Praxis' sodium blocker cuts seizures in 2 epilepsy types in phase 2

Praxis Precision Medicines recently achieved another success in their midphase epilepsy trials, demonstrating the effectiveness of their sodium channel inhibitor in reducing seizures among children suffering from two specific types of epilepsy. This follows their previous midphase success earlier in the year.

The EMBOLD study, conducted by Praxis, involved 16 patients aged between 2 and 18 years. These patients were diagnosed with early-onset SCN2A-DEE or SCN8A-DEE, forms of epilepsy that currently lack approved treatments. They were either administered a placebo or relutrigine, a drug that inhibits persistent sodium current—a major factor in triggering seizure symptoms in these types of epilepsy.

According to a press release from Praxis dated September 3, patients treated with relutrigine experienced an average reduction of 46% in their seizure frequency during the double-blind portion of the study. Additionally, clinicians observed a 23% improvement in disrupted movement by Week 16, a 31% improvement in communication, and a 62% reduction in the severity and intensity of seizures.

A particularly noteworthy outcome was that five patients on relutrigine went without a seizure for a continuous period of 28 days. In contrast, none of the participants in the placebo group achieved this result. The primary goal of the trial was to assess the safety of the drug, and Praxis reported that no patients had to discontinue treatment due to adverse events. The company indicated that relutrigine was "generally safe and well tolerated," with seven patients able to increase their daily dosage from 0.5 mg/kg to 1 mg/kg during the trial. The most commonly reported adverse events included infections, vomiting, pyrexia, drowsiness, and constipation.

Marcio Souza, CEO of Praxis, highlighted the significance of these findings in the release, noting the substantial reduction in seizure frequency from baseline rates. “Seizure freedom is the ultimate goal for patients, and we were humbled by the progress made with relutrigine during the EMBOLD study with over 30% of patients achieving this life-altering milestone,” Souza stated.

Earlier in March, Praxis celebrated another midphase achievement in epilepsy treatment with their next-generation NaV blocker PRAX-628. In that study, a high dose of PRAX-628 was associated with a 100% complete response rate in epilepsy patients who exhibited photoparoxysmal response, a condition related to photosensitivity.

Overall, these promising results from the EMBOLD study and other trials signal significant advances in the development of more effective treatments for epilepsy, offering hope to patients struggling with these severe neurological disorders.