UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, D.C. 20549
FORM 10-K
[X] ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934
FOR THE FISCAL YEAR ENDED DECEMBER 31, 2016
[ ] TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934
FOR THE TRANSITION PERIOD FROM ______________ TO ______________.
COMMISSION FILE NUMBER: 001-37348
Corbus Pharmaceuticals Holdings, Inc.
(Exact name of registrant as specified in its charter)
Delaware
(State or other jurisdiction of
incorporation or organization)
100 River Ridge Drive
Norwood, Massachusetts
(Address of principal executive offices)
46-4348039
(I.R.S. Employer
Identification No.)
02062
(Zip Code)
(617) 963-0100
Registrant’s telephone number, including area code:
Securities registered pursuant to Section 12(b) of the Act:
Title of Each Class
Common Stock, par value $0.0001 per share
Name of Each Exchange on which Registered
NASDAQ Global Market
Securities registered pursuant to Section 12(g) of the Act:
Title of each class
Common Stock, par value $0.0001 per share
Indicate by check mark if the registrant is a well-known seasoned issuer, as defined in Rule 405 of the Securities Act. Yes [ ] No
Indicate by check mark if the registrant is not required to file reports pursuant to Section 13 or Section 15(d) of the Act. Yes [ ]
[X]
No [X]
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 [X] 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 [X] No [ ]
Indicate by check mark if disclosure of delinquent filers pursuant to Item 405 of Regulation S-K is not contained herein, and will
not be contained, to the best of the 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 [ ]
Accelerated filer
Non-accelerated filer [ ] (Do not check if a smaller reporting company)
Smaller reporting company
[ ]
[X]
EXPLANATORY NOTE
The registrant met the “accelerated filer” requirements as of the end of its 2016 fiscal year pursuant to Rule 12b-2 of the Securities
Exchange Act of 1934, as amended. However, pursuant to Rule 12b-2 and SEC Release No. 33-8876, the registrant (as a smaller reporting
company transitioning to the larger reporting company system based on its public float as of June 30, 2016) is not required to satisfy the
larger reporting company requirements until its first quarterly report on Form 10-Q for the 2017 fiscal year and thus is eligible to check the
“Smaller Reporting Company” box on the cover of this Form 10-K.
Indicate by check mark whether the registrant is a shell company (as defined in Rule 12b-2 of the Act). Yes [ ] No [X]
As of June 30, 2016, the last business day of the registrant’s most recently completed second fiscal quarter, the aggregate market
value of the common stock held by non-affiliates of the registrant was approximately $121,536,402, based on the closing price of the
registrant’s common stock on June 30, 2016.
As of March 6, 2017, the number of shares outstanding of the registrant’s common stock, $0.0001 par value per share, was
48,701,626.
Portions of the registrant’s proxy statement for the 2017 annual meeting of stockholders to be filed pursuant to Regulation 14A
within 120 days after the registrant’s fiscal year ended December 31, 2016, are incorporated by reference in Part III of this Form 10-K.
Documents incorporated by reference
CORBUS PHARMACEUTICALS HOLDINGS, INC.
ANNUAL REPORT ON FORM 10-K
FOR THE YEAR ENDED DECEMBER 31, 2016
TABLE OF CONTENTS
ITEM
Business
Risk Factors
Unresolved Staff Comments
Properties
Legal Proceedings
Mine Safety Disclosures
PART I
PART II
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
Directors, Executive Officers and Corporate Governance
Executive Compensation
Security Ownership of Certain Beneficial Owners and Management and Related Stockholder Matters
Certain Relationships and Related Transactions, and Director Independence
Principal Accounting Fees and Services
PART III
Exhibits, Financial Statement Schedules
Form 10-K Summary
PART IV
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1B.
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7A.
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9A.
9B.
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SPECIAL NOTE REGARDING FORWARD-LOOKING STATEMENTS
PART I
This report on Form 10-K contains forward-looking statements made pursuant to the safe harbor provisions of the Private
Securities Litigation Reform Act of 1995 under Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities
Exchange Act of 1934, as amended. Forward-looking statements include statements with respect to our beliefs, plans, objectives, goals,
expectations, anticipations, assumptions, estimates, intentions and future performance, and involve known and unknown risks, uncertainties
and other factors, which may be beyond our control, and which may cause our actual results, performance or achievements to be materially
different from future results, performance or achievements expressed or implied by such forward-looking statements. All statements other
than statements of historical fact are statements that could be forward-looking statements. You can identify these forward-looking
statements through our use of words such as “may,” “can,” “anticipate,” “assume,” “should,” “indicate,” “would,” “believe,”
“contemplate,” “expect,” “seek,” “estimate,” “continue,” “plan,” “point to,” “project,” “predict,” “could,” “intend,” “target,” “potential”
and other similar words and expressions of the future.
There are a number of important factors that could cause the actual results to differ materially from those expressed in any
forward-looking statement made by us. These factors include, but are not limited to:
● our lack of operating history;
● our current and future capital requirements and our ability to satisfy our capital needs;
● our ability to complete required clinical trials of our product and obtain approval from the FDA or other regulatory agents in
different jurisdictions;
● our ability to maintain or protect the validity of our patents and other intellectual property;
● our ability to retain key executive members;
● our ability to internally develop new inventions and intellectual property;
● interpretations of current laws and the passages of future laws;
● acceptance of our business model by investors;
● the accuracy of our estimates regarding expenses and capital requirements; and
● our ability to adequately support growth.
The foregoing does not represent an exhaustive list of matters that may be covered by the forward-looking statements contained
herein or risk factors that we are faced with that may cause our actual results to differ from those anticipate in our forward-looking
statements. Please see “Risk Factors” for additional risks which could adversely impact our business and financial performance.
All forward-looking statements are expressly qualified in their entirety by this cautionary notice. You are cautioned not to place
undue reliance on any forward-looking statements, which speak only as of the date of this report or the date of the document incorporated
by reference into this report. We have no obligation, and expressly disclaim any obligation, to update, revise or correct any of the forward-
looking statements, whether as a result of new information, future events or otherwise. We have expressed our expectations, beliefs and
projections in good faith and we believe they have a reasonable basis. However, we cannot assure you that our expectations, beliefs or
projections will result or be achieved or accomplished.
Item 1.
BUSINESS
Overview
This report and the information incorporated herein by reference contain references to trademarks, service marks and trade names
owned by us or other companies. Solely for convenience, trademarks, service marks and trade names referred to in this report and the
information incorporated herein, including logos, artwork, and other visual displays, may appear without the ® or ™ symbols, but such
references are not intended to indicate, in any way, that we will not assert, to the fullest extent under applicable law, our rights or the rights
of the applicable licensor to these trademarks, service marks and trade names. We do not intend our use or display of other companies’
trade names, service marks or trademarks to imply a relationship with, or endorsement or sponsorship of us by, any other companies. Other
trademarks, trade names and service marks appearing in this report are the property of their respective owners.
1
We are a clinical stage pharmaceutical company, focused on the development and commercialization of novel therapeutics to treat
rare, chronic and serious inflammatory and fibrotic diseases with clear unmet medical needs. Our product JBT-101 is a novel synthetic oral
endocannabinoid-mimetic drug that is intended to resolve chronic inflammation and halt fibrotic processes without causing
immunosuppression. JBT-101 is currently being developed to treat four life-threatening diseases: systemic sclerosis, cystic fibrosis, diffuse
cutaneous, skin-predominant dermatomyositis and systemic lupus erythematosus (“SLE’’).
In November 2016, we reported positive clinical data in a Phase 2 JBT-101 study for the treatment of systemic sclerosis. We
conducted an end of Phase 2 meeting with the FDA in late February 2017 and we expect to begin a Phase 3 clinical program in systemic
sclerosis by the end of the third quarter of 2017. Based on the positive Phase 2 clinical results in systemic sclerosis, we also filed an
application with the FDA in March 2017 for Breakthrough Therapy designation.
In December 2016, we completed a second Phase 2 study in cystic fibrosis study and expect to report top-line data from this study
by the end of March 2017. A third Phase 2 study in dermatomyositis is expected to be completed in the third quarter of 2017 and a fourth
Phase 2 in SLE is planned to start during the second quarter of 2017. The United States Food and Drug Administration (“FDA”) has
granted JBT-101 Orphan Designation as well as Fast Track Status for both cystic fibrosis and systemic sclerosis.
Since our inception, we have devoted substantially all of our efforts to business planning, research and development, recruiting
management and technical staff, acquiring operating assets and raising capital. Our research and development activities have included
conducting pre-clinical studies, developing manufacturing methods and the manufacturing of our drug JBT-101 for clinical trials and
conducting clinical studies in patients. Three of the four clinical programs for JBT-101 are being supported by non-dilutive awards and
grants. The NIH is funding the majority of the Phase 2 clinical development costs for the dermatomyositis and SLE Phase clinical trials and
the Phase 2 clinical trial in cystic fibrosis has been supported by a $5 million award from the Cystic Fibrosis Foundation Therapeutics, Inc.
(“CFFT”), a non-profit drug discovery and development affiliate of the Cystic Fibrosis Foundation.
JBT-101 is a synthetic, rationally-designed oral small molecule drug that selectively binds to the cannabinoid receptor type 2, or
CB2, found on activated immune cells, fibroblasts and muscle cells. JBT-101 stimulates the production of Specialized Pro-Resolving Lipid
Mediators (SPMs) that act to resolve inflammation, and halt fibrosis by activating endogenous pathways. These endogenous resolution
pathways are normally activated in healthy individuals during the course of normal immune responses but are dysfunctional in chronic
inflammatory and fibrotic diseases. Through its’ activation of the CB2 receptor, JBT-101 is designed to drive innate immune responses
from the activation phase through completion of the resolution phase. The CB2 receptor plays an endogenous role in modulating and
resolving inflammation by, in effect, turning heightened inflammation “off” and restoring homeostasis.
A key aspect of the body’s innate immune response is its activation phase is the recruitment of inflammatory cells to the site of
tissue infection/injury whereupon these cells act to destroy the infection and/or repair tissue damage. The next phase in a normal innate
immune response is its resolution phase, during which the nature of the infiltrating immune cells changes from pro-inflammatory to pro-
resolving, the infectious pathogens are eliminated, residual cellular debris and immune cells are cleared from the tissue, and tissue repair
processes are eventually halted when they are no longer needed. In chronic inflammatory and fibrotic diseases, the innate immune
responses are “stuck” in the initial activation phase. This failure to progress through the resolution phase causes chronic tissue infiltration
with inflammatory cells and chronic activation of healing processes that cause tissue scarring, or fibrosis. The key event that propels an
innate immune response from its activation phase to its resolution phase is a “class switch” from production of pro-inflammatory lipid
mediators such as prostaglandins and leukotrienes to a family of SPMs (Figure 1) which include lipoxins, resolvins, protectins, and
marescins. If an innate immune response persists in the activation phase and does not progress through resolution, chronic inflammation and
fibrosis can result, causing organ dysfunction, organ failure, severe morbidity and even death. There are hundreds of inflammatory
diseases, many of which are chronic, life-long and incurable.
2
Figure 1. JBT-101’s Mechanism of Action
JBT-101 is designed to restore immune system homeostasis on a daily basis, by using the body’s own physiologic pathways to
transition an innate immune response from the activation phase to the resolution phase. If an innate immune response is “stuck” in the
activation phase, tissue damage, fibrosis and persistent infection are expected consequences. Endogenous progression of the innate immune
response through its resolution phase has been shown to clear inflammation, stop fibrosis, and promote pathogen clearance. JBT-101’s
unique mechanism of action is different than anti-inflammatory drugs which inhibit production or functions of certain pro-inflammatory
mediators that initiate or are active during the activation phase. Activation of an innate immune response is necessary to clear infections, so
that drugs that interfere with the activation phase carry the risk of immunosuppression and may have other undesirable side effects. In
contrast, JBT-101 is designed to transition an innate immune response from its activation phase to resolution phase. JBT-101’s CB2 agonist
activity initiates a class switch in bioactive lipid mediators from inflammation-activating mediators to pro-resolving mediators.
The development status of JBT-101 is summarized below:
Figure 2: Drug developmental pipeline
3
Positive Clinical Results in Systemic Sclerosis
On November 14, 2016, we announced positive topline results from our Phase 2 study evaluating JBT-101 for the treatment of
diffuse cutaneous systemic sclerosis (“systemic sclerosis”). JBT-101 out-performed placebo in the American College of Rheumatology
(ACR) Combined Response Index in diffuse cutaneous Systemic Sclerosis (CRISS) score, reaching 33% at week 16, versus 1% for placebo.
The higher the CRISS score the greater the improvement. The difference in CRISS scores between JBT-101 and placebo groups over the
trial period was significant (p = 0.044), 1-sided mixed model repeated measures using rank transformed data. Differences in categorical
levels of CRISS responses and changes from baseline in the five individual domains of the CRISS score also supported clinical benefit of
JBT-101.
The multi-center, double-blind, randomized, placebo-controlled Phase 2 study evaluated JBT-101’s clinical benefit and safety in
27 subjects who received JBT-101 and 15 who received placebo. Subjects had disease duration up to 6 years and were allowed to receive
stable doses of immunosuppressive drugs during this study. Subjects were randomized (2 to 1 overall JBT-101 to placebo ratio) to receive
JBT-101 for the first four weeks at 5 mg once a day (n = 9), 20 mg once a day (n = 9), or 20 mg twice a day (n = 9) or placebo for the first
four weeks, then all JBT-101 subjects received 20 mg twice a day for the next 8 weeks. All subjects were followed off study drug from
weeks 13 through 16. The primary efficacy objective was to evaluate clinical benefit in all subjects who received JBT-101 versus subjects
who received placebo using the ACR CRISS score, a measure of improvement in systemic sclerosis. The CRISS is an exponentially
weighted algorithm of change from baseline that includes the modified Rodnan skin score (mRSS), a measure of skin thickening, physician
global assessment (MDGA), patient global assessment (PtGA), and Health Assessment Questionnaire - Disability Index (HAQ-DI), and
forced vital capacity (FVC).
Results:
The median (25th percentile, 75th percentile) CRISS scores for the combined JBT-101 group and the placebo group at weeks 4, 8,
12, and 16 are provided in the table below. The difference in CRISS scores between JBT-101 and placebo groups over the trial period was
significant (p = 0.044), 1-sided mixed model repeated measures using rank transformed data.
Group
JBT-101
n = 26
Placebo
n = 15
Median CRISS Score1, % (25th percentile, 75th percentile)
Week 4
Week 8
Week 12
Week 16
3%
(0.6%, 11.4%)
19%
(0.3%, 69.2%)
27.5%
(1.9%, 67.8%)
33%
(0.8%, 82.1%)
1%
(0.3%, 8.8%)
1%
(0.1%, 15.2%)
1%
(0.1%, 60.1%)
1%
(0.1%, 16%)
1) Modified intent to treat population, last observation carried forward
There were no serious, severe, or unexpected adverse events related to JBT-101. One of 27 subjects (3.7% of subjects) who
received JBT-101 withdrew from the study for an adverse event which was moderate dizziness.
We conducted an end of Phase 2 meeting with the FDA in late February 2017 and we expect to begin a Phase 3 clinical program in
systemic sclerosis by the end of the third quarter of 2017. Based on the positive Phase 2 clinical results in systemic sclerosis, we also filed
an application with the FDA in March 2017 for Breakthrough Therapy designation.
Market Opportunity in Inflammatory and Fibrotic Diseases
There are many different chronic, serious inflammatory and fibrotic diseases. Some examples of chronic, serious diseases
characterized by inflammation with variable degrees of fibrosis include genetic diseases such as cystic fibrosis, nonalcoholic steatohepatitis
(“NASH”), autoimmune diseases including systemic sclerosis, systemic lupus erythematosus, myositis, rheumatoid arthritis, vasculitis,
primary biliary cirrhosis and lung diseases including idiopathic pulmonary fibrosis, bronchiolitis obliterans, and sarcoidosis.
According to Global Business Intelligence Research, the global market for drugs to treat chronic inflammation in 2010 was
approximately $58 billion and is expected to grow to approximately $86 billion by 2017. While some chronic inflammatory diseases are
very common, for example, about 28.5 million Americans have chronic sinusitis and about 18.7 million Americans have asthma, our initial
focus is on chronic, serious inflammatory and fibrotic diseases that are rare or uncommon and have significant unmet medical need. Some
of these diseases can be categorized as “orphan diseases” in the U.S., meaning they affect no more than 200,000 patients each. Examples
include cystic fibrosis, systemic sclerosis and idiopathic pulmonary fibrosis. The advantage of targeting these serious uncommon diseases
is that they have the potential for accelerated regulatory approval through orphan drug designation, fast track designation and breakthrough
therapy designation.
4
JBT-101 Market Opportunity for Current Indications Being Developed
Autoimmune Disorders
Systemic Sclerosis
Systemic sclerosis is a chronic, systemic autoimmune disease characterized by activation of innate and adaptive immune systems,
an obliterative, proliferative vasculopathy of small blood vessels, and fibrosis of the skin and multiple internal organs. Approximately
90,000 people in the United States and Europe have systemic sclerosis. The disease affects mainly adults (80% of systemic sclerosis
patients are women) with mean age of onset about 46 years of age in the United States and the majority of patients between 45-64 years of
age.
A commonly used system classifies systemic sclerosis patients into those with more wide-spread skin thickening (diffuse
cutaneous systemic sclerosis, about 45% of patients) and those with more restricted skin thickening (limited cutaneous systemic sclerosis,
about 55% of patients). There is significant overlap in the clinical manifestations for these two groups of systemic sclerosis patients and no
known significant differences in disease pathogenesis.
Systemic sclerosis can affect multiple internal organs in the body, including the lungs, heart, kidneys, joints, muscles, esophagus,
stomach and intestines. Clinically apparent organ involvement that occurs in more than a third of these patients includes thickened skin,
Raynaud’s phenomenon, esophageal symptoms, pulmonary fibrosis, restrictive lung disease, edematous skin, joint contractures, digital
ulcers, and muscle weakness. Less frequently occurring, yet life-threatening manifestations include pulmonary artery hypertension (about 1
in 5 patients), cardiac conduction blocks (about 1 in 10 patients), and renal crisis (about 1 in 50 patients). In the US, systemic sclerosis is the
most deadly of the systemic autoimmune diseases. The median disease duration for an individual who dies of systemic sclerosis is 7.1 years
from the onset of symptoms. About 85% of deaths caused by systemic sclerosis are the result of pulmonary fibrosis, pulmonary artery
hypertension, or cardiovascular disease, such as sudden death.
In systemic sclerosis the innate immune system fails to transition from the activation phase to the resolution phase. Individuals
with systemic sclerosis who have interstitial lung disease have an imbalance of bioactive lipid mediators, causing a predominance of
inflammatory mediators versus resolving mediators. The preponderance of inflammatory mediators correlates positively with the degree of
inflammation in the lungs and negatively with forced vital capacity, a measure of lung fibrosis. Excessive activation of the pathways which
cause fibrosis including TGFβ, myofibroblast accumulation, and production of collagen and other extracellular matrix proteins are all
present in systemic sclerosis.
Currently, there are no FDA-approved therapies specifically for systemic sclerosis, except for the pulmonary artery hypertension
associated with this disease. Immunosuppressants with significant toxicities are also commonly used to treat systemic sclerosis, however
there is a general absence of clinical data to support their use. For example, systemic corticosteroids are used frequently in systemic
sclerosis patients despite concerns about toxic side effect and precipitation of renal crisis.
We believe there is general agreement in the systemic sclerosis community that an effective anti-inflammatory and anti-fibrotic
drug would address a significant unmet medical need in systemic sclerosis, especially a drug that is orally administered, can be used
chronically with other commonly prescribed medications for systemic sclerosis, and is not immunosuppressive. We believe such a therapy
would be positively received by the market.
Dermatomyositis
Dermatomyositis is a serious and rare autoimmune idiopathic inflammatory myopathy with characteristic cutaneous findings.
About 30,000 individuals in the U.S. suffer from dermatomyositis. Dermatomyositis usually strikes adults, with most common age of adult
onset between 50-60 years.
This systemic disorder most frequently affects the skin and muscles, and dermatomyositis can also include interstitial lung
disease/restrictive lung disease, arthritis, gastrointestinal and cardiac involvement. Inflammatory muscle disease can cause discomfort and
significant weakness of the proximal muscles of the arms and legs and of the trunk. Dermatomyositis can include damaging inflammation
elsewhere in the body, for example: lung inflammation that leads to lung fibrosis and restrictive lung disease; heart inflammation that
causes arrhythmia, congestive heart failure, and pericarditis, inflammation of muscles in the esophagus that causes swallowing problems or
aspiration pneumonia, and arthritis. Dermatomyositis patients may have active skin disease despite successful treatment of their muscle
and/or lung disease. The skin findings in dermatomyositis can be disfiguring and are inflammatory rashes characterized by redness and
itching in exposed areas of the skin, around the eyes, on the hands, and in a “shawl” distribution on the scalp, hands, upper back, and photo-
exposed areas. With this chronic inflammation, patients with dermatomyositis have an increased risk of malignancy, most commonly in
older patients By itself, skin involvement in dermatomyositis has a large negative impact on quality of life, comparable to that of cutaneous
lupus erythematosus and vulvodynia, and much higher than those of many dermatologic diseases. The pathophysiology of dermatomyositis
is also consistent with inability of patients to adequately resolve innate immune responses.
5
Therapy for dermatomyositis involves both general measures and specific measures to control the muscle disease and the skin
disease. In addition, some patients with dermatomyositis need treatment for other systemic manifestations or complications. The muscle
component is treated by administering corticosteroids, typically with an immunosuppressive agent. The skin disease is treated by avoiding
sun exposure and by using sunscreens and photoprotective clothing, as well as with topical corticosteroids, and antimalarial agents.
Antimalarial therapy frequently is ineffective or can cause drug reactions. Antimalarial-refractory disease is then treated with systemic
therapies that may additionally cause toxicity, including systemic glucocorticoids, immunosuppressive therapies such as methotrexate,
mycophenolate mofetil, or intravenous immunoglobulin.
We believe that an effective drug that controls inflammation in the skin, muscles, and other organs will address a significant
unmet medical need in dermatomyositis, particularly a drug that is orally administered, can be used chronically with other commonly
prescribed medications for the disease, and is not immunosuppressive.
Systemic Lupus Erythematosus
Systemic lupus erythematosus, or SLE, is a prototypical autoimmune disease with a wide array of clinical manifestations,
including arthritis, rash, photosensitivity, oral ulcers, pleuritis, pericarditis, kidney problems, seizures and psychosis and blood cell
abnormalities. The musculoskeletal system is the most commonly involved system in SLE. Patients with SLE have an increased frequency
of related autoimmune problems, such as Sjogren’s syndrome and antiphospholipid syndrome that require additional treatments. SLE may
occur with other autoimmune conditions, such as thyroiditis, hemolytic anemia, and idiopathic thrombocytopenia purpura. Accelerated
atherosclerosis among SLE patients is responsible for premature mortality.
The pathology of SLE involves chronic activation of the innate immune system by immune complexes, with activation of
complement, increased production of type 1 interferons and other mediators of inflammation and resultant tissue inflammation and damage.
Drugs specifically approved by the FDA for SLE are limited to aspirin, corticosteroids, hydroxychloroquine and belimumab.
Physicians commonly treat SLE disease manifestations with immunosuppressive or corticosteroid therapies that have significant toxicities.
We believe that an effective drug that controls inflammation in the joints and skin as well as improves overall disease activity will
address a significant unmet medical need in SLE, particularly a drug that is orally administered, can be used chronically with other
commonly prescribed medications for the disease, and is not immunosuppressive.
Cystic Fibrosis
Cystic fibrosis is a life-long, progressive, debilitating, and life-threatening autosomal recessive disease. Cystic fibrosis is caused
by mutations in the gene Cystic Fibrosis Transmembrane Conductance Regulator or CFTR. The CFTR serves as a central hub to modulate
transport, trafficking, and signaling in cells. Given multiple roles of CFTR in cellular activation and homeostasis, mutation of the CFTR
give rise to multiple disorders in respiratory, digestive and reproductive organs.
The CFTR mutations lead to defective ion transport, with reduced chloride and bicarbonate secretion and sodium hyper-absorption,
followed by water hyper-absorption, by airway epithelia and other cell types. The resultant reduced height of epithelial lining fluid and
decreased hydration of mucus results in abnormally thick and sticky mucus, which obstructs the lumen into which the mucus is secreted and
reduces mucociliary clearance of bacteria. The dysfunction in ion transport in CF patients is reflected in abnormal sweat chloride levels.
The negative effects caused by CFTR mutations are not restricted to ion channels, but also extend to dysfunction of the innate
immune system. The nature of the abnormalities in CF are consistent with inability of innate immune responses to make the transition out
of the activation phase and into and through the resolution phase. Bioactive lipid mediators (SPMs) that initiate the transition to resolution
phase of innate immune responses have been found to be deficient relative to pro-inflammatory lipid mediators that initiate its activation
phase, and this reduction correlates with poor recovery of lung function following an acute pulmonary exacerbation in children. The
preponderance of activated neutrophils and pro-inflammatory macrophages in inflamed tissue, reduced neutrophil apoptosis, high levels of
neutrophil proteases that reflect persistent neutrophil activation, reduced clearance of neutrophils by macrophages, ineffective clearance of
certain bacteria such as P. aeruginosa, and excessive activation of fibrotic pathways all show the inability of individuals with CF to resolve
their innate immune responses.
An overview of the disease progression in cystic fibrosis is provided in Figure 3.
6
Figure 3: Factors involved in cystic fibrosis progression
As a result of obstructing secretions, recurrent infections, hyper-inflammation, and activated fibrotic pathways in the lungs,
individuals with CF develop bronchiectasis, pulmonary fibrosis, mixed obstructive/restrictive lung disease, and, eventually, respiratory
failure. They may also have chronic sinusitis and nasal polyps. The same pathophysiologic events of obstruction, infection, chronic
inflammation, and tissue damage/fibrosis occur in the gastrointestinal system, which can lead to bowel obstructions, fat malabsorption,
bacterial overgrowth, gut dysmotility, malnutrition, growth retardation, low weight, pancreatic insufficiency, cystic fibrosis-related
diabetes, gallstones, and liver failure including cirrhosis. Adult males with cystic fibrosis have degeneration of the ductus deferens and
sterility. End-stage organ involvement in cystic fibrosis is sometimes treated with transplantation, especially lung transplantation.
The median current life expectancy of cystic fibrosis patients is about 40 years. According to the Cystic Fibrosis Foundation,
30,000 Americans and a total of 75,000 people in the United States and Europe suffer from cystic fibrosis.
Current therapies for cystic fibrosis include mucolytics to breakdown mucus, antibiotics to fight bacterial infection, and drugs that
act to restore some functionality to the faulty CFTR protein in specific genetic sub-populations of patients, including Kalydeco™ and
Orkambi™. Kalydeco™ was approved for treatment of cystic fibrosis in January 2012 and Orkambi was approved in June 2015. With
about 2,000 different known mutations in the CFTR gene, Kalydeco™ can be used by approximately 4,000 patients who suffer from a
limited number of “gating mutations,” including the G551D mutation. Orkambi™ can be used by approximately 25,000 adult patients
carrying two copies (homozygotes) of the delta508 CFTR mutation.
Of importance, drugs that are sufficient to correct ion channel functions of mutant CFTR protein are not necessarily able to correct
the dysfunction of the innate immune system. For example, ivacaftor treatment has not been associated with reduction in sputum
neutrophils or neutrophil derived proteases in CF patients. Thus correction of ion channel dysfunction does not necessarily translate into
correction of dysfunction of the innate immune system.
All CF patients appear to have dysfunction in resolution of the innate immune system, no matter which CFTR mutations a given
patient has. Currently, there is no drug that is used commonly to address this basic problem in CF, other than antibiotics to control infection
and, indirectly, control inflammation. The use of high dose ibuprofen as an anti-inflammatory treatment, which has been shown to be
beneficial, is limited to about 3-5% of CF patients since there is a need to monitor levels closely in children and due to side effect risks,
primarily gastrointestinal bleeding. The Cystic Fibrosis Foundation Strategic Plan Report, 2014-2018, includes a strategic priority to
identify new treatments for CF that allow all patients to better manage the symptoms of their disease and improve their health, with a
specific mention of the need for new agents that can resolve inflammation.
We believe there is general agreement in the CF community that an effective drug that will reduce hyper-inflammation and help
clear infections would address a significant unmet medical need in CF, especially a drug that is orally administered, can be used chronically
with other prescribed medications for CF, is not immunosuppressive, and has anti-fibrotic effects.
7
Current Treatment Alternatives for Chronic, Serious Diseases Characterized by Chronic Inflammation and Fibrosis
Overview
Drugs currently used to treat chronic, serious diseases with chronic inflammation and fibrosis are divided broadly into several
groups: non-steroidal anti-inflammatory drugs (NSAIDS), anti-malarial agents, systemic corticosteroids, and immunosuppressive agents.
The choice of agent or combination generally depends upon the underlying disease, and physician and patient preferences.
The potency of NSAIDs in the treatment of chronic, serious diseases with chronic inflammatory and fibrotic diseases is often too
limited to control disease activity, requiring patients to receive additional treatment with anti-malarial drugs, systemic corticosteroids or
immunosuppressive agents. Anti-malarial therapy is used as a baseline treatment for chronic inflammation in certain autoimmune diseases,
typically SLE and dermatomyositis, especially in patients with milder manifestations of disease. Anti-malarial therapy frequently is
ineffective in controlling chronic, serious inflammation, or can cause drug reactions. Antimalarial-refractory disease is then treated with
systemic therapies that may additionally cause toxicity, including systemic corticosteroids and immunosuppressive agents.
Systemic corticosteroids are commonly prescribed for treatment of chronic, serious diseases characterized by chronic
inflammation and fibrosis, such as cystic fibrosis, systemic sclerosis, and dermatomyositis. Chronic corticosteroid use is limited by
toxicities that include growth retardation, iatrogenic Cushings’s Disease, hypertension, high glucose levels/diabetes, obesity, brittle
bones/osteoporosis, aseptic necrosis of bone, immunosuppression/increased infection, glaucoma, depression, and psychosis. Thus, safer yet
potent alternatives to steroids have long been sought.
Multiple other immunosuppressive drugs are used to treat chronic, serious, inflammatory diseases, to achieve disease control and
to reduce or avoid the need for corticosteroids. These include biological agents, such as monoclonal antibodies or fusion proteins, which
target a very specific molecule in a key disease pathway. These drugs have a number of disadvantages including that the drugs must be
administered by parenterally and they are associated with increased incidence of malignancy and infection. Non-biologic
immunosuppressive agents that are used to treat chronic, serious inflammation include methotrexate, mycophenolate, leflunomide,
cyclophosphamide, and azathioprine, among others. Intravenous immunoglobulin is used occasionally to treat refractory chronic, serious
inflammatory diseases.
Autoimmune Disorders
Systemic Sclerosis
Cytotoxic and immunosuppressive medications are used to control overall disease activity in systemic sclerosis. In a study of 2,739
systemic sclerosis patients in the United States, in one year 44.3% received corticosteroids, 4.8% received mycophenolate mofetil, 2.7%
received cyclophosphamide, and 0.5% received cyclosporine. In a report of 7,655 patients in the European Scleroderma Trials and
Research Group database, immunosuppressant treatments used to treat systemic sclerosis and the percentage of patients receiving them
were: prednisone (43.5%) with median dose of 8 mg/day; cyclophosphamide (15.9%); methotrexate (13.7%); azathioprine (6.4%);
mycophenolate mofetil (4.2%), d-penicillamine (2.1%), and rituximab (1%).
Dermatomyositis
Current medications for dermatomyositis involve both treatments to reduce overall disease activity and specific treatments to
control the muscle disease and the skin disease. The muscle component is treated by administering corticosteroids, typically with an
immunosuppressive agent. The skin disease is treated by avoiding sun exposure and by using sunscreens and photoprotective clothing, as
well as with topical corticosteroids, antimalarial agents such as hydroxychloroquine and immunosuppressive medications such as
methotrexate, azathioprine, mycophenolate mofetil, or intravenous immunoglobulin.
Systemic Lupus Erythematosus
Similar to dermatomyositis, current medications for SLE involve treatments to reduce overall disease activity and specific
treatments for a given organ involvement. Commonly used medications include NSAIDs, topical corticosteroids, antimalarial agents,
prednisone, belimumab, and other
immunosuppressive medications such as mycophenolate, methotrexate, azathioprine, and
cyclophosphamide.
