- U.S. Food and Drug Administration (FDA) provides a clear
regulatory pathway to Accelerated Approval for isaralgagene
civaparvovec using data from ongoing Phase 1/2 STAAR study,
avoiding requirement for additional registrational study and
accelerating estimated time to potential approval by approximately
three years.
- FDA confirms that estimated glomerular filtration rate (eGFR)
slope data at one year across all Phase 1/2 patients can serve as
primary basis for approval under Accelerated Approval pathway.
- Data to support Accelerated Approval pathway available in
first half of 2025, with a potential Biologics License Application
(BLA) submission expected in the second half of 2025.
Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine
company, today announced the outcome of a recent successful
interaction with the U.S. FDA, providing a clear regulatory pathway
to Accelerated Approval for isaralgagene civaparvovec, or ST-920,
its wholly owned gene therapy product candidate for the treatment
of Fabry disease.
The FDA has agreed in a Type B interaction that data from the
ongoing Phase 1/2 STAAR study can serve as the primary basis for
approval under the Accelerated Approval Program, using eGFR slope
at 52 weeks across all patients as an intermediate clinical
endpoint. The complete dataset to support an Accelerated Approval
pathway will be available in the first half of 2025. This approach
unlocks a potential BLA submission in the second half of 2025,
three years ahead of previous estimates, and avoids the requirement
for an additional registrational study to establish clinical
efficacy.
Sangamo engaged with the FDA on alternative pathways to
potential approval following analysis of clinical data from the
Phase 1/2 STAAR study showing encouraging safety and efficacy data,
including promising preliminary evidence of improved kidney
function. Renal manifestations, such as proteinuria or a decreased
glomerular filtration rate, occur early in life in almost all male,
and in many female, patients with Fabry disease, and can lead to
end-stage renal disease and early death. In the 18 male and female
patients treated with isaralgagene civaparvovec with more than one
year of follow-up data, statistically significant improvements were
observed in both mean and median eGFR levels, resulting in a
positive annualized eGFR slope. Based on these latest data, the FDA
agreed that eGFR slope at 52 weeks can serve as an intermediate
clinical endpoint to support a potential Accelerated Approval. The
FDA also advised that eGFR slope at 104 weeks may be assessed to
verify clinical benefit.
“Fabry is a debilitating disease, for which there is a serious
unmet medical need,” said Sandy Macrae, Chief Executive Officer of
Sangamo. “I strongly believe in the potential for ST-920 to
alleviate many manifestations of Fabry disease and am delighted to
have a clear regulatory pathway that could bring this treatment to
patients significantly sooner than originally anticipated”.
Dosing was completed in the Phase 1/2 STAAR study in April 2024,
with a total of 33 patients dosed. The longest treated patient
recently achieved four years of follow-up. The 18th and final
patient who started the study on Enzyme Replacement Therapy (ERT)
was successfully withdrawn from ERT in September 2024, and all 18
patients remain off ERT as of today. The 52-week eGFR slope data
from all enrolled patients in the Phase 1/2 STAAR study will be
available in the first half of 2025. A potential BLA submission is
anticipated in the second half of 2025.
Sangamo has begun to execute BLA readiness activities for
isaralgagene civaparvovec, while continuing to advance ongoing
business development discussions with potential collaboration
partners.
About the STAAR Study
The Phase 1/2 STAAR study is a global open-label, single-dose,
dose-ranging, multicenter clinical study designed to evaluate the
safety and tolerability of isaralgagene civaparvovec, or ST-920, a
gene therapy product candidate in patients with Fabry disease.
Isaralgagene civaparvovec requires a one-time infusion without
preconditioning. The STAAR study enrolled male and female patients
who are on ERT, are ERT pseudo-naïve (defined as having been off
ERT for six or more months), or who are ERT-naïve. The FDA has
granted Orphan Drug, Fast Track and RMAT designations to
isaralgagene civaparvovec, which has also received Orphan Medicinal
Product designation and PRIME eligibility from the EMA and
Innovative Licensing and Access Pathway from U.K. Medicines and
Healthcare products Regulatory Agency.
About Fabry Disease
Fabry disease is a lysosomal storage disorder caused by
mutations in the galactosidase alpha gene (GLA), which leads to
deficient alpha-galactosidase A (α-Gal A) enzyme activity, which is
necessary for metabolizing globotriaosylceramide (Gb3). The buildup
of Gb3 in the cells can cause serious damage to vital organs,
including the kidney, heart, nerves, eyes, gut and skin. Symptoms
of Fabry disease can include decreased or absent sweat production,
heat intolerance, angiokeratoma (skin blemishes), vision problems,
kidney disease, heart failure, gastrointestinal disturbance, mood
disorders, neuropathic pain and tingling in the extremities.
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 in 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
our current expectations. These forward-looking statements include,
without limitation, statements relating to: the safety and efficacy
and therapeutic potential of isaralgagene civaparvovec; the
potential for isaralgagene civaparvovec to qualify for the FDA’s
Accelerated Approval program, including the adequacy of data
generated in the Phase 1/2 STAAR study to support any such
approval; expectations concerning the availability of additional
data to support a potential BLA submission for isaralgagene
civaparvovec, and the timing of such submission; the potential to
accelerate the expected timeline to approval and bring isaralgagene
civaparvovec to patients sooner than previously expected; the
anticipated advancement of isaralgagene civaparvovec to
registration, including Sangamo’s plans to seek a potential
collaboration partner; 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
our lack of capital resources to obtain regulatory approval for and
commercialize our product candidates in a timely manner or at all,
including our ability to secure a partnership; the uncertain timing
and unpredictable nature of clinical trial results, including the
risk that the therapeutic effects observed in the latest
preliminary clinical data from the Phase 1/2 STAAR study will not
be durable in patients and that final clinical trial data from the
study will not validate the safety and efficacy of isaralgagene
civaparvovec, including that the 52-week data from the Phase 1/2
STAAR study will not support a BLA submission and/or that the
104-week data from such study will not verify the clinical benefit
of isaralgagene civaparvovec or support FDA approval, and that the
patients withdrawn from ERT will remain off ERT; our need for
substantial additional funding to execute our operating plan and to
continue to operate as a going concern; the effects of
macroeconomic factors or financial challenges on the global
business environment, healthcare systems and our business and
operations; the research and development process; the unpredictable
regulatory approval process for product candidates across multiple
regulatory authorities; the potential for technological
developments that obviate technologies used by Sangamo; our
reliance on collaborators and our potential inability to secure
additional collaborations; and our ability to achieve expected
future financial performance.
There can be no assurance that we and our current or potential
future 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 our collaborators. These risks and uncertainties are described
more fully in our Securities and Exchange Commission, or SEC,
filings and reports, including in our Annual Report on Form 10-K
for the year ended December 31, 2023, as supplemented by our
Quarterly Report on Form 10-Q for the quarter ended June 30, 2024,
each filed with the SEC, and future filings and reports that
Sangamo makes from time to time with the SEC. Forward-looking
statements contained in this announcement are made as of this date,
and we undertake no duty to update such information except as
required under applicable law.
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version on businesswire.com: https://www.businesswire.com/news/home/20241022733270/en/
Investor Relations & Media
Inquiries Louise Wilkie ir@sangamo.com media@sangamo.com
Grafico Azioni Sangamo Therapeutics (NASDAQ:SGMO)
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Grafico Azioni Sangamo Therapeutics (NASDAQ:SGMO)
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