Zogenix (ZGNX) announced new data from its first Phase 3 trial (Study 1) of its investigational drug, ZX008 (low-dose fenfluramine hydrochloride), for the treatment of Dravet syndrome, a type of epilepsy.
Top-line results from Study 1 were previously reported in September 2017. The updated Study 1 results, as well as additional data supporting the further investigation of ZX008 in refractory epilepsies, were presented at the 71st American Epilepsy Society (AES) Annual Meeting, taking place this week in Washington, D.C.
The new Study 1 results presented at AES showed the odds of achieving a clinically meaningful (=50%) or substantial (=75%) reduction in convulsive seizure frequency were 29 and 50 times higher, respectively, among patients treated with ZX008 0.8 mg/kg/day than in patients treated with placebo. The study also measured improvement on the Clinical Global Impression (CGI-C) rating. Fifty-five percent of patients treated with ZX008 0.8 mg/kg/day were rated by parents/caregivers as very much improved or much improved in overall condition on the CGI-C compared to 10% of the placebo group and 62.5% of patients treated with ZX008 0.8 mg/kg/day were rated by investigators as very much improved or much improved in overall condition on the CGI-C compared to 10% of the placebo group.
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).
Zognix CEO Stehpen Farr said "The overall data to date reinforce the potential of ZX008 to be an important new treatment for seizure control in children and young adults with Dravet syndrome, and we look forward to sharing top-line results from our second pivotal Phase 3 trial, Study 1504, in the second quarter of 2018."
Today's sell-off may be due to concerns around potential safety issues, but that is uncelar. The company said 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.
The company is hosting a call with Pediatric Epilepsy expert Joseph Sullivan, M.D. on Dravet Syndrome and ZX008 today at 4:30 PM ET.
ZGNX surged over 200% after the positive data from Study was originally announced in late September. The stock had been trading in a narrow range in between the upper-$30s and lower $40s since then.
Zogenix plans to initiate a Phase 3 clinical trial of ZX008 in children and adults with Lennox-Gastaut Syndrome (a different type of epilepsy) during fourth quarter of 2017.
Zogenix has ~$300 million in cash after selling 6.7 million shares in an offering in early October. The company has an enterprise value of $1.1 billion and 14% of the 28 million share float is sold short.