Avidity Biosciences' Duchenne Muscular Dystrophy Treatment Shows Promise in Phase 1/2 Trial

Avidity Biosciences reported positive results from its Phase 1/2 trial of AOC 1044, a potential treatment for Duchenne muscular dystrophy (DMD). The trial showed that the therapy demonstrated disease reversal, with patients experiencing improvements in muscle strength and function. The company's pipeline includes other RNA therapeutics for myotonic dystrophy type 1 and facioscapulohumeral muscular dystrophy.

Avidity Biosciences, Inc. (Nasdaq: RNA) has announced positive data from its Phase 1/2 trial of AOC 1044, a potential treatment for Duchenne muscular dystrophy (DMD). The trial, known as EXPLORE44®, demonstrated that the therapy led to disease reversal and significant improvements in muscle strength and function. The company's pipeline also includes other RNA therapeutics for myotonic dystrophy type 1 and facioscapulohumeral muscular dystrophy.

The EXPLORE44® trial, which enrolled 26 participants with DMD44 mutations amenable to exon 44 skipping, showed that AOC 1044 delivered phosphorodiamidate morpholino oligomers (PMOs) to skeletal muscle and heart tissue. This resulted in sustained muscle fiