6/28/23, 12:02 AM ADAPT Pathway Feedback Search Clinical Topics Home Studies ADAPT ADAPT Disease Myasthenia gravis Trial question What is the role of efgartigimod in patients with generalized myasthenia gravis? Study design Multi-center Double blinded RCT Population Characteristics of study participants 71.0% female N = 167 29.0% male 167 patients (118 female, 49 male) Inclusion criteria: adult patients with myasthenia gravis and a Myasthenia Gravis Activities of Daily Living score 5 Key exclusion criteria: receipt of rituximab or eculizumab within 6 months; thymectomy within 3 months; IVIG or plasma exchange within 1 month; active hepatitis B; hepatitis C seropositivity; HIV seropositivity with low CD4 count; serum IgG levels < 6 g/L; pregnancy Interventions N=84 efgartigimod (intravenous dose of 10 mg/kg, administered as four infusions per cycle; one infusion per week) N=83 placebo (intravenous administration of matching placebo) https://web.pathway.md/studies/recgjSyIfTE4nQRGB 1/2 6/28/23, 12:02 AM ADAPT Pathway Primary outcome Percentage of positive anti-acetylcholine receptor antibody patients with a clinically meaningful reduction in Myasthenia Gravis Activities of Daily Living score at cycle 1 68.0 % 68 51.0 % 34.0 % 30 Significant increase 17.0 % NNT = 3 0.0 % Efgartigimod Placebo Significant increase in the percentage of positive anti-AChR antibody patients with a clinically meaningful reduction in Myasthenia Gravis Activities of Daily Living score at cycle 1 (68% vs. 30%; OR 4.95, 95% CI 2.21 to 11.53) Secondary outcomes Significant increase in the percentage of patients with a clinically meaningful reduction in Quantitative Myasthenia Gravis score (63% vs. 14%; OR 10.84, 95% CI 4.18 to 31.2) Significant increase in the percentage of all patients with a clinically meaningful reduction in Myasthenia Gravis Activities of Daily Living score at cycle 1 (68% vs. 37%; OR 3.7, 95% CI 1.85 to 7.58) Significant increase in the percentage of patients with a clinically meaningful reduction in Myasthenia Gravis Activities of Daily Living score at day 126 (48.7% vs. 26.6%; AD 22.1%, 95% CI 11.06 to 33.14) Safety outcomes No significant difference in adverse events. Conclusion In adult patients with myasthenia gravis and a Myasthenia Gravis Activities of Daily Living score 5, efgartigimod was superior to placebo with respect to the percentage of positive anti-AChR antibody patients with a clinically meaningful reduction in Myasthenia Gravis Activities of Daily Living score at cycle 1. Reference James F Howard Jr, Vera Bril, Tuan Vu et al. Safety, efficacy, and tolerability of efgartigimod in patients with generalised myasthenia gravis (ADAPT): a multicentre, randomised, placebo- controlled, phase 3 trial. Lancet Neurol. 2021 Jul;20(7):526-536. Open reference URL https://web.pathway.md/studies/recgjSyIfTE4nQRGB 2/2