Pfizer Inc. (NYSE: PFE) and Sangamo Therapeutics, Inc. (Nasdaq:
SGMO), a genomic medicines company, today announced that the first
participant has been dosed in the Phase 3 AFFINE study of
giroctocogene fitelparvovec (SB-525), an investigational gene
therapy for hemophilia A patients.
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AFFINE is a global Phase 3, open-label, multicenter, single arm
study that will evaluate the efficacy and safety of giroctocogene
fitelparvovec in patients with moderately severe to severe
hemophilia A. The primary endpoint is impact on annual bleed rate
(ABR) through 12 months following treatment with giroctocogene
fitelparvovec, compared to ABR on Factor VIII (FVIII) replacement
therapy collected in the Phase 3 lead-in study period. Participants
will be analyzed throughout the 5-year study period following the
single infusion to further assess the durability and efficacy.
“The initiation of the pivotal Phase 3 dosing study of
giroctocogene fitelparvovec is a significant achievement for Pfizer
as we continue our longstanding commitment to improving care for
the hemophilia community,” said Brenda Cooperstone, Chief
Development Officer, Rare Disease, Pfizer Global Product
Development. “Enrollment in the lead-in study is progressing well
and recruitment is on track for Phase 3. Given the Phase 1/2 study
findings to date, we believe that giroctocogene fitelparvovec has
the potential to sustain factor levels and reduce annual bleed
rates, suggesting this one-time gene therapy could potentially
transform the standard of care for eligible patients
worldwide.”
Data from the Phase 3 lead-in study will provide a baseline for
patients evaluated in the Phase 3 study. Updated Phase 1/2 data
announced at a Pfizer investor event on September 15, 2020
demonstrated that giroctocogene fitelparvovec was generally well
tolerated. Each of the five patients in the high dose cohort
sustained FVIII activity levels without bleeds or the need for
prophylactic factor through up to 85 weeks. Factor VIII activity
levels were sustained at a clinically meaningful level, with a
geometric mean of ~71% when measured between the weeks of 9 and
52.
“We are encouraged that findings from the Phase 1/2 Alta study
met two critically important measures for the hemophilia A patient
community, showing clinically meaningful factor levels and reduced
bleeds,” said Bettina M. Cockroft, M.D., M.B.A, Chief Medical
Officer of Sangamo. “The progress of this program, the most
advanced of our gene therapy product candidates, into Phase 3 is an
important milestone for Sangamo, as it represents our first asset
in a registrational trial.”
Per the terms of the collaboration agreement, Sangamo has now
earned a $30 million milestone payment. The giroctocogene
fitelparvovec collaboration was established in May 2017. Under the
terms, Pfizer is now operationally and financially responsible for
research, development, manufacturing and commercialization
activities for giroctocogene fitelparvovec following the transfer
of the Investigational New Drug (IND) from Sangamo to Pfizer in
December 2019. Sangamo is eligible to receive total potential
milestone payments of up to $300 million for the development and
commercialization of giroctocogene fitelparvovec, and up to $175
million for additional hemophilia A gene therapy product candidates
that may be developed under the collaboration. Sangamo will,
additionally, receive tiered royalties starting in the low teens
and up to 20% of annual net sales of giroctocogene
fitelparvovec.
About the AFFINE study
The Phase 3 AFFINE (efficAcy and saFety
Factor vIii geNe thErapy in hemophilia
A patients; NCT04370054) study is an open-label, multicenter,
single arm study to evaluate the efficacy and safety of a single
infusion of giroctocogene fitelparvovec in more than 60 adult (ages
18-64 years) male participants with moderately severe to severe
hemophilia A. Eligible study participants will have completed at
least six months of routine FVIII prophylaxis therapy during the
lead-in Phase 3 study (NCT03587116) in order to collect
pretreatment data for efficacy and selected safety parameters.
The primary endpoint is impact on ABR through 12 months
following treatment with giroctocogene fitelparvovec, as compared
to ABR on prior FVIII prophylaxis replacement therapy. The
secondary endpoint is FVIII activity level after the onset of
steady state and through 12 months following infusion of
giroctocogene fitelparvovec.
