AUT00201

AUT00201 is a novel, orally active small molecule developed by Autifony Therapeutics for the treatment of central nervous system disorders. It acts as a potent positive allosteric modulator of Kv3 potassium channels—specifically Kv3.1 and Kv3.2—which are implicated in neuronal excitability and brain circuit function. By enhancing the activity of these channels, AUT00201 aims to restore normal neuronal firing patterns disrupted in diseases such as progressive myoclonic epilepsy type 7 (EPM7), ataxia, schizophrenia, hearing disorders, and Fragile X syndrome. Preclinical studies have shown that AUT00201 can normalize cerebellar dysfunction and reduce seizure susceptibility in relevant animal models[1][6][7][8]. The drug has received orphan drug designation for EPM7 from regulatory authorities.

Other names

2,4-Imidazolidinedione, 5-ethyl-3-[6-[(7-methylspiro[benzofuran-3(2H),1′-cyclopropan]-4-yl)oxy]-3-pyridinyl]-, (5R)-