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Taysha Gene Therapies Provides Clinical Updates for Investigational Programs TSHA-120 in Giant Axonal Neuropathy (GAN) and TSHA-102 in Rett Syndrome at R&D Day
Company views that results of comprehensive data analysis of TSHA-120 and development of disease progression model (DPM) address U.S. Food and Drug
About this update from Taysha Gene Therapies, Inc.
[{"type":"text","content":"Company views that results of comprehensive data analysis of TSHA-120 and development of disease progression model (DPM) address U.S. Food and Drug Administration (FDA) feedback regarding the effort-dependent nature of MFM32 as primary endpoint in an unblinded study and heterogeneity of GAN; Taysha plans to review potential regulatory pathway for TSHA-120 at a formal meeting with the FDA expected in Q3 2023 New GAN analysis identified multiple functional, electrophysiological and biological measurements that demonstrate a clinically meaningful and objective measurement of TSHA-120 treatment effect on disease progression Encouraging initial clinical observations seen in the first adult patient with Rett syndrome recently dosed with TSHA-102 in REVEAL Phase 1/2 trial; safety and efficacy update and Independent Data Monitoring Committee (IDMC) approval to dose second patient expected in early Q3 2023 Detailed updates will be presented at virtual R&D Day today at 10:00 AM ET DALLAS, June 28, 2023 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS), announced new data analyses for TSHA-120 in GAN and initial clinical observations for TSHA-102 in Rett syndrome. Taysha will host a virtual R&D Day today at 10:00 AM ET to discuss these updates. The webcast link can be accessed on the Events and Presentations section of Taysha’s website. “Late last year, the company submitted and discussed with the FDA a subset of available evidence supporting the potential therapeutic benefit and safety profile for TSHA-120 in patients with GAN, an ultra-rare disease with currently no approved treatments. FDA feedback included the need to address the heterogeneity of disease progression in GAN and the effort-dependent nature of MFM32 as a primary endpoint, considering the unblinded study design,” said Sean P. Nolan, Chairman and Chief Executive Officer of Taysha. “Given the FDA also indicated it is open to regulatory flexibility in a controlled trial setting and willing to consider alternative study designs, we undertook an extensive analysis of the totality of data available to determine a feasible regulatory path forward for TSHA-120.” Mr. Nolan continued, “We be...