I haven’t the slightest idea of how their medicine works. In my head, this company is smoking joints all day, discussing the virtues of cannabis. In reality, they’re probably smoking joints and also developing some kick ass treatments.
At any rate, this stock is up 126% in the pre-market, making it must-read news for today’s session. Whether this creates a halo-effect around biotech remains to be seen. But this data looks incredible.
“The results of this Epidiolex pivotal trial are important and exciting as they represent the first placebo-controlled evidence to support the safety and efficacy of pharmaceutical cannabidiol in children with Dravet syndrome, one of the most severe and difficult-to-treat types of epilepsy,” said Orrin Devinsky, M.D., of New York University Langone Medical Center’s Comprehensive Epilepsy Center. “These data demonstrate that Epidiolex delivers clinically important reductions in seizure frequency together with an acceptable safety and tolerability profile, providing the epilepsy community with the prospect of an appropriately standardized and tested pharmaceutical formulation of cannabidiol being made available by prescription in the future.”
“The positive outcome of this Phase 3 trial is a significant milestone in the development of Epidiolex as a potential new treatment for patients suffering from Dravet syndrome. We are excited about the potential for Epidiolex to become the first FDA approved treatment option specifically for Dravet syndrome patients and their families,” stated Justin Gover, GW’s Chief Executive Officer. “In light of this positive data, we will now request a pre-NDA meeting with the FDA to discuss our proposed regulatory submission. We also look forward with excitement to the upcoming results from the two Phase 3 trials in Lennox-Gastaut syndrome and the second pivotal trial in Dravet syndrome.”
“Dravet syndrome is one of the most catastrophic types of epilepsy in children and safe and effective treatments are desperately needed. We are thrilled to learn of these positive results, which bring much needed hope to the children and families who have been living with these debilitating seizures,” said Mary Anne Meskis, Executive Director of the Dravet Syndrome Foundation.
- In this study, Epidiolex achieved the primary endpoint of a significant reduction in convulsive seizures assessed over the entire treatment period compared with placebo (p=0.01).
- Co will now request a pre-NDA meeting with the FDA to discuss its proposed regulatory submission.
- In this study, patients taking Epidiolex achieved a median reduction in monthly convulsive seizures of 39 percent compared with a reduction on placebo of 13 percent, which was highly statistically significant (p=0.01). Results from secondary efficacy endpoints reinforced the overall effectiveness observed with Epidiolex.
- Epidiolex was generally well tolerated in this study.
- The most common adverse events (occurring in greater than 10 percent of Epidiolex-treated patients) were: somnolence, diarrhea, decreased appetite, fatigue, pyrexia, vomiting, lethargy, upper respiratory tract infection and convulsion.
- Of those patients on Epidiolex that reported an adverse event, 84 percent reported it to be mild or moderate. Ten patients on Epidiolex experienced a serious adverse event compared with three patients on placebo.
- Eight patients on Epidiolex discontinued treatment due to adverse events compared with one patient on placebo.
- The first Phase 3 trial is a placebo-controlled trial of Epidiolex (at a dose of 20 mg/kg) over a 14-week treatment period and has randomized 171 patients.
- This trial is expected to report top-line results in the second quarter of 2016.
- The second placebo-controlled trial has randomized a total of 225 patients and is expected to report top-line results mid-2016.
“decreased appetite”