The little-known small cap Corbus Pharmaceuticals ($CRBP), last year touted as a rival to Vertex ($VRTX), has published positive top-line results from its experimental med resunab in patients with a disease that can cause major fibrosis throughout the body.
The Norwood, MA-based biotech saw its med show a “clear signal of clinical benefit” in a Phase II test in patients with systemic sclerosis. Its shares jumped 70% premarket this morning on the news.
Resunab, also known as JBT-101, out-performed placebo in the American College of Rheumatology Combined Response Index in diffuse cutaneous Systemic Sclerosis (CRISS) score--reaching 33% at week 16, versus 0% for placebo.
The double-blind, randomized, placebo-controlled Phase II looked at the clinical benefit and safety in 27 subjects who received Corbus’ med and 15 who received placebo.
It was however a close-run thing as the difference in CRISS scores between resunab and a dummy therapy across all groups over the trial period was only just significant (p = 0.044).
The company notes however that the study wasn’t powered for P value, so getting it was a positive.
The drug, which has FDA orphan and fast track tags, will now be tested in subjects enrolled in a one-year open label extension, designed to gather data on long-term safety and durability of response.
This test, which is open-label, will allow all participants in the study to receive Corbus’ med for an additional 12 months.
“We are excited about these positive clinical outcomes in the JBT-101 group in this study, especially given its short duration and relatively small number of diverse systemic sclerosis subjects,” said Barbara White, the biotech’s CMO.
“With these clinical data and findings of acceptable safety and tolerability, we plan to reach out to regulatory authorities to confirm the next steps forward.”
The drug, an endocannabinoid-mimetic, targets the CB2 receptor pathway found in lymphocytes, dendritic cells, macrophages, glial cells and other immune cells and is believed to turn off a signal for chronic inflammation and fibrosis.
Yuval Cohen, CEO of the biotech, said: “The positive results of this study exceed our expectations and validate the unique mechanism of action of JBT-101. Our drug previously demonstrated clear and consistent proof of activity in cellular and animal models as well as in healthy volunteers, repeatedly showing that its engagement with the CB2 receptor and its activation of inflammatory resolution translates into a potent effect on inflammation and fibrosis.
“With the data from this Phase II study, we now show that this mechanism of action provided clinical benefit in patients with systemic sclerosis in this trial. We look forward to the next stages in the clinical development of this drug. We are sincerely grateful to the patients, their physicians and the clinical staff who participated in this trial.”
Last spring, the biotech moved from the OTC market into Nasdaq with a market cap of less than $80 million--but this has now grown to $240 million.
In May 2015, it was handed a notable award worth up to $5 million from the nonprofit Cystic Fibrosis Foundation Therapeutics to help cover the costs of a Phase II study of resunab in CF.
It is also in tests for skin-predominant dermatomyositis, while set for an NIH-sponsored trial in systemic lupus erythematosus, which is planned to begin during the first half of next year.