UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM 10-K
(Mark One)
☒
ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934
For the fiscal year ended December 31, 2019
OR
☐
TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934
For the transition period from to
Commission File Number 001-38360
Solid Biosciences Inc.
(Exact name of Registrant as specified in its Charter)
Delaware
(State or other jurisdiction of
incorporation or organization)
141 Portland Street, Fifth Floor
Cambridge, MA
(Address of principal executive offices)
90-0943402
(I.R.S. Employer
Identification No.)
02139
(Zip Code)
Registrant’s telephone number, including area code: (617) 337-4680
Securities registered pursuant to Section 12(b) of the Act:
Title of each class
Common Stock $0.001 par value per share
Trading Symbol
SLDB
Name of exchange on which registered
The Nasdaq Global Select Market
Securities registered pursuant to Section 12(g) of the Act:
None
(Title of class)
Indicate by check mark if the registrant is a well-known seasoned issuer, as defined in Rule 405 of the Securities Act. YES ☐ NO ☒
Indicate by check mark if the registrant is not required to file reports pursuant to Section 13 or Section 15(d) of the Act. YES ☐ NO ☒
Indicate by check mark whether the Registrant: (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities Exchange Act of 1934 during the preceding 12 months (or for such shorter period
that the Registrant was required to file such reports), and (2) has been subject to such filing requirements for the past 90 days. YES ☒ NO ☐
Indicate by check mark whether the Registrant has submitted electronically every Interactive Data File required to be submitted pursuant to Rule 405 of Regulation S-T (§232.405 of this chapter) during the
preceding 12 months (or for such shorter period that the Registrant was required to submit such files). YES ☒ NO ☐
Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, a smaller reporting company, or an emerging growth company. See the definitions of “large
accelerated filer,” “accelerated filer,” “smaller reporting company,” and “emerging growth company” in Rule 12b-2 of the Exchange Act.
Large accelerated filer
Non-accelerated filer
☐
☐
Accelerated filer
Smaller reporting company
Emerging growth company
☒
☒
☒
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided
pursuant to Section 13(a) of the Exchange Act. ☒
Indicate by check mark whether the Registrant is a shell company (as defined in Rule 12b-2 of the Act). YES ☐ NO ☒
As of June 28, 2019, the last business day of the Registrant’s most recently completed second fiscal quarter, the aggregate market value of the Registrant’s common stock held by non-affiliates was
$100.9 million, based on the last reported sale price of such stock on the Nasdaq Global Select Market as of such date.
The number of shares of Registrant’s common stock outstanding as of February 15, 2020 was 46,068,049.
Table of Contents
PART I
Item 1.
Item 1A.
Item 1B.
Item 2.
Item 3.
Item 4.
Business
Risk Factors
Unresolved Staff Comments
Properties
Legal Proceedings
Mine Safety Disclosures
PART II
Item 5.
Item 6.
Item 7.
Item 7A.
Item 8.
Item 9.
Item 9A.
Item 9B.
Market for Registrant’s Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities
Selected Financial Data
Management’s Discussion and Analysis of Financial Condition and Results of Operations
Quantitative and Qualitative Disclosures About Market Risk
Financial Statements and Supplementary Data
Changes in and Disagreements With Accountants on Accounting and Financial Disclosure
Controls and Procedures
Other Information
PART III
Item 10.
Item 11.
Item 12.
Item 13.
Item 14.
Directors, Executive Officers and Corporate Governance
Executive Compensation
Security Ownership of Certain Beneficial Owners and Management and Related Stockholder Matters
Certain Relationships and Related Transactions, and Director Independence
Principal Accounting Fees and Services
PART IV
Item 15.
Item 16.
Exhibits, Financial Statement Schedules
Form 10-K Summary
SIGNATURES
CONSOLIDATED FINANCIAL STATEMENTS
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F-2
CAUTIONARY NOTE REGARDING FORWARD-LOOKING STATEMENTS
This Annual Report on Form 10-K includes forward-looking statements, which involve risks and uncertainties. These forward-looking statements
can be identified by the use of forward-looking terminology, including the terms “believe,” “estimate,” “project,” “anticipate,” “expect,” “seek,” “predict,”
“aim,” “continue,” “possible,” “intend,” “may,” “might,” “will,” “could,” “would” or “should” or, in each case, their negative, or other variations or
comparable terminology. These forward-looking statements include all matters that are not historical facts. They appear in a number of places throughout
this Annual Report on Form 10-K. We derive many of our forward-looking statements from our operating budgets and forecasts, which are based upon
many detailed assumptions. While we believe that our assumptions are reasonable, we caution that it is very difficult to predict the impact of known
factors, and, of course, it is impossible for us to anticipate all factors that could affect our actual results. All forward-looking statements are based upon
information available to us on the date of this Annual Report on Form 10-K.
The forward-looking statements in this Annual Report on Form 10-K include, among other things, statements about:
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the timing, progress and results of preclinical studies and clinical trials for SGT-001 and our other product candidates;
our ability to resolve the clinical hold on SGT-001 and the requirements for and timing of any such resolution;
our ability to obtain and maintain U.S. regulatory approval of SGT-001, and the timing and scope thereof;
our ability to obtain and maintain foreign regulatory approvals, and the timing and the scope thereof;
the size of the patient populations and potential market opportunity for SGT-001 and our other product candidates, if approved for commercial
use;
our manufacturing capabilities and strategy, including the scalability and commercial viability of our manufacturing methods and processes;
our plans to develop and commercialize SGT-001 and our other product candidates, if approved;
the pricing and reimbursement of SGT-001 and any other product candidates we may develop, if approved;
the establishment of sales, marketing and distribution capabilities and entry into agreements with third parties to market and sell SGT-001 or
our other product candidates, if approved;
the rate and degree of market acceptance and clinical utility of SGT-001 and any other product candidates we may develop and for which we
may receive approval;
our expectations related to our use of capital resources;
our estimates regarding expenses, ongoing losses, future revenue, capital requirements and need for and ability to obtain additional financing;
our intellectual property position;
our competitive and market position;
developments relating to our competitors and our industry; and
the impact of laws and regulations on our operations.
By their nature, forward-looking statements involve risks and uncertainties because they relate to events and depend on circumstances that may or
may not occur in the future. We caution you that forward-looking statements are not guarantees of future performance and that our actual results of
operations, financial condition, business and prospects may differ materially from those made in or suggested by the forward-looking statements contained
in this Annual Report on Form 10-K. In addition, even if our results of operations, financial condition, business and prospects are consistent with the
forward-looking statements contained in this Annual Report on Form 10-K, those results may not be indicative of results in subsequent periods.
You should read this Annual Report on Form 10-K completely and with the understanding that our actual future results may be materially different
from what we expect. We qualify all of our forward-looking statements by these cautionary statements.
As used in this Annual Report on Form 10-K, the terms “Solid,” “the Company,” “we,” “us” and “our” refer to Solid Biosciences Inc. unless the
context indicates otherwise.
ii
Item 1.
Business.
Overview
PART I
Our mission is to cure Duchenne muscular dystrophy, or DMD, a genetic muscle-wasting disease predominantly affecting boys, with symptoms that
usually manifest between three and five years of age. DMD is a progressive, irreversible and ultimately fatal disease that affects approximately one in every
3,500 to 5,000 live male births and has an estimated prevalence of 10,000 to 15,000 cases in the United States alone. DMD is caused by mutations in the
dystrophin gene, which result in the absence or near-absence of dystrophin protein. Dystrophin protein works to strengthen muscle fibers and protect them
from daily wear and tear. Without functioning dystrophin and certain associated proteins, muscles suffer excessive damage from normal daily activities and
are unable to regenerate, leading to the build-up of fibrotic, or scar, and fat tissue. There is no cure for DMD and, for the vast majority of patients, there are
no satisfactory symptomatic or disease-modifying treatments. Our efforts are focused on our lead product candidate, SGT-001, a gene transfer candidate
under investigation for its ability to drive functional dystrophin protein expression in patients’ muscles and improve the course of the disease. Based on our
preclinical program that included multiple animal species of different phenotypes and genetic variations, as well as preliminary clinical trial biomarker
results, we believe that SGT-001, has the potential to slow or even halt the progression of DMD, regardless of the type of genetic mutation or stage of the
disease.
For patients suffering from DMD, symptoms usually begin to manifest between three and five years of age, when they fail to reach developmental
milestones or experience motor function challenges, such as difficulty walking or climbing stairs. As the disease progresses, patients with DMD experience
frequent falls; can no longer run, play sports or perform most daily functions; and are further weakened by physical activity. By their early teens, DMD
patients typically lose their ability to walk and ultimately become dependent on a wheelchair for mobility. By their 20s, patients essentially become
paralyzed from the neck down and require a ventilator to breathe. Though disease severity and life expectancy vary, a DMD patient’s quality of life
dramatically decreases over time, with death typically occurring by early adulthood from either cardiac or respiratory complications.
Our founders, who are personally touched by the disease, created a biotechnology company purpose-built to accelerate the discovery and
development of meaningful therapies for all patients affected by DMD. Through this disease-focused business model, our research team, led by experts in
DMD biology and drug development, along with key opinion leaders in DMD, continuously evaluate emerging science to identify high-potential product
candidates. Our selection process includes extensive diligence and initial pharmacology research with highly specific, predefined criteria, which provide us
with confidence in our development program decisions. Through this data-driven selection process, we have evaluated a number of programs and identified
gene therapy as a potentially beneficial approach for DMD, and thus initiated development of our lead product candidate SGT-001.
Our product candidates
SGT-001 is our lead gene transfer candidate and the focus of our research and development efforts. Gene transfer, a type of gene therapy, is
designed to address diseases caused by mutated genes through the delivery of functional versions of those genes, called transgenes. The transgenes are then
utilized by the body to produce proteins that are absent or not functional prior to treatment, potentially offering long-lasting beneficial clinical effects. SGT-
001 is designed to address the underlying genetic cause of DMD by delivering a synthetic transgene that produces dystrophin-like protein that is only
expressed in muscles of the body, including cardiac and respiratory muscles. Our SGT-001 vector is derived from a naturally occurring, non-pathogenic
virus called adeno-associated virus, or AAV, which was selected for its ability to efficiently enter skeletal, diaphragm and cardiac muscle tissues. The vector
is designed to carry a synthetic dystrophin transgene construct, called microdystrophin, that retains the most critical components of the full-size dystrophin
gene yet is small enough to fit within AAV packaging constraints. These components not only include the domains necessary to confer protection against
muscle damage, but also to drive the expression of key dystrophin associated proteins, such as neuronal Nitric Oxide Synthase, or nNOS. Inclusion of this
nNOS coding region of the dystrophin protein may result in microdystrophin protein that has unique activity, restore nitric oxide production in the muscle
and potentially provide important functional benefits such as diminished muscle fatigue and protection against ischemic muscle damage. In our
Investigational New Drug Application-, or IND, enabling preclinical program, we have studied the efficacy, safety and durability of SGT-001 in multiple
preclinical models and its functional benefits in DMD animal studies. In contrast to other therapeutic approaches that are designed to target specific
mutations in the dystrophin gene, we believe SGT-001 is a mutation agnostic approach.
SGT-001 has been granted Rare Pediatric Disease Designation, or RPDD, and Fast Track Designation, in the United States and Orphan Drug
Designations in both the United States and European Union. The safety and efficacy of SGT-001 are under evaluation in a randomized, controlled, open-
label, single-ascending dose Phase I/II clinical trial called IGNITE DMD, which is currently on clinical hold.
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In the fourth quarter of 2017, we initiated IGNITE DMD to evaluate SGT-001 in ambulatory and non-ambulatory males with DMD aged four to 17
years. The primary objectives of IGNITE DMD are to assess the safety and tolerability of SGT-001, as well as efficacy as defined by SGT-001
microdystrophin protein expression. The clinical trial is also designed to assess other parameters of muscle function and mass, respiratory and
cardiovascular function, serum and muscle biomarkers associated with SGT-001 microdystrophin production, SGT-001 microdystrophin associated
biochemical properties (e.g. nNOS binding) and patient and parent reported outcomes and quality of life measures, among other endpoints. In February
2019, we announced preliminary findings based on three-month biopsy data from the first three patients dosed with 5E13 vg/kg of SGT-001, the lowest
dose outlined in the trial protocol. In one patient, SGT-001 microdystrophin was detected via western blot below the five percent level of quantification of
the assay and in approximately 10 percent of fibers via immunofluorescence. Due to these findings, and in consultation with the Data Safety Monitoring
Board, or DSMB, in May 2019 we announced that we had initiated dosing of the next cohort of patients at 2E14 vg/kg. In August 2019, we amended our
protocol to remove the matched patient control arm for the rest of the 2E14 vg/kg cohort in the IGNITE DMD trial and to provide measures to potentially
improve safety, such as initially proceeding to dose patients weighing 25 kg or less.
In November 2019, IGNITE DMD was placed on clinical hold by the U.S. Food and Drug Administration, or FDA, as a result of a serious adverse
event, or SAE, experienced by the third patient dosed in the 2E14 vg/kg cohort. The SAE, deemed related to the study drug, was characterized by
complement activation, thrombocytopenia, a decrease in red blood cell count, acute kidney injury, and cardio-pulmonary insufficiency. Neither cytokine-
nor coagulopathy-related abnormalities were observed. In December 2019, we reported that the SAE was fully resolved and the patient had resumed his
normal activities. We continue to make progress to address the clinical hold and determine the path forward for SGT-001.
In December 2019, we announced preliminary findings based on three-month biopsy data from the first two patients dosed with 2E14 vg/kg of
SGT-001. Using two independent immunofluorescence assays, 10% to 20% of microdystrophin positive muscle fibers were determined to express SGT-001
microdystrophin in the fourth patient and 50% to 70% of microdystrophin positive muscle fibers in the fifth patient. Immunofluorescence also showed clear
stabilization and co-localization of nNOS and beta-sarcoglycan with SGT-001 microdystrophin in both patients. Using western blot, the expression levels
for the fourth patient were detectable and estimated to be near the assay’s level of quantification which is 5% of non-dystrophic control samples, with one
assay replicate at 5.5%. Expression for the fifth patient was 17.5% of normal control samples. The levels of serum creatine kinase, a highly variable
biochemical marker of muscle damage, declined from baseline in both patients.
In March 2020, we announced data from the third patient dosed in the 2E14 vg/kg dose cohort of IGNITE DMD, including three-month biopsy
data. Using immunofluorescence assays, 50% to70% of the muscle fibers were determined to express SGT-001 microdystrophin. Immunofluorescence also
showed stabilization and co-localization of nNOS and beta-sarcoglycan with SGT-001 microdystrophin. Using western blot, microdystrophin expression
was 8% of normal control samples. In addition, the level of serum creatine kinase decreased from baseline.
We believe the unique functionality of our SGT-001 microdystrophin may result in functional benefits including diminished muscle fatigue and
protection against ischemic muscle damage, which can lead to loss of functional muscle. Collectively, we believe these data provide evidence supporting
the biological activity of SGT-001 and provide support for continued development.
Taking into account the prevalence and incidence of DMD and the anticipated dosing requirements for gene transfer, we anticipate that there will be
a need for a substantial supply of SGT-001 for clinical trials and, if approved, for commercial markets. Through significant targeted investments to address
this challenge, we have developed a manufacturing process that we believe can scale to adequately support our needs for clinical trials, commercial launch
and beyond. Our in-house scientists are continuing to work to increase the productivity, efficiency and purity of our manufacturing process.
In conjunction with our development of SGT-001, we believe it is critical to continue to invest time and resources in tools and technologies
designed to help us more effectively understand DMD, accurately monitor disease progression and assist patients in daily life. As part of this goal, we are
developing biomarkers and sensors that may allow us to identify treatment targets faster, measure the therapeutic impact of potential product candidates
better and reach decision points earlier.
We seek to protect our proprietary and intellectual property position through a combination of patents, trade secret laws, proprietary know-how,
continuing technological innovation, and entering into non-disclosure, confidentiality and invention assignment agreements. For our gene transfer
programs, we have exclusively licensed four issued U.S. patents, and nine issued patents and ten pending patent applications in foreign jurisdictions. We
have filed eight pending U.S. patent applications, two pending PCT international patent applications, and one pending patent application in Taiwan. We
intend to continue building out our intellectual property protection to further strengthen our position in the DMD field.
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Who we are
Solid Biosciences was founded in 2013 by our Chief Executive Officer, Ilan Ganot, our Chairman of the Board, Andrey Zarur, our former President,
Gilad Hayeem, our board member Matthew Arnold and our Head of Patient Advocacy Annie Ganot, with the goal of developing meaningful therapies for
patients with DMD. Solid is the English translation of Eytani, the Hebrew name of Ilan and Annie Ganot’s son, who was diagnosed with the disease in
2012. Our founders, unsatisfied with the existing therapeutic landscape, proceeded to raise funds to execute on our disease-focused business model. We
assembled a passionate management team and scientific advisory board composed of individuals with extensive experience in DMD, gene therapy, product
discovery, research and development, manufacturing, business strategy and finance.
In 2015, we began exclusively licensing the elements of the construct for SGT-001 and other elements of our gene transfer program from the
University of Michigan, the University of Missouri and the University of Washington. Since then, we have continued to use our extensive network across
the academic, business and patient communities to identify, vet and pursue high-potential complementary product candidates to address the needs of DMD
patients.
Since our inception, we have raised private capital from a group of top-tier corporate and private investors. In addition, three leading U.K.-based
DMD charities provided initial seed funding for our gene transfer program in return for equity in our company, and we have accepted additional
contributions from several DMD charities to fund our early-stage research programs. In January 2018, we completed our initial public offering resulting in
net proceeds of $129.1 million, after deducting underwriting discounts and commissions and offering expenses. In July 2019, we completed a private
placement of shares of our common stock and pre-funded warrants to purchase shares of our common stock resulting in net proceeds to us of $57.9 million,
after deducting offering costs.
We operated as a Delaware limited liability company under the name Solid Biosciences, LLC until immediately prior to the effectiveness of our
registration statement on Form S-1 on January 25, 2018, at which time we converted into a Delaware corporation pursuant to a statutory conversion and
changed our name to Solid Biosciences Inc.
Mission
Our mission, which guides every aspect of our operations, is to cure DMD. Underscoring this mission, our disease-focused business model is
founded on the following fundamental values:
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identify and develop meaningful therapies for all patients with DMD;
bring together the leading experts in DMD, science, technology, disease management and care; and
be guided by the needs of DMD patients.
About Duchenne muscular dystrophy
DMD is an X-chromosome-linked, muscle-wasting disease, predominantly affecting boys. Progressive, irreversible and ultimately fatal, DMD
occurs in approximately one in every 3,500 to 5,000 live male births and has an estimated prevalence of 10,000 to 15,000 cases in the United States alone.
In DMD, mutations in the dystrophin gene result in the body’s inability to produce functioning dystrophin protein, which works to strengthen muscle fibers
and protect them from daily wear and tear. Dystrophin protein also serves as the cornerstone of the dystrophin glycoprotein complex, or DGC, a group of
proteins that links the inner and outer components of muscle cells to ensure proper muscle function.
Without dystrophin and the DGC, muscles suffer excessive damage from normal daily activities and are unable to regenerate, leading to the build-
up of scar and fat tissue. More than 1,000 dystrophin gene mutations, which can be inherited or can occur spontaneously, have been identified in people
with DMD.
For patients suffering from DMD, symptoms usually begin to manifest between three and five years of age, when they fail to reach developmental
milestones or experience motor function challenges, such as difficulty walking or climbing stairs. Muscle wasting initially presents in the legs and pelvic
area, then in the muscles of the shoulders, neck and arms. As the disease progresses, patients with DMD experience frequent falls, can no longer run, play
sports or perform most daily functions, and are further weakened by physical activity. In addition to physical challenges, DMD also commonly involves
cognitive difficulties and behavioral challenges.
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By their early teens, DMD patients typically lose their ability to walk and become dependent on a wheelchair for mobility. By their 20s, patients
essentially become paralyzed from the neck down and require a ventilator to breathe. Though disease severity and life expectancy vary, a patient’s quality
of life dramatically decreases over time, with death typically occurring by early adulthood from either cardiac or respiratory complications.
Need for effective therapies
There is no cure for DMD and, for the vast majority of patients, there are no satisfactory symptomatic or disease-modifying treatments.
Glucocorticoid treatment, the current standard-of-care, has been shown to temporarily improve muscle strength, prolong the period of ambulation
and slow the progression of DMD. However, glucocorticoid use is associated with well-known adverse events, such as severe weight gain, stunted growth,
weakening of bone structure and metabolic dysfunctions, among others. The most commonly used glucocorticoids include prednisone and deflazacort
(EMFLAZA).
In recent years, certain regulators have conditionally approved three new therapies which target specific mutations in the dystrophin gene. These
therapies are indicated for only a small portion of the DMD patient population, and their respective efficacy profiles still need to be fully understood.
Current best practices for treating DMD patients also dictate a multidisciplinary approach to disease management, which includes physical and
occupational therapy to preserve strength, function and flexibility, orthopedic management to reduce the risk of scoliosis and other bone and joint problems,
pulmonary, cardiac and gastrointestinal management, and psychosocial management to support behavior and learning.
Burden of disease
Despite recent therapeutic advances, DMD represents a significant societal and economic burden. The economic burden, estimated at $1.2 billion
annually in the United States (excluding costly mortality and end-of-life care expenses), includes costs associated with hospital admissions, medication,
frequent doctor visits and investment in assistive devices, as well as indirect costs related to productivity losses for the caregivers and costs due to pain,
anxiety and social handicap. Of this amount, approximately 45% is represented by indirect costs. Only a small proportion of DMD patients are employed
and many caregivers reduce their hours or stop working altogether to care for their children, who progressively require more help with everyday tasks, such
as eating, dressing and using the bathroom. In some cases, patients also experience serious mental health issues that require additional support and
treatment.
Gene transfer—A corrective therapy
Gene therapy is a therapeutic approach that aims to address diseases caused by gene mutations. A gene is a portion of deoxyribonucleic acid, or
DNA, that provides the instructions for the body to construct proteins that perform functions needed for life. Genes are prone to mutations, which can either
be inherited or occur spontaneously. While many mutations are harmless, some lead to the absence of crucial proteins, resulting in serious genetic diseases
like DMD.
Gene transfer, a type of gene therapy, is designed to address diseases caused by mutated genes through the delivery of functional versions of those
genes, called transgenes. The transgenes are then utilized by the body to produce proteins that are absent or not functional prior to treatment, potentially
offering long-lasting beneficial effects.
We have focused our efforts on gene transfer because we believe it has the greatest potential to address the root cause of DMD: the absence or near-
absence of dystrophin protein. If successful, we believe gene transfer can slow or stop the progression of DMD in a majority of patients, irrespective of
their genetic mutation, by producing long-term, muscle-specific expression of a functional dystrophin-like protein.
Our gene transfer candidate, or vector, includes three components:
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a viral capsid—a protein shell utilized as a vehicle to deliver a transgene to cells in the body;
a transgene—a functional gene intended to produce a functional protein; and
a promoter—a specialized DNA sequence that directs cells to produce the protein in specific tissues.
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SGT-001
SGT-001, our lead gene transfer candidate, is designed to preserve muscle function in DMD patients after a single administration and is based on
some of the most recent understanding of dystrophin biology in the field. The SGT-001 vector is comprised of a functional transgene and a muscle-specific
promoter, which are delivered via an AAV capsid. We believe that the SGT-001 construct is differentiated from other gene transfer candidates and may
provide unique clinical benefit.
The vector is modified to no longer self-replicate, yet retains its ability to effectively introduce new genetic material directly into patients’ cells.
AAV vectors have been extensively studied in human clinical trials in multiple disease indications, including in clinical trials of high-dose, systemically
delivered AAV gene therapies being conducted by third parties.
Capsid: The capsid of the SGT-001 vector is derived from a naturally occurring, non-pathogenic virus called AAV. There are several subtypes of
AAV capsids that differ based on the proteins that make up their structure. These capsids have affinities for different sites in the body. We selected the
AAV9 serotype capsid for clinical development based on our preclinical data, which demonstrated the capsid’s ability to efficiently enter skeletal,
diaphragm and cardiac muscle tissues, as well as its favorable tolerability reported in other gene transfer clinical programs.
Transgene: Dystrophin, the largest gene in the body, exceeds the carrying capacity of AAV vectors. To overcome this challenge, we advanced
development of the SGT-001 transgene, a synthetic, dystrophin-like gene that fits into AAV and has the ability to drive functional protein expression in
skeletal, diaphragm and cardiac muscle tissue.
The concept of a modified therapeutic dystrophin gene originated from research on Becker muscular dystrophy, or BMD, where researchers
discovered that certain BMD patients had mutations in the dystrophin gene that drove expression of a functional form of dystrophin protein, allowing
patients to live relatively normal lives. This discovery led scientists to engineer a number of synthetic, dystrophin transgene constructs, called
microdystrophins, that retained only the most critical components of the full-size dystrophin gene yet were small enough to fit within AAV packaging
constraints. There are several types of microdystrophins that differ based on the configuration of their components. Microdystrophins were subsequently
demonstrated to functionally protect muscle in mouse models of DMD.
Our SGT-001 microdystrophin construct is based on three decades of development and optimization work at the University of Missouri and the
University of Washington as well as other academic institutions. In preclinical studies, the laboratories of Jeffrey Chamberlain, Ph.D., from the University
of Washington, and Dongsheng Duan, Ph.D., from the University of Missouri, identified a proprietary configuration of genetic components that, when
administered systemically, produces functional microdystrophin protein expression that not only stabilizes muscle membranes and protects muscle against
injury, but also simultaneously restores the localization of DGC to the muscle membrane, notably increasing neuronal nitric oxide synthase, or nNOS,
concentration. In subsequent published studies, Drs. Duan and Chamberlain demonstrated in animal models that, in comparison to earlier configurations,
nNOS-restoring microdystrophins were more effective in improving muscle function and resistance to fatigue. We believe the unique functionality of our
SGT-001 microdystrophin may result in functional benefits including diminished muscle fatigue and protection against ischemic muscle damage, which can
lead to loss of functional muscle.
Promoter: The expression of the SGT-001 microdystrophin transgene is regulated by a modified, synthetic muscle-specific promoter cassette called
CK8, which is derived from the naturally occurring muscle creatine kinase promoter. Regulatory cassettes, such as CK8, are used to prompt gene
expression specifically in muscle tissues. In comparison to other regulatory cassettes, we chose CK8 due to its small size and its ability to drive
microdystrophin transgene expression in skeletal, diaphragm and cardiac muscle tissues. In our preclinical studies in small and large animal models, CK8
restricted microdystrophin transgene expression to these muscles.
SGT-001 preclinical program
Our comprehensive preclinical program for SGT-001 was comprised of studies that inform efficacy, durability and safety, as well as dose response
and the kinetics of transgene expression. Our program includes three different animal species: mice, dogs and non-human primates, or NHPs. Our
preclinical studies were performed by third-party collaborators.
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Well-established mouse and dog disease models for DMD offered us the opportunity to better evaluate the potential translatability of SGT-001 to
humans. While studies in dystrophic mice, such as the mdx mouse, provide important efficacy rationale, we chose to perform additional functional studies
in dystrophic dogs because they exhibit a more severe dystrophic phenotype and progress similarly to human patients at earlier stages of the disease. Dog
models enabled us to assess various endpoints, including biodistribution, expression, durability and function in a large animal species.
Because DMD is a disease defined by a lack of dystrophin protein, it is important to reliably detect microdystrophin expression in muscle after
SGT-001 treatment. As part of our core preclinical program, we developed well-characterized and well-recognized analytic approaches to confirm
transgene expression and localization, using the following assays:
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Immunofluorescence: A qualitative method to determine if a transgene is expressed and localized to muscle membrane.
Western blot: A recognized method to quantify dystrophin expression.
Mass spectrometry: A highly sensitive analytical method to quantify transgene expression.
We also employed immunofluorescence to confirm if our microdystrophin construct restored the DGC, including key proteins such as sarcoglycan
and nNOS.
Efficacy in dystrophic mice
Multiple studies in both dystrophic, or mdx, and healthy, or wild-type, mice have demonstrated that a single intravenous administration of SGT-001
induces measurable levels of microdystrophin protein expression. Muscle strength assessments in mild and severe mouse models of DMD demonstrated
functional improvements in animals treated with SGT-001 in comparison to untreated dystrophic controls. In all studies, microdystrophin protein
expression was measured using immunofluorescence, western blot and mass spectrometry.
In an mdx dose-response study, a clear dose-dependent pattern of transgene expression was observed at day 28 by all three assays. As an example,
at a dose of 1E14 vg/kg, transgene expression as quantified by positive immunofluorescence staining in the quadriceps and heart muscle tissues was 50%
and 80%, respectively, of the full-length dystrophin levels quantified in healthy wild-type control muscles. Similar levels of microdystrophin expression
were found in all mdx studies completed to date. Efficacy studies performed in dystrophic mice treated with SGT-001 demonstrated significant, dose-
responsive improvements in both muscle morphology and multiple physiological parameters. In a blinded efficacy study performed in mdx mice dosed at
approximately six weeks of age, SGT-001 treatment showed statistically significant improvements in multiple muscle strength parameters as well as
resistance to treadmill-induced fatigue.
Efficacy in dystrophic dogs
Two independent studies in dystrophic dogs assessed durability of microdystrophin expression and efficacy, respectively. These studies were
performed in two distinct dystrophic dog models (mixed breed dystrophic dogs, or cDMD, and Golden Retriever Muscular Dystrophy, or GRMD),
collectively encompassing a number of genetic mutations that lead to the absence of dystrophin protein. This enabled us to assess SGT-001 across multiple
mutations, which is more reflective of the composition of the DMD patient population. Both studies used a canine-optimized version of the
microdystrophin gene.
In a long-term dose-ranging study, five three-month-old, juvenile cDMD dogs received an intravenous dose of either 5E13 vg/kg (n=1), 1E14 vg/kg
(n=2), 3E14 vg/kg (n=1) or 5E14 vg/kg (n=1). Robust transgene expression was detected by immunofluorescence at all biopsy time points and at all of the
dose levels. In animals dosed with 1E14 vg/kg, approximately 70-90% of the muscle fibers were positive for microdystrophin and correlated to the
restoration of DGC associated proteins, including nNOS.
A blinded dose-ranging study in the GRMD model assessed the general safety and efficacy of the canine construct of SGT-001. The three dose
levels (1E13, 1E14 and 2E14 vg/kg) were administered at three months of age and animals were followed for three months following administration.
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Dose-dependent transgene expression was detected in interim biopsies of skeletal muscles at day 28 and 45 and at the end of the study at day 91 in
skeletal, diaphragm and cardiac muscles. A blinded histological evaluation of the muscle tissue revealed a reduction of dystrophic pathology at the higher
dose levels. In the mid- and high-dose groups, all muscles biopsied at the end of the study exhibited improved pathology compared to low dose and
untreated controls. Biodistribution studies demonstrated dose dependent transgene expression that was only detectable in the muscle tissues.
The observed dose response was detectable by both immunofluorescence and western blot. Quantification by western blot averaged less than 10%
of wild-type in the low-dose (1E13 vg/kg) animals. In the mid-dose animals, the level of expression among the skeletal muscles ranged from an average of
approximately 20% to approximately 50% of wild-type control muscles. At 2E14 vg/kg, the level of expression ranged from 30% to 70% of wild-type
dystrophin. This data also correlates to quantification of microdystrophin via mass spectrometry.
Dose-dependent, sustained expression of microdystrophin not only correlated with histological improvements in muscle, but also provided
statistically significant improvements in measures of muscle function. At day 90, muscle force generation was improved in both the 1E14 vg/kg and 2E14
vg/kg cohorts, indicating that the microdystrophin produced by SGT-001 is highly protective in a large animal dystrophic species.
The efficacy data collectively described above in both dystrophic mouse and dog models was incorporated into an overall nonclinical model to
inform dose selection for our clinical program. All doses were well tolerated and there was no observed immune response to the transgene.
Manufacturing comparability
As part of our manufacturing process development, we have run comparability studies at each stage of our process scale-up. These comparability
studies were carried out using in vivo mouse models to ensure that our drug product produced at different scales is comparable to each other.
Safety
As part of our preclinical program, we performed necessary good laboratory practices, or GLP, toxicology studies to establish the overall safety
profile of SGT-001 in wild-type mice and NHPs. The data and our conclusions from these studies were included in our IND submission to the
FDA. Systemic administration of SGT-001 was generally well tolerated in both species. We observed no evidence of test-article-related toxicity for up to 13
weeks after systemic administration of SGT-001 in either species that would prevent us from initiating clinical trials. In the NHP study, test-article-related
effects were self-limited, mild chemistry and hematology changes with no microscopic correlates at the end of the study. There was a transient and
asymptomatic increase in liver function enzymes observed in NHPs starting on day 9, which returned to normal levels by day 21. We believe there were no
other relevant test-article-related adverse events associated with SGT-001 administration in either GLP study. In the NHP toxicology study, a single animal
from the high dose cohort was euthanized after it did not recover from an anesthetic procedure. We believe this event was attributed to procedural
errors. However, AAV vector cannot be completely ruled out as a contributing factor to the toxicity that gave rise to the event.
Clinical development of SGT-001
We are developing SGT-001 for the treatment of DMD through a single intravenous administration. In the fourth quarter of 2017, we announced the
initiation of IGNITE DMD, a randomized, controlled, open-label, single-ascending dose Phase I/II clinical trial designed to evaluate SGT-001 in
ambulatory and non-ambulatory males with DMD aged four to 17 years. The primary objectives of IGNITE DMD are to assess the safety and tolerability
of SGT-001, as well as efficacy as defined by microdystrophin protein expression. The clinical trial is also designed to assess muscle function and mass,
respiratory and cardiovascular function, serum and muscle biomarkers associated with microdystrophin production, SGT-001 microdystrophin associated
biochemical properties (e.g., nNOS binding), functional outcome, patient and parent reported outcomes and quality of life measures, among other
endpoints. Key inclusion criteria include: established clinical diagnosis of DMD and documented dystrophin gene mutation predictive of DMD phenotype;
anti-AAV9 antibodies below pre-specified thresholds; stable cardiac and pulmonary function; and a stable daily dose of oral corticosteroids for 24 weeks.
There is no enrollment restriction in the clinical trial protocol based on a patient’s underlying dystrophin gene mutation.
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In the current IGNITE DMD protocol, participants are all assigned to open label treatment with SGT-001. The selection of the original starting dose
in our first cohort, 5E13 vg/kg, was based on safety and efficacy data observed in our preclinical studies. Dose escalation and decisions regarding clinical
trial progression occur after review by the DSMB. Efficacy is being assessed by comparing microdystrophin protein expression in muscle biopsy before
and 12 months after treatment for each patient. An intermediate biopsy at either 45 days, three months, six months or nine months will inform the time
course of microdystrophin expression. Long-term follow up will continue per regulatory guidelines.
In February 2019, we announced preliminary findings based on three-month biopsy data from the first three patients dosed with 5E13 vg/kg of
SGT-001, the lowest dose outlined in the trial protocol. In one patient, SGT-001 microdystrophin was detected via western blot below the five percent level
of quantification of the assay and in approximately 10 percent of fibers via immunofluorescence. Due to these findings, and in consultation with the Data
Safety Monitoring Board, or DSMB, in May 2019 we announced that we had initiated dosing of the next cohort of patients at 2E14 vg/kg. To date we have
dosed 3 patients with the 2E14 vg/kg dose. In August 2019, we amended our protocol to remove the matched patient control arm for the rest of the 2E14
vg/kg cohort in the IGNITE DMD trial and to provide measures to potentially improve safety, such as initially proceeding to dose patients weighing 25 kg
or less.
In November 2019, IGNITE DMD was placed on clinical hold by the FDA as a result of an SAE experienced by the third patient dosed in the 2E14
vg/kg cohort. The SAE, deemed related to the study drug, was characterized by complement activation, thrombocytopenia, a decrease in red blood cell
count, acute kidney injury, and cardio-pulmonary insufficiency. Neither cytokine- nor coagulopathy-related abnormalities were observed. In December
2019, we reported that the SAE was fully resolved and the patient had resumed his normal activities. We continue to make progress to address the clinical
hold and determine the path forward for SGT-001.
In December 2019, we announced preliminary findings based on three-month biopsy data from the first two patients dosed with 2E14 vg/kg of
SGT-001. Using two independent immunofluorescence assays, 10% to 20% of microdystrophin positive muscle fibers were determined to express SGT-001
microdystrophin in the fourth patient and 50% to 70% of microdystrophin positive muscle fibers in the fifth patient. Immunofluorescence also showed clear
stabilization and co-localization of nNOS and beta-sarcoglycan with SGT-001 microdystrophin in both patients. Using western blot, the expression levels
for the fourth patient were detectable and estimated to be near the assay’s level of quantification which is 5% of non-dystrophic control samples, with one
assay replicate at 5.5%. Expression for the fifth patient was 17.5% of normal control samples. The levels of serum creatine kinase, a highly variable
biochemical marker of muscle damage, declined from baseline in both patients.
In March 2020, we announced data from the third patient dosed in the 2E14 vg/kg dose cohort of IGNITE DMD, including three-month biopsy
data. Using immunofluorescence assays, 50% to 70% of the muscle fibers were determined to express SGT-001 microdystrophin. Immunofluorescence also
showed stabilization and co-localization of nNOS and beta-sarcoglycan with SGT-001 microdystrophin. Using western blot, microdystrophin expression
was 8% of normal control samples. In addition, the level of serum creatine kinase decreased from baseline.
We believe the unique functionality of our SGT-001 microdystrophin may result in functional benefits including diminished muscle fatigue and
protection against ischemic muscle damage, which can lead to loss of functional muscle. Collectively, we believe these data provide evidence supporting
the biological activity of SGT-001 and provide support for continued development. Based on data from the clinical trial, we will determine next steps for
SGT-001 clinical development, including additional clinical trials that may include other patient populations, as well as the need for larger confirmatory
clinical trials.
Manufacturing SGT-001
The prevalence and incidence of DMD, combined with average patient weight and anticipated dosing requirements for SGT-001, result in a
substantial supply need for clinical trials and, if approved, for commercial markets. To address this challenge, we developed a manufacturing process that
we believe will be scalable to support clinical and commercial production needs for SGT-001.
Our suspension-based process is founded on seminal work by scientists at the University of Florida and has been optimized for manufacturability
by our internal process development scientists with the support of our Contract Development Manufacturing Organization, or CDMO, partners. The
process consists of three steps. First, we produce two replication-incompetent Herpes Simplex Virus, or HSV, stocks, one containing our microdystrophin
construct and the other containing the critical elements of the AAV9. Second, we then use these two HSV stocks to coinfect suspension-adapted human
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embryonic kidney cells (HEK-293). Third, these cells are then purified and concentrated in our downstream process to produce our gene transfer candidate.
Our team has developed the analytical testing methods needed to support consistency and strict standards of quality and potency. We believe that this
approach will increase our speed of development, ensure consistent quality and regulatory compliance, and reduce the risk of delay or unexpected
production costs.
We believe that our decision to invest early in our manufacturing process was key to developing a scalable process designed to support clinical
development and potential commercialization. We are supplying our clinical development program for SGT-001 with drug product produced at current
good manufacturing practices, or cGMP, - compliant facilities located at partner CDMOs. We have operated at 250-liter scale and have successfully
produced multiple drug product batches at this scale. Our in-house scientists are continuing to work to increase the productivity, efficiency and purity of
our manufacturing process.
Complementary disease-modifying therapy: Anti-LTBP4
While we believe gene transfer may be able to slow or halt DMD disease progression, many patients would still suffer from the manifestations of
the disease, such as tissue damage to their muscles, impaired muscle strength, inflammation, cardiac dysfunction and fibrosis.
Anti-LTBP4 is our complementary disease modifying program with the goal of identifying and developing a monoclonal antibody intended to
reduce fibrosis and inflammation by targeting and stabilizing the LTBP4 protein. LTBP4 is highly expressed in muscle and, when stable, prevents fibrosis
and inflammation by inhibiting the activation of the TGF-beta pathway.
The rationale for targeting LTBP4 originated from observations in DMD natural history studies. Researchers found that subsets of patients with
genetic variants in the LTBP4 gene maintained their ability to walk longer compared to patients in the study who did not. Researchers discovered that these
genetic variants lead to reduced TGF-beta signaling. Elizabeth McNally, M.D., Ph.D., Director of the Center for Genetic Medicine at Northwestern
University, hypothesized that stabilization of the LTBP4 protein in DMD patients could mimic the effect.
In order to assess the efficacy of potential human antibody clinical candidates in preclinical models, mice expressing the human version of LTBP4
were crossed with mdx mice to generate a DMD model that expressed human LTBP4 (hLTBP4:mdx). Preliminary studies showed that the hLTBP4:mdx
animals treated with an anti-LTBP4 antibody showed significantly lower levels of fibrosis and inflammation due to the stabilization of the LTBP4 protein.
In order to focus our efforts on addressing the underlying cause of DMD through our SGT-001 program, we have significantly curtailed investment
in our other programs, including anti-LTBP4.
Tools to accelerate discovery and development
In conjunction with our development of SGT-001, we believe it is critical to continue to invest time and research into tools designed to help us more
effectively measure disease progression and the therapeutic impact of our product candidates. We are developing biomarkers and sensors that will allow us
to identify treatment targets faster, better measure the therapeutic impact of potential product candidates and reach therapeutic decision points earlier.
Non-invasive biochemical and imaging biomarkers
We are working to identify non-invasive biochemical and imaging biomarkers that could potentially reduce or eliminate the need for muscle
biopsies in clinical trials, reducing stress on patients and allowing better evaluation of potential product candidates. We are developing a platform
technology that may enable the non-invasive measurement of changes associated with increased dystrophin and dystrophin-like protein expression in DMD
patients by using established imaging techniques. We are also currently using leading, robust platforms to perform extensive analysis on patient samples to
establish molecular signatures based on various stages of DMD disease progression.
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Assistive devices
Solid Suit
In partnership with Silicon Valley’s Seismic and Parent Project Muscular Dystrophy (PPMD), we have advanced development of wearable assistive
devices that may have both functional and therapeutic benefits, with the goal of helping patients perform day-to-day activities with greater ease and
preserving their muscle function. We refer to these devices as the Solid Suit. This work was done with the insight and oversight of technology innovators
and engineering and disease experts, and each step is informed by input from the patient community. The Solid Suit utilizes cutting-edge technologies to
power soft, light-weight comfortable exoskeletons with the potential to offset muscle fatigue and augment muscle strength. The Solid Suit is comprised of
three separate components, all of which are currently in prototype development.
In order to focus our efforts on addressing the underlying cause of DMD through our SGT-001 program, we have significantly curtailed investment
in our other programs, including the Solid Suit.
Intellectual property
Our commercial success depends in part on our ability to obtain and maintain proprietary or intellectual property protection for our product
candidates, including SGT-001, and other know-how, to operate without infringing, misappropriating or otherwise violating the intellectual property rights
of others, and to prevent others from infringing, misappropriating or otherwise violating our intellectual property rights. We also rely on patents, trade
secrets, know-how and continuing technological innovation to develop and maintain our proprietary and intellectual property position.
As of February 15, 2020, we have filed eight pending U.S. patent applications, two pending PCT international patent applications, and one pending
patent application in Taiwan. For our gene transfer programs, we have exclusively licensed four issued U.S. patents, two pending U.S. non-provisional
patent applications, and ten granted patents and ten pending patent applications in foreign jurisdictions. The issued U.S. patents are projected to expire
between 2021 and 2036, excluding any patent term adjustments and any patent term extensions, and any U.S. patents that may issue from the pending U.S.
non-provisional patent application and U.S. provisional patent applications (assuming U.S. non-provisional patent applications are timely filed with respect
to such provisional patent applications and all other applicable requirements are satisfied) would be projected to expire in 2036, excluding any patent term
adjustments and any patent term extensions.
With respect to our gene transfer programs, we exclusively licensed patent families that relate to microdystrophin genes. With respect to SGT-001,
we exclusively licensed two issued U.S. patents and two pending U.S. non-provisional patent applications, which generally claim the structural elements of
SGT-001 and the promoter sequence used in SGT-001. These issued U.S. patents are projected to expire in 2028 and 2036, excluding any patent term
adjustments and any patent term extensions.
Relating to SGT-001, we also own seven pending U.S. provisional patent applications, one pending U.S. patent application, and one pending PCT
international patent application. Any patents that may be issued from the pending PCT and our pending U.S. provisional patent application (assuming a
U.S. non-provisional patent application is timely filed with respect to such provisional patent application and all other applicable requirements are satisfied)
would be projected to expire between 2036 and 2041, excluding any patent term adjustments and any patent term extensions. Substantive prosecution of
our patent applications has not yet commenced at the U.S. Patent and Trademark Office, or USPTO. We cannot predict whether such pending patent
applications will result in the issuance of a patent that effectively protects SGT-001, or if such issued patent or any of our licensor’s issued patents will
effectively prevent others from commercializing competitive products. In any event, patent prosecution is a lengthy process, during which the scope of the
claims initially submitted for examination by the patent offices in various jurisdictions are often significantly narrowed by the time they issue, if they issue
at all.
With respect to our Anti-LTBP4 program, we do not currently own or in-license any issued patents or patent applications relating to such product
candidate. We have exercised an exclusive option to negotiate for licenses of certain patents and patent applications relating to Anti-LTBP4 from Ikaika
Therapeutics, LLC. Ikaika Therapeutics, LLC is only required to negotiate the terms of a potential license agreement with us for certain specified periods of
time and we may be unable to enter into such a definitive license agreement within the required timeframe or under terms that are acceptable to us. If we
are unable to enter into such a definitive license agreement, we will not have any license to such patents and patent applications.
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The term of individual patents depends upon the legal term for patents in the countries in which they are obtained. In most countries, including the
United States, the patent term is 20 years from the earliest filing date of a non-provisional patent application. In the United States, a patent’s term may be
lengthened by patent term adjustment, which compensates a patentee for administrative delays by the USPTO in examining and granting a patent, or may
be shortened if a patent is terminally disclaimed over an earlier filed patent. The term of a patent that covers a drug or biological product may also be
eligible for patent term extension when FDA approval is granted, subject to certain limitations and provided statutory and regulatory requirements are met
(for more information, please see “Business— Government regulation and product approval —U.S. patent term restoration and marketing exclusivity”). In
the future, if and when our product candidates receive approval from the FDA or foreign regulatory authorities, we expect to apply for patent term
extensions on issued patents we may obtain in the future covering those products, depending upon the length of the clinical trials for each product and other
factors. There can be no assurance that any of our pending patent applications will issue or that we will benefit from any patent term extension or favorable
adjustment to the term of any of our patents.
As with other biotechnology and pharmaceutical companies, our ability to maintain and solidify our proprietary and intellectual property position
for our product candidates will depend on our success in obtaining effective patent claims and enforcing those claims if granted. However, our owned and
licensed pending patent applications, and any patent applications that we may in the future file or license from third parties may not result in the issuance of
patents. We also cannot predict the breadth of claims that may be allowed or enforced in our patents. Any issued patents that we may receive in the future
may be challenged, invalidated or circumvented. In addition, because of the extensive time required for clinical development and regulatory review of a
product candidate we may develop, it is possible that, before any of our product candidates can be commercialized, any related patent may expire or remain
in force for only a short period following commercialization, thereby limiting protection such patent would afford the respective product and any
competitive advantage such patent may provide.
In addition to patents, we rely upon unpatented trade secrets and know-how and continuing technological innovation to develop and maintain our
competitive position. We seek to protect our proprietary information, in part, by executing confidentiality agreements with our collaborators and scientific
advisors, and non-competition, non-solicitation, confidentiality, and invention assignment agreements with our employees and consultants. We have also
executed agreements requiring assignment of inventions with selected scientific advisors and collaborators. The confidentiality agreements we enter into
are designed to protect our proprietary information and the agreements or clauses requiring assignment of inventions to us are designed to grant us
ownership of technologies that are developed through our relationship with the respective counterparty. We cannot guarantee, however, that these
agreements will afford us adequate protection of our intellectual property and proprietary information rights.
We also seek trademark protection in the United States and internationally where available and when appropriate. We currently own U.S. federal
registrations for the marks SOLID, SOLID GT and SOLID BIOSCIENCES and a European Union registration for the mark SOLID GT.
Strategic partnerships and collaborations/licenses
We have certain obligations under licensing agreements with third parties that include annual maintenance fees and payments that are contingent
upon achieving various development, commercial and regulatory milestones. Pursuant to many of these license agreements, we are required to make
milestone payments if certain development, regulatory and commercial sales milestones are achieved, and may have certain additional research funding
obligations. Also, pursuant to the terms of many of these license agreements, when and if commercial sales of a licensed product commence, we must pay
royalties to our licensors on net sales of the respective licensed products.
University of Washington License Agreement
In 2015, we entered into a license agreement with the University of Washington, acting through UW CoMotion, under which we obtained an
exclusive, royalty-bearing, sublicensable, worldwide license under certain patent applications owned by the University of Washington relating to novel
micro-dystrophins to develop, manufacture, and commercialize products for use in the treatment of DMD and related disease indications caused by a lack
of functional dystrophin. We have the right to grant sublicenses to third parties contingent upon written approval by the University of Washington prior to
executing such sublicense, which approval may not be unreasonably withheld.
11
In consideration for the rights granted by the agreement, we paid a one-time, non-refundable license fee, which was recorded as a research and
development expense in 2015. We are required to reimburse the University of Washington for costs incurred in applying for, prosecuting and maintaining
patents and pay up to an aggregate of approximately $1 million upon the achievement of certain milestones. There were no milestones achieved during the
years ended December 31, 2019 and 2018. In October 2017, the first milestone was achieved under this agreement. The milestone payment was recorded as
a research and development expense in the fourth quarter of 2017. We must also pay royalties of a low single digit percentage of future sales by us and our
sublicensees of products developed under the licensed patent rights. In addition, we must pay an annual maintenance fee until certain milestones are
achieved, at which time a minimum annual royalty requirement will replace such maintenance fee and will apply to us and our sublicensees.
We are obligated to use our commercially reasonable efforts, consistent with sound and reasonable business practices and judgment, to
commercialize the inventions covered by the licensed patent rights and to make and sell products based on that patent as soon as practicable and maximize
sales thereof.
The University of Washington controls the prosecution and maintenance of the licensed patents in consultation with us and at our expense. In
countries in which we have not requested prosecution or maintenance of licensed patents, the University of Washington may prosecute and maintain such
licensed patents at its own cost. We have the first right to enforce such licensed patents at our expense. However, we may not enter into any settlement in
any manner relating to the licensed patents without the University of Washington’s prior written consent.
The license agreement remains in effect until the expiration of the last-to-expire patent licensed under the agreement. We may terminate the
agreement at any time upon providing sixty days’ written notice to the University of Washington. The University of Washington may terminate the
agreement upon our uncured, material breach of the agreement or if we enter into an insolvency-related event.
The University of Missouri License Agreement
In 2015, we entered into a license agreement with the Curators of the University of Missouri, or the University of Missouri, a public corporation of
Missouri, under which we obtained an exclusive, royalty-bearing, sublicensable, worldwide license under certain patents and patent applications owned by
the University of Missouri relating to a novel synthetic microdystrophin gene to make, sell and distribute products for use in the treatment of DMD and
related disease indications resulting from a lack of functional dystrophin.
In consideration for the rights granted by the agreement, we paid a one-time, non-refundable license fee, which was recorded as a research and
development expense in 2015. We are required to reimburse the University of Missouri for costs incurred in applying for, prosecuting and maintaining the
licensed patents and pay up to an aggregate of approximately $1 million upon the achievement of certain milestones for each product developed based on
the licensed patents. There were no milestones achieved during the years ended December 31, 2019 and 2018. In October 2017, the first milestone was
achieved under this agreement. The milestone payment was recorded as a research and development expense in the fourth quarter of 2017. We must pay a
royalty of a low single digit percentage of future sales by us or our sublicensees of products developed using the licensed patents. In addition, we must pay
an annual maintenance fee until certain milestones are achieved, after which time a minimum annual royalty will replace such maintenance fee.
Under the agreement, we granted the University of Missouri a non-exclusive, royalty-free, irrevocable, paid-up license, with the right to grant
sublicenses to non-profit, academic, educational or governmental institutions, to practice and use improvements made by us using the licensed patent
rights, solely for non-commercial research purposes.
We are obligated to use our reasonable best efforts to introduce products based on the licensed patent rights into the commercial market as soon as
possible, consistent with sound and reasonable business practices and judgment, and thereafter to keep such products reasonably available to the public.
The University of Missouri controls the prosecution and maintenance of the licensed patents in consultation with us and at our expense. In countries
in which we have not requested prosecution or maintenance of licensed patents, the University of Missouri may prosecute and maintain such licensed
patents at its own cost. We have the first right to enforce such licensed patents at our expense. However, any settlement, consent judgment or other
voluntary disposition of litigation that materially limits the scope, validity or enforceability of the licensed patent or admits fault or wrongdoing on the part
of the University of Missouri must be pre-approved in writing by the University of Missouri.
12
The license agreement remains in effect until the expiration of the last-to-expire patent or the abandonment of the last to be abandoned patent
application licensed under the agreement. The University of Missouri may terminate the agreement, or render the license granted thereunder non-exclusive,
in individual countries if we and our sublicensees fail to achieve certain milestones. We may terminate the license agreement at any time upon providing
six months’ written notice to the University of Missouri and paying a termination fee. Each of the University of Missouri and we may also terminate the
agreement for an uncured default or breach of the agreement by the other party. Our ability to cure such breach only applies to the first two notices of such
breach provided by the University of Missouri, and thereafter, the University of Missouri may terminate the agreement for our default or breach of the
agreement upon thirty days’ written notice without an opportunity to cure such default or breach.
The University of Michigan License Agreement
In 2016, we entered into a license agreement with the Regents of the University of Michigan, or the University of Michigan, a constitutional
corporation of Michigan, under which we obtained an exclusive, royalty-bearing, sublicensable, worldwide license to make, sell and distribute products
under certain patents owned by the University of Michigan related to microdystrophin and utrophin spectrin-like nucleic acid sequences for any use that,
but for this agreement, would comprise an infringement of a valid claim included in the licensed patent rights.
In consideration for the rights granted by the agreement, we paid a one-time license fee and a separate fee to cover past patent prosecution costs. We
recorded the upfront license fee as a research and development expense in 2016. We are required to reimburse the University of Michigan for costs incurred
in applying for, prosecuting and maintaining patents, and pay up to an aggregate of approximately $1 million upon the achievement of certain milestones.
There were no milestones achieved during the years ended December 31, 2019, 2018 and 2017. We must also pay royalties of a low single-digit percentage
of future sales by us or our sublicensees of products developed using the licensed rights, with a minimum annual royalty after certain milestones are
achieved. In addition, we must pay an annual maintenance fee in any year in which the minimum annual royalty is not reached.
Under the agreement, the University of Michigan reserves for itself and its affiliates the right to use the licensed rights for non-commercial research,
public service, internal and educational purposes and the right to grant the same limited non-commercial rights to other non-profit research institutions.
We are obligated to use commercially reasonable efforts to bring one or more products based on the licensed patents to market through a diligence
program for utilizing the licensed patents, to continue diligent marketing efforts throughout the term of the agreement, and to make reasonable amounts of
such products commercially available, in each case consistent with prudent business practices and judgment.
The University of Michigan controls the prosecution and maintenance of the licensed patents in consultation with us and at our expense. In
countries in which we have not requested prosecution or maintenance of licensed patents, the University of Michigan may prosecute and maintain such
licensed patents at its own cost. We have the first right to enforce such licensed patents at our expense. However, we may only enter into a settlement with
the advice and consent of the University of Michigan.
The license agreement remains in effect until the expiration of the last-to-expire patent licensed under the agreement. The University of Michigan
may terminate the agreement upon our uncured material breach of the agreement, including failure to make required payments under the agreement or to
achieve certain milestones, or if we become insolvent or bankrupt. We may terminate the license agreement at any time upon providing sixty days’ written
notice to the University of Michigan.
Harvard College License Agreements
In 2016 and 2017, we entered into license agreements with the President and Fellows of Harvard College, or Harvard College, under which we
obtained non-exclusive, royalty-bearing, sublicensable, worldwide licenses to use certain intellectual property owned by Harvard College to develop,
manufacture, and commercialize products for use in the treatment of DMD.
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In consideration for the rights granted by each agreement, we paid one-time, non-refundable license fees, which were recorded as a research and
development expense in 2016 and 2017. We are required to pay an annual license maintenance fee until certain milestones are achieved, after which time
the annual maintenance fee will increase annually. Such annual maintenance fees will further increase if we grant certain rights to a sublicensee or strategic
partner with whom we collaborate on the development and commercialization of licensed products. The annual maintenance fees are creditable against
royalty payments. We also must pay milestone payments within thirty days after achieving certain milestones. There were no milestones achieved during
the years ended December 31, 2019, 2018 and 2017 under either agreement. We must pay a royalty on future sales by us or our sublicensees of products
developed using the licensed technology.
The license agreements each remain in effect for an initial term of fifteen years, with automatic three-year renewal periods thereafter unless one of
the parties provides notice of non-renewal. We may terminate the license agreements at any time upon providing sixty days’ written notice to Harvard
College. Harvard College may terminate the agreements in the event we become bankrupt or insolvent. Both Harvard College and we may also terminate
the agreements for an uncured material breach of the agreements by the other party.
Other License Agreements
In 2016, we entered into a license agreement with Life Technologies Corporation, or Life Technologies. In consideration for obtaining a non-
exclusive, royalty-free, worldwide license to use certain technologies and associated know-how to develop our product candidates, we paid a one-time,
non-refundable license fee. This fee was recorded as a research and development expense in 2016. The license agreement will remain effective in
perpetuity unless earlier terminated. Life Technologies has the right to terminate the agreement upon our material, uncured breach of the agreement or in
the event that it determines that continued performance of the agreement may violate any laws. We are obligated to diligently pursue regulatory approval
necessary for the development, manufacture and sale of the licensed products. We have the right to terminate the agreement at any time upon providing
thirty days’ written notice to Life Technologies.
Competition
The biotechnology and pharmaceutical industries are characterized by rapidly changing technologies, significant competition and a strong emphasis
on intellectual property. This is also true in treatments of DMD, as well as in gene therapy. While we believe that our focus, strength of team, expertise in
gene therapy, scientific knowledge and intellectual property provide us with competitive advantages, we face competition from several different sources,
including large and small biopharmaceutical companies, academic research institutions, government agencies and public and private research institutions.
Not only must we compete with other companies that are focused on gene transfer technology, but any product candidates that we successfully develop and
commercialize will compete with existing therapies and new therapies that may become available in the future.
Many of our competitors have significantly greater financial resources and expertise in research and development, manufacturing, preclinical
testing, clinical trials, regulatory approvals and product marketing than we do. These competitors also compete with us in recruiting and retaining qualified
scientific and management personnel and establishing clinical trial sites and patient registration for clinical trials, as well as in acquiring technologies
complementary to, or necessary for, our programs. Mergers and acquisitions in the pharmaceutical and biotechnology industries may result in even more
resources being concentrated among a smaller number of our competitors. Smaller or early stage companies may also prove to be significant competitors,
particularly through collaborative arrangements with large and established companies.
We are aware of several companies and research institutions focused on developing systemic gene transfers for DMD, including Pfizer Inc., with a
product candidate currently in Phase 1 clinical development, and Sarepta Therapeutics, Inc., with a product candidate currently in Phase I/II clinical trial
development. In addition, there are three therapies, which are intended to be disease modifying, that are currently approved for DMD by certain regulators.
These products are eteplirsen (EXONDYS 51) and ataluren (Translarna), each of which is indicated for approximately 13% of DMD patients, and
golodirsen (VYONDYS 53), which is indicated for approximately 8% of DMD patients. Any advances in gene transfer technology made by a competitor
may be used to develop therapies that could compete with our lead product candidate or other product candidates we may develop.
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Government regulation and product approval
U.S. government regulation and product approval
In the United States, biologic products including gene therapy products, such as our lead product candidate, are licensed for marketing by the FDA
under the Public Health Service Act, or PHS Act, and regulated by the FDA under the Federal Food, Drug, and Cosmetic Act, or FD&C Act, as well as by
other federal, state and local statutes and regulations. Both the FD&C Act and the PHS Act and their corresponding rules and regulations govern, among
other things, the testing, manufacturing, safety, efficacy, labeling, packaging, storage, record keeping, distribution, reporting, advertising and other
promotional practices involving biologic products. FDA approval must be obtained before conducting human clinical testing of biologic products.
Additionally, each clinical trial protocol for a gene therapy product candidate is reviewed by the FDA and, in limited instances, the U.S. National Institutes
of Health, or the NIH, through its Office of Biotechnology Activities’ Recombinant DNA Advisory Committee, or RAC. FDA must license a biologic
product before it may be marketed within the United States.
Within the FDA, the Center for Biologics Evaluation and Research, or the CBER, regulates gene therapy products. Within CBER, the review of
gene therapy and related products is consolidated in the Office of Tissues and Advanced Therapies, or the OTAT, and the FDA has established the Cellular,
Tissue and Gene Therapies Advisory Committee to advise CBER on its reviews. CBER, which works closely with the NIH and the RAC, makes
recommendations to the NIH on gene therapy issues and engages in a public discussion of scientific, safety, ethical and societal issues related to proposed
and ongoing gene therapy protocols. The FDA has licensed human gene therapies products for sale in the United States, and the agency has provided
guidance for the development of other gene therapy products. This guidance includes a growing body of guidance documents on chemistry, manufacturing
and control, or CMC, clinical investigations and other areas of gene therapy development, all of which are intended to facilitate the industry’s development
of gene therapy products.
U.S. biologic products development process
The process required by the FDA before a biologic product may be marketed in the United States generally involves the following:
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completion of preclinical laboratory tests and in vivo studies according to the FDA’s GLP requirements and applicable requirements for the
humane use of laboratory animals or other applicable regulations;
submission to the FDA of an application for an IND, which allows human clinical trials to begin unless the FDA objects within 30 days;
approval by an institutional review board, or IRB, reviewing each clinical site before each clinical trial may be initiated;
approval by an institutional biosafety committee, or IBC, assessing the safety of the clinical research and identifying any potential risk to
public health or the environment;
performance of adequate and well controlled human clinical trials according to the FDA’s regulations commonly referred to as good clinical
practices, or GCPs, and any additional requirements for the protection of human research subjects and their health information, to establish the
safety, potency and purity of the proposed biologic product for its intended use;
preparation and submission to the FDA of a biologics license application, or BLA, for marketing approval that includes substantive evidence
of safety, purity and potency from results of preclinical testing and clinical trials, and detailed information about the CMC for the product,
reports of the outcomes and full data sets of the clinical trials and proposed labeling and packaging for the product;
review of the product candidate by an FDA advisory committee, if applicable;
satisfactory completion of an FDA inspection of the manufacturing facility or facilities where the biologic product candidate is produced to
assess compliance with cGMP requirements and to assure that the facilities, methods and controls are adequate to preserve the biologic
product candidate’s identity, safety, strength, quality and purity;
potential FDA audit of the nonclinical and clinical trial sites that generated the data in support of the BLA;
payment of user fees;
FDA review and licensure of the BLA; and
compliance with any post-approval requirements, including the potential requirement to implement a Risk Evaluation and Mitigation Strategy,
or REMS, and the potential requirement to conduct post-approval studies.
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Before testing any biologic product candidate in humans, including a gene therapy product candidate, the product candidate must undergo
preclinical testing. Preclinical tests, also referred to as nonclinical studies, include laboratory evaluations of product chemistry, toxicity and formulation, as
well as in vivo studies to assess the potential safety and activity of the product candidate and to establish a rationale for therapeutic use. The conduct of
certain nonclinical studies must comply with federal regulations and requirements, including GLPs.
If a gene therapy trial is conducted at, or sponsored by, institutions receiving NIH funding for recombinant DNA research, prior to the submission
of an IND to the FDA, a protocol and related documents must be submitted to, and the study registered with, the NIH Office of Biotechnology Activities,
or OBA, pursuant to the NIH Guidelines for Research Involving Recombinant DNA Molecules, or NIH Guidelines. Compliance with the NIH Guidelines
is mandatory for investigators at institutions receiving NIH funds for research involving recombinant DNA. However, many companies and other
institutions, not otherwise subject to the NIH Guidelines, voluntarily follow them. NIH is responsible for convening the RAC that discusses protocols that
raise novel or particularly important scientific, safety or ethical considerations at one of its quarterly public meetings. The OBA will notify the FDA of the
RAC’s decision regarding the necessity for full public review of a gene therapy protocol. RAC proceedings and reports are posted to the OBA website and
may be accessed by the public.
The clinical trial sponsor must submit the results of the preclinical tests, together with manufacturing information, analytical data, any available
clinical data or literature and a proposed clinical protocol, to the FDA as part of the IND. An IND is an exemption from the FD&C Act that allows an
unapproved product to be shipped in interstate commerce for use in an investigational clinical trial and a request for FDA authorization to administer an
investigational product to humans. Some preclinical tests may continue even after the IND is submitted. The IND automatically becomes effective 30 days
after receipt by the FDA, unless the FDA places the clinical trial on a full clinical hold or partial clinical hold. In such a case, the IND sponsor and the FDA
must resolve any outstanding concerns before the clinical trial can begin. With gene therapy protocols, if the FDA allows the IND to proceed, but the RAC
decides that full public review of the protocol is warranted, the FDA will request at the completion of its IND review that the sponsor delay initiation of the
protocol until after completion of the RAC review process. The FDA also may impose clinical holds on a biologic product candidate at any time before or
during clinical trials due to safety concerns or non-compliance. If the FDA imposes a clinical hold, trials may not recommence without FDA authorization
and then only under terms authorized by the FDA.
In addition, the FDA may impose a partial clinical hold at any time before or during clinical trials. A partial clinical hold is a delay or suspension of
only part of the clinical work requested under the IND (e.g., a specific protocol or part of a protocol is not allowed to proceed; however, other protocols or
parts of the protocol are allowed to proceed under the IND). If the FDA requires that progress to the next study is contingent on (i) FDA review of
additional data and (ii) subsequent specific permission for the study to proceed, this represents a partial clinical hold.
Human clinical trials under an IND
Clinical trials involve the administration of the biologic product candidate to healthy volunteers or subjects under the supervision of qualified
investigators, generally physicians not employed by, or under the control of, the trial sponsor. Clinical trials are conducted under written study protocols
detailing, among other things, the objectives of the clinical trial, dosing procedures, subject selection and exclusion criteria, and the parameters to be used
to monitor subject safety, including stopping rules that assure a clinical trial will be stopped if certain adverse events should occur. Each protocol and any
amendments to the protocol must be submitted to the FDA as part of the IND. Clinical trials must be conducted and monitored in accordance with the
FDA’s regulations comprising the GCP requirements, including the requirement that all research subjects provide informed consent.
Further, each clinical trial must be reviewed and approved by an IRB at or servicing each institution at which the clinical trial will be conducted. An
IRB is charged with protecting the welfare and rights of trial participants and considers such items as whether the risks to individuals participating in the
clinical trials are minimized and are reasonable in relation to anticipated benefits. The IRB also approves the form and content of the informed consent that
must be signed by each clinical trial subject or his or her legal representative, reviews and approves the study protocol and must monitor the clinical trial
until completed. Additionally, some trials are overseen by an independent group of qualified experts organized by the trial sponsor, known as a data safety
monitoring board or committee, or DSMB. This group provides authorization as to whether or not a trial may move forward at designated check points
based on access that only the group maintains to available data from the study. Clinical trials involving recombinant DNA also must be reviewed by an IBC
a local institutional committee that reviews and oversees basic and clinical research and utilizes recombinant DNA at that institution. The IBC assesses the
safety of the research and identifies any potential risk to public health or the environment.
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Human clinical trials are typically conducted in three sequential phases that may overlap or be combined:
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Phase I. The investigational biologic product is initially introduced into a small group of healthy human subjects and tested for safety, dosage
tolerance, absorption, metabolism, distribution, excretion and, if possible, to gain an early understanding of its effectiveness. In the case of
some product candidates for severe or life-threatening diseases, especially when the product candidate may be too inherently toxic to ethically
administer to healthy volunteers, the initial human testing is often conducted in patients. Phase I clinical trials of gene therapies are typically
conducted in patients rather than healthy volunteers.
Phase II. The biologic product candidate is evaluated in a limited patient population to identify possible adverse effects and safety risks, to
preliminarily evaluate the efficacy of the product for specific targeted diseases and to determine dosage tolerance, optimal dosage and dosing
schedule.
Phase III. Phase III clinical trials are commonly referred to as “pivotal” studies, which typically denotes a study that presents the data that the
FDA or other relevant regulatory agency will use to determine whether or not to approve a biologic product. In Phase III clinical trials, the
investigational biologic product is administered to an expanded patient population, generally at multiple geographically dispersed clinical trial
sites in adequate and well controlled clinical trials to generate sufficient data to statistically confirm the potency and safety of the product for
approval. These clinical trials are intended to establish the overall risk/benefit ratio of the product candidate and provide an adequate basis for
product labeling.
Post-approval clinical trials, sometimes referred to as Phase IV clinical trials, may be conducted after initial approval. These clinical trials are used
to gain additional experience from the treatment of patients in the intended therapeutic indication, particularly for long-term safety follow-up.
During all phases of clinical development, regulatory agencies require extensive monitoring and auditing of all clinical activities, clinical data and
clinical trial investigators. Annual progress reports detailing the results of the clinical trials must be submitted to the FDA.
Written IND safety reports must be promptly submitted to the FDA, the NIH and the investigators for serious and unexpected adverse events, any
findings from other trials, in vivo laboratory tests or in vitro testing that suggest a significant risk for human subjects, or any clinically important increase in
the rate of a serious suspected adverse reaction over that listed in the protocol or investigator brochure. The sponsor must submit an IND safety report
within 15 calendar days after the sponsor determines that the information qualifies for reporting. The sponsor also must notify the FDA of any unexpected
fatal or life-threatening suspected adverse reaction within seven calendar days after the sponsor’s initial receipt of the information.
The FDA or the sponsor or its DSMB may suspend a clinical trial at any time on various grounds, including a finding that the research subjects or
patients are being exposed to an unacceptable health risk. Similarly, an IRB can suspend or terminate approval of a clinical trial at its institution if the
clinical trial is not being conducted in accordance with the IRB’s requirements or if the biologic product candidate has been associated with unexpected
serious harm to patients.
Information about certain clinical trials must be submitted within specific timeframes to the NIH for public dissemination on its ClinicalTrials.gov
website. Similar requirements for posting clinical trial information are present in the European Union (EudraCT) website: https://eudract.ema.europa.eu/
and other countries, as well.
Expanded Access to an Investigational Drug for Treatment Use
Expanded access, sometimes called “compassionate use,” is the use of investigational new drug products outside of clinical trials to treat patients
with serious or immediately life-threatening diseases or conditions when there are no comparable or satisfactory alternative treatment options. The rules
and regulations related to expanded access are intended to improve access to investigational drugs for patients who may benefit from investigational
therapies. FDA regulations allow access to investigational drugs under an IND by the company or the treating physician for treatment purposes on a case-
by-case basis for: individual patients (single-patient IND applications for treatment in emergency settings and non-emergency settings); intermediate-size
patient populations; and larger populations for use of the drug under a treatment protocol or Treatment IND Application.
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When considering an IND application for expanded access to an investigational product with the purpose of treating a patient or a group of patients,
the sponsor and treating physicians or investigators will determine suitability when all of the following criteria apply: patient(s) have a serious or
immediately life-threatening disease or condition, and there is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or
condition; the potential patient benefit justifies the potential risks of the treatment and the potential risks are not unreasonable in the context or condition to
be treated; and the expanded use of the investigational drug for the requested treatment will not interfere with initiation, conduct, or completion of clinical
investigations that could support marketing approval of the product or otherwise compromise the potential development of the product.
On December 13, 2016, the 21st Century Cures Act established (and the 2017 Food and Drug Administration Reauthorization Act later amended) a
requirement that sponsors of one or more investigational drugs for the treatment of a serious disease(s) or condition(s) make publicly available their policy
for evaluating and responding to requests for expanded access for individual patients. Although these requirements were rolled out over time, they have
now come into full effect. This provision requires drug and biologic companies to make publicly available their policies for expanded access for individual
patient access to products intended for serious diseases. Sponsors are required to make such policies publicly available upon the earlier of initiation of a
Phase II or Phase III study; or 15 days after the drug or biologic receives designation as a breakthrough therapy, fast track product, or regenerative medicine
advanced therapy.
In addition, on May 30, 2018, the Right to Try Act, was signed into law. The law, among other things, provides a federal framework for certain
patients to access certain investigational new drug products that have completed a Phase I clinical trial and that are undergoing investigation for FDA
approval. Under certain circumstances, eligible patients can seek treatment without enrolling in clinical trials and without obtaining FDA permission under
the FDA expanded access program. There is no obligation for a drug manufacturer to make its drug products available to eligible patients as a result of the
Right to Try Act, but the manufacturer must develop an internal policy and respond to patient requests according to that policy.
