ARCA biopharma submits Gencaro trial protocol for review under the FDA’s SPA process

ARCA biopharma, Inc. (Nasdaq: ABIO) today announced that it has submitted a study protocol for review under the U.S. Food and Drug Administration’s (FDA) Special Protocol Assessment (SPA) process for the design of a clinical trial to assess the safety and efficacy of Gencaro^TM, in approximately 3,000 patients with chronic heart failure who have the genotype that appears to respond most favorably to Gencaro. Gencaro is the Company’s investigational, pharmacologically unique beta-blocker and mild vasodilator. The FDA has previously designated the investigation of Gencaro for the reduction of cardiovascular mortality and cardiovascular hospitalizations in a genotype-defined heart failure population as a fast-track development program. An SPA is an agreement with the FDA that the proposed trial protocol design, clinical endpoints and statistical analyses are acceptable to support regulatory approval. For further information, please visit the FDA website, www.fda.gov.

“We believe that Gencaro has the potential to provide a needed advancement in the treatment of patients with chronic heart failure, a disease afflicting approximately 6 million people in the United States with approximately 550,000 new cases diagnosed each year.”

“Submission of our trial protocol for review under the FDA’s SPA process is an important milestone for ARCA and Gencaro,” said Michael R. Bristow, President and Chief Executive Officer of ARCA. “We believe that Gencaro has the potential to provide a needed advancement in the treatment of patients with chronic heart failure, a disease afflicting approximately 6 million people in the United States with approximately 550,000 new cases diagnosed each year.”

The proposed trial protocol includes a superiority comparison to the beta-blocker metoprolol CR/XL, which is approved for heart failure and other indications. The proposed trial protocol also includes an interim data analysis at a pre-specified number of primary endpoints, the results of which could allow ARCA to formally submit a complete response to the FDA’s Complete Response Letter and could serve as the clinical effectiveness basis for FDA approval if the results meet certain predefined criteria agreed upon with the FDA during the SPA process. If the proposed protocol and criteria are 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 SPA submission proposes 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.

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.