Applied Genetic Technologies Corporation
2017
ANNUAL REPORT
Dear Shareholder:
This past year at AGTC has been marked by multiple corporate and clinical milestones as we continue our
efforts to develop transformational genetic therapies for patients suffering from rare and debilitating diseases.
2017 was a landmark year in the field of ocular gene therapy, highlighted by the first ever FDA approval for an
inherited retinal disease. We are energized by this progress and are more committed than ever to advancing our
clinical programs to deliver novel gene-based therapies for orphan indications.
Our pipeline includes clinical-stage ophthalmology programs in X-linked retinoschisis (XLRS),
achromatopsia (ACHM), X-linked retinitis pigmentosa (XLRP), as well as a preclinical optogenetics
collaboration. In addition to these programs, we are working in several non-ophthalmology indications including
adrenoleukodystrophy (ALD) and a preclinical otology initiative focused on treating genetic causes of hearing
loss.
Our most advanced product candidate, a potential treatment for XLRS, is being evaluated in an ongoing
Phase 1/2 clinical trial as part of the company’s collaboration with Biogen. To date, the company has completed
enrollment of 12 patients in the dose escalation phase of the trial. We are also enrolling adult patients in the
expansion group at the highest dose level and three children between the ages of 6 and 18 at the mid-dose level.
To date, a total of 21 patients have been enrolled in this trial.
We are also actively pursuing clinical development of novel gene-based treatments for two forms of ACHM
that account for approximately 75 percent of the affected patient population. A Phase 1/2 clinical trial evaluating
the company’s ACHM CNGB3 product candidate is currently enrolling patients. The company completed
enrollment of four patients in one dose group before electing to adjust the dose downward and continue enrolling
patients in the dose ranging study. We are also currently enrolling patients in a Phase 1/2 clinical trial evaluating
the company’s ACHM CNGA3 product candidate.
Additionally, in August of this year, the company filed an IND with the FDA to support the initiation of a
Phase 1/2 clinical trial of the company’s gene therapy product candidate for the treatment of XLRP caused by
mutations in the RPGR gene. For this trial, we are currently screening patients for enrollment.
We have been excited to have four ongoing clinical trials seeking novel gene therapy treatments. Managing
those trials, however, with our lean organization in the novel gene therapy field has also posed challenges, and,
as we have discussed, the time to enroll patients has taken us longer than anticipated. As a result, we have more
than doubled the size of our clinical team by hiring staff at all levels of the organization. In the XLRS and XLRP
programs, we have strengthened our collaboration with the Biogen clinical team. We have also engaged a leading
global contract research organization to provide additional support. All of these activities are being driven by a
single overarching goal for 2018 to meet our clinical timelines.
Although our clinical trials are the priority for 2018, our ongoing collaborations remain an important focal
point for our company. We recently announced a partnership with Foundation Fighting Blindness to support the
organizations’ shared mission to advance gene therapy research to treat inherited retinal diseases. As part of our
collaboration, AGTC will provide grant funding to support the Foundation’s My Retina Tracker® registry as well
as a genetic testing study designed to identify barriers that are preventing greater patient participation in genetic
testing. With AGTC’s support, additional registry participants who have been diagnosed with XLRS, ACHM or
XLRP will have the opportunity to receive a genetic test to support their diagnosis and determine their potential
eligibility for one of the AGTC sponsored gene therapy clinical trials.
We would like to express our deep appreciation to those patients who have participated in our clinical trials,
and are also grateful to our employees, collaborators and shareholders for their dedication and continued support.
I look forward to a productive year and providing updates on our progress in the months ahead.
Sincerely,
Sue Washer
President and Chief Executive Officer, Applied Genetic Technologies Corporation
January 29, 2018
Forward Looking Statements
This letter contains forward-looking statements that reflect AGTC’s plans, estimates, assumptions and beliefs.
Forward-looking statements include information concerning possible or assumed future results of operations,
business strategies and operations, preclinical and clinical product development and regulatory progress.
Forward-looking statements include all statements that are not historical facts and can be identified by terms
such as “anticipates,” “believes,” “could,” “seeks,” “estimates,” “expects,” “intends,” “may,” “plans,”
“potential,” “predicts,” “projects,” “should,” “will,” “would” or similar expressions and the negatives of
those terms. Actual results could differ materially from those discussed in the forward-looking statements, due to
a number of important factors. Risks and uncertainties that may cause actual results to differ materially from
those described in the forward-looking statements are set forth under the heading “Risk Factors” in the
Company’s Annual Report on Form 10-K, as amended, for the fiscal year ended June 30, 2017, as amended and
filed with the SEC. Given these uncertainties, you should not place undue reliance on these forward-looking
statements. Also, forward-looking statements represent management’s plans, estimates, assumptions and beliefs
only as of the date of this letter. Except as required by law, we assume no obligation to update these forward-
looking statements publicly or to update the reasons actual results could differ materially from those anticipated
in these forward-looking statements, even if new information becomes available in the future.
UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, DC 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 June 30, 2017
OR
(cid:4) TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934
Commission File Number: 001-36370
APPLIED GENETIC TECHNOLOGIES
CORPORATION
(Exact Name of Registrant as Specified in Its Charter)
Delaware
(State or Other Jurisdiction of
Incorporation or Organization)
59-3553710
(I.R.S. Employer
Identification No.)
14193 NW 119th Terrace
Suite 10
Alachua, Florida 32615
(Address of Principal Executive Offices, Including Zip Code)
(386) 462-2204
(Registrant’s Telephone Number, Including Area Code)
Securities registered pursuant to Section 12(b) of the Act:
Title of class
Common Stock, $.001 par value
Name of exchange on which registered
NASDAQ Global Market
Securities registered pursuant to Section 12(g) of the Act: None
Indicate by check mark if the registrant is a well-known seasoned issuer, as defined in Rule 405 of the Securities Act. Yes (cid:4) 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 (cid:4) 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 (cid:4)
Indicate by check mark whether the registrant has submitted electronically and posted on its corporate Web site, if any, every Interactive Data File required to be
submitted and posted 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 and post such files). Yes ⌧ No (cid:4)
Indicate by check mark if disclosure of delinquent filers pursuant to Item 405 of Regulation S-K (§ 229.405 of this chapter) is not contained herein, and will not
be contained, to the best of registrant’s knowledge, in definitive proxy or information statements incorporated by reference in Part III of this Form 10-K or any
amendment to this Form 10-K. ⌧
Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, or a smaller reporting company. See the
definitions of “large accelerated filer,” “accelerated filer” and “smaller reporting company” in Rule 12b-2 of the Exchange Act. (Check one):
Large accelerated filer
(cid:4)
Non-accelerated filer
(cid:4)(cid:5)(cid:5)(Do not check if a smaller reporting company)
Accelerated filer
Smaller reporting company
⌧
(cid:4)
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 Exchange Act). Yes (cid:4) No ⌧
The aggregate market value of the voting common shares held by non-affiliates of the registrant was approximately $154.8 million, computed by reference to
the closing sale price of the common stock as reported by The NASDAQ Global Market on December 31, 2016, the last trading day of the registrant’s most recently
completed second fiscal quarter. The Company has no non-voting common shares.
The number of shares of the registrant’s common stock outstanding as of August 31, 2017 was 18,087,785.
DOCUMENTS INCORPORATED BY REFERENCE
Certain information required to be provided in Part III of this Annual Report on Form 10-K will be provided by an amendment to our Annual Report on Form
10-K/A (the “Annual Report Amendment”) to be filed with the Securities and Exchange Commission on or before October 27, 2017 .
APPLIED GENETIC TECHNOLOGIES CORPORATION
ANNUAL REPORT ON FORM 10-K
FOR FISCAL YEAR ENDED JUNE 30, 2017
TABLE OF CONTENTS
Page
PART I
Item 1.
Item 1A.
Item 1B.
Item 2.
Item 3.
Item 4.
PART II
Business ..............................................................................................................................................................
Risk Factors.........................................................................................................................................................
Unresolved Staff Comments ...............................................................................................................................
Properties ............................................................................................................................................................
Legal Proceedings ...............................................................................................................................................
Mine Safety Disclosures .....................................................................................................................................
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 Accountant Fees and Services .............................................................................................................
PART IV
Item 15.
Exhibits and Financial Statement Schedules ......................................................................................................
SIGNATURES ..............................................................................................................................................................................
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CAUTIONARY NOTE REGARDING FORWARD-LOOKING STATEMENTS
This Annual Report on Form 10-K, including the sections entitled “Business,” “Risk Factors” and “Management’s Discussion
and Analysis of Financial Condition and Results of Operations,” contains forward-looking statements. These statements may relate
to, but are not limited to, expectations of our future results of operations, business strategies and operations, financing plans,
potential growth opportunities, potential market opportunities and the effects of competition, as well as assumptions relating to the
foregoing. Forward-looking statements are inherently subject to risks and uncertainties, some of which cannot be predicted or
quantified. These risks and other factors include, but are not limited to, those listed under “Risk Factors.” In some cases, you can
identify forward-looking statements by terminology such as “may,” “will,” “should,” “could,” “expect,” “plan,” “anticipate,”
“believe,” “estimate,” “predict,” “intend,” “potential,” “might,” “would,” “continue” or the negative of these terms or other
comparable terminology. These statements are only predictions. Actual events or results may differ materially.
There may be events in the future that we are not able to accurately predict or control and that may cause our actual results to
differ materially from the expectations we describe in our forward-looking statements. Except as required by applicable law,
including the securities laws of the United States and the rules and regulations of the SEC, we do not plan to publicly update or revise
any forward-looking statements contained in this Annual Report on Form 10-K after we file it, whether as a result of any new
information, future events or otherwise. Before you invest in our common stock, you should be aware that the occurrence of any of the
events described in the “Risk Factors” section and elsewhere in this Annual Report on Form 10-K could harm our business,
prospects, operating results and financial condition. Although we believe that the expectations reflected in the forward-looking
statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.
As used herein, except as otherwise indicated by context, references to “we,” “us,” “our,” or the “Company” refer to Applied
Genetic Technologies Corporation.
ITEM 1.
BUSINESS
Overview
PART I
Applied Genetic Technologies Corporation (“AGTC”) is a clinical-stage biotechnology company that uses a proprietary gene
therapy platform to develop transformational genetic therapies for patients suffering from rare and debilitating diseases. Our initial
focus is in the field of ophthalmology, where we have active clinical programs in X-linked retinoschisis (XLRS), X-linked retinitis
pigmentosa (XLRP), and achromatopsia (ACHM) and a preclinical program in optogenetics. In addition to ophthalmology, we have
recently initiated preclinical programs in adrenoleukodystrophy (ALD), which is a disease of the central nervous system (CNS) and
otology. With a number of important clinical milestones on the horizon, we believe we are well positioned to advance multiple
programs towards pivotal studies. In addition to our product pipeline, we have also developed broad technological capabilities both
internally and through our collaborations with 4D Molecular Therapeutics (4DMT), Synpromics Limited (Synpromics), and the
University of Florida, which provide us with expertise in vector design and manufacturing as well as synthetic promoter development
and optimization. Finally, our partnership with Biogen, which includes our clinical XLRS and XLRP programs, a discovery program
in ALD and two ophthalmology programs, validates our approach and technology, and provides us with cash runway to advance our
wholly owned candidates.
Our strategy
Our objective is to become a leader in developing and commercializing gene therapy treatments for patients with severe
diseases, with an initial focus in ophthalmology, and thereby provide a better life for patients with these diseases. Our strategy to
accomplish this goal is to:
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Develop and commercialize gene therapies in orphan ophthalmology. Our lead product candidates are treatments for
the severe orphan eye diseases XLRS, ACHM, and XLRP. Given the severity of these diseases and the current lack of
treatment options, a one-time-treatment alternative that corrects the underlying genetic defect would provide superior
long-term value for patients, their families and the healthcare system more broadly.
Expand our position in ophthalmology.
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Continue our leadership position in orphan ophthalmology. We have developed significant experience in the
orphan ophthalmology space through our work on XLRS, ACHM, XLRP and Lebers Congenital Amourosis Type 2
(LCA2) and have significantly advanced our knowledge and experience through the programs that we have
advanced to the clinic since our initial public offering.
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Seek opportunities for strategic partnerships and acquisitions in ophthalmology gene therapy. On July 1,
2015, we entered into a broad collaboration and license agreement with Biogen to develop gene-based therapies for
our XLRS and XLRP programs and three discovery programs. In February 2017, we entered into a collaboration
agreement with Bionic Sight to develop an optogenetic product candidate for patients with advanced retinal disease.
We will seek to develop a new optogenetic therapy that leverages our deep experience in gene therapy and
ophthalmology and Bionic Sight's innovative neuro-prosthetic device and algorithm for retinal coding. We believe
there may be additional opportunities for us to partner with companies and academic groups. We expect that our
breadth of experience in research, manufacturing, clinical and regulatory matters will help us to identify and execute
in-licenses, co-development agreements, intellectual property acquisitions or manufacturing agreements that would
further extend our leadership position in ophthalmology gene therapy.
Extend our expertise in adeno-associated virus, or AAV, vector design, manufacturing and delivery. We believe that
our understanding of our target indications and our robust internal expertise in viral vector design including the
identification of novel capsids and the optimization of genes and promoters, physical vector delivery, vector
manufacturing, clinical trial design and clinical trial conduct are significant competitive advantages. We intend to
continue to devote substantial resources both internally and with others, such as our external research collaborations with
4DMT and University of Florida, to identify next generation capsids and to develop optimized promoters through our
collaboration with Synpromics. We are also expanding our discovery capabilities to further enhance our ability to develop
next generation products.
Expand our manufacturing capabilities. We continue to invest in the development and expansion of our internal
manufacturing capabilities. Our new process development and pilot manufacturing facility is now operational, and as we
advance further into clinical development we plan to further develop our internal manufacturing capabilities. We have
decreased our dependence on a single contract manufacturer by qualifying and contracting with multiple backup
contractors. Further, we continue to invest in process and analytical improvements that we expect to lead to higher
manufacturing yields and robust quality control of our final products. We believe these investments will facilitate the
more rapid advancement of our product candidates through regulatory approval while reducing risk, and will enhance the
therapeutic and commercial potential of our gene therapy platform.
Pursue orphan indications with high unmet medical need and strong probability of a streamlined clinical,
regulatory and commercial pathway. We will continue to focus on diseases for which the underlying genetic defect is
well characterized and can be addressed by correcting or inserting a single gene, for which predictive animal models exist
and for which clinical endpoints are objective and are accepted by the FDA. We believe that focusing on these types of
indications will enable us to obtain data more rapidly and accelerate clinical studies and regulatory approval of our
products. Given the relatively low prevalence of patients who have each of these orphan diseases and the strong key
opinion leader communities and patient advocacy groups around them, we also believe these markets can be served with a
small, targeted commercial infrastructure.
Evaluate opportunities to leverage our gene therapy platform to address indications outside of ophthalmology. We
intend to develop and partner selectively to expand the scope of our pipeline and the utilization of our gene therapy
platform beyond ophthalmology. The adaptability of our platform also presents an opportunity for us to selectively form
collaborative alliances to expand our capabilities and product offerings into a range of genetically defined diseases and
potentially to accelerate the development and commercialization of gene therapy products more broadly. Also, we have
ongoing preclinical efforts in otology and a partnered programs in CNS for which we intend to use our platform
capabilities to develop potential therapies.
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Our Focus in Ophthalmology
Sight is critical to the human experience. Many people fear blindness more than premature death. Consequently, we have
decided to focus our expertise in gene therapy on orphan diseases in ophthalmology. These orphan indications have patient
populations that are small enough to allow for clinical trials on a manageable scale but have a sufficient prevalence to provide
substantial commercial opportunity. By focusing initially on orphan ophthalmology product candidates, we are also able to leverage
our experience and develop strong relationships within the relevant scientific and medical community. Our clinical trials are
conducted mainly at academic test centers and by working with the principal investigators and surgeons at these test centers, we have
realized a number of important synergies.
2
Our most advanced products consist of four ophthalmology development programs across three targets: XLRS caused by
mutations in the RS1 gene, ACHM, caused by mutations in either the CNGB3 gene or the CNGA3 gene, and XLRP caused by
mutations in the RPGR gene. These three inherited orphan diseases of the eye are caused by mutations in single genes that
significantly affect visual function and currently lack effective medical treatments.
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XLRS is characterized by abnormal splitting of the layers of the retina, resulting in poor visual function in young boys,
which can progress to legal blindness in adult men. Additionally, approximately 40% of patients are at risk of vitreous
hemorrhage or detachment. Initial safety data from our Phase 1/2 clinical trial were presented in June 2017 and
demonstrated that our product candidate was generally well tolerated and demonstrated across treatment groups.
According to a published study, the incidence rate for XLRS is between one in 5,000 and one in 20,000 males. Using an
incidence rate of 1 in 11,500 and assuming half the population is male, we estimate that there are about 35,000 patients in
the United States and Europe combined. We currently are enrolling patients in a Phase 1/2 clinical trial for our XLRS
product candidate.
ACHM is characterized by the absence of cone photoreceptor function, resulting in extremely poor visual acuity, light
sensitivity, day blindness and complete loss of color discrimination. According to a published study, the incidence rate for
ACHM is approximately one in 30,000 people, and we therefore estimate that there are about 27,000 patients in the
United States and Europe combined. Of these patients, about 75% have the form of disease caused by mutations in the
CNGB3 gene or the CNGA3 gene. We currently are enrolling patients in Phase 1/2 clinical trials for both our ACHM
CNGB3 product candidate and our ACHM CNGA3 product candidate.
XLRP is a disease of the rod and cone photoreceptors characterized by progressive degeneration of the retina, which can
lead to total blindness in adult men. According to a published study, the incidence rate for retinitis pigmentosa is about
one in 4,000 people and we estimate that there are about 200,000 patients in the United States and Europe combined. It is
estimated that about ten percent, or 20,000, of these people have XLRP. We filed an Investigational New Drug
application (IND) with the FDA for this product candidate on August 9, 2017 and are initiating a Phase 1/2 clinical trial at
multiple sites in the United States.
In addition to these clinical-stage ophthalmology programs, we have a pre-clinical program in collaboration with Bionic Sight to
develop an optogenetic product candidate for patients with advanced retinal disease.
Corporate History and Significant Milestones
On April 1, 2014, we completed our initial public offering in which we sold 4,791,667 shares of common stock and now trade
on NASDAQ under the ticker symbol AGTC.
On July 30, 2014, we completed a follow on public offering in which we sold 2,000,000 shares of common stock.
On June 4, 2015, we started enrolling patients in a Phase 1/2 trial designed to test the safety and efficacy of our gene-therapy
product candidate for the treatment of XLRS.
In July 2015, we entered into a collaboration agreement with Biogen for the development and commercialization of product
candidates in our XLRS and XLRP programs and for the option to license three discovery programs as well as certain access rights to
our manufacturing system.
On May 26, 2016, we started enrolling patients in a Phase 1/2 trial designed to test the safety and efficacy of our gene-therapy
product candidate for the treatment of ACHM caused by mutations in the CNGB3 gene.
In November 2016, we completed the manufacture of our XLRP product candidate clinical trial material, bringing the total
number of batches of clinical trial material that we have produced to seven over a number of clinical trials and disease indications.
In February 2017, we entered into a collaboration agreement with Bionic Sight, LLC to develop a gene therapy treatment to be
used with Bionic Sight’s innovative neuro-prosthetic device and algorithm for retinal coding. Together we are working to develop an
optogenetic therapy for patients with visual deficits or blindness due to retinal disease. This approach could be beneficial to a wide
range of patients as it is independent of the patients’ specific genetic mutation.
In June 2017, we published the results of a follow up study demonstrating durability of treatment with our alpha-1 anti-trypsin
product candidate. The study showed that treatment with our alpha one anti-trypsin product candidate resulted in sustained expression
of alpha one anti-trypsin for five years after intramuscular administration.
3
On July 27, 2017, we started enrolling patients in a Phase 1/2 trial designed to test the safety and efficacy of our gene-therapy
product candidate for the treatment of ACHM caused by mutations in the CNGA3 gene.
In August 2017, we filed an IND to conduct a Phase 1/2 clinical trial designed to test the safety and efficacy of our gene-therapy
product candidate for the treatment of XLRP caused by mutations in the RPGR gene.
Our Strengths
We believe the combination of our technology expertise and product development know-how position us well to be leaders in
the gene therapy field. We believe our strengths include:
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Product candidates in clinical development, including four ongoing Phase 1/2 clinical trials with sufficient capital to
complete enrollment and initial data analysis on all four of these trials;
Significant relationships with key opinion leaders in the fields of ophthalmology, otology, CNS, and AAV production;
Robust preclinical product development pipeline including ophthalmology, otology and CNS disorders;
Biogen collaboration for the development and commercialization of two clinical programs (XLRS and XLRP) and rights
for them to develop three additional discovery programs (ALD and two ophthalmology programs);
A collaboration with Bionic Sight for the development and commercialization of an optogenetic gene therapy and a
neuroprosthetic device;
Proprietary gene therapy manufacturing system capable of making significant quantities of high quality viral vectors and
execution on the system in accordance with Good Manufacturing Practice (GMP) standards over seven different clinical
trials;
Product candidates which, to date, use recombinant AAV vector technology, which is a well-studied, versatile and
efficient gene therapy approach;
Technical expertise in manufacturing and analytical techniques, synthetic promoter development, engineered capsids,
optimized capsids and specialized formulation and delivery techniques; and
Initial safety data in our XLRS Phase 1/2 trial showing that our most advanced gene therapy candidate is generally safe
and well tolerated across treatment groups.
Our gene therapy platform
Although the concept of gene therapy is relatively straightforward, the process of developing and manufacturing vectors capable
of delivering the genetic material safely into a patient’s own cells is highly technical and demands significant expertise, experience
and know-how. Our approach to gene therapy product development is built on our core competencies in three key areas: vector
design, vector manufacturing and vector delivery, each of which is described in further detail below. One of our key capabilities is our
depth of understanding of the complex interplay between the clinical disease, the cells in the patient’s body that need treatment, the
selection of a capsid and a promoter, the design of the gene construct and the physical administration method. We have spent more
than 15 years conducting research on the best combinations of these elements with the aim of developing safe and effective product
candidates.
Vector Selection Overview. The success of a gene therapy platform is highly dependent on the vector selected. Our platform is
based on the use of a modified version of the non-replicating AAV to deliver the correct DNA directly to the nucleus of the cells
affected by the disease. As an underlying platform, we believe that AAV vectors are particularly well suited for treating our target
diseases and have advantages over other viral vectors, such as adenovirus, herpes virus and lentivirus. These advantages include:
(cid:129) AAV is a small, simple non-enveloped virus with only two native genes, which makes the virus easy to engineer as an
effective vector;
(cid:129) AAV is inherently stable and resistant to degradation;
(cid:129) AAV vectors are capable of delivering functional genes in a manner that supports long-term production of protein, leading
to sustained therapeutic effect, without altering the patient’s native DNA;
(cid:129) AAV vectors have a demonstrated safety profile across multiple human clinical trials in several indications; and
(cid:129) AAV vectors are versatile, having the ability to carry therapeutic gene sequences of up to 4,000 base pairs in length into a
patient’s cell. As more than 90% of human genes have coding sequences less than 3,000 base pairs in length, this allows
AAV vectors to be used in a wide variety of indications.
4
Vector design. After the selection of the vector type, there are other critical factors to be considered to maximize the safety and
efficacy of the final gene therapy product:
(cid:129) Gene of Interest: The first step in vector design is to identify the therapeutic protein that we want the patient’s own cells to
produce. The protein is expressed from a DNA sequence that defines the gene of interest. In many cases the DNA sequence
must be engineered to be stable during manufacturing and delivery.
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Promoter: Production of the protein requires a promoter, which is a genetic element that drives expression. Certain
promoters function well only in certain cell types, whereas other promoters function well in almost any cell type. We make
our selection by comparing different promoters in the specific type of cells that are affected in each disease target, ideally in
an animal whose physiology is close to that of humans, to find the promoter that best enables production of therapeutic
levels of protein in that cell type. Not only do we engineer these promoters in-house, we also work with a collaborator,
Synpromics, that specializes in designing promoter elements to optimize therapeutic constructs for maximum expression
with a smaller size, better expression and increased cell specificity.
Capsid: after the promoter and gene of interest are selected, these elements must be packaged into the AAV capsid. There
are thousands of variations of AAV capsids with different abilities to bind to and enter varying cell types. Not only do we
engineer these capsids in-house, we also collaborate with commercial and academic researchers to develop novel capsids
that efficiently enter the type of cells that are affected by each of our targeted diseases.
Vector manufacturing
We have developed a proprietary, high-yield vector manufacturing process using scalable technologies. While our
manufacturing method uses the herpes virus as a helper in the first step of a four-step AAV vector manufacturing process, there is no
herpes virus in the final product. Our propriety process for AAV manufacturing uses robust cell lines that are well characterized and
have been included in several regulatory submissions in the United States and Europe. This process is highly efficient and selective,
generating more packaged vector with higher fidelity of target sequences than other production systems. We have developed and
transferred over 30 robust and quantitative product-specific assays consistent with expected requirements for regulatory approval, and
we supplement our deep manufacturing experience with first-in-class characterization of the resulting candidate therapeutics.
Our manufacturing process has been reviewed by the FDA, the Irish Medicines Board, and the Israeli Ministry of Health and has
been authorized for production of product candidates for use in clinical trials in the United States, Europe and Israel. To date we have
successfully manufactured clinical trial material for seven different indications using three different manufacturing vendors, to ensure
sufficient capacity, and believe we are the only AAV gene therapy company with this level of experience. Our manufacturing process
is reproducible and scalable. Our new process development facility is operational and we are conducting equipment evaluation runs
with multiple vendors to support development of commercial processes for our product candidates. Further we have completed design
work on an internal cGMP facility at our Florida headquarters.
We own or have licensed 64 issued patents and 12 pending patent applications covering our manufacturing technology. We
believe that our core competency and intellectual property estate in vector manufacturing differentiate us competitively and provide a
key element of our gene therapy platform.
Vector delivery
Our gene therapy platform allows for vector delivery by a variety of methods, and we select the method that is best suited for the
disease and cell type that we are targeting.
In ophthalmology, the product candidate can best be delivered to cells in the eye by either injecting the product candidate into
the vitreous of the eye, an intravitreal injection, or by injecting the product candidate under the retina, a subretinal injection. Our
ongoing clinical trial for our XLRS product candidate uses intravitreal injection, which we also expect to use as the method of delivery
for our optogenetic product candidate. We are using subretinal injection as the method of delivery for our ACHM and XLRP product
candidates in our ongoing clinical trials.
Surgical techniques used to introduce AAV in otology indications include microinjection into the cochlea via an apical
cochleostomy or through the round window membrane. Like the eye, the inner ear sensory organ – the organ of Corti – is bathed by
fluid-filled spaces, enabling accessible vector administration.
Once a product candidate is identified in our CNS discovery program, we expect it will be administered by intrathecal delivery,
which is an injection into the spinal canal.
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Our Product Candidate Pipeline
The graphic below summarizes our current gene therapy programs:
Our four most advanced product candidates address ophthalmology indications XLRS, ACHM B3, ACHM A3, and XLRP,
which are orphan diseases of the eye that are caused by mutations in single genes, significantly affect visual function starting at birth
and currently lack effective medical treatments. Ophthalmology is attractive to us as a clinical stage company because treatments for
diseases affecting vision have clearly defined, objective clinical endpoints with validated measurement tools that are accepted by the
United States FDA. Other orphan drug companies have spent considerable time and resources working with the FDA to identify
acceptable clinical endpoints and develop measurement tools in sometimes ill-defined diseases. In ophthalmology four accepted
endpoints—visual acuity, visual fields, contrast sensitivity and color vision—are well understood by clinicians. In addition, the FDA
consistently applies these endpoints and works with industry to provide guidance on how much improvement is required for clinical
relevancy. We believe that these end points could help accelerate the process of clinical study and regulatory approval for our
ophthalmic products.
Finally, through our internal research work and in collaboration with partners, we have obtained preliminary safety data in
clinical trials using delivery routes by both intravitreal and subretinal injection. In June 2017, we released topline safety data from the
dose escalation portion of our XLRS trial indicating that our product candidate delivered by intravitreal injection was generally safe
and well tolerated across treatment groups. Subretinal injections of AAV vectors have also been studied in other clinical trials
conducted by us (wet AMD and LCA2) and there were no reports of serious adverse events attributed to the vector used in either of
those studies. Although there were differences in clinical outcomes in the initial three patients enrolled in our ACHM B3 trial, these
results were most likely due to differences in surgical technique rather than the candidate drug product. We have made changes to our
clinical study to address the differences in outcomes by using the same surgeon in the near term to treat patients in this trial.
Our lead programs
X-linked Retinoschisis (XLRS)
XLRS is an inherited retinal disease, meaning that children are born with the defective gene that causes poor visual function that
significantly affects daily living activities. XLRS is specifically caused by mutations in the RS1 gene, which is located on the
X chromosome and encodes the retinoschisin, or RS1, protein. Retinoschisin is expressed and secreted primarily from photoreceptor
cells and binds strongly and specifically to the surface of photoreceptor and bipolar cells in the retina. Mutated forms of retinoschisin
are unable to bind properly, resulting in schisis, or splitting of the layers of the retina, primarily in the macula. The disease begins
early in childhood, and affected boys typically have best-corrected visual acuity of 20/60 to 20/120 at initial diagnosis. Complications
such as vitreous hemorrhage or retinal detachment occur in up to 40% of patients, especially in older patients. According to a
published study, the incidence rate for XLRS is between one in 5,000 and one in 20,000 males. Using a conservative incidence rate of
1 in 11,500 and assuming half the population is male, we estimate that there are about 35,000 persons with XLRS in the United States
and Europe combined with XLRS.
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The diagnosis of XLRS is made based on clinical findings and results of imaging studies and Electroretinogram (ERG). ERG is
a clinical test that measures the electrical function of the retina. Clinical findings include reduced visual acuity and a characteristic
spoke-wheel appearance of the macula when viewed by an ophthalmoscope, which is the instrument commonly used by
ophthalmologists and optometrists to view the retina. Images obtained by optical coherence tomography, or OCT, a method of
viewing layers of the eye somewhat like a sonogram, show spaces between the layers of the retina within the macula and fovea in
most school-age boys with XLRS. These spaces mean that electrical signals cannot move from the photoreceptors to other retinal
neurons and on to the brain, resulting in poor vision.
There is currently no approved treatment for. Management of disease manifestations includes low vision aids such as large-print
textbooks, preferential seating in the front of the classroom and use of handouts with high contrast. Surgery may be required to
address complications of vitreous hemorrhage or full-thickness retinal detachment, which occur in approximately 40% of patients.
Previous anecdotal evidence has indicated that treatment with carbonic anhydrase inhibitors may provide a reduction in the
degree of schisis. However, data from our natural history study demonstrated that topical carbonic anhydrase inhibitors provided
reduction in the degree of schisis in only some patients. In any case, the absence of controlled clinical trials makes interpretation of
these data difficult. In addition, treatment with carbonic anhydrase inhibitors does not address the fundamental genetic defect in
persons affected by XLRS. Neither carbonic anhydrase inhibitors nor any other medicinal products have been approved by regulatory
agencies for treatment of XLRS.
Our XLRS product candidate
Our gene therapy approach involves using an AAV vector to insert a functional copy of the RS1 gene into the patient’s retinal
cells, thereby inducing those cells to produce the normal retinoschisin protein. Our XLRS product candidate contains the RS1 gene
and a promoter that has been shown to work well in primate retinal cells, and is packaged in a proprietary engineered AAV capsid that
is able to efficiently enter target cells in the inner layers of the retina after intravitreal injection.
Preclinical proof of concept for our XLRS product candidate
In mouse models of XLRS, our gene therapy approach restores to normal the abnormal ERG response that is characteristic of
XLRS. Mouse models of XLRS have been developed by deactivating, or knocking out, the RS1 gene. These “knockout” mice have
clinical features similar to humans with XLRS, including reduced visual acuity, schisis cavities detected by OCT, and a markedly
abnormal ERG response.
A study published in 2005 looked at staining for retinoschisin and for nuclei in retinal cells in a normal mouse, a RS1 knockout
mouse in the absence of treatment, and a RS1 knockout mouse treated with an AAV-RS1 vector. The knockout mouse retina had no
expression of retinoschisin and had splitting and disorganization of the layers of the retina. After treatment, RS1 staining was present
in a normal fashion and the nuclear staining showed restoration of the organization of the cell layers in the retina. In another
experiment, treatment by injection of an AAV vector expressing either mouse or human RS1 in these knockout mice improved visual
function.
Because of the fragile nature of the patient’s eyes we concluded that intravitreal injection is the preferred route of administration
for the AAV-RS1 vector. We therefore evaluated intravitreal injection of an AAV vector expressing a marker protein packaged in
several different AAV capsids in monkeys and demonstrated that a vector packaged in an engineered capsid was able to target
expression to the primate macula, which is the primary area in which retinoschisis occurs.
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Clinical development of our XLRS product candidate
We are currently enrolling patients in a Phase 1/2 clinical trial, the primary endpoint of which is safety. Secondary endpoints
include efficacy analyses and biological activity. The current clinical protocol, illustrated below, anticipates enrollment of up to 27
patients.
The Phase 1/2 clinical trial is currently being conducted at six clinical sites that specialize in inherited retinal diseases. We have
completed treatment of patients in groups 1a, 1b, 2 and 3 of the dose escalation phase of our Phase 1/2 clinical trial and we currently
are enrolling adult patients in group 4 at the highest dose level and children between 6 and 18 in group 2b at the mid-dose group prior
to making a decision to dose children between 6 and 18 at the highest dose level.
In June 2017, Mark Pennesi, M.D., Ph.D., one of our investigators, presented safety data at the Macula Society Annual Meeting
from the first twelve patients, some of which have been followed for more than one year. The data demonstrates that our XLRS
product candidate has been generally well tolerated. We did observe mild to moderate ocular inflammation in the majority of patients
in the treated eye, which resolved either without treatment or after treatment with topical or oral corticosteroids. We amended the
protocol to provide for standard prophylactic corticosteroids versus the original protocol that allowed for treatment with
corticosteroids only after inflammation was observed. No treatment related serious adverse events have been reported.
We have also completed a natural history study in persons affected by XLRS. The results from 56 participants with XLRS were
evaluated on cross sectional and longitudinal measures of visual acuity, visual field sensitivity, microperimetry, cyst volume and ERG.
The data suggest that patients with XLRS show a spectrum of visual function, with baseline visual acuity ranging from 0 to 82
ETDRS letters (average = 55.5; approximately 20/80). Changes in measures of visual function were minor and, in fact, stable over
time and weakly correlated to changes in cyst volume or age.
In the patients dosed to date in our Phase 1/2 trial, we have continued to note variation in visual function from patient to
patient. We believe that in addition to the three patients initially enrolled in the high-dose group, the full group of patients in the
expansion group need to be enrolled and followed for at least six months before meaningful conclusions can be drawn regarding
biological activity. We expect to fully enroll this expansion group by the first quarter of 2018 and will present complete data after the
last patient has been followed for six months. To date, we have not seen evidence of biological activity in this group of patients,
which is not unexpected given the small patient pool, the relatively short period of observation in the high dose group and the
variability in the patient population.
Completion of the Phase 1/2 clinical trial and the natural history study will guide us, and our collaborator Biogen, in finalizing
the design of a pivotal clinical trial. In the planned pivotal trial, we would expect to enroll between 40 and 75 patients who will be
evaluated for changes in visual function over a 12-month period. If successful, we believe the results of this second trial could support
submission of a Biologics License Application, or BLA, to the FDA in the United States and a Marketing Authorization Application,
or MAA, to the EMA in Europe for our XLRS product candidate.
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As a part of our collaboration, Biogen has obtained worldwide commercialization rights for the XLRS program. We will be
responsible for the clinical development program through product approval. Biogen will support the clinical development costs,
subject to certain conditions, following the first-in-human study. We have an option to share development costs and profits after the
initial clinical trial data are available.
Achromatopsia (ACHM)
ACHM is an inherited retinal disease, meaning that children are born with the defective gene that causes poor visual function,
which significantly affects daily activities. ACHM is present from birth and throughout life and is characterized by a lack of cone
photoreceptor function. Cone photoreceptors which are concentrated in the macula and the fovea, respond to moderate or bright
intensity light and mediate fine visual acuity. Individuals with ACHM have markedly reduced visual acuity, photophobia or light
sensitivity, and complete loss of color discrimination. Their only functioning photoreceptors are rod photoreceptors, which respond to
low intensity light conditions and mediate night vision but cannot achieve fine visual acuity. Best-corrected visual acuity in persons
affected by ACHM, even under subdued light conditions, is usually about 20/200, a level at which people are considered legally blind.
They also experience extreme light sensitivity resulting in even worse visual acuity under normal daylight conditions, or day
blindness.
ACHM can be caused by mutations in any of at least five genes that are required for normal cone photoreceptor function. The
most common causes are mutations in the CNGB3 gene (about half of all cases) or CNGA3 gene (about one-fourth of all cases).
These genes encode the CNGB3 and CNGA3 proteins, which combine to form a channel in the photoreceptor membrane that is
required for phototransduction, the process of converting light into electrical signals that the brain can understand. According to
published reports, the incidence rate for ACHM is approximately one in 30,000 people, and we therefore estimate that there are about
10,000 people in the United States and about 17,000 people in Europe with ACHM. Of these, about half, or a total of 13,500 in the
United States and Europe combined, have the form of the disease caused by mutations in the CNGB3 gene and approximately 6,800
people have the form of the disease caused by mutations in the CNGA3 gene.
Our ACHM product candidates
Our gene therapy approach to treatment of ACHM involves using an AAV vector to insert a functional copy of the CNGB3 or
CNGA3 gene into the patient’s photoreceptor cells. Our ACHM product candidates contain either the CNGB3 or the CNGA3 gene
and a proprietary cone specific promoter that has been shown in preclinical studies to drive efficient gene expression in all three types
of primate cone photoreceptors and restores cone photoreceptor function in dog, mouse and sheep models of ACHM.
Preclinical proof of concept for our ACHM product candidates
In mouse, dog and sheep models of ACHM, our product candidates were able to restore photoreceptor function, improve visual
acuity and mitigate photophobia and day blindness.
ACHM naturally occurs in two breeds of dogs. Treatment by subretinal injection of an AAV vector expressing human CNGB3
restored cone function in both breeds of dogs. Cone-specific ERG responses were undetectable in these dogs before treatment but
were clearly detected after treatment. Day blindness was demonstrated before treatment by testing the ability of the dogs to navigate a
maze under progressively brighter conditions. Before treatment, it took the ACHM dogs progressively longer to navigate the maze as
the ambient light increased from dim light to normal room lighting and even longer with normal outdoor daytime lighting. After
treatment, the day blindness was substantially eliminated, and the treated ACHM dogs were able to navigate the maze under bright
light conditions at almost the same speed as normal dogs.
Untreated ACHM dogs also demonstrated photophobia and day blindness when outdoors in daylight, which severely limited
their ability to interact with people and objects in their environment. After treatment, there was a dramatic improvement in these
important clinical manifestations of ACHM. The restored function persisted for more than 2.5 years (the longest duration tested).
In addition, a mouse model of ACHM was developed by knocking out the CNGB3 gene. Treatment by subretinal injection of an
AAV vector expressing human CNGB3 in the knockout mice improved cone-specific ERG responses to nearly normal levels and
improved visual acuity, as measured by their ability to follow a rotating pattern of vertical stripes of varying thickness.
There are also sheep models of ACHM caused by mutations in the CNGA3 gene. These sheep have clinical characteristics
similar to human ACHM patients, with day blindness and absence of retinal cone function as measured by ERG. In collaboration with
scientists at three academic centers in Israel, we conducted a preclinical study evaluating the safety and efficacy of our product
candidate for CNGA3-related ACHM using one of the sheep models, which uses the same promoter and capsid used in our CNGB3
product candidate, administered by subretinal injection. Cone-specific ERG responses, which were undetectable in these sheep before
treatment, were clearly detected after treatment. Day blindness was demonstrated before treatment by testing the ability of the sheep to
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navigate a maze under bright light conditions. After treatment, the day blindness was substantially reduced, and the treated sheep were
able to navigate the maze under bright light conditions. At both the higher and lower doses tested, the maze transit time was similar,
and clearly improved over the pre-dose performance.
Humans and non-human primates (NHP), unlike lower mammals, have three sets of cones, so we also conducted studies in NHP
to select both a capsid and promoter that would allow expression of the protein in all three primate cone types. These studies showed
that our proprietary promoter allowed for robust expression in red, green and blue cones.
Clinical development of our CNGB3-related ACHM product candidate
We are currently enrolling patients in a Phase 1/2 clinical trial in which the primary endpoint is safety. Secondary endpoints
include efficacy analysis and biological activity. The current clinical protocol, illustrated below, anticipates enrollment of up to a total
of 26 ACHM CNGB3 patients.
The Phase 1/2 clinical trial is being conducted at four clinical sites that specialize in inherited retinal diseases. An additional site
is performing advanced optical testing on patients.
We are continuing to enroll the low dose group. Due to differences in surgical technique in the first three patients, we expanded
group 1 to include an additional two patients bringing the total number of patients treated in group 1 to five. In order to minimize
differences in surgical technique, we decided to use the same surgeon to treat the next two patients such that three of the first five
patients will have been treated by the same surgeon. This surgeon will treat both additional patients prior to presenting data to the
DSMC.
We have completed enrollment in a natural history study in persons affected by ACHM caused by CNGB3 mutations and results
from the study will be presented in appropriate scientific meetings and publications.
Completion of the Phase 1/2 clinical study and the natural history study will guide us in finalizing the design of a pivotal clinical
trial. In the planned pivotal trial, we expect that between 40 and 75 patients will be enrolled and evaluated for changes in visual
function over a 12-month period following treatment. If successful, we believe the results of this pivotal trial could support our
submission of a BLA to the FDA and of an MAA to the EMA for our ACHM-B3 product candidate.
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Clinical development of our CNGA3-related ACHM product candidate
A Phase 1/2 clinical trial in which the primary endpoint is safety is open and patient scheduling is ongoing. Secondary
endpoints include efficacy analysis and biological activity. The current clinical protocol, illustrated below, anticipates enrollment of
up to a total of 24 ACHM CNGA3 patients.
The Phase 1/2 clinical trial is being conducted at five sites that specialize in inherited retinal diseases, four of which are in the
United States and one of which is in Israel. An additional site is performing advanced optical testing on patients. Based on our ACHM
CNGB3 experience, we have made adjustments to this trial and intend to use the single surgeon discussed above with regard to our
ACHM CNGB3 trial for the near term.
We have nearly completed enrollment in a natural history study in persons affected by ACHM caused by CNGA3 mutations and
results from the study will be presented in appropriate scientific meetings and publications.
Completion of the Phase 1/2 clinical study and the natural history study will guide us in finalizing the design of a pivotal clinical
trial. In the planned pivotal trial, we expect that between 40 and 75 patients will be enrolled and evaluated for changes in visual
function over a 12-month period following treatment. If successful, we believe the results of this pivotal trial could support our
submission of a BLA to the FDA and of an MAA to the EMA for our ACHM-A3 product candidate.
X-linked Retinitis Pigmentosa (XLRP)
Retinitis pigmentosa is an inherited retinal disease with progressive loss of vision, meaning children are born with defective
genes that cause poor visual function that significantly affects daily activities and worsens over time. XLRP is commonly first
observed in boys and young men who notice problems with vision under low light conditions, or night blindness, followed by a
restriction of peripheral visual fields, or tunnel vision, leading to poor central vision and eventually to total blindness.
The incidence rate for retinitis pigmentosa is about one in 4,000 people, according to a published study, and we estimate that
there are about 75,000 people in the United States and 125,000 people in Europe with retinitis pigmentosa, or 200,000 people in the
United States and Europe. According to a published study, about 10% of cases of retinitis pigmentosa are XLRP, from which we
therefore estimate that there are about 20,000 persons with XLRP in the United States and Europe combined.
Our XLRP product candidates
Our gene therapy approach to treatment of XLRP involves using an AAV vector to insert a functional copy of the RPGR gene
into the patient’s photoreceptor cells. Our XLRP product candidates contain an optimized and stabilized RPGR gene and a promoter
that has been shown in preclinical studies to drive efficient gene expression in primate rods, cones and photoreceptors and restores
photoreceptor function dog and mouse models of XLRP.
Pre-clinical development of our XLRP Product
Preclinical studies in a dog model of XLRP caused by mutations in the RPGR gene demonstrated a reduction in the rate of
disease progression in eyes that received a subretinal injection of an AAV vector expressing a codon-optimized form of the RPRG-
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ORF15 gene. A comparative dose range finding study of our XLRP product candidate in dogs demonstrated expression of the RPGR
protein in both cone and rod photoreceptors, and improvement in structural and functional parameters associated with disease
progression in this model. IND-enabling good laboratory practices, or GLP, toxicology studies have been completed in two relevant
disease models (a dog model of the disease and in an RPGR knockout mouse model), providing guidance on the appropriate choice of
dose levels to test in initial clinical studies.
Clinical development of our XLRP product candidate
On August 9, 2017, we filed an IND with the FDA in support of a Phase 1/2 trial for this product candidate.
The Phase 1/2 clinical trial is expected to be conducted at four clinical sites that specialize in inherited retinal diseases. We are
currently conducting site initiation activities at the four clinical sites.
We are conducting a natural history study in patients with XLRP caused by RPGR mutations. This study documents the
progression of the disease in the absence of treatment and is providing important information about the best methods for measuring
visual function and other parameters in these patients, which will guide us in the design of subsequent clinical trials in which our
product candidate will be tested for safety and efficacy.
Completion of the Phase 1/2 clinical study and the natural history study will guide us, and our collaborator Biogen, in finalizing
the design of a pivotal clinical trial. In the planned pivotal trial, we expect that between 80 and 120 patients will be enrolled and
evaluated for changes in visual function over a 24-month period following treatment. If successful, we believe the results of this
pivotal trial could support our submission of a BLA to the FDA and of an MAA to the EMA for our XLRP product candidate.
As a part of our collaboration, Biogen obtained worldwide commercialization rights for our XLRP program. We will lead the
clinical development program through the completion of first-in-human trials. Biogen will support the clinical development costs,
subject to certain conditions, following the IND-enabling studies. We have an option to share development costs and profits after the
initial clinical trial data are available, and an option to co-promote the second of two products (XLRS and XLRP) to be approved in
the United States.
Advanced Retinal Disease
As part of our collaboration with Bionic Sight we are developing an optogenetic candidate treatment for individuals having
retinitis pigmentosa (RP) who have lost light sensitivity. RP is a large group of inherited retinal disorders in which progressive
degeneration of photoreceptors or retinal pigment epithelium (RPE) leads to vision loss which is independent of a patients’ genetic
mutation. In Europe and the United States, about 200,000 patients suffer from RP and every year between 15,000 and 20,000 patients
with RP suffer vision loss. The clinical manifestations of affected individuals present first as defective dark adaptation or “night
blindness,” followed by reduction of peripheral visual fields and, eventually, loss of central vision. While the photoreceptor cell layers
of these patients degenerate, the ganglion cell layer remains intact.
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Optogenetics is a biological technique by which cells are modified to express light-sensitive proteins. These light sensitive
proteins open or close in response to light and allow millisecond-scale temporal manipulation of electrical events. Therefore,
optogenetics provides a way to manipulate the activity of cells by controlling these proteins with light. A number of studies have been
published on the effectiveness of these proteins for treating RP in animal models. Other studies have shown their effectiveness
supplemented with a vision-enhancing device known as a retinal prosthesis. The device receives images, processes them, and then
converts them into the same patterns of electrical pulses that the brain normally receives from normally functioning photoreceptors.
The candidate treatment being developed by AGTC and Bionic Sight is a recombinant AAV that expresses an optogenetic
protein, ChronosFP, in the retinal ganglion cells. Light then activates the ChronosFP to send electrical signals from the retinal
ganglion cells to the brain. Bionic Sight is developing a device with a retinal code that can provide light signals to the retinal ganglion
cells that will result in an image the brain can recognize and may significantly enhance vision in patients who have received the
optogenetic treatment. Our collaborator, Bionic Sight, plans to file an IND for this product candidate for the treatment of RP in 2018.
Other opportunities in ophthalmology
We believe our current gene therapy platform will enable us to develop and test new AAV vectors that carry gene sequences for
other inherited diseases in ophthalmology (it is estimated that approximately 290 genes causing inherited retinal disease have been
identified), and that by leveraging our work on our lead programs we can reduce the need for early research work. In this way, we
anticipate being able to move products efficiently through preclinical studies and into clinical development. As an example, in January
2016 we announced a research effort in collaboration with the Blue Cone Monochromacy Family Foundation, in blue cone
monochromacy (BCM), a rare genetic disease of the retina that almost exclusively affects males. We also believe that there are large-
market ophthalmology diseases where AAV vectors may provide benefit, such as wet AMD.
Central Nervous System
Our collaboration with Biogen includes options for three discovery programs, two in ophthalmic diseases and one in a non-
ophthalmic condition. On February 8, 2016, we announced that Biogen had selected adrenoleukodystrophy (ALD) as the indication
for the non-ophthalmic discovery program.
ALD is an X-linked disorder of fatty acid metabolism that leads to accumulation of very long chain fatty acids in tissues
throughout the body, mainly in the central nervous system and the adrenal gland. Clinically, ALD is a heterogeneous disorder with
several distinct phenotypes, including rapidly declining neurological function and early death in young boys or progressive muscular
weakness leading to lower limb paralysis in adults. There are approximately 14,000 patients with ALD in the United States. We are
working with Biogen on vector design, animal model proof of concept and targeting studies in non-human primates in order to obtain
data to support moving a potential product candidate to IND enabling studies.
Otology
We continue developing and improving our products through pre-clinical work in order to add to our clinical candidate pipeline.
On May 9, 2016, we announced a new initiative in the area of otology in which we will use our capabilities to develop potential gene
therapy products that can address genetic causes of hearing loss. Hearing loss is one of the most common human sensory deficits and
it is estimated that nearly half of the cases are suspected to have a genetic origin. Of the inherited forms of hearing loss, more than 300
genetic causes have been defined with the specific gene identified for more than 70. Despite the impairment that can be caused by
deafness, very little progress has been made in developing therapies that go beyond the temporary and partial solutions provided by
hearing aids and cochlear implants. In multiple academic research studies, replacement of defective genes in animal models with
normal copies has been shown to improve sound propagation in the auditory hair cells, making this a potentially promising application
of AAV gene therapy. Additionally, the inner ear shares many of the characteristics that make ophthalmology attractive: it is
anatomically well defined and is a small, well contained space where the target cells to be treated are easily identified. In addition, the
clinical outcome measures for treatments for hearing loss are well defined. Developing product candidates for conditions having these
characteristics is a natural complement to our ophthalmology portfolio strategy as we apply our core capabilities and expertise to a
new disease field. As part of our efforts in otology, we have formed a scientific advisory board to help guide our target selection and
will announce product candidates when we formally begin development plans.
Research Programs
Additionally, in order to develop optimized next generation capsids to target specific cell populations within the human retina
we continue to collaborate with 4DMT. After extensive library screening in non-human primates, a shortlist of potential novel capsid
variants with improved penetration after intravitreal injection, to all cell layers of the retina, in particular photoreceptors, are being
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further characterized. We have an option to enter into a licensing agreement if the confirmatory studies are successful. We also have a
licensing arrangement with the University of Florida where we are screening their extensive library in a similar fashion.
We continue to collaborate with Synpromics to use their proprietary technology to develop and optimize synthetic
promoters. Promising novel synthetic promoter candidates specific for retinal cell types, such as RPE, have been identified and are
undergoing further testing in vivo. We have an option to enter into a licensing agreement if the Synpromics develops improved
promoters that we decide to use in our clinical candidates.
Manufacturing
Historically, there has been a lack of manufacturing infrastructure to enable the production of gene therapies in a reliable and
reproducible manner at a commercially viable scale. The historical challenges for gene therapy manufacturing relate to the difficulty
of developing constructs that provide the necessary helper functions, and in having systems that provide adequate yield, scalability and
purity. We have made significant investments in developing improved manufacturing processes, which include the following:
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Our proprietary manufacturing method for AAV production is highly efficient and selective, generating more packaged
vector with higher fidelity of target sequences than other methods.
We have developed proprietary AAV vector manufacturing processes and techniques that produce a more purified and
concentrated product candidate,
Our propriety platform for AAV production uses robust cell substrates that are well characterized and have been in several
regulatory submissions in the US, Israel and Europe.
We do not need a specially cloned and isolated cell line for each of our disease targets; we instead use specially
engineered replication-incompetent herpes simplex helpers, or HSV helpers, which are stable and straightforward to
clone.
We have developed over 30 assays to accurately characterize our process and the HSV and AAV vectors we produce.
We have developed a purification system applicable to multiple AAV capsids.
We are investing in the development of mid- to large-scale manufacturing processes to enable the manufacture of our
product candidates at commercial scale.
We believe these improvements and our continued investment in our manufacturing platform will enable us to develop best-in-
class, next generation gene therapy products and that our viral vector production platform for AAV-based gene therapies offers
significant benefits in comparison with the methods used by others to manufacture AAV vectors.
Strategic collaborations
We have formed strategic alliances in which both parties contribute expertise to enable the discovery and development of
potential gene therapy product candidates. To access the substantial funding and other resources required to develop and
commercialize gene therapy products, we intend to seek additional opportunities to form strategic alliances with collaborators who can
augment our industry-leading gene therapy expertise.
On July 1, 2015, we entered into a broad collaboration and license agreement with Biogen to develop gene-based therapies for
multiple ophthalmic diseases. Biogen made an upfront payment to us in the amount of $124.0 million, which included a $30.0 million
equity investment and certain prepaid research and development expenditures. Biogen was granted a license to the XLRS and XLRP
programs and the option to license discovery programs for two additional ophthalmic indications and one non-ophthalmic indication at
the time of clinical candidate selection. In February 2016, we announced Biogen’s selection of adrenoleukodystrophy, or ALD, as the
non-ophthalmic indication of the three discovery programs. Under the collaboration, we are eligible to receive upfront and milestone
payments exceeding $1 billion. This includes up to $472.5 million collectively for the two lead programs, which also will carry
royalties in the high single digit to mid-teen percentages of annual net sales. In addition, Biogen may make payments up to $592.5
million across the discovery programs, along with royalties in the mid-single digits to low teen percentages of annual net sales for the
discovery programs. In October 2015, Biogen paid us an additional $5.0 million upon achievement of the first enrollment milestone
for XLRS.
Biogen will also receive an exclusive license to use our proprietary manufacturing technology platform to make AAV vectors
for up to six genes, three of which are chosen at our discretion, in exchange for payment of milestones and royalties.
