Taysha Gene Therapies Announces Pivotal Part B Trial Design Details for TSHA-102 in Rett Syndrome Enabled by IRSF Natural History Data and Positive Clinical Data from Part A of the REVEAL Adult/Adolescent and Pediatric Trials Evaluating TSHA-102
05-28-2025 | PDF Version
Natural history data analysis established patients ≥ six years of age are in developmental plateau, with a ~0% likelihood of gaining/regaining developmental milestones across the core functional domains of Rett syndrome
Written alignment from FDA supports single-arm, open label pivotal trial with primary endpoint of developmental milestone gain/regain in the developmental plateau population (≥ 6 years, intend N=15) with each patient serving as their own control
100% of patients in REVEAL Part A (N=10, 6-21 years) gained/regained ≥ one developmental milestone post-TSHA-102 with concordant improvements across multiple outcome measures; high dose consistently outperformed low dose, with dose-dependent effects deepening over time
No treatment-related SAEs or DLTs following low dose and high dose of TSHA-102
FDA advised the Company to submit pivotal Part B trial protocol and SAP as an amendment to the IND application, which is expected to occur in the current quarter; pivotal trial initiation activities anticipated in Q3 2025
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