Item 2. Management’s Discussion and Analysis
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operations
Certain statements contained in this Quarterly Report on Form 10-Q may constitute forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. The words “may,” “might,” “can,” “will,” “to be,” “could,” “would,” “should,” “expect,” “intend,” “plan,” “objective,” “anticipate,” “believe,” “estimate,” “predict,” “project,” “potential,” “likely,” “continue,” “ongoing” or similar expressions, or the negative of such words, are intended to identify “forward-looking statements.” We have based these forward-looking statements on our current expectations and projections about future events. Because such statements include risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Factors that could cause or contribute to these differences include those below in this Quarterly Report on Form 10-Q and those in our Annual Report on Form 10-K, in each case under the caption “Risk Factors,” and in our other filings with the Securities and Exchange Commission, or SEC. Statements made herein are as of the date of the filing of this Form 10-Q with the SEC and should not be relied upon as of any subsequent date. Unless otherwise required by applicable law, we do not undertake, and we specifically disclaim, any obligation to update any forward-looking statements to reflect occurrences, developments, unanticipated events or circumstances after the date of such statement.
The following discussion and analysis of our financial condition and results of operations should be read in conjunction with our unaudited condensed consolidated financial statements and related notes that appear in Item 1 of this Quarterly Report on Form 10-Q and with our audited consolidated financial statements and related notes for the year ended December 31, 2020, which are included in our Annual Report on Form 10-K filed with the SEC on February 25, 2021.
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.
Clinical Programs
Zunsemetinib (ATI-450), an Investigational Oral MK2 Inhibitor
We submitted an Investigational New Drug Application, or IND, in April 2019 for zunsemetinib, 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 zunsemetinib as a potential alternative to injectable anti-TNF/IL1/IL6 biologics and JAK inhibitors for treating certain immuno-inflammatory diseases. Zunsemetinib has been adopted as the nonproprietary name for ATI-450.
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 zunsemetinib in 77 healthy subjects in August 2019 (ATI-450-PKPD-101). Final data from this trial demonstrated that zunsemetinib resulted in marked inhibition of TNFα, IL1β, IL8 and IL6. We also observed that zunsemetinib 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. Zunsemetinib was generally well-tolerated at all doses tested in the trial. The most common adverse events (reported by 2 or more subjects who received zunsemetinib) were dizziness, headache, upper respiratory tract infection, constipation, abdominal pain and nausea.
20
Table of Contents
Zunsemetinib was also evaluated at 80mg and 120mg doses twice daily in a second Phase 1 clinical trial in healthy subjects (ATI-450-PKPD-102). Final 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 zunsemetinib relative to doses tested in the first Phase 1 trial. 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 zunsemetinib) were headache, dizziness, nausea, parasthesia and, in the post-dosing follow-up period of the trial, dry skin. These adverse events were all mild in severity.
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 zunsemetinib in subjects with moderate to severe rheumatoid arthritis (ATI-450-RA-201). In the trial, which consisted of a 12-week treatment period and a 4-week follow-up period, 19 subjects were randomized in a 3:1 ratio and received either zunsemetinib at 50 mg twice daily or placebo, in combination with methotrexate, for 12 weeks.
The final per-protocol analysis, which consisted of the 17 subjects who completed the treatment period (15 in the treatment arm and two in the placebo arm), confirmed that zunsemetinib 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. Zunsemetinib 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, and ventricular extrasystoles, all of which were determined to be unrelated to treatment except for one UTI. Two subjects withdrew from the trial during the treatment period, one in the treatment arm and one in the placebo arm. The subject in the treatment arm withdrew due to an elevated creatine phosphokinase, or CPK, level, which was determined by the site investigator to be treatment-related; this subject also had palpitations and ventricular extrasystoles, which were unrelated to the trial medication. The subject in the placebo arm withdrew as a result of prohibited medication needed to treat muscle strain. There was also one non-treatment-related serious adverse event (COVID-19) reported in the 4-week follow-up period of the trial in a subject who was no longer receiving treatment; the subject withdrew during the 4-week follow-up period of the trial.
