RARE

Ultragenyx (RARE) reported earnings 30 days ago. What's next for the stock?

Ultragenyx plunges 46% after its phase III Angelman syndrome study fails to achieve key efficacy goals, putting apazunersen's future in question.

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) stock is tumbling on Thursday after reporting late-stage clinical trial failures for its Angelman syndrome therapy, apazunersen, prompting the biopharmaceutical firm to review the program's future and plan significant corporate expense reductions.

Ultragenyx Pharmaceuticals looks to cut costs following the trial failure, while attention remains squared on its burgeoning commercial business.

Shares of Ultragenyx Pharmaceutical plunged more than 40% in premarket on Thursday after its neurodevelopmental disorder treatment failed a closely watched ​late-stage trial, dealing a major blow to one of ‌its biggest growth bets.

Phase 3 Aspire did not achieve the primary endpoint of change from Baseline in Bayley-4 cognitive raw score nor the key secondary endpoint of net response in Multidomain Responder Index (MDRI)

At Week 96, participants across treatment and crossover groups experienced mean reduction in daily cornstarch intake of 61% while maintaining glycemic control, with most participants achieving reduction of at least 50%

ALNY, BMRN, RARE and BBIO offer rare-disease growth potential through strong products, pipeline catalysts and high unmet medical needs.

NOVATO, Calif., Aug. 25, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultra-rare diseases, today reported the grant of 45,984 restricted stock units of the company's common stock to 23 newly hired non-executive officers of the company.

Ultragenyx stock rises as Genglycos wins FDA's accelerated approval as the first treatment targeting the underlying cause of GSDIa, boosting its rare-disease portfolio.