Ultragenyx Pharma plunges after rare disease drug trial failure
03/09/2026 09:54
BUZZ-Ultragenyx Pharma plunges after rare disease drug trial failure ** Shares of Ultragenyx Pharmaceutical RARE.O fall 45.5% to $14.4 premarket
** Co's drug apazunersen failed to meet its main goal in a late-stage trial for Angelman syndrome, a condition that affects the nervous system and impairs typical brain development in childhood
** The drug did not show meaningful improvement in children's cognitive abilities or overall development compared with placebo
** Ultragenyx said it reviews the future of the apazunersen program
** The readout is "unambiguously negative" and the failure "raises risk for competitors" like Ionis IONS.O, who have their own similar late-stage Angelman trials ongoing
** As of last close, stock up 15.4% YTD
(Reporting by Kamal Choudhury in Bengaluru)
Recent news on Ultragenyx Pharmaceutical
See all newsBrief: Ultragenyx Pharmaceutical Enters Settlement Agreement With Navinta On Dojolvi Patent Litigation - SEC Filing
Ultragenyx gains on $210 million US FDA voucher sale
Brief: Ultragenyx Pharmaceutical - Under Deal With U.S. Department Of Health Co To Pay 20% Of Prv Sale Proceeds To Nih
Brief: Ultragenyx Enters Into Agreement To Sell Rare Pediatric Disease Priority Review Voucher For $210 Million
U.S. STOCKS ON THE MOVE-CoreWeave, StubHub, Ultragenyx