On February 25, 2021 ADC Therapeutics SA (NYSE:ADCT), a late clinical-stage oncology-focused biotechnology company pioneering the development and commercialization of highly potent and targeted antibody drug conjugates (ADCs) for patients with hematological malignancies and solid tumors, reported that Chris Martin, Chief Executive Officer, will participate in a fireside chat at the Cowen 41st Annual Health Care Conference on Thursday, March 4, 2021, at 12:50 p.m. ET (Press release, ADC Therapeutics, FEB 25, 2021, View Source [SID1234575676]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

A live webcast of the fireside chat will be available via the Events & Presentations page in the Investors section of ADC Therapeutics’ website, ir.adctherapeutics.com. A replay of the webcast will be available for approximately 30 days.

On February 25, 2021 Radius Health, Inc. ("Radius" or the "Company") (Nasdaq: RDUS), reported its financial results for the fourth quarter ended December 31, 2020 as well as full year 2020 results (Press release, Radius, FEB 25, 2021, View Source [SID1234575675]). In addition, a number of brief business updates are included.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Q4 2020 FINANCIAL AND BUSINESS HIGHLIGHTS:

Q4 2020 U.S. net sales of TYMLOS were $60 million, an 8% year-over-year increase vs. Q4 2019
TYMLOS U.S. new patient starts increased by 26% in Q4 2020 vs. Q3 2020
FY 2020 FINANCIAL AND BUSINESS HIGHLIGHTS:

FY 2020 U.S. TYMLOS net product revenue was $208 million vs. FY 2019 net product revenue of $173 million – year-over-year growth of 20%
Balance sheet: approx. $115 million of cash, cash equivalents, and marketable securities at year end
Reduced actual total company headcount by 25+% vs. full year 2020 budget
Reduced real estate footprint and infrastructure by approximately 50%
Pivoted U.S. commercial effort to focus on post-menopausal high risk fracture patients
Increased U.S. commercial productivity (net rev. per sales person) by approximately 50%
Completed elacestrant business development out-licensing transaction with the Menarini Group
Completed RAD140 business development out-licensing transaction with Ellipses Pharma Limited
Completed abaloparatide business development out-licensing transction with Paladin Labs in Canada
Completed RAD011 business development acquisition transaction for global rights
OTHER BUSINESS HIGHLIGHTS:

Abaloparatide

ATOM phase 3 study, evaluating abaloparatide for use in osteoporotic men at high risk for fracture, remains on track for 2H 2021 read-out
wearABLe phase 3 study, evaluating the effects on bone mineral density of abaloparatide delivered via a novel transdermal system, remains on track for 2H 2021 read-out
In Japan, our partner Teijin continues to make progress within the Japan regulatory and market processes
Our partner in Canada – Paladin Labs – is advancing with plans to progress the abaloparatide molecule through the regulatory process
In the EU, we continue to advance meetings and dialogue regarding a possible re-submission of abaloparatide
RAD011

A dedicated and experienced team has been created to advance the molecule and asset
Our initial focus: Prader Willi Syndrome (PWS) for the U.S. and other global markets
Assessing additional product pathways with focus on other orphan metabolic, endocrine indications
Elacestrant

EMERALD phase 3 Study, with partner Menarini Group, remains on track for a 2H 2021 read-out
Fourth Quarter 2020 Financial Results

Three Months Ended December 31, 2020

Net Loss
For the three months ended December 31, 2020, Radius reported a net loss of $21.4 million, or $0.46 per share, compared to a net loss of $24.7 million, or $0.54 per share, for the three months ended December 31, 2019.

For the three months ended December 31, 2020, non-GAAP adjusted net income, was $10.8 million, or $0.23 per share, compared to non-GAAP adjusted net loss of $13.3 million, or $0.29 per share, for the three months ended December 31, 2019.

Revenue
For the three months ended December 31, 2020, TYMLOS net product revenues were $59.9 million compared to approximately $55.7 million for the three months ended December 31, 2019.

Costs and Expenses
For the three months ended December 31, 2020, research and development expense was $36.4 million compared to $34.5 million for the three months ended December 31, 2019, an increase of $1.9 million, or 5%. This increase was primarily driven by a $16.0 million increase in RAD011 program expenses, a $3.4 million increase in abaloparatide transdermal system program costs, which was partially offset by a $18.4 million decrease in elacestrant program costs.

