ARCA biopharma Submits Special Protocol Assessment Request to FDA for Gencaro Phase 3 Atrial Fibrillation Clinical Trial
September 18 2018 - 8:30AM
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 a Special Protocol Assessment (SPA)
request to the U.S. Food and Drug Administration (FDA). The
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 heart failure (HF) patients at risk for atrial
fibrillation (AF).
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
risk of bringing a drug to market. The SPA submission is the next
step in beginning a Phase 3 program for Gencaro after an
End-of-Phase 2 Meeting with FDA in June 2018.
The protocol included with the SPA request
incorporates guidance ARCA received from the FDA regarding a Phase
3 program that could provide sufficient evidence of the efficacy
and safety of Gencaro in the treatment of atrial fibrillation.
"A successful SPA agreement with the FDA should
help us solidify the development and regulatory pathway for
Gencaro, including the details of our planned PRECISION-AF Phase 3
clinical trial," said Michael R. Bristow, MD, PhD, Chief Executive
Officer of ARCA biopharma. "We greatly appreciate the FDA’s
commentary and guidance on the Gencaro development program.
Gencaro is potentially the first genetically-targeted treatment for
heart failure patients at risk for atrial fibrillation and we are
excited to potentially move the program into Phase 3
development."
The ARCA 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 HFrEF patients (HF with left ventricular
ejection fraction (LVEF) < 50%). Eligible patients will have
HFrEF, an AF event within the prior 180 days and the genotype which
responds most favorably to Gencaro. The primary endpoint of
the submitted trial will utilize AF Burden (AFB) methodology.
AFB is defined as the amount of time per day a patient experienced
AF, as measured by an implanted cardiac electronic device (CIED).
In the recently completed GENETIC-AF Phase 2 clinical trial,
Gencaro showed a trend for 25% benefit over Toprol-XL in reducing
AF recurrence as measured by AFB in a subgroup of patients with
continuous monitoring with a CIED. For U.S. patients in this
substudy, a trend for 51% benefit was observed. The proposed
trial will have an adaptive design with an interim analysis and use
standard significance criteria (p < 0.05) for the primary
endpoint. Subject to FDA approval of the 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, 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
A photo accompanying this announcement is available at
http://www.globenewswire.com/NewsRoom/AttachmentNg/af496e97-20da-420a-bf93-e51b3a3ed740
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