Pfizer Inc. (NYSE: PFE) today announced positive top-line
results from the Phase 3 BENEGENE-2 study (NCT03861273) evaluating
fidanacogene elaparvovec, an investigational gene therapy, for the
treatment of adult males with moderately severe to severe
hemophilia B.
The BENEGENE-2 study met its primary endpoint of non-inferiority
and superiority in the annualized bleeding rate (ABR) of total
bleeds post-fidanacogene elaparvovec infusion versus prophylaxis
regimen with Factor IX (FIX), administered as part of usual care.
The results demonstrated superiority with a mean ABR for all bleeds
of 1.3 for the 12 months from week 12 to month 15 compared to an
ABR of 4.43 during the lead-in pre-treatment period of at least six
months, resulting in a 71% reduction in ABR (p<0.0001) after a
single dose of 5e11 vg/kg of fidanacogene elaparvovec. Key
secondary endpoints demonstrated a 78% reduction in treated ABR
(p=0.0001) and a 92% reduction in annualized infusion rate
(p<0.0001). Mean FIX activity was 27% at 15 months by one-stage
SynthASil assay and 25% at 24 months. The mean steady-state FIX:C
was significantly higher than the pre-specified threshold of 5%
(p<0.0001).
Fidanacogene elaparvovec was generally well-tolerated, with a
safety profile consistent with Phase 1/2 results. Fourteen serious
adverse events (SAEs) were reported in seven (16%) patients, with
two assessed as related to treatment, a duodenal ulcer hemorrhage
occurring in the setting of corticosteroid use, and an
immune-mediated elevation of liver aminotransferase levels. No
deaths, SAEs associated with infusion reactions, thrombotic events,
or FIX inhibitors were reported.
Fidanacogene elaparvovec is a novel, investigational vector that
contains a bio-engineered adeno-associated virus (AAV) capsid
(protein shell) and a high-activity human coagulation FIX gene. The
goal of this gene therapy for people living with hemophilia B, once
treated, is that they will be able to produce FIX via this one-time
treatment rather than having to regularly receive exogenous FIX. In
this Phase 3 trial, eligible study participants (n=45) completed a
minimum six months of routine exogeneous FIX prophylaxis therapy
during the lead-in study (NCT03587116) and then received one
intravenous dose of fidanacogene elaparvovec at a dose of 5e11
vg/kg. Participants in the BENEGENE-2 study were screened with a
validated assay designed to identify individuals who test negative
for neutralizing antibodies to the gene therapy vector. Clinical
trial participants will be evaluated as part of a long-term study
over the course of 15 years.
“Pfizer has more than 30 years of experience in developing and
commercializing therapies for hematological disorders, and a deep
understanding of the significant challenges that people living with
hemophilia continually face. We are proud to advance the latest
innovation for people living with hemophilia B and are encouraged
by the potential of this investigational gene therapy,” said Chris
Boshoff, M.D., Ph.D., Chief Development Officer, Oncology and Rare
Disease, Pfizer Global Product Development. “We are extremely
appreciative of those who are participating in the trial and to the
investigators contributing to this innovative research as we work
to unlock the full potential of gene therapies for people living
with hemophilia.”
Pfizer currently has three Phase 3 programs investigating gene
therapy in populations where there is a high unmet need: hemophilia
B, hemophilia A, and Duchenne muscular dystrophy. A Phase 3 trial
is also ongoing investigating marstacimab, a potential novel
subcutaneous therapy option being studied for the treatment of
people with hemophilia A and B with and without inhibitors.
“The burden people living with hemophilia B face is significant,
with many receiving routine infusions or injections which can
interfere with their ability to take part in day-to-day activities
that many take for granted,” said Adam Cuker, M.D., M.S., Director,
Penn Comprehensive and Hemophilia Thrombosis Program. “The
BENEGENE-2 data demonstrate the promise of this gene therapy
candidate as a potential one-time option for people living with
hemophilia B as a means of reducing the clinical and treatment
burden over the long term.”
Fidanacogene elaparvovec has been granted breakthrough,
regenerative medicines advance therapy (RMAT), and orphan drug
designations from the US Food and Drug Administration, as well as
PRIority MEdicines (PRIME) and orphan drug designation from the
European Medicines Agency. Pfizer will discuss these data with
regulatory authorities in early 2023.
Additional key data will be presented at a scientific conference
in early 2023 as well.
About fidanacogene elaparvovec
Fidanacogene elaparvovec is a novel, investigational gene
therapy that contains a bio-engineered AAV capsid and a
high-activity human coagulation FIX gene. The goal of this
investigational treatment for people living with hemophilia B, once
treated, is that they will be able to produce FIX themselves via
this one-time treatment rather than having to receive exogenous
FIX.
Pfizer licensed SPK-9001 (fidanacogene elaparvovec), from Spark
Therapeutics pursuant to a December 2014 agreement under which
Spark Therapeutics was responsible for conducting all Phase 1/2
studies for the investigational gene therapy while Pfizer assumed
responsibility for pivotal studies, any regulatory activities, and
potential global commercialization.
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.
According to the World Federation of Hemophilia, more than
38,000 people worldwide were living with hemophilia B in 2021.
People with hemophilia B have a deficiency in clotting FIX, a
specific protein in the blood. Hemophilia B also is called
congenital FIX deficiency or Christmas disease. The current
standard of care requires recurrent intravenous infusions of either
plasma-derived or recombinant FIX to control and prevent bleeding
episodes.
Pfizer Inc.: Breakthroughs that Change Patients’
Lives
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, including innovative medicines and vaccines. 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
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providers, governments and local communities to support and expand
access to reliable, affordable health care around the world. For
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DISCLOSURE NOTICE: The information contained in this release is
as of December 29, 2022. 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
fidanacogene elaparvovec, Pfizer’s other gene therapy candidates,
marstacimab and the potential of gene therapy, 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 endpoints, commencement and/or completion
dates for our clinical trials, regulatory submission dates,
regulatory approval dates and/or launch dates, as well as the
possibility of unfavorable new clinical data and further analyses
of existing clinical data; the risk that clinical trial data are
subject to differing interpretations and assessments by regulatory
authorities; whether regulatory authorities will be satisfied with
the design of and results from our clinical studies; whether and
when drug applications for any potential indications for
marstacimab or any of Pfizer’s other gene therapy candidates may be
filed in any jurisdictions; whether and when regulatory authorities
in any jurisdictions may approve any such applications, which will
depend on myriad factors, including making a determination as to
whether the product's benefits outweigh its known risks and
determination of the product's efficacy and, if approved, whether
fidanacogene elaparvovec, marstacimab or any of Pfizer’s other gene
therapy candidates will be commercially successful; decisions by
regulatory authorities impacting labeling, manufacturing processes,
safety and/or other matters that could affect the availability or
commercial potential of fidanacogene elaparvovec, marstacimab or
any of Pfizer’s other gene therapy candidates; uncertainties
regarding the impact of COVID-19 on Pfizer’s business, operations
and financial results; 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, 2021 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.
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