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125th Patient Randomized into
the GENETIC-AF Phase 2B/3 Clinical Trial Evaluating Gencaro as
Potentially First Genetically-Targeted Treatment for Atrial
Fibrillation
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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
developing genetically-targeted therapies for cardiovascular
diseases, today reported financial results for the quarter ended
September 30, 2016, and provided a business update.
“Atrial fibrillation is considered an epidemic cardiovascular
disease impacting an estimated 33 million patients globally,”
commented Dr. Michael Bristow, ARCA’s President and CEO. “Our
on-going GENETIC-AF Phase 2B/3 clinical trial is evaluating
GencaroTM as potentially the first genetically-targeted treatment
for atrial fibrillation. With the recent enrollment of the 125th
patient in the trial, we are approaching two important milestones –
the enrollment of the 150th patient, anticipated in the first
quarter of 2017, and the outcome of the GENETIC-AF Data and Safety
Monitoring Board Phase 2B interim efficacy analysis, projected in
the third quarter of 2017. We believe 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 heart failure with reduced ejection fraction patients.”
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 approximately 620
patients. Eligible patients will have heart failure with reduced
left ventricular ejection fraction (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/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, Canada and certain European
countries. To date, 125 patients have been randomized into the
trial.
The GENETIC-AF Data and Safety Monitoring Board (DSMB) will
conduct a pre-specified interim analysis of study endpoints for
efficacy, safety and futility to recommend whether or not 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. Based on the results of the interim
analysis, the DSMB may recommend that the trial proceed to Phase 3,
the trial be completed as a Phase 2B study, or termination of the
trial due to futility. ARCA, in collaboration with the GENETIC-AF
Steering Committee, will determine the next steps for the trial
based on the DSMB recommendation from this interim analysis and on
the Company’s available financing. Based on ARCA’s current
enrollment projections, the Company now expects to reach 150
patients enrolled in the trial during the first quarter of 2017.
The Company projects that the outcome of the DSMB interim analysis
and recommendation will be available in the third quarter of
2017.
Third Quarter 2016 Summary Financial Results
Cash, cash equivalents and marketable securities totaled
$27.4 million as of September 30, 2016, compared to $38.8
million as of December 31, 2015. The Company believes that its
current cash, cash equivalents and marketable securities will be
sufficient to fund its operations, at its projected cost structure,
through at least the end of 2017. ARCA had approximately 9.1
million outstanding shares of common stock as of September 30,
2016.
Research and development (R&D) expense for the three
months ended September 30, 2016 was $3.7 million compared to $1.7
million for the corresponding period of 2015, an increase of
approximately $2.0 million. R&D expense for the nine months
ended September 30, 2016 was $9.2 million compared to $5.2 million
for the corresponding period of 2015, an increase of approximately
$4.1 million. The increase in R&D expense in the three and nine
month periods ended September 30, 2016 is due, primarily, to the
increased clinical expense of the GENETIC-AF clinical trial. The
Company expects R&D expense 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) expense for the
three months ended September 30, 2016 was $1.0 million compared to
$1.1 million for the corresponding period in 2015. G&A expense
was $3.1 million for the nine months ended September 30, 2016 as
compared to $3.1 million for the corresponding period in 2015. The
Company expects G&A expense in 2016 will be higher than in 2015
as the Company increases administrative activities to support the
GENETIC-AF clinical trial.
Total operating expense for the three months ended
September 30, 2016 was $4.7 million compared to $2.8 million for
the corresponding period in 2015. Total operating expense for the
nine months ended September 30, 2016 was $12.3 million compared to
$8.3 million for the corresponding period in 2015.
Net loss was $4.7 million, or $0.51 per share, for the
third quarter of 2016, compared to $2.8 million, or $0.31 per
share, for the third quarter of 2015. For the first nine months of
2016, net loss was $12.2 million, or $1.35 per share, compared to
$8.3 million, or $1.54 per share, for the first nine months of
2015.
About ARCA biopharma
ARCA biopharma is dedicated to developing genetically-targeted
therapies for cardiovascular diseases through a precision medicine
approach to drug development. 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.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 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)
(unaudited)
September 30,
2016
December 31,
2015
Cash, cash equivalents and marketable securities $27,435 $38,802
Working capital $21,632 $37,412 Total assets $28,679 $39,574 Total
stockholders’ equity $26,480 $38,070
ARCA BIOPHARMA, INC.
STATEMENTS OF OPERATIONS AND
COMPREHENSIVE LOSS
(unaudited)
Three Months Ended Nine Months Ended
September 30, September 30, 2016
2015 2016 2015 (in thousands,
except share and per share amounts) Costs and expenses:
Research and development $ 3,720 $ 1,716 $ 9,227 $ 5,170 General
and administrative 992 1,101 3,094
3,110 Total costs and expenses 4,712 2,817
12,321 8,280 Loss from operations (4,712 )
(2,817 ) (12,321 ) (8,280 ) Interest and other
income 53 4 121 7 Interest expense — (1 ) —
(4 ) Net loss $ (4,659 ) $ (2,814 ) $ (12,200 ) $ (8,277 )
Change in unrealized loss on marketable securities
(19 ) — (6 ) — Comprehensive loss $ (4,678 ) $
(2,814 ) $ (12,206 ) $ (8,277 ) Net loss per share: Basic
and diluted $ (0.51 ) $ (0.31 ) $ (1.35 ) $ (1.54 ) Weighted
average shares outstanding: Basic and diluted 9,068,376 9,034,016
9,062,516 5,358,629
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version on businesswire.com: http://www.businesswire.com/news/home/20161114006118/en/
ARCA biopharmaInvestor & Media Contact:Derek
Cole, 720-940-2163derek.cole@arcabio.com
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