January 30, 2015 11:58am EST
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MIAMI--(BUSINESS WIRE)--
OPKO Health, Inc. (NYSE:OPK) announced the submission of an
Investigational New Drug Application (IND) to the U.S. Food and Drug
Administration (FDA) to conduct a Phase 2a study of OPKO's long-acting
version of coagulation Factor VIIa (Factor VIIa-CTP) for the treatment
of bleeding episodes in hemophilia A or B patients with inhibitors to
Factor VIII or Factor IX.
Factor VIIa-CTP is a novel, long-acting recombinant Factor VIIa
utilizing OPKO’s proprietary technology to extend its circulatory
half-life without the use of polymers, encapsulation techniques, or
nanoparticles. The technology is based on a naturally occurring peptide,
the C-terminal peptide (CTP) of the beta chain of human chorionic
gonadotropin. The CTP technology is also used in OPKO’s hGH-CTP, its
long-acting recombinant human growth hormone product which is being
evaluated in Phase 3 clinical trials for adults and Phase 2 trials for
children with growth hormone deficiencies. OPKO recently announced a
global agreement with Pfizer for the development and commercialization
of hGH-CTP.
Currently, Factor VIIa therapy is available only as an intravenous (IV)
formulation which, due to Factor VIIa’s short half-life, requires
multiple infusions to treat a bleeding episode. In addition, frequent
infusions are onerous when used as preventative prophylactic therapy,
especially for children.
Pre-clinical studies of intravenous and subcutaneous formulations of our
product in hemophilic animal models demonstrated its duration of action
and significantly increased survival.
“We look forward to beginning clinical trials of Factor VIIa-CTP,”
stated Phillip Frost, M.D., Chairman and CEO of OPKO Health. “A longer
acting Factor VII administered either by IV or subcutaneous
administration, could change the Factor VIIa market by permitting
children and adults to easily self-administer at home on a prophylactic
basis,” continued Dr. Frost.
Factor VIIa-CTP has been granted orphan drug designation in the U.S. and
Europe.
ABOUT OPKO HEALTH, INC.
OPKO is a multinational biopharmaceutical and diagnostics company that
seeks to establish industry-leading positions in large, rapidly growing
markets by leveraging its discovery, development and commercialization
expertise and novel and proprietary technologies. For more information,
visit http://www.opko.com .
This press release contains "forward-looking statements," as that
term is defined under the Private Securities Litigation Reform Act of
1995 (PSLRA), regarding product development efforts and other
non-historical facts about our expectations, beliefs or intentions
regarding our business, technologies and products, financial condition,
strategies or prospects, including statements regarding our expectations
about our long-acting version of clotting Factor VIIa (Factor VIIa-CTP)
for the treatment of bleeding episodes in patients with hemophilia A or
B with inhibitors to Factor VIII or Factor IX, whether Factor VIIa-CTP
has the potential for substantial improvement of the quality of life of
patients, via both IV and subcutaneous (SC) administration, whether
Factor VIIa-CTP will be able to be administered subcutaneously using a
simple injection and allow children and adults with hemophilia to easily
self-administer the drug at home on a prophylactic basis, whether we
will be able to initiate a Phase IIa trial with our IV formulation in
hemophilia patients and the timing for doing so, whether we will be in a
position to be first to market with a longer acting Factor VIIa product,
expectations about market potential for Factor VIIa-CTP, whether we will
be able to successfully develop, obtain approval for and launch sales of
the Factor VIIa-CTP, and the expected completion dates for our trials.
Many factors could cause our actual activities or results to differ
materially from the activities and results anticipated in
forward-looking statements. These factors include those described in our
filings with the Securities and Exchange Commission, as well as risks
inherent in funding, developing and obtaining regulatory approvals of
new, commercially-viable and competitive products and treatments,
including the risks that clinical trials for Factor VIIa-CTP may not be
successful or achieve the expected results or effectiveness, and may not
generate data that would support the approval or marketing of this
product for the indications being studied, that others may develop
products which are superior to Factor VIIa-CTP, and that Factor VIIa-CTP
may not have advantages or prove to be superior over presently marketed
products. In addition, forward-looking statements may also be adversely
affected by general market factors, competitive product development,
product availability, federal and state regulations and legislation, the
regulatory process for new products and indications, manufacturing
issues that may arise, patent positions and litigation, among other
factors. The forward-looking statements contained in this press release
speak only as of the date the statements were made and we do not
undertake any obligation to update forward-looking statements. We intend
that all forward-looking statements be subject to the safe-harbor
provisions of the PSLRA.
OPKO Health, Inc.
Steven D. Rubin, 305-575-4100
or
Adam
Logal, 305-575-4100
Source: OPKO Health, Inc.
Released January 30, 2015
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