AstraZeneca drug Ultomiris misses primary endpoint in late stage clinical trial

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AstraZeneca said its rare disease drug Ultomiris failed a late-stage trial in patients with a blood vessel complication after a stem cell transplant, missing the primary endpoint of event-free survival at 26 weeks.
The company said it is discussing next steps with global health authorities for adults, while a separate children's trial showed positive survival results and will move forward with regulatory filings.
The failure adds to a string of recent pipeline setbacks for the drugmaker, though its experimental gastric cancer drug met a key goal and the company beat second-quarter profit expectations.
The outcome matters because AstraZeneca is relying on up to 20 new drug launches to reach $80 billion in annual revenue by 2030.

Outcome of AstraZeneca's discussions with global health authorities on potential next steps for the adult indication
Progress of regulatory filings for Ultomiris use in children
Whether further trial failures deepen concerns about the company's trial design

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Named United States · Japan · Camizestrant · Ravulizumab · Sonesitatug Vedotin · Ultomiris · AstraZeneca · US regulatory panel

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