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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.
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 shareand
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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version on businesswire.com: http://www.businesswire.com/news/home/20160317005928/en/
ARCA biopharma, Inc.Investor & Media
Contact:Derek Cole,
720-940-2163derek.cole@arcabiopharma.com
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