Zogenix says primary endpoint achieved in second Phase 3 clinical trial of ZX008
Zogenix (ZGNX) reported positive top-line results from its second confirmatory Phase 3 study for its investigational drug, ZX008, for the treatment of children and young adults with Dravet syndrome.
Dravet syndrome is a rare, catastrophic, lifelong form of epilepsy that begins in the first year of life with frequent and/or prolonged seizures. Previously known as Severe Myoclonic Epilepsy of Infancy (SMEI), it affects one out of 15,700 individuals, 80% of whom have a mutation in their SCN1A gene.
The study results, which are consistent with those reported in Study 1, Zogenix’s first pivotal Phase 3 study, successfully met the primary endpoint and all key secondary endpoints, demonstrating that ZX008, at a dose of 0.5 mg/kg/day, is superior to placebo when added to a stiripentol regimen. Key Findings: Patients taking ZX008 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 ZX008 group compared to 1.2% in placebo patients. ZX008 also demonstrated statistically significant improvement versus placebo in both key secondary measures, including patients with clinically meaningful reductions 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.
Secondary endpoints assessed ZX008 compared to placebo in terms of the proportions of patients who achieved greater than or equal to 50% reductions and greater than or equal to 75% reductions in monthly convulsive seizures, as well as the median of the longest convulsive 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.
The incidence of treatment emergent adverse events was similar in both the treatment and placebo groups, with 97.7% of patients receiving ZX008 experiencing at least one treatment emergent adverse event compared to 95.5% of patients in the placebo group.
The most common adverse events in the ZX008 group were decreased appetite, diarrhea, pyrexia, fatigue, and nasopharyngitis.
The incidence of serious adverse events was similar in both the treatment and placebo groups, with 14% of patients in the ZX008 group experiencing at least one treatment emergent serious adverse event compared to 15.9% of patients in the placebo group. T
wo patients in the ZX008 group had an adverse event leading to study discontinuation compared to one in the placebo group.
ZGNX closed at $46.30, it last traded at $55.30.
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