GW Pharmaceuticals is going to increase its focus on the development of its investigational, cannabinoid-based therapy GWP42006 to treat autism spectrum disorders (ASD), including Fragile X, the company announced.
According to a press release, the company’s decision is based on recent results of a clinical trial in which the therapy failed to improve symptoms of focal seizures in adult patients.
The randomized, placebo-controlled Phase 2 trial (NCT02365610) included 162 adults with focal seizures across several sites in Europe. Led by Josemir W. Sander, MD, PhD, professor at the Institute of Neurology of University College London, the trial showed that focal seizures were reduced by roughly 40 percent in both the GWP42006 and placebo groups.
GW is currently analyzing the trial data to understand what factors could have contributed to the increased response in the placebo group.
“GW’s R&D focus has evolved in the last few years towards pediatric neurology where we have generated significant positive clinical data,” Justin Gover, CEO of GW, said in the release.
“Whilst the results of this adult focal seizure study for GWP42006 are disappointing, we remain committed to advancing this pipeline compound to address unmet needs in the field of autism spectrum disorders, in which a promising body of pre-clinical data has already been generated, as well as continuing to explore the product’s potential within the field of epilepsy,” he said.
Data from a previous Phase 1 trial (NCT01918735) in 66 healthy volunteers showed that GWP42006 is well-tolerated, even when given at the highest tested dose, with no significant adverse side effects reported.
Preclinical studies in models of both general and syndromic ASD, including Rett syndrome and Fragile X, have shown promising data on improving cognitive and social features of the diseases, as well as repetitive behaviors.
GW is planning to advance various clinical initiatives to evaluate the potential of GWP42006 in treating ASDs. These will include a physician-led, expanded access investigative new drug (IND) study in 10 patients with autism, and open-label and placebo-controlled Phase 2 trials in Rett syndrome. Topline data from the IND study are expected later in 2018.
GWP42006 is a formulation of a non-psychoactive compound, so-called cannabidivarin (CBDV), which is extracted from the cannabis plant. The U.S. Food and Drug Administration granted orphan drug designation to GWP42006 for treatment in Rett syndrome.