Ultomiris Met Primary Endpoint in CHAMPION-NMOSD Phase III Trial in Adults with Neuromyelitis Optica Spectrum Disorder

Positive high-level results from the open-label Phase III CHAMPION-NMOSD trial showed that Ultomiris (ravulizumab-cwvz) achieved a statistically significant and clinically meaningful reduction in the risk of relapse in adults with anti-aquaporin-4 (AQP4) antibody-positive (Ab+) neuromyelitis optica spectrum disorder (NMOSD) compared to the external placebo arm from the pivotal Soliris PREVENT clinical trial.

Ultomiris, the first and only long-acting C5 complement inhibitor, met the primary endpoint of time to first on-trial relapse, as confirmed by an independent adjudication committee. Notably, no relapse was observed in 58 patients over a median treatment duration of 73 weeks.

NMOSD is a rare and devastating autoimmune disease that affects the central nervous system (CNS), including the spine and optic nerves.^1-3 Most people living with NMOSD often experience unpredictable relapses, a new onset of neurologic symptoms or worsening of existing neurologic symptoms, also referred to as attacks, which tend to be severe and recurrent and may result in permanent disability.^4-6

Sean J. Pittock, MD, Director of Mayo Clinic’s Center for Multiple Sclerosis and Autoimmune Neurology and of Mayo’s Neuroimmunology Laboratory and lead primary investigator in the CHAMPION-NMOSD trial, said: “Every NMOSD relapse can have debilitating and irreversible consequences, so reducing relapses is critical. Patients on Ultomiris remained relapse free over a median treatment duration of 73 weeks in the trial.”

Marc Dunoyer, Chief Executive Officer, Alexion, said: “Soliris established the role of complement inhibition in preventing relapses in NMOSD, and with Ultomiris, we continue to innovate for patients with a more convenient every eight-week dosing schedule. These trial results show that Ultomiris may help patients move towards eliminating relapses, which is an important advancement in the treatment of NMOSD.”

The safety and tolerability of Ultomiris in the Champion-NMOSD trial were consistent with previous clinical studies and other approved indications. Fifty-six patients are continuing to receive treatment in a long-term extension period, which is ongoing.

The data will be presented at a forthcoming medical meeting and submitted to global health authorities as rapidly as possible to bring forward Ultomiris to the NMOSD community.