Taysha Gene Therapies Halts its TSHA-120 Program for Giant Axonal Neuropathy Treatment
21 September 2023
Collaborations, Mergers & Acquisitions
Due to the challenges with the feasibility study design for a potential BLA submission, Taysha is discontinuing the development of TSHA-120 in GAN following the Type C meeting feedback from the FDA. To potentially enable further program development, Taysha will pursue external strategic options for the TSH-120 program.
Taysha Gene Therapies, a clinical-stage gene therapy company, has announced that because of the Type C meeting feedback from the FDA, the Company will be discontinuing the development of its TSHA-120 program in evaluation for the treatment of giant axonal neuropathy. Furthermore, Astellas has decided not to exercise its option to obtain an exclusive license to TSHA-120 under the Option Agreement between Taysha and Astellas.
Richard Wilson, Senior Vice President, Primary Focus Lead of Genetic Regulation of Astellas, stated:
“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.”
Primarily, the Company is focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system.
“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 CEO of Taysha.
In 2022, Taysha submitted and reviewed with the FDA in a Type B end-of-Phase II meeting subset of available evidence from a Phase I/II clinical trial investigating TSHA-120 for the treatment of GAN, which was initiated by the NIH. FDA feedback emphasized the need to address the heterogeneity of disease progression in GAN and the effort-dependent nature of MFM32 as a primary outcome in an unblinded study.
“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,” continued Nolan.
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. Acknowledged in 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 consider the potential of bias.
Prioritization of the program’s strategy could reduce operating expenses and is anticipated to extend the cash runway into the fourth quarter of 2025 to support the continued development of TSHA-102 in evaluation for Rett syndrome.
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