Pfizer Inc. (NYSE:PFE) announced today enrollment of the first patient
in a multicenter Phase II clinical trial of the investigational compound
PF-06252616 in boys with Duchenne muscular dystrophy (DMD), a genetic
disorder characterized by progressive muscle degeneration and weakness.
PF-06252616 is an experimental, infused, anti-myostatin monoclonal
antibody. Myostatin is a naturally occurring protein in muscles that
helps control muscle growth; it is believed that blocking the activity
of myostatin may have potential therapeutic application in treating
muscle wasting diseases such as DMD.
“DMD is a devastating and debilitating disease impacting approximately 1
in 3,500 male births worldwidei with no current treatment
options,” said Kevin Lee, Ph.D., senior vice president and chief
scientific officer of Pfizer’s Rare Disease Research Unit. “We are
pleased to be taking this important next step in the development of
PF-06252616 as an investigational therapy for DMD in the hopes of
potentially bringing a much-needed therapy to individuals and families
with this devastating disease.”
The phase 2 clinical trial will evaluate the safety, tolerability and
efficacy of PF-06252616 in boys aged 6 to <10 years old diagnosed with
DMD regardless of genotype. Based on the proposed mechanism of action of
PF-06252616, Pfizer is exploring whether there is the potential to
increase muscle mass and function in boys with DMD who are weak and have
lost muscle.
“We are enthusiastic about the potential for myostatin inhibitors to
stimulate increases in muscle mass and strength for people living with
Duchenne muscular dystrophy. This approach could potentially add an
important angle in our fight against this disease and we are pleased to
see the time and great care that Pfizer has expended on its
development,” said Dr. Sharon Hesterlee, vice president of Research for
Parent Project Muscular Dystrophy (PPMD). “Our Duchenne community is
proud to have the extraordinary passion and expertise of the Pfizer team
fighting with us.”
PF-06252616 was granted Orphan Drug designation in July 2012 and Fast
Track Designation in November 2012 by the U.S. Food and Drug
Administration (FDA). The FDA’s Fast Track Designation is a process
designed to facilitate the development and expedite the review of new
drugs and biologics intended to treat or prevent serious conditions and
that address an unmet medical need.ii The European Medicines
Agency (EMA) granted the investigational candidate Orphan Medical
Product designation in February 2013.
About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a genetic disorder characterized by
progressive muscle degeneration and weakness. DMD is caused by an
absence of dystrophin, a protein that helps keep muscle cells intact.
Symptom onset is in early childhood, usually between the ages of 3 and
5. The disease primarily affects boys, but in rare cases it can affect
girls. Muscle weakness can begin as early as age 3, first affecting the
muscles of the hips, pelvic area, thighs and shoulders, and later the
skeletal (voluntary) muscles in the arms, legs and trunk. The calves
often are enlarged. By the early teens, the heart and respiratory
muscles are also affected.iii
Pfizer and Rare Diseases
Rare diseases are among the most serious of all illnesses and impact
millions of patients worldwide, representing an opportunity to apply our
knowledge and expertise to help make a significant impact in addressing
unmet medical needs. The Pfizer focus on rare diseases builds on more
than a decade of experience and a global portfolio of 22 medicines
approved worldwide that treat rare diseases in the areas of hematology,
neuroscience, inherited metabolic disorders, pulmonology, and oncology.
Pfizer Inc.: Working together for a healthier world®
At Pfizer, we apply science and our global resources to bring therapies
to people that extend and significantly improve their lives. We strive
to set the standard for quality, safety and value in the discovery,
development and manufacture of health care products. Our global
portfolio includes medicines and vaccines as well as many of the world's
best-known consumer health care products. Every day, Pfizer colleagues
work across developed and emerging markets to advance wellness,
prevention, treatments and cures that challenge the most feared diseases
of our time. Consistent with our responsibility as one of the world's
premier innovative biopharmaceutical companies, we collaborate with
health care providers, governments and local communities to support and
expand access to reliable, affordable health care around the world. For
more than 150 years, Pfizer has worked to make a difference for all who
rely on us. To learn more, please visit us at www.pfizer.com.
DISCLOSURE NOTICE: The information contained in
this release is as of December 17, 2014. 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, PF-06252616, 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.
Such risks and uncertainties include, among other things, the
uncertainties inherent in research and development, including the
ability to meet anticipated clinical study commencement and completion
dates as well as the possibility of unfavorable study results; whether
and when new drug applications may be filed in any jurisdictions for
PF-06252616; whether and when any such applications may be approved by
regulatory authorities, which will depend on the assessment by such
regulatory authorities of the benefit-risk profile suggested by the
totality of the efficacy and safety information submitted; decisions by
regulatory authorities regarding labeling and other matters that could
affect the availability or commercial potential of PF-06252616; 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, 2013 and in its subsequent reports on Form 10-Q, including in the
sections thereof captioned “Risk Factors” and “Forward-Looking
Information That May Affect Future Results”, as well as in its
subsequent reports on Form 8-K, all of which are filed with the SEC and
available at www.sec.gov
and www.pfizer.com.
Copyright Business Wire 2014