GW Starts Phase II/III Trial of Epidiolex in Dravet Syndrome

GW Pharmaceuticals plc announced that it has commenced a Phase II/III clinical trial evaluating Epidiolex in patients with Dravet syndrome, a rare and catastrophic treatment-resistant form of childhood epilepsy.

The two-part trial will test the safety, tolerability, pharmacokinetic and efficacy of Epidiolex. The first part will determine the ideal dosage for the drug and the second part will evaluate the drug’s safety and efficacy in this patient population.

Texas Children’s Hospital announced that it will be a site for the clinical trial. Beginning this month, the trial will enroll 30 patients worldwide to determine the correct dosages for the drug and later, with a second set of 80 patients, to determine if the drug helps to reduce or eliminate their seizures. Participants in the first part of the study will be evaluated over a 3-week period and participants in the second part of the study will be evaluated over a three-month period. All patients who complete the study will be eligible to receive Epidiolex under a long-term extension study. GW anticipates starting an additional Phase III trial in Dravet syndrome in the first quarter of 2015 in parallel with part two of the first Phase II/III trial.

“As one of the largest epilepsy centers in the country, our focus has always been to find new and innovative ways to treat and cure children with various forms of epilepsy,” said Dr. Angus Wilfong, neurologist at Texas Children’s Hospital. “Initial trials of Epidiolex have shown promising signals of efficacy in children with treatment-resistant epilepsy, and we are pleased to have the opportunity to partner with GW Pharmaceuticals in the first worldwide trial for this group of patients with such a catastrophic form of epilepsy.”

Epidiolex is a highly purified extract of the cannabis plant, containing pure Cannabidiol (CBD). It is a first-of-its-kind form of CBD, and does not contain any tetrahydrocannabinol (THC), the major psychoactive ingredient in marijuana. Texas Children’s first began using the drug in April under compassionate use protocols for certain patients with catastrophic forms of epilepsy who were not responding to other drugs.

Dravet syndrome occurs in about one in every 30,000 births. Children with the condition experience a diminished quality of life. It is not uncommon for patients with Dravet syndrome to experience dozens or even hundreds of seizures per day. These patients are also prone to prolonged life-threatening seizures called status epilepticus. Children with Dravet syndrome are often not responsive to medications that are currently available and their seizures are difficult to control. Children with the condition often struggle with developmental delays, a variety of health problems and have an increased risk of Sudden Unexplained Death in Epilepsy (SUDEP).

“We are pleased to have advanced Epidiolex into the pivotal stage of clinical development. The start of this Phase 2/3 trial represents a significant milestone for children that suffer with Dravet syndrome for which there remains a substantial unmet medical need,” said Justin Gover, GW’s Chief Executive Officer. “Epidiolex is the first plant-based CBD medicine to be studied in a FDA-authorized, placebo-controlled trial and we look forward to working with leading pediatric epilepsy centers across the US to advance this clinical program as rapidly as possible.”

Sources: GW Pharmaceuticals plc; Texas Children’s Hospital

Last updated: 10/30/14; 2:30pm EST