On February 2, 2017, we entered into a strategic research and development collaboration agreement with Bionic Sight to
develop therapies for patients with visual deficits and blindness due to retinal disease. Through the AGTC-Bionic Sight collaboration,
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the companies seek to develop a new optogenetic therapy that leverages AGTC’s deep experience in gene therapy and ophthalmology
and Bionic Sight’s innovative neuro-prosthetic device and algorithm for retinal coding.
Under the agreement, AGTC made an initial $2.0 million payment to Bionic Sight for an equity interest in that company. This
initial investment represents an approximate 5 percent equity interest in Bionic Sight. In addition to the initial investment, AGTC will
contribute to ongoing research and development support costs through additional payments or other in-kind contributions. These
payments and contributions will be made over time, up to the date that Bionic Sight has received both investigational new drug
clearance from the FDA and receipt of written approval from an internal review board to conduct clinical trials from at least one
clinical site for that product.
AGTC will receive additional equity, based on the valuation in place at the beginning of the agreement, for its cash and in-kind
research and development contributions and will be obligated to purchase additional equity in Bionic Sight for $4.0 million, upon the
filing of an IND for the product at a pre-determined valuation.
We have also entered into an agreement with SAFC, one of our current contract manufacturing organizations, for cGMP
manufacture of clinical grade material for third parties. This arrangement allows us to approach other gene therapy companies that
might benefit from our manufacturing and vector design capabilities. Under such an arrangement, we could potentially license our
manufacturing technology and receive upfront payments, milestones and royalties. SAFC would do the manufacturing of commercial
grade material.
We will continue to in-license additional intellectual property to support our current programs, to establish new development
programs and to support our manufacturing technology. Additionally, we will seek to partner with other commercial gene therapy
companies and academic institutions to leverage our expertise in vector design, research, manufacturing and the regulatory process.
The goal of these collaborations would be to forge strategic partnerships around technologies and programs that would fit with our
current and future development pipeline. In general, we would seek new intellectual property, development programs in rare diseases,
pipeline products where the regulatory pathway is understood, partners with strong scientific, clinical and management expertise, and
programs that have synergy with our current knowledge base and product pipeline that would add to our industry leadership. We
would also be looking at programs where the disease being treated has a large enough patient population that there would be adequate
financial returns for the investment of resources.
Our relationship with the University of Florida
All of our scientific founders spent part of their careers at the University of Florida, or UF, and three are still UF faculty
members. Since our inception we have licensed significant technology from and funded research at multiple labs at UF. Pursuant to
four agreements, we have licensed three U.S. patents and multiple pending applications covering inventions made at UF. UF has
multiple capabilities in genetic cloning, gene therapy manufacturing, novel capsid identification, animal model development and
facilities for both small and large animal testing, and in certain instances we have benefited from the ability to conduct important
research at UF without having to expand in-house facilities and personnel. We interact frequently with the Powell Gene Therapy
Center at UF and have an excellent working relationship with the UF Office of Technology Licensing.
In May 2013, we and UF were jointly awarded an $8.3 million grant from the National Eye Institute to support development of
our ACHM CNGB3 product candidate, with Dr. William Hauswirth, one of our scientific founders a Professor and holder of the
Rybaczki-Bullard Chair in the Department of Ophthalmology at UF, as principal investigator. As a sub-awardee, we expect to receive
approximately $3.8 million over five years under this grant. As of June 30, 2017, we have received payments in the aggregate amount
of $2.8 million under this grant.
Our relationships with patient advocacy groups and academic centers
We have long believed that when developing products to treat orphan indications it is important to form strong relationships
with patient advocacy groups, and we have done this successfully with both the Foundation Fighting Blindness, or FFB, and the
Alpha-1 Foundation. Both organizations are well known for their advocacy of patients’ interests in obtaining diagnosis, developing
treatments and providing for reimbursement. Both actively support research into treatment, and we have been awarded three research
grants totaling $1.6 million from the FFB and one grant of $0.3 million from the Alpha-1 Foundation. More importantly, both
organizations have been instrumental in assisting us in forming ties with disease experts, recruiting patients into clinical trials and
helping us to understand the needs, wants and concerns of patients. We also have relationships with other advocacy organizations
such as Achroma Corp, the BCM Family Foundation, MOMS For Sight, Curing Retinal Blindness Foundation, Sofia Sees Hope,
National Organization for Rare Disorders, ALD Connect, The Alpha-one Project and Global Genes.
In addition, we have formed strong relationships with key academic centers across the United States that have core
competencies in gene therapy, orphan ophthalmology and AAT deficiency. These centers conduct sponsored research, act as advisors
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and collaborate with us on grant proposals. Since our inception, we have been awarded a variety of grant funding, either independently
or with our collaborators. This funding has provided peer-reviewed scientific validation of our programs and has facilitated critical
early stage research for our lead product candidates.
Intellectual property
We strive to protect and enhance the proprietary technology, inventions, and improvements that are commercially important to
the development of our business, including seeking, maintaining and defending patent rights, whether developed internally or licensed
from third parties and seeking patent term extensions where available. We also rely on trade secrets relating to our proprietary
technology platform and on know-how, continuing technological innovation and in-licensing opportunities to develop, strengthen and
maintain our proprietary position in the field of gene therapy that may be important for the development of our business. In addition to
IP and trade secrets, we also will rely on regulatory protection afforded through orphan drug designations, data exclusivity and market
exclusivity for our products, when possible.
Our commercial success may depend in part on our ability to obtain and maintain patent and other proprietary protection for
commercially important technology, inventions and know-how related to our business; defend and enforce our patents; preserve the
confidentiality of our trade secrets; and operate without infringing the valid enforceable patents and proprietary rights of third parties.
Our ability to stop third parties from making, using, selling, offering to sell or importing our products may depend on the extent to
which we have rights under valid and enforceable patents or trade secrets that cover these activities. With respect to both licensed and
company-owned intellectual property, we cannot be sure that patents will be granted with respect to any of our pending patent
applications or with respect to any patent applications filed by us in the future, nor can we be sure that any of our existing patents or
any patents that may be granted to us in the future will be commercially useful in protecting our commercial products and methods of
manufacturing the same.
We have developed or in-licensed numerous patents and patent applications and possess substantial know-how and trade secrets
relating to the development, commercialization and manufacture of gene therapy products. Our proprietary intellectual property,
including patent and non-patent intellectual property, is generally directed to, for example, certain genes and promoters, methods of
transferring genetic material into cells, processes to manufacture our AAV-based product candidates and other proprietary
technologies and processes related to our lead product candidates.
As of August 21, 2017, our patent portfolio included approximately 63 patents and patent applications that we own and
approximately 54 patents and patent applications that we have licensed. More specifically, we own seven U.S. patents, seven pending
U.S. applications, 24 foreign patents and 25 foreign patent applications. We have licensed six U.S. patents, two pending U.S.
applications, 35 foreign patents and 11 pending foreign patent applications. Of the patents and patent applications that we own or
license, 77 cover methods to manufacture AAV vectors, the longest lived and most significant of which is expected to expire in 2025.
Two of the patents and 18 of the patent applications that we own are directed to small cone promoters and uses thereof. A patent
issuing from this group could have an expiration date in 2034.
Our objective is to continue to expand our portfolio of patents and patent applications in order to protect our gene therapy
product candidates and AAV manufacturing process. Our owned and licensed patent portfolio includes patents and patent applications
directed to our XLRS, ACHM, and XLRP programs, as well as our foundational AAV production platform. See also “—License
agreements.
In addition to the above, we have established expertise and development capabilities focused in the areas of preclinical research
and development, manufacturing and manufacturing process scale-up, quality control, quality assurance, regulatory affairs and clinical
trial design and implementation. We believe that our focus and expertise will help us develop products based on our proprietary
intellectual property and to expand our intellectual property portfolio.
The term of individual patents depends upon the legal term of the patents in the countries in which they are obtained. In most
countries in which we file, the patent term is 20 years from the date of filing the non-provisional 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 United
States Patent and Trademark Office in granting a patent, or may be shortened if a patent is terminally disclaimed over an earlier-filed
patent. The issued patents that are material to our business are expected to expire on various dates from 2018 to 2029.
The term of a patent that covers an FDA-approved drug may also be eligible for patent term extension, which permits patent
term restoration of a U.S. patent as compensation for the patent term lost during the FDA regulatory review process. The Hatch-
Waxman Act permits a patent term extension of up to five years beyond the expiration of the patent. The length of the patent term
extension is related to the length of time the drug is under regulatory review. A patent term extension cannot extend the remaining
term of a patent beyond a total of 14 years from the date of product approval and only one patent per approved drug may be extended.
Moreover, a patent can only be extended once, and thus, if a single patent is applicable to multiple products, it can only be extended
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based on one product. Similar provisions are available in Europe and other foreign jurisdictions to extend the term of a patent that
covers an approved drug. When possible, depending upon the length of clinical trials and other factors involved in the filing of a BLA,
we expect to apply for patent term extensions for patents covering our product candidates and their methods of use.
We may rely, in some circumstances, on trade secrets to protect our technology. However, trade secrets can be difficult to
protect. We seek to protect our proprietary technology and processes, in part, by entering into confidentiality agreements with our
employees, consultants, scientific advisors and contractors. 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 individuals, organizations and systems, agreements or security measures may 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. To the extent that our consultants, contractors or collaborators use intellectual property
owned by others in their work for us, disputes may arise as to the rights in related or resulting know-how and inventions.
License agreements
We have rights to use and exploit multiple technologies disclosed in issued and pending patents under licenses from other
entities. We consider the commercial terms of these licenses, which provide for modest milestone and royalty payments, and their
provisions regarding diligence, insurance, indemnification and other similar matters, to be reasonable and customary for our industry.
Information about our principal licenses is set forth below.
The University of Florida
We currently have three agreements with the University of Florida Research Foundation, or UFRF, an affiliate of UF, of which
the principal licenses are as follows:
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A joint license from UFRF and Johns Hopkins University, or JHU, signed in October 2003 relates to a particular HSV
construct and various compositions thereof. We have an exclusive license in all fields of use.
Under the terms of this license, we made cash and stock-based up-front payments to UFRF and JHU and are required to
make payments ranging from the mid-five figures to the low-six figures based upon development, regulatory and
commercial milestones for any products covered by the in-licensed intellectual property. Assuming that we meet each of
the specified development, regulatory and commercial milestones not more than once for each product, which we expect
will be the case, the maximum aggregate milestone payments payable under this license with respect to any individual
product that we commercialize will be $0.5 million. We will also be required to pay a royalty on net sales of products
covered by the in-licensed intellectual property in the low-single digits. We have the right to sublicense our rights under
this agreement, and we will be required to pay a percentage of such license income in the low-double digits. We are
required to make annual maintenance payments in the low four figures under this license, which payments are creditable
against royalty payments on a year-by-year basis.
This license will terminate upon the earlier to occur of the expiration of all of the patents subject to the license and the
date on which royalty payments, once commenced, cease for more than three calendar quarters. Additionally, UFRF and
JHU may terminate this license upon certain breaches by us of the terms of the license and we may terminate the license at
any time by submitting written notice to UFRF.
The longest-lived patent covered by this license is expected to expire in 2022.
Two licenses from UFRF, signed in September and November 2012, respectively, relate to the use of engineered AAV
capsids. We have an exclusive license to the patents covered by the November 2012 license in the fields of ACHM, XLRS
and XLRP and a semi-exclusive license in all other fields of orphan ophthalmology. We have a non-exclusive license in
all fields of use with respect to the patents covered by the September 2012 license. Currently these patents are most useful
for ACHM, XLRS and XLRP but could be important for treating a wide variety of diseases as the mutant capsids have
been shown to be able to enter cells more effectively than standard AAV capsids.
Under the terms of these licenses, we made cash up-front payments to UFRF and are required to make payments ranging
from the mid-five figures to the low-six figures based upon development, regulatory and commercial milestones for any
products covered by the in-licensed intellectual property. Assuming that we meet each of the specified development,
regulatory and commercial milestones not more than once for each product, which we expect will be the case, the
maximum aggregate milestone payments payable under these licenses with respect to any individual product that we
commercialize will be $0.6 million. We will also be required to pay a royalty on net sales of products covered by the in-
licensed intellectual property in the low-single digits. We have the right to sublicense our rights under these agreements,
and we will be required to pay a percentage of such license income in the mid-single digits. We are required to make
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annual maintenance payments in the mid four figures under these licenses, which payments are creditable against royalty
payments on a year-by-year basis.
These licenses will continue until the expiration of all of the patents subject to the licenses, provided or, if later, a date
specified in the license. Additionally, UFRF may terminate this license upon certain breaches by us of the terms of the
licenses and we may terminate the licenses at any time by submitting written notice to UFRF.
The longest-lived patent covered by these licenses is expected to expire in 2029. There are also patent applications
pending under these licenses.
The University of Alabama at Birmingham
A license agreement from the UAB Research Foundation affiliated with The University of Alabama at Birmingham signed in
2006, relates to one U.S. patent with claims covering the use of HSV helpers to produce AAV vectors. The patent is expected to
expire in 2025. Effective in July 2015, we modified the license from co-exclusive to exclusive.
Under the terms of this license, we made a cash up-front payment to the UAB Research Foundation, and we will be required to
make payments ranging from the mid-five figures to the low-six figures based upon development and regulatory milestones for any
products covered by the in-licensed intellectual property. Assuming that we meet each of these development and regulatory milestones
not more than once for each product, which we expect will be the case, the maximum aggregate milestone payments payable under
this license with respect to any individual product that we commercialize will be $0.5 million. We will also be required to pay a
royalty on net sales of products covered by the in-licensed intellectual property in the low-single digits. We have the right to
sublicense our rights under this agreement, and we will be required to pay a percentage of such license income in the mid-single digits.
We are required to make annual maintenance payments in the mid-four figures under this license, which payments are creditable
against royalty payments on a year-by-year basis.
This license will terminate upon the expiration of all of the patents subject to the license. Additionally, the UAB Research
Foundation may terminate this license upon certain breaches by us of the terms of the license and we may terminate the license at any
time by submitting written notice to the UAB Research Foundation.
Collaborations with 4DMT and Synpromics
We continue to collaborate with 4DMT and Synpromics in order to develop optimized capsids to target specific cell populations
and novel synthetic promoters, respectively.
Competition
The biotechnology and pharmaceutical industries are characterized by intense and rapidly changing competition to develop new
technologies and proprietary products, and any product candidates that we successfully develop and commercialize will have to
compete with existing therapies and new therapies that may become available in the future. While we believe that our proprietary
technology estate and scientific expertise in the gene therapy field provide us with competitive advantages, we face potential
competition from many different sources, including larger and better-funded pharmaceutical, specialty pharmaceutical and
biotechnology companies, as well as from academic institutions and governmental agencies and public and private research
institutions that may develop potentially competitive products or technologies.
Currently there are no approved products for any of our lead orphan ophthalmology indications of XLRS, ACHM and XLRP,
although at least one company is developing a candidate gene therapy treatment intended to treat each indication. We believe the key
competitive factors that will affect the success of our product candidates, if approved, are likely to be their efficacy, safety,
convenience of administration and delivery, price, the level of generic competition and the availability of reimbursement from
government and other third-party payors.
Government regulation
Biological products, including gene therapy products, are subject to regulation under the Federal Food, Drug, and Cosmetic Act,
or FD&C Act, and the Public Health Service Act, or PHS Act, and other federal, state, local and foreign statutes and regulations. Both
the FD&C Act and the PHS Act and their corresponding regulations govern, among other things, the testing, manufacturing, safety,
efficacy, labeling, packaging, storage, record keeping, distribution, reporting, advertising and other promotional practices involving
biological products. Before clinical testing of biological products may begin, we must submit an IND which must go into effect, and
each clinical trial protocol for a gene therapy product candidate is reviewed by the FDA and, in some instances, the NIH, through its
Recombinant DNA Advisory Committee, or RAC. FDA approval of a BLA also must be obtained before marketing of biological
products. The process of obtaining regulatory approvals and the subsequent compliance with appropriate federal, state, local and
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foreign statutes and regulations require the expenditure of substantial time and financial resources and we may not be able to obtain
the required regulatory approvals.
Within the FDA, the Center for Biologics Evaluation and Research, or CBER, regulates gene therapy products. CBER works
closely with the NIH and its RAC, which 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 and the
NIH have published guidance documents with respect to the development and submission of gene therapy protocols. The FDA also
has published guidance documents related to, among other things, gene therapy products in general, their preclinical assessment,
observing subjects involved in gene therapy studies for delayed adverse events, potency testing, and chemistry, manufacturing and
control information in gene therapy INDs.
Ethical, social and legal concerns about gene therapy, genetic testing and genetic research have led to the enactment of
legislation such as the Genetic Information Nondiscrimination Act of 2008 and could result in additional regulations restricting or
prohibiting the processes we may use. Federal and state agencies, congressional committees and foreign governments have expressed
interest in further regulating biotechnology. More restrictive regulations or claims that our products 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.
Recent developments in regulation of gene therapy
In 2017, the FDA approved a gene therapy product called Kymriah. Although this is the only gene therapy product that has
been approved in the United States, the FDA has provided guidance for the development of gene therapy products. For example, the
FDA has established the Office of Cellular, Tissue and Gene Therapies, or OCTGT, within CBER, to consolidate the review of gene
therapy and related products, and the Cellular, Tissue and Gene Therapies Advisory Committee, or CTGTAC, to advise CBER on its
reviews. In addition, the FDA has issued a growing body of clinical guidelines, chemistry, manufacturing and control, or CMC,
guidelines and other guidelines, all of which are intended to facilitate industry’s development of gene therapy products. As a result of
those guidelines and the increasing number of gene therapy products in clinical development, we believe that a number of gene
therapy products will be submitted to the FDA for approval over the next several years.
In Europe, two gene therapy products have been approved. In 2012, the EMA approved a gene therapy product called Glybera,
which is the first gene therapy product approved by regulatory authorities anywhere in the Western world. Most recently, Strimvelis
became the second gene therapy product approved by the EMA.
United States biological products development process
The process required by the FDA before a biological product candidate may be marketed in the United States generally involves
the following:
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completion of nonclinical laboratory tests and animal studies according to good laboratory practices, or GLP,
requirements and applicable requirements for the humane use of laboratory animals or other applicable regulations;
submission to the FDA of an IND, which must become effective before human clinical trials may begin;
performance of adequate and well-controlled human clinical trials according to good clinical practice, or GCP, standards
and IND and human subject protection regulations, and requirements to ensure the privacy and confidentiality of human
research subjects and their health information, to establish the safety and efficacy of the proposed biological product
candidate for its intended use;
submission to the FDA of a Biologics License Application, or BLA, for marketing approval that includes substantial
evidence of safety, purity, and potency from results of nonclinical testing and clinical trials;
satisfactory completion of an FDA inspection of the manufacturing facility or facilities where the biological product
candidate is produced to assess compliance with GMP requirements, to assure that the facilities, methods and controls are
adequate to preserve the biological product candidate’s identity, strength, quality and purity;
potential FDA audit of the nonclinical and clinical trial sites that generated the data in support of the BLA; and
FDA review and approval, or licensure, of the BLA prior to any commercial marketing or sale of the product candidate in
the United States.
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Before testing any biological product candidate, including a gene therapy product candidate, in humans, the product candidate
enters the preclinical testing stage. Preclinical tests, also referred to as nonclinical studies, include laboratory evaluations of product
chemistry, toxicity and formulation, as well as animal studies to assess the potential safety and activity of the product candidate. The
conduct of the preclinical tests must comply with federal regulations and requirements including GLP requirements.
When a gene therapy trial is conducted at, or sponsored by, institutions receiving NIH funding for recombinant DNA research,
along with the submission of an IND to the FDA, the protocol and related documentation is submitted to and the trial is registered with
the NIH Office of Science Policy, or OSP, pursuant to the NIH Guidelines for Research Involving Recombinant or Synthetic Nucleic
Acid 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. The oversight bodies at the initial clinical site(s) (Institutional Review Board (IRB) and
Institutional Biosafety Committee (IBC)) are responsible for recommending whether or not the clinical study requires public review
by the RAC, a federal advisory committee, which discusses protocols that raise novel or particularly important scientific, safety or
ethical considerations. This recommendation is included with the protocol and related documentation submitted to the OSP as part of
the trial registration process. The OSP may agree or disagree with that recommendation. Should the oversight bodies recommend
public RAC review and the OSP concurs, the protocol will then be reviewed at one of the NIH RAC’s quarterly meetings for which
information must be submitted at least eight weeks in advance. RAC proceedings and reports are posted to the OBA web site 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. Some preclinical testing
may continue even after the IND is submitted. The IND automatically becomes effective 30 days after receipt by the FDA, although
IND sponsors generally wait until the FDA affirmatively provides notice that the agency has no issues with the IND. If the FDA
places the clinical trial on a clinical hold within that 30-day time period. 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, and
the trial oversight bodies and the OSP decide that full public review of the protocol is warranted, initiation of the protocol will be
delayed until after completion of the RAC review process. The FDA may also impose clinical holds on a biological 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. Accordingly, we cannot be sure that
submission of an IND will result in the FDA allowing clinical trials to begin, or that, once begun, issues will not arise that suspend or
terminate such trials.
Clinical trials involve the administration of the biological product candidate to healthy volunteers or patients under the
supervision of qualified investigators, generally physicians not employed by or under the trial sponsor’s control. Clinical trials are
conducted under 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 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 GCP standards, human subject protection
requirements, and FDA’s investigational new drug requirements, including the requirement that all research subjects provide informed
consent. Further, each clinical trial must be reviewed and approved by an independent institutional review board, or 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 and must monitor the clinical trial until completed. Clinical trials also
must be reviewed by an institutional biosafety committee, or IBC, a local institutional committee that reviews and oversees basic and
clinical research conducted at that institution. The IBC assesses the safety of the research and identifies any potential risk to public
health or the environment.
Human clinical trials are typically conducted in three sequential phases that may overlap, be combined, or be bifurcated into two
parts:
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Phase 1. The biological product candidate is initially introduced into healthy human subjects and tested for safety. 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.
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Phase 2. The biological 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 candidate for specific targeted diseases and to
determine dosage tolerance, optimal dosage and dosing schedule.
Phase 3. Clinical trials are undertaken to further evaluate dosage, clinical efficacy, potency, and safety in an expanded
patient population at geographically dispersed clinical trial sites. These clinical trials are intended to establish the overall
risk/benefit ratio of the product candidate and provide an adequate basis for product approval and labeling.
Post-approval clinical trials, sometimes referred to as Phase 4 clinical trials, may be required as a condition of approval or may
be recommended after initial marketing approval if required. 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. Depending on the type of
product and mechanism of action, the FDA may recommend 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, of trial subjects.
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 suspected adverse reactions, any findings from other trials, tests in laboratory animals 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. Phase 1, Phase
2 and Phase 3 clinical trials may not be completed successfully within any specified period, if at all. The FDA or the sponsor, or its
data safety monitoring board 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 biological product
candidate has been associated with unexpected serious harm to patients.
Human gene therapy products are a new category of therapeutics. Although the FDA recently approved Kymriah, the first gene
therapy product in the United States, gene therapy remains a relatively new and expanding area of novel therapeutic interventions.
Consequently, there can be no assurance as to the length of the trial period, the number of patients the FDA will require to be enrolled
in the trials in order to establish the safety, efficacy, purity and potency of human gene therapy products, or that the data generated in
these trials will be acceptable to the FDA to support marketing approval. The NIH and the FDA have a publicly accessible database,
the Genetic Modification Clinical Research Information System, which includes information on gene transfer trials and serves as an
electronic tool to facilitate the reporting and analysis of adverse events on these trials. Over the last several years the FDA has issued
helpful guidance on development of gene therapy products and shown a willingness to work closely with developers, especially with
those working in orphan disease areas.
Concurrent with clinical trials, companies usually complete additional animal studies and must also develop additional
information about the physical characteristics of the biological product candidate as well as finalize a process for manufacturing the
product candidate in commercial quantities in accordance with GMP requirements. To help reduce the risk of the introduction of
adventitious agents with the use of biological 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 things, the sponsor must develop methods for testing the identity, strength, quality, potency
and purity of the final biological product. Additionally, appropriate packaging must be selected and tested and stability studies must be
conducted to demonstrate that the biological product candidate does not undergo unacceptable deterioration over its shelf life.
United States review and approval processes
After the completion of clinical trials of a biological product candidate, FDA approval of a BLA must be obtained before
commercial marketing of the biological product candidate. The BLA must include results of product development, laboratory and
animal studies, human trials, information on the manufacture and composition of the product candidate, proposed labeling and other
relevant information. In addition, under the Pediatric Research Equity Act, or PREA, a BLA or supplement to a BLA must contain
data to assess the safety and effectiveness of the biological product candidate for the claimed indications in all relevant pediatric
subpopulations and to support dosing and administration for each pediatric subpopulation for which the product candidate is safe and
effective. The FDA may grant deferrals for submission of data or full or partial waivers. Unless otherwise required by regulation,
PREA does not apply to any biological product candidate for an indication for which orphan designation has been granted. The testing
and approval processes require substantial time and effort and there can be no assurance that the FDA will accept the BLA for filing
and, even if filed, that any approval will be granted on a timely basis, if at all.
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Under the Prescription Drug User Fee Act, or PDUFA, as amended, each BLA must be accompanied by a user fee. The FDA
adjusts the PDUFA user fees on an annual basis. According to the FDA’s fee schedule for fiscal year 2017, which becomes effective
October 1, 2016, the user fee for an application requiring clinical data, such as a BLA, is $2,038,100. PDUFA also imposes an annual
product fee for biologics ($97,750) and an annual establishment fee ($512,200) on facilities used to manufacture prescription
biologics. 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.
Within 60 days following submission of the application, the FDA reviews a BLA submitted 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 candidate is safe and potent, or effective, for its intended use, and has an acceptable purity
profile, and whether the product candidate is being manufactured in accordance with GMP regulations to assure and preserve the
product candidate’s identity, safety, strength, quality, potency and purity. The FDA may refer applications for novel biological
products or biological 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 biological product approval process, the FDA also will determine
whether a Risk Evaluation and Mitigation Strategy, or REMS, is necessary to assure the safe use of the biological product candidate.
A REMS may be imposed to ensure safe use of the drug, and could include medication guides, physician communication plans, or
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 GMP
requirements and adequate to assure consistent production of the product candidate within required specifications. Additionally,
before approving a BLA, the FDA may inspect one or more clinical sites to assure that the clinical trials were conducted in
compliance with IND trial requirements and GCP requirements. To assure GMP and GCP compliance, an applicant must incur
significant expenditure of time, money and effort in the areas of training, record keeping, 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 we interpret the same data. 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 candidate 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 candidate. 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 4 clinical trials, designed
to further assess a biological product’s safety and effectiveness, and testing and surveillance programs to monitor the safety of
approved products that have been commercialized.
One of the performance goals agreed to by the FDA under the PDUFA is to review 90% of standard BLAs in 10 months and
90% of 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.
Orphan drug designation
Under the Orphan Drug Act, the FDA may grant orphan designation to a drug or biological product candidate intended to treat a
rare disease or condition, which is defined as a disease or condition that affects fewer than 200,000 individuals in the United States, or
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more than 200,000 individuals in the United States if there is no reasonable expectation that the cost of developing and making a drug
or biological product available in the United States for this type of disease or condition will be recovered from sales of the product
candidate. Orphan product designation must be requested before submitting an NDA or 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 candidate that has orphan designation subsequently receives the first FDA approval for the disease or condition for
which it has such designation, the product is entitled to orphan product exclusivity, which means that the FDA may not approve any
other applications to market the same drug or biological product for the same indication for seven years; however, the FDA has not yet
established what characteristics of a gene therapy product are relevant to determining whether two gene therapy products would be
considered the same for purposes of orphan drug market exclusivity. The FDA may approve a second drug or biological product
during an exclusivity period in limited circumstances, such as a showing of clinical superiority to the product with orphan exclusivity.
Competitors, however, may receive approval of different products for the indication for which the orphan product has exclusivity or
obtain approval for the same product but for a different indication for which the orphan product does not have exclusivity. Orphan
product exclusivity also could block the approval of one of our products for seven years if a competitor obtains approval of the same
biological product as defined by the FDA or if our product candidate is determined to be contained within the competitor’s product for
the same indication or disease. If a drug or biological product designated as an orphan product receives marketing approval for an
indication broader than what is designated, it may not be entitled to orphan product exclusivity. Orphan drug status in the European
Union has similar, but not identical, benefits.
Expedited development and review programs
The FDA has a Fast Track program that is intended to expedite or facilitate the process for reviewing new drugs and biological
products that meet certain criteria. Specifically, new drugs and biological products are eligible for Fast Track designation if they are
intended to treat a serious or life-threatening disease or condition and demonstrate the potential to address unmet medical needs for the
disease or condition. Fast Track designation applies to the combination of the product candidate and the specific indication for which
it is being studied. The sponsor of a new drug or biologic may request the FDA to designate the drug or biologic as a Fast Track
product candidate at any time during the clinical development of the product candidate. Unique to a Fast Track product candidate, the
FDA may consider for review sections of the marketing application on a rolling basis before the complete application is submitted, if
the sponsor provides a schedule for the submission of the sections of the application, the FDA agrees to accept sections of the
application and determines that the schedule is acceptable, and the sponsor pays any required user fees upon submission of the first
section of the application.
Any product candidate 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 priority review and accelerated approval. Any product
candidate is eligible for priority review if it has the potential to provide safe and effective therapy where no satisfactory alternative
therapy exists or a significant improvement in the treatment, diagnosis or prevention of a disease compared to marketed products. The
FDA will attempt to direct additional resources to the evaluation of an application for a new drug or biological product candidate
designated for priority review in an effort to facilitate the review, and aims to review such applications within six months as opposed
to ten months for standard review. Additionally, a product candidate may be eligible for accelerated approval. Drug or biological
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, which means that they 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, or 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 biological
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, which could adversely impact
the timing of the commercial launch of the product.
Lastly, under the provisions of the new Food and Drug Administration Safety and Innovation Act, or FDASIA, enacted in 2012,
a sponsor can request designation of a product candidate as a “breakthrough therapy.” A breakthrough therapy is defined as a drug that
is intended, alone or in combination with one or more other drugs, to treat a serious or life-threatening disease or condition, and
preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more
clinically significant endpoints, such as substantial treatment effects observed early in clinical development. Drugs designated as
breakthrough therapies are also eligible for accelerated approval and receive the same benefits as drugs with Fast Track designation.
The FDA must take certain actions, such as holding timely meetings and providing advice, intended to expedite the development and
review of an application for approval of a breakthrough therapy.
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Fast Track designation, priority review, accelerated approval and breakthrough therapy designation do not change the standards
for approval but may expedite the development or approval process.
Post-approval requirements
Maintaining compliance with applicable federal, state, and local statutes and regulations requires the expenditure of substantial
time and financial resources. Rigorous and extensive FDA regulation of biological products continues after approval, particularly with
respect to GMP requirements. We will rely, and expect to continue to rely, on third parties for the production of clinical and
commercial quantities of any products that we may commercialize. Manufacturers of our products are required to comply with
applicable requirements in the GMP regulations, including quality control and quality assurance and maintenance of records and
documentation. Other post-approval requirements applicable to biological products include reporting of GMP deviations that may
affect the identity, potency, purity and overall safety of a distributed product, record-keeping requirements, reporting of adverse
effects, reporting updated safety and efficacy information, and complying with electronic record and signature requirements. After a
BLA is approved, the product may also be subject to official lot release. In this case, as part of the manufacturing process, the
manufacturer is required to perform certain tests on each lot of the product before it is released for distribution. If the product is
subject to official release by the FDA, the manufacturer submits samples of each lot of product to the FDA together with a release
protocol showing a summary of the history of manufacture of the lot and the results of all of the manufacturer’s tests performed on the
lot. The FDA also may perform certain confirmatory tests on lots of some products, such as viral vaccines, before releasing the lots for
distribution by the manufacturer. In addition, the FDA conducts laboratory research related to the regulatory standards on the safety,
purity, potency, and effectiveness of biological products.
We also must comply with the FDA’s advertising and promotion requirements, such as the prohibition of preapproval
promotion, requirements related to direct-to-consumer advertising, the prohibition on promoting products for uses or in patient
populations that are not described in the product’s approved labeling (known as “off-label use”), limitations on industry-sponsored
scientific and educational activities, and promotional activities involving the internet. Discovery of previously unknown problems or
the failure to comply with the applicable regulatory requirements may result in restrictions on the marketing of a product or
withdrawal of the product from the market as well as possible civil or criminal sanctions. Failure to comply with the applicable U.S.
requirements at any time during the product development process, approval process or after approval, may subject an applicant or
manufacturer to administrative or judicial civil or criminal sanctions and adverse publicity. FDA sanctions could include refusal to
approve pending applications, withdrawal of an approval, clinical hold, warning or untitled letters, product recalls, product seizures,
total or partial suspension of production or distribution, injunctions, fines, refusals of government contracts, mandated corrective
advertising or communications with doctors, debarment, restitution, disgorgement of profits, or civil or criminal penalties. Any agency
or judicial enforcement action could have a material adverse effect on us.
Biological product manufacturers and other entities involved in the manufacture and distribution of approved biological
products are required to register their establishments with the FDA and certain state agencies, and are subject to periodic unannounced
inspections by the FDA and certain state agencies for compliance with GMPs and other laws. Accordingly, manufacturers must
continue to expend time, money, and effort in the area of production and quality control to maintain GMP compliance. Discovery of
problems with a product after approval may result in restrictions on a product, manufacturer, or holder of an approved BLA, including
withdrawal of the product from the market. In addition, changes to the manufacturing process or facility generally require prior FDA
approval before being implemented and other types of changes to the approved product, such as adding new indications and additional
labeling claims, are also subject to further FDA review and approval.
United States patent term restoration and marketing exclusivity
Depending upon the timing, duration and specifics of product development and the FDA review of a BLA, some of our U.S.
patents may be eligible for limited patent term extension under the Drug Price Competition and Patent Term Restoration Act of 1984,
commonly referred to as the Hatch-Waxman Amendments. The Hatch-Waxman Amendments permit a patent restoration term of up to
five years as compensation for patent term lost during product development and the 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 is generally 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 biological
product is eligible for the extension and the application for the extension must be submitted prior to the expiration of the patent. The
United States Patent and Trademark Office, in consultation with the FDA, reviews and approves the application for any patent term
extension or restoration. In the future, we may apply for restoration of patent term for one or more of our currently owned or licensed
patents to add patent life beyond its current expiration date, depending on the expected length of the clinical trials and other factors
involved in the filing of the relevant BLA.
A biological product can obtain pediatric market exclusivity in the United States. Pediatric exclusivity, if granted, adds six
months to existing exclusivity periods and patent terms. This six-month exclusivity, which runs from the end of other exclusivity
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protection or patent term, may be granted based on the voluntary completion of a pediatric study in accordance with an FDA-issued
“Written Request” for such a study.
The Patient Protection and Affordable Care Act, as amended by the Health Care and Education Reconciliation Act, or
Affordable Care Act, signed into law on March 23, 2010, includes a subtitle called the Biologics Price Competition and Innovation
Act of 2009, which created an abbreviated approval pathway for biological products shown to be similar to, or interchangeable with,
an FDA-licensed reference biological product. This amendment to the PHS Act attempts to minimize duplicative testing.
Biosimilarity, which requires that there be no clinically meaningful differences between the biological product and the reference
product in terms of safety, purity, and potency, can be shown through analytical studies, animal studies, and a clinical trial or trials.
Interchangeability requires that a product is biosimilar to the reference product and the product must demonstrate that it can be
expected to produce the same clinical results as the reference product and, for products administered multiple times, 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. However, complexities associated with the larger, and often
more complex, structure of biological products, as well as the process by which such products are manufactured, pose significant
hurdles to implementation that are still being worked out by the FDA.
A reference biologic is granted twelve years of exclusivity from the time of first licensure of the reference product. On April 10,
2013, President Obama released his proposed budget for fiscal year 2014 and proposed to cut this twelve-year period of exclusivity
down to seven years. He also proposed to prohibit additional periods of exclusivity for brand biologics due to minor changes in
product formulations, a practice often referred to as “evergreening.” The first biologic product submitted under the biosimilar
abbreviated approval pathway that is determined to be interchangeable with the reference product has exclusivity against other
biologics submitting under the abbreviated approval pathway for the lesser of (i) one year after the first commercial marketing, (ii) 18
months after approval if there is no legal challenge, (iii) 18 months after the resolution in the applicant’s favor of a lawsuit challenging
the biologics’ patents if an application has been submitted, or (iv) 42 months after the application has been approved if a lawsuit is
ongoing within the 42-month period.
Pharmaceutical Coverage, Pricing and Reimbursement
Sales of our products, when and if approved for marketing, will depend, in part, on the extent to which our products will be
covered by third-party payors, such as federal, state, and foreign government health care programs, commercial insurance and
managed healthcare organizations. These third-party payors are increasingly reducing reimbursements for medical products, drugs and
services. In addition, the U.S. government, state legislatures and foreign governments have continued implementing cost containment
programs, including price controls, restrictions on coverage and reimbursement and requirements for substitution of generic products.
Adoption of price controls and cost containment measures, and adoption of more restrictive policies in jurisdictions with existing
controls and measures, could further limit our net revenue and results. Decreases in third-party reimbursement for our product
candidates or a decision by a third-party payor not to cover our product candidates could reduce physician usage of our products once
approved and have a material adverse effect on our sales, results of operations and financial condition. Given the potential for long
term durable therapeutic benefit from the single administration of a gene therapy product, the question of appropriate pricing and
method of payment, including annuity payments and “pay for performance” schemes, is currently an active discussion and, depending
on outcome, could affect the use of our products and our financial performance.
Other Healthcare Laws
Although we currently do not have any products on the market, we may be subject to additional healthcare regulation and
enforcement by the federal government and by authorities in the states and foreign jurisdictions in which we conduct our business.
Such laws include, without limitation, state and federal anti-kickback, fraud and abuse, false claims, privacy and security and
physician sunshine laws and regulations, many of which may become more applicable to us if our product candidates are approved
and we begin commercialization. If our operations are found to be in violation of any of such laws or any other governmental
regulations that apply to us, we may be subject to penalties, including, without limitation, civil and criminal penalties, damages, fines,
the curtailment or restructuring of our operations, exclusion from participation in federal and state healthcare programs and
imprisonment, any of which could adversely affect our ability to operate our business and our financial results.
Employees
As of June 30, 2017, we had 72 full-time employees, 46 of whom have Ph.D., M.D. or other post-graduate degrees. Of these
full-time employees, 53 are engaged in research and development activities and 19 are engaged in finance, human resources, facilities
and general management.
All of our personnel are co-employees of AGTC and a professional human resource service organization, TriNet HR
Corporation, or TriNet. Under our agreement with TriNet, TriNet is a co-employer of our personnel, and is responsible for
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administering all payroll functions, including tax withholding, and providing health insurance and other benefits for these individuals.
We reimburse TriNet for these costs and pay TriNet an administrative fee for its services. We are responsible for, and control, all
aspects of the hiring, retention, compensation, management and supervision of our personnel. We consider the terms of our contract
with TriNet to be reasonable and customary and believe this arrangement provides substantial benefit to us, in the form of lower costs
for employee benefits and a reduced administrative burden on us.
We have no collective bargaining agreements with our employees and we have not experienced any work stoppages. We
consider our relations with our employees to be good.
Corporate information
We were incorporated in Florida in January 1999 and reincorporated in Delaware in October 2003. On April 1, 2014, we
completed our initial public offering of our common stock, which is traded on The NASDAQ Global Market under the symbol
“AGTC.” Our principal executive offices are located at 14193 NW 119th Terrace, Suite 10, Alachua, Florida 32615, and our telephone
number is (386) 462-2204. Our corporate website address is www.agtc.com. Information contained on or accessible through our
website is not a part of this annual report.
We use “AGTC” and the double helix logo as trademarks in the United States and other countries. As of June 30, 2016, these
trademarks have been registered in the United States, European Union and Japan.
This annual report contains references to our trademarks and to trademarks belonging to other entities. Solely for convenience,
trademarks and trade names referred to in this annual report, including logos, artwork, and other visual displays, may appear without
the or ™ symbols, but such references are not intended to indicate that we will not assert, to the fullest extent under applicable law,
our rights or the rights of the applicable licensor to these trademarks and trade names. We do not intend our use or display of other
companies’ trade names or trademarks to imply a relationship with, or endorsement or sponsorship of us by, any such companies.
Implications of being an emerging growth company
As a company with less than $1 billion in revenue during our last fiscal year, we qualify as an “emerging growth company” as
defined in the Jumpstart Our Business Startups Act of 2012, or the JOBS Act. As an emerging growth company, we may take
advantage of specified reduced disclosure and other requirements that are otherwise applicable generally to public companies. These
provisions include:
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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 about our executive compensation arrangements;
no non-binding advisory votes on executive compensation or golden parachute arrangements; and
exemption from the auditor attestation requirement in the assessment of our internal controls over financial reporting.
We may take advantage of these exemptions for up to five years from the date of our initial public offering of common stock or
such earlier time that we are no longer an emerging growth company. We would cease to be an emerging growth company if we have
more than $1 billion in annual revenue, we have more than $700 million in market value of our stock held by non-affiliates or we
issue more than $1 billion of non-convertible debt over a three-year period. We may choose to take advantage of some, but not all, of
the available exemptions.
In addition, the JOBS Act provides that an emerging growth company can take advantage of an extended transition period for
complying with new or revised accounting standards. This allows an emerging growth company to delay the adoption of certain
accounting standards until those standards would otherwise apply to private companies. We have irrevocably elected not to avail
ourselves of this exemption from new or revised accounting standards and, therefore, we will be subject to the same new or revised
accounting standards as other public companies that are not emerging growth companies.
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Item 1A.
Risk Factors
You should carefully consider the risks and uncertainties described below, together with the information included elsewhere in this
Annual Report on Form 10-K and other documents we file with the SEC. The risks and uncertainties described below are those that
we have identified as material, but are not the only risks and uncertainties facing us. Our business is also subject to general risks and
uncertainties that affect many other companies, such as overall U.S. and non-U.S. economic and industry conditions including a global
economic slowdown, geopolitical events, changes in laws or accounting rules, fluctuations in interest and exchange rates, terrorism,
international conflicts, major health concerns, natural disasters or other disruptions of expected economic and business conditions.
Additional risks and uncertainties not currently known to us or that we currently believe are immaterial also may impair our business
operations and liquidity.
Risks related to our financial condition and capital requirements
We have incurred significant losses since our inception and anticipate that we will continue to incur significant losses for the
foreseeable future.
We are a clinical-stage biotechnology company, and we have not yet generated revenues from product sales. With the exception
of the fiscal year ended June 30, 2017, in which we reported net income of $1.7 million due in part to the amortization associated with
our collaboration agreement with Biogen, we have incurred losses from operations in each year since our inception in 1999. We
reported net losses of $1.4 million and $24.3 million for each of the fiscal years ended June 30, 2016 and 2015, respectively. As of
our most recent fiscal year ended June 30, 2017, we had an accumulated deficit of $88.3 million. Our prior losses, combined with
expected future losses, have had and may continue to have an adverse effect on our stockholders’ equity and working capital.
We have devoted most of our financial resources to research and development, including our clinical and preclinical
development activities. To date, we have financed our operations primarily through the sale of equity securities and, to a lesser extent,
through research grants from third parties or milestone payments from a collaborator. The amount of our future net losses will depend,
in part, on the rate of our future expenditures and our ability to obtain funding through equity or debt financings, strategic
collaborations or additional grants. We anticipate that it will be several years, if ever, before we have a product candidate ready for
commercialization. Even if we obtain regulatory approval to market a product candidate, our future revenues will depend upon the
size of any markets in which our product candidates have received approval, and our ability to achieve sufficient market acceptance,
reimbursement from third-party payors and adequate market share for our product candidates in those markets.
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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continue our research and preclinical and clinical development of our product candidates;
expand the scope of our current clinical trials for our product candidates;
initiate additional preclinical studies, clinical trials or other studies for our product candidates;
further develop our gene therapy platform, including the process for design, delivery and manufacturing of our vectors for
our product candidates;
change or add additional manufacturers or suppliers;
seek regulatory and marketing approvals for our product candidates that successfully complete clinical trials;
establish a sales, marketing and distribution infrastructure to commercialize any products for which we may obtain
marketing approval;
seek to identify and validate additional product candidates;
acquire or in-license other product candidates and technologies;
make milestone or other payments under any in-license agreements;
maintain, protect and expand our intellectual property portfolio;
attract and retain skilled personnel;
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create additional infrastructure to support our operations as a public company and our product development and planned
future commercialization efforts; and
experience any delays or encounter issues with any of the above.
The net losses we incur may fluctuate significantly from quarter to quarter and year to year, such that a period-to-period
comparison of our results of operations may not be a good indication of our future performance. In any particular quarter or quarters,
our operating results could be below the expectations of securities analysts or investors, which could cause our stock price to decline.
Our ability to generate revenue from product sales is highly uncertain and we may never achieve or sustain profitability,
which could depress the market price of our common stock, and could cause you to lose part or all of your investment.
All of our revenue generated to date has come from research grants from third parties or license fees or milestone payments
from collaborations. Our ability to generate substantial revenue and achieve profitability depends on our ability, alone or with strategic
collaboration partners such as Biogen and Bionic Sight, to successfully complete the development of, and obtain the regulatory
approvals necessary to commercialize, our product candidates. We do not anticipate generating revenues from product sales for at
least the next several years, if ever. If any of our product candidates fail in clinical trials or do not gain regulatory approval, or if any
of our product candidates, if approved, fail to achieve market acceptance, we may never become profitable. Even if we achieve
profitability in the future, we may not be able to sustain profitability in subsequent periods. Our ability to generate future revenues
from product sales depends heavily on our success in:
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completing research and preclinical and clinical development of our product candidates;
seeking and obtaining regulatory and marketing approvals for product candidates for which we complete clinical trials;
establishing and maintaining supply and manufacturing relationships with third parties that can provide adequate (in
amount and quality) products and services to support clinical development and the market demand for our product
candidates, if approved;
launching and commercializing product candidates for which we obtain regulatory and marketing approval, either by
collaborating with a partner or, if launched independently, by establishing a sales, marketing and distribution
infrastructure;
obtaining and maintaining adequate coverage and reimbursement from third-party payors for our product candidates;
obtaining market acceptance of our product candidates and gene therapy as a viable treatment option;
addressing any competing technological and market developments;
implementing additional internal systems and infrastructure, as needed;
identifying and validating new gene therapy product candidates;
negotiating favorable terms in any collaboration, licensing or other arrangements into which we may enter;
maintaining, protecting and expanding our portfolio of intellectual property rights, including patents, trade secrets and
know-how; and
attracting, hiring and retaining qualified personnel.
Even if one or more of the product candidates that we develop is approved for commercial sale, we anticipate incurring
significant costs associated with commercializing any approved product candidate, particularly to the extent that we seek to
commercialize any product for an indication, such as wet AMD, that has a patient population significantly larger than those addressed
by our current lead product candidates. Our expenses could increase beyond expectations if we are required by the FDA, the EMA or
other regulatory agencies, domestic or foreign, to perform clinical trials and other studies in addition to those that we currently
anticipate. Even if we are able to generate revenues from the sale of any approved products, we may not become profitable and may
need to obtain additional funding to continue operations. Our failure to become and remain profitable would depress the market price
of our common stock and could impair our ability to raise capital, expand our business, diversify our product offerings or continue our
operations.
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In order to obtain regulatory approval for and commercialize our product candidates, we will need to raise additional
funding in the future, which may not be available on acceptable terms, or at all. Failure to obtain necessary capital when needed
may force us to delay, limit or terminate our product development efforts or other operations.
Other than our product candidates for the treatment of XLRS, ACHM CNGB3 and ACHM CNGA3, all of our lead programs in
orphan ophthalmology and otology are currently in preclinical development. Developing gene therapy products is expensive, and we
expect our research and development expenses to increase substantially as we advance our current product candidates in clinical trials
and as we undertake preclinical studies of new product candidates.
Our operations have consumed substantial amounts of cash since inception. As of June 30, 2017, and 2016, our cash and cash
equivalents and investments amounted to $138.4 million and $172.7 million, respectively. Our research and development expenses
were $26.2 million, $38.9 million and $16.5 million for the fiscal years ended June 30, 2017, 2016 and 2015, respectively. We believe
that our existing cash and cash equivalents at June 30, 2017 will be sufficient to enable us to advance planned preclinical studies and
clinical trials for our lead product candidates for at least the next two years. In order to complete the process of obtaining regulatory
approval for our lead product candidates and to build the sales, marketing and distribution infrastructure that we believe will be
necessary to commercialize our lead product candidates, if approved, we will require substantial additional funding. Also, our current
operating plan may change as a result of many factors currently unknown to us, and we may need to seek additional funds sooner than
planned, through public or private equity or debt financings, government or other third-party funding, marketing and distribution
arrangements and other collaborations, strategic alliances and licensing arrangements, or a combination of these approaches.
Any such fundraising efforts may divert our management from their day-to-day activities, which may adversely affect our
ability to develop and commercialize our product candidates. In addition, financing may not be available to us in the future in
sufficient amounts or on terms acceptable to us, if at all. Moreover, the terms of any financing may adversely affect the holdings or the
rights of our stockholders and the issuance of additional securities, whether equity or debt, by us, or the possibility of such issuance,
may cause the market price of our shares to decline. The sale of additional equity or convertible securities would dilute all of our
stockholders. The incurrence of indebtedness would result in increased fixed payment obligations and a portion of our operating cash
flows, if any, being dedicated to the payment of principal and interest on such indebtedness, and we may be required to agree to
certain restrictive covenants, such as limitations on our ability to incur additional debt, limitations on our ability to acquire, sell or
license intellectual property rights and other operating restrictions that could adversely impact our ability to conduct our business. We
could also be required to seek funds through arrangements with collaborative partners or otherwise at an earlier stage than otherwise
would be desirable or on terms that are less favorable than might otherwise be available, and we may be required to relinquish or
license on unfavorable terms rights to some of our technologies or product candidates or otherwise agree to terms unfavorable to us,
any of which may have a material adverse effect on our business, financial condition, results of operations and prospects and cause the
price of our common stock to decline.
If we are unable to obtain needed funding on a timely basis, we may be required to significantly curtail, delay or discontinue one
or more of our research or development programs or the commercialization of any product candidates or be unable to expand our
operations or otherwise capitalize on our business opportunities, as desired, which could materially affect our business, financial
condition, results of operations and prospects and cause the price of our common stock to decline.
Risks related to the discovery and development of our product candidates
All of our product candidates are in preclinical or clinical development. Clinical drug development is expensive, time
consuming and uncertain, and we may ultimately not be able to obtain regulatory approvals for the commercialization of some or
all of our product candidates.
The research, testing, manufacturing, labeling, approval, selling, marketing and distribution of drug products are subject to
extensive regulation by the FDA and other regulatory authorities, which regulations differ from country to country. Our product
candidates are in various stages of development and are subject to the risks of failure typical of drug development. The development
and approval process is expensive and can take many years to complete, and its outcome is inherently uncertain. We have not
submitted an application for or received marketing approval for any of our product candidates. We have limited experience in
conducting and managing the later-stage clinical trials necessary to obtain regulatory approvals, including approval by the FDA. To
receive regulatory approval, we must, among other things, demonstrate with substantial evidence from clinical trials that the product
candidate is safe, pure and effective for each indication for which approval is sought, and failure can occur in any stage of
development. Satisfaction of the approval requirements typically takes several years and the time needed to satisfy them may vary
substantially, based on the type, complexity and novelty of the pharmaceutical product. We cannot predict if or when we might
receive regulatory approvals for any of our product candidates currently under development.
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The FDA and foreign regulatory authorities also have substantial discretion in the drug approval process. The number and types
of preclinical studies and clinical trials that will be required for regulatory approval varies depending on the product candidate, the
disease or condition that the product candidate is designed to address, and the regulations applicable to any particular product
candidate. Approval policies, regulations, or the type and amount of clinical data necessary to gain approval may change during the
course of a product candidate’s clinical development and may vary among jurisdictions, and there may be varying interpretations of
data obtained from preclinical studies or clinical trials, either of which may cause delays or limitations in the approval or the decision
not to approve an application. Regulatory agencies can delay, limit or deny approval of a product candidate for many reasons,
including:
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the FDA or comparable foreign regulatory authorities may disagree with the design or implementation of our clinical
trials;
we may be unable to demonstrate to the satisfaction of the FDA or comparable foreign regulatory authorities that a
product candidate is safe and effective for its proposed indication;
the results of clinical trials may not meet the level of statistical or clinical significance required by the FDA or comparable
foreign regulatory authorities for approval;
the patients recruited for a particular clinical program may not be sufficiently broad or representative to assure safety in
the full population for which we seek approval;
the results may not confirm the positive results from earlier preclinical studies or clinical trials;
we may be unable to demonstrate that a product candidate’s clinical and other benefits outweigh its safety risks;
the FDA or comparable foreign regulatory authorities may disagree with our interpretation of data from preclinical studies
or clinical trials;
the data collected from clinical trials of our product candidates may not be sufficient to the satisfaction of FDA or
comparable foreign regulatory authorities to support the submission of a biologics license application, or BLA, or other
comparable submission in foreign jurisdictions or to obtain regulatory approval in the United States or elsewhere;
regulatory agencies might not approve or might require changes to our manufacturing processes or facilities; or
regulatory agencies may change their approval policies or adopt new regulations in a manner rendering our clinical data
insufficient for approval.
Any delay in obtaining or failure to obtain required approvals could materially adversely affect our ability to generate revenue
from the particular product candidate, which likely would result in significant harm to our financial position and adversely impact our
stock price. Furthermore, any regulatory approval to market a product may be subject to limitations on the indicated uses for which we
may market the product. These limitations may limit the size of the market for the product.
We are not permitted to market our product candidates in the United States or in other countries until we receive approval of a
BLA from the FDA or marketing approval from applicable regulatory authorities outside the United States. Obtaining approval of a
BLA can be a lengthy, expensive and uncertain process. If we fail to obtain FDA approval to market our product candidates, we will
be unable to sell our product candidates in the United States, which will significantly impair our ability to generate any revenues. In
addition, failure to comply with FDA and non-U.S. regulatory requirements may, either before or after product approval, if any,
subject our company to administrative or judicially imposed sanctions, including:
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restrictions on our ability to conduct clinical trials, including full or partial clinical holds on ongoing or planned trials;
restrictions on the products, manufacturers or manufacturing process;
warning letters;
civil and criminal penalties;
injunctions;
suspension or withdrawal of regulatory approvals;
product seizures, detentions or import bans;
voluntary or mandatory product recalls and publicity requirements;
total or partial suspension of production;
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imposition of restrictions on operations, including costly new manufacturing requirements; and
refusal to approve pending BLAs or supplements to approved BLAs.
