Ultragenyx Pharmaceutical Inc. announced that its Phase 3 Aspire study for apazunersen (GTX-102) in patients with Angelman syndrome did not meet its primary or key secondary endpoints. Following this outcome, the company will evaluate the future of the drug program and plans to implement significant expense reductions.
Key Details
- Trial Outcome: The study failed to demonstrate efficacy, not achieving its primary endpoint (change in Bayley-4 cognitive raw score) or its key secondary endpoint (net response in Multidomain Responder Index).
- Company Response: The company will re-evaluate the apazunersen program and assess its operations to implement significant cost savings while supporting its existing commercial business.
- Safety Profile: The safety results observed in the Phase 3 trial were consistent with those from the earlier Phase 1/2 study.