Zogenix Announces New Positive Efficacy and Safety Data from Pivotal Phase 3 Clinical Trial of ZX008 in Dravet Syndrome
ZX008 Patients More Likely to Achieve Clinically Meaningful Reduction in Seizure Frequency Compared to Those on Placebo
Parents/Caregivers and Investigators Rated Patients Treated with ZX008 as Very Much Improved or Much Improved in Overall Condition Compared to Placebo
Company to Host Callwith Pediatric Epilepsy Expert Joseph Sullivan, M.D.,
on Dravet Syndrome and ZX008, Today at
The new Study 1 results presented at
“Dravet syndrome is a rare form of intractable epilepsy for which a significant unmet medical need currently exists,” said
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 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). Patients taking ZX008 0.8 mg/kg/day achieved a 63.9% reduction in mean monthly convulsive seizures compared to placebo (p<0.001). The median percent reduction in monthly convulsive seizure frequency was 72.4% among ZX008 0.8 mg/kg/day patients, compared to 17.4% in placebo patients.
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). ZX008 0.8 mg/kg/day and ZX008 0.2 mg/kg/day also demonstrated statistically significant improvements versus placebo in additional key secondary measures, including the proportion of patients with clinically meaningful reductions in seizure frequency and longest seizure-free interval.
The most common treatment emergent adverse events (>10% in any treatment group) in Study 1 include diarrhea, vomiting, fatigue, pyrexia, nasopharyngitis, upper respiratory tract infection, fall, weight decreased, decreased appetite, lethargy, seizure and somnolence. Prospective cardiac safety monitoring throughout the study demonstrated trace regurgitation on mitral or aortic valves were recorded on at least one echocardiogram in >10% of subjects among all three treatment groups, placebo included. There was no clinical or echocardiographic evidence of cardiac valvulopathy or pulmonary hypertension. No patient discontinued participation or required a change in monitoring in the study due to cardiac factors.
“Our confidence in the potential of ZX008 as an effective treatment for seizures associated with Dravet syndrome continues to strengthen with the new data showing that both caregivers and clinicians perceived patients’ overall condition to be very much or much improved following treatment with ZX008,” said
Additional posters supporting the continued investigation of ZX008 in refractory epilepsies were also presented in the main exhibit hall at the
Data were also presented from a drug-drug interaction study assessing the pharmacokinetics (PK) and safety of ZX008 administered with and without a combination regimen of stiripentol, clobazam and valproic acid. Lastly, three posters were presented from studies on the caregiver burden and pharmacoeconomic impact related to severe childhood epilepsies. These data demonstrated that Dravet syndrome caregivers face substantial physical, emotional and time burdens, including elevated levels of anxiety/depression and financial burden.
ZX008 is designated as an orphan drug in both the U.S. and
Conference Call Details
To access the call with pediatric epilepsy expert
Conference ID: 7828122
Audio replays available through
Replay PIN: 7828122
About Study 1
The randomized, double blind, placebo controlled, Phase 3 study enrolled 119 patients across sites in the U.S.,
Forward Looking Statements
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Source: Zogenix, Inc.