A final analysis, which consisted of the 17 subjects, 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 treatment 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 4-week 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 zunsemetinib into a Phase 2b trial in moderate to severe rheumatoid arthritis in the fourth quarter of 2021.
Psoriatic Arthritis and Moderate to Severe Hidradenitis Suppurativa
As part of the planned expansion of our Phase 2 immuno-inflammatory clinical development programs, we also plan to progress zunsemetinib into Phase 2 trials in psoriatic arthritis and moderate to severe hidradenitis suppurativa.
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.
21
Table of Contents
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). In the trial, which consisted of a 4-week treatment period and a 2-week follow-up period during which no treatment was given, 50 subjects with moderate to severe atopic dermatitis were randomized in a 1:1 ratio into one of two arms: ATI-1777 topical solution 2.0% w/w or vehicle applied twice daily. In June 2021, we announced that the trial achieved its primary endpoint, which was the percent change from baseline in the modified Eczema Area and Severity Index, or mEASI, score at week 4, with a high degree of statistical significance (p<0.001) (one-sided p-value), which corresponded to a 74.4% reduction in mEASI score from baseline at week 4 in subjects applying ATI-1777 compared to a 41.4% reduction in subjects applying vehicle. The final data was based on the full analysis set, or FAS, which was comprised of 48 subjects randomized and documented to have received at least one dose of trial medication. Positive trends in favor of ATI-1777 were observed in key secondary efficacy endpoints, such as improvement in itch, percent of mEASI-50 responders, investigator’s global assessment responder analysis, and reduction in body surface area impacted by disease. In addition, the FAS analysis also showed positive trends in favor of ATI-1777 in percent of mEASI-75 responders (65.2% for ATI-1777 compared to 24.0% for vehicle) and mEASI-90 responders (30.4% for ATI-1777 compared to 20.0% for vehicle). These secondary efficacy endpoints were not powered for statistical significance. Based on an analysis of pharmacokinetic plasma samples in the ATI-1777 arm at multiple timepoints, minimal systemic exposure was observed which supports a “soft” topical JAK inhibitor approach.
ATI-1777 was generally well tolerated. No serious adverse events were reported. The most common adverse events (reported in at least 2 subjects in the trial) were increased blood CPK levels and headache in subjects in the ATI-1777 arm and urinary tract infection (one in each of the ATI-1777 and the vehicle arm); none of these adverse events in the ATI-1777 arm were determined by the clinical trial investigators to be related to ATI-1777. One treatment-related adverse event, application site pruritus, was reported in one subject in the ATI-1777 arm.
We plan to submit the final trial results for publication in a peer-reviewed scientific journal.
Based on the results observed in the Phase 2a trial, we intend to progress ATI-1777 into a Phase 2b trial in moderate to severe atopic dermatitis in the first half of 2022. In this trial, we plan to explore multiple concentrations of twice daily treatment with ATI-1777 and a single concentration of once daily treatment with ATI-1777.
Preclinical Programs
ATI-2138, an Investigational ITJ Inhibitor
We are 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 submitted an IND for ATI-2138 for the treatment of psoriasis in October 2021. If allowed, we plan to progress to a first-in-human Phase 1 single ascending dose trial of ATI-2138 in healthy volunteers.
ATI-2231, an Investigational Oral MK2 Inhibitor
We are exploring the use of ATI-2231, an investigational oral MK2 inhibitor compound designed to have a long half-life, as a potential treatment for pancreatic cancer and metastatic breast cancer as well as in preventing bone loss in patients with metastatic breast cancer. We are currently conducting IND-enabling studies.
Discovery Programs
We are developing oral gut-restricted JAK inhibitors with limited systemic exposure as potential treatments for inflammatory bowel disease. In addition, we are engaged in research to identify brain penetrant kinase inhibitor candidates as potential treatments for neurodegenerative diseases.