Special Regulations and Guidance Governing Gene Therapy Products
The FDA has defined a gene therapy product as one that mediates its effects by transcription and/or translation of transferred genetic material
and/or by integrating into the host genome and which is administered as nucleic acids, viruses, or genetically engineered microorganisms. The products
may be used to modify cells in vivo or transferred to cells ex vivo prior to administration to the recipient. Within the FDA, the Center for Biologics
Evaluation and Research, or CBER, regulates gene therapy products. Within CBER, the review of gene therapy and related products is consolidated in the
OTAT, and the FDA has established the Cellular, Tissue and Gene Therapies Advisory Committee to advise CBER on its reviews. CBER works closely
with the Local Biosafety Board, a federal advisory committee, in reviewing proposed and ongoing gene therapy protocols and engaging in a public
discussion of scientific, safety, ethical, and societal issues related to those protocols. The NIH and the Recombinant DNA Advisory Committee, or RAC, a
federal advisory committee, also advise the FDA on gene therapy issues and other issues related to emerging technologies. The FDA and the NIH have
published guidance documents with respect to the development and submission of gene therapy protocols.
The FDA has issued various guidance documents regarding gene therapies, including recent final guidance documents released in January 2020
relating to chemistry, manufacturing and controls information for gene therapy INDs, gene therapies for rare diseases and gene therapies for retinal
disorders. Although the FDA has indicated that these and other guidance documents it previously issued are not legally binding, we believe that our
compliance with them is likely necessary to gain approval for any gene therapy product candidate we may develop. The guidance documents provide
additional factors that the FDA will consider at each of the above stages of development and relate to, among other things, the proper preclinical
assessment of gene therapies; the chemistry, manufacturing, and control information that should be included in an IND application; the proper design of
tests to measure product potency in support of an IND or BLA application; and measures to observe delayed adverse effects in subjects who have been
exposed to investigational gene therapies when the risk of such effects is high. Further, the FDA usually recommends that sponsors observe subjects for
potential gene therapy-related delayed adverse events for a 15-year period, including a minimum of five years of annual examinations followed by 10 years
of annual queries, either in person or by questionnaire.
If a gene therapy trial is conducted at, or sponsored by, institutions receiving the NIH funding for recombinant DNA research, a protocol and related
documentation must be submitted to, and the study registered with, the NIH Office of Biotechnology Activities, or OBA, pursuant to the NIH Guidelines
for Research Involving Recombinant DNA Molecules prior to the submission of an IND to the FDA. In addition, many companies and other institutions
not otherwise subject to the NIH Guidelines voluntarily follow them. The NIH will convene the RAC to discuss protocols that raise novel or particularly
important scientific, safety or ethical considerations at one of its quarterly public meetings. The OBA will notify the FDA of
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the RAC’s decision regarding the necessity for full public review of a gene therapy protocol. RAC proceedings and reports are posted to the OBA website
and may be accessed by the public.
Further, to facilitate adverse event reporting and dissemination of additional information about gene therapy trials, the FDA and the NIH established
the Genetic Modification Clinical Research Information System, or GeMCRIS. Investigators and sponsors of human gene transfer trials can utilize this
web-based system to report serious adverse events and to provide annual reports. GeMCRIS also allows members of the public to access basic reports
about human gene transfer trials registered with the NIH and to search for information such as trial location, the names of investigators conducting trials,
and the names of gene transfer products being studied.
Finally, for a gene therapy product, the FDA also will not approve the product if the manufacturer is not in compliance with good tissue practices,
or GTP. These standards are found in FDA regulations and guidances that govern the methods used in, and the facilities and controls used for, the
manufacture of human cells, tissues, and cellular and tissue based products, or HCT/Ps, which are human cells or tissue intended for implantation,
transplant, infusion, or transfer into a human recipient. The primary intent of the GTP requirements is to ensure that cell and tissue based products are
manufactured in a manner designed to prevent the introduction, transmission, and spread of communicable disease. FDA regulations also require tissue
establishments to register and list their HCT/Ps with the FDA and, when applicable, to evaluate donors through screening and testing.
Compliance with cGMP requirements
Manufacturers of biologics must comply with applicable cGMP regulations, including quality control and quality assurance and maintenance of
records and documentation. Manufacturers and others involved in the manufacture and distribution of such products also must register their establishments
with the FDA and certain state agencies. Both domestic and foreign manufacturing establishments must register and provide additional information to the
FDA upon their initial participation in the manufacturing process. Establishments may be subject to periodic, unannounced inspections by government
authorities to ensure compliance with cGMP requirements and other laws. Discovery of problems may result in a government entity placing restrictions on
a product, manufacturer or holder of an approved BLA, and may extend to requiring withdrawal of the product from the market. The FDA will not approve
a BLA unless it determines that the manufacturing processes and facilities are in compliance with cGMP requirements and adequate to assure consistent
production of the product within required specification.
Concurrent with clinical trials, companies usually complete additional preclinical studies and must also develop additional information about the
physical characteristics of the biologic product candidate as well as finalize a process for manufacturing the product candidate in commercial quantities in
accordance with cGMP requirements. To help reduce the risk of the introduction of adventitious agents or of causing other adverse events with the use of
biologic products, the PHS Act emphasizes the importance of manufacturing control for products whose attributes cannot be precisely defined. The
manufacturing process must be capable of consistently producing quality batches of the product candidate and, among other requirements, the sponsor must
develop methods for testing the identity, strength, quality, potency and purity of the final biologic product. Additionally, appropriate packaging must be
selected and tested, and stability studies must be conducted to demonstrate that the biologic product candidate does not undergo unacceptable deterioration
over its shelf life.
U.S. review and approval processes
After the completion of clinical trials of a biologic product, FDA licensure of a BLA must be obtained before commercial marketing of the biologic
product. The BLA must include results of product development, laboratory and animal studies, human studies, information on the manufacture and
composition of the product, proposed labeling and other relevant information. In addition, under the Pediatric Research Equity Act, a BLA or supplement
to a BLA must contain data to assess the safety and effectiveness of the biologic product for the claimed indications in all relevant pediatric subpopulations
and to support dosing and administration for each pediatric subpopulation for which the product is safe and effective. The FDA may grant deferrals for
submission of data or full or partial waivers.
Under the Prescription Drug User Fee Act, or PDUFA, as amended, each BLA must be accompanied by a significant user fee. Under federal law,
the submission of most NDAs is subject to an application user fee, which for federal fiscal year 2020 is $2,942,965 for an application requiring clinical
data. The sponsor of an approved NDA is also subject to an annual program fee, which for fiscal year 2020 is $325,424. Fee waivers or reductions are
available in certain circumstances, including a waiver of the application fee for the first application filed by a small business. Additionally, no user fees are
assessed on BLAs for product candidates designated as orphan drugs, unless the product candidate also includes a non-orphan indication.
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The FDA reviews a BLA within 60 days of submission to determine if it is substantially complete before the agency accepts it for filing. The FDA
may refuse to file any BLA that it deems incomplete or not properly reviewable at the time of submission and may request additional information. In this
event, the BLA must be resubmitted with the additional information. The resubmitted application also is subject to review before the FDA accepts it for
filing. Once the submission is accepted for filing, the FDA begins an in-depth substantive review of the BLA.
The FDA reviews the BLA to determine, among other things, whether the proposed product is safe and potent, or effective, for its intended use, and
has an acceptable purity profile, and whether the product is being manufactured in accordance with cGMP to assure and preserve the product’s identity,
safety, strength, quality, potency and purity. The FDA may refer applications for novel biologic products or biologic products that present difficult
questions of safety or efficacy to an advisory committee, typically a panel that includes clinicians and other experts, for review, evaluation and a
recommendation as to whether the application should be approved and under what conditions. The FDA is not bound by the recommendations of an
advisory committee, but it considers such recommendations carefully when making decisions. During the biologic product approval process, the FDA also
will determine whether a REMS, is necessary to assure the safe use of the biologic product. REMS use risk minimization strategies beyond the professional
labeling to ensure that the benefits of the product outweigh the potential risks. To determine whether a REMS is needed, the FDA will consider the size of
the population likely to use the product, seriousness of the disease, expected benefit of the product, expected duration of treatment, seriousness of known or
potential adverse events and whether the product is a new molecular entity. A REMS could include medication guides, physician communication plans and
elements to assure safe use, such as restricted distribution methods, patient registries and other risk minimization tools. If the FDA concludes a REMS is
needed, the sponsor of the BLA must submit a proposed REMS; the FDA will not approve the BLA without a REMS, if required.
Before approving a BLA, the FDA will inspect the facilities at which the product candidate is manufactured. The FDA will not approve the product
candidate unless it determines that the manufacturing processes and facilities are in compliance with cGMP requirements and adequate to assure consistent
production of the product within required specifications. Additionally, before approving a BLA, the FDA will typically inspect one or more clinical sites to
assure that the clinical trials were conducted in compliance with IND study requirements and GCP requirements. cGMP, GLP and GCP compliance
requires significant expenditure of time, money and effort in the areas of training, recordkeeping, production and quality control.
Notwithstanding the submission of relevant data and information, the FDA may ultimately decide that the BLA does not satisfy its regulatory
criteria for approval and deny approval. Data obtained from clinical trials are not always conclusive and the FDA may interpret data differently than how
we would interpret the same data. On the basis of the BLA and accompanying information, including the results of the inspection of the manufacturing
facilities, the FDA may issue an approval letter or a complete response letter. An approval letter authorizes commercial marketing of the biologic product
with specific prescribing information for specific indications. A complete response letter generally outlines the deficiencies in the submission and may
require substantial additional testing or information in order for the FDA to reconsider the application. If and when those deficiencies have been addressed
to the FDA’s satisfaction in a resubmission of the BLA, the FDA will issue an approval letter. If the agency decides not to approve the BLA in its present
form, the FDA will issue a complete response letter that usually describes all of the specific deficiencies in the BLA identified by the FDA. The
deficiencies identified may be minor, for example, requiring labeling changes; or major, for example, requiring additional clinical trials. Additionally, the
complete response letter may include recommended actions that the applicant might take to place the application in a condition for approval. If a complete
response letter is issued, the applicant may either resubmit the BLA, addressing all of the deficiencies identified in the letter, or withdraw the application.
If a product receives regulatory approval, the approval may be significantly limited to specific diseases and dosages or the indications for use may
otherwise be limited, which could restrict the commercial value of the product. Further, the FDA may require that certain contraindications, warnings or
precautions be included in the product labeling. The FDA may impose restrictions and conditions on product distribution, prescribing or dispensing in the
form of a REMS, or otherwise limit the scope of any approval. In addition, the FDA may require post-marketing clinical trials, sometimes referred to as
Phase IV clinical trials, designed to further assess a biologic product’s safety and effectiveness, and testing and surveillance programs to monitor the safety
of approved products that have been commercialized.
The FDA has agreed to specified performance goals in the review of BLAs under the PDUFA. One such goal is to review standard BLAs in
ten months after the FDA accepts the BLA for filing, and priority BLAs in six months, whereupon a review decision is to be made. The FDA does not
always meet its PDUFA goal dates for standard and priority BLAs and its review goals are subject to change from time to time. The review process and the
PDUFA goal date may be extended by three months if the FDA requests or the BLA sponsor otherwise provides additional information or clarification
regarding information already provided in the submission within the last three months before the PDUFA goal date.
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Biosimilars and exclusivity
The Patient Protection and Affordable Care Act and the companion Health Care and Education Reconciliation Act, or the Health Care Reform Law,
which was signed into law on March 23, 2010, included a subtitle called the Biologics Price Competition and Innovation Act of 2009, or BPCIA. That Act
established a regulatory scheme authorizing the FDA to approve biosimilars and interchangeable biosimilars.
Under the BPCIA, a manufacturer may submit an application for licensure of a biologic product that is “biosimilar to” or “interchangeable with” a
previously approved biological product or “reference product.” In order for the FDA to approve a biosimilar product, it must find that there are no clinically
meaningful differences between the reference product and proposed biosimilar product in terms of safety, purity and potency. For the FDA to approve a
biosimilar product as interchangeable with a reference product, the agency must find that the biosimilar product can be expected to produce the same
clinical results as the reference product, and (for products administered multiple times) that the biologic and the reference biologic may be switched after
one has been previously administered without increasing safety risks or risks of diminished efficacy relative to exclusive use of the reference biologic.
Under the BPCIA, an application for a biosimilar product may not be submitted to the FDA until four years following the date of approval of the
reference product. The FDA may not approve a biosimilar product until 12 years from the date on which the reference product was approved. Even if a
product is considered to be a reference product eligible for exclusivity, another company could market a competing version of that product if the FDA
approves a full BLA for such product containing the sponsor’s own preclinical data and data from adequate and well-controlled clinical trials to
demonstrate the safety, purity and potency of their product. The BPCIA also created certain exclusivity periods for biosimilars approved as interchangeable
products. At this juncture, it is unclear whether products deemed “interchangeable” by the FDA will, in fact, be readily substituted by pharmacies, which
are governed by state pharmacy law.
As of January 1, 2020, the FDA has approved 26 biosimilar products for use in the United States. No interchangeable biosimilars, as described
below, have been approved. The FDA has issued several guidance documents outlining an approach to review and approval of biosimilars. Additional
guidances are expected to be finalized by the FDA.
Pediatric exclusivity
Pediatric exclusivity is another type of non-patent exclusivity in the United States and, if granted, provides for the attachment of an additional six
months of marketing protection to the term of any existing regulatory exclusivity or patent protection, including the non-patent and orphan exclusivity. This
six-month exclusivity may be granted if an application sponsor submits pediatric data that fairly respond to a written request from the FDA for such data.
The data do not need to show the product to be effective in the pediatric population studied; rather, if the clinical trial is deemed to fairly respond to the
FDA’s request, the additional protection is granted. If reports of requested pediatric studies are submitted to and accepted by the FDA within the statutory
time limits, whatever statutory or regulatory periods of exclusivity or patent protection cover the product are extended by six months. Thus, pediatric
exclusivity adds six months to existing exclusivity periods applicable to biological products under the BPCIA—namely, the four-year period during which
the FDA will not consider an application for a biosimilar product, and the 12-year period during which the FDA will not approve a biosimilar application.
Orphan drug designation
Under the Orphan Drug Act, the FDA may designate a biologic product as an “orphan drug” if it is intended to treat a rare disease or condition
(generally meaning that it affects fewer than 200,000 individuals in the United States, or more in cases in which there is no reasonable expectation that the
cost of developing and making a biologic product available in the United States for treatment of the disease or condition will be recovered from sales of the
product). Orphan product designation must be requested before submitting a BLA. After the FDA grants orphan product designation, the identity of the
therapeutic agent and its potential orphan use are disclosed publicly by the FDA. Orphan product designation does not convey any advantage in, or shorten
the duration of, the regulatory review and approval process.
If a product with orphan status receives the first FDA approval for the disease or condition for which it has such designation, the product is entitled
to orphan product exclusivity, meaning that the FDA may not approve any other applications to market the same drug or biologic product for the same
indication for seven years, except in limited circumstances, such as a showing of clinical superiority to the product with orphan exclusivity or if the party
holding the exclusivity fails to assure the availability of sufficient quantities of the drug to meet the needs of patients with the disease or condition for
which the drug was designated. This is the case despite an earlier court opinion holding that the Orphan Drug Act unambiguously required the FDA to
recognize orphan exclusivity regardless of a showing of clinical superiority.
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Orphan exclusivity does not block the approval of a different product for the same rare disease or condition, nor does it block the approval of the
same product for different indications. In particular, the concept of what constitutes the "same drug" for purposes of orphan drug exclusivity remains in
flux in the context of gene therapies, and the FDA has issued recent draft guidance suggesting that it would not consider two gene therapy products to be
different drugs solely based on minor differences in the transgenes or vectors. If a product designated as an orphan drug ultimately receives marketing
approval for an indication broader than what was designated in its orphan drug application, it may not be entitled to exclusivity. Orphan medicinal product
status in the European Union has similar, but not identical, benefits.
Expedited development and review programs
The FDA is authorized to expedite the review of BLAs in several ways. Under the Fast Track program, the sponsor of a biologic product candidate
may request the FDA to designate the product for a specific indication as a Fast Track product concurrent with or after the filing of the IND. Biologic
products are eligible for Fast Track designation if they are intended to treat a serious or life-threatening condition and demonstrate the potential to address
unmet medical needs for the condition. Fast Track designation applies to the combination of the product candidate and the specific indication for which it is
being studied. In addition to other benefits, such as the ability to have greater interactions with the FDA, the FDA may initiate review of sections of a Fast
Track BLA before the application is complete, a process known as rolling review.
Any product submitted to the FDA for marketing, including under a Fast Track program, may be eligible for other types of FDA programs intended
to expedite development and review, such as breakthrough therapy designation, priority review and accelerated approval.
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Breakthrough therapy designation. To qualify for the breakthrough therapy program, product candidates must be intended to treat a serious or
life-threatening disease or condition and preliminary clinical evidence must indicate that such product candidates may demonstrate substantial
improvement on one or more clinically significant endpoints over existing therapies. The FDA will seek to ensure the sponsor of a
breakthrough therapy product candidate receives intensive guidance on an efficient drug development program, intensive involvement of
senior managers and experienced staff on a proactive, collaborative and cross-disciplinary review and rolling review.
Priority review. A product candidate is eligible for priority review if it treats a serious condition and, if approved, it would be a significant
improvement in the safety or effectiveness of the treatment, diagnosis or prevention compared to marketed products. FDA aims to complete its
review of priority review applications within six months as opposed to 10 months for standard review.
Accelerated approval. Drug or biologic products studied for their safety and effectiveness in treating serious or life-threatening illnesses and
that provide meaningful therapeutic benefit over existing treatments may receive accelerated approval. Accelerated approval means that a
product candidate may be approved on the basis of adequate and well controlled clinical trials establishing that the product candidate has an
effect on a surrogate endpoint that is reasonably likely to predict a clinical benefit, or on the basis of an effect on a clinical endpoint other than
survival or irreversible morbidity or mortality or other clinical benefit, taking into account the severity, rarity and prevalence of the condition
and the availability or lack of alternative treatments. As a condition of approval, the FDA may require that a sponsor of a drug or biologic
product candidate receiving accelerated approval perform adequate and well controlled post-marketing clinical trials. In addition, the FDA
currently requires as a condition for accelerated approval pre-approval of promotional materials.
Regenerative advanced therapy. With passage of the 21st Century Cures Act, or the Cures Act, in December 2016, Congress authorized the
FDA to accelerate review and approval of products designated as regenerative advanced therapies. A product is eligible for this designation if
it is a regenerative medicine therapy that is intended to treat, modify, reverse or cure a serious or life-threatening disease or condition and
preliminary clinical evidence indicates that the product candidate has the potential to address unmet medical needs for such disease or
condition. The benefits of a regenerative advanced therapy designation include early interactions with the FDA to expedite development and
review, benefits available to breakthrough therapies, potential eligibility for priority review and accelerated approval based on surrogate or
intermediate endpoints.
None of these expedited programs change the standards for approval but they may help expedite the development or approval process of product
candidates.
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Post-approval requirements
After regulatory approval of a product is obtained, there may be a number of post-approval requirements. For example, as a condition of approval of
a BLA, the FDA may require post-marketing testing and surveillance to monitor the product’s safety or efficacy. In addition, holders of an approved BLA
are required to keep extensive records, to report certain adverse reactions and production problems to the FDA, to provide updated safety and efficacy
information and to comply with requirements concerning advertising and promotional labeling for their products. Also, quality control and manufacturing
procedures must continue to conform to cGMP regulations and practices, as well as the manufacturing conditions of approval set forth in the BLA. The
FDA periodically inspects manufacturing facilities to assess compliance with cGMP requirements, which impose certain procedural, substantive and
recordkeeping requirements. Accordingly, manufacturers must continue to expend time, money and effort in the area of production and quality control to
maintain compliance with cGMP and other aspects of regulatory compliance.
The FDA strictly regulates marketing, labeling, advertising and promotion of products that are placed on the market. Products may be promoted
only for the approved indications and in accordance with the provisions of the approved label. The FDA and other agencies actively enforce the laws and
regulations prohibiting the promotion of off-label uses, and a company that is found to have improperly promoted off-label uses may be subject to
significant liability. If a company is found to have promoted off-label uses, it may become subject to adverse public relations and administrative and
judicial enforcement by the FDA, the Department of Justice, or the Office of the Inspector General of the Department of Health and Human Services, as
well as state authorities. This could subject a company to a range of penalties that could have a significant commercial impact, including civil and criminal
fines and agreements that materially restrict the manner in which a company promotes or distributes drug products.
U.S. patent term restoration and marketing exclusivity
Depending upon the timing, duration and specifics of FDA approval of product candidates, some of a sponsor’s U.S. patents may be eligible for
limited patent term extension under the Drug Price Competition and Patent Term Restoration Act of 1984, or the Hatch-Waxman Amendments. The Hatch-
Waxman Amendments permit a patent restoration term of up to five years as compensation for patent terms lost during product development and FDA
regulatory review process. However, patent term restoration cannot extend the remaining term of a patent beyond a total of 14 years from the product’s
approval date. The patent term restoration period generally is one-half the time between the effective date of an IND and the submission date of a BLA plus
the time between the submission date of a BLA and the approval of that application. Only one patent applicable to an approved biologic product is eligible
for the extension, the application for the extension must be submitted prior to the expiration of the patent, and only those claims covering the approved
drug, a method for using it or a method for manufacturing it may be extended. Moreover, a given patent may only be extended once based on a single
product. The USPTO in consultation with the FDA, reviews and approves the application for any patent term extension or restoration.
Government regulation outside of the U.S.
In addition to regulations in the United States, a manufacturer is subject to a variety of regulations in foreign jurisdictions to the extent it chooses to
sell any products in those foreign countries. Even if a manufacturer obtains FDA approval of a product, it must still obtain the requisite approvals from
regulatory authorities in foreign countries prior to the commencement of clinical trials or marketing of the product in those countries. Because biologically
sourced materials are subject to unique contamination risks, their use may also be restricted in some countries.
Clinical trial approval in the European Union
Pursuant to the currently applicable Clinical Trials Directive 2001/20/EC and the Directive 2005/28/EC on Good Clinical Practice, an applicant
must obtain approval from the competent national authority of the European Union Member State, or the EU Member State, in which the clinical trial is to
be conducted. If the clinical trial is conducted in different EU Member States, the competent authorities in each of these EU Member States must provide
their approval for the conduct of the clinical trial. Furthermore, the applicant may only start a clinical trial at a specific study site after the competent ethics
committee has issued a favorable opinion. In April 2014, the European Union adopted a new Clinical Trials Regulation (EU) No 536/2014, which is set to
replace the current Clinical Trials Directive 2001/20/EC. The new Clinical Trials Regulation will be directly applicable to and binding in all EU Member
States without the need for any national implementing legislation. Clinical Trials Regulation (EU) No 536/2014 was published on June 16, 2014 but was
not expected to apply until later in 2019.
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Specifically, the new Regulation aims at simplifying and streamlining the approval of clinical trials in the European Union. Under the new
coordinated procedure for the approval of clinical trials, the sponsor of a clinical trial to be conducted in more than one EU Member State will only be
required to submit a single application for approval of a clinical trial to a reporting EU Member State. The Clinical Trials Regulation also aims to
streamline and simplify the rules on safety reporting for clinical trials. As of January 1, 2020, the website of the European Commission reported that the
implementation of the Clinical Trials Regulation was dependent on the development of a fully functional clinical trials portal and database, which would be
confirmed by an independent audit, and that the new legislation would come into effect six months after the European Commission publishes a notice of
this confirmation. The website indicated that the audit was expected to commence in December 2020.
PRIME designation
In March 2016, the EMA, launched the PRIority MEdicines, or PRIME, initiative to foster research and development of medicines that may offer a
major therapeutic advantage over existing treatments, or benefit patients without treatment options. PRIME aims to strengthen clinical trial designs to
facilitate the generation of high-quality data for the evaluation of an application for marketing authorization. To be accepted for PRIME, a medicine has to
show its potential to benefit patients with unmet medical needs based on preclinical and/or early clinical data. These medicines are considered priority
medicines within the European Union.
After an investigational candidate has been selected for PRIME, developers are assigned a rapporteur from the CHMP, to provide continuous
support and help to build knowledge ahead of a marketing authorization application, or MAA. A multidisciplinary group of experts will provide broader
guidance on the overall development plan and regulatory strategy of the product. Companies are also eligible for accelerated assessment at the time of their
regulatory application.
Marketing authorization
In the European Union, marketing authorizations for medicinal products may be obtained through several different procedures founded on the same
basic regulatory process.
The centralized procedure provides for the grant of a single marketing authorization by the European Commission that is valid across the European
Economic Area (i.e. the EU as well as Iceland, Liechtenstein and Norway). The centralized procedure is compulsory for medicinal products produced by
certain biotechnological processes, products designated as orphan medicinal products, and products with a new active substance indicated for the treatment
of certain diseases. It is optional for those products that are highly innovative or for which a centralized process is in the interest of patients. Under the
centralized procedure in the European Union, the maximum timeframe for the evaluation of an MAA is 210 days, excluding clock stops, when additional
written or oral information is to be provided by the applicant in response to questions asked by the CHMP. Accelerated evaluation may be granted by the
CHMP in exceptional cases. These are defined as circumstances in which a medicinal product is expected to be of a “major public health interest.” Three
cumulative criteria must be fulfilled in such circumstances: the seriousness of the disease, such as severely disabling or life-threatening diseases, to be
treated; the absence or insufficiency of an appropriate alternative therapeutic approach; and anticipation of high therapeutic benefit. In these circumstances,
the EMA ensures that the opinion of the CHMP is given within 150 days.
Specifically, the grant of marketing authorization in the European Union for products containing viable human tissues or cells such as gene therapy
medicinal products is governed by Regulation 1394/2007/EC on advanced therapy medicinal products, read in combination with Directive 2001/83/EC of
the European Parliament and of the Council, commonly known as the Community code on medicinal products. Regulation 1394/2007/EC lays down
specific rules concerning the authorization, supervision, and pharmacovigilance of gene therapy medicinal products, somatic cell therapy medicinal
products, and tissue engineered products. Manufacturers of advanced therapy medicinal products must demonstrate the quality, safety, and efficacy of their
products to EMA which provides an opinion regarding the application for marketing authorization. The European Commission grants or refuses marketing
authorization in light of the opinion delivered by EMA.
The decentralized procedure provides for approval by one or more other concerned EU Member States of an assessment of an application for
marketing authorization conducted by one EU Member State, known as the reference EU Member State. In accordance with this procedure, an applicant
submits an application for marketing authorization to the reference EU Member State and the concerned EU Member States. This application is identical to
the application that would be submitted to the EMA for authorization through the centralized procedure. The reference EU Member State prepares a draft
assessment and drafts of the related materials within 120 days after receipt of a valid application. The resulting assessment report is submitted to the
concerned EU Member States which, within 90 days of receipt, must decide whether to approve the assessment report and related materials.
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If a concerned EU Member State cannot approve the assessment report and related materials due to concerns relating to a potential serious risk to
public health, disputed elements may be referred to the European Commission, whose decision is binding on all EU Member States. In accordance with the
mutual recognition procedure, the sponsor applies for national marketing authorization in one EU Member State. Upon receipt of this authorization the
sponsor can then seek the recognition of this authorization by other EU Member States. Authorization in accordance with either of these procedures will
result in authorization of the medicinal product only in the reference EU Member State and in the other concerned EU Member States.
A marketing authorization may be granted only to an applicant established in the European Union. Regulation No. 1901/2006 provides that, prior to
obtaining a marketing authorization in the European Union, an applicant must demonstrate compliance with all measures included in a Pediatric
Investigation Plan, or PIP, approved by the Pediatric Committee of the EMA, covering all subsets of the pediatric population, unless the EMA has granted a
product-specific waiver, class waiver, or a deferral for one or more of the measures included in the PIP.
In specific circumstances, E.U. legislation on Conditional Marketing Authorizations for Medicinal Products for Human Use, or conditional
marketing authorization, enables applicants to obtain a conditional marketing authorization prior to obtaining the comprehensive clinical data required for
an application for a full marketing authorization. Such conditional approvals may be granted for product candidates (including medicines designated as
orphan medicinal products) if the risk-benefit balance of the product candidate is positive, it is likely that the applicant will be in a position to provide the
required comprehensive clinical trial data, the product fulfills unmet medical needs and the benefit to public health of the immediate availability on the
market of the medicinal product concerned outweighs the risk inherent in the fact that additional data are still required. A conditional marketing
authorization may contain specific obligations to be fulfilled by the marketing authorization holder, including obligations with respect to the completion of
ongoing or new studies, and with respect to the collection of pharmacovigilance data.
Conditional marketing authorization
Conditional marketing authorizations are valid for one year, and may be renewed annually, if the risk-benefit balance remains positive, and after an
assessment of the need for additional or modified conditions and/or specific obligations. The timelines for the centralized procedure described above also
apply with respect to the review by the CHMP of applications for a conditional marketing authorization.
The requirements and processes governing the conduct of clinical trials, product licensing, pricing and reimbursement vary from country to country.
In all cases, the clinical trials are conducted in accordance with GCPs and the applicable regulatory requirements of the country or countries in which the
clinical trial is performed, as well as the ethical principles that have their origin in the Declaration of Helsinki (whichever provides the greater protection to
the clinical trial participants).
Specialized Procedures for Gene Therapies
The grant of marketing authorization in the European Union for gene therapy products is governed by Regulation 1394/2007/EC on advanced
therapy medicinal products, read in combination with Directive 2001/83/EC of the European Parliament and of the Council, commonly known as the
Community code on medicinal products. Regulation 1394/2007/EC includes specific rules concerning the authorization, supervision, and
pharmacovigilance of gene therapy medicinal products. Manufacturers of advanced therapy medicinal products must demonstrate the quality, safety, and
efficacy of their products to the EMA, which provides an opinion regarding the MAA. The European Commission grants or refuses marketing
authorization in light of the opinion delivered by the EMA.
General Data Protection Regulation
The collection, use, disclosure, transfer, or other processing of personal data regarding individuals in the EU, including personal health data, is
subject to the EU General Data Protection Regulation, or the GDPR, which became effective on May 25, 2018. The GDPR is wide-ranging in scope and
imposes numerous requirements on companies that process personal data, including requirements relating to processing health and other sensitive data,
obtaining consent of the individuals to whom the personal data relates, providing information to individuals regarding data processing activities,
implementing safeguards to protect the security and confidentiality of personal data, providing notification of data breaches, and taking certain measures
when engaging third-party processors. The GDPR also imposes strict rules on the transfer of personal data to countries outside the EU, including the U.S.,
and permits data protection authorities to impose large penalties for violations of the GDPR, including potential fines of up to €20 million or 4% of annual
global revenues, whichever is greater. The GDPR also confers a private right of action on data subjects and consumer associations to lodge complaints with
supervisory authorities, seek judicial remedies, and obtain compensation for damages resulting from violations of the GDPR. Compliance with the GDPR
will be a rigorous and time-intensive process that may increase the cost of doing business or require companies to change their business practices to ensure
full compliance.
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Brexit and the Regulatory Framework in the United Kingdom
On June 23, 2016, the electorate in the United Kingdom voted in favor of leaving the European Union, commonly referred to as Brexit. Following
protracted negotiations, the United Kingdom left the European Union on January 31, 2020. Under the withdrawal agreement, there is a transitional period
until December 31, 2020 (extendable up to two years). Discussions between the United Kingdom and the European Union have so far mainly focused on
finalizing withdrawal issues and transition agreements but have been extremely difficult to date. To date, only an outline of a trade agreement has been
reached. Much remains open but the Prime Minister has indicated that the United Kingdom will not seek to extend the transitional period beyond the end
of 2020. If no trade agreement has been reached before the end of the transitional period, there may be significant market and economic disruption. The
Prime Minister has also indicated that the UK will not accept high regulatory alignment with the EU.
Since the regulatory framework for pharmaceutical products in the United Kingdom covering quality, safety, and efficacy of pharmaceutical
products, clinical trials, marketing authorization, commercial sales, and distribution of pharmaceutical products is derived from European Union directives
and regulations, Brexit could materially impact the future regulatory regime that applies to products and the approval of product candidates in the United
Kingdom. Any delay in obtaining, or an inability to obtain, any marketing approvals, as a result of Brexit or otherwise, may force us to restrict or delay
efforts to seek regulatory approval in the United Kingdom and/or European Union for our product candidates, which could significantly and materially
harm our business.
Healthcare law and regulation
Healthcare providers and third-party payors play a primary role in the recommendation and prescription of products that are granted marketing
approval. Arrangements with providers, consultants, third-party payors and customers are subject to broadly applicable fraud and abuse, anti-kickback,
false claims laws, reporting of payments to physicians and teaching physicians and patient privacy laws and regulations and other healthcare laws and
regulations that may constrain business and/or financial arrangements. Restrictions under applicable federal and state healthcare laws and regulations,
include the following:
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the federal Anti-Kickback Statute, which prohibits, among other things, persons and entities from knowingly and willfully soliciting, offering,
paying, receiving or providing remuneration, directly or indirectly, in cash or in kind, to induce or reward either the referral of an individual
for, or the purchase, order or recommendation of, any good or service, for which payment may be made, in whole or in part, under a federal
healthcare program such as Medicare and Medicaid;
the federal civil and criminal false claims laws, including the civil False Claims Act, and civil monetary penalties laws, which prohibit
individuals or entities from, among other things, knowingly presenting, or causing to be presented, to the federal government, claims for
payment that are false, fictitious or fraudulent or knowingly making, using or causing to made or used a false record or statement to avoid,
decrease or conceal an obligation to pay money to the federal government;
the federal Health Insurance Portability and Accountability Act of 1996, or HIPAA, which created additional federal criminal laws that
prohibit, among other things, knowingly and willfully executing, or attempting to execute, a scheme to defraud any healthcare benefit program
or making false statements relating to healthcare matters;
HIPAA, as amended by the Health Information Technology for Economic and Clinical Health Act, and their respective implementing
regulations, including the Final Omnibus Rule published in January 2013, which impose obligations, including mandatory contractual terms,
with respect to safeguarding the privacy, security and transmission of individually identifiable health information;
the federal false statements statute, which prohibits knowingly and willfully falsifying, concealing or covering up a material fact or making
any materially false statement in connection with the delivery of or payment for healthcare benefits, items or services;
the federal transparency requirements known as the federal Physician Payments Sunshine Act, under the Health Care Reform Law, which
requires certain manufacturers of drugs, devices, biologics and medical supplies to report annually to the Centers for Medicare & Medicaid
Services, or CMS, within the United States Department of Health and Human Services, information related to payments and other transfers of
value made by that entity to physicians and teaching hospitals, as well as ownership and investment interests held by physicians and their
immediate family members; and
analogous state and foreign laws and regulations, such as state anti-kickback and false claims laws, which may apply to healthcare items or
services that are reimbursed by non-governmental third-party payors, including private insurers.
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Some state laws require pharmaceutical companies to comply with the pharmaceutical industry’s voluntary compliance guidelines and the relevant
compliance guidance promulgated by the federal government in addition to requiring manufacturers to report information related to payments to physicians
and other health care providers or marketing expenditures. State and foreign laws also govern the privacy and security of health information in some
circumstances, many of which differ from each other in significant ways and often are not preempted by HIPAA, thus complicating compliance efforts.
Pharmaceutical insurance coverage and health care reform
In the United States and markets in other countries, patients who are prescribed treatments for their conditions and providers performing the
prescribed services generally rely on third-party payors to reimburse all or part of the associated health care costs. Significant uncertainty exists as to the
coverage and reimbursement status of products approved by the FDA and other government authorities. Thus, even if a product candidate is approved,
sales of the product will depend, in part, on the extent to which third-party payors, including government health programs in the United States such as
Medicare and Medicaid, commercial health insurers and managed care organizations, provide coverage and establish adequate reimbursement levels for,
the product. The process for determining whether a payor will provide coverage for a product may be separate from the process for setting the price or
reimbursement rate that the payor will pay for the product once coverage is approved. Third-party payors are increasingly challenging the prices charged,
examining the medical necessity and reviewing the cost-effectiveness of medical products and services and imposing controls to manage costs. Third-party
payors may limit coverage to specific products on an approved list, also known as a formulary, which might not include all of the approved products for a
particular indication.
The Trump Administration has also taken executive actions to undermine or delay implementation of the Health Care Reform Law. Since January
2017, President Trump has signed two Executive Orders designed to delay the implementation of certain provisions of the Health Care Reform Law or
otherwise circumvent some of the requirements for health insurance mandated by the Health Care Reform Law. One Executive Order directs federal
agencies with authorities and responsibilities under the Health Care Reform Law to waive, defer, grant exemptions from, or delay the implementation of
any provision of the Health Care Reform Law that would impose a fiscal or regulatory burden on states, individuals, healthcare providers, health insurers,
or manufacturers of pharmaceuticals or medical devices. The second Executive Order terminates the cost-sharing subsidies that reimburse insurers under
the Health Care Reform Law. Several state Attorneys General filed suit to stop the administration from terminating the subsidies, but their request for a
restraining order was denied by a federal judge in California on October 25, 2017. In addition, CMS has recently proposed regulations that would give
states greater flexibility in setting benchmarks for insurers in the individual and small group marketplaces, which may have the effect of relaxing the
essential health benefits required under the Health Care Reform Law for plans sold through such marketplaces. Further, on June 14, 2018, U.S. Court of
Appeals for the Federal Circuit ruled that the federal government was not required to pay more than $12 billion in Health Care Reform Law risk corridor
payments to third-party payors who argued were owed to them. This decision is under review by the U.S. Supreme Court during its current term. The full
effect of this gap in reimbursement on third-party payors, the viability of the Health Care Reform Law marketplace, providers, and potentially our business,
are not yet known.
More recently, with enactment of the Tax Cuts and Jobs Act of 2017, which was signed by the President on December 22, 2017, Congress repealed
the “individual mandate.” The repeal of this provision, which requires most Americans to carry a minimal level of health insurance, will become effective
in 2019. According to the Congressional Budget Office, the repeal of the individual mandate will cause 13 million fewer Americans to be insured in 2027
and premiums in insurance markets may rise. Additionally, the 2020 federal spending package permanently eliminated, effective January 1, 2020, the
Health Care Reform Law-mandated “Cadillac” tax on high-cost employer-sponsored health coverage and medical device tax and, effective January 1,
2021, also eliminates the health insurer tax. Further, the Bipartisan Budget Act of 2018, among other things, amended the Health Care Reform Law,
effective January 1, 2019, to increase from 50 percent to 70 percent the point-of-sale discount that is owed by pharmaceutical manufacturers who
participate in Medicare Part D and to close the coverage gap in most Medicare drug plans, commonly referred to as the “donut hole.” The Congress may
consider other legislation to replace elements of the Health Care Reform Law during the next Congressional session.
In addition, on December 14, 2018, a U.S. District Court judge in the Northern District of Texas ruled that the individual mandate portion of the
Health Care Reform Law is an essential and inseverable feature of the Health Care Reform Law, and therefore because the mandate was repealed as part of
the Tax Cuts and Jobs Act, the remaining provisions of the Health Care Reform Law are invalid as well. The Trump administration and CMS have both
stated that the ruling will have no immediate effect, and on December 30, 2018 the same judge issued an order staying the judgment pending appeal. The
Trump Administration recently represented to the Court of Appeals considering this judgment that it does not oppose the lower court’s ruling. On July 10,
2019, the Court of Appeals for the Fifth Circuit heard oral argument in this case. On December 18, 2019, that court affirmed the lower court’s ruling that
the individual mandate portion of the Health Care Reform Law is unconstitutional and it remanded the case to the district court for reconsideration of the
severability question and additional analysis of the provisions of the Health Care Reform Law. On January 21, 2020, the U.S. Supreme Court declined to
review this decision on an expedited basis. Litigation and legislation over the Health Care Reform Law are likely to continue, with unpredictable and
uncertain results.
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Further, there have been several recent U.S. congressional inquiries and proposed federal and proposed and enacted state legislation designed to,
among other things, bring more transparency to drug pricing, review the relationship between pricing and manufacturer patient programs, reduce the costs
of drugs under Medicare and reform government program reimbursement methodologies for drug products. For example, there have been several recent
U.S. congressional inquiries and proposed federal and proposed and enacted state legislation designed to, among other things, bring more transparency to
drug pricing, review the relationship between pricing and manufacturer patient programs, reduce the costs of drugs under Medicare and reform government
program reimbursement methodologies for drug products. At the federal level, Congress and the Administration have each indicated that it will continue to
seek new legislative and/or administrative measures to control drug costs. For example, on May 11, 2018, the Administration issued a plan to lower drug
prices. Under this blueprint for action, the Administration indicated that the Department of Health and Human Services will: take steps to end the gaming
of regulatory and patent processes by drug makers to unfairly protect monopolies; advance biosimilars and generics to boost price competition; evaluate the
inclusion of prices in drug makers’ ads to enhance price competition; speed access to and lower the cost of new drugs by clarifying policies for sharing
information between insurers and drug makers; avoid excessive pricing by relying more on value-based pricing by expanding outcome-based payments in
Medicare and Medicaid; work to give Part D plan sponsors more negotiation power with drug makers; examine which Medicare Part B drugs could be
negotiated for a lower price by Part D plans, and improving the design of the Part B Competitive Acquisition Program; update Medicare’s drug-pricing
dashboard to increase transparency; prohibit Part D contracts that include “gag rules” that prevent pharmacists from informing patients when they could
pay less out-of-pocket by not using insurance; and require that Part D plan members be provided with an annual statement of plan payments, out-of-pocket
spending, and drug price increases. In addition, on December 23, 2019, the Trump Administration published a proposed rulemaking that, if finalized, would
allow states or certain other non-federal government entities to submit importation program proposals to FDA for review and approval. Applicants would
be required to demonstrate their importation plans pose no additional risk to public health and safety and will result in significant cost savings for
consumers. At the same time, FDA issued draft guidance that would allow manufacturers to import their own FDA-approved drugs that are authorized for
sale in other countries (multi-market approved products).
At the state level, individual states are increasingly aggressive in passing legislation and implementing regulations designed to control
pharmaceutical and biological product pricing, including price or patient reimbursement constraints, discounts, restrictions on certain product access and
marketing cost disclosure and transparency measures, and, in some cases, designed to encourage importation from other countries and bulk purchasing. In
addition, regional health care authorities and individual hospitals are increasingly using bidding procedures to determine what pharmaceutical products and
which suppliers will be included in their prescription drug and other health care programs. These measures could reduce the ultimate demand for our
products, once approved, or put pressure on our product pricing. We expect that additional state and federal healthcare reform measures will be adopted in
the future, any of which could limit the amounts that federal and state governments will pay for healthcare products and services, which could result in
reduced demand for our product candidates or additional pricing pressures.
Environmental regulations
We are also subject to regulation under the Occupational Safety and Health Act, the Environmental Protection Act, the Toxic Substances Control
Act, the Resource Conservation and Recovery Act and other present and potential federal, state or local regulations. These and other laws govern our use,
handling and disposal of various biological and chemical substances used in, and waste generated by, our operations. Our research and development
involves the controlled use of hazardous materials, chemicals and viruses.
Employees
As of December 31, 2019, we employed 121 full-time employees, including 97 in research and development and 24 in general and administrative
positions. 34 of our employees hold Ph.D. or M.D. degrees.
In January 2020, we announced a reduction in workforce by approximately one third as part of a strategic plan designed to create a leaner company
focused on advancing SGT-001.
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Corporate Information
We were originally formed as SOLID Ventures Management, LLC in March 2013 as a Delaware limited liability company. In October 2013, we
changed our name to Solid Ventures, LLC and in June 2015 we changed our name to Solid Biosciences, LLC. Immediately prior to the effectiveness of our
registration statement on Form S-1 on January 25, 2018, we converted into a Delaware corporation pursuant to a statutory conversion and changed our
name to Solid Biosciences Inc. In addition, immediately following the statutory conversion, entities formed solely for the purpose of holding membership
interests in our limited liability company were merged with and into us. We refer to the corporate conversion and the mergers collectively as the Corporate
Conversion. Our principal executive offices are located at 141 Portland Street, Fifth Floor, Cambridge, Massachusetts 02139 and our telephone number is
(617) 337-4680. Our website address is www.solidbio.com. The information contained in, or accessible through, our website does not constitute a part of
this Annual Report on Form 10-K.
We make available free of charge on our website our Annual Reports on Form 10-K, Quarterly Reports on Form 10-Q, Current Reports on Form 8-
K and amendments to those reports filed or furnished pursuant to Section 13(a) or 15(d) of the Exchange Act, as soon as reasonably practicable after we
electronically file such material with, or furnish it to, the SEC.
Item 1A.
Risk Factors.
You should carefully consider the following risk factors, in addition to the other information contained in this annual report on Form 10-K,
including the section of this report titled “Management’s Discussion and Analysis of Financial Condition and Results of Operations” and our financial
statements and related notes. If any of the events described in the following risk factors and the risks described elsewhere in this annual report on Form 10-
K occurs, our business, operating results and financial condition could be seriously harmed and the trading price of our common stock could decline. This
annual report on Form 10-K also contains forward-looking statements that involve risks and uncertainties. Our actual results could differ materially from
those anticipated in the forward-looking statements as a result of factors that are described below and elsewhere in this annual report on Form 10-K.
Risks related to our financial position and need for capital requirements
We have incurred significant net losses since inception and anticipate that we will continue to incur net losses for the foreseeable future and may never
achieve or maintain profitability.
Since inception, we have incurred significant net losses. Our net losses were $117.2 million, $74.8 million and $53.2 million for the years ended
December 31, 2019, 2018 and 2017, respectively. As of December 31, 2019, we had an accumulated deficit of $316.3 million. To date, we have devoted
substantially all of our efforts to research and development, including clinical development of our gene transfer product candidate, SGT-001, as well as to
building out our management team and infrastructure. We expect that it could be several years, if ever, before we have a commercialized product. We
expect to continue to incur significant expenses and increasing operating losses for the foreseeable future. We anticipate that our expenses will increase
substantially if, and as, we:
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seek to resolve the clinical hold on and determine next steps for IGNITE DMD and, if and when possible, resume our clinical development of
SGT-001;
move other product candidates into clinical trials;
continue research and preclinical development of other product candidates;
seek to identify additional product candidates;
seek marketing approvals for our product candidates that successfully complete clinical trials, if any;
establish a sales, marketing and distribution infrastructure to commercialize any products for which we may obtain marketing approval;
arrange for manufacture of larger quantities of our product candidates for clinical development and potential commercialization;
maintain, expand, protect and enforce our intellectual property portfolio;
hire and retain additional clinical, quality control and scientific personnel;
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build out new facilities or expand existing facilities to support our activities;
acquire or in-license other drugs, technologies and intellectual property; and
add operational, financial and management information systems and personnel.
To become and remain profitable, we must develop and eventually commercialize one or more product candidates with significant market potential.
This will require us to be successful in a range of challenging activities, and our expenses will increase substantially as we seek to resolve the clinical hold,
resume IGNITE DMD and complete clinical trials of SGT-001, obtain marketing approval for SGT-001, develop and validate commercial-scale
manufacturing processes, manufacture, market and sell any future product candidates for which we may obtain marketing approval and satisfy any post-
marketing requirements. We may never succeed in any or all of these activities and, even if we do, we may never generate revenue that is significant or
large enough to achieve profitability. If we do achieve profitability, we may not be able to sustain or increase profitability on a quarterly or annual basis.
Our failure to become and remain profitable would decrease the value of our company and could impair our ability to raise capital, maintain our research
and development efforts, expand our business or continue our operations. A decline in the value of our company also could cause stockholders to lose all or
part of their investment.
We will need additional funding, which may not be available on acceptable terms, or at all. Failure to obtain this necessary capital when needed may
force us to delay, limit or terminate our product development efforts or other operations.
We expect our expenses to increase in connection with our ongoing activities, particularly as we continue the research and development of, conduct
clinical trials of, and seek marketing approval for, SGT-001 and our other product candidates. In addition, if we obtain marketing approval for SGT-001 and
our other product candidates, we expect to incur significant expenses related to product sales, marketing, manufacturing and distribution. We also incur
additional costs associated with operating as a public company. While we believe that our cash, cash equivalents and available-for-sale securities as of
December 31, 2019 will enable us to fund our operating expenses and capital expenditure requirements into 2021, we have based this estimate on
assumptions that may prove to be wrong, and we could use our available capital resources sooner than we currently anticipate. In addition, we anticipate
that we will need additional funding to complete the development of SGT-001 and our other product candidates.
Our future capital requirements will depend on many factors, including:
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if and when we are able to favorably resolve the clinical hold on and resume IGNITE DMD;
the progress and results of IGNITE DMD and future clinical trials of SGT-001 and our other product candidates;
the costs, timing and outcome of regulatory review of SGT-001 and our other product candidates;
the scope, progress, results and costs of discovery, laboratory testing, manufacturing, preclinical development and clinical trials for other
product candidates that we may pursue in the future, if any;
the costs associated with our manufacturing process development and evaluation of third-party manufacturers;
whether we decide to construct and validate our own manufacturing facility and the associated costs;
revenue, if any, received from commercial sale of SGT-001 or our other product candidates, should any of our product candidates receive
marketing approval;
the costs of preparing, filing and prosecuting patent applications, maintaining, defending and enforcing our intellectual property rights and
defending intellectual property-related claims;
the outcome of any lawsuits filed against us;
the terms of our current and any future license agreements and collaborations; and
the extent to which we acquire or in-license other product candidates, technologies and intellectual property.
Identifying potential product candidates and conducting preclinical testing and clinical trials is a time-consuming, expensive and uncertain process
that takes years to complete, and we may never generate the necessary data or results required to obtain marketing approval and achieve product sales. In
addition, our product candidates, if approved, may not achieve commercial success. Our product revenue, if any, will be derived from or based on sales of
product candidates that may not be commercially available for many years, if at all. Accordingly, we will need to continue to rely on additional financing to
achieve our business objectives. Adequate additional financing may not be available to us on acceptable terms, or at all.
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Raising additional capital may cause dilution to our existing stockholders, restrict our operations or require us to relinquish rights to our technologies,
SGT-001 or our other product candidates.
We may seek additional capital through a combination of public and private equity offerings, debt financings, strategic partnerships and alliances
and licensing arrangements. To the extent that we raise additional capital through the sale of equity or convertible debt securities, ownership of our
common stock will be diluted and the terms may include liquidation or other preferences that adversely affect the rights of our current stockholders. The
incurrence of indebtedness would result in increased fixed payment obligations and could involve restrictive covenants, such as limitations on our ability to
incur additional debt, limitations on our ability to acquire or license intellectual property rights and other operating restrictions that could adversely impact
our ability to conduct our business. If we raise additional funds through strategic partnerships and alliances and licensing arrangements with third parties,
we may have to relinquish valuable rights to our technologies, SGT-001 or our other product candidates, or grant licenses on terms unfavorable to us.
We have never generated revenue from product sales and do not expect to do so for the next several years, if ever.
Our ability to generate revenue from product sales and achieve profitability depends on our ability, alone or with collaborative partners, to
successfully complete the development of, and obtain the regulatory approvals necessary to commercialize, SGT-001 and any other product candidates that
we may pursue in the future. We do not anticipate generating revenue from product sales for the next several years, if ever. Our ability to generate future
revenue from product sales depends heavily on our success in:
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favorably resolving the clinical trial hold on the Phase I/II clinical trial for SGT-001;
completing research and development of SGT-001 and our other product candidates in a timely and successful manner;
seeking and obtaining regulatory and marketing approvals for any product candidates for which we complete clinical trials;
launching and commercializing SGT-001 and any other product candidates for which we obtain regulatory and marketing approval by
establishing a sales force and marketing and distribution infrastructure or, alternatively, collaborating with a commercialization partner;
maintaining and enhancing a commercially viable, sustainable, scalable, reproducible and transferable manufacturing process for SGT-001 and
our other product candidates that is compliant with cGMPs;
establishing and maintaining supply and manufacturing relationships with third parties that can provide adequate, in both amount and quality,
products and services to support clinical development and the commercial demand for SGT-001 and our other product candidates, if approved;
obtaining market acceptance, if and when approved, of SGT-001 or any other product candidate as a viable treatment option by patients, the
medical community and third-party payors;
qualifying for coverage and adequate reimbursement by government and third-party payors for SGT-001 and our other product candidates
both in the U.S. and internationally;
effectively addressing any competing technological and market developments;
negotiating favorable terms in any collaboration, licensing or other arrangements into which we may enter and performing our obligations
under such arrangements;
maintaining, protecting, enforcing and expanding our portfolio of intellectual property rights, including patents, trademarks, trade secrets and
know-how;
avoiding and defending against intellectual property infringement, misappropriation and other claims;
implementing additional internal systems and infrastructure, as needed; and
attracting, hiring and retaining qualified personnel.
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Our limited operating history may make it difficult for our stockholders to evaluate the success of our business to date and to assess our future viability.
We are a development-stage company founded in 2013. Our operations to date, with respect to the development of SGT-001 and other potential
product candidates, have been limited to organizing and staffing our company, business planning, raising capital, acquiring rights to our technology,
identifying SGT-001 as a potential gene transfer product candidate and undertaking preclinical studies and a clinical trial of that product candidate and
establishing research and development and manufacturing collaborations. We have not yet demonstrated the ability to complete clinical trials of SGT-001
or any other product candidate, obtain marketing approvals, manufacture a commercial-scale product or conduct sales and marketing activities necessary
for successful commercialization. Consequently, any predictions our stockholders make about our prospects may not be as accurate as they could be if we
had a longer operating history.
Our independent registered public accounting firm has included an explanatory paragraph relating to our ability to continue as a going concern in its
report on our audited financial statements included in our Annual Report on Form 10-K.
The report from our independent registered public accounting firm for the year ended December 31, 2019 includes an explanatory paragraph stating
that our losses from operations and required additional funding to finance our operations raise substantial doubt about our ability to continue as a going
concern. If we are unable to obtain sufficient funding, our business, prospects, financial condition and results of operations will be materially and adversely
affected and we may be unable to continue as a going concern. If we are unable to continue as a going concern, we may have to liquidate our assets and
may receive less than the value at which those assets are carried on our audited financial statements, and it is likely that investors will lose all or a part of
their investment. If we seek additional financing to fund our business activities in the future and there remains substantial doubt about our ability to
continue as a going concern, investors or other financing sources may be unwilling to provide additional funding to us on commercially reasonable terms or
at all.
Risks related to the development of our product candidates
SGT-001 is a gene transfer candidate based on a novel technology, which makes it difficult to predict the time and cost of development and of
subsequently obtaining regulatory approval. To our knowledge, a limited number of gene transfer products have been approved for commercialization
in the United States and the European Union.
We have concentrated our research and development efforts on SGT-001 for the treatment of DMD and our future success depends on our
successful development of that product candidate. Our risk of failure is high. We have experienced, and may in the future experience, problems or delays in
developing SGT-001. Any such problems or delays would cause unanticipated costs, and any development problems may not be solved. For example, we
or another party may uncover a previously unknown risk associated with SGT-001, the adeno-associated virus, or AAV, vector, toxicity or other issues that
may be more problematic than we currently believe and this may prolong the period of observation required for obtaining, or result in the failure to obtain,
regulatory approval or may necessitate additional clinical testing.
In addition, the product specifications and the clinical trial requirements of the FDA, the European Commission, the European Medicines Agency,
or the EMA, and other regulatory authorities and the criteria these regulators use to determine the safety and efficacy of a product candidate vary
substantially according to the type, complexity, novelty and intended use and market of such product candidate. The regulatory approval process for novel
product candidates such as ours is unclear and can be more expensive and take longer than for other, better known or more extensively studied product
candidates. To our knowledge, a limited number of gene transfer products have been approved for commercialization in the United States and the European
Union. As a result, it is difficult to determine how long it will take or how much it will cost to obtain regulatory approvals for SGT-001 in either the United
States or the European Union. Approvals by the European Commission may not be indicative of what the FDA may require for approval and vice versa.
The FDA has placed IGNITE DMD on clinical hold after we reported a serious adverse event in the clinical trial. We may not conduct any clinical
trials of SGT-001 until such clinical hold is lifted by the FDA. Our business may be adversely affected if the clinical hold is not timely and favorably
resolved or if such regulatory concerns lead to more burdensome preclinical studies or clinical trials that cause significant delays in or end
development of SGT-001.
In November 2019, the FDA placed a clinical hold on SGT-001 following a serious adverse event in our Phase I/II clinical trial, called IGNITE
DMD. The third patient in the 2E14 vg/kg cohort of IGNITE DMD, dosed in late October 2019, experienced a serious adverse event deemed related to the
study drug that was characterized by complement activation, thrombocytopenia, decrease in red blood cell count, acute kidney injury, and cardio-
pulmonary insufficiency. We reported the event to the FDA and the study Data Safety Monitoring Board. Following notification, the FDA placed IGNITE
DMD on
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clinical hold. We continue to make progress to address the clinical hold and determine the path forward for SGT-001. We cannot assure our stockholders
that the FDA will lift the clinical hold or that we will be able to resume development of SGT-001 as planned, or at all. Further, even if the FDA lifts the
clinical hold, or if the FDA or other regulatory agencies continue to express safety concerns even after the hold is lifted, additional preclinical studies or
clinical trials involving SGT-001, further amendments to the SGT-001 enrollment criteria and/or clinical trial protocol or changes to our manufacturing
process may be needed and difficult to implement and/or complete. In such instance, our progress in the development of SGT-001 may be significantly
slowed or stopped and the associated costs may be significantly increased, adversely affecting our business, and our stock price would likely decline.
In addition, we may not be able to obtain institutional review board committee, or IRB, or data safety monitoring board committee approvals for
IGNITE DMD as a result of the clinical hold or any related risks, which could further delay our ability to open new trial sites and enroll patients into the
clinical trial. Any delay in enrolling patients or inability to continue or complete our clinical trial of SGT-001, as a result of the clinical hold or otherwise,
will delay or terminate our clinical development plans for SGT-001, may require us to incur additional clinical development costs and could impair our
ability to ultimately obtain FDA approval for SGT-001. Delays in the completion of any clinical trial of SGT-001, our lead product candidate, or any other
product candidate will increase our costs, slow down our product candidate development and approval process and delay or potentially jeopardize our
ability to commence product sales and generate revenue. In addition, many of the factors that cause, or lead to, a delay in the commencement or completion
of clinical trials may also ultimately lead to the denial of regulatory approval of SGT-001 or our other product candidates.
Our product candidates may cause undesirable side effects or have other properties that could delay or prevent their regulatory approval, limit their
commercial potential or result in significant negative consequences following any potential marketing approval.
During the conduct of clinical trials, patients may experience changes in their health, including illnesses, injuries, discomforts or a fatal outcome.
Often, it is not possible to determine whether the product candidate being studied caused these conditions. For instance, recently we reported a serious
adverse event in IGNITE DMD, which resulted in a clinical hold, and previously IGNITE DMD has been on clinical hold after reporting another serious
adverse event. In addition, it is possible that as we test SGT-001 or our other product candidates in larger, longer and more extensive clinical programs, or
as use of these product candidates becomes more widespread if they receive regulatory approval, illnesses, injuries, discomforts and other adverse events
that were observed in earlier clinical trials, as well as conditions that did not occur or went undetected in previous clinical trials, will be reported by
subjects. Many times, side effects are only detectable after investigational products are tested in large-scale, Phase III clinical trials or, in some cases, after
they are made available to patients on a commercial scale after approval. If additional clinical experience indicates that SGT-001 or any other product
candidate has side effects or causes serious or life-threatening side effects, the development of the product candidate may fail or be delayed, or, if the
product candidate has received regulatory approval, such approval may be revoked.
There have been several significant adverse side effects in gene therapy treatments in the past, including reported cases of leukemia and death seen
in other clinical trials using other vectors. While new recombinant vectors have been developed with the intent to reduce these side effects, gene therapy is
still a relatively new approach to disease treatment and additional adverse side effects could develop. Patients will create antibodies to the AAV vector and
a second administration of gene transfer might not be successful. There also is the potential risk of delayed adverse events following exposure to gene
therapy products due to persistent biologic activity of the genetic material or other components of products used to carry the genetic material. Possible
adverse side effects that may occur with treatment with gene therapy products include an immunologic reaction early after administration that could
substantially limit the effectiveness of the treatment or represent safety risks for patients. Additionally, in previous clinical trials involving AAV vectors for
gene therapy, some subjects experienced the development of a positive ELISPOT test associated with T-cell responses, which is of unclear clinical
translatability. If T-cells are activated, the cellular immune response system may trigger the removal of transduced cells. If our gene transfer candidate
demonstrates a similar effect, we may decide or be required to halt or delay further clinical development of SGT-001.
As part of our preclinical program, we performed necessary good laboratory practices, or GLP, toxicology studies to establish the overall safety
profile of SGT-001 in wild-type mice and non-human primates, or NHPs. The data and our conclusions from these studies were included in our IND
submission to the FDA. Systemic administration of SGT-001 was generally well tolerated in both species. We observed no evidence of test-article-related
toxicity for up to 13 weeks after systemic administration of SGT-001 in either species that would prevent us from initiating clinical trials. In the NHP study,
test-article-related effects were self-limited, mild chemistry and hematology changes with no microscopic correlates at the end of the study. There was a
transient and asymptomatic increase in liver function enzymes observed in NHPs starting on
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day 9, which returned to normal levels by day 21. We believe there were no other relevant test-article-related adverse events associated with SGT-001
administration in either GLP study. In the NHP toxicology study, a single animal from the high dose cohort was euthanized after it did not recover from an
anesthetic procedure. We believe this event was attributed to procedural errors. However, AAV vector cannot be completely ruled out as a contributing
factor to the toxicity that gave rise to the event.
In addition to side effects caused by SGT-001 and our other product candidates, the administration process or related procedures also can cause
adverse side effects. For example, integration of AAV DNA into the host cell’s genome has been reported to occur. Further, our AAV delivery system has
not been validated in human clinical trials previously, and if such delivery system does not meet the safety criteria or cannot provide the desired efficacy
results, then we may be forced to suspend or terminate our development of SGT-001. In addition, the relatively high dosing requirements for SGT-001 may
amplify the risk of adverse side effects relating to the AAV vector.When James M. Wilson, M.D., Ph.D., resigned from our Scientific Advisory Board he
cited emerging concerns about the possible risks of high systemic dosing of AAV. If any such adverse side effects were to occur in the future and we are
unable to demonstrate that they were not caused by the administration process or related procedures, the FDA, the European Commission, the EMA or
other regulatory authorities could order us to cease further development of, or deny approval of, SGT-001 or any other product candidate for any or all
targeted indications. Even if we are able to demonstrate that any serious adverse events are not product-related, such occurrences could affect patient
recruitment or the ability of enrolled patients to complete the clinical trial.
Additionally, if SGT-001 or our other product candidates receive marketing approval, the FDA could require us to adopt a Risk Evaluation and
Mitigation Strategy, or REMS, to ensure that the benefits outweigh the risks, which may include, among other things, a medication guide outlining the risks
of the product for distribution to patients and a communication plan to health care practitioners. Furthermore, if we or others later identify undesirable side
effects caused by SGT-001 or our other product candidates, several potentially significant negative consequences could result, including:
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regulatory authorities may suspend or withdraw approvals of such a product candidate;
regulatory authorities may require additional warnings on the label;
we may be required to change the way a product candidate is administered or conduct additional clinical trials;
we could be sued and held liable for harm caused to patients; and
our reputation may suffer.
We have never completed a clinical trial, and may be unable to do so for any product candidates we may develop, including SGT-001.
We will need to successfully complete clinical trials in order to obtain FDA approval to market SGT-001 or our other product candidates. We have
limited experience in preparing, submitting and prosecuting regulatory filings, and have not previously submitted a biologics license application, or BLA,
for any product candidate. In November 2019, the FDA placed SGT-001 on a clinical hold. If the clinical hold is not lifted on our IGNITE DMD clinical
trial, we will not be able to evaluate or fully evaluate the safety, tolerability and efficacy of SGT-001.We cannot be sure that submission of an IND will
result in the FDA allowing clinical trials to begin or to begin as proposed, or that, once begun, issues will not arise that suspend or terminate such clinical
trials. Carrying out later-stage clinical trials and the submission of a successful BLA is a complicated process. This may be particularly true for design of a
pivotal trial for the treatment of DMD as the FDA has not given clear guidance as to the necessary endpoints for approval of a treatment for DMD. In
addition, we have had limited interactions with the FDA and cannot be certain how many clinical trials of SGT-001 or our other product candidates will be
required or how such trials should be designed. Consequently, we may be unable to successfully and efficiently execute and complete necessary clinical
trials in a way that leads to BLA submission and approval of SGT-001 or our other product candidates. We may require more time and incur greater costs
than our competitors and may not succeed in obtaining regulatory approvals of product candidates that we develop. Failure to commence or complete, or
delays in, clinical trials, could prevent us from or delay us in commercializing SGT-001 and our other product candidates.
In the past, we have made changes to the IGNITE DMD protocol, and these changes, and any other such changes that may be made in the future,
may impact our development timeline and result in increased costs and expenses.
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Success in preclinical studies or early clinical trials, including our IGNITE DMD clinical trial, may not be indicative of results obtained in later trials.
Results from preclinical studies or early clinical trials, including our IGNITE DMD clinical trial, are not necessarily predictive of future clinical
trial results and are not necessarily indicative of final results. There is a high failure rate for gene therapy and biologic products proceeding through clinical
trials. Many companies in the pharmaceutical and biotechnology industries have suffered significant setbacks in late-stage clinical trials even after
achieving promising results in preclinical testing and earlier-stage clinical trials. Data obtained from preclinical and clinical activities are subject to varying
interpretations, which may delay, limit or prevent regulatory approval. We also may experience regulatory delays or rejections as a result of many factors,
including due to changes in regulatory policy during the period of our product candidate development. Our preclinical studies for SGT-001 in animals have
been limited and we have only recently begun dosing human patients with SGT-001. SGT-001 or our other product candidates may fail to show the desired
safety and efficacy in clinical development despite positive results in preclinical studies. This failure would cause us to abandon SGT-001 or our other
product candidates.
We may encounter substantial delays in our clinical trials or we may fail to demonstrate safety and efficacy to the satisfaction of applicable regulatory
authorities.
Before obtaining marketing approval from regulatory authorities for the sale of SGT-001 or our other product candidates, we must conduct
extensive clinical trials to demonstrate the safety and efficacy of the product candidate for its intended indications. Clinical testing is expensive, time-
consuming and uncertain as to outcome. We cannot guarantee that any clinical trials will be conducted as planned or completed on schedule, if at all. A
failure of one or more clinical trials can occur at any stage of testing. Events that may prevent successful or timely completion of clinical development
include:
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delays in reaching a consensus with regulatory authorities on trial design;
delays in or the inability to successfully resolve the clinical hold placed on IGNITE DMD for SGT-001;
delays in reaching agreement with the appropriate external parties on dose escalation;
delays in enrolling patients in IGNITE DMD for SGT-001;
delays in reaching agreement on acceptable terms with prospective contract research organizations, or CROs, and clinical trial sites;
delays in opening clinical trial sites or obtaining required IRB or independent ethics committee approval at each clinical trial site;
delays in recruiting suitable subjects to participate in our clinical trials, including because such trials may be placebo-controlled trials and
patients are not guaranteed to receive treatment with our product candidates;
failure by us, any CROs we engage or any other third parties to adhere to clinical trial requirements;
failure to perform in accordance with FDA good clinical practices, or GCPs, or applicable regulatory guidelines in the European Union and
other countries;
delays in the testing, validation, manufacturing and delivery of SGT-001 or our other product candidates to the clinical sites, including delays
by third parties with whom we have contracted to perform certain of those functions;
delays in subjects completing participation in a trial or returning for post-treatment follow-up;
clinical trial sites or subjects dropping out of a trial;
selection of clinical endpoints that require prolonged periods of clinical observation or analysis of the resulting data;
imposition of a clinical hold by regulatory authorities as a result of a serious adverse event or after an inspection of our clinical trial
operations, trial sites or manufacturing facilities;
occurrence of serious adverse events in trials of the same class of agents conducted by other sponsors; or
changes in regulatory requirements and guidance that require amending or submitting new clinical protocols.
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Additionally, if the results of any clinical trials are inconclusive or if there are safety concerns or serious adverse events associated with SGT-001 or
our other product candidates, we may:
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be delayed or fail in obtaining marketing approval for SGT-001 or our other product candidates;
obtain approval for indications or patient populations that are not as broad as we intended or desired;
obtain approval with labeling that includes significant use or distribution restrictions or safety warnings;
be subject to changes in the way our products, if approved, are administered;
be required to perform additional clinical trials to support approval or be subject to additional post-marketing testing requirements;
have regulatory authorities withdraw, or suspend, their approval of the product or impose restrictions on its distribution in the form of a
modified REMS;
be sued and held liable for harm caused to patients; or
experience damage to our reputation.
Our product development costs will increase if we experience delays in testing or marketing approvals. In addition, if we make manufacturing or
other changes to SGT-001 or our other product candidates, we may need to conduct additional studies to bridge our modified product candidates to earlier
versions. We do not know whether any of our preclinical studies or clinical trials will begin as planned, will need to be restructured or will be completed on
schedule, or at all. We may also determine to change the design or protocol of one or more of our clinical trials, which could result in delays. Significant
preclinical study or clinical trial delays also could shorten any periods during which we may have the exclusive right to commercialize our product
candidates or allow our competitors to bring products to market before we do and impair our ability to successfully commercialize our product candidates.
If our third-party clinical trial vendors fail to comply with strict regulations, the clinical trials for SGT-001 or our other product candidates may be
delayed or unsuccessful.
We do not have the personnel capacity to conduct or manage the clinical trials that will be necessary for the development of SGT-001 or our other
product candidates. For IGNITE DMD we are relying, and for any future clinical trials we expect we will rely, on third parties to assist us in managing,
monitoring and conducting our clinical trials. If these third parties fail to comply with applicable regulations or do not adequately fulfill their obligations
under the terms of our agreements with them, we may not be able to enter into alternative arrangements without undue delay or additional expenditures
and, therefore, the clinical trials for SGT-001 or our other product candidates may be delayed or unsuccessful.
Furthermore, the FDA can be expected to inspect some or all of the clinical sites participating in our clinical trials to determine if our clinical trials
are being conducted according to GCPs. If the FDA determines that these clinical sites are not in compliance with applicable regulations, we may be
required to delay, repeat or terminate the clinical trials.
We may find it difficult to enroll patients in our clinical trials, which could delay or prevent us from proceeding with clinical trials of SGT-001 or our
other product candidates.
Identifying and qualifying patients to participate in any clinical trials of SGT-001 and our other product candidates is critical to our success. The
timing of any clinical trials depends on our ability to recruit patients to participate as well as complete required follow-up periods. If patients are unwilling
or unable to participate in our gene therapy clinical trials, including because of negative publicity from adverse events related to our product candidates,
other approved gene therapies or the biotechnology or gene therapy fields, or due to competitive clinical trials for similar patient populations, clinical trials
in products employing our vector or our platform or for other reasons, the timeline for recruiting patients, conducting clinical trials and obtaining regulatory
approval of SGT-001 may be delayed. We may also experience delays if patients withdraw from the clinical trial or do not complete the required
monitoring period. These delays could result in increased costs, delays in advancing SGT-001 or our other product candidates, delays in testing the
effectiveness of SGT-001 and our other product candidates or termination of clinical trials altogether.
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We may not be able to identify, recruit and enroll a sufficient number of patients, or those with required or desired characteristics, to complete any
clinical trials in a timely manner. Patient enrollment and trial completion is affected by many factors, including:
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size of the patient population and the process for identifying subjects;
design of the trial protocol;
eligibility and exclusion criteria, including that some patients may have pre-existing antibodies to AAV vectors precluding them from being
able to receive AAV-mediated gene transfer;
restrictions on our ability to conduct clinical trials, including full and partial clinical holds on ongoing or planned clinical trials;
perceived risks and benefits of the product candidate under study;
perceived risks and benefits of gene therapy-based approaches to the treatment of diseases;
release or disclosure of data from our completed or ongoing clinical trials;
availability of competing therapies and clinical trials;
severity of the disease;
proximity and availability of clinical trial sites for prospective subjects;
ability to obtain and maintain subject consent;
risk that enrolled subjects will drop out before completion of the trial;
patient referral practices of physicians;
ability to monitor subjects adequately during and after treatment; and
in the case of pivotal trials, the risk that patients may opt not to enroll because they are not assured treatment with our product candidate.
In November 2019, the FDA placed our IGNITE DMD clinical trial of SGT-001 on clinical hold following our report of a serious adverse event in
the clinical trial. If we are unable to satisfy any requests of the FDA in a timely manner, or at all, or if the FDA does not lift the clinical hold in a timely
manner, or at all, we would be further delayed or prevented from enrolling patients in our IGNITE DMD clinical trial of SGT-001.
Our ability to successfully initiate, enroll and complete a clinical trial in any foreign country is subject to numerous risks unique to conducting
business in foreign countries, including:
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different standards for the conduct of clinical trials;
absence in some countries of established groups with sufficient regulatory expertise for review of gene therapy protocols;
difficulty in identifying and partnering with qualified local consultants, physicians and partners; and
the potential burden of complying with a variety of foreign laws, medical standards and regulatory requirements, including the regulation of
pharmaceutical and biotechnology research and products.
