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.
Updates
· Reuters — Shares fell more than 40% premarket; at least five brokerages cut price targets; company announced significant expense reductions.
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.