On May 7, 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 first quarter of 2021 and provided a corporate update (Press release, Aclaris Therapeutics, MAY 7, 2021, View Source [SID1234579427]).

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"We’re very pleased with the progress of our novel immuno-inflammatory drug development pipeline and look forward to reporting data from our Phase 2a trial of ATI-1777 in the second quarter of 2021," said Dr. Neal Walker, President & CEO of Aclaris. "ATI-1777 is our second development program generated by KINect, our proprietary drug discovery platform. After generating proof of mechanism in inhibiting TNFα, IL1β and IL6 in our Phase 2a trial of ATI-450 in moderate to severe rheumatoid arthritis, we are planning to move ATI-450 forward with a Phase 2b trial in moderate to severe rheumatoid arthritis in the second half of 2021 and planning to initiate two additional trials of ATI-450 in hidradenitis suppurativa and psoriatic arthritis."

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 19 subjects with moderate to severe rheumatoid arthritis. The trial consisted of a 12-week treatment period and a 4-week follow-up period. Two subjects withdrew from the trial during the treatment period, one in the treatment arm and one in the placebo arm.
Final per-protocol analysis, which consisted of 17 subjects who completed the treatment period (15 in the treatment arm and two in the placebo arm), confirmed that ATI-450 demonstrated durable clinical activity, as defined by a marked and sustained reduction in DAS28-CRP and improvement of ACR20/50/70 responses over 12 weeks.
Overall, ATI-450 was generally well tolerated. There were no treatment-related serious adverse events and all adverse events were mild to moderate. There was 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.
Aclaris intends to progress ATI-450 into a Phase 2b trial in moderate to severe rheumatoid arthritis in the second half of 2021.
As part of its planned expansion of its Phase 2 immuno-inflammatory clinical development programs, Aclaris also plans to progress ATI-450 into Phase 2 trials in hidradenitis suppurativa and psoriatic arthritis.
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 50 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.
Enrollment in this trial was completed in March 2021.
Data from this trial are now expected in the second quarter of 2021.
ATI-2138, an investigational oral ITK/TXK/JAK3 (ITJ) inhibitor compound:
Currently being developed as a potential treatment for T-cell mediated diseases such as psoriasis and/or inflammatory bowel disease.
Submission of Investigational New Drug Application is expected in the second half of 2021.
Aclaris is also expanding its Scientific Advisory Board with the addition of Dr. Philip Mease. Dr. Mease, a rheumatologist, currently serves as a Director of the Division of Rheumatology Clinical Research at the Swedish Medical Center/Providence St. Joseph Health and is a Clinical Professor at the University of Washington in Seattle. His major clinical and research focus is psoriatic arthritis and axial spondyloarthritis.

Financial Highlights:

Liquidity and Capital Resources

As of March 31, 2021, Aclaris had aggregate cash, cash equivalents and marketable securities of $142.7 million compared to $54.1 million as of December 31, 2020. The primary factors for the change in cash, cash equivalents and marketable securities during the three months ended March 31, 2021 included:

Net proceeds of $103.3 million from a public offering in January 2021 in which Aclaris sold 6.3 million shares of common stock.

Net cash used in operating activities of $12.2 million resulting from net loss of $28.8 million and changes in operating assets and liabilities of $2.9 million, partially offset by non-cash adjustments of $19.4 million which was primarily related to a $16.4 million charge for the revaluation of contingent consideration.
Aclaris anticipates that its cash, cash equivalents and marketable securities as of March 31, 2021 will be sufficient to fund its operations through the end of 2023, including estimated costs for the Phase 2b trial of ATI-450 for moderate to severe rheumatoid arthritis and the planned expansion of its Phase 2 immuno-inflammatory clinical development programs for hidradenitis suppurativa and psoriatic arthritis, without giving effect to any potential business development transactions or financing activities.

Financial Results

First Quarter 2021

Net loss was $28.8 million for the first quarter of 2021 compared to $15.6 million for the first quarter of 2020.

Total revenue was $1.8 million for the first quarter of 2021 compared to $1.4 million for the first quarter of 2020.

