EMERYVILLE, Calif.—Zogenix Inc., a pharmaceutical company developing therapies for the treatment of rare central nervous system (CNS) disorders, in July reported positive top-line results from its second confirmatory Phase 3 study (Study 1504) for its investigational drug, ZX008 (low-dose fenfluramine hydrochloride), for the treatment of children and young adults with Dravet syndrome.
According to Zogenix, the study successfully met the primary endpoint and all key secondary endpoints, demonstrating that ZX008, at a dose of 0.5 mg/kg/day (maximum 20 mg/day), is superior to placebo when added to a stiripentol regimen. The results are consistent with those reported in Study 1, Zogenix’s first pivotal Phase 3 study, the company adds.
Keys finding include:
- Patients taking ZX008 achieved a 54.7-percent greater reduction in mean monthly convulsive seizures compared to placebo. The median reduction in monthly convulsive seizure frequency was 62.7 percent in the ZX008 group, compared to 1.2 percent in placebo patients.
- ZX008 also demonstrated statistically significant improvement vs. placebo in both key secondary measures, including patients with clinically meaningful reductions (more than 50 percent) in seizure frequency and longest seizure-free interval.
- ZX008 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.
“These impressive study results show the significant impact the addition of ZX008 made in reducing the burden of convulsive seizures for patients who are not adequately controlled using stiripentol, the standard of care for the treatment of Dravet syndrome in Europe,” said Dr. Rima Nabbout of the Department of Pediatric Neurology, Reference Center for Rare Epilepsies at Necker Enfants Malades Hospital, who was the principal investigator of Study 1504. “If approved, ZX008 has the potential to be a transformative treatment for Dravet syndrome, a rare and serious form of epilepsy with few available treatment options.”