Subgroup outcomes from RAISE: a randomised, Phase 3 trial of zilucoplan in generalised myasthenia gravis

• Published in: Journal of neurology, neurosurgery and psychiatry Vol. 94; no. Suppl 1; p. A81
• Main Authors: Hewamadduma Channa, Bresch Saskia, Juntas-Morales Raul, Leite, Isabel, Maniaol Angelina, Zajda Malgorzata, Boroojerdi Babak, Duda Petra, de la Borderie Guillemette, Howard, James
• Format: Journal Article
• Language: English
• Published: London BMJ Publishing Group LTD 01.11.2023
• Subjects: Myasthenia gravis
• ISSN: 0022-3050; 1468-330X
• DOI: 10.1136/JNNP-2023-ABN.251

BackgroundRAISE (NCT04115293) was a Phase 3, multicentre, double-blind, placebo-controlled study of the complement C5 inhibitor zilucoplan that demonstrated statistically significant and clinically meaningful improvement in MG-related efficacy endpoints in patients with AChR Ab+ gMG.Design/MethodsAdults (MGFA Disease Class II–IV gMG, AChR Ab+, MG-ADL score ≥6, QMG score ≥12) were randomised 1:1 to daily subcutaneous doses of zilucoplan 0.3 mg/kg or placebo for 12 weeks. Primary efficacy endpoint: change from baseline (CFB) at Week 12 in MG-ADL score. Efficacy outcomes were assessed according to pre-specified subgroups: MG-ADL ≤9 or ≥10, QMG ≤17 or ≥18, and disease duration <5 or ≥5 years at baseline.ResultsOverall, 174 participants were randomised to zilucoplan (n=86) or placebo (n=88). At Week 12, mean CFB in MG-ADL for zilucoplan vs placebo was consistently improved in each subgroup: baseline MG-ADL ≤9: −3.88 vs −2.48 and ≥10: −5.24 vs −3.06; baseline QMG ≤17: −4.19 vs −2.81 and ≥18: −5.11 vs −2.88; and duration of disease <5 years: −3.92 vs −3.04 and ≥5 years: −5.38 vs −2.62. Zilucoplan showed a favourable safety profile and was well-tolerated.ConclusionZilucoplan demonstrated consistent improvements in MG-specific efficacy outcomes irre- spective of disease severity or duration. Funding: UCB Pharma.