June 23, 2014 2:00pm EDT
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Interim efficacy results show that a single weekly injection of
Lagova (hGH-CTP) can replace seven consecutive daily injections of
currently marketed human growth hormone (hGH)
Conference Call and Slide Presentation Webcast Scheduled Tuesday at
8:30am ET / 7:30am CT
MIAMI--(BUSINESS WIRE)--
OPKO Health, Inc. (NYSE:OPK), a multinational biopharmaceutical and
diagnostics company, today announced 6 month results of a Phase 2
dose-finding study evaluating the safety and efficacy of its novel
long-acting human growth hormone product (Lagova) to treat pediatric
growth hormone deficiency disorder (GHD).
All three Lagova once-weekly doses demonstrate strong catch-up growth
during the six months treatment. The annualized growth rates are above
12 cm in all three doses. The results are supported by excellent dose
dependent pharmacokinetics (PK) and pharmacodynamics (PD) profiles.
Lagova shows a promising safety profile with no serious adverse events
reported. Glucose and lipid metabolism markers are within the normal
ranges. No lipoatrophy was observed in any patients dosed, and no
clinically significant local tolerability issues were identified.
“The interim results further affirm that a once-weekly administration of
Lagova can replace daily injections of marketed hGH in pediatric GHD
patients. The results enable dose selection for the company’s upcoming
Phase 3 pediatric trial,” said Dr. Ron Rosenfeld, clinical advisor on
the study and professor of Pediatrics (emeritus), Stanford University
and professor of Pediatrics at Oregon Health and Science University
(emeritus). “Because Lagova consists of native human growth hormone
attached to a C-Terminal Peptide of endogenous hormone, one would
anticipate low immunogenicity,” Rosenfeld noted.
“Based on these encouraging safety and efficacy results, OPKO plans to
move aggressively into a single confirmatory pivotal Phase 3 study for
pediatric GHD patients. We hope to make Lagova available to pediatric
GHD patients as soon as possible,” said CEO, Phillip Frost, M.D. “Lagova
is one of a family of important products being developed at OPKO
Biologics designed to improve compliance and offer ease of
administration to patients.”
Study Design
The randomized, comparator-controlled Phase 2 study was conducted in up
to 56 pre-pubertal, naïve GHD children receiving one of three Lagova
doses as once-weekly regimen (0.25, 0.48, 0.66mg/Kg/week; equivalent of
0.18, 0.35, 0.48 mg/Kg/week of hGH) or daily hGH (34µg/Kg/day)
subcutaneously. In order to introduce naïve patients to the allocated
Lagova dose in a gradual manner, a stepwise dose increase approach was
implemented. Once patients reached the targeted doses, Lagova, GH, IGF-1
and IGF-BP3 concentrations were measured and PK-PD analysis was
conducted utilizing a population based approach.
Study Results
An interim analysis of 6 months data demonstrated that all doses of
Lagova used in the study provided strong catch-up growth response better
than historical controls of daily growth hormone therapy.
The baseline characteristics of all patients were comparable among all
groups. Interim analysis of the PK profile following administration of
Lagova demonstrates a significantly extended half-life as reflected by
the T1/2 and AUC respectively. A dose dependent PD (IGF-1) response was
observed between Lagova cohorts, reaching steady state with no
accumulation or excessive levels. All cohorts demonstrated promising
“catch-up” growth, in line with reported age and GHD severity-matched
data. The annualized height velocities are more than 12 cm, which
correlates with the PK/PD profile in those patients.
Conference Call and Webcast
OPKO will hold a conference call and live webcast on Tuesday, June 24,
2014 at 8:30 a.m. EDT (7:30 a.m. CDT). The dial-in numbers are
1-877-407-0789 for domestic callers and 1-201-689-8562 for international
callers. To join the live webcast of the presentation, please register
for ‘OPKO Health: ENDO Conference’ on June 24, 2014, at 8:30 a.m. EDT
After the webcast, the call will remain available on the OPKO website, www.opko.com ,
for 30 days.
About Lagova (hGH-CTP)
In June 2013, OPKO initiated a pivotal Phase 3 clinical trial in adults
for its proprietary long-acting version of hGH-CTP (Lagova). Lagova has
been awarded orphan drug designation in the U.S. and Europe for both
adults and children with growth hormone deficiency.
ABOUT OPKO HEALTH
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 .
SAFE HARBOR STATEMENT
This press release contains "forward-looking statements," as that
term is defined under the Private Securities Litigation Reform Act of
1995 (PSLRA), which statements may be identified by words such as
"expects," "plans," "projects," "will," "may," "anticipates,"
"believes," "should," "intends," "estimates," and other words of similar
meaning, including statements regarding expected results and benefits of
Lagova, including its safety and efficacy, whether OPKO's clinical
trials for adult and pediatric growth hormone deficiency will generate
data to support marketing approval, whether a single injection of Lagova
can replace seven consecutive daily injections of currently marketed hGH,
whether Lagova will have low immunogenecity, the expected
commencement date for the Phase 3 clinical trial for Lagova in pediatric
patients, whether Lagova will be successfully developed or
commercialized, expectations regarding the product and its market
potential, as well as other non-historical statements about our
expectations, beliefs or intentions regarding our business, technologies
and products, financial condition, strategies or prospects. 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 the risks inherent in funding,
developing and obtaining regulatory approvals of new,
commercially-viable and competitive products and treatments, including
the risks that the Phase 3 clinical trials for the Lagova product 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 Lagova, and that Lagova may not have
advantages or prove to be superior over presently marketed products or
products introduced in the future. 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.
FTI Consulting
Investor Relations
Barbara Ryan,
Managing Director
or
Media
Relations
Kimberly Ha, 212-850-5612
Senior Director
Source: OPKO Health, Inc.
Released June 23, 2014
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