CEO Letter to Stockholders
Dear Rexahn Stockholders,
Our mission at Rexahn is to improve the lives of cancer patients by discovering and developing
novel, highly targeted, next generation cancer therapies that are designed to maximize anti-
cancer activity while minimizing the side effects and toxicity traditionally experienced with
cancer treatment.
I am pleased to report that we made solid progress towards this objective in 2015 and are well
positioned to continue our momentum in 2016.
Last year we made important strides advancing each of our clinical-stage oncology programs -
RX-3117, Supinoxin™ and Archexin® and generated what we believe are promising interim
clinical data which were presented at prominent medical meetings. Though preliminary, we are
encouraged by the accumulating clinical data which show evidence of a potential clinical
benefit of each of these novel investigational anti-cancer compounds. We were gratified and
excited by the early clinical results, which suggest the potential to delay disease progression
(evidenced as stable disease), and, in some cases, produce tumor shrinkage. These results
fortify our confidence in the merits of our approach and strengthen our commitment to
developing Rexahn’s novel portfolio of potentially first-in-class or best-in-class cancer
therapeutics.
In 2016 we will continue to advance each of our clinical oncology programs and plan to more
comprehensively evaluate RX-3117 and Supinoxin in Phase Ib/IIa proof-of-concept clinical
studies. Based on the existing high unmet medical need and promising clinical and preclinical
data accumulated thus far, we plan to initially evaluate RX-3117 in patients with relapsed and
refractory pancreatic cancer and advanced bladder cancer. In the case of Supinoxin, we are
targeting patients with triple negative breast cancer (TNBC) and platinum resistant ovarian
cancer.
Our Archexin program achieved an important milestone in 2015 following the conclusion of
Stage 1 of our ongoing Phase Ib/IIa clinical trial. Not only did we achieve our intended goal – to
determine a maximum tolerated dose of Archexin for further clinical evaluation, but we saw
promising evidence of both a dose-dependent and duration-of-treatment-dependent reduction in
the size of certain patients’ kidney cancer tumors. We were very pleased to observe potential
early evidence of the clinical activity of Archexin and, earlier this year, we commenced the
randomized portion (Stage 2) of the Phase Ib/IIa clinical trial, which will evaluate Archexin, in
combination with everolimus (a widely used cancer drug) in patients with metastatic renal cell
carcinoma.
I am very encouraged by our progress, and the interim data we are generating, and look forward
to continuing to advance our three clinical-stage oncology programs in 2016 in the hopes of
improving the quality of life and longevity of patients living with cancer.
Moving on, our research engine continued to be very productive in 2015. Our scientists and
academic and clinical collaborators generated intriguing new preclinical data supporting the
anti-cancer effects of our programs. Through this research we confirmed the broad applicability
of RX-3117 against a range of human solid tumors in preclinical models. We were also very
excited to observe the ability of RX-3117 to achieve significant tumor inhibition in gemcitabine-
resistant cancers in preclinical models. This could suggest a potential role for RX-3117 in
treating the approximately 25% to 40% of cancer patients that develop resistance to standard-
of-care therapy with gemcitabine. We also presented new data for Supinoxin elucidating a
unique mechanism of action that is highly cancer-specific. Further, we showed that Supinoxin
dose-dependently decreased the migration of TNBC cells, potentially suggesting the utility of
Supinoxin in the treatment and prevention of TNBC metastases. Additionally, new data were
presented showing Supinoxin’s potent tumor inhibition effects in a xenograft mouse model of
TNBC, further supporting our choice of TNBC as one of the initial clinical indications for the
Supinoxin program.
Finally, we were honored to have been selected by the National Cancer Institute’s
Nanotechnology Characterization Laboratory for funding of the further preclinical development
of Rexahn’s RX-21101 nanotechnology anti-cancer program.
We are fortunate to have a rich diversity of oncology assets at Rexahn and the determination to
progress these programs through preclinical and clinical development with the ultimate goal of
improving the lives of cancer patients. We recognize that our people are the backbone of our
success and took steps to further augment our leadership structure in 2015. Notably, Dr. Ely
Benaim was appointed to the newly-created position of Chief Medical Officer. In addition, Peter
Brandt was appointed as our new Chairman of the Board.
On behalf of our Board of Directors and the employees at Rexahn, I wish to thank you for your
continued interest and support of our Company. We look forward to continuing our progress in
2016. We thank you for joining us on this important journey as we endeavor to improve the
lives of patients with cancer.
Sincerely,
Peter D. Suzdak, Ph.D.
Chief Executive Officer
Rexahn Pharmaceuticals - Oncology Pipeline Overview
With a strong foundation in cancer research and discovery, Rexahn has built a diverse
portfolio of novel oncology assets that includes three clinical-stage investigational anti-
cancer compounds currently in human clinical trials, and a robust oncology research
platform. Our mission is to develop highly targeted cancer therapies with the potential for
enhanced efficacy and safety – an approach which we believe could improve the lives of
cancer patients.
Clinical-Stage Oncology Programs:
RX-3117 – An orally bioavailable, cancer cell specific nucleoside agent that has
shown efficacy against a broad range of human tumor cell types, including
gemcitabine resistant tumors.
Supinoxin™ - A potent, first-in-class, orally bioavailable, small molecule that
inhibits the growth of cancer cells by targeting phosphorylated-p68 (P-p68), which
is highly overexpressed in cancer cells.
Archexin® - A unique anti-sense oncology drug candidate that specifically inhibits
the cancer cell signaling protein Akt-1, which is highly overexpressed in cancer
cells.
RX-3117 – A Novel Next Generation Nucleoside Compound
RX-3117 is an orally bioavailable, small molecule, investigational anti-cancer therapy that
targets a unique biological pathway implicated in the development and metastasis of
numerous cancers.
A novel, cancer-cell specific nucleoside analogue, RX-3117 is a prodrug activated by the
enzyme Uridine Cytidine Kinase, or ‘UCK2’, which is only present in cancer cells. Once
activated by UCK2, RX-3117 inhibits DNA and RNA synthesis leading to cancer cell
death. Because UCK2 is overexpressed in multiple human tumors – but has a very limited
presence in normal tissues, RX-3117 offers the potential for a targeted anti-cancer therapy
with an improved efficacy and safety profile.
that have become resistant
Preclinical studies of RX-3117 in patient-derived, cancer cell xenograft models have
demonstrated broad anti-tumor activity and – most importantly, an ability to treat cancer
cells
to gemcitabine, a widely-used chemotherapy.
Unfortunately, resistance to gemcitabine occurs in up to approximately 25% to 40% of
cancer patients over time, reducing overall treatment efficacy and further limiting options
for patients.
RX-3117 has shown broad spectrum anti-tumor activity against over 100 different human
cancer cell lines and efficacy in 17 different mouse xenograft models. In preclinical mouse
1
xenograft studies, RX-3117 demonstrated superior efficacy to gemcitabine. In addition,
RX-3117 retained its full anti-tumor activity in human cancer cell lines made resistant to
the anti-tumor effects of gemcitabine, supporting a unique, highly-targeted mechanism of
action.
An exploratory Phase I clinical trial of RX-3117 showed that oral administration of a 50
mg dose of RX-3117 achieved oral bioavailability of 56% and a plasma half-life (T1/2) of
14 hours. In addition, RX-3117 appeared to be safe and well tolerated in all subjects
throughout the dose range tested.
Interim results from a separate Phase Ib clinical trial of RX-3117 in patients with advanced
and metastatic solid tumors were presented in September 2015 at the European Cancer
Congress. The results showed that, at the dose levels tested to date, RX-3117 appeared to
show evidence of tumor reduction (9%), which was observed in one patient, and stable
disease, which was observed in five patients persisting from between 112 and 276 days
before disease progression occurred. In addition, RX-3117 appeared to be safe and well
tolerated with a predictable pharmacokinetic profile. The most frequently reported
treatment emergent adverse events in the Phase Ib trial were mild to moderate fatigue,
gastrointestinal disturbances, anemia, pyrexia, decreased appetite and dehydration.
RX-3117 is currently being evaluated in a Phase Ib/IIa multi-center, open-label clinical
study in patients with relapsed or refractory pancreatic cancer and advanced bladder
cancer. The study has a two-stage design. In the initial stage, 10 patients with relapsed or
refractory pancreatic cancer and 10 patients with advanced bladder cancer will be enrolled.
If responses are observed within the first 10 response-evaluable subjects, 40 additional
subjects could be enrolled. RX-3117 has received Orphan Drug designation for the
treatment of pancreatic cancer – one of the reasons we have selected this indication for
initial clinical study.
In the Phase Ib/IIa study, RX-3117 is being administered orally five times weekly on a
three weeks on, one week off dosing schedule. The primary endpoint of the Phase Ib/IIa
trial is an assessment of the progression free survival rate or an objective clinical response
rate and reduction in tumor size. Secondary endpoints include time to disease progression,
overall response rate and duration of response, as well as pharmacokinetic assessments and
safety parameters.
Supinoxin – A Potential First-in-Class Inhibitor of a Unique Cancer Protein
Supinoxin™ (RX-5902) is an orally administered, potential first-in-class, small molecule
inhibitor of a unique cancer protein – phosphorylated-p68 (P-p68) which is selectively
overexpressed in cancer cells and absent in normal tissue. P-p68 is believed to increase the
activity of multiple cancer related genes including, cyclin D1, c-jun and c-myc, and play a
prominent role in tumor progression and metastasis. Over-expression of P-p68 has been
observed in many solid tumors, including, melanoma, colon, ovarian and lung tumors.
2
In preclinical studies, Supinoxin has been shown to inhibit proliferation of cancer cells in
over 100 different human cancer cell lines, including, breast, ovarian, colon, pancreas, and
stomach cancers, and has shown potent activity in drug-resistant cancer cells. In preclinical
animal models, where human cancer cells from triple negative breast cancer, ovarian,
melanoma, pancreas, or renal tumors were grafted into animals, treatment with Supinoxin
resulted in a significant reduction in tumor growth.
Supinoxin is currently being evaluated in a Phase I multi-center, dose-finding, open-label,
single agent clinical study in patients with advanced or metastatic solid tumors.
Interim results from the Phase I clinical trial were presented at the European Cancer
Congress in September 2015. The results showed that, at the dose levels tested to date,
clinical evidence of single-agent activity of Supinoxin was observed in approximately 29%
of patients (4/14 patients) who showed stable disease persisting from between 255 and 497
days (as of September 14, 2015.)
In addition, Supinoxin, administered orally, appeared to be safe and well tolerated with no
Grade 3 or Grade 4 adverse events and only one unrelated Grade 2 adverse event. The
most frequently reported drug related adverse events were mild nausea, vomiting and
fatigue. Pharmacokinetic analyses of the current data show both a predictable and
desirable pharmacokinetic profile for an orally-administered route of therapy.
The Phase I study of Supinoxin is currently ongoing and Rexahn anticipates commencing a
Phase Ib/IIa Proof-of-Concept clinical trial in the first half of 2016 in patients with triple
negative breast cancer and platinum resistant ovarian cancer to more comprehensively
evaluate Supinoxin’s clinical activity.
Archexin – A Potential Best-in-Class Akt-1 Inhibitor
Archexin is a unique antisense oncology drug candidate that specifically inhibits the cancer
cell signaling protein Akt-1, which is highly overexpressed in cancer cells. Archexin is the
only specific inhibitor of Akt-1 in clinical development. The activated form of Akt-1,
which is involved in cancer cell growth, survival, angiogenesis, and drug resistance, has
been shown to be present or elevated in more than 12 different human cancer cell lines,
including pancreatic and renal cell carcinoma.
We believe the overall safety profile of Archexin may be superior to existing cytotoxic
compounds and chemotherapeutic drugs that affect growth in both cancerous and non-
cancerous cells. In two clinical trials, Archexin appeared to be safe and well tolerated at
all dose levels tested with no dose-limiting toxicities. The FDA has granted Orphan Drug
Designation to Archexin in the treatment of five cancers: renal cell, pancreatic, ovarian,
stomach, and glioblastoma.
In a small Phase IIa trial, Archexin appeared to be safe and well tolerated and showed
preliminary signs of efficacy in advanced pancreatic cancer patients when used in
combination with gemcitabine. Median survival for patients dosed with Archexin plus
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gemcitabine was 9.1 months as compared to historical survival data of 5.7 months for
gemcitabine alone.
Following consultation with key opinion leaders in oncology, Rexahn initiated a Phase
Ib/IIa clinical trial of Archexin in patients with metastatic renal cell carcinoma. The strong
scientific evidence, high unmet clinical need, and achievement of Orphan Drug
Designation for renal cell carcinoma provided a firm rationale for this approach.
In addition, resistance to the anti-cancer effects of mTOR inhibitors such as everolimus
(Afinitor® ), a widely used chemotherapy drug which is used as second line therapy in
renal cell carcinoma patients, has been attributed to an increase in Ak-1 activity. Thus, we
believe that treatment with Archexin could provide a synergistic treatment effect –
inhibiting the growth of renal cell carcinoma and overcoming the resistance to mTOR
inhibitors, resulting in enhanced efficacy.
Rexahn is currently conducting a Phase Ib/IIa clinical trial of Archexin in patients with
metastatic renal cell carcinoma (RCC). This is a multi-center study designed to evaluate
the efficacy of Archexin in combination with everolimus to treat metastatic renal cell
carcinoma patients. This trial is being conducted in two stages. The first stage is a dose
ranging study to determine the maximum tolerated dose of Archexin in combination with
everolimus. The first stage of the study was completed in 2015 and a maximum tolerated
dose of Archexin was identified.
Notably, the results from Stage 1 of the Phase Ib/IIa trial showed early evidence of the
potential clinical activity of Archexin in combination with everolimus. Among the patients
enrolled in the study, four patients had stable disease which persisted for 383 days, 191
days, 176 days and 106 days, respectively. In addition, compared to baseline CT scans,
three of these patients experienced reductions in the size of their tumors which ranged from
16% to 36%. At the lowest dose level of Archexin administered (125 mg/m2/day) one
patient had a 16% tumor reduction after 4 cycles of treatment. At the second dose level
(200 mg/m2/day) one patient experienced a 36% tumor reduction after 2 cycles of
treatment. At the highest dose level (250 mg/m2/day), which has been determined to be the
maximum tolerated dose, one patient had a 32% reduction in their tumor size following 6
cycles of treatment.
At the dose levels tested to date, Archexin appeared to be safe and well tolerated. The
most commonly reported adverse event in patients taking both Archexin and everolimus
was thrombocytopenia. To date, no adverse events have been dose limiting.
Stage 2 of the Phase IIa clinical study, which commenced enrolling patients in 2016, is a
randomized, open-label, two-arm dose expansion study of Archexin in combination with
everolimus, versus everolimus alone, to determine safety and efficacy of the combination.
The trial is anticipated to enroll up to 30 metastatic RCC patients who will be randomized
to receive either Archexin in combination with everolimus, or everolimus alone, in a ratio
of 2:1. The maximum tolerated dose of 250 mg/m2/day of Archexin – identified in Stage
1, will be administered along with 10 mg of everolimus versus 10 mg everolimus alone.
4
The primary endpoint of Stage 2 is the percentage of progression free patients following
eight cycles of therapy. Patients are scanned (CT or MRI) for the assessment of tumor
progression after every 2 cycles of therapy. Secondary endpoints include pharmacokinetic
profile, incidence of adverse events, changes in clinical laboratory tests and vital signs over
time, tumor response, duration of response, time to response, and response rate.
Exploratory endpoints include blood levels of Akt pathway biomarkers, tumor apoptosis
biomarkers, or other relevant biomarkers.
Metastatic RCC represents an attractive market opportunity with an estimated annual
incidence of 90,000 patients worldwide. Metastatic RCC patients receiving standard of
care treatment have a poor prognosis with an overall survival of less than two years.
Rexahn has received U.S. Food and Drug Administration (FDA) Orphan Drug Designation
for Archexin for metastatic RCC as well as four other cancers.
Oncology Research Programs
Nano-Drug Delivery Platform for FDA-Approved Chemotherapy Drugs:
Rexahn’s Nano-Polymer-Drug Conjugate System (NPDCS) combines FDA-approved
chemotherapies with a proprietary polymer carrier that is designed to deliver chemotherapy
drugs directly into the tumor while bypassing healthy cells. This approach minimizes the
level of freely-circulating drug in the body while maximizing the drug exposure at the
tumor site, potentially increasing efficacy and minimizing toxic side effects.
RX-21101 – A Proprietary Nano-Polymer Anti-Cancer Therapeutic
RX-21101 combines Rexahn’s nano-drug delivery system with docetaxel, a widely-used,
FDA-approved chemotherapy drug. RX-21101 may enhance efficacy while reducing the
systemic toxicity of traditional docetaxel delivery by specifically targeting the tumor site
and reducing drug exposure elsewhere in the body. Potential indications for RX-21101
include breast, ovarian, prostate and lung cancer. In 2015, the National Cancer Institute’s
(NCI) Nanotechnology Characterization Laboratory selected RX-21101 for funding of the
further preclinical development of this program, under NCI’s preclinical characterization
program.
5
_________________________________________________________________________________________________
UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM 10-K
(Mark One)
ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF
THE
SECURITIES EXCHANGE ACT OF 1934
For the fiscal year ended December 31, 2015
OR
TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d)
OF
THE SECURITIES EXCHANGE ACT OF 1934
For the transition period from to
Commission File No.:001-34079
Rexahn Pharmaceuticals, Inc.
(Exact name of registrant as specified in its charter)
Delaware
(State or other jurisdiction of incorporation or organization)
11-3516358
(I.R.S. Employer Identification Number)
15245 Shady Grove Road, Suite 455
Rockville, MD 20850
(Address of principal executive offices, including zip code)
Telephone: (240) 268-5300
(Registrant’s telephone number, including area code)
Indicate by check mark if the registrant is a well-known seasoned issuer, as defined by 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
Exchange Act
Yes No
Indicate by check mark whether the registrant (1) has filed all reports required to be filed by Section 13 or 15(d) of
the Securities Exchange Act of 1934 during the preceding 12 months (or for such shorter period that the registrant
was required to file such reports), and (2) has been subject to such filing requirements for the past 90 days. Yes
No
Indicate by check mark whether the registrant has submitted electronically and posted on its corporate Web site, if
any, every Interactive Data File required to be submitted and posted pursuant to Rule 405 of Regulation S-T
(§232.405 of this chapter) during the preceding 12 months (or for such shorter period that the registrant was required
to submit and post such files). Yes No
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 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 definition of ―accelerated filer,‖ ―large accelerated filer‖ and ―smaller reporting
company‖ in Rule 12b-2 of the Exchange Act. (Check one):
Large Accelerated Filer
Non-Accelerated Filer
(Do not check if a smaller reporting company)
Accelerated Filer
Smaller reporting company
Indicate by check mark whether the registrant is a shell company (as defined in Rule 12b-2 of the Exchange Act)
Yes No
State the aggregate market value of the voting and non-voting common equity held by non-affiliates computed by
reference to the price at which the common equity was last sold, or the average bid and asked price of such common
equity, as of the last business day of the registrant‘s most recently completed second fiscal quarter: As of June 30,
2015, the aggregate market value of the registrant’s common stock held by non-affiliates of the registrant was
$105,800,912 based on the closing price reported on NYSE MKT.
Indicate the number of shares outstanding of each of the issuer‘s classes of common stock, as of the latest
practicable date:
Class
Common Stock, $0.0001 par value per share
Outstanding as of March 11, 2016
213,113,785 shares
DOCUMENTS INCORPORATED BY REFERENCE
Certain portions of the registrant‘s Definitive Proxy Statement for its 2016 Annual Meeting of
Stockholders, which is expected to be filed with the U.S. Securities and Exchange Commission within 120 days
after the end of the registrant‘s fiscal year ended December 31, 2015, are incorporated by reference into Part III of
this Annual Report on Form 10-K.
Cautionary Statement Regarding Forward-Looking Statements.
This Annual Report on Form 10-K contains statements (including certain projections and
business trends) accompanied by such phrases as ―believe,‖ ―estimate,‖ ―expect,‖ ―anticipate,‖ ―will,‖
―intend‖ and other similar expressions, that are ―forward-looking statements‖ as defined in the Private
Securities Litigation Reform Act of 1995. We caution that forward-looking statements are based largely
on our expectations and are subject to a number of known and unknown risks and uncertainties that are
subject to change based on factors which are, in many instances, beyond our control. Actual results,
performance or achievements may differ materially from those contemplated, expressed or implied by the
forward-looking statements.
Although we believe that the expectations reflected in our forward-looking statements are
reasonable as of the date we make them, actual results could differ materially from those currently
anticipated due to a number of factors, including risks relating to:
our understandings and beliefs regarding the role of certain biological mechanisms and processes
in cancer;
our drug candidates being in early stages of development, including in pre-clinical development;
our ability to initially develop drug candidates for orphan indications to reduce the time-to-market
and take advantage of certain incentives provided by the U.S. Food and Drug Administration;
our ability to transition from our initial focus on developing drug candidates for orphan
indications to candidates for more highly prevalent indications;
our ability to successfully and timely complete clinical trials for our drug candidates in clinical
development;
uncertainties related to the timing, results and analyses related to our drug candidates in
pre-clinical development;
our ability to obtain the necessary U.S. and international regulatory approvals for our drug
candidates;
our reliance on third-party contract research organizations and other investigators and
collaborators for certain research and development services;
our ability to maintain or engage third-party manufacturers to manufacture, supply, store and
distribute supplies of our drug candidates for our clinical trials;
our ability to form strategic alliances and partnerships with pharmaceutical companies and other
partners for sales and marketing of certain of our product candidates;
demand for and market acceptance of our drug candidates;
the scope and validity of our intellectual property protection for our drug candidates and our
ability to develop our candidates without infringing the intellectual property rights of others;
our lack of profitability and the need for additional capital to operate our business; and
other risks and uncertainties, including those set forth herein under the caption ―Risk Factors‖ and
those detailed from time to time in our filings with the Securities and Exchange Commission.
These forward-looking statements are made only as of the date hereof, and we undertake no obligation to
update or revise the forward-looking statements, whether as a result of new information, future events or
otherwise.
REXAHN PHARMACEUTICALS, INC.
TABLE OF CONTENTS
PART I
Item 1 Description of Business
Item 1A Risk Factors
Item 1B Unresolved Staff Comments
Item 2 Description of Property
Item 3
Item 4 Mine Safety Disclosures
Legal Proceedings
Selected Financial Data
PART II
Item 5 Market for Common Equity, Related Stockholder Matters, and Issuer Purchases of Equity Securities
Item 6
Item 7 Management‘s Discussion and Analysis of Financial Condition and Results of Operations
Item 7A Qualitative and Quantitative Disclosures About Market Risk
Financial Statements and Supplementary Data
Item 8
Item 9
Changes In and Disagreements with Accountants on Accounting and Financial Disclosure
Item 9A Controls and Procedures
Item 9B Other Information
PART III
Item 10 Directors, Executive Officers, and Corporate Governance
Item 11 Executive Compensation
Item 12 Security Ownership of Certain Beneficial Owners and Management and Related Stockholder Matters
Item 13 Certain Relationships and Related Transactions; and Director Independence
Item 14 Principal Accounting Fees and Services
Item 15 Exhibits, Financial Statement Schedules
SIGNATURES
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Unless the context requires otherwise, any references in this Annual Report on Form 10-K to “we,” “us,”
“our,” the “Company” or “Rexahn” refers to Rexahn Pharmaceuticals, Inc.
PART I
Item 1. Description of Business
Overview
We are a clinical stage biopharmaceutical company dedicated to the discovery, development and
commercialization of innovative treatments for cancer. Our mission is to improve the lives of cancer
patients by developing next generation cancer therapies that are designed to maximize efficacy while
minimizing the toxicity and side effects traditionally associated with cancer treatment. Our clinical
pipeline features one oncology product candidate in Phase II clinical development, two oncology product
candidates in Phase I clinical development, and additional compounds in pre-clinical development. Our
strategy is to continue building a significant pipeline of innovative oncology product candidates that we
intend to commercialize alone or with partners. Our three clinical stage drug candidates in active
development are Archexin® , RX-3117 and SupinoxinTM (RX-5902).
Archexin is a potential best-in-class, potent inhibitor of the protein kinase Akt-1, which we
believe plays a critical role in cancer cell proliferation, survival, angiogenesis, metastasis and
drug resistance. Archexin has received ―orphan drug‖ designation from the U.S. Food and Drug
Administration (the ―FDA‖) for renal cell carcinoma (―RCC‖), glioblastoma, ovarian cancer,
stomach cancer and pancreatic cancer. Orphan drug designation provides tax incentives for
clinical research and a waiver from user fees under certain circumstances. In addition, an orphan
drug receives seven years of exclusivity after approval, during which the FDA generally cannot
approve another product with the same active moiety for the same indication. We have
completed a pilot Phase IIa clinical trial of Archexin for the treatment of pancreatic cancer. We
are currently conducting a Phase IIa proof-of-concept clinical trial of Archexin in patients with
metastatic renal cell carcinoma to evaluate its safety and efficacy.
RX-3117 is a small molecule nucleoside compound with an anti-metabolite mechanism of action,
and we believe it has therapeutic potential in a broad range of cancers, including pancreatic,
bladder, colon, and lung cancer. We completed an exploratory Phase I clinical study of
RX-3117 that showed a level of oral bioavailability of RX-3117 in humans with no adverse
effects reported. We are currently conducting a Phase Ib clinical trial to study the safety and
efficacy of RX-3117 in patients with solid tumors. RX-3117 has received orphan drug
designation from the FDA for pancreatic cancer.
Supinoxin, or RX-5902,
inhibitor of
phosphorylated-p68, a protein that we believe plays a key role in cancer cell growth, progression
and metastasis. We are currently conducting a Phase I clinical trial of Supinoxin to evaluate its
safety and efficacy in patients with solid tumors.
small molecule
is a potential
first-in-class
We also have one drug candidate
in pre-clinical development: RX-21101, an
N-(2-Hydroxypropyl) methacrylamide-docetaxel-folate, which we believe may provide increased efficacy
against tumors with potentially fewer side effects as a result of specific tumor targeting and increased
stability in the body.
In addition to our drug development efforts, we are also working on proprietary research
technologies, including our multi-target aimed ligands platform and nano-based drug delivery systems.
Our unique ligand discovery platform, The Inhibitors of Multi-Expression Signals (―TIMES‖), permits us
to identify potentially important targets that control multiple genes or signaling events in cancer cells. Our
3-D Gateway of Ligand Discovery (―3-D GOLD‖) integrates three-dimensional molecular modeling with
databases of chemicals and proteins and ligand filtering and generation, which helps us discover novel
lead compounds. Leveraging this system, we believe that we are able to effectively develop predictive
models, formulate and test hypotheses for optimizing efficacy, and increase drug safety and
bioavailability early in the drug discovery process. Our nano-based drug delivery systems, such as those
1
used in the multiple nanoliposomal- and nanopolymer-based anti-cancer drugs that we are currently
testing, may increase the availability of a drug at the disease site, minimize adverse reactions, and provide
longer duration of action.
Company Background
We trace our history to the March 2001 founding of Rexahn, Corp, which in 2005 merged with
and into Rexahn Pharmaceuticals, Inc. (formerly Corporate Road Show.com Inc.). Dr. Peter Suzdak,
our Chief Executive Officer since February 2013, has extensive experience in drug development,
particularly in the field of oncology. Dr. Chang Ahn, our founder, Chief Scientist and Chairman
Emeritus of our Board of Directors, is a former FDA reviewer and National Cancer Institute (NCI)
research scientist. He guided our initial research and commercialization efforts in targeted oncology
drugs.
Our common stock is currently listed on the NYSE MKT under the trading symbol ―RNN.‖ Our
principal corporate office is located at 15245 Shady Grove Road, Suite 455, Rockville, Maryland 20850
in Maryland‘s I-270 technology corridor. Our telephone number is (240) 268-5300.
Industry and Disease Markets
Market Overview
Our primary research and development focus is on oncology therapeutics. A key component of
our strategy is to develop innovative drugs that are potential first-in-class or market-leading compounds
for the treatment of cancer. According to the Centers for Disease Control and Prevention, cancer claims
the lives of more than half a million Americans each year and is the second leading cause of death among
Americans. In 2014, the World Economic Forum and the Harvard School of Public Health estimated
that the 13.3 million new cases of cancer diagnosed worldwide in 2010 cost $290 billion, projected 21.5
million new cancer cases occurring in 2030 at a cost of $458 billion; and a 2016 American Cancer Society
report projected that an estimated 1.7 million new cancer cases will be diagnosed in the United States in
2016. In 2015, Evaluate Pharma projected that global annual sales of cancer drugs would grow to $153
billion by 2020.
Current Cancer Treatments
Traditional cancer treatments involve surgery, radiation therapy and chemotherapy. Surgery is
widely used to treat cancer, but may result in related or significant complications and may be ineffective
if metastasis has occurred. Radiation therapy, or radiotherapy, can be highly effective in treating certain
types of cancer. In radiation therapy, ionizing radiation deposits energy that injures or destroys cells in
the area being treated by damaging their genetic material, making it impossible for these cells to continue
to grow. Although radiation damages both cancer cells and normal cells, the normal cells are generally
able to repair themselves and function properly. Chemotherapy involves the use of cytotoxic cancer
drugs to destroy cancer cells by interfering with various stages of the cell division process. For certain
cancers and in certain patients, these drugs have limited efficacy and debilitating adverse side effects.
Administration of cytotoxic cancer drugs may also result in the development of multiple drug, or
multi-drug, resistance, which is a condition that results when certain tumor cells that have survived
treatment with cytotoxic drugs are no longer susceptible to treatment by those and other drugs.
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Unmet Needs in Cancer
Despite significant advances in cancer research and treatments, many unmet needs still remain
including:
Long-term management of cancers: Surgery, radiation therapy or chemotherapy may not
result in long-term remission, although surgery and radiation therapies are considered
effective methods for some cancers. There is a need for more effective drugs and adjuvant
therapies to treat relapsed and refractory cancers.
Multi-drug resistance: Multi-drug resistance is a major obstacle to effectively treating
various cancers with chemotherapy.
Debilitating toxicity by chemotherapy: Chemotherapy as a mainstay of cancer treatment can
induce severe adverse reactions and toxicities, adversely affecting quality of life or life itself.
