Phase III study (Study 1504) for ZX 008 meets primary endpoint in Dravet syndrome.- Zogenix.

Zogenix has reported positive top-line results from its second confirmatory Phase III study (Study 1504) for ZX 008 (low-dose fenfluramine hydrochloride), for the treatment of children and young adults with Dravet syndrome. The study results, which are consistent with those reported in Study 1, Zogenix�s first pivotal Phase III study, successfully met the primary endpoint and all key secondary endpoints, demonstrating that ZX 008, at a dose of 0.5 mg/kg/day (maximum 20 mg/day), is superior to placebo when added to a stiripentol regimen.

It showed that patients taking ZX 008 achieved a 54.7% greater reduction in mean monthly convulsive seizures compared to placebo. The median reduction in monthly convulsive seizure frequency was 62.7% in the ZX 008 group compared to 1.2% in placebo patients. ZX 008 also demonstrated statistically significant improvement versus placebo in both key secondary measures, including patients with clinically meaningful reductions (more than 50%) in seizure frequency and longest seizure-free interval. It found that ZX 008 was generally well-tolerated in this study with the adverse events consistent with those observed in Study 1 and the known safety profile of fenfluramine. No patient exhibited cardiac valvulopathy or pulmonary hypertension at any time in the study.

Comment: ZX 008 is designated as an orphan drug in both the U.S. and Europe, and has received Breakthrough Therapy designation in the U.S. for the treatment of Dravet syndrome. Earlier this year, Zogenix conducted a positive meeting with the FDA regarding the ZX 008 clinical development program and planned New Drug Application submission in Dravet syndrome in which FDA affirmed Study 1 and Study 1504 were suitable as the clinical basis for the NDA submission.