British cannabinoid company GW Pharmaceuticals ($GWPH) was up about 15% in early trading on news that its lead candidate, Epidiolex (cannabidiol or CBD), had positive Phase III data in the treatment of a rare and severe kind of epilepsy. In March, it also released positive Phase III data for the candidate to treat Dravet syndrome; the candidate has Orphan Drug Designation from the FDA in both indications.
The company said it expects to submit an NDA for Epidiolex to the FDA during the first half of next year. The latest data were in Lennox-Gastaut syndrome (LGS) patients, who are difficult to treat, with most not getting an adequate response from existing treatments.
“Epidiolex represents an exciting potential new class of anti-seizure drug, which may lead to meaningful seizure management and otherwise treatment resistant patients in the setting of a well-tolerated safety profile,” said GW Pharma CEO Justin Gover on a June 27 call about the data.
With the latest data alongside the data from March, “We believe that these two events together have remarkably changed the profile and future prospects with GW,” he added.
LGS is a rare and severe form of childhood-onset epilepsy. The 171-patient randomized, double-blind, placebo-controlled Phase III trial found that Epidiolex had a statistically significant median reduction in monthly drop seizures of 44%, as compared with 22% for placebo, during the 14-week treatment period. That was the primary endpoint of the trial. Patients in the trial had already been treated with an average of 9 antiepileptic drugs.
Specifically, drop seizures are atonic, tonic and tonic-clonic seizures involving the entire body, trunk or head that led or could have led to a fall, injury, slumping in a chair or hitting the patient’s head on a surface.
Epidiolex patients reported an 86% rate of adverse events, as compared with 69% for placebo patients. The most common, occurring in more than 10% of patients, were diarrhea, somnolence, decreased appetite, pyrexia and vomiting.
The company expects to report on a second, 225-patient Phase III trial in this indication. This is a dose-ranging trial with three treatment arms with top-line data expected around the end of the third quarter.
It has previously said that it may submit to the FDA for Epidiolex together for both Lennox-Gastaut and Dravet syndromes. This spring, it also started a Phase III trial for the candidate in a third indication: tuberous sclerosis complex.
GW has an upcoming pre-NDA meeting with the FDA on Epidiolex in Dravet syndrome, and it will request a separate meeting for the candidate in Lennox-Gastaut syndrome. But it does believe the indications are related and may be reviewed together.
“With this positive LGS data now in hand, we anticipate requesting a pre-NDA meeting for Epidiolex in Lennox-Gastaut syndrome with the FDA to discuss these results,” said CMO Dr. Stephen Wright on the call about the data. “We're also expecting to have our pre-NDA meeting regarding Dravet syndrome very soon. It's pretty conventional that you hold a separate pre-NDA meeting for each indication in which you are developing your drug. … So the chances are that they will--they may put a different review onto one indication, then on to another,” he added.
“But as we signaled, we clearly think there's a relationship between the two syndromes that we've been looking at, many of the seizure types of over that, many of the patients are in the instant chart and adolescent age group. We would certainly hope and aspire to be discussing the Lennox-Gastaut results with FDA at the pre-NDA meeting for Dravet,” concluded Wright.
The company already markets Sativex, a cannabinoid to treat spasticity due to multiple sclerosis, outside the U.S.; it is also in development in cancer pain.
GW Pharma got a big bump up on the data in Dravet syndrome in March; it’s gained almost 50% so far this year to bring its market cap to more than $1.5 billion.
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