On March 18, 2021 ARCA biopharma, Inc. (Nasdaq: ABIO), a biopharmaceutical company applying a precision medicine approach to developing genetically targeted therapies for cardiovascular diseases, reported 2020 financial results and provided a corporate update (Press release, Arca biopharma, MAR 18, 2021, View Source [SID1234576863]).

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Dr. Michael Bristow, ARCA’s President and Chief Executive Officer, commented, "We are currently actively enrolling patients in a Phase 2b clinical trial evaluating rNAPc2 as a potential treatment for patients hospitalized with COVID-19 who are at high risk for thrombotic complications, as indicated by elevated D-dimer levels, which are elevated in nearly 70% of hospitalized COVID-19 patients and are associated with adverse clinical outcomes. We believe rNAPc2’s combination of anticoagulant, anti-inflammatory and antiviral properties, give it the potential to be effective in addressing the impact of COVID-19 from multiple pathways. We look forward to sharing the trial results in the third quarter of this year. As a therapeutic aimed at a host response to a disease syndrome, we believe rNAPc2 has therapeutic potential for future viral outbreaks beyond the current pandemic, even after safe and effective vaccines for SARS-CoV-2 are successfully deployed."

Pipeline Update

rNAPc2 (AB201) – a small recombinant protein being developed as a potential treatment for RNA virus associated disease, initially focusing on COVID-19.

Phase 2b clinical trial evaluating rNAPc2 as a potential treatment for patients hospitalized with COVID-19 initiated in December 2020.
Phase 2b topline data anticipated in the third quarter of 2021.
U.S. Food and Drug Administration (FDA) designated the investigation of rNAPc2 as a potential treatment for COVID-19 as a Fast Track development program.
GencaroTM (bucindolol hydrochloride) – a pharmacologically unique beta-blocker and mild vasodilator being developed as a potential genetically-targeted treatment for atrial fibrillation (AF) in patients with heart failure (HF).

In February 2021, ARCA was issued a new patent by the United States Patent and Trademark Office (USPTO) for use of Gencaro in treating AF in the HF population that ARCA plans to enroll in Phase 3 development, a population that includes more than half of all HF patients in the United States and Europe and has few approved or effective drug therapies. The Company believes this patent would provide effective patent coverage in the United States into 2039. ARCA has filed similar patent applications in other countries.
The FDA has issued a Special Protocol Assessment (SPA) agreement for a single Phase 3 clinical trial (PRECISION-AF) to examine Gencaro as a genetically targeted therapy for the prevention of AF recurrence in certain heart failure patients. We continue to evaluate the feasibility and potential timing for initiation of PRECISION-AF relative to the COVID-19 pandemic and the ability to recruit patients for a cardiovascular clinical trial.
Full Year 2020 Summary Financial Results

Cash and cash equivalents were $49.1 million as of December 31, 2020, compared to $8.4 million as of December 31, 2019. ARCA believes that its current cash and cash equivalents, together with the $23.3 million of net proceeds raised through February 2021 from sales of the common stock under the Company’s "at-the-market" offering, will be sufficient to fund its operations through 2022.

Research and development (R&D) expenses were $5.0 million for the year ended December 31, 2020, compared to $1.8 million for 2019. The $3.2 million increase in R&D expenses in 2020 as compared to 2019 was primarily related to the initiation of the rNAPc2 clinical trial in the second half of 2020. R&D expenses in 2021 are expected to be higher than 2020, as the Company continues the rNAPc2 Phase 2b clinical trial.

General and administrative (G&A) expenses were $4.8 million for the year ended December 31, 2020, compared to $4.0 million for 2019, an increase of approximately $0.8 million. The increase in expenses during 2020 was comprised primarily of increased legal and professional costs, insurance costs and higher personnel costs in 2020, as compared to 2019. G&A expenses in 2021 are expected to be consistent with those in 2020 as the Company maintains administrative activities to support our ongoing operations.

Total operating expenses for the year ended December 31, 2020 were $9.8 million compared to $5.8 million in 2019.

Net loss for the year ended December 31, 2020 was $9.7 million, or $2.07 per basic and diluted share, compared to $5.5 million, or $4.15 per basic and diluted share in 2019.

On March 18, 2021 Applied Therapeutics, Inc. (Nasdaq: APLT), a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need, reported financial results for the fourth quarter and full year ended December 31, 2020 (Press release, Applied Therapeutics, MAR 18, 2021, View Source [SID1234576862]).

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"The fourth quarter was a productive period of internal planning and execution," said Shoshana Shendelman, PhD, Founder, CEO and Chair of the Board of Applied Therapeutics. "We are excited for what lies ahead in 2021, where our focus will be on advancing our late stage assets toward commercialization while initiating clinical development of our two new rare disease programs."