22
Table of Contents
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 $68.1 million for the nine months ended September 30, 2021 and $51.0 million for the year ended December 31, 2020. As of September 30, 2021, we had an accumulated deficit of $572.6 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 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. We also expect to add additional personnel to support our expanding and advancing development pipeline. 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.
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.
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 zunsemetinib as a potential treatment for moderate to severe rheumatoid arthritis and ATI-1777 as a potential treatment for moderate to severe atopic dermatitis and the supply of active pharmaceutical ingredients and drug product for our clinical trials. 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 spread of the disease, the duration of the pandemic, 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
23
Table of Contents
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 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.
Components of Our Results of Operations
Revenue
Contract Research
We earn revenue from the provision of laboratory services. 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.
Other Revenue
Other revenue primarily consists of royalties earned on net sales of RHOFADE pursuant to the asset purchase agreement with EPI Health described above.
24
Table of Contents
Cost of Revenue
Cost of revenue consists of the costs incurred in connection with the provision of contract research services. 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; and
● laboratory materials and supplies used to support our research activities.
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 zunsemetinib as a potential treatment for moderate to severe rheumatoid arthritis and 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, clinical trial 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 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;
25
Table of Contents
● 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 and travel expenses.
Revaluation of Contingent Consideration
Revaluation of contingent consideration consists of changes in the fair value of our contingent consideration liability between reporting dates.
Other Expense, Net
Other 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
This discussion and analysis of our financial condition and results of operations is based on our condensed consolidated financial statements, which have been prepared in accordance with generally accepted accounting principles in the United States. The preparation of these financial statements requires us to make estimates and judgments that affect the reported amounts of assets, liabilities, revenues and expenses and the disclosure of contingent assets and liabilities in our condensed consolidated financial statements. 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 or conditions. Except as described below, there have been no material changes to our significant accounting policies and use of estimates as disclosed in the footnotes to our audited consolidated financial statements for the year ended December 31, 2020 included in our Annual Report on Form 10-K filed with the SEC on February 25, 2021.
Contingent Consideration
We initially recorded a contingent consideration liability at fair value on the date of acquisition related to future potential payments resulting from the acquisition of Confluence based upon significant unobservable inputs including the achievement of development, regulatory and commercial milestones, as well as estimated future sales levels and the discount rates applied to calculate the present value of the potential payments. Significant judgement was involved in determining the appropriateness of these assumptions. These assumptions are considered Level 3 inputs. Revaluation of our contingent consideration liability can result from changes to one or more of these assumptions. We evaluate the fair value estimate of our contingent consideration liability on a quarterly basis with changes, if any, recorded as income or
26
Table of Contents
expense in our condensed consolidated statement of operations. Any such changes could have a material impact on our financial results.
The fair value of contingent consideration is estimated using a probability-weighted expected payment model for regulatory milestone payments and a Monte Carlo simulation model for commercial milestone and royalty payments and then applying a risk-adjusted discount rate to calculate the present value of the potential payments. Significant assumptions used in our estimates include the probability of achieving regulatory milestones and commencing commercialization, which are based on an asset’s current stage of development and a review of existing clinical data. Probability of success assumptions ranged between 4% and 40%. Additionally, estimated future sales levels and the risk-adjusted discount rate applied to the potential payments are also significant assumptions used in calculating the fair value. The discount rate ranged between 5.9% and 7.8% depending on the year of each potential payment.
During the nine months ended September 30, 2021, we updated assumptions for probability of success and estimated future sales levels as a result of the completion of a Phase 2a clinical trial of zunsemetinib in subjects with moderate to severe rheumatoid arthritis and as a result of the completion of a Phase 2a clinical trial of ATI-1777 in subjects with moderate to severe atopic dermatitis. We also included estimated future sales of zunsemetinib as a potential treatment for psoriatic arthritis and moderate to severe hidradenitis suppurativa, which are additional planned indications for zunsemetinib. These updates resulted in a charge of $22.1 million.
