- Phase 3 atrial fibrillation trial planned in population with no effective or FDA approved therapies
- PRECISION-AF, planned Phase 3 clinical trial in patients with genotype that responds most favorably to Gencaro
WESTMINSTER, Colo., Dec. 20, 2018 (GLOBE NEWSWIRE) -- ARCA biopharma, Inc. (Nasdaq: ABIO), a biopharmaceutical company applying a precision medicine
approach to developing genetically-targeted therapies for cardiovascular diseases, today announced that it has submitted an
amendment to its Special Protocol Assessment (SPA) request to the U.S. Food and Drug Administration (FDA).
The amendment addresses FDA feedback and guidance on the target population for ARCA’s planned Phase 3 clinical trial. The SPA
request is part of the Company’s ongoing interaction with the FDA focused on the planned Phase 3 clinical development program of
GencaroTM (bucindolol hydrochloride) as a genetically-targeted treatment for atrial fibrillation (AF) in patients with
heart failure (HF).
The FDA’s SPA process is designed to facilitate the FDA’s review and approval of drugs by allowing FDA to
evaluate the proposed design and size of certain clinical trials that are intended to form the primary basis for determining a drug
product’s efficacy and safety. Upon specific request by a clinical trial sponsor, FDA will evaluate the protocol and respond to a
sponsor’s questions regarding, among other things, primary efficacy endpoints, trial conduct and data analysis, within 45 days of
receipt of the request. FDA ultimately assesses whether the protocol design and planned analysis of the trial are acceptable
to support regulatory approval of the product candidate for the indication studied. An SPA agreement can potentially reduce the
regulatory risk of bringing a drug to market.
ARCA’s amended SPA incorporates guidance received from FDA during a December meeting regarding a Phase 3
clinical development program that could potentially provide sufficient evidence of the efficacy and safety of Gencaro in the
treatment of atrial fibrillation in patients with heart failure.
"We greatly appreciate the FDA’s commentary and guidance on the Gencaro clinical development program. We
believe a successful SPA agreement with the FDA could help us solidify the development and regulatory pathway for Gencaro," said
Michael R. Bristow, MD, PhD, Chief Executive Officer of ARCA biopharma. "Based on our analysis of Phase 2B data and our
interactions with the FDA, we believe with our proposed Phase 3 clinical development plan we have the opportunity to potentially
provide a new treatment for heart failure patients at risk for recurrent atrial fibrillation, who otherwise currently do not have
good pharmacologic therapeutic options."
The ARCA amended SPA submission details a single adequate and well-controlled Phase 3 clinical trial
(PRECISION-AF) designed as a double-blind, active-controlled, multicenter, international, study comparing Gencaro with Toprol-XL
(metoprolol succinate) for the prevention of recurrent AF or all-cause mortality (ACM) in HF patients with mid-range ejection
fraction (HFmrEF), which is defined as HF with a left ventricular ejection fraction (LVEF) ? 40% and <50%. Eligible
patients will have HFmrEF, a recent AF event and the genotype which responds most favorably to Gencaro. The primary endpoint
of the submitted trial will be time to first event of atrial fibrillation/atrial flutter (AF/AFL) or ACM during the 26-week
Follow-up Period. In the recently completed GENETIC-AF Phase 2 clinical trial, Gencaro showed a trend for benefit over
Toprol-XL in reducing AF recurrence in a subgroup of patients with HFmrEF (hazard ratio = 0.42; 95% CI: 0.21, 0.86; p = 0.017).
Subject to FDA approval of the amended SPA and securing additional financing, ARCA anticipates initiating PRECISION-AF in the
second half of 2019.
About ARCA biopharma
ARCA biopharma is dedicated to developing genetically-targeted therapies for cardiovascular diseases through a
precision medicine approach to drug development. ARCA’s lead product candidate, GencaroTM (bucindolol hydrochloride), is
an investigational, pharmacologically unique beta-blocker and mild vasodilator being developed for the potential treatment of heart
failure (HF) patients at risk for atrial fibrillation (AF). ARCA has identified common genetic variations that it believes predict
individual patient response to Gencaro, giving it the potential to be the first genetically-targeted AF prevention treatment.
The Gencaro development program has been granted Fast Track designation by FDA. ARCA is also developing AB171, a
thiol-substituted isosorbide mononitrate, as a potential genetically-targeted treatment for peripheral arterial disease (PAD) and
for heart failure (HF). For more information, please visit www.arcabio.com.
Safe Harbor Statement
This press release contains "forward-looking statements" for purposes of the safe harbor provided by the
Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, potential future development
plans for Gencaro, ARCA’s ability to enter into a SPA agreement with the FDA or complete any Phase 3 clinical trial thereunder, the
expected features and characteristics of Gencaro, including the potential for genetic variations to predict individual patient
response to Gencaro, Gencaro’s potential to treat AF, future treatment options for patients with AF, and the potential for Gencaro
to be the first genetically-targeted AF prevention treatment. Such statements are based on management's current
expectations and involve risks and uncertainties. Actual results and performance could differ materially from those projected
in the forward-looking statements as a result of many factors, including, without limitation, the risks and uncertainties
associated with: ARCA’s financial resources and whether they will be sufficient to meet its business objectives and operational
requirements; ARCA may not be able to raise sufficient capital on acceptable terms, or at all, to continue development of Gencaro
or to otherwise continue operations in the future; ARCA cannot guarantee that the FDA will issue an agreement on the SPA, and even
if ARCA does obtain the FDA’s agreement, a SPA would not guarantee approval of Gencaro or any other particular outcome from
regulatory review; results of earlier clinical trials may not be confirmed in future trials; the protection and market exclusivity
provided by ARCA’s intellectual property; risks related to the drug discovery and the regulatory approval process; and, the impact
of competitive products and technological changes. These and other factors are identified and described in more detail in
ARCA’s filings with the Securities and Exchange Commission, including without limitation ARCA’s annual report on Form 10-K for the
year ended December 31, 2017, and subsequent filings. ARCA disclaims any intent or obligation to update these forward-looking
statements.
Investor & Media Contact:
Derek Cole
720.940.2163
derek.cole@arcabio.com