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UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM 10-K
ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF
THE SECURITIES EXCHANGE ACT OF 1934
For the fiscal year ended December 31, 2020
Commission file number 001-37581
ACLARIS THERAPEUTICS, INC.
Incorporated under the Laws of the
State of Delaware
I.R.S. Employer Identification No.
46-0571712
640 Lee Road, Suite 200
Wayne, PA 19087
(484) 324-7933
Securities registered pursuant to Section 12(b) of the Act:
Title of Each Class:
Common Stock, $0.00001 par value
Trading Symbol(s)
ACRS
Name of Each Exchange on which Registered
The Nasdaq Stock Market, LLC
Securities registered pursuant to Section 12(g) of the Act:
None
Indicate by check mark if the registrant is a well-known seasoned issuer, as defined in Rule 405 of the Securities Act. Yes ⌧ No ☐
Indicate by check mark if the registrant is not required to file reports pursuant to Section 13 or 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 every Interactive Data File required to be submitted pursuant to Rule 405 of Regulation S-T
(§232.405 of this chapter) during the preceding 12 months (or for such shorter period that the registrant was required to submit such files). Yes ⌧ No ☐
Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, a smaller reporting company or emerging growth
company. See definitions of “large accelerated filer,” “accelerated filer”, “smaller reporting company” and “emerging growth company” in Rule 12b-2 of the Exchange Act.
Large accelerated filer ☐
Accelerated filer ☐
Non-accelerated filer ⌧
Smaller reporting company ⌧
Emerging growth company ☐
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised
financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐
Indicate by check mark whether the registrant has filed a report on and attestation to its management’s assessment of the effectiveness of its internal control over financial
reporting under Section 404(b) of the Sarbanes-Oxley Act (15 U.S.C. 7262(b)) by the registered public accounting firm that prepared or issued its audit report. ☐
Indicate by check mark whether the registrant is a shell company (as defined in Rule 12b-2 of the Exchange Act). Yes ☐ No ⌧
As of June 30, 2020, the last business day of the registrant’s last completed second quarter, the aggregate market value of the registrant’s common stock held by non-
affiliates of the registrant was approximately $65.5 million based on the closing price of the registrant’s common stock, as reported by the Nasdaq Global Select Market, on such
date.
As of January 31, 2021, 51,804,258 shares of common stock, $0.00001 par value, were outstanding.
DOCUMENTS INCORPORATED BY REFERENCE
Portions of the Company's definitive proxy statement, to be filed pursuant to Regulation 14A under the Securities Exchange Act of 1934, for its 2021 Annual Meeting of
Stockholders are incorporated by reference in Part III of this Form 10-K.
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SPECIAL NOTE REGARDING FORWARD-LOOKING STATEMENTS
This Annual Report on Form 10-K, or this Annual Report, contains forward-looking statements within the
meaning of Section 27A of the Securities Act of 1933, as amended, or the Securities Act, and Section 21E of the Securities
Exchange Act of 1934, as amended, or the Exchange Act, that involve substantial risks and uncertainties. The forward-
looking statements are contained principally in Part I, Item 1. “Business,” Part I, Item 1A. “Risk Factors,” and Part II,
Item 7. “Management’s Discussion and Analysis of Financial Condition and Results of Operations,” but are also contained
elsewhere in this Annual Report. In some cases, you can identify forward-looking statements by the words “may,” “might,”
“can,” “will,” “to be,” “could,” “would,” “should,” “expect,” “intend,” “plan,” “objective,” “anticipate,” “believe,”
“estimate,” “predict,” “project,” “potential,” “likely,” “continue” and “ongoing,” or the negative of these terms, or other
comparable terminology intended to identify statements about the future. These statements involve known and unknown
risks, uncertainties and other factors that may cause our actual results, levels of activity, performance or achievements to be
materially different from the information expressed or implied by these forward-looking statements. Although we believe
that we have a reasonable basis for each forward-looking statement contained in this Annual Report, we caution you that
these statements are based on a combination of facts and factors currently known by us and our expectations of the future,
about which we cannot be certain. Forward-looking statements include statements about:
● our plans to develop our drug candidates;
● the timing of our planned clinical trials of our drug candidates and the reporting of the results from these trials;
● the clinical utility of our drug candidates;
● our plans and expectations related to manufacturing capabilities and strategy;
● our expectations regarding coverage and reimbursement of our drug candidates, if approved;
● the timing of our regulatory filings and approvals for our drug candidates;
● our intellectual property position;
● our plans to pursue strategic alternatives, including identifying and consummating transactions with third-party
partners, to further develop, obtain marketing approval for and/or commercialize our drug candidates, and earn
revenue from such arrangements;
● our expectations regarding competition;
● our expectations regarding our continued reliance on third parties;
● the impacts of the COVID-19 pandemic on our business;
● our expectations regarding our use of capital; and
● our estimates regarding future revenue, expenses and needs for additional financing.
You should refer to Part I, Item 1A. “Risk Factors” in this Annual Report for a discussion of important factors that
may cause our actual results to differ materially from those expressed or implied by our forward-looking statements. As a
result of these factors, we cannot assure you that the forward-looking statements in this Annual Report will prove to be
accurate, and you should not place undue reliance on these forward-looking statements. Furthermore, if our forward-
looking statements prove to be inaccurate, the inaccuracy may be material. In light of the significant uncertainties in these
forward-looking statements, you should not regard these statements as a representation or warranty by us or any other
person that we will achieve our objectives and plans in any specified time frame, or at all. The forward-looking statements
in this Annual Report represent our views as of the date of this Annual Report. We anticipate that subsequent events and
developments may cause our views to change. However, while we may elect to update these forward-looking statements at
some point in the future, we undertake no obligation to publicly update any forward-looking statements, whether as a result
of new information, future events or otherwise, except as required by law. You should, therefore, not rely on these forward-
looking statements as representing our views as of any date subsequent to the date of this Annual Report.
All brand names or trademarks appearing in this Annual Report, including KINect, ESKATA and RHOFADE are
the property of their respective owners. Unless the context requires otherwise, references in this report to “Aclaris,” the
“Company,” “we,” “us,” and “our” refer to Aclaris Therapeutics, Inc. and its subsidiaries.
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PART I
Item 1. Business
Item 1A. Risk Factors
Item 1B. Unresolved Staff Comments
Item 2. Properties
Item 3. Legal Matters
Item 4. Mine Safety Disclosures
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PART II
Item 5. Market for Registrant’s Common Equity, Related Stockholder Matters and Issuer Purchases of Equity
Securities
Item 6. Selected Consolidated Financial Data
Item 7. Management’s Discussion and Analysis of Financial Condition and Results of Operations
Item 7A. Quantitative and Qualitative Disclosure About Market Risk
Item 8. Financial Statements and Supplementary Data
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 Accountant Fees and Services
PART IV
Item 15. Exhibits, Financial Statement Schedules
Item 16. Form 10-K Summary
Signatures
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Item 1. Business
Overview
PART I
We are a clinical-stage biopharmaceutical company focused on developing novel drug candidates for immuno-
inflammatory diseases. In addition to developing our novel drug candidates, we are pursuing strategic alternatives,
including identifying and consummating transactions with third-party partners, to further develop, obtain marketing
approval for and/or commercialize our novel drug candidates.
Our Drug Candidates Currently in Development
Our pipeline of drug candidates that we are currently developing is summarized in the table below. These
investigational drugs were developed internally utilizing our proprietary KINect drug discovery platform.
Drug Candidate /
Program
Target
Route of
Administration
Indication
Development
Phase
Immuno-Inflammatory Diseases
ATI-450
MK2 inhibitor
oral
ATI-1777
ATI-2138
Undisclosed- Gut
Restricted
Program
Undisclosed- Gut
Restricted
Program
“soft” JAK 1/3
inhibitor
ITK/TXK/JAK3
inhibitor
JAK1/JAK3
inhibitor
ITK/TXK/JAK3
inhibitor
topical
oral
oral
oral
rheumatoid arthritis
(moderate to severe)
additional immuno-inflammatory
diseases
COVID-19**
atopic dermatitis
(moderate to severe)
psoriasis
inflammatory bowel disease
Phase 2
Phase 2*
Phase 2
Phase 2
Pre-IND
inflammatory bowel disease
Discovery
inflammatory bowel disease
Discovery
* We are currently evaluating additional potential immuno-inflammatory indications which we expect to progress
directly into Phase 2.
** This is an investigator-initiated trial sponsored by the University of Kansas Medical Center.
MK2 Inhibitors, JAK Inhibitors and ITK Inhibitors as Potential Treatments for Immuno-Inflammatory
Diseases
In 2017, we acquired Confluence Life Sciences, Inc. (now known as Aclaris Life Sciences, Inc.), or
Confluence. The acquisition of Confluence added small molecule drug discovery and preclinical development capabilities,
including KINect, a proprietary drug discovery platform. This allowed us to bring early-stage research and development
activities in-house that we previously outsourced to third parties. We intend to leverage these capabilities and KINect to
identify potential drug candidates that we may develop independently or in collaboration with third parties. As part of the
Confluence acquisition we also acquired our investigational drug candidates, ATI-450, an inhibitor of the mitogen-
activated protein kinase-activated protein kinase 2, or MK2, signaling pathway, and ATI-1777, a topical “soft” Janus
kinase, or JAK, inhibitor, as well as several other preclinical drug candidates including inhibitors of interleukin-2-
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inducible T cell kinase, or ITK. We also earn revenue from Confluence’s provision of contract research services to third
parties.
ATI-450, an Investigational Oral MK2 Inhibitor
We submitted an Investigational New Drug Application, or IND, in April 2019 for ATI-450, an investigational
oral, novel, small molecule selective MK2 inhibitor compound, for the treatment of rheumatoid arthritis, which was
allowed by the U.S. Food and Drug Administration, or FDA, in May 2019. MK2 is a key regulator of pro-inflammatory
mediators including TNFα, IL1β, IL6, IL8 and other essential pathogenic signals in chronic immuno-inflammatory
diseases, as well as in oncology. As an oral drug candidate, we are developing ATI-450 as a potential alternative to
injectable anti-TNF/IL1/IL6 biologics and JAK inhibitors for treating certain immuno-inflammatory diseases.
We initiated a Phase 1 single (at 10mg, 30mg, 50mg and 100mg doses) and multiple ascending (at 10mg, 30mg
and 50mg doses) dose clinical trial evaluating ATI-450 in 77 healthy subjects in August 2019 (ATI-450-PKPD-101). Final
data from this trial demonstrated that ATI-450 resulted in marked inhibition of TNFα, IL1β, IL8 and IL6. We also observed
that ATI-450 had dose-proportional pharmacokinetics with a terminal half-life of 9-12 hours in the multiple ascending dose
cohort, and had no meaningful food effect or drug-drug interaction with methotrexate. ATI-450 was generally well-
tolerated at all doses tested in the trial. The most common adverse events (reported by 2 or more subjects who received
ATI-450) were dizziness, headache, upper respiratory tract infection, constipation, abdominal pain and nausea.
ATI-450 was also evaluated at 80mg and 120mg doses twice daily in a second Phase 1 clinical trial in healthy
subjects (ATI-450-PKPD-102). Preliminary topline data from this trial showed that no dose-limiting toxicity was
observed. Ex vivo analysis of blood samples from this Phase 1 trial also showed that increased cytokine inhibition was
achieved with these higher doses of ATI-450. No serious adverse events were reported and all adverse events were mild to
moderate. The most common adverse events (reported by 2 or more subjects who received ATI-450) were headache,
dizziness, nausea, parasthesia and, in the post-dosing safety follow-up phase of the trial, dry skin. These adverse events
were all mild in severity. A final analysis of this trial is underway.
Moderate to Severe Rheumatoid Arthritis
Following the completion of the first Phase 1 clinical trial, in March 2020 we initiated a 12-week, Phase 2a,
multicenter, randomized, investigator and patient-blind, sponsor-unblinded, parallel group, placebo-controlled clinical trial
to investigate the safety, tolerability, pharmacokinetics and pharmacodynamics of ATI-450 in subjects with moderate to
severe rheumatoid arthritis (ATI-450-RA-201). In the trial, 19 subjects were randomized in a 3:1 ratio (seventeen subjects
[15 in the treatment arm and two in the placebo arm] completed treatment) and received either ATI-450 at 50 mg twice
daily or placebo, in combination with methotrexate, for 12 weeks. Preliminary topline data from this trial showed that ATI-
450 demonstrated durable clinical activity, as defined by a marked and sustained reduction in DAS28-CRP and
improvement of ACR20/50/70 responses over 12 weeks. ATI-450 was generally well tolerated. All adverse events were
mild to moderate. The most common adverse events (each reported in 2 subjects) were urinary tract infection, or UTI,
elevated lipids and ventricular extrasystoles, all of which were determined to be unrelated to treatment except for one UTI.
Two subjects withdrew from the trial, one in the treatment arm and one in the placebo arm. The subject in the treatment
arm withdrew due to palpitations, which were unrelated to the trial medication, and an elevated creatine phosphokinase, or
CPK, which was determined by the site investigator to be treatment-related. The subject in the placebo arm withdrew as a
result of prohibited medication needed to treat muscle strain. There was one non-treatment-related serious adverse event
(COVID-19) reported in the four-week safety follow-up phase of the trial in a subject who was no longer receiving
treatment.
An interim analysis (11 treatment, two placebo) of ex vivo stimulated cytokines from blood samples taken from
the treatment arm showed a marked and durable inhibition of TNFα, IL1β, IL6, and IL8 over the 12 week dosing period.
Similarly, analysis of endogenous inflammation biomarkers also demonstrated a marked and sustained inhibition of median
concentrations of hsCRP, TNFα, IL6, IL8 and MIP1β in the treatment arm over the 12-week period.
We plan to submit for publication a full analysis of the Phase 2a data in a peer-reviewed scientific journal which
will include data from other secondary and exploratory endpoints evaluated in the trial, including the four-week safety
follow-up data and a full analysis of MRI, pharmacodynamic and pharmacokinetic data. Based on the results observed in
the Phase 2a trial, we intend to progress ATI-450 into a Phase 2b trial in moderate to severe rheumatoid arthritis in the
second half of 2021.
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Cryopyrin-associated Periodic Syndrome
In November 2020, we initiated a Phase 2a multicenter, open-label, single-arm clinical trial to investigate the
safety, tolerability, efficacy and pharmacodynamics of ATI-450 for the maintenance of remission in subjects with
cryopyrin-associated periodic syndrome, or CAPS, previously managed with anti-IL1 therapy (ATI-450-CAPS-201). Due
to the COVID-19 pandemic, subject enrollment in this trial was paused. As a result of the ongoing pandemic and given the
positive preliminary topline data from the ATI-450-RA-201 trial, we have decided to focus our efforts and resources on
other immuno-inflammatory diseases.
COVID-19
We also supported an investigator-initiated Phase 2a, randomized, double-blind, placebo-controlled clinical trial to
investigate the safety and efficacy of ATI-450, when used in addition to standard of care therapy, as a potential treatment
for cytokine release syndrome in hospitalized patients with COVID-19. The primary endpoint in this trial is the proportion
of subjects who are free from respiratory failure by day 14. We provided funding and clinical drug supply to the University
of Kansas Medical Center, the sponsor of the trial. The trial included 20 subjects and is completed. We expect data to be
available in the first half of 2021.
ATI-1777, an Investigational Topical “Soft” JAK 1/3 Inhibitor
In June 2020, we submitted an IND for ATI-1777, an investigational topical “soft” JAK 1/3 inhibitor compound,
for the treatment of moderate to severe atopic dermatitis. “Soft” JAK inhibitors are designed to be topically applied and
active in the skin, but rapidly metabolized and inactivated when they enter the bloodstream, which may result in low
systemic exposure.
In October 2020, we initiated a Phase 2a, multicenter, randomized, double-blind, vehicle-controlled, parallel-
group clinical trial to determine the efficacy, safety, tolerability and pharmacokinetics of ATI-1777 in subjects with
moderate to severe atopic dermatitis (ATI-1777-AD-201). We expect data to be available mid-year 2021.
ATI-2138, an Investigational ITJ Inhibitor
We are also developing ATI-2138, an investigational oral ITK/TXK/JAK3, or ITJ, inhibitor compound, as a
potential treatment for psoriasis and/or inflammatory bowel disease, which are both T-cell mediated autoimmune diseases.
The ITJ compound interrupts T cell signaling through the combined inhibition of ITK/TXK/JAK3 pathways in
lymphocytes. We expect to file an IND for ATI-2138 in the second half of 2021.
Our Other Drug Candidates
We continue to seek third-party partners for our dermatology investigational drug candidate A-101 45% Topical
Solution as a potential treatment for common warts (verruca vulgaris).
Manufacturing and Supply
We do not have any manufacturing facilities. We rely on third parties for the manufacture of preclinical and
clinical supplies for our drug candidates.
Competition
The pharmaceutical industry is characterized by rapidly advancing technologies, intense competition and a strong
emphasis on proprietary drugs. While we believe that our knowledge, experience and scientific resources provide us with
competitive advantages, we face potential competition from many different sources, including major pharmaceutical,
biotechnology and specialty pharmaceutical companies, academic institutions and governmental agencies and public and
private research institutions. Our drug candidates, if approved, will compete with existing treatments and new treatments
that may become available in the future.
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With respect to ATI-450 as a potential treatment for moderate to severe rheumatoid arthritis, there are several
different types of therapies in the rheumatoid arthritis market. Medications for the treatment of rheumatoid arthritis
currently fall into two categories: drugs that ease symptoms such as nonsteroidal anti-inflammatory drugs and drugs that
slow disease activity. Drugs that slow disease activity include corticosteroids and disease-modifying anti-rheumatic drugs,
or DMARDs. Disease-modifying drugs include conventional DMARDs such as methotrexate, sulfasalazine, leflunomide,
hydroxychloroquine, biologic DMARDs (monoclonal antibodies which inhibit targets such as TNF, IL1, IL6 and
costimulatory signaling mechanisms), and targeted synthetic DMARDs such as JAK inhibitors. These types of drugs are
produced and sold by large pharmaceutical companies, including AbbVie, Amgen, Bristol Myers Squibb, Eli Lilly, Johnson
& Johnson, Merck, Pfizer, and Roche, among others. In addition, we are aware of a number of companies developing and
conducting clinical trials for investigational drug candidates, including biosimilars, that, if approved, could compete with
ATI-450, if approved, for the treatment of rheumatoid arthritis.
With respect to ATI-1777 as a potential treatment for moderate to severe atopic dermatitis, there are several
different types of therapies in the atopic dermatitis market, such as biologics, oral and topical corticosteroids, injectable and
oral methotrexate products, oral and topical calcineurin inhibitors, oral mycophenolate products, other JAK inhibitors,
other oral antibiotics and antihistamines and phototherapy. There are also several prescription, non-prescription and over-
the-counter, or OTC, topical products, including PDE4 inhibitors, utilized to treat atopic dermatitis. These types of drugs
are produced and sold by large pharmaceutical companies, including Sanofi and Regeneron Pharmaceuticals, Inc., and
Pfizer. In addition, we are aware of a number of companies including large pharmaceutical companies, such as AbbVie, Eli
Lilly, Novartis, Incyte, Pfizer and LEO Pharma A/S developing and conducting clinical trials for investigational drug
candidates, that, if approved, could compete with ATI-1777, if approved, for the treatment of atopic dermatitis.
The commercial opportunity for our drug candidates, if approved, could be reduced or eliminated if our
competitors develop and commercialize drugs that are safer, more effective, have fewer or less severe side effects, are more
convenient or are less expensive than any drug we may develop. Our competitors also may obtain FDA or other regulatory
approval for their drug candidates more rapidly than our potential third-party partners may obtain approval for our drug
candidates, which could result in our competitors establishing a strong market position before our drug candidates are able
to enter the market.
Many of the companies against which we are competing, or against which we may compete in the future, have
significantly greater financial resources and expertise in research and development, manufacturing, and preclinical and
clinical development than we do. Mergers and acquisitions in the pharmaceutical and biotechnology industries may result
in even more resources being concentrated among a smaller number of our competitors. Smaller or early-stage companies
may also prove to be significant competitors, particularly through collaborative arrangements with large and established
companies. These competitors also compete with us in recruiting and retaining qualified scientific and management
personnel and establishing clinical trial sites and subject registration for clinical trials, as well as in acquiring technologies
complementary to, or that may be necessary for, our development programs.
Intellectual Property
Our success depends in large part upon our ability to obtain and maintain proprietary protection for our drug
candidates and to operate without infringing the proprietary rights of others. We seek to avoid the latter by monitoring
patents and publications that may affect our business, and to the extent we identify such developments, evaluating and
taking appropriate courses of action. Our policy is to protect our proprietary position by, among other methods, filing
patent applications on inventions that are important to the development and conduct of our business with the U.S. Patent
and Trademark Office, or USPTO, and its foreign counterparts.
With respect to our MK2 signaling pathway inhibitor development program, we own numerous issued patents and
pending applications to novel MK2 pathway inhibitors, including our lead candidate ATI-450, and various methods of use
that expire, or would expire, between 2031 and 2041, subject to any applicable patent term adjustment or extension that
may be available in a particular country. For example, we own two U.S. patents and pending applications in the European
Union and other foreign countries directed to ATI-450 and analogs thereof and certain methods of using the same. The
U.S. patents expire in 2034 and any claims that may issue from the pending applications expire in 2034, subject to any
applicable adjustment or extension. We also own a Patent Cooperation Treaty, or PCT, application directed to deuterated
forms of ATI-450 and methods of use, which, if issued, would expire in 2040, subject to any applicable adjustment or
extension. Further, we own numerous provisional applications directed to certain methods of using ATI-
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450, methods of manufacturing ATI-450 and crystal forms of ATI-450, which, if issued, would each expire in 2041, subject
to any applicable adjustment or extension.
With respect to our “soft” JAK inhibitor development program, we own one issued U.S. patent and numerous
pending applications in the U.S. and foreign countries to novel “soft” JAK inhibitors and various methods of use that
expire, or would expire, between 2038 and 2041, subject to any applicable patent term adjustment or extension that may be
available in a particular country. For example, we own one allowed U.S. application and pending applications in the
European Union and other foreign countries directed to various novel inhibitors of JAK1 and/or JAK3, including ATI-
1777, and methods of using the same, which, if issued, would expire in 2038, subject to any applicable adjustment or
extension. We also own two provisional applications directed to topical formulations and crystal forms of ATI-1777, which,
if issued, would expire in 2041, subject to any applicable adjustment or extension.
With respect to our ITK inhibitor development program, we own numerous issued U.S. patents and pending
applications in the U.S. and foreign countries directed to novel inhibitors of ITK and methods of use that expire, or would
expire, between 2035 and 2039, subject to any applicable patent term adjustment or extension that may be available in a
particular country. We also own pending U.S., European Union and other foreign country applications directed to ATI-2138
and analogs thereof and methods of using the same, which, if issued, would expire in 2039, subject to any applicable
adjustment or extension.
Patents extend for varying periods according to the date of patent filing or grant and the legal term of patents in
various countries where patent protection is obtained. The actual protection afforded by a patent, which can vary from
country to country, depends on the type of patent, the scope of its coverage and the availability of legal remedies in the
country. In most countries in which we file, the patent term is 20 years from the earliest date of filing a non-provisional
patent application. In the United States, a patent term may be shortened if a patent is terminally disclaimed over another
patent or as a result of delays in patent prosecution by the patentee, and a patent’s term may be lengthened by patent term
adjustment, which compensates a patentee for administrative delays by the USPTO in granting a patent or by patent term
extension, which compensates a patentee for delays at the FDA. The patent term of a European patent is 20 years from its
filing date; however, unlike in the United States, the European patent does not grant patent term adjustments. The European
Union does have a compensation program similar to patent term extension called supplementary patent certificate that
would effectively extend patent protection for up to five years.
We also use other forms of protection, such as trademark, copyright, and trade secret protection, to protect our
intellectual property, particularly where we do not believe patent protection is appropriate or obtainable. We aim to take
advantage of all of the intellectual property rights that are available to us and believe that this comprehensive approach will
provide us with proprietary positions for our drug candidates, where available.
We also protect our proprietary information by requiring our employees, consultants, contractors and other
advisors to execute nondisclosure and assignment of invention agreements upon commencement of their respective
employment or engagement. Agreements with our employees also prevent them from bringing the proprietary rights of
third parties to us. In addition, we also require confidentiality or service agreements from third parties that receive our
confidential information or materials.
Acquisition and License Agreements
Agreement and Plan of Merger with Confluence
In August 2017, we entered into an Agreement and Plan of Merger, or the Confluence Agreement, with
Confluence, Aclaris Life Sciences, Inc., our wholly-owned subsidiary, or Merger Sub, and Fortis Advisors LLC, as
representative of the former equity holders of Confluence. Pursuant to the terms of the Confluence Agreement, the Merger
Sub merged with and into Confluence, with Confluence surviving as our wholly-owned subsidiary, resulting in our
acquisition of 100% of the outstanding shares of Confluence. We paid closing consideration of $10.3 million in cash and
issued 349,527 shares of our common stock with a fair value of $9.7 million to the former Confluence equity holders.
In November 2018, a development milestone specified in the Confluence Agreement was achieved, as a result of
which we paid the former Confluence equity holders $2.5 million in cash and issued 253,208 shares of our common stock
with a fair value of $2.2 million. Under the Confluence Agreement, we also agreed to pay the former Confluence equity
holders aggregate remaining contingent consideration of up to $75.0 million based upon the achievement of specified
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regulatory and commercial milestones set forth in the Confluence Agreement. In addition, we have agreed to pay the
former Confluence equity holders future royalty payments calculated as a low single-digit percentage of annual net sales,
subject to specified reductions, limitations and other adjustments, until the date that all of the patent rights for that product
have expired, as determined on a country-by-country and product-by-product basis or, in specified circumstances, ten years
from the first commercial sale of such product. In addition to the payments described above, if we sell, license or transfer
any of the intellectual property acquired from Confluence pursuant to the Confluence Agreement to a third party, we will
be obligated to pay the former Confluence equity holders a portion of any consideration received from such sale, license or
transfer in specified circumstances.
Government Regulation and Product Approval
Governmental authorities in the United States, at the federal, state and local level, and analogous authorities in
other countries extensively regulate, among other things, the research, development, testing, manufacture, safety
surveillance, efficacy, quality control, labeling, packaging, distribution, record keeping, promotion, storage, advertising,
distribution, marketing, sale, export and import, and the reporting of safety and other post-market information of products
such as the ones we are developing. A drug candidate must be approved by the FDA before it may be legally promoted in
the United States and by comparable foreign regulatory authorities before marketing in other jurisdictions. The process of
obtaining regulatory approvals and the subsequent compliance with applicable federal, state, local and foreign statutes and
regulations require the expenditure of substantial time and resources. Failure to comply with the applicable U.S.
requirements at any time during the product development process, approval process or after approval may subject an
applicant and/or sponsor to a variety of administrative or judicial sanctions, including refusal by regulatory authorities to
approve applications, withdrawal of an approval, imposition of a clinical hold, import/export delays, issuance of warning
letters and untitled letters, product recalls, product seizures, total or partial suspension of production or distribution,
injunctions, fines, refusals of government contracts, restitution, disgorgement of profits, or civil or criminal investigations
and penalties brought by FDA and the Department of Justice or other governmental entities.
United States Government Regulation
NDA Approval Processes
In the United States, the FDA regulates drug and medical device products under the Federal Food, Drug, and
Cosmetic Act, or FDCA, and its implementing regulations. The FDA’s Center for Drug Evaluation and Research has
primary jurisdiction over the premarket development, review and approval of our drug candidates. Accordingly, we are
investigating our drug candidates pursuant to IND applications and expect that any potential third-party partners that we
may consummate a transaction with would seek approval through the New Drug Application, or NDA, pathway.
An applicant seeking approval to market and distribute a new drug product in the United States must typically
undertake the following:
● completion of preclinical laboratory tests, animal studies and formulation studies in compliance with the FDA’s
good laboratory practice regulations;
● submission to the FDA of an IND which must take effect before clinical trials may begin;
● approval by an independent institutional review board, or IRB, representing each clinical site before clinical
testing may be initiated at the clinical site;
● performance of adequate and well-controlled clinical trials in accordance with good clinical practice, or GCP,
regulations to establish the safety and efficacy of the proposed drug product for each indication;
● preparation and submission to the FDA of an NDA;
● review of the NDA by an FDA advisory committee, if applicable;
● satisfactory completion of one or more FDA inspections of the manufacturing facility or facilities at which the
product or its components are produced to assess compliance with current good manufacturing practices, or
cGMP, and regulations to assure that the facilities, methods and controls are adequate to preserve the product’s
identity, strength, quality and purity;
● payment of user fees and securing FDA approval of the NDA; and
● compliance with any post-approval requirements, including potential requirements for a risk evaluation and
mitigation strategy and post-approval studies required by the FDA.
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Once a drug candidate is identified for development, it enters the preclinical or nonclinical testing stage.
Preclinical studies include laboratory evaluations of product chemistry, pharmacology, toxicity and formulation. An IND
sponsor must submit the results of the preclinical studies, together with manufacturing information and analytical data, to
the FDA as part of the IND. Some preclinical studies may continue even after the IND is submitted. In addition to
including the results of the preclinical studies, the IND will also include a protocol detailing, among other things, the
objectives of the clinical trial, the parameters to be used in monitoring safety and the effectiveness criteria to be evaluated
if the first phase lends itself to an efficacy determination. The IND automatically becomes effective 30 days after receipt by
the FDA, unless the FDA, within the 30-day time period, places the IND on clinical hold. In such a case, the IND sponsor
and the FDA must resolve any outstanding concerns before clinical trials can begin. A clinical hold may occur at any time
during the life of an IND, and may affect one or more specific clinical trials or all clinical trials conducted under the IND.
All clinical trials must be conducted under the supervision of one or more qualified investigators in accordance
with current GCP regulations. They must be conducted under protocols detailing the objectives of the trial, dosing
procedures, research subject selection and exclusion criteria, and the safety and effectiveness criteria to be evaluated. Each
protocol must be submitted to the FDA as part of the IND, and progress reports detailing the status of the clinical trials
must be submitted to the FDA annually. Sponsors also must timely report to the FDA serious and unexpected adverse
reactions, any clinically important increase in the rate of a serious suspected adverse reaction over that listed in the protocol
or investigator brochure, and any findings from other studies or animal or in vitro testing that suggest a significant risk in
humans exposed to the drug. An IRB at each institution participating in the clinical trial must review and approve the
protocol before the clinical trial commences at that institution and must also approve the information regarding the trial and
the consent form that must be provided to each research subject or the subject’s legal representative, monitor the study until
completed and otherwise comply with IRB regulations.
Clinical trials are typically conducted in three sequential phases that may overlap or be combined:
● Phase 1. The drug is initially introduced into healthy human subjects and tested for safety, dosage tolerance,
absorption, metabolism, distribution and elimination. In the case of some products for severe or life-threatening
diseases, such as cancer, and especially when the product may be inherently too toxic to ethically administer to
healthy volunteers, the initial human testing is often conducted in patients who already have the condition.
● Phase 2. Clinical trials are performed on a limited patient population intended to identify possible adverse effects
and safety risks, to preliminarily evaluate the efficacy of the product for specific targeted diseases and to
determine dosage tolerance and optimal dosage.
● Phase 3. If a drug candidate is found to be potentially effective and to have an acceptable safety profile in Phase
2 clinical trials, the clinical trial program will be expanded to Phase 3 clinical trials to further evaluate dosage,
clinical efficacy and safety in an expanded patient population at geographically dispersed clinical trial sites. These
trials are intended to establish the overall risk-benefit ratio of the product and provide an adequate basis for
product approval and labeling claims.
Phase 4 clinical trials are conducted after approval to gain additional experience from the treatment of patients in
the intended therapeutic indication and to document a clinical benefit in the case of drugs approved under accelerated
approval regulations, or when otherwise requested by the FDA in the form of post-market requirements or commitments.
Failure to promptly conduct any required Phase 4 clinical trials could result in withdrawal of approval.
Clinical trials are inherently uncertain, and Phase 1, Phase 2 and Phase 3 testing may not be successfully
completed. The FDA or the sponsor may suspend a clinical trial at any time for a variety of reasons, including a finding
that the research subjects or patients are being exposed to an unacceptable health risk. Similarly, an IRB can suspend or
terminate approval of a clinical trial at its institution if the clinical trial is not being conducted in accordance with the IRB’s
requirements or if the drug has been associated with unexpected serious harm to patients. In some cases, clinical trials are
overseen by an independent group of qualified experts organized by the trial sponsor, which is called the clinical
monitoring board or data safety monitoring board. This group provides authorization for whether or not a trial may move
forward at designated check points. These decisions are based on the limited access to data from the ongoing trial.
During the development of a new drug, sponsors are given opportunities to meet with the FDA at certain points.
These points may be prior to the submission of an IND, at the end of Phase 2 and before an NDA is submitted. Meetings at
other times may be requested. These meetings can provide an opportunity for the sponsor to share information about the
data gathered to date and for the FDA to provide advice on the next phase of development. Sponsors typically use the
meeting at the end of Phase 2 to discuss their Phase 2 clinical trial results and present their plans for the pivotal Phase 3
clinical trial or trials that they believe will support the approval of the new drug.
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Concurrent with clinical trials, sponsors usually complete additional animal safety studies and also develop
additional information about the chemistry and physical characteristics of the drug and finalize a process for manufacturing
commercial quantities of the product in accordance with cGMP requirements. The manufacturing process must be capable
of consistently producing quality batches of the drug and the manufacturer must develop methods for testing the quality,
purity and potency of the drug. Additionally, appropriate packaging must be selected and tested, and stability studies must
be conducted to demonstrate that the drug candidate does not undergo unacceptable deterioration over its proposed shelf-
life.
The results of product development, preclinical studies and clinical trials, along with descriptions of the
manufacturing process, analytical tests and other control mechanisms, proposed labeling and other relevant information are
submitted to the FDA as part of an NDA requesting approval to market the product. The submission of an NDA is subject
to the payment of user fees, but a waiver of such fees may be obtained under specified circumstances. The FDA reviews all
NDAs submitted for a period of 60 days to ensure that they are sufficiently complete for substantive review before it
accepts them for filing. It may request additional information rather than accept an NDA for filing. In this event, the NDA
must be resubmitted with the additional information. The resubmitted application also is subject to review before the FDA
accepts it for filing.
During the approval process, the FDA also will determine whether a risk evaluation and mitigation strategy, or
REMS, is necessary to assure the safe use of the product. If the FDA concludes a REMS is needed, the sponsor of the NDA
must submit a proposed REMS, and the FDA will not approve the application without an approved REMS, if required. A
REMS can substantially increase the costs of obtaining approval. The FDA could also require a special warning, known as
a boxed warning, to be included in the product label in order to highlight a particular safety risk.
Once the submission is accepted for filing, the FDA begins an in-depth review. The FDA reviews an NDA to
determine, among other things, whether a product is safe and effective for its intended use and whether its manufacturing is
cGMP-compliant. The FDA may refer the NDA to an advisory committee for review and recommendation as to whether
the application should be approved and under what conditions. The FDA is not bound by the recommendation of an
advisory committee, but it generally follows such recommendations. NDAs receive either standard or priority review. A
drug representing a significant improvement in treatment, prevention or diagnosis of disease may receive priority review. A
priority review designation is intended to direct overall attention and resources to the evaluation of such applications, and
to shorten the FDA’s goal for taking action on the NDA from ten months to six months from filing of the NDA. After the
FDA evaluates the NDA and conducts inspections of manufacturing facilities where the drug product and/or its active
pharmaceutical ingredient will be produced, it may issue an approval letter or a Complete Response Letter. An approval
letter authorizes commercial marketing of the drug with specific prescribing information for specific indications. A
Complete Response Letter indicates that the review cycle of the application is complete, and the application is not ready for
approval. A Complete Response Letter may require additional clinical data and/or an additional pivotal Phase 3 clinical
trial(s), and/or other significant, expensive and time-consuming requirements related to clinical trials, preclinical studies or
manufacturing. Even if such data and information are submitted, the FDA may ultimately decide that the NDA does not
satisfy the criteria for approval.
Post-approval Requirements
Drugs manufactured or distributed pursuant to FDA approvals are subject to pervasive and continuing regulation
by the FDA and other governmental agencies, including, among other things, requirements relating to recordkeeping,
periodic reporting, product sampling and distribution, advertising and promotion, and reporting of adverse experiences
with the product. Once an approval is granted, the FDA may withdraw the approval if compliance with regulatory
requirements is not maintained or if problems occur after the product reaches the market. Later discovery of previously
unknown problems with a product may result in restrictions on the product or even complete withdrawal of the product
from the market. After approval, some types of changes to the approved product, such as adding new indications,
manufacturing changes and additional labeling claims, are subject to further FDA review and approval. There are also
continuing, annual user fee requirements for products and the establishments at which such products are manufactured, as
well as new application fees for certain supplemental applications. In addition, the FDA may require testing and
surveillance programs to monitor the effect of approved products that have been commercialized, and the FDA has the
power to prevent or limit further marketing of a product based on the results of these post-marketing programs.
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Drug manufacturers and other entities involved in the manufacture and distribution of approved drugs are required
to register their establishments with the FDA and certain state agencies, and are subject to periodic unannounced
inspections by the FDA and some state agencies for compliance with GMP regulations and other laws. The FDA has
promulgated specific requirements for drug cGMPs and device cGMPs embodied in the Quality System Regulation.
Changes to the manufacturing process are strictly regulated and often require prior FDA approval before being
implemented. FDA regulations also require investigation and correction of any deviations from cGMP requirements and
impose reporting and documentation requirements upon the sponsor and any third-party manufacturers that the sponsor
may decide to use. Accordingly, manufacturers must continue to expend time, money, and effort in the area of production
and quality control to maintain cGMP compliance.
Failure to comply with the applicable U.S. requirements at any time during the product development process or
approval process, or after approval, may subject us to administrative or judicial sanctions, any of which could have a
material adverse effect on us. These sanctions could include:
● refusal to approve pending applications;
● withdrawal of an approval;
● imposition of a clinical hold;
● warning letters;
● product seizures or detention, or refusal to permit the import or export of products;
● restrictions on the marketing or manufacturing of the product;
● total or partial suspension of production or distribution or product recalls; or
● injunctions, fines, disgorgement, or civil or criminal penalties.
The FDA strictly regulates the marketing, labeling, advertising and promotion of drug products that are placed on
the market. Drugs may be promoted only for the approved indications and in accordance with the provisions of the
approved label. However, companies may share truthful and not misleading information that is otherwise consistent with
the product’s FDA approved labeling. The FDA and other agencies actively enforce the laws and regulations prohibiting
the promotion of off-label uses, and a company that is found to have improperly promoted off-label uses may be subject to
significant liability. However, physicians may, in their independent medical judgment, prescribe legally available products
for off-label uses. The FDA does not regulate the behavior of physicians in their choice of treatments but the FDA does
restrict manufacturer’s communications on the subject of off-label use of their products.
From time to time, legislation is drafted, introduced and passed in Congress that could significantly change the
statutory provisions governing the approval, manufacturing and marketing of products regulated by the FDA. In addition,
FDA regulations and guidance are often issued revised or reinterpreted by the agency in ways that may significantly affect
our business and our drug candidates. It is impossible to predict whether legislative changes will be enacted, or whether
FDA regulations, guidance or interpretations will be issued or changed or what the impact of such changes, if any, may be.
Non-patent Exclusivity
The FDCA provides a five-year period of non-patent marketing exclusivity within the United States to the first
applicant to obtain approval of an NDA for a new chemical entity, or NCE. A drug is an NCE if the FDA has not
previously approved any other new drug containing the same active moiety, which is the molecule or ion responsible for
the action of the drug substance. If market exclusivity is granted for an NCE, during the exclusivity period, the FDA may
not accept for review or approve an abbreviated new drug application, or ANDA, or a 505(b)(2) NDA submitted by
another company for another version of such drug where the applicant does not own or have a legal right of reference to all
the data required for approval. However, an application may be submitted after four years if it contains a certification of
patent invalidity or non-infringement to one of the patents listed with the FDA by the innovator NDA holder.
The FDCA also provides three years of marketing exclusivity for an NDA, or supplement to an existing NDA if
new clinical investigations, other than bioavailability studies, that were conducted or sponsored by the applicant are
deemed by the FDA to be essential to the approval of the application, for example new indications, dosages, dosage forms
or strengths of an existing drug. This three-year exclusivity covers only the conditions associated with the new clinical
investigations and prohibits the FDA from approving an ANDA or a 505(b)(2) NDA submitted by another company with
overlapping conditions associated with the new clinical investigations for the three-year period. Clinical investigation
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exclusivity does not prohibit the FDA from approving ANDAs for drugs containing the original active agent. Five-year and
three-year exclusivity will not delay the submission or approval of an NDA for the same drug. However, an applicant
submitting an NDA would be required to conduct or obtain a right of reference to all of the preclinical studies and adequate
and well-controlled clinical trials necessary to demonstrate safety and effectiveness.
Orphan Drug Designation
Under the Orphan Drug Act, the FDA may grant orphan drug designation to products intended to treat a rare
disease or condition—generally one that affects fewer than 200,000 individuals in the United States. Orphan drug
designation must be requested before submitting the NDA. After the FDA grants orphan drug designation, the FDA
publicly discloses the drug’s identity and its intended orphan use. Orphan drug designation does not convey any advantage
in, or shorten the duration of, the regulatory review and approval process. The first active moiety to be approved to treat a
disease with FDA’s orphan drug designation is entitled to a seven-year period of marketing exclusivity in the United States
for that product, for that indication. During the seven-year exclusivity period, the FDA may not approve any other
applications to market the same drug for the same orphan indication, regardless of patent status, except in limited
circumstances, such as a showing of clinical superiority to the product with orphan exclusivity or if the FDA finds that the
holder of the orphan exclusivity has not shown that it can assure the availability of sufficient quantities of the orphan drug
to meet the needs of patients with the disease or condition for which the drug was designated. Orphan drug exclusivity does
not prevent the FDA from approving a different chemical/biological entity for the same disease or condition. An orphan
drug designation also does not preclude the same drug from being developed for a different disease or condition. Among
the other benefits of orphan drug designation are tax credits for certain research expenses and a waiver of the application
user fee.
Regulation Outside of the United States
Even if we obtain FDA approval for a drug candidate, we must obtain approval by the comparable regulatory
authorities of countries outside of the United States before we can commence clinical trials in such countries, and our
potential third-party partners must obtain approval of the regulators of such countries or economic areas, such as the
European Union, before they may market any of our drug candidates in those countries or areas. The approval process and
requirements governing the conduct of clinical trials, product licensing and promotion, pricing and reimbursement vary
greatly by geographic region, and the time may be longer or shorter than that required for FDA approval.
In the European Economic Area, or EEA, which is composed of the Member States of the European Union plus
Norway, Iceland and Liechtenstein, medicinal products can only be commercialized after obtaining a Marketing
Authorization, or MA.
There are two types of MAs:
● The Community MA, which is issued by the European Commission through the Centralized Procedure, based on
the opinion of the Committee for Medicinal Products for Human Use, or CHMP, of the European Medicines
Agency, or EMA, and which is valid throughout the entire territory of the EEA. The Centralized Procedure is
mandatory for certain types of products, such as biotechnology medicinal products, orphan medicinal products,
and medicinal products indicated for the treatment of AIDS, cancer, neurodegenerative disorders, diabetes, auto-
immune and viral diseases. The Centralized Procedure is optional for products containing a new active substance
not yet authorized in the EEA, or for products that constitute a significant therapeutic, scientific or technical
innovation or which are in the interest of public health in the European Union. Under the Centralized Procedure,
the maximum timeframe for the evaluation of a marketing authorization application is 210 days (excluding clock
stops, when additional written or oral information is to be provided by the applicant in response to questions
asked by the CHMP). Accelerated evaluation might be granted by the CHMP in exceptional cases, when the
authorization of a medicinal product is of major interest from the point of view of public health and, in particular,
from the viewpoint of therapeutic innovation. Under the accelerated procedure, the standard 210 days review
period is reduced to 150 days.
● National MAs, which are issued by the competent authorities of the Member States of the EEA and only cover
their respective territory, are available for products not falling within the mandatory scope of the Centralized
Procedure. Where a product has already been authorized for marketing in a Member State of the EEA, this
National MA can be recognized in another Member State through the Mutual Recognition Procedure. If the
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product has not received a National MA in any Member State at the time of application, it can be approved
simultaneously in various Member States through the Decentralized Procedure.
In the EEA, upon receiving marketing authorization, new chemical entities generally receive eight years of data
exclusivity and an additional two years of market exclusivity. If granted, data exclusivity prevents regulatory authorities in
the EEA from referencing the innovator’s data to assess a generic application. During the additional two-year period of
market exclusivity, a generic marketing authorization can be submitted, and the innovator’s data may be referenced, but no
generic product can be marketed until the expiration of the market exclusivity. However, there is no guarantee that a
product will be considered by the EEA’s regulatory authorities to be a new chemical entity, and products may not qualify
for data exclusivity.
Other Health Care Laws
Health care providers, physicians and third-party payors in the United States and elsewhere will play a primary
role in the recommendation and prescription of any of our drug candidates for which marketing approval is obtained. Our
potential third-party partners’ arrangements with third-party payors, health care professionals and customers may expose
them to broadly applicable fraud and abuse and other health care laws and regulations, including, without limitation, the
federal Anti-Kickback Statute and the federal civil False Claims Act, that may constrain the business or financial
arrangements and relationships through which they sell, market and distribute any drug candidates for which marketing
approval is obtained. In addition, we and our potential third-party partners may be subject to transparency laws and patient
privacy regulation by the federal government and by the U.S. states and foreign jurisdictions in which we or they conduct
business.
The federal Anti-Kickback Statute makes it illegal for any person or entity, including a prescription drug
manufacturer (or a party acting on its behalf) to knowingly and willfully, directly or indirectly, solicit, receive, offer, or pay
any remuneration that is intended to induce the referral of business, including the purchase, order, or lease of any good,
facility, item or service for which payment may be made under a federal health care program, such as Medicare or
Medicaid. The term “remuneration” has been broadly interpreted to include anything of value. The Anti-Kickback Statute
has been interpreted to apply to arrangements between pharmaceutical manufacturers on one hand and prescribers,
purchasers, formulary managers, and beneficiaries on the other. Although there are a number of statutory exceptions and
regulatory safe harbors protecting some common activities from prosecution, the exceptions and safe harbors are drawn
narrowly. Practices that involve remuneration that may be alleged to be intended to induce prescribing, purchases or
recommendations may be subject to scrutiny if they do not qualify for an exception or safe harbor. Failure to meet all of the
requirements of a particular applicable statutory exception or regulatory safe harbor does not make the conduct per se
illegal under the Anti-Kickback Statute. Instead, the legality of the arrangement will be evaluated on a case-by-case basis
based on a cumulative review of all its facts and circumstances. Several courts have interpreted the statute’s intent
requirement to mean that if any one purpose of an arrangement involving remuneration is to induce referrals of federal
health care covered business, the Anti-Kickback Statute has been violated. Violations of this law are punishable by up to
ten years in prison, and can also result in criminal fines, civil monetary penalties, administrative penalties and exclusion
from participation in federal health care programs.
Additionally, the intent standard under the Anti-Kickback Statute was amended by the Patient Protection and
Affordable Care Act of 2010, as amended by the Health Care and Education Reconciliation Act of 2010, or collectively the
Affordable Care Act, to a stricter standard such that a person or entity no longer needs to have actual knowledge of the
statute or specific intent to violate it in order to have committed a violation. In addition, the Affordable Care Act codified
case law that a claim including items or services resulting from a violation of the federal Anti-Kickback Statute constitutes
a false or fraudulent claim for purposes of the federal civil False Claims Act.
Federal false claims and false statement laws, including the federal civil False Claims Act, prohibits, among other
things, any person or entity from knowingly presenting, or causing to be presented, for payment to, or approval by, federal
programs, including Medicare and Medicaid, claims for items or services, including drugs, that are false or fraudulent or
not provided as claimed. Entities can be held liable under these laws if they are deemed to “cause” the submission of false
or fraudulent claims by, for example, providing inaccurate billing or coding information to customers, promoting a product
off-label, or for providing medically unnecessary services or items. In addition, activities relating to the sale and marketing
of products are subject to scrutiny under this law. Penalties for federal civil False Claims Act violations may include up to
three times the actual damages sustained by the government, plus mandatory civil penalties for each separate false claim,
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the potential for exclusion from participation in federal health care programs, and, although the federal civil False Claims
Act is a civil statute, False Claims Act violations may also implicate various federal criminal statutes.
The federal Health Insurance Portability and Accountability Act of 1996, or HIPAA, prohibits among other
actions, knowingly and willfully executing, or attempting to execute, a scheme to defraud any health care benefit program,
including private third-party payors, knowingly and willfully embezzling or stealing from a health care benefit program,
willfully obstructing a criminal investigation of a health care offense, and knowingly and willfully falsifying, concealing or
covering up a material fact or making any materially false, fictitious or fraudulent statement in connection with the delivery
of or payment for health care benefits, items or services. Like the Anti-Kickback Statute, the Affordable Care Act amended
the intent standard for the health care fraud statute under HIPAA such that a person or entity no longer needs to have actual
knowledge of the statute or specific intent to violate it in order to have committed a violation.
The civil monetary penalties statute imposes penalties against any person or entity that, among other things, is
determined to have presented or caused to be presented a claim to a federal health program that the person knows or should
know is for an item or service that was not provided as claimed or is false or fraudulent.
Also, many states have similar fraud and abuse statutes or regulations that may be broader in scope and may apply
regardless of payor, in addition to items and services reimbursed under Medicaid and other state programs. Additionally, to
the extent that a product is sold in a foreign country, the seller may be subject to similar foreign laws.
In addition, legislation imposing marketing restrictions and transparency requirements on pharmaceutical
manufacturers has been enacted at the state and federal levels. For example, the Affordable Care Act imposed, among
other things, annual reporting requirements to the Centers for Medicare & Medicaid Services, or CMS, for covered
manufacturers for certain payments and other transfers of value provided to physicians (defined to include doctors, dentists,
optometrists, podiatrists and chiropractors) and teaching hospitals, as well as ownership and investment interests held by
physicians and their immediate family members. Beginning in 2022, applicable manufacturers will also be required to
report information regarding payments and other transfers of value provided during the previous year to physician
assistants, nurse practitioners, clinical nurse specialists, certified nurse anesthetists, anesthesiologist assistants, and certified
nurse-midwives. Failure to submit timely, accurately and completely the required information for all payments, transfers of
value and ownership or investment interests may result in civil monetary penalties for “knowing failures.” Certain states
also mandate implementation of compliance programs, impose restrictions on drug manufacturer marketing practices,
require registration of certain employees engaged in marketing activities in the location, and/or require the tracking and
reporting of gifts, compensation and other remuneration to physicians.
We have developed a comprehensive compliance program that establishes internal controls to facilitate adherence
to the rules and program requirements to which we are subject. Although the development and implementation of
compliance programs designed to establish internal controls and facilitate compliance can mitigate the risk of investigation,
prosecution, and penalties assessed for violations of these laws, or any other laws that may apply to us, the risks cannot be
entirely eliminated. If our operations are found to be in violation of any such laws or any other governmental regulations,
we may be subject to significant penalties, including, without limitation, administrative, civil, and criminal penalties,
damages, fines, disgorgement, imprisonment, contractual damages, reputational harm, diminished profits and future
earnings, the curtailment or restructuring of our operations, exclusion from participation in federal and state health care
programs, additional reporting requirements and oversight if we become subject to a corporate integrity agreement or
similar agreement to resolve allegations of non-compliance with these laws and individual imprisonment, any of which
could adversely affect our ability to operate our business and our financial results.
We may also be subject to data privacy and security regulation by both the federal government and the states in
which we conduct our business. HIPAA, as amended by the Health Information Technology for Economic and Clinical
Health Act, or HITECH, and their implementing regulations, including the final omnibus rule published on January 25,
2013, mandates, among other things, the adoption of uniform standards for the electronic exchange of information in
common health care transactions, as well as standards relating to the privacy and security of individually identifiable health
information, which require the adoption of administrative, physical and technical safeguards to protect such information.
Among other things, HITECH makes HIPAA’s security standards directly applicable to “business associates”, namely
independent contractors or agents of HIPAA covered entities that create, receive or obtain protected health information in
connection with providing a service for or on behalf of a covered entity and their subcontractors that use, disclose, access,
or otherwise process protected health information. HITECH also increased the civil and criminal penalties that may be
imposed against covered entities and business associates, and gave state attorneys general new authority to file civil actions
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for damages or injunctions in federal courts to enforce the federal HIPAA laws and seek attorneys’ fees and costs
associated with pursuing federal civil actions. In addition, certain state laws govern the privacy and security of health
information in certain circumstances, some of which are more stringent than HIPAA and many of which differ from each
other in significant ways and may not have the same effect, thus complicating compliance efforts. Failure to comply with
these laws, where applicable, can result in the imposition of significant civil and/or criminal penalties.
Health Care Reform
In the United States, there have been and continue to be a number of significant legislative initiatives to contain
health care costs. For example, in March 2010, the Affordable Care Act was passed, which has had, and is expected to
continue to have, a significant impact on the health care industry. The Affordable Care Act was designed to expand
coverage for the uninsured and at the same time contain overall health care costs. With regard to pharmaceutical products,
among other things, the Affordable Care Act expanded and increased industry rebates for drugs covered under Medicaid
programs; addressed a new methodology by which rebates owed by manufacturers under the Medicaid Drug Rebate
Program are calculated for drugs that are inhaled, infused, instilled, implanted or injected; extended the rebate program to
individuals enrolled in Medicaid managed care organizations; established annual fees and taxes on manufacturers of certain
branded prescription drugs; made changes to the coverage requirements under the Medicare prescription drug benefit; and
established a new Medicare Part D coverage gap discount program, in which manufacturers, as a condition for their
outpatient drugs to be covered under Medicare Part D, must agree to offer 70% point-of-sale discounts off negotiated prices
of applicable brand drugs to eligible beneficiaries during their coverage gap period. Moreover, the Affordable Care Act
provided incentives to programs that increase the federal government’s comparative effectiveness research and
implemented payment system reforms including a national pilot program on payment bundling meant to encourage
hospitals, physicians and other providers to improve the coordination, quality and efficiency of certain health care services.
There remain judicial and Congressional challenges to certain aspects of the Affordable Care Act. While Congress
has not passed comprehensive repeal legislation, several bills affecting the implementation of certain taxes under the
Affordable Care Act have been signed into law. The Tax Cuts and Jobs Act of 2017 includes a provision repealing,
effective January 1, 2019, the tax-based shared responsibility payment imposed by the Affordable Care Act on certain
individuals who fail to maintain qualifying health coverage for all or part of a year that is commonly referred to as the
“individual mandate”. In addition, the 2020 federal spending package permanently eliminated, effective January 1, 2020,
the Affordable Care Act-mandated “Cadillac” tax on high-cost employer-sponsored health coverage and medical device tax
and, effective January 1, 2021, also eliminated the health insurer tax. Further, the Bipartisan Budget Act of 2018, or the
BBA, among other things, amended the Affordable Care Act, effective January 1, 2019, to close the coverage gap in most
Medicare drug plans, commonly referred to as the “donut hole”. On December 14, 2018, a Texas U.S. District Court Judge
ruled that the Affordable Care Act is unconstitutional in its entirety because the “individual mandate” was repealed by
Congress as part of the Tax Cuts and Jobs Act of 2017. Additionally, on December 18, 2019, the U.S. Court of Appeals for
the Fifth Circuit upheld the District Court ruling that the individual mandate was unconstitutional and remanded the case
back to the District Court to determine whether the remaining provisions of the Affordable Care Act are invalid as well.
The U.S. Supreme Court is currently reviewing the case, although it is unknown when a decision will be made. Further,
although the U.S. Supreme Court has not yet ruled on the constitutionality of the Affordable Care Act, on January 28,
2021, President Biden issued an executive order to initiate a special enrollment period from February 15, 2021 through
May 15, 2021 for purposes of obtaining health insurance coverage through the Affordable Care Act marketplace. The
executive order also instructs certain governmental agencies to review and reconsider their existing policies and rules that
limit access to healthcare, including among others, reexamining Medicaid demonstration projects and waiver programs that
include work requirements, and policies that create unnecessary barriers to obtaining access to health insurance coverage
through Medicaid or the Affordable Care Act. It is unclear how the Supreme Court ruling, other such litigation, and the
health care reform measures of the Biden administration will impact the Affordable Care Act and our business.
