Spark Therapeutics (NASDAQ:ONCE), a fully integrated gene therapy
company dedicated to challenging the inevitability of genetic
disease, and Pfizer Inc. (NYSE:PFE), today announced that The New
England Journal of Medicine has published interim data as of July
25, 2017, from the Phase 1/2 clinical trial of SPK-9001, an
investigational gene therapy for hemophilia B. With a cumulative
follow-up of 492 weeks’ observation of the first 10 adult male
participants, the mean steady-state factor IX activity was 34
percent of normal (range of 14-81 percent) following a single
administration of investigational SPK-9001. The annualized bleeding
rate (ABR) was reduced 97 percent, from a mean rate of 11.1 events
per year before vector administration to 0.4 events per year after
vector administration (p=0.02), while factor IX concentrate use was
reduced 99 percent (p=0.004).
“People who live with hemophilia today face a lifelong need for
vigilant monitoring and recurrent factor concentrate infusions to
prevent spontaneous, potentially life-threatening bleeds and to
protect their joints. The discipline required to execute the usual
prophylactic regimen can exact a heavy toll on quality of life, and
these regimens result in significant costs to patients, families
and the health care system,” said Katherine A. High, M.D.,
president and head of Research and Development at Spark
Therapeutics and co-author of the paper. “The data suggest a
one-time infusion of SPK-9001 has the potential to safely sustain
factor IX coagulant activity level that may result in the
termination of baseline prophylaxis factor infusions, significantly
reduce bleeding, and nearly eliminate the need for exogenous factor
IX concentrate infusions.”
In this open-label, non-randomized, multicenter Phase 1/2
clinical trial, there were no serious adverse events during or
following infusion of SPK-9001, and no participants experienced
thrombotic events or developed factor IX inhibitors. Two
participants developed an asymptomatic and transient increase in
liver enzymes that resolved with a tapering dose of oral
corticosteroids. One participant with severe joint disease has
administered factor for suspected bleeding, but overall factor use
for this participant was 91 percent lower than before SPK-9001
infusion.
Please refer to the paper, “Hemophilia B Gene Therapy with a
High Specific Activity Factor IX Variant,” for the full description
of the design and interim results of this study.
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.
About the SPK-FIX Program
and SPK-9001 SPK-9001 is a novel investigational
vector that contains a bio-engineered adeno-associated virus (AAV)
capsid and a codon-optimized, high-activity human factor IX gene
enabling endogenous production of factor IX.
Spark Therapeutics and Pfizer entered into a collaboration
in December 2014 for the SPK-FIX program,
including SPK-9001, under which Spark
Therapeutics is responsible for conducting all Phase 1/2
studies for any product candidates, while Pfizer will assume
responsibility for pivotal studies, any regulatory activities and
potential global commercialization of any products that may result
from the collaboration.
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 directed to the retina and liver, and currently have
four programs in clinical trials or under regulatory review,
including the first potential gene therapy for a genetic disease in
the United States and 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.
Cautionary note on forward-looking statements
This 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) our lead SPK-FIX product
candidate, SPK-9001, may not produce sufficient data in our
Phase 1/2 clinical trial to warrant further development; (ii) our
overall collaboration with Pfizer may not be successful; (iii) we
may not receive any additional milestone payments from Pfizer; and
(iv) any one or more of our product candidates in preclinical or
clinical development will not successfully be developed and
commercialized. 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 Securities and Exchange
Commission. All information in this press release is as of the date
of the release, and Spark undertakes no duty to update this
information unless required by law.
About Pfizer: 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.
Pfizer Disclosure
Notice: The information contained in
this release is as of Dec. 6, 2017. 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 SPK-9001 and the SPK-FIX program,
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 Phase 1/2
study for SPK-9001 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 SPK-9001; 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 SPK-9001 will be
commercially successful; decisions by regulatory authorities
regarding labeling and other matters that could affect the
availability or commercial potential of SPK-9001; 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, 2016 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.
Investor Relations Contact:Ryan Asay
ryan.asay@sparktx.com (215) 239-6424
Media Contact:Monique da
Silvacommunications@sparktx.com (215) 282-7470
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