ARCA biopharma Updates Special Protocol Assessment Request to FDA for Gencaro Phase 3 Atrial Fibrillation Clinical Trial
December 20 2018 - 4:01PM
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 Cole720.940.2163derek.cole@arcabio.com
ARCA Biopharma (NASDAQ:ABIO)
Historical Stock Chart
From Aug 2024 to Sep 2024
ARCA Biopharma (NASDAQ:ABIO)
Historical Stock Chart
From Sep 2023 to Sep 2024