Taysha Gene Therapies, Inc. (NASDAQ: TSHA) witnessed a significant surge of 19.70% in its stock value on the US market, closing at $3.16, following the announcement of a clinical trial update. The update pertains to the progress of the TSHA-102 program aimed at treating Rett syndrome. The Independent Data Monitoring Committee (IDMC) greenlit Taysha’s request to escalate doses in the REVEAL Phase 1/2 trials, both for adolescents and adults and for pediatric patients.
In the REVEAL Phase 1/2 trials, TSHA-102, administered via lumbar intrathecal injection, is being evaluated for safety and efficacy in females aged 12 and above with Rett syndrome caused by MECP2 loss-of-function mutation. The recent approval from the IDMC allows Taysha to proceed with dose escalation earlier than anticipated, hastening the process of gathering crucial data for clinical development and regulatory planning.
Moreover, the expansion of the trial from Canada into the United States further broadens the demographic range of patients being studied, aligning with the company’s objective of assessing TSHA-102’s effectiveness across various ages and disease stages in Rett syndrome patients.
Taysha expressed optimism regarding the safety and efficacy data observed thus far with TSHA-102. They emphasized the significance of being able to dose the second patient in the pediatric trial and moving towards the high dose cohort in the adolescent and adult trial. This acceleration in the trial process is expected to expedite the formulation of the clinical development strategy.
The first-in-human, open-label, randomized, dose-escalation, and dose-expansion strategy is used in the REVEAL studies. After Part A has determined the maximum administered dosage (MAD) or maximum tolerated dose (MTD), Part B will include giving this dose for additional assessment.
The latest events highlight Taysha’s dedication to meeting the unmet needs of Rett syndrome patients and their families. Taysha hopes to provide hope to a greater range of individuals suffering from this crippling illness by advancing the clinical studies and broadening their scope.