Even if we complete the necessary clinical trials, we cannot predict when, or if, we will obtain regulatory approval to commercialize SGT-001 or our
other product candidates and the approval may be for a more narrow indication than we seek.
We cannot commercialize SGT-001 or our other product candidates until the appropriate regulatory authorities have reviewed and approved the
product candidate. The process of obtaining regulatory approvals and the subsequent compliance with appropriate federal, state and local statutes and
regulations require the expenditure of substantial time and financial resources and we may not be able to obtain the required regulatory approvals. Even if
our product candidates meet their safety and efficacy endpoints in clinical trials, the regulatory authorities may not complete their review processes in a
timely manner, or we may not be able to obtain regulatory approval. Additional delays may result if an FDA advisory committee or other regulatory
authority recommends non-approval or restrictions on approval. In addition, we may experience delays or rejections based upon additional government
regulation from future legislation or administrative action or changes in regulatory authority policy during the period of product development, clinical trials
and the regulatory review process.
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Even if we receive regulatory approval, regulatory authorities may approve a product candidate for more limited indications than requested or they
may impose significant limitations in the form of narrow indications, warnings or a REMS. Regulatory authorities may require precautions or contra-
indications with respect to conditions of use or they may grant approval subject to the performance of costly post-marketing clinical trials. In addition,
regulatory authorities may not approve the labeling claims that are necessary or desirable for the successful commercialization of our product candidates.
Any of the foregoing scenarios could materially harm the commercial prospects for our product candidates.
Even if we obtain regulatory approval for a product candidate, our product candidates will remain subject to regulatory oversight.
Even if we obtain any regulatory approval for SGT-001 or our other product candidates, we will be subject to ongoing regulatory requirements for
manufacturing, labeling, packaging, storage, advertising, promotion, sampling, record-keeping and submission of safety and other post-market information.
Any regulatory approvals that we receive for our product candidates may also be subject to a REMS, limitations on the approved indicated uses for which
the product may be marketed or conditions of approval, or requirements for potentially costly post-marketing testing, including Phase IV clinical trials, and
surveillance to monitor the quality, safety and efficacy of the product. Advertising and promotional materials must comply with FDA rules and are subject
to FDA review, in addition to other potentially applicable federal and state laws.
If we fail to comply with applicable regulatory requirements following approval of SGT-001 or our other product candidates, a regulatory authority
may, among other things, suspend or withdraw regulatory approval, narrow the product label, restrict the marketing or manufacturing of the product,
suspend any ongoing clinical trials or seize or detain the product or otherwise require the withdrawal of the product from the market.
Even if we obtain and maintain approval for SGT-001 or our other product candidates from the FDA, we may never obtain approval for our product
candidates outside of the United States, which would limit our market opportunities and adversely affect our business.
Even if we receive FDA approval of SGT-001 or our other product candidates in the United States, approval of a product candidate in the United
States by the FDA does not ensure approval of such product candidate by regulatory authorities in other countries or jurisdictions, and approval by one
foreign regulatory authority does not ensure approval by regulatory authorities in other foreign countries or by the FDA. Future sales of our product
candidates outside of the United States will be subject to foreign regulatory requirements governing clinical trials, manufacturing and marketing approval.
Approval procedures vary among jurisdictions and can involve requirements and administrative review periods different from, and more onerous than,
those in the United States, including additional preclinical studies or clinical trials. In many countries outside the United States, a product candidate must
be approved for reimbursement before it can be approved for sale in that country. We intend to submit a marketing authorization application, or MAA, to
the EMA for approval of SGT-001 in the European Union, but obtaining such approval from the European Commission following the opinion of the EMA
is a lengthy and expensive process. Regulatory authorities in countries outside of the United States and the European Union also have requirements for
approval of product candidates with which we must comply prior to marketing in those countries. Obtaining foreign regulatory approvals and compliance
with foreign regulatory requirements could result in significant delays, difficulties and costs for us and could delay or prevent the introduction of SGT-001
or our other product candidates in certain countries.
Further, clinical trials conducted in one country may not be accepted by regulatory authorities in other countries. Also, regulatory approval for SGT-
001 or our other product candidates may be withdrawn. If we fail to comply with the regulatory requirements, our target market will be reduced, and our
ability to realize the full market potential of our product candidates will be harmed.
Regulatory requirements governing gene therapy products are periodically updated and may continue to change in the future.
The FDA has established the Office of Tissues and Advanced Therapies, or the OTAT, within the Center for Biologics Evaluation and Research, or
the CBER, to consolidate the review of gene therapy and related products, and has established the Cellular, Tissue and Gene Therapies Advisory
Committee to advise CBER in its review. Gene therapy clinical trials conducted at institutions that receive funding for recombinant DNA research from the
U.S. National Institutes of Health, or the NIH, also are potentially subject to review by the Office of Biotechnology Activities’ Recombinant DNA
Advisory Committee, or the RAC; however, the NIH announced that the RAC will soon only publicly review clinical trials if the trials cannot be evaluated
by standard oversight bodies and pose unusual risks. Although the FDA decides whether individual gene
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therapy protocols may proceed, the RAC public review process, if undertaken, can delay the initiation of a clinical trial, even if the FDA has reviewed the
trial design and details and approved its initiation. Conversely, the FDA can put an IND on a clinical hold even if the RAC has provided a favorable review
or an exemption from in-depth, public review. If we were to engage an NIH-funded institution to conduct a clinical trial, that institution’s institutional
biosafety committee, or IBC, as well as its IRB would need to review the proposed clinical trial to assess the safety of the trial. In addition, adverse
developments in clinical trials of gene therapy products conducted by others may cause the FDA or other oversight bodies to change the requirements for
approval of our product candidates.
The FDA has issued various guidance documents regarding gene therapies, including recent final guidance documents released in January 2020
relating to chemistry, manufacturing and controls information for gene therapy INDs, gene therapies for rare diseases and gene therapies for retinal
disorders. Although the FDA has indicated that these and other guidance documents it previously issued are not legally binding, we believe that our
compliance with them is likely necessary to gain approval for any gene therapy product candidate we may develop. The guidance documents provide
additional factors that the FDA will consider at each of the above stages of development and relate to, among other things, the proper preclinical
assessment of gene therapies; the chemistry, manufacturing, and control information that should be included in an IND application; the proper design of
tests to measure product potency in support of an IND or BLA application; and measures to observe delayed adverse effects in subjects who have been
exposed to investigational gene therapies when the risk of such effects is high. Further, the FDA usually recommends that sponsors observe subjects for
potential gene therapy-related delayed adverse events for a 15-year period, including a minimum of five years of annual examinations followed by 10 years
of annual queries, either in person or by questionnaire.
Further, for a gene therapy product, the FDA also will not approve the product if the manufacturer is not in compliance with good tissue practices,
or GTP. These standards are found in FDA regulations and guidances that govern the methods used in, and the facilities and controls used for, the
manufacture of human cells, tissues, and cellular and tissue based products, or HCT/Ps, which are human cells or tissue intended for implantation,
transplant, infusion, or transfer into a human recipient. The primary intent of the GTP requirements is to ensure that cell and tissue based products are
manufactured in a manner designed to prevent the introduction, transmission, and spread of communicable disease. FDA regulations also require tissue
establishments to register and list their HCT/Ps with the FDA and, when applicable, to evaluate donors through screening and testing.
Similarly, the EMA may issue new guidelines concerning the development and marketing authorization for gene therapy products and require that
we comply with these new guidelines. The grant of marketing authorization in the European Union for gene therapy products is governed by
Regulation 1394/2007/EC on advanced therapy medicinal products, read in combination with Directive 2001/83/EC of the European Parliament and of the
Council, commonly known as the Community code on medicinal products. Regulation 1394/2007/EC includes specific rules concerning the authorization,
supervision, and pharmacovigilance of gene therapy medicinal products. Manufacturers of advanced therapy medicinal products must demonstrate the
quality, safety, and efficacy of their products to the EMA, which provides an opinion regarding the MAA. The European Commission grants or refuses
marketing authorization in light of the opinion delivered by the EMA.
Finally, ethical, social and legal concerns about gene therapy, genetic testing and genetic research could result in additional regulations or
prohibiting the processes we may use. Federal and state agencies, congressional committees and foreign governments have expressed their intentions to
further regulate biotechnology. More restrictive regulations or claims that our product candidates are unsafe or pose a hazard could prevent us from
commercializing any products. New government requirements may be established that could delay or prevent regulatory approval of our product candidates
under development. It is impossible to predict whether legislative changes will be enacted, regulations, policies or guidance changed, or interpretations by
agencies or courts changed, or what the impact of such changes, if any, may be.
As we advance our product candidates through clinical development, we will be required to consult with these regulatory and advisory groups, and
comply with applicable guidelines. These regulatory review committees and advisory groups and any new guidelines they promulgate may lengthen the
regulatory review process, require us to perform additional studies, increase our development costs, lead to changes in regulatory positions and
interpretations, delay or prevent approval and commercialization of SGT-001 or our other product candidates or lead to significant post-approval limitations
or restrictions. Delay or failure to obtain, or unexpected costs in obtaining, the regulatory approval necessary to bring a potential product to market could
decrease our ability to generate sufficient product revenue.
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We may not be able to benefit from orphan drug designation for SGT-001 or any of our product candidates.
The FDA and EMA granted SGT-001 orphan drug designation for the treatment of DMD in August 2016 and September 2016, respectively. The
designation of SGT-001 as an orphan drug does not guarantee that any regulatory agency will accelerate regulatory review of, or ultimately approve, that
product candidate, nor does it limit the ability of any regulatory agency to grant orphan drug designation to product candidates of other companies that treat
the same indications as our product candidate prior to our product candidate receiving exclusive marketing approval.
We may lose orphan drug exclusivity if the FDA or EMA determines that the request for designation was materially defective or if we cannot assure
sufficient quantity of the applicable drug to meet the needs of patients with DMD.
Even if we maintain orphan drug exclusivity for SGT-001 or obtain orphan drug exclusivity for any other product candidate, the exclusivity may not
effectively protect the product candidate from competition because regulatory authorities still may authorize different drugs for the same condition or the
same drug for the same condition if it is determined by the FDA to be clinically superior to the product with orphan drug exclusivity. Moreover, the concept
of what constitutes the “same drug” for purposes of orphan drug exclusivity remains in flux in the context of gene therapies, and the FDA issued draft
guidance in January 2020 suggesting that it would not consider two gene therapy products to be different drugs solely based on minor differences in the
transgenes or vectors.
We may seek a breakthrough therapy designation for SGT-001 or our other product candidates, but we might not receive such designation, and even if
we do, such designation may not lead to a faster development or regulatory review or approval process.
We may seek a breakthrough therapy designation for SGT-001 or our other product candidates; however, we cannot assure our stockholders that
SGT-001 or our other product candidates will meet the criteria for that designation. A breakthrough therapy is defined as a therapy that is intended, alone or
in combination with one or more other therapies, to treat a serious condition, and preliminary clinical evidence indicates that the therapy may demonstrate
substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in
clinical development. For therapies and biologics that have been designated as breakthrough therapies, interaction and communication between the FDA
and the sponsor of the trial can help to identify the most efficient path for clinical development while minimizing the number of patients placed in
ineffective control regimens. Therapies designated as breakthrough therapies by the FDA may also be eligible for priority review if supported by clinical
data at the time the new drug application is submitted to the FDA.
Designation as a breakthrough therapy is within the discretion of the FDA. Accordingly, even if we believe that one of our product candidates meets
the criteria for designation as a breakthrough therapy, the FDA may disagree and instead determine not to make such designation. Even if we receive
breakthrough therapy designation, the receipt of such designation for a product candidate may not result in a faster development or regulatory review or
approval process compared to drugs considered for approval under conventional FDA procedures and does not assure ultimate approval by the FDA. In
addition, even if one or more of our product candidates qualifies as a breakthrough therapy, the FDA may later decide that the product candidate no longer
meets the conditions for qualification or decide that the time period for FDA review or approval will not be shortened.
Accelerated approval by the FDA, even if granted for SGT-001 or our other product candidates, may not lead to a faster development or regulatory
review or approval process and it does not increase the likelihood that our product candidates will receive marketing approval.
We may seek approval of SGT-001 or our other product candidates using the FDA’s accelerated approval pathway. A product may be eligible for
accelerated approval if it treats a serious or life-threatening condition and generally provides a meaningful advantage over available therapies. In addition,
it must demonstrate an effect on a surrogate endpoint that is reasonably likely to predict clinical benefit or on a clinical endpoint that can be measured
earlier than irreversible morbidity or mortality, or IMM, that is reasonably likely to predict an effect on IMM or other clinical benefit. The FDA or other
applicable regulatory agency makes the determination regarding whether a surrogate endpoint is reasonably likely to predict long-term clinical benefit.
Given that expression of microdystrophin has not yet been established to predict long-term clinical benefit, it is not currently accepted, and it is possible the
FDA and/or other applicable regulatory agencies could decide never to accept it, as a surrogate endpoint for the accelerated approval pathway.
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As a condition of approval, the FDA may require that a sponsor of a drug or biologic product candidate receiving accelerated approval perform
adequate and well-controlled post-marketing clinical trials. These confirmatory trials must be completed with due diligence. In addition, the FDA currently
requires as a condition for accelerated approval pre-approval of promotional materials, which could adversely impact the timing of the commercial launch
of the product.
Even if we do receive accelerated approval, we may not experience a faster development or regulatory review or approval process and receiving
accelerated approval does not provide assurance of ultimate FDA approval.
A potential regenerative medicine advanced therapy designation by the FDA for our product candidates may not lead to a faster development or
regulatory review or approval process, and it does not increase the likelihood that our product candidates will receive marketing approval.
We may seek a regenerative medicine advanced therapy designation, or RMAT, for some of our product candidates. A regenerative medicine
advanced therapy is defined as cell therapies, therapeutic tissue engineering products, human cell and tissue products, and combination products using any
such therapies or products. Gene therapies, including genetically modified cells, that lead to a durable modification of cells or tissues may meet the
definition of a regenerative medicine therapy. The regenerative medicine advanced therapy program is intended to facilitate efficient development and
expedite review of regenerative medicine advanced therapies, which are intended to treat, modify, reverse, or cure a serious or life-threatening disease or
condition. A new drug application or a BLA for a regenerative medicine advanced therapy may be eligible for priority review or accelerated approval
through (1) surrogate or intermediate endpoints reasonably likely to predict long-term clinical benefit or (2) reliance upon data obtained from a meaningful
number of sites. Benefits of such designation also include early interactions with the FDA to discuss any potential surrogate or intermediate endpoint to be
used to support accelerated approval. A regenerative medicine therapy that is granted accelerated approval and is subject to post-approval requirements
may fulfill such requirements through the submission of clinical evidence, clinical studies, patient registries, or other sources of real world evidence, such
as electronic health records; the collection of larger confirmatory data sets; or post-approval monitoring of all patients treated with such therapy prior to its
approval.
Designation as a regenerative medicine advanced therapy is within the discretion of the FDA. Accordingly, even if we believe one of our product
candidates meets the criteria for designation as a regenerative medicine advanced therapy, the FDA may disagree and instead determine not to make such
designation. In any event, the receipt of a regenerative medicine advanced therapy designation for a product candidate may not result in a faster
development process, review or approval compared to drugs considered for approval under conventional FDA procedures and does not assure ultimate
approval by the FDA. In addition, even if one or more of our product candidates qualify as regenerative medicine advanced therapies, the FDA may later
decide that the biological products no longer meet the conditions for qualification.
The FDA has granted Rare Pediatric Disease Designation, or RPDD, to SGT-001; however, a BLA for SGT-001 may not meet the eligibility criteria for
a priority review voucher upon approval.
The FDA has granted RPDD to SGT-001. RPDD does not guarantee that a BLA for such drug will meet the eligibility criteria for a rare pediatric
disease priority review voucher at the time the application is approved. We will need to request a rare pediatric disease priority review voucher in our BLA
for SGT-001. The use of a priority review voucher allows for a drug or biologic to be reviewed by the FDA within six months. However, the FDA may
determine that a BLA for SGT-001 does not meet the eligibility criteria for a priority review voucher upon approval. Moreover, even if SGT-001 does
satisfy those criteria, the product will need to be licensed before September 30, 2022 in order to be granted a rare disease priority review voucher.
The FDA has granted fast track designation for SGT-001. However, such designation may not actually lead to a faster development or regulatory
review or approval process. We might not receive such designation for our other product candidates.
If a therapy is intended for the treatment of a serious condition and nonclinical or clinical data demonstrate the potential to address unmet medical
need for this condition, a drug sponsor may apply for FDA fast track designation. The FDA has granted fast track designation to SGT-001; however, fast
track designation does not ensure that we will receive marketing approval or that approval will be granted within any particular timeframe. We may not
experience a faster development or regulatory review or approval process with fast track designation compared to conventional FDA procedures. In
addition, the FDA may withdraw fast track designation if it believes that the designation is no longer supported by data from our clinical development
program. Fast track designation alone does not guarantee qualification for the FDA’s priority review procedures.
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We may seek priority review designation for SGT-001 or our other product candidates, but we might not receive such designation, and even if we do,
such designation may not lead to a faster development or regulatory review or approval process.
If the FDA determines that a product candidate offers a treatment for a serious condition and, if approved, the product would provide a significant
improvement in safety or effectiveness, the FDA may designate the product candidate for priority review. A priority review designation means that the goal
for the FDA to review an application is six months, rather than the standard review period of ten months. We may request priority review for our product
candidates, however, we cannot assume that SGT-001 or our other product candidates will meet the criteria for that designation. The FDA has broad
discretion with respect to whether or not to grant priority review status to a product candidate, so even if we believe a particular product candidate is
eligible for such designation or status, the FDA may decide not to grant it. Moreover, a priority review designation does not necessarily mean a faster
development or regulatory review or approval process or necessarily confer any advantage with respect to approval compared to conventional FDA
procedures. Receiving priority review from the FDA does not guarantee approval within the six-month review cycle or at all.
We face significant competition and our competitors may achieve regulatory approval before us or develop therapies that are more advanced or
effective than ours, which may adversely affect our ability to successfully market or commercialize SGT-001 or our other product candidates.
We operate in a highly competitive segment of the biopharmaceutical market. We face competition from many different sources, including larger
and better-funded pharmaceutical, specialty pharmaceutical and biotechnology companies, as well as from academic institutions, government agencies and
private and public research institutions. Our product candidates, if successfully developed and approved, will compete with established therapies as well as
with new treatments that may be introduced by our competitors. There are a variety of product candidates, including gene therapies, in development for
DMD. Many of our competitors have significantly greater financial, product candidate development, manufacturing and marketing resources than we do.
Large pharmaceutical and biotechnology companies have extensive experience in clinical testing and obtaining regulatory approval for their products, and
mergers and acquisitions within these industries may result in even more resources being concentrated among a smaller number of larger competitors.
Smaller and other early-stage companies may also prove to be significant competitors, particularly through collaborative arrangements with large and
established companies. These third parties compete with us in recruiting and retaining qualified scientific and management personnel, establishing clinical
trial sites and patient registration for clinical trials, as well as in acquiring technologies complementary to, or necessary for, our programs.
For example, we are aware of several companies and research institutions focused on developing systemic gene transfers for DMD, including Pfizer
Inc., with a product candidate currently in Phase 1 clinical development, and Sarepta Therapeutics, Inc., with a product candidate currently in Phase I/II
clinical trial development.
Our commercial opportunity could be reduced or eliminated if competitors develop and commercialize products that are safer, more effective, have
fewer or less severe side effects, have broader market acceptance, are more convenient or are less expensive than any product candidate that we may
develop.
We are aware of several companies focused on developing gene therapies in various indications, as well as several companies addressing other
methods for modifying genes and regulating gene expression. Any advances in gene therapy technology made by a competitor may be used to develop
therapies that could compete against SGT-001 or any future gene therapy product candidates we develop.
We may fail to capitalize on other potential product candidates that may represent a greater commercial opportunity or for which there is a greater
likelihood of success.
The success of our business depends upon our ability to develop and commercialize SGT-001 and our other product candidates. Because we have
limited resources, we may forego or delay pursuit of opportunities with certain programs or product candidates or for indications that later prove to have
greater commercial potential than SGT-001 or our other product candidates. For example, in January 2020, in connection with implementing our strategic
plan to create a leaner company focused on advancing SGT-001, we curtailed certain activities supporting our other research and development programs.
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Our spending on current and future research and development programs may not yield any commercially viable product candidates. If we do not
accurately evaluate the commercial potential for a particular product candidate, we may relinquish valuable rights to that product candidate through
strategic collaborations, licensing or other arrangements in cases in which it would have been more advantageous for us to retain sole development and
commercialization rights to such product candidate. Alternatively, we may allocate internal resources to a product candidate in a therapeutic area in which
it would have been more advantageous to enter into a partnering arrangement. If any of these events occur, we may be forced to abandon our development
efforts with respect to a particular product candidate or fail to develop a potentially successful product candidate.
Risks related to the manufacturing and commercialization of SGT-001 and our other product candidates
We may not be successful in finding strategic collaborators for continuing development of SGT-001 or our other product candidates or successfully
commercializing or competing in the market for certain indications.
We may seek to establish strategic partnerships for developing SGT-001 or our other product candidates due to capital costs required to develop,
manufacture and commercialize our product candidates. We may not be successful in our efforts to establish such strategic partnerships or other alternative
arrangements because our research and development pipeline may be insufficient, SGT-001 may be deemed to be at too early of a stage of development for
collaborative effort or third parties may not view SGT-001 as having the requisite potential to demonstrate safety and efficacy. We cannot be certain that,
following a strategic transaction, we will achieve an economic or business benefit that justifies such transaction.If we seek to but are unable to reach
agreements with suitable collaborators on a timely basis, on acceptable terms or at all, we may have to curtail, reduce or delay the development of a product
candidate, delay its potential commercialization, reduce the scope of any sales or marketing activities or increase our expenditures and undertake
development, manufacturing or commercialization activities independently. If we elect to fund our own independent development or commercialization
activities, we will need to obtain additional expertise and additional capital, which may not be available to us on acceptable terms or at all. If we fail to
enter into collaborations and do not have sufficient funds or expertise to undertake the necessary development, manufacturing and commercialization
activities, we may not be able to further develop SGT-001 or our other product candidates.
We have limited gene transfer manufacturing experience and could experience production problems and delays in obtaining regulatory approval of our
manufacturing processes, which could result in delays in the development or commercialization of SGT-001 or our other product candidates.
The manufacturing process we use to produce SGT-001 is complex and has not been validated for commercial use. We have no experience
manufacturing SGT-001 and our other product candidates. Building our own manufacturing facility, if we decide to do so in the future, would require
substantial additional investment, would be time-consuming and may be subject to delays, including those resulting from compliance with regulatory
requirements. In addition, building a manufacturing facility may cost more than we currently anticipate. Although we may establish our own manufacturing
facility to support a commercial launch, if we are unable to do so or otherwise decide not to do so, we may be unable to produce commercial materials or
meet demand, if any should develop, for SGT-001 and our other product candidates. Any such failure could delay or prevent our commercialization of
SGT-001 or our other product candidates. The production of SGT-001 requires processing steps that are more complex than those required for most
chemical pharmaceuticals. Moreover, unlike chemical pharmaceuticals, the physical and chemical properties of a gene transfer product candidate such as
ours generally cannot be fully characterized. As a result, assays of the finished product may not be sufficient to ensure that the product will perform in the
intended manner. Accordingly, we employ multiple steps to control our manufacturing process to assure that the process works and that SGT-001 is made
strictly and consistently in compliance with the process. As a result of the limited number of FDA approvals for gene transfer products to date, the
timeframe required for us to obtain approval for a cGMP gene therapy manufacturing facility in the United States is uncertain. We must supply all
necessary documentation in support of a BLA or MAA on a timely basis and must adhere to the FDA’s and the European Union’s cGMP requirements
before we can obtain marketing approval for SGT-001 and our other product candidates. In order to obtain approval, we will need to ensure that all of our
processes, methods and equipment are compliant with cGMP requirements, and perform extensive audits of contract laboratories, manufacturers and
suppliers.
We currently rely on third-party manufacturers for our SGT-001 supply. In order to produce sufficient quantities of SGT-001 for clinical trials and
initial U.S. commercial demand, we will need to increase the scale of our manufacturing process at our third-party manufacturers, and potentially through
our own commercial-scale manufacturing facility. We may need to change our current manufacturing process. We may not be able to produce sufficient
quantities of SGT-001 due to several factors, including equipment malfunctions, facility contamination, material shortages or contamination, natural
disasters, a public health issue (for example, an outbreak of a contagious disease such as the novel coronavirus), disruption in utility services, human error
or disruptions in the operations of our suppliers. For example, through our contract manufacturer
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we have performed and released within specifications manufacturing runs of SGT-001 for clinical supply and have experienced variability with respect to
the success and yield of these runs. We continue to engage in process development activities to improve the reproducibility, reliability and consistency of
yields of our manufacturing process. While we are able to produce for more than one patient from a single batch, additional manufacturing runs will be
required to produce necessary or adequate supply for IGNITE DMD and there is no guarantee that all of those runs will be within specifications or produce
adequate supply. If we are not able to produce sufficient supply on the timeline expected, our overall development schedule for SGT-001 could be delayed,
and we could incur additional expense.
If supply from a manufacturing facility is interrupted, including as a result of equipment malfunctions, facility contamination, material shortages or
contamination, natural disasters, a public health issue, disruption in utility services or human error, there could be a significant disruption in supply of SGT-
001 or our other product candidates. In such instance, we may need to locate appropriate replacement third-party manufacturers, and we may not be able to
enter into arrangements with such additional third-party manufacturers on favorable terms or at all. Use of new third-party manufacturers could increase the
risk of delays in production or insufficient supplies of our product candidates as we transfer our manufacturing technology to these manufacturers and as
they gain experience manufacturing our product candidates.
In addition, product manufacturers and their facilities are subject to payment of user fees and continual review and periodic inspections by the FDA
and other regulatory authorities for compliance with cGMP requirements and adherence to commitments made in the BLA or foreign marketing
application. If we, or a regulatory authority, discover previously unknown problems with a product, such as adverse events of unanticipated severity or
frequency, or problems with the facility where the product is manufactured, a regulatory authority may impose restrictions relative to that product, the
manufacturing facility or us, including requiring recall or withdrawal of the product from the market or suspension of manufacturing.
In addition, the FDA, the EMA and other foreign regulatory authorities may require us to submit samples of any lot of any approved product
together with the protocols showing the results of applicable tests at any time. Under some circumstances, the FDA, the EMA or other foreign regulatory
authorities may require that we not distribute a lot until the agency authorizes its release. Lot failures or product recalls could cause us to delay or abandon
clinical trials or product launches.
We also may encounter problems hiring and retaining the experienced specialist scientific, quality control and manufacturing personnel needed to
operate our manufacturing process, which could result in delays in our production or difficulties in maintaining compliance with applicable regulatory
requirements.
Any problems in our manufacturing process or facilities could make us a less attractive collaborator for potential partners, including larger
pharmaceutical companies and academic research institutions, which could limit our access to additional attractive development programs. Problems in our
manufacturing process or facilities also could restrict our ability to meet market demand for SGT-001, our other product candidates or future product
candidates.
Although we may establish our own SGT-001 manufacturing facility, we expect to utilize third parties to conduct our product manufacturing for the
foreseeable future. Therefore, we are subject to the risk that these third parties may not perform satisfactorily or meet regulatory requirements.
Until such time, if ever, as we establish a manufacturing facility that has been properly validated to comply with FDA cGMP requirements, we will
not be able to independently manufacture material for our current and future clinical programs. For clinical trials of SGT-001, we intend to utilize materials
manufactured by cGMP-compliant third-party suppliers. Even following our potential establishment of a validated cGMP manufacturing facility, we intend
to utilize third-party manufacturing capabilities in order to provide multiple sources of supply. In the event that the establishment of our own manufacturing
facility is delayed or not otherwise pursued and if these third-party manufacturers do not successfully carry out their contractual duties, meet expected
deadlines or manufacture SGT-001 in accordance with regulatory requirements or if there are disagreements between us and these third-party
manufacturers, we may not be able to complete, or may be delayed in completing, the clinical trials required for approval of SGT-001. In such instances,
we may need to locate an appropriate replacement third-party relationship, which may not be readily available or on acceptable terms, which would cause
additional delay or increased expense prior to the approval of our product candidates.
Additionally, we rely on our third-party manufacturers for their compliance with the cGMP and their maintenance of adequate quality control,
quality assurance and qualified personnel. Furthermore, all of our third-party suppliers and manufacturers are engaged with other companies to supply
and/or manufacture materials or products for such companies, which exposes them to regulatory risks for the production of such materials and products.
FDA inspections may identify compliance issues at third-party manufacturer facilities or at the facilities of third-party suppliers that may disrupt production
or distribution, or require substantial resources to correct and prevent recurrence of any deficiencies, and could result in fines or penalties by regulatory
authorities. In addition, discovery of problems with a product or the failure to comply with applicable requirements may result in restrictions on a product,
manufacturer or holder of an approved BLA, including withdrawal or recall of the product from the market or other voluntary, FDA-initiated or judicial
action, including fines,
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injunctions, civil penalties, license revocations, seizure, total or partial suspension of production or criminal penalties, any of which could significantly and
adversely affect supplies of our product candidates.
In addition, we do not currently have long-term supply or manufacturing arrangements in place for the production of SGT-001 at commercial scale.
Although we intend to establish additional sources for long-term supply, potentially including our own commercial-scale cGMP-compliant manufacturing
facility and one or more third-party manufacturers, if the gene therapy industry were to grow, we may encounter increasing competition for the materials
necessary for the production of SGT-001. We may experience difficulties in scaling up production beyond clinical batches. Furthermore, demand for third-
party cGMP manufacturing facilities may grow at a faster rate than existing manufacturing capacity, which could disrupt our ability to find and retain third-
party manufacturers capable of producing sufficient quantities of SGT-001 for future clinical trials or to meet initial commercial demand in the United
States. We currently rely, and expect to continue to rely, on additional third parties to manufacture materials for our product candidates and to perform
quality testing. Even following the potential establishment of our own cGMP-compliant manufacturing capabilities, we intend to maintain third-party
manufacturers for these materials, as well as to serve as additional sources of SGT-001, which will expose us to risks including:
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reduced control of manufacturing activities;
the inability of certain contract manufacturing organizations, or CMOs, to produce our product candidates in the necessary quantities, or in
compliance with current cGMP or in compliance with pertinent regulatory requirements and within our planned time frame and cost
parameters;
termination or nonrenewal of manufacturing and service agreements with third parties in a manner or at a time that is costly or damaging to
us; and
disruptions to the operations of our third-party manufacturers and our and their suppliers caused by conditions unrelated to our business or
operations, including the bankruptcy of the manufacturer or supplier, natural disasters or public health issues.
Any of these events could lead to clinical trial delays or failure to obtain regulatory approval or impact our ability to successfully commercialize
SGT-001 or our other product candidates. Some of these events could be the basis for FDA action, including injunction, recall, seizure or total or partial
suspension of product manufacture.
If we are unable to establish sales, distribution and marketing capabilities or enter into agreements with third parties to market and sell SGT-001 and
our other product candidates, we will be unable to generate any product revenue.
We currently have no sales, distribution or marketing organization. To successfully commercialize any product candidate that may result from our
development programs, we will need to develop these capabilities, either on our own or with others. The establishment and development of our own
commercial team or the establishment of a contract sales force to market any product candidate we may develop will be expensive and time-consuming and
could delay any product launch. Moreover, we cannot be certain that we will be able to successfully develop this capability. We may enter into
collaborations regarding SGT-001 and our other product candidates with other entities to utilize their established marketing and distribution capabilities,
but we may be unable to enter into such agreements on favorable terms, if at all. If any future collaborators do not commit sufficient resources to
commercialize our product candidates, or we are unable to develop the necessary capabilities on our own, we will be unable to generate sufficient product
revenue to sustain our business. We compete with many companies that currently have extensive, experienced and well-funded sales, distribution and
marketing operations to recruit, hire, train and retain marketing and sales personnel. We also face competition in our search for third parties to assist us with
the sales and marketing efforts of SGT-001 and our other product candidates. Without an internal team or the support of a third party to perform marketing
and sales functions, we may be unable to compete successfully against these more established companies.
If we are unable to establish medical affairs capabilities, we will be unable to establish an educated market of physicians to administer SGT-001 or our
other product candidates.
We currently have no medical affairs team. If we are unable to successfully build a medical affairs team to address scientific and medical questions
and provide expert guidance and education in the application, administration and utilization of SGT-001 and our other product candidates to physicians, we
may not be able to establish an educated market for our products. The establishment and development of our own medical affairs team will be expensive
and time-consuming and could delay any product launch. Moreover, we cannot be certain that we will be able to successfully develop this capability.
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If the market opportunities for SGT-001 are smaller than we believe they are, our revenue prospects may be adversely affected and our business may
suffer.
We currently focus our research and product development on treatments for DMD. Our understanding of the patient population with this disease is
based on estimates in published literature and by DMD foundations. These estimates may prove to be incorrect and new studies may reduce the estimated
incidence or prevalence of this disease. The number of patients in the United States, the European Union and elsewhere may turn out to be lower than
expected, patients may not be otherwise amenable to treatment with our product candidate or patients may become increasingly difficult to identify and
access.
Further, there are several factors that could contribute to making the actual number of patients who receive SGT-001 less than the potentially
addressable market. These include the lack of widespread availability of, and limited reimbursement for, new therapies in many underdeveloped markets.
Further, the severity of the progression of a degenerative disease such as DMD up to the time of treatment will likely diminish the therapeutic benefit
conferred by a gene therapy due to irreversible cell damage.
Certain patients’ immune systems might prohibit the successful delivery of certain gene therapy products, thereby potentially limiting the population of
patients amenable to gene transfer.
As with many AAV-mediated gene therapy approaches, certain patients’ immune systems might prohibit the successful delivery of certain gene
therapy products, thereby potentially limiting the population of patients amenable to gene transfer. While we are working to better understand the
prevalence of antibodies to AAV, or seroprevalence, as it relates to gene therapies for DMD, the exact DMD-wide seroprevalence is currently unknown and
it varies by AAV serotype and age. We may not be able to address this potentially limiting factor for gene therapy as a treatment for certain patients.
The commercial success of any of our product candidates, including SGT-001, if approved, will depend upon market acceptance by physicians, patients,
third-party payors and others in the medical community.
Even with the requisite approvals from the FDA in the United States, the European Commission in the European Union and other regulatory
authorities internationally, the commercial success of SGT-001 will depend, in part, on the acceptance of physicians, patients and health care payors of gene
therapy products in general, and SGT-001 in particular, as medically necessary, cost-effective and safe. Any product that we commercialize may not gain
acceptance by physicians, patients, health care payors and others in the medical community due to ethical, social, medical and legal concerns. If these
products do not achieve an adequate level of acceptance, we may not generate significant product revenue and may not become profitable. The degree of
market acceptance of gene therapy products and, in particular, SGT-001, if approved for commercial sale, will depend on multiple factors, including:
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the efficacy and safety of SGT-001 as demonstrated in clinical trials;
the efficacy and potential and perceived advantages of SGT-001 over alternative treatments;
the cost of treatment relative to alternative treatments;
the clinical indications for which SGT-001 is approved by the FDA or the European Commission;
the willingness of physicians to prescribe new therapies;
the willingness of the target patient population to try new therapies;
the prevalence and severity of any side effects;
product labeling or product insert requirements of the FDA, the EMA or other regulatory authorities, including any limitations or warnings
contained in a product’s approved labeling;
relative convenience and ease of administration;
the strength of marketing and distribution support;
the timing of market introduction of competitive products;
the availability of products to meet market demand;
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publicity concerning our product candidates or competing products and treatments;
any restrictions on the use of our products together with other medications; and
favorable third-party payor coverage and adequate reimbursement.
Even if a potential product candidate displays a favorable efficacy and safety profile in preclinical studies and clinical trials, market acceptance of
the product will not be fully known until after it is launched.
Our efforts to educate the medical community and third-party payors on the benefits of SGT-001 and our other product candidates may require
significant resources and may never be successful. Such efforts may require more resources than are typically required due to the complexity and
uniqueness of our potential product candidates. If SGT-001 or our other product candidates are approved but fail to achieve market acceptance among
physicians, patients or third-party payors, we will not be able to generate significant revenue from any such product.
Our gene transfer approach utilizes a vector derived from a virus, which may be perceived as unsafe or may result in unforeseen adverse events.
Negative public opinion and increased regulatory scrutiny of gene therapy may damage public perception of the safety of our SGT-001 gene transfer
product candidate and adversely affect our ability to conduct our business or obtain regulatory approvals for SGT-001.
Gene transfer remains a novel technology and public perception may be influenced by claims that gene transfer is unsafe, and gene transfer may not
gain the acceptance of the public or the medical community. In particular, our success will depend upon physicians who specialize in the treatment of DMD
prescribing treatments that involve the use of SGT-001 in lieu of, or in addition to, other treatments with which they are more familiar and for which greater
clinical data may be available. More restrictive government regulations or negative public opinion may delay or impair the development and
commercialization of SGT-001 or demand for any product candidate we may develop. A public backlash developed against gene therapy following the
death of a patient in 1999 during a gene therapy clinical trial of research subjects with ornithine transcarbamylase, or OTC, deficiency, a rare disorder in
which the liver lacks a functional copy of the OTC gene. The death of the clinical trial subject was due to complications of adenovirus vector
administration. Dr. James M. Wilson, former chair of our Scientific Advisory Board, was a co-investigator of the 1999 trial while he was Director of the
Institute for Human Gene Therapy of the University of Pennsylvania. Serious adverse events in our clinical trials, including the events that led to the
current clinical hold on IGNITE DMD and the previously-lifted clinical hold on IGNITE DMD or other clinical trials involving gene transfer products or
our competitors’ products, even if not ultimately attributable to the relevant product candidates, and the resulting publicity, could result in increased
government regulation, unfavorable public perception, potential regulatory delays in the testing or approval of SGT-001, stricter labeling requirements for
SGT-001 if approved and a decrease in demand for SGT-001.
Failure to comply with ongoing regulatory requirements could cause us to suspend production or put in place costly or time-consuming remedial
measures.
The regulatory authorities may, at any time following approval of a product for sale, audit the manufacturing facilities for such product. If any such
inspection or audit identifies a failure to comply with applicable regulations, or if a violation of product specifications or applicable regulations occurs
independent of such an inspection or audit, the relevant regulatory authority may require remedial measures that may be costly or time-consuming to
implement and that may include the temporary or permanent suspension of a clinical trial or commercial sales or the temporary or permanent closure of a
manufacturing facility.
Any contamination in our manufacturing process, shortages of materials or failure of any of our key suppliers to deliver necessary components could
result in interruption in the supply of our product candidates and delays in our clinical development or commercialization schedules.
Given the nature of biologics manufacturing, there is a risk of contamination in our manufacturing processes. Any contamination could materially
adversely affect our ability to produce SGT-001 on schedule and could cause reputational damage.
Some of the materials required in our manufacturing process are derived from biologic sources. Such materials are difficult to procure and may be
subject to contamination or recall. A material shortage, contamination, recall or restriction on the use of biologically derived substances in the manufacture
of SGT-001 could adversely impact or disrupt the commercial manufacturing or the production of clinical material, which could materially and adversely
affect our development timelines.
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The insurance coverage and reimbursement status of newly approved products is uncertain. Failure to obtain or maintain adequate coverage and
reimbursement for our product candidates, if approved, could limit our ability to market those products and decrease our ability to generate product
revenue.
There is significant uncertainty related to third-party coverage and reimbursement of newly approved products. We expect the cost of a single
administration of gene transfer products, such as those we are developing, to be substantial, when and if they achieve regulatory approval. We expect that
coverage and reimbursement by government and private payors will be essential for most patients to be able to afford these treatments. Accordingly, sales
of SGT-001, if approved, will depend substantially, both domestically and abroad, on the extent to which the costs of SGT-001 will be paid by health
maintenance, managed care, pharmacy benefit and similar health care management organizations, or will be reimbursed by government authorities, private
health coverage insurers and other third-party payors. Coverage and reimbursement by a third-party payor may depend upon several factors, including the
third-party payor’s determination that use of a product is:
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a covered benefit under its health plan;
safe, effective and medically necessary;
appropriate for the specific patient;
cost-effective;
durable and a one-time treatment; and
neither experimental nor investigational.
Obtaining coverage and reimbursement for a product from third-party payors is a time-consuming and costly process that could require us to
provide to the payor supporting scientific, clinical and cost-effectiveness data. If coverage and reimbursement are not available, or are available only at
limited levels, we may not be able to successfully commercialize SGT-001 and our other product candidates. Even if coverage is provided, the approved
reimbursement amount may not be adequate to realize a sufficient return on our investment.
To our knowledge, a limited number of gene transfer products have been approved for coverage and reimbursement by the Centers for Medicare &
Medicaid Services, or the CMS, the agency responsible for administering the Medicaid program. It is difficult to predict what the CMS will decide with
respect to coverage and reimbursement for fundamentally novel products such as ours, as there is no body of established practices and precedents for these
types of products either in the United States or the European Union. For example, several cancer drugs have been approved for reimbursement in the
United States and have not been approved for reimbursement in certain European Union member states and vice versa. It is difficult to predict what third-
party payors will decide with respect to the coverage and reimbursement for SGT-001 and our other product candidates.
Governments outside the United States tend to impose strict price controls, which may adversely affect our revenue, if any.
Outside the United States, international operations generally are subject to extensive government price controls and other market regulations, and
increasing emphasis on cost-containment initiatives in the European Union, Canada and other countries may put pricing pressure on us. In general, the
prices of therapeutics outside the United States are substantially lower than in the United States. Other countries may allow companies to fix their own
prices for therapeutics, but monitor and control company profits. Additional foreign price controls or other changes in pricing regulations could restrict the
amount that we are able to charge for our product candidates. Accordingly, in markets outside the United States, the reimbursement for our product
candidates may be reduced compared with the United States and may be insufficient to generate commercially reasonable product revenue.
Additionally, in countries where the pricing of gene therapy products is subject to governmental control, pricing negotiations with governmental
authorities can take considerable time after the receipt of marketing approval for a product. Political, economic and regulatory developments may further
complicate pricing negotiations, and pricing negotiations may continue after reimbursement has been obtained. Reference pricing used by various European
Union member states and parallel distribution, or arbitrage between low-priced and high-priced member states, can further reduce prices. To obtain
reimbursement or pricing approval in some countries, we may be required to conduct a clinical trial that compares the cost-effectiveness of our product
candidate to other available therapies. Reimbursement of our products may be unavailable or limited in scope or amount, which would adversely affect our
revenue, if any.
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If we obtain approval to commercialize SGT-001 and our other product candidates outside of the United States, in particular in the European Union, a
variety of risks associated with international operations could materially adversely affect our business.
We expect that we will be subject to additional risks in commercializing SGT-001 and our other product candidates outside the United States,
including:
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different regulatory requirements for approval of therapeutics in foreign countries;
reduced protection for intellectual property rights;
the existence of additional third-party patent rights of potential relevance to our business;
unexpected changes in tariffs, trade barriers and regulatory requirements;
economic weakness, including inflation, or political instability in particular foreign economies and markets;
compliance with tax, employment, immigration and labor laws for employees living or traveling abroad;
foreign currency fluctuations, which could result in increased operating expenses and reduced revenue, and other obligations incident to doing
business in another country;
foreign reimbursement, pricing and insurance regimes;
production shortages resulting from any events affecting material supply or manufacturing capabilities abroad; and
business interruptions resulting from geopolitical actions, including war and terrorism or natural disasters including earthquakes, typhoons,
floods and fires.
The failure to comply with applicable foreign regulatory requirements may result in, among other things, fines, suspension, variation or withdrawal
of regulatory approvals, product recalls, seizure of products, operating restrictions and criminal prosecution.
Additionally, on June 23, 2016, the electorate in the United Kingdom voted in favor of leaving the European Union, commonly referred to as
Brexit. Following protracted negotiations, the United Kingdom left the European Union on January 31, 2020. Under the withdrawal agreement, there is a
transitional period until December 31, 2020, which period is extendable up to two years. Discussions between the United Kingdom and the European
Union have so far mainly focused on finalizing withdrawal issues and transition agreements but have been extremely difficult to date. To date, only an
outline of a trade agreement has been reached. Much remains open but the United Kingdom’s Prime Minister has indicated that the United Kingdom will
not seek to extend the transitional period beyond the end of 2020. If no trade agreement has been reached before the end of the transitional period, there
may be significant market and economic disruption. The Prime Minister has also indicated that the UK will not accept high regulatory alignment with the
EU.
Since the regulatory framework for pharmaceutical products in the United Kingdom covering quality, safety, and efficacy of pharmaceutical
products, clinical trials, marketing authorization, commercial sales, and distribution of pharmaceutical products is derived from European Union directives
and regulations, Brexit could materially impact the future regulatory regime that applies to products and the approval of product candidates in the United
Kingdom. Any delay in obtaining, or an inability to obtain, any marketing approvals, as a result of Brexit or otherwise, may force us to restrict or delay
efforts to seek regulatory approval in the United Kingdom and/or European Union for our product candidates, which could significantly and materially
harm our business.
If we engage in future acquisitions or strategic collaborations, this may increase our capital requirements, dilute our stockholders, cause us to incur
debt or assume contingent liabilities and subject us to other risks.
We may evaluate various acquisitions and strategic collaborations, including licensing or acquiring complementary products, intellectual property
rights, technologies or businesses. Any potential acquisition or strategic collaboration may entail numerous risks, including:
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increased operating expenses and cash requirements;
the assumption of additional indebtedness or contingent liabilities;
assimilation of operations, intellectual property and products of an acquired company, including difficulties associated with integrating new
personnel;
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the diversion of our management’s attention from our existing product candidates and initiatives in pursuing such acquisition or strategic
collaboration;
retention of key employees, the loss of key personnel and uncertainties in our ability to maintain key business relationships;
risks and uncertainties associated with the other party to such a transaction, including the prospects of that party and their existing products or
product candidates and regulatory approvals; and
our inability to generate revenue from acquired technology and/or products sufficient to meet our objectives in undertaking the acquisition or
collaboration or even to offset transaction costs.
In addition, if we undertake acquisitions, we may issue dilutive securities, assume or incur debt obligations, incur large one-time expenses and
acquire intangible assets that could result in significant future amortization expense. Moreover, we may not be able to locate suitable acquisition or
collaboration opportunities and this inability could impair our ability to grow or obtain access to technology or products that may be important to the
development of our business.
Risks related to our business operations
Our future success depends on our ability to retain key employees, consultants and advisors and to attract, retain and motivate qualified personnel.
We are highly dependent on members of our executive team, the loss of whose services may adversely impact the achievement of our objectives.
While we have entered into employment agreements with certain of our executive officers, any of them could leave our employment at any time. We
currently do not have “key person” insurance on any of our employees. The loss of the services of one or more of our current key employees might impede
the achievement of our research, development and commercialization objectives.
Recruiting and retaining other qualified employees, consultants and advisors for our business, including scientific and technical personnel, also will
be critical to our success. There currently is a shortage of skilled individuals with substantial gene therapy experience, which is likely to continue. As a
result, competition for skilled personnel, including in gene therapy research and vector manufacturing, is intense and the turnover rate can be high. We may
not be able to attract and retain personnel on acceptable terms given the competition among numerous pharmaceutical and biotechnology companies and
academic institutions for individuals with similar skill sets. In addition, the failure to succeed in preclinical or clinical trials or applications for marketing
approval may make it more challenging to recruit and retain qualified personnel. The inability to recruit, or loss of services of certain executives, key
employees, consultants or advisors, may impede the progress of our research, development and commercialization objectives.
Our strategic plan and the associated workforce reduction announced in January 2020 may not result in anticipated savings, could result in total costs
and expenses that are greater than expected and could disrupt our business.
In January 2020, we announced a reduction in workforce by approximately one third as part of a strategic plan designed to create a leaner company
focused on advancing SGT-001. We may not realize, in full or in part, the anticipated benefits, savings and improvements in our cost structure from our
restructuring efforts due to unforeseen difficulties, delays or unexpected costs. If we are unable to realize the expected operational efficiencies and cost
savings from the restructuring, our operating results and financial condition would be adversely affected. We also cannot guarantee that we will not have to
undertake additional workforce reductions or restructuring activities in the future. Furthermore, our strategic restructuring plan may be disruptive to our
operations. For example, our workforce reductions could yield unanticipated consequences, such as attrition beyond planned staff reductions, or increase
difficulties in our day-to-day operations. Our workforce reductions could also harm our ability to attract and retain qualified management, scientific,
clinical, manufacturing and sales and marketing personnel who are critical to our business. Any failure to attract or retain qualified personnel could prevent
us from successfully developing and commercializing our product candidates in the future.
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If we are unable to manage growth in the scale and complexity of our operations, our performance may suffer.
If we are successful in executing our business strategy, we will need to expand our managerial, operational, financial and other systems and
resources to manage our operations, continue our research and development activities and, in the longer term, build a commercial infrastructure to support
commercialization of SGT-001 and any other product candidate that is approved for sale. Future growth would impose significant added responsibilities on
members of management. It is likely that our management, finance, development personnel, systems and facilities currently in place may not be adequate to
support this future growth. Our need to effectively manage our operations, growth and any future product candidates requires that we continue to develop
more robust business processes and improve our systems and procedures in each of these areas and to attract and retain sufficient numbers of talented
employees. We may be unable to successfully implement these tasks on a larger scale and, accordingly, may not achieve our research, development and
growth goals.
Our employees may engage in misconduct or other improper activities, including non-compliance with regulatory standards and requirements, which
could cause significant liability for us and harm our reputation.
We are exposed to the risk of employee fraud or other misconduct, including intentional failures to comply with FDA regulations or similar
regulations of comparable foreign regulatory authorities, provide accurate information to the FDA or comparable foreign regulatory authorities, comply
with manufacturing standards, comply with federal and state healthcare fraud and abuse laws and regulations and similar laws and regulations established
and enforced by comparable foreign regulatory authorities, report financial information or data accurately or disclose unauthorized activities to us.
Employee misconduct could also involve the improper use of information obtained in the course of clinical trials, which could result in regulatory sanctions
and serious harm to our reputation. This could include violations of the federal Health Insurance Portability and Accountability Act of 1996, or HIPAA,
other U.S. federal and state law, and requirements of non-U.S. jurisdictions, including the European Union Data Protection Directive. It is not always
possible to identify and deter employee misconduct, and the precautions we take to detect and prevent this activity may not be effective in controlling
unknown or unmanaged risks or losses or in protecting us from governmental investigations or other actions or lawsuits stemming from a failure to be in
compliance with such laws, standards, regulations, guidance or codes of conduct. If any such actions are instituted against us, and we are not successful in
defending ourselves or asserting our rights, those actions could have a significant impact on our business and results of operations, including the imposition
of significant fines or other sanctions.
Enacted and future legislation may increase the difficulty and cost for us to obtain marketing approval of and commercialize our product candidates
and may affect the prices we may set.
Our business and financial prospects could be affected by changes in health care spending and policy in the United States and abroad. We operate in
a highly regulated industry and new laws or judicial decisions, or new interpretations of existing laws or decisions, related to health care availability, the
method of delivery or payment for health care products and services could negatively impact our business, operations and financial condition.
For example, in the United States there is significant interest in promoting health care reform, as evidenced by the enactment of the Patient
Protection and Affordable Care Act and the companion Health Care and Education Reconciliation Act, or the Health Care Reform Law. The Health Care
Reform Law increased federal oversight of private health insurance plans and included a number of provisions designed to reduce Medicare expenditures
and the cost of health care generally, to reduce fraud and abuse, and to provide access to increased health coverage.
The Health Care Reform Law also imposed substantial changes to the U.S. system for paying for health care, including programs to extend medical
benefits to millions of individuals who have lacked insurance coverage. Generally, implementation of the Health Care Reform Law has thus far included
significant cost-saving, revenue and payment reduction measures with respect to, for example, several government health care programs that might cover
our products in the United States, should they be commercialized, including Medicaid and Medicare. Additional downward pricing pressure associated
with the Health Care Reform Law includes that the Health Care Reform Law established and provided significant funding for a Patient-Centered Outcomes
Research Institute to coordinate and fund Comparative Effectiveness Research, as those terms are defined in the Health Care Reform Law. While the stated
intent of Comparative Effectiveness Research is to develop information to guide providers to the most efficacious therapies, outcomes of Comparative
Effectiveness Research could influence the reimbursement or coverage for therapies that are determined to be less cost-effective than others. Should any of
our products be approved for sale, but then determined to be less cost-effective than alternative therapies, the levels of reimbursement for these products, or
the willingness to reimburse at all, could be adversely impacted.
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Another provision of the Health Care Reform Law, generally referred to as the Physician Payment Sunshine Act or Open Payments Program, has
imposed new reporting and disclosure requirements for pharmaceutical and medical device manufacturers and distributors with certain FDA-approved
products, such as approved vaccines, with regard to payments or other transfers of value made to certain U.S. health care practitioners, such as physicians
and academic medical centers, and with regard to certain ownership interests held by physicians in reporting entities. The CMS publishes information from
these reports on a publicly available website, including amounts transferred and the physician and teaching hospital identities.
Under the Physician Payment Sunshine Act, we are required to collect and report detailed information regarding certain financial relationships we
have with physicians and teaching hospitals if we obtain FDA approval for any of our products and receive reimbursement from the federal government.
Our compliance with these rules may also impose additional costs.
With enactment of the Tax Cuts and Jobs Act of 2017, which was signed by the President on December 22, 2017, Congress repealed the “individual
mandate.” The repeal of this provision, which requires most Americans to carry a minimal level of health insurance, became effective in 2019.
Additionally, the 2020 federal spending package permanently eliminated, effective January 1, 2020, the Health Care Reform Law-mandated “Cadillac” tax
on high-cost employer-sponsored health coverage and medical device tax and, effective January 1, 2021, also eliminates the health insurer tax. Further, the
Bipartisan Budget Act of 2018, among other things, amended the Health Care Reform Law, effective January 1, 2019, to increase from 50 percent to 70
percent the point-of-sale discount that is owed by pharmaceutical manufacturers who participate in Medicare Part D and to close the coverage gap in most
Medicare drug plans, commonly referred to as the “donut hole.” The Congress may consider other legislation to replace elements of the Health Care
Reform Law during the next Congressional session.
We will continue to evaluate the effect that the Health Care Reform Law and its possible repeal and replacement could have on our business. It is
possible that repeal and replacement initiatives, if enacted into law, could ultimately result in fewer individuals having health insurance coverage or in
individuals having insurance coverage with less generous benefits. While the timing and scope of any potential future legislation to repeal and replace
Health Care Reform Law provisions is uncertain in many respects, it is also possible that some of the Health Care Reform Law provisions that generally
are not favorable for the research-based pharmaceutical industry could also be repealed along with Health Care Reform Law coverage expansion
provisions. Accordingly, such reforms, if enacted, could have an adverse effect on anticipated revenue from product candidates that we may successfully
develop and for which we may obtain marketing approval and may affect our overall financial condition and ability to develop commercialize product
candidates.
The Trump administration has also taken executive actions to undermine or delay implementation of the Health Care Reform Law. Since January
2017, President Trump has signed two Executive Orders designed to delay the implementation of certain provisions of the Health Care Reform Law or
otherwise circumvent some of the requirements for health insurance mandated by the Health Care Reform Law. One Executive Order directs federal
agencies with authorities and responsibilities under the Health Care Reform Law to waive, defer, grant exemptions from, or delay the implementation of
any provision of the Health Care Reform Law that would impose a fiscal or regulatory burden on states, individuals, healthcare providers, health insurers,
or manufacturers of pharmaceuticals or medical devices. The second Executive Order terminates the cost-sharing subsidies that reimburse insurers under
the Health Care Reform Law. Several state Attorneys General filed suit to stop the administration from terminating the subsidies, but their request for a
restraining order was denied by a federal judge in California on October 25, 2017. In addition, CMS has recently proposed regulations that would give
states greater flexibility in setting benchmarks for insurers in the individual and small group marketplaces, which may have the effect of relaxing the
essential health benefits required under the Health Care Reform Law for plans sold through such marketplaces. Further, on June 14, 2018, U.S. Court of
Appeals for the Federal Circuit ruled that the federal government was not required to pay more than $12 billion in Health Care Reform Law risk corridor
payments to third-party payors who argued such payments were owed to them. This decision is under review by the U.S. Supreme Court during its current
term. The full effect of this gap in reimbursement on third-party payors, the viability of the Health Care Reform Law marketplace, providers, and
potentially our business, are not yet known.
In addition, the CMS has proposed regulations that would give states greater flexibility in setting benchmarks for insurers in the individual and
small group marketplaces, which may have the effect of relaxing the essential health benefits required under the Health Care Reform Law for plans sold
through such marketplaces. On November 30, 2018, CMS announced a proposed rule that would amend the Medicare Advantage and Medicare Part D
prescription drug benefit regulations to reduce out-of-pocket costs for plan enrollees and allow Medicare plans to negotiate lower rates for certain drugs.
Among other things, the proposed rule changes would allow Medicare Advantage plans to use pre authorization, or PA, and step therapy, or ST, for six
protected classes of drugs, with certain exceptions; permit plans to implement PA and ST in Medicare Part B drugs; and change the definition of
“negotiated prices” as well as add a definition of “price concession” in the regulations. It is unclear whether these proposed changes will be accepted, and if
so, what effect such changes will have on our business.
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Further, on December 14, 2018, a U.S. District Court judge in the Northern District of Texas ruled that the individual mandate portion of the Health
Care Reform Law is an essential and inseverable feature of the Health Care Reform Law, and therefore because the mandate was repealed as part of the Tax
Cuts and Jobs Act of 2017, the remaining provisions of the Health Care Reform Law are invalid as well. The Trump administration and the CMS have both
stated that the ruling will have no immediate effect, and on December 30, 2018, the same judge issued an order staying the judgment pending appeal. The
Trump administration thereafter represented to the Court of Appeals considering this judgment that it does not oppose the district court’s ruling. On July 10,
2019, the Court of Appeals for the Fifth Circuit heard oral argument in this case. In those arguments, the Trump administration argued in support of
upholding the lower court decision. On December 18, 2019, the Court of Appeals for the Fifth Circuit affirmed the lower court’s ruling that the individual
mandate portion of the Health Care Reform Law is unconstitutional and it remanded the case to the district court for reconsideration of the severability
question and additional analysis of the provisions of the Health Care Reform Law. On January 21, 2020, the U.S. Supreme Court declined to review this
decision on an expedited basis. It is unclear how this decision and any subsequent appeals and other efforts to repeal and replace the Health Care Reform
Law will impact the Health Care Reform Law and our business. Litigation and legislation over the Health Care Reform Law are likely to continue, with
unpredictable and uncertain results.
The costs of prescription pharmaceuticals in the United States has also been the subject of considerable discussion in the United States, and
members of Congress and the Administration have stated that they will address such costs through new legislative and administrative measures. The
pricing of prescription pharmaceuticals is also subject to governmental control outside the United States. In these countries, pricing negotiations with
governmental authorities can take considerable time after the receipt of marketing approval for a product. To obtain reimbursement or pricing approval in
some countries, we may be required to conduct a clinical trial that compares the cost effectiveness of our product candidates to other available therapies. If
reimbursement of our products is unavailable or limited in scope or amount, or if pricing is set at unsatisfactory levels, our ability to generate revenues and
become profitable could be impaired.
Specifically, there have been several recent U.S. congressional inquiries and proposed federal and proposed and enacted state legislation designed
to, among other things, bring more transparency to drug pricing, review the relationship between pricing and manufacturer patient programs, reduce the
costs of drugs under Medicare and reform government program reimbursement methodologies for drug products. At the federal level, Congress and the
Trump administration have each indicated that it will continue to seek new legislative and/or administrative measures to control drug costs. For example,
on May 11, 2018, the Administration issued a plan to lower drug prices. Under this blueprint for action, the Administration indicated that the Department of
Health and Human Services will: take steps to end the gaming of regulatory and patent processes by drug makers to unfairly protect monopolies; advance
biosimilars and generics to boost price competition; evaluate the inclusion of prices in drug makers’ ads to enhance price competition; speed access to and
lower the cost of new drugs by clarifying policies for sharing information between insurers and drug makers; avoid excessive pricing by relying more on
value-based pricing by expanding outcome-based payments in Medicare and Medicaid; work to give Part D plan sponsors more negotiation power with
drug makers; examine which Medicare Part B drugs could be negotiated for a lower price by Part D plans, and improving the design of the Part B
Competitive Acquisition Program; update Medicare’s drug-pricing dashboard to increase transparency; prohibit Part D contracts that include “gag rules”
that prevent pharmacists from informing patients when they could pay less out-of-pocket by not using insurance; and require that Part D plan members be
provided with an annual statement of plan payments, out-of-pocket spending, and drug price increases. In addition, on December 23, 2019, the Trump
administration published a proposed rulemaking that, if finalized, would allow states or certain other non-federal government entities to submit importation
program proposals to the FDA for review and approval. Applicants would be required to demonstrate their importation plans pose no additional risk to
public health and safety and will result in significant cost savings for consumers. At the same time, the FDA issued draft guidance that would allow
manufacturers to import their own FDA-approved drugs that are authorized for sale in other countries (multi-market approved products).
At the state level, individual states are increasingly aggressive in passing legislation and implementing regulations designed to control
pharmaceutical and biological product pricing, including price or patient reimbursement constraints, discounts, restrictions on certain product access and
marketing cost disclosure and transparency measures, and, in some cases, designed to encourage importation from other countries and bulk purchasing. In
addition, regional health care authorities and individual hospitals are increasingly using bidding procedures to determine what pharmaceutical products and
which suppliers will be included in their prescription drug and other health care programs. These measures could reduce the ultimate demand for our
products, if approved, or put pressure on our product pricing. We expect that additional state and federal healthcare reform measures will be adopted in the
future, any of which could limit the amounts that federal and state governments will pay for healthcare products and services, which could result in reduced
demand for our product candidates or additional pricing pressures.
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The Drug Supply Chain Security Act imposes new obligations on manufacturers of pharmaceutical products related to product tracking and tracing.
Legislative and regulatory proposals have been made to expand post-approval requirements and restrict sales and promotional activities for pharmaceutical
products. We are not sure whether additional legislative changes will be enacted, or whether the current regulations, guidance or interpretations will be
changed, or what the impact of such changes on our business, if any, may be.
There have been a number of federal and state legislative changes made over the last few years regarding the pricing of pharmaceutical and biologic
products. Concerns about drug pricing have been expressed by members of Congress and the President.
It is likely that federal and state legislatures within the United States and foreign governments will continue to consider changes to existing health
care legislation. We cannot predict the reform initiatives that may be adopted in the future or whether initiatives that have been adopted will be repealed or
modified. The continuing efforts of the government, insurance companies, managed care organizations and other health care payors of to contain or reduce
costs of health care may adversely affect:
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the demand for any product candidates for which we may obtain regulatory approval;
our ability to set a price that we believe is fair for our products;
our ability to obtain coverage and reimbursement approval for a product;
our ability to generate revenue and achieve or maintain profitability; and
the level of taxes that we are required to pay.
Our relationships with customers, physicians and third-party payors will be subject, directly or indirectly, to federal and state health care fraud and
abuse laws, false claims laws, health information privacy and security laws, and other health care laws and regulations. If we are unable to comply, or
have not fully complied, with such laws, we could face substantial penalties.
If we obtain FDA approval for SGT-001 or our other product candidates and begin commercializing those products in the United States, our
operations will be directly or indirectly through our prescribers, customers and purchasers, subject to various federal and state fraud and abuse laws and
regulations, including, without limitation, the federal Health Care Program Anti-Kickback Statute, the federal civil and criminal laws and the Physician
Payment Sunshine Act and regulations. These laws will impact, among other things, our proposed sales, marketing and educational programs. In addition,
we may be subject to patient privacy laws by both the federal government and the states in which we conduct our business. The laws that will affect our
operations include, but are not limited to:
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the federal Health Care Program Anti-Kickback Statute, which prohibits, among other things, persons or entities from knowingly and willfully
soliciting, receiving, offering or paying any remuneration (including any kickback, bribe or rebate), directly or indirectly, overtly or covertly,
in cash or in kind, in return for the purchase, recommendation, leasing or furnishing of an item or service reimbursable under a federal health
care program, such as the Medicare and Medicaid programs. This statute has been interpreted to apply to arrangements between
pharmaceutical manufacturers on the one hand, and prescribers, purchasers and formulary managers on the other. The Health Care Reform
Law amended the intent requirement of the federal Anti-Kickback Statute. A person or entity no longer needs to have actual knowledge of this
statute or specific intent to violate it;
federal civil and criminal false claims laws and civil monetary penalty laws, which prohibit, among other things, individuals or entities from
knowingly presenting, or causing to be presented, claims for payment or approval from Medicare, Medicaid or other government payors that
are false or fraudulent. The Health Care Reform Law provides and recent government cases against pharmaceutical and medical device
manufacturers support the view that Federal Anti-Kickback Statute violations and certain marketing practices, including off-label promotion,
may implicate the False Claims Act;
HIPAA, which created new federal criminal statutes that prohibit a person from knowingly and willfully executing a scheme or from making
false or fraudulent statements to defraud any health care benefit program, regardless of the payor (e.g., public or private);
HIPAA, as amended by the Health Information Technology for Economic and Clinical Health Act, or HITECH, and its implementing
regulations, and as amended again by the final HIPAA omnibus rule, Modifications to the HIPAA Privacy, Security, Enforcement, and Breach
Notification Rules Under HITECH and the Genetic Information Nondiscrimination Act; Other Modifications to HIPAA, published in January
2013, which imposes certain requirements relating to the privacy, security and transmission of individually identifiable health information
without appropriate authorization by entities subject to the rule, such as health plans, health care clearinghouses and health care providers;
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federal transparency laws, including the federal Physician Payment Sunshine Act, that require certain manufacturers of drugs, devices,
biologics and medical supplies for which payment is available under Medicare, Medicaid or the Children’s Health Insurance Program, with
specific exceptions, to report annually to the CMS information related to: (i) payments or other “transfers of value” made to physicians and
teaching hospitals and (ii) ownership and investment interests held by physicians and their immediate family members;
state and foreign law equivalents of each of the above federal laws, state laws that require drug manufacturers to report information related to
payments and other transfers of value to physicians and other health care providers or marketing expenditures and state laws governing the
privacy and security of health information in certain circumstances, many of which differ from each other in significant ways and may not
have the same effect, thus complicating compliance efforts in certain circumstances, such as specific disease states; and
state and foreign laws that govern the privacy and security of health information in some circumstances, many of which differ from each other
in significant ways and often are not preempted by HIPAA, thus complicating compliance efforts.
Because of the breadth of these laws and the narrowness of the statutory exceptions and safe harbors available, it is possible that some of our
business activities could be subject to challenge under one or more of such laws. If our operations are found to be in violation of any of the laws described
above or any other government regulations that apply to us, we may be subject to penalties, including civil and criminal penalties, damages, fines,
exclusion from participation in government health care programs, such as Medicare and Medicaid, imprisonment and the curtailment or restructuring of our
operations.
The risk of our being found in violation of these laws is increased by the fact that many of them have not been fully interpreted by the regulatory
authorities or the courts, and their provisions are open to a variety of interpretations. Any action against us for violation of these laws, even if we
successfully defend against it, could cause us to incur significant legal expenses and divert our management’s attention from the operation of our business.
The shifting compliance environment and the need to build and maintain robust and expandable systems to comply with multiple jurisdictions with
different compliance and/or reporting requirements increases the possibility that we may run afoul of one or more of the requirements.
Compliance with global privacy and data security requirements could result in additional costs and liabilities to us or inhibit our ability to collect and
process data globally, and the failure to comply with such requirements could subject us to significant fines and penalties, which may have a material
adverse effect on our business, financial condition or results of operations.
The regulatory framework for the collection, use, safeguarding, sharing, transfer and other processing of information worldwide is rapidly evolving
and is likely to remain uncertain for the foreseeable future. Globally, virtually every jurisdiction in which we operate has established its own data security
and privacy frameworks with which we must comply. For example, the collection, use, disclosure, transfer, or other processing of personal data regarding
individuals in the European Union, including personal health data, is subject to the General Data Protection Regulation, or GDPR, which took effect across
all member states of the European Economic Area, or EEA, in May 2018. The GDPR is wide-ranging in scope and imposes numerous requirements on
companies that process personal data, including requirements relating to processing health and other sensitive data, obtaining consent of the individuals to
whom the personal data relates, providing information to individuals regarding data processing activities, implementing safeguards to protect the security
and confidentiality of personal data, providing notification of data breaches, and taking certain measures when engaging third-party processors. The GDPR
increases our obligations with respect to clinical trials conducted in the EEA by expanding the definition of personal data to include coded data and
requiring changes to informed consent practices and more detailed notices for clinical trial subjects and investigators. In addition, the GDPR also imposes
strict rules on the transfer of personal data to countries outside the European Union, including the United States and, as a result, increases the scrutiny that
clinical trial sites located in the EEA should apply to transfers of personal data from such sites to countries that are considered to lack an adequate level of
data protection, such as the United States. The GDPR also permits data protection authorities to require destruction of improperly gathered or used personal
information and/or impose substantial fines for violations of the GDPR, which can be up to four percent of global revenues or 20 million Euros, whichever
is greater, and it also confers a private right of action on data subjects and consumer associations to lodge complaints with supervisory authorities, seek
judicial remedies, and obtain compensation for damages resulting from violations of the GDPR. In addition, the GDPR provides that European Union
member states may make their own further laws and regulations limiting the processing of personal data, including genetic, biometric or health data.
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Given the breadth and depth of changes in data protection obligations, preparing for and complying with the GDPR’s requirements is rigorous and
time intensive and requires significant resources and a review of our technologies, systems and practices, as well as those of any third-party collaborators,
service providers, contractors or consultants that process or transfer personal data collected in the European Union. The GDPR and other changes in laws or
regulations associated with the enhanced protection of certain types of sensitive data, such as healthcare data or other personal information from our
clinical trials, could require us to change our business practices and put in place additional compliance mechanisms, may interrupt or delay our
development, regulatory and commercialization activities and increase our cost of doing business, and could lead to government enforcement actions,
private litigation and significant fines and penalties against us and could have a material adverse effect on our business, financial condition or results of
operations. Similarly, failure to comply with federal and state laws regarding privacy and security of personal information could expose us to fines and
penalties under such laws. Even if we are not determined to have violated these laws, government investigations into these issues typically require the
expenditure of significant resources and generate negative publicity, which could harm our reputation and our business.
Product liability lawsuits against us could cause us to incur substantial liabilities and could limit commercialization of any product candidate that we
may develop.
We face an inherent risk of product liability exposure related to the testing of SGT-001, our other product candidates and any future product
candidate in preclinical studies and clinical trials and may face an even greater risk if we commercialize any product candidate that we may develop. If we
cannot successfully defend ourselves against claims that our product candidates caused injuries, we could incur substantial liabilities. Regardless of merit
or eventual outcome, liability claims may result in:
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decreased demand for any product candidate that we may develop;
loss of revenue;
substantial monetary awards to trial participants or patients;
significant time and costs to defend the related litigation;
withdrawal of clinical trial participants;
the inability to commercialize any of our product candidates; and
injury to our reputation and significant negative media attention.
Although we maintain product liability insurance coverage, such insurance may not be adequate to cover all liabilities that we may incur. We
anticipate that we will need to increase our insurance coverage each time we commence a clinical trial and if we successfully commercialize any product
candidate. Insurance coverage is increasingly expensive. We may not be able to maintain insurance coverage at a reasonable cost or in an amount adequate
to satisfy any liability that may arise.
If we fail to comply with environmental, health and safety laws and regulations, we could become subject to fines or penalties or incur costs that could
have a material adverse effect on the success of our business.
We are subject to numerous environmental, health and safety laws and regulations, including those governing laboratory procedures and the
generation, handling, use, storage, treatment, manufacture, transportation and disposal of, and exposure to, hazardous materials and wastes, as well as laws
and regulations relating to occupational health and safety. Our operations involve the use of hazardous and flammable materials, including chemicals and
viruses and other biologic materials. Our operations also produce hazardous waste products. We generally contract with third parties for the disposal of
these materials and wastes. We cannot eliminate the risk of contamination or injury from these materials. In the event of contamination or injury resulting
from our use of hazardous materials, we could be held liable for any resulting damages. We also could incur significant costs associated with civil or
criminal fines and penalties. We do not carry specific biological or hazardous waste insurance coverage, and our property, casualty and general liability
insurance policies specifically exclude coverage for damages and fines arising from biological or hazardous waste exposure or contamination. Although we
maintain workers’ compensation insurance for certain costs and expenses we may incur due to injuries to our employees resulting from the use of
hazardous materials or other work-related injuries, this insurance may not provide adequate coverage against potential liabilities. Accordingly, in the event
of contamination or injury, we could be held liable for damages or be penalized with fines in an amount exceeding our resources, and our clinical trials or
regulatory approvals could be suspended.
