After failing in an ulcerative colitis test a few years back, GW Pharma’s cannabis-derived orphan drug treatment has had better luck in a small oncology study for an aggressive type of brain tumor.
In the phase 2 study, the U.K. biopharma’s cannabidiol (CBD) and tetrahydrocannabinol (THC) candidate, both of which are active ingredients derived from cannabis, showed positive top-line results from an exploratory placebo-controlled trial in 21 patients with recurrent glioblastoma multiforme, or GBM, a particularly aggressive brain tumor that has a poor prognosis.
The London-based company’s trial showed that those with recurrent GBM treated with THC:CBD had an 83% one-year survival rate, compared with 53% for patients in the placebo cohort (although the p-value was close at 0.042).
After an initial safety test, the trial went into a controlled phase where 12 patients were randomized to THC:CBD as add-on therapy, compared with nine patients randomized to placebo (plus standard of care).
“Median survival for the THC:CBD group was greater than 550 days compared with 369 days in the placebo group,” the biopharma said in a statement, adding that THC:CBD “was generally well tolerated with treatment emergent adverse events leading to discontinuation in two patients in each group.”
The company believes that, in glioma, THC and CBD “appear to act via distinct signalling pathways,” backing up what it had seen in preclinical animal studies.
The most common adverse events were vomiting and dizziness. GW said that the results of some biomarker analyses “are still awaited.”
“We believe that the signals of efficacy demonstrated in this study further reinforce the potential role of cannabinoids in the field of oncology and provide GW with the prospect of a new and distinct cannabinoid product candidate in the treatment of glioma,” said Justin Gover, GW’s CEO.
“These data are a catalyst for the acceleration of GW’s oncology research interests and over the coming months, we expect to consult with external experts and regulatory agencies on a pivotal clinical development program for THC:CBD in GBM and to expand our research interests in other forms of cancer.”
This will be welcome news for GW Pharmaceuticals after an oral capsule form of its drug missed its primary endpoint in a midstage trial for ulcerative colitis. It had however always had hope that its med could help treat an array of diseases.
The company was up more than 3.5% premarket this morning on the news. Last fall it saw its shares up around 20% on a report from Reuters that it has been approached by more than one potential acquirer, and hired investment bank Morgan Stanley to manage the process.
Its lead pipeline candidate is cannabidiol Epidiolex, which is seeking to gain an approval to treat Dravet syndrome and Lennox-Gastaut syndrome, both of which are rare forms of childhood-onset epilepsy.
The company already markets Sativex, a cannabinoid to treat spasticity due to multiple sclerosis, outside the U.S. and is also in development in cancer pain.