Ultragenyx Reports Phase 3 Trial Failure for Apazunersen in Angelman Syndrome
Ultragenyx Pharmaceutical (RARE) announced that its Phase 3 Aspire trial of apazunersen, a drug candidate for Angelman syndrome, did not meet the primary endpoint or key secondary endpoints. The study aimed to assess changes from baseline using cognitive raw scores as measured by Bayley-4. The failure marks a setback in Ultragenyx's pipeline development and could impact investor confidence in its rare disease therapies. Apazunersen had previously shown promise in earlier trials, but the Phase 3 results indicate it may not be effective enough to warrant regulatory approval for this indication.
Tickers: $RARE