Market Opportunity
There are several factors that are favorable for commercializing new cancer drugs that may be
first-in-class or market leaders, including:
Expedited Regulatory or Commercialization Pathways. Drugs for life-threatening diseases such
as cancer are often candidates for fast track designation, breakthrough therapy designation,
priority review and accelerated approval, each of which can lead to approval sooner than would
otherwise be the case.
Favorable Environment for Formulary Access and Reimbursement. We believe cancer drugs
with proven efficacy would gain rapid market uptake, formulary listing and third-party payor
reimbursement. Drugs with orphan designations are generally reimbursed by third-party payors
because there are few, if any, alternatives.
Focus on Specialty Markets. The marketing of new drugs to specialty physicians can be
accomplished with a specialty sales force that requires fewer personnel and lower related costs
than a typical sales force that markets widely to primary care physicians and general practitioners.
Our Strategy
Our strategy is to continue building a significant product pipeline of innovative drug candidates
that we intend to commercialize alone or with partners. This strategy has several key components.
Develop Innovative Therapeutics with the Potential to be First-in-Class or Market Leaders
We plan to focus our research and development pipeline on potential first-in-class or
market-leading compounds for the treatment of cancer. By expanding the breadth and depth of our
oncology pipeline, we aim to develop an industry-leading oncology therapeutics franchise. Our pipeline
spans several major classes of cancer drugs, including molecular targeted therapies, signal transduction
and multi-kinase inhibitors, and nano-medicines for targeted delivery of compounds and small molecule
cytotoxic compounds. Differentiated target product profiles and proprietary discovery and research
technology platforms further support these strategic efforts.
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Clinically Develop Drug Candidates as Orphan Drugs
We intend to initially develop drug candidates for cancers that are orphan indications. Under the
Orphan Drug Act, the FDA may grant orphan drug designation to new drugs developed to treat diseases
generally affecting less than 200,000 patients. Incentives associated with orphan drug designation
include tax incentives for research and development and an exemption from user fees under certain
circumstances. Although the standards for orphan drug approval are not different than for non-orphan
products, the path to approval may be faster because clinical trials may be smaller due to the smaller
patient population. Further, a drug that is approved for its orphan-designated indication generally receives
seven years of orphan drug exclusivity, during which the FDA generally may not approve any other
application for a product containing the same active moiety and proposed for the same indication. An
approved orphan drug also may qualify for an exemption from the branded prescription drug fee. We
plan to develop drug candidates for cancers that are orphan indications in order to take advantage of the
benefits of orphan drug designation during development and the exclusivity available under the Orphan
Drug Act for approved products, as well as the potential for reduced time to market. Drugs intended to
treat rare diseases or conditions also may qualify for fast track designation, breakthrough therapy
designation, accelerated approval and/or priority review, any or all of which can speed the approval
process.
Target Signal Transduction Molecules with Multiple Drug Candidates
We plan to expand our research and development pipeline to introduce new signal inhibitor drugs
into clinical trials in the future. By identifying and characterizing the genes and proteins that control the
signaling pathways and gene expression of cancer cells, we seek to develop DNA/RNA-based and
small-molecule drugs to treat a broad range of diseases caused by abnormal expression or functions of
those genes and proteins.
Establish Partnerships with Large Pharmaceutical Companies
We seek to establish strategic alliances and partnerships with large pharmaceutical companies for
the development of our drug candidates.
In-License Unique Technology
We continually review opportunities to in-license and advance compounds in oncology that have
value creating potential and will strengthen our clinical development pipeline.
Capitalize on Our Management Team’s Expertise for Drug Development
Our management team possesses clinical development experience in oncology and several other
therapeutic areas which facilitates strategic approaches to and competitive advantages in, the design, risk
assessment and implementation of drug development programs. Our management team also has prior
experience in pharmaceutical alliances, product launches and marketing.
Our Pipeline Drug Candidates
Clinical Stage Pipeline
Archexin: Potential Best-in-class Anti-Cancer Akt-1 Inhibitor
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Archexin is a potential best-in-class, potent inhibitor of the protein kinase Akt-1, which we
believe plays a critical role in cancer cell proliferation, survival, angiogenesis, metastasis and drug
resistance. Archexin has received orphan drug designation from the FDA for RCC, glioblastoma, ovarian
cancer, stomach cancer and pancreatic cancer. We believe Archexin is differentiated from other Akt-1
inhibitors by its ability to inhibit both activated and inactivated forms of Akt-1, and as a result it is not
expected to lead to drug resistance, which has been observed with other protein kinase inhibitors. Other
targeted drugs may only inhibit inactivated Akt-1 and may also cause drug resistance. Akt-1 is
over-activated in patients with many cancers, including breast, colorectal, gastric, pancreatic, prostate and
melanoma cancers. Akt-1 activity may be inhibited by signaling molecules upstream of Akt-1 in cancer
cells through the use of vascular endothelial growth factor and epidermal growth factor receptor inhibitors,
but this treatment only indirectly affects the activity of native Akt-1. Because signal transmission for
cancer progression and resistance occurs when Akt-1 is activated, we believe it is also important to inhibit
activated Akt-1. We believe Archexin inhibits both activated and native Akt-1.
Archexin is an antisense oligonucleotide compound that is complementary to Akt-1 mRNA and
highly selective for inhibiting mRNA expression, which leads to reduced production of Akt-1 protein.
Archexin preliminarily appeared to be safe and well tolerated with minimal side effects in a Phase I study
in patients with advanced cancers, where Grade 3 fatigue was the only dose-limiting toxicity and no
significant hematological abnormalities were observed. The main objectives of the Phase I study were to
determine the maximum tolerated dose, dose limiting toxicity and pharmacokinetic parameters for
Archexin monotherapy. The Archexin Phase I study design was an open label, single arm ascending
dose, safety and tolerability study.
We have completed an open label, two-stage Phase IIa clinical trial for Archexin that was designed
to assess the safety and efficacy of Archexin in combination with gemcitabine. Gemcitabine is used to treat
pancreatic, breast, ovarian, and lung cancers, and may be used for other cancers as well. Gemcitabine is a
member of a group of chemotherapy drugs known as anti-metabolites, which prevent cells from making
DNA and RNA, which in turn stops cell growth and causes cells to die. Stage 1 was the dose-finding
portion of the study, and Stage 2 was the dose-expansion portion of the study using the dose identified in
Stage 1 administered together with gemcitabine. The study enrolled 31 subjects aged 18 to 65 with
metastatic pancreatic cancer at nine centers in the United States and India. The primary endpoint was
overall survival following four cycles of therapy with a six month follow-up. For those evaluable patients,
the study showed that treatment with Archexin in combination with gemcitabine provided a median
survival rate of 9.1 months compared to the historical survival data of 5.7 months for standard single agent
gemcitabine therapy. The most frequently reported adverse events were constipation, nausea, abdominal
pain and pyrexia, regardless of relatedness.
We are conducting an ongoing Phase IIa proof-of-concept clinical trial of Archexin to study its
safety and efficacy in patients with metastatic RCC. In this trial, Archexin is being administered in
combination with Afinitor® (everolimus). The trial is being conducted in two stages. Stage 1 was a dose
ranging study, with up to three dose groups with three RCC patients each, to determine its maximum
tolerated dose (―MTD‖) in combination with everolimus. In January 2016, we completed Stage 1 of the
study and commenced enrollment in Stage 2, which is a randomized, open-label, two-arm dose expansion
study of everolimus versus Archexin in combination with everolimus to determine safety and efficacy of
the combination. This phase of the trial (Stage 2) is anticipated to enroll up to 30 RCC patients who will be
randomized to receive either Archexin in combination with everolimus, or everolimus alone, in a ratio of
2:1 The MTD was determined to be 250 mg/m2/day of Archexin, which was identified in Stage 1 and will
be administered in Stage 2 along with 10 mg of everolimus compared to 10 mg everolimus alone.
We have been issued a U.S. patent for Archexin that covers composition of matter and broad
claims for the nucleotide sequences of the antisense compounds that target and inhibit the expression of
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Akt-1 in human tissues or cells, and for the method of using the compounds to induce cytotoxicity in
cancer cells.
RX-3117: Oral Small Molecule Nucleoside
RX-3117 is a novel, investigational oral small molecule nucleoside compound. In pre-clinical
models when activated (phosphorylated) by uridine-cytidine kinase 2 (―UCK2‖), a protein that is
overexpressed in various human cancer cells, RX-3117 is incorporated into DNA or RNA of cells and
inhibits both DNA and RNA synthesis, which induces apoptotic cell death of tumor cells. We believe
RX-3117 has therapeutic potential in a broad range of cancers including pancreatic, bladder, lung,
cervical, non-small cell lung cancer and colon cancer. RX-3117 has received orphan drug designation
from the FDA for the treatment of patients with pancreatic cancer. RX-3117 has also been shown in
animal models to inhibit the growth of gemcitabine-resistant human cancers and improve overall survival.
RX-3117 has demonstrated broad spectrum anti-tumor activity against over 100 different human
cancer cell lines and efficacy in 17 different mouse xenograft models. Notably, the efficacy of RX-3117
in the mouse xenograft models was superior to that of gemcitabine. Further, RX-3117 still retains its full
anti-tumor activity in human cancer cell lines made resistant to the anti-tumor effects of gemcitabine. In
August 2012, Rexahn reported the completion of an exploratory Phase I clinical trial of RX-3117 in
cancer patients conducted in Europe, to investigate the oral bioavailability, safety and tolerability of the
compound. In this study, oral administration of a 50 mg dose of RX-3117 demonstrated an oral
bioavailability of 56% and a plasma half-life (T1/2) of 14 hours. In addition, RX-3117 appeared to be safe
and well tolerated in all subjects throughout the dose range tested.
We are currently evaluating RX-3117 in a Phase Ib clinical trial to study the safety, tolerability,
dose-limiting toxicities and MTD in patients with solid tumors. Secondary endpoints include
pharmacokinetic analysis and an evaluation of the preliminary anti-tumor effects of RX-3117.
Preliminary results from the Phase Ib clinical trial of RX-3117 were presented in September 2015
at the 2015 European Cancer Congress. The results showed that, at the dose levels tested to date, RX-3117
administered orally appeared to be safe and well tolerated with a predictable pharmacokinetic profile. The
most frequently reported treatment emergent adverse events were mild to moderate fatigue,
gastrointestinal disturbances, anemia, pyrexia, decreased appetite and dehydration.
In addition, preliminary anti-tumor activity was seen in the Phase Ib clinical trial, with evidence of
tumor reduction observed in one patient and stable disease observed in five patients persisting from
between 112 and 276 days before disease progression occurred.
Patient enrollment has been completed in nine dose groups (30mg, 60mg, 100mg, 150mg, 200mg,
500mg, 1,000mg, 1,500mg and 2,000 mg). Based on the favorable safety and pharmacokinetic profile
seen at the highest dose levels, we initiated a dosing schedule modification to increase patients‘ daily
exposure of RX-3117 in the ongoing Phase Ib clinical trial. All newly enrolled patients are now receiving
RX-3117 five times weekly. We anticipate that the new dosing paradigm will increase drug exposure,
maximizing potential therapeutic activity and enable more rapid determination of the MTD for further
clinical study.
Based on the progress of the RX-3117 clinical development program and the level of interest
expressed from a number of oncology-focused pharmaceutical companies, we are continuing discussions
with multiple companies to explore collaborative business structures in an effort to maximize the potential
value of the program
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Supinoxin: Potential First-in-Class p68 RNA Helicase Inhibitor
Supinoxin is a potential first-in-class small molecule inhibitor of phosphorylated-p68, a protein
that we believe plays a key role in cancer growth, progression and metastasis. Phosphorylated p68, which
is highly expressed in cancer cells, but not in normal cells, results in up-regulation of cancer-related genes
and a subsequent proliferation of cancer cells and tumor growth. Supinoxin selectively blocks
phosphorylated p68, thereby decreasing the proliferation or growth of cancer cells. In pre-clinical tissue
culture models and in-vivo xenograft models, Supinoxin has exhibited single-agent tumor growth
inhibition, potential synergy with cytotoxic agents and activity against drug resistant cancer cells. In
particular, in in-vivo xenograft models of human triple negative breast cancer and pancreatic cancer,
treatment with Supinoxin on days one through 20 in mouse models produced a dose-dependent inhibition
of tumor growth and a survival benefit.
Supinoxin is currently being evaluated in a Phase I dose-escalation clinical trial in cancer patients
with solid tumors designed to evaluate the safety, tolerability, dose-limiting toxicities and MTD. Secondary
endpoints include pharmacokinetic analyses and an evaluation of the preliminary anti-tumor effects of
Supinoxin. Patients have received doses up to 25, 50, 100, 150, 225, 300, 425, 575, and 775 mg. Based
on the favorable safety and pharmacokinetic profile seen at the highest dose levels (575 mg and 775 mg),
we have initiated a dosing schedule modification to increase patients' daily exposure of Supinoxin. All
newly enrolled patients are now receiving Supinoxin seven times weekly as opposed to once weekly. We
anticipate that the new dosing paradigm will increase drug exposure and enable more rapid determination
of the MTD for further clinical study.
Preliminary results from the Phase I clinical trial of Supinoxin were presented in September 2015 at
the 2015 European Cancer Congress.
The results showed that, at the dose levels tested to date, Supinoxin administered orally appeared to
be safe and well tolerated with no Grade 3 or Grade 4 adverse events and several unrelated Grade 2 adverse
events. The most frequently reported drug related adverse events were mild nausea, vomiting and fatigue.
Pharmacokinetic analyses of the current data demonstrate both a predictable and desirable pharmacokinetic
profile for an orally-administered route of therapy.
Clinical evidence of single-agent activity of Supinoxin was also observed in four patients who
showed stable disease persisting from between 255 and 497 days as of September 14, 2015. At that time,
three of the four patients exhibiting stable disease remained on active treatment. Those patients continue
to be followed in the study.
Based on the progress of the Supinoxin clinical development program and the level of interest
expressed from a number of oncology-focused pharmaceutical companies, we are continuing our
discussions with multiple companies to explore collaborative business structures in an effort to maximize
the potential commercial value of the program.
Pre-Clinical Pipeline
RX-21101: Nano-polymer Anti-cancer Drug
RX-21101 is an investigational anti-cancer nano-polymer drug that we believe can mitigate some
of the limitations of cytotoxic compounds, such as poor solubility and severe adverse reactions.
Conjugating water-soluble and non-toxic N-(2-Hydroxypropyl)methacrylamide
to conventional
anti-cancer compounds may bolster efficacy while lowering toxicity by specific tumor targeting and
increased stability in the body. In June 2015, RX-21101 was selected by the National Cancer Institute's
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Nanotechnology Characterization Laboratory for its pre-clinical characterization program to facilitate the
advancement of RX-21101 towards human clinical trials.
Research and Development Process
We have engaged third-party contract research organizations and other investigators and
collaborators, such as universities and medical institutions, to conduct our pre-clinical studies, toxicology
studies and clinical trials. Engaging third-party contract research organizations is typical practice in our
industry. However, relying on such organizations means that the clinical trials and other studies described
above are being conducted at external locations and that the completion of these trials and studies is not
within our direct control. Trials and studies may be delayed due to circumstances outside our control, and
such delays may result in additional expenses for us.
Competition
We compete against fully integrated pharmaceutical companies and smaller companies that are
collaborating with larger pharmaceutical companies, as well as academic institutions, government
agencies and other public and private research organizations. Many of these competitors, either alone or
together with their collaborative partners, operate larger research and development programs or have
substantially greater financial resources than we do, as well as more experience in:
developing drugs;
undertaking pre-clinical testing and human clinical trials;
obtaining FDA and other regulatory approvals of drugs;
formulating and manufacturing drugs; and
launching, marketing and selling drugs.
Large pharmaceutical companies currently sell both generic and proprietary compounds for the
treatment of cancer. In addition, companies developing oncology therapies represent substantial
competition. Many of these organizations have substantially greater capital resources, larger research and
development staff and facilities, longer drug development history in obtaining regulatory approvals and
greater manufacturing and marketing capabilities than we do. These organizations also compete with us to
attract qualified personnel, parties for acquisitions, joint ventures or other collaborations.
We are aware of products under development by our competitors that target the same indications
as our clinical stage drug candidates. If approved, Archexin could compete with other Akt-1 inhibitors,
such as MK-2206 and GSK-2141795, which are under development by Merck & Company, Inc. and
GlaxoSmithKline, respectively. Archexin will also compete with carbozantinib, a multi-kinase inhibitor
under development by Exelixis for RCC. If approved, RX-3117 could compete with other compounds
with an anti-metabolite mechanism of action in cancers, such as sapacitabine which is under development
by Cyclacel. We are not currently aware of known inhibitors of phosphorylated p68 that would compete
with Supinoxin if Supinoxin were approved. Our competitors may succeed in developing products that
are more safe and/or effective than ours, which could render our product candidates less competitive prior
to recovery by us of expenses incurred with respect to their development.
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Government Regulation
Regulation by governmental authorities in the United States and in other countries is a significant
consideration in our product development, manufacturing and marketing strategies. We expect that all
of our drug candidates will require regulatory approval by the FDA and by similar regulatory authorities
in foreign countries prior to commercialization and will be subjected to rigorous pre-clinical, clinical, and
post-approval testing to demonstrate safety and effectiveness, as well as other significant regulatory
requirements and restrictions in each jurisdiction in which we would seek to market our products.
U.S. federal laws and regulations govern the testing, development, manufacture, quality control, safety,
effectiveness, approval, storage, labeling, record keeping, reporting, distribution, import, export and
marketing of all biopharmaceutical products intended for therapeutic purposes. We believe that we and
the third parties that work with us are in compliance in all material respects with currently applicable
rules and regulations. Those rules and regulations are subject to change, however, and in any event, a
failure to comply could have a material negative impact on our ability to successfully develop and
commercialize our products, and therefore on our financial performance.
Obtaining governmental approvals and maintaining ongoing compliance with applicable
regulations are expected to require the expenditure of significant financial and human resources not
currently at our disposal. We plan to fulfill our short-term needs through consulting agreements and
joint ventures with academic or corporate partners while developing our own internal infrastructure for
long-term corporate growth.
Development and Approval
The process to obtain approval for biopharmaceutical compounds for commercialization in the
United States and many other countries is lengthy, complex and expensive, and the outcome is far from
certain. Although foreign requirements for conducting clinical trials and obtaining approval may be
different than in the United States, they often are equally rigorous and the outcome cannot be predicted
with confidence. A key component of any submission for approval in any jurisdiction is pre-clinical and
clinical data demonstrating the product‘s safety and effectiveness.
Pre-clinical Testing. Before testing any compound in humans in the United States, a company
must develop pre-clinical data, generally including laboratory evaluation of product chemistry and
formulation, as well as toxicological and pharmacological studies in animal species to assess safety and
quality. Certain types of animal studies must be conducted in compliance with the FDA‘s Good
Laboratory Practice (―GLP‖) regulations and the Animal Welfare Act, which is enforced by the
Department of Agriculture.
IND Application. In the United States, FDA regulations require that the person or entity
sponsoring or conducting a clinical study for the purpose of investigating a candidate‘s safety and
effectiveness submit to the FDA an investigational new drug (―IND‖) application, which contains
pre-clinical testing results and provides a basis for the FDA to conclude that there is an adequate basis for
testing the drug in humans. If the FDA does not object to the IND application within 30 days of
submission, the clinical testing proposed in the IND may begin. Even after the IND has gone into effect
and clinical testing has begun, the FDA may put the clinical trials on ―clinical hold,‖ suspending (or in
some cases, ending) them because of safety concerns or for other reasons.
Clinical Trials. Clinical trials involve administering a drug to human volunteers or patients,
under the supervision of a qualified clinical investigator. Clinical trials are subject to extensive
regulation. In the United States, this includes compliance with the FDA‘s bioresearch monitoring
regulations and Good Clinical Practice (―GCP‖) requirements, which establish standards for conducting,
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recording data from, and reporting the results of, clinical trials, with the goal of assuring that the data and
results are credible and accurate and that study participants‘ rights, safety and well-being are protected.
Each clinical trial must be conducted under a protocol that details the study objectives, parameters for
monitoring safety and the efficacy criteria, if any, to be evaluated. The protocol is submitted to the FDA
as part of the IND and reviewed by the agency before the study begins. Additionally, each clinical trial
must be reviewed, approved and conducted under the auspices of an Institutional Review Board (―IRB‖)
at the institution at which the trial is being conducted. The sponsor of a clinical trial, the investigators
and IRBs each must comply with requirements and restrictions that govern obtaining informed consent
from each study subject, complying with the protocol and investigational plan, adequately monitoring the
clinical trial, and timely reporting adverse events. Foreign studies conducted under an IND must meet
the same requirements applicable to studies conducted in the United States. However, if a foreign study
is not conducted under an IND, the data may still be submitted to the FDA in support of a product
application, if the study was conducted in accordance with GCP and the FDA is able to validate the data.
Sponsors of clinical trials are required to make public certain information about active clinical
trials and trial results by posting the information on government or independent websites, such as
http://clinicaltrials.gov. Clinical testing is typically performed in three phases.
In Phase I, the drug is administered to a small number of human subjects to confirm its safety and
to develop detailed profiles of its pharmacological and pharmacokinetic actions (i.e., absorption,
distribution, metabolism, and excretion). Although Phase I trials typically are conducted in healthy
human subjects, in some instances (including, for example, with some cancer therapies) the study subjects
are patients with the targeted disease or condition.
In Phase II, the drug is administered to groups of patients (usually no more than several hundred)
to develop initial data regarding efficacy against the targeted disease and determine the requisite dose and
dose intervals, and generate additional information regarding the drug‘s safety. In a typical development
program, additional animal toxicology studies precede this phase. In some cases, the trial can be split
into Phase IIa and IIb studies in order to test smaller subject pools. Some Phase I clinical studies may
proceed in parallel with some Phase II studies.
In Phase III, the drug is administered to a larger group of patients (usually from several hundred
to several thousand or more). Phase III studies also can include patients with concomitant diseases and
medications. Larger patient populations are evaluated in Phase III at multiple study sites and registration
studies may be conducted concurrently for the sake of time and efficiency. The extensive clinical testing
is intended to obtain additional information about product safety and effectiveness necessary to evaluate
the drug‘s overall risk-benefit profile and to provide a basis for physician labeling. Phase III data often
form the core basis on which the FDA evaluates the product‘s safety and effectiveness when considering
an application to market the drug.
The study sponsor, the FDA or an IRB may suspend or terminate a clinical trial at any time on
various grounds, including a determination that study subjects are being exposed to an unacceptable
health risk. Additionally, success in early-stage clinical trials does not assure success in later-stage
clinical trials, and data from clinical trials are not always conclusive and may be subject to alternative
interpretations that could delay, limit or prevent approval.
NDA Submission and Review. After completing the clinical studies, a sponsor seeking approval
to market a drug in the United States submits to the FDA a New Drug Application (―NDA‖). The NDA
is a comprehensive, multi-volume application intended to demonstrate the product‘s safety and
effectiveness and includes, among other things, pre-clinical and clinical data, information about the drug‘s
composition, the sponsor‘s plans for manufacturing and packaging and proposed labeling. When an
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NDA is submitted, the FDA makes an initial determination as to whether the application is sufficiently
complete to be accepted for review. If the application is not, the FDA may refuse to accept the NDA for
filing and request additional information. A refusal to file, which requires resubmission of the NDA
with the requested additional information, delays review of the application.
FDA performance goals regarding the timeliness of NDA review generally provide for action on
an NDA within 12 months of its submission. That deadline can be extended under certain
circumstances, including by FDA requests for additional information. The targeted action date can also
be shortened to eight months after submission, for products that are intended to treat serious or
life-threatening conditions and demonstrate the potential to address unmet medical needs. Additionally,
the FDA has programs for enhanced communication and consultation and other steps to expedite
submission and consideration of such products. For example, the Fast Track program is intended to
expedite or facilitate the process for reviewing new drugs that demonstrate the potential to address unmet
medical needs involving serious or life-threatening diseases or conditions. If a drug receives Fast Track
designation, the FDA may consider reviewing sections of the NDA on a rolling basis, rather than
requiring the entire application to be submitted to begin the review. Products with Fast Track designation
also may be eligible for more frequent meetings and correspondence with the FDA about the product's
development. Other FDA programs intended to expedite development and review include Accelerated
Approval (i.e., approval on the basis of a surrogate endpoint that is reasonably likely to predict clinical
benefit) and Breakthrough Therapy designation, which is available for drugs under development for
serious or life-threatening conditions and where preliminary clinical evidence shows that the drug may
have substantial improvement on at least one clinically significant endpoint over available therapy. If a
drug receives Breakthrough Therapy designation, it will be eligible for all of the benefits of Fast Track
designation, as well as for more intensive guidance from the FDA on an efficient drug development
program and a commitment from the agency to involve senior FDA managers in such guidance. Even if
a product qualifies for Fast Track designation or Breakthrough Therapy designation, the FDA may later
decide that the product no longer meets the conditions for qualification, and/or may determine that the
product does not meet the standards for approval. We anticipate, but cannot ensure, that our products
will qualify for such programs.
If it concludes that an NDA does not meet the regulatory standards for approval, the FDA
typically issues a Complete Response letter, which communicates the reasons for the agency‘s decision
not to approve the application and may request additional information, including additional clinical data.
An NDA may be resubmitted with the deficiencies addressed, but that does not guarantee approval.
Data from clinical trials are not always conclusive, and the FDA‘s interpretation of data may differ from
the sponsor‘s. Obtaining approval can take years, requires substantial resources and depends on a
number of factors, including the severity of the targeted disease or condition, the availability of
alternative treatments, and the risks and benefits demonstrated in clinical trials. Additionally, as a
condition of approval, the FDA may impose restrictions that could affect the commercial prospects of a
product, such as a Risk Evaluation and Mitigation Strategy, and could require post-approval commitments
to conduct additional studies or conduct surveillance programs to monitor the drug‘s effects.
Moreover, once a product is approved, information about its safety or effectiveness from actual
use can limit or prevent successful commercialization, either because of regulatory action or market
forces. Post-approval modifications to a drug product, such as changes in indications, labeling or
manufacturing processes or facilities, may require development and submission of additional information
or data in a new or supplemental NDA, which would also require FDA approval.
One of our drug candidates, Archexin is an antisense oligonucleotide (―ASO‖) compound. To
date, the FDA has not approved any NDAs for any ASO compounds for cancer treatment; however, the
FDA has approved the ASO compounds fomivirsen (marketed as Vitravene® ) as a treatment for
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cytomegalovirus retinitis, and mipomersen sodium (marketed as Kynamro® ), as a treatment for
homozygous familial hypercholesterolemia. In addition, Archexin is in a drug class known as Akt-1
inhibitors, and drugs from this class have not been approved by the FDA to date.
We have not submitted an NDA for any of our drug candidates.
Exclusivity and Patent Protection. In the United States and elsewhere, certain regulatory
exclusivities and patent rights can provide an approved drug product with protection from certain
competitors‘ products for a period of time and within a certain scope. In the United States, those
protections include exclusivity under the Orphan Drug Act, which is available for drugs intended to treat
rare diseases or conditions, which generally are diseases or conditions that affect fewer than
200,000 persons in the United States. If a sponsor demonstrates that a drug is intended to treat a rare
disease or condition, the FDA grants orphan drug designation to the product for that use. A product that
has received orphan drug designation is eligible for research and development tax credits and is exempt
from user fees under certain circumstances.
Additionally, a drug that is approved for its
orphan-designated indication generally receives seven years of orphan drug exclusivity. During that
period, FDA generally may not approve any other application for a product containing the same active
moiety and proposed for the same indication. There are exceptions, however, most notably when the
later product is shown to be clinically superior to the product with exclusivity. An approved orphan
drug also may qualify for an exemption from the branded prescription drug fee. Products that qualify for
orphan designation may also qualify for other FDA programs that are intended to expedite the
development and approval process and, as a practical matter, clinical trials for orphan products may be
smaller, simply because of the smaller patient population. Nonetheless, the same approval standards
apply to orphan-designated products as for other drugs.
Archexin has received orphan drug designation from the FDA for RCC, glioblastoma, ovarian
cancer, stomach cancer and pancreatic cancer. RX-3117 received orphan drug designation for
pancreatic cancer in September 2014.
Generic Competition. Any drug candidates approved for commercial marketing under an NDA
would be subject to the provisions of the Drug Price Competition and Patent Term Restoration Act of
1984, known as the Hatch-Waxman Act. Among other things, the Hatch-Waxman Act establishes two
abbreviated approval pathways for drug products that are in some way follow-on versions of already
approved NDA products, including generic versions of the approved product, which may be approved
under an Abbreviated New Drug Application by a showing that the generic product is the ―same as‖ the
approved product in key respects. Those abbreviated approval pathways generally are available,
however, after expiration of certain periods of regulatory exclusivity and/or extended patent protection,
which the Hatch-Waxman Act also provides. These protections include: (1) five years of regulatory
exclusivity for a new chemical entity (generally, the first approval of a product containing a particular
active moiety), during which an application for a follow-on product cannot be accepted for review; (2)
three years of exclusivity for the approval of an NDA or supplemental NDA that contains data from new
clinical investigations that were necessary for approval, during which the follow-on product may not
receive final approval; and (3) up to five years‘ extension of the term of a patent covering a drug that
contains an active ingredient not previously approved. The Hatch-Waxman Act also provides a means
for the sponsor of an already approved product to act before approval of the follow-on product to sue to
protect patents claiming the drug substance, drug product, or an approved method of using the drug. The
laws of other key markets likewise create both opportunities for exclusivity periods and patent protections
and the possibility of generic competition once such periods or protections have either expired or have
been successfully challenged by generic entrants.
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Post-Approval Regulation
Once approved, products are subject to continuing extensive regulation by the FDA. If ongoing
regulatory requirements are not met, or if safety problems occur after a product reaches market, the FDA
may take actions to change the conditions under which the product is marketed, including suspending or
even withdrawing approval. In addition to FDA regulation, the healthcare industry, and therefore our
business, is also subject to extensive federal, state, local and foreign regulation.
Good Manufacturing Practices. Companies engaged in manufacturing drug products or their
components must comply with applicable current Good Manufacturing Practice (―cGMP‖) requirements,
which include requirements regarding organization and training of personnel, building and facilities,
equipment, control of components and drug product containers, closures, production and process controls,
packaging and labeling controls, holding and distribution, laboratory controls and records and reports.
