ARCA biopharma, Inc. (Nasdaq:ABIO) today announced that the U.S. Food and Drug Administration (FDA) has designated as a Fast Track development program the investigation of GencaroTM, the Company’s investigational, pharmacologically unique beta-blocker and mild vasodilator, for the reduction of cardiovascular mortality and cardiovascular hospitalizations in a genotype-defined heart failure population. 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.
According to the FDA’s Fast Track Guidance document, Fast Track programs are designed to facilitate the development and expedite the review of new drugs that are intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs.
“Fast Track designation for the Gencaro development program is an important acknowledgement of the need for advancements 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,” said Michael R. Bristow, President and Chief Executive Officer of ARCA. “If the SPA is approved by the FDA and the Company is able to obtain financing, this proposed clinical trial would be the first full sized cardiovascular trial performed in a genetically defined subpopulation to predict efficacy enhancement by the tested drug. As such, the proposed trial would be a landmark undertaking in pharmacogenetic drug development.”
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.
Fast Track drug development designation is included in the FDA Modernization Act of 1997 (FDAMA) as a formal process to enhance interactions with the FDA during drug development. A drug development program with Fast Track designation would be eligible for consideration for some or all of the following programs for expediting development and review: scheduled meetings to seek FDA input into development plans, priority review of the New Drug Application (NDA), the option of submitting portions of an NDA prior to submission of the complete application and potential accelerated approval.