GENETIC-AF Phase 2B/3 Clinical Trial
Evaluating Gencaro as Potentially First Genetically-Targeted
Treatment for Atrial Fibrillation in Heart Failure Patients
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Outcome of GENETIC-AF Phase 2B Interim
Efficacy Analysis Anticipated in the Third Quarter of 2017
ARCA biopharma, Inc. (Nasdaq: ABIO), a biopharmaceutical company
applying a precision medicine approach to developing
genetically-targeted therapies for cardiovascular diseases, today
reported financial results for the year ended December 31, 2016,
and provided a business update.
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Dr. Michael Bristow, President and CEO,
ARCA biopharma, Inc. (Photo: Business Wire)
“During 2016, we continued to make progress advancing the
GENETIC-AF clinical trial evaluating Gencaro as potentially the
first genetically-targeted treatment for atrial fibrillation,”
commented Dr. Michael Bristow, ARCA’s President and Chief Executive
Officer. “The American Heart Association estimates that
approximately 5.2 million Americans had atrial fibrillation in
2015, with medical and indirect costs totaling an estimated $31
billion. We believe that a precision medicine approach to drug
development, tailoring medical treatment to the individual genetic
characteristics of each patient, can potentially enable more
effective therapies, improve patient outcomes and reduce healthcare
costs. The GENETIC-AF trial interim efficacy analysis outcome
expected in the third quarter of this year will be a major
milestone for ARCA, the Gencaro development program and our
targeted approach to cardiovascular drug development.”
2016 Summary Financial Results
Cash, cash equivalents and marketable securities totaled
$23.5 million as of December 31, 2016, compared to $38.8 million as
of December 31, 2015. ARCA believes that its current cash, cash
equivalents and marketable securities will be sufficient to fund
its operations, at its projected cost structure, through the end of
2017.
Research and development (R&D) expenses for the year
ended December 31, 2016 totaled $12.3 million compared to $7.1
million for 2015. The $5.3 million increase in research and
development expenses in 2016 as compared to 2015 was primarily due
to increased expenses for the GENETIC-AF clinical trial, including
contract research organization costs, clinical site initiation and
monitoring activities, patient visit costs and increased costs to
support expanding the trial into Europe. The Company expects
R&D expenses in 2017 to be higher than 2016 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, 2016 were $4.3 million compared to $4.4
million in 2015. The decrease in expenses during 2016 was comprised
primarily of 2015 costs related to a reverse stock split in
September 2015, with no corresponding transaction in 2016, and
lower corporate franchise taxes in 2016 as compared to 2015. ARCA
expects G&A expenses in 2017 to be consistent with those in
2016 as it maintains administrative activities to support the
GENETIC-AF clinical trial.
Total operating expenses for the year ended December 31,
2016 were $16.6 million compared to $11.5 million in 2015. The
increase in total operating expenses for 2016 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 $16.4 million, or $1.81 per share, for 2016
compared to $11.4 million, or $1.82 per share, for 2015.
GENETIC-AF Clinical Trial
GENETIC-AF is an adaptive, seamless design Phase 2B/3,
multi-center, randomized, double-blind, clinical superiority trial
comparing the safety and efficacy of Gencaro to TOPROL-XL
(metoprolol succinate) for the treatment and prevention of
recurrent atrial fibrillation or flutter (AF/AFL) in heart failure
patients with reduced left ventricular ejection fraction (HFrEF).
Eligible patients will have HFrEF, 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 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/AFL
or all-cause mortality. The trial is currently enrolling patients
in the United States, Canada and Europe.
Phase 2B Interim Efficacy Analysis
GENETIC-AF, as an adaptive seamless design Phase 2B/3
superiority trial, includes an interim data analysis of efficacy
and safety. The GENETIC-AF Data and Safety Monitoring Board (DSMB)
will perform a pre-specified interim analysis of unblinded efficacy
data when at least 150 patients have evaluable data. A randomized
patient has evaluable data either when they experience their first
composite endpoint event, AF/AFL or all-cause mortality, or after
completion of the 24-week primary endpoint follow-up period. The
analysis will be conducted for detection of evidence of safety and
superior efficacy of Gencaro versus the active comparator,
metoprolol succinate (TOPROL-XL).
The prospectively defined features of this analysis include an
estimate of Gencaro effectiveness relative to TOPROL-XL and an
assessment of safety as characterized by adverse events. The
relative benefit estimate will utilize Bayesian statistical methods
to calculate the predictive probability of the Phase 3 patient
cohort hazard ratio (a measure of an effect of an intervention on
an outcome of interest over time) based on the interim Phase 2B
data. Prospectively defined ranges of predictive probabilities have
been predetermined to define three potential outcomes based on the
projection of the Phase 2B interim results:
1) transition the trial to Phase 3 based on a
likelihood of achieving a statistically significant hazard ratio in
favor of Gencaro (evidence of an effectiveness signal consistent
with pretrial assumptions) and enroll up to a total of 620 patients
(including the Phase 2B patients); 2) completion of the Phase 2B
stage of the trial including 24-week follow-up of all randomized
subjects (approximately 250 patients), based on an intermediate
result that is potentially favorable but does not support
transition of the trial to Phase 3 or; 3) immediate termination of
the trial due to futility.
ARCA, in collaboration with the GENETIC-AF Steering Committee,
will determine the most appropriate path forward for the trial
based on the DSMB recommendation from this interim analysis and the
Company’s available capital. The unblinded statistical data
available to the DSMB will not be disclosed to ARCA or the public.
ARCA projects that the outcome of the DSMB interim analysis and
recommendation will be available in the third quarter of 2017.
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 2017
American Heart Association report on Cardiovascular Disease,
approximately 5.2 million people in the United States had atrial
fibrillation in 2015. 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 through a precision medicine
approach to drug development. ARCA’s lead product candidate,
GencaroTM (bucindolol hydrochloride), is an investigational,
pharmacologically unique beta-blocker and mild vasodilator being
developed for the potential treatment of patients with atrial
fibrillation and chronic heart failure. 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.arcabio.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 and related recommendations of the
DSMB, the potential that the data from at least 150 patients will
support a recommendation that the GENETIC-AF trial transition to
Phase 3, or completion of Phase 2B, 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, 2016, and subsequent
filings. The Company disclaims any intent or obligation to update
these forward-looking statements.
ARCA BIOPHARMA, INC.
BALANCE SHEET DATA
(in thousands)
December 31,
2016
December 31,
2015
Cash, cash equivalents & marketable securities $23,515 $38,802
Working capital $19,049 $37,412 Total assets $24,629 $39,574 Total
stockholders’ equity $22,194 $38,070
ARCA BIOPHARMA, INC.
STATEMENTS OF OPERATIONS AND
COMPREHENSIVE LOSS
Years Ended December 31, 2016
2015 (in thousands, except share
and per share amounts)
Costs and expenses: Research and development $ 12,348 $ 7,063
General and administrative 4,265 4,392 Total costs
and expenses 16,613 11,455 Loss from operations
(16,613
)
(11,455 ) Interest and other income 169 14 Interest
expense — (4 ) Net loss $ (16,444 ) $ (11,445 )
Change in unrealized loss on marketable securities
(19 ) — Comprehensive loss $ (16,463 ) $ (11,445 )
Net loss per share: Basic and diluted $ (1.81 ) $ (1.82 ) Weighted
average shares outstanding: Basic and diluted 9,067,438 6,289,305
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ARCA biopharmaInvestor & Media Contact:Derek
Cole, 720-940-2163derek.cole@arcabio.com
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