Spark Therapeutics Announces $15 Million Milestone Payment from Pfizer for Progress in Hemophilia B Gene Therapy Program
January 04 2017 - 7:30AM
Spark Therapeutics (NASDAQ:ONCE), a fully integrated gene therapy
company dedicated to challenging the inevitability of genetic
disease, today announced that it has earned a $15 million
payment from Pfizer Inc. (NYSE:PFE) for achieving a
pre-specified safety and efficacy profile development milestone in
the ongoing hemophilia B Phase 1/2 trial of investigational
SPK-9001. SPK-9001 has received breakthrough therapy and orphan
product designations from the U.S. Food and Drug Administration.
“We continue to make strong, tangible progress with our
hemophilia pipeline, and achievement of this second milestone marks
further advancement in the development of our investigational gene
therapy for hemophilia B,” said Jeffrey D. Marrazzo, chief
executive officer of Spark Therapeutics. “We look forward to
reporting additional data as we continue to document the clinical
experience with SPK-9001.”
Under the terms of the license agreement with Pfizer, Spark
Therapeutics received a $20 million upfront payment upon
entering into the agreement in 2014, and a $15 million milestone
payment in December 2015 for progress with the development program.
The company is eligible to receive up to an additional $230
million in aggregate for achieving future development and
commercial milestones, as well as royalties calculated as a
low-teen percentage of net sales on any potential SPK-FIX products.
Spark Therapeutics maintains responsibility for the clinical
development of SPK-FIX product candidates through the
completion of Phase 1/2 trials. Thereafter, Pfizer has
responsibility for further clinical development, achieving
regulatory approvals and potential global commercialization.
The SPK-FIX program leverages a more than two-decade long
history of hemophilia gene therapy research and clinical
development conducted by Spark Therapeutics and its founding
scientific team. 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.
About Spark TherapeuticsSpark Therapeutics, a
fully integrated company, strives to challenge the inevitability of
genetic disease by discovering, developing, and delivering gene
therapies that address inherited retinal diseases (IRDs),
neurodegenerative diseases, as well as diseases that can be
addressed by targeting the liver. 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 (which also has received
both breakthrough therapy and orphan product designations) in a
Phase 1/2 trial for hemophilia B being developed in collaboration
with Pfizer, and SPK-8011, in a Phase 1/2 trial 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.
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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