ARCA biopharma, Inc. has 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 58 th Annual Scientific Session being held March 29-31, 2009 in Orlando, Florida.
The trial results demonstrated that, in a demographically-diverse group of primarily U.S. 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 percent of the projected primary endpoint events available and average follow-up shortened by 12 months.
Analyzed in accordance with the U.S. 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 percent (hazard ratio (HR) 0.87; p=0.053). All eight secondary endpoints of the trial were positive and statistically significant. As pre-specified 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.¡±
About BEST
The Beta Blocker Evaluation of Survival Trial (BEST) was a randomized, placebo-controlled 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. BEST was jointly funded by the Veteran's Administration Cooperative Clinical Studies Program (VA CCSP) and the National Heart, Lung and Blood Institute (NHLBI). As a result of the quality of data from existing studies in patients with heart failure and its tolerability, Gencaro was chosen as the beta-blocker for evaluation in this trial.