Twelve Months Ended December 31, 2020

Net Loss
For the twelve months ended December 31, 2020, Radius reported a net loss of $109.2 million, or $2.35 per share, compared to a net loss of $133.0 million, or $2.89 per share, for the twelve months ended December 31, 2019.

For the twelve months ended December 31, 2020, non-GAAP adjusted net loss was $62.3 million, or $1.34 per share, compared to non-GAAP adjusted net loss of $90.9 million, or $1.98 per share, for the twelve months ended December 31, 2019.

Revenue
For the twelve months ended December 31, 2020, TYMLOS net product revenues were $208.4 million compared to approximately $173.3 million for the twelve months ended December 31, 2019.

Costs and Expenses
For the twelve months ended December 31, 2020, research and development expense was $159.7 million compared to $116.8 million for the twelve months ended December 31, 2019, an increase of $43.0 million, or 37%. This increase was primarily a result of an increase of $39.9 million in program spending for the abaloparatide transdermal system project costs and an increase of $16.0 million in other costs due to the Benuvia licensing expense. These increases were partially offset by a $11.9 million decrease in program spending for elacestrant research which is a result of $39.3 million of reimbursable expenses offsetting year to date expenses.

For the twelve months ended December 31, 2020, selling, general and administrative expenses were $144.2 million compared to $152.7 million for the twelve months ended December 31, 2019, a decrease of $8.6 million, or 6%. This decrease was primarily the result of a $6.3 million decrease in professional fees, a $4.0 million decrease compensation related expenses. These decreases were offset by a $1.7 million increase in other costs.

As of December 31, 2020, Radius had $115.3 million in cash, cash equivalents, restricted cash, and marketable securities. Based upon our cash, cash equivalents and marketable securities balance as of December 31, 2020, we believe that, prior to the consideration of potential proceeds from partnering and/or collaboration activities, we have sufficient capital to fund our development plans, U.S. commercial and other operational activities for at least twelve months from the date of this press release.

Webcast and Conference Call

In connection with today’s reporting of Fourth Quarter 2020 Financial Results, Radius will host a conference call and live audio webcast at 8:30 a.m. ET today, February 25, 2021, to review the commercial, research and development, and financial highlights and provide a Company update.

A live audio webcast of the call can be accessed from the Investors section of the Company’s website, www.radiuspharm.com. The full text of the announcement and financial results will also be available on the Company’s website.

For those unable to participate in the conference call or webcast, a replay will be available on Thursday, February 25th at 11:30 a.m. ET and will be archived on the Company’s website for 90 days. To access the replay, dial (855) 859-2056 for U.S. or (404) 537-3406 for International, using conference ID number 8266586.

On February 25, 2021 Compugen Ltd. (Nasdaq: CGEN), a clinical-stage cancer immunotherapy company and a leader in predictive target discovery, reported data from its Phase 1 dose escalation and expansion study of COM701 as a monotherapy, and in a dose escalation combination study with Opdivo (nivolumab) (Press release, Compugen, FEB 25, 2021, View Source [SID1234575674]). COM701 is a first-in-class investigational therapeutic antibody targeting PVRIG, a novel immune checkpoint discovered computationally by Compugen.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"The data generated to date across our COM701 clinical program suggest that PVRIG may be an important immune checkpoint in patients who are unresponsive or refractory to currently available immunotherapies," said Anat Cohen-Dayag, Ph.D., President and CEO of Compugen. "We are highly encouraged by our updated results from the COM701 plus Opdivo combination dose escalation, which now include a confirmed complete response in a patient with prior progression on Opdivo and a previously reported patient with a durable confirmed partial response for almost a year. Combined with a disease control rate of 66.7% and ongoing durable signals of activity beyond or approaching one year in multiple patients and across indications, these results leave us increasingly confident that dual blockade of PVRIG and PD-1 may be key to driving anti-tumor immune responses in certain patient populations. Based on these encouraging results, we will be further evaluating this dual combination regimen in patients with ovarian, breast, endometrial and microsatellite-stable colorectal cancers with the initiation of the COM701 and Opdivo cohort expansion study in the second quarter of 2021, as part of our collaboration with Bristol Myers Squibb."

