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)
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