The FDA inspects equipment, facilities and manufacturing processes before approval and conducts
periodic re-inspections after approval. Failure to comply with applicable cGMP requirements or the
conditions of the product‘s approval may lead the FDA to take administrative enforcement action.
Although we periodically monitor the FDA compliance of the third parties on which we rely for
manufacturing our drug products, we cannot be certain that our present or future third-party
manufacturers will consistently comply with cGMP or other applicable FDA regulatory requirements.
Sales and Marketing. Once a product is approved, its advertising, promotion and marketing will
be subject to close regulation, including with regard to promotion to healthcare practitioners,
direct-to-consumer advertising, communications regarding unapproved uses, industry-sponsored scientific
and educational activities and promotional activities involving the internet. In addition to FDA
restrictions on marketing of pharmaceutical products, state and federal fraud and abuse laws have been
applied to restrict certain marketing practices in the pharmaceutical industry in recent years. Some of
the pertinent laws have not been definitively interpreted by the regulatory authorities or the courts, and
their provisions are open to a variety of interpretations. In addition, these laws and their interpretations are
subject to change.
Other Requirements. Companies that manufacture or distribute drug products pursuant to
approved NDAs must meet numerous other regulatory requirements, including adverse event reporting,
submission of periodic reports, and record-keeping obligations
Fraud and Abuse Laws. At such time as we market, sell and distribute any products for which
we obtain marketing approval, it is possible that our business activities could be subject to scrutiny and
enforcement under one or more federal or state health care fraud and abuse laws and regulations, which
could affect our ability to operate our business. These restrictions under applicable federal and state
health care fraud and abuse laws and regulations that may affect our ability to operate include:
The federal Anti-Kickback Law, which prohibits, among other things, knowingly or willingly
offering, paying, soliciting or receiving remuneration, directly or indirectly, in cash or in kind,
to induce or reward the purchasing, leasing, ordering or arranging for or recommending the
purchase, lease or order of any health care items or service for which payment may be made,
in whole or in part, by federal healthcare programs such as Medicare and Medicaid. This
statute has been interpreted to apply to arrangements between pharmaceutical companies on
one hand and prescribers, purchasers and formulary managers on the other. Further, the
Patient Protection and Affordable Care Act, as amended by the Health Care and Education
Reconciliation Act of 2010, together the Affordable Care Act, clarified among other things
that liability may be established under the federal Anti-Kickback Law without proving actual
knowledge of the statute or specific intent to violate it. In addition, the Affordable Care Act
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amended the Social Security Act to provide that the government may assert that a claim
including items or services resulting from a violation of the federal Anti-Kickback Law
constitutes a false or fraudulent claim for purposes of the federal civil False Claims Act.
Although there are a number of statutory exemptions and regulatory safe harbors to the
federal Anti-Kickback Law protecting certain common business arrangements and activities
from prosecution or regulatory sanctions, the exemptions and safe harbors are drawn
narrowly, and practices that do not fit squarely within an exemption or safe harbor, or for
which no exception or safe harbor is available, may be subject to scrutiny;
The federal civil False Claims Act, which prohibits, among other things, individuals or
entities from knowingly presenting, or causing to be presented, a false or fraudulent claim for
payment of government funds or knowingly making, using or causing to be made or used, a
false record or statement material to an obligation to pay money to the government or
knowingly concealing or knowingly and improperly avoiding, decreasing or concealing an
obligation to pay money to the federal government. Many pharmaceutical and other
healthcare companies have been investigated and have reached substantial financial
settlements with the federal government under the civil False Claims Act for a variety of
alleged improper marketing activities, including: providing free product to customers with the
expectation that the customers would bill federal programs for the product; providing sham
consulting fees, grants, free travel and other benefits to physicians to induce them to prescribe
the company‘s products; and inflating prices reported to private price publication services,
which are used to set drug payment rates under government healthcare programs. In addition,
in recent years the government has pursued civil False Claims Act cases against a number of
pharmaceutical companies for causing false claims to be submitted as a result of the
marketing of their products for unapproved, and thus non-reimbursable, uses. Pharmaceutical
and other healthcare companies also are subject to other federal false claim laws, including,
among others, federal criminal healthcare fraud and false statement statutes that extend to
non-government health benefit programs;
Analogous state laws and regulations, such as state anti-kickback and false claims laws, may
apply to items or services reimbursed under Medicaid and other state programs or, in several
states, apply regardless of the payor. Some state laws also require pharmaceutical companies
to report expenses relating to the marketing and promotion of pharmaceutical products and to
report gifts and payments to certain healthcare providers in the states. Other states prohibit
providing meals to prescribers or other marketing related activities. In addition, California,
Connecticut, Nevada and Massachusetts require pharmaceutical companies to implement
compliance programs or marketing codes of conduct.
The federal Physician Payment Sunshine Act, being implemented as the Open Payments
Program, requires certain pharmaceutical manufacturers to engage in extensive tracking of
payments and other transfers of value to physicians and teaching hospitals, and to submit
such data to the Centers for Medicare and Medicaid Services (―CMS‖), which will then make
all of this data publicly available on the CMS website. Pharmaceutical manufacturers with
products for which payment is available under Medicare, Medicaid or the State Children‘s
Health Insurance Program are required to track reportable payments and must submit a report
to CMS on or before the 90th day of each calendar year disclosing reportable payments made
in the previous calendar year. Failure to comply with the reporting obligations may result in
civil monetary penalties;
The federal Foreign Corrupt Practices Act of 1997 and other similar anti-bribery laws in other
jurisdictions generally prohibit companies and their intermediaries from providing money or
anything of value to officials of foreign governments, foreign political parties, or
international organizations with the intent to obtain or retain business or seek a business
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advantage. Recently, there has been a substantial increase in anti-bribery law enforcement
activity by U.S. regulators, with more frequent and aggressive investigations and enforcement
proceedings by both the Department of Justice and the U.S. Securities and Exchange
Commission (the ―SEC‖). Violations of United States or foreign laws or regulations could
result in the imposition of substantial fines, interruptions of business, loss of supplier, vendor
or other third-party relationships, termination of necessary licenses and permits and other
legal or equitable sanctions. Other internal or government investigations or legal or regulatory
proceedings, including lawsuits brought by private litigants, may also follow as a
consequence.
Violations of any of the laws described above or any other governmental regulations are punishable by
significant civil, criminal and administrative penalties, damages, fines and exclusion from
government-funded healthcare programs, such as Medicare and Medicaid. Although compliance
programs can mitigate the risk of investigation and prosecution for violations of these laws, the risks
cannot be entirely eliminated. Moreover, achieving and sustaining compliance with applicable federal
and state privacy, security and fraud laws may prove costly.
Privacy Laws. We are also subject to laws and regulations covering data privacy and the
protection of health-related and other personal information. The legislative and regulatory landscape for
privacy and data protection continues to evolve, and there has been an increasing focus on privacy and
data protection issues which may affect our business, including recently enacted laws in all jurisdictions
where we operate. Numerous federal and state laws, including state security breach notification laws,
state health information privacy laws and federal and state consumer protection laws, govern the
collection, use and disclosure of personal information. In addition, if we successfully commercialize our
drug candidates, we may obtain patient health information from healthcare providers who prescribe our
products and research institutions we collaborate with, and they are subject to privacy and security
requirements under the Health Insurance Portability and Accountability Act of 1996, as amended by the
Health Information Technology for Economic and Clinical Health Act (―HIPAA‖). Although we are not
directly subject to HIPAA other than with respect to providing certain employee benefits, we could
potentially be subject to criminal penalties if we knowingly obtain or disclose individually identifiable
health information maintained by a HIPAA-covered entity in a manner that is not authorized or permitted
by HIPAA.
Coverage and Reimbursement
Significant uncertainty exists as to the coverage and reimbursement status of any drug candidates
for which we may obtain regulatory approval. The regulations that govern marketing approvals, pricing
and reimbursement for new drug products vary widely from country to country. Current and future
legislation may significantly change the approval requirements in ways that could involve additional costs
and cause delays in obtaining approvals. Some countries require approval of the sale price of a drug before
it can be marketed. In many countries, the pricing review period begins after marketing or product licensing
approval is granted. In some foreign markets, prescription pharmaceutical pricing remains subject to
continuing governmental control even after initial approval is granted. As a result, we might obtain
marketing approval for a product in a particular country, but then be subject to price regulations that delay
our commercial launch of the product, possibly for lengthy time periods, which could negatively impact the
revenues we are able to generate from the sale of the product in that particular country. Adverse pricing
limitations may hinder our ability to recoup our investment in one or more product candidates even if our
product candidates obtain marketing approval.
Our ability to commercialize any products successfully also will depend in part on the extent to
which coverage and adequate reimbursement for these products and related treatments will be available in a
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timely manner from government third-party payors, including government healthcare programs such as
Medicare and Medicaid, commercial health insurers and managed care organizations. Government
authorities and other third-party payors, such as private health insurers and health maintenance
organizations, determine which medications they will cover and establish reimbursement levels.
Third-party payors may limit coverage to specific products on an approved list, or formulary, which
might not include all of the FDA approved products for a particular indication. The process for
determining whether a payor will provide coverage for a product may be separate from the process for
setting the price or reimbursement rate that the payor will pay for the product once coverage is approved.
A primary trend in the U.S. healthcare industry and elsewhere is cost containment. Government
healthcare programs and other third-party payors are increasingly challenging the prices charged for
medical products and services and examining the medical necessity and cost-effectiveness of medical
products and services, in addition to their safety and efficacy, and have attempted to control costs by
limiting coverage and the amount of reimbursement for particular medications. Increasingly, third-party
payors are requiring that drug companies provide them with predetermined discounts from list prices and
are challenging the prices charged for medical products. We cannot be sure that coverage and
reimbursement will be available promptly or at all for any product that we commercialize and, if
reimbursement is available, what the level of reimbursement will be. Moreover, eligibility for coverage and
reimbursement does not imply that any drug will be paid for in all cases. Limited coverage may impact the
demand for, or the price of, any product candidate for which we obtain marketing approval. If coverage and
reimbursement are not available or reimbursement is available only to limited levels, we may not
successfully commercialize any product candidate for which we obtain marketing approval.
Payors also are increasingly considering new metrics as the basis for reimbursement rates, such as
average sales price (―ASP‖), average manufacturer price (―AMP‖) and actual acquisition cost. The existing
data for reimbursement based on these metrics is relatively limited, although certain states have begun to
survey acquisition cost data for the purpose of setting Medicaid reimbursement rates. CMS surveys and
publishes retail community pharmacy acquisition cost information in the form of National Average Drug
Acquisition Cost (―NADAC‖) files to provide state Medicaid agencies with a basis of comparison for their
own reimbursement and pricing methodologies and rates. It may be difficult to project the impact of these
evolving reimbursement mechanics on the willingness of payors to cover our products for which we receive
regulatory approval.
If we successfully commercialize any of our products, we may participate in the Medicaid Drug
Rebate Program. Participation is required for federal funds to be available for our products under
Medicaid and Medicare Part B. Under the Medicaid Drug Rebate Program, we would be required to pay a
rebate to each state Medicaid program for our covered outpatient drugs that are dispensed to Medicaid
beneficiaries and paid for by a state Medicaid program as a condition of having federal funds being made
available to the states for our drugs under Medicaid and Part B of the Medicare program.
Federal law requires that any company that participates in the Medicaid Drug Rebate Program also
participate in the Public Health Service‘s 340B drug pricing program in order for federal funds to be
available for the manufacturer‘s drugs under Medicaid and Medicare Part B. The 340B drug pricing
program requires participating manufacturers to agree to charge statutorily-defined covered entities no
more than the 340B ―ceiling price‖ for the manufacturer‘s covered outpatient drugs. These 340B covered
entities include a variety of community health clinics and other entities that receive health services grants
from the Public Health Service, as well as hospitals that serve a disproportionate share of low-income
patients.
In addition, in order to be eligible to have its products paid for with federal funds under the
Medicaid and Medicare Part B programs and purchased by certain federal agencies and grantees, a
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manufacturer also must participate in the Department of Veterans Affairs Federal Supply Schedule
(―FSS‖), pricing program, established by Section 603 of the Veterans Health Care Act of 1992 (the
―VHCA‖). Under this program, the manufacturer is obligated to make its innovator and single source
products available for procurement on an FSS contract and charge a price to four federal agencies,
Department of Veterans Affairs, Department of Defense (―DoD‖), Public Health Service, and Coast Guard,
that is no higher than the statutory Federal Ceiling Price. Moreover, pursuant to regulations issued by the
DoD. TRICARE Management Activity (―TMA‖), now the Defense Health Agency (―DHA‖), to implement
Section 703 of the National Defense Authorization Act for Fiscal Year 2008, manufacturers are required to
provide rebates on utilization of their innovator and single source products that are dispensed to TRICARE
beneficiaries by TRICARE network retail pharmacies. The formula for determining the rebate is
established in the regulations and is based on the difference between the annual non-federal average
manufacturer price and the Federal Ceiling Price (these price points are required to be calculated by us
under the VHCA). The requirements under the 340B, FSS, and TRICARE programs could reduce the
revenue we may generate from any products that are commercialized in the future and could adversely
affect our business and operating results.
There may be significant delays in obtaining coverage and reimbursement for newly approved
drugs, and coverage may be more limited than the purposes for which the drug is approved by the FDA or
comparable foreign regulatory authorities. Moreover, eligibility for coverage and reimbursement does not
imply that a drug will be paid for in all cases or at a rate that covers our costs, including research,
development, manufacture, sale and distribution. Interim reimbursement levels for new drugs, if applicable,
may also not be sufficient to cover our costs and may only be temporary. Reimbursement rates may vary
according to the use of the drug and the clinical setting in which it is used, may be based on reimbursement
levels already set for lower cost drugs and may be incorporated into existing payments for other services.
Net prices for drugs may be reduced by mandatory discounts or rebates required by government healthcare
programs or private payors and by any future relaxation of laws that presently restrict imports of drugs from
countries where they may be sold at lower prices than in the United States. Limited coverage may impact
the demand for, or the price of, any product candidate for which we obtain marketing approval.
Third-party payors also may seek additional clinical evidence, including expensive pharmacoeconomic
studies, beyond the data required to obtain marketing approval, demonstrating clinical benefits and value
in specific patient populations, before covering our products for those patients. If reimbursement is
available only for limited indications, we may not be able to successfully commercialize any product
candidate for which we obtain marketing approval. Our inability to promptly obtain coverage and
profitable reimbursement rates from both government-funded and private payors for any approved products
that we develop could have a material adverse effect on our operating results, our ability to raise capital
needed to commercialize products and our overall financial condition.
United States Healthcare Reform
The United States and many foreign jurisdictions have enacted or proposed legislative and
regulatory changes affecting the healthcare system that could prevent or delay marketing approval of our
product candidates, restrict or regulate post-approval activities and affect our ability to profitably sell any
product candidate for which we obtain marketing approval. The United States government, state
legislatures and foreign governments also have shown significant
implementing
cost-containment programs to limit the growth of government-paid healthcare costs, including price
controls, restrictions on reimbursement and requirements for substitution of generic products for branded
prescription drugs.
interest
in
In recent years, Congress has considered reductions in Medicare reimbursement levels for drugs
administered by physicians. CMS, the agency that administers the Medicare and Medicaid programs, also
has authority to revise reimbursement rates and to implement coverage restrictions for some drugs. Cost
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reduction initiatives and changes in coverage implemented through legislation or regulation could decrease
utilization of and reimbursement for any approved products, which in turn would affect the price we can
receive for those products. While Medicare regulations apply only to drug benefits for Medicare
beneficiaries, private payors often follow Medicare coverage policy and payment limitations in setting their
own reimbursement rates. Therefore, any reduction in reimbursement that results from federal legislation or
regulation may result in a similar reduction in payments from private payors.
In March 2010, President Obama signed into law the Affordable Care Act. This law substantially
changes the way healthcare is financed by both governmental and private insurers, and significantly
impacts the pharmaceutical industry. The Affordable Care Act is intended to broaden access to health
insurance, reduce or constrain the growth of healthcare spending, enhance remedies against healthcare
fraud and abuse, add new transparency requirements for healthcare and health insurance industries, impose
new taxes and fees on pharmaceutical and medical device manufacturers, and impose additional health
policy reforms. Among other things, the Affordable Care Act expanded manufacturers‘ rebate liability
under the Medicaid Drug Rebate Program by increasing the minimum Medicaid rebate for both branded
and generic drugs, expanded the 340B program, and revised the definition of AMP, which could increase
the amount of Medicaid drug rebates manufacturers are required to pay to states. The legislation also
extended Medicaid drug rebates, previously due only on fee-for-service Medicaid utilization, to include the
utilization of Medicaid managed care organizations as well and created an alternative rebate formula for
certain new formulations of certain existing products that is intended to increase the amount of rebates due
on those drugs. On February 1, 2016, CMS issued final regulations to implement the changes to the
Medicaid Drug Rebate program under the Affordable Care Act. These regulations become effective on
April 1, 2016. It appears likely that the Affordable Care Act will continue the pressure on pharmaceutical
pricing, especially under the Medicare and Medicaid programs, and may also increase our regulatory
burdens and operating costs.
The Affordable Care Act requires pharmaceutical manufacturers of branded prescription drugs to
pay a branded prescription drug fee to the federal government. Each individual pharmaceutical
manufacturer pays a prorated share of the branded prescription drug fee of $3.0 billion in 2016, based on the
dollar value of its branded prescription drug sales to certain federal programs identified in the law.
Furthermore, the new law requires manufacturers to provide a 50% discount off the negotiated price of
prescriptions filled by beneficiaries in the Medicare Part D coverage gap, referred to as the ―donut hole.‖
The Affordable Care Act also expanded the Public Health Service‘s 340B drug pricing discount
program. As noted above, the 340B drug pricing program requires participating manufacturers to agree to
charge statutorily-defined covered entities no more than the 340B ―ceiling price‖ for the manufacturer‘s
covered outpatient drugs. The Affordable Care Act expanded the 340B program to include additional
types of covered entities: certain free-standing cancer hospitals, critical access hospitals, rural referral
centers and sole community hospitals, each as defined by the Affordable Care Act. The Affordable Care
Act exempts ―orphan drugs‖—those designated under section 526 of the Food, Drug, and Cosmetic
Act—from the ceiling price requirements for these newly-eligible entities. Because the 340B ceiling price
is determined based on AMP and Medicaid drug rebate data, the revisions to the Medicaid rebate formula
and AMP definition described above could cause the required 340B discounts to increase.
In addition, other legislative changes have been proposed and adopted since the Affordable Care
Act was enacted. For example, recent legislative enactments have resulted in Medicare payments being
subject to a two percent reduction, referred to as sequestration, until 2025. Continuation of sequestration
or enactment of other reductions in Medicare reimbursement for drugs could affect our ability to achieve
a profit on any candidate products that are approved for marketing.
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We expect that the Affordable Care Act, as well as other healthcare reform measures that have and
may be adopted in the future, may result in more rigorous coverage criteria and in additional downward
pressure on the price that we receive for any approved product and could seriously harm our future
revenues. Any reduction in reimbursement from Medicare, Medicaid, or other government programs may
result in a similar reduction in payments from private payors. The implementation of cost containment
measures or other healthcare reforms may prevent us from being able to generate revenue, attain
profitability or commercialize our products.
Foreign Regulation
In addition to regulations in the United States, we will be subject to a number of significant
regulations in other jurisdictions regarding clinical trials, approval, manufacturing, marketing and
promotion and safety reporting. These requirements and restrictions vary from country to country, but
in many instances are similar to the United States requirements, and failure to comply with them could
have the same negative effects as noncompliance in the United States.
Sales and Marketing
We do not currently have the sales and marketing infrastructure in place that would be necessary
to sell and market products. As our drug candidates progress in clinical trials, we may build the
commercial infrastructure that would be needed to successfully market and sell any successful drug
candidate. For drug candidates that may require larger clinical trials or sales efforts, we intend to establish
strategic alliances and partnerships with large pharmaceutical companies during the development process.
Research Technologies
Our research technologies are focused on our proprietary multi-target aimed ligands platform and
nano-based drug delivery, which are described further below. For a discussion of collaboration
arrangements pursuant to which we obtain research and development services from universities, research
institutions and other organizations, see ―Collaboration and License Agreements‖ in this Item 1.
The Inhibitors of Multi-Expression Signals (TIMES)
TIMES is our platform for discovering ligands, which are molecules coordinated to a central
atom or molecule in a larger chemical complex, that target multi-expression signals. Because cancer is a
complex disease caused by multiple factors as well as genetic modifications, cancer treatment involves a
combination of drugs with different mechanisms of action, which may result in compounding the degree
and extent of toxicities to which a patient is exposed. TIMES permits us to control multiple targets
important for cancer proliferation with a single agent. In doing so, we utilize a proprietary,
genomics-based integrated, gene expression system to identify potentially important targets that control
multiple genes or signaling events in cancer cells.
3-D Gateway of Ligand Discovery (3-D GOLD)
3-D GOLD is a drug discovery platform that integrates three-dimensional (―3D‖) molecular
modeling, databases of chemicals and proteins and ligand filtering and generation. The chemical database
contains 3D structures of approximately seven million compounds. Our proprietary docking tools
quantitative structure-activity relationship tool for innovative discovery are parts of the platform. Ligand
filtering highlights similarities in pharmacophore and 3D fingerprinting, while ligand generation helps
optimize the identification of such similarities.
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Nano-medicine Drug Delivery
We have developed unique proprietary drug delivery nano-systems that we believe may increase
the availability of a drug at the disease site, minimize adverse reactions, and provide longer duration of
action. We are currently testing multiple nanoliposomal- and nanopolymer-based anti-cancer drugs.
RX-21101 is an investigational nanoliposomal-based drug.
Manufacturing and Distribution
We have no experience in drug formulation or manufacturing, and we lack the resources and
expertise to formulate or manufacture our own drug candidates internally. Therefore, we rely on
third-party expertise to support us in this area. We have entered into contracts with third-party
manufacturers to manufacture, supply, store and distribute supplies of our drug candidates for our clinical
trials. If any of our drug candidates receive FDA approval, we expect to rely on third-party contractors to
manufacture our drugs. We have no current plans to build internal manufacturing capacity for any
product, and we have no long-term supply arrangements.
Intellectual Property
We generally seek proprietary patent and intellectual property (―IP‖) protection for our drug
candidates, processes, and other know-how. In addition to patent protection, we rely upon trade secrets,
know-how, continuing technological innovation and licensing opportunities to develop and safeguard and
maintain our IP.
We hold U.S. and foreign patents for our drug candidates that expire from 2020 to 2030. We hold
U.S. patents for Archexin, RX-3117 and Supinoxin. We also hold multiple foreign patents for Archexin,
RX-3117, and Supinoxin.
In 2015, we were granted a U.S. patent from the United States Patent and Trademark Office for
claims related to the synthesis of our novel anti-cancer investigational drug candidate, RX-3117.
Additional U.S. and foreign patent applications related to Archexin, RX-3117, Supinoxin, and RX-21101
are pending.
Collaboration and License Arrangements
We have numerous collaborative research and development relationships with universities,
research institutions pharmaceutical companies and other organizations.
The University of Maryland Baltimore (“UMB”)
In July 2013, we entered into an exclusive license agreement with UMB for a novel drug delivery
platform, Nano-Polymer-Drug Conjugate Systems. This platform combines existing chemotherapeutic
agents with a proprietary polymer carrier that contains a signaling moiety to direct the agents into a
tumor. RX-21101 is our first drug candidate utilizing this platform and is a conjugated form of
docetaxel, a common chemotherapy agent. This agreement requires us to make payments to UMB if
RX-21101 or any other products developed from the licensed delivery platform achieve development
milestones.
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The Ohio State University
In October 2013, we entered into an exclusive license agreement with the Ohio State Innovation
Foundation, an affiliate of The Ohio State University, for a novel oligonucleotide drug delivery platform,
Lipid-Coated Albumin Nanoparticle (―LCAN‖). The LCAN platform incorporates both cationic lipid
and cationized albumin that can form an electrostatic complex with oligonucleotides and be
co-encapsulated by lipids. The agreement requires us to make payments to the Ohio State Innovation
Foundation if any products from the licensed delivery platform achieve development milestones.
Korea Research Institute of Chemical Technology (“KRICT”)
In June 2009, we entered into a license agreement with KRICT to acquire all intellectual property
related to quinoxaline-piperazine derivatives, which includes Supinoxin. We paid an initial license fee
of $100,000 in July 2009, and will pay $1,000,000 to KRICT upon marketing approval from the FDA for
the first commercial product stemming from the agreement.
Rexgene Biotech Co., Ltd. (“Rexgene”)
In February 2003, we entered into a research collaboration agreement with Rexgene, which is
engaged in the development of pharmaceutical products in Asia. Rexgene has agreed to assist us with the
research, development and clinical trials necessary for registration of Archexin in Asia. Under the
agreement, Rexgene has exclusive rights to license, sublicense, make, have made, use, sell and import
Archexin in Asia. In accordance with the agreement, Rexgene paid us a one-time fee of $1,500,000 in
2003. Rexgene also agreed to pay us a royalty fee of 3% of net sales of licensed products related to
Archexin in all countries in Asia by Rexgene or any sublicensee of Rexgene.
The agreement expires upon the last to expire of all U.S. and foreign patents presently or in the
future issued that cover Archexin, or, if no licensed patent is issued, within 20 years from the date of
execution of the agreement. A breach of the agreement by either party give the non-breaching party the
right to terminate the agreement upon 90 days written notice of termination specifying the obligations
breached, provided that within said 90 days the breaching party does not remedy the breach.
Total Research and Development Costs
We have incurred research and development costs of $12,148,226, $7,015,901 and $3,253,139 for
the years ended December 31, 2015, 2014 and 2013 respectively. Research and development costs
primarily consist of clinical trials and pre-clinical development costs, as well as payroll costs for research
and development personnel.
Employees
We currently have 20 full-time employees, all of whom are based either at our Rockville,
Maryland office or our Gaithersburg, Maryland lab facility. Our employees are not covered by any
collective bargaining agreement and we have never experienced a work stoppage. We believe our
relationships with our employees are satisfactory.
Available Information
Under the Securities Exchange Act of 1934, as amended (the ―Exchange Act‖), we are required to
file annual, quarterly and current reports, proxy statements and other information with the SEC. Any
document we file with the SEC may be read and copied at the SEC‘s Public Reference Room at 100 F
Street, N.E., Washington, D.C. 20549. Please call the SEC at (800) SEC-0330 for further information about
21
the public reference room. The SEC maintains a website at www.sec.gov that contains reports, proxy and
information statements and other information regarding issuers that file electronically with the SEC.
We make available, free of charge, on our website at www.rexahn.com our Annual Report on Form
10-K, Quarterly Reports on Form 10-Q and Current Reports on Form 8-K and all amendments thereto, as
soon as reasonably practicable after they are filed with or furnished to the SEC. Investors are encouraged to
access these reports and the other information about our business on our website. Information found on our
website is not part of this Annual Report on Form 10-K (this ―Annual Report‖). We will also provide copies
of this Annual Report, free of charge, upon written request to the Investor Relations Department at our main
address, 15245 Shady Grove Road, Suite 455, Rockville MD, 20850.
Also posted on our website, and available in print upon written request of any shareholder to our
Investor Relations Department, are the charters of the standing committees of our Board.
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Item 1A. Risk Factors.
You should carefully consider the risks described below together with the other information included in
this Form 10-K. Our business, financial condition or results of operations could be adversely affected by
any of these risks. If any of these risks occur, the value of our common stock could decline.
Risks Related to Our Financial Position and Capital Needs
We currently have no product revenues, have incurred negative cash flows from operations since
inception and will need to raise additional capital to operate our business.
To date, we have generated no product revenues and have incurred negative cash flow from
operations. Until we receive approval from the FDA or other regulatory authorities for our drug
candidates, we cannot sell our drugs and will not have product revenues. We expect to continue to incur
significant development and other expenses related to our ongoing operations. Therefore, for the
foreseeable future, we will have to fund all of our operations and capital expenditures from the net
proceeds of equity or debt offerings, cash on hand, licensing fees and grants, if any. If we are not able to
raise sufficient funds, we will have to reduce our research and development activities. We will first reduce
research and development activities associated with our pre-clinical compounds. To the extent necessary,
we will then reduce our research and development activities related to some or all of our clinical stage
product candidates.
Unforeseen events, difficulties, complications and delays may occur that could cause us to utilize
our existing capital at a faster rate than projected, including the progress of our research and development
efforts, the cost and timing of regulatory approvals and the costs of protecting our intellectual property
rights. We may seek additional financing to implement and fund other drug candidate development,
clinical trial and research and development efforts, including clinical trials for other new drug candidates,
as well as other research and development projects.
We will need additional financing to continue to develop our drug candidates, which may not be
available on favorable terms, if at all. If we are unable to secure additional financing in the future on
acceptable terms, or at all, we may be unable to complete our planned pre-clinical and clinical trials or
obtain approval of our drug candidates from the FDA and other regulatory authorities. In addition, we
may be forced to reduce or discontinue product development or product licensing, reduce or forego sales
and marketing efforts and forego attractive business opportunities in order to improve our liquidity to
enable us to continue operations. Any additional sources of financing will likely involve the sale of our
equity securities or securities convertible into our equity securities, which may have a dilutive effect on
our stockholders.
We are not currently profitable and may never become profitable.
To date, we have generated no product revenues and have incurred negative cash flow from
operations. Our accumulated deficit as of December 31, 2015 and 2014 was $105,716,864 and
$91,332,308, respectively. For the years ended December 31, 2015, 2014, and 2013, we had net losses of
$14,384,556, $18,521,601 and $9,499,424, respectively. Even if we succeed in developing and
commercializing one or more of our drug candidates, we expect to incur substantial losses for the
foreseeable future and may never become profitable. We also expect to continue to incur significant
operating and capital expenditures and anticipate that our expenses will increase substantially in the
foreseeable future, based on the following considerations:
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continued pre-clinical development and clinical trials for our current and new drug candidates;
finding suitable partners to help us research, develop and commercialize new drug candidates;
efforts to seek regulatory approvals for our drug candidates;
implementing additional internal systems and infrastructure;
in-licensing additional technologies to develop; and
hiring additional personnel or entering into relationships with third parties to perform functions
that we are unable to perform on our own.