Dr. Cohen-Dayag continued, "Our monotherapy cohort expansion study was an important milestone in our COM701 monotherapy evaluation. This data, together with data from our previously reported dose escalation study, which includes a confirmed partial response with treatment ongoing for over one year as of our new data cutoff date, demonstrate durable signals of antitumor activity in tumor types typically unresponsive to immune checkpoint inhibitors, including patients with prior progression on these treatments. We will leverage these data, along with future data from our ongoing correlative assessments of biological samples from patients, to inform our clinical approach and next steps as we execute across our broad combination strategy, which includes dual and triple blockade regimens of COM701 with TIGIT and PD-1. Importantly, as the only company with wholly owned clinical candidates targeting both PVRIG and TIGIT, we are uniquely capable and on track to conduct this comprehensive evaluation of the synergistic blockade of the DNAM axis with PD-1, and we look forward to continued progress in potentially expanding the reach of immunotherapy."

Data highlights from the Phase 1 dose escalation studies as of the data cutoff of December 14, 2020 include:

COM701 and Opdivo combination dose escalation arm:

In 15 patients with a median of five prior anticancer therapies (range of 2-10), COM701 in combination with Opdivo was well-tolerated with no reported dose-limiting toxicities up to the fifth and final dose cohort of COM701 20 mg/kg and Opdivo 480 mg, both IV Q4 weeks.
The disease control rate (DCR) was 66.7% (N=10) with best responses of complete response (CR) 6.7% (N=1), partial response (PR) 6.7% (N=1) and stable disease (SD) 53.3% (N=8).
A patient with anal squamous cell carcinoma with confirmed SD as reported at American Association for Cancer Research (AACR) (Free AACR Whitepaper) 2020, now with confirmed CR and remains on treatment at 79 weeks. This patient progressed on Opdivo prior to enrolling in our study.
A patient with microsatellite stable (MSS)-colorectal cancer with durable confirmed partial response previously reported at AACR (Free AACR Whitepaper) 2020 remained on study treatment at 44 weeks.
Durable responses of confirmed SD of six months or more in three patients. One patient with renal cell carcinoma remains on treatment at 58 weeks, and one patient with non-small cell lung cancer (NSCLC) (squamous) who failed prior treatment with immune checkpoint inhibitors remained on treatment at 36 weeks, and one patient with endometrial cancer remained on treatment at 46 weeks.
COM701 monotherapy arm dose escalation update since AACR (Free AACR Whitepaper) 2020:

The patient with primary peritoneal cancer (platinum resistant, MSS) with durable confirmed partial response remains on study treatment at 62 weeks.
The patient with pancreatic cancer, refractory to all three prior lines of standard of care (SOC) therapy with durable confirmed SD was on study treatment for 31 weeks.
Data highlights from the monotherapy expansion cohort as of the data cutoff of December 14, 2020 include:

20 patients enrolled in biomarker and data informed indications; four patients of each: endometrial cancer, NSCLC, ovarian cancer, breast cancer and colorectal cancer.
Six of the 20 patients (30%) had best responses of SD, one patient with endometrial cancer, three patients with NSCLC and two patients with ovarian cancer.
Two patients with SD remain on treatment as of the data cutoff date; one patient with NSCLC who had >3 prior lines of SOC therapy; including prior treatment with immune checkpoint inhibitors with treatment ongoing at 26 weeks, and one patient with ovarian cancer with treatment ongoing at 20 weeks.
Two additional patients remain on treatment as of the data cutoff date.
No new safety findings were observed.
Additional clinical data and initial correlative assessments of biological samples from patients are planned to be presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2021 annual meeting, to which an abstract was submitted.

Opdivo is a registered trademark of Bristol Myers Squibb.