Cystic Fibrosis
The importance of treating inflammation in cystic fibrosis is confirmed in the Cystic Fibrosis Foundation’s Strategic Plan, 2014-
2018. While treatment with systemic corticosteroids and ibuprofen are effective in improving the symptoms of cystic fibrosis, the side
effects associated with chronic treatment using these drugs are significant. Specifically, long term usage of oral corticosteroids in children
are associated with glucose intolerance, cataract formation, multiple bone fractures secondary to osteoporosis or osteopenia, Cushing
disease effects, and anorexia nervosa as well as growth retardation. The use of high dose ibuprofen is limited by the years of treatment it
takes to show benefit, a need to monitor levels closely in the patient, and the increased risk of gastrointestinal bleeding. As a result, these
drugs have limited long-term use in cystic fibrosis.
8
Other therapies routinely used by cystic fibrosis patients include antibiotics, such as Cayston from Gilead and TOBI from Novartis,
and mucolytics, such as Pulmozyme from Genentech. In addition, Vertex currently markets the only approved drugs that specifically target
the defective CFTR protein, Kalydeco and Orkambi.
JBT-101’s Mechanism of Action is Distinct from Anti-Inflammatory Drugs, Steroids and Immunosuppressive Agents
Corticosteroids and NSAIDs exert their effect by inhibiting the activation of inflammation. In simple terms, both classes of drugs
inhibit inflammation by “interfering” with the biochemical pathways in the cell that promote and sustain inflammation. For example,
NSAIDs directly inhibit the activity of the COX 1 and COX 2 enzymes that are responsible for generating pro-inflammatory eicosanoids. A
drawback of this approach is that it only inhibits one arm of the eicosanoid pathway (e.g. COX but not LOX) resulting in a buildup in LOX-
derived inflammatory mediators which leads to gastrointestinal and cardiovascular side effects (termed “molecular shunting”).
JBT-101 on the other hand triggers an endogenous pathways that resolves inflammation and halts fibrosis without
immunosuppression. JBT-101 acts to impact and activate multiple pathways including:
● Increase in production of SPMs and anti-inflammatory eicosanoids, with a concomitant decrease in production of pro-
inflammatory eicosanoids.
● Increase in production of anti-inflammatory cytokines, coupled with a decrease in production of pro-inflammatory cytokines
and pro-fibrotic growth factors.
● Increase in influx of non-inflammatory macrophages with a decrease in influx and accumulation of inflammatory cells and pro-
fibrotic myofibroblasts.
● Increase in bacterial clearance. SPMs stimulate production of bactericidal peptides, enhance phagocytosis and killing of bacteria
by neutrophils and macrophages.
● Increase in apoptosis of inflammatory cells, including neutrophil and pro-fibrotic cells, including fibroblasts.
● Increase in clearance of apoptotic cells and cellular debris by non-inflammatory macrophages.
JBT-101 potentially offers a new and unique mechanism to treat a spectrum of rare, chronic, serious inflammatory and fibrotic
diseases.
Clinical Trials
Overview
We have completed Phase 2 randomized, double-blind, placebo-controlled clinical studies in systemic sclerosis and cystic fibrosis
and are currently conducting a Phase 2 study in dermatomyositis that is expected to be completed in the third quarter of 2017. In addition
we plan to commence a Phase 2 clinical study during the second quarter of 2017 in Systemic Lupus Erythematosus, or SLE.
We have obtained Orphan Drug Designations for both CF and systemic sclerosis in the United States and European Union and
Fast Track status for both CF and systemic sclerosis in the United States. Fast Track designation is eligible for some or all of the following:
(i) more frequent meetings with FDA to discuss the drug’s development plan and ensure collection of appropriate data needed to support
drug approval; (ii) more frequent written correspondence from FDA about things such as the design of the proposed clinical trial and the
use of biomarkers; and (iii) eligibility for Accelerated Approval and Priority Review, if relevant criteria are met. Based on the positive
Phase 2 clinical results in systemic sclerosis, we filed an application with the FDA in March 2017 for Break Through Therapy designation.
Systemic Sclerosis (Scleroderma)
In August 2015, we initiated a Phase 2 double-blind placebo-controlled clinical study in patients suffering from diffuse cutaneous
systemic sclerosis that was completed in October 2016. The Principal Investigator was Dr. Robert Spiera of the Hospital of Special
Surgery, New York City, New York.
9
This multi-center, double-blind, randomized, placebo-controlled Phase 2 study evaluated JBT-101’s clinical benefit and safety in
27 subjects who received JBT-101 and 15 who received placebo. Subjects had disease duration up to 6 years and were allowed to receive
stable doses of immunosuppressive drugs during this study. Subjects were randomized (2 to 1 overall JBT-101 to placebo ratio) to receive
JBT-101 for the first four weeks at 5 mg once a day (n = 9), 20 mg once a day (n = 9), or 20 mg twice a day (n = 9) or placebo for the first
four weeks, then all JBT-101 subjects received 20 mg twice a day for the next 8 weeks. All subjects were followed off study drug from
weeks 13 through 16. The primary efficacy objective was to evaluate clinical benefit in all subjects who received JBT-101 versus subjects
who received placebo using the ACR CRISS score, a measure of improvement in systemic sclerosis. The CRISS is an exponentially
weighted algorithm of change from baseline that includes the modified Rodnan skin score (mRSS), a measure of skin thickening, physician
global assessment (MDGA), patient global assessment (PtGA), and Health Assessment Questionnaire - Disability Index (HAQ-DI), and
forced vital capacity (FVC). . Secondary endpoint measurements include JBT-101 concentrations and metabolites, other patient-reported
outcomes, biomarkers of inflammation and fibrosis in the blood and skin, and metabolipidomic profiles.
In November 2016, we announced positive topline results from this study. JBT-101 out-performed placebo in the American
College of Rheumatology (ACR) Combined Response Index in diffuse cutaneous Systemic Sclerosis (CRISS) score, reaching 33% at week
16, versus 1% for placebo. The higher the CRISS score the greater the improvement. The difference in CRISS scores between JBT-101 and
placebo groups over the trial period was significant (p = 0.044), 1-sided mixed model repeated measures using rank transformed data.
Differences in categorical levels of CRISS responses and changes from baseline in the five individual domains of the CRISS score also
supported clinical benefit of JBT-101.
Top-Line Results:
The median (25th percentile, 75th percentile) CRISS scores for the combined JBT-101 group and the placebo group at weeks 4, 8,
12, and 16 are provided in the table below. The difference in CRISS scores between JBT-101 and placebo groups over the trial period was
significant (p = 0.044), 1-sided mixed model repeated measures using rank transformed data.
Group
JBT-101
n = 26
Placebo
n = 15
Median CRISS Score1, % (25th percentile, 75th percentile)
Week 4
Week 8
Week 12
Week 16
3%
(0.6%, 11.4%)
19%
(0.3%, 69.2%)
27.5%
(1.9%, 67.8%)
33%
(0.8%, 82.1%)
1%
(0.3%, 8.8%)
1%
(0.1%, 15.2%)
1%
(0.1%, 60.1%)
1%
(0.1%, 16%)
1) Modified intent to treat population, last observation carried forward
Results of secondary efficacy outcome measures supported the finding of clinical benefit of JBT-101, including numerical
superiority of JBT-101 in each of the five domains of the CRISS score, with divergence starting early at Week 4 or Week 8.
There were no serious, severe, or unexpected adverse events related to JBT-101. One of 27 subjects (3.7% of subjects) who
received JBT-101 withdrew from the study for an adverse event which was moderate dizziness.
We conducted an end of Phase 2 meeting with the FDA in late February 2017 and we expect to begin a Phase 3 clinical program in
systemic sclerosis by the end of the third quarter of 2017. Based on the positive Phase 2 clinical results in systemic sclerosis, we also filed
an application with the FDA in March 2017 for Breakthrough Therapy designation.
In April 2016, the FDA granted approval for a one year open-label extension to the Phase 2 clinical study in systemic sclerosis.
The objective of the open-label extension study is to provide all subjects (treatment and placebo) with the option of receiving JBT-101 for a
period of one year after they complete the Phase 2 study. The same safety and efficacy endpoints evaluated in the Phase 2 study are being
assessed throughout the one year extension study. The Company expects to report data from this study in the second half of 2017.
Cystic Fibrosis
In September 2015, we initiated a Phase 2 study in JBT-101 in patients suffering from CF that was completed in December 2016.
The study was a multi-center U.S. and European trial. The lead Principal Investigator in the U.S. was Dr. James Chmiel of the Rainbow
Babies & Children’s Hospital, Cleveland, Ohio and the Co-Investigator in Europe was Dr. Stuart Elborn of Queens University Hospital,
Belfast, Ireland. We expect to report top line results by the end of March 2017.
The primary objective of the study is to test safety and tolerability of JBT-101 in adults with CF who had forced expiratory
volume in 1 second (FEV1) percent predicted at least 40%, without regard to their CFTR mutation, infecting pathogen, or baseline
treatment. Secondary objectives are to evaluate changes in pro-inflammatory and pro-resolving lipid mediators as a marker of mechanism
of action of JBT-101 and to evaluate efficacy with FEV1 and Cystic Fibrosis Questionnaire Revised – Respiratory Symptom Score.
Exploratory outcomes include effects of JBT-101 on biomarkers of inflammation and the sputum microbiome. Eighty-five subjects on
stable standard-of-care medications were dosed with study product at 21 CF centers in the U.S. and Europe and treated with study product
daily for a period of 84 days, with a follow-up period of 28 days.
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Dermatomyositis
In June 2015, we initiated a Phase 2 double-blind placebo-controlled clinical study evaluating JBT-101 in patients with skin-
predominant dermatomyositis. The study is a U.S. single center trial and is being funded by a NIH grant. The Principal Investigator is Dr.
Victoria Werth of the University of Pennsylvania, Philadelphia, PA. We expect to complete the study in the third quarter of 2017.
Patients in the dermatomyositis study have active skin involvement, relatively inactive muscle disease, and are permitted to remain
on their baseline mediations. JBT-101 is being evaluated at two dosing levels - 20 mg once a day and 20 mg twice a day - versus placebo.
The trial design has a 28 day screening period, followed by 84 days treatment period and a 28 day safety follow-up. The primary goals of
this trial are to evaluate safety and efficacy of JBT-101 in 22 patients with skin-predominant dermatomyositis. Patients are being monitored
for safety and tolerability throughout the study. Safety is being assessed with physical examinations, adverse effects, laboratory safety
testing, electrocardiograms and testing of psychoactivity. Efficacy is being assessed using the Cutaneous Dermatomyositis Disease Area
and Severity Index. Secondary endpoint measurements include JBT-101 concentrations, other patient-reported outcomes, biomarkers of
inflammation and disease activity in the blood and skin, and metabolipidomic profiles.
In November 2016 we commenced a one-year, open-label extension study to the ongoing Phase 2 clinical study. The application to
conduct a one year open-label extension was submitted to and reviewed by the FDA. The objective of the open-label extension study is to
provide all subjects (treatment and placebo) with the option of receiving JBT-101 for a period of one year after they complete the Phase 2
study. The same safety and efficacy endpoints evaluated in the Phase 2 study will continue to be assessed throughout the one year
extension study.
Systemic Lupus Erythematosus
We plan to commence a Phase 2 double blind placebo-controlled study evaluating JBT-101 in the treatment of SLE in the second
quarter of 2017. The study is being funded by an NIH Grant to the Feinstein Institute for Medical Research and will test the efficacy,
safety, tolerability and biologic effects of JBT-101 as a novel, non-immunosuppressive oral treatment to improve signs and symptoms of
SLE. The study plans to enroll about 100 adult SLE patients with active musculoskeletal disease and will be carried out at approximately 10
sites in the United States with 75 patients in the treatment group and 25 patients in the placebo group. The patients will receive either
placebo or three different doses (5 mg a day, 20 mg a day or 20 mg twice a day) of JBT-101 daily for 84 days with 28 days follow-up. The
primary endpoints are the safety and tolerability of JBT-101 and the evaluation of inflammatory joint pain.
Prior Human Safety Data
Two Phase 1 and one Phase 2 clinical trials have been conducted by prior licensees Atlantic Pharmaceuticals and Indevus
Pharmaceuticals, Inc. who were developing JBT-101 as an analgesia therapy (pain relief) rather than an anti-inflammatory therapy. Based
on their review of the preclinical and clinical data in pain relief, Indevus Pharmaceuticals elected not to continue with further clinical trials
and its license rights were terminated in December 2008. Upon termination, the rights reverted back to Dr. Sumner Burstein who then
assigned the rights to us in April 2009.
The first Phase 1 study evaluated the safety, tolerability and pharmacokinetics of a single oral dose of JBT-101 in healthy adult
male subjects over a dose range from 1 mg to 10 mg, in 4 dose groups of 8 subjects each. JBT-101 had satisfactory oral bioavailability, was
well tolerated, and exhibited linear pharmacokinetics over the dose range tested. There were no life-threatening or serious adverse events in
this study. Two subjects in the 3 mg group and one subject in the 6 mg group out of a total of twenty-four subjects reported adverse events
of mild to moderate intensity. None of the remaining subjects in the 3 mg and 6 mg groups and no subjects in the 1 mg, 10 mg and placebo
groups experienced any adverse events. The two subjects in the 3 mg group reported blurred vision, difficulty in remembering, mild
euphoria, impression of slower movements, dry mouth and difficulty in concentrating. The one subject in the 6 mg group reported
orthostatic vagal fainting, feeling dizzy, and nausea immediately after the first blood draw, which occurred prior to the administration of the
drug.
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The second Phase 1 study evaluated the safety, tolerability and pharmacokinetics of single and multiple ascending doses of JBT-
101 at higher doses. Each dose level had 8 subjects randomized at a ratio of 6 receiving JBT-101 and 2 receiving placebo. Doses ranged
from 10 to 240 mg single dose, and 20, 40 and 80 mg three times a day, or tid, for 7 days multi-dose. JBT-101 showed good tolerability at
single doses up to 120 mg and multiple doses up to 40 mg three times a day for 7 days. For the single dose treatment stage, the most
frequently occurring treatment–emergent adverse events, or TEAEs, occurring in > 10% of all subjects treated with JBT-101, in decreasing
order of frequency, were: dizziness (15 out of 48), nausea (10 out of 48), vomiting (7 out of 48), pallor (6 out of 48), dry mouth (5 out of
48), headache (5 out of 48), somnolence (5 out of 48), tremor (5 out of 48), and disorientation (5 out of 48). All TEAEs were mild to
moderate and the majority of these events occurred in subjects treated at dose levels of 120 mg and above. For the multiple dose stage,
there were no TEAEs at 20 mg tid while in the 40 and 80 mg tid dose groups the most frequently occurring TEAEs, occurring in > 10% of
all subjects treated with JBT-101, were at the 40 and 80 mg tid dose respectively: dizziness (4 out of 6 and 3 out of 6), nausea (2 out of 6
and 1 out of 6), somnolence (2 out of 6 and 1 out of 6), dry mouth (1 out of 6 and 1 out of 6), fatigue (0 out of 6 and 2 out of 6), feeling
abnormal (0 out of 6 and 2 out of 6), anorexia (0 out of 6 and 2 out of 6), inappropriate affect (0 out of 6 and 2 out of 6), and orthostatic
hypotension (0 out of 6 and 2 out of 6). The majority of these events were mild and occurred in subjects treated at the highest dose level of
approximately 80 mg three times a day, for 7 days (i.e., subjects treated with 80 mg tid were reduced to 60 mg tid starting with the second
dose on Day 2 continuing through Day 7). In some elderly patients over 65 years of age, changes in electrocardiogram readings were noted;
however no differences in readings were observed between subjects treated with JBT-101 versus and placebo thus this was not deemed to
be of clinical relevance. JBT-101 exhibited linear pharmacokinetics over the dose range tested.
A Phase 2 crossover design study was conducted for JBT-101 in refractory neuropathic pain patients, or NP. In this study, 21 NP
patients were treated with either JBT-101 or placebo at a dose of 20 and 40 mg twice a day (8 hours apart) for 1 week followed by a 1 week
washout and a cross over. JBT-101 reduced the 3 hour pain index by up to 28% (p<0.03) in one cohort, and was effective at reducing pain
by >30% in 50% of this same cohort (P<0.03) of refractory pain patients although its analgesic effect wore off between 3-8 hours post-
dose. JBT-101 was well tolerated and there were no safety issues of note in this study.
While JBT-101 showed a clear analgesic signal in this phase 2 study in refractory neuropathic pain patients, we are not relying on
these efficacy results to support our clinical program as this study was designed to measure pain while our future studies will have a
completely different efficacy endpoint.
Safety & Toxicology
To date, JBT-101 has undergone an extensive battery of animal safety and toxicology studies in support of Phase II clinical
development. Unlike other CB2 agonists, JBT-101 exhibits limited blood brain barrier penetration (30%) and negligible CB1 activity in
vivo (12 to1 binding affinity for CB2 vs. CB1) resulting in limited central nervous system side effects. Results of the safety pharmacology
studies in animals assessing the central nervous system, cardiovascular and respiratory systems, renal system and gastrointestinal system
were all negative and support that there should be a significant safety margin at anticipated therapeutics doses of JBT-101 in patients.
Animal toxicological profiles of orally administered JBT-101 in single and multi-dose studies in mice, rats, and dogs, and a battery
of in vitro and in vivo genetic toxicity studies, have been conducted and successfully completed. The results of these studies showed no
major toxicological concerns and an excellent safety margin based on drug exposure levels in animals compared to human exposure. In
support of Phase 3 clinical trials, Good Laboratory Practice (“GLP”) chronic toxicology studies have been completed in rats and dogs.
Based on the results of these studies and prior clinical trials, the safety margin in humans is estimated to be between 8 and 32 times greater
than the doses in our clinical trials.
Pre-clinical Studies Demonstrate Mechanism of Action
In pre-clinical animal studies, with both prophylactic and therapeutic administration, JBT-101 has demonstrated clear efficacy at
resolving inflammation. Inflammation was induced in animal pharmacology models by a variety of stimuli that trigger acute innate immune
responses or mimic established inflammation including arachidonic acid, zymosin, platelet activating factor, IL-1β and TNFα, Freund’s
complete adjuvant, and bleomycin. At the histological level, JBT-101 suppressed inflammation in all of these disease models in which it
was studied. At the cellular level, JBT-101 reduced the numbers of inflammatory cells infiltrating the tissue, especially neutrophils. JBT-
101 also induced non-inflammatory cell-death, or apoptosis, of activated T cells. At the molecular level, JBT-101 stimulated the production
of the SPM, lipoxin A4 and the anti-inflammatory eicosanoid prostaglandin J2, while reducing production of pro-inflammatory eicosanoids
leukotriene B4 and prostaglandin E2. JBT-101 reduced production of pro-inflammatory cytokines IL-6, TNFα, IL-1β, and type 1
interferons.
JBT-101 also demonstrated clear efficacy at stopping fibrotic processes in pre-clinical animal studies, with both prophylactic and
therapeutic administration. Fibrosis was induced in joints by Freund’s complete adjuvant, in skin by bleomycin and TGFβ receptor 1
activation, and in lungs by bleomycin. At the histological level, JBT-101 inhibited the development or progression of fibrosis in each
model, with reduced joint ankylosis, skin thickness, and lung fibrosis. For example, at a low dose of 0.1 mg/kg three days a week, JBT-101
significantly inhibited joint ankylosis (fibrosis) in adjuvant-induced arthritis. At a cellular level, myofibroblast accumulation was reduced
(along with inflammatory cell infiltration). At the molecular level, JBT-101 reduced cellular activation through Erg-1, reduced expression
of the pro-fibrotic growth factors TGF-β and connective tissue growth factor, and reduced production of hydroxyproline and collagen.
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Effect of JBT-101 in a Human Model of Inflammatory Resolution
Dr. Gilroy, Professor of Experimental Inflammation and Pharmacology at University College of London has been evaluating the effects of
JBT-101 in a clinical research model of inflammation and its resolution in healthy volunteers. In this model, inflammation was triggered in
healthy individuals by the subcutaneous injection of heat-killed E. coli. Blood flow to the site of inflammation was measured with laser
Doppler techniques. Suction blisters were generated over the site of inflammation, and cells and inflammatory mediators were measured in
the blister fluid at different times after the injection of E. coli. Subjects received either JBT-101 or placebo prior to the procedure. The first
set of data was collected on 15 subjects (5 on placebo, 5 on 5 mg JBT-101 twice a day and 5 on 20 mg JBT-101 twice a day). The top dose
of JBT-101 in this study is the same as the top dose in the Phase 2 clinical trials currently underway and recently completed with JBT-101
in cystic fibrosis, systemic sclerosis and dermatomyositis, respectively.
The data demonstrated that both doses of JBT-101 exerted potent anti-inflammatory effects by inhibiting neutrophil infiltration, and
increased the clearance of bacteria as measured by local endotoxin levels, both key determinants of inflammation severity. JBT-101
correspondingly decreased micro-vascular blood flow around the site of inflammation at 4 hours post stimulus. These two phenomena are
related to a decrease in the inflammatory activation phase of this model. The investigators also found that JBT-101 progressively increased
micro-vascular flow around the site of inflammation during the early phases of resolution (10-24 hours post stimulus), an event believed to
drive an efficient acute inflammatory response and signal its timely resolution. These results are consistent with previous findings from
experiments that tested JBT-101’s effects in animal models of inflammation and support JBT-101’s potential to deliver therapeutic benefit
in chronic inflammatory diseases as a first-in-class “pro-resolution” drug.
Figure 5: Reduction of Neutrophils and Endotoxin in Human Model of Inflammatory Resolution
Effect of JBT-101 in CF Mouse Model
Since patients with CF have chronic inflammation and their lungs are chronically colonized with bacteria, pre-clinical studies were
conducted by Case Western Reserve University to evaluate JBT-101’s overall impact on Pseudomonas aeruginosa colonization.
Pseudomonas aeruginosa infection in the lungs was initiated in wildtype mice (control group) and CFTR-deficient mice in which the CFTR
gene was knocked out. Each group then received treatments with JBT-101 at escalating doses 24 hours post infection over a ten day period.
At day 10, animals were euthanized and evaluated for bacteria load (colony forming units or “CFUs”), total and differential
bronchoalveolar lavage (BAL) white blood cell counts (WBCs). The results of the study demonstrated that in the CFTR-deficient mice
group, JBT-101 improved survival, decreased weight loss, reduced the numbers of neutrophils and white blood cells in the lung and
improved the ability of animals to resolve pulmonary infection as assessed by lung bacterial CFUs, compared to control treatment (see
Figures 4 & 5 below).
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Figure 5: JBT-101 resolves lung inflammation in Pseudomonas Aeruginosa infected mouse model
Figure 5: JBT-101 enhances resolution of lung infection in CF mice infected with Pseudomonas aeruginosa
Effect of JBT-101 in Fibrotic Disease Animal Models
The efficacy of JBT-101 was investigated in three mouse models of scleroderma. Oral administration of JBT-101 once-a-day at 1
mg/kg/day inhibited dermal fibrosis in the three models as measured by reductions in dermal thickness, markers of collagen production,
and myofibroblast abundance (cells that produce collagen). Histopathologic evaluation revealed that JBT-101 inhibited the fibrosis of the
skin tissue in each of these models. In addition, oral administration of JBT-101 was effective at inhibiting lung fibrosis in a bleomycin-
induced mouse model of systemic sclerosis (Figure 6). Collectively, we believe this data supports the development of JBT-101 as a potent
and novel anti-fibrotic/anti-inflammatory drug with the potential to treat some of the key manifestations of human scleroderma.
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Figure 6: JBT-101 inhibits skin thickening in bleomycin-induced model of fibrosis in systemic sclerosis
To translate these in vivo effects of JBT-101 on fibrosis in animal models to humans, we determined the anti-fibrotic effects of
JBT-101 on dermal fibroblasts isolated from diffuse cutaneous systemic sclerosis patients. Dermal fibroblasts from systemic sclerosis
patients overproduce extracellular matrix proteins like collagen and the pro-fibrotic cytokine TGF- β. Further supporting its anti-fibrotic
activity, JBT-101 inhibited new collagen synthesis, reduced TGF-β and increased anti-inflammatory (resolving) eicosanoid levels in this
ex-vivo model of human systemic sclerosis. These responses were statistically significant with a p<0.001 as determined using the Student-
Newmen Keuls post-hoc test for multiple comparisons. TGF-β has been identified in published scientific literature to be an important
cytokine in promoting inflammation and fibrosis in multiple diseases and conditions including systemic sclerosis and cystic fibrosis. While
no pre-clinical model is entirely predictive of clinical efficacy, the results from these pre-clinical studies provide a credible rationale for
further clinical development.
Competition
For autoimmune disorders such as systemic sclerosis, dermatomyositis and SLE, physicians treat patients with a number of drugs
including potent immunosuppressants and cytotoxics to try to reduce the autoimmune response characteristic of the disease. These drugs
have not proven to be very effective thus there remains a high unmet need for safe and effective drugs to treat these autoimmune disorders.
Several companies, including Roche, Boehringer Ingelheim, Bristol Myers, Sanofi, Promedior and Digna Biotech, are actively working to
develop new drugs for treating the inflammation and/or fibrosis in systemic sclerosis. To the best of our knowledge, JBT-101 offers a
unique mode of action to treat systemic sclerosis being one of the few oral drugs with the potential to resolve inflammation and halt fibrosis
without immunosuppression.
There are numerous drug therapies currently used to treat CF patients, targeting different aspects of this complex disease. Inhaled
and oral antibiotics address the pulmonary microbial infection. Mucolytics address the accumulation of mucus in the lungs.
Bronchodilators and hydration agents are also used to help improve pulmonary function. Targeting of the inflammatory component of the
disease is currently done by high dose Ibuprofen and oral corticosteroids. While these offer some clinical benefit, they are not used
chronically due to their adverse side effects which include immunosuppression and metabolic changes (steroids) as well as the risk of
gastrointestinal bleeding (ibuprofen). Thus, there is a clear and urgent unmet medical need for safe and effective inflammation-targeting
drugs for the chronic treatment of CF that could potentially have a beneficial impact on morbidity and mortality.
An emerging area of CF therapy is the development of correctors and potentiators of CFTR. In January 2012, Vertex launched
Kalydeco™, or ivacaftor, the first ever cystic fibrosis drug specifically targeting the underlying genetic defect in the CFTR ion channel.
Kalydeco is a small synthetic oral molecule that helps potentiate the function of the G551D mutant CFTR protein, resulting in improved
forced expiratory volume in one second (a measure of obstruction of airflow in the lungs) by approximately 10% in cystic fibrosis patients.
With at least 2,000 different known mutations in the CFTR gene, Kalydeco can be used by approximately 4,000 patients (ages 2 and above)
who suffer from a limited number of “gating mutations,” including the G551D mutation.
A new combination drug from Vertex, Orkambi™ (lumacaftor/ivacaftor) combination treatment targets a larger population of
homozygote ΔF508 CFTR mutation patients. Orkambi was approved by the FDA on July 2, 2015. In clinical studies, the lung function of
patients taking Orkambi improved by a range of 2.6 percentage points to 3 percentage points, compared with that of patients receiving
placebo. Orkambi™ can be used by approximately 25,000 adult patients carrying two copies of the delta508 CFTR mutation.
15
Several other companies are developing drugs for CF targeting CFTR either as a protein or mRNA transcript. These are
highlighted in the table below:
Selected CF Products in Development
Company
Drug
PTC Therapeutics
UK CF Gene Therapy Consortium
Novartis
Ataluren
pGM169/GL67A Gene therapy
QBW251
Potentiator
Mechanism
Ribosome read thru
(nonsense mutations)
Delivery
Oral
Inhaled
Oral
Bayer
Riociguat
stimulates sGC enzyme
Oral
Nivalis Therapeutics (formerly N30)
Flatley Discovery Lab
N91115
FDL169
GSNOR inhibitor
Corrector
IV and Oral
Oral
Galapagos / AbbVie
ProQR
Celtaxsys
Proteostasis
Research and Development
GLPG1837 /
ABBV-974
Potentiator
Oral
QR-010
RNA oligonucleotide
Inhaled
Acebilustat
PTI130
Anti-inflammatory-inhibits
production of LTB4
CFTR amplifier
Oral
Oral
Mutation
Class 1,
nonsense
All
Gating
Stage
Phase 3
Phase 2b
Phase 2
F508del
homozygous Phase 2
Phase 2
F508del
(failed)
homozygous
Phase 1
F508del
Gating
Phase 1
F508del
homozygous Phase 2
N/A
All
Phase 2
Phase 2
We incurred expenses of approximately $15,437,000 and $5,889,000 for research and development activities for the years ended
December 31, 2016 and 2015, respectively. These expenses include cash and non-cash expenses relating to the development of our clinical
and pre-clinical programs for JBT-101.
Intellectual Property
We have filed patent applications directed to JBT-101, compositions and methods for treating disease using JBT-101. If granted,
the resulting patents would expire on dates ranging from 2030 to 2034, subject to extension under certain circumstances. The patent
application filings are directed to:
● Compositions including an improved ultrapure version of JBT-101 and uses of the compositions for the treatment of fibrotic
conditions and inflammatory conditions;
● The use of JBT-101 in the treatment of fibrotic diseases; and
● JBT-101 formulations and uses of the formulations for the treatment of disease.
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We do not have any issued patents for JBT-101 for the treatment of cystic fibrosis, systemic sclerosis, dermatomyositis, SLE or
any other indications for which we are currently seeking commercial approval.
JBT-101 has been granted Orphan Drug Designation for both cystic fibrosis and systemic sclerosis in the U.S. and in the European
Union. We will be seeking orphan drug status for JBT-101in dermatomyositis and possibly other orphan inflammatory diseases from the
U.S. Food and Drug Administration and in Europe. Orphan drug status provides seven years of market exclusivity in the U.S. and ten years
in Europe beginning on the date of drug approval.
Our commercial success depends in part on our ability to obtain and maintain patent and other proprietary protection for JBT-101
and to operate without infringing the proprietary right of others and to prevent others from infringing our proprietary rights. We strive to
protect our intellectual property through a combination of patents and trademarks as well as through the confidentiality provisions in our
contracts. With respect to JBT-101, we endeavor to obtain and maintain patent protection in the U.S. and internationally on all patentable
aspects of the drug. We cannot be sure that the patents will be granted with respect to any patent applications we may own or license in the
future, nor can we be sure that any patents issued or licensed to us in the future will be useful in protecting our technology. For this and
more comprehensive risks related to our intellectual property, please see “Risk Factors—Risks Relating to Our Intellectual Property
Rights.”
In addition to patent protection, we rely on trade secrets and know-how to develop and maintain our competitive position. For
example, aspects of our proprietary technology platform are based on unpatented trade secrets and know-how related to the manufacturing
of JBT-101. Trade secrets and know-how can be difficult to protect. We seek to protect our proprietary technology and processes, in part,
by confidentiality agreements and invention assignment agreements with our employees, consultants, scientific advisors, contractors and
commercial partners. These agreements are designed to protect our proprietary information and, in the case of the invention assignment
agreements, to grant us ownership of technologies that are developed through a relationship with a third party. 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 contractors 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.
We also plan to seek trademark protection in the U.S. and outside of the U.S. where available and when appropriate. We intend to
use these registered marks in connection with our pharmaceutical research and development as well as our product candidates.
Manufacturing and Supply for JBT-101
We have developed and validated a good manufacturing practice, or GMP, process to manufacture JBT-101 active pharmaceutical
ingredient (“API”) and drug product through our contract manufacturers. The existing API contract manufacturer has produced multi-Kg
scale bulk batches of the under GMP for our Phase 2 clinical studies and we have engaged a second contract manufacturer to produce
sufficient active ingredient for all the clinical studies required prior to submitting an NDA filing with the FDA. We do not own or operate
manufacturing facilities for the production of JBT-101. We expect to depend on third-party suppliers and manufacturing organizations for
all of our clinical trial quantities of raw materials and drug substance. JBT-101 is a synthetic molecule and there are readily available
supplies of all raw materials needed for the manufacture of JBT-101.
Regulatory Matters
Government Regulation
Any product development activities related to JBT-101 or products or assets that we may develop or acquire in the future will be
subject to extensive regulation by various government authorities, including the FDA and other federal, state and local statutes and
regulations and comparable regulatory authorities in other countries, which regulate the design, research, clinical and non-clinical
development, testing, manufacturing, storage, distribution, import, export, labeling, advertising and marketing of pharmaceutical products
and devices. Generally, before a new drug can be sold, considerable data demonstrating its quality, safety and efficacy must be obtained,
organized into a format specific to each regulatory authority, submitted for review and approved by the regulatory authority. The data are
often generated in two distinct development states: pre-clinical and clinical. JBT-101 or other products that we may develop or acquire in
the future must be approved by the FDA through the IND process before they may be legally marketed in the United States. For new
chemical entities, the pre-clinical development stage generally involves synthesizing the active component, developing the formulation and
determining the manufacturing process, as well as carrying out non-human toxicology, pharmacology and drug metabolism studies which
support subsequent clinical testing.
