Biotech Values

[haysaw]

GW Pharmaceuticals Reports Positive Phase 3 Pivotal Trial Results for EPIDIOLEX® (cannabidiol) Oral Solution in Patients with Seizures Associated With Tuberous Sclerosis Complex
GlobeNewswire•May 6, 2019
- Achieved primary efficacy measure with both EPIDIOLEX doses as compared to placebo -

- Represents the fifth consecutive positive Phase 3 pivotal trial for EPIDIOLEX -

- Expect to file sNDA in Q4 2019 -

https://finance.yahoo.com/news/gw-pharmaceuticals-reports-positive-phase-200100663.html

CARLSBAD, Calif., May 06, 2019 (GLOBE NEWSWIRE) -- GW Pharmaceuticals plc and its U.S. subsidiary Greenwich Biosciences Inc. (NASDAQ: GWPH, GW, the Company or the Group), the world leader in the science, development, and commercialization of cannabinoid prescription medicines, today announced positive top-line results of a randomized, double-blind, placebo-controlled Phase 3 clinical trial of EPIDIOLEX® (cannabidiol or CBD) CV in the treatment of seizures associated with Tuberous Sclerosis Complex (TSC), a rare and severe form of childhood-onset epilepsy. In this trial, EPIDIOLEX met its primary endpoint, which was the reduction in seizure frequency compared to baseline of the Epidiolex 25 mg/kg/day dose group vs placebo (p=0.0009). Results for both the 25 and 50 mg/kg/day dose groups were similar, with seizure reductions of 48.6% and 47.5% from baseline respectively, vs 26.5% for placebo (50 mg/kg/day vs placebo, p=0.0018). All key secondary endpoints were supportive of the effects on the primary endpoint. The safety profile observed is consistent with findings from previous studies, with no new safety risks identified.

“The positive outcome in this trial of EPIDIOLEX in patients with Tuberous Sclerosis Complex expands both our knowledge of this newly available medicine and its potential utility beyond the current indications,” stated Elizabeth Thiele, M.D., Ph.D., Director of the Herscot Center for Tuberous Sclerosis Complex at Massachusetts General Hospital, Professor of Neurology at Harvard Medical School and the lead investigator of the trial. “Data from previous controlled clinical trials of EPIDIOLEX have shown clinically meaningful seizure reductions and consistent safety and tolerability in children and adults with Lennox-Gastaut syndrome and Dravet syndrome. Based on the positive results of this trial in TSC patients, EPIDIOLEX, if approved for this additional indication, may become an important treatment option also in this disease state with significant unmet medical need.”

“The positive results from this trial represent the fifth positive Phase 3 trial for EPIDIOLEX and follows the recent U.S. launch of EPIDIOLEX for the treatment of seizures associated with Lennox-Gastaut syndrome and Dravet syndrome. These new data show EPIDIOLEX reduced TSC-associated seizures, which include both focal and generalized seizures types, expanding the body of reliable science supporting the use of EPIDIOLEX,” stated Justin Gover, GW’s CEO. “With these data, we look forward to submitting an sNDA to the FDA in the fourth quarter with the goal of expanding the product label in 2020 to help the lives of patients suffering with TSC.”

“Some of the most challenging and frustrating aspects of tuberous sclerosis complex (TSC) are seizures that cannot be effectively controlled by existing medications,” explained Kari Luther Rosbeck, President and CEO of the Tuberous Sclerosis Alliance, “A new safe and effective treatment option such as Epidiolex is desperately needed. Further, we are grateful to GW, the researchers and the members of the TSC community who participated in this clinical trial. We are truly excited about the potential approval of Epidiolex to treat seizures in TSC as it brings much needed hope to those with TSC and their families who live daily with this difficult disorder.”

Trial Overview and Results

Patients aged 1-65 years with a confirmed diagnosis of treatment-resistant TSC were eligible to participate in this Phase 3, randomized double-blind placebo-controlled trial. The trial randomized 224 patients into three arms, where EPIDIOLEX 25 mg/kg/day (n=75), EPIDIOLEX 50 mg/kg/day (n=73) or placebo (n=76) was added to current anti-epileptic drug (AED) treatment. The average age of trial participants was 14 years (range 1-57). On average, patients were taking 3 AEDs, having previously tried and discontinued 4 other AEDs. The most common concomitant AEDs in this trial were valproic acid (45 percent), vigabatrin (33 percent), levetiracetam (29 percent), and clobazam (27 percent).