Fulcrum’s Losmapimod Improves Multiple Measures, But Not Primary Endpoint

Fulcrum Therapeutics Inc. is arguing that although losmapimod failed to meet the primary endpoint in the Phase IIb ReDUX4 study in facioscapulohumeral muscular dystrophy (FSHD), it met other endpoints measuring clinical benefit that are more important. Whether it can convince the US Food and Drug Administration or will need to conduct another trial could set an important standard for other companies with DUX4-targeting drugs for FSHD currently in preclinical development.

The ReDUX4 results were presented 24 June at the FSHD International Research Congress, showing what Cambridge, MA-based Fulcrum said were clinically relevant and statistically significant benefits compared with placebo on multiple measures of structural and functional disease progression and patient-reported outcomes at 48 weeks