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GENETIC-AF Trial Evaluating Gencaro as a Potential Treatment for
Atrial Fibrillation
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Outcome of Interim Analysis of Phase 2B Efficacy Data Anticipated in
the First Half of 2017
ARCA biopharma, Inc. (Nasdaq: ABIO), a biopharmaceutical company
developing genetically-targeted therapies for cardiovascular diseases,
today reported financial results for the year ended December 31, 2015,
and provided a business update.
“During 2015, we made good progress implementing our corporate strategy
based on the belief that a precision 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,” commented Dr.
Michael Bristow, ARCA’s President and CEO. “In the ongoing GENETIC-AF
clinical trial, we are evaluating Gencaro as potentially the first
genetically-targeted treatment for atrial fibrillation. We believe we
are on track to enroll sufficient patients by year-end for the DSMB to
begin the pre-specified interim analysis with the outcome of the
analysis anticipated in the first half of 2017.”
GENETIC-AF Clinical Trial
GENETIC-AF is a Phase 2B/Phase 3, multi-center, randomized,
double-blind, adaptive design clinical trial comparing the safety and
efficacy of Gencaro to Toprol-XL (metoprolol succinate) for the
treatment of atrial fibrillation (AF) in a combined total of
approximately 620 patients. Eligible patients will have heart failure
with reduced left ventricular ejection fraction (HFREF), have a history
of paroxysmal AF (episodes lasting 7 days or less) or persistent AF
(episodes lasting more than 7 days and less than 1 year) in the past 6
months, and have the beta-1 389 arginine homozygous genotype that the
Company believes responds most favorably to Gencaro. The primary
endpoint of the study is time to first event of symptomatic AF/atrial
flutter (AFL) or all-cause mortality. The combined Phase 2B/Phase 3
trial is designed for 90 percent power at a p-value of less than 0.01
significance level to detect a 25 percent reduction in the primary
endpoint for patients in the Gencaro arm compared to patients in the
Toprol-XL arm. The trial is currently enrolling patients in the United
States and Canada.
The GENETIC-AF Data Safety Monitoring Board (DSMB) will conduct a
pre-specified interim analysis of study endpoints for efficacy, safety
and futility to recommend whether the trial should proceed to Phase 3.
The DSMB will make its recommendation based on a predictive probability
analysis of certain trial data after at least 150 patients have
evaluable endpoint data. An enrolled patient has evaluable endpoint data
either when they experience their first endpoint event, or after they
complete the 24-week follow up period. The DSMB interim analysis will
focus on analyses of the AF/AFL endpoints in the trial using both
clinical-based intermittent monitoring and device-based continuous
monitoring techniques. Should the DSMB interim analysis indicate that
the data are consistent with pre-trial statistical assumptions and the
potential for achieving statistical significance for the Phase 3
endpoint, the DSMB may recommend that the study proceed to Phase 3. The
DSMB may also halt the study for futility. Based on the current
enrollment rate, the Company expects to enroll at least 150 patients by
the end of 2016. The Company expects the outcome of the DSMB interim
analysis and recommendation regarding the potential transition to Phase
3 in the first half of 2017.
In February 2016, the GENETIC-AF protocol was amended to simplify
certain operational aspects of the trial. The Company believes these
modifications will facilitate site recruitment and enrollment in
existing trial sites and potential sites in European countries, where
the Company anticipates expanding the study to support both the later
portion of Phase 2B, as well as the potential Phase 3 portion of the
trial. The Company believes inclusion of European investigative sites
may support potential European regulatory submissions and partnering
discussions. The Company received no objection from the United States
Food and Drug Administration (FDA) or Health Canada on this protocol
amendment.
2015 Summary Financial Results
Cash and cash equivalents totaled $38.8 million as of December
31, 2015, compared to $15.4 million as of December 31, 2014. The Company
believes that its current cash and cash equivalents will be sufficient
to fund its operations, at its projected cost structure, through at
least the end of 2017.
