Ultragenyx announces completion of enrollment in Phase 1/2 trial of GTX-102

Ultragenyx Pharmaceutical announced the completion of patient enrollment in its Phase 1/2 clinical trial of GTX-102 for the treatment of pediatric patients with Angelman syndrome, AS. The dose-expansion cohorts have enrolled 53 patients for a total of 74 patients enrolled globally in the Phase 1/2 trial. GTX-102 is an investigational antisense oligonucleotide delivered via intrathecal administration and is designed to target and inhibit expression of UBE3A antisense transcript, UBE3A-AS. “With completion of enrollment in the Phase 1/2 trial, we remain on track to report results in the first half of 2024 from at least 20 expansion cohort patients on therapy for a minimum of 6 months. We are confident that the cumulative safety and efficacy data will allow for dose and endpoint selection to support our Phase 3 program,” said Eric Crombez, M.D., chief medical officer at Ultragenyx. “We appreciate the support of the Angelman community, including the patients, families and healthcare providers, as we urgently work together to develop a new treatment option that may be able to improve the quality of life of those impacted by this devastating disease.”