Even if we do receive regulatory approval to market a product candidate, any such approval may be subject to limitations on the
indicated uses for which we may market the product. It is possible that none of our existing product candidates or any product
candidates we may seek to develop in the future will ever obtain the appropriate regulatory approvals necessary for us or our
collaborators to commence product sales. Any delay in obtaining, or an inability to obtain, applicable regulatory approvals would
prevent us from commercializing our product candidates, generating revenues and achieving and sustaining profitability.
Our gene therapy product candidates are based on a novel technology, which makes it difficult to predict the time and cost of
product candidate development and subsequently obtaining regulatory approval. At the moment, only one gene therapy product
has been approved in the United States and only two such products have been approved in Europe.
We have concentrated our product research and development efforts on our gene therapy platform, and our future success
depends on the successful development of this approach. There can be no assurance that any development problems we experience in
the future related to our gene therapy platform will not cause significant delays or unanticipated costs, or that such development
problems can be solved. We may also experience unanticipated problems or delays in expanding our manufacturing capacity or
transferring our manufacturing process to commercial partners, which may prevent us from completing our clinical trials or
commercializing our products on a timely or profitable basis, if at all.
In addition, the clinical trial requirements of the FDA, the EMA and other regulatory agencies 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 candidates. The regulatory approval process for novel product candidates such as ours can be
more expensive and take longer than for other, better known or extensively studied pharmaceutical or other product candidates.
Regulatory requirements governing gene and cell therapy products have changed frequently and may continue to change in the
future. For example, the FDA has established the Office of Cellular, Tissue and Gene Therapies within its Center for Biologics
Evaluation and Research, or CBER, to consolidate the review of gene therapy and related products, and the Cellular, Tissue and Gene
Therapies Advisory Committee to advise CBER on its review. Gene therapy clinical trials conducted at institutions that receive
funding for recombinant DNA research from the United States National Institutes of Health, or the NIH, are also subject to review by
the NIH Office of Biotechnology Activities’ Recombinant DNA Advisory Committee, or the RAC. Although the FDA decides
whether individual gene therapy protocols may proceed, the RAC review process 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 clinical hold even if
the RAC has provided a favorable review of the drug. Also, before a clinical trial can begin at an NIH-funded institution, that
institution’s institutional review board, or IRB, and its Institutional Biosafety Committee, or IBC, have 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 regulatory bodies to change the requirements for approval of any of our product candidates.
These regulatory review committees and advisory groups and the 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 these product candidates or lead to significant post-approval
limitations or restrictions. As we advance our product candidates, we will be required to consult with these regulatory and advisory
groups, and comply with applicable guidelines. If we fail to do so, we may be required to delay or discontinue development of our
product candidates. These additional processes may result in a review and approval process that is longer than we otherwise would
have expected for orphan ophthalmology product candidates. 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
to maintain our business.
Success in animal studies or early clinical trials may not be indicative of results obtained in later trials.
Trial designs and results from animal studies or previous clinical trials are not necessarily predictive of our future clinical trial
designs or results, and interim results of a clinical trial are not necessarily indicative of final results. Our product candidates may also
fail to show the desired safety and efficacy in clinical development despite demonstrating positive results in animal studies or having
successfully advanced through initial clinical trials. There can be no assurance that the success we achieved in the animal studies for
our lead product candidates will result in success in our clinical trials of those product candidates.
There is a high failure rate for drugs and biological products proceeding through clinical trials. A number of companies in the
pharmaceutical and biotechnology industries have suffered significant setbacks in later stage clinical trials even after achieving
promising results in earlier stage clinical trials. Data obtained from preclinical and clinical activities are subject to varying
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interpretations, which may delay, limit or prevent regulatory approval. In addition, we 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.
We may find it difficult to enroll patients in our clinical trials, which could delay or prevent clinical trials of our product
candidates.
Identifying and qualifying patients to participate in clinical trials of our product candidates is critical to our success. The timing
of our clinical trials depends on the speed at which we can recruit patients to participate in testing our product candidates as well as
completion of required follow-up periods. If patients are unwilling to participate in our gene therapy studies because of negative
publicity from adverse events in the biotechnology or gene therapy industries or for other reasons, including competitive clinical trials
for similar patient populations, the timeline for recruiting patients, conducting studies and obtaining regulatory approval of our
product candidates may be delayed. For example, trials using early versions of lentiviral vectors, which integrate with, and thereby
alter, the host cell’s DNA, have led to several well-publicized adverse events, including reported cases of leukemia. If there are delays
in accumulating the required number of clinical events in trials for our product candidates where clinical events are a primary
endpoint, there may be delays in completing the trial. These delays could result in increased costs, delays in advancing our product
candidates, delays in testing the effectiveness of our technology or termination of the clinical trials altogether.
We may not be able to identify, recruit and enroll a sufficient number of patients, or those with the required or desired
characteristics to achieve diversity in a trial, to complete our clinical trials in a timely manner. For example, enrolling eligible patients
in novel orphan drug trials can be challenging and we have encountered slower-than-expected enrollment in our phase 1/2 clinical trial
for our XLRS product candidate as a result of patients not meeting one or more study eligibility criteria. Challenges such as these in
enrolling a sufficient number of patients to conduct our clinical trials as planned, may cause us to delay, limit or terminate ongoing or
planned clinical trials, any of which would have an adverse effect on our business. We could also encounter delays if physicians
encounter unresolved ethical issues associated with enrolling patients in clinical trials of our product candidates
In particular, each of the conditions for which we plan to evaluate our product candidates are rare genetic disorders with limited
patient pools from which to draw for clinical trials. The eligibility criteria of our clinical trials will further limit the pool of available
trial participants.
Patient enrollment is affected by factors including:
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severity of the disease under investigation;
design of the clinical trial protocol;
size and nature of the patient population;
eligibility criteria for the trial in question;
perceived risks and benefits of the product candidate under trial;
proximity and availability of clinical trial sites for prospective patients;
availability of competing therapies and clinical trials;
clinicians’ and patients’ perceptions as to the potential advantages of the drug being studied in relation to other available
therapies, including any new drugs that may be approved for the indications we are investigating;
efforts to facilitate timely enrollment in clinical trials;
patient referral practices of physicians; and
our ability to monitor patients adequately during and after treatment.
We plan to seek initial marketing approval for our product candidates in the United States and the European Economic Area, or
EEA. We may not be able to initiate or continue clinical trials if we cannot enroll a sufficient number of eligible patients to participate
in the clinical trials required by the FDA, the EMA or other foreign regulatory authorities. 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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difficulty in establishing or managing relationships with contract research organizations, or CROs, and physicians;
different standards for conducting clinical trials;
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our inability to locate 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 products and treatments.
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 our product candidates, we must conduct
extensive clinical trials to demonstrate the safety and efficacy of such product candidates in humans. 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. Our clinical trials have and may continue to be delayed by the necessity to re-test study agent, the decision to use
a single surgeon to treat all patients in our ACHM CNGB3 and ACHM CNGA3 trials in the near-term and a protocol amendment that
required approval by institutional review boards at the clinical sites. 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 raising, or inability to raise, sufficient capital to fund the planned clinical trials;
inability to generate sufficient preclinical, toxicology, or other data to support the initiation of human clinical trials;
delays in reaching a consensus with regulatory agencies on trial design;
identifying, recruiting and training suitable clinical investigators;
delays in reaching agreement on acceptable terms with prospective CROs and clinical trial sites, the terms of which can be
subject to extensive negotiation and may vary significantly among different CROs and trial sites;
delays in obtaining required IRB approval at each clinical trial site;
delays in recruiting suitable patients to participate in our clinical trials;
delays due to changing standard of care for the diseases we are targeting;
adding new clinical trial sites;
imposition of a clinical hold by regulatory agencies, after review of an IND application or equivalent application or an
inspection of our clinical trial operations or trial sites;
failure by our CROs, other third parties or us to adhere to clinical trial requirements;
loss of product due to shipping delays or delays in customs in connection with delivery to foreign countries for use in
clinical trials;
failure to perform in accordance with the FDA’s good clinical practices, or GCP requirements or applicable regulatory
guidelines in other countries;
delays in the manufacture, testing, release, import or export for use of sufficient quantities of our product candidates for use
in clinical trials by our vendors, such as the vendor testing errors previously experienced in our ongoing clinical trials;
failure by us or our vendors to manufacture our product candidate in accordance with the FDA’s good manufacturing
practice, or GMP, requirements or applicable regulatory guidelines in other countries;
delays by us or our contract vendors in the testing, validation and delivery of our product candidates to the clinical trial
sites;
delays in having patients complete participation in a trial or return for post-treatment follow-up;
clinical trial sites deviating from trial protocol or clinical trial sites or patients dropping out of a trial;
occurrence of serious adverse events associated with the product candidate that are viewed to outweigh its potential
benefits;
changes in regulatory requirements and guidance that require amending or submitting new clinical protocols;
the costs of clinical trials of our product candidates may be greater than we anticipate; or
clinical trials of our product candidates may produce negative or inconclusive results, and we may decide, or regulators
may require us, to conduct additional clinical trials or abandon drug development programs.
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Further, a clinical trial may be suspended or terminated by us, our collaborators, the IRBs, in the institutions in which such trials
are being conducted, the Data Safety Monitoring Board, or DSMB, for such trial, or by the FDA or other regulatory authorities due to
a number of factors, including failure to conduct the clinical trial in accordance with regulatory requirements or our clinical protocols,
inspection of the clinical trial operations or trial site by the FDA or other regulatory authorities resulting in the imposition of a clinical
hold, unforeseen safety issues or adverse side effects, failure to demonstrate a benefit from using a product candidate, changes in
governmental regulations or administrative actions or lack of adequate funding to continue the clinical trial. In appropriate
circumstances, we may also elect to temporarily suspend an ongoing clinical trial to further study unexpected results, even if those
results would not require us to formally suspend the trial under the applicable regulatory requirements or clinical protocols. Such
temporary suspension could include further testing of trial materials and the need to review subject responses to ensure safety. If we
experience termination of, or delays in the completion of, any clinical trial of our product candidates, the commercial prospects of our
product candidates will be harmed, and our ability to generate product revenues from any of these product candidates will be delayed.
Furthermore, 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 our product candidates.
Any inability to successfully complete preclinical and clinical development could result in additional costs to us or impair our
ability to generate revenues from product sales, regulatory and commercialization milestones and royalties. In addition, if we or our
third-party collaborators make manufacturing or formulation changes to product candidates, we or they may need to conduct
additional trial to bridge the modified product candidates to earlier versions. Clinical trial delays could also 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, which could impair our ability to successfully commercialize our product candidates and may harm our business and
results of operations.
If the results of our clinical trials are inconclusive or if there are safety concerns or adverse events associated with our product
candidates, we may:
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be delayed in obtaining marketing approval for our product candidates, if at all;
obtain approval for indications or patient populations that are not as broad as intended or desired;
obtain approval with labeling that includes significant use or distribution restrictions or safety warnings;
be subject to changes with the way the product is administered;
be required to perform additional clinical trials to support approval or be subject to additional post-marketing testing
requirements;
have regulatory authorities withdraw their approval of the product or impose restrictions on its distribution in the form of
a modified risk evaluation and mitigation strategy;
be subject to the addition of labeling statements, such as warnings or contraindications;
be sued; or
experience damage to our reputation.
Any of these events could prevent us from achieving or maintaining market acceptance of our product candidates and impair our
ability to commercialize our product candidates.
Our product candidates may cause undesirable side effects or have other properties that could delay or prevent their
regulatory approval, limit the commercial profile of an approved label, or result in significant negative consequences following any
potential marketing approval.
As with many pharmaceutical and biological products, treatment with our product candidates may produce undesirable side
effects or adverse reactions or events. These adverse events may occur despite our belief that our AAV vectors have an improved
safety profile over prior such treatments.
Known adverse side effects that could occur with treatment with AAV vectors include an immunologic reaction to the capsid
protein or gene at early time points after administration. In previous clinical trials involving AAV viral vectors for gene therapy, some
subjects experienced serious adverse events, including the development of T-cell response due to immune response against the vector
capsid proteins. If our vectors demonstrate a similar effect, or other adverse events, we may be required to halt or delay further clinical
development of our product candidates. In addition, theoretical adverse side effects of AAV vectors include replication and spread of
the virus to other parts of the body and insertional oncogenesis, which is the process whereby the insertion of a functional gene near a
gene that is important in cell growth or division results in uncontrolled cell division, also known as cancer, which could potentially
enhance the risk of malignant transformation. Potential procedure-related adverse events, including inflammation, can also occur and
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have, in fact, been observed in our XLRS and ACHM CNGB3 trials. There is also the potential risk of delayed adverse events
following exposure to gene therapy products due to persistent biological activity of the genetic material or other components of
products used to carry the genetic material. If any such adverse events occur, our clinical trials could be suspended or terminated and
the FDA, the EMA or other foreign regulatory authorities could order us to cease further development of or deny approval of our
product candidates for any or all targeted indications. The product-related side effects could affect patient recruitment or the ability of
enrolled patients to complete the trial. If we elect or are required to delay, suspend or terminate any clinical trial of any of our product
candidates, the commercial prospects of such product candidates will be harmed and our ability to generate product revenues from any
of these product candidates will be delayed or eliminated. Any of these occurrences may harm our business, financial condition and
prospects significantly.
Additionally, if any of our 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 its risks, which may include, among other things, a medication
guide outlining the risks of gene therapies for distribution to patients and a communication plan to health care practitioners.
Furthermore, if we or others later identify undesirable side effects caused by our product candidate, a number of potentially significant
negative consequences could result, including:
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regulatory authorities may withdraw approvals of such product candidate;
regulatory authorities may require additional warnings on the label;
we may be required to create a medication guide outlining the risks of such side effects for distribution to patients;
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.
Any of these events could prevent or delay us from achieving or maintaining market acceptance of our product candidates and
could significantly harm our business, prospects, financial condition and results of operations.
We may be unable to obtain orphan product designation or exclusivity for some of our product candidates. If our competitors
are able to obtain orphan product exclusivity for their products that are the same as our product candidates, we may not be able to
have competing products approved by the applicable regulatory authority for a significant period of time.
Regulatory authorities in some jurisdictions, including the United States and Europe, may designate drugs for relatively small
patient populations as orphan drugs. Under the Orphan Drug Act of 1983, the FDA may designate a product candidate as an orphan
drug if it is intended to treat a rare disease or condition, which is generally defined as having a patient population of fewer than
200,000 individuals diagnosed annually in the United States, or a patient population greater than 200,000 in the United States where
there is no reasonable expectation that the cost of developing the drug will be recovered from sales in the United States. In the
European Union, the EMA’s Committee for Orphan Medicinal Products, or COMP, grants orphan drug designation to promote the
development of products that are intended for the diagnosis, prevention or treatment of a life-threatening or chronically debilitating
condition affecting not more than 5 in 10,000 persons in the European Union Community. Additionally, designation is granted for
products intended for the diagnosis, prevention or treatment of a life-threatening, seriously debilitating or serious and chronic
condition and when, without incentives, it is unlikely that sales of the drug in the European Union would be sufficient to justify the
necessary investment in developing the drug or biological product. Our product candidates for the treatment of XLRS, ACHM (in the
form caused by mutations in the CNGB3 and CNGA3 genes) have been granted orphan drug designations by the FDA, and our
product candidates for the treatment of XLRS, ACHM (in the form caused by mutations in the CNGB3 and CNGA3 genes), XLRP (in
the form caused by mutations in the RPGR gene) and have been granted orphan medicinal product designation by the European
Commission. We may request orphan drug designation for our other product candidates in the future but there can be no assurances
that the FDA will grant any of our product candidates such designation. Additionally, the designation by the FDA of any of our
product candidates as an orphan drug does not guarantee that the FDA will accelerate regulatory review of or ultimately approve that
product candidate.
Generally, if a product candidate with an orphan drug designation subsequently receives the first marketing approval for the
indication for which it has such designation, the product is entitled to a period of marketing exclusivity, which precludes the EMA or
the FDA from approving another marketing application for the same drug and indication for that time period, except in limited
circumstances. The FDA has not defined the meaning of “same drug” specifically for gene therapy products and it is possible that the
FDA could conclude that no two gene therapy products could be considered the same. The applicable period is seven years in the
United States and 10 years in Europe. The European exclusivity period can be reduced to six years if a product no longer meets the
criteria for orphan drug designation or if the product is sufficiently profitable so that market exclusivity is no longer justified. Orphan
drug exclusivity may be lost if the FDA or EMA determines that the request for designation was materially defective or if the
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manufacturer is unable to assure sufficient quantity of the product to meet the needs of patients with the rare disease or condition, or if
a gene product considered to be the same as our product candidate is superior in certain respects.
Even if we obtain orphan drug exclusivity for a product candidate, that exclusivity may not effectively protect the product
candidate from competition because different drugs can be approved for the same condition. In the United States, even after an orphan
drug is approved, the FDA can subsequently approve another drug for the same condition if the FDA concludes that the later drug is
clinically superior in that it is shown to be safer, more effective or makes a major contribution to patient care.
Even if we complete the necessary clinical trials, we cannot predict when or if we will obtain regulatory approval to
commercialize a product candidate or the approval may be for a narrower indication than we expect.
We cannot commercialize a product candidate until the appropriate regulatory authorities have reviewed and approved the
product candidate. Even if our product candidates demonstrate safety and efficacy in clinical trials, the regulatory agencies 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 agency policy during the period of product development, clinical trials and the review process. Regulatory
agencies also may approve a product candidate for fewer or more limited indications than requested, may not approve the price we
intend to charge for our product candidate, may impose significant limitations in the form of narrow indications, warnings, precautions
or contra-indications with respect to conditions of use or may grant approval subject to the performance of costly post-marketing
clinical trials. In addition, regulatory agencies 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 products will remain subject to regulatory scrutiny.
Even if we obtain regulatory approval in a jurisdiction for our product candidates, they 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
limitations on the approved indicated uses for which the product may be marketed or to the conditions of approval, or contain
requirements for potentially costly post-marketing testing, including Phase 4 clinical trials, and surveillance to monitor the safety and
efficacy of the product. For example, the holder of an approved BLA is obligated to monitor and report adverse events and any failure
of a product to meet the specifications in the BLA. FDA guidance advises that patients treated with some types of gene therapy
undergo follow-up observations for potential adverse events for as long as 15 years. The holder of an approved BLA must also submit
new or supplemental applications and obtain FDA approval for certain changes to the approved product, product labeling or
manufacturing process. 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.
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 GMP requirements and adherence to commitments made
in the BLA or foreign marketing application. If we or a regulatory agency discovers 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 or
disagrees with the promotion, marketing or labeling of that product, a regulatory agency 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.
If we fail to comply with applicable regulatory requirements following approval of any of our product candidates, a regulatory
agency may:
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issue a warning letter asserting that we are in violation of the law;
seek an injunction or impose civil or criminal penalties or monetary fines;
suspend or withdraw regulatory approval;
suspend any ongoing clinical trials;
refuse to approve a pending BLA or comparable foreign marketing application (or any supplements thereto) submitted by
us or our strategic partners;
restrict the marketing or manufacturing of the product;
seize or detain product or otherwise require the withdrawal of product from the market;
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refuse to permit the import or export of products; or
refuse to allow us to enter into supply contracts, including government contracts.
Any government investigation of alleged violations of law could require us to expend significant time and resources in response
and could generate negative publicity. The occurrence of any event or penalty described above may inhibit our ability to
commercialize our product candidates and generate revenues.
In addition, the FDA’s policies may change and additional government regulations may be enacted that could prevent, limit or
delay regulatory approval of our product candidates. We cannot predict the likelihood, nature or extent of government regulation that
may arise from future legislation or administrative action, either in the United States or abroad. If we are slow or unable to adapt to
changes in existing requirements or the adoption of new requirements or policies, or if we are not able to maintain regulatory
compliance, we may lose any marketing approval that we may have obtained and we may not achieve or sustain profitability, which
would adversely affect our business, prospects, financial condition and results of operations.
Even if we obtain and maintain approval for our 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.
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. Sales of our product candidates outside of the United States will be
subject to foreign regulatory requirements governing clinical trials and marketing approval. Even if the FDA grants marketing
approval for a product candidate, comparable regulatory authorities of foreign countries must also approve the manufacturing and
marketing of the product candidates in those countries. Approval procedures vary among jurisdictions and can involve requirements
and administrative review periods different from, and greater 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. In some cases, the price that we intend to charge for our products, if approved, is also subject to
approval. We intend to submit a marketing authorization application to the EMA for approval in the EEA, but obtaining such approval
is a lengthy and expensive process and the EMA has its own procedures for approval of product candidates. Even if a product
candidate is approved, the FDA or the EMA, as the case may be, may limit the indications for which the product may be marketed,
require extensive warnings on the product labeling or require expensive and time-consuming clinical trials or reporting as conditions
of approval. Regulatory authorities in countries outside of the United States and the EEA 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 our product candidates in certain countries.
Further, clinical trials conducted in one country may not be accepted by regulatory authorities in other countries and regulatory
approval of a product candidate in one country does not ensure approval in any other country, while a failure or delay in obtaining
regulatory approval in one country may have a negative effect on the regulatory approval process in others. Also, regulatory approval
for any of our product candidates may be withdrawn. If we fail to comply with the regulatory requirements in international markets
and/or receive applicable marketing approvals, our target market will be reduced and our ability to realize the full market potential of
our product candidates will be harmed and our business will be adversely affected.
Risks related to our reliance on third parties
We expect to rely on third parties to conduct aspects of our product manufacturing and protocol development, and these
third parties may not perform satisfactorily.
We do not expect to independently conduct all aspects of our vector production, product manufacturing, protocol development,
and monitoring and management of our ongoing and planned preclinical and clinical programs. We have expanded our internal
capabilities to include a full-scale pilot facility to facilitate continued improvement in our manufacturing process. We have completed
the design phase for a cGMP facility at our Florida headquarters to support later stage clinical development. We currently rely, and
expect to continue to rely, to a significant degree, on third parties for the production of our clinical trial materials. In such cases, we
expect to control only certain aspects of their activities.
Under certain circumstances, these third parties may be entitled to terminate their engagements with us or we may seek to
terminate our engagement with them. Because of the complexities inherent in gene therapy manufacturing, we expect that any
engagement by us of a new third-party manufacturer for our product candidates would take a substantial amount of time to establish.
Accordingly, if we need to enter into alternative arrangements, it could delay our product development activities. We are currently
negotiating with and conducting pilot work at three alternative third-party manufacturers to expand our capacity and mitigate risk.
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Our reliance on these third parties for research and development activities will reduce our control over these activities but will not
relieve us of our responsibility to ensure compliance with all required regulations and study and trial protocols. If these third parties do
not successfully carry out their contractual duties, meet expected deadlines or conduct our studies in accordance with regulatory
requirements or our stated study and trial plans and protocols, or if there are disagreements between us and these third parties, we will
not be able to complete, or may be delayed in completing, the preclinical studies and clinical trials required to support future IND
submissions and approval of our product candidates. In some such cases, 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 with respect to the
approval of our product candidates and would thereby have a material adverse effect on our business, financial condition, results of
operations and prospects.
In addition, reliance on third-party manufacturers entails risks to which we would not be subject if we manufactured the product
candidates ourselves, including:
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the inability to negotiate manufacturing agreements with third parties under commercially reasonable terms;
delays in the production of our product candidates associated with transitioning to a new third-party manufacturer;
reduced control as a result of using third-party manufacturers for all aspects of manufacturing activities;
termination or nonrenewal of manufacturing 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 or suppliers caused by conditions unrelated to our business
or operations, including the bankruptcy of the manufacturer or supplier.
Any of these events could lead to clinical trial delays or failure to obtain regulatory approval, or impact our ability to
successfully commercialize future 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.
We and our contract manufacturer are subject to significant regulatory oversight with respect to manufacturing our
products. The manufacturing facilities on which we rely may not continue to meet regulatory requirements and may have limited
capacity.
All parties involved in the preparation of therapeutics for clinical trial or commercial sale are subject to extensive regulation.
Components of a finished therapeutic product approved for commercial sale or used in late-stage clinical trials must be manufactured
in accordance with GMP requirements. These regulations govern manufacturing processes and procedures (including record keeping)
and the implementation and operation of quality systems to control and assure the quality of investigational products and products
approved for sale. Poor control of production processes can lead to the introduction of adventitious agents or other contaminants, or to
inadvertent changes in the properties or stability of our product candidates that may not be detectable in final product testing. We or
our contract manufacturers must supply all necessary documentation in support of a BLA on a timely basis and must adhere to the
FDA’s GMP requirements enforced by the FDA through its facilities inspection program. Our facilities and quality systems and the
facilities and quality systems of some or all of our third-party contractors must pass a pre-approval inspection for compliance with the
applicable regulations as a condition of regulatory approval of our product candidates. In addition, the regulatory authorities may, at
any time, audit or inspect a manufacturing facility involved with the preparation of our product candidates or the associated quality
systems for compliance with the regulations applicable to the activities being conducted. If these facilities do not pass a pre-approval
plant inspection, FDA approval of the products will not be granted.
The regulatory authorities also may, at any time following approval of a product for sale, audit our manufacturing facilities or
those of our third-party manufacturers. If any such inspection or audit identifies a failure to comply with applicable regulations or if a
violation of our product specifications or applicable regulations occurs independent of such an inspection or audit, we or the relevant
regulatory authority may require remedial measures that may be costly and/or time-consuming for us or our third-party manufacturers
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 such remedial measures imposed upon us or third parties with whom we contract
could materially harm our business.
As described above in “Business,” we have encountered delays in clinical trial material availability as a result of difficulties in
proper testing. If we or any of our third-party manufacturers or testing contractors fail to maintain regulatory compliance, the FDA can
impose regulatory sanctions including, among other things, refusal to approve a pending application for a new product candidate, or
revocation of a pre-existing approval. Such an occurrence may cause our business, financial condition and results of operations to be
materially harmed.
38
Additionally, if supply from an approved manufacturer is interrupted, there could be a significant disruption in commercial
supply of our products. While we are currently working to establish a relationship with alternative third-party manufacturers, we do
not currently have a backup manufacturer of our product candidate supply for clinical trials or commercial sale. Because of the
complexities inherent in our gene therapy manufacturing, we expect that there will be a significant period of time following our
engagement of an alternative third-party manufacturer before that manufacturer will be in a position to provide an adequate supply of
our product candidates for our clinical trials. In addition, any alternative manufacturer will also need to be qualified through a BLA
supplement which could result in further delay. The regulatory agencies may also require additional trials if a new manufacturer is
relied upon for commercial production. Switching manufacturers may involve substantial costs and is likely to result in a delay in our
desired clinical and commercial timelines.
These factors could cause the delay of clinical trials, regulatory submissions, required approvals or commercialization of our
product candidates, cause us to incur higher costs and prevent us from commercializing our products successfully. Furthermore, if our
suppliers fail to meet contractual requirements, and we are unable to secure one or more replacement suppliers capable of production
at a substantially equivalent cost, our clinical trials may be delayed or we could lose potential revenue.
We expect to rely on third parties to conduct and supervise our clinical trials, and if these third parties perform in an
unsatisfactory manner, it may harm our business.
We expect to continue to rely on academic research institutions and other CROs along with clinical trial sites to ensure our
clinical trials are conducted properly and on time. While we will have agreements governing their activities, we will have limited
influence over their actual performance and will control only certain aspects of our CROs’ activities. Nevertheless, we will be
responsible for ensuring that each of our clinical trials is conducted in accordance with the applicable protocol, legal, regulatory and
scientific standards, and our reliance on the CROs does not relieve us of our regulatory responsibilities.
We and our CROs are required to comply with the FDA’s and other regulatory authorities’ GCP, GMP and good laboratory
practice, or GLP, requirements for conducting, recording and reporting the results of our preclinical studies and clinical trials to assure
that the data and reported results are credible and accurate and that the rights, integrity and confidentiality of clinical trial participants
are protected. The FDA enforces these requirements through periodic inspections of study sponsors, principal investigators and
clinical trial sites. If we or our CROs fail to comply with applicable GCP requirements, the clinical data generated in our future
clinical trials may be deemed unreliable and the FDA may require us to perform additional clinical trials before approving any
marketing applications. Upon inspection, the FDA may determine that our clinical trials did not comply with GCP requirements,
which may render the data generated in those trials unreliable. In addition, our future clinical trials will require a sufficient number of
test subjects to evaluate the safety and effectiveness of our product candidates. Accordingly, if our CROs fail to comply with these
regulations or fail to recruit a sufficient number of patients, we may be required to repeat such clinical trials, which would delay the
regulatory approval process.
Our CROs are not our employees, and, except for remedies available to us under our agreements with such CROs, we are
therefore unable to directly monitor whether or not they devote sufficient time and resources to our clinical and nonclinical programs.
Our CROs also have relationships with other commercial entities, including our competitors, for whom they may also be conducting
clinical trials or other drug development activities that could harm our competitive position. If our CROs do not successfully carry out
their contractual duties or obligations, fail to meet expected deadlines, or if the quality or accuracy of the clinical data they obtain is
compromised due to the failure to adhere to our clinical protocols or regulatory requirements, or for any other reasons, our clinical
trials may be extended, delayed or terminated, and we may not be able to obtain regulatory approval for, or successfully
commercialize our product candidates. As a result, our financial results and the commercial prospects for our product candidates
would be harmed, our costs could increase, and our ability to generate revenues could be delayed.
Switching or adding CROs involves substantial cost and requires extensive management time and focus. In addition, there is a
natural transition period when a new CRO commences work. As a result, delays occur, which can materially impact our ability to meet
our desired clinical development timelines. Though we carefully manage our relationships with our CROs, there can be no assurance
that we will not encounter similar challenges or delays in the future or that these delays or challenges will not have a material adverse
impact on our business, prospects, financial condition and results of operations.
We also expect to rely on other third parties to store and distribute our vectors and products for any clinical trials that we may
conduct. Any performance failure on the part of our distributors could delay clinical development, regulatory review or marketing
approval of our product candidates or commercialization of our products, if approved, producing additional losses and depriving us of
potential product revenue.
Collaborations with third parties, such as our collaboration with Biogen, may be important to our business. If these
collaborations are not successful, our business could be adversely affected.
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We have a collaboration with Biogen to develop, seek regulatory approval for and commercialize gene therapy products to treat
XLRS and XLRP. The collaboration agreement also provides for discovery programs targeting three indications whereby we will
conduct discovery, research and development activities for those additional drug candidates through the stage of clinical candidate
designation, after which, Biogen may exercise an option to continue to develop, seek regulatory approval for and commercialize the
designated clinical candidate. In addition, under our manufacturing agreement with Biogen, we granted Biogen an exclusive license
to use our proprietary technology platform outside of the collaboration to make AAV vectors for up to three available genes and three
additional genes that we may approve in our discretion. An unsuccessful outcome in pending and future clinical trials for which
Biogen is responsible could be harmful to the public perception and prospects of our gene therapy platform.
Our license relationships with Biogen and any other current collaboration or collaboration we enter into in the future, may pose
a number of risks, including the following:
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collaborators have significant discretion in determining the efforts and resources that they will apply to these
collaborations;
collaborators may not perform their obligations as expected;
exclusivity rights we negotiate with our collaborators may be unenforceable in certain jurisdictions;
collaborators may not pursue development and commercialization of any product candidates that achieve regulatory
approval or may elect not to continue or renew development or commercialization programs based on clinical trial results,
changes in the collaborators’ strategic focus or available funding, or external factors, such as an acquisition, that divert
resources or create competing priorities;
collaborators may delay clinical trials, provide insufficient funding for a clinical trial program, stop a clinical trial or
abandon a product candidate, repeat or conduct new clinical trials or require a new formulation of a product candidate for
clinical testing;
collaborators may decide not to continue the development of collaboration products and could independently develop, or
develop with third parties, products that compete directly or indirectly with our products or product candidates if the
collaborators believe that competitive products are more likely to be successfully developed or can be commercialized
under terms that are more economically attractive than ours;
product candidates discovered in collaboration with us may be viewed by our collaborators as competitive with their own
product candidates or products, which may cause collaborators to cease to devote resources to the commercialization of
our product candidates;
take-over or step-in rights granted to a collaborator with respect to one or more of our product candidates, may cause us to
have limited control over future development activities and/or realize diminished economic or other benefits upon the
ultimate commercialization of that product candidate;
a collaborator with marketing, distribution and commercialization rights to one or more of our product candidates that
achieve regulatory approval may not commit sufficient resources to the marketing and distribution of any such product
candidate;
if we fail to obtain orphan product designation for a partnered product, we may realize diminished economic benefit upon
the ultimate commercialization of that product candidate;
restrictions and commitments contained in collaborations may have the effect of preventing us from independently
undertaking development and other efforts that may appear to be attractive to us;
disagreements with collaborators, including disagreements over proprietary rights, contract interpretation or the preferred
course of development of any product candidates, might cause delays or termination of the research, development or
commercialization of such product candidates, might lead to additional responsibilities for us with respect to such product
candidates, or might result in litigation or arbitration, any of which would be time-consuming and expensive;
collaborators may not properly maintain or defend our intellectual property rights or may use our proprietary information
in such a way as to invite litigation that could jeopardize or invalidate our intellectual property or proprietary information
or expose us to potential litigation;
collaborators may infringe the intellectual property rights of third parties, which may expose us to litigation and potential
liability;
collaborations may be terminated at the convenience of the collaborator or for a material breach by either party, and, if a
collaboration is terminated, we could be required to make payments to the collaborator or have our potential payments
under the collaboration reduced; and
in the event of the termination of a collaboration, we could be required to raise additional capital to pursue further
development or commercialization of the product candidates returned to us by our former collaborator.
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If our collaborations do not result in the successful development and commercialization of products or if one of our
collaborators terminates its agreement with us, we may not receive any future research funding or milestone or royalty payments under
the collaboration. If we do not receive the funding we expect under these agreements, our development of our gene therapy platform
and product candidates could be delayed and we may need additional resources to develop product candidates and gene therapy
platform. As a result of these or other factors, we may not receive the benefits that we expect from our collaborations.
In the event Biogen terminates the collaboration for a material breach by us, we will be restricted from competing with Biogen
for two years in the terminated programs with Biogen having the right, in lieu of termination, to elect to maintain the license from us
and reduce the royalties and milestones in a manner specified in the agreement.
Additionally, subject to its contractual obligations to us, if one of our collaborators is involved in a business combination, the
collaborator might deemphasize or terminate the development or commercialization of any product candidate licensed to it by us. If
one of our collaborators terminates its agreement with us, we may find it more difficult to attract new collaborators and our perception
in the business and financial communities could be adversely affected.
We may in the future determine to collaborate with other pharmaceutical and biotechnology companies for development and
potential commercialization of product candidates other than those covered by our collaboration with Biogen. These relationships or
those like them may require us to incur non-recurring and other charges, increase our near- and long-term expenditures, issue
securities that dilute our existing stockholders or disrupt our management and business. In addition, we could face significant
competition in seeking appropriate collaborators and the negotiation process is time-consuming and complex. Our ability to reach a
definitive collaboration agreement with any such new party will depend, among other things, upon our assessment of the
collaborator’s resources and expertise, the terms and conditions of the proposed collaboration and the proposed collaborator’s
evaluation of a number of factors. Moreover, we may not be successful in our efforts to establish a strategic partnership or other
alternative arrangements for our product candidates because our research and development pipeline may be insufficient, our product
candidates may be deemed to be at too early of a stage of development for collaborative effort and third parties may not view our
product candidates as having the requisite potential to demonstrate safety and efficacy. If we license product candidates, we may not
be able to realize the benefit of such transactions if we are unable to successfully integrate them with our existing operations and
company culture. We cannot be certain that, following a strategic transaction or license, we will achieve the revenues or specific net
income that justifies such transaction.
If we are unable to reach agreements with suitable collaborators on a timely basis, on acceptable terms, or at all, we may have to
curtail the development of a product candidate, reduce or delay its development program or one or more of our other development
programs, delay its potential commercialization or reduce the scope of any sales or marketing activities, or increase our expenditures
and undertake development or commercialization activities at our own expense. If we elect to fund and undertake development or
commercialization activities on our own, we may 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 and commercialization activities, we may not be able to further develop our product candidates
or bring them to market or continue to develop our gene therapy platform and our business may be materially and adversely affected.
Our reliance on third parties requires us to share our trade secrets, which increases the possibility that a competitor will
discover them or that our trade secrets will be misappropriated or disclosed.
Because we rely on third parties to manufacture our viral vectors and our product candidates, and because we collaborate with
various organizations and academic institutions on the advancement of our gene therapy platform, we must, at times, share trade
secrets with them. We seek to protect our proprietary technology in part by entering into confidentiality agreements and, if applicable,
material transfer agreements, collaborative research agreements, consulting agreements or other similar agreements with our
collaborators, advisors, employees and consultants prior to beginning research or disclosing proprietary information. These
agreements typically limit the rights of the third parties to use or disclose our confidential information, such as trade secrets.
Despite the contractual provisions employed when working with third parties, the need to share trade secrets and other
confidential information increases the risk that such trade secrets become known by our competitors, are inadvertently incorporated
into the technology of others, or are disclosed or used in violation of these agreements. Given that our proprietary position is based, in
part, on our know-how and trade secrets, a competitor’s discovery of our trade secrets or other unauthorized use or disclosure would
impair our competitive position and may have a material adverse effect on our business.
In addition, these agreements typically restrict the ability of our collaborators, advisors, employees and consultants to publish
data potentially relating to our trade secrets. Our academic collaborators typically have rights to publish data, provided that we are
notified in advance and may delay publication for a specified time in order to secure our intellectual property rights arising from the
collaboration. In other cases, publication rights are controlled exclusively by us, although in some cases we may share these rights
with other parties. We also conduct joint research and development programs that may require us to share trade secrets under the terms
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of our research and development partnerships or similar agreements. Despite our efforts to protect our trade secrets, our competitors
may discover our trade secrets, either through breach of these agreements, independent development or publication of information
including our trade secrets in cases where we do not have proprietary or otherwise protected rights at the time of publication. A
competitor’s discovery of our trade secrets would impair our competitive position and have an adverse impact on our business.
Risks related to commercialization of our product candidates
If we are unable to establish sales and marketing capabilities or enter into agreements with third parties to market and sell
our product candidates, we may be unable to generate any revenues.
We currently have no sales and marketing organization and have no experience selling and marketing our product candidates.
To successfully commercialize any products 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 sales force or the establishment of a
contract sales force to market any products we may develop will be expensive and time-consuming, particularly to the extent that we
seek to commercialize any product for an indication, such as wet AMD, that has a patient population significantly larger than those
addressed by our current lead product candidates, 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 with other entities to utilize their mature marketing and
distribution capabilities, but we may be unable to enter into marketing agreements on favorable terms, if at all. If our future
collaborators do not commit sufficient resources to commercialize our future products, if any, and we are unable to develop the
necessary marketing capabilities on our own, we will be unable to generate sufficient product revenue to sustain our business. We will
be competing with many companies that currently have extensive and well-funded marketing and sales 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 our 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.
We face intense competition and rapid technological change and the possibility that our competitors may develop therapies
that are more advanced or effective than ours, which may adversely affect our financial condition and our ability to successfully
commercialize our product candidates.
The biotechnology and pharmaceutical industries are characterized by intense and rapidly changing competition to develop new
technologies and proprietary products, and any product candidates that we successfully develop and commercialize will have to
compete with existing therapies and new therapies that may become available in the future. While we believe that our proprietary
technology estate and scientific expertise in the gene therapy field provide us with competitive advantages, we face potential
competition from many different sources, including larger and better-funded pharmaceutical, specialty pharmaceutical and
biotechnology companies, as well as from academic institutions and governmental agencies and public and private research
institutions that may develop potentially competitive products or technologies.
Currently there are no approved products for any of our lead orphan ophthalmology indications of XLRS, ACHM and XLRP.
We believe the key competitive factors that will affect the success of our product candidates, if approved, are likely to be their
efficacy, safety, convenience of administration and delivery, price, the level of generic competition and the availability of
reimbursement from government and other third-party payors.
A number of companies have announced that they are working on AAV-based gene therapy technology and there are companies
developing gene therapies in the field of orphan ophthalmology, on which we are currently focused, which have programs that are at
the clinical and pre-clinical stages. Other companies could also potentially seek to enter this field.
Many of our potential competitors, alone or with their strategic partners, have substantially greater financial, technical and
human resources than we do and significantly greater experience in the discovery and development of product candidates, obtaining
FDA and other regulatory approvals of treatments and the commercialization of those treatments. To the extent that we develop
product candidates for indications with larger patient populations, such as wet AMD, we expect to experience particularly intense
competition from larger and better funded pharmaceutical companies. Mergers and acquisitions in the biotechnology and
pharmaceutical industries may result in even more resources being concentrated among a smaller number of our competitors. Our
commercial opportunity could be reduced or eliminated if our competitors develop and commercialize products that are safer, more
effective, have fewer or less severe side effects, are more convenient or are less expensive than any products that we may develop.
Our competitors also may obtain FDA or other regulatory approval for their products more rapidly than we may obtain approval for
ours, which could result in our competitors establishing a strong market position before we are able to enter the market. Additionally,
market exclusivity provisions for products with orphan designation could severely limit the sales potential for any of our product
candidates that do not gain first-to-market approval.
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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 products, if approved, could limit our ability to market those products and decrease
our ability to generate revenue.
We expect the cost of a single administration of gene therapy products such as those we are developing to be substantial, when
and if they achieve regulatory approval. We expect that coverage and reimbursement by governmental and private payors will be
essential for most patients to be able to afford these treatments. Accordingly, sales of our product candidates will depend substantially,
both domestically and abroad, on the extent to which the costs of our product candidates will be paid by health maintenance, managed
care, pharmacy benefit and similar healthcare management organizations, or reimbursed by government authorities, private health
coverage insurers and other third-party payors. Coverage and reimbursement by a third-party payor may depend upon a number of
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; and
neither experimental nor investigational.
Obtaining coverage and reimbursement for a product from governmental and private payors is a time-consuming and costly
process that could require us to provide to the payor supporting scientific, clinical and cost-effectiveness data for the use of our
products. We may not be able to provide data sufficient to receive a positive coverage determination. If coverage and reimbursement
are not available, or are available only to limited levels, we may not be able to successfully commercialize our product candidates.
Even if coverage is provided, the approved reimbursement amount may not be high enough to allow us to establish or maintain pricing
sufficient to realize a sufficient return on our investment.
There is significant uncertainty related to third-party coverage and reimbursement of newly approved products. In the United
States, third-party payors, including private and governmental payors, such as the Medicare and Medicaid programs, play an important
role in determining the extent to which new drugs and biologics will be covered and reimbursed. The Medicare and Medicaid
programs increasingly are used as models for how private payors and other governmental payors develop their coverage and
reimbursement policies for drugs and biologics. Currently, no gene therapy products have been approved for coverage under the
Medicare program. The Centers for Medicare & Medicaid Services, or CMS, the agency responsible for administering the Medicare
program, covers some items and services nationally through National Coverage Determinations. More frequently, coverage
determinations for new products are made by the individual Medicare Administrative Contractors (MACs) that operate the program on
a day-to-day basis in their awarded geographic jurisdictions. It is difficult to predict what CMS or the local MACs 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 new products. Moreover, Medicare reimbursement is determined in part based on where the drug or biologic is
administered. Drugs or biologics administered in the inpatient setting are bundled along with other services into Diagnosis Related
Groups for payment purposes. In the outpatient setting drugs and biologics such as our product candidates are generally reimbursed at
Average Sales Price (ASP) + 6 %. Outside of the United States, agencies in Europe may be more conservative than CMS with respect
to reimbursement. For example, a number of cancer drugs have been approved for reimbursement in the United States and have not
been approved for reimbursement in certain European countries. It is difficult to predict at this time what third-party payors will
decide with respect to the coverage and reimbursement for our product candidates.
Outside the United States, international operations are generally subject to extensive governmental price controls and other
market regulations, and we believe the increasing emphasis on cost-containment initiatives in Europe, Canada, and other countries has
and will continue to put pressure on the pricing and usage of our product candidates. In many countries, the prices of medical products
are subject to varying price control mechanisms as part of national health systems. In general, the prices of medicines under such
systems are substantially lower than in the United States. Other countries allow companies to fix their own prices for medical
products, but monitor and control company profits. Additional foreign price controls or other changes in pricing regulation could
restrict the amount that we are able to charge for our product candidates and delay their commercial launch. Accordingly, in markets
outside the United States, the reimbursement for our products may be reduced or delayed compared with the United States and may be
insufficient to generate commercially reasonable revenues and profits.
Moreover, increasing efforts by governmental and third-party payors in the United States and abroad to cap or reduce healthcare
costs may cause such organizations to limit both coverage and the level of reimbursement for new products approved and, as a result,
they may not cover or provide adequate payment for our product candidates. We expect to experience pricing pressures in connection
with the sale of any of our product candidates, due to the trend toward managed healthcare, the increasing influence of health
maintenance organizations and potential legislative changes on both the federal and state levels. The downward pressure on healthcare
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costs in general, particularly prescription drugs and surgical procedures and other treatments, has become very intense. As a result,
increasingly high barriers are being erected to the entry of new products.
Negative public opinion and increased regulatory scrutiny of gene therapy and genetic research may damage public
perception of our product candidates or adversely affect our ability to conduct our business or obtain regulatory approvals for our
product candidates.
Gene therapy remains a novel technology, with only one gene therapy product approved to date in the United States and only
two gene therapy products approved to date in Europe. Public perception may be influenced by claims that gene therapy is unsafe, and
gene therapy may not gain the acceptance of the public or the medical community. In particular, our success will depend upon
physicians specializing in the treatment of those diseases that our product candidates target prescribing treatments that involve the use
of our product candidates in lieu of, or in addition to, existing treatments they are already familiar with and for which greater clinical
data may be available. More restrictive government regulations or negative public opinion would have a negative effect on our
business or financial condition and may delay or impair the development and commercialization of our product candidates or demand
for any products we may develop. For example, trials using early versions of lentiviral vectors, which integrate with, and thereby alter,
the host cell’s DNA, have led to several well-publicized adverse events, including reported cases of leukemia. Although none of our
current product candidates utilize lentiviral vectors, our product candidates use a viral delivery system. Adverse events in our clinical
trials or the clinical trials of other gene therapy companies, even if not ultimately attributable to our product candidates, and the
resulting publicity could result in increased governmental regulation, unfavorable public perception, potential regulatory delays in the
testing or approval of our product candidates, stricter labeling requirements for those product candidates that are approved and a
decrease in demand for any such product candidates.
Healthcare legislative reform measures may have a material adverse effect on our business and results of operations.
In the United States, there have been and continue to be a number of legislative initiatives to contain healthcare costs. For
example, in March 2010, the Patient Protection and Affordable Care Act, as amended by the Health Care and Education
Reconciliation Act or 2010, or the ACA, was passed, which substantially changes the way health care is financed by both
governmental and private insurers, and significantly impacts the U.S. pharmaceutical industry. The ACA, among other things, subjects
biologic products to potential competition by lower-cost biosimilars, addresses a new methodology by which rebates owed by
manufacturers under the Medicaid Drug Rebate Program are calculated for drugs that are inhaled, infused, instilled, implanted or
injected, increases the minimum Medicaid rebates owed by manufacturers under the Medicaid Drug Rebate Program and extends the
rebate program to individuals enrolled in Medicaid managed care organizations, establishes annual fees and taxes on manufacturers of
certain branded prescription drugs, and subjects additional drugs to lower pricing under the 340B drug pricing program by adding new
entities to the program.
In addition, other legislative changes have been proposed and adopted in the United States since the ACA was enacted. On
August 2, 2011, the Budget Control Act of 2011 among other things, created measures for spending reductions by Congress. A Joint
Select Committee on Deficit Reduction, tasked with recommending a targeted deficit reduction of at least $1.2 trillion for the years
2013 through 2021, was unable to reach required goals, thereby triggering the legislation’s automatic reduction to several government
programs. This includes aggregate reductions of Medicare payments to providers up to 2% per fiscal year, which went into effect on
April 1, 2013. 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.
Some of the provisions of the Affordable Care Act have yet to be fully implemented, while certain provisions have been subject
to judicial and Congressional challenges, and we expect there to be further challenges in the future. On January 20, 2017, President
Trump signed an Executive Order directing federal agencies with authorities and responsibilities under the ACA to waive, defer, grant
exemptions from, or delay the implementation of any provision of the ACA that would impose a fiscal burden on states or a cost, fee,
tax, penalty or regulatory burden on individuals, healthcare providers, health insurers, or manufacturers of pharmaceuticals or medical
devices. In May 2017, following the passage of the budget resolution for fiscal year 2017, the U.S. House of Representatives passed
legislation known as the American Health Care Act, which, if enacted, would amend or repeal significant portions of the ACA. The
U.S. Senate has not adopted the American Health Care Act as passed by the U.S. House of Representatives or other legislation to
amend or replace elements of the ACA. Thus, it is uncertain when or if the American Health Care Act will become law. In the coming
years, additional legislative and regulatory changes could be made to governmental health programs that could significantly impact
our business. The implications of these actions for our and our partners’ business and financial condition, if any, are not yet clear.
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The commercial success of any of our product candidates will depend upon its degree of market acceptance by physicians,
patients, third-party payors and others in the medical community.
Ethical, social and legal concerns about gene therapy and genetic research could result in additional regulations restricting or
prohibiting the products and processes we may use. Even with the requisite approvals from the FDA in the United States and other
government bodies internationally, the commercial success of our product candidates will depend in part on the medical community’s,
patients’, and third-party payors’ acceptance of gene therapy products in general, and our product candidates in particular, as
medically necessary, cost-effective, and safe. Any product that we bring to the market may not gain market acceptance by physicians,
patients, third-party payors and others in the medical community. 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 our product
candidates, if approved for commercial sale, will depend on a number of factors, including:
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the efficacy and safety of such product candidates as demonstrated in clinical trials;
the potential and perceived advantages of product candidates over alternative treatments;
the clinical indications for which the product candidate is approved;
the safety of product candidates seen in a broader patient group, including its use outside the approved indications;
the prevalence and severity of any side effects;
product labeling or product insert requirements of the FDA or other regulatory authorities, including any limitations or
warnings contained in a product’s approved labeling;
the cost of treatment relative to alternative treatments;
relative convenience and ease of administration;
the willingness of the target patient population to try new therapies and of physicians to prescribe these therapies;
the strength of marketing and distribution support;
label limitations required by regulatory authorities, which could limit size of market;
the timing of market introduction of competitive products;
publicity concerning our products or competing products and treatments; and
sufficient third-party insurance coverage and reimbursement.
Even if a potential product 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 the product candidates may require significant resources and may never be successful. Such efforts to
educate the marketplace may require more resources than are required by the conventional technologies marketed by our competitors.
If any of our product candidates is approved but fails to achieve market acceptance among physicians, patients, or health care payors,
we will not be able to generate significant revenues from such product, which could have a material adverse effect on our business,
prospects, financial condition and results of operations.
If we obtain approval to commercialize our product candidates outside of the United States, a variety of risks associated with
international operations could materially adversely affect our business.
If any of our product candidates are approved for commercialization, we may enter into agreements with third parties to market
them on a worldwide basis or in more limited geographical regions. We expect that we will be subject to additional risks related to
entering into international business relationships, including:
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different regulatory requirements for approval of drugs and biologics in foreign countries;
the potential for so-called parallel importing, which is what happens when a local seller, faced with high or higher local
prices, opts to import goods from a foreign market (with low or lower prices) rather than buying them locally;
challenges enforcing our contractual and intellectual property rights, especially in those foreign countries that do not
respect and protect intellectual property rights to the same extent as the United States;
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;
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foreign currency fluctuations, which could result in increased operating expenses and reduced revenues, and other
obligations incident to doing business in another country;
difficulties staffing and managing foreign operations;
workforce uncertainty in countries where labor unrest is more common than in the United States;
potential liability under the Foreign Corrupt Practices Act of 1977 or comparable foreign regulations;
production shortages resulting from any events affecting raw 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.
These and other risks associated with our international operations may materially adversely affect our ability to attain or
maintain profitable operations.
We may not be successful in our efforts to identify or discover additional product candidates.
The success of our business depends primarily upon our ability to identify, develop and commercialize product candidates based
on our gene therapy platform. Although certain of our product candidates are currently in clinical or preclinical development, we may
fail to identify other potential product candidates for clinical development for a number of reasons. For example, our research
methodology may be unsuccessful in identifying potential product candidates or our potential product candidates may be shown to
have harmful side effects or may have other characteristics that may make the products unmarketable or unlikely to receive marketing
approval.
If any of these events occur, we may be forced to abandon our development efforts with respect to a particular product
candidate, which would have a material adverse effect on our business and could potentially cause us to cease operations. Research
programs to identify new product candidates require substantial technical, financial and human resources. We may focus our efforts
and resources on potential programs or product candidates that ultimately prove to be unsuccessful.
Risks related to our business operations
We incur significant increased costs as a result of operating as a public company, and our management devotes substantial
time to new compliance initiatives.
As a public company, we have incurred and will continue to incur significant legal, accounting and other expenses that we did
not incur as a private company. In addition, the Sarbanes-Oxley Act of 2002, or the Sarbanes-Oxley Act, as well as rules subsequently
implemented by the Securities and Exchange Commission, or SEC, and The NASDAQ Global Market impose various requirements
on public companies. In July 2010, the Dodd-Frank Wall Street Reform and Consumer Protection Act, or the Dodd-Frank Act, was
enacted. There are significant corporate governance and executive compensation related provisions in the Dodd-Frank Act that require
the SEC to adopt additional rules and regulations in these areas. Stockholder activism, the current political environment and the
current high level of government intervention and regulatory reform may lead to substantial new regulations and disclosure
obligations, which may lead to additional compliance costs and impact the manner in which we operate our business in ways we
cannot currently anticipate.
We may not be successful in complying with these obligations, and compliance with these obligations could be time-consuming
and expensive. If these requirements divert the attention of our management and personnel from other business concerns, they could
have a material adverse effect on our business, financial condition and results of operations. 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, which in turn could make it more difficult for us to attract and retain qualified members of our board of directors. The
increased costs will decrease our net income or increase our net loss, and may require us to reduce costs in other areas of our business
or increase the prices of our products or services.
We have identified material weaknesses in our internal control over financial reporting, and if we are unable to maintain
effective internal control over financial reporting, investors could lose confidence in our financial statements and our company
which could have a material adverse effect on our business and our stock price.
Our management previously determined that as of June 30, 2016, 2015 and 2014, we had material weaknesses in our internal
control over financial reporting, which related to the design and operation of our closing and financial reporting processes and our
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accounting for debt, equity and convertible instruments (as described in our Annual Reports on Form 10-K for such fiscal years).
Management has determined that as of June 30, 2017, a material weakness in our internal control over financial reporting related to
the design and operation of our closing and financial reporting processes still existed, as described in Item 9A of this Annual Report
on Form 10-K.
If we fail to maintain effective internal controls in the future, it could result in a material misstatement of our financial
statements, which could cause investors to lose confidence in our financial statements and our company or cause our stock price to
decline. Our independent registered public accounting firm has not assessed the effectiveness of our internal control over financial
reporting and, under the JOBS Act, will not be required to provide an attestation report on the effectiveness of our internal control
over financial reporting so long as we qualify as an emerging growth company, which may increase the risk that weaknesses or
deficiencies in our internal control over financial reporting go undetected.
