ARCA biopharma, Inc, a biopharmaceutical company developing genetically-targeted therapies for heart failure and other cardiovascular diseases, announced that data from the pivotal phase-3 BEST trial of Gencaro (bucindolol hydrochloride) in patients with advanced chronic heart failure (CHF) was presented at the American College of Cardiology 58th Annual Scientific Session in Orlando, Florida. The trial results demonstrated that, in a demographically-diverse group of primarily US patients with New York Heart Association (NYHA) class III and IV heart failure, Gencaro treatment resulted in a near significant overall survival benefit and statistically significant benefits in slowing progression of heart failure. These results were observed despite the premature termination of the trial, with only 92 per cent of the projected primary endpoint events available and average follow-up shortened by 12 months.
Analyzed in accordance with the US Food and Drug Administration (FDA)-reviewed, pre-specified statistical analysis plan, the primary endpoint of mortality was reduced in all BEST trial patients on Gencaro by 13 per cent (hazard ratio (HR) 0.87; p=0.053). All eight secondary endpoints of the trial were positive and statistically significant. As prespecified with the FDA, the composite endpoint of heart failure progression was the most important secondary endpoint, with Gencaro shown to be superior to placebo for slowing progression of heart failure (HR 0.80, p=0.00003), and for the endpoint’s components of heart failure-related mortality (HR 0.85, p=0.042), heart failure-related hospital admission (HR 0.77, p=0.00002), and heart failure-related emergency room visits (HR
0.74, p=0.024).
“The results of the BEST trial, analyzed according to the pre-specified regulatory statistical analysis plan, are the foundation of our New Drug Application currently under review by the FDA with a PDUFA date of May 31, 2009,” commented Dr Michael R Bristow, ARCA’s founder and chief science and medical officer. “We believe there is a substantial need for new heart failure therapies for which response can be better predicted pharmacogenetically prior to the onset of therapy, and that Gencaro may help to address these needs.”
The Beta Blocker Evaluation of Survival Trial (BEST) was a randomized, placebocontrolled trial in patients with moderate to severe heart failure (NYHA Class III or IV), testing the hypothesis that beta-blockers reduce mortality and morbidities in patients with heart failure.
ARCA biopharma is dedicated to developing and commercializing genetically targeted therapies for heart failure and other cardiovascular disease.