Recent Highlights

Announced Restart of Pediatric Galactosemia Study. In February 2021, the Company announced that the FDA lifted the clinical hold on the AT-007 ACTION-Galactosemia Kids pediatric clinical study, and the study resumed immediately. Applied Therapeutics worked closely with FDA to modify the trial, with the shared goal of ensuring that all patients have the opportunity to receive clinical benefit, and remains on target to submit an NDA no later than Q3 2021.

Announced the Launch of a Galactosemia Awareness and Education Initiative. Galactosemia Together is the first and only industry-led Galactosemia awareness and education campaign. Developed in partnership with the Galactosemia community, this initiative aims to address gaps in education by providing updated, reliable and credible resources to help connect, educate and support those families impacted by this disease.

Announced Magnetic Resonance Spectroscopy (MRS) Data from ACTION-Galactosemia Study. In December 2020, the Company shared MRS data on reduction of galactitol levels in the brain of Galactosemia patients treated with AT-007 in the ACTION-Galactosemia adult study. Overall, plasma reduction in galactitol correlated with brain reduction in galactitol. At the two doses which demonstrated statistically significant reduction in plasma galactitol, 20 and 40mg/kg, 3 out of 4 patients displayed substantial galactitol reduction ranging from 61.94% to 69.80% reduction from baseline.

Closed Public Offering of 3,000,000 Shares of Common Stock at a Price to the Public of $23.00 Per Share. In February 2021, the Company completed an underwritten public offering of 3,450,000 shares of common stock, including the exercise in full of the underwriters’ option to purchase 450,000 additional shares of common stock at a price of $23.00 per share, resulting in aggregate net proceeds of approximately $74.3 million.
Financial Results

Cash and cash equivalents and short-term investments totaled $96.8 million as of December 31, 2020, compared with $38.9 million at December 31, 2019. This does not include approximately $74.3 million in aggregate net proceeds the Company received from the underwritten public offering of common stock in February 2021.

Research and development expenses for the year ended December 31, 2020 were $61.8 million, compared to $32.4 million for the year ended December 31, 2019. The increase of $29.4 million related to an increase in clinical and pre-clinical expense of $11.3 million, primarily related to the progression of the AT-007 ACTION-Galactosemia adult extension and the AT-001 Phase 3 ARISE-HF clinical studies, as well as the commencement and progression of the AT-007 ACTION-Galactosemia Kids pediatric registrational study; an increase in drug manufacturing and formulation expenses of $15.4 million primarily related to the commencement of the 2020 manufacturing campaigns and the associated raw material deliveries and AT-007 and AT-001 drug product batch releases; an increase in personnel expenses of $0.6 million due to the increase in headcount in support of our clinical program pipeline; a decrease in stock-based compensation of $0.2 million due to the stock option modification expense recognized during the year ended December 31, 2019 for the acceleration of certain options vesting following the IPO; offset by an increase in expense recognized during the year ended December 31, 2020 due to new stock option and restricted stock grants; and an increase of regulatory and other expenses of $2.3 million primarily related to University of Miami license fees and increased clinical consulting fees recognized during the year ended December 31, 2020.

General and administrative expenses were $32.7 million for the year ended December 31, 2020, compared to $13.2 million for the year ended December 31, 2019. The increase of $19.4 million was an increase in professional and legal fees of $4.1 million due to increased operations and costs associated with being a public company for a full year; an increase of $5.7 million related to the establishment of a commercial department; an increase in personnel expenses of $4.2 million and an increase in stock-based compensation of $2.0 million due to an increase in headcount; an increase of insurance expenses of $1.8 million related to increased D&O insurance costs; and an increase in other expenses of $1.6 million, primarily relating to increased costs of rent and other office expenses.

Net loss for the year ended December 31, 2020 was $94.0 million, or $4.28 per basic and diluted common share, compared to a net loss of $45.5 million, or $3.55 per basic and diluted common share, for the year ended December 31, 2019.

On March 18, 2021 Alpine Immune Sciences, Inc. (NASDAQ: ALPN), a leading clinical-stage immunotherapy company focused on developing innovative treatments for cancer and autoimmune/inflammatory diseases, reported financial results for the fourth quarter and year ended December 31, 2020 (Press release, Alpine Immune Sciences, MAR 18, 2021, View Source [SID1234576861]).

