AstraZeneca said on Monday its rare disease drug Ultomiris ​failed a late-stage trial in patients with a blood ‌vessel complication following a stem cell transplant, ​the latest in a series of ⁠setbacks this year that have fuelled concerns about the drugmaker’s pipeline.Here are some details:* Ultomiris did not ‌achieve statistical significance for event-free survival at 26 weeks in patients with haematopoietic stem cell ‌transplant-associated thrombotic microangiopathy, though it showed a ‌trend ⁠toward benefit.* AstraZeneca said it is ⁠in ongoing discussions with global health authorities over potential next steps for the adult indication.* In a separate trial involving ​children, Ultomiris showed an ‌overall survival rate of 87.2% at 26 weeks and 73.4% at 52 weeks, and the drugmaker said it will advance regulatory filings for ‌use in children.* The data comes on ​the heels of a failed pivotal heart-disease trial earlier this month, which have ⁠cast doubts over the drugmaker’s trial design.* In May, a U.S. regulatory panel rejected its ‌breast cancer drug camizestrant on trial design grounds.* AstraZeneca also reported that its experimental gastric cancer drug sonesitatug vedotin met a key goal, showing a statistically significant and clinically meaningful improvement in overall survival in advanced gastric cancer patients.* The ‌drugmaker, which topped second-quarter profit expectations on strong demand ​for its cancer and rare disease therapies, is counting on up to 20 new ⁠drug launches to help generate $80 billion in annual revenue ⁠by 2030.* Ultomiris, known chemically as ravulizumab, is already approved in the United ‌States, the EU and Japan for treating certain rare blood disorders and neurological conditions. Published on July 27, 2026