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Monepantel is a small molecule anthelmintic belonging to the amino-acetonitrile derivative (AAD) class. It is primarily used in veterinary medicine for the treatment and control of gastrointestinal nematodes in sheep and cattle, including strains resistant to other anthelmintic classes[3][6]. Monepantel acts as a positive allosteric modulator of a nematode-specific clade of nicotinic acetylcholine receptor subunits (notably ACR-23/MPTL‑1), leading to persistent activation, depolarization, paralysis, and death of susceptible nematodes[1][3][6]. The drug has minimal toxicity in mammals due to the absence of its molecular target in these species. Recently, monepantel has been investigated for repurposing as a treatment for amyotrophic lateral sclerosis (ALS), where it inhibits the mTOR signaling pathway involved in protein homeostasis[5].
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