About giroctocogene fitelparvovec
Giroctocogene fitelparvovec (SB-525 or PF-07055480) comprises a
recombinant adeno-associated virus serotype 6 vector (AAV6)
encoding the complementary deoxyribonucleic acid for B domain
deleted human FVIII. The giroctocogene fitelparvovec expression
cassette was designed for optimal liver-specific expression of
FVIII protein and supports production of high yields of the vector.
The giroctocogene fitelparvovec transcriptional cassette
incorporates multi-factorial modifications to the liver-specific
promoter module, FVIII transgene, synthetic polyadenylation signal
and vector backbone sequence.
The U.S. Food and Drug Administration has granted Orphan Drug,
Fast Track, and regenerative medicine advanced therapy (RMAT)
designations to giroctocogene fitelparvovec, which also received
Orphan Medicinal Product designation from the European Medicines
Agency. Giroctocogene fitelparvovec is being developed as part of a
global collaboration between Sangamo and Pfizer.
About Hemophilia A
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. 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. For people who live with hemophilia A, 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 Sangamo Therapeutics
Sangamo Therapeutics is committed to translating ground-breaking
science into genomic medicines with the potential to transform
patients’ lives using gene therapy, ex vivo gene-edited cell
therapy, and in vivo genome editing and gene regulation. For more
information about Sangamo, visit www.sangamo.com.
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.
SANGAMO DISCLOSURE NOTICE:
This press release contains forward-looking statements regarding
Sangamo's current expectations. These forward-looking statements
include, without limitation, statements relating to the potential
to develop, obtain regulatory approvals for and commercialize
SB-525 as a safe and effective therapy to treat hemophilia A, the
potential long-term durability of SB-525 therapy, anticipated plans
and timelines for conducting phase 3 clinical trials and sharing
additional clinical data, the potential for Sangamo to earn
milestone payments and royalties under its collaboration with
Pfizer and the timing of such payments and royalties and other
statements that are not historical fact. These statements are not
guarantees of future performance and are subject to risks and
uncertainties that are difficult to predict. Sangamo’s actual
results may differ materially and adversely from those expressed.
There can be no assurance that Sangamo will earn any additional
milestone or royalty payments under the Pfizer collaboration.
Factors that could cause actual results to differ include, but are
not limited to, risks and uncertainties related to: the evolving
COVID-19 pandemic and its impact on the global business
environment, healthcare systems and the business and operations of
Sangamo and Pfizer; the research and development process; the
uncertain timing and unpredictable results of clinical trials,
including whether final clinical trial data will validate the
safety and efficacy of SB-525; the unpredictable regulatory
approval process for product candidates across multiple regulatory
authorities; the manufacturing of products and product candidates;
the commercialization of approved products; the potential for
technological developments that obviate technologies used by
Sangamo and Pfizer in SB-525; the potential for Pfizer to terminate
the SB-525 program or to breach or terminate its collaboration
agreement with Sangamo; and the potential for Sangamo for fail to
realize its expected benefits of its collaboration with Pfizer.
These risks and uncertainties are described more fully in Sangamo's
filings with the U.S. Securities and Exchange Commission, including
its most recent Quarterly Report on Form 10-Q for the quarter ended
June 30, 2020 and Annual Report on Form 10-K for the year ended
December 31, 2019. The information contained in this release is as
of October 7, 2020, and Sangamo undertakes no duty to update
forward-looking statements contained in this release except as
required by applicable laws.
PFIZER DISCLOSURE NOTICE:
The information contained in this release is as of October 7,
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 therapy, giroctocogene fitelparvovec
(SB-525, or PF-07055480), 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; risks
associated with interim 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
giroctocogene fitelparvovec 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 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 giroctocogene fitelparvovec; 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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version on businesswire.com: https://www.businesswire.com/news/home/20201007005049/en/
Pfizer Media Contact: Steve Danehy 212-733-1538
Steven.Danehy@pfizer.com
Pfizer Investor Contact: Chuck Triano 212-733-3901
Charles.E.Triano@pfizer.com
Sangamo Media Contact: Aron Feingold 510-970-6000, x421
afeingold@sangamo.com
Sangamo Investor Contact: McDavid Stilwell 510-970-6000, x219
mstilwell@sangamo.com
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