Positive Results from Phase 3 Trial of Epidiolex

Epidiolex has significantly reduced convulsive seizure frequency in children taking multiple anti-epileptic drugs with poor seizure control

GW Pharmaceuticals plc, the world leader in the science, development, and commercialization of cannabinoid prescription medicines, along with U.S. subsidiary Greenwich Biosciences, announced today that JAMA Neurology has published results from the second positive Phase 3 trial (GWPCARE2) of EPIDIOLEX® (cannabidiol) oral solution CV in children with seizures associated with Dravet syndrome.

The article has been published online and will be included in the May 2020 print issue of the journal. EPIDIOLEX, a pharmaceutical formulation of highly purified cannabidiol (CBD), is the first prescription, plant-derived cannabis-based medicine approved by the U.S. Food and Drug Administration (FDA) for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS) or Dravet syndrome in patients two years of age or older.

In the study, two doses of EPIDIOLEX, 10 and 20 mg/kg/day, significantly reduced convulsive seizure frequency compared to placebo in children two to 18 years of age with highly treatment-resistant Dravet syndrome, meeting the study's primary endpoint. The primary endpoint outcomes for the 10 and 20 mg/kg/day arms were similar, with seizure reductions of 49% and 46% from baseline, respectively, vs 27% for placebo (10 mg/kg/day, p=0.0095 and 20 mg/kg/day, p=0.0299).

"Dravet syndrome is one of the most difficult-to-treat forms of epilepsy and patients are highly individualized in their symptoms and dosing needs," said Ian Miller, M.D., Chief of Neurology at Nicklaus Children's Hospital in Miami, FL and lead author. "The data published by JAMA Neurologyshow that EPIDIOLEX 10 and 20 mg/kg/day were both efficacious and significantly reduced convulsive seizures. Drug-resistant seizures are common with Dravet syndrome and it is valuable to have a range of approved doses that offer physicians the flexibility to adjust treatment to a patient's specific needs."

Results from key secondary endpoints also showed:

  • Significant reductions in total seizure frequency from baseline: 56% for 10 mg/kg/day and 47% for 20 mg/kg/day vs 30% for placebo (p=0.0003 and p=0.0255, respectively).
     
  • Significantly more patients taking EPIDIOLEX (44% on 10 mg/kg/day and 49% on 20 mg/kg/day) achieved a 50 percent or greater reduction in convulsive seizures from baseline during the treatment period compared to placebo (26%; p=0.0332 and p=0.0069, respectively).

  • Compared with placebo, caregivers of patients treated with EPIDIOLEX were significantly more likely to report an improvement in overall condition as measured by the Caregiver Global Impression of Change (CGIC) scale at last visit (10 mg/kg/day, p=0.0009 and 20 mg/kg/day, p=0.0279).

The most common adverse reactions in the study (occurring in at least 10% of patients in any group) included decreased appetite, diarrhea, somnolence, pyrexia, and fatigue. Elevated liver transaminases occurred more frequently on 20 mg/kg/day than 10 mg/kg/day cannabidiol, with all affected patients on concomitant valproate.

"EPIDIOLEX continues to represent an important advancement in the treatment of difficult-to-treat pediatric-onset epilepsies such as Dravet syndrome where there are few FDA-approved therapies" Justin Gover, GW CEO.

"We are pleased that the full results of our GWPCARE2 study in Dravet syndrome are now available to the greater neurology community. The continued publication of our data is a testament to our groundbreaking research in the field of cannabinoids, which we plan to continue to advance in an effort to bring more novel treatments to patients in need."

Study Overview

The pivotal Phase 3 study of EPIDIOLEX – GWPCARE2 – was a randomized, double-blind placebo-controlled trial of patients aged 2-18 years with a confirmed diagnosis of drug-resistant Dravet syndrome currently uncontrolled on one or more concomitant anti-epileptic drugs (AEDs).

The trial randomized 199 patients into three arms, where EPIDIOLEX 10 mg/kg/day (n=67), EPIDIOLEX 20 mg/kg/day (n=67), or placebo (n=65) was added to current AED treatment. On average, patients were taking three AEDs, having previously tried and discontinued on average, four other AEDs. The average age of trial participants was 9 years. The median baseline convulsive seizure frequency per month was 12 and the median baseline total seizure frequency per month was 35.

"When scientific research is validated by esteemed journals like JAMA Neurology, it's a win for every member of the Dravet community seeking more information and a greater understanding of treatments for their condition," said Mary Anne Meskis, Executive Director of the Dravet Syndrome Foundation. "Patients and their families are in need of treatment options that work for this devastating illness and it's exciting to see further proof that EPIDIOLEX has the potential to make a meaningful difference in patients' lives."

Results from the first positive Phase 3 pivotal trial of EPIDIOLEX in patients with Dravet syndrome were published in The New England Journal of Medicine.

The EPIDIOLEX clinical program now includes five positive randomized, controlled Phase 3 clinical trials in Lennox-Gastaut Syndrome, Dravet syndrome and tuberous sclerosis complex.

To learn more about GW Pharmaceuticals visit their Company HQ here

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Niki Mohazeb
Niki Mohazeb

Niki is a Sydney based writer, with a passion for promoting the health benefits of medicinal cannabis. Niki also enjoys researching and writing about the future of cannabis along with the many other benefits that the plant provides, such as the diverse utilities of hemp.

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