Ultragenyx's Angelman Syndrome Drug Fails Phase 3, Stock Near 52-Week Low
RARE is trading near its 52-week low of $18.29 (19% below the low).
Summary
Ultragenyx's Phase 3 Aspire trial for apazunersen in Angelman syndrome failed its primary and key secondary endpoints. This is a major setback for a key pipeline asset in a rare neurodevelopmental disorder with high unmet need. The stock is already trading near its 52-week low, and this failure removes a significant near-term catalyst. The company's recent FDA approval of GENGLYCOS for GSDIa provides some offset, but the Angelman program was a major growth driver. Investors will now focus on the UX111 PDUFA date of September 19 and any strategic updates on the Angelman franchise.
At the time of this announcement, RARE was trading at $14.75 on NASDAQ in the Life Sciences sector, with a market capitalization of approximately $2.6B. The 52-week trading range was $18.29 to $39.89. This news item was assessed with negative market sentiment and an importance score of 9 out of 10. Source: Reuters.