Encouraging GTX-102 Trial Results in Angelman Syndrome Patients from Phase 1/2 Study by Ultragenyx
April 15, 2024 —In a recent study, patients with Angelman syndrome demonstrated significant improvements in various areas after treatment with GTX-102, a drug developed by Ultragenyx Pharmaceutical Inc. The Phase 1/2 clinical trial results indicated that the drug led to rapid enhancements in cognition, behavior, sleep, and motor function, with benefits either matching or surpassing those observed in earlier dose-escalation cohorts. Notably, the long-term data revealed that these improvements were sustained, offering hope for a condition that currently lacks an approved disease-modifying treatment.
The study's findings were presented in detail during a corporate event and are set to be discussed further at the 76th Annual American Academy of Neurology Meeting. The positive outcomes are particularly encouraging as they align with the experiences reported by families of the patients, suggesting that the improvements are not only statistically significant but also meaningful in the patients' daily lives.
The data from the Expansion Cohorts at Day 170 and the Dose-escalation Cohorts up to Day 758 showed consistent results across multiple domains, with the Multi-domain Responder Index (MDRI) analysis indicating a substantial overall response. The safety profile of GTX-102 was also reassuring, with no unexpected serious adverse events reported. Ultragenyx is planning to meet with the FDA and other health authorities to discuss the next steps, which include initiating a Phase 3 pivotal study. The company's commitment to developing therapies for rare genetic diseases is evident in its portfolio and the urgency it places on delivering effective treatments.
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