Ultomiris Trial Fails Primary Endpoint in HSCT-TMA Patients
A Phase III clinical trial for Ultomiris (ravulizumab) did not achieve statistical significance for its primary endpoint of event-free survival in patients with thrombotic microangiopathy after hematopoietic stem cell transplant (HSCT-TMA).
AstraZeneca has announced that its drug Ultomiris did not meet the primary goal in a pivotal Phase III clinical trial. The study, designated ALXN1210-TMA-313, evaluated Ultomiris in adults and adolescents aged 12 years and older suffering from thrombotic microangiopathy following a hematopoietic stem cell transplant (HSCT-TMA).
The trial's main objective was to demonstrate event-free survival through 26 weeks compared to a placebo. High-level results indicated that Ultomiris (ravulizumab) did not achieve statistical significance on this primary endpoint, which was defined as the time from randomization until the occurrence of specific events.
HSCT-TMA is a severe and uncommon complication that can occur after a stem cell transplant, often leading to significant morbidity and mortality. The lack of statistically significant benefit in this trial suggests Ultomiris did not provide a clear advantage over placebo for this patient group in preventing events within the specified timeframe.
Full data and analyses from the trial are yet to be released by AstraZeneca, with plans to present detailed findings at future scientific meetings. The implications of these results for the future development of Ultomiris in the HSCT-TMA indication are still under evaluation.