Praxis Precision receives FDA Breakthrough Therapy Designation for relutrigine

Praxis Precision (PRAX) Medicines announced that the U.S. Food and Drug Administration has granted BTD for relutrigine, a sodium channel functional state modulator for pediatric use for the treatment of patients with SCN2A and SCN8A DEEs. The BTD enables expedited development and regulatory review for drugs that are intended to treat a serious condition, and preliminary clinical evidence indicates that relutrigine may demonstrate substantial improvement on a clinically significant endpoint over existing therapies. The BTD for relutrigine was supported by the positive data from cohort 1 in the Phase 2 EMBOLD study, as well as 11-month data from the open-label extension period of the trial. Data from the double-blind period showed a placebo-adjusted monthly motor seizure reduction of 46% during the double-blind period and seizure freedom in over 30% of patients while on relutrigine. Additionally, meaningful gains were observed in alertness, communication and seizure severity as noted by both clinicians and caregivers. In May 2025, Praxis provided a data update from the ongoing OLE part of the trial during a virtual investor event. At month 11, an average of approximately 90% seizure reduction was observed in patients, and a sustained and continuous improvement in seizure-free periods was observed, with a mean of 67 days without seizures compared to 3 days in the baseline period. There were no new safety signals, drug related serious adverse events or dose reductions needed. The EMBOLD registrational cohort 2 is currently ongoing and continues to enroll, with topline results expected no later than the first half of 2026, followed by a potential NDA filing. Praxis has recently initiated the EMERALD study evaluating relutrigine in patients across all DEEs. EMERALD is a registrational, 16-week, placebo-controlled study evaluating seizure reduction in patients diagnosed with developmental epilepsies.