Go to Top

ARCA biopharma, Inc. (ABIO): Phase 3 Trial for Gencaro in Atrial Fibrillation

ARCA biopharma, Inc. (NASDAQ: ABIO) is dedicated to developing genetically-targeted therapies for cardiovascular diseases. The Company’s lead product candidate, GencaroTM (bucindolol hydrochloride), is an investigational, pharmacologically unique beta-blocker and mild vasodilator being developed for the prevention of atrial fibrillation and treatment of heart failure in patients with heart failure and reduced left ventricular ejection fraction (“HFREF”).

ABIO today provided an update on a proposed, genetically-targeted clinical trial in atrial fibrillation (“AF”) of the Company’s lead developmental drug, Gencaro (bucindolol hydrochloride). The Company also announced that Medtronic, Inc., a leader in medical technologies to improve the treatment of chronic diseases, including cardiac rhythm disorders, has signed a non-binding Letter of Intent to collaborate on the initial, Phase 2B portion of the proposed trial.

The trial, known as GENETIC-AF, is projected to be a 620-patient, Phase 3 study comparing Gencaro to metoprolol CR/XL for prevention of AF in patients with heart failure and left ventricular dysfunction (“HFREF”).

The primary endpoint of GENETIC-AF is planned to be the combination endpoint of recurrent symptomatic AF and all-cause mortality, over 24 weeks post electrical cardioversion for persistent AF. Commencement of the GENETIC-AF is conditional on receipt of the necessary funding, which ARCA intends to secure through equity financing or a strategic partnership.

ARCA believes that Gencaro has potential as a treatment for AF, based on placebo-controlled data from the Phase 3 heart failure BEST study. A retrospective analysis of data from the BEST Trial shows that patients with the genotype that ARCA plans to enroll in GENETIC-AF had a 74% reduction in the risk of AF compared to placebo (p = 0.0003). These same patients experienced a 38% reduction in the risk of all cause mortality (p < 0.05) and statistically significant improvements on other major clinical efficacy endpoints.

ARCA has received guidance from the Food and Drug administration (“FDA”) regarding a Phase 3 clinical study comparing Gencaro to metoprolol for the prevention of AF in approximately 600 HFREF patients, with a design similar to GENETIC-AF, but without an adaptive feature. Based on this FDA guidance, the Company believes that a successful Phase 3 clinical study similar to GENETIC-AF, with a p-value of less than 0.01, could be sufficient evidence of efficacy upon which to base a New Drug Application (“NDA”) for the approval of Gencaro for an AF indication in HFREF patients. ARCA plans to obtain further guidance from the FDA, which may affect the trial’s design.

Dr. Michael Bristow, MD, PhD., President and Chief Executive Officer of ARCA, commented:

We at ARCA are excited about the new adaptive design for GENETIC-AF and the potential to use AF burden measured by previously implanted devices as well as by newly inserted implantable loop recorders, which we believe represent the next generation of diagnosis and treatment options for patients at risk for AF. AF is a serious disorder that increases the risk of stroke and mortality, and in HFREF patients often heralds the worsening of heart failure. There is a need for new treatment options, particularly for patients with HFREF. The GENETIC-AF trial has the potential to result in an approvable new therapy that is safe and effective for HFREF patients at high risk for AF. We believe that the use of continuous monitoring devices including implantable loop recorders to monitor AF burden in the Phase 2B substudy will result in many more clinically-relevant events, which will potentially increase the power and usefulness of the data. We believe that AF burden used in conjunction with drug therapy will become an increasingly important tool in the diagnosis and treatment of AF.