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.
“We appreciate the FDA’s insight and input into the development pathway for Gencaro and look forward to continuing our interactions with the FDA,” said Michael R. Bristow, President and Chief Executive Officer of ARCA. “As a genetically targeted drug candidate for heart failure patients, we believe Gencaro has the potential to provide physicians and patients with a treatment with greater predictability of drug response and improved clinical outcomes for a disease that leads to approximately 12 to 15 million annual visits to physicians, 6.5 million annual hospital days and over $34 billion in direct and indirect annual healthcare costs.”
In recent months, the Company has negotiated early termination agreements relating to property leases that have resulted in a significant reduction in the Company’s financial obligations. The Company ended the third quarter of 2009 with $10.6 million in cash and cash equivalents. Due to recent actions to reduce operating expenses and corporate liabilities, the Company believes that its cash and cash equivalents are adequate to fund operations, at its current cost structure, through at least the first quarter of 2010.
Due to the substantial costs anticipated for the proposed trial, the Company is evaluating potential sources of funding including partnering or licensing Gencaro and the Company’s other product candidates, including NU-172 and rNAPc2. The Company is also evaluating other potential financing sources including equity capital and government grant funding.