ARCA biopharma, Inc. (Nasdaq: ABIO), a biopharmaceutical company
developing genetically-targeted therapies for cardiovascular diseases,
today announced that the U.S. Food and Drug Administration (FDA) has
designated as a Fast Track development program the investigation of
GencaroTM for the prevention of atrial fibrillation/atrial
flutter in a genetically modified heart failure population (heart
failure patients with reduced left ventricular ejection fraction,
HFREF). Gencaro is the Company’s investigational, pharmacologically
unique beta-blocker and mild vasodilator.
Dr. Michael Bristow, CEO, ARCA biopharma (Photo: Business Wire)
According to the FDA’s Fast Track Guidance document, Fast Track programs
are designed to facilitate the development and expedite the review of
new drugs that are intended to treat serious or life-threatening
conditions and that demonstrate the potential to address unmet medical
needs.
Gencaro is currently being evaluated as a potential treatment for atrial
fibrillation in a genetically-defined heart failure (HFREF) population
in GENETIC-AF, a Phase 2B/3 adaptive design clinical trial. ARCA
anticipates that enrollment of approximately 200 patients in the Phase
2B portion of the trial will be completed by the end of 2016.
“We view Fast Track designation for the Gencaro development program as
an important acknowledgement of the need for advancements in the
treatment of atrial fibrillation in heart failure patients,” said
Michael R. Bristow, President and Chief Executive Officer of ARCA.
“Atrial fibrillation afflicts over 2.7 million people in the United
States with 250,000 to 500,000 new cases diagnosed each year. We believe
we have a significant opportunity to improve the treatment options for
heart failure patients living with atrial fibrillation.”
Fast Track drug development designation was included in the FDA
Modernization Act of 1997 (FDAMA) as a formal process to enhance
interactions with the FDA during drug development. A drug development
program with Fast Track designation is eligible for consideration for
some or all of the following programs for expediting development and
review: scheduled meetings to seek FDA input into development plans,
priority review of the New Drug Application (NDA), the option of
submitting portions of an NDA for review prior to submission of the
complete application and potential accelerated approval.
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 2015 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 million to 6.1 million
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.
About Pharmacogenomics
Pharmacogenomics is the study of genetic polymorphisms that underlie
individual differences in responses to therapeutics drugs.
Pharmacogenomics includes identifying candidate genes and polymorphisms,
correlating these polymorphisms with possible therapies, predicting drug
response and clinical outcomes, reducing adverse events and selection,
and selecting dosing of therapeutic drugs on the basis of genotype. One
goal of pharmacogenomics is to customize drugs for defined
sub-populations of patients.
A DNA sub-study of patients from the BEST Phase 3 heart failure
mortality trial of Gencaro indicated that the combinations of beta-1 389
and alpha-2C polymorphisms in individual patients in the trial appeared
to influence the response to Gencaro.
About ARCA biopharma
ARCA biopharma is dedicated to developing genetically-targeted therapies
for cardiovascular diseases. The Company's lead product candidate,
GencaroTM (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, potential timeline for GENETIC-AF trial activities, 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, 2014, and subsequent filings. The Company disclaims
any intent or obligation to update these forward-looking statements.
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