Research and development (R&D) expenses were $7.8 million for the quarter ended March 31, 2021 compared to $7.7 million for the prior year period.
The quarter-over-quarter increase of $0.1 million was primarily the result of continued investment in the further development of Aclaris’ immuno-inflammatory drug development pipeline, including ATI-450, ATI-1777 and ATI-2138, partially offset by a reduction in spend for legacy dermatology assets and personnel costs.
General and administrative (G&A) expenses were $4.8 million for the quarter ended March 31, 2021 compared to $6.2 million for the prior year period.
The quarter-over-quarter decrease of $1.4 million was primarily the result of lower personnel and non-cash stock-based compensation expenses.
Revaluation of contingent consideration charges related to the Confluence acquisition was $16.4 million for the quarter ended March 31, 2021 compared to $1.8 million for the prior year period.
The quarter-over-quarter increase in contingent consideration of $14.7 million primarily resulted from updates to probability of success and estimated future sales level assumptions following the completion of a Phase 2a clinical trial of ATI-450 in subjects with moderate to severe rheumatoid arthritis.

On May 7, 2021 Verrica Pharmaceuticals Inc. ("Verrica") (Nasdaq: VRCA), a dermatology therapeutics company developing medications for skin diseases requiring medical interventions, reported financial results for the first quarter ended March 31, 2021 (Press release, Verrica Pharmaceuticals, MAY 7, 2021, View Source [SID1234579473]).

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"This is a significant time for the Company as we ramp up our commercial readiness plans in preparation for potential FDA approval this year of VP-102 for the treatment of molluscum, a common, highly contagious skin disease with no FDA-approved treatments," said Ted White, Verrica’s President and Chief Executive Officer. "In parallel, we strengthened our financial position, raising funds and generating licensing revenues that we believe will support planned operations at least through the second quarter of 2023."

Ted White continued: "Further, we expanded our global reach by granting Torii an exclusive license to develop and commercialize VP-102 in Japan for the treatment of molluscum and common warts, executing the first strategic step in potentially bringing VP-102 to global markets."

Business Highlights and Recent Developments

The Company continued to expand its U.S. commercial operations during the quarter in preparation for the potential FDA approval of VP-102 (cantharidin 0.7% Topical Solution), and has made key hires in marketing, sales and payor functions to support product launch and commercialization. The Company has been assigned a Prescription Drug User Fee Act (PDUFA) goal date of June 23, 2021 for its NDA for VP-102. If approved, VP-102 will be marketed in the United States under the conditionally accepted brand name YCANTH.
On March 25, the Company closed an underwritten public offering of 2,033,899 shares of its common stock at a price to the public of $14.75 per share. The gross proceeds from the offering to Verrica were approximately $30.0 million, before offering expenses.
The Company entered into a Collaboration and License Agreement (the "Torii Agreement") with Torii Pharmaceutical Co., Ltd. ("Torii") granting Torii an exclusive license to develop and commercialize VP-102 in Japan for the treatment of molluscum and common warts. Under the terms of the Agreement, Torii made an up-front payment of $11.5 million to Verrica and has also agreed to make up to an additional $58 million in aggregate payments contingent on achievement of specified development, regulatory, and sales milestones. Torii will also make tiered transfer price payments for supply of product in the range of the mid-30s to mid-40s as a percent of net sales. Torii is responsible for all development activities and costs in support of obtaining regulatory approval in Japan.
Positive pooled results from the pivotal CAMP trials evaluating the safety and efficacy of VP-102 in the treatment of molluscum were published in the March 2021 issue of the American Journal of Clinical Dermatology.
Financial Results

First Quarter 2021 Financial Results

Verrica recognized license revenues of $12.0 million in the first quarter of 2021 related to the Torii Agreement. There were no license revenues recognized in 2020.
Research and development expenses were $5.4 million in the first quarter of 2021, compared to $4.9 million for the same period in 2020. The increase was primarily attributable to a one-time $2.3 million milestone payment to Lytix Biopharma AS upon the achievement of a regulatory milestone for LTX-315, partially offset by decreased Chemistry, Manufacturing and Controls (CMC) and clinical costs related to Verrica’s development of VP-102 for molluscum contagiosum, external genital warts, and common warts in 2020.
General and administrative expenses were $6.6 million in the first quarter of 2021, compared to $5.0 million for the same period in 2020. The increase was primarily a result of expenses related to increased headcount, an increase in insurance, professional fees and other operating costs, and an increase in expenses related to pre-commercial activities for VP-102.
For the first quarter of 2021, net loss on a GAAP basis was $0.9 million, or $0.04 per share, compared to a net loss of $9.8 million, or $0.39 per share, for the same period in 2020.
For the first quarter of 2021, non-GAAP net income was $0.6 million, or $0.02 per share, compared to a non-GAAP net loss of $8.8 million, or $0.35 per share, for the same period in 2020.
As of March 31, 2021, Verrica had aggregate cash, cash equivalents, and marketable securities of $87.7 million. The Company believes that its existing cash, cash equivalents, and marketable securities as of March 31, 2021, combined with the $11.5 million up-front payment received pursuant to the Torii Agreement in April 2021, will be sufficient to support planned operations at least through the second quarter of 2023.
Non-GAAP Financial Measures

