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ARCA biopharma Announces 175th Patient Randomized into the GENETIC-AF Phase 2B/3 Clinical Trial


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 175th patient has been randomized into
GENETIC-AF, a seamless design Phase 2B/3 clinical trial evaluating
Gencaro™ (bucindolol hydrochloride) as a potential treatment for atrial
fibrillation (AF).

"We are pleased with the increased rate of enrollment in the GENETIC-AF
clinical trial and believe it is reflective of the unmet need for new AF
treatments for patients with heart failure," commented Dr. Michael
Bristow, ARCA's President and CEO. "We would like to express our
gratitude to the patients and clinical sites that are participating in
this potentially groundbreaking clinical trial evaluating Gencaro as
possibly the first genetically-targeted treatment for atrial

The GENETIC-AF Data Safety Monitoring Board (DSMB) will conduct a Phase
2B interim efficacy, safety and futility analysis of evaluable data from
at least 150 patients. The Company expects the outcome of this interim
analysis in the third quarter of 2017.

Phase 2B Interim Efficacy Analysis

GENETIC-AF is a seamless Phase 2B/3 adaptive design superiority trial
evaluating the effectiveness of Gencaro for the prevention of recurrent
atrial fibrillation or flutter (AF/AFL) in heart failure patients with
reduced left ventricular ejection fraction (HFrEF). The DSMB will
perform a pre-specified interim analysis of unblinded efficacy data when
at least 150 patients have 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 analysis will be
conducted for detection of evidence of safety and superior efficacy of
Gencaro versus the active comparator, metoprolol succinate (TOPROL-XL).

The prospectively defined features of this analysis include: 1) an
estimate of Gencaro effectiveness relative to TOPROL-XL; and, 2) an
assessment of safety as characterized by adverse events. The relative
benefit estimate will utilize Bayesian statistical methods to calculate
the predictive probability of the Phase 3 patient cohort hazard ratio (a
measure of an effect of an intervention on an outcome of interest over
time) based on the interim Phase 2B data. Prospectively defined ranges
of predictive probabilities have been predetermined to define three
potential outcomes based on the projection of the Phase 2B interim

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.

The Company, 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 and the Company's
available capital. The unblinded statistical data available to the DSMB
will not be disclosed to the Company or the public.

GENETIC-AF Clinical Trial

GENETIC-AF is a seamless Phase 2B/3, adaptive design, multi-center,
randomized, double-blind, superiority clinical trial comparing the
safety and efficacy of Gencaro to Toprol-XL (metoprolol succinate) for
the prevention of recurrent AF/AFL in HFrEF patients. 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 the Company 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 trial is currently enrolling patients
in the United States, Canada and Europe.

About ARCA biopharma

ARCA biopharma is dedicated to developing genetically-targeted therapies
for cardiovascular diseases through a precision medicine approach to
drug development. The Company's lead product candidate, Gencaro™
(bucindolol hydrochloride), is an investigational, pharmacologically
unique beta-blocker and mild vasodilator being developed for atrial
fibrillation. 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. For more information, please visit

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 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 the Company's capital to
support its operations, the potential for genetic variations to predict
individual patient response to Gencaro, Gencaro's potential to treat
atrial fibrillation, future treatment options for patients with atrial
fibrillation, and the potential for Gencaro to be the first
genetically-targeted atrial fibrillation prevention treatment.
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: the Company's financial resources and
whether they will be sufficient to meet the Company's business
objectives and operational requirements; results of earlier clinical
trials may not be confirmed in future trials, the protection and market
exclusivity provided by the Company's intellectual property; risks
related to the drug discovery and the regulatory approval process; and,
the impact of competitive products and technological changes.
and other factors are identified and described in more detail in ARCA's
filings with the Securities and Exchange Commission, including without
limitation the Company's annual report on Form 10-K for the year ended
December 31, 2015, and subsequent filings. The Company disclaims any
intent or obligation to update these forward-looking statements.

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