London, UK; 14 March 2016: GW Pharmaceuticals plc (Nasdaq: GWPH, AIM: “GWP,” “GW,” “the Company” or “the Group”), a biopharmaceutical company focused on discovering, developing and commercializing novel therapeutics from its proprietary cannabinoid product platform, announces the positive results of the first pivotal Phase 3 study of its investigational medicine Epidiolex® (cannabidiol or CBD) for the treatment of Dravet syndrome. In this study, Epidiolex achieved the primary endpoint of a significant reduction in convulsive seizures assessed over the entire treatment period compared with placebo (p=0.01). Epidiolex has both Orphan Drug Designation and Fast Track Designation from the U.S. Food and Drug Administration (FDA) in the treatment of Dravet syndrome, a rare and debilitating type of epilepsy for which there are currently no treatments approved in the U.S.
See the full announcement at GW Pharmaceuticals
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The Phase 3 study randomized 120 patients into two arms, Epidiolex 20mg/kg/day (n=61) and placebo (n=59). Epidiolex or placebo was added to current anti-epileptic drug (AED) treatment regimens. On average, patients were taking approximately 3 AEDs, having previously tried and failed an average of more than 4 other AEDs. The average age of trial participants was 10 years and 30 percent of patients were less than 6 years of age. The median baseline convulsive seizure frequency per month was 13.
The primary efficacy endpoint was a comparison between Epidiolex and placebo measuring the percentage change in the monthly frequency of convulsive seizures during the 14-week treatment period compared with the 4-week baseline observation period. In this study, patients taking Epidiolex achieved a median reduction in monthly convulsive seizures of 39 percent compared with a reduction on placebo of 13 percent, which was highly statistically significant (p=0.01). A series of sensitivity analyses of the primary endpoint confirmed the robustness of this result. The difference between Epidiolex and placebo emerged during the first month of treatment and was sustained during the entire treatment period.
Results from secondary efficacy endpoints reinforced the overall effectiveness observed with Epidiolex.
Epidiolex was generally well tolerated in this study. The most common adverse events (occurring in greater than ten percent of Epidiolex-treated patients) were: somnolence, diarrhea, decreased appetite, fatigue, pyrexia, vomiting, lethargy, upper respiratory tract infection and convulsion. Of those patients on Epidiolex that reported an adverse event, 84 percent reported it to be mild or moderate. Ten patients on Epidiolex experienced a serious adverse event compared with three patients on placebo. Eight patients on Epidiolex discontinued treatment due to adverse events compared with one patient on placebo.
Further data will be presented in future publications and medical meetings.
In addition to this first Phase 3 trial, GW is conducting a second Phase 3 trial in Dravet syndrome which is recruiting 150 patients.
GW Clinical Trial Programs in Lennox-Gastaut Syndrome and Tuberous Sclerosis Complex
In addition to the Dravet syndrome trials, GW is conducting the largest global pivotal clinical trial program to date in Lennox-Gastaut syndrome, another rare and severe form of epilepsy. The first Phase 3 trial is a placebo-controlled trial of Epidiolex (at a dose of 20 mg/kg) over a 14 week treatment period and has randomized 171 patients. This trial is expected to report top-line results in the second quarter of 2016. The second placebo-controlled trial has randomized a total of 225 patients and is expected to report top-line results mid-2016. The primary measure of efficacy in these two trials will be the comparison between Epidiolex and placebo in the percentage change in number of monthly drop seizures during the 14-week treatment period compared with the 4-week baseline observation period.
Based on the findings of the physician-led expanded access program, GW continues to identify additional development targets for Epidiolex within the field of pediatric epilepsy. A Phase 3 trial in a third pediatric epilepsy indication, Tuberous Sclerosis Complex, is due to commence imminently and clinical development in a fourth indication is expected to commence in the second half of 2016.