Another Praxis win provides much-needed progress in rare genetic epilepsies

Shares of Praxis Precision Medicine rose 31% on Friday after a Phase II/III study of the company’s candidate for SCN2A and SCN8A developmental and epileptic encephalopathy (DEE) was stopped early for efficacy. The success of the small molecule is a bright spot during a challenging year for rare disease drug development and represents a step forward in the treatment of genetic forms of autism.

Praxis Precision Medicine Inc. (NASDAQ:PRAX) reported that relutrigine, a sodium channel modulator selective for disease-associated overactive channels, led to positive results in the second, registrational cohort of the EMBOLD study in patients with rare variants in SCN2A or de novo variants in SCN8A and associated seizures. The top-line results will be presented at the American Epilepsy Society Annual Meeting on Saturday, and Praxis will meet with FDA to determine the timing of an NDA submission...