Taysha Gene Therapies Provides Update on TSHA-120 Program in Giant Axonal Neuropathy (GAN)
September 19 2023 - 4:01PM
Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a clinical-stage gene
therapy company focused on developing and commercializing AAV-based
gene therapies for the treatment of monogenic diseases of the
central nervous system (CNS), today announced that subsequent to
the receipt of Type C meeting feedback from the United States
(U.S.) Food and Drug Administration (FDA) regarding a
registrational path for TSHA-120, the Company will discontinue the
development of its TSHA-120 program in evaluation for the treatment
of giant axonal neuropathy (GAN). Further, Taysha announced that
Astellas Gene Therapies, Inc. (f/k/a Audentes Therapeutics, Inc.
(d/b/a Astellas Gene Therapy)) (Astellas) has elected not to
exercise its option to obtain an exclusive license to TSHA-120
under the Option Agreement between Astellas and Taysha.
“We believe we have made significant progress in demonstrating
the therapeutic potential of TSHA-120 and identifying a potential
registrational path. Following FDA feedback, we have made the
decision to discontinue further development of the program due to
challenges related to the feasibility of the study designs to
support a potential BLA submission in this ultra-rare
neurodegenerative disease,” said Sean P. Nolan, Chairman and Chief
Executive Officer of Taysha. “I want to express our gratitude to
the patients and families who participated in the trial, the GAN
community, and the National Institutes of Health (NIH) for their
partnership in establishing the foundation for a potential
treatment option in GAN. We plan to pursue external strategic
options for TSHA-120 that may enable further development of
TSHA-120 and help patients with this devastating disease.”
“This strategic program prioritization is expected to extend our
cash runway into the fourth quarter of 2025 to support the
continued clinical development of TSHA-102 in Rett syndrome, a rare
neurodevelopmental disorder with no approved treatments that target
the genetic root cause of the disease. We remain focused on
continuing to evaluate the therapeutic potential of TSHA-102 in our
ongoing REVEAL Phase 1/2 trial in adults and our planned pediatric
trial,” concluded Mr. Nolan.
Richard Wilson, Senior Vice President, Primary Focus Lead of
Genetic Regulation of Astellas, added, “While Astellas has declined
to exercise its option for the GAN program, we remain focused on
the needs of patients impacted by devastating diseases and look
forward to continuing our relationship with Taysha.”
In 2022, Taysha submitted and reviewed with the FDA in a Type B
end-of-Phase 2 meeting, a subset of available evidence from a Phase
1/2 clinical trial investigating TSHA-120 for the treatment of GAN,
which was initiated by the NIH. FDA feedback included the need to
address the heterogeneity of disease progression in GAN and the
effort-dependent nature of MFM32 as a primary endpoint in an
unblinded study. To further discuss a potential regulatory path
forward for TSHA-120, Taysha submitted a new comprehensive analysis
of the totality of data from the natural history and interventional
trial comparing functional and biological measurements against a
Disease Progression Model (DPM) as part of a Type C meeting request
to the FDA in June 2023.
FDA Type C meeting feedback indicated that the FDA continues to
recommend a randomized, double-blind, placebo-controlled trial as
the optimal path to demonstrate efficacy in TSHA-120. Among other
areas of feedback, the FDA also provided a potential path for a
single-arm trial with an external control group matched with to-be
treated patients by multiple prognostic factors and recommended
longer term follow up to account for potential bias.
About Taysha Gene
Therapies Taysha
Gene Therapies (Nasdaq: TSHA) is on a mission to eradicate
monogenic CNS disease. With a singular focus on developing curative
medicines, we aim to rapidly translate our treatments from bench to
bedside. We have combined our team’s proven experience in gene
therapy drug development and commercialization with the world-class
UT Southwestern Gene Therapy Program. Together, we leverage our
fully integrated platform with a goal of dramatically improving
patients’ lives. More information is available
at www.tayshagtx.com.
Forward-Looking StatementsThis press release
contains forward-looking statements within the meaning of the
Private Securities Litigation Reform Act of 1995. Words such as
“anticipates,” “believes,” “expects,” “intends,” “projects,”
“plans,” and “future” or similar expressions are intended to
identify forward-looking statements. Forward-looking statements
include statements concerning the potential of our product
candidates to positively impact quality of life and alter the
course of disease in the patients we seek to treat, our research,
development and regulatory plans for our product candidates, the
potential for these product candidates to receive regulatory
approval from the FDA or equivalent foreign regulatory agencies,
and whether, if approved, these product candidates will be
successfully distributed and marketed, our ability to pursue
strategic options for TSHA-120, anticipated cost savings due to the
discontinuation of development of TSHA-120, our expected cash
runway, the potential benefits of Taysha’s collaboration with
Astellas and the potential for Astellas to exercise any of the
options granted to it by Taysha. Forward-looking statements are
based on management’s current expectations and are subject to
various risks and uncertainties that could cause actual results to
differ materially and adversely from those expressed or implied by
such forward-looking statements. Accordingly, these forward-looking
statements do not constitute guarantees of future performance, and
you are cautioned not to place undue reliance on these
forward-looking statements. Risks regarding our business are
described in detail in our Securities and Exchange Commission
(“SEC”) filings, including in our Annual Report on Form 10-K for
the full-year ended December 31, 2022, which is available on the
SEC’s website at www.sec.gov. Additional information will be made
available in other filings that we make from time to time with the
SEC. Such risks may be amplified by the impacts of the COVID-19
pandemic. These forward-looking statements speak only as of the
date hereof, and we disclaim any obligation to update these
statements except as may be required by law.
Company Contact:Hayleigh Collins Director, Head
of Corporate CommunicationsTaysha Gene Therapies,
Inc.hcollins@tayshagtx.com
Media Contact:Carolyn HawleyCanale
Communicationscarolyn.hawley@canalecomm.com
Taysha Gene Therapies (NASDAQ:TSHA)
Historical Stock Chart
From Jun 2024 to Jul 2024
Taysha Gene Therapies (NASDAQ:TSHA)
Historical Stock Chart
From Jul 2023 to Jul 2024