Taysha Gene Therapies has discontinued its TSHA-120 development for gene therapy following a Type C meeting with the US Food and Drug Administration (FDA).
The FDA requested adjustments to the trial design when laying out the registrational path for TSHA-120. The recommended changes include the trial design to be a randomised, double-blind, placebo-controlled trial with patients matched for multiple prognostic factors and long-term follow-up to reduce any potential bias.
The news led to an over 10% drop in Taysha stock in today's pre-market trading. In addition, Astellas Gene Therapies, Taysha’s partner for developing TSHA-120, chose not to exercise its right to obtain an exclusive licence for the drug.
TSHA-120 is a gene therapy being investigated for the treatment of giant axonal neuropathy (GAN), a rare peripheral and central nervous system disorder.
The drug development pathway has been a turbulent one. Last year, the FDA wanted Taysha to amend its primary endpoint to address the heterogeneity of disease progression in GAN. In June, the company submitted new data including the natural history and interventional trial comparing functional and biological measurements against a disease progression model (DPM) ahead of the Type C meeting.
Taysha CEO and chairman Sean P Nolan said: “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 [biologics licence application] submission in this ultra-rare neurodegenerative disease.
“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.”
The company was quick to point out that it is shifting its focus to its other gene therapy programme, TSHA-102, for treating Rett syndrome, a rare neurodevelopment disorder. The drug is currently in Phase I/II of clinical development.
Taysha revised its cash value projections following the trial termination and expected its cash runway to last into Q4 2025. The company has a market cap of $609.49m.
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