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| Other names | AUT-00063 |
| Routes of administration | Oral |
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| Formula | C20H21N3O4 |
| Molar mass | 367.405 g·mol−1 |
AUT00063 (also called AUT-00063) is an investigational small-molecule compound developed by Autifony Therapeutics for hearing loss and tinnitus. [1]
AUT00063 was identified as a modulator of Kv3 (Shaw) family potassium channels, which play a role in the firing properties of auditory neurons and are implicated in central auditory hyper-activity hypotheses of tinnitus. [2]
In June 2014 Autifony announced that it had been awarded a £2.2 million grant from the UK's Technology Strategy Board (now Innovate UK) toward a Phase IIa clinical trial in tinnitus patients. [3]
A randomised, double-blind, placebo-controlled Phase IIa study (EudraCT 2014–002179–27) titled "QUIET-1" enrolled participants with subjective tinnitus to assess efficacy and safety of AUT00063 versus placebo. [4]
However, the trial was terminated early for futility: Autifony announced on 28 April 2016 that headline data from QUIET-1 "confirm lack of efficacy of AUT00063 in the treatment of people with mild-to-moderate tinnitus.". [4]
AUT00063 targets Kv3 (Shaw) potassium channels, which are voltage-gated channels expressed in the central auditory pathway and help control rapid repolarization and high-frequency firing of neurons. By modulating these channels, the drug aimed to reduce abnormal spontaneous activity (a proposed substrate of tinnitus) in central auditory circuits. [5]