On February 25, 2021 Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat cancer and genetically defined diseases, reported business highlights and financial results for the fourth quarter and year ended December 31, 2020 (Press release, Agios Pharmaceuticals, FEB 25, 2021, View Source [SID1234575673]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"This past year was a transformative one for Agios," said Jackie Fouse, Ph.D., chief executive officer at Agios. "Despite the extraordinary challenges brought on by the global COVID-19 pandemic and civil and political unrest in the U.S., we remain hopeful for the future of our country and the promise of our industry and more confident than ever in our ability to execute on our plans on behalf of patients. The sale of our oncology business to Servier on attractive terms both allows our oncology portfolio to grow and flourish and facilitates our new laser focus on genetically defined diseases, where we anticipate a catalyst-rich year ahead for mitapivat across our three initial disease indications. In addition to our plans to file for approval for mitapivat in adults with PK deficiency in the U.S. and EU and launching our Phase 3 thalassemia trials later this year, we’re pleased to share our pivotal strategy for mitapivat in sickle cell disease which we believe will enable us to pursue a broad label for patients who desperately need new treatment options."

FOURTH QUARTER 2020 & RECENT HIGHLIGHTS

Entered into definitive agreement to sell commercial, clinical and research-stage oncology portfolio to Servier, an independent global pharmaceutical company, in December.
Achieved TIBSOVO (ivosidenib tablets) net sales of $39.1 million for the quarter and $121.1 million for the year, exceeding the $115 million updated net revenue target and representing a 102% increase in net sales year-over-year.
Announced topline results from two Phase 3 studies of mitapivat in adults with pyruvate kinase (PK) deficiency who were not regularly transfused (ACTIVATE) and who were regularly transfused (ACTIVATE-T ). Both studies achieved statistical significance for their primary endpoint with ACTIVATE demonstrating a clinically meaningful sustained increase in hemoglobin compared to placebo and ACTIVATE-T demonstrating a clinically meaningful reduction in transfusion burden. Statistical significance was also achieved for all pre-specified key secondary endpoints for ACTIVATE demonstrating an improvement compared to placebo, including in patient-reported outcomes (PRO) based on changes from baseline in pyruvate kinase deficiency diary (PKDD) score and pyruvate kinase deficiency impact assessment (PKDIA) score at Week 24. The safety profile was consistent with results from prior studies.
Completed U.S. and EU regulatory interactions on the pivotal development plan for mitapivat in sickle cell disease, resulting in the Phase 2/3 trial design being announced today.
The Phase 2 will randomize 69 patients 1:1:1 to 50 mg mitapivat BID, 100 mg mitapivat BID or matched placebo. The primary endpoint is hemoglobin response defined as ≥1 g/dL change from baseline to Week 12.
The Phase 3 will randomize 198 patients 2:1 to the selected Phase 2 dose of mitapivat or matched placebo. The study includes two primary endpoints: hemoglobin response defined as ≥1 g/dL change from baseline to Week 52 and annualized rate of sickle cell pain crises.
Potential regulatory approval with a broad label based on the operationally seamless Phase 2/3 trial expected by the end of 2026.
Appointed Darrin Miles, previously senior vice president, U.S. commercial and global marketing, to role of chief commercial officer.
Presented final data, including mature overall survival (OS) results, from the Phase 3 ClarIDHy study of TIBSOVO in patients with previously treated isocitrate dehydrogenase 1 (IDH1) mutated cholangiocarcinoma at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Gastrointestinal Cancers Symposium (ASCO-GI) in January.
Presented updated Phase 1 data for mitapivat in sickle cell disease at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December.
Launched Anemia ID, a program providing no-cost genetic testing for patients with suspected hereditary anemias, including PK deficiency in November.
ANTICIPATED 2021 KEY MILESTONES

Corporate

Complete sale of oncology portfolio to Servier in a transaction worth up to $2 billion plus royalties, following a shareholder vote on March 25, and execute a meaningful portion of the planned $1.2 billion capital return by year-end.
Genetically Defined Diseases

File for regulatory approval for mitapivat in adults with PK deficiency: submit new drug application (NDA) in the U.S. in the second quarter of 2021 and marketing authorization application (MAA) in the EU in mid-2021.
Initiate two Phase 3 studies of mitapivat, ENERGIZE and ENERGIZE-T, in not regularly transfused and regularly transfused adults with thalassemia in the second half of 2021.
Initiate Phase 2/3 study of mitapivat in sickle cell disease by year-end.
Prioritize new indications for pyruvate kinase R (PKR) and pyruvate kinase M2 (PKM2) activator clinical development by year-end.
Genetically Defined Disease Data Presentations

Submit data from the following clinical studies for presentation at the EHA (Free EHA Whitepaper) Virtual Congress, hosted June 9-17, 2021:
Phase 3 ACTIVATE study of mitapivat in adults with PK deficiency who do not receive regular transfusions
Phase 3 ACTIVATE-T study of mitapivat in adults with PK deficiency who receive regular transfusions
Phase 2 study of mitapivat in adults with α- and β-thalassemia who do not receive regular transfusions
Submit data from ongoing clinical studies of mitapivat in sickle cell disease for presentation at medical meetings throughout 2021.
Present data from the single ascending dose (SAD) and multiple ascending dose (MAD) cohorts of the Phase 1 study of AG-946, the company’s next-generation PKR activator, in healthy volunteers by year-end.
Oncology

Submit supplemental new drug application (sNDA) in the U.S. for TIBSOVO in patients with previously treated IDH1-mutant cholangiocarcinoma in Q1 2021.
Enrollment in the Phase 3 AGILE trial of TIBSOVO in combination with azacitidine in adult patients with previously untreated IDH1-mutant acute myeloid leukemia is expected to complete by year-end.
Enrollment in the relapsed or refractory myelodysplastic syndrome arm of the TIBSOVO Phase 1 study of IDH1-mutant advanced hematologic malignancies is expected to complete by year-end.
Full-year 2021 net product revenue for TIBSOVO is expected to be $160-170 million.
FOURTH QUARTER AND FULL YEAR 2020 FINANCIAL RESULTS

Revenue: Total revenue for the fourth quarter of 2020 was $44.0 million, which includes $2.0 million in collaboration revenue, $39.1 million of net product revenue from sales of TIBSOVO and $2.9 million in royalty revenue from net global sales of IDHIFA under our collaboration agreement with Celgene. This compares to $35.4 million for the fourth quarter of 2019, which included $12.9 million in collaboration revenue, $19.6 million of net product revenue from U.S. sales of TIBSOVO and $3.0 million in royalty revenue from net global sales of IDHIFA. Total revenue for the year ended December 31, 2020 was $203.2 million compared to $117.9 million for the year ended December 31, 2019. The increase in 2020 revenue was primarily driven by a 102% increase in TIBSOVO net product revenue and higher collaboration revenue due to recognition of the remainder of the deferred revenue balance related to the completion of the metabolic immuno-oncology collaboration with Celgene Corporation, a wholly owned subsidiary of Bristol Myers Squibb Company.

Cost of Sales: Cost of sales were $1.0 million for the fourth quarter of 2020 compared to $0.3 million for the fourth quarter of 2019, and $2.8 million for the year ended December 31, 2020 compared to $1.3 million for the year ended December 31, 2019.

Research and Development (R&D) Expenses: R&D expenses were $95.7 million for the fourth quarter of 2020 compared to $106.2 million for the fourth quarter of 2019 and $367.5 million for the year ended December 31, 2020 compared to $410.9 million for the year ended December 31, 2019. The decrease in R&D expense was primarily attributable to a decrease in TIBSOVO clinical development costs, including winding down the ClarIDHy Phase 3 study.

Selling, General and Administrative (SG&A) Expenses: SG&A expenses were $39.8 million for the fourth quarter of 2020 compared to $34.8 million for the fourth quarter of 2019, and $149.1 million for the year ended December 31, 2020 compared to $132.0 million for the year ended December 31, 2019. The increase in SG&A expense was primarily attributable to increased workforce expenses and professional fees related to the Servier transaction, partially offset by a decrease in external spending due to cost savings initiatives and reduced employee travel related expenses due to restrictions.

Net Loss: Net loss was $97.7 million for the fourth quarter of 2020 compared to $102.4 million for the fourth quarter of 2019, and $327.4 million for the year ended December 31, 2020 compared to a net loss of $411.5 million for the year ended December 31, 2019.