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The clinical stage of development can generally be divided into three sequential phases that may overlap, Phase 1, Phase 2 and
Phase 3 clinical trials. In Phase 1, generally, small numbers of healthy volunteers are initially exposed to single escalating doses and then
multiple escalating doses of the product candidate. The primary purpose of these studies is to assess the metabolism, pharmacologic action,
side effect tolerability and safety of the drug. Phase 2 trials typically involve studies in disease-affected patients to determine the dose
required to produce the desired benefits, while Phase 2b trials are designed to determine efficacy. At the same time, safety and further
pharmacokinetic and pharmacodynamic information is collected. In some instances, formal Phase 1 and Phase 2 trials may not be deemed
necessary or required by the FDA. Such is often the case when the safety and efficacy of an active ingredient is considered to be well
understood by the FDA. Under established regulatory frameworks, pharmaceutical products with active ingredients equal or similar to those
known by the FDA often enter more streamlined development programs than compounds entirely new to the agency.
Post-approval studies, sometimes referred to as Phase 4 clinical trials, may be conducted after initial marketing approval.
Sometimes, these studies are used to gain additional experience from the treatment of patients in the intended therapeutic condition, then
often referred to as Phase 4 clinical trials. In certain instances, the FDA may mandate the performance of Phase 4 studies. In other
situations, post-approval studies aim to gain additional indications for a medication.
Development of Drugs in the United States
In the United States, the process of obtaining regulatory approvals and the subsequent compliance with appropriate federal, state,
local and foreign statutes and regulations require the expenditure of substantial time and financial resources. Failure to comply with the
applicable United States requirements at any time during the product development process, approval process or after approval, may subject
an applicant to administrative or judicial sanctions. These sanctions could include the FDA’s refusal to approve pending applications,
withdrawal of an approval, a clinical hold, warning letters, product recalls or withdrawals from the market, product seizures, total or partial
suspension of production or distribution, injunctions, fines, refusals of government contracts, restitution, disgorgement, or civil or criminal
penalties. Any agency or judicial enforcement action could have a material adverse effect on us.
Prior to the start of human clinical studies for a new drug in the United States, pre-clinical laboratory and animal tests are often
performed under the FDA’s Good Laboratory Practices regulations. The sponsor must submit the result of the pre-clinical tests, together
with manufacturing information, analytical data and any available clinical data or literature and a proposed clinical protocol to the FDA as
part of an IND application, which is a request for authorization from the FDA to administer an investigational drug or biological product to
humans. Similar filings are required in other countries. The amount of data that must be supplied in the IND depends on the phase of the
study. Phase 1 studies typically require less data than larger Phase 2 and 3 studies. A clinical plan must be submitted to the FDA prior to
commencement of a clinical trial. If the FDA has concerns about the clinical plan or the safety of the proposed studies, they may suspend
or terminate the study at any time. Studies must be conducted in accordance with good clinical practice and regulator reporting of study
progress and any adverse experiences is required. Studies are also subject to review by independent institutional review boards responsible
for overseeing studies at particular sites and protecting human research study subjects. An independent institutional review board may also
suspend or terminate a study once initiated. 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 could cause the trial to be suspended or terminated.
Review and Approval in the United States
Following pivotal or Phase 3 trial completion, data are analyzed to determine safety and efficacy. Data are then filed with the FDA
in a New Drug Application, or an NDA, along with proposed labeling for the product and information about the manufacturing and testing
processes and facilities that will be used to ensure product quality. In the United States, FDA approval of an NDA must be obtained before
marketing a pharmaceutical product. The NDA must contain proof of safety, purity, potency and efficacy, which entails extensive pre-
clinical and clinical testing.
The FDA will likely re-analyze the clinical trial data, which could result in extensive discussions between the FDA and us during
the review process. The review and evaluation of applications by the FDA is extensive and time consuming and may take several years to
complete. The FDA may conduct a pre-approval inspection of the manufacturing facilities for the new product to determine whether they
comply with current good manufacturing practice requirements and may also audit data from clinical and pre-clinical trials.
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There is no assurance that the FDA will act favorably or quickly in making such reviews and significant difficulties or costs may
be encountered in our efforts to obtain FDA approvals. The FDA may require that certain contraindications, warnings or precautions be
including in the product labeling, or may condition the approval of the NDA on other changes to the proposed labeling, development of
adequate controls and specifications, or a commitment to conduct post-marketing testing or clinical trials and surveillance programs to
monitor the safety of approved products that have been commercialized. Further, the FDA may place conditions on approvals including the
requirement for a risk evaluation and mitigation strategy, or REMS, to assure the safe use of the drug. If the FDA concludes a REMS is
needed, the sponsor of the NDA must submit a proposed REMS; the FDA will not approve the NDA without an approved REMS, if
required. A REMS 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. Any of these limitations on approval or marketing could restrict
the commercial promotion, distribution, prescription or dispensing of products. Product approvals maybe withdrawn for non-compliance
with regulatory standards or if problems occur, following the initial marketing of the product.
Orphan Drug Designation
Under the Orphan Drug Act, the FDA may grant orphan drug designation to a drug intended to treat a rare disease or condition,
which is generally a disease or condition that affects fewer than 200,000 individuals in the United States and for which there is no
reasonable expectation that the cost of developing and making a drug available in the Unites States for this type of disease or condition will
be recovered from sales of the product. Orphan product designation must be requested before submitting an NDA. After the FDA grants
orphan drug 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 regulatory review and approval process. In addition to the
potential period of exclusivity, orphan designation makes a company eligible for grant funding of up to $400,000 per year for four years to
defray costs of clinical trial expenses, tax credits for clinical research expenses and potential exemption from the FDA application user fee.
If a product 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 drug exclusivity, which means the FDA may not approve any other applications to
market the same drug for the same indication for seven years, except in limited circumstances, such as (i) the drug’s orphan designation is
revoked; (ii) its marketing approval is withdrawn; (iii) the orphan exclusivity holder consents to the approval of another applicant’s
product; (iv) the orphan exclusivity holder is unable to assure the availability of a sufficient quantity of drug; or (v) a showing of clinical
superiority to the product with orphan exclusivity by a competitor product. If a drug designated as an orphan product receives marketing
approval for an indication broader than what is designated, it may not be entitled to orphan drug exclusivity. There can be no assurance that
we will receive orphan drug designation for JBT-101 for dermatomyositis, or other orphan inflammatory diseases.
Drug Development in Europe
In the European Union, our future products may also be subject to extensive regulatory requirements. Similar to the United States,
the marketing of medicinal products has been subject to the granting of marketing authorizations by regulatory agencies. Particular
emphasis is also being placed on more sophisticated and faster procedures for reporting of adverse events to the competent authorities.
As in the United States, the various phases of pre-clinical and clinical research in the European Union are subject to significant
regulatory controls. Although the regulatory controls on clinical research are currently undergoing a harmonization process following the
adoption of the Clinical Trials Directive 2001/20/EC, there are currently significant variations in the member state regimes. All member
states, however, currently require independent institutional review board approval of interventional clinical trials. Except for the United
Kingdom Phase 1 studies in health volunteers, all clinical trials require either prior governmental notification or approval. Most regulators
also require the submission of adverse event reports during a study and a copy of the final study report.
Review and Approval in the European Union
In the European Union, approval of new medicinal products can be obtained through one of three processes: the mutual
recognition procedure, the centralized procedure and the decentralized procedure. We intend to determine which process we will follow, if
any, in the future.
Mutual Recognition Procedure : An applicant submits an application in one European Union member state, known as the reference
member state. Once the reference member state has granted the marketing authorization, the applicant may choose to submit applications in
other concerned member states, requesting them to mutually recognize the marketing authorizations already granted. Under this mutual
recognition process, authorities in other concerned member states have 55 days to raise objections, which must then be resolved by
discussion among the concerned member states, the reference member state and the applicant within 90 days of the commencement of the
mutual recognition procedure. If any disagreement remains, all considerations by authorities in the concerned member states are suspended
and the disagreement is resolved through an arbitration process. The mutual recognition procedure results in separate national marketing
authorizations in the reference member state.
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Centralized Procedure: This procedure is currently mandatory for products developed by means of a biotechnological process and
optional for new active substances and other “innovative medicinal products with novel characteristics.” Under this procedure, an
application is submitted to the European Agency for the Evaluation of Medical Products. Two European Union member states are
appointed to conduct an initial evaluation of each application. These countries each prepare an assessment report that is then used as the
basis of a scientific opinion of the Committee on Proprietary Medical Products. If this opinion is favorable, it is sent to the European
Commission, which drafts a decision. After consulting with the member states, the European Commission adopts a decision and grants a
marketing authorization, which is valid throughout the European Union and confers the same rights and obligations in each of the member
states as a marketing authorization granted by that member state.
Decentralized Procedure: The most recently introduced of the three processes for obtaining approval of new medicinal processes
in the European Union, the decentralized procedure is similar to the mutual recognition procedure described above, but with differences in
the timing that key documents are provided to concerned member states by the reference member state, the overall timing of the procedure
and the possibility of, among other things, “clock stops” during the procedure.
Post-Marketing Requirements
Following approval of a new product, a pharmaceutical company and the approved product are subject to continuing regulation by
the FDA and other federal and state regulatory authorities, including, among other things, monitoring and recordkeeping activities,
reporting to applicable regulatory authorities of adverse experiences with the product, providing the regulatory authorities with updated
safety and efficacy information, product sampling and distribution requirements, and complying with promotion and advertising
requirements, which include, among others, standards for direct-to-consumer advertising, restrictions on promoting drugs for uses or in
patient populations not described in the drug’s approved labeling (known as “off-label use”), limitations on industry-sponsored scientific
and educational activities, and requirements for promotional activities involving the internet. Although physicians may prescribe legally
available drugs for off-label uses, manufacturers may not market or promote such off-label uses. Modifications or enhancements to the
products or labeling or changes of site of manufacture are often subject to the approval of the FDA and other regulators, which may or may
not be received or may result in a lengthy review process.
Prescription drug advertising is subject to federal, state and foreign regulations. In the United States, the FDA regulates
prescription drug promotion, including direct-to-consumer advertising. Prescription drug promotion materials must be submitted to the FDA
in conjunction with their first use. Any distribution of prescription drug products and pharmaceutical samples must comply with the U.S.
Prescription Drug Marketing Act, a part of the U.S. Federal Food, Drug and Cosmetic Act. Once a product is approved, its manufacture is
subject to comprehensive and continuing regulations by the FDA. The FDA regulations require the products be manufactured in specific
approved facilities and in accordance with current good manufacturing practices, and NDA holders must list their products and register their
manufacturing establishments with the FDA. These regulations also impose certain organizational, procedural and documentation
requirements with respect to manufacturing and quality assurance activities. Drug manufacturers and other entities involved in the
manufacture and distribution of approved drugs are subject to periodic unannounced inspections by the FDA and certain state agencies for
compliance with current good manufacturing practice and other laws. NDA holders using contract manufacturers, laboratories or packagers
are responsible for the selection and monitoring of qualified firms. These firms are subject to inspections by the FDA at any time, and the
discovery of violative conditions could result in enforcement actions that interrupt the operation of any such facilities or the ability to
distribute products manufactured, processed or tested by them.
Special Protocol Assessment
The Federal Food, Drug and Cosmetic Act directs the FDA to meet with sponsors, pursuant to a sponsor’s written request, for the
purpose of reaching agreement on the design and size of clinical trials intended to form the primary basis of an efficacy claim in an NDA. If
an agreement is reached, the FDA will reduce the agreement to writing and make it part of the administrative record. This agreement is
called a special protocol assessment, or SPA. While the FDA’s guidance on SPAs states that documented SPAs should be considered
binding on the review division, the FDA has latitude to change its assessment if certain exceptions apply. Exceptions include public health
concerns emerging that were unrecognized at the time of the protocol assessment, identification of a substantial scientific issue essential to
the safety or efficacy testing that later comes to light, a sponsor’s failure to follow the protocol agreed upon, or the FDA’s reliance on data,
assumptions or information that are determined to be wrong.
Other Regulatory Matters
Manufacturing, sales, promotion and other activities following product approval are also subject to regulation by numerous
regulatory authorities in addition to the FDA, including, in the United States, the Centers for Medicare & Medicaid Services, or CMS, other
divisions of the Department of Health and Human Services, the Drug Enforcement Administration, the Consumer Product Safety
Commission, the Federal Trade Commission, the Occupational Safety & Health Administration, the Environmental Protection Agency, and
state and local governments. Sales, marketing and scientific/educational programs must also comply with federal and state fraud and abuse
laws. Pricing and rebate programs must comply with the Medicaid rebate requirements of the U.S. Omnibus Budget Reconciliation Act of
1990. If products are made available to authorized users of the Federal Supply Schedule of the General Services Administration, additional
laws and requirements apply. The handling of any controlled substances must comply with the U.S. Controlled Substances Act and
Controlled Substances Import and Export Act. Products must meet applicable child-resistant packaging requirements under the U.S. Poison
Prevention Packaging Act. Manufacturing, sales, promotion and other activities are also potentially subject to federal and state consumer
protection and unfair completion laws.
20
In the United States, our product candidate, JBT-101, is currently classified as Schedule I controlled substance as defined in the
Controlled Substance Act (“CSA”). This designation is based on JBT-101’s chemical structure and pharmacology (namely, it being a
synthetic endocannabinoid mimetic that binds to the CB2 receptor). Even though JBT-101’s mechanism of action is to modulate the
immune system and results to date from clinical studies have demonstrated the drug has no psychotropic effects (which we believe is unlike
other members of its chemical class), the DEA classifies JBT-101 as a Schedule I substance.
Schedule I controlled substances are pharmaceutical products subject to specific regulations under the CSA, that establishes,
among other things, certain registration, manufacturing quotas, security, recordkeeping, reporting, import, export and other requirements
administered by the DEA. All parties responsible for the manufacturing, distribution and testing the drug in clinical studies must apply for
and obtain a license from the DEA before they are permitted to perform these activities with JBT-101. Furthermore, these parties must have
the security, control, recordkeeping, reporting and inventory mechanisms required by the DEA to prevent drug loss and diversion. All
licensed facilities are required to renew their registrations annually if they intend to continue to work with our drug. The DEA conducts
periodic inspections of certain registered establishments that handle controlled substances. We have been working with our manufacturers,
distributors, exporters and clinical sites to obtain the necessary licenses to work with JBT-101. The parties responsible for the
manufacturing, distribution and export of JBT-101 have already applied for and have been granted DEA licenses and a number of
institutions responsible for conducting our Phase 2 clinical studies have also been granted DEA licenses
Individual states have also established controlled substance laws and regulations. Though state-controlled substances laws often
mirror federal law, because the states are separate jurisdictions, they may separately schedule drugs, as well. While some states
automatically schedule a drug based on federal action, other states schedule drugs through rulemaking or a legislative action. The
requirement for state registrations could also result in delay of the manufacturing, distribution of JBT-101 or in the completion of the Phase
2 clinical studies. We and our manufacturing vendors and clinical sites must also obtain separate state registrations, permits or licenses in
order to be able to obtain, handle, and distribute controlled substances for clinical trials or commercial sale, and failure to meet applicable
regulatory requirements could lead to enforcement and sanctions by the states in addition to those from the DEA or otherwise arising under
federal law.
The distribution of pharmaceutical products is subject to additional requirements and regulations, including extensive record-
keeping, licensing, storage and security requirements intended to prevent the unauthorized sale of pharmaceutical products.
Third-Party Payer Coverage and Reimbursement
Significant uncertainty exists as to the coverage and reimbursement status of any of our drug candidates that ultimately may obtain
regulatory approval. In both the United States and foreign markets, our ability to commercialize our product candidates successfully, and to
attract commercialization partners for our product candidates, depends in significant part on the availability of adequate financial coverage
and reimbursement from third-party payers, including, in the United States, governmental payers such as the Medicare and Medicaid
programs, managed care organizations, and private health insurers. Medicare is a federally funded program managed by the CMS, through
local fiscal intermediaries and carriers that administer coverage and reimbursement for certain healthcare items and services furnished to
the elderly and disabled. Medicaid is an insurance program for certain categories of patients whose income and assets fall below state
defined levels and who are otherwise uninsured that is both federally and state funded and managed by each state. The federal government
sets general guidelines for Medicaid and each state creates specific regulations that govern its individual program. Each payer has its own
process and standards for determining whether it will cover and reimburse a procedure or particular product. Private payers often rely on
the lead of the governmental payers in rendering coverage and reimbursement determinations. Therefore, achieving favorable CMS
coverage and reimbursement is usually a significant gating issue for successful introduction of a new product. The competitive position of
some of our products will depend, in part, upon the extent of coverage and adequate reimbursement for such products and for the
procedures in which such products are used. Prices at which we or our customers seek reimbursement for our product candidates can be
subject to challenge, reduction or denial by the government and other payers.
21
The United States Congress and state legislatures may, from time to time, propose and adopt initiatives aimed at cost containment,
which could impact our ability to sell our product candidates profitably. For example, in March 2010, President Obama signed into law the
Patient Protection and Affordable Care Act and the associated reconciliation bill, which we refer to collectively as the Health Care Reform
Law, a sweeping law intended to broaden access to health insurance, reduce or constrain the growth of healthcare spending, enhance
remedies against fraud and abuse, add new transparency requirements for healthcare and health insurance industries, impose new taxes and
fees on the health industry and impose additional health policy reforms. Effective October 1, 2010, the Health Care Reform Law revised
the definition of “average manufacturer price” for reporting purposes, which could increase the amount of Medicaid drug rebates to states
once the provision is effective. Further, the law imposes a significant annual fee on companies that manufacture or import branded
prescription drug products. Substantial new provisions affecting compliance have also been enacted, which may require us to modify our
business practices with healthcare practitioners. We will not know the full effects of the Health Care Reform Law until applicable federal
and state agencies issue regulations or guidance under the new law. Although it is too early to determine the effect of the Health Care
Reform Law, the new law appears likely to continue the pressure on pharmaceutical pricing, especially under the Medicare program, and
may also increase our regulatory burdens and operating costs. Moreover, in the coming years, additional changes could be made to
governmental healthcare programs that could significantly impact the success of our product candidates.
The cost of pharmaceuticals continues to generate substantial governmental and third-party payer interest. We expect that the
pharmaceutical industry will experience pricing pressures due to the trend toward managed healthcare, the increasing influence of managed
care organizations and additional legislative proposals. Our results of operations could be adversely affected by current and future
healthcare reforms.
Some third-party payers also require pre-approval of coverage for new or innovative devices or drug therapies before they will
reimburse healthcare providers that use such therapies. While we cannot predict whether any proposed cost-containment measures will be
adopted or otherwise implemented in the future, the announcement or adoption of these proposals could have a material adverse effect on
our ability to obtain adequate prices for our product candidates and operate profitably.
In addition, in some foreign countries, the proposed pricing for a drug must be approved before it may be lawfully marketed. The
requirements governing drug pricing vary widely from country to country. For example, the European Union provides options for its
member states to restrict the range of medicinal products for which their national health insurance systems provide reimbursement and to
control the prices of medicinal products for human use. A member state may approve a specific price for the medicinal product or it may
instead adopt a system of direct or indirect controls on the profitability of the company placing the medicinal product on the market. There
can be no assurance that any country that has price controls or reimbursement limitations for pharmaceutical products will allow favorable
reimbursement and pricing arrangements for any of our products. Historically, products launched in the European Union do not follow price
structures of the United States and generally tend to be significantly lower.
Other Healthcare Laws and Compliance Requirements
In the United States, our activities are potentially subject to regulation by various federal, state and local authorities in addition to
the FDA, including the CMS, other divisions of the United States Department of Health and Human Services (e.g., the Office of Inspector
General), the United States Department of Justice and individual United States Attorney offices within the Department of Justice, and state
and local governments. These regulations include:
● the federal healthcare program anti-kickback law which prohibits, among other things, persons from soliciting, receiving or
providing remuneration, directly or indirectly, to induce either the referral of an individual, for an item or service or the
purchasing or ordering of a good or service, for which payment may be made under federal healthcare programs such as the
Medicare and Medicaid programs;
● federal false claims laws which prohibit, among other things, individuals or entities from knowingly presenting, or causing to be
presented, claims for payment from Medicare, Medicaid, or other government reimbursement programs that are false or
fraudulent. The government may assert that a claim including items or services resulting from a violation of the federal
healthcare program anti-kickback law or related to off-label promotion constitutes a false or fraudulent claim for purposes of
the federal false claims laws;
● the federal Health Insurance Portability and Accountability Act of 1996 which prohibits executing a scheme to defraud any
healthcare benefit program or making false statements relating to healthcare matters and which also imposes certain
requirements relating to the privacy, security and transmission of individually identifiable health information;
● the federal transparency requirements under the Health Care Reform Law requires manufacturers of drugs, devices, biologics,
and medical supplies to report to the Department of Health and Human Services information related to physician payments and
other transfers of value and physician ownership and investment interests;
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● t h e Federal Physician Payments Sunshine Act within the ACA, and its implementing regulations, require that certain
manufacturers of drugs, devices, biological and medical supplies for which payment is available under Medicare, Medicaid or
the Children’s Health Insurance Program (with certain exceptions) to report information related to certain payments or other
transfers of value made or distributed to physicians and teaching hospitals, or to entities or individuals at the request of, or
designated on behalf of, the physicians and teaching hospitals and to report annually certain ownership and investment interests
held by physicians and their immediate family members; and
● state law equivalents of each of the above federal laws, such as anti-kickback and false claims laws, which may apply to items
o r services reimbursed by any third party payer, including commercial insurers, 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 often are
not preempted by federal laws, thus complicating compliance efforts.
In addition, we may be subject to data privacy and security regulation by both the federal government and the states in which we
conduct our business. The Health Insurance Portability and Accountability Act, or HIPAA, as amended by the Health Information
Technology for Economic and Clinical Health Act, or HITECH, and its implementing regulations, imposes certain requirements relating to
the privacy, security and transmission of individually identifiable health information. Among other things, HITECH makes HIPAA’s
privacy and security standards directly applicable to “business associates”—independent contractors or agents of covered entities that
receive or obtain protected health information in connection with providing a service on behalf of a covered entity. HITECH also created
four new tiers of civil monetary penalties, amended HIPAA to make civil and criminal penalties directly applicable to business associates
and possibly other persons, and gave state attorneys general new authority to file civil actions for damages or injunctions in federal courts to
enforce the federal HIPAA laws and seek attorneys’ fees and costs associated with pursuing federal civil actions. In addition, state laws
govern 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.
Post-Marketing Regulations
Following approval of a new product, a pharmaceutical company and the approved product are subject to continuing regulation by
the FDA and other federal and state regulatory authorities, including, among other things, monitoring and recordkeeping activities,
reporting to applicable regulatory authorities of adverse experiences with the product, providing the regulatory authorities with updated
safety and efficacy information, product sampling and distribution requirements, and complying with promotion and advertising
requirements, which include, among others, standards for direct-to-consumer advertising, restrictions on promoting drugs for uses or in
patient populations not described in the drug’s approved labeling (known as “off-label use”), limitations on industry-sponsored scientific
and educational activities, and requirements for promotional activities involving the internet. Although physicians may prescribe legally
available drugs for off-label uses, manufacturers may not market or promote such off-label uses. Modifications or enhancements to the
products or labeling or changes of site of manufacture are often subject to the approval of the FDA and other regulators, which may or may
not be received or may result in a lengthy review process.
Employees
We had 25 full time employees at December 31, 2016. All of our employees are engaged in administration, finance, clinical,
manufacturing, regulatory and business development functions. We believe our relations with our employees are good. We anticipate that
the number of employees will grow as we continue to develop our product candidates. In addition, we utilize and will continue to utilize
consultants, clinical research organizations and third parties to perform our pre-clinical studies, clinical studies, manufacturing and
regulatory functions.
Emerging Growth Company
We are an “emerging growth company,” as defined in the Jumpstart Our Business Startups Act of 2012, or the JOBS Act. We will
remain an emerging growth company until the earlier of (1) January 1, 2020, (2) the last day of the first fiscal year in which our annual
gross revenues exceed $1 billion, (3) the date on which we become a “large accelerated filer” as defined in Rule 12b-2 under the Securities
Exchange Act of 1934, as amended, which would occur if the market value of our common stock that is held by non-affiliates exceeds $700
million as of the last business day of our most recently completed second fiscal quarter or (4) the date on which we have issued more than
$1 billion in non-convertible debt during the preceding three-year period.
For as long as we remain an “emerging growth company,” we may take advantage of certain exemptions from various reporting
requirements that are applicable to public companies that are not “emerging growth companies” including, but not limited to, not being
required to comply with the auditor attestation requirements of Section 404 of the Sarbanes-Oxley Act, reduced disclosure obligations
regarding executive compensation and financial statements in our periodic reports and proxy statements, and exemptions from the
requirements of holding a nonbinding advisory vote to approve executive compensation and shareholder approval of any golden parachute
payments not previously approved. We are choosing to “opt out” of the extended transition periods available under the JOBS Act for
complying with new or revised accounting standards, and intend to take advantage of the other reporting exemptions until we are no longer
an “emerging growth company.”
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Corporate Information
Corbus Pharmaceuticals, Inc. (formerly known as JB Therapeutics Inc.), was incorporated on April 24, 2009 under the laws of the
State of Delaware. On April 11, 2014, JB Therapeutics, Inc. completed a merger with Corbus Pharmaceuticals Holdings, Inc. and changed
its name to Corbus Pharmaceuticals, Inc. Upon the consummation of the merger, Corbus Pharmaceuticals, Inc. became a wholly owned
subsidiary of Corbus Pharmaceuticals Holdings, Inc. which continues to operate the business of Corbus Pharmaceuticals, Inc. Our principal
executive offices are located at 100 River Ridge Drive, Norwood, Massachusetts 02062, and our telephone number is (619) 963-0100. Our
website address is www.corbuspharma.com.
We make available free of charge on or through the Investor Relations link on our website, www.corbuspharma.com, access to
press releases and investor presentations, as well as all materials that we file electronically with the SEC, including our annual report on
Form 10-K, quarterly reports on Form 10-Q, current reports on Form 8-K and amendments to those reports, filed or furnished pursuant to
Section 13(a) or 15(d) of the Exchange Act as soon as reasonably practicable after electronically filing such materials with, or furnishing
them to, the SEC. During the period covered by this Form 10-K, we made all such materials available through our website as soon as
reasonably practicable after filing such materials with the SEC. You may also read and copy any materials filed by us with the SEC at the
SEC’s Public Reference Room at 100 F Street, N.E., Washington, D.C. 20549, and you may obtain information on the operation of the
Public Reference Room by calling the SEC in the United States at 1-800-SEC-0330. In addition, the SEC maintains an Internet website,
www.sec.gov, that contains reports, proxy and information statements and other information that we file electronically with the SEC.
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Item 1A. RISK FACTORS
An investment in our common stock is speculative and illiquid and involves a high degree of risk including the risk of a loss of your entire
investment. You should carefully consider the risks and uncertainties described below and the other information contained in this report
and our other reports filed with the Securities and Exchange Commission. The risks set forth below are not the only ones facing us.
Additional risks and uncertainties may exist that could also adversely affect our business, operations and financial condition. If any of the
following risks actually materialize, our business, financial condition and/or operations could suffer. In such event, the value of our
common stock could decline, and you could lose all or a substantial portion of the money that you pay for our common stock.
Risk Related to our Company and our Business
Risks Related to Our Financial Position and Need for Capital
We are a clinical stage pharmaceutical company with a limited operating history.
We are a clinical stage pharmaceutical company with a limited operating history. We must obtain FDA clearance of our
Investigational New Drug applications, or INDs, before clinical trials can commence, and must receive regulatory approval of our New
Drug Applications, or NDAs, before commercial sales of a product can commence. The likelihood of success of our business plan must be
considered in light of the problems, substantial expenses, difficulties, complications and delays frequently encountered in connection with
developing and expanding early-stage businesses and the regulatory and competitive environment in which we operate. Pharmaceutical
product development is a highly speculative undertaking, involves a substantial degree of risk and is a capital-intensive business.
Accordingly, you should consider our prospects in light of the costs, uncertainties, delays and difficulties frequently encountered
by companies in the early stages of development, especially clinical pharmaceutical companies such as ours. Potential investors should
carefully consider the risks and uncertainties that a company with a limited operating history will face. In particular, potential investors
should consider that we cannot assure you that we will be able to:
● receive FDA approval of INDs for commencing our clinical trials;
● successfully implement or execute our current business plan, or that our business plan is sound;
● Successfully manufacture our clinical product and establish commercial drug supply;
● obtain DEA licenses necessary for the manufacturing of JBT-101 and for evaluating JBT-101 in our clinical trials;
● successfully complete clinical trials and obtain regulatory approval for the marketing of JBT-101;
● secure market exclusivity and/or adequate intellectual property protection for JBT-101;
● attract and retain an experienced management and advisory team;
● secure acceptance of JBT-101 in the medical community and with third party payors and consumers;
● launch commercial sales of JBT-101, whether alone or in collaboration with others; and
● raise sufficient funds in the capital markets to effectuate our business plan including clinical development, regulatory approval
and commercialization for JBT-101.
If we cannot successfully execute any one of the foregoing, our business may not succeed and your investment will be adversely
affected.
We have incurred operating losses in each year since our inception and expect to continue to incur substantial losses for the foreseeable
future. We may never become profitable or, if achieved, be able to sustain profitability.
We expect to incur substantial expenses without corresponding revenues unless and until we are able to obtain regulatory approval
and successfully commercialize JBT-101. We have been engaged in developing JBT-101 since 2009. To date, we have not generated any
revenue from JBT-101 and we expect to incur significant expense to complete our clinical program for JBT-101 in the United States and
elsewhere. We may never be able to obtain regulatory approval for the marketing of JBT-101 in any indication in the United States or
internationally. Even if we are able to commercialize JBT-101 or any other product candidate, there can be no assurance that we will
generate significant revenues or ever achieve profitability. Our net losses for the years ended December 31, 2016 and December 31, 2015
were approximately $19,999,000 and $8,851,000, respectively. As of December 31, 2016, we had an accumulated deficit of approximately
$33,276,000.
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If we were to obtain FDA approval for JBT-101, we would expect that our research and development expenses will continue to
increase as we advance to clinical trials for indications for the treatment of cystic fibrosis, systemic sclerosis and dermatomyositis. We may
elect to pursue FDA approval for JBT-101 in other indications, which will result in significant additional research and development
expenses. As a result, we expect to continue to incur substantial losses for the foreseeable future, and these losses will increase. We are
uncertain when or if we will be able to achieve or sustain profitability. If we achieve profitability in the future, we may not be able to
sustain profitability in subsequent periods. Failure to become and remain profitable would impair our ability to sustain operations and
adversely affect the price of our common stock and our ability to raise capital.
Our cash or cash equivalents will only fund our operations for a limited time and we will need to raise additional capital to support our
development and commercialization efforts.
We are currently operating at a loss and expect our operating costs will increase significantly as we incur costs related to the
clinical trials for JBT-101. As of December 31, 2016, our consolidated cash balance was approximately $15.0 million. On February 28,
2017, we entered into a securities purchase agreement providing for the issuance and sale of 3,887,815 shares of our common stock in a
registered direct offering to institutional and accredited investors at a purchase price of $7.00 per share with gross proceeds to us totaling
$27.2 million less estimated issuance costs of approximately $0.1 million (“February 2017 Registered Direct Offering”). We expect our
cash on hand of approximately $15.0 million at December 31, 2016 together with the proceeds from the February 2017 Registered Direct
Offering and the remaining milestone payment of $500,000 from the CFFT, which we expect to receive in the second quarter of 2017, to be
sufficient to meet our operating and capital requirements into the fourth quarter of 2018 based on current planned expenditures.
We do not currently have any arrangements or credit facilities in place as a source of funds, and there can be no assurance that we
will be able to raise sufficient additional capital on acceptable terms, or at all, and if we are not successful in raising additional capital, we
may not be able to continue as a going concern. We may seek additional capital through a combination of private and public equity
offerings, debt financings and strategic collaborations. Debt financing, if obtained, may involve agreements that include covenants limiting
or restricting our ability to take specific actions, such as incurring additional debt, and could increase our expenses and require that our
assets secure such debt.
Equity financing, if obtained, could result in dilution to our then existing stockholders and/or require such stockholders to waive
certain rights and preferences. If such financing is not available on satisfactory terms, or is not available at all, we may be required to delay,
scale back or eliminate the development of business opportunities and our operations and financial condition may be materially adversely
affected. We can provide no assurances that any additional sources of financing will be available to us on favorable terms, if at all. In
addition, if we are unable to secure sufficient capital to fund our operations, we might have to enter into strategic collaborations that could
require us to share commercial rights to JBT-101 with third parties in ways that we currently do not intend or on terms that may not be
favorable to us. If we choose to pursue additional indications and/or geographies for JBT-101 or otherwise expand more rapidly than we
presently anticipate we may also need to raise additional capital sooner than expected.
