- Based on positive results from Phase 3 AFFINE
trial, which met primary and secondary endpoints, Sangamo plans to
explore all options to commercialize the asset, including seeking a
potential new collaboration partner
Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine
company, today announced it will regain development and
commercialization rights to giroctocogene fitelparvovec, an
investigational gene therapy product candidate for the treatment of
adults with moderately severe to severe hemophilia A that it has
co-developed with, and licensed to Pfizer Inc., following a
decision by Pfizer to terminate the global collaboration and
license agreement between the parties. Sangamo intends to explore
all options to advance the program, including seeking a potential
new collaboration partner. Pfizer has indicated to Sangamo that
this termination reflects its decision not to proceed with the
Biologics License Application (BLA) and Marketing Authorisation
Application (MAA) submissions for, or to pursue commercialization
of, giroctocogene fitelparvovec.
In July 2024, Pfizer announced positive topline results from the
Phase 3 AFFINE trial evaluating giroctocogene fitelparvovec that
demonstrated the trial met primary and key secondary objectives of
superiority compared to prophylaxis. On December 9, 2024, Pfizer
presented detailed AFFINE data in an oral presentation at the 66th
ASH Annual Meeting and Exposition, with the presentation selected
as part of the “Best of ASH” program, which highlights the most
cutting-edge science presented during the Annual Meeting. In
November 2024, Pfizer indicated to Sangamo that Pfizer was
discussing these data with regulatory authorities. Pfizer
previously indicated to Sangamo that anticipated BLA and MAA
submissions were expected to occur in early 2025.
“Giroctocogene fitelparvovec has demonstrated the potential to
be a life changing gene therapy treatment for hemophilia A
patients, and following positive results from the Phase 3 AFFINE
trial, we believe it is well positioned for regulatory submissions
and potential commercialization,” said Sandy Macrae, Chief
Executive Officer of Sangamo Therapeutics. “While we were surprised
and extremely disappointed by Pfizer’s decision to end our
collaboration so close to the anticipated BLA and MAA submissions,
especially given the compelling pivotal clinical trial data, we
appreciate their collaboration in leading a robust and successful
clinical development program and for advancing the asset to this
important stage. We are committed to exploring the optimal path
forward for this important treatment, including seeking the right
partner with the focus and understanding of the genomic medicine
commercial environment to bring this medicine to patients. In
parallel, we remain focused on advancing our wholly owned neurology
genomic medicine pipeline and progressing our Fabry gene therapy
program towards a potential BLA submission in the second half of
2025.”
The collaboration and license agreement with Pfizer will
terminate effective April 21, 2025, at which time Pfizer will be
required to transition the giroctocogene fitelparvovec program back
to Sangamo. All trial participants will continue to be monitored as
planned during the transition period.
Sangamo believes that its recently announced partnerships with
Genentech and Astellas, and advanced business development
discussions for its Fabry gene therapy program, will allow it to
chart a path forward for its neurology genomic medicine pipeline as
it prepares to initiate expected enrollment of patients in the
Phase 1/2 study of ST-503 for idiopathic small fiber neuropathy in
mid-2025, and file an anticipated Clinical Trial Authorisation
(CTA) submission for the prion disease program in Q4 2025, each
subject to securing adequate additional funding.
About the AFFINE Trial
The Phase 3 AFFINE (NCT04370054) trial is an open-label,
multicenter, single-arm trial to evaluate the efficacy and safety
of a single infusion of giroctocogene fitelparvovec in adult male
participants (n=75 dosed participants) with moderately severe to
severe hemophilia A. Trial participants included in the assessments
of the key endpoints of the primary efficacy analysis (n=50)
completed a minimum six months of routine FVIII replacement
prophylaxis therapy during the lead-in study (NCT03587116)
providing data to compare with post giroctocogene fitelparvovec
treatment.
In July 2024, Pfizer announced that the AFFINE trial achieved
its primary objective of non-inferiority, as well as superiority,
of total annualized bleeding rate (ABR) from Week 12 through at
least 15 months of follow up post-infusion compared with routine
Factor VIII (FVIII) replacement prophylaxis treatment. Following a
single 3e13 vg/kg dose, giroctocogene fitelparvovec demonstrated a
statistically significant reduction in mean total ABR compared to
the pre-infusion period (1.24 vs 4.73; one-sided p-value=0.0040).
Key secondary endpoints as defined by the trial protocol were met
and also demonstrated superiority compared to prophylaxis. In the
AFFINE trial, giroctocogene fitelparvovec was generally well
tolerated.
Giroctocogene fitelparvovec has been developed as part of a
collaboration agreement for the global development and
commercialization of gene therapies for hemophilia A between
Sangamo Therapeutics and Pfizer. In late 2019, Sangamo transferred
the manufacturing technology and the Investigational New Drug
application to Pfizer. Under the agreement, Pfizer assumed
responsibility for pivotal studies, any regulatory activities, and
potential global commercialization of giroctocogene
fitelparvovec.