We also expect to continue to experience negative cash flow for the foreseeable future as we fund
our operations and capital expenditures. Until we have the capacity to generate revenues, we are relying
upon outside funding resources to fund our cash flow requirements. If these resources are depleted or
unavailable, we may be unable to continue to expand our operations or otherwise capitalize on our
business opportunities, and our business, financial condition and results of operations would be materially
adversely affected.
We have a limited operating history, and we have not demonstrated an ability to commercialize drug
candidates.
We are a clinical-stage company with a limited number of drug candidates. We currently do not
have any products that have gained regulatory approval, and we have not demonstrated an ability to
perform the functions necessary for the successful commercialization of any of our drug candidates. The
successful commercialization of our drug candidates will require us to first perform a variety of functions,
including:
conducting pre-clinical and clinical trials;
participating in regulatory approval processes;
formulating and manufacturing products; and
conducting sales and marketing activities.
To date, our operations have been limited to organizing and staffing the Company, acquiring,
developing and securing our proprietary technology, and undertaking drug candidate research and
development, including pre-clinical trials and clinical trials of our principal drug candidates. These
operations provide a limited basis for assessing our ability to commercialize drug candidates.
Several of our drug candidates are in clinical trials, which are very expensive, time-consuming and
difficult to design and implement.
Our drug candidates are in various stages of development and require extensive clinical testing.
Such testing is expensive and time-consuming and requires specialized knowledge and expertise.
Archexin entered a Phase IIa clinical trial in January 2014, RX-3117 entered a Phase Ib clinical trial in
January 2014, and Supinoxin entered a Phase I clinical trial in August 2013.
24
Human clinical trials are very expensive and difficult to design and implement, in part because
they are subject to rigorous regulatory requirements. The clinical trial process is also time-consuming, and
the outcome is not certain; the results of pre-clinical studies and early clinical trials may not be predictive
of the results of later-stage clinical trials. We estimate that clinical trials of our current drug candidates
will take multiple years to complete. Furthermore, failure can occur at any stage of a clinical trial, and we
could encounter problems that cause us to abandon or repeat clinical trials. The commencement and
completion of clinical trials may be delayed or precluded by a number of factors, including:
delay or failure in reaching agreement with the FDA or a foreign regulatory authority on the
design of a given trial, or in obtaining authorization to commence a trial;
delay or failure in reaching agreement on acceptable terms with prospective contract research
organizations and clinical trial sites;
delay or failure in obtaining approval of an IRB to conduct a clinical trial at a given site;
withdrawal of clinical trial sites from our clinical trials as a result of changing standards of care of
the ineligibility of a site to participate;
delay or failure in recruiting and enrolling study subjects;
delay or failure in having subjects complete a clinical trial or return for post-treatment follow up;
clinical sites or investigators deviating from trial protocol, failing to conduct the trial in
accordance with applicable regulatory requirements, or dropping out of a trial;
inability to identify and maintain a sufficient number of trial sites;
failure of third-party clinical trial managers to meet their contractual obligations or deadlines;
the need to modify a study protocol;
unforeseen safety issues;
emergence of dosing issues;
lack of effectiveness during clinical trials;
change in the standard of care of the indication being studied;
reliance on third-party suppliers for the clinical trial supply of drug candidates;
inability to monitor patients adequately during or after treatment;
lack of sufficient funding to finance the clinical trials; and
changes in governmental regulations or administrative action.
We, the FDA or an IRB may suspend a clinical trial at any time if it appears that we are exposing
participants to unacceptable health risks or if the FDA finds deficiencies in our IND applications or the
25
conduct of these trials. Additionally, we may have difficulty enrolling patients in our clinical trials. If
we experience such difficulties, we may not be able to complete a clinical trial or we may experience
significant delays in completing a clinical trial.
If the results of our clinical trials fail to support the approval of any of our drug candidates, the
completion of development of that candidate may be significantly delayed, or we may be forced to
abandon development altogether, which will significantly impair our ability to generate product
revenues.
Even if our clinical trials are completed as planned, we cannot be certain that clinical results will
support approval of our drug candidates. Success in pre-clinical testing and early clinical trials does not
ensure that later clinical trials will be successful, and we cannot be sure that the results of later clinical
trials will replicate the results of prior clinical trials and pre-clinical testing. The clinical trial process may
fail to demonstrate that one or more of our drug candidates is safe and effective for indicated uses. As a
result, we may have to conduct additional clinical trials or may decide to abandon a drug candidate, in
which case we may never recognize any revenue related to such candidate. Standard of care treatments
may change, which may require additional clinical trials. Repeating clinical trials or conducting additional
clinical trials will increase our development costs and delay the filing of an NDA and, ultimately, delay
our ability to commercialize our drug candidates and generate product revenues.
We may not obtain the necessary U.S. or worldwide regulatory approvals to commercialize our drug
candidates, and we cannot guarantee how long it will take the FDA or other comparable regulatory
agencies to review applications for our drug candidates.
We will need FDA approval to commercialize our drug candidates in the United States and
approvals from the comparable regulatory authorities to commercialize our drug candidates in foreign
jurisdictions.
The time it takes to obtain approval, either in the United States or foreign jurisdictions, is
unpredictable, but typically takes many years, depending upon a variety of factors, including the type,
complexity and novelty of the drug candidate. Obtaining approval requires substantial resources and is
subject to regulatory authorities‘ substantial discretion. In addition, approval policies, regulations or the
type and amount of clinical data necessary to gain approval may change during the course of a product
candidate‘s development and may vary among jurisdictions. We cannot guarantee that any of our drug
candidates will ultimately be approved by the FDA or any other regulatory authority, or the length of time
obtaining approval will take. One of our drug candidates, Archexin, is an ASO compound. To date, the
FDA has approved very few ASO compounds. In addition, Archexin is in the drug class known as
Akt-1 inhibitors that to date have not been approved by the FDA, nor have we submitted an NDA for an
Akt-1 inhibitor.
Our product candidates could fail to receive regulatory approval from the FDA or a comparable
foreign authority for a variety of reasons, including:
disagreement with the design or implementation of our clinical trials;
failure to demonstrate to the authority‘s satisfaction that the product candidate is safe and
effective for the proposed indication;
failure of clinical trials to meet the level of statistical significance required for approval;
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failure to demonstrate that the product‘s benefits outweigh its risks;
disagreement with our interpretation of pre-clinical or clinical data; and
inadequacies in the manufacturing facilities or processes of third-party manufacturers.
The FDA or a comparable foreign authority may require us to conduct additional pre-clinical and
clinical testing, which may delay or prevent approval and our commercialization plans or cause us to
abandon the development program. Further, any approval we receive may be for fewer or more limited
indications than we request, may not include labeling claims necessary for successful commercialization
of the product candidate, or may be contingent upon our conducting costly post-marketing clinical trials.
Any of these scenarios could materially harm the commercial prospects of a product candidate.
Even if our product candidates obtain approval, they may face future development and regulatory
difficulties that can negatively affect commercial prospects.
Even if we obtain approval for a product candidate, it would be subject to ongoing regulatory
requirements and restrictions of the FDA and comparable regulatory authorities regarding manufacturing,
quality control, further development, labeling, packaging, storage, distribution, safety surveillance,
import, export, advertising, promotion, recordkeeping and reporting. Failure by us or any of the third
parties on which we rely to meet those requirements can lead to enforcement action, among other
consequences, that could significantly impair our ability to successfully commercialize a given product.
If the FDA or a comparable regulatory authority becomes aware of new safety information, it can impose
additional restrictions on how the product is marketed or may seek to withdraw marketing approval
altogether.
There is no assurance that any of our products that has received or will receive orphan drug
designation will subsequently obtain orphan drug exclusivity, or that any such exclusivity will provide
the desired benefit.
Although we have obtained orphan drug designation for several uses of Archexin and one use of
RX-3117 and may obtain additional orphan drug designation for these or other product candidates, we are
not assured of being awarded orphan drug exclusivity or realizing the benefits of such exclusivity, even if
any of these products is approved for its orphan-designated use. If another company also holding orphan
drug designation for a product containing the same active moiety intended for the same rare disease or
condition receives approval before our orphan-designated product, approval of our product could be
precluded for seven years because of that product‘s orphan drug exclusivity, unless we could demonstrate
our product to be clinically superior to the earlier-approved product. Similarly, even if our orphan
designated drug were approved first and awarded seven-year orphan drug exclusivity, it would not block
approval of the other product if that product were shown to be clinically superior, or if we fail to assure a
sufficient quantity of our orphan drug. Additionally, because orphan drug exclusivity is product- and
indication-specific, it does not prevent approval of another drug for the same orphan indication or the
same drug for a different use.
If physicians and patients do not accept and use our drugs, our ability to generate revenue from sales
of our products will be materially impaired.
Even if the FDA approves our drug candidates, physicians and patients may not accept and use
them. Future acceptance and use of our products will depend upon a number of factors including, but not
limited to:
27
awareness of a drug‘s availability and benefits;
perceptions by members of the health care community, including physicians, about the safety and
effectiveness of our drugs;
pharmacological benefit and cost-effectiveness of our products relative to competing products;
availability of reimbursement for our products from government or other third-party payors;
effectiveness of marketing and distribution efforts by us and our licensees and distributors, if any;
and
the price at which we sell our products.
Because we expect sales of our current drug candidates, if approved, to generate substantially all
of our product revenues for the foreseeable future, the failure of any of these drugs to find market
acceptance would harm our business and could require us to seek additional financing.
Even if we are able to commercialize any of our product candidates, these products may become subject
to unfavorable pricing regulations, third-party reimbursement practices or healthcare reform initiatives,
which could harm our business.
The regulations that govern marketing approvals, pricing and reimbursement for new drug products
vary widely from country to country. Current and future legislation may significantly change the approval
requirements in ways that could involve additional costs and cause delays in obtaining approvals. Some
countries require approval of the sale price of a drug before it can be marketed. In many countries, the
pricing review period begins after marketing or product licensing approval is granted. In some foreign
markets, prescription pharmaceutical pricing remains subject to continuing governmental control even after
initial approval is granted. As a result, we might obtain marketing approval for a product in a particular
country, but then be subject to price regulations that delay our commercial launch of the product, possibly
for lengthy time periods, which could negatively impact the revenues we are able to generate from the sale
of the product in that particular country. Adverse pricing limitations may hinder our ability to recoup our
investment in one or more product candidates even if our product candidates obtain marketing approval.
Our ability to commercialize any products successfully also will depend in part on the extent to
which coverage and adequate reimbursement for these products and related treatments will be available in a
timely manner from government third-party payors, including governmental healthcare programs such as
Medicare and Medicaid, commercial health insurers and managed care organizations. Government
authorities and other third-party payors, such as private health insurers and health maintenance
organizations, determine which medications they will cover and establish reimbursement levels.
Third-party payors may limit coverage to specific products on an approved list, or formulary, which
might not include all of the FDA approved products for a particular indication. The process for
determining whether a payor will provide coverage for a product may be separate from the process for
setting the price or reimbursement rate that the payor will pay for the product once coverage is approved.
A primary trend in the U.S. healthcare industry and elsewhere is cost containment. Government
healthcare programs and other third-party payors are increasingly challenging the prices charged for
medical products and services and examining the medical necessity and cost-effectiveness of medical
products and services, in addition to their safety and efficacy, and have attempted to control costs by
limiting coverage and the amount of reimbursement for particular medications. Increasingly, third-party
payors are requiring that drug companies provide them with predetermined discounts from list prices and
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are challenging the prices charged for medical products. We cannot be sure that coverage and
reimbursement will be available promptly or at all for any product that we commercialize and, if
reimbursement is available, what the level of reimbursement will be. Moreover, eligibility for coverage and
reimbursement does not imply that any drug will be paid for in all cases. Limited coverage may impact the
demand for, or the price of, any product candidate for which we obtain marketing approval. If coverage and
reimbursement are not available or reimbursement is available only to limited levels, we may not
successfully commercialize any product candidate for which we obtain marketing approval.
In the past, payors have implemented reimbursement metrics and periodically revised those metrics
as well as the methodologies used as the basis for reimbursement rates, such as ASP, AMP and actual
acquisition cost. The existing data for reimbursement based on these metrics is relatively limited, although
certain states have begun to survey acquisition cost data for the purpose of setting Medicaid reimbursement
rates. CMS, surveys and publishes retail community pharmacy acquisition cost information in the form of
NADAC files to provide state Medicaid agencies with a basis of comparison for their own reimbursement
and pricing methodologies and rates. It may be difficult to project the impact of these evolving
reimbursement mechanics on the willingness of payors to cover our products for which we receive
regulatory approval.
If we successfully commercialize any of our products, we may participate in the Medicaid Drug
Rebate program. Participation is required for federal funds to be available for our products under
Medicaid and Medicare Part B. Under the Medicaid Drug Rebate Program, we would be required to pay a
rebate to each state Medicaid program for our covered outpatient drugs that are dispensed to Medicaid
beneficiaries and paid for by a state Medicaid program as a condition of having federal funds being made
available to the states for our drugs under Medicaid and Part B of the Medicare program.
Federal law requires that any company that participates in the Medicaid Drug Rebate Program also
participate in the Public Health Service‘s 340B drug pricing program in order for federal funds to be
available for the manufacturer‘s drugs under Medicaid and Medicare Part B. The 340B drug pricing
program requires participating manufacturers to agree to charge statutorily-defined covered entities no
more than the 340B ―ceiling price‖ for the manufacturer‘s covered outpatient drugs. These 340B covered
entities include a variety of community health clinics and other entities that receive health services grants
from the Public Health Service, as well as hospitals that serve a disproportionate share of low-income
patients.
In addition, in order to be eligible to have its products paid for with federal funds under the
Medicaid and Medicare Part B programs and purchased by certain federal agencies and grantees, a
manufacturer also must participate in the FSS, pricing program, established by Section 603 of the VHCA.
Under this program, the manufacturer is obligated to make its innovator and single source products
available for procurement on an FSS contract and charge a price to four federal agencies, Department of
Veterans Affairs, DoD, Public Health Service, and Coast Guard, that is no higher than the statutory Federal
Ceiling Price. Moreover, pursuant to regulations issued by the DoD TMA, now the DHA, to implement
Section 703 of the National Defense Authorization Act for Fiscal Year 2008, manufacturers are required to
provide rebates on utilization of their innovator and single source products that are dispensed to TRICARE
beneficiaries by TRICARE network retail pharmacies. The formula for determining the rebate is
established in the regulations and is based on the difference between the annual non-federal average
manufacturer price and the Federal Ceiling Price (these price points are required to be calculated by us
under the VHCA). The requirements under the 340B, FSS, and TRICARE programs could reduce the
revenue we may generate from any products that are commercialized in the future and could adversely
affect our business and operating results.
There may be significant delays in obtaining coverage and reimbursement for newly approved
drugs, and coverage may be more limited than the purposes for which the drug is approved by the FDA or
comparable foreign regulatory authorities. Moreover, eligibility for coverage and reimbursement does not
29
imply that a drug will be paid for in all cases or at a rate that covers our costs, including research,
development, manufacture, sale and distribution. Interim reimbursement levels for new drugs, if applicable,
may also not be sufficient to cover our costs and may only be temporary. Reimbursement rates may vary
according to the use of the drug and the clinical setting in which it is used, may be based on reimbursement
levels already set for lower cost drugs and may be incorporated into existing payments for other services.
Net prices for drugs may be reduced by mandatory discounts or rebates required by government healthcare
programs or private payors and by any future relaxation of laws that presently restrict imports of drugs from
countries where they may be sold at lower prices than in the United States. Limited coverage may impact
the demand for, or the price of, any product candidate for which we obtain marketing approval.
Third-party payors also may seek additional clinical evidence, including expensive pharmacoeconomic
studies beyond the data required to obtain marketing approval, demonstrating clinical benefits and value
in specific patient populations, before covering our products for those patients. If reimbursement is
available only for limited indications, we may not be able to successfully commercialize any product
candidate for which we obtain marketing approval. Our inability to promptly obtain coverage and
profitable reimbursement rates from both government-funded and private payors for any approved products
that we develop could have a material adverse effect on our operating results, our ability to raise capital
needed to commercialize products and our overall financial condition.
Changes in healthcare law and implementing regulations, including those based on recently enacted
and future legislation, as well as changes in healthcare policy, may increase the difficulty and cost for us
to commercialize our product candidates and affect the prices we may obtain.
The United States and many foreign jurisdictions have enacted or proposed legislative and
regulatory changes affecting the healthcare system that could prevent or delay marketing approval of our
product candidates, restrict or regulate post-approval activities and affect our ability to profitably sell any
product candidate for which we obtain marketing approval. The United States government, state
legislatures and foreign governments also have shown significant interest in implementing
cost-containment programs to limit the growth of government-paid healthcare costs, including price
controls, restrictions on reimbursement and requirements for substitution of generic products for branded
prescription drugs.
In recent years, Congress has considered reductions in Medicare reimbursement levels for drugs
administered by physicians. CMS also has authority to revise reimbursement rates and to implement
coverage restrictions for some drugs. Cost reduction initiatives and changes in coverage implemented
through legislation or regulation could decrease utilization of and reimbursement for any approved
products, which in turn would affect the price we can receive for those products. While Medicare
regulations apply only to drug benefits for Medicare beneficiaries, private payors often follow Medicare
coverage policy and payment limitations in setting their own reimbursement rates. Therefore, any reduction
in reimbursement that results from federal legislation or regulation 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 Reconciliation Act of 2010 (collectively, the ―Affordable
Care Act‖). This law substantially changes the way healthcare is financed by both governmental and
private insurers, and significantly impacts the pharmaceutical industry. The Affordable Care Act is
intended to broaden access to health insurance, reduce or constrain the growth of healthcare spending,
enhance remedies against healthcare fraud and abuse, add new transparency requirements for healthcare
and health insurance industries, impose new taxes and fees on pharmaceutical and medical device
manufacturers, and impose additional health policy reforms. Among other things, the Affordable Care Act
expanded manufacturers‘ rebate liability under the Medicaid Drug Rebate Program by increasing the
minimum Medicaid rebate for both branded and generic drugs, expanded the 340B program, and revised
the definition of AMP. The legislation also extended Medicaid drug rebates, previously due only on
30
fee-for-service Medicaid utilization, to include the utilization of Medicaid managed care organizations as
well and created an alternative rebate formula for certain new formulations of certain existing products that
is intended to increase the amount of rebates due on those drugs. On February 1, 2016, CMS issued final
regulations to implement the changes to the Medicaid Drug Rebate program under the Affordable Care Act.
These regulations become effective on April 1, 2016. It appears likely that the Affordable Care Act will
continue the pressure on pharmaceutical pricing, especially under the Medicare and Medicaid programs,
and may also increase our regulatory burdens and operating costs.
The Affordable Care Act requires pharmaceutical manufacturers of branded prescription drugs to
pay a branded prescription drug fee to the federal government. Each individual pharmaceutical
manufacturer pays a prorated share of the branded prescription drug fee of $3.0 billion in 2016, based on the
dollar value of its branded prescription drug sales to certain federal programs identified in the law.
Furthermore, the new law requires manufacturers to provide a 50% discount off the negotiated price of
prescriptions filled by beneficiaries in the Medicare Part D coverage gap, referred to as the ―donut hole.‖
The Affordable Care Act also expanded the Public Health Service‘s 340B drug pricing discount
program. The 340B drug pricing program requires participating manufacturers to agree to charge
statutorily-defined covered entities no more than the 340B ―ceiling price‖ for the manufacturer‘s covered
outpatient drugs. The Affordable Care Act expanded the 340B program to include additional types of
covered entities: certain free-standing cancer hospitals, critical access hospitals, rural referral centers and
sole community hospitals, each as defined by the Affordable Care Act. The Affordable Care Act exempts
―orphan drugs‖—those designated under section 526 of the Food, Drug, and Cosmetic Act—from the
ceiling price requirements for these newly-eligible entities. Because the 340B ceiling price is determined
based on AMP and Medicaid drug rebate data, the revisions to the Medicaid rebate formula and AMP
definition described above could cause the required 340B discounts to increase.
In addition, other legislative changes have been proposed and adopted since the Affordable Care
Act was enacted. For example, recent legislative enactments have resulted in Medicare payments being
subject to a two percent reduction, referred to as sequestration, until 2025. Continuation of sequestration or
enactment of other reductions in Medicare reimbursement for drugs could affect our ability to achieve a
profit on any candidate products that are approved for marketing.
We expect that the Affordable Care Act, as well as other healthcare reform measures that have
and may be adopted in the future, may result in more rigorous coverage criteria and in additional
downward pressure on the price that we receive for any approved product and could seriously harm our
future revenues. Any reduction in reimbursement from Medicare, Medicaid, or other government
programs may result in a similar reduction in payments from private payors. The implementation of cost
containment measures or other healthcare reforms may prevent us from being able to generate revenue,
attain profitability or commercialize our products.
If we are able to successfully commercialize any of our products and if we participate in the Medicaid
drug rebate program or other governmental pricing programs, failure to comply with reporting and
payment obligations under these programs could result in additional reimbursement requirements,
penalties, sanctions and fines which could have a material adverse effect on our business, financial
condition, results of operations and growth prospects.
The Medicaid Drug Rebate Program and other governmental pricing programs require
manufacturers to report pricing data to the government. Pricing calculations vary among products and
programs and include average manufacturer price and best price for the Medicaid Drug Rebate Program,
average sales price for certain categories of drugs that are paid under Part B of the Medicare program, and
non-federal average manufacturer price for the FSS pricing program. If we successfully commercialize
any of our products and participate in such governmental pricing programs, we will be liable for errors
31
associated with our submission of pricing data. That liability could be significant. For example, if we
are found to have knowingly submitted false average manufacturer price, average sales price, best price,
or non-federal average manufacturer price information to the government, we may be liable for civil
monetary penalties in the amount of $100,000 per item of false information. If we are found to have
made a misrepresentation in the reporting of average sales price, the statute provides for civil monetary
penalties of up to $10,000 for each misrepresentation for each day in which the misrepresentation was
applied. Our failure to submit monthly/quarterly average manufacturer price, average sales price, and
best price, and quarterly/annual non-federal average manufacturer price data on a timely basis could result
in a civil monetary penalty of $10,000 per day for each day the information is late beyond the due date.
Such failure also could be grounds for other sanctions, such as termination from the Medicaid Drug
Rebate Program.
Our relationships with customers and third-party payors will be subject to applicable anti-kickback,
fraud and abuse, transparency and other healthcare laws and regulations, which could expose us to
criminal sanctions, civil penalties, contractual damages, reputational harm, administrative burdens and
diminished profits and future earnings.
Healthcare providers, physicians and third-party payors play a primary role in the recommendation
and prescription of any product candidates for which we obtain marketing approval. Our future
arrangements with third-party payors and customers may expose us to broadly applicable fraud and abuse
and other healthcare laws and regulations that may constrain the business or financial arrangements and
relationships through which we market, sell and distribute our products for which we obtain marketing
approval. Restrictions under applicable federal and state healthcare laws and regulations, include the
following:
the federal Anti-Kickback Law prohibits persons from, among other things, knowingly and
willfully soliciting, offering, receiving or providing remuneration, directly or indirectly, in
cash or in kind, to induce or reward, or in return for, the referral of an individual for the
furnishing or arranging for the furnishing, or the purchase, lease or order, or arranging for
or recommending purchase, lease or order, any good or service for which payment may be
made under a federal healthcare program such as Medicare and Medicaid;
the federal civil False Claims Act imposes penalties, including through civil whistleblower
or qui tam actions, against individuals or entities for, among other things, knowingly
presenting, or causing to be presented, to the federal government, claims for payment that
are false or fraudulent or making a false statement material to an obligation to pay money
to the government or knowingly concealing or knowingly and improperly avoiding,
decreasing, or concealing an obligation to pay money to the federal government;
the federal Health Insurance Portability and Accountability Act of 1996, or HIPAA,
imposes criminal liability for knowingly and willfully executing a scheme to defraud any
healthcare benefit program, knowingly and willfully embezzling or stealing from a health
care benefit program, willfully obstructing a criminal investigation of a health care offense,
or knowingly and willfully making false statements relating to healthcare matters;
HIPAA, as amended by the Health Information Technology for Economic and Clinical
Health Act of 2009 and its implementing regulations, also imposes obligations on certain
covered entity health care providers, health plans and health care clearinghouses as well as
their business associates that perform certain services involving the use or disclosure of
individually identifiable health information, including mandatory contractual terms, with
32
respect to safeguarding the privacy, security and transmission of individually identifiable
health information;
the federal Open Payments program, created under Section 6002 of the Affordable Care
Act and its implementing regulations, requires manufacturers of drugs, devices, biologics
and medical supplies for which payment is available under Medicare, Medicaid or the
Children‘s Health Insurance Program (with certain exceptions) to report annually to the
CMS information related to ―payments or other transfers of value‖ made to physicians
(defined to include doctors, dentists, optometrists, podiatrists and chiropractors) and
teaching hospitals, and applicable manufacturers and applicable group purchasing
organizations to report annually CMS ownership and investment interests held by
physicians (as defined above) and their immediate family members; and
analogous state and foreign laws and regulations, such as state anti-kickback and false
claims laws, which may apply to sales or marketing arrangements and claims involving
healthcare items or services reimbursed by non-governmental third-party payors, including
private insurers; state and foreign laws that require pharmaceutical companies to comply
with the pharmaceutical industry‘s voluntary compliance guidelines and the relevant
compliance guidance promulgated by the federal government or otherwise restrict
payments that may be made to certain healthcare providers; state and foreign laws that
require drug manufacturers to report information related to payments and other transfers of
value to physicians and other healthcare providers or marketing expenditures; and state and
foreign laws that govern 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 HIPAA, thus complicating compliance efforts.
Efforts to ensure that our business arrangements with third parties will comply with applicable
healthcare laws and regulations will involve substantial costs. It is possible that governmental authorities
will conclude that our business practices may not comply with current or future statutes, regulations or case
law interpreting applicable fraud and abuse or other healthcare laws and regulations. If our operations are
found to be in violation of any of these laws or any other governmental regulations that may apply to us, we
may be subject to significant civil, criminal and administrative penalties, damages, fines, imprisonment,
exclusion from government funded healthcare programs, such as Medicare and Medicaid, and the
curtailment or restructuring of our operations. If any of the physicians or other healthcare providers or
entities with whom we expect to do business is found not to be in compliance with applicable laws, that
person or entity may be subject to criminal, civil or administrative sanctions, including exclusions from
government funded healthcare programs. For a fuller discussion of the applicable anti-kickback fraud and
abuse, transparency and other healthcare laws and regulations applicable to our business, see Item 1,
‗Description of Business – Government Regulation‖
Developments by competitors may render our products or technologies obsolete or non-competitive.
We compete against fully integrated pharmaceutical companies and smaller companies that are
collaborating with larger pharmaceutical companies as well as academic institutions, government
agencies and other public and private research organizations. Many of these competitors, either alone or
together with their collaborative partners, operate larger research and development programs or have
substantially greater financial resources than we do, as well as more experience in:
developing drugs;
undertaking pre-clinical testing and human clinical trials;
33
obtaining FDA and other regulatory approvals of drugs;
formulating and manufacturing drugs; and
launching, marketing and selling drugs.
Large pharmaceutical companies currently sell both generic and proprietary compounds for the
treatment of cancer. In addition, companies developing oncology therapies represent substantial
competition. Many of these organizations have substantially greater capital resources, larger research and
development staff and facilities, longer drug development history in obtaining regulatory approvals and
greater manufacturing and marketing capabilities than we do. These organizations also compete with us to
attract qualified personnel, parties for acquisitions, joint ventures or other collaborations. Our competitors
may succeed in developing products that are more effective and/or safe than ours, which could render our
product candidates less competitive prior to recovery by us of expenses incurred with respect to their
development.
If we are unable to successfully manage our growth, our business may be harmed.
In addition to our own internally developed drug candidates, we are actively seeking
opportunities to in-license compounds in oncology and other therapeutic areas that are strategic additions
to our product pipeline. Such additional drug candidates could significantly increase our capital
requirements and place further strain on our resources, including on the time of our existing personnel,
which may delay or otherwise adversely affect the development of our existing drug candidates. As of
December 31, 2015, we had 20 full-time employees. We may need to hire more employees as our product
pipeline and operations expand, further increasing the size of our organization and related expenses. If we
are unable to manage our growth effectively, we may not efficiently use our resources, which may delay
the development of our drug candidates and negatively impact our business, results of operations and
financial condition.
We may not be able to attract and retain qualified personnel necessary for the development and
commercialization of our drug candidates. Our success may be negatively impacted if key personnel
leave.
Attracting and retaining qualified personnel is critical to our future success. We compete for
qualified individuals with numerous biopharmaceutical companies, universities and other research
institutions. Competition for such individuals is intense, and we cannot assure you that we will be
successful in engaging personnel with the skills and experience to support our business and research and
development activities.
Our key personnel, especially Dr. Chang H. Ahn, our Chairman Emeritus and Chief Scientist, Dr.
Peter Suzdak, our Chief Executive Officer, Dr. Ely Benaim, our Chief Medical Officer, and Dr. Tae
Heum Jeong, our Chief Financial Officer, provide critical technical knowledge and expertise. The loss
of Dr. Ahn, Dr. Suzdak, Dr. Benaim, Dr. Jeong, or any of the other members of our management team,
could result in delays in product development and diversion of management resources, which could
adversely affect our operating results. We do not have ―key person‖ life insurance policies for any of our
executive officers.
Risks Related to Reliance on Third Parties
Much of our drug development program depends upon third-party researchers, and thus the conduct
34
and completion of our clinical trials are, to some extent, beyond our control.
We have engaged third-party contract research organizations and other investigators and
collaborators, such as universities and medical institutions, to conduct our pre-clinical studies, toxicology
studies and clinical trials. Engaging third-party contract research organizations is typical practice in our
industry. However, relying on such organizations means that the clinical trials and other studies described
above are being conducted at external locations and that the completion of these trials and studies is not
within our direct control. Trials and studies may be delayed due to circumstances outside our control, and
such delays may result in additional expenses for us.
