Avidity Biosciences Reports AOC 1020 Data Showing 50% Reduction in FSHD Trial

Jun 12, 2024, 11:40 AM

Avidity Biosciences ($RNA) has announced initial clinical data from their FSHD program, presenting unprecedented results from the Phase 1/2 FORTITUDE trial. The AOC 1020 data show a greater than 50 percent reduction in DUX4 regulated genes and trends of functional improvement in people living with Facioscapulohumeral Muscular Dystrophy (FSHD). This marks the first time convincing measures of DUX4 target engagement have been demonstrated in humans, despite technical challenges in the biopsy process. Management has guided to expect changes in DUX4 target gene expression, but potentially not for all patients due to these challenges. The results highlight the potential of antibody-oligonucleotide conjugates in treating FSHD.