Risks Related to Product Development, Regulatory Approval, Manufacturing and Commercialization
We depend entirely on the success of JBT-101. If we are unable to generate revenues from JBT-101, our ability to create stockholder
value will be limited.
Our only product candidate currently is JBT-101, which has successfully completed Phase 1 safety studies and is being evaluated
in clinical studies. We do not generate revenues from any FDA approved drug products and have no other product candidates in
development. There is no guarantee that our clinical trials will be successful or that we will continue with clinical studies to support an
approval from the FDA for any indication. We note that most drug candidates never reach the clinical development stage and even those
that do reach clinical development have only a small chance of successfully completing clinical development and gaining regulatory
approval. Therefore, our business currently depends entirely on the successful development, regulatory approval and commercialization of
JBT-101, which may never occur.
If we are not able to obtain any required regulatory approvals for JBT-101, we will not be able to commercialize our only product
candidate and our ability to generate revenue will be limited.
We must successfully complete clinical trials for JBT-101 before we can apply for marketing approval. Even if we complete our
clinical trials, it does not assure FDA approval. Our clinical trials may be unsuccessful, which would materially harm our business. Even if
these clinical trials are successful, we are required to conduct additional clinical trials to establish JBT-101’s safety and efficacy, before a
New Drug Application, or NDA, can be filed with the FDA for marketing approval of JBT-101.
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Clinical testing is expensive, is difficult to design and implement, can take many years to complete and is uncertain as to outcome.
Success in early phases of pre-clinical and clinical trials does not ensure that later clinical trials will be successful, and interim results of a
clinical trial do not necessarily predict final results. A failure of one or more of our clinical trials can occur at any stage of testing. We may
experience numerous unforeseen events during, or as a result of, the clinical trial process that could delay or prevent our ability to receive
regulatory approval or commercialize JBT-101. The research, testing, manufacturing, labeling, packaging, storage, approval, sale,
marketing, advertising and promotion, pricing, export, import and distribution of drug products are subject to extensive regulation by the
FDA and other regulatory authorities in the United States and other countries, which regulations differ from country to country. We are not
permitted to market JBT-101 as a prescription pharmaceutical product in the United States until we receive approval of an NDA from the
FDA, or in any foreign countries until we receive the requisite approval from such countries. In the United States, the FDA generally
requires the completion of clinical trials of each drug to establish its safety and efficacy and extensive pharmaceutical development to
ensure its quality before an NDA is approved. Regulatory authorities in other jurisdictions impose similar requirements. Of the large
number of drugs in development, only a small percentage result in the submission of an NDA to the FDA and even fewer are eventually
approved for commercialization. We have never submitted an NDA to the FDA or comparable applications to other regulatory authorities.
If our development efforts for JBT-101, including regulatory approval, are not successful for its planned indications, or if adequate demand
for JBT-101 is not generated, our business will be harmed.
Our success depends on the receipt of regulatory approval and the issuance of such regulatory approvals is uncertain and subject to
a number of risks, including the following:
● the FDA or comparable foreign regulatory authorities or institutional review boards, or IRBs, may disagree with the design or
implementation of our clinical trials;
● we may not be able to provide acceptable evidence of JBT-101’s safety and efficacy;
● the results of our clinical trials may not be satisfactory or may not meet the level of statistical or clinical significance required
by the FDA, European Medicines Agency, or EMA, or other comparable foreign regulatory authorities for marketing approval;
● the dosing of JBT-101 in a particular clinical trial may not be at an optimal level;
● patients in our clinical trials may suffer adverse effects for reasons that may or may not be related to JBT-101;
● the data collected from clinical trials may not be sufficient to support the submission of an NDA or other submission or to
obtain regulatory approval in the United States or elsewhere;
● the FDA or comparable foreign regulatory authorities may fail to approve the manufacturing processes or facilities of third-
party manufacturers with which we contract for clinical and commercial supplies; and
● the approval policies or regulations of the FDA or comparable foreign regulatory authorities may significantly change in a
manner rendering our clinical data insufficient for approval.
Failure to obtain regulatory approval for JBT-101 for the foregoing or any other reasons will prevent us from commercializing this
product candidate as a prescription product, and our ability to generate revenue will be materially impaired. We cannot guarantee that
regulators will agree with our assessment of the results of the clinical trials we intend to conduct in the future or that such trials will be
successful. The FDA, EMA and other regulators have substantial discretion in the approval process and may refuse to accept any
application or may decide that our data is insufficient for approval and require additional clinical trials, or pre-clinical or other studies. In
addition, varying interpretations of the data obtained from pre-clinical and clinical testing could delay, limit or prevent regulatory approval
of a product candidate.
We are a clinical stage company and we have not submitted an NDA or received regulatory approval to market JBT-101 in any
jurisdiction. We have only limited experience in filing the applications necessary to gain regulatory approvals and expect to rely on
consultants and third party contract research organizations, or CROs, with expertise in this area to assist us in this process. Securing FDA
approval requires the submission of pre-clinical, clinical, and/or pharmacokinetic data, information about product manufacturing processes
and inspection of facilities and supporting information to the FDA for each therapeutic indication to establish a product candidate’s safety
and efficacy for each indication. JBT-101 may prove to have undesirable or unintended side effects, toxicities or other characteristics that
may preclude our obtaining regulatory approval or prevent or limit commercial use with respect to one or all intended indications.
The process of obtaining regulatory approvals is expensive, often takes many years, if approval is obtained at all, and can vary
substantially based upon, among other things, the type, complexity and novelty of the product candidates involved, the jurisdiction in
which regulatory approval is sought and the substantial discretion of the regulatory authorities. Changes in the regulatory approval policy
during the development period, changes in or the enactment of additional statutes or regulations, or changes in regulatory review for a
submitted product application may cause delays in the approval or rejection of an application. Regulatory approval obtained in one
jurisdiction does not necessarily mean that a product candidate will receive regulatory approval in all jurisdictions in which we may seek
approval, but the failure to obtain approval in one jurisdiction may negatively impact our ability to seek approval in a different jurisdiction.
Failure to obtain regulatory marketing approval for JBT-101 in any indication will prevent us from commercializing the product candidate,
and our ability to generate revenue will be materially impaired.
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JBT-101 is our only product candidate in development. If we fail to successfully commercialize JBT-101, we may need to acquire
additional product candidates and our business will be adversely affected.
We have never commercialized any product candidates and do not have any other compounds in pre-clinical testing, lead
optimization or lead identification stages beyond JBT-101. We cannot be certain that JBT-101 will prove to be sufficiently effective and
safe to meet applicable regulatory standards for any indication. If we fail to successfully commercialize JBT-101 as a treatment for cystic
fibrosis, systemic sclerosis, dermatomyositis or any other indication, whether as a stand-alone therapy or in combination with other
treatments, our business would be adversely affected.
Even if we receive regulatory approval for JBT-101, we still may not be able to successfully commercialize this product, and the revenue
that we generate from its sales, if any, may be limited.
If approved for marketing, the commercial success of JBT-101 will depend upon its acceptance by the medical community,
including physicians, patients and health care payors. The degree of market acceptance of JBT-101 will depend on a number of factors,
including:
● demonstration of clinical safety and efficacy;
● relative convenience, pill burden and ease of administration;
● the prevalence and severity of any adverse effects;
● the willingness of physicians to prescribe JBT-101 and of the target patient population to try new therapies;
● efficacy of JBT-101 compared to competing products;
● the introduction of any new products that may in the future become available to treat indications for which JBT-101 may be
approved;
● new procedures or methods of treatment that may reduce the incidences of any of the indications in which JBT-101 may show
utility;
● pricing and cost-effectiveness;
● the inclusion or omission of JBT-101 in applicable treatment guidelines;
● the effectiveness of our or any future collaborators’ sales and marketing strategies;
● limitations or warnings contained in FDA-approved labeling;
● our ability to obtain and maintain sufficient third-party coverage or reimbursement from government health care programs,
including Medicare and Medicaid, private health insurers and other third-party payors; and
● the willingness of patients to pay out-of-pocket in the absence of third-party coverage or reimbursement.
If JBT-101 is approved, but does not achieve an adequate level of acceptance by physicians, health care payors and patients, we
may not generate sufficient revenue and we may not be able to achieve or sustain profitability. Our efforts to educate the medical
community and third-party payors on the benefits of JBT-101 may require significant resources and may never be successful.
In addition, even if we obtain regulatory approvals, the timing or scope of any approvals may prohibit or reduce our ability to
commercialize JBT-101 successfully. For example, if the approval process takes too long, we may miss market opportunities and give
other companies the ability to develop competing products or establish market dominance. Any regulatory approval we ultimately obtain
may be limited or subject to restrictions or post-approval commitments that render JBT-101 not commercially viable. For example,
regulatory authorities may approve JBT-101 for fewer or more limited indications than we request, may not approve the price we intend to
charge for JBT-101, may grant approval contingent on the performance of costly post-marketing clinical trials, or may approve JBT-101
with a label that does not include the labeling claims necessary or desirable for the successful commercialization of that indication. Further,
the FDA or comparable foreign regulatory authorities may place conditions on approvals, such as risk management plans and a Risk
Evaluation and Mitigation Strategy, or REMS, to assure the safe use of the drug. If the FDA concludes a REMS is needed, the sponsor of
the NDA must submit a proposed REMS; the FDA will not approve the NDA without an approved REMS, if required. A REMS 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. The FDA may also require a REMS for an approved product when new safety information
emerges. Any of these limitations on approval or marketing could restrict the commercial promotion, distribution, prescription or
dispensing of JBT-101. Moreover, product approvals may be withdrawn for non-compliance with regulatory standards or if problems occur
following the initial marketing of the product. Any of the foregoing scenarios could materially harm the commercial success of JBT-101.
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Even if we obtain marketing approval for JBT-101, we will be subject to ongoing obligations and continued regulatory review, which
may result in significant additional expense. Additionally, JBT-101 could be subject to labeling and other restrictions and withdrawal
from the market and we may be subject to penalties if we fail to comply with regulatory requirements or if we experience unanticipated
problems with JBT-101.
Even if we obtain United States regulatory approval of JBT-101 for an indication, the FDA may still impose significant
restrictions on its indicated uses or marketing or the conditions of approval, or impose ongoing requirements for potentially costly and
time-consuming post-approval studies, including Phase 4 clinical trials, and post-market surveillance to monitor safety and efficacy. JBT-
101 will also be subject to ongoing regulatory requirements governing the manufacturing, labeling, packaging, storage, distribution, safety
surveillance, advertising, promotion, recordkeeping and reporting of adverse events and other post-market information. These requirements
include registration with the FDA, as well as continued compliance with current Good Clinical Practices regulations, or cGCPs, for any
clinical trials that we conduct post-approval. In addition, manufacturers of drug products and their facilities are subject to continual review
and periodic inspections by the FDA and other regulatory authorities for compliance with current Good Manufacturing Practices, or cGMP,
requirements relating to quality control, quality assurance and corresponding maintenance of records and documents.
The FDA has the authority to require a risk evaluation and mitigation strategy, or REMS, as part of an NDA or after approval,
which may impose further requirements or restrictions on the distribution or use of an approved drug, such as limiting prescribing to certain
physicians or medical centers that have undergone specialized training, limiting treatment to patients who meet certain safe-use criteria or
requiring patient testing, monitoring and/or enrollment in a registry.
With respect to sales and marketing activities by us or any future partner, advertising and promotional materials must comply with
FDA rules in addition to other applicable federal, state and local laws in the United States and similar legal requirements in other countries.
In the United States, the distribution of product samples to physicians must comply with the requirements of the U.S. Prescription Drug
Marketing Act. Application holders must obtain FDA approval for product and manufacturing changes, depending on the nature of the
change. We may also be subject, directly or indirectly through our customers and partners, to various fraud and abuse laws, including,
without limitation, the U.S. Anti-Kickback Statute, U.S. False Claims Act, and similar state laws, which impact, among other things, our
proposed sales, marketing, and scientific/educational grant programs. If we participate in the U.S. Medicaid Drug Rebate Program, the
Federal Supply Schedule of the U.S. Department of Veterans Affairs, or other government drug programs, we will be subject to complex
laws and regulations regarding reporting and payment obligations. All of these activities are also potentially subject to U.S. federal and state
consumer protection and unfair competition laws. Similar requirements exist in many of these areas in other countries.
In addition, if JBT-101 is approved for an indication, our product labeling, advertising and promotion would be subject to
regulatory requirements and continuing regulatory review. The FDA strictly regulates the promotional claims that may be made about
prescription products. In particular, a product may not be promoted for uses that are not approved by the FDA as reflected in the product’s
approved labeling. If we receive marketing approval for JBT-101, physicians may nevertheless legally prescribe our products to their
patients in a manner that is inconsistent with the approved label. If we are found to have promoted such off-label uses, we may become
subject to significant liability and government fines. The FDA and other agencies actively enforce the laws and regulations prohibiting the
promotion of off-label uses by a company, and any company that is found to have improperly promoted off-label uses may be subject to
significant sanctions. The federal government has levied large civil and criminal fines against companies for alleged improper promotion
and has enjoined several companies from engaging in off-label promotion. The FDA has also requested that companies enter into consent
decrees of permanent injunctions under which specified promotional conduct is changed or curtailed.
If we or a regulatory agency discover previously unknown problems with a product, such as adverse events of unanticipated
severity or frequency, problems with the facility where the product is manufactured, or if we or our manufacturers fail to comply with
applicable regulatory requirements, we may be subject to the following administrative or judicial sanctions:
●
restrictions on the marketing or manufacturing of the product, withdrawal of the product from the market, or voluntary or
mandatory product recalls;
● issuance of warning letters or untitled letters;
29
● clinical holds;
● injunctions or the imposition of civil or criminal penalties or monetary fines;
● suspension of any ongoing clinical trials;
● refusal to approve pending applications or supplements to approved applications filed by us, or suspension or revocation of
product license approvals;
● suspension or imposition of restrictions on operations, including costly new manufacturing requirements; or
● product seizure or detention or refusal to permit the import or export of product.
The occurrence of any event or penalty described above may inhibit our ability to commercialize JBT-101 and generate revenue.
Adverse regulatory action, whether pre-or post-approval, can also potentially lead to product liability claims and increase our product
liability exposure.
We currently have no sales and marketing organization. If we are unable to secure a sales and marketing partner or establish
satisfactory sales and marketing capabilities, we may not successfully commercialize JBT-101.
At present, we have no sales or marketing personnel. In order to commercialize products that are approved for commercial sales,
we must either collaborate with third parties that have such commercial infrastructure or develop our own sales and marketing
infrastructure. If we are not successful entering into appropriate collaboration arrangements, or recruiting sales and marketing personnel or
in building a sales and marketing infrastructure, we will have difficulty successfully commercializing JBT-101, which would adversely
affect our business, operating results and financial condition.
We may not be able to enter into collaboration agreements on terms acceptable to us or at all. In addition, even if we enter into
such relationships, we may have limited or no control over the sales, marketing and distribution activities of these third parties. Our future
revenues may depend heavily on the success of the efforts of these third parties. If we elect to establish a sales and marketing infrastructure
we may not realize a positive return on this investment. In addition, we will have to compete with established and well-funded
pharmaceutical and biotechnology companies to recruit, hire, train and retain sales and marketing personnel. Factors that may inhibit our
efforts to commercialize JBT-101 without strategic partners or licensees include:
● our inability to recruit and retain adequate numbers of effective sales and marketing personnel;
● the inability of sales personnel to obtain access to or persuade adequate numbers of physicians to prescribe JBT-101;
● the lack of complementary products to be offered by sales personnel, which may put us at a competitive disadvantage relative
to companies with more extensive product lines; and
● unforeseen costs and expenses associated with creating an independent sales and marketing organization.
We face competition from other biotechnology and pharmaceutical companies and our operating results will suffer if we fail to compete
effectively.
The biotechnology and pharmaceutical industries are intensely competitive and subject to rapid and significant technological
change. We have competitors in a number of jurisdictions, many of which have substantially greater name recognition, commercial
infrastructures and financial, technical and personnel resources than we have. Established competitors may invest heavily to quickly
discover and develop novel compounds that could make JBT-101 obsolete or uneconomical. Any new product that competes with an
approved product may need to demonstrate compelling advantages in efficacy, cost, convenience, tolerability and safety to be
commercially successful. Other competitive factors, including generic competition, could force us to lower prices or could result in reduced
sales. In addition, new products developed by others could emerge as competitors to JBT-101. If we are not able to compete effectively
against our current and future competitors, our business will not grow and our financial condition and operations will suffer.
Recently enacted and future legislation may increase the difficulty and cost for us to obtain marketing approval of and commercialize
JBT-101 and affect the prices we may obtain.
In the United States and some foreign jurisdictions, there have been a number of legislative and regulatory changes and proposed
changes regarding the healthcare system that could prevent or delay marketing approval for JBT-101, restrict or regulate post-approval
activities and affect our ability to profitably sell JBT-101. Legislative and regulatory proposals have been made to expand post-approval
requirements and restrict sales and promotional activities for pharmaceutical products. We do not know whether additional legislative
changes will be enacted, or whether the FDA regulations, guidance or interpretations will be changed, or what the impact of such changes
on the marketing approvals of JBT-101, if any, may be. In addition, increased scrutiny by the U.S. Congress of the FDA’s approval process
may significantly delay or prevent marketing approval, as well as subject us to more stringent product labeling and post-marketing testing
and other requirements.
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In the United States, the Medicare Modernization Act, or MMA, changed the way Medicare covers and pays for pharmaceutical
products. The legislation expanded Medicare coverage for drug purchases by the elderly and introduced a new reimbursement methodology
based on average sales prices for drugs. In addition, this legislation authorized Medicare Part D prescription drug plans to use formularies
where they can limit the number of drugs that will be covered in any therapeutic class. As a result of this legislation and the expansion of
federal coverage of drug products, we expect that there will be additional pressure to contain and reduce costs. These cost reduction
initiatives and other provisions of this legislation could decrease the coverage and price that we receive for JBT-101 and could seriously
harm our business. While the MMA applies only to drug benefits for Medicare beneficiaries, private payors often follow Medicare coverage
policy and payment limitations in setting their own reimbursement rates, and any reduction in reimbursement that results from the MMA
may result in a similar reduction in payments from private payors.
In March 2010, President Obama signed into law the Patient Protection and Affordable Care Act, as amended by the Health Care
and Education Affordability Reconciliation Act of 2010 or, collectively, the Health Care Reform Law, a sweeping law intended to broaden
access to health insurance, reduce or constrain the growth of healthcare spending, enhance remedies against fraud and abuse, add new
transparency requirements for healthcare and health insurance industries, impose new taxes and fees on the health industry and impose
additional health policy reforms. Effective October 1, 2010, the Health Care Reform Law revised the definition of “average manufacturer
price” for reporting purposes, which could increase the amount of Medicaid drug rebates to states. Further, the new law imposed a
significant annual fee on companies that manufacture or import branded prescription drug products. Substantial new provisions affecting
compliance have also been enacted, which may require us to modify our business practices with healthcare practitioners, and incur
substantial costs to ensure compliance. As a result of the 2016 presidential election, U.S. lawmakers have made statements about potentially
repealing or replacing the Affordable Care Act, although no such specific legislation has yet been introduced. While we are unable to
predict what legislation, if any, may potentially be enacted, to the extent that future changes affect how our product candidates could be
paid for and/or reimbursed by the government and private payers, our business could be adversely affected.
Despite initiatives to invalidate the Health Care Reform Law, at this time it appears the implementation of the Health Care Reform
Law will continue. We will not know the full effects of the Health Care Reform Law until applicable federal and state agencies issue
regulations or guidance under the new law. Although it is too early to determine the effect of the Health Care Reform Law, the new law
appears likely to continue the pressure on pharmaceutical pricing, especially under the Medicare program, and may also increase our
regulatory burdens and operating costs.
In addition, other legislative changes have been proposed and adopted in the United States since the Health Care Reform Law 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 to Medicare payments to providers of up to 2% per fiscal year, starting in 2013. On January 2,
2013, President Obama signed into law the American Taxpayer Relief Act of 2012, or the ATRA, which delayed for another two months
the budget cuts mandated by these sequestration provisions of the Budget Control Act of 2011. The ATRA, among other things, also
reduced Medicare payments to several providers, including hospitals, imaging centers and cancer treatment centers, and increased the
statute of limitations period for the government to recover overpayments to providers from three to five years. We expect that additional
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, and in turn could significantly reduce the projected value of certain development projects and
reduce our profitability.
Our future growth depends, in part, on our ability to penetrate foreign markets, where we would be subject to additional regulatory
burdens and other risks and uncertainties.
Our future profitability will depend, in part, on our ability to commercialize JBT-101 in foreign markets for which we intend to
rely on collaborations with third parties. If we commercialize JBT-101 in foreign markets, we would be subject to additional risks and
uncertainties, including:
● our customers’ ability to obtain reimbursement for JBT-101 in foreign markets;
● our inability to directly control commercial activities because we are relying on third parties;
● the burden of complying with complex and changing foreign regulatory, tax, accounting and legal requirements;
● different medical practices and customs in foreign countries affecting acceptance in the marketplace;
● import or export licensing requirements;
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● longer accounts receivable collection times;
● longer lead times for shipping;
● language barriers for technical training;
● reduced protection of intellectual property rights in some foreign countries;
● foreign currency exchange rate fluctuations; and
● the interpretation of contractual provisions governed by foreign laws in the event of a contract dispute.
Foreign sales of JBT-101 could also be adversely affected by the imposition of governmental controls, political and economic
instability, trade restrictions and changes in tariffs, any of which may adversely affect our results of operations.
If we market JBT-101 in a manner that violates healthcare fraud and abuse laws, or if we violate government price reporting laws, we
may be subject to civil or criminal penalties.
The FDA enforces laws and regulations which require that the promotion of pharmaceutical products be consistent with the
approved prescribing information. While physicians may prescribe an approved product for a so-called “off label” use, it is unlawful for a
pharmaceutical company to promote its products in a manner that is inconsistent with its approved label and any company which engages in
such conduct may be subject to significant liability. Similarly, industry codes in the European Union and other foreign jurisdictions prohibit
companies from engaging in off-label promotion and regulatory agencies in various countries enforce violations of the code with civil
penalties. While we intend to ensure that our promotional materials are consistent with our label, regulatory agencies may disagree with our
assessment and may issue untitled letters, warning letters or may institute other civil or criminal enforcement proceedings. In addition to
FDA restrictions on marketing of pharmaceutical products, several other types of state and federal healthcare fraud and abuse laws have
been applied in recent years to restrict certain marketing practices in the pharmaceutical industry. These laws include the U.S. Anti-
Kickback Statute, U.S. False Claims Act and similar state laws. Because of the breadth of these laws and the narrowness of the safe
harbors, it is possible that some of our business activities could be subject to challenge under one or more of these laws.
The U.S. Anti-Kickback Statute prohibits, among other things, knowingly and willfully offering, paying, soliciting or receiving
remuneration to induce, or in return for, purchasing, leasing, ordering or arranging for the purchase, lease or order of any healthcare item or
service reimbursable under Medicare, Medicaid or other federally financed healthcare programs. This statute has been interpreted broadly
to apply to arrangements between pharmaceutical manufacturers on the one hand and prescribers, purchasers and formulary managers on
the other. Although there are several statutory exemptions and regulatory safe harbors protecting certain common activities from
prosecution, the exemptions and safe harbors are drawn narrowly, and practices that involve remuneration intended to induce prescribing,
purchasing or recommending may be subject to scrutiny if they do not qualify for an exemption or safe harbor. Our practices may not, in all
cases, meet all of the criteria for safe harbor protection from anti-kickback liability. Moreover, recent health care reform legislation has
strengthened these laws. For example, the Health Care Reform Law, among other things, amends the intent requirement of the U.S. Anti-
Kickback Statute and criminal health care fraud statutes; a person or entity no longer needs to have actual knowledge of this statute or
specific intent to violate it. In addition, the Health Care Reform Law provides that the government may assert that a claim including items
or services resulting from a violation of the U.S. Anti-Kickback Statute constitutes a false or fraudulent claim for purposes of the U.S. False
Claims Act. Federal false claims laws prohibit any person from knowingly presenting, or causing to be presented, a false claim for payment
to the federal government or knowingly making, or causing to be made, a false statement to get a false claim paid.
Over the past few years, pharmaceutical and other healthcare companies have been prosecuted under these laws for a variety of
alleged promotional and marketing activities, such as: allegedly providing free trips, free goods, sham consulting fees and grants and other
monetary benefits to prescribers; reporting to pricing services inflated average wholesale prices that were then used by federal programs to
set reimbursement rates; engaging in off-label promotion that caused claims to be submitted to Medicare or Medicaid for non-covered, off-
label uses; and submitting inflated best price information to the Medicaid Rebate Program to reduce liability for Medicaid rebates. Most
states also have statutes or regulations similar to the U.S. Anti-Kickback Statute and the U.S. False Claims Act, which apply to items and
services reimbursed under Medicaid and other state programs, or, in several states, apply regardless of the payor. Sanctions under these
federal and state laws may include substantial civil monetary penalties, exclusion of a manufacturer’s products from reimbursement under
government programs, substantial criminal fines and imprisonment.
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We are, and will be, completely dependent on third parties to manufacture JBT-101, and our commercialization of JBT-101 could be
halted, delayed or made less profitable if those third parties fail to obtain manufacturing approval from the FDA or comparable foreign
regulatory authorities, fail to provide us with sufficient quantities of JBT-101 or fail to do so at acceptable quality levels or prices.
We do not currently have, nor do we plan to acquire, the capability or infrastructure to manufacture the active pharmaceutical
ingredient, or the finished JBT-101 drug product in tablet form, for use in our clinical trials or for commercial product, if any. As a result,
we will be obligated to rely on contract manufacturers, if and when JBT-101 is approved for commercialization.
The facilities used by our contract manufacturers to manufacture JBT-101 must be approved by the FDA pursuant to inspections
that will be conducted after we submit our NDA to the FDA. We do not control the manufacturing process of, and are completely
dependent on, our contract manufacturing partners for compliance with cGMPs for manufacture of both active drug substances and finished
drug products. These cGMP regulations cover all aspects of the manufacturing, testing, quality control and record keeping relating to JBT-
101. If our contract manufacturers cannot successfully manufacture material that conforms to our specifications and the strict regulatory
requirements of the FDA or others, they will not be able to secure and/or maintain regulatory approval for their manufacturing facilities. If
the FDA or a comparable foreign regulatory authority does not approve these facilities for the manufacture of JBT-101 or if it withdraws
any such approval in the future, we may need to find alternative manufacturing facilities, which would significantly impact our ability to
develop, obtain regulatory approval for or market JBT-101, if approved.
Our contract manufacturers will be subject to ongoing periodic unannounced inspections by the FDA and corresponding state and
foreign agencies for compliance with cGMPs and similar regulatory requirements. We will not have control over our contract
manufacturers’ compliance with these regulations and standards. Failure by any of our contract manufacturers to comply with applicable
regulations could result in sanctions being imposed on us, including fines, injunctions, civil penalties, failure to grant approval to market
JBT-101, delays, suspensions or withdrawals of approvals, operating restrictions and criminal prosecutions, any of which could
significantly and adversely affect our business. In addition, we will not have control over the ability of our contract manufacturers to
maintain adequate quality control, quality assurance and qualified personnel. Failure by our contract manufacturers to comply with or
maintain any of these standards could adversely affect our ability to develop, obtain regulatory approval for or market JBT-101.
If for any reason, these third parties are unable or unwilling to perform, we may not be able to terminate our agreements with
them, and we may not be able to locate alternative manufacturers or formulators or enter into favorable agreements with them and we
cannot be certain that any such third parties will have the manufacturing capacity to meet future requirements. If these manufacturers or
any alternate manufacturer of finished drug product experiences any significant difficulties in its respective manufacturing processes for
our API or finished JBT-101 product or should cease doing business with us, we could experience significant interruptions in the supply of
JBT-101 or may not be able to create a supply of JBT-101 at all. Were we to encounter manufacturing issues, our ability to produce a
sufficient supply of JBT-101 might be negatively affected. Our inability to coordinate the efforts of our third party manufacturing partners,
or the lack of capacity available at our third party manufacturing partners, could impair our ability to supply JBT-101 at required levels.
Because of the significant regulatory requirements that we would need to satisfy in order to qualify a new bulk or finished product
manufacturer, if we face these or other difficulties with our current manufacturing partners, we could experience significant interruptions in
the supply of JBT-101 if we decided to transfer the manufacture of JBT-101 to one or more alternative manufacturers in an effort to deal
with the difficulties.
Any manufacturing problem or the loss of a contract manufacturer could be disruptive to our operations and result in lost sales.
Additionally, we rely on third parties to supply the raw materials needed to manufacture our potential products. Any reliance on suppliers
may involve several risks, including a potential inability to obtain critical materials and reduced control over production costs, delivery
schedules, reliability and quality. Any unanticipated disruption to a future contract manufacturer caused by problems at suppliers could
delay shipment of JBT-101, increase our cost of goods sold and result in lost sales.
We cannot guarantee that our manufacturing and supply partners will be able to reduce the costs of commercial scale
manufacturing of JBT-101 over time. If the commercial-scale manufacturing costs of JBT-101 are higher than expected, these costs may
significantly impact our operating results. In order to reduce costs, we may need to develop and implement process improvements.
However, in order to do so, we will need, from time to time, to notify or make submissions with regulatory authorities, and the
improvements may be subject to approval by such regulatory authorities. We cannot be sure that we will receive these necessary approvals
or that these approvals will be granted in a timely fashion. We also cannot guarantee that we will be able to enhance and optimize output in
our commercial manufacturing process. If we cannot enhance and optimize output, we may not be able to reduce our costs over time.
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Our product candidate JBT-101, is currently classified as a Schedule I controlled substance subject to U.S. controlled substance laws
and regulations, including regulations of the Drug Enforcement Agency and the U.S. Food and DrugAdministration. Failure to obtain
the necessary licenses and registrations and failure to comply with these laws could result in the delay in the manufacturing and
distribution of JBT-101 and could delay the completion of clinical studies. Such delays and the cost of compliance with these laws and
regulations, could adversely affect our business operations and our financial condition.
In the United States, our product candidate, JBT-101, is currently classified as Schedule I controlled substance as defined in the
Controller Substance Act (“CSA”). This designation is based on JBT-101’s chemical structure and pharmacology (namely, it being a
synthetic endocannabinoid mimetic that binds to the CB2 receptor). Even though JBT-101’s mechanism of action is to modulate the
immune system and results to date from clinical studies have demonstrated the drug has no psychotropic effects (which we believe is unlike
other members of its chemical class), the DEA classifies JBT-101 as a Schedule I substance.
Schedule I controlled substances are pharmaceutical products subject to specific regulations under the CSA, that establishes,
among other things, certain registration, manufacturing quotas, security, recordkeeping, reporting, import, export and other requirements
administered by the DEA. All parties responsible for the manufacturing, distribution and testing the drug in clinical studies must apply for
and obtain a license from the DEA before they are permitted to perform these activities with JBT-101. Furthermore, these parties must have
the security, control, recordkeeping, reporting and inventory mechanisms required by the DEA to prevent drug loss and diversion. All
licensed facilities are required to renew their registrations annually if they intend to continue to work with our drug. The DEA conducts
periodic inspections of certain registered establishments that handle controlled substances. We have been working with our manufacturers,
distributors, exporters and clinical sites to obtain the necessary licenses to work with JBT-101. The parties responsible for the
manufacturing, distribution and export of JBT-101 have already applied for and have been granted DEA licenses and a number of
institutions responsible for conducting our Phase 2 clinical studies have also been granted DEA licenses. However the failure to maintain
the necessary registrations and the delay or failure of additional clinical sites to obtain DEA registrations, could delay the manufacturing,
distribution and export of JBT-101 and could delay the completion of the Phase 2 clinical studies. Furthermore, failure to maintain
compliance with the CSA, particularly non-compliance resulting in loss or diversion, could result in regulatory action that could have a
material adverse effect on our business, financial condition and results of operations. The DEA may seek civil penalties, refuse to renew
necessary registrations, or initiate proceedings to restrict, suspend or revoke those registrations. In certain circumstances, violations could
lead to criminal proceedings. In addition, if the FDA, DEA, or any foreign regulatory authority determines that JBT-101 may have potential
for abuse, it may require us to generate more clinical or other data than we currently anticipate to establish whether or to what extent the
substance has an abuse potential, which could increase the cost and/or delay the launch of JBT-101.
