Horizon Therapeutics plc (Nasdaq: HZNP) today announced new findings from a post hoc analysis of the N-MOmentum Phase 3 pivotal trial of UPLIZNA supporting the medicine’s efficacy in Europeans living with NMOSD. These data are being presented during the 8 th Congress of the European Academy of Neurology (EAN), June 25-28 in Vienna. UPLIZNA received marketing authorization from the European Commission (EC) on April 25, 2022 and is the first and only targeted CD19+ B-celldepleting monotherapy proven to reduce attacks in adult patients with NMOSD who are anti-aquaporin-4 immunoglobulin G seropositive (AQP4- IgG+).
This post hoc analysis compared attack rates, disability-related outcomes and safety among 50 trial participants from the European Union (EU) (including participants from Bulgaria, Czech Republic, Estonia, Germany, Hungary and Poland) versus 163 non-EU participants. “People living with NMOSD in Europe need novel treatment options that have been shown to reduce attacks that can cause irreversible and debilitating damage, such as vision loss and paralysis,” said Friedemann Paul, M.D., study author and head, Clinical Neuroimmunology Research Group, NeuroCure Clinical Research Center, and head, Clinical and Experimental Neuroimmunology, Experimental and Clinical Research Center, Charité–Universitätsmedizin Berlin and Max Delbrueck Center for Molecular Medine, Berlin, Germany. “With UPLIZNA, physicians have a treatment option that can be given twice a year after initial dosing to help prevent NMOSD attacks by specifically targeting CD19 B-cells, which play a central role in the pathogenesis of the disease.”
Key analysis findings:
- Participants in the EU who were treated with UPLIZNA experienced fewer attacks (12.5%) compared to those treated with placebo (30%), sharing similar results with non-EU participants receiving UPLIZNA (10.7%) or placebo (45.2%).
-No significant differences in Expanded Disability Status Scale (EDSS) worsening were found between participants in the EU (15%) versus non-EU participants (14.9%).
-Fewer NMOSD-related hospitalisations were reported among those receiving UPLIZNA compared to those treated with placebo (mean, EU: 1.0 vs 2.0; non-EU: 1.0 vs 1.33).
“The UPLIZNA pivotal trial is the largest in NMOSD and clearly demonstrates the merits of targeting CD19 B-cells, including plasmablasts and plasma cells, to provide broad, deep and durable B-cell depletion,” said Karl Boegl, M.D. Ph.D., executive director, EMEA regional medical affairs lead, Horizon. “We believe these data provide treating physicians with greater certainty that a targeted monotherapy like UPLIZNA can be a valuable option for the treatment of NMOSD patients in Europe.”