In evaluating the operating performance of its business, Verrica’s management considers non-GAAP income from operations, non-GAAP net income (loss) and non-GAAP net income (loss) per share. These non-GAAP financial measures exclude stock-based compensation charges and non-cash interest expense that are required by GAAP. Verrica believes that non-GAAP income from operations, non-GAAP net income (loss) and non-GAAP net income (loss) per share provides useful information to both management and investors by excluding the effect of certain non-cash expenses and items that Verrica believes may not be indicative of its operating performance, because either they are unusual and Verrica does not expect them to recur in the ordinary course of its business, or they are unrelated to the ongoing operation of the business in the ordinary course. non-GAAP income from operations, non-GAAP net income (loss) and non-GAAP net income (loss) per share should be considered in addition to results prepared in accordance with GAAP, but should not be considered a substitute for, or superior to, GAAP results. Non-GAAP income from operations, non-GAAP net income (loss) and non-GAAP net income (loss) per share have been reconciled to the nearest GAAP measure in the tables following the financial statements in this press release.

About VP-102

Verricaʼs lead product candidate, VP-102, is a proprietary drug-device combination product that contains a GMP-controlled formulation of cantharidin (0.7% w/v) delivered via a single-use applicator that allows for precise topical dosing and targeted administration. VP-102 is currently under U.S. Food and Drug Administration (FDA) review, with a PDUFA goal date of June 23, 2021, and could potentially be the first product approved by the FDA to treat molluscum contagiosum ― a common, highly contagious skin disease that affects an estimated six million people in the United States, primarily children. If approved, VP-102 will be marketed in the United States under the conditionally accepted brand name YCANTH. In addition, Verrica has successfully completed a Phase 2 study of VP-102 for the treatment of common warts and a Phase 2 study of VP-102 for the treatment of external genital warts.

About Molluscum Contagiosum (Molluscum)

There are currently no FDA-approved treatments for molluscum, a highly contagious viral skin disease that affects approximately six million people — primarily children — in the United States. Molluscum is caused by a pox virus that produces distinctive raised, skin-toned-to-pink-colored lesions that can cause pain, inflammation, itching and bacterial infection. It is easily transmitted through direct skin-to-skin contact or through fomites (objects that carry the disease like toys, towels or wet surfaces) and can spread to other parts of the body or to other people, including siblings. The lesions can be found on most areas of the body and may carry substantial social stigma. Without treatment, molluscum can last for an average of 13 months, and in some cases, up to several years.

On May 7, 2021 OCI reported thet the company has decided to invest in Panolos Bioscience, a domestic bio venture company with anticancer drug candidates and multifunctional recombinant protein technology (Press release, Panolos Bioscience, MAY 7, 2021, View Source [SID1234633686]).

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On the same day, OCI announced on the 26th that it had signed an investment contract worth 5 billion won with Panolos Bioscience. "Through this strategic partnership, we will be able to secure expertise in the field of anticancer drugs," said OCI President Kim Taek-joong.

Panolos Bioscience is developing new biological therapeutics by utilizing αARTTM (Anti-angiogenesis-based Artifact Re-targeting Tri-specifics platform), a multi-specific drug generation platform. OCI explained that it is a platform that can reduce the side effects of single-target protein therapeutics and has excellent potential for expanding new drug pipelines through multiple targeting.

”PB101”, a next-generation anti-cancer drug candidate based on the αART platform, is a candidate for all types of Vascular Endothelial Growth Factor (VEGF)2, which is excessively produced around cancer cells (VEGF-A, VEGF-B, Placental Growth Factor). factor) to inhibit the growth of cancer cells.

Unlike conventional VEGF-inhibiting drugs that target only a portion of VEGF, it blocks all delivery pathways to reduce drug resistance and increase efficacy. PB101 is currently in the state of development of a process capable of mass production. Panolos Biosciences aims to start preclinical 3 in May and enter phase 1 clinical trial in 2022.

