ARCA biopharma Announces GENETIC-AF Data and Safety Monitoring Board Recommendation to Complete Phase 2B GENETIC-AF Clinical ...
August 09 2017 - 4:30PM
ARCA Estimates Reporting Top-line Phase
2B Data in the First Quarter of 2018Gencaro
Development Program Has Been Granted Fast Track Designation by U.S.
FDA
ARCA biopharma, Inc. (Nasdaq:ABIO), a biopharmaceutical company
applying a precision medicine approach to developing
genetically-targeted therapies for cardiovascular diseases, today
announced that the GENETIC-AF trial Data and Safety Monitoring
Board (DSMB) completed its pre-specified Phase 2B interim analysis.
Based on its efficacy and safety review, the DSMB recommended
completion of the Phase 2B trial with no changes to the trial
design. GENETIC-AF is a clinical trial evaluating GencaroTM
(bucindolol hydrochloride) as a potential treatment for atrial
fibrillation (AF). The Gencaro development program has been
granted Fast Track designation by the U.S. Food and Drug
Administration (FDA).
“The DSMB stated that there were no safety
concerns and that the efficacy results met the prespecified
criteria for continuing the Phase 2B trial to completion. We are
pleased with the execution of the study thus far with major
pretrial assumptions reflected in the trial to date, including
genotype frequency, AF/AFL event rate and patient screening
rates. We believe the DSMB recommendation is encouraging, as
the trial is testing for superior efficacy over the active
comparator, TOPROL-XL,” commented Dr. Michael Bristow, ARCA’s
President and CEO. “We are now focused on completing the
Phase 2B trial, which we estimate should include approximately 50%
more events than were available at the interim analysis. We
look forward to sharing the top-line trial results late in the
first quarter of next year, and reviewing the findings with the
FDA.”
GENETIC-AF Clinical Trial
GENETIC-AF is a Phase 2B, adaptive design,
multi-center, randomized, double-blind, superiority clinical trial
comparing the safety and efficacy of Gencaro to TOPROL-XL
(metoprolol succinate) for the treatment and prevention of
recurrent atrial fibrillation or flutter (AF/AFL) in heart failure
patients with reduced left ventricular ejection fraction (HFrEF).
Eligible patients will have HFrEF, a history of paroxysmal AF
(episodes lasting 7 days or less) or persistent AF (episodes
lasting more than 7 days and less than 1 year) in the past 6
months, and the beta-1 389 arginine homozygous genotype that ARCA
believes responds most favorably to Gencaro. The primary
endpoint of the study is time to first event of symptomatic AF/AFL
or all-cause mortality. The GENETIC-AF trial design has been
reviewed by the FDA. The trial is enrolling patients in the
United States, Canada and Europe. The Company estimates
reporting top-line Phase 2B data late in the first quarter of
2018. ARCA believes that its current cash, cash equivalents
and marketable securities will be sufficient to fund its
operations, at its projected cost structure, into the second
quarter of 2018.
Interim Efficacy Analysis
The DSMB performed a pre-specified interim
analysis of unblinded efficacy data from all patients randomized as
of June 19, 2017. The primary analysis on the data from these
patients was conducted to evaluate the evidence of safety and
superior efficacy of Gencaro versus the active comparator,
metoprolol succinate (TOPROL-XL).
The prospectively defined features of this
analysis included an estimate of Gencaro effectiveness relative to
TOPROL-XL and an assessment of safety as characterized by adverse
events. The primary analysis method generated predictive
probability of success (PPoS) values that were compared to
prespecified PPoS boundaries constructed from Bayesian statistical
modeling. Prospectively defined PPoS ranges had been
predetermined to define three potential outcomes based on the
projection of the Phase 2B interim results:
1) transition the trial to Phase 3;2) completion
of the Phase 2B stage of the trial, based on an intermediate result
that is potentially favorable but does not support immediate
transition of the trial to Phase 3; or,3) immediate termination of
the trial due to futility, if the PPoS results fall below the
boundary for completion as a Phase 2 trial.
Based on the efficacy and safety data of the
interim analysis, the DSMB recommended completing the Phase 2B
trial with no changes to the trial design. In order to maintain the
integrity of the on-going double-blind clinical trial, the
unblinded statistical data available to the DSMB has not and will
not be disclosed to ARCA, the trial leadership, or the
investigators until the Phase 2B trial is complete. ARCA estimates
reporting top-line data for the Phase 2B trial late in the first
quarter of 2018.
About Gencaro
Gencaro (bucindolol hydrochloride) is an
investigational, pharmacologically unique beta-blocker and mild
vasodilator being developed for the treatment of AF. Gencaro is
considered part of the beta-blocker class of compounds because of
its property of blocking both beta-1 and beta-2, receptors in the
heart. The blocking of these receptors prevents them from binding
with other molecules, primarily the neurotransmitter
norepinephrine, or NE, which activate these receptors. ARCA
believes that Gencaro is well-tolerated in cardiovascular patients
because of its mild vasodilator effects. Originally developed by
Bristol-Myers Squibb the active pharmaceutical ingredient in
Gencaro, bucindolol hydrochloride, has been tested clinically in
approximately 4,500 patients, including over 3,000 patients in
seven clinical trials in HFrEF patients. Gencaro was the subject of
a Phase 3 HF mortality trial in 2,708 patients, mostly in the
United States, or the BEST trial. The BEST trial included a DNA
bank of over 1,000 patients, which was used to evaluate the effect
of genetic variation on patients’ response to Gencaro.
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 patients
with atrial fibrillation and HFrEF, currently in a Phase 2B
clinical trial. 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 atrial
fibrillation prevention treatment. ARCA has a collaboration
with Medtronic, Inc. for support of the GENETIC-AF trial. The
Gencaro development program has been granted Fast Track designation
by the FDA. 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, statements regarding, the
potential that the outcome of the Phase 2B interim analysis is
suggestive of a potentially positive outcome for the full Phase 2
trial, or sufficient to transition to a Phase 3 trial in the
future, the potential timeline for GENETIC-AF trial activities,
potential timing for the announcement of topline data for the Phase
2B portion of the GENETIC-AF trial, the sufficiency of ARCA’s
capital to support its operations, 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; 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, 2016, 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
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