Results of Operations
Comparison of Three and Nine Months Ended September 30, 2021 and 2020
Three Months Ended September 30,
Nine Months Ended September 30,
(In thousands)
2021
2020
Change
2021
2020
Change
Revenues:
Contract research
$
1,415
$
1,331
$
84
$
4,556
$
4,373
$
183
Other revenue
244
118
126
704
529
175
Total revenue
1,659
1,449
210
5,260
4,902
358
Costs and expenses:
Cost of revenue
1,099
1,189
(90)
3,564
3,847
(283)
Research and development
13,976
6,240
7,736
29,711
20,382
9,329
General and administrative
5,979
3,859
2,120
16,676
15,632
1,044
Revaluation of contingent consideration
900
626
274
22,139
2,393
19,746
Total costs and expenses
21,954
11,914
10,040
72,090
42,254
29,836
Loss from operations
(20,295)
(10,465)
(9,830)
(66,830)
(37,352)
(29,478)
Other expense, net
(851)
(194)
(657)
(1,231)
(205)
(1,026)
Loss from continuing operations
(21,146)
(10,659)
(10,487)
(68,061)
(37,557)
(30,504)
Loss from discontinued operations
—
—
—
—
(285)
285
Net loss
$
(21,146)
$
(10,659)
$
(10,487)
$
(68,061)
$
(37,842)
$
(30,219)
Revenue
Contract research revenue was $1.4 million and $1.3 million for the three months ended September 30, 2021 and 2020, respectively, and was comprised of fees earned from the provision of laboratory services. The $0.1 million increase was driven by a higher average bill rate offset by a reduction in overall hours billed. Other revenue for the three months ended September 30, 2021 and 2020 primarily consisted of $0.2 million and $0.1 million of royalties earned on net sales of RHOFADE, respectively.
27
Table of Contents
Contract research revenue was $4.6 million and $4.4 million for the nine months ended September 30, 2021 and 2020, respectively, and was comprised of fees earned from the provision of laboratory services. The $0.2 million increase was driven by a higher average bill rate. Other revenue for the nine months ended September 30, 2021 and 2020 primarily consisted of $0.6 million and $0.5 million of royalties earned on net sales of RHOFADE, respectively.
Cost of Revenue
Cost of revenue was $1.1 million and $3.6 million for the three and nine months ended September 30, 2021, respectively, and $1.2 million and $3.8 million for the three and nine months ended September 30, 2020, respectively, and in each case related to providing laboratory services to our customers. The decrease in cost of revenue in the three and nine months ended September 30, 2021 compared to the three and nine months ended September 30, 2020 was primarily the result of COVID-19 employee-retention tax credits. Changes in cost of revenue generally correlate to changes in contract research revenue.
Research and Development Expenses
The following table summarizes our research and development expenses by drug candidate or, for unallocated expenses, by type:
Three Months Ended
Nine Months Ended
September 30,
September 30,
(In thousands)
2021
2020
Change
2021
2020
Change
Zunsemetinib
$
8,207
2,216
$
5,991
$
12,532
4,925
$
7,607
ATI-1777
450
694
(244)
1,894
2,553
(659)
ATI-2138
980
599
381
3,330
1,753
1,577
ATI-2231
263
—
263
333
—
333
Discovery
935
455
480
2,401
1,700
701
Other research and development
469
398
71
1,268
2,068
(800)
Personnel
1,733
1,441
292
4,984
5,191
(207)
Stock-based compensation
939
437
502
2,969
2,192
777
Total research and development expenses
$
13,976
$
6,240
$
7,736
$
29,711
$
20,382
$
9,329
Zunsemetinib
The increase in expenses for zunsemetinib during the three and nine months ended September 30, 2021 compared to the three and nine months ended September 30, 2020 was primarily due to costs associated with drug product manufacturing and clinical development activities for a Phase 2b trial in subjects with moderate to severe rheumatoid arthritis and a Phase 2 trial in subjects with moderate to severe hidradenitis suppurativa .
