AUT00063

AUT00063 is an orally active, experimental small molecule drug developed by Autifony Therapeutics. It acts as a modulator of Kv3 (Shaw) voltage-gated potassium channels, specifically targeting the Kv3.1 subunit in central auditory cortical neurons. The drug was designed to suppress spontaneous neuronal hyperactivity and was investigated for its potential to treat hearing disorders such as age-related hearing loss (presbycusis), tinnitus, ototoxicity, and other forms of hearing impairment. Despite promising preclinical results in rodent models—where it reduced hyperactivity associated with tinnitus—the compound did not demonstrate clinical efficacy in human trials for tinnitus or age-related hearing loss but was found to be safe and well tolerated[1][2][3][5][6].