Zogenix (ZGNX) : positive results for the treatment of Dravet syndrome

• 9/27:  #15;  vol.  900K

Zogenix reports positive top-line results from its first Phase 3 trial for its investigational drug, ZX008 for the treatment of Dravet syndrome; trial met its primary endpoint

"Dravet syndrome is a rare, but catastrophic form of epilepsy that can be devastating for patients and their families," said Joseph Sullivan M.D., director of the Pediatric Epilepsy Center in UCSF Benioff Children's Hospital San Francisco, and Principal Investigator of Study 1 in the U.S. "These results are truly exciting and demonstrate, in a large multicenter controlled trial, the impressive efficacy of low-dose fenfluramine for patients with Dravet syndrome. If approved, ZX008 could play an important role in treating this devastating condition."

• The trial met its primary objective of demonstrating that ZX008, 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. ZX008 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 ZX008 dose versus placebo also demonstrated statistically significant improvement compared with placebo.
  
• A key secondary endpoint was the same analysis for a comparison of ZX008 0.2 mg/kg/day and placebo. Patients taking ZX008 0.2 mg/kg/day achieved a reduction in mean monthly convulsive seizures of 33.7% compared to placebo (p=0.019). Collectively, these top-line data suggest a dose-response relationship for ZX008 in the adjunctive treatment of convulsive seizures in Dravet syndrome.

** charts before  **

 

** daily **

** monthly **

*****

GW Pharma (GWPH) down 10% after competitor Zogenix (ZGNX) reported positive Phase 3 data for Dravet syndrome (severe myoclonic epilepsy of infancy)

• In August, GWPH said its NDA rolling submission for Dravet and LGS epilepsy indications was under way.