ATI-1777
The decrease in expenses for ATI-1777 during the three months ended September 30, 2021 compared to the three months ended September 30, 2020 was primarily due to lower costs associated with a Phase 2a clinical trial in subjects with moderate to severe atopic dermatitis, which concluded in the second quarter 2021.
The decrease in expenses for ATI-1777 during the nine months ended September 30, 2021 compared to the nine months ended September 30, 2020 was primarily due to a decrease in development costs, including toxicology studies, partially offset by an increase in costs associated with a Phase 2a clinical trial in subjects with moderate to severe atopic dermatitis, which began in the third quarter of 2020 and concluded in the second quarter of 2021.
ATI-2138
Expenses for ATI-2138 were higher during the three and nine months ended September 30, 2021 compared to the three and nine months ended September 30, 2020 primarily due to preclinical development activities and IND-enabling studies as we progressed towards an IND submission.
28
Table of Contents
ATI-2231
Expenses for ATI-2231 were higher during the three and nine months ended September 30, 2021 compared to the three and nine months ended September 30, 2020 primarily due to preclinical development activities and IND-enabling studies.
Discovery and other research and development
Expenses related to discovery increased during the three and nine months ended September 30, 2021 compared to the three and nine months ended September 30, 2020 due to continued investment in our discovery-stage programs.
Other research and development expenses, which primarily include expenses for our legacy dermatology assets and medical affairs activities, were lower during the nine months ended September 30, 2021 compared to September 30, 2020 due to a decrease in costs for our legacy dermatology assets following the decision to discontinue investment in those programs.
Personnel and stock-based compensation
Compensation related expenses increased during the three months ended September 30, 2021 compared to the three months ended September 30, 2020 primarily due to an increase in stock-based compensation expense associated with new equity awards granted in 2021 as well as higher average headcount.
Compensation related expenses increased during the nine months ended September 30, 2021 compared to the nine months ended September 30, 2020 primarily due to an increase in stock-based compensation expense associated with new equity awards granted in 2021, partially offset by lower average headcount and lower payroll taxes resulting from COVID-19 employee-retention tax credits taken during the nine months ended September 30, 2021.
General and Administrative Expenses
The following table summarizes our general and administrative expenses:
Three Months Ended
Nine Months Ended
September 30,
September 30,
(In thousands)
2021
2020
Change
2021
2020
Change
Personnel
$
1,099
$
861
$
238
$
3,205
$
4,130
$
(925)
Professional and legal fees
1,207
907
300
3,898
2,857
1,041
Facility and support services
557
362
195
1,369
1,294
75
Other general and administrative
559
441
118
1,751
1,568
183
Stock-based compensation
2,557
1,288
1,269
6,453
5,783
670
Total general and administrative expenses
$
5,979
$
3,859
$
2,120
$
16,676
$
15,632
$
1,044
Personnel and stock-based compensation
Compensation related expenses increased during the three months ended September 30, 2021 compared to the three months ended September 30, 2020 primarily due to an increase in stock-based compensation expense associated with new equity awards granted in 2021 and higher incentive compensation-related accruals.
Compensation related expenses decreased during the nine months ended September 30, 2021 compared to the nine months ended September 30, 2020 primarily due to lower average headcount partially offset by an increase in stock-based compensation expense associated with new equity awards granted in 2021.
29
Table of Contents
Professional and legal fees
Professional and legal fees, including accounting, investor relations and corporate communication costs, were higher during the three and nine months ended September 30, 2021 compared to the three and nine months ended September 30, 2020 primarily as a result of increased costs associated with Sarbanes-Oxley compliance and other professional fees for temporary staffing.