If we are unable to manage expected 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 continue 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 sales force and commercial infrastructure to support commercialization of any of our product candidates that are approved for
sale. Future growth would impose significant added responsibilities on members of management. It is possible 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 products 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.
We may enter into or seek to enter into business partnerships, combinations and/or acquisitions which may be difficult to
integrate, disrupt our business, divert management attention or dilute stockholder value.
A key element of our strategy is to enter into business partnerships, combinations and/or acquisitions. We have limited
experience in making acquisitions, which are typically accompanied by a number of risks, including:
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the difficulty of integrating the operations and personnel of the acquired companies;
the potential disruption of our ongoing business and distraction of management;
potential unknown liabilities and expenses;
the failure to achieve the expected benefits of the combination or acquisition;
the maintenance of acceptable standards, controls, procedures and policies; and
the impairment of relationships with employees as a result of any integration of new management and other personnel.
If we are not successful in completing acquisitions that we may pursue in the future, we would be required to reevaluate our
business strategy and we may have incurred substantial expenses and devoted significant management time and resources in seeking
to complete the acquisitions. In addition, we could use substantial portions of our available cash as all or a portion of the purchase
price, or we could issue additional securities as consideration for these acquisitions, which could cause our stockholders to suffer
significant dilution.
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 our executive officers, the loss of whose services may adversely impact the achievement of our
objectives. Recruiting and retaining other qualified employees, consultants and advisors for our business, including scientific and
technical personnel, will also be critical to our success. There is currently a shortage of skilled executives and scientific personnel in
our industry, which is likely to continue. As a result, competition for skilled personnel 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 for individuals with similar skill sets. In addition, failure to succeed in preclinical studies or clinical trials
may make it more challenging to recruit and retain qualified personnel. The inability to recruit or loss of the services of any executive,
key employee, consultant or advisor may impede the progress of our research, development and commercialization objectives.
In order to induce valuable employees to remain at AGTC, in addition to salary and cash incentives, we have provided stock
options that vest over time. The value to employees of stock options that vest over time may be significantly affected by movements in
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our stock price that are beyond our control, and may at any time be insufficient to counteract more lucrative offers from other
companies.
Despite our efforts to retain valuable employees, members of our management, scientific and development teams have in the
past and may in the future terminate their employment with us. The loss of the services of any of our executive officers or other key
employees and our inability to find suitable replacements could potentially harm our business, prospects, financial condition or results
of operations. We do not maintain “key man” insurance policies on the lives of these individuals or any of our other employees. Our
success also depends on our ability to continue to attract, retain and motivate highly skilled junior, mid-level, and senior managers as
well as junior, mid-level, and senior scientific and medical personnel.
Many of the other biotechnology and pharmaceutical companies that we compete against for qualified personnel have greater
financial and other resources, different risk profiles and a longer history in the industry than we do. They may also provide more
diverse opportunities and better chances for career advancement. Some of these characteristics may be more appealing to high quality
candidates than what we can offer. If we are unable to continue to attract and retain high quality personnel, the rate and success at
which we can discover, develop and commercialize product candidates will be limited.
Our employees, principal investigators, consultants and commercial partners may engage in misconduct or other improper
activities.
We are exposed to the risk that our employees, CROs, principal investigators, consultants and commercial partners may engage
in fraudulent conduct or other illegal activity or may fail to disclosure unauthorized activities to us. Misconduct by these parties could
include intentional, reckless and/or negligent failures to comply with:
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the laws and regulations of the FDA and non-U.S. regulators, including those laws requiring the reporting of true,
complete and accurate information to such regulatory bodies;
manufacturing standards we have established;
healthcare fraud and abuse laws and regulations in the United States and similar foreign laws; or
laws requiring the accurate reporting of financial information or data or the disclosure of unauthorized activities to us.
In particular, sales, marketing and business arrangements in the healthcare industry are subject to extensive laws and regulations
intended to prevent fraud, misconduct, kickbacks, self-dealing and other abusive practices. These laws and regulations may restrict or
prohibit a wide range of pricing, discounting, marketing and promotion, sales commission, customer incentive programs and other
business arrangements. Such misconduct could also involve the improper use of information obtained in the course of clinical trials,
which could result in regulatory sanctions and cause serious harm to our reputation. We have adopted a code of conduct applicable to
all of our employees, but 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 comply with these laws or regulations. 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, including the imposition of significant fines or other sanctions.
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We may be subject, directly or indirectly, to federal and state healthcare fraud and abuse laws, false claims laws and health
information privacy and security laws. If we are unable to comply, or have not fully complied, with such laws, we could face
substantial penalties.
Our operations may be directly, or indirectly through our prescribers, customers and purchasers, subject to various federal and
state fraud and abuse laws. If we obtain FDA approval for any of our product candidates and begin commercializing those products in
the United States, many of these laws will become more directly applicable to our operations, including, without limitation, the federal
Health Care Program Anti-Kickback Statute, the federal civil and criminal False Claims Acts and Physician Payments Sunshine Act
and regulations. These laws may 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
may affect our ability to operate 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 healthcare program, such as the Medicare and Medicaid
programs;
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 payers that are false or fraudulent;
the federal Health Insurance Portability and Accountability Act of 1996, or 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 healthcare 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;
federal transparency laws, including the federal Physician Payment Sunshine Act that requires disclosure of payments and
other transfers of value provided to physicians and teaching hospitals, and ownership and investment interests held by
physicians and other healthcare providers and their immediate family members and applicable group purchasing
organizations;
the Patient Protection and Affordable Care Act, as amended by the Health Care and Education Reconciliation Act, or the
Affordable Care Act, and its implementing regulations, which may impact, among other things, reimbursement rates by
federal health care programs and commercial insurers;
federal consumer protection and unfair competition laws, which broadly regulate marketplace activities and activities that
potentially harm consumers;
federal government price reporting laws, which require us to calculate and report complex pricing metrics to government
programs, where such reported prices may be used in the calculation of reimbursement and/or discounts on our marketed
drugs, when and if approved; participation in these programs and compliance with the applicable requirements may
subject us to potentially significant discounts on our products, when and if approved, increased infrastructure costs and
potentially limit our ability to offer certain marketplace discounts; and
state law equivalents of each of the above federal laws, such as anti-kickback and false claims laws which may apply to
items or services reimbursed by any third-party payor, including commercial insurers; state laws that require
pharmaceutical companies to comply with the industry’s voluntary compliance guidelines and the applicable compliance
guidance promulgated by the federal government, or otherwise restrict certain payments that may be made to healthcare
providers and other potential referral sources; state laws that require drug manufacturers to report information related to
payments and other transfers of value to physicians and other healthcare 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.
In addition, any sale of our products or product candidates, if commercialized outside of the United States, may also subject us
to foreign laws governing prescription drug marketing and fraud and abuse, including laws similar to the U.S. healthcare laws
mentioned above. Because of the breadth of these laws and the narrowness of the statutory exceptions and safe harbors available, it is
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possible that some of our business activities could be subject to challenge under one or more of such laws. In addition, recent health
care reform legislation has strengthened these laws. For example, the PPACA, among other things, amends the intent requirements of
the federal Anti-Kickback Statute and the criminal statute governing healthcare fraud. A person or entity can now be found guilty of
violating the Anti-Kickback Statute and the federal criminal healthcare fraud statute without actual knowledge of the statute or
specific intent to violate it. In addition, the PPACA provides that the government may assert that a claim including items or services
resulting from a violation of the federal Anti-Kickback Statute constitutes a false or fraudulent claim for purposes of the federal False
Claims Act.
If our operations are found to be in violation of any of the laws described above or any other governmental 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, disgorgement, contractual damages, reputational harm, diminished
profits and future earnings, imprisonment, and the curtailment or restructuring of our operations, any of which could adversely affect
our ability to operate our business and our results of operations.
If the use of our product candidates harms patients, we could be subject to costly and damaging product liability claims.
The use of our product candidates in clinical trials and the sale of any products for which we obtain marketing approval exposes
us to the risk of product liability claims. Product liability claims might be brought against us by consumers, healthcare providers,
pharmaceutical companies or others selling or otherwise coming into contact with our products. For example, we may be sued if any
product candidate we develop allegedly causes injury or is found to be otherwise unsuitable during clinical testing, manufacturing,
marketing or sale. Any such product liability claims may include allegations of defects in manufacturing, defects in design, a failure to
warn of dangers inherent in the product, negligence, strict liability, and a breach of warranties. Claims could also be asserted under
state consumer protection acts. If we cannot successfully defend against product liability claims, we could incur substantial liability
and costs. In addition, regardless of merit or eventual outcome, product liability claims may result in:
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impairment of our business reputation;
withdrawal of clinical trial participants;
initiation of investigations by regulators;
costs due to related litigation;
distraction of management’s attention from our primary business;
substantial monetary awards to trial participants, patients or other claimants;
loss of revenue;
exhaustion of any available insurance and our capital resources;
the inability to commercialize our product candidates; and
decreased demand for our product candidates, if approved for commercial sale.
Our inability to obtain and retain sufficient product liability insurance at an acceptable cost to protect against potential product
liability claims could prevent or inhibit the commercialization of products we develop. While we believe our product liability
insurance coverage is sufficient in light of our current clinical programs. The amount of the product liability coverage that we carry
varies from time to time, depending on a number of factors, the most significant of which are the nature and scope of the clinical trials
in which we are engaged and the number of patients being treated with our product candidates in these trials. This amount may
increase or decrease in the future. We may not be able to maintain insurance coverage at a reasonable cost or in sufficient amounts to
protect us against losses due to liability and any claim that may be brought against us could result in a court judgment or settlement in
an amount that is not covered, in whole or in part, by our insurance or that is in excess of the limits of our insurance coverage. If and
when we obtain marketing approval for product candidates, we intend to expand our insurance coverage to include the commercial
sale of our products; however, we may be unable to obtain product liability insurance on commercially reasonable terms or in
adequate amounts. Our insurance policies also have various exclusions, and we may be subject to a product liability claim for which
we have no coverage. A successful product liability claim or series of claims brought against us could cause our stock price to decline
and, if judgments exceed our insurance coverage, could adversely affect our results of operations and business.
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 handling, use, storage, treatment, manufacture and disposal of hazardous materials and wastes. Our operations
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involve the use of hazardous and flammable materials, including chemicals and biological materials. Our operations also produce
hazardous waste products. We generally contract with third parties for the disposal of these materials and wastes. Although we believe
that our procedures for using, handling, storing and disposing of these materials comply with legally prescribed standards, 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, and any liability could exceed our resources. We also could
incur significant costs associated with civil or criminal fines and penalties.
Although we maintain workers’ compensation insurance to cover us for 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. In addition, we may incur substantial costs in order to comply with current or future
environmental, health and safety laws and regulations. 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.
We rely on our relationship with a professional employer organization for our human relations function and as a co-
employer of our personnel, and if that party failed to perform its responsibilities under that relationship, our relations with our
employees could be damaged and we could incur liabilities that could have a material adverse effect on our business.
All of our personnel, including our executive officers, are co-employees of AGTC and a professional employer organization,
TriNet HR Corporation, or TriNet. Under the terms of our arrangement, TriNet is the formal employer of all of our personnel, and is
responsible for administering all payroll, including tax withholding, and providing health insurance and other benefits for these
individuals. We reimburse TriNet for these costs, and pay TriNet an administrative fee for its services. If TriNet fails to comply with
applicable laws, or its obligations under this arrangement, our relationship with our employees could be damaged. We could, under
certain circumstances, be held liable for a failure by TriNet to appropriately pay, or withhold and remit required taxes from payments
to, our employees. In such a case, our potential liability could be significant and could have a material adverse effect on our business.
We or the third parties upon whom we depend may be adversely affected by natural disasters and our business continuity and
disaster recovery plans may not adequately protect us from a serious disaster.
Substantially all of our manufacturing operations and a majority of our research and development operations are conducted from
our headquarters located near Gainesville, Florida. Hurricanes or other natural disasters could severely disrupt our operations, damage
our research facilities or destroy stored research materials that could be difficult to replace, and otherwise have a material adverse
effect on our business, results of operations, financial condition and prospects. In addition, despite the implementation of security
measures, our internal computer systems and those of our current and any future CROs and other contractors and consultants and
collaborators are vulnerable to damage from computer viruses, unauthorized access, natural disasters, terrorism, war and
telecommunication and electrical failures. If such an event were to occur and cause interruptions in our operations, it could result in a
material disruption of our development programs and our business operations. If a natural disaster, power outage or other event
occurred that prevented us from using all or a significant portion of our headquarters, that damaged critical infrastructure or that
otherwise disrupted our operations or the operations of our third-party contract manufacturer, it may be difficult or, in certain cases,
impossible for us to continue our business for a substantial period of time. For example, the loss of clinical trial data from our clinical
trials could result in delays in our regulatory approval efforts and significantly increase our costs to recover or reproduce the data. If
our security measures, disaster recovery and business continuity plans are not adequate in the event of such a breach, serious disaster
or similar event, we could incur substantial expenses and the further development and commercialization of our product candidates
could be delayed, which could have a material adverse effect on our business.
Interruptions in the supply of product or inventory loss may adversely affect our operating results and financial condition.
Our product candidates are manufactured using technically complex processes requiring specialized facilities, highly specific
raw materials and other production constraints. The complexity of these processes, as well as strict company and government
standards for the manufacture and storage of our products, subjects us to production risks. While product batches released for use in
clinical trials or for commercialization undergo sample testing, such testing is subject to human error and some defects may only be
identified following product release. In addition, process deviations or unanticipated effects of approved process changes may result in
these intermediate products not complying with stability requirements or specifications. Most of our product candidates must be stored
and transported at temperatures within a certain range. If these environmental conditions deviate, our product candidates’ remaining
shelf lives could be impaired or their efficacy and safety could become adversely affected, making them no longer suitable for use.
The occurrence or suspected occurrence of production and distribution difficulties can lead to lost inventories, and in some cases
product recalls, with consequential reputational damage and the risk of product liability. The investigation and remediation of any
identified problems can cause production delays, substantial expense, lost sales and delays of new product launches. Any interruption
in the supply of finished products or the loss thereof could hinder our ability to timely distribute our products and satisfy customer
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demand. Any unforeseen failure in the storage of the product or loss in supply could delay our clinical trials and, if our product
candidates are approved, result in a loss of our market share and negatively affect our revenues and operations.
We may use our financial and human resources to pursue a particular research program or product candidate and fail to
capitalize on programs or product candidates that may be more profitable or for which there is a greater likelihood of success.
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. Our resource allocation decisions may cause us to fail to
capitalize on viable commercial products or profitable market opportunities. Our spending on current and future research and
development programs for product candidates may not yield any commercially viable products. If we do not accurately evaluate the
commercial potential or target market for a particular product candidate, we may relinquish valuable rights to that product candidate
through strategic collaboration, licensing or other royalty arrangements in cases in which it would have been more advantageous for us
to retain sole development and commercialization rights to such product candidate, or 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.
Our ability to use our net operating loss carryforwards may be subject to limitation.
Under Section 382 of the Internal Revenue Code of 1986, as amended, substantial changes in our ownership may limit the
amount of net operating loss carryforwards that could be utilized annually in the future to offset our taxable income. Specifically, this
limitation may arise in the event of a cumulative change in ownership of our company of more than 50% within a three-year period.
Any such annual limitation may significantly reduce the utilization of our net operating loss carryforwards before they expire. We
believe it is likely that transactions that have occurred in the past and other transactions that may occur in the future, could trigger an
ownership change pursuant to Section 382, which could limit the amount of net operating loss carryforwards that could be utilized
annually in the future to offset our taxable income, if any.
Cyber attacks or other breaches of network or other information technology security could have an adverse effect on our
business.
Cyber attacks or other breaches of network or information technology security may cause equipment failures or disruptions to
our operations. While, to date, we have not been subject to cyber attacks or other cyber incidents which, individually or in the
aggregate, have been material to our operations or financial condition, the preventative actions we take to prevent or detect the risk of
cyber incidents and protect our information technology and networks may be insufficient to prevent or detect a major cyber attack in
the future. If we fail to prevent the theft of valuable information such as financial data, sensitive information about the us, our patients
or our intellectual property, or if we fail to protect the privacy of patient and employee confidential data against breaches of network
or information technology security, it would result in damage to our reputation, which could adversely impact the confidence of our
partners, investors and employees. Any of these occurrences could result in a material adverse effect on our results of operations and
financial condition.
Risks related to our intellectual property
If we are unable to obtain and maintain patent protection for our technology and products or if the scope of the patent
protection obtained is not sufficiently broad, our competitors could develop and commercialize technology and products similar or
identical to ours, and our ability to successfully commercialize our technology and products may be impaired.
Our success depends in large part on our ability to obtain and maintain patent protection in the United States and other countries
with respect to our proprietary technology and products. We seek to protect our proprietary position by filing patent applications in the
United States and abroad related to our novel technologies and product candidates.
The patent prosecution process is expensive and time-consuming, and we may not be able to file and prosecute all necessary or
desirable patent applications at a reasonable cost or in a timely manner. 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. Moreover, in some circumstances, we
do not have the right to control the preparation, filing and prosecution of patent applications, or to maintain the patents, covering
technology that we license from third parties. Therefore, these patents and applications may not be prosecuted and enforced in a
manner consistent with the best interests of our business.
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. In addition, the laws of foreign countries may not protect
our rights to the same extent as the laws of the United States. For example, European patent law restricts the patentability of methods
of treatment of the human body more than United States law does. Publications of discoveries in the scientific literature often lag
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behind the actual discoveries, and patent applications in the United States and other jurisdictions are typically not published until 18
months after filing, or in some cases not at all. Therefore, we cannot know with certainty whether we were the first to make the
inventions claimed in our owned or licensed patents or pending patent applications, or that we were the first to file for patent
protection of such inventions. As a result, the issuance, scope, validity, enforceability and commercial value of our patent rights are
highly uncertain. Our pending and future patent applications may not result in issued patents that protect our technology or products,
in whole or in part, or which effectively prevent others from commercializing competitive technologies and products. Changes in
either the patent laws or interpretation of the patent laws in the United States and other countries may diminish the value of our patents
or narrow the scope of our patent protection.
Moreover, we may be subject to a third-party preissuance submission of prior art to the United States Patent and Trademark
Office, or become involved in opposition, derivation, reexamination, inter partes review, post-grant review or interference
proceedings challenging our patent rights or the patent rights of others. An adverse determination in any such submission, proceeding
or litigation could reduce the scope of, or invalidate, our patent rights, allow third parties to commercialize our technology or products
and compete directly with us, without payment to us, or result in our inability to manufacture or commercialize products without
infringing third-party patent rights. In addition, if the breadth or strength of protection provided by our patents and patent applications
is threatened, it could dissuade companies from collaborating with us to license, develop or commercialize current or future product
candidates.
Even if our owned and licensed patent applications issue as patents, they may not issue in a form that will provide us with any
meaningful protection, prevent competitors from competing with us or otherwise provide us with any competitive advantage. Our
competitors may be able to circumvent our owned or licensed patents by developing similar or alternative technologies or products in
a non-infringing manner.
In addition, the issuance of a patent is not conclusive as to its inventorship, scope, validity or enforceability, and our owned and
licensed patents may be challenged in the courts or patent offices in the United States and abroad. Such challenges 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 or identical technology and products, or limit the duration
of the patent protection of our technology and products. 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 owned and licensed patent portfolio may not provide us with sufficient rights to exclude others from
commercializing products similar or identical to ours.
Third parties may initiate legal proceedings alleging claims of intellectual property infringement, the outcome of which
would be uncertain and could have a material adverse effect on the success of our business.
Our commercial success depends in part on our avoiding infringement of the patents and proprietary rights of third parties.
There is a substantial amount of litigation, both within and outside the United States, involving patent and other intellectual property
rights in the biotechnology and pharmaceutical industries, including patent infringement lawsuits, interferences, oppositions and inter
partes reexamination proceedings before the United States Patent and Trademark Office and corresponding foreign patent offices.
Numerous United States and foreign issued patents and pending patent applications, which are owned by third parties, exist in the
fields in which we are pursuing development candidates. As the biotechnology and pharmaceutical industries expand and more patents
are issued, the risk increases that our product candidates may be subject to claims of infringement of the patent rights of third parties.
Third parties may assert that we are employing their proprietary technology without authorization. There may be third-party
patents or patent applications with claims to materials, formulations, methods of manufacture or methods for treatment related to the
use or manufacture of our product candidates. Because patent applications can take many years to issue, there may be currently
pending patent applications that may later result in issued patents that our product candidates may infringe. In addition, third parties
may obtain patents in the future and claim that use of our technologies infringes upon these patents. If any third-party patents were
held by a court of competent jurisdiction to cover the manufacturing process of any of our product candidates, any molecules formed
during the manufacturing process or any final product itself, the holders of any such patents may be able to block our ability to
commercialize such product candidate unless we obtained a license under the applicable patents, or until such patents expire.
Similarly, if any third-party patents were held by a court of competent jurisdiction to cover aspects of our formulations, methods
for manufacture or methods of use, including combination therapy, the holders of any such patents may be able to block our ability to
develop and commercialize the applicable product candidate unless we obtained a license or until such patent expires. In either case,
such a license may not be available 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 access to the same technologies licensed to us.
Parties making claims against us may obtain injunctive or other equitable relief, which could effectively block our ability to
further develop and commercialize one or more of our product candidates. Defense of these claims, regardless of their merit, would
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involve substantial litigation expense and would be a substantial diversion of employee resources from our business. In the event of a
successful claim of infringement against us, we may have to pay substantial damages, including treble damages and attorneys’ fees for
willful infringement, pay royalties, redesign our infringing products or obtain one or more licenses from third parties, which may be
impossible or require substantial time and monetary expenditure.
We may not be successful in obtaining or maintaining necessary rights to gene therapy product components and processes
for our development pipeline through acquisitions and in-licenses.
Presently we have rights to the intellectual property to develop our gene therapy product candidates. Because a key element of
our business strategy is to pursue in-licensing and intellectual property acquisitions for additional product candidates that may require
the use of proprietary rights held by third parties, the growth of our business will likely depend in part on our ability to acquire, in-
license or use these proprietary rights. In addition, our product candidates may require specific formulations to work effectively and
efficiently and these rights may be held by others. We may be unable to acquire or in-license any compositions, methods of use,
processes or other third-party intellectual property rights from third parties that we identify on terms that we find acceptable, or at all.
The licensing and acquisition of third-party intellectual property rights is a competitive area, and a number of more established
companies are also pursuing 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, cash resources and greater clinical
development and commercialization capabilities.
For example, we sometimes collaborate with United States and foreign academic institutions to accelerate our preclinical
research or development under written agreements with these institutions. Typically, these institutions 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 right of first
negotiation for intellectual property, we may be unable to negotiate a license within the specified time frame 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.
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. If we are unable to successfully obtain rights to required third-party intellectual property rights, our business, financial
condition and prospects for growth could suffer.
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.
Licensing of intellectual property is of critical importance to our business and involves complex legal, business and scientific
issues and is complicated by the rapid pace of scientific discovery in our industry. We are a party to intellectual property license
agreements with the University of Florida Research Foundation, an affiliate of the University of Florida, Johns Hopkins University
and the UAB Research Foundation, an affiliate of The University of Alabama at Birmingham, each of which is important to our
business, and we expect to enter into additional license agreements in the future. Our existing license agreements impose, and we
expect that future license agreements will impose, various diligence, milestone payment, royalty and other obligations on us. If we fail
to comply with our obligations under these agreements, or we are subject to a bankruptcy, the licensor may have the right to terminate
the license, in which event we would not be able to market products covered by the license.
We may need to obtain licenses from third parties to advance our research or allow commercialization of our product
candidates, and we have done so from time to time. It is possible that we may fail to obtain any of these 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 develop or license
replacement technology. If we are unable to do so, we may be unable to develop or commercialize the affected product candidates,
which could harm our business significantly. We cannot provide any assurances that third-party patents do not exist which might be
enforced against our current product candidates or future products, resulting in either an injunction prohibiting our sales, or, with
respect to our sales, an obligation on our part to pay royalties and/or other forms of compensation to third parties.
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In many cases, patent prosecution of our licensed technology is controlled solely by the licensor. 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. In certain cases, we control the prosecution of patents resulting from licensed technology. In the event we breach
any of our obligations related to such prosecution, we may incur significant liability to our licensing partners. Disputes may arise
regarding intellectual property subject to a licensing agreement, including:
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the scope of rights granted under the license agreement and other interpretation-related issues;
the extent to which our technology and 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;
our diligence obligations under the license agreement and what activities satisfy those diligence obligations;
the 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 patented technology.
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 the affected product candidates.
We may become involved in lawsuits to protect or enforce our patents or other intellectual property or the patents or other
intellectual property of our licensors, which could be expensive, time-consuming and ultimately unsuccessful.
Competitors may infringe our patents or other intellectual property or the patents or other intellectual property of our licensors.
In response, we may be required to file infringement claims, which can be expensive and time-consuming. Any claims we assert
against perceived infringers could provoke these parties to assert counterclaims against us, alleging that we infringe their patents.
Additionally, if the party against whom we bring a claim of infringement has a relationship with one or more of our collaborators,
licensors or other strategic counterparties, our relationship with that counterparty may be harmed. Similarly, because our intellectual
property is potentially useful for the treatment of serious diseases, any third party infringers may be viewed sympathetically by the
public and our assertion of an infringement claim against them may hurt our reputation. In addition, in a patent infringement
proceeding, a court may decide that a patent of ours or our licensors is invalid or unenforceable, in whole or in part, construe the
patent’s claims narrowly or refuse to stop the other party from using the technology at issue on the grounds that our patents do not
cover the technology in question. An adverse result in any litigation or defense proceeding could put one or more of our patents at risk
of being invalidated or interpreted narrowly and could put our patent applications at risk of not issuing.
Interference proceedings provoked by third parties or brought by us may be necessary to determine the priority of inventions
with respect to our patents or patent applications or those of our licensors. An unfavorable outcome could require us to cease using the
related technology or to attempt to license rights to it from the prevailing party. Our business could be harmed if the prevailing party
does not offer us a license on commercially reasonable terms. Our defense of litigation or interference proceedings may fail and, even
if successful, may result in substantial costs and distract our management and other employees. We may not be able to prevent, alone
or with our licensors, misappropriation of our intellectual property rights, particularly in countries where the laws may not protect
those rights as fully as in the United States.
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. There could also be
public announcements of the results of hearings, motions or other interim proceedings or developments. If securities analysts or
investors perceive these results to be negative, it could have a material adverse effect on the price of our common stock.
Issued patents covering our product candidates or methods of manufacturing could be found invalid or unenforceable if
challenged in court.
If we or one of our licensing partners initiate legal proceedings against a third party to enforce a patent covering one of our
product candidates, or methods of manufacturing our product candidates, the defendant could counterclaim that the patent covering
our product candidate or method is invalid or unenforceable. In patent litigation in the United States, defendant counterclaims alleging
invalidity or unenforceability are commonplace. Grounds for a validity challenge could be an alleged failure to meet any of several
statutory requirements, including lack of novelty, obviousness or non-enablement. Grounds for an unenforceability assertion could be
an allegation that someone connected with prosecution of the patent withheld relevant information from the United States Patent and
Trademark Office, or made a misleading statement, during prosecution. Third parties may also raise similar claims before
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administrative bodies in the United States or abroad, even outside the context of litigation. Such mechanisms include re-examination,
post grant review, and equivalent proceedings in foreign jurisdictions. Such proceedings could result in the revocation of or
amendment to our patents in such a way that they no longer cover our product candidates or manufacturing methods. The outcome
following legal assertions of invalidity and unenforceability is unpredictable. With respect to the validity question, for example, we
cannot be certain that there is no invalidating prior art, of which we and the patent examiner were unaware during prosecution. If a
defendant were to prevail on a legal assertion of invalidity or unenforceability, we could lose at least part, and perhaps all, of the
patent protection on one or more of our product candidates or methods of manufacturing our products. Such a loss of patent protection
could have a material adverse impact on our business.
We may be subject to claims that our employees, consultants or independent contractors have wrongfully used or disclosed
confidential information of third parties or that our employees have wrongfully used or disclosed alleged trade secrets of their
former employers.
We employ individuals who were previously employed at universities or other biotechnology or pharmaceutical companies,
including our competitors or potential competitors. Although we have enacted policies and procedures designed to ensure that our
employees, consultants and independent contractors do not use the proprietary information or know-how of others in their work for us,
we may be subject to claims that we or our employees, consultants or independent contractors have inadvertently or otherwise used or
disclosed intellectual property, including trade secrets or other proprietary information, of any of our employee’s former employer or
other third parties. 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, which could adversely impact our business.
Even if we are successful in defending against such claims, litigation could result in substantial costs and be a distraction to
management and other employees.
We may be subject to claims challenging the inventorship or ownership of our patents and other intellectual property.
We may be subject to claims that former employees, collaborators or other third parties have an ownership interest in our
patents or other intellectual property. While it is our policy to require our employees and contractors who may be involved in the
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 develops intellectual property that we regard as our own. We could be
subject to ownership disputes arising, for example, from conflicting obligations of consultants or others who are involved in
developing our product candidates. Litigation may be necessary to defend against these and other claims challenging inventorship or
ownership. If we fail in defending any such claims, in addition to paying monetary damages, we may lose valuable intellectual
property rights, such as exclusive ownership of, or right to use, valuable intellectual property. Such an outcome could have a material
adverse effect on our business. Even if we are successful in defending against such claims, litigation could result in substantial costs
and be a distraction to management and other employees.
Intellectual property litigation could cause us to spend substantial resources and distract our personnel from their normal
responsibilities.
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. 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 conduct
such litigation or proceedings adequately. 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. Uncertainties resulting from the initiation and continuation
of patent litigation or other proceedings could compromise 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 governmental 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 governmental fees on patents and/or applications will be
due to be paid to the United States Patent and Trademark Office and various governmental patent agencies outside of the United States
in several stages over the lifetime of the patents and/or applications. We rely on our outside counsel to pay these fees due to non-U.S.
patent agencies. The United States Patent and Trademark Office and various non-U.S. governmental patent agencies require
compliance with a number of procedural, documentary, fee payment and other similar provisions during the patent application
process. We employ reputable law firms and other professionals to help us comply, and 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. However, there are situations in which non-
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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.
Changes in United States patent law could diminish the value of patents in general, thereby impairing our ability to protect
our products.
As is the case with other biotechnology companies, our success is heavily dependent on intellectual property, particularly
patents. Obtaining and enforcing patents in the biotechnology industry involve both technological and legal complexity, and therefore
obtaining and enforcing biotechnology patents is costly, time-consuming and inherently uncertain.
Recent patent reform legislation could increase the uncertainties and costs surrounding the prosecution of our patent applications
and the enforcement or defense of our 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 a number of significant changes to U.S. patent law. These include
provisions that affect the way patent applications are prosecuted and may also affect patent litigation. The United States Patent and
Trademark Office recently 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. 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, all of which could have a material adverse
effect on our business and financial condition.
Moreover, recent United States Supreme Court rulings have narrowed the scope of patent protection available in certain
circumstances and weakened the rights of patent owners in certain situations. In addition to increasing uncertainty with regard to our
ability to obtain patents in the future, this combination of events has created uncertainty with respect to the value of patents, once
obtained. Depending on decisions by the United States Congress, the federal courts, and the United States Patent and Trademark
Office, the laws and regulations governing patents could change in unpredictable ways that would weaken our ability to obtain new
patents or to enforce our existing patents and patents that we might obtain in the future.
We have not yet sought FDA approval of names for any of our product candidates and failure to secure such approvals could
adversely affect our business.
Any name we propose to use with our product candidates in the United States must be approved by the FDA, regardless of
whether we have registered it, or applied to register it, as a trademark. The FDA typically conducts a review of proposed product
names, including an evaluation of potential for confusion with other product names. If the FDA objects to any of our proposed
proprietary product names, we may be required to expend significant additional resources in an effort to identify a suitable substitute
name that would qualify under applicable trademark laws, not infringe the existing rights of third parties and be acceptable to the
FDA.
We may not be able to protect our intellectual property rights throughout the world.
Filing, prosecuting 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 can be less extensive than those in 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. Consequently, we may not be able to prevent third parties from practicing our inventions in all
countries outside the United States, or from selling or importing products made using our inventions in and into the United States or
other jurisdictions. Competitors may use our technologies in jurisdictions where we have not obtained patent protection to develop
their own products and further, may export otherwise infringing products to territories where we have patent protection, but
enforcement is not as strong as that in the United States. These products may compete with our products 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 protection, particularly those relating to biotechnology products, which could make it
difficult for us to stop the infringement of our patents or marketing of competing products in violation of our proprietary rights
generally. Proceedings to enforce our patent rights in foreign jurisdictions could result in substantial costs and divert our efforts and
attention from other aspects of our business, could put our patents at risk of being invalidated or interpreted narrowly and our patent
applications at risk of not issuing and could 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
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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.
Risks related to ownership of our common stock
The market price for our common stock may be volatile, which could contribute to the loss of your investment.
Fluctuations in the price of our common stock could contribute to the loss of all or part of your investment. The trading price of
our common stock is likely to continue to be highly volatile and could be subject to wide fluctuations in response to various factors,
some of which are beyond our control. Any of the factors listed below could have a material adverse effect on your investment in our
common stock. In such circumstances, the trading price of our common stock may not recover and may experience a further decline.
Factors affecting the trading price of our common stock may include:
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our failure to develop and commercialize our product candidates;
actual or anticipated fluctuations in our quarterly financial results or the quarterly financial results of companies perceived
to be similar to us;
changes in the market’s expectations about our operating results;
adverse results or delays in our preclinical studies or clinical trials;
reports of adverse events in other gene therapy products or clinical studies of such products;
our decision to initiate a clinical trial, not to initiate a clinical trial or to terminate an existing clinical trial;
any delay in filing an IND or BLA for any of our product candidates and any adverse development or perceived adverse
development with respect to the FDA’s review of that IND or BLA;
adverse regulatory decisions, including failure to receive regulatory approval for any of our product candidates;
success of competitive products;
adverse developments concerning our collaborations and our manufacturers;
inability to obtain adequate product supply for any product candidate for clinical trials or commercial sale or inability to
do so at acceptable prices;
the termination of a collaboration or the inability to establish additional collaborations;
unanticipated serious safety concerns related to the use of any of our product candidates;
our ability to effectively manage our growth;
the size and growth, if any, of the orphan ophthalmology and other targeted markets;
our operating results failing to meet the expectation of securities analysts or investors in a particular period or failure of
securities analysts to publish reports about us or our business;
changes in financial estimates and recommendations by securities analysts concerning our company, the gene therapy
market, or the biotechnology and pharmaceutical industries in general;
operating and stock price performance of other companies that investors deem comparable to us;
overall performance of the equity markets;
announcements by us or our competitors of acquisitions, new product candidates or programs, significant contracts,
commercial relationships or capital commitments;
our ability to successfully market our product candidates;
changes in laws and regulations affecting our business, including but not limited to clinical trial requirements for
approvals;
disputes or other developments relating to proprietary rights, including patents, litigation matters and our ability to obtain
patent protection for our product candidates and gene therapy platform;
commencement of, or involvement in, litigation involving our company, our general industry, or both;
58
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changes in our capital structure, such as future issuances of securities or the incurrence of additional debt;
the volume of shares of our common stock available for public sale;
additions or departures of key scientific or management personnel;
any major change in our board or management;
changes in accounting practices;
ineffectiveness of our internal control over financial reporting;
sales of substantial amounts of common stock by our directors, executive officers or significant stockholders or the
perception that such sales could occur; and
general economic and political conditions such as recessions, interest rates, fuel prices, international currency fluctuations
and acts of war or terrorism.
Broad market and industry factors may materially harm the market price of our common stock irrespective of our operating
performance. The stock market in general, and The NASDAQ Global Market and the market for biotechnology companies in
particular, have experienced extreme price and volume fluctuations that have often been unrelated or disproportionate to the operating
performance of the particular companies affected. The trading prices and valuations of these stocks, and of ours, may not be
predictable. A loss of investor confidence in the market for technology or software stocks or the stocks of other companies which
investors perceive to be similar to us, the opportunities in the digital simulation market or the stock market in general, could depress
our stock price regardless of our business, prospects, financial conditions or results of operations.
If securities analysts do not publish research or reports about our business or if they downgrade our stock, the price of our
common stock could decline.
The trading market for our common stock relies in part on the research and reports that industry or financial analysts publish
about us, our business, our markets and our competitors. We do not control these analysts. As a newly public company, we have only
limited coverage by securities analysts. If securities analysts do not continue to cover our common stock, the lack of research coverage
may adversely affect the market price of our common stock. Furthermore, if one or more of the analysts who do cover us downgrade
our stock or if those analysts issue other unfavorable commentary about us or our business, our stock price would likely decline. If one
or more of these analysts cease coverage of us or fails to regularly publish reports on us, we could lose visibility in the market and
interest in our stock could decrease, which in turn could cause our stock price or trading volume to decline and may also impair our
ability to expand our business with existing customers and attract new customers.
We are an “emerging growth company,” and the reduced reporting requirements applicable to emerging growth companies
may make our common stock less attractive to investors.
We are an “emerging growth company,” as defined in the Jumpstart Our Business Startups Act of 2012, or the JOBS Act. For as
long as we continue to be an emerging growth company, we may take advantage of exemptions from various reporting requirements
that are applicable to other public companies that are not emerging growth companies, including not being required to comply with the
auditor attestation requirements of Section 404 of the Sarbanes-Oxley Act t, reduced disclosure obligations regarding executive
compensation and exemptions from the requirements of holding a nonbinding advisory vote on executive compensation and
stockholder approval of any golden parachute payments not previously approved. We could be an emerging growth company through
June 30, 2019, which is the fifth anniversary of the end of the fiscal year in which our initial public offering closed. However,
circumstances could cause us to lose that status earlier, including if the market value of our common stock held by non-affiliates
exceeds $700.0 million as of any December 31 before that time or if we have total annual gross revenue of $1.0 billion or more during
any fiscal year before that time, in which cases we would no longer be an emerging growth company as of the following June 30 or, if
we issue more than $1.0 billion in non-convertible debt during any three-year period before that time, we would cease to be an
emerging growth company immediately. Even after we no longer qualify as an emerging growth company, we may still qualify as a
“smaller reporting company” which would allow us to take advantage of many of the same exemptions from disclosure requirements,
including not being required to comply with the auditor attestation requirements of Section 404 of the Sarbanes-Oxley Act and
reduced disclosure obligations regarding executive compensation. We cannot predict if investors will find our common stock less
attractive because we may rely on 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.
Under the JOBS Act, emerging growth companies can also delay adopting new or revised accounting standards until such time
as those standards apply to private companies. We have irrevocably elected not to avail ourselves of this exemption from new or
revised accounting standards and, therefore, will be subject to the same new or revised accounting standards as other public companies
that are not emerging growth companies. As a result, changes in rules of U.S. generally accepted accounting principles or their
59
interpretation, the adoption of new guidance or the application of existing guidance to changes in our business could significantly
affect our financial position and results of operations.
Future sales and issuances of our common stock or rights to purchase common stock, including pursuant to our equity
incentive plans, could result in additional dilution of the percentage ownership of our stockholders and could cause our stock price
to fall.
We expect that significant additional capital may be needed in the future to continue our planned operations, including
conducting clinical trials, commercialization efforts, expanded research and development activities, potential acquisitions, in-licenses,
or collaborations and costs associated with operating a public company. To raise capital, we may sell common stock, convertible
securities or other equity securities in one or more transactions at prices and in a manner we determine from time to time. If we sell
common stock, convertible securities or other equity securities in more than one transaction, investors may be materially diluted by
subsequent sales. Such sales may also result in material dilution to our existing stockholders, and new investors could gain rights,
preferences and privileges senior to the holders of our common stock.
We do not currently intend to pay dividends on our common stock and, consequently, your ability to achieve a return on your
investment will depend on the appreciation in the price of our common stock.
We have never declared or paid any cash dividends on our common stock. We currently intend to retain any future earnings to
fund our future growth and do not expect to declare or pay any dividend on shares of our common stock in the foreseeable future. As a
result, you may only receive a return on your investment in our common stock if the market price of our common stock appreciates
and you sell your shares at a price above your cost.
We could be subject to securities class action litigation.
In the past, securities class action litigation has often been brought against a company following a decline in the market price of
its securities. This risk is especially relevant for us because biotechnology companies have experienced significant stock price
volatility in recent years. If we face such litigation, it could result in substantial costs and a diversion of management’s attention and
resources, which could harm our business.
Anti-takeover provisions in our amended and restated certificate of incorporation and amended and restated bylaws, as well
as provisions in Delaware law, might discourage, delay or prevent a change of control of our company or changes in our
management and, therefore, depress the trading price of our common stock.
Our certificate of incorporation, bylaws and Delaware law contain provisions that could have the effect of rendering more
difficult or discouraging an acquisition deemed undesirable by our board of directors, even if doing so would benefit our stockholders
or remove our current management. Our corporate governance documents include provisions:
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providing for three classes of directors with the term of office of one class expiring each year, commonly referred to as a
staggered board;
authorizing blank check preferred stock, which could be issued with voting, liquidation, dividend and other rights superior
to our common stock;
limiting the liability of, and providing indemnification to, our directors and officers;
eliminating the ability of our stockholders to call and bring business before special meetings and to take action by written
consent in lieu of a meeting;
requiring advance notice of stockholder proposals for business to be conducted at meetings of our stockholders and for
nominations of candidates for election to our board of directors;
controlling the procedures for the conduct and scheduling of board and stockholder meetings;
limiting the determination of the number of directors on our board and the filling of vacancies or newly created seats on
the board to our board of directors then in office; and
providing that directors may be removed by stockholders only for cause.
These provisions, alone or together, could delay hostile takeovers and changes in control or changes in our management.
As a Delaware corporation, we are also subject to provisions of Delaware law, including Section 203 of the Delaware General
Corporation Law, which prohibits a publicly-held Delaware corporation from engaging in a business combination with an interested
60
stockholder, generally a person which together with its affiliates owns, or within the last three years has owned, 15% of our voting
stock, for a period of three years after the date of the transaction in which the person became an interested stockholder, unless the
business combination is approved in a prescribed manner.
The existence of the foregoing provisions and anti-takeover measures could limit the price that investors might be willing to pay
in the future for shares of our common stock. They could also deter potential acquirers of our company, thereby reducing the
likelihood that you could receive a premium for your common stock in an acquisition.
ITEM 1B. UNRESOLVED STAFF COMMENTS
None.
ITEM 2.
PROPERTIES
Alachua, Florida
Our corporate headquarters are located in Alachua, Florida. In January 2016, we moved into a new combined-use facility
consisting of approximately 21,000 square feet of laboratory and office space. The initial lease term for this facility is 10 years and
we have options to extend the term of the lease for three additional five-year periods. Our prior leased facilities encompassed
approximately 7,000 square feet of office and laboratory space. Operating leases associated with the prior facilities expired in
December 2015.
Cambridge, Massachusetts
In August 2015, we entered into a two-year lease to occupy approximately 3,000 square feet of office and laboratory space in
Cambridge, Massachusetts. On July 31, 2017, we entered into a new lease to increase our office and laboratory space in Cambridge by
approximately 5,000 square feet to a total of approximately 8,000 square feet and extend the term of the lease for an additional seven
years, with an option to further extend the lease for one additional three-year term. The Cambridge facility primarily focuses on
business development, pharmacology, and basic research and development.
ITEM 3.
LEGAL PROCEEDINGS
We are not a party to any pending legal proceedings.
ITEM 4. MINE SAFETY DISCLOSURES
Not applicable.
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 listed on The NASDAQ Global Market under the symbol “AGTC” since March 27, 2014. Prior to
that date, there was no public market for our common stock. The following table sets forth, for the periods indicated, the high and low
intraday sales prices of our common stock as reported by The NASDAQ Global Market:
First fiscal quarter
Second fiscal quarter
Third fiscal quarter
Fourth fiscal quarter
2017
2016
High
Low
High
Low
$
$
$
$
$
17.00
10.85
$
10.15 $
7.05 $
$
8.50
6.35
$
6.08 $
4.70 $
$
20.10
21.43
$
20.13 $
19.86 $
12.43
12.04
12.12
12.76
As of August 31, 2017, a total of 18,087,785 shares of our common stock were outstanding and we had 35 holders of record of
our common stock.
61
Dividend Policy
We have never declared or paid any cash dividends on our capital stock. We currently intend to retain all available funds and
future earnings, if any, to finance the growth and development of our business. We do not expect to pay any cash dividends on our
common stock in the foreseeable future.
Securities Authorized for Issuance Under Equity Compensation Plans
For information regarding securities authorized for issuance under our equity compensation plans, see Part III, Item 12,
“Security Ownership of Certain Beneficial Owners and Management and Related Stockholder Matters.”
62
Comparative Stock Performance
The following stock performance graph compares the cumulative total return to stockholders for our common stock for the
period commencing March 27, 2014 (the date on which our common stock commenced trading on The NASDAQ Global Market) and
ended June 30, 2017 against the cumulative total return of the NASDAQ Composite Index and the NASDAQ Biotechnology Index.
The calculation of total cumulative returns assumes a $100 investment in our common stock, the NASDAQ Composite Index and the
NASDAQ Biotechnology Index, and assumes reinvestment of all dividends, if any. The historical information set forth below is not
necessarily indicative of future performance.
COMPARISON OF 39 YEAR CUMULATIVE TOTAL RETURN*
Among Applied Genetic Technologies Corporation, the NASDAQ Composite Index
and the NASDAQ Biotechnology Index
$180
$160
$140
$120
$100
$80
$60
$40
$20
$0
3/27/14
6/30/14
6/30/15
6/30/16
6/30/17
Applied Genetic Technologies Corporation
NASDAQ Composite
NASDAQ Biotechnology
*$100 invested on 3/27/14 in stock or 3/31/14 in index, including reinvestment of dividends.
Fiscal year ending June 30.
Applied Genetic Technologies Corporation
NASDAQ Composite
NASDAQ Biotechnology
3/14
100.00
100.00
100.00
6/14
156.50
105.23
109.25
6/15
103.93
119.93
149.07
6/16
95.73
117.34
107.52
6/17
34.55
149.68
124.72
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ITEM 6:
SELECTED FINANCIAL DATA
The following selected financial data should be read in conjunction with our financial statements and related notes in Part II,
Item 8 of this Annual Report on Form 10-K and with our “Management’s Discussion and Analysis of Financial Condition and Results
of Operations” in Part II, Item 7 of this Annual Report on Form 10-K.
Our selected statement of operations data for the fiscal years ended June 30, 2017, 2016 and 2015 and our selected balance sheet
data as of June 30, 2017 and 2016 are derived from our audited financial statements included elsewhere in this report. The selected
statements of operations data for the fiscal years ended June 30, 2014 and 2013 and the balance sheet data as of June 30, 2015, 2014
and 2013 are derived from our audited financial statements not included in this Annual Report on Form 10-K. Our historical results
are not necessarily indicative of results to be expected for any future period. The selected financial data in this section are not intended
to replace our financial statements and the related notes.
Selected Financial Data
Statement of Operations Data:
Revenue:
Collaboration Revenue
Grant revenue
Sponsored research and other revenue
Total revenue
Operating expenses:
Research and development
General and administrative
Total operating expenses
Income (loss) from operations
Other income (expense):
Investment income, net
Interest expense
Fair value adjustments to warrant liabilities
Fair value adjustments to Series B purchase
rights
Other
Total other income (expense), net
Income (loss) before provision for income taxes
Provision for income taxes
Net income (loss)
Net income (loss) per share, basic (1)
Net income (loss) per share, diluted (1)
Weighted-average shares outstanding, basic
Weighted-average shares outstanding, diluted
2017
39,282 $
191
-
39,473
26,217
11,354
37,571
1,902
952
—
—
—
(47)
905
2,807
2,400
407 $
0.02 $
0.02 $
18,072
18,385
$
$
$
$
2016
Fiscal Year Ended June 30,
2015
(in thousands except per share data)
2014
46,751 $
610
-
47,361
39,376
10,074
49,450
(2,089)
711
—
—
—
(3)
708
(1,381)
—
(1,381) $
(0.08) $
(0.08) $
17,810
17,810
$
—
1,682
672
2,354
— $
917
212
1,129
18,118
8,768
26,886
(24,532)
8,503
5,182
13,685
(12,556)
216
—
—
—
(2)
214
(24,318)
—
(24,318) $
(1.50) $
(1.50) $
16,253
16,253
42
—
(441)
(2,904)
(49)
(3,352)
(15,908)
—
(15,908) $
(4.46) $
(4.46) $
3,568
3,568
2013
—
439
503
942
3,133
1,403
4,536
(3,594)
10
(191)
(8)
(1,207)
—
(1,396)
(4,990)
—
(4,990)
(45.78)
(45.78)
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Balance Sheet Data:
Cash and cash equivalents
Short and long-term investments
Total assets
Current liabilities
Convertible preferred stock
Total stockholders’ equity (deficit)
2017
2016
As of June 30,
2015
(in thousands)
2014
2013
$
$
$
$
$
$
30,706 $
107,743 $
147,923 $
28,156 $
— $
115,329 $
28,868 $
143,847 $
180,797 $
54,743 $
— $
109,288 $
39,187
46,083
90,174
4,642
—
85,532
$
$
$
$
$
$
8,623 $
64,450 $
77,407 $
2,534 $
— $
74,873 $
8,893
14,000
25,490
3,460
58,103
(36,183)
(1)
See Note 2 to the AGTC Financial Statements for a description of the method used to calculate basic and diluted net loss per share.
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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
the financial statements and notes included in Part IV, Item 15 of this Annual Report on Form 10-K. In addition to historical financial
information, the following discussion contains forward-looking statements that reflect our plans, estimates and beliefs. Our actual
results could differ materially from those discussed in the forward-looking statements. Factors that could cause or contribute to these
differences include those discussed below and elsewhere in this Annual Report, including but not limited to those set forth in
“Cautionary Note Regarding Forward-Looking Statements” and “Risk Factors.”
Overview
We are a clinical-stage biotechnology company that uses a proprietary gene therapy platform to develop transformational
genetic therapies for patients suffering from rare and debilitating diseases. Our initial focus is in the field of ophthalmology, where we
have active clinical programs in X-linked retinoschisis (XLRS), X-linked retinitis pigmentosa (XLRP), and achromatopsia (ACHM)
and a preclinical program in optogenetics. In addition to ophthalmology, we have recently initiated preclinical programs in
adrenoleukodystrophy (ALD) and otology. With a number of important clinical milestones on the horizon, we believe we are well
positioned to advance multiple programs towards pivotal studies. In addition to our product pipeline, we have also developed broad
technological capabilities through our collaborations with 4D Molecular Therapeutics (4DMT), Synpromics Limited (Synpromics),
and the University of Florida, which provide us with expertise in vector design and manufacturing as well as synthetic promoter
development and optimization. Finally, our partnership with Biogen, which includes our clinical XLRS and XLRP programs, a
discovery program in ALD and two ophthalmology programs, validates our approach and technology, and provides us with a
significant cash runway to advance our wholly-owned candidates.
Since our inception in 1999, we have devoted substantially all of our resources to development efforts relating to our proof-of-
concept programs in ophthalmology and alpha-1 antitrypsin deficiency, or AAT deficiency, an inherited orphan lung disease,
including activities to manufacture product in compliance with good manufacturing practices, preparing to conduct and conducting
clinical trials of our product candidates, providing general and administrative support for these operations and protecting our
intellectual property. We do not have any products approved for sale and have not generated any revenue from product sales. To date,
we have funded our operations primarily through the private placement of preferred stock, common stock, convertible notes and
warrants to purchase preferred stock, through our public offerings consummated in April 2014 and July/August 2014, and through
upfront and milestone payments from our partners. We have also been the recipient, either independently or with our collaborators, of
grant funding administered through federal, state, and local governments and agencies, including the United States Food and Drug
Administration, or FDA, and by patient advocacy groups such as The Foundation Fighting Blindness, or FFB, and the Alpha-1
Foundation.
Although we recorded income from operations of $1.9 million for the year ended June 30, 2017 due in part to the amortization
of revenue associated with our collaboration agreement with Biogen, we have incurred losses from operations in each other year since
inception. Our net income for the fiscal year ended June 30, 2017 was $0.4 million, compared to net losses of $1.4 million and $24.3
million for each of the fiscal years ended June 30, 2016 and 2015, respectively. Substantially all our net losses resulted from costs
incurred in connection with our research and development programs and from general and administrative costs associated with our
operations. We expect to continue to incur significant operating expenses for at least the next several years and anticipate that such
expenses will increase substantially in connection with our ongoing activities, as we:
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conduct preclinical studies and clinical trials for our XLRS, ACHM and XLRP product candidates;
continue our research and development efforts, including exploration through early preclinical studies of potential
applications of our gene therapy platform in::
o
o
o
orphan ophthalmology indications;
non-orphan ophthalmology indications including wet AMD and other inherited retinal diseases; and
other inherited diseases, such as otology and CNS indications.
manufacture clinical trial materials and develop larger-scale manufacturing capabilities;
seek regulatory approval for our product candidates;
further develop our gene therapy platform;
add personnel to support our collaboration, product development and commercialization efforts; and
continue to operate as a public company.
65
As of June 30, 2017, we had cash and cash equivalents and investments totaling $138.4 million.
We do not expect to generate revenue from product sales unless and until we successfully complete development and obtain
regulatory approval for one or more of our product candidates, which we expect will take a number of years and which we believe is
subject to significant uncertainty. We believe that our existing cash and cash equivalents and investments at June 30, 2017, will be will
be sufficient to allow us to generate data from our ongoing clinical programs, to move our pre-clinical optogenetic program in
collaboration with Bionic Sight into the clinic and to fund our currently planned research and discovery programs for at least the next
two years. In order to complete the process of obtaining regulatory approval for our lead product candidates and to build the sales,
marketing and distribution infrastructure that we believe will be necessary to commercialize our lead product candidates, if approved,
we will require substantial additional funding. Also, our current operating plan may change as a result of many factors currently
unknown to us, and we may need to seek additional funds sooner than planned, through public or private equity or debt financings,
government or other third-party funding, marketing and distribution arrangements and other collaborations, strategic alliances and
licensing arrangements or a combination of these approaches. However, we may be unable to raise additional funds or enter into such
other arrangements when needed on favorable terms or at all. Our failure to raise capital or enter into such other arrangements as and
when needed would have a negative impact on our financial condition and our ability to develop our products.
