Praxis Precision Medicines said its experimental epilepsy drug vormatrigine failed the main goal of a Phase 2/3 study in adults with focal onset seizures. The company is pausing enrollment in another study while it reassesses the program. Shares fell more than 12 percent in extended trading.
Praxis Precision Medicines' experimental epilepsy drug vormatrigine failed the main goal of a Phase 2/3 study, sending shares down more than 12 percent.
"While the results for POWER1 were not what we hoped for, we are encouraged by the signal we saw on the higher dose arm, the low discontinuation rate and solid safety profile," Chief Executive Officer Marcio Souza said.
The POWER1 study tested vormatrigine in adults with focal onset seizures, a type of epilepsy where seizures begin in one part of the brain. Patients were already taking one to three anti-seizure medications. The drug missed the primary endpoint of reducing monthly seizure frequency from baseline but met a secondary measure where more patients saw at least a 50 percent reduction in seizures. Seizure reduction was more pronounced in the second half of the study when patients received the higher 30 mg dose, suggesting a potential dose-response relationship. Vormatrigine was generally well-tolerated, with fewer than 10 percent of patients discontinuing due to adverse events. About 90 percent of patients from the vormatrigine arm moved into the open-label extension study.
Vormatrigine is a small molecule designed to target the hyperexcitable state of sodium channels in the brain. Praxis was developing it as a once-daily oral treatment for adult focal onset seizures and generalized epilepsy.
The failure is a setback for Praxis, which is pausing enrollment in the ongoing POWER2 study while it reassesses the program. Piper Sandler analyst Yasmeen Rahimi said the pause was the "right call," noting that investors had not been giving much credit to the asset. The company said it continues to focus on preparing for planned launches of its other pipeline candidates, relutrigine and ulixacaltamide.
The decline puts Praxis shares at their lowest level since the announcement. The company's next catalyst will be its reassessment of the vormatrigine program and any potential modifications to the study design, which could determine whether the drug advances or the company shifts resources to its other late-stage candidates.
This article is for informational purposes only and does not constitute investment advice.
