All four subjects received one-time
administration of SPK-9001 at initial low dose for a cumulative
58-weeks of follow-up without the need for
immunosuppression
Spark Therapeutics (NASDAQ:ONCE) announced today updated results of
the first cohort from the ongoing Phase 1/2 clinical trial of
SPK-9001, the lead investigational candidate in its SPK-FIX
program, which is being studied for the treatment of hemophilia
B. The company also provided an update on preclinical data in
its SPK-FVIII program for the treatment of hemophilia A.
“Spark’s validated platform has now successfully delivered gene
therapies with proof of concept data in both the eye and the liver.
We have made strong, tangible progress throughout our entire
hemophilia franchise,” said Jeffrey D. Marrazzo, co-founder and
chief executive officer of Spark. “The clinical data emerging
from our hemophilia B program, which is partnered with Pfizer, and
the strong parallels to the preclinical data from our rapidly
progressing program in hemophilia A, give us early confidence in
achieving our goal of eliminating the need for regular infusions to
control and prevent bleeding episodes in patients with these
diseases through a potentially one-time, intravenous administration
of highly optimized gene therapies."
Hemophilia B Data Overview
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. SPK-9001 is being developed
in collaboration with Pfizer Inc. (NYSE:PFE). Data from the
Phase 1/2 clinical trial of SPK-9001 were presented on June 11 at
the 21st Congress European Hematology Association (EHA) by Dr.
Spencer Sullivan, an Assistant Professor of Pediatrics and Medicine
at the University of Mississippi Medical Center and a trial
investigator, and were an extension of the data released on May
19th in the initial EHA abstract.
Data presented today show that the low dose cohort of four
subjects enrolled in the study experienced consistent and sustained
factor IX activity levels following a single administration of
SPK-9001 at the initial dose level (5 x 1011 vg/kg) studied in the
trial. Factor IX activity in the first two subjects, which had no
history of liver disease, rose consistently and have stabilized at
28% of normal through the first approximately twenty-six weeks
post-administration in the first subject, and at 41% of normal at
fifteen weeks post-administration in the second. Factor IX activity
level in the third subject, with a history of liver disease, also
rose consistently and was at 26% of normal at approximately ten
weeks post-administration. The fourth subject, also with a history
of liver disease and not included in the previously disclosed data,
saw a clinical response similar to the earlier subjects and was at
33% of normal through approximately seven weeks.
To date, over a combined 58 weeks of observation, none of the
first four subjects received regular infusions of factor IX
concentrates to prevent bleeding events. One precautionary infusion
took place due to a suspected ankle bleed in subject number three
two days after administration. Based on their pre-enrollment
histories, it is estimated that the four subjects followed to date
would have received more than 100 infusions of recombinant factor
IX over the period of the study to prevent or treat bleeds as part
of their normal care.
Across the cohort, we saw no sustained elevation in liver enzyme
levels and no drop in factor IX levels. To date, SPK-9001 has
been well-tolerated and no subjects have needed, or received,
immunosuppression.
Hemophilia A Program
Spark today highlighted preclinical results from SPK-8011, the
lead investigational candidate in its SPK-FVIII program for
hemophilia A. SPK-8011 was developed using Spark's
proprietary technology platform and uses Spark200, a novel
bio-engineered adeno-associated virus (AAV) capsid optimized for
more efficient transduction of human hepatocytes, and contains an
optimized B-domain deleted FVIII expression cassette. Spark retains
global commercialization rights to its SPK-FVIII program for
hemophilia A.
Data presented at the meeting of the American Society of Gene
and Cell Therapy in May demonstrated strong preclinical data,
specifically FVIII expression in the 15% to 35% range in
preclinical models at clinically relevant doses of 2 x 1012
vg/kg.
Today, the company released additional preclinical data,
utilizing an in vitro assay, of the Spark200 capsid demonstrated
higher efficiency in transducing human hepatocytes compared to
Spark100, the capsid used in SPK-9001.
“We believe that the key to the hemophilia B results was the
optimization of SPK-9001, which allowed us to observe therapeutic
levels of FIX activity utilizing a low dose. The data
emerging from our hemophilia A program show parallels to the
preclinical data in hemophilia B,” said Katherine A. High, MD,
co-founder, president and chief scientific officer of Spark. "We
remain on track to rapidly progress SPK-8011 for hemophilia A into
a Phase 1/2 clinical trial in the second half of 2016, and to
potentially see the first human data from this program in the first
half of 2017.”
Conference Call InformationSpark management
will discuss these data in a conference call on Monday, June
13, 2016 at 8:30 a.m. ET. A slide presentation will
also be available on the Events page of the company's website,
www.sparktx.com, to accompany the conference call. The call
can be accessed by dialing (855) 851-4526 (domestic) or (720)
634-2901 (international), and entering passcode 27643823.
About Hemophilia A and Hemophilia BHemophilia
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. Hemophilia A is
more common than hemophilia B. The incidence of hemophilia A is one
in 5,000 male births. People with hemophilia A have a deficiency in
clotting factor VIII, a specific protein in the blood. Hemophilia A
is also called congenital factor VIII deficiency or classic
hemophilia. Current standard of care requires recurrent intravenous
infusions of either plasma-derived or recombinant factor VIII to
control and prevent bleeding episodes. 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 and SPK-FVIII ProgramsSpark's
proprietary technology platform for selecting, designing,
manufacturing and formulating highly optimized gene therapies was
applied to developing compounds in the SPK-FIX and SPK-FVIII
programs. The SPK-FIX and SPK-FVIII programs both leverage a long
track record of hemophilia gene therapy research and clinical
development conducted by Spark 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 and Pfizer entered into a collaboration in 2014 for the
SPK-FIX program, including SPK-9001, under which Spark will be
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-8011, the lead investigational candidate from our SPK-FVIII
program in hemophilia A, is a novel bio-engineered adeno-associated
virus (AAV) capsid optimized for more efficient transduction of
human hepatocytes expressing a codon-optimized B-domain deleted
factor VIII transgene enabling endogenous production of factor
VIII. Spark retains global commercialization rights to its
SPK-FVIII program.
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’s 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’s validated platform has successfully delivered gene
therapies with proof-of-concept data in the eye and liver. 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’s 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; (ii) our overall collaboration with Pfizer may not be
successful; and (iii) preclinical SPK-8011 data may not be
predictive of clinical results, if any, seen in human clinical
trials. 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.
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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