Strategic Collaborations
Biogen
In July 2015, we entered into a collaboration agreement with Biogen, which we refer to as the collaboration agreement, pursuant
to which we and Biogen will collaborate to develop, seek regulatory approval for and commercialize gene therapy products to treat
XLRS, XLRP, and discovery programs targeting three indications based on our adeno-associated virus vector technologies. The
Collaboration Agreement became effective on August 14, 2015.Under the collaboration agreement, we will conduct all development
activities through regulatory approval in the United States for the XLRS program (with activities through Phase 1/2 completion being
pre-funded under the agreement and any further activities subject to incremental consideration), and all development activities through
the completion of the first in human clinical trial for the XLRP program (with activities through filing the IND being pre-funded under
the agreement and any further activities subject to incremental consideration). In addition, the collaboration agreement provides for
discovery programs targeting three indications whereby we will conduct discovery, research and development activities for those
additional drug candidates through the stage of clinical candidate designation, after which, Biogen may exercise an option to continue
to develop, seek regulatory approval for and commercialize the designated clinical candidate. In February 2016, we announced
Biogen’s selection of adrenoleukodystrophy as the non-ophthalmic indication of the discovery programs. Under the terms of the
collaboration agreement, we, in part through our participation in joint committees with Biogen, will participate in overseeing the
development and commercialization of these specific programs.
Under the collaboration agreement, we received a non-refundable upfront payment of $94.0 million in August 2015. As a result
of the upfront payment made by Biogen, we became liable to our various research partner institutions for sub-license payments, which
led us to record an expense for collaboration-related license fees of $12.0 million. We paid these license fees in full during the fiscal
year ended June 30, 2016.
During 2017 and 2016, we recognized revenue of approximately $39.3 million and $46.8 million, respectively from our
collaboration with Biogen. We are also eligible to receive payments of up to $467.5 million based on the successful achievement of
future milestones under the two lead programs and up to $592.5 million based on the exercise of the option for and the successful
achievement of future milestones under the three discovery programs. Biogen will pay revenue-based royalties for each licensed
product at tiered rates ranging from high single digit to mid-teen percentages of annual net sales of the XLRS or XLRP products and
at rates ranging from mid-single digit to low-teen percentages of annual net sales for the discovery products. Due to the uncertainty
surrounding the achievement of the future milestones, such payments were not considered fixed or determinable at the inception of the
collaboration agreement and accordingly, will not be recognized as revenue unless and until they become earned. We achieved the
first milestone under the XLRS program in late August 2015, which triggered a milestone payment from Biogen of $5.0 million and
the recording of milestone revenue. We are not able to reasonably predict if and when any of the remaining milestones will be
achieved.
In addition to the collaboration agreement, on July 1, 2015, we also entered into an equity agreement with Biogen. Under the
terms of the equity agreement, Biogen purchased 1,453,957 shares of our common stock, at a purchase price equal to $20.63 per share,
for an aggregate cash purchase price of $30.0 million. We received these cash proceeds from Biogen in August 2015. The shares
issued to Biogen constitute restricted securities that may not be resold by Biogen other than in a transaction registered under the
Securities Act of 1933, as amended, or pursuant to an exemption from such registration requirement.
Bionic Sight, LLC
In February 2017, we entered into a collaboration agreement with Bionic Sight to develop a gene therapy treatment to be
used with Bionic Sight’s innovative neuroprosthetic device and algorithm for retinal coding. Under the agreement, AGTC made an
initial $2.0 million payment to Bionic Sight for an equity interest in that company. This initial investment represents an approximate 5
66
percent equity interest in Bionic Sight. In addition to the initial investment, we will contribute to ongoing research and development
support costs through additional payments or other in-kind contributions. These payments and contributions will be made over time,
up to the date that Bionic Sight has received both investigational new drug clearance from the FDA and receipt of written approval
from an internal review board to conduct clinical trials from at least one clinical site for that product (the “IND Trigger”.) If the IND
Trigger is attained, we will receive additional equity, based on the valuation in place at the beginning of the agreement, for its cash
and research and development contributions and will be obligated to purchase additional equity in Bionic Sight for $4.0 million upon
the filing of the IND for the product at a pre-determined valuation.
Synpromics
In December 2015, we entered into an agreement with Synpromics Limited (“Synpromics”) to utilize Synpromics' proprietary
technology to develop and optimize synthetic promoters. In the event that results from the research are promising, we have options to
license promoters that result from the collaboration efforts.
4DMT
In April 2015, we signed an agreement with 4DMT to conduct research on optimized next generation capsids to target specific
target cell populations within the human retina using 4DMT’s Directed Evolution AAV vector discovery platform. If promising
capsids result from the research, we have an option to enter into a licensing agreement.
Financial operations overview
Revenue
Our ability to generate product revenue and become profitable depends upon our ability to successfully commercialize products.
To date, we have not generated any revenues from the sales of products. During the fiscal years ended June 30, 2017 and 2016, we
recognized $39.3 million and $46.8 million of revenue from our collaboration with Biogen, respectively. In the fiscal year ended
June 30, 2015, all our revenues were derived from grants and sponsored research arrangements. Revenues from grants and sponsored
research arrangements are recognized when there is reasonable assurance that they will be received and we have complied with the
terms of the grant or the sponsored research arrangement.
Research and development expenses
Research and development expenses consist primarily of costs incurred for the development of our product candidates, which
include:
(cid:129)
(cid:129)
(cid:129)
(cid:129)
(cid:129)
employee-related expenses, including salaries, benefits, travel and share-based compensation expense;
expenses incurred under agreements with academic research centers, contract research organizations, or CROs, and
investigative sites that conduct our clinical trials;
license and sublicense fees and collaboration expenses;
the cost of acquiring, developing, and manufacturing clinical trial materials; and
facilities, depreciation, and other expenses, which include direct and allocated expenses for rent and maintenance of
facilities, insurance, and other supplies.
Research and development costs are expensed as incurred. Costs for certain development activities are recognized based on an
evaluation of the progress to completion of specific tasks using information and data provided to us by our vendors and our clinical
sites.
67
We cannot determine with certainty the duration and completion costs of the current or future clinical trials of our product
candidates or if, when, or to what extent we will generate revenues from the commercialization and sale of any of our product
candidates that obtain regulatory approval. We may never succeed in achieving regulatory 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:
(cid:129)
(cid:129)
(cid:129)
(cid:129)
(cid:129)
(cid:129)
(cid:129)
(cid:129)
(cid:129)
(cid:129)
(cid:129)
the scope, rate of progress, and expense of our ongoing as well as any additional clinical trials and other research and
development activities;
the timing and level of activity as determined by us or jointly with our partners;
the level of funding received from our partners;
whether or not we elect to cost share with our partners;
the countries in which trials are conducted;
future clinical trial results;
uncertainties in clinical trial enrollment rates or drop-out or discontinuation rates of patients;
potential additional safety monitoring or other studies requested by regulatory agencies or elected as best practice by us;
increased cost and delay associated with manufacturing or testing issues, including ongoing quality assurance, qualifying
new vendors and developing in-house capabilities;
significant and changing government regulation; and
the timing and receipt of any regulatory approvals.
A change in the outcome of any of these variables with respect to the development of a product candidate could mean a
significant change in the costs and timing associated with the development of that product candidate. For example, if the FDA, or
another regulatory authority were to require us to conduct clinical trials beyond those that we currently anticipate will be required for
the completion of clinical development of a product candidate or if we experience significant delays in enrollment in or execution of
any of our clinical trials, we could be required to expend significant additional financial resources and time on the completion of
clinical development.
As of June 30, 2017, we had a total of 53 research and development personnel. From inception through June 30, 2017, we
have incurred approximately $137.0 million in research and development expenses. We expect our research and development
expenses to increase for the foreseeable future as we continue the development of our product candidates and explore potential
applications of our gene therapy platform in other indications.
General and administrative expenses
General and administrative expenses consist primarily of salaries and related costs for personnel, including share-based
compensation and travel expenses for our employees in executive, operational, legal, business development, finance and human
resource functions. Other general and administrative expenses include costs to support employee training and development, board of
directors costs, depreciation, insurance expenses, facility-related costs not otherwise included in research and development expense,
professional fees for legal services, including patent-related expenses, and accounting, investor relations, corporate communications
and information technology services. We anticipate that our general and administrative expenses will continue to increase in the future
as we hire additional employees to support our continued research and development efforts, collaboration arrangements, and the
potential commercialization of our product candidates. Additionally, if and when we believe a regulatory approval of the first product
candidate appears likely, we anticipate an increase in payroll and related expenses as a result of our preparation for commercial
operations, especially as it relates to the sales and marketing of our product candidates.
Other income (expense), net
Other income and expense consists primarily of interest earned on cash and cash equivalents and our held-to-maturity
investments.
68
Critical accounting policies and estimates
The following discussion of critical accounting policies identifies the accounting policies that require application of
management’s most difficult, subjective or complex judgments, often as a result of the need to make estimates about the effect of
matters that are inherently uncertain and may change in subsequent periods. It is not intended to be a comprehensive list of all of our
significant accounting policies, which are more fully described in Note 2 of the notes to the financial statements appearing elsewhere
in this annual report on Form 10-K. In many cases, the accounting treatment of a particular transaction is specifically dictated by
generally accepted accounting principles, with no need for management’s judgment in their application. There are also areas in which
the selection of an available alternative policy would not produce a materially different result.
Revenue recognition
We have generated revenue primarily through sponsored research arrangements with nonprofit organizations for the
development and commercialization of product candidates and revenues from federal research and development grant programs. We
recognize revenue when amounts are realized or realizable and earned. Revenue is considered realizable and earned when the
following criteria are met: (1) persuasive evidence of an arrangement exists; (2) delivery has occurred or services have been rendered;
(3) the price is fixed or determinable; and (4) collection of the amounts due are reasonably assured.
We evaluate the terms of sponsored research agreement grants and federal grants to assess our obligations and if our obligations
are satisfied by the passage of time, revenue is recognized on a straight-line basis. In situations where the performance of our
obligations has been satisfied when the grant is received, revenue is recognized upon receipt of the grant. Certain grants contain refund
provisions. We review those refund provisions to determine the likelihood of repayment. If the likelihood of repayment of the grant is
determined to be remote, the grant is recognized as revenue. If the probability of repayment is determined to be more than remote, we
record the grant as a deferred revenue liability, until such time that the grant requirements have been satisfied.
Collaboration revenue
As described above, on July 1, 2015, we entered into a collaboration agreement with Biogen. The terms of this agreement and
other potential collaboration or commercialization agreements we may enter into generally contain multiple elements, or deliverables,
which may include, among others, (i) licenses, or options to obtain licenses, to our technology, and (ii) research and development
activities to be performed on behalf of the collaborative partner. Payments made under such arrangements typically include one or
more of the following: non-refundable, up-front license fees; option exercise fees; funding of research and/or development efforts;
milestone payments; and royalties on future product sales.
Multiple element arrangements are analyzed to determine whether the deliverables within the agreement can be separated or
whether they must be accounted for as a single unit of accounting.
Deliverables under an agreement are required to be accounted for as separate units of accounting provided that (i) a delivered
item has value to the customer on a stand-alone basis; and (ii) if the agreement includes a general right of return relative to the
delivered item, delivery or performance of the undelivered item is considered probable and substantially in the control of the vendor.
The allocation of consideration amongst the deliverables under the agreement is derived using a “best estimate of selling price” if
vendor specific objective evidence and third party evidence of fair value is not available. If the delivered element does not have stand-
alone value or if the fair value of any of the undelivered elements cannot be determined, the arrangement is then accounted for as a
single unit of accounting, and we recognize the consideration received under the arrangement as revenue on a straight-line basis over
our estimated period of performance.
We determine the estimated selling price for deliverables within each agreement using vendor-specific objective evidence, or
VSOE, of selling price, if available, third-party evidence, or TPE, of selling price if VSOE is not available, or best estimate of selling
price, or BESP, if neither VSOE nor TPE are available. Determining the best estimate of selling price for a deliverable requires
significant judgment. We use BESP to estimate the selling price related to licenses to our proprietary technology, since we often do
not have VSOE or TPE of selling price for these deliverables. In those circumstances where we utilize BESP to determine the
estimated selling price of a license to our proprietary technology, we consider market conditions as well as entity-specific factors,
including those factors contemplated in negotiating the agreements as well as internally developed models that include assumptions
related to the market opportunity, estimated development costs, probability of success and the time needed to commercialize a product
candidate pursuant to the license. In validating our best estimate of selling price, we evaluate whether changes in the key assumptions
used to determine the best estimate of selling price will have a significant effect on the allocation of arrangement consideration among
multiple deliverables.
If the delivered element does not have stand-alone value or if the fair value of any of the undelivered elements cannot be
determined, the arrangement is then accounted for as a single unit of accounting, and we recognize the consideration received under
the arrangement as revenue on a straight-line basis over our estimated period of performance. Our anticipated periods of performance,
typically the terms of our research and development obligations, are subject to estimates by management and may change over the
69
course of the collaboration agreement. Such changes could have a material impact on the amount of revenue we record in future
periods.
Milestone revenue
We apply the milestone method of accounting to recognize revenue from milestone payments when earned, as evidenced by
written acknowledgement from the collaborator or other persuasive evidence that the milestone has been achieved and the payment is
non-refundable, provided that the milestone event is substantive. A milestone event is defined as an event (i) that can only be
achieved based in whole or in part on either our performance or on the occurrence of a specific outcome resulting from our
performance; (ii) for which there is substantive uncertainty at the inception of the arrangement that the event will be achieved; and (iii)
that would result in additional payments being due to us. Events for which the occurrence is either contingent solely upon the passage
of time or the result of a counterparty’s performance are not considered to be milestone events. A milestone event is substantive if all
of the following conditions are met: (i) the consideration is commensurate with either our performance to achieve the milestone, or the
enhancement of the value to the delivered item(s) as a result of a specific outcome resulting from our performance to achieve the
milestone; (ii) the consideration relates solely to past performance; and (iii) the consideration is reasonable relative to all the
deliverables and payment terms (including other potential milestone consideration) within the arrangement.
We assess whether a milestone is substantive at the inception of the arrangement. If a milestone is deemed non-substantive, we
account for that milestone payment in accordance with the multiple element arrangements guidance and recognize revenue consistent
with the related units of accounting for the arrangement over the related performance period.
Research and development expenses
Research and development costs include costs incurred in identifying, developing and testing product candidates and generally
comprise compensation and related benefits and non-cash share-based compensation to research related employees; laboratory costs;
animal and laboratory maintenance and supplies; rent; utilities; clinical and pre-clinical expenses; and payments for sponsored
research, scientific and regulatory consulting fees and testing.
As part of the process of preparing our financial statements, we are required to estimate our accrued expenses. This process
involves reviewing quotations and contracts, identifying services that have been performed on our behalf and estimating the level of
service performed and the associated cost incurred for the service when we have not yet been invoiced or otherwise notified of the
actual cost. The majority of our service providers invoice us monthly in arrears for services performed or when contractual milestones
are met. We make estimates of our accrued expenses as of each balance sheet date in our financial statements based on facts and
circumstances known to us at that time. The significant estimates in our accrued research and development expenses are related to
expenses incurred with respect to academic research centers, CROs, and other vendors in connection with research and development
activities for which we have not yet been invoiced.
There may be instances in which our service providers require advance payments at the inception of a contract or in which
payments made to these vendors will exceed the level of services provided, resulting in a prepayment of the research and development
expense. Such prepayments are charged to research and development expense as and when the service is provided or when a specific
milestone outlined in the contract is reached.
Share-based compensation
We account for share-based awards issued to employees in accordance with Accounting Standard Codification (“ASC”) Topic
718, Compensation—Stock Compensation (“ASC 718”) generally recognize share-based compensation expense on a straight-line basis
over the periods during which the employees and non-employee directors are required to provide service in exchange for the award.
In addition, we issue stock options and restricted shares of common stock to non-employees in exchange for consulting services and
account for these in accordance with the provisions of ASC Subtopic 505-50, Equity-Based Payments to Non-employees (“ASC 505-
50”). Under ASC 505-50, share-based awards to non-employees are subject to periodic fair value re-measurement over their vesting
terms.
For purposes of calculating stock-based compensation, we estimate the fair value of stock options using a Black-Scholes option-
pricing model. The determination of the fair value of share-based payment awards utilizing the Black-Scholes model is affected by our
stock price and a number of assumptions, including expected volatility, expected life, risk-free interest rate and expected dividends.
The expected volatility is primarily based on the historical volatility of peer company data while the expected life of the stock options
is based on historical and other economic data trended into the future. The risk-free interest rate assumption is based on observed
interest rates appropriate for the expected terms of our stock options. The dividend yield assumption is based on our history and
expectation of no dividend payouts. If factors change and we employ different assumptions, stock-based compensation expense may
differ significantly from what we have recorded in the past. If there is a difference between the assumptions used in determining stock-
based compensation expense and the actual factors which become known over time, specifically with respect to anticipated forfeitures,
70
we may change the input factors used in determining stock-based compensation costs for future grants. These changes, if any, may
materially impact our results of operations in the period such changes are made.
Income taxes
We use the asset and liability method for accounting for income taxes. Under this method, deferred tax assets and liabilities are
recognized for the estimated future tax consequences attributable to differences between the financial statement carrying amounts of
existing assets and liabilities and their respective income tax bases. Deferred tax assets and liabilities are measured using enacted rates
expected to apply to taxable income in the years in which those temporary differences are expected to be recovered or settled.
As required by U.S. GAAP, we recognize the financial statement benefit of a tax position only after determining that the
relevant tax authority would more likely than not sustain the position following an audit. For tax positions meeting the more-likely-
than-not threshold, the amount recognized in the financial statements is the largest benefit that has a greater than 50 percent likelihood
of being realized upon ultimate settlement with the relevant tax authority. Any interest and penalties related to uncertain tax postions
will be reflected in income tax expense. The Company is subject to examination of its income tax returns in the federal and state
income tax jurisdictions in which it operates. On December 28, 2015, the United States Internal Revenue Service, or IRS, notified the
Company of an income tax audit for the tax period ending June 30, 2014. As of June 30, 2017, the IRS audit was closed and the
Company incurred no penalties or payment liabilities for its income tax positions.
For the fiscal year ended June 30, 2017, the Company recorded an income tax provision, related to the Company’s Federal
alternative minimum taxable income (“AMTI”) and state income tax in multiple states where the Company is doing business. The
Company calculates its AMTI using the alternative minimum tax (“AMT”) system. The Company’s federal income tax liability is the
greater of the tax computed using the regular tax system or the tax under the AMT system. Corporations are exempt from AMT for all
prior years in which their annual gross receipts for the 3-year period ending before the current tax year did not exceed $7.5
million. For the fiscal year ending June 30, 2017, the Company no longer qualifies for the small company exclusion. The AMT system
limits the use of net operating losses used by taxpayers to offset taxable income.
Recent Accounting Pronouncements
In May 2017, the FASB issued Accounting Standards Update (“ASU”) No. 2017-09, Scope of Modification Accounting, which
amends ASC Topic 718, Compensation – Stock Compensation. The amendments in this Update provide guidance about which
changes to the terms or conditions of a share-based payment award require an entity to apply modification accounting in Topic
718. The amendments are effective for fiscal years beginning after December 15, 2017, and interim periods within those fiscal years
and early adoption is permitted. We are currently in the process of evaluating the impact of adoption of this standard on our financial
statements.
In March 2016, the FASB issued ASU No. 2016-09, Improvements to Employee Share-Based Payment Accounting, which
amends ASC Topic 718, Compensation – Stock Compensation. The amendments simplify several aspects of the accounting for share-
based payment transactions, including the income tax consequences, classification of awards as either equity or liabilities, forfeitures,
and classification on the statement of cash flows. The amendments are effective for fiscal years beginning after December 15, 2016,
and interim periods within those fiscal years and early adoption is permitted. We have adopted this standard for our 2018 fiscal year
and do not expect it to have a material effect on our balance sheets, results of operations or cash flows.
In February 2016, the FASB issued ASU No. 2016-02, Leases (Topic 842) in order to increase transparency and comparability
among organizations by recognizing lease assets and lease liabilities on the balance sheet for those leases previously classified as
operating leases under GAAP. The standard requires, in most instances, a lessee to recognize on its balance sheet a liability to make
lease payments (the lease liability) and also a right-of-use asset representing its right to use the underlying asset for the lease
term. The amendments are effective for fiscal years beginning after December 15, 2018, including interim periods within those
periods, using a modified retrospective approach and early adoption is permitted. We are currently in the process of evaluating the
impact of adoption of this standard on our financial statements.
In May 2014, the FASB issued ASU No. 2014-09, Revenue from Contracts with Customers (Topic 606) which replaces the
existing accounting standards for revenue recognition with a single comprehensive five-step model. The core principle is to recognize
revenue upon the transfer of goods or services to customers at an amount that reflects the consideration expected to be received. It also
requires enhanced disclosures about revenue, provides guidance for transactions that were not previously addressed comprehensively,
and improves guidance for multiple-element arrangements. The guidance applies to any entity that either enters into contracts with
customers to transfer goods or services or enters into contracts for the transfer of nonfinancial assets unless those contracts are within
the scope of other standards. Since its issuance, the FASB has amended several aspects of the new guidance, including provisions that
address revenue recognition associated with the licensing of intellectual property. In July 2015, the FASB delayed the effective date of
this guidance by one year. The guidance is now effective for public companies for annual periods beginning after December 15, 2017
as well as interim periods within those annual periods using either the full retrospective approach or modified retrospective approach.
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We are working through an adoption plan which will include a review of collaboration agreements, applying the five-step model of
the new standard and comparing results to our current accounting. As part of this, we are evaluating the method of adoption and
assessing changes that might be necessary to our processes, internal controls and address changes in financial reporting. Because of
the nature of the work that remains, at this time we are unable to reasonably estimate the impact of adoption on our financial
statements.
Emerging growth company status
The JOBS Act permits an “emerging growth company” such as ours to take advantage of an extended transition period to
comply with new or revised accounting standards applicable to public companies. We have chosen to “opt out” of this provision and,
as a result, we will comply with new or revised accounting standards as required when they are adopted. This decision to opt out of the
extended transition period under the JOBS Act is irrevocable.
72
Results of operations
Comparison of the fiscal years ended June 30, 2017 and 2016
Revenue
Fiscal year ended June 30,
Collaboration revenue
Grant revenue
Total revenue
2017
$
$
39,282 $
191
39,473 $
2016
(dollars in thousands)
46,751 $
610
47,361 $
(7,469)
(419)
(7,888)
(16)%
(69)%
(17)%
Increase
(Decrease)
% Increase
(Decrease)
Total revenue for fiscal year 2017 decreased by $7.9 million to $39.5 million compared to fiscal year 2016. This decrease is
primarily due to recognizing $5.0 million of milestone revenue in fiscal year 2016 associated with achieving a XLRS patient
enrollment milestone under our collaboration arrangement with Biogen, and to a lesser extent due to reduced revenue associated with
the amortization of upfront fees associated with the Biogen collaboration due to certain delays which have extended our estimate of
period of performance and reduced research and development activities under grant-funded projects.
Research and development expenses
Research and development expenses for fiscal year 2017 decreased by $13.2 million to $26.2 million compared to fiscal year
2016. This decrease was primarily driven by a $12.0 million reduction in sublicense expenses associated with our collaboration
arrangement with Biogen on the XLRS and XLRP programs and a $3M reduction in licensing and milestones fees paid to 4D
Molecular Therapeutics and Synpromics Limited associated with our research and discovery programs. These decreases were
partially offset by increased employee-related expenses related to the hiring of additional employees to support clinical trial execution
and research and development activities.
The following table summarizes our research and development expenses by product candidate or program for the fiscal year
ended June 30, 2017 and 2016:
External research and development expenses
ACHM
XLRS
XLRP
Research and discovery programs
Total external research and development expenses
Internal research and development expenses
Employee-related costs
Share-based compensation
Other
Total internal research and development expenses
Total research and development expense
Fiscal Year Ended June 30,
Increase
% Increase
2017
2016
(dollars in thousands)
(Decrease)
(Decrease)
$
$
3,699 $
2,062
1,788
3,677
11,226
7,808
2,546
4,637
14,991
26,217 $
3,648 $
9,237
8,604
6,911
28,400
5,631
2,147
3,198
10,976
39,376 $
51
(7,175)
(6,816)
(3,234)
(17,174)
2,177
399
1,439
4,015
(13,159)
1%
(78)%
(79)%
(47)%
(202)%
39%
19%
45%
37%
(33)%
In fiscal year 2016, we began tracking external spending for our most advanced product candidates. External research and
development costs consist of collaboration, licensing, manufacturing, testing, and other miscellaneous expenses that are directly
attributable to our most advanced product candidates and discovery programs. We do not allocate personnel-related costs, including
stock-based compensation, costs associated with broad technology platform improvements or other indirect costs, to specific
programs, as they are deployed across multiple projects under development and, as such, are separately classified as internal research
and development expenses in the table above.
73
General and administrative expenses
Fiscal Year Ended June 30,
2017
2016
(dollars in thousands)
Increase
(Decrease)
% Increase
(Decrease)
Employee-related costs
Share-based compensation
Legal and professional fees
Other general and administrative expenses
Total general and administrative expenses
$
$
3,952 $
3,060
1,058
3,284
11,354 $
2,827 $
2,860
1,144
3,243
10,074 $
1,125
200
(86)
41
1,280
40%
7%
(8)%
1%
13%
General and administrative expenses for fiscal year 2017 increased by $1.3 million to $11.4 million compared to fiscal year
2016. The increase was primarily driven by the hiring of additional employees which resulted in higher share-based compensation
and other employee-related costs.
Other income (expense), net
For fiscal year 2017, other income (expense), net, which was primarily comprised of investment income, increased to $0.9
million from $0.7 million generated in 2016 due largely to modestly better investment performance.
Provision for income taxes
Income tax expense was $2.4 million for the year ended June 30, 2017 compared to no tax expense in fiscal year 2016. The
fiscal year 2017 income tax expense results from the recognition of revenue related to the Biogen agreement for tax purposes, which is
accelerated compared to our GAAP revenue, resulting in significantly more taxable income than GAAP net income. While our
taxable income is largely offset by the use of NOLs, our income tax expense is primarily due to federal alternative minimum tax
expense, the apportionment of income to certain state jurisdictions where we do not have NOLs and the recognition of a reserve for
uncertain tax positions.
74
Comparison of the fiscal years ended June 30, 2016 and 2015
Revenue
Fiscal year ended June 30,
Increase
% Increase
Collaboration revenue
Grant revenue
Sponsored research and other revenue
Total revenue
2016
$
$
46,751 $
610
—
47,361 $
(Decrease)
2015
(dollars in thousands)
— $
1,682
672
2,354 $
46,751
(1,072)
(672)
45,007
(Decrease)
n/m
(64)%
n/m
n/m
Total revenue for fiscal year 2016 was $47.4 million compared to $2.4 million generated during fiscal year 2015. The increase
was driven by revenue generated from our collaboration with Biogen, primarily comprised of the amortization of upfront fees and $5.0
million of milestone revenue earned during the fiscal year following achievement of a patient enrollment-based milestone under the
terms of the collaboration agreement. Grant revenue generated during fiscal year 2016 decreased compared to 2015 due largely to
reduced research and development activities under grant-funded projects. Other revenue recorded in 2015 related primarily to
sponsored research revenue earned from a patient advocacy group and income that was generated from a right of reference agreement
entered into with a strategic partner during that period.
Research and development expenses
Collaboration costs
Outside program costs
Employee-related costs
Licenses, milestones and related fees
Share-based compensation
Other
Total research and development expenses
Fiscal Year Ended June 30,
Increase
% Increase
2016
2015
(dollars in thousands)
(Decrease)
(Decrease)
$
$
12,034
11,965
5,631
4,402
2,147
3,197
39,376
$
$
—
10,800
2,923
1,590
908
1,897
18,118
$
$
12,034
1,165
2,708
2,812
1,239
1,300
21,258
n/m
11%
93%
177%
136%
69%
117%
Research and development expenses for fiscal year 2016 increased by $21.3 million to $39.4 million compared to fiscal year
2015, driven largely by $12.0 million of incremental sublicensing costs associated with our collaboration arrangement with Biogen.
Also contributing to the higher research and development expenses were $3.0 million of increased license and milestone fees from
new collaboration arrangements with external partners 4D Molecular Therapeutics and Synpromics Limited. In addition, employee-
related and share-based compensation costs also increased year-over-year due primarily to the hiring of additional employees to
support this increased level of research and development activity and the impact of new share-based incentives awarded during the
year.
General and administrative expenses
Employee-related costs
Share-based compensation
Legal and professional fees
Other
Total general and administrative expenses
Fiscal Year Ended June 30,
Increase
(Decrease)
% Increase
(Decrease)
2015
(dollars in thousands)
2016
$
$
2,827 $
2,860
1,144
3,243
10,074 $
2,231 $
1,912
1,909
2,716
8,768 $
596
948
(765)
527
1,306
27%
50%
(40)%
19%
15%
General and administrative expenses for fiscal year 2016 increased by $1.3 million to $10.1 million compared to fiscal year
2015. The increase was primarily driven by the hiring of additional employees which resulted in higher share-based compensation
and other employee-related costs. Other administrative expenses were also higher compared to 2015 due primarily to our ongoing
expansion and the increasing costs of operating as a publicly-traded company. The impact of these incremental costs was partially
offset by decreased legal and professional fees.
75
Other income (expense), net
For fiscal year 2016, other income (expense), net, which was primarily comprised of investment income, increased to $708,000
from $214,000 generated in 2015 due largely to comparatively higher cash and investment balances.
Liquidity and capital resources
We have incurred cumulative losses and negative cash flows from operations since our inception in 1999, and as of June 30,
2017, we had an accumulated deficit of $89.6 million. It will be several years, if ever, before we have a product candidate ready for
commercialization. We expect that our research and development and general and administrative expenses will continue to increase
and as a result, we anticipate that we will require additional capital to fund our operations, which we may raise through a combination
of equity offerings, debt financings, other third-party funding, marketing and distribution arrangements and other collaborations,
strategic alliances and licensing arrangements.
During fiscal year 2016, we received a non-refundable upfront cash payment of $94.0 million under our collaboration
arrangement with Biogen. Contemporaneous with this collaboration arrangement, we also entered into an equity agreement with
Biogen under which we received an additional $30.0 million in cash in exchange for 1,453,957 shares of common stock that we issued
to Biogen at a purchase price of $20.63 per share. During fiscal year 2017, we made an initial $2.0 million payment to Bionic Sight
for an equity interest in that company. Cash in excess of immediate requirements is invested in accordance with our investment policy
which primarily seeks to maintain adequate liquidity and preserve capital by generally limiting investments to certificates of deposit
and investment-grade debt securities that mature within 24 months. As of June 30, 2017, we had cash and cash equivalents and
investments of $138.4 million.
As of June 30, 2017, our cash and cash equivalents were held in bank accounts and money market funds, while our short and
long-term investments consisted of certificates of deposit and corporate and government bonds, none of which mature more than 24
months after the balance sheet date, consistent with our investment policy that seeks to maintain adequate liquidity and preserve
capital.
Cash flows
The following table sets forth the primary sources and uses of cash for each of the periods set forth below:
Net cash provided by (used in):
Operating activities
Investing activities
Financing activities
Net (decrease) increase in cash and cash equivalents
2017
Fiscal Year Ended June 30,
2016
(in thousands)
2015
$
$
(31,001)
32,811
28
1,838
$
$
70,991 $
(100,804)
19,494
(10,319) $
(19,485)
17,892
32,157
30,564
Operating activities. Cash used in operating activities of $31.0 million during the fiscal year ended June 30, 2017 was primarily
due to changes in operating assets and liabilities of $38.4 million, partially offset by non-cash items of $7.0 million, including $5.6
million of stock-based compensation expense, and net income of $0.4 million. The change in operating assets and liabilities was
primarily due to the amortization of upfront fees associated with our Biogen collaboration. For fiscal year 2016, net cash provided by
operating activities of $80.0 million was primarily associated with the upfront cash proceeds of $104.8 million received in connection
with the entry into our collaboration with Biogen, which included an allocation of $10.8 million from the equity agreement, and a
milestone payment from Biogen of $5.0 million. These proceeds were partially offset by the impact of our net loss, including the
amortization of deferred revenue and payments to certain research partner institutions in the aggregate amount of $12.0 million for
sub-license, milestone and other costs which were all associated with the Biogen collaboration, and changes during the period in our
working capital accounts. Cash used in operating activities of $19.5 million during the fiscal year ended June 30, 2015 was primarily
a result of our $24.3 million net loss, partially offset by non-cash items of $3.2 million, including $2.8 million of stock-based
compensation expense, and changes in operating assets and liabilities of $1.6 million.
Investing activities. Cash provided by investing activities of $32.8 million for the fiscal year ended June 30, 2017 was primarily
due to maturity of investments of $103.2 million, partially offset by purchases of investments of $67.5 million, a $2.0 million payment
to Bionic Sight for an equity interest in that company and purchases of property and equipment of $0.7 million. For the fiscal year
2016, net cash used in investing activities consisted primarily of cash outflows of $208.2 million related to the purchase of
investments and $2.6 million related to the purchase of property and equipment and the acquisition of intellectual property, including
leasehold improvements at our new facility in Alachua, Florida. These cash outflows were partially offset by $110.0 million of
76
proceeds from the maturity of investments. Net cash provided by investing activities during fiscal year 2015 of $17.9 million was
primarily comprised of proceeds totaling $121.1 million from the maturity of investments, partially offset by cash outflows of $102.9
million related to the purchase of investments and $323,000 related to the acquisition and maintenance of intellectual property and
purchase of property and equipment.
Financing activities. Net cash provided by financing activities was $28,000 during fiscal year 2017 and was due to proceeds
received from the exercise of stock options. Net cash provided by financing activities during fiscal year 2016 was $19.5 million and
consisted of $19.2 million of cash received in connection with our sale of shares of common stock to Biogen pursuant to the equity
agreement executed on July 1, 2015 and $283,000 of cash received in connection with the exercise of stock options. Net cash
provided by financing activities during fiscal year 2015 was $32.2 million, of which $32.0 million was related to the follow on public
offering that we completed during our first quarter ended September 30, 2014 and $148,000 consisted of cash received in connection
with the exercise of stock options.
Operating capital requirements
To date, we have not generated any revenue from product sales. We do not know when, or if, we will generate any revenue from
product sales. We do not expect to generate significant revenue from product sales unless and until we obtain regulatory approval of
and commercialize one of our current or future product candidates. We anticipate that we will continue to generate losses for the
foreseeable future as we continue the development of, and seek regulatory approvals for, our product candidates, and begin to
commercialize any approved products. We are subject to all of the risks incident in the development of new gene therapy products,
and we may encounter unforeseen expenses, difficulties, complications, delays and other unknown factors that may adversely affect
our business.
We believe that our existing cash and cash equivalents and investments at June 30, 2017, will be will be sufficient to allow us to
generate data from our ongoing clinical programs, to move our pre-clinical optogenetic program in collaboration with Bionic Sight
into the clinic and to fund our currently planned research and discovery programs for at least the next two years. In order to complete
the process of obtaining regulatory approval for our lead product candidates and to build the sales, marketing and distribution
infrastructure that we believe will be necessary to commercialize our lead product candidates, if approved, we will require substantial
additional funding.
We have based our projections of operating capital requirements on assumptions that may prove to be incorrect and we may use
all of our available capital resources sooner than we expect. Because of the numerous risks and uncertainties associated with research,
development and commercialization of pharmaceutical products, we are unable to estimate the exact amount of our operating capital
requirements. Our future funding requirements will depend on many factors, including, but not limited to:
(cid:129)
(cid:129)
(cid:129)
(cid:129)
(cid:129)
(cid:129)
(cid:129)
(cid:129)
(cid:129)
(cid:129)
(cid:129)
(cid:129)
(cid:129)
(cid:129)
the timing and costs our planned clinical trials for our XLRS and ACHM product candidates;
the timing and costs of our planned preclinical studies of our XLRP product candidate;
the timing and level of activity as determined by us or jointly with our partners;
the level of funding received from our partners;
whether or not we elect to cost share with our partners;
the initiation, progress, timing, costs and results of preclinical studies relating to potential applications of our gene therapy
platform in other indications;
our success in scaling our manufacturing method and expanding our manufacturing capabilities;
the number and characteristics of product candidates that we pursue;
the outcome, timing and costs of seeking regulatory approvals;
subject to receipt of marketing approval, revenue received from commercial sales of our product candidates;
the terms and timing of any future collaborations, licensing, consulting or other arrangements that we may establish;
the amount and timing of any payments we may be required to make, or that we may receive, in connection with the
licensing, filing, prosecution, defense and enforcement of any patents or other intellectual property rights;
the costs of preparing, filing and prosecuting patent applications, maintaining and protecting our intellectual property
rights and defending against intellectual property related claims; and
the extent to which we in-license or acquire other products and technologies.
77
Contractual obligations and commitments
The following table summarizes our contractual obligations at June 30, 2017:
In thousands
Operating lease obligations (1)
Purchase obligations (2)
Total
Less
than 1
Year
Total
1 to 3
Years
3 to 5
Years
More
than 5
Years
$
$
13,021 $
919
13,940 $
1,157 $
165
1,322 $
2,983 $
162
3,145 $
3,094 $
173
3,267 $
5,787
419
6,206
(1) Our current leased facilities encompass approximately 21,000 square feet of laboratory and office space in Alachua, Florida
under a lease arrangement that will expire in December 31, 2025. In addition, we occupy approximately 3,000 square feet
of office and laboratory space in Cambridge, Massachusetts. On July 31, 2017, we entered into a new lease to increase our
office and laboratory space in Cambridge by approximately 5,000 square feet to a total of approximately 8,000 square feet
and extend the term of the lease for an additional seven years, with an option to further extend the lease for one additional
three-year term.
(2) Consists of minimum annual royalties and maintenance fees under license agreements with third parties. In addition to these
minimum annual payments, we may be required to make future payments related to milestones or royalties on future sales
of specified products. These contingent payments generally become due and payable only upon achievement of specified
developmental, regulatory or commercial milestones. The amount and timing of any of such payments are not known due to
the uncertainty surrounding the successful research, development and commercialization of the products and, as such, have
not been included in the above table.
Contingent contractual obligations
We also have obligations arising under our license agreements to make future payments to third parties that become due and
payable on the achievement of certain development, regulatory and commercial milestones (such as the start of a clinical trial, filing of
a Biologics License Application, or BLA, approval by the FDA or product launch). We have not included these obligations on our
balance sheet or in the table above because the achievement and timing of these milestones is not fixed nor determinable. These
obligations include:
(cid:129)
(cid:129)
Under each of our various licenses with the University of Florida Research Foundation, or UFRF, covering the AAV
construct containing the AAT gene and the method to treat AAT deficiency using this construct, a small cone cell specific
promoter, and the use of engineered capsids and under our joint license with UFRF and Johns Hopkins University
covering a particular HSV construct and various compositions thereof, we will be required to make payments based upon
development, regulatory and commercial milestones for any products covered by the in-licensed intellectual property. We
will also be required to pay a royalty on net sales of products covered by the in-licensed intellectual property. We have
the right to sublicense our rights under this agreement, and we will be required to pay a percentage of such license
income. We are required to make annual maintenance payments under these licenses, which payments are creditable
against royalty payments on a year-by-year basis.
Under our license agreement with the UAB Research Foundation pursuant to which we license a patent covering the use
of HSV helpers to produce AAV vectors, we will be required to make payments based upon development and regulatory
milestones for any products covered by the in-licensed intellectual property. We will also be required to pay a royalty on
net sales of products covered by the in-licensed intellectual property. We have the right to sublicense our rights under this
agreement, and we will be required to pay a percentage of such license income. We are required to make annual
maintenance payments under this license, which payments are creditable against royalty payments on a year-by-year
basis.
If any of our product candidates that utilize technology licensed under these agreements reached commercialization, we will be
obligated to make royalty payments ranging from 0.5% to 4.0% of our net sales of the applicable product. We are responsible for a
portion of the costs related to the preparation, filing, issuance, prosecution and maintenance of the patents covered by the license
agreements. In fiscal years 2017, 2016 and 2015, we paid annual royalty and milestone payments in the aggregate amounts of
$54,000, $963,000 and $122,000, respectively.
78
We enter into contracts in the normal course of business with contract research organizations for preclinical research studies,
research supplies and other services and products for operating purposes. These contracts generally provide for termination on notice,
and therefore are cancelable contracts and not included in the table of contractual obligations and commitments.
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 Securities and Exchange Commission.
ITEM 7A. QUANTITATIVE AND QUALITATIVE DISCLOSURES ABOUT MARKET RISK
Interest Rate Sensitivity
Our financial instruments at June 30, 2017 consisted primarily of cash and cash equivalents and short-term and long-term
investments totaling $138.4 million. These financial instruments are exposed to the impact of interest rate changes which may result
in fluctuations to our interest income. Due to the nature of our investments in money market funds, certificates of deposits, and debt
instruments of corporations and U.S. government agencies, all of which generally mature within a two-year period of their purchase
date, the estimated fair values of these financial instruments approximate their carrying amounts at June 30, 2017.
We maintain our investment portfolio in accordance with our investment policy. The primary objectives of this investment policy are
to maintain adequate liquidity, preserve capital and to meet our operating needs. Although our investments are subject to credit risk,
our investment policy specifies credit quality standards for our investments and limits the amount of credit exposure from any single
issue, issuer or type of investment. Our investments are also subject to interest rate risk and may decrease in value if market interest
rates increase. However, due to the conservative nature of our investments and relatively short investment periods, we believe interest
rate risk is mitigated and an immediate 10% increase in interest rates would not have a material effect on the fair market value of our
portfolio. We do not own derivative financial instruments. Accordingly, we do not believe that there is any material market risk
exposure with respect to derivative or other financial instruments.
79
ITEM 8.
FINANCIAL STATEMENTS AND SUPPLEMENTARY DATA
APPLIED GENETIC TECHNOLOGIES CORPORATION
INDEX TO FINANCIAL STATEMENTS
Report of Independent Registered Public Accounting Firm ..................................................................................................
81
Page(s)
Financial Statements
Balance Sheets at June 30, 2017 and 2016...................................................................................................................
Statements of Operations for the fiscal years ended June 30, 2017, 2016 and 2015 ...................................................
Statements of Stockholders’ Equity for the fiscal years ended June 30, 2017, 2016 and 2015 ..................................
Statements of Cash Flows for the fiscal years ended June 30, 2017, 2016 and 2015 ..................................................
Notes to Financial Statements ......................................................................................................................................
82
83
84
85
86-108
Schedule II—Valuation and Qualifying Accounts.................................................................................................................
116
80
The Board of Directors and Stockholders of Applied Genetic Technologies Corporation
Report of Independent Registered Public Accounting Firm
We have audited the accompanying balance sheets of Applied Genetic Technologies Corporation (the Company) as of June 30, 2017
and 2016, and the related statements of operations, stockholders’ equity and cash flows for each of the three years in the period ended
June 30, 2017. Our audits also included the financial statement schedule of the Company listed in Item 15(a). These financial
statements and financial statement schedule are the responsibility of the Company's management. Our responsibility is to express an
opinion on these financial statements and schedule based on our audits.
We conducted our audits in accordance with the standards of the Public Company Accounting Oversight Board (United States). Those
standards require that we plan and perform the audit to obtain reasonable assurance about whether the financial statements are free of
material misstatement. The Company is not required to have, nor were we engaged to perform, an audit of its internal control over
financial reporting. Our audits included consideration of internal control over financial reporting as a basis for designing audit
procedures that are appropriate in the circumstances, 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. An audit also includes examining, on a
test basis, evidence supporting the amounts and disclosures in the financial statements, assessing the accounting principles used and
significant estimates made by management, and evaluating the overall financial statement presentation. We believe that our audits
provide a reasonable basis for our opinion.
In our opinion, the financial statements referred to above present fairly, in all material respects, the financial position of Applied
Genetic Technologies Corporation as of June 30, 2017 and 2016, the results of its operations and its cash flows for each of the three
years in the period ended June 30, 2017, in conformity with U.S. generally accepted accounting principles. Also, in our opinion, the
related financial statement schedule, when considered in relation to the basic financial statements taken as a whole, presents fairly in
all material respects the information set forth therein.
/s/ RSM US LLP
Raleigh, North Carolina
September 13, 2017
81
APPLIED GENETIC TECHNOLOGIES CORPORATION
BALANCE SHEETS
In thousands, except per share data
ASSETS
Current assets:
Cash and cash equivalents
Investments
Grants receivable
Prepaid and other current assets
Total current assets
Investments, net of current portion
Property and equipment, net
Intangible assets, net
Investment in Bionic Sight
Other assets
Total assets
LIABILITIES AND STOCKHOLDERS' EQUITY
Current liabilities:
Accounts payable
Accrued and other liabilities
Deferred revenue
Total current liabilities
Deferred revenue, net of current portion
Total liabilities
Stockholders' equity:
$
$
$
Common stock, par value $.001 per share, 150,000 shares authorized; 18,088 and
18,053 shares issued and outstanding at June 30, 2017 and June 30, 2016, respectively
Additional paid-in capital
Accumulated deficit
Total stockholders' equity
Total liabilities and stockholders' equity
$
At June 30,
2017
2016
30,706 $
95,994
174
3,361
130,235
11,749
2,661
1,219
2,000
59
147,923 $
998 $
6,162
20,996
28,156
4,438
32,594
18
204,937
(89,626)
115,329
147,923 $
28,868
69,664
954
3,089
102,575
74,183
2,627
1,321
—
91
180,797
1,331
6,514
46,898
54,743
16,766
71,509
18
199,303
(90,033)
109,288
180,797
The accompanying notes are an integral part of the financial statements.
82
APPLIED GENETIC TECHNOLOGIES CORPORATION
STATEMENTS OF OPERATIONS
In thousands, except per share amounts
Revenue:
Collaboration revenue
Grant revenue
Sponsored research and other revenue
Total revenue
Operating expenses:
Research and development
General and administrative
Total operating expenses
Income (loss) from operations
Other income (expense):
Investment income, net
Other expense
Total other income (expense), net
Income (loss) before provision for income taxes
Provision for income taxes
Net income (loss)
Net income (loss) per share, basic
Net income (loss) per share, diluted
Weighted average shares outstanding, basic
Weighted average shares outstanding, diluted
$
$
$
$
For the fiscal years ended June 30,
2016
2015
2017
$
39,282
191
—
39,473
26,217
11,354
37,571
1,902
952
(47)
905
2,807
2,400
407
0.02
0.02
18,072
18,385
$
$
$
$
46,751
610
—
47,361
39,376
10,074
49,450
(2,089)
711
(3)
708
(1,381)
—
(1,381) $
(0.08) $
(0.08) $
17,810
17,810
—
1,682
672
2,354
18,118
8,768
26,886
(24,532)
216
(2)
214
(24,318)
—
(24,318)
(1.50)
(1.50)
16,253
16,253
The accompanying notes are an integral part of the financial statements.
83
APPLIED GENETIC TECHNOLOGIES CORPORATION
STATEMENTS OF STOCKHOLDERS’ EQUITY
Common Stock
Additional
In thousands
Balance, June 30, 2014
Issuance of common stock, net of issuance costs
Share-based compensation expense
Shares issued under employee plans
Exercise of Warrants
Net loss
Balance, June 30, 2015
Issuance of common stock, net of issuance costs
Share-based compensation expense
Shares issued under employee plans
Exercise of warrants
Net loss
Balance, June 30, 2016
Share-based compensation expense
Shares issued under employee plans
Exercise of warrants
Net income
Balance, June 30, 2017
Outstanding
Shares
Amount
$
$
14,082
2,300
—
94
15
—
16,491
1,494
—
59
9
—
18,053
$
—
31
4
—
18,088 $
$
$
$
Paid-in
Capital
139,193
32,007
2,820
148
—
—
174,168
19,805
5,007
283
40
—
199,303
Accumulated
Deficit
(64,334)
—
—
—
—
(24,318)
(88,652)
—
—
—
—
(1,381)
(90,033)
$
$
5,606
28
—
204,937 $
$
—
—
—
407
(89,626) $
$
$
14
2
—
—
—
—
16
2
—
—
—
—
$
18
—
—
—
—
18 $
Total
74,873
32,009
2,820
148
—
(24,318)
85,532
19,807
5,007
283
40
(1,381)
109,288
5,606
28
—
407
115,329
The accompanying notes are an integral part of the financial statements.
84
APPLIED GENETIC TECHNOLOGIES CORPORATION
STATEMENTS OF CASH FLOWS
In thousands
Cash flows from operating activities
Net income (loss)
Adjustments to reconcile net income (loss) to net cash used in operating
activities:
Share-based compensation expense
Share-based collaboration expense
Depreciation and amortization
Investment premium accretion
Loss on disposal of property, plant and equipment
Changes in operating assets and liabilities:
Decrease (increase) in grants receivable
Increase (decrease) in prepaid and other assets
(Decrease) increase in accounts payable
Increase (decrease) in deferred revenues
Increase in accrued and other liabilities
Net cash (used in) provided by operating activities
Cash flows from investing activities
Purchase of property and equipment
Purchase of and capitalized costs related to intangible assets
Investment in Bionic Sight
Maturity of investments
Purchase of investments
Net cash provided by (used in) investing activities
Cash flows from financing activities
Proceeds from issuance of common stock, net of issuance costs
Proceeds from exercise of common stock options
Net cash provided by financing activities
Net (decrease) increase in cash and cash equivalents
Cash and cash equivalents, beginning of period
Cash and cash equivalents, end of period
Supplemental disclosure of cash flow information
Cash paid during the year for income taxes
For the fiscal years ended June 30,
2016
2015
2017
$
407
$
(1,381) $
(24,318)
5,606
—
912
402
47
780
(240)
(333)
(38,230)
(352)
(31,001)
(739)
(152)
(2,000)
103,244
(67,542)
32,811
—
28
28
1,838
28,868
30,706
$
5,007
636
567
448
—
409
(1,562)
140
63,664
3,063
70,991
(2,471)
(121)
—
109,968
(208,180)
(100,804)
19,211
283
19,494
(10,319)
39,187
28,868
$
2,820
—
376
158
—
(876)
261
228
—
1,866
(19,485)
(225)
(98)
—
121,079
(102,864)
17,892
32,009
148
32,157
30,564
8,623
39,187
887 $
—
$
—
$
$
The accompanying notes are an integral part of the financial statements.
85
APPLIED GENETIC TECHNOLOGIES CORPORATION
NOTES TO FINANCIAL STATEMENTS— (Continued)
FOR THE YEARS ENDED JUNE 30, 2017, 2016 AND 2015
(1) Organization and Operations:
Applied Genetic Technologies Corporation (the “Company” or “AGTC”) was incorporated as a Florida corporation on
January 19, 1999 and reincorporated as a Delaware corporation on October 24, 2003. The Company is a clinical-stage
biotechnology company that uses a proprietary gene therapy platform to develop transformational genetic therapies for patients
suffering from rare and debilitating diseases.
In July 2015, the Company entered into a collaboration agreement (the “Collaboration Agreement”) with Biogen MA, Inc., a
wholly owned subsidiary of Biogen Inc. (“Biogen”), pursuant to which the Company and Biogen will collaborate to develop,
seek regulatory approval for and commercialize gene therapy products to treat X-linked retinoschisis (“XLRS”), X-linked
retinitis pigmentosa (“XLRP”), and discovery programs targeting three indications based on the Company’s adeno-associated
virus vector technologies. The Collaboration Agreement became effective in August 2015. The Collaboration Agreement and
other transactions with Biogen are discussed further in Note 7 to these financial statements.
The Company has devoted substantially all of its efforts to research and development, including clinical trials. The Company
has not completed the development of any products. The Company has generated revenue from collaboration agreements,
sponsored research payments and grants, but has not generated product revenue to date and is subject to a number of risks
similar to those of other early stage companies in the biotechnology industry, including dependence on key individuals, the
difficulties inherent in the development of commercially viable products, the need to obtain additional capital necessary to fund
the development of its products, development by the Company or its competitors of technological innovations, risks of failure of
clinical studies, protection of proprietary technology, compliance with government regulations and ability to transition to large-
scale production of products. As of June 30, 2017, the Company had an accumulated deficit of $89.6 million. While the
Company expects to continue to generate some revenue from partnering, including under the collaboration with Biogen, the
Company expects to incur losses for the foreseeable future. The Company has funded its operations to date primarily through
public offerings of its common stock, private placements of its preferred stock, and collaborations. At June 30, 2017, the
Company had cash and cash equivalents and investments of $138.4 million.
(2) Summary of Significant Accounting Policies:
Basis of Presentation
The accompanying financial statements have been prepared in conformity with accounting principles generally accepted in the
United States of America (“GAAP”) and, in the opinion of management, include all adjustments necessary for a fair presentation
of the Company’s financial position, results of operations, and cash flows for each period presented.
Certain amounts reported previously have been reclassified to conform to the current year presentation, with no effect on
stockholders’ equity or net loss as previously presented.
Segment Reporting
Operating segments are identified as components of an enterprise about which separate discrete financial information is available
for evaluation by the chief operating decision-maker in making decisions regarding resource allocation and assessing
performance. To date, we have viewed our operations and managed our business as one segment.
Use of estimates
The preparation of financial statements in conformity with GAAP requires management to make estimates and assumptions that
affect the reported amounts of assets and liabilities, the disclosure of contingent assets and liabilities at the date of the financial
statements and the reported amount of revenues and expenses during the reporting period. Actual results could differ from those
estimates.
Cash and cash equivalents
Cash consists of funds held in bank accounts. Cash equivalents consist of short-term, highly liquid investments with original
maturities of 90 days or less at the time of purchase and generally include money market accounts.
86
APPLIED GENETIC TECHNOLOGIES CORPORATION
NOTES TO FINANCIAL STATEMENTS— (Continued)
FOR THE YEARS ENDED JUNE 30, 2017, 2016 AND 2015
Investments
The Company’s investments consist of certificates of deposit and debt securities classified as held-to-maturity. Management
determines the appropriate classification of debt securities at the time of purchase and reevaluates such designation as of each
balance sheet. Debt securities are classified as held-to-maturity when the Company has the positive intent and ability to hold the
securities to maturity. Held-to-maturity securities are stated at amortized cost, adjusted for amortization of premiums and
accretion of discounts to maturity. Such amortization is included in investment income. Interest on securities classified as held-
to-maturity is included in investment income.
The Company uses the specific identification method to determine the cost basis of securities sold.
Investments are considered to be impaired when a decline in fair value is judged to be other-than-temporary. The Company
evaluates an investment for impairment by considering the length of time and extent to which market value has been less than
cost or amortized cost, the financial condition and near-term prospects of the issuer as well as specific events or circumstances
that may influence the operations of the issuer and the Company’s intent to sell the security or the likelihood that it will be
required to sell the security before recovery of the entire amortized cost. Once a decline in fair value is determined to be other-
than-temporary, an impairment charge is recorded to other income (expense) and a new cost basis in the investment is
established.
