ARCA biopharma Announces Screening of First Patient in Phase 2B/3 Clinical Trial Evaluating Gencaro in Atrial Fibrillation

  ARCA biopharma Announces Screening of First Patient in Phase 2B/3 Clinical
  Trial Evaluating Gencaro in Atrial Fibrillation

     Gencaro Potentially First Genetically Targeted Treatment for Atrial
                                 Fibrillation

 Atrial Fibrillation Estimated Global Prevalence of 33.5 Million Patients in
                                     2010

Business Wire

WESTMINSTER, Colo. -- April 17, 2014

ARCA biopharma, Inc. (Nasdaq: ABIO), a biopharmaceutical company developing
genetically targeted therapies for cardiovascular diseases, today announced
that the first patient has been genetically screened in GENETIC-AF, its Phase
2B/3 adaptive design clinical trial. The GENETIC-AF trial will evaluate
Gencaro^TM (bucindolol hydrochloride) as a potential treatment for the
prevention of atrial fibrillation (AF) in patients with heart failure and/or
left ventricular dysfunction who also have a specific genotype of the beta-1
adrenergic receptor (389 arginine homozygous), which is believed to be present
in approximately 50% of the U.S. population. There are currently seven active
clinical trial sites recruiting patients for GENETIC-AF. The Company
anticipates having approximately 50 clinical trial sites for the Phase 2B
portion of the trial. Last week, ARCA hosted the initial GENETIC-AF
Investigator Meeting which brought together the physicians and research
coordinators participating in the trial for information sharing and
study-specific training.

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)

Gencaro is an investigational, pharmacologically unique beta-blocker and mild
vasodilator. ARCA has identified common genetic variations that it believes
may predict individual patient response to Gencaro, giving it the potential to
be the first genetically targeted AF prevention treatment. Currently, no
beta-blocker has been approved by the U.S. Food and Drug Administration for
the prevention of AF.

AF is considered an epidemic cardiovascular disease. 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 2.7 to
6.1 million. AF increases the risk of stroke and may also contribute to
worsening heart failure.

“This is a major milestone for the company and for the development of
Gencaro,” said Dr. Michael R. Bristow, President and Chief Executive Officer
of ARCA. “We believe we have an opportunity to potentially improve the
treatment options for patients at risk for, or living with, atrial
fibrillation by addressing an unmet medical need in patients who also have
heart failure and/or left ventricular dysfunction. The Gencaro development
program, if successful in GENETIC-AF, would also herald a new era of
pharmacogenetically developed cardiovascular drugs.”

ARCA and Medtronic, Inc. (NYSE: MDT), a leader in medical technologies to
improve the treatment of chronic diseases, including cardiac rhythm disorders,
entered into a collaboration agreement in May 2013 to support GENETIC-AF.

Laboratory Corporation of America^® Holdings (LabCorp^®) (NYSE: LH) and ARCA
have collaborated to develop a companion diagnostic for identifying
prospective patients’ genotypes. LabCorp is conducting the genotyping for the
GENETIC-AF trial.

GENETIC-AF Clinical Trial

GENETIC-AF is a Phase 2B/3, multi-center, randomized, double-blind clinical
trial comparing Gencaro to Toprol-XL for prevention of symptomatic AF or
atrial flutter (AFL) in patients with heart failure and/or reduced left
ventricular ejection fraction (HFREF). 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 plans to initiate it as a Phase 2B trial in approximately
200 patients and then, depending on the results of an interim analysis by the
trial Data Safety Monitoring Board (DSMB) and availability of additional
financing, potentially transition the trial to a Phase 3 trial and enroll an
estimated additional 420 patients.

Under the collaboration with Medtronic, ARCA plans to conduct a substudy that
will include continuous monitoring of the cardiac rhythms of patients enrolled
during the Phase 2B portion of GENETIC-AF. These substudy patients will have
heart rhythm monitoring via a Medtronic device, either a previously implanted
cardiac resynchronization or defibrillation device, or a previously or newly
inserted Reveal LINQ^TM Insertable Cardiac Monitor (ICM) System. The substudy
will measure AF burden, defined as a patient’s actual time in AF regardless of
symptoms.

For the DSMB interim analysis, AF event rates, AF burden and components of the
primary endpoint of the study, time to recurrence of symptomatic AF/AFL or
death after electrical cardioversion to sinus rhythm will be reviewed by the
DSMB to determine if there is sufficient potential for a statistically
significant efficacy signal in the overall Phase 2B/3 study population.

Atrial Fibrillation (AF)

Atrial fibrillation 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. These clots may travel from the heart and become lodged in
the arteries leading to the brain and other organs, thereby blocking necessary
blood flow and potentially resulting in stroke. In addition, in patients with
HFREF, new onset AF may also contribute to worsening heart failure and
increased risk of death.

Gencaro Data in Atrial Fibrillation

Clinical data analysis from a review of patient forms from the BEST trial, a
Phase 3 trial in 2,708 patients with advanced heart failure, indicates that
Gencaro may have a potentially enhanced and pharmacogenetically influenced
effect in reducing and preventing AF. In that trial, patients in the Gencaro
arm had a reduction in the risk of new onset AF time to event compared to
patients in the placebo arm of 41% (AF measured as an adverse event/serious
adverse event or as detected on surveillance ECGs, time to event analysis, p =
0.0004). In a 1,040 patient DNA substudy of BEST, Gencaro exhibited potential
pharmacogenetic enhancement and differentiation for AF prevention in patients
with a specific genotype (beta[1]389 arginine homozygous (Arg/Arg) adrenergic
receptor (AR); approximately 47% of the patients). These patients experienced
a 74% (p = 0.0003) reduction in risk of AF versus no detectable reduction in
patients who had alternative genotypes (beta[1]389 Gly carriers; 53% of the
patients) who had received placebo. The Company believes this data supports
the potential ability of Gencaro to prevent AF in patients who have the
genotype the Company believes responds most favorably to Gencaro.

In prior placebo controlled trials of beta-blockers in chronic HFREF, most
studies have detected a positive signal for prevention of AF, with an event
rate reduction averaging approximately 27%. However, AF had not been a
pre-specified primary or secondary endpoint in these studies.

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, the role of AF burden in diagnosis and
treatment of 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:

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