– If approved, marstacimab has potential to be
a best-in-class treatment option among novel non-factor agents
–
Pfizer Inc. (NYSE: PFE) today announced that the first
participant has been dosed in the Phase 3 BASIS study of
marstacimab (PF-06741086), an anti-tissue factor pathway inhibitor
(anti-TFPI) being evaluated for the treatment of people with severe
hemophilia A or B, with or without inhibitors.
BASIS is a global Phase 3, open-label, multicenter study that
will evaluate annualized bleed rate (ABR) through 12 months on
prophylaxis treatment with marstacimab, an investigational, novel
subcutaneous therapy, in adolescents and adults with hemophilia A
or B compared to a run-in period on replacement therapy with FVIII
or FIX clotting factor, respectively, or bypass therapy (i.e.,
treatments that “bypass” the need for clotting factor treatment to
help the body form a normal clot). The primary endpoint is impact
on ABR through 12 months following prophylaxis treatment with
marstacimab. The incidence and severity of thrombotic events will
also be assessed.
“Our approach to hemophilia research includes the investigation
of multiple mechanisms to help address the needs of all people with
hemophilia, including those with hemophilia A or B, and with or
without inhibitors, and targeting TFPI provides a novel approach to
improve blood coagulation,” said Brenda Cooperstone, Chief
Development Officer, Rare Disease, Pfizer Global Product
Development. “Based on the Phase 2 study findings to date,
marstacimab may have the potential to offer improved bleed control
via subcutaneous injection and potentially eliminate the need for
prophylactic factor replacement, providing an enhanced treatment
option compared to factor replacement therapy.”
The completed Phase 2 study results demonstrated that treatment
with marstacimab showed significant (>75%) reductions in ABR for
all participants in the study population. The participants were
monitored in a long-term extension study, which showed sustained
efficacy up to 12 months and no thrombotic events or
treatment-related serious adverse events in 20 participants
receiving weekly subcutaneous marstacimab doses at or above the
dose to be studied in the BASIS Phase 3 pivotal trial (300 mg
subcutaneous loading followed by 150 mg subcutaneous weekly).
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 or B
(defined as factor VIII or factor IX activity <1%,
respectively), with or without inhibitors. Approximately 20% of
participants will be adolescents (ages between 12 to <18 years
old). This study is comparing treatment with a run-in period on
patients’ prescribed factor replacement therapy or bypass therapy
during a 6-month Observational Phase with a 12-month Active
Treatment Phase, during which participants will 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.
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). Marstacimab is in
development as a prophylactic treatment to prevent or reduce the
frequency of bleeding episodes in individuals with severe
hemophilia A or B (defined as factor VIII or factor IX activity
<1%, respectively) with or without inhibitors. In September
2019, the U.S. Food and Drug Administration (FDA) granted Fast
Track designation to marstacimab for use in combination with
inhibitors as a potential treatment for hemophilia A and B.
About Hemophilia
Hemophilia is a genetic hematological rare disease that results
in a deficiency of a protein that is required for normal blood
clotting—clotting factor VIII in hemophilia A and clotting factor
IX in hemophilia B. The severity of hemophilia that a person has is
determined by the amount of factor in the blood. The lower the
amount of the factor, the more likely it is that bleeding will
occur which can lead to serious health problems.
Hemophilia A occurs in approximately one in every 5,000-10,000
male births worldwide, and the incidence of hemophilia B is one in
25,000 male births. For people who live with hemophilia, there is
an increased risk of spontaneous bleeding as well as bleeding
following injuries or surgery. It is a lifelong disease that
requires constant monitoring and therapy.
About Pfizer Rare Disease
Rare disease includes some of the most serious of all illnesses
and impacts millions of patients worldwide, representing an
opportunity to apply our knowledge and expertise to help make a
significant impact on addressing unmet medical needs. The Pfizer
focus on rare disease builds on more than two decades of
experience, a dedicated research unit focusing on rare disease, and
a global portfolio of multiple medicines within a number of disease
areas of focus, including rare hematologic, neurologic, cardiac and
inherited metabolic disorders.
Pfizer Rare Disease combines pioneering science and deep
understanding of how diseases work with insights from innovative
strategic collaborations with academic researchers, patients, and
other companies to deliver transformative treatments and solutions.
We innovate every day leveraging our global footprint to accelerate
the development and delivery of groundbreaking medicines and the
hope of cures.
Click here to learn more about our Rare Disease portfolio and
how we empower patients, engage communities in our clinical
development programs, and support programs that heighten disease
awareness.
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
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 150 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.
PFIZER DISCLOSURE NOTICE:
The information contained in this release is as of November 23,
2020. 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 an
investigational hemophilia A / B therapy, marstacimab, including
its 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 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 marstacimab 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;
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, 2019 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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Pfizer Media: Steve Danehy 212-733-1538 Steven.Danehy@pfizer.com
Pfizer Investor: Chuck Triano 212-733-3901
Charles.E.Triano@pfizer.com
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