Zogenix, Inc has announced the top-line results from a Phase 3 study of the investigational drug ZX008 (low-dose fenfluramine hydrochloride) for the treatment of young patients with Dravet syndrome. The source article can be read here, at Globe Newswire.
About Dravet Syndrome
Dravet syndrome is a type of epilepsy, and people with the condition typically experience frequent seizures, and may also have a range of other conditions, including speech impairment, developmental delays, and sleep disturbances, amongst others.
Dravet syndrome has been linked to genetic changes that affect the sodium ion channel, which controls the movement of sodium in to and out of cells and is important for electrical neurone signalling.
About the Study
The Phase 3 clinical trial (Study 1504) involved 87 patients between the ages of two and nineteen. Of these patients, 43 were assigned to the group that received the experimental drug ZX008, and 44 were assigned to a placebo group. Both groups also took stiripentol and other antiepileptic drugs. The study took place at sites across Europe, the US, and Canada, and treatment lasted for around fifteen weeks.
The study reached its primary goal of significantly reducing the number of convulsive seizures that patients taking ZX008 experienced compared to those taking the placebo. The study also found statistically significant results in other measures.
The study found that, compared to the placebo group, the group taking ZX008 had a 54.7% larger reduction in their average (mean) monthly convulsive seizures. In addition, patients taking ZX008 also showed less frequent seizures and longer seizure-free intervals compared to the placebo group.
The safety profile of the experimental drug was also similar to what had been found in a previous study of it, and the safety profile of fenfluramine. The most common adverse events in patients taking ZX008 were reduced appetite, fatigue, diarrhoea, pyrexia (a fever), and nasopharyngitis (a cold). Patients were also monitored for signs of cardiac valvulopathy (a condition that affects the heart valves) and pulmonary hypertension (high blood pressure linked to the lungs) throughout the study, but no patient showed signs of these conditions.