ARCA biopharma, Inc. (Nasdaq: ABIO), a biopharmaceutical company
developing genetically-targeted therapies for cardiovascular diseases,
today announced that the U.S. Food and Drug Administration (FDA) Office
of Orphan Products Development has granted orphan drug designation to
rNAPc2 as a potential treatment of viral hemorrhagic fever post-exposure
to Ebola virus. The drug candidate has previously demonstrated
post-exposure efficacy in non-human primate models of Ebola hemorrhagic
fever virus (HFV). rNAPc2 was originally developed as a cardiovascular
therapy for thrombosis and other indications. As a result, it has an
extensive human clinical record, and has been safely tested in over 700
human patients in nine Phase 1 and 2 clinical trials for cardiovascular
disease.
Orphan drug designation is granted by the FDA Office of Orphan Products
Development to novel drugs or biologics that treat rare diseases or
conditions affecting fewer than 200,000 individuals in the U.S. The
designation provides the drug developer with a seven-year period of U.S.
marketing exclusivity upon marketing approval, as well as certain
financial incentives that can help support its development.
About rNAPc2
rNAPc2 (Recombinant Nematode Anticoagulation Protein c2) is a potent and
selective inhibitor of tissue factor (TF), the protein responsible for
initiating the extrinsic coagulation pathway, the primary coagulation
mechanism in humans. The rationale for rNAPc2 as a HFV therapy arises
from the role of TF in HFV mediated disseminated intravascular
coagulation (DIC), an often fatal complication of the HFV disease
syndrome that leads to spontaneous hemorrhage. Previous research studies
have shown that abnormal activation of TF is a common element of the
host response to infection from Ebola, Marburg and other HFVs. This
activation of TF results in a systemic consumption coagulopathy and
related inflammation, which is believed to be partially responsible for
the high morbidity and mortality caused by these infections. rNAPc2 was
identified as a potential therapeutic for HFV infection because of its
unique mechanism of action in inhibiting TF.
Previously, pilot studies of rNAPc2 in non-human primates demonstrated
potential efficacy against two of the most deadly known HFVs, Ebola and
Marburg. The results of these studies demonstrated that, when
administered as a post-exposure therapy, rNAPc2 showed evidence of
efficacy in improving survival and inhibiting the DIC process.
Additionally, in these studies, rNAPc2 demonstrated anti-inflammatory
and anti-viral properties.
ARCA is currently exploring options for the development of rNAPc2
including seeking development partners, out-licensing the compound and
applying for grant or government funding. ARCA is not actively
developing rNAPc2 for any indications and has no plans to begin such
development without securing a partnership, an out-licensing agreement
or receiving grant or government funding.
rNAPc2 Publications
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Geisbert TW, Hensley LE, Jahrling PB, Larsen T, Geisbert JB, Paragas
J, Young HA, Fredeking TM, Rote WE, Vlasuk GP. Treatment of Ebola
virus infection with a recombinant inhibitor of factor VIIa/tissue
factor: a study in rhesus monkeys. Lancet. 2003;362(9400):1953-8.
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Geisbert TW, Daddario-DiCaprio KM, Geisbert JB, Young HA, Formenty P,
Fritz EA, Larsen T, Hensley LE. Marburg virus Angola infection of
rhesus macaques: pathogenesis and treatment with recombinant nematode
anticoagulant protein c2. J Infect Dis. 2007;196 Suppl 2:S372-81.
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, the potential for rNAPc2 to be developed for, or to
effectively treat hemorrhagic fever viruses, including Ebola, 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.
Copyright Business Wire 2014