[Pubmed] Effect of Zilucoplan on Myasthenia Gravis-Specific Outcome Subdomain Scores in RAISE: A Phase 3 Study (S34.004)

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Neurology. 2025 Apr 8;104(7_Supplement_1):1803. doi: 10.1212/WNL.0000000000208755. Epub 2025 Apr 7.

ABSTRACT

OBJECTIVE: To evaluate the effect of zilucoplan, a macrocyclic peptide complement component 5 inhibitor, on Myasthenia Gravis Activities of Daily Living (MG-ADL) and Quantitative Myasthenia Gravis (QMG) subdomain scores (ocular, bulbar, respiratory and limb/axial muscle groups) in patients with generalized myasthenia gravis (gMG).

BACKGROUND: In RAISE (NCT04115293), zilucoplan demonstrated rapid and clinically meaningful improvements in MG-ADL and QMG total scores vs placebo in adults with acetylcholine receptor autoantibody-positive gMG. Since gMG affects a range of muscle groups, analysis of efficacy within specific muscle groups will enhance understanding of zilucoplan treatment in patients with gMG.

DESIGN/METHODS: Patients in RAISE, a Phase 3, multicenter, double blind, placebo-controlled study, were randomized to self-administer daily subcutaneous injections of zilucoplan 0.3 mg/kg (n=86) or placebo (n=88) for 12 weeks. Primary endpoint: change from baseline (CFB) in MG-ADL total score at Week 12. This exploratory analysis of RAISE data assessed CFB in MG-ADL and QMG subdomain scores for patients with baseline score ≥1 in that subdomain.

RESULTS: At Week 12, least-squares mean (LSM) (95% CI) CFB in MG-ADL total score was: zilucoplan -4.39 (-5.28, -3.50) and placebo -2.30 (-3.17, -1.43); difference -2.09 [-3.24, -0.95]; p=0.0004. LSM CFB in QMG total score was: zilucoplan -6.19 (-7.29, -5.08) and placebo -3.25 (-4.32, -2.17); difference -2.94 (-4.39, -1.49); p

Source: https://pubmed.ncbi.nlm.nih.gov/4019408 ... t9+e462414