JAMA Neurology Publishes Phase 3 Study of Zogenix Investigational Drug FINTEPLA® in Dravet Syndrome Patients Taking Stiripentol-Containing Antiepileptic Drug Regimens
- Addition of FINTEPLA to stiripentol-inclusive regimens provided a 54% greater reduction in study patients’ mean monthly convulsive seizure frequency compared to placebo
- A significantly greater proportion of study patients taking FINTEPLA compared to placebo experienced clinically meaningful (≥ 50%) or profound (≥ 75%) seizure reduction
- Patients in the FINTEPLA treatment group also experienced significantly longer periods of seizure freedom
“Because it is common for physicians to use stiripentol for their Dravet syndrome patients, it was important to evaluate the benefit and tolerability of adding FINTEPLA to a stiripentol-inclusive treatment regimen in those patients who were still experiencing frequent convulsive seizures,” said study author, Professor
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 (n=44) or FINTEPLA 0.4 mg/kg/day (n=43)*. The study was conducted at 28 centers in
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 (95% CI, 35.6%-67.2%; p<0.001). Additionally, 54% of patients treated with FINTEPLA experienced a clinically meaningful (>50%) reduction in MCSF versus 5% with placebo (p<0.001). Profound seizure reduction (>75% reduction in MCSF) was experienced by 35% of FINTEPLA-treated patients compared to 2% with placebo (p=0.003). The median longest seizure-free interval was 22 days (3.0-105.0) with FINTEPLA and 13 days (1.0-40.0) with placebo (p=0.004).
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 (Study 1503). The most common adverse events in Study 1504 were decreased appetite (19 patients taking fenfluramine [44%] vs 5 taking placebo [11%]), fatigue (11 [26%] vs 2 [5%]), diarrhea (10 [23%] vs 3 [7%]), and pyrexia (11 [26%] vs 4 [9%]). Across all three studies, no patient exhibited clinical or echocardiographic evidence of valvular heart disease or pulmonary arterial hypertension.
“Given the devastating and lifelong impacts associated with the frequent convulsive seizures experienced by Dravet syndrome patients, we are very much encouraged by the results of this study in patients still having many seizures on a stiripentol-containing medication regimen and by its publication in JAMA Neurology,” said
Zogenix is a global pharmaceutical company committed to developing and commercializing transformative therapies to improve the lives of patients and their families living with rare diseases. The company has two late-stage development programs underway: FINTEPLA® (ZX008, fenfluramine oral solution) for the treatment of seizures associated with Dravet and Lennox-Gastaut syndromes, two rare and often-catastrophic childhood-onset epilepsies, and MT1621, a novel substrate enhancement therapy for the treatment of a rare genetic disorder called TK2 deficiency. The company’s New Drug Application for FINTEPLA for Dravet syndrome has been accepted for review by the
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Source: Zogenix, Inc