ARCA biopharma, Inc (Nasdaq: ABIO) announced the release of final results for the Phase 3 BEST trial of Gencaro(TM) (bucindolol hydrochloride) in patients with advanced chronic heart failure (CHF). Gencaro showed a near significant benefit on overall mortality (hazard ratio (HR) 0.87; p=0.053), the trial’s primary endpoint. The drug demonstrated a significant benefit in slowing progression of heart failure (HR 0.80, p=0.00003), the most important secondary endpoint as specified by the FDA. Gencaro is currently under review by the FDA with a PDUFA date (the date by which the FDA will take action on the new drug application) of May 31, 2009. Although the BEST trial was conducted from 1995 to 1999 enrolling 2,708 patients, these full trial results analyzed according to the FDA-negotiated pre-specified statistical analysis plan had never been released.
ARCA had previously released results in September 2008 for 1000 BEST patients in a prospectively-designed DNA sub-study which categorized patients based on expected response to Gencaro through genetic variations in two adrenergic receptors involved in the regulation of cardiac function. For the sub-study, patients were classified into three categories or genotypes based on these genetic variations expressing the expected response to Gencaro: very favorable, favorable, or unfavorable. Patients in the very favorable group experienced the most benefit from treatment, including significant reductions in all cause mortality, all cause mortality or transplant, cardiovascular mortality, heart failure hospitalization, and cardiovascular hospitalization. Treatment benefits were also observed in patients in the favorable group. ARCA, headquartered in Broomfield, Colorado, is focused on the development of genetically targeted therapies for cardiovascular conditions.