In addition, other legislative changes have been proposed and adopted since the Affordable Care Act was enacted.
For example, in August 2011, President Obama signed into law the Budget Control Act of 2011, which, among other
things, created the Joint Select Committee on Deficit Reduction to recommend to Congress proposals in spending
reductions. The Joint Select Committee on Deficit Reduction did not achieve a targeted deficit reduction of at least $1.2
trillion for fiscal years 2012 through 2021, triggering the legislation’s automatic reduction to several government programs.
This includes aggregate reductions in Medicare payments to providers of 2% per fiscal year, which went into effect
beginning on April 1, 2013 and, due to subsequent legislative amendments to the statute, including the BBA, will stay in
effect through 2030 with the exception of a temporary suspension from May 1, 2020 through March 31, 2021, unless
additional Congressional action is taken. Additionally, in January 2013, the American Taxpayer Relief Act of 2012 was
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signed into law, which, among other things, reduced Medicare payments to several providers, including hospitals, cancer
treatment centers and imaging centers. Moreover, the Drug Supply Chain Security Act imposes new obligations on
manufacturers of pharmaceutical products related to product tracking and tracing. Legislative and regulatory proposals
have been made to expand post-approval requirements and restrict sales and promotional activities for pharmaceutical
products.
More recently, there has been heightened governmental scrutiny over the manner in which manufacturers set
prices for their marketed products. Such scrutiny has resulted in several recent Congressional inquiries and proposed and
enacted federal and state legislation designed to, among other things, bring more transparency to product pricing, review
the relationship between pricing and manufacturer patient programs, and reform government program reimbursement
methodologies for products. At the federal level, the Trump administration used several means to propose or implement
drug pricing reform, including through federal budget proposals, executive orders and policy initiatives. For example, on
July 24, 2020 and September 13, 2020, President Trump announced several executive orders related to prescription drug
pricing that seek to implement several of the administration’s proposals. As a result, the FDA released a final rule on
September 24, 2020, effective November 30, 2020 providing guidance for states to build and submit importation plans for
drugs from Canada. Further, on November 20, 2020, the Department of Health and Human Services, or HHS, finalized a
regulation removing safe harbor protection for price reductions from pharmaceutical manufacturers to plan sponsors under
Part D, either directly or through pharmacy benefit managers, unless the price reduction is required by law. The
implementation of the rule has been delayed by the Biden administration from January 1, 2022 to January 1, 2023 in
response to ongoing litigation. The rule also created a new safe harbor for price reductions reflected at the point-of-sale, as
well as a new safe harbor for certain fixed fee arrangements between pharmacy benefit managers and manufacturers, the
implementation of which have also been delayed pending review by the Biden administration until March 22, 2021. On
November 20, 2020, CMS issued an interim final rule implementing the President Trump’s Most Favored Nation executive
order, which would tie Medicare Part B payments for certain physician-administered drugs to the lowest price paid in other
economically advanced countries, effective January 1, 2021. On December 28, 2020, the U.S. District Court in Northern
California issued a nationwide preliminary injunction against implementation of the interim final rule. It is unclear whether
the Biden administration will work to reverse these measures or pursue similar policy initiatives. At the state level,
legislatures have become increasingly active in passing legislation and implementing regulations designed to control
pharmaceutical and biological product pricing, including price or patient reimbursement constraints, discounts, restrictions
on certain product access and marketing cost disclosure and transparency measures, and, in some cases, designed to
encourage importation from other countries and bulk purchasing.
The Affordable Care Act, as well as other federal and state health care reform measures that have been and may
be adopted in the future, could harm our future revenue. Additional legislative actions may be taken in the future which
may change current regulations, guidance and interpretations. Further, it is also possible that additional governmental
action is taken in response to the COVID-19 pandemic. The impact of such actions on our business, if any, cannot
presently be determined.
The Hatch Waxman Amendments to the FDCA
Orange Book Listing
In seeking approval for a drug through an NDA, applicants are required to list with the FDA each patent whose
claims cover the applicant’s product or a method of using the product. Upon approval of a drug, each of the patents listed in
the application for the drug is then published in the FDA’s Approved Drug Products with Therapeutic Equivalence
Evaluations, commonly known as the Orange Book. Drugs listed in the Orange Book can, in turn, be cited by potential
competitors in support of approval of an ANDA or an application covered by Section 505(b)(2) of the FDCA. An ANDA
provides for marketing of a drug product that has the same active ingredients, generally in the same strengths and dosage
form, as the listed drug and has been shown through pharmacokinetic, or PK, testing to be bioequivalent to the listed drug.
Drugs approved in this way are commonly referred to as “generic equivalents” to the listed drug, and can often be
substituted by pharmacists under prescriptions written for the original listed drug. Other than the requirement for
bioequivalence testing, ANDA applicants are generally not required to conduct, or submit results of, preclinical studies or
clinical tests to prove the safety or effectiveness of their drug product. Section 505(b)(2) applications provide for marketing
of a drug product that may have the same active ingredients as the listed drug and contains full safety and effectiveness
data as an NDA, but at least some of this information comes from studies not conducted by or for the applicant. This
alternate regulatory pathway enables the applicant to rely, in part, on the FDA’s findings of safety and efficacy for an
existing product, or published literature, in support of its application. The FDA may then approve the new drug candidate
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for all or some of the labeled indications for which the referenced product has been approved, as well as for any new
indication sought by the 505(b)(2) applicant.
The ANDA or Section 505(b)(2) applicant is required to certify to the FDA concerning any patents listed for the
approved product in the FDA’s Orange Book. Specifically, the applicant must certify that: (i) the required patent
information has not been filed; (ii) the listed patent has expired; (iii) the listed patent has not expired, but will expire on a
particular date and approval is sought after patent expiration; or (iv) the listed patent is invalid or will not be infringed by
the new product. The ANDA or Section 505(b)(2) applicant may also elect to submit a statement certifying that its
proposed ANDA label does not contain, or carves out, any language regarding a patented method of use rather than certify
to such listed method of use patent. If the applicant does not challenge the listed patents by filing a certification that the
listed patent is invalid or will not be infringed by the new product, the ANDA or Section 505(b)(2) application will not be
approved until all the listed patents claiming the referenced product have expired.
A certification that the new product will not infringe the already approved product’s listed patents, or that such
patents are invalid, is called a Paragraph IV certification. If the ANDA or Section 505(b)(2) applicant has provided a
Paragraph IV certification to the FDA, the applicant must also send notice of the Paragraph IV certification to the NDA
and patent holders once the ANDA or Section 505(b)(2) application has been accepted for filing by the FDA. The NDA
and patent holders may then initiate a patent infringement lawsuit in response to the notice of the Paragraph IV
certification. The filing of a patent infringement lawsuit within 45 days of the receipt of a Paragraph IV certification
automatically prevents the FDA from approving the ANDA or Section 505(b)(2) application until the earliest of 30 months,
expiration of the patent, settlement of the lawsuit, and a decision in the infringement case that is favorable to the ANDA or
Section 505(b)(2) applicant. This prohibition is generally referred to as the 30-month stay. Thus, approval of an ANDA or
505(b)(2) NDA could be delayed for a significant period of time depending on the patent certification the applicant makes
and the reference drug sponsor’s decision to initiate patent litigation.
The ANDA or Section 505(b)(2) application also will not be approved until any applicable non-patent exclusivity
listed in the Orange Book for the referenced product has expired.
Patent Term Extension
In the United States, after NDA approval, owners of relevant drug patents may apply for up to a five year patent
extension, which provides patent term restoration as compensation for the patent term lost during the FDA regulatory
review process for the first permitted commercial marketing of a drug product. The Drug Price Competition and Patent
Term Restoration Act of 1984, or the Hatch-Waxman Act, permits a patent term extension of up to five years beyond the
expiration of the patent. The allowable patent term extension is calculated as half of the drug’s testing phase, which is the
time between the IND submission becoming effective and the NDA submission, and all of the review phase, which is the
time between NDA submission and approval, up to a maximum extension of five years. The time can be shortened if the
FDA determines that the applicant did not pursue approval with due diligence. Patent extension cannot extend the
remaining term of a patent beyond a total of 14 years from the date of product approval and only one patent applicable to
an approved drug may be extended.
Similar provisions are available in the European Union and other foreign jurisdictions to extend the term of a
patent that covers an approved drug. For example, in Japan, it may be possible to extend the patent term for up to five years
and in the European Union, it may be possible to obtain a supplementary patent certificate that would effectively extend
patent protection for up to five years.
Coverage and Reimbursement
We believe the success of our drug candidates, if approved, will depend on obtaining and maintaining coverage
and adequate reimbursement as a prescription treatment or in the absence of coverage and adequate reimbursement, on the
extent to which patients will be willing to pay out of pocket for our prescription drug products.
Third-party payors determine which prescription drug products they will cover and establish reimbursement
levels. Reimbursement by a third-party payor may depend upon a number of factors, including: the third-party payor’s
determination that a product is safe, effective, and medically necessary; appropriate for the specific patient; cost-effective;
supported by peer-reviewed medical journals or current clinical practice guidelines; and whether there are competitive
products, either branded or generic, and the pricing of those products. Many private third-party payors, such as managed
care plans, manage access to drug products’ coverage partly to control costs for their plans, and may use drug formularies
and medical policies to limit their exposure. Obtaining and maintaining favorable reimbursement can be a time-consuming
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and expensive process, and our potential third-party partners may not be able to negotiate or continue to negotiate
reimbursement or pricing terms for our drug candidates, if approved, with third-party payors at levels that are profitable to
us, or at all.
In addition to uncertainties surrounding coverage policies, there are periodic changes to reimbursement. Third-
party payors regularly update reimbursement amounts and also from time to time revise the methodologies used to
determine reimbursement amounts. Accordingly, these updates could impact the demand for our drug candidates, if
approved. Our drug candidates, if approved, may not be considered cost effective, and government and third-party private
health insurance coverage and reimbursement may not be available to patients or sufficient to allow our potential third-
party partners to sell our drug candidates, if approved, on a competitive and profitable basis. Our results of operations
could be adversely affected by the Affordable Care Act and by other health care reforms that may be enacted or adopted in
the future. In addition, increasing emphasis on managed care in the United States will continue to put pressure on the
pricing of pharmaceutical products. Cost control initiatives could decrease the price that our potential third-party partners
could receive for any of our drug candidates, if approved, and could adversely affect our profitability. We cannot predict
how pending and future health care legislation will impact our business, and any changes in coverage and reimbursement
that further restricts coverage of our drug candidates could harm our business.
Foreign governments also have their own health care reimbursement systems, which vary significantly by country
and region, and we cannot be sure that coverage and adequate reimbursement will be made available with respect to our
drug candidates, if approved, under any foreign reimbursement system. In some foreign countries, including major
markets in the European Union and Japan, the pricing of prescription pharmaceuticals is subject to governmental
control. In these countries, pricing negotiations with governmental authorities can take up to 12 months or longer after the
receipt of regulatory marketing approval for a product. To obtain reimbursement or pricing approval in some countries, we
may be required to conduct a pharmacoeconomic study that compares the cost-effectiveness of our drug candidate to other
available therapies. Such pharmacoeconomic studies can be costly and the results uncertain. Our business could be
harmed if reimbursement of our drug candidates, if approved, is unavailable or limited in scope or amount or if pricing is
set at unsatisfactory levels.
Employees and Human Capital Resources
As of December 31, 2020, we had 60 total employees, of which 57 were full-time employees. All of our
employees are located in the United States. None of our employees are represented by a labor union or covered by a
collective bargaining agreement. We consider our relationship with our employees to be good.
Our human capital resources objectives include, as applicable, identifying, recruiting, retaining, incentivizing and
integrating our existing and new employees, advisors and consultants. The principal purposes of our equity incentive plans
are to attract, retain and reward personnel through the granting of stock-based compensation awards in order to increase
stockholder value and the success of our company by motivating such individuals to perform to the best of their abilities
and achieve our objectives.
Corporate Information
We were incorporated under the laws of the State of Delaware in July 2012. Our principal executive offices are
located at 640 Lee Road, Suite 200, Wayne, PA 19087. Our telephone number is (484) 324-7933. Our common stock is
listed on the Nasdaq Global Select Market under the symbol “ACRS.”
Available Information
Our internet website address is www.aclaristx.com. In addition to the information contained in this Annual
Report, information about us can be found on our website. Our website and information included in or linked to our
website are not part of this Annual Report.
Our annual reports on Form 10-K, quarterly reports on Form 10-Q, current reports on Form 8-K and amendments
to those reports filed or furnished pursuant to Section 13(a) or 15(d) of the Exchange Act are available free of charge
through our website as soon as reasonably practicable after they are electronically filed with or furnished to the Securities
and Exchange Commission, or SEC. The SEC also maintains a website that contains our reports, proxy and information
statements and other information. The address of the SEC’s website is www.sec.gov.
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Item 1A. Risk Factors
Our business is subject to numerous risks. You should carefully consider the following risks and all other
information contained in this Annual Report, as well as general economic and business risks, together with any other
documents we file with the SEC. If any of the following events actually occur or risks actually materialize, it could have a
material adverse effect on our business, operating results and financial condition and cause the trading price of our
common stock to decline.
Summary of Risk Factors
● We have incurred significant losses since our inception. We expect to incur losses over the next several years
and may never achieve or maintain profitability.
● We will need substantial additional funding to meet our financial obligations and to pursue our business
objectives. If we are unable to raise capital when needed, we could be forced to curtail our planned
operations.
● Our business is dependent on the successful development of our investigational drug candidate, ATI-450.
● We have a limited history as a clinical-stage biopharmaceutical company developing and partnering our drug
candidates, which may make it difficult to evaluate the success of our business to date and to assess our
future viability.
● If we are unable to successfully develop our drug candidates and to pursue strategic alternatives, including
identifying and consummating transactions with third-party partners, to further develop, obtain marketing
approval for and/or commercialize our drug candidates, or experience significant delays in doing so, our
business will be harmed.
● Our future success depends on our ability to retain key executives and to attract, retain and motivate qualified
personnel.
● We intend to pursue strategic alternatives, including identifying and consummating transactions with third-
party partners, to further develop, obtain marketing approval for and/or commercialize our drug candidates.
If those arrangements are not successful, we may not be able to capitalize on the market potential of these
drug candidates.
● Our business has been adversely impacted and could continue to be adversely affected by the evolving and
ongoing COVID-19 global pandemic in regions where we or third parties on which we rely have
manufacturing facilities, clinical trial sites or other business operations. The COVID-19 pandemic could
adversely affect our operations, including at our headquarters and at our clinical trial sites, as well as the
business or operations of our manufacturers, contract research organizations or other third parties with whom
we conduct business.
● If we are unable to obtain and maintain patent protection for our drug candidates, or if the scope of the patent
protection obtained is not sufficiently broad, our competitors could develop and commercialize technology
and drugs similar or identical to ours, and our ability to successfully pursue strategic alternatives, including
identifying and consummating transactions with potential third-party partners, to commercialize our
technology and drug candidates may be impaired.
● We face substantial competition, which may result in others discovering, developing or commercializing
drugs before or more successfully than we do.
Risks Related to Our Business, Our Financial Position and Capital Needs
We have incurred significant losses since our inception. We expect to incur losses over the next several years
and may never achieve or maintain profitability.
Since inception, we have incurred significant net losses. We incurred net losses of $51.0 million and $161.4
million for the years ended December 31, 2020 and 2019, respectively. As of December 31, 2020, we had an accumulated
deficit of $504.5 million. We have financed our operations over the last several years primarily from sales of equity
securities and incurring indebtedness in the form of loans from commercial lenders.
We have devoted substantially all of our financial resources and efforts to the development of our drug candidates,
including preclinical studies and clinical trials, and from 2018 to October 2019, to the commercialization of our products.
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Our net losses may fluctuate significantly from quarter to quarter and year to year. We expect to continue to incur
significant expenses and operating losses in the near term as we:
● pursue strategic alternatives, including identifying and seeking to consummate transactions with third-party
partners, to further develop, obtain marketing approval for and/or commercialize our drug candidates;
● continue the clinical development of ATI-450, our MK2 inhibitor, as a potential treatment for moderate to
severe rheumatoid arthritis and potentially other immuno-inflammatory diseases, and ATI-1777, our “soft”
JAK inhibitor, as a potential treatment for moderate to severe atopic dermatitis;
● continue to develop our preclinical drug candidates, including ATI-2138, our ITJ inhibitor;
● continue to discover and develop additional drug candidates;
● maintain, expand and protect our intellectual property portfolio; and
● incur legal, accounting, investor relations and other administrative expenses in operating as a public
company.
To become and remain profitable, we must succeed in a range of challenging activities, including completing
preclinical testing and clinical trials of our drug candidates and pursuing strategic alternatives, including identifying and
consummating transactions with third-party partners, for the further development and/or commercialization of our drug
candidates, as well as discovering and developing additional drug candidates. We are in the early stages of most of these
activities. We may never succeed in these activities and, even if we do, may never earn revenue from our drug candidates
that is significant enough to achieve profitability.
For any of our drug candidates, our revenue will be dependent, in part, upon our ability to identify and
consummate transactions with third-party partners to further develop, obtain marketing approval for and/or commercialize
those drug candidates. Further, we will be dependent on our potential third-party partners’ ability to obtain marketing
approval and successfully commercialize the product, upon the size of the markets in the territories where marketing
approval is obtained, the accepted price for the product, and the ability to obtain coverage and reimbursement, if any. If we
fail to identify and enter into partnerships with third parties to further develop, obtain marketing approval for and/or
commercialize our drug candidates, any partnerships we enter into do not result in the successful development, marketing
approval for and commercialization of our drug candidates, the number of addressable patients is not as significant as
estimated by our potential third-party partners, the indication approved by regulatory authorities is narrower than expected,
or the treatment population is narrowed by competition, physician choice or treatment guidelines, we may not earn
significant revenue from agreements with potential third-party partners for such drug candidates, even if the drug
candidates are approved for marketing.
Because of the numerous risks and uncertainties associated with drug development, we are unable to accurately
predict the timing or amount of expenses or when, or if, we will be able to achieve profitability. If we are required by
regulatory authorities to perform studies in addition to those expected, or if there are any delays in the initiation and
completion of our clinical trials, the development of any of our drug candidates or the identification and consummation of
transactions with third-party partners to further develop, obtain marketing approval for and/or commercialize our drug
candidates, our expenses could increase.
Even if we achieve profitability, we may not be able to sustain or increase profitability on a quarterly or annual
basis. Our failure to become and remain profitable would depress the value of our company and could impair our ability to
raise capital, expand our business, maintain our development efforts, diversify our offerings or continue our operations. A
decline in the value of our company could also cause you to lose all or part of your investment.
We will need substantial additional funding to meet our financial obligations and to pursue our business
objectives. If we are unable to raise capital when needed, we could be forced to curtail our planned operations.
Identifying potential drug candidates and conducting preclinical testing and clinical trials is a time-consuming,
expensive and uncertain process that takes years to complete, and we may never generate the necessary data or results
required to identify and consummate transactions with third-party partners to further develop, obtain marketing approval
for and/or commercialize our drug candidates. We expect to incur significant expenses and operating losses for the
foreseeable future as we advance our drug candidates from discovery through preclinical and clinical development. In
addition, we may not be able to identify and consummate transactions with third-party partners to further develop, obtain
marketing approval for and/or commercialize our drug candidates, and our drug candidates, if approved, may not achieve
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commercial success. Furthermore, we incur and expect to continue to incur significant costs associated with operating as a
public company, including legal, accounting, investor relations and other expenses.
As of December 31, 2020, we had cash, cash equivalents and marketable securities of $54.1 million. We believe
that our existing cash, cash equivalents and marketable securities as of the date of this Annual Report will enable us to fund
our operating expenses and capital expenditure requirements for a period greater than 12 months from the date of this
report based on our current operating assumptions. These assumptions may prove to be wrong, and we could use our
available capital resources sooner than we expect. Changes may occur beyond our control that would cause us to consume
our available capital before that time, including changes in and progress of our development activities, acquisitions of
additional products or drug candidates, and changes in regulation. Our future capital requirements will depend on many
factors, including:
● the number and development requirements of the drug candidates that we may pursue;
● the scope, progress, results and costs of preclinical development, laboratory testing and conducting
preclinical and clinical trials for our drug candidates;
● the costs, timing and outcome of regulatory review of our drug candidates;
● the extent to which we in-license or acquire additional drug candidates and technologies;
● the costs and timing of preparing, filing and prosecuting patent applications, maintaining and enforcing our
intellectual property rights and defending any intellectual property-related claims;
● the impact on the timing of our preclinical studies, on the recruitment, enrollment, conduct and timing of our
clinical trials, and on our business, due to the COVID-19 pandemic;
● our ability to identify and consummate transactions with third-party partners to further develop, obtain
marketing approval for and/or commercialize our drug candidates, and earn revenue from such arrangements;
and
● the revenue earned from our commercial products as a result of licenses to, or partnerships with, third parties.
We will require additional capital to complete the clinical development of ATI-450 and ATI-1777, to develop our
preclinical compounds and to support our discovery efforts. Additional funds may not be available on a timely basis, on
commercially acceptable terms, or at all, and such funds, if raised, may not be sufficient to enable us to continue to
implement our long-term business strategy. Our ability to raise additional capital may be adversely impacted by potential
worsening global economic conditions and the recent disruptions to, and volatility in, the credit and financial markets in the
United States and worldwide resulting from the ongoing COVID-19 pandemic. If we are unable to raise sufficient
additional capital, we could be forced to curtail our planned operations.
Our business is dependent on the successful development of our drug candidate, ATI-450.
Our pipeline includes ATI-450, our investigational oral, novel, selective MK2 inhibitor compound, which we are
developing as a potential treatment for moderate to severe rheumatoid arthritis and potentially other immuno-inflammatory
diseases. The success of our business will significantly depend on our successful development of and/or our ability to
pursue strategic alternatives for, including identifying and consummating transactions with third-party partners, to further
develop, obtain marketing approval for and/or commercialize, ATI-450.
Raising additional capital may cause dilution to our stockholders, restrict our operations or require us to
relinquish rights to our technologies, intellectual property, potential future revenue streams or drug candidates.
Until such time, if ever, as we can earn substantial revenue, we expect to finance our cash needs through a
combination of equity offerings, debt financings and license and partnership agreements. To the extent that we raise
additional capital through the sale of equity securities or convertible debt securities, your ownership interest will be diluted,
and the terms of these securities may include liquidation or other preferences that adversely affect your rights as a common
stockholder. Debt financing and preferred equity financing, if available, may involve agreements that include covenants
limiting or restricting our ability to take specific actions, such as incurring additional debt, making capital expenditures or
declaring dividends.
If we raise additional funds through partnerships, strategic alliances or marketing, distribution or licensing
arrangements with potential third-party partners, we may be required to relinquish valuable rights to our technologies,
intellectual property, potential future revenue streams, or drug candidates or grant licenses on terms that may not be
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favorable to us. If we are unable to raise additional funds through equity or debt financings or other arrangements with
third parties when needed, we may be required to delay, limit, reduce or terminate our drug development efforts or grant
rights to third parties to develop technologies, intellectual property, or drug candidates that we would otherwise prefer to
develop ourselves.
We have a limited history as a clinical-stage biopharmaceutical company developing and partnering our drug
candidates, which may make it difficult to evaluate the success of our business to date and to assess our future viability.
Our operations over the last several years have been largely focused on raising capital, undertaking preclinical
studies and conducting clinical trials, and acquiring new drug candidates and related intellectual property. We have had
limited time to demonstrate our ability to successfully develop, manufacture and identify and consummate transactions
with third-party partners to further develop, obtain marketing approval for and/or commercialize our drug candidates.
Consequently, any predictions you make about our future success or viability may not be as accurate as they could be if we
had a longer history of being a clinical-stage biopharmaceutical company focused on developing and partnering drugs. We
may also encounter unforeseen expenses, difficulties, complications, delays and other known or unknown factors in
achieving our business objectives.
Our business has been adversely impacted and could continue to be adversely affected by the evolving and
ongoing COVID-19 global pandemic in regions where we or third parties on which we rely have manufacturing
facilities, clinical trial sites or other business operations. The COVID-19 pandemic could adversely affect our
operations, including at our headquarters and at our clinical trial sites, as well as the business or operations of our
manufacturers, contract research organizations or other third parties with whom we conduct business.
Our business has been adversely affected by the effects of the COVID-19 pandemic, which has resulted in travel
and other restrictions in order to reduce the spread of the disease, which, among other things, direct businesses and
governmental agencies to cease non-essential operations at physical locations, prohibit certain non-essential gatherings,
and order cessation of non-essential travel. In response to these public health directives and orders, we have implemented a
virtual operations strategy, including teleworking and other alternative work arrangements for all employees. The effects of
our alternative work arrangement policies may negatively impact productivity, disrupt our business and delay our
preclinical drug development and clinical trials and timelines, the magnitude of which will depend, in part, on the length
and severity of the restrictions and other limitations on our ability to conduct our business in the ordinary course. These
and similar, and perhaps more severe, disruptions in our operations could negatively impact our business, operating results
and financial condition.
Quarantines, executive and similar government orders, and business shutdowns, or the perception that such orders,
shutdowns or other restrictions on the conduct of business operations could occur, related to COVID-19 or other infectious
diseases could impact personnel at third-party manufacturing facilities in the United States and other countries, or the
availability or cost of materials, which would disrupt our supply chain. Some of our third-party manufacturers which we
use for the supply of materials for our drug candidates or other materials necessary to manufacture drug product to conduct
preclinical studies and clinical trials are located in countries affected by COVID-19, and should they experience
disruptions, such as temporary closures or suspension of services, we would likely experience delays in advancing these
studies and trials.
In addition, our clinical trials have been and may continue to be affected by the COVID-19 pandemic. Clinical site
initiation and patient enrollment may be delayed due to prioritization of hospital resources toward the COVID-19
pandemic. Some subjects may not be able to comply with clinical trial protocols if quarantines impede patient movement
or interrupt healthcare services. Similarly, our ability to recruit and retain subjects and principal investigators and site staff
who, as healthcare providers, may have heightened exposure to COVID-19, may adversely impact our clinical trial
operations. For example, due to the COVID-19 pandemic subject enrollment in our ATI-450-CAPS-201 trial was paused as
a result of which, among other reasons, we have decided to focus our efforts and resources on other immuno-inflammatory
diseases.
The spread of COVID-19, which has caused a broad impact globally, may materially affect us economically.
While the potential economic impact brought by, and the duration of, the COVID-19 pandemic may be difficult to assess or
predict, the widespread pandemic could result in significant disruption of global financial markets, reducing our ability to
access capital, which could in the future negatively affect our liquidity and impact our ability to make scheduled payments
pursuant to our Loan and Security Agreement with Silicon Valley Bank, or SVB. In addition, a recession or
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market correction resulting from the further spread of COVID-19 could materially affect our business and the value of our
common stock.
The global COVID-19 pandemic continues to rapidly evolve. The extent to which the COVID-19 pandemic
impacts our business, our preclinical and clinical development and our regulatory efforts will depend on future
developments that are highly uncertain and cannot be predicted, such as the geographic spread of the disease, the duration
of the outbreak, travel restrictions, quarantines, stay-at-home orders, social distancing requirements and business closures,
and supply chain and other disruptions in the United States and other countries, and the effectiveness of actions taken in the
United States and other countries to contain and treat the disease, including the success of mass vaccination efforts.
Accordingly, we do not yet know the full extent of the impacts on our business, our preclinical and clinical development
and regulatory activities, healthcare systems or the global economy as a whole. However, these impacts could adversely
affect our business, financial condition, results of operations and growth prospects.
In addition, to the extent the ongoing COVID-19 pandemic adversely affects our business and results of
operations, it may also have the effect of heightening many of the other risks and uncertainties described herein.
We may not be able to generate sufficient cash to service our indebtedness, including the Loan and Security
Agreement with SVB.
In March 2020, we entered into the Loan and Security Agreement with SVB, pursuant to which we borrowed
$11.0 million. Our obligations under the Loan and Security Agreement are secured by substantially all of our assets except
for our intellectual property, and restrict us from encumbering our intellectual property without SVB’s prior written
consent. The Loan and Security Agreement contains customary representations, warranties and covenants by us, which
covenants, among other things, limit our ability, subject to specified exceptions, to convey, sell, lease, transfer, assign or
otherwise dispose of our assets; engage in any business other than the businesses currently engaged in by us or reasonably
related thereto; liquidate or dissolve; undergo specified change of control events; create, incur, assume or be liable for
indebtedness; create, incur, allow or suffer any liens on our property; pay dividends and make other restricted payments;
make investments; or enter into any material transactions with our affiliates. Our obligations under the Loan and Security
Agreement are subject to acceleration upon the occurrence of specified events of default, including a material adverse
change in our business, operations or financial condition. We may also enter into other debt agreements in the future which
may contain similar or more restrictive terms.
Our ability to make scheduled monthly payments or to refinance our debt obligations depends on numerous
factors, including the amount of our cash reserves and our actual and projected financial and operating performance. These
amounts and our performance are subject to certain financial and business factors, as well as prevailing economic and
competitive conditions, including the impact of the COVID-19 pandemic, some of which may be beyond our control. We
cannot guarantee that we will maintain a level of cash reserves or cash flows from operating activities sufficient to permit
us to pay the principal, premium, if any, and interest on our existing or future indebtedness. If our cash flows and capital
resources are insufficient to fund our debt service obligations, we may be forced to reduce or delay capital expenditures,
sell assets or operations, seek additional capital or restructure or refinance our indebtedness. We cannot guarantee that we
would be able to take any of these actions, or that these actions would permit us to meet our scheduled debt service
obligations. Failure to comply with the covenants and conditions of the Loan and Security Agreement could result in an
event of default, which could result in an acceleration of amounts due under the Loan and Security Agreement. We may not
have sufficient funds or may be unable to arrange for additional financing to repay our indebtedness or to make any
accelerated payments, and SVB could seek to enforce security interests in the collateral securing such indebtedness, which
would harm our business.
Risks Related to the Development and Potential Commercialization of Our Drug Candidates
If we are unable to successfully develop our drug candidates and to pursue strategic alternatives, including
identifying and consummating transactions with third-party partners, to further develop, obtain marketing approval for
and/or commercialize our drug candidates, or experience significant delays in doing so, our business will be harmed.
We have invested significant efforts and financial resources in the development of our drug candidates and the
identification of potential drug candidates. Our ability to earn substantial revenue from our drug candidates will depend
heavily on our ability to successfully develop and pursue strategic alternatives, including identifying and consummating
transactions with third-party partners, to further develop, obtain marketing approval for and/or commercialize these drug
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candidates. The success of any drug candidates that we develop, including ATI-450, will depend on several factors,
including:
● successful completion of preclinical studies and our clinical trials;
● successful development of manufacturing processes;
● receipt of timely approvals from applicable regulatory authorities;
● the identification and consummation of transactions with third-party partners to further develop, obtain
marketing approval for and/or commercialize our drug candidates;
● the commercial launch of our drug candidates, if approved, by a potential third-party partner;
● our potential third-party partners’ ability to achieve acceptance of our drug candidates, if approved, by
patients, the medical community and third-party payors, and willingness of patients to pay out of pocket for
our drug candidates when third-party payor coverage and reimbursement is limited or unavailable;
● our potential third-party partners’ ability to achieve success in educating physicians and patients about the
benefits, administration and use of our drug candidates, if approved;
● the prevalence and severity of adverse events experienced with our drug candidates;
● the availability, perceived advantages, cost, safety and efficacy of alternative treatments for the proposed
indications of our drug candidates;
● obtaining and maintaining patent, trademark and trade secret protection and regulatory exclusivity for our
drug candidates and otherwise protecting the intellectual property portfolio;
● maintaining compliance with regulatory requirements, including current good manufacturing practices, or
cGMPs;
● our potential third-party partners’ ability to compete effectively with other treatment procedures; and
● our potential third-party partners’ ability to maintain a continued acceptable safety, tolerability and efficacy
profile of our drug candidates following marketing approval.
Whether marketing approval will be granted is unpredictable and depends upon numerous factors, including the
substantial discretion of the regulatory authorities. Our drug candidates’ success in clinical trials will not guarantee
marketing approval. Following submission, the NDA for any drug candidate may not be accepted for substantive review, or
even if it is accepted for substantive review the FDA or other comparable foreign regulatory authorities may require
additional studies or clinical trials, additional data, or additional manufacturing steps, or require other conditions before
they will reconsider or approve the application, which could increase costs and cause delays in the marketing approval
process and which may require the expenditure of additional resources. These delays would also impact our ability to
identify and consummate transactions with third-party partners to further develop, obtain marketing approval for and/or
commercialize our drug candidates. In addition, the FDA or other comparable foreign regulatory authorities may not
consider sufficient any additional required studies, clinical trials, data or information that we perform and complete or
generate, or we may decide to abandon the program.
It is possible that our drug candidates currently in development will never obtain marketing approval. If we do not
achieve one or more of these factors in a timely manner or at all, we could experience significant delays or an inability to
successfully pursue strategic alternatives, including identifying and consummating transactions with third-party partners, to
further develop, obtain marketing approval for and/or commercialize our drug candidates, which would harm our business.
Clinical drug development involves a lengthy and expensive process, with an uncertain outcome. We may incur
additional costs or experience delays in completing, or ultimately be unable to complete, the development of and pursue
strategic alternatives, including identifying and consummating transactions with third-party partners, to further
develop, obtain marketing approval for and/or commercialize our drug candidates.
The risk of failure for our drug candidates is high. It is impossible to predict when or if any of our drug candidates
will prove effective or safe in humans or will receive marketing approval. Before obtaining regulatory approval for the sale
of any drug candidate, we must complete preclinical development and then conduct extensive clinical trials to demonstrate
the safety and efficacy of our drug candidates in humans for use in the target indication. Clinical testing is expensive,
difficult to design and implement, can take many years to complete and is inherently uncertain as to outcome.
A failure of one or more clinical trials can occur at any stage of testing. The outcome of preclinical testing and
early clinical trials may not be predictive of the success of later clinical trials, and interim results of a clinical trial do not
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necessarily predict final results. Moreover, preclinical and clinical data are often susceptible to varying interpretations and
analyses, and many companies that have believed their drug candidates performed satisfactorily in preclinical studies and
clinical trials have nonetheless failed to obtain marketing approval of their drugs.
We may experience numerous unforeseen events during or as a result of clinical trials that could delay or prevent
our ability to pursue strategic alternatives, including identifying and consummating transactions with third-party partners,
to further develop, obtain marketing approval for and/or commercialize our drug candidates, including:
● regulators or IRBs may not authorize us or our investigators to commence a clinical trial or conduct a clinical
trial at a prospective trial site;
● we may experience delays in reaching, or fail to reach, agreement on acceptable clinical trial contracts or
clinical trial protocols with prospective trial sites or prospective contract research organizations, or CROs,
the terms of which can be subject to extensive negotiation and may vary significantly among different CROs
and trial sites;
● clinical trials of our drug candidates may produce negative or inconclusive results, including failure to
demonstrate statistical significance, and we may decide, or regulators may require us, to conduct additional
clinical trials or abandon drug development programs;
● the number of patients required for clinical trials of our drug candidates may be larger than we anticipate,
enrollment in these clinical trials may be slower than we anticipate, or participants may drop out of these
clinical trials or fail to return for post-treatment follow-up at a higher rate than we anticipate;
● the COVID-19 pandemic may impact the recruitment, enrollment, conduct and timing of our clinical trials;
● our drug candidates may have undesirable side effects or other unexpected characteristics, causing us or our
investigators, regulators or IRBs to suspend or terminate the trials;
● our third-party contractors may fail to comply with regulatory requirements or meet their contractual
obligations to us in a timely manner, or at all;
● regulators or IRBs may require that we or our investigators suspend or terminate clinical development for
various reasons, including noncompliance with regulatory requirements or a finding that the participants are
being exposed to unacceptable health risks;
● the cost of clinical trials of our drug candidates may be greater than we anticipate; and
● the supply or quality of our drug candidates or other materials necessary to conduct clinical trials of our drug
candidates may be insufficient or inadequate.
We could also encounter delays if a clinical trial is suspended or terminated by us, by the IRBs of the institutions
in which such trials are being conducted, by a data safety monitoring board for such trial or by the FDA or other regulatory
authorities. Such authorities may impose such a suspension or termination due to a number of factors, including failure to
conduct the clinical trial in accordance with regulatory requirements or our clinical protocols, inspection of the clinical trial
operations or trial site by the FDA or other regulatory authorities resulting in the imposition of a clinical hold, unforeseen
safety issues or adverse side effects, failure to demonstrate a benefit from using a drug, changes in governmental
regulations or administrative actions or lack of adequate funding to continue the clinical trial. If we experience delays in
the completion of, or termination of, any clinical trial of our drug candidates, our costs will increase, our drug candidate
development process will be slowed, the commercial prospects of our drug candidates will be harmed, and our ability to
pursue strategic alternatives, including identifying and consummating transactions with third-party partners, to further
develop, obtain marketing approval for and/or commercialize our drug candidates will be delayed. Any of these
occurrences may harm our business, financial condition and prospects significantly. In addition, many of the factors that
cause, or lead to, a delay in the commencement or completion of clinical trials may also ultimately lead to the denial of
marketing approval of our drug candidates. If we are required to conduct additional clinical trials or other testing of our
drug candidates beyond those that we currently contemplate, if we are unable to successfully complete clinical trials of our
drug candidates or other testing, if the results of these trials or tests are not favorable or if there are safety concerns, we
may not be able to pursue strategic alternatives, including identifying and consummating transactions with third-party
partners, to further develop, obtain marketing approval for and/or commercialize our drug candidates, and our potential
third-party partners may:
● be delayed in obtaining marketing approval for our drug candidates;
● not obtain marketing approval at all;
● obtain marketing approval for indications or patient populations that are not as broad as intended or desired;
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● obtain marketing approval with labeling that includes significant use or distribution restrictions or safety
warnings;
● be subject to additional post-marketing testing requirements; or
● have the drug removed from the market after obtaining marketing approval.
Our drug development costs will also increase if we experience delays in testing. We do not know whether any of
our preclinical studies or clinical trials will begin as planned, will need to be restructured or will be completed on schedule,
or at all. Significant preclinical study or clinical trial delays also could shorten any periods during which our potential
third-party partners may have the exclusive right to commercialize our drug candidates or allow competitors to bring drugs
to market before such third-party partners do, which would impact our ability to successfully identify and consummate
transactions with third-party partners to further develop, obtain marketing approval for and/or commercialize our drug
candidates.
If we experience delays or difficulties in the enrollment of subjects in clinical trials, our ability to pursue
strategic alternatives, including identifying and consummating transactions with third-party partners, to further
develop, obtain marketing approval for and/or commercialize our drug candidates could be delayed or prevented.
Successful and timely completion of clinical trials will require that we enroll a sufficient number of subjects.
Subject enrollment, a significant factor in the timing of clinical trials, is affected by many factors including the size and
nature of the patient population. Trials may be subject to delays as a result of subject enrollment taking longer than
anticipated or subject withdrawal, including as a result of factors beyond our control, such as the COVID-19 pandemic. We
may not be able to initiate or continue clinical trials for our drug candidates if we are unable to locate and enroll a sufficient
number of eligible patients to participate in these trials as required by the FDA or similar regulatory authorities outside the
United States. We cannot predict how successful we will be at enrolling subjects in future clinical trials. Subject enrollment
is affected by other factors including:
● the eligibility criteria for the trial in question;
● the perceived risks and benefits of the drug candidate in the trial;
● the availability of drugs approved to treat the disease in the trial;
● the efforts to facilitate timely enrollment in clinical trials;
● the patient referral practices of physicians;
● the ability to monitor patients adequately during and after treatment; and
● the proximity and availability of clinical trial sites for prospective patients.
Our inability to enroll a sufficient number of subjects for clinical trials would result in significant delays and could
require us or them to abandon one or more clinical trials altogether. Enrollment delays in these clinical trials may result in
increased development costs for our drug candidates, which would cause the value of our company to decline and limit our
ability to obtain additional financing. Furthermore, we rely on and expect to continue to rely on CROs and clinical trial
sites to ensure the proper and timely conduct of our clinical trials and we will have limited influence over their
performance. Any delays in completing clinical trials would delay or prevent our ability to pursue strategic alternatives,
including identifying and consummating transactions with third-party partners, to further develop, obtain marketing
approval for and/or commercialize our drug candidates.
Our clinical trials may fail to demonstrate the safety and efficacy of our drug candidates, or serious adverse or
unacceptable side effects may be identified during the development of our drug candidates, which could increase our
costs or necessitate the abandonment or limitation of the development of our drug candidates or prevent or delay our
ability to pursue strategic alternatives, including identifying and consummating transactions with third-party partners,
to further develop, obtain marketing approval for and/or commercialize our drug candidates.
If our drug candidates are associated with side effects in clinical trials or have characteristics that are unexpected,
our costs could increase or we may need to abandon their development or limit development to more narrow uses in which
the side effects or other characteristics are less prevalent, less severe or more acceptable from a risk-benefit perspective.
The FDA or an IRB may also require that we suspend, discontinue, or limit our clinical trials based on safety information.
Such findings could further result in regulatory authorities failing to provide marketing authorization for our drug
candidates. Many drug candidates that initially showed promise in early stage testing have later been found to cause side
effects that prevented further development of the drug candidate.
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Before any potential third-party partners can obtain marketing approvals for the commercial sale of our drug
candidates, we must demonstrate through lengthy, complex and expensive preclinical testing and clinical trials that our
drug candidates are both safe and effective for use in each target indication, and failures can occur at any stage of testing.
Clinical trials often fail to demonstrate safety and efficacy of the drug candidate studied for the target indication.
Additionally, if we or others identify undesirable side effects caused by our drugs, a number of potentially
significant negative consequences could result, including:
● we may need to abandon the development or limit the further development of our drug candidates, including
in various populations and for certain indications;
● regulatory authorities may withdraw approval to market such product;
● regulatory authorities may require additional warnings on the labels;
● a medication guide outlining the risks of such side effects for distribution to patients may be required;
● we could be sued and held liable for harm caused to patients;
● our reputation and physician or patient acceptance of our drug candidates, if approved, may suffer; and
● our ability to pursue strategic alternatives, including identifying and consummating transactions with third-
party partners, to further develop, obtain marketing approval for and/or commercialize our drug candidates
would be harmed.
For example, we are supporting an investigator-initiated trial of ATI-450 in hospitalized patients with COVID-19.
Although the COVID-19 trial is not sponsored by us, the use of ATI-450 in a hospitalized and severely ill patient
population may be associated with adverse events and risks that could jeopardize our development of ATI-450 in other
populations and indications, including our trials in subjects with moderate to severe rheumatoid arthritis.
Any of these events could prevent us from pursuing strategic alternatives, including identifying and
consummating transactions with third-party partners, to further develop, obtain marketing approval for and/or
commercialize the particular drug candidate and could significantly harm our business, results of operations and prospects.
Interim, topline and preliminary data from our clinical trials that we announce or publish from time to time
may change as more subject data become available and are subject to audit and verification procedures that could result
in material changes in the final data.
From time to time, we may publicly disclose interim, topline or preliminary data from our clinical trials, such as
the preliminary topline results from our Phase 2a trial of ATI-450 in subjects with moderate to severe rheumatoid arthritis,
which are based on a preliminary analysis of then-available data, and the results and related findings and conclusions are
subject to change following a full analysis of all data related to the particular trial. We also make assumptions, estimations,
calculations and conclusions as part of our analyses of data, and we may not have received or had the opportunity to fully
and carefully evaluate all data. In addition, we may report preliminary analyses of only certain endpoints rather than all
endpoints. As a result, the interim, topline or preliminary results that we report may differ from future results of the same
trials, or different conclusions or considerations may qualify such results, once additional data have been received and fully
evaluated. Topline data also remain subject to audit and verification procedures that may result in the final data being
materially different from the preliminary data we previously published. As a result, interim, topline and preliminary data
should be viewed with caution until the final data are available. We may also disclose interim data from our clinical trials.
Interim data from clinical trials that we may complete are subject to the risk that one or more of the clinical outcomes may
materially change as subject enrollment continues and more subject data become available. Adverse differences between
interim, topline or preliminary data and final data could significantly harm our reputation and business prospects. Further,
disclosure of interim, topline or preliminary data by us or by our competitors could result in volatility in the price of our
common stock.
Further, others, including regulatory agencies, may not accept or agree with our assumptions, estimates,
calculations, conclusions or analyses or may interpret or weigh the importance of data differently, which could impact the
potential of the particular program, the likelihood of marketing approval or commercialization of the particular drug
candidate, any approved product, and our company in general. In addition, the information we choose to publicly disclose
regarding a particular study or clinical trial is derived from information that is typically extensive, and you or others may
not agree with what we determine is material or otherwise appropriate information to include in our disclosure, and any
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information we determine not to disclose may ultimately be deemed significant with respect to future decisions,
conclusions, views, activities or otherwise regarding a particular program, drug candidate or our business.
If the interim, topline or preliminary data that we report differ from actual results, or if others, including
regulatory authorities, disagree with the conclusions reached, our ability to pursue strategic alternatives, including
identifying and consummating transactions with third-party partners, to further develop, obtain marketing approval for
and/or commercialize our drug candidates may be harmed, which could harm our business, operating results, prospects or
financial condition.
Changes in methods of drug candidate manufacturing or formulation may result in additional costs or delay.
As drug candidates are developed through preclinical studies to late-stage clinical trials towards approval and
commercialization, it is common that various aspects of the development program, such as manufacturing methods and
formulation, are altered along the way in an effort to optimize processes and results. Such changes carry the risk that they
will not achieve these intended objectives, and may also require additional testing, FDA notification or FDA
approval. Any of these changes could cause our drug candidates to perform differently and affect the results of planned
clinical trials or other future clinical trials conducted with the altered materials. This could delay completion of clinical
trials, require the conduct of bridging clinical trials or the repetition of one or more clinical trials, increase clinical trial
costs, delay approval of our drug candidates and jeopardize our ability to pursue strategic alternatives, including identifying
and consummating transactions with third-party partners, to further develop, obtain marketing approval for and/or
commercialize our drug candidates.
We may not be successful in our efforts to increase our pipeline of drug candidates, including by in-licensing
or acquiring additional drug candidates.
A key element of our strategy is to build and expand our pipeline of drug candidates. To build our pipeline, we
may seek to in-license or acquire additional drug candidates. We may not be able to identify or develop drug candidates
that are safe, tolerable and effective. Even if we are successful in continuing to build our pipeline, the potential drug
candidates that we develop, in-license or acquire may not be suitable for clinical development, including as a result of
being shown to have harmful side effects or other characteristics that indicate that they are unlikely to be drugs that will
receive marketing approval and achieve market acceptance.
We may expend our limited resources to pursue a particular drug candidate or indication and fail to capitalize
on drug candidates or indications that may be more profitable or for which there is a greater likelihood of success.
Because we have limited financial and management resources, we focus on development programs and drug
candidates that we identify for specific indications. As such, we are currently primarily focused on the development of
ATI-450 as a potential treatment for moderate to severe rheumatoid arthritis and potentially other immuno-inflammatory
diseases and ATI-1777 as a potential treatment for moderate to severe atopic dermatitis. As a result, we may forego or
delay pursuit of opportunities with other drug candidates or for other indications that later prove to have greater
commercial potential. Our resource allocation decisions may cause us to fail to capitalize on viable commercial drugs or
profitable market opportunities. Our spending on current and future development programs and drug candidates for specific
indications may not yield any commercially viable drugs. If we do not accurately evaluate the commercial potential or
target market for a particular drug candidate, we may relinquish valuable rights to that drug candidate through partnerships,
licensing or other arrangements in cases in which it would have been more advantageous for us to retain sole development
and commercialization rights to such drug candidate.
For any of our drug candidates that receive marketing approval, our potential third-party partners may fail to
achieve the degree of market acceptance by physicians, patients, third-party payors and others in the medical
community necessary for commercial success.
For any of our drug candidates that receive marketing approval, our potential third-party partners may fail to gain
sufficient market acceptance by physicians, patients, third-party payors and others in the medical community. If such third-
party partners fail to obtain an adequate level of acceptance for our drug candidates, we may not earn significant revenue
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and we may not become profitable. The degree of market acceptance of any drug candidate, if approved, will depend on a
number of factors, including:
● the efficacy, safety and potential advantages compared to alternative treatments;
● our potential third-party partners’ ability to offer the products for sale at competitive prices;
● the convenience and ease of administration compared to alternative treatments;
● the willingness of the target patient population to try new treatments and of physicians to prescribe these
treatments;
● the ability of our potential third-party partners to retain a sales force;
● the strength of our potential third-party partners’ marketing and distribution support;
● the availability of third-party payor coverage and adequate reimbursement or the willingness of patients to
pay for these products;
● the prevalence and severity of any side effects; and
● any restrictions on the use of our products together with other medications.
We face substantial competition, which may result in others discovering, developing or commercializing drugs
before or more successfully than we do.
The development and commercialization of new drugs is highly competitive. We will face competition with
respect to any drug candidates that we may seek to develop or through our potential third-party partners, commercialize, in
the future, from many different sources, including major pharmaceutical, biotechnology and specialty pharmaceutical
companies, academic institutions and governmental agencies and public and private research institutions.
With respect to ATI-450 as a potential treatment for moderate to severe rheumatoid arthritis, there are several
different types of therapies in the rheumatoid arthritis market. Medications for the treatment of rheumatoid arthritis
currently fall into two categories: drugs that ease symptoms such as nonsteroidal anti-inflammatory drugs and drugs that
slow disease activity. Drugs that slow disease activity include corticosteroids and DMARDs. Disease-modifying drugs
include conventional DMARDs such as methotrexate, sulfasalazine, leflunomide, hydroxychloroquine, biologic DMARDs
(monoclonal antibodies which inhibit targets such as TNF, IL1, IL6 and costimulatory signaling mechanisms), and targeted
synthetic DMARDs such as JAK inhibitors. These types of drugs are produced and sold by large pharmaceutical
companies, including AbbVie, Amgen, Bristol Myers Squibb, Eli Lilly, Johnson & Johnson, Merck, Pfizer, and Roche,
among others. In addition, we are aware of a number of companies developing and conducting clinical trials for
investigational drug candidates, including biosimilars, that, if approved, could compete with ATI-450, if approved, for the
treatment of rheumatoid arthritis.
With respect to ATI-1777 as a potential treatment for moderate to severe atopic dermatitis, there are several
different types of therapies in the atopic dermatitis market, such as biologics, oral and topical corticosteroids, injectable and
oral methotrexate products, oral and topical calcineurin inhibitors, oral mycophenolate products, other JAK inhibitors,
other oral antibiotics and antihistamines and phototherapy. There are also several prescription, non-prescription and OTC
topical products, including PDE4 inhibitors, utilized to treat atopic dermatitis. These types of drugs are produced and sold
by large pharmaceutical companies, including Sanofi and Regeneron Pharmaceuticals, Inc., and Pfizer. In addition, we are
aware of a number of companies including large pharmaceutical companies, such as AbbVie, Eli Lilly, Novartis, Incyte,
Pfizer and LEO Pharma A/S developing and conducting clinical trials for investigational drug candidates, that, if approved,
could compete with ATI-1777, if approved, for the treatment of atopic dermatitis.
The commercial opportunity for our drug candidates, if approved, could be reduced or eliminated if our
competitors develop and commercialize drugs that are safer, more effective, have fewer or less severe side effects, are more
convenient or are less expensive than a drug that we may develop. Our competitors also may obtain FDA or other
regulatory approval for their drugs more rapidly than our potential third-party partners’ may obtain approval for our drug
candidates, which could result in our competitors establishing a strong market position before our drug candidates are able
to enter the market.
Many of the companies against which we are competing, or against which we may compete in the future, have
significantly greater financial resources and expertise in research and development, manufacturing, and preclinical and
clinical development than we do. Mergers and acquisitions in the pharmaceutical and biotechnology industries may result
in even more resources being concentrated among a smaller number of our competitors. Smaller or early-stage companies
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may also prove to be significant competitors, particularly through collaborative arrangements with large and established
companies. These competitors also compete with us in recruiting and retaining qualified scientific and management
personnel and establishing clinical trial sites and patient registration for clinical trials, as well as in acquiring technologies
complementary to, or that may be necessary for, our development programs.
The success of our drug candidates, if approved, will depend significantly on coverage and adequate
reimbursement or the willingness of patients to pay for these products.
We believe the success of our drug candidates, if approved, will depend on obtaining and maintaining coverage
and adequate reimbursement as a prescription treatment or in the absence of coverage and adequate reimbursement, on the
extent to which patients will be willing to pay out of pocket for these prescription drug products.
Third-party payors determine which prescription drug products they will cover and establish reimbursement
levels. Reimbursement by a third-party payor may depend upon a number of factors, including: the third-party payor’s
determination that a product is safe, effective, and medically necessary; appropriate for the specific patient; cost-effective;
supported by peer-reviewed medical journals or current clinical practice guidelines; and whether there are competitive
products, either branded or generic, and the pricing of those products. Many private third-party payors, such as managed
care plans, manage access to drug products’ coverage partly to control costs for their plans, and may use drug formularies
and medical policies to limit their exposure. Obtaining and maintaining favorable reimbursement can be a time-consuming
and expensive process, and our potential third-party partners may not be able to negotiate or continue to negotiate
reimbursement or pricing terms for our products with third-party payors at levels that are profitable to us, or at all.
In addition to uncertainties surrounding coverage policies, there are periodic changes to reimbursement. Third-
party payors regularly update reimbursement amounts and also from time to time revise the methodologies used to
determine reimbursement amounts. Accordingly, these updates could impact the demand for our drug candidates, if
approved. Our drug candidates, if approved, may not be considered cost effective, and government and third-party private
health insurance coverage and reimbursement may not be available to patients or sufficient to allow our potential third-
party partners to sell our drug candidates, if approved, on a competitive and profitable basis. Our results of operations
could be adversely affected by the Affordable Care Act and by other health care legislative reforms that may be enacted or
adopted in the future. In addition, increasing emphasis on managed care in the United States will continue to put pressure
on the pricing of pharmaceutical products. Cost control initiatives could decrease the price that our potential third-party
partners could receive for any of our drug candidates, if approved, and could adversely affect our profitability. We cannot
predict how pending and future health care legislation will impact our business, and any changes in coverage and
reimbursement that further restricts coverage of our drug candidates could harm our business.
Foreign governments also have their own healthcare reimbursement systems, which vary significantly by country
and region, and we cannot be sure that coverage and adequate reimbursement will be made available with respect to our
drug candidates, if approved, under any foreign reimbursement system. In some foreign countries, including major
markets in the European Union and Japan, the pricing of prescription pharmaceuticals is subject to governmental
control. In these countries, pricing negotiations with governmental authorities can take up to 12 months or longer after the
receipt of regulatory marketing approval for a product. To obtain reimbursement or pricing approval in some countries, we
may be required to conduct a pharmacoeconomic study that compares the cost-effectiveness of our drug candidate to other
available therapies. Such pharmacoeconomic studies can be costly and the results uncertain. Our business could be
harmed if reimbursement of our drug candidates, if approved, is unavailable or limited in scope or amount or if pricing is
set at unsatisfactory levels.
Product liability lawsuits against us could cause us to incur substantial liabilities and to limit
commercialization of any of our drug candidates that we may develop and are commercialized by our potential third-
party partners or impact any commercial products that we have previously sold or are being sold by third-party partners.
We face an inherent risk of product liability exposure related to the testing of our drug candidates in human
clinical trials and an even greater risk relating to any of our commercial products that we have previously sold or are being
sold by third-party partners. If we cannot successfully defend ourselves against claims that our commercial products that
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we have previously sold or are being sold by third-party partners, or drug candidates, caused injuries, we will incur
substantial liabilities. Regardless of merit or eventual outcome, product liability claims may result in:
● decreased demand for any drug candidates that we may develop and, if approved, are commercialized by our
potential third-party partners;
● injury to our reputation and significant negative media attention;
● withdrawal of clinical trial participants;
● significant costs to defend the related litigation;
● substantial monetary awards paid to trial participants or patients;
● loss of revenue;
● reduced resources of our management to pursue our business strategy; and
● our inability to pursue strategic alternatives, including identifying and consummating transactions with third-
party partners, to further develop, obtain marketing approval for and/or commercialize our drug candidates.
We currently hold $10 million in product liability insurance coverage in the aggregate, with a per incident limit of
$10 million, which may not be adequate to cover all liabilities that we may incur. Insurance coverage is increasingly
expensive. We may need to increase our insurance coverage and we may not be able to maintain insurance coverage at a
reasonable cost or in an amount adequate to satisfy any liability that may arise.
