Comparison of Uplizna vs Azathioprine + Immunosuppressives vs Placebo for AQP4+ NMOSD

Comparative analysis of data from the N-MOmentum clinical trial (NCT02200770) open-label extension (OLE) and previously published NMOSD studies demonstrated statistically significant differences in efficacy in terms of time to onset of NMOSD attack for Uplizna (inebilizumab-cdon, Horizon Therapeutics, Dublin, Ireland) treatment vs azathioprine and other immunosuppressive therapies and vs placebo for patients with NMOSD. The results were presented at the 9th joint meeting of the European and American Committees for Treatment and Research in Multiple Sclerosis (ECTRIMS-ACTRIMS).

N-MOmentum was a multicenter, double-blind, randomized placebo-controlled phase 2/3 clinical trial evaluating the safety and efficacy of Uplizna for treatment of patients with NMOSD. For their new study, researchers assessed the long-term efficacy of Uplizna by comparing data from the anti-aquaporin-4 (AQP4) antibody positive subpopulation of the N-MOmentum OLE with 2 historical comparator groups (HCGs) from published NMOSD studies. The first HCG received azathioprine and other immunosuppresive therapies ([AZA/IST] N=132), and the second HCG received placebo only (N=106). Hazard ratios (HR) were estimated using a Cox proportional hazards regression, and time to NMOSD attack was analyzed using parametric and flexible survival (spline) models.

• The HR for time to NMOSD attack for patients treated with Uplizna was 0.29 (95% CI: 0.17, 0.42; P<.001) vs 0.15 (95% CI: 0.10, 0.21; P<.001) for patients treated from both HCGs, including those taking AZA/IST and those taking placebo only.
• The spline model estimated an attack-free survival of 77% (95% CI: 71, 83) for the Uplizna group, 36% (95% CI: 27, 46) for the azathioprine and other immunosuppresive therapies group, and 12% (95% CI: 7, 20) for the placebo only group.

Researchers who are part of this study represent a variety of institutions, including the UCSF Weill Institute for Neurosciences, Eversana, Pierre Wertheimer Hospital, UTHealth Houston, Research Institute and Hospital of National Cancer Center, Horizon Therapeutics, and the Max Delbrück Center for Molecular Medicine.