ARCA Biopharma Announces Database Lock for GENETIC-AF Phase 2B Interim Efficacy Analysis – DSMB Recommendation Anticipated ...
June 20 2017 - 8:30AM
GENETIC-AF Evaluating Gencaro as
Potentially First Genetically-Targeted Treatment
for Atrial Fibrillation
ARCA biopharma, Inc. (Nasdaq:ABIO), a biopharmaceutical company
applying a precision medicine approach to developing
genetically-targeted therapies for cardiovascular diseases, today
announced database lock for the GENETIC-AF Phase 2B interim
efficacy analysis, to be conducted by the trial Data Safety
Monitoring Board (DSMB). GENETIC-AF is a seamless
design Phase 2B/3 clinical trial evaluating GencaroTM (bucindolol
hydrochloride) as a potential treatment for atrial fibrillation
(AF). The Company expects to announce the DSMB’s recommendation
based on this interim analysis in August 2017. The Gencaro
development program has previously been granted Fast Track
designation by the U.S. Food and Drug Administration (FDA).
A photo accompanying this announcement is available at
http://www.globenewswire.com/NewsRoom/AttachmentNg/af496e97-20da-420a-bf93-e51b3a3ed740
“Reaching database lock for the interim analysis
and the collection and processing of trial data for the DSMB mark
important points in the development of Gencaro. We are hopeful a
positive DSMB recommendation will further support our data-driven
rationale for development of the first genetically-targeted
treatment candidate for atrial fibrillation in heart failure
patients,” commented Dr. Michael Bristow, ARCA’s President and
CEO. “Based on our view of the strength of prior AF
prevention data from the BEST trial and pursuant to discussions
with the FDA, we have incorporated a seamless design feature for
this trial. Beyond the potential for a positive recommendation of
completing GENETIC-AF as a Phase 2B trial, the interim analysis
allows for a recommendation to advance directly to Phase 3 if the
analyzed Phase 2B cohort is responding favorably to treatment, with
retention of these patients’ data in the final Phase 3 analysis.
This approach substantially reduces the number of patients required
to complete Phase 3, potentially allowing for a more rapid
development program devoid of the risks of starting a new trial
dissociated from a positive Phase 2 experience.”
GENETIC-AF Clinical Trial
GENETIC-AF is an adaptive, seamless design Phase
2B/3, 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 currently enrolling
patients in the United States, Canada and Europe.
Phase 2B Interim Efficacy
Analysis
The DSMB will perform a pre-specified interim
analysis of unblinded efficacy data from a minimum of 150 patients
with evaluable data. A randomized patient has evaluable data
either when they experience their first composite endpoint event,
AF/AFL or all-cause mortality, or after completion of the 24-week
primary endpoint follow-up period. The primary analysis will
include data from all randomized subjects at the time of database
lock.
The primary analysis will be conducted to
evaluate the evidence for safety and superior efficacy of Gencaro
versus the active comparator, metoprolol succinate (TOPROL-XL).
The prospectively defined features of this
analysis include an estimate of Gencaro effectiveness relative to
TOPROL-XL and an assessment of safety as characterized by adverse
events. The primary analysis method will generate predictive
probability of success (PPoS) values that will be compared to
prespecified PPoS boundaries constructed from Bayesian statistical
modeling. Prospectively defined PPoS ranges have been
predetermined to define three potential outcomes based on the
projection of the Phase 2B interim results:
1) transition the trial to Phase 3 based on a
likelihood of achieving a statistically significant hazard ratio in
favor of Gencaro (evidence of an effectiveness signal consistent
with pretrial assumptions) and enroll up to a total of 620 patients
(including the Phase 2B patients);2) completion of the Phase 2B
stage of the trial including 24-week follow-up of all randomized
subjects (approximately 250 patients), based on an intermediate
result that is potentially favorable but does not support
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.
ARCA, in collaboration with the trial Steering
Committee, will determine the most appropriate path forward for the
trial based on the DSMB recommendation from this interim
analysis. The unblinded statistical data available to the
DSMB will not be disclosed to the Company or the public.
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. 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 U.S. Food and Drug
Administration (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 data from at least 150 patients will support a
recommendation that the GENETIC-AF trial transition to Phase 3, the
potential timeline for GENETIC-AF trial activities and related
recommendations of the DSMB, potential timing for patient
enrollment in 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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