Zogenix announces Phase 3 Fintepla data published
Zogenix announced that JAMA Neurology has published the results of Zogenix's Phase 3 study of the investigational drug, Fintepla, in Dravet syndrome patients whose antiepileptic drug treatment regimens included stiripentol but who were still experiencing a high number of convulsive seizures. Dravet syndrome is a rare, severe and difficult to treat infantile-onset epilepsy characterized by frequent, disabling seizures. The study demonstrated that adding Fintepla to these patients' treatment regimens led to a significant and clinically meaningful reduction in monthly convulsive seizure frequency, or MCSF. Study 1504 was an international, double-blind, placebo-controlled Phase 3 study of 87 Dravet syndrome patients age 2-19 taking background anti-epileptic drug regimens that included stiripentol, randomized to placebo or Fintepla 0.4 mg/kg/day. The study was conducted at 28 centers in Canada, France, Germany, the Netherlands, Spain, the United Kingdom and the United States. Eligible patients in the trial were experiencing seizures that were poorly controlled with their current anti-seizure medications consisting of stiripentol plus clobazam and/or valproic acid. After a 6-week period to establish baseline seizure frequency, patients were randomized to receive Fintepla starting at a dose of 0.2 mg/kg/day, twice-daily with gradual blinded titration over a three week period to 0.4 mg/kg/d over three weeks. Patients maintained their regimen for an additional 12 weeks at a stable dose, then either continued treatment in an open-label extension study or discontinued treatment. The study met its primary efficacy endpoint and all key secondary endpoints. Patients treated with Fintepla achieved a 54% greater reduction in mean MCSF than those receiving the placebo. Additionally, 54% of patients treated with Fintepla experienced a clinically meaningful reduction in MCSF versus 5% with placebo. Profound seizure reduction was experienced by 35% of Fintepla-treated patients compared to 2% with placebo. The median longest seizure-free interval was 22 days with Fintepla and 13 days with placebo. In the study, Fintepla was generally well-tolerated and demonstrated a safety profile consistent with the findings of Zogenix's first Phase 3 study of Fintepla in Dravet syndrome, called Study 1, as well as with findings from an analysis of the company's ongoing open-label extension study. The most common adverse events in Study 1504 were decreased appetite, fatigue and pyrexia. Across all three studies, no patient exhibited clinical or echocardiographic evidence of valvular heart disease or pulmonary arterial hypertension.