Taysha Says

Taysha Says FDA Grants Breakthrough Therapy Designation To In Rett Syndrome

Biotechnology company Taysha Gene Therapies, Inc. (TSHA) announced the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to TSHA-102, an intrathecally delivered AAV9 gene therapy with disease modifying potential, for the treatment of Rett syndrome.

Additionally, the Company announced that it has finalized alignment with the FDA on the REVEAL pivotal trial protocol and statistical analysis plan (SAP) that are intended to support the planned Biologics License Application (BLA) submission for TSHA-102, following the resolution of remaining clinical and statistical queries.

The Breakthrough Therapy designation and finalized FDA alignment on the pivotal trial protocol and SAP were based on the FDA's review of positive clinical evidence from Part A of the REVEAL Phase 1/2 adolescent/adult and pediatric trials.

Clinical evidence supporting Breakthrough Therapy designation for TSHA-102 included previously disclosed Part A REVEAL clinical data demonstrating a generally well-tolerated safety profile and a 100% response rate post-TSHA-102 for the pivotal trial primary endpoint of the gain/regain of = one defined developmental milestone, with <6.7% likelihood of being achieved without treatment based on natural history data.

These findings were corroborated by dose-dependent improvements in multiple outcome measures, including Revised Motor Behavior Assessment (R-MBA) and Clinician Global Impression - Improvement (CGI-I).