ARCA biopharma, Inc. is a Colorado-based biopharmaceutical company developing genetically-targeted therapies for cardiovascular diseases.
ARCA’s principal focus is to develop personalized therapies for the treatment of cardiovascular disease through the use of genetics. The Company’s business focus combines expertise in cardiovascular pathophysiology, molecular genetics and clinical development.
The Company's lead product candidate, GencaroTM (bucindolol hydrochloride), is an investigational, pharmacologically unique beta-blocker and mild vasodilator being developed for the prevention of atrial fibrillation in patients with heart failure. ARCA has identified common genetic variations in the cardiovascular system that it believes interact with Gencaro’s pharmacology and may predict individual patient response to Gencaro, giving it the potential to be the first genetically-targeted atrial fibrillation prevention treatment.
We are planning to initiate a Phase 3 clinical study of Gencaro in AF patients with heart failure and left ventricular dysfunction. We believe AF is an attractive indication for Gencaro because data from BEST, the previously conducted Phase 3 HF trial involving Gencaro in 2,708 HF patients, suggest Gencaro may have a potentially significant effect in reducing and/or preventing AF. Based on the BEST trial we believe Gencaro's prevention of AF in HF patients is pharmacogenetically regulated, similar to the effects on HF clinical endpoints, and plan to enroll approximately 300-400 patients with recent onset AF who have the genotype that appears to respond most favorably to Gencaro. We anticipate that the trial could begin approximately 6 months after we obtain sufficient funding.
In September 2008, the U.S. Food and Drug Administration, or FDA, formally accepted for filing our New Drug Application, or NDA, for Gencaro as a potential treatment for HF. In May 2009, the FDA notified us through a Complete Response Letter, or CRL, that our NDA for Gencaro was not approvable in its current form, and specified additional actions and information required for approval of the NDA including conducting an additional Phase 3 clinical trial. In May 2010, we reached agreement with the FDA on a Special Protocol Assessment, or SPA, for the design of an additional Phase 3 clinical trial to assess the safety and efficacy of Gencaro in approximately 3,200 patients with HF who have the genotype that appears to respond most favorably to Gencaro. We believe that the SPA agreement would permit this trial, if successful, to serve as the clinical effectiveness basis for the approval of Gencaro in HF. In light of the substantial costs associated with the Phase 3 clinical HF trial, the Company does not plan to initiate the trial until government funding, or a strategic transaction, such as a strategic combination or partnership is secured or the planned AF trial is completed.
