Phase III study of ZX 008 showed robust anti-seizure activity in Dravet syndrome.- Zogenix.

Zogenix announced additional data from analyses of its first Phase III trial (Study 1) of ZX 008 (low-dose fenfluramine hydrochloride), for the adjunctive treatment of seizures associated with Dravet syndrome. Top-line results from Study 1 were previously reported in September 2017.

In the results of a post-hoc analysis evaluating the effect of ZX 008 in enrolled patients in Study 1 who had previously failed treatment with stiripentol, ZX 008 demonstrated robust antiseizure activity in patients. The efficacy and tolerability results in this subgroup were comparable to those achieved in the full Study 1 population. In the post-hoc analysis, patients taking ZX 008 0.8 mg/kg/day achieved a 60.8% greater reduction in mean monthly convulsive seizures compared to placebo (p=0.002). In the full Study 1 population, patients taking ZX 008 0.8 mg/kg/day achieved a 63.9% greater reduction in mean monthly convulsive seizures compared to placebo (p<0.001).>

The post-hoc analysis showed a consistent safety and tolerability profile, as previously seen in the full Phase III trial population. The most common treatment emergent adverse events in this subgroup of patients included decreased appetite, lethargy, fatigue, diarrhea, upper respiratory tract infection, nasopharyngitis, pyrexia, and somnolence. The most common treatment emergent adverse events in the full Study 1 population included diarrhea, vomiting, fatigue, pyrexia, nasopharyngitis, upper respiratory tract infection, fall, weight decreased, decreased appetite, lethargy, seizure, and somnolence. The additional Study 1 results were presented at the Emerging Science session at the 2018 American Academy of Neurology Annual Meeting.