While we make every effort internally to oversee the work of third-party contractors, these
collaborators are not our employees, and we cannot control the effort, time or other resources that they
devote to our programs. Third parties may not assign priority to our programs or pursue them as diligently
as we would if we were undertaking them ourselves. If outside collaborators fail to devote sufficient time
and resources to our drug-development programs, or if their performance is substandard, the approval of
our FDA applications and introduction of new drugs to the market may be delayed. These collaborators
may also have relationships with other commercial entities, some of which may compete with us. If our
collaborators assist our competitors at our expense, our competitive position would be harmed.
We rely exclusively on third parties to formulate and manufacture our drug candidates, which exposes
us to a number of risks that may delay development, regulatory approval and commercialization of our
products or result in higher product costs.
We have no experience in drug formulation or manufacturing and we lack the resources and
expertise to formulate or manufacture our own drug candidates internally. Therefore, we rely on
third-party expertise to support us in this area. We have entered into contracts with third-party
manufacturers to manufacture, supply, store and distribute supplies of our drug candidates for our clinical
trials. If any of our drug candidates receives FDA approval, we expect to rely on third-party contractors to
manufacture our drugs. We have no current plans to build internal manufacturing capacity for any
product, and we have no long-term supply arrangements.
Our reliance on third-party manufacturers exposes us to the following potential risks:
We may be unable to contract with third-party manufacturers on acceptable terms, or at all,
because the number of potential manufacturers is limited and potential manufacturers are subject
to FDA approval. FDA approval requires testing and compliance inspections. In addition, any
new manufacturer would have to be qualified and approved to produce our products after receipt
of FDA approval, if any;
Our third-party manufacturers might be unable to formulate and manufacture our drugs in the
volume and of the quality required to meet our clinical and commercial needs, if any;
Our third-party manufacturers may not perform as agreed or may not remain in the contract
manufacturing business for the time required to supply our clinical trials through completion or to
successfully produce, store and distribute our commercial products, if approved;
Drug manufacturers are subject to ongoing periodic unannounced inspection by the FDA and
other government agencies to ensure compliance with cGMP and other government regulations
and corresponding foreign standards. We do not have control over third-party manufacturers‘
compliance with these regulations and standards, but we may ultimately be responsible for any of
35
their failures;
If any third-party manufacturer makes improvements in the manufacturing process for our
products, we may not own, or may have to share, the intellectual property rights to such
improvements; and
A third-party manufacturer may gain knowledge from working with us that could be used to
supply one of our competitors with a product that competes with ours.
Each of these risks could delay or have other adverse impacts on our clinical trials and the
approval and commercialization of our drug candidates, potentially resulting in higher costs, reduced
revenues or both.
We have no experience selling, marketing or distributing products and currently no internal capability
to do so.
We currently have no sales, marketing or distribution capabilities. While we intend to have a role
in the commercialization of our products, we do not anticipate having the resources in the foreseeable
future to develop global sales and marketing capabilities for all of our proposed products. Our future
success depends, in part, on our ability to enter into and maintain collaborative relationships with other
companies that have sales, marketing and distribution capabilities, a strategic interest in the products
under development, and the ability to successfully market and sell our products. To the extent that we
decide not to, or are unable to, enter into collaborative arrangements with respect to the sales and
marketing of our proposed products, significant capital expenditures, management resources and time will
be required to establish and develop an in-house marketing and sales force with the necessary expertise.
We cannot assure you that we will be able to establish or maintain relationships with third-party
collaborators or develop in-house sales and distribution capabilities. To the extent that we depend on third
parties for marketing and distribution, any revenues we receive will depend upon the efforts of such third
parties, as well as the terms of our agreements with such third parties, which cannot be predicted at this
early stage of our development. We cannot assure you that such efforts will be successful. In addition, we
cannot assure you that we will be able to market and sell our products in the United States or overseas.
We may incur substantial liabilities and may be required to limit commercialization of our products in
response to product liability lawsuits.
The testing and marketing of medical products entail an inherent risk of product liability. Product
liability claims may be brought against us by subjects enrolled in our clinical trials, patients, healthcare
providers or others using, administering or selling our products. Large judgments have been awarded in
class action lawsuits based on drugs that had unanticipated side effects. If we cannot successfully defend
ourselves against product liability claims, we may incur substantial liabilities or be required to limit
commercialization of our products. Our inability to obtain sufficient product liability insurance at an
acceptable cost to protect against potential product liability claims could prevent or inhibit the
commercialization of pharmaceutical products we develop, alone or with collaborators. Although we
currently carry clinical trial insurance and product liability insurance we, or any collaborators, may not be
able to maintain such insurance at a reasonable cost. Even if our agreements with any future collaborators
entitle us to indemnification against losses, such indemnification may not be available or adequate should
any claims arise.
Risks Related to Our Intellectual Property
If we fail to adequately protect or enforce our intellectual property rights or secure rights to patents of
36
others, the value of our intellectual property rights would diminish, and our business and competitive
position would suffer.
Our success, competitive position and future revenues will depend in part on our ability and the
abilities of our licensors and licensees to obtain and maintain patent protection for our products, methods,
processes and other technologies, to preserve our trade secrets, to prevent third parties from infringing on
our proprietary rights and to operate without infringing the proprietary rights of third parties. We have an
active patent protection program that includes filing patent applications on new compounds, formulations,
delivery systems and methods of making and using products and prosecuting these patent applications in
the United States and abroad. As patents issue, we also file continuation applications as appropriate.
Although we have taken steps to build a strong patent portfolio, we cannot predict:
the degree and range of protection any patents will afford us against competitors, including
whether third parties find ways to invalidate or otherwise circumvent our licensed patents;
if and when patents will issue in the United States or any other country;
whether or not others will obtain patents claiming aspects similar to those covered by our licensed
patents and patent applications;
whether we will need to initiate litigation or administrative proceedings to protect our intellectual
property rights, which may be costly whether we win or lose;
whether any of our patents will be challenged by our competitors alleging invalidity or
unenforceability and, if opposed or litigated, the outcome of any administrative or court action as
to patent validity, enforceability or scope;
whether a competitor will develop a similar compound that is outside the scope of protection
afforded by a patent or whether the patent scope is inherent in the claims modified due to
interpretation of claim scope by a court;
whether there were activities previously undertaken by a licensor that could limit the scope,
validity or enforceability of licensed patents and intellectual property; or
whether a competitor will assert infringement of its patents or intellectual property, whether or
not meritorious, and what the outcome of any related litigation or challenge may be.
Our success also depends upon the skills, knowledge and experience of our scientific and
technical personnel, our consultants and advisors as well as our licensors, sublicensees and contractors.
To help protect our proprietary know-how and our inventions for which patents may be unobtainable or
difficult to obtain, we rely on trade secret protection and confidentiality agreements. To this end, we
require all employees to enter into agreements that prohibit the disclosure of confidential information and,
where applicable, require disclosure and assignment to us of the ideas, developments, discoveries and
inventions important to our business. These agreements may not provide adequate protection for our trade
secrets, know-how or other proprietary information in the event of any unauthorized use or disclosure or
the lawful development by others of such information. If any of our trade secrets, know-how or other
proprietary information is disclosed, the value of our trade secrets, know-how and other proprietary rights
would be significantly impaired, and our business and competitive position would suffer.
37
Due to legal and factual uncertainties regarding the scope and protection afforded by patents and other
proprietary rights, we may not have meaningful protection from competition.
Our long-term success will substantially depend upon our ability to protect our proprietary
technologies from infringement, misappropriation, discovery and duplication and avoid infringing the
proprietary rights of others. Our patent rights, and the patent rights of biopharmaceutical companies in
general, are highly uncertain and include complex legal and factual issues. These uncertainties also mean
that any patents that we own or may obtain in the future could be subject to challenge, and even if not
challenged, may not provide us with meaningful protection from competition. Patents already issued to us
or our pending applications may become subject to dispute, and any dispute could be resolved against us.
In connection with the process of seeking patent protection for Supinoxin in Japan, we filed a
patent application including claims covering Supinoxin with the Japanese Patent Office (―JPO‖) for
examination. The JPO initially agreed that the claims covering the compound for Supinoxin were
allowable, but as a result of a mistake in the patent application filing as prepared and submitted by our
Japanese patent attorneys and incomplete review by the JPO‘s patent examiner, the JPO issued a decision
to grant a patent with claims that did not include Supinoxin. We appealed this decision with the JPO to
request withdrawal of the decision to grant so that the correct claims would be allowed, but the JPO
refused to withdraw its decision. As a result, and in accordance with Japanese law and procedure for
appealing patent application decisions, we have filed a lawsuit against the JPO in Tokyo District Court to
cause the JPO to reverse its decision to grant the errant patent and to allow a patent that includes claims
covering Supinoxin. The patent application at issue remains pending subject to the outcome of this
action. There can be no guarantee that we will be successful in winning the appeal to correct the error in
the patent registration that would exclude the compound for Supinoxin.
If we infringe the rights of third parties, we could be prevented from selling products and be forced to
defend against litigation and pay damages.
If our products, methods, processes and other technologies infringe the proprietary rights of other
parties, we could incur substantial costs and may have to:
obtain licenses, which may not be available on commercially reasonable terms, if at all;
redesign our products or processes to avoid infringement;
stop using the subject matter claimed in patents held by others, which could cause us to lose the
use of one or more of our drug candidates;
pay damages; or
defend litigation or administrative proceedings that may be costly whether we win or lose and
that could result in a substantial diversion of our management resources.
Although we have not received any claims of infringement by any third parties to date, we expect
that as our drug candidates move further into clinical trials and commercialization and our public profile
is raised, we may be subject to such claims.
38
Risks Related to Ownership of Our Common Stock
An investment in shares of our common stock is very speculative and involves a very high degree of
risk.
To date, we have generated no revenues from product sales and only minimal revenues from a
research agreement with a minority shareholder and interest on bank account balances and short-term
investments. Our accumulated deficit as of December 31, 2015 and 2014 was $105,716,864 and
$91,332,308, respectively. For the years ended December 31, 2015, 2014, and 2013, we had net losses of
$14,384,556, $18,521,601 and $9,499,424, respectively, partially as a result of expenses incurred through
a combination of research and development activities related to the various technologies under our control
and expenses supporting those activities. Until we receive approval from the FDA and other regulatory
authorities for our drug candidates, we cannot sell our drugs and will not have product revenues.
The market price of our common stock may fluctuate significantly.
The market price of our common stock may fluctuate significantly in response to factors, some of
which are beyond our control, such as:
the announcement of new products or product enhancements by us or our competitors;
changes in our relationships with our licensors or other strategic partners;
developments concerning intellectual property rights and regulatory approvals;
variations in our and our competitors‘ results of operations;
changes in earnings estimates or recommendations by securities analysts;
changes in the structure of healthcare payment systems; and
developments and market conditions in the pharmaceutical and biotechnology industries.
Further, the stock market, in general, and the market for biotechnology companies, in particular,
have experienced extreme price and volume fluctuations. Continued market fluctuations could result in
extreme volatility in the price of our common stock, which may be unrelated or disproportionate to our
operating performance and which could cause a decline in the value of our common stock. You should
also be aware that price volatility might be worse if the trading volume of our common stock is low.
We will require additional capital funding the receipt of which may impair the value of our common
stock.
Our future capital requirements depend on many factors, including our research, development,
sales and marketing activities. We will need to raise additional capital through public or private equity or
debt offerings or through arrangements with strategic partners or other sources in order to continue to
develop our drug candidates. There can be no assurance that additional capital will be available when
needed or on terms satisfactory to us, if at all. To the extent we raise additional capital by issuing equity
securities, our stockholders may experience substantial dilution and the new equity securities may have
39
greater rights, preferences or privileges than our existing common stock.
We have not paid dividends to our stockholders in the past, and we do not anticipate paying dividends
to our stockholders in the foreseeable future.
We have not declared or paid cash dividends on our common stock. We currently intend to retain
all future earnings, if any, to fund the continuing operation of our business, and therefore we do not
anticipate paying dividends on our common stock in the foreseeable future. As a result, you will not
realize any income from an investment in our common stock until and unless you sell your shares at a
profit.
We may be subject to securities litigation, which is expensive and could divert management attention.
The market price of our common stock may be volatile, and in the past companies that have
experienced volatility in the market price of their stock have been subject to securities class action
litigation. We may be the target of this type of litigation in the future. Securities litigation against us
could result in substantial costs and direct our management‘s attention from other business concerns,
which could seriously harm our business.
Item 1B. Unresolved Staff Comments.
None
Item 2. Description of Property.
We lease approximately 7,103 square feet of office space in Rockville, Maryland. We also lease
approximately 1,100 square feet of laboratory space in Gaithersburg, Maryland. The laboratory space is
equipped with the requisite laboratory services required to conduct our business and we believe our existing
facilities are adequate to meet our needs for the foreseeable future. The office lease, which commenced on
June 29, 2009, expires in June 2019. The laboratory lease, which commenced on July 1, 2015 expires in
June 2020. We do not own any real property.
Item 3. Legal Proceedings.
None
Item 4. Mine Safety Disclosures
Not Applicable
40
PART II
Item 5. Market for Common Equity, Related Stockholder Matters and Issuer Purchases of Equity
Securities.
Our common stock is traded on the NYSE MKT, under the ticker symbol ―RNN‖. As of March
11, 2016, there were approximately 66 stockholders of record of our common stock. The following
table sets forth the high and low sales prices of our common shares as reported on the NYSE MKT during
the periods indicated.
Period
2014
First Quarter
Second Quarter
Third Quarter
Fourth Quarter
2015
First Quarter
Second Quarter
Third Quarter
Fourth Quarter
Dividends
High
Low
1.85
1.29
0.89
0.82
0.96
0.81
0.66
0.53
0.50
0.78
0.65
0.65
0.69
0.60
0.48
0.35
We have not paid any cash dividends on common stock and do not expect to do so in the
foreseeable future. We anticipate that any earnings generated from future operations will be used to
finance our operations. No restrictions exist upon our ability to pay dividends.
Purchase of Equity Securities by the Issuer and Affiliated Purchasers
There were no repurchases of equity securities in 2015.
Recent Sales of Unregistered Equity Securities
As previously disclosed, pursuant to an engagement letter agreement, dated November 5, 2015,
with H.C. Wainwright & Co., LLC, on November 12, 2015, we issued warrants to purchase 833,333
shares of our common stock (the ―Placement Agent Warrants‖) to designees of Rodman & Renshaw
(―Rodman‖), a unit of H.C. Wainwright & Co., LLC, as partial consideration for Rodman‘s services as
placement agent in connection with a registered direct offering of our common stock and warrants. The
common stock issuable upon exercise of the warrants is expected to be issued and the Placement Agent
Warrants were offered and sold by us in reliance on the exemption from registration provided by Section
4(a)(2) of the Securities Act as transactions not involving any public offering. The Placement Agent
Warrants have an exercise price of $0.53 per share and are exercisable at any time from May 13, 2016 to
November 6, 2020.
41
Performance Graph
The following graph compares the cumulative total stockholder return on $100 of our common
stock for the period beginning December 31, 2010 through December 31, 2015, with the cumulative total
return over such period for an identical investment in i) the NYSE Arca Biotechnology Index or ii) the
NYSE MKT Composite Index. This graph is not deemed to be ―filed‖ with the SEC or subject to the
liabilities of Section 18 of the Exchange Act, and the graph shall not be deemed to be incorporated by
reference into any prior or subsequent filing by us under the Securities Act or the Exchange Act.
42
$- $50 $100 $150 $200 $250 $30012/1012/1112/1212/1312/1412/15Rexahn Pharmaceuticals, Inc.NYSE Arca Biotechnology IndexNYSE MKT Composite Index
Item 6. Selected Financial Data.
The following selected data should be read in conjunction with ―Item 7. Management‘s
Discussion and Analysis of Financial Condition and Results of Operations‖ and our financial statements
included elsewhere in this Annual Report.
Statement of Operations Data:
Revenues
$
Expenses:
General and administrative
Research and development
Total expenses
Loss from operations
Other Income (Expense), net
Net Loss
Net Loss per share, basic and
diluted
Weighted average shares
outstanding, basic and diluted
$
$
Balance Sheet Data:
Cash, Cash Equivalents, and
Marketable Securities
Working Capital
Total Assets
Warrant Liabilities
Accumulated Deficit
Total Stockholders' Equity
Common shares outstanding
$
$
$
$
$
$
2015
For the Year Ended December 31,
2014
2013
2012
2011
- $
- $
- $
- $
-
6,115,210
12,148,226
18,263,436
(18,263,436)
3,878,880
6,253,328
7,015,901
13,269,229
(13,269,229)
(5,252,372)
(14,384,556) $ (18,521,601) $
(0.11) $
(0.08) $
4,725,699
3,253,139
7,978,838
(7,978,838)
(1,520,586)
(9,499,424) $
(0.07) $
3,186,634
3,392,896
6,579,530
(6,579,530)
352,860
4,138,926
11,992,087
16,131,013
(16,131,013)
4,786,063
(6,226,670) $ (11,344,950)
(0.12)
(0.06) $
182,388,226
176,106,981
128,649,303
97,138,233
93,048,490
2015
2014
2013
2012
2011
As of December 31,
23,439,526 $ 32,698,296 $ 18,788,031 $ 13,586,543 $ 11,811,488
22,000,046 $ 30,970,020 $ 18,361,438 $ 12,923,514 $ 10,977,936
24,805,029 $ 33,533,060 $ 19,556,498 $ 14,919,308 $ 13,689,648
868,725
2,739,163 $
(105,716,864) $ (91,332,308) $ (72,810,707) $ (63,311,283) $ (57,084,613)
9,533,989 $ 10,706,130
95,345,656
18,775,548 $ 26,580,491 $ 12,625,488 $
178,253,318
197,413,785
5,034,058 $
2,842,065 $
3,768,351 $
146,717,795
119,428,989
43
Item 7. Management’s Discussion and Analysis of Financial Condition and Results of Operations.
You should read the following discussion and analysis of our results of operations, financial condition
and liquidity in conjunction with our financial statements and the related notes, which are included in this
Annual Report. Some of the information contained in this discussion and analysis or set forth elsewhere
in this Annual Report, including information with respect to our plans and strategies for our business,
statements regarding the industry outlook, our expectations regarding the future performance of our
business, and the other non-historical statements contained herein are forward-looking statements. See
“Cautionary Statement Regarding Forward-Looking Statements.” You should also review the “Risk
Factors” section under this Item 1A of this Annual Report for a discussion of important factors that could
cause actual results to differ materially from the results described herein or implied by such
forward-looking statements.
OVERVIEW
We are a clinical stage biopharmaceutical company dedicated to the discovery, development and
commercialization of innovative treatments for cancer. Our mission is to improve the lives of cancer
patients by developing next-generation cancer therapies that are designed to maximize efficacy while
minimizing the toxicity and side effects traditionally associated with cancer treatment. Our clinical
pipeline features one product candidate in Phase II clinical development, two product candidates in Phase
I clinical development and additional compounds in pre-clinical development. Our strategy is to continue
building a significant pipeline of innovative oncology product candidates that we will commercialize
alone or with partners.
Since our inception, our efforts and resources have been focused primarily on developing our
pharmaceutical technologies, raising capital and recruiting personnel. We have no product sales to date,
and we will not generate any product sales until we receive approval from the FDA or equivalent foreign
regulatory bodies to begin selling our pharmaceutical candidates. Our major sources of working capital
have been proceeds from various private and public financings, and licensing and collaboration
agreements with our strategic investors and partners.
Critical Accounting Policies
A ―critical accounting policy‖ is one which is both important to the portrayal of our financial
condition and results and requires our management‘s most difficult, subjective or complex judgments,
often as a result of the need to make estimates about the effect of matters that are inherently uncertain.
Our accounting policies are in accordance with U.S. generally accepted accounting principles and their
basis of application is consistent with that of the previous year. Our significant estimates include
assumptions made in estimating the fair values of stock-based compensation, warrant liabilities,
marketable securities, and our assessment relating to costs incurred on research and development
contracts.
Research and Development
Research and development costs are expensed as incurred. Research and development expenses
consist primarily of third party service costs under research and development agreements, salaries and
related personnel costs, as well as stock compensation related to these costs, costs to acquire
pharmaceutical products and product rights for development and amounts paid to contract research
organizations, hospitals and laboratories for the provision of services and materials for drug development
and clinical trials.
44
Costs incurred in obtaining the license rights to technology in the research and development stage
that have no alternative future uses and are for unapproved product compounds are expensed as incurred.
Fair Value of Financial Instruments
The carrying amounts reported in the accompanying financial statements for cash and cash
equivalents, prepaid expenses and other current assets and accounts payable and accrued expenses
approximate fair value because of the short-term maturity of these financial instruments. The fair value
methodology for our warrant liabilities and marketable securities is described in detail in Item 8 of this
Annual Report on Form 10-K.
Income Taxes
We account for income taxes in accordance with Accounting Standards Codification (―ASC‖)
740, ―Income Taxes.‖ Deferred tax assets and liabilities are recorded for differences between the
financial statement and tax basis of the assets and liabilities that will result in taxable or deductible
amounts in the future based on enacted tax laws and rates. ASC 740 requires that a valuation allowance
be established when it is more likely than not that all portions of a deferred tax asset will not be realized.
A review of all positive and negative evidence needs to be considered, including a company‘s current and
past performance, the market environment in which the company operates, length of carryback and
carryforward periods and existing contracts that will result in future profits. Income tax expense is
recorded for the amount of income tax payable or refundable for the period, increased or decreased by the
change in deferred tax assets and liabilities during the period.
As a result of our significant cumulative losses, we determined that it was appropriate to establish
a valuation allowance for the full amount of our deferred tax assets.
The calculation of our tax liabilities involves the inherent uncertainty associated with the
application of complex tax laws. We are subject to examination by various taxing authorities. We
believe that as a result of our losses sustained to date, any examination would result in a reduction of our
net operating loss carryforward rather than a tax liability. As such, we have not provided for additional
taxes estimated under ASC 740.
Warrant Liabilities
In accordance with ASC 480, ―Distinguishing Liabilities from Equity,‖ we record warrant
liabilities at fair value due to provisions in our warrant agreements, as discussed further in Note 12,
Warrants, in the Notes to the Financial Statements of Item 8 of this Annual Report. We reevaluate the
fair value of our warrants at each reporting period, and changes in the fair value between reporting
periods is recorded as ―unrealized gain (loss) on fair value of warrants‖ in the statement of operations.
Stock-Based Compensation
In accordance with ASC 718, ―Stock Compensation‖ compensation costs related to share-based
payment transactions, including employee stock options, are to be recognized in the financial statements. In
addition, we adhere to the guidance set forth within SEC Staff Accounting Bulletin No. 107 (―SAB 107‖),
which provides the Staff‘s views regarding the interaction between ASC 718 and certain SEC rules and
regulations, and provides interpretations with respect to the valuation of share-based payments for public
companies.
Concentration of Credit Risk
ASC 825, ―Financial Instruments,‖ requires disclosure of any significant off-balance sheet risk and
credit risk concentration. We do not have significant off-balance sheet risk or credit concentration. We
maintain cash and short-term investments with major financial institutions. From time to time we have
funds on deposit with commercial banks that exceed federally insured limits. The balances are insured by
45
the Federal Deposit Insurance Corporation up to $250,000. At December 31, 2015, our uninsured cash
balance was $9,554,878. Management does not consider this to be a significant credit risk as the banks are
large, established financial institutions.
Recently Issued Accounting Standards
See Note 2, ―Summary of Significant Accounting Policies in the Notes to the Financial
Statements‖, in the Notes to Financial Statements of this Annual Report for a discussion of recent
accounting pronouncements.
Results of Operations
Comparison of the Years Ended December 31, 2015 and December 31, 2014
Total Revenues
We had no revenues for the years ended December 31, 2015 or 2014.
General and Administrative Expenses
General and administrative expenses consist primarily of salaries and related expenses for
executive, finance and other administrative personnel, recruitment expenses, professional fees and other
corporate expenses, including business development, investor relations, and general legal activities.
General and administrative expenses decreased $138,118, or 2.2%, to $6,115,210 for the year
ended December 31, 2015 from $6,253,328 for the year ended December 31, 2014. The year over year
decrease is primarily attributable to a decrease in professional fees.
Research and Development Expenses
Research and development expenses increased $5,132,325, or 73.2%, to $12,148,226 for the year
ended December 31, 2015, from $7,015,901 for the year ended December 31, 2014. The increase is
primarily attributable to the advancement of our drug candidates. During the year ended December 31,
2015, we incurred additional clinical trial and drug manufacturing costs as we have advanced our clinical
trials for Archexin, RX-3117 and Supinoxin. The increase is also partially attributable to an increase in
personnel expenses.
46
The table below summarizes the approximate amounts incurred on each of our research and
development projects for the years ended December 31, 2015 and 2014:
Clinical Candidates:
Archexin
RX-3117
Supinoxin
For the Year Ended December 31,
2015
2014
$
1,547,000 $
4,062,000
2,839,000
1,215,000
1,897,000
1,351,000
Pre-clinical, Personnel and Overhead
3,700,226
2,552,901
Total Research and Development Expenses
$
12,148,226 $
7,015,901
Interest Income
Interest income decreased $30,638 or 22.9% to $103,269 for the year ended December 31, 2015
from $133,907 for the year ended December 31, 2014. The decrease is primarily attributable to lower
aggregate balances of cash, cash equivalents, and marketable securities for the year ended December 31,
2015 compared to the year ended December 31, 2014.
Unrealized Gain (Loss) on Fair Value of Warrants
Our warrants are recorded as liabilities at fair value, and the warrants are valued using a lattice
model. Changes in the fair value of warrants are recorded as an unrealized gain or loss in our statement
of operations. During the years ended December 31, 2015 and 2014, we recorded unrealized gains
(losses) on the fair value of our warrants of $3,986,727 and $(5,180,107) respectively. Estimating fair
values of warrants requires the development of significant and subjective estimates that may, and are
likely to, change over the duration of the warrant with related changes to external market factors. The
unrealized gain for the year ended December 31, 2015 primarily resulted from a decreased stock price
underlying the common stock at December 31, 2015, while the unrealized loss for the year ended
December 31, 2014 primarily resulted from an increased price of the underlying common stock at
December 31, 2014 and on the dates during the year when warrant holders exercised their warrants.
Financing Expense
We incurred $211,116 and $206,172 of financing expenses during the years ended December 31,
2015 and 2014, respectively, related to our registered direct offerings in November 2015 and January
2014, respectively.
Net Loss
As a result of the above, net loss for the years ended December 31, 2015 and 2014 was
$14,384,556 and $18,521,601 or $0.08 and $0.11 per share, respectively.
47
Comparison of the Years Ended December 31, 2014 and December 31, 2013
Total Revenues
We had no revenues for the years ended December 31, 2014 or 2013.
General and Administrative Expenses
General and administrative expenses increased $1,527,629, or 32.3%, to $6,253,328 for the year
ended December 31, 2014 from $4,725,699 for the year ended December 31, 2013. The year over year
increase is primarily attributable to an increase in professional fees and personnel expenses.
Professional fees increased approximately $1,015,000 during the year ended December 31, 2014 due to
increased legal and accounting fees regarding corporate organizational matters, consulting, proxy
solicitation fees, and investor relations fees and compensatory stock. For the year ended December 31,
2014, general and administrative expenses also increased approximately $175,000 due to an increase in
personnel, and $150,000 due to an increase in insurance coverage.
Research and Development Expenses
Research and development expenses increased $3,762,762, or 115.7%, to $7,015,901 for the year
ended December 31 2014, from $3,253,139 for the year ended December 31, 2013. The increase is
primarily attributable to our drug candidates advancing further through clinical development. During the
year ended December 31, 2014, one of our drug candidates, Archexin, entered a Phase IIa clinical trial to
study its safety and efficacy in patients with metastatic renal cell carcinoma (―RCC‖) and another drug
candidate, RX-3117, entered a Phase Ib clinical trial to study its safety and efficacy in patients with solid
tumors. Our Phase I trial for Supinoxin was initiated in August 2013 and continued through 2014.
Research and development personnel and overhead increased due to the hiring of additional personnel.
The table below summarizes the approximate amounts spent on each of our research and
development projects for the years ended December 31, 2014 and 2013:
Clinical Candidates:
Archexin
RX-3117
Supinoxin
For the Year Ended December 31,
2014
2013
$
1,215,000 $
1,897,000
1,351,000
144,300
402,000
784,800
Pre-clinical, Personnel and Overhead
2,552,901
1,922,039
Total Research and Development Expenses
$
7,015,901 $
3,253,139
Interest Income
Interest income increased $84,627, or 171.7% to $133,907 for the year ended December 31, 2014
from $49,280 for the year ended December 31, 2013. The increase is primarily attributable to higher
cash and cash equivalents and marketable securities balances due to our registered direct offering in
January 2014 and the exercise of warrants in 2014.
48
Unrealized Gain (Loss) on Fair Value of Warrants
Our warrants are recorded as liabilities at fair value, and the warrants are valued using a lattice
model. Changes in the fair value of warrants are recorded as an unrealized gain or loss in our statement
of operations. During the years ended December 31, 2014 and 2013, we recorded unrealized losses on
the fair value of our warrants of $5,180,107 and $1,365,654, respectively. Estimating fair values of
warrants requires the development of significant and subjective estimates that may, and are likely to,
change over the duration of the warrant with related changes to external market factors. The large
unrealized loss for the year ended December 31, 2014 primarily resulted from an increased stock price of
the underlying common stock at December 31, 2014 and on the dates in 2014 when warrant holders
exercised their warrants.
Financing Expense
We incurred $206,172 and $204,212 of financing expenses during the years ended December 31,
2014 and 2013, respectively, related to our registered direct offerings in January 2014, October, 2013 and
July 2013.
Net Loss
As a result of the above, net loss for the years ended December 31, 2014 and 2013 was
$18,521,601 and $9,499,424 or $0.11 and $0.07 per share, respectively.
