Drug intelligence / Profile preview

gaboxadol

Development stage
Phase 3
Lead developer
Healx
Modality
Small Molecules
Administration
Oral
01

Overview

Gaboxadol is a small molecule experimental drug that acts as a highly selective agonist at extrasynaptic GABAA receptors containing the α4 and δ subunits. Unlike benzodiazepines and other sedative-hypnotics that act primarily at synaptic GABAA receptors, gaboxadol targets delta-containing GABAA receptor subtypes located outside the synapse (extrasynaptic), particularly those with α4β3δ composition. This mechanism enhances tonic inhibition in the brain and is thought to promote slow-wave sleep by modulating thalamic neuronal activity. Gaboxadol was originally developed as a sleep aid for insomnia by Lundbeck and Merck but its development was discontinued in 2007 due to concerns about safety and efficacy. More recently, it has been investigated for neurological disorders such as Angelman syndrome, fragile X syndrome, and epilepsy[1][2][3][4][5].

Other names
4,5,6,7-tetrahydroisoxazolo(5,4-c)pyridin-3-ol
02

Targets

GABAAR α4β3δ (GABA-A receptor α4β3δ subtype)GABA_A receptor α4/α6δ (Gamma-aminobutyric acid type A receptor alpha-4/alpha-6 delta family)GABA-A receptor α1β3γ2 (Gamma-aminobutyric acid type A receptor alpha-1 beta-3 gamma-2)GABRA5 (Gamma-aminobutyric acid type A receptor subunit alpha-5)GABRR (GABA-A receptor subunit rho)GABA-A receptor α1β2γ2 (GABA_A receptor α1β2γ2 – N-methyllaurotetanine)

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