Together, all nine participants have reduced
infusions of factor IX concentrates by 99 percent over cumulative
1,650 days
Spark Therapeutics (NASDAQ:ONCE) and Pfizer Inc. (NYSE:PFE)
announced updated preliminary data today from the first nine
infused participants in the ongoing Phase 1/2 clinical trial of
investigational SPK-9001 for hemophilia B, who received a single
administration of 5 x 1011 vector genomes (vg)/kg body weight.
These data will be presented at today’s media briefing and on
Sunday, Dec. 4 at the Plenary Scientific Session of the 58th
American Society of Hematology (ASH) Annual Meeting, in San Diego.
The first participant to reach one year in the study, who has
been followed for 52 weeks post-infusion of SPK-9001, has reduced
to zero his number of intravenous factor IX infusions without
having any bleeds. In the year before administration of
SPK-9001, he infused factor IX concentrates prophylactically a
total of 98 times and still experienced four traumatic bleeds. As
of Nov. 30, 2016, his steady-state factor IX activity level was 33
percent of normal.
As of Nov. 30, 2016, the total clotting factor IX concentrates
consumption in all nine infused participants over a cumulative
1,650 patient days following vector administration was reduced by
1.13 million international units based on their factor IX
concentrates usage in the year before vector administration. Seven
of the nine infused participants, who have progressed to at least
12 weeks post-vector administration as of Nov. 30, 2016,
experienced consistent and sustained factor IX activity levels,
with a mean steady-state level greater than 28 percent. In the
study to date, no participants developed factor IX inhibitors or
experienced thrombotic events after the vector administration. No
serious adverse events after SPK-9001 administration have been
reported.
Two of the nine participants experienced asymptomatic, transient
elevation in liver enzymes associated with an immune response to
the Spark100 vector capsid during the first four to eight weeks
following administration. In one participant, this response was
accompanied by a decline in factor IX activity level from 32 to 12
percent, as of Nov. 30, 2016. In the other participant, to whom
steroids were administered more promptly, this response was
accompanied by a decline from a peak factor IX activity level of 71
to 68 percent, as of Nov. 30, 2016. Both participants were put on a
tapering course of corticosteroids, and, to date, neither
participant has experienced any bleeds nor has required infusion of
replacement factor IX concentrates.
“We will monitor these two participants carefully as we continue
to taper the corticosteroids,” said Katherine A. High, M.D.,
president and chief scientific officer at Spark Therapeutics. “It’s
important to remember that the immune response to the capsid
typically is transient, and in both cases, seems to have been
arrested by corticosteroids. Once corticosteroids have been
discontinued altogether, levels of expression will be the best
measure of the efficacy of this approach. The experience we have
gained in immuno-monitoring and in clinical management of the
immune response in the hemophilia B trial will further inform our
upcoming hemophilia studies.”
Eight of the infused participants have required no factor IX
concentrates to prevent or control bleeding events since the day
after vector administration. One participant with severe joint
disease self-administrated a precautionary infusion two days after
administration of SPK-9001 for a suspected ankle bleed and again at
week 35, post the data cut-off date and despite a factor IX
activity level of 36 percent, for a suspected knee bleed.
“Giving people living with hemophilia B treatment options that
potentially minimize or eliminate the need for infusions of factor
concentrates is one of the main goals of conducting clinical
studies of investigational gene therapies,” said Dr. High. “While
continued observation and larger cohorts are needed, these updated
preliminary data continue to be encouraging and suggest the
potential of investigational SPK-9001 to deliver a consistent,
sustained and therapeutically meaningful level of factor IX
activity through a one-time intravenous administration.”
Additionally, to date, six of seven participants reported
increased physical activity and improved quality of life based on
the Haemophilia Quality of Life Questionnaire for Adults, a
validated instrument that measures health-related quality of life
in adults (≥ 17 years of age) with hemophilia. Two participants
received the gene therapy product too recently to evaluate
quality-of-life measures.
Presentation Details:ASH Annual Meeting Media
BriefingPresenter: Katherine A. High, M.D., president and
chief scientific officer at Spark TherapeuticsDate: Saturday, Dec.
3, 2016Briefing time: 8-9 a.m. PSTLocation: San Diego Convention
Center, Room 22
SPK-9001: Adeno-Associated Virus Mediated Gene Transfer for
Hemophilia B Achieves Sustained Mean Factor IX Activity Levels of
>30% without Immunosuppression (Abstract # 91358)Presenter:
Lindsey George, M.D., Children’s Hospital of PhiladelphiaDate:
Sunday, Dec. 4, 2016
Session time: 2-4 p.m. PSTLocation: San
Diego Convention Center, Hall AB
Spark Therapeutics Conference Call:Spark
Therapeutics management will also host a conference call on Monday,
Dec. 5, 2016, at 8 a.m. ET to discuss the data presented at the
meeting. The conference call can be accessed by dialing (855)
851-4526 (domestic) or (720) 634-2901 (international), and entering
passcode 29837698. To access a live audio webcast, please
visit the “Investors” section at www.sparktx.com.
A replay of the call will be available for one week following
the call and can be accessed by dialing (855) 859-2056 (domestic)
or (404) 537-3406 (international), and entering passcode 29837698
or by visiting the “Investors” section at www.sparktx.com.
About Hemophilia BHemophilia, 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, is common almost exclusively 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. 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 and SPK-9001Spark
Therapeutics' proprietary technology platform for selecting,
designing, manufacturing and formulating 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, investigational 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. SPK-9001 has received breakthrough therapy and
orphan product designations from the U.S. Food and Drug
Administration.
About Spark TherapeuticsSpark Therapeutics, a
fully integrated company, is challenging the inevitability of
genetic disease by discovering, developing, and delivering gene
therapies that address inherited retinal diseases (IRDs),
liver-mediated diseases such as hemophilia, and neurodegenerative
diseases. Our validated platform successfully has delivered
proof-of-concept data with investigational gene therapies in the
retina and liver. Our most advanced investigational candidate,
voretigene neparvovec, in development for the treatment of
RPE65-mediated IRD, has received orphan designations in the U.S.
and European Union, and breakthrough therapy designation in the
U.S. The pipeline also includes SPK-7001, in a Phase 1/2 trial for
choroideremia, and two hemophilia development programs: SPK-9001 in
a Phase 1/2 trial for hemophilia B being developed in collaboration
with Pfizer (which also has received both breakthrough therapy and
orphan product designations) and SPK-8011, a preclinical candidate
for hemophilia A to which Spark Therapeutics retains global
commercialization rights. To learn more about us and our growing
pipeline, visit www.sparktx.com.
Spark Cautionary Note on Forward-looking
StatementsThis 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 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 Dec. 3, 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.
Spark Therapeutics Corporate Contacts
Stephen W. Webster, Chief Financial Officer
Daniel Faga, Chief Business Officer
(855) SPARKTX (1-855-772-7589)
Media Contact
Dan Quinn
Ten Bridge Communications
(781) 475-7974
dan@tenbridgecommunications.com
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