About Hemophilia A
Hemophilia is an inherited, rare bleeding disorder that causes
people to bleed for longer than normal due to a deficiency of a
protein required for normal blood clotting, known as clotting
Factor VIII (FVIII) in hemophilia A. The severity of hemophilia is
determined by the amount of the 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 25 in every 100,000 male
births worldwide. Approximately 55-75% of males with hemophilia A
have a moderate to severe form of the disease. 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 a genomic medicine company dedicated to
translating ground-breaking science into medicines that transform
the lives of patients and families afflicted with serious
neurological diseases who do not have adequate or any treatment
options. Sangamo believes that its zinc finger epigenetic
regulators are ideally suited to potentially address devastating
neurological disorders and that its capsid discovery platform can
expand delivery beyond currently available intrathecal delivery
capsids, including the central nervous system. Sangamo’s pipeline
also includes multiple partnered programs and programs with
opportunities for partnership and investment. To learn more, visit
www.sangamo.com and connect with us on LinkedIn and X.
Forward-Looking Statements
This press release contains forward-looking statements regarding
Sangamo’s current expectations. These forward-looking statements
include, without limitation, statements relating to: Sangamo’s
plans to explore options to commercialize giroctocogene
fitelparvovec and advance the giroctocogene fitelparvovec program,
including through seeking a potential new collaboration partner;
the potential of giroctocogene fitelparvovec to be a life changing
gene therapy treatment for hemophilia A patients; Sangamo’s belief
that giroctocogene fitelparvovec is well positioned for regulatory
submissions and potential commercialization; the effects of the
termination of the collaboration and license agreement with Pfizer,
including with respect to the transition of giroctocogene
fitelparvovec back to Sangamo; expectations concerning Sangamo’s
Fabry program, including plans for a potential BLA submission and
the timing thereof and expectations concerning Sangamo’s ability to
secure a potential partnership for such program; Sangamo’s plans to
initiate enrollment of patients in the Phase 1/2 study of ST-503
for idiopathic small fiber neuropathy and the anticipated timing
thereof; Sangamo’s plans to submit a CTA for the prion disease
program and the anticipated timing thereof; Sangamo’s belief that
its recently announced partnerships with Genentech and Astellas,
and advanced business development discussions for its Fabry gene
therapy program, will allow it to chart a path forward for its
neurology genomic medicine pipeline; and other statements that are
not historical fact. These statements are not guarantees of future
performance and are subject to certain risks and uncertainties that
are difficult to predict. Factors that could cause actual results
to differ include, but are not limited to, risks and uncertainties
related to Sangamo’s lack of capital resources and need for
substantial additional funding to execute its operating plan and to
continue to operate as a going concern, including the risk that
Sangamo will be unable to obtain funding or partnerships or
additional collaboration partners necessary to advance its
preclinical and clinical programs and to otherwise operate as a
going concern, in which case Sangamo may be required to cease
operations entirely, liquidate all or a portion of its assets
and/or seek protection under the U.S. Bankruptcy Code; the
uncertain and costly research and development process; risks
related to Sangamo’s reliance on partners to advance its Fabry and
giroctocogene fitelparvovec programs, including the risks that
Sangamo may be unable to identify and engage any new potential
collaboration partners for such programs on commercially reasonable
terms or at all, and even if Sangamo is able to do so, such
potential partners may be unable to meaningfully advance such
programs, whether due to internal portfolio prioritization
decisions or otherwise; the risk that Sangamo will not realize the
benefits of, including receipt of potential payments under, its
collaboration agreements; the effects of macroeconomic factors or
financial challenges, including as a result of the ongoing overseas
conflicts, current or potential future bank failures, inflation and
high interest rates, on the global business environment, healthcare
systems and business and operations of Sangamo and its
collaborators; the uncertain timing and unpredictable nature of
clinical trial results, including the risk that therapeutic effects
in the Phase 3 AFFINE trial will not be durable in patients; the
unpredictable regulatory approval process for product candidates
across multiple regulatory authorities; reliance on results of
early clinical trials, which results are not necessarily predictive
of future clinical trial results, including the results of any
registrational trial of Sangamo’s product candidates; and the
potential for technological developments that obviate technologies
used by Sangamo. There can be no assurance that Sangamo and its
collaborators will be able to develop commercially viable products.
Actual results may differ materially from those projected in these
forward-looking statements due to the risks and uncertainties
described above and other risks and uncertainties that exist in the
operations and business environments of Sangamo and its
collaborators. These risks and uncertainties are described more
fully in Sangamo’s Securities and Exchange Commission, or SEC,
filings and reports, including in Sangamo’s Annual Report on Form
10-K for the year ended December 31, 2023, as supplemented by
Sangamo’s Quarterly Report on Form 10-Q for the quarter ended
September 30, 2024, and subsequent filings and reports that Sangamo
makes from time to time with the SEC. Forward-looking statements
contained in this press release are made as of this date, and
Sangamo undertakes no duty to update such information except as
required under applicable law.
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Investor Relations and Media
Inquiries Louise Wilkie ir@sangamo.com media@sangamo.com
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