Research and Development Projects
Research and development costs are expensed as incurred. These costs consist primarily of
salaries and related personnel costs, costs to acquire pharmaceutical products and product rights for
development and amounts paid to contract research organizations, hospitals and laboratories for the
provision of services and materials for drug development and clinical trials. Costs incurred in obtaining
the license rights to technology in the research and development stage that have no alternative future uses
are expensed as incurred. Our research and development programs are related to our oncology clinical
stage drug candidates, Archexin, RX-3117 and Supinoxin, and our pre-clinical stage drug candidate,
RX-21101. As we expand our clinical studies, we expect to enter into additional development
agreements. Significant additional expenditures will be required if we complete our clinical trials, start
new trials, apply for regulatory approvals, continue development of our technologies, expand our
operations and bring our products to market. The eventual total cost of each clinical trial is dependent on
a number of uncertainties such as trial design, the length of the trial, the number of clinical sites and the
number of patients. The process of obtaining and maintaining regulatory approvals for new therapeutic
products is lengthy, expensive and uncertain. Because the successful development of our most advanced
drug candidates, Archexin, RX-3117 and Supinoxin, is uncertain, and because RX-21101 is in early-stage
development, we are unable to estimate the costs of completing our research and development programs,
the timing of bringing such programs to market and, therefore, when material cash inflows could
commence from the sale of these drug candidates, if any. If these projects are not completed as planned,
our results of operations and financial condition would be negatively affected.
Archexin
Archexin is a potential best-in-class, potent inhibitor of the protein kinase Akt-1, which we
believe plays a critical role in cancer cell proliferation, survival, angiogenesis, metastasis and drug
resistance. Additional information about Archexin, including about the ongoing two-stage Phase IIa
proof-of-concept clinical trial of Archexin in patients with metastatic renal cell carcinoma to evaluate its
49
safety and efficacy, can be found in Item 1 to this Annual Report. We expect that expenses related to
Archexin will increase slightly in 2016 compared to 2015 as we begin Stage 2 of the trial.
RX-3117
RX-3117 is a novel, investigational oral small molecule nucleoside compound. We believe
RX-3117 has therapeutic potential in a broad range of cancers including pancreatic, bladder, lung,
cervical, non-small cell lung cancer and colon cancer. Additional information about RX-3117, including
about the current Phase Ib clinical trial to study the safety, tolerability, dose-limiting toxicities and MTD in
patients with solid tumors, can be found in Item 1 of this Annual Report. We expect that expenses
related to RX-3117 will increase slightly in 2016 compared to 2015 as we expect to reach the
MTD/recommended Phase Ib/IIa dose in the first half of 2016 and will continue development with a two
arm Phase Ib/IIa study.
Supinoxin (RX-5902)
Supinoxin is a potential first-in-class small molecule inhibitor of phosphorylated-p68, a protein
that we believe plays a key role in cancer growth, progression and metastasis. Phosphorylated p68 results
in up-regulation of cancer-related genes and a subsequent proliferation of cancer cells and tumor growth.
Additional information about Supinoxin, including about the current Phase I dose-escalation clinical trial
in cancer patients with solid tumors designed to evaluate the safety, tolerability, dose-limiting toxicities and
MTD, can be found in Item 1 of this Annual Report. We expect that expenses related to Supinoxin will
increase slightly in 2016 compared to 2015 as we expect to reach the MTD/recommended Phase Ib/IIa
dose in the first half of 2016 and will continue development with a two arm Phase Ib/IIa study.
Pre-clinical Pipeline
We expect that expenses related to our pre-clinical pipeline, including RX-21101, will remain flat
in 2016 compared to 2015 as we continue testing and development.
Research and Development Process
We have engaged third-party contract research organizations and other investigators and
collaborators, such as universities and medical institutions, to conduct our pre-clinical studies, toxicology
studies and clinical trials. Engaging third-party contract research organizations is typical practice in our
industry. However, relying on such organizations means that the clinical trials and other studies described
above are being conducted at external locations and that the completion of these trials and studies is not
within our direct control. Trials and studies may be delayed due to circumstances outside our control, and
such delays may result in additional expenses for us.
50
Liquidity and Capital Resources
Cash Flows
The table below summarizes our net cash flow activity:
Net Cash Used in Operating Activities
Net Cash Provided by (Used In) Investing Activities
Net Cash Provided by Financing Activities
Net Increase (Decrease) in Cash and Cash Equivalents
For the Year Ended December 31,
2014
2015
$ (17,351,950) $ (11,041,211) $ (7,984,856)
845,522
12,340,822
5,201,488
(22,661,045)
24,840,470
(8,861,786) $
9,554,394
8,170,751
373,195 $
2013
$
Cash used in operating activities was $17,351,950 for the year ended December 31, 2015. The
operating cash flows during the year ended December 31, 2015 reflect our net loss of $14,384,556, an
unrealized gain on the fair value of warrants of $3,986,727 and a net decrease of cash components of
working capital and non-cash charges totaling $1,019,333. Cash used in operating activities was
$11,041,211 for the year ended December 31, 2014. The operating cash flows during the year ended
December 31, 2014 reflect our net loss of $18,521,601, which includes an unrealized loss on fair value of
warrants of $5,180,107 and a net increase of cash components of working capital and other non-cash
charges totaling $2,300,283. Cash used in operating activities was $7,984,856 for the year ended
December 31, 2013, which reflects our net loss of $9,499,424 and a net increase of cash components of
working capital and non-cash charges totaling $1,514,568. The significant increases in cash used in
operating activities for the years ended December 31, 2015 and 2014 were primarily due to increases in
research and development expenses as we continued to develop our product candidates.
Cash provided by investing activities was $9,554,394 for the year ended December 31, 2015,
which consisted of $17,525,000 from the redemption of marketable securities, offset by $7,908,304 and
$62,302 for the purchases of marketable securities and equipment, respectively. Cash used in investing
activities was $22,661,045 for the year ended December 31, 2014, which consisted of $26,075,926 and
$41,249 for the purchases of marketable securities and equipment, respectively, offset by a decrease in
restricted cash equivalents of $196,130 and $3,260,000 from the redemption of marketable securities.
Cash provided by investing activities for the year ended December 31, 2013 was $845,522, which
consisted of a decrease in restricted cash equivalents of $895,671, offset by $50,149 for the purchase of
equipment.
Cash provided by financing activities was $8,170,751 for the year ended December 31, 2015,
which consisted of net proceeds of $7,439,809 from our registered direct public offering and our at
market issuance agreement, and proceeds of $708,617 and $22,325 received from the exercise of stock
options and stock warrants, respectively. Cash provided by financing activities was $24,840,470 for the
year ended December 31, 2014, which consisted of net proceeds of $18,634,247 from our registered
direct public offering in January 2014, $258,955 from the exercise of stock options and $5,947,268 from
the exercise of warrants. Cash provided by financing activities was $12,340,822 for the year ended
December 31, 2013, which consisted of net proceeds of $10,041,155 from our registered direct public
offerings in July and October, 2013, $90,000 from the exercise of stock options, and $2,209,667 from the
exercise of warrants.
51
Financings
On July 26, 2013 we closed a registered direct public offering of 11,400,000 shares of common
stock and warrants to purchase up to 3,990,000 shares of common stock. The common stock and
warrants were sold in units consisting of one share of common stock and a warrant to purchase 0.35
shares of common stock at a price of $0.50 per unit, and the warrants have an exercise price of $0.59 per
share. The total gross proceeds of the offering were $5,700,000. The warrants issued are exercisable
beginning six months after the closing date until the five-year anniversary of the closing date.
On October 16, 2013 we closed a registered direct public offering of 10,192,309 shares of
common stock and warrants to purchase up to 3,567,309 shares of common stock. The common stock
and warrants were sold in units consisting of one share of common stock and a warrant to purchase 0.35
shares of common stock at a price of $0.52 per unit, and the warrants have an exercise price of $0.575 per
share. The total gross proceeds of the offering were $5,300,001. The warrants issued are exercisable
beginning six months after the closing date until the five-year anniversary of the closing date.
On January 21, 2014 we closed a registered direct public offering of 19,047,620 shares of
common stock and warrants to purchase up to 4,761,905 shares of common stock. The common stock
and warrants were sold in units consisting of one share of common stock and a warrant to purchase 0.25
shares of common stock, at a price of $1.05 per unit, and the warrants have an exercise price of $1.28 per
share. The total gross proceeds of the offering were $20,000,001. The warrants issued are exercisable
beginning six months and one day after the closing date until the five-year anniversary of the closing date.
On November 12, 2015 we closed a registered direct public offering of 16,666,667 shares of
common stock and warrants to purchase up to 12,500,000 shares of common stock. The common stock
and warrants were sold in units consisting of one share of common stock and a warrant to purchase 0.75
shares of common stock at a price of $0.42 per unit, and the warrants have an exercise price of $0.53 per
share. The total gross proceeds of the offering were $7,000,000. The warrants issued are exercisable
beginning six months after the closing date until the five-year anniversary of the initial exercise date.
On March 2, 2016 we closed a registered direct public offering of 15,625,000 shares of common
stock and warrants to purchase up to 11,718,750 shares of common stock. The common stock and
warrants were sold in units consisting of one share of common stock and a warrant to purchase 0.75
shares of common stock at a price of $0.32 per unit, and the warrants have an exercise price of $0.42 per
share. The total gross proceeds of the offering were $5,000,000. The warrants are exercisable
beginning six months after the closing date until the five-year anniversary of the initial exercise date.
We will need to raise additional capital through public or private equity or debt offerings or
through arrangements with strategic partners or other sources in order to continue to develop our drug
candidates. There can be no assurance that additional capital will be available when needed or on terms
satisfactory to us, if at all. If we are not able to raise sufficient additional capital, we will have to reduce
our research and development activities. We will first reduce research and development activities
associated with our pre-clinical compounds. To the extent necessary, we will then reduce our research
and development activities related to some or all of our clinical drugs.
At Market Issuance Sales Agreement
On March 16, 2015, we entered into an at market issuance sales agreement (the ―Sales
Agreement‖) with MLV & Co. LLC (―MLV‖), pursuant to which we may issue and sell shares of our
52
common stock having an aggregate offering price of up to $40 million from time to time, at our option,
through MLV as our sales agent, subject to certain terms and conditions. Any shares sold will be sold
pursuant to our effective shelf registration statement on Form S-3 (File No. 333-196255), as
supplemented by a prospectus supplement dated March 16, 2015. We will pay MLV a commission of
3.0% of the gross proceeds of the sale of any shares sold through MLV. During the year ended
December 31, 2015, we sold 1,407,072 shares of common stock pursuant to the Sales Agreement for
$1,042,573 in gross proceeds at a weighted average price of $0.7410 per share. Net proceeds to us were
$1,005,715 after deducting commissions and other transaction costs. We are not obligated to make any
further sales under the Sales Agreement and no assurance can be given that we will sell any further shares
under the Sales Agreement, or, if we do, as to the price or amount of shares that we will sell, or the dates
on which any such sales will take place. Pursuant to the securities purchase agreement entered into in
connection with our registered direct offering, which closed on March 2, 2016, we are prohibited from
selling shares under the Sales Agreement until March 2, 2017.
Contractual Obligations
The following table summarizes our contractual obligations as of December 31, 2015:
Operating Leases
Total
951,296 $
$
Less than 1
year
269,733 $
1 -3 Years
3-5 Years
More than 5
years
494,140 $
187,423 $
-
We also have obligations under various license agreements that become due and payable on the
achievement of certain development, regulatory, or commercial milestones. We have not included these
commitments on our balance sheet or in the above table of contractual obligations because the
achievement and timing of these events is neither fixed nor determinable.
We have contracted with various vendors for research and development services, the terms of
which require payments over the term of the agreements, usually ranging from two to 36 months. The
costs to be incurred are estimated and are subject to revision. As of December 31, 2015, the total
estimated cost to complete these agreements was approximately $9,380,000. All of these agreements
may be terminated by either party upon appropriate notice as stipulated in the respective agreements, and
therefore, are not included in the above table of contractual obligations.
Current and Future Financing Needs
We have incurred negative cash flow from operations since we started our business. We have
spent, and expect to continue to spend, substantial amounts in connection with implementing our business
strategy, including our planned product development efforts, our clinical trials and our research and
development efforts. We will need to raise additional capital through public or private equity or debt
offerings or through arrangements with strategic partners or other sources in order to continue to develop
our drug candidates. There can be no assurance that additional capital will be available when needed or
on terms satisfactory to us, if at all. If we are not able to raise sufficient additional capital, we will have
to reduce our research and development activities. We believe our cash, cash equivalents, and marketable
securities will be sufficient to cover our cash flow requirements for our current activities for at least the
next 12 months.
53
The actual amount of funds we will need to operate is subject to many factors, some of which are
beyond our control. These factors include the following:
the progress of our product development activities;
the number and scope of our product development programs;
the progress of our pre-clinical and clinical trial activities;
the progress of the development efforts of parties with whom we have entered into
collaboration agreements;
our ability to maintain current collaboration programs and to establish new collaboration
arrangements;
the costs involved in prosecuting and enforcing patent claims and other intellectual
property rights; and
the costs and timing of regulatory approvals.
Off-Balance Sheet Arrangements
We do not have any off-balance sheet arrangements or holdings in variable interest entities.
54
Item 7A. Quantitative and Qualitative Disclosures About Market Risk.
For the year ended December 31, 2015, we are exposed to the following market risks:
Interest Rate Risk
We invest our cash in a variety of financial instruments. At December 31, 2015, our cash was
invested primarily in short term bank deposits and municipal obligations, all of which were denominated
in U.S. dollars. Due to the conservative nature of these investments, which primarily bear interest at
fixed rates, we do not believe we have material exposure to interest rate risk.
Foreign Currency Risk
We are exposed to risks associated with foreign currency transactions on contracts with vendors
associated outside of the United States. Accordingly changes in the value of the U.S. dollar, relative to
other currencies, may have an impact on our financial statements and earnings. The number and dollar
amount of contracts denominated in foreign currency is immaterial; therefore, we believe we do not have
material exposure to foreign currency risk.
Item 8. Financial Statements and Supplementary Data.
Our financial statements and the Report of the Independent Registered Public Accounting Firm
thereon filed pursuant to this Item 8 and are included in this Annual Report on Form 10-K beginning on
page F-1.
55
Item 9. Changes In and Disagreements With Accountants on Accounting and Financial Disclosure.
None.
Item 9A. Controls and Procedures.
Evaluation of Disclosure Controls and Procedures. Under the supervision and with the
participation of our management, including our Chief Executive Officer and Chief Financial Officer, we
evaluated the effectiveness of the design and operation of our disclosure controls and procedures (as
defined in Rule 13a-15(e) and 15d-15(e) under the Exchange Act) as of the end of the period covered by this
report. Based upon that evaluation, our Chief Executive Officer and Chief Financial Officer concluded that
our disclosure controls and procedures as of the end of the period covered by this report were effective such
that the information required to be disclosed by us in reports filed under the Exchange Act is (i) recorded,
processed, summarized and reported within the time periods specified in the SEC‘s rules and forms and
(ii) accumulated and communicated to our management, including our Chief Executive Officer and Chief
Financial Officer, as appropriate to allow timely decisions regarding disclosure. A controls system cannot
provide absolute assurance, however, that the objectives of the controls system are met, and no evaluation
of controls can provide absolute assurance that all control issues and instances of fraud, if any, within a
company have been detected.
Changes in Internal Control Over Financial Reporting. During the most recent quarter ended
December 31, 2015, there has been no change in our internal control over financial reporting (as defined in
Rule 13a-15(f) and 15d-15(f) under the Exchange Act) ) that has materially affected, or is reasonably likely
to materially affect, our internal control over financial reporting.
56
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 Rule 13a-15(f) under the Exchange Act). Our internal control over
financial reporting is a process designed to provide reasonable assurance regarding the reliability of
financial reporting and the preparation of financial statements for external purposes in accordance with
generally accepted accounting principles and includes those policies and procedures that:
Pertain to the maintenance of records that, in reasonable detail, accurately and fairly
reflect the transactions and the dispositions of our assets;
Provide reasonable assurance that transactions are recorded as necessary to permit
preparation of financial statements in accordance with generally accepted
accounting principles, and that our receipts and expenditures are being made only in
accordance with authorization of our management and the board of directors; and
Provide reasonable assurance regarding prevention or timely detection of
unauthorized acquisition, use, or disposition of our assets that could have a material
effect on the financial statements.
Because of its inherent limitations, internal control over financial reporting may not prevent or detect
misstatements. Also, projections of any evaluations of effectiveness to future periods are subject to risk
that controls may become inadequate because of changes in conditions or because of declines in the degree
of compliance with the policies or procedures.
Our management, with the participation of the Chief Executive Officer and Chief Financial Officer,
assessed the effectiveness of our internal control over financial reporting as of December 31, 2015. In
making this assessment, our management used the criteria set forth by the Committee of Sponsoring
Organizations of the Treadway Commission in the Internal Control-Integrated Framework (2013).
Based on this evaluation, our management, with the participation of the Chief Executive Officer and
Chief Financial Officer, concluded that, as of December 31, 2015 our internal control over financial
reporting was effective.
Management‘s assessment of the effectiveness of the Company‘s internal control over financial
reporting has been audited by Baker Tilly Virchow Krause, LLP, an independent registered public
accounting firm. Baker Tilly Virchow Krause, LLP has issued an attestation report on the effectiveness of
the Company‘s internal control over financial reporting, which appears herein.
57
Report of Independent Registered Public Accounting Firm
Board of Directors and Stockholders
Rexahn Pharmaceuticals, Inc.
We have audited Rexahn Pharmaceuticals Inc.‘s internal control over financial reporting as of December
31, 2015, based on criteria established in Internal Control—Integrated Framework (2013 framework)
issued by the Committee of Sponsoring Organizations of the Treadway Commission (COSO). Rexahn
Pharmaceuticals Inc.‘s management is responsible for maintaining effective internal control over financial
reporting, and for its assessment of the effectiveness of internal control over financial reporting, included in
the accompanying Management‘s Report on Internal Control over Financial Reporting. Our responsibility
is to express an opinion on the entity‘s internal control over financial reporting based on our audit.
We conducted our audit 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 effective internal control over financial reporting was maintained in all material
respects. Our audit of internal control over financial reporting included obtaining an understanding of
internal control over financial reporting, assessing the risk that a material weakness exists, and testing and
evaluating the design and operating effectiveness of internal control based on the assessed risk. Our audit
also included performing such other procedures as we considered necessary in the circumstances. We
believe that our audit provides a reasonable basis for our opinion.
An entity‘s internal control over financial reporting is a process designed to provide reasonable assurance
regarding the reliability of financial reporting and the preparation of financial statements for external
purposes in accordance with accounting principles generally accepted in the United States of America. An
entity‘s internal control over financial reporting includes those policies and procedures that (1) pertain to
the maintenance of records that, in reasonable detail, accurately and fairly reflect the transactions and
dispositions of the assets of the entity; (2) provide reasonable assurance that transactions are recorded as
necessary to permit preparation of financial statements in accordance with generally accepted accounting
principles, and that receipts and expenditures of the entity are being made only in accordance with
authorizations of management and directors of the entity; and (3) provide reasonable assurance regarding
prevention or timely detection of unauthorized acquisition, use, or disposition of the entity‘s assets that
could have a material effect on the financial statements.
Because of its inherent limitations, internal control over financial reporting may not prevent or detect
misstatements. Also, projections of any evaluation of effectiveness to future periods are subject to the risk
that controls may become inadequate because of changes in conditions, or that the degree of compliance
with the policies or procedures may deteriorate.
In our opinion, Rexahn Pharmaceuticals Inc. maintained, in all material respects, effective internal control
over financial reporting as of December 31, 2015, based on criteria established in Internal
Control—Integrated Framework (2013 framework) issued by the Committee of Sponsoring Organizations
of the Treadway Commission (COSO).
58
We have also audited, in accordance with the standards of the Public Company Accounting Oversight
Board (United States), the balance sheet and the related statements of operations, comprehensive loss,
stockholders‘ equity, and cash flows of Rexahn Pharmaceuticals Inc., and our report dated March 14, 2016
expressed an unqualified opinion.
/s/ Baker Tilly Virchow Krause, LLP
Wyomissing, Pennsylvania
March 14, 2016
59
Item 9B. Other Information.
None.
60
Item 10. Directors, Executive Officers and Corporate Governance.
PART III
The information required by this Item is set forth in our 2016 Proxy Statement to be filed with the
SEC within 120 days of December 31, 2015 and is incorporated into this Annual Report by reference.
Item 11. Executive Compensation.
The information required by this Item is set forth in our 2016 Proxy Statement to be filed with the
SEC within 120 days of December 31, 2015 and is incorporated into this Annual Report by reference.
Item 12. Security Ownership of Certain Beneficial Owners and Management and Related
Stockholder Matters.
The information required by this Item is set forth in our 2016 Proxy Statement to be filed with the
SEC within 120 days of December 31, 2015 and is incorporated into this Annual Report by reference.
Item 13. Certain Relationships and Related Transactions; and Director Independence.
The information required by this Item is set forth in our 2016 Proxy Statement to be filed with the
SEC within 120 days of December 31, 2015 and is incorporated into this Annual Report by reference.
Item 14. Principal Accounting Fees and Services.
The information required by this Item is set forth in our 2016 Proxy Statement to be filed with the
SEC within 120 days of December 31, 2015 and is incorporated into this Annual Report by reference.
61
Item 15. Exhibits, Financial Statement Schedules.
(a) The following documents are filed as a part of this Annual Report:
(1) The following documents are filed as a part of this Annual Report:
Report of Baker Tilly Virchow Krause, LLP
Balance Sheet as of December 31, 2015 and December 31, 2014
Statement of Operations for the years ended December 31, 2015, 2014 and 2013
F-1
F-2
F-3
Statement of Comprehensive Loss for the years ended December 31, 2015, 2014 and
2013
F-4
Statement of Stockholders‘ Equity for the years ended December 31, 2015, 2014 and 2013 F-5
Statement of Cash Flows for the years ended December 31, 2015, 2014 and 2013
Notes to the Financial Statements
F-6
F-7
(2) All financial statement schedules have been omitted because they are not applicable or not
required or because the information is included elsewhere in the financial statements or the
Notes thereto.
(3) See the accompanying Index to Exhibits filed as a part of this Annual Report, which list is
incorporated by reference in this Item.
(b) See the accompanying Index to Exhibits filed as a part of this Annual Report.
(c) Other schedules are not applicable.
62
SIGNATURES
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.
REXAHN PHARMACEUTICALS, INC.
By: /s/ Peter D. Suzdak
Peter D. Suzdak
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:
Name
Title
Date
/s/ Peter Suzdak*
Peter Suzdak
/s/ Tae Heum Jeong*
Tae Heum Jeong
/s/ Peter Brandt*
Peter Brandt
/s/ Chang H. Ahn*
Chang H. Ahn
/s/ Charles Beever*
Charles Beever
/s/ Kwang Soo Cheong*
Kwang Soo Cheong
/s/ Mark Carthy*
Mark Carthy
/s/ Richard J. Rodgers*
Richard J. Rodgers
Chief Executive Officer and
Director (Principal Executive
Officer)
March 14, 2016
Chief Financial Officer, and
Secretary (Principal Financial and
Accounting Officer)
March 14, 2016
Chairman
March 14, 2016
Director
Director
Director
Director
Director
March 14, 2016
March 14, 2016
March 14, 2016
March 14, 2016
March 14, 2016
* By: /s/ Tae Heum Jeong, Attorney-in Fact
Tae Heum Jeong, Attorney-in-Fact**
** By authority of the power of attorney filed as Exhibit 24 hereto.
63
Report of Independent Registered Public Accounting Firm
Board of Directors and Stockholders
Rexahn Pharmaceuticals, Inc.
We have audited the accompanying balance sheet of Rexahn Pharmaceuticals, Inc. as of December 31, 2015
and 2014, and the related statements of operations, comprehensive loss, stockholders‘ equity, and cash flows
for each of the years in the three-year period ended December 31, 2015. These financial statements are the
responsibility of the entity‘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. 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 financial
position of Rexahn Pharmaceuticals, Inc. as of December 31, 2015 and 2014, and the results of its operations
and its cash flows for each of the years in the three-year period ended December 31, 2015, in conformity with
accounting principles generally accepted in the United States of America.
We also have audited, in accordance with the standards of the Public Company Accounting Oversight Board
(United States), Rexahn Pharmaceuticals, Inc.‘s internal control over financial reporting as of December 31,
2015, based on criteria established in Internal Control—Integrated Framework (2013 framework) issued by
the Committee of Sponsoring Organizations of the Treadway Commission (COSO), and our report dated
March 14, 2016 expressed an unqualified opinion.
/s/ Baker Tilly Virchow Krause, LLP
Wyomissing, Pennsylvania
March 14, 2016
F-1
REXAHN PHARMACEUTICALS, INC.
Balance Sheet
Current Assets:
Cash and cash equivalents
Marketable securities
Prepaid expenses and other current assets
Total Current Assets
Security Deposits
Equipment, Net
Total Assets
Current Liabilities:
December 31, 2015
December 31, 2014
ASSETS
$
$
10,199,440 $
13,240,086
1,221,818
24,661,344
30,785
112,900
24,805,029 $
9,826,245
22,872,051
730,987
33,429,283
25,681
78,096
33,533,060
LIABILITIES AND STOCKHOLDERS’ EQUITY
Accounts payable and accrued expenses
$
2,661,298 $
2,459,263
Deferred Research and Development Arrangement
525,000
600,000
Other Liabilities
Warrant Liabilities
Total Liabilities
Commitments and Contingencies (note 14)
Stockholders’ Equity:
Preferred stock, par value $0.0001, 100,000,000 authorized
shares, none issued and outstanding
Common stock, par value $0.0001, 500,000,000 authorized
shares, 197,413,785 and 178,366,533 issued and
197,413,785 and 178,253,318 outstanding
Additional paid-in capital
Accumulated other comprehensive loss
Accumulated deficit
Treasury stock, 0 and 113,215 shares, at cost
104,020
124,955
2,739,163
3,768,351
6,029,481
6,952,569
-
-
19,741
124,490,712
(18,041)
(105,716,864)
-
17,837
118,057,019
(33,647)
(91,332,308)
(128,410)
Total Stockholders’ Equity
18,775,548
26,580,491
Total Liabilities and Stockholders’ Equity
$
24,805,029 $
33,533,060
(See accompanying notes to the financial statements)
F-2
REXAHN PHARMACEUTICALS, INC.
Statement of Operations
Revenues:
Expenses:
For the Year Ended December 31,
2015
2014
2013
$
- $
- $
-
General and administrative
Research and development
6,115,210
12,148,226
6,253,328
7,015,901
4,725,699
3,253,139
Total Expenses
18,263,436
13,269,229
7,978,838
Loss from Operations
(18,263,436)
(13,269,229)
(7,978,838)
Other Income (Expense)
Interest income
103,269
133,907
49,280
Unrealized gain (loss) on fair value of warrants
3,986,727
(5,180,107)
(1,365,654)
Financing expense
Total Other Income (Expense)
(211,116)
(206,172)
(204,212)
3,878,880
(5,252,372)
(1,520,586)
Net Loss Before Provision for Income Taxes
(14,384,556)
(18,521,601)
(9,499,424)
Provision for income taxes
Net Loss
-
-
-
$
(14,384,556) $
(18,521,601) $
(9,499,424)
Net loss per share, basic and diluted
$
(0.08) $
(0.11) $
(0.07)
Weighted average shares outstanding, basic and diluted
182,388,226
176,106,981
128,649,303
(See accompanying notes to the financial statements)
F-3
REXAHN PHARMACEUTICALS, INC.
Statement of Comprehensive Loss
For the Year Ended December 31,
2014
2015
2013
Net Loss
$
(14,384,556) $
(18,521,601) $
(9,499,424)
Unrealized gain (loss) on available-for-sale securities
15,606
(33,647)
-
Comprehensive Loss
$
(14,368,950) $
(18,555,248) $
(9,499,424)
(See accompanying notes to the financial statements)
F-4
REXAHN PHARMACEUTICALS, INC.
Statement of Stockholders‘ Equity
For the Year Ended December 31, 2015, 2014 and 2013
Balances at
December 31, 2012
Issuance of common
stock and units
Stock issuance costs
Common stock issued
in exchange for services
Stock options exercised
Stock warrants
exercised
Stock based
compensation
Net loss
Balances at
December 31, 2013
Issuance of common
stock and units
Stock issuance costs
Common stock issued
in exchange for services
Stock options exercised
Shares surrendered for
net stock option
exercise
Stock warrants
exercised
Stock based
compensation
Net loss
Other comprehensive
loss
Balances at
December 31, 2014
Issuance of common
stock and units
Stock issuance costs
Common stock issued
in exchange for services
Stock options exercised
Stock warrants
exercised
Stock based
compensation
Retirement of treasury
stock
Net loss
Other comprehensive
loss
Balances at
December 31, 2015
Common Stock
Treasury Stock
Number of
Shares
Amount
Additional
Paid-in
Capital
Accumulated
Deficit
Number
of Shares Amount
Accumulated
Other
Comprehensive
Loss
Total
Stockholders'
Equity
119,443,194
11,944
72,861,738
(63,311,283)
14,205
(28,410)
21,592,309
-
2,159
-
8,631,696
(952,490)
640,000
375,000
64
38
306,736
89,962
4,681,497
468
3,946,862
-
-
-
-
-
-
-
-
-
565,428
-
-
(9,499,424)
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
9,533,989
8,633,855
(952,490)
306,800
90,000
3,947,330
565,428
(9,499,424)
146,732,000 $
14,673 $ 85,449,932 $ (72,810,707)
14,205 $ (28,410) $
- $
12,625,488
19,047,620
-
1,905
-
16,306,667
(1,159,582)
400,000
448,693
40
45
408,960
358,910
-
-
-
11,738,220
1,174
16,083,337
-
-
-
-
-
-
-
-
-
-
-
-
608,795
-
-
(18,521,601)
-
-
-
-
-
-
- -
-
-
-
99,010
(100,000)
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
16,308,572
(1,159,582)
409,000
358,955
(100,000)
16,084,511
608,795
(18,521,601)
(33,647)
(33,647)
178,366,533 $
17,837 $
118,057,019 $ (91,332,308)
113,215 $
(128,410) $
(33,647) $
26,580,491
18,073,739
-
1,807
-
5,248,266
(566,065)
150,000
889,428
47,300
15
89
4
101,985
708,528
31,699
-
-
1,037,679
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
(113,215)
-
(11)
-
(128,399)
-
-
(14,384,556)
(113,215)
-
128,410
-
-
-
-
-
-
-
-
-
5,250,073
(566,065)
102,000
708,617
31,703
1,037,679
-
(14,384,556)
-
-
-
-
197,413,785 $
19,741 $
124,490,712 $
(105,716,864)
-
- $
-
15,606
15,606
- $
(18,041) $
18,775,548
(See accompanying notes to the financial statements)
F-5
REXAHN PHARMACEUTICALS, INC.
