Zogenix Announces Presentation of New Efficacy and Safety Data from its First Pivotal Phase 3 Clinical Trial of ZX008 in Dravet Syndrome
Efficacy and Safety Data in Patients on ZX008 Who Previously Failed Stiripentol Treatment Prior to Entry into First Phase 3 Trial (Study 1) Comparable to Results from Full Study 1 Population
Patients on ZX008 Also Achieved Significant, Clinically Meaningful Reduction in Total Seizure Frequency Compared to Those on Placebo
Data Presented at 2018
As previously reported, Study 1 met its primary objective of demonstrating that ZX008, at a dose of 0.8 mg/kg/day, is superior to placebo as an 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 (p<0.001).
The first poster, presented by
“It is highly encouraging to see that the efficacy and tolerability in this subgroup of patients who had previously failed treatment with stiripentol was comparable to the full Study 1 population,” said
In this subgroup of patients, ZX008 demonstrated robust antiseizure activity in patients who failed prior treatment with stiripentol. 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 ZX008 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 ZX008 0.8 mg/kg/day achieved a 63.9% greater reduction in mean monthly convulsive seizures compared to placebo (p<0.001). Other select endpoint comparisons are presented below.
|Subgroup Population: Subjects
Who Failed Treatment with
Stiripentol Prior to Study 1
|Full Study 1 Population|
|ZX008 0.8 mg/kg/day
|ZX008 0.8 mg/kg/day
|Patients with ≥50% reduction in monthly convulsive seizures||73%
|Patients with ≥75% reduction in monthly convulsive seizures||50%
|Longest seizure-free interval (median)||24.5 days
|9 days||20.5 days
|% rated as Much Improved or Very Much Improved on CGI-C* Rated by Parents / Caregivers||41%
|% rated as Much Improved or Very Much Improved on CGI-C* Rated by Investigator||64%
* Clinical Global Impression (CGI-C) of Improvement indicates rating of very much improved or much Improved in overall condition.
The post-hoc analysis showed a consistent safety and tolerability profile, as previously seen in the full Phase 3 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.
As reported for the full Study 1 population, there was no clinical and/or echocardiographic evidence of cardiac valvulopathy or pulmonary hypertension in any patient at any time during the study. No patient discontinued participation or required a change in monitoring in the study due to cardiac factors in this subgroup or the full Study 1 population.
A second poster presented by
“The efficacy and safety data generated from ZX008 in Study 1 continue to be extremely compelling,” said
ZX008 is designated as an orphan drug in both the U.S. and
About Study 1
The randomized, double blind, placebo controlled, Phase 3 study enrolled 119 patients across sites in the U.S.,
Forward Looking Statements
646-597-6987 | Andrew@lifesciadvisors.com
858-449-9575 | email@example.com
Source: Zogenix, Inc.