February 21, 2017
By Mark Terry, BioSpace.com Breaking News Staff
Norwood, Mass. – Corbus Pharmaceuticals had a terrific 2016, with stock climbing 412 percent. Jim Crumly, writing for The Motley Fool, takes a look at the company and whether it can continue that trend for 2017.
Corbus focuses on rare, chronic and serious inflammatory and fibrotic diseases. Its lead product candidate, Resunab, is a synthetic oral endocannabinoid-mimetic drug that targets chronic inflammation and fibrotic processes by triggering a pathway called “Resolution of Inflammation.” What has caught some attention among investors is the “cannabinoid” part.
There has been some interest by investors in companies that are investigating and developing drugs based on marijuana. Those include GW Pharmaceuticals PLC , Insys Therapeutics , Cara Therapeutics and Zynerba Pharmaceuticals .
That connection to Corbus is a bit misleading. Resunab has nothing to do with the marijuana plant. The drug is a synthetic compound that mimics some cannabinoid activity, preferentially binding to the CB2 receptor that is expressed on activated immune cells and fibroblasts. It is not psychoactive. It is being evaluated in cystic fibrosis, diffuse cutaneous systemic sclerosis (scleroderma), dermatomyositis and systemic lupus erythematosus.
Crumly writes, “If the approach works, the implications will be massive. A number of diseases involve an imbalance between the ‘on’ and ‘off’ switches of the immune system that result in an inability to resolve inflammation. The first Phase II trial that produced efficacy results tested Resunab on patients with systemic sclerosis, a rare auto-immune disease that affects 90,000 patients in the U.S. and Europe, which has 40% to 60% mortality within 10 years and for which there is no FDA-approved treatment.”
The biggest market, for which it is being evaluated, is lupus, which has 500,000 patients in the U.S. and Europe.
The company completed its Phase II study of Resunab in cystic fibrosis at the end of December, but hasn’t released topline data yet, although it is expected to in the first quarter of this year.
It did have data in mid-November on the trial for systemic sclerosis. Primarily a safety trial, there was some statistically significant improvement in patients’ health, which caused the company stock to jump 50 percent in a single day.
Crumly notes, “Resunab is far from proven at this point. The Phase II trial turned a positive result with a p-value of .044, where a value below .05 is generally considered significant. In other words, the results barely met the statistical threshold. That doesn’t mean that the drug barely worked; the trial was just too small to give greater confidence, and up to now the only evidence that the drug would work came from animal studies and observations from pre-clinical studies.”
The drug was developed in the 1990s. Two other companies tried to develop it to treat pain and abandoned their efforts.
On January 17, 2017, the European Commission granted Orphan Designation for systemic sclerosis. It received Orphan Drug Designation and Fast Track status from the U.S. Food and Drug Administration for systemic sclerosis and cystic fibrosis, as well as Orphan Designation for CF in the EU as well.
So the company definitely shows promise. But it’s also a unicorn—a one-product company—and everything depends on it. Crumly writes, “The odds might be better for Resunab given the encouraging results so far, but given that the company is betting its entire future on this single molecule, risk-averse investors should be wary. And commercialization is still years away, even assuming the drug proves out.”
Corbus is currently trading for $9.
