Following Type C meeting feedback from the
Taysha will pursue external strategic options for the TSHA-120 program to potentially enable further program development
Strategic program prioritization will reduce operating expenses and is anticipated to extend cash runway into the fourth quarter of 2025 to support the continued development of TSHA-102 in evaluation for Rett syndrome
“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
“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
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
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.
This 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
Director, Head of Corporate Communications
Source: Taysha Gene Therapies, Inc.