Spark Therapeutics Presents Updated Preliminary Data from Hemophilia B Phase 1/2 Trial Suggesting Consistent and Sustained Le...
April 06 2017 - 7:30AM
As of data cutoff, the annualized bleeding rate
(ABR) has been reduced by 96 percent and the annualized infusion
rate (AIR) reduced by 99 percent
Spark Therapeutics (NASDAQ:ONCE) announced updated preliminary data
today from 10 infused participants in the ongoing Phase 1/2
clinical trial of investigational SPK-9001 for hemophilia B. All
participants have experienced consistent and sustained increases in
factor IX activity following administration of the investigational
therapy. These data will be presented at the Hemostasis and
Thrombosis Research Society (HTRS) 2017 Scientific Symposium in
Scottsdale, Arizona on Friday, April 7, by Adam Cuker, M.D.,
assistant professor of medicine at the Perelman School of Medicine
of the University of Pennsylvania and a clinical investigator at
Children’s Hospital of Philadelphia.
Data as of March 24, 2017 will be presented on 10 participants
in the study, who were dosed with a single administration of 5 x
1011 vector genomes (vg)/kg body weight. All participants have
discontinued routine infusions of factor IX concentrates. Based on
individual patient history prior to the study, ABR was reduced by
96 percent to a mean of 0.39 annual bleeds, compared with 9.2
bleeds before SPK-9001 administration. AIR was reduced 99 percent
to a mean of 0.98 annual infusions, compared with 68.5 infusions
before SPK-9001 administration.
As of the data cutoff, nine of the 10 infused participants have
not taken factor IX concentrates to prevent or control bleeding
events since vector administration. As previously reported, one
participant with severe joint disease has self-administered
precautionary infusions for persistent knee pain. The mean
steady-state factor IX activity level post 12 weeks treatment for
the 10 participants was a sustained 33 percent (range as of the
data cutoff: 14 to 81 percent). In the study to date, no serious
adverse events have been reported, including no factor IX
inhibitors and no thrombotic events. These data represent more than
2,400 cumulative patient days of exposure from the start of the
trial.
Two of the 10 participants experienced an asymptomatic,
transient elevation in liver enzymes, or decline in FIX activity,
potentially indicative of an immune response to the Spark100 vector
capsid, that occurred several weeks post infusion. Both
participants received a tapering dose of oral corticosteroids,
after which their alanine aminotransferase (ALT) levels returned to
baseline. The activity level of one of these participants has
stabilized at approximately 15 percent for more than nine weeks
post corticosteroid use. The other participant had a factor IX
activity level between 70 to 80 percent at completion of steroid
use.
“The additional preliminary data continue to support our initial
observations that a single intravenous administration of SPK-9001
has resulted in consistent and sustained levels of factor IX
activity for trial participants,” said Katherine A. High,
M.D., president and chief scientific officer at Spark Therapeutics.
“Notably, all participants to date have consistently achieved
our targeted therapeutic range of FIX activity. As we continue to
glean more insights from these preliminary data, our analysis
suggests that a tapering course of oral corticosteroids has been
well-tolerated and may help control potential capsid immune
responses following SPK-9001 infusion.”
These data from the Phase 1/2 clinical trial
of SPK-9001 will be presented during a poster session on
Friday, April 7, from 5:15-6:15 p.m. MST.
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.
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 Spark
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 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 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 biallelic 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 by the
FDA, and access to the PRIority MEdicines (PRIME) Program by the
EMA) 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.
Investor Contact:
Ryan Asay
Ryan.asay@sparktx.com
(215) 239-6424
Media Contact:
Monique da Silva
Monique.dasilva@sparktx.com
(215) 282-7470
Onconetix (NASDAQ:ONCE)
Historical Stock Chart
From Aug 2024 to Sep 2024
Onconetix (NASDAQ:ONCE)
Historical Stock Chart
From Sep 2023 to Sep 2024