GW Pharmaceuticals plc (GWPH) just changed the game…
Shares of GW Pharmaceuticals plc (GWPH) are up over 100% after announcing positive results for the first Phase 3 Clinical study for its Epidiolex product (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. Epidiolex has both Orphan Drug Designation and Fast Track Designation from the FDA.
About the Study
The Phase 3 study randomized 120 patients, Epidiolex and a placebo. Either Epidiolex or the placebo was added to the current anti-epileptic treatment regimen. On average, patients were taking approximately 3 drugs in their regimen, having previously tried and failed an average of 4+ other drugs. The average age of trial participants was 10 years and 30% of patients were less than 6 years of age.
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.
Patients taking Epidiolex achieved a median reduction in monthly convulsive seizures of 39% compared with a reduction on placebo of 13%, which was highly statistically significant. 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.
Reiterate GWPH as our Top Pick
GWPH released topline data on its phase 3 trial of Epidiolex in Dravet Syndrome showing a significant reduction in seizure frequency by 39% versus placebo reduction by 13%. Adverse events were reported in more than 10% of patients included somnolence, diarrhea, decreased appetite, fatigue, pyrexia, vomiting, lethargy, upper respiratory tract infection and convulsion.
We view the trial as a momentous positive because we expect that the marijuana-derived drug needs clear-cut efficacy and safety profiles for approval.
The company will seek pre-NDA meetings with the FDA on the back of the positive data and before the second phase 3 trials in Dravet Syndrome.
Outlook is Bright
GWPH plans to readout three more pivotal trials during 2016. The 2nd phase 3 Dravet Syndrome trial will readout in the second half of 2016 and aims to enroll 150 patients. Two more pivotal phase 3 trials are ongoing in another rare form of pediatric epilepsy called Lennox-Gastaut Syndrome and are scheduled to readout topline data in second quarter of 2016.
We think that today’s positive data and additional readouts will lead to a 2017 Epidiolex launch.