Individual states have also established controlled substance laws and regulations. Though state-controlled substances laws often
mirror federal law, because the states are separate jurisdictions, they may separately schedule drugs, as well. While some states
automatically schedule a drug based on federal action, other states schedule drugs through rulemaking or a legislative action. The
requirement for state registrations could also result in delay of the manufacturing, distribution of JBT-101 or in the completion of the Phase
2 clinical studies. We and our manufacturing vendors and clinical sites must also obtain separate state registrations, permits or licenses in
order to be able to obtain, handle, and distribute controlled substances for clinical trials or commercial sale, and failure to meet applicable
regulatory requirements could lead to enforcement and sanctions by the states in addition to those from the DEA or otherwise arising under
federal law.
The manufacturing and distribution of JBT-101 is subject to the DEA’s annual manufacturing and procurement quota
requirements. The annual quota allocated to us or our contract manufacturers for the controlled substances in JBT-101 may not be
sufficient to complete clinical trials. Consequently, any delay or refusal by the DEA in establishing our, or our contract manufacturers,
procurement and/or production quota for controlled substances could delay or stop our clinical trials or product launches, which could have
a material adverse effect on our business, financial position and operations.
Delays in shipping JBT-101 could have a material adverse effect on our business, results of operations and financial condition.
The import and export of JBT-101 requires import and export licenses. However, because JBT-101 is currently a Schedule I
controlled substance in the United States, in addition to the FDA and U.S. Customs and Border Protection, its import and export is also
regulated by the DEA. We may not be granted, or if granted, maintain, such licenses for import or export from the authorities these
regulatory agencies. Even if we obtain the relevant licenses, shipments of JBT-101 may be held up in transit by any of these authorities,
which could cause significant delays and may lead to product batches which no longer meet specifications for use in clinical trials or
commercial distribution. Such events could result in delayed development timelines, increased expenses and partial or total loss of revenue
from JBT-101.
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We expect that we will rely on third parties to assist the Company in conducting clinical trials for JBT-101. If these third parties do not
successfully carry out their contractual duties or meet expected deadlines, we may not be able to obtain regulatory approval for or
commercialize JBT-101 and our business would be substantially harmed.
We expect to enter into agreements with third-party CROs to assist us in conducting and managing our clinical programs including
contracting with clinical sites to perform our clinical studies. We plan to rely on these parties for execution of clinical studies for JBT-101
and we will control only certain aspects of conducting the clinical studies. Nevertheless, we will be responsible for ensuring that each of
our studies is conducted in accordance with the applicable protocol, legal, regulatory and scientific standards, and our reliance on CROs and
clinical sites will not relieve us of our regulatory responsibilities. We and our CROs will be required to comply with cGCPs, which are
regulations and guidelines enforced by the FDA, the Competent Authorities of the Member States of the European Economic Area and
comparable foreign regulatory authorities for any products in clinical development. The FDA enforces these cGCP regulations through
periodic inspections of trial sponsors, principal investigators and trial sites. If we or our CROs fail to comply with applicable cGCPs, the
clinical data generated in our clinical trials may be deemed unreliable and the FDA or comparable foreign regulatory authorities may
require us to perform additional clinical trials before approving our marketing applications. We cannot assure you that, upon inspection, the
FDA will determine that any of our clinical trials comply with cGCPs. In addition, our clinical trials must be conducted with products
produced under cGMP regulations and will require a large number of test subjects. Our failure or the failure of our CROs or clinical sites to
comply with these regulations may require us to repeat clinical trials, which would delay the regulatory approval process and could also
subject us to enforcement action up to and including civil and criminal penalties.
Although we intend to design the clinical trials for JBT-101 in consultation with CROs, we expect that the CROs will manage all
of the clinical trials conducted at contracted clinical sites. As a result, many important aspects of our drug development programs would be
outside of our direct control. In addition, the CROs and clinical sites may not perform all of their obligations under arrangements with us or
in compliance with regulatory requirements. If the CROs or clinical sites do not perform clinical trials in a satisfactory manner, breach their
obligations to us or fail to comply with regulatory requirements, the development and commercialization of JBT-101 for the subject
indication may be delayed or our development program materially and irreversibly harmed. We cannot control the amount and timing of
resources these CROs and clinical sites will devote to our program or JBT-101. If we are unable to rely on clinical data collected by our
CROs, we could be required to repeat, extend the duration of, or increase the size of our clinical trials, which could significantly delay
commercialization and require significantly greater expenditures.
If any of our relationships with these third-party CROs or clinical sites terminate, we may not be able to enter into arrangements
with alternative CROs or clinical sites. If CROs do not successfully carry out their contractual duties or obligations or meet expected
deadlines, if they need to be replaced or if the quality or accuracy of the clinical data they obtain is compromised due to the failure to
adhere to our clinical protocols, regulatory requirements or for other reasons, any such clinical trials may be extended, delayed or
terminated, and we may not be able to obtain regulatory approval for or successfully commercialize JBT-101. As a result, our financial
results and the commercial prospects for JBT-101 would be harmed, our costs could increase and our ability to generate revenue could be
delayed.
Any termination or suspension of or delays in the commencement or completion of any necessary studies of JBT-101 for any
indications could result in increased costs to us, delay or limit our ability to generate revenue and adversely affect our commercial
prospects.
The commencement and completion of clinical studies can be delayed for a number of reasons, including delays related to:
● the FDA failing to grant permission to proceed and placing the clinical study on hold;
● subjects failing to enroll or remain in our trials at the rate we expect;
● a facility manufacturing JBT-101 being ordered by the FDA or other government or regulatory authorities to temporarily or
permanently shut down due to violations of cGMP requirements or other applicable requirements, or cross-contaminations of
product in the manufacturing process;
● any changes to our manufacturing process that may be necessary or desired;
● subjects choosing an alternative treatment for the indications for which we are developing JBT-101, or participating in
competing clinical studies;
● subjects experiencing severe or unexpected drug-related adverse effects;
● reports of similar technologies and products raising safety and/or efficacy concerns;
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● third-party clinical investigators losing their license or permits necessary to perform our clinical trials, not performing our
clinical trials on our anticipated schedule or employing methods consistent with the clinical trial protocol, cGCP requirements,
or other third parties not performing data collection and analysis in a timely or accurate manner;
● inspections of clinical study sites by the FDA or IRBs finding regulatory violations that require us to undertake corrective
action, result in suspension or termination of one or more sites or the imposition of a clinical hold on the entire study, or that
prohibit us from using some or all of the data in support of our marketing applications;
● third-party contractors becoming debarred or suspended or otherwise penalized by the FDA or other government or regulatory
authorities for violations of regulatory requirements, in which case we may need to find a substitute contractor, and we may not
be able to use some or any of the data produced by such contractors in support of our marketing applications;
● one or more IRBs refusing to approve, suspending or terminating the study at an investigational site precluding enrollment of
additional subjects, or withdrawing its approval of the trial; 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;
● deviations of the clinical sites from trial protocols or dropping out of a trial;
● adding new clinical trial sites;
● the inability of the CRO to execute any clinical trials for any reason; and
● government or regulatory delays or “clinical holds” requiring suspension or termination of a trial.
Product development costs for JBT-101 will increase if we have delays in testing or approval or if we need to perform more or
larger clinical studies than planned. Additionally, changes in regulatory requirements and policies may occur and we may need to amend
study protocols to reflect these changes. Amendments may require us to resubmit our study protocols to the FDA and IRBs for
reexamination, which may impact the costs, timing or successful completion of that study. If we experience delays in completion of, or if
we, the FDA or other regulatory authorities, the IRB, or other reviewing entities, or any of our clinical study sites suspend or terminate any
of our clinical studies of JBT-101, its commercial prospects may be materially harmed and our ability to generate product revenues will be
delayed. Any delays in completing our clinical trials will increase our costs, slow down our development and approval process and
jeopardize our ability to commence product sales and generate revenues. Any of these occurrences may harm our business, financial
condition and prospects significantly. In addition, many of the factors that cause, or lead to, termination or suspension of, or a delay in the
commencement or completion of, clinical studies may also ultimately lead to the denial of regulatory approval of JBT-101. In addition, if
one or more clinical studies are delayed, our competitors may be able to bring products to market before we do, and the commercial
viability of JBT-101 could be significantly reduced.
Clinical drug development involves a lengthy and expensive process with an uncertain outcome, and results of earlier studies and trials
may not be predictive of future trial results.
Clinical testing is expensive and can take many years to complete, and its outcome is inherently uncertain. Failure can occur at any
time during the clinical trial process. The results of pre-clinical studies and early clinical trials may not be predictive of the results of later-
stage clinical trials. We cannot assure you that the FDA will view the results as we do or that any future trials of JBT-101 will achieve
positive results. Product candidates in later stages of clinical trials may fail to show the desired safety and efficacy traits despite having
progressed through pre-clinical studies and initial clinical trials. A number of companies in the pharmaceutical industry have suffered
significant setbacks in advanced clinical trials due to lack of efficacy or adverse safety profiles, notwithstanding promising results in earlier
trials. Any future clinical trial results for JBT-101 may not be successful.
In addition, a number of factors could contribute to a lack of favorable safety and efficacy results for JBT-101. For example, such
trials could result in increased variability due to varying site characteristics, such as local standards of care, differences in evaluation period
and surgical technique, and due to varying patient characteristics including demographic factors and health status.
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We may not be able to obtain or maintain orphan drug designation or exclusivity for our product candidates.
We have been granted orphan drug designation in the United States and in the European Union for JBT-101 for the treatment of
cystic fibrosis and systemic sclerosis. We also intend to seek orphan drug status for JBT-101 for the treatment of dermatomyositis. Upon
receipt of regulatory approval, orphan drug status will provide us with seven years of market exclusivity in the United States under the
Orphan Drug Act. However, there is no guarantee that the FDA will grant orphan drug designation for JBT-101 for dermatomyositis or any
other indication, which would make us ineligible for the additional exclusivity and other benefits of orphan drug designation. Moreover,
there can be no assurance that another company also holding orphan drug designation for the same indication or which may receive orphan
drug designation in the future will not receive approval prior to us, in which our competitor would have the benefit of the seven year market
exclusivity, and we would be unable to commercialize our product for the same indication until the expiration of the seven-year period.
Even if we are the first to obtain approval for the orphan drug indication, there are circumstances under which a competing product may be
approved for the same indication during our seven-year period of exclusivity.
Under the Orphan Drug Act, the FDA may grant orphan drug designation to a drug intended to treat a rare disease or condition,
which is generally a disease or condition that affects fewer than 200,000 individuals in the United States and for which there is no
reasonable expectation that the cost of developing and making a drug available in the Unites States for this type of disease or condition will
be recovered from sales of the product. Orphan drug designation must be requested before submitting an NDA. After the FDA grants
orphan drug 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 regulatory review and approval process. In addition to the
potential period of exclusivity, orphan designation makes a company eligible for grant funding of up to $400,000 per year for four years to
defray costs of clinical trial expenses, tax credits for clinical research expenses and potential exemption from the FDA application user fee.
If a product 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 drug exclusivity, which means the FDA may not approve any other applications to
market the same drug for the same indication for seven years, except in limited circumstances, such as (i) the drug’s orphan designation is
revoked; (ii) its marketing approval is withdrawn; (iii) the orphan exclusivity holder consents to the approval of another applicant’s
product; (iv) the orphan exclusivity holder is unable to assure the availability of a sufficient quantity of drug; or (v) a showing of clinical
superiority to the product with orphan exclusivity by a competitor product. If a drug designated as an orphan product receives marketing
approval for an indication broader than what is designated, it may not be entitled to orphan drug exclusivity. There can be no assurance that
we will receive orphan drug designation for JBT-101 for the treatment of dermatomyositis, or other inflammatory disease indications, if we
elect to seek such applications.
Any fast track designation or grant of priority review status by the FDA may not actually lead to a faster development or regulatory
review or approval process, nor will it assure FDA approval of our product candidates. Additionally, our product candidates may treat
indications that do not qualify for priority review vouchers.
We have received fast track designation for JBT-101 for the treatment of cystic fibrosis and systemic sclerosis in the United States
and European Union and may seek fast track designation or priority review of applications for approval of our product candidate for certain
indications. If a drug is intended for the treatment of a serious or life-threatening condition and the drug demonstrates the potential to
address unmet medical needs for this condition, the drug sponsor may apply for FDA fast track designation. If a product candidate offers
major advances in treatment, the FDA may designate it eligible for priority review. The FDA has broad discretion whether or not to grant
these designations, so even if we believe a particular product candidate is eligible for these designations, we cannot assure you that the
FDA would decide to grant them. Even if we do receive fast track designation or priority review, we may not experience a faster
development process, review or approval compared to conventional FDA procedures. The FDA may withdraw fast track designation if it
believes that the designation is no longer supported by data from our clinical development program.
Any breakthrough therapy designation granted by the FDA for our product candidate may not lead to a faster development or
regulatory review or approval process, and it does not increase the likelihood that our product candidate will receive marketing
approval.
We may seek a breakthrough therapy designation for our product candidate. 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. For drugs and biologics that have been
designated as breakthrough therapies, interaction and communication between the FDA and the sponsor of the trial can help to identify the
most efficient path for clinical development while minimizing the number of patients placed in ineffective control regimens. Drugs
designated as breakthrough therapies by the FDA may also be eligible for accelerated approval if the relevant criteria are met.
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Designation as a breakthrough therapy is within the discretion of the FDA. Accordingly, even if we believe our product candidate
meets the criteria for designation as a breakthrough therapy, the FDA may disagree and instead determine not to make such designation. In
any event, the receipt of a breakthrough therapy designation for a product candidate may not result in a faster development process, review
or approval compared to drugs considered for approval under conventional FDA procedures and does not assure ultimate approval by the
FDA. In addition, even if one or more of our product candidates qualify as breakthrough therapies, the FDA may later decide that the
products no longer meet the conditions for qualification or decide that the time period for FDA review or approval will not be shortened.
Third-party coverage and reimbursement and health care cost containment initiatives and treatment guidelines may constrain our
future revenues.
Our ability to successfully market JBT-101 will depend in part on the level of reimbursement that government health
administration authorities, private health coverage insurers and other organizations provide for the cost of our products and related
treatments. Countries in which JBT-101 is expected to be sold through reimbursement schemes under national health insurance programs
frequently require that manufacturers and sellers of pharmaceutical products obtain governmental approval of initial prices and any
subsequent price increases. In certain countries, including the United States, government-funded and private medical care plans can exert
significant indirect pressure on prices. We may not be able to sell JBT-101 profitably if adequate prices are not approved or coverage and
reimbursement is unavailable or limited in scope. Increasingly, third-party payors attempt to contain health care costs in ways that are likely
to impact our development of products including:
● failing to approve or challenging the prices charged for health care products;
● introducing reimportation schemes from lower priced jurisdictions;
● limiting both coverage and the amount of reimbursement for new therapeutic products;
● denying or limiting coverage for products that are approved by the regulatory agencies but are considered to be experimental or
investigational by third-party payors; and
● refusing to provide coverage when an approved product is used in a way that has not received regulatory marketing approval.
Risks Relating to Our Intellectual Property Rights
It is difficult and costly to protect our intellectual property rights, and we cannot ensure the protection of these rights.
Our commercial success will depend, in part, on obtaining and maintaining patent protection for our technologies, products and
processes, successfully defending these patents against third-party challenges and successfully enforcing these patents against third party
competitors. The patent positions of pharmaceutical companies can be highly uncertain and involve complex legal, scientific and factual
questions for which important legal principles remain unresolved. Changes in either the patent laws or in interpretations of patent laws may
diminish the value of our intellectual property. Accordingly, we cannot predict the breadth of claims that may be allowable in our pending
applications or, if we obtain patents from our applications, enforceable. Our pending patent applications for JBT-101 and its uses may
never be approved by United States or foreign patent offices and the existing patent applications relating to JBT-101 and related
technologies may be challenged, invalidated or circumvented by third parties and might not protect us against competitors with similar
products or technologies. We do not have any issued patents for JBT-101 for the treatment of cystic fibrosis, systemic sclerosis,
dermatomyositis, SLE or any other indications for which we are currently seeking commercial approval.
The degree of future protection for our proprietary rights is uncertain, because legal means afford only limited protection and may
not adequately protect our rights, permit us to gain or keep our competitive advantage, or provide us with any competitive advantage at all.
For example, others have filed, and in the future are likely to file, patent applications covering products and technologies that are similar,
identical or competitive to JBT-101, or important to our business. We cannot be certain that any patent application owned by a third party
will not have priority over patent applications filed by us, or that we will not be involved in interference, opposition or invalidity
proceedings before United States or foreign patent offices.
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We also rely on trade secrets to protect technology, especially in cases when we believe patent protection is not appropriate or
obtainable. However, trade secrets are difficult to protect. While we require employees, academic collaborators, consultants and other
contractors to enter into confidentiality agreements, we may not be able to adequately protect our trade secrets or other proprietary or
licensed information. Typically, research collaborators and scientific advisors have rights to publish data and information in which we may
have rights. If we cannot maintain the confidentiality of our proprietary technology and other confidential information, our ability to
receive patent protection and our ability to protect valuable information owned by us may be imperiled. Enforcing a claim that a third-party
entity illegally obtained and is using any of our trade secrets is expensive and time consuming, and the outcome is unpredictable. In
addition, courts are sometimes less willing to protect trade secrets than patents. Moreover, our competitors may independently develop
equivalent knowledge, methods and know-how.
If we fail to obtain or maintain patent protection or trade secret protection for JBT-101 or our technologies, third parties could use
our proprietary information, which could impair our ability to compete in the market and adversely affect our ability to generate revenues
and attain profitability.
We may also rely on the trademarks we may develop to distinguish our products from the products of our competitors. We cannot
guarantee that any trademark applications filed by us or our business partners will be approved. Third parties may also oppose such
trademark applications, or otherwise challenge our use of the trademarks. In the event that the trademarks we use are successfully
challenged, we could be forced to rebrand our products, which could result in loss of brand recognition, and could require us to devote
resources to advertising and marketing new brands. Further, we cannot provide assurance that competitors will not infringe the trademarks
we use, or that we will have adequate resources to enforce these trademarks.
JBT-101 may infringe the intellectual property rights of others, which could increase our costs and delay or prevent our development
and commercialization efforts.
Our success depends in part on avoiding infringement of the proprietary technologies of others. The pharmaceutical industry has
been characterized by frequent litigation regarding patent and other intellectual property rights. Identification of third party patent rights
that may be relevant to our proprietary technology is difficult because patent searching is imperfect due to differences in terminology
among patents, incomplete databases and the difficulty in assessing the meaning of patent claims. Additionally, because patent applications
are maintained in secrecy until the application is published, we may be unaware of third-party patents that may be infringed by
commercialization of JBT-101 or any future product candidate. There may be certain issued patents and patent applications claiming
subject matter that we may be required to license in order to research, develop or commercialize JBT-101, and we do not know if such
patents and patent applications would be available to license on commercially reasonable terms, or at all. Any claims of patent infringement
asserted by third parties would be time-consuming and may:
● result in costly litigation;
● divert the time and attention of our technical personnel and management;
● prevent us from commercializing a product until the asserted patent expires or is held finally invalid or not infringed in a court
of law;
● require us to cease or modify our use of the technology and/or develop non-infringing technology; or
● require us to enter into royalty or licensing agreements.
Although no third party has asserted a claim of infringement against us, others may hold proprietary rights that could prevent JBT-
101 from being marketed. Any patent-related legal action against us claiming damages and seeking to enjoin commercial activities relating
to JBT-101 or our processes could subject us to potential liability for damages and require us to obtain a license to continue to manufacture
or market JBT-101 or any future product candidates. We cannot predict whether we would prevail in any such actions or that any license
required under any of these patents would be made available on commercially acceptable terms, if at all. In addition, we cannot be sure that
we could redesign JBT-101 or any future product candidates or processes to avoid infringement, if necessary. Accordingly, an adverse
determination in a judicial or administrative proceeding, or the failure to obtain necessary licenses, could prevent us from developing and
commercializing JBT-101 or a future product candidate, which could harm our business, financial condition and operating results.
A number of companies, including several major pharmaceutical companies, have conducted research on anti-inflammatory and
anti-fibrosis therapies which resulted in the filing of many patent applications related to this research. If we were to challenge the validity of
these or any issued United States patent in court, we would need to overcome a statutory presumption of validity that attaches to every
issued United States patent. This means that, in order to prevail, we would have to present clear and convincing evidence as to the invalidity
of the patent’s claims.
39
If we were to challenge the validity of these or any issued United States patent in an administrative trial before the Patent Trial and
Appeal Board in the United States Patent and Trademark Office, we would have to prove that the claims are unpatentable by a
preponderance of the evidence. There is no assurance that a jury and/or court would find in our favor on questions of infringement, validity
or enforceability.
We may be subject to claims that we have wrongfully hired an employee from a competitor or that we or our employees have wrongfully
used or disclosed alleged confidential information or trade secrets of their former employers.
As is commonplace in our industry, we employ individuals who were previously employed at other pharmaceutical companies,
including our competitors or potential competitors. Although no claims against us are currently pending, we may be subject in the future to
claims that our employees or prospective employees are subject to a continuing obligation to their former employers (such as non-
competition or non-solicitation obligations) or claims that our employees or we have inadvertently or otherwise used or disclosed trade
secrets or other proprietary information of their former employers. Litigation may be necessary to defend against these claims. Even if we
are successful in defending against these claims, litigation could result in substantial costs and be a distraction to management.
General Company-Related Risks
We will need to grow the size of our organization, and we may experience difficulties in managing this growth.
As of December 31, 2016, we had 25 full-time employees. As our development and commercialization plans and strategies
develop, we will need to expand the size of our employee base for managerial, operational, sales, marketing, financial and other resources.
Future growth would impose significant added responsibilities on members of management, including the need to identify, recruit,
maintain, motivate and integrate additional employees. In addition, our management may have to divert a disproportionate amount of its
attention away from our day-to-day activities and devote a substantial amount of time to managing these growth activities. Our future
financial performance and our ability to commercialize JBT-101 and any other future product candidates and our ability to compete
effectively will depend, in part, on our ability to effectively manage our future growth.
Future capital raises may dilute our existing stockholders’ ownership and/or have other adverse effects on our operations.
If we raise additional capital by issuing equity securities, our existing stockholders’ percentage ownership will be reduced and
these stockholders may experience substantial dilution. We may also issue equity securities that provide for rights, preferences and
privileges senior to those of our common stock. If we raise additional funds by issuing debt securities, these debt securities would have
rights senior to those of our common stock and the terms of the debt securities issued could impose significant restrictions on our
operations, including liens on our assets. If we raise additional funds through collaborations and licensing arrangements, we may be
required to relinquish some rights to our technologies or candidate products, or to grant licenses on terms that are not favorable to us.
If we are not successful in attracting and retaining highly qualified personnel, we may not be able to successfully implement our
business strategy. In addition, the loss of the services of certain key employees, including Yuval Cohen, our CEO, Mark Tepper, our
President and Chief Scientific Officer, Barbara White, our Chief Medical Officer and Sean Moran, our Chief Financial Officer would
adversely impact our business prospects.
Our ability to compete in the highly competitive pharmaceuticals industry depends in large part upon our ability to attract highly
qualified managerial, scientific and medical personnel. In order to induce valuable employees to remain with us, we intend to provide
employees with stock options that vest over time. The value to employees of stock options that vest over time will be significantly affected
by movements in our stock price that we will not be able to control and may at any time be insufficient to counteract more lucrative offers
from other companies.
Our management team has expertise in many different aspects of drug development and commercialization. However, we will
need to hire additional personnel as we further develop JBT-101. Competition for skilled personnel in our market is intense and
competition for experienced scientists may limit our ability to hire and retain highly qualified personnel on acceptable terms. Despite our
efforts to retain valuable employees, members of our management, scientific and medical teams may terminate their employment with us on
short notice. In connection with the Merger, we entered into employment agreements with certain of our executive officers. However, these
employment arrangements provide for at-will employment, which means that any of our employees could leave our employment at any
time, with or without notice. The loss of the services of any of our executive officers or other key employees could potentially harm our
business, operating results or financial condition. In particular, we believe that the loss of the services of Yuval Cohen Ph.D., our Chief
Executive Officer, Mark Tepper Ph.D., our President and Chief Scientific Officer, Barbara White, M.D., our Chief Medical Officer and
Sean Moran, C.P.A., M.B.A., our Chief Financial Officer, would have a material adverse effect on our business. 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.
40
Other pharmaceutical companies with which we compete for qualified personnel have greater financial and other resources,
different risk profiles, and a longer history in the industry than we do. They also may 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 have to offer. If we
are unable to continue to attract and retain high-quality personnel, the rate and success at which we can develop and commercialize product
candidates would be limited.
If product liability lawsuits are brought against us, we may incur substantial liabilities and may be required to limit commercialization
of JBT-101.
We face a potential risk of product liability as a result of the clinical testing of JBT-101 and will face an even greater risk if we
commercialize JBT-101 or any other future product. For example, we may be sued if any product we develop, including JBT-101, or any
materials that we use in our products allegedly causes injury or is found to be otherwise unsuitable during product 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 ourselves against product liability claims, we may incur substantial liabilities or be
required to limit commercialization of JBT-101. Even successful defense would require significant financial and management resources.
Regardless of the merits or eventual outcome, liability claims may result in:
● decreased demand for JBT-101 or any future products that we may develop;
● injury to our reputation;
● withdrawal of clinical trial participants;
● costs to defend the related litigation;
● a diversion of management’s time and our resources;
● substantial monetary awards to trial participants or patients;
● product recalls, withdrawals or labeling, marketing or promotional restrictions;
● the inability to commercialize JBT-101; and
● a decline in the value of our stock.
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. We intend to obtain product liability insurance
covering our clinical trials. Although we will maintain such insurance, 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. Our insurance policies also have various exclusions, and we may be subject to a product liability claim for which we
have no coverage. We may have to pay any amounts awarded by a court or negotiated in a settlement that exceed our coverage limitations
or that are not covered by our insurance, and we may not have, or be able to obtain, sufficient capital to pay such amounts.
We may acquire businesses, assets or products, or form strategic alliances, in the future, and we may not realize the benefits of such
acquisitions.
We may acquire additional businesses, assets or products, form strategic alliances or create joint ventures with third parties that
we believe will complement or augment our existing business. If we acquire businesses with promising markets or technologies, we may
not be able to realize the benefit of acquiring such businesses if we are unable to successfully integrate them with our existing operations
and company culture. We may encounter numerous difficulties in developing, manufacturing and marketing any new delay or prevent us
from realizing their expected benefits or enhancing our business. We cannot assure you that, following any such acquisition, we will
achieve the expected synergies to justify the transaction.
41
Risks Related to our Common Stock
Our affiliates may control our company for the foreseeable future, including the outcome of matters requiring stockholder approval.
Our officers, directors, and five percent stockholders collectively owned approximately 20.1% of our outstanding shares of
common stock as of December 31, 2016. This concentration of voting power and control could have a significant effect in delaying,
deferring or preventing an action that might otherwise be beneficial to our other stockholders and be disadvantageous to our stockholders
with interests different from those entities and individuals. Certain of these individuals also have significant control over our business,
policies and affairs as officers or directors of our company. Therefore, you should not invest in reliance on your ability to have any control
over our company.
An active, liquid trading market for our common stock may not be sustained.
Presently, our common stock is traded on the Nasdaq Global Market and as we are in our early stages, an investment in our
company may require a long-term commitment, with no certainty of return. . If we are unable to maintain an active, liquid active trading
market:
● investors may have difficulty buying and selling or obtaining market quotations;
● market visibility for shares of our common stock may be limited; and
● a lack of visibility for shares of our common stock may have a depressive effect on the market price for shares of our common
stock.
The lack of an active market could impair your ability to sell your shares at the time you wish to sell them or at a price that you
consider reasonable. The lack of an active market may also reduce the fair market value of your shares. An inactive market may also impair
our ability to raise capital to continue to fund operations by selling shares and may impair our ability to acquire additional intellectual
property assets by using our shares as consideration.
We are currently listed on the Nasdaq Global Market. If we are unable to maintain listing of our securities on the Nasdaq Global
Market or any stock exchange, our stock price could be adversely affected and the liquidity of our stock and our ability to obtain
financing could be impaired and it may be more difficult for our stockholders to sell their securities.
Although our common stock is currently listed on the Nasdaq Global Market, we may not be able to continue to meet the
exchange’s minimum listing requirements or those of any other national exchange. If we are unable to maintain listing on the Nasdaq
Global Market or if a liquid market for our common stock does not develop or is sustained, our common stock may remain thinly traded.
The Listing Rules of the Nasdaq Global Market require listing issuers to comply with certain standards in order to remain listed on
its exchange. If, for any reason, we should fail to maintain compliance with these listing standards and Nasdaq should delist our securities
from trading on its exchange and we are unable to obtain listing on another national securities exchange, a reduction in some or all of the
following may occur, each of which could have a material adverse effect on our stockholders:
● the liquidity of our common stock;
● the market price of our common stock;
● our ability to obtain financing for the continuation of our operations;
● the number of institutional and general investors that will consider investing in our common stock;
● the number of investors in general that will consider investing in our common stock;
● the number of market makers in our common stock;
● the availability of information concerning the trading prices and volume of our common stock; and
● the number of broker-dealers willing to execute trades in shares of our common stock.
The market price of our common stock may be significantly volatile.
Even if an active market for our common stock develops, of which no assurances can be given, the market price for our common
stock may be volatile and subject to wide fluctuations in response to factors including the following:
● actual or anticipated fluctuations in our quarterly or annual operating results;
● changes in financial or operational estimates or projections;
● conditions in markets generally;
42
● changes in the economic performance or market valuations of companies similar to ours; and
● general economic or political conditions in the United States or elsewhere.
In particular, the market prices of biotechnology companies like ours have been highly volatile due to factors, including, but not
limited to:
● any delay or failure to conduct a clinical trial for our product or receive approval from the FDA and other regulatory agencies;
● developments or disputes concerning our product’s intellectual property rights;
● our or our competitors’ technological innovations;
● changes in market valuations of similar companies;
● announcements by us or our competitors of significant contracts, acquisitions, strategic partnerships, joint ventures, capital
commitments, new technologies, or patents; and
● failure to complete significant transactions or collaborate with vendors in manufacturing our product.
The securities market has from time to time experienced significant price and volume fluctuations that are not related to the
operating performance of particular companies. These market fluctuations may also materially and adversely affect the market price of
shares of our common stock.
Future sales of shares by existing stockholders could cause our stock price to decline.
As of December 31, 2016, we had outstanding options to purchase an aggregate of 6,610,179 shares of our common stock at a
weighted average exercise price of $2.54 per share and warrants to purchase an aggregate of 1,288,500 shares of our common stock at a
weighted average exercise price of $1.00 per share. The exercise of such outstanding options and warrants will result in further dilution of
your investment. If our existing stockholders sell substantial amounts of our common stock in the public market, or if the public perceives
that such sales could occur, this could have an adverse impact on the market price of our common stock, even if there is no relationship
between such sales and the performance of our business.
We are an “emerging growth company,” and will be able take advantage of reduced disclosure requirements applicable to “emerging
growth companies,” which could 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 JOBS Act, and, for as
long as we continue to be an “emerging growth company,” we intend to take advantage of certain exemptions from various reporting
requirements applicable to other public companies but not to “emerging growth companies,” including, but not limited to, not being
required to comply with the auditor attestation requirements of Section 404 of the Sarbanes-Oxley Act, reduced disclosure obligations
regarding executive compensation in our periodic reports and proxy statements, 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 will remain an emerging growth company until the earlier of (1) January 1, 2020, (2) the last day of the first fiscal year in which our
annual gross revenues exceed $1 billion, (3) the date on which we become a “large accelerated filer” as defined in Rule 12b-2 under the
Securities Exchange Act of 1934, as amended, which would occur if the market value of our common stock that is held by non-affiliates
exceeds $700 million as of the last business day of our most recently completed second fiscal quarter or (4) the date on which we have
issued more than $1 billion in non-convertible debt during the preceding three-year period.
We intend to take advantage of these reporting exemptions described above until we are no longer an “emerging growth
company.” 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, we will be subject to the same new or revised accounting standards as other public companies
that are not “emerging growth companies.”
We cannot predict if investors will find our common stock less attractive if we choose to rely on these exemptions. If some
investors find our common stock less attractive as a result of any choices to reduce future disclosure, there may be a less active trading
market for our common stock and our stock price may be more volatile.
43
We will incur significantly increased costs and devote substantial management time as a result of operating as a public company
particularly after we are no longer an “emerging growth company.”