Cash Position and Guidance: Cash, cash equivalents and marketable securities as of December 31, 2020 were $670.5 million compared to $717.8 million as of December 31, 2019. The company expects that its cash, cash equivalents and marketable securities as of December 31, 2020, together with anticipated product and royalty revenue, interest income and expense reimbursements under our collaboration agreements, but excluding any additional program-specific milestone payments, will enable the company to fund its planned operating expenses and capital expenditure requirements to the end of 2022. Following the closing of the Servier transaction and net of the planned capital return, Agios expects to be able to fund its operation through major catalysts and to cash-flow positivity without the need to raise additional equity.

CONFERENCE CALL INFORMATION

Agios will host a conference call and live webcast with slides today at 8:00 a.m. ET to discuss fourth quarter and full year 2020 financial results and recent business activities. To participate in the conference call, please dial 1-877-377-7098 (domestic) or 1-631-291-4547 (international) and referring to conference ID 8442238. The live webcast can be accessed under "Events & Presentations" in the Investors section of the company’s website at www.agios.com. The archived webcast will be available on the company’s website beginning approximately two hours after the event.

On February 25, 2021 Aclaris Therapeutics, Inc. (NASDAQ: ACRS), a clinical-stage biopharmaceutical company focused on developing novel drug candidates for immuno-inflammatory diseases, reported its financial results for the fourth quarter and full year of 2020 and provided a corporate update (Press release, Aclaris Therapeutics, FEB 25, 2021, View Source [SID1234575672]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"As we reflect on 2020, we are proud of how we have progressed our immuno-inflammatory development programs," said Dr. Neal Walker, President & CEO of Aclaris. "We are very excited about the platform potential of ATI-450, an oral MK2 inhibitor which we have shown to inhibit TNFα, IL1β, and IL6. Given its novel mechanism, there are several immuno-inflammatory indications that ATI-450 may potentially address. We are also excited about the potential of our pipeline which is internally generated from KINect, our proprietary drug discovery platform, for various immuno-inflammatory indications. We look forward to building on this momentum in 2021."

Research and Development Highlights:

The global COVID-19 pandemic continues to rapidly evolve and has caused and may continue to cause Aclaris to experience disruptions that could impact the timing of its research and development and regulatory activities listed below.

ATI-450, an investigational oral small molecule MK2 inhibitor compound:

ATI-450-RA-201: A 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.
Positive preliminary topline data announced in January 2021. In this trial, 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. This clinical activity was further supported by pharmacodynamic analyses showing a marked and durable inhibition of TNFα, IL1β, IL6, and IL8 in ex vivo stimulated samples as well as an endogenous inflammation biomarker analysis which 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.
ATI-450 was generally well tolerated. The most common adverse events (AE) (each reported in 2 subjects) were urinary tract infection (UTI), elevated lipids and ventricular extrasystoles, all of which were determined to be unrelated to treatment except for one UTI. 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.
Aclaris intends to progress ATI-450 into a Phase 2b trial in moderate to severe rheumatoid arthritis in the second half of 2021.
Aclaris is currently evaluating additional potential indications driven by TNFα, IL1β and IL6 as part of its planned expansion of its Phase 2 immuno-inflammatory clinical development programs.

ATI-450-PKPD-102: A Phase 1, placebo-controlled, randomized, observer-blind clinical trial to investigate the safety, tolerability, pharmacokinetics, and pharmacodynamics of ATI-450 at 80 mg and 120mg twice daily in health subjects.
Positive preliminary topline data announced in January 2021. Pharmacodynamic analysis demonstrated incremental cytokine suppression at these higher doses. ATI-450 was generally well tolerated. The most common AEs (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 AEs were all mild in severity.
A final analysis of this trial is underway.

ATI-450-CAPS-201: 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 (CAPS) previously managed with anti-IL1 therapy. 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, Aclaris has decided to focus its efforts and resources on other immuno-inflammatory diseases.
ATI-1777, an investigational topical "soft" Janus Kinase (JAK) 1/3 inhibitor compound:

ATI-1777-AD-201: An ongoing Phase 2a, multicenter, randomized, double-blind, vehicle-controlled, parallel-group clinical trial to investigate the efficacy, safety, tolerability and pharmacokinetics of ATI-1777 in subjects with moderate to severe atopic dermatitis. The primary endpoint is the percentage change from baseline in the Eczema Area and Severity Index (EASI) score at week 4. Data from this trial are expected mid-year 2021.
ATI-2138, an investigational oral ITK/TXK/JAK3 (ITJ) inhibitor compound:

