Safety, Efficacy, And Pharmacokinetics of Argx-117 in Adults With Multifocal Motor Neuropathy: A Global, Multicenter, Placebo Controlled Phase 2 Study (Arda)

Objective To report the study design of a Phase 2, multicenter, randomized, placebo controlled, parallel-group (ARDA, NCT05225675 ) will assess safety, efficacy, PK, and PD ARGX-117 in adults with MMN. Background Multifocal motor neuropathy (MMN) is chronic, immune-mediated characterized by progressive asymmetric weakness predominantly affecting distal upper limbs. MMN often associated anti-GM1 IgM antibodies targeting axolemma at node Ranvier, leading to activation classical complement pathway which drives subsequent damage axon. IVIg only proven effective therapy. novel monoclonal antibody that inhibits factor 2 (C2). Using an vitro model for MMN, was shown block IgM-mediated on both neurons Schwann cells. being studied study. Design/Methods Forty-eight participants be recruited assigned one two dosing cohorts (24 each) randomized 2:1 within each cohort receive either or placebo. Key inclusion criteria include: diagnosis probable definite per 2010 EFNS/PNS guidelines, stable regimen, treatment dependency. The primary objective safety based adverse event monitoring other assessments. Secondary objectives include assessment efficacy measured as strength functional disability, pharmacokinetics, pharmacodynamics (C2 CH50), immunogenicity ARGX-117. After completing 16-week period, may enroll open-label extension study, enter follow-up period. Results This ongoing phase Conclusions results this inform direct future studies patient population.