As a public company, we incur significant legal, accounting and other expenses that we did not incur as a private company. For
example, we are required to comply with certain of the requirements of the Sarbanes-Oxley Act and the Dodd-Frank Wall Street Reform
and Consumer Protection Act, as well as rules and regulations subsequently implemented by the SEC, including the establishment and
maintenance of effective disclosure and financial controls and changes in corporate governance practices. We expect that compliance with
these requirements will increase our legal and financial compliance costs and will make some activities more time consuming and costly. In
addition, we expect that our management and other personnel will need to divert attention from operational and other business matters to
devote substantial time to these public company requirements. In particular, we expect to incur significant expenses and devote substantial
management effort toward ensuring compliance with the requirements of Section 404 of the Sarbanes-Oxley Act. In addition, after we are
no longer qualify as an “emerging growth company,” we expect to incur additional management time and cost to comply with the more
stringent reporting requirements applicable to companies that are deemed accelerated filers or large accelerated filers, including complying
with the auditor attestation requirements of Section 404 of the Sarbanes-Oxley Act. We currently do not have an internal audit function, and
we will need to hire or contract for additional accounting and financial staff with appropriate public company experience and technical
accounting knowledge.
We cannot predict or estimate the amount of additional costs we may incur as a result of becoming a public company or the timing
of such costs.
There may be limitations on the effectiveness of our internal controls, and a failure of our control systems to prevent error or fraud may
materially harm our company.
Proper systems of internal controls over financial accounting and disclosure are critical to the operation of a public company. As
of December 31, 2016, we had 25 full-time employees which results in a lack of segregation of duties and we may be unable to effectively
establish such systems, especially in light of the fact that we expect to operate as a publicly reporting company. This would leave us without
the ability to reliably assimilate and compile financial information about our company and significantly impair our ability to prevent error
and detect fraud, all of which would have a negative impact on our company from many perspectives.
Moreover, we do not expect that disclosure controls or internal control over financial reporting, will prevent all error and all fraud.
A control system, no matter how well designed and operated, can provide only reasonable, not absolute, assurance that the control system’s
objectives will be met. Further, the design of a control system must reflect the fact that there are resource constraints and the benefits of
controls must be considered relative to their costs. Because of the inherent limitations in all control systems, no evaluation of controls can
provide absolute assurance that all control issues and instances of fraud, if any, have been detected. Failure of our control systems to
prevent error or fraud could materially adversely impact us.
We do not currently intend to pay dividends on our common stock in the foreseeable future, and consequently, your ability to achieve a
return on your investment will depend on appreciation in the price of our common stock.
We have never declared or paid cash dividends on our common stock and do not anticipate paying any cash dividends to holders
of our common stock in the foreseeable future. Consequently, investors must rely on sales of their common stock after price appreciation,
which may never occur, as the only way to realize any future gains on their investments. There is no guarantee that shares of our common
stock will appreciate in value or even maintain the price at which our investors have purchased their shares.
We may be unable to complete our analysis of our internal controls over financial reporting in a timely manner, or these internal
controls may not be determined to be effective, which may adversely affect investor confidence in our company and, as a result, the
value of our common stock.
We are required, pursuant to Section 404 of the Sarbanes-Oxley Act, to furnish a report by our management on, among other
things, the effectiveness of our internal control over financial reporting. This assessment will need to include disclosure of any material
weaknesses identified by our management in our internal control over financial reporting.
We may not be able to complete our evaluation, testing and any required remediation in a timely fashion. During the evaluation
and testing process, if we identify one or more material weaknesses in our internal control over financial reporting, we will be unable to
assert that our internal controls are effective.
If we are unable to assert that our internal control over financial reporting is effective, or, if applicable, our independent registered
public accounting firm is unable to express an opinion on the effectiveness of our internal controls, we could lose investor confidence in
the accuracy and completeness of our financial reports, which would cause the price of our common stock to decline, and we may be
subject to investigation or sanctions by the SEC. We will also be required to disclose changes made in our internal control and procedures
on a quarterly basis.
44
Our remediation efforts may not enable us to avoid a material weakness in our internal control over financial reporting in the
future. Any of the foregoing occurrences, should they come to pass, could negatively impact the public perception of our company, which
could have a negative impact on our stock price.
Upon dissolution of our company, you may not recoup all or any portion of your investment.
In the event of a liquidation, dissolution or winding-up of our company, whether voluntary or involuntary, the proceeds and/or
assets of our company remaining after giving effect to such transaction, and the payment of all of our debts and liabilities and distributions
required to be made to holders of any outstanding preferred stock will then be distributed to the stockholders of common stock on a pro rata
basis. There can be no assurance that we will have available assets to pay to the holders of common stock, or any amounts, upon such a
liquidation, dissolution or winding-up of our Company. In this event, you could lose some or all of your investment.
Our ability to use our net operating loss carryforwards and certain other tax attributes may be limited.
As a result of our merger in April 2014 with Corbus Pharmaceuticals, Inc., our wholly-owned subsidiary, our ability to utilize our
federal net operating loss, carryforwards and federal tax credit prior to that date may be limited under Sections 382 of the Internal Revenue
Code. The limitations apply if an “ownership change,” as defined by Section 382, occurs. Generally, an ownership change occurs if the
percentage of the value of the stock that is owned by one or more direct or indirect “five percent shareholders” increases by more than 50
percentage points over their lowest ownership percentage at any time during the applicable testing period (typically three years). In
addition, future changes in our stock ownership, which may be outside of our control, may trigger an “ownership change” and,
consequently, Section 382 limitations. As a result, if we earn net taxable income, our ability to use our pre-change net operating loss
carryforwards and other tax attributes to offset United States federal taxable income may be subject to limitations, which could potentially
result in increased future tax liability to us.
Our certificate of incorporation, as amended, allows for our board to create new series of preferred stock without further approval by
our stockholders, which could adversely affect the rights of the holders of our common stock.
Our board of directors has the authority to fix and determine the relative rights and preferences of preferred stock. We anticipate
that our board of directors will have the authority to issue up to 10,000,000 shares of our preferred stock without further stockholder
approval. As a result, our board of directors could authorize the issuance of a series of preferred stock that would grant to holders the
preferred right to our assets upon liquidation and the right to receive dividend payments before dividends are distributed to the holders of
common stock. In addition, our board of directors could authorize the issuance of a series of preferred stock that has greater voting power
than our common stock or that is convertible into our common stock, which could decrease the relative voting power of our common stock
or result in dilution to our existing stockholders.
Item 1B.
UNRESOLVED STAFF COMMENTS
Not applicable.
Item 2.
PROPERTIES
Our principal offices are located at 100 River Ridge Drive, Norwood, MA 02062. In September 2016, our lease for this office
space was amended for our expansion into an additional 4,088 square feet of office space within the existing building for an aggregate total
of 10,414 square feet of leased office space. The lease for this office space terminates in January 2021. As of December 31, 2016, our
operating lease commitment through the end of the lease was approximately $1,030,000. We believe our facilities are adequate for our
foreseeable needs.
Item 3.
LEGAL PROCEEDINGS
We are not currently subject to any material legal proceedings. However, we may from time to time become a party to various legal
proceedings arising in the ordinary course of our business.
Item 4.
MINE SAFETY DISCLOSURES
Not applicable.
45
PART II
Item 5.
MARKET FOR REGISTRANT’S COMMON EQUITY, RELATED STOCKHOLDER MATTERS AND ISSUER
PURCHASES OF EQUITY SECURITIES
Market Information
Our common stock is currently listed on the Nasdaq Global Market under the symbol “CRBP.” Our shares of common stock
began trading on the Nasdaq Capital Market under the symbol “CRBP” effective April 16, 2015. Prior to April 16, 2015, our common stock
was quoted on the Over-the Counter Markets (the “OTC.QB”) under the symbol “CRBP.” Our shares of common stock began being
quoted on the OTC.QB effective October 24, 2014.
The following table contains information about the range of high and low sale prices for our common stock for each quarter
during the last two years. The source of these high and low sales prices was the Nasdaq Global Market, the Nasdaq Capital Market and the
OTC.QB.
Fiscal Year Ended December 31, 2016
First Quarter,
Second Quarter
Third Quarter
Fourth Quarter
Fiscal Year Ended December 31, 2015
First Quarter
Second Quarter
Third Quarter
Fourth Quarter
Dividends
High Sales Price Low Sales Price
1.01
1.95 $
$
1.78
3.85 $
$
2.68
7.88 $
$
4.65
10.78 $
$
High Sales Price Low Sales Price
2.00
3.25 $
$
2.63
4.31 $
$
1.45
4.22 $
$
1.50
2.55 $
$
We have never declared or paid cash dividends on our common stock. We do not intend to declare or pay cash dividends on our
common stock for the foreseeable future, but currently intend to retain any future earnings to fund the development and growth of our
business. The payment of cash dividends if any, on the common stock will rest solely within the discretion of our board of directors and
will depend, among other things, upon our earnings, capital requirements, financial condition, and other relevant factors.
Record Holders
As of March 6, 2017, there are approximately 114 record holders of shares of common stock.
Item 6.
SELECTED FINANCIAL DATA
Not applicable.
46
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 together with our financial
statements and the related notes and the other financial information included elsewhere in this Annual Report. This discussion contains
forward-looking statements that involve risks and uncertainties. Our actual results could differ materially from those anticipated in these
forward-looking statements as a result of various factors, including those discussed below and elsewhere in this Annual Report,
particularly those under “Risk Factors.”
Overview
We are a clinical stage pharmaceutical company, focused on the development and commercialization of novel therapeutics to treat
rare, chronic and serious inflammatory and fibrotic diseases with clear unmet medical needs. Our product JBT-101 is a novel synthetic oral
endocannabinoid-mimetic drug that is intended to resolve chronic inflammation and halt fibrotic processes without causing
immunosuppression. JBT-101 is currently being developed to treat four life-threatening diseases: systemic sclerosis, cystic fibrosis, diffuse
cutaneous, skin-predominant dermatomyositis and systemic lupus erythematosus (“SLE’’).
In November 2016, we reported positive clinical data in a Phase 2 JBT-101 study for the treatment of systemic sclerosis. We
conducted an end of Phase 2 meeting with the FDA in late February 2017 and we expect to begin a Phase 3 clinical program in systemic
sclerosis by the end of the third quarter of 2017. Based on the positive Phase 2 clinical results in systemic sclerosis, we also filed an
application with the FDA in March 2017 for Breakthrough Therapy designation.
In December 2016, we completed a second Phase 2 study in cystic fibrosis study and expect to report top-line data from this study
by the end of March 2017. A third Phase 2 study in dermatomyositis is expected to be completed in the third quarter of 2017 and a fourth
Phase 2 in systemic lupus erythematosus is planned to start during the second quarter of 2017. The United States Food and Drug
Administration (“FDA”) has granted JBT-101 Orphan Designation as well as Fast Track Status for both cystic fibrosis and systemic
sclerosis.
Financial Operations Overview
We are a clinical stage pharmaceutical company and have not generated any revenues from the sale of products. We have never
been profitable and, from inception through December 2016, our losses from operations have been approximately $33.3 million. Our net
losses for the years ended December 31, 2016 and 2015 were approximately $19,999,000 and $8,851,000, respectively. We expect to
continue to incur significant expenses and increasing operating losses for the foreseeable future. We expect our expenses to increase
significantly in connection with our ongoing activities to develop, seek regulatory approval and commercialization of JBT-101.
Furthermore, we expect to incur additional costs associated with operating as a public company. Accordingly, we will need additional
financing to support our continuing operations. We will seek to fund our operations through public or private equity or debt financings or
other sources, which may include government grants and collaborations with third parties. Adequate additional financing may not be
available to us on acceptable terms, or at all. Our failure to raise capital as and when needed would have a negative impact on our financial
condition and our ability to pursue our business strategy. We will need to generate significant revenues to achieve profitability, and we may
never do so.
We expect to continue to incur significant expenses and increasing operating losses for at least the next several years. We expect
our expenses will increase substantially in 2016 and in the future in connection with our ongoing activities, as we:
● conduct clinical trials for JBT-101 in scleroderma, cystic fibrosis, systemic lupus erythematosus and other indications;
● continue our research and development efforts;
● manufacture clinical study materials and develop commercial scale manufacturing capabilities;
● seek regulatory approval for our product candidates;
● add personnel to support development of our product candidates; and
● operate as a public company.
47
Critical Accounting Policies and Estimates
Our consolidated financial statements have been prepared in accordance with accounting principles generally accepted in the
United States of America. The preparation of these financial statements requires management to make estimates and assumptions that
affect the reported amounts of assets and liabilities and the disclosure of contingent assets and liabilities at the date of the financial
statements and the reported amounts of revenues and expenses during the reporting period.
On an ongoing basis, we evaluate our estimates and judgments for all assets and liabilities, including those related to stock-based
compensation expense and the fair value determined for stock purchase warrants classified as derivative liabilities. We base our estimates
and judgments on historical experience, current economic and industry conditions and on various other factors that are believed to be
reasonable under the circumstances. This forms the basis for making judgments about the carrying values of assets and liabilities that are
not readily apparent from other sources. Actual results may differ from these estimates under different assumptions or conditions.
We believe that full consideration has been given to all relevant circumstances that we may be subject to, and the consolidated
financial statements accurately reflect our best estimate of the results of operations, financial position and cash flows for the periods
presented.
Revenue
To date, we have not generated any revenues from the sales of products. We do not expect to generate revenue from product sales
unless and until we successfully complete development and obtain regulatory approval for the marketing of JBT-101, which we expect will
take a number of years and is subject to significant uncertainty.
We have recorded $1,911,424 and $648,382 of collaboration revenue in the year ended December 31, 2016 and December 31,
2015, respectively, related to an award agreement we entered into in fiscal 2015 with the CFFT, pursuant to which we received a
development award (the “Award”) for up to $5 million in funding. The funding from the Award is supporting the Phase 2 clinical trial of
JBT-101 in adults with cystic fibrosis. We have billed and received a total of $4.5 million in payments since the inception of the Award as
outlined below. The payments received under the Award have been recorded as deferred revenue and are being amortized on a straight-line
basis over the expected duration of the performance period under the Award, which is expected to conclude in the second quarter of 2017.
Upon the execution of the Award agreement, we received a payment of $1,250,000 in May 2015. In November 2015, we received
a second payment of $1,250,000 upon the achievement of a milestone for dosing the first patient. In August 2016, we received a third
payment from the CFFT in the amount of $1,000,000 for achieving a milestone in July 2016 related to dosing the median clinical trial
patient. In January 2017, we received a fourth payment from the CFFT in the amount of $1,000,000 for achieving a milestone in December
2016 related to completing the final visit for the final patient. We expect that the last milestone payment of $500,000 under the Award will
be recognized in the second quarter of 2017 upon the achievement of the final milestone related to the Phase 2 CF clinical trial, as set forth
in the Award agreement.
Research and Development
Research and development expenses are incurred for the development of JBT-101 and consist primarily of payroll, and payments
to contract research and development companies. To date, these costs are related to generating pre-clinical data and the cost of
manufacturing JBT-101 for clinical trials and conducting clinical trials. These costs are expected to increase significantly in the future as
JBT-101 is continued to be evaluated in additional later stage clinical trials.
General and Administrative Expenses
General and administrative expenses consist primarily of payroll, rent and professional services. Other general and administrative
expenses include accounting and legal services. We anticipate that our general and administrative expenses will increase significantly
during 2017 and in the future as we increase our headcount to support our continued research and development and the potential
commercialization of our product candidates. We also anticipate increased expenses related to audit, legal, and tax-related services
associated with maintaining compliance with NASDAQ exchange listing and SEC requirements, director and officer insurance, and investor
relations costs associated with being a public company.
48
Total Other Income (Expense)
Total other income (expense) consists primarily of interest income we earn on interest-bearing accounts, interest expense incurred
on our outstanding debt, and gains or losses related to foreign currency exchange rate fluctuations.
Accrued Research and Development Expenses
As part of the process of preparing financial statements, we are required to estimate and accrue expenses, the largest of which are
research and development expenses. This process involves: communicating with our applicable personnel to identify 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 actual cost; estimating and accruing expenses in our financial statements as of each balance
sheet date based on facts and circumstances known to us at the time; and periodically confirming the accuracy of our estimates with
selected service providers and making adjustments, if necessary.
Examples of estimated research and development expenses that we accrue include:
●
●
●
●
fees paid to CROs in connection with nonclinical studies;
fees paid to contract manufacturers in connection with the production of JBT-101 for clinical trials ;
fees paid to CRO and research institutions in connection with conducting of clinical studies; and
professional service fees for consulting and related services.
We base our expense accruals related to clinical studies on our estimates of the services performed pursuant to contracts with
multiple research institutions and clinical research organizations that conduct and manage clinical studies on our behalf. The financial terms
of these agreements vary from contract to contract and may result in uneven payment flows. Payments under some of these contracts
depend on factors, such as the successful enrollment of patients and the completion of clinical study milestones. Our service providers
invoice us monthly in arrears for services performed. In accruing service fees, we estimate the time period over which services will be
performed and the level of effort to be expended in each period. If we do not identify costs that we have begun to incur or if we
underestimate or overestimate the level of services performed or the costs of these services, our actual expenses could differ from our
estimates.
To date, we have not experienced significant changes in our estimates of accrued research and development expenses following
each applicable reporting period. However, due to the nature of estimates, we cannot assure you that we will not make changes to our
estimates in the future as we become aware of additional information regarding the status or conduct of our clinical studies and other
research activities.
Stock-Based Compensation
Stock options are granted with an exercise price at no less than fair market value at the date of the grant. The stock options
normally expire ten years from the date of grant. Stock option awards vest upon terms determined by our board of directors.
We recognize compensation costs resulting from the issuance of stock-based awards to employees, members of our Board of
directors and consultants. The fair value of each option grant was estimated as of the date of grant using the Black-Scholes option-pricing
model. The fair value is amortized as compensation cost on a straight-line basis over the requisite service period of the awards, which is
generally the vesting period. Due to our limited operating history and limited volume of sales of our common stock, we estimated our
volatility in consideration of a number of factors, including the volatility of comparable public companies and, commencing in 2015, we
also included the volatility of our own common stock. We use historical data, as well as subsequent events occurring prior to the issuance
of the consolidated financial statements, to estimate option exercise and employee forfeitures within the valuation model. The expected
term of options granted to employees under our stock plans is based on the average of the contractual term (generally 10 years) and the
vesting period (generally 48 months). The expected term of options granted under the 2014 Plan, all of which qualify as “plain vanilla” per
SEC Staff Accounting Bulletin 107, is based on the average of the 6.25 years. For non-employee options, the expected term is the
contractual term and stock options granted to non-employee consultants are revalued at the end of each reporting period until vested and
changes in their fair value are recorded as adjustments to expense over the related vesting period. The risk-free rate is based on the yield of
a U.S. Treasury security with a term consistent with the option. The risk-free rate is based on the yield of a U.S. Treasury security with a
term consistent with the expected term of the option. We estimate the forfeiture rate at the time of grant and revise it, if necessary, in
subsequent periods if actual forfeitures differ from those estimates. Forfeitures were estimated based on management’s expectation through
industry knowledge and historical data. We have never paid dividends on our common stock and do not anticipate paying dividends on our
common stock in the foreseeable future. Accordingly, we have assumed no dividend yield for purposes of estimating the fair value of our
share-based compensation.
49
The following assumptions were used to estimate the fair value of stock options granted using the Black-Scholes option pricing
model for the years ended December 31, 2016 and 2015 is as follows:
Risk free interest rate
Expected dividend yield
Expected term in years
Expected volatility
Estimated forfeiture rate
Emerging Growth Company Status
2016
2015
1.70%
0%
6.66
90.39%
5.00%
1.85%
0%
6.73
90.68%
4.83%
Under Section 107(b) of the Jumpstart Our Business Startups Act of 2012, emerging growth companies can 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, we will be subject to the same new or revised
accounting standards as other public companies that are not emerging growth companies.
Results of Operations
Comparison of Year Ended 2016 to 2015
Collaboration Revenue. We have recorded $1,911,424 and $648,382 of collaboration revenue in the year ended December 31, 2016 and
December 31, 2015, respectively, related to an award agreement we entered into in fiscal 2015 with the CFFT, pursuant to which we
received a development award (the “Award”) for up to $5 million in funding. The funding from the Award is supporting the Phase 2
clinical trial of JBT-101 in adults with cystic fibrosis. We have billed and received a total of $4.5 million in payments since the inception of
the Award as outlined below. The payments received under the award have been recorded as deferred revenue and are being amortized on a
straight-line basis over the expected duration of the performance period under the Award, which is expected to conclude in the second
quarter of 2017.
Upon the execution of the Award agreement, we received a payment of $1,250,000 in May 2015. In November 2015, we received
a second payment of $1,250,000 upon the achievement of a milestone for dosing the first patient. In August 2016, we received a third
payment from the CFFT in the amount of $1,000,000 for achieving a milestone in July 2016 related to dosing the median clinical trial
patient. In January 2017, we received a fourth payment from the CFFT in the amount of $1,000,000 for achieving a milestone in December
2016 related to completing the final visit for the final patient. We expect that the last milestone payment of $500,000 under the Award will
be recorded in the second quarter of 2017 upon the achievement of the final milestone related to the Phase 2 CF clinical trial, as set forth in
the Award agreement.
Research and Development. Research and Development expenses for the year ended December 31, 2016 totaled approximately
$15,437,000, an increase of $9,548,000 over the $5,889,000 recorded for the year ended December 31, 2015. The increase in fiscal 2016 as
compared to fiscal 2015 was primarily attributable to increases of $7,132,000 in clinical trial costs, $1,447,000 in compensation costs, and
$969,000 in stock-based compensation expense.
General and Administrative. General and Administrative expense for the year ended December 31, 2016 totaled approximately
$6,460,000, an increase of $2,847,000 over the $3,613,000 recorded for year ended December 31, 2015. The increase in fiscal 2016 as
compared to fiscal 2015 was primarily attributable to increases of approximately $1,251,000 in stock-based compensation expense,
$997,000 in compensation costs, $375,000 in investor relations costs, and $213,000 in legal costs.
Other Income (Loss), Net. Other loss, net for fiscal 2016 was approximately $14,000 as compared to other income, net of approximately
$3,000 recorded for fiscal 2015 and was primarily attributable to an increase in foreign currency exchange transaction losses recorded
during fiscal 2016.
Liquidity and Capital Resources
Since inception, we have experienced negative cash flows from operations. We have financed our operations primarily through
sales of equity-related securities. In addition, the majority of the costs of the dermatomyositis and systemic lupus erythematosus clinical
trials are being funded by NIH grants and our cystic fibrosis clinical trial has been partially funded by a $5 million award from the CFFT.
At December 31, 2016, our accumulated deficit since inception was approximately $33,276,000.
50
At December 31, 2016, we had total current assets of approximately $17,403,000 and current liabilities of approximately
$8,899,000 resulting in working capital of $8,504,000. Net cash used in operating activities for the year ended December 31, 2016 was
approximately $13,571,000, which includes a net loss of approximately $19,999,000, non-cash expenses of approximately $3,341,000
principally related to the increase in stock-based compensation expense, and approximately $3,086,000 of cash provided from a change in
net working capital items principally related to the increase in accounts payable and accrued expenses.
Cash used in investing activities for the year ended December 31, 2016 totaled approximately $353,000 for the purchase of
property and equipment.
Cash provided by financing activities for the year ended December 31, 2016 totaled approximately $16,742,000. In June 2016 we
sold 5,960,000 shares of our common stock in a registered direct offering to investors in June 2016 at a purchase price of $2.50 per share
which resulted in net proceeds to us totaling approximately $14,875,000. In December 2016, we entered into a sales agreement with Cantor
Fitzgerald under which we may direct Cantor Fitzgerald as our placement agent to sell common stock under an “At the Market Offering”
(“Sales Agreement). Sales of common stock under the Sales Agreement are made pursuant to an effective registration statement for an
aggregate offering of up to $35 million. In 2016, we sold 188,695 shares of our common stock under the Sales Agreement at an average
selling price of $8.54 per share (net of 3% commission paid to Cantor Fitzgerald) which resulted in gross proceeds of approximately
$1,621,000 of which $330,413 was classified in stock subscriptions receivable as of December 31, 2016 because the Company did not
receive these proceeds until January 2017.
During the year ended December 31, 2016, we also received proceeds of approximately $468,000 from the issuance of 927,916
shares of our common stock upon the exercise of stock options and warrants. Cash provided by financing activities for the year ended
December 31, 2016 included proceeds from issuances of notes payable of $349,000, partially offset by principal payments on notes payable
of approximately $239,000 in connection with our loan agreement with a financing company. The terms of the loan that we entered into in
October 2016 stipulate equal monthly payments of principal and interest payments of $39,114 over a nine-month period. Interest accrues
on this loan at an annual rate of 2.25%.
At December 31, 2016, we had cash and cash equivalents of approximately $14,992,000. On February 28, 2017, we entered into a
securities purchase agreement providing for the issuance and sale of 3,887,815 shares of our common stock in a registered direct offering to
institutional and accredited investors at a purchase price of $7.00 per share with gross proceeds to us totaling $27,214,705 less estimated
issuance costs of approximately $100,000 (“February 2017 Registered Direct Offering”). We expect our cash on hand of $14,992,257 at
December 31, 2016 together with the proceeds from the February 2017 Registered Direct Offering and the remaining milestone payment of
$500,000 from the CFFT, which we expect to receive in the second quarter of 2017, to be sufficient to meet our operating and capital
requirements into the fourth quarter of 2018 based on current planned expenditures.
We will need to raise significant additional capital to continue to fund operations and the clinical trials for JBT-101. We may seek
to sell common or preferred equity or convertible debt securities, enter into a credit facility or another form of third-party funding, or seek
other debt financing. In addition, we may seek to raise cash through collaborative agreements or from government grants. The sale of
equity and convertible debt securities may result in dilution to our stockholders and those securities may have rights senior to those of our
common shares. If we raise additional funds through the issuance of preferred stock, convertible debt securities or other debt financing,
these securities or other debt could contain covenants that would restrict our operations. Any other third-party funding arrangement could
require us to relinquish valuable rights.
The source, timing and availability of any future financing will depend principally upon market conditions, and, more specifically,
on the progress of our clinical development programs. Funding may not be available when needed, at all, or on terms acceptable to us. Lack
of necessary funds may require us, among other things, to delay, scale back or eliminate expenses including some or all of our planned
clinical trials.
51
Contractual Obligations and Commitments
The following table presents information about our known contractual obligations as of December 31, 2016. It does not reflect
contractual obligations that may have arisen or may arise after that date. Except for historical facts, the information in this section is
forward-looking information.
Payments due by period
Contractual Obligations
Operating lease obligations (1)
Capital lease obligations (2)
Obligation with related party(3)
Total
Total
$ 1,030,118 $
9,465
50,000
$ 1,089,583 $
2017
244,295 $
4,543
50,000
298,838 $
Fiscal 2018-2019
Fiscal 2020-2021
After Fiscal 2021
504,211 $
4,922
—
509,133 $
281,612 $
—
—
281,612 $
—
—
—
—
(1)
(2)
(3)
In September 2016, our lease for this office space was amended for our expansion into an additional 4,088 square feet of office
space within the existing building for an aggregate total of 10,414 square feet of leased office space. The lease for this office space
terminates in January 2021.
On December 30, 2015, we entered into a lease agreement for a copier machine. The machine was placed in service in January
2016. The lease is for a three-year term and includes a bargain purchase option at the end of the term.
On September 21, 2016, we entered into a consulting agreement with Orchestra Medical Ventures, LLC (“Orchestra”), of which
David Hochman, a member of our Board of Directors, is Managing Partner. The agreement provides that Orchestra would render a
variety of consulting and advisory services relating principally to identifying and evaluating strategic relationships, licensing
opportunities, and business strategies. Pursuant to the terms of this agreement, we will pay to Orchestra cash compensation in an
aggregate amount of $100,000, payable in equal monthly installments, of which $50,000 was paid during fiscal 2016 and the
remaining $50,000 will be paid in the first quarter of 2017.
We may enter into contracts in the normal course of business with clinical research organizations for clinical trials and clinical
supply manufacturing and with vendors for pre-clinical research studies, research supplies and other services and products for operating
purposes. These contracts generally provide for termination on notice, and therefore, we believe that our non-cancelable obligations under
these agreements are not material. As of December 31, 2016, other than our leases in the table above, we had no material Contractual
Obligations or Commitments that will affect our future liquidity.
Off-Balance Sheet Arrangements
We do not have any off-balance sheet arrangements that have or are reasonably likely to have a current or future effect on our
financial condition, changes in financial condition, revenues or expenses, results of operations, liquidity, capital expenditures or capital
resources that is material to investors.
Item 7A.
QUANTITATIVE AND QUALITATIVE DISCLOSURES ABOUT MARKET RISK
Not applicable.
Item 8.
FINANCIAL STATEMENTS AND SUPPLEMENTARY DATA
See pages F-1 through F-20 following the Exhibit Index of this Annual Report on Form 10-K.
Item 9.
CHANGES IN AND DISAGREEMENTS WITH ACCOUNTANTS ON ACCOUNTING AND FINANCIAL
DISCLOSURE
None.
Item 9A.
CONTROLS AND PROCEDURES
Evaluation of Our Disclosure Controls
We maintain disclosure controls and procedures that are designed to provide reasonable assurance that material information
required to be disclosed in our periodic reports filed under the Exchange Act is recorded, processed, summarized, and reported within the
time periods specified in the SEC’s rules and forms and to provide reasonable assurance that such information is accumulated and
communicated to our management, our chief executive officer and our chief financial officer, to allow timely decisions regarding required
disclosure. We carried out an evaluation, under the supervision and with the participation of our management, including our principal
executive and principal financial officer, of the effectiveness of the design and operation of our disclosure controls and procedures, as
defined in Rule 13(a)-15(e) under the Exchange Act. Based on this evaluation, our principal executive officer and principal financial officer
concluded that, as of December 31, 2016, our disclosure controls and procedures were effective.
52
Management’s Report on Internal Control Over Financial Reporting
Our management is responsible for establishing and maintaining adequate internal control over financial reporting as defined in
Rules 13a-15(f) and 15d-15(f) under the Exchange Act. Under the supervision and with the participation of our management, including our
Chief Executive Officer and Chief Financial Officer, we conducted an evaluation of the effectiveness of our internal control over financial
reporting based on criteria established in the framework in Internal Control — Integrated Framework (2013) issued by the Committee of
Sponsoring Organizations of the Treadway Commission. Based on this evaluation, our management concluded that our internal control over
financial reporting was effective as of December 31, 2016.
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 risks that controls may become inadequate because of
changes in conditions, or that the degree of compliance with the policies or procedures may deteriorate.
This Annual Report does not include an attestation report of our independent registered public accounting firm because we are an
“emerging growth company,” and may take advantage of certain exemptions from various reporting requirements that are applicable to
public companies that are not “emerging growth companies” including, but not limited to, not being required to comply with the auditor
attestation requirements of Section 404 of the Sarbanes-Oxley Act.
Changes in Internal Controls over Financial Reporting
During the year ended December 31, 2016, there have been no changes in our internal control over financial reporting that have
materially affected or are reasonably likely to materially affect our internal controls over financial reporting. From time to time, we make
changes to our internal control over financial reporting that are intended to enhance its effectiveness and which do not have a material effect
on our overall internal control over financial reporting.
Item 9B.
OTHER INFORMATION
None
53
Item 10.
DIRECTORS, EXECUTIVE OFFICERS AND CORPORATE GOVERNANCE
PART III
Information required by this item is incorporated by reference to our Proxy Statement for the 2017 Annual Meeting of
Stockholders.
Item 11.
EXECUTIVE COMPENSATION
Information required by this item is incorporated herein by reference to our Proxy Statement for the 2017 Annual Meeting of
Stockholders.
Item 12.
SECURITY OWNERSHIP OF CERTAIN BENEFICIAL OWNERS AND MANAGEMENT AND RELATED
STOCKHOLDER MATTERS
Information required by this item is incorporated by reference to our Proxy Statement for the 2017 Annual Meeting of
Stockholders.
Item 13.
CERTAIN RELATIONSHIPS AND RELATED TRANSACTIONS, AND DIRECTOR INDEPENDENCE
Information required by this item is incorporated by reference to our Proxy Statement for the 2017 Annual Meeting of
Stockholders.
Item 14.
PRINCIPAL ACCOUNTING FEES AND SERVICES
Information required by this item is incorporated by reference to our Proxy Statement for the 2017 Annual Meeting of
Stockholders.
54
Item 15.
EXHIBITS, FINANCIAL STATEMENT SCHEDULES
PART IV
(a) List of Documents filed as part of this Report
(1) Consolidated Financial Statements
The financial statements and related notes, together with the report of EisnerAmper LLP appear at pages F-1 through F-19
following the Exhibit List as required by Part II, Item 8 “Financial Statements and Supplementary Data” of this Form 10-K.
(2) Financial Statement Schedules.
Schedules are omitted because they are either not required, not applicable, or the information is otherwise included.
(3) Exhibits
The Company has filed with this report or incorporated by reference herein certain exhibits as specified below pursuant to Rule
12b-32 under the Exchange Act. See Exhibit Index following the signature page to this report for a complete list of documents filed with
this report.
