— Results demonstrated potential to reduce current dosing frequency from once-daily to a single weekly injection —
MIAMI and NEW YORK, October 21, 2019 – OPKO Health Inc. (NASDAQ: OPK) and Pfizer Inc.
(NYSE: PFE) announced today that the global Phase 3 trial evaluating somatrogon dosed onceweekly in pre-pubertal children with growth hormone deficiency (GHD) met its primary endpoint of
non-inferiority to daily GENOTROPIN® (somatropin) for injection, as measured by annual height
velocity at 12 months.
Top-line results from the study demonstrated that treatment with somatrogon dosed once-weekly
in pre-pubertal children with GHD was non-inferior to somatropin dosed once-daily with respect to
height velocity at 12 months of treatment (the primary endpoint); the least square mean was higher
in the somatrogon group (10.12 cm/year) than in the somatropin group (9.78 cm/year); the
treatment difference (somatrogon – somatropin) in height velocity (cm/year) was 0.33 with a twosided 95% confidence interval of the difference of (-0.39, 1.05). In addition, change in height
standard deviation scores at six and 12 months, key secondary endpoints, were higher in the
somatrogon dosed once-weekly cohort in comparison to the somatropin dosed once-daily cohort.
Moreover, at six months, change in height velocity, another key secondary endpoint, was higher in
the somatrogon dosed once-weekly cohort in comparison to the somatropin dosed once-daily
cohort. These common measures of growth are employed in the clinical setting to measure the
potential level of catch-up growth that subjects may experience relative to heights of age and
gender matched peers.
Somatrogon was generally well tolerated in the study and comparable to that of somatropin dosed
once-daily with respect to the types, numbers and severity of the adverse events observed between
the treatment arms. Immunogenicity testing and analysis of additional data are ongoing, and full
results of the study will be submitted for presentation at a future scientific meeting.
“We are very pleased to announce these favorable top-line results demonstrating the efficacy of
somatrogon dosed once-weekly in the pediatric patient population with GHD,” said Phillip Frost,
M.D., Chairman and Chief Executive Officer of OPKO. “Somatrogon is a new molecular entity
incorporating OPKO’s proprietary long-acting technology. We believe somatrogon represents a
significant advance in the treatment of children with GHD compared to the current standard of one
injection per day that could enhance a patient’s adherence to treatment and quality of life.”
“We’re encouraged by these data and look forward to the possibility of bringing this longer-acting
therapy to children. If approved, somatrogon could reduce the daily disease burden on children and
their caregivers, potentially increasing treatment adherence,” said Brenda Cooperstone, M.D.,
Chief Development Officer, Rare Disease, Pfizer Global Product Development. “At Pfizer, we strive
each and every day to advance treatment options that better serve the needs of patients with rare
diseases.”
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.
About the Study
The somatrogon Phase 3 trial is a randomized, open-label, active-controlled study conducted in
over 20 countries. This study enrolled and treated 224 pre-pubertal, treatment-naïve children with
growth hormone deficiency who were randomized 1:1 into two arms: somatrogon dosed onceweekly vs daily GENOTROPIN® (somatropin) for injection. The primary endpoint of the trial was
height velocity at 12 months. Secondary endpoints included change in height standard deviation at
six and 12 months, safety and pharmacodynamic measures. Children completing this study had
the opportunity to enroll in a global, open-label, multicenter, long-term extension study, in which
they were able to either continue receiving or switch to somatrogon. Approximately 95% of the
patients switched into the open-label extension study and received somatrogon treatment.
About Somatrogon
Somatrogon is a new molecular entity that contains the natural sequence of growth hormone and
one copy of the C-terminal peptide (CTP) from the beta chain of human chorionic hCG at the Nterminus 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 man-made, prescription 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 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 just like the natural growth hormone that our bodies make and has an established
safety profile.
Important GENOTROPIN® Safety Information
- Growth hormone should not be used to increase height in children after the growth plates
have closed.
- Growth hormone should not be used in patients with diabetes who have certain types of
diabetic retinopathy (eye problems).
- Growth hormone should not be used in patients with cancer or who are being treated for
cancer. Growth hormone deficiency can be caused by brain tumors. So, the presence of
these brain tumors should be ruled out before treatment is started. Growth hormone should
not be used if it is shown that a previous brain tumor has come back or is getting larger.
- Growth hormone should not be used in patients who are critically ill because of surgery,
trauma, or respiratory failure.
- Growth hormone should not be used in children with Prader-Willi syndrome who are very
overweight or have severe breathing problems.
- GENOTROPIN should not be used by patients who have had an allergy or bad reaction to
somatropin or any of the other ingredients in GENOTROPIN.
- Some patients have developed diabetes mellitus while taking GENOTROPIN. Dosage of
diabetes medicines may need to be adjusted during growth hormone treatment. Patients
should be watched carefully if growth hormone is given along with glucocorticoid therapy
and/or other drugs that are processed by the body in the same way.
- In childhood cancer survivors, treatment with growth hormone may increase the risk of a
new tumor, particularly certain benign brain tumors. This risk may be higher in patients who
were treated with cranial radiation. Also, patients and their doctors should check regularly
for any skin changes.
- A small number of patients treated with growth hormone have had increased pressure in
the brain. This can cause headaches and problems with vision. Treatment should be
stopped and reassessed in these patients. Patients with Turner syndrome and Prader-Willi
syndrome may be at higher risk of developing increased pressure in the brain.