Concentrations of Credit Risk
The Company maintains its cash and cash equivalents and certificates of deposit with two financial institutions that are federally
insured. Some of these financial instruments are in excess of federally insured limits and as a result, could potentially expose the
Company to significant concentrations of credit risk. To date, the Company has not experienced any losses associated with this
credit risk and continues to believe that this exposure is not significant. The Company invests its excess cash primarily in money
market funds, certificates of deposit, and debt instruments of corporations and U.S. government agencies. These investments
generally mature within a two-year period from their purchase date, in line with the Company’s investment policy that seeks to
maintain adequate liquidity and preserve capital.
Inventory
Purchases of clinical materials stored for master and working viral banks that remain at the sites in anticipation of their future
use at that site are charged to expense when they are incurred. Since the Company can use each of the raw materials in only a
single product, each raw material is deemed to have no future economic value independent of the development status of that
single drug.
87
APPLIED GENETIC TECHNOLOGIES CORPORATION
NOTES TO FINANCIAL STATEMENTS— (Continued)
FOR THE YEARS ENDED JUNE 30, 2017, 2016 AND 2015
Fair value of financial instruments
The Company is required to disclose information on all assets and liabilities reported at fair value that enables an assessment of
the inputs used in determining the reported fair values. The Financial Accounting Standards Board (“FASB”) Accounting
Standard Codification (“ASC”) Topic 820, Fair Value Measurements and Disclosures (“ASC 820”), establishes a hierarchy of
inputs used when available. Observable inputs are inputs that market participants would use in pricing the asset or liability based
on market data obtained from sources independent of the Company. Unobservable inputs are those that reflect the Company’s
assumptions about the inputs that market participants would use in pricing the asset or liability, and are developed based on the
best information available in the circumstances. The fair value hierarchy applies only to the valuation inputs used in determining
the reported fair value of financial instruments and is not a measure of the investment credit quality. The three levels of the fair
value hierarchy are described below:
Level 1—Valuations based on unadjusted quoted prices in active markets for identical assets or liabilities that the Company has
the ability to access at the measurement date.
Level 2—Valuations based on quoted prices for similar assets or liabilities in markets that are not active or for which all
significant inputs are observable, either directly or indirectly.
Level 3—Valuations that require inputs that reflect the Company’s own assumptions that are both significant to the fair value
measurement and are unobservable.
To the extent that valuation is based on models or inputs that are less observable or unobservable in the market, the
determination of fair value requires more judgment. Accordingly, the degree of judgment exercised by the Company in
determining fair value is greatest for instruments categorized in Level 3. A financial instrument’s level within the fair value
hierarchy is based on the lowest level of any input that is significant to the fair value measurement.
Property and equipment
Property and equipment, consisting of laboratory equipment, furniture and fixtures, computer equipment and leasehold
improvements, are recorded at cost. Depreciation is computed using the straight-line method over the estimated useful lives of
the assets, which are generally three to seven years. Leasehold improvements are stated at cost and are amortized over the
shorter of the estimated useful lives of the assets or the lease term, including any renewal periods that are deemed to be
reasonably assured. Repair and maintenance costs that do not improve service potential or extend an asset’s economic life are
recorded as an expense when incurred.
Intangible assets
Intangible assets primarily include licenses and patents. The Company obtains licenses from third parties and capitalizes the
costs related to exclusive licenses that have alternative future use in multiple potential programs. The Company also capitalizes
costs related to filing, issuance, and prosecution of patents. The Company reviews its capitalized costs periodically to determine
that such costs relate to patent applications that have future value and an alternative future use, and writes off any costs
associated with patents that are no longer being actively pursued or that have no future benefit.
Amortization expense is computed using the straight-line method over the estimated useful lives of the assets, which are
generally eight to twenty years. The Company amortizes in-licensed patents and patent applications from the date of the
applicable license and internally developed patents and patent applications from the date of the initial application. Licenses and
patents converted to research use only are immediately written off to expense.
Impairment of long-lived assets
The Company reviews its long-lived assets for impairment when impairment indicators are present. If impairment indicators
exist, management determines whether impairment in value has occurred by comparing the estimated undiscounted cash flows
from future operations with the carrying values of the assets. Management considers several indicators in assessing impairment,
including trends and prospects, as well as the effects of obsolescence, demand, competition and other economic factors. No
impairment charges were recorded for each of the fiscal years ended June 30, 2017, 2016, and 2015.
88
APPLIED GENETIC TECHNOLOGIES CORPORATION
NOTES TO FINANCIAL STATEMENTS— (Continued)
FOR THE YEARS ENDED JUNE 30, 2017, 2016 AND 2015
Revenue recognition
The Company has primarily generated revenue through collaboration agreements, sponsored research arrangements with
nonprofit organizations for the development and commercialization of product candidates and revenues from federal research
and development grant programs. The Company recognizes revenue when amounts are realized or realizable and earned.
Revenue is considered realizable and earned when the following criteria are met: (1) persuasive evidence of an arrangement
exists; (2) delivery has occurred or services have been rendered; (3) the price is fixed or determinable; and (4) collection of the
amounts due are reasonably assured.
Amounts received prior to satisfying the revenue recognition criteria are recorded as deferred revenue in the Company’s balance
sheets. Amounts expected to be recognized as revenue within the 12 months following the balance sheet date are classified as
current liabilities. The Company recognizes revenue for reimbursements of research and development costs under collaboration
agreements as the services are performed. The Company records these reimbursements as revenue and not as a reduction of
research and development expenses, as the Company has the risks and rewards as the principal in the research and development
activities.
The Company evaluates the terms of sponsored research agreement grants and federal grants to assess the Company’s
obligations and if the Company’s obligations are satisfied by the passage of time, revenue is recognized on a straight-line basis.
In situations where the performance of the Company’s obligations has been satisfied when the grant is received, revenue is
recognized upon receipt of the grant. Certain grants contain refund provisions. The Company reviews those refund provisions to
determine the likelihood of repayment. If the likelihood of repayment of the grant is determined to be remote, the grant is
recognized as revenue. If the probability of repayment is determined to be more than remote, the Company records the grant as a
deferred revenue liability, until such time that the grant requirements have been satisfied.
Collaboration revenue
On July 1, 2015, the Company entered into a Collaboration Agreement with Biogen. This collaboration is discussed further in
Note 7 of notes to the financial statements. The terms of this agreement and other potential collaboration or commercialization
agreements the Company may enter into generally contain multiple elements, or deliverables, which may include, among others,
(i) licenses, or options to obtain licenses, to its technology, and (ii) research and development activities to be performed on
behalf of the collaborative partner. Payments made under such arrangements typically include one or more of the following:
non-refundable, up-front license fees; option exercise fees; funding of research and/or development efforts; milestone payments;
and royalties on future product sales.
Multiple element arrangements are analyzed to determine whether the deliverables within the agreement can be separated or
whether they must be accounted for as a single unit of accounting. Deliverables under an agreement are required to be accounted
for as separate units of accounting provided that (i) a delivered item has value to the customer on a stand-alone basis; and (ii) if
the agreement includes a general right of return relative to the delivered item, delivery or performance of the undelivered item is
considered probable and substantially in the control of the vendor. The allocation of consideration amongst the deliverables
under the agreement is derived using a “best estimate of selling price” if vendor specific objective evidence and third-party
evidence of fair value is not available. If the delivered element does not have stand-alone value or if the fair value of any of the
undelivered elements cannot be determined, the arrangement is then accounted for as a single unit of accounting, and the
Company recognizes the consideration received under the arrangement as revenue on a straight-line basis over the estimated
period of performance.
The Company determines the estimated selling price for deliverables within each agreement using vendor-specific objective
evidence, or VSOE, of selling price, if available, third-party evidence, or TPE, of selling price if VSOE is not available, or best
estimate of selling price, or BESP, if neither VSOE nor TPE are available. Determining the best estimate of selling price for a
deliverable requires significant judgment. The Company uses BESP to estimate the selling price related to licenses to its
proprietary technology, since it often does not have VSOE or TPE of selling price for these deliverables. In those circumstances
where it utilizes BESP to determine the estimated selling price of a license to our proprietary technology, the Company considers
market conditions as well as entity-specific factors, including those factors contemplated in negotiating the agreements as well as
internally developed models that include assumptions related to the market opportunity, estimated development costs, probability
of success and the time needed to commercialize a product candidate pursuant to the license. In validating its best estimate of
selling price, the Company evaluates whether changes in the key assumptions used to determine the best estimate of selling price
will have a significant effect on the allocation of arrangement consideration among multiple deliverables.
89
APPLIED GENETIC TECHNOLOGIES CORPORATION
NOTES TO FINANCIAL STATEMENTS— (Continued)
FOR THE YEARS ENDED JUNE 30, 2017, 2016 AND 2015
If the delivered element does not have stand-alone value or if the fair value of any of the undelivered elements cannot be
determined, the arrangement is then accounted for as a single unit of accounting, and the Company recognizes the consideration
received under the arrangement as revenue on a straight-line basis over our estimated period of performance. The Company’s
anticipated periods of performance, typically the terms of our research and development obligations, are subject to estimates by
management and may change over the course of the collaboration agreement. Such changes could have a material impact on the
amount of revenue we record in future periods.
Milestone revenue
The Company applies the milestone method of accounting to recognize revenue from milestone payments when earned, as
evidenced by written acknowledgement from the collaborator or other persuasive evidence that the milestone has been achieved
and the payment is non-refundable, provided that the milestone event is substantive. A milestone event is defined as an event (i)
that can only be achieved based in whole or in part on either the Company’s performance or on the occurrence of a specific
outcome resulting from the Company’s performance; (ii) for which there is substantive uncertainty at the inception of the
arrangement that the event will be achieved; and (iii) that would result in additional payments being due to the Company. Events
for which the occurrence is either contingent solely upon the passage of time or the result of a counterparty’s performance are
not considered to be milestone events. A milestone event is substantive if all of the following conditions are met: (i) the
consideration is commensurate with either the Company’s performance to achieve the milestone, or the enhancement of the
value to the delivered item(s) as a result of a specific outcome resulting from the Company’s performance to achieve the
milestone; (ii) the consideration relates solely to past performance; and (iii) the consideration is reasonable relative to all the
deliverables and payment terms (including other potential milestone consideration) within the arrangement.
The Company assesses whether a milestone is substantive at the inception of the arrangement. If a milestone is deemed non-
substantive, the Company accounts for that milestone payment in accordance with the multiple element arrangements guidance
and recognizes revenue consistent with the related units of accounting for the arrangement over the related performance period.
During the fiscal year ended June 30, 2016 the Company recognized milestone revenue in the amount of $5.0 million. No
milestone revenues were recognized during the fiscal years ended June 30, 2017 or 2015.
Income taxes
The Company uses the asset and liability method for accounting for income taxes. Under this method, deferred tax assets and
liabilities are recognized for the estimated future tax consequences attributable to differences between the financial statement
carrying amounts of existing assets and liabilities and their respective income tax bases. Deferred tax assets and liabilities are
measured using enacted rates expected to apply to taxable income in the years in which those temporary differences are expected
to be recovered or settled.
For the fiscal year ended June 30, 2017, the Company recorded an income tax provision of $2.4 million, related to the
Company’s federal alternative minimum taxable income (“AMTI”) and state income tax in multiple states where the Company is
doing business. The Company calculates its AMTI using the alternative minimum tax (“AMT”) system. The Company’s federal
income tax liability is the greater of the tax computed using the regular tax system or the tax under the AMT system.
Corporations are exempt from AMT for all prior years in which their annual gross receipts for the 3-year period ending before
the current tax year did not exceed $7.5 million. For the fiscal year ending June 30, 2017, the Company no longer qualifies for
the small company exclusion. The AMT system limits the use of net operating losses used by taxpayers to offset taxable income.
As required by U.S. GAAP, the Company recognizes the financial statement benefit of a tax position only after determining that
the relevant tax authority would more likely than not sustain the position following an audit. For tax positions meeting the more-
likely-than-not threshold, the amount recognized in the financial statements is the largest benefit that has a greater than 50
percent likelihood of being realized upon ultimate settlement with the relevant tax authority. Any interest and penalties related to
uncertain tax positions will be reflected in income tax expense. The Company is subject to examination of its income tax returns
in the federal and state income tax jurisdictions in which it operates. On December 28, 2015, the United States Internal Revenue
Service, or IRS, notified the Company of an income tax audit for the tax period ending June 30, 2014. As of June 30, 2017, the
IRS audit was closed and the Company incurred no penalties or payment liabilities for its income tax positions.
90
APPLIED GENETIC TECHNOLOGIES CORPORATION
NOTES TO FINANCIAL STATEMENTS— (Continued)
FOR THE YEARS ENDED JUNE 30, 2017, 2016 AND 2015
For the year ended June 30, 2017, the Company’s $2.4 million of tax expense includes an uncertain tax position liability of
$950,000 related to uncertainty in how states may tax income from the Biogen collaboration agreement. This collaboration
agreement is described in more detail in Note 7 of the notes to the financial statements. No similar liability was recorded for the
years ended June 30, 2016 and 2015.
Research and development expenses
Research and development costs include costs incurred in identifying, developing and testing product candidates and generally
comprise compensation and related benefits and non-cash share-based compensation to research related employees; laboratory
costs; animal and laboratory maintenance and supplies; rent; utilities; clinical and pre-clinical expenses; and payments for
sponsored research, scientific and regulatory consulting fees and testing.
As part of the process of preparing financial statements, the Company is required to estimate its accrued expenses. This process
involves reviewing quotations and contracts, identifying services that have been performed on its behalf and estimating the level
of service performed and the associated cost incurred for the service when the Company has not yet been invoiced or otherwise
notified of the actual cost. The majority of the Company’s service providers invoice the Company monthly in arrears for
services performed or when contractual milestones are met. The Company makes estimates of its accrued expenses as of each
balance sheet date in its financial statements based on facts and circumstances known to it at that time. The significant estimates
in the Company’s accrued research and development expenses are related to expenses incurred with respect to academic research
centers, contract research organizations (“CROs”), and other vendors in connection with research and development activities for
which it has not yet been invoiced.
There may be instances in which the Company’s service providers require advance payments at the inception of a contract or in
which payments made to these vendors will exceed the level of services provided, resulting in a prepayment of the research and
development expense. Such prepayments are charged to research and development expense as and when the service is provided
or when a specific milestone outlined in the contract is reached.
Prepayments related to research and development activities were $1.5 million and $2.0 million at June 30, 2017 and 2016,
respectively, and are included within the Prepaid and other current assets line item on the balance sheets.
Share-based compensation
The Company accounts for share-based awards issued to employees in accordance with ASC Topic 718, Compensation—Stock
Compensation (“ASC 718”) and generally recognizes share-based compensation expense on a straight-line basis over the periods
during which the employees are required to provide service in exchange for the award. In addition, the Company issues stock
options and restricted shares of common stock to non-employees in exchange for consulting services and accounts for these in
accordance with the provisions of ASC Subtopic 505-50, Equity-Based Payments to Non-employees (“ASC 505-50”). Under
ASC 505-50, share-based awards to non-employees are subject to periodic fair value re-measurement over their vesting terms.
For purposes of calculating stock-based compensation, the Company estimates the fair value of stock options using a Black-
Scholes option-pricing model. The determination of the fair value of share-based payment awards utilizing the Black-Scholes
model is affected by the Company’s stock price and a number of assumptions, including expected volatility, expected life, risk-
free interest rate and expected dividends. The expected volatility is primarily based on the historical volatility of peer company
data while the expected life of the stock options is based on historical and other economic data trended into the future. The risk-
free interest rate assumption is based on observed interest rates appropriate for the expected terms of the Company’s stock
options. The dividend yield assumption is based on the Company’s history and expectation of no dividend payouts. If factors
change and the Company employs different assumptions, stock-based compensation expense may differ significantly from what
has been recorded in the past. If there is a difference between the assumptions used in determining stock-based compensation
expense and the actual factors which become known over time, specifically with respect to anticipated forfeitures, the Company
may change the input factors used in determining stock-based compensation costs for future grants. These changes, if any, may
materially impact the Company’s results of operations in the period such changes are made.
Net earnings (loss) per share
91
APPLIED GENETIC TECHNOLOGIES CORPORATION
NOTES TO FINANCIAL STATEMENTS— (Continued)
FOR THE YEARS ENDED JUNE 30, 2017, 2016 AND 2015
Basic net earnings (loss) per share is calculated by dividing net earnings (loss) by the weighted average shares outstanding
during the period, without consideration for common stock equivalents. Diluted net earnings (loss) per share is calculated by
adjusting weighted average shares outstanding for the dilutive effect of common stock equivalents outstanding for the period,
determined using the treasury-stock method. For purposes of the diluted net earnings (loss) per share calculation, stock options
and warrants are considered to be common stock equivalents. The dilutive impact of stock options and warrants for the fiscal
year ended June 30, 2017 totaled 0.3 million shares. The dilutive impact of stock options and warrants have been excluded from
the calculation of diluted net loss per share for the fiscal years ended June 30, 2016 and 2015, as their effect would be anti-
dilutive. Therefore, for the fiscal years ended June 30, 2016 and 2015, basic and diluted net loss per share are the same.
92
APPLIED GENETIC TECHNOLOGIES CORPORATION
NOTES TO FINANCIAL STATEMENTS— (Continued)
FOR THE YEARS ENDED JUNE 30, 2017, 2016 AND 2015
Comprehensive loss
Comprehensive loss consists of net loss and changes in equity during a period from transactions and other equity and
circumstances generated from non-owner sources. The Company’s net loss equals comprehensive loss for all periods presented.
New Accounting Pronouncements
In May 2017, the FASB issued Accounting Standards Update (“ASU”) No. 2017-09, Scope of Modification Accounting, which
amends ASC Topic 718, Compensation – Stock Compensation. The amendments in this Update provide guidance about which
changes to the terms or conditions of a share-based payment award require an entity to apply modification accounting in Topic
718. The amendments are effective for fiscal years beginning after December 15, 2017, and interim periods within those fiscal
years and early adoption is permitted. The Company is currently in the process of evaluating the impact of adoption of this
standard on its financial statements.
In March 2016, the FASB issued ASU No. 2016-09, Improvements to Employee Share-Based Payment Accounting, which
amends ASC Topic 718, Compensation – Stock Compensation. The amendments simplify several aspects of the accounting for
share-based payment transactions, including the income tax consequences, classification of awards as either equity or liabilities,
forfeitures, and classification on the statement of cash flows. The amendments are effective for fiscal years beginning after
December 15, 2016, and interim periods within those fiscal years and early adoption is permitted. The Company has adopted this
standard for its 2018 fiscal year and does not expect it to have a material effect on its balance sheets, results of operations or cash
flows.
In February 2016, the FASB issued ASU No. 2016-02, Leases (Topic 842) in order to increase transparency and comparability
among organizations by recognizing lease assets and lease liabilities on the balance sheet for those leases previously classified as
operating leases under GAAP. The standard requires, in most instances, a lessee to recognize on its balance sheet a liability to
make lease payments (the lease liability) and also a right-of-use asset representing its right to use the underlying asset for the
lease term. The amendments are effective for fiscal years beginning after December 15, 2018, including interim periods within
those periods, using a modified retrospective approach and early adoption is permitted. The Company is currently in the process
of evaluating the impact of adoption of this standard on its financial statements.In May 2014, the FASB issued ASU No. 2014-
09, Revenue from Contracts with Customers (Topic 606) which replaces the existing accounting standards for revenue
recognition with a single comprehensive five-step model. The core principle is to recognize revenue upon the transfer of goods
or services to customers at an amount that reflects the consideration expected to be received. It also requires enhanced
disclosures about revenue, provides guidance for transactions that were not previously addressed comprehensively, and improves
guidance for multiple-element arrangements. The guidance applies to any entity that either enters into contracts with customers
to transfer goods or services or enters into contracts for the transfer of nonfinancial assets unless those contracts are within the
scope of other standards. Since its issuance, the FASB has amended several aspects of the new guidance, including provisions
that address revenue recognition associated with the licensing of intellectual property. In July 2015, the FASB delayed the
effective date of this guidance by one year. The guidance is now effective for public companies for annual periods beginning
after December 15, 2017 as well as interim periods within those annual periods using either the full retrospective approach or
modified retrospective approach. The Company is working through an adoption plan which will include a review of
collaboration agreements, applying the five-step model of the new standard and comparing the results to the Company’s current
accounting. As part of this, the Company is evaluating the method of adoption and assessing changes that might be necessary to
its processes, internal controls and address changes in financial reporting. Effective July 1, 2018, the Company will be revising
its revenue recognition accounting policy and expanding revenue disclosures to reflect the requirements of the amended revenue
recognition guidance. Because of the nature of the work that remains, at this time the Company is unable to reasonably estimate
the impact of adoption on its consolidated financial statements.
93
APPLIED GENETIC TECHNOLOGIES CORPORATION
NOTES TO FINANCIAL STATEMENTS— (Continued)
FOR THE YEARS ENDED JUNE 30, 2017, 2016 AND 2015
(3)
Investments:
Cash in excess of our immediate requirements is invested in accordance with the Company’s investment policy that primarily
seeks to maintain adequate liquidity and preserve capital.
The following table summarizes the Company’s investments by category as of June 30, 2017 and 2016:
In thousands
Investments - Current:
Certificates of deposit
Debt securities - held-to-maturity
Investments - Noncurrent:
Certificates of deposit
Debt securities - held-to-maturity
June 30,
2017
June 30,
2016
$
$
$
$
3,500 $
92,494
95,994 $
2,111 $
9,638
11,749 $
18,093
51,571
69,664
2,544
71,639
74,183
A summary of the Company’s debt securities classified as held-to-maturity is as follows:
In thousands
Investments - Current:
U.S. government and agency obligations
Corporate obligations
Investments - Noncurrent:
U.S. government and agency obligations
Corporate obligations
In thousands
Investments - Current:
U.S. government and agency obligations
Corporate obligations
Investments - Noncurrent:
U.S. government and agency obligations
Amortized Cost
$
$
$
$
$
$
$
92,494
-
92,494
7,552
2,086
9,638
$
Amortized Cost
$
$
$
$
40,609
10,962
51,571
$
$
71,639
71,639
$
$
At June 30, 2017
Gross
Unrealized
Gains
Gross
Unrealized
Losses
Fair Value
-
-
-
-
-
-
$
$
$
$
(147) $
-
(147) $
(52) $
(12)
(64) $
92,347
-
92,347
7,500
2,074
9,574
At June 30, 2016
Gross
Unrealized
Gains
Gross
Unrealized
Losses
Fair Value
(2) $
(1)
(3) $
40,619
10,964
51,583
(11) $
(11) $
71,681
71,681
12
3
15
$
$
53
53
$
$
The amortized cost and fair value of held-to-maturity debt securities as of June 30, 2017, by contractual maturity, were as
follows:
In thousands
Due in one year or less
Due after one year through two years
Amortized Cost
Fair Value
$
$
92,494 $
9,638
102,132 $
92,347
9,574
101,921
94
APPLIED GENETIC TECHNOLOGIES CORPORATION
NOTES TO FINANCIAL STATEMENTS— (Continued)
FOR THE YEARS ENDED JUNE 30, 2017, 2016 AND 2015
The Company believes that the unrealized losses disclosed above were primarily driven by interest rate changes rather than by
unfavorable changes in the credit ratings associated with these securities and as a result, the Company continues to expect to
collect the principal and interest due on its debt securities that have an amortized cost in excess of fair value. At each reporting
period, the Company evaluates securities for impairment when the fair value of the investment is less than its amortized cost.
The Company evaluated the underlying credit quality and credit ratings of the issuers, noting neither a significant deterioration
since purchase nor other factors leading to an other-than-temporary impairment. Therefore, the Company believes these losses
to be temporary. As of June 30, 2017, the Company did not have any intent to sell any of the securities that were in an
unrealized loss position at that date.
(4)
Fair Value Measurements:
Certain assets and liabilities are measured at fair value in the Company’s financial statements or have fair values disclosed in the
notes to the financial statements. These assets and liabilities are classified into one of three levels of a hierarchy defined by
GAAP. The Company’s assessment of the significance of a particular item to the fair value measurement in its entirety requires
judgment, including the consideration of inputs specific to the asset or liability.
The following methods and assumptions were used to estimate the fair value and determine the fair value hierarchy
classification of each class of financial instrument included in the table below:
Cash and Cash Equivalents. The carrying value of cash and cash equivalents approximates fair value as maturities are less
than three months.
Certificates of Deposit. The Company’s certificates of deposit are placed through an account registry service. The fair value
measurement of the Company’s certificates of deposit is considered Level 2 of the fair value hierarchy as the inputs are based on
quoted prices for identical assets in markets that are not active. The carrying amounts of the Company’s certificates of deposit
reported in the balance sheets approximate fair value.
Debt securities – held-to-maturity. The Company’s investments in debt securities classified as held-to-maturity generally
include U.S. Treasury Securities, government agency obligations and corporate obligations. U.S. Treasury Securities are valued
using quoted market prices. Valuation adjustments are not applied. Accordingly, U.S. Treasury Securities are considered Level
1 of the fair value hierarchy. The fair values of U.S. government agency obligations and corporate obligations are generally
determined using recently executed transactions, broker quotes, market price quotations where these are available or other
observable market inputs for the same or similar securities. As such, the Company classifies its investments in U.S. government
agency obligations and corporate obligations within Level 2 of the hierarchy.
95
APPLIED GENETIC TECHNOLOGIES CORPORATION
NOTES TO FINANCIAL STATEMENTS— (Continued)
FOR THE YEARS ENDED JUNE 30, 2017, 2016 AND 2015
The following fair value hierarchy table presents information about each major category of the Company’s financial assets and
liabilities measured at fair value on a recurring basis:
In thousands
June 30, 2017
Cash and cash equivalents
Certificates of deposit
Held-to-maturity investments:
Corporate obligations
U.S. government and agency obligations
Total assets
June 30, 2016
Cash and cash equivalents
Certificates of deposit
Held-to-maturity investments:
Corporate obligations
U.S. government and agency obligations
Total assets
Quoted prices
in active
markets
(Level 1)
Significant
other
observable
inputs
(Level 2)
Significant
unobservable
inputs
(Level 3)
Total Fair
Value
Total
Carrying
Value
$
30,706 $
—
— $
5,601
— $
—
30,706 $
5,601
30,706
5,610
—
79,476
110,182 $
2,074
20,372
28,047 $
—
—
— $
2,074
99,848
138,229
$
2,086
100,046
138,448
28,868 $
—
— $
20,626
— $
—
28,868 $
20,626
28,868
20,637
—
73,809
102,677 $
10,964
38,491
70,081 $
—
—
— $
10,964
112,300
172,758
$
10,962
112,248
172,715
$
$
$
96
APPLIED GENETIC TECHNOLOGIES CORPORATION
NOTES TO FINANCIAL STATEMENTS— (Continued)
FOR THE YEARS ENDED JUNE 30, 2017, 2016 AND 2015
(5)
Property and Equipment, Net:
Property and equipment consists of the following:
In thousands
Laboratory equipment
Leasehold improvements
Office equipment
Property and equipment, gross
Less: Accumulated depreciation and amortization
Property and equipment, net
At June 30,
2017
2016
$
$
2,645
1,198
470
4,313
(1,652)
2,661
$
$
2,414
883
514
3,811
(1,184)
2,627
Depreciation expense of $657,000, $319,000 and $126,000 was recorded for each of the fiscal years ended June 30, 2017, 2016
and 2015, respectively.
(6)
Intangible Assets, Net:
Intangible assets subject to amortization consist of the following:
In thousands
Patents
Licenses
Other
Intangible assets, net
In thousands
Patents
Licenses
Other
Intangible assets, net
At June 30, 2017
Net of
Cost
Accumulated
Amortization
Accumulated
Amortization
2,135 $
1,240
54
3,429 $
(1,205) $
(981)
(24)
(2,210) $
930
259
30
1,219
At June 30, 2016
Net of
Cost
Accumulated
Amortization
Accumulated
Amortization
1,982 $
1,240
87
3,309 $
(1,035) $
(907)
(46)
(1,988) $
947
333
41
1,321
$
$
$
$
Amortization expense related to intangible assets for the years ended June 30, 2017, 2016 and 2015 was $255,000, $248,000 and
$250,000, respectively.
Estimated amortization expense (in thousands) for the next five years and thereafter is as follows:
Fiscal Year Ending June 30,
2018
2019
2020
2021
2022
Thereafter
$
$
Amount
246
202
203
179
71
318
1,219
97
APPLIED GENETIC TECHNOLOGIES CORPORATION
NOTES TO FINANCIAL STATEMENTS— (Continued)
FOR THE YEARS ENDED JUNE 30, 2017, 2016 AND 2015
(7) Collaboration Agreements
Biogen
On July 1, 2015, the Company entered into a Collaboration Agreement with Biogen, pursuant to which the Company and
Biogen will collaborate to develop, seek regulatory approval for and commercialize gene therapy products to treat XLRS,
XLRP, and discovery programs targeting three indications based on the Company’s adeno-associated virus vector
technologies. The Collaboration Agreement became effective on August 14, 2015.
Under the Collaboration Agreement, the Company will conduct all development activities through regulatory approval in the
United States for the XLRS program (with activities through Phase 1/2 completion being pre-funded under the agreement and
any further activities subject to incremental consideration), and all development activities through the completion of the first in
human clinical trial for the XLRP program (with activities through filing the IND being pre-funded under the agreement and any
further activities subject to incremental consideration). In addition, the Collaboration Agreement provides for discovery
programs targeting three indications whereby the Company will conduct discovery, research and development activities for
those additional drug candidates through the stage of clinical candidate designation, after which, Biogen may exercise an option
to continue to develop, seek regulatory approval for and commercialize the designated clinical candidate. In February 2016, the
Company announced Biogen’s selection of adrenoleukodystrophy as the non-ophthalmic indication of the discovery
programs. Under the terms of the Collaboration Agreement, the Company, in part through its participation in joint committees
with Biogen, will participate in overseeing the development and commercialization of these specific programs.
The Company has granted to Biogen with respect to the XLRS and XLRP programs, and upon exercise of the option for the
applicable discovery program, an exclusive, royalty-bearing license, with the right to grant sublicenses, to use adeno-associated
virus vector technology and other technology controlled by the Company for the licensed products or discovery programs
developed under the Collaboration Agreement. Biogen and the Company have also granted each other worldwide licenses, with
the right to grant sublicenses, of their respective interests in other intellectual property developed under the collaboration outside
the licensed products or discovery programs.
Activities under the Collaboration Agreement were evaluated under ASC 605-25, Revenue Recognition—Multiple Element
Arrangements, as amended by ASU 2009-13, Revenue Recognition ("ASC 605-25"), to determine if they represented a multiple
element revenue arrangement. The Collaboration Agreement includes the following significant deliverables:
(1) for each of the XLRS and XLRP programs, exclusive, royalty-bearing licenses, with the right to grant sublicenses, to use
adeno-associated virus vector technology and other technology controlled by the Company for the purpose of researching,
developing, manufacturing and commercializing licensed products developed under the arrangement (the “License
Deliverables”);
(2) for each of the discovery programs, exercisable options to obtain exclusive licenses to develop, seek regulatory approval for
and commercialize any of the designated clinical candidates under such discovery programs (the “Option Deliverables”); and
(3) the performance obligations to conduct research and development activities through (a) regulatory approval in the United
States, in the case of the XLRS program; (b) completion of the first in human clinical trial, in the case of the XLRP program;
and (c) the stage of clinical candidate designation, in the case of each of the discovery programs (the “R&D Activity
Deliverables”).
The R&D Activity Deliverables for each program are further segmented by those that are “Pre-Funded Activities” and those that
are “Post-Funding Development Activities”. Pre-Funded Activities are those R&D activities for which the Company has
primary responsibility and the consideration to be received under the agreement was received at the inception of the
arrangement. Post-Funding Development Activities are those activities that may occur after the Pre-Funded Activities and for
which the Company is entitled to additional compensation under the agreement from Biogen. Biogen has final decision-making
authority for all matters related to the conduct of the Post-Funding Development Activities. Because Biogen is not contractually
obligated to continue the programs beyond the Pre-Funded Activities, and due to the uncertain outcome of the discovery,
research and development activities, the Post-Funding Development Activities are not considered deliverables at the inception
of the arrangement and the associated fees and milestones are not included in the allocable arrangement consideration. The
Company has determined that the additional fees it could receive under the arrangement for Post-Funding Development
Activities are not priced at a significant and incremental discount.
The Company determined that both the License Deliverables and Option Deliverables do not have stand-alone value and do not
meet the criteria to be accounted for as separate units of accounting under ASC 605-25. The factors considered by the Company
in making this determination included, among other things, the unique and specialized nature of its proprietary technology and
98
APPLIED GENETIC TECHNOLOGIES CORPORATION
NOTES TO FINANCIAL STATEMENTS— (Continued)
FOR THE YEARS ENDED JUNE 30, 2017, 2016 AND 2015
intellectual property, and the development stages of each of the XLRS, XLRP and the discovery programs targeting three
indications. Accordingly, the License Deliverables under each of the XLRS and XLRP programs and the Option Deliverables
under each of the discovery programs have been combined with the initial, Pre-Funded Activities deliverables associated with
each related program and as a result, the Company’s separate units of accounting under its collaboration with Biogen, comprise
the XLRS program, the XLRP program, and each of the discovery programs.
Under the Collaboration Agreement, the Company received a non-refundable upfront payment of $94.0 million in August 2015
which it recorded as deferred revenue. This upfront payment of $94.0 million was allocated among the separate units of
accounting discussed above using the relative selling price method. In addition to the Collaboration Agreement, on July 1,
2015, the Company also entered into an equity agreement with Biogen. Under the terms of this equity agreement, Biogen
purchased 1,453,957 shares of the Company’s common stock at a price of $20.63 per share, for an aggregate cash purchase price
of $30.0 million which the Company also received in August 2015. The shares issued to Biogen represented approximately
8.1% of the Company’s outstanding common stock on a post-issuance basis, calculated on the number of shares that were
outstanding at June 30, 2015, and constitute restricted securities that may not be resold by Biogen other than in a transaction
registered under, or pursuant to an exemption from the registration requirements of, the Securities Act of 1933, as amended.
Accounting standards for multiple element arrangements contain a presumption that separate contracts negotiated or entered into
at or near to the same time with the same entity were likely negotiated as a package and should be evaluated as a single
agreement. The Company determined that the price of $20.63 paid by Biogen included a premium of $7.45 per share over the
fair value of the company’s stock price, calculated based upon the stock price on the date of close of the agreement and adjusted
for lack of marketability due to restrictions. Accordingly, the total premium of $10.8 million was also recorded as deferred
revenue and, together with the $94.0 million, allocated to the separate units of accounting identified above using the relative
selling price method as discussed in Note 2 to these financial statements. The Company will record revenue based on the
revenue recognition criteria applicable to each separate unit of accounting. For amounts received up-front and initially deferred,
the Company will recognize the deferred revenue on a straight-line basis over the estimated service periods in which it is
required to perform the research and development activities associated with each unit of accounting. At the inception of the
Collaboration Agreement, the Company initially estimate the service periods to range between 2 and 3 years. However, due to
certain delays which have extended our estimated period of performance, the estimated service periods are currently anticipated
to be between 2 and 4 years from the inception of the Collaboration Agreement.
During the fiscal years ended June 30, 2017 and 2016, the Company recognized revenue of approximately $39.3 million and
$46.8 million, respectively from its collaboration with Biogen. Below is a summary of the components of the collaboration
revenue:
Fiscal year ended June 30,
2017
2016
Amortization of non-refundable upfront fees
Milestone revenue
Other
Total collaboration revenue
$
$
$
(dollars in thousands)
38,230
—
1,052
39,282
$
41,166
5,000
585
46,751
During the fiscal year ended June 30, 2016, the Company recorded and received $5.0 million of milestone revenue after having
achieved a patient enrollment-based milestone under the Collaboration Agreement. Other revenue is primarily comprised of
reimbursable costs for post-funding development activities that were conducted by the Company during the fiscal year.
As a result of the upfront payment of $94.0 million made by Biogen and achievement of the $5.0 million milestone as discussed
above, the Company became liable to various research partner institutions for sub-license and other payments under existing
agreements with such institutions. These agreements obligate the Company to pay to each research partner institution, amounts
that range from 5% to 10% of certain proceeds received from collaboration and other arrangements, including any milestone
payments received under such arrangements. Accordingly, the Company recorded total collaboration costs of approximately
$12.0 million associated with such obligations, including $636,000 of expense that was settled during fiscal year 2016 by the
issuance of 40,000 shares of the Company’s common stock to a research partner institution, pursuant to the terms of the existing
agreement with that institution. The remainder of these sub-license and milestone fees were fully paid in cash during the fiscal
year ended June 30, 2016.
99
APPLIED GENETIC TECHNOLOGIES CORPORATION
NOTES TO FINANCIAL STATEMENTS— (Continued)
FOR THE YEARS ENDED JUNE 30, 2017, 2016 AND 2015
The Company is also eligible to receive payments of up to $467.5 million based on the successful achievement of future
milestones under its XLRS and XLRP programs. For XLRS, the Company is eligible to receive up to: (i) $40 million in
milestone payments based upon the successful achievement of clinical milestones (relating to dosing in specified trials), (ii)
$155 million in milestone payments based upon the achievement of regulatory approvals and first commercial sale in specified
territories and (iii) $65 million in milestone payments based upon the achievement of worldwide sales targets. For XLRP, the
Company is eligible to receive up to: (i) $42.5 million in milestone payments based upon successful achievement of clinical
milestones (relating to dosing in specified trials), (ii) $102.5 million in milestone payments based upon the achievement of
regulatory approvals and first commercial sale in specified territories and (iii) $62.5 million in milestone payments based upon
the achievement of worldwide sales targets. In addition, the Company is eligible to receive payments of up to $592.5 million
based on the exercise of the option for and the successful achievement of future milestones under its discovery programs. Each
discovery program is categorized as Category A, Category B or Category C depending on the nature of the indication it seeks to
address. For Category A, the Company is eligible to receive payments of up to: (i) $20 million based upon the successful
achievement of clinical milestones (relating to dosing in specified trials) and (ii) $70 million in milestone payments based upon
the achievement of regulatory approvals and first commercial sale in specified territories. For Category B, the Company is
eligible to receive payments of up to: (i) $27.5 million based upon the successful achievement of clinical milestones (relating to
dosing in specified trials) and (ii) $105 million in milestone payments based upon the achievement of regulatory approvals and
first commercial sale in specified territories. For Category C, the Company is eligible to receive payments of up to: (i) $40
million based upon the successful achievement of clinical milestones (relating to dosing in specified trials) and (ii) $140 million
in milestone payments based upon the achievement of regulatory approvals and first commercial sale in specified territories.
Under certain limited circumstances, if there are discovery products from more than one discovery program in any of Category
A, Category B or Category C, then the milestone payments under the applicable category shall be payable for the applicable
discovery product from each such discovery program to achieve the specified milestones.
Biogen will also pay revenue-based royalties for each licensed product at tiered rates ranging from high single digit to mid-teen
percentages of annual net sales of the XLRS or XLRP products and at rates ranging from mid-single digit to low-teen
percentages of annual net sales for the discovery products. The Company has elected to apply the guidance in ASC 605-28 to
the milestones. These milestones, if achieved, are substantive as they relate solely to past performance, are commensurate with
estimated enhancement of value associated with the achievement of each milestone as a result of the Company’s performance
and are reasonable when compared to other consideration amounts payable under the Collaboration Agreement; however, there
can be no assurance that the Company will achieve the milestones or that the Company will receive the related revenue. Due to
the uncertainty surrounding the achievement of the future milestones, such payments were not considered fixed or determinable
at the inception of the Collaboration Agreement and accordingly, will not be recognized as revenue unless and until they
become earned. The Company is not able to reasonably predict if and when the remaining milestones will be achieved.
Bionic Sight
On February 2, 2017, the Company entered into a strategic research and development collaboration agreement with Bionic
Sight, LLC (“Bionic Sight”), to develop therapies for patients with visual deficits and blindness due to retinal disease. Through
the AGTC-Bionic Sight collaboration, the companies seek to develop a new optogenetic therapy that leverages AGTC’s deep
experience in gene therapy and ophthalmology and Bionic Sight’s innovative neuro-prosthetic device and algorithm for retinal
coding.
Under the agreement, AGTC made an initial $2.0 million payment to Bionic Sight for an equity interest in that company. This
initial investment represents an approximate 5% equity interest in Bionic Sight. In addition to the initial investment, AGTC will
contribute to ongoing research and development support costs through additional payments or other in-kind contributions
(AGTC Ongoing R&D Support). The AGTC Ongoing R&D Support payments and in-kind contributions will be made over
time, up to the date that Bionic Sight has received both IND clearance from the FDA and receipt of written approval from an
internal review board to conduct clinical trials from at least one clinical site for that product (the “IND Trigger”.)
If the IND Trigger is attained, AGTC will receive additional equity, based on the valuation in place at the beginning of the
agreement, for the AGTC Ongoing R&D Support payments and in-kind contributions, and will be obligated to purchase
additional equity in Bionic Sight for $4.0 million, at a pre-determined valuation. Due to the uncertainty of achieving the IND
Trigger, the Company is expensing the AGTC Ongoing R&D Support payments and in-kind contributions made under the
collaboration agreement. Such amounts are included as a component of research and development expenses in the Company’s
financial statements.
100
APPLIED GENETIC TECHNOLOGIES CORPORATION
NOTES TO FINANCIAL STATEMENTS— (Continued)
FOR THE YEARS ENDED JUNE 30, 2017, 2016 AND 2015
The Company recorded its initial $2.0 million investment in Bionic Sight using the cost method of accounting for investments,
which is recorded as its own line item on the Company’s balance sheet. The ongoing research and development costs and
contributions will be recorded as a periodic cost until such time when or if the IND Trigger is achieved.
The collaboration agreement grants to AGTC, subject to achievement by Bionic Sight of certain development milestones, an
option to exclusively negotiate for a limited period of time to acquire (i) a majority equity interest in Bionic Sight, (ii) the Bionic
Sight assets to which the collaboration agreement relates, or (iii) an exclusive license with respect to the product to which the
collaboration agreement relates.
(8)
Share-based Compensation Plans:
The Company uses stock options and awards of restricted stock to provide long-term incentives for its employees, non-
employee directors and certain consultants. The Company has two equity compensation plans under which awards are currently
authorized for issuance, the 2013 Employee Stock Purchase Plan and the 2013 Equity and Incentive Plan. No awards have been
issued to date under the 2013 Employee Stock Purchase Plan and all of the 128,571 shares previously authorized under this plan
remain available for issuance. As of June 30, 2017, the total number of shares available for issuance under the 2013 Equity and
Incentive Plan was 543,626.
The Compensation Committee of the Board of Directors, as the plan administrator, has the authority to select the individuals to
whom share-based awards are granted and to determine the terms of each award, including (i) the number of shares of common
stock subject to a stock option or restricted share award; (ii) the date on which the stock option becomes exercisable; (iii) the
option exercise price, which, in the case of incentive stock options, must be at least 100% (110% in the case of incentive stock
options granted to a stockholder owning in excess of 10% of the Company’s stock) of the fair market value of the common stock
as of the date of grant; (iv) the vesting term; and (v) the duration of the option (which, in the case of incentive stock options,
may not exceed ten years). Employee options typically vest over a three- or four-year period.
A summary of the stock option activity is as follows:
2017
For the years ended June 30,
2016
2015
In thousands, except per share amounts
Outstanding, beginning of year
Granted
Exercised
Forfeited
Expired
Outstanding, end of year
Exercisable, end of year
Weighted average fair value of options granted during
the year
$
Weighted
Average
Exercise
Price
Weighted
Average
Exercise
Price
Shares
Weighted
Average
Exercise
Price
Shares
Shares
2,037 $
842
(31)
(101)
(31)
2,716 $
1,431
13.71
10.92
0.91
13.97
16.17
12.95
1,484 $
675
(59)
(57)
(6)
2,037 $
872
11.83
17.08
4.77
13.90
15.50
13.71
1,024 $
615
(46)
(109)
1,484 $
424
6.21
19.58
3.24
6.43
11.83
7.54
$
11.83
$
14.39
101
APPLIED GENETIC TECHNOLOGIES CORPORATION
NOTES TO FINANCIAL STATEMENTS— (Continued)
FOR THE YEARS ENDED JUNE 30, 2017, 2016 AND 2015
The following table summarizes information about stock options outstanding:
(Number of options in thousands; remaining lives in years)
Exercise Price
$0.35 to $4.90
$4.95 to $10.55
$12.00 to $15.90
$16.00 to $19.87
$20.00 to $24.62
At June 30,
2017
Weighted
Average
Contractual Life
Remaining
Number of
Options
2016
Weighted
Average
Number of
Options
Contractual Life
Remaining
475
467
744
864
166
2,716
5.57
9.48
8.11
7.83
7.57
509
—
412
940
176
2,037
The following table summarizes information about stock options exercisable:
Exercise Price
$0.35 to $4.90
$4.95 to $10.55
$12.00 to $15.90
$16.00 to $19.87
$20.00 to $24.62
At June 30,
2017
2016
Number of Options
(in thousands)
460
24
355
482
110
1,431
6.48
—
8.35
8.89
8.58
396
—
160
239
77
872
As of June 30, 2017, the aggregate intrinsic value of all outstanding stock options was $11.0 million and for exercisable stock
options was $11.0 million. The intrinsic value per option at June 30, 2017 is calculated as the difference between the exercise
price of the underlying option and the closing price of the Company’s common stock of $5.10 on that date, and applies only to
those awards having an exercise price currently below this closing price. The total fair value of options that vested during the
fiscal years ended June 30, 2017, 2016, and 2015 was $6.0 million, $4.5 million, and $1.9 million, respectively.
Unrecognized compensation expense related to non-vested employee stock options amounted to $11.2 million as of June 30,
2016. Such compensation expense is expected to be recognized over a weighted-average period of 2.40 years.
In accounting for stock options to non-employees, the fair value of services related to the options granted are generally recorded
as an expense as these services are provided to the Company over the relating service periods. The Company re-measures any
non-vested, non-employee options to fair value at the end of each reporting period using the Black-Scholes pricing model.
102
APPLIED GENETIC TECHNOLOGIES CORPORATION
NOTES TO FINANCIAL STATEMENTS— (Continued)
FOR THE YEARS ENDED JUNE 30, 2017, 2016 AND 2015
Share-based compensation expense related to stock options awarded to employees, non-employee directors and consultants
amounted to $5.6 million, $4.9 million and $2.3 million for the fiscal years ended June 30, 2017, 2016 and 2015, respectively.
Share-based compensation expense related to restricted shares of common stock awarded to employees and consultants
amounted to $5,000, $167,000 and $524,000 for the fiscal years ended June 30, 2017, 2016 and 2015, respectively.
Total share-based expense associated with stock options and restricted shares of common stock was allocated as follows:
In thousands
General and administrative
Research and development
For the fiscal years ended June 30,
2016
2015
2017
$
$
3,136 $
2,470
5,606 $
2,860 $
2,147
5,007 $
1,912
908
2,820
The fair value of each option granted is estimated on the grant date using the Black-Scholes stock option pricing model. The
following assumptions were made in estimating fair value:
Assumption
Dividend yield
Expected term
Risk-free interest rate
Expected volatility
2017
Fiscal Years Ended June 30,
2016
2015
0.00%
0.00%
0.00%
6.00 to 6.25 years
1.08% to 2.11%
6.00 to 6.25 years
1.41% to 1.86%
6.00 to 10.00 years
2.04% to 2.31%
79.18%
78.85%
85.68%
The dividend yield is based upon the assumption that the Company will not declare a dividend over the life of the options. Since
adopting ASC 718, the Company has been unable to use historical employee exercise and option expiration data to estimate the
expected term assumption for the Black-Scholes grant-date valuation. The Company therefore has utilized the “simplified”
method, as prescribed by the SEC’s Staff Accounting Bulletin No. 107, Share-Based Payment, to estimate on a formula basis
the expected term of our stock options considered to have “plain vanilla” characteristics. The risk-free interest rate is based on
the U.S. Treasury yield curve on the date of valuation. As a relatively new public company, the Company does not have
sufficient history to estimate the volatility of its common stock price or the expected life of the options. The Company calculates
expected volatility based on reported data for similar publicly traded companies for which historical information is available and
will continue to do so until the historical volatility of its common stock is sufficient to measure expected volatility for future
option grants. The group of similar publicly traded companies was determined based upon companies considered to be direct
competition or having been presented by independent parties as a “comparable” company based upon market sector. In
determining this group, the Company has excluded “large-cap” entities. Forfeitures are estimated at the time of the grant and
revised, if necessary, in subsequent periods if actual forfeitures differ from those estimates. Share-based compensation expense
recognized in the statements of operations for the fiscal years ended June 30, 2017, 2016 and 2015 does not reflect tax related
effects on stock-based compensation given the Company’s historical and anticipated operating losses and offsetting changes in
its valuation allowance that fully reserves against potential deferred tax assets.
103
APPLIED GENETIC TECHNOLOGIES CORPORATION
NOTES TO FINANCIAL STATEMENTS— (Continued)
FOR THE YEARS ENDED JUNE 30, 2017, 2016 AND 2015
(9) Commitments and Contingencies:
Operating Leases
Alachua, Florida
The Company’s corporate headquarters are located in Alachua, Florida. In January 2016, the Company moved into a new
combined-use facility consisting of approximately 21,000 square feet of laboratory and office space. The initial lease term for
this facility is 10 years and the Company has options to extend the term of the lease for three additional five-year periods. The
Company’s prior leased facilities encompassed approximately 7,000 square feet of office and laboratory space. The operating
leases associated with the prior facilities expired in December 2015.
Cambridge, Massachusetts
In August 2015, the Company entered into a two-year lease to occupy approximately 3,000 square feet of office and laboratory
space in Cambridge, Massachusetts. In July 2017, we entered into a new lease to increase our office and laboratory space in Cambridge
by approximately 5,000 square feet to a total of approximately 8,000 square feet and extend the term of the lease for an additional seven years,
with an option to further extend the lease for one additional three-year term. This additional facility primarily focuses on business
development, pharmacology, and basic research and development.
For the fiscal years ended June 30, 2017, 2016, and 2015, rent expense under these operating leases amounted to $845,000,
$587,000 and $167,000, respectively. Future annual minimum lease payments (in thousands) under these non-cancelable
operating leases are as follows:
Fiscal Year Ending June 30,
2018
2019
2020
2021
2022
Thereafter
$
$
Amount
1,157
1,478
1,505
1,533
1,561
5,787
13,021
License and Other Agreements
Under various agreements, the Company will be required to pay royalties and milestone payments upon the successful
development and commercialization of products. The Company has entered into funding agreements with various not-for-profit
organizations. The Company may become obligated to pay royalties on net product sales of any collaboration product that it
successfully develops and subsequently commercializes or, if it out-licenses rights to a collaboration product, a specified
percentage of certain payments it receives from its licensee. The Company is not obligated to make such payments unless and
until annual sales of a collaboration product exceed a designated threshold. The Company’s obligation to make such payments
would end upon its payment of a specified amount.
The Company is also party to various agreements entered into in the ordinary course of its business, principally relating to
licensed technology. At June 30, 2017, the Company had seven license agreements with six different entities, including four
with the University of Florida Research Foundation. The Company is responsible for all costs related to preparation, filing,
issuance, prosecution and maintenance of the underlying patents covered in the license agreements. The Company is required to
pay minimum annual royalty and license maintenance for all licenses until such time when the license is terminated by either
expiration of underlying patents or voluntary termination by either party per the agreement.
These license agreements also require future payments related to milestones or royalties on future sales of specified products.
Payments under these agreements generally become due and payable only upon achievement of certain developmental,
regulatory or commercial milestones. Because the achievement of these milestones had not occurred as of June 30, 2016, such
contingencies have not been recorded in the Company’s financial statements. Amounts related to contingent milestone payments
are not considered contractual obligations as they are contingent on the successful achievement of certain development,
regulatory and commercial milestones. There is uncertainty regarding the various activities and outcomes needed to reach these
104
APPLIED GENETIC TECHNOLOGIES CORPORATION
NOTES TO FINANCIAL STATEMENTS— (Continued)
FOR THE YEARS ENDED JUNE 30, 2017, 2016 AND 2015
milestones, and they may not be achieved. The Company may terminate its license agreements with zero to ninety days written
notice depending upon the terms of each specific agreement.
The Company’s expense associated with annual royalty and milestone payments was $54,000, $963,000 and $122,000 for each
of the fiscal years ended June 30, 2017, 2016 and 2015, respectively. All royalty and milestone payments are included within
research and development expenses in the statement of operations.
The Company enters into standard indemnification agreements in the ordinary course of business. Pursuant to these agreements,
the Company indemnifies, holds harmless, and agrees to reimburse the indemnified party for losses suffered or incurred by the
indemnified party, generally the Company’s business partners or customers, in connection with any U.S. patent or any copyright
or other intellectual property infringement claim by any third party with respect to the Company’s products. The term of these
indemnification agreements is generally perpetual. The maximum potential amount of future payments the Company could be
required to make under these indemnification agreements is unlimited. The Company has never incurred costs to defend
lawsuits or settle claims related to these indemnification agreements. From time to time, the Company may be involved in
claims and legal actions that arise in the normal course of business. Management has no reason to believe that the outcome of
any such legal actions would have a significant adverse effect on the Company’s financial position, results of operations or cash
flows.
(10)
Income Taxes:
For the fiscal years ended June 30, 2016 and 2015, the Company did not record a current or deferred income tax expense or
benefit. For the fiscal year ended June 30, 2017, the federal and state income tax provision (benefit) summarized as follows:
In thousands
Current provision:
Federal
State
Deferred tax liabilities:
Federal
State
Provision for income taxes
At June 30,
2017
2016
$
13,706 $
1,774
15,480
(12,949)
(131)
(13,080)
$
2,400 $
1,199
127
1,326
(1,199)
(127)
(1,326)
—
Deferred taxes are recognized for temporary differences between the basis of assets and liabilities for financial statement and
income tax purposes. The significant components of the Company’s deferred tax assets (liabilities) are comprised of the
following:
In thousands
Deferred tax assets:
Net operating loss carryforwards
Tax credit carryforwards
Accruals and other
Gross deferred tax assets
Deferred tax asset valuation allowance
Total deferred tax assets, net of valuation allowance
Deferred tax liabilities:
Depreciation and amortization
Total deferred tax liabilities
Net deferred tax asset (liability)
105
At June 30,
2017
2016
$
$
9,124 $
19,195
11,987
40,306
(40,303)
3
(3)
(3)
— $
27,103
8,771
1,599
37,473
(37,412)
61
(61)
(61)
—
APPLIED GENETIC TECHNOLOGIES CORPORATION
NOTES TO FINANCIAL STATEMENTS— (Continued)
FOR THE YEARS ENDED JUNE 30, 2017, 2016 AND 2015
As of June 30, 2017, the Company had net operating losses of approximately $24.0 million that may be applied against future
taxable income and expire in various years ranging from 2022 to 2037. As of June 30, 2017, the Company also had research
and development tax credits of approximately $18.4 million that may provide future tax benefits and expire from 2027 to 2046,
and alternative minimum tax credits of approximately $0.7 million, that may provide future tax benefits.
