Biopharmaceutical company GW Pharma (GWPH 131.21, +7.16) advances about 5.8% on Monday after this morning’s positive top-line results for the company’s cannabinoid-based childhood-onset epilepsy treatment EPIDIOLEX.
This morning GW announced positive top-line results of the second randomized, double-blind, placebo-controlled Phase 3 clinical trial of EPIDIOLEX (cannabidiol or CBD) CV in the treatment of seizures associated with Dravet syndrome, a rare and severe form of childhood-onset epilepsy. In this trial, EPIDIOLEX, when added to the patient’s current treatment, achieved the primary endpoint of reduction in convulsive seizures for both dose levels (10 mg/kg per day and 20 mg/kg per day) with high statistical significance compared to placebo. Both EPIDIOLEX doses also demonstrated statistically significant improvements on all key secondary endpoints.
During the treatment period, patients taking EPIDIOLEX 20 mg/kg/day demonstrated a 46% reduction in convulsive seizures while patients taking EPIDIOLEX 10 mg/kg/day achieved a reduction of 49%, compared to a 27% reduction in patients taking placebo. Results from the key secondary efficacy endpoints (reduction in total seizures) also showed statistical significance of both dose groups of EPIDIOLEX compared to placebo. In the EPIDIOLEX 20 mg/kg/day group, 49% of patients achieved a 50% or greater reduction in convulsive seizures from baseline over the treatment period, compared to 44% of patients taking EPIDIOLEX 10 mg/kg/day, and 26% of patients taking placebo.
The currently available safety data from this trial are consistent with the previous Phase 3 clinical trials. The data represents the fourth positive Phase 3 pivotal trial for EPIDIOLEX in Dravet syndrome and Lennox-Gastaut Syndrome. EPIDIOLEX was recently launched in the US and is now available by prescription.
Dravet syndrome is a severe infantile-onset and highly treatment-resistant epileptic encephalopathy frequently associated with genetic mutations in the SCN1A sodium channels. Onset of Dravet syndrome occurs typically during the first year of life in previously healthy and developmentally normal infants. Initial seizures are often body temperature related, severe, and long-lasting. Over time, patients with Dravet syndrome often develop multiple types of seizures, including tonic-clonic, myoclonic, and atypical absences and are prone to bouts of prolonged seizures including status epilepticus, which can be life threatening.
GWPH still holds losses of less than 1.0% on the year and sits about 27% lower from the late September 2018 highs.