Potential Cystic Fibrosis Treatment Does Not Meet Primary Endpoint

Corbus Pharmaceuticals announced topline results from its 28-week Phase 2b study of lenabasum in patients with cystic fibrosis (CF). The study enrolled patients in the U.S., Canada and Europe at high risk for recurrent pulmonary exacerbations (PEx). Subjects received lenabasum or placebo added to their background treatments for CF.

The CF-002 Phase 2b trial did not meet the primary endpoint of a statistically significant reduction in rate of new PEx per subject per 28 weeks. Lenabasum treatment had a favorable safety profile and was well-tolerated.

“We are very disappointed that the study did not meet the primary endpoint. We look forward to providing more details of study results starting tomorrow at NACFC. We thank the participants, the staff at study sites, the Cystic Fibrosis Foundation, and the European Cystic Fibrosis Society Clinical Trials for their support and partnership throughout this study,” Barbara White, M.D., Chief Medical Officer and Head of Research of Corbus, said.

“We are immensely grateful to the Cystic Fibrosis Foundation for their invaluable support of this program from its inception. It has been a privilege to work with the CF community throughout this development program,” Yuval Cohen, Ph.D., Chief Executive Officer of Corbus, said.

CF-002 was a multinational Phase 2b study evaluating the efficacy and safety of lenabasum in CF. This was a double-blind, randomized, placebo-controlled study, with dosing of lenabasum at 5 mg twice per day, lenabasum 20 mg twice per day or placebo twice per day for 28 weeks, with 4 weeks safety follow-up off active treatment. The primary efficacy endpoint was the event rate of new PEx per subject per 28 weeks, when the primary definition of new PEx was physician diagnosis of PEx, prescription of new antibiotics for that PEx starting more than 28 days after completion of the last antibiotic course for any previous PEx, with 4 out of 12 Fuch’s criteria present in the subject. The Phase 2b CF study was funded in part by a Development Award for up to $25 Million from the Cystic Fibrosis Foundation.

Lenabasum is a novel, oral, small molecule that selectively binds as an agonist to the cannabinoid receptor type 2 (CB2) and resolves inflammation and limits fibrosis in animal and human models of disease. CB2 is preferentially expressed on activated immune cells and on fibroblasts, muscle cells, and endothelial cells. Lenabasum has demonstrated acceptable safety and tolerability profiles in clinical studies to date.