Efficacy of nipocalimab in patients with moderate-to-severe ocular symptoms of generalized myasthenia gravis in the phase 3 Vivacity-MG3 study.

BACKGROUND: In the global Vivacity-MG3 study, nipocalimab (30-mg/kg loading dose; 15-mg/kg every-2-weeks) demonstrated sustained disease control versus placebo plus standard-of-care for generalized myasthenia gravis (gMG); this post-hoc analysis evaluated nipocalimab efficacy for participants with moderate-to-severe ocular symptoms (baseline score of ≥2-points on either ocular-domain item of MG-Activities-of-Daily-Living [MG-ADL]). RESEARCH DESIGN AND METHODS: Mean MG-ADL and Quantitative Myasthenia Gravis total scores change-from-baseline (CFB) through Week (W)24 were assessed using ANCOVA. W24% of participants with ≥2-point improvement in MG-ADL total (meaningful-within-person-improvement) or ocular-domain scores were compared using odds ratios (OR). Longitudinal odds of ≥2-point ocular-domain improvement were evaluated using generalized-estimating-equations logistic models. RESULTS: Nipocalimab (n = 54/77) and placebo-treated (n = 51/76) participants with moderate-to-severe ocular symptoms had similar baseline (mean[SD]) MG-ADL total (10.1[2.8];9.4[1.9]); ocular-domain scores were 4.1 (1.2);3.5 (1.0). At W24, LS-mean(SE) CFB for nipocalimab versus placebo were -4.71 (0.50) versus -3.24 (0.50), p = 0.042 (MG-ADL total) and median (IQR) -2.00 (-3.0 to -0.5) versus -1.00 (-1.5 to 0.0), p = 0.024 (ocular-domain). W24 ≥ 2-point ocular-domain improvements were observed with nipocalimab (40.7%) versus placebo (19.6%; OR = 3.47 [95% CI = 1.34-8.97]; p = 0.010). Adverse events: 77.8% (nipocalimab); 76.5% (placebo). CONCLUSION: These hypothesis-generating results suggest nipocalimab may provide sustained disease control, reduce ocular symptoms, and improve activities-of-daily-living versus placebo in participants with gMG and moderate-to-severe ocular symptoms. CLINICAL TRIAL REGISTRATION: www.clinicaltrials.govidentifier is NCT04951622;www.clinicaltrialsregister.euidentifier is 2020-005732-29.