Drug intelligence / Profile preview

AUT2

Development stage
Preclinical
Lead developer
Autifony Therapeutics
Modality
Small Molecules
01

Overview

AUT2 is a small-molecule positive allosteric modulator of the voltage-gated potassium channels Kv3.1 (KCNC1) and Kv3.2 (KCNC2). Developed by Autifony, AUT2 acts by shifting the voltage dependence of channel activation to more negative potentials, thereby increasing channel opening at lower membrane potentials. At higher concentrations, it also shifts steady-state inactivation to more negative potentials. AUT2 has been primarily investigated as a preclinical research tool in animal models of Fragile X syndrome (FXS), where it has been shown to normalize the firing patterns of auditory brainstem neurons and rescue auditory brainstem responses (ABR) in Fmr1-knockout mice, suggesting potential therapeutic utility in treating sensory hypersensitivity and hearing disorders.

Other names
4-({5-[(4R)-4-ethyl-2,5-dioxo-1-imidazolidinyl]-2-pyridinyl}oxy)-2-(1-methylethyl)benzonitrile
02

Targets

KCNC1 (Kv3.1)KCNC2 (Potassium voltage-gated channel subfamily C member 2)

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