Risks Related to Our Dependence on Third Parties
We rely on third parties to conduct clinical trials for our drug candidates, and those third parties may not
perform satisfactorily, including failing to meet deadlines for the completion of such trials.
We engage CROs to conduct clinical trials of our drug candidates. We expect to continue to rely on third parties,
such as clinical data management organizations, medical institutions and clinical investigators, to conduct those clinical
trials. If any of our relationships with these third parties terminate, we may not be able to timely enter into arrangements
with alternative third parties or to do so on commercially reasonable terms, if at all. In addition, any third parties
conducting our clinical trials will not be our employees, and except for remedies available to us under our agreements with
such third parties, we cannot control whether or not they devote sufficient time and resources to our clinical programs. If
these third parties do not successfully carry out their contractual duties or obligations or meet expected deadlines, if they
need to be replaced or if the quality or accuracy of the clinical data they obtain is compromised due to the failure to adhere
to our clinical protocols, regulatory requirements or for other reasons, our clinical trials may be extended, delayed or
terminated and we may not be able to identify and consummate transactions with third-party partners to further develop,
obtain marketing approval for and/or commercialize our drug candidates. Consequently, our results of operations and the
commercial prospects for our drug candidates would be harmed, our costs could increase substantially and our ability to
earn revenue from those partnerships could be delayed significantly.
Switching or adding CROs involves substantial cost and requires management time and focus. In addition, there is
a natural transition period when a new CRO commences work. As a result, delays occur, which can materially impact our
ability to meet our desired clinical development timelines. Though we intend to carefully manage our relationships with
our CROs, there can be no assurance that we will not encounter challenges or delays in the future or that these delays or
challenges will not have a material adverse impact on our business, financial condition and prospects.
We rely on these parties for execution of our preclinical studies and clinical trials, and generally do not control
their activities. Our reliance on these third parties for research and development activities will reduce our control over these
activities but will not relieve us of our responsibilities. For example, we will remain responsible for ensuring that each of
our clinical trials is conducted in accordance with the general investigational plan and protocols for the trial. Moreover, the
FDA requires us to comply with standards, commonly referred to as good clinical practices, or GCPs, for conducting,
recording and reporting the results of clinical trials to assure that data and reported results are credible and accurate and
that the rights, integrity and confidentiality of trial participants are protected. We also are required to register ongoing
clinical trials and post the results of completed clinical trials on a government-sponsored database, ClinicalTrials.gov,
within specified timeframes. Failure to do so can result in fines, adverse publicity and civil and criminal sanctions. If we or
any of our CROs fail to comply with applicable GCPs, the clinical data generated in our clinical trials may be deemed
unreliable and the FDA or comparable foreign regulatory authorities may require us to perform additional clinical trials
before approving marketing applications. We cannot assure you that upon inspection by a given regulatory
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authority, such regulatory authority will determine that any of our clinical trials complies with GCP regulations. In
addition, our clinical trials must be conducted with drug product produced under cGMP regulations. Our failure to comply
with these regulations may require us to repeat clinical trials, which would delay the marketing approval process for our
potential third-party partners.
We also rely on other third parties to store and distribute drug supplies for our clinical trials. Any performance
failure on the part of our distributors could delay clinical development or marketing approval of our drug candidates or
commercialization of our drug candidates, if approved, producing additional losses and depriving us of potential revenue.
We contract with third parties for the manufacture and supply of our drug candidates for preclinical and
clinical testing. This reliance on third parties increases the risk that we will not have sufficient quantities of our drug
candidates or such quantities at an acceptable cost, which could delay, prevent or impair our development efforts.
We do not have any manufacturing facilities. We currently rely, and expect to continue to rely, on third parties for
the manufacture and supply of our drug candidates for preclinical and clinical testing. This reliance on third parties
increases the risk that we will not have sufficient quantities of our drug candidates at an acceptable cost and/or quality,
which could delay, prevent or impair our ability to timely conduct our clinical trials or our other development efforts.
The facilities used by our contract manufacturers to manufacture our drug candidates must be approved by the
FDA or comparable foreign regulatory authorities pursuant to inspections that will be conducted after the NDA or
comparable marketing application is submitted to the FDA or other regulatory authority. We do not have control over a
supplier’s or manufacturer’s compliance with laws, regulations and applicable cGMP standards and other laws and
regulations, such as those related to environmental health and safety matters. If our contract manufacturers cannot
successfully manufacture material that conforms to our specifications and the strict regulatory requirements of the FDA or
others, they will not be able to secure and maintain regulatory approval for their manufacturing facilities. In addition, we
have no control over the ability of our contract manufacturers to maintain adequate quality control, quality assurance and
qualified personnel. If the FDA or a comparable foreign regulatory authority does not approve these facilities for the
manufacture of our drug candidates or if it withdraws any such approval in the future, we may need to find alternative
manufacturing facilities, which could significantly impact our ability to develop, and identify and consummate transactions
with third-party partners to further develop, obtain marketing approval for and/or commercialize, our drug candidates.
We may be unable to establish any agreements with future third-party manufacturers or do so on acceptable terms.
Even if we are able to establish agreements with third-party manufacturers, reliance on third-party manufacturers entails
additional risks, including:
● reliance on the third party for regulatory compliance and quality assurance;
● the possible breach of the manufacturing agreement by the third party;
● the possible misappropriation of our proprietary information, including our trade secrets and know-how;
● the possible increase in costs by our third-party suppliers for the active pharmaceutical ingredients for our
drug candidates; and
● the possible termination or nonrenewal of the agreement by the third party at a time that is costly or
inconvenient for us.
Third-party manufacturers may not be able to comply with cGMP regulations or similar regulatory requirements
outside the United States. Our failure, or the failure of our third-party manufacturers, to comply with applicable regulations
could result in sanctions being imposed on us, including clinical holds, fines, injunctions, civil penalties, delays, suspension
or withdrawal of approvals, license revocation, seizures or recalls of products, operating restrictions and criminal
prosecutions, any of which could significantly and adversely affect supplies of our drug candidates.
Our drug candidates may compete with other products and drug candidates for access to manufacturing facilities.
There are a limited number of manufacturers that operate under cGMP regulations and that might be capable of
manufacturing for us. Any performance failure on the part of our existing or future manufacturers could delay clinical
development or marketing approval of our drug candidates.
If our current contract manufacturers cannot perform as agreed, we may be required to replace such
manufacturers. We may incur added costs and delays in identifying and qualifying any such replacement. We do not
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currently have arrangements in place for redundant supply or a second source for the active pharmaceutical ingredients
and/or drug product for our drug candidates.
We expect to continue to depend on third-party contract manufacturers for the foreseeable future. Our current and
anticipated future dependence upon others for the manufacture of our drug candidates may adversely affect our future
profit margins and our ability to pursue strategic alternatives, including identifying and consummating transactions with
third-party partners, to further develop, obtain marketing approval for and/or commercialize our drug candidates on a
timely and competitive basis.
We intend to pursue strategic alternatives, including identifying and consummating transactions with third-
party partners, to further develop, obtain marketing approval for and/or commercialize our drug candidates. If those
arrangements are not successful, we may not be able to capitalize on the market potential of these drug candidates.
We intend to pursue strategic alternatives, including identifying and consummating transactions with third-party
partners, to further develop, obtain marketing approval for and/or commercialize our drug candidates. Our likely partners
for any such arrangements include large and mid-size pharmaceutical companies, regional and national pharmaceutical
companies and biotechnology companies. If we do enter into any such arrangements with any third parties, we will likely
have limited control over the amount and timing of resources that our partners dedicate to the development or
commercialization of our drug candidates. Our ability to earn revenue from these arrangements will depend on our
partners’ abilities to successfully perform the functions assigned to them in these arrangements.
Partnerships involving our drug candidates would pose the following risks to us:
● partners have significant discretion in determining the efforts and resources that they will apply to these
arrangements;
● partners may not perform their obligations as expected;
● partners may not pursue development, marketing approval or commercialization of any drug candidates that
achieve marketing approval or may elect not to continue or renew development or commercialization
programs based on clinical trial results, changes in the partners’ strategic focus or available funding, or
external factors, such as an acquisition, that divert resources or create competing priorities;
● partners may delay clinical trials, provide insufficient funding for a clinical trial program, stop a clinical trial
or abandon a drug candidate, repeat or conduct new clinical trials or require a new formulation of a drug
candidate for clinical testing;
● partners could independently develop, or develop with third parties, products that compete directly or
indirectly with our drug candidates if the partners believe that competitive products are more likely to be
successfully developed or can be commercialized under terms that are more economically attractive than
ours;
● drug candidates discovered in collaboration with us may be viewed by our collaborators as competitive with
their own products or drug candidates, which may cause our partners to cease to devote resources to the
development and/or commercialization of our drug candidates, if approved;
● a partner with marketing and distribution rights to one or more of our drug candidates that achieve marketing
approval may not commit sufficient resources to the marketing and distribution of such drug candidates;
● disagreements with partners, including disagreements over proprietary rights, contract interpretation or the
preferred course of development or commercialization, might cause delays or termination of the research,
development or commercialization of drug candidates, might lead to additional responsibilities for us with
respect to drug candidates, or might result in litigation or arbitration, any of which would be time-consuming
and expensive;
● partners may not properly maintain or defend our or their intellectual property rights or may use our or their
proprietary information in such a way as to invite litigation that could jeopardize or invalidate such
intellectual property or proprietary information or expose us to potential litigation;
● partners may infringe the intellectual property rights of third parties, which may expose us to litigation and
potential liability; and
● partnerships may be terminated for the convenience of the partner and, if terminated, we could be required to
raise additional capital to pursue further development and/or commercialization of the applicable drug
candidates.
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Partnership agreements may not lead to development, marketing approval or commercialization of drug
candidates in the most efficient manner or at all. If a present or future partner of ours were to be involved in a business
combination, the continued pursuit and emphasis on our drug development or commercialization program could be
delayed, diminished or terminated.
If we are not able to establish partnerships, we may have to alter our development and commercialization
plans.
Our drug development programs for our drug candidates will require substantial additional capital. We intend to
partner with pharmaceutical and biotechnology companies for the further development and/or commercialization of our
drug candidates.
We face significant competition in seeking appropriate partners. Whether we reach a definitive agreement for a
partnership will depend, among other things, upon our assessment of the partner’s resources and expertise, the terms and
conditions of the proposed arrangement and the proposed partner’s evaluation of a number of factors. Those factors may
include the design or results of clinical trials, the likelihood of approval by the FDA or similar regulatory authorities
outside the United States, the potential market for the subject drug candidate, the costs and complexities of manufacturing
and delivering such drug candidate to patients, the potential of competing products, the existence of uncertainty with
respect to our ownership of technology, which can exist if there is a challenge to such ownership without regard to the
merits of the challenge, and industry and market conditions generally. The partner may also consider alternative drug
candidates or technologies for similar indications that may be available to partner on and whether such a partnership could
be more attractive than the one with us for our drug candidate. Partnerships are complex and time-consuming to negotiate
and document. In addition, there have been a significant number of recent business combinations among large
pharmaceutical companies that have resulted in a reduced number of potential future partners.
We may not be able to negotiate partnerships on a timely basis, on acceptable terms, or at all. If we are unable to
do so, we may have to curtail the development of such drug candidate, or reduce or delay its development program or one
or more of our other development programs, or increase our expenditures and undertake development or commercialization
activities at our own expense. If we elect to increase our expenditures to fund development or commercialization activities
on our own, we may need to obtain additional capital, which may not be available to us on acceptable terms or at all. If we
do not have sufficient funds, we may not be able to further develop our drug candidates or bring them to market and
generate revenue.
We may not have access to all information regarding our drug candidates that are subject to partnership
agreements. Consequently, our ability to inform our stockholders about the status of our drug candidates that are
subject to these agreements, and our ability to make business and operational decisions, may be limited.
We may not have access to all information regarding our drug candidates that may become subject to agreements
with partners, including potentially material information about clinical trial design, execution and timing, safety and
efficacy, clinical trial results, regulatory affairs, manufacturing, marketing, sales and other areas known by our potential
partners. In addition, we may have confidentiality obligations under our agreements with such partners. Therefore, our
ability to keep our stockholders informed about the status of our drug candidates will be limited by the degree to which our
partners keep us informed and by the degree to which our partners allow us to disclose information to the public or provide
such information to the public themselves. If our partners do not timely inform us about the status of our drug candidates
that are the subject of the partnership, we may make operational and investment decisions that we would not have made
had we been fully informed, which may have an adverse impact on our business, prospects, financial condition and results
of operations.
Our sublease could terminate if the master lease is terminated for any reason, thus terminating our rights to
our corporate headquarters.
We sublease space for our corporate headquarters. While the term of the sublease extends until October 2023, if
for any reason the master lease is terminated or expires prior to October 2023, our sublease will also automatically
terminate. In such an event, we would need to obtain a new direct lease with the master landlord or negotiate and enter
into a new lease for office space at a different location, which we may not be able to do on commercially reasonable terms,
if at all.
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Risks Related to Our Intellectual Property
If we are unable to obtain and maintain patent protection for our drug candidates, or if the scope of the patent
protection obtained is not sufficiently broad, our competitors could develop and commercialize technology and drugs
similar or identical to ours, and ability to successfully identify a potential third-party partner to commercialize our
technology and drug candidates may be impaired.
Our success depends in large part on our ability to obtain and maintain patent protection in the United States and
other countries with respect to our drug candidates. We seek to protect our proprietary position by filing patent applications
in the United States and abroad related to our drug candidates.
The patent prosecution process is expensive and time-consuming, however, and we may not be able to file and
prosecute all necessary or desirable patent applications at a reasonable cost or in a timely manner. It is also possible that we
will fail to identify patentable aspects of our development output before it is too late to obtain patent protection. We may
not have the right to control the preparation, filing and prosecution of patent applications, or to maintain the rights to
patents licensed to third parties. Therefore, these patents and applications may not be prosecuted and enforced in a manner
consistent with the best interests of our business.
The patent position of biotechnology and pharmaceutical companies generally is highly uncertain, involves
complex legal and factual questions and has in recent years been the subject of much litigation. In addition, the laws of
foreign countries may not protect our rights to the same extent as the laws of the United States or vice versa. For example,
European patent law restricts the patentability of methods of treatment of the human body more than U.S. law does.
Publications of discoveries in the scientific literature often lag behind the actual discoveries, and patent applications in the
United States and other jurisdictions are typically not published until 18 months after filing, or in some cases not at all.
Therefore, we cannot know with certainty whether we or our licensors were the first to make the inventions claimed in our
patents or pending patent applications, or that we or our licensors were the first to file for patent protection of such
inventions. As a result, the issuance, scope, validity, enforceability and commercial value of our patent rights are highly
uncertain. Our pending and future patent applications may not result in patents being issued that protect our technology or
drug candidates, in whole or in part, or which effectively prevent others from commercializing competitive technologies
and drug candidates. Changes in either the patent laws or interpretation of the patent laws in the United States and other
countries may diminish the value of our patents or narrow the scope of our patent protection.
Moreover, we may be subject to a third-party preissuance submission of prior art to the U.S. Patent and Trademark
Office, or USPTO, or become involved in opposition, derivation, reexamination, inter partes review, post-grant review or
interference proceedings challenging our patent rights or the patent rights of others. An adverse determination in any such
submission, proceeding or litigation could reduce the scope of, or invalidate, our patent rights, allow third parties to
commercialize our technology or drug candidates and compete directly with us, without payment to us, or result in the
inability of our potential third-party partners to manufacture or commercialize our drug candidates without infringing third-
party patent rights. In addition, if the breadth or strength of protection provided by our patents and patent applications that
we own or license is threatened, it could dissuade companies from partnering with us to license, develop and/or
commercialize our drug candidates.
Even if our patent applications that we own or license issue as patents, they may not issue in a form that will
provide us with any meaningful protection, prevent competitors from competing with us or our potential third-party
partners or otherwise provide us or our potential third-party partners with any competitive advantage. Competitors may be
able to circumvent our patents by developing similar or alternative technologies or drugs in a non-infringing manner.
In addition, the issuance of a patent is not conclusive as to its inventorship, scope, validity or enforceability, and
our patents may be challenged in the courts or patent offices in the United States and abroad. Such challenges may result in
loss of exclusivity or freedom to operate or in patent claims being narrowed, invalidated or held unenforceable, in whole or
in part, which could limit the ability to stop others from using or commercializing similar or identical technology and drug
candidates, or limit the duration of the patent protection of our technology and drug candidates. Our issued U.S. patents
covering our lead inhibitor of the MK2 signaling pathway, ATI-450, expire in 2034 and other issued patents covering
different MK2 signaling pathway inhibitors expire in 2031 and 2032. We currently do not have any patents issued directed
to our lead “soft” JAK inhibitor, ATI-1777, but any claims that may issue would expire in 2038. Our issued patent covering
other novel “soft” JAK inhibitors expires in 2038. We currently do not have any patents issued directed to our lead ITK
inhibitor, ATI-2138, but any claims that may issue would expire in 2039. Our issued patents covering other
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novel inhibitors of ITK expire between 2035 and 2038. Given the amount of time required for the development, testing and
regulatory review of new drug candidates, patents protecting such candidates might expire before or shortly after such
candidates are commercialized. As a result, our patent portfolio may not provide us or our potential third-party partners
with sufficient rights to exclude others from commercializing drugs similar or identical to ours.
We may become involved in lawsuits to protect or enforce our patents or other intellectual property, which
could be expensive, time-consuming and unsuccessful. Further, our issued patents could be found invalid or
unenforceable if challenged in court.
Competitors may infringe our issued patents or other intellectual property. Our pending applications cannot be
enforced against third parties practicing the technology claimed in such applications unless and until a patent issues from
such applications. To counter infringement or unauthorized use, we may be required to file infringement claims, which can
be expensive and time-consuming. Any claims we assert against perceived infringers could provoke these parties to assert
counterclaims against us alleging that we infringe their patents or that our patents are invalid or unenforceable. Grounds for
a validity challenge could be an alleged failure to meet any of several statutory requirements, including lack of novelty,
obviousness, non-enablement or insufficient written description. Grounds for an unenforceability assertion could be an
allegation that someone connected with prosecution of the patent withheld relevant information from the USPTO or made a
misleading statement during prosecution. Third parties may also raise similar claims before the USPTO, in post-grant
proceedings such as ex parte reexaminations, inter partes review, or post-grant review, or oppositions or similar
administrative proceedings outside the United States, in parallel with litigation or, even outside the context of litigation.
The outcome following legal assertions of invalidity and unenforceability is unpredictable. With respect to the validity
question, for example, we cannot be certain that there is no invalidating prior art of which we and the patent examiner were
unaware during prosecution. If a defendant were to prevail on a legal assertion of invalidity or unenforceability, we would
lose at least part, and perhaps all, of the patent protection on our drug candidates. Such a loss of patent protection would
harm our business.
In such a proceeding, a court or administrative board may decide that a patent of ours is invalid or unenforceable,
in whole or in part, construe the patent’s claims narrowly or refuse to stop the other party from using the technology at
issue on the grounds that our patents do not cover the technology. An adverse result in any such proceeding could put one
or more of our patents at risk of being invalidated or interpreted narrowly. We may find it impractical or undesirable to
enforce our intellectual property against some third parties.
Interference proceedings provoked by third parties or brought by us or declared by the USPTO may be necessary
to determine the priority of inventions with respect to our patents or patent applications. An unfavorable outcome could
require us to cease using the related technology or to attempt to license rights to it from the prevailing party. Our business
could be harmed if the prevailing party does not offer us a license on commercially reasonable terms.
Furthermore, because of the substantial amount of discovery required in connection with intellectual property
litigation, there is a risk that some of our confidential information could be compromised by disclosure during this type of
litigation.
We may not be able to protect our intellectual property rights throughout the world.
Filing, prosecuting and defending patents on our drug candidates in all countries throughout the world would be
prohibitively expensive, and our intellectual property rights in some countries outside the United States can be less
extensive than those in the United States. For example, our lead inhibitor of the MK2 signaling pathway, ATI-450, is
currently covered by patents and applications in the United States, European Union and other foreign markets. We
currently do not have any patents issued directed to our lead “soft” JAK inhibitor, ATI-1777, or our lead ITK inhibitor,
ATI-2138, rather we have pending applications in the United States, European Union and other foreign markets directed to
each of ATI-1777 and ATI-2138.
Many companies have encountered significant problems in protecting and defending intellectual property rights in
foreign jurisdictions. The legal systems of some countries do not favor the enforcement of patents and other intellectual
property protection, which could make it difficult for us to stop the infringement of our patents generally. Proceedings to
enforce our patent rights in foreign jurisdictions could result in substantial costs and divert our efforts and attention from
other aspects of our business, could put our patents at risk of being invalidated or interpreted narrowly and our patent
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applications at risk of not issuing and could provoke third parties to assert claims against us. We may not prevail in any
lawsuits that we initiate, and the damages or other remedies awarded, if any, may not be commercially meaningful.
Many countries, including European Union countries, India, Japan and China, have compulsory licensing laws
under which a patent owner may be compelled under specified circumstances to grant licenses to third parties. In those
countries, we may have limited remedies if patents are infringed or if we are compelled to grant a license to a third party,
which could materially diminish the value of those patents. This could limit our ability to pursue strategic alternatives,
including identifying and consummating transactions with potential third-party partners, to further develop, obtain
marketing approval for and/or commercialize our drug candidates, and consequently our potential revenue opportunities.
Accordingly, our efforts to enforce our intellectual property rights around the world may be inadequate to obtain a
significant commercial advantage from the intellectual property that we develop or license.
We may need to license intellectual property from third parties, and such licenses may not be available or may
not be available on commercially reasonable terms.
A third party may hold intellectual property, including patent rights, that are important or necessary to the
development and/or commercialization of our drug candidates. It may be necessary for our potential third-party partners to
use the patented or proprietary technology of third parties to further develop and/or commercialize our drug candidates. If
our potential third-party partners are not able to obtain a license from these third parties on commercially reasonable terms,
our business could be harmed, possibly materially, and even if they are able to, it may result in the reduction of revenue we
earn from such partner as a result of payment obligations such partner has to the licensor.
Third parties may initiate legal proceedings alleging that we are infringing their intellectual property rights,
the outcome of which would be uncertain and could have a material adverse effect on the success of our business.
Our success depends upon our ability to pursue strategic alternatives, including identifying and consummating
transactions with potential third-party partners, to develop, obtain marketing approval for and/or commercialize our drug
candidates and earn revenue from those partnerships, and for our proprietary technologies to be used without infringing the
proprietary rights of third parties. There is considerable intellectual property litigation in the biotechnology and
pharmaceutical industries. We may become party to, or threatened with, future adversarial proceedings or litigation
regarding intellectual property rights with respect to our drug candidates and technologies, including interference or
derivation proceedings before the USPTO. Numerous U.S. and foreign issued patents and pending patent applications
owned by third parties exist in the fields in which we are developing our drug candidates. Third parties may assert
infringement claims against us based on existing patents or patents that may be granted in the future.
If we or our potential third-party partners are found to infringe a third party’s intellectual property rights, we or
such partners could be required to obtain a license from such third party to continue developing or commercializing our
drug candidates and technology. However, we or our potential third-party partners may not be able to obtain any required
license on commercially reasonable terms or at all. Even if we or our potential third-party partner were able to obtain a
license, it could be non-exclusive, thereby giving competitors access to the same technologies licensed to us or our partner.
Consequently, we or our potential third-party partner could be forced, including by court order, to cease developing or
commercializing the infringing technology or drug candidate. In addition, we or our potential third-party partner could be
found liable for monetary damages, including treble damages and attorneys’ fees if we or such partner are found to have
willfully infringed a patent. A finding of infringement could prevent our potential third-party partners from
commercializing our drug candidates, if approved, or force such partners to cease some of their business operations. In the
event of a successful claim of infringement against us or our potential third-party partners, we or our potential third-party
partners may have to pay substantial damages, including treble damages and attorneys’ fees for willful infringement, pay
royalties, redesign our infringing drug candidate or obtain one or more licenses from third parties, which may be
impossible or require substantial time and monetary expenditure. Claims that we have misappropriated the confidential
information or trade secrets of third parties could have a similar negative impact on our business.
We may be subject to claims by third parties asserting that we, our employees or our licensors have
misappropriated their intellectual property, or claiming ownership of what we regard as our own intellectual property.
Many of our employees and our licensors’ employees were previously employed at other biotechnology or
pharmaceutical companies. Although we and our licensors try to ensure that our employees and our licensors’ employees
do not use the proprietary information or know-how of others in their work for us, we or our licensors may be subject to
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claims that these employees, our licensors or we have used or disclosed intellectual property, including trade secrets or
other proprietary information, of any such employee’s former employer. Litigation may be necessary to defend against
these claims.
In addition, while it is our policy to require our employees and contractors who may be involved in the
development of intellectual property to execute agreements assigning such intellectual property to us, we may be
unsuccessful in executing such an agreement with each party who in fact develops intellectual property that we regard as
our own. Our and their assignment agreements may not be self-executing or may be breached, and we may be forced to
bring claims against third parties, or defend claims they may bring against us, to determine the ownership of what we
regard as our intellectual property.
If we or our licensors fail in prosecuting or defending any such claims, in addition to paying monetary damages,
we may lose valuable intellectual property rights or personnel. Even if we and our licensors are successful in prosecuting or
defending against such claims, litigation could result in substantial costs and be a distraction to management.
Intellectual property litigation could cause us to spend substantial resources and distract our personnel from
their normal responsibilities.
Even if resolved in our favor, litigation or other legal proceedings relating to intellectual property claims may
cause us to incur significant expenses, and could distract our technical and management personnel from their normal
responsibilities. In addition, there could be public announcements of the results of hearings, motions or other interim
proceedings or developments and if securities analysts or investors perceive these results to be negative, it could have a
substantial adverse effect on the price of our common stock. Such litigation or proceedings could substantially increase our
operating losses and reduce the resources available for development activities. We may not have sufficient financial or
other resources to conduct such litigation or proceedings adequately. Some of our competitors may be able to sustain the
costs of such litigation or proceedings more effectively than we can because of their greater financial resources. Some of
our competitors are larger than we are and have substantially greater resources. They are, therefore, likely to be able to
sustain the costs of complex patent litigation longer than we could. Accordingly, despite our efforts, we may not be able to
prevent third parties from infringing upon or misappropriating our intellectual property. Litigation could result in
substantial costs and diversion of management resources, which could harm our business. In addition, the uncertainties
associated with litigation could compromise our ability to compete in the marketplace, including compromising our ability
to raise the funds necessary to continue our clinical trials, continue our internal research programs, or pursue strategic
alternatives, including identifying and consummating transactions with third-party partners, to further develop, obtain
marketing approval for and/or commercialize our drug candidates.
If we are unable to protect the confidentiality of our trade secrets, our business and competitive position would
be harmed.
In addition to seeking and maintaining patents for our drug candidates, we also rely on trade secrets, including
unpatented know-how, technology and other proprietary information, to maintain our competitive position. We seek to
protect our trade secrets, in part, by entering into non-disclosure and confidentiality agreements with parties who have
access to them, such as our employees, corporate collaborators, outside scientific collaborators, contract manufacturers,
consultants, advisors and other third parties. We also enter into confidentiality and invention or patent assignment
agreements with our employees and consultants. Despite these efforts, any of these parties may breach the agreements and
disclose our proprietary information, including our trade secrets, and we may not be able to obtain adequate remedies for
such breaches. Enforcing a claim that a party illegally disclosed or misappropriated a trade secret is difficult, expensive and
time-consuming, and the outcome is unpredictable. In addition, some courts inside and outside the United States are less
willing or unwilling to protect trade secrets. If any of our trade secrets were to be lawfully obtained or independently
developed by a competitor, we would have no right to prevent them, or those to whom they communicate it, from using
that technology or information to compete with us. If any of our trade secrets were to be disclosed to or independently
developed by a competitor, our competitive position would be harmed.
The validity, scope and enforceability of any of our patents that cover any of our commercial products or any of
our drug candidates can be challenged by competitors.
The likelihood that a third party will challenge the patents covering a commercial product is increased because it
is a marketed product. The challenge may come in the form of a patent office proceeding, such as an inter partes review,
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challenging the validity of the patents or a district court proceeding, such as a paragraph IV litigation arising out of the
filing of an ANDA.
If a third party files an ANDA or 505(b)(2) application for a generic of a commercial product, and relies in whole
or in part on studies conducted by or for us, the third party will be required to certify to the FDA that either: (1) there is no
patent information listed in the FDA’s Orange Book with respect to our NDA for the applicable approved drug; (2) the
patents listed in the Orange Book have expired; (3) the listed patents have not expired, but will expire on a particular date
and approval is sought after patent expiration; or (4) the listed patents are invalid or will not be infringed by the
manufacture, use or sale of the third party's generic drug. A certification that the new drug will not infringe the Orange
Book-listed patents for the applicable approved drug, or that such patents are invalid, is called a paragraph IV certification.
If the third party submits a paragraph IV certification to the FDA, a notice of the paragraph IV certification must also be
sent to us once the third party’s ANDA is accepted for filing by the FDA. We may then initiate a lawsuit to defend the
patents identified in the notice. The filing of a patent infringement lawsuit within 45 days of receipt of the notice
automatically prevents the FDA from approving the third party’s ANDA until the earliest of 30 months or the date on
which the patent expires, the lawsuit is settled, or the court reaches a decision in the infringement lawsuit in favor of the
third party. If we do not file a patent infringement lawsuit within the required 45-day period, the third party’s ANDA will
not be subject to the 30-month stay of FDA approval. Litigation or other proceedings to enforce or defend intellectual
property rights are often very complex in nature, may be very expensive and time-consuming, may divert our
management's attention from our core business, and may result in unfavorable results that could limit our ability to prevent
third parties from competing with our commercial products.
If any of our drug candidates advance through development or are approved by the FDA, one or more third parties
may challenge the current patents, or patents that may issue in the future, within our portfolio covering these drug
candidates. Any such challenge could result in the invalidation of, or render unenforceable, some or all of the relevant
patent claims or a finding of non-infringement, which would harm our ability to pursue strategic alternatives, including
identifying and consummating transactions with third-party partners, to further develop, obtain marketing approval for
and/or commercialize our drug candidates, and earn revenue from such arrangements. In addition, any such challenge on
any divested product could harm our ability to earn revenue from the arrangements for such product.
If we do not obtain protection under the Hatch-Waxman Act by extending the patent term and obtaining data
exclusivity for our drug candidates, our business may be materially harmed.
Our success will largely depend on our ability to obtain and maintain patent and other intellectual property in the
United States and other countries with respect to our proprietary technology, drug candidates and our target
indications. Our issued U.S. patents covering ATI-450, our lead inhibitor of the MK2 signaling pathway, expire in 2034,
and other issued patents covering different MK2 signaling pathway inhibitors expire in 2031 and 2032. We currently do not
have any patents issued directed to our lead “soft” JAK inhibitor, ATI-1777, but any claims that may issue would expire in
2038. Our issued patent covering other novel “soft” JAK inhibitors expires 2038. We currently do not have any patents
issued directed to our lead ITK inhibitor, ATI-2138, but any claims that may issue would expire in 2039. Our issued
patents covering other novel inhibitors of ITK expire between 2035 and 2038. Given the amount of time required for the
development, testing and regulatory review of new drug candidates, patents protecting our drug candidates might expire
before or shortly after such candidates begin to be commercialized. We expect to seek extensions of patent terms in the
United States and, if available, in other countries where we are prosecuting patents.
Depending upon the timing, duration and specifics of FDA marketing approval of our drug candidates, one or
more of our U.S. patents may be eligible for limited patent term extension under The Drug Price Competition and Patent
Term Restoration Act of 1984, or the Hatch-Waxman Act, for a drug candidate. The Hatch-Waxman Act permits a patent
extension term of up to five years beyond the normal expiration of the patent as compensation for patent term lost during
development and the FDA regulatory review process, which is limited to the approved indication (or any additional
indications approved during the period of extension). However, the total patent term including the period of extension
cannot exceed 14 years from the product’s approval date. Furthermore, this extension is limited to only one patent per
regulatory review period that covers the approved product. However, the applicable authorities, including the FDA and the
USPTO in the United States, and any equivalent regulatory authority in other countries, may not agree with our assessment
of whether such extensions are available, and may refuse to grant extensions to our patents, or may grant more limited
extensions than we request. We may not be granted an extension because of, for example, failing to apply within
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applicable deadlines, failing to apply prior to expiration of relevant patents or otherwise failing to satisfy applicable
requirements. Moreover, the applicable time period or the scope of patent protection afforded could be less than we
request.
If we are unable to extend the expiration date of our existing patents or obtain new patents with longer expiry
dates, our competitors may be able to take advantage of our investment in development and clinical trials by referencing
our clinical and preclinical data to obtain approval of competing products following our patent expiration and launch their
product earlier than might otherwise be the case.
Any trademarks we have obtained or may obtain may be infringed or successfully challenged, resulting in
harm to our business.
We expect to rely on trademarks as one means to distinguish our products, services or technologies from those of
our competitors. Once we select new trademarks and apply to register them, our trademark applications may not be
approved. Third parties may oppose or attempt to cancel our trademark applications or trademarks, or otherwise challenge
our use of the trademarks. In such an event, we may need to negotiate a settlement agreement with such third party over the
use of our trademarks, which we may not be able to do on commercially reasonable terms, if at all. In the event that our
trademarks are successfully challenged, our products, services or technologies may need to be rebranded, which could
result in loss of brand recognition and could require us to devote resources to advertising and marketing new brands. Our
competitors may infringe our trademarks and we may not have adequate resources to enforce our trademarks.
Outside of the United States we cannot be certain that any country’s patent or trademark office will not
implement new rules that could seriously affect how we draft, file, prosecute and maintain patents, trademarks and
patent and trademark applications.
We cannot be certain that the patent or trademark offices of countries outside the United States will not implement
new rules that increase costs for drafting, filing, prosecuting and maintaining patents, trademarks and patent and trademark
applications or that any such new rules will not restrict our ability to file for patent protection. For example, we may elect
not to seek patent protection in some jurisdictions or for some drug candidates in order to save costs. We may be forced to
abandon or return the rights to specific patents due to a lack of financial resources.
Intellectual property rights do not necessarily address all potential threats to our competitive advantage.
The degree of future protection afforded by our intellectual property rights is uncertain because intellectual
property rights have limitations, and may not adequately protect our business, or permit us to maintain our competitive
advantage. The following examples are illustrative:
● we, our licensors or any potential third-party partners might not have been the first to make the inventions
covered by the issued patents or pending patent applications that we own;
● we, our licensors or any potential third-party partners might not have been the first to file patent applications
covering certain of our inventions;
● others may independently develop similar or alternative technologies or duplicate any of our technologies
without infringing our intellectual property rights;
● it is possible that our pending patent applications will not lead to issued patents;
● issued patents that we own or exclusively license may not provide us with any competitive advantages, or
may be held invalid or unenforceable as a result of legal challenges;
● our competitors might conduct research and development activities in the United States and other countries
that provide a safe harbor from patent infringement claims for certain research and development activities, as
well as in countries where we do not have patent rights, and then use the information learned from such
activities to develop competitive products for sale in major commercial markets; and
● we may not develop additional proprietary technologies that are patentable.
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Risks Related to Regulatory Approval of Our Drug Candidates and Other Legal Compliance Matters
If our potential third-party partners are not able to obtain, or if there are delays in obtaining, required
regulatory approvals, our drug candidates will not be able to be commercialized, and our ability to earn revenue from
arrangements with such third-party partners will be materially impaired.
Our drug candidates and the activities associated with their development and commercialization, including their
design, testing, manufacture, safety, efficacy, recordkeeping, labeling, storage, approval, advertising, promotion, sale and
distribution, are subject to comprehensive regulation by the FDA, other regulatory agencies in the United States and similar
regulatory authorities outside the United States. Failure to obtain marketing approval for a drug candidate will prevent our
potential third-party partners from commercializing the drug candidate. We have only limited experience in filing and
supporting the applications necessary to gain marketing approvals. Securing marketing approval requires the submission of
extensive preclinical and clinical data and supporting information to regulatory authorities for each therapeutic indication
to establish the drug candidate’s safety and efficacy. Securing marketing approval also requires the submission of
information about the drug manufacturing process to, and inspection of manufacturing facilities by, the regulatory
authorities. Our drug candidates may not be effective, may be only moderately effective or may prove to have undesirable
or unintended side effects, toxicities or other characteristics that may preclude our potential third-party partners from
obtaining marketing approval or prevent or limit commercial use. If any of our drug candidates receive marketing approval,
the accompanying label may limit the approved use of our product in this way, which could limit sales of the product.
The process of obtaining marketing approvals, both in the United States and abroad, is expensive and may take
many years if additional clinical trials are required, if approval is obtained at all, and can vary substantially based upon a
variety of factors, including the type, complexity and novelty of the drug candidates involved. Changes in marketing
approval policies during the development period, changes in or the enactment of additional statutes or regulations, or
changes in regulatory review for each submitted drug application, may cause delays in the approval or rejection of an
application. Regulatory authorities have substantial discretion in the approval process and may refuse to accept any
application or may decide that our data is insufficient for approval and require additional preclinical, clinical or other
studies. In addition, varying interpretations of the data obtained from preclinical and clinical testing could delay, limit or
prevent marketing approval of a drug candidate. Any marketing approval our potential third-party partners ultimately
obtain may be limited or subject to restrictions or post-approval commitments that render the approved drug not
commercially viable.
If our potential third-party partners experience delays in obtaining approval or if they fail to obtain approval of our
drug candidates, the commercial prospects for our drug candidates may be harmed and our ability to earn revenue from
arrangements with such third-party partners will be materially impaired.
Failure to obtain marketing approval in international jurisdictions would prevent our drug candidates from
being marketed abroad.
In order to market and sell our drugs in the European Union and any other jurisdictions outside the United States,
our potential third-party partners must obtain separate marketing approvals and comply with numerous and varying
regulatory requirements. The approval procedure varies among countries and can involve additional testing. The time
required to obtain approval may differ substantially from that required to obtain FDA approval. The regulatory approval
process outside the United States generally includes all of the risks associated with obtaining FDA approval. In addition, in
many countries outside the United States, it is required that the drug be approved for reimbursement before the drug can be
approved for sale in that country. Our potential third-party partners may not obtain approvals from regulatory authorities
outside the United States on a timely basis, if at all. Approval by the FDA does not ensure approval by regulatory
authorities in other countries or jurisdictions, and approval by one regulatory authority outside the United States does not
ensure approval by regulatory authorities in other countries or jurisdictions or by the FDA. However, failure to obtain
approval in one jurisdiction may impact our potential third-party partners’ ability to obtain approval elsewhere. Our
potential third-party partners may not be able to file for marketing approvals and may not receive necessary approvals to
commercialize our drug candidates in any market.
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A variety of risks associated with marketing our drug candidates by our potential third-party partners
internationally could harm our business.
If our drug candidates, if approved, are marketed internationally by our potential third-party partners, our potential
third-party partners would be subject to additional risks related to operating in foreign countries, including:
● differing regulatory requirements in foreign countries;
● the potential for so-called parallel importing, which is what happens when a local seller, faced with high or
higher local prices, opts to import goods from a foreign market (with low or lower prices) rather than buying
them locally;
● unexpected changes in tariffs, trade barriers, price and exchange controls and other regulatory requirements;
● economic weakness, including inflation, or political instability in particular foreign economies and markets;
● foreign reimbursement, pricing and insurance regimes;
● compliance with tax, employment, immigration and labor laws for employees living or traveling abroad;
● foreign taxes, including withholding of payroll taxes;
● foreign currency fluctuations, which could result in increased operating expenses and reduced revenue, and
other obligations incident to doing business in another country;
● difficulties staffing and managing foreign operations;
● workforce uncertainty in countries where labor unrest is more common than in the United States;
● potential liability under the U.S. Foreign Corrupt Practices Act of 1977, as amended, or the FCPA, or
comparable foreign regulations;
● challenges enforcing contractual and intellectual property rights, especially in those foreign countries that do
not respect and protect intellectual property rights to the same extent as the United States;
● production shortages resulting from any events affecting raw material supply or manufacturing capabilities
abroad;
● logistical challenges resulting from distributing our drug candidates to foreign countries; and
● business interruptions resulting from geo-political actions, including war and terrorism.
These and other risks associated with international operations may compromise our ability to earn revenue from
arrangements with potential third-party partners for our drug candidates.
Any drug candidate for which our potential third-party partners obtain marketing approval could be subject to
post-marketing restrictions or recall or withdrawal from the market, and our potential third-party partners may be
subject to penalties if they fail to comply with regulatory requirements or if they experience unanticipated problems with
our drug candidates, when and if any of them are approved.
Any drug candidate for which our potential third-party partners obtain marketing approval, along with the
manufacturing processes, post-approval clinical data, labeling, advertising and promotional activities for such drug
candidate, will be subject to continual requirements of and review by the FDA and other regulatory authorities. These
requirements include submissions of safety and other post-marketing information and reports, registration and listing
requirements, cGMP requirements relating to manufacturing, quality control, quality assurance and corresponding
maintenance of records and documents, requirements regarding the distribution of samples to physicians and
recordkeeping. Even if marketing approval of a drug candidate is granted, the approval may be subject to limitations on the
indicated uses for which the drug candidate may be marketed or to the conditions of approval, including the requirement to
implement a risk evaluation and mitigation strategy. If any of our drug candidates receives marketing approval, the
accompanying label may limit the approved use of our drug, which could limit sales of the drug by our potential third-party
partners.
The FDA may also impose requirements for costly post-marketing studies or clinical trials and surveillance to
monitor the safety or efficacy of the drug. The FDA closely regulates the post-approval marketing and promotion of drugs
to ensure drugs are marketed only for the approved indications and in accordance with the provisions of the approved
labeling. The FDA imposes stringent restrictions on manufacturers’ communications regarding off-label use and if our
potential third-party partners do not market our drugs for their approved indications, they may be subject to enforcement
action for off-label marketing. Physicians, on the other hand, may prescribe products for off-label uses. Although the FDA
and other regulatory agencies do not regulate a physician’s choice of drug treatment made in the physician’s independent
medical judgment, they do restrict promotional communications from companies or their sales force with respect to off-
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label uses of products for which marketing clearance has not been issued. However, companies may share truthful and not
misleading information that is otherwise consistent with the product’s FDA approved labeling. Violations of the FDCA
relating to the promotion of prescription drugs may lead to investigations alleging violations of federal and state health care
fraud and abuse laws, as well as state consumer protection laws.
In addition, later discovery of previously unknown adverse events or other problems with our drugs,
manufacturers or manufacturing processes, or failure to comply with regulatory requirements, may have negative
consequences, including:
● restrictions on such drugs, manufacturers or manufacturing processes;
● restrictions on the labeling or marketing of a drug;
● restrictions on drug distribution or use;
● requirements to conduct post-marketing studies or clinical trials;
● warning letters;
● recall or withdrawal of the drugs from the market;
● refusal to approve pending applications or supplements to approved applications;
● clinical holds;
● fines, restitution or disgorgement of profits or revenue;
● suspension or withdrawal of marketing approvals;
● refusal to permit the import or export of our drugs;
● drug seizure; or
● injunctions or the imposition of civil or criminal penalties.
Non-compliance with the European Union’s requirements regarding safety monitoring or pharmacovigilance, and
with requirements related to the development of drugs for the pediatric population, can also result in significant financial
penalties. Similarly, failure to comply with the European Union’s requirements regarding the protection of personal
information can also lead to significant penalties and sanctions. These and other risks associated with the failure by our
potential third-party partners to comply with regulatory requirements may compromise our ability to earn revenue from
arrangements with such third-party partners for our drug candidates.
Our potential third-party partners’ relationships with third-party payors, health care professionals and
customers in the United States and elsewhere may be subject, directly or indirectly, to applicable anti-kickback, fraud
and abuse, false claims, physician payment transparency, health information privacy and security and other health care
laws and regulations, and any failure to comply with such laws and regulations could have a material adverse effect on
our ability to earn revenue from arrangements with such third-party partners for our drug candidates.
Health care providers, physicians and third-party payors in the United States and elsewhere will play a primary
role in the recommendation and prescription of any of our drug candidates for which marketing approval is obtained. Our
potential third-party partners’ arrangements with third-party payors, health care professionals and customers may expose
them to broadly applicable fraud and abuse and other health care laws and regulations, including, without limitation, the
federal Anti-Kickback Statute and the federal civil False Claims Act, that may constrain the business or financial
arrangements and relationships through which they sell, market and distribute any drug candidates for which marketing
approval is obtained. In addition, we and our potential third-party partners may be subject to transparency laws and patient
privacy regulation by the federal government and by the U.S. states and foreign jurisdictions in which we or they conduct
business. The applicable federal, state and foreign health care laws and regulations that may affect our or our potential
third-party partners’ ability to operate include the following:
● the federal Anti-Kickback Statute, which prohibits, among other things, persons and entities from 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, either the referral of an individual for, or the purchase, order or
recommendation of, any good or service, for which payment may be made under federal and state health care
programs such as Medicare and Medicaid. Further, several courts have interpreted the statute’s intent
requirement to mean that if any one purpose of an arrangement involving remuneration is to induce referrals
of federal health care covered business, the Anti-Kickback Statute has been violated. The intent standard
was further amended by the Affordable Care Act, to a stricter standard such that a person or entity no longer
needs to have actual knowledge of the statute or specific intent to violate it in order to have committed a
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violation. Moreover, the government may assert that a claim including items or services resulting from a
violation of the federal Anti-Kickback Statute constitutes a false or fraudulent claim for purposes of the False
Claims Act;
● federal civil and criminal false claims laws, including, without limitation, the federal civil False Claims Act
(that can be enforced through civil whistleblower or qui tam actions), and the civil monetary penalties law,
which impose criminal and civil penalties, against individuals or entities for knowingly presenting, or
causing to be presented, to the federal government, including the Medicare and Medicaid programs, claims
for payment that are false or fraudulent or making a false statement to avoid, decrease or conceal an
obligation to pay money to the federal government;
● federal Health Insurance Portability and Accountability Act of 1996, or HIPAA, which imposes criminal and
civil liability for, among other things, executing a scheme to defraud any health care benefit program or
making false statements relating to health care matters. Similar to the federal Anti-Kickback Statute, a person
or entity does not need to have actual knowledge of the statute or specific intent to violate it to have
committed a violation;
● HIPAA, as amended by HITECH, and their respective implementing regulations, which impose obligations
on covered health care providers, health plans, and health care clearinghouses, as well as their business
associates that create, receive, maintain or transmit individually identifiable health information for or on
behalf of a covered entity and their subcontractors that use, disclose, access, or otherwise process protected
health information, with 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 (commonly
known as the Physician Payments Sunshine Act) and its implementing regulations, which requires specified
manufacturers of drugs, devices, biologics or medical supplies for which payment is available under
Medicare, Medicaid or the Children’s Health Insurance Program, with specific exceptions, to report annually
to CMS information related to payments or other “transfers of value” made to physicians, which is defined to
include doctors, dentists, optometrists, podiatrists and chiropractors and teaching hospitals, as well as
applicable manufacturers to report annually to CMS ownership and investment interests held by physicians
and their immediate family members, and, beginning in 2022, will require applicable manufacturers to report
information related to payments and other transfers of value provided in the previous year to physician
assistants, nurse practitioners, clinical nurse specialists, certified
registered nurse anesthetists,
anesthesiologist assistants and certified nurse midwives. All such reported information is publicly available;
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 health care 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 health care providers; state, local and foreign laws that require drug manufacturers to
report information related to payments and other transfers of value to physicians and other health care
providers or marketing expenditures; state laws that require drug manufacturers to report pricing information
regarding certain drugs; and/or that require registration of certain employees engaged in marketing activities
in the location; and state and foreign laws governing the privacy and security of health information in certain
circumstances, many of which differ from each other in significant ways and often are not preempted by
HIPAA, thus complicating compliance efforts.
Efforts to ensure that our or our potential third-party partners’ business arrangements with third parties will
comply with applicable health care laws and regulations may involve substantial costs. It is possible that governmental
authorities will conclude that our or our potential third-party partners’ business practices, including relationships with
physicians and other health care providers, some of whom may recommend, purchase and/or prescribe our drug candidates,
if approved, may not comply with current or future statutes, regulations or case law involving applicable fraud and abuse or
other health care laws and regulations. By way of example, some of our consulting arrangements with physicians may not
meet all of the criteria of the personal services safe harbor under the federal Anti-Kickback Statute. Accordingly, they may
not qualify for safe harbor protection from government prosecution. A business arrangement that does not substantially
comply with a safe harbor, however, is not necessarily illegal under the Anti-Kickback Statute, but may be subject to
additional scrutiny by the government.
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If our or our potential third-party partners’ operations are found to be in violation of any of these laws or any other
governmental regulations that may apply to us or them, we or our potential third-party partners may be subject to
significant civil, criminal and administrative penalties, including, without limitation, damages, fines, disgorgement,
imprisonment, exclusion from participation in government health care programs, such as Medicare and Medicaid,
additional reporting requirements and oversight if we or they become subject to a corporate integrity agreement or similar
agreement to resolve allegations of non-compliance with these laws and the curtailment or restructuring of our or their
operations, which could have a material adverse effect on our ability to earn revenue from arrangements with such third-
party partners for our drug candidates. If any physician or other health care provider or entity with whom we or our
potential third-party partners expect to do business is found not to be in compliance with applicable laws, it may be subject
to significant criminal, civil or administrative sanctions, including exclusions from participation in government health care
programs, which could also materially affect our ability to earn revenue from arrangements with such third-party partners
for our drug candidates.
Recently enacted and future legislation may increase the difficulty and cost for our potential third-party
partners to obtain marketing approval of our drug candidates and commercialize our drug candidates, if approved, and
affect the prices our potential third-party partners may obtain.
In the United States, and some foreign jurisdictions, there have been a number of legislative and regulatory
changes and proposed changes regarding the health care system that could prevent or delay marketing approval of our drug
candidates, restrict or regulate post-approval activities and affect our potential third-party partners’ ability to profitably sell
any of our drug candidates for which our potential third-party partners obtain marketing approval, and consequently affect
our ability to earn revenue from arrangements with such third-party partners for our drug candidates.
Among policy makers and payors in the United States and elsewhere, there is significant interest in promoting
changes in health care systems with the stated goals of containing health care costs, improving quality and/or expanding
access. In the United States, the pharmaceutical industry has been a particular focus of these efforts and has been
significantly affected by major legislative initiatives. The Affordable Care Act, which was signed into law in 2010, is a
sweeping law intended to broaden access to health insurance, reduce or constrain the growth of health care spending,
enhance remedies against fraud and abuse, add new transparency requirements for the health care and health insurance
industries, impose new taxes and fees on the health industry and impose additional health policy reforms.
Among the provisions of the Affordable Care Act of importance to commercial products are the following:
● an annual, nondeductible fee on any entity that manufactures or imports certain branded prescription drugs
and biologic agents, apportioned among these entities according to their market share in certain government
health care programs;
● an increase in the statutory minimum rebates a manufacturer must pay under the Medicaid Drug Rebate
Program to 23.1% and 13.0% of the average manufacturer price for branded and generic drugs, respectively;
● expansion of health care fraud and abuse laws, including the False Claims Act and the Anti-Kickback
Statute, which include, among other things, new government investigative powers and enhanced penalties for
non-compliance;
● a new Medicare Part D coverage gap discount program, in which manufacturers must agree to offer 70%
point-of-sale discounts off negotiated prices of applicable brand drugs to eligible beneficiaries during their
coverage gap period, as a condition for the manufacturer’s outpatient drugs to be covered under Medicare
Part D;
● extension of manufacturers’ Medicaid rebate liability to covered drugs dispensed to individuals who are
enrolled in Medicaid managed care organizations;
● expansion of eligibility criteria for Medicaid programs by, among other things, allowing states to offer
Medicaid coverage to additional individuals, thereby potentially increasing manufacturers’ Medicaid rebate
liability;
● expansion of the entities eligible for discounts under the Public Health Service pharmaceutical pricing
program;
● the new requirements under the federal Open Payments program and its implementing regulations;
● a new requirement to annually report drug samples that manufacturers and distributors provide to physicians;
and
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● a new Patient-Centered Outcomes Research Institute to oversee, identify priorities in, and conduct
comparative clinical effectiveness research, along with funding for such research.
There remain judicial and Congressional challenges to certain aspects of the Affordable Care Act. While Congress
has not passed comprehensive repeal legislation, several bills affecting the implementation of certain taxes under the
Affordable Care Act have been signed into law. The Tax Cuts and Jobs Act of 2017, or the 2017 Tax Act, includes a
provision that repealed, effective January 1, 2019, the tax-based shared responsibility payment imposed by the Affordable
Care Act on certain individuals who fail to maintain qualifying health coverage for all or part of a year that is commonly
referred to as the “individual mandate”. Additionally, the 2020 federal spending package permanently eliminated, effective
January 1, 2020, the Affordable Care Act-mandated “Cadillac” tax on high-cost employer-sponsored health coverage and
medical device tax and, effective January 1, 2021, also eliminated the health insurer tax. Further, the Bipartisan Budget
Act of 2018, or the BBA, among other things, amended the Affordable Care Act, effective January 1, 2019, to close the
coverage gap in most Medicare drug plans, commonly referred to as the “donut hole”. On December 14, 2018, a Texas
U.S. District Court Judge ruled that the Affordable Care Act is unconstitutional in its entirety because the “individual
mandate” was repealed by Congress as part of the 2017 Tax Act. Additionally, on December 18, 2019, the U.S. Court of
Appeals for the Fifth Circuit upheld the District Court ruling that the individual mandate was unconstitutional and
remanded the case back to the District Court to determine whether the remaining provisions of the Affordable Care Act are
invalid as well. The U.S. Supreme Court is currently reviewing the case, although it is unknown when a decision will be
made. Further, although the U.S. Supreme Court has not yet ruled on the constitutionality of the Affordable Care Act, on
January 28, 2021, President Biden issued an executive order to initiate a special enrollment period from February 15, 2021
through May 15, 2021 for purposes of obtaining health insurance coverage through the Affordable Care Act marketplace.
The executive order also instructs certain governmental agencies to review and reconsider their existing policies and rules
that limit access to healthcare, including among others, reexamining Medicaid demonstration projects and waiver programs
that include work requirements, and policies that create unnecessary barriers to obtaining access to health insurance
coverage through Medicaid or the Affordable Care Act. It is unclear how the Supreme Court ruling, other such litigation,
and the health care reform measures of the Biden administration will impact the Affordable Care Act and our business.
In addition, other legislative changes have been proposed and adopted since the Affordable Care Act was enacted.
These changes included aggregate reductions to Medicare payments to providers of 2% per fiscal year that became
effective on April 1, 2013 and, due to subsequent legislative amendments to the statute, including the BBA, will stay in
effect through 2030 with the exception of a temporary suspension from May 1, 2020 through March 31, 2021 unless
additional Congressional action is taken. The American Taxpayer Relief Act of 2012, which was signed into law in
January 2013, among other things, further reduced Medicare payments to several providers, and increased the statute of
limitations period for the government to recover overpayments to providers from three to five years. Any similar new laws
may result in additional reductions in Medicare and other health care funding, which could have a material adverse effect
on our ability to earn revenue from arrangements with our potential third-party partners for our drug candidates.
We expect that the Affordable Care Act, as well as other health care reform measures that may be adopted in the
future, may result in more rigorous coverage criteria and in additional downward pressure on the price that our potential
third-party partners receive for any approved drug candidate. Any reduction in reimbursement from Medicare or other
government programs may result in a similar reduction in payments from private payors. The implementation of cost
containment measures or other health care reforms may prevent our potential third-party partners from being able to
generate revenue, attain profitability, or commercialize our drug candidates, if approved, which in turn may impact our
ability to earn revenue from arrangements with such third-party partners for our drug candidates. Further, it is also possible
that additional governmental action is taken in response to the COVID-19 pandemic.
Legislative and regulatory proposals have been made to expand post-approval requirements and restrict sales and
promotional activities for drugs. In addition, there has been heightened governmental scrutiny in the United States of
pharmaceutical pricing practices in light of the rising cost of prescription drugs and biologics. Such scrutiny has resulted in
several recent Congressional inquiries and proposed and enacted federal and state legislation designed to, among other
things, bring more transparency to drug pricing, review the relationship between pricing and manufacturer patient
programs, and reform government program reimbursement methodologies for drugs. At the federal level, the Trump
administration used several means to propose or implement drug pricing reform, including through federal budget
proposals, executive orders and policy initiatives. For example, on July 24, 2020 and September 13, 2020, President Trump
announced several executive orders related to prescription drug pricing that seek to implement several of the
administration’s proposals. As a result, the FDA released a final rule on September 24, 2020, effective November 30,
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2020, providing guidance for states to build and submit importation plans for drugs from Canada. Further, on November
20, 2020, HHS finalized a regulation removing safe harbor protection for price reductions from pharmaceutical
manufacturers to plan sponsors under Part D, either directly or through pharmacy benefit managers, unless the price
reduction is required by law. The implementation of the rule has been delayed by the Biden administration from January 1,
2022 to January 1, 2023 in response to ongoing litigation. The rule also creates a new safe harbor for price reductions
reflected at the point-of-sale, as well as a new safe harbor for certain fixed fee arrangements between pharmacy benefit
managers and manufacturers, the implementation of which have also been delayed pending review by the Biden
administration until March 22, 2021. On November 20, 2020, CMS issued an interim final rule implementing President
Trump’s Most Favored Nation executive order, which would tie Medicare Part B payments for certain physician-
administered drugs to the lowest price paid in other economically advanced countries, effective January 1, 2021. On
December 28, 2020, the U.S. District Court in Northern California issued a nationwide preliminary injunction against
implementation of the interim final rule. It is unclear whether the Biden administration will work to reverse these measures
or pursue similar policy initiatives. At the state level, legislatures have become increasingly active in passing legislation
and implementing regulations designed to control pharmaceutical and biological product pricing, including price or patient
reimbursement constraints, discounts, restrictions on certain drug access and marketing cost disclosure and transparency
measures, and, in some cases, designed to encourage importation from other countries and bulk purchasing. We cannot be
sure whether additional legislative changes will be enacted, or whether the FDA regulations, guidance or interpretations
will be changed, or what the impact of such changes on obtaining marketing approvals for our drug candidates, if any, may
be. In addition, increased scrutiny by the U.S. Congress of the FDA’s approval process may significantly delay or prevent
marketing approval, as well as subject our potential third-party partners to more stringent drug labeling and post-marketing
testing and other requirements. These risks may compromise our ability to earn revenue from arrangements with such
third-party partners for our drug candidates.
Governments outside the United States tend to impose strict price controls, which may adversely affect our
revenue.
In some countries, particularly the countries of the European Union, the pricing of prescription pharmaceuticals is
subject to governmental control. In these countries, pricing negotiations with governmental authorities can take
considerable time after the receipt of marketing approval for a drug. To obtain coverage and reimbursement or pricing
approval in some countries, our potential third-party partners may be required to conduct a clinical trial that compares the
cost-effectiveness of our drug candidate to other available procedures. If reimbursement of our drug candidates is
unavailable or limited in scope or amount, or if pricing is set at unsatisfactory levels, our potential third-party partners may
not able to generate revenue, which in turn may adversely affect our ability to earn revenue from arrangements with such
third-party partners for our drug candidates.
If we fail to comply with environmental, health and safety laws and regulations, we could become subject to
fines or penalties or incur costs that could harm our business.
We are subject to numerous environmental, health and safety laws and regulations, including those governing
laboratory procedures and the handling, use, storage, treatment and disposal of hazardous materials and wastes. Our
operations involve the use of hazardous and flammable materials, including chemicals and biological materials. Our
operations also produce hazardous waste products. We generally contract with third parties for the disposal of these
materials and wastes. We cannot eliminate the risk of contamination or injury from these materials. In the event of
contamination or injury resulting from our use of hazardous materials, we could be held liable for any resulting damages,
and any liability could exceed our resources. We also could incur significant costs associated with civil or criminal fines
and penalties for failure to comply with such laws and regulations.
Although we maintain workers’ compensation insurance to cover us for costs and expenses we may incur due to
injuries to our employees resulting from the use of hazardous materials, this insurance may not provide adequate coverage
against potential liabilities. We do not maintain insurance for environmental liability or toxic tort claims that may be
asserted against us in connection with our storage or disposal of biological, hazardous or radioactive materials.
In addition, we may incur substantial costs in order to comply with current or future environmental, health and
safety laws and regulations. These current or future laws and regulations may impair our development or manufacturing
efforts. Our failure to comply with these laws and regulations also may result in substantial fines, penalties or other
sanctions.
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We are subject to governmental economic sanctions and export and import controls that could impair our
potential third-party partners’ ability to compete in international markets or subject us or our potential third-party
partners to liability if we or they are not in compliance with applicable laws.
As a U.S. company, we are subject to U.S. import and export controls and economic sanctions laws and
regulations, and we are required to import and export our drug candidates, technology and services in compliance with
those laws and regulations, including the U.S. Export Administration Regulations, the International Traffic in Arms
Regulations, and economic embargo and trade sanction programs administered by the Treasury Department’s Office of
Foreign Assets Control.
U.S. economic sanctions and export control laws and regulations prohibit the shipment of certain products and
services to countries, governments and persons targeted by U.S. sanctions. While we are currently taking precautions to
prevent doing any business, directly or indirectly, with countries, governments and persons targeted by U.S. sanctions and
to ensure that our drug candidates are not exported or used by countries, governments and persons targeted by U.S.
sanctions, such measures may be circumvented.
Furthermore, if we or our potential third-party partners export our drug candidates, the exports may require
authorizations, including a license, a license exception or other appropriate government authorization. Complying with
export control and sanctions regulations may be time-consuming and may result in the delay or loss of sales opportunities.
Failure to comply with export control and sanctions regulations may expose us or our potential third-party partners to
government investigations and penalties.
If we are found to be in violation of U.S. sanctions or import or export control laws, it could result in civil and
criminal, monetary and non-monetary penalties, including possible incarceration for those individuals responsible for the
violations, the loss of export or import privileges and reputational harm.
We and our potential third-party partners are subject to anti-corruption and anti-money laundering laws with
respect to our and their operations and non-compliance with such laws can subject us to criminal and/or civil liability
and harm our business.
We and our potential third-party partners are subject to the FCPA, the U.S. domestic bribery statute contained in
18 U.S.C. § 201, the U.S. Travel Act, the USA PATRIOT Act and possibly other anti-bribery and anti-money laundering
laws in countries in which we conduct activities. Anti-corruption laws are interpreted broadly and prohibit companies and
their employees and third-party intermediaries from authorizing, offering or providing, directly or indirectly, improper
payments or benefits to recipients in the public or private sector. We or our potential third-party partners may engage third-
party intermediaries in connection with the development or commercialization of our drug candidates, if approved, and to
obtain necessary permits, licenses and other regulatory approvals. We, our potential third-party partners or the third-party
intermediaries may have direct or indirect interactions with officials and employees of government agencies or state-owned
or affiliated entities. We can be held liable for the corrupt or other illegal activities of these third-party intermediaries, our
employees, representatives, contractors, partners and agents, even if we do not explicitly authorize such activities.
Noncompliance with anti-corruption and anti-money laundering laws could subject us to whistleblower
complaints, investigations, sanctions, settlements, prosecution, other enforcement actions, disgorgement of profits,
significant fines, damages, other civil and criminal penalties or injunctions, suspension and/or debarment from contracting
with certain persons, the loss of export privileges, reputational harm, adverse media coverage and other collateral
consequences. Responding to any action will likely result in a materially significant diversion of management’s attention
and resources and significant defense costs and other professional fees.
Risks Related to Employee Matters and Managing Our Growth
Our future success depends on our ability to retain key executives and to attract, retain and motivate qualified
personnel.
We are highly dependent on the management, development, clinical, financial, legal and business development
expertise of Dr. Neal Walker, our Chief Executive Officer, Dr. David Gordon, our Chief Medical Officer, Frank Ruffo, our
Chief Financial Officer, and Kamil Ali-Jackson, our Chief Legal Officer, as well as the other members of our scientific and
clinical teams. Although we have entered into employment agreements with certain of our executive officers, each of
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them may currently terminate their employment with us or resign at any time. We do not maintain “key person” insurance
for any of our key executives other than for Dr. Walker.
Recruiting and retaining qualified scientific, manufacturing and clinical personnel will also be critical to our
success. The loss of the services of our executive officers or other key employees could impede the achievement of our
development objectives and seriously harm our ability to successfully implement our business strategy. Furthermore,
replacing executive officers and key employees may be difficult and may take an extended period of time because of the
limited number of individuals in our industry with the breadth of skills and experience required to successfully develop and
partner drug candidates. Competition to hire from this limited pool is intense, and we may be unable to hire, train, retain or
motivate these key personnel on acceptable terms given the competition among numerous pharmaceutical and
biotechnology companies for similar personnel. We also experience competition for the hiring of scientific and clinical
personnel from universities and research institutions. In addition, we rely on consultants and advisors, including scientific
and clinical advisors, to assist us in formulating our development strategy. Our consultants and advisors may have
commitments under employment, consulting or advisory contracts with other entities that may limit their availability to us.
If we are unable to continue to attract and retain high quality personnel, our ability to pursue our growth strategy will be
limited.
Our employees, independent contractors, consultants, third-party partners, principal investigators, CROs and
vendors may engage in misconduct or other improper activities, including non-compliance with regulatory standards
and requirements.
We are exposed to the risk that our employees, independent contractors, consultants, third-party partners, principal
investigators, CROs and vendors may engage in fraudulent conduct or other illegal activity. Misconduct by these parties
could include intentional, reckless and/or negligent conduct or disclosure of unauthorized activities to us that violates FDA
regulations, including those laws requiring the reporting of true, complete and accurate information to the FDA,
manufacturing standards, federal and state health care laws and regulations, and laws that require the true, complete and
accurate reporting of financial information or data. In particular, sales, marketing and business arrangements by our
potential third-party partners in the health care industry are subject to extensive laws and regulations intended to prevent
fraud, kickbacks, self-dealing and other abusive practices. These laws and regulations may restrict or prohibit a wide range
of pricing, discounting, marketing and promotion, sales commission, customer incentive programs and other business
arrangements. Misconduct by these parties could also involve the improper use of individually identifiable information,
including, without limitation, information obtained in the course of clinical trials, which could result in regulatory
sanctions and serious harm to our reputation. We have adopted a code of business conduct and ethics, but it is not always
possible to identify and deter misconduct, and the precautions we take to detect and prevent this activity may not be
effective in controlling unknown or unmanaged risks or losses or in protecting us from governmental investigations or
other actions or lawsuits stemming from a failure to be in compliance with such laws or regulations. If any such actions are
instituted against us, and we are not successful in defending ourselves or asserting our rights, those actions could have a
significant impact on our business, including the imposition of significant civil, criminal and administrative penalties,
including, without limitation, damages, fines, disgorgement, imprisonment, exclusion from participation in government
health care programs, such as Medicare and Medicaid, additional reporting obligations and oversight if we are subject to a
corporate integrity agreement or other agreement to resolve allegations of non-compliance with these laws, and the
curtailment or restructuring of our operations.
Risks Related to Ownership of Our Common Stock
The trading price of the shares of our common stock has been and is likely to continue to be volatile.
Our stock price has been and is likely to continue to be volatile. The stock market in general and the market for
biotechnology companies in particular have experienced extreme volatility that has often been unrelated to the operating
performance of particular companies. As a result of this volatility, investors may not be able to sell their common stock at
or above the price paid for the shares. The market price for our common stock may be influenced by many factors,
including:
● the commencement, enrollment or results of any clinical trials we may conduct, or changes in the
development status of our drug candidates;
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● any delay in our regulatory filings for any of our drug candidates and any adverse development or perceived
adverse development with respect to the applicable regulatory authority’s review of such filings, including
without limitation the FDA’s issuance of a “refusal to file” letter or a request for additional information;
● adverse results from, delays in or termination of clinical trials;
● adverse regulatory decisions, including failure of any of our drug candidates to receive marketing approval;
● unanticipated serious safety concerns related to the use of any drug candidate or previously sold commercial
product;
● changes in financial estimates by us or by any securities analysts who might cover our stock;
● conditions or trends in our industry;
● changes in the structure of health care payment systems;
● changes in the market valuations of similar companies;
● stock market price and volume fluctuations of comparable companies and, in particular, those that operate in
the biotechnology industry;
● publication of research reports about us or our industry or positive or negative recommendations or
withdrawal of research coverage by securities analysts;
● announcements by us or our competitors of significant acquisitions, strategic partnerships or divestitures;
● announcements of investigations or regulatory scrutiny of our operations or lawsuits filed against us;
● investors’ general perception of our company and our business;
● recruitment or departure of key personnel;
● overall performance of the equity markets;
● trading volume of our common stock;
● disputes or other developments relating to proprietary rights, including patents, litigation matters and our
ability to obtain patent protection for our technologies;
● significant lawsuits, including patent or stockholder litigation;
● general political and economic conditions;
● the evolution of the COVID-19 pandemic and success of mass vaccination efforts; and
● other events or factors, many of which are beyond our control.
In the past, stockholders have initiated class action lawsuits against pharmaceutical companies following periods
of volatility in the market prices of these companies’ stock. For example, two purported class action complaints were filed
against us and certain of our executive officers alleging violations of certain federal securities laws and two stockholder
derivative actions were filed against certain of our executive officers and directors alleging breaches of fiduciary duties.
We and the other defendants dispute the plaintiffs’ claims and intend to defend these matters vigorously. We have entered
into indemnification agreements with our executive officers and directors which provide, among other things, that we will
indemnify such officer or director, under the circumstances and to the extent provided for therein, for expenses, damages,
judgments, fines and settlements he or she may be required to pay in actions or proceedings which he or she is or may be
made a party by reason of his or her position as our director, officer or other agent, and otherwise to the fullest extent
permitted under Delaware law and our bylaws. These cases, and additional litigation, if instituted against us, could cause us
to incur substantial costs and divert management’s attention and resources from our business.
Sales of a substantial number of shares of our common stock into the market could cause the market price of
our common stock to drop significantly, even if our business is doing well.
Sales of a substantial number of shares of our common stock in the public market could occur at any time. If our
stockholders sell, or the market perceives that our stockholders intend to sell, substantial amounts of our common stock in
the public market, the market price of our common stock could decline significantly.
In addition, we have filed registration statements on Form S-8 under the Securities Act registering the issuance of
shares of common stock subject to options or other equity awards issued or reserved for future issuance under our equity
incentive plans. Shares registered under these registration statements are available for sale in the public market subject to
vesting arrangements and exercise of options, and the restrictions of Rule 144 under the Securities Act in the case of our
affiliates.
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Provisions in our corporate charter documents and under Delaware law may prevent or frustrate attempts by
our stockholders to change our management and hinder efforts to acquire a controlling interest in us, and the market
price of our common stock may be lower as a result.
There are provisions in our certificate of incorporation and bylaws that may make it difficult for a third party to
acquire, or attempt to acquire, control of our company, even if a change of control was considered favorable by some or all
of our stockholders. For example, our board of directors has the authority to issue up to 10,000,000 shares of preferred
stock. The board of directors can fix the price, rights, preferences, privileges, and restrictions of the preferred stock without
any further vote or action by our stockholders. The issuance of shares of preferred stock may delay or prevent a change of
control transaction. As a result, the market price of our common stock and the voting and other rights of our stockholders
may be adversely affected. An issuance of shares of preferred stock may result in the loss of voting control to other
stockholders.
Our charter documents also contain other provisions that could have an anti-takeover effect, including:
● only one of our three classes of directors is elected each year;
● stockholders are not entitled to remove directors other than by a 66 2/3% vote and only for cause;
● stockholders are not permitted to take actions by written consent;
● stockholders cannot call a special meeting of stockholders; and
● stockholders must give advance notice to nominate directors or submit proposals for consideration at
stockholder meetings.
In addition, we are subject to the anti-takeover provisions of Section 203 of the Delaware General Corporation
Law, which regulates corporate acquisitions by prohibiting Delaware corporations from engaging in specified business
combinations with particular stockholders of those companies. These provisions could discourage potential acquisition
proposals and could delay or prevent a change of control transaction. They could also have the effect of discouraging
others from making tender offers for our common stock, including transactions that may be in your best interests. These
provisions may also prevent changes in our management or limit the price that investors are willing to pay for our stock.
We are a “smaller reporting company” and, as a result of the reduced disclosure and governance requirements
applicable to smaller reporting companies, our common stock may be less attractive to investors.
We are a “smaller reporting company” as defined in Item 10(f)(1) of Regulation S-K, and we intend to take
advantage of some of the exemptions from reporting requirements that are applicable to other public companies that are not
smaller reporting companies, including:
● not being required to comply with the auditor attestation requirements in the assessment of our internal
control over financial reporting; and
● reduced disclosure obligations regarding executive compensation in our periodic reports, proxy statements
and registration statements.
We may take advantage of these reporting exemptions until we are no longer a smaller reporting company. We
will remain a smaller reporting company until the last day of any fiscal year for so long as either (1) the market value of
our shares of common stock held by non-affiliates does not equal or exceed $250.0 million as of the prior June 30th, or (2)
our annual revenues did not equal or exceed $100.0 million during such completed fiscal year and the market value of our
shares of common stock held by non-affiliates did not equal or exceed $700.0 million as of the prior June 30th.
We cannot predict if investors will find our common stock less attractive because we will rely on these
exemptions. If some investors find our common stock less attractive as a result, there may be a less active trading market
for our common stock and our stock price may be more volatile.
If we fail to maintain proper and effective internal controls, our ability to produce accurate financial
statements on a timely basis could be impaired.
We are subject to the reporting requirements of the Exchange Act, the Sarbanes-Oxley Act and the rules and
regulations of the stock market on which our common stock is listed. The Sarbanes-Oxley Act requires, among other
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things, that we maintain effective disclosure controls and procedures and internal control over financial reporting, and
perform system and process evaluation and testing of our internal control over financial reporting to allow management to
report on the effectiveness of our internal control over financial reporting. This requires that we incur substantial additional
professional fees and internal costs to expand our accounting and finance functions and that we expend significant
management efforts.
We may identify weaknesses in our system of internal financial and accounting controls and procedures that could
result in a material misstatement of our consolidated financial statements. Our internal control over financial reporting will
not prevent or detect all errors and all fraud. A control system, no matter how well designed and operated, can provide only
reasonable, not absolute, assurance that the control system’s objectives will be met. Because of the inherent limitations in
all control systems, no evaluation of controls can provide absolute assurance that misstatements due to error or fraud will
not occur or that all control issues and instances of fraud will be detected.
If we are unable to maintain proper and effective internal controls, we may not be able to produce timely and
accurate financial statements, and we may conclude that our internal control over financial reporting is not effective. If that
were to happen, the market price of our stock could decline, and we could be subject to sanctions or investigations by the
stock exchange on which our common stock is listed, the SEC, or other regulatory authorities.
We might not be able to utilize a significant portion of our net operating loss carryforwards and research and
development tax credit carryforwards.
As of December 31, 2020, we had federal and state net operating loss carryforwards, or NOLs, of $367.6 million
and $369.6 million, respectively, which will begin to expire in 2032. Under the 2017 Tax Act, as modified by the
Coronavirus Aid, Relief, and Economic Security Act, or CARES Act, federal NOL carryforwards generated in tax years
beginning after December 31, 2017 may be carried forward indefinitely but, in the case of tax years beginning after 2020,
may only be used to offset 80% of our taxable income annually. Federal NOL carryforwards generated in taxable years
beginning in 2018, 2019 and 2020 will similarly carry forward indefinitely but will not be subject to such 80% of annual
taxable income limitation. It is uncertain if and to what extent various states will conform to the federal tax law. As of
December 31, 2020, we also had federal research and development tax credit carryforwards of $8.6 million which will
begin to expire in 2032, and state research and development tax credit carryforwards of $0.1 million which will begin to
expire in 2022. We also have $0.2 million of loss carryforwards in the United Kingdom which can be carried forward
indefinitely. These net operating loss and tax credit carryforwards could expire unused or due to limitation on use be
unavailable to offset future income tax liabilities. In addition, under Section 382 of the Internal Revenue Code of 1986, as
amended, and corresponding provisions of state law, if a corporation undergoes an “ownership change,” which is generally
defined as a greater than 50% change, by value, in its equity ownership over a three-year period, the corporation’s ability to
use its pre-change net operating loss carryforwards and other pre-change tax attributes to offset its post-change income may
be limited. We have completed an analysis under Section 382 for NOLs generated from July 13, 2012 through July 20,
2020. Although we have experienced Section 382 ownership changes since 2012, we have concluded that we should have
sufficient ability to utilize NOLs accumulated during the periods tested. We have not yet determined if a Section 382
ownership change has occurred after July 20, 2020. In addition, we may experience ownership changes in the future as a
result of subsequent shifts in our stock ownership, some of which may be outside of our control. If we determine that an
ownership change has occurred and our ability to use our historical net operating loss and tax credit carryforwards is
materially limited, it might harm our future operating results by effectively increasing our future tax obligations.
We do not anticipate paying any cash dividends on our common stock in the foreseeable future and our stock
may not appreciate in value.
We have not declared or paid cash dividends on our common stock to date. We currently intend to retain our
future earnings, if any, to fund the development and growth of our business. There is no guarantee that shares of our
common stock will appreciate in value or that the price at which our stockholders have purchased their shares will be able
to be maintained.
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Exclusive forum provisions in our amended and restated certificate of incorporation and amended and restated
bylaws could limit our stockholders’ ability to obtain a favorable judicial forum for disputes with us or our directors,
officers or other employees.
Our amended and restated certificate of incorporation provides that the Court of Chancery of the State of
Delaware is the exclusive forum for the following types of actions or proceedings under Delaware statutory or common
law: (i) any derivative action or proceeding brought on our behalf, (ii) any action asserting a claim for breach of a fiduciary
duty owed by any of our directors, officers or other employees to us or our stockholders, (iii) any action asserting a claim
arising pursuant to any provision of the Delaware General Corporation Law, our amended and restated certificate of
incorporation or our amended and restated bylaws or (iv) any action asserting a claim governed by the internal affairs
doctrine. This provision would not apply to suits brought to enforce a duty or liability created by the Exchange Act.
Furthermore, Section 22 of the Securities Act creates concurrent jurisdiction for federal and state courts over all such
Securities Act actions. Accordingly, both state and federal courts have jurisdiction to entertain such claims. To prevent
having to litigate claims in multiple jurisdictions and the threat of inconsistent or contrary rulings by different courts,
among other considerations, our amended and restated bylaws provide the federal district courts of the United States of
America will be the exclusive forum for resolving any complaint asserting a cause of action arising under the Securities
Act. While the Delaware courts have determined that such choice of forum provisions are facially valid, a stockholder may
nevertheless seek to bring a claim in a venue other than those designated in the exclusive forum provisions. In such
instance, we would expect to vigorously assert the validity and enforceability of the exclusive forum provisions of our
amended and restated certificate of incorporation and our amended and restated bylaws. This may require significant
additional costs associated with resolving such action in other jurisdictions and there can be no assurance that the
provisions will be enforced by a court in those other jurisdictions.
Our amended and restated certificate of incorporation and amended and restated bylaws further provide any
person or entity purchasing or otherwise acquiring any interest in shares of our common stock is deemed to have notice of
and consented to the foregoing provisions. These exclusive forum provisions may limit a stockholder’s ability to bring a
claim in a judicial forum that it finds favorable for disputes with us or our directors, officers, or other employees, which
may discourage lawsuits against us and our directors, officers and other employees. If a court were to find either exclusive-
forum provision to be inapplicable or unenforceable in an action, we may incur further significant additional costs
associated with resolving the dispute in other jurisdictions, all of which could seriously harm our business.
General Risk Factors
Our business and operations would suffer in the event of computer system failures, cyber-attacks or a
deficiency in our cyber-security.
Despite the implementation of security measures, our internal computer systems, and those of third parties on
which we rely, are vulnerable to damage from computer viruses, malware, natural disasters, terrorism, war,
telecommunication and electrical failures, cyber-attacks or cyber-intrusions over the Internet, attachments to emails,
persons inside our organization, or persons with access to systems inside our organization. The risk of a security breach or
disruption, particularly through cyber-attacks or cyber intrusion, including by computer hackers, foreign governments, and
cyber terrorists, has generally increased as the number, intensity and sophistication of attempted attacks and intrusions from
around the world have increased. If such an event were to occur and cause interruptions in our operations, it could result in
a material disruption of our drug development programs. For example, the loss of clinical trial data from completed or
ongoing or planned clinical trials could result in delays in obtaining marketing approval for our drug candidates and
significantly increase our costs to recover or reproduce the data. To the extent that any disruption or security breach was to
result in a loss of or damage to our data or applications, or inappropriate disclosure of confidential or proprietary
information, we could incur material legal claims and liability, damage to our reputation, and the further development or
commercialization of our drug candidates by a potential third-party partner could be delayed.
An active trading market for our common stock may not be sustained.
Although our common stock is listed on The Nasdaq Global Select Market, we cannot assure you that an active
trading market for our shares will be sustained. If an active market for our common stock is not sustained, it may be
difficult for investors in our common stock to sell shares without depressing the market price for the shares or to sell the
shares at all.
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If equity research analysts do not publish research or reports, or publish unfavorable research or reports,
about us, our business or our market, our stock price and trading volume could decline.
The trading market for our common stock is influenced by the research and reports that equity research analysts
publish about us or our business, our market and our competitors. Equity research analysts may elect not to initiate or
continue to provide research coverage of our common stock, and such lack of research coverage may adversely affect the
market price of our common stock. Even if we have equity research analyst coverage, we will not have any control over the
analysts or the content and opinions included in their reports. The price of our stock could decline if one or more equity
research analysts downgrade our stock or issue other unfavorable commentary or research. If one or more equity research
analysts ceases coverage of our company or fails to publish reports on us regularly, demand for our stock could decrease,
which in turn could cause our stock price or trading volume to decline.
The issuance of additional stock in connection with financings, acquisitions, investments, our equity incentive
plan or otherwise will dilute all other stockholders.
Our certificate of incorporation authorizes us to issue up to 100,000,000 shares of common stock and up to
10,000,000 shares of preferred stock with such rights and preferences as may be determined by our board of directors.
Subject to compliance with applicable rules and regulations, we may issue our shares of common stock or securities
convertible into our common stock from time to time in connection with a financing, acquisition, investment, our equity
incentive plan or otherwise. Any such issuance could result in substantial dilution to our existing stockholders and cause
the trading price of our common stock to decline.
Changes in tax laws or regulations that are applied adversely to us may have a material adverse effect on our
business, cash flow, financial condition or results of operations.
New income, sales, use or other tax laws, statutes, rules, regulations or ordinances could be enacted at any time,
which could adversely affect our business operations and financial performance. Further, existing tax laws, statutes, rules,
regulations or ordinances could be interpreted, changed, modified or applied adversely to us. For example, the 2017 Tax
Act enacted many significant changes to the U.S. tax laws. Future guidance from the Internal Revenue Service and other
tax authorities with respect to the 2017 Tax Act (as modified by the CARES Act) may affect us, and certain aspects of the
2017 Tax Act could be repealed or modified in future legislation. In addition, it is uncertain if and to what extent various
states will conform to the 2017 Tax Act or any newly enacted federal tax legislation. Changes in corporate tax rates, the
realization of net deferred tax assets relating to our operations, the taxation of foreign earnings, and the deductibility of
expenses under the 2017 Tax Act or future reform legislation could have a material impact on the value of our deferred tax
assets, could result in significant one-time charges, and could increase our future U.S. tax expense.
We incur significant costs and demands upon management as a result of being a public company.
As a public company listed in the United States, we incur, and will continue to incur, particularly after we cease to
be a “smaller reporting company,” significant legal, accounting and other costs. These costs could negatively affect our
financial results. In addition, changing laws, regulations and standards relating to corporate governance and public
disclosure, including regulations implemented by the SEC and The Nasdaq Stock Market, may increase legal and financial
compliance costs and make some activities more time-consuming. These laws, regulations and standards are subject to
varying interpretations and, as a result, their application in practice may evolve over time as new guidance is provided by
regulatory and governing bodies. We intend to invest resources to comply with evolving laws, regulations and standards,
and this investment may result in increased general and administrative expenses and a diversion of management’s time and
attention from revenue-generating activities to compliance activities. If notwithstanding our efforts to comply with new
laws, regulations and standards, we fail to comply, regulatory authorities may initiate legal proceedings against us and our
business may be harmed.
Failure to comply with these rules might also make it more difficult for us to obtain some types of insurance,
including director and officer liability insurance, and we might be forced to accept reduced policy limits and coverage or
incur substantially higher costs to obtain the same or similar coverage. The impact of these events could also make it more
difficult for us to attract and retain qualified persons to serve on our board of directors, on committees of our board of
directors or as members of senior management.
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Item 1B. Unresolved Staff Comments
None.
Item 2. Properties
We sublease 33,019 square feet of space for our headquarters in Wayne, Pennsylvania, which we use for our
therapeutics business. The sublease has a term through October 2023. If for any reason the master lease is terminated or
expires prior to October 2023, our sublease will automatically terminate.
We also sublease 20,433 square feet of office and laboratory space in St. Louis, Missouri, which we use for our
therapeutics and contract research businesses. The sublease has an initial term through June 2029. We have the option to
extend the initial term for two additional five-year periods.
We believe that our facilities are suitable and adequate to meet our current needs.
Item 3. Legal Proceedings
From time to time we are subject to litigation and claims arising in the ordinary course of business including
intellectual property and product liability litigation, but, except as stated below, we are not currently a party to any material
legal proceedings and we are not aware of any other pending or threatened legal proceeding against us that we believe
could have a material adverse effect on our business, operating results, cash flows or financial condition.
Securities Class Action
On July 30, 2019, plaintiff Linda Rosi, or Rosi, filed a putative class action complaint captioned Rosi v. Aclaris
Therapeutics, Inc., et al. in the U.S. District Court for the Southern District of New York against us and certain of our
executive officers. The complaint alleges that the defendants violated federal securities laws by, among other things,
failing to disclose an alleged likelihood that regulators would scrutinize advertising materials related to ESKATA
(hydrogen peroxide) topical solution, 40% (w/w), or ESKATA, our non-marketed FDA-approved product, and find that the
materials minimized the risks or overstated the efficacy of the product. The complaint seeks unspecified compensatory
damages on behalf of Rosi and all other persons and entities that purchased or otherwise acquired our securities between
May 8, 2018 and June 20, 2019.
On September 5, 2019, an additional plaintiff, Robert Fulcher, or Fulcher, filed a substantially identical putative
class action complaint captioned Fulcher v. Aclaris Therapeutics, Inc., et al. in the same court against the same defendants.
On November 6, 2019, the court consolidated the Rosi and Fulcher actions, or together, the Consolidated
Securities Action, and appointed Fulcher “lead plaintiff” for the putative class.
On January 24, 2020, Fulcher filed a consolidated amended complaint in the Consolidated Securities Action,
naming two additional executive officers as defendants, extending the putative class period to August 12, 2019, and adding
allegations concerning, among other things, alleged statements and omissions throughout the putative class period
concerning ESKATA’s risks, tolerability and effectiveness. The defendants filed a motion to dismiss the consolidated
amended complaint on April 17, 2020. Fulcher filed an opposition to the defendants’ motion on June 15, 2020, and the
defendants filed a reply to such opposition on August 4, 2020. Oral argument on the pending motion to dismiss is
scheduled for February 25, 2021. The motion remains under judicial consideration.
We and the other defendants dispute plaintiffs’ claims in the Consolidated Securities Action and intend to defend
the matter vigorously.
Stockholder Derivative Action
On November 15, 2019, plaintiff Keith Allred, or Allred, filed a derivative stockholder complaint captioned Allred
v. Walker et al. in the U.S. District Court for the Southern District of New York against certain of our
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directors and executive officers. The complaint alleges that the defendants, among other things, breached their fiduciary
duties as directors and/or officers in connection with the claims alleged in the Consolidated Securities Action. The
complaint seeks, among other things, unspecified compensatory damages on behalf of our company.
On November 25, 2019, an additional plaintiff, Bruce Brown, or Brown, filed a substantially identical complaint
captioned Brown v. Walker et al. in the same court against the same defendants.
On December 12, 2019, the court consolidated the Allred and Brown actions under the caption In re Aclaris
Therapeutics, Inc. Derivative Litigation, or the Consolidated Derivative Action, and directed that future derivative cases
filed in or transferred to the court arising out of substantially the same transactions or events be similarly consolidated.
Thereafter, on January 11, 2020, the court stayed – subject to certain conditions – all deadlines in the Consolidated
Derivative Action pending resolution of the defendants’ then-anticipated motion to dismiss the Consolidated Securities
Action.
The defendants dispute plaintiffs’ claims in the Consolidated Derivative Action and intend to defend the matter
vigorously.
Item 4. Mine Safety Disclosures
Not applicable.
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Item 5. Market for Registrant’s Common Equity, Related Stockholder Matters and Issuer Purchases of Equity
Securities
PART II
Market Information for Common Stock
Our common stock is listed on the Nasdaq Global Select Market under the symbol “ACRS.”
Dividend Policy
We have never declared or paid any dividends on our common stock. We anticipate that we will retain all of our
future earnings, if any, for use in the operation and expansion of our business and do not anticipate paying cash dividends
in the foreseeable future.
Stockholders
As of January 31, 2021, we had 51,804,258 shares of common stock outstanding held by 56 holders of record. The
actual number of stockholders is greater than this number of record holders and includes stockholders who are beneficial
owners but whose shares are held in street name by brokers and other nominees. This number of holders of record also
does not include stockholders whose shares may be held in trust by other entities.
Recent Sales of Unregistered Securities
None.
Purchases of Equity Securities by the Issuer and Affiliated Parties
None.
Item 6. Selected Consolidated Financial Data
Not applicable.
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Item 7. Management’s Discussion and Analysis of Financial Condition and Results of Operations
You should read the following discussion and analysis of our financial condition and results of operations in
conjunction with the consolidated financial statements and the related notes to those statements included later in this
Annual Report. In addition to historical financial information, the following discussion contains forward-looking
statements that reflect our plans, estimates, beliefs and expectations that involve risks and uncertainties. Our actual results
and the timing of events could differ materially from those discussed in these forward-looking statements. Factors that
could cause or contribute to these differences include those discussed below and elsewhere in this Annual Report,
particularly in Part I, Item 1A. “Risk Factors,” and “Special Note Regarding Forward-Looking Statements.”
Overview
We are a clinical-stage biopharmaceutical company focused on developing novel drug candidates for immuno-
inflammatory diseases. In addition to developing our novel drug candidates, we are pursuing strategic alternatives,
including identifying and consummating transactions with third-party partners, to further develop, obtain marketing
approval for and/or commercialize our novel drug candidates.
ATI-450, an Investigational Oral MK2 Inhibitor
We submitted an Investigational New Drug Application, or IND, in April 2019 for ATI-450, an investigational
oral, novel, small molecule selective mitogen-activated protein kinase-activated protein kinase 2, or MK2, inhibitor
compound, for the treatment of rheumatoid arthritis, which was allowed by the U.S. Food and Drug Administration, or
FDA, in May 2019. MK2 is a key regulator of pro-inflammatory mediators including TNFα, IL1β, IL6, IL8 and other
essential pathogenic signals in chronic immuno-inflammatory diseases, as well as in oncology. As an oral drug candidate,
we are developing ATI-450 as a potential alternative to injectable anti-TNF/IL1/IL6 biologics and JAK inhibitors for
treating certain immuno-inflammatory diseases.
We initiated a Phase 1 single (at 10mg, 30mg, 50mg and 100mg doses) and multiple ascending (at 10mg, 30mg
and 50mg doses) dose clinical trial evaluating ATI-450 in 77 healthy subjects in August 2019 (ATI-450-PKPD-101). Final
data from this trial demonstrated that ATI-450 resulted in marked inhibition of TNFα, IL1β, IL8 and IL6. We also observed
that ATI-450 had dose-proportional pharmacokinetics with a terminal half-life of 9-12 hours in the multiple ascending dose
cohort, and had no meaningful food effect or drug-drug interaction with methotrexate. ATI-450 was generally well-
tolerated at all doses tested in the trial. The most common adverse events (reported by 2 or more subjects who received
ATI-450) were dizziness, headache, upper respiratory tract infection, constipation, abdominal pain and nausea.
ATI-450 was also evaluated at 80mg and 120mg doses twice daily in a second Phase 1 clinical trial in healthy
subjects (ATI-450-PKPD-102). Preliminary topline data from this trial showed that no dose-limiting toxicity was
observed. Ex vivo analysis of blood samples from this Phase 1 trial showed that increased cytokine inhibition was achieved
with these higher doses of ATI-450. No serious adverse events were reported and all adverse events were mild to moderate.
The most common adverse events (reported by 2 or more subjects who received ATI-450) were headache, dizziness,
nausea, parasthesia and, in the post-dosing safety follow-up phase of the trial, dry skin. These adverse events were all mild
in severity. A final analysis of this trial is underway.
Moderate to Severe Rheumatoid Arthritis
Following the completion of the first Phase 1 clinical trial, in March 2020 we initiated a 12-week, Phase 2a,
multicenter, randomized, investigator and patient-blind, sponsor-unblinded, parallel group, placebo-controlled clinical trial
to investigate the safety, tolerability, pharmacokinetics and pharmacodynamics of ATI-450 in subjects with moderate to
severe rheumatoid arthritis (ATI-450-RA-201). In the trial, 19 subjects were randomized in a 3:1 ratio (seventeen subjects
[15 in the treatment arm and two in the placebo arm] completed treatment) and received either ATI-450 at 50 mg twice
daily or placebo, in combination with methotrexate, for 12 weeks. Preliminary topline data from this trial showed that ATI-
450 demonstrated durable clinical activity, as defined by a marked and sustained reduction in DAS28-CRP and
improvement of ACR20/50/70 responses over 12 weeks. ATI-450 was generally well tolerated. All adverse events were
mild to moderate. The most common adverse events (each reported in 2 subjects) were urinary tract infection, or UTI,
elevated lipids and ventricular extrasystoles, all of which were determined to be unrelated to treatment except for one UTI.
Two subjects withdrew from the trial, one in the treatment arm and one in the placebo arm. The subject in the treatment
arm withdrew due to palpitations, which were unrelated to the trial medication, and an elevated creatine phosphokinase,
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or CPK, which was determined by the site investigator to be treatment-related. The subject in the placebo arm withdrew as
a result of prohibited medication needed to treat muscle strain. There was one non-treatment-related serious adverse event
(COVID-19) reported in the four-week safety follow-up phase of the trial in a subject who was no longer receiving
treatment.
An interim analysis (11 treatment, two placebo) of ex vivo stimulated cytokines from blood samples taken from
the treatment arm showed a marked and durable inhibition of TNFα, IL1β, IL6, and IL8 over the 12 week dosing period.
Similarly, analysis of endogenous inflammation biomarkers also demonstrated a marked and sustained inhibition of median
concentrations of hsCRP, TNFα, IL6, IL8 and MIP1β in the treatment arm over the 12-week period.
We plan to submit for publication a full analysis of the Phase 2a data in a peer-reviewed scientific journal which
will include data from other secondary and exploratory endpoints evaluated in the trial, including the four-week safety
follow-up data and a full analysis of MRI, pharmacodynamic and pharmacokinetic data. Based on the results observed in
the Phase 2a trial, we intend to progress ATI-450 into a Phase 2b trial in moderate to severe rheumatoid arthritis in the
second half of 2021.
Cryopyrin-associated Periodic Syndrome
In November 2020, we initiated a Phase 2a multicenter, open-label, single-arm clinical trial to investigate the
safety, tolerability, efficacy and pharmacodynamics of ATI-450 for the maintenance of remission in subjects with
cryopyrin-associated periodic syndrome, or CAPS, previously managed with anti-IL1 therapy (ATI-450-CAPS-201). Due
to the COVID-19 pandemic, subject enrollment in this trial was paused. As a result of the ongoing pandemic and given the
positive preliminary topline data from the ATI-450-RA-201 trial, we have decided to focus our efforts and resources on
other immuno-inflammatory diseases.
ATI-1777, an Investigational Topical “Soft” JAK 1/3 Inhibitor
In June 2020, we submitted an IND for ATI-1777, an investigational topical “soft” JAK 1/3 inhibitor compound,
for the treatment of moderate to severe atopic dermatitis. “Soft” JAK inhibitors are designed to be topically applied and
active in the skin, but rapidly metabolized and inactivated when they enter the bloodstream, which may result in low
systemic exposure.
In October 2020, we initiated a Phase 2a, multicenter, randomized, double-blind, vehicle-controlled, parallel-
group clinical trial to determine the efficacy, safety, tolerability and pharmacokinetics of ATI-1777 in subjects with
moderate to severe atopic dermatitis (ATI-1777-AD-201). We expect data to be available mid-year 2021.
ATI-2138, an Investigational ITJ Inhibitor
We are also developing ATI-2138, an investigational oral ITK/TXK/JAK3, or ITJ, inhibitor compound, as a
potential treatment for psoriasis and/or inflammatory bowel disease, which are both T-cell mediated autoimmune diseases.
The ITJ compound interrupts T cell signaling through the combined inhibition of ITK/TXK/JAK3 pathways in
lymphocytes. We expect to file an IND for ATI-2138 in the second half of 2021.
Our Other Drug Candidates
We continue to seek third-party partners for our dermatology investigational drug candidate A-101 45% Topical
Solution as a potential treatment for common warts (verruca vulgaris).
Financial Overview
Since our inception, we have incurred significant operating losses. Our net loss was $51.0 million for the year
ended December 31, 2020 and $161.4 million for the year ended December 31, 2019. As of December 31, 2020, we had an
accumulated deficit of $504.5 million. We expect to incur significant expenses and operating losses for the foreseeable
future as we advance our drug candidates from discovery through preclinical and clinical development. In addition, our
drug candidates, even if they are approved by regulatory agencies for marketing, may not achieve commercial success. We
may also not be successful in pursuing strategic alternatives, including identifying and consummating transactions with
third-party partners, to further develop, obtain marketing approval for and/or commercialize our drug
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candidates. Furthermore, we have incurred and expect to continue to incur significant costs associated with operating as a
public company, including legal, accounting, investor relations and other expenses. As a result, we will need substantial
additional funding to support our continuing operations.
We have historically financed our operations primarily with sales of equity securities and incurring indebtedness
in the form of loans from commercial lenders. In the near term, we expect to finance our operations through these and
other capital sources, including potential partnerships with other companies or other strategic transactions. We may be
unable to raise additional funds or enter into such other agreements or arrangements when needed on commercially
acceptable terms, or at all. If we fail to raise capital or enter into such agreements as, and when, needed, we may have to
significantly delay, scale back or discontinue the development of one or more of our drug candidates.
Recent Developments
January 2021 Public Offering
In January 2021, we closed a public offering in which we sold 6,306,271 shares of common stock at a price to the
public of $17.50 per share, for aggregate gross proceeds of $110.4 million. We paid underwriting discounts and
commissions of $6.6 million, and also incurred expenses of $0.3 million in connection with the offering. As a result, the
net offering proceeds received by us, after deducting underwriting discounts, commissions and offering expenses, were
$103.5 million.
Impact of COVID-19 on Our Business
The impacts of the global COVID-19 pandemic continue to evolve. We have implemented a virtual operations
strategy, including teleworking and other alternative work arrangements for our employees, intended to protect the health
and safety of our employees while enabling us to continue to develop our drug candidates and provide contract research
services to our clients. We are focused on ensuring the continuity of our operations. However, COVID-19 has caused
disruptions to our business. For example, due to the COVID-19 pandemic subject enrollment in our ATI-450-CAPS-201
trial was paused as a result of which, among other reasons, we have decided to focus our efforts and resources on other
immuno-inflammatory diseases.
If COVID-19 continues to spread, we may experience additional disruptions that could severely impact our
business, results of operations and prospects, including the timing of our development programs and our clinical trials,
including our trials of ATI-450 as a potential treatment for moderate to severe rheumatoid arthritis and ATI-1777 as a
potential treatment for moderate to severe atopic dermatitis. The extent to which the COVID-19 pandemic impacts our
business, our preclinical and clinical development and our regulatory efforts will depend on future developments that are
highly uncertain and cannot be predicted, such as the geographic spread of the disease, the duration of the outbreak, travel
restrictions, quarantines, stay-at-home orders, social distancing requirements, business closures and supply chain and other
disruptions in the United States and other countries, and the effectiveness of actions taken in the United States and other
countries to contain and treat the disease, including the administration of vaccines. Accordingly, we do not yet know the
full extent of the potential impacts on our business, our preclinical and clinical development and regulatory activities.
Acquisition and License Agreements
Agreement and Plan of Merger with Confluence
In August 2017, we entered into an Agreement and Plan of Merger, or the Confluence Agreement, with
Confluence Life Sciences, Inc. (now known as Aclaris Life Sciences, Inc.), or Confluence, Aclaris Life Sciences, Inc., our
wholly-owned subsidiary, or Merger Sub, and Fortis Advisors LLC, as representative of the equity holders of Confluence.
Pursuant to the terms of the Confluence Agreement, the Merger Sub merged with and into Confluence, with Confluence
surviving as our wholly-owned subsidiary. We paid closing consideration of $10.3 million in cash and issued 349,527
shares of our common stock with a fair value of $9.7 million to the former Confluence equity holders.
In November 2018, a development milestone specified in the Confluence Agreement was achieved, as a result of
which we paid the former Confluence equity holders $2.5 million in cash and issued 253,208 shares of our common stock
with a fair value of $2.2 million. Under the Confluence Agreement, we also agreed to pay the former Confluence equity
holders aggregate remaining contingent consideration of up to $75.0 million based upon the achievement of specified
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regulatory and commercial milestones set forth in the Confluence Agreement. In addition, we have agreed to pay the
former Confluence equity holders future royalty payments calculated as a low single-digit percentage of annual net sales,
subject to specified reductions, limitations and other adjustments, until the date that all of the patent rights for that product
have expired, as determined on a country-by-country and product-by-product basis or, in specified circumstances, ten years
from the first commercial sale of such product. In addition to the payments described above, if we sell, license or transfer
any of the intellectual property acquired from Confluence pursuant to the Confluence Agreement to a third party, we will
be obligated to pay the former Confluence equity holders a portion of any consideration received from such sale, license or
transfer in specified circumstances.
Asset Purchase Agreement with EPI Health
In October 2019, we entered into an asset purchase agreement with EPI Health, LLC, or EPI Health, pursuant to
which we sold the worldwide rights to RHOFADE (oxymetazoline hydrochloride) cream, 1%, or RHOFADE, which
included the assignment of certain licenses for related intellectual property assets, or the Disposition.
Pursuant to the asset purchase agreement, EPI Health paid us an upfront payment of $35.0 million, $1.75 million
of which was placed in escrow, and $0.2 million for inventory. In addition, EPI Health has agreed to pay us (i) potential
sales milestone payments of up to $20.0 million in the aggregate upon the achievement of specified levels of net sales of
products covered by the agreement, (ii) a specified high single-digit royalty calculated as a percentage of net sales, on a
product-by-product and country-by-country basis, until the date that the patent rights related to a particular product, such as
RHOFADE, have expired, provided, that with respect to sales of RHOFADE in any territory outside of the United
States, such royalty shall be paid on a country-by-country basis until the date that the RHOFADE patent rights in the
particular country have expired or, if later, 10 years from the date of the first commercial sale of RHOFADE in such
country and (iii) 25% of any upfront, license, milestone, maintenance or fixed payment received by EPI Health in
connection with any license or sublicense of the assets transferred in the Disposition in any territory outside of the United
States, subject to specified exceptions. In addition, EPI Health has agreed to assume our obligation to pay specified
royalties and milestone payments under certain agreements with third parties.
Asset Purchase Agreement with Allergan
In November 2018, we acquired RHOFADE, which included an exclusive license to certain intellectual property
for RHOFADE, as well as additional intellectual property, from Allergan Sales, LLC, or Allergan, pursuant to an asset
purchase agreement. At the closing of the acquisition, we paid total cash consideration of $66.1 million.
Components of Our Results of Operations
Revenue
Product Sales, net
We sold RHOFADE in the United States during the years ended December 31, 2019 and 2018. We relied on
Allergan to distribute RHOFADE on our behalf pursuant to the terms of a transition services agreement. We sold
RHOFADE to wholesalers in the United States, which, in turn, distributed it to pharmacies that ultimately filled patient
prescriptions. We also entered into, or were subject to, arrangements with third-party payors, including pharmacy benefit
managers and government agencies, as well as group purchasing organizations, or GPOs, which provided for government
mandated or privately negotiated rebates, chargebacks, and discounts with respect to the purchase of RHOFADE. We
never sold RHOFADE outside of the United States. We sold the worldwide rights to RHOFADE to EPI Health in October
2019.
During the years ended December 31, 2019 and 2018, we sold ESKATA (hydrogen peroxide) topical solution,
40% (w/w), or ESKATA, our non-marketed FDA-approved product, to one wholesaler, McKesson Specialty Care
Distribution, or McKesson, which in turn resold ESKATA to health care providers. We also entered into agreements with
two GPOs that provided for administrative fees and discounted pricing in the form of volume-based rebates and
chargebacks. We never sold ESKATA outside of the United States. We discontinued sales of ESKATA in the United States
in August 2019.
Product sales, net is presented in discontinued operations for all periods presented.
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Contract Research
We earn revenue from the provision of laboratory services to clients through Confluence, our wholly-owned
subsidiary. Contract research revenue is generally evidenced by contracts with clients which are on an agreed upon fixed-
price, fee-for-service basis and are generally billed on a monthly basis in arrears for services rendered.
We have also received revenue from grants under the Small Business Innovation Research program of the
National Institutes of Health, or NIH. During the year ended December 31, 2018, we had two active grants from NIH
related to early-stage research. There are no remaining funds available to us under the grants.
Cost of Revenue
Cost of revenue consists of the costs incurred in connection with the provision of contract research services to our
clients through Confluence. Cost of revenue primarily includes:
● employee-related expenses, which include salaries, benefits and stock-based compensation;
● outsourced professional scientific services;
● depreciation of laboratory equipment;
● facility-related costs; and
● laboratory materials and supplies used to support the services provided.
Research and Development Expenses
Research and development expenses consist of expenses incurred in connection with the discovery and
development of our drug candidates. These expenses primarily include:
● expenses incurred under agreements with contract research organizations, or CROs, as well as clinical trial
sites and consultants that conduct our clinical trials and preclinical studies, and investigator-initiated trials;
● manufacturing scale-up expenses and the cost of acquiring and manufacturing active pharmaceutical
ingredients and preclinical and clinical trial materials;
● outsourced professional scientific development services;
● medical affairs expenses related to our drug candidates;
● employee-related expenses, which include salaries, benefits and stock-based compensation;
● depreciation of manufacturing equipment;
● payments made under agreements with third parties under which we have acquired or licensed intellectual
property;
● expenses relating to regulatory activities, including filing fees paid to regulatory agencies;
● laboratory materials and supplies used to support our research activities; and
● non-cash charges related to the revaluation of contingent consideration.
Research and development activities are central to our business model. Drug candidates in later stages of clinical
development generally have higher development costs than those in earlier stages of clinical development, primarily due to
the increased size and duration of later-stage clinical trials. We expect to continue to incur research and development
expenses in the near term as we continue the clinical development of ATI-450 as a potential treatment for moderate to
severe rheumatoid arthritis and potentially other immuno-inflammatory diseases and ATI-1777 as a potential treatment for
moderate to severe atopic dermatitis, continue the development of our preclinical compounds, and continue to discover and
develop additional drug candidates. We expense research and development costs as incurred. Our direct research and
development expenses primarily consist of external costs including fees paid to CROs, consultants, investigator sites,
regulatory agencies and third parties that manufacture our preclinical and clinical trial materials, and are tracked on a
program-by-program basis. We do not allocate personnel costs, facilities or other indirect expenses, to specific research
and development programs.
The successful development of our drug candidates is highly uncertain. We cannot reasonably estimate or know
the nature, timing and costs of the efforts that will be necessary to complete the remainder of the development of, or when,
if ever, material net cash inflows may commence from any of our drug candidates. This uncertainty is due to the numerous
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risks and uncertainties associated with the duration and cost of clinical trials, which vary significantly over the life of a
project as a result of many factors, including:
● the number of clinical sites included in the trials;
● the length of time required to enroll suitable subjects;
● the number of subjects that ultimately participate in the trials;
● the number of doses subjects receive;
● the impact on the recruitment, enrollment, conduct and timing of our clinical trials due to the COVID-19
pandemic;
● the duration of subject follow-up; and
● the results of our clinical trials.
Our expenditures are subject to additional uncertainties, including the preparation of regulatory filings for our
drug candidates, and the expense of filing, prosecuting, defending and enforcing any patent claims or other intellectual
property rights. We may obtain unexpected results from our clinical trials or other development activities. We may elect to
discontinue, delay or modify the development, including clinical trials, of some drug candidates or focus on others. A
change in the outcome of any of these variables with respect to the development of a drug candidate could mean a
significant change in the costs and timing associated with the development of that drug candidate. For example, if the FDA
or other regulatory authorities were to require us to conduct clinical trials beyond those that we currently anticipate, or if
we experience significant delays in enrollment in any of our clinical trials, we could be required to expend significant
additional financial resources and time on the completion of clinical development.
General and Administrative Expenses
General and administrative expenses consist principally of salaries and related costs, including stock-based
compensation, for personnel in executive, administrative, finance and legal functions. General and administrative expenses
also include facility-related costs, patent filing and prosecution costs, professional fees for legal, auditing and tax services,
investor relations costs, insurance costs, travel expenses, as well as a milestone payment under a third party agreement.
Other Income (Expense), net
Other income (expense), net primarily consists of interest earned on our cash, cash equivalents and marketable
securities, interest expense related to our debt obligations, and gains and losses on transactions denominated in foreign
currencies.
Critical Accounting Policies and Significant Judgments and Estimates
Our consolidated financial statements are prepared in accordance with generally accepted accounting principles in
the United States. The preparation of our consolidated financial statements and related disclosures requires us to make
estimates and judgments that affect the reported amounts of assets and liabilities, disclosure of contingent assets and
liabilities at the date of the financial statements, and the reported amounts of expenses during the reported period. We base
our estimates on historical experience, known trends and events and various other factors that we believe to be reasonable
under the circumstances, the results of which form the basis for making judgments about the carrying values of assets and
liabilities that are not readily apparent from other sources. We evaluate our estimates and judgments on an ongoing basis.
Our actual results may differ from these estimates under different assumptions and conditions.
While our significant accounting policies are described in more detail in the notes to our consolidated financial
statements appearing elsewhere in this Annual Report on Form 10-K, we believe that the following accounting policies are
those most critical to the judgments and estimates used in the preparation of our consolidated financial statements.
Revenue Recognition
We account for revenue in accordance with Accounting Standards Codification, or ASC, Topic 606, Revenue from
Contracts with Customers. Under ASC Topic 606, revenue is recognized when a customer obtains control of promised
goods or services in an amount that reflects the consideration to which we expect to be entitled in exchange for
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those goods or services.
To determine revenue recognition in accordance with ASC Topic 606, we perform the following five steps: (i)
identify the contract(s) with a customer, (ii) identify the performance obligations in the contract, (iii) determine the
transaction price, (iv) allocate the transaction price to the performance obligations in the contract, and (v) recognize
revenue when (or as) performance obligations are satisfied. We recognize revenue when collection of the consideration we
are entitled to under a contract with a customer is probable. At contract inception, we assess the goods or services
promised within a contract with a customer to identify the performance obligations, and to determine if they are distinct.
We recognize revenue that is allocated to each distinct performance obligation when (or as) that performance obligation is
satisfied. We only recognize revenue when collection of the consideration we are entitled to under a contract with a
customer is probable.
Product Sales, net
We recognized revenue from product sales at the point the customer obtained control, which generally occurred
upon delivery. We also included estimates of variable consideration in the same period revenue was recognized.
Components of variable consideration included trade discounts and allowances, product returns, government rebates,
discounts and rebates, other incentives such as patient co-pay assistance, and other fee for service amounts. Variable
consideration was recorded on the consolidated balance sheet as either a reduction of accounts receivable, if payable to a
customer, or as a current liability, if payable to a third-party other than a customer. We considered all relevant information
when estimating variable consideration such as contractual and statutory requirements, specific known market events and
trends, industry data and forecasted customer buying and payment patterns. The amount of net revenue that can be
recognized is constrained by estimates of variable consideration which are included in the transaction price. Payment
terms with customers did not exceed one year and, therefore, we did not account for a financing component in our
arrangements. We expensed incremental costs of obtaining a contract with a customer, including sales commissions, when
incurred as the period of benefit was less than one year.
