February 23, 2016 8:30am EST
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MIAMI--(BUSINESS WIRE)--
OPKO Health, Inc. (NYSE:OPK) today announced dosing of the first patient
in a Phase 2a study evaluating the safety of a long-acting Factor VIIa
in hemophilia patients. The Phase 2a study is a dose escalation study to
determine safety and explore efficacy endpoints in patients with 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. The study is intended to enroll
24 patients in the United States.
“This is a significant step in the development of another drug candidate
utilizing our CTP technology,” commented Phillip Frost, MD, Chairman and
Chief Executive Officer of OPKO. “Later this year, we expect to initiate
a second Phase 2a trial for Factor VIIa-CTP in a subcutaneous
formulation that, if successful, could provide prophylactic therapy to
prevent bleeding episodes and thereby change the treatment paradigm for
patients with hemophilia.”
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 hemophilia A or B
patients with inhibitors. In addition, frequent infusions are onerous
when used as 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.
About Factor VIIa-CTP
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.
Factor VIIa-CTP has been granted orphan drug designation in the U.S. and
Europe.
About OPKO Health, Inc.
OPKO Health, Inc. is a diversified healthcare company that seeks to
establish industry-leading positions in large, rapidly growing markets.
Our diagnostics business includes Bio-Reference Laboratories, the
nation's third-largest clinical laboratory with a core genetic testing
business and a 420-person sales force to drive growth and leverage new
products, including the 4Kscore® prostate cancer test and the Claros®1
in-office immunoassay platform. Our pharmaceutical business features
Rayaldee™, a treatment for SHPT in stage 3-4 CKD patients with vitamin D
insufficiency (March 29, 2016 PDUFA date) and VARUBI™ for
chemotherapy-induced nausea and vomiting (oral formulation launched by
partner Tesaro, IV formulation in Phase 3). Our biologics business
includes hGH-CTP, a once-weekly human growth hormone injection (in Phase
3 and partnered with Pfizer), and a long-acting Factor VIIa drug for
hemophilia (entering Phase 2a). We also have production and distribution
assets worldwide, multiple strategic investments and an active business
development strategy. More information is available at 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 in a position to be first to market with a longer acting Factor
VIIa product, expectations about market potential for Factor VIIa-CTP
and its ability to provide prophylactic therapy to prevent bleeding
episodes and thereby change the treatment paradigm for patients with
hemophilia, 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.
View source version on businesswire.com: http://www.businesswire.com/news/home/20160223006105/en/
OPKO Health, Inc.
Tara Mackay, 305-575-4100
Investor Relations
or
Media
Rooney
& Associates
Terry Rooney, 212-223-0689
or
Marion
Janic, 212-223-4017
or
Investors
LHA
Anne
Marie Fields, 212-838-3777
or
Bruce
Voss, 310-691-7100
Source: OPKO Health, Inc.
Released February 23, 2016
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