Phase 3 lead-in study initiated following
completion of the transfer of Spark Therapeutics’ hemophilia B gene
therapy program to Pfizer
Pfizer Inc. (NYSE:PFE) and Spark Therapeutics (NASDAQ:ONCE)
announced today that Pfizer initiated a Phase 3 open-label,
multi-center, lead-in study (NCT03587116) to evaluate the efficacy
and safety of current factor IX prophylaxis replacement therapy in
the usual care setting. The factor IX prophylaxis efficacy data
obtained in the lead-in study will serve as the within-subject
control group for those patients that enroll into the next part of
the Phase 3 study, which will evaluate the investigational gene
therapy fidanacogene elaparvovec for the treatment of hemophilia B.
The interventional portion of this pivotal Phase 3 study will
enroll patients who have completed at least six months in the
lead-in study. Fidanacogene elaparvovec is the official United
States Adopted Name (USAN) and will become the Recommended
International Nonproprietary Name (INN) for the therapy formerly
known as SPK-9001 and PF-06838435.
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The Phase 3 program was initiated following the transfer of the
responsibility for Spark Therapeutics’ hemophilia B gene therapy
program to Pfizer. Fidanacogene elaparvovec is a novel,
investigational vector that contains a bio-engineered
adeno-associated virus (AAV) capsid (protein shell) and a
high-activity human coagulation factor IX gene. It is hoped that,
once treated, patients will be able to produce factor IX
themselves, rather than having to regularly inject factor IX.
"With the lead-in study now open and actively recruiting
patients, we are excited to begin our Phase 3 program evaluating
fidanacogene elaparvovec for the treatment of hemophilia B,” said
Brenda Cooperstone, MD, Senior Vice President and Chief Development
Officer, Rare Disease, Pfizer Global Product Development. “The
current data suggest immense promise for the use of this potential
one-time treatment option. We look forward to the opportunity to
continue the progress achieved by Spark Therapeutics for patients
living with hemophilia B.”
“We are pleased to have transitioned fidanacogene elaparvovec to
Pfizer following the positive results of the ongoing Phase 1/2
clinical trial,” said Katherine A. High, MD, President and Head of
Research & Development, Spark Therapeutics. “The initiation of
the Phase 3 program marks an important milestone toward our goal of
one day potentially freeing patients with hemophilia B of the need
for regular infusions, while potentially eliminating spontaneous
bleeding.”
In May 2018, Pfizer and Spark Therapeutics announced data for 15
participants in the ongoing Phase 1/2 clinical trial of
fidanacogene elaparvovec for the treatment of severe or moderately
severe (FIX:C < 2 percent) hemophilia B. The findings showed all
15 patients had discontinued routine infusions of factor IX
concentrates with no reported serious adverse events or thrombotic
events as of the May 7, 2018 data cutoff.
About the Pfizer and Spark Therapeutics Agreement
Pfizer and Spark Therapeutics entered into a License Agreement
in December 2014 for the hemophilia B gene therapy program. Under
the terms of the agreement, Pfizer will now assume sole
responsibility for all subsequent pivotal studies, all regulatory
activities, manufacturing and global commercialization of any
products resulting from the hemophilia B gene therapy program.
About Hemophilia B
Hemophilia, a rare genetic bleeding disorder that causes the
blood to take a long time to clot because of a deficiency in one of
several blood clotting factors, is almost exclusively found in
males. People with hemophilia are at risk for excessive and
recurrent bleeding from modest injuries, which have the potential
to be life threatening. People with severe hemophilia often bleed
spontaneously into their muscles or joints, or rarely into other
critical closed spaces such as the intracranial space, where
bleeding can be fatal. The incidence of hemophilia B is one in
25,000 male births. People with hemophilia B have a deficiency in
clotting factor IX, a specific protein in the blood. Hemophilia B
also is called congenital factor IX deficiency or Christmas
disease. The current standard of care requires recurrent
intravenous infusions of either plasma-derived or recombinant
factor IX to control and prevent bleeding episodes. There exists a
significant need for novel therapeutics to treat people living with
hemophilia.
Pfizer Rare Disease
Rare disease includes some of the most serious of all illnesses
and impacts millions of patients worldwide, representing an
opportunity to apply our knowledge and expertise to help make a
significant impact on addressing unmet medical needs.1 The Pfizer
focus on rare disease builds on more than two decades of
experience, a dedicated research unit focusing on rare disease, and
a global portfolio of multiple medicines within a number of disease
areas of focus, including hematology, neuromuscular, and inherited
metabolic disorders.1
Pfizer Rare Disease combines pioneering science and deep
understanding of how diseases work with insights from innovative
strategic collaborations with academic researchers, patients, and
other companies to deliver transformative treatments and solutions.
