AstraZeneca revealed Monday that its rare disease medication Ultomiris did not achieve its main objective in an advanced clinical trial testing the drug in patients who developed a blood vessel complication following a stem cell transplant.
Specifically, Ultomiris failed to reach statistical significance for event-free survival at 26 weeks among patients with haematopoietic stem cell transplant-associated thrombotic microangiopathy — a serious blood vessel disorder. While the drug did show a trend pointing toward benefit, it was not enough to meet the trial’s primary benchmark.
The company stated it is currently in discussions with health regulators around the world to determine possible next steps for the adult version of the treatment.
Results were more encouraging on the pediatric side. A separate trial involving children showed an overall survival rate of 87.2% at the 26-week mark and 73.4% at 52 weeks. Based on those findings, AstraZeneca said it plans to move forward with regulatory filings seeking approval for use in children.
This latest disappointment follows a failed pivotal heart disease trial earlier this month, which has raised questions about the company’s approach to trial design. Back in May, a U.S. regulatory panel also rejected AstraZeneca’s breast cancer drug camizestrant, again citing trial design concerns.
On a more positive note, AstraZeneca reported that its experimental gastric cancer drug sonesitatug vedotin hit a key milestone, demonstrating a statistically significant and clinically meaningful improvement in overall survival among patients with advanced gastric cancer.
The drugmaker, which beat second-quarter profit expectations thanks to strong demand for its cancer and rare disease treatments, has set an ambitious target of launching up to 20 new drugs to help it reach $80 billion in annual revenue by 2030.
Ultomiris, whose chemical name is ravulizumab, already holds regulatory approval in the United States, the European Union, and Japan for treating certain rare blood disorders and neurological conditions.
