Ultragenyx Shares Drop After Phase 3 Trial for Apazunersen Fails to Meet Key Endpoints
Ultragenyx Pharmaceuticals (NASDAQ:RARE) experienced a significant drop in its stock price after announcing that the pivotal Phase 3 Aspire study of apazunersen, an investigational treatment for Angelman syndrome, did not meet either primary or key secondary endpoints. The trial's main goal was to assess cognitive improvement using the Bayley-4 score and multidomain response index; however, no meaningful differences were observed between the drug and placebo groups. Following these results, several analysts downgraded their ratings and price targets for Ultragenyx. Evercore ISI reduced its rating from Outperform to In Line with a new $16 target (from $34). Baird also cut its rating to Neutral with a similar revised target of $16. JPMorgan lowered the stock's status from Overweight to Neutral, while William Blair removed GTX-102’s contribution from its valuation model due to ongoing cash burn and future exclusivity risks for Crysvita. Ultragenyx shares fell 44.7% in premarket trading to $14.67, below their previous 52-week low of $18.29.
Tickers: $RARE
Topics: biotech drug_development stock_movement