We innovate every day leveraging our global footprint to accelerate
the development and delivery of groundbreaking medicines and the
hope of cures.
Click here to learn more about our Rare Disease portfolio and
how we empower patients, engage communities in our clinical
development programs, and support programs that heighten disease
awareness.
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, we have worked to make a difference for
all who rely on us. We routinely post information that may be
important to investors on our website at www.pfizer.com. In
addition, to learn more, please visit us on www.pfizer.com and
follow us on Twitter at @Pfizer and @Pfizer_News, LinkedIn, YouTube
and like us on Facebook at Facebook.com/Pfizer.
About Spark Therapeutics
At Spark Therapeutics, a fully integrated company committed to
discovering, developing and delivering gene therapies, we challenge
the inevitability of genetic diseases, including blindness,
hemophilia and neurodegenerative diseases. We have successfully
applied our technology in the first FDA-approved gene therapy in
the U.S. for a genetic disease, and currently have three programs
in clinical trials, including product candidates that have shown
promising early results in patients with hemophilia. At Spark, we
see the path to a world where no life is limited by genetic
disease. For more information, visit www.sparktx.com, and follow us
on Twitter and LinkedIn.
Spark Therapeutics Cautionary note on forward-looking
statements
This press release contains "forward-looking statements" within
the meaning of the Private Securities Litigation Reform Act of
1995, including statements regarding the company's SPK-FIX program.
The words "anticipate," "believe," "expect," "intend," "may,"
"plan," "predict," "will," "would," "could," "should," "continue"
and similar expressions are intended to identify forward-looking
statements, although not all forward-looking statements contain
these identifying words. We may not actually achieve the plans,
intentions or expectations disclosed in our forward-looking
statements, and you should not place undue reliance on our
forward-looking statements. Any forward-looking statements are
based on management's current expectations of future events and are
subject to a number of risks and uncertainties that could cause
actual results to differ materially and adversely from those set
forth in, or implied by, such forward-looking statements. These
risks and uncertainties include, but are not limited to, the risk
that: (i) SPK-9001 may not free patients with hemophilia B of the
need for regular infusions, while eliminating spontaneous bleeding
and (ii) our overall collaboration with Pfizer may not be
successful. For a discussion of other risks and uncertainties, and
other important factors, any of which could cause our actual
results to differ from those contained in the forward-looking
statements, see the "Risk Factors" section, as well as discussions
of potential risks, uncertainties and other important factors, in
our Annual Report on Form 10-K, our Quarterly Reports on Form 10-Q
and other filings we make with the U.S. Securities and Exchange
Commission. All information in this press release is as of the date
of the press release, and Spark undertakes no duty to update this
information unless required by law.
DISCLOSURE NOTICE: The information contained in this release is
as of July 16, 2018. 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
fidnacogene elparvovec, the fidnacogene elparvovec program and the
License Agreement between Pfizer and Spark, 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 study commencement and completion dates as
well as the possibility of unfavorable study results, including
unfavorable new clinical data and additional analyses of existing
clinical data; risks associated with initial data, including the
risk that the final results of the fidnacogene elparvovec lead-in
study and the subsequent Phase 3 study and/or additional clinical
trials may be different from (including less favorable than) the
initial data results and may not support further clinical
development; the risk that clinical trial data are subject to
differing interpretations, and, even when we view data as
sufficient to support the safety and/or effectiveness of a product
candidate, regulatory authorities may not share our views and may
require additional data or may deny approval altogether; whether
regulatory authorities will be satisfied with the design of and
results from our clinical studies; whether and when any
applications may be filed with regulatory authorities for
fidnacogene elparvovec; whether and when regulatory authorities may
approve any such applications, 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 and, if approved, whether fidnacogene elparvovec will be
commercially successful; decisions by regulatory authorities
regarding labeling and other matters that could affect the
availability or commercial potential of fidnacogene elparvovec; 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, 2017 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.
________________________________1 Pfizer Inc. Rare disease.
http://www.pfizer.com/health-and-wellness/health-topics/rare-diseases/areas-of-focus.
Accessed July 2018.
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version on businesswire.com: https://www.businesswire.com/news/home/20180716005453/en/
Pfizer Inc.Media Relations:Neha Wadhwa,
212-733-2835Neha.Wadhwa@pfizer.comorInvestors:Chuck Triano,
212-733-3901Charles.E.Triano@pfizer.com
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