Ultragenyx Plans Cost Cuts After Angelman Drug Fails Phase 3
RARE is trading near its 52-week low of $13.81 (7.3% above the low) on elevated volume (4.0× avg).
Summary
Ultragenyx's Phase 3 Aspire trial for apazunersen in Angelman syndrome failed its primary and key secondary endpoints, sending shares down 47.57% to $13.91 in premarket trading. The company now plans to evaluate the program's future and implement significant expense reductions while supporting its commercial business. This follows the FDA approval of GENGLYCOS for GSDIa in August, which provides a revenue base but does not offset the loss of the Angelman opportunity. The restructuring signals a shift in strategy as the company prioritizes its approved gene therapy portfolio. Investors will watch for details on the scope of cost cuts and any further pipeline adjustments.
At the time of this announcement, RARE was trading at $14.83 on NASDAQ in the Life Sciences sector, with a market capitalization of approximately $1.5B. The 52-week trading range was $13.81 to $39.89. This news item was assessed with negative market sentiment and an importance score of 7 out of 10. Source: Benzinga.