Following last October’s announcement of successful results of the
global Phase 3 study of once-weekly somatrogon, an investigational
agent for the treatment of pediatric growth hormone deficiency
(pGHD), OPKO Health Inc. (NASDAQ: OPK) announced today that the
Japan Phase 3 clinical trial met its primary and secondary
objectives, and demonstrated that the efficacy and safety of
somatrogon administered weekly was comparable to GENOTROPIN®
(somatropin) for injection administered once-daily as measured by
annual height velocity after 12 months of treatment in
treatment-naïve Japanese pre-pubertal children with GHD. The
findings were consistent with the results previously reported in
the Phase 3 global study.
Using the Full Analysis Set, the least squared
means for the annual height velocity was higher in the somatrogon
group (9.65 cm/year) than in the GENOTROPIN group (7.87 cm/year).
The point estimate of the treatment difference was 1.79 cm/year
with the two-sided 95% confidence interval of (0.97, 2.60).
Similarly, greater change in height standard deviation score from
baseline at 12 months was observed for the somatrogon group (0.94)
compared to the GENOTROPIN group (0.52), indicating that both
somatrogon and GENOTROPIN were associated with catch-up growth in
treated patients.
Most adverse events were mild to moderate in
severity with no notable differences between the treatment groups,
and weekly somatrogon administration was generally well-tolerated.
A total of six serious adverse events were reported in four
patients (two in the somatrogon arm and two in the GENOTROPIN arm).
One patient in the GENOTROPIN group discontinued treatment in the
study due to adverse events.
“We are pleased to announce positive top-line
results from the Japanese Phase 3 clinical study of somatrogon. We
have reached an additional clinical development milestone of
somatrogon with the demonstration of comparable efficacy and safety
of somatrogon administered once-weekly to daily administration of
GENOTROPIN in pediatric patients with growth hormone deficiency,”
said Phillip Frost, M.D., Chairman and Chief Executive Officer of
OPKO. “Positive results in both the Japanese and pivotal global
Phase 3 pediatric trials are promising for children with pGHD.
Treatment with once-weekly somatrogon represents a significant
advance, which has the potential to enhance patient adherence and
quality of life.”
“The data from the Japan Phase 3 trial are very
encouraging and point to the potential of bringing a once-weekly
treatment option to children with pGHD. We are committed to
furthering treatment advances for people living with rare growth
hormone disorders and this is an exciting advance that may help
ease the daily disease burden for children and their caregivers,”
said Brenda Cooperstone, M.D., Chief Development Officer, Rare
Disease, Pfizer Global Product Development.
In 2014, Pfizer and OPKO entered into a
worldwide agreement for the development and commercialization of
somatrogon for the treatment of GHD. Under the agreement, OPKO is
responsible for conducting the clinical program and Pfizer is
responsible for registering and commercializing the product. The
companies will evaluate the potential for additional pediatric and
adult indications, as appropriate.
About the Study
The Phase 3 study of somatrogon in 44
treatment-naïve Japanese pre-pubertal children with pGHD was a
12-month, open-label, randomized, active-controlled, parallel-group
study of the efficacy and safety of weekly somatrogon compared to
recombinant human growth hormone (r-hGH), GENOTROPIN® (somatropin)
for injection treatment administered once-daily. Eligible patients
were randomized in a 1:1 ratio to receive either once-weekly
somatrogon or GENOTROPIN administered once-daily (reference
therapy, 0.025 mg/kg/day which is equivalent to 0.175 mg/kg/week).
To obtain pharmacokinetic information of three different weekly
doses in Japanese pGHD patients, somatrogon treated patients
received 0.25 mg/kg/week for 2 weeks, followed by 0.48 mg/kg/week
for 2 weeks followed by 0.66 mg/kg/week for the remaining 46
weeks. Somatrogon was administered subcutaneously using a
single patient use, multi-dose, disposable, pre-filled pen, the
same pen used in the global study, while GENOTROPIN was
administered using approved commercial products in Japan.
