GW Pharmaceuticals recently presented positive results from two Phase 3 clinical trials evaluating its lead cannabidiol therapeutic candidate, Epidiolex, in patients with lennox-gastaut syndrome (LGS) and Dravet syndrome (DS), two treatment-resistant epilepsy syndromes. The presentations took place during the American Epilepsy Society (AES) Annual Meeting in Houston, Texas Dec. 2-6.
Epidiolex is an oral formulation of pure plant-derived cannabidiol (CBD), being initially developed for severe, orphan, early-onset, treatment-resistant epilepsy syndromes including Dravet syndrome, LGS, tuberous sclerosis complex (TSC) and infantile spasms (IS). The drug has both Orphan Drug Designation and Fast Track Designation from the U.S. Food and Drug Administration (FDA) in the treatment of Dravet syndrome, for which there are no treatments approved in the United States.
“We are very excited to share these data from our Phase 3 programs with the epilepsy community. These presentations are the culmination of intensive effort by the GW in-house team and more than 50 participating sites over the past two years, and reflect the courage and commitment of patients and their families. We can now focus on the ultimate goal, which is to gain approval for Epidiolex and make this much-needed treatment available,” Justin Gover, GW’s chief executive officer, said in a press release.
“We have also reached an ideal moment to introduce our new U.S. identity, Greenwich Biosciences, which will be the face of our company to patients and their families, clinicians and the general public in the United States,” Gover said.
At the AES meeting, the company presented the results of two Phase 3 trials, one in DS and one in LGS, showing that treatment with Epidiolex led to significantly greater reductions in specific seizure types for patients taking the drug compared to those taking placebo.
The presentation titled “Cannabidiol (CBD) significantly reduces drop seizure frequency in Lennox-Gastaut syndrome: results of a multi-center, randomized, double-blind, placebo-controlled trial (GWPCARE4),” focused on the results reported in June of the first randomized, double-blind, placebo-controlled Phase 3 clinical trial (NCT02224690) Epidiolex for the treatment of Lennox-Gastaut syndrome (LGS).
In this trial, Epidiolex, when added as an adjunct to the patient’s current treatment, achieved the primary endpoint of a significant reduction in the monthly frequency of drop seizures assessed over the entire 14-week treatment period, compared with placebo.
The company also presented the results of GWPCARE1, a Phase 3 study (NCT02091375) that randomized 120 patients into two arms, Epidiolex 20mg/kg/day or placebo. Epidiolex or placebo was added to current anti-epileptic drug (AED) treatment regimen. Results announced in March showed that in this study Epidiolex achieved the primary endpoint of a significant reduction in convulsive seizures assessed over the entire treatment period, compared with placebo in patients with Dravet syndrome.
During the meeting, GW also presented additional laboratory results of the effects of its pure cannabidiol product on human CNS-expressed voltage-gated sodium channels, as well as multiple posters covering the use of Epidiolex in children and adults.
More information about the presentations can be found here.