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Background In this interim analysis of RAISE-XT ( NCT04225871), an ongoing, Phase 3, open-label extension study, we evaluate the long-term safety and efficacy of zilucoplan, a macrocyclic peptide complement component 5 inhibitor, in patients with acetylcholine receptor autoantibody-positive generalised myasthenia gravis (gMG).Methods Adults with gMG who completed a qualifying double-blind study ( NCT03315130/NCT04115293) self-administered daily subcutaneous zilucoplan 0.3mg/kg. Primary outcome: incidence of treatment-emergent adverse events (TEAEs). Change from baseline (CFB) to Week 120 in MG Activities of Daily Living (MG-ADL) score was analysed for pooled data from participants who received zilucoplan 0.3mg/kg or placebo in the qualifying studies.Results 200 patients enrolled in RAISE-XT. At data cut-off (11 November 2023), median (range) exposure to zilucoplan was 2.2 (0.1–5.6) years. TEAEs occurred in 194 (97.0%) patients; 81 (40.5%) experienced a serious TEAE. The most common TEAEs were COVID-19 (71 [35.5%]) and MG worsening (59 [29.5%]). Of 183 patients who received zilucoplan 0.3mg/kg or placebo in the qualifying study, 93 continued zilucoplan and 90 switched from placebo to zilucoplan. At Week 120, mean CFB in MG-ADL score in pooled zilucoplan 0.3mg/kg patients was –7.14 (standard error 0.44).Conclusions Zilucoplan demonstrated a favourable long-term safety profile with sustained efficacy up to Week 120.maria.leite@ndcn.ox.ac.uk