Exhibit No.
Description
3.1
Certificate of Incorporation (incorporated by reference to Exhibit 3.1 of the Company’s Registration Statement on Form S-1
filed with the SEC on September 3, 2014).
3.2
Certificate of Amendment (incorporated by reference to Exhibit 3.2 of the Company’s Registration Statement on Form S-1
filed with the SEC on September 3, 2014).
3.3
Bylaws (incorporated by reference to Exhibit 3.3 of the Company’s Registration Statement on Form S-1 filed with the SEC
on September 3, 2014).
4.1
Form of Merger Warrant (incorporated by reference to Exhibit 4.1 of the Company’s Registration Statement on Form S-1
filed with the SEC on September 3, 2014).
4.2
Form of Replacement Warrant (incorporated by reference to Exhibit 4.2 of the Company’s Registration Statement on Form
S-1 filed with the SEC on September 3, 2014).
4.3
Form of Investor Warrant (incorporated by reference to Exhibit 4.3 of the Company’s Registration Statement on Form S-1
filed with the SEC on September 3, 2014).
4.4
Form of Additional Replacement Warrant (incorporated by reference to Exhibit 4.4 of the Company’s Registration
Statement on Form S-1 filed with the SEC on September 3, 2014).
4.5
Form of Placement Agent Warrant (incorporated by reference to Exhibit 4.5 of the Company’s Registration Statement on
Form S-1 filed with the SEC on September 3, 2014).
4.6
Registration Rights Agreement (incorporated by reference to Exhibit 4.6 of the Company’s Registration Statement on Form
S-1 filed with the SEC on September 3, 2014).
4.7
Specimen Common Stock Certificate, $0.0001 par value (incorporated herein by reference to Exhibit 4.1 of the Company’s
Registration Statement on Form S-3 filed with the SEC on November 10, 2015).
10.1
Placement Agency Agreement, dated March 27, 2014, between the Company and Aegis Capital Corporation (incorporated
by reference to Exhibit 10.1 of the Company’s Registration Statement on Form S-1 filed with the SEC on September 3,
2014).
10.2
Consulting Agreement, dated March 21, 2014, between the Company and Orchestra Medical Ventures (incorporated by
reference to Exhibit 10.2 of the Company’s Registration Statement on Form S-1 filed with the SEC on September 3, 2014).
55
Exhibit No
Description
10.3
Form of Subscription Agreement for the Company’s 2014 Private Placement (incorporated by reference to Exhibit 10.3 of
the Company’s Registration Statement on Form S-1 filed with the SEC on September 3, 2014).
10.4
Form of Voting Agreement, dated April 11, 2014, by and among the Company and the stockholders named therein
(incorporated by reference to Exhibit 10.4 of the Company’s Registration Statement on Form S-1 filed with the SEC on
September 3, 2014).
10.5
2014 Equity Compensation Plan (incorporated by reference to Exhibit 10.5 of the Company’s Registration Statement on
Form S-1 filed with the SEC on September 3, 2014).
10.6
Form of Incentive Stock Option Agreement (incorporated by reference to Exhibit 10.6 of the Company’s Registration
Statement on Form S-1 filed with the SEC on September 3, 2014).
10.7
Form of Non-Qualified Stock Option Agreement (incorporated by reference to Exhibit 10.7 of the Company’s Registration
Statement on Form S-1 filed with the SEC on September 3, 2014).
10.8
Form of Restricted Stock Agreement (incorporated by reference to Exhibit 10.8 of the Company’s Registration Statement
on Form S-1 filed with the SEC on September 3, 2014).
10.9
Employment Agreement, dated April 11, 2014, between the Company and Yuval Cohen (incorporated by reference to
Exhibit 10.9 of the Company’s Registration Statement on Form S-1 filed with the SEC on September 3, 2014).
10.10
Employment Agreement, dated April 11, 2014, between the Company and Mark Tepper (incorporated by reference to
Exhibit 10.10 of the Company’s Registration Statement on Form S-1 filed with the SEC on September 3, 2014).
10.11
10.12
10.13
10.14
Amended and Restated Employment Agreement, dated June 19, 2014, between the Company and Sean Moran (incorporated
by reference to Exhibit 10.11 of the Company’s Registration Statement on Form S-1 filed with the SEC on September 3,
2014).
Agreement and Plan of Merger, dated March 27, 2014, by and among the Company, Corbus Pharmaceuticals Acquisition,
Inc. and JB Therapeutics, Inc. (incorporated by reference to Exhibit 10.12 of the Company’s Registration Statement on
Form S-1 filed with the SEC on September 3, 2014).
Subscription Agreement, dated April 2009, between Sumner Burstein and JB Therapeutics, Inc. (which is now known as
Corbus Pharmaceuticals, Inc.) (incorporated by reference to Exhibit 10.13 of the Company’s Registration Statement on
Form S-1 filed with the SEC on September 3, 2014).
Letter Agreement, dated April 29, 2009, between JB Therapeutics, Inc. (which is now known as Corbus Pharmaceuticals,
Inc.) and Sumner Burstein (incorporated by reference to Exhibit 10.14 of the Company’s Registration Statement on Form S-
1 filed with the SEC on September 3, 2014).
10.15
Form of Indemnification Agreement (incorporated by reference to Exhibit 10.15 of the Company’s Registration Statement
on Amendment No. 1 to Form S-1 filed with the SEC on September 30, 2014).
10.16
Letter Agreement, dated August 18, 2014, between the Company and Barbara White (incorporated herein by reference to
Exhibit 10.15 of the Company’s Post-Effective Amendment No. 1 to Form S-1 filed with the SEC on
March 31, 2015).
10.17
Award Agreement, dated April 9, 2015, between Cystic Fibrosis Foundation Therapeutics, Inc. and the Company
(incorporated herein by reference to Exhibit 10.1 of the Company’s Quarterly Report on Form 10-Q filed with the SEC on
May 13, 2015).#
10.18
Amendment No.1 to Employment Agreement, dated April 11, 2016, between the Company and Yuval Cohen (incorporated
by reference to Exhibit 10.1 of the Company’s Current Report on Form 8-K filed with the SEC on April 15, 2016).
10.19
Amendment No.1 to Employment Agreement, dated April 11, 2016, between the Company and Mark Tepper (incorporated
by reference to Exhibit 10.2 of the Company’s Current Report on Form 8-K filed with the SEC on April 15, 2016).
10.20
Amendment No.1 to Amended and Restated Employment Agreement, dated April 11, 2016, between the Company and
Sean Moran (incorporated by reference to Exhibit 10.3 of the Company’s Current Report on Form 8-K filed with the SEC
on April 15, 2016).
10.21
Employment Agreement, dated April 11, 2016, between the Company and Barbara White (incorporated by reference to
Exhibit 10.4 of the Company’s Current Report on Form 8-K filed with the SEC on April 15, 2016).
10.22
Securities Purchase Agreement, dated June 10, 2016, between Company and each purchaser identified on the signature
pages thereto (incorporated by reference to Exhibit 10.1 of the Company’s Current Report on Form 8-K filed with the SEC
on June 10, 2016).
56
Exhibit No
Description
10.23
Consulting Agreement, dated September 20, 2016, between Company and Orchestra Medical Ventures, LLC (incorporated
by reference to Exhibit 10.1 of the Company’s Current Report on Form 8-K filed with the SEC on September 21, 2016).
10.24
Lease, dated May 30, 2014, between Corbus Pharmaceuticals, Inc. and River Ridge Limited Partnership (incorporated by
reference to Exhibit 10.2 of the Company’s Current Report on Form 8-K filed with the SEC on November 10, 2016).
10.25
10.26
10.27
10.28
10.29
First Amendment to Lease, dated August 27, 2015, between Corbus Pharmaceuticals, Inc. and River Ridge Limited
Partnership (incorporated by reference to Exhibit 10.3 of the Company’s Current Report on Form 8-K filed with the SEC
on November 10, 2016).
Second Amendment to Lease, dated March 30, 2016, between Corbus Pharmaceuticals, Inc. and River Ridge Limited
Partnership (incorporated by reference to Exhibit 10.4 of the Company’s Current Report on Form 8-K filed with the SEC
on November 10, 2016).
Third Amendment to Lease, dated September 13, 2016, between Corbus Pharmaceuticals, Inc. and River Ridge Limited
Partnership (incorporated by reference to Exhibit 10.5 of the Company’s Current Report on Form 8-K filed with the SEC
on November 10, 2016).
Controlled Equity OfferingS M Sales Agreement, dated November 23, 2016, by and between Corbus Pharmaceuticals
Holdings, Inc. and Cantor Fitzgerald & Co. (incorporated by reference to Exhibit 1.1 of the Company’s Current Report on
Form 8-K filed with the SEC on November 23, 2016.)
Securities Purchase Agreement, dated February 28, 2017, between Company and each purchaser identified on the signature
pages thereto (incorporated by reference to Exhibit 10.1 of the Company’s Current Report on Form 8-K filed with the SEC
on February 28, 2017).
21.1
List of Subsidiaries of the Company (incorporated by reference to Exhibit 21.1 of the Company’s Registration Statement on
Form S-1 filed with the SEC on September 3, 2014).
23.1
Consent of EisnerAmper LLP.*
31.1
Certification of Chief Executive Officer pursuant to Rule 13a-14(a) or Rule 15d-14(a).*
31.2
Certification of Chief Financial Officer pursuant to Rule 13a-14(a) or Rule 15d-14(a).*
32.1
Certification of Chief Executive Officer pursuant to Rule 13a-14(b) or Rule 15d-14(b).*
32.2
Certification of Chief Financial Officer pursuant to Rule 13a-14(b) or Rule 15d-14(b).*
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.*
*
#
Filed herewith.
Confidential treatment has been granted with respect to certain portions of this exhibit. Omitted portions have been submitted
separately to the SEC.
Item 16.
Form 10-K Summary.
None.
57
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
Date: March 8, 2017
CORBUS PHARMACEUTICALS HOLDINGS, INC.
/s/ YUVAL COHEN
By:
Name: Yuval Cohen
Title: Chief Executive Officer
Pursuant to the requirements of the Securities Exchange Act of 1934, this report has been signed below by the following persons
on behalf of the registrant and in the capacities and on the dates indicated.
Signature
/s/ YUVAL COHEN
Yuval Cohen
/s/ SEAN MORAN
Sean Moran
/s/ ALAN HOLMER
Alan Holmer
/s/ DAVID HOCHMAN
David Hochman
/s/ RENU GUPTA
Renu Gupta
/s/ AVERY CATLIN
Avery Catlin
Title
Date
Chief Executive Officer and Director (Principal
March 8, 2017
Executive Officer)
Chief Financial Officer (Principal Financial and
March 8, 2017
Accounting Officer)
Director
Director
Director
Director
58
March 8, 2017
March 8, 2017
March 8, 2017
March 8, 2017
INDEX TO FINANCIAL STATEMENTS
Report of Independent Registered Public Accounting Firm
Corbus Pharmaceuticals Holdings, Inc. Financial Statements- December 31, 2016:
Consolidated Balance Sheets as of December 31, 2016 and December 31, 2015
Consolidated Statements of Operations for the Years Ended December 31, 2016 and 2015
Consolidated Statements of Stockholders’ Equity for the Years Ended December 31, 2016 and 2015
Consolidated Statements of Cash Flows for the Years Ended December 31, 2016 and 2015
Notes to Consolidated Financial Statements
F-1
Page Number
F-2
F-3
F-4
F-5
F-6
F-7
REPORT OF INDEPENDENT REGISTERED PUBLIC ACCOUNTING FIRM
The Board of Directors and Stockholders
Corbus Pharmaceuticals Holdings, Inc.
We have audited the accompanying consolidated balance sheets of Corbus Pharmaceuticals Holdings, Inc. and Subsidiary (the
“Company”) as of December 31, 2016 and 2015, and the related consolidated statements of operations, stockholders’ equity, and cash
flows for each of the years then ended. The financial statements are the responsibility of the Company’s management. Our responsibility is
to express an opinion on these financial statements 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 includes examining, on a test basis, evidence
supporting the amounts and disclosures in the financial statements. An audit also includes assessing the accounting principles used and
significant estimates made by management, as well as 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 consolidated financial position
of Corbus Pharmaceuticals Holdings, Inc. and Subsidiary as of December 31, 2016 and 2015, and the consolidated results of their
operations and their cash flows for each of the years then ended, in conformity with accounting principles generally accepted in the United
States of America.
/s/ EisnerAmper LLP
Iselin, New Jersey
March 8, 2017
F-2
Corbus Pharmaceuticals Holdings, Inc.
Consolidated Balance Sheets
ASSETS
Current assets:
Cash and cash equivalents
Restricted cash
Grants receivable
Stock subscriptions receivable
Prepaid expenses
Total current assets
Restricted cash
Property and equipment, net
Total assets
LIABILITIES AND STOCKHOLDERS’ EQUITY
Current liabilities:
Notes payable
Accounts payable
Accrued expenses
Deferred revenue, current
Deferred rent, current
Total current liabilities
Deferred revenue, noncurrent
Deferred rent, noncurrent
Other liabilities
Total liabilities
Commitments and Contingencies
Stockholders’ equity
December 31,
2016
2015
$
$
14,992,257 $
150,000
1,000,000
330,413
930,261
17,402,931
50,000
435,251
17,888,182 $
$
271,757 $
3,419,921
3,256,455
1,940,195
10,263
8,898,591
—
65,724
4,632
8,968,947
12,338,275
—
—
—
376,515
12,714,790
36,375
124,138
12,875,303
162,019
1,314,377
562,279
1,591,358
—
3,630,033
260,260
—
—
3,890,293
Preferred Stock $0.0001 par value:10,000,000 shares authorized, no shares issued and
outstanding at December 31, 2016 and 2015
Common stock, $0.0001 par value; 150,000,000 shares authorized, 44,681,745 and
37,605,134 shares issued and outstanding at December 31, 2016 and 2015
Additional paid-in capital
Accumulated deficit
Total stockholders’ equity
Total liabilities and stockholders’ equity
$
—
—
4,468
42,191,256
(33,276,489)
8,919,235
17,888,182 $
3,761
22,259,063
(13,277,814)
8,985,010
12,875,303
The accompanying notes are an integral part of these Consolidated Financial Statements.
F-3
Corbus Pharmaceuticals Holdings, Inc.
Consolidated Statements of Operations
Collaboration revenue
Operating expenses:
Research and development
General and administrative
Total operating expenses
Operating loss
Other income (expense):
Interest income, net
Foreign currency exchange gain (loss)
Other income (loss), net
Net loss
Net loss per share, basic and diluted
Weighted average number of common shares outstanding, basic and diluted
For the Years Ended
December 31,
2016
2015
$
1,911,424 $
648,382
15,436,735
6,459,747
21,896,482
(19,985,058)
477
(14,094)
(13,617)
(19,998,675) $
(0.49) $
41,137,518
5,888,659
3,613,416
9,502,075
(8,853,693)
977
1,977
2,954
(8,850,739)
(0.28)
31,350,145
$
$
The accompanying notes are an integral part of these Consolidated Financial Statements.
F-4
Corbus Pharmaceuticals Holdings, Inc.
Statements of Stockholders’ Equity
Common Stock
Shares
Amount
Additional
Paid-in
Capital
Accumulated
Deficit
Total
Stockholders’
Equity
25,938,332 $
2,594 $
10,287,214 $
1,153,302
(4,427,075) $
11,615,674
1,162
10,812,963
51,128
5
5,584
(8,850,739)
5,862,733
1,153,302
10,814,125
5,589
(8,850,739)
37,605,134 $
3,761 $
22,259,063 $ (13,277,814) $
3,163,534
8,985,010
3,163,534
6,148,695
615
16,300,309
16,300,924
601,030
326,886
60
32
1,190
467,160
44,681,745 $
4,468 $
42,191,256 $ (33,276,489) $
(19,998,675)
1,250
467,192
(19,998,675)
8,919,235
Balance at December 31, 2014
Stock compensation expense
Issuance of common stock upon exercise of
warrants, net of issuance costs of $509,215
Issuance of common stock upon exercise of
stock options
Net Loss
Balance at December 31, 2015
Stock compensation expense
Issuance of common stock, net of issuance
costs of $260,179
Issuance of common stock upon exercise of
warrants
Issuance of common stock upon exercise of
stock options
Net Loss
Balance at December 31, 2016
The accompanying notes are an integral part of these Consolidated Financial Statements.
F-5
Corbus Pharmaceuticals Holdings, Inc.
Consolidated Statements of Cash Flows
Cash flows from operating activities:
Net loss
Adjustments to reconcile net loss to net cash used in operating activities:
Year Ended December 31,
2015
2016
$
(19,998,675) $
(8,850,739)
Share-based compensation expense
Depreciation and amortization
Loss (gain) on foreign exchange
Deferred rent
Changes in operating assets and liabilities:
Increase in grants receivable
Increase in prepaid expenses
Increase in accounts payable
Increase in accrued expenses
Increase in deferred revenue
Net cash used in operating activities
Cash flows from investing activities:
Purchases of property and equipment
Net cash used in investing activities
Cash flows from financing activities:
Proceeds from issuance of notes payable
Principal payments on notes payable
Proceeds from issuance on common stock
Issuance costs paid for common stock financings
Principal payments under capital lease obligations
Net cash provided by financing activities
Net increase in cash, cash equivalents, and restricted cash
Cash, cash equivalents, and restricted cash at beginning of the year
Cash, cash equivalents, and restricted cash at end of the year
Supplemental disclosure of cash flow information and non cash transactions:
Cash paid during the period for interest
Assets acquired under capital lease obligation
Purchases of property and equipment included in accounts payable or accrued expenses
Unpaid stock issuance costs
$
$
$
$
3,163,534
87,664
14,094
75,987
(1,000,000)
(553,745)
1,890,876
2,660,461
88,577
(13,571,227)
(353,032)
(353,032)
348,750
(239,012)
16,699,133
(63,830)
(3,175)
16,741,866
2,817,607
12,374,650
15,192,257 $
5,586 $
11,638 $
34,107 $
196,349 $
1,153,302
43,943
(1,977)
—
—
(105,959)
972,194
312,788
1,851,618
(4,624,830)
(114,037)
(114,037)
207,750
(190,120)
11,328,929
(509,215)
—
10,837,344
6,098,477
6,276,173
12,374,650
—
—
—
—
The accompanying notes are an integral part of these Consolidated Financial Statements.
F-6
Corbus Pharmaceuticals Holdings, Inc.
Notes to Consolidated Financial Statements
1.
NATURE OF OPERATIONS
Business
Corbus Pharmaceuticals Holdings, Inc. (“CPHI” or “the Company”) is a clinical stage pharmaceutical company, focused on the
development and commercialization of novel therapeutics to treat rare, chronic, and serious inflammatory and fibrotic diseases.
Since its inception, the Company has devoted substantially all of its efforts to business planning, research and development,
recruiting management and technical staff, acquiring operating assets and raising capital. . Our research and development activities
have included conducting pre-clinical studies, developing manufacturing methods and manufacturing of our lead drug JBT-101 for
clinical trials and conducting clinical studies in patients. The Company’s business is subject to significant risks and uncertainties and
the Company will be dependent on raising substantial additional capital before it becomes profitable and it may never achieve
profitability.
2.
LIQUIDITY
The Company anticipates operating losses to continue for the foreseeable future due to, among other things, costs related to research
funding, development of its product candidates and its preclinical and clinical programs, strategic alliances and the development of
its administrative organization. The Company has incurred recurring losses since inception and as of December 31, 2016, had an
accumulated deficit of $33,276,489. In June 2016, the Company completed a sale of shares of its common stock pursuant to the
terms of a securities purchase agreement under which the Company sold an aggregate of 5,960,000 shares of its common stock in a
registered direct offering to investors at a purchase price of $2.50 per share with net proceeds to the Company totaling
approximately $14,875,000. On February 28, 2017, the Company entered in a securities purchase agreement providing for the
issuance and sale by the Company of 3,887,815 shares of its common stock in a registered direct offering to institutional and
accredited investors at a purchase price of $7.00 per share with gross proceeds to the Company totaling $27,214,705 less estimated
issuance costs of approximately $100,000 (“February 2017 Registered Direct Offering”) (See Note 15). The Company expects the
cash on hand of $14,992,257 at December 31, 2016 together with the proceeds from the February 2017 Registered Direct Offering
and the remaining milestone payment of $500,000 from the Cystic Fibrosis Foundation Therapeutics, Inc. (“CFFT”), which the
Company expects to receive in the second quarter of 2017 (See Note 14), to be sufficient to meet our operating and capital
requirements into the fourth quarter of 2018 based on current planned expenditures.
Should the Company be unable to raise sufficient additional capital, the Company may be required to undertake cost-cutting
measures including delaying or discontinuing certain clinical activities. The Company will need to raise significant additional
capital to continue to fund the clinical trials for JBT-101. The Company may seek to sell common or preferred equity or convertible
debt securities, enter into a credit facility or another form of third-party funding, or seek other debt financing. The sale of equity and
convertible debt securities may result in dilution to the Company’s stockholders and certain of those securities may have rights
senior to those of the Company’s common shares. If the Company raises additional funds through the issuance of preferred stock,
convertible debt securities or other debt financing, these securities or other debt could contain covenants that would restrict the
Company’s operations. Any other third-party funding arrangement could require the Company to relinquish valuable rights.
The source, timing and availability of any future financing will depend principally upon market conditions, and, more specifically,
on the progress of the Company’s clinical development programs. Funding may not be available when needed, at all, or on terms
acceptable to the Company. Lack of necessary funds may require the Company, among other things, to delay, scale back or
eliminate some or all of the Company’s planned clinical trials.
3.
SIGNIFICANT ACCOUNTING POLICIES
A summary of the significant accounting policies followed by the Company in the preparation of the financial statements is as
follows:
Use of Estimates
The process of preparing financial statements in conformity with accounting principles generally accepted in the United States of
America requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities and
disclosure of assets and liabilities at the date of financial statements and the reported amounts of revenues and expenses during the
reporting period. Actual results could differ from those estimates and changes in estimates may occur. The most significant
estimates are related to stock based compensation, the value of derivative instruments and the accrual of research and clinical
obligations.
F-7
Prior to the registration of its common stock and the subsequent public listing of the common stock, the Company had granted stock
options at exercise prices not less than the fair value of its common stock as determined by the board of directors, with input from
management. The Company’s board of directors determined the estimated fair value of the common stock based on a number of
objective and subjective factors, including external market conditions affecting the biotechnology industry sector and the historic
prices at which the Company sold shares of preferred stock.
Cash, Cash Equivalents, and Restricted Cash
The Company considers only those investments which are highly liquid, readily convertible to cash, and that mature within three
months from date of purchase to be cash equivalents. Marketable investments are those with original maturities in excess of three
months. At December 31, 2016 and 2015, cash equivalents were comprised of money market funds. The Company had no
marketable investments at December 31, 2016 and 2015.
Restricted cash as of December 31, 2016 included a $150,000 collateral account for the Company’s corporate credit cards and is
classified in current assets. Additionally, as of December 31, 2016 and 2015 restricted cash included a stand-by letter of credit
issued in favor of a landlord for $50,000 and $36,375, respectively (See Note 5) and is classified in noncurrent assets.
Cash, cash equivalents, and restricted cash consists of the following:
Cash
Money market fund
Cash and cash equivalents
Restricted cash, current
Restricted cash, noncurrent
Restricted cash
$
December 31,
2016
1,127,530 $
13,864,727
14,992,257
2015
255,943
12,082,332
12,338,275
150,000
50,000
200,000
—
36,375
36,375
Total cash, cash equivalents, and restricted cash
shown in the statement of cash flows
$
15,192,257 $
12,374,650
Financial Instruments
The carrying amounts reported in the consolidated balance sheet for cash and cash equivalents and accounts payable approximate
fair value based on the short-term nature of these instruments. The carrying value of loans payable approximate their fair value due
to their market terms.
Property and Equipment
The estimated life for the Company’s property and equipment is as follows: three years for computer hardware and software and
three to five years for office furniture and equipment. The Company’s leasehold improvements and assets under capital lease are
amortized over the shorter of their useful lives or the terms of the respective leases. See Note 4 for details of property and
equipment and Note 5 for operating and capital lease commitments.
Research and Development Expenses and Collaborative Research Agreements
Costs incurred for research and development are expensed as incurred.
For amounts received under the development award received from the CFFT during 2015 and 2016 (See Note 14), the Company is
amortizing these amounts on a straight-line basis over the expected duration of the performance period of the development program
under the award, which is expected to conclude in the second quarter of 2017.
Accruals for Research and Development Expenses and Clinical Trials
As part of the process of preparing its financial statements, the Company is required to estimate its expenses resulting from its
obligations under contracts with vendors, clinical research organizations and consultants and under clinical site agreements in
connection with conducting clinical trials. The financial terms of these contracts are subject to negotiations, which vary from
contract to contract and may result in payment terms that do not match the periods over which materials or services are provided
under such contracts. The Company’s objective is to reflect the appropriate expenses in its financial statements by matching those
expenses with the period in which services are performed and efforts are expended. The Company accounts for these expenses
according to the timing of various aspects of the expenses. The Company determines accrual estimates through financial models
taking into account discussion with applicable personnel and outside service providers as to the progress of clinical trials, or the
services completed. During the course of a clinical trial, the Company adjusts its clinical expense recognition if actual results differ
from its estimates. The Company makes estimates of its accrued expenses as of each balance sheet date based on the facts and
circumstances known to it at that time. The Company’s clinical trial accruals are dependent upon the timely and accurate reporting
of contract research organizations and other third-party vendors. Although the Company does not expect its estimates to be
materially different from amounts actually incurred, its understanding of the status and timing of services performed relative to the
actual status and timing of services performed may vary and may result in it reporting amounts that are too high or too low for any
particular period. For the years ended December 31, 2016 and 2015, there were no material adjustments to the Company’s prior
period estimates of accrued expenses for clinical trials.
F-8
Concentrations of Credit Risk
The Company has no significant off-balance-sheet concentration of credit risk such as foreign exchange contracts, option contracts
or other hedging arrangements. The Company may from time to time have cash in banks in excess of Federal Deposit Insurance
Corporation insurance limits.
Segment Information
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, or decision making group, in making decisions regarding resource allocation
and assessing performance. To date, the Company has viewed its operations and manages its business as principally one operating
segment, which is developing and commercializing therapeutics to treat rare life-threatening inflammatory fibrotic diseases. As of
December 31, 2016 and 2015, all of the Company’s assets were located in the United States.
Income Taxes
For federal and state income taxes, deferred tax assets and liabilities are recognized based upon temporary differences between the
financial statement and the tax basis of assets and liabilities. Deferred income taxes are based upon prescribed rates and enacted
laws applicable to periods in which differences are expected to reverse. A valuation allowance is recorded to reduce a net deferred
tax benefit when it is more likely than not that the tax benefit from the deferred tax assets will not be realized. Accordingly, given
the cumulative losses since inception, the Company has provided a valuation allowance equal to 100% of the tax benefit in order to
eliminate the deferred tax assets amounts. Tax positions taken or expected to be taken in the course of preparing the Company’s tax
returns are required to be evaluated to determine whether the tax positions are “more-likely-than-not” of being sustained by the
applicable tax authority.
Tax positions not deemed to meet a more-likely-than-not threshold, as well as accrued interest and penalties, if any, would be
recorded as a tax expense in the current year. There were no uncertain tax positions that require accrual or disclosure to the
financial statements as of December 31, 2016 or 2015.
Impairment of Long-lived Assets
The Company continually monitors events and changes in circumstances that could indicate that carrying amounts of long-lived
assets may not be recoverable. An impairment loss is recognized when expected cash flows are less than an asset’s carrying value.
Accordingly, when indicators of impairment are present, the Company evaluates the carrying value of such assets in relation to the
operating performance and future undiscounted cash flows of the underlying assets. The Company’s policy is to record an
impairment loss when it is determined that the carrying value of the asset may not be recoverable. No impairment charges were
recorded for the years ended December 31, 2016 and 2015.
Share-based Payments
The Company recognizes compensation costs resulting from the issuance of stock-based awards to employees, non-employees and
directors as an expense in the statement of operations over the service period based on a measurement of fair value for each stock-
based award. The fair value of each option grant is estimated as of the date of grant using the Black-Scholes option-pricing model.
The fair value is amortized as compensation cost on a straight-line basis over the requisite service period of the awards, which is
generally the vesting period. Stock options granted to non-employee consultants are revalued at the end of each reporting period
until vested and the changes in their fair value are recorded as adjustments to expense over the related vesting period.
F-9
Net Loss Per Common Share
Basic net loss per share of the Company’s common stock has been computed by dividing net loss by the weighted average number
of shares outstanding during the period. Diluted net income per share of the Company’s common stock has been computed by
dividing net income by the weighted average number of shares outstanding plus the dilutive effect, if any, of outstanding stock
options, warrants and convertible securities. Diluted net loss per share of the Company’s common stock has been computed by
dividing the net loss for the period by the weighted average number of shares of the Company’s common stock outstanding during
such period. For years in which there is a net loss, options, warrants and convertible securities are anti-dilutive and therefore
excluded from diluted loss per share calculations. The following table sets forth the computation of basic and diluted earnings per
share for the years ended December 31, 2016 and 2015:
Basic and diluted net loss per share of common stock:
Net loss
Weighted average shares of common stock outstanding
Net loss per share of common stock-basic and diluted
Years Ended December 31,
2015
2016
$
$
(19,998,675) $
41,137,518
(0.49) $
(8,850,739)
31,350,145
(0.28)
The following potentially dilutive securities outstanding at December 31, 2016 and 2015 have been excluded from the computation
of dilutive weighted average shares outstanding as the inclusion would be antidilutive.
Warrants
Stock options
Recent Accounting Pronouncements
Revenue Recognition
December 31,
2016
1,288,500
6,610,179
7,898,679
2015
1,969,250
3,982,065
5,951,315
In May 2014, the Financial Accounting Standards Board (the “FASB”) issued guidance codified in Accounting Standards
Codification (ASC) 606, Revenue Recognition — Revenue from Contracts with Customers (“ASC 606”) which amends the guidance
in former ASC 605, Revenue Recognition, and is effective for public companies for annual and interim periods beginning after
December 15, 2017. The Company plans to adopt the standard in the first quarter of 2018 and believes that its adoption may have an
impact on the Company’s consolidated financial statements. Specifically, the new standard differs from the current accounting
standard in many respects, such as in the accounting for variable consideration received, including milestone payments or
contingent payments. Under the Company’s current accounting policy, milestone payments are recognized as revenue in the period
that the payment-triggering event occurred or was achieved (See Note 8). ASC 606, however, may require the Company to
recognize these payments before the payment-triggering event is completely achieved, subject to management’s assessment of
whether it is probable that the triggering event will be achieved and that a significant reversal in the amount of cumulative revenue
recognized will not occur when the uncertainty associated with the variable consideration is subsequently resolved.
Accounting for Share-Based Payments
In June 2014, the FASB issued Accounting Standards Update (“ASU”) No. 2014-12, Accounting for Share-Based Payments When
the Terms of an Award Provide That a Performance Target Could Be Achieved after the Requisite Service Period (a consensus of
the FASB Emerging Issues Task Force) (“ASU 2014-12”). ASU 2014-12 clarifies that entities should treat performance targets that
can be met after the requisite service period of a share-based payment award as performance conditions that affect vesting.
Therefore, an entity would not record compensation expense (measured as of the grant date without taking into account the effect of
the performance target) related to an award for which transfer to the employee is contingent on the entity’s satisfaction of a
performance target until it becomes probable that the performance target will be met. There are no new disclosures required under
ASU 2014-12. ASU 2014-12 is effective for fiscal years, and interim periods within those years, beginning after December 15,
2015. The Company’s adoption of ASU 2014-12 in the first quarter of 2016 had no impact on its financial position, results of
operations, cash flows, or disclosures.
F-10
Reporting of Going-Concern Uncertainties
In August 2014, the FASB issued ASU No. 2014-15, Presentation of Financial Statements—Going Concern (“ASU 2014-15),
which states management should evaluate whether there are conditions or events, considered in the aggregate, that raise a
substantial doubt about the entity’s ability to continue as a going concern within one year after the date that the financial statements
are issued. Management’s evaluation should be based on relevant conditions and events that are known and likely to occur at the
date that the financial statements are issued. ASU 2014-15 was effective for the annual period ending after December 15, 2016 and
its adoption did not have any impact on the Company’s financial position, results of operations, cash flows, or disclosures.