Statement of Cash Flows
Cash Flows from Operating Activities:
Net loss
Adjustments to reconcile net loss to net cash used in operating activities:
Compensatory stock
Depreciation and amortization
Amortization of premiums and discounts on marketable securities, net
Stock-based compensation
Amortization of deferred research and development arrangements
Unrealized (gain) loss on fair value of warrants
Financing expense
Amortization of deferred lease incentive
Deferred lease expenses
Changes in assets and liabilities:
Prepaid expenses and other assets
Accounts payable and accrued expenses
Net Cash Used in Operating Activities
Cash Flows from Investing Activities:
Restricted cash equivalents
Purchase of equipment
Purchase of marketable securities
Redemption of marketable securities
Net Cash Provided by (Used In) Investing Activities
Cash Flows from Financing Activities:
Issuance of common stock and units, net of issuance costs
Proceeds from exercise of stock options
Proceeds from exercise of stock warrants
Net Cash Provided by Financing Activities
Net Increase (Decrease) in Cash and Cash Equivalents
Cash and Cash Equivalents – beginning of period
Cash and Cash Equivalents - end of period
Supplemental Cash Flow Information
Non-cash financing and investing activities:
Warrants issued
Warrant liability extinguishment from exercise of warrants
Shares withheld for net stock option exercise
Leasehold improvement incentive
Retirement of treasury stock
For the Year Ended December 31,
2013
2014
2015
$ (14,384,556) $ (18,521,601) $
(9,499,424)
102,000
27,498
30,875
1,037,679
(75,000)
(3,986,727)
211,116
(12,443)
(8,492)
409,000
28,325
10,228
608,795
(233,630)
5,180,107
206,172
(12,443)
7,834
306,800
37,133
-
565,428
(792,370)
1,365,654
204,212
(16,222)
25,709
(495,935)
202,035
(17,351,950)
(249,503)
1,525,505
(11,041,211)
(263,697)
81,921
(7,984,856)
-
(62,302)
(7,908,304)
17,525,000
9,554,394
196,130
(41,249)
(26,075,926)
3,260,000
(22,661,045)
895,671
(50,149)
-
-
845,522
7,439,809
708,617
22,325
8,170,751
373,195
9,826,245
10,199,440 $
$
18,634,247
258,955
5,947,268
24,840,470
(8,861,786)
18,688,031
9,826,245 $
10,041,155
90,000
2,209,667
12,340,822
5,201,488
13,486,543
18,688,031
$
$
$
$
$
2,966,917 $
9,378 $
- $
- $
128,410 $
3,691,429 $
10,137,243 $
100,000 $
- $
- $
2,564,002
1,737,663
-
54,660
-
(See accompanying notes to the financial statements)
F-6
REXAHN PHARMACEUTICALS, INC.
Notes to Financial Statements
1. Operations and Organization
Operations
Rexahn Pharmaceuticals, Inc. (the ―Company,‖), a Delaware corporation, is a biopharmaceutical
company whose principal operations are the discovery, development and commercialization of
innovative treatments for cancer. The Company had an accumulated deficit of $105,716,864 at
December 31, 2015 and anticipates incurring losses through fiscal year 2016 and beyond. The
Company has not yet generated commercial revenues and has funded its operating losses to date
through the sale of shares of its common stock and warrants to purchase shares of its common stock,
convertible debt, financings, interest income from cash, cash equivalents and marketable securities,
and proceeds from reimbursed research and development costs. The Company believes that its
cash, cash equivalents, and marketable securities, will be sufficient to cover its cash flow
requirements for its current activities for at least the next 12 months. Management believes it has
the capability of managing the Company‘s operations within existing cash available by focusing on
select research and development activities, selecting projects in conjunction with potential
financings and milestones, and efficiently managing its general and administrative affairs.
2. Summary of Significant Accounting Policies
a) Cash and Cash Equivalents
Cash and cash equivalents include cash on hand and short-term investments purchased with
remaining maturities of three months or less at acquisition.
b) Marketable Securities
Marketable securities are considered ―available-for-sale‖ in accordance with Financial Statement
Accounting Board (―FASB‖) Accounting Standards Codification (―ASC‖) 320, ―Debt and Equity
Securities‖, and thus are reported at fair value in the Company‘s accompanying balance sheet, with
unrealized gains and losses excluded from earnings and reported as a separate component of
stockholders‘ equity. Amounts reclassified out of accumulated other comprehensive loss into
realized gains and losses are accounted for on the basis of specific identification and are included in
other income or expense in the statement of operations. The Company classifies such investments
as current on the balance sheet as the investments are readily marketable and available for use in
Rexahn Pharmaceuticals‘ current operations.
c) Equipment
Equipment is stated at cost less accumulated depreciation. Depreciation, based on the lesser of the
term of the lease or the estimated useful life of the assets, is provided as follows:
Life Depreciation Method
Furniture and fixtures
Office equipment
Lab equipment
Computer equipment
Leasehold improvements
7 years straight line
5 years straight line
5-7 years straight line
3-5 years straight line
3-5 years straight line
F-7
REXAHN PHARMACEUTICALS, INC.
Notes to Financial Statements
d) Research and Development
Research and development costs are expensed as incurred. Research and development expenses
consist primarily of third party service costs under research and development agreements, salaries
and related personnel costs, as well as stock compensation related to these costs, costs to acquire
pharmaceutical products and product rights for development and amounts paid to contract research
organizations, hospitals and laboratories for the provision of services and materials for drug
development and clinical trials.
Costs incurred in obtaining the licensing rights to technology in the research and development stage
that have no alternative future uses and are for unapproved product compounds are expensed as
incurred.
e) Use of Estimates
The preparation of financial statements in conformity with accounting principles generally
accepted in the United States 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. These estimates are based on management‘s best knowledge of current events
and actions the Company may undertake in the future. Actual results may ultimately differ from
these estimates. These estimates are reviewed periodically and as adjustments become necessary,
they are reported in earnings in the period in which they become available.
f) Fair Value of Financial Instruments
The carrying amounts reported in the accompanying financial statements for cash and cash
equivalents, prepaid expenses and other current assets, the security deposit and accounts payable
and accrued expenses approximate fair value because of the short-term maturity of these financial
instruments. The fair value for marketable securities, warrant liabilities, and certain other assets
and liabilities is discussed in Notes 3, 12, and 15, respectively.
F-8
REXAHN PHARMACEUTICALS, INC.
Notes to Financial Statements
g) Income Taxes
The Company accounts for income taxes in accordance with ASC 740, ―Income Taxes‖. Deferred
tax assets and liabilities are recorded for differences between the financial statement and tax basis
of the assets and liabilities that will result in taxable or deductible amounts in the future based on
enacted tax laws and rates. ASC 740 requires that a valuation allowance be established when it is
more likely than not that all portions of a deferred tax asset will not be realized. A review of all
positive and negative evidence needs to be considered, including a company‘s current and past
performance, the market environment in which the company operates, length of carryback and
carryforward periods and existing contracts that will result in future profits. Income tax expense is
recorded for the amount of income tax payable or refundable for the period, increased or decreased
by the change in deferred tax assets and liabilities during the period.
As a result of the Company‘s significant cumulative losses, the Company determined that it was
appropriate to establish a valuation allowance for the full amount of deferred tax assets.
The calculation of the Company‘s tax liabilities involves the inherent uncertainty associated with
the application of complex tax laws. The Company is subject to examination by various taxing
authorities. The Company believes that, as a result of its loss carryforward sustained to date, any
examination would result in a reduction of its net operating losses rather than a tax liability. As
such, the Company has not provided for any additional taxes that would be estimated under ASC
740.
h) Stock-Based Compensation
In accordance with ASC 718, ―Stock Compensation,‖ compensation costs related to share-based
payment transactions, including employee stock options, are to be recognized in the financial
statements. In addition, the Company adheres to the guidance set forth within Securities and
Exchange Commission (―SEC‖) Staff Accounting Bulletin (―SAB‖) No. 107, which provides the
Staff‘s views regarding the interaction between ASC 718 and certain SEC rules and regulations,
and provides interpretations with respect to the valuation of share-based payments for public
companies.
i) Concentration of Credit Risk
ASC 825, ―Financial Instruments,‖ requires disclosure of any significant off balance sheet risk and
credit risk concentration. The Company does not have significant off-balance sheet risk or credit
concentration. The Company maintains cash and cash equivalents with major financial
institutions. From time to time the Company has funds on deposit with commercial banks that
exceed federally insured limits. The balances are insured by the Federal Deposit Insurance
Corporation up to $250,000. At December 31, 2015, the Company‘s uninsured cash balance was
$9,554,878. Management does not consider this to be a significant credit risk as the banks are large,
established financial institutions.
j) Reclassification
Certain amounts in the prior year‘s financial statements have been reclassified to conform to the
current year presentation with no material effect on the financial statements.
F-9
REXAHN PHARMACEUTICALS, INC.
Notes to Financial Statements
k) Recent Accounting Pronouncements Affecting the Company
Revenue from Contracts with Customers
In May 2014, the FASB issued Accounting Standards Update (―ASU‖) 2014-09, ―Revenue from
Contracts with Customers,‖ a comprehensive new revenue recognition standard that will
supersede nearly all existing revenue recognition guidance under accounting principles generally
accepted in the United States of America. The standard‘s core principle is that a company
should recognize revenue when it transfers goods or services to customers in an amount that
reflects the consideration to which the company expects to be entitled in exchange for those
goods and services, and provides a revenue recognition framework in accordance with this
principle. On August 12, 2015, the FASB issued ASU 2015-14, which defers the effective date
of ASU 2014-09 by one year to December 15, 2017 for annual reporting periods beginning after
that date and interim periods therein. Early adoption of the standard is permitted, but not before
the original effective date of December 15, 2016. The Company is currently evaluating the
impact that the adoption of this guidance will have on its financial statements and future
operating results.
Disclosure of Uncertainties about an Entity’s Ability to Continue as a Going Concern
In August 2014, the FASB issued ASU 2014-15, ―Disclosure of Uncertainties about an Entity‘s
Ability to Continue as a Going Concern,‖ which requires management to perform interim and
annual assessments as to the entity‘s ability to continue as a going concern and provides related
disclosure guidance. ASU 2014-15 will be effective for reporting periods beginning after
December 15, 2016, with early adoption permitted. The Company is currently evaluating the
impact the adoption of this guidance will have on its financial statements.
Leases
In February 2016, the FASB issued ASU 2016-02, ―Leases,‖ which requires that lease
arrangements for a period greater than 12 months result in an entity recognizing an asset and a
liability. ASU 2016-02 will be effective for reporting periods beginning after December 15,
2016, with early adoption permitted. We have not evaluated the impact that the adoption of this
guidance will have on our financial statements.
F-10
REXAHN PHARMACEUTICALS, INC.
Notes to Financial Statements
3. Marketable Securities
The following table shows the Company‘s marketable securities‘ adjusted cost, gross unrealized gains
and losses, and fair value by significant investment category as of December 31, 2015 and 2014:
Certificates of Deposit
Commercial Paper
Corporate Bonds
Total Marketable Securities
Certificates of Deposit
Commercial Paper
Corporate Bonds
Total Marketable Securities
December 31, 2015
Cost
Basis
$
6,240,000 $
2,981,307
4,036,820
$ 13,258,127 $
Gross
Unrealized
Gains
571 $
-
-
571 $
Gross
Unrealized
Losses
Fair
Value
6,234,996
(5,575) $
2,977,570
(3,737)
4,027,520
(9,300)
(18,612) $ 13,240,086
December 31, 2014
Cost
Basis
$ 18,865,000 $
1,998,001
2,042,697
$ 22,905,698 $
Gross
Unrealized
Gains
60 $
62
-
122 $
Fair
Value
Gross
Unrealized
Losses
(26,789) $ 18,838,271
1,997,910
(153)
(6,827)
2,035,870
(33,769) $ 22,872,051
The Company typically invests in highly-rated securities, with the primary objective of minimizing
the potential risk of principal loss. As of December 31, 2015, the Company had 15 certificates of
deposit with a fair value of $3,594,425 and unrealized losses of $5,575, three commercial papers with
a fair value of $2,977,570 and unrealized losses of $3,737, and three corporate bonds with a fair value
of $3,025,320 and unrealized losses of $7,625, all of which have been unrealized losses for less than
12 months. The Company has one corporate bond with a fair value of $1,002,200 and unrealized
losses of $1,675 which has been an unrealized loss for greater than 12 months. The Company does
not intend to sell its marketable securities in an unrealized loss position. Based upon the Company‘s
securities‘ fair value relative to the cost, high ratings, and volatility of fair value, the Company
considers the declines in market value of its marketable securities to be temporary in nature and does
not consider any of its investments other-than-temporarily impaired, and anticipates that it will
recover the entire amortized cost basis.
As of December 31, 2015, all of the Company‘s marketable securities are expected to mature in less
than one year.
F-11
REXAHN PHARMACEUTICALS, INC.
Notes to Financial Statements
4. Prepaid Expenses and Other Current Assets
December 31, December 31,
2015
2014
Deposits on contracts
Prepaid expenses and other current assets
$
501,170 $
720,648
369,811
361,176
$
1,221,818 $
730,987
Deposits on contracts consist of deposits on research and development contracts for services that had
not been incurred as of the balance sheet date. Prepaid expenses and other assets include prepaid
general and administrative expenses, such as insurance, rent, investor relations fees and compensatory
stock issued for services not yet incurred as of the balance sheet date.
5. Equipment, Net
Furniture and fixtures
Office and computer equipment
Lab equipment
Leasehold improvements
December 31, December 31,
2015
2014
$
78,794 $
105,266
431,650
133,762
70,320
57,893
425,195
133,762
Total equipment
Less: Accumulated depreciation and amortization
749,472
(636,572)
687,170
(609,074)
Net carrying amount
$
112,900 $
78,096
6. Accounts Payable and Accrued Expenses
Trade payables
Accrued expenses
Accrued research and development contract costs
Payroll liabilities
F-12
December 31, December 31,
2015
2014
$
774,543 $
92,752
1,515,151
278,852
706,781
56,884
1,078,532
617,066
$
2,661,298 $
2,459,263
REXAHN PHARMACEUTICALS, INC.
Notes to Financial Statements
7. Deferred Research and Development Arrangements
Rexgene Biotech Co., Ltd.
In 2003, the Company entered into a collaborative research agreement with Rexgene Biotech Co.,
Ltd. (―Rexgene‖), a shareholder. Rexgene is engaged in the development of pharmaceutical
products in Asia and has agreed to assist the Company with the research, development and clinical
trials necessary for registration of the Company‘s drug candidate Archexin in Asia. This
agreement provides Rexgene with exclusive rights to license, sublicense, make, have made, use, sell
and import Archexin in Asia. In accordance with the agreement, Rexgene paid the Company a
one-time fee of $1,500,000 in 2003. The agreement terminates at the later of 20 years or the term
of the patent. The amortization reduces research and development expenses for the periods
presented.
The Company is using 20 years as its basis for recognition and accordingly research and
development expenses were reduced by $75,000 for each of the years ended December 31, 2015,
2014 and 2013. The remaining $525,000 and $600,000 to be amortized at December 31, 2015 and
2014, respectively, are reflected as deferred research and development arrangements on the balance
sheet. The payment from Rexgene is being used in the cooperative funding of the costs of
development of Archexin. Royalties of 3% of net sales of licensed products will become payable by
Rexgene to the Company on a quarterly basis once commercial sales of Archexin begin in Asia.
The product is still under development and commercial sales in Asia are not expected to begin until
at least 2017. Under the terms of the agreement, Rexgene does not receive royalties on the
Company‘s net sales outside of Asia.
Teva Pharmaceutical Industries, Ltd.
The Company previously had an arrangement with Teva Pharmaceutical Industries Limited
(―Teva‖) where Teva provided funds for the pre-clinical development of RX-3117. The proceeds
received from Teva were recorded as restricted cash and as a deferred research and development
arrangement on the balance sheet. Costs paid for the development of RX-3117 reduced the
deferred research and development arrangement and therefore were not an expense in the
Company‘s statement of operations. During the years ended December 31, 2014 and 2013,
$158,630 and $717,730 was reduced from deferred research and development arrangements. As of
December 31, 2014, there were no proceeds remaining, and therefore, no deferred research and
development liability relating to Teva.
F-13
REXAHN PHARMACEUTICALS, INC.
Notes to Financial Statements
8. Other Liabilities
Deferred Lease Incentive
On June 29, 2009, the Company entered into a five-year office lease agreement which is further
discussed in Note 14. The lessor agreed to grant a leasehold improvement allowance of $100,000
to the Company to be used for the construction cost of improvements to the leased property, which
included architectural and engineering fees, government agency plan check, permit and other fees,
sales and use taxes, testing and inspection costs and telephone and data cabling and wiring in the
premises. The Company accounted for the benefit of the leasehold improvement allowance as a
reduction of rental expense over the five-year term of the office lease.
On June 7, 2013, the Company entered into the first amendment to the lease agreement, also
discussed in Note 14. According to the terms of the amendment, the Company extended the lease
term until June 30, 2019. The lessor agreed to grant an additional leasehold improvement
allowance of $54,660 to the Company to be used for further construction of the leased property,
furniture and equipment. The Company accounts for this benefit, including the unamortized
portion from the original lease agreement, as a reduction of rental expense over the six-year amended
term of the lease.
The following table sets forth the cumulative deferred lease incentive:
Deferred lease incentive
Less accumulated amortization
Balance
Deferred Office Lease Expense
December 31,
2015
December 31,
2014
$
$
154,660 $
(111,108)
154,660
(98,665)
43,552 $
55,995
The lease agreement, as amended, provided for an initial annual base rent with annual increases over
the following six years. The Company recognizes rental expense on a straight-line basis over the
term of the lease, which resulted in a deferred rent liability of $60,468 and $68,960 as of December
31, 2015 and 2014, respectively.
9. Net Loss per Common Share
Basic loss per common share is computed by dividing net loss by the weighted average number of
shares of common stock outstanding for the period. Diluted loss per common share is computed by
dividing net loss by the weighted average number of shares of common stock outstanding, plus the
number of common share equivalents that would be dilutive. As of December 31, 2015, 2014 and
2013, there were stock options and warrants to acquire, in the aggregate, 39,082,886, 24,606,677
and 34,325,663 shares of the Company‘s common stock, respectively, that are potentially dilutive.
However, diluted loss per share for all periods presented is the same as basic loss per share because
the inclusion of common share equivalents would be anti-dilutive.
F-14
REXAHN PHARMACEUTICALS, INC.
Notes to Financial Statements
10. Common Stock
The following transactions occurred during the years ended December 31, 2015, 2014 and 2013:
Public Offerings
July 2013
On July 26, 2013 the Company closed on a registered direct public offering to issue and sell
11,400,000 shares of common stock and warrants to purchase up to 3,990,000 shares of common
stock. The common stock and warrants were sold in units, consisting of common stock and a
warrant to purchase 0.35 shares of common stock, at a price of $0.50 per share, and the warrants
have an exercise price of $0.59 per share. The total gross proceeds of the offering were $5,700,000.
The warrants issued are exercisable beginning six months after the closing date until the five-year
anniversary of the closing date and were recorded as liabilities at fair value.
A summary of the allocation of the proceeds of the offering is shown below:
Gross Proceeds:
$
5,700,000
Allocated to warrant liabilities:
Allocated to common stock and additional paid-in capital
1,295,952
4,404,048
Total allocated gross proceeds:
$
5,700,000
The closing costs of $637,334 included 456,000 warrants valued at $110,489 and $526,845 for
placement agent and other fees. Based upon the estimated fair value of the stock and warrants in
the units, the Company allocated $112,559 to financing expense and $524,775 as stock issuance
costs.
October 2013
On October 16, 2013, the Company closed on a registered direct public offering to issue and sell
10,192,309 shares of common stock and warrants to purchase up to 3,567,309 shares of common
stock. The common stock and warrants were sold in units, consisting of common stock and a
warrant to purchase 0.35 shares of common stock, at a price of $0.52 per share, and the warrants
have an exercise price of $0.575 per share. The total gross proceeds of the offering were
$5,300,001. The warrants issued are exercisable beginning six months after the closing date until
the five-year anniversary of the closing date and were recorded as liabilities at fair value.
A summary of the allocation of the proceeds of the offering is shown below:
Gross Proceeds:
$
5,300,001
Allocated to warrant liabilities:
Allocated to common stock and additional paid-in capital
1,070,193
4,229,808
Total allocated gross proceeds:
$
5,300,001
The closing costs of $519,368 included 407,692 warrants valued at $87,368 and $432,000 for
placement agent and other fees. Based upon the estimated fair value of the stock and warrants in
the units, the Company allocated $91,653 to financing expense and $427,715 as stock issuance costs.
F-15
REXAHN PHARMACEUTICALS, INC.
Notes to Financial Statements
January 2014
On January 21, 2014 the Company closed on a registered direct public offering to issue and sell
19,047,620 shares of common stock and warrants to purchase up to 4,761,905 shares of common
stock. The common stock and warrants were sold in units, consisting of common stock and a
warrant to purchase 0.25 shares of common stock, at a price of $1.05 per share, and the warrants
have an exercise price of $1.28 per share. The total gross proceeds of the offering were
$20,000,001. The warrants issued are exercisable beginning six months and one day after the
closing date until the five-year anniversary of the closing date and were recorded as liabilities at fair
value.
A summary of the allocation of the proceeds of the offering is shown below:
Gross Proceeds:
$
20,000,001
Allocated to warrant liabilities:
Allocated to common stock and additional paid-in capital
3,691,429
16,308,572
Total allocated gross proceeds:
$
20,000,001
The total closing costs of the offering were $1,365,754, which consisted of placement agent and
other professional fees. Based upon the estimated fair value of the stock and warrants in the units,
the Company allocated $206,172 to financing expense and $1,159,582 as stock issuance costs.
November 2015
On November 12, 2015, the Company closed on a registered direct public offering to issue and sell
16,666,667 shares of common stock and warrants to purchase up to 12,500,000 shares of common
stock. The common stock and warrants were sold in units, consisting of common stock and a
warrant to purchase 0.75 shares of common stock, at a price of $0.42 per share, and the warrants
have an exercise price of $0.53 per share. The total gross proceeds of the offering were $7,000,000.
The warrants issued are exercisable beginning six months after the closing date until the five-year
anniversary of the initial exercise date and were recorded as liabilities at fair value.
A summary of the allocation of the proceeds of the offering is shown below:
Gross Proceeds:
$
7,000,000
Allocated to warrant liabilities:
Allocated to common stock and additional paid-in capital
2,792,500
4,207,500
Total allocated gross proceeds:
$
7,000,000
The closing costs of $740,323 included 833,333 warrants valued at $174,417 and $565,906 for
placement agent and other fees. Based upon the estimated fair value of the stock and warrants in
the units, the Company allocated $211,116 to financing expense and $529,207 as stock issuance
costs.
F-16
REXAHN PHARMACEUTICALS, INC.
Notes to Financial Statements
At Market Offering
On March 16, 2015, the Company entered into an at market (―ATM‖) issuance sales agreement (the
―Sales Agreement‖) with MLV & Co. LLC (―MLV‖) pursuant to which the Company may issue and
sell shares of its common stock having an aggregate offering price of up to $40 million from time to
time, at its option, through MLV as its sales agent, subject to certain terms and conditions. Any
shares sold will be sold pursuant to the Company‘s effective shelf registration statement on Form S-3
(File No. 333-196255), as supplemented by a prospectus supplement dated March 16, 2015. The
Company will pay MLV a commission of 3.0% of the gross proceeds of the sale of any shares sold
through MLV. For the year ended December 31, 2015, the Company sold 1,407,072 shares of
common stock pursuant to the Sales Agreement for $1,042,573 in gross proceeds at a weighted
average price of $0.7410 per share. Net proceeds to the Company were $1,005,715 after deducting
commissions and other transaction costs. Pursuant to the securities purchase agreement entered
into in connection with the Company‘s registered direct offering that closed on March 2, 2016, the
Company is prohibited from selling shares under the Sales Agreement until March 2, 2017. See
Note 17, ―Subsequent Events‖, for information on this registered direct offering.
Compensatory Shares
The Company has issued shares to vendors in exchange for services. A summary of the shares
issued, and the related market value is shown below:
Number of Shares
Issued
Market Value Per
Share
Total Market Value of Share
Issuance
37,200
100,000
63,600
106,000
306,800
336,000
73,000
409,000
56,250
45,750
102,000
Date of Issuance
May 10, 2013
June 10, 2013
August 1, 2013
October 1, 2013
2013 Total
120,000 $
200,000
120,000
200,000
640,000
0.31 $
0.50
0.53
0.53
$
February 10, 2014
300,000 $
1.12 $
August 1, 2014
2014 Total
100,000
400,000
0.73
$
February 10, 2015
75,000 $
0.75 $
July 1, 2015
2015 Total
75,000
150,000
0.61
$
F-17
REXAHN PHARMACEUTICALS, INC.
Notes to Financial Statements
Stock Option and Stock Warrant Exercises
The table below summarizes stock options and stock warrants exercised:
Stock Option Exercises
Number of shares issued
Total cash received
Stock Warrant Exercises
Number of shares issued
Total cash received
Treasury Stock Transactions
$
$
For the Year Ended December 31,
2015
2014
2013
889,428
708,617 $
448,693
258,955 $
375,000
90,000
47,300
22,325 $
11,738,220
5,947,268 $
4,681,497
2,209,667
On April 14, 2014, an option holder exercised stock options by a net exercise. The Company
withheld 99,010 shares in treasury as payment for the $100,000 aggregate exercise price.
On December 3, 2015, the Company retired 113,215 shares of treasury stock with an aggregate
purchase price of $128,410.
F-18
REXAHN PHARMACEUTICALS, INC.
Notes to Financial Statements
11. Stock-Based Compensation
As of December 31, 2015, the Company had 12,590,982 options outstanding.
At the Company‘s Annual Meeting of the Stockholders held on June 10, 2013, the Company‘s
stockholders voted to approve the Rexahn Pharmaceuticals, Inc. 2013 Stock Option Plan (the ―2013
Plan‖). Under the 2013 Plan, the Company grants stock options to key employees, directors and
consultants of the Company. A total of 17,000,000 shares of common stock have been reserved for
issuance pursuant to the 2013 Plan. As of December 31, 2015, there were 6,912,482 options
outstanding under the 2013 Plan, and 10,080,018 shares were available for issuance.
On August 5, 2003, the Company established a stock option plan (the ―2003 Plan‖). Under the
2003 Plan, the Company granted stock options to key employees, directors and consultants of the
Company. With the adoption of the 2013 Plan, no new stock options may be issued under the 2003
Plan, but previously issued options under the 2003 Plan remain outstanding until their expiration.
As of December 31, 2015, there were 5,678,500 outstanding options under the 2003 Plan.
For the majority of the grants to employees, the vesting period is either i) 30%, 30% and 40% on the
first, second and third anniversaries, of the grant date, respectively, or ii) 25% each on the first four
anniversaries. Options expire between five and ten years from the date of grant. For grants to
non-employee consultants of the Company, the vesting period is between one and three years,
subject to the fulfillment of certain conditions in the individual stock agreements, or 100% upon the
occurrence of certain events specified in the individual stock agreements.
Accounting for Awards
The Company‘s results of operations for the years ended December 31, 2015, 2014 and 2013 include
stock-based compensation expense totaling $1,037,679, $608,795 and $565,428 respectively. Such
amounts have been included in the statement of operations in general and administrative and
research and development expenses. No income tax benefit has been recognized in the statement of
operations for stock-based compensation arrangements as the Company has provided for a 100%
valuation allowance on its deferred tax assets.
Stock option compensation expense is the estimated fair value of options granted amortized on a
straight-line basis over the requisite vesting service period for the entire portion of the award.
F-19
REXAHN PHARMACEUTICALS, INC.
Notes to Financial Statements
Summary of Stock Compensation Expense Recognized
Total stock-based compensation recognized by the Company for the years ended December 31,
2015, 2014 and 2013 is as follows:
For the Year Ended December 31,
2014
2013
2015
Statement of operations line item:
General and administrative
Research and development
$
665,063 $
372,616
457,128 $
151,667
503,076
62,352
Total
$
1,037,679 $
608,795 $
565,428
Summary of Stock Option Transactions
There were 4,201,316 stock options granted at exercise prices ranging from $0.54 to $0.89 with an
aggregate fair value of $1,994,893 during the year ended December 31, 2015. There were
2,528,499 stock options granted at exercise prices ranging from $0.68 to $1.35 with an aggregate fair
value of $1,737,087 during the year ended December 31, 2014. There were 2,450,000 stock
options granted at exercise prices ranging from $0.31 to $0.61 with an aggregate fair value of
$681,752 during the year ended December 31, 2013.
The fair value of options at the date of grant was estimated using the Black-Scholes option pricing
model. The Company took into consideration guidance under ASC 718, ―Compensation-Stock
Compensation‖ and Staff Accounting Bulletin No. 107 (―SAB 107‖) when reviewing and updating
assumptions. The expected volatility is based upon historical volatility of the Company‘s stock.
The expected term is based upon the simplified method as allowed under SAB 107.
The assumptions made in calculating the fair values of options are as follows:
Year Ended December 31,
2014
2013
2015
Black-Scholes assumptions
Expected dividend yield
Expected volatility
Risk free interest rate
Expected term (in years)
0 %
72-80 %
1.2-1.7 %
5-6 years
0 %
92-96 %
1.49-1.75 %
5 years
0 %
94-96 %
0.75-1.75 %
5 years
F-20
REXAHN PHARMACEUTICALS, INC.
