Zogenix Inc. (NASDAQ: ZGNX) saw its shares more than double early on Friday after the company reported positive top-line results from its most recent late-stage trial in the treatment of Dravet syndrome. Considering the stock has more than doubled, it’s obvious this Phase 3 trial was passed with flying colors.
The Phase 3 trial for the investigational drug, ZX008, met its primary objective of demonstrating that it 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 six-week baseline observation period and the 14-week treatment period.
Patients taking ZX008 achieved a 63.9% reduction in mean monthly convulsive seizures compared to a placebo. The median reduction in monthly convulsive seizure frequency was 72.4% among ZX008 patients, compared to 17.4% in placebo patients.
Not only did Zogenix nail its primary endpoint, but ZX008 also demonstrated statistically significant improvements versus the placebo in all key secondary measures, including the proportion of patients with clinically meaningful reductions in seizure frequency and longest seizure-free interval. These included:
- Patients taking ZX008 achieved a reduction in mean monthly convulsive seizures of 33.7% compared to placebo.
- The proportion of patients who achieved ≥50% reductions in monthly convulsive seizures.
- The median of the longest convulsive seizure-free interval.
Joseph Sullivan M.D., director of the Pediatric Epilepsy Center in UCSF Benioff Children’s Hospital San Francisco, and Principal Investigator of Study 1 in the United States, commented:
Dravet syndrome is a rare, but catastrophic form of epilepsy that can be devastating for patients and their families. These results are truly exciting and demonstrate, in a large multicenter controlled trial, the impressive efficacy of low-dose fenfluramine for patients with Dravet syndrome. If approved, ZX008 could play an important role in treating this devastating condition.
Shares of Zogenix closed Thursday down about 8% at $12.88, with a consensus analyst price target of $24.67 and a 52-week range of $7.50 to $16.50. Following the announcement the stock was up more than 150% at $32.25 in early trading indications Friday.