Drug Development

GW Pharma’s rally rolls on as cannabinoid drug scores again in PhIII Epidiolex anti-convulsive study

GW Pharmaceuticals’ late-stage cannabinoid high is on a roll.

The UK biotech posted strong efficacy data for its experimental anti-convulsive therapy Epidiolex in treating Lennox-Gastaut syndrome, three months after getting the party started with a big rally on its pivotal data for Dravet syndrome, a rare form of severe epilepsy.

Investigators said their drug exactly doubled the 22% median reduction in drop seizures among a group of LGS patients with an average age of 15 — 44% vs 22%.

The London-based company looks to be setting the stage to wrap up a slate of new drug applications for Epidiolex, and investors responded with a 20% spike in the biotech’s share price this morning. The surge came after the Brexit vote rattled markets at the end of last week, sending biotech shares down as investors fled risk in search of a port in the storm.

Back in March the company excited investors with results for the first of four late-stage studies on Epidiolex, with the drug reducing convulsions in children with treatment-resistant Dravet syndrome by a median average of 39% compared to only 13% in the placebo arm.

There are currently no drugs approved for the disease. And if GW can continue to rack up positive data, it may well prove to be the pioneer for cannabinoid treatments. Epidiolex uses a pure form of CBD, a compound found in marijuana. The plan now is to follow up with pre-NDA meetings with the FDA with a filing planned next year.

The company declined to speculate on a potential price for the drug in a call this morning with analysts, but some of the most bullish analysts covering the company have issued some dizzying peak sales estimates ranging from $2 billion to $3 billion a year.

GW will have to explain an unfavorable safety profile, though. Researchers reported that 20 patients on Epidiolex “experienced a serious adverse event (nine of which were deemed treatment related) compared with four patients on placebo (one of which was deemed treatment related). Twelve patients on Epidiolex discontinued treatment due to adverse events compared with one patient on placebo. There was one death in the Epidiolex group, which was deemed unrelated to treatment. Of the patients who completed this trial, 100 percent have opted to continue into an open-label extension trial.”

The type of serious events tracked in this second pivotal study is “very similar” to what was seen in the first trial, noted CMO Stephen Wright in the call. There were no unexpected serious adverse events and no changes in the safety profile.

GW also has a dose-ranging study for Lennox-Gastaut syndrome that should read out later in the year with a Phase III trial of Epidiolex in Tuberous Sclerosis Complex already underway and a late-stage study in infantile spasms due to launch in the fourth quarter.


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