GeneTx, Ultragenyx Resume Angelman Syndrome Trial After FDA Lift
GeneTx Biotherapeutics and Ultragenyx Pharmaceutical have resumed a Phase 1/2 study, KIK-AS, testing GTX-102 in children and adolescents with Angelman syndrome. The FDA lifted the clinical hold after addressing safety concerns. The study aims to assess the safety, tolerability, and preliminary efficacy of GTX-102 in up to 20 patients aged 4-17.
The study will take place in the U.S., U.K., and Canada over the next several months. In the U.S., a dose-loading phase will involve eight treatment-naïve patients aged 4 to 7, split into an active treatment group and an age-matched control group. In the U.K. and Canada, about 12 patients will be split into two age groups, receiving different doses of GTX-102.
GTX-102 is an antisense oligonucleotide designed to block the UBE3A antisense transcript, reactivating the silent paternal copy of UBE3A in the brain. This aims to compensate for the missing maternal copy of the UBE3A gene, which causes Angelman syndrome. The study was previously placed on a clinical hold due to five patients experiencing serious, but reversible, muscle weakness after receiving the highest dose of GTX-102. The resumed study has a modified protocol, including a narrowed dose range and intrathecal administration.
GeneTx Biotherapeutics is responsible for dosing GTX-102 in participants in the U.S. The study aims to provide valuable data on the safety and efficacy of GTX-102 in treating Angelman syndrome. Results are expected to guide further development of the therapy.