Patients who completed the 12-month Main Study
Period and met the inclusion criteria were given the opportunity to
continue in a long-term open label extension period with
once-weekly somatrogon until marketing approval. All patients in
the GENOTROPIN group have been switched to weekly somatrogon
treatment.
About Somatrogon
Somatrogon is a new molecular entity that
contains the natural sequence of human growth hormone and one copy
of the C-terminal peptide (CTP) from the beta chain of human
chorionic hCG at the N-terminus and two copies at the C-terminus.
The CTPs extend the half-life of the molecule. Somatrogon received
Orphan Drug designation in the U.S. and the EU for the treatment of
children and adults with growth hormone deficiency.
About Growth Hormone
Deficiency
Growth hormone deficiency is a rare disease
characterized by the inadequate secretion of growth hormone from
the pituitary gland and affects one in approximately 4,000 to
10,000 people. In children, this disease can be caused by genetic
mutations or acquired after birth. Because the patient's pituitary
gland secretes inadequate levels of somatropin, the hormone that
causes growth, his or her height may be affected and puberty may be
delayed. Without treatment, he or she will have persistent growth
attenuation, a very short height in adulthood and may experience
other health problems.
About GENOTROPIN®
GENOTROPIN is a recombinant growth hormone as a
replacement treatment option, approved in the United States for
children who do not make enough growth hormone on their own, have
the genetic condition called Prader-Willi syndrome (PWS), were born
smaller than most other babies, have the genetic condition called
Turner syndrome (TS) or have idiopathic short stature (ISS).
GENOTROPIN is also approved in the United States to treat adults
with growth hormone deficiency. GENOTROPIN is taken by injection
just below the skin and is available in a wide range of devices to
fit a range of individual dosing needs. GENOTROPIN is
distributed by Pharmacia and Upjohn Co., a division of Pfizer,
Inc.
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
www.opko.com.
Cautionary Statement Regarding
Forward-Looking Statements
This release contains forward-looking
information about a product candidate, somatrogon dosed once-weekly
in pre-pubertal children with growth hormone deficiency (or GHD),
including its potential benefits, that involves substantial risks
and uncertainties that could cause actual results to differ
materially from those expressed or implied by such statements.
Risks and uncertainties include, among other things, the
uncertainties inherent in research and development, including the
ability to meet anticipated clinical endpoints, commencement and/or
completion dates for our clinical trials, regulatory submission
dates, regulatory approval dates and/or launch dates, as well as
the possibility of unfavorable new clinical data and further
analyses of existing clinical data; the risk that clinical trial
data are subject to differing interpretations and assessments by
regulatory authorities; whether regulatory authorities will be
satisfied with the design of and results from our clinical studies;
whether and when drug applications may be filed in any
jurisdictions for somatrogon; whether and when any such
applications may be approved by regulatory authorities, which will
depend on myriad factors, including making a determination as to
whether the product's benefits outweigh its known risks and
determination of the product's efficacy and, if approved, whether
somatrogon will be commercially successful; decisions by regulatory
authorities impacting labeling, manufacturing processes, safety
and/or other matters that could affect the availability or
commercial potential of somatrogon; and competitive
developments.
A further description of risks and uncertainties
can be found in OPKO’s Annual Reports on Form 10-K for the fiscal
year ended December 31, 2019 and in its subsequent reports on Form
10-Q, including in the sections thereof captioned “Risk Factors”,
“Forward-Looking Information and Factors That May Affect Future
Results”, “Cautionary Statement Regarding Forward-Looking
Statements”, as well as in its subsequent reports on Form 8-K, all
of which are filed with the U.S. Securities and Exchange Commission
and available at www.sec.gov and www.OPKO.com.
Contacts:
LHA Investor RelationsYvonne
Briggs, 310-691-7100ybriggs@lhai.com orBruce Voss,
310-691-7100bvoss@lhai.com
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