Praxis Announces Positive Elsunersen Phase 1/2 Results and FDA Priority Review for Relutrigine NDA

Praxis Precision Medicines announced positive topline results from Part A of the EMBRAVE Phase 1/2 trial evaluating elsunersen in pediatric patients with SCN2A developmental and epileptic encephalopathy (DEE). The trial met its objectives, demonstrating a 77% placebo-adjusted seizure reduction from baseline (p=0.015). Notably, 57% of patients achieved at least a 28-day period of seizure freedom, and improvements were observed across motor function, sleep, and attention. Elsunersen was well-tolerated with no drug-related serious adverse events or discontinuations reported at doses up to 8 mg. Efficacy was sustained for up to one year in the open-label extension. Additionally, the company announced that the FDA has accepted its New Drug Application (NDA) for relutrigine for the treatment of SCN2A and SCN8A DEEs. The FDA granted the application Priority Review and established a PDUFA target action date of September 27, 2026. This dual update represents a significant regulatory and clinical milestone for the company's precision medicine pipeline in rare epilepsy.