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"This past year has been transformative for the company. We are building on our strong momentum to accelerate development of innovative therapies for patients living with cancer and autoimmune/inflammatory disease," said Mitchell H. Gold, M.D., Executive Chairman and Chief Executive Officer of Alpine. "Over the next year we will continue to execute on our clinical-stage programs and plan to initiate an international Phase 2 trial of ALPN-101 in adults with systemic lupus erythematosus (SLE), and further progress our ongoing NEON-1 Phase 1 study of ALPN-202 in advanced malignancies. We also plan to progress key pipeline programs, including the initiation of a Phase 1 healthy volunteer study for ALPN-303. In summary, I believe 2021 will be another year of significant opportunity for Alpine as we advance our pipeline of innovative therapeutic candidates."

Full Year 2020 and Recent Corporate and Clinical Updates

ALPN-101: Dual CD28/ICOS inhibitor
Announced Option and License Agreement with AbbVie: In June 2020, Alpine announced an exclusive worldwide option and license agreement for ALPN-101. Under the terms of the agreement, Alpine received an upfront payment of $60 million and is eligible to receive up to an aggregate of $805 million for exercise of the option and success-based development, regulatory, and commercial milestones, including up to $75 million in pre-option development milestones. In addition, Alpine is eligible to receive tiered royalties on net sales of ALPN-101.
Preparation for a global Phase 2 study in SLE is ongoing with an intent to initiate the study in mid-2021.
ALPN-202: Conditional CD28 costimulator and dual checkpoint inhibitor
Presentation of NEON-1 trial design at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) Virtual Annual Meeting 2020: The design of NEON-1, our first-in-class conditional CD28 costimulator and dual checkpoint inhibitor, was presented at the AACR (Free AACR Whitepaper) Virtual Annual Meeting I, in the Phase I Trials in Progress Virtual Poster Session.
Enrollment in NEON-1, a first in human monotherapy Phase 1 clinical trial, continues to progress through its dose escalation cohorts. Alpine plans to share interim data at an appropriate scientific forum and to determine expansion cohorts later this year.
Alpine is also targeting the initiation of NEON-2, a Phase 1 combination study of ALPN-202 and a PD-1 inhibitor, later this year.
ALPN-303: Dual APRIL/BAFF inhibitor
Presentation of Preclinical Data on Novel B cell Modulatory Domains, Alone or in Combination with Novel Domains that Inhibit T Cell Costimulation, for Autoimmune Diseases: In June 2020, Alpine presented a poster at the European Congress of Rheumatology (EULAR) E-Congress detailing preclinical work with novel B cell modulatory variant TNF Receptor domains (vTDs), alone or in combination with variant Ig Domains (vIgDtm) that inhibit T cell costimulation. This work included ALPN-303, a novel dual BAFF/APRIL inhibitor, in multiple preclinical models, including the NZB/NZW F1 murine lupus model.
Alpine is targeting completion of activities to support initiation of a Phase 1 healthy volunteer study with ALPN-303 in the fourth quarter of this year.
General Corporate
Raised $60 Million in a Private Placement: In July 2020, Alpine raised $60 million in gross proceeds through a private placement led by Omega Funds with participation from Avidity Partners, EcoR1 Capital, LLC, Invus Public Equities, L.P., and Samsara BioCapital, among others.
Appointed industry veteran Natasha Hernday to the Board of Directors: Ms. Hernday brings more than 20 years of experience in corporate development and corporate strategy, and currently serves as Executive Vice President, Corporate Development and as a member of the Executive Committee for Seagen, Inc.
Promotion of Remy Durand, Ph.D. to Chief Business Officer: Dr. Durand joined Alpine Immune Sciences in 2018 as Vice President, Business Development, and has played a leading role in building the company’s partnerships with AbbVie and Adaptimmune, and has represented the company at investor meetings and conferences.
Appointed Pamela Holland, Ph.D. as Senior Vice President, Research: Dr. Holland is an experienced cancer biologist with a proven track record of successfully discovering and progressing multiple preclinical therapeutics into clinical development, most recently at Surface Oncology and Amgen.
Full Year 2020 Financial Results

As of December 31, 2020, we had cash, cash equivalents, and investments totaling $131.4 million. Net cash provided by operating activities for the year ended December 31, 2020 was $30.1 million compared to net cash used in operating activities of $35.3 million for the year ended December 31, 2019. We recorded net losses of $27.9 million and $41.9 million for the years ended December 31, 2020 and 2019, respectively.

Collaboration revenue for the year ended December 31, 2020 was $9.3 million compared to $1.7 million for the year ended December 31, 2019. The increase was primarily attributable to the revenue recognized under our AbbVie Agreement.

Research and development expenses for the year ended December 31, 2020 were $27.2 million compared to $35.8 million for the year ended December 31, 2019. The decrease was primarily attributable to decreases in contract manufacturing and process development of our product candidates and other direct research activities. The decreases were partially offset by increases in clinical trial activity for NEON-1.

