FibroGen Duchenne Study Misses Endpoint

FibroGen says its Phase 3 LELANTOS-2 trial of pamrevlumab for treating ambulatory patients with Duchenne muscular dystrophy did not meet the primary endpoint of change in the North Star Ambulatory Assessment total score from baseline to Week 52. Secondary endpoints (four-stair climb velocity, 10-meter walk/run test, time to stand, time to loss of ambulation, and proportion of patients with greater than 10 seconds in the 10-meter walk/run test) were also not met. The company says it is evaluating the results, including other pre-specified endpoints, to determine next steps.

Pamrevlumab is described as a potential first-in-class antibody being developed to inhibit the activity of connective tissue growth factor, a common factor in fibrotic and proliferative disorders characterized by persistent and excessive scarring that can lead to organ dysfunction and failure. Pamrevlumab is also being studied as a treatment for pancreatic cancer.