FibroGen, Inc. announced topline data from the Phase 3 LELANTOS-1 placebo-controlled trial of pamrevlumab for the treatment of non-ambulatory patients with Duchenne Muscular Dystrophy (DMD) on background corticosteroids. The study did not meet the primary endpoint of Performance of the Upper Limb 2.0 (PUL 2.0) score at week 52 compared to baseline. Pamrevlumab was generally safe and well tolerated and the majority of treatment emergent adverse events were mild or moderate.

FibroGen plans to present the complete results of the LELANTOS-1 study at an upcoming medical conference and to publish the full results. Topline data from the Phase 3 LELANTOS-2 clinical trial of pamrevlumab for the treatment of ambulatory patients with DMD is expected Third Quarter 2023. A total of ninety-nine (99) DMD patients aged 12 years and older were enrolled in LELANTOS-1, a global Phase 3, randomized, double-blind, trial of pamrevlumab or placebo in combination with systemic corticosteroids to evaluate the efficacy and safety of pamrevlumab in patients with non-ambulatory DMD.

The U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease, Orphan Drug, and Fast Track Designation to pamrevlumab for the treatment of DMD. In the EU, FibroGen has been granted Orphan Drug Designation to pamrevlumab for the treatment of DMD. The Phase 3 clinical development program for pamrevlumab for DMD includes two studies, LELANTOS-1 and LELANTOS-2. These randomized, double-blind global Phase 3 trials are designed to evaluate the efficacy and safety of pamrevlumab in combination with systemic corticosteroids in patients with either non-ambulatory or ambulatory DMD.

For more information about LELANTOS-2, which is fully enrolled. Pamrevlumab is a potential first-in-class antibody being developed by FibroGen to inhibit the activity of connective tissue growth factor (CTGF), a common factor in fibrotic and proliferative disorders characterized by persistent and excessive scarring that can lead to organ dysfunction and failure. Pamrevlumab is in Phase 3 clinical development for the treatment of idiopathic pulmonary fibrosis (IPF), locally advanced unresectable pancreatic cancer (LAPC), and Duchenne muscular dystrophy (DMD), and in Phase 2/3 for the treatment of metastatic pancreatic cancer.

The U.S. Food and Drug Administration has granted Orphan Drug Designation, and Fast Track designation to pamrevlumab for the treatment of patients with IPF, DMD, and LAPC. The U.S. Food and Drug Administration has also granted Rare Pediatric Disease Designation to pamrevlumab for the treatment of patients with DMD. Pamrevlumab has demonstrated a safety and tolerability profile that has supported ongoing clinical investigation in IPF, DMD, and LAPC.

Pamrevlumab is an investigational drug and not approved for marketing by any regulatory authority.