Praxis AES Wrap-up: Best-in-Class Potential Across Rare Pediatric and Adult Epilepsies

Core Insights - Praxis Precision Medicines presented significant clinical updates at the American Epilepsy Society Annual Meeting, highlighting the potential of relutrigine and vormatrigine in treating epilepsy [2][3] Group 1: Relutrigine for Developmental and Epileptic Encephalopathies (DEEs) - The EMBOLD study demonstrated a 53% placebo-adjusted reduction in seizures (p < 0.0002) and a 66% increase in motor seizure-free days (p = 0.034) [8] - Relutrigine was well-tolerated, showing rapid and significant seizure reduction over time, with broad functional improvements in behavior, alertness, communication, and overall status [4][8] - The company plans to meet with the FDA to discuss the next steps for the New Drug Application (NDA) [4] Group 2: Vormatrigine for Focal Onset Seizures (FOS) and Generalized Epilepsy - The RADIANT study indicated a 54% median reduction in seizures for patients taking vormatrigine over 8 weeks, with 58% achieving at least a 50% reduction in week 1, increasing to 61% by week 8 [9] - FOS patients reached a 100% median weekly seizure reduction after 8 weeks, maintained through 16 weeks, with over 11% experiencing seizure freedom for the entire 8-week period [9] - Vormatrigine is positioned as a best-in-class therapy with fast-acting efficacy, sustained reduction, and favorable safety profiles [5] Group 3: Company Commitment and Future Studies - The company is committed to accelerating treatment options for patients and advancing the broader DEE study, EMERALD, along with ASO programs and FOS studies toward registration [3] - Praxis has completed recruitment for the POWER1 pivotal study in FOS and is on track to complete the POWER2 study in the second half of 2026, with the POWER3 study set to begin in the first half of 2026 [10]