Praxis Precision announces topline results from EMBRAVE Part A trial

Praxis Precision (PRAX) announced topline results from the EMBRAVE Part A trial of elsunersen in pediatric patients with early-seizure onset SCN2A developmental and epileptic encephalopathy. Elsunersen treatment led to a 77% placebo-adjusted seizure reduction from baseline. A total of 57% of patients had at least a 28-day period of seizure freedom. Efficacy was sustained in the OLE for up to one year. All of elsunersen patients improved across sleep, motor function, muscle tone, attention or neuropsychomotor development compared to no improvements in placebo group. Elsunersen was well-tolerated, with no drug-related SAEs, no discontinuations and no neuroinflammation signals at doses up to 8 mg Most treatment-emergent adverse events were mild to moderate. TEAEs were consistent with the EMBRAVE Part 1 study.