Having already claimed a win for the phase 2 study of its seizure drug, Praxis Precision Medicines is hoping fresh efficacy data will form the backbone of an approval application for the sodium channel inhibitor.
Last year, the Boston-based biotech revealed that the Embold study of the drug, called relutrigine, had hit its primary endpoint of safety. The trial enrolled 16 individuals ages 2 to 18 with early-onset SCN2A-DEE or SCN8A-DEE, forms of epilepsy for which there are no approved treatments.
Praxis has continued with the study, alongside discussions with the FDA about the possibility of using its efficacy results as the basis for an approval application that had been penciled in for early 2026.
Against this backdrop, the company announced yesterday that it has ended the Embold study early on the recommendation of the trial’s data monitoring committee after it was clear relutrigine’s efficacy had been demonstrated.
The company’s stock jumped 30% in premarket trading Friday to $247 per share from a Thursday closing price of $189.97.
“SCN2A and SCN8A DEEs are devastating conditions with extremely high mortality due to the debilitating seizure burden they impose on patients, and there are currently no approved treatment options,” Praxis’ CEO Marcio Souza said in the Dec. 4 post-market release.
“Our progress represents an important milestone towards delivering the first therapy ever designed for these children and their families,” added Souza, who said the company will share the results at the American Epilepsy Society Annual Meeting tomorrow.
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In the same release, Praxis said the FDA has agreed to a meeting “in the coming weeks … to review the data and discuss next steps.”
“Praxis will make a determination on the timing for filing an NDA for relutrigine after the meeting,” the biotech added.
When Praxis rolled out the top-line results in September 2024, the company explained in a presentation that SCN2A and SCN8A are among “the most severe and refractory forms” of developmental and epileptic encephalopathies (DEEs).
There are roughly 5,000 patients in the U.S. with the SCN2A/SCN8A DEEs, representing a market opportunity of about $500 million, the company said at the time.
Praxis launched another trial of relutrigine in patients across all DEEs this year. The hope is that data from the study—which is due to complete in the second half of 2026—would form the basis of a supplemental new drug application. In the September presentation, the company said the peak market opportunity for all developmental epilepsies with a high seizure burden could surpass $2 billion.
Meanwhile, the company scored another midphase epilepsy win this year when its next-generation NaV blocker vormatrigine was linked to a 100% complete response rate in epilepsy patients with photoparoxysmal response, a form of photosensitivity.
