Pfizer Inc. (NYSE:PFE) announced today two strategic decisions to expand
the company’s rare disease research and development activities through
the establishment of a gene therapy platform to investigate potential
treatments for patients. First is an agreement with Spark Therapeutics
to develop SPK-FIX, a program incorporating a bio-engineered AAV vector
for the potential treatment of Hemophilia B expected to enter Phase 1/2
clinical trials in the first half of 2015. Additionally, Pfizer has
appointed Michael Linden, Ph.D., Professor at King’s College London and
Director of the University College London Gene Therapy Consortium, who
will be with the company for a two-year secondment to lead gene therapy
research in the rare disease area.
“The fundamental understanding of the biology of hereditary rare
diseases, coupled with advances in the technology to harness disarmed
viruses as gene delivery vehicles, provide a ripe opportunity to
investigate the next wave of potential life-changing therapies for
patients,” said Mikael Dolsten, M.D., Ph.D., president of Worldwide
Research and Development at Pfizer. “By establishing our gene therapy
capabilities, we hope to gain a deeper understanding of the mechanisms
that could potentially bring true disease modification for those
suffering from devastating hematologic and neuromuscular diseases.”
Agreement with Spark Therapeutics for
Hemophilia Research
Philadelphia-based Spark Therapeutics and Pfizer will collaborate to
progress the clinical program for SPK-FIX, a program incorporating a
bio-engineered AAV vector for the potential treatment of hemophilia B.
Pfizer has a long-standing commitment to the hemophilia community and
has been providing hemophilia products to patients for more than 17
years.
“Pfizer strives to provide meaningful enhancements to the lives of
patients with hemophilia, and the agreement with Spark Therapeutics
offers an important expansion of Pfizer’s commitment to the bleeding
disorder community and builds on our leading hemophilia portfolio,” said
Geno Germano, group president, Global Innovative Pharma Business at
Pfizer. “We believe the SPK-FIX program could add to our existing
portfolio of hemophilia products and could pioneer a potential new
treatment technology for patients with bleeding disorders.”
Under the terms of the agreement, Spark will maintain responsibility for
clinical development through Phase 1/2 studies. Pfizer will assume
responsibility for pivotal studies, any regulatory approvals and
potential global commercialization of the product.
Establishment of Gene Therapy Research in
Pfizer Rare Disease
Effective December 1, 2014, Professor Michael Linden has joined Pfizer
from his current position at King’s College London, for a two-year
secondment to lead gene therapy research within the company’s rare
disease research area.
“The establishment of a gene therapy group under the leadership of
Professor Linden will help Pfizer explore the potential of this
important technology that could possibly benefit patients living with
serious diseases,” said Kevin Lee, Ph.D., senior vice president and
chief scientific officer of Pfizer’s Rare Disease Research Unit.
“Professor Linden brings to Pfizer his extensive expertise in AAV
technology obtained from over 20 years working in the field.”
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 3, 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 an agreement
between Pfizer and Spark Therapeutics to jointly develop SPK-FIX 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 drug applications may be filed in any jurisdictions for any
potential product candidates or combination therapies; 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 any of such product candidates or combination
therapies; 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