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In addition, we may incur substantial costs in order to comply with current or future environmental, health and safety laws and regulations, which
have tended to become more stringent over time. These current or future laws and regulations may impair our research, development or production efforts.
Failure to comply with these laws and regulations also may result in substantial fines, penalties or other sanctions or liabilities.
Our internal computer systems, or those of our collaborators, contractors or consultants, may fail or suffer security breaches, which could result in a
material disruption of our product development.
Despite the implementation of security measures, our internal computer systems and those of our current and any future collaborators and other
contractors or consultants are vulnerable to damage from computer viruses, unauthorized access, natural disasters, terrorism, war and telecommunication
and electrical failures. Such systems are also vulnerable to service interruptions or to security breaches from inadvertent or intentional actions by our
employees, third-party vendors and/or business partners, or from cyber-attacks by malicious third parties. Cyber-attacks are increasing in their frequency,
sophistication and intensity, and have become increasingly difficult to detect. Cyber-attacks could include the deployment of harmful malware,
ransomware, denial-of-service attacks, social engineering and other means to affect service reliability and threaten the confidentiality, integrity and
availability of information. Cyber-attacks also could include phishing attempts or e-mail fraud to cause payments or information to be transmitted to an
unintended recipient.
While we are not aware of any such material system failure, accident, cyber-attack or security breach to date, if such an event were to occur and
cause interruptions in our or our collaborators’, contractors’ or consultants’ operations, it could result in a material disruption of our development programs
and our business operations, whether due to a loss of our trade secrets or other proprietary information or other similar disruptions. For example, the loss of
clinical trial data from preclinical studies or clinical trials could result in delays in our regulatory approval efforts and significantly increase our costs to
recover or reproduce the data. To the extent that any disruption or security breach were to result in a loss of, or damage to, our data or applications, or
inappropriate disclosure of confidential or proprietary information, we could incur liability, our competitive position could be harmed and the further
development and commercialization of SGT-001 and our other product candidates could be delayed.
Risks related to our intellectual property
We heavily rely on certain in-licensed patents and other intellectual property rights in connection with our development of SGT-001 and may be
required to acquire or license additional patents or other intellectual property rights to continue to develop and commercialize SGT-001.
Our ability to develop and commercialize SGT-001 and other product candidates is heavily dependent on licenses to patent rights and other
intellectual property granted to us by third parties. In particular, we have licensed certain patents and patent applications from the University of Michigan,
the University of Missouri and the University of Washington that are important or necessary to the development of SGT-001 and other elements of our gene
transfer program. Our existing license agreements impose, and we expect that future license agreements will impose, various diligence, development and
commercialization obligations, milestone payments, royalties and other obligations on us. If we fail to comply with our obligations under these agreements,
we may be subject to damages, which may be significant, and the licensor may have the right to terminate the license, in which event we may not be able to
develop or market product candidates or technologies covered by the license, including SGT-001. In addition, certain of these license agreements are not
assignable by us without the consent of the respective licensor, which may have an adverse effect on our ability to engage in certain transactions.
Under our existing license agreements, we do not have, and under future license agreements we may not have, the right to control the preparation,
filing and prosecution of patent applications, or the maintenance, enforcement and defense of the patents and patent applications that we license from third
parties. For example, under our inbound license agreements with the University of Michigan, the University of Missouri and the University of Washington,
each of the applicable licensors controls the prosecution of patent applications and the maintenance of patents and patent applications. Therefore, we cannot
be certain that these patents and applications will be prosecuted, maintained, enforced and defended in a manner consistent with the best interests of our
business. If our licensors fail to maintain, enforce or defend such patents, or lose rights to those patents or patent applications, the rights we have licensed
may be reduced or eliminated and our right to develop and commercialize any of our product candidates that are the subject of such licensed rights,
including SGT-001, could be adversely affected. For more information, see Part I, Item 1, “Business—Strategic partnerships and collaborations/licenses” of
this annual report on Form 10-K.
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Moreover, licenses to additional third-party intellectual property, technology and materials are required for our development programs but may not
be available in the future or may not be available on commercially reasonable terms. For example, we are aware of certain third-party patents related to
certain microdystrophin constructs, which, if in force at the time of SGT-001’s commercialization, may be claimed by third parties to cover SGT-001. In
addition, third parties may claim that the AAV vector we are developing for use in SGT-001 is covered by patents held by them. We believe that we would
have valid defenses to any such claims; however, if any such claims were ultimately successful, we might require a license to continue to use and sell SGT-
001 and such AAV vector. Such licenses may not be available on commercially reasonable terms, or at all. The licensing or acquisition of third-party
intellectual property rights is a competitive area, and several more established companies may pursue strategies to license or acquire third-party intellectual
property rights that we may consider attractive. These established companies may have a competitive advantage over us due to their size, capital resources
and greater clinical development and commercialization capabilities. In addition, companies that perceive us to be a competitor may be unwilling to assign
or license rights to us. We also may be unable to license or acquire third-party intellectual property rights on terms that would allow us to make an
appropriate return on our investment. Moreover, even if we are able to obtain such licenses, they may only be non-exclusive, which could permit
competitors and other third parties to use the same intellectual property in competition with us.
We may collaborate with non-profit and academic institutions to accelerate our preclinical research or development under written agreements with
these institutions. These institutions may provide us with an option to negotiate a license to any of the institution’s rights in technology resulting from the
collaboration. Regardless of such option, we may be unable to negotiate a license within the required timeframe or under terms that are acceptable to us. If
we are unable to do so, the institution may offer the intellectual property rights to other parties, potentially blocking our ability to pursue our program.
If we are unable to successfully obtain rights to any third-party intellectual property rights that are required for the development and
commercialization of SGT-001 or any of our other product candidates, and such third-party intellectual property rights are successfully asserted against us,
we may be liable for damages, which may be significant, and we may be required to cease the development and commercialization of SGT-001 or our other
product candidates.
If we are unable to obtain and maintain patent protection for our product candidates, or if the scope of the patent protection obtained is not sufficiently
broad, our competitors could develop and commercialize products similar or identical to ours, and our ability to successfully commercialize our product
candidates may be adversely affected.
Our success depends, in large part, on our and our licensors’ ability to seek, obtain, maintain, enforce and defend patent rights in the United States
and other countries with respect to SGT-001, our other product candidates and our future innovation related to our manufacturing technology. Our licensors
and we have sought, and we intend to continue to seek, to protect our proprietary position by filing patent applications in the United States and, in at least
some cases, one or more countries outside the United States related to SGT-001 and certain other product candidates that are important to our business.
However, we cannot predict whether the patent applications we and our licensors are currently pursuing will issue as patents or whether the claims of any
issued patents will provide us with a competitive advantage.
Moreover, although we have pending patent applications in the United States and abroad, we cannot predict whether or in which jurisdictions the
pending applications will result in issuance of patents that effectively protect any of our product candidates or will effectively prevent others from
commercializing competitive products. Further, each of the provisional patent applications is not eligible to become an issued patent until, among other
things, we file a non-provisional patent application within 12 months of the filing date of each provisional patent application. If we do not timely file a non-
provisional patent application in respect of a provisional patent application, we may lose our priority date with respect to such provisional patent
application and any patent protection on the inventions disclosed in such provisional patent application. While we intend to timely file non-provisional
patent applications relating to our provisional patent applications, we cannot predict whether such future patent applications will result in the issuance of
patents that effectively protect any of our product candidates or will effectively prevent others from commercializing competitive products.
We may not be able to file, prosecute, maintain, enforce, defend or license all patents that are necessary to our business.
The patent prosecution process is expensive, time-consuming and complex, and we and our licensors may not be able to file, prosecute, maintain,
enforce, defend or license all necessary or desirable patents and patent applications at a reasonable cost or in a timely manner.
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It is also currently unknown what claims may, if ever, issue from pending applications included in our patent rights. Additionally, certain of our in-
licensed U.S. patent rights lack corresponding foreign patents or patent applications, and therefore we will be unable to obtain patent protection for our
product candidates in certain jurisdictions. We or our licensors may not be able to obtain or maintain patent protection with respect to SGT-001 or our other
product candidates.
Changes in either the patent laws or their interpretation in the United States and other countries may diminish our ability to protect our inventions,
obtain, maintain and enforce our intellectual property rights, and more generally, could affect the value of our intellectual property rights or narrow the
scope of our licensed patents or future owned patents.
It is also possible that we will fail to identify patentable aspects of our research and development output before it is too late to obtain patent
protection. Although we enter into non-disclosure and confidentiality agreements with parties who have access to confidential or patentable aspects of our
research and development output, such as our employees, corporate collaborators, outside scientific collaborators, CROs, contract manufacturers,
consultants, advisors and other third parties, any of these parties may breach the agreements and disclose such output before a patent application is filed,
thereby jeopardizing our ability to seek patent protection.
The patent position of biotechnology and pharmaceutical companies generally is highly uncertain, involves complex legal and factual questions and
has, in recent years, been the subject of much litigation. As a result, the issuance, scope, validity, enforceability and commercial value of our patent rights
are highly uncertain. Patent applications included in our current and future patent rights may not result in patents being issued that protect our product
candidates, effectively prevent others from commercializing competitive products or otherwise provide any competitive advantage. In fact, patent
applications may not issue as patents at all. Even assuming patents issue from patent applications in which we have rights, changes in either the patent laws
or interpretation of the patent laws in the United States and other jurisdictions may diminish the value of our patents or narrow the scope of our patent
protection.
Other parties have developed products that may be related or competitive to our own and such parties may have filed or may file patent
applications, or may have received or may receive patents, claiming inventions that may overlap or conflict with those claimed in our patent applications or
issued patents. We may not be aware of all third-party intellectual property rights potentially relating to SGT-001 or our other current or future product
candidates. In addition, we cannot provide any assurances that any of the inventions disclosed in our patent applications will be found to be patentable,
including over third-party or our own prior art patents, publications or other disclosures, or will issue as patents. Even if our patent applications issue as
patents, we cannot provide any assurances that such patents will not be challenged or ultimately held to be invalid or unenforceable. Publications of
discoveries in the scientific literature often lag behind the actual discoveries, and patent applications in the United States and in other jurisdictions are
typically not published until 18 months after filing, or, in some cases, at all. Therefore, we cannot know with certainty whether the inventors of our licensed
patents and applications were the first to make the inventions claimed in those patents or pending patent applications, or that they were the first to file for
patent protection of such inventions. Similarly, should we own any issued patents or patent applications in the future, we may not be certain that we were
the first to file for patent protection for the inventions claimed in such patents or patent applications. Furthermore, given the differences in patent laws in
the United States, Europe and other foreign jurisdictions, for example, the availability of grace periods for filing patent applications and what can be
considered as prior art, we cannot make any assurances that any claims in our pending and future patent applications in the United States or other
jurisdictions will issue, or if they do issue, whether they will issue in a form that provides us with any meaningful competitive advantage. Similarly, we
cannot make any assurances that if the patentability, validity, enforceability or scope of our pending or future patents and patent applications in the United
States or foreign jurisdictions are challenged by any third party, that the claims of such pending or future patents and patent applications will survive any
such challenge in a form that provides us with any meaningful competitive advantage. For example, we are aware of certain third-party patents and
publications related to certain microdystrophin constructs. While we believe that our owned or in-licensed patents and patent applications claim novel and
non-obvious features of microdystrophin constructs that are not described in such third-party patents or publications, such third-party patents and
publications may have earlier priority or publication dates and may be asserted as prior art against our owned or in-licensed patents and applications. Any
such challenge, if successful, could limit or eliminate patent protection for our products and product candidates or otherwise materially harm our business.
As a result, the issuance, scope, validity, enforceability and commercial value of our patent rights cannot be predicted with any certainty.
Moreover, the coverage claimed in a patent application can be significantly reduced before the patent is issued, and its scope can be reinterpreted
after issuance. Even if patent applications we license or may own in the future do issue as patents, they may not issue in a form that will provide us with
any meaningful protection, prevent competitors or other third parties from competing with us or otherwise provide us with any competitive advantage. Any
patents that we license or may own in the future may be challenged, narrowed, circumvented, or invalidated by third parties. Consequently, we do not know
whether any of our product candidates will be protectable or remain protected by valid and enforceable patents. Our competitors or other third parties may
be able to circumvent our patents by developing similar or alternative products in a non-infringing manner.
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The degree of patent protection we require to successfully compete in the marketplace may be unavailable. We cannot provide any assurances that
any of the patents or patent applications included in our patent rights include or will include claims with a scope sufficient to protect SGT-001 and our other
product candidates or otherwise provide any competitive advantage. In addition, the laws of foreign countries may not protect our proprietary rights to the
same extent as the laws of the United States. Furthermore, patents have a limited lifespan. In the United States, the natural expiration of a patent is
generally twenty years after it is filed. Certain extensions may be available, however, the life of a patent, and the protection it affords, is limited. Given the
amount of time required for the development, testing and regulatory review of new product candidates, patents protecting such candidates might expire
before or shortly after such candidates are commercialized. As a result, our patent rights may not provide us with adequate and continuing patent protection
sufficient to exclude others from commercializing products similar or identical to our product candidates, including biosimilar versions of such products.
Our licensed patents, and any patents we may own in the future, may be challenged, narrowed, invalidated or held unenforceable.
Even if we acquire patent protection that we expect should enable us to maintain some competitive advantage, third parties, including competitors,
may challenge the validity, enforceability or scope thereof, which may result in such patents being narrowed, invalidated or held unenforceable. In
litigation, a competitor could claim that our in-licensed patents or any patents we may own in the future are not valid or enforceable for a number of
reasons. If a court agrees, we would lose our rights to those challenged patents. Third parties also may raise similar claims before administrative bodies in
the United States or abroad, even outside the context of litigation. Such proceedings could result in the revocation or cancellation of or amendment to our
licensed patents and any patents we may own in the future in such a way that they no longer cover SGT-001 or our other product candidates.
Even if issued, the issuance of a patent is not conclusive as to its inventorship, scope, validity or enforceability, and our current and future patent
rights may be challenged in the courts or patent offices in the United States and abroad. For example, we may be subject to a third-party submission of
prior art to the U.S. Patent and Trademark Office, or USPTO, challenging the validity of one or more claims of patents included in our patent rights. Such
submissions may also be made prior to a patent’s issuance, precluding the granting of a patent based on one of the pending patent applications included in
our patent rights. We may become involved in opposition, derivation, revocation, reexamination, post-grant and inter partes review, or interference
proceedings challenging one or more patents included in our patent rights. For example, competitors may claim that they invented the inventions claimed in
patents or patent applications included in our patent rights, such as the microdystrophin we use in SGT-001, prior to the inventors of such patents or patent
applications, or may have filed one or more patent applications before the filing of the patents or patent applications included in our patent rights. A
competitor who can establish an earlier filing or invention date may also assert that we are infringing their patents and that we therefore cannot practice our
technology related to our product candidates as claimed in the patents or patent applications included in our patent rights. Competitors may also contest
patents or patent applications included in our patent rights by showing that the claimed subject matter was not patent-eligible, was not novel or was obvious
or that the patent claims failed any other requirement for patentability or enforceability. In addition, we may in the future be subject to claims by our or our
licensors’ current or former employees or consultants asserting an ownership right in the patents or patent applications included in our patent rights as an
inventor or co-inventor, as a result of the work they performed.
An adverse determination in any such submission or proceeding may result in loss of exclusivity or freedom to operate or in patent claims being
narrowed, invalidated or held unenforceable, in whole or in part, which could limit our ability to stop others from using or commercializing similar
therapeutics, without payment to us, or could limit the duration of the patent protection covering our product candidates. Such challenges may also result in
our inability to manufacture or commercialize our product candidates without infringing third-party patent rights, and we may be required to obtain a
license from third parties, which may not be available on commercially reasonable terms or at all, or we may need to cease the development, manufacture
and commercialization of one or more of our product candidates. In addition, if the breadth or strength of protection provided by the patents and patent
applications included in our patent rights is threatened, it could dissuade companies from collaborating with us to license, develop or commercialize current
or future product candidates. Such proceedings also may result in substantial cost and require significant time from our scientists and management, even if
the eventual outcome is favorable to us.
Even if they are unchallenged, the patents and pending patent applications included in our patent rights may not provide us with any meaningful
protection or prevent competitors from designing around our patent claims to circumvent our patent rights by developing similar or alternative therapeutics
in a non-infringing manner. For example, a third party may develop a competitive therapeutic that provides benefits similar to one or more of our product
candidates but that uses a vector or an expression construct that falls outside the scope of our patent protection. If the patent protection provided by the
patents and patent applications we license or pursue with respect to our product candidates is not sufficiently broad to impede such competition, our ability
to successfully commercialize our product candidates could be negatively affected.
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Our intellectual property licenses with third parties may be subject to disagreements over contract interpretation, which could narrow the scope of our
rights to the relevant intellectual property or technology or increase our financial or other obligations to our licensors.
We currently depend, and will continue to depend, on our license, collaboration and other similar agreements. Further development and
commercialization of SGT-001 and our other current and future product candidates may require us to enter into additional license, collaboration or other
similar agreements. The agreements under which we currently license intellectual property or technology from third parties are complex, and certain
provisions in such agreements may be susceptible to multiple interpretations. The resolution of any contract interpretation disagreement that may arise
could narrow what we believe to be the scope of our rights to the relevant intellectual property or technology, or increase what we believe to be our
financial or other obligations under the relevant agreement. Moreover, if disputes over intellectual property that we have licensed prevent or impair our
ability to maintain our current licensing arrangements on commercially acceptable terms, we may be unable to successfully develop and commercialize the
affected product candidates.
If any of our licenses or material relationships are terminated or breached, we may:
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lose our rights to develop and market SGT-001 or our other product candidates;
lose patent protection for SGT-001 or our other product candidates;
experience significant delays in the development or commercialization of SGT-001 or our other product candidates;
not be able to obtain any other licenses on acceptable terms, if at all; or
incur liability for damages.
These risks apply to any agreements that we may enter into in the future for SGT-001 and our other current and future product candidates.
If we fail to comply with our obligations in the agreements under which we license intellectual property rights from third parties or otherwise
experience disruptions to our business relationships with our licensors, we could lose license rights that are important to our business.
We have certain obligations under licensing agreements with third parties that include annual maintenance fees and payments that are contingent
upon achieving various development, commercial and regulatory milestones. Pursuant to many of these license agreements, we are required to make
milestone payments if certain development, regulatory and commercial sales milestones are achieved, and may have certain additional research funding
obligations. Also, pursuant to the terms of many of these license agreements, when and if commercial sales of a licensed product commence, we must pay
royalties to our licensors on net sales of the respective licensed products.
We have entered into license agreements with third parties and may need to obtain additional licenses from one or more of these same third parties
or from others to advance our research or allow our commercialization of SGT-001 or other product candidates. It is possible that we may be unable to
obtain additional licenses at a reasonable cost or on reasonable terms, if at all. In that event, we may be required to expend significant time and resources to
redesign SGT-001, our other product candidates or the methods for manufacturing them or to develop or license replacement products, all of which may not
be feasible on a technical or commercial basis. If we are unable to do so, we may be unable to develop or commercialize SGT-001 or our other product
candidates. We cannot provide any assurances that third-party patents or other intellectual property rights do not exist that might be enforced against our
manufacturing methods, product candidates or any technologies we may develop, resulting in either an injunction prohibiting our manufacture or sales, or,
with respect to our sales, an obligation on our part to pay royalties and/or other forms of compensation to third parties.
In each of our existing license agreements, and we expect in our future agreements, patent prosecution of our licensed technology is controlled
solely by the licensor, and we may be required to reimburse the licensor for their costs of patent prosecution. If our licensors fail to obtain and maintain
patent or other protection for the proprietary intellectual property we license from them, we could lose our rights to the intellectual property or our
exclusivity with respect to those rights, and our competitors could market competing products using the intellectual property. Further, in each of our license
agreements our licensors have the first right to bring any actions against any third party for infringing on the patents we have licensed. Our license
agreements also require us to meet development thresholds to maintain the license, including establishing a set timeline for developing and
commercializing product candidates. Disputes may arise regarding intellectual property subject to our licensing agreements, including:
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the scope of rights granted under the license agreement and other interpretation-related issues;
the extent to which our products or processes infringe on intellectual property of the licensor that is not subject to the licensing agreement;
the sublicensing of patent and other rights under our collaborative development relationships;
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our diligence obligations under the license agreement and what activities satisfy those diligence obligations;
the inventorship or ownership of inventions and know-how resulting from the joint creation or use of intellectual property by our licensors and
us and our partners; and
the priority of invention of licensed patented inventions.
If disputes over intellectual property that we have licensed prevent or impair our ability to maintain our current licensing arrangements on
acceptable terms, we may be unable to successfully develop and commercialize SGT-001 or our other product candidates. In spite of our best efforts, our
licensors might conclude that we have materially breached our license agreements and might therefore terminate the license agreements, thereby resulting
in disputes or litigation, which could cause us to incur substantial costs and distract management’s time, and if we are unsuccessful, we could lose our
ability to develop and commercialize products covered by these license agreements. If these licenses are ultimately terminated by the licensor, or if the
underlying patents fail to provide the intended exclusivity, competitors would have the freedom to seek regulatory approval of, and to market, products
identical to ours.
Third parties may initiate legal proceedings alleging that we are infringing, misappropriating or otherwise violating their intellectual property rights,
the outcome of which would be uncertain and could have a material adverse effect on the success of our business.
Our commercial success depends upon our ability and the ability of our future collaborators to develop, manufacture, market and sell SGT-001 and
our other current and future product candidates without infringing, misappropriating or otherwise violating the proprietary rights and intellectual property
of third parties. The biotechnology and pharmaceutical industries are characterized by extensive and complex litigation regarding patents and other
intellectual property rights. We or our licensors may in the future become party to, or be threatened with, adversarial proceedings or litigation regarding
intellectual property rights with respect to SGT-001 or our other product candidates, including interference proceedings, post grant review and inter partes
review before the USPTO. Our competitors or other third parties may assert infringement claims against us, alleging that, among other things, our
therapeutics, manufacturing methods, formulations or administration methods are covered by their patents.
Given the vast number of patents in our field of technology, we cannot be certain or guarantee that a court would hold that SGT-001 or any of our
other product candidates does not infringe an existing patent or a patent that may be granted in the future. Many companies and institutions have filed, and
continue to file, patent applications related to gene therapy and related manufacturing methods. Some of these patent applications have already been
allowed or issued and others may issue in the future. Since this area is competitive and of strong interest to pharmaceutical and biotechnology companies,
there will likely be additional patent applications filed and additional patents granted in the future, as well as additional research and development programs
expected in the future. Furthermore, because patent applications can take many years to issue, may be confidential for 18 months or more after filing and
can be revised before issuance, there may be applications now pending that may later result in issued patents that may be infringed by the manufacture, use,
sale or importation of our product candidates and we may or may not be aware of such patents. If a patent holder believes the manufacture, use, sale or
importation of one of our product candidates infringes its patent, the patent holder may sue us even if we have licensed other patent protection for our
product candidates. Moreover, we may face patent infringement claims from non-practicing entities that have no relevant product revenue and against
whom our licensed patent portfolio may therefore have no deterrent effect.
It is also possible that we have failed to identify relevant third-party patents or applications for which we may need a license to develop and
commercialize SGT-001 and our other product candidates. For example, applications filed before November 29, 2000 and certain applications filed after
that date that will not be filed outside the United States remain confidential until patents issue. Moreover, it is difficult for industry participants, including
us, to identify all third-party patent rights that may be relevant to our product candidates because patent searching is imperfect due to differences in
terminology among patents, incomplete databases and the difficulty in assessing the meaning of patent claims. We may fail to identify relevant patents or
patent applications or may identify pending patent applications of potential interest but incorrectly predict the likelihood that such patent applications may
issue with claims of relevance to our product candidates. In addition, we may be unaware of one or more issued patents that would be infringed by the
manufacture, sale or use of a current or future product candidate, or we may incorrectly conclude that a third-party patent is invalid, unenforceable or not
infringed by our activities. Additionally, pending patent applications that have been published can, subject to certain limitations, be later amended in a
manner that could cover our product candidates.
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Third parties may assert infringement claims against us based on existing patents or patents that may be granted in the future, regardless of their
merit. There is a risk that third parties may choose to engage in litigation with us to enforce or to otherwise assert their patent or other intellectual property
rights against us. For example, as discussed above, third parties may claim that the microdystrophin or the AAV vector we are developing for use in SGT-
001 is covered by patents held by them. Even if we believe such claim, or other intellectual property claims alleged by third parties, are without merit, there
is no assurance that we would be successful in defending such claims. A court of competent jurisdiction could hold that these third-party patents are valid,
enforceable and infringed, which could materially and adversely affect our ability to commercialize SGT-001 or our other product candidates covered by
the asserted third-party patents. In order to successfully challenge the validity of any such U.S. patent in federal court, we would need to overcome a
presumption of validity. As this burden is a high one requiring us to present clear and convincing evidence as to the invalidity of any such U.S. patent
claim, there is no assurance that a court of competent jurisdiction would invalidate the claims of any such U.S. patent. Similarly, there is no assurance that a
court of competent jurisdiction would find that SGT-001 or our other product candidates did not infringe a third-party patent.
Patent and other types of intellectual property litigation can involve complex factual and legal questions, and their outcome is uncertain. If we are
found, or believe there is a risk that we may be found, to infringe, misappropriate or otherwise violate a third party’s intellectual property rights, and we are
unsuccessful in demonstrating that such intellectual property rights are invalid or unenforceable, we could be required or may choose to obtain a license
from such third party to continue developing, manufacturing and marketing our product candidates. However, we may not be able to obtain any required
license on commercially reasonable terms or at all. Even if we were able to obtain a license, it could be non-exclusive, thereby giving our competitors and
other third parties access to the same technologies licensed to us, and it could require us to make substantial licensing and royalty payments. We could be
forced, including by court order, to cease developing, manufacturing and commercializing the infringing product candidate, including SGT-001. In
addition, we could be found liable for monetary damages, including treble damages and attorneys’ fees, if we are found to have willfully infringed a patent
or other intellectual property right. A finding of infringement, misappropriation or other violation of intellectual property rights, or claims that we have
done so, could prevent us from manufacturing and commercializing our product candidates or force us to cease some or all of our business operations.
Intellectual property litigation could cause us to spend substantial resources and distract our personnel from their normal responsibilities.
Litigation or other legal proceedings relating to intellectual property claims, with or without merit, is unpredictable and generally expensive and
time-consuming. Competitors may infringe patents that we may own in the future or the patents of our licensing partners or we may be required to defend
against claims of infringement. Even if resolved in our favor, litigation or other legal proceedings relating to intellectual property claims may cause us to
incur significant expenses and could distract our technical and management personnel from their normal responsibilities. Furthermore, because of the
substantial amount of discovery required in connection with intellectual property litigation, there is a risk that some of our confidential information could
be compromised by disclosure during this type of litigation. In addition, there could be public announcements of the results of hearings, motions or other
interim proceedings or developments and if securities analysts or investors perceive these results to be negative, it could have a substantial adverse effect
on the price of our common stock. Such litigation or proceedings could substantially increase our operating losses and reduce the resources available for
development activities or any future sales, marketing or distribution activities.
We may not have sufficient financial or other resources to adequately conduct such litigation or proceedings. Some of our competitors may be able
to sustain the costs of such litigation or proceedings more effectively than we can because of their greater financial resources and more mature and
developed intellectual property portfolios. Accordingly, despite our efforts, we may not be able to prevent third parties from infringing or misappropriating
or successfully challenging our intellectual property rights. Uncertainties resulting from the initiation and continuation of patent litigation or other
proceedings could have a material adverse effect on our ability to compete in the marketplace.
Obtaining and maintaining our patent protection depends on compliance with various procedural, document submission, fee payment and other
requirements imposed by government patent agencies, and our patent protection could be reduced or eliminated for non-compliance with these
requirements.
Periodic maintenance fees, renewal fees, annuity fees and various other government fees on patents and/or applications will be due to be paid to the
USPTO and various government patent agencies outside of the United States over the lifetime of our licensed patents and applications and any patents and
patent applications we may own in the future. The USPTO and various non-U.S. government patent agencies require compliance with several procedural,
documentary, fee payment and other similar provisions during the patent application process. We employ reputable intellectual property law firms and other
professionals to help us comply and we are also dependent on our licensors to take the necessary action to comply with these
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requirements with respect to our licensed intellectual property. In many cases, an inadvertent lapse can be cured by payment of a late fee or by other means
in accordance with the applicable rules. There are situations, however, in which non-compliance can result in abandonment or lapse of the patent or patent
application, resulting in partial or complete loss of patent rights in the relevant jurisdiction. In such an event, potential competitors might be able to enter
the market and this circumstance could have a material adverse effect on our business.
Some intellectual property that we have in-licensed may have been discovered through government-funded programs and thus may be subject to federal
regulations such as “march-in” rights, certain reporting requirements, and a preference for U.S. manufacturing. Compliance with such regulations
may limit our exclusive rights, and limit our ability to contract with non-U.S. manufacturers.
Some of the intellectual property rights we have licensed, including such rights licensed from the University of Michigan, the University of
Missouri and the University of Washington, are stated to have been generated through the use of U.S. government funding and may therefore be subject to
certain federal regulations. As a result, the U.S. government may have certain rights to intellectual property embodied in our current or future product
candidates pursuant to the Bayh-Dole Act of 1980, or Bayh-Dole Act. These U.S. government rights in certain inventions developed under a government-
funded program include a non-exclusive, non-transferable, irrevocable worldwide license to use inventions for any governmental purpose. In addition, the
U.S. government has the right to require us to grant exclusive, partially exclusive or non-exclusive licenses to any of these inventions to a third party if it
determines that: (i) adequate steps have not been taken to commercialize the invention, (ii) government action is necessary to meet public health or safety
needs or (iii) government action is necessary to meet requirements for public use under federal regulations (also referred to as “march-in rights”). The U.S.
government also has the right to take title to these inventions if we, or the applicable licensor, fail to disclose the invention to the government and fail to file
an application to register the intellectual property within specified time limits. Intellectual property generated under a government funded program is also
subject to certain reporting requirements, compliance with which may require us or the applicable licensor to expend substantial resources. In addition, the
U.S. government requires that any products embodying the subject invention or produced through the use of the subject invention be manufactured
substantially in the United States. The manufacturing preference requirement can be waived if the owner of the intellectual property can show that
reasonable but unsuccessful efforts have been made to grant licenses on similar terms to potential licensees that would be likely to manufacture
substantially in the United States or that under the circumstances domestic manufacture is not commercially feasible. This preference for U.S.
manufacturers may limit our ability to contract with non-U.S. product manufacturers for products covered by such intellectual property. To the extent any
of our current or future intellectual property is generated through the use of U.S. government funding, the provisions of the Bayh-Dole Act may similarly
apply.
We may not be able to protect our intellectual property and proprietary rights throughout the world.
Filing, prosecuting, maintaining, enforcing and defending patents on product candidates in all countries throughout the world would be
prohibitively expensive, and our intellectual property rights in some countries outside the United States could be less extensive than those in the United
States. Although our license agreements grant us worldwide rights, certain of our in-licensed U.S. patents lack corresponding foreign patents or patent
applications. For example, the issued U.S. patents we license from the University of Michigan do not have any corresponding foreign patents or patent
applications. Thus, we will not have the opportunity to obtain patent protection for the subject matter of such patents outside the United States. In addition,
the laws of some foreign countries do not protect intellectual property rights to the same extent as federal and state laws in the United States even in
jurisdictions where we and our licensors pursue patent protection. Consequently, we and our licensors may not be able to prevent third parties from
practicing our inventions in all countries outside the United States, even in jurisdictions where we and our licensors pursue patent protection, or from
selling or importing products made using our inventions in and into the United States or other jurisdictions. Competitors may use our inventions in
jurisdictions where we and our licensors have not pursued and obtained patent protection to develop their own products and may export otherwise
infringing products to territories where we have patent protection, but where enforcement is not as strong as it is in the United States. These products may
compete with our product candidates and our patents or other intellectual property rights may not be effective or sufficient to prevent them from competing.
Many companies have encountered significant problems in protecting and defending intellectual property rights in foreign jurisdictions. The legal
systems of certain countries, particularly certain developing countries, do not favor the enforcement of patents, trade secrets and other intellectual property
rights, particularly those relating to biotechnology products, which could make it difficult for us to stop the infringement of our patents, if pursued and
obtained, or the marketing of competing products in violation of our intellectual property and proprietary rights generally. Proceedings to enforce our
intellectual property and proprietary rights in foreign jurisdictions could (i) result in substantial costs and divert our efforts and attention from other aspects
of our business, (ii) put our patents at risk of being invalidated or interpreted narrowly and our patent applications at risk of not issuing and (iii) provoke
third parties to assert claims against us. We may not prevail in any lawsuits that we initiate and the damages or other remedies awarded, if any, may not be
commercially meaningful. Accordingly, our efforts to enforce our intellectual property rights around the world may be inadequate to obtain a significant
commercial advantage from the intellectual property that we develop or license.
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If we are unable to protect the confidentiality of our trade secrets, our business and competitive position would be harmed.
In addition to the protection afforded by patents, we rely on trade secret protection and confidentiality agreements to protect proprietary know-how
that is not patentable or that we elect not to patent, processes for which patents are difficult to enforce and any other elements of the discovery and
development processes of SGT-001 and our other product candidates that involve proprietary know-how, information or technology that is not covered by
patents. Our manufacturing process is protected by trade secrets. However, trade secrets can be difficult to protect and some courts inside and outside the
United States are less willing or unwilling to protect trade secrets.
We seek to protect our proprietary know-how, trade secrets and processes, in part, by entering into confidentiality agreements and, if applicable,
material transfer agreements, consulting agreements or other similar agreements with our employees, consultants, scientific advisors, CROs, manufacturers
and contractors. These agreements typically limit the rights of third parties to use or disclose our confidential information. However, we may not be able to
prevent the unauthorized disclosure or use of our technical know-how or other trade secrets by the parties to these agreements, despite the existence
generally of confidentiality agreements and other contractual restrictions. We cannot guarantee that we have entered into such agreements with each party
that may have or have had access to our trade secrets or proprietary processes. Monitoring unauthorized uses and disclosures is difficult and we do not
know whether the steps we have taken to protect our proprietary know-how and trade secrets will be effective. If any of our employees, collaborators,
CROs, manufacturers, consultants, advisors and other third parties who are parties to these agreements breaches or violates the terms of any of these
agreements, we may not have adequate remedies for any such breach or violation. Enforcing a claim that a party illegally disclosed or misappropriated a
trade secret is difficult, expensive, and time-consuming, and the outcome is unpredictable. As a result, we could lose our trade secrets. We also seek to
preserve the integrity and confidentiality of our data and trade secrets by maintaining physical security of our premises and physical and electronic security
of our information technology systems. While we have confidence in these security measures, they may still be breached, and we may not have adequate
remedies for any breach.
In addition, our trade secrets may otherwise become known or be independently discovered by competitors. Competitors could purchase our
product candidates, if approved, and attempt to replicate some or all of the competitive advantages we derive from our development efforts, willfully
infringe, misappropriate or otherwise violate our intellectual property rights, design around our protected know-how and trade secrets, or develop their own
competitive technologies that fall outside of our intellectual property rights. If any of our trade secrets were to be lawfully obtained or independently
developed by a competitor, we would have no right to prevent them, or those to whom they communicate such trade secrets, from using that technology or
information to compete with us. If our trade secrets are not adequately protected so as to protect our market against competitors’ products and technologies,
our competitive position could be adversely affected.
We may be subject to claims asserting that our employees, consultants or advisors have wrongfully used or disclosed alleged trade secrets of their
current or former employers or claims asserting ownership of what we regard as our own intellectual property.
Certain of our employees, consultants or advisors are currently, or were previously, employed at universities or other biotechnology or
pharmaceutical companies, including our competitors or potential competitors, as well as our academic partners. Although we try to ensure that our
employees, consultants and advisors do not use the proprietary information or know-how of others in their work for us, we may be subject to claims that
these individuals or we have used or disclosed intellectual property, including trade secrets or other proprietary information, of any such individual’s
current or former employer. Litigation may be necessary to defend against these claims. If we fail in defending any such claims, in addition to paying
monetary damages, we may lose valuable intellectual property rights or personnel. An inability to incorporate such technologies or features would have a
material adverse effect on our business and may prevent us from successfully commercializing our product candidates. Moreover, any such litigation or the
threat of such litigation may adversely affect our ability to hire employees or contract with independent contractors. A loss of key personnel or their work
product could hamper or prevent our ability to commercialize our product candidates. Even if we are successful in defending against such claims, litigation
could result in substantial costs and be a distraction to management.
In addition, while it is our policy to require our employees and contractors who may be involved in the conception or development of intellectual
property to execute agreements assigning such intellectual property to us, we may be unsuccessful in executing such an agreement with each party who, in
fact, conceives or develops intellectual property that we regard as our own. Moreover, even when we obtain agreements assigning intellectual property to
us, the assignment of intellectual property rights may not be self-executing or the assignment agreements may be breached, and we may be forced to bring
claims against third parties, or defend claims that they may bring against us, to determine the ownership of what we regard as our intellectual property.
Moreover, individuals executing agreements with us may have preexisting or competing obligations to a third party, such as an academic institution, and
thus an agreement with us may be ineffective in perfecting ownership of inventions developed by that individual.
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Changes in U.S. patent law could diminish the value of patents in general, thereby impairing our ability to protect our product candidates.
Changes in either the patent laws or the interpretation of the patent laws in the United States could increase the uncertainties and costs surrounding
the prosecution of patent applications and the enforcement or defense of issued patents. On September 16, 2011, the Leahy-Smith America Invents Act, or
the Leahy-Smith Act, was signed into law. The Leahy-Smith Act includes several significant changes to U.S. patent law. Prior to March 2013 in the United
States, assuming that other requirements for patentability are met, the first to make the claimed invention was entitled to the patent, while outside the
United States, the first to file a patent application was entitled to the patent. After March 2013, under the Leahy-Smith Act, the United States transitioned to
a first inventor to file system in which, assuming that other requirements for patentability are met, the first inventor to file a patent application will be
entitled to the patent on an invention regardless of whether a third party was the first to invent the invention. The Leahy-Smith Act also includes a number
of significant changes that affect the way patent applications will be prosecuted and may also affect patent litigation. These include allowing third-party
submission of prior art to the USPTO during patent prosecution and additional procedures to attack the validity of a patent through various post-grant
proceedings administered by the USPTO. The USPTO developed new regulations and procedures to govern administration of the Leahy-Smith Act, and
many of the substantive changes to patent law associated with the Leahy-Smith Act, and in particular, the first to file provisions, only became effective on
March 16, 2013. Accordingly, it is not clear what, if any, impact the Leahy-Smith Act will have on the operation of our business as, among other reasons,
the USPTO must still implement various regulations. However, the Leahy-Smith Act and its implementation could increase the uncertainties and costs
surrounding the prosecution of our patent applications and the enforcement or defense of our issued patents.
The patent positions of companies engaged in the development and commercialization of biologics and pharmaceuticals are particularly uncertain.
Two cases involving diagnostic method claims and “gene patents” have been decided by the Supreme Court of the United States, or the Supreme Court. On
March 20, 2012, the Supreme Court issued a decision in Mayo Collaborative Services v. Prometheus Laboratories, Inc., or Prometheus, a case involving
patent claims directed to a process of measuring a metabolic product in a patient to optimize a drug dosage for the patient. According to the Supreme Court,
the addition of well understood, routine or conventional activity such as “administering” or “determining” steps was not enough to transform an otherwise
patent-ineligible natural phenomenon into patent-eligible subject matter. On July 3, 2012, the USPTO issued a guidance memo to patent examiners
indicating that process claims directed to a law of nature, a natural phenomenon or a naturally occurring relation or correlation that do not include
additional elements or steps that integrate the natural principle into the claimed invention such that the natural principle is practically applied and the patent
claim amounts to significantly more than the natural principle itself should be rejected as directed to patent-ineligible subject matter. On June 13, 2013, the
Supreme Court issued its decision in Association for Molecular Pathology v. Myriad Genetics, Inc., or Myriad, a case involving patent claims held by
Myriad Genetics, Inc. relating to the breast cancer susceptibility genes BRCA1 and BRCA2. Myriad held that an isolated segment of naturally occurring
DNA, such as the DNA constituting the BRCA1 and BRCA2 genes, is not patent-eligible subject matter, but that complementary DNA may be patent-
eligible.
In 2014, the USPTO issued a guidance to its patent examiners for evaluating claims for patent subject matter eligibility under the relevant statute
(35 U.S.C. § 101). This guidance was in response to a series of decisions from the U.S. Supreme Court on patent claims reciting judicial exceptions,
including Abstract Ideas, Laws of Nature/Natural Principles, Natural Phenomena and/or Natural Products. Based on judicial decisions and public feedback,
several supplements to this guidance and additional memoranda and materials have since been issued and are continually being issued, while the current
eligibility guidance has been incorporated into the latest (9th) edition of the MPEP (Manual for Patent Examination Procedure), last revised in January
2018. The current subject matter eligibility guideline instructs USPTO examiners to follow a two-part test, set forth in the U.S. Supreme Court decisions
Alice/Mayo, as the only test that should be used to evaluate the eligibility of claims under examination, including claims directed to natural products and
principles including all naturally occurring nucleic acids. Certain claims of our licensed patents and patent applications contain, and any future patents we
may obtain may contain, claims that relate to specific recombinant DNA sequences that are naturally occurring at least in part and, therefore, could be the
subject of future challenges made by third parties. In addition, the current USPTO subject matter eligibility guidance and the constantly evolving case law,
together with contemplated congressional action, could all impact our ability to pursue similar patent claims in patent applications we may prosecute in the
future.
We cannot assure our stockholders that our efforts to seek patent protection for our product candidates will not be negatively impacted by the
decisions described above, rulings in other cases or changes in guidance or procedures issued by the USPTO. We cannot fully predict what impact the
Supreme Court’s decisions in Prometheus and Myriad may have on the ability of life science companies to obtain or enforce patents relating to their
products in the future. These decisions, the guidance issued by the USPTO and rulings in other cases or changes in USPTO guidance or procedures could
have a material adverse effect on our existing patent rights and our ability to protect and enforce our intellectual property in the future.
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Moreover, although the Supreme Court has held in Myriad that isolated segments of naturally occurring DNA are not patent-eligible subject matter,
certain third parties could allege that activities that we may undertake infringe other gene-related patent claims, and we may deem it necessary to defend
ourselves against these claims by asserting non-infringement and/or invalidity positions, or paying to obtain a license to these claims. In any of the
foregoing or in other situations involving third-party intellectual property rights, if we are unsuccessful in defending against claims of patent infringement,
we could be forced to pay damages or be subjected to an injunction that would prevent us from utilizing the patented subject matter.
If we do not obtain patent term extension for patents relating to SGT-001 or our other product candidates, our business may be materially harmed.
Depending upon the timing, duration and specifics of any FDA marketing approval of SGT-001 and our other product candidates, one or more U.S.
patents that we license or may own in the future may be eligible for limited patent term extension under the Drug Price Competition and Patent Term
Restoration Act of 1984, or the Hatch-Waxman Amendments. The Hatch-Waxman Amendments permit a patent extension term of up to five years as
compensation for patent term lost during the FDA regulatory review process based on the first regulatory approval for a particular drug or biologic. A
patent term extension cannot extend the remaining term of a patent beyond a total of 14 years from the date of product approval, only one patent may be
extended and only those claims covering the approved drug, a method for using it or a method for manufacturing it may be extended. However, we may not
be granted an extension because of, for example, failing to exercise due diligence during the testing phase or regulatory review process, failing to apply
within applicable deadlines, failing to apply prior to expiration of relevant patents or otherwise failing to satisfy applicable requirements. Moreover, the
applicable time period or the scope of patent protection afforded could be less than we request. In addition, to the extent we wish to pursue patent term
extension based on a patent that we in-license from a third party, we would need the cooperation of that third party. If we are unable to obtain patent term
extension or the term of any such extension is less than we request, our competitors may be able to enter the market sooner.
If our trademarks and trade names are not adequately protected, then we may not be able to build name recognition and our business may be adversely
affected.
We have registered trademarks with the USPTO for the marks “SOLID BIOSCIENCES”, “SOLID GT” and “SOLID”. Once registered, our
trademarks or trade names may be challenged, infringed, diluted, tarnished, circumvented or declared generic or determined to be infringing on other
marks. We may not be able to protect our rights to these trademarks and trade names, which we need to build name recognition among potential partners or
customers in our markets of interest. At times, competitors may adopt trade names or trademarks similar to ours, thereby impeding our ability to build
brand identity and possibly leading to market confusion. In addition, there could be potential trade name or trademark infringement, dilution or tarnishment
claims brought by owners of other registered trademarks or trademarks that incorporate variations of our registered or unregistered trademarks or trade
names. Over the long term, if we are unable to establish name recognition based on our trademarks and trade names, then we may not be able to compete
effectively and our business may be adversely affected. Our efforts to enforce or protect our proprietary rights related to trademarks, trade secrets, domain
names, copyrights or other intellectual property may be ineffective and could result in substantial costs and diversion of resources.
Intellectual property rights and regulatory exclusivity rights do not necessarily address all potential threats.
The degree of future protection afforded by our intellectual property rights is uncertain because intellectual property rights have limitations, and
may not adequately protect our business or permit us to maintain our competitive advantage. For example:
•
•
•
•
others may be able to make gene therapy products that are similar to our product candidates but that are not covered by the claims of the
patents that we license or may own in the future;
we, or our current or future license partners or collaborators, might not have been the first to make the inventions covered by the issued patent
or pending patent applications that we license or may own in the future;
we, or our current and future license partners or collaborators, might not have been the first to file patent applications covering certain of our
or their inventions;
others may independently develop similar or alternative products or duplicate any of our processes without infringing our owned or licensed
intellectual property rights;
67
•
•
•
•
•
•
•
•
others may circumvent our regulatory exclusivities, such as by pursuing approval of a competitive product candidate via the traditional
approval pathway based on their own clinical data, rather than relying on the abbreviated pathway provided for biosimilar applicants;
it is possible that our pending licensed patent applications or those that we may own in the future will not lead to issued patents;
issued patents that we hold rights to now or in the future may be held invalid or unenforceable, including as a result of legal challenges by our
competitors;
others may have access to the same intellectual property rights licensed to us;
our competitors might conduct research and development activities in countries where we do not have patent rights and then use the
information learned from such activities to develop competitive products for sale in our major commercial markets;
we may not develop additional proprietary technologies that are patentable;
the patents or other intellectual property rights of others may have an adverse effect on our business; and
we may choose not to file a patent for certain trade secrets or know how, and a third party may subsequently file a patent covering such
intellectual property.
Risks related to ownership of our common stock
Our executive officers, directors and principal stockholders maintain the ability to control all matters submitted to our stockholders for approval.
Our executive officers and directors and principal stockholders, in the aggregate, own shares representing approximately 55.9% of our capital stock
as of February 15, 2020. As a result, if these stockholders were to choose to act together, they would be able to control all matters submitted to our
stockholders for approval, as well as our management and affairs. For example, these persons, if they choose to act together, would control the election of
directors and approval of any merger, consolidation or sale of all or substantially all of our assets.
This concentration of voting power may:
•
•
•
delay, defer or prevent a change in control;
entrench our management and our board of directors; or
delay or prevent a merger, consolidation, takeover or other business combination involving us on terms that other stockholders may desire.
A significant number of our total outstanding shares may be sold into the market in the near future, which could cause the market price of our
common stock to drop significantly, even if our business is performing well.
Sales of a substantial number of shares of our common stock in the public market could occur at any time. These sales, or the perception in the
market that holders of a large number of shares intend to sell shares, could reduce the market price of our common stock. Our outstanding shares of
common stock may be freely sold in the public market at any time to the extent permitted by Rules 144 and 701 under the Securities Act of 1933, as
amended, or the Securities Act, or to the extent such shares have already been registered under the Securities Act and are held by non-affiliates of ours.
Moreover, as of February 15, 2020, holders of an aggregate of approximately 28.2 million shares of our common stock and approximately 2.3 million
shares of our common stock issuable upon the exercise of pre-funded warrants have rights, subject to certain conditions, to require us to file registration
statements covering their shares or to include their shares in registration statements that we may file for ourselves or other stockholders.
In July 2019, we completed a private placement of 10,607,525 shares of our common stock and 2,295,699 pre-funded warrants to purchase shares of
our common stock to several accredited investors. We have filed a registration statement covering the resale of these shares by the purchasers in the private
placement and have agreed to keep such registration statement effective until the date the shares covered by the registration statement have been sold or can be
resold without restriction under Rule 144 of the Securities Act.
68
In addition, on January 29, 2018, we filed a Registration Statement on Form S-8 to register approximately 5.0 million shares reserved for future
issuance under our 2018 Omnibus Incentive Plan will become eligible for sale in the public market in the future, subject to certain legal and contractual
limitations. These shares can be freely sold in the public market upon issuance, subject to black-out periods and volume limitations applicable to affiliates.
The price of our common stock has been, and in the future is likely to be, volatile and fluctuate substantially, which could result in substantial losses
for holders of our common stock.
Our stock price has been, and in the future is likely to be, volatile. The stock market in general and the market for biopharmaceutical or
pharmaceutical companies in particular, has experienced extreme volatility that has often been unrelated to the operating performance of particular
companies. As a result of this volatility, our stockholders may not be able to sell their shares of common stock at or above the price they paid for their
shares. The market price for our common stock may be influenced by many factors, including:
•
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•
•
•
•
•
•
•
•
•
•
•
•
•
results of or developments in preclinical studies and clinical trials of SGT-001 or our other product candidates or those of our competitors;
the success of competitive products or technologies;
regulatory or legal developments in the United States, the European Union and other countries;
the recruitment or departure of key personnel;
the level of expenses related to any of our product candidates, or our clinical development programs and our commercialization efforts;
the results of our efforts to discover, develop, acquire or in-license additional product candidates;
actual or anticipated changes in our development timelines;
our ability to raise additional capital;
our inability to obtain or delays in obtaining adequate product supply for any approved product or inability to do so at acceptable prices;
disputes or other developments relating to proprietary rights, including patents, litigation matters and our ability to obtain patent protection for
our product candidates;
significant lawsuits, including patent or stockholder litigation;
variations in our financial results or those of companies that are perceived to be similar to us;
changes in the structure of health care payment systems;
market conditions in the pharmaceutical and biotechnology sectors;
general economic, industry and market conditions; and
the other factors described in this “Risk Factors” section.
If our quarterly operating results fall below the expectations of investors or securities analysts, the price of our common stock could decline
substantially. Furthermore, any quarterly fluctuations in our operating results may, in turn, cause the price of our stock to fluctuate substantially.
In the past, following periods of volatility in the market price of a company’s securities, securities class-action litigation often has been instituted
against that company. We and certain of our executive officers and board members have been named as defendants in purported class action lawsuits. Any
such litigation instituted against us could cause us to incur substantial costs to defend such claims and divert management’s attention and resources.
69
An active trading market for our common stock may not be sustained.
Prior to our initial public offering, which occurred on January 26, 2018, there was no public market for our common stock. Although our common
stock is listed on the Nasdaq Global Select Market, given the limited trading history of our common stock, there is a risk that an active trading market for
our shares may not continue to develop or be sustained. If an active market for our common stock does not continue to develop or is not sustained, it may
be difficult for our stockholders to sell shares without depressing the market price for the shares, or at all.
We are an “emerging growth company,” and the reduced disclosure requirements applicable to emerging growth companies may make our common
stock less attractive to investors.
We are an “emerging growth company,” or EGC, as defined in the Jumpstart our Business Startups Act of 2012, or the JOBS Act. We will remain
an EGC until the earliest of: (i) the last day of the fiscal year in which we have total annual gross revenues of $1.07 billion or more; (ii) December 31,
2023; (iii) the date on which we have issued more than $1.0 billion in nonconvertible debt during the previous three years; and (iv) the date on which we
are deemed to be a large accelerated filer under the rules of the Securities and Exchange Commission, or the SEC. For so long as we remain an EGC, we
are permitted and intend to rely on exemptions from certain disclosure requirements that are applicable to other public companies that are not emerging
growth companies. These exemptions include:
•
•
•
•
•
not being required to comply with the auditor attestation requirements of Section 404 of the Sarbanes-Oxley Act, or Section 404;
not being required to comply with any requirement that may be adopted by the Public Company Accounting Oversight Board regarding
mandatory audit firm rotation or a supplement to the auditor’s report providing additional information about the audit and the financial
statements;
being permitted to provide only two years of audited financial statements, in addition to any required unaudited interim financial statements,
with correspondingly reduced “Management’s Discussion and Analysis of Financial Condition and Results of Operations” disclosure;
reduced disclosure obligations regarding executive compensation; and
an exemption from the requirement to seek nonbinding advisory votes on executive compensation or golden parachute arrangements.
We may choose to take advantage of some, but not all, of the available exemptions. We have taken advantage of reduced reporting burdens in our
other filings with the Securities and Exchange Commission. In particular, we have not included all of the executive compensation information that would
be required if we were not an EGC. We cannot predict whether investors will find our common stock less attractive if we rely on certain or all of these
exemptions. If some investors find our common stock less attractive as a result, there may be a less active trading market for our common stock and our
stock price may be more volatile.
We incur increased costs as a result of operating as a public company, and our management is now required to devote substantial time to new
compliance initiatives.
As a public company, and particularly after we are no longer an EGC, we incur significant legal, accounting and other expenses that we did not
incur as a private company. In addition, the Sarbanes-Oxley Act and rules subsequently implemented by the SEC and Nasdaq have imposed various
requirements on public companies, including establishment and maintenance of effective disclosure and financial controls and corporate governance
practices. Our management and other personnel devote a substantial amount of time to these compliance initiatives. Moreover, these rules and regulations
will increase our legal and financial compliance costs and will make some activities more time-consuming and costly. For example, we expect that these
rules and regulations may make it more difficult and more expensive for us to obtain director and officer liability insurance.
Pursuant to Section 404, we are required to furnish a report by our management on our internal control over financial reporting. However, while we
remain an EGC, we will not be required to include an attestation report on internal control over financial reporting issued by our independent registered
public accounting firm. To achieve compliance with Section 404 within the prescribed period, we will be engaged in a process to document and evaluate
our internal control over financial reporting, which is both costly and challenging. In this regard, we will need to continue to dedicate internal resources,
engage outside consultants and adopt a detailed work plan to assess and document the adequacy of internal control over financial reporting, continue steps
to improve control processes, validate through testing that controls are functioning as documented and implement a continuous reporting and improvement
process for internal control over financial reporting. Despite our efforts, there is a risk that neither we nor our independent registered public accounting firm
will be able to conclude within the prescribed timeframe that our internal control over financial reporting is effective as required by Section 404. This could
result in an adverse reaction in the financial markets due to a loss of confidence in the reliability of our financial statements.
70
Provisions in our certificate of incorporation and our bylaws and under Delaware law could make an acquisition of us, which may be beneficial to our
stockholders, more difficult and may prevent attempts by our stockholders to replace or remove our current management.
Provisions in our certificate of incorporation and our bylaws may discourage, delay or prevent a merger, acquisition or other change in control of us
that stockholders may consider favorable, including transactions in which our stockholders might otherwise receive a premium for their shares. These
provisions also could limit the price that investors might be willing to pay in the future for shares of our common stock, thereby depressing the market price
of our common stock. In addition, because our board of directors is responsible for appointing the members of our management team, these provisions may
frustrate or prevent any attempts by our stockholders to replace or remove our current management by making it more difficult for stockholders to replace
members of our board of directors. Among other things, these provisions:
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•
establish a classified board of directors such that not all members of our board are elected at one time;
allow the authorized number of our directors to be changed only by resolution of our board of directors;
limit the manner in which stockholders can remove directors from the board;
establish advance notice requirements for stockholder proposals that can be acted on at stockholder meetings and nominations to our board of
directors;
require that stockholder actions must be effected at a duly called stockholder meeting and prohibit actions by our stockholders by written
consent;
limit who may call stockholder meetings;
authorize our board of directors to issue preferred stock without stockholder approval, which could be used to institute a stockholder rights
plan, or so-called “poison pill,” that would work to dilute the stock ownership of a potential hostile acquirer, effectively preventing
acquisitions that have not been approved by our board of directors; and
require the approval of the holders of at least two-thirds of the votes that all our stockholders would be entitled to cast to amend or repeal
certain provisions of our certificate of incorporation or bylaws.
Moreover, because we are incorporated in Delaware, we are governed by the provisions of Section 203 of the Delaware General Corporation Law,
or the DGCL, which prohibits a person who owns in excess of 15% of our outstanding voting stock from merging or combining with us for a period of
three years after the date of the transaction in which the person acquired in excess of 15% of our outstanding voting stock, unless the merger or
combination is approved in a prescribed manner.
Because we do not anticipate paying any cash dividends on our capital stock in the foreseeable future, capital appreciation, if any, is the only sole
source of gain for an investment in our common stock.
We have never declared or paid cash dividends on our capital stock. We currently intend to retain all of our future earnings, if any, to finance the
growth and development of our business. In addition, the terms of any future debt agreements may preclude us from paying dividends. As a result, capital
appreciation, if any, of our common stock will be the sole source of gain for an investor for the foreseeable future.
Our certificate of incorporation provides that the Court of Chancery of the State of Delaware is the exclusive forum for certain litigation that may be
initiated by our stockholders, which could limit our stockholders’ ability to obtain a favorable judicial forum for such disputes with us or our directors,
officers or employees.
Our certificate of incorporation provides that, unless we consent in writing to the selection of an alternative forum, the Court of Chancery of the
State of Delaware is the exclusive forum for (i) any derivative action or proceeding brought on our behalf, (ii) any action asserting a claim for breach of a
fiduciary duty owed by any of our directors, officers or other employees to us or our stockholders, (iii) any action asserting a claim arising pursuant to any
provision of the DGCL, our certificate of incorporation or our bylaws or (iv) any action asserting a claim governed by the internal affairs doctrine. We do
not intend to have this choice of forum provision apply to, and this choice of forum provision will not apply to, actions arising under the Securities Act or
the Exchange Act. The choice of forum provision may limit a stockholder’s ability to bring a claim in a judicial forum that it finds favorable for disputes
with us or our directors, officers or other employees, which may discourage such lawsuits against us and our directors, officers and other employees.
Alternatively, if a court were to find the choice of forum provision contained in our certificate of incorporation to be inapplicable or unenforceable in an
action, we may incur additional costs associated with resolving such action in other jurisdictions.
71
Item 1B.
Unresolved Staff Comments.
None.
Item 2.
Properties.
We lease our corporate headquarters, which consists of approximately 16,000 square feet in Cambridge, Massachusetts. The lease for our corporate
headquarters has an initial term of approximately 4 years that expires in February 2022. In addition, we lease our primary laboratory space, which consists
of 9,500 square feet in Cambridge, Massachusetts, under a lease with an initial term of five years that expires in April 2023 and includes an option to
extend for one additional two-year term. In addition, we lease smaller laboratory and office space.
Item 3.
Legal Proceedings.
None.
Item 4.
Mine Safety Disclosures.
Not applicable.
72
PART II
Item 5.
Market for Registrant’s Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities.
Market Information
Our common stock has been publicly traded on the Nasdaq Global Select Market under the symbol “SLDB” since January 26, 2018 in connection
with our initial public offering. Prior to that date, there was no established public trading market for our common stock.
The following graph compares the performance of our common stock to The Nasdaq Composite Index and to The Nasdaq Biotechnology Index
from January 26, 2018 (the first date that shares of our common stock were publicly traded) through December 31, 2019. The comparison assumes $100
was invested after the market closed on January 26, 2018 in our common stock and in each of the foregoing indices, and it assumes reinvestment of
dividends, if any. The stock price performance included in this graph is not necessarily indicative of future stock price performance.
COMPARISON OF CUMULATIVE TOTAL RETURN
Among The Nasdaq Composite Index, The Nasdaq Biotechnology Index and Solid Biosciences Inc.
Holders
As of February 15, 2020, we had approximately 71 holders of record of our common stock. This number does not include beneficial owners whose
shares were held in street name. The actual number of holders of our common stock is greater than this number of record holders and includes stockholders
who are beneficial owners, but whose shares are held in street name by brokers or held by other nominees. This number of holders of record also does not
include stockholders whose shares may be held in trust by other entities.
Information about our Equity Compensation Plans
Information about our equity compensation plans is incorporated herein by reference to the section entitled “Securities authorized for issuance
under equity compensation plans” in Item 12 of Part III of this Annual Report on Form 10-K.
Purchase of Equity Securities
We did not purchase any of our registered equity securities during the period covered by this Annual Report on Form 10-K.
73
Recent Sales of Unregistered Securities
We did not sell any securities, during the year ended December 31, 2019 that were not registered under the Securities Act of 1933, as amended, or
the Securities Act, and that have not otherwise been described in a Quarterly Report on Form 10-Q or in a Current Report on Form 8-K.
Use of Proceeds from Initial Public Offering
On January 30, 2018, we closed our initial public offering, in which we issued and sold 8,984,375 shares of common stock, including 1,171,875
shares of our common stock issued upon the exercise in full of the underwriters’ over-allotment option, at a public offering price of $16.00 per share. The
aggregate gross proceeds to us from our initial public offering were approximately $143.8 million. All of the shares of common stock issued and sold in our
initial public offering were registered under the Securities Act pursuant to a registration statement on Form S-1 (Registration No. 333-222357), which was
declared effective by the SEC on January 25, 2018 and a registration statement on Form S-1 MEF (Registration No. 333-222705) filed pursuant to Rule
462(b) of the Securities Act. J.P. Morgan Securities LLC, Goldman Sachs & Co. LLC and Leerink Partners LLC were joint book-running managers for the
initial public offering. The offering commenced on January 25, 2018 and did not terminate until the sale of all of the shares offered. The aggregate net
proceeds to us were approximately $129.1 million, after deducting underwriting discounts and commissions and offering expenses payable by us of
approximately $14.7 million.
Except as set forth below, no offering expenses were paid directly or indirectly to any of our directors or officers (or their associates) or persons
owning ten percent or more of any class of our equity securities or to any other affiliates, JPMC Strategic Investments II Corporation, or JPMC, owned in
excess of 10% of our issued and outstanding common stock immediately prior to our initial public offering, and JPMC is an affiliate of J.P. Morgan
Securities LLC, which was a book running manager in our initial public offering. In addition, Mr. Robert Huffines, one of our directors, is an employee of
J.P. Morgan Securities LLC.
There has been no material change in our planned use of the net proceeds from the offering as described in our final prospectus filed with the SEC
on January 29, 2018 pursuant to Rule 424(b) under the Securities Act. We have been using and plan to continue to use the net offering proceeds to fund
research, development and clinical trial expenses, and the remainder for general and administrative expenses and other general corporate purposes.
Item 6.
Selected Financial Data.
The statement of operations data for the years ended December 31, 2019, 2018 and 2017 and the selected balance sheet data as of December 31,
2019 and 2018 are derived from our audited consolidated financial statements included elsewhere in this Annual Report on Form 10-K. The statement of
operations data for the years ended December 31, 2016 and 2015 and the selected balance sheet data as of December 31, 2017, 2016 and 2015 is derived
from our audited consolidated financial statements that are not included in this Annual Report on Form 10-K.
74
Our historical results are not necessarily indicative of results that should be expected in any future period. The following selected financial data
should be read in conjunction with the section of this report entitled “Management’s Discussion and Analysis of Financial Condition and Results of
Operations” and our financial statements and the related notes included elsewhere in this Annual Report on Form 10-K.
(in thousands, except shares and per share data)
Consolidated statements of operations data:
Revenue
Operating expenses:
Research and development
General and administrative
Total operating expenses
Loss from operations
Other income (expense):
Revaluation of preferred unit tranche rights
Interest and Other income
Total other income (expense), net
Net loss
Net loss attributable to Solid Biosciences Inc.
Net loss attributable to common stockholders
Net loss per share attributable to common stockholders,
basic and diluted (1)
Weighted average common stock outstanding, basic
and diluted (1)
(in thousands)
Consolidated balance sheet data:
Cash, cash equivalents and available-for-sale securities
Working capital (2)
Total assets
Finance lease obligations, net of current portion
Redeemable preferred units
Accumulated deficit
Total equity/(deficit)
For the Year Ended December 31,
2019
2018
2017
2016
2015
$
- $
- $
- $
- $
-
94,737
24,581
119,318
(119,318)
-
2,095
2,095
(117,223) $
57,965
17,722
75,687
(75,687)
-
889
889
(74,798) $
39,905
14,952
54,857
(54,857)
459
1,220
1,679
(53,178) $
20,116
5,460
25,576
(25,576)
1,163
640
1,803
(23,773) $
(117,223) $
(74,798) $
(52,118) $
(21,539) $
(117,223) $
(74,798) $
(39,317) $
(17,230) $
4,192
2,372
6,564
(6,564)
(103)
3
(100)
(6,664)
(6,377)
(6,445)
(2.91) $
(2.25) $
(2.88) $
(10.14) $
(7.61)
$
$
$
$
40,289,290 33,262,597 13,649,485
1,698,904
846,569
2019
2018
2017
2016
2015
As of December 31,
$
83,524 $
68,026
103,471
733
-
(316,279)
80,048
122,464 $
116,158
139,597
859
-
(199,056)
125,183
69,094 $
59,387
76,193
-
124,179
(124,258)
(59,257)
37,658 $
33,099
40,636
-
71,649
(84,941)
(37,886)
55,387
41,772
55,696
-
61,697
(67,711)
(19,925)
(1)
See Note 16 to our consolidated financial statements in this Annual Report on Form 10-K for details on the calculation of basic and diluted net loss
per share attributable to common stockholders.
(2) We define working capital as current assets less current liabilities.
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Item 7.
Management’s Discussion and Analysis of Financial Condition and Results of Operations.
The following discussion and analysis of our financial condition and results of operations should be read in conjunction with our financial
statements and related notes appearing elsewhere in this Annual Report on Form 10-K. Some of the information contained in this discussion and analysis
or set forth elsewhere in this Annual Report on Form 10-K, including information with respect to our plans and strategy for our business, includes forward-
looking statements that involve risks and uncertainties. As a result of many factors, including those factors set forth in the “Risk Factors” section of this
Annual Report on Form 10-K, our actual results could differ materially from the results described, in or implied, by these forward-looking statements.
Overview
Our mission is to cure Duchenne muscular dystrophy, or DMD, a genetic muscle-wasting disease predominantly affecting boys, with symptoms that
usually manifest between three and five years of age. DMD is a progressive, irreversible and ultimately fatal disease that affects approximately one in every
3,500 to 5,000 live male births and has an estimated prevalence of 10,000 to 15,000 cases in the United States alone. DMD is caused by mutations in the
dystrophin gene, which result in the absence or near-absence of dystrophin protein. Dystrophin protein works to strengthen muscle fibers and protect them
from daily wear and tear. Without functioning dystrophin and certain associated proteins, muscles suffer excessive damage from normal daily activities and
are unable to regenerate, leading to the build-up of fibrotic, or scar, and fat tissue. There is no cure for DMD and, for the vast majority of patients, there are
no satisfactory symptomatic or disease-modifying treatments. Our efforts are focused on our lead product candidate, SGT-001, a gene transfer candidate
under investigation for its ability to drive functional dystrophin protein expression in patients’ muscles and improve the course of the disease. Based on our
preclinical program that included multiple animal species of different phenotypes and genetic variations, as well as preliminary clinical trial biomarker
results, we believe that SGT-001, has the potential to slow or even halt the progression of DMD, regardless of the type of genetic mutation or stage of the
disease.
Since our inception, we have devoted substantial resources to identifying and developing SGT-001 and our other product candidates, developing our
manufacturing processes, organizing and staffing our company and providing general and administrative support for these operations. We have incurred
significant losses every year since our inception. We do not have any products approved for sale. To date, we have not generated any revenue. Our ability to
eventually generate any product revenue sufficient to achieve profitability will depend on the successful development, approval and eventual
commercialization of SGT-001 and our other product candidates. If successfully developed and approved, we intend to commercialize SGT-001 in the
United States and European Union and may enter into licensing agreements or strategic collaborations in other markets. If we generate product sales or
enter into licensing agreements or strategic collaborations, we expect that any revenue we generate will fluctuate from quarter to quarter and year to year as
a result of the timing and amount of any product sales, license fees, milestone payments and other payments. If we fail to complete the development of
SGT-001 and our other product candidates in a timely manner or obtain regulatory approval of them, our ability to generate future revenue, and our results
of operations and financial position, would be materially adversely affected.
SGT-001 has been granted Rare Pediatric Disease Designation, or RPDD, and Fast Track Designation, in the United States and Orphan Drug
Designations in both the United States and European Union. The safety and efficacy of SGT-001 are currently being evaluated in a Phase I/II clinical trial
called IGNITE DMD, which is currently on clinical hold.
Due to our significant research and development expenditure, licensing and patent investment, and general administrative costs associated with our
operations, we have generated substantial operating losses in each period since our inception. Our net losses were $117.2 million, $74.8 million and
$53.2 million for the years ended December 31, 2019, 2018 and 2017, respectively. As of December 31, 2019, we had an accumulated deficit of $316.3
million. We expect to incur significant expenses and operating losses for the foreseeable future.
As we seek to develop and commercialize SGT-001 or any other product candidates, we anticipate that our expenses will increase significantly and
that we will need substantial additional funding to support our continuing operations. Until such time as we can generate significant revenue from product
sales, if ever, we expect to finance our operations through a combination of public or private equity financings, debt financings or other sources, which may
include licensing agreements or strategic collaborations. We may be unable to raise additional funds or enter into such agreements or arrangements when
needed on favorable terms, if at all. If we fail to raise capital or enter into such agreements as and when needed, we may have to significantly delay, scale
back or discontinue the development or commercialization of SGT-001 or our other product candidates.
76
Because of the numerous risks and uncertainties associated with product development, we are unable to predict the timing or amount of increased
expenses or determine when or if we will be able to achieve or maintain profitability. Even if we are able to generate revenue from product sales, we may
not become profitable. If we fail to become profitable or are unable to sustain profitability on a continuing basis, then we may be unable to continue our
operations at planned levels and be forced to reduce or terminate our operations.
On January 30, 2018, we completed our initial public offering in which we sold 8,984,375 shares of our common stock, including shares of our
common stock issued upon the exercise in full of the underwriters’ over-allotment option, at a public offering price of $16.00 per share, resulting in net
proceeds of $129.1 million, after deducting underwriting discounts and commissions and offering expenses.
In July 2019, we completed a private placement of shares of our common stock and pre-funded warrants to purchase shares of our common stock
resulting in net proceeds to us of $57.9 million, after deducting offering costs.
As of December 31, 2019, we had cash, cash equivalents and available-for-sale securities of $83.5 million. We believe that our cash, cash
equivalents and available-for-sale securities as of December 31, 2019 will enable us to fund our operating expenses and capital expenditure requirements
into 2021. We have based this estimate on assumptions that may prove to be wrong, and we could use our available capital resources sooner than we
currently anticipate.
In January 2020, we announced a reduction in workforce by approximately one third as part of a strategic plan designed to create a leaner company
focused on advancing SGT-001.
Corporate conversion
We operated as a Delaware limited liability company under the name Solid Biosciences, LLC until immediately prior to the effectiveness of our
registration statement on Form S-1 on January 25, 2018, at which time we converted into a Delaware corporation pursuant to a statutory conversion and
changed our name to Solid Biosciences Inc. In addition, immediately following the statutory conversion, entities formed solely for the purpose of holding
membership interests in our limited liability company were merged with and into us. We refer to the corporate conversion and the mergers, collectively as
the Corporate Conversion. As a result of the Corporate Conversion, the holders of the Series 1 and 2 Senior Preferred and Junior Preferred Units and Series
A, B, C and D Common Units of Solid Biosciences, LLC became holders of common stock of Solid Biosciences Inc.
The consolidated financial statements included elsewhere in this Annual Report on Form 10-K are those of Solid Biosciences Inc. and its
subsidiaries.
Merger and recapitalization
We historically owned 100% of the voting units of our wholly owned subsidiary, Solid GT, LLC, or Solid GT, and the results of Solid GT are
included in our consolidated financial statements. Solid GT was organized in Delaware in August 2014 and was engaged in the business of developing
disease-modifying interventions for DMD through gene therapy. In November 2015, Solid GT issued voting units to new investors, which decreased our
voting ownership in Solid GT to 77%. We consolidated the results of Solid GT as we owned a majority voting interest in Solid GT and we directed the
activities of Solid GT.
Net loss attributable to non-controlling interests in our consolidated statement of operations and comprehensive loss consists of the portion of the
net income or loss of Solid GT that is not allocated to us. Changes in the amount of net loss attributable to non-controlling interests are directly impacted
by changes in the net income or loss of Solid GT. On March 29, 2017, we merged the operations of Solid GT into the company and Solid GT ceased to
exist as a separate legal entity. As a result, for periods subsequent to March 29, 2017, we no longer report any non-controlling interests related to Solid GT.
