Avidity Biosciences Reports Promising Phase 1/2 Data for AOC 1020 with 50% Reduction in DUX4

Jun 12, 2024, 12:26 PM

Avidity Biosciences ($RNA) announced promising results from its Phase 1/2 FORTITUDE trial for AOC 1020, targeting facioscapulohumeral muscular dystrophy (FSHD). The data showed a greater than 50 percent reduction in DUX4 regulated genes and trends of functional improvement in patients. This marks the first convincing measure of DUX4 target engagement in humans. The announcement has led to a 21.6% pre-market increase in Avidity Biosciences' stock price. Comparisons were made to Fulcrum Therapeutics' data, suggesting Avidity's drug may be more potent, though the data is at an earlier time point (16 weeks vs. 48 weeks). The results are significant for the FSHD treatment landscape, indicating potential efficacy and safety of AOC 1020. Additionally, the data derisks RWS as a more reliable endpoint for Fulcrum's upcoming readout in Q4.