Ultragenyx Pharmaceutical has dosed the first participant in Aurora, a study evaluating GTX-102 (apazunersen) for angelman syndrome (AS).
The rare, neurogenetic condition results from the loss of function of the maternally inherited UBE3A gene allele.
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The company stated that Aurora will broaden the treated population to include younger and older participants and individuals with non-deletion genotypes not part of the Phase III Aspire trial, which focuses on aged four to 17 years with full maternal ubiquitin-protein ligase E3A (UBE3A) gene deletion.
The Aurora study will enrol 60 subjects aged one to under 65 years worldwide, including countries and regions not represented in the Aspire Phase III trial.
Cohorts A, B, and C of the trial are single arm. Cohort D has a 2:1 randomisation to GTX-102 or a no treatment group, with the latter switching to GTX-102 dosing at week 24.
Each cohort has a 48-week efficacy assessment period, after which long-term extension options exist.
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By GlobalDataUltragenyx chief medical officer Eric Crombez said: “The open-label basket design of the Aurora study will enable us to efficiently evaluate the safety and efficacy of GTX-102 in younger and older patients with Angelman syndrome and those with other genotypes.
“While the Aspire study population represents the largest group of Angelman patients, we recognise the importance of bringing this potential new treatment to all patients, across genotypes and ages, as quickly as possible.”
As of July 2025, Aspire study enrolment concluded with 129 subjects aged four to 17 years across 28 global sites. Outcomes are anticipated during the second half of 2026.
GTX-102 is an investigational antisense oligonucleotide (ASO) therapy that is administered intrathecally and designed to inhibit and target UBE3A-AS.
It holds FDA rare paediatric disease, orphan drug, breakthrough therapy, and fast track designations, as well as orphan and PRIME designations from the European Medicines Agency (EMA).
