To describe a proof-of-concept study investigating the safety, tolerability, and exploratory efficacy of gaboxadol (OV101) in Fragile X syndrome (FXS).

FXS is a rare neurodevelopmental condition, caused by a genetic mutation and associated with intellectual disability, autism spectrum disorder, anxiety, and maladaptive behaviors. Individuals with FXS are at increased risk for a range of behavioral problems that may limit their academic performance, adaptive functioning, daily living skills, and social interactions, as well as impact their quality of life. Interventions include speech, physical, and occupational therapy, as well as educational resources. Currently, all approved medications for FXS target behavioral and psychiatric symptoms and not the underlying core brain deficits.

Gaboxadol is a highly selective orthosteric agonist that activates the δ-containing subunit of extrasynaptic GABA_A receptors. Preclinical studies on the use of gaboxadol for treatment of FXS has been conducted in mouse models of the condition.