Financial operations overview
Revenue
We have not generated any revenue to date and do not expect to generate any revenue from the sale of our products for the next few years, if ever. If
our development efforts for SGT-001 or our other product candidates are successful and result in marketing approval or if we enter into collaboration or
license agreements with third parties, we may generate revenue in the future from a combination of product sales or payments from those collaboration or
license agreements.
77
Operating expenses
We classify our operating expenses into two categories: research and development, and general and administrative expenses. Personnel costs,
including salaries, benefits, bonuses and equity-based compensation expense, comprise a significant component of each of these expense categories. We
allocate expenses associated with personnel costs based on the nature of work associated with these resources.
Research and development expenses
Research and development expenses consist primarily of costs incurred for our research activities, including our discovery efforts, and the
development of SGT-001 and our other product candidates and include:
•
•
•
•
•
•
•
expenses incurred under agreements with third parties, including CROs, that conduct research and preclinical activities on our behalf, as well
as CMOs, that manufacture SGT-001 and our other product candidates for use in our preclinical studies and clinical trials;
salaries, benefits and other related costs, including equity-based compensation expense, for personnel engaged in research and development
functions;
costs of outside consultants, engaged to assist in our research and development activities, including their fees, equity-based compensation and
related travel expenses;
costs of laboratory supplies and acquiring, developing and manufacturing preclinical study and clinical trial materials;
costs incurred in seeking regulatory approval of SGT-001 and our other product candidates;
expenses incurred under our intellectual property licenses; and
facility-related research and development expenses, which include direct depreciation costs and allocated expenses for rent and maintenance
of facilities and other operating costs.
We expense research and development expenses as incurred. We recognize costs for certain development activities, such as preclinical research and
development and clinical trial costs, based on an evaluation of the progress to completion of specific tasks using information and data provided to us by our
vendors, collaborators and third-party service providers. Payments for these activities are based on the terms of the individual agreements, which may differ
from the pattern of costs incurred, and are reflected in our consolidated financial statements as prepaid or accrued research and development expenses.
We typically use our employee and infrastructure resources across our product candidates. We track outsourced development costs and milestone
payments made under our licensing arrangements by product candidates, but we do not allocate personnel costs, license payments made under our licensing
arrangements or other internal costs to product candidates on a program-specific basis. These costs are included in unallocated research and development
expenses in the table below.
The following table summarizes our research and development expenses by product candidates for the respective periods:
SGT-001
Other product candidates
Unallocated research and development expenses
Personnel related expenses
External expenses
Total unallocated research and development expenses
Total research and development expenses
For the Year Ended December 31,
2018
2017
2019
$
45,281 $
4,941
29,033 $
3,506
29,372
15,143
44,515
94,737 $
18,511
6,915
25,426
57,965 $
$
24,674
1,619
7,931
5,681
13,612
39,905
78
We cannot determine with certainty the duration, costs and timing of clinical trials of SGT-001 and our other product candidates or if, when or to
what extent we will generate revenue from the commercialization and sale of any of our product candidates for which we obtain marketing approval or our
other research and development expenses. We may never succeed in obtaining marketing approval for any of our product candidates. The duration, costs
and timing of clinical trials and development of our product candidates will depend on a variety of factors, including:
•
•
•
•
•
•
•
the scope, rate of progress, expense and results of any clinical trials of SGT-001 or other product candidates and other research and
development activities that we may conduct;
the imposition of regulatory restrictions on clinical trials, including full and partial clinical holds and the time and activities required to lift any
such holds;
uncertainties in clinical trial design and patient enrollment or drop out or discontinuation rates;
significant and changing government regulation and regulatory guidance;
potential additional studies or clinical trials requested by regulatory agencies;
the timing and receipt of any marketing approvals; and
the expense of filing, prosecuting, defending and enforcing any patent claims and other intellectual property rights.
Research and development activities are central to our business model. Product candidates in later stages of clinical development generally have
higher development costs than those in earlier stages of clinical development, primarily due to the increased size and duration of later-stage clinical trials.
We expect that our research and development expenses will decrease in calendar year 2020, as a result of the organizational changes announced in January
2020, to create a leaner organization focused on advancing SGT-001.
General and administrative expenses
General and administrative expenses consist primarily of salaries and other related costs, including equity-based compensation, for personnel in our
executive, finance, business development and administrative functions. General and administrative expenses also include legal fees relating to patent and
corporate matters, professional fees for accounting, auditing, tax and consulting services, insurance costs, travel expenses, and facility-related expenses,
which include direct depreciation costs and allocated expenses for rent and maintenance of office facilities and other operating costs.
We expect that our general and administrative expenses will increase in the future as we expect to continue to incur increased expenses associated
with being a public company, including costs of accounting, audit, legal, regulatory and tax-related services associated with maintaining compliance with
exchange listing and SEC requirements, director and officer insurance costs and investor and public relations costs.
Other income (expense)
Revaluation of preferred unit tranche right
The terms of the Series 1 Senior Preferred Unit Purchase Agreement, as amended on September 1, 2017, contained a right, which we refer to as the
Series 1 Tranche Right. The Series 1 Tranche Right obligated the holders of the Series 1 Senior Preferred Units to purchase 1,973,430 Series 2 Senior
Preferred Units at a purchase price of $12.67 per unit in the event we achieved certain preclinical milestones. In addition, the holders of a majority of the
Series 1 Senior Preferred Units had the right to require the holders of the Series 1 Senior Preferred Units to purchase the Series 2 Senior Preferred Units at
any time prior to December 1, 2017. The Series 1 Tranche Right was subject to certain transfer rights.
We concluded that the Series 1 Tranche Right met the definition of a freestanding financial instrument as the Series 1Tranche Right was legally
detachable and separately exercisable from the Series 1 Senior Preferred Units. Therefore, we allocated the net proceeds between the Series 1 Tranche
Right and the Series 1 Senior Preferred Units. The Series 1 Tranche Right was initially recorded at fair value and was re-measured at fair value each
reporting period. Changes in the fair market value were recognized as a component of other income (expense), net, in the consolidated statements of
operations.
In October 2017, the Series 1 Tranche Right was settled in connection with the closing of the Series 2 Senior Preferred Financing.
79
Interest income
Interest income consists of interest income earned on our cash, cash equivalents and available-for-sale securities.
Other income
We have received funding from charitable organizations, which is not considered to be an ongoing major or central part of our business. The
amounts received are recorded as other income as services are performed and research expenses are incurred in the consolidated statements of operations.
Income taxes
From our inception in 2013 until January 25, 2018, we were organized as a Delaware limited liability company for federal and state income tax
purposes and treated as a partnership for U.S. income tax purposes. As such, we were not viewed as a taxpaying entity in any jurisdiction and did not
require a provision for income taxes. Each member of our company was responsible for the tax liability, if any, related to its proportionate share of our
taxable income.
As a result of the Corporate Conversion, we are now treated as a corporation for U.S. income tax purposes and thus are subject to U.S. federal, state
and local income taxes and are taxed at the prevailing corporate tax rates. Among other things, we expect to generate net operating losses at the corporate
level. We account for income taxes using an asset and liability approach, which requires recognition of deferred tax assets and liabilities for the expected
future tax consequences of events that have been recognized in the consolidated financial statements, but have not been reflected in taxable income. A
valuation allowance is established to reduce deferred tax assets to their estimated realizable value.
We account for uncertainty in income taxes recognized in the financial statements by applying a two-step process to determine the amount of tax
benefit to be recognized. First, the tax position must be evaluated to determine the likelihood that it will be sustained upon external examination by the
taxing authorities. If the tax position is deemed more-likely-than-not to be sustained, the tax position is then assessed to determine the amount of benefit to
recognize in the financial statements. The amount of the benefit that may be recognized is the largest amount that has a greater than 50% likelihood of
being realized upon ultimate settlement. The provision for income taxes includes the effects of any resulting tax reserves, or unrecognized tax benefits, that
are considered appropriate as well as the related net interest and penalties.
Critical accounting policies and use of estimates
Our management’s discussion and analysis of financial condition and results of operations is based on our consolidated financial statements, which
have been prepared in accordance with generally accepted accounting principles in the United States. The preparation of our consolidated financial
statements and related disclosures requires us to make estimates and assumptions that affect the reported amounts of assets and liabilities, costs and
expenses and the disclosure of contingent assets and liabilities in our consolidated financial statements. We base our estimates on historical experience,
known trends and events and various other factors that we believe are reasonable under the circumstances, the results of which form the basis for making
judgments about the carrying values of assets and liabilities that are not readily apparent from other sources. We evaluate our estimates and assumptions on
an ongoing basis. Our actual results may differ from these estimates.
While our significant accounting policies are described in more detail in the notes to our consolidated financial statements appearing at the end of
this Annual Report on Form 10-K, we believe that the following accounting policies are those most critical to the judgments and estimates used in the
preparation of our consolidated financial statements.
80
Accrued research and development expenses
As part of the process of preparing our consolidated financial statements, we are required to estimate our accrued research and development
expenses. This process involves reviewing open contracts and purchase orders, communicating with our personnel to identify services that have been
performed on our behalf and estimating the level of service performed and the associated costs incurred for the services when we have not yet been
invoiced or otherwise notified of the actual costs. The majority of our service providers invoice us in arrears for services performed, on a pre-determined
schedule or when contractual milestones are met; however, some require advanced payments. We make estimates of our accrued expenses as of each
balance sheet date in our consolidated financial statements based on facts and circumstances known to us at that time. Examples of estimated accrued
research and development expenses include fees paid to:
•
•
•
•
CROs in connection with performing research activities on our behalf and conducting clinical trials and preclinical studies on our behalf;
vendors in connection with preclinical development activities;
vendors related to product manufacturing and development and distribution of clinical and preclinical supplies; and
third parties under our intellectual property licenses.
We base our expenses related to preclinical studies and clinical trials on our estimates of the services received and efforts expended pursuant to
quotes and contracts with multiple CROs that conduct and manage preclinical studies and clinical trials on our behalf. The financial terms of these
agreements are subject to negotiation, vary from contract to contract and may result in uneven payment flows. There may be instances in which payments
made to our vendors will exceed the level of services provided and result in a prepayment of the expense. In accruing fees, we estimate the time period over
which services will be performed, and the level of effort to be expended in each period. If the actual timing of the performance of services or the level of
effort varies from our estimate, we adjust the accrual or amount of prepaid expense accordingly. Although we do not expect our estimates to be materially
different from amounts actually incurred, our understanding of the status and timing of services performed relative to the actual status and timing of
services performed may vary and may result in us reporting amounts that are too high or too low in any particular period. To date, we have not made any
material adjustments to our prior estimates of accrued research and development expenses.
Equity-based compensation
In connection with the completion of our initial public offering, we adopted the 2018 Omnibus Incentive Plan, which provides for the issuance of
share-based awards, including options to purchase common stock. The 2018 Omnibus Incentive Plan provides for the awarding of up to 5,001,000 shares
of common stock for equity awards.
We measure all stock options and other stock-based awards granted to employees, directors and non-employees based on the fair value on the date
of the grant and recognize compensation expense of those awards, over the requisite service period, which is generally the vesting period of the respective
award. Forfeitures are accounted for as they occur. We apply the straight-line method of expense recognition to all awards with only service-based vesting
conditions. We have not issued any awards with performance-based vesting conditions. For stock-based awards granted to non-employees, compensation
expense is recognized over the period during which services are rendered by such non-employees until completed.
The fair value of each stock option grant is estimated on the date of grant using the Black-Scholes option-pricing model. We historically have been
a private company and lack company-specific historical and implied volatility information. Therefore, we estimate our expected stock volatility based on
the historical volatility of a publicly traded set of peer companies and expect to continue to do so until such time as we have adequate historical data
regarding the volatility of our own traded stock price. For options with service-based vesting conditions, the expected term of our stock options has been
determined utilizing the “simplified” method for awards that qualify as “plain-vanilla” options. Through December 31, 2018, the expected term of stock
options granted to non-employees is equal to the contractual term of the option award and effective January 1, 2019, the “simplified” method is used. The
risk-free interest rate is determined by reference to the U.S. Treasury yield curve in effect at the time of grant of the award for time periods approximately
equal to the expected term of the award. Expected dividend yield is based on the fact that we have never paid cash dividends and do not expect to pay any
cash dividends in the foreseeable future, if ever.
81
Results of operations
Comparison of the years ended December 31, 2019 and 2018
The following table summarizes our results of operations for the years ended December 31, 2019 and 2018:
(in thousands)
Revenue
Operating expenses:
Research and development
General and administrative
Total operating expenses
Loss from operations
Other income (expense):
Interest income
Other income
Total other income (expense)
Net loss
Research and development expenses
(in thousands)
SGT-001
Other product candidates
Unallocated research and development expenses
Personnel related expenses
External expenses
For the Year Ended December 31,
2019
2018
Increase
(decrease)
$
- $
- $
-
94,737
24,581
119,318
(119,318)
1,580
515
2,095
(117,223) $
57,965
17,722
75,687
(75,687)
619
270
889
(74,798) $
36,772
6,859
43,631
(43,631)
961
245
1,206
(42,425)
$
For the Year Ended December 31,
2019
2018
Increase
(decrease)
$
45,281 $
4,941
29,033 $
3,506
29,372
15,143
44,515
94,737 $
18,511
6,915
25,426
57,965 $
16,248
1,435
10,861
8,228
19,089
36,772
Total unallocated research and development expenses
Total research and development expenses
$
Research and development expenses for the year ended December 31, 2019 were $94.7 million, compared to $58.0 million for the year ended
December 31, 2018. The increase of $36.8 million in research and development costs was due to total unallocated research and development costs of $19.1
million primarily due to personnel and facility related expenses, including costs incurred to operate our lab facility, a $16.2 million increase in costs related
to our lead product candidate SGT-001 driven by higher clinical development and manufacturing activities of $13.2 million and contract cancellation fees
of $3.0 million as well as $1.4 million increase in costs related to our other product candidates.
General and administrative expenses
General and administrative expenses were $24.6 million for the year ended December 31, 2019, compared to $17.7 million for the year ended
December 31, 2018. The increase of $6.9 million was driven by a $4.4 million increase in equity-based compensation as a result of grants issued during the
year ended December 31, 2019, and other personnel and facility related expenses of $1.6 million as well as an increase of $0.9 million for professional
services and other corporate expenses.
Interest income
Interest income was $1.6 million and $0.6 million for the years ended December 31, 2019 and 2018, respectively. The increase in interest income
was due to an increase in available-for-sale securities in our portfolio during the first half of 2019.
82
Other income
Other income for the year ended December 31, 2019 was $0.5 million compared to $0.3 million for the year ended December 31, 2018. The
increase of $0.2 million was due to an increase of income from charitable organizations. We do not expect these contributions to significantly increase in
future periods.
Comparison of the years ended December 31, 2018 and 2017
The following table summarizes our results of operations for the years ended December 31, 2018 and 2017:
(in thousands)
Revenue
Operating expenses:
Research and development
General and administrative
Total operating expenses
Loss from operations
Other income (expense):
Revaluation of preferred unit tranche right
Interest income
Other income
Total other income (expense)
Net loss
$
Research and development expenses
(in thousands)
SGT-001
Other product candidates
Unallocated research and development expenses
Personnel related expenses
External expenses
Total unallocated research and development expenses
Total research and development expenses
$
For the Year Ended December 31,
2018
2017
Increase
(decrease)
$
- $
- $
-
57,965
17,722
75,687
(75,687)
-
619
270
889
(74,798) $
39,905
14,952
54,857
(54,857)
459
219
1,001
1,679
(53,178) $
18,060
2,770
20,830
(20,830)
(459)
400
(731)
(790)
(21,620)
For the Year Ended December 31,
2018
2017
Increase
(decrease)
$
29,033 $
3,506
24,674 $
1,619
18,511
6,915
25,426
57,965 $
7,931
5,681
13,612
39,905 $
4,359
1,887
10,580
1,234
11,814
18,060
Research and development expenses for the year ended December 31, 2018 were $58.0 million, compared to $39.9 million for the year ended
December 31, 2017. The increase of $18.1 million in research and development costs was due to total unallocated research and development costs of $11.8
million primarily due to personnel and facility related expenses, including costs incurred to operate the new lab facility, a net $4.4 million increase in costs
related to our lead product candidate SGT-001 driven by higher clinical development and manufacturing activities of $6.7 million offset by a $2.3 million
reduction in preclinical costs, and a $1.9 million increase in costs related to our other product candidates.
General and administrative expenses
General and administrative expenses were $17.7 million for the year ended December 31, 2018, compared to $15.0 million for the year ended
December 31, 2017. The increase of $2.7 million was driven by personnel and facility related expenses of $3.1 million as well as higher professional
services and other corporate expenses of $1.9 million associated with being a public company, partially offset by a decrease in equity-based compensation
of $2.3 million. The reduction in equity-based compensation of $2.3 million was primarily due to a charge associated with the exchange of certain of our
vested common units in connection with the recapitalization of Solid Biosciences, LLC and our merger with Solid GT on March 29, 2017.
83
Revaluation of preferred unit tranche right
We issued the Series 1 Tranche Right on March 29, 2017 and it was settled in October 2017 in connection with the Series 2 senior preferred unit
financing. The revaluation of the Series 1 Tranche Right resulted in a gain of $0.5 million in the year ended December 31, 2017 due to the settlement of the
tranche right.
Interest income
Interest income was $0.6 million and $0.2 million for the years ended December 31, 2018 and 2017 respectively. The increase in interest income
was due to an increase in available-for-sale securities in our portfolio.
Other income
Other income for the year ended December 31, 2018 was $0.3 million compared to $1.0 million for the year ended December 31, 2017. The
decrease of $0.7 million was due a reduction of income from charitable organizations. We do not expect these contributions to significantly increase in
future periods.
Liquidity and capital resources
Sources of liquidity
To date, we have financed our operations primarily through private placements of preferred units and our initial public offering as well as a private
placement of shares of our common stock and pre-funded warrants to purchase shares of our common stock. Through December 31, 2019, we raised an
aggregate of $144.6 million of gross proceeds from our sales of preferred units prior to the completion of our initial public offering, an aggregate of $129.1
million of net proceeds from the sale of our common stock after deducting underwriting discounts and commission and offering expenses in our initial
public offering and an aggregate $57.9 million of net proceeds, after deducting offering costs, from our July 2019 private placement.
We completed our initial public offering on January 30, 2018, in which we sold 8,984,375 shares of common stock, including the underwriters’
over-allotment option, at a public offering price of $16.00 per share, resulting in net proceeds of $129.1 million.
On July 30, 2019, we issued and sold in a private placement (i) 10,607,525 shares of our common stock at a price per share of $4.65 and (ii)
2,295,699 pre-funded warrants to purchase shares of our common stock at a price per warrant of $4.64. Each pre-funded warrant is exercisable for one
share of common stock at an exercise price of $0.01 and the pre-funded warrants have no expiration date. We received $57.9 million of net proceeds from
the private placement after deducting offering costs.
As of December 31, 2019, we had cash, cash equivalents and available-for-sale securities of $83.5 million and had no debt outstanding.
Cash flows
The following table summarizes our sources and uses of cash for each of the periods presented:
(in thousands)
Cash used in operating activities
Cash provided by (used in) investing activities
Cash provided by financing activities
Net (decrease) increase in cash and cash equivalents
For the Year Ended December 31,
2018
2017
2019
$
$
(92,714) $
24,516
57,875
(10,323) $
(70,197) $
(26,762)
131,507
34,548 $
(43,224)
10,448
77,078
44,302
84
Operating activities
During the year ended December 31, 2019, operating activities used $92.7 million of cash, primarily resulting from our net loss of $117.2 million
offset by non-cash charges of $16.8 million due primarily to equity-based compensation of $14.2 million and depreciation expense of $2.8 million as well
as cash provided by changes in our operating assets and liabilities. Net cash provided by changes in our operating assets and liabilities consisted primarily
of a decrease in prepaid expenses and other assets of $4.5 million which was primarily due to a decrease in prepayments related to research and
development activities. Net cash provided by changes in our operating assets and liabilities also included an increase in accounts payable of $3.8 million
due to the timing of payments.
During the year ended December 31, 2018, operating activities used $70.2 million of cash, primarily resulting from our net loss of $74.8 million
and cash used in changes in our operating assets and liabilities of $2.8 million offset by non-cash charges of $7.4 million due primarily to equity-based
compensation of $6.0 million and depreciation expense of $1.6 million. Net cash used in changes in our operating assets and liabilities consisted primarily
of an increase in prepaid expenses and other assets of $4.9 million which was primarily due to an increase in prepayments related to research and
development activities. Net cash used in changes in our operating assets and liabilities also included a decrease in accounts payable of $1.6 million due to
the timing of payments. These changes were offset by an increase in accrued expenses and other liabilities of $2.9 million as well as proceeds received
from our landlord for tenant improvements related to the buildout of our lab space of $0.8 million.
During the year ended December 31, 2017, operating activities used $43.2 million of cash, primarily resulting from our net loss of $53.2 million
offset by net non-cash charges of $5.5 million due primarily to equity-based compensation of $5.3 million, which included $3.7 million associated with the
exchange of Series A common units into Series B and D common units, and cash provided by changes in our operating assets and liabilities of $4.4 million.
Net cash provided by changes in our operating assets and liabilities during the year ended December 31, 2017 consisted of a decrease in prepaid expenses
and other current assets of $0.8 million due to the timing of prepaid research and development expense payments and net increase in accounts payable and
accrued expenses of $3.6 million due to the timing of payments and the increase in the overall activity of the company.
Investing activities
During the year ended December 31, 2019, investing activities provided $24.5 million of cash, consisting primarily of net proceeds on the sale and
maturity of available-for-sale securities partially offset by purchases of property and equipment.
During the year ended December 31, 2018, investing activities used $26.8 million of cash, consisting primarily of net purchases of investments as
well as the acquisition of property and equipment.
During the year ended December 31, 2017, investing activities provided $10.4 million of cash, consisting primarily of the net proceeds on the sale
and maturity of available-for-sale securities partially offset by purchases of property and equipment.
Financing activities
During the year ended December 31, 2019, net cash provided by financing activities was $57.9 million, due to the net proceeds from our private
placement of shares of our common stock and pre-funded warrants to purchase shares of our common stock that we completed in July 2019.
During the year ended December 31, 2018, net cash provided by financing activities was $131.5 million, primarily due to the net proceeds from our
initial public offering.
During the year ended December 31, 2017, net cash provided by financing activities was $77.1 million, primarily due to the net proceeds from our
sale of Series 1 and 2 Senior Preferred Units of $24.5 million and $55.0 million, respectively, partially offset by payments made in connection with our
initial public offering.
85
Funding requirements
We expect our expenses to increase substantially in connection with our ongoing development activities related to SGT-001. In addition, we expect
to incur additional costs associated with operating as a public company. We expect that our expenses will increase substantially if and as we:
•
•
•
•
•
•
•
•
•
•
•
•
seek to resolve the clinical hold on IGNITE DMD and, if and when lifted, continue to enroll patients in IGNITE DMD and continue clinical
development of SGT-001;
move other product candidates into clinical trials;
continue research and preclinical development of our other product candidates;
seek to identify additional product candidates;
seek marketing approvals for our product candidates that successfully complete clinical trials, if any;
establish a sales, marketing and distribution infrastructure to commercialize any products for which we may obtain marketing approval;
arrange for manufacture of larger quantities of our product candidates for clinical development and potential commercialization;
maintain, expand, protect and enforce our intellectual property portfolio;
hire and retain additional clinical, quality control and scientific personnel;
build out new facilities or expand existing facilities to support our activities;
acquire or in-license other drugs, technologies and intellectual property; and
add operational, financial and management information systems and personnel.
On January 30, 2018, we completed our initial public offering in which we sold 8,984,375 shares of common stock, including shares of common
stock issued upon the exercise in full of the underwriters’ over-allotment option, at a public offering price of $16.00 per share, resulting in net proceeds of
$129.1 million, after deducting underwriting discounts and commissions and offering expenses.
On July 30, 2019, we issued and sold in a private placement (i) 10,607,525 shares of our common stock at a price per share of $4.65 and (ii)
2,295,699 pre-funded warrants to purchase shares of our common stock at a price per warrant of $4.64. Each pre-funded warrant is exercisable for one
share of common stock at an exercise price of $0.01 and the pre-funded warrants have no expiration date. We received $57.9 million of net proceeds from
the private placement after deducting offering costs.
As of December 31, 2019, we had cash, cash equivalents and available-for-sale securities of $83.5 million. We believe that our cash, cash
equivalents and available-for-sale securities as of December 31, 2019, will enable us to fund our operating expenses and capital expenditure requirements
into 2021. We have based this estimate on assumptions that may prove to be wrong, and we could use our available capital resources sooner than we
currently anticipate.
Because of the numerous risks and uncertainties associated with the development of SGT-001 and other product candidates and programs and
because the extent to which we may enter collaborations with third parties for development of our product candidates is unknown, we are unable to
estimate the timing and amounts of increased capital outlays and operating expenses associated with completing the research and development of our
product candidates. Our future capital requirements will depend on many factors, including:
•
•
•
•
•
if and when we are able to favorably resolve the clinical hold on and resume IGNITE DMD;
the progress and results of IGNITE DMD and future clinical trials of SGT-001 and our other product candidates;
the costs, timing and outcome of regulatory review of SGT-001 and our other product candidates;
the scope, progress, results and costs of discovery, laboratory testing, manufacturing, preclinical development and clinical trials for other
product candidates that we may pursue in the future, if any;
the costs associated with our manufacturing process development and evaluation of third-party manufacturers;
86
•
•
•
•
•
•
whether we decide to construct and validate our own manufacturing facility and the associated costs;
revenue, if any, received from commercial sale of SGT-001 or other product candidates, should any of our product candidates receive
marketing approval;
the costs of preparing, filing and prosecuting patent applications, maintaining, defending and enforcing our intellectual property rights and
defending intellectual property-related claims;
the outcome of any lawsuits filed against us;
the terms of our current and any future license agreements and collaborations; and
the extent to which we acquire or in-license other product candidates, technologies and intellectual property.
We intend to supply our clinical development program for SGT-001 with drug product produced at a cGMP compliant facility located at one of our
Contract Development Manufacturing Organization partners. We intend to establish the capability and capacity to supply SGT-001 at commercial scale
from multiple sources, including potentially building our own GMP facility to ensure redundancy and reliability. We expect that such a facility would
require capital expenditures of between $60.0 million to $75.0 million to commence operations. We expect to finalize plans to potentially build our own
GMP facility after we have additional data from IGNITE DMD.
Developing pharmaceutical products, including conducting preclinical studies and clinical trials, is a time-consuming, expensive and uncertain
process that takes years to complete, and we may never generate the necessary data or results required to obtain marketing approval for any product
candidates or generate revenue from the sale of any products for which we may obtain marketing approval. In addition, our product candidates, if approved,
may not achieve commercial success. Our commercial revenues, if any, will be derived from sales of products that we do not expect to be commercially
available for many years, if ever. Accordingly, we will need to obtain substantial additional funds to achieve our business objectives.
Adequate additional funds may not be available to us on acceptable terms, or at all. We do not currently have any committed external source of
funds. To the extent that we raise additional capital through the sale of equity securities, our existing stockholders’ ownership interest may be diluted. Any
debt or preferred equity financing, if available, may involve agreements that include restrictive covenants that may limit our ability to take specific actions,
such as incurring additional debt, making capital expenditures or declaring dividends, which could adversely impact our ability to conduct our business,
and may require the issuance of warrants, which could potentially dilute existing stockholders’ ownership interests.
If we raise additional funds through licensing agreements and strategic collaborations with third parties, we may have to relinquish valuable rights
to our technology, future revenue streams, research programs, or product candidates or grant licenses on terms that may not be favorable to us. If we are
unable to raise additional funds, we may be required to delay, limit, reduce and/or terminate development of our product candidates or any future
commercialization efforts or grant rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves.
Contractual obligations and commitments
The following table summarizes our contractual obligations at December 31, 2019 and the effects that such obligations are expected to have on our
liquidity and cash flows in future periods:
(in thousands)
Operating lease commitments (1)
Finance lease commitments (2)
Unconditional purchase commitments (3)
Total
$
Total
7,740 $
1,128
3,079
$
11,947 $
Less Than
1 Year
Payments due by period
1 - 3
Years
3 - 5
Years
More Than
5 Years
2,408 $
276
1,496
4,180 $
3,868 $
552
1,583
6,003 $
1,106 $
300
-
1,406 $
358
-
-
358
(1)
Represents minimum payments due for our leases of office and laboratory space in Cambridge, Massachusetts.
In January 2018, we executed a lease agreement for lab space in Cambridge, Massachusetts. The lease consists of approximately 9,500 square feet
with an initial term of five years with the option to extend the term for one additional two-year term.
87
In January 2018, we executed a lease agreement for office space in Cambridge, Massachusetts. The lease serves as our corporate headquarters and
consists of approximately 16,000 square feet. The term of the lease runs through February 2022.
In January 2019, we executed a lease agreement for additional office space in Cambridge, Massachusetts. The space serves as office space
supporting the Company’s lab operations and consists of approximately 5,000 square feet. The term of the lease runs through October 2025.
(2)
Represents minimum payments due for our lease of lab equipment which is in use in our lab facility in Cambridge, Massachusetts.
In November 2018, we executed a finance lease for lab equipment.
(3)
Unconditional purchase commitments represent minimum payments due to purchase goods that are enforceable and legally binding and that specify
all significant terms, including fixed or minimum quantities to be purchased, fixed or variable price provisions, and the approximate timing of the
transaction.
Under various agreements with third-party licensors, we have agreed to make milestone payments and pay royalties to third parties based on
specific milestones. We have not included any such contingent payment obligations in the table above as the amount, timing and likelihood of such
payments are not known. See “Business—Strategic partnerships and collaborations/licenses.”
We enter into contracts in the normal course of business with CROs and CMOs for clinical trials, preclinical research studies and testing,
manufacturing and other services and products for operating purposes. These contracts are cancelable by us upon prior notice of 30 days and, as a result,
are not included in the table of contractual obligations above.
Off-balance sheet arrangements
We did not have during the periods presented, and we do not currently have, any off-balance sheet arrangements, as defined in the rules and
regulations of the SEC.
Recently issued accounting pronouncements
We have reviewed all recently issued standards and have determined that, other than as disclosed in Note 2 to our consolidated financial statements
appearing at the end of this Annual Report on Form 10-K, such standards will not have a material impact on our consolidated financial statements or do not
otherwise apply to our operations.
Emerging growth company status
The JOBS Act permits an emerging growth company such as us to take advantage of an extended transition period to comply with new or revised
accounting standards applicable to public companies until those standards would otherwise apply to private companies. We have irrevocably elected to opt
out of this provision and, as a result, we will comply with new or revised accounting standards when they are required to be adopted by public companies
that are not emerging growth companies.
Item 7A.
Quantitative and Qualitative Disclosures About Market Risk.
Interest Rate Risk
We are exposed to market risk related to changes in interest rates. As of December 31, 2019, our available-for-sale securities consisted of corporate
bond securities and commercial paper that have contractual maturities of one year or less. Our primary exposure to market risk is interest income
sensitivity, which is affected by changes in the general level of U.S. interest rates. However, because of the short-term nature of our portfolio, an immediate
10% change in market interest rates would not have a material impact on the fair market value of our investment portfolio or on our financial position or
results of operations.
Inflation Risk
We do not believe that inflation had a material effect on our business, financial condition or results of operations in the last three years.
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Item 8.
Financial Statements and Supplementary Data.
The information required by this Item 8 is included at the end of this Annual Report on Form 10-K beginning on page F-1.
Item 9.
Changes in and Disagreements With Accountants on Accounting and Financial Disclosure.
Not applicable.
Item 9A.
Controls and Procedures.
Evaluation of Disclosure Controls and Procedures
Our management, with the participation of our Chief Executive Officer and Chief Financial Officer (our principal executive officer and principal
financial officer, respectively), evaluated the effectiveness of our disclosure controls and procedures as of December 31, 2019. The term "disclosure
controls and procedures," as defined in Rules 13a-15(e) and 15d-15(e) under the Securities Exchange Act of 1934, as amended, or the Exchange Act, refers
to controls and other procedures of a company that are designed to ensure that information required to be disclosed by a company in the reports that it files
or submits under the Exchange Act is recorded, processed, summarized and reported, within the time periods specified in the SEC's rules and forms.
Disclosure controls and procedures include, without limitation, controls and procedures designed to ensure that such information required to be disclosed
by a company in the reports that it files or submits under the Exchange Act is accumulated and communicated to a company's management, including its
principal executive and principal financial officers, or persons performing similar functions, as appropriate to allow timely decisions regarding required
disclosure.
In designing and evaluating our disclosure controls and procedures, management recognizes that disclosure controls and procedures, no matter how
well designed and operated, can provide only reasonable, not absolute, assurance that the objectives of the disclosure controls and procedures are met.
Additionally, in designing disclosure controls and procedures, our management necessarily was required to apply its judgment in evaluating the cost-benefit
relationship of possible disclosure controls and procedures. The design of any system of controls also is based in part upon certain assumptions about the
likelihood of future events, and there can be no assurance that any design will succeed in achieving its stated goals under all potential future conditions;
over time, controls may become inadequate because of changes in conditions, or the degree of compliance with policies or procedures may deteriorate.
Because of the inherent limitations in a control system, misstatements due to error or fraud may occur and not be detected.
Based on the evaluation of our disclosure controls and procedures, our Chief Executive Officer and Chief Financial Officer concluded that, as of
December 31, 2019, our disclosure controls and procedures were effective at the reasonable assurance level.
Management's Annual Report on Internal Control over Financial Reporting
Our management is responsible for establishing and maintaining adequate internal control over financial reporting as defined in Rules 13a-15(f) and
15d-15(f) under the Exchange Act. Our internal control over financial reporting is a process designed to provide reasonable assurance regarding the
reliability of financial reporting and the preparation of financial statements for external purposes in accordance with generally accepted accounting
principles. Because of its inherent limitations, internal control over financial reporting may not prevent or detect misstatements. Also, projections of any
evaluation of effectiveness to future periods are subject to the risk that controls may become inadequate because of changes in conditions, or that the degree
of compliance with the policies or procedures may deteriorate.
Management conducted an evaluation of the effectiveness of our internal control over financial reporting as of December 31, 2019 based on the
framework in Internal Control-Integrated Framework (2013) issued by the Committee of Sponsoring Organizations of the Treadway Commission (COSO).
Based on that evaluation, management has concluded that the Company's internal control over financial reporting was effective as of December 31, 2019.
This Annual Report on Form 10-K does not include an attestation report of our independent registered public accounting firm due to a transition
period established by rules of the SEC for “emerging growth companies”.
89
Remediation of Previously-Identified Material Weaknesses in Internal Control over Financial Reporting
We previously identified and disclosed in our Annual Report on Form 10-K for the period ended December 31, 2018, as well as in our Quarterly
Reports on Form 10-Q for each interim period in 2019, material weaknesses in our internal control over financial reporting relating to the following:
•
•
•
We did not design or maintain an effective control environment commensurate with our financial reporting requirements. We lacked a
sufficient number of professionals with an appropriate level of accounting knowledge, training and experience to appropriately analyze, record
and disclose accounting matters timely and accurately. Additionally, the limited personnel resulted in our inability to consistently establish
appropriate authorities and responsibilities in pursuit of our financial reporting objectives, as demonstrated by, among other things, our
insufficient segregation of duties in our finance and accounting functions. This material weakness contributed to the additional material
weaknesses detailed below.
We did not design and maintain formal accounting policies, procedures and controls to achieve complete, accurate and timely financial
accounting, reporting and disclosures, including controls over the preparation and review of account reconciliations and journal entries.
Additionally, we did not design and maintain controls over the appropriate cut-off, classification and presentation of accounts and disclosures
in the financial statements.
We did not design and maintain formal accounting policies, processes and controls to analyze, account for and disclose complex transactions.
Specifically, we did not design and maintain controls to analyze, account for and disclose complex transactions, including variable interest
entities, preferred units, the preferred unit tranche right and equity-based compensation.
Our management, with oversight from our audit committee, has implemented the following remediation steps to address the previously disclosed
material weaknesses and to improve our internal control over financial reporting:
•
•
•
•
hired additional full-time accounting resources and financial planning and analysis resources with appropriate levels of experience, including a
new Assistant Controller and Senior Accountant, and reallocated responsibilities across the accounting organization to ensure that the
appropriate level of knowledge and experience is applied based on risk and complexity of transactions and tasks under review;
strengthened our internal policies, processes and reviews, including formal documentation thereof;
implemented a financial close policy and monitoring program, including the formation of a disclosure committee comprised of members of
our senior management team, financial management and representatives from our accounting and legal departments to review and approve
SEC filings and investor communications, the results of which are discussed with the audit committee quarterly; and
engaged a professional accounting services firm to help us assess, document, design and implement control activities related to internal
control over financial reporting.
Management concluded that, as a result of the implementation of these actions, and the results of our testing over the design and operating
effectiveness of controls, our remediation efforts have been successful and that the previously-identified material weaknesses in our internal control over
financial reporting have been remediated as of December 31, 2019.
Changes in Internal Control over Financial Reporting
There have been no changes in our internal control over financial reporting (as defined in Rules 13a-15(f) and 15d-15(f) under the Exchange Act)
that occurred during the three months ended December 31, 2019 that have materially affected, or are reasonably likely to materially affect, our internal
control over financial reporting.
Item 9B.
Other Information.
Not applicable.
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Item 10.
Directors, Executive Officers and Corporate Governance.
Set forth below are the names, ages and positions of our current executive officers and directors as of February 15, 2020.
PART III
Name
Executive Officers
Ilan Ganot
Lynette Herscha
Carl Morris, Ph.D.
Joel Schneider, Ph.D.
Jennifer Ziolkowski
Non-Employee Directors
Andrey Zarur, Ph.D.
Matthew Arnold
Martin Freed, M.D., F.A.C.P.
Robert Huffines
Adam Koppel, M.D., Ph.D.
Sukumar Nagendran, M.D.
Rajeev Shah
Adam Stone
Lynne Sullivan
Executive officers
Age
Position(s) held
46
48
50
35
45
49
50
59
54
50
54
42
40
53
Co-founder, President, Chief Executive Officer and Director
Chief Legal Officer and Secretary
Chief Scientific Officer
Chief Technology Officer and Head of Exploratory Research and Development
Chief Financial Officer and Treasurer
Co-founder and Chairman of the Board of Directors
Director
Director
Director
Director
Director
Director
Director
Director
Ilan Ganot is one of our co-founders and has served as our Chief Executive Officer and as a member of our board of directors since our inception in
2013. Mr. Ganot has served as our President since June 2018. Previously, Mr. Ganot served as an investment banker at JPMorgan Chase & Co., a leading
global financial services firm, from September 2011 to September 2013. From October 2008 to August 2011, Mr. Ganot served as a banker at Nomura
Securities Co., Ltd., a securities and investment banking company, and from September 2003 to September 2008, at Lehman Brothers, a global financial
services firm. Mr. Ganot received his M.B.A. from London Business School and holds law and business degrees from the Interdisciplinary Center in
Herzliya, Israel. Mr. Ganot also practiced corporate law in Israel and was a Captain in the Israeli Defense Forces. He is qualified to serve on our board of
directors because of his personal dedication to improving treatments available for DMD patients and his extensive leadership experience.
Carl Morris, Ph.D. has served as our Chief Scientific Officer since June 2017, and previously served as our Senior Vice President of Research and
Development from September 2015 to June 2017. Prior to joining us, Dr. Morris held various leadership positions within Pfizer Inc.’s, or Pfizer’s, Rare
Disease Research Unit from January 2010 to August 2015, including serving as a Senior Director, Director and Senior Principal Scientist. Prior to Pfizer,
Dr. Morris held various positions within the Tissue Repair unit at Wyeth Pharmaceuticals, Inc., a pharmaceutical company acquired by Pfizer. Dr. Morris
was an Assistant Professor at Boston University School of Medicine and a founding faculty member of the Muscle and Aging Research Unit. He is also co-
founder and a member of the board of directors of Breed Nutrition Inc. Dr. Morris holds a B.A. in Biology from Franklin Pierce College and a Ph.D. in
Physiology from UCLA.
Lynette Herscha has served as our Chief Legal Officer and Secretary since January 2019. Prior to joining us, she served as General Counsel,
Secretary and a member of the Executive Team at Concert Pharmaceuticals, Inc., a public biopharmaceutical company, from 2017 to 2018 and its Vice
President and Assistant Secretary Legal from 2014 to 2017. Before joining Concert Pharmaceuticals, Ms. Herscha held various senior legal positions at
Momenta Pharmaceuticals, Inc., a public biotechnology company, from 2006 to 2014 and Phase Forward, Inc., a public technology company, from
2000 to 2006. She began her career at the law offices of Fullbright & Jaworski. Ms. Herscha holds a J.D. from Boston University School of Law and a
B.A. from Boston University.
Joel Schneider, Ph.D. has served as our Chief Technology Officer and Head of Exploratory Research and Development since June 2017.
Dr. Schneider also served as an Analyst from March 2014 to March 2015, a Director from March 2015 to January 2017 and our Vice President of Research
and Development from January 2017 to June 2017. Prior to joining us, Dr. Schneider completed a postdoctoral fellowship at Harvard University in the
Department of Stem Cell and Regenerative Biology from January 2013 to 2014. He holds a Ph.D. in Cell Biology and Molecular Medicine from Rutgers
University and a B.A. in Biology from Brandeis University.
91
Jennifer Ziolkowski has served as our Chief Financial Officer and Treasurer since May 2017. Prior to joining us, she served as the Head of Sales
Operations, North America for Philips Healthcare, a healthcare company, from 2014 to 2017 and as its Senior Director of Finance, North America from
2012 to 2014. Ms. Ziolkowski served as Controller of Medical Consumables and Sensors from 2010 to 2012, Director of Finance of Imaging Systems from
2008 to 2010, Senior Director of Finance and Corporate Controller from 2007 to 2008 at TransMedics, Inc., a medical device company, and held various
finance and corporate development leadership positions at Cytyc Corporation, a medical technology company, from 2001 to 2007. From 1996 to 2001,
Ms. Ziolkowski gained significant experience at PricewaterhouseCoopers LLP where she served as a Senior Transaction Services Consultant and as Audit
Senior and Staff in the Boston Technology Group. Ms. Ziolkowski holds a B.S. in Accounting from Boston College and is a Certified Public Accountant.
Non-employee directors
Andrey Zarur, Ph.D. is one of our co-founders and has served as the Chairman of our board of directors since our inception in 2013. Dr. Zarur co-
founded GreenLight Biosciences Inc., a biotechnology company, in August 2008, and currently serves as its Chairman and Chief Executive Officer. From
January 2006 to August 2014, he served as Managing General Partner of Kodiak Venture Partners, a venture capital firm. Dr. Zarur also serves as a member
of the board of directors for a number of private companies. Dr. Zarur holds an M.S. and a Ph.D. from Massachusetts Institute of Technology and an
undergraduate degree from Universidad Nacional Autónoma de México. Mr. Zarur is qualified to serve on our board of directors based on his over 20 years
of experience in leading companies from clinical-stage drug development to global commercialization.
Matthew Arnold is a founding member of Solid and has served as a member of our board of directors since our inception in 2013. A former energy
executive, since 2009, Mr. Arnold has been actively working with startup businesses in the United Kingdom and Europe, primarily in the technology and
clean tech sectors. He holds an M.S. from the University of Virginia and a B.A. from Duke University. Mr. Arnold is qualified to serve on our board of
directors because of his extensive management and board experience with startup companies and his background in finance.
Martin Freed, M.D., F.A.C.P. has served as a member of our board of directors since June 2018. Dr. Freed has served as an independent consultant
to several private pharmaceutical, biotechnology, and healthcare companies, specializing in clinical and general pharmaceutical development and clinical
and regulatory strategy since February 2015. He co-founded and served as chief medical officer of Civitas Therapeutics, Inc., a biopharmaceutical company
acquired by Acorda Therapeutics, Inc., or Acorda, from December 2010 to October 2014, and as senior vice president, clinical development of Acorda
from October 2014 through January 2015. Dr. Freed spent nearly 14 years at GlaxoSmithKline and its predecessor, SmithKline Beecham Pharmaceuticals
or SmithKline Beecham, where he served numerous roles including vice president, clinical development and medical affairs in the metabolism therapeutic
area. Dr. Freed currently serves on the board of directors of Dicerna Pharmaceuticals, Inc. Dr. Freed has been Board Certified in Internal Medicine,
Nephrology and Clinical Pharmacology. He performed his internal medicine residency at Temple University Hospital and nephrology fellowship at Yale-
New Haven Hospital. A Fellow of the American College of Physicians, Dr. Freed received a B.S. with distinction in biology from the University of
Delaware and an M.D. from Pennsylvania State University’s College of Medicine. Dr. Freed is qualified to serve on our board of directors because of his
extensive leadership experience, his public company board experience and his experience working in the healthcare sector.
Robert Huffines has served as a member of our board of directors since December 2013. Mr. Huffines joined J.P. Morgan, a leading global financial
services firm, in 1991 and currently serves as the Global Chairman of Investment Banking, a position he has held since February 2017. Throughout his
career at J.P. Morgan, Mr. Huffines has held various leadership positions, including serving as Co-Head of the Global Healthcare Investment Banking
Group from 2002 to 2010 and Vice Chairman from 2011 to January 2017. Mr. Huffines received an M.B.A. from the University of Virginia and a B.A.
from the University of North Carolina. Mr. Huffines is qualified to serve on our board of directors based on his over 25 years of experience advising
healthcare companies and his leadership experience.
Adam Koppel, M.D., Ph.D. has served as a member of our board of directors since October 2017. Dr. Koppel rejoined Bain Capital, a global
investment firm, in 2016 as a Managing Director of Bain Capital Life Sciences. He initially joined Bain Capital Public Equity in 2003 where he was a
leader within the healthcare sector until mid-2014. During the period from mid-2014 to mid-2016, Dr. Koppel worked at Biogen Inc., or Biogen, a
biotechnology company, where he served as EVP of Corporate Development and Chief Strategy Officer. Prior to joining Bain Capital in 2003, Dr. Koppel
was an Associate Principal at McKinsey & Co., a management consulting firm, where he served a variety of healthcare companies. Dr. Koppel currently
serves on the board of directors of Aptinyx Inc. and Dicerna Pharmaceuticals, Inc., both public biotechnology companies. Previously, Dr. Koppel served of
the board of directors of Trevena Inc. and PTC Therapeutics,
92
Inc. Dr. Koppel also serves as a member of the board of directors for a number of private companies. Dr. Koppel received an M.D. and Ph.D. in
Neuroscience from the University of Pennsylvania School of Medicine. He also received an M.B.A. from The Wharton School at the University of
Pennsylvania, where he was a Palmer Scholar. He graduated magna cum laude from Harvard University with an A.B. and A.M. in History and Science.
Dr. Koppel is qualified to serve on our board of directors because of his extensive leadership experience, his public company board experience and his
experience working in the healthcare sector.
Sukumar Nagendran, M.D., has served as a member of our board of directors since September 2018. Since February 2020, Dr. Nagendran has
served as Chief Medical Officer of an unnamed biopharmaceutical holding company. Prior to that, he was most recently the Chief Medical Officer &
Senior Vice President of AveXis Inc., a clinical-stage gene therapy company, or AveXis, from September 2015 to July 2018, prior to the company’s
acquisition by Novartis. Prior to AveXis, Dr. Nagendran was Vice President of Medical Affairs of Quest Diagnostics from March 2013 to September
2015. Prior to Quest Diagnostics, Dr. Nagendran held key leadership positions at Pfizer, Novartis, Daiichi Sankyo, and Reata Pharmaceuticals. Prior to
moving to the biotech industry, Dr. Nagendran practiced internal medicine, with a focus on diabetes and cardiovascular disease. He is a Mayo Alumni
Laureate and founding member of the Robert Wood Johnson Legacy Society. He is also the sponsor for the Fonseca-Nagendran Scholar award at the
American Diabetes Association to enhance research in minority populations. Dr. Nagendran received his undergraduate degree in Biochemistry from
Rutgers University and his M.D. from Rutgers Medical School and trained in Internal Medicine at Mayo Clinic, Rochester. Dr. Nagendran is qualified to
serve on our board of directors because of his extensive leadership experience and his experience working in the healthcare sector.
Rajeev Shah has served as a member of our board of directors since March 2017. Mr. Shah has been a managing partner at RA Capital
Management, L.P., a multi-stage investment manager dedicated to evidence-based investing in public and private healthcare and life science companies that
are developing drugs, medical devices, and diagnostics, since 2004. Mr. Shah is also a member of the board of directors of Eidos Therapeutics, Inc., Kala
Pharmaceuticals, Inc., Satsuma Pharmaceuticals, Inc., Ra Pharmaceuticals, Inc. and Black Diamond Therapeutics, Inc. Mr. Shah was previously a member
of the board of directors of KalVista Pharmaceuticals, Inc., a public pharmaceutical company, from June 2015 through April 2018. Mr. Shah received a
B.A. in Chemistry from Cornell University. Mr. Shah is qualified to serve on our board of directors because of his extensive leadership experience, his
public company board experience and his experience investing in life science companies.
Adam Stone has served as a member of our board of directors since November 2015. Mr. Stone is currently the Chief Investment Officer of
Perceptive Advisors, a life science focused hedge fund, where he has worked since May 2006. Mr. Stone received a B.A. from Princeton University.
Mr. Stone is qualified to serve on our board of directors because of his extensive experience developing early-stage biotech and health care companies.
Lynne Sullivan has served as a member of our board of directors since November 2015. Ms. Sullivan served as the Chief Financial Officer for
Compass Therapeutics, LLC, a biotechnology company, or Compass, from December 2018 to August 2019. Prior to Compass, Ms. Sullivan served as
Biogen’s Senior Vice President of Finance from 2016 to December 2018, where she also served as Vice President of Tax and Corporate Finance from
February 2015 to March 2016 and Vice President of Tax from April 2008 to February 2015. Ms. Sullivan is currently a member of the board of directors of
resTORbio, Inc. and BiomX Inc., both of which are public biopharmaceutical companies. Ms. Sullivan also serves on the board of Inheris Biopharma, Inc.,
a private biopharmaceutical company. She received an M.S. in Taxation from Bentley University and a B.S.B.A. from Suffolk University. Ms. Sullivan was
a Certified Public Account for over 20 years. Ms. Sullivan is qualified to serve on our board of directors because of her extensive experience in public
accounting and financial expertise and her experience working in the healthcare sector.
Delinquent Section 16(a) Reports
Section 16(a) of the Exchange Act requires our directors and executive officers and holders of more than 10% of our common stock to file with the
SEC initial reports of ownership and reports of changes in ownership of our common stock and other equity securities. These Section 16 reporting persons
are required by SEC regulation to furnish us with copies of all Section 16(a) forms they file.
Based solely on a review of reports furnished to us, or written representations from reporting persons, we believe all directors, executive officers,
and 10% owners timely filed all reports regarding transactions in our securities required to be filed for the fiscal year ended December 31, 2019 by Section
16(a) under the Exchange Act, with the exception of a Form 4/A filed by Ilan Ganot on February 12, 2020 to report an option grant to Mr. Ganot’s wife
that, at the time of filing the original Form 4 on January 29, 2020, was inadvertently omitted.
93
Audit Committee
Our board of directors has established an audit committee, which operates under a charter that has been approved by our board of directors and that
is available on the Investor Relations portion of our website, www.solidbio.com Our audit committee consists of Ms. Sullivan, Mr. Arnold and Dr. Koppel,
with Ms. Sullivan serving as chair of the audit committee. Our board of directors has determined that each of these individuals meets the independence
requirements of the Sarbanes-Oxley Act, Rule 10A-3 under the Exchange Act, and the applicable listing standards of Nasdaq. Each member of our audit
committee can read and understand fundamental financial statements in accordance with Nasdaq audit committee requirements. In arriving at this
determination, the board has examined each audit committee member’s employment and other experience. Our board of directors has determined that
Ms. Sullivan qualifies as an audit committee financial expert within the meaning of SEC regulations and meets the financial sophistication requirements of
the Nasdaq listing rules. In making this determination, our board has considered Ms. Sullivan’s formal education and previous and current experience in
financial roles. Both our independent registered public accounting firm and management periodically meet privately with our audit committee.
The functions of our audit committee include, among other things:
•
•
•
•
•
•
•
•
•
•
•
•
•
evaluating the performance, independence and qualifications of our independent auditors and determining whether to retain our existing
independent auditors or engage new independent auditors;
reviewing and approving the engagement of our independent auditors to perform audit services and any permissible non-audit services;
monitoring the rotation of partners of our independent auditors on our engagement team as required by law;
prior to engagement of any independent auditor, and at least annually thereafter, reviewing relationships that may reasonably be thought to
bear on their independence, and assessing and otherwise taking the appropriate action to oversee the independence of our independent auditor;
reviewing our annual and quarterly financial statements and reports, including the disclosures contained under the caption “Management’s
Discussion and Analysis of Financial Condition and Results of Operations,” and discussing the statements and reports with our independent
auditors and management;
reviewing with our independent auditors and management any significant issues that arise regarding accounting principles and financial
statement presentation and matters concerning the scope, adequacy and effectiveness of our financial controls;
reviewing with management and our auditors any earnings announcements and other public announcements regarding material financial
developments;
establishing procedures for the receipt, retention and treatment of complaints received by us regarding financial controls, accounting or
auditing matters and other matters;
preparing the audit committee report that the SEC requires in our annual proxy statement;
reviewing and providing oversight of any related-person transactions in accordance with our related-person transaction policy and reviewing
and monitoring compliance with legal and regulatory requirements, including our code of business conduct and ethics;
reviewing our major financial risk exposures, including the guidelines and policies to govern the process by which risk assessment and risk
management is implemented;
reviewing on a periodic basis our investment policy; and
reviewing and evaluating on an annual basis the performance of the audit committee and the audit committee charter.
Our board of directors has determined that the composition and functioning of our audit committee complies with all applicable requirements of the
Sarbanes-Oxley Act, and all applicable SEC and Nasdaq rules and regulations.
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Code of business conduct and ethics
We have adopted a Code of Business Conduct and Ethics, or the Code of Conduct, that applies to our directors, executive officers and employees,
including our principal executive officer, principal financial officer, principal accounting officer or controller, or persons performing similar functions. The
Code of Conduct is available on the Investor Relations portion of our website, www.solidbio.com. The nominating and corporate governance committee of
our board of directors is responsible for overseeing the Code of Conduct and must approve any waivers of the Code of Conduct for employees, executive
officers and directors. In addition, we intend to post on our website all disclosures that are required by law or the listing standards of Nasdaq concerning
any amendments to, or waivers of, any provision of the Code of Conduct.
Item 11.
Executive Compensation.
Compensation of our Named Executive Officers
The following information describes the material elements of compensation awarded to, earned by or paid to each of our named executive officers,
or the Named Executive Officers. The Named Executive Officers for the year ended December 31, 2019 are:
•
•
•
Ilan Ganot, our Chief Executive Officer;
Jorge Quiroz, our former Chief Medical Officer; and
Alvaro Amorrortu, our former Chief Operating Officer.
Summary Compensation Table for Fiscal Year 2019
The following table contains information about the compensation paid to or earned by each of our Named Executive Officers during the most
recently completed fiscal year.
Name and Principal Position
Ilan Ganot, President and Chief Executive
Officer
Jorge Quiroz, M.D., Former Chief Medical
Officer (3)
Alvaro Amorrortu, Former Chief Operating
Officer (4)
Year
Salary
($)
Bonus
($) (1)
Option Awards
($) (2)
Total ($)
2019
2018
2019
2019
2018
520,000
450,000
193,050
247,500
2,622,879
5,286,270
3,335,929
5,983,770
412,500
113,400
1,015,308
1,541,208
400,000
330,000
108,000
132,000
1,015,308
2,380,736
1,523,308
2,842,736
(1)
(2)
(3)
(4)
Represents annual bonuses paid to the Named Executive Officers granted after the completion of each calendar year at the discretion of the Board
of Directors.
The amount in this column represents the aggregate grant date fair value of the award as computed in accordance with Financial Accounting
Standard Board Accounting Standards Codification Topic 718. The assumptions used in calculating the grant date fair value of the award reported
in this column are set forth in Note 12 to our audited consolidated financial statements appearing elsewhere in this Annual Report on Form 10-K.
Dr. Quiroz’s employment terminated on January 15, 2020.
Mr. Amorrortu’s employment terminated on January 15, 2020.
Narrative to Summary Compensation Table
Base Salary. In 2019, we paid our Named Executive Officers base salaries as follows: Mr. Ganot: $520,000; Dr. Quiroz: $412,500; and Mr.
Amorrortu: $400,000. For 2020, our board of directors increased the base salary amount for Mr. Ganot to $535,000.
We use base salaries to recognize the experience, skills, knowledge and responsibilities required of all our employees, including our Named
Executive Officers. None of our Named Executive Officers is currently party to an employment agreement or other agreement or arrangement that provides
for automatic or scheduled increases in base salary.
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Annual Bonus. Our board of directors may, in its discretion, award bonuses to our Named Executive Officers from time to time. Our employment
agreements with our Named Executive Officers provide (or provided, in the case of Dr. Quiroz and Mr. Amorrortu) that they will be eligible for annual
performance-based bonuses up to a specified percentage of their salary, subject to approval by our board of directors. Performance-based bonuses, which
are calculated as a percentage of base salary, are designed to motivate our employees to achieve annual goals based on our strategic, financial and operating
performance objectives. From time to time, our board of directors has approved discretionary annual cash bonuses to our Named Executive Officers with
respect to their prior year performance.
With respect to 2019, our board of directors awarded discretionary bonuses of $193,050, $113,400 and $108,000 to Mr. Ganot, Dr. Quiroz and Mr.
Amorrortu, respectively.
Equity Incentives. Although we do not have a formal policy with respect to the grant of equity incentive awards to our executive officers, or any
formal equity ownership guidelines applicable to them, we believe that equity grants provide our executives with a strong link to our long-term
performance, create an ownership culture and help to align the interests of our executives and our stockholders. In addition, we believe that equity grants
with a time-based vesting feature promote executive retention because this feature incents our executive officers to remain in our employment during the
vesting period. Accordingly, our board of directors periodically reviews the equity incentive compensation of our Named Executive Officers and from time
to time may grant equity incentive awards to them in the form of stock options.
In January 2019, we granted options to purchase 155,000 shares of our common stock to Mr. Ganot and 60,000 shares of our common stock to each
of Dr. Quiroz and Mr. Amorrortu. These options vest in equal annual installments over a term of four years from the date of grant.
Our employees and executives are eligible to receive stock options and other stock-based awards pursuant to our 2018 Omnibus Incentive Plan, or
the 2018 Plan.
We use stock options to compensate our executive officers in the form of initial grants in connection with the commencement of employment and
also at various times, often but not necessarily annually, if we have performed as expected or better than expected. None of our executive officers is
currently party to an employment agreement that provides for automatic award of stock options. We have granted stock options to our executive officers
with time-based vesting. The options that we have granted to our executive officers typically become exercisable as to 25% of the shares underlying the
option on the first anniversary of the grant date and as to an additional 25% of the original number of shares underlying the option annually thereafter.
Vesting rights cease upon termination of employment and exercise rights cease shortly after termination, except that vesting is fully accelerated upon
certain terminations in connection with a change of control and exercisability is extended in the case of death or disability. Prior to the exercise of an
option, the holder has no rights as a stockholder with respect to the shares subject to such option, including no voting rights and no right to receive
dividends or dividend equivalents.
The exercise price of all stock options granted after the closing of our initial public offering is equal to the fair market value of shares of our
common stock on the date of grant, which is determined by reference to the closing market price of our common stock on the date of grant.
Employment Agreements
We have entered into employment agreements with each of our Named Executive Officers. The employment agreements set forth the terms of the
Named Executive Officers’ compensation, including their base salary, and annual performance bonus opportunity. In addition, the employment agreements
provide that, subject to eligibility requirements under the plan documents governing such programs and our policies, the Named Executive Officers are (or
were, in the case of Dr. Quiroz and Mr. Amorrortu) entitled, on the same basis as our other employees, to participate in and receive benefits under, any
medical, vision and dental insurance policy maintained by us and we will pay, consistent with our then-current employee benefit policy, a portion of the
cost of the premiums for any such insurance policy in which the Named Executive Officer elects to participate. Each Named Executive Officer will also be
eligible (or was eligible, in the case of Dr. Quiroz and Mr. Amorrortu) to receive paid vacation time, sick time, and Company holidays consistent with our
policies as then in effect from time to time and equity awards at such times and on such terms and conditions as the board of directors may
determine. Each Named Executive Officer’s employment is (or was, in the case of Dr. Quiroz and Mr. Amorrortu) at will.
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Employment Agreement with Ilan Ganot
On January 25, 2019, we entered into an employment agreement with Mr. Ganot, our President and Chief Executive Officer, which employment
agreement amended and restated the terms of his existing agreement (with the exception of the restrictive covenant provisions contained therein).
Pursuant to his employment agreement, Mr. Ganot is entitled to an annual base salary of $520,000, effective as of January 1, 2019, which base
salary will be reviewed by the board of directors from time to time and is subject to change in the discretion of the board of directors. Mr. Ganot is also
eligible to earn an annual performance bonus, with a target bonus amount equal to up to 55% of his base salary, based upon the board’s assessment of his
performance and the Company’s attainment of targeted goals as set by the board in its sole discretion. The bonus may be in the form of cash, equity
award(s), or a combination of cash and equity.
Mr. Ganot will remain bound by proprietary rights, non-disclosure, developments, non-competition and non-solicitation obligations pursuant to the
restrictive covenants in his existing employment agreement, which provisions shall remain in full force and effect. Under these restrictive covenants, he has
agreed not to compete with us during his employment and for a period of one year after the termination of his employment (provided that he is not
restricted from promoting treatments for, or endeavoring to cure, DMD), not to solicit our employees, consultants, or actual or prospective customers or
business relations during his employment and for a period of one year after the termination of his employment, and to protect our confidential and
proprietary information indefinitely.
Mr. Ganot’s employment agreement and his employment may be terminated: (1) upon his death or at our election due to his “disability”; (2) at our
election, with or without “cause”; and (3) at his election, with or without “good reason” (as such terms are defined in his employment agreement).
In the event of the termination of Mr. Ganot’s employment by us without cause, or by Mr. Ganot for good reason, prior to or more than twelve
months following a “change in control” (as defined in his employment agreement), Mr. Ganot is entitled to receive his base salary that has accrued and to
which he is entitled as of the termination date, to the extent consistent with Company policy, accrued but unused paid time off through and including the
termination date, unreimbursed business expenses for which expenses he has timely submitted appropriate documentation, and other amounts or benefits to
which he is entitled in accordance with the terms of the benefit plans then-sponsored by us, which we refer to collectively as the Ganot Accrued
Obligations. In addition, subject to his execution and nonrevocation of a release of claims in our favor, Mr. Ganot is entitled to (1) continued payment of his
base salary, in accordance with our regular payroll procedures, for a period of 12 months and (2) provided he is eligible for and timely elects to continue
receiving group medical insurance under COBRA and the payments would not result in the violation of nondiscrimination requirements of applicable law,
payment by us of the portion of health coverage premiums we pay for similarly-situated, active employees who receive the same type of coverage, for a
period of up to 12 months following his date of termination.
In the event of the termination of Mr. Ganot’s employment by us without cause, or by Mr. Ganot for good reason, within twelve months following a
change in control, he is entitled to receive the Ganot Accrued Obligations. In addition, subject to his execution and nonrevocation of a release of claims in
our favor, he is entitled to (1) continued payment of his base salary, in accordance with our regular payroll procedures, for a period of 18 months, (2)
provided he is eligible for and timely elects to continue receiving group medical insurance under COBRA and the payments would not result in the
violation of nondiscrimination requirements of applicable law, payment by us of the portion of health coverage premiums we pay for similarly-situated,
active employees who receive the same type of coverage, for a period of up to 18 months following his date of termination, (3) a lump sum payment equal
to 150% of his target bonus for the year in which his employment is terminated or, if higher, his target bonus immediately prior to the change in control and
(4) full vesting acceleration of any then-unvested equity awards that vest based solely based on the passage of time held by Mr. Ganot, such that any such
equity awards held by him become fully exercisable or non-forfeitable as of the termination date.
If Mr. Ganot’s employment is terminated for any other reason, including as a result of his death or disability, for cause, or voluntarily by him
without good reason, our obligations under the employment agreement cease immediately, and he is only entitled to receive the Ganot Accrued Obligations.
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Employment Agreement with Jorge Quiroz
On January 25, 2019, we entered into an employment agreement with Dr. Quiroz, our former Chief Medical Officer, which employment agreement
amended and restated the terms of his existing agreement (with the exception of the restrictive covenant provisions contained therein).
Pursuant to his employment agreement, Dr. Quiroz was entitled to an annual base salary of $412,500, effective as of January 1, 2019, which base
salary was subject to change in the discretion of the board of directors. Dr. Quiroz was also eligible to earn an annual performance bonus in the form of
cash, equity award(s), or a combination of cash and equity, with a target bonus amount equal to up to 40% of his base salary, based upon the board’s
assessment of his performance and the Company’s attainment of targeted goals as set by the board in its sole discretion.
Dr. Quiroz will remain bound by proprietary rights, non-disclosure, developments, non-competition and non-solicitation obligations pursuant to the
restrictive covenants in his existing employment agreement, which provisions shall remain in full force and effect. Under these restrictive covenants, he has
agreed not to compete with us during his employment and for a period of one year after the termination of his employment, not to solicit our employees,
consultants, or actual or prospective customers or business relations during his employment and for a period of one year after the termination of his
employment, and to protect our confidential and proprietary information indefinitely.
In the event of the termination of Dr. Quiroz’s employment by us without cause, or by Dr. Quiroz for good reason, prior to or more than twelve
months following a “change in control” (as defined in his employment agreement), Dr. Quiroz is entitled to receive his base salary that has accrued and to
which he is entitled as of the termination date, to the extent consistent with Company policy, accrued but unused paid time off through and including the
termination date, unreimbursed business expenses for which expenses he has timely submitted appropriate documentation, and other amounts or benefits to
which he is entitled in accordance with the terms of the benefit plans then-sponsored by us. In addition, subject to his execution and nonrevocation of a
release of claims in our favor, Dr. Quiroz is entitled to (1) continued payment of his base salary, in accordance with our regular payroll procedures, for a
period of 12 months and (2) provided he is eligible for and timely elects to continue receiving group medical insurance under COBRA and the payments
would not result in the violation of nondiscrimination requirements of applicable law, payment by us of the portion of health coverage premiums we pay for
similarly-situated, active employees who receive the same type of coverage, for a period of up to 12 months following his date of termination.
Dr. Quiroz’s employment was terminated by us without cause effective January 15, 2020. In connection with his departure, we will pay Dr. Quiroz,
pursuant to the terms of his employment agreement, the severance amounts described above. In addition, we agreed to extend the time he will have to
exercise his outstanding stock options to January 15, 2021.
Employment Agreement with Alvaro Amorrortu
On January 25, 2019, we entered into an employment agreement with Mr. Amorrortu, our former Chief Operating Officer, which employment
agreement amended and restated the terms of his existing agreement (with the exception of the restrictive covenant provisions contained therein).
Pursuant to his employment agreement, Mr. Amorrortu was entitled to an annual base salary of $400,000, effective as of January 1, 2019, which
base salary was subject to change in the discretion of the board of directors. Mr. Amorrortu was also eligible to earn an annual performance bonus in the
form of cash, equity award(s), or a combination of cash and equity, with a target bonus amount equal to up to 40% of his base salary, based upon the
board’s assessment of his performance and the Company’s attainment of targeted goals as set by the board in its sole discretion.
Mr. Amorrortu will remain bound by proprietary rights, non-disclosure, developments, non-competition and non-solicitation obligations pursuant to
the restrictive covenants in his existing employment agreement, which provisions shall remain in full force and effect. Under these restrictive covenants, he
has agreed not to compete with us during his employment and for a period of one year after the termination of his employment, not to solicit our
employees, consultants, or actual or prospective customers or business relations during his employment and for a period of one year after the termination of
his employment, and to protect our confidential and proprietary information indefinitely.
In the event of the termination of Mr. Amorrortu’s employment by us without cause, or by Mr. Amorrortu for good reason, prior to or more than
twelve months following a “change in control” (as defined in his employment agreement), Mr. Amorrortu is entitled to receive his base salary that has
accrued and to which he is entitled as of the termination date, to the extent consistent with Company policy, accrued but unused paid time off through and
including the termination date,
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unreimbursed business expenses for which expenses he has timely submitted appropriate documentation, and other amounts or benefits to which he is
entitled in accordance with the terms of the benefit plans then-sponsored by us. In addition, subject to his execution and nonrevocation of a release of
claims in our favor, Mr. Amorrortu is entitled to (1) continued payment of his base salary, in accordance with our regular payroll procedures, for a period of
12 months and (2) provided he is eligible for and timely elects to continue receiving group medical insurance under COBRA and the payments would not
result in the violation of nondiscrimination requirements of applicable law, payment by us of the portion of health coverage premiums we pay for similarly-
situated, active employees who receive the same type of coverage, for a period of up to 12 months following his date of termination.
Mr. Amorrortu’s employment was terminated by us without cause effective January 15, 2020. In connection with his departure, we will pay Mr.
Amorrortu, pursuant to the terms of his employment agreement, the severance amounts described above. In addition, we agreed to extend the time he will
have to exercise his outstanding stock options to January 15, 2021.
2019 Outstanding Equity Awards at Fiscal Year-End
The following table sets forth information regarding equity awards held by our Named Executive Officers as of December 31, 2019.
Name
Ilan Ganot
Jorge Quiroz
Alvaro Amorrortu
Number of securities
underlying
unexercised
options
(#) exercisable
48,250
—
(1 )
11,765
—
(3 )
33,664
—
(3 )
Option Awards
Number of securities
underlying unexercised
options (#)
unexercisable
144,750
155,000
(1 )
(2 )
Option
exercise
price ($)
37.89
22.93
Option expiration
date
July 25, 2028
January 23, 2029
Stock Awards
Number
of shares of
stock that
have not
vested (#)
Market value of
shares of stock
that have not
vested ($) (8)
35,296
60,000
(3 )
(2 )
26.23
22.93
February 14, 2028
January 23, 2029
100,993
60,000
(3 )
(2 )
26.23
22.93
February 14, 2028
January 23, 2029
19,169
23,960
(4 )
(5 )
85,302
106,622
8,419
10,029
(6 )
(7 )
37,465
44,629
(1)
(2)
(3)
(4)
(5)
(6)
(7)
(8)
This option was granted on July 25, 2018 under the 2018 Plan and is subject to vesting in equal annual installments over four years from the vesting
start date through and including July 25, 2022.
This option was granted on January 23, 2019 under the 2018 Plan and is subject to vesting in equal annual installments over four years from the
vesting start date through and including January 23, 2023.
This option was granted on February 14, 2018 under the 2018 Plan and is subject to vesting in equal annual installments over four years from the
vesting start date through and including February 14, 2022.
Consists of restricted stock awards granted under our Solid Biosciences, LLC Amended and Restated Equity Incentive Plan. The grant was made on
September 12, 2017 and is subject to vesting in equal annual installments over four years from the vesting start date through and including
September 12, 2021.
Consists of restricted stock awards granted under our Solid Biosciences, LLC Amended and Restated Equity Incentive Plan. The grant was made on
December 7, 2017, vested as to 25% on the first anniversary of the vesting start date and is subject to equal semi-annual vesting installments
thereafter through and including February 14, 2022.
Consists of restricted stock awards granted under our Solid Biosciences, LLC Amended and Restated Equity Incentive Plan. The grant was made on
September 12, 2017, vested as to 25% on the first anniversary of the vesting start date and is subject to equal monthly vesting installments thereafter
through and including September 12, 2021.
Consists of restricted stock awards granted under our Solid Biosciences, LLC Amended and Restated Equity Incentive Plan. The grant was made on
December 7, 2017, vested as to 25% on the first anniversary of the vesting start date and is subject to equal monthly vesting installments thereafter
through and including January 30, 2022.
Based on the $4.45 closing sale price of our common stock on December 31, 2019 as reported by the Nasdaq Global Select Market.
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On January 27, 2020, we granted a stock option to Mr. Ganot for the right to buy 142,000 shares of our common stock. The options have an
exercise price of $3.47 per share, vest in four equal installments beginning on January 27, 2021 and expire on January 27, 2030. In addition, we granted
Mr. Ganot 71,000 restricted stock units on January 27, 2020. The restricted stock units vest in two equal installments. Fifty percent will vest six months
after the grant date with the remaining fifty percent vesting on the first anniversary of the grant date.
Equity Incentive Plans
Solid Biosciences, LLC Amended and Restated Equity Incentive Plan
Prior to the closing of our initial public offering, Solid Biosciences, LLC maintained its Amended and Restated Equity Incentive Plan, or the
Existing Plan, under which Solid Biosciences, LLC granted Series D Common Units to its employees, consultants and other service providers. The Existing
Plan was frozen in connection with our initial public offering. No further awards will be granted under the Existing Plan, but awards granted prior to the
freeze date will continue in accordance with their terms and the terms of the Existing Plan.
2018 Omnibus Incentive Plan
Prior to our initial public offering, the board of managers of Solid Biosciences, LLC adopted the Solid Biosciences Inc. 2018 Omnibus Incentive
Plan, or 2018 Plan, contingent upon the consummation of our initial public offering. The unitholders of Solid Biosciences, LLC approved the 2018 Plan
contingent upon the consummation of our initial public offering.
The material terms of the 2018 Plan are summarized below. The following summary is qualified in its entirety by reference to the complete text of
the 2018 Plan, a copy of which was filed as Exhibit 99.1 to our registration statement on Form S-8 filed with the SEC on January 29, 2018 and is
incorporated herein by reference.
Administration of the plan
The board of managers of Solid Biosciences, LLC appointed the compensation committee of our board of directors as the committee under the 2018
Plan with the authority to administer the 2018 Plan. We refer to our board of directors or compensation committee, as applicable, as the Administrator. The
Administrator is authorized to grant awards to eligible employees, consultants and non-employee directors.
Number of authorized shares and award limits
The aggregate number of our shares of common stock that may be issued or used for reference purposes under the 2018 Plan may not exceed
5,001,000 shares (subject to adjustment as described below). Our shares of common stock that are subject to awards will be counted against the overall
limit as one share for every share granted or covered by an award. If any award is cancelled, expires or terminates unexercised for any reason, the shares
covered by such award will again be available for the grant of awards under the 2018 Plan, except that any shares that are not issued as the result of a net
exercise or settlement or that are used to pay any exercise price or tax withholding obligation will not be available for the grant of awards. Shares of
common stock that we repurchase on the open market with the proceeds of an option exercise price also will not be available for the grant of awards.
Awards that may be settled solely in cash will not be deemed to use any shares.
The maximum number of our shares of common stock that may be granted pursuant to awards under the 2018 Plan during any fiscal year to any
non-employee director is 1,000,200 shares. The foregoing individual participant limit is cumulative; that is, to the extent that shares of common stock that
may be granted to an individual in a fiscal year are not granted, the number of shares of common stock that may be granted to such individual is increased
in the subsequent fiscal years during the term of the 2018 Plan until used.
The Administrator will, in accordance with the terms of the 2018 Plan, make appropriate adjustments to the above aggregate and individual limits
(other than cash limitations), to the number and/or kind of shares or other property (including cash) underlying awards and to the purchase price of shares
underlying awards, in each case, to reflect any change in our capital structure or business by reason of any stock split, reverse stock split, stock dividend,
combination or reclassification of shares, any recapitalization, merger, consolidation, spin off, split off, reorganization or any partial or complete
liquidation, any sale or transfer of all or part of our assets or business, or any other corporate transaction or event that would be considered an “equity
restructuring” within the meaning of Financial Accounting Standards Board Accounting Standards Codification Topic 718. In addition, the Administrator
may take similar action with respect to other extraordinary events.
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Eligibility and participation
All of our current and prospective employees and consultants, as well as our non-employee directors, are eligible to be granted non-qualified stock
options, restricted stock, performance-based cash awards and other stock-based awards under the 2018 Plan. Only our and our subsidiaries’ employees are
eligible to be granted incentive stock options, or ISOs, under the 2018 Plan. Eligibility for awards under the 2018 Plan is determined by the Administrator
in its discretion. In addition, each member of our board of directors who is not an employee of the company or any of our affiliates is expected to be
eligible to receive awards under the 2018 Plan.
Types of awards
Stock options. The 2018 Plan authorizes the Administrator to grant ISOs to eligible employees and non-qualified stock options to purchase shares to
employees, consultants, prospective employees, prospective consultants and non-employee directors. The Administrator will determine the number of
shares of common stock subject to each option, the term of each option, the exercise price (which may not be less than the fair market value of the shares of
common stock at the time of grant, or 110% of fair market value in the case of ISOs granted to 10% stockholders), the vesting schedule and the other terms
and conditions of each option. Options will be exercisable at such times and subject to such terms as are determined by the Administrator at the time of
grant. The maximum term of options under the 2018 Plan is ten years (or five years in the case of ISOs granted to 10% stockholders). Upon the exercise of
an option, the participant must make payment of the full exercise price (a) in cash or by check, bank draft or money order; (b) solely to the extent permitted
by law and authorized by the Administrator, through the delivery of irrevocable instructions to a broker, reasonably acceptable to us, to promptly deliver to
us an amount equal to the aggregate exercise price; or (c) on such other terms and conditions as may be acceptable to the Administrator (including, without
limitation, the relinquishment of options or by payment in full or in part in the form of shares of common stock).
Restricted stock. The 2018 Plan authorizes the Administrator to grant restricted stock. Recipients of restricted stock will be required to enter into an
agreement with us subjecting the restricted stock to transfer and other restrictions and providing the criteria or dates on which such awards vest and such
restrictions lapse. The restrictions on restricted stock may lapse and the awards may vest over time, based on performance criteria or other factors, as
determined by the Administrator at the time of grant. Except as otherwise determined by the Administrator, a holder of restricted stock has all of the
attendant rights of a stockholder including the right to receive dividends, if any, subject to and conditioned upon vesting and restrictions lapsing on the
underlying restricted stock, the right to vote shares and, subject to and conditioned upon the vesting and restrictions lapsing for the underlying shares, the
right to tender such shares. However, the Administrator may in its discretion provide at the time of grant that the right to receive dividends on restricted
stock will not be subject to the vesting or lapsing of the restrictions on the restricted stock.
Other stock-based awards. The 2018 Plan authorizes the Administrator to grant awards of shares of common stock and other awards that are valued
in whole or in part by reference to, or are payable in or otherwise based on, shares of common stock, including, but not limited to, shares of common stock
awarded purely as a bonus and not subject to any restrictions or conditions; shares of common stock in payment of the amounts due under an incentive or
performance plan sponsored or maintained by us or an affiliate; stock appreciation rights; stock equivalent units; restricted stock units; performance awards
entitling participants to receive a number of shares of common stock (or cash in an equivalent value) or a fixed dollar amount, payable in cash, stock or a
combination of both, with respect to a designated performance period; or awards valued by reference to book value of our shares of common stock. In
general, other stock-based awards that are denominated in shares of common stock will include the right to receive dividends, if any, subject to and
conditioned upon vesting and restrictions lapsing on the underlying award, but the Administrator may in its discretion provide at the time of grant that the
right to receive dividends on a stock-denominated award will not be subject to the vesting or lapsing of the restrictions on the performance award.
Performance-based cash awards. The 2018 Plan authorizes the Administrator to grant cash awards that are payable or otherwise based on the
attainment of pre-established performance goals during a performance period. These performance goals will be based on the attainment of a certain target
level of, or a specified increase in (or decrease where noted), criteria selected by the Administrator.
Such performance goals may be based upon the attainment of specified levels of company, affiliate, subsidiary, division, other operational unit,
business segment or administrative department performance relative to the performance of other companies. The Administrator may designate additional
business criteria on which the performance goals may be based or adjust, modify or amend those criteria. Unless the Administrator determines otherwise,
the Administrator will disregard and exclude the impact of special, unusual or non-recurring items, events, occurrences or circumstances; discontinued
operations or the disposal of a business; the operations of any business that we acquire during the fiscal year or other applicable performance period; or a
change in accounting standards required by generally accepted accounting principles or changes in applicable law or regulations.
101
Effect of certain transactions; Change in control
In the event of a change in control, as defined in the 2018 Plan, except as otherwise provided by the Administrator, unvested awards will not vest.
Instead, the Administrator may, in its sole discretion, provide that outstanding awards will be: assumed and continued; purchased based on the price per
share paid in the change in control transaction (less, in the case of options and stock appreciation rights, or SARs, the exercise price), as adjusted by the
Administrator for any contingent purchase price, escrow obligations, indemnification obligations or other adjustments to the purchase price; and/or in the
case of stock options or other stock-based appreciation awards where the change in control price is less than the applicable exercise price, cancelled.
However, the Administrator may in its sole discretion provide for the acceleration of vesting and lapse of restrictions of an award at any time including in
connection with a change in control.
Non-transferability of awards
Except as the Administrator may permit, at the time of grant or thereafter, awards granted under the 2018 Plan are generally not transferable by a
participant other than by will or the laws of descent and distribution. Shares of common stock acquired by a permissible transferee will continue to be
subject to the terms of the 2018 Plan and the applicable award agreement.
Term
Awards under the 2018 Plan may not be made after December 18, 2027, but awards granted prior to such date may extend beyond that date.
Amendment and termination
Subject to the rules referred to in the balance of this paragraph, our board of directors or the Administrator (to the extent permitted by law) may at
any time amend, in whole or in part, any or all of the provisions of the 2018 Plan, or suspend or terminate it entirely, retroactively or otherwise. Except as
required to comply with applicable law, no such amendment, suspension or termination may reduce the rights of a participant with respect to awards
previously granted without the consent of such participant. In addition, without the approval of stockholders, no amendment may be made that would:
increase the aggregate number of shares of common stock that may be issued under the 2018 Plan; increase the maximum individual participant share
limitations for a fiscal year or year of a performance period; change the classification of individuals eligible to receive awards under the 2018 Plan; extend
the maximum term of any option; reduce the exercise price of any option or SAR or cancel any outstanding “in-the-money” option or SAR in exchange for
cash; substitute any option or SAR in exchange for an option or SAR (or similar other award) with a lower exercise price; alter the performance goals; or
require stockholder approval in order for the 2018 Plan to continue to comply with Section 162(m) or Section 422 of the Internal Revenue Code of 1986, as
amended, or the Code.
Registration of shares
On January 29, 2018, we filed a registration statement on Form S-8 under the Securities Act of 1933, as amended, or the Securities Act, to register
the full number of shares of common stock that will be available for issuance under the 2018 Plan, as described in the section titled “Number of authorized
shares and award limits” above.
Non-employee director compensation
During 2019, we did not provide any compensation to Mr. Ganot, our President and Chief Executive Officer, for his service as a member of our
board. Mr. Ganot’s compensation as an executive officer is set forth above under “Executive Compensation-2019 Summary Compensation Table.”
Non-employee director compensation is set by our board of directors at the recommendation of our compensation committee. In 2019, the
compensation committee retained Radford, an AON Hewitt company, to assist in assessing our non-employee director compensation program and provide
recommendations in order to establish the compensation program.
Under our current director compensation program, we pay our non-employee directors a cash retainer for their service on the board of directors and
for their service on each committee of which the director is a member. The chairs of each committee receive higher retainers for such service. These fees
are payable in arrears in equal semi-annual installments not later than the 15th business day following the end of the second and fourth calendar quarters,
provided that the amount of
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such payment will be prorated for any portion of such semi-annual period that the director is not serving on the board, on such committee or in such
position, and no fee shall be payable in respect of any period prior to the completion of our initial public offering. The fees paid to non-employee directors
for their service on the board of directors and for their service on each committee of the board of directors of which the director is a member are as follows:
Committee
Board of Directors
Audit Committee
Clinical Committee
Compensation Committee
Nominating and Governance Committee
$
Member
Annual Fee
Chairperson
Incremental
Annual Fee
35,000 $
7,500
7,500
5,000
4,000
35,000
7,500
7,500
5,000
4,000
We also reimburse our non-employee directors for reasonable out-of-pocket business expenses incurred in connection with the performance of their
duties as directors, including travel expenses in connection with their attendance in person at our board of director and committee meetings.
In addition, under our current director compensation program, each new non-employee director elected to our board of directors receives an option
to purchase 20,000 shares of our common stock under our 2018 Plan. Each of these options vest in equal annual installments over a three-year period
measured from the date of grant, subject to the director’s continued service as a director. Further, on the date of our annual meeting of stockholders, each
non-employee director that has served on our board of directors for at least six months prior to such annual meeting receives an option to purchase 10,000
shares of our common stock under our 2018 Plan. Each of these options vest in full on the earlier to occur of the one-year anniversary of the grant date and
immediately prior to our first annual meeting of stockholders occurring after the grant date, subject to the director’s continued service as a director. All
options granted to our non-employee directors under our director compensation program will be issued at exercise prices equal to the fair market value of
our common stock on the date of grant and will become exercisable in full in the event of a change in control.
This program is intended to provide compensation for our non-employee directors in a manner that enables us to attract and retain outstanding
director candidates and reflects the substantial time commitment necessary to oversee our affairs. We also seek to align the interests of our directors and
our stockholders, and we have chosen to do so by compensating our non-employee directors with a mix of cash and equity-based compensation.
The table below shows the compensation paid to our non-employee directors during 2019.
Name
Andrey Zarur, Ph.D.
Matthew Arnold
Martin Freed, M.D., F.A.C.P.
Robert Huffines
Adam Koppel, M.D., Ph.D.
Sukumar Nagendran, M.D.
Rajeev Shah
Adam Stone
Lynne Sullivan
Option
Awards
($) (1)(2)
Fees
Earned
or
Paid in
Cash
($)
70,000 2,369,704 (3)
39,965 (4)
42,500
39,965 (4)
54,000
39,965 (4)
35,000
39,965 (4)
60,000
39,965 (4)
42,500
39,965 (4)
40,000
39,965 (4)
48,000
39,965 (4)
54,000
Total ($)
All Other
Compensation
($)
100,000 (5) 2,539,704
82,465
93,965
74,965
99,965
82,465
79,965
87,965
93,965
-
-
-
-
-
-
-
-
(1)
The amount in this column represents the aggregate grant date fair value of the award as computed in accordance with Financial Accounting
Standard Board Accounting Standards Codification Topic 718. The assumptions used in calculating the grant date fair value of the award reported
in this column are set forth in Note 12 to our audited consolidated financial statements appearing elsewhere in this Annual Report on Form 10-K.
103
(2)
(3)
(4)
(5)
As of December 31, 2019, our non-employee directors held options to purchase shares of our common stock as follows: Dr. Zarur: 130,000 shares;
Mr. Arnold: 20,000 shares; Dr. Freed: 30,000 shares; Mr. Huffines: 20,000 shares; Dr. Koppel: 20,000 shares; Dr. Nagendran: 30,000 shares; Mr.
Shah: 20,000 shares; Mr. Stone: 20,000 shares; and Ms. Sullivan: 20,000 shares.
Consists of: (i) in respect of his services as a consultant to us for the year ended December 31, 2019, an option to purchase 10,000 shares of our
common stock granted on January 2, 2019 that vested in its entirety on the first anniversary of the grant and (ii) in respect of his services as a
consultant to us for the three years ended December 31, 2018, an option to purchase 100,000 shares of our common stock granted on January 2,
2019 which is subject to vesting in equal annual installments over three years from the vesting start date of January 2, 2020 through and including
January 2, 2023. The compensation arrangements we have with Dr. Zarur for his services to us as a consultant are described below in Item 13,
“Certain Relationships and Related Transactions, and Director Independence—Other arrangements.”
Consists of an option to purchase 10,000 shares of our common stock granted on June 13, 2019.
Consists of payments made to Dr. Zarur in consideration for his consulting services provided to the company.
Compensation committee interlocks and insider participation
None of the current members of our compensation committee has ever been an executive officer or employee of ours. None of our executive
officers currently serves, or has served during the last completed fiscal year, on the compensation committee or board of directors of any other entity that
has one or more executive officers serving as a member of our board of directors or compensation committee.
Item 12.
Security Ownership of Certain Beneficial Owners and Management and Related Stockholder Matters.
Security Ownership of Certain Beneficial Owners and Management
The following table sets forth information regarding the beneficial ownership of our common stock as of February 15, 2020 by (i) each person
whom we know to beneficially own more than 5% of our outstanding common stock, or a 5% stockholder, (ii) each director, (iii) each Named Executive
Officer and (iv) all current directors and executive officers as a group. Unless otherwise indicated, the address of each executive officer and director is c/o
Solid Biosciences, 141 Portland Street, Fifth Floor, Cambridge, MA 02139.
The number of shares of common stock “beneficially owned” by each stockholder is determined under rules issued by the SEC regarding the
beneficial ownership of securities. This information is not necessarily indicative of beneficial ownership for any other purpose. Under these rules,
beneficial ownership of shares of our common stock includes (1) any shares as to which the person or entity has sole or shared voting power or investment
power and (2) any shares as to which the person or entity has the right to acquire beneficial ownership within 60 days after February 15, 2020. The
percentage of beneficial ownership in the table below is based on 46,068,049 shares of common stock deemed to be outstanding as of February 15, 2020.
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Unless otherwise indicated below, and subject to community property laws where applicable, to our knowledge, all persons named in the table have
sole voting and investment power with respect to their shares of common stock.
b
5% Stockholders:
Perceptive Life Sciences Master Fund LTD (1)
Entities affiliated with RA Capital Management, L.P. (2)
Gilad Hayeem (3)
BCLS SB Investco, LP (4)
Boxer Capital, LLC (5)
Named Executive Officers and Directors:
Ilan Ganot (6)
Alvaro Amorrortu (7)
Jorge Quiroz, M.D. (8)
Andrey Zarur, Ph.D. (9)
Matthew Arnold (10)
Martin Freed, M.D., F.A.C.P. (11)
Robert Huffines (12)
Adam Koppel, M.D., Ph.D. (13)
Sukumar Nagendran, M.D. (14)
Rajeev Shah (15)
Adam Stone (16)
Lynne Sullivan (17)
All current directors and executive officers as a group
(14 persons) (18)
Number of
Shares
Beneficially
Owned
Percentage of
Shares
Beneficially
Owned
6,749,803
4,571,164
4,441,972
3,871,164
3,379,269
1,504,482
236,074
266,622
746,294
3,510,997
60,429
10,000
3,881,164
40,924
4,581,164
10,000
10,000
14.65%
9.92%
9.64%
8.40%
6.99%
3.26%
*
*
1.62%
7.62%
*
*
8.42%
*
9.94%
*
*
15,410,390
33.12%
*
(1)
(2)
(3)
Less than one percent.
Consists of shares held by Perceptive Life Sciences Master Fund LTD, or the Master Fund. Perceptive Advisors LLC is the investment manager to
Master Fund and may be deemed to beneficially own the securities directly held by the Master Fund. Joseph Edelman is the managing member of
Perceptive Advisors LLC. Perceptive Advisors LLC and Mr. Edelman may be deemed to beneficially own the shares held by the Master Fund. The
address of Perceptive is 51 Astor Place, 10th Floor, New York, NY 10003. Perceptive reports that it holds shared voting power and shared
dispositive power with respect to all shares held by it. Based on information set forth in a Schedule 13G/A filed with the SEC on February 14, 2020.
Consists of (a) 2,962,610 shares held by RA Capital Healthcare Fund, L.P. (“RA Capital Fund”), (b) 608,554 shares held by Blackwell Partners
LLC—Series A (“Blackwell”) and (c) 1,000,000 shares that RA Capital Fund and Blackwell purchased in connection with the closing of our initial
public offering. RA Capital Management, L.P. (“RA Capital”) is the investment manager for RA Capital fund and Blackwell. The general partner of
RA Capital Management, L.P. is RA Capital Management GP, LLC, of which Dr. Peter Kolchinsky and Mr. Shah are the managing members.
Investment decisions with respect to the shares held by RA Capital Fund and Blackwell are made by a portfolio management team at RA Capital of
which Rajeev Shah, a member of our board of directors, is a member. RA Capital Management, L.P., RA Capital Management GP, LLC, Dr.
Kolchinsky and Mr. Shah may be deemed indirect beneficial owners of the shares held by RA Capital Healthcare Fund, L.P. and Blackwell Partners
LLC – Series A. RA Capital Management, L.P., RA Capital Management GP, LLC, Dr. Kolchinsky and Mr. Shah expressly disclaim beneficial
ownership over all shares held by RA Capital Healthcare Fund, L.P. and Blackwell Partners LLC – Series A, except to the extent of their pecuniary
interest therein, and disclaim any pecuniary interest in the shares held by Blackwell Partners LLC – Series A. The address for each of RA Capital
Fund, Blackwell, and RA Capital is c/o 200 Berkeley Street, 18th Floor, Boston, MA 02116. Entities affiliated with RA Capital report that they hold
shared voting power and shared dispositive power with respect to all shares held by them.
All shares are held by DTMG Bermuda Limited (“DTMG”), which is owned and controlled by Gilad Hayeem. Mr. Hayeem and DTMG report that
they hold shared voting power and shared dispositive power with respect to all shares held. The address for Mr. Hayeem and DTMG is c/o Hunton
Andrews Kurth LLP, Attn: Eric Markus, 2200 Pennsylvania Avenue, NW, Washington, DC 20037. In connection with estate planning activities, Mr.
Hayeem sold units of the Company equivalent to approximately 353,050 shares to a sub-trust of an employee-benefit trust
105
established by a former employer of Mr. Hayeem. Such sub-trust has as its beneficiaries Mr. Hayeem and his family. Because Mr. Hayeem does not
exercise investment or voting control of the shares held by such sub-trust, such shares do not appear in the table above. Based on information set
forth in a Schedule 13G/A filed with the SEC on February 14, 2020 and on information provided to us by Mr. Hayeem
Consists of shares held by BCLS SB Investco, LP (“BCLS”). The governance, investment strategy and decision-making process with respect to
investments held by BCLS is directed by Bain Capital Life Sciences Investors, LLC, whose managers are Jeffrey Schwartz and Adam Koppel, a
member of our board of directors. As a result, each of Bain Capital Life Sciences Investors, LLC, Mr. Schwartz and Dr. Koppel may be deemed to
share voting and dispositive power over the shares held by BCLS. The address of BCLS is c/o Bain Capital Life Sciences, LP, 200 Clarendon
Street, Boston, Massachusetts 02116.
Consists of (a) 1,031,872 shares held by Boxer Capital, LLC (“Boxer Capital”) for which Boxer Capital, Boxer Asset Management Inc. (“Boxer
Management”) and Joe Lewis hold shared voting power and shared dispositive power, (b) 2,158,329 shares of common stock which may be
acquired upon the exercise of warrants held by Boxer Capital within 60 days of February 15, 2020 for which Boxer Capital, Boxer Management and
Joe Lewis hold shared voting power and shared dispositive power, (c) 11,488 shares held by Braslyn Ltd. (“Braslyn”) for which Braslyn and Joe
Lewis hold shared voting power and shared dispositive power, (d) 40,210 shares held by MVA Investors, LLC (“MVA Investors”) for which MVA
Investors and Aaron Davis hold shared voting power and shared dispositive power and (e) 137,370 shares of common stock which may be acquired
upon the exercise of warrants held by MVA Investors within 60 days of February 15, 2020 for which MVA Investors and Aaron Davis hold shared
voting power and shared dispositive power. Boxer Management is the managing member and majority owner of Boxer Capital. Joe Lewis is the
sole indirect beneficial owner of and controls Boxer Management and Braslyn. MVA Investors is the independent, personal investment vehicle of
certain employees of Boxer Capital. Aaron Davis is a member of and has voting and dispositive power over securities held by MVA Investors. The
address of Boxer Capital, MVA Investors, LLC and Aaron Davis is 11682 El Camino Real, Suite 320, San Diego, CA 92130. The address of Boxer
Management, Braslyn and Joe Lewis is Cay House, EP Taylor Drive N7776, Lyford Cay, New Providence, Bahamas. Based on information set
forth in a Schedule 13G/A filed with the SEC on February 14, 2020.
Consists of (a) 1,063,000 shares held by Mr. Ganot as an individual, (b) 60,631 shares held by Mr. Ganot and Ms. Ganot as joint tenants with right
of survivorship, (c) 290,914 shares held by Mr. Adam Ganot and Ms. Ganot, as trustees for the Ilan Ganot 2017 Irrevocable Trust, (d) 87,000 shares
of common stock underlying options held by Mr.Ganot that are exercisable as of February 15, 2020 or will become exercisable within 60 days after
such date and (e) 2,937 shares held by Mr. Ganot’s wife.
Consists of (a) 153,746 shares of common stock owned by Mr. Amorrortu and (b) 82,238 shares of common stock underlying options held by Mr.
Amorrortu that are exercisable as of February 15, 2020 or will become exercisable within 60 days after such date.
Consists of (a) 228,092 shares of common stock owned by Dr. Quiroz and (b) 38,530 shares of common stock underlying options held by Dr.
Quiroz that are exercisable as of February 15, 2020 or will become exercisable within 60 days after such date.
Consists of (a) 691,105 shares held by Mr. Zarur as an individual, (b) 45,000 shares of common stock underlying options held by Mr. Zarur that are
exercisable as of February 15, 2020 or will become exercisable within 60 days after such date and (c) 10,189 shares held by Mr. Zarur’s wife.
Consists of (a) 3,500,997 shares of common stock owned by Mr. Arnold and (b) 10,000 shares of common stock underlying options held by Mr.
Arnold that are exercisable as of February 15, 2020 or will become exercisable within 60 days after such date.
Consists of (a) 53,763 shares of common stock owned by Dr. Freed and (b) 6,666 shares of common stock underlying options held by Dr. Freed that
are exercisable as of February 15, 2020 or will become exercisable within 60 days after such date.
Consists of 10,000 shares of common stock underlying options held by Mr. Huffines that are exercisable as of February 15, 2020 or will become
exercisable within 60 days after such date.
Consists of shares held by BCLS. Dr. Koppel is a manager of Bain Capital Life Sciences Investors, LLC and as a result, by virtue of the
relationships described in footnote (4) above, may be deemed to share beneficial ownership of the shares held by BCLS. The address of Dr. Koppel
is c/o Bain Capital Life Sciences, LP, 200 Clarendon Street, Boston, Massachusetts 02116. In addition, the amount consists of 10,000 shares of
common stock underlying options held by Dr. Koppel that are exercisable as of February 15, 2020 or will become exercisable within 60 days after
such date.
Consists of (a) 34,258 shares of common stock owned by Dr. Nagendran and (b) 6,666 shares of common stock underlying options held by Dr.
Nagendran that are exercisable as of February 15, 2020 or will become exercisable within 60 days after such date.
(4)
(5)
(6)
(7)
(8)
(9)
(10)
(11)
(12)
(13)
(14)
106
(15)
Consists of shares held by RA Capital as described in Footnote (2) above. Mr. Shah disclaims beneficial ownership of all shares held by RA Capital
Fund and Blackwell, except to the extent of his pecuniary interest therein, and disclaims any pecuniary interest in the shares held by Blackwell
Partners LLC – Series A. The address for each of RA Capital Fund, Blackwell, and RA Capital is c/o 200 Berkeley Street, 18th Floor, Boston, MA
02116. Entities affiliated with RA Capital report that they hold shared voting power and shared dispositive power with respect to all shares held by
them. In addition, the amount consists of 10,000 shares of common stock underlying options held by Mr. Shah that are exercisable as of February
15, 2020 or will become exercisable within 60 days after such date.
(16) Mr. Stone is Chief Investment Officer of Perceptive Advisors LLC. Mr. Stone disclaims beneficial ownership of the shares held by Perceptive. The
address of Mr. Stone is 51 Astor Place, 10th Floor, New York, NY 10003. In addition, the amount consists of 10,000 shares of common stock
underlying options held by Mr. Stone that are exercisable as of February 15, 2020 or will become exercisable within 60 days after such date.
Consists of 10,000 shares of common stock underlying options held by Ms. Sullivan that are exercisable as of February 15, 2020 or will become
exercisable within 60 days after such date.
Includes 571,882 shares of common stock underlying options that are exercisable as of February 15, 2020 or will become exercisable within 60
days after such date.
(18)
(17)
Securities Authorized for Issuance under Equity Compensation Plans
The following table provides information about our equity compensation plans as of December 31, 2019.
Plan Category
Equity compensation plans approved by security
holders (1)
Equity compensation plans not approved by
security holders
Total
(a)
Number of
securities
to be issued upon
the exercise of
outstanding
options,
warrants and
rights
(b)
Weighted-
average
exercise price
of outstanding
options,
warrants
and rights (2)
©
Number of securities
remaining available
for future issuance
under equity
compensation plans
(excluding securities
reflected in column
(a))
2,856,075 $
22.18
2,144,925
-
2,856,075 $
-
22.18
-
2,144,925
(1)
(2)
Reflects shares issuable upon exercise of options and settlement of RSUs.
The weighted-average exercise price does not include RSUs, which have no exercise price.
Item 13.
Certain Relationships and Related Transactions, and Director Independence.
In addition to the executive officer and director compensation arrangements discussed above under “Compensation of our executive officers and
directors,” we describe transactions since January 1, 2018 to which we have been or will be a participant, in which the amount involved in the transaction
exceeds the lesser of $120,000 or 1% of our total assets at year end for each of the last two completed fiscal years and in which any of our directors,
executive officers or beneficial holders of more than 5% of any class of our capital stock, or 5% Security Holders, or any immediate family member of, or
person sharing the household with, any of these individuals, had or will have a direct or indirect material interest.
Limited liability company agreement of Solid Biosciences, LLC
Solid Biosciences, LLC was party to a limited liability company agreement, or the LLC Agreement, with its members that terminated upon the
Corporate Conversion. Under the terms of the LLC Agreement, Series A Common Unit holders were entitled to designate two individuals to serve on our
board of managers. Pursuant to this provision, the two board appointees were Mssrs. Arnold and Huffines. Mr. Huffines is an employee of J.P. Morgan
Securities LLC, a participating underwriter in our initial public offering. An affiliate of J.P. Morgan Securities LLC owned in excess of 10% of our issued
and outstanding common stock immediately prior to our initial public offering. See “Underwriting—Conflicts of Interest” in our prospectus filed with the
SEC filed on January 29, 2018 for a description of services that the underwriters provided to us in connection with our initial public offering.
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Corporate conversion
In connection with our Corporate Conversion, Solid Biosciences, LLC unitholders received 26,498,559 shares of common stock for all units held
immediately prior to the Corporate Conversion. The existing units held by our executive officers, directors and 5% Security Holders were converted on the
same basis as all other holders of such units.
As result of the Corporate Conversion, the holders of Series A Common Units of Solid Biosciences, LLC became holders of shares of our common
stock.
Amended and restated registration rights agreement
We are party to an Amended and Restated Registration Rights Agreement, or the Registration Rights Agreement, dated March 29, 2017, with
certain of our stockholders, or the Investors, which includes our 5% Security Holders and entities affiliated with certain of our directors. The Registration
Rights Agreement provides the Investors the right, subject to certain conditions, to demand that we file a registration statement or to request that their
shares be covered by a registration statement that we are otherwise filing.
Participation in initial public offering
In our initial public offering, certain of our 5% stockholders and their affiliates purchased an aggregate of 2,300,000 shares of our common stock.
Each of those purchases was made through the underwriters at the initial public offering price of $16.00 per share. The following table sets forth the
aggregate number of shares of our common stock that these 5% stockholders and their affiliates purchased in our initial public offering:
Purchaser (1)
RA Capital Management L.P.
Perceptive Advisors LLC
BCLS SB Investco, LP
Shares of
common stock
1,000,000 $
1,000,000 $
300,000 $
Total
purchase price
16,000,000
16,000,000
4,800,000
(1)
See “Item 12. Security Ownership of Certain Beneficial Owners and Management and Related Stockholder Matters” for more information about the
shares held by the below identified entities.
108
Private placement
On July 25, 2019, we entered into a definitive agreement with respect to the private placement of (i) 10,607,525 shares of our common stock at a
price per share of $4.65 and (ii) 2,295,699 pre-funded warrants to purchase shares of our common stock at a price per warrant of $4.64, to a group of
accredited investors. Each pre-funded warrant is exercisable for one share of common stock at an exercise price of $0.01 and the pre-funded warrants have
no expiration date. We completed this private placement on July 30, 2019, resulting in approximately $60.0 million in gross proceeds to us, before
deducting offering costs of $2.1 million. The number of shares that each of our directors, executive officers and holders of more than 5% of our voting
securities purchased and the aggregate purchase price paid for such shares is set forth in the table below.
Name
DTMG Bermuda Limited
Perceptive Life Sciences Master
Fund, Ltd.
BCLS SB Investco, LP
RA Capital Healthcare Fund, L.P.
Blackwell Partners LLC - Series A
Matthew B. Arnold
Martin I. Freed
Sukumar Nagendran
Ilan Ganot
Carl Morris
MVA Investors, LLC
Boxer Capital, LLC
Number of
Shares of
Common
Stock
Purchased
903,226
2,822,581
1,881,720
1,593,629
288,091
677,419
53,763
32,258
21,505
21,505
Number of
Warrant
Shares
Underlying
Pre-Funded
Warrants
Purchased
Purchase Price
$ 4,200,000.90
$ 13,125,001.65
$ 8,749,998.00
$ 7,410,374.85
$ 1,339,623.15
$ 3,149,998.35
249,997.95
$
149,999.70
$
99,998.25
$
99,998.25
$
637,396.80
137,370$
2,158,329$ 10,014,646.56
Other arrangements
We employ Annie Ganot, one of our Co-Founders and the wife of Ilan Ganot, as Director, Patient Advocacy. Mr. Ganot is our CEO and a member
of our board of directors. Ms. Ganot receives an annual salary and bonus payments of less than $200,000.
In respect of his services as a consultant to us for the three years ended December 31, 2018, on January 2, 2019, we granted Dr. Zarur an option to
purchase 100,000 shares of our common stock. In respect of his services as a consultant to us for the year ended December 31, 2019, (i) on January 2,
2019, we granted Dr. Zarur an option to purchase 10,000 shares of our common stock, and (ii) we paid him $100,000. In respect of his services as a
consultant to us for the year ending December 31, 2020, (i) on January 2, 2020, we granted Dr. Zarur an option to purchase 10,000 shares of our common
stock, and (ii) we will pay him $100,000.
In connection with the termination of his employment, we entered into a consulting agreement with Dr. Quiroz, effective as of January 15, 2020,
pursuant to which Dr. Quiroz will assist with the transition of his duties to our executive management team. Dr. Quiroz will be compensated at a rate of
$500 per hour for his services under the consulting agreement. The term of the consulting agreement will continue until July 15, 2020. Either we or Dr.
Quiroz will be able to terminate the consulting agreement at any time, with or without cause (as defined therein).
In connection with the termination of his employment, we entered into a consulting agreement with Mr. Amorrortu, effective as of January 15,
2020, pursuant to which Mr. Amorrortu will assist with the transition of his duties to our executive management team. Mr. Amorrortu will be compensated
at a rate of $500 per hour for his services under the consulting agreement. The term of the consulting agreement will continue until July 15, 2020. Either we
or Mr. Amorrortu will be able to terminate the consulting agreement at any time, with or without cause (as defined therein).
109
Indemnification agreements
We entered into agreements to indemnify our directors and executive officers in connection with our initial public offering. These agreements
require us, among other things, to indemnify these individuals for certain expenses (including attorneys’ fees), judgments, fines and settlement amounts
reasonably incurred by such persons in any action or proceeding, including any action by or in our right, on account of any services undertaken by any such
person on behalf of our company or that person’s status as a member of our board of directors to the maximum extent allowed under Delaware law.
Policy for approval of related-person transactions
We have adopted a written related-person transaction policy that sets forth our procedures for the identification, review, consideration and approval
or ratification of any transaction, arrangement or relationship in which we are a participant, the amount involved exceeds $120,000 and one of our
executive officers, directors, director nominees or 5% stockholders (or their immediate family members), each of whom we refer to as a “related person,”
has a direct or indirect material interest.
If a related person proposes to enter into such a transaction, arrangement or relationship, which we refer to as a “related-person transaction,” the
related person must report the proposed related-person transaction to our general counsel. The policy calls for the proposed related-person transaction to be
reviewed by and if deemed appropriate approved by, the audit committee of our board of directors. Whenever practicable, the reporting, review and
approval will occur prior to entry into the transaction. If advance review and approval is not practicable, the audit committee will review and, in its
discretion, may ratify the related-person transaction. The policy also permits the chair of the audit committee to review, and if deemed appropriate approve,
proposed related-person transactions that arise between audit committee meetings, subject to ratification by the audit committee at its next meeting. Any
related-person transactions that are ongoing in nature will be reviewed annually.
A related-person transaction reviewed under the policy will be considered approved or ratified if it is authorized by the audit committee after full
disclosure of the related person’s interest in the transaction. As appropriate for the circumstances, the committee will review and consider:
•
•
•
•
•
•
•
the related person’s interest in the related-person transaction;
the approximate dollar amount involved in the related-person transaction;
the approximate dollar amount of the related person’s interest in the transaction without regard to the amount of any profit or loss;
whether the transaction was undertaken in the ordinary course of our business;
whether the terms of the transaction are no less favorable to us than terms that could have been reached with an unrelated third party;
the purpose of, and the potential benefits to us of, the related-person transaction; and
any other information regarding the related-person transaction or the related person in the context of the proposed transaction that would be
material to investors in light of the circumstances of the particular transaction.
The audit committee may approve or ratify the transaction only if the audit committee determines that, under all of the circumstances, the
transaction is not inconsistent with our best interests. The audit committee may impose any conditions on the related-person transaction that it deems
appropriate.
The policy provides that transactions involving compensation of executive officers will be reviewed and approved by the compensation committee
of our board of directors in the manner specified in its charter.
Director independence
Applicable Nasdaq rules require a majority of a listed company’s board of directors to be comprised of independent directors within one year of
listing. In addition, Nasdaq rules require that, subject to specified exceptions, each member of a listed company’s audit, compensation and nominating and
corporate governance committees be independent under the Exchange Act. Audit committee members also satisfy independence criteria set forth in
Rule 10A-3 under the Exchange Act and compensation committee members must also satisfy the independence criteria set forth in Rule 10C-1 under the
Exchange Act. The Nasdaq independence definition includes a series of objective tests, such as that the director is not, and has not been for at least three
years, one of our employees and that neither the director nor any of his or her family members
110
has engaged in various types of business dealings with us. In addition, under applicable Nasdaq rules, a director will only qualify as an “independent
director” if, in the opinion of the listed company’s board of directors, that person does not have a relationship that would interfere with the exercise of
independent judgment in carrying out the responsibilities of a director. In order to be considered independent for purposes of Rule 10A-3, a member of an
audit committee of a listed company may not, other than in his or her capacity as a member of the audit committee, the board of directors, or any other
board committee, accept, directly or indirectly, any consulting, advisory, or other compensatory fee from the listed company or any of its subsidiaries or
otherwise be an affiliated person of the listed company or any of its subsidiaries. In order to be considered independent for purposes of Rule 10C-1, the
board must consider, for each member of a compensation committee of a listed company, all factors specifically relevant to determining whether a director
has a relationship to such company which is material to that director’s ability to be independent from management in connection with the duties of a
compensation committee member, including, but not limited to: (1) the source of compensation of the director, including any consulting advisory or other
compensatory fee paid by such company to the director; and (2) whether the director is affiliated with the company or any of its subsidiaries or affiliates.
Our board of directors has determined that all members of the board of directors, except Ilan Ganot and Andrey Zarur, are independent directors, as
defined under applicable Nasdaq rules. In making such determination, our board of directors considered the relationships that each such non-employee
director has with our Company and all other facts and circumstances that our board of directors deemed relevant in determining his or her independence,
including the beneficial ownership of our common stock by each non-employee director.
Our board of directors has determined that the composition of our committees currently complies with all applicable independence requirements of
Nasdaq and the rules and regulations of the SEC.
Item 14.
Principal Accounting Fees and Services.
The following table represents aggregate fees billed to us by PricewaterhouseCoopers LLP, or PwC, for the fiscal years ended December 31, 2019
and 2018:
Audit fees
Audit-related fees
Tax fees
All other fees
Total
2019
2018
$
$
646,500 $
-
20,148
2,756
669,404 $
698,300
20,000
75,158
2,756
796,214
The services rendered by PwC in connection with the fees presented above were as follows:
Audit Fees
Audit fees consist of amounts for professional services rendered for audit and quarterly reviews of our 2019 and 2018 financial statements as well
as review of our registration statements on Form S-3 and Form S-1 in 2019 and 2018, respectively.
Audit-Related Fees
Audit-related fees consist of amounts for professional services rendered related to the implementation of an accounting standard in the year prior to
its adoption.
Tax Fees
Tax fees consist of fees for professional services related to tax compliance and consultations.
All other fees
All other fees include license fees for web-based accounting research tools.
Pre-approval policies
The audit committee has not adopted policies and procedures for the pre-approval of audit and non-audit services rendered by our independent
registered public accounting firm and, consequently, all audit and non-audit services are pre-approved by the whole audit committee or the chair of the
audit committee.
111
Item 15.
Exhibits, Financial Statement Schedules.
(1) Financial Statements:
PART IV
Report of Independent Registered Public Accounting Firm
Consolidated Balance Sheets at December 31, 2019 and 2018
Consolidated Statements of Operations for the Years Ended December 31, 2019, 2018 and 2017
Consolidated Statements of Comprehensive Loss for the Years Ended December 31, 2019, 2018 and 2017
Consolidated Statements of Preferred Units and Stockholders’/Members’ Equity/(Deficit) for the Years Ended December 31, 2019, 2018 and
2017
Consolidated Statements of Cash Flows for the Years Ended December 31, 2019, 2018 and 2017
Notes to Consolidated Financial Statements
Page
F-1
F-2
F-3
F-4
F-5
F-6
F-7
(2) Financial Statement Schedules:
All financial statement schedules have been omitted because they are not applicable, not required or the information required is shown in the
financial statements or the notes thereto.
(3) Exhibits. The following is a list of exhibits filed as part of this Annual Report on Form 10-K.
112
Exhibit
Number
2.1
2.2
2.3
3.1
3.2
4.1
4.2*
10.1†
10.2†
10.3†
10.4†
10.5†
10.6†
10.7†
10.8†
10.9†
10.10#
10.11#
10.12#
10.13#
Description
Agreement and Plan of Merger, dated March 29, 2017, by and between Solid Biosciences, LLC and Solid GT, LLC (incorporated by
reference to Exhibit 2.1 to the Registration Statement on Form S-1 filed on December 29, 2017).
Plan of Conversion, dated January 25, 2018 (incorporated by reference to Exhibit 2.2 to the Annual Report on Form 10-K filed on March 29,
2018).
Agreement and Plan of Merger, dated January 25, 2018, by and among Solid Biosciences Inc., Bain Capital Life Sciences Fund, L.P., BCIP
Life Sciences Associates, LP, BCLS Solid Bio, Inc., Foresite Capital Fund III, L.P. and FC Fund III Solid Holdings, Inc. (incorporated by
reference to Exhibit 2.3 to the Annual Report on Form 10-K filed on March 29, 2018).
Certificate of Incorporation of Solid Biosciences Inc. (incorporated by reference to Exhibit 4.1 to the Registration Statement on Form S-8
filed on January 29, 2018).
Bylaws of Solid Biosciences Inc. (incorporated by reference to Exhibit 4.2 to the Registration Statement on Form S-8 filed on January 29,
2018).
Specimen Common Stock Certificate (incorporated by reference to Exhibit 4.1 to the Registration Statement on Form S-1 filed on December
29, 2017).
Description of the Company’s Securities Registered under Section 12 of the Exchange Act.
Amended and Restated Registration Rights Agreement dated March 29, 2017 by and among Solid Biosciences, LLC and certain investors
(incorporated by reference to Exhibit 10.17 to the Registration Statement on Form S-1 filed on December 29, 2017).
Employment Agreement, dated as of January 25, 2019, by and between Solid Biosciences Inc. and Ilan Ganot. (incorporated by reference to
Exhibit 10.2 to the Annual Report on Form 10-K filed on March 13, 2019)
Employment Agreement, dated as of January 25, 2019, by and between Solid Biosciences Inc. and Alvaro Amorrortu (incorporated by
reference to Exhibit 10.3 to the Annual Report on Form 10-K filed on March 13, 2019)
Employment Agreement, dated as of January 25, 2019, by and between Solid Biosciences Inc. and Carl Morris. Ph.D. (incorporated by
reference to Exhibit 10.4 to the Annual Report on Form 10-K filed on March 13, 2019).
Solid Biosciences, LLC Amended and Restated Equity Incentive Plan and form of unit restriction agreement (incorporated by reference to
Exhibit 10.7 to the Annual Report on Form 10-K filed on March 29, 2018).
Solid Biosciences Inc. 2018 Omnibus Incentive Plan (incorporated by reference to Exhibit 99.1 to the Registration Statement on Form S-8
filed on January 29, 2018).
Form of Incentive Stock Option Agreement under 2018 Omnibus Incentive Plan. (incorporated by reference to Exhibit 10.7 to the Annual
Report on Form 10-K filed on March 13, 2019).
Form of Nonqualified Stock Option Agreement under 2018 Omnibus Incentive Plan. (incorporated by reference to Exhibit 10.8 to the
Annual Report on Form 10-K filed on March 13, 2019).
Form of Restricted Stock Agreement under 2018 Omnibus Incentive Plan (incorporated by reference to Exhibit 10.9 to the Registration
Statement on Form S-1 filed on December 29, 2017).
Exclusive Patent License Agreement, dated as of October 16, 2015, by and between Solid GT, LLC and the University of Washington
(incorporated by reference to Exhibit 10.10 to the Registration Statement on Form S-1 filed on December 29, 2017).
Patent License Agreement, dated as of March 10, 2016, by and between Solid GT, LLC and the Regents of the University of Michigan
(incorporated by reference to Exhibit 10.11 to Amendment No. 1 to the Registration Statement on Form S-1 filed on January 16, 2018).
License Agreement, dated as of October 15, 2015, by and between Solid GT, LLC and The Curators of the University of Missouri
(incorporated by reference to Exhibit 10.12 to Amendment No. 1 to the Registration Statement on Form S-1 filed on January 16, 2018).
Cell Line License Agreement, dated as of November 20, 2016, by and between Solid Biosciences, LLC and Life Technologies Corporation
(incorporated by reference to Exhibit 10.13 to Amendment No. 1 to the Registration Statement on Form S-1 filed on January 16, 2018).
113
10.14#
10.15#
10.16
10.17
10.18
10.19
10.20†
10.21
10.22
10.23
10.24*†
10.25*†
10.26*†
10.27*†
21.1*
23.1*
31.1*
31.2*
32.1**
32.2**
License Agreement, dated as of June 23, 2016, by and between Solid GT, LLC and the President and Fellows of Harvard College
(incorporated by reference to Exhibit 10.14 to the Registration Statement on Form S-1 filed on December 29, 2017).
License Agreement, dated as of August 3, 2017, by and between Solid Biosciences, LLC and the President and Fellows of Harvard College
(incorporated by reference to Exhibit 10.15 to the Registration Statement on Form S-1 filed on December 29, 2017).
First Amendment to Patent License Agreement, dated as of March 15, 2017, by and between Solid GT, LLC and the Regents of the
University of Michigan (incorporated by reference to Exhibit 10.16 to Amendment No. 1 to the Registration Statement on Form S-1 filed on
January 16, 2018).
Form of Indemnification Agreement for Directors and Officers (incorporated by reference to Exhibit 10.16 to the Registration Statement on
Form S-1 filed on December 29, 2017).
Sublease, dated as of January 30, 2018, by and between Solid Biosciences, LLC and Twitter, Inc. (incorporated by reference to Exhibit 10.20
to the Annual Report on Form 10-K filed on March 29, 2018).
Lease Agreement, dated as of December 22, 2017, by and between Solid Biosciences, LLC and ARE-MA Region No. 59, LLC (incorporated
by reference to Exhibit 10.21 to the Annual Report on Form 10-K filed on March 29, 2018).
Summary of Non-Employee Director Compensation Program. (incorporated by reference to Exhibit 10.20 to the Annual Report on Form 10-
K filed on March 13, 2019).
Securities Purchase Agreement, dated July 25, 2019, by and among the Company and the other parties thereto (incorporated by reference to
Exhibit 10.1 to the Current Report on Form 8-K filed on July 26, 2019).
Form of Pre-Funded Warrant to Purchase Common Stock to be issued pursuant to the Securities Purchase Agreement (incorporated by
reference to Exhibit 10.2 to the Current Report on Form 8-K filed on July 26, 2019).
Registration Rights Agreement, dated July 25, 2019, by and among the Company and the other parties thereto (incorporated by reference to
Exhibit 10.3 to the Current Report on Form 8-K filed on July 26, 2019).
Consulting Agreement, dated as of January 1, 2020, by and between Solid Biosciences Inc. and Andrey Zarur.
Consulting Agreement, dated as of January 15, 2020, by and between Solid Biosciences Inc. and Alvaro Amorrortu.
Consulting Agreement, dated as of January 15, 2020 by and between Solid Biosciences Inc. and Jorge Quiroz.
Employment Agreement, dated as of January 25, 2019, by and between Solid Biosciences Inc. and Jorge Quiroz.
Subsidiaries of Solid Biosciences Inc.
Consent of PricewaterhouseCoopers LLP, Independent Registered Public Accounting Firm.
Certification of Chief Executive Officer of the Registrant Pursuant to Section 302 of the Sarbanes-Oxley Act of 2002.
Certification of Chief Financial Officer of the Registrant Pursuant to Section 302 of the Sarbanes-Oxley Act of 2002.
Certification of Chief Executive Officer of the Registrant Pursuant to 18 U.S.C. Section 1350, as adopted pursuant to Section 906 of the
Sarbanes-Oxley Act of 2002.
Certification of Chief Financial Officer of the Registrant Pursuant to 18 U.S.C. Section 1350, as adopted pursuant to Section 906 of the
Sarbanes-Oxley Act of 2002.
*
**
†
#
Filed herewith.
Furnished herewith.
Indicates management contract or compensatory plan.
Confidential treatment has been granted as to certain portions, which portions the Registrant has omitted and filed separately with the SEC.
Item 16.
Form 10-K Summary.
Not applicable.
114
Pursuant to the requirements of Section 13 or 15(d) of the Securities Exchange Act of 1934, as amended, the Registrant has duly caused this Report
to be signed on its behalf by the undersigned, thereunto duly authorized.
SIGNATURES
Date: March 12, 2020
SOLID BIOSCIENCES INC.
By:
/s/ Ilan Ganot
Ilan Ganot
President, Chief Executive Officer and Director
(Principal Executive Officer)
Pursuant to the requirements of the Securities Exchange Act of 1934, as amended, this Report has been signed below by the following persons on
behalf of the Registrant in the capacities and on the dates indicated.
/s/ Ilan Ganot
Ilan Ganot
/s/ Jennifer Ziolkowski
Jennifer Ziolkowski
/s/ Andrey Zarur, Ph.D.
Andrey Zarur, Ph.D.
/s/ Matthew Arnold
Matthew Arnold
/s/ Martin Freed
Martin Freed
/s/ Robert Huffines
Robert Huffines
/s/ Adam Koppel
Adam Koppel, M.D., Ph.D.
/s/ Sukumar Nagendran
Sukumar Nagendran
/s/ Rajeev Shah
Rajeev Shah
/s/ Adam Stone
Adam Stone
/s/ Lynne Sullivan
Lynne Sullivan
President, Chief Executive Officer and Director
(Principal Executive Officer)
Chief Financial Officer
(Principal Financial and Accounting Officer)
March 12, 2020
March 12, 2020
Chairman of the Board
March 12, 2020
Director
Director
Director
Director
Director
Director
Director
Director
115
March 12, 2020
March 12, 2020
March 12, 2020
March 12, 2020
March 12, 2020
March 12, 2020
March 12, 2020
March 12, 2020
Report of Independent Registered Public Accounting Firm
To the Board of Directors and Stockholders of Solid Biosciences Inc.
Opinion on the Financial Statements
We have audited the accompanying consolidated balance sheets of Solid Biosciences Inc. and its subsidiaries (the “Company”) as of December 31, 2019
and 2018, and the related consolidated statements of operations, of comprehensive loss, of preferred units and stockholders’/members’ equity (deficit) and
of cash flows for each of the three years in the period ended December 31, 2019, including the related notes (collectively referred to as the “consolidated
financial statements”). In our opinion, the consolidated financial statements present fairly, in all material respects, the financial position of the Company as
of December 31, 2019 and 2018, and the results of its operations and its cash flows for each of the three years in the period ended December 31, 2019 in
conformity with accounting principles generally accepted in the United States of America.
Substantial Doubt About the Company’s Ability to Continue as a Going Concern
The accompanying consolidated financial statements have been prepared assuming that the Company will continue as a going concern. As discussed in
Note 1 to the consolidated financial statements, the Company has incurred losses and negative cash flows from operations since inception, that raise
substantial doubt about its ability to continue as a going concern. Management’s plans in regard to these matters are also described in Note 1. The
consolidated financial statements do not include any adjustments that might result from the outcome of this uncertainty.
Change in Accounting Principle
As discussed in Note 2 to the consolidated financial statements, the Company changed the manner in which it accounts for leases in 2019.
Basis for Opinion
These consolidated financial statements are the responsibility of the Company’s management. Our responsibility is to express an opinion on the Company’s
consolidated financial statements based on our audits. We are a public accounting firm registered with the Public Company Accounting Oversight Board
(United States) (PCAOB) and are required to be independent with respect to the Company in accordance with the U.S. federal securities laws and the
applicable rules and regulations of the Securities and Exchange Commission and the PCAOB.
We conducted our audits of these consolidated financial statements in accordance with the standards of the PCAOB. Those standards require that we plan
and perform the audit to obtain reasonable assurance about whether the consolidated financial statements are free of material misstatement, whether due to
error or fraud. The Company is not required to have, nor were we engaged to perform, an audit of its internal control over financial reporting. As part of our
audits we are required to obtain an understanding of internal control over financial reporting but not for the purpose of expressing an opinion on the
effectiveness of the Company's internal control over financial reporting. Accordingly, we express no such opinion.
Our audits included performing procedures to assess the risks of material misstatement of the consolidated financial statements, whether due to error or
fraud, and performing procedures that respond to those risks. Such procedures included examining, on a test basis, evidence regarding the amounts and
disclosures in the consolidated financial statements. Our audits also included evaluating the accounting principles used and significant estimates made by
management, as well as evaluating the overall presentation of the consolidated financial statements. We believe that our audits provide a reasonable basis
for our opinion.
/s/ PricewaterhouseCoopers LLP
Boston, Massachusetts
March 12, 2020
We have served as the Company’s auditor since 2017.
F-1
SOLID BIOSCIENCES INC.
CONSOLIDATED BALANCE SHEETS
(In thousands, except share and per share data)
Assets
Current assets:
Cash and cash equivalents
Available-for-sale securities
Prepaid expenses and other current assets
Total current assets
Operating lease, right of use asset
Property and equipment, net
Other non-current assets
Restricted cash
Total assets
Liabilities and Stockholders' Equity
Current liabilities:
Accounts payable
Accrued expenses
Operating lease liabilities
Finance lease liabilities
Other current liabilities
Total current liabilities
Operating lease liabilities, excluding current portion
Finance lease obligations, excluding current portion
Other non-current liabilities
Total liabilities
Commitments and Contingencies (Note 14)
Stockholders’ Equity:
Preferred stock, $0.001 par value; 10,000,000 shares authorized at December 31, 2019 and
December 31, 2018; no shares issued and outstanding at December 31, 2019 and December 31, 2018
Common stock, $0.001 par value; 300,000,000 shares authorized at December 31, 2019 and
December 31, 2018; 45,987,571 shares issued and outstanding at December 31, 2019 and 35,432,460
shares issued and outstanding at December 31, 2018; 2,295,699 pre-funded warrants outstanding at
December 31, 2019 and no pre-funded warrants outstanding at December 31, 2018
Additional Paid-in Capital
Accumulated other comprehensive gain (loss)
Accumulated deficit
Total stockholders' equity
Total liabilities and stockholders' equity
December 31,
2019
2018
$
$
$
76,043
7,481
2,778
86,302
4,988
11,645
209
327
103,471
7,124
9,178
1,736
186
52
18,276
4,414
733
-
23,423
86,366
36,098
6,175
128,639
-
10,422
209
327
139,597
3,691
8,235
-
173
382
12,481
-
859
1,074
14,414
-
-
48
396,278
1
(316,279)
80,048
103,471
$
35
324,209
(5)
(199,056)
125,183
139,597
$
$
$
$
The accompanying notes are an integral part of these consolidated financial statements.
F-2
SOLID BIOSCIENCES INC.
CONSOLIDATED STATEMENTS OF OPERATIONS
(In thousands, except share and per share data)
Revenue
Operating expenses:
Research and development
General and administrative
Total operating expenses
Loss from operations
Other income (expense):
Revaluation of preferred unit tranche right
Interest income
Other income
Total other income (expense), net
Net loss
Net loss attributable to non-controlling interest
Net loss attributable to Solid Biosciences Inc.
Accretion of preferred units to redemption value
Redemption of preferred units
Redemption of redeemable interest from non-controlling interest in
Solid GT
Net loss attributable to common stockholders
Net loss per share attributable to common stockholders, basic
and diluted
Weighted average common stock outstanding, basic and diluted
2019
Year Ended December 31,
2018
2017
$
- $
- $
-
94,737
24,581
119,318
(119,318)
-
1,580
515
2,095
(117,223) $
-
57,965
17,722
75,687
(75,687)
-
619
270
889
(74,798) $
-
(117,223) $
(74,798) $
-
-
-
-
-
-
(117,223) $
(74,798) $
39,905
14,952
54,857
(54,857)
459
219
1,001
1,679
(53,178)
(1,060)
(52,118)
(959)
15,685
(1,925)
(39,317)
(2.91) $
(2.25) $
(2.88)
40,289,290
33,262,597
13,649,485
$
$
$
$
The accompanying notes are an integral part of these consolidated financial statements.
F-3
SOLID BIOSCIENCES INC.
CONSOLIDATED STATEMENTS OF COMPREHENSIVE LOSS
(In thousands)
Net loss
Other comprehensive income (loss):
Unrealized gain (loss) on available-for-sale securities
Comprehensive loss
Comprehensive loss attributable to non-controlling interest
Comprehensive loss attributable to Solid Biosciences Inc.
2019
Year Ended December 31,
2018
2017
$
(117,223) $
(74,798) $
(53,178)
6
(117,217)
-
8
(74,790)
-
$
(117,217) $
(74,790) $
(36)
(53,214)
(1,060)
(52,154)
The accompanying notes are an integral part of these consolidated financial statements.
F-4
SOLID BIOSCIENCES INC.
CONSOLIDATED STATEMENTS OF PREFERRED UNITS AND STOCKHOLDERS’/MEMBERS’ EQUITY/(DEFICIT)
(In thousands except for share data)
Redeemable
Preferred
Units
Amount
Series 2
Senior
Preferred
Units
Amount
Series 1
Senior
Preferred
Units
Junior
Preferred
Units
Amount
Amount
Series
A, B, C
and D
Common
Units
Amount
Common
Stock
Amount
Additional
paid
in capital
Accumulated
other
comprehensive
income (loss)
Accumulated
Deficit
Total
Members’/
Stockholders’
Equity
(Deficit)
Non-
controlling
Interest
Total
Equity
(Deficit)
Balance at
December 31, 2016 17,100,000 $ 71,649
-
-
-
-
-
-
5,123,917 $
558
-
-
- $
23 $
(84,941 ) $
(84,360 ) $
46,474 $ (37,886 )
Issuance of
Series 1
senior
preferred
units, net
of issuance
costs of $500
and tranche
right
of $459
Accretion of
Series 1
senior
preferred
units to
redemption
value
Redemption
of preferred
units
Equity-based
compensation
Net loss
Issuance of
Series B
common
units in
exchange
for Series A
common
units
Issuance of
Series D
common
units in
exchange
for Series A
common
units
Issuance of
Series A
common
units in
exchange
for
redeemable
preferred
units
Issuance of
junior
preferred
units in
redemption
of Class D
non-
controlling
interest in
Solid GT
Issuance of
Series C
common
units in
exchange
for Class B
non-
controlling
interest in
Solid GT
Issuance of
Series D
common
units in
exchange
for Class C
non-
controlling
interest in
Solid GT
Issuance of
Series D
common
units
Issuance of
Series 2
senior
preferred
units,
Unrealized
loss on
available-for-
sale securities
Balance at
December 31, 2017
Conversion
of units into
shares of
common
stock
Issuance of
common
stock upon
initial public
offering,
net of
issuance
costs of
$4,592
Equity-based
compensation
Net loss
Forfeiture of
restricted
stock/unit
awards
Unrealized
gain on
available-for-
sale securities
Balance at
-
-
-
- 2,500,000 $ 24,041
-
-
-
-
-
-
-
-
-
-
-
-
-
-
- (15,685 )
-
-
-
-
-
-
-
-
-
-
-
-
-
959
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
5,030
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
(959 )
(959 )
-
(959 )
15,685
15,685
-
15,685
-
(52,118 )
5,030
(52,118 )
300
(1,060 )
5,330
(53,178 )
-
-
-
-
-
-
-
-
(1,301,520 )
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
(160,954 )
-
-
-
-
-
-
-
-
-
(17,100,000 ) (55,964 )
-
-
-
-
-
- 12,219,299 55,964
-
-
-
-
-
55,964
-
55,964
-
-
-
-
-
- 4,414,356 $ 44,177
-
-
-
-
-
-
(1,925 )
(1,925 )
(42,252 )
(44,177 )
-
-
-
-
-
-
-
-
1,635,916
2,053
-
-
-
-
-
2,053
(2,053 )
-
-
-
-
-
-
-
-
-
1,083,205
1,409
-
-
-
-
-
-
-
-
-
-
-
838,689
-
-
-
-
-
-
-
1,409
(1,409 )
-
-
-
-
- 4,886,000 $ 55,002
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
- 4,886,000 55,002 2,500,000 25,000 4,414,356 44,177 19,438,552 $ 65,014
-
-
-
-
-
- $
(36 )
-
(36 )
-
(36 )
(13 ) $
(124,258 ) $
(59,257 ) $
— $ (59,257 )
-
- (4,886,000 ) (55,002 ) (2,500,000 ) (25,000 ) (4,414,356 ) (44,177 ) (19,429,620 ) (65,180 ) 26,498,559
26
189,333
-
-
124,179
- 124,179
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
- 8,984,375 $
9
129,087
166
-
-
-
5,789
-
-
-
-
-
-
-
-
-
(8,932 )
-
(50,474 )
-
-
-
-
-
-
-
129,096
- 129,096
-
(74,798 )
5,955
(74,798 )
-
-
5,955
(74,798 )
-
-
-
-
-
-
-
-
-
—
-
—
-
—
-
—
-
—
-
—
-
—
-
-
— 35,432,460
- $
35
-
324,209
8
(5 )
-
(199,056 )
8
125,183
-
8
— 125,183
December 31, 2018
Equity-based
compensation
Sale of
common
stock,
net of
issuance
costs
of $2,102
Sale of pre-
funded
warrants
Net loss
Forfeiture of
restricted
stock awards
Unrealized
gain on
available-for-
sale securities
Balance at
December 31, 2019
-
-
-
-
-
-
-
-
-
-
—
14,207
-
-
14,207
-
14,207
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
- $
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
- $
-
-
-
-
-
-
-
- 10,607,525 $
11
47,212
-
-
- 2,295,699
-
2
10,650
-
-
(52,414 )
-
-
-
-
-
-
-
47,223
-
47,223
-
(117,223 )
10,652
(117,223 )
-
10,652
- (117,223 )
-
-
-
-
6
-
-
6
-
-
- $
-
6
-
- $
- 48,283,270
48 $ 396,278 $
1 $
(316,279 ) $
80,048 $
- $
80,048
The accompanying notes are an integral part of these consolidated financial statements.
F-5
SOLID BIOSCIENCES INC.
CONSOLIDATED STATEMENTS OF CASH FLOWS
(In thousands)
Cash flows from operating activities:
Net loss
Adjustments to reconcile net loss to net cash used in operating activities:
Amortization of (discount)/premium on available-for-sale securities
Equity-based compensation expense
Depreciation expense
Loss on sale of property and equipment
(Gain) from revaluation of preferred unit tranche right
Changes in operating assets and liabilities:
Proceeds from landlord lease incentive for tenant improvements
Prepaid expenses and other current assets
Accounts payable
Accrued expenses and other current liabilities
Net cash used in operating activities
Cash flows from investing activities:
Purchases of property and equipment
Proceeds from sales and maturities of available-for-sale securities
Purchases of available-for-sale securities
Net cash provided by (used in) investing activities
Cash flows from financing activities:
Proceeds from issuance of Series 1 Senior preferred units
Proceeds from issuance of Series 2 Senior preferred units
Payment of offering costs
Proceeds from issuance of common stock
Proceeds from issuance of pre-funded warrants
Proceeds from initial public offering of common stock, net of commissions and
underwriting discounts
Principal payments under capital lease obligation
Net cash provided by financing activities
Net (decrease) increase in cash, cash equivalents and restricted cash
Cash, cash equivalents, and restricted cash at beginning of period
Cash, cash equivalents, and restricted cash at end of period
Supplemental disclosure of non-cash investing and financing activities:
Conversion of Series A, B, C and D common units into shares of
common stock
Conversion of Series 2 Senior Preferred units into shares of common stock
Conversion of Series 1 Senior Preferred units into shares of common stock
Conversion of Junior Preferred units into shares of common stock
Accretion to redemption value for redeemable preferred units
Redemption of preferred units
Redemption of redeemable interest from non-controlling interest in Solid GT
Deferred offering costs included in accounts payable and accrued expenses
Property and equipment included in accounts payable and accruals
Property and equipment acquired through a capital lease
Issuance of Series D common units in exchange for Series A common units
Issuance of Series A common units in exchange for Redeemable
preferred units
Issuance of Junior preferred units upon redemption of Class D non-controlling
interest in Solid GT
Issuance of Series C common units in exchange for Class B non-controlling
interest in Solid GT
Issuance of Series D common units in exchange for Class C non-controlling
interest in Solid GT
2019
Year Ended December 31,
2018
2017
$
(117,223) $
(74,798) $
(53,178)
(279)
14,207
2,824
2
-
-
4,486
3,779
(510)
(92,714)
(4,387)
60,399
(31,496)
24,516
-
-
(2,102)
49,325
10,652
-
-
57,875
(10,323)
86,693
76,370
$
$
$
$
$
$
$
-
-
-
-
-
-
-
-
490
-
-
-
-
-
-
(90)
5,955
1,566
4
-
823
(4,930)
(1,610)
2,883
(70,197)
(7,776)
25,335
(44,321)
(26,762)
-
-
(2,168)
-
-
133,688
(13)
131,507
34,548
52,145
86,693
$
65,180
55,002
25,000
44,177
-
-
-
-
952
1,023
-
$
$
$
$
$
$
-
$
-
$
-
$
-
$
206
5,330
448
-
(459)
-
815
1,579
2,035
(43,224)
(2,276)
31,621
(18,897)
10,448
24,500
55,002
(2,424)
-
-
-
-
77,078
44,302
7,843
52,145
-
-
-
-
(959)
15,685
(1,925)
682
265
-
638
55,964
44,177
2,053
1,409
$
$
The accompanying notes are an integral part of these consolidated financial statements.
F-6
SOLID BIOSCIENCES INC.
NOTES TO CONSOLIDATED FINANCIAL STATEMENTS
(Amounts in thousands, except share and per share data)
1. Nature of the Business and Basis of Presentation
Nature of Business
Solid Biosciences Inc. was organized in March 2013 under the name SOLID Ventures Management, LLC. In October 2013, the company changed its name
to Solid Ventures, LLC and in June 2015, the company changed its name to Solid Biosciences, LLC. The company operated as a Delaware limited liability
company under the name Solid Biosciences, LLC until immediately prior to the effectiveness of its registration statement on Form S-1 on January 25, 2018,
at which time it completed a statutory corporate conversion into a Delaware corporation (the “Corporate Conversion”) and changed its name to Solid
Biosciences Inc. (the “Company”) In addition, entities formed solely for the purpose of holding membership interests in the Company’s limited liability
company were merged with and into the Company. As a result of the Corporate Conversion, all of the Series 1 and 2 Senior Preferred, Junior Preferred
Units, Series A, B, C and D Common Units of Solid Biosciences, LLC converted into shares of common stock of Solid Biosciences Inc. on a one for
0.8485 basis and all of the unit holders of Solid Biosciences, LLC became holders of common stock of Solid Biosciences Inc.
The Company’s mission is to cure Duchenne muscular dystrophy (“DMD”), a genetic muscle-wasting disease predominantly affecting boys. It is caused by
mutations in the dystrophin gene, which result in the absence or near-absence of dystrophin protein. Dystrophin protein works to strengthen muscle fibers
and protect them from daily wear and tear. Without functioning dystrophin and certain associated proteins, muscles suffer excessive damage from normal
daily activities and are unable to regenerate, leading to the build-up of fibrotic, or scar, and fat tissue. The Company’s lead product candidate, SGT-001, is a
gene transfer candidate under investigation for its ability to drive functional dystrophin protein expression in patients’ muscles and improve the course of
the disease. SGT-001 has been granted Rare Pediatric Disease Designation and Fast Track in the United States and Orphan Drug Designations in both the
United States and European Union. The Company filed an Investigational New Drug application (“IND”) in September 2017 and initiated a Phase I/II for
SGT-001 in the United States during the fourth quarter of 2017, which is called IGNITE DMD. In November 2019, IGNITE DMD was placed on clinical
hold by the U.S. Food and Drug Administration.
The Company is subject to risks and uncertainties common to early-stage companies in the biotechnology industry, including, but not limited to,
development by competitors of new technological innovations, dependence on licenses, protection of proprietary technology, dependence on key personnel,
compliance with government regulations and the need to obtain additional financing to fund operations. Product candidates currently under development
will require significant additional research and development efforts, including extensive pre-clinical studies and clinical trials and regulatory approval, prior
to commercialization. These efforts require significant amounts of additional capital, adequate personnel infrastructure and extensive compliance and
reporting capabilities.
The Company’s product candidates are in development. There can be no assurance that the Company’s research and development will be successfully
completed, that adequate protection for the Company’s intellectual property will be obtained, that any products developed will obtain necessary
government regulatory approval or that any approved products will be commercially viable. Even if the Company’s product development efforts are
successful, it is uncertain when, if ever, the Company will generate significant revenue from product sales. The Company operates in an environment of
rapid change in technology and substantial competition from, among others, other pharmaceutical and biotechnology companies. In addition, the Company
is dependent upon the services of its employees, partners and consultants.
Initial Public Offering in January 2018
On January 30, 2018, the Company completed its initial public offering with the sale of 8,984,375 shares of common stock, including shares of common
stock issued upon the exercise in full of the underwriters’ over-allotment option, at a public offering price of $16.00 per share, resulting in net proceeds of
$129,096, after deducting underwriting discounts and commissions and offering expenses.
Liquidity
The accompanying consolidated financial statements have been prepared on a basis that assumes the Company will continue as a going concern and which
contemplates the realization of assets and satisfaction of liabilities and commitments in the ordinary course of business. Through December 31, 2019, the
Company has funded its operations primarily with the proceeds from the sale of redeemable preferred units and member units as well as the sale of
common stock and prefunded warrants to purchase shares of its common stock in private placements and the sale of common stock in its initial public
offering.
F-7
On July 30, 2019, the Company issued and sold in a private placement (i) 10,607,525 shares of its common stock at a price per share of $4.65 and (ii)
2,295,699 pre-funded warrants to purchase shares of its common stock at a price per warrant of $4.64. Each pre-funded warrant is exercisable for one share
of common stock at an exercise price of $0.01 and the pre-funded warrants have no expiration date. The Company received gross proceeds from the private
placement of $59,977, before deducting offering costs of $2,102.
In accordance with ASC 205-40, Going Concern, the Company has evaluated whether there are conditions and events, considered in the aggregate, that
raise substantial doubt about the Company’s ability to continue as a going concern within one year after the date the financial statements are issued. As of
December 31, 2019, the Company had an accumulated deficit of $316,279. During the year ended December 31, 2019, the Company incurred a net loss of
$117,223 and used $92,714 of cash in operations. The Company expects to continue to generate operating losses in the foreseeable future. Based upon its
current operating plan, the Company expects that its cash, cash equivalents and available-for-sale securities of $83,524 will be sufficient to fund its
operating expenses and capital requirements into 2021.
In accordance with the requirements of ASC 205-40, the Company determined that there is substantial doubt about the Company’s ability to continue as a
going concern within twelve months of the issuance date of these financial statements. The financial statements do not include any adjustments that might
result from the outcome of this uncertainty. Although the Company has been successful in raising capital in the past, there is no assurance that it will be
successful in obtaining such additional financing on terms acceptable to the Company, if at all, nor is it considered probable under the accounting standards.
As such, under the requirements of ASC 205-40, management may not consider the potential for future capital raises or management plans to reduce costs
that are not considered probable in its assessment of the Company’s ability to meet its obligations for the next twelve months. If the Company is unable to
obtain funding, the Company would be forced to delay, reduce or eliminate some or all of its research and development programs, pre-clinical and clinical
testing or commercialization efforts, which could adversely affect its business prospects.
Merger and Recapitalization in March 2017
The Company had historically owned 100% of the voting units of its wholly owned subsidiary, Solid GT, LLC (“Solid GT”), and the results of Solid GT
were included in the Company’s consolidated financial statements. In November 2015, Solid GT issued voting units to new investors which decreased the
Company’s voting ownership in Solid GT to 77%. The Company continued to consolidate the results of Solid GT into its financial statements as the
Company owned a majority voting interest in Solid GT and directed the activities of Solid GT. However, because the Company controlled but owned less
than 100% of Solid GT, the Company recorded a non-controlling ownership interest at its fair value at inception and recognizes the net loss or profit
attributable to non-controlling interests in the consolidated statements of operations based on a profit and loss sharing arrangement between the Company
and the non-controlling interests. The Company also presented the change in equity related to equity-based compensation issued to Solid GT employees by
Solid GT, in non-controlling interest.
