- Study demonstrates statistically significant and clinically
relevant reduction in annualized bleeding rate compared to
prophylaxis and on-demand intravenous regimens
- Marstacimab, if approved, has the potential to become the first
once-weekly subcutaneous treatment for people living with
hemophilia B and the first treatment administered as a flat dose
for people living with hemophilia A or B
- A novel, investigational anti-tissue factor pathway inhibitor,
marstacimab’s mechanism of action is differentiated from the
current standard for hemophilia
Pfizer Inc. (NYSE: PFE) today announced that the pivotal Phase 3
BASIS clinical trial (NCT03938792) evaluating marstacimab has met
its primary endpoints, having demonstrated statically significant
and clinically meaningful effects. Marstacimab, a novel,
investigational anti-tissue factor pathway inhibitor (anti-TFPI)
being studied for the treatment of hemophilia A or B for people
without inhibitors to Factor VIII (FVIII) or Factor IX (FIX), was
administered weekly with flat (not weight-based) dosing in the
trial as a subcutaneous 300 mg loading dose followed by 150 mg once
weekly.
“Despite significant treatment advances in recent years, many
people living with hemophilia unfortunately continue to experience
bleeding episodes and are required to manage their disease with
frequent intravenous infusions,” said Chris Boshoff, M.D., Ph.D.,
Chief Development Officer, Oncology and Rare Disease, Pfizer Global
Product Development. “These results support the potential for
marstacimab to become the first once-weekly non-factor treatment
for people with hemophilia B and a treatment option that helps
address the diverse needs of patients with hemophilia A or B
without inhibitors. These needs include preventing excessive or
potentially life-threatening bleeds, while at the same time
reducing the burden of treatment with once-weekly, subcutaneous
administration.”
The BASIS trial demonstrated that prophylactic treatment with
marstacimab resulted in a statistically significant and clinically
relevant reduction in annualized bleeding rate (ABR) in people
living with severe hemophilia A and moderately severe to severe
hemophilia B without inhibitors. 116 people living with hemophilia
were treated with marstacimab during a 12-month period versus a
prophylaxis and on-demand intravenous regimen with FVIII or FIX,
administered as part of usual care in the six-month lead-in period.
In the cohort of patients treated with on-demand factor replacement
intravenous therapy in the lead-in period, marstacimab demonstrated
superiority (P< 0.0001) with a 92% reduction in bleeds. The
results also showed superiority (p=0.0376) with marstacimab
compared to prophylaxis, with a 35% reduction in ABR.
The safety profile for marstacimab was consistent with Phase 1/2
results and treatment was generally well-tolerated. No deaths were
reported and there have been no thromboembolic events or events of
consumptive coagulopathy recorded in hemophilia patients that have
been enrolled in clinical trials investigating marstacimab.
Discovered by Pfizer scientists, marstacimab has a mechanism of
action that is differentiated from FVIII and FIX replacement
treatments that are the current treatment standard for hemophilia.
Instead of replacing missing or insufficient clotting factors,
marstacimab targets TFPI, one of the body’s natural mechanisms that
inhibits the initiation of blood clotting. By targeting the
Kunitz-2 domain of TFPI, marstacimab may help re-establish balance
between bleeding and blood clot formation.
Marstacimab is in development as a prophylactic treatment to
prevent or reduce the frequency of bleeding episodes in individuals
with severe hemophilia A or moderately severe to severe hemophilia
B, in each case with or without inhibitors. The inhibitor cohort of
the BASIS trial is completing enrollment and is expected to read
out as early as late 2024.
“These encouraging results from the BASIS trial demonstrate
marstacimab’s potential as a promising subcutaneous treatment
option that could offer improvement over the current standard of
care for many living with hemophilia,” said Davide Matino, M.D.,
M.Sc., Assistant Professor of Medicine, McMaster University. “If
approved, marstacimab may both prevent bleeds and reduce the
treatment burden that many people living with hemophilia A or B
without inhibitors face today.”
Analyses of the full Phase 3 dataset are ongoing, and results
will be presented at an upcoming scientific conference. Pfizer will
discuss these data with regulatory authorities, with the goal of
initiating regulatory filings in the coming months.
Pfizer currently has three Phase 3 programs investigating novel
treatment options for people living with hemophilia. In addition to
the BASIS study, fidanacogene elaparvovec and giroctocogene
fitelparvovec are investigational gene therapy treatments being
studied for the treatment of adults living with hemophilia B and
hemophilia A, respectively.
About the BASIS study
BASIS is a global Phase 3, open-label, multicenter study
evaluating annualized bleed rate through 12 months on treatment
with marstacimab, an investigational, novel subcutaneous therapy
option, in approximately 145 adolescent and adult participants
between ages 12 to <75 years with severe hemophilia A (defined
as FVIII <1%) or moderately severe to severe hemophilia B
(defined as FIX activity <=2%), with or without inhibitors.
Approximately 20% of participants are adolescents (ages between 12
to <18 years old). This study is comparing treatment with a
run-in period for patients’ prescribed factor replacement therapy
or bypass therapy during a 6-month Observational Phase with a
12-month Active Treatment Phase, during which participants receive
prophylaxis (a 300 mg subcutaneous loading dose of marstacimab,
followed by 150 mg subcutaneously once weekly) with potential for
dose escalation to 300 mg once weekly.
Pfizer is also conducting BASIS KIDS, an open-label study
investigating the safety and efficacy of marstacimab in children
<18 years of age with severe hemophilia A or moderately severe
to severe hemophilia B with or without inhibitors. The study will
compare 12 months of historical standard treatment to marstacimab
prophylaxis.
About Marstacimab (PF-06741086)
Marstacimab (PF-06741086) is a human monoclonal immunoglobulin G
isotype, subclass 1 (IgG1) that targets the Kunitz 2 domain of
tissue factor pathway inhibitor (TFPI), a natural anticoagulation
protein that functions to prevent the formation of blood clots.
Marstacimab is in development as a prophylactic treatment to
prevent or reduce the frequency of bleeding episodes in individuals
with severe hemophilia A or moderately severe to severe hemophilia
B with or without inhibitors.
In September 2019, the U.S. Food and Drug Administration (FDA)
granted Fast Track designation to marstacimab for routine
prophylaxis to prevent or reduce the frequency of bleeding episodes
in hemophilia A with inhibitors or hemophilia B with
inhibitors.
About Hemophilia
Hemophilia is a family of rare genetic blood diseases caused by
a clotting factor deficiency (FVIII in hemophilia A, FIX in
hemophilia B) which prevents normal blood clotting. Hemophilia is
diagnosed in early childhood and impacts more than 400,000 people
worldwide.1 The inability of the blood to clot properly can
increase the risk of painful bleeding inside the joints, which can
cause joint scarring and damage. People living with hemophilia can
suffer permanent joint damage following repeated bleeding
episodes.1,2
For decades, the most common treatment approach for hemophilia A
and B has been factor replacement therapy, which replaces the
missing clotting factors. Factor replacement therapies increase the
amount of clotting factor in the body to levels that improve
clotting, resulting in less bleeding.2,3 Approximately 25-30% of
people with hemophilia A and 3-5% of people with hemophilia B are
unable to continue taking factor replacement therapies because they
develop inhibitors to FVIII and FIX.4,5
About Pfizer: 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
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 170 years, we have worked to make a difference for all
who rely on us. We routinely post information that may be important
to investors on our website at www.pfizer.com. In addition, to
learn more, please visit us on www.pfizer.com and follow us on
Twitter at @Pfizer and @Pfizer_News, LinkedIn, YouTube and like us
on Facebook at Facebook.com/Pfizer.
DISCLOSURE NOTICE: The information contained in this release is
as of May 30, 2023. 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
marstacimab, an investigational anti-tissue factor pathway
inhibitor, and Pfizer’s hemophilia programs for fidanacogene
elaparvovec and giroctocogene fitelparvovec, 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; whether or when the inhibitor cohort of
the BASIS trial will be successful; 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 any applications may be filed with regulatory
authorities for marstacimab, fidanacogene elaparvovec or
giroctocogene fitelparvovec; whether and when regulatory
authorities 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 marstacimab,
fidanacogene elaparvovec and giroctocogene fitelparvovec 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 marstacimab, fidanacogene elaparvovec and giroctocogene
fitelparvovec; uncertainties regarding the impact of COVID-19 on
our 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, 2022 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.
______________________________ 1 Srivastava A, Santagostino E,
Dougall A, et al. WFH guidelines for the management of hemophilia,
3rd Edition; 2020. Haemophilia, 26(S6), 1–158.
https://doi.org/10.1111/hae.14046. 2 Centers for Disease Control
and Prevention. Hemophilia. Last Reviewed: April 2023.
https://www.cdc.gov/dotw/hemophilia/index.html. 3 Weyand AC, Pipe
SW. New therapies for hemophilia. Blood 2019;133(5):389–398. doi:
https://doi.org/10.1182/blood-2018-08-872291. 4 Centers for Disease
Control and Prevention. Inhibitors and hemophilia. Last reviewed:
April 2023. https://www.cdc.gov/ncbddd/hemophilia/inhibitors.html.
5 Peyvandi F, Garagiola I, Seregni S. Future of coagulation factor
replacement therapy. J Throm Haemost. 2013;11 (Suppl. 1):84–98.
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