Phase III trial of ZX 008 (low-dose fenfluramine hydrochloride) in Dravet syndrome meets primary endpoints.- Zogenix.

Zogenix reported positive top-line results from its first Phase III trial (Study 1) for its investigational drug, ZX 008 (low-dose fenfluramine hydrochloride), for the treatment of Dravet syndrome. The trial met its primary objective of demonstrating that ZX 008, at a dose of 0.8 mg/kg/day, is superior to placebo as adjunctive therapy in the treatment of Dravet syndrome in children and young adults based on change in the frequency of convulsive seizures between the 6-week baseline observation period and the 14-week treatment period. Patients taking ZX 008 0.8 mg/kg/day achieved a 63.9% reduction in mean monthly convulsive seizures compared to placebo. The median percent reduction in monthly convulsive seizure frequency was 72.4% among ZX008 0.8 mg/kg/day patients compared to 17.4% in placebo patients.

ZX 008 0.8 mg/kg/day also demonstrated statistically significant improvements versus placebo in all key secondary measures, including the proportion of patients with clinically meaningful reductions in seizure frequency and longest seizure-free interval. The same analyses comparing a 0.2 mg/kg/day ZX 008 dose versus placebo also demonstrated statistically significant improvement compared with placebo. ZX008 was generally well-tolerated in this study with the adverse events consistent with the known safety profile of fenfluramine.

The company expects top-line results from its second pivotal Phase III trial, Study 1504, which is nearing full enrollment, in the first half of 2018 and is on track to submit applications for regulatory approvals in the U.S. and Europe in the second half of 2018.