Trade Discounts and Allowances - We provided customers with trade discounts, rebates, allowances and/or other
incentives. We recorded estimates for these items as a reduction of revenue in the same period the revenue was recognized.
Government and Payor Rebates – We contracted with, or were subject to arrangements with, certain third-party
payors, including pharmacy benefit managers and government agencies, for the payment of rebates with respect to
utilization of our commercial products. We also entered into agreements with GPOs that provided for administrative fees
and discounted pricing in the form of volume-based rebates. We were also subject to discount and rebate obligations under
state Medicaid programs and Medicare. We recorded estimates for these discounts and rebates as a reduction of revenue in
the same period the revenue was recognized.
Other Incentives – We maintained a co-pay assistance program which was intended to provide financial assistance
to qualified commercially-insured patients with prescription drug co-payments required by third-party payors. We
estimated and recorded accruals for these incentives as a reduction of revenue in the period the revenue was recognized.
Our estimated amounts for co-pay assistance were based upon the number of claims and the cost per claim that we
expected to receive associated with product that had been sold to customers but remained in the distribution channel at the
end of each reporting period.
Product Returns - Consistent with industry practice, we have a product returns policy for RHOFADE which may
provide customers a right of return for product purchased within a specified period prior to and subsequent to the product’s
expiration date. The right of return lapses upon shipment of the product to a patient. We recorded an estimate for the
amount of product which may be returned as a reduction of revenue in the period the related revenue was recognized. Our
estimates for product returns were based upon available industry data and our own sales information, including visibility
into the inventory remaining in the distribution channel. There is no return liability associated with sales of ESKATA as
we had a no returns policy for ESKATA.
Product sales, net is presented in discontinued operations for all periods presented.
Contract Research
Revenue related to laboratory services is generally recognized as the laboratory services are performed, based
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upon the rates specified in the contracts. Under ASC Topic 606, we elected to apply the “right to invoice” practical
expedient when recognizing contract research revenue and as such, recognize revenue in the amount which we have the
right to invoice. ASC Topic 606 also provides an optional exemption, which we have elected to apply, from disclosing
remaining performance obligations when revenue is recognized from the satisfaction of the performance obligation in
accordance with the “right to invoice” practical expedient.
We recognize revenue related to grants as amounts become reimbursable under each grant, which is generally
when research is performed, and the related costs are incurred.
Other Revenue
Licenses of Intellectual Property – We recognize revenue received from non-refundable, upfront fees related to the
licensing of intellectual property when the intellectual property is determined to be distinct from the other performance
obligations identified in the arrangement, the license has been transferred to the customer, and the customer is able to use
and benefit from the license.
Milestone Payments – At the inception of each arrangement that includes milestone payments, we evaluate
whether the milestones are considered probable of being reached and estimate the amount to be included in the transaction
price using the most likely amount method. If it is probable that a significant revenue reversal would not occur, the
associated milestone value is included in the amount allocated to the license of intellectual property. Milestone payments
that are not within our control or the control of the customer, such as regulatory approvals, are not considered probable of
being achieved until those approvals are received.
Intangible Assets
Our intangible assets include both definite-lived and indefinite-lived assets. Our definite-lived intangible assets
consist of a drug discovery platform acquired through the acquisition of Confluence, and prior to the disposition in 2019,
also included the intellectual property rights related to RHOFADE. Definite-lived intangible assets are amortized over
their estimated useful life based on the pattern over which the intangible assets are consumed or otherwise used up. If that
pattern cannot be reliably determined, the straight-line method of amortization is used. Our indefinite-lived intangible
assets consist of an in-process research and development, or IPR&D, drug candidate also acquired through the acquisition
of Confluence. IPR&D assets are considered indefinite-lived until the completion or abandonment of the associated
research and development efforts. The cost of IPR&D assets is either amortized over their estimated useful life beginning
when the underlying drug candidate is approved and launched commercially, or expensed immediately if development of
the drug candidate is abandoned.
Definite-lived intangible assets are tested for impairment when events or changes in circumstances indicate that
the carrying value of the asset may not be recoverable. Indefinite-lived intangible assets are tested for impairment at least
annually, which we perform during the fourth quarter, or when indicators of an impairment are present. We recognize an
impairment loss when and to the extent that the estimated fair value of an intangible asset is less than its carrying value.
During the year ended December 31, 2019, we performed an impairment analysis of the RHOFADE intangible
asset due to our decision to discontinue commercial operations and actively seek a commercialization partner for
RHOFADE. Our impairment analysis, which primarily utilized a third-party indication of fair value, resulted in a fair value
for the RHOFADE intangible asset which was less than its carrying value. As a result, we recorded an impairment charge
of $27.6 million to adjust the carrying value of the RHOFADE intangible asset to its net realizable value.
Goodwill
Goodwill is not amortized, but rather is subject to testing for impairment at least annually, which we perform
either during the fourth quarter or when indicators of an impairment are present. We consider each of our operating
segments, therapeutics and contract research, to be a reporting unit since this is the lowest level for which discrete financial
information is available. The impairment test is a qualitative assessment based upon the then current facts and
circumstances related to operations of the reporting unit. If our qualitative assessment indicates an impairment may be
present, we would perform the required quantitative analysis and an impairment charge would be recognized to the extent
that the estimated fair value of the reporting unit is less than its carrying amount. However, any loss recognized would not
exceed the total amount of goodwill allocated to that reporting unit.
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During the year ended December 31, 2019, we performed an impairment analysis due to a decline in our stock
price, which was considered a triggering event to evaluate goodwill for impairment. Our impairment analysis, which
utilized a market approach, noted that our stock price, including a reasonable control premium, resulted in a fair value for
the therapeutics reporting unit which was less than its carrying value. As a result, we recorded an impairment charge of
$18.5 million.
Leases
Leases represent a company’s right to use an underlying asset and a corresponding obligation to make payments to
a lessor for the right to use those assets. We evaluate leases at their inception to determine if they are an operating lease or
a finance lease. A lease is accounted for as a finance lease if it meets one of the following five criteria: the lease has a
purchase option that is reasonably certain of being exercised, the present value of the future cash flows are substantially all
of the fair market value of the underlying asset, the lease term is for a significant portion of the remaining economic life of
the underlying asset, the title to the underlying asset transfers at the end of the lease term, or if the underlying asset is of
such a specialized nature that it is expected to have no alternative uses to the lessor at the end of the term. Leases that do
not meet the finance lease criteria are accounted for as an operating lease.
We recognize assets and liabilities for leases at their inception based upon the present value of all payments due
under the lease. We use an implicit interest rate to determine the present value of finance leases, and our incremental
borrowing rate to determine the present value of operating leases. We determine incremental borrowing rates by
referencing collateralized borrowing rates for debt instruments with terms similar to the respective lease. We recognize
expense for operating and finance leases on a straight-line basis over the term of each lease, and interest expense related to
finance leases is recognized over the lease term based on the effective interest method. We include estimates for any
residual value guarantee obligations under our leases in lease liabilities recorded on our consolidated balance sheet.
Right-of-use assets are included in other assets and property and equipment, net on our consolidated balance sheet
for operating and finance leases, respectively. Obligations for lease payments are included in current portion of lease
liabilities and other liabilities on our consolidated balance sheet for both operating and finance leases.
Contingent Consideration
We initially recorded a contingent consideration liability related to future potential payments resulting from the
acquisition of Confluence based upon the achievement of certain development, regulatory and commercial milestones, as
well as future projected sales performance, at its estimated fair value on the date of acquisition. The ultimate amount of
future payments, if any, is based on criteria such as sales performance and the achievement of certain regulatory and sales
milestones. We estimate the fair value of the contingent consideration liability related to the achievement of regulatory
milestones by assigning an achievement probability to each potential milestone and discounting the associated cash
payment to its present value using a credit-risk-adjusted interest rate. We estimate the fair value of the contingent
consideration liability associated with sales milestones and royalties by estimating future sales levels, assigning an
achievement probability and discounting the associated cash payment amounts to their present values using a risk-adjusted
rate of return. Significant assumptions used in our estimates include the probability of success of both achieving regulatory
milestones and commencing commercialization, which are based upon an asset’s current stage of development and ranged
between 4% and 15%. We evaluate fair value estimates of contingent consideration liabilities on a quarterly basis. Any
change in fair value reflects new information about the likelihood of the payment of the contingent consideration and the
passage of time. For example, if the timing of the development of an acquired drug candidate, or the size of potential
commercial opportunities related to an acquired drug candidate, differ from our assumptions, then the fair value of
contingent consideration would be adjusted accordingly. Future changes in the fair value of the contingent consideration, if
any, will be recorded as income or expense in our consolidated statement of operations.
During the year ended December 31, 2020, we updated our assumptions for contingent consideration related to the
acquisition of Confluence as a result of the successful completion of a Phase 1 clinical trial for ATI-450 and the submission
and allowance of an IND for ATI-1777, which resulted in a charge of $2.4 million. During the year ended December 31,
2019, we updated our assumptions for contingent consideration related to the acquisition of Confluence as a result of the
submission and allowance of an IND for ATI-450, which resulted in a charge of $0.7 million.
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Research and Development Expenses
As part of the process of preparing our consolidated financial statements, we are required to estimate our research
and development expenses. This process involves reviewing open contracts and purchase orders, communicating with our
applicable personnel to identify services that have been performed on our behalf and estimating the level of service
performed and the associated cost incurred for the service when we have not yet been invoiced or otherwise notified of
actual costs. The majority of our preclinical development activities and clinical trials are performed pursuant to quotes and
contracts with multiple vendors, including research institutions and CROs, that conduct and manage such activities on our
behalf. Many of the contracts with our vendors require advance payments; while others invoice us in arrears for services
performed, or on a pre-determined schedule, or upon the successful enrollment of subjects, or when contractual milestones
are met. We record expenses for preclinical development activities and clinical trials based upon estimates of the total cost
of the services to be provided by the vendor and the time period over which the vendor is to perform those services.
Estimates of research and development expenses included in our consolidated financial statements are based on facts and
circumstances known to us at that time. The financial terms of our agreements are subject to negotiation, vary from
contract to contract, and may result in uneven payment flows. There may be times when payments made to a vendor
exceed the level of services provided, resulting in a prepayment for work to be performed. We may confirm the accuracy
of our estimates with the service providers, or make adjustments to our estimates based upon new or updated facts and
circumstances, as necessary. For example, if the timing and/or cost of services to be performed is materially different from
our previous estimates, we would make a prospective adjustment for the change in our estimates in the period in which we
become aware of the new cost and/or timing. Although we do not expect our estimates to be materially different from
actual amounts incurred, our understanding of the status and timing of services performed relative to the actual status and
timing of services performed may vary and may result in reporting amounts that are too high or too low in any particular
period. To date, we have not made any material adjustments to our estimates of research and development expenses.
Stock-Based Compensation
We measure the compensation expense of stock-based awards granted to employees and directors using the grant
date fair value of the award. We have issued stock options and restricted stock unit, or RSU, awards with service-based
vesting conditions, as well as with performance-based vesting conditions. We have not issued awards that include market-
based conditions. For service-based awards, we recognize stock-based compensation expense on a straight-line basis over
the requisite service period. For performance-based awards, we recognize stock-based compensation expense on a
straight-line basis over the requisite service period beginning in the period that it becomes probable the performance
conditions will occur. At each balance sheet date, we evaluate whether any performance conditions related to a
performance-based award have changed. The effect of any change in performance conditions would be recognized as a
cumulative catch-up adjustment in the period such change occurs, and any remaining unrecognized compensation expense
would be recognized on a straight-line basis over the remaining requisite service period. The impact of forfeitures is
recognized in the period in which they occur.
We measure the compensation expense of stock-based awards granted to consultants using the grant date fair
value of the award. We recognize compensation expense over the period during which services are rendered by the
consultant.
We estimate the fair value of each stock option grant using the Black-Scholes option-pricing model. We estimate
expected volatility based on historical volatility of a set of peer companies, which are publicly traded, and we expect to
continue to do so until we have adequate historical data regarding the volatility of our own publicly-traded stock price. The
expected term of our stock options has been determined using the “simplified” method for awards that qualify as “plain
vanilla” options. The expected term of stock options we granted to non-employees is equal to the contractual term of the
option award. The risk-free interest rate is determined by reference to the U.S. Treasury yield curve in effect at the time of
grant of the award for time periods approximately equal to the expected term of the award. We use an expected dividend
yield of zero because we have not paid cash dividends to date, and have no intention of paying cash dividends in the future.
The fair value of each RSU is measured using the closing price of our common stock on the date of grant.
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Income Taxes
Since our inception, we have not recorded U.S. federal or state income tax benefits for the net operating losses we
have incurred in each year or for our earned research and development tax credits, due to our uncertainty of realizing a
benefit from those items.
Results of Operations
Comparison of Years Ended December 31, 2020 and 2019
(In thousands)
Revenues:
Contract research
Other revenue
Total revenue
Costs and expenses:
Cost of revenue
Research and development
General and administrative
Goodwill impairment
Total costs and expenses
Loss from operations
Other expense, net
Loss from continuing operations before income taxes
Income tax benefit
Loss from continuing operations
Income (loss) from discontinued operations, net of tax
Net loss
Revenue
Year Ended December 31,
2020
2019
Change
$
$
5,786
696
6,482
4,227
—
4,227
$
1,559
696
2,255
5,133
31,731
20,530
—
57,394
(50,912)
4,055
64,899
27,827
18,504
115,285
(111,058)
1,078
(33,168)
(7,297)
(18,504)
(57,891)
60,146
(424)
(51,336)
(182)
(51,154)
139
2,060
62,206
(182)
62,388
47,951
$ (51,015) $ (161,354) $ 110,339
(2,484)
(113,542)
—
(113,542)
(47,812)
Contract research revenue was $5.8 million and $4.2 million for the years ended December 31, 2020 and 2019,
respectively, and was comprised primarily of fees earned from the provision of laboratory services to our clients through
Confluence. Other revenue for the year ended December 31, 2020 consisted of $0.7 million of royalties earned on net sales
of RHOFADE pursuant to the asset purchase agreement with EPI Health.
Cost of Revenue
Cost of revenue was $5.1 million and $4.1 million for the years ended December 31, 2020 and 2019, respectively,
and related to providing laboratory services to our clients through Confluence. The increase in cost of revenue was driven
primarily by increased personnel and lab supply costs as well as an increase in overhead expenses.
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Research and Development Expenses
The following table summarizes our research and development expenses:
Year Ended
December 31,
(In thousands)
ATI-450
ATI-1777
ATI-2138
Other JAK inhibitors
A-101 45% Topical Solution
Discovery
Other research and development
Personnel
Stock-based compensation
Milestones and licensing
Contingent consideration
Total research and development expenses
ATI-450
Change
2020
$ 8,268 $
3,993
2,412
265
611
2,141
1,564
7,165
2,919
—
2,393
$ 31,731
2019
7,839 $
3,612
1,771
11,102
13,309
4,152
2,177
9,612
5,091
5,500
734
$ 64,899
429
381
641
(10,837)
(12,698)
(2,011)
(613)
(2,447)
(2,172)
(5,500)
1,659
$ (33,168)
Research and development expenses for ATI-450 during the year ended December 31, 2020 primarily consisted of
costs associated with multiple clinical trials, including a Phase 2a trial in subjects with moderate to severe rheumatoid
arthritis. ATI-450 expenses during the year ended December 31, 2019 primarily consisted of preclinical development
activities and activities related to a Phase 1 clinical trial that was completed in January 2020. ATI-450 expenses increased
during the year ended December 31, 2020 due to an increase in costs associated with various ongoing clinical trials,
partially offset by lower preclinical development activities.
ATI-1777
Expenses for ATI-1777 were higher during the year ended December 31, 2020 primarily due to costs associated
with a Phase 2a clinical trial in subjects with moderate to severe atopic dermatitis which commenced in 2020, partially
offset by lower preclinical development activities.
ATI-2138
Expenses for ATI-2138 were higher during the year ended December 31, 2020 primarily due to preclinical
development activities and IND-enabling studies.
Other JAK inhibitors
The decrease in expenses related to other JAK inhibitors primarily resulted from lower expenses in the year ended
December 31, 2020 following the completion of multiple Phase 2 clinical trials of our legacy JAK inhibitors, ATI-501 and
ATI-502, in 2019.
A-101 45% Topical Solution
Expenses related to A-101 45% Topical Solution decreased primarily due to lower expenses in the year ended
December 31, 2020 following the completion of our Phase 3 clinical trials in 2019.
Discovery and other research and development
Research and development expenses related to discovery decreased during the year ended December 31, 2020 as
our discovery-stage assets matured to clinical-stage assets. As a result of this transition, we focused fewer resources on our
discovery-stage assets in 2020 compared to 2019. Other research and development expenses, which primarily included
expenses for medical affairs activities, were lower primarily due to a decrease in activities for our A-101 45% Topical
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Solution, ATI-501 and ATI-502 clinical trials that were completed in 2019. Additionally, travel expenses were lower
during the year ended December 31, 2020 due to the COVID-19 pandemic.
Personnel and stock-based compensation
Personnel expenses and stock-based compensation decreased due to lower headcount primarily resulting from the
restructuring announced in September 2019.
Milestones and licensing and contingent consideration
In 2019, we recorded $5.5 million of expenses for payments under a license agreement upon the achievement of a
development milestone and a fee related to a contract amendment. The change in contingent consideration during the year
ended December 31, 2020 was the result of updates to our assumptions as a result of the completion of a successful Phase 1
clinical trial for ATI-450 and the submission and allowance of an IND for ATI-1777, while the change in contingent
consideration during the year ended December 31, 2019 was the result of updates to our assumptions as a result of the
submission and allowance of an IND for ATI-450.
General and Administrative Expenses
The following table summarizes our general and administrative expenses:
Year Ended
December 31,
(In thousands)
Personnel
Professional and legal fees
Facility and support services
Other general and administrative
Stock-based compensation
Total general and administrative expenses
Change
2020
$ 5,671 $
3,671
1,743
2,103
7,342
$ 20,530
2019
8,342 $ (2,671)
(324)
3,995
(831)
2,574
(525)
2,628
(2,946)
10,288
$ (7,297)
$ 27,827
Personnel and stock-based compensation expenses decreased due to lower headcount primarily resulting from the
restructuring announced in September 2019. Professional and legal fees included accounting, legal, investor relations and
corporate communication costs, as well as legal fees related to patents and current lawsuits described in this report.
Professional and legal fees were lower year-over-year primarily due to lower corporate legal fees. Facility and support
services included general office expenses, information technology costs and other expenses, and decreased primarily due to
lower operational and other overhead costs. Other general and administrative expenses was lower primarily due to reduced
travel-related activities in light of the COVID-19 pandemic.
Goodwill Impairment
During the year ended December 31, 2019, we performed an impairment analysis due to a decline in our stock
price. Our impairment analysis noted that the fair value for the therapeutics reporting unit was less than its carrying
value. As a result, we recorded an impairment charge of $18.5 million.
Other Expense, net
The $2.1 million decrease in other expense, net was primarily due to lower interest expense and fees associated
with outstanding debt balances.
Loss from Discontinued Operations
The decrease in loss from discontinued operations was due to the divestiture of RHOFADE (see Note 3 to the
consolidated financial statements included in this report for additional information). We recorded income from
discontinued operations of $0.1 million during the year ended December 31, 2020 which consisted of $0.4 million of
RHOFADE product sales due to a reversal of previously accrued product sales-related reserves, partially offset by $0.3
million of expenses.
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Comparison of Years Ended December 31, 2019 and 2018
(In thousands)
Revenues:
Contract research
Other revenue
Total revenue
Costs and expenses:
Cost of revenue
Research and development
General and administrative
Goodwill impairment
Total costs and expenses
Loss from operations
Other income (expense), net
Loss from continuing operations before income taxes
Income tax benefit
Loss from continuing operations
Loss from discontinued operations, net of tax
Net loss
Revenue
Year Ended December 31,
2019
2018
Change
$
$
4,227
—
4,227
4,651
1,500
6,151
$
(424)
(1,500)
(1,924)
4,055
64,899
27,827
18,504
115,285
(111,058)
4,329
60,841
25,761
—
90,931
(84,780)
(274)
4,058
2,066
18,504
24,354
(26,278)
(2,484)
(113,542)
—
(113,542)
(47,812)
(5,160)
(31,438)
—
(31,438)
2,822
$ (161,354) $ (132,738) $ (28,616)
2,676
(82,104)
—
(82,104)
(50,634)
Contract research revenue was $4.2 million and $4.7 million for the years ended December 31, 2019 and 2018,
respectively, and was comprised primarily of fees earned from the provision of laboratory services to our clients through
Confluence. Other revenue for the year ended December 31, 2018 related to payments associated with a license agreement
and consisted of an upfront payment of $1.0 million and $0.5 million earned upon the achievement of a specified
regulatory milestone.
Cost of Revenue
Cost of revenue was $4.1 million and $4.3 million for the years ended December 31, 2019 and 2018, respectively,
and related to providing laboratory services to our clients through Confluence.
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Research and Development Expenses
The following table summarizes our research and development expenses:
Year Ended
December 31,
(In thousands)
ATI-450
ATI-1777
ATI-2138
Other JAK inhibitors
A-101 45% Topical Solution
Discovery
Other research and development
Personnel
Stock-based compensation
Milestones and licensing
Contingent consideration
Total research and development expenses
ATI-450
2019
$ 7,839 $
3,612
1,771
11,102
13,309
4,152
2,177
9,612
5,091
5,500
734
$ 64,899
2018
4,068 $
8
—
22,449
10,114
4,082
4,035
8,332
6,481
—
1,272
$ 60,841
$
Change
3,771
3,604
1,771
(11,347)
3,195
70
(1,858)
1,280
(1,390)
5,500
(538)
4,058
The increase in expenses for ATI-450 resulted primarily from preclinical development activities as well as a Phase
1 clinical trial, which was initiated and near completion during the year ended December 31, 2019.
ATI-1777
Expenses related to ATI-1777 increased during the year ended December 31, 2019 due to higher preclinical
activities and IND-enabling preclinical studies.
ATI-2138
Expenses for ATI-2138 were higher during the year ended December 31, 2019 primarily due to preclinical
development activities.
Other JAK inhibitors
Development expenses related to other JAK inhibitors decreased primarily due to lower expenses in the year
ended December 31, 2019 following the completion of multiple Phase 2 clinical trials of ATI-501 and ATI-502 in 2019.
A-101 45% Topical Solution
Expenses related to A-101 45% Topical Solution increased primarily due to higher expenses associated with our
two pivotal Phase 3 clinical trials, which were initiated during 2018 and were completed in 2019.
Discovery and other research and development
Discovery expenses were consistent year-over-year and reflected our efforts to progress various programs to
candidate selection and clinical-stage. The decrease in other research and development expenses was primarily due to
lower costs related to legacy dermatology assets and lower travel expenses.
Personnel and stock-based compensation
The increase in personnel expenses, which includes restructuring expenses, was primarily the result of increased
headcount prior to our restructuring in 2019. Restructuring expenses primarily included the cost of termination benefits
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given to employees that were involuntarily terminated during the year ended December 31, 2019. The decrease in stock-
based compensation expense was primarily the result of forfeitures by certain employees during 2019.
Milestones and licensing and contingent consideration
In 2019, we recorded $5.5 million of expenses for payments under a license agreement upon the achievement of a
development milestone and a fee related to a contract amendment. The change in contingent consideration during the year
ended December 31, 2019 was the result of updates to our assumptions as a result of the submission and allowance of an
IND for ATI-450. The change in contingent consideration during the year ended December 31, 2018 was the result of
updates to our assumptions related to ATI-1777 that reflected the achievement of a specified development milestone in
November 2018 under the Confluence Agreement.
General and Administrative Expenses
The following table summarizes our general and administrative expenses:
Year Ended
December 31,
(In thousands)
Personnel
Professional and legal fees
Facility and support services
Other general and administrative
Stock-based compensation
Total general and administrative expenses
2019
$ 8,342 $
3,995
2,574
2,628
10,288
$ 27,827
2018
7,006 $
5,091
2,349
1,998
9,317
$ 25,761
$
Change
1,336
(1,096)
225
630
971
2,066
Personnel expenses, which includes restructuring expenses, and stock-based compensation expenses increased due
to higher headcount prior to our restructuring. Restructuring expenses primarily include the costs of termination benefits
given to employees that were involuntarily terminated during the year ended December 31, 2019. Professional and legal
fees included accounting, legal, investor relations and corporate communication costs, as well as legal fees related to
patents and business development. The decrease in professional and legal fees was primarily related to lower corporate
communications costs as well as lower legal costs incurred related to patents. Facility and support services included
general office expenses and information technology costs, which increased due to our new office and laboratory facility in
St. Louis, which we moved into in 2019, as well as higher headcount prior to our restructuring. Other general and
administrative expenses included insurance, travel costs, depreciation and other miscellaneous expenses.
Goodwill Impairment
During the year ended December 31, 2019, we performed an impairment analysis due to a decline in our stock
price. Our impairment analysis noted that the fair value for the therapeutics reporting unit was less than its carrying
value. As a result, we recorded an impairment charge of $18.5 million.
Other Income (Expense), net
The $5.2 million decrease in other income (expense), net was primarily due to higher interest expense incurred on
outstanding debt balances.
Loss from Discontinued Operations
In September 2019, we announced the completion of a strategic review and our decision to refocus on our
immuno-inflammatory development programs and to actively seek partners for our commercial products. The decrease in
loss from discontinued operations was driven by higher product sales, net partially offset by higher expenses (see Note 18
to the consolidated financial statements included in this report for additional information).
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Liquidity and Capital Resources
Since our inception, we have incurred net losses and negative cash flows from our operations. Prior to our
acquisition of Confluence in August 2017, we did not generate any revenue. We have financed our operations over the last
several years primarily through sales of our equity securities in public offerings and a private placement transaction, as well
as debt financings. We may engage in additional debt and equity financing transactions in order to raise additional funds.
In addition, to the extent we are able to consummate transactions with potential third-party partners to further develop,
obtain marketing approval for and/or commercialize our drug candidates, we may receive upfront payments, milestone
payments or royalties from such arrangements that would increase our liquidity.
As of December 31, 2020, we had cash, cash equivalents and marketable securities of $54.1 million. Subsequent
to December 31, 2020, we raised net proceeds of $103.5 million through a public offering of common stock. Cash in
excess of immediate requirements is invested in accordance with our investment policy, primarily with a view towards
liquidity and capital preservation.
We currently have no ongoing material financing commitments, such as lines of credit or guarantees, that are
expected to affect our liquidity, other than our term loan facility, lease obligations, and contingent obligations under the
Confluence Agreement, which is summarized above under “Overview—Acquisition and License Agreements.”
Equity Financing
January 2021 Public Offering
In January 2021, we closed a public offering in which we sold 6,306,271 shares of common stock at a price to the
public of $17.50 per share, for aggregate gross proceeds of $110.4 million. We paid underwriting discounts and
commissions of $6.6 million, and we also incurred expenses of $0.3 million in connection with the offering. As a result,
the net offering proceeds received by us, after deducting underwriting discounts, commissions and offering expenses, were
$103.5 million.
Equity Purchase Agreement with Lincoln Park Capital Fund, LLC
In August 2020, we entered into a purchase agreement, or the Purchase Agreement, with Lincoln Park. The
Purchase Agreement provided that, upon the terms and subject to the conditions and limitations set forth therein, we could
sell to Lincoln Park, at our discretion, up to $15.0 million of shares of our common stock over the 36-month term of the
Purchase Agreement. Upon execution of the Purchase Agreement, we issued 121,584 shares of our common stock to
Lincoln Park as commitment shares in accordance with the closing conditions contained within the Purchase Agreement.
The commitment shares were valued using the closing price of our common stock on the effective date of the Purchase
Agreement resulting in an aggregate fair value of $0.3 million. Through December 31, 2020, we sold 2,111,170 shares of
our common stock to Lincoln Park under the Purchase Agreement for net proceeds of $7.7 million. We did not sell any
additional shares prior to terminating the Purchase Agreement in January 2021 in connection with the public offering of
common stock described above.
October 2018 Public Offering
In October 2018, we closed a public offering in which we sold 9,941,750 shares of common stock at a price to the
public of $10.75 per share, for aggregate gross proceeds of $106.9 million. We paid underwriting discounts and
commissions of $6.4 million to the underwriters, and we incurred expenses of $0.3 million in connection with the offering.
As a result, the net offering proceeds received by us, after deducting underwriting discounts, commissions and offering
expenses, were $100.2 million.
Debt Financing
Loan and Security Agreement with Silicon Valley Bank
In March 2020, we entered into a Loan and Security Agreement with SVB. The Loan and Security Agreement
provides for $11.0 million in term loans, of which we borrowed the entire amount on March 30, 2020. The Loan and
Security Agreement is secured by substantially all of our assets other than intellectual property.
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The term loan repayment schedule provides for interest only payments beginning April 1, 2020 and continuing
through March 1, 2022, followed by 24 consecutive equal monthly installments of principal, plus monthly payments of
accrued interest, starting on April 1, 2022 and continuing through the maturity date of March 1, 2024. All outstanding
principal and accrued and unpaid interest will be due and payable on the maturity date. The Loan and Security Agreement
provides for an annual interest rate equal to the greater of (i) the prime rate then in effect as reported in The Wall Street
Journal plus 2% and (ii) 6.75%.
The Loan and Security Agreement includes a final payment fee equal to 5% of the original principal amount
borrowed. We have the option to prepay the outstanding balance of the term loans in full, subject to a prepayment
premium of (i) 3% of the original principal amount borrowed for any prepayment on or prior to the first anniversary of
March 30, 2020, (ii) 2% of the original principal amount borrowed for any prepayment after the first anniversary and on or
before the second anniversary of March 30, 2020 or (iii) 1% of the original principal amount borrowed for any prepayment
after the second anniversary of March 30, 2020 but before March 1, 2024.
The Loan and Security Agreement contains a customary covenant that limits our ability, subject to specified
exceptions, to incur additional indebtedness without the prior written consent of SVB.
Loan and Security Agreement with Oxford Finance LLC
In October 2018, we entered into a Loan and Security Agreement with Oxford. The Loan and Security
Agreement provided for up to $65.0 million in term loans, of we borrowed $30.0 million in October 2018. In October
2019, we repaid in full the $30.0 million that was outstanding under the Loan and Security Agreement, together with all
accrued and unpaid interest and fees.
Cash Flows
The following table summarizes our cash flows for each of the periods presented:
(In thousands)
Net cash used in operating activities
Net cash provided by investing activities
Net cash provided by (used in) financing activities
Net increase (decrease) in cash, cash equivalents and restricted cash
Operating Activities
2020
Year Ended December 31,
2019
$ (38,633) $ (96,445) $ (100,811)
9,367
105,679
128,261
(30,316)
36,817
$ (13,874) $ (21,082) $
6,387
18,372
2018
During the year ended December 31, 2020, operating activities used $38.6 million of cash primarily resulting
from our net loss of $51.0 million and changes in our operating assets and liabilities of $2.4 million, partially offset by non-
cash adjustments of $14.7 million. Net cash used by changes in our operating assets and liabilities during the year ended
December 31, 2020 consisted of a $8.9 million decrease in accounts payable and accrued expenses, partially offset by a
$4.9 million decrease in accounts receivable and a $1.7 million decrease in prepaid expenses and other assets. The net
decrease in accounts payable and accrued expenses was primarily driven by lower overall operating expenses as well as
expenses incurred, but not yet paid, as of December 31, 2020 compared to the year ended December 31, 2019. The
decrease in accounts receivable was primarily the result of cash received from Allergan related to sales of RHOFADE
made during the year ended December 31, 2019. The decrease in prepaid expenses and other assets was primarily due to
amortization of the premiums for our corporate insurance policies, which we expense equally over the policy term,
partially offset by prepayment of policies for 2021. Non-cash expenses of $14.7 million were primarily composed of
stock-based compensation expense of $11.2 million, a charge of $2.4 million related to the change in the fair value of
contingent consideration and depreciation and amortization expense of $1.3 million.
During the year ended December 31, 2019, operating activities used $96.4 million of cash primarily resulting
from our net loss of $161.4 million and changes in our operating assets and liabilities of $2.7 million, partially offset by
non-cash adjustments of $67.6 million. Net cash used by changes in our operating assets and liabilities during the year
ended December 31, 2019 consisted of a $5.1 million decrease in accounts payable and accrued expenses and a $0.8
million increase in accounts receivable, which were partially offset by a $3.2 million decrease in prepaid expenses and
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other assets. The decrease in accounts payable and accrued expenses was primarily driven by lower levels of expenses,
including sales discounts and allowances, as the result of the disposition of RHOFADE, and lower research and
development expenses as a result of the completion of our two pivotal Phase 3 clinical trials for A-101 45% Topical
Solution, as well as the timing of vendor invoicing and payments. The increase in accounts receivable was primarily the
result of the timing of cash receipts from our contract research customers. The decrease in prepaid expenses and other
assets was due to research and development activities primarily related to preclinical development activities for ATI-450
and legacy JAK inhibitors, which concluded during the year ended December 31, 2019, and the elimination of sales and
marketing activities as a result of the disposition of RHOFADE in October 2019. Non-cash expenses of $67.6 million were
composed of an intangible asset impairment charge of $27.6 million, a goodwill impairment charge of $18.5 million, stock-
based compensation expense of $16.2 million, a charge of $0.7 million related to the change in the fair value of contingent
consideration and depreciation and amortization expense of $6.4 million, partially offset by a gain of $1.9 million
recognized on the disposition of RHOFADE.
During the year ended December 31, 2018, operating activities used $100.8 million of cash primarily resulting
from our net loss of $132.7 million, partially offset by changes in our operating assets and liabilities of $9.4 million, and
non-cash adjustments of $22.5 million. Net cash provided by changes in our operating assets and liabilities during the year
ended December 31, 2018 consisted of a $13.8 million increase in accounts payable and accrued expenses, which was
partially offset by a $4.4 million increase in accounts receivable. The increase in accounts payable and accrued expenses
was primarily driven by expenses incurred, but not yet paid, as of December 31, 2018, as well as the timing of vendor
invoicing and payments. Expenses incurred, but not yet paid, as of December 31, 2018 primarily included sales and
marketing expenses related to the commercial launch of ESKATA in the United States in May 2018, amounts payable for
copay assistance and commercial rebates related to sales of RHOFADE which we began selling in December 2018, as well
as expenses related to our Phase 3 clinical trials for A-101 45% Topical Solution and our Phase 2 clinical trials for our
legacy JAK inhibitors. The increase in accounts receivable was the result of the commercial launch of ESKATA in May
2018 and sales of RHOFADE which we acquired in November 2018. Non-cash expenses of $22.5 million were primarily
composed of stock-based compensation expense.
Investing Activities
During the year ended December 31, 2020, investing activities provided $6.4 million of cash consisting of
proceeds from sales and maturities of marketable securities of $54.6 million, offset by purchases of marketable securities of
$47.7 million and purchases of equipment and leasehold improvements of $0.5 million.
During the year ended December 31, 2019, investing activities provided $105.7 million of cash consisting of
proceeds from sales and maturities of marketable securities of $210.5 million and $34.2 million from the disposition of
RHOFADE, offset by purchases of marketable securities of $137.4 million and purchases of equipment of $1.6 million.
During the year ended December 31, 2018, investing activities provided $9.4 million of cash consisting of
proceeds from sales and maturities of marketable securities of $239.4 million, offset by purchases of marketable securities
of $161.6 million, $67.1 million for the acquisition of RHOFADE, and purchases of equipment of $1.4 million.
Financing Activities
During the year ended December 31, 2020, financing activities provided $18.4 million of cash consisting of $10.9
million of net borrowings pursuant to the Loan and Security Agreement with SVB and $7.7 million of proceeds from the
issuance of common stock in connection with the Purchase Agreement with Lincoln Park, partially offset by $0.1 million
of finance lease payments and $0.2 million of deferred issuance costs.
During the year ended December 31, 2019, financing activities used $30.3 million of cash consisting of $30.0
million for the repayment of our term loan with Oxford and $0.5 million related to finance lease payments, offset by $0.2
million of cash received from the exercise of employee stock options.
During the year ended December 31, 2018, financing activities provided $128.3 million of cash consisting of net
proceeds of $100.2 million received from our public offering of common stock in October 2018, $29.9 million of net
borrowings pursuant to the Loan and Security Agreement with Oxford and $0.6 million of cash received from the exercise
of employee stock options, offset by $1.8 million paid to the former Confluence equity holders as a result of the
achievement of a development milestone and $0.6 million of finance lease payments.
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Funding Requirements
We anticipate we will incur net losses in the near term as we continue the clinical development of ATI-450 as a
potential treatment for moderate to severe rheumatoid arthritis and potentially other immuno-inflammatory diseases and
ATI-1777 as a potential treatment for moderate to severe atopic dermatitis, continue the development of our preclinical
compounds, and continue to discover and develop additional drug candidates. We may not be able to generate revenue
from these programs if, among other things, our clinical trials are not successful, the FDA does not approve our drug
candidates currently in clinical trials when we expect, or at all, or we are not able to identify and consummate transactions
with third-party partners to further develop, obtain marketing approval for and/or commercialize our drug candidates.
Our primary uses of capital are, and we expect will continue in the near term to be, compensation and related
expenses, clinical costs, external research and development services, laboratory and related supplies, legal and other
regulatory expenses, and administrative and overhead costs. Our future funding requirements will be heavily determined
by the resources needed to support the development of our drug candidates.
As a publicly traded company, we incur and will continue to incur significant legal, accounting and other
expenses. In addition, the Sarbanes-Oxley Act of 2002, as well as rules adopted by the SEC and the Nasdaq Stock Market
LLC, requires public companies to implement specified corporate governance practices.
We believe our existing cash, cash equivalents and marketable securities are sufficient to fund our operating and
capital expenditure requirements for a period greater than 12 months from the date of issuance of our consolidated financial
statements that appear in Item 8 of this Annual Report on Form 10-K based on our current operating assumptions. We will
require additional capital to complete the clinical development of ATI-450 and ATI-1777, to develop our preclinical
compounds, and to support our discovery efforts. Additional funds may not be available on a timely basis, on
commercially acceptable terms, or at all, and such funds, if raised, may not be sufficient to enable us to continue to
implement our long-term business strategy. Our ability to raise additional capital may be adversely impacted by potential
worsening global economic conditions and the recent disruptions to, and volatility in, the credit and financial markets in the
United States and worldwide resulting from the ongoing COVID-19 pandemic. If we are unable to raise sufficient
additional capital or generate revenue from transactions with potential third-party partners for the development and/or
commercialization of our drug candidates, we may need to substantially curtail our planned operations.
We may raise additional capital through the sale of equity or debt securities. In such an event, our stockholders’
ownership will be diluted, and the terms of these securities may include liquidation or other preferences that adversely
affect the rights of a holder of our common stock.
Because of the numerous risks and uncertainties associated with research and development of pharmaceutical
drugs, we are unable to estimate the exact amount of our working capital requirements. Our funding requirements in the
near term will depend on many factors, including:
● the number and development requirements of the drug candidates that we may pursue;
● the scope, progress, results and costs of preclinical development, laboratory testing and conducting
preclinical and clinical trials for our drug candidates;
● the costs, timing and outcome of regulatory review of our drug candidates;
● the extent to which we in-license or acquire additional drug candidates and technologies;
● the costs and timing of preparing, filing and prosecuting patent applications, maintaining and enforcing our
intellectual property rights and defending any intellectual property-related claims;
● the impact on the timing of our preclinical studies, the recruitment, enrollment, conduct and timing of our
clinical trials and our business due to the COVID-19 pandemic;
● our ability to identify and consummate transactions with third-party partners to further develop, obtain
marketing approval for and/or commercialize our drug candidates, and earn revenue from such arrangements;
and
● the revenue earned from our commercial products as a result of licenses to, or partnerships with, third parties.
See “Risk Factors” for additional risks associated with our substantial capital requirements.
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Contractual Obligations and Commitments
We occupy space for our headquarters in Wayne, Pennsylvania under a sublease agreement which has a term
through October 2023. In December 2020, we entered into a sub-sublease agreement under which we sub-subleased 8,115
square feet. The sub-sublease term runs concurrent with the original sublease agreement. We occupy office and laboratory
space in St. Louis, Missouri under a sublease agreement which has a term through June 2029.
In March 2020, we borrowed $11.0 million under the Loan and Security Agreement with SVB. Amounts
borrowed under the Loan and Security Agreement are subject to interest only through March 2022, after which we will be
required to make principal and interest payments through the maturity date of March 2024.
We enter into contracts in the normal course of business with CROs and contract manufacturing organizations for
clinical trials, preclinical studies and testing, manufacturing and other services and products for operating purposes. These
contracts generally provide for termination upon notice, and therefore we believe that our non-cancelable obligations under
these agreements are not material.
Segment Information
We have two reportable segments, therapeutics and contract research. The therapeutics segment is focused on
identifying and developing innovative therapies to address significant unmet needs for immuno-inflammatory diseases.
The contract research segment earns revenue from the provision of laboratory services to clients through Confluence, our
wholly-owned subsidiary.
Off-Balance Sheet Arrangements
We did not have during the periods presented, and we do not currently have, any off-balance sheet arrangements,
as defined in the rules and regulations of the SEC.
Recently Issued Accounting Pronouncements
In November 2018, the Financial Accounting Standards Board, or FASB, issued Accounting Standards Update, or
ASU, 2018-18, Collaborative Arrangements (Topic 808): Clarifying the Interaction Between Topic 808 and Topic 606,
which, among other things, provides guidance on how to assess whether certain collaborative arrangement transactions
should be accounted for under Topic 606. We adopted this standard as of January 1, 2020, the impact of which on our
consolidated financial statements was not significant.
In August 2018, the FASB issued ASU 2018-15, Intangibles—Goodwill and Other—Internal-Use Software
(Subtopic 350-40). ASU 2018-15 requires a customer in a cloud computing arrangement that is a service contract to follow
the internal-use software guidance in Accounting Standards Codification, or ASC, 350-40 to determine which
implementation costs to capitalize as assets or expense as incurred. We adopted this standard as of January 1, 2020, the
impact of which on our consolidated financial statements was not significant.
In August 2018, the FASB issued ASU 2018-13, Fair Value Measurement (Topic 820). The FASB developed the
amendments to ASC 820 as part of its broader disclosure framework project, which aims to improve the effectiveness of
disclosures in the notes to financial statements by focusing on requirements that clearly communicate the most important
information to users of the financial statements. This update eliminates certain disclosure requirements for fair value
measurements for all entities, requires public entities to disclose certain new information and modifies some of the existing
disclosure requirements. We adopted this standard as of January 1, 2020, the impact of which on our consolidated financial
statements was not significant.
In June 2018, the FASB, issued ASU 2018-07, Compensation—Stock Compensation (Topic 718). The
amendments in this ASU expand the scope of Topic 718 to include stock-based compensation arrangements with non-
employees except for specific guidance on option pricing model inputs and cost attribution. We adopted this standard as of
January 1, 2019, the impact of which on our consolidated financial statements was not significant.
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In February 2016, the FASB issued ASU 2016-02, Leases (Topic 842). In July 2018, the FASB issued ASU 2018-
10, Codification Improvements to Topic 842, Leases, and ASU 2018-11, Targeted Improvements, both of which included a
number of technical corrections and improvements, including additional options for transition. The new standard
establishes a right-of-use model that requires a lessee to record a right-of-use asset and a lease liability on the balance sheet
for all leases with terms longer than 12 months. Leases are classified as either finance or operating, with classification
affecting the pattern of expense recognition in the income statement. The amendments in ASU 2016-02 must be applied to
all leases existing at the date a company initially applies the standard.
We adopted the new standard as of January 1, 2019, using the effective date as the date of initial application, and
we used the modified retrospective approach. In addition, we elected the practical expedients permitted under the
transition guidance within the new standard, which, among other things, allowed us to carry forward the historical lease
identification and classification. We also elected the practical expedient to not separate lease and non-lease components, as
well as the short-term lease exemption which allowed us to not capitalize leases with terms less than 12 months that do not
contain a reasonably certain purchase option. Our consolidated financial statements have not been updated, and disclosures
required by the new standard have not been provided, for periods before January 1, 2019.
The adoption of ASU 2016-02 resulted in us recording additional assets and liabilities of $2.1 million and $2.3
million, respectively, upon adoption on January 1, 2019. The adoption of ASU 2016-02 did not have a material impact on
our consolidated statement of operations or cash flows.
Item 7A. Quantitative and Qualitative Disclosures about Market Risk
Our cash equivalents and marketable securities consist of money market funds, asset-backed securities,
commercial paper, corporate debt securities and government agency debt. Our primary exposure to market risk is interest
rate sensitivity, which is affected by changes in the general level of U.S. interest rates. Our marketable securities are
subject to interest rate risk and will fall in value if market interest rates increase. However, due to the short-term nature and
low-risk profile of our investment portfolio, we do not expect that an immediate 10% change in market interest rates would
have a material effect on the fair market value of our investment portfolio. We have the ability to hold our marketable
securities until maturity, and therefore we would not expect our operating results or cash flows to be affected to any
significant degree by the effect of a change in market interest rates on our investments.
The Loan and Security Agreement with SVB provides for an annual interest rate equal to the greater of (i) the
prime rate then in effect as reported in The Wall Street Journal plus 2% and (ii) 6.75%. To the extent that any present or
future credit facilities that we enter into are based on a floating interest rate, we will be subject to risks relating to changes
in market interest rates. In periods of rising interest rates when we have such debt outstanding, our interest expense would
increase. Based upon our debt outstanding of $11.0 million as of December 31, 2020, a 100 basis-point increase in the
interest rate on our loan with SVB would result in $0.1 million of additional interest expense on an annualized basis.
The uncertainty that exists with respect to the economic impact of the global COVID-19 pandemic has introduced
significant volatility in the financial markets during and subsequent to the year ended December 31, 2020.
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Item 8. Financial Statements and Supplementary Data
INDEX TO CONSOLIDATED FINANCIAL STATEMENTS
Report of Independent Registered Public Accounting Firm
Consolidated Balance Sheets as of December 31, 2020 and 2019
Consolidated Statements of Operations and Comprehensive Loss for the years ended December 31, 2020, 2019 and
2018
Consolidated Statements of Stockholders’ Equity for the years ended December 31, 2020, 2019 and 2018
Consolidated Statements of Cash Flows for the years ended December 31, 2020, 2019 and 2018
Notes to Consolidated Financial Statements
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87
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REPORT OF INDEPENDENT REGISTERED PUBLIC ACCOUNTING FIRM
To the Board of Directors and Stockholders of
Aclaris Therapeutics, Inc.
Opinion on the Financial Statements
We have audited the accompanying consolidated balance sheets of Aclaris Therapeutics, Inc. and its subsidiaries
(the “Company”) as of December 31, 2020 and 2019, and the related consolidated statements of operations and
comprehensive loss, of stockholders’ equity and of cash flows for each of the three years in the period ended December 31,
2020, including the related notes (collectively referred to as the “consolidated financial statements”). In our opinion, the
consolidated financial statements present fairly, in all material respects, the financial position of the Company as of
December 31, 2020 and 2019, and the results of its operations and its cash flows for each of the three years in the period
ended December 31, 2020 in conformity with accounting principles generally accepted in the United States of America.
Basis for Opinion
These consolidated financial statements are the responsibility of the Company’s management. Our responsibility
is to express an opinion on the Company’s consolidated financial statements based on our audits. We are a public
accounting firm registered with the Public Company Accounting Oversight Board (United States) (PCAOB) and are
required to be independent with respect to the Company in accordance with the U.S. federal securities laws and the
applicable rules and regulations of the Securities and Exchange Commission and the PCAOB.
We conducted our audits of these consolidated financial statements in accordance with the standards of the
PCAOB. Those standards require that we plan and perform the audit to obtain reasonable assurance about whether the
consolidated financial statements are free of material misstatement, whether due to error or fraud. The Company is not
required to have, nor were we engaged to perform, an audit of its internal control over financial reporting. As part of our
audits we are required to obtain an understanding of internal control over financial reporting but not for the purpose of
expressing an opinion on the effectiveness of the Company's internal control over financial reporting. Accordingly, we
express no such opinion.
Our audits included performing procedures to assess the risks of material misstatement of the consolidated
financial statements, whether due to error or fraud, and performing procedures that respond to those risks. Such procedures
included examining, on a test basis, evidence regarding the amounts and disclosures in the consolidated financial
statements. Our audits also included evaluating the accounting principles used and significant estimates made by
management, as well as evaluating the overall presentation of the consolidated financial statements. We believe that our
audits provide a reasonable basis for our opinion.
Critical Audit Matters
The critical audit matter communicated below is a matter arising from the current period audit of the consolidated
financial statements that was communicated or required to be communicated to the audit committee and that (i) relates to
accounts or disclosures that are material to the consolidated financial statements and (ii) involved our especially challenging,
subjective, or complex judgments. The communication of critical audit matters does not alter in any way our opinion on the
consolidated financial statements, taken as a whole, and we are not, by communicating the critical audit matter below,
providing a separate opinion on the critical audit matter or on the accounts or disclosures to which it relates.
Fair Value of Contingent Consideration Liability
As described in Notes 2 and 4 to the consolidated financial statements, the Company initially recorded a
contingent consideration liability related to future potential payments resulting from the acquisition of Confluence based
upon the achievement of certain development, regulatory and commercial milestones, as well as future projected sales
performance, at its estimated fair value on the date of acquisition. Management evaluates fair value estimates of contingent
consideration liabilities on a quarterly basis. Management estimates the fair value of the contingent consideration liability
associated with sales milestones and royalties by estimating future sales levels, assigning an achievement probability and
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discounting the associated cash payment to its present value using a risk-adjusted rate of return. Management estimates the
fair value of the contingent consideration liability for regulatory milestones by assigning an achievement probability to
each potential milestone and discounting the associated cash payments to their present values using a credit-risk-adjusted
interest rate. Changes in the fair value of the contingent consideration are recorded as income or expense in the Company’s
consolidated statement of operations and comprehensive loss. Significant assumptions used in management’s estimates
include the probability of success of both achieving regulatory milestones and commencing commercialization, which are
based upon an asset’s current stage of development. As of and for the year ended December 31, 2020, management
recorded a contingent consideration liability of $4.1 million and expense of $2.4 million.
The principal considerations for our determination that performing procedures relating to the fair value of the
contingent consideration liability is a critical audit matter are the significant judgment by management when developing
the fair value estimate, which in turn led to a high degree of auditor judgment, subjectivity and effort in evaluating the
significant assumptions related to the probability of success of both achieving regulatory milestones and commencing
commercialization. Also, the audit effort involved the use of professionals with specialized skill and knowledge.
Addressing the matter involved performing procedures and evaluating audit evidence in connection with forming
our overall opinion on the consolidated financial statements. These procedures included, among others, testing
management’s process for developing the fair value of the contingent consideration liability and evaluating the
reasonableness of the valuation model and assumptions related to the probability of success of both achieving regulatory
milestones and commencing commercialization. Evaluating management’s assumptions related to the probability of
success of both achieving regulatory milestones and commencing commercialization involved assessing whether the
assumptions used by management were reasonable considering the agreements associated with the transaction and the
consistency with industry studies and the stage of product development. Professionals with specialized skill and knowledge
were used to assist in evaluating the appropriateness of management’s valuation model.
/s/ PricewaterhouseCoopers LLP
Philadelphia, Pennsylvania
February 25, 2021
We have served as the Company’s auditor since 2015.
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ACLARIS THERAPEUTICS, INC.
CONSOLIDATED BALANCE SHEETS
(In thousands, except share and per share data)
Assets
Current assets:
Cash and cash equivalents
Restricted cash
Marketable securities
Accounts receivable, net
Prepaid expenses and other current assets
Discontinued operations - current assets
Total current assets
Property and equipment, net
Intangible assets
Other assets
Total assets
Liabilities and Stockholders’ Equity
Current liabilities:
Accounts payable
Accrued expenses
Current portion of lease liabilities
Discontinued operations - current liabilities
Total current liabilities
Other liabilities
Long-term debt, net
Contingent consideration
Deferred tax liability
Total liabilities
Commitments and contingencies (Note 20)
Stockholders’ Equity:
Preferred stock, $0.00001 par value; 10,000,000 shares authorized and no shares issued
or outstanding at December 31, 2020 and December 31, 2019
Common stock, $0.00001 par value; 100,000,000 shares authorized at
December 31, 2020 and December 31, 2019; 45,109,314 and 41,485,638 shares issued
and outstanding at December 31, 2020 and December 31, 2019, respectively
Additional paid‑in capital
Accumulated other comprehensive loss
Accumulated deficit
Total stockholders’ equity
Total liabilities and stockholders’ equity
December 31,
2020
December 31,
2019
$
$
$
$
$
$
22,063
—
32,068
772
2,590
—
57,493
1,654
7,123
4,514
70,784
5,254
5,906
603
3,111
14,874
3,179
10,653
4,061
367
33,134
34,187
1,750
39,078
704
3,118
4,966
83,803
2,470
7,199
4,825
98,297
9,917
7,721
637
4,157
22,432
3,736
—
1,668
549
28,385
—
—
—
542,286
(94)
(504,542)
37,650
70,784
$
$
—
523,505
(66)
(453,527)
69,912
98,297
The accompanying notes are an integral part of these consolidated financial statements.
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ACLARIS THERAPEUTICS, INC.
CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS
(In thousands, except share and per share data)
Revenues:
Contract research
Other revenue
Total revenue
Costs and expenses:
Cost of revenue
Research and development
General and administrative
Goodwill impairment
Total costs and expenses
Loss from operations
Other income (expense), net
Loss from continuing operations before income taxes
Income tax benefit
Loss from continuing operations
Income (loss) from discontinued operations, net of tax
Net loss
Net loss per share, basic and diluted
Weighted average common shares outstanding, basic and diluted
Other comprehensive income (loss):
Unrealized gain (loss) on marketable securities, net of tax of $0
Foreign currency translation adjustment
Total other comprehensive income (loss)
Comprehensive loss
Year Ended
December 31,
2019
2018
2020
$
5,786
696
6,482
$
4,227
—
4,227
4,651
1,500
6,151
5,133
31,731
20,530
—
57,394
(50,912)
4,055
64,899
27,827
18,504
115,285
(111,058)
4,329
60,841
25,761
—
90,931
(84,780)
(424)
(51,336)
(182)
(51,154)
139
(51,015) $
(2,484)
(113,542)
—
(113,542)
(47,812)
(161,354) $
2,676
(82,104)
—
(82,104)
(50,634)
(132,738)
$
$
(1.20) $
$
42,539,293
(3.90) $
(4.03)
32,909,762
41,323,921
$
$
(2) $
(26)
(28)
(51,043) $
$
28
(25)
3
(161,351) $
145
32
177
(132,561)
The accompanying notes are an integral part of these consolidated financial statements.
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ACLARIS THERAPEUTICS, INC.
CONSOLIDATED STATEMENTS OF STOCKHOLDERS’ EQUITY
(In thousands, except share data)
Common Stock
Par
Additional
Paid‑in
Value Capital
Accumulated
Other
Comprehensive Accumulated
Loss
$ — $ 384,943
$
(246) $
Balance at December 31, 2017
Issuance of common stock in connection with public
offering, net of offering costs of $6,669
Issuance of common stock in connection with the
Confluence development milestone
Exercise of stock options and vesting of restricted stock
units
Unrealized gain on marketable securities
Foreign currency translation adjustment
Stock-based compensation expense
Net loss
Balance at December 31, 2018
Exercise of stock options and vesting of restricted stock
units
Unrealized gain on marketable securities
Foreign currency translation adjustment
Stock-based compensation expense
Net loss
Balance at December 31, 2019
Exercise of stock options and vesting of restricted stock
units
Issuance of common stock in connection with an equity
purchase agreement, net of offering costs of $168
Fair value of warrants issued in connection with debt
financing
Unrealized loss on marketable securities
Foreign currency translation adjustment
Stock-based compensation expense
Net loss
Balance at December 31, 2020
Shares
30,856,505
9,941,750
253,181
—
—
100,205
2,215
159,289
—
—
—
—
41,210,725
—
—
—
—
—
(52)
—
—
20,055
—
$ — $ 507,366
274,913
—
—
—
—
41,485,638
1,390,922
2,232,754
—
—
—
—
—
45,109,314
—
—
—
—
—
(38)
—
—
16,177
—
$ — $ 523,505
—
—
(669)
7,865
—
—
—
—
—
378
—
—
11,207
—
$ — $ 542,286
$
$
$
Deficit
(159,435) $
—
—
—
—
—
—
(132,738)
(292,173) $
—
—
—
—
(161,354)
(453,527) $
—
—
—
—
—
145
32
—
—
(69) $
—
28
(25)
—
—
(66) $
—
—
—
(2)
(26)
—
—
(94) $
—
—
—
—
(51,015)
(504,542) $
Total
Stockholders’
Equity
225,262
100,205
2,215
(52)
145
32
20,055
(132,738)
215,124
(38)
28
(25)
16,177
(161,354)
69,912
(669)
7,865
378
(2)
(26)
11,207
(51,015)
37,650
The accompanying notes are an integral part of these consolidated financial statements.