The Company has evaluated the positive and negative evidence bearing upon the realizability of its deferred tax assets. Based on
its history of operating losses, the Company has concluded that as of June 30, 2017, it is more likely than not that the benefit of
its deferred tax assets will not be realized. Therefore, any tax benefits to be realized in future years as a result of the utilization
of the Company’s net operating loss carry forwards as of June 30, 2017, computed based on statutory federal and state rates, are
completely offset by valuation allowances established because realization of the deferred tax benefits are not considered more
likely than not as of that date. On July 1, 2015, the Company entered into a collaboration agreement with Biogen, under which it
received a non-refundable upfront payment of $94.0 million. This collaboration agreement is discussed in more detail in Note 7
of notes to the financial statements. As of June 30, 2017, deferred revenue of $25.4 million recorded in the accompanying
balance sheets will be recognized as income as required under tax laws. The valuation allowance increased by approximately
$2.9 million during the fiscal year ended June 30, 2017, due primarily to the net increase in federal tax credits reduced by the
utilization in net operating loss carryforwards from the recognition in deferred revenue.
A reconciliation of income tax expense computed at the statutory federal income tax rate to income taxes as reflected in the
financial statements is as follows:
Federal income tax benefit at statutory rate
State income tax, net of federal benefit
Permanent differences
Research and development tax credits
Other
Change in valuation allowance
Effective income tax rate
2017
For the years ended June 30,
2016
2015
34%
4%
130%
(240)%
416%
(259)%
85%
(34)%
(4)%
52%
(60)%
85%
(39)%
0%
(34)%
(4)%
9%
(18)%
1%
46%
0%
Under the provisions of the Internal Revenue Code, the Company’s net operating loss and tax credit carry forwards are subject
to review and possible adjustment by the Internal Revenue Service and state tax authorities. Net operating loss and tax credit
carry forwards may become subject to an annual limitation in the event of certain cumulative changes in the ownership interest
of significant shareholders over a three-year period in excess of 50 percent, as defined under Sections 382 and 383 of the
Internal Revenue Code, respectively, as well as similar state provisions. This could limit the amount of tax attributes that can be
utilized annually to offset future taxable income or tax liabilities. The amount of the annual limitation is determined based on
the value of the Company immediately prior to the ownership change. Since its inception, the Company has completed several
financings and sales of common stock which have resulted in a change in control as defined by Sections 382 and 383 of the
Internal Revenue Code. Subsequent ownership changes may further affect the limitation in future years.
For fiscal years through June 30, 2017, the Company generated research credits but has not conducted a study to document the
qualified activities. This study may result in an adjustment to the Company’s research and development tax credit carry
forwards; however, until a study is completed and any adjustment is known, no amounts are being presented as an uncertain tax
position at June 30, 2017 or 2016. A full valuation allowance has been provided against the Company’s research and
development tax credits and, if an adjustment were to be required, this adjustment would be offset by an adjustment to the
deferred tax asset established for the tax credit carry forwards and the valuation allowance.
The Company files income tax returns in the United States and in multiple states. The federal and state returns are generally
subject to tax examinations for the tax years ended June 30, 2013 through June 30, 2017. To the extent the Company has tax
attribute carry forwards, the tax years in which the attribute was generated may still be adjusted upon examination by the
Internal Revenue Service, or state authorities, to the extent such attributes are utilized in a future period. On December 28,
2015, the United States Internal Revenue Service, or IRS, notified the Company of an income tax audit for the tax period ending
June 30, 2014. As of June 30, 2017, the IRS audit was closed and the Company incurred no penalties or payment liabilities for
its income tax positions.
106
APPLIED GENETIC TECHNOLOGIES CORPORATION
NOTES TO FINANCIAL STATEMENTS— (Continued)
FOR THE YEARS ENDED JUNE 30, 2017, 2016 AND 2015
The Company recognizes a tax benefit from an uncertain tax position when it is more likely than not that the position will be
sustained upon examination. For the year ended June 30, 2017, the Company recorded an uncertain tax position reserve of
$950,000. No similar reserve was recorded for the years ended June 30, 2016 and 2015. The entire amount of the reserve would
reduce the annual effective tax rate if recognized. The Company does not anticipate that the amount of unrecognized tax benefits
as of June 30, 2017 will significantly change within the next twelve months. The Company’s practice is to recognize interest
and/or penalties related to uncertain income tax positions in income tax expense. The Company had no interest and/or penalties
accrued on the Company’s balance sheets at June 30, 2017 and 2016, and the Company did not recognize any interest and/or
penalties in the statement of operations for the years ended June 30, 2017, 2016 and 2015 related to uncertain tax positions.
The following table provides a reconciliation of changes in unrecognized tax benefits (in thousands):
Balance at beginning of period
Additions related to current period tax positions
Additions related to prior period tax positions
Reductions related to prior period tax positions
Reductions related to lapse of statute of limitations
Balance at end of period
2017
Years Ended June 30,
2016
2015
$
$
— $
950
—
—
—
950 $
— $
—
—
—
—
— $
—
—
—
—
—
—
(11) Accrued Expenses:
Accrued expenses as of June 30, 2017 and 2016 consisted of the following:
In thousands
Research and development-related
Compensation-related
Accrued tax liabilities
(12) Defined Contribution Plan:
At June 30,
2017
2016
$
$
2,868 $
1,780
1,514
6,162 $
4,923
1,591
—
6,514
The Company sponsors an employee 401(k) salary deferral plan (“401(k) Plan”) that covers substantially all of its employees and is
administered through its staff leasing company. Under the 401(k) Plan, employees may elect to defer up to 25% of their
compensation per year (subject to a maximum limit prescribed by federal tax law) and the Company matches a portion of such
employee contributions up to a maximum of 4% of the eligible salary. The Company’s matching contributions to the 401(k) Plan
amounted to $235,000, $162,000 and $90,000 for each of the fiscal years ended June 30, 2017, 2016 and 2015, respectively.
(13) Common Stock and Stockholders’ Equity:
Common Stock
In connection with its entry into the Collaboration Agreement with Biogen described in Note 7 of these financial statements, the
Company also entered into an equity agreement with Biogen on July 1, 2015. Under the terms of this equity agreement, Biogen
purchased 1,453,957 shares of the Company’s common stock, at a price of $20.63 per share, for an aggregate cash purchase
price of $30.0 million. These cash proceeds were received from Biogen in August 2015. The shares issued to Biogen constitute
restricted securities that may not be resold by Biogen other than in a transaction registered under the Securities Act of 1933, as
amended, or pursuant to an exemption from such registration requirement.
As of June 30, 2017, there were 150,000,000 shares of $0.001 par value common stock that were authorized to be issued. As of
that date, a total of 18,087,785 shares of common stock were issued and outstanding.
107
APPLIED GENETIC TECHNOLOGIES CORPORATION
NOTES TO FINANCIAL STATEMENTS— (Continued)
FOR THE YEARS ENDED JUNE 30, 2017, 2016 AND 2015
The following shares of common stock were reserved for future issuance as of June 30, 2017:
In thousands
Common stock warrants
Stock options issued and outstanding
Authorized for future grant under the 2013 Employee Stock Purchase Plan
Authorized for future grant under the 2013 Equity and Incentive Plan
June 30, 2017
3
2,716
129
546
3,394
(14) Quarterly Financial Information (Unaudited):
Summarized quarterly information for the two fiscal years ended June 30, 2017 and 2016, respectively, is as follows:
In thousands, except per share data
Revenue
Income (loss) from operations
Net income (loss)
Net earnings (loss) per common share, basic
Net earnings (loss) per common share, diluted
In thousands, except per share data
Revenue
Income (loss) from operations
Net income (loss)
Net earnings (loss) per common share, basic
Net earnings (loss) per common share, diluted
First
Fiscal Year 2017 by Quarter:
Third
Second
Fourth
11,806 $
3,625 $
3,025 $
0.17 $
0.16 $
10,933 $
2,379 $
1,779 $
0.10 $
0.10 $
8,388 $
(600) $
(1,200) $
(0.07) $
(0.07) $
8,345
(2,597)
(3,197)
(0.18)
(0.18)
First
Fiscal Year 2016 by Quarter:
Third
Second
Fourth
11,062 $
(9,213) $
(9,123) $
(0.53) $
(0.53)
12,189 $
2,870 $
3,045 $
0.17 $
0.17
11,997 $
1,767 $
1,969 $
0.11 $
0.11
12,113
2,487
2,728
0.15
0.15
$
$
$
$
$
$
$
$
$
$
108
ITEM 9.
CHANGES IN AND DISAGREEMENTS WITH ACCOUNTANTS ON ACCOUNTING AND FINANCIAL
DISCLOSURE
None.
ITEM 9A. CONTROLS AND PROCEDURES
Disclosure Controls and Procedures
We maintain disclosure controls and procedures that are designed to ensure that information required to be disclosed in our
reports under the Securities Exchange Act of 1934, as amended (the “Exchange Act”), is recorded, processed, summarized and
reported within the time periods specified in the rules and forms, and that such information is accumulated and communicated to us,
including our Chief Executive Officer and Chief Financial Officer, as appropriate, to allow timely decisions regarding required
disclosure. In designing and evaluating our disclosure controls and procedures, we recognize that any controls and procedures, no
matter how well designed and operated, can provide only reasonable assurance of achieving the desired control objectives, as ours are
designed to do, and we necessarily were required to apply our judgment in evaluating whether the benefits of the controls and
procedures that we adopt outweigh their costs.
As required by Rule 13a-15(b) under the Exchange Act, an evaluation of the effectiveness of our disclosure controls and
procedures (as defined in Rule 13a-15(e) under the Exchange Act) as of June 30, 2017 was conducted under the supervision and with
the participation of our management, including our Chief Executive Officer and Chief Financial Officer. As a result of the material
weakness in internal control over financial reporting relating to the design and operation of our closing and financial reporting
processes disclosed below, our Chief Executive Officer and Chief Financial Officer have concluded that our disclosure controls and
procedures were not effective at the reasonable assurance level as of June 30, 2017.
Management’s Report on Internal Control Over Financial Reporting
Management is responsible for establishing and maintaining adequate “internal control over financial reporting,” as such term is
defined under Rule 13a-15(f) of the Exchange Act. We maintain internal control over financial reporting 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. Internal control over financial reporting includes maintaining records that
in reasonable detail accurately and fairly reflect our transactions; providing reasonable assurance that transactions are recorded as
necessary for preparation of our financial statements; providing reasonable assurance that receipts and expenditures of our assets are
made in accordance with management’s authorization; and providing reasonable assurance that unauthorized acquisition, use or
disposition of our assets that could have a material effect on the financial statements would be prevented or detected on a timely basis.
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. Therefore, internal control
over financial reporting determined to be effective provides only reasonable assurance regarding the reliability of financial reporting
and the preparation of financial statements for external purposes in accordance with generally accepted accounting principles.
Management assessed the effectiveness of the Company’s internal control over financial reporting as of June 30, 2017. In
making this assessment, management used the criteria set forth by the Committee of Sponsoring Organizations of the Treadway
Commission (COSO) in Internal Control — Integrated Framework (2013). Based on this assessment, management has concluded
that, as of June 30, 2017, a material weakness in internal control over financial reporting relating to the design and operation of our
closing and financial reporting processes still existed and, as a result, the Company did not maintain effective internal control over
financial reporting as of June 30, 2017. Notwithstanding this material weakness in internal control over financial reporting relating to
the design and operation of our closing and financial reporting processes, our management has concluded that the financial statements
included in this Annual Report on Form 10-K present fairly, in all material respects, our financial position, results of operations and
cash flows for the periods presented in conformity with accounting principles generally accepted in the United States.
We are not required to comply with the auditor attestation requirement of Section 404 of the Sarbanes-Oxley Act while we
qualify as an “emerging growth company” as defined in the JOBS Act. Subject to certain limitations, we expect to remain an
emerging growth company under the JOBS Act for up to five years from April 1, 2014, the date of our initial public offering.
Material Weaknesses in Internal Control over Financial Reporting
Management previously identified and disclosed a material weakness in our internal control over financial reporting at June 30,
2016 which related to the design and operation of our closing and financial reporting processes. Specifically, management concluded
109
that this material weakness in our internal control over financial reporting was due to the fact that we did not have the appropriate
resources with the appropriate level of experience and technical expertise to oversee our closing and financial reporting processes.
In order to address this material weakness, we took the following actions in fiscal year 2017:
(cid:129)
(cid:129)
(cid:129)
(cid:129)
we hired an additional employee during the second half of fiscal year 2017 to provide further support to our finance and
accounting team. The finance and accounting team now comprises of a total of eight employees, including our Chief
Financial Officer and our Controller;
we restructured our finance team to better align the functional areas to the overall strategy of the company, while at the
same time providing more focus for the accounting team in maintaining proper control over the financial reporting process
commensurate to support standalone external financial reporting under public company or SEC requirements;
we continued to provide functional and system training to employees and to prepare detailed documentation to clearly
define key tasks and actions, as well as the positions responsible for those tasks and actions. During fiscal year 2017, we
also engaged the services of a consulting firm to assist in documenting and formalizing our accounting policies and
internal control processes and to help strengthen supervisory reviews by our management; and
we continued to design and implement monthly manual controls to manage our financial reporting close processes and to
help ensure an adequate level of segregation of duties within our finance and accounting function. We are also designing
additional controls around identification, documentation and application of technical accounting guidance with particular
emphasis on events outside the ordinary course of business. These controls include the implementation of additional
supervision and review activities by qualified personnel, the preparation of formal accounting memoranda to support our
conclusions on technical accounting matters, and the development and use of checklists and research tools to assist in
compliance with GAAP with regard to complex accounting issues.
We have completed the documentation and review of the corrective actions described above and, as of June 30, 2017, our
management has concluded that there still existed significant flaws in the design and operation of our closing and financial reporting
processes and therefore that this previously identified material weakness had not been fully remediated as of June 30, 2017. In order
to fully remediate this material weakness, we will continue to implement the corrective actions described above, including additional
procedures to improve the capture, review, approval, and recording of all transactions in the appropriate accounting period.
Management will also continue to review and make necessary changes to the overall design of our internal control environment, and
implement policies and procedures that improve the overall effectiveness of our internal controls over financial reporting.
Changes in Internal Control over Financial Reporting
Except for those remedial actions described above, there was no change in our internal control over financial reporting identified
in connection with the evaluation required by Rule 13a-15(d) and 15d-15(d) of the Exchange Act that occurred during the period
covered by this report that has materially affected, or is reasonably likely to materially affect, our internal control over financial
reporting.
ITEM 9B. OTHER INFORMATION
None.
PART III
ITEM 10. DIRECTORS, EXECUTIVE OFFICERS AND CORPORATE GOVERNANCE
The information required by this item will be set forth under the captions “Election of Directors,” “Executive Officers,” “Code
of Ethics,” “Directors—Audit Committee Financial Expert” and “Corporate Governance” in an amendment to our Annual Report on
Form 10-K/A to be filed with the Securities and Exchange Commission no later than 120 days after the end of our fiscal year (the
“Annual Report Amendment”), and is incorporated herein by reference.
We are also required under Item 405 of Regulation S-K to provide information concerning delinquent filers of reports under
Section 16 of the Securities and Exchange Act of 1934, as amended. This information will be set forth under the caption “Section
16(a) Beneficial Ownership Reporting Compliance” in our Annual Report Amendment, and is incorporated herein by reference.
110
ITEM 11.
EXECUTIVE COMPENSATION
The information required by this item will be set forth under the captions “Executive Officers—Executive Compensation” in our
Annual Report Amendment and is incorporated herein by reference.
ITEM 12.
SECURITY OWNERSHIP OF CERTAIN BENEFICIAL OWNERS AND MANAGEMENT AND RELATED
STOCKHOLDER MATTERS
The information required by Item 403 of Regulation S-K will be set forth under the caption “Security Ownership of Certain
Beneficial Owners and Management” in our Annual Report Amendment, and is incorporated herein by reference.
We have two equity compensation plans under which awards are currently authorized for issuance, our 2013 Equity and
Incentive Plan and our 2013 Employee Stock Purchase Plan. In connection with the consummation of our initial public offering in
April 2014, our board of directors terminated any new offerings under our 2001 Stock Option Plan and our 2011 Stock Incentive Plan.
Each of our 2013 Equity and Incentive Plan, our 2013 Employee Stock Purchase Plan, our 2001 Stock Option Plan and our 2011
Stock Incentive Plan was approved by our stockholders prior to our initial public offering in 2014. The following table provides
information regarding securities authorized for issuance as of June 30, 2017 under our equity compensation plans.
Plan category
Equity compensation plans approved by security holders
Equity compensation plans not approved by
security holders
Total
2,714,602 $
— $
2,714,602 $
12.96
—
12.96
Number of securities to be
issued upon exercise of
outstanding options,
warrants and rights, and Weighted-average exercise
vesting of outstanding
restricted stock units
(a)
warrants and rights
(b)
price of outstanding options, (excluding securities reflected
Number of securities
remaining available for
future issuance under equity
compensation plans
in column (a))
(c)
672,197 (1)
—
672,197
(1)
Includes 543,626 shares issuable under our 2013 Equity and Incentive Plan, which may be issued in the form of options,
restricted stock, unrestricted stock, performance share awards or other equity-based awards, and 128,571 shares issuable under
our 2013 Employee Stock Purchase Plan.
ITEM 13. CERTAIN RELATIONSHIPS AND RELATED TRANSACTIONS, AND DIRECTORS INDEPENDENCE
The information required by this item will be set forth under the caption “Executive Officers—Certain Relationships and
Related Transactions” and “Corporate Governance” in our Annual Report Amendment, and is incorporated herein by reference.
ITEM 14.
PRINCIPAL ACCOUNTANT FEES AND SERVICES
The information required by this item will be set forth under the caption “Independent Registered Public Accounting Firm” in
our Annual Report Amendment, and is incorporated herein by reference.
ITEM 15.
EXHIBITS AND FINANCIAL STATEMENT SCHEDULES
(a) Documents filed as a part of this Report:
PART IV
(1) Financial Statements —See Index to Financial Statements and Financial Statement Schedule at Item 8 on page 104 of this
Annual Report on Form 10-K.
(2) Financial Statement Schedules —See Index to Financial Statements and Financial Statement Schedule at Item 8 on
page 104 of this Annual Report on Form 10-K. All other schedules are omitted because they are not applicable or not required.
111
(3) Index to Exhibits.
Exhibit
number
Description
3.1
3.2
4.1
10.1
10.2*
10.3*
10.4*
10.5*
10.6*
10.7†
10.8
10.9†
10.10†
10.11†
10.12†
Fifth Amended and Restated Certificate of Incorporation of the Company (incorporated by reference to Exhibit 3.1 to the
Company’s Current Report on Form 8-K filed with the SEC on April 1, 2014)
Amended and Restated Bylaws of the Company (incorporated by reference to Exhibit 3.2 to the Company’s Current
Report on Form 8-K filed with the SEC on April 1, 2014)
Specimen certificate evidencing shares of common stock (incorporated by reference to Exhibit 4.1 to the Company’s
Registration Statement on Form S-1 (File No. 333-193309))
Lease Agreement made as of April 10, 2015, by and between Alachua Foundation Park Holding Company, LLC and
Applied Genetic Technologies Corporation (incorporated by reference to Exhibit 10.1 to the Company’s Annual Report
on Form 10-K for the year ending June 30, 2015 (File No. 001-36370))
Employment Agreement dated as of May 27, 2015 between Applied Genetic Technologies Corporation and Mark S.
Shearman (incorporated by reference to Exhibit 10.2 to the Company’s Annual Report on Form 10-K for the year ending
June 30, 2015 (File No. 001-36370))
Employment Agreement dated as of January 29, 2015 between Applied Genetic Technologies Corporation and Stephen
W. Potter (incorporated by reference to Exhibit 10.1 to the Company’s Quarterly Report on Form 10-Q for the quarter
ended March 31, 2015 (File No. 001-36370))
Separation Agreement dated as of March 3, 2015 between Applied Genetic Technologies Corporation and Daniel L.
Menichella (incorporated by reference to Exhibit 10.1 to the Company’s Quarterly Report on Form 10-Q/A (Amendment
No. 1) for the quarter ended March 31, 2015 (File No. 001-36370))
Employment Agreement dated as of September 26, 2014 between Applied Genetic Technologies Corporation and
Susan B. Washer (incorporated by reference to Exhibit 10.1 to the Company’s Current Report on Form 8-K, event date
September 26, 2014, filed on October 2, 2014 (File No. 001-36370))
Employment Agreement dated as of September 26, 2014 between Applied Genetic Technologies Corporation and Jeffrey
D. Chulay (incorporated by reference to Exhibit 10.2 to the Company’s Current Report on Form 8-K, event date
September 26, 2014, filed on October 2, 2014 (File No. 001-36370))
Collaboration and License Agreement dated as of July 1, 2015 by and between Biogen MA Inc., and Applied Genetic
Technologies Corporation (incorporated by reference to Exhibit 10.7 to the Company’s Annual Report on Form 10-K for
the year ending June 30, 2015 (File No. 001-36370))
Common Stock Purchase Agreement dated as of July 1, 2015 by and between Biogen MA Inc., and Applied Genetic
Technologies Corporation (incorporated by reference to Exhibit 10.8 to the Company’s Annual Report on Form 10-K for
the year ending June 30, 2015 (File No. 001-36370))
Manufacturing License and Technology Transfer Agreement dated as of July 1, 2015 by and between Biogen MA Inc.,
and Applied Genetic Technologies Corporation (incorporated by reference to Exhibit 10.9 to the Company’s Annual
Report on Form 10-K for the year ending June 30, 2015 (File No. 001-36370))
Second Amendment to Non-exclusive License Agreement, made and effective as of June 29, 2015, by and between The
UAB Research Foundation, Inc. and Applied Genetic Technologies Corporation (incorporated by reference to Exhibit
10.10 to the Company’s Annual Report on Form 10-K for the year ending June 30, 2015 (File No. 001-36370))
Omnibus Amendment to Standard Exclusive License Agreement with Sublicensing Terms, made and effective as of July
1, 2015, by and between the University of Florida Research Foundation, Inc., the University of Florida Board of
Trustees, John Hopkins University and Applied Genetic Technologies Corporation (incorporated by reference to Exhibit
10.11 to the Company’s Annual Report on Form 10-K for the year ending June 30, 2015 (File No. 001-36370))
Omnibus Amendment to Standard Exclusive License Agreement with Know How and Standard Non-Exclusive License
Agreement, made and effective as of June 30, 2015, by and between the University of Florida Research Foundation, Inc.
and Applied Genetic Technologies Corporation (incorporated by reference to Exhibit 10.12 to the Company’s Annual
Report on Form 10-K for the year ending June 30, 2015 (File No. 001-36370))
112
Exhibit
number
10.13
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*
10.28
Description
Lease Agreement made as of September 19, 2011, by and between Thomson-Davis Enterprises, LLC and Applied
Genetic Technologies Corporation (incorporated by reference to Exhibit 10.1 to the Company’s Registration Statement
on Form S-1 (File No. 333-193309))
Exclusive License Agreement with Sublicensing Terms, effective as of September 25, 2001, by and between the
University of Florida Research Foundation, Inc. and Applied Genetic Technologies Corporation (incorporated by
reference to Exhibit 10.2 to the Company’s Registration Statement on Form S-1 (File No. 333-193309))
Restated Amendment to License Agreement made and, effective as of January 31, 2005, by and between the University
of Florida Research Foundation, Inc. and Applied Genetic Technologies Corporation (incorporated by reference to
Exhibit 10.3 to the Company’s Registration Statement on Form S-1 (File No. 333-193309))
First Amendment After Restated Amendment to License Agreement, made and effective as of November 28, 2007, by
and between the University of Florida Research Foundation, Inc. and Applied Genetic Technologies Corporation
(incorporated by reference to Exhibit 10.4 to the Company’s Registration Statement on Form S-1 (File No. 333-193309))
Standard Exclusive License Agreement with Sublicensing Terms, effective as of October 7, 2003, by and between the
University of Florida Research Foundation, Inc., Johns Hopkins University and Applied Genetic Technologies
Corporation (incorporated by reference to Exhibit 10.5 to the Company’s Registration Statement on Form S-1 (File No.
333-193309))
First Amendment to Standard Exclusive License Agreement with Sublicensing Terms, made as of November 2004, by
and between the University of Florida Research Foundation, Inc., Johns Hopkins University and Applied Genetic
Technologies Corporation (incorporated by reference to Exhibit 10.6 to the Company’s Registration Statement on Form
S-1 (File No. 333-193309))
Second Amendment to Standard Exclusive License Agreement with Sublicensing Terms, made as of February 25, 2009,
by and among Applied Genetic Technologies Corporation, the University of Florida Research Foundation, Inc. and
Johns Hopkins University (incorporated by reference to Exhibit 10.7 to the Company’s Registration Statement on Form
S-1 (File No. 333-193309))
Non-Exclusive License Agreement with Sublicensing Terms, made as of January 19, 2006, by and between The UAB
Research Foundation, Inc. and Applied Genetic Technologies Corporation (incorporated by reference to Exhibit 10.8 to
the Company’s Registration Statement on Form S-1 (File No. 333-193309))
Standard Non-Exclusive License Agreement, effective as of September 18, 2012, by and between the University of
Florida Research Foundation, Inc. and Applied Genetic Technologies Corporation (incorporated by reference to Exhibit
10.10 to the Registrant’s Registration Statement on Form S-1 (File No. 333-193309))
Standard Exclusive License Agreement with Know How, effective as of November 5, 2012, by and between the
University of Florida Research Foundation, Inc. and Applied Genetic Technologies Corporation (incorporated by
reference to Exhibit 10.11 to the Company’s Registration Statement on Form S-1 (File No. 333-193309))
Amended and Restated Investor Rights Agreement, dated as of November 15, 2012 (incorporated by reference to Exhibit
10.12 to the Company’s Registration Statement on Form S-1 (File No. 333-193309))
Applied Genetic Technologies Corporation 2001 Stock Option Plan, as amended (incorporated by reference to Exhibit
10.13 to the Company’s Registration Statement on Form S-1 (File No. 333-193309))
Applied Genetic Technologies Corporation 2011 Stock Incentive Plan, as amended, and forms of Incentive Stock Option
Agreement and Nonstatutory Stock Option Agreement thereunder (incorporated by reference to Exhibit 10.14 to the
Company’s Registration Statement on Form S-1 (File No. 333-193309))
Applied Genetic Technologies Corporation 2013 Equity And Incentive Plan (incorporated by reference to Exhibit 10.15
to the Company’s Registration Statement on Form S-1 (File No. 333-193309))
Applied Genetic Technologies Corporation 2013 Employee Stock Purchase Plan (incorporated by reference to Exhibit
10.16 to the Company’s Registration Statement on Form S-1 (File No. 333-193309))
Form of Applied Genetic Technologies Corporation Warrant to Purchase Shares of Series A-1 Preferred Stock
(incorporated by reference to Exhibit 10.17 to the Company’s Registration Statement on Form S-1 (File No. 333-
193309))
113
Exhibit
number
10.29
10.30
10.31
10.32
10.33
10.34
10.35
10.36†
10.37†
10.38*
10.39†
10.40*
10.41
23.1**
31.1**
31.2**
32.1**
Description
Form of Applied Genetic Technologies Corporation Warrant to Purchase Shares of Series B-1 Preferred Stock
(incorporated by reference to Exhibit 10.18 to the Company’s Registration Statement on Form S-1 (File No. 333-
193309))
Warrant to Purchase Shares of Series A-1 Preferred Stock of Applied Genetic Technologies Corporation issued to
Silicon Valley Bank and effective on September 23, 2005 (incorporated by reference to Exhibit 10.19 to the Company’s
Registration Statement on Form S-1 (File No. 333-193309))
Warrant to Purchase Shares of Series A-1 Preferred Stock of Applied Genetic Technologies Corporation issued to
Silicon Valley Bank and effective on June 30, 2006 (incorporated by reference to Exhibit 10.20 to the Company’s
Registration Statement on Form S-1 (File No. 333-193309))
Warrant to Purchase Shares of Series A-1 Preferred Stock of Applied Genetic Technologies Corporation issued to Square
1 Bank on July 6, 2010 (incorporated by reference to Exhibit 10.21 to the Company’s Registration Statement on Form S-
1 (File No. 333-193309))
Warrant to Purchase Shares of Series B-1 Preferred Stock of Applied Genetic Technologies Corporation issued to Square
1 Bank on August 31, 2012 (incorporated by reference to Exhibit 10.22 to the Company’s Registration Statement on
Form S-1 (File No. 333-193309))
Form of Indemnification Agreement for Directors Associated with an Investment Fund (incorporated by reference to
Exhibit 10.23 to the Company’s Registration Statement on Form S-1 (File No. 333-193309))
Form of Indemnification Agreement for Directors Not Associated with an Investment Fund (incorporated by reference to
Exhibit 10.24 to the Company’s Registration Statement on Form S-1 (File No. 333-193309))
Second Amendment After Restated Amendment to License Agreement, made and effective as of January 10, 2014, by
and between the University of Florida Research Foundation, Inc. and Applied Genetic Technologies Corporation
(incorporated by reference to Exhibit 10.25 to the Company’s Registration Statement on Form S-1 (File No. 333-
193309))
Fourth Amendment to Standard Exclusive License Agreement with Sublicensing Terms, made as of December 17, 2013
by and between the University of Florida Research Foundation, Inc., Johns Hopkins University and Applied Genetic
Technologies Corporation (incorporated by reference to Exhibit 10.26 to the Company’s Registration Statement on Form
S-1 (File No. 333-193309))
Letter Agreement dated July 22, 2013 by and between the Company and Dan Menichella (incorporated by reference to
Exhibit 10.26 to the Company’s Registration Statement on Form S-1 (File No. 333-197385))
First Amendment to Non-Exclusive License, made as of March 28, 2014, by and between the UAB Research
Foundation, Inc. and Applied Genetic Technologies Corporation (incorporated by reference to Exhibit 10.27 to the
Company’s Registration Statement on Form S-1 (File No. 333-197385))
Letter Agreement dated January 22, 2014 by and between the Company and Larry Bullock (incorporated by reference to
Exhibit 10.28 to the Company’s Registration Statement on Form S-1 (File No. 333-197385))
Employment Letter Agreement dated as of October 29, 2016 between Applied Genetic Technologies Corporation and
Michael Goldstein (incorporated by reference to Exhibit 10.1 to the Company’s Quarterly Report on Form 10-Q for the
quarter ended December 31, 2016 (File No. 001-36370))
Consent of Independent Registered Public Accounting Firm
Certification of Principal Executive Officer pursuant to Rule 13a-14(a) or Rule 15d-14(a) of the Securities Exchange Act
of 1934, as adopted pursuant to Section 302 of the Sarbanes-Oxley Act of 2002
Certification of Principal Financial Officer pursuant to Rule 13a-14(a) or Rule 15d-14(a) of the Securities Exchange Act
of 1934, as adopted pursuant to Section 302 of the Sarbanes-Oxley Act of 2002
Certification of Principal Executive Officer and Principal Financial Officer pursuant to 18 U.S.C. Section 1350, as
adopted pursuant to Section 906 of the Sarbanes-Oxley Act of 2002
114
Exhibit
number
Description
101.INS** XBRL Instance Document
101.SCH** XBRL Taxonomy Extension Schema Document
101.CAL** XBRL Taxonomy Extension Calculation Linkbase Document
101.DEF** XBRL Taxonomy Extension Definition Linkbase Document
101.LAB** XBRL Taxonomy Extension Label Linkbase Document
101.PRE** XBRL Taxonomy Extension Presentation Linkbase Document
*
**
†
Management contract or compensatory plan or arrangement
Filed herewith
We have omitted portions of this exhibit, for which confidential treatment has been granted.
115
SCHEDULE II—VALUATION AND QUALIFYING ACCOUNTS
In thousands
Deferred Tax Valuation Allowance
Year 2017
Year 2016
Year 2015
Additions
Charge
To (from)
Beginning
of Period
(Benefit) to
Expenses
Other
Accounts
Deductions
End of
Period
$
$
$
37,412 $
36,845 $
24,086 $
2,891 $
567 $
12,759 $
— $
— $
— $
— $
— $
— $
40,303
37,412
36,845
116
Pursuant to the requirements of Section 13 or 15(d) of the Securities Exchange Act of 1934, the registrant has duly caused this
report to be signed on its behalf by the undersigned thereunto duly authorized.
SIGNATURES
APPLIED GENETIC TECHNOLOGIES CORPORATION
By:
Date:
/s/ Susan B. Washer
Susan B. Washer
President and Chief Executive Officer
September 13, 2017
Pursuant to the requirements of the Securities Exchange Act of 1934, this Annual Report has been signed below by the
following persons on behalf of the Company and in the capacities and on the dates indicated.
Signature
Title
Date
/s/ Susan B. Washer
Susan B. Washer
/s/William A. Sullivan
William A. Sullivan
/s/ Scott Koenig
Scott Koenig
/s/ David Guyer
David Guyer
/s/ Ed Hurwitz
Ed Hurwitz
/s/ Ivana Magovcevic-Liebisch
Ivana Magovcevic-Liebisch
/s/ Anne VanLent
Anne VanLent
/s/ James Rosen
James Rosen
Chief Executive Officer, President and
Director (Principal Executive Officer)
September 13, 2017
Chief Financial Officer (Principal Financial and
Accounting Officer)
September 13, 2017
September 13, 2017
September 13, 2017
September 13, 2017
September 13, 2017
September 13, 2017
September 13, 2017
Director
Director
Director
Director
Director
Director
117
UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, DC 20549
FORM 10-K/A
(Amendment No. 1)
(Mark One)
È ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE
ACT OF 1934
‘ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES
For the Fiscal Year Ended June 30, 2017
OR
EXCHANGE ACT OF 1934
Commission File Number: 001-36370
APPLIED GENETIC TECHNOLOGIES
CORPORATION
(Exact Name of Registrant as Specified in Its Charter)
Delaware
(State or Other Jurisdiction of
Incorporation or Organization)
59-3553710
(I.R.S. Employer
Identification No.)
14193 NW 119th Terrace
Suite 10
Alachua, Florida 32615
(Address of Principal Executive Offices, Including Zip Code)
(386) 462-2204
(Registrant’s Telephone Number, Including Area Code)
Securities registered pursuant to Section 12(b) of the Act:
Title of class
Common Stock, $.001 par value
Name of exchange on which registered
NASDAQ Global Market
Securities registered pursuant to Section 12(g) of the Act: None
Indicate by check mark if the registrant is a well-known seasoned issuer, as defined in Rule 405 of the Securities
Act. Yes ‘ No È
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 È
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 È No ‘
Indicate by check mark whether the registrant has submitted electronically and posted on its corporate Web site, if any, every
Interactive Data File required to be submitted and posted 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 and post such files). Yes È No ‘
Indicate by check mark if disclosure of delinquent filers pursuant to Item 405 of Regulation S-K (§ 229.405 of this chapter) is not
contained herein, and will not be contained, to the best of registrant’s knowledge, in definitive proxy or information statements
incorporated by reference in Part III of this Form 10-K or any amendment to this Form 10-K. È
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. (Check one):
Large accelerated filer ‘
Non-accelerated filer ‘
È
Accelerated filer
Smaller reporting company ‘
Emerging growth company È
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. È
Indicate by check mark whether the registrant is a shell company (as defined in Rule 12b-2 of the Exchange Act). Yes ‘ No È
The aggregate market value of the voting common shares held by non-affiliates of the registrant was approximately $154.8 million,
computed by reference to the closing sale price of the common stock as reported by The NASDAQ Global Market on December 31, 2016,
the last trading day of the registrant’s most recently completed second fiscal quarter. The Company has no non-voting common shares.
As of October 15, 2017, a total of 18,093,235 shares of the registrant’s common stock, $0.001 par value per share, were outstanding.
DOCUMENTS INCORPORATED BY REFERENCE
None.
EXPLANANTORY NOTE
This Amendment No. 1 to Form 10-K (this “Amendment”) amends the Annual Report on Form 10-K for the
fiscal year ended June 30, 2017 originally filed on September 13, 2017 (the “Original Filing”) by Applied
Genetic Technologies Corporation (“AGTC”, the “Company”, “we”, or “us”). We are filing this Amendment to
present the information required by Part III of Form 10-K as we will not file our definitive proxy statement
within 120 days of the end of our fiscal year ended June 30, 2017.
Except as described above, this Amendment does not amend, update or change any other items or
disclosures in the Original Filing, and accordingly, should be read in conjunction with the Original Filing. As
required by Rule 12b-15 under the Securities and Exchange Act of 1934, as amended (the “Exchange Act”), new
certifications by our principal executive officer and our principal financial officer are filed as exhibits to this
Amendment under Item 15 of Part IV hereof.
APPLIED GENETIC TECHNOLOGIES CORPORATION
ANNUAL REPORT ON FORM 10-K
FOR FISCAL YEAR ENDED JUNE 30, 2017
TABLE OF CONTENTS
PART III
Item 10. Directors, Executive Officers and Corporate Governance . . . . . . . . . . . . . . . . . . . . . . . . . . .
Item 11. Executive Compensation . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
Item 12. Security Ownership of Certain Beneficial Owners and Management and Related
Stockholder Matters . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
Item 13. Certain Relationships and Related Transactions, and Director Independence . . . . . . . . . . . .
Item 14. Principal Accounting Fees and Services . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
PART IV
Item 15. Exhibits and Financial Statement Schedules
. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
SIGNATURES . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
Page
1
6
15
18
20
21
26
PART III
ITEM 10. DIRECTORS, EXECUTIVE OFFICERS AND CORPORATE GOVERNANCE
The following biographical descriptions set forth certain information with respect to our directors and our
executive officers who are not directors.
Name
Age
Position
. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
Susan Washer
William Sullivan . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
Stephen Potter . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
Mark Shearman, Ph.D.
. . . . . . . . . . . . . . . . . . . . . . . .
Andrew Ashe . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
Matthew Feinsod, M.D. . . . . . . . . . . . . . . . . . . . . . . . .
David R. Guyer, M.D. (2) . . . . . . . . . . . . . . . . . . . . . .
Ed Hurwitz (3) . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
Scott Koenig, M.D., Ph.D. (1) . . . . . . . . . . . . . . . . . . .
Ivana Magovcevic-Liebisch, Ph.D. (1) (3) . . . . . . . . .
James Rosen (1) . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
Anne VanLent (3) . . . . . . . . . . . . . . . . . . . . . . . . . . . .
President, Chief Executive Officer and Director
Interim Chief Medical Officer
56
46 Chief Financial Officer
61 Chief Business Officer
56 Chief Scientific Officer
51 General Counsel
47
57 Director
53 Director
65 Chairman of the Board of Directors
50 Director
48 Director
69 Director
(1) Member of the Compensation Committee.
(2) Member of the Nominating and Corporate Governance Committee.
(3) Member of the Audit Committee.
Executive Officers
Susan Washer has served as our president and chief executive officer since March 2002 and as a member of
our board of directors since November 2003. Prior to becoming our president and chief executive officer,
Ms. Washer served as our chief operating officer from October 2001 to March 2002. From August 1996 to
October 2001, Ms. Washer was president and chief executive officer of Scenic Productions Inc., a specialty
construction firm providing sculpting, painting and construction services to the entertainment industry. From
June 1994 to August 1996, Ms. Washer served as the Founding Executive Director and then Business Advisor for
the North Florida Technology Innovation Center, a public-private organization financing and providing services
to entrepreneurial companies licensing technology from Florida universities. From October 1983 to June 1994,
Ms. Washer served in various research and pharmaceutical management positions with Abbott Laboratories and
Eli Lilly and Company. Ms. Washer received a B.S. in biochemistry from Michigan State University and an
M.B.A. from the University of Florida. We believe that Ms. Washer’s education and professional background in
science and business management, her years of experience in the pharmaceutical and biotechnology industries,
her service as a senior executive of entrepreneurial companies and her extensive knowledge of our company and
its business qualify her to serve as a member of our board of directors.
William Sullivan has served as our chief financial officer since August 2017. Prior to joining AGTC,
Mr. Sullivan worked at Merrimack Pharmaceuticals Inc. from November 2007 to April of 2017 where he held a
number of positions of increasing responsibility including controller, vice president of finance, treasurer, chief
financial officer and head of finance. Previously, Mr. Sullivan served as corporate controller of Vette Corp., a
thermal management solutions company, from 2004 to 2007. Mr. Sullivan began his career at Arthur Andersen
LLP, where he obtained his certified public accountant license. Mr. Sullivan holds an M.B.A. and an M.S. in
accounting from Northeastern University’s Graduate School of Professional Accounting and a B.A. from
Williams College.
Stephen Potter has served as our vice president and chief business officer since January 2015. Prior to
joining us, Mr. Potter was employed most recently by NeoStem, Inc., a developer of cell-based therapeutics,
1
where he served as Executive Vice President from July 2013 to January 2015, and was a member of the Board of
Directors from January 2013 to July 2013. Previously, Mr. Potter was Senior Vice President of Operations and
Corporate Development for Osiris Therapeutics, Inc., from February 2011 to November 2012, where he was
managed the bio-surgery business unit and also had operational oversight for multiple functional areas including
manufacturing, human resources, IT, legal, and business development. From 2006 through 2010, Mr. Potter
served as Senior Vice President of Corporate and Business Development at Genzyme Corporation and as Vice
President of Corporate and Business Development. Mr. Potter has also held positions at DuPont Pharmaceuticals,
E.I. Dupont de Nemours and Company, Inc., and Booz Allen & Hamilton. Mr. Potter earned a B.S. from
University of Massachusetts and an M.B.A. from Harvard Business School.
Mark Shearman Mark Shearman has served as our chief scientific officer since June 1, 2015. From August
2009 until June 2015, Dr. Shearman served as Senior Vice-President of Research & Early Development of EMD
Serono, Inc., the U.S. and Canadian subsidiary of Merck KGaA. Prior his time at EMD Serono, Dr. Shearman
was Executive Director of Merck & Co. Research Laboratories, Boston, from January 2006 to July 2009 and
Senior Director at the Merck Sharp & Dohme Research Laboratories Neuroscience Research Centre, U.K. from
January 2004 to December 2005. Dr. Shearman earned a B.Sc. from the University of Bristol, a Ph.D. from the
University of Nottingham and conducted academic research at institutes in Japan and Germany.
Andrew Ashe has served as our General Counsel since August 2017. From 2003 to 2016, Mr. Ashe served
in various leadership positions at Dyax Corp., most recently serving as Executive Vice President, Administration,
Corporate Secretary and General Counsel. During his tenure at Dyax, Mr. Ashe managed various operational and
administrative functions, including legal, business development, human resources, IT and facilites. Previously,
Mr. Ashe was a member of the business law practices at Prince, Lobel & Tye LLP, and Nutter, McClennen &
Fish LLP. He has also served as a Trading Specialist and Senior Analyst of Corporate Listings for the New
York and American Stock Exchanges. He holds a B.A. in Finance from the Isenberg School of Management,
University of Massachusetts and a J.D. from George Washington University.
Matthew Feinsod, M.D. has served as our interim Chief Medical Officer since September 2017.
Dr. Feinsod, a board-certified ophthalmologist, joined AGTC in July 2014 and has played key roles in
developing and implementing clinical and regulatory strategy, due diligence and licensing. Prior to joining
AGTC, Dr. Feinsod co-founded and led Imagen Biotech, a venture-backed company dedicated to developing
ophthalmology treatments for sight-threatening diseases, from 2011 to 2013. Prior to Imagen, Dr. Feinsod served
in various roles including Senior Vice President of Strategy and Product Development at Eyetech
Pharmaceuticals from 2003 to 2007, during which time he helped to develop and launch Macugen. Dr. Feinsod
served as a medical officer in the ophthalmology division of the U.S. Food and Drug Administration
(FDA) from 2002 to 2003. He holds a B.S. from the Wharton School of Business at the University of
Pennsylvania and an M.D. from the George Washington University School of Medicine.
Directors
David R. Guyer, M.D. has served as a member of our board of directors since June 2014. Dr. Guyer has
served as Chairman of the board of directors of Ophthotech Corporation since January 2007 and served as Chief
Executive Officer of Ophthotech from April 2013 until July 2017. Dr. Guyer, served as a Partner at SV Life
Sciences, a venture capital firm, from December 2009 to April 2013, and as a Venture Partner at SV Life
Sciences from May 2006 to December 2009. In April 2013, Dr. Guyer resumed his role as Venture Partner at SV
Life Sciences Advisers, LLC. Dr. Guyer co-founded Eyetech Pharmaceuticals Inc. and served as Chief Executive
Officer and as a member of its board of directors from 2000 to 2006. Prior to co-founding Eyetech
Pharmaceuticals, Dr. Guyer was a Professor and served as Chairman of the Department of Ophthalmology at
New York University School of Medicine. Dr. Guyer received a B.S. from Yale College and an M.D. from Johns
Hopkins Medical School. Dr. Guyer completed his ophthalmology residency at Wilmer Ophthalmological
Institute, Johns Hopkins Hospital and a retinal fellowship at the Massachusetts Eye and Ear Infirmary at Harvard
Medical School. We believe that Dr. Guyer’s extensive experience in developing and commercializing
2
ophthalmologic therapies and his service as an executive officer and director of other biotechnology companies
qualify him to serve as a member of our board of directors.
Ed Hurwitz has served as a member of our board of directors since November 2012. Mr. Hurwitz is a
Managing Director of MPM Capital, a healthcare venture capital firm, and a Managing Director of Precision
Bioventures, LLC, a consulting and investment advisory firm founded by Mr. Hurwitz. He was a director at Alta
Partners from 2002 through December 2014, and served as a consultant to Alta Partners during 2013 and 2014.
Mr. Hurwitz currently serves as Chairman of the board of directors of ViewPoint Therapeutics, a privately-held,
biotechnology company, and as a member of the board of directors of MacroGenics, Inc. Prior to joining Alta,
Mr. Hurwitz served as Senior Vice President and CFO of Affymetrix from 1997 to 2002. From 1994 to 1997,
Mr. Hurwitz was a biotechnology research analyst for Robertson Stephens & Company, and from 1992 to 1994,
was a biotechnology research analyst for Smith Barney Shearson. From 1990 to 1992, he practiced commercial
law at Cooley Godward LLP. Mr. Hurwitz earned a J.D. and M.B.A. from the University of California,
Berkeley’s Boalt School of Law and Haas School of Business, respectively. He also holds a B.A. in Molecular
Biology from Cornell University. We believe that Mr. Hurwitz’s education and professional background in
science, business management and law, his work as a lawyer, research analyst and senior executive in the
biotechnology industry and his experience as a director of other public and private biotechnology companies
qualify him to serve as a member of our board of directors.
Scott Koenig, M.D., Ph.D. has served as a member of our board of directors since April 2002 and as
chairman of our board of directors since April 2004. Dr. Koenig has served as the President and Chief Executive
Officer and a director of MacroGenics, Inc., a publicly traded biopharmaceutical company, since September 2001
and was one of its co-founders. Prior to joining MacroGenics, Dr. Koenig served as Senior Vice President of
Research at MedImmune Inc., a biopharmaceutical company, where he participated in the selection and
maturation of its product pipeline. From 1984 to 1990, he worked in the Laboratory of Immunoregulation at the
National Institute of Allergy and Infectious Diseases at the National Institutes of Health, or NIH, where he
investigated the immune response to retroviruses and studied the pathogenesis of AIDS. Dr. Koenig currently
serves as a member of the board of directors of each of GlycoMimetics, Inc. (GLYC), The International
Biomedical Research Alliance, and the Biotechnology Industry Organization (BIO). Dr. Koenig received his
A.B. and Ph.D. from Cornell University and his M.D. from the University of Texas Health Science Center in
Houston. He completed his residency in Internal Medicine at the Hospital of the University of Pennsylvania, and
is Board certified in Internal Medicine and Allergy and Immunology. We believe that Dr. Koenig’s education
and professional background in science and medicine, his experience as chief executive officer of MacroGenics
and as a scientist and senior executive at other life science companies and research organizations and his service
as a director of other biopharmaceutical companies, medical institutions and industry groups qualify him to serve
as a member of our board of directors.
Ivana Magovcevic-Liebisch has served as a member of our board of directors since June 2014.
Dr. Magovcevic-Liebisch has served as Executive Vice President, Chief Strategy and Corporate Development
Officer for Axcella Health Inc. since May 2017. From April 2013 through May 2017, Dr. Magovcevic-Liebisch
served as Senior Vice President, Head of Global Business Development for Teva Pharmaceutical Industries Ltd.,
or Teva. Prior to joining Teva, Dr. Magovcevic-Liebisch held several senior positions within Dyax Corp., or
Dyax, from April 2001 through March 2013, most recently serving as Executive Vice President and Chief
Operating Officer. Prior to joining Dyax, Dr. Magovcevic-Liebisch was Director of Intellectual Property and
Patent Counsel for Transkaryotic Therapies, Inc. from November 1999 until March 2001. Dr. Magovcevic-
Liebisch received her J.D. from Suffolk University Law School and her Ph.D. in genetics from Harvard
University. We believe that Dr. Magovcevic-Liebisch’s extensive experience in biopharmaceutical business
development and operations qualify her to serve as a member of our board of directors.
James Rosen has served as a member of our board of directors since March 2010; he is currently
President & CEO of Artizan Biosciences. Artizan Biosciences is engaging in early-stage immunobiology
research and development for the treatment of unmet medical needs. From February 2015 through August 2016,
3
Mr. Rosen served as Deputy Director, Venture Investing at the Bill & Melinda Gates Foundation. Prior to that,
Mr. Rosen was a partner at Intersouth Partners, a venture capital firm, from January 2007 to December 2014.
Prior to joining Intersouth, he spent 15 years in clinical, research and financial positions in the health care and
biotechnology sectors, including serving as an equity research analyst at Brean Murray & Co., from 2000 to
2003, covering biopharmaceuticals, genomics, generics, drug delivery and medical device companies. Mr. Rosen
holds a B.A. from Duke University, an M.B.A. from the University of North Carolina-Chapel Hill’s Kenan-
Flagler School of Business and an M.S.P.H. from the University of North Carolina School of Public Health. We
believe that Mr. Rosen’s education and professional background in science, business management and finance
and his operational experience as a scientist and executive in the healthcare and biotechnology industries and as a
venture capitalist concentrating on those industries, qualify him to serve as a member of our board of directors.
Anne VanLent has served as a member of our board of directors and chair of the audit committee since
August 2016. Ms. VanLent is President of AMV Advisors, providing corporate strategy and financial consulting
services to emerging growth life sciences companies. Ms. VanLent had been Executive Vice President and Chief
Financial Officer of Barrier Therapeutics, Inc., a publicly traded pharmaceutical company that developed and
marketed prescription dermatology products, from May 2002 through April 2008. From July 1997 to October
2001, she was the Executive Vice President—Portfolio Management for Sarnoff Corporation, a multidisciplinary
research and development firm. From 1985 to 1993, she served as Senior Vice President and Chief Financial
Officer of The Liposome Company, Inc., a publicly-traded biopharmaceutical company. Ms. VanLent currently
serves as lead director, chair of the Audit Committee, and member of the Nominating and Governance
Committee of Aviragen Therapeutics, Inc.; and as a director, chair of the Audit Committee, and chair of the
Nominating and Governance Committee of Ocera Therapeutics, Inc. (formerly Tranzyme Pharma, Inc.), each a
NASDAQ-listed pharmaceuticals company. From April 2013 through June 2017 she served a director, member
of the Audit Committee (serving as Chair from April 2013 through 2016, and member of the Compliance
Committee of Novelion Pharmaceuticals, Inc. (previously Aegerion Pharmaceuticals, Inc.). From July 2013 to
May 2016, Ms. VanLent served as a director, chair of the Audit Committee, and member of the Compensation
Committee of Onconova Therapeutics, Inc., a NASDAQ-listed pharmaceuticals company; and was as a director
of Integra Life Sciences Holdings, Inc., a NASDAQ-listed medical device company, where she served as chair of
the Audit Committee from 2006 to 2012. Ms. VanLent received a B.A. degree in Physics from Mount Holyoke
College. Our Board of Directors believes that Ms. VanLent’s qualifications to sit on our Board of Directors
include her extensive leadership and finance experience, and her extensive experience serving as a board
member, audit committee member and audit committee chair of public companies in the life sciences industry.
CORPORATE GOVERNANCE
Code of Business Conduct and Ethics; Corporate Governance Guidelines
We have adopted a written code of business conduct and ethics that applies to our directors, executive
officers and employees, as well as corporate governance guidelines. Copies of the code of business conduct and
ethics and our corporate governance guidelines are posted on the Corporate Governance section of our website,
which is located at www.agtc.com. If we make any substantive amendments to, or grant any waivers from, the
code of business conduct and ethics for any officer or director, we will disclose the nature of such amendment or
waiver on our website.
Section 16(a) Beneficial Ownership Reporting Compliance
Section 16(a) of the Securities Exchange Act of 1934 requires our directors and executive officers, and
persons who beneficially own more than ten percent of a registered class of our equity securities, to file reports of
ownership of, and transactions in, our securities with the Securities and Exchange Commission. These directors,
executive officers and ten-percent stockholders are also required to furnish us with copies of all
Section 16(a) forms they file.
4
Based solely on a review of the copies of such forms received by us, and on written representations from
certain reporting persons, we believe that during fiscal year 2017 our directors, executive officers and ten-percent
stockholders complied with all applicable Section 16(a) filing requirements, except that Mr. Potter filed a late
Form 4 with respect to open market purchases of our common stock on September 16, 2016 and September 20,
2016
Audit Committee
The Company has a standing audit committee consisting of Ms. VanLent, its chairperson, Mr. Hurwitz and
Dr. Magovcevic-Liebisch. Among other things, the audit committee assists our board of directors in its oversight
of: the integrity of our financial statements; our compliance with legal and regulatory requirements; the
qualifications and independence of our independent registered public accounting firm; and the performance of
our independent registered public accounting firm. Our board of directors has determined that each member of
the audit committee satisfies the NASDAQ Stock Market independence standards and the independence
standards of Rule 10A-3(b)(1) of the Securities Exchange Act. Each of the members of our audit committee
meets the requirements for financial literacy under applicable rules and regulations of the SEC and the NASDAQ
Stock Market. The board of directors has also determined that Ms. VanLent qualifies as an “audit committee
financial expert,” as defined by applicable rules of the NASDAQ Stock Market and the SEC.
5
ITEM 11. EXECUTIVE COMPENSATION
Executive Summary
The compensation of our executive officers is determined by the compensation committee of our Board of
Directors, and discussed by the committee throughout the year. Our formal annual compensation review process
generally takes place during the first quarter of each fiscal year, after the results of the previous fiscal year are
known. Annual variable compensation and discretionary cash bonuses for the completed fiscal year, if any, and
long-term equity-based incentive compensation, if any, are awarded by the committee on a discretionary basis,
generally during the first fiscal quarter, after a review of the previous fiscal year’s results.
