[Pubmed] Efficacy of zilucoplan in patients with generalised myasthenia gravis who have not previously received immunogl

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J Neurol Sci. 2025 May 16;474:123550. doi: 10.1016/j.jns.2025.123550. Online ahead of print.

ABSTRACT

BACKGROUND: RAISE (NCT04115293; N = 174) was a randomised, double-blind, placebo-controlled, Phase 3 study of zilucoplan, a macrocyclic peptide complement component 5 inhibitor, in patients with anti-acetylcholine receptor antibody-positive (AChR Ab+) generalised myasthenia gravis (gMG). Zilucoplan showed clinically meaningful improvements in myasthenia gravis (MG)-specific outcomes versus placebo in the overall population. We assessed the profiles of RAISE patients without and with prior immunoglobulin (intravenous and subcutaneous) or plasma exchange (PLEX) use.

METHODS: Adults with anti-AChR Ab+ gMG were randomised 1:1 to daily, self-administered, subcutaneous zilucoplan 0.3 mg/kg or placebo for 12 weeks. The primary efficacy endpoint was change from baseline (CFB) to Week 12 in Myasthenia Gravis Activities of Daily Living (MG-ADL) score. We conducted a prespecified, descriptive analysis of subgroups without and with prior immunoglobulin/PLEX.

RESULTS: At baseline, 54 patients had no prior immunoglobulin/PLEX (zilucoplan: n = 29; placebo: n = 25); this subgroup had experienced milder disease and shorter disease duration from diagnosis than the subgroup with prior immunoglobulin/PLEX. Mean (standard error) CFB in MG-ADL score was -4.22 (0.71) with zilucoplan versus -2.61 (0.50) with placebo in the subgroup without prior immunoglobulin/PLEX, and - 4.93 (0.54) versus -2.94 (0.50) in the subgroup with prior immunoglobulin/PLEX. Zilucoplan was well tolerated with a similar safety profile in both subgroups.

CONCLUSIONS: In the RAISE study, patients without prior immunoglobulin/PLEX, who had milder disease, experienced improvements in MG-specific outcomes with zilucoplan that were comparable to those seen in patients with prior immunoglobulin/PLEX. These findings may support early use of zilucoplan in the gMG treatment paradigm.

PMID:40450840 | DOI:10.1016/j.jns.2025.123550


Source: https://pubmed.ncbi.nlm.nih.gov/4045084 ... t9+e462414