September 29, 2017
By Alex Keown, BioSpace.com Breaking News Staff
EMERYVILLE, Calif. – Shares of Zogenix, Inc. have more than doubled since the stock market’s opening bell this morning following the company announcement its investigational Dravet syndrome drug met its primary endpoints in cutting seizure rates in juvenile patients.
Dravet syndrome, also known as Severe Myoclonic Epilepsy of Infancy, is a rare form of epilepsy that is characterized by frequent and prolonged seizures. Zogenix’ investigational drug, ZX008, a low-dose of fenfluramine hydrochloride, was able to reduce the frequency of convulsive seizures in trial patients.
With the positive trial data, Zogenix is looking at filing for regulatory approval with the U.S. Food and Drug Administration in the second half of 2018. The company is awaiting results from a second Phase III trial and expects top-line results in the first half of 2018.
The positive trial results certainly pleased investors who sent share prices skyrocketing this morning. Prices rose from Thursday’s close of $12.40 per share to a high of $31.55 this morning. Prices have settled back a bit to $28.65 as of 11:18 a.m. In pre-market trading, prices went even higher, hitting $40, Reuters reported.
Stephen Farr, president and chief executive officer of Zogenix, praised the top-line efficacy and safety results from the study. He said the data reinforced the potential of “ZX008 to be an important new treatment for seizure control in children with Dravet syndrome.” Farr said the company plans to present additional data in publications and at medical conferences.
In the Phase III trial, ZX008 proved superior to placebo as an adjunctive therapy based on change in the frequency of convulsive seizures between the 6-week baseline observation period and the 14-week treatment period. Patients in the trial experienced approximately 40 seizures per month. Those taking ZX008 saw a 63.9 percent reduction in mean monthly convulsive seizures compared to placebo, Zogenix said. Additionally, Zogenix said the drug met secondary endpoints “with clinically meaningful reductions in seizure frequency and longest seizure-free interval.” The median percent reduction in monthly convulsive seizure frequency was 72.4 percent for ZX008 patients compared to 17.4 percent in placebo patients.
The median age of the 119 patient trial was eight years old.
Zogenix said ZX008, which is a mild dose of the controversial obesity drug Phen fen, was well tolerated in the trial.
ZX008 is designated as an orphan drug in both the U.S. and Europe, and has received Fast Track designation in the U.S. for the treatment of Dravet syndrome, the company said.
Zogenix’ Dravet syndrome drug is well ahead of Ovid Therapeutics and Takeda Pharmaceuticals ’ TAK-935, a novel, potent and highly selective CH24H inhibitor. The drug moved into a Phase Ib/IIa study earlier this year.
In addition to studying ZX008 in Dravet syndrome, Zogenix is also exploring the drug’s impact on another form of childhood epilepsy, Lennox Gastaut Syndrome. In June, the FDA granted orphan drug designation to ZX008 for this designation.
