GT-002

GT-002 is a selective small molecule positive allosteric modulator (PAM) of the GABAA receptor, developed by Gabather and originally discovered at Lund University. Unlike classic dopamine-blocking antipsychotics, GT-002 targets a novel binding site on the GABAA receptor distinct from benzodiazepines and elicits both tonic and phasic inhibitory currents. Preclinical studies have shown that GT-002 improves learning and memory, restores ketamine-induced cognitive deficits, inhibits behavioral effects induced by phencyclidine in schizophrenia models, is anxiolytic, promotes social interaction in rats, and does not produce withdrawal symptoms or drug abuse liability after long-term use. In clinical trials (three completed Phase I studies), it has demonstrated safety, tolerability, favorable pharmacokinetics for once-daily oral dosing, and target engagement as evidenced by increased EEG alpha band power—a marker associated with cognitive activity such as attention and relaxation. The drug is being developed primarily for neuropsychiatric disorders including schizophrenia (Phase I/II), behavioral disorders (Phase I), cognitive dysfunction (preclinical), dementia (preclinical), and frontotemporal dementia (planned pilot study). It is considered a new molecular entity.