Praxis Precision Medicines released another batch of topline data from its epilepsy program on Tuesday, this time from the Phase 2 EMBOLD study investigating relutrigine in SCN2A and SCN8A developmental and epileptic encephalopathy patients.
In the study, participants were 2 to 18 years old with a diagnosis of early-onset developmental or epileptic encephalopathy. Sixteen patients were randomized to the drug, and 13 decided to continue on into the open-label study.
Patients on the drug saw a 46% reduction in countable motor seizures, and caregivers and clinicians also reported improvements in disruptive behavior, communication, alertness and the severity and intensity of seizures. Eight patients completed at least 28 days in the extension arm of the study and saw a median reduction in seizures of 75%, while five patients went 28 days without a seizure compared to none on placebo.
The most common adverse events included infections, vomiting and constipation but none of the patients discontinued treatment due to adverse events.
During an investor call on Tuesday, Praxis president and CEO Marcio Souza said that the trial results “propelled” the company to “initiate an expanded registration cohort aiming to further validate the drug’s benefit and support its journey towards regulatory approval.”
With 5,000 potential patients in the US and no approved treatment, the peak revenue potential for relutrigine is more than $500 million, according to Souza. He said the company plans to meet with regulators by the beginning of next year.
Earlier this year, Praxis touted topline data from a Phase 2a study in photosensitive epilepsy patients. Based on that positive data, the company said it planned to start a focal epilepsy efficacy study for PRAX-628 in the second half of this year. It also has a third epilepsy candidate called PRAX-222 in Phase 1.