Praxis Precision Medicines Inc. (PRAX) released positive topline results from its EMBOLD Phase 2 proof-of-concept study evaluating relutrigine in patients with SCN2A and SCN8A developmental and epileptic encephalopathy (DEE). These rare and severe neurological disorders are characterized by seizures, encephalopathy (brain dysfunction), and intellectual disability.
The study demonstrated a significant reduction in seizures among patients receiving relutrigine. Patients experienced a placebo-adjusted reduction of 46% in countable motor seizures. The study also revealed improvements in other areas related to DEE. For example, caregiver and clinician assessments at week 16 showed improvements in disruptive behavior (29% and 23%, respectively), communication (43% and 31%), seizure severity and intensity (71% and 62%), and alertness (57% and 69%).
Furthermore, the long-term extension of EMBOLD showed encouraging results. Eight patients completed at least one 28-day period, with a median reduction in motor seizures of 75%. Notably, five patients achieved a 28-day seizure-free status while on relutrigine, compared to none on placebo.
Relutrigine was generally safe and well-tolerated during the EMBOLD study. Seven patients increased their daily dose from 0.5 to 1 mg/kg/day during the double-blind period. No patient required a dose reduction. The most common adverse events (AEs) in patients receiving relutrigine were infections, vomiting, pyrexia, somnolence, and constipation. Importantly, no patients discontinued the study due to an AE.
These positive results offer hope for patients with DEE, who currently lack effective treatment options. Praxis Precision Medicines is continuing to advance relutrigine in clinical development, with plans for further studies.
PRAX stock closed at $51.61 on Tuesday, down 2.90%.
