Rare Daily Staff
Zogenix reported that its second late-stage trial of its experimental drug ZX008 met its primary endpoint as a treatment for Dravet syndrome, a rare and serious form of epilepsy.
The study showed ZX008 was superior to a placebo as adjunctive therapy to stiripentol in the treatment of children and young adults with Dravet syndrome. The results were consistent with those reported in a previous pivotal late-stage study.
Patients taking ZX008 achieved more than a 54 percent greater reduction in mean monthly convulsive seizures compared to placebo. The median reduction in monthly convulsive seizure frequency was nearly 63 percent in the ZX008 group compared to a little more than 1 percent in placebo patients.
ZX008 also demonstrated statistically significant improvement versus placebo in both key secondary measures, including patients with clinically meaningful reductions of 50 percent or more in seizure frequency and longest seizure-free interval.
ZX008 was generally well tolerated. The incidence of serious adverse events was similar in both the treatment and placebo groups, with 14 percent of patients in the ZX008 group experiencing at least one treatment emergent serious adverse event compared to nearly 16 percent of patients in the placebo group. Two patients in the ZX008 group had an adverse event leading to study discontinuation compared to one in the placebo group.
ZX008 is designated as an orphan drug in both the United States and Europe and has received Breakthrough Therapy designation in the United States for the treatment of Dravet syndrome.
“Based on these highly compelling top-line results from both of our pivotal studies, we are now focused on submitting applications for regulatory approvals in the U.S. and Europe in the fourth quarter of 2018,” said Stephen Farr, president and CEO of Zogenix. “We are excited about ZX008’s potential to have a major impact in the treatment of patients with Dravet syndrome and their families.”
July 12, 2018
Photo: Stephen Farr, president and CEO of Zogenix