Research and development (R&D) expenses for the year ended
December 31, 2015 totaled $7.1 million compared to $5.6 million for
2014. The $1.4 million increase in research and development expenses in
2015 as compared to 2014 was primarily due to increased clinical
expenses for the GENETIC-AF clinical trial, increased R&D personnel
costs and increased manufacturing process development costs. The Company
expects R&D expenses in 2016 to be higher than 2015 as it activates new
clinical sites and enrolls additional patients in the GENETIC-AF
clinical trial.
General and administrative (G&A) expenses for the year ended
December 31, 2015 were $4.4 million compared to $4.1 million in 2014.
The approximately $325,000 increase in G&A expenses during 2015 was
comprised primarily of increased personnel costs, franchise taxes and
outside services related to the Company’s reverse stock split, partially
offset by decreased non-cash, stock-based compensation expense,
insurance and facility costs. The Company expects G&A expenses in 2016
to be consistent with those in 2015 as it maintains administrative
activities to support the GENETIC-AF clinical trial.
Total operating expenses for the year ended December 31, 2015
were $11.5 million compared to $9.7 million in 2014. The increase in
total operating expenses for 2015 was primarily due to the increase in
R&D expense due to the increased clinical expense of the GENETIC-AF
clinical trial.
Net loss was $11.4 million, or $1.82 per share, for 2015 compared
to $9.7 million, or $3.31 per share, for 2014.
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 2016 American Heart Association report on
Heart Disease and Stroke Statistics, the estimated number of individuals
with AF in the United States in 2010 ranged from 2.7 million to
6.1 million people. Hospitalization rates for AF increased by 23% among
U.S. adults from 2000 to 2010 and hospitalizations account for the
majority of the economic cost burden associated with AF.
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, 2015, and subsequent filings. The Company
disclaims any intent or obligation to update these forward-looking
statements.
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ARCA BIOPHARMA, INC. BALANCE SHEET DATA (in
thousands)
|
|
|
|
|
|
|
|
|
|
|
|
|
December 31, 2015
|
|
|
December 31, 2014
|
|
Cash and cash equivalents
|
|
|
$
|
38,802
|
|
|
$
|
15,354
|
|
Working capital
|
|
|
$
|
37,412
|
|
|
$
|
14,100
|
|
Total assets
|
|
|
$
|
39,574
|
|
|
$
|
16,132
|
|
Total stockholders’ equity
|
|
|
$
|
38,070
|
|
|
$
|
14,741
|
|
|
|
|
|
|
|
|
|
|
|
ARCA BIOPHARMA, INC. STATEMENTS OF OPERATIONS AND
COMPREHENSIVE LOSS
|
|
|
|
|
|
|
|
Years Ended December 31,
|
|
|
|
2015
|
|
|
2014
|
|
|
|
(in thousands, except share and per share amounts)
|
Costs and expenses:
|
|
|
|
|
|
|
|
|
Research and development
|
|
|
$
|
7,063
|
|
|
|
$
|
5,625
|
|
General and administrative
|
|
|
|
4,392
|
|
|
|
|
4,068
|
|
Total costs and expenses
|
|
|
|
11,455
|
|
|
|
|
9,693
|
|
Loss from operations
|
|
|
|
(11,455
|
)
|
|
|
|
(9,693
|
)
|
|
|
|
|
|
|
|
|
|
Interest and other income
|
|
|
|
14
|
|
|
|
|
7
|
|
Interest expense
|
|
|
|
(4
|
)
|
|
|
|
(3
|
)
|
Net loss and comprehensive loss
|
|
|
$
|
(11,445
|
)
|
|
|
$
|
(9,689
|
)
|
|
|
|
|
|
|
|
|
|
Net loss available to common stockholders per share:
|
|
|
|
|
|
|
|
|
Basic and diluted
|
|
|
$
|
(1.82
|
)
|
|
|
$
|
(3.31
|
)
|
Weighted average shares outstanding:
|
|
|
|
|
|
|
|
|
Basic and diluted
|
|
|
|
6,289,305
|
|
|
|
|
2,928,746
|
|
|
|
|
|
|
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