Lenabasum Study for Systemic Sclerosis Does Not Meet Primary Endpoint

Corbus Pharmaceuticals has announced topline results from the 52-week Phase 3 RESOLVE-1 study of lenabasum in patients with diffuse cutaneous systemic sclerosis (SSc). SSc is a rare and life-threatening multi-system autoimmune disease for which there are currently no U.S. Food and Drug Administration (FDA)-approved treatments for overall disease.

Topline data showed no significant differences in the primary and secondary endpoints when comparing lenabasum to placebo, both added to background drug therapy.

For the primary endpoint, the median American College of Rheumatology Combined Response Index for Systemic Sclerosis (ACR CRISS) scores at Week 52 were 0.887 in the placebo arm and 0.888 in the lenabasum 20 mg twice daily arm. ACR CRISS is a composite endpoint that reflects the probability of patient improvement. The maximum achievable ACR CRISS score is 1.0.

RESOLVE-1 is the first 52-week, randomized, placebo-controlled Phase 3 trial that tested the efficacy and safety of lenabasum in 365 patients with diffuse cutaneous SSc in a multinational, double-blind, randomized, placebo-controlled study, with dosing of lenabasum at 20 mg twice daily, lenabasum at 5 mg twice daily, or placebo twice daily for 52 weeks. The majority of enrolled patients (84%) were receiving background immunosuppressive drugs, reflecting recent trends in clinical practice.

Similar proportions of placebo-treated and lenabasum-treated subjects had at least one treatment emergent adverse event (AEs), 86.2% in the placebo arm and 91.7% in the lenabasum 20 mg twice daily arm. Serious AEs occurred in 14.6% of subjects in the control arm and 9.2% of subjects in the lenabasum 20 mg twice daily arm. Severe AEs occurred in 13% of subjects in the control arm and 5.8% of subjects in the lenabasum arm. No subjects receiving lenabasum withdrew from the study because of an AE-related to study drug. Lenabasum treatment was well-tolerated in this study. No evidence of lenabasum-related immunosuppression or new safety signals for lenabasum were observed.

Further analyses of these data are underway, and once Corbus has a fuller understanding of the data, the Company would like to engage with the FDA to determine potential next steps in this clinical development program. The data will be presented at upcoming medical conferences.

“We are surprised and deeply disappointed that the RESOLVE-1 trial did not meet its primary endpoint. I would like to extend my gratitude to the participants in the study and the clinical staff at the study sites, as well as to the Corbus employees, for their hard work and dedication. We now look forward to upcoming topline results from our study of lenabasum in patients with cystic fibrosis,” Yuval Cohen, Ph.D., Chief Executive Officer of Corbus said.

Lenabasum was granted Orphan Drug designation and Fast Track designation for the treatment of SSc from the FDA and Orphan Designation for the treatment of SSc from the European Medicines Agency. Lenabasum is currently being evaluated in a Phase 3 DETERMINE study in dermatomyositis, a Phase 2 study in systemic lupus erythematosus, and a Phase 2b study in cystic fibrosis.

Systemic sclerosis, a form of scleroderma, is a chronic, rare, debilitating autoimmune disease affecting approximately 200,000 people in the North America, EU and Japan. Systemic sclerosis is considered one of the most life-threatening rheumatic diseases. The disease affects affects the skin and internal organs and is driven by inflammation and fibrosis (scarring of tissue) which can lead to severe damage and failure of multiple organs including the skin, joints, tendons, gastrointestinal tract, lungs, heart, blood vessels and kidneys. There is no cure for systemic sclerosis, and current treatments address the clinical manifestations of the disease, not the underlying mechanisms that drive inflammation and fibrosis.