Praxis Precision Medicines (NASDAQ:PRAX) has announced positive topline results from its EMBOLD Phase II proof of concept study evaluating relutrigine in SCN2A and SCN8A developmental and epileptic encephalopathy patients.
The study results indicated that relutrigine was generally safe and well tolerated by patients. A placebo-adjusted monthly motor seizure reduction of 45% was reported during the double-blind period, and more than 30% of patients achieved seizure freedom status while on relutrigine. Observations also showed meaningful improvements in alertness, communication, and seizure severity, as well as a 75% reduction in median seizure rate for patients in the long-term extension.
“We are thrilled to see the combination of positive efficacy and tolerability of relutrigine for SCN2A and 8A, where there are no approved treatments. When comparing to the baseline rates, patients in EMBOLD had over 2,000 fewer seizures since the beginning of the study. This kind of remarkable impact keeps us focused on advancing our programs,” said Marcio Souza, president and CEO of Praxis. “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. Most importantly, we are grateful to the patients, families, caregivers and clinicians who participated in the trial.”
