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CONFERENCE UPDATE

Ravulizumab shows sustained efficacy and safety in preventing relapses in AQP4+ NMOSD: Interim results from the CHAMPION-NMOSD trial

NEUROLOGY

24 Jul 2025

STUDY DESIGN

Aquaporin-4 antibody-positive neuromyelitis optica spectrum disorder (AQP4+ NMOSD) is a rare autoimmune disease of the central nervous system characterized by recurrent and unpredictable relapses, which can lead to significant and irreversible neurological disability.¹ Ravulizumab is a long-acting C5 complement inhibitor designed to prevent relapses in patients with AQP4+ NMOSD.¹ This interim analysis presents long-term outcomes from the CHAMPION-NMOSD study, evaluating the efficacy and safety of ravulizumab in adults with AQP4+ NMOSD over a median follow-up period of 170.3 weeks.¹

The CHAMPION-NMOSD trial is an ongoing, global, open-label, phase 3 study evaluating ravulizumab in patients with AQP4+ NMOSD.¹ A total of 58 patients were enrolled in the primary treatment period (PTP), with 56 patients entering the long-term extension (LTE) phase as of the data cutoff on June 14, 2024.¹ Adult participants in PTP received an intravenous weight-based loading dose of ravulizumab on day 1, followed by maintenance doses starting on day 15 and every 8 weeks thereafter.¹ The placebo arm (n=47) of the PREVENT study, which evaluated eculizumab in patients with AQP4+ NMOSD, was used as an external control group.¹ After completing the PTP for up to 2.5 years, patients were eligible to enter the LTE phase.¹ Baseline demographics and clinical characteristics were generally similar between the ravulizumab group in CHAMPION-NMOSD and those in the placebo group from the PREVENT study.¹

The primary endpoints of the study was the time to first adjudicated on-trial relapse and relapse risk reduction.¹ Secondary endpoints included adjudicated relapse rate, and changes from baseline in Hauser Ambulation Index (HAI) and Expanded Disability Status Scale (EDSS) scores and safety profile.¹

“These findings support the long-term clinical benefit of ravulizumab in preventing relapses and preserving neurological function in adults with AQP4+ NMOSD"

Dr. Sean J. Pittock

Mayo Clinic,

Minnesota, United States

FINDINGS

Primary endpoint:

The primary endpoint of the study was time to first adjudicated on-trial relapse and relapse risk reduction¹

After a median follow-up of 170.3 weeks and a total of 186.6 patient-years of follow-up across both PTP and LTE groups, no adjudicated on-trial relapses were observed in patients treated with ravulizumab. This result corresponds to a 98.9% reduction in the risk of relapse as compared to placebo (p<0.0001)¹

Secondary endpoints:

Secondary endpoints included adjudicated on-trial relapse rate, and changes from baseline in HAI and EDSS scores¹

There is a significantly reduced rate of adjudicated on-trial relapse with ravulizumab compared to placebo (HR=0.011; 95% CI: 0.000-0.062; p<0.0001)¹

A total of 94.8% of patients treated with ravulizumab demonstrated stable or clinically improved scores on the HAI as of the LTE cutoff¹

89.7% of patients treated with ravulizumab showed no clinically important worsening in EDSS scores as of the LTE cutoff¹

Safety:

The safety profile of ravulizumab observed in this interim analysis remained consistent with findings from prior analyses¹

Treatment-emergent adverse events (TEAEs) occurred in 94.8% of patients, mostly mild or moderate and unrelated to ravulizumab; serious adverse events were reported in 25.9% of patients¹

Two cases of meningococcal infection were observed in vaccinated patients in the ravulizumab group during the PTP. Both were treated and recovered without any sequelae¹

Discontinuation of ravulizumab due to adverse events occurred in only one patient¹

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