Facility and support services and other general and administrative
Facility and support services, including general office expenses, information technology costs and other expenses, increased during the three and nine months ended September 30, 2021 compared to the three and nine months ended September 30, 2020 primarily due to an increase in information technology costs resulting from higher headcount and infrastructure technology improvements.
Other general and administrative expenses increased during the three and nine months ended September 30, 2021 compared to the three and nine months ended September 30, 2020 primarily due to an increase in insurance premiums resulting from additional coverage in 2021 as compared to the prior year.
Revaluation of Contingent Consideration
The increase in revaluation of contingent consideration during the three months ended September 30, 2021 compared to the three months ended September 30, 2020 resulted from updates to the probability of success and estimated future sales level assumptions as a result of the completion of a Phase 2a clinical trial of ATI-1777 in subjects with moderate to severe atopic dermatitis.
The increase in revaluation of contingent consideration during the nine months ended September 30, 2021 compared to the nine months ended September 30, 2020 resulted from updates to the probability of success and estimated future sales level assumptions as a result of the completion of a Phase 2a clinical trial of zunsemetinib in subjects with moderate to severe rheumatoid arthritis, as well as the completion of a Phase 2a clinical trial of ATI-1777 in subjects with moderate to severe atopic dermatitis. Additionally, the inclusion of estimated future sales of zunsemetinib as a potential treatment for psoriatic arthritis and moderate to severe hidradenitis suppurativa, which are additional planned indications for zunsemetinib, also contributed to the increase.
Other Expense, net
Other expense, net increased during the three months ended September 30, 2021 compared to the three months ended September 30, 2020 primarily due to interest and fees associated with a payoff of the Silicon Valley Bank Loan and Security Agreement.
Other expense, net increased during the nine months ended September 30, 2021 compared to the nine months ended September 30, 2020 primarily due to interest and fees associated with a payoff of the Silicon Valley Bank Loan and Security Agreement and lower interest income.
30
Table of Contents
Liquidity and Capital Resources
Overview
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 and incurring indebtedness in the form of loans from commercial lenders. We may engage in additional debt and equity financing transactions in order to raise 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 September 30, 2021, we had cash, cash equivalents and marketable securities of $243.6 million. 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 contingent obligations under the Confluence Agreement, which is summarized above under “Overview—Acquisition and License Agreements, ” and our lease obligations.
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 also incurred expenses of $0.4 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 $103.3 million.
June 2021 Public Offering
In June 2021, we closed a public offering in which we sold 8,098,592 shares of common stock at a price to the public of $17.75 per share, for aggregate gross proceeds of $143.8 million. We paid underwriting discounts and commissions of $8.6 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 $134.9 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 Capital Fund, LLC, or Lincoln Park, which 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. U pon 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 terminated the Purchase Agreement in January 2021 in connection with the public offering of common stock described above. We did not sell any additional shares prior to terminating the Purchase Agreement.
31
Table of Contents
Debt Financing
Loan and Security Agreement with Silicon Valley Bank
In March 2020, we entered into a Loan and Security Agreement with Silicon Valley Bank, or SVB. The Loan and Security Agreement provided for $11.0 million in term loans, of which we borrowed the entire amount on March 30, 2020. In July 2021, we repaid in full the $11.0 million that was outstanding under the Loan and Security Agreement, together with all accrued and unpaid interest and fees as of the payoff date, for a total payment of $11.7 million.
Liquidity and Cash Flows
Cash and cash equivalents were $53.6 million as of September 30, 2021 compared to $22.1 million as of December 31, 2020. We also had $190.0 million in short- and long-term marketable securities as of September 30, 2021 compared to $32.1 million as of December 31, 2020.
