FibroGen's One Study For Duchenne Muscular Dystrophy Fails To Hit Main Goal

FibroGen Inc FGEN announced topline data from the Phase 3 LELANTOS-1 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 the Performance of the Upper Limb 2.0 (PUL 2.0) score at week 52 compared to baseline. 

"While disappointed with these results, we look forward to sharing the data at a future medical conference to contribute towards the understanding of this devastating disease," CEO Enrique Conterno said in a statement.

Pamrevlumab was generally safe and well tolerated, and most 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 treating ambulatory patients with DMD is expected in Q3 2023.

Last month, FibroGen's MATTERHORN Phase 3 trial of roxadustat for anemia in patients with transfusion-dependent lower-risk myelodysplastic syndromes (MDS) did not meet its primary endpoint. 

Price Action: FGEN shares are down 4.68% at $17.84 on the last check Wednesday.