{"id":766601,"date":"2023-06-28T08:03:48","date_gmt":"2023-06-28T12:03:48","guid":{"rendered":"https:\/\/www.marketnewsdesk.com\/index.php\/taysha-gene-therapies-provides-clinical-updates-for-investigational-programs-tsha-120-in-giant-axonal-neuropathy-gan-and-tsha-102-in-rett-syndrome-at-rd-day\/"},"modified":"2023-06-28T08:03:48","modified_gmt":"2023-06-28T12:03:48","slug":"taysha-gene-therapies-provides-clinical-updates-for-investigational-programs-tsha-120-in-giant-axonal-neuropathy-gan-and-tsha-102-in-rett-syndrome-at-rd-day","status":"publish","type":"post","link":"https:\/\/www.marketnewsdesk.com\/index.php\/taysha-gene-therapies-provides-clinical-updates-for-investigational-programs-tsha-120-in-giant-axonal-neuropathy-gan-and-tsha-102-in-rett-syndrome-at-rd-day\/","title":{"rendered":"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"},"content":{"rendered":"
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\n 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<\/em>\u00a0<\/p>\n

\n 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<\/em>\n <\/p>\n

\n 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<\/em>\u00a0<\/p>\n

\n Detailed updates will be presented at virtual R&D Day today at 10:00 AM ET<\/em>\u00a0<\/p>\n

DALLAS, June 28, 2023 (GLOBE NEWSWIRE) — \u00a0Taysha Gene Therapies, Inc.\u00a0(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<\/a> section of Taysha\u2019s website.<\/p>\n

\u201cLate 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,\u201d said Sean P. Nolan, Chairman and Chief Executive Officer of Taysha. \u201cGiven 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.\u201d\u00a0<\/p>\n

Mr. Nolan continued, \u201cWe believe the new analyses may help support an approval pathway for TSHA-120 for the treatment of GAN. Our newly developed disease progression model demonstrates predictable and homogenous disease progression in classic GAN, which in our view supports the use of natural history data as an external control. Additionally, we identified objective functional, electrophysiological and biological measurements that demonstrated a clinically meaningful treatment effect, which is also accompanied by over seven years of clinical data supporting the safety profile. We\u2019ve requested a formal FDA meeting to discuss these new developments to support a potential regulatory path forward for TSHA-120. We expect the meeting to take place in the third quarter of this year.\u201d\u00a0<\/p>\n

\u201cFor our TSHA-102 program in Rett syndrome, we are encouraged by the initial clinical observations of the first adult patient recently dosed in the REVEAL Phase 1\/2 trial,\u201d said Sukumar Nagendran, M.D., President, and Head of R&D. \u201cWe look forward to providing further clinical updates on the safety and efficacy observations for the first patient early in the third quarter of this year, following the required IDMC adjudication of the initial clinical data. Subsequent REVEAL trial updates will be provided quarterly, thereafter. We remain on track to submit a CTA to the UK MHRA in pediatric patients in mid-2023 and to submit an IND application to the FDA in the second half of 2023.\u201d\u00a0<\/p>\n

\n Key R&D Day Highlights<\/strong>\n <\/p>\n

\n TSHA-120:<\/strong> a self-complimentary intrathecally delivered AAV9 gene therapy being evaluated in an open-label, dose-escalation, non-randomized Phase 1\/2 trial for GAN, an ultra-rare inherited genetic neurodegenerative disorder with no approved treatments.<\/em><\/p>\n