designer491/iStock via Getty Images Shares of Taysha Gene Therapies ( NASDAQ: TSHA ) slipped in the premarket Tuesday after the gene therapy developer announced data from its lead clinical program, TSHA-102, in patients with Rett syndrome, a rare neurodevelopmental disorder. The readout comprised long-term results from Taysha’s ( TSHA ) REVEAL Phase 1/2 adolescent and adult trial and initial data from the company’s REVEAL Phase 1/2 pediatric trial. Citing up to 52 weeks of data from the first two adult participants who received a low TSHA-102 dose, the company said there were no dose-limiting toxicities, and the study drug was well-tolerated with no serious adverse events ((SAEs)).
Meanwhile, data from the pediatric trial suggests that the first two patients who received TSHA-102 at a lower dose generally well-tolerated the drug with no SAEs up to 22 weeks from starting the therapy. The second patient experienced two SAEs, which Taysha ( TSHA ) said were not related to the experimental drug. Efficacy data up to 52 weeks from both trials suggest improvements across multiple clinical measures, including those related to motor skills and communication/socialization.
Taysha ( TSHA ) said REVEAL’s data monitoring committee has cleared its request to advance the pediatric trial to a high-dose cohort. Its dosing is expected to begin in Q3 2024, and initial data will be available in H2 2024. More on Taysha Gene Therapies Taysha Gene Therapies, Inc.
(TSHA) Q1 2024 Earnings Call .
