Praxis Reports Promising Phase 2 Data From RADIANT Trial: Vormatrigine Shows Strong Efficacy And Tolerability In Focal Epilepsy

Praxis Precision Medicines, Inc. has announced topline results from its Phase 2 RADIANT study evaluating vormatrigine, a promising therapy for adults with focal onset seizures. Vormatrigine is a next-generation, orally administered small molecule that selectively targets hyperexcitable sodium channels in the brain, aiming to restore neuronal balance in central nervous system (CNS) disorders.

“We are thrilled by the best-in-disease topline results of this first cohort for the RADIANT study and for the potential to deliver a fast-acting, highly efficacious and tolerable therapy to patients living with focal epilepsy. Many of the over three million patients living with common epilepsy in the U.S. need to manage multiple anti-seizure medications, and vormatrigine is the only drug in development aiming to become first line and address the market at large,” said Marcio Souza, president and chief executive officer of Praxis.

He also added, “These findings build on our earlier clinical data showing a differentiated profile for vormatrigine as a fast-acting, no-titration, once-daily oral drug with no requirement to be taken with food, and a favorable DDI profile, all of which are unseen in ASMs currently in the market or in development. We are well on track to complete the pivotal, 12-week POWER1 study in Q4 and, based on the results from RADIANT, expect to initiate the POWER2 study shortly. We are deeply grateful to the patients, caregivers and clinicians who are contributing to the vormatrigine trials.” 

Unlike standard treatments, vormatrigine’s functional selectivity allows it to focus on disease-specific sodium channel hyperactivity without broadly affecting normal neuronal activity. Preclinical studies have shown its superior selectivity and significant efficacy in the maximal electroshock seizure (MES) model, one of the most predictive preclinical models for focal epilepsy treatment success.

“While several sodium channel therapies are already used to treat focal onset seizures, there remains substantial room for improvement within this class. A next-generation sodium channel blocker has the potential to represent an important step forward in addressing that gap. I'm encouraged by the initial results of the RADIANT study and hopeful that, in the future, we may be able to offer patients another effective treatment option,” said Jacqueline French, MD, Professor at NYU Langone Health's Comprehensive Epilepsy Center.

The initial patient cohort from the RADIANT study demonstrated a strong reduction in seizure frequency, along with a safety and tolerability profile consistent with clinical expectations. These early findings suggest vormatrigine could become a best-in-class treatment for focal epilepsy if further validated in larger trials.