Corbus Pharmaceuticals says that its sole clinical-stage drug came through in a small Phase II study for systemic sclerosis, an autoimmune disease of connective tissue, with the patients taking the treatment posting a significant response compared to the placebo arm.
Using a composite score to gauge a response on a slate of key measures like lung function, the combined drug arm including patients on a range of doses of resunab (JBT-101) posted a median 33% CRISS score after 16 weeks of therapy, compared to 0% in the placebo group. And that marks a significant benefit for patients, says the biotech, with a p-value of 0.044 — not a great number, but within the margin needed to declare a win.
The trial listing on clinicaltrials.gov, though, does list 12-week results as the marker for the primary endpoint, when the CRISS score was a lower 27.5%. Also, one patient had to drop out of the drug arm after complaining of dizziness. CEO Yuval Cohen tells me that any CRISS score over 20% would be “medically meaningful.”
“The p=0.044 isn’t for week 16,” he adds in an email. “It is a comparison between the drug and placebo group across the entire study.”
Shares of Corbus rocketed up 95% this morning in pre-market trading as investors’ temperaments changed dramatically on their first take of the data.
Norwood, MA-based Corbus $CRBP had been on a roll in the lead up to the trial results, with its shares rising steadily right up to the end of October, when TheStreet’s Adam Feuerstein ran a column presenting an anonymous short attack that was in circulation, speculating the resunab was unlikely to demonstrate an effect in Phase II. The drug has bounced around for years among different companies and the data is “weak and inconsistent.” And similar drugs that target the CB2 receptor have failed as well.
That message resonated with investors, and Corbus’ shares cratered in the days after the Feuerstein column was posted, dropping 40%.
On Monday, investigators and company execs were pushing back with a much different message on the study, which included a total of 42 patients in both arms.
“This is the first double-blind, randomized, placebo-controlled trial in diffuse cutaneous systemic sclerosis to demonstrate a clinical benefit using the CRISS as an endpoint, with a drug that was safe and well tolerated. These results bring hope to patients and their physicians that JBT-101 may be an effective drug for systemic sclerosis where currently there are no proven treatments,” said Robert Spiera, the principal investigator and director of the Vasculitis and Scleroderma Program at the Hospital for Special Surgery, Weill Cornell Medical College.
A one-year extension study is now under way to test safety and long-term durability of the drug as the company looks at “next steps” with regulators.
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