The sources and uses of cash that contributed to the change in cash and cash equivalents were:
Nine Months Ended
September 30,
(In thousands)
2021
2020
Cash and cash equivalents beginning balance
$
22,063
$
35,937
Net cash used in operating activities
(35,059)
(29,776)
Net cash provided by (used in) investing activities
(158,456)
8,692
Net cash provided by financing activities
225,054
10,543
Cash and cash equivalents ending balance
$
53,602
$
25,396
Operating Activities
Operating activities use of cash was the result of:
Nine Months Ended
September 30,
(In thousands)
2021
2020
Net loss
$
(68,061)
$
(37,842)
Non-cash adjustments to reconcile net loss to net cash used in operating activities
33,826
12,869
Change in accounts payable and accrued expenses
(777)
(10,889)
Change in accounts receivable
(39)
4,783
Change in prepaid expenses and other assets
(8)
1,303
Net cash used in operating activities
$
(35,059)
$
(29,776)
Net cash used in operating activities increased for the nine months ended September 30, 2021 compared to the nine months ended September 30, 2020 primarily as a result of higher net losses after adjusting for non-cash items, an increase in cash paid for prepaid expenses, and a reduction of cash collected from outstanding accounts receivable . The increase was partially offset by a decrease in cash paid to settle outstanding accounts payable balances.
The change in prepaid expenses and other assets was the result of higher prepaid research and development balances relative to the prior year period primarily associated with drug product manufacturing, clinical trials and other preclinical development activities. The change in accounts payable and accrued expenses was primarily driven by the timing of receipt and payment of invoices around quarter-end relative to the prior-year period. The change in accounts receivable was primarily the result of cash received during the nine months ended September 30, 2020 from Allergan Sales, LLC related to sales of RHOFADE that occurred after the date we sold RHOFADE to EPI Health.
32
Table of Contents
Investing Activities
Cash flow from investing activities was the result of:
Nine Months Ended
September 30,
(In thousands)
2021
2020
Purchases of property and equipment
$
(108)
$
(445)
Purchases of marketable securities
(199,862)
(39,898)
Proceeds from sales and maturities of marketable securities
41,514
49,035
Net cash provided by (used in) investing activities
$
(158,456)
$
8,692
The change in net cash used in investing activities for the nine months ended September 30, 2021 compared to net cash provided by investing activities for the nine months ended September 30, 2020 primarily resulted from purchases of marketable securities following our January and June 2021 public offerings.
Financing Activities
Financing activities use of cash was the result of:
Nine Months Ended
September 30,
(In thousands)
2021
2020
Proceeds from issuance of common stock in connection with public offerings, net of issuance costs
$
238,200
$
—
Proceeds from debt financing (including warrants), net of issuance costs
—
10,913
Repayment of debt
(11,483)
—
Restricted stock unit employee tax withholdings
(3,122)
—
Finance lease payments
—
(128)
Deferred issuance costs
—
(242)
Proceeds from exercise of employee stock options and the issuance of stock
1,459
—
Net cash provided by financing activities
$
225,054
$
10,543
Cash provided by financing activities increased for the nine months ended September 30, 2021 compared to September 30, 2020 primarily due to our January 2021 and June 2021 public offerings. The increase was partially offset by a decrease in proceeds from debt financing, an increase in debt repayments, and an increase in cash used for tax withholdings in connection with the vesting of restricted stock units.
Funding Requirements
We anticipate we will incur net losses in the near term as we continue the clinical development of zunsemetinib as a potential treatment for moderate to severe rheumatoid arthritis and 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.
33
Table of Contents
As a publicly traded company, we incur and will continue to incur significant legal, accounting and other similar 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 that could increase our compliance costs.
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 condensed consolidated financial statements that appear in Item 1 of this Quarterly Report on Form 10-Q based on our current operating assumptions. We will require additional capital to complete the clinical development of zunsemetinib 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.
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 concurrently 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. In July 2021, we repaid in full the $11.0 million that was outstanding under the Loan and Security Agreement, together with all accrued and unpaid interest and fees as of the payoff date, for a total payment of $11.7 million.
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.
34
Table of Contents
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.
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.
Text extracted from the filing as submitted to EDGAR. Formatting, tables and exhibits are simplified for reading; the original document is authoritative for anything you rely on.