- First patient was dosed in pivotal Phase 3
AFFINE study in October 2020
Pfizer Inc. (NYSE: PFE) and Sangamo Therapeutics, Inc. (Nasdaq:
SGMO), a genomic medicines company, today announced updated
follow-up data from the Phase 1/2 Alta study of giroctocogene
fitelparvovec (SB-525 or PF-07055480), an investigational gene
therapy for patients with severe hemophilia A. These data are being
presented today at the 62nd American Society for Hematology Annual
meeting taking place virtually from December 5th - 8th. The oral
presentation slides, which include follow-up data up to 85 weeks
for the longest treated patient, are available on Sangamo’s website
in the Investors and Media section under Events and
Presentations.
This press release features multimedia. View
the full release here:
https://www.businesswire.com/news/home/20201207005251/en/
All five patients in the high dose 3 x 1013 vg/kg cohort have
had at least one year of follow-up and showed sustained factor VIII
(FVIII) activity levels, with a group median FVIII activity of
56.9% and a group geometric mean FVIII activity of 70.4% via
chromogenic assay from week 9 to 52. Steady-state FVIII activity
was achieved for all patients in the 3 x 1013 vg/kg cohort within 9
weeks of treatment with giroctocogene fitelparvovec, with no
bleeding events and no FVIII infusions (beyond 3 weeks
post-infusion) within the first year. As of the cutoff date of
August 31, 2020, one patient had one target joint bleed requiring
FVIII therapy, occurring after week 52.
“It is promising to see how quickly all five patients in the 3 x
1013 vg/kg cohort achieved steady-state FVIII activity levels, with
no bleeding events and no factor usage within the first year and
only one target joint bleed after 52 weeks,” said Andrew D.
Leavitt, MD, Professor of Medicine, University of California, San
Francisco, CA, and investigator of the Alta and AFFINE studies.
“Our focus now is to confirm these exciting findings in the Phase 3
study, and to gather long-term data by following these patients and
others in the Phase 3 study over a longer period of time.”
Giroctocogene fitelparvovec was generally well tolerated. As
previously reported, one patient in the 3 x 1013 vg/kg dose cohort
had a treatment-related serious adverse event of hypotension (grade
3) and fever (grade 2), with symptoms of headache and tachycardia,
which occurred six hours post-infusion with giroctocogene
fitelparvovec, and which fully resolved within 24 hours. No other
treatment-related serious adverse events were reported as of the
cutoff date. Among the five patients in the 3 x 1013 vg/kg dose
cohort, four received corticosteroids for liver enzyme (alanine
aminotransferase, ALT) elevations. Three patients had subsequent
ALT elevations that responded to corticosteroids. All episodes of
ALT elevations fully resolved with oral corticosteroids, and as of
the cutoff date no participants were on corticosteroids and no
corticosteroid use has been initiated after week 52.
“We continue to be encouraged by the findings from this Phase
1/2 study, which now include durable factor VIII expression through
one year of follow-up, and we look forward to continuing to follow
these patients,” said Seng Cheng, Senior Vice President and Chief
Scientific Officer of Pfizer’s Rare Disease Research Unit. “With
the first patient dosed in the Phase 3 AFFINE study in October
2020, we are on track for a readout from this pivotal Phase 3 trial
in 2022, which will allow us to better assess the potential of our
gene therapy across a larger sample size.”
“These latest results demonstrate that this gene therapy may
bring clinical benefit to patients and has the potential to serve
as an alternative to the burdensome standard of care for patients
with hemophilia A,” said Bettina Cockroft, M.D., M.B.A, Chief
Medical Officer of Sangamo. “We look forward to continuing to
support our collaboration partners at Pfizer as they conduct the
Phase 3 AFFINE study and assess the full potential of this
promising therapy.”
Pfizer and Sangamo plan to present further follow-up data from
the Alta study when all five patients in the 3 x 1013 vg/kg dose
cohort have been followed for at least two years.
About the Alta study
The Phase 1/2 Alta study is an open-label, dose-ranging,
multicenter clinical trial designed to assess the safety and
tolerability of giroctocogene fitelparvovec in patients with severe
hemophilia A. The mean age of the 11 male patients assessed across
four dose cohorts (9x1011 vg/kg - 2 patients, 2 x 1012 vg/kg - 2
patients, 1x1013 vg/kg - 2 patients and 3 x 1013 vg/kg - 5
patients) is 30 years (range 18-47 years). After one year of
follow-up for all patients in the study, participants will be
assessed every 6 months until they enroll into a long-term
follow-up study.
About the AFFINE study
The Phase 3 AFFINE (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 annualized bleeding rate (ABR)
through 12 months following treatment with giroctocogene
fitelparvovec. This will be compared to ABR on prior FVIII
prophylaxis replacement therapy. The secondary endpoints include
FVIII activity level after the onset of steady state and through 12
months following infusion of giroctocogene fitelparvovec.
About giroctocogene fitelparvovec
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
collaboration agreement for the global development and
commercialization of gene therapies for hemophilia A between
Sangamo and Pfizer. In late 2019, Sangamo transferred the
manufacturing technology and the Investigational New Drug (IND)
application to 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 Pfizer Rare Disease
Rare diseases include some of the most serious of all illnesses
and impact 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 www.facebook.com/Pfizer.
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 genome regulation. For more
information about Sangamo, visit www.sangamo.com.
PFIZER DISCLOSURE NOTICE:
The information contained in this release is as of December 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 and the
anticipated timing and readout of the phase 3 clinical trial, 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.
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 therapeutic
potential of giroctocogene fitelparvovec (SB-525), including its
potential clinical benefit to patients with hemophilia A and its
potential as an alternative to the standard of care for patients
with hemophilia A, the anticipated readout from the Phase 3 AFFINE
study and the expected timing thereof, the plan and related
timelines for sharing additional clinical data 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 nature of clinical trial results, including the risks
that therapeutic effects observed in clinical trial results will
not be durable in patients and that final clinical trial data will
not validate the safety and efficacy of giroctocogene
fitelparvovec; reliance on results of early clinical trials, such
as the Phase 1/2 Alta study, which results are not necessarily
predictive of future clinical trial results, including the results
in the Phase 3 AFFINE study; 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 giroctocogene fitelparvovec; the potential
for Pfizer to terminate the giroctocogene fitelparvovec program or
to breach or terminate its collaboration agreement with Sangamo;
and the potential for Sangamo to fail to realize its expected
benefits of its collaboration with Pfizer, including the risk that
Sangamo may not earn any additional milestone or royalty payments
under 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 September 30,
2020. The information contained in this release is as of December
7, 2020, and Sangamo undertakes no duty to update forward-looking
statements contained in this release except as required by
applicable laws.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20201207005251/en/
Pfizer Media:
Steve Danehy 212.733.1538 Steven.danehy@pfizer.com
Pfizer Investor
Relations: Bryan
Dunn 212.733.8917
Bryan.dunn@pfizer.com
Sangamo Investor & Media
Relations Aron Feingold 628.252.7494
afeingold@sangamo.com
Sangamo Therapeutics (NASDAQ:SGMO)
Historical Stock Chart
From Mar 2024 to Apr 2024
Sangamo Therapeutics (NASDAQ:SGMO)
Historical Stock Chart
From Apr 2023 to Apr 2024