- Thyroid function should be checked regularly during growth hormone therapy. Thyroid
hormone replacement therapy should be started or adjusted if needed.
- Patients treated with growth hormone should be checked regularly if they are receiving
standard hormone replacement therapy to treat a lack of more than one hormone.
- In children experiencing rapid growth, curvature of the spine may develop or worsen. This
is also called scoliosis. Patients with scoliosis should be checked regularly to make sure
their scoliosis does not get worse during their growth hormone therapy.
- In children experiencing rapid growth, limping or hip or knee pain may occur. If a child
getting growth hormone therapy starts to limp or gets hip or knee pain, the child’s doctor
should be notified and the child should be examined.
- Growth hormone should only be used during pregnancy if clearly needed. It should be used
with caution in nursing mothers because it is not known whether growth hormone is passed
into human milk.
- Use a different place on the body each day for growth hormone injections. This can help
to prevent skin problems such as lumpiness or soreness.
- Some cases of pancreatitis (inflamed pancreas) have been reported rarely in children and
adults receiving growth hormone. There is some evidence that there is a greater risk of this
in children than in adults. Literature suggests that girls who have Turner syndrome may
have a greater risk of pancreatitis than other children taking growth hormone. In any child
who develops lasting, severe abdominal pain, pancreatitis should be considered.
- In studies of GENOTROPIN in children with GHD, side effects included injection site
reactions, such as pain, redness/swelling, inflammation, bleeding, scarring, lumps, or rash.
Other side effects were fat loss, headache, blood in the urine, low thyroid activity, and
mildly increased blood sugar.
- In studies of GENOTROPIN in children born SGA, side effects included temporarily
elevated blood sugar, increased pressure in the brain, early puberty, abnormal jaw growth,
injection site reactions, growth of moles, and worsening of scoliosis (curvature of the
spine).
- Deaths have been reported with the use of growth hormone in children with Prader-Willi
syndrome. These children were extremely overweight, had breathing problems, and/or
lung infection. All patients with Prader-Willi syndrome should be examined for these
problems. They should also establish healthy weight control.
- In studies of GENOTROPIN in children with PWS, side effects included fluid retention,
aggressiveness, joint and muscle pain, hair loss, headache, and increased pressure in the
brain.
- Turner syndrome patients taking growth hormone therapy may be more likely to get ear
infections. This is also called otitis media.
- In studies of GENOTROPIN in children with Turner syndrome, side effects included flu,
throat, ear, or sinus infection, runny nose, joint pain, and urinary tract infection.
- In studies of GENOTROPIN in children with ISS, side effects included respiratory illnesses,
flu, throat infection, inflammation of the nose and throat, stomach pain, headaches,
increased appetite, fever, fracture, mood changes, and joint pain.
- Women who are taking estrogen by mouth may take GENOTROPIN. They may need a
larger dose of growth hormone.
- GENOTROPIN may be taken by the elderly. Elderly patients may be more likely to have
side effects with growth hormone therapy.
- In studies of GENOTROPIN in adults with GHD, side effects included fluid retention, joint
or muscle pain, stiffness, and changes in sensation. Usually these side effects did not last
long and depended on the dose of GENOTROPIN being taken.
- GENOTROPIN cartridges contain m-Cresol and should not be used by patients allergic to
it.
- A health care provider will help you with the first injection. He or she will also train you on
how to inject GENOTROPIN.
You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088.
For the full Prescribing Information for GENOTROPIN, please visit http://labeling.pfizer.com/ShowLabeling.aspx?id=577.
GENOTROPIN Indications and Usage in the U.S.
GENOTROPIN is a prescription product for the treatment of growth failure in children:
- Who do not make enough growth hormone on their own. This condition is called growth
hormone deficiency (GHD).
- With a genetic condition called Prader-Willi syndrome (PWS). Growth hormone is not right
for all children with PWS. Check with your doctor.
- Who were born smaller than most other babies born after the same number of weeks of
pregnancy. Some of these babies may not show catch-up growth by age 2 years. This
condition is called small for gestational age (SGA).
- With a genetic condition called Turner syndrome (TS).
- With idiopathic short stature (ISS), which means that they are shorter than 98.8% of other
children of the same age and sex; they are growing at a rate that is not likely to allow them
to reach normal adult height, and their growth plates have not closed. Other causes of short
height should be ruled out. ISS has no known cause.
GENOTROPIN is a prescription product for the replacement of growth hormone in adults with
growth hormone deficiency (GHD) that started either in childhood or as an adult. Your doctor should
do tests to be sure you have GHD, as appropriate.
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.
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PFIZER DISCLOSURE NOTICE: The information contained in this release is as of October 21,
2019. Pfizer assumes no obligation to update forward-looking statements contained in this release
as the result of new information or future events or developments.
This release contains forward-looking information about a product candidate, somatrogon dosed
once-weekly in pre-pubertal children with growth hormone deficiency (or GHD) and Pfizer’s rare
disease portfolio, including their 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 Pfizer’s Annual Report on Form 10-
K for the fiscal year ended December 31, 2018 and in its subsequent reports on Form 10-Q,
including in the sections thereof captioned “Risk Factors” and “Forward-Looking Information and
Factors That May Affect Future Results”, 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.pfizer.com.
Media Contact:
Steve Danehy
212-733-1538
Steven.Danehy@pfizer.com
Investor Contact:
Chuck Triano
212-733-3901
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