- U.S. Food and Drug Administration (FDA) advises that a single
study with up to 25 patients, in combination with confirmatory
evidence, may be acceptable pathway to Biologics License
Application (BLA) submission for isaralgagene civaparvovec, which
would significantly reduce anticipated complexity, cost and time to
potential approval.
- European Medicines Agency (EMA) granted priority medicines
(PRIME) eligibility to isaralgagene civaparvovec, which includes
enhanced regulatory support and scientific guidance.
- Sangamo is actively seeking a collaboration partner to advance
isaralgagene civaparvovec through potential registration and
commercialization.
Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine
company, today announced important U.S. and European regulatory
updates 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 D meeting that data from a single,
adequate, and well-controlled study may form the primary basis of
approval of a BLA for isaralgagene civaparvovec. The proposed study
would enroll up to 25 patients, both male and female, without the
need for a control arm. A head-to-head comparison with Enzyme
Replacement Therapy (ERT) is not part of the proposed study design
deemed acceptable by the FDA. This approach enables a potentially
more rapid, efficient and cost-effective pathway to BLA submission
than originally anticipated.
Additionally, the EMA has granted PRIME eligibility to
isaralgagene civaparvovec. PRIME is a program designed to enhance
support for the development of medicines that target an unmet
medical need and is intended to optimize development plans and
expedite review and approval processes so that these medicines may
reach patients as early as possible. Isaralgagene civaparvovec has
already received Orphan Medicinal Product designation from the EMA
as well as Orphan Drug, Fast Track and RMAT designations from the
FDA.
“The U.S. and European regulatory support for ST-920 and the
serious unmet medical need in Fabry Disease signal the important
role that ST-920 could play in improving the lives of Fabry
patients across the globe,” said Nathalie Dubois Stringfellow,
Ph.D., Chief Development Officer of Sangamo. “We are thankful for
the FDA’s support and alignment on a regulatory pathway that could
potentially deliver a new treatment option for Fabry disease
patients on an expedited, cost-effective timeline. Similarly, we
appreciate the support from the EMA and the opportunity to advance
our development plans in Europe. Fabry is a debilitating disease in
need of new medicines, and we are grateful that regulatory agencies
across geographies recognize this and support our proposed
development plans.”
Updated Phase 1/2 STAAR study data showing sustained clinical
benefit and a differentiated safety profile across 24 patients were
shared at the 20th Annual WORLDSymposiumTM in San Diego, CA on
Wednesday, February 7, 2024. A total of 29 patients have been
treated to date in the Phase 1/2 STAAR study. All 13 patients
withdrawn from ERT remain off ERT as of February 12, 2024.
Screening and enrollment are complete in the study and dosing of
the remaining enrolled patients is expected in the first half of
2024. Sangamo is deferring additional investments in planning for a
registrational trial until a collaboration partnership is
secured.
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 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.
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’s zinc finger epigenetic regulators are ideally
suited to potentially address devastating neurological disorders
and Sangamo’s capsid discovery platform is making progress toward
potentially expanding 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 Twitter/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 and commercial potential of isaralgagene
civaparvovec, the anticipated plans and timelines for conducting
our ongoing and potential future clinical trials and presenting
clinical data from our clinical trials, expectations regarding the
conclusion of dosing in our Phase 1/2 STAAR study, the anticipated
advancement of isaralgagene civaparvovec to late-stage development,
including Sangamo’s plans to seek a potential partner to proceed
with potential future registrational studies of isaralgagene
civaparvovec, the design of any potential future studies of
isaralgagene civaparvovec, the potential impact of FDA and EMA
feedback on the regulatory pathway for isaralgagene civaparvovec ,
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 fully develop, obtain regulatory approval for and
commercialize our product candidates, including our ability to
secure a partnership required to initiate a potential
registrational study of isaralgagene civaparvovec in a timely
manner or at all; 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, including as a result of the ongoing overseas conflict,
current or potential future bank failures, inflation and elevated
interest rates, on the global business environment, healthcare
systems and business and operations of Sangamo and our
collaborators, including the operation of clinical trials; the
research and development process, including the operation and
results of clinical trials and the presentation of clinical data;
the impacts of clinical trial delays, pauses and holds on clinical
trial timelines and commercialization of product candidates; 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, and that the patients withdrawn from ERT
will remain off ERT; 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 studies of our product
candidates; 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, 2022, as supplemented by our
Quarterly Report on Form 10-Q for the quarter ended September 30,
2023, 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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Investor Relations & Media
Inquiries Louise Wilkie ir@sangamo.com media@sangamo.com
Sangamo Therapeutics (NASDAQ:SGMO)
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