Zogenix Announces Positive Top-line Results from Second Pivotal Phase 3 Clinical Trial of ZX008 in Dravet Syndrome
Primary Endpoint Achieved - Statistically Significant Convulsive Seizure Reduction for ZX008 versus Placebo for Adjunctive Treatment of Seizures
ZX008 Also Demonstrated Statistical Significance in All Key Secondary Endpoints
U.S. and EU Regulatory Submissions on Track for Fourth Quarter of 2018
- Patients taking ZX008 achieved a 54.7% greater reduction in mean monthly convulsive seizures compared to placebo (p<0.001). 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 (>50%) 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.
“These impressive study results show the significant impact the addition of ZX008 made in reducing the burden of convulsive seizures for patients who are not adequately controlled using stiripentol, the standard of care for the treatment of Dravet syndrome in Europe,” said Professor
Secondary endpoints assessed ZX008 compared to placebo in terms of the proportions of patients who achieved ≥50% reductions and ≥75% reductions in monthly convulsive seizures, as well as the median of the longest convulsive seizure-free interval. These results are shown in the following table.
|Patients with ≥50% reduction in
monthly convulsive seizures*
|Patients with ≥75% reduction in
monthly convulsive seizures
|Longest seizure-free interval
*Key secondary endpoints
“Patients with Dravet syndrome can often experience frequent, severe convulsive seizures that dramatically impact quality of life for them and their families,” said
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% (n=42) of patients receiving ZX008 experiencing at least one treatment emergent adverse event compared to 95.5% (n=42) 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% (n=6) of patients in the ZX008 group experiencing at least one treatment emergent serious adverse event compared to 15.9% (n=7) 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.
Prospective cardiac safety monitoring throughout the study did not identify clinical or echocardiographic evidence of cardiac valvulopathy or pulmonary hypertension in any patient. This confirms the observations from Study 1 which also reported no valvulopathy or pulmonary hypertension in any patient. Furthermore, approximately 300 patients are currently enrolled in the ongoing open-label safety extension study (Study 1503), some of whom have been treated with ZX008 on a daily basis for over 2 years. In all studies, no safety signal of any cardiovascular abnormality has been identified to date.
The double blind, placebo controlled, Phase 3 study (Study 1504) randomized 87 patients, with a median age of 9 years (range, 2-19 years), across sites in
ZX008 is designated as an orphan drug in both the U.S. and Europe, and has received Breakthrough Therapy designation in the U.S. for the treatment of Dravet syndrome. Earlier this year,
“I would like to extend my gratitude to the patients, families and investigators involved in Study 1504,” said
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Source: Zogenix, Inc.