On March 29, 2017, the Company merged the operations of Solid GT into the Company and Solid GT ceased to exist as a legal entity. See Note 3, Merger
and Recapitalization, for additional information.
The proportionate share of the loss attributed to the non-controlling interest amounted to $0, $0 and $1,060, for the years ended December 31, 2019, 2018
and 2017, respectively.
There was no non-controlling interest at December 31, 2019 and 2018.
The accompanying consolidated financial statements have been prepared in accordance with accounting principles generally accepted in the United States
of America (“GAAP”). The accompanying consolidated financial statements include the accounts of Solid Biosciences Inc. and its wholly owned or
controlled subsidiaries. All intercompany accounts and transactions have been eliminated.
F-8
2. Summary of Significant Accounting Policies
Use of Estimates
The preparation of the Company’s consolidated financial statements in conformity with GAAP requires management to make estimates, judgments and
assumptions that affect the reported amounts of assets and liabilities, the disclosure of contingent assets and liabilities at the date of the consolidated
financial statements and the reported amounts of expenses during the reporting periods. Significant estimates and assumptions reflected in these
consolidated financial statements include, but are not limited to, the recognition of research and development expenses and equity-based compensation.
Estimates are periodically reviewed in light of changes in circumstances, facts and experience. Changes in estimates are recorded in the period in which
they become known. Actual results could differ from the Company’s estimates.
Cash Equivalents
The Company considers all short-term, highly liquid investments with original maturities of 90 days or less at acquisition date to be cash equivalents.
Restricted Cash
The Company held restricted cash of $327 in separate restricted bank accounts as security deposits for leases of the Company’s facilities as of December
31, 2019 and December 31, 2018. The Company has included restricted cash of $327 as a non-current asset as of December 31, 2019 and December 31,
2018. A reconciliation of the amounts of cash and cash equivalents and restricted cash from the cash flow statement to the balance sheet is as follows:
Cash and cash equivalents
Restricted cash, current
Restricted cash, non-current
Cash and cash equivalents and restricted cash
Available-for-Sale Securities
December 31,
2019
December 31,
2018
December 31,
2017
$
$
76,043 $
-
327
76,370 $
86,366 $
-
327
86,693 $
52,080
65
-
52,145
Available-for-sale securities consist of investments with original maturities greater than 90 days at acquisition date. The Company has classified its
investments with maturities beyond one year as short term, based on their highly liquid nature and because such available-for-sale securities represent the
investment of cash that is available for current operations.
The Company classifies all of its investments as available-for-sale securities. The Company’s investments are measured and reported at fair value using
quoted prices in active markets for similar securities. Unrealized gains and losses on available-for-sale debt securities are reported as a separate component
of stockholders’/members’ equity/(deficit). The cost of debt securities sold is determined on a specific identification basis, and realized gains and losses are
included in other income (expense) within the consolidated statement of operations. If any adjustment to fair value reflects a decline in the value of the
investment that the Company considers to be “other than temporary,” the Company reduces the investment to fair value through a charge to the
consolidated statement of operations. No such adjustments were necessary during the periods presented.
Concentration of Credit Risk and of Significant Suppliers
Financial instruments that potentially expose the Company to concentrations of credit risk consist primarily of cash, cash equivalents and available-for-sale
securities. Periodically, the Company maintains deposits in accredited financial institutions in excess of federally insured limits. The Company maintains
each of its cash, cash equivalents and available-for-sale securities balances with high-quality and accredited financial institutions and accordingly, such
funds are not exposed to significant credit risk. The Company does not believe that it is subject to unusual credit risk beyond the normal credit risk
associated with commercial banking relationships.
The Company is dependent on third-party manufacturers to supply products for research and development activities of its programs, including clinical and
pre-clinical testing. These programs could be adversely affected by a significant interruption in the supply of such drug substance products.
F-9
Fair Value Measurements
Certain assets and liabilities are carried at fair value under GAAP. Fair value is defined as the exchange price that would be received for an asset or paid to
transfer a liability (an exit price) in the principal or most advantageous market for the asset or liability in an orderly transaction between market participants
on the measurement date. Valuation techniques used to measure fair value must maximize the use of observable inputs and minimize the use of
unobservable inputs. Financial assets and liabilities carried at fair value are to be classified and disclosed in one of the following three levels of the fair
value hierarchy, of which the first two are considered observable and the last is considered unobservable:
•
•
•
Level 1—Quoted prices in active markets for identical assets or liabilities.
Level 2—Observable inputs (other than Level 1 quoted prices), such as quoted prices in active markets for similar assets or liabilities, quoted
prices in markets that are not active for identical or similar assets or liabilities or other inputs that are observable or can be corroborated by
observable market data.
Level 3—Unobservable inputs that are supported by little or no market activity and that are significant to determining the fair value of the
assets or liabilities, including pricing models, discounted cash flow methodologies and similar techniques.
The Company’s cash equivalents and available-for-sale securities are carried at fair value, determined according to the fair value hierarchy described above.
See Note 4, Fair Value of Financial Assets and Liabilities, for additional information. The carrying values of the Company’s accounts payable and accrued
expenses and other current liabilities approximate their fair value due to the short-term nature of these liabilities.
Leases
At inception of a contract, the Company determines if a contract meets the definition of a lease. A lease is a contract, or part of a contract, that conveys the
right to control the use of identified property, plant, or equipment (an identified asset) for a period of time in exchange for consideration. The Company
determines if the contract conveys the right to control the use of an identified asset for a period of time. The Company assesses throughout the period of use
whether the Company has both of the following: (1) the right to obtain substantially all of the economic benefits from use of the identified asset and (2) the
right to direct the use of the identified asset. This determination is reassessed if the terms of the contract are changed. Leases are classified as operating or
finance leases based on the terms of the lease agreement and certain characteristics of the identified asset. Right-of-use assets and lease liabilities are
recognized at the lease commencement date based on the present value of the minimum future lease payments. The Company’s policy is to not record
leases with an original term of twelve months or less on the consolidated balance sheets. The Company recognizes lease expense for these short-term leases
on a straight-line basis over the lease term. Certain lease agreements include rental payments that are adjusted periodically for inflation or other variables.
In addition to rent, the leases may require the Company to pay additional amounts for taxes, insurance, maintenance and other expenses, which are
generally referred to as non-lease components. Such adjustments to rental payments and variable non-lease components are treated as variable lease
payments and recognized in the period in which the obligation for these payments was incurred. Variable lease components and variable non-lease
components are not measured as part of the right of use asset and liability. Only when lease components and their associated non-lease components are
fixed are they accounted for as a single lease component and recognized as part of a right of use asset and liability. Total contract consideration is allocated
to the combined fixed lease and non-lease components.
Property and Equipment
Property and equipment are stated at cost less accumulated depreciation. Depreciation expense is recognized using the straight-line method over the useful
life of the asset. Laboratory equipment is depreciated over five years. Computer equipment is depreciated over three years. Computer software is
depreciated over two years. Furniture and office equipment are depreciated over five years. Leasehold improvements are amortized over the shorter of the
lease term or the estimated useful life of the related asset. Expenditures for repairs and maintenance of assets are charged to expense as incurred. Upon
retirement or sale, the cost and related accumulated depreciation of assets disposed of are removed from the accounts and any resulting gain or loss is
included in loss from operations. Equipment under a finance lease is stated at fair value at the inception of the lease less accumulated depreciation and is
depreciated over the remaining lease term or the estimated useful life of the equipment.
F-10
Impairment of Long-Lived Assets
Long-lived assets, comprised of property and equipment, to be held and used are tested for recoverability whenever events or changes in business
circumstances indicate that the carrying amount of the assets may not be fully recoverable. Factors that the Company considers in deciding when to
perform an impairment review include significant underperformance of the business in relation to expectations, significant negative industry or economic
trends and significant changes or planned changes in the use of the assets. If an impairment review is performed to evaluate a long-lived asset for
recoverability, the Company compares forecasts of undiscounted cash flows expected to result from the use and eventual disposition of the long-lived asset
to its carrying value. An impairment loss would be recognized when estimated undiscounted future cash flows expected to result from the use of an asset
are less than its carrying amount. The impairment loss would be based on the excess of the carrying value of the impaired asset over its fair value,
determined based on discounted cash flows. To date, the Company has not recorded any impairment losses or disposals on long-lived assets.
Research and Development Costs
Research and development costs are expensed as incurred. Research and development expenses include salaries, equity-based compensation and benefits of
employees, third-party license fees and other operational costs related to the Company’s research and development activities, including allocated facility-
related expenses and external costs of outside vendors engaged to conduct both pre-clinical studies and clinical trials. Non-refundable pre-payments for
goods or services that will be used or rendered for future research and development activities are deferred and capitalized. Such amounts are recognized as
expense as the goods or services are delivered or the related services are performed, or until it is no longer expected that the goods will be delivered or the
services rendered.
Research Contract Costs and Accruals
The Company has entered into various research and development contracts with research institutions and other companies. These agreements are generally
cancelable, and related payments are recorded as research and development expenses as incurred. The Company records accruals for estimated ongoing
research costs. When evaluating the adequacy of the accrued liabilities, the Company analyzes progress of the studies, including the phase or completion of
events, invoices received and contracted costs. Significant judgments and estimates are made in determining the accrued balances at the end of any
reporting period. Actual results could differ from the Company’s estimates. The Company’s historical accrual estimates have not been materially different
from the actual costs.
Patent Costs
All patent-related costs incurred for filing and prosecuting patent applications are expensed as incurred due to the uncertainty about the recovery of the
expenditure. Amounts incurred are classified as general and administrative expenses.
Equity-Based Compensation
In connection with the completion of the Company’s initial public offering, the Company adopted the 2018 Omnibus Incentive Plan, which provides for the
issuance of share-based awards, including options to purchase common stock. The 2018 Omnibus Incentive Plan provides for the awarding of up to
5,001,000 shares of common stock for equity awards.
The Company measures all stock options and other stock-based awards granted to employees, directors and non-employees based on the fair value on the
date of the grant and recognizes compensation expense of those awards, over the requisite service period, which is generally the vesting period of the
respective award. Forfeitures are accounted for as they occur. The Company applies the straight-line method of expense recognition to all awards with only
service-based vesting conditions. The Company has not issued any awards with performance-based vesting conditions. For stock-based awards granted to
non-employees, compensation expense is recognized over the period during which services are rendered by such non-employees until completed.
The fair value of each stock option grant is estimated on the date of grant using the Black-Scholes option-pricing model. The Company historically has
been a private company and lacks company-specific historical and implied volatility information. Therefore, it estimates its expected stock volatility based
on the historical volatility of a publicly traded set of peer companies and expects to continue to do so until such time as it has adequate historical data
regarding the volatility of its own traded stock price. For options with service-based vesting conditions, the expected term of the Company’s stock options
has been determined utilizing the “simplified” method for awards that qualify as “plain-vanilla” options. Through December 31, 2018, the expected term of
stock options granted to non-employees is equal to the contractual term of the option award and effective January 1, 2019, the “simplified” method is used.
The risk-free interest rate is determined by reference to the U.S. Treasury yield curve in effect at the time of grant of the award for time periods
approximately equal to the expected term of the award. Expected dividend yield is based on the fact that the Company has never paid cash dividends and
does not expect to pay any cash dividends in the foreseeable future.
F-11
The Company classifies stock-based compensation expense in its consolidated statement of operations in the same manner in which the award recipient’s
payroll costs are classified or in which the award recipient’s service payments are classified.
Income Taxes
Income taxes are accounted for under the asset and liability method. Under the asset and liability method, deferred tax assets and liabilities are recognized
for the future tax consequences attributable to differences between the financial statement carrying amounts of existing assets and liabilities and their
respective tax bases. Deferred tax assets and liabilities are measured using enacted tax rates expected to apply to taxable income in the years in which those
temporary differences are expected to be recovered or settled. The effect on deferred tax assets and liabilities of a change in tax rates is recognized in
income in the period that includes the enactment date. The Company records valuation allowances to reduce deferred income tax assets to the amount that
is more likely than not to be realized. The Company determines whether it is more likely than not that a tax position will be sustained upon examination. If
it is not more likely than not that a position will be sustained, no amount of benefit attributable to the position is recognized. The tax benefit to be
recognized of any tax position that meets the more likely than not recognition threshold is calculated as the largest amount that is more than 50% likely of
being realized upon resolution of the contingency.
Prior to January 25, 2018, the Company had not been subject to U.S. federal income taxes as the Company was organized as a limited liability company. As
such, the taxable income or loss was passed through to and included in the tax returns of the members. Since January 25, 2018, the Company’s income has
since been subject to U.S. federal, state, local, and foreign income taxes and taxed at the prevailing corporate tax rates.
Segment Data
The Company manages its operations as a single segment for the purposes of assessing performance and making operating decisions. The Company’s
singular focus is on developing treatments through gene therapy and other means for patients with DMD. All of the Company’s tangible assets are held in
the United States.
Comprehensive Loss
Comprehensive loss includes net loss, as well as other changes in stockholders’/members’ equity/(deficit) that result from transactions and economic events
other than those with members. The Company’s only element of other comprehensive income (loss) in all periods presented was unrealized gains (losses)
from available-for-sale securities.
Net Loss per Share
The Company follows the two-class method when computing net loss per share, as the Company has issued shares that meet the definition of participating
securities. The two-class method determines net loss per share for each class of common and participating securities according to dividends declared or
accumulated and participation rights in undistributed earnings. The two-class method requires income available to common stockholders for the period to
be allocated between common and participating securities based upon their respective rights to receive dividends as if all income for the period had been
distributed.
Basic net loss per share attributable to common stockholders is computed by dividing the net loss attributable to common stockholders by the weighted
average number of shares of common stock and pre-funded warrants outstanding for the period. Diluted net loss attributable to common stockholders is
computed by adjusting net loss attributable to common stockholders to reallocate undistributed earnings based on the potential impact of dilutive securities.
Diluted net loss per share attributable to common stockholders is computed by dividing the diluted net loss attributable to common stockholders by the
weighted average number of shares of common stock and pre-funded warrants outstanding for the period, including potential dilutive shares of common
stock assuming the dilutive effect of common stock equivalents.
The Company’s preferred stock contractually entitles the holders of such shares to participate in dividends but does not contractually require the holders of such
shares to participate in losses of the Company. Accordingly, in periods in which the Company reports a net loss, such losses are not allocated to such
participating securities. In periods in which the Company reports a net loss attributable to common stockholders, diluted net loss per share attributable to
common stockholders is the same as basic net loss per share attributable to common stockholders, since dilutive shares of common stock are not assumed to
have been issued if their effect is anti-dilutive.
F-12
Funding from Charitable Organizations
The Company has received funding from charitable organizations to perform research and development services to identify therapies for people with DMD.
The amounts received are recognized as services are performed and research expenses are incurred. These are included in other income in the consolidated
statements of operations as the arrangement between the Company and the charitable organizations are not part of the Company’s on-going, major or
central operations. Any amount received in advance of services performed is recorded in other current liabilities in the consolidated balance sheets if the
services are expected to be performed within the next twelve months.
The Company recognized other income of $515, $270 and $1,001 for the years ended December 31, 2019, 2018 and 2017, respectively, which is included
in the consolidated statements of operations.
Contingencies
Loss contingency provisions are recorded if the potential loss from any claim, asserted or unasserted, or legal proceeding, is considered probable and the
amount can be reasonably estimated, or a range of loss can be determined. These accruals represent the Company’s best estimate of probable loss.
Disclosure also is provided when it is reasonably possible that a loss will be incurred or when it is reasonably possible that the amount of a loss will exceed
the recorded provision. The Company reviews the status of each significant matter and assesses its potential financial exposure. Significant judgment is
required in both the determination of probability and the determination as to whether an exposure is reasonably estimable. Because of uncertainties related
to these matters, accruals are based only on the best information available at the time. As additional information becomes available, the Company
reassesses the potential liability related to pending claims and may change its estimates. These changes in the estimates of the potential liabilities could
have a material impact on the Company’s consolidated results of operations and financial position.
Recently Adopted Accounting Pronouncements
The Company adopted Accounting Standards Update (“ASU”) No. 2016-02, Leases (Topic 842), as of January 1, 2019 using the modified retrospective
transition approach with no restatement of prior periods or cumulative adjustment to accumulated deficit. Upon adoption, the Company elected the package
of transition practical expedients, which allowed the Company to carry forward prior conclusions related to whether any expired or existing contracts are or
contain leases, the lease classification for any expired or existing leases and initial direct costs for existing leases. The Company also made an accounting
policy election not to recognize leases with an initial term of 12 months or less within its consolidated balance sheets and to recognize those lease payments
on a straight-line basis in its consolidated statements of operations over the lease term. Upon adoption of ASU 2016-02, the Company recognized operating
lease, right-of-use assets of approximately $4,600 and a corresponding operating lease liability of approximately $5,900, which are included in the
Company’s consolidated balance sheet. The adoption of ASU 2016-02 did not have any impact on the Company’s consolidated statements of operations
and comprehensive loss.
Recently Issued Accounting Pronouncements
In August 2018 the Financial Accounting Standards Board issued ASU No. 2018-13, Fair Value Measurement (Topic 820): Disclosure Framework -
Changes to the Disclosure Requirements for Fair Value Measurement. This new standard modifies certain disclosure requirements on fair value
measurements. This new standard will be effective for the Company on January 1, 2020. The adoption of this new standard will not have a material impact
on the disclosures.
3. Merger and Recapitalization
On March 29, 2017, the Company completed a series of transactions, which included the issuance of Series 1 Senior Preferred Units pursuant to the Senior
Preferred Unit Purchase Agreement (the “Senior Preferred Unit Purchase Agreement”) and the merger of Solid GT into the Company pursuant to the
merger agreement between the Company and Solid GT (the “Merger Agreement”), collectively referred to as the “Merger and Recapitalization.” As part of
the Merger and Recapitalization, the Company (a) issued 2,500,000 Series 1 Senior Preferred Units to new investors at $10.00 per unit resulting in gross
proceeds to the Company of $25,000, (b) merged operations of Solid GT into the Company, effected through the exchange of Solid GT units held by non-
controlling interests of the Company into new classes of the Company units, and (c) exchanged existing Redeemable Preferred Units and Series A
Common Units of the Company into new units. The details of each component of the Merger and Recapitalization are as follows:
F-13
(a)
Issuance of Series 1 Senior Preferred Units
Pursuant to the Senior Preferred Unit Purchase Agreement, the Company issued 2,500,000 Series 1 Senior Preferred Units to new investors at $10.00 per
unit resulting in gross proceeds to the Company of $25,000.
See Note 10, Redeemable Preferred Units, Series 2 and Series 1 Senior Preferred Units and Junior Preferred Units, for additional information.
(b)
Merger of Solid GT into the Company
Prior to the Merger and Recapitalization, the Company issued Class B Non-Voting and Class D Voting Units of Solid GT to holders which represent non-
controlling interests of the Company. On March 29, 2017, in connection with the Merger and Recapitalization, the non-controlling interests were eliminated
as follows:
•
•
50,000 Class B Non-Voting Units of Solid GT (“Solid GT Class B Units”) were exchanged for 1,635,916 Series C Common Units of the
Company; and
134,920 Class D Voting Units of Solid GT (“Solid GT Class D Units”) were exchanged for 4,414,356 Junior Preferred Units of the
Company.
In addition, the Class C Non-Voting Units of Solid GT (“Solid GT Class C Restricted Units”) were exchanged for Series D Common Units of the
Company. The Solid GT Class C Restricted Units were held by employees and consultants of Solid GT. See Note 12, Equity-Based Compensation, for
additional information.
Since there was no change in control in connection with the Solid GT merger, the exchange of Solid GT Class B Units, Class C Restricted Units and
Class D Units was accounted for as an equity transaction. In addition, because Solid GT Class D Units represented preferred units with preference over the
other classes of Solid GT Units, the difference between the carrying value of the Solid GT Class D Units and the fair value of Junior Preferred Units was
recorded as a deemed dividend in members’ deficit, which impacts net loss attributable to common unitholders. See Note 16, Net Loss per Share, for
additional information.
(c)
Exchange of the Company’s existing Redeemable Preferred Units and Series A Common Units
In connection with the Merger and Recapitalization, the Company exchanged its existing Redeemable Preferred Units and Series A Common Units as
follows:
•
•
•
17,100,000 Redeemable Preferred Units of the Company were exchanged for 12,219,299 Series A Common Units of the Company. See
Note 10, Redeemable Preferred Units, Series 2 and Series 1 Senior Preferred Units and Junior Preferred Units, for additional information.
4,560,000 Series A Common Units of the Company were exchanged for 3,258,480 Series B Common Units of the Company. See Note 11,
Members’ Deficit, for additional information.
563,917 Series A Common Units of the Company were exchanged for 402,963 Series D Common Units of the Company. See Note 11,
Members’ Deficit, for additional information.
The table below displays the pre-merger and post-merger capitalization structure of the Company:
Entity
Company
Company
Company
Solid GT
Solid GT
Solid GT
Solid GT
Company
(Total)
Pre-Merger and Recapitalization
Class
Redeemable Preferred
Series A Common (Founders)
Series A Common (Others)
Class A Voting
Class B Non-Voting
Class C Non-Voting
Class D Voting
Issued
17,100,000
4,560,000
563,917
450,000
50,000
33,107
134,920
Common Units (Series A)
5,123,917
F-14
Entity
Company
Company
Company
Company
Company
Company
Company
(Total)
Post-Merger and Recapitalization
Class
Series A Common
Series B Common
Series D Common
Ceased to exist
Series C Common
Series D Common
Junior Preferred
Common Units
(Series A, B, C and D)
Issued
12,219,299
3,258,480
402,963
1,635,916
1,083,205
4,414,356
18,599,863
4. Fair Value of Financial Assets and Liabilities
The following tables present information about the Company’s assets and liabilities that are measured at fair value on a recurring basis and indicate the
level of the fair value hierarchy utilized to determine such fair values:
Assets:
Cash equivalents
Available-for-sale securities
Assets:
Cash equivalents
Available-for-sale securities
Fair Value Measurements as of December 31, 2019 Using:
Level 1
Level 2
Level 3
Total
- $
-
- $
50,037 $
7,481
57,518 $
- $
-
- $
50,037
7,481
57,518
Fair Value Measurements as of December 31, 2018 Using:
Level 1
Level 2
Level 3
Total
- $
-
- $
54,423 $
36,098
90,521 $
- $
-
- $
54,423
36,098
90,521
$
$
$
$
As of December 31, 2019 and 2018, the fair values of the Company’s available-for-sale securities were determined using level two inputs. At December
31, 2019, the portfolio consisted of corporate bond securities and commercial paper. At December 31, 2018, the portfolio consisted of U.S. government
agency securities, corporate bond securities and commercial paper. During the years ended December 31, 2019 and 2018, there were no transfers between
Level 1, Level 2 and Level 3.
The fair value of the Company’s cash, restricted cash, accounts payable, and accrued expenses and other current liabilities approximate their carrying value
due to their short-term maturities.
5. Available-for-Sale Securities
As of December 31, 2019 and 2018, the fair value of available-for-sale debt securities by type of security was as follows:
Investments:
Corporate bond securities
Commercial paper
Investments:
U.S. government agency securities
Corporate bond securities
Commercial paper
Amortized
Cost
December 31, 2019
Gross
Unrealized
Gain
Gross
Unrealized
Loss
Fair
Value
1,502 $
5,978
$
7,480
1 $
-
$
1
- $
-
$
-
1,503
5,978
7,481
Amortized
Cost
December 31, 2018
Gross
Unrealized
Gain
Gross
Unrealized
Loss
Fair
Value
13,543 $
12,860
9,700
$
36,103
- $
2
-
$
2
(2) $
(5)
-
(7) $
13,541
12,857
9,700
36,098
$
$
$
$
F-15
The estimated fair value and amortized cost of the Company’s available-for-sale securities by contractual maturity are summarized as follows:
Due in one year or less
Total available-for-sale securities
December 31, 2019
December 31, 2018
Amortized
Cost
Fair
Value
Amortized
Cost
Fair
Value
$
$
7,480 $
7,480 $
7,481 $
7,481 $
36,103 $
36,103 $
36,098
36,098
The average maturity of the Company’s available-for-sale securities as of December 31, 2019 and 2018 was approximately 0.2 years and 0.3 years,
respectively.
6. Prepaid Expenses and Other Current Assets
Prepaid expenses and other current assets consist of the following:
Prepaid research and development expenses
Prepaid expenses and other assets
7. Property and Equipment
Property and equipment consists of the following:
Furniture and fixtures
Laboratory equipment
Leasehold improvements
Computer equipment
Computer software
Construction in process
Less accumulated depreciation
December 31,
2019
2018
1,290 $
1,488
2,778 $
4,365
1,810
6,175
December 31,
2019
2018
203 $
9,425
4,686
428
372
1,322
16,436
4,791
11,645 $
187
5,998
4,585
166
123
1,351
12,410
1,988
10,422
$
$
$
$
Depreciation expense was $2,824, $1,566 and $448 for the years ended December 31, 2019, 2018, and 2017 respectively.
8. Accrued Expenses
Accrued expenses and other current liabilities consist of the following:
Accrued research and development
Accrued compensation
Accrued other
December 31,
2019
2018
$
$
3,742 $
3,583
1,853
9,178 $
3,529
3,534
1,172
8,235
F-16
9. Preferred Unit Tranche Right
Included in the terms of the Series 1 Senior Preferred Unit Purchase Agreement was a Tranche Right which obligated the investors to purchase additional
preferred units under certain conditions. The Tranche Right also provided the investors with the right to purchase these additional units. The Company
concluded that the Tranche Right met the definition of a freestanding financial instrument as the Tranche Right was legally detachable and separately
exercisable from the Series 1 Senior Preferred Units. Therefore, the Company allocated the net proceeds to the Tranche Right and the Series 1 Senior
Preferred Units based on the fair value at the date of issuance with the remaining proceeds being allocated to the Series 1 Senior Preferred Units.
The estimated fair value of the Tranche Right was determined using a probability-weighted present value model that considered the probability of closing
the tranche through achievement of the preclinical milestones, estimated to be 50% on the date of issue and the estimated future value of Series 1 Senior
Preferred Units at closing. The Company converted future values to present value using a discount rate appropriate for probability adjusted cash flows. The
estimates were based, in part, on subjective assumptions. Changes to these assumptions can have a significant impact on the fair value of the Tranche
Right. The Tranche Right was settled in connection with the closing of the Series 2 Senior Preferred Unit financing on October 26, 2017. There were no
tranche rights outstanding as of December 31, 2019, 2018 or 2017.
A roll-forward of the Tranche Right is as follows:
Balance at December 31, 2016
Issuance
Change in fair value
Balance at December 31, 2017
Series 1 Senior
Preferred Unit
Tranche Right
$
$
-
459
(459)
-
10. Redeemable Preferred Units, Series 2 and Series 1 Senior Preferred Units and Junior Preferred Units
Redeemable Preferred Units
The Company issued redeemable preferred units (“Redeemable Preferred Units”). The Redeemable Preferred Units were classified outside of members’
deficit because the units contained redemption features that are not solely within the control of the Company.
In December 2013, the Company issued 3,420,000 Redeemable Preferred Units at an issuance price of $1.00 per unit for proceeds of $3,420.
In December 2014, the Company issued 3,420,000 Redeemable Preferred Units at an issuance price of $1.00 per unit for proceeds of $3,420.
In October 2015, the Company issued 6,840,000 Redeemable Preferred Units at an issuance price of $1.00 per unit for proceeds of $6,840.
In November and December 2016, the Company issued an aggregate of 3,420,000 Redeemable Preferred Units at $1.00 per unit for proceeds of
$3,420.
On March 29, 2017, the Redeemable Preferred Units were exchanged to Series A Common Units. See Note 3, Merger and Recapitalization, for additional
information. The Redeemable Preferred Units, which were carried at fair value due to their fair value redemption feature, were remeasured for a final time
to their redemption value on March 29, 2017 and then were reclassified to members’ deficit. There were no Redeemable Preferred Units authorized, issued
or outstanding as of December 31, 2019, 2018 or 2017.
The holders of the Redeemable Preferred Units had the following rights and preferences:
F-17
Redemption
The Redeemable Preferred Units were redeemable on or after December 27, 2022 at the option of the Redeemable Preferred unitholder. The Redeemable
Preferred Units were redeemable at the fair market value on the redemption date.
Conversion
The Redeemable Preferred Units had no conversion rights.
Voting Rights
The holders of Redeemable Preferred Units were entitled to vote as a single class with the holders of the Series A Common Units on certain matters,
including the election of managers, with each Redeemable Preferred Unit and Series A Common Unit carrying one vote per unit.
Distributions
The Company’s Board of Managers had authority to determine the amount, if any, of proceeds available for distribution to the unitholders. Prior to the
conversion of the Redeemable Preferred Units on March 29, 2017, such proceeds were to be distributed in accordance with the following order of priority:
•
•
First, to the holders of Redeemable Preferred Units, pro rata in proportion to the remaining amount to be distributed to each such holder, until
each such holder has received distributions in an amount equal to the cumulative capital contributions since inception in respect of the
Redeemable Preferred Units.
Thereafter, to all Redeemable Preferred Unitholders, Series A Common Units held by the Company’s founders, Series A Common Units
issued to non-founders between December 27, 2013 and December 26, 2014, and vested Series A Restricted Common Unitholders issued
subsequent to December 26, 2014 pro rata in proportion to their percentage interest at the time of distribution.
No distributions were made in 2017.
Liquidation
In the event of any liquidation, dissolution, or winding-up of the Company, the assets of the Company would have been distributed in accordance with the
same order of priority that applied to distributions.
Series 2 Senior Preferred Units
On October 26, 2017, the Company completed the sale of 4,886,000 Series 2 Senior Preferred Units at a price of $11.26 per unit resulting in net proceeds
of $55,002. There were no Series 2 Senior Preferred Units authorized, issued or outstanding as of December 31, 2019 or 2018.
Series 1 Senior Preferred Units
On March 29, 2017, the Company issued 2,500,000 Series 1 Senior Preferred Units at an issuance price of $10.00 per unit for proceeds of $25,000. See
Note 3, Merger and Recapitalization, for additional information. There were no Series 1 Senior Preferred Units authorized, issued or outstanding as of
December 31, 2019 or 2018.
Junior Preferred Units
On March 29, 2017, 134,920 Solid GT Class D Units were exchanged for 4,414,356 Junior Preferred Units of the Company. See Note 3, Merger and
Recapitalization, for additional information. There were no Junior Preferred Units authorized, issued or outstanding as of December 31, 2019 or 2018.
The holders of the Series 1 and Series 2 Senior Preferred Units and Junior Preferred Units had the following rights and preferences:
F-18
Tranche Right
The holders of Series 1 Senior Preferred Units were obligated to purchase 1,973,430 Series 2 Senior Preferred Units at $12.67 per unit for gross proceeds
of $25,000 in the event the Company achieves certain pre-clinical milestones. In addition, the holders of a majority of the Series 1 Senior Preferred Units
had the right to require the holders of the Series 1 Senior Preferred Units to purchase the Series 2 Senior Preferred Units at any time prior to September 1,
2017, which in August 2017, was extended to December 1, 2017. The Tranche Right was subject to certain transfer rights. See Note 9, Preferred Unit
Tranche Right, for additional information. The Tranche Right was settled in connection with the closing of the Series 2 Senior Preferred Unit financing on
October 26, 2017.
Redemption
The Series 1 and Series 2 Senior Preferred Units were redeemable on or after March 29, 2022 at the option of the holder at a redemption price equal to the
original purchase price of $10.00 and $11.26 per unit, respectively, plus any declared but unpaid distributions. The Company presented Series 1 and Series
2 Senior Preferred Units outside of permanent equity since the redemption of Series 1 and Series 2 Senior Preferred Units was outside the control of the
Company.
The consent of the Junior Preferred unitholders along with Series 1 and Series 2 Senior Preferred unitholders could have effected a deemed liquidation
event. Therefore, the Company presented the Junior Preferred Units outside of permanent equity.
Voting Rights
The holders of the Series 1 and Series 2 Senior Preferred Units and Junior Preferred Units were entitled to vote together, and not as separate classes, with
each Series 1 and Series 2 Senior Preferred Unit, Junior Preferred Unit, Series A Common Unit and Series B Common Unit carrying one vote per unit.
Subject to maintaining certain ownership levels, the Series 1 and Series 2 Senior Preferred unitholders as a class were entitled to elect two of the nine board
members while such units were outstanding. The Junior Preferred unitholders as a class are entitled to elect two of the nine board members while such units
were outstanding.
Dividends
The holders of Series 1 and Series 2 Senior Preferred Units were entitled to an 8% annual dividend based on the Series 1 and Series 2 Senior Preferred Unit
issuance price of $10.00 and $11.26 per unit, respectively, when and if declared by the Board of Managers. No dividends were declared or paid to Series 1
or Series 2 Senior Preferred unitholders.
The holders of the Junior Preferred Units were entitled to an 8% annual dividend based on the Junior Preferred Unit issuance price of $9.63 per unit, when
and if declared by the Board of Managers. No dividends were declared or paid to Junior Preferred unitholders.
Distributions
The Company’s Board of Managers had authority to determine the amount, if any, of proceeds available for distribution. Such proceeds were to be
distributed in accordance with the following order of priority:
•
•
•
First, the Series 2 Senior Preferred unitholders were entitled to an amount distributed, on a pro rata basis, equal to the Series 2 Senior
Preferred Unit price of $11.26 per unit and any declared but unpaid Series 2 Senior Preferred dividends.
Second, the Series 1 Senior Preferred and the Junior Preferred unitholders were entitled to an amount distributed, on a pro rata basis, equal to
the Series 1 Senior Preferred Unit price of $10.00 per unit and any declared but unpaid Series 1 Senior Preferred dividends and the Junior
Preferred Unit price of $9.63 per unit and any declared but unpaid Junior Preferred dividends, respectively.
Third, the Series A, B, C and D Common unitholders were entitled to an amount distributed, on a pro rata basis, subject to certain limitations,
until the cumulative amount distributed with respect to one Series A Common Unit, Series B Common Unit, Series C Common Unit and
vested Series D Common Unit equaled the cumulative amount distributed to one Junior Preferred Unit.
F-19
•
•
Fourth, the Junior Preferred unitholders and the Series A, B, C and vested D Common unitholders were entitled to an amount distributed on a
pro rata basis, subject to certain limitations, until the cumulative amount distributed with respect to one Junior Preferred Unit, Series A
Common Unit, Series B Common Unit, Series C Common Unit and vested Series D Common Unit equaled the cumulative amount distributed
to one Series 1 Senior Preferred Unit.
Fifth, the Series 1 and Series 2 Senior Preferred, the Junior Preferred and the Series A, B, C and vested D Common unitholders were entitled
to participate on a pro rata basis in cumulative distributions, subject to certain limitations, in the remaining proceeds available for distribution.
As a result of the issuance of the Series 2 Senior Preferred Units on October 26, 2017, the Series 2 Senior Preferred unitholders were entitled to cumulative
amounts distributed equal to the amount paid per unit for the Series 2 Senior Preferred Units and any declared but unpaid Series 2 Senior Preferred
cumulative dividends, prior to and with priority over any distributions to any other unitholders. In addition, upon the issuance of the Senior Series 2
Preferred units, the holders of the Junior Preferred Units no longer shared pro rata in the order of distributions with the Senior Series 1 Preferred
unitholders and were subordinate to distributions made to Series 1 Senior Preferred unitholders.
No distributions were made during the year ended December 31, 2017.
Liquidation
In the event of any liquidation, dissolution, or winding-up of the Company, the assets of the Company would have been distributed in accordance with the
same order of priority that applied to distributions.
Corporate Conversion
Immediately prior to the effectiveness of the Company’s registration statement on Form S-1, which occurred on January 25, 2018, the Company completed
the Corporate Conversion whereby all the Series 1 and Series 2 Senior Preferred, Junior Preferred Units converted into shares of common stock.
11. Members’ Deficit
Series A, B, C and D Common Units
There were no Series A, B, C and D Common Units authorized, issued or outstanding as of December 31, 2019 or 2018.
Series A Common Units
Founders Series A Common Units
On December 27, 2013, the Company issued 4,560,000 restricted Series A Common Units to its founders with time-based vesting conditions.
On March 29, 2017, in connection with the Merger and Recapitalization, the 4,560,000 founders’ restricted Series A Common Units were exchanged for
3,258,480 restricted Series B Common Units. All restricted Series B Common Units continued to vest pursuant to the original vesting terms under the
restricted Series A Common Units agreements and the Company continued to recognize compensation expense over the related service period.
In addition, in connection with the exchange of the founders’ restricted Series A Common Units into restricted Series B Common Units, the Company
recognized $2,710 of equity based compensation expense for vested units, which represents the incremental fair value of the units before and after the
Merger and Recapitalization. The Company has recorded the additional compensation expense in the amount of $904 over the remaining vesting period of
the Series B Common Units during the year ended December 31, 2017.
Non-Founder Series A Common Units
On March 29, 2017, in connection with the Merger and Recapitalization, 563,917 non-founder restricted Series A Common Units were exchanged for
402,963 restricted Series D Common Units. All restricted Series D Common Units continued to vest pursuant to their original vesting period, which was
generally four years, under the restricted Series A Common Units agreement, and the Company will continue to recognize compensation expense over the
related service period.
F-20
In addition, in connection with the exchange of the non-founders’ restricted Series A Common Units into restricted Series D Common Units, the Company
recognized $140 of equity-based compensation expense for vested units, which represents the incremental fair value of the units before and after the
Merger and Recapitalization. The Company will record additional compensation expense in the amount of $115 over the remaining vesting period of the
Series D Common units of which $13, $54 and $48 were recognized during the years ended December 31, 2019, 2018 and 2017, respectively.
The holders of the Series A, B, C and D Common Units were entitled to the following rights and priorities:
Voting Rights
Holders of Series A and B Common Units had the right to one vote per unit held by such member. The Series A Common unitholders as a class were
entitled to elect two of the eight board members while such units are outstanding. The Series B Common unitholders as a class were entitled to elect three
of the eight board members while such units are outstanding.
Holders of Series C and D Common Units did not have the right to vote for the election of board members.
Redemption
The Series A, B, C and D Common Units were not redeemable.
Distributions and Liquidation Preference
The holders of the Series A, B, C and D Common Units were entitled to participate in distributions after preferential distributions were made to the Series 1
and Series 2 Senior Preferred and Junior Preferred unitholders as follows:
•
•
•
•
The Series A, B, C and D Common unitholders were entitled to participate in distributions on a pro rata basis, subject to certain limitations,
until the cumulative amount distributed with respect to one Series A Common Unit, Series B Common Unit, Series C Common Unit and
vested Series D Common Unit equaled the cumulative amount distributed to one Junior Preferred Unit.
The Junior Preferred unitholders and the Series A, B, C and D Common unitholders were entitled to participate in distributions on a pro rata
basis, subject to certain limitations, until the cumulative amount distributed with respect to one Junior Preferred Unit, Series A Common
Unit, Series B Common Unit, Series C Common Unit and vested Series D Common Unit equaled the cumulative amount distributed to one
Series 1 Senior Preferred Unit.
The Series 1 Senior Preferred, Junior Preferred unitholders and the Series A, B, C and D Common unitholders were entitled to participate in
distributions on a pro rata basis, subject to certain limitations, until the cumulative amount distributed with respect to one Series 1 Senior
Preferred Unit, Junior Preferred Unit, Series A Common Unit, Series B Common Unit, Series C Common Unit and vested Series D
Common Unit equaled the cumulative amount distributed to one Series 2 Senior Preferred Unit.
All unitholders were entitled to participate on a pro rata basis in cumulative distributions, subject to certain limitations, in the remaining
proceeds available for distribution.
No distributions were made to the Series A, B, C or D Common unitholders during the years ended December 31, 2019, 2018 and 2017.
F-21
Corporate Conversion
Immediately prior to the effectiveness of the Company’s registration statement on Form S-1, which occurred on January 25, 2018, the Company completed
the Corporate Conversion whereby all the Series A, B, C, and D Common Units converted into shares of common stock.
12. Equity-Based Compensation
2018 Omnibus Incentive Plan
In connection with the closing of the Company’s initial public offering, the board of directors and stockholders approved the 2018 Omnibus Incentive Plan,
which provides for the reservation of 5,001,000 shares of common stock for equity awards.
Stock Options
The following table summarizes the Company’s stock option activity for the year ended December 31, 2019:
Outstanding at December 31, 2018
Granted
Forfeitures
Outstanding at December 31, 2019
Vested and expected to vest as of December 31, 2019
Exercisable at December 31, 2019
Number of
Options
1,153,198 $
1,700,899
(243,122)
2,610,975
2,610,975 $
403,003 $
Weighted
Average
Exercise
Price
28.73
17.72
22.06
22.18
22.18
24.56
At December 31, 2019, the Company had an aggregate of $26,394 of unrecognized equity-based compensation cost related to stock options outstanding
which is expected to be recognized over a weighted average period of 2.7 years. The intrinsic value of stock options was $0 and $2,260 as of December 31,
2019 and 2018, respectively. There were no stock options outstanding as of December 31, 2017.
The fair value of each option award is estimated on the date of grant using the Black-Scholes option-pricing model using the assumptions noted in the
following table for the years ended December 31:
Expected volatility
Expected dividends
Expected term (in years)
Risk-free rate
2019
85.1% - 104.7%
0.0%
5.10 - 6.25
1.5% - 2.6%
2018
73.8% - 86.4%
0.0%
5.25 - 6.25
2.6% - 3.1%
The weighted average fair value of options to purchase shares of common stock granted during the year ended December 31, 2019 and 2018 was $13.13
and $19.94, respectively.
F-22
Restricted Stock Units
In August 2019, the board of directors issued restricted stock units to employees. The restricted stock units vest in two equal installments with fifty percent
vesting six months from the grant date and the remaining fifty percent on the first anniversary of the grant date.
The following table summarizes the Company’s restricted stock unit activity for the year ended December 31, 2019:
Unvested at December 31, 2018
Granted
Forfeitures
Outstanding at December 31, 2019
Unvested as of December 31, 2019
Weighted-
Average
Grant Date
Fair Value
-
5.81
5.81
5.81
5.81
Units
- $
265,800
(20,700)
245,100 $
245,100 $
At December 31, 2019, the Company had an aggregate of $878 of unrecognized equity-based compensation cost related to restricted stock units
outstanding. The unrecognized expense for the restricted stock units is expected to be recognized over a weighted average period of 0.6 years.
Restricted Common Stock
In connection with the Company’s Corporate Conversion on January 25, 2018, all restricted Series B and D common units were converted to restricted
shares of common stock. The following table summarizes the Company’s unvested restricted shares of common stock activity for the year ended December
31, 2019:
Unvested restricted Series D Common Units at
December 31, 2018
Releases
Forfeitures
Unvested restricted Series D Common Units at
December 31, 2019
Weighted-
Average
Grant Date
Fair Value
6.40
6.72
5.21
8.07
Units
743,564 $
(338,198)
(52,414)
352,952 $
The aggregate intrinsic value of restricted common units that vested during the years ended December 31, 2019, 2018, and 2017 were $135, $8,721, and
$3,358 respectively.
At December 31, 2019, the Company had an aggregate of $2,285 of unrecognized equity-based compensation related restricted shares of common stock,
which is expected to be recognized over a weighted average period of 1.4 years.
The Company recorded equity-based compensation expense related to all of its share and unit-based awards to employees and non-employees in the
following captions within its consolidated statements of operations for the years ended December 31, 2019, 2018 and 2017
Research and development expenses
General and administrative expenses
For the Year Ended December 31,
2018
2017
2019
$
$
8,006 $
6,201
14,207 $
4,180 $
1,775
5,955 $
1,206
4,124
5,330
F-23
13. Leases
The Company leases real estate, including laboratories and office space, and certain equipment related to its research and development. The Company’s
leases have remaining lease terms ranging from one to six years. Certain leases include an option to renew, exercisable at the Company’s sole discretion,
with renewal terms that can extend the lease for two years. The Company evaluated the renewal options in its leases to determine if it was reasonably
certain that the renewal option would be exercised, and therefore should be included in the calculation of the operating lease assets and operating lease
liabilities. Given the Company’s current business structure, uncertainty of future growth, and the associated impact to real estate, the Company concluded
that it is not reasonably certain that any renewal options would be exercised. Therefore, the operating lease assets and lease liabilities only contemplate the
initial lease terms.
In January 2018, the Company executed a lease agreement for lab space in Cambridge, Massachusetts. The lease consists of approximately 9,500 square
feet with an initial term of five years with the option to extend the term for one additional two year term. The future minimum rent commitment for the
initial five-year term is approximately $2,600. In addition to rent, the lease requires the Company to pay additional amounts for taxes, insurance,
maintenance and other operating expenses.
In January 2018, the Company executed a lease agreement for office space in Cambridge, Massachusetts. The space serves as the Company’s corporate
headquarters and consists of approximately 16,000 square feet. The term of the lease runs through February 2022. The future minimum rent commitment
for the lease term is approximately $2,700. In addition to rent, the lease requires the Company to pay additional amounts for taxes, insurance, maintenance
and other operating expenses.
In November 2018, the Company entered into a 48-month capital lease for certain lab equipment to be used at its facility in Cambridge Massachusetts. The
future minimum lease commitment for the lease term is approximately $1,100.
In January 2019, the Company executed a lease agreement for additional office space in Cambridge, Massachusetts. The space serves as office space
supporting the Company’s lab operations and consists of approximately 5,000 square feet. The term of the lease runs through October 2025. The future
minimum rent commitment for the lease term is approximately $2,300.
As of December 31, 2019, minimum future lease payments for these operating and finance leases were as follows:
2020
2021
2022
2023
Thereafter
Total
Less: Imputed Interest
Total Lease Liabilities
$
$
Finance Leases
Operating Leases
2,408
2,444
1,424
686
778
7,740
1,590
6,150
276 $
276
276
300
—
1,128
209
919 $
As of December 31, 2018, minimum future lease payments for these operating and finance leases were as follows:
Finance Leases
2019
2020
2021
2022
2023
Thereafter
Total
$
$
Operating Leases
1,934
2,017
2,056
1,023
278
—
7,308
276 $
276
276
485
—
—
1,313 $
The Company recorded rent expense of $2,500, $2,524 and $1,301 for the years ended December 31, 2019, 2018 and 2017, respectively.
Short-term lease and variable lease costs were not material for the year ended December 31, 2019.
F-24
The supplemental disclosure of cash flow information related to the Company’s leases and the weighted average remaining lease term and weighted
average discount rate of the Company’s leases are as follows:
Other information
Cash paid for amounts included in the measurement of lease liabilities
Operating lease liabilities arising from obtaining right-of-use-assets
Finance lease liabilities arising from obtaining right-of-use assets
Weighted-average remaining lease term (in years)
Operating lease
Finance lease
Weighted-average discount rate
Operating lease
Finance lease
14. Commitments and Contingencies
Letter of Credit
For the Year Ended
December 31,
2019
$
$
$
2,288
1,629
—
3.5
3.3
12.5%
10.7%
The Company had outstanding letters of credit in the amounts of $327 and $327 at December 31, 2019 and 2018, respectively, which were required as a
condition of the Company’s office and laboratory leases.
Indemnification Agreements
In the ordinary course of business, the Company may provide indemnification of varying scope and terms to vendors, lessors, business partners and other
parties with respect to certain matters, including, but not limited to, losses arising out of breach of such agreements or from intellectual property
infringement claims made by third parties. In addition, the Company has entered into indemnification agreements with its executive officers and members
of its board of directors that require the Company, among other things, to indemnify them against certain liabilities that may arise by reason of their status
or service as executive officers or directors. The maximum potential amount of future payments the Company could be required to make under these
indemnification agreements is, in many cases, unlimited. To date, the Company has not incurred any material costs as a result of such indemnification
arrangements.
The Company does not believe that the outcome of any claims under indemnification arrangements will have a material effect on its financial position,
results of operations or cash flows, and it has not accrued any liabilities related to such obligations in its consolidated financial statements as of
December 31, 2019 and 2018.
Legal Proceedings
On March 28, 2018, a purported stockholder of the Company, filed a putative class action complaint alleging violations of the federal securities laws, in the
Business Litigation Section of the Superior Court of the Commonwealth of Massachusetts (Civil Action No. 1884-00984), against the Company, Ilan
Ganot, Jennifer Ziolkowski, the Company’s directors and certain of the underwriters in the Company’s initial public offering. The plaintiff in this suit
claims to represent purchasers of the Company’s common stock in or traceable to the Company’s January 25, 2018 initial public offering and seeks
unspecified damages arising out of the alleged failure to disclose risks associated with toxicity and potential for adverse events related to the Company’s
lead product candidate. On April 30, 2018, all defendants including the Company moved to stay the proceedings in favor of the prior-filed federal court
securities class action. The plaintiff filed his opposition to this motion on May 14, 2018, and defendants filed a reply in support of their motion on May 24,
2018. After oral argument on June 13, 2018, the court issued an order on June 22, 2018 allowing the motion to stay and directing the parties to advise the
court of the status of the federal court action every six months. On December 21, 2018, the parties filed a joint status report informing the court of the
voluntary dismissal of the federal actions and of plaintiff’s intent to move to vacate the stay. On June 28, 2019, the plaintiff voluntarily dismissed his claims
without prejudice.
The Company may periodically become subject to other legal proceedings and claims arising in connection with ongoing business activities, including
claims or disputes related to patents that have been issued or that are pending in the field of research on which the Company is focused. Other than the
above action, the Company is not aware of any other material claims as of December 31, 2019.
F-25
15. License Agreements
University of Washington License Agreement
In 2015, the Company entered into a license agreement with the University of Washington, acting through UW CoMotion, under which the Company
obtained an exclusive, royalty-bearing, sublicensable, worldwide license under a patent application owned by the University of Washington relating to
novel micro-dystrophins and all patents claiming priority to such patent to develop, manufacture, and commercialize products for use in the treatment of
DMD and related disease indications caused by a lack of functional dystrophin. The Company has the right to grant sublicenses to third parties contingent
upon written approval by the University of Washington prior to executing such sublicense, which approval may not be unreasonably withheld.
In consideration for the rights granted by the agreement, the Company paid a one-time, non-refundable license fee, which was recorded as a research and
development expense in 2015. The Company is required to reimburse the University of Washington for costs incurred in applying for, prosecuting and
maintaining patents and pay up to an aggregate of approximately $1,000 upon the achievement of certain milestones. In October 2017, the first milestone
was achieved under this agreement. The milestone payment was recorded as a research and development expense in the fourth quarter of 2017. There were
no milestones achieved during the years ended December 31, 2019 and 2018. The Company must also pay royalties of a low single digit percentage of
future sales by the Company and its sublicensees of products developed under the licensed patent rights. In addition, the Company must pay an annual
maintenance fee until certain milestones are achieved, at which time a minimum annual royalty requirement will replace such maintenance fee and will
apply to the Company and its sublicensees.
The license agreement remains in effect until the expiration of the last-to-expire patent licensed under the agreement. The Company may terminate the
agreement at any time upon providing sixty days’ written notice to the University of Washington. The University of Washington may terminate the
agreement upon the Company’s uncured, material breach of the agreement or if the Company enters into an insolvency-related event.
The Company recorded research and development expense in the amount of $38, $47, and $135 for the years ended December 31, 2019, 2018 and 2017,
respectively, under the agreement.
The University of Missouri License Agreement
In 2015, the Company entered into a license agreement with the Curators of the University of Missouri (the “University of Missouri”), a public corporation
of Missouri, under which the Company obtained an exclusive, royalty-bearing, sublicensable, worldwide license under certain patent and patent
applications owned by the University of Missouri relating to a novel synthetic microdystrophin gene to make, sell and distribute products for use in the
treatment of DMD and related disease indications resulting from a lack of functional dystrophin.
In consideration for the rights granted by the agreement, the Company paid a one-time, non-refundable license fee, which was recorded as a research and
development expense in 2015. The Company is required to reimburse the University of Missouri for costs incurred in applying for, prosecuting and
maintaining the licensed patents and pay up to an aggregate of approximately $1,000 upon the achievement of certain milestones for each product
developed based on the licensed patents. In October 2017, the first milestone was achieved under this agreement. The milestone payment was recorded as a
research and development expense in the fourth quarter of 2017. There were no milestones achieved during the years ended December 31, 2019 and 2018.
The Company must pay a royalty of a low single digit percentage of future sales or by its sublicensees of products developed using the licensed patents. In
addition, the Company must pay an annual maintenance fee until certain milestones are achieved, after which time a minimum annual royalty will replace
such maintenance fee.
Under the agreement, the Company granted the University of Missouri a non-exclusive, royalty-free, irrevocable, paid-up license, with the right to grant
sublicenses to non-profit, academic, educational or governmental institutions, to practice and use improvements made by the Company using the licensed
patent rights, solely for non-commercial research purposes.
The license agreement remains in effect until the expiration of the last-to-expire patent or the abandonment of the last to be abandoned patent application
licensed under the agreement. The University of Missouri may terminate the agreement, or render the license granted thereunder non-exclusive, in
individual countries if the Company’s sublicensees fail to achieve certain milestones. The Company may terminate the license agreement at any time upon
providing six months’ written notice to the University of Missouri and paying a termination fee. Each of the University of Missouri and the Company may
also terminate the agreement for an uncured default or breach of the agreement by the other party. The Company’s ability to cure such breach only applies
to the first two notices of such breach provided by the University of Missouri, and thereafter, the University of Missouri may terminate the agreement for
the Company’s default or breach of the agreement upon thirty days’ written notice without an opportunity to cure such default or breach.
F-26
The Company recorded research and development expense in the amount of $23, $10, and $11 for the years ended December 31, 2019, 2018 and 2017,
respectively, under the agreement.
The University of Michigan License Agreement
In 2016, the Company entered into a license agreement with the Regents of the University of Michigan, (the “University of Michigan”), a constitutional
corporation of Michigan, under which the Company obtained an exclusive, royalty-bearing, sublicensable, worldwide license to make, sell and distribute
products under certain patents owned by the University of Michigan related to microdystrophin and utrophin spectrin-like nucleic acid sequences for any
use that, but for this agreement, would comprise an infringement of a valid claim included in the licensed patent rights.
In consideration for the rights granted by the agreement, the Company paid a one-time license fee and a separate fee to cover past patent prosecution costs,
which the Company recorded as a research and development expense in 2016. The Company is required to reimburse the University of Michigan for costs
incurred in applying for, prosecuting and maintaining patents, and pay up to an aggregate of approximately $1,000 upon the achievement of certain
milestones. There were no milestones achieved during the years ended December 31, 2019, 2018 and 2017. The Company must also pay a royalty of a low
single digit percentage on future sales by the Company or its sublicensees of products developed using the licensed rights, with a minimum annual royalty
after certain milestones are achieved. In addition, the Company must pay an annual maintenance fee in any year in which the minimum annual royalty is
not reached.
Under the agreement, the University of Michigan reserves for itself and its affiliates the right to use the licensed rights for non-commercial research, public
service, internal and educational purposes and the right to grant the same limited non-commercial rights to other non-profit research institutions.
The license agreement remains in effect until the expiration of the last-to-expire patent licensed under the agreement. The University of Michigan may
terminate the agreement upon the Company’s uncured material breach of the agreement, including failure to make required payments under the agreement
or to achieve certain milestones, or if the Company becomes insolvent or bankrupt. The Company may terminate the license agreement at any time upon
providing sixty days’ written notice to the University of Michigan.
The Company recorded and research and development expense in the amount of $39, $35 and $4 for the years ended December 31, 2019, 2018 and 2017,
respectively, under the agreement.
Harvard College License Agreements
In 2016, the Company entered into a license agreement with the President and Fellows of Harvard College, (“Harvard College”), under which the Company
obtained a non-exclusive, royalty-bearing, sublicensable, worldwide license to use certain intellectual property owned by Harvard College to develop,
manufacture, and commercialize products for use in the treatment of DMD.
In consideration for the rights granted by the agreement, the Company paid a one-time, non-refundable license fee, which was recorded as a research and
development expense in 2016. The Company is required to pay an annual license maintenance fee until certain milestones are achieved, after which time
the annual maintenance fee will increase annually. Such annual maintenance fee will further increase if the Company grants certain rights to a sublicensee
or strategic partner with whom the Company collaborates on the development and commercialization of licensed products. The annual maintenance fee is
creditable against royalty payments. The Company also must pay a milestone payment within thirty days after achieving certain milestones. There were no
milestones achieved during the years ended December 31, 2019, 2018 and 2017. The Company must pay a royalty of a low single digit percentage on
future sales by the Company or its sublicensees of products developed using the licensed technology.
The license agreement remains in effect for an initial term of fifteen years, with automatic three-year renewal periods thereafter unless one of the parties
provides notice of non-renewal. The Company may terminate the license agreement at any time upon providing sixty days’ written notice to Harvard
College. Harvard College may terminate the agreement in the event the Company becomes bankrupt or insolvent. Both Harvard College and the Company
may also terminate the agreement for an uncured material breach of the agreement by the other party.
F-27
The Company recorded research and development expense in the amount of $10, $20 and $45 for the years ended December 31, 2019, 2018 and 2017,
respectively, under the agreement.
In August 2017, the Company entered into another license agreement with Harvard College, under which the Company obtained a non-exclusive, royalty-
bearing, sublicensable, worldwide license to use certain intellectual property owned by Harvard College to develop, manufacture, and commercialize
products for use in the treatment of DMD.
In consideration for the rights granted by the agreement, the Company paid a one-time, non-refundable license fee, which was recorded as a research and
development expense in 2017. The Company is required to pay an annual license maintenance fee until certain milestones are achieved, after which time
the annual maintenance fee will increase annually. Such annual maintenance fee will further increase if the Company grants certain rights to a sublicensee
or strategic partner with whom the Company collaborates on the development and commercialization of licensed products. The annual maintenance fee is
creditable against royalty payments. The Company also must pay a milestone payment within thirty days after achieving certain milestones. There were no
milestones achieved during the years ended December 31, 2019, 2018 and 2017. The Company must pay a royalty of a low single digit percentage on
future sales by the Company or its sublicensees of products developed using the licensed technology.
The license agreement remains in effect for an initial term of fifteen years, with automatic three-year renewal periods thereafter unless one of the parties
provides notice of non-renewal. The Company may terminate the license agreement at any time upon providing sixty days’ written notice to Harvard
College. Harvard College may terminate the agreement in the event the Company becomes bankrupt or insolvent. Both Harvard College and the Company
may also terminate the agreement for an uncured material breach of the agreement by the other party.
The Company recorded research and development expense in the amount of $5, $5 and $23 for the years ended December 31, 2019, 2018 and 2017,
respectively, under the agreement.
Other License Agreements
In 2016, the Company entered into a license agreement with Life Technologies Corporation, (“Life Technologies”). In consideration for obtaining a non-
exclusive, royalty-free, worldwide license to use certain technologies and associated know-how to develop product candidates, the Company paid a one-
time, non-refundable license fee. This fee was recorded as a research and development expense in 2016. The license agreement will remain effective in
perpetuity unless earlier terminated. Life Technologies has the right to terminate the agreement upon the Company’s material, uncured breach of the
agreement or in the event that it determines that continued performance of the agreement may violate any laws. The Company is obligated to diligently
pursue regulatory approval necessary for the development, manufacture and sale of the licensed products. The Company has the right to terminate the
agreement at any time upon providing thirty days’ written notice to Life Technologies.
16. Net Loss per Share
Basic and diluted net loss per share attributable to common stockholders were calculated as follows:
The numerator for basic and diluted net loss per share attributable to common stockholders is as follows:
Net loss
Net loss attributable to non-controlling interest
Net loss attributable to Solid Biosciences Inc.
Decretion (accretion) of preferred units to redemption
value
Redemption of preferred units
Redemption of redeemable interest from non-controlling
interest in Solid GT
Net loss attributable to common stockholders
For the Year Ended December 31,
2018
2019
(117,223) $
-
(117,223) $
$
$
(74,798) $
-
(74,798) $
2017
(53,178)
(1,060)
(52,118)
-
-
-
-
(959)
15,685
-
(117,223) $
-
(74,798) $
(1,925)
(39,317)
$
F-28
The denominator is as follows:
Weighted average common stock outstanding, basic and
diluted
Weighted average pre-funded warrants to purchase common stock
Total
39,326,983
962,307
40,289,290
33,262,597
-
33,262,597
13,649,485
-
13,649,485
For the Year Ended December 31,
2018
2017
2019
Net loss per share attributable to common stockholders, basic and diluted is as follows:
Net loss per share attributable to common stockholders,
basic and diluted
$
(2.91) $
(2.25) $
(2.88)
For the Year Ended December 31,
2018
2017
2019
The following potential common stock equivalents, presented based on amounts outstanding at each period end, were excluded from the calculation of
diluted net loss per share attributable to common stockholders for the periods indicated because including them would have had an anti-dilutive effect:
Options to purchase shares of common stock
Unvested restricted stock units
Unvested shares of common stock
Series D common units
For the Year Ended December 31,
2018
1,153,198
-
743,564
-
1,896,762
2019
2,610,975
245,100
352,952
-
3,209,027
2017
-
-
-
1,404,265
1,404,265
17. Income Taxes
The Company recorded no tax benefit for the years ended December 31, 2019 and 2018 for the net operating losses incurred due to its uncertainty of
realizing a benefit from those items.
A reconciliation of income taxes computed using the U.S. federal statutory rate to that reflected in operations as of December 31, 2019 and 2018 is as
follows:
Income tax computed at federal statutory tax rate
State taxes, net of federal benefit
Permanent differences
Tax credits
Loss taxed as a partnership
Conversion to a C-Corporation
Other
Valuation allowance
December 31, 2019
December 31, 2018
21.0%
6.1%
(0.4)%
10.9%
-
-
(0.1)%
(37.5)%
0.0%
21.0%
5.5%
(0.8)%
9.2%
(1.7)%
0.5%
(0.1)%
(33.6)%
0.0%
F-29
The Company established deferred tax assets and liabilities on identified book to tax temporary differences as of the date of conversion to a C-corporation.
Deferred income taxes reflect the net tax effects of these temporary differences between the carrying amounts of assets and liabilities for financial reporting
purposes and the amounts used for income tax purposes. Significant components of the Company’s net deferred tax assets as of December 31, 2019 and
2018 are as follows:
Deferred tax assets:
Tax loss carryforwards
Tax credit carryforwards
Deferred expenses
Accrued expenses
Stock compensation
Other
Total deferred tax assets
Right of use asset
Depreciation
Valuation allowance
Net deferred taxes
December 31, 2019
December 31, 2018
$
$
43,885
19,717
1,920
921
4,159
257
70,859
(1,355)
(333)
(69,171)
-
$
$
16,417
6,903
372
935
856
177
25,660
-
(406)
(25,254)
-
As of December 31, 2019 and 2018, the Company has federal net operating loss carryforwards of $160,568 and $59,357 and tax credits of $18,715 and
$6,349, respectively which may be used to offset future federal income and tax liability, respectively. In addition, the Company has state net operating loss
carryforwards of approximately $157,104 and $59,453 and tax credits of $1,269 and $702 for the years ended December 31, 2019 and 2018, respectively,
which may be used to offset future state income and tax liability, respectively. The Company’s ability to utilize these federal and state carryforwards may
be limited in the future if the Company experiences an ownership change pursuant to Internal Revenue Code Section 382. Ownership changes, as defined
in the Internal Revenue Code, including those resulting from the issuance of common stock in connection with the Company’s public offerings, may limit
the amount of net operating loss and tax credit carryforwards that can be utilized to offset future taxable income or tax liability. The Company has not
conducted a study to assess whether a change of control has occurred or whether there have been multiple changes of control since inception due to the
significant complexity and cost associated with such a study. If the Company has experienced a change of control, as defined by Section 382, at any time
since inception, utilization of the net operating loss carryforwards tax credit carryforwards would be subject to an annual limitation under Section 382. Any
limitation may result in expiration of a portion of the net operating loss carryforwards or research and development tax credit carryforwards before
utilization. Further, until a study is completed and any limitation is known, no amounts are being presented as an uncertain tax position.
A valuation allowance is recorded against deferred tax assets if it is more likely than not that some or all of the deferred tax assets will not be realized. The
Company has evaluated the positive and negative evidence bearing upon the realizability of the deferred tax assets. The Company concluded, in accordance
with the applicable accounting standards, that it is more likely than not that the Company will not realize the benefit of its deferred tax assets. Accordingly,
the Company has recorded a full valuation allowance against its deferred tax assets. The Company had approximately $69,171 and $25,254 in valuation
allowances recorded against its deferred tax assets as of December 31, 2019 and 2018, respectively.
The Company files tax returns as prescribed by the tax laws of the jurisdictions in which it operates. In the normal course of business, the Company is
subject to examination by federal and state jurisdictions, where applicable. There are currently no pending income tax examinations. The Company’s C-
Corporation tax years beginning with the year ended December 31, 2018 are open under statute. Any tax credit or net operating loss carryforward can be
adjusted in future periods after the respective year of generation’s statute of limitation has closed.
As of December 31, 2019 and 2018, the Company did not have unrecognized tax benefits. The Company recognizes interest and penalties related to income
taxes as a component of income tax expense. As of December 31, 2019 and 2018, no interest and penalties have been recorded.
F-30
18. Retirement Plan
The Company has a defined contribution savings plan under Section 401(k) of the Internal Revenue Code. The plan covers substantially all employees who
meet minimum age and service requirements and allows participants to defer a portion of their annual compensation on a pretax basis. Company
contributions to the plan may be made at the discretion of the Company’s board of managers. The Company made no contributions to the plan during the
years ended December 31, 2019, 2018 and 2017.
19. Selected Quarterly Financial Information (Unaudited)
Selected quarterly results from operations for the years ended December 31, 2019 and 2018 are as follows:
2019 Quarter Ended
March
31
June
30
September
30
December
31
Revenues
Operating expenses
Loss from operations
Net loss
Net loss per share attributable to common stockholders
Revenues
Operating expenses
Loss from operations
Net loss
Net loss per share attributable to common
stockholders
20. Subsequent Events
$
$
$
- $
30,302
(30,302)
(29,582)
(0.85) $
(in thousands, except for per share data)
- $
26,969
(26,969)
(26,525)
(0.76) $
- $
29,717
(29,717)
(29,255)
(0.67) $
-
32,330
(32,330)
(31,861)
(0.67)
2018 Quarter Ended
March
31
June
30
September
30
December
31
- $
15,973
(15,973)
(15,877)
(in thousands, except for per share data)
- $
18,178
(18,178)
(17,980)
- $
19,150
(19,150)
(19,020)
-
22,386
(22,386)
(21,921)
$
(0.54) $
(0.52) $
(0.55) $
(0.63)
In January 2020, the Company implemented changes to its organizational structure to create a leaner company focused on advancing SGT-001. In
connection with the restructuring, the Company made changes to its management team and reduced headcount by approximately 30 percent. The Company
expects to substantially complete the restructuring in the first quarter of 2020. The Company estimates total restructuring costs of approximately $2.1
million related to severance and other employee termination benefits. The Company expects that approximately $1.2 million would be paid during the
three months ended March 31, 2020 and approximately $0.9 million would be paid during the remainder of 2020.
F-31
Exhibit 4.2
DESCRIPTION OF SECURITIES REGISTERED UNDER SECTION 12 OF THE SECURITIES EXCHANGE ACT OF 1934, AS
AMENDED
The following description of the securities of Solid Biosciences Inc. (“us,” “our,” “we” or the “Company”) registered under Section 12 of the
Securities Exchange Act of 1934, as amended (the “Exchange Act”), is intended as a summary only and therefore is not a complete description. This
description is based upon, and is qualified by reference to, our certificate of incorporation (“charter”), our bylaws and applicable provisions of the
Delaware General Corporation Law (the “DGCL”). You should read our charter and bylaws, which are incorporated by reference as Exhibit 3.1 and Exhibit
3.2, respectively, to the Annual Report on Form 10-K of which this Exhibit 4.2 is a part, for the provisions that are important to you.
Authorized Capital Stock
Our authorized capital stock consists of 300,000,000 shares of common stock, par value $0.001 per share, and 10,000,000 shares of preferred stock,
par value $0.001 per share, all of which preferred stock is undesignated. Our common stock is registered under Section 12(b) of the Exchange Act.
Common Stock
Voting Rights. Each holder of our common stock is entitled to one vote for each share on all matters submitted to a vote of the stockholders,
including the election of directors. Our stockholders do not have cumulative voting rights in the election of directors. Accordingly, holders of a majority of
the voting shares are able to elect all of the directors.
Dividends. Subject to preferences that may be applicable to any then-outstanding preferred stock, holders of our common stock are entitled to
receive dividends, if any, as may be declared from time to time by our board of directors out of legally available funds.
Liquidation. In the event of our liquidation, dissolution or winding up, holders of our common stock will be entitled to share ratably in the net assets
legally available for distribution to stockholders after the payment of all of our debts and other liabilities and the satisfaction of any liquidation preference
granted to the holders of any then-outstanding shares of preferred stock.
Other Rights. Holders of our common stock have no preemptive, conversion, subscription or other similar rights, and there are no redemption or
sinking fund provisions applicable to our common stock. The rights, preferences and privileges of the holders of our common stock are subject to and may
be adversely affected by the rights of the holders of shares of any series of our preferred stock that we may designate in the future.
Preferred Stock
We are authorized to issue up to 10,000,000 shares of “blank check” preferred stock, which may be issued in one or more series upon
authorization of our board of directors. Our board of directors is authorized to fix the designations, powers, preferences and the relative, participating,
optional or other special rights and any qualifications, limitations and restrictions of the shares of each series of preferred stock. The authorized shares of
our preferred stock are available for issuance without further action by our stockholders, unless such action is required by applicable law or the rules of any
stock exchange on which our securities may be listed. If the approval of our stockholders is not required for the issuance of shares of our preferred stock,
our board may determine not to seek stockholder approval. A series of our preferred stock could, depending on the terms of such series, impede the
completion of a merger, tender offer or other takeover attempt.
Provisions of Our Certificate of Incorporation and Bylaws and the DGCL That May Have Anti-Takeover Effects
Board of Directors Vacancies: Our charter and bylaws authorize only our board of directors to fill vacant directorships, including newly created
seats. In addition, the number of directors constituting our board of directors may only be set by a resolution adopted by a majority vote of our entire board
of directors. These provisions would prevent a stockholder from increasing the size of our board of directors and then gaining control of our board of
directors by filling the resulting vacancies with its own nominees. This makes it more difficult to change the composition of our board of directors but
promotes continuity of management.
Classified Board: Our charter and bylaws provide that our board of directors will be classified into three classes of directors, each with staggered
three-year terms. A third party may be discouraged from making a tender offer or otherwise attempting to obtain control of us as it is more difficult and
time consuming for stockholders to replace a majority of the directors on a classified board of directors.
Stockholder Action; Special Meetings of Stockholders: Our charter provides that our stockholders may not take action by written consent, but
may only take action at annual or special meetings of our stockholders. As a result, a holder controlling a majority of our capital stock would not be able to
amend our bylaws or remove directors without holding a meeting of our stockholders called in accordance with our bylaws. Further, our bylaws and charter
will provide that special meetings of our stockholders may be called only by a majority of our board of directors, the Chairman of our board of directors or
our Chief Executive Officer, thus prohibiting a stockholder from calling a special meeting. These provisions might delay the ability of our stockholders to
force consideration of a proposal or for stockholders controlling a majority of our capital stock to take any action, including the removal of directors.
Advance Notice Requirements for Stockholder Proposals and Director Nominations: Our bylaws provide advance notice procedures for
stockholders seeking to bring matters before our annual meeting of stockholders or to nominate candidates for election as directors at our annual meeting of
stockholders. Our bylaws also specify certain requirements regarding the form and content of a stockholder’s notice. These provisions might preclude our
stockholders from bringing matters before our annual meeting of stockholders or from making nominations for directors at our annual meeting of
stockholders if the proper procedures are not followed. We expect that these provisions might also discourage or deter a potential acquirer from conducting
a solicitation of proxies to elect the acquirer’s own slate of directors or otherwise attempting to obtain control of our company.
Supermajority Voting: The DGCL provides, generally, that the affirmative vote of a majority of the shares entitled to vote on any matter is required
to amend a corporation’s certificate of incorporation or bylaws, unless a corporation’s certificate of incorporation or bylaws, as the case may be, requires a
greater percentage. Our bylaws may be amended or repealed by a majority vote of our board of directors or the affirmative vote of the holders of at least
two-thirds of the votes that all our stockholders would be entitled to cast in an annual election of directors. In addition, the affirmative vote of the holders of
at least two-thirds of the votes that all our stockholders would be entitled to cast in an election of directors is required to amend or repeal or to adopt certain
provisions of our charter.
No Cumulative Voting: The DGCL provides that stockholders are not entitled to the right to cumulate votes in the election of directors unless a
corporation’s certificate of incorporation provides otherwise. Our charter does not provide for cumulative voting.
