ARCA biopharma Provides Business Update

  ARCA biopharma Provides Business Update

    GENETIC-AF Trial Evaluating Gencaro™ as Potential Treatment for Atrial
         Fibrillation Planned to Begin Patient Enrollment in Q1 2014

 ARCA and Medtronic, Inc. are Collaborating on Phase 2B Portion of GENETIC-AF

Business Wire

BROOMFIELD, Colo. -- July 17, 2013

ARCA biopharma, Inc. (Nasdaq: ABIO), a biopharmaceutical company developing
genetically-targeted therapies for cardiovascular diseases, today provided a
general business update and clinical development guidance.

ARCA was founded on the belief that 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. ARCA’s lead development program
is intended to be a direct implementation of those ideas. Gencaro (bucindolol
hydrochloride) is being developed as a potential treatment for atrial
fibrillation (AF). 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. AF is
considered an epidemic cardiovascular disease with an estimated prevalence of
at least 2.7 million Americans in 2010.

“We believe we have a significant opportunity to improve the treatment options
for patients living with atrial fibrillation,” said Dr. Michael R. Bristow,
President and Chief Executive Officer of ARCA. “With the completion of our
recent capital raise, and the support of Medtronic, we now have the resources
to advance Gencaro into Phase 2B clinical evaluation. We believe there is an
unmet medical need for new atrial fibrillation treatments that have fewer side
effects than currently available therapies.”

In June, the Company completed an equity offering, raising net proceeds of
approximately $18 million. As of June 30, 2013, the Company had approximately
$20.4 million in cash and cash equivalents.

ARCA and Medtronic, Inc. (NYSE:MDT), a leader in medical technologies to
improve the treatment of chronic diseases, including cardiac rhythm disorders,
have entered into a collaboration agreement for the Phase 2B portion of
GENETIC-AF, the Company’s Phase 2B/3 trial evaluating Gencaro ^ as a potential
treatment for AF.

GENETIC-AF Clinical Trial

GENETIC-AF is planned as a Phase 2B/3, multi-center, randomized, double-blind
clinical trial comparing Gencaro to metoprolol CR/XL for prevention of AF in
patients with heart failure and 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 study in approximately 200 patients and then,
depending on the results of an interim analysis by the trial Data Safety
Monitoring Board (DSMB), expand the trial to a Phase 3 study by enrolling an
estimated additional 420 patients. The Company now anticipates that patient
enrollment in GENETIC-AF will begin in the first quarter of 2014.

Under the collaboration with Medtronic, ARCA plans to conduct a substudy that
will include continuous monitoring of the cardiac rhythms of all 200 patients
enrolled during the Phase 2B portion of GENETIC-AF. Each patient 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^® loop recorder. The collaboration substudy will measure AF
burden, defined as a patient’s actual time in AF regardless of symptoms. For
the DSMB interim analysis, AF burden and the primary endpoint of the study,
time to recurrence of symptomatic AF after electrical cardioversion, or death,
will be reviewed by the DSMB to determine if there is sufficient potential for
a statistically significant efficacy signal to be determined for all patients
enrolled in the Phase 2B/3 study.

Upcoming GENETIC-AF Trial Implementation Milestones

  -   GENETIC-AF Implementation Team Additions                July 2013
    -     GENETIC-AF Steering Committee Formalization               July 2013
    -     GENETIC-AF Data Safety Monitoring Board Formalization     Aug 2013
    -     Gencaro AF Investigational New Drug (IND) Application     Q3 2013
          Submission
    -     GENETIC-AF Clinical Research Organization Selection       Q3/Q4 2013
    -     GENETIC-AF Patient Enrollment Initiation                  Q1 2014
                                                                    

Our forecast of the time periods to achieve these milestones is a
forward-looking statement and involves risks and uncertainties, and actual
results could vary as a result of a number of factors, including the factors
discussed in “Risk Factors” in our periodic filings.

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 post-hoc review of patient forms from the from
BEST trial, a Phase 3 trial in 2,708 patients with advanced heart failure,
indicate 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 comparing beta-blockers to placebo have detected a positive signal for
prevention of AF, with an event rate reduction averaging approximately 27%,
although AF had not been a pre-specified primary or secondary endpoint in
these studies. Currently, no beta-blocker has been approved by the U.S. Food
and Drug Administration for the prevention of AF in heart failure patients.

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 Phase 2B portion 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
upcoming GENETIC-AF trial implementation milestones, 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, 2012, and subsequent filings. The Company
disclaims any intent or obligation to update these forward-looking statements.

Contact:

ARCA biopharma, Inc.
Derek Cole, 720-940-2163
Investor Relations Advisory Solutions
derek.cole@arcabiopharma.com
 
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