Praxis Precision Medicines Inc. (PRAX) announced topline results from its Phase 2/3 POWER1 study of Vormatrigine in adults with focal onset seizures.
Focal onset seizures (FOS) are a common form of epilepsy, often requiring multiple anti-seizure medications. Patients with highly refractory disease face limited treatment options and ongoing quality-of-life challenges.
The POWER1 trial was a double-blind, randomized, multicenter study evaluating Vormatrigine in adults with FOS who were concurrently taking one to three anti-seizure medications. Patients received either once-daily Vormatrigine (20 mg for six weeks, then 30 mg for six weeks) or placebo for 12 weeks. The primary endpoint measured percent change in monthly seizure frequency from baseline.
The study did not meet its primary endpoint. However, the secondary measure—the 50% responder rate—was achieved, and seizure reduction was more pronounced during the second half of the study at the higher 30 mg dose. Vormatrigine was generally well tolerated, with adverse event-related discontinuations under 10%. Approximately 90% of patients transitioned into and remain enrolled in the open-label extension study.
Marcio Souza, President and CEO of Praxis, stated: "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. We will take some time to review these results to ensure we have the best path forward for developing vormatrigine and the ongoing POWER2 study."
Praxis confirmed it is pausing enrolment in the POWER2 study to reassess the program and determine potential modifications. The company continues to advance preparations for planned launches of relutrigine and ulixacaltamide. Vormatrigine is a next-generation, functionally selective small molecule targeting sodium-channels hyperexcitability in the brain. Preclinical and translational data suggest differentiation from standard of care, with potential best-in-class efficacy for focal epilepsy.
PRAX is trading at $37.19 and $366.52 over the past year. The stock closed Monday's trading at $335.16, down 4.23%. In pre-market trading, the stock is at $310.00, down 7.51%.
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