Removal of Directors Only for Cause: Our charter provides that stockholders may remove directors only for cause and only by the affirmative vote
of the holders of at least two-thirds of our outstanding common stock.
Delaware Business Combination Statute. We are subject to the provisions of Section 203 of the DGCL, regulating corporate takeovers. In general,
DGCL Section 203 prohibits a publicly held Delaware corporation from engaging in a business combination with an interested stockholder for a period of
three years following the date on which the person became an interested stockholder unless (i) prior to the date of the transaction, the board of directors of
the corporation approved either the business combination or the transaction which resulted in the stockholder becoming an interested stockholder; (ii) upon
consummation of the transaction that resulted in the stockholder becoming an interested stockholder, the interested stockholder owned at least 85% of the
voting stock of the corporation outstanding at the time the transaction commenced, excluding for purposes of determining the voting stock outstanding, but
not the outstanding voting stock owned by the interested stockholder, (a) shares owned by persons who are directors and also officers and (b) shares owned
by employee stock plans in which employee participants do not have the right to determine confidentially whether shares held subject to the plan will be
tendered in a tender or exchange offer; or (iii) at or subsequent to the date of the transaction, the business combination is approved by the board of directors
of the corporation and authorized at an annual or special meeting of stockholders, and not by written consent, by the affirmative vote of at least two-thirds
of the outstanding voting stock that is not owned by the interested stockholder.
Generally, a business combination includes a merger, asset or stock sale, or other transaction or series of transactions together resulting in a financial
benefit to the interested stockholder. An interested stockholder is a person who, together with affiliates and associates, owns or, within three years prior to
the determination of interested stockholder status, did own 15% or more of a corporation’s outstanding voting stock. We expect the existence of this
provision to have an anti-takeover effect with respect to transactions our board of directors does not approve in advance. We also anticipate that DGCL
Section 203 may also discourage attempts that might result in a premium over the market price for the shares of common stock held by stockholders.
Exclusive Forum Selection. Our charter provides that the Court of Chancery of the State of Delaware will be the exclusive forum for: any derivative
action or proceeding brought on our behalf; any action asserting a breach of fiduciary duty; any action asserting a claim against us arising pursuant to the
DGCL, our charter or our bylaws; any action to interpret, apply, enforce or determine the validity of our charter or our bylaws; or any action asserting a
claim against us that is governed by the internal affairs doctrine. We do not intend to have this choice of forum provision apply to, and this choice of forum
provision will not apply to, actions arising under the Securities Act of 1933, as amended, or the Exchange Act.
2
CONSULTING AGREEMENT
Exhibit 10.24
In consideration and as a condition of Andrey Zarur, an individual having an address at [*] (the “Consultant”),
providing consulting services to Solid Biosciences Inc., a Delaware corporation (together with its affiliates, the “Company”),
having an address at 141 Portland Street, Fifth Floor, Cambridge, MA 02139, the Consultant hereby agrees with the Company as
follows:
1.
Relationship. During the Term (as defined below), Consultant shall devote reasonable time and efforts to the
Company’s business, consistent with Consultant’s other business responsibilities. Consultant’s efforts on behalf of the Company
shall, accordingly, be on a part‑time basis. Consultant’s duties are described in Exhibit A hereto (the “Services”). Consultant
represents that his service as a consultant to the Company and Consultant’s performance of the Services and all of the terms of
this Agreement do not and will not violate any other legal obligation or any agreement to which Consultant is or shall become a
party.
2.
Compensation.
(a)
The Company shall pay Consultant a consulting fee at the annual rate of $100,000, payable in
equal monthly instalments in arrears. Company shall reimburse Consultant for all reasonable travel and out-of-pocket expenses
incurred by Consultant in performing Services pursuant to this Agreement, upon submission of appropriate supporting
documentation.
(b)
In addition, Company shall grant to Consultant, subject to the approval of the Company’s Board of
Directors, a nonqualified stock option under Company’s 2018 Omnibus Incentive Plan for the purchase of 10,000 shares of
Company’s common stock at an exercise price per share equal to the fair market value of a share of such common stock on the
date of grant, as calculated under such Plan.
3.
Work-Made-for Hire.
(a)
All right, title and interest in any and all writings, ideas, inventions, know-how, designs, improvements or
other property created during Consultant’s consulting relationship relating directly to the business of the Company, constituting
copyrights, patents, trademarks, service marks and related rights or other forms of proprietary rights or information (regardless of
whether any such copyrights, patents, trademarks and service marks or other rights have or may be registered) that are created,
adapted or improved by Consultant (whether alone or in conjunction with any other person or employee), and all material created
during Consultant’s consulting relationship that includes any of the foregoing (collectively, “Covered Material”), shall be owned
by the Company and to the extent that it includes copyrightable subject matter, shall be deemed a work made for hire for the
Company within the meaning of the United States Copyright Act of 1976 and for all other purposes. If any Covered Material is
deemed not to be work made for hire, such Covered Material is hereby assigned by Consultant to the Company and Consultant
shall not have or claim to have, under this Agreement or otherwise, any right, title or interest of any kind or nature whatsoever in
such Covered Material.
- 2 -
(b)
The Company shall have the right to apply for and obtain registrations in the United States Copyright Office
and the United States Patent and Trademark Office, in its own or its designee’s name, of its rights in any or all of the Covered
Material. If for any reason the rights in any Covered Material are registered, or applied to be registered, in Consultant’s name,
Consultant shall assign in writing such application or registration to the Company and hereby authorizes and appoints the
Company its agent for the purpose of recording such assignment.
(c)
Whenever the Company shall so request, whether during or after Consultant’s consulting relationship,
Consultant shall execute, acknowledge and deliver all applications, assignments or other instruments; make or cause to be made
all rightful oaths; testify in all legal proceedings; communicate all known facts which relate to such works, copyrights,
inventions, ideas, discoveries, designs and improvements; perform all lawful acts and otherwise render all such assistance as the
Company may deem necessary to protect the Company’s interest therein including any assistance which the Company shall deem
necessary in connection with any proceeding or litigation involving the same. The Company shall reimburse Consultant for all
reasonable out-of-pocket costs, incurred by Consultant in rendering any such assistance requested by the Company pursuant to
this Section.
4.
Nondisclosure. Consultant shall not at any time, whether during or after the termination of Consultant’s
consulting relationship, regardless of the reason for such termination, reveal to any person or entity any Confidential Information
except to employees of the Company who need to know such Confidential Information for the purposes of their employment, or
as otherwise authorized by the Company in writing. For purposes of this Agreement, the term “Confidential Information” means
any and all information and derivative information, in whatever form or medium, including oral information, concerning or
relating to the Company or information of any third party that the Company is under an obligation to keep confidential or that is
maintained by the Company as confidential, including, without limitation, intellectual property of the Company, such as, but not
limited to, patent applications, copyrights, copyright applications, and trade secrets; information regarding or resulting from
research and development activities performed by or on behalf of the Company and other projects (such as, but not limited to,
preclinical and clinical data, design details and specifications, engineering information, and works in process); and business and
financial information (such as, but not limited to, current, future, and proposed products and services, financial information and
models, information relating to procurement requirements, purchasing, manufacturing, investors, customer lists, customers,
suppliers, facilities, product plans, product ideas, business strategies, marketing or business plans, financial or personnel matters,
investors, employees, business and contractual relationships, business forecasts, sales, strategies, operations, policies, procedures,
commercialization capabilities, and information regarding third parties). Notwithstanding the foregoing, Confidential Information
does not include information that Consultant can demonstrate: (a) is publicly known and generally available in the public domain
other than in consequence of improper action by any person; or (b) was acquired by Consultant free and clear of any duty of
confidentiality or restricted use and without improper action by the transferor of such information or any other
person. Consultant shall keep confidential all matters entrusted to Consultant by or on behalf of the Company and shall not use
or attempt to use any Confidential Information except as may be required in the ordinary course of performing Consultant’s
duties as a consultant to the Company, and Consultant shall not use any Confidential Information in any manner that may injure
or cause loss or may be calculated to injure or cause loss to the Company, whether directly or indirectly.
- 3 -
Nothing in this Agreement prohibits Consultant from reporting possible violations of federal or state law or regulations to any
governmental agency or entity or self-regulatory institution, including but not limited to the Equal Employment Opportunity
Commission, the National Labor Relations Board, the Department of Justice, the Securities and Exchange Commission,
Congress, and any Inspector General, or making other disclosures that are protected under the whistleblower provisions of federal
or state law or regulation. Prior authorization of the Company shall not be required to make any such reports or disclosures and
Consultant is not required to notify the Company that Consultant has made such reports or disclosures.
5.
Term of Consulting Relationship. The term of this Agreement shall be from January 1, 2020 to December 31,
2020 (the “Term”). Notwithstanding the foregoing, either party may terminate Consultant’s consulting relationship and this
Agreement at any time, for any or no reason, on at least thirty (30) days’ prior notice, and the Company may terminate
Consultant’s consulting relationship and this Agreement for “Cause” immediately upon notice. For purposes of this Agreement,
a termination shall be for Cause if any one or more of the following has occurred:
(i) Consultant has committed an act of fraud, embezzlement, misappropriation or breach of fiduciary duty
against the Company;
(ii) Consultant has been convicted of, or pleaded guilty or nolo contendere to, any crime triable upon indictment
or involving moral turpitude; or
(iii) Consultant has engaged in the unlawful use (including being under the influence) or possession of illegal
drugs on the Company’s premises.
6.
Independent Contractor.
Consultant is an independent contractor and not an employee of the
Company. Consultant shall be responsible for all taxes arising from compensation and other amounts paid under this
Agreement. Neither federal, state or local income tax, nor payroll tax of any kind, shall be withheld or paid by the Company on
Consultant’s behalf. Consultant will not be eligible for, and shall not participate in, any employee pension, health, welfare, or
other fringe benefit plan of the Company.
7.
Waiver; Amendments. Any waiver by the Company of a breach of any provision of this Agreement shall not
operate or be construed as a waiver of any subsequent breach of such provision or any other provision hereof. In addition, any
amendment to or modification of this Agreement or any waiver of any provision hereof must be agreed to in writing by both
parties.
8.
Severability. Consultant agrees that each provision and the subparts of each provision herein shall be treated
as separate and independent clauses, and the unenforceability of any one clause shall in no way impair the enforceability of any
of the other clauses of the Agreement. Moreover, if one or more of the provisions contained in this Agreement shall for any
reason be held to be excessively broad as to scope, activity, subject or otherwise so as to be unenforceable at law, such provision
or provisions shall be construed by the appropriate judicial body by limiting or reducing it or them, so as to be enforceable to the
maximum extent compatible with the applicable law as it shall then appear. Consultant hereby further agrees that the language of
all parts of this Agreement shall in all cases be construed as a whole according to its fair meaning and not strictly for or against
either of the parties.
- 4 -
9.
Survival. Consultant’s obligations under Sections 3 and 4 of this Agreement shall survive the termination of
Consultant’s consulting relationship regardless of the reason for or manner of such termination and shall be binding upon
Consultant’s successors, heirs, executors, administrators and legal representatives.
10.
Assignment. The Company shall have the right to assign this Agreement to its successors and assigns, and
all covenants and agreements hereunder shall inure to the benefit of and be enforceable by said successors or assigns. Consultant
may not assign this Agreement.
11.
Governing Law. This Agreement shall be governed by and construed in accordance with the laws of the
Commonwealth of Massachusetts and shall in all respects be interpreted, enforced and governed under the internal and domestic
laws of Massachusetts, without giving effect to the principles of conflicts of laws of such state. The laws of the Commonwealth
of Massachusetts shall govern any claims or legal actions by one party against the other arising out of the relationship between
the parties contemplated herein (whether or not arising under this Agreement).
12.
Entire Agreement. This Agreement sets forth the complete, sole and entire agreement between the parties
with respect to the subject matter herein and supersedes any and all other agreements, negotiations, discussions, proposals, or
understandings, whether oral or written, previously entered into, discussed or considered by the parties.
/s/ Andrey Zarur
Andrey Zarur
Solid Biosciences Inc.
By:
/s/ Ilan Ganot
Ilan Ganot, CEO
The role will entail the following responsibilities: Work closely with the CEO in a number of key areas, which may include
advice on strategic, financial, intellectual property, clinical and manufacturing issues.
EXHIBIT A
Description of Services
CONSULTING AGREEMENT
Exhibit 10.25
In consideration and as a condition of Alvaro Amorrortu, an individual having an address at [*] (the “Consultant”),
providing consulting services to Solid Biosciences Inc., a Delaware corporation (together with its affiliates, the “Company”),
having an address at 141 Portland Street, Fifth Floor, Cambridge, MA 02139, the Consultant hereby agrees with the Company as
follows:
1.
Relationship. During the Term (as defined below), Consultant shall devote reasonable time and efforts to the
Company’s business, consistent with Consultant’s other business responsibilities. Consultant’s efforts on behalf of the Company
shall, accordingly, be on a part‑time basis. Consultant’s duties are described in Exhibit A hereto and shall be performed on an as-
needed basis, as requested by the Company (the “Services”). Consultant represents that his service as a consultant to the
Company and Consultant’s performance of the Services and all of the terms of this Agreement do not and will not violate any
other legal obligation or any agreement to which Consultant is or shall become a party. In performing the Services, Consultant
shall comply, to the best of his knowledge, with all business conduct, regulatory, ethical and health and safety guidelines
established by any governmental authority with respect to the Company’s business.
2.
Compensation. Consultant shall invoice the Company monthly in arrears on the basis of time dedicated to the
Services, and such invoice shall include a description of activities performed, including the date or dates of the Services. The
Company shall pay Consultant the consulting fees within thirty (30) days after receipt of the applicable invoice. The consulting
fee will be $500 per hour. Company shall reimburse Consultant for all reasonable travel and out-of-pocket expenses incurred by
Consultant in performing Services pursuant to this Agreement that are preapproved by the Company in writing, upon submission
of appropriate supporting documentation. Air travel of over five hours will be reimbursed at business class rates, and all other air
travel at economy rates. The Consultant must obtain prior written authorization from the Company before booking such travel.
3.
Work-Made-for Hire.
(a)
All right, title and interest in any and all writings, ideas, inventions, know-how, designs, improvements or
other property created during Consultant’s consulting relationship relating in any way to the assets, business or operations of the
Company, constituting copyrights, patents, trademarks, service marks and related rights or other forms of proprietary rights or
information (regardless of whether any such copyrights, patents, trademarks and service marks or other rights have or may be
registered) that are created, adapted or improved by Consultant (whether alone or in conjunction with any other person or
employee), and all material created during Consultant’s consulting relationship that includes any of the foregoing (collectively,
“Covered Material”), shall be owned by the Company and to the extent that it includes copyrightable subject matter, shall be
deemed a work made for hire for the Company within the meaning of the United States Copyright Act of 1976 and for all other
purposes. If any Covered Material is deemed not to be work made for hire, such Covered Material is hereby assigned by
Consultant to the Company and Consultant shall not have or claim to have, under this Agreement or otherwise, any right, title or
interest of any kind or nature whatsoever in such Covered Material.
- 2 -
(b)
The Company shall have the right to apply for and obtain registrations in the United States Copyright Office
and the United States Patent and Trademark Office, in its own or its designee’s name, of its rights in any or all of the Covered
Material. If for any reason the rights in any Covered Material are registered, or applied to be registered, in Consultant’s name,
Consultant shall assign in writing such application or registration to the Company and hereby authorizes and appoints the
Company its agent for the purpose of recording such assignment.
(c)
Whenever the Company shall so request, whether during or after Consultant’s consulting relationship,
Consultant shall execute, acknowledge and deliver all applications, assignments or other instruments; make or cause to be made
all rightful oaths; testify in all legal proceedings; communicate all known facts which relate to such works, copyrights,
inventions, ideas, discoveries, designs and improvements; perform all lawful acts and otherwise render all such assistance as the
Company may deem necessary to protect the Company’s interest therein including any assistance which the Company shall deem
necessary in connection with any proceeding or litigation involving the same. The Company shall reimburse Consultant for all
reasonable out-of-pocket costs (such as travel, meals, and lodging), incurred by Consultant in rendering any such assistance
requested by the Company pursuant to this Section.
4.
Nondisclosure. Consultant shall not at any time, whether during or after the termination of Consultant’s
consulting relationship, regardless of the reason for such termination, directly or indirectly reveal to any person or entity any
Confidential Information except to employees of the Company who need to know such Confidential Information for the purposes
of their employment, or as otherwise authorized by the Company in writing. For purposes of this Agreement, the term
“Confidential Information” means any and all information and derivative information, in whatever form or medium, including
oral information, concerning or relating to the Company or information of any third party that the Company is under an
obligation to keep confidential or that is maintained by the Company as confidential or proprietary, including, without limitation,
intellectual property of the Company, such as, but not limited to, patent applications, copyrights, copyright applications, and trade
secrets; information regarding or resulting from research and development activities performed by or on behalf of the Company
and other projects (such as, but not limited to, preclinical and clinical data, design details and specifications, engineering
information, and works in process); and business and financial information (such as, but not limited to, current, future, and
proposed products and services, financial information and models, information relating to procurement requirements, purchasing,
manufacturing, investors, customer lists, customers, suppliers, facilities, product plans, product ideas, business strategies,
marketing or business plans, financial or personnel matters, investors, employees, business and contractual relationships, business
forecasts, sales, strategies, operations, policies, procedures, commercialization capabilities, and information regarding third
parties). Notwithstanding the foregoing, Confidential Information does not include information that Consultant can demonstrate:
(a) is publicly known and generally available in the public domain other than in consequence of improper action by any person;
or (b) was acquired by Consultant free and clear of any duty of confidentiality or restricted use and without improper action by
the transferor of such information or any other person. Consultant shall keep confidential all matters entrusted to Consultant by
or on behalf of the Company and shall not, directly or indirectly, for himself or for any other person or entity, use or attempt to
use any Confidential Information except as may be required in the ordinary course of performing Consultant’s duties as a
consultant to the Company, and Consultant
- 3 -
shall not, directly or indirectly, use any Confidential Information in any manner that may injure or cause loss or may be
calculated to injure or cause loss to the Company, whether directly or indirectly. Nothing in this Agreement prohibits Consultant
from reporting possible violations of federal or state law or regulations to any governmental agency or entity or self-regulatory
institution, including but not limited to the Equal Employment Opportunity Commission, the National Labor Relations Board, the
Department of Justice, the Securities and Exchange Commission, Congress, and any Inspector General, or making other
disclosures that are protected under the whistleblower provisions of federal or state law or regulation. Prior authorization of the
Company shall not be required to make any such reports or disclosures and Consultant is not required to notify the Company that
Consultant has made such reports or disclosures.
5.
Company Property. Consultant agrees that during Consultant’s consulting relationship Consultant shall not
make, use or permit to be used any Company Property otherwise than for the benefit of the Company. The term “Company
Property” shall include all Confidential Information; the Company’s records, files and data; all Company computers, cellular
telephones, personal digital assistants, credit and/or calling cards, keys, access cards and the like; and all other documentation or
materials of any nature and in any form, whether written, printed, electronic or in digital format or otherwise, relating to any
matter within the scope of the business of the Company or concerning any of its dealings or affairs and any other Company
property in Consultant’s possession, custody or control. Consultant further agrees that Consultant shall not, after the termination
of Consultant’s consulting relationship, regardless of the reason for such termination, use or permit others to use any such
Company Property. Consultant acknowledges and agrees that all Company Property shall be and remain the sole and exclusive
property of the Company. Immediately upon the termination of Consultant’s consulting relationship Consultant shall deliver all
Company Property in Consultant’s possession, and all copies thereof, to the Company.
6.
Term of Consulting Relationship. The term of this Agreement shall be from January 15, 2020 through July
15, 2020 (the “Term”). Notwithstanding the foregoing, either the Company or Consultant may terminate Consultant’s consulting
relationship and this Agreement at any time, for any or no reason, on at least ten (10) days’ prior notice.
7.
Independent Contractor.
Consultant is an independent contractor and not an employee of the
Company. Consultant shall be responsible for all taxes arising from compensation and other amounts paid under this
Agreement. Neither federal, state or local income tax, nor payroll tax of any kind, shall be withheld or paid by the Company on
Consultant’s behalf. Consultant will not be eligible for, and shall not participate in, any employee pension, health, welfare, or
other fringe benefit plan of the Company.
8.
Waiver; Amendments. Any waiver by the Company of a breach of any provision of this Agreement shall not
operate or be construed as a waiver of any subsequent breach of such provision or any other provision hereof. In addition, any
amendment to or modification of this Agreement or any waiver of any provision hereof must be agreed to in writing by both
parties.
- 4 -
9.
Severability. Consultant agrees that each provision and the subparts of each provision herein shall be treated
as separate and independent clauses, and the unenforceability of any one clause shall in no way impair the enforceability of any
of the other clauses of the Agreement. Moreover, if one or more of the provisions contained in this Agreement shall for any
reason be held to be excessively broad as to scope, activity, subject or otherwise so as to be unenforceable at law, such provision
or provisions shall be construed by the appropriate judicial body by limiting or reducing it or them, so as to be enforceable to the
maximum extent compatible with the applicable law as it shall then appear. Consultant hereby further agrees that the language of
all parts of this Agreement shall in all cases be construed as a whole according to its fair meaning and not strictly for or against
either of the parties.
10.
Survival. Consultant’s obligations under Section 3 through 10 of this Agreement shall survive the
termination of Consultant’s consulting relationship regardless of the reason for or manner of such termination and shall be
binding upon Consultant’s successors, heirs, executors, administrators and legal representatives.
11.
Assignment. The Company shall have the right to assign this Agreement to its successors and assigns, and
all covenants and agreements hereunder shall inure to the benefit of and be enforceable by said successors or assigns. Consultant
may not assign this Agreement.
12.
Governing Law. This Agreement shall be governed by and construed in accordance with the laws of the
State of Delaware and shall in all respects be interpreted, enforced and governed under the internal and domestic laws of
Delaware, without giving effect to the principles of conflicts of laws of such state. The laws of the State of Delaware shall
govern any claims or legal actions by one party against the other arising out of the relationship between the parties contemplated
herein (whether or not arising under this Agreement).
13.
Equity.
The section of each of Consultant’s Series D Common Unit Restriction Agreements with
the Company dated September 12, 2017 and December 7, 2017 (the “URA”), entitled “Forfeiture of Units”, is hereby amended to
provide that Consultant’s provision of Services pursuant to this Agreement shall be deemed the equivalent of employment by the
Company solely for purposes of the URA. For the avoidance of doubt, as provided by Section 2.58 of the Company’s 2018
Omnibus Incentive Plan, Consultant’s stock options with the Company shall continue to vest so long as he provides Services to
the Company pursuant to this Agreement.
14.
Entire Agreement. This Agreement sets forth the complete, sole and entire agreement between the parties
with respect to the subject matter herein and supersedes any and all other agreements, negotiations, discussions, proposals, or
understandings, whether oral or written, previously entered into, discussed or considered by the parties; provided, however, the
parties acknowledge that the terms and conditions of the Employment Agreement, dated January 25, 2019, by and between the
Company and Consultant, and the Restrictive Covenant Agreement (as defined in such Employment Agreement) survive, and
shall not be impacted by the terms and conditions of this Agreement.
IN WITNESS HEREOF, Consultant has executed this Agreement as of the date first written below.
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By:
/s/ Alvaro Amorrortu
Alvaro Amorrortu
Date:
January 10, 2020
Accepted and agreed to as of
the date set forth above:
Solid Biosciences Inc.
By:
/s/ Ilan Ganot
Ilan Ganot, CEO
Certain consulting and transition services as requested by the Company from time to time.
EXHIBIT A
Description of Services
ActiveUS 177635953v.2
CONSULTING AGREEMENT
Exhibit 10.26
In consideration and as a condition of Jorge Quiroz, an individual having an address at [*] (the “Consultant”),
providing consulting services to Solid Biosciences Inc., a Delaware corporation (together with its affiliates, the “Company”),
having an address at 141 Portland Street, Fifth Floor, Cambridge, MA 02139, the Consultant hereby agrees with the Company as
follows:
1.
Relationship. During the Term (as defined below), Consultant shall devote reasonable time and efforts to the
Company’s business, consistent with Consultant’s other business responsibilities. Consultant’s efforts on behalf of the Company
shall, accordingly, be on a part‑time basis. Consultant’s duties are described in Exhibit A hereto and shall be performed on an as-
needed basis, as requested by the Company (the “Services”). Consultant represents that his service as a consultant to the
Company and Consultant’s performance of the Services and all of the terms of this Agreement do not and will not violate any
other legal obligation or any agreement to which Consultant is or shall become a party. In performing the Services, Consultant
shall comply, to the best of his knowledge, with all business conduct, regulatory, ethical and health and safety guidelines
established by any governmental authority with respect to the Company’s business.
2.
Compensation. Consultant shall invoice the Company monthly in arrears on the basis of time dedicated to the
Services, and such invoice shall include a description of activities performed, including the date or dates of the Services. The
Company shall pay Consultant the consulting fees within thirty (30) days after receipt of the applicable invoice. The consulting
fee will be $500 per hour. Company shall reimburse Consultant for all reasonable travel and out-of-pocket expenses incurred by
Consultant in performing Services pursuant to this Agreement that are preapproved by the Company in writing, upon submission
of appropriate supporting documentation. Air travel of over five hours will be reimbursed at business class rates, and all other air
travel at economy rates. The Consultant must obtain prior written authorization from the Company before booking such travel.
3.
Work-Made-for Hire.
(a)
All right, title and interest in any and all writings, ideas, inventions, know-how, designs, improvements or
other property created during Consultant’s consulting relationship relating in any way to the assets, business or operations of the
Company, constituting copyrights, patents, trademarks, service marks and related rights or other forms of proprietary rights or
information (regardless of whether any such copyrights, patents, trademarks and service marks or other rights have or may be
registered) that are created, adapted or improved by Consultant (whether alone or in conjunction with any other person or
employee), and all material created during Consultant’s consulting relationship that includes any of the foregoing (collectively,
“Covered Material”), shall be owned by the Company and to the extent that it includes copyrightable subject matter, shall be
deemed a work made for hire for the Company within the meaning of the United States Copyright Act of 1976 and for all other
purposes. If any Covered Material is deemed not to be work made for hire, such Covered Material is hereby assigned by
Consultant to the Company and Consultant shall not have or claim to have, under this Agreement or otherwise, any right, title or
interest of any kind or nature whatsoever in such Covered Material.
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(b)
The Company shall have the right to apply for and obtain registrations in the United States Copyright Office
and the United States Patent and Trademark Office, in its own or its designee’s name, of its rights in any or all of the Covered
Material. If for any reason the rights in any Covered Material are registered, or applied to be registered, in Consultant’s name,
Consultant shall assign in writing such application or registration to the Company and hereby authorizes and appoints the
Company its agent for the purpose of recording such assignment.
(c)
Whenever the Company shall so request, whether during or after Consultant’s consulting relationship,
Consultant shall execute, acknowledge and deliver all applications, assignments or other instruments; make or cause to be made
all rightful oaths; testify in all legal proceedings; communicate all known facts which relate to such works, copyrights,
inventions, ideas, discoveries, designs and improvements; perform all lawful acts and otherwise render all such assistance as the
Company may deem necessary to protect the Company’s interest therein including any assistance which the Company shall deem
necessary in connection with any proceeding or litigation involving the same. The Company shall reimburse Consultant for all
reasonable out-of-pocket costs (such as travel, meals, and lodging), incurred by Consultant in rendering any such assistance
requested by the Company pursuant to this Section.
4.
Nondisclosure. Consultant shall not at any time, whether during or after the termination of Consultant’s
consulting relationship, regardless of the reason for such termination, directly or indirectly reveal to any person or entity any
Confidential Information except to employees of the Company who need to know such Confidential Information for the purposes
of their employment, or as otherwise authorized by the Company in writing. For purposes of this Agreement, the term
“Confidential Information” means any and all information and derivative information, in whatever form or medium, including
oral information, concerning or relating to the Company or information of any third party that the Company is under an
obligation to keep confidential or that is maintained by the Company as confidential or proprietary, including, without limitation,
intellectual property of the Company, such as, but not limited to, patent applications, copyrights, copyright applications, and trade
secrets; information regarding or resulting from research and development activities performed by or on behalf of the Company
and other projects (such as, but not limited to, preclinical and clinical data, design details and specifications, engineering
information, and works in process); and business and financial information (such as, but not limited to, current, future, and
proposed products and services, financial information and models, information relating to procurement requirements, purchasing,
manufacturing, investors, customer lists, customers, suppliers, facilities, product plans, product ideas, business strategies,
marketing or business plans, financial or personnel matters, investors, employees, business and contractual relationships, business
forecasts, sales, strategies, operations, policies, procedures, commercialization capabilities, and information regarding third
parties). Notwithstanding the foregoing, Confidential Information does not include information that Consultant can demonstrate:
(a) is publicly known and generally available in the public domain other than in consequence of improper action by any person;
or (b) was acquired by Consultant free and clear of any duty of confidentiality or restricted use and without improper action by
the transferor of such information or any other person. Consultant shall keep confidential all matters entrusted to Consultant by
or on behalf of the Company and shall not, directly or indirectly, for himself or for any other person or entity, use or attempt to
use any Confidential Information except as may be required in the ordinary course of performing Consultant’s duties as a
consultant to the Company, and Consultant shall not, directly or indirectly, use any Confidential Information in any manner that
may injure or
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cause loss or may be calculated to injure or cause loss to the Company, whether directly or indirectly. Nothing in this Agreement
prohibits Consultant from reporting possible violations of federal or state law or regulations to any governmental agency or entity
or self-regulatory institution, including but not limited to the Equal Employment Opportunity Commission, the National Labor
Relations Board, the Department of Justice, the Securities and Exchange Commission, Congress, and any Inspector General, or
making other disclosures that are protected under the whistleblower provisions of federal or state law or regulation. Prior
authorization of the Company shall not be required to make any such reports or disclosures and Consultant is not required to
notify the Company that Consultant has made such reports or disclosures.
5.
Company Property. Consultant agrees that during Consultant’s consulting relationship Consultant shall not
make, use or permit to be used any Company Property otherwise than for the benefit of the Company. The term “Company
Property” shall include all Confidential Information; the Company’s records, files and data; all Company computers, cellular
telephones, personal digital assistants, credit and/or calling cards, keys, access cards and the like; and all other documentation or
materials of any nature and in any form, whether written, printed, electronic or in digital format or otherwise, relating to any
matter within the scope of the business of the Company or concerning any of its dealings or affairs and any other Company
property in Consultant’s possession, custody or control. Consultant further agrees that Consultant shall not, after the termination
of Consultant’s consulting relationship, regardless of the reason for such termination, use or permit others to use any such
Company Property. Consultant acknowledges and agrees that all Company Property shall be and remain the sole and exclusive
property of the Company. Immediately upon the termination of Consultant’s consulting relationship Consultant shall deliver all
Company Property in Consultant’s possession, and all copies thereof, to the Company.
6.
Term of Consulting Relationship. The term of this Agreement shall be from January 15, 2020 through July
15, 2020 (the “Term”). Notwithstanding the foregoing, either the Company or Consultant may terminate Consultant’s consulting
relationship and this Agreement at any time, for any or no reason, on at least ten (10) days’ prior notice.
7.
Independent Contractor.
Consultant is an independent contractor and not an employee of the
Company. Consultant shall be responsible for all taxes arising from compensation and other amounts paid under this
Agreement. Neither federal, state or local income tax, nor payroll tax of any kind, shall be withheld or paid by the Company on
Consultant’s behalf. Consultant will not be eligible for, and shall not participate in, any employee pension, health, welfare, or
other fringe benefit plan of the Company.
8.
Waiver; Amendments. Any waiver by the Company of a breach of any provision of this Agreement shall not
operate or be construed as a waiver of any subsequent breach of such provision or any other provision hereof. In addition, any
amendment to or modification of this Agreement or any waiver of any provision hereof must be agreed to in writing by both
parties.
9.
Severability. Consultant agrees that each provision and the subparts of each provision herein shall be treated
as separate and independent clauses, and the unenforceability of any one clause shall in no way impair the enforceability of any
of the other clauses of the Agreement. Moreover, if one or more of the provisions contained in this Agreement shall for any
- 4 -
reason be held to be excessively broad as to scope, activity, subject or otherwise so as to be unenforceable at law, such provision
or provisions shall be construed by the appropriate judicial body by limiting or reducing it or them, so as to be enforceable to the
maximum extent compatible with the applicable law as it shall then appear. Consultant hereby further agrees that the language of
all parts of this Agreement shall in all cases be construed as a whole according to its fair meaning and not strictly for or against
either of the parties.
10.
Survival. Consultant’s obligations under Section 3 through 10 of this Agreement shall survive the
termination of Consultant’s consulting relationship regardless of the reason for or manner of such termination and shall be
binding upon Consultant’s successors, heirs, executors, administrators and legal representatives.
11.
Assignment. The Company shall have the right to assign this Agreement to its successors and assigns, and
all covenants and agreements hereunder shall inure to the benefit of and be enforceable by said successors or assigns. Consultant
may not assign this Agreement.
12.
Governing Law. This Agreement shall be governed by and construed in accordance with the laws of the
State of Delaware and shall in all respects be interpreted, enforced and governed under the internal and domestic laws of
Delaware, without giving effect to the principles of conflicts of laws of such state. The laws of the State of Delaware shall
govern any claims or legal actions by one party against the other arising out of the relationship between the parties contemplated
herein (whether or not arising under this Agreement).
13.
Equity.
The section of each of Consultant’s Series D Common Unit Restriction Agreements with
the Company dated September 12, 2017 and December 7, 2017 (the “URA”), entitled “Forfeiture of Units”, is hereby amended to
provide that Consultant’s provision of Services pursuant to this Agreement shall be deemed the equivalent of employment by the
Company solely for purposes of the URA. For the avoidance of doubt, as provided by Section 2.58 of the Company’s 2018
Omnibus Incentive Plan, Consultant’s stock options with the Company shall continue to vest so long as he provides Services to
the Company pursuant to this Agreement.
14.
Entire Agreement. This Agreement sets forth the complete, sole and entire agreement between the parties
with respect to the subject matter herein and supersedes any and all other agreements, negotiations, discussions, proposals, or
understandings, whether oral or written, previously entered into, discussed or considered by the parties; provided, however, the
parties acknowledge that the terms and conditions of the Employment Agreement, dated January 25, 2019, by and between the
Company and Consultant, and the Restrictive Covenant Agreement (as defined in such Employment Agreement) survive, and
shall not be impacted by the terms and conditions of this Agreement.
IN WITNESS HEREOF, Consultant has executed this Agreement as of the date first written below.
- 5 -
By:
/s/ Jorge Quiroz
Jorge Quiroz
Date: January 10, 2020
Accepted and agreed to as of
the date set forth above:
Solid Biosciences Inc.
By:
/s/ Ilan Ganot
Ilan Ganot, CEO
EXHIBIT A
Description of Services
Certain consulting and transition services as requested by the Company from time to time.
THIS EMPLOYMENT AGREEMENT (the "Agreement"), is made as of January 25, 2019 by and between Solid Biosciences Inc. (the
"Company"), and Jorge A. Quiroz, M.D. (the "Executive") (together, the "Parties").
EMPLOYMENT AGREEMENT
Exhibit 10.27
WHEREAS, the Company desires to continue to employ the Executive as its Chief Medical Officer; and
RECITALS
WHEREAS, the Company and the Executive are party to a letter agreement dated November 17, 2015 (the "Existing Agreement") and desire
to amend and restate the Existing Agreement with the exception of Exhibit A, as amended, attached to the Existing Agreement (the "Restrictive Covenant
Agreement"), which shall not be so amended or restated and shall remain in full force and effect; and
WHEREAS, the Executive has agreed to continue employment with the Company on the terms and conditions set forth in this Agreement;
NOW, THEREFORE, in consideration of the foregoing and of the respective covenants and agreements of the Parties herein contained, the
Parties hereto agree as follows:
Agreement. This Agreement shall be effective as of the date first set forth above (the "Effective Date"). Following the Effective Date, the
1.
Executive shall continue to be an employee of the Company until such employment relationship is terminated in accordance with Section 6 hereof (the
"Term of Employment").
2.
Company's office in Cambridge, Massachusetts, and travelling as reasonably required by the Executive's job duties.
Position. During the Term of Employment, the Executive shall serve as the Chief Medical Officer of the Company, working out of the
3.
Scope of Employment. During the Term of Employment, the Executive shall be responsible for the performance of those duties consistent with
the Executive's position as Chief Medical Officer. The Executive shall report to the Chief Executive Officer of the Company and/or the Company's board of
directors (the "Board") and shall perform and discharge faithfully, diligently, and to the best of the Executive's ability, the Executive's duties and
responsibilities hereunder. The Executive shall devote substantially all of the Executive's business time, loyalty, attention and efforts to the business
and affairs of the Company and its affiliates. Membership on boards of directors of up to two other companies or community, charitable or industry
organizations will be permitted only with the express approval of the Chief Executive Officer, provided that such activities do not create a conflict of
interest or otherwise interfere with the Executive's performance of the Executive's duties hereunder. Membership on boards of directors of any other
companies or community, charitable or industry organizations will be permitted only with the express approval of the Board. The Executive agrees to abide
by the rules, regulations, instructions, personnel practices and policies of the Company and any changes therein that may be adopted :from time to time by
the Company.
ActiveUS 171347422
4.
Employment, the Company will provide to the Executive the following:
Compensation. As full compensation for all services rendered by the Executive to the Company and any affiliate thereof, during the Term of
(a)
Base Salary. Effective as of January 1, 2019, the Executive shall receive a base salary at the annualized rate of $412,500.00
(the "Base Salary"). The Executive's Base Salary shall be paid in equal installments in accordance with the Company's regularly established payroll
procedures. The Executive's Base Salary will be reviewed from time to time by the Board and is subject to change in the discretion of the Board.
(b)
Annual Discretionary Bonus. Effective as of January I, 2019, the Executive will be eligible to earn an annual performance
bonus of up to 40% of the Executive's Base Salary (the "Target Bonus"), based upon the Board's assessment of the Executive's performance and the
Company's attainment of targeted goals as set by the Board in its sole discretion. The Board may determine to provide the bonus in the form of cash, equity
award(s), or a combination of cash and equity. Following the close of each calendar year, the Board will determine whether the Executive has earned a
performance bonus, and the amount of any performance bonus, based on the set criteria. No amount of the annual bonus is guaranteed, and the Executive
must be an employee in good standing on the date of payment in order to be eligible for any annual bonus, except as specifically set forth below. The
Executive's bonus eligibility will be reviewed from time to time by the Board and is subject to change in the discretion of the Board.
as the Board shall, in its sole discretion, determine.
(c)
Equity Award. The Executive will be eligible to receive equity awards, if any, at such times and on such terms and conditions
(d)
Paid Time Off The Executive shall receive paid vacation time plus sick time, consistent with the Company's policies as in effect
from time to time. In addition, the Executive shall receive paid time off for Company holidays which are set annually and in accordance with Company
policy.
(e)
Benefits. Subject to eligibility requirements and the Company's polices, the Executive shall have the right, on the same basis
as other employees of the Company, to participate in, and to receive benefits under, any medical, vision and dental insurance policy maintained by the
Company and the Company shall pay a portion of the cost of the premiums for such medical, vision and dental insurance that is consistent with the
Company's then current employee benefit policy if the Executive elects to participate in such plans.
(t) Withholdings. All compensation payable to the Executive shall be subject to applicable taxes and withholdings.
5.
the provisions of Section 3 of Exhibit A attached hereto.
Expenses. The Executive will be reimbursed for his actual, necessary and reasonable business expense pursuant to Company policy, subject to
6.
Employment Termination. This Agreement and the employment of the Executive shall terminate upon the occurrence of any of the following:
(a)
Upon the death of the Executive or at the election of the Company due to the Executive's "Disability". As used in this
Agreement, the term "Disability" shall mean a physical or mental illness or disability that prevents the Executive from performing the duties of the
Executive's position for a period of more than any three consecutive months or for periods aggregating more than twenty-six weeks during any twelve-month
period. The Company shall determine in good faith and in its sole discretion whether the Executive is unable to perform the services provided for herein.
ActiveUS 171347422
to the Executive. As used in this Agreement, "Cause" shall mean a finding by the Company's Chief Executive Officer or the Board that the Executive:
(b)
At the election of the Company, with or without "Cause" (as defined below), immediately upon written notice by the Company
(i)
(ii)
(iii)
(iv)
(v)
(vi)
(vii)
performed his duties, in the good faith opinion of the Company's Chief Executive Officer or the Board, in a grossly
negligent or reckless manner or with willful malfeasance;
exhibited habitual drunkenness or engaged in substance abuse;
committed any material violation of any state or federal law relating to the workplace environment (including, without
limitation, laws relating to sexual harassment or age, sex or other prohibited discrimination) or any material violation of
any material Company policy;
willfully failed or refused to perform in the usual manner at the usual time those duties which he regularly and routinely
performed in connection with the business of the Company or such other duties reasonably related to the capacity in
which the Executive is employed hereunder which may be assigned to the Executive by the Company's Chief Executive
Officer or the Board, which failure or refusal continues for a period of more than seven (7) days after written notice
thereof has been provided to the Executive by the Company's Chief Executive Officer or the Board, such notice to set
forth in reasonable detail the nature of such failure or refusal;
performed any material action when specifically and reasonably instructed not to do so by the Company's Chief
Executive Officer or the Board;
committed any fraud or used or appropriated for his personal use or benefit any funds, properties or opportunities of the
Company not authorized by the Company's Chief Executive Officer or the Board to be so used or appropriated; or
was convicted of, or pled guilty or "no contest" to, any felony or any other crime related to the Executive's employment or
involving moral turpitude.
(c)
At the election of the Executive, with or without "Good Reason" (as defined below), immediately upon written notice by the
Executive to the Company (subject, if it is with Good Reason, to the timing provisions set forth in the definition of Good Reason). As used in this
Agreement, "Good Reason" shall mean (without the Executive's consent):
(i)
(ii)
(iii)
(iv)
a material diminution in the nature or scope of Executive's duties, responsibilities, or authority;
a material diminution of the Executive's base compensation;
the Company's requiring Executive to relocate Executive's primary office more than fifty (50) miles from the Executive's
then-current primary office; or
any material breach of this Agreement by the Company not otherwise covered by this paragraph;
provided, however, that in each case, the Company shall have a period of not less than thirty (30) days to cure any act constituting Good Reason following
Executive's delivery to the Company of written notice within sixty (60) days of the action or omission constituting Good Reason and that the Executive
actually terminates employment within thirty (30) days following the expiration of the Company's cure period.
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7.
Effect of Termination.
(a)
All Terminations Other Than by the Company Without Cause or by the Executive With Good Reason. If the Executive's
employment is terminated under any circumstances other than a Qualifying Termination (as defined below) (including a voluntary termination by the
Executive without Good Reason pursuant to Section 6(c), a termination by the Company for Cause pursuant to Section 6(b) or due to the Executive's death or
Disability pursuant to Section 6(a)), the Company's obligations under this Agreement shall immediately cease and the Executive shall only be entitled to
receive (i) the Base Salary that has accrued and to which the Executive is entitled as of the effective date of such termination and to the extent consistent with
general Company policy, accrued but unused paid time off through and including the effective date of such termination, to be paid in accordance with the
Company's established payroll procedure and applicable law but no later than the next regularly scheduled pay period, (ii) unreimbursed business expenses
for which expenses the Executive has timely submitted appropriate documentation in accordance with Section 5 hereof, and (iii) any amounts or benefits to
which the Executive is then entitled under the terms of the benefit plans then-sponsored by the Company in accordance with their terms (and not accelerated
to the extent acceleration does not satisfy Section 409A of the Internal Revenue Code of 1986, as amended, (the "Code") (the payments described in this
sentence, the "Accrued Obligations").
(b)
Termination by the Company Without Cause or by the Executive With Good Reason Prior to or More Than Twelve Months
Following a Change in Control. If the Executive's employment is terminated by the Company without Cause pursuant to Section 6(b) or by the Executive
with Good Reason pursuant to Section 6(c) (in either case, a "Qualifying Termination") prior to or more than twelve (12) months following a Change in
Control (as defined below), the Executive shall be entitled to the Accrued Obligations. In addition, and subject to Exhibit A and the conditions of Section
7(d), the Company shall: (i) continue to pay to the Executive, in accordance with the Company's regularly established payroll procedures, the Executive's
Base Salary for a period of twelve (12) months and (ii) provided the Executive is eligible for and timely elects to continue receiving group medical insurance
pursuant to the "COBRA" law, continue to pay (but in no event longer than twelve (12) months following the Executive's termination date) the share of the
premium for health coverage that is paid by the Company for active and similarly-situated employees who receive the same type of coverage, unless the
Company's provision of such COBRA payments will violate the nondiscrimination requirements of applicable law, in which case this benefit will not apply
(collectively, the "Severance Benefits").
(c)
Termination by the Company Without Cause or by the Executive With Good Reason Within Twelve Months Following a Change in
Control. If a Qualifying Termination occurs within twelve (12) months following a Change in Control, then the Executive shall be entitled to the
Accrued Obligations. In addition, and subject to Exhibit A and the conditions of Section 7(d), the Company shall: (i) continue to pay to the Executive, in
accordance with the Company's regularly established payroll procedures, the Executive's Base Salary for a period of twelve (12) months; (ii) pay to the
Executive, in a single lump sum on the Payment Date (as defined below) an amount equal to 100% of the Executive's Target Bonus for the year in which
termination occurs or the Executive's Target Bonus immediately prior to the Change in Control, if higher, (iii) provided the Executive is eligible for and
timely elects to continue receiving group medical insurance pursuant to the "COBRA" law, continue to pay (but in no event longer than twelve (12) months
following the Executive's termination date) the share of the premium for health coverage that is paid by the Company for active and similarly-situated
employees who receive the same type of coverage, unless the Company's provision of such COBRA payments will violate the nondiscrimination
requirements of applicable law, in which case this benefit will not apply, and (iv) provide that the vesting of the Executive's then-unvested equity awards that
vest based solely on the passage of time shall be accelerated, such that all then unvested time-based equity awards shall vest and become fully exercisable or
non-forfeitable as of the termination date (collectively, the "Change in Control Severance Benefits").
ActiveUS 171347422
(d)
Severance and Release of Claims Agreement. As a condition of the Executive's receipt of the Severance Benefits or the Change
in Control Severance Benefits, as applicable, the Executive must execute and deliver to the Company a severance and release of claims agreement in a form
to be provided by the Company (the "Severance Agreement"), which Severance Agreement must become irrevocable within 60 days following the date of the
Executive's termination of employment (or such shorter period as may be directed by the Company). The Severance Benefits or the Change in Control
Severance Benefits, as applicable, will be paid or commence to be paid in the first regular payroll beginning after the Severance Agreement becomes
effective, provided that if the foregoing 60 day period (or shorter period as may be directed by the Company) would end in a calendar year subsequent to the
year in which the Executive's employment ends, the Severance Benefits or Change in Control Severance Benefits, as applicable, will not be paid or begin to
be paid before the first payroll of the subsequent calendar year (the date the Severance Benefits or Change in Control Severance Benefits, as applicable,
commence pursuant to this sentence, the "Payment Date").
(e)
Change in Control Definition. For purposes of this Agreement, "Change in Control" shall mean the occurrence of any of the
following events, provided that such event or occurrence constitutes a change in the ownership or effective control of the Company, or a change in the
ownership of a substantial portion of the assets of the Company, as defined in Treasury Regulation §§ 1.409A-3(i)(5)(v), (vi) and (vii): (i) the acquisition by
an individual, entity or group (within the meaning of Section 13(d)(3) or 14(d)(2) of the Securities Exchange Act of 1934 (the "Exchange Act")) (a "Person")
of beneficial ownership of any capital stock of the Company if, after such acquisition, such Person beneficially owns (within the meaning of Rule 13d-3
under the Exchange Act) fifty percent (50%) or more of either (x) the then-outstanding shares of common stock of the Company (the "Outstanding
Company Common Stock") or (y) the combined voting power of the then-outstanding securities of the Company entitled to vote generally in the election of
directors (the "Outstanding Company Voting Securities"); provided, however, that for purposes of this subsection (i), the following acquisitions shall not
constitute a Change in Control: (1) any acquisition directly from the Company or (2) any acquisition by any entity pursuant to a Business Combination (as
defined below) which complies with clauses (x) and (y) of subsection (iii) of this definition; or (ii) a change in the composition of the Board that results in the
Continuing Directors (as defined below) no longer constituting a majority of the Board (or, if applicable, the Board of Directors of a successor corporation to
the Company), where the term "Continuing Director" means at any date a member of the Board (x) who was a member of the Board on the Effective Date or
(y) who was nominated or elected subsequent to such date by at least a majority of the directors who were Continuing Directors at the time of such
nomination or election or whose election to the Board was recommended or endorsed by at least a majority of the directors who were Continuing Directors at
the time of such nomination or election; provided, however, that there shall be excluded from this clause (y) any individual whose initial assumption of office
occurred as a result of an actual or threatened election contest with respect to the election or removal of directors or other actual or threatened solicitation of
proxies or consents, by or on behalf of a person other than the Board; or (iii) the consummation of a merger, consolidation, reorganization, recapitalization or
share exchange involving the Company, or a sale or other disposition of all or substantially all of the assets of the Company (a "Business Combination"),
unless, immediately following such Business Combination, each of the following two (2) conditions is satisfied: (x) all or substantially all of the individuals
and entities who were the beneficial owners of the Outstanding Company Common Stock and Outstanding Company Voting Securities immediately prior to
such Business Combination beneficially own, directly or indirectly, more than fifty percent (50%) of the then-outstanding shares of common stock and the
combined voting power of the then-outstanding securities entitled to vote generally in the election of directors, respectively, of the resulting or acquiring
corporation in such Business Combination (which shall include, without limitation, a corporation which as a result of such transaction owns the Company or
substantially all of the Company's assets either directly or through one (1) or more subsidiaries) (such resulting or acquiring corporation is referred to herein
as the "Acquiring Corporation") in substantially the same proportions as their ownership of the Outstanding Company Common Stock and Outstanding
Company Voting Securities, respectively, immediately prior to such Business Combination and (y) no Person (excluding any employee benefit plan (or
related trust) maintained or sponsored by the Company or by the Acquiring Corporation) beneficially owns, directly or indirectly, fifty percent (50%) or more
of the then-outstanding shares of common stock of the Acquiring Corporation, or of the combined voting power of the then-outstanding securities of such
corporation entitled to vote generally in the election of directors (except to the extent that such ownership existed prior to the Business Combination); or (iv)
the liquidation or dissolution of the Company.
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8.
Company undergoes a "Change in Ownership or Control" (as defined below), the following provisions shall apply:
Modified Section 280G Cutback. Notwithstanding any other provision of this Agreement, except as set forth in Section 8(b), in the event that the
(a)
The Company shall not be obligated to provide to the Executive any portion of any "Contingent Compensation Payments" (as
defined below) that the Executive would otherwise be entitled to receive to the extent necessary to eliminate any "excess parachute payments" (as defined in
Section 280G(b)(1) of the Code) for the Executive. For purposes of this Section 8, the Contingent Compensation Payments so eliminated shall be referred to
as the "Eliminated Payments" and the aggregate amount (determined in accordance with Treasury Regulation Section 1.280G-1, Q/A-30 or any successor
provision) of the Contingent Compensation Payments so eliminated shall be referred to as the "Eliminated Amount."
(b)
Notwithstanding the provisions of Section 8(a), no such reduction in Contingent Compensation Payments shall be made if (1)
the Eliminated Amount (computed without regard to this sentence) exceeds (2) 100% of the aggregate present value (determined in accordance with
Treasury Regulation Section l.280G-l, Q/A-31 and Q/A-32 or any successor provisions) of the amount of any additional taxes that would be incurred by the
Executive if the Eliminated Payments (determined without regard to this sentence) were paid to the Executive (including state and federal income taxes on
the Eliminated Payments, the excise tax imposed by Section 4999 of the Code payable with respect to all of the Contingent Compensation Payments in
excess of the Executive's "base amount" (as defined in Section 280G(b)(3) of the Code), and any withholding taxes). The override of such reduction in
Contingent Compensation Payments pursuant to this Section 8(b) shall be referred to as a "Section 8(b) Override." For purpose of this paragraph, if any
federal or state income taxes would be attributable to the receipt of any Eliminated Payment, the amount of such taxes shall be computed by multiplying the
amount of the Eliminated Payment by the maximum combined federal and state income tax rate provided by law.
(c)
For purposes of this Section 8 the following terms shall have the following respective meanings:
(i)
"Change in Ownership or Control" shall mean a change in the ownership or effective control of the Company or in the
ownership of a substantial portion of the assets of the Company determined in accordance with Section 280G(b)(2) of the Code.
(ii)
"Contingent Compensation Payment" shall mean any payment (or benefit) in the nature of compensation that is made or
made available (under this Agreement or otherwise) to or for the benefit of a "disqualified individual" (as defined in Section 280G(c) of
the Code) and that is contingent (within the meaning of Section 280G(b)(2)(A)(i) of the Code) on a Change in Ownership or Control of the
Company.
(d)
Any payments or other benefits otherwise due to the Executive following a Change in Ownership or Control that could
reasonably be characterized (as determined by the Company) as Contingent Compensation Payments (the "Potential Payments") shall not be made until the
dates provided for in this Section 8(d). Within thirty (30) days after each date on which the Executive first becomes entitled to receive (whether or not then
due) a Contingent Compensation Payment relating to such Change in Ownership or Control, the Company shall determine and notify the Executive (with
reasonable detail regarding the basis for its determinations) (I) which Potential Payments constitute Contingent Compensation Payments, (2) the Eliminated
Amount and (3) whether the Section 8(b) Override is applicable. Within thirty (30) days after delivery of such notice to the Executive, the Executive shall
deliver a response to the Company (the "Executive Response") stating either (A) that the Executive agrees with the Company's determination pursuant to the
preceding sentence or (B) that the Executive disagrees with such determination, in which case the Executive shall set forth (x) which Potential Payments
should be characterized as Contingent Compensation Payments, (y) the Eliminated Amount, and (z) whether the Section 8(b) Override is applicable. In the
event that the Executive fails to deliver an Executive Response on or before the required date, the Company's initial determination shall be final. If the
Executive states in the Executive Response
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that the Executive agrees with the Company's determination, the Company shall make the Potential Payments to the Executive within three (3) business days
following delivery to the Company of the Executive Response (except for any Potential Payments which are not due to be made until after such date, which
Potential Payments shall be made on the date on which they are due). If the Executive states in the Executive Response that the Executive disagree with the
Company's determination, then, for a period of sixty (60) days following delivery of the Executive Response, the Executive and the Company shall use good
faith efforts to resolve such dispute. If such dispute is not resolved within such 60-day period, such dispute shall be settled exclusively by arbitration in
Cambridge, Massachusetts, in accordance with the rules of the American Arbitration Association then in effect. Judgment may be entered on the arbitrator's
award in any court having jurisdiction. The Company shall, within three (3) business days following delivery to the Company of the Executive Response,
make to the Executive those Potential Payments as to which there is no dispute between the Company and the Executive regarding whether they should be
made (except for any such Potential Payments which are not due to be made until after such date, which Potential Payments shall be made on the date on
which they are due). The balance of the Potential Payments shall be made within three (3) business days following the resolution of such dispute.
(e)
The Contingent Compensation Payments to be treated as Eliminated Payments shall be determined by the Company by
determining the "Contingent Compensation Payment Ratio" (as defined below) for each Contingent Compensation Payment and then reducing the
Contingent Compensation Payments in order beginning with the Contingent Compensation Payment with the highest Contingent Compensation Payment
Ratio. For Contingent Compensation Payments with the same Contingent Compensation Payment Ratio, such Contingent Compensation Payment shall be
reduced based on the time of payment of such Contingent Compensation Payments with amounts having later payment dates being reduced first. For
Contingent Compensation Payments with the same Contingent Compensation Payment Ratio and the same time of payment, such Contingent Compensation
Payments shall be reduced on a pro rata basis (but not below zero) prior to reducing Contingent Compensation Payment with a lower Contingent
Compensation Payment Ratio. The term "Contingent Compensation Payment Ratio" shall mean a fraction the numerator of which is the value of the
applicable Contingent Compensation Payment that must be taken into account by the Executive for purposes of Section 4999(a) of the Code, and the
denominator of which is the actual amount to be received by the Executive in respect of the applicable Contingent Compensation Payment. For example, in
the case of an equity grant that is treated as contingent on the Change in Ownership or Control because the time at which the payment is made or the
payment vests is accelerated, the denominator shall be determined by reference to the fair market value of the equity at the acceleration date, and not in
accordance with the methodology for determining the value of accelerated payments set forth in Treasury Regulation Section 1.280G-1 Q/A-24(b) or (c)).
Agreement or any other agreement or plan under which the Executive receives Contingent Compensation Payments.
(f)
The provisions of this Section 8 are intended to apply to any and all payments or benefits available to the Executive under this
Absence of Restrictions. The Executive represents and warrants that the Executive is not bound by any employment contracts, restrictive
9.
covenants or other restrictions that prevent the Executive from entering into employment with, or carrying out the Executive's responsibilities for, the
Company, or which are in any way inconsistent with any of the terms of this Agreement.
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10.
Notice. Any notice delivered under this Agreement shall be deemed duly delivered three (3) business days after it is sent by registered or
certified mail, return receipt requested, postage prepaid, one (I) business day after it is sent for next-business day delivery via a reputable nationwide
overnight courier service, or immediately upon hand delivery, in each case to the address of the recipient set forth below.
To Executive:
At the address set forth in the Executive's personnel file To
Company:
Solid Biosciences Inc.
141 Portland Street Fifth
Floor
Cambridge, MA 02139
Either Party may change the address to which notices are to be delivered by giving notice of such change to the other Party in the manner set forth in this
Section 10.
Applicable Law; Jury Trial Waiver. This Agreement shall be governed by and construed in accordance with the laws of the State of Delaware
11.
(without reference to the conflict of laws provisions thereof). Any action, suit or other legal proceeding arising under or relating to any provision of this
Agreement shall be commenced only in a court of the State of Delaware (or, if appropriate, a federal court located within the State of Delaware), and the
Company and the Executive each consents to the jurisdiction of such a court. The Company and the Executive each hereby irrevocably waives any right to
a trial by jury in any action, suit or other legal proceeding arising under or relating to any provision of this Agreement.
Successors and Assigns. This Agreement shall be binding upon and inure to the benefit of both Parties and their respective successors and
12.
assigns, including any corporation with which or into which the Company may be merged or which may succeed to its assets or business; provided,
however, that the obligations of the Executive are personal and shall not be assigned by the Executive.
At-Will Employment. During the Term of Employment, the Executive will continue to be an at-will employee of the Company, which means
13.
that, notwithstanding any other provision set forth herein, the employment relationship can be terminated by either Party for any reason, at any time, with
or without prior notice and with or without Cause.
Acknowledgment. The Executive states and represents that the Executive has had an opportunity to fully discuss and review the terms of this
14.
Agreement with an attorney. The Executive further states and represents that the Executive has carefully read this Agreement, understands the contents
herein, freely and voluntarily assents to all of the terms and conditions hereof, and signs the Executive's name of the Executive's own free act.
No Oral Modification, Waiver, Cancellation or Discharge. This Agreement may be amended or modified only by a written instrument executed
15.
by both the Company and the Executive. No delay or omission by the Company in exercising any right under this Agreement shall operate as a waiver of
that or any other right. A waiver or consent given by the Company on any one occasion shall be effective only in that instance and shall not be construed as
a bar to or waiver of any right on any other occasion.
Captions and Pronouns. The captions of the sections of this Agreement are for convenience of reference only and in no way define, limit or
16.
affect the scope or substance of any section of this Agreement. Whenever the context may require, any pronouns used in this Agreement shall include the
corresponding masculine, feminine or neuter forms, and the singular forms of nouns and pronouns shall include the plural, and vice versa.
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Interpretation. The Parties agree that this Agreement will be construed without regard to any presumption or rule requiring construction or
17.
interpretation against the drafting Party. References in this Agreement to "include" or "including" should be read as though they said "without limitation" or
equivalent forms. References in this Agreement to the "Board" shall include any authorized committee thereof.
Severability. Each provision of this Agreement must be interpreted in such manner as to be effective and valid under applicable law, but if any
18.
provision of this Agreement is held to be prohibited by or invalid under applicable law, such provision will be ineffective only to the extent of such
prohibition or invalidity, without invalidating the remainder of such provision or the remaining provisions of this Agreement. Moreover, if a court of
competent jurisdiction determines any of the provisions contained in this Agreement to be unenforceable because the provision is excessively broad in
scope, whether as to duration, activity, geographic application, subject or otherwise, it will be construed, by limiting or reducing it to the extent legally
permitted, so as to be enforceable to the extent compatible with then applicable law to achieve the intent of the Parties.
Entire Agreement. This Agreement constitutes the entire agreement between the Parties and supersedes all prior agreements and
19.
understandings, whether written or oral, relating to the subject matter of this Agreement, provided that, for the avoidance of doubt, the Restrictive Covenant
Agreement is not amended hereby and remains in full force and effect.
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[Signatures on Page Following]
IN WITNESS WHEREOF, the Parties hereto have executed this Agreement as of the day and year set forth above.
SOLID BIOSCIENCES INC.
By:
Name:
Title:
/s/ Ilan Ganot
Ilan Ganot
CEO
EXECUTIVE
/s/ Jorge Quiroz
Jorge A. Quiroz M.D.
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EXHIBIT A
Payments Subject to Section 409A
1.
Subject to this Exhibit A, any severance payments that may be due under the Agreement shall begin only upon the date of the
Executive's "separation from service" (determined as set forth below) which occurs on or after the termination of the Executive's employment. The
following rules shall apply with respect to distribution of the severance payments, if any, to be provided to the Executive under the Agreement, as
applicable:
(a)
(b)
(c)
It is intended that each installment of the severance payments provided under the Agreement shall be treated as a separate "payment"
for purposes of Section 409A of the Internal Revenue Code ("Section 409A"). Neither the Company nor the Executive shall have the
right to accelerate or defer the delivery of any such payments except to the extent specifically permitted or required by Section
409A.
If, as of the date of the Executive's "separation from service" from the Company, the Executive is not a "specified employee" (within
the meaning of Section 409A), then each installment of the severance payments shall be made on the dates and terms set forth in the
letter agreement.
If, as of the date of the Executive's "separation from service" from the Company, the Executive is a "specified employee" (within the
meaning of Section 409A), then:
(i)
(ii)
Each installment of the severance payments due under the Agreement that, in accordance with the dates and terms set
forth herein, will in all circumstances, regardless of when the Executive's separation from service occurs, be paid within
the short-term deferral period (as defined under Section 409A) shall be treated as a short-term deferral within the
meaning of Treasury Regulation Section 1.409A-l(b)(4) to the maximum extent permissible under Section 409A and
shall be paid on the dates and terms set forth in the Agreement; and
Each installment of the severance payments due under the Agreement that is not described in this Exhibit A, Section l(c)
(i) and that would, absent this subsection, be paid within the six-month period following the Executive's "separation
from service" from the Company shall not be paid until the date that is six months and one day after such separation
from service (or, if earlier, the Executive's death), with any such installments that are required to be delayed being
accumulated during the six-month period and paid in a lump sum on the date that is six months and one day following
the Executive's separation from service and any subsequent installments, if any, being paid in accordance with the dates
and terms set forth herein; provided, however, that the preceding provisions of this sentence shall not apply to any
installment of payments if and to the maximum extent that that such installment is deemed to be paid under a separation
pay plan that does not provide for a deferral of compensation by reason of the application of Treasury Regulation
1.409A-l(b)(9)(iii) (relating to separation pay upon an involuntary separation from service). Any installments that
qualify for the exception under Treasury Regulation Section 1.409A-l(b)(9)(iii) must be paid no later than the last day of
the Executive's second taxable year following the taxable year in which the separation from service occurs.
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2.
The determination of whether and when the Executive's separation from service from the Company has occurred shall be made and
in a manner consistent with, and based on the presumptions set forth in, Treasury Regulation Section 1.409A-l(h). Solely for purposes of Section 2 of this
Exhibit A, "Company" shall include all persons with whom the Company would be considered a single employer under Section 414(b) and 414(c) of the
Code.
3.
All reimbursements and in-kind benefits provided under the Agreement shall be made or provided in accordance with the
requirements of Section 409A to the extent that such reimbursements or in-kind benefits are subject to Section 409A, including, where applicable, the
requirements that (i) any reimbursement is for expenses incurred during the Executive's lifetime (or during a shorter period of time specified in the
Agreement), (ii) the amount of expenses eligible for reimbursement during a calendar year may not affect the expenses eligible for reimbursement in any
other calendar year, (iii) the reimbursement of an eligible expense will be made on or before the last day of the calendar year following the year in which
the expense is incurred and (iv) the right to reimbursement is not subject to set off or liquidation or exchange for any other benefit.
4.
The Company makes no representation or warranty and shall have no liability to the Executive or to any other person if any of the
provisions of the Agreement (including this Exhibit A) are determined to constitute deferred compensation subject to Section 409A but that do not satisfy
an exemption from, or the conditions of, that section.
5.
The Agreement is intended to comply with, or be exempt from, Section 409A and shall be interpreted accordingly .
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Subsidiaries of Solid Biosciences Inc.
Name of Subsidiary
Solid Biosciences UK Limited
Solid Biosciences Securities Corporation
Exhibit 21.1
Jurisdiction of Organization
United Kingdom
Massachusetts
CONSENT OF INDEPENDENT REGISTERED PUBLIC ACCOUNTING FIRM
We hereby consent to the incorporation by reference in the Registration Statements on Form S-8 (No. 333-222763) and Form S-3 (No. 333-233594 and No.
333-230228) of Solid Biosciences Inc. of our report dated March 10, 2020 relating to the financial statements, which appears in this Form 10-K.
Exhibit 23.1
/s/ PricewaterhouseCoopers LLP
Boston, Massachusetts
March 12, 2020
CERTIFICATION OF PERIODIC REPORT UNDER SECTION 302 OF
THE SARBANES-OXLEY ACT OF 2002
Exhibit 31.1
I, Ilan Ganot, certify that:
1. I have reviewed this Annual Report on Form 10-K of Solid Biosciences Inc.;
2. Based on my knowledge, this report does not contain any untrue statement of a material fact or omit to state a material fact necessary to make the
statements made, in light of the circumstances under which such statements were made, not misleading with respect to the period covered by this report;
3. Based on my knowledge, the financial statements, and other financial information included in this report, fairly present in all material respects the
financial condition, results of operations and cash flows of the registrant as of, and for, the periods presented in this report;
4. The registrant’s other certifying officer and I are responsible for establishing and maintaining disclosure controls and procedures (as defined in Exchange
Act Rules 13a-15(e) and 15d-15(e)) and internal control over financial reporting (as defined in Exchange Act Rules 13a-15(f) and 15d-15(f)) for the
registrant and have:
a) Designed such disclosure controls and procedures, or caused such disclosure controls and procedures to be designed under our supervision, to ensure that
material information relating to the registrant, including its consolidated subsidiaries, is made known to us by others within those entities, particularly
during the period in which this report is being prepared;
b) Designed such internal control over financial reporting, or caused such internal control over financial reporting to be designed under our supervision, to
provide reasonable assurance regarding the reliability of financial reporting and the preparation of financial statements for external purposes in accordance
with generally accepted accounting principles;
c) Evaluated the effectiveness of the registrant’s disclosure controls and procedures and presented in this report our conclusions about the effectiveness of
the disclosure controls and procedures, as of the end of the period covered by this report based on such evaluation; and
d) Disclosed in this report any change in the registrant’s internal control over financial reporting that occurred during the registrant’s most recent fiscal
quarter (the registrant’s fourth fiscal quarter in the case of an annual report) that has materially affected, or is reasonably likely to materially affect, the
registrant’s internal control over financial reporting; and
5. The registrant’s other certifying officer and I have disclosed, based on our most recent evaluation of internal control over financial reporting, to the
registrant’s auditors and the audit committee of the registrant’s board of directors (or persons performing the equivalent functions):
a) All significant deficiencies and material weaknesses in the design or operation of internal control over financial reporting which are reasonably likely to
adversely affect the registrant’s ability to record, process, summarize and report financial information; and
b) Any fraud, whether or not material, that involves management or other employees who have a significant role in the registrant’s internal control over
financial reporting.
Date: March 12, 2020
By:
/s/ Ilan Ganot
Ilan Ganot
President and Chief Executive Officer
(Principal Executive Officer)
CERTIFICATION OF PERIODIC REPORT UNDER SECTION 302 OF
THE SARBANES-OXLEY ACT OF 2002
Exhibit 31.2
I, Jennifer Ziolkowski, certify that:
1. I have reviewed this Annual Report on Form 10-K of Solid Biosciences Inc.;
2. Based on my knowledge, this report does not contain any untrue statement of a material fact or omit to state a material fact necessary to make the
statements made, in light of the circumstances under which such statements were made, not misleading with respect to the period covered by this report;
3. Based on my knowledge, the financial statements, and other financial information included in this report, fairly present in all material respects the
financial condition, results of operations and cash flows of the registrant as of, and for, the periods presented in this report;
4. The registrant’s other certifying officer and I are responsible for establishing and maintaining disclosure controls and procedures (as defined in Exchange
Act Rules 13a-15(e) and 15d-15(e)) and internal control over financial reporting (as defined in Exchange Act Rules 13a-15(f) and 15d-15(f)) for the
registrant and have:
a) Designed such disclosure controls and procedures, or caused such disclosure controls and procedures to be designed under our supervision, to ensure that
material information relating to the registrant, including its consolidated subsidiaries, is made known to us by others within those entities, particularly
during the period in which this report is being prepared;
b) Designed such internal control over financial reporting, or caused such internal control over financial reporting to be designed under our supervision, to
provide reasonable assurance regarding the reliability of financial reporting and the preparation of financial statements for external purposes in accordance
with generally accepted accounting principles;
c) Evaluated the effectiveness of the registrant’s disclosure controls and procedures and presented in this report our conclusions about the effectiveness of
the disclosure controls and procedures, as of the end of the period covered by this report based on such evaluation; and
d) Disclosed in this report any change in the registrant’s internal control over financial reporting that occurred during the registrant’s most recent fiscal
quarter (the registrant’s fourth fiscal quarter in the case of an annual report) that has materially affected, or is reasonably likely to materially affect, the
registrant’s internal control over financial reporting; and
5. The registrant’s other certifying officer and I have disclosed, based on our most recent evaluation of internal control over financial reporting, to the
registrant’s auditors and the audit committee of the registrant’s board of directors (or persons performing the equivalent functions):
a) All significant deficiencies and material weaknesses in the design or operation of internal control over financial reporting which are reasonably likely to
adversely affect the registrant’s ability to record, process, summarize and report financial information; and
b) Any fraud, whether or not material, that involves management or other employees who have a significant role in the registrant’s internal control over
financial reporting.
Date: March 12, 2020
By:
/s/ Jennifer Ziolkowski
Jennifer Ziolkowski
Chief Financial Officer
(Principal Financial and Accounting Officer)
CERTIFICATION PURSUANT TO
18 U.S.C. SECTION 1350, AS ADOPTED PURSUANT TO
SECTION 906 OF THE SARBANES-OXLEY ACT OF 2002
Exhibit 32.1
In connection with the Annual Report of Solid Biosciences Inc. (the “Company”) on Form 10-K for the year ended December 31, 2019 as filed with
the Securities and Exchange Commission on the date hereof (the “Report”), I, Ilan Ganot, Chief Executive Officer of the Company, certify, pursuant to 18
U.S.C. § 1350, as adopted pursuant to § 906 of the Sarbanes-Oxley Act of 2002, that, to my knowledge:
(1)
(2)
The Report fully complies with the requirements of Section 13(a) or 15(d) of the Securities Exchange Act of 1934; and
The information contained in the Report fairly presents, in all material respects, the financial condition and result of operations of the
Company.
Date: March 12, 2020
By:
/s/ Ilan Ganot
Ilan Ganot
President and Chief Executive Officer
(Principal Executive Officer)
CERTIFICATION PURSUANT TO
18 U.S.C. SECTION 1350, AS ADOPTED PURSUANT TO
SECTION 906 OF THE SARBANES-OXLEY ACT OF 2002
Exhibit 32.2
In connection with the Annual Report of Solid Biosciences Inc. (the “Company”) on Form 10-K for the year ended December 31, 2019 as filed with
the Securities and Exchange Commission on the date hereof (the “Report”), I, Jennifer Ziolkowski, Chief Financial Officer of the Company, certify,
pursuant to 18 U.S.C. § 1350, as adopted pursuant to § 906 of the Sarbanes-Oxley Act of 2002, that, to my knowledge:
(1)
(2)
The Report fully complies with the requirements of Section 13(a) or 15(d) of the Securities Exchange Act of 1934; and
The information contained in the Report fairly presents, in all material respects, the financial condition and result of operations of the
Company.
Date: March 12, 2020
By:
/s/ Jennifer Ziolkowski
Jennifer Ziolkowski
Chief Financial Officer
(Principal Financial and Accounting Officer)