{
  "abstract": "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",
  "authors": [
    {
      "affiliations": [
        "Nuffield Department of Clinical Neurosciences, University of Oxford, Oxford, UK"
      ],
      "name": "Leite Maria Isabel"
    },
    {
      "affiliations": [
        "The Ohio State University Wexner Medical Center, Columbus, OH, USA"
      ],
      "name": "Freimer Miriam"
    },
    {
      "affiliations": [
        "Clinical Research Unit, The Montreal Neurological Institute, Montreal, QC, Canada"
      ],
      "name": "Genge Angela"
    },
    {
      "affiliations": [
        "Sheffield Teaching Hospitals NHS Foundation Trust and University of Sheffield, Sheffield, UK"
      ],
      "name": "Hewamadduma Channa"
    },
    {
      "affiliations": [
        "Department of Neurology, The University of North Carolina at Chapel Hill, Chapel Hill, NC, USA"
      ],
      "name": "Howard James F"
    },
    {
      "affiliations": [
        "Department of Neurology, Dell Medical School, The University of Texas at Austin, Austin, TX, USA"
      ],
      "name": "Hussain Yessar"
    },
    {
      "affiliations": [
        "Department of Neurology, Oslo University Hospital, Oslo, Norway"
      ],
      "name": "Maniaol Angelina"
    },
    {
      "affiliations": [
        "Istituto Nazionale Neurologico Carlo Besta, Milan, Italy"
      ],
      "name": "Mantegaza Renato"
    },
    {
      "affiliations": [
        "Department of Neurology, University of South Florida Morsani College of Medicine, Tampa, FL, USA"
      ],
      "name": "Vu Tuan"
    },
    {
      "affiliations": [
        "Nuffield Department of Clinical Neurosciences, University of Oxford, Oxford, UK"
      ],
      "name": "et al"
    }
  ],
  "title": "2 Long-term safety and efficacy of zilucoplan in generalised myasthenia gravis: 120-week interim analysis of RAISE-XT",
  "uid": "55c2e584-d025-556d-8ea8-db358077eb70"
}