AstraZeneca says Ultomiris met main goal in late-stage study for rare autoimmune disorder
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AstraZeneca (NASDAQ:AZN) announced on Thursday that its late-stage study for Ultomiris in neuromyelitis optica spectrum disorder (NMOSD) met the primary endpoint indicating a statistically significant and clinically meaningful decline in the risk of relapse in adult patients.
The open-label Phase III CHAMPION-NMOSD trial was a global trial that involved 58 patients across North America, Europe, Asia-Pacific and Japan.
Ultomiris, a long-acting C5 complement inhibitor, reached the primary endpoint of time to first on-trial relapse when compared to the external placebo arm in the pivotal Soliris PREVENT clinical trial, the company said. No relapse was seen in 58 subjects over a 73-week median treatment duration.
NMOSD is a rare autoimmune disorder in which the immune system acts against the healthy tissues and cells in the central nervous system.
Safety and tolerability data of the trial were in line with previous study findings for Ultomiris and 56 patients are currently undergoing the treatment in a long-term extension period.
AstraZeneca (AZN) plans to present data at a future medical meeting and make regulatory submissions to make the drug available for NMOSD patients.
The Anglo-Swedish pharma giant added Ultomiris to the pipeline as part of the acquisition of U.S.-based Alexion Pharmaceuticals in 2021.