Positive phase III trial of Epidiolex to treat tuberous sclerosis complex.- GW Pharma

GW Pharmaceuticals plc and its U.S. subsidiary Greenwich Biosciences Inc., the world leader in the science, development, and commercialization of cannabinoid prescription medicines, announced positive top-line results of a randomized, double-blind, placebo-controlled Phase III clinical trial of Epidiolex (cannabidiol or CBD) CV in the treatment of seizures associated with Tuberous Sclerosis Complex (TSC), a rare and severe form of childhood-onset epilepsy.

In this trial, Epidiolex met its primary endpoint, which was the reduction in seizure frequency compared to baseline of the Epidiolex 25 mg/kg/day dose group vs placebo (p=0.0009). Results for both the 25 and 50 mg/kg/day dose groups were similar, with seizure reductions of 48.6% and 47.5% from baseline respectively, vs 26.5% for placebo (50 mg/kg/day vs placebo, p=0.0018). All key secondary endpoints were supportive of the effects on the primary endpoint. The safety profile observed is consistent with findings from previous studies, with no new safety risks identified.

Comment: Tuberous Sclerosis Complex (TSC) is a genetic disorder that causes non-malignant tumors to form in many different organs, with the brain and skin being the most commonly affected tissues. There are approximately 50,000 in the United States and nearly 1 million people worldwide estimated to have TSC. Epilepsy is the most common presenting symptom in TSC and is also the most common medical disorder in TSC..