ARCA biopharma, Inc. (Nasdaq: ABIO) today announced its planned next steps in response to the May 29, 2009 Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) for ARCA’s New Drug Application (NDA) seeking approval for GencaroTM (bucindolol hydrochloride) for the treatment of patients with chronic heart failure. Following receipt of the CRL, the Company and the FDA have held a series of meetings to discuss how best to address the requirements identified in the CRL.
The Company intends to submit a study protocol for review under the FDA’s Special Protocol Assessment (SPA) process for the design of a clinical trial to assess the safety and efficacy of Gencaro in approximately 3,000 patients with chronic heart failure who have the genotype that appears to respond most favorably to Gencaro. ARCA anticipates that the proposed trial protocol will be a superiority comparison to the beta-blocker metoprolol CR/XL, which is approved for heart failure and other indications. The Company believes that the proposed trial protocol will involve an interim data analysis at a pre-specified number of primary endpoints, which could serve as the clinical effectiveness basis for FDA approval if the results meet certain predefined criteria. If agreed to by the FDA, the Company anticipates that the proposed trial could reach the specified number of endpoint events as soon as approximately two years after the trial begins. The Company expects that the SPA submission will propose that a composite of cardiovascular mortality and cardiovascular hospitalization serve as the primary endpoint of the trial. Any proposed trial protocol must be reviewed and agreed on by the FDA and the final trial protocol may be significantly different from the Company’s initial SPA submission.
The Company anticipates that it will submit the study protocol for review under the SPA process in the fourth quarter of 2009. Subject to the timing and outcome of the FDA’s review of the SPA submission, and subject to the Company’s ability to obtain sufficient funding, the Company currently expects it could begin the proposed trial in late 2010 or the first half of 2011. The Company continues to actively work on, and believes it will be able to successfully address, the other issues raised in the CRL which must also be resolved prior to any marketing approval for Gencaro.