New phase III results with Fintepla (ZX 008) in Dravet Syndrome presented.- Zogenix.

Zogenix announced that data will be presented on the use of Fintepla (low-dose fenfluramine), in children and young adults with Dravet syndrome. Results from the phase III Study 1504 is a follow-up to top-line results that were released in July 2018. Patients (n=87) in Study 1504 were taking a background anti-epileptic drug (AED) medication regimen that included stiripentol and were randomized to placebo (n=44) or FINTEPLA 0.5 mg/kg/day (n=43). Consistent with Study 1, Study 1504 met the primary endpoint and all key secondary endpoints. Results demonstrated the statistically significant efficacy of Fintepla when added to a stiripentol regimen in children and young adults with Dravet syndrome. Children and young adults treated with Fintepla achieved a 54.0% greater reduction in mean monthly convulsive seizures compared to placebo. The median reduction in monthly convulsive seizure frequency was 63.1% in the Fintepla group compared to 1.1% in placebo patients. The Study 1504 results showed the odds of achieving a clinically meaningful (at least 50%) or substantial (at least 75%) reduction in convulsive seizure frequency were 26 and 24 times higher, respectively, among patients treated with Fintepla 0.5 mg/kg/day than in patients treated with placebo. The study also demonstrated statistically significant differences in seizure-free intervals, with a median longest seizure-free interval of 22 days in patients treated with Fintepla 0.5 mg/kg/day vs.13 days for patients in the placebo group (p=0.004). The incidence of serious adverse events was similar in both the treatment and placebo groups.

In addition, data from Study 1503 that focuses on effectiveness and overall tolerability of Fintepla and on the long-term cardiovascular assessments and observations were presented. A total of 232 patients in Study 1503 were included in the interim analysis of the ongoing OLE trial. A total of 22 (9.5%) patients discontinued treatment: lack of efficacy (16), subject withdrawal (2), adverse event (1), Sudden Unexpected Death in Epilepsy (SUDEP) (1), physician decision (1), and withdrawal by caregiver (1). More than 90% of patients remained in the study. The median percent reduction in monthly convulsive seizure frequency over the entire OLE treatment period was 66.8% (compared with baseline frequency established in the core Phase III studies). Over the same period, 64.4% of children and young adults demonstrated a greater than 50% reduction in convulsive seizure frequency and 41.2% demonstrated a greater than 75% reduction. Reductions in convulsive seizure frequency was noted at the first timepoint measured, one month, and continued over the entire treatment period analysed.

The occurrence of adverse events was consistent with the Phase III placebo-controlled studies.The most common adverse events occurring in more than 10% of children and young adults were pyrexia (22%), nasopharyngitis (20%), decreased appetite (16%), influenza (12%), diarrhea (11%), and upper respiratory tract infection (10%). The data will be presented at the 72nd American Epilepsy Society (AES) Annual Meeting.