London's GW Pharmaceuticals ($GWPH) said its cannabis-based treatment for epilepsy came through in a late-stage trial, sending the biotech's share price skyrocketing and improving its odds of FDA approval.
The drug, Epidiolex, is a liquid formulation of cannabidiol, a part of the marijuana plant that doesn't induce a high, that GW believes can reduce seizures in some severe forms of childhood epilepsy.
And in a Phase III trial on the rare Dravet syndrome, it did just that, the company said. The trial enrolled 120 patients with Dravet, giving roughly half Epidiolex and the rest placebo. After 14 weeks, patients in the treatment arm recorded a 39% median reduction in monthly seizures, significantly beating out the 13% seen with placebo, GW said.
On the safety side, 8 patients in the treatment arm dropped out due to side effects compared to just one from the placebo group, the company said. But of the Epidiolex patients who reported an adverse event, 84% said it was mild or moderate, according to GW.
The data roughly doubled GW's share value in premarket trading Monday, sending the biotech to its highest point since December.
The company is now planning to request a meeting with the FDA to map out a path to submitting Epidiolex for approval. GW is working through a second Phase III trial in Dravet with plans to enroll 150 patients, and the company has a late-stage program testing the drug in Lennox-Gastaut syndrome, another epilepsy-related rare disease. GW is also planning a Phase III Epidiolex trial in tuberous sclerosis complex, a third epilepsy indication.
GW is betting big on Epidiolex's future in the U.S. Last year, the company sent CEO Justin Gover to Southern California and appointed Allergan ($AGN) veteran Julian Gangolli to head up its North American business unit, all with the goal of laying the groundwork for a stateside commercial operation to get Epidiolex on the market as soon as possible.
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