Ultragenyx Pharmaceutical announced that its Phase 3 Aspire trial of apazunersen (GTX-102) in Angelman syndrome failed to meet its primary and key secondary cognitive and functional endpoints, prompting a review of the program and planned significant expense reductions. The company's stock fell 40.3% following the news. On the same day, the FDA confirmed that Ultragenyx's newly approved GENGLYCOS gene therapy for glycogen storage disease type Ia qualifies for a rare pediatric disease priority review voucher, highlighting a contrast between a major pipeline setback and progress in its commercial gene therapy portfolio. The company's narrative projects $1.2 billion revenue and $43.8 million earnings by 2029, with a fair value estimate of $27.00, implying a 76% upside to its current price. However, the failure raises questions about execution and the concentration of value in a few late-stage programs.
UltragenyxPhase 3 trial failure of apazunersen in Angelman syndrome missed endpoints, prompting program review and expense cuts.