General and administrative expenses for the year ended December 31, 2020 were $10.9 million compared to $9.5 million for the year ended December 31, 2019. The increase was primarily attributable to increases in professional and legal services, stock-based compensation and insurance and facility costs to support the growth and expansion of our business.

Alpine expects that its current cash resources, combined with the potential $75 million in pre-option exercise milestones payable under its option and license agreement with AbbVie, for the development and commercialization of ALPN-101, are sufficient to fund Alpine’s planned operations through 2023.

Fourth Quarter and Full Year 2020 Conference Call and Webcast Details

Alpine will host a conference call and live webcast to discuss the fourth quarter and full year performance today, March 18, 2021 at 4:30 p.m. ET/1:30 p.m. PT.

To access the live call by phone, dial (800) 816-3005 (domestic) or (857) 770-0069 (international) and reference conference ID: 2539338. A live webcast of the presentation will be available online in the investor relations section of the company’s website at View Source A replay of the presentation will be available on the company website for 90 days following the webcast.

On March 18, 2021 Alligator Bioscience (Nasdaq Stockholm: ATORX) reported that the Board of Directors has appointed Søren Bregenholt, Ph.D. as the company’s new Chief Executive Officer (CEO), to strengthen Alligator’s business development activities and clinical progress on an international level (Press release, Alligator Bioscience, MAR 18, 2021, View Source [SID1234576860]). Dr. Søren Bregenholt will take up the position on 1 June 2021, succeeding Per Norlén, who will remain in the company. In order to facilitate the transition, Malin Carlsson, Alligator’s COO, is appointed as interim CEO.

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"We’re delighted to have Søren onboard. In line with Alligator’s strategy and increased focus on business development and its clinical assets, Alligator is now entering a phase where it will be commercially crucial to further develop and leverage our international network. We are convinced that Søren’s broad global network across pharma, biotech, venture capital and academia, together with his extensive deal experience from both small and large companies, will be invaluable for Alligator as the company continues to assess favorable partnership and out-licensing opportunities, activities which are highly prioritized for Alligator", said Peter Benson, Chairman of Alligator’s Board of Directors.

Søren Bregenholt has more than 20 years of international experience from operational and strategic leadership positions in global pharma and the biotech industry. He most recently served as CEO and Board Director at Macrophage Pharma Ltd in the UK. Previous experiences include Corporate Vice President positions at Novo Nordisk A/S between 2010 and 2018, and COO at Symphogen A/S where he worked from 2002 to 2010. During his career Søren Bregenholt has negotiated and operationalized numerous licensing, collaboration and co-development agreements. Søren received his PhD from University of Copenhagen and did his post-doctoral training at Institute Pasteur in Paris. He is the author and co-author of more than 45 papers in peer reviewed scientific journals. "I am really looking forward to the new challenge of leading Alligator Bioscience, a very dynamic and respected company in the exciting field of immuno-oncology. I am impressed by the robust quality of the clinical portfolio as well as the research and development capabilities that has brought the company to this stage. It will be truly exciting to lead and work closely with the competent team at Alligator and take the company further towards clinical and commercial success", said Søren Bregenholt.

This information is information that Alligator Bioscience AB is obliged to make public pursuant to the EU Market Abuse Regulation. The information was submitted for publication, through the agency of the contact person set out above, at 7:00 p.m. CET on March 18, 2021.

On March 18, 2021 VAXIMM AG, a Swiss/German biotech company focused on developing an oral plug and play DNA vaccination technology to stimulate patients’ cytotoxic T-cells targeting a wide range of cancer-related antigens, reported that the Company will participate in several scientific and industry events in the coming weeks (Press release, Vaximm, MAR 18, 2021, View Source [SID1234576853]). All conferences will be held virtually.

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15th Annual International Partnering Conference BIO-Europe Spring
March 22-25, 2021
Dr. Heinz Lubenau, Chief Executive Officer and Co-Founder, will give a company presentation and participate in the online partnering system. To request a meeting, please sign up through the event’s partneringONE meeting system.

European Biotech Investor Days 2021
April 7-8, 2021
Dr. Lubenau will give a company presentation at 5pm CEST, 7 April, and be available for one-on-one meetings.

American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting
April 10-15 and May 17-21, 2021
Dr. Lubenau will attend the conference. To schedule a meeting with the Company, please contact [email protected].

Alliance for Cancer Gene Therapy Summit 2021
April 29, 2021
Dr. Lubenau will attend the conference. To schedule a meeting with the Company, please contact [email protected].

Upcoming events in which VAXIMM will participate are updated on a regular basis. See the Events section of the Company’s website for more information.