Accounting for Leases
In February 2016, the FASB issued ASU No. 2016-02, Leases (Topic 842) (“ASU 2016-02”). Under ASU 2016-02, a lessee will be
required to recognize assets and liabilities for leases with lease terms of more than 12 months. Consistent with current GAAP, the
recognition, measurement, and presentation of expenses and cash flows arising from a lease by a lessee primarily will depend on its
classification as a finance or operating lease. However, unlike current GAAP, which requires only capital leases to be recognized on
the balance sheet, ASU 2016-02 will require both types of leases to be recognized on the balance sheet. ASU 2016-02 will take
effect for fiscal years, and interim periods within those fiscal years, beginning after December 15, 2018, with early application
permitted. Management has not yet determined if it will adopt ASU 2016-02 earlier than the required adoption date. The adoption
of ASU 2016-02 will have an impact on the Company’s financial position, results of operations, cash flows, and disclosures as the
Company has an operating lease commitment for office space as of December 31, 2016 in the amount of $1,030,118 (see Note 5)
for which ASU 2016-02 would apply.
Employee Share-Based Payment Accounting
On March 30, 2016, the FASB issued ASU No. 2016-09, Compensation—Stock Compensation (Topic 718): Improvements to
Employee Share-Based Payment Accounting (“ASU 2016-09”). ASU 2016-09 simplifies several aspects of the accounting for
employee share-based payment transactions including the accounting for income taxes, forfeitures, and statutory tax withholding
requirements, as well as classification in the statement of cash flows. ASU 2016-09 will take effect for fiscal years, and interim
periods within those fiscal years, beginning after December 15, 2017, with early application permitted. Management does not expect
the adoption of ASU 2016-09 to have a material impact on the Company’s consolidated financial statements, although there may be
additional disclosures upon adoption.
Restricted Cash Presentation
On November 17, 2016, the FASB issued ASU No. 2016-18, Restricted Cash (a consensus of the FASB Emerging Issues Task
Force) (“ASU 2016-18”), which addresses classification and presentation of changes in restricted cash on the statement of cash
flows. ASU 2016-18 requires an entity’s reconciliation of the beginning-of-period and end-of-period total amounts shown on the
statement of cash flows to include in cash and cash equivalents amounts generally described as restricted cash and restricted cash
equivalents. ASU 2016-18 is effective for public business entities for annual and interim periods in fiscal years beginning after
December 15, 2017. Early adoption is permitted, including adoption in an interim period. If an entity early adopts the amendments
in an interim period, adjustments should be reflected at the beginning of the fiscal year that includes that interim period. The
Company early adopted ASU 2016-18 for the fiscal year ended December 31, 2016 using a retrospective transition method for each
period presented.
4.
PROPERTY AND EQUIPMENT
Property and Equipment consists of the following:
Computer hardware and software
Office furniture and equipment
Leasehold improvements
Construction in progress
Property and equipment, gross
Less: accumulated depreciation
Property and equipment, net
December 31,
2016
2015
$
$
96,131 $
259,138
188,219
—
543,488
(108,237)
435,251 $
40,202
35,209
19,310
83,765
178,486
(54,348)
124,138
Depreciation expense was approximately $88,000 and $44,000 for the years ended December 31, 2016 and 2015, respectively. At
December 31, 2015, construction in progress consisted of purchased property and equipment not placed in service until the
Company’s relocation into 6,326 square feet of office space in January 2016 (See Note 5). In connection with this relocation, the
Company wrote off approximately $34,482 of fully depreciated property and equipment.
On December 30, 2015, the Company entered into a lease agreement for a copier machine. The cost of the machine was
approximately $12,000 and is included in office furniture and equipment category in the table above. The lease payments
commenced when the machine was placed in service in January 2016. The machine is being amortized over the life of the lease,
which is for a three-year term and includes a bargain purchase option at the end of the term. See Note 5 for details of this capital
lease commitment.
F-11
5.
COMMITMENTS AND CONTINGENCIES
Operating Lease Commitment
On May 30, 2014, the Company entered into a commercial lease for 2,387 square feet of office space in Norwood, MA. The lease
commenced on July 1, 2014, had a three-year term, and required a standby letter of credit of $13,728 payable in favor of the
landlord. In August 2015, the lease was amended for the relocation of the Company into 6,326 square feet of office space within
the existing building (“August 2015 Amendment”). In January 2016, the Company began occupying the space under this lease
amendment, which was for a five-year term. The amendment also required an increase in the standby letter of credit to $36,375 (See
Note 3).
In September 2016, the lease was amended for the Company’s expansion into an additional 4,088 square feet of office space within
the existing building (“September 2016 Amendment”). The Company began occupying this space in early November 2016 and the
final lease payment per the terms of the September 2016 Amendment is due in January 2021. Additionally, the September 2016
Amendment requires an increase in the standby letter of credit to $50,000 (See Note 3).
The Company records the total rent payable during the lease term on a straight-line basis over the term of the lease and records the
difference between the rents paid and the straight-line rent as deferred rent, which is classified in deferred rent, current and deferred
rent, noncurrent in the Company’s balance sheet as of December 31, 2016.
Pursuant to the terms of the Company’s non-cancelable lease agreements in effect at December 31, 2016, the future minimum rent
commitments are as follows:
2017
2018
2019
2020
2021
Thereafter
Total
$
$
244,295
249,502
254,709
259,916
21,696
—
1,030,118
Total rent expense for the years ended December 31, 2016 and 2015 was $229,705 and $55,496, respectively.
Capital Lease Commitment
On December 30, 2015, the Company entered into a capital lease agreement for a copier machine. The lease payments commenced
when the machine was placed in service in January 2016. The lease is for a three-year term and includes a bargain purchase option
at the end of the term. In the accompanying balance sheet as of December 31, 2016, the current portion of this capital lease
obligation is classified in accrued expenses and the long-term portion of the capital lease obligation is classified in other long-term
liabilities. Pursuant to the terms of this capital lease agreement, the future minimum capital lease commitments are as follows as of
December 31, 2016:
2017
2018
2019
Total future minimum lease payments
Less: interest
Future capital lease obligations
Less: current portion
Long-term portion
$
$
4,543
4,543
379
9,465
(1,002)
8,463
(3,831)
4,632
Interest expense for this capital lease obligation for the years ended December 31, 2016 and 2015 was $1,286 and $0, respectively.
6.
NOTES PAYABLE
In November 2015, the Company entered into a loan agreement with a financing company for $207,750 to finance one of the
Company’s insurance policies. The terms of the loan stipulated equal monthly payments of principal and interest payments of
$23,397 over a nine month period. Interest on this loan was accrued at an annual rate of 3.25%. This loan was fully repaid in July
2016.
F-12
In October 2016, the Company entered into a loan agreement with a financing company for $348,750 to finance one of the
Company’s insurance policies. The terms of the loan stipulate equal monthly payments of principal and interest payments of
$39,114 over a nine-month period. Interest accrues on this loan at an annual rate of 2.25%.
Interest expense for notes payable for the years ended December 31, 2016 and 2015 totaled $3,115 and $2,440, respectively.
Notes payable consisted of the following:
Notes payable
Less: current portion
7.
ACCRUED EXPENSES
Accrued expenses consisted of the following:
Accrued clinical operations and trials costs
Accrued product development costs
Accrued compensation
Accrued other
Total
8.
DEFERRED REVENUE
December 31,
2016
2015
$
$
271,757 $
(271,757)
— $
162,019
(162,019)
—
December 31,
2016
2015
$
$
1,647,490 $
713,426
778,250
117,289
3,256,455 $
365,188
152,018
—
45,073
562,279
In May 2015, the Company received $1,250,000 upon signing the CFFT award agreement and in the fourth quarter of 2015, the
Company received $1,250,000 from the CFFT upon the achievement of a milestone for dosing the first patient. In August 2016, the
Company received a third payment from the CFFT for achieving a milestone in July 2016 related to dosing the median clinical trial
patient as per the terms of the Award in the amount of $1,000,000. In January 2017, the Company received a fourth payment from
the CFFT for achieving a milestone in December 2016 related to completing the final visit for the final patient as per the terms of
the Award in the amount of $1,000,000 (See Note 3 and Note 14), which was billed by the Company to CFFT in December 2016
and was classified in grants receivable as of December 31, 2016. The Company recorded these four milestone payments received
from the CFFT totaling $4,500,000 as deferred revenue and is amortizing the deferred revenue and recognizing revenue on a
straight-line basis over the performance period for the development program under the Award, which is expected to conclude
during the second quarter of 2017. The Company recorded $1,911,424 and $648,382 of revenue during the year ended December
31, 2016 and 2015, respectively. Deferred revenue consists of the following:
Deferred revenue
Less: current portion
Long term portion
9.
INCOME TAXES
$
December 31,
2016
1,940,195 $
(1,940,195)
—
2015
1,851,618
(1,591,358)
260,260
No provision or benefit for federal or state income taxes has been recorded, as the Company has incurred a net loss for all of the
periods presented, and the Company has provided a full valuation allowance against its deferred tax assets.
At December 31, 2016, and 2015, the Company had federal and Massachusetts net operating loss carryforwards of approximately
$55,217,000 and $22,416,000, respectively, of which federal carryforwards will expire in varying amounts beginning in 2029. As of
December 31, 2016 and 2015, approximately $1,432,000 and $762,000, respectively, of Federal and Massachusetts net operating
loss carryforwards are from excess stock-based compensation, which are not included in the deferred tax asset as of December 31,
2016 and 2015. Massachusetts net operating losses began to expire in 2014. Utilization of net operating losses may be subject to
substantial annual limitations due to the “change in ownership” provisions of the Internal Revenue Code, and similar state
provisions. The annual limitations may result in the expiration of net operating losses before utilization. The Company has not yet
conducted a study to determine if any such changes have occurred that could limit our ability to use the net operating losses and tax
credit carryforwards. The Company also had research and development tax credit carryforwards at December 31, 2016 and 2015 of
approximately $986,000 and $441,000, respectively.
F-13
Significant components of the Company’s net deferred tax asset are as follows:
NOL carryforward
Tax credits
Stock based compensation
Accrued expenses
Other temporary differences
Subtotal
Valuation allowance
Net deferred tax asset
December 31,
2016
10,860,828 $
673,690
1,177,650
302,943
225,214
13,240,325
(13,240,325)
— $
2015
4,505,965
406,888
453,906
—
12,581
5,379,340
(5,379,340)
—
$
$
The Company has maintained a full valuation allowance against its deferred tax assets in all periods presented. A valuation
allowance is required to be recorded when it is more likely than not that some portion or all of the net deferred tax assets will not be
realized. Since the Company cannot determine that it is more likely than not that it will generate taxable income, and thereby
realize the net deferred tax assets, a full valuation allowance has been provided. The valuation allowance increased by $7,860,985
and $3,615,002 in 2016 and 2015, respectively, due to the increase in deferred tax assets, primarily due to net operating loss
carryforwards. The Company has no uncertain tax positions at December 31, 2016 and 2015 that would affect its effective tax rate.
The Company does not anticipate a significant change in the amount of uncertain tax positions over the next twelve months. Since
the Company is in a loss carryforward position, the Company is generally subject to U.S. federal and state income tax examinations
by tax authorities for all years for which a loss carryforward is available.
Income tax benefits computed using the federal statutory income tax rate differs from the Company’s effective tax rate primarily
due to the following:
Tax provision at statutory rate
State taxes, net of federal benefit
Permanent differences
Tax credits
Other
Decrease in valuation reserve
Total
10.
COMMON STOCK
December 31,
2016
2015
34.00%
4.76%
-0.65%
1.33%
-0.13%
-39.31%
0.00%
34.00%
4.76%
-0.62%
2.67%
0.04%
-40.85%
0.00%
The Company has authorized 150,000,000 shares of common stock, $0.0001 par value per share, of which 44,681,745 shares and
37,605,134 shares were issued and outstanding as of December 31, 2016 and 2015, respectively.
In June 2016, the Company completed a sale of shares of its common stock pursuant to the terms of a securities purchase agreement
under which the Company sold an aggregate of 5,960,000 shares of its common stock in a registered direct offering to investors at a
purchase price of $2.50 per share with gross proceeds to the Company totaling approximately $14,900,000 less issuance costs of
$25,222. On February 28, 2017, the Company entered in a securities purchase agreement providing for the issuance and sale by the
Company of 3,887,815 shares of its common stock in a registered direct offering to institutional and accredited investors at a
purchase price of $7.00 per share with gross proceeds to the Company totaling $27,214,705 less estimated issuance costs of
approximately $100,000 (See Note 15).
In November 2016, the Company entered into a sales agreement with Cantor Fitzgerald under which the Company may direct
Cantor Fitzgerald as its placement agent to sell common stock under an “At the Market Offering” (“Sales Agreement”). Sales of
common stock under the Sales Agreement are made pursuant to an effective registration statement for an aggregate offering of up to
$35 million. In 2016, we sold 188,695 shares of our common stock under the Sales Agreement at an average selling price of
approximately $8.54 per share (net of 3% commission paid to Cantor Fitzgerald) which resulted in proceeds of approximately
$1,621,182 and net proceeds of approximately $1,426,145 net of incurred issuance costs. Approximately $330,413 of these
proceeds was classified in stock subscriptions receivable as of December 31, 2016 because the Company did not receive these
proceeds until January 2017.
During the year ended December 31, 2016, the Company issued 927,916 shares of common stock upon the exercise of stock options
and warrants to purchase common stock and the Company received net proceeds of $468,442 from these exercises.
F-14
11.
STOCK OPTIONS
In April 2014, the Company adopted the Corbus Pharmaceuticals Holdings, Inc. 2014 Equity Incentive Plan (the “2014 Plan”).
Pursuant to the 2014 Plan, the Company’s Board of Directors may grant incentive and nonqualified stock options and restricted
stock to employees, officers, directors, consultants and advisors. On January 1, 2016, pursuant to an annual evergreen provision
contained in the 2014 Plan, the number of shares reserved for future grants was increased by 1,250,000 shares, respectively. As of
December 31, 2016, there was a total of 9,916,017 shares reserved for issuance under the 2014 Plan and there were 2,840,133 shares
available for future grants. Options issued under the 2014 Plan are exercisable for up to 10 years from the date of issuance.
Pursuant to the terms of an annual evergreen provision in the 2014 Plan, the number of shares of common stock available for
issuance under the 2014 Plan shall automatically increase on January 1 of each year by at least seven percent (7%) of the total
number of shares of common stock outstanding on December 31st of the preceding calendar year, or, pursuant to the terms of the
2014 Plan, in any year, the Board of Directors may determine that such increase will provide for a lesser number of shares. In
accordance with the terms of the 2014 Plan, effective as of January 1, 2017, the number of shares of common stock available for
issuance under the 2014 Plan increased by 3,127,722 shares, which was seven percent (7%) of the outstanding shares of common
stock on December 31, 2016. As of January 1, 2017, the 2014 Plan had a total reserve of 13,043,739 shares and there were
5,967,855 shares available for future grants.
Share-based Compensation
For stock options issued and outstanding for the years ended December 31, 2016 and 2015, the Company recorded non-cash, stock-
based compensation expense of $3,163,534 and $1,153,302, respectively, net of estimated forfeitures.
The fair value of each option award is estimated on the date of grant using the Black-Scholes option pricing model that uses the
assumptions noted in the following table. Due to its limited operating history and limited number of sales of its common stock, the
Company estimated its volatility in consideration of a number of factors, including the volatility of comparable public companies
and, commencing in 2015, the Company also included the volatility of its own common stock. The Company uses historical data, as
well as subsequent events occurring prior to the issuance of the financial statements, to estimate option exercises and employee
terminations within the valuation model. The expected term of options granted under the 2014 Plan, all of which qualify as “plain
vanilla” per SEC Staff Accounting Bulletin 107, is based on the average of the 6.25 years. For non-employee options, the expected
term is the contractual term. The risk-free rate is based on the yield of a U.S. Treasury security with a term consistent with the
option.
The weighted average assumptions used principally in determining the fair value of options granted were as follows:
Risk free interest rate
Expected dividend yield
Expected term in years
Expected volatility
Estimated forfeiture rate
2016
2015
1.70%
0%
6.66
90.39%
5.00%
1.85%
0%
6.73
90.68%
4.83%
F-15
A summary of option activity for years ended December 31, 2016 and 2015 is presented below:
Options
Outstanding at December 31, 2014
Granted
Exercised
Forfeited
Outstanding at December 31, 2015
Granted
Exercised
Forfeited
Outstanding at December 31, 2016
Vested at December 31, 2016
Shares
3,556,691 $
600,002 $
(51,128) $
(123,500)
3,982,065 $
3,020,000 $
(326,886) $
(65,000) $
6,610,179 $
2,782,551 $
Weighted
Average
Exercise
Price
Weighted
Average
Remaining
Contractual
Term in
Years
Intrinsic
Value
0.83
2.10
0.11
1.00
1.03
4.42
1.43
2.44
2.54
1.10
8.22 $
7.21 $
39,192,254
20,439,089
The weighted average grant-date fair value of options granted during the years ended December 31, 2016 and 2015 was $3.81 and
$1.41 per share, respectively. The aggregate intrinsic value of options exercised during the years ended December 31, 2016 and
2015 was approximately $1,004,321 and $152,531, respectively. The total fair value of options that were vested as of December 31,
2016 was $2,561,877. As of December 31, 2016 there was approximately $9,232,486 of total unrecognized compensation expense,
related to non-vested share-based option compensation arrangements. The unrecognized compensation expense is estimated to be
recognized over a period of 3.03 years at December 31, 2016.
12.
WARRANTS
At December 31, 2016, there were warrants outstanding to purchase 1,288,500 shares of common stock with a weighted average
exercise price of $1.00 and a weighted average remaining life of 2.41 years. During the year ended December 31, 2016, warrants to
purchase 679,500 shares of common stock were exercised on a cashless basis resulting in the issuance of 599,780 shares and 1,250
shares of common stock were exercised on a for cash basis. During the year ended December 31, 2015, warrants to purchase
11,615,674 shares of common stock were exercised for net proceeds of approximately $10,814,125 which included warrants to
purchase 371,250 shares of common stock that were exercised in cashless exercises, in accordance with the warrant agreements,
resulting in the issuance of 255,724 shares. There were no warrants issued or cancelled during the year ended December 31, 2016 or
2015.
13.
RELATED PARTY TRANSACTIONS
In connection with the formation of Corbus Pharmaceutical Holdings, Inc. in December 2013, certain affiliates of Aegis Capital
Corp. (the “Placement Agent”) and certain other parties not affiliated with us or the Placement Agent subscribed for an aggregate of
6,000,000 shares of common stock for which they paid an aggregate of $120,000 ($0.02 per share), including David Hochman, one
of our directors who purchased 450,000 shares and whose family trust purchased 90,000 shares of common stock.
Following the Initial Closing of the 2014 Private Placement, which took place on April 11, 2014, the Placement Agent had a right to
appoint one member of the Company’s board of directors for a two-year term (the “Aegis Nominee”). David Hochman was
appointed as the Aegis Nominee.
On June 21, 2014, the Company entered into a consulting agreement with Orchestra Medical Ventures, LLC (“Orchestra”), of
which David Hochman is Managing Partner. The agreement provided that Orchestra would render a variety of consulting and
advisory services relating principally to identifying and evaluating strategic relationships, licensing opportunities, and business
strategies. Orchestra was compensated at a rate of $5,000 per month for twelve months, payable quarterly in advance. During the
year ended December 31, 2015, the Company paid Orchestra $15,000. The consulting agreement expired on April 11, 2015 and the
Company was not obligated to make future payments. On September 20, 2016, the Company entered into a new consulting
agreement with Orchestra for similar services as provided under the previous agreement (the “2016 Consulting Agreement”). The
term of the 2016 Consulting Agreement commenced on September 20, 2016 and will expire on March 20, 2017, subject to renewal
upon mutual agreement of the parties. Pursuant to the terms of the 2016 Consulting Agreement, the Company will pay to Orchestra
cash compensation in an aggregate amount of $100,000, payable in equal monthly installments over the six-month term of the 2016
Consulting Agreement. In connection with this agreement, the Company granted an equity incentive award to Orchestra consisting
of options to purchase 50,000 shares of the Company’s common stock at an exercise price of $7.14 per share pursuant to the
Company’s 2014 Equity Compensation Plan, which amounted to stock-based compensation expense of approximately $222,000
during the year ended December 31, 2016.
F-16
The Company entered into a non-exclusive financial advisory agreement with Aegis under which the Company paid Aegis
$200,000 upon the execution of the agreement, which commenced on September 1, 2015 and expired on November 30, 2015. The
Company also paid Aegis a warrant solicitation fee of $309,215 in connection with the exercise of warrants that were called and
exercised in the third quarter of 2015.
One of the former members of the Company’s scientific advisory board was considered an affiliate of the Company as he owned
more than 10% of the Company’s common stock as of December 31, 2015. This individual’s ownership of the Company’s common
stock was less than 10% as of December 31, 2016.
14.
DEVELOPMENT AWARD
On April 20, 2015, the Company entered into an award agreement with the CFFT, a non-profit drug discovery and development
affiliate of the Cystic Fibrosis Foundation, pursuant to which it received a development award (the “Award”) for up to $5 million in
funding. The funding from the Award is supporting a first-in-patient Phase 2 clinical trial of the Company’s oral anti-inflammatory
drug JBT-101 in adults with cystic fibrosis (“CF”). The Company has billed and received a total of $4.5 million in payments since
the inception of the Award as outlined below. The payments received under the award have been recorded as deferred revenue and
are being amortized on a straight-line basis over the expected duration of the performance period under the Award, which is
expected to conclude in the second quarter of 2017.
Upon the execution of the Award agreement, the Company received a payment of $1,250,000 in May 2015. In November 2015, the
Company received a second payment of $1,250,000 upon the achievement of a milestone for dosing the first patient. In August
2016, the Company received a third payment from the CFFT in the amount of $1,000,000 for achieving a milestone in July 2016
related to dosing the median clinical trial patient. In January 2017, the Company received a fourth payment from the CFFT in the
amount of $1,000,000 for achieving a milestone in December 2016 related to completing the final visit for the final patient), which
was billed by the Company to CFFT in December 2016 and was classified in grants receivable as of December 31, 2016. The
Company expects that the last milestone payment of $500,000 under the Award will be recorded in the second quarter of 2017 upon
the achievement of the final milestone related to the Phase 2 CF clinical trial, as set forth in the Award agreement.
Pursuant to the terms of the Award agreement, the Company is obligated to make royalty payments to CFFT contingent upon
commercialization of JBT-101 in the Field of Use (as defined in the Award agreement) including a royalty payment equal to five
times the amount the Company receives under the Award agreement, up to $25 million, payable in three equal annual installments
following the first commercial sale of JBT-101, the first of which is due within 90 days following the first commercial sale of JBT-
101. The Company is also obligated to make a royalty payment to CFFT equal to the amount the Company receives under the
Award agreement, up to $5 million, due in the first calendar year in which the aggregate cumulative net sales of JBT-101 in the
Field of Use exceed $500 million. Lastly, the Company is obligated to make royalty payment(s) to CFFT of up to approximately
$15 million if the Company transfers, sells or licenses JBT-101 in the Field of Use other than for certain clinical or development
purposes, or if the Company enters into a change of control transaction, with such payment(s) to be credited against the royalty
payments due upon commercialization. The Field of Use is defined in the Award as the treatment in humans of CF, asbestosis,
bronchiectasis, byssinosis, chronic bronchitis/COPD hypersensitivity pneumonitis, pneumoconiosis, primary ciliary dyskinesis,
sarcoidosis and silicosis. Either CFFT or the Company may terminate the agreement for cause, which includes the Company’s
material failure to achieve certain commercialization and development milestones. The Company’s payment obligations survive the
termination of the Award agreement.
15.
SUBSEQUENT EVENTS
Evergreen Provision
Pursuant to the terms of an annual evergreen provision in the 2014 Plan, the number of shares of common stock available for
issuance under the 2014 Plan shall automatically increase on January 1 of each year by at least seven percent (7%) of the total
number of shares of common stock outstanding on December 31st of the preceding calendar year, or, pursuant to the terms of the
2014 Plan, in any year, the Board of Directors may determine that such increase will provide for a lesser number of shares. In
accordance with the terms of the 2014 Plan, effective as of January 1, 2017, the number of shares of common stock available for
issuance under the 2014 Plan increased by 3,127,722 shares, such amount being seven percent (7%) of the outstanding shares of
common stock on December 31, 2016. As of January 1, 2017, the 2014 Plan had a total reserve of 13,043,739 shares and there were
5,967,855 shares available for future grants.
Registered Direct Offering
On February 28, 2017, the Company entered in a securities purchase agreement with institutional and accredited investors providing
for the issuance and sale by the Company of 3,887,815 shares of its common stock in a registered direct offering to investors at a
purchase price of $7.00 per share with gross proceeds to the Company totaling approximately $27,214,705 less estimated issuance
costs of approximately $100,000.
F-17
CONSENT OF INDEPENDENT REGISTERED PUBLIC ACCOUNTING FIRM
We consent to the incorporation by reference in the Registration Statements of Corbus Pharmaceuticals Holdings, Inc. and Subsidiary on
Form S-3 (No. 333-207936) and Form S-8 (Nos. 333-200350 and 333-201898 and 333-210428) of our report dated March 8, 2017, on our
audits of the consolidated financial statements as of December 31, 2016 and 2015 and for each of the years then ended, which report is
included in this Annual Report on Form 10-K to be filed on or about March 8, 2017.
Exhibit 23.1
/s/ EISNERAMPER LLP
Iselin, New Jersey
March 8, 2017
I, Yuval Cohen, certify that:
CERTIFICATION OF CHIEF EXECUTIVE OFFICER PURSUANT
TO SECTION 302 OF THE SARBANES-OXLEY ACT OF 2002
Exhibit 31.1
1
2.
3
4
I have reviewed this annual report on Form 10-K for the period ended December 31, 2016 of Corbus Pharmaceuticals Holdings,
Inc.;
Based on my knowledge, this report does not contain any untrue statement of a material fact or omit to state a material fact
necessary to make the statements made, in light of the circumstances under which such statements were made, not misleading with
respect to the period covered by this report;
Based on my knowledge, the financial statements, and other financial information included in this report, fairly present in all
material respects the financial condition, results of operations and cash flows of the registrant as of, and for, the periods presented
in this report;
The registrant’s other certifying officer(s) and I are responsible for establishing and maintaining disclosure controls and procedures
(as defined in Exchange Act Rules 13a-15(e) and 15d-15(e)) and internal control over financing reporting (as defined in Exchange
Act Rules 13a-15(f) and 15d-15(f)) for the registrant and have:
a) Designed such disclosure controls and procedures, or caused such disclosure controls and procedures to be designed under our
supervision, to ensure that material information relating to the registrant, including its consolidated subsidiaries, is made
known to us by others within those entities, particularly during the period in which this report is being prepared;
b) Designed such internal control over financial reporting, or caused such internal control over financial reporting to be designed
under our supervision, to provide reasonable assurance regarding the reliability of financial reporting and the preparation of
financial statements for external purposes in accordance with generally accepted accounting principles;
c) Evaluated the effectiveness of the registrant’s disclosure controls and procedures and presented in this report our conclusions
about the effectiveness of the disclosure controls and procedures, as of the end of the period covered by this report based on
such evaluation; and
d) Disclosed in this report any change in the registrant’s internal control over financial reporting that occurred during the
registrant’s most recent fiscal quarter (the registrant’s fourth fiscal quarter in the case of an annual report) that has materially
affected, or is reasonably likely to materially affect, the registrant’s internal control over financial reporting; and
5.
The registrant’s other certifying officer(s) and I have disclosed, based on our most recent evaluation of internal control over
financial reporting, to the registrant’s auditors and the audit committee of the registrant’s board of directors (or persons performing
the equivalent functions):
a) All significant deficiencies and material weaknesses in the design or operation of internal control over financial reporting
which are reasonably likely to adversely affect the registrant’s ability to record, process, summarize and report financial
information; and
b) Any fraud, whether or not material, that involves management or other employees who have a significant role in the registrant’s
internal control over financial reporting.
Date: March 8, 2017
/s/ Yuval Cohen
Yuval Cohen
Chief Executive Officer
(Principal Executive Officer)
CERTIFICATION OF CHIEF FINANCIAL OFFICER PURSUANT
TO SECTION 302 OF THE SARBANES-OXLEY ACT OF 2002
I, Sean M. Moran, certify that:
Exhibits 31.2
1.
2.
3.
4.
I have reviewed this annual report on Form 10-K for the period ended December 31, 2016 of Corbus Pharmaceuticals Holdings,
Inc.;
Based on my knowledge, this report does not contain any untrue statement of a material fact or omit to state a material fact
necessary to make the statements made, in light of the circumstances under which such statements were made, not misleading with
respect to the period covered by this report;
Based on my knowledge, the financial statements, and other financial information included in this report, fairly present in all
material respects the financial condition, results of operations and cash flows of the registrant as of, and for, the periods presented
in this report;
The registrant’s other certifying officer(s) and I are responsible for establishing and maintaining disclosure controls and
procedures (as defined in Exchange Act Rules 13a-15(e) and 15d-15(e)) and internal control over financing reporting (as defined in
Exchange Act Rules 13a-15(f) and 15d-15(f)) for the registrant and have:
a. Designed such disclosure controls and procedures, or caused such disclosure controls and procedures to be designed under our
supervision, to ensure that material information relating to the registrant, including its consolidated subsidiaries, is made
known to us by others within those entities, particularly during the period in which this report is being prepared;
b. Designed such internal control over financial reporting, or caused such internal control over financial reporting to be designed
under our supervision, to provide reasonable assurance regarding the reliability of financial reporting and the preparation of
financial statements for external purposes in accordance with generally accepted accounting principles;
c. Evaluated the effectiveness of the registrant’s disclosure controls and procedures and presented in this report our conclusions
about the effectiveness of the disclosure controls and procedures, as of the end of the period covered by this report based on
such evaluation; and
d. Disclosed in this report any change in the registrant’s internal control over financial reporting that occurred during the
registrant’s most recent fiscal quarter (the registrant’s fourth fiscal quarter in the case of an annual report) that has materially
affected, or is reasonably likely to materially affect, the registrant’s internal control over financial reporting; and
5.
The registrant’s other certifying officer(s) and I have disclosed, based on our most recent evaluation of internal control over
financial reporting, to the registrant’s auditors and the audit committee of the registrant’s board of directors (or persons performing
the equivalent functions):
a. All significant deficiencies and material weaknesses in the design or operation of internal control over financial reporting
which are reasonably likely to adversely affect the registrant’s ability to record, process, summarize and report financial
information; and
b. Any fraud, whether or not material, that involves management or other employees who have a significant role in the registrant’s
internal control over financial reporting.
Date: March 8, 2017
/s/ Sean Moran
Sean Moran
Chief Financial Officer
(Principal Accounting and Financial Officer)
Certification of Chief Executive Officer Pursuant to
18 U.S.C. Section 1350,
as Adopted Pursuant to
Section 906 of the Sarbanes-Oxley Act of 2002
Exhibit 32.1
This Certification is being filed pursuant to 18 U.S.C. Section 1350, as adopted by Section 906 of the Sarbanes-Oxley Act of 2002. This
Certification is included solely for the purposes of complying with the provisions of Section 906 of the Sarbanes-Oxley Act and is not
intended to be used for any other purpose. In connection with the accompanying Annual Report on Form 10-K of Corbus Pharmaceuticals
Holdings, Inc. for the year ended December 31, 2016, each of the undersigned hereby certifies in his capacity as an officer of Corbus
Pharmaceuticals Holdings, Inc. that to such officer’s knowledge:
(1) The Annual Report fully complies with the requirements of Section 13(a) or 15(d) of the Securities Exchange Act of 1934; and
(2) The information contained in the Annual Report fairly presents, in all material respects, the financial condition and results of
operations of the Company.
Dated: March 8, 2017
By:/s/ Yuval Cohen
Yuval Cohen
Chief Executive Officer
(Principal Executive Officer)
Certification of Chief Financial Officer Pursuant to
18 U.S.C. Section 1350,
as Adopted Pursuant to
Section 906 of the Sarbanes-Oxley Act of 2002
Exhibit 32.2
This Certification is being filed pursuant to 18 U.S.C. Section 1350, as adopted by Section 906 of the Sarbanes-Oxley Act of 2002. This
Certification is included solely for the purposes of complying with the provisions of Section 906 of the Sarbanes-Oxley Act and is not
intended to be used for any other purpose. In connection with the accompanying Annual Report on Form 10-K of Corbus Pharmaceuticals
Holdings, Inc. for the year ended December 31, 2016, each of the undersigned hereby certifies in his capacity as an officer of Corbus
Pharmaceuticals Holdings, Inc. that to such officer’s knowledge:
(1) The Annual Report fully complies with the requirements of Section 13(a) or 15(d) of the Securities Exchange Act of 1934; and
(2) The information contained in the Annual Report fairly presents, in all material respects, the financial condition and results of
operations of the Company.
Dated: March 8, 2017
By: /s/ Sean Moran
Sean Moran
Chief Financial Officer
(Principal Accounting and Financial Officer)