Data include eleventh participant in the trial
Annualized bleeding rate (ABR) for all 11 participants was reduced
by 97 percent, while annualized infusion rate (AIR) was
reduced 99 percent as of Nov. 29, 2017 data cutoff
PHILADELPHIA and NEW YORK, Dec. 11, 2017 (GLOBE NEWSWIRE) -- 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, with a
cumulative follow-up of more than 13 patient years of observation, all 11 participants in the ongoing Phase 1/2 clinical trial of
investigational SPK-9001 for the treatment of patients with hemophilia B had discontinued routine infusions of
factor IX concentrates and shown sustained steady-state factor IX activity levels with no serious adverse events, thrombotic events
or factor IX inhibitors observed. Based on individual participant history for the year prior to the study, the overall annualized
bleeding rate (ABR) was reduced by 97 percent (calculated based on data after week four; 95 percent based on data after infusion)
to a mean of 0.3 (0.5) annual bleeds, compared to a mean of 10.5 bleeds annually before SPK-9001 administration.
Overall annualized infusion rate (AIR) was reduced 99 percent (calculated based on data after week four; 97 percent based on data
after infusion) to a mean of 0.8 (1.7) annual infusions, compared to a mean of 62.5 infusions per year
before SPK-9001 administration. Data on all 11 participants were presented today by Lindsey A. George,
M.D., attending physician in the Division of Hematology at Children’s Hospital of Philadelphia and principal investigator
of the trial, at the 59th American Society of Hematology (ASH) Annual Meeting and Exposition in Atlanta.
“We believe these longer-term data are meeting critical goals of our hemophilia programs,” said Katherine A. High, M.D.,
president and head of research and development at Spark Therapeutics. “Now, with four participants followed for more than
18 months, we continue to see consistent levels of factor IX activity, no serious adverse events, as well as a sustained reduction
in both the symptoms of hemophilia and the prophylactic and disease management protocols that were used prior to infusion with
SPK-9001.”
As of the Nov. 29, 2017 data cutoff, the mean steady-state factor IX activity level at 12 weeks post-administration for the 11
participants was 36 percent of normal (range as of the data cutoff: 15 to 78 percent). As of the data cutoff, the last participant
to be infused, who received SPK-9001 manufactured using an enhanced process, was out eight months following
SPK-9001 infusion, with a mean factor IX activity level of 60 percent. Spark Therapeutics will enroll up to four
additional participants in the current Phase 1/2 clinical trial who will receive SPK-9001 manufactured using an enhanced
process to test its comparability to the SPK-9001 received by the first 10 participants enrolled in the ongoing trial.
In this open-label, non-randomized and multicenter Phase 1/2 clinical trial, there were no serious adverse events during or
following infusion with 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 administered factor for suspected joint bleeding, while a second
participant recorded one spontaneous bleed.
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.
Spark Therapeutics 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; and (iii) 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 U.S. 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
December 11, 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 Silva
communications@sparktx.com
(215) 282-7470
