AUT-00063

AUT00063 is an investigational small-molecule compound developed by Autifony Therapeutics for hearing loss and tinnitus.

Background and development

AUT00063 was identified as a modulator of Kv3 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.
In June 2014 Autifony announced that it had been awarded a £2.2 million grant from the UK's Technology Strategy Board toward a Phase IIa clinical trial in tinnitus patients.

Clinical trials

Tinnitus (QUIET-1)

A randomised, double-blind, placebo-controlled Phase IIa study titled "QUIET-1" enrolled participants with subjective tinnitus to assess efficacy and safety of AUT00063 versus placebo.
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.".

Mechanism of action

AUT00063 targets Kv3 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 in central auditory circuits.