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Adimanebart (ARGX-119) is a first-in-class humanized agonist monoclonal antibody targeting muscle-specific kinase (MuSK). Developed by argenx, it is designed to treat various neuromuscular diseases by binding with high affinity to the Frizzled-like domain of MuSK. Its mechanism of action involves activating MuSK without interfering with its natural ligand, neural agrin, which leads to the clustering of acetylcholine receptors (AChR) and the stabilization of the neuromuscular junction (NMJ). Adimanebart is currently being evaluated in Phase 3 clinical trials for congenital myasthenic syndromes (CMS) and is in early-stage clinical development for amyotrophic lateral sclerosis (ALS) and spinal muscular atrophy (SMA).
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