Spark Therapeutics (NASDAQ:ONCE) and Pfizer Inc. (NYSE:PFE)
announced today that the U.S. Food and Drug Administration (FDA)
has granted breakthrough therapy designation to SPK-9001, the lead
investigational candidate in the companies’ SPK-FIX program, in
development for the treatment of hemophilia B. SPK-9001, a novel
bio-engineered adeno-associated virus (AAV) capsid expressing a
codon-optimized, high-activity human factor IX variant, is being
investigated in an ongoing Phase 1/2 trial as a potential one-time
therapy.
Breakthrough therapy designation is intended to expedite the
development and FDA review of drugs to treat a serious or
life-threatening disease or condition. The designation requires
preliminary clinical evidence that the investigational therapy may
offer substantial improvement over existing therapies on at least
one clinically significant endpoint.
In addition to SPK-9001, Spark Therapeutics previously received
breakthrough therapy designation for voretigene neparvovec, which
is being developed for the potential treatment of inherited
retinal disease (IRD) caused by mutations in the RPE65 gene.
“We are extremely pleased to have been granted breakthrough
therapy designation for SPK-9001, which has shown early promise in
achieving our goal of eliminating the need for regular infusions to
control and prevent bleeding episodes in patients with hemophilia B
through a potentially one-time, intravenous administration of a
highly optimized gene therapy,” said Jeffrey D. Marrazzo,
chief executive officer of Spark Therapeutics. “Together with
Pfizer, we look forward to working closely with the FDA to bring
SPK-9001 to patients as quickly and responsibly as possible.”
About Hemophilia B
Hemophilia is a rare genetic bleeding disorder that causes the
blood to take a long time to clot as a result of a deficiency in
one of several blood clotting factors, and occurs almost
exclusively in males. People with hemophilia face specific risks as
they are not able to form blood clots efficiently and 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.
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 is also
called congenital factor IX deficiency or Christmas disease.
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
Spark Therapeutics' proprietary technology platform for
selecting, designing, manufacturing and formulating highly
optimized gene therapies was applied to developing compounds in
the SPK-FIX program. The SPK-FIX program leverages a
long history of hemophilia gene therapy research and clinical
development conducted by Spark Therapeutics and its founding
scientific team over nearly three decades. SPK-9001 is a novel
bio-engineered adeno-associated virus (AAV) capsid expressing a
codon-optimized, high-activity human factor IX variant enabling
endogenous production of factor IX. SPK-9001 is being
developed under a collaboration with Pfizer. Spark Therapeutics and
Pfizer entered into a collaboration in 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
Spark Therapeutics, a fully integrated gene therapy company, is
seeking to transform the lives of patients with debilitating
genetic diseases by developing one-time, life-altering treatments.
Spark Therapeutics’ validated gene therapy platform is being
applied to a range of clinical and preclinical programs addressing
serious genetic diseases, including inherited retinal diseases,
liver-associated diseases, such as hemophilia, and
neurodegenerative diseases. Spark Therapeutics’ validated and
proprietary technology platform for selecting, designing,
manufacturing and formulating highly optimized gene therapies has
successfully delivered gene therapies with proof-of-concept data in
the eye and liver. Spark Therapeutics’ most advanced product
candidate, voretigene neparvovec (formerly referred to as
SPK-RPE65), which has received both breakthrough therapy and orphan
product designation, reported positive top-line results from a
pivotal Phase 3 clinical trial for the treatment of rare blinding
conditions. Spark Therapeutics’ hemophilia franchise has two lead
assets: SPK-9001, in a Phase 1/2 trial for hemophilia B
and SPK-8011, a preclinical candidate for hemophilia A. To
learn more, please visit www.sparktx.com.
Spark 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. 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; 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 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.
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 two decades of experience, a dedicated
research unit focusing on rare diseases, and a global portfolio of
more than 20 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. For more information, please visit us at
www.pfizer.com. In addition, to learn more, 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 July 21,
2016. 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 I/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;
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; 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, 2015 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.
Contacts
Corporate Contacts:
Stephen W. Webster
Chief Financial Officer
Spark Therapeutics, Inc.
Daniel Faga
Chief Business Officer
Spark Therapeutics, Inc.
(855) SPARKTX (1-855-772-7589)
Media Contact:
Ten Bridge Communications
Dan Quinn
(781) 475-7974
dan@tenbridgecommunications.com
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