Our compensation committee is comprised entirely of non-employee Directors, each of whom our Board of
Directors has determined is independent within the meaning of the rules of The NASDAQ Stock Market. The
members of the compensation committee have substantial managerial experience and wide contacts in the
biotechnology and biopharmaceutical industries and in the broader healthcare industry, upon which they rely in
making their determinations. The committee also takes into account publicly available information concerning
the compensation practices of other companies in the biotechnology industry. This information is used by the
committee informally and primarily for purposes of comparison to ascertain whether our compensation practices
for our executive officers are broadly competitive.
Our Chief Executive Officer makes recommendations with regard to the compensation of our executive
officers other than herself, which are reviewed by the compensation committee. Executive officers do not
participate in the process of establishing their own annual compensation.
The committee does not have a formal benchmarking policy or a practice of establishing the amount of any
element of our executive officers’ compensation by reference to a fixed range of percentages or percentiles of the
compensation of any peer or comparison group. As a result, the determinations made by the members of our
compensation committee are guided to a significant degree by their collective judgment and experience. During
fiscal year 2017, the committee retained a compensation consultant, Aon Consulting’s Radford Surveys +
Consulting, or Radford, to assist the committee in assessing the form and amount of compensation paid to our
executives.
Our compensation committee has reviewed our compensation programs and believes that our compensation
programs have not encouraged or rewarded excessive or inappropriate risk taking.
6
Summary Compensation Table for Fiscal Year 2017
The following table sets forth information regarding compensation earned by our President and Chief
Executive Officer, our Chief Financial Officer and our three next most highly paid executive officers who served
during fiscal year 2017. We refer to these individuals as our named executive officers.
Name
Year
Salary ($)
Option
Awards
($)(1)
Bonus
($)(2)
Susan Washer . . . . . . . . . . . . . . . . . .
President and Chief Executive
2017
2016
487,000
464,000
853,391
1,030,046
Officer
Lawrence Bullock (4) . . . . . . . . . . . .
Chief Financial Officer
Jeffrey Chulay, M.D. (5) . . . . . . . . . .
Chief Medical Officer /
Executive Director of
Clinical Strategy
2017
2016
2017
2016
341,000
327,000
400,000
381,500
373,358
476,396
373,358
914,165
—
—
—
—
—
—
Non-equity
incentive
plan
compensation
($)
149,996
196,504
—
93,849
70,000
100,144
Other
($)(3)
Total ($)
8,161
8,507
1,498,548
1,699,057
6,648
6,816
11,098
11,291
721,006
904,061
854,456
1,407,100
Stephen Potter . . . . . . . . . . . . . . . . . .
Chief Business Officer
Mark Shearman, Ph.D.
. . . . . . . . . . .
Chief Scientific Officer
2017
2016
2017
2016
331,000
314,150
370,000
350,000
533,369
656,654
533,369
656,654
—
—
—
50,000
86,888
82,464
115,255
102,900
11,120
13,994
8,639
14,000
962,377
1,067,262
1,027,263
1,173,554
(1) Represents the grant date fair value of option awards granted in fiscal years 2016 and 2017 in accordance
with Accounting Standards Codification Topic 718, “Compensation—Stock Compensation” (“ASC 718”).
See Note 8 of the notes to our financial statements included in the Original Filing for a discussion of the
relevant assumptions used in calculating these amounts.
(2) Amount consists of a signing bonus paid to Dr. Shearman in connection with his initial employment.
(3) Consists of 401(k) matching contributions.
(4) Mr. Bullock retired in August 2017.
(5) Dr. Chulay served as our Chief Medical Officer until November 2016 and continues to serve as our
Executive Director of Clinical Strategy.
Narrative Disclosure to Summary Compensation Table
We review compensation annually for all of our employees, including our executives. In setting executive
base salaries and bonuses and granting equity incentive awards, we consider compensation for comparable
positions in the market, the historical compensation levels of our executives, individual performance as compared
to our expectations and objectives, our desire to motivate our employees to achieve short- and long-term results
that are in the best interests of our stockholders, and a long-term commitment to our company. We do not target a
specific competitive position or a specific mix of compensation among base salary, bonus or long-term
incentives.
Our compensation committee reviews and discusses management’s proposed compensation with the chief
executive officer for all executives other than our chief executive officer. Based on those discussions and its
discretion, the compensation committee then determines the compensation and benefits of our executive officers.
In fiscal year 2017, our compensation committee engaged Radford to assist us with the identification of an
appropriate peer group of companies for purposes of benchmarking the competitiveness of our executive
compensation. Our compensation committee will evaluate the need for revisions to our executive compensation
program to ensure that our program is competitive with the companies with which we compete for executive
talent and that it is appropriate for a public company.
7
Outstanding Equity Awards at Year End
The following table sets forth information regarding outstanding stock options held by our named executive
officers as of June 30, 2017.
Name
Susan Washer . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
Lawrence Bullock (2)
. . . . . . . . . . . . . . . . . . . . . . .
Jeffrey Chulay, M.D. . . . . . . . . . . . . . . . . . . . . . . . .
Stephen Potter . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
Mark Shearman, Ph.D. . . . . . . . . . . . . . . . . . . . . . . .
Number of
Number of
Securities
Securities
Underlying
Underlying
Unexercised
Unexercised
Options (#)
Options (#)
unexercisable
exercisable
—
19,541
—
3,877
—
120,686
8,750
131,250 (1)
78,498 (1) 20,658
40,672 (1) 15,108
38,333 (1) 41,667
18,333 (1) 61,667
83,766 (3) 16,754
417
2,085 (3)
50,665 (3) 10,134
17,729 (1) 19,271
8,020 (1) 26,980
—
2,142
—
10,511
2,539
38,072 (1)
4,007
15,223 (1)
16,953 (1)
6,297
34,020 (1) 36,980
8,020 (1) 26,980
79,175 (4) 51,874
24,437 (5) 26,563
11,458 (1) 38,542
55,000 (4) 55,000
24,437 (6) 26,563
11,458 (1) 38,542
Option
Exercise
Price ($)
3.50
3.50
0.35
4.90
14.08
16.00
18.48
15.57
12.00
14.08
16.00
18.48
15.57
3.50
0.35
4.90
14.08
16.00
18.48
15.57
24.62
18.48
15.57
19.50
18.48
15.57
Option
Expiration
Date
9/18/2019
11/1/2021
1/6/2023
9/18/2023
4/17/2024
7/31/2024
7/21/2025
7/7/2026
3/26/2024
4/17/2024
7/31/2024
7/21/2025
7/7/2026
9/18/2019
1/6/2023
9/18/2023
4/17/2024
7/31/2024
7/21/2025
7/7/2026
1/29/2025
7/21/2025
7/7/2026
6/1/2025
7/21/2025
7/7/2026
Option
Grant Date
9/18/2009
11/1/2011
1/6/2013
9/18/2013
4/17/2014
7/31/2014
7/21/2015
7/7/2016
3/26/2014
4/17/2014
7/31/2014
7/21/2015
7/7/2016
9/18/2009
1/6/2013
9/18/2013
4/17/2014
7/31/2014
7/21/2015
7/7/2016
1/29/2015
7/21/2015
7/7/2016
6/1/2015
7/21/2015
7/7/2016
(1) This option becomes exercisable in equal monthly installments over four years from the date of grant.
(2) Mr. Bullock retired in August 2017.
(3) This option became exercisable for 25% of the underlying shares on February 3, 2015, and thereafter
becomes exercisable in equal monthly installments over 36 months, resulting in the option being exercisable
for 100% of the underlying shares on February 3, 2018.
(4) This option becomes exercisable for 25% of the underlying shares on the first anniversary of the grant date,
and thereafter becomes exercisable for the remaining underlying shares in equal monthly installments over
three years, resulting in the option being exercisable for 100% of the underlying shares on the fourth
anniversary of the grant date.
(5) This option becomes exercisable for 6/48 of the underlying shares on January 21, 2016, and thereafter
becomes exercisable for the remaining underlying shares in equal monthly installments over four years from
the date of grant.
(6) This option becomes exercisable for 11/48 of the underlying shares on June 21, 2016, and thereafter
becomes exercisable for the remaining underlying shares in equal monthly installments over four years from
the date of grant.
8
Employment Agreements, Severance and Change in Control Arrangements
Agreements with Ms. Washer and Dr. Chulay
On September 26, 2014, we entered into an employment agreement with Ms. Washer. Under the terms of
her employment agreement, if we terminate Ms. Washer’s employment without “cause” or if she terminates her
employment with us for “good reason” prior to a change of control or during the 12-month period following a
“change of control,” in each case as those terms are defined in her employment agreement, she will be entitled to
receive severance benefits, payable in a single lump sum, as follows:
• An amount equal to the sum of (a) her then current annual base salary and (b) the product of her target
bonus in effect immediately prior to the date of termination multiplied by a fraction equal to the
quotient of (i) the number of days elapsed as of the termination date during the year in which the
termination occurs divided by (ii) 365.
•
•
•
She will also be entitled to continue to participate in our benefits plans for a period of up to 12 months
following the effective date of the termination of her employment on substantially the same terms as
were in effect immediately prior to her termination.
In addition, if Ms. Washer’s employment is terminated by us without cause or by Ms. Washer during
the 12 months following a change of control for good reason, all unvested equity awards previously
granted to her will become fully vested as of the date of the termination of her employment.
In the event Ms. Washer terminates her employment for good reason other than during the 12-month
period following a change of control, each unvested equity award previously granted to her will
immediately vest with respect to 50% of the shares that are unvested as of the effective date of the
termination of her employment.
• To the extent that the vesting of any unvested awards held by Ms. Washer at the time of the termination
of her employment is contingent upon the attainment of any corporate or market performance condition
that has not been satisfied as of that date, the condition will be deemed to have been satisfied as of the
date of termination
•
•
at the 100% level, in the case of a termination by us without cause or by Ms. Washer during the 12
months following a change of control for good reason, or
the 50% level, in the case of a termination by Ms. Washer for good reason other than during the
12 months following a change of control for good reason.
On September 26, 2014, we also entered into an employment agreement with Dr. Chulay. Under the terms
of his employment agreement, if we terminate Dr. Chulay’s employment without “cause” or if Dr. Chulay
terminates his employment for with us for “good reason” during the 12-month period following a “change of
control,” in each case as those terms are defined in his employment agreement, he will be entitled to receive
severance benefits, payable in a single lump sum, as follows:
• An amount equal to the sum of (a) 75% of his then current annual base salary and (b) the product of his
target bonus in effect immediately prior to the date of termination multiplied by a fraction equal to the
quotient of (i) the number of days elapsed as of the termination date during the year in which the
termination occurs divided by (ii) 365.
• He will also be entitled to continue to participate in our benefits plans for a period of up to nine months
following the effective date of such a termination of his employment on substantially the same terms as
were in effect immediately prior to his termination.
•
In addition, if Dr. Chulay’s employment is terminated during the 12 months following a change of
control by us without cause or by Dr. Chulay for good reason, all unvested equity awards
previously granted to Dr. Chulay will become fully vested as of the date of such a termination of
his employment.
9
•
In the event we terminate Dr. Chulay’s employment without cause at any time other than during
the 12-month period following a change of control, each unvested equity award previously
granted to Dr. Chulay will immediately vest with respect to 50% of the shares that are unvested as
of the effective date of the termination of his employment.
• To the extent that the vesting of any unvested awards held by Dr. Chulay at the time of the
termination of his employment is contingent upon the attainment of any corporate or market
performance condition that has not been satisfied as of that date, the condition will be deemed to
have been satisfied as of the date of termination
•
•
at the 100% level, in the case of a termination during the 12 months following a change of
control by us without cause or by Dr. Chulay for good reason, or
the 50% level, in the case of a termination by us without cause other than during the 12
months following a change of control.
Agreements with Dr. Shearman and Mr. Potter
We entered into offer letters with each of Dr. Shearman and Mr. Potter in connection with their employment
by us. Pursuant to the terms of the offer letters, under certain circumstances, we may be required to make
severance payments to Dr. Shearman and Mr. Potter following a termination of their respective employment by
us. If, at any time following the date that is six months following the start of his respective employment with us,
either of Dr. Shearman’s or Mr. Potter’s employment by us is terminated (i) by us without cause or (ii) by either
of Dr. Shearman or Mr. Potter, as applicable, following a sale of all or substantially all of our stock or assets,
whether by merger, acquisition or otherwise, in which he is not offered a position with the successor entity with
substantially equivalent responsibilities and with total compensation, benefits and severance rights at least
equivalent to those he received from us immediately prior to such event, which we refer to as a change of control
termination, the affected individual will receive:
•
in the case of Dr. Shearman,
•
•
•
an amount equal to six-months of his then-current base salary and earned bonus, if the termination
occurs prior to the first anniversary of the commencement of his employment, or
an amount equal to nine-months of his then-current base salary and earned bonus, if the
termination occurs on or after the first anniversary of the commencement of his employment; and
if the termination is a change of control termination, all of Dr. Shearman’s outstanding unvested
options will immediately vest and become exercisable; or
•
in the case of Mr. Potter, an amount equal to six-months of his then-current base salary.
Agreement with Mr. Bullock
We also entered into an offer letter with Mr. Bullock in connection with his employment as our Chief
Financial Officer. However, Mr. Bullock retired as our Chief Financial Officer effective as of August 7, 2017
pursuant to the terms of a separation agreement. The terms of the separation agreement are described in more
detail in a current report on Form 8-K filed with the Commission on August 2, 2017. Though no longer
applicable, prior to his retirement, pursuant to the terms of his offer letter, under certain circumstances we would
have been required to make severance payments to Mr. Bullock following a termination of his employment by
us. If, at any time following the date that was six months following the start of his employment with us,
Mr. Bullock was terminated (i) by us without cause or (ii) by him following a sale of all or substantially all of our
stock or assets, whether by merger, acquisition or otherwise, in which he was not offered a position with the
successor entity with substantially equivalent responsibilities and with total compensation, benefits and
severance rights at least equivalent to those he received from us immediately prior to such event, which we refer
to as a change of control termination, he would have been entitled to receive:
•
an amount equal to six-months of his then-current base salary and earned bonus, if the termination
occurred prior to the first anniversary of the commencement of his employment, or
10
•
•
an amount equal to nine-months of his then-current base salary and earned bonus, if the
termination occurred on or after the first anniversary of the commencement of his employment, or
an amount equal to twelve months of base salary and bonus, if the termination was a result of a
change in control of AGTC and Mr. Bullock was not offered the position of chief financial officer
of the acquiring company.
Additionally, in the event there was a change of control of AGTC and Mr. Bullock was not offered the
position of chief financial officer of the acquiring company, then all of his then-outstanding options would have
vested in full upon the change of control and remained exercisable until the earlier of the second anniversary of
the change of control and their scheduled expiration.
The following table provides information regarding the estimated amounts payable to our named executive
officers upon the occurrence of the triggering events described, in each case assuming that the trigger event
occurred on June 30, 2017 (including for Mr. Bullock, notwithstanding his retirement on August 7, 2017 as
described above), the last day of our most recently completed fiscal year, assuming that their employment
agreements had been in effect as of that date. The amounts shown as payable upon the triggering events
described do not include amounts earned by the individual and accrued before the occurrence of the triggering
event but payable after the triggering event, such as accrued and unpaid salary or the value of accrued but unused
paid-time-off.
11
Payments Due Upon Termination of Employment
Name and Trigger Event
Susan B. Washer
Cash ($)
Equity
($)(1)
Perquisites/
Benefits ($)
Total ($)
Termination of employment by us without cause . . . . . . . . . . . . . . .
Termination of employment by Ms. Washer for good reason during
the 12 months following a change of control . . . . . . . . . . . . . . . .
Termination of employment by Ms. Washer for good reason other
754,850
1,750
— (2) 756,600
754,850
1,750
— (2) 756,600
than during the 12 months following a change of control . . . . . .
754,850
875
— (2) 755,725
Lawrence E. Bullock (3)
Termination of employment by us without cause . . . . . . . . . . . . . . .
Change of control termination . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
Change of change of control of AGTC in which Mr. Bullock is not
offered the position of chief financial officer of the acquiring
company . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
Termination of employment by Mr. Bullock following a change of
control of AGTC in which he is not offered the position of chief
financial officer of the acquiring company . . . . . . . . . . . . . . . . .
Jeffrey D. Chulay, M.D.
Termination of employment by us without cause other than during
the 12 months following a change of control . . . . . . . . . . . . . . . .
Termination of employment by us without cause or by Dr. Chulay
for good reason during the 12 months following a change of
control . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
Stephen A. Potter
375,100 —
375,100 —
—
—
—
—
—
375,100
375,100
—
460,350 —
—
460,350
440,000
254
4,587 (4) 444,841
440,000
508
4,587 (4) 445,095
Termination of employment by us without cause . . . . . . . . . . . . . . .
Change of control termination . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
165,500 —
165,500 —
Mark Shearman, Ph.D.
Termination of employment by us without cause . . . . . . . . . . . . . . .
Change of control termination . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
407,000 —
407,000 —
—
—
—
—
165,500
165,500
407,000
407,000
(1) Value represents the number of shares underlying unvested in-the-money stock options that would have
been accelerated multiplied by the difference between the exercise prices of such options minus the closing
price of our common stock on the last trading day of the year.
(2) As of June 30, 2017, Ms. Washer did not participate in our medical, dental or vision insurance benefit
programs.
(3) Mr. Bullock retired in August 2017.
(4) Represents the value of medical, dental and vision insurance benefit continuation for nine months after
termination.
Director Compensation
Our non-employee directors other than Dr. Guyer receive equity-based compensation and cash fees as
follows:
•
•
•
each non-employee director receives an annual cash fee in the amount of $35,000;
our chairman receives an additional cash fee in the amount of $35,000;
the chairperson of each of our board committees receives an additional annual cash fee as follows:
audit committee chair, $15,000; compensation committee chair, $10,000; and nominating and corporate
governance committee chair, $7,500; and
12
•
each other member of a board committee receives an additional annual cash fee as follows: audit
committee, $7,500; compensation committee, $5,000; and nominating and corporate governance
committee, $3,750.
The cash fees described above are paid quarterly in arrears. Non-employee directors are also reimbursed
upon request for travel and other out-of-pocket expenses incurred in connection with their attendance at meetings
of the board and of committees on which they serve.
Upon initial election to our board of directors, our non-employee directors other than Dr. Guyer are entitled
to receive a non-qualified stock option, vesting in equal installments on each of the first three anniversaries of the
date of grant, to purchase 16,000 shares of our common stock. In addition, each non-employee director other than
Dr. Guyer remaining in office receives annually a non-qualified stock option, vesting on the first anniversary of
the date of grant, to purchase 8,000 shares of our common stock. Each such initial or annual stock option is
granted with an exercise price equal to the fair value of our common stock on the date of grant.
We have agreed to pay to Dr. Guyer an annual cash fee in the amount of $75,000 (reduced from $95,000 for fiscal
year 2017) for his service on our board of directors in lieu of the cash payments and option grants described above.
The following table sets forth information regarding compensation awarded to, earned by or paid to our
non-employee directors who served during fiscal year 2017. We do not pay any compensation to our president
and chief executive officer in connection with her service on our board of directors. See “Executive
Compensation” for a discussion of the compensation of Ms. Washer.
Name
. . . . . . . . . . . . . . .
Scott Koenig, M.D., Ph.D.
David R. Guyer, M.D. . . . . . . . . . . . . . . . . . . .
Ed Hurwitz . . . . . . . . . . . . . . . . . . . . . . . . . . .
Ivana Magovcevic-Liebisch, Ph.D. . . . . . . . . .
Arnold L. Oronsky, Ph.D. (3) . . . . . . . . . . . . .
James Rosen . . . . . . . . . . . . . . . . . . . . . . . . . .
Anne VanLent (4) . . . . . . . . . . . . . . . . . . . . . .
Fees earned or paid in
cash ($)(1)
Option awards ($)(2)
Total ($)
$75,000
$98,333
$48,750
$44,167
$ 8,750
$48,333
$44,649
$ 43,327
—
$ 43,327
$ 43,327
$ 43,327
$ 43,327
$207,125
$118,327
$ 98,333
$ 92,077
$ 87,494
$ 52,077
$ 91,660
$251,774
(1) Represents amount earned or paid for service as a director during fiscal year 2017.
(2) Represents the grant date fair value of option awards granted in fiscal year 2017 in accordance with ASC
718. See Note 8 of the notes to our financial statements included in the Original Filing for a discussion of
the relevant assumptions used in calculating these amounts.
(3) Mr. Oronsky resigned from our board of directors effective as of August 14, 2017.
(4) Ms. VanLent joined the board of directors on August 17, 2016.
The table below shows the aggregate number of option awards held as of June 30, 2017 by each of our
current non-employee directors who was serving as of that date.
Name
Scott Koenig, M.D., Ph.D. . . . . . . . . . . .
David R. Guyer, M.D. . . . . . . . . . . . . . .
Ed Hurwitz . . . . . . . . . . . . . . . . . . . . . . .
Ivana Magovcevic-Liebisch, Ph.D. . . . .
Arnold L. Oronsky, Ph.D. . . . . . . . . . . .
James Rosen . . . . . . . . . . . . . . . . . . . . .
Anne VanLent (1) . . . . . . . . . . . . . . . . .
Number of Options Outstanding at
June 30, 2017
85,993
—
28,263
28,263
28,263
28,263
24,000
(1) Ms. VanLent joined the board of directors on August 17, 2016.
13
Compensation Committee Interlocks and Insider Participation
The members of our compensation committee for fiscal year 2017 were Dr. Koenig, Dr. Magovcevic-
Liebisch and Mr. Rosen. None of our executive officers serves, or in the past has served, as a member of the
board of directors or compensation committee, or other committee serving an equivalent function, of any entity
that has one or more executive officers who serve as members of our board of directors or our compensation
committee. None of the members of our compensation committee is an officer or employee of our company, nor
has any of them ever been an officer or employee of our company.
14
ITEM 12. SECURITY OWNERSHIP OF CERTAIN BENEFICIAL OWNERS AND MANAGEMENT
AND RELATED STOCKHOLDER MATTERS
The following table sets forth certain information with respect to beneficial ownership of our common
stock, as of October 15, 2017, by:
•
•
•
•
each person or entity, or group of affiliated persons or entities, known by us to beneficially own more
than 5% of our common stock;
each of our directors;
each of our named executive officers; and
all of our executive officers and directors as a group.
Beneficial ownership is determined in accordance with the rules of the SEC. In computing the number of
shares beneficially owned by a person and the percentage ownership of that person, shares of common stock
subject to options or warrants held by that person that are currently exercisable or exercisable within 60 days of
October 15, 2017 are deemed outstanding, but are not deemed outstanding for computing the percentage
ownership of any other person. To our knowledge, except as set forth in the footnotes to this table and subject to
applicable community property laws, each person named in the table has sole voting and investment power with
respect to the shares set forth opposite such person’s name. Except as otherwise indicated, the address of each of
the persons in this table is c/o Applied Genetic Technologies Corporation, 14193 NW 119th Terrace, Suite 10,
Alachua, Florida 32615.
Each stockholder’s percentage ownership is determined in accordance with Rule 13d-3 under the Exchange
Act and is based on 18,093,235 shares of our common stock outstanding as of October 15, 2017. The number of
outstanding shares beneficially owned by each stockholder below was obtained from the most recent publicly
filed information, as applicable. Amounts under the heading “Right to Acquire” represent shares that may be
acquired upon exercise of outstanding stock options or warrants exercisable within 60 days of October 15, 2017.
Name of Beneficial Owner
Shares
Outstanding Right to Acquire
Total
Percentage
of Shares
Outstanding
Entities affiliated with InterWest Partners (1) . . . . . . . . . .
Biogen Inc. (2) . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
S.R. One, Limited (3) . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
Intersouth Partners VI, L.P. (4) . . . . . . . . . . . . . . . . . . . . .
Alta Partners VIII, L.P. (5) . . . . . . . . . . . . . . . . . . . . . . . . .
FMR LLC (6) . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
MedImmune Ventures, Inc. (7) . . . . . . . . . . . . . . . . . . . . .
Susan Washer (8) . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
Lawrence E. Bullock (9)
. . . . . . . . . . . . . . . . . . . . . . . . . .
Jeffrey D. Chulay, M.D. (10) . . . . . . . . . . . . . . . . . . . . . . .
Stephen Potter (11) . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
Mark Shearman, Ph.D. (12) . . . . . . . . . . . . . . . . . . . . . . . .
David Guyer, M.D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
Edward Hurwitz (13)(14) . . . . . . . . . . . . . . . . . . . . . . . . . .
Scott Koenig, M.D., Ph.D. (15) . . . . . . . . . . . . . . . . . . . . .
Ivana Magovcevic-Liebisch, Ph.D. (16)
. . . . . . . . . . . . . .
James Rosen (17) . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
Anne VanLent (18) . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
All executive officers and directors (11 persons) (19) . . . .
1,452,216
1,453,957
1,436,448
1,214,457
1,129,551
1,114,092
1,011,300
20,328
10,000
24,400
1,400
3,000
—
1,800
2,228
3,000
—
—
66,156
11,895
—
—
—
—
—
—
494,413
190,971
143,676
140,284
116,208
—
20,263
77,993
20,263
20,263
5,333
1,229,667
1,464,111
1,453,957
1,436,448
1,214,457
1,129,551
1,114,092
1,011,300
514,741
200,971
168,076
141,684
119,208
—
22,063
80,221
23,263
20,263
5,333
1,295,823
8.1%
8.0%
7.9%
6.7%
6.2%
6.2%
5.6%
2.8%
*
*
*
*
—
*
*
*
*
*
7.2%
(1) This information is based on a Schedule 13G filed with the SEC on February 14, 2017 by InterWest Partners
VIII, L.P., InterWest Investors VIII, L.P., InterWest Investors Q VIII, L.P., InterWest Management Partners
VIII, LLC, Harvey B. Cash, Philip T. Gianos, W. Stephen Holmes III, Gilbert H. Kliman and Arnold L.
15
Oronsky. InterWest Management Partners VIII, LLC is the general partner of InterWest Partners VIII, L.P.,
InterWest Investors VIII, L.P., InterWest Investors Q VIII, L.P., and has sole voting and investment control
over the shares held by each of them. Harvey B. Cash, Philip T. Gianos, W. Stephen Holmes, Gilbert H.
Kliman and Arnold L. Oronsky, a member of our board of directors, are the managing directors of InterWest
Management Partners VIII, LLC. Each of the managing directors share voting and dispositive power over
the shares held by the entities affiliated with InterWest Partners. The address for these entities is c/o
InterWest Partners, 2710 Sand Hill Road, Suite 200, Menlo Park, California 94025. Includes 11,895 shares
of common stock issuable upon exercise of stock purchase warrants exercisable within 60 days of the date
of this table.
(2) This information is based on a Schedule 13G filed with the SEC on August 20, 2015 by Biogen Inc. and
Biogen MA Inc. The address of Biogen Inc. is 225 Binney Street, Cambridge MA 02142 and the address of
Biogen MA Inc. is 250 Binney Street, Cambridge, MA 02142. Biogen Inc. and Biogen MA Inc. share voting
and dispositive power with respect to all of the shares of our common stock reported as beneficially owned
by them.
(3) This information is based on a Schedule 13D/A filed with the SEC on February 24, 2015 by
GlaxoSmithKline plc, the indirect parent of S.R. One, Limited. The address of S.R. One, Limited is 161
Washington Street, Suite 500, Conshohocken, Pennsylvania 19428.
(4) The address of Intersouth Partners VI, L.P. is 102 City Hall Plaza, Suite 200, Durham, North Carolina
27701. Mitchell Mumma and Dennis Dougherty are the managing members of Intersouth Associates VI,
LLC, the sole general partner of Intersouth Partners VI, L.P., and share the power to vote or direct the
voting of and to dispose or direct the disposition of the shares of our common stock held by Intersouth
Partners VI, L.P.
(5) This information is based on a Schedule 13D/A filed with the SEC on April 20, 2015 by Alta Partners VIII,
L.P., Alta Partners Management, LLC, Farah Champsi, Daniel Janney and Guy Nohra. The address of Alta
Partners VIII, L.P. is One Embarcadero Center, 37th Floor, San Francisco, California 94111. Alta Partners
Management VIII, LLC is the general partner of Alta Partners VIII, L.P. and shares voting and dispositive
power over 1,129,551 shares of our common stock held by Alta Partners VIII, L.P. Farah Champsi, Daniel
Janney, and Guy Nohra are the managing directors of Alta Partners Management VIII, LLC and share
dispositive and voting control over the shares of our common stock held by Alta Partner VIII, L.P. The
amount included in the table above excludes 12,436 shares of our common stock reported as owned directly
by Ms. Champsi, over which she exercises sole dispositive and voting control.
(6) This information is based on a Schedule 13G/A filed with the SEC on February 14, 2017 by FMR LLC and
Abigail P. Johnson. FMR LLC reported that it has sole dispositive power over 1,115,494 shares of our
common stock and sole voting power over 367 shares of our common stock. The address provided for FMR
LLC and Ms. Johnson is 245 Summer Street, Boston, MA 02210.
(7) This information is based on a Schedule 13G filed with the SEC on February 17, 2015 by Astrazeneca plc
and Medimmune Ventures, Inc., which reported that they shared voting and dispositive power with respect
to 1,114,092 shares of our common stock. The address of Astrazeneca plc is 2 Kingdom Street, London W2
6BD and the address of MedImmune Ventures, Inc. is One MedImmune Way, Gaithersburg, Maryland
20878.
(8) Excludes 104,627 shares subject to outstanding stock options that are not exercisable within 60 days of the
date of the table.
(9) Excludes 44,850 shares subject to outstanding stock options that are not exercisable within 60 days of the
date of the table.
(10) Excludes 58,068 shares subject to outstanding stock options that are not exercisable within 60 days of the
date of the table.
(11) Excludes 91,765 shares subject to outstanding stock options that are not exercisable within 60 days of the
date of the table.
16
(12) Excludes 94,792 shares subject to outstanding stock options that are not exercisable within 60 days of the
date of the table.
(13) Excludes 8,000 shares subject to outstanding stock options that are not exercisable within 60 days of the
date of the table.
(14) Includes 1,800 shares held by the Hurwitz/Lichtenfeld Revocable Trust over which Mr. Hurwitz, as a trustee
and a beneficiary, may be deemed to exercise voting and investment control.
(15) Excludes 8,000 shares subject to outstanding stock options that are not exercisable within 60 days of the
date of the table.
(16) Excludes 8,000 shares subject to outstanding stock options that are not exercisable within 60 days of the
date of the table.
(17) Excludes 8,000 shares subject to outstanding stock options that are not exercisable within 60 days of the
date of the table.
(18) Excludes 18,667 shares subject to outstanding stock options that are not exercisable within 60 days of the
date of the table.
(19) Excludes 444,769 shares subject to outstanding stock options that are not exercisable within 60 days of the
date of the table.
17
ITEM 13. CERTAIN RELATIONSHIPS AND RELATED TRANSACTIONS, AND DIRECTORS
INDEPENDENCE
Policies and Procedures for Related-Person Transactions
We have adopted a written related-person transactions policy that sets forth our policies and procedures
regarding the identification, review, consideration, approval and oversight of “related-person transactions.” For
purposes of our policy only, a “related-person transaction” is a past, present or future transaction, arrangement or
relationship (or any series of similar transactions, arrangements or relationships) in which we and any “related
person” are participants involving an amount that exceeds $120,000.
Transactions involving compensation for services provided to us by an employee, consultant or director are
not considered related-person transactions under this policy. A “related person,” as determined since the
beginning of our last fiscal year, is any executive officer, director or a holder of more than five percent of our
common stock, including any of their immediate family members and any entity owned or controlled by such
persons.
The policy imposes an affirmative duty upon each director and executive officer to identify any transaction
involving them, their affiliates or immediate family members that may be considered a related party transaction
before such person engages in the transaction. Under the policy, where a transaction has been identified as a
related-person transaction, management must present information regarding the proposed related-person
transaction to our audit committee (or, where review by our audit committee would be inappropriate, to another
independent body of our board of directors) for review. The presentation must include a description of, among
other things, the material facts, the direct and indirect interests of the related persons, the benefits of the
transaction to us and whether any alternative transactions are available.
Our audit committee is responsible for reviewing and approving in advance any related-person transactions.
In determining whether to approve a related-person transaction, the audit committee will take into account,
among other factors it deems appropriate, whether the related-person transaction is on terms no less favorable
than terms generally available to an unaffiliated third-person under the same or similar circumstances and the
extent of the related person’s interest in the transaction.
Agreements with Our Stockholders
Biogen Collaboration and License Agreement
On July 1, 2015, we entered into a Collaboration and License Agreement, which we refer to as the
collaboration agreement, with Biogen MA Inc., a wholly owned subsidiary of Biogen Inc. (“Biogen”), pursuant
to which we and Biogen agreed to collaborate to develop, seek regulatory approval for and commercialize gene
therapy products to treat X-linked juvenile retinoschisis (“XLRS”), X-linked retinitis pigmentosa (“XLRP”) and
discovery programs targeting three indications based on our adeno-associated virus vector technologies. The
collaboration agreement became effective in August 2015.
Under the collaboration agreement, we will be eligible to receive upfront payments, option exercise fees and
milestone payments aggregating over $1 billion, including an upfront license fee of $94.0 million which we
received on August 19, 2015 and a portion of which was allocated to fund our costs incurred in connection with
budgeted research and development activities for the XLRS and XLRP programs as well as the discovery
programs. During fiscal year 2017, we also earned and received a $5.0 million milestone payment from Biogen
for the XLRS program and we recorded other revenue of $1,052,000, primarily comprised of reimbursable costs
for post-funding development activities that we conducted pursuant to the terms of this collaboration agreement
with Biogen. In the event that Biogen exercises its option to obtain an exclusive commercial license for one or
more discovery products that are designated as clinical candidates, we are eligible to receive an option exercise
fee for each drug candidate. In addition, we are eligible to receive development milestone payments upon the
18
achievement of specified regulatory, clinical development and commercialization milestones of up to
$472.5 million collectively for the two lead programs and, together with option exercise fees, up to
$592.5 million across the discovery programs. Biogen also has the right to substitute up to two discovery
programs, with a limited ability to reinstate such substituted programs within six months. In the event Biogen
elects to reinstate such substituted programs, we are eligible to receive additional option exercise fees and
potential development, regulatory and sales milestone payments.
Budgeted development expenses for the programs are funded by Biogen through an allocation of the upfront
license fee described below, subject to cost sharing for budget overruns and additional clinical trials that may be
required prior to a pivotal trial for each of the XLRS and XLRP programs. During our development of the XLRS
and XLRP products, Biogen retains the right to step in and take over the remaining development activities under
specified circumstances. When Biogen assumes development responsibility, budgeted development expenses are
paid by Biogen, subject to cost sharing for additional development activities. For each of the XLRS and XLRP
programs, we have an option to share in development costs and resulting profits as well as an option to
co-promote the second product approved in the United States. The collaboration agreement also provides for
discovery programs targeting three indications whereby we will conduct discovery, research and development
activities for those additional drug candidates through the stage of clinical candidate designation, after which,
Biogen may exercise an option to continue to develop, seek regulatory approval for and commercialize the
designated clinical candidate.
Under the collaboration agreement, we granted Biogen an exclusive, royalty-bearing license, with the right
to grant sublicenses, to use adeno-associated virus vector technology and other technology controlled by us for
the purpose of researching, developing, manufacturing and commercializing licensed products developed under
the agreement. We also granted Biogen a non-exclusive, worldwide, royalty-free, fully paid license, with the
right to grant sublicenses, of our interest in other intellectual property developed pursuant to the collaboration
agreement. Biogen agreed to pay royalties for each licensed product at tiered rates ranging from high single digit
to mid-teen percentages of annual net sales of the XLRS or XLRP products and rates ranging from mid-single
digit to low-teen percentages of annual net sales for the discovery products.
Director Independence
Our Board of Directors has determined that, with the exception of Ms. Washer who is our employee, all of
the members of our Board of Directors are “independent directors” under the applicable rules of the Nasdaq
Stock Market. Our Board of Directors has also determined that each member of our Audit Committee,
Compensation Committee and Nominating and Corporate Governance Committee is an “independent director”
under the rules of the Nasdaq Stock Market applicable to such committees.
19
ITEM 14. PRINCIPAL ACCOUNTANT FEES AND SERVICES
Our Audit Committee engaged RSM US LLP to serve as our independent registered public accounting firm
for the fiscal year ended June 30, 2017. The selection of RSM US LLP was approved by our stockholders at the
2016 annual meeting of stockholders. Our Audit Committee has also engaged RSM US LLP to serve as our
independent registered public accounting firm for the fiscal year ending June 30, 2018.
Audit and Other Fees
The following table shows fees for professional audit services, audit-related fees, tax fees and other services
rendered by RSM US LLP, including its affiliates, for the audit of our annual financial statements for the fiscal
years ended June 30, 2016 and 2017:
Fee Category
Fiscal 2017
Fiscal 2016
Audit fees . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
Audit-related fees . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
Tax fees . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
All other fees . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
$218,486
60,000
—
9,036
$187,683
31,000
40,250
—
Total fees . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
$287,522
$258,933
Audit fees. Audit fees consist of fees and related expenses billed for professional services rendered for the
audit of the financial statements and services that are normally provided by RSM US LLP in connection with
statutory and regulatory filings or engagements and include fees for professional services rendered in connection
with quarterly and annual reports. The audit fees for fiscal years 2017 and 2016 also include fees and related
expenses associated with the issuance of consents by RSM US LLP to be named in our registration statements
and to the use of their audit report in the registration statements.
Audit-related fees. Audit-related fees represent fees for assurance and related services performed by RSM
US LLP that are reasonably related to the performance of the audit or review of our financial statements,
including consultation on accounting standards or accounting for specific transactions.
Tax fees. Tax fees represent fees for professional services performed by RSM US LLP with respect to tax
compliance, tax advice and tax planning and related expenses. The company engages a separate professional
services firm for these services, including assistance with the preparation of federal, state, and foreign income tax
returns.
All other fees. All other fees represent fees for products and services provided by RSM US LLP, other than
those disclosed above.
Pre-Approval Policies and Procedures
Our audit committee’s pre-approval policies or procedures do not allow our management to engage RSM
US LLP to provide any specified services without specific audit committee pre-approval of the engagement for
those services. All of the services provided by RSM US LLP during fiscal year 2017 were pre-approved.
20
ITEM 15. EXHIBITS AND FINANCIAL STATEMENT SCHEDULES
(a) Documents filed as a part of this Report:
PART IV
(1) Financial Statements —See Index to Financial Statements and Financial Statement Schedule at Item 8
on page 80 of the Original Filing.
(2) Financial Statement Schedules —See Index to Financial Statements and Financial Statement Schedule
at Item 8 on page 80 of the Original Filing. All other schedules are omitted because they are not applicable
or not required.
21
(3) Index to Exhibits.
Exhibit
number
3.1
3.2
4.1
10.1
10.2*
10.3*
10.4*
10.5*
10.6*
10.7†
10.8
10.9†
Description
Fifth Amended and Restated Certificate of Incorporation of the Company (incorporated by
reference to Exhibit 3.1 to the Company’s Current Report on Form 8-K filed with the SEC on
April 1, 2014)
Amended and Restated Bylaws of the Company (incorporated by reference to Exhibit 3.2 to the
Company’s Current Report on Form 8-K filed with the SEC on April 1, 2014)
Specimen certificate evidencing shares of common stock (incorporated by reference to Exhibit 4.1
to the Company’s Registration Statement on Form S-1 (File No. 333-193309))
Lease Agreement made as of April 10, 2015, by and between Alachua Foundation Park Holding
Company, LLC and Applied Genetic Technologies Corporation (incorporated by reference to
Exhibit 10.1 to the Company’s Annual Report on Form 10-K for the year ending June 30, 2015
(File No. 001-36370))
Employment Agreement dated as of May 27, 2015 between Applied Genetic Technologies
Corporation and Mark S. Shearman (incorporated by reference to Exhibit 10.2 to the Company’s
Annual Report on Form 10-K for the year ending June 30, 2015 (File No. 001-36370))
Employment Agreement dated as of January 29, 2015 between Applied Genetic Technologies
Corporation and Stephen W. Potter (incorporated by reference to Exhibit 10.1 to the Company’s
Quarterly Report on Form 10-Q for the quarter ended March 31, 2015 (File No. 001-36370))
Separation Agreement dated as of March 3, 2015 between Applied Genetic Technologies
Corporation and Daniel L. Menichella (incorporated by reference to Exhibit 10.1 to the Company’s
Quarterly Report on Form 10-Q/A (Amendment No. 1) for the quarter ended March 31, 2015 (File
No. 001-36370))
Employment Agreement dated as of September 26, 2014 between Applied Genetic Technologies
Corporation and Susan B. Washer (incorporated by reference to Exhibit 10.1 to the Company’s
Current Report on Form 8-K, event date September 26, 2014, filed on October 2, 2014 (File
No. 001-36370))
Employment Agreement dated as of September 26, 2014 between Applied Genetic Technologies
Corporation and Jeffrey D. Chulay (incorporated by reference to Exhibit 10.2 to the Company’s
Current Report on Form 8-K, event date September 26, 2014, filed on October 2, 2014 (File
No. 001-36370))
Collaboration and License Agreement dated as of July 1, 2015 by and between Biogen MA Inc.,
and Applied Genetic Technologies Corporation (incorporated by reference to Exhibit 10.7 to the
Company’s Annual Report on Form 10-K for the year ending June 30, 2015 (File No. 001-36370))
Common Stock Purchase Agreement dated as of July 1, 2015 by and between Biogen MA Inc., and
Applied Genetic Technologies Corporation (incorporated by reference to Exhibit 10.8 to the
Company’s Annual Report on Form 10-K for the year ending June 30, 2015 (File No. 001-36370))
Manufacturing License and Technology Transfer Agreement dated as of July 1, 2015 by and
between Biogen MA Inc., and Applied Genetic Technologies Corporation (incorporated by
reference to Exhibit 10.9 to the Company’s Annual Report on Form 10-K for the year ending
June 30, 2015 (File No. 001-36370))
10.10†
Second Amendment to Non-exclusive License Agreement, made and effective as of June 29, 2015,
by and between The UAB Research Foundation, Inc. and Applied Genetic Technologies
Corporation (incorporated by reference to Exhibit 10.10 to the Company’s Annual Report on Form
10-K for the year ending June 30, 2015 (File No. 001-36370))
22
(3) Index to Exhibits.
Exhibit
number
10.11†
10.12†
10.13
10.14†
10.15†
10.16†
10.17†
10.18†
10.19†
10.20†
10.21†
Description
Omnibus Amendment to Standard Exclusive License Agreement with Sublicensing Terms, made
and effective as of July 1, 2015, by and between the University of Florida Research Foundation,
Inc., the University of Florida Board of Trustees, John Hopkins University and Applied Genetic
Technologies Corporation (incorporated by reference to Exhibit 10.11 to the Company’s Annual
Report on Form 10-K for the year ending June 30, 2015 (File No. 001-36370))
Omnibus Amendment to Standard Exclusive License Agreement with Know How and Standard
Non-Exclusive License Agreement, made and effective as of June 30, 2015, by and between the
University of Florida Research Foundation, Inc. and Applied Genetic Technologies Corporation
(incorporated by reference to Exhibit 10.12 to the Company’s Annual Report on Form 10-K for the
year ending June 30, 2015 (File No. 001-36370))
Lease Agreement made as of September 19, 2011, by and between Thomson-Davis Enterprises,
LLC and Applied Genetic Technologies Corporation (incorporated by reference to Exhibit 10.1 to
the Company’s Registration Statement on Form S-1 (File No. 333-193309))
Exclusive License Agreement with Sublicensing Terms, effective as of September 25, 2001, by and
between the University of Florida Research Foundation, Inc. and Applied Genetic Technologies
Corporation (incorporated by reference to Exhibit 10.2 to the Company’s Registration Statement on
Form S-1 (File No. 333-193309))
Restated Amendment to License Agreement made and, effective as of January 31, 2005, by and
between the University of Florida Research Foundation, Inc. and Applied Genetic Technologies
Corporation (incorporated by reference to Exhibit 10.3 to the Company’s Registration Statement on
Form S-1 (File No. 333-193309))
First Amendment After Restated Amendment to License Agreement, made and effective as of
November 28, 2007, by and between the University of Florida Research Foundation, Inc. and
Applied Genetic Technologies Corporation (incorporated by reference to Exhibit 10.4 to the
Company’s Registration Statement on Form S-1 (File No. 333-193309))
Standard Exclusive License Agreement with Sublicensing Terms, effective as of October 7, 2003,
by and between the University of Florida Research Foundation, Inc., Johns Hopkins University and
Applied Genetic Technologies Corporation (incorporated by reference to Exhibit 10.5 to the
Company’s Registration Statement on Form S-1 (File No. 333-193309))
First Amendment to Standard Exclusive License Agreement with Sublicensing Terms, made as of
November 2004, by and between the University of Florida Research Foundation, Inc., Johns
Hopkins University and Applied Genetic Technologies Corporation (incorporated by reference to
Exhibit 10.6 to the Company’s Registration Statement on Form S-1 (File No. 333-193309))
Second Amendment to Standard Exclusive License Agreement with Sublicensing Terms, made as
of February 25, 2009, by and among Applied Genetic Technologies Corporation, the University of
Florida Research Foundation, Inc. and Johns Hopkins University (incorporated by reference to
Exhibit 10.7 to the Company’s Registration Statement on Form S-1 (File No. 333-193309))
Non-Exclusive License Agreement with Sublicensing Terms, made as of January 19, 2006, by and
between The UAB Research Foundation, Inc. and Applied Genetic Technologies Corporation
(incorporated by reference to Exhibit 10.8 to the Company’s Registration Statement on Form S-1
(File No. 333-193309))
Standard Non-Exclusive License Agreement, effective as of September 18, 2012, by and between
the University of Florida Research Foundation, Inc. and Applied Genetic Technologies Corporation
(incorporated by reference to Exhibit 10.10 to the Registrant’s Registration Statement on Form S-1
(File No. 333-193309))
23
(3) Index to Exhibits.
Exhibit
number
10.22†
10.23
10.24*
10.25*
10.26*
10.27*
10.28
10.29
10.30
10.31
10.32
10.33
10.34
10.35
10.36†
Description
Standard Exclusive License Agreement with Know How, effective as of November 5, 2012, by and
between the University of Florida Research Foundation, Inc. and Applied Genetic Technologies
Corporation (incorporated by reference to Exhibit 10.11 to the Company’s Registration Statement
on Form S-1 (File No. 333-193309))
Amended and Restated Investor Rights Agreement, dated as of November 15, 2012 (incorporated
by reference to Exhibit 10.12 to the Company’s Registration Statement on Form S-1
(File No. 333-193309))
Applied Genetic Technologies Corporation 2001 Stock Option Plan, as amended (incorporated by
reference to Exhibit 10.13 to the Company’s Registration Statement on Form S-1
(File No. 333-193309))
Applied Genetic Technologies Corporation 2011 Stock Incentive Plan, as amended, and forms of
Incentive Stock Option Agreement and Nonstatutory Stock Option Agreement thereunder
(incorporated by reference to Exhibit 10.14 to the Company’s Registration Statement on Form S-1
(File No. 333-193309))
Applied Genetic Technologies Corporation 2013 Equity And Incentive Plan (incorporated by
reference to Exhibit 10.15 to the Company’s Registration Statement on Form S-1
(File No. 333-193309))
Applied Genetic Technologies Corporation 2013 Employee Stock Purchase Plan (incorporated by
reference to Exhibit 10.16 to the Company’s Registration Statement on Form S-1
(File No. 333-193309))
Form of Applied Genetic Technologies Corporation Warrant to Purchase Shares of Series A-1
Preferred Stock (incorporated by reference to Exhibit 10.17 to the Company’s Registration
Statement on Form S-1 (File No. 333-193309))
Form of Applied Genetic Technologies Corporation Warrant to Purchase Shares of Series B-1
Preferred Stock (incorporated by reference to Exhibit 10.18 to the Company’s Registration
Statement on Form S-1 (File No. 333-193309))
Warrant to Purchase Shares of Series A-1 Preferred Stock of Applied Genetic Technologies
Corporation issued to Silicon Valley Bank and effective on September 23, 2005 (incorporated by
reference to Exhibit 10.19 to the Company’s Registration Statement on Form S-1
(File No. 333-193309))
Warrant to Purchase Shares of Series A-1 Preferred Stock of Applied Genetic Technologies
Corporation issued to Silicon Valley Bank and effective on June 30, 2006 (incorporated by
reference to Exhibit 10.20 to the Company’s Registration Statement on Form S-1 (File
No. 333-193309))
Warrant to Purchase Shares of Series A-1 Preferred Stock of Applied Genetic Technologies
Corporation issued to Square 1 Bank on July 6, 2010 (incorporated by reference to Exhibit 10.21 to
the Company’s Registration Statement on Form S-1 (File No. 333-193309))
Warrant to Purchase Shares of Series B-1 Preferred Stock of Applied Genetic Technologies
Corporation issued to Square 1 Bank on August 31, 2012 (incorporated by reference to Exhibit
10.22 to the Company’s Registration Statement on Form S-1 (File No. 333-193309))
Form of Indemnification Agreement for Directors Associated with an Investment Fund
(incorporated by reference to Exhibit 10.23 to the Company’s Registration Statement on Form S-1
(File No. 333-193309))
Form of Indemnification Agreement for Directors Not Associated with an Investment Fund
(incorporated by reference to Exhibit 10.24 to the Company’s Registration Statement on Form S-1
(File No. 333-193309))
Second Amendment After Restated Amendment to License Agreement, made and effective as of
January 10, 2014, by and between the University of Florida Research Foundation, Inc. and Applied
Genetic Technologies Corporation (incorporated by reference to Exhibit 10.25 to the Company’s
Registration Statement on Form S-1 (File No. 333-193309))
24
(3) Index to Exhibits.
Exhibit
number
10.37†
10.38*
10.39†
10.40*
10.41*
Description
Fourth Amendment to Standard Exclusive License Agreement with Sublicensing Terms, made as
of December 17, 2013 by and between the University of Florida Research Foundation, Inc., Johns
Hopkins University and Applied Genetic Technologies Corporation (incorporated by reference to
Exhibit 10.26 to the Company’s Registration Statement on Form S-1 (File No. 333-193309))
Letter Agreement dated July 22, 2013 by and between the Company and Dan Menichella
(incorporated by reference to Exhibit 10.26 to the Company’s Registration Statement on Form S-1
(File No. 333-197385))
First Amendment to Non-Exclusive License, made as of March 28, 2014, by and between the
UAB Research Foundation, Inc. and Applied Genetic Technologies Corporation (incorporated by
reference to Exhibit 10.27 to the Company’s Registration Statement on Form S-1
(File No. 333-197385))
Letter Agreement dated January 22, 2014 by and between the Company and Larry Bullock
(incorporated by reference to Exhibit 10.28 to the Company’s Registration Statement on Form S-1
(File No. 333-197385))
Employment Letter Agreement dated as of October 29, 2016 between Applied Genetic
Technologies Corporation and Michael Goldstein (incorporated by reference to Exhibit 10.1 to the
Company’s Quarterly Report on Form 10-Q for the quarter ended December 31, 2016 (File
No. 001-36370))
23.1***
Consent of Independent Registered Public Accounting Firm
31.1**
31.2**
Certification of Principal Executive Officer pursuant to Rule 13a-14(a) or Rule 15d-14(a) of the
Securities Exchange Act of 1934, as adopted pursuant to Section 302 of the Sarbanes-Oxley Act of
2002
Certification of Principal Financial Officer pursuant to Rule 13a-14(a) or Rule 15d-14(a) of the
Securities Exchange Act of 1934, as adopted pursuant to Section 302 of the Sarbanes-Oxley Act of
2002
32.1***
Certification of Principal Executive Officer and Principal Financial Officer pursuant to 18 U.S.C.
Section 1350, as adopted pursuant to Section 906 of the Sarbanes-Oxley Act of 2002
101.INS*** XBRL Instance Document
101.SCH*** XBRL Taxonomy Extension Schema Document
101.CAL*** XBRL Taxonomy Extension Calculation Linkbase Document
101.DEF*** XBRL Taxonomy Extension Definition Linkbase Document
101.LAB*** XBRL Taxonomy Extension Label Linkbase Document
101.PRE*** XBRL Taxonomy Extension Presentation Linkbase Document
Management contract or compensatory plan or arrangement
Filed herewith
*
**
*** Previously filed
†
We have omitted portions of this exhibit, for which confidential treatment has been granted.
25
Pursuant to the requirements of Section 13 or 15(d) of the Securities Exchange Act of 1934, the registrant
has duly caused this report to be signed on its behalf by the undersigned thereunto duly authorized.
SIGNATURES
APPLIED GENETIC TECHNOLOGIES
CORPORATION
By:
/s/ Susan B. Washer
Susan B. Washer
President and Chief Executive Officer
Date: October 27, 2017
26
Management Team
Board of Directors
Susan B. Washer
President, Chief Executive Officer and Director
William A. Sullivan
Chief Financial Officer
Matthew Feinsod, M.D.
Interim Chief Medical Officer
Stephen W. Potter
Chief Business Officer
Mark S. Shearman, Ph.D.
Chief Scientific Officer
Andrew Ashe
General Counsel
Scott Koenig, M.D., Ph.D.
President, Chief Executive Officer and Director of
Macrogenics, Inc.
Susan B. Washer
President, Chief Executive Officer and Director of
Applied Genetic Technologies Corporation
Ed Hurwitz
Managing Director, MPM Capital
Ivana Magovcevic-Liebisch, Ph.D.
Executive Vice President, Chief Strategy and
Corporate Development Officer of Axcella Health Inc.
James Rosen
President and Chief Executive Officer of Artizan
Biosciences, Inc.
Anne VanLent
President of AMV Advisors
CORPORATE AND STOCKHOLDER INFORMATION
Corporate Headquarters
Applied Genetic Technologies Corporation
14193 NW 119th Terrace, Suite 10
Alachua, Florida 32615
www.agtc.com
Common Stock Listing
Our common stock is traded on the NASDAQ Global
Market under the symbol “AGTC.”
Independent Registered Public Accounting Firm
Ernst & Young LLP
200 Clarendon Street
Boston, Massachusetts 02116
Annual Meeting
The Company’s annual meeting of stockholders will
be held at 2:00 p.m., Eastern time, on February 28,
2018 at the offices of Foley Hoag LLP located at 155
Seaport Boulevard, Boston, Massachusetts 02210.
Investor Inquiries
The 2017 Annual Report, Form 10-K, as amended,
and other investor information are available free of
charge through the investor portion of our website at
ir.agtc.com.
Legal Counsel
Foley Hoag LLP
Seaport West
155 Seaport Boulevard
Boston, Massachusetts 02210
Transfer Agent
Computershare Trust Company, N.A.
250 Royall Street
Canton, Massachusetts 02021