ARCA biopharma, Inc., a biopharmaceutical company applying a precision medicine approach to developing genetically targeted therapies for cardiovascular diseases, announced that the U.S. Food and Drug Administration (FDA) has designated as a Fast Track development program the investigation of AB201 as a potential treatment for COVID-19. The Company intends to initiate a Phase 2b clinical trial (ASPEN-COVID-19) of AB201 in approximately 100 patients hospitalized with COVID-19 in December 2020, with topline trial data anticipated in the second quarter of 2021.

According to the FDA’s Fast Track Guidance document, Fast Track programs are designed to facilitate the development and expedite the review of new drugs that are intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs.

“Fast Track designation for the AB201 development program is an important acknowledgement of the critical need for treatments for hospitalized COVID-19 patients, whether effective vaccines are approved and available or not,” said Dr Michael Bristow, ARCA’s President and Chief Executive Officer, who is also an American Heart Association (AHA) funded COVID-19 investigator. “We believe AB201’s combination of anticoagulant, anti-inflammatory and antiviral effects may favorably impact clinical recovery of patients hospitalized with COVID-19 and look forward to beginning the ASPEN-COVID-19 trial to evaluate AB201’s potential efficacy in this patient population."

Fast Track drug development designation is included in the FDA Modernization Act of 1997 (FDAMA) as a formal process to enhance interactions with the FDA during drug development. A drug development program with Fast Track designation would be eligible for consideration for some or all of the following programs for expediting development and review: scheduled meetings to seek FDA input into development plans, priority review of the New Drug Application (NDA), the option of submitting portions of an NDA prior to submission of the complete application and potential accelerated approval. ARCA believes that AB201 is the only anticoagulant class new chemical entity in development for COVID-19 that has a Fast Track designation.

AB201 is a small recombinant protein being developed as a potential treatment for RNA virus-associated diseases, initially focusing on COVID-19. AB201 is a potent, selective inhibitor of tissue factor (TF), which has been identified as playing a central role in the inflammatory response to viral infections and in the process of viral dissemination. Its unique mechanism of action gives AB201 a combination of anticoagulant, anti-inflammatory and antiviral properties, and therefore the potential to be effective in addressing the impact of viral infections from multiple pathways. AB201 has previously undergone Phase 1 and Phase 2 testing in more than 700 patients, including in clinical studies for prevention of venous and arterial thrombosis, where it showed efficacy in inhibiting the TF pathway and was well tolerated at therapeutic doses.

Recent research suggests that the disease syndrome caused by coronavirus may have much in common with other severe infections in which the infection process causes inappropriate activation of the coagulation system and other aspects of the immune response, resulting in serious complications. Recent mechanistic discoveries, as well as prior data from studies in non-human primates (NHPs) given lethal doses of Ebola or Marburg filoviruses demonstrating morbidity and mortality reductions, decreases in inflammatory biomarkers and reduction in viral load, indicate that AB201 may have important antiviral and anti-inflammatory activity in addition to its anticoagulant effects. The Company believes that collectively these observations provide a strong rationale for investigating AB201 as a treatment for COVID-19 and other RNA virus associated diseases.