Praxis Precision Medicines, Inc. (NASDAQ:PRAX) stock experienced a volatile trading session on Monday, after the company announced positive topline results from its Phase 2 RADIANT study evaluating vormatrigine in patients with focal onset seizures and generalized epilepsy.
The stock initially surged on the news, but then reversed course and is currently trading down approximately 9%.
The central nervous system (CNS) disorders-focused company said the topline results from the Phase 2 RADIANT study included data from 37 patients.
“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,” said Marcio Souza, president and CEO of Praxis.
In an investor presentation on the company website, Praxis noted that the trial showed a median seizure reduction of around 56.3%, with 60% of the patients achieving at least a 50% reduction in seizures.
This positive outcome has encouraged the company to move forward with a Phase 2/3 trial, even though 23% of patients discontinued the study.
54% of patients achieved at least a 50% seizure reduction threshold in Week 1 and 67% in Week 8. In the last month of the dataset, 22% of the patients experienced a 100% reduction in seizure frequency.
The company added that most adverse events were mild to moderate and transient. All severe and serious adverse events (AEs) were recovered and resolved.
The investor presentation noted that the investigators had the option to reduce the dose of the background medication to manage AEs; when done (6 patients), no discontinuation was observed.
The company said it is on track to complete the pivotal, 12-week POWER1 study in the fourth quarter of 2025 and, based on the results from RADIANT, it expects to initiate the POWER2 study shortly.
On Monday, the company reported cash and investments of approximately $447 million and maintains a cash runway into 2028.
In July, the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation for Praxis Precision’s relutrigine, a sodium channel functional state modulator for pediatric use for SCN2A and SCN8A developmental and epileptic encephalopathies (DEEs).
The EMBOLD cohort 2 pivotal trial is on track for topline results in the first half of 2026, with NDA filing to follow.
