Taysha Gene Therapies Reports First Quarter 2025 Financial Results and Provides Corporate Update

Core Insights - Taysha Gene Therapies has made significant regulatory and clinical progress with its TSHA-102 program for Rett syndrome, having reached alignment with the FDA on key elements of the pivotal Part B trial design [3][4] - The FDA has advised the company to submit the pivotal Part B trial protocol and statistical analysis plan as an amendment to the IND application, which is expected to expedite study initiation and registration [3][4] - TSHA-102 has shown to be generally well tolerated in patients, with no treatment-related serious adverse events or dose-limiting toxicities reported in the initial cohorts [4] Regulatory Progress - The company achieved alignment with the FDA on the pivotal Part B trial design for TSHA-102, following discussions under the Regenerative Medicine Advanced Therapy (RMAT) pathway [4] - The FDA's guidance allows Taysha to submit the trial protocol directly as an IND amendment, eliminating the need for a formal end-of-phase meeting, which may accelerate the process [3][4] Clinical Data - As of April 10, 2025, TSHA-102 has been administered to 10 patients (6 in the high dose cohort and 4 in the low dose cohort) across two REVEAL Phase 1/2 trials, with no serious adverse events or dose-limiting toxicities reported [4] - A comprehensive update on the pivotal trial design, natural history data analysis, and clinical data from the REVEAL trials is anticipated in Q2 2025 [4][13] Financial Highlights - For Q1 2025, research and development expenses were $15.6 million, down from $20.7 million in Q1 2024, primarily due to reduced manufacturing activities [7] - General and administrative expenses increased to $8.2 million in Q1 2025 from $7.1 million in Q1 2024, attributed to higher compensation and legal fees [8] - The net loss for Q1 2025 was $21.5 million, or $0.08 per share, compared to a net loss of $24.1 million, or $0.10 per share, in Q1 2024 [9][17] Cash Position - As of March 31, 2025, Taysha had $116.6 million in cash and cash equivalents, which is expected to support operations into Q4 2026 [9][19] Upcoming Events - Taysha will present three abstracts related to TSHA-102 at the 2025 International Rett Syndrome Foundation Scientific Meeting scheduled for June 9-11, 2025 [4][5]