GW Pharmaceuticals' Epidiolex hits main goal of second Phase III study for Lennox-Gastaut syndrome

GW Pharmaceuticals reported Monday that a Phase III study of Epidiolex (cannabidiol) for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS) achieved the primary endpoint at both dose levels tested with high statistical significance. The company reaffirmed that it expects to submit a US filing for the therapy in the first half of 2017. Shares in GW Pharmaceuticals rose as much as 16 percent on the news.

Earlier this year, the company announced positive results from another late-stage trial of Epidiolex in LGS, as well as a Phase III study in the treatment of seizures associated with Dravet syndrome (for additional analysis, see ViewPoints: Second successful Phase III study may open up many new doors for GW’s Epidiolex). CEO Justin Gover commented "as a body of data we now have three positive Phase III trial results," adding "these are very robust findings and provide a compelling case to support the efficacy and safety of the drug with US regulators."

In the latest study, 225 patients with drug-resistant LGS currently uncontrolled on at least one anti-epileptic drug were randomised to treatment with Epidiolex, at a dose of 10 mg/kg/day or 20 mg/kg/day, or placebo, both in addition to current anti-epileptic drug therapy. The main goal of the trial was the percentage change in the monthly frequency of drop seizures during the 14-week treatment period versus the four-week period prior to randomisation.

Results illustrated that the low and high doses of Epidiolex were associated with 37 percent and 42 percent reductions in seizure frequency, respectively, versus a decline of 17 percent in the placebo arm. GW Pharmaceuticals explained that the difference in efficacy between Epidiolex and placebo emerged in the first month of treatment and persisted throughout the treatment period. The drugmaker added that the findings for secondary endpoints reinforced the efficacy of the drug. The company noted that further results from the study will be presented in future research conferences and medical publications. 

Commenting on the news, Morgan Stanley analyst Andrew Berens remarked that the latest study findings further de-risk the Epidiolex development programme (for further analysis, read ViewPoints: GW Pharmaceuticals' Epidiolex passes another test). Earlier this month, speculation arose that GW Pharmaceuticals could be a takeover target after the company reportedly hired an investment banker after being approached to several potential suitors (for related analysis, see ViewPoints: Positive neurologist feedback on GW Pharmaceuticals' Epidiolex validates reported takeout interest.

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