Notes to Financial Statements
The following table summarizes share-based transactions:
Weighted
Average
Exercise
Price
Weighted Average
Remaining
Contractual Term
5.2 years $
Aggregate
Intrinsic
Value
842,300
Outstanding, January 1, 2015
Granted
Exercised
Expired
Cancelled
Number of
Options
11,400,806 $
4,201,316
(889,428)
(1,741,879)
(379,833)
Outstanding, December 31, 2015
Exercisable, December 31, 2015
12,590,982 $
6,702,550 $
0.93
0.69
0.80
1.19
0.88
0.83
0.91
6.8 years $
5.0 years $
26,500
16,500
The total intrinsic value of the options exercised was $99,895, $115,528 and $91,300 for the years
ended December 31, 2015, 2014 and 2013, respectively. The weighted average fair value of the
options granted was $0.47, $0.69 and $0.28 for the years ended December 31, 2015, 2014 and 2013,
respectively.
A summary of the Company‘s unvested options as of December 31, 2015 and changes during the
year ended December 31, 2015 is presented below:
Unvested at January 1, 2015
Granted
Vested
Cancelled
Unvested at December 31, 2015
2015
Number of Options
Weighted Average Fair
Value at Grant Date
3,233,499 $
4,201,316 $
(1,308,050) $
(238,333) $
5,888,432 $
0.60
0.47
0.58
0.57
0.51
As of December 31, 2015 there was $2,233,363 of total unrecognized compensation cost related to
unvested stock options, which is expected to be recognized over a weighted average vesting period
of 2.5 years.
F-21
REXAHN PHARMACEUTICALS, INC.
Notes to Financial Statements
12. Warrants
As of December 31, 2015, warrants to purchase 26,491,904 shares were outstanding, having exercise
prices ranging from $0.41 to $1.50 and expiration dates ranging from July 5, 2016 to May 13, 2021.
2015
2014
Number of
warrants
Weighted
average exercise
price
Balance, January 1
Issued during the period
Exercised during the period
Expired during the period
13,205,871 $
13,333,333 $
(47,300) $
- $
1.07
0.53
0.47
-
Number of
warrants
24,968,868 $
4,761,905 $
(12,058,871) $
(4,466,031) $
Balance, December 31
26,491,904 $
0.80
13,205,871 $
Weighted average
exercise price
0.86
1.28
0.52
1.59
1.07
At December 31, 2015 the weighted average remaining contractual life of the outstanding warrants
was 3.8 years.
The warrants issued to investors in the March 2011, December 2012, November 2015 and previous
offerings contain a provision for net cash settlement in the event that there is a fundamental
transaction (contractually defined as a merger, sale of substantially all assets, tender offer or share
exchange). If a fundamental transaction occurs in which the consideration issued consists
principally of cash or stock in a non-public company, then the warrant holder has the option to
receive cash, equal to the fair value of the remaining unexercised portion of the warrant. Due to
this contingent redemption provision, the warrants require liability classification in accordance with
ASC 480 and are recorded at fair value. The warrants issued to investors in the July 2013, October
2013 and January 2014 offerings contain a fundamental transaction provision, but the warrant
holders only have an option as to the type of consideration received if the holders of common stock
receive an option as to their consideration. In addition, the warrants issued in the March 2011,
December 2012, July 2013, October 2013, January 2014, November 2015 and previous offerings
contain a cashless exercise provision that is exercisable only in the event that a registration statement
is not effective. That provision may not be operative if an effective registration statement is not
available because an exemption under the U.S. securities laws may not be available to issue
unregistered shares. As a result, net cash settlement may be required, and the warrants require
liability classification.
ASC 820 provides requirements for disclosure of liabilities that are measured at fair value on a
recurring basis in periods subsequent to the initial recognition. Fair values for warrants are
determined using the Binomial Lattice (―Lattice‖) valuation technique. The Lattice model provides
for dynamic assumptions regarding volatility and risk-free interest rates within the total period to
maturity. Accordingly, within the contractual term, the Company provided multiple date intervals
over which multiple volatilities and risk free interest rates were used. These intervals allow the
Lattice model to project outcomes along specific paths that consider volatilities and risk free rates
that would be more likely in an early exercise scenario.
F-22
REXAHN PHARMACEUTICALS, INC.
Notes to Financial Statements
Significant assumptions are determined as follows:
Trading market values—Published trading market values;
Exercise price—Stated exercise price;
Term—Remaining contractual term of the warrant;
Volatility—Historical trading volatility for periods consistent with the remaining terms; and
Risk-free rate—Yields on zero coupon government securities with remaining terms consistent with
the remaining terms of the warrants.
Due to the fundamental transaction provision, which could provide for early redemption of the
warrants, the model also considered the probability the Company would enter into a fundamental
transaction during the remaining term of the warrant. Because the Company is not yet achieving
positive cash flow, management believes the probability of a fundamental transaction occurring over
the term of the warrant is unlikely and therefore estimates the probability of entering into a
fundamental transaction to be 5%. For valuation purposes, the Company also assumed that if such
a transaction did occur, it was more likely to occur towards the end of the term of the warrants.
The significant unobservable inputs used in the fair value measurement of the warrants include
management‘s estimate of the probability that a fundamental transaction may occur in the future.
Significant increases (decreases) in the probability of occurrence would result in a significantly
higher (lower) fair value measurement.
The following table summarizes the fair value of the warrants as of the respective balance sheet
dates:
Warrant Issuance:
March 31, 2011 financing:
Warrants to institutional investors
December 4, 2012 financing:
Warrants to institutional investors
Warrants to placement agent
July 26, 2013 financing:
Warrants to institutional investors
Warrants to placement agent
October 16, 2013 financing:
Warrants to institutional investors
Warrants to placement agent
January 21, 2014 financing:
Warrants to institutional investors
November 12, 2015 financing:
Warrants to institutional investors
Warrants to placement agent
Total:
Fair Value as of:
December 31, 2015 December 31, 2014
$
30 $
319,277
9,818
1,206
121,420
384
169,349
970
90,052
14,595
788,314
30,594
949,756
96,563
131,476
1,479,200
2,169,375
135,135
2,739,163 $
-
-
3,768,351
$
F-23
REXAHN PHARMACEUTICALS, INC.
Notes to Financial Statements
The following table summarizes the number of shares indexed to the warrants as of the respective
balance sheet dates:
Warrant Issuance
March 31, 2011 financing:
Warrants to institutional investors
December 4, 2012 financing:
Warrants to institutional investors
Warrants to placement agent
July 26, 2013 financing:
Warrants to institutional investors
Warrants to placement agent
October 16, 2013 financing:
Warrants to institutional investors
Warrants to placement agent
January 21, 2014 financing:
Warrants to institutional investors
November 12, 2015 financing:
Warrants to institutional investors
Warrants to placement agent
Total:
Number of Shares indexed as of:
December 31, 2015
December 31, 2014
3,333,333
3,333,333
174,300
40,000
2,000,000
124,032
2,317,309
407,692
221,600
40,000
2,000,000
124,032
2,317,309
407,692
4,761,905
4,761,905
12,500,000
833,333
26,491,904
-
-
13,205,871
The assumptions used in calculating the fair values of the warrants are as follows:
Trading market prices
Estimated future volatility
Dividend
Estimated future risk-free rate
Equivalent volatility
Equivalent risk-free rate
December 31, 2015 December 31, 2014
$
$
0.36
105 %
-
0.82-2.38 %
44-65 %
0.22-1.11 %
0.70
108 %
-
0.74-1.90 %
65-78 %
0.18-0.63 %
F-24
REXAHN PHARMACEUTICALS, INC.
Notes to Financial Statements
Changes in the fair value of the warrant liabilities, carried at fair value, as reported as ―unrealized
gain (loss) on fair value of warrants‖ in the statement of operations:
$
Exercised and Expired Warrants
March 31, 2011 financing:
Warrants to institutional investors
December 4, 2012 financing:
Warrants to institutional investors
Warrants to placement agent
July 26, 2013 financing:
Warrants to institutional investors
Warrants to placement agent
October 16, 2013 financing:
Warrants to institutional investors
Warrants to placement agent
January 21, 2014 financing:
Warrants to institutional investors
November 12, 2015 financing:
Warrants to institutional investors
Warrants to placement agent
Total:
$
Year Ended December 31,
2015
- $
2014
(277,769) $
2013
104,887
319,247
(7,917)
(5,027)
70,856
13,389
(4,120,103)
(514,881)
(1,598,195)
(75,062)
666,894
30,210
(1,272,731)
(234,877)
780,407
95,593
(940,100)
(23,956)
147,562
26,681
18,739
14,761
1,347,724
2,212,227
-
623,125
39,282
3,986,727 $
-
-
(5,180,107) $
-
-
(1,365,654)
F-25
REXAHN PHARMACEUTICALS, INC.
Notes to Financial Statements
13. Income Taxes
No provision for federal and state income taxes was required for the years ended December 31,
2015, 2014 and 2013 due to the Company‘s operating losses and increased deferred tax asset
valuation allowance. At December 31, 2015 and 2014, the Company had unused net operating loss
carry-forwards of approximately $98,954,000 and $81,619,000, respectively, which expire at various
dates through 2035. Some of this amount may be subject to annual limitations under certain
provisions of the Internal Revenue Code related to ―changes in ownership.‖
As of December 31, 2015 and 2014, the deferred tax assets related to the aforementioned
carry-forwards have been fully offset by valuation allowances, because significant utilization of such
amounts is not presently expected in the foreseeable future.
Deferred tax assets and valuation allowances consist of:
December 31,
2015
December 31,
2014
Net Operating Loss Carryforwards
Stock Compensation Expense
Book tax differences on assets and liabilities
Valuation Allowance
Net Deferred Tax Assets
$
$
38,592,000 $
1,891,000
380,000
(40,863,000)
31,831,000
2,221,000
416,000
(34,468,000)
- $
-
The Company files income tax returns in the U.S. federal and Maryland state jurisdictions. Tax
years for fiscal 2012 through 2015 are open and potentially subject to examination by the federal and
Maryland state taxing authorities.
F-26
REXAHN PHARMACEUTICALS, INC.
Notes to Financial Statements
14. Commitments and Contingencies
a) The Company has contracted with various vendors for research and development services, the
terms which require payments over the term of the agreements, usually ranging from two to 36
months. The costs to be incurred are estimated and are subject to revision. As of December 31,
2015, the total estimated cost to complete these agreements was approximately $9,380,000.
All of these agreements may be terminated by either party upon appropriate notice as stipulated
in the respective agreements.
b) On June 22, 2009, the Company entered into a License Agreement with Korea Research
Institute of Chemical Technology (―KRICT‖) to acquire the rights to all intellectual property
related to quinoxaline-piperazine derivatives that were synthesized under a Joint Research
Agreement. The initial license fee was $100,000, all of which was paid as of December 31,
2009. The agreement with KRICT calls for a one-time milestone payment of $1,000,000
within 30 days after the first achievement of marketing approval of the first commercial
product arising out of or in connection with the use of KRICT‘s intellectual properties. As of
December 31, 2015, the milestone has not occurred.
c) Office Space Lease
On June 29, 2009, the Company signed a five-year commercial lease agreement for 5,466
square feet of office space in Rockville, Maryland. Under the lease agreement, the Company
pays its allocable portion of real estate taxes and common area operating charges. Rent paid
under the Company‘s lease during the years ended December 31, 2015, 2014 and 2013 was
$202,529, $155,057 and $117,977, respectively.
On June 7, 2013, the Company entered into the first amendment to the lease agreement.
According to the terms of this amendment, the Company extended the lease term until June 30,
2019. The amended base rent was $100,210 and is subject to annual base bent increases the
remaining term of the lease.
On July 26, 2014 the Company entered into the second amendment to the lease agreement.
According to the terms of this amendment, the Company leased an additional 1,637 square feet
of office space, beginning on September 1, 2014 and ending on August 31, 2015. On May 6,
2015, the Company renewed the lease for this space for an additional year, beginning on
September 1, 2015 and ending on August 31, 2016.
Prior Laboratory Lease
On August 26, 2014 and June 24, 2013, the Company signed one-year renewals to use
laboratory space commencing on July 1, 2014 and 2013, respectively. The lease required
monthly rental payments of $4,554. Rent paid under the Company‘s lease during the years
ended December 31, 2015, 2014 and 2013 was $27,324, $54,648, and $54,648 respectively.
Current Laboratory Lease
On April 20, 2015, the Company signed a five-year lease agreement for 2,552 square feet of
laboratory space commencing on July 1, 2015 and ending on June 30, 2020. Under the lease
agreement, the Company pays its allocable portion of real estate taxes and common area
operating charges. Rent paid under this lease during the year ended December 31, 2015 was
$30,624.
F-27
REXAHN PHARMACEUTICALS, INC.
Notes to Financial Statements
Future rental payments over the next five years for all leases are as follows:
For the year ending December 31:
2016
2017
2018
2019
2020
269,733
260,217
233,923
152,955
34,468
Total
$
951,296
d) The Company has established a 401(k) plan for its employees. The Company has elected to
match 100% of the first 3% of an employee’s compensation plus 50% of an additional 2% of
the employee’s deferral. Expense related to this matching contribution aggregated to $121,519,
$91,241 and $78,487 for the years ended December 31, 2015, 2014 and 2013, respectively.
e)
In July 2013, the Company entered into an exclusive license agreement with the University of
Maryland, Baltimore for a novel drug delivery platform, Nano-Polymer Drug Conjugate
Systems. RX-21101 is the Company’s first drug candidate utilizing this platform. The
agreement requires the Company to make payments to the University of Maryland if
RX-21101 or any products from the licensed delivery platform achieve development
milestones. As of December 31, 2015, no development milestones have occurred.
f) In October 2013, the Company signed an exclusive license agreement with the Ohio State
Innovation Foundation, for a novel oligonucleotide drug delivery platform, Lipid-Coated
Albumin Nanoparticle. The agreement requires the Company to make payments to the Ohio
State Innovation Foundation or any products from the licensed delivery platform achieve
development milestones. As of December 31, 2015, no development milestones have
occurred.
F-28
REXAHN PHARMACEUTICALS, INC.
Notes to Financial Statements
15. Fair Value Measurements
ASC 820 defines fair value as the price that would be received to sell an asset or paid to transfer a
liability in an orderly transaction between market participants at the measurement date, not adjusted
for transaction costs. ASC 820 also establishes a fair value hierarchy that prioritizes the inputs to
valuation techniques used to measure fair value into three broad levels giving the highest priority to
quoted prices in active markets for identical assets or liabilities (Level 1) and the lowest priority to
unobservable inputs (Level 3).
The three levels are described below:
Level 1 Inputs
—
Unadjusted quoted prices in active markets for identical assets or
liabilities that are accessible by the Company;
Level 2 Inputs
—
Quoted prices in markets that are not active or financial instruments for
which all significant inputs are observable, either directly or indirectly;
Level 3 Inputs
—
Unobservable inputs for the asset or liability including significant
assumptions of the Company and other market participants.
The following tables present assets and liabilities that are measured at fair value on a recurring
basis and are categorized using the fair value hierarchy. There have been no changes in the
methodologies used at December 31, 2015 and 2014.
Total
Fair Value Measurements at December 31, 2015
Level 3
Level 2
Level 1
Assets:
Certificates of Deposit
Commercial Paper
Corporate Bonds
Total Assets:
Liabilities:
Warrant Liabilities
Assets:
Certificates of Deposit
Commercial Paper
Corporate Bonds
Total Assets:
Liabilities:
Warrant Liabilities
$
$
6,234,996 $
2,977,570
4,027,520
13,240,086 $
- $
-
-
- $
6,234,996 $
2,977,570
4,027,520
13,240,086 $
-
-
-
-
$
2,739,163
-
- $
2,739,163
Total
$
$
18,838,271 $
1,997,910
2,035,870
22,872,051 $
Fair Value Measurements at December 31, 2014
Level 3
Level 2
Level 1
- $
-
-
- $
18,838,271 $
1,997,910
2,035,870
22,872,051 $
-
-
-
-
$
3,768,351
-
- $
3,768,351
F-29
REXAHN PHARMACEUTICALS, INC.
Notes to Financial Statements
The fair value of the Company‘s Level 2 marketable securities is determined by using quoted prices
from independent pricing services that use market data for comparable securities in active or inactive
markets. A variety of data inputs, including benchmark yields, interest rates, known historical
trades and broker dealer quotes are using with pricing models to determine the quoted prices.
The fair value methodology for the warrant liabilities is disclosed in Note 12.
The carrying amounts reported in the financial statements for cash and cash equivalents (Level 1),
prepaid expenses, and other assets and accounts payable and accrued expenses approximate fair
value because of the short term maturity of these financial instruments.
The following table sets forth a reconciliation of changes in the years ended December 31, 2015 and
2014 in the fair value of the liabilities classified as Level 3 in the fair value hierarchy:
Balance at January 1, 2015
Additions
Unrealized gains, net
Transfers out of level 3
Balance at December 31, 2015
Balance at January 1, 2014
Additions
Unrealized losses, net
Transfers out of level 3
Balance at December 31, 2014
Warrant Liabilities
3,768,351
2,966,917
(3,986,727)
(9,378)
2,739,163
Warrant Liabilities
5,034,058
3,691,429
5,180,107
(10,137,243)
3,768,351
$
$
$
$
Additions consist of the fair value of warrant liabilities upon issuance. Transfers out of Level 3 for
warrant liabilities consist of warrant exercises, where the liability is converted to additional paid-in
capital upon exercise. The Company‘s policy is to recognize transfers in and transfers out as of the
actual date of the event or change in circumstance that caused the transfer.
F-30
REXAHN PHARMACEUTICALS, INC.
Notes to Financial Statements
16. Select Quarterly Data (Unaudited)
Revenues
Expenses
Loss from Operations
Other Income (Expense)
Net Loss
Net Loss per share, basic and diluted
March 31
$
- $
4,417,708
(4,417,708)
145,997
(4,271,711) $
(0.02) $
$
$
2015
For the Quarter Ended
June 30
- $
4,819,940
(4,819,940)
1,585,780
(3,234,160) $
(0.02) $
September 30 December 31
-
- $
4,367,233
(4,367,233)
1,515,078
(2,852,155)
(0.02)
4,658,555
(4,658,555)
632,025
(4,026,530) $
(0.02) $
Revenues
Expenses
Loss from Operations
Other Income (Expense)
2014
For the Quarter Ended
March 31
$
- $
June 30
September 30 December 31
-
- $
- $
2,766,094
3,508,936
3,141,706
3,852,493
(2,766,094)
(3,508,936)
(3,141,706)
(3,852,493)
(11,834,405)
3,703,400
1,236,258
1,642,375
Net (Loss) Income per share
$
(14,600,499) $
194,464 $
(1,905,448) $
(2,210,118)
Net (Loss) Income per share, basic and diluted $
(0.09) $
0.00 $
(0.01) $
(0.01)
17. Subsequent Events
Since December 31, 2015, the Company granted 3,337,090 stock options to officers, employees and
consultants.
On March 2, 2016, the Company issued 15,625,000 shares of its common stock and warrants exercisable
for up to 11,718,750 shares of its common stock in a registered direct offering for gross proceeds of $5
million. The shares and warrants were sold in units, consisting of one share of common stock and a
warrant to purchase 0.75 of a share of common stock, at an offering price of $0.32 per unit. Subject to
certain ownership limitations, the warrants are exercisable beginning six months after the closing date
until the five-year anniversary of the initial exercise date. The warrants have an exercise price of $0.42
per share. The Company also issued warrants to purchase 781,250 shares of the Company‘s common
stock, at an exercise price of $0.40 per share, to the placement agent in the offering and its designees in
reliance on the exemption from registration provided by Section 4(a)(2) under the Securities Act as
transactions not involving any public offering.
F-31
3.1
3.2
4.1
4.2
4.3
4.4
4.5
4.6
4.7
4.8
4.9
*10.1
*10.2
*10.3
*10.4
EXHIBIT INDEX
Amended and Restated Certificate of Incorporation, filed as Appendix G to the Company‘s
Definitive Proxy Statement on Schedule 14A (File No. 000-50590) dated April 29, 2005, is
incorporated herein by reference.
Amended and Restated Bylaws, as amended, through March 21, 2014, filed as exhibit 3.2 to
the Company‘s Annual Report on Form 10-K on March 21, 2014, is incorporated herein by
reference.
Specimen Certificate for the Company‘s Common Stock, par value $.0001 per share, filed
as Exhibit 4.3 to the Company‘s Registration Statement on Form S-8 (File No. 333-129294)
dated October 28, 2005, is incorporated herein by reference.
Form of Warrant for the Company‘s Common Stock Purchase Warrants, filed as Exhibit 4.1
to the Company‘s Current Report on Form 8-K filed on March 30, 2011, is incorporated
herein by reference.
Form of Warrant for the Company‘s Common Stock Purchase Warrants, filed as Exhibit 4.1
to the Company‘s Current Report on Form 8-K filed on November 30, 2012, is incorporated
herein by reference.
Form of Warrant for the Company‘s Common Stock Purchase Warrants, filed as Exhibit 4.2
to the Company‘s Current Report on Form 8-K filed on November 30, 2012, is incorporated
herein by reference.
Form of Warrant for the Company‘s Common Stock Purchase Warrants, filed as Exhibit 4.1
to the Company‘s Current Report on Form 8-K filed on July 24, 2013, is incorporated
herein by reference.
Form of Warrant for the Company‘s Common Stock Purchase Warrants, filed as Exhibit 4.1
to the Company‘s Current Report on Form 8-K filed on October 16, 2013, is incorporated
herein by reference.
Form of Warrant for the Company‘s Common Stock Purchase Warrants, filed as Exhibit 4.1
to the Company‘s Current Report on Form 8-K filed on January 15, 2014, is incorporated
herein by reference.
Form of Warrant for the Company‘s Common Stock Purchase Warrants, filed as Exhibit 4.1
to the Company‘s Current Report on Form 8-K filed on November 6, 2015, is incorporated
herein by reference.
Form of Warrant for the Company‘s Common Stock Purchase Warrants, filed as Exhibit 4.1
to the Company‘s Current Report on Form 8-K filed on February 26, 2016, is incorporated
herein by reference.
Rexahn Pharmaceuticals, Inc. Stock Option Plan, as amended, filed as Exhibit 4.4 to the
Company‘s Registration Statement on Form S-8 (File No. 333-129294) dated October 28,
2005, is incorporated herein by reference.
Form of Stock Option Grant Agreement for Employees, filed as Exhibit 4.5.1 to the
Company‘s Registration Statement on Form S-8 (File No. 333-129294) dated October 28,
2005, is incorporated herein by reference.
Form of Stock Option Grant Agreement for Non-Employee Directors and Consultants, filed
as Exhibit 4.5.2 to the Company‘s Registration Statement on Form S-8 (File No.
333-129294) dated October 28, 2005, is incorporated herein by reference.
Rexahn Pharmaceuticals, Inc. 2013 Stock Option Plan, filed as Exhibit 10.1 to the
Company‘s Registration Statement on Form S-8 (File No. 333-189240) dated June 11,
*10.5
*10.6
*10.7
*10.8
*10.9
10.10
10.11
10.12
10.13
10.14
10.15
10.16
10.17
2013, is incorporated herein by reference.
Form of Stock Option Grant Agreement under Rexahn Pharmaceuticals, Inc. 2013 Stock
Option Plan.
Employment Agreement, dated as of September 9, 2010, by and between Rexahn
Pharmaceuticals, Inc. and T. H. Jeong, filed as Exhibit 10.3 to the Company‘s Current
Report on Form 8-K filed on September 10, 2010, is incorporated herein by reference.
Employment Agreement, dated as of February 4, 2013, by and between Rexahn
Pharmaceuticals, Inc. and Peter Suzdak, filed as Exhibit 10.1 to the Company‘s Current
Report on Form 8-K filed on January 22, 2013, is incorporated herein by reference.
Employment Agreement, dated as of March 25, 2013, by and between Rexahn
Pharmaceuticals, Inc. and Chang H. Ahn, filed as Exhibit 10.1 to the Company‘s Current
Report on Form 8-K filed on March 29, 2013, is incorporated herein by reference.
Employment Agreement, dated as of February 2, 2015, by and between Rexahn
Pharmaceuticals, Inc. and Ely Benaim, M.D., filed as Exhibit 10.1 to the Company‘s
Current Report on Form 8-K filed on May 8, 2015, is incorporated herein by reference.
Lease Agreement, dated June 5, 2009, by and between Rexahn Pharmaceuticals, Inc. and
The Realty Associates Fund V, L.P., filed as Exhibit 10.4 to the Company‘s Quarterly
Report on Form 10-Q for the quarterly period ended June 30, 2009, is incorporated herein
by reference.
First Amendment to Lease Agreement, dated June 7, 2013, by and between Rexahn
Pharmaceuticals, Inc. and SG Plaza Holdings, LLC, filed as Exhibit 10.1 to the Company‘s
Quarterly Report on Form 10-Q for the quarterly period ended June 30, 2013, is
incorporated herein by reference.
Second Amendment to Lease Agreement, dated July 26, 2014, by and between Rexahn
Pharmaceuticals, Inc. and SG Plaza Holdings, LLC, filed as Exhibit 10.1 to the Company‘s
Quarterly Report on Form 10-Q for the quarterly period ended September 30, 2014, is
incorporated herein by reference.
Third Amendment to Lease Agreement, dated May 6, 2015, by and between Rexahn
Pharmaceuticals, Inc. and SG Plaza Holdings, LLC, filed as Exhibit 10.1 to the Company‘s
Quarterly Report on Form 10-Q for the quarterly period ended June 30, 2015, is
incorporated herein by reference.
Form of Securities Purchase Agreement, dated as of July 23, 2013, by and between Rexahn
Pharmaceuticals, Inc. and the purchasers identified on the signature pages thereto, filed as
Exhibit 10.1 to the Company‘s Current Report on Form 8-K filed on July 24, 2013, is
incorporated herein by reference.
Form of Securities Purchase Agreement, dated as of October 10, 2013, by and between
Rexahn Pharmaceuticals, Inc. and the purchasers identified on the signature pages thereto,
filed as Exhibit 10.1 to the Company‘s Current Report on Form 8-K filed on October 16,
2014, is incorporated herein by reference.
Form of Securities Purchase Agreement, dated as of January 15, 2014, by and between
Rexahn Pharmaceuticals, Inc. and the purchasers identified on the signature pages thereto,
filed as Exhibit 10.1 to the Company‘s Current Report on Form 8-K filed on January 21,
2014, is incorporated herein by reference.
Form of Securities Purchase Agreement, dated as of November 6, 2015, by and between
Rexahn Pharmaceuticals, Inc. and the purchasers identified on the signature pages thereto,
filed as Exhibit 10.1 to the Company‘s Current Report on Form 8-K filed on November 6,
10.18
10.19
12.1
23.1
24.1
31.1
31.2
32.1
32.2
2015, is incorporated herein by reference.
Form of Securities Purchase Agreement, dated as of February 26, 2016, by and between
Rexahn Pharmaceuticals, Inc. and the purchasers identified on the signature pages thereto,
filed as Exhibit 10.1 to the Company‘s Current Report on Form 8-K filed on February 26,
2015, is incorporated herein by reference.
At Market Issuance Sales Agreement, dated March 16, 2015, by and between Rexahn
Pharmaceuticals, Inc. and MLV & Co. LLC, filed as Exhibit 10.18 to the Company‘s
Annual Report on Form 10-K for the year ended December 31, 2014, is incorporated herein
by reference.
Statement Regarding the Computation of Ratio of Earnings to Combined Fixed Charges and
Preferred Stock Dividends
Consent of Baker Tilly Virchow Krause, LLP, independent registered public accounting
firm
Power of Attorney
Certification of Chief Executive Officer Pursuant to Rule 13a-14(a) or Rule 15d-14(a)
Certification of Chief Financial Officer Pursuant to Rule 13a-14(a) or Rule 15d-14(a)
Certification of Chief Executive Officer of Periodic Report Pursuant to 18 U.S.C.
Section 1350
Certification of Chief Financial Officer of Periodic Report Pursuant to 18 U.S.C.
Section 1350
101.INS
XBRL Instance Document
101.SCH
XBRL Taxonomy Extension Schema
101.CAL
XBRL Taxonomy Calculation Linkbase
101.DEF
XBRL Taxonomy Definition Linkbase
101.LAB
XBRL Taxonomy Label Linkbase
101.PRE
XBRL Taxonomy Presentation Linkbase
*Indicates management contract or compensatory plan or arrangement
CORPORATE INFORMATION
BOARD OF DIRECTORS
CORPORATE HEADQUARTERS
Peter Brandt, Chairman
Former President and Chief Executive
Officer, Noven Pharmaceuticals
Chang H. Ahn, Ph.D. Chairman Emeritus
Chief Scientist, Rexahn Pharmaceuticals
Charles Beever, Director
Former Vice President, PwC Strategy&
Mark Carthy, Director
Managing Partner, Orion Equity Partners
Kwang Soo Cheong, Ph.D. Director
Associate Professor, Johns Hopkins
University
Richard J. Rodgers, Director
Former Executive Vice President and Chief
Financial Officer, TESARO
Peter D. Suzdak, Ph.D. Director
Chief Executive Officer, Rexahn
Pharmaceuticals, Inc.
EXECUTIVE OFFICERS
Peter D. Suzdak, Ph.D.
Chief Executive Officer
Tae Heum (Ted) Jeong, D. Mgt.
Sr. Vice President, Chief Financial Officer
and Secretary
Ely Benaim, M.D.
Chief Medical Officer
Rexahn Pharmaceuticals, Inc.
15245 Shady Grove Road, Suite 455
Rockville, MD 20850
Phone: 240-268-5300
www.rexahn.com
TRANSFER AGENT
Olde Monmouth Stock Transfer Co., Inc.
Matthew J. Troster
200 Memorial Parkway
Atlantic Highlands, NJ 07716
Phone: 732-872-2727
LEGAL COUNSEL
Hogan Lovells US LLP
100 International Drive, Suite 2000
Baltimore, MD 21202
INDEPENDENT REGISTERED
PUBLIC ACCOUNTING FIRM
Baker Tilly Virchow Krause, LLP
2609 Keiser Blvd
Wyomissing, PA 19610-3338
SECURITIES INFORMATION
TRADING MARKET: NYSE MKT
SYMBOL: RNN
FOR INVESTOR RELATIONS INQUIRIES OR
TO REQUEST ADDITIONAL COPIES OF THIS
ANNUAL REPORT, CONTACT:
LifeSci Advisors, LLC
Matthew P. Duffy
(212)-915-0685
matthew@lifesciadvisors.com
Stockholders may obtain a copy of any
exhibit to our Form 10-K free of charge by
writing to the company at our corporate
headquarters address above.