Sign in Subscribe
Anticonvulsants

Praxis Precision Medicines Sees Encouraging Results from Midphase Trial of Vormatrigine

Strategic Insights

— 1 min read

In a midphase trial, Praxis Precision Medicines observed a 56.3% median reduction in seizure frequency from baseline, a promising sign for the company's sodium-channel modulator vormatrigine in treating epilepsy. The Radiant study involved 37 patients with focal onset seizures who received vormatrigine, with 23% of patients discontinuing due to adverse events or lack of background therapy dose adjustment, despite protocol guidance. However, the majority of patients who continued with the treatment saw significant improvements, with 60% experiencing at least a 50% reduction in seizure frequency over eight weeks. The company plans to start a monotherapy trial in the first half of 2026 and is set to initiate a follow-up study to the Radiant trial, enrolling a larger patient population.

Key Takeaways:

  • The Radiant study demonstrated a 56.3% median reduction in seizure frequency from baseline for patients receiving vormatrigine, a significant finding for the treatment of epilepsy.
  • Despite the high discontinuation rate of 23%, the majority of patients who continued with the treatment saw significant improvements, with 60% experiencing at least a 50% reduction in seizure frequency over eight weeks.
  • Vormatrigine was generally well-tolerated, with adverse events and discontinuations "avoided entirely" in six instances where investigators proactively reduced background treatment.
  • The company observed consistent efficacy across subgroups of patients on different background therapies, a strength of the study as highlighted by Steven Petrou, Ph.D., chief scientific officer at Praxis.
  • Praxis plans to start a monotherapy trial in the first half of 2026 and is set to initiate a follow-up study to the Radiant trial, enrolling a larger patient population to receive placebo or one of three doses of vormatrigine.
  • During the trial, the efficacy of vormatrigine was not impacted by the dialing back of other medications, as stated by Praxis CEO Marcio Souza.

Statistics:

  • 56.3% median reduction in seizure frequency from baseline for patients receiving vormatrigine.
  • 23% of patients discontinued the study due to adverse events or lack of background therapy dose adjustment.
  • 60% of patients experienced at least a 50% reduction in seizure frequency over eight weeks.
  • 54% of patients hit the 50% threshold in Week 1.
  • 22% of patients experienced a 100% reduction in seizure frequency in the last month of the dataset.
  • More than 80% of participants were taking a sodium channel blocker.
  • 30% of participants were on SK Life Science’s Xcopri.

Sources:

  • Praxis Precision Medicines (PDF)
  • August 4 release (no date specified)

Read more