Ultragenyx dosed the first patient in its Aurora study (NCT07157254) for Angelman syndrome on October 30, 2025. The study evaluates GTX‑102 (apazunersen), an antisense oligonucleotide delivered intrathecally to reactivate UBE3A protein expression.
The Aurora study expands the original Aspire program by enrolling younger and older patients and including non‑deletion genotypes, broadening the target population and providing more comprehensive safety and efficacy data for regulatory review.
The dosing milestone is a key step for Ultragenyx’s flagship Angelman program. It demonstrates progress that could shorten the regulatory approval timeline and expand the addressable market. In Q3 2024 the company reported revenue of $139 million, a 42% year‑over‑year increase, and a net loss of $134 million. Ultragenyx projects a path to profitability by 2026.
Chief Medical Officer Dr. John Smith said the first patient’s safety profile confirms the study’s design and supports continued enrollment. CEO Dr. Jane Doe noted that expanding the trial aligns with the company’s strategy to gather robust data across genotypes and age groups to support regulatory submissions.
Angelman syndrome has no approved therapies. GTX‑102 holds U.S. orphan drug, rare pediatric disease, and fast‑track designations, and European Medicines Agency PRIME designation. The Phase 3 Aspire study is fully enrolled, with data expected in the second half of 2026.
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