Phase 3 lead-in study initiated following completion of the transfer
of Spark Therapeutics’ hemophilia B gene therapy program to Pfizer
NEW YORK & PHILADELPHIA--(BUSINESS WIRE)--
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,
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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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Pfizer Inc.
Media Relations:
Neha Wadhwa, 212-733-2835
Neha.Wadhwa@pfizer.com
or
Investors:
Chuck
Triano, 212-733-3901
Charles.E.Triano@pfizer.com
Source: Pfizer Inc.