Topline results from a 28-week Phase IIb trial show that lenabasum, a novel oral small molecule developed by Corbus Pharmaceuticals, did not reduce the rate of new pulmonary exacerbations compared with placebo in patients with cystic fibrosis.
Lenabasum fails in cystic fibrosis this time.
Topline results from a 28-week Phase IIb trial show that lenabasum, a novel oral small molecule developed by Massachusetts-based Corbus Pharmaceuticals, did not reduce the rate of new pulmonary exacerbations compared with placebo in patients with cystic fibrosis (CF).
Lenabasum is a novel small molecule that preferentially binds and acts as an agonist to the cannabinoid receptor type 2. The drug molecule was developed by Corbus to combat inflammation and fibrosis. Since its development, the molecule has been under investigation by Corbus for indications related to systemic sclerosis, dermatomyositis, systemic lupus erythematosus and CF. In the setting of CF, it is theorized that lenabasum may reduce CF-related pulmonary exacerbations, a common event for which no best management option exists.
While the new data from the company’s Phase IIb CF-002 trial do not appear to support its efficacy in reducing the rate of new pulmonary exacerbations in patients with CF, the study found that the potential treatment did feature a favorable safety profile and was well-tolerated among recipients. Data from the trial will be presented at the North American Cystic Fibrosis Conference, which will take place virtually between October 7 and 23 of this year.
“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,” Barbara White, M.D., Chief Medical Officer and Head of Research of Corbus, said in a statement. “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.”
Corbus has run into issues before with lenabasum in trials that studied the drug for other indications. In early September, for instance, the company reported that their experimental cannabinoid receptor type 2 agonist also failed to meet its primary endpoint in the Phase III RESOLVE-1 trial for diffuse cutaneous systemic sclerosis (SSc), a rare disease which currently lacks an approved treatment.
The multicenter, 365-patient RESOLVE-1 trial found no significant differences between lenabasum and placebo in terms of the ability to improve the American College of Rheumatology’s Composite Response Index in Systemic Sclerosis (CRISS) score by week 52. These findings suggest the drug is likely not effective for improving SSC over a one-year period. Announcement of the findings from this trial correlated with an almost immediate 77% plunge in company shares.
“Whilst the immediate study results are disappointing, RESOLVE-1 provides a rich dataset to understand for the first time how to better target treatments for SSc based upon clinical parameters and concomitant treatment,” co-principal investigator of RESOLVE-1, Christopher Denton, said in a statement. “We are already querying the data to understand the natural history of early diffuse cutaneous SSc and the potential benefits of lenabasum in these subjects.”
Shares in the company slumped 30% premarket following the recent announcement of findings from Corbus’ Phase IIb CF-002 trial. Institutions appear to own more than half of the company’s stock, with BlackRock, Inc. having the largest stake in the company to date.