Aclaris is developing ATI-2138 as a potential treatment for T-cell mediated diseases such as psoriasis and/or inflammatory bowel disease and expects to submit an Investigational New Drug Application for ATI-2138 in the second half of 2021.
Financial Highlights:

Liquidity and Capital Resources

As of December 31, 2020, Aclaris had aggregate cash, cash equivalents, restricted cash and marketable securities of $54.1 million compared to $75.0 million as of December 31, 2019. The changes in cash and cash equivalents and marketable securities during the year included:

Net cash used in operating activities was $38.6 million resulting from net loss of $51.0 million and changes in operating assets and liabilities of $2.4 million, partially offset by non-cash adjustments of $14.7 million.

Net borrowings of $10.9 million pursuant to a Loan and Security Agreement.

Net proceeds of $7.7 million from the sale of 2.1 million shares of common stock under an equity line of credit agreement.
In January 2021, Aclaris closed a public offering in which it sold approximately 6.3 million shares of common stock. Proceeds from the offering were $103.5 million, net of underwriting discounts, commissions and offering expenses. Aclaris anticipates that its cash, cash equivalents and marketable securities as of December 31, 2020 in combination with the proceeds from the January 2021 public offering will be sufficient to fund its operations through the end of 2023, without giving effect to any potential business development transactions or financing activities.

Financial Results

Fourth Quarter 2020

Net loss was $13.2 million for the fourth quarter of 2020 compared to $18.6 million for the fourth quarter of 2019. Loss from continuing operations was $13.6 million for the quarter ended December 31, 2020 compared to $19.2 million for the prior year period.
Total revenue was $1.6 million for the fourth quarter of 2020 compared to $1.1 million for the fourth quarter of 2019.

Research and development (R&D) expenses were $9.0 million for the quarter ended December 31, 2020 compared to $11.5 million for the prior year period.

The quarter-over-quarter decrease of $2.6 million was primarily the result of expenses related to Aclaris’ legacy JAK inhibitors ATI-501 and ATI-502, including the substantial completion of Aclaris’ various Phase 2 clinical trials, and the substantial completion of two pivotal Phase 3 clinical trials of A-101 45% Topical Solution in 2019.
General and administrative (G&A) expenses were $4.9 million for the quarter ended December 31, 2020 compared to $5.8 million for the prior year period.

The quarter-over-quarter decrease of $0.9 million was primarily the result of lower non-cash stock-based compensation expense resulting from headcount reductions. Stock-based compensation expense was $1.6 million compared to $2.6 million in the prior year period.
Full Year 2020

Net loss was $51.0 million for the year ended December 31, 2020 compared to $161.4 million for the year ended December 31, 2019. Loss from continuing operations was $51.2 million for the year ended December 31, 2020 compared to $113.5 million for the prior year period. Income from discontinued operations was $0.1 million for the year ended December 31, 2020 compared to a loss from discontinued operations of $47.8 million for the year ended December 31, 2019.

Total revenue was $6.5 million for the year ended December 31, 2020 compared to $4.2 million for the year ended December 31, 2019.

R&D expenses were $31.7 million for the year ended December 31, 2020 compared to $64.9 million for the prior year period.

The decrease of $33.2 million was primarily the result of expenses related to ATI-501 and ATI-502, including the substantial completion of Aclaris’ various Phase 2 clinical trials, and the substantial completion of two pivotal Phase 3 clinical trials of A-101 45% Topical Solution in 2019.
R&D expenses in 2020 included non-cash stock-based compensation expense of $2.9 million compared to $5.1 million in the prior year period.
G&A expenses were $20.5 million for the year ended December 31, 2020 compared to $27.8 million for the prior year period.

The decrease of $7.3 million was primarily the result of lower personnel and non-cash stock-based compensation resulting from headcount reductions.
G&A expenses in 2020 included non-cash stock-based compensation expense of $7.3 million compared to $10.3 million in the prior year period.
For the year ended December 31, 2019, there was also a $18.5 million non-cash charge for the impairment of goodwill.