Subjects received one-time administration of a
highly optimized gene therapy at initial low dose without the need
for immunosuppression
Spark Therapeutics (NASDAQ:ONCE) and Pfizer Inc. (NYSE:PFE)
announced today that new data will be presented on June 11 at the
European Hematology Association’s (EHA) 21st Congress. These data
will show encouraging initial observations for the first subjects
dosed in the Phase 1/2 clinical trial of SPK-9001, the lead
investigational compound in the SPK-FIX program, which is being
studied 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, was
developed using Spark’s proprietary technology platform for
selecting, designing, manufacturing and formulating highly
optimized gene therapies. The conference abstract, including a
figure demonstrating Factor IX activity levels (as % of normal)
expressed by subject over time, was made available today:
http://www.ehaweb.org/congress-and-events/21st-congress/key-information-3/
Data available today demonstrate that the first three subjects
enrolled in the study experienced AAV-mediated Factor IX activity
levels following one administration of SPK-9001 at the initial dose
level (5 x 1011 vg/kg) studied in the trial. Factor IX activity
levels in the first two subjects, without prior history of liver
disease, rose consistently through the first four weeks
post-administration. At the time of abstract submission, the
first subject stabilized at 28% of normal at eighteen weeks, and
the second subject at 30% of normal at seven weeks
post-administration. Factor IX activity level in the third subject,
with a history of liver disease, also rose consistently and was at
16% of normal at three weeks post-administration. Data from a
natural history of patients with hemophilia suggest that
circulating factor activity levels sustained at a threshold of
greater than or equal to 12% of normal generally are considered to
be sufficient to reduce the risk of joint bleeds and the need for
prophylactic clotting factor infusions.
Over the combined 28 weeks of observation reported in the
abstract, none of the three subjects received regular infusions of
Factor IX concentrates to prevent bleeding events. Only one
precautionary infusion has taken place due to a suspected ankle
bleed in one subject two days after administration of vector.
SPK-9001 has been well-tolerated and no subjects have needed, or
received, immunosuppression.
“We are highly encouraged by these initial data, which are
supportive of the target profile of a potential gene therapy
product capable of eliminating the need for regular
infusions to control and prevent bleeding episodes
through a one-time, intravenous administration,” said
Katherine A. High, MD, co-founder, president and chief scientific
officer of Spark. “Our hypothesis had been that delivery of a
highly optimized gene therapy at low doses could allow expression
of therapeutic levels of FIX while avoiding the need for
immunosuppression. The data summarized in the abstract of SPK-9001
appear to support this hypothesis, although we will continue to
monitor and investigate the validity of the hypothesis as well
as assess long-term efficacy and safety of the product
candidate.”
Spark and Pfizer entered into a collaboration in 2014, under
which Spark will be responsible for conducting all Phase 1/2
studies for any product candidates that may be developed under the
SPK-FIX program, while Pfizer will assume responsibility for
pivotal studies, any regulatory activities and potential global
commercialization of any products that may result from the
collaboration.
“Although these results are early, we believe that the initial
SPK-9001 data are promising,” said Greg LaRosa, Chief Scientific
Officer, Rare Disease Research Unit, Pfizer. “Pfizer has a
longstanding commitment to the hemophilia community and strong
history of providing hemophilia products to patients for nearly two
decades. We look forward to our continued collaboration with Spark
as we work toward our shared goal of bringing to market a novel
potential therapy for hemophilia patients around the world.”
Up-to-date results from the trial will be presented at the EHA
Congress on June 11 by Dr. Spencer Sullivan, an Assistant Professor
of Pediatrics and Medicine at the University of Mississippi Medical
Center, one of the trial investigators. The trial is led by Dr.
Lindsey George of the Children’s Hospital of Philadelphia. In
addition, the work has been selected by the EHA for press briefing
at the Congress scheduled for June 11, 8:30 am CET.
About Hemophilia B and the
SPK-FIX ProgramHemophilia
B is a rare genetic blood disorder that affects approximately
4,000 males in the U.S. and 26,000 males worldwide. Current
treatment requires recurrent intravenous infusions of either
plasma-derived or recombinant Factor IX to control and prevent
bleeding episodes. Spark’s proprietary technology platform for
selecting, designing, manufacturing and formulating highly
optimized gene therapies was applied to developing SPK-9001, 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, with the potential to be a
one-time therapy. The SPK-FIX program leverages a long track record
of hemophilia B gene therapy research and clinical development
conducted by Spark and its founding scientific team over nearly
three decades. SPK-9001 is being developed under a partnership with
Pfizer.
About Spark TherapeuticsSpark is a gene therapy
leader seeking to transform the lives of patients with debilitating
genetic diseases by developing one-time, life-altering treatments.
Spark’s validated gene therapy platform is being applied to a range
of clinical and preclinical programs addressing serious genetic
diseases, including inherited retinal dystrophies, hematologic
disorders and neurodegenerative diseases. Spark’s most advanced
product candidate, SPK-RPE65 (voretigene neparvovec), 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 builds
on two decades of research, development and manufacturing at The
Children’s Hospital of Philadelphia, including human trials
conducted across diverse therapeutic areas and routes of
administration. 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 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 DiseasesRare 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 May 19, 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, including its 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
Investor Relations
Spark Therapeutics, Inc.
Stephen W. Webster
Chief Financial Officer
(855) SPARKTX (1-855-772-7589)
Media
Ten Bridge Communications
Dan Quinn
(781) 475-7974
dan@tenbridgecommunications.com
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