From GlobeNewswire: 2024-11-09 10:30:00

In Novato, California, Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced Phase 1/2 data supporting the Phase 3 Aspire study for GTX-102, an investigational treatment for Angelman syndrome. The data show improvements in cognition and other domains, confirming that the Phase 3 study is well-powered to establish efficacy. Enrollment for the Phase 3 study is expected to begin by the end of the year, with a focus on cognition and other key endpoints. The Phase 3 Aspire study will include approximately 120 patients with Angelman syndrome and aims to assess improvements in cognition and other domains over a 48-week period.

The Phase 1/2 data from the Dose Expansion Cohorts showed continued improvement at Week 48, with a mean change in cognitive scores exceeding the important difference threshold. Additionally, the key secondary endpoint of MDRI showed significant improvement in approximately 80% of patients. These findings confirm that the Phase 3 study is well-equipped to evaluate the efficacy of GTX-102 in improving cognition and other domains in patients with Angelman syndrome.

GTX-102, an investigational treatment for Angelman syndrome, has shown a consistent and acceptable safety profile as of the latest data cutoff. The Phase 3 Aspire study is expected to establish the efficacy of GTX-102 on cognition and other key endpoints, with data indicating strong power to detect treatment effects. For more information on Ultragenyx and GTX-102, visit the company’s website.



Read more at GlobeNewswire: Ultragenyx Presents Positive Update on GTX-102 Angelman