PRAX-020

PRAX-020 is a small molecule investigational drug candidate developed for the treatment of KCNT1-related epilepsy. It is designed as a selective inhibitor of the potassium sodium-activated channel subfamily T member 1 (KCNT1, also known as KNa1.1 or KCa4.1). KCNT1-related epilepsies are rare, severe forms of epilepsy that currently lack approved therapies. PRAX-020 originates from Praxis Precision Medicines’ research platform and has been advanced in partnership with UCB, which holds an exclusive option for global development and commercialization of resulting KCNT1-targeting small molecules. The program leverages genetic insights and aims to restore the balance of neuronal excitation and inhibition in affected patients[2][3][9][10].