Corbus Pharmaceuticals has posted data from a phase 2 trial of its lead drug in the inflammatory disease dermatomyositis. The study linked synthetic oral endocannabinoid-mimetic anabasum to a statistically significant drop on a skin disease scale, resulting in the trial hitting its primary efficacy endpoint with a p value of 0.04.
Investigators enrolled 22 dermatomyositis patients with Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) scores of 14 or greater. The subjects received anabasum—formerly known as resunab and JBT-101—or placebo for 16 weeks, in most cases on top of immunosuppressive drugs.
At the end of the trial, CDASI scores in the treatment arm had dropped 9.3 points, compared to a 3.7 decline in the placebo cohort. That was enough for the trial to just hit its efficacy endpoint. And, in the eyes of one of the trial’s investigators, suggested anabasum can improve outcomes in a population short on options.
“The results signal a clear clinical benefit associated with anabasum,” Victoria Werth, M.D., of the University of Pennsylvania, said in a statement. “I have extensive experience treating patients with dermatomyositis and we currently have little to offer patients with moderate to severe disease activity except immunosuppressive therapies with often limited efficacy and significant side effects.”
Treatments being used include corticosteroids and rituximab—but there is limited data on their effect on CDASI. In 2011, an 11-patient, single-arm study linked a low dose of methotrexate to an 8.35 mean improvement in CDASI. Four of the patients in that trial would have been ineligible for the Corbus study as their CDASI scores were below 14 at baseline.
Corbus said its trial linked anabasum to “improvements” across multiple secondary endpoints. Management at Corbus now plans to discuss the data with regulators with a view to figuring out a path forward for anabasum, which is also in development as a treatment for systemic sclerosis and cystic fibrosis.
Shares in Corbus climbed more than 20% in premarket trading.