Ultragenyx Plunges 40% as Angelman Drug Fails Phase 3, Analysts Slash Targets
RARE is trading near its 52-week low of $18.29 (18% below the low) on elevated volume (3.0× avg).
Summary
Ultragenyx shares cratered more than 40% in premarket trading after its Phase 3 Aspire trial for apazunersen in Angelman syndrome failed both primary and key secondary endpoints. The company said there were no differences between treatment and placebo, and it will review the program's future. This is the second consecutive late-stage trial failure for Ultragenyx, following the earlier setback, and at least five brokerages cut price targets. The company announced significant expense reductions to manage high operating costs. Analysts called the failure 'unambiguously negative' and said it removes a key growth driver, shifting the investment case to a commercial and expense story. Ionis Pharmaceuticals is mentioned as a competitor with a similar program, but the direct impact is on Ultragenyx.
At the time of this announcement, RARE was trading at $14.94 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 8 out of 10. Source: Reuters.