On May 7, 2021 Applied DNA Sciences, Inc. (NASDAQ: APDN) ("Applied DNA" or the "Company"), a leader in Polymerase Chain Reaction (PCR)-based DNA manufacturing, reported that it will report fiscal 2021 second quarter financial results after market close on Thursday, May 13, 2021 (Press release, Applied DNA Sciences, MAY 7, 2021, View Source [SID1234579454]). The Company’s management will discuss the results during a conference call and simultaneous webcast at 4:30 p.m. ET that same day. Presentation slides will also be posted to the ‘IR Calendar’ section of the Company’s corporate website and embedded into the live webcast.

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Conference Call and Webcast Information – Replay
A telephonic replay of the conference call will be available for one week beginning one hour after the end of the live conference call.

Webcast: View Source

Availability: Telephonic replay: until Thursday, May 20, 2021; webcast replay: 1 year

The webcast and accompanying PowerPoint presentation will also be archived on the ‘IR Calendar’ page listed under the Investor Relations drop-down menu on the Company’s website.

On May 7, 2021 Cytokinetics, Incorporated (Nasdaq: CYTK) reported financial results for the first quarter of 2021. Net loss for the first quarter was $47.1 million, or $0.66 per share, compared to net loss for the first quarter of 2020 of $39.4 million, or $0.66 per share. Cash, cash equivalents and investments totaled $460.2 million at March 31, 2021 (Press release, Cytokinetics, MAY 7, 2021, View Source,million%2C%20or%20%240.66%20per%20share. [SID1234579474]).

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"We continued to execute well in the first quarter against our ambitious plans. We recently convened a meeting with FDA related to omecamtiv mecarbil and GALACTIC-HF and are proceeding towards additional planned meetings this quarter with an expected NDA submission in the second half of the year," said Robert I. Blum, Cytokinetics’ President and Chief Executive Officer. "We look forward to the presentation of a secondary analysis from GALACTIC-HF which will shed further light on the impact of ejection fraction on patient outcomes. Additionally, we were pleased to complete enrollment in Cohort 2 of REDWOOD-HCM and more recently begin enrollment in the open-label extension study. We expect results from REDWOOD-HCM mid-year and are planning to initiate a pivotal Phase 3 trial by year-end."

Q1 and Recent Highlights

Cardiac Muscle Programs

omecamtiv mecarbil (cardiac myosin activator)

Met with the U.S. Food and Drug Administration (FDA) in Q1 and anticipate additional regulatory interactions in Q2 to inform plans to submit a New Drug Application (NDA) for omecamtiv mecarbil in 2H 2021. The planned regulatory filing is expected to be based on a single pivotal trial, GALACTIC-HF, which demonstrated a positive effect on the primary composite endpoint of cardiovascular death or heart failure events in patients receiving standard of care plus omecamtiv mecarbil

Announced that data from a secondary analysis of GALACTIC-HF (Global Approach to Lowering Adverse Cardiac Outcomes Through Improving Contractility in Heart Failure) assessing the effect of omecamtiv mecarbil on clinical outcomes in relationship to patient baseline ejection fraction will be presented in a Late Breaking Clinical Trial session at the American College of Cardiology 70th Annual Scientific Session & Expo (ACC.21).

Continued conduct of METEORIC-HF (Multicenter Exercise Tolerance Evaluation of Omecamtiv Mecarbil Related to Increased Contractility in Heart Failure), the second Phase 3 trial of omecamtiv mecarbil. Expect to complete enrollment in Q2 and report results in early 2022.

Conducted market research, forecasting and other planning activities in support of the potential commercialization of omecamtiv mecarbil.

Published a manuscript entitled, "Effect of Varying Degrees of Renal Impairment on the Pharmacokinetics of Omecamtiv Mecarbil" in Clinical Pharmacokinetics.
CK-3773274 (CK-274, cardiac myosin inhibitor)

Dosed the first patient in Cohort 2 of REDWOOD-HCM (Randomized Evaluation of Dosing With CK-274 in Obstructive Outflow Disease in HCM), the Phase 2 clinical trial designed to determine the safety and tolerability of CK-274 in patients with obstructive hypertrophic cardiomyopathy (oHCM). We subsequently completed enrollment in Cohort 2 of REDWOOD-HCM in Q1.

Opened enrollment in Cohort 3 of REDWOOD-HCM for patients whose background therapy includes disopyramide.

Activated the first site for enrollment in REDWOOD-HCM OLE, the open label extension clinical study designed to assess the long-term safety and tolerability of CK-274 in patients with symptomatic oHCM who have participated previously in REDWOOD-HCM.

Received orphan drug designation for CK-274 for the potential treatment of symptomatic HCM from the FDA.

Prepared for regulatory interactions with FDA to occur in Q2 and continuing into the second half of 2021 to inform preparations for a pivotal Phase 3 clinical trial of CK-274 in HCM, expected to begin by year-end.

Recently presented data related to the optimization of CK-274, including the first disclosure of its chemical structure, at the American Chemical Society Spring 2021 Virtual Meeting.

Enrolled the first patient in a Phase 1 study of CK-274 in China under the License and Collaboration Agreement between Cytokinetics and Ji Xing Pharmaceuticals Limited.
Skeletal Muscle Program

reldesemtiv (next-generation fast skeletal muscle troponin activator (FSTA))

Conducted start-up activities, including regulatory and institutional review board submissions, for COURAGE-ALS (Clinical Outcomes Using Reldesemtiv on ALSFRS-R in a Global Evaluation in ALS), the planned Phase 3 clinical trial of reldesemtiv in patients with ALS, in preparation for the potential opening of the trial to patient enrollment in 2H 2021.

Completed joint research program under collaboration with Astellas directed to the discovery of next-generation skeletal muscle activators.

Published a manuscript entitled "Reldesemtiv in Patients with Spinal Muscular Atrophy: A Phase 2 Hypothesis-Generating Study" in Neurotherapeutics.
Pre-Clinical Development and Ongoing Research

Continued to advance new chemical entities and to conduct IND-enabling studies with expectation of our potentially advancing 1-2 potential drug candidates in development over the next year.

Continued research activities directed to our other muscle biology research programs.
Corporate

Conducted planning activities related to the termination of the Collaboration and Option Agreement with Amgen and the transition of rights to develop and commercialize omecamtiv mecarbil, effective May 20, 2021.

Joined with the European Organisation for Rare Diseases (EURORDIS) and the National Organization for Rare Disorders (NORD) to recognize Rare Disease Day, an international campaign elevating the public understanding of rare diseases.

Awarded Cytokinetics Communications Fellowship Grants to patient advocacy organizations serving the heart failure, HCM, ALS and SMA communities to support increased capacity in communications, disease awareness and community engagement.

Convened inaugural Heart Failure Advisory Council meeting with patients and caregivers with heart failure to inform the ongoing development of the company’s heart failure directed pipeline.
Financials

Revenues for the first quarter 2021 increased to $6.5 million from $3.8 million for the first quarter 2020 due to increased research and development revenue from our collaborations with Amgen and Astellas.

Research and development expenses for the first quarter 2021 increased to $31.6 million from $21.7 million for the first quarter of 2020. The changes were primarily due to increases in spending for COURAGE-ALS and our clinical development activities for our cardiac muscle inhibitor programs.

General and administrative expenses for the first quarter of 2021 increased to $15.6 million from $12.4 million for the first quarter in 2020, due primarily to an increase in personnel related costs including stock-based compensation and higher outside spending for commercial readiness.

We expect to revise our financial guidance mid-year once we finalize strategies and potential commercial launch plans for omecamtiv mecarbil. Executing on those strategies and plans may result in our incurring significant additional expenses that were not included in our current financial guidance. We expect that some or all of those potential expenses could be covered by our accessing additional capital through strategic partnership(s) with near term cash infusions or by equity and/or debt financings if deemed appropriate.

Conference Call and Webcast Information

Members of Cytokinetics’ senior management team will review the company’s first quarter 2021 results via a webcast and conference call today at 4:30 PM Eastern Time. The webcast can be accessed through the Investors & Media section of the Cytokinetics website at www.cytokinetics.com. The live audio of the conference call can also be accessed by telephone by dialing either (866) 999-CYTK (2985) (United States and Canada) or +1 (706) 679-3078 (international) and typing in the passcode 6097958.

An archived replay of the webcast will be available via Cytokinetics’ website until May 20, 2021. The replay will also be available via telephone by dialing (855) 859-2056 (United States and Canada) or +1 (404) 537-3406 (international) and typing in the passcode 6097958 from May 6, 2021 at 7:30 PM Eastern Time until May 20, 2021.