GENETIC-AF Trial Evaluating Gencaro ™ as
Potential Treatment for Atrial Fibrillation
Protocol Amended to Broaden Trial Population
and Increase Enrollment Rate
Phase 2B of GENETIC-AF Anticipated to
Complete Enrollment by Year-End 2016
ARCA biopharma, Inc. (Nasdaq: ABIO), a biopharmaceutical company
developing genetically-targeted therapies for cardiovascular
diseases, today provided an update on GENETIC-AF, the Company’s
Phase 2B/3 clinical trial evaluating Gencaro (bucindolol
hydrochloride) as a potential treatment for atrial fibrillation
(AF).
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 is being developed as a
potential treatment for 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.
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 (metoprolol succinate) for the treatment of AF
in patients with heart failure and left ventricular systolic
dysfunction (HFREF patients). The primary endpoint of GENETIC-AF is
time to symptomatic AF/atrial flutter (AFL). ARCA is enrolling only
HFREF patients with the genetic variant of the cardiac beta-1
adrenergic receptor, which the Company believes responds most
favorably to Gencaro, the 389 arginine homozygous genotype (ADRB1
Arg389Arg). GENETIC-AF has an adaptive design, under which the
Company initiated the trial as a Phase 2B trial seeking to enroll
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.
There are currently 38 active clinical trial sites in the United
States and Canada. The Company anticipates that approximately 65
sites will be activated for the Phase 2B portion of GENETIC-AF.
Trial enrollment, the number of patients randomized into one of the
study’s two treatment arms, has not met the Company’s original
projections, with the trial having screened 37 patients who met the
general clinical inclusion criteria and signed informed consent for
genotyping, resulting in 12 patients who were randomized into the
trial. The percentage of screened patients who have the targeted
genotype is consistent with pre-trial assumptions of approximately
50%. The Company believes the original trial eligibility criteria
overly restricted the potential pool of appropriate patients for
the trial and contributed to the number of screened patients who
subsequently became ineligible for randomization. To address these
issues, the Company, in consultation with the GENETIC-AF Steering
Committee comprised of experts in the fields of electrophysiology,
heart failure and clinical trial methodology, has implemented
amendments to the trial protocol that the Company believes may
expand the eligible target population, increase the patient
screening and enrollment rate, and simplify trial procedures.
Under the revised protocol, patients in sinus rhythm who have
experienced symptomatic AF in the past 120 days are now eligible
for inclusion in the trial, as are patients with AF episodes
lasting 7 days or less (i.e., paroxysmal AF). Previously, these
patients were not eligible to be enrolled in the trial. The Company
believes this expanded target population, which is substantially
larger than the original target population, has the potential to
improve trial screening and enrollment rates and broaden the
potential commercial market for Gencaro should it achieve
regulatory approval in the future. The amendments to the protocol
do not fundamentally alter or impact the original endpoints of the
clinical trial. Based on the projected impact of the expanded
patient population and the current enrollment rate, the Company
anticipates that the enrollment of 200 patients for the Phase 2B
portion of the trial may be completed by the end of 2016.
The Company has met with the U.S. Food and Drug Administration
to confirm the acceptability of the amendments to the protocol and
received no objections.
The revised clinical trial protocol has been distributed to
participating clinical trial sites for review by their respective
institutional review boards (IRB), or similar committee. IRBs
review and approve clinical trial protocols for trials in which
their organizations participate, a process with timelines that vary
significantly from IRB to IRB and is out of the Company’s control.
The Company does not anticipate a significant change to the current
enrollment rate until the revised protocol is adopted by clinical
trial sites. The Company anticipates the first trial sites to be
approved, trained and operating under the revised protocol in June
2015.
“We greatly appreciate the tremendous support and feedback from
the clinical investigator community participating in GENETIC-AF,”
said Dr. Michael R. Bristow, President and Chief Executive Officer
of ARCA. “The adaptive nature of the clinical trial design for
GENETIC-AF is an important feature that allows us to adapt certain
aspects of the trial without compromising basic assumptions and the
central goal, which is to test the hypothesis that Gencaro is safe
and effective in preventing recurrent AF in a genetically defined
HFREF population. We believe we have a significant opportunity to
improve the treatment options for patients living with atrial
fibrillation and look forward to providing future updates on the
progress of GENETIC-AF.”
GENETIC-AF Trial Phase 2B Projected Timeline
-- Revised protocol distributed to participating clinical
trial sites March 2015 -- GENETIC-AF Trial
Investigators meeting and training May 2015 -- First trial sites
operating under revised protocol June 2015 -- Approximately 65
clinical trial sites active in U.S. & Canada Q4 2015 -- Trial
Enrollment Completion YE 2016 -- DSMB Interim Analysis Decision 1H
2017
The Company’s forecast of the time periods to achieve these
milestones is a forward-looking statement and involves risks and
uncertainties, and actual results are likely to vary as a result of
a number of factors, including the factors discussed in “Risk
Factors” in the Company’s periodic SEC filings.
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 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, 2013, and subsequent
filings. The Company disclaims any intent or obligation to update
these forward-looking statements.
ARCA biopharma, Inc.Investor & Media
Contact:Derek Cole,
720-940-2163derek.cole@arcabiopharma.com
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