ARCA biopharma Announces Health Canada Acceptance of Genetic-AF Clinical Trial Application

  ARCA biopharma Announces Health Canada Acceptance of Genetic-AF Clinical
  Trial Application

   Canadian Site Activation for Phase 2B/3 Clinical Trial to Begin in 2014

 Gencaro Potentially the First Genetically-Targeted Cardiovascular Treatment

Business Wire

WESTMINSTER, Colo. -- August 18, 2014

ARCA biopharma, Inc. (Nasdaq: ABIO), a biopharmaceutical company developing
genetically targeted therapies for cardiovascular diseases, today announced
that the Company’s Clinical Trial Application (CTA) for the GENETIC-AF
clinical trial evaluating Gencaro^TM as a potential treatment for atrial
fibrillation (AF) has been accepted by Health Canada. ARCA anticipates that
clinical trial sites in Canada will be active in the fourth quarter of 2014.

Dr. Michael R. Bristow, President and CEO, ARCA biopharma, Inc. (Photo:
Business Wire)

Dr. Michael R. Bristow, President and CEO, ARCA biopharma, Inc. (Photo:
Business Wire)

ARCA is evaluating Gencaro, a pharmacologically unique beta-blocker and mild
vasodilator, as a potential treatment for AF in the Phase 2B/3 GENETIC-AF
clinical trial, which is currently enrolling patients in the United States.
ARCA has identified common genetic variations that it believes predict
individual patient response to Gencaro, giving it potential to be the first
genetically targeted therapy for the prevention of atrial fibrillation.

Dr. Michael R. Bristow, Founder and CEO of ARCA, commented, “At ARCA, we
believe a personalized medicine approach to drug development, tailoring
medical treatment to the individual genetic characteristics of each patient,
can enable more effective therapies, improve patient outcomes and reduce
healthcare costs. If the GENETIC-AF trial successfully confirms the atrial
fibrillation data analysis from a prior Phase 3 clinical trial, Gencaro has
the potential to be the first genetically targeted treatment for the
prevention of this important cardiovascular disorder and provide a much needed
treatment option for patients in an area of high unmet medical need.”

About Atrial Fibrillation (AF)

Atrial fibrillation, the most common sustained cardiac arrhythmia, is
considered an epidemic cardiovascular disease and a major public health
burden. The estimated number of individuals with AF globally in 2010 was 33.5
million. According to the 2014 American Heart Association report on Heart
Disease and Stroke Statistics, the estimated number of individuals with AF in
the U.S. in 2010 ranged from 2.7million to 6.1million people.
Hospitalization rates for AF increased by 23% among US adults from 2000 to
2010 and hospitalizations account for the majority of the economic cost burden
associated with AF.

AF is a disorder in which the normally regular and coordinated contraction
pattern of the heart’s two small upper chambers (the atria) becomes irregular
and uncoordinated. The irregular contraction pattern associated with AF causes
blood to pool in the atria, predisposing the formation of clots potentially
resulting in stroke. AF increases the risk of mortality and morbidity due to
stroke, congestive heart failure and impaired quality of life. The approved
therapies for the treatment or prevention AF have certain disadvantages in
patients with heart failure and/or reduced left ventricular ejection fraction
(HFREF) patients. These include toxic or cardiovascular adverse effects, and
most of the approved drugs for AF are contra indicated or have warnings in
their prescribing information for such patients. The Company believes there is
an unmet medical need for new AF treatments that have fewer side effects than
currently available therapies and are more effective, particularly in HFREF
patients.

GENETIC-AF Clinical Trial

GENETIC-AF is a Phase 2B/3, multi-center, randomized, double-blind clinical
trial comparing the safety and efficacy of Gencaro to Toprol-XL for prevention
of symptomatic AF/atrial flutter in HFREF patients. ARCA plans to enroll only
patients with the genetic variant of the beta-1 cardiac receptor which the
Company believes responds most favorably to Gencaro. GENETIC-AF has an
adaptive design, under which the Company initiated the trial as a Phase 2B
trial in approximately 200 patients. The GENETIC-AF Data Safety Monitoring
Board (DSMB) will analyze certain data from the Phase 2B portion of the trial
and recommend, based on a comparison to the pre-trial statistical assumptions,
whether the trial should proceed to Phase 3 and seek to enroll an additional
420 patients.

The AF indication for Gencaro was chosen based on clinical data from the
previously conducted Phase 3 heart failure trial of 2,708 patients (the BEST
trial). The Company believes data from the BEST trial indicate that Gencaro
may have a genetically regulated effect in reducing or preventing AF, whereas
the Company believes the therapeutic benefit of Toprol-XL does not appear to
be enhanced in patients with this genotype. A retrospective analysis of data
from the BEST trial shows that the entire cohort of patients in the BEST trial
treated with Gencaro had a 41% reduction in the risk of new onset AF
(time-to-event) compared to placebo (p = 0.0004). In the BEST DNA substudy,
patients with the beta-1 389 arginine homozygous genotype experienced a 74% (p
= 0.0003) reduction in risk of AF when receiving Gencaro, based on the same
analysis.

About ARCA biopharma

ARCA biopharma is dedicated to developing genetically-targeted therapies for
cardiovascular diseases. The Company's lead product candidate, Gencaro^TM
(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 www.arcabiopharma.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,
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. 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:
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. These and other
factors are identified and described in more detail in ARCA’s filings with the
SEC, including without limitation the Company’s annual report on Form 10-K for
the year ended December 31, 2013, and subsequent filings. The Company
disclaims any intent or obligation to update these forward-looking statements.

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Contact:

ARCA biopharma
Investor & Media Contact:
Derek Cole, 720-940-2163
derek.cole@arcabiopharma.com
 
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