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 reduced left ventricular ejection fraction ("HFREF"), known as GENETIC-AF. 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. The initial phase of GENETIC-AF is planned as a 200 patient, Phase 2B study comparing Gencaro to metoprolol CR/XL for prevention of recurrent AF post electrical cardioversion in HFREF patients. The results from first 200 patients would be used in an adaptive design to determine if the trial goes to Phase 3 and full enrollment of 620 patients. We believe that this adaptive feature of GENETIC-AF is consistent with current FDA guidance on optimal clinical trial design. ARCA is in discussions with potential academic and industry partners to secure the funding for GENETIC-AF. We anticipate that the trial could begin approximately 6 months after we obtain sufficient funding.
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.