Shares of Cambridge, UK based GW Pharmaceuticals ($GWPH) shot higher to the tune of over 10% on Tuesday after the biopharmaceutical marijuana company announced the results of its completed Phase 3 trials for two forms of epilepsy, which will be presented at the American Epilepsy Society (AES) annual meeting taking place in Washington D.C. December 1-5th.
GW’s flagship purified CBD product, Epidiolex, is being developed for the treatment of rare childhood-onset epilepsy disorders.
The company’s cannabidiol (CBD) treatment has shown efficacy in patients with Lennox-Gastaut syndrome (LGS) and Dravet syndrome, and is expected to gain FDA approval for a 2018 launch. To date, GW has received Orphan Drug Designation from the FDA for treatment of Dravet Syndrome and LGS, and has received Fast Track Designation from the FDA.
Abstracts released today include the following highlights:
- Responder analyses from pooled Phase 3 data of LGS patients on/off clobazam
- Data from two expanded access sites in patients on/off clobazam
- Long-term maintenance treatment effect and safety in the open label extension study and the expanded access program
- Exposure-response analysis of CBD in Phase 3 LGS studies
In a Tuesday press release, GW’s CEO Justin Glover stated “We continue to see a robust flow of data from the Epidiolex clinical program and look forward to sharing these new data with the epilepsy community at AES. Importantly, data in the abstracts show long-term maintenance of safety and efficacy, as well as that patients on Epidiolex who achieved clinically meaningful responses did so regardless of concomitant clobazam therapy,” said Justin Gover, GW’s Chief Executive Officer. “Having recently completed our New Drug Application submission to the FDA, we are now focused on the goal of gaining approval for Epidiolex in mid-2018 and making this much-needed, first-in-class medicine available to the patients who need it.”
The non-psychoactive CBD cannabinoid found in Marijuana, was famously used to treat seizures in Charlotte Figi, a Colorado child who suffered from 30 minute seizures suspected to be Dravet syndrome.
The twins were 3 months old when the Figis’ lives changed forever. Charlotte had just had a bath, and Matt was putting on her diaper.“She was laying on her back on the floor,” he said, “and her eyes just started flickering.” –CNN
As the years went on, Charlotte got worse.
The strain was low in THC (which gets you high), and high in CBD (non-psychoactive component).
By then, Charlotte had lost the ability to walk, talk, and eat, and was having 300 grand mal seizures a week. Charlotte’s heart had stopped several times – only to be resuscitated over and over.
At just five years old, the Figis turned to CBD cannabinoid treatment as a last resort.
“(But) they had exhausted all of her treatment options,” Shackelford said. “There really weren’t any steps they could take beyond what they had done. Everything had been tried — except cannabis.”
The results were stunning…
“When she didn’t have those three, four seizures that first hour, that was the first sign,” Paige recalled. “And I thought well, ‘Let’s go another hour, this has got to be a fluke.’ ”
The seizures stopped for another hour. And for the following seven days.
Paige said she couldn’t believe it. Neither could Matt. But their supply was running out.
Enter the Stanley Brothers
With their expensive supply of CBD oil marijuana running out, one of Colorado’s largest marijuana growers, the Stanley Brothers, stepped up and created a strain of marijuana high in CBD and low in THC, just for Charlotte, and named it Charlotte’s web.
“The biggest misconception about treating a child like little Charlotte is most people think that we’re getting her high, most people think she’s getting stoned,” Josh Stanley said, stressing his plant’s low THC levels. “Charlotte is the most precious little girl in the world to me. I will do anything for her.”
Years later, Charlotte was thriving – only having 2-3 seizures per month, mostly in her sleep. Not only can she walk, but she’s riding a bicycle, feeding herself, and talking.
“I literally see Charlotte’s brain making connections that haven’t been made in years,” Matt said. “My thought now is, why were we the ones that had to go out and find this cure? This natural cure? How come a doctor didn’t know about this? How come they didn’t make me aware of this?”
And now, GW Pharmaceuticals has been given an FDA fast-track for their CBD seizure treatment.