Praxis Stock Up as Epilepsy Candidate Shows Reduction in Seizures

Core Viewpoint - Praxis Precision Medicines announced positive results from its mid-stage EMBOLD study for relutrigine, leading to a 4.2% increase in share price on September 4 [1] Group 1: EMBOLD Study Results - The phase II EMBOLD study showed a placebo-adjusted reduction of 46% in monthly motor seizures for patients treated with relutrigine [2] - Over 30% of evaluable patients achieved a 28-day seizure-free status while on relutrigine, compared to none on placebo [2] - Patients also reported significant improvements in disruptive behavior, communication, seizure severity, and alertness after 16 weeks of treatment [3] Group 2: Safety and Efficacy - Relutrigine was found to be "generally safe and well tolerated," with no patients requiring dose reduction during the study [5] - Seven patients increased their daily dose to the maximum allowed without adverse effects leading to discontinuation [5] - Adverse events were mostly mild to moderate in severity [5] Group 3: Future Developments - Based on the positive outcomes, Praxis has initiated the registrational phase of the EMBOLD study for SCN2A and SCN8A DEE, with no current approved treatments for these conditions [6] - Relutrigine is a first-in-class investigational drug targeting persistent sodium current, a key driver of seizures in the specified conditions [7] Group 4: Pipeline and Competitors - Praxis is also developing ulixacaltamide for essential tremor, with top-line results expected in the second half of 2024 [8] - Another candidate, PRAX-628, is being evaluated for focal onset seizures, with plans for four studies to support global regulatory registrations [9] - Competitor Xenon Pharmaceuticals is developing therapies for epilepsy, including a selective sodium channel inhibitor currently in phase II studies [10]