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ACLARIS THERAPEUTICS, INC.
CONSOLIDATED STATEMENTS OF CASH FLOWS
(In thousands)
Cash flows from operating activities:
Net loss
Adjustments to reconcile net loss to net cash used in operating activities:
Depreciation and amortization
Stock-based compensation expense
Change in fair value of contingent consideration
Goodwill impairment charge
Intangible asset impairment charge
Payment of Confluence development milestone
Gain on sale of RHOFADE
Deferred taxes
Changes in operating assets and liabilities:
Accounts receivable
Prepaid expenses and other assets
Accounts payable
Accrued expenses
Net cash used in operating activities
Cash flows from investing activities:
Purchases of property and equipment
Acquisition of RHOFADE
Disposition of RHOFADE
Purchases of marketable securities
Proceeds from sales and maturities of marketable securities
Net cash provided by investing activities
Cash flows from financing activities:
Proceeds from issuance of common stock in connection with public offering, net of issuance costs
Proceeds from issuance of common stock in connection with an equity purchase agreement, net of
issuance costs
Proceeds from debt financing (including warrants), net of issuance costs
Repayment of debt
Payment of Confluence development milestone
Finance lease payments
Deferred issuance costs
Proceeds from exercise of employee stock options and the issuance of stock
Net cash provided by (used in) financing activities
Net increase (decrease) in cash, cash equivalents and restricted cash
Cash, cash equivalents and restricted cash at beginning of period
Cash, cash equivalents and restricted cash at end of period
Supplemental disclosure of non-cash investing and financing activities:
Additions to property and equipment included in accounts payable
Fair value of warrants issued in connection with debt financing
Property and equipment obtained pursuant to finance lease financing arrangements
Fair value of stock issued in connection with Confluence development milestone
Offering costs included in accounts payable
Operating lease asset recorded as a result of new accounting standard
Fair value of common stock issued in connection with an equity purchase agreement
Year Ended
December 31,
2019
2018
2020
$
(51,015)
$
(161,354)
$
(132,738)
1,324
11,207
2,393
—
—
—
—
(182)
4,898
1,689
(5,219)
(3,728)
(38,633)
(453)
—
—
(47,714)
54,554
6,387
6,409
16,177
734
18,504
27,638
—
(1,850)
—
(809)
3,233
(3,160)
(1,967)
(96,445)
(1,613)
—
34,186
(137,385)
210,491
105,679
1,879
20,055
1,272
—
—
(717)
—
—
(4,380)
62
6,964
6,792
(100,811)
(1,356)
(67,122)
—
(161,598)
239,443
9,367
—
—
100,205
7,737
10,913
—
—
(137)
(211)
70
18,372
(13,874)
35,937
22,063
$
—
—
(30,000)
—
(523)
—
207
(30,316)
(21,082)
57,019
35,937
$
—
29,910
—
(1,783)
(648)
—
577
128,261
36,817
20,202
57,019
— $
378
$
— $
— $
— $
— $
$
263
124
$
— $
— $
— $
— $
$
— $
2,132
161
—
2,131
2,215
210
—
—
$
$
$
$
$
$
$
$
The accompanying notes are an integral part of these consolidated financial statements.
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ACLARIS THERAPEUTICS, INC.
NOTES TO CONSOLIDATED FINANCIAL STATEMENTS
1. Organization and Nature of Business
Overview
Aclaris Therapeutics, Inc. was incorporated under the laws of the State of Delaware in 2012. In July 2015, Aclaris
Therapeutics International Limited (“ATIL”) was established under the laws of the United Kingdom as a wholly-owned
subsidiary of Aclaris Therapeutics, Inc. In March 2016, Vixen Pharmaceuticals, Inc. (“Vixen”) became a wholly-owned
subsidiary of Aclaris Therapeutics, Inc., and in September 2018, Vixen was dissolved. In August 2017, Confluence Life
Sciences, Inc. (now known as Aclaris Life Sciences, Inc.) (“Confluence”) was acquired by Aclaris Therapeutics, Inc. and
became a wholly-owned subsidiary thereof. Aclaris Therapeutics, Inc., ATIL, Vixen and Confluence are referred to
collectively as the “Company.” The Company is a clinical-stage biopharmaceutical company focused on developing novel
drug candidates for immuno-inflammatory diseases. In addition to developing its novel drug candidates, the Company is
pursuing strategic alternatives, including identifying and consummating transactions with third-party partners, to further
develop, obtain marketing approval for and/or commercialize its novel drug candidates.
Liquidity
The Company’s consolidated financial statements have been prepared on the basis of continuity of operations,
realization of assets and the satisfaction of liabilities in the ordinary course of business. As of December 31, 2020, the
Company had cash, cash equivalents and marketable securities of $54.1 million and an accumulated deficit of $504.5
million. Since inception, the Company has incurred net losses and negative cash flows from its operations. Prior to the
acquisition of Confluence in August 2017, the Company had never generated revenue. There can be no assurance that
profitable operations will ever be achieved, and, if achieved, will be sustained on a continuing basis. In addition,
development activities, including clinical and preclinical testing of the Company’s drug candidates, will require significant
additional financing. The future viability of the Company is dependent on its ability to successfully develop its drug
candidates and to generate revenue from identifying and consummating transactions with third-party partners to further
develop, obtain marketing approval for and/or commercialize its development assets or to raise additional capital to finance
its operations. The Company will require additional capital to complete the clinical development of ATI-450 and ATI-
1777, to develop its preclinical compounds, and to support its discovery efforts.
The Company has taken a number of actions to support its operations and meet its liquidity needs. In September
2019, the Company announced the completion of a strategic review and its decision to refocus its resources on its immuno-
inflammatory development programs and to actively seek partners for its drug candidates and commercial products. As a
result of this decision, the Company restructured its operations and reduced its workforce, which lowered operating costs.
In October 2019, the Company sold the worldwide rights to RHOFADE (oxymetazoline hydrochloride) cream, 1%
(“RHOFADE”) to further its focus on its development programs and improve cash flow. In March 2020, the Company
borrowed $11.0 million under a term loan facility with Silicon Valley Bank. In August 2020, the Company entered into an
equity purchase agreement (the “Purchase Agreement”) with Lincoln Park Capital Fund, LLC (“Lincoln Park”). As of
December 31, 2020, the Company had sold 2,111,170 shares of common stock to Lincoln Park under the Purchase
Agreement for net proceeds of $7.7 million. The Company did not sell any additional shares prior to terminating the
Purchase Agreement in January 2021 in connection with a public offering of common stock in which it sold 6,306,271
shares of its common stock for net proceeds of $103.5 million.
The Company’s plans to further address its liquidity needs primarily include its ability to control the timing and
spending on its research and development programs. The Company may also consider other plans to fund its operations
including: (1) raising additional capital through debt or equity financings; (2) identifying third-party partners to further
develop, obtain marketing approval for and/or commercialize its drug candidates, which may generate revenue and/or
milestone payments; (3) reducing spending on one or more research and development programs by delaying or
discontinuing development; and/or (4) further restructuring its operations to change its overhead structure.
Additional funds may not be available on a timely basis, on commercially acceptable terms, or at all, and such
funds, if raised, may not be sufficient to enable the Company to continue to implement its long-term business strategy.
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The Company’s ability to raise additional capital may be adversely impacted by potential worsening global economic
conditions and the recent disruptions to, and volatility in, the credit and financial markets in the United States and
worldwide resulting from the ongoing COVID-19 pandemic. If the Company is unable to raise sufficient additional capital
or generate revenue from transactions with potential third-party partners for the development and/or commercialization of
its drug candidates, it may need to substantially curtail planned operations. The Company’s failure to raise capital as and
when needed could have a negative impact on its financial condition and ability to pursue its business strategies.
In accordance with Accounting Standards Update (“ASU”) 2014-15, Disclosure of Uncertainties about an Entity’s
Ability to Continue as a Going Concern (Subtopic 205-40), the Company evaluated whether there are conditions and
events, considered in the aggregate, that raise substantial doubt about the Company’s ability to continue as a going concern
within one year after the date that its consolidated financial statements are issued. As of the report date, the Company does
not believe that substantial doubt exists about its ability to continue as a going concern. The Company believes its existing
cash, cash equivalents and marketable securities are sufficient to fund its operating and capital expenditure requirements
for a period greater than 12 months from the date of issuance of these consolidated financial statements.
2. Summary of Significant Accounting Policies
Basis of Presentation
The accompanying consolidated financial statements have been prepared in conformity with generally accepted
accounting principles in the United States (“GAAP”). The consolidated financial statements of the Company include the
accounts of the operating parent company, Aclaris Therapeutics, Inc., and its wholly-owned subsidiaries, Confluence,
ATIL, and Vixen (for periods prior to its dissolution in 2018). All significant intercompany transactions have been
eliminated. Based upon the Company’s revenue, the Company believes that gross profit does not provide a meaningful
measure of profitability and, therefore, has not included a line item for gross profit on the consolidated statement of
operations.
Reclassifications
Certain prior year amounts have been reclassified to conform to the current year financial statement presentation.
Discontinued Operations
In September 2019, the Company announced the completion of a strategic review and its decision to refocus its
resources on its immuno-inflammatory development programs and to actively seek partners for its commercial products.
The Company also announced a plan to terminate 86 employees (see Note 17).
Use of Estimates
The preparation of financial statements in conformity with GAAP requires management to make estimates and
assumptions that affect the reported amounts of assets and liabilities, the disclosure of contingent assets and liabilities at the
date of the financial statements and the reported amounts of expenses during the reporting periods. Significant estimates
and assumptions reflected in these financial statements include, but are not limited to, research and development expenses,
contingent consideration and the valuation of stock-based awards. Estimates are periodically reviewed in light of changes
in circumstances, facts and experience. The COVID-19 pandemic has resulted in a global slowdown in economic activity.
As of the date of issuance of these financial statements, the Company is not aware of any specific event or circumstance
that would require an update to its estimates, assumptions and judgments or revise the carrying value of its assets or
liabilities. Actual results could differ from the Company’s estimates.
Revenue Recognition
The Company accounts for revenue in accordance with Accounting Standards Codification (“ASC”) Topic 606,
Revenue from Contracts with Customers. Under ASC Topic 606, revenue is recognized when a customer obtains control
of promised goods or services in an amount that reflects the consideration to which the Company expects to be entitled in
exchange for those goods or services.
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To determine revenue recognition in accordance with ASC Topic 606, the Company performs the following five
steps: (i) identify the contract(s) with a customer, (ii) identify the performance obligations in the contract, (iii) determine
the transaction price, (iv) allocate the transaction price to the performance obligations in the contract, and (v) recognize
revenue when (or as) performance obligations are satisfied. At contract inception, the Company assesses the goods or
services promised within a contract with a customer to identify the performance obligations, and to determine if they are
distinct. The Company recognizes the revenue that is allocated to each distinct performance obligation when (or as) that
performance obligation is satisfied. The Company only recognizes revenue when collection of the consideration it is
entitled to under a contract with a customer is probable.
Product Sales, net
The Company sold RHOFADE and ESKATA (hydrogen peroxide) topical solution, 40% (w/w) (“ESKATA”), its
non-marketed product approved by the U.S. Food and Drug Administration, during the years ended December 31, 2019
and 2018 to a limited number of wholesalers in the United States (collectively, its “Customers”). These Customers
subsequently resold the Company’s products to pharmacies and health care providers. In addition to distribution
agreements with Customers, the Company entered into, or was subject to, arrangements with third-party payors, including
pharmacy benefit managers and government agencies, as well as group purchasing organizations (“GPOs”), which
provided for government mandated or privately negotiated rebates, chargebacks, and discounts with respect to the purchase
of the Company’s commercial products. The Company discontinued selling ESKATA in August 2019. The Company sold
the worldwide rights to RHOFADE in October 2019 (see Note 3). Product sales, net is presented in discontinued
operations for all periods presented.
The Company recognized revenue from product sales at the point the Customer obtained control of the product,
which generally occurred upon delivery. The Company also included estimates of variable consideration in the same
period revenue was recognized. Components of variable consideration include trade discounts and allowances, product
returns, government rebates, discounts and rebates, other incentives such as patient co-pay assistance, and other fee for
service amounts. Variable consideration was recorded on the consolidated balance sheet as either a reduction of accounts
receivable, if payable to a Customer, or as a current liability, if payable to a third party other than a Customer. The
Company considered all relevant information when estimating variable consideration such as contractual and statutory
requirements, specific known market events and trends, industry data and forecasted customer buying and payment
patterns. The amount of net revenue that can be recognized is constrained by estimates of variable consideration which are
included in the transaction price. Payment terms with Customers did not exceed one year and, therefore, the Company did
not account for a financing component in its arrangements. The Company expensed incremental costs of obtaining a
contract with a Customer, including sales commissions, when incurred as the period of benefit was less than one year.
Trade Discounts and Allowances - The Company provided Customers with trade discounts, rebates, allowances
and/or other incentives. The Company recorded estimates for these items as a reduction of revenue in the same period the
revenue was recognized.
Government and Payor Rebates - The Company contracted with, or was subject to arrangements with, certain
third-party payors, including pharmacy benefit managers and government agencies, for the payment of rebates with respect
to utilization of its commercial products. The Company also entered into agreements with GPOs that provided for
administrative fees and discounted pricing in the form of volume-based rebates. The Company was also subject to discount
and rebate obligations under state Medicaid programs and Medicare. The Company recorded estimates for these discounts
and rebates as a reduction of revenue in the same period the revenue was recognized.
Other Incentives - The Company maintained a co-pay assistance program which was intended to provide financial
assistance to qualified commercially-insured patients with prescription drug co-payments required by third-party payors.
The Company estimated and recorded accruals for these incentives as a reduction of revenue in the period the revenue was
recognized. The Company estimated amounts for co-pay assistance based upon the number of claims and the cost per
claim that the Company expected to receive associated with product that had been sold to Customers but remained in the
distribution channel at the end of each reporting period.
Product Returns - Consistent with industry practice, the Company has a product returns policy for RHOFADE that
provides Customers a right of return for product purchased within a specified period prior to and subsequent to the
product’s expiration date. The right of return lapses upon shipment of the product to a patient. The Company recorded an
estimate for the amount of its products which may be returned as a reduction of revenue in the period the related revenue
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was recognized. The Company’s estimate for product returns was based upon available industry data and its own sales
information, including its visibility into the inventory remaining in the distribution channel. There is no return liability
associated with sales of ESKATA as the Company had a no returns policy for ESKATA when it was commercialized.
Contract Research
The Company earns contract research revenue from the provision of laboratory services to clients through
Confluence, its wholly-owned subsidiary. Contract research revenue is generally evidenced by contracts with clients which
are on an agreed upon fixed-price, fee-for-service basis and are generally billed on a monthly basis in arrears for services
rendered. Revenue related to these contracts is generally recognized as the laboratory services are performed, based upon
the rates specified in the contracts. Under ASC Topic 606, the Company elected to apply the “right to invoice” practical
expedient when recognizing contract research revenue and as such, recognizes revenue in the amount which it has the right
to invoice. ASC Topic 606 also provides an optional exemption, which the Company has elected to apply, from disclosing
remaining performance obligations when revenue is recognized from the satisfaction of the performance obligation in
accordance with the “right to invoice” practical expedient.
The Company also received revenue from grants under the Small Business Innovation Research program of the
National Institutes of Health, or NIH. During the year ended December 31, 2018, the Company had two active grants from
NIH related to early-stage research. There are no remaining funds available under the grants.
Other Revenue
Licenses of Intellectual Property – The Company recognizes revenue received from non-refundable, upfront fees
related to the licensing of intellectual property when the intellectual property is determined to be distinct from the other
performance obligations identified in the arrangement, the license has been transferred to the customer, and the customer is
able to use and benefit from the license.
Milestone Payments – At the inception of each arrangement that includes milestone payments, the Company
evaluates whether the milestones are considered probable of being reached and estimates the amount to be included in the
transaction price using the most likely amount method. If it is probable that a significant revenue reversal would not occur,
the associated milestone value is included in the amount allocated to the license of intellectual property. Milestone
payments that are not within the control of the Company or the counterparty, such as regulatory approvals, are not
considered probable of being achieved until those approvals are received.
Cash, Cash Equivalents and Restricted Cash
The Company considers all short-term, highly liquid investments with original maturities of three months or less
at acquisition date to be cash equivalents. Cash equivalents, which have consisted of money market accounts and
commercial paper, are stated at fair value. Total cash, cash equivalents and restricted cash as shown in the consolidated
statements of cash flows as of December 31, 2020 and 2019 includes $0 and $1.8 million, respectively, of restricted cash,
consisting of funds in escrow pursuant to the asset purchase agreement with EPI Health, LLC (“EPI Health”) (see Note
15).
Marketable Securities
Marketable securities with original maturities of greater than three months and remaining maturities of less than
one year from the balance sheet date are classified as short-term. Marketable securities with remaining maturities of greater
than one year from the balance sheet date are classified as long-term.
The Company classifies all of its marketable securities as available-for-sale securities. The Company’s marketable
securities are measured and reported at fair value using either quoted prices in active markets for identical securities or
quoted prices in markets that are not active for identical or similar securities. Unrealized gains and losses are reported as a
separate component of stockholders’ equity. The cost of securities sold is determined on a specific identification basis, and
realized gains and losses, if any, are included in other income (expense), net within the consolidated statement of
operations and comprehensive loss. If any adjustment to fair value reflects a decline in the value of the investment, the
Company considers available evidence to evaluate the extent to which the decline is “other than
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temporary” and reduces the investment to fair value through a charge to the statement of operations and comprehensive
loss.
Property and Equipment
Property and equipment are stated at cost less accumulated depreciation. Depreciation expense is recognized using
the straight-line method over the useful life of the asset. Computer equipment is depreciated over three years.
Manufacturing and laboratory equipment is depreciated over five years. Furniture and fixtures are depreciated over five
years. Leasehold improvements are depreciated over the shorter of the lease term or their useful life. Expenditures for
repairs and maintenance of assets are charged to expense as incurred. Upon retirement or sale, the cost and related
accumulated depreciation of assets disposed of are removed from the accounts and any resulting gain or loss is included in
loss from continuing operations.
Impairment of Long-Lived Assets
Long-lived assets consist of property and equipment. Long-lived assets to be held and used are tested for
recoverability whenever events or changes in business circumstances indicate that the carrying amount of the assets may
not be fully recoverable. Factors that the Company considers in deciding when to perform an impairment review include
significant underperformance of the business in relation to expectations, significant negative industry or economic trends
and significant changes or planned changes in the use of the assets. If an impairment review is performed to evaluate a
long-lived asset for recoverability, the Company compares forecasts of undiscounted cash flows expected to result from the
use and eventual disposition of the long-lived asset to its carrying value. An impairment loss would be recognized when
estimated undiscounted future cash flows expected to result from the use of an asset are less than its carrying amount. The
impairment loss would be based on the excess of the carrying value of the impaired asset over its fair value, determined
based on discounted cash flows.
Intangible Assets
Intangible assets include both definite-lived and indefinite-lived assets. Definite-lived intangible assets consist of
a drug discovery platform the Company acquired through the acquisition of Confluence, and prior to the disposition in
2019, also included the intellectual property rights related to RHOFADE. Definite-lived intangible assets are amortized
over their estimated useful life based on the pattern over which the intangible assets are consumed or otherwise used up. If
that pattern cannot be reliably determined, the straight-line method of amortization is used. Indefinite-lived intangible
assets consist of an in-process research and development (“IPR&D”) drug candidate acquired through the acquisition of
Confluence. IPR&D assets are considered indefinite-lived until the completion or abandonment of the associated research
and development efforts. The cost of IPR&D is either amortized over its estimated useful life beginning when the
underlying drug candidate is approved and launched commercially, or expensed immediately if development of the drug
candidate is abandoned or otherwise impaired.
Definite-lived intangible assets are tested for impairment when events or changes in circumstances indicate that
the carrying value of the asset may not be recoverable. Indefinite-lived intangible assets are tested for impairment at least
annually, which the Company performs during the fourth quarter, or when indicators of an impairment are present. The
Company recognizes impairment losses when and to the extent that the estimated fair value of an intangible asset is less
than its carrying value.
During the year ended December 31, 2019, the Company performed an impairment analysis of the RHOFADE
intangible asset due to its decision to discontinue commercial operations and actively seek a commercialization partner for
RHOFADE. The Company’s impairment analysis, which primarily utilized a market-participant’s indication of fair value,
resulted in a fair value for the RHOFADE intangible asset which was less than its carrying value. As a result, the Company
recorded an impairment charge of $27.6 million, which is included in discontinued operations on the consolidated
statement of operations, to adjust the carrying value of the RHOFADE intangible asset to its net realizable value (see Note
3).
During the years ended December 31, 2020 and 2019, the Company did not record an IPR&D impairment.
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Goodwill
Goodwill is not amortized, but rather is subject to testing for impairment at least annually, which the Company
performs either during the fourth quarter or when indicators of an impairment are present. The Company considers each of
its operating segments, therapeutics and contract research, to be a reporting unit since this is the lowest level for which
discrete financial information is available. The impairment test performed by the Company is a qualitative assessment
based upon the then current facts and circumstances related to operations of the reporting unit. If the qualitative
assessment indicates an impairment may be present, the Company would perform the required quantitative analysis and an
impairment charge would be recognized to the extent that the estimated fair value of the reporting unit is less than its
carrying amount. However, any loss recognized would not exceed the total amount of goodwill allocated to that reporting
unit.
During the year ended December 31, 2019, the Company performed an impairment analysis due to a decline in its
stock price, which was considered a triggering event to evaluate goodwill for impairment. The Company’s impairment
analysis, using a market approach, noted that its stock price, including a reasonable control premium, resulted in a fair
value for the therapeutics reporting unit which was less than its carrying value. As a result, the Company recorded an
impairment charge of $18.5 million.
Leases
Leases represent a company’s right to use an underlying asset and a corresponding obligation to make payments to
a lessor for the right to use those assets. The Company evaluates leases at their inception to determine if they are an
operating lease or a finance lease. A lease is accounted for as a finance lease if it meets one of the following five criteria:
the lease has a purchase option that is reasonably certain of being exercised, the present value of the future cash flows are
substantially all of the fair market value of the underlying asset, the lease term is for a significant portion of the remaining
economic life of the underlying asset, the title to the underlying asset transfers at the end of the lease term, or if the
underlying asset is of such a specialized nature that it is expected to have no alternative uses to the lessor at the end of the
term. Leases that do not meet the finance lease criteria are accounted for as an operating lease.
The Company recognizes assets and liabilities for leases at their inception based upon the present value of all
payments due under the lease. The Company uses an implicit interest rate to determine the present value of finance leases,
and its incremental borrowing rate to determine the present value of operating leases. The Company determines
incremental borrowing rates by referencing collateralized borrowing rates for debt instruments with terms similar to the
respective lease. The Company recognizes expense for operating and finance leases on a straight-line basis over the term
of each lease, and interest expense related to finance leases is recognized over the lease term based on the effective interest
method. The Company includes estimates for any residual value guarantee obligations under its leases in lease liabilities
recorded on its consolidated balance sheet.
Right-of-use assets are included in other assets and property and equipment, net on the Company’s consolidated
balance sheet for operating and finance leases, respectively. Obligations for lease payments are included in current portion
of lease liabilities and other liabilities on the Company’s consolidated balance sheet for both operating and finance leases.
Contingent Consideration
The Company initially recorded a contingent consideration liability related to future potential payments resulting
from the acquisition of Confluence based upon the achievement of certain development, regulatory and commercial
milestones, as well as future projected sales performance, at its estimated fair value on the date of acquisition. The ultimate
amount of future payments, if any, is based on criteria such as sales performance and the achievement of certain regulatory
and sales milestones. The Company estimates the fair value of the contingent consideration liability related to the
achievement of regulatory milestones by assigning an achievement probability to each potential milestone and discounting
the associated cash payment to its present value using a credit-risk-adjusted interest rate. The Company estimates the fair
value of the contingent consideration liability associated with sales milestones and royalties by estimating future sales
levels, assigning an achievement probability and discounting the associated cash payments to their present values using a
risk-adjusted rate of return. Significant assumptions used in the Company’s estimates include the probability of success of
both achieving regulatory milestones and commencing commercialization, which are based upon an asset’s current stage of
development and ranged between 4% and 15%. The Company evaluates fair value estimates of contingent consideration
liabilities on a quarterly basis. Any change in fair value reflects new information about the likelihood of the payment of the
contingent consideration and the passage of time. For example, if the timing of the development of an
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acquired drug candidate, or the size of potential commercial opportunities related to an acquired drug candidate, differ from
the Company’s assumptions, then the fair value of contingent consideration would be adjusted accordingly. Future changes
in the fair value of the contingent consideration, if any, will be recorded as income or expense in the Company’s
consolidated statement of operations and comprehensive loss.
Research and Development Costs
Research and development costs are expensed as incurred. Research and development expenses include salaries,
stock-based compensation and benefits of employees, fees paid under licensing agreements, fees paid under a third party
assignment agreement and other operational costs related to the Company’s research and development activities, including
depreciation expenses and the cost of research and development contracts which the Company has entered into with
outside vendors to conduct both preclinical studies and clinical trials. Significant judgment and estimates are made in
determining the amount of research and development costs recognized in each reporting period. The Company analyzes
the progress of its preclinical studies and clinical trials, completion of milestone events, invoices received and contracted
costs when estimating research and development costs. Actual results could differ from the Company’s estimates. The
Company’s historical estimates for research and development costs have not been materially different from the actual costs.
Stock-Based Compensation
The Company measures the compensation expense of stock-based awards granted to employees and directors
using the grant date fair value of the award. The Company has issued stock options and restricted stock unit (“RSU”)
awards with service-based vesting conditions, as well as with performance-based vesting conditions. The Company has
not issued awards that include market-based conditions. For service-based awards the Company recognizes stock-based
compensation expense on a straight-line basis over the requisite service period. For performance-based awards the
Company recognizes stock-based compensation expense on a straight-line basis over the requisite service period beginning
in the period that it becomes probable the performance conditions will occur. At each balance sheet date, the Company
evaluates whether any performance conditions related to a performance-based award have changed. The effect of any
change in performance conditions would be recognized as a cumulative catch-up adjustment in the period such change
occurs, and any remaining unrecognized compensation expense would be recognized on a straight-line basis over the
remaining requisite service period. The impact of forfeitures is recognized in the period in which they occur.
The Company measures the compensation expense of stock-based awards granted to consultants using the grant
date fair value of the award. The Company recognizes compensation expense over the period during which services are
rendered by the consultant.
The Company classifies stock-based compensation expense in its statement of operations and comprehensive loss
in the same manner in which the award recipient’s payroll costs are classified or in which the award recipients’ service
payments are classified.
The fair value of each stock option grant is estimated on the date of grant using the Black-Scholes option-pricing
model. The Company estimates its expected stock volatility based on the historical volatility of a set of peer companies,
which are publicly traded, and expects to continue to do so until it has adequate historical data regarding the volatility of its
own publicly-traded stock price. The expected term of the Company’s stock options has been determined using the
“simplified” method for awards that qualify as “plain vanilla” options. The expected term of stock options granted to non-
employees is equal to the contractual term of the option award. The risk-free interest rate is determined by reference to the
U.S. Treasury yield curve in effect at the time of grant of the award for time periods approximately equal to the expected
term of the award. The Company uses an expected dividend yield of zero based on the fact that the Company has never
paid cash dividends and does not expect to pay cash dividends in the future.
The fair value of each RSU is measured using the closing price of the Company’s common stock on the date of
grant.
Patent Costs
All patent related costs incurred in connection with filing and prosecuting patent applications are expensed as
incurred due to the uncertainty about the recovery of the expenditure. Amounts incurred are classified as general and
administrative expenses.
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Foreign Currency Translation
The reporting currency of the Company is the U.S. Dollar. The functional currency of ATIL, the Company’s
wholly-owned subsidiary, is the British Pound. Assets and liabilities of ATIL are translated into U.S. Dollars based on
exchange rates at the end of each reporting period. Revenues and expenses are translated at average exchange rates during
the reporting period. Gains and losses arising from the translation of assets and liabilities are included as a component of
accumulated other comprehensive loss within the Company’s consolidated balance sheet. Gains and losses resulting from
foreign currency transactions are reflected within the Company’s consolidated statement of operations. The Company has
not utilized foreign currency hedging strategies to mitigate the effect of its foreign currency exposure.
Income Taxes
The Company accounts for income taxes using the asset and liability method, which requires the recognition of
deferred tax assets and liabilities for the expected future tax consequences of events that have been recognized in the
financial statements or in the Company’s tax returns. Deferred taxes are determined based on the difference between the
financial statement and tax basis of assets and liabilities using enacted tax rates in effect in the years in which the
differences are expected to reverse. Changes in deferred tax assets and liabilities are recorded in the provision for income
taxes. The Company assesses the likelihood that its deferred tax assets will be recovered from future taxable income and,
to the extent it believes, based upon the weight of available evidence, that it is more likely than not that all or a portion of
the deferred tax assets will not be realized, a valuation allowance is established through a charge to income tax expense.
Potential for recovery of deferred tax assets is evaluated by estimating the future taxable profits expected and considering
prudent and feasible tax planning strategies.
The Company accounts for uncertainty in income taxes recognized in the consolidated financial statements by
applying a two-step process to determine the amount of tax benefit to be recognized. First, the tax position must be
evaluated to determine the likelihood that it will be sustained upon external examination by the taxing authorities. If the tax
position is deemed more likely than not to be sustained, the tax position is then assessed to determine the amount of benefit
to recognize in the consolidated financial statements. The amount of the benefit that may be recognized is the largest
amount that has a greater than 50% likelihood of being realized upon ultimate settlement. The provision for income taxes
includes the effects of any resulting tax reserves, or unrecognized tax benefits, that are considered appropriate as well as
the related net interest and penalties.
Comprehensive Loss
Comprehensive loss includes net loss as well as other changes in stockholders’ equity that result from transactions
and economic events other than those with stockholders. Comprehensive loss is comprised of net loss, foreign currency
translation adjustments and unrealized gains (losses) on marketable securities.
Net Loss per Share
Basic net loss per share is computed using the weighted average number of common shares outstanding during the
period. Diluted net loss per share is computed using the sum of the weighted average number of common shares
outstanding during the period, plus the weighted average number of potential shares of common stock from the assumed
exercise of stock options and warrants and the assumed vesting of RSUs, if dilutive. Since the Company was in a net loss
position, basic and diluted net loss per share was the same for each of the periods presented.
Fair Value Measurements
Certain assets and liabilities are carried at fair value under GAAP. Fair value is defined as the exchange price that
would be received for an asset or paid to transfer a liability in the principal or most advantageous market for the asset or
liability in an orderly transaction between market participants on the measurement date. Valuation techniques used to
measure fair value must maximize the use of observable inputs and minimize the use of unobservable inputs. Financial
assets and liabilities carried at fair value are to be classified and disclosed in one of the following three levels of the fair
value hierarchy, of which the first two are considered observable and the last is considered unobservable:
● Level 1 — Quoted prices in active markets for identical assets or liabilities.
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● Level 2 — Observable inputs (other than Level 1 quoted prices), such as quoted prices in active markets for
similar assets or liabilities, quoted prices in markets that are not active for identical or similar assets or
liabilities, or other inputs that are observable or can be corroborated by observable market data.
● Level 3 — Unobservable inputs that are supported by little or no market activity and that are significant to
determining the fair value of the assets or liabilities, including pricing models, discounted cash flow
methodologies and similar techniques.
The Company’s cash equivalents, marketable securities and contingent consideration are carried at fair value,
determined according to the fair value hierarchy described above. The carrying value of the Company’s accounts payable
and accrued expenses approximate fair value due to the short-term nature of these liabilities. The carrying value of the
Company’s debt approximates fair value due to the debt bearing a variable interest rate which is reflective of current
market rates.
Concentration of Credit Risk and of Significant Suppliers
Financial instruments that potentially expose the Company to concentrations of credit risk consist primarily of
cash, cash equivalents and marketable securities. The Company holds all cash, cash equivalents and marketable securities
balances at one accredited financial institution, in amounts that exceed federally insured limits. The Company does not
believe that it is subject to unusual credit risk beyond the normal credit risk associated with commercial banking
relationships.
The Company is dependent on third-party manufacturers to supply drug product, including all underlying
components, for its research and development activities, including preclinical and clinical testing. These activities could be
adversely affected by a significant interruption in the supply of active pharmaceutical ingredients or other components.
Segment Reporting
Operating segments are components of a company for which separate financial information is available and
evaluated regularly by the chief operating decision maker in assessing performance and deciding how to allocate resources.
The Company has two reportable segments, therapeutics and contract research. The therapeutics segment is focused on
identifying and developing innovative therapies to address significant unmet needs for immuno-inflammatory diseases.
The contract research segment earns revenue from the provision of laboratory services to clients through Confluence, the
Company’s wholly-owned subsidiary. Contract research revenue is generally evidenced by contracts with clients which are
on an agreed upon fixed-price, fee-for-service basis. The Company does not report balance sheet information by segment
since it is not reviewed by the chief operating decision maker, and all of the Company’s tangible assets are held in the
United States.
Recently Issued Accounting Pronouncements
In November 2018, the Financial Accounting Standards Board (“FASB”) issued ASU 2018-18, Collaborative
Arrangements (Topic 808): Clarifying the Interaction Between Topic 808 and Topic 606, which, among other things,
provides guidance on how to assess whether certain collaborative arrangement transactions should be accounted for under
Topic 606. The Company adopted this standard as of January 1, 2020, the impact of which on its consolidated financial
statements was not significant.
In August 2018, the FASB issued ASU 2018-15, Intangibles—Goodwill and Other—Internal-Use Software
(Subtopic 350-40). ASU 2018-15 requires a customer in a cloud computing arrangement that is a service contract to follow
the internal-use software guidance in ASC 350-40 to determine which implementation costs to capitalize as assets or
expense as incurred. The Company adopted this standard as of January 1, 2020, the impact of which on its consolidated
financial statements was not significant.
In August 2018, the FASB issued ASU 2018-13, Fair Value Measurement (Topic 820). The FASB developed the
amendments to ASC 820 as part of its broader disclosure framework project, which aims to improve the effectiveness of
disclosures in the notes to financial statements by focusing on requirements that clearly communicate the most important
information to users of the financial statements. This update eliminates certain disclosure requirements for fair value
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measurements for all entities, requires public entities to disclose certain new information and modifies some of the existing
disclosure requirements. The Company adopted this standard as of January 1, 2020, the impact of which on its
consolidated financial statements was not significant.
In June 2018, the FASB issued ASU 2018-07, Compensation—Stock Compensation (Topic 718). The
amendments in this ASU expand the scope of Topic 718 to include stock-based compensation arrangements with
nonemployees except for specific guidance on option pricing model inputs and cost attribution. The Company adopted this
standard as of January 1, 2019, the impact of which on its consolidated financial statements was not significant.
In February 2016, the FASB issued ASU 2016-02, Leases (Topic 842). In July 2018, the FASB issued ASU 2018-
10, Codification Improvements to Topic 842, Leases, and 2018-11, Targeted Improvements, which included a number of
technical corrections and improvements, including additional options for transition. The new standard establishes a right-
of-use model that requires a lessee to record a right-of-use asset and a lease liability on the balance sheet for all leases with
terms longer than 12 months. Leases are classified as either finance or operating, with classification affecting the pattern of
expense recognition in the income statement. The amendments in ASU 2016-02 must be applied to all leases existing at
the date a company initially applies the standard. The Company adopted the new standard as of January 1, 2019, using the
effective date as the date of its initial application, and used the modified retrospective approach. The adoption of ASU
2016-02 resulted in the Company recording additional assets and liabilities of $2.1 million and $2.3 million, respectively,
upon adoption on January 1, 2019. The adoption of ASU 2016-02 did not have a material impact on the Company’s
consolidated statement of operations and comprehensive loss or cash flows.
3. RHOFADE
Disposition - Asset Purchase Agreement with EPI Health, LLC
In October 2019, the Company entered into an asset purchase agreement with EPI Health pursuant to which the
Company sold the worldwide rights to RHOFADE, which included the assignment of certain licenses for related
intellectual property assets (the “Disposition”).
Pursuant to the asset purchase agreement, EPI Health paid the Company an upfront payment of $35.2 million. In
addition, EPI Health agreed to pay the Company (i) potential sales milestone payments of up to $20.0 million in the
aggregate upon the achievement of specified levels of net sales of products as defined in the asset purchase agreement, (ii)
a specified high single-digit royalty calculated as a percentage of net sales, on a product-by-product and country-by-
country basis, until the date that the patent rights related to a particular product, such as RHOFADE, have expired,
provided, that with respect to sales of RHOFADE in any territory outside of the United States, such royalty shall be paid
until the date that the RHOFADE patent rights in the particular country have expired or, if later, 10 years from the date of
the first commercial sale of RHOFADE in such country and (iii) 25% of any upfront, license, milestone, maintenance or
fixed payment received by EPI Health in connection with any license or sublicense of the assets transferred in the
Disposition in any territory outside of the United States, subject to specified exceptions. Finally, EPI Health agreed to
assume the Company’s obligation to pay specified royalties and milestone payments under certain agreements with third
parties.
Acquisition – Asset Purchase Agreement with Allergan Sales, LLC
In November 2018, the Company acquired the worldwide rights to RHOFADE, which included an exclusive
license to certain intellectual property, from Allergan Sales, LLC (“Allergan”) pursuant to an asset purchase agreement.
The acquisition of RHOFADE was accounted for as an asset acquisition in accordance with FASB ASC 805-50.
The following table summarizes the fair value of assets acquired in the acquisition of RHOFADE:
(In thousands)
Inventory
Intangible assets, net
Total assets acquired
$
$
893
66,229
67,122
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4. Fair Value of Financial Assets and Liabilities
The following tables present information about the fair value measurements of the Company’s financial assets and
liabilities which are measured at fair value on a recurring and non-recurring basis, and indicate the level of the fair value
hierarchy utilized to determine such fair values:
(In thousands)
Assets:
Cash equivalents
Marketable securities
Total assets
Liabilities:
Acquisition-related contingent consideration
Total liabilities
(In thousands)
Assets:
Cash equivalents
Marketable securities
Total assets
Level 1 Level 2
Level 3
Total
December 31, 2020
$ 14,955
—
$ 14,955
$
1,500
32,068
$ 33,568
$
$
— $ 16,455
—
32,068
— $ 48,523
$
$
— $
— $
— $
— $
4,061
4,061
$
$
4,061
4,061
Level 1 Level 2 Level 3
Total
December 31, 2019
$ 21,277
—
$ 21,277
$
— $
39,078
$ 39,078
$
— $ 21,277
39,078
—
— $ 60,355
Liabilities:
Acquisition-related contingent consideration
Total liabilities
$
$
— $
— $
— $ 1,668
— $ 1,668
$
$
1,668
1,668
As of December 31, 2020 and 2019, the Company’s cash equivalents included a money market fund, which was
valued based upon Level 1 inputs. Cash equivalents as of December 31, 2020 also included commercial paper, which was
valued based upon Level 2 inputs. The Company’s marketable securities as of December 31, 2020 and 2019 included U.S.
government agency debt securities, commercial paper and asset-backed debt securities, which were valued based upon
Level 2 inputs. Marketable securities as of December 31, 2019 also included corporate debt securities, which were valued
based upon Level 2 inputs.
In determining the fair value of its Level 2 investments, the Company relied on quoted prices for identical
securities in markets that are not active. These quoted prices were obtained by the Company with the assistance of a third-
party pricing service based on available trade, bid and other observable market data for identical securities. Quarterly, the
Company compares the quoted prices obtained from the third-party pricing service to other available independent pricing
information to validate the reasonableness of the quoted prices provided. The Company evaluates whether adjustments to
third-party pricing are necessary and, historically, the Company has not made adjustments to quoted prices obtained from
the third-party pricing service. During the years ended December 31, 2020 and 2019, there were no transfers between
Level 1, Level 2 and Level 3.
The increase in contingent consideration of $2.4 million during the year ended December 31, 2020 was primarily
due to updates to the Company’s assumptions resulting from the successful completion of a Phase 1 clinical trial for ATI-
450 and the submission and allowance of an Investigational New Drug Application (“IND”) for ATI-1777. The change in
acquisition-related contingent consideration of $0.7 million during the year ended December 31, 2019 was the result of
updates to the Company’s assumptions as a result of the submission and allowance of an IND for ATI-450.
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As of December 31, 2020 and 2019, the fair value of the Company’s available-for-sale marketable securities by
type of security was as follows:
(In thousands)
Marketable securities:
Commercial paper
Asset-backed debt securities
U.S. government agency debt securities
Total marketable securities
(In thousands)
Marketable securities:
Corporate debt securities
Commercial paper
Asset-backed debt securities
U.S. government agency debt securities
Total marketable securities
5. Property and Equipment, Net
December 31, 2020
Gross
Gross
Amortized Unrealized Unrealized
Gain
Loss
Cost
Fair
Value
$ 20,483
4,036
7,547
$ 32,066
$
$
— $
1
1
2
$
— $ 20,483
4,037
—
—
7,548
— $ 32,068
December 31, 2019
Gross
Gross
Amortized
Cost
Unrealized Unrealized
Gain
Loss
Fair
Value
$ 7,815
15,129
8,004
8,126
$ 39,074
$
$
2
—
4
1
7
$
$
— $ 7,817
— 15,129
8,008
—
8,124
(3)
$ 39,078
(3)
Property and equipment, net consisted of the following:
(In thousands)
Computer equipment
Finance lease right-of-use assets
Lab equipment
Furniture and fixtures
Leasehold improvements
Property and equipment, gross
Accumulated depreciation
Property and equipment, net
December 31,
2020
December 31,
2019
$
$
1,197 $
—
1,340
617
1,123
4,277
(2,623)
1,654
$
1,315
435
1,250
647
889
4,536
(2,066)
2,470
Depreciation expense was $1.1 million, $1.5 million and $1.2 million for the years ended December 31, 2020,
2019 and 2018, respectively.
6. Intangible Assets
Intangible assets consisted of the following:
Gross Cost
Accumulated Amortization
(In thousands, except years)
Other intangible assets
IPR&D
Total intangible assets
Remaining December 31, December 31, December 31, December 31,
Life (years)
6.6
na
2020
2019
2019
2020
751
6,629
7,380 $
751
6,629
7,380 $
257
—
257 $
181
—
181
$
Amortization expense was $75 thousand for each of the years ended December 31, 2020, 2019 and 2018.
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As of December 31, 2020, estimated future amortization expense is as follows:
(In thousands)
2021
2022
2023
2024
2025
Thereafter
Total
$
Year Ending
December 31,
75
75
75
75
75
119
494
$
7. Accrued Expenses
Accrued expenses consisted of the following:
(In thousands)
Employee compensation expenses
Research and development expenses
Other
Total accrued expenses
8. Debt
Loan and Security Agreement – Silicon Valley Bank
December 31, December 31,
2020
2019
$
$
3,971 $
761
1,174
5,906 $
3,321
2,857
1,543
7,721
In March 2020, the Company entered into a Loan and Security Agreement with Silicon Valley Bank (“SVB”).
The Loan and Security Agreement provides for $11.0 million in term loans, of which the Company borrowed the entire
amount on March 30, 2020. The Loan and Security Agreement is secured by substantially all of the assets of the Company
other than intellectual property. In connection with the Loan and Security Agreement, the Company issued to SVB a
warrant to purchase up to 460,251 shares of common stock (the “Warrant”) (see Note 9). The proceeds of the Loan and
Security Agreement were allocated to the term loan and Warrant using a relative fair value approach.
The term loan repayment schedule provides for interest only payments beginning April 1, 2020 and continuing
through March 1, 2022, followed by 24 consecutive equal monthly installments of principal, plus monthly payments of
accrued interest, starting on April 1, 2022 and continuing through the maturity date of March 1, 2024. All outstanding
principal and accrued and unpaid interest will be due and payable on the maturity date. The Loan and Security Agreement
provides for an annual interest rate equal to the greater of (i) the prime rate then in effect as reported in The Wall Street
Journal plus 2% and (ii) 6.75%.
The Loan and Security Agreement includes a final payment fee equal to 5% of the original principal amount
borrowed. The Company has the option to prepay the outstanding balance of the term loans in full, subject to a prepayment
premium of (i) 3% of the original principal amount borrowed for any prepayment on or prior to the first anniversary of
March 30, 2020, (ii) 2% of the original principal amount borrowed for any prepayment after the first anniversary and on or
before the second anniversary of March 30, 2020 or (iii) 1% of the original principal amount borrowed for any prepayment
after the second anniversary of March 30, 2020 but before March 1, 2024.
Loan and Security Agreement – Oxford Finance LLC
In October 2018, the Company entered into a Loan and Security Agreement with Oxford Finance LLC. The Loan
and Security Agreement provided for up to $65.0 million in term loans, of which the Company borrowed $30.0 million in
October 2018. In October 2019, the Company repaid in full the $30.0 million that was outstanding under the Loan and
Security Agreement, together with all accrued and unpaid interest and fees.
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9. Stockholders’ Equity
Preferred Stock
As of December 31, 2020 and 2019, the Company’s amended and restated certificate of incorporation authorized
the Company to issue 10,000,000 shares of undesignated preferred stock. There were no shares of preferred stock
outstanding as of December 31, 2020 and 2019.
Common Stock
As of December 31, 2020 and 2019, the Company’s amended and restated certificate of incorporation authorized
the Company to issue 100,000,000 shares of $0.00001 par value common stock.
Each share of common stock entitles the holder to one vote on all matters submitted to a vote of the Company’s
stockholders. Common stockholders are entitled to receive dividends, as may be declared by the board of directors, if any,
subject to any preferential dividend rights of any series of preferred stock that may be outstanding. No dividends have
been declared through December 31, 2020.
October 2018 Public Offering
In October 2018, the Company entered into an underwriting agreement pursuant to which the Company issued
and sold 9,941,750 shares of common stock under registration statements on Form S-3, including the underwriters’ full
exercise of their option to purchase additional shares. The shares of common stock were sold to the public at a price of
$10.75 per share, for gross proceeds of $106.9 million. The Company paid underwriting discounts and commissions of
$6.4 million to the underwriters in connection with the offering. In addition, the Company incurred expenses of $0.3
million in connection with the offering. The net offering proceeds received by the Company, after deducting underwriting
discounts and commissions and offering expenses, were $100.2 million.
Warrants
The Warrant issued to SVB in March 2020 had an initial exercise price of $0.956 per share, subject to adjustment
as provided in the Warrant. The Warrant became immediately exercisable in full upon the funding of the term loan facility.
The Company assigned a fair value of $0.4 million to the Warrant using a Black-Scholes valuation methodology, and also
concluded that the Warrant was indexed to its own stock and therefore classified the Warrant as an equity instrument. In
January 2021, SVB net exercised the Warrant in full, and the Company issued to SVB 388,119 shares of common stock.
Equity Purchase Agreement with Lincoln Park Capital Fund, LLC
In August 2020, the Company entered into the Purchase Agreement with Lincoln Park which provided that, upon
the terms and subject to the conditions and limitations set forth therein, the Company may sell to Lincoln Park, at its
discretion, up to $15.0 million of shares of its common stock over the 36-month term of the Purchase Agreement. Upon
execution of the Purchase Agreement, the Company issued 121,584 shares of its common stock to Lincoln Park as
commitment shares in accordance with the closing conditions contained within the Purchase Agreement. The commitment
shares were valued using the closing price of the Company’s common stock on the effective date of the Purchase
Agreement resulting in an aggregate fair value of $0.3 million. Through December 31, 2020, the Company sold 2,111,170
shares of its common stock to Lincoln Park under the Purchase Agreement for net proceeds of $7.7 million. The Company
terminated the Purchase Agreement in January 2021. The Company did not sell any additional shares prior to terminating
the Purchase Agreement in January 2021 in connection with the public offering of common stock described below (see
Note 21).
10. Stock-Based Awards
2015 Equity Incentive Plan
In September 2015, the Company’s board of directors adopted the 2015 Equity Incentive Plan (the “2015 Plan”),
and the Company’s stockholders approved the 2015 Plan. The 2015 Plan became effective in connection with the
Company’s initial public offering. Beginning at the time the 2015 Plan became effective, no further grants may be made
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under the Company’s 2012 Equity Compensation Plan, as amended and restated (the “2012 Plan”). The 2015 Plan
provides for the grant of incentive stock options, nonqualified stock options, stock appreciation rights, restricted stock
awards, RSU awards, performance stock awards, cash-based awards and other stock-based awards. The number of shares
initially reserved for issuance under the 2015 Plan was 1,643,872 shares of common stock. The number of shares of
common stock that may be issued under the 2015 Plan will automatically increase on January 1 of each year ending on
January 1, 2025, in an amount equal to the lesser of (i) 4.0% of the shares of the Company’s common stock outstanding on
December 31 of the preceding calendar year or (ii) an amount determined by the Company’s board of directors. The shares
of common stock underlying any awards that expire, are otherwise terminated, settled in cash or repurchased by the
Company under the 2015 Plan and the 2012 Plan will be added back to the shares of common stock available for issuance
under the 2015 Plan. As of December 31, 2020, 2,423,020 shares remained available for grant under the 2015 Plan. As of
January 1, 2021, the number of shares of common stock that may be issued under the 2015 Plan was automatically
increased by 1,804,372 shares.
2017 Inducement Plan
In July 2017, the Company’s board of directors adopted the 2017 Inducement Plan (the “2017 Inducement Plan”).
The 2017 Inducement Plan is a non-stockholder approved stock plan adopted pursuant to the “inducement exception”
provided under Nasdaq listing rules. The Company had 443,000 stock options and 28,895 RSUs outstanding as of
December 31, 2020 under the 2017 Inducement Plan. All shares of common stock that were eligible for issuance under the
2017 Inducement Plan after October 1, 2018, including any shares underlying any awards that expire or are otherwise
terminated, reacquired to satisfy tax withholding obligations, settled in cash or repurchased by the Company in the future
that would have been eligible for re-issuance under the 2017 Inducement Plan, were retired.
2012 Equity Compensation Plan
Upon the 2015 Plan becoming effective, no further grants can be made under the 2012 Plan. The Company
granted a total of 1,140,524 stock options under the 2012 Plan, of which 549,561 and 745,735 were outstanding as of
December 31, 2020 and 2019, respectively. Stock options granted under the 2012 Plan vested over four years and expire
after ten years.
Stock Option Valuation
The weighted average assumptions the Company used to estimate the fair value of stock options granted during
the years ended December 31, 2020, 2019 and 2018 were as follows:
Risk-free interest rate
Expected term (in years)
Expected volatility
Expected dividend yield
2020
0.87 %
6.1
Year Ended
December 31,
2019
2.27 %
6.2
85.19 % 99.36 % 96.78 %
0 %
2018
2.66 %
6.3
0 %
0 %
The Company recognizes compensation expense for awards over their vesting period. Compensation expense for
awards includes the impact of forfeiture in the period when they occur.
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Stock Options
The following table summarizes stock option activity for the years ended December 31, 2020, 2019 and 2018:
Weighted
Average
Exercise
Price
Weighted
Average
Remaining
Contractual
Term
(in years)
8.3
Aggregate
Intrinsic
Value
$ 19,812
724
(In thousands, except share and per share data and years)
Outstanding as of December 31, 2017
Granted
Exercised
Forfeited and cancelled
Outstanding as of December 31, 2018
Granted
Exercised
Forfeited and cancelled
Outstanding as of December 31, 2019
Granted
Exercised
Forfeited and cancelled
Outstanding as of December 31, 2020
Options vested and expected to vest as of December 31, 2020
Options exercisable as of December 31, 2020
Number
of Shares
3,328,757
1,459,800
(59,450)
(447,026)
4,282,081
44,500
(142,779)
(1,081,581)
3,102,221
734,800
(53,737)
(911,786)
2,871,498
2,871,498
1,844,197
$
$
$
$
$
$
20.69
20.97
9.70
24.62
20.53
5.75
1.33
23.01
20.33
1.30
1.30
22.41
15.16
15.16
18.92
7.9
$
2,404
112
6.6
$
148
145
4,890
4,890
1,424
6.8
6.8
5.8
$
$
$
The weighted average grant date fair value of stock options granted during the years ended December 31, 2020,
2019 and 2018 was $0.93, $4.63 and $16.55 per share, respectively.
Restricted Stock Units
The following table summarizes RSU activity for the years ended December 31, 2020, 2019 and 2018.
Weighted
Average
Grant Date Aggregate
Number
Fair Value
Intrinsic
(In thousands, except share and per share data)
Outstanding as of December 31, 2017
Granted
Vested
Forfeited and cancelled
Outstanding as of December 31, 2018
Granted
Vested
Forfeited and cancelled
Outstanding as of December 31, 2019
Granted
Vested
Forfeited and cancelled
Outstanding as of December 31, 2020
103
of Shares
283,553 $
552,060
(140,497)
(68,709)
626,407 $
3,650,942
(173,444)
(510,990)
3,592,915 $
1,168,805
(1,804,429)
(713,134)
2,244,157 $
Per Share
27.02
19.03
27.22 $
23.65
20.30
3.56
21.31 $
10.63
4.62
1.36
3.33 $
4.77
3.83
Value
2,158
799
2,607
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Stock-Based Compensation
Stock-based compensation expense included in total costs and expenses on the consolidated statement of
operations included the following:
(In thousands)
Cost of revenue
Research and development
General and administrative
Total stock-based compensation expense
Year Ended
December 31,
2019
703
5,091
10,288
$ 16,082
2018
$
766
6,480
9,317
$ 16,563
2020
$
946 $
2,919
7,342
$ 11,207
As of December 31, 2020, the Company had unrecognized stock-based compensation expense for stock options
and RSUs of $4.2 million and $5.5 million, respectively, which is expected to be recognized over weighted average periods
of 1.3 years and 1.8 years, respectively.
11. Net Loss per Share
Basic and diluted net loss per share is summarized in the following table:
(In thousands, except for share and per share data)
Numerator:
Net loss
Denominator:
Year Ended
December 31,
2019
2018
2020
$
(51,015)
$
(161,354)
$
(132,738)
Weighted average shares of common stock outstanding, basic and
diluted
Net loss per share, basic and diluted
42,539,293
(1.20)
$
41,323,921
(3.90)
$
32,909,762
(4.03)
$
The Company’s potentially dilutive securities, which included stock options, RSUs and warrants, have been
excluded from the computation of diluted net loss per share since the effect would be to reduce the net loss per share.
Therefore, the weighted average number of shares of common stock outstanding used to calculate both basic and diluted
net loss per share attributable to common stockholders is the same. The following table presents potential shares of
common stock excluded from the calculation of diluted net loss per share attributable to common stockholders for the years
ended December 31, 2020, 2019 and 2018. All share amounts presented in the table below represent the total number
outstanding as of December 31 of each year.
Options to purchase common stock
Restricted stock unit awards
Warrants
Total potential shares of common stock
12. Leases
2020
2,871,498
2,244,157
460,251
5,575,906
December 31,
2019
2018
3,102,221 4,282,081
626,407
3,592,915
—
4,908,488
—
6,695,136
The Company has operating leases for office space and laboratory facilities, and had finance leases for its
laboratory equipment. As a result of the Company’s decision to actively seek partners for its commercial products, the
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Company terminated the finance leases for its fleet vehicles and recognized a loss on lease termination of $0.2 million
during the year ended December 31, 2019. The components of lease expense were as follows:
(In thousands)
Operating lease expense
Finance Leases:
Amortization of right-to-use assets
Interest expense
Total finance lease expenses
Year Ended
December 31,
2020
2019
$
1,013 $
808
$
$
113
5
118
$
$
443
87
530
Rent expense was $1.0 million, $1.0 million and $0.9 million for the years ended December 31, 2020, 2019 and
2018, respectively, which was recognized on a straight-line basis over the term of the lease.
Operating Leases
Agreements for Office Space
The Company has a sublease agreement with Auxilium Pharmaceuticals, LLC (the “Sublandlord”) pursuant to
which it subleases 33,019 square feet of office space for its headquarters in Wayne, Pennsylvania. The sublease has a term
that runs through October 2023. If for any reason the lease between Chesterbrook Partners, LP (“Landlord”) and
Sublandlord is terminated or expires prior to October 2023, the Company’s sublease will automatically terminate. In
December 2020, the Company entered into a sub-sublease agreement under which it sub-subleased 8,115 square feet. The
sub-sublease term runs concurrent with the original sublease agreement.
In February 2019, the Company entered into a sublease agreement with a third party for 20,433 square feet of
office and laboratory space in St. Louis, Missouri. The lease commenced in June 2019 and has a term that runs through
June 2029.
Supplemental balance sheet information related to operating leases is as follows:
(In thousands)
Operating Leases:
Gross cost
Accumulated amortization
Other assets
Current portion of lease liabilities
Other liabilities
Total operating lease liabilities
Finance Leases
Laboratory Equipment
December 31, December 31,
2020
2019
$
$
$
$
5,240 $
(1,111)
4,129 $
603 $
2,894
3,497 $
5,213
(480)
4,733
526
3,548
4,074
The Company leased laboratory equipment which it used in its laboratory space in St. Louis, Missouri
under two finance lease financing arrangements which the Company entered into in August 2017 and October 2017, and
which terms ended in October 2020 and December 2020, respectively.
Fleet Vehicles
The Company leased automobiles for its sales force and other field-based employees under the terms of a master
lease agreement with a third party. The lease term for each automobile began on the date the Company took delivery and
continued for a period of four years. The Company returned all leased vehicles during the year ended December 31, 2019.
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Supplemental balance sheet information related to finance leases is as follows:
(In thousands)
Finance Leases:
Property and equipment, gross
Accumulated depreciation
Property and equipment, net
Current portion of lease liabilities
Other liabilities
Total finance lease liabilities
Supplemental information related to operating and finance leases is as follows:
(In thousands, except for years and percentages)
Supplemental Cash Flow Lease Information:
Operating cash flows from operating leases
Operating cash flows from finance leases
Financing cash flows from finance leases
Leased assets obtained in exchange for new operating lease liabilities
Weighted-Average Remaining Lease Term (in years):
Operating leases
Finance leases
Weighted-Average Discount Rate:
Operating leases
Finance leases
December 31, December 31,
2020
2019
$
$
$
$
$
$
$
$
— $
—
— $
— $
—
— $
435
(322)
113
111
21
132
Year Ended
December 31,
2020
2019
907
5
137
$
$
$
755
87
523
— $
3,060
6.0
—
6.8
0.9
10.1 %
10.0 %
10.1%
10.0%
Future minimum lease payments under operating and finance lease agreements are as follows:
(In thousands)
Year Ending December 31,
2021
2022
2023
2024
2025
Thereafter
Total undiscounted lease payments
Less: unrecognized interest
Total lease liability
13. Income Taxes
Operating
Leases
924
949
866
343
352
1,301
4,735
(1,238)
3,497
$
$
During the years ended December 31, 2020, 2019 and 2018, the Company did not record an income tax benefit for
net operating losses incurred in each year due to the uncertainty of realizing a benefit from those items.
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Loss before income taxes is allocated as follows:
Year Ended December 31,
(In thousands)
U.S. operations
Foreign operations
Loss before income taxes
2020
2019
$ (51,215) $ (161,192) $ (132,473)
(265)
$ (51,197) $ (161,354) $ (132,738)
(162)
2018
18
A reconciliation of the U.S. federal statutory income tax rate to the Company’s effective income tax rate is as
follows:
Federal statutory income tax rate
State taxes, net of federal benefit
Research and development tax credits
Permanent differences
Change in deferred tax asset valuation allowance
Effective income tax rate
Year Ended December 31,
2019
(21.0)%
(6.6)
(1.5)
3.0
26.2
0.1 %
2020
(21.0)%
(7.5)
(2.6)
2.6
28.1
(0.4)%
2018
(21.0)%
(3.5)
(2.1)
0.8
25.7
(0.1)%
Deferred tax liabilities, net consisted of the following:
(In thousands)
Deferred tax assets:
Net operating loss carryforwards
Capitalized start-up costs
Research and development tax credit carryforwards
Capitalized research and development expense
Stock‑based compensation expense
Accrued compensation
Lease liabilities
Other
Total deferred tax assets
Deferred tax liabilities:
Property and equipment
Intangible asset
Right-to-use assets
Other
Total deferred tax liabilities
Valuation allowance
Deferred tax liabilities, net
December 31,
2020
2019
$
101,277
6,509
8,732
4,611
14,526
745
888
602
137,890
$
90,298
6,904
7,417
4,456
12,973
588
945
618
124,199
(209)
(2,033)
(1,026)
(430)
(3,698)
(134,559)
(367)
$
(206)
(1,741)
(1,235)
(600)
(3,782)
(120,966)
(549)
$
As of December 31, 2020, the Company had federal and state net operating loss (“NOL”) carryforwards of $367.6
million and $369.6 million, respectively, which will begin to expire in 2032. As of December 31, 2020, the Company also
had federal research and development tax credit carryforwards of $8.6 million which will begin to expire in 2032, and state
research and development tax credit carryforwards of $0.1 million which will begin to expire in 2022. The Company also
has $0.2 million of loss carryforwards in the United Kingdom which can be carried forward indefinitely. Utilization of the
NOLs and research and development tax credit carryforwards in the United States may be subject to a substantial annual
limitation under Section 382 of the Internal Revenue Code of 1986 due to ownership changes that may have occurred
previously or that could occur in the future. These ownership changes may limit the amount of carryforwards that can be
utilized annually to offset future taxable income. In general, an ownership change, as defined by Section 382, results from
transactions increasing the ownership of certain stockholders or public groups in the stock of a corporation by more than
50% over a three-year period. The Company has completed an analysis under Section 382 for NOLs generated from July
13, 2012 through July 20, 2020. Although the Company has experienced Section 382 ownership changes since 2012, the
Company has concluded that it should have sufficient ability to utilize NOLs accumulated during the periods
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tested. The Company has not yet determined if a Section 382 ownership change has occurred after July 20, 2020. In
addition, the Company may experience ownership changes in the future as a result of subsequent shifts in its stock
ownership, some of which may be outside of the Company’s control.
The Company has evaluated the positive and negative evidence bearing upon its ability to realize the deferred tax
assets. The Company considered its history of cumulative net losses incurred since inception, its lack of substantial
revenue generated to date, and its forecasted future operating losses and concluded that it is more likely than not that the
Company will not realize the benefits of its deferred tax assets. Accordingly, a full valuation allowance has been
established against the deferred tax assets as of December 31, 2020 and 2019. The Company evaluates positive and
negative evidence of its ability to realize deferred tax assets at each reporting period.
Changes in the valuation allowance for deferred tax assets during the years ended December 31, 2020, 2019 and
2018 related primarily to the increases in NOLs, capitalized start-up costs, and research and development tax credit
carryforwards and were as follows:
(In thousands)
Valuation allowance at beginning of year
Decreases recorded as benefit to income tax provision
Decreases recorded to opening balance sheet
Increases recorded to income tax provision
Valuation allowance as of end of year
2020
2018
Year Ended December 31,
2019
$ (120,966) $ (80,985) $ (46,878)
—
—
(34,107)
$ (80,985)
—
58
(13,651)
$ (134,559)
—
—
(39,981)
$ (120,966)
The Company files tax returns as prescribed by the tax laws of the jurisdictions in which it operates. In the
normal course of business, the Company is subject to examination by federal and state jurisdictions, where applicable.
There are currently no pending income tax examinations. The Company’s tax years are still open under statute from 2017
to the present. All open years may be examined to the extent that tax credit or NOLs are used in future periods. The
Company’s policy is to record interest and penalties related to income taxes as part of its income tax provision.
14. Related Party Transactions
Mallinckrodt plc
In April 2018, Bryan Reasons was appointed to the Company’s board of directors. Subsequently, in March 2019,
Mr. Reasons became the Chief Financial Officer of Mallinckrodt plc. Prior to Mr. Reasons joining Mallinckrodt plc, in
November 2018 the Company entered into a master services agreement with a subsidiary of Mallinckrodt plc pursuant to
which Confluence provides laboratory services to the subsidiary (“Mallinckrodt”) in the ordinary course of business. Mr.
Reasons was not involved in the negotiation or execution of the agreement, but may be deemed to have an interest in the
ongoing transactions based on his employment as an executive officer of Mallinckrodt plc. During the years ended
December 31, 2020 and 2019, the Company invoiced Mallinckrodt for $0.3 million and $0.1 million, respectively, under
the master services agreement. As of December 31, 2020 and 2019, the Company had $0 of outstanding accounts
receivable balances from Mallinckrodt. Mr. Reasons had no financial interest in these transactions.
15. Agreements Related to Intellectual Property
Asset Purchase Agreement – EPI Health, LLC
In October 2019, the Company sold RHOFADE to EPI Health pursuant to an asset purchase agreement. EPI
Health agreed to pay the Company a high single-digit royalty calculated as a percentage of net sales on a country-by-
country basis until the date that the patent rights related to RHOFADE have expired or, if later, ten years from the date of
the first commercial sale of RHOFADE in such country. The Company recorded royalty income under the asset purchase
agreement of $0.7 million and $0 during the years ended December 31, 2020 and 2019, respectively. Royalty income is
included in other revenue on the consolidated statements of operations and comprehensive loss. EPI Health has also
agreed to pay the Company potential sales milestone payments of up to $20.0 million in the aggregate upon the
achievement of specified levels of net sales of products covered by the asset purchase agreement, and 25% of any upfront,
license, milestone, maintenance or fixed payment received by EPI Health in connection with any license or sublicense of
the assets transferred in the disposition in any territory outside of the United States, subject to specified exceptions.
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Asset Purchase Agreement – Allergan Sales, LLC
In November 2018, the Company acquired RHOFADE from Allergan pursuant to an asset purchase agreement.
The Company agreed to pay Allergan specified royalties, ranging from a mid-single digit percentage to a mid-teen
percentage of net sales, subject to specified reductions, limitations and other adjustments. The Company incurred royalties
earned by Allergan under the asset purchase agreement of $0, $1.4 million and $0.1 million during the years ended
December 31, 2020, 2019 and 2018, respectively.
Agreement and Plan of Merger - Confluence
In August 2017, the Company entered into an Agreement and Plan of Merger, pursuant to which it acquired
Confluence (the “Confluence Agreement”). In November 2018, a development milestone specified in the Confluence
Agreement was achieved, as a result of which the Company paid the former Confluence equity holders $2.5 million in cash
and issued 253,208 shares of its common stock with a fair value of $2.2 million. Under the Confluence Agreement, the
Company also agreed to pay the former Confluence equity holders aggregate remaining contingent consideration of up to
$75.0 million, based upon the achievement of specified regulatory and commercial milestones set forth in the Confluence
Agreement. In addition, the Company agreed to pay the former Confluence equity holders future royalty payments
calculated as a low single-digit percentage of annual net sales, subject to specified reductions, limitations and other
adjustments, until the date that all of the patent rights for that product have expired, as determined on a country-by-country
and product-by-product basis or, in specified circumstances, ten years from the first commercial sale of such product. In
addition to the payments described above, if the Company sells, licenses or transfers any of the intellectual property
acquired from Confluence pursuant to the Confluence Agreement to a third party, the Company will be obligated to pay the
former Confluence equity holders a portion of any consideration received from such sale, license or transfer in specified
circumstances.
License and Collaboration Agreement – Rigel Pharmaceuticals, Inc.
In August 2015, the Company entered into an exclusive, worldwide license and collaboration agreement with
Rigel Pharmaceuticals, Inc. (“Rigel”) for the development and commercialization of products containing two specified
JAK inhibitors, which the Company refers to as ATI-501 and ATI-502. During the year ended December 31, 2019, the
Company made a milestone payment of $4.0 million to Rigel upon the achievement of a specified development milestone
which is included in research and development expenses on the Company’s consolidated statement of operations. In
connection with an amendment of the agreement with Rigel in October 2019, the Company paid Rigel an amendment fee
of $1.5 million during the year ended December 31, 2020.
16. Retirement Savings Plan
The Company has a defined contribution savings plan under Section 401(k) of the Internal Revenue Code. This
plan covers substantially all employees who meet minimum age and service requirements and allows participants to defer a
portion of their annual compensation on a pre-tax basis. Company contributions to the plan may be made at the discretion
of the Company’s board of directors. The Company has elected to match 100% of employee contributions to the 401(k)
Plan up to 4% of the employee’s earnings, subject to certain limitations. Company contributions under the 401(k) Plan
were $0.4 million, $0.7 million and $0.7 million for the years ended December 31, 2020, 2019 and 2018, respectively.
17. Restructuring Charges
In September 2019, the Company announced the completion of a strategic review and its decision to refocus on its
immuno-inflammatory development programs and to actively seek partners for its commercial products. As a result, the
Company terminated 63 employees (“terminated employees”) and gave notice to an additional 23 employees (“noticed
employees”) who were asked to provide transition services through termination dates ranging between 4 to 10 months from
the date notice was given. The terminated employees were entitled to receive cash severance payments as well as cash
payments in lieu of sixty days’ notice required by the Worker Adjustment and Retraining Notification Act (the “WARN
Act”). The noticed employees were entitled to receive one-time cash severance payments which were not contingent upon
providing additional services to the Company. In addition, certain noticed employees earned retention bonuses if they
continued to be employed by the Company through certain termination dates. The Company recorded a restructuring
charge for the one-time severance and WARN Act payments, which was triggered immediately upon either
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terminating or giving notice to the impacted employees. The Company expensed the cost of retention bonuses for noticed
employees over their respective service terms. During the year ended December 31, 2020, the Company recognized
aggregate expenses of $0.1 million and made payments of $0.3 million related to termination benefits for employees.
During the year ended December 31, 2019, the Company recognized aggregate expenses of $2.7 million and made
payments of $2.3 million related to termination benefits for employees.
18. Discontinued Operations
The components of loss from discontinued operations as reported in the Company’s consolidated statement of
operations were as follows:
(In thousands, except share and per share data)
Revenues:
Product sales, net
Total revenue, net
Costs and expenses:
Cost of revenue (excludes amortization)
Research and development
Sales and marketing
General and administrative
Intangible asset impairment
Amortization of definite-lived intangible
Total costs and expenses
Income (loss) from operations
Year Ended
December 31,
2019
2018
2020
$
424 $
424
13,896
13,896
$
3,940
3,940
—
1
283
1
—
—
285
139
—
139
—
139
$
4,522
503
23,112
2,929
27,638
4,426
63,130
(49,234)
1,422
(47,812)
—
(47,812)
$
1,969
2,168
47,827
2,058
—
552
54,574
(50,634)
—
(50,634)
—
(50,634)
Other income, net
Income (loss) from discontinued operations before income taxes
Income tax benefit
Net income (loss) from discontinued operations
$
Net income (loss) from discontinued operations per share, basic and
diluted
Weighted average common shares outstanding, basic and diluted
$
0.00
42,539,293
$
(1.16)
41,323,921
$
(1.54)
32,909,762
The following table presents the details of product sales, net included in discontinued operations:
(In thousands)
ESKATA
RHOFADE
Total product sales, net
Year Ended
December 31,
2019
2020
$
$
— $
424
424
$
312
13,584
13,896
$
$
2018
2,804
1,136
3,940
The Company recorded $0.4 million of RHOFADE product sales, net during the year ended December 31, 2020
due to a reversal of previously accrued product sales-related reserves.
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The following table presents information related to assets and liabilities reported as discontinued operations in the
Company’s consolidated balance sheet:
(In thousands)
Accounts receivable, net
Discontinued operations - current assets
Accounts payable
Accrued expenses
Discontinued operations - current liabilities
December 31,
2020
December 31,
2019
$
$
$
$
— $
— $
1,175
1,936
3,111
$
$
4,966
4,966
1,705
2,452
4,157
The Company relied on Allergan to distribute RHOFADE on its behalf pursuant to the terms of a transition
services agreement. Accounts receivable, net as of December 31, 2019 included $5.0 million related to amounts invoiced
by Allergan for sales of RHOFADE.
The following table presents certain non-cash items related to discontinued operations, which are included in the
Company’s consolidated statement of cash flows:
(In thousands)
Depreciation and amortization
Stock-based compensation expense
Intangible asset impairment charge
Loss on disposal of property and equipment
Gain on sale of RHOFADE
Total non-cash items
Year Ended
December 31,
2020
2019
— $
—
—
—
—
—
— $
313
95
27,638
248
28,294
1,670
26,624
$
$
As a result of the Company’s decision to actively seek partners for its commercial products, the Company
terminated the finance leases for its fleet vehicles and recognized a loss on lease termination of $0.2 million in the year
ended December 31, 2019, which is included in other income, net in the Company’s consolidated statement of operations.
During the year ended December 31, 2019, the Company performed an impairment analysis of the RHOFADE
intangible asset due to its decision to discontinue commercial operations and actively seek a commercialization partner for
RHOFADE. The Company’s impairment analysis, which primarily utilized a third-party indication of fair value, resulted
in a fair value for the RHOFADE intangible asset which was less than its carrying value. As a result, the Company
recorded an impairment charge of $27.6 million to adjust the carrying value of the RHOFADE intangible asset to its net
realizable value.
19. Segment Information
The Company has two reportable segments, therapeutics and contract research. The therapeutics segment is
focused on identifying and developing innovative therapies to address significant unmet needs for immuno-inflammatory
diseases. The contract research segment earns revenue from the provision of laboratory services to clients through
Confluence, the Company’s wholly-owned subsidiary. Contract research revenue is generally evidenced by contracts with
clients which are on an agreed upon fixed-price, fee-for-service basis. Corporate and other includes general and
administrative expenses as well as eliminations of intercompany transactions. The Company does not report balance sheet
information by segment since it is not reviewed by the chief operating decision maker, and all of the Company’s tangible
assets are held in the United States.
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The Company’s results of operations by segment for the years ended December 31, 2020, 2019 and 2018 are
summarized in the tables below:
(In thousands)
Year Ended December 31, 2020
Total revenue
Cost of revenue
Research and development
General and administrative
Loss from operations
Income (loss) from discontinued operations
Year Ended December 31, 2019
Total revenue
Cost of revenue
Research and development
General and administrative
Goodwill impairment
Loss from operations
Loss from discontinued operations
Year Ended December 31, 2018
Revenue, net
Cost of revenue
Research and development
General and administrative
Loss from operations
Loss from discontinued operations
Intersegment Revenue
Therapeutics Research
$ 13,319
$
12,228
—
2,794
$ (1,703)
$
Contract Corporate
and Other
$ (7,533)
(7,095)
(439)
17,736
$ (17,735)
(1)
696
—
32,170
—
(31,474)
140
— $
$
$
Therapeutics Research
— $ 16,824
$
16,253
—
—
65,298
2,738
620
—
18,504
$ (2,167)
(84,422)
$
(46,305)
Contract Corporate
and Other
$ (12,597)
(12,198)
(399)
24,469
—
$ (24,469)
— $ (1,507)
$
$
Therapeutics Research
$ 13,135
$
11,399
—
2,181
(445)
Contract Corporate
and Other
$ (8,484)
(7,070)
(1,414)
23,420
$ (23,420)
— $ (2,058)
1,500
—
62,255
160
(60,915)
(48,576)
$
$
$
$
Total
Company
6,482
$
5,133
31,731
20,530
(50,912)
139
$
$
Total
Company
4,227
$
4,055
64,899
27,827
18,504
$ (111,058)
(47,812)
$
Total
Company
6,151
$
4,329
60,841
25,761
(84,780)
(50,634)
$
$
Revenue for the contract research segment included $7.5 million, $12.6 million and $8.5 million for services
performed on behalf of the therapeutics segment for the years ended December 31, 2020, 2019 and 2018, respectively. All
intersegment revenue has been eliminated in the Company’s consolidated statement of operations.
20. Legal Proceedings
Securities Class Action
On July 30, 2019, plaintiff Linda Rosi (“Rosi”) filed a putative class action complaint captioned Rosi v. Aclaris
Therapeutics, Inc., et al. in the U.S. District Court for the Southern District of New York against the Company and certain
of its executive officers. The complaint alleges that the defendants violated federal securities laws by, among other things,
failing to disclose an alleged likelihood that regulators would scrutinize advertising materials related to ESKATA and find
that the materials minimized the risks or overstated the efficacy of the product. The complaint seeks unspecified
compensatory damages on behalf of Rosi and all other persons and entities that purchased or otherwise acquired the
Company’s securities between May 8, 2018 and June 20, 2019.
On September 5, 2019, an additional plaintiff, Robert Fulcher (“Fulcher”), filed a substantially identical putative
class action complaint captioned Fulcher v. Aclaris Therapeutics, Inc., et al. in the same court against the same defendants.
On November 6, 2019, the court consolidated the Rosi and Fulcher actions (together, the “Consolidated Securities
Action”) and appointed Fulcher “lead plaintiff” for the putative class.
On January 24, 2020, Fulcher filed a consolidated amended complaint in the Consolidated Securities Action,
naming two additional executive officers as defendants, extending the putative class period to August 12, 2019, and adding
allegations concerning, among other things, alleged statements and omissions throughout the putative class period
concerning ESKATA’s risks, tolerability and effectiveness. The defendants filed a motion to dismiss the consolidated
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amended complaint on April 17, 2020. Fulcher filed an opposition to the defendants’ motion on June 15, 2020, and the
defendants filed a reply to such opposition on August 4, 2020. Oral argument on the pending motion to dismiss is
scheduled for February 25, 2021. The motion remains under judicial consideration.
The Company and the other defendants dispute plaintiffs’ claims in the Consolidated Securities Action and intend
to defend the matter vigorously. At this time, the Company cannot reasonably predict the outcome or potential loss, if any,
that could result from this matter.
Stockholder Derivative Action
On November 15, 2019, plaintiff Keith Allred (“Allred”) filed a derivative stockholder complaint captioned Allred
v. Walker et al. in the U.S. District Court for the Southern District of New York against certain of the Company’s directors
and executive officers. The complaint alleges that the defendants, among other things, breached their fiduciary duties as
directors and/or officers in connection with the claims alleged in the Consolidated Securities Action. The complaint seeks,
among other things, unspecified compensatory damages on behalf of the Company.
On November 25, 2019, an additional plaintiff, Bruce Brown (“Brown”), filed a substantially identical complaint
captioned Brown v. Walker et al. in the same court against the same defendants.
On December 12, 2019, the court consolidated the Allred and Brown actions under the caption In re Aclaris
Therapeutics, Inc. Derivative Litigation (the “Consolidated Derivative Action”) and directed that future derivative cases
filed in or transferred to the court arising out of substantially the same transactions or events be similarly consolidated.
Thereafter, on January 11, 2020, the court stayed – subject to certain conditions – all deadlines in the Consolidated
Derivative Action pending resolution of the defendants’ anticipated motion to dismiss the Consolidated Securities Action.
The defendants dispute plaintiffs’ claims in the Consolidated Derivative Action and intend to defend the matter
vigorously. At this time, the Company cannot reasonably predict the outcome or potential loss, if any, that could result
from this matter.
Product Liability Lawsuit
On December 18, 2020, plaintiff Daurie Mancini filed an amended complaint under the caption Daurie Mancini v.
Aclaris Therapeutics, Inc. et al in the Superior Court of New Jersey Ocean County against the Company and certain third
parties alleging injuries as a result of the plaintiff’s alleged treatment with ESKATA in 2019. The amended complaint seeks
unspecified compensatory and punitive damages. On January 19, 2021, the Company’s deadline to answer, move against or
otherwise respond to the amended complaint was extended until March 15, 2021.
The Company disputes plaintiff’s claims and intends to defend the matter vigorously. At this time, the Company
cannot reasonably predict the outcome or potential loss, if any, that could result from this matter.
21. Subsequent Events
January 2021 Public Offering
In January 2021, the Company closed a public offering in which it sold 6,306,271 shares of common stock at a
price to the public of $17.50 per share, for aggregate gross proceeds of $110.4 million. The Company paid underwriting
discounts and commissions of $6.6 million, and also incurred expenses of $0.3 million in connection with the offering. As
a result, the net offering proceeds received by the Company, after deducting underwriting discounts, commissions and
offering expenses, were $103.5 million.
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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 of and with the participation of our management, including our chief executive officer, who
is our principal executive officer, and our chief financial officer, who is our principal financial officer, we conducted an
evaluation of the effectiveness of our disclosure controls and procedures as of December 31, 2020, the end of the period
covered by this Annual Report. The term “disclosure controls and procedures,” as set forth in Rules 13a-15(e) and 15d-
15(e) under the Securities Exchange Act of 1934, as amended, or the Exchange Act, means controls and other procedures
of a company that are designed to provide reasonable assurance that information required to be disclosed by a company in
the reports that it files or submits under the Exchange Act is recorded, processed, summarized and reported, within the time
periods specified in the rules and forms promulgated by the SEC. Disclosure controls and procedures include, without
limitation, controls and procedures designed to ensure that information required to be disclosed by a company in the
reports that it files or submits under the Exchange Act is accumulated and communicated to the company’s management,
including its principal executive and principal financial officers, as appropriate to allow timely decisions regarding required
disclosure. Management recognizes that any controls and procedures, no matter how well designed and operated, can
provide only reasonable assurance of achieving their objectives, and management necessarily applies its judgment in
evaluating the cost-benefit relationship of possible controls and procedures. Based on the evaluation of our disclosure
controls and procedures as of December 31, 2020, our chief executive officer and chief financial officer concluded that, as
of such date, our disclosure controls and procedures were effective at the reasonable assurance level.
Changes in Internal Control over Financial Reporting
There was no change in our internal control over financial reporting identified in connection with the evaluation
required by Rules 13a-15(d) and 15d-15(d) of the Exchange Act that occurred during the quarter ended December 31, 2020
that has materially affected, or is reasonably likely to materially affect, our internal control over financial reporting.
Management’s Report on Internal Control over Financial Reporting and Attestation Report of the Registered
Public Accounting Firm
Our management is responsible for establishing and maintaining an adequate system of internal control over
financial reporting, as defined in Rule 13a-15(f) of the Exchange Act. Management conducted an assessment of our
internal control over financial reporting based on the framework established in 2013 by the Committee of Sponsoring
Organizations of the Treadway Commission in Internal Control—Integrated Framework. Based on the assessment,
management concluded that, as of December 31, 2020, our internal control over financial reporting was effective.
This Annual Report does not include an attestation report of our registered public accounting firm regarding
internal control over financial reporting as required by Section 404(b) of the Sarbanes-Oxley Act of 2002. Because we are
a smaller reporting company and a non-accelerated filer under the SEC rules, management's report was not subject to
attestation by our independent registered public accounting firm.
Item 9B. Other Information
Not applicable.
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PART III
We will file a definitive Proxy Statement for our 2021 Annual Meeting of Stockholders, or the 2021 Proxy
Statement, with the SEC, pursuant to Regulation 14A, not later than 120 days after the end of our fiscal year. Accordingly,
certain information required by Part III has been omitted under General Instruction G(3) to Form 10-K. Only those sections
of the 2021 Proxy Statement that specifically address the items set forth herein are incorporated by reference.
Item 10. Directors, Executive Officers and Corporate Governance
The information required by Item 10 is hereby incorporated by reference to the sections of the 2021 Proxy
Statement under the captions “Information Regarding the Board of Directors and Corporate Governance,” “Election of
Directors” and “Information about our Executive Officers.”
Item 11. Executive Compensation
The information required by Item 11 is hereby incorporated by reference to the sections of the 2021 Proxy
Statement under the captions “Executive Compensation” and “Non-Employee Director Compensation.”
Item 12. Security Ownership of Certain Beneficial Owners and Management and Related Stockholder Matters
The information required by Item 12 is hereby incorporated by reference to the sections of the 2021 Proxy
Statement under the captions “Security Ownership of Certain Beneficial Owners and Management” and “Securities
Authorized for Issuance under Equity Compensation Plans.”
Item 13. Certain Relationships and Related Transactions, and Director Independence
The information required by Item 13 is hereby incorporated by reference to the sections of the 2021 Proxy
Statement under the captions “Transactions with Related Persons” and “Independence of the Board of Directors.”
Item 14. Principal Accountant Fees and Services
The information required by Item 14 is hereby incorporated by reference to the sections of the 2021 Proxy
Statement under the caption “Ratification of Selection of Independent Registered Public Accounting Firm.”
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PART IV
Item 15. Exhibits, Financial Statement Schedules
(a) The following documents are filed as part of this report:
(1) Financial Statements
Our consolidated financial statements are listed in the “Index to Consolidated Financial Statements”
under Part II. Item 8 of this Annual Report on Form 10-K.
(2) Financial Statement Schedules
Financial statement schedules have been omitted in this report because they are not applicable, not
required under the instructions, or the information required is set forth in the consolidated financial
statements or related notes thereto.
(3) Exhibits
See exhibits listed under part (b) below.
(b) Exhibits
Exhibit
Number
Description of Document
2.1# Agreement and Plan of Merger, dated as of August 3, 2017, by and among the Registrant, Aclaris Life
Sciences, Inc., Confluence Life Sciences, Inc. and Fortis Advisors LLC (incorporated by reference to Exhibit
2.1 to the Registrant’s Quarterly Report on Form 10-Q (File No. 001-37581), filed with the SEC on
November 7, 2017).
2.2˄& Asset Purchase Agreement, by and between the Registrant and EPI Health, LLC, dated as of October 10,
2019 (incorporated by reference to Exhibit 2.1 to the Registrant’s Current Report on Form 8-K (File No. 001-
37581), filed with the SEC on October 11, 2019).
3.1 Amended and Restated Certificate of Incorporation of the Registrant (incorporated by reference to
Exhibit 3.1 to the Registrant’s Current Report on Form 8-K (File No. 001-37581), filed with the SEC on
October 13, 2015).
3.2 Amended and Restated Bylaws of the Registrant (incorporated by reference to Exhibit 3.1 to the Registrant’s
4.1
Current Report on Form 8-K (File No. 001-37581), filed with the SEC on June 24, 2020).
Specimen stock certificate evidencing shares of Common Stock (incorporated by reference to Exhibit 4.1 to
Amendment No. 2 to the Registrant’s Registration Statement on Form S-1 (File No. 333-206437), filed with
the SEC on September 25, 2015).
4.2* Description of Securities.
10.1+ Amended and Restated 2012 Equity Compensation Plan (incorporated by reference to Exhibit 10.7 to
10.2+
10.3+
10.4+
10.5+
Amendment No. 1 to the Registrant’s Registration Statement on Form S-1 (File No. 333-206437), filed with
the SEC on September 4, 2015).
Form of Stock Option Grant under Amended and Restated 2012 Equity Compensation Plan (incorporated by
reference to Exhibit 10.8 to the Registrant’s Registration Statement on Form S-1 (File No. 333-206437), filed
with the SEC on August 17, 2015).
2015 Equity Incentive Plan (incorporated by reference to Exhibit 4.6 to the Registrant’s Registration
Statement on Form S-8 (File No. 333-207434), filed with the SEC on October 15, 2015).
Form of Stock Option Grant Notice and Stock Option Agreement under 2015 Equity Incentive Plan
(incorporated by reference to Exhibit 10.10 to Amendment No. 2 to the Registrant’s Registration Statement
on Form S-1 (File No. 333-206437), filed with the SEC on September 25, 2015).
Form of Restricted Stock Unit Grant Notice and Restricted Stock Unit Award Agreement under 2015 Equity
Incentive Plan (incorporated by reference to Exhibit 10.11 to Amendment No. 2 to the Registrant’s
Registration Statement on Form S-1 (File No. 333-206437), filed with the SEC on September 25, 2015).
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10.6+
10.7+
Form of Performance Stock Option Grant Notice and Stock Option Agreement used in connection with the
2015 Equity Incentive Plan (incorporated by reference to Exhibit 10.11 to the Registrant’s Annual Report on
Form 10-K (File No. 001-37581), filed with the SEC on March 18, 2019).
Form of Performance Restricted Stock Unit Grant Notice and Restricted Stock Unit Award Agreement used
in connection with the 2015 Equity Incentive Plan (incorporated by reference to Exhibit 10.12 to the
Registrant’s Annual Report on Form 10-K (File No. 001-37581), filed with the SEC on March 18, 2019).
10.8+ Aclaris Therapeutics, Inc. Inducement Plan (incorporated herein by reference to Exhibit 10.1 to the
10.9+
10.10+
10.11+
Registrant’s Current Report on Form 8-K (File No. 001-37581), filed with the SEC on August 1, 2017).
Form of Stock Option Grant Notice and Stock Option Agreement used in connection with the Aclaris
Therapeutics, Inc. Inducement Plan (incorporated herein by reference to Exhibit 10.2 to the Registrant’s
Current Report on Form 8-K (File No. 001-37581), filed with the SEC on August 1, 2017).
Form of Restricted Stock Unit Grant Notice and Restricted Stock Unit Award Agreement used in connection
with the Aclaris Therapeutics, Inc. Inducement Plan (incorporated herein by reference to Exhibit 10.3 to the
Registrant’s Current Report on Form 8-K (File No. 001-37581), filed with the SEC on August 1, 2017).
Form of Indemnification Agreement (incorporated by reference to Exhibit 10.12 to the Registrant’s
Registration Statement on Form S-1 (File No. 333-206437), filed with the SEC on August 17, 2015).
Fourth Amended and Restated Non-Employee Director Compensation Policy.
10.12+*
10.13+ Amended and Restated Employment Agreement, by and between the Registrant and Neal Walker, dated as of
October 5, 2015 (incorporated by reference to Exhibit 10.1 to the Registrant’s Quarterly Report on Form 10-
Q (File No. 001-37581), filed with the SEC on November 18, 2015).
10.14+ Employment Agreement with Kamil Ali-Jackson, dated as of September 17, 2015 (incorporated by reference
to Exhibit 10.1 to the Registrant’s Quarterly Report on Form 10-Q (File No. 001-37581), filed with the SEC
on May 9, 2017).
10.15+ Employment Agreement with Frank Ruffo, dated as of September 17, 2015 (incorporated by reference to
Exhibit 10.20 to the Registrant’s Annual Report on Form 10-K (File No. 001-37581), filed with the SEC on
February 25, 2020).
10.16+* Change in Control Severance Benefit Plan, effective January 1, 2017, as amended by First Amendment to
10.17
10.18
10.19
Change in Control Severance Benefit Plan, effective October 2, 2019.
Sublease, dated November 2, 2017, by and between the Registrant and Auxilium Pharmaceuticals, LLC
(incorporated by reference to Exhibit 10.1 to the Registrant’s Current Report on Form 8-K (File No. 001-
37581), filed with the SEC on November 2, 2017).
First Amendment to Sublease, dated as of December 13, 2017, by and between the Registrant and Auxilium
Pharmaceuticals, LLC (incorporated by reference to Exhibit 10.27 to the Registrant’s Annual Report on Form
10-K (File No. 001-37581), filed with the SEC on March 18, 2019).
Second Amendment to Sublease, dated as of April 29, 2020, by and between the Registrant and Auxilium
Pharmaceuticals, LLC (incorporated by reference to Exhibit 10.5 to the Registrant’s Quarterly Report on
Form 10-Q (File No. 001-37581), filed with the SEC on May 7, 2020).
10.20 Loan and Security Agreement, by and among the Registrant, Confluence Discovery Technologies, Inc. and
Silicon Valley Bank, dated as of March 30, 2020 (incorporated by reference to Exhibit 10.1 to the
Registrant’s Current Report on Form 8-K (File No. 001-37581), filed with the SEC on March 31, 2020).
Subsidiaries of the Registrant.
21.1*
23.1* Consent of PricewaterhouseCoopers LLP, independent registered public accounting firm.
24.1*
31.1* Certification of Principal Executive Officer pursuant to Rules 13a-14(a) and 15d-14(a) promulgated under the
Power of Attorney (contained on signature page hereto).
Securities Exchange Act of 1934, as adopted pursuant to Section 302 of the Sarbanes-Oxley Act of 2002.
31.2* Certification of Principal Financial Officer pursuant to Rules 13a-14(a) and 15d-14(a) promulgated under the
Securities Exchange Act of 1934, as adopted pursuant to section 302 of the Sarbanes-Oxley Act of 2002.
32.1 *† Certification of Principal Executive Officer and Principal Financial Officer pursuant to Rules 13a-14(b) and
15d-14(b) promulgated under the Securities Exchange Act of 1934 and 18 U.S.C. Section 1350, as adopted
pursuant to section 906 of The Sarbanes-Oxley Act of 2002.
101.INS XBRL Instance Document (the instance document does not appear in the Interactive Data File because its
XBRL tags are embedded within the Inline XBRL document)
101.SCH Inline XBRL Taxonomy Extension Schema Document
101.CAL Inline XBRL Taxonomy Extension Calculation Linkbase Document
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Inline XBRL Taxonomy Extension Definition Linkbase Document
101.DEF
101.LAB Inline XBRL Taxonomy Extension Label Linkbase Document
101.PRE Inline XBRL Taxonomy Extension Presentation Linkbase Document
104 Cover Page Interactive Data File (formatted as Inline XBRL and contained in Exhibit 101)
*
†
Filed herewith.
This certification is being furnished solely to accompany this Annual Report pursuant to 18 U.S.C. Section 1350, and
are not being filed for purposes of Section 18 of the Securities Exchange Act of 1934, as amended, and is not to be
incorporated by reference into any filing of the Registrant, whether made before or after the date hereof, regardless of
any general incorporation language in such filing.
Indicates management contract or compensatory plan.
+
# Confidential treatment has been granted with respect to portions of this exhibit (indicated by asterisks) and those
˄
portions have been separately filed with the SEC.
Pursuant to Item 601(a)(5) of Regulation S-K promulgated by the SEC, certain exhibits and schedules to this
agreement have been omitted. The Company hereby agrees to furnish supplementally to the SEC, upon its request, any
or all of such omitted exhibits or schedules.
& Pursuant to Item 601(b)(2)(ii) of Regulation S-K promulgated by the SEC, certain portions of this exhibit have been
redacted because such portions, indicated by asterisks, are both not material and would likely cause competitive harm
to the Company if publicly disclosed. The Company hereby agrees to furnish supplementally to the SEC, upon its
request, an unredacted copy of the exhibit.
Item 16. Form 10-K Summary.
Not applicable.
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Pursuant to the requirements of Section 13 or 15(d) of the Securities Exchange Act of 1934, as amended, the
registrant has duly caused this report to be signed on its behalf by the undersigned, thereunto duly authorized.
SIGNATURES
ACLARIS THERAPEUTICS, INC.
By:
/s/ Neal Walker
Neal Walker
President and Chief Executive Officer
Date: February 25, 2021
KNOW ALL PERSONS BY THESE PRESENTS, that each person whose signature appears below constitutes
and appoints Neal Walker, Kamil Ali-Jackson and Frank Ruffo, jointly and severally, as his or her true and lawful
attorneys-in-fact and agents, with full power of substitution and resubstitution, for him or her and in his or her name, place
and stead, in any and all capacities, to sign this Annual Report on Form 10-K of Aclaris Therapeutics, Inc., and any or all
amendments thereto, and to file the same, with all exhibits thereto, and other documents in connection therewith, with the
Securities and Exchange Commission, granting unto said attorneys-in-fact and agents full power and authority to do and
perform each and every act and thing requisite or necessary to be done in and about the premises hereby ratifying and
confirming all that said attorneys-in-fact and agents, or his, her or their substitute or substitutes, may lawfully do or cause
to be done by virtue hereof.
Pursuant to the requirements of the Securities Exchange Act of 1934, as amended, this report has been signed
below by the following persons on behalf of the registrant and in the capacities and on the dates indicated.
Signature
/s/ Neal Walker
Neal Walker
/s/ Frank Ruffo
Frank Ruffo
Title
President, Chief Executive Officer and Director
(Principal Executive Officer)
Chief Financial Officer
(Principal Financial Officer and Principal
Accounting Officer)
Date
February 25, 2021
February 25, 2021
/s/ Christopher Molineaux
Christopher Molineaux
/s/ Anand Mehra, M.D.
Anand Mehra, M.D.
/s/ William Humphries
William Humphries
/s/ Andrew Powell
Andrew Powell
/s/ Andrew Schiff
Andrew Schiff
/s/ Bryan Reasons
Bryan Reasons
/s/ Maxine Gowen
Maxine Gowen
/s/ Vincent Milano
Vincent Milano
Chairman of the Board of Directors
February 25, 2021
Director
Director
Director
Director
Director
Director
Director
119
February 25, 2021
February 25, 2021
February 25, 2021
February 25, 2021
February 25, 2021
February 25, 2021
February 25, 2021
Exhibit 4.2
DESCRIPTION OF ACLARIS THERAPEUTICS, INC. CAPITAL STOCK
The following description of the common stock of Aclaris Therapeutics, Inc., or the Company, is a summary and does
not purport to be complete. This summary is qualified in its entirety by reference to the provisions of the Delaware
General Corporation Law, or the DGCL, and the complete text of the Company’s amended and restated certificate of
incorporation, or the certificate of incorporation, and amended and restated bylaws or the bylaws, which are
incorporated by reference as Exhibits 3.1 and 3.2, respectively of the Company’s Annual Report on Form 10-K to
which this description is also an exhibit. The Company encourages you to read that law and those documents carefully.
Common Stock
Under the certificate of incorporation, the Company authorized to issue up to 100,000,000 shares of common
stock, $0.00001 par value per share, and 10,000,000 shares of preferred stock, $0.00001 par value per share, all of
which shares of preferred stock are undesignated. The Company’s board of directors may establish the rights and
preferences of the preferred stock from time to time.
Voting Rights
Each holder of common stock is entitled to one vote for each share on all matters submitted to a vote of the
stockholders, including the election of directors. Under the certificate of incorporation and the bylaws, common
stockholders do not have cumulative voting rights. Because of this, the holders of a majority of the shares of common
stock entitled to vote in any election of directors can elect all of the directors standing for election, if they should so
choose.
Dividends
Subject to preferences that may be applicable to any then-outstanding preferred stock, holders of common stock
are entitled to receive ratably those dividends, if any, as may be declared from time to time by the board of directors
out of legally available funds.
Liquidation
In the event of the Company’s liquidation, dissolution or winding up, holders of common stock will be entitled to
share ratably in the net assets legally available for distribution to stockholders after the payment of all of debts and
other liabilities and the satisfaction of any liquidation preference granted to the holders of any then-outstanding shares
of preferred stock.
Rights and Preferences
Holders of common stock have no preemptive, conversion or subscription rights and there are no redemption or
sinking fund provisions applicable to the common stock. The rights, preferences and privileges of the holders of
common stock are subject to, and may be adversely affected by, the rights of the holders of shares of any series of
preferred stock that the Company may designate in the future.
Anti-Takeover Provisions
Section 203 of the DGCL
The Company is subject to Section 203 of the DGCL, which prohibits a Delaware corporation from engaging in
any business combination with any interested stockholder for a period of three years after the date that such
stockholder became an interested stockholder, with the following exceptions:
● before such date, the board of directors of the corporation approved either the business combination or the
transaction that resulted in the stockholder becoming an interested stockholder;
● upon completion of the transaction that resulted in the stockholder becoming an interested stockholder, the
interested stockholder owned at least 85% of the voting stock of the corporation outstanding at the time the
transaction began, excluding for purposes of determining the voting stock outstanding, but not the outstanding
voting stock owned by the interested stockholder, those shares owned (i) by persons who are directors and
also officers and (ii) employee stock plans in which employee participants do not have the right to determine
confidentially whether shares held subject to the plan will be tendered in a tender or exchange offer; or
● on or after such date, the business combination is approved by the board of directors and authorized at an
annual or special meeting of the stockholders, and not by written consent, by the affirmative vote of at least
66 2/3% of the outstanding voting stock that is not owned by the interested stockholder.
In general, Section 203 defines a “business combination” to include the following:
● any merger or consolidation involving the corporation and the interested stockholder;
● any sale, transfer, pledge or other disposition of 10% or more of the assets of the corporation involving the
interested stockholder;
● subject to certain exceptions, any transaction that results in the issuance or transfer by the corporation of any
stock of the corporation to the interested stockholder;
● any transaction involving the corporation that has the effect of increasing the proportionate share of the stock
or any class or series of the corporation beneficially owned by the interested stockholder; or
● the receipt by the interested stockholder of the benefit of any loans, advances, guarantees, pledges or other
financial benefits by or through the corporation.
In general, Section 203 defines an “interested stockholder” as an entity or person who, together with the person’s
affiliates and associates, beneficially owns, or within three years prior to the time of determination of interested
stockholder status did own, 15% or more of the outstanding voting stock of the corporation.
Certificate of Incorporation and Bylaws
The certificate of incorporation provides for the Company’s board of directors to be divided into three classes with
staggered three-year terms. Only one class of directors will be elected at each annual meeting of stockholders, with the
other classes continuing for the remainder of their respective three-year terms. Because the Company’s stockholders do
not have cumulative voting rights, stockholders holding a majority of the shares of common stock outstanding will be
able to elect all of the Company’s directors. The certificate of incorporation and bylaws also provide that directors may
be removed by the stockholders only for cause upon the vote of 66 2/3% or more of outstanding common stock.
Furthermore, the authorized number of directors may be changed only by resolution of the board of directors, and
vacancies and newly created directorships on the board of directors may, except as otherwise required by law or
determined by the board, only be filled by a majority vote of the directors then serving on the board, even though less
than a quorum.
The certificate of incorporation and bylaws also provide that all stockholder actions must be effected at a duly
called meeting of stockholders and will eliminate the right of stockholders to act by written consent without a meeting.
The bylaws also provide that only the Company’s chairman of the board, chief executive officer or the board of
directors pursuant to a resolution adopted by a majority of the total number of authorized directors may call a special
meeting of stockholders.
The bylaws also provide that stockholders seeking to present proposals before a meeting of stockholders or to
nominate candidates for election as directors at a meeting of stockholders must provide timely advance notice in
writing, and specify requirements as to the form and content of a stockholder’s notice.
The certificate of incorporation and bylaws provide that the stockholders cannot amend many of the provisions
described above except by a vote of 66 2/3% or more of outstanding common stock.
The combination of these provisions make it more difficult for the Company’s existing stockholders to replace the
board of directors as well as for another party to obtain control of the Company by replacing its board of directors.
Since the Company’s board of directors has the power to retain and discharge the Company’s officers, these provisions
also make it more difficult for existing stockholders or another party to effect a change in management. In addition, the
authorization of undesignated preferred stock makes it possible for the Company’s board of directors to issue preferred
stock with voting or other rights or preferences that could impede the success of any attempt to change the Company’s
control.
These provisions are intended to enhance the likelihood of continued stability in the composition of the
Company’s board of directors and its policies and to discourage coercive takeover practices and inadequate takeover
bids. These provisions are also designed to reduce the Company’s vulnerability to hostile takeovers and to discourage
certain tactics that may be used in proxy fights. However, such provisions could have the effect of discouraging others
from making tender offers for the Company’s shares and may have the effect of delaying changes in its control or
management. As a consequence, these provisions may also inhibit fluctuations in the market price of the Company’s
stock that could result from actual or rumored takeover attempts. The Company believes that the benefits of these
provisions, including increased protection of its potential ability to negotiate with the proponent of an unfriendly or
unsolicited proposal to acquire or restructure the company, outweigh the disadvantages of discouraging takeover
proposals, because negotiation of takeover proposals could result in an improvement of their terms.
Choice of Forum
The Company’s certificate of incorporation provides that the Court of Chancery of the State of Delaware is the
exclusive forum for the following types of actions or proceedings under Delaware statutory or common law: (i) any
derivative action or proceeding brought on the Company’s behalf, (ii) any action asserting a claim for breach of a
fiduciary duty owed by any of the Company’s directors, officers or other employees to the Company or its
stockholders, (iii) any action asserting a claim arising pursuant to any provision of the DGCL, the Company’s
certificate of incorporation or bylaws or (iv) any action asserting a claim governed by the internal affairs
doctrine. However, this exclusive forum provision would not apply to suits brought to enforce a duty or liability
created by the Securities Act of 1933, as amended, or the Securities Act, or the Securities Exchange Act of 1934, as
amended.
In addition, the Company’s bylaws provide that unless the Company consents in writing to the selection of an
alternative forum, the federal district courts of the United States of America shall, to the fullest extent permitted by
law, be the sole and exclusive forum for the resolution of any complaint asserting a cause of action arising under the
Securities Act, and that any person or entity purchasing or otherwise acquiring or holding any interest in shares of
capital stock of the Company shall be deemed to have notice of and consented to the federal forum selection provision.
Transfer Agent and Registrar
The transfer agent and registrar for the Company’s common stock is Broadridge Corporate Issuer Solutions, Inc.
The transfer agent’s address is 1717 Arch Street, Suite 1300, Philadelphia, Pennsylvania 19103.
Listing on the NASDAQ Global Select Market
The Company’s common stock is listed on the Nasdaq Global Select Market under the symbol “ACRS.”
Exhibit 10.12
ACLARIS THERAPEUTICS, INC.
FOURTH AMENDED & RESTATED
NON-EMPLOYEE DIRECTOR COMPENSATION POLICY
Each member of the Board of Directors (the “Board”) who is not also serving as an employee of Aclaris
Therapeutics, Inc. (the “Company”) (each such member, an “Eligible Director”) will receive the compensation
described in this Fourth Amended & Restated Non-Employee Director Compensation Policy (this “Policy”) for his or
her Board service effective as of January 1, 2021 (the “Effective Date”). An Eligible Director may decline all or any
portion of his or her compensation by giving notice to the Company prior to the date cash is to be paid or equity
awards are to be granted, as the case may be. This Policy may be amended at any time in the sole discretion of the
Board or the Compensation Committee of the Board. The terms and conditions of this Policy shall supersede any prior
Non-Employee Director Compensation Policy of the Company.
Annual Cash Compensation
The annual cash compensation amount set forth below is payable in equal quarterly installments, payable in arrears on
the last day of each fiscal quarter in which the service occurred. If an Eligible Director joins the Board or a committee
of the Board at a time other than effective as of the first day of a fiscal quarter, each annual retainer set forth below will
be pro-rated based on days served in the applicable fiscal year, with the pro-rated amount paid for the first fiscal
quarter in which the Eligible Director provides the service, and regular full quarterly payments thereafter. All annual
cash fees are vested upon payment.
1.
2.
Annual Board Service Retainer:
a.
All Eligible Directors: $40,000
Annual Committee Member Service Retainer:
a.
b.
c.
Member of the Audit Committee: $7,500
Member of the Compensation Committee: $6,000
Member of the Nominating and Corporate Governance Committee: $4,500
3.
Annual Committee Chair Service Retainer (in addition to Committee Member Service Retainer):
a.
b.
c.
Chairman of the Audit Committee: $12,500
Chairman of the Compensation Committee: $8,000
Chairman of the Nominating and Corporate Governance Committee: $4,500
4.
Annual Chairman of the Board Service Retainer (in addition to Board Service Retainer): $30,000
Equity Compensation
The equity compensation set forth below will be granted under the Company’s 2015 Equity Incentive Plan (the
“Plan”). All stock options granted under this Policy will be nonstatutory stock options, with an exercise price per
share equal to 100% of the Fair Market Value (as defined in the Plan) of the Company’s underlying common stock (the
“Common Stock”) on the date of grant, and a term of ten years from the date of grant (subject to earlier termination in
connection with a termination of service as provided in the Plan).
1.
Initial Grant: On the date of the Eligible Director’s initial election to the Board, for each Eligible Director
who is first elected to the Board following the Effective Date (or, if such date is not a market trading day, the first
market trading day thereafter), the Eligible Director will be automatically, and without further action by the Board or
Compensation Committee of the Board, granted a stock option to purchase 44,600 shares of the Company’s Common
Stock, with an exercise price per share equal to 100% of the Fair Market Value of the Company’s Common Stock on
the date of grant. The shares subject to each such stock option will vest in equal monthly
installments for 36 months, subject to the Eligible Director’s Continuous Service (as defined in the Plan) through such
vesting date[s].
Annual Grant: On the date of each annual stockholders meeting of the Company held on and after the
2.
Effective Date, each Eligible Director who continues to serve as a non-employee member of the Board following such
stockholders meeting will be automatically, and without further action by the Board or Compensation Committee of
the Board, granted (a) a stock option to purchase 22,300 shares of the Company’s Common Stock, with an exercise
price per share equal to 100% of the Fair Market Value of the Company’s Common Stock on the date of grant or (b) if
approved by the Board or the Compensation Committee of the Board prior to any such meeting, a number of restricted
stock units at a ratio to the number of shares such Eligible Director would have received under clause (a) as
determined by the Board or the Compensation Committee (or any combination of clause (a) and this clause (b)). The
shares subject to each such stock option will vest in equal monthly installments for 12 months and the restricted stock
units will vest in one installment on the first anniversary of the grant date, subject to the Eligible Director’s Continuous
Service through such vesting date[s].
ACLARIS THERAPEUTICS, INC.
CHANGE IN CONTROL SEVERANCE BENEFIT PLAN
Exhibit 10.16
Section 1.
INTRODUCTION.
The Aclaris Therapeutics, Inc. Change in Control Severance Benefit Plan (the “Plan”) is hereby established
effective January 1, 2017 (the “Effective Date”). The purpose of the Plan is to provide for the payment of severance benefits
to selected employees of Aclaris Therapeutics, Inc. (the “Company”) that constitute a select group of management or highly
compensated employees in the event that such employees become subject to involuntary employment terminations in
connection with an acquisition of the Company. This Plan document also is the Summary Plan Description for the Plan.
For purposes of the Plan, the following terms are defined as follows:
(a)
“Affiliate” means any “parent” or “subsidiary” of the Company as such terms are defined in Rule
405 of the Securities Act of 1933, as amended. The Plan Administrator shall have the authority to determine the time or
times at which “parent” or “subsidiary” status is determined within the foregoing definition. For purposes of this Agreement,
NeXeption, LLC, NeXeption, Inc., NST, LLC and NST Consulting, LLC shall not be deemed to be Affiliates of the
Company.
(b)
“Annual Base Salary” means the annualized base pay amount (excluding incentive pay, premium
pay, commissions, overtime, bonuses and other forms of variable compensation) as in effect immediately prior to a Covered
Termination.
(c)
“Board” means the Board of Directors of the Company; provided, however, that if the Board has
delegated authority to administer the Plan to the Compensation Committee of the Board, then “Board” shall also mean the
Compensation Committee.
(d)
“Cause” means cause or misconduct as defined in the Individual Severance Arrangement as in
effect on the Eligible Employee’s Termination Date, or in the absence of any such applicable definition, any of the following
with respect to the employee: (i) the Eligible Employee’s conviction of, or guilty plea to, a crime of moral turpitude (whether
or not a felony) or a felony (other than traffic violations); (ii) any act(s) or omission(s) by the Eligible Employee which
constitutes gross negligence or a material breach of the Eligible Employee’s duty of loyalty; (iii) any material breach by the
Eligible Employee of the Company’s personnel policies, including but not limited to those prohibiting acts of discrimination,
harassment or retaliation; (iv) any act constituting dishonesty, fraud, immoral or disreputable conduct; (v) refusal to follow
or implement a clear and reasonable directive of the Company; (vi) breach of fiduciary duty; or (vii) a material breach by the
Eligible Employee of the Eligible Employee’s Employment Agreement or any other agreement between the parties. The
determination whether a termination is for Cause shall be made by the Plan Administrator in its sole and exclusive judgment
and discretion.
(e)
“Change in Control” means, in each case as approved by the Board and the requisite stockholders
of the Company, (i) any consolidation or merger of the Company with or into any other corporation or other Entity or
person, or any other corporate reorganization, in which the stockholders of the Company immediately prior to such
consolidation, merger or reorganization, own, in the aggregate, less than 50% of the surviving Entity’s voting power and/or
outstanding capital stock
1.
immediately after such consolidation, merger or reorganization, or any transaction or series of related transactions (including
any transaction which results from an option agreement or binding letter of intent with a third party) to which the Company
or any of its stockholders is a party in which in excess of 50% of the Company’s voting power and/or outstanding capital
stock is transferred, or pursuant to which any person or group of affiliated persons obtains in excess of 50% of the
Company’s voting power and/or outstanding capital stock, excluding any consolidation or merger effected exclusively to
change the domicile of the Company; or (ii) any sale, lease or other disposition (including through a Board and stockholder
approved division or spin-off transaction) of all or substantially all of the assets of the Company and/or any of its
subsidiaries or any sale, lease, exclusive license (or substantially exclusive license or agreement) or other disposition of all
or substantially all of the Company’s intellectual property, as reasonably determined based upon the potential earning power
of the assets or intellectual property; provided, however that none of the following shall constitute a Change in Control: (A)
transfers of capital stock by an existing stockholder as a result of death or otherwise for estate planning purposes or to such
stockholder’s affiliates or to any of Employer’s other existing stockholders, and (B) issuances of equity securities of the
Company in connection with financings for working capital and other general corporate purposes.
(f)
“Closing” means the initial closing of the Change in Control as defined in the definitive agreement
executed in connection with the Change in Control. In the case of a series of transactions constituting a Change in Control,
“Closing” means the first closing that satisfies the threshold of the definition for a Change in Control.
(g)
(h)
“COBRA” means the Consolidated Omnibus Budget Reconciliation Act of 1985.
“Code” means the Internal Revenue Code of 1986, as amended.
(i)
Entity resulting from such event.
“Company” means Aclaris Therapeutics, Inc. or, following a Change in Control, the surviving
(j)
“Covered Period” means the period commencing sixty (60) days prior to the Closing of a Change in
Control and ending twelve (12) months following the Closing of a Change in Control.
(k)
“Covered Termination” means an employee’s termination from all positions he or she then holds
with the Company, which termination constitutes a “separation from service” (as defined under Treasury Regulation Section
1.409A-1(h) without regard to any alternative definition thereunder), and (i) which is due to a termination by the Company
without Cause and other than as a result of death or disability and (ii) which occurs within the Covered Period.
(l)
“Director” means a member of the Board.
(m)
“Eligible Employee” means an employee of the Company that meets all the requirements to be
eligible to receive Plan benefits as set forth in Section 2, including timely provision of an effective Release (as such term is
defined in Section 2(b)).
(n)
“Employment Agreement” means any individual employment offer letter, contract or agreement
that an Eligible Employee has with the Company.
2.
(o)
“Entity” means a corporation, partnership, limited liability company or other entity.
(p)
“Individual Severance Arrangement” means any Employment Agreement providing for severance
benefits to an Eligible Employee or any other severance arrangement between the Eligible Employee and the Company other
than the Plan, in each case that remains in effect through the date of a Covered Termination.
(q)
“Plan Administrator” means the Board prior to the Closing and the Representative upon and
following the Closing.
(r)
“Qualified Plan” means a plan sponsored by the Company or an Affiliate that is intended to be
qualified under Section 401(a) of the Internal Revenue Code.
(s)
“Representative” means one or more members of the Board or other persons or Entities designated
by the Board prior to or in connection with a Change in Control that will have authority to administer and interpret the Plan
upon and following the Closing as provided in Section 8(a).
(t)
“Target Bonus” means, with respect to an Eligible Employee, the Eligible Employee’s target annual
bonus under the Company’s annual cash bonus plan or policy, or if the Company does not maintain such a plan or policy, the
target annual bonus under the terms of the Eligible Employee’s Employment Agreement or other agreement with the
Company, in each case applicable to such Eligible Employee for the calendar year in which the Covered Termination of such
Eligible Employee occurs. If no cash bonus plan or policy or such an agreement is in effect for the employee for the year in
which the Covered Termination occurs, the Target Bonus for such employee will be $0.
(u)
“Termination Date” means the effective date of an Eligible Employee’s Covered Termination.
Section 2.
ELIGIBILITY FOR BENEFITS.
(a)
Eligible Employee. An employee of the Company is eligible to participate in the Plan if (i) the
employee is a Vice President or higher level officer on any date within the Covered Period and/or the sum of the employee’s
Annual Base Salary and expected Target Bonus equals or exceeds $250,000; (ii) the Board has designated such employee as
eligible to participate in the Plan; (iii) such employee’s employment with the Company terminates due to a Covered
Termination; and (iii) such employee meets the other Plan eligibility requirements set forth in this Section 2 and the Plan.
The determination of whether an employee is an Eligible Employee shall be made by the Plan Administrator, in its sole
discretion, and such determination shall be binding and conclusive on all persons. For the avoidance of doubt, the
Company’s President and Chief Executive Officer, Chief Operating Officer, Chief Scientific Officer, Chief Legal Officer and
Chief Financial Officer shall each not be considered to be an Eligible Employee for purposes of the Plan.
(b)
Release Requirement. In order to be eligible to receive benefits under the Plan, the employee also
must execute a general waiver and release in substantially the form attached hereto as Exhibit A, Exhibit B or Exhibit C, as
appropriate (the “Release”), within the applicable time period set forth therein, but in no event more than fifty (50) days
following the Termination Date of the applicable Covered Termination, and such Release must become effective in
accordance with its terms. The
3.
Company, in its sole discretion, may modify the form of the Release to comply with applicable law. The Release may be
incorporated into a termination agreement or other agreement with the employee.
(c)
No Duplicative Benefits Provided Under Plan. This Plan does not supersede the terms of any
Individual Severance Arrangement. Unless otherwise determined by the Plan Administrator in its discretion, if an employee
is an Eligible Employee and otherwise eligible to receive severance benefits under this Plan that are of the same category
and would otherwise duplicate the benefits available under the terms of any Individual Severance Arrangement
(“Duplicative Benefits”) such Eligible Employee will receive severance benefits under the Individual Severance
Arrangement in lieu of any Plan benefits to the extent such benefits are Duplicative Benefits, and severance benefits will be
provided under the Plan only to the extent, if any, that Plan benefits are not Duplicative Benefits.
(d)
Exceptions to Benefit Entitlement. An employee who otherwise is an Eligible Employee will not
receive benefits under the Plan in the following circumstances, as determined by the Plan Administrator in its sole
discretion:
(1)
The employee is terminated by the Company for any reason (including due to the
employee’s death or disability) or voluntarily terminates employment with the Company in any manner, and in either case,
such termination does not constitute a Covered Termination. Voluntary terminations include, but are not limited to,
resignation, retirement or failure to return from a leave of absence on the scheduled date.
employment with another entity that is wholly or partly owned (directly or indirectly) by the Company or an Affiliate.
(2)
The employee voluntarily terminates employment with the Company in order to accept
The employee is offered an identical or substantially equivalent or comparable position with
the Company or an Affiliate. For purposes of the foregoing, a “substantially equivalent or comparable position” is one that
provides the employee substantially the same level of responsibility and compensation.
(3)
(4)
The employee is offered immediate reemployment by a successor to the Company or an
Affiliate or by a purchaser of the Company’s assets, as the case may be, following a Change in Control in a “substantially
equivalent or comparable position” as defined in Section 2(d)(3). For purposes of the foregoing, “immediate reemployment”
means that the employee’s employment with the successor to the Company or an Affiliate or the purchaser of its assets, as
the case may be, results in uninterrupted employment such that the employee does not incur a lapse in pay or benefits as a
result of the change in ownership of the Company or the sale of its assets. For the avoidance of doubt, an employee who
becomes immediately reemployed as described in this Section 2(d)(4) by a successor to the Company or an Affiliate or by a
purchaser of the Company’s assets, as the case may be, following a Change in Control shall continue to be an Eligible
Employee following the date of such reemployment.
prior to the date benefits under the Plan are scheduled to commence.
(5)
The employee is rehired by the Company or an Affiliate and recommences employment
Section 3.
AMOUNT OF BENEFIT.
(a)
Severance Benefit. Benefits under the Plan shall be provided to an Eligible Employee as follows,
subject to any delay in payment that may be required by Section 5:
4.
(1)
Severance Pay. The Eligible Employee will be entitled to receive a single lump sum cash
payment equal to (A) 50% of the Eligible Employee’s Annual Base Salary, plus (B) an amount equal to the Eligible
Employee’s Target Bonus, pro-rated based on the number of days actually served in the calendar year during which the
Termination Date occurs (the sum of (A) and (B), the “Severance Pay”). The Severance Pay will be payable to the Eligible
Employee within ten (10) business days following the later of (i) the effective date of the Release, or (ii) the effective date of
the Closing.
(2)
Accelerated Vesting of Stock Awards. Effective as of the later of the effective date of the
Release or the effective date of the Closing, to the extent not previously vested: (A) the vesting and exercisability of all
outstanding stock options to purchase the Company’s common stock that are held by the Eligible Employee on such date
shall be accelerated in full, (B) any reacquisition or repurchase rights held by the Company in respect of common stock
issued pursuant to any other stock award granted to the Eligible Employee by the Company shall lapse in full, and (C) the
vesting of any other stock awards granted to the Eligible Employee by the Company, and any issuance of shares triggered by
the vesting of such stock awards, shall be accelerated in full; provided, however, that the foregoing provisions shall not
apply to stock awards issued under or held in any Qualified Plan. For purposes of determining the number of shares that will
vest pursuant to the foregoing provision with respect to any performance based vesting award that has multiple vesting
levels depending upon the level of performance, vesting acceleration shall occur with respect to the number of shares subject
to the award as if the applicable performance criteria had been attained at a 100% level. In order to give effect to the intent
of the foregoing provision, notwithstanding anything to the contrary set forth in the Eligible Employee’s stock award
agreements or the applicable equity incentive plan under which such stock award was granted that provides that any then
unvested portion of the award will immediately expire upon the Eligible Employee’s termination of service, the stock awards
referred to in this Section 3(a)(2) shall remain outstanding after an Eligible Employee’s Covered Termination to the extent
necessary to give effect to the potential vesting acceleration in this Section 3(a)(2) (e.g., if an Eligible Employee’s Covered
Termination occurs prior to the Closing). Notwithstanding anything to the contrary set forth herein, the Eligible Employee’s
stock awards shall remain subject to the terms of the applicable Company plan and award documents under which such
stock award was granted, including any provision for earlier termination of such stock awards upon a “corporate
transaction,” “change in control” or similar event under the terms of the applicable Company equity incentive plan or
substantially equivalent provisions applicable to such stock option or stock award.
(3)
Payment of Continued Group Health Plan Benefits.
(i)
Provided that the Eligible Employee is a participant in the Company’s group health
plans as of immediately prior to the Covered Termination, if the Eligible Employee timely elects continued group health plan
continuation coverage under COBRA with respect to such group health plans, the Company shall directly pay to the
Company’s group health care provider an amount equal to the COBRA premiums, less the amount the Eligible Employee
would have had to pay to receive group health coverage for the Eligible Employee and the Eligible Employee’s covered
dependents based on the cost sharing levels in effect on the Eligible Employee’s Termination Date (which for the avoidance
of doubt the Eligible Employee shall be required to pay directly to the Company’s group health care provider together with
any administrative or additional costs due with respect to such COBRA coverage), for the Eligible Employee and the
Eligible Employee’s covered dependents under such plans during the period commencing on the Termination Date and
ending upon the last day of the six (6) month period following the Covered Termination (the “COBRA Payment Period”).
Upon the conclusion of such period of insurance premium payments made by the Company, the Eligible Employee will be
responsible for the entire payment of premiums (or payment for the cost of coverage) required under
5.
COBRA for the duration of the Eligible Employee’s eligible COBRA coverage period. For purposes of this Section, (A)
references to COBRA shall be deemed to refer also to analogous provisions of state law and (B) any applicable insurance
premiums that are paid by the Company shall not include any amounts payable by the Eligible Employee under an Internal
Revenue Code Section 125 health care reimbursement plan, which amounts, if any, are the Eligible Employee’s sole
responsibility.
(ii)
Notwithstanding the foregoing, if at any time the Company determines, in its sole
discretion, that it cannot provide the COBRA premium benefits without potentially incurring financial costs or penalties
under applicable law (including, without limitation, Section 2716 of the Public Health Service Act), then in lieu of paying
COBRA premiums on the Eligible Employee’s behalf, the Company will instead pay the Eligible Employee on the last day
of each remaining month of the COBRA Payment Period a fully taxable cash payment equal to the COBRA premium for
that month, subject to applicable tax withholding (such amount, the “Special Severance Payment”), such Special Severance
Payment to be made without regard to the Eligible Employee’s election of COBRA coverage or payment of COBRA
premiums and without regard to the Eligible Employee’s continued eligibility for COBRA coverage during the COBRA
Payment Period. Such Special Severance Payment shall end upon expiration of the COBRA Payment Period.
(b)
Additional Benefits. Notwithstanding the foregoing, the Company may, in its sole discretion,
provide benefits to employees who are not Eligible Employees (“Non-Eligible Employees”) chosen by the Board, in its sole
discretion, and the provision of any such benefits to a Non-Eligible Employee shall in no way obligate the Company to
provide such benefits to any other Non-Eligible Employee, even if similarly situated. If benefits under the Plan are provided
to a Non-Eligible Employee, references in the Plan to “Eligible Employee” (and similar references) shall be deemed to refer
to such Non-Eligible Employee.
(c)
Certain Reductions. The Company, in its sole discretion, shall have the authority to reduce an
Eligible Employee’s severance benefits, in whole or in part, by pay and benefits provided during a period following written
notice of a plant closing or mass layoff, pay and benefits in lieu of such notice, or other similar benefits payable to the
Eligible Employee by the Company or an Affiliate that become payable in connection with the Eligible Employee’s
termination of employment pursuant to (i) any applicable legal requirement, including, without limitation, the Worker
Adjustment and Retraining Notification Act or any other similar state law, (ii) any Company policy or practice providing for
the Eligible Employee to remain on the payroll for a limited period of time after being given notice of the termination of the
Eligible Employee’s employment, or (iii) any other severance benefit agreement or arrangement between the Company and
the Eligible Employee, and the Plan Administrator shall so construe and implement the terms of the Plan, in each case to the
extent compliant with Section 409A of the Code. Any such reductions that the Company determines to make pursuant to
this Section 3(c) shall be made such that any benefit under the Plan shall be reduced solely by any similar type of benefit
under such legal requirement, agreement, policy or practice (i.e., any cash severance benefits under the Plan shall be reduced
solely by any cash payments or severance benefits under such legal requirement, agreement, policy or practice, and any
continued insurance benefits under the Plan shall be reduced solely by any continued insurance benefits under such legal
requirement, agreement, policy or practice). The Company’s decision to apply such reductions to the severance benefits of
one Eligible Employee and the amount of such reductions shall in no way obligate the Company to apply the same
reductions in the same amounts to the severance benefits of any other Eligible Employee, even if similarly situated. In the
Company’s sole discretion, such reductions may be applied on a retroactive basis, with severance benefits previously paid
being re-characterized as payments pursuant to the Company’s statutory obligation.
6.
(d)
Parachute Payments. The following provisions shall not supersede any provisions to the contrary
provided under any Individual Severance Arrangement, if applicable:
(1)
Any provision of the Plan to the contrary notwithstanding, if any payment or benefit an
Eligible Employee would receive from the Company pursuant to the Plan or otherwise (“Payment”) would (i) constitute a
“parachute payment” within the meaning of Section 280G of the Code, and (ii) but for this sentence, be subject to the excise
tax imposed by Section 4999 of the Code (the “Excise Tax”), then such Payment will be equal to the Reduced Amount
(defined below). The “Reduced Amount” shall be either (x) the largest portion of the Payment that would result in no
portion of the Payment being subject to the Excise Tax or (y) the largest portion, up to and including the total, of the
Payment, whichever amount, after taking into account all applicable federal, state and local employment taxes, income taxes,
and the Excise Tax (all computed at the highest applicable marginal rate), results in the Eligible Employee’s receipt, on an
after-tax basis, of the greater economic benefit notwithstanding that all or some portion of the Payment may be subject to the
Excise Tax. If a reduction in payments or benefits constituting “parachute payments” is necessary so that the Payment equals
the Reduced Amount, reduction shall occur in the manner that results in the greatest economic benefit for the Eligible
Employee. If more than one method of reduction will result in the same economic benefit, the items so reduced will be
reduced pro rata.
(2)
In the event it is subsequently determined by the Internal Revenue Service that some portion
of the Reduced Amount as determined pursuant to clause (x) in the preceding paragraph is subject to the Excise Tax, the
Eligible Employee agrees to promptly return to the Company a sufficient amount of the Payment so that no portion of the
Reduced Amount is subject to the Excise Tax. For the avoidance of doubt, if the Reduced Amount is determined pursuant to
clause (y) in the preceding paragraph, the Eligible Employee will have no obligation to return any portion of the Payment
pursuant to the preceding sentence.
(3)
Unless the Eligible Employee and the Company agree on an alternative accounting firm or
law firm, the accounting firm engaged by the Company for general tax compliance purposes as of the day prior to the
effective date of the Change in Control shall perform the foregoing calculations. If the accounting firm so engaged by the
Company is serving as accountant or auditor for the individual, entity or group effecting the Change in Control, the
Company shall appoint a nationally recognized accounting or law firm to make the determinations required hereunder. The
Company shall bear all expenses with respect to the determinations by such accounting or law firm required to be made
hereunder.
Section 4.
RETURN OF COMPANY PROPERTY.
An Eligible Employee will not be entitled to any severance benefit under the Plan unless and until the
Eligible Employee returns all Company Property. For this purpose, “Company Property” means all Company documents
(and all copies thereof) and other Company property which the Eligible Employee had in his or her possession at any time,
including, but not limited to, Company files, notes, drawings, records, plans, forecasts, reports, studies, analyses, proposals,
agreements, financial information, research and development information, sales and marketing information, operational and
personnel information, specifications, code, software, databases, computer-recorded information, tangible property and
equipment (including, but not limited to, computers, facsimile machines, mobile telephones, servers), credit cards, entry
cards, identification badges and keys; and any materials of any kind which contain or embody any proprietary or confidential
information of the Company (and all reproductions thereof in whole or in part).
7.
Section 5.
TAX WITHHOLDING; OFFSET; SECTION 409A.
All payments under the Plan will be subject to applicable withholding for federal, state and local taxes. If an
Eligible Employee is indebted to the Company on his or her termination date, the Company reserves the right to offset any
severance payments under the Plan by the amount of such indebtedness. All severance benefits provided under the Plan are
intended to satisfy the requirements for an exemption from application of Section 409A of the Code to the maximum extent
that an exemption is available and any ambiguities herein shall be interpreted accordingly.
Notwithstanding anything to the contrary set forth herein, any payments and benefits provided under the
Plan that constitute “deferred compensation” within the meaning of Section 409A of the Code and the regulations and other
guidance thereunder and any state law of similar effect (collectively “Section 409A”) shall not commence in connection with
an Eligible Employee’s termination of employment unless and until the Eligible Employee has also incurred a “separation
from service,” as such term is defined in Treasury Regulations Section 1.409A-1(h) (“Separation from Service”), unless the
Company reasonably determines that such amounts may be provided to the Eligible Employee without causing the Eligible
Employee to incur the adverse personal tax consequences under Section 409A.
It is intended that (i) each installment of any benefits payable under the Plan to an Eligible Employee be
regarded as a separate “payment” for purposes of Treasury Regulations Section 1.409A-2(b)(2)(i), (ii) all payments of any
such benefits under the Plan satisfy, to the greatest extent possible, the exemptions from the application of Section 409A
provided under Treasury Regulations Sections 1.409A-1(b)(4) and 1.409A-1(b)(9)(iii), and (iii) any such benefits consisting
of COBRA premiums also satisfy, to the greatest extent possible, the exemption from the application of Section 409A
provided under Treasury Regulations Section 1.409A-1(b)(9)(v). However, if the Company determines that any such
benefits payable under the Plan constitute “deferred compensation” under Section 409A and the Eligible Employee is a
“specified employee” of the Company, as such term is defined in Section 409A(a)(2)(B)(i), then, solely to the extent
necessary to avoid the imposition of the adverse personal tax consequences under Section 409A, (A) the timing of such
benefit payments shall be delayed until the earlier of (1) the date that is six (6) months and one (1) day after the Eligible
Employee’s Separation from Service and (2) the date of the Eligible Employee’s death (such applicable date, the “Delayed
Initial Payment Date”), and (B) the Company shall (1) pay the Eligible Employee a lump sum amount equal to the sum of
the benefit payments that the Eligible Employee would otherwise have received through the Delayed Initial Payment Date if
the commencement of the payment of the benefits had not been delayed pursuant to this paragraph and (2) commence paying
the balance, if any, of the benefits in accordance with the applicable payment schedule.
In no event shall payment of any benefits under the Plan be made prior to an Eligible Employee’s
Termination Date or prior to the effective date of the Release. If the Company determines that any payments or benefits
provided under the Plan constitute “deferred compensation” under Section 409A, and the Eligible Employee’s Separation
from Service occurs at a time during the calendar year when the Release could become effective in the calendar year
following the calendar year in which the Eligible Employee’s Separation from Service occurs, then regardless of when the
Release is returned to the Company and becomes effective, the Release will not be deemed effective any earlier than the
latest permitted effective date.
8.
Section 6.
REEMPLOYMENT.
In the event of an Eligible Employee’s reemployment by the Company during the period of time in respect
of which severance benefits pursuant to the Plan have been paid, the Company, in its sole and absolute discretion, may
require such Eligible Employee to repay to the Company all or a portion of such severance benefits as a condition of
reemployment.
Section 7.
TRANSFER AND ASSIGNMENT.
The rights and obligations of an Eligible Employee under this Plan may not be transferred or assigned without the prior
written consent of the Company. The Plan shall be binding upon any entity or person who is a successor by merger,
acquisition, consolidation or otherwise to the business formerly carried on by the Company without regard to whether or not
such entity or person actively assumes the obligations hereunder and without regard to whether or not a Change in Control
occurs.
Section 8.
RIGHT TO INTERPRET AND ADMINISTER PLAN; AMENDMENT AND TERMINATION.
(a)
Interpretation and Administration. Prior to the Closing, the Board shall be the Plan
Administrator and shall have the exclusive discretion and authority to establish rules, forms, and procedures for the
administration of the Plan and to construe and interpret the Plan and to decide any and all questions of fact, interpretation,
definition, computation or administration arising in connection with the operation of the Plan, including, but not limited to,
the eligibility to participate in the Plan and amount of benefits paid under the Plan. The rules, interpretations, computations
and other actions of the Board shall be binding and conclusive on all persons. Upon and after the Closing, the Plan will be
interpreted and administered in good faith by the Representative who shall be the Plan Administrator during such period.
All actions taken by the Representative in interpreting the terms of the Plan and administering the Plan upon and after the
Closing will be final and binding on all Eligible Employees. Any references in this Plan to the “Board” or “Plan
Administrator” with respect to periods following the Closing shall mean the Representative.
(b)
Amendment. The Plan Administrator reserves the right to amend this Plan at any time in its
discretion; provided, however, that any amendment of the Plan that would adversely affect a particular employee will not be
effective as to such employee without his or her written consent if at the time of such amendment such employee previously
has been terminated in a Covered Termination.
(c)
Termination. The Plan will automatically terminate following satisfaction of all the Company’s
obligations under the Plan. The Plan may be earlier terminated at any time at the discretion of the Plan Administrator,
provided, however, that no such discretionary termination by the Plan Administrator may be implemented with respect to
any employee without his or her written consent if at such time the employee previously has been terminated in a Covered
Termination.
Section 9.
NO IMPLIED EMPLOYMENT CONTRACT.
The Plan shall not be deemed (i) to give any employee or other person any right to be retained in the employ
of the Company or (ii) to interfere with the right of the Company to discharge any employee or other person at any time,
with or without cause, which right is hereby reserved.
9.
Section 10.
LEGAL CONSTRUCTION.
This Plan is intended to be governed by and shall be construed in accordance with the Employee Retirement
Income Security Act of 1974 (“ERISA”) and, to the extent not preempted by ERISA, the laws of the Commonwealth of
Pennsylvania.
Section 11.
CLAIMS, INQUIRIES AND APPEALS.
(a)
Applications for Benefits and Inquiries. Any application for benefits, inquiries about the Plan or
inquiries about present or future rights under the Plan must be submitted to the Plan Administrator in writing by an applicant
(or his or her authorized representative). The Plan Administrator is:
Aclaris Therapeutics, Inc.
Board of Directors
101 Lindenwood Drive, Suite 400
Malvern, PA 19355
(b)
Denial of Claims. In the event that any application for benefits is denied in whole or in part, the
Plan Administrator must provide the applicant with written or electronic notice of the denial of the application, and of the
applicant’s right to review the denial. Any electronic notice will comply with the regulations of the U.S. Department of
Labor. The notice of denial will be set forth in a manner designed to be understood by the applicant and will include the
following:
(1)
(2)
(3)
the specific reason or reasons for the denial;
references to the specific Plan provisions upon which the denial is based;
a description of any additional information or material that the Plan Administrator needs to
complete the review and an explanation of why such information or material is necessary; and
an explanation of the Plan’s review procedures and the time limits applicable to such
procedures, including a statement of the applicant’s right to bring a civil action under Section 502(a) of ERISA following a
denial on review of the claim, as described in Section 11(d) below.
(4)
This notice of denial will be given to the applicant within ninety (90) days after the Plan Administrator
receives the application, unless special circumstances require an extension of time, in which case, the Plan Administrator has
up to an additional ninety (90) days for processing the application. If an extension of time for processing is required, written
notice of the extension will be furnished to the applicant before the end of the initial ninety (90) day period.
This notice of extension will describe the special circumstances necessitating the additional time and the
date by which the Plan Administrator is to render its decision on the application.
(c)
Request for a Review. Any person (or that person’s authorized representative) for whom an
application for benefits is denied, in whole or in part, may appeal the denial by submitting a request for a review to the Plan
Administrator within sixty (60) days after the application is denied. A request for a review shall be in writing and shall be
addressed to:
10.
Aclaris Therapeutics, Inc.
Board of Directors
101 Lindenwood Drive, Suite 400
Malvern, PA 19355
A request for review must set forth all of the grounds on which it is based, all facts in support of the request and any other
matters that the applicant feels are pertinent. The applicant (or his or her representative) shall have the opportunity to submit
(or the Plan Administrator may require the applicant to submit) written comments, documents, records, and other
information relating to his or her claim. The applicant (or his or her representative) shall be provided, upon request and free
of charge, reasonable access to, and copies of, all documents, records and other information relevant to his or her claim. The
review shall take into account all comments, documents, records and other information submitted by the applicant (or his or
her representative) relating to the claim, without regard to whether such information was submitted or considered in the
initial benefit determination.
(d)
Decision on Review. The Plan Administrator will act on each request for review within sixty (60)
days after receipt of the request, unless special circumstances require an extension of time (not to exceed an additional sixty
(60) days), for processing the request for a review. If an extension for review is required, written notice of the extension will
be furnished to the applicant within the initial sixty (60) day period. This notice of extension will describe the special
circumstances necessitating the additional time and the date by which the Plan Administrator is to render its decision on the
review. The Plan Administrator will give prompt, written or electronic notice of its decision to the applicant. Any electronic
notice will comply with the regulations of the U.S. Department of Labor. In the event that the Plan Administrator confirms
the denial of the application for benefits in whole or in part, the notice will set forth, in a manner calculated to be understood
by the applicant, the following:
(1)
(2)
(3)
the specific reason or reasons for the denial;
references to the specific Plan provisions upon which the denial is based;
a statement that the applicant is entitled to receive, upon request and free of charge,
reasonable access to, and copies of, all documents, records and other information relevant to his or her claim; and
(4)
a statement of the applicant’s right to bring a civil action under Section 502(a) of ERISA.
(e)
Rules and Procedures. The Plan Administrator will establish rules and procedures, consistent with
the Plan and with ERISA, as necessary and appropriate in carrying out its responsibilities in reviewing benefit claims. The
Plan Administrator may require an applicant who wishes to submit additional information in connection with an appeal from
the denial of benefits to do so at the applicant’s own expense.
(f)
Exhaustion of Remedies. No legal action for benefits under the Plan may be brought until the
applicant (i) has submitted a written application for benefits in accordance with the procedures described by Section 11(a)
above, (ii) has been notified by the Plan Administrator that the application is denied, (iii) has filed a written request for a
review of the application in accordance with the appeal procedure described in Section 11(c) above, and (iv) has been
notified that the Plan Administrator has denied the appeal. Notwithstanding the foregoing, if the Plan Administrator does
not respond to an
11.
Eligible Employee’s claim or appeal within the relevant time limits specified in this Section 11, the Eligible Employee may
bring legal action for benefits under the Plan pursuant to Section 502(a) of ERISA.
Section 12.
BASIS OF PAYMENTS TO AND FROM PLAN.
The Plan shall be unfunded, and all cash payments under the Plan shall be paid only from the general assets
of the Company.
Section 13. OTHER PLAN INFORMATION.
(a)
Employer and Plan Identification Numbers. The Employer Identification Number assigned to
the Company (which is the “Plan Sponsor” as that term is used in ERISA) by the Internal Revenue Service is 46-0571712.
The Plan Number assigned to the Plan by the Plan Sponsor pursuant to the instructions of the Internal Revenue Service is
[___].
(b)
Ending Date for Plan’s Fiscal Year. The date of the end of the fiscal year for the purpose of
maintaining the Plan’s records is December 31.
(c)
Agent for the Service of Legal Process. The agent for the service of legal process with respect to
the Plan is:
Aclaris Therapeutics, Inc.
101 Lindenwood Drive, Suite 400
Malvern, PA 19355
In addition, service of legal process may be made upon the Plan Administrator.
(d)
Plan Sponsor. The “Plan Sponsor” is:
Aclaris Therapeutics, Inc.
101 Lindenwood Drive, Suite 400
Malvern, PA 19355
(484) 324-7933
(e)
Plan Administrator. The Plan Administrator is the Board prior to the Closing and the
Representative upon and following the Closing. The Plan Administrator’s contact information is:
Aclaris Therapeutics, Inc.
Board of Directors or Representative
101 Lindenwood Drive, Suite 400
Malvern, PA 19355
(484) 324-7933
The Plan Administrator is the named fiduciary charged with the responsibility for administering the Plan.
12.
Section 14.
STATEMENT OF ERISA RIGHTS.
Participants in this Plan (which is a welfare benefit plan sponsored by Aclaris Therapeutics, Inc.) are entitled
to certain rights and protections under ERISA. If you are an Eligible Employee, you are considered a participant in the Plan
and, under ERISA, you are entitled to:
(a)
Receive Information About Your Plan and Benefits
(1)
Examine, without charge, at the Plan Administrator’s office and at other specified locations,
such as worksites, all documents governing the Plan and a copy of the latest annual report (Form 5500 Series), if applicable,
filed by the Plan with the U.S. Department of Labor and available at the Public Disclosure Room of the Employee Benefits
Security Administration;
Obtain, upon written request to the Plan Administrator, copies of documents governing the
operation of the Plan and copies of the latest annual report (Form 5500 Series), if applicable, and an updated (as necessary)
Summary Plan Description. The Plan Administrator may make a reasonable charge for the copies; and
(2)
Administrator is required by law to furnish each Eligible Employee with a copy of this summary annual report.
(3)
Receive a summary of the Plan’s annual financial report, if applicable. The Plan
(b)
Prudent Actions by Plan Fiduciaries. In addition to creating rights for Eligible Employees,
ERISA imposes duties upon the people who are responsible for the operation of the employee benefit plan. The people who
operate the Plan, called “fiduciaries” of the Plan, have a duty to do so prudently and in the interest of you and other Eligible
Employees and beneficiaries. No one, including your employer, your union or any other person, may fire you or otherwise
discriminate against you in any way to prevent you from obtaining a Plan benefit or exercising your rights under ERISA.
(c)
Enforce Your Rights. If your claim for a Plan benefit is denied or ignored, in whole or in part, you
have a right to know why this was done, to obtain copies of documents relating to the decision without charge, and to appeal
any denial, all within certain time schedules.
Under ERISA, there are steps you can take to enforce the above rights. For instance, if you request
a copy of Plan documents or the latest annual report from the Plan, if applicable, and do not receive them within thirty (30)
days, you may file suit in a Federal court. In such a case, the court may require the Plan Administrator to provide the
materials and pay you up to $110 a day until you receive the materials, unless the materials were not sent because of reasons
beyond the control of the Plan Administrator.
a state or Federal court.
If you have a claim for benefits which is denied or ignored, in whole or in part, you may file suit in
If you are discriminated against for asserting your rights, you may seek assistance from the U.S.
Department of Labor, or you may file suit in a Federal court. The court will decide who should pay court costs and legal
fees. If you are successful, the court may order the person you have sued to pay these costs and fees. If you lose, the court
may order you to pay these costs and fees, for example, if it finds your claim is frivolous.
13.
(d)
Assistance with Your Questions. If you have any questions about the Plan, you should contact the
Plan Administrator. If you have any questions about this statement or about your rights under ERISA, or if you need
assistance in obtaining documents from the Plan Administrator, you should contact the nearest office of the Employee
Benefits Security Administration, U.S. Department of Labor, listed in your telephone directory or the Division of Technical
Assistance and Inquiries, Employee Benefits Security Administration, U.S. Department of Labor, 200 Constitution Avenue
N.W., Washington, D.C. 20210. You may also obtain certain publications about your rights and responsibilities under
ERISA by calling the publications hotline of the Employee Benefits Security Administration.
14.
For Eligible Employees Age 40 or Older
Individual Termination
EXHIBIT A
RELEASE AGREEMENT
I understand and agree completely to the terms set forth in the Aclaris Therapeutics, Inc. Change in Control
Severance Benefit Plan (the “Plan”).
I understand that this Release Agreement (the “Release”), together with the Plan, constitutes the complete, final and
exclusive embodiment of the entire agreement between Aclaris Therapeutics, Inc. (the “Company”), Affiliates of the
Company and me with regard to the subject matter hereof. I am not relying on any promise or representation by the
Company or an Affiliate of the Company that is not expressly stated therein. Certain capitalized terms used in this Release
are defined in the Plan.
I hereby confirm my obligations under my Confidentiality and Invention Rights, Non-competition and Non-
solicitation Agreement with the Company and/or an Affiliate of the Company.
In consideration of the severance benefits and other consideration provided to me under the Plan that I am not
otherwise entitled to receive, I hereby generally and completely release the Company and its Affiliates, and their parents,
subsidiaries, successors, predecessors and affiliates, and their current and former partners, members, directors, officers,
employees, stockholders, shareholders, agents, attorneys, predecessors, successors, insurers, affiliates and assigns, from any
and all claims, liabilities and obligations, both known and unknown, that arise out of or are in any way related to events,
acts, conduct, or omissions occurring at any time prior to and including the date I sign this Release (collectively, the
“Released Claims”). The Released Claims include, but are not limited to: (a) all claims arising out of or in any way related
to my employment with the Company and its Affiliates, or their affiliates, or the termination of that employment; (b) all
claims related to my compensation or benefits, including salary, bonuses, commissions, vacation pay, expense
reimbursements, severance pay, fringe benefits, stock, stock options, or any other ownership, equity, or profits interests in
the Company and its Affiliates, or their affiliates; (c) all claims for breach of contract, wrongful termination, and breach of
the implied covenant of good faith and fair dealing; (d) all tort claims, including claims for fraud, defamation, emotional
distress, and discharge in violation of public policy; and (e) all federal, state, and local statutory claims, including but not
limited to claims for discrimination, harassment, retaliation, attorneys’ fees, or other claims arising under the federal Civil
Rights Act of 1964 (as amended), the federal Americans with Disabilities Act of 1990 (as amended), the federal Age
Discrimination in Employment Act (as amended) (“ADEA”), the federal Family and Medical Leave Act, the federal
Employee Retirement Income Security Act of 1974 (as amended), the Pennsylvania Human Relations Act, the Pennsylvania
Wage Payment and Collection Law, the Pennsylvania Whistleblower Law and the Pennsylvania Equal Pay Law.
Notwithstanding the foregoing, I understand that the following rights or claims are not included in the Released
Claims: (a) any rights or claims for indemnification I may have pursuant to any written indemnification agreement with the
Company or its Affiliates to which I am a party; the charter, bylaws, or operating agreements of the Company or its
Affiliates; or under applicable law; or (b) any rights that cannot be waived as a matter of law. In addition, I understand that
nothing in this Release prevents me from filing, cooperating with, or participating in any proceeding before the Equal
Employment Opportunity Commission, the Department of Labor, the National Labor Relations Board, the Securities and
Exchange Commission, or any other federal, state or local government agency or commission (collectively, the
“Government Agencies”). This Release does not limit my ability to communicate with any Government Agencies or
otherwise participate in any investigation or proceeding that may be
1.
conducted by any Government Agencies, including providing documents or other information, without notice to the
Company. While this Release does not limit my right to receive an award for information provided to the Securities and
Exchange Commission, I understand and agree that, to maximum extent permitted by law, I am otherwise waiving any and
all rights I may have to individual relief based on any claims that I have released and any rights that I have waived by
signing this Release. I hereby represent and warrant that, other than the claims identified in this paragraph, I am not aware of
any claims I have or might have that are not included in the Released Claims.
I acknowledge that I am knowingly and voluntarily waiving and releasing any rights I may have under the ADEA,
and that the consideration given under the Plan for the waiver and release in this paragraph is in addition to anything of
value to which I was already entitled. I further acknowledge that I have been advised by this writing, as required by the
ADEA, that: (a) my waiver and release do not apply to any rights or claims that may arise after the date I sign this Release;
(b) I should consult with an attorney prior to signing this Release (although I may choose voluntarily not do so); (c) I have
twenty-one (21) days to consider this Release (although I may choose voluntarily to sign this Release earlier); (d) I have
seven (7) days following the date I sign this Release to revoke the Release by providing written notice to an officer of the
Company; and (e) this Release shall not be effective until the date upon which the revocation period has expired, which shall
be the eighth day after I sign this Release provided I have not revoked it.
I hereby represent that I have been paid all compensation owed and for all hours worked; I have received all the
leave and leave benefits and protections for which I am eligible pursuant to the Family and Medical Leave Act or otherwise;
and I have not suffered any on-the-job injury for which I have not already filed a workers’ compensation claim.
I acknowledge that to become effective, I must sign and return this Release to the Company so that it is received not
later than twenty-one (21) days following the date it is provided to me or such other date as specified by the Company.
ELIGIBLE EMPLOYEE
Printed Name:
Signature:
Date:
2.
For Eligible Employees Age 40 or Older
Group Termination
EXHIBIT B
RELEASE AGREEMENT
I understand and agree completely to the terms set forth in the Aclaris Therapeutics, Inc. Change in Control
Severance Benefit Plan (the “Plan”).
I understand that this Release Agreement (the “Release”), together with the Plan, constitutes the complete, final and
exclusive embodiment of the entire agreement between the Aclaris Therapeutics, Inc. (the “Company”), Affiliates of the
Company and me with regard to the subject matter hereof. I am not relying on any promise or representation by the
Company or an Affiliate of the Company that is not expressly stated therein. Certain capitalized terms used in this Release
are defined in the Plan.
I hereby confirm my obligations under my Confidentiality and Invention Rights, Non-competition and Non-
solicitation Agreement with the Company and/or an affiliate of the Company.
In consideration of the severance benefits and other consideration provided to me under the Plan that I am not
otherwise entitled to receive, I hereby generally and completely release the Company and its Affiliates, and their parents,
subsidiaries, successors, predecessors and affiliates, and its and their current and former partners, members, directors,
officers, employees, stockholders, shareholders, agents, attorneys, predecessors, successors, insurers, affiliates and assigns,
from any and all claims, liabilities and obligations, both known and unknown, that arise out of or are in any way related to
events, acts, conduct, or omissions occurring at any time prior to and including the date I sign this Release (collectively, the
“Released Claims”). The Released Claims include, but are not limited to: (a) all claims arising out of or in any way related
to my employment with the Company and its Affiliates, or their affiliates, or the termination of that employment; (b) all
claims related to my compensation or benefits, including salary, bonuses, commissions, vacation pay, expense
reimbursements, severance pay, fringe benefits, stock, stock options, or any other ownership, equity, or profits interests in
the Company and its Affiliates, or their affiliates; (c) all claims for breach of contract, wrongful termination, and breach of
the implied covenant of good faith and fair dealing; (d) all tort claims, including claims for fraud, defamation, emotional
distress, and discharge in violation of public policy; and (e) all federal, state, and local statutory claims, including but not
limited to claims for discrimination, harassment, retaliation, attorneys’ fees, or other claims arising under the federal Civil
Rights Act of 1964 (as amended), the federal Americans with Disabilities Act of 1990 (as amended), the federal Age
Discrimination in Employment Act (as amended) (“ADEA”), the federal Family and Medical Leave Act, the federal
Employee Retirement Income Security Act of 1974 (as amended), the Pennsylvania Human Relations Act, the Pennsylvania
Wage Payment and Collection Law, the Pennsylvania Whistleblower Law and the Pennsylvania Equal Pay Law.
Notwithstanding the foregoing, I understand that the following rights or claims are not included in the Released
Claims: (a) any rights or claims for indemnification I may have pursuant to any written indemnification agreement with the
Company or its Affiliates to which I am a party; the charter, bylaws, or operating agreements of the Company or its
Affiliates; or under applicable law; or (b) any rights that cannot be waived as a matter of law. In addition, I understand that
nothing in this Release prevents me from filing, cooperating with, or participating in any proceeding before the Equal
Employment Opportunity Commission, the Department of Labor, the National Labor Relations Board, the Securities and
Exchange Commission, or any other federal, state or local government agency or commission (collectively, the
“Government Agencies”). This Release does not limit my ability to communicate with any Government Agencies or
otherwise participate in any investigation or proceeding that may be conducted by any Government Agencies, including
providing documents or other information, without
1.
notice to the Company. While this Release does not limit my right to receive an award for information provided to the
Securities and Exchange Commission, I understand and agree that, to maximum extent permitted by law, I am otherwise
waiving any and all rights I may have to individual relief based on any claims that I have released and any rights that I have
waived by signing this Release. I hereby represent and warrant that, other than the claims identified in this paragraph, I am
not aware of any claims I have or might have that are not included in the Released Claims.
I acknowledge that I am knowingly and voluntarily waiving and releasing any rights I may have under the ADEA,
and that the consideration given under the Plan for the waiver and release in this paragraph is in addition to anything of
value to which I was already entitled. I further acknowledge that I have been advised by this writing, as required by the
ADEA, that: (a) my waiver and release do not apply to any rights or claims that may arise after the date I sign this Release;
(b) I should consult with an attorney prior to signing this Release (although I may choose voluntarily not to do so); (c) I have
forty-five (45) days to consider this Release (although I may choose voluntarily to sign this Release earlier); (d) I have seven
(7) days following the date I sign this Release to revoke the Release by providing written notice to an officer of the
Company; (e) this Release shall not be effective until the date upon which the revocation period has expired, which shall be
the eighth day after I sign this Release provided I have not revoked it; and (f) I have received with this Release all of the
information required by the ADEA, including without limitation a detailed list of the job titles and ages of all employees
who were terminated in this group termination and the ages of all employees of the Company in the same job classification
or organizational unit who were not terminated.
I hereby represent that I have been paid all compensation owed and for all hours worked; I have received all the
leave and leave benefits and protections for which I am eligible pursuant to the Family and Medical Leave Act or otherwise;
and I have not suffered any on-the-job injury for which I have not already filed a workers’ compensation claim.
I acknowledge that to become effective, I must sign and return this Release to the Company so that it is received not
later than forty-five (45) days following the date it is provided to me or such other date as specified by the Company.
ELIGIBLE EMPLOYEE
Printed Name:
Signature:
Date:
2.
For Eligible Employees Under Age 40
Individual or Group Termination
EXHIBIT C
RELEASE AGREEMENT
I understand and agree completely to the terms set forth in the Aclaris Therapeutics, Inc. Change in Control
Severance Benefit Plan (the “Plan”).
I understand that this Release Agreement (the “Release”), together with the Plan, constitutes the complete, final and
exclusive embodiment of the entire agreement between Aclaris Therapeutics, Inc. (the “Company”), Affiliates of the
Company and me with regard to the subject matter hereof. I am not relying on any promise or representation by the
Company or an Affiliate of the Company that is not expressly stated therein. Certain capitalized terms used in this Release
are defined in the Plan.
I hereby confirm my obligations under my Confidentiality and Invention Rights, Non-competition and Non-
solicitation Agreement with the Company and/or an Affiliate of the Company.
In consideration of the severance benefits and other consideration provided to me under the Plan that I am not
otherwise entitled to receive, I hereby generally and completely release the Company and its Affiliates, and their parents,
subsidiaries, successors, predecessors and affiliates, and its and their current and former partners, members, directors,
officers, employees, stockholders, shareholders, agents, attorneys, predecessors, successors, insurers, affiliates and assigns,
from any and all claims, liabilities and obligations, both known and unknown, that arise out of or are in any way related to
events, acts, conduct, or omissions occurring at any time prior to and including the date I sign this Release (collectively, the
“Released Claims”). The Released Claims include, but are not limited to: (a) all claims arising out of or in any way related
to my employment with the Company and its Affiliates, or their affiliates, or the termination of that employment; (b) all
claims related to my compensation or benefits, including salary, bonuses, commissions, vacation pay, expense
reimbursements, severance pay, fringe benefits, stock, stock options, or any other ownership, equity, or profits interests in
the Company and its Affiliates, or their affiliates; (c) all claims for breach of contract, wrongful termination, and breach of
the implied covenant of good faith and fair dealing; (d) all tort claims, including claims for fraud, defamation, emotional
distress, and discharge in violation of public policy; and (e) all federal, state, and local statutory claims, including but not
limited to claims for discrimination, harassment, retaliation, attorneys’ fees, or other claims arising under the federal Civil
Rights Act of 1964 (as amended), the federal Americans with Disabilities Act of 1990 (as amended), the federal Family and
Medical Leave Act, the federal Employee Retirement Income Security Act of 1974 (as amended), the Pennsylvania Human
Relations Act, the Pennsylvania Wage Payment and Collection Law, the Pennsylvania Whistleblower Law and the
Pennsylvania Equal Pay Law.
Notwithstanding the foregoing, I understand that the following rights or claims are not included in the Released
Claims: (a) any rights or claims for indemnification I may have pursuant to any written indemnification agreement with the
Company or its Affiliates to which I am a party; the charter, bylaws, or operating agreements of the Company or its
Affiliates; or under applicable law; or (b) any rights that cannot be waived as a matter of law. In addition, I understand that
nothing in this Release prevents me from filing, cooperating with, or participating in any proceeding before the Equal
Employment Opportunity Commission, the Department of Labor, the National Labor Relations Board, the Securities and
Exchange Commission, or any other federal, state or local government agency or commission (collectively, the
“Government Agencies”). This Release does not limit my ability to communicate with
1.
any Government Agencies or otherwise participate in any investigation or proceeding that may be conducted by any
Government Agencies, including providing documents or other information, without notice to the Company. While this
Release does not limit my right to receive an award for information provided to the Securities and Exchange Commission, I
understand and agree that, to maximum extent permitted by law, I am otherwise waiving any and all rights I may have to
individual relief based on any claims that I have released and any rights that I have waived by signing this Release. I hereby
represent and warrant that, other than the claims identified in this paragraph, I am not aware of any claims I have or might
have that are not included in the Released Claims.
I hereby represent that I have been paid all compensation owed and for all hours worked; I have received all the
leave and leave benefits and protections for which I am eligible pursuant to the Family and Medical Leave Act or otherwise;
and I have not suffered any on-the-job injury for which I have not already filed a workers’ compensation claim.
I acknowledge that to become effective, I must sign and return this Release to the Company so that it is received not
later than fourteen (14) days following the date it is provided to me or such other date as specified by the Company.
ELIGIBLE EMPLOYEE
Printed Name:
Signature:
Date:
2.
ACLARIS THERAPEUTICS, INC.
FIRST AMENDMENT TO
CHANGE IN CONTROL SEVERANCE BENEFIT PLAN
WHEREAS, Aclaris Therapeutics, Inc., a Delaware Corporation (the “Company”), has adopted and maintains the
“Aclaris Therapeutics, Inc. Change in Control Severance Benefit Plan” (the “Plan”) for the benefit of “Eligible Employees,”
as that term is defined in the Plan; and
WHEREAS, the Plan Administrator (as defined in the Plan) desires to amend the requirements to be an “Eligible
Employee.”
NOW THEREFORE, pursuant to the power of amendment contained in Section 8(b) of the Plan, the Plan is hereby
amended as follows:
The first sentence of Section 2(a) of the Plan is hereby deleted in its entirety and replaced with the following, which
1.
shall be effective for employees of the Company whose employment commences after October 2, 2019:
“An employee of the Company is eligible to participate in the Plan if (i) the employee is an officer or other
employee who reports directly to the Chief Executive Officer of the Company on any date within the Covered
Period; (ii) the Board has designated such employee as eligible to participate in the Plan; (iii) such employee’s
employment with the Company terminates due to a Covered Termination; and (iv) such employee meets the other
Plan eligibility requirements set forth in this Section 2 and the Plan.
Each reference in the Plan to “101 Lindenwood Drive, Suite 400, Malvern, PA 19355” is hereby deleted in its
2.
entirety and replaced with the following:
“640 Lee Road, Suite 200
Wayne, PA 19087”
*
*
*
*
*
Subsidiaries of Aclaris Therapeutics, Inc.
Exhibit 21.1
Name of Subsidiary
Aclaris Therapeutics International Limited
Aclaris Life Sciences, Inc.
Confluence Discovery Technologies, Inc.
Jurisdiction of Incorporation or
Organization
United Kingdom
Delaware
Delaware
Exhibit 23.1
CONSENT OF INDEPENDENT REGISTERED PUBLIC ACCOUNTING FIRM
We hereby consent to the incorporation by reference in the Registration Statements on Form S-3 (No. 333-212095) and
Form S-8 (Nos. 333-238079, 333-230614, 333-223922, 333-220149, 333-216703, 333-210379 and 333-207434) of Aclaris
Therapeutics, Inc. of our report dated February 25, 2021 relating to the financial statements, which appears in this Form 10-
K.
/s/ PricewaterhouseCoopers LLP
Philadelphia, Pennsylvania
February 25, 2021
Exhibit 31.1
CERTIFICATION OF PRINCIPAL EXECUTIVE OFFICER
PURSUANT TO SECTION 302 OF THE SARBANES-OXLEY ACT OF 2002
I, Neal Walker, certify that:
1. I have reviewed this annual report on Form 10-K of Aclaris Therapeutics, Inc. (the “registrant”);
2. Based on my knowledge, this report does not contain any untrue statement of a material fact or omit to state a material fact necessary
to make the statements made, in light of the circumstances under which such statements were made, not misleading with respect to
the period covered by this report;
3. Based on my knowledge, the financial statements, and other financial information included in this report, fairly present in all
material respects the financial condition, results of operations and cash flows of the registrant as of, and for, the periods presented in
this report;
4. The registrant’s other certifying officer(s) and I are responsible for establishing and maintaining disclosure controls and procedures
(as defined in Exchange Act Rules 13a-15(e) and 15d-15(e)) and 15d-15(e)) and internal control over financial reporting (as defined
in Exchange Act Rules 13a-15(f) and 15d-15(f)) for the registrant and have:
(a) Designed such disclosure controls and procedures, or caused such disclosure controls and procedures to be designed under our
supervision, to ensure that material information relating to the registrant, including its consolidated subsidiaries, is made known
to us by others within those entities, particularly during the period in which this report is being prepared;
(b) Designed such internal control over financial reporting, or caused such internal control over financial reporting to be designed
under our supervision, to provide reasonable assurance regarding the reliability of financial reporting and the preparation of
financial statements for external purposes in accordance with generally accepted accounting principles;
(c) Evaluated the effectiveness of the registrant’s disclosure controls and procedures and presented in this report our conclusions
about the effectiveness of the disclosure controls and procedures, as of the end of the period covered by this report based on
such evaluation; and
(d) Disclosed in this report any change in the registrant’s internal control over financial reporting that occurred during the
registrant’s most recent fiscal quarter (the registrant’s fourth fiscal quarter in the case of an annual report) that has materially
affected, or is reasonably likely to materially affect, the registrant’s internal control over financial reporting; and
5. The registrant’s other certifying officer(s) and I have disclosed, based on our most recent evaluation of internal control over financial
reporting, to the registrant’s auditors and the audit committee of the registrant’s board of directors (or persons performing the
equivalent functions):
(a) All significant deficiencies and material weaknesses in the design or operation of internal control over financial reporting which
are reasonably likely to adversely affect the registrant’s ability to record, process, summarize and report financial information;
and
(b) Any fraud, whether or not material, that involves management or other employees who have a significant role in the registrant’s
internal control over financial reporting.
Date: February 25, 2021
/s/ Neal Walker
Neal Walker
President & Chief Executive Officer
(principal executive officer)
Exhibit 31.2
CERTIFICATION OF PRINCIPAL EXECUTIVE OFFICER
PURSUANT TO SECTION 302 OF THE SARBANES-OXLEY ACT OF 2002
I, Frank Ruffo, certify that:
1. I have reviewed this annual report on Form 10-K of Aclaris Therapeutics, Inc. (the “registrant”);
2. Based on my knowledge, this report does not contain any untrue statement of a material fact or omit to state a material fact necessary
to make the statements made, in light of the circumstances under which such statements were made, not misleading with respect to
the period covered by this report;
3. Based on my knowledge, the financial statements, and other financial information included in this report, fairly present in all
material respects the financial condition, results of operations and cash flows of the registrant as of, and for, the periods presented in
this report;
4. The registrant’s other certifying officer(s) and I are responsible for establishing and maintaining disclosure controls and procedures
(as defined in Exchange Act Rules 13a-15(e) and 15d-15(e)) and 15d-15(e)) and internal control over financial reporting (as defined
in Exchange Act Rules 13a-15(f) and 15d-15(f)) for the registrant and have:
(a) Designed such disclosure controls and procedures, or caused such disclosure controls and procedures to be designed under our
supervision, to ensure that material information relating to the registrant, including its consolidated subsidiaries, is made known
to us by others within those entities, particularly during the period in which this report is being prepared;
(b) Designed such internal control over financial reporting, or caused such internal control over financial reporting to be designed
under our supervision, to provide reasonable assurance regarding the reliability of financial reporting and the preparation of
financial statements for external purposes in accordance with generally accepted accounting principles;
(c) Evaluated the effectiveness of the registrant’s disclosure controls and procedures and presented in this report our conclusions
about the effectiveness of the disclosure controls and procedures, as of the end of the period covered by this report based on
such evaluation; and
(d) Disclosed in this report any change in the registrant’s internal control over financial reporting that occurred during the
registrant’s most recent fiscal quarter (the registrant’s fourth fiscal quarter in the case of an annual report) that has materially
affected, or is reasonably likely to materially affect, the registrant’s internal control over financial reporting; and
5. The registrant’s other certifying officer(s) and I have disclosed, based on our most recent evaluation of internal control over financial
reporting, to the registrant’s auditors and the audit committee of the registrant’s board of directors (or persons performing the
equivalent functions):
(a) All significant deficiencies and material weaknesses in the design or operation of internal control over financial reporting which
are reasonably likely to adversely affect the registrant’s ability to record, process, summarize and report financial information;
and
(b) Any fraud, whether or not material, that involves management or other employees who have a significant role in the registrant’s
internal control over financial reporting.
Date: February 25, 2021
/s/ Frank Ruffo
Frank Ruffo
Chief Financial Officer
(principal financial officer and principal accounting officer)
CERTIFICATIONS OF
PRINCIPAL EXECUTIVE OFFICER AND PRINCIPAL FINANCIAL OFFICER
PURSUANT TO 18 U.S.C. SECTION 1350,
AS ADOPTED PURSUANT TO
SECTION 906 OF THE SARBANES-OXLEY ACT OF 2002
Exhibit 32.1
Pursuant to the requirement set forth in Rule 13a-14(b) of the Securities Exchange Act of 1934, as amended (the
“Exchange Act”), and Section 1350 of Chapter 63 of Title 18 of the United States Code (18 U.S.C. §1350), Neal Walker,
President and Chief Executive Officer of Aclaris Therapeutics, Inc. (the “Company”), and Frank Ruffo, Chief Financial
Officer of the Company, each hereby certifies that, to the best of his knowledge:
1. The Company’s Annual Report on Form 10-K for the period ended December 31, 2020 (the “Annual Report”),
to which this Certification is attached as Exhibit 32.1, fully complies with the requirements of Section 13(a) or
Section 15(d) of the Exchange Act, and
2. The information contained in the Annual Report fairly presents, in all material respects, the financial condition
of the Company as of the end of the period covered by the Annual Report and results of operations of the
Company for the periods covered by the Annual Report.
In Witness Whereof, the undersigned have set their hands hereto as of the 25th day of February 2021.
/s/ Neal Walker
Neal Walker
President & Chief Executive Officer
/s/ Frank Ruffo
Frank Ruffo
Chief Financial Officer
* This certification accompanies the Form 10-K to which it relates, is not deemed filed with the Securities and Exchange
Commission and is not to be incorporated by reference into any filing of the Company under the Securities Act of 1933,
as amended, or the Exchange Act (whether made before or after the date of the Form 10-K), irrespective of any general
incorporation language contained in such filing.