June 25, 2014 3:32pm EDT
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MIAMI--(BUSINESS WIRE)--
OPKO Health, Inc. (NYSE:OPK), a multinational biopharmaceutical and
diagnostics company, yesterday hosted a webcast presenting outstanding
interim six-month results from a Phase 2 study of its long-acting human
growth hormone product Lagova to treat pediatric growth hormone
deficiency disorder (GHD).
During the webcast, Dr. Ron Rosenfeld, Chairman of OPKO Biologic’s
Scientific Advisory Board and professor emeritus of Pediatrics at both
Stanford University and Oregon Health and Science University,
highlighted the superior interim results from the study. In particular,
Dr. Rosenfeld, a pioneer in the recombinant human growth hormone field,
noted that the mean height velocity achieved with each of the three
different doses of Lagova administered in the study was “actually quite
dramatic, ranging from 12.25 centimeters to almost 15.5 centimeters.
Every child enrolled in the study experienced a significant improvement
in height velocity from weekly administration of Lagova,” he noted.
“OPKO believes there is a significant market opportunity with Lagova, as
compliance with daily injections is problematic. Non-compliance leads to
a significant decrease in growth response in both adults and children,
which is well evidenced by current clinical literature,” said OPKO’s
CEO, Phillip Frost, M.D. “The outstanding interim Phase II results allow
for dose selection for a Phase III study which is anticipated to start
in 2015.”
Unlike other pediatric long-acting growth hormone deficiency clinical
trials, the Lagova Phase II trials also included a direct comparator arm
at the same population with similar baseline values. Children were given
daily administration of growth hormone at a dosage of 0.24 mg/kg per
week, but administered daily in a traditional manner. There was no
statistically significant difference among any of the three dosage
strengths of Lagova used in the study versus the comparator arm.
As any Phase III trial for a long-acting recombinant human growth
hormone will likely require proof of non-inferiority against a
comparator arm of daily administered growth hormone, this is an
important distinction that gives OPKO great confidence in designing its
non-inferiority pivotal Phase III study for Lagova. Had OPKO utilized
historical database comparisons as utilized in other pediatric GHD
clinical trials versus an actual comparator arm, annualized height
velocity for Lagova at all three dosage strengths would have been
notably superior, exceeding comparable age-matched historical controls,
as published by Bakker (2008) and Ranke (2010) for the same GHD patient
population by approximately 30%.
Lagova uses OPKO Biologics’ novel CTP technology which attaches a short
naturally occurring peptide to the natural human growth hormone.
Approximately 75% of the injected protein in Lagova is the actual native
growth hormone, altogether resulting in a highly water soluble compound
that can be delivered in high concentrations using a very thin 31 gauge
needle to patients of a wide range of body weights and ages by a single,
weekly injection. As expected, the safety profile from the six-month
data was excellent. No serious adverse events were reported. There were
no episodes of either lipoatrophy or lipodystrophy and there were no
injection site tolerability issues. More importantly, long term safety
is supported by the pharmacokinetic and pharmacodynamics profiles; there
was no accumulation and no over-exposure of IGF-1.
A replay of the webcast is available on OPKO’s website, www.opko.com ,
for the next 30 days.
About Lagova (hGH-CTP)
In June 2013, OPKO initiated a multi-center worldwide 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.
For OPKO Health, Inc.
FTI Consulting
Barbara
Ryan, 212-850-5679
Managing Director
or
Media
FTI Consulting
Kimberly Ha, 212-850-5612
Senior
Director
Source: OPKO Health, Inc.
Released June 25, 2014
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