New results from the ALXN1210-TMA-313 phase 3 clinical trial showed that ravulizumab, developed by Alexion, AstraZeneca’s rare disease division, did not achieve statistical significance for the primary endpoint of event-free survival in adults and adolescents.
The trial was one part of a wider announcement covering AstraZeneca’s ravulizumab programme in thrombotic microangiopathy following haematopoietic stem cell transplant (HSCT-TMA), a serious complication that can cause organ failure and death.
In adults and adolescents aged 12 years or older, the placebo-controlled ALXN1210-TMA-313 trial did not meet its primary endpoint of event free survival, defined as the time to TMA related clinical worsening or death. There was a trend toward treatment benefit at 26 weeks compared with placebo, though this fell short of significance. Discussions with health authorities were ongoing regarding how the findings, alongside real-world evidence, should be interpreted.
In the open-label ALXN1210-TMA-314 paediatric trial, the drug was associated with overall survival of 87.2% at 26 weeks and 73.4% at 52 weeks, supported further by external control data from the ALX-TMA-502 study. Alexion said it was advancing regulatory filings for paediatric HSCT-TMA based on these results.
Prof Christopher Dvorak, Chief of the Division of Paediatric Allergy, Immunology and Bone Marrow Transplantation, UCSF Benioff Children’s Hospitals, said: “Children who develop HSCT-TMA have an extremely poor prognosis without targeted treatment. The overall survival results observed in this phase 3 paediatric trial are clinically meaningful for this young patient population with significant unmet need.”
Prof Vincent Ho, Director of Clinical Operations, Adult Haematopoietic Stem Cell Transplantation, Dana-Farber Cancer Institute, said: “While the phase 3 trial in adults and adolescents did not meet the primary endpoint, the results add new, important insights to advance the field. Continued evaluation of these data together with real-world experience will further advance clinical understanding of this complex transplant complication.”
Marc Dunoyer, Chief Executive Officer, Alexion, said: “These trials have demonstrated the potential of [ravulizumab] to improve survival in paediatric patients with this complex condition. We are moving forward with regulatory filings while continuing engagement with global health authorities on potential next steps for the adult indication.”
Alexion said the safety profile observed across both trials was consistent with the known safety profile of ravulizumab and with that seen in patients undergoing HSCT. The company plans to present full data at a forthcoming medical meeting.
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