Ultragenyx is trading 47% down at $14.03 after apazunersen (GTX-102) failed its Phase 3 Aspire trial in Angelman syndrome.
- The treatment missed both its primary cognitive endpoint and key secondary MDRI endpoint, with no efficacy difference versus placebo.
- The company is reviewing the program and planning significant expense reductions.
- The biotech sector is higher, making this a company-specific selloff.