An international team led by Prof. Lih-Chu Chiou (College of Medicine, National Taiwan University), has identified a promising drug candidate, the α6-containing GABAA receptor-selective positive allosteric modulator. If successfully approved, it will be the first-in-class therapy for migraine schizophrenia and Tourette syndrome with excellent pharmacokinetics and safety pharmacology profile.

This small molecule compound is a selective ligand of α6GABAAR. These receptors are limitedly expressed in the nervous system, eg. the cerebellum and trigeminal ganglia. Thus, it unlikely has the side effects as those non-selective ligands of GABAAR, like benzodiazepines, including tolerance, sedation etc. If approved, it will be a first-in-class therapy for migraine and neuropsychiatric disorders.

The candidate compound has promising safety pharmacology and pharmacokinetic properties, and may emerge as novel drug candidate for migraine, schizophrenia and Tourette syndrome. It is ready to enter pre-clinical IND-enabling studies. If successfully approved in the future, it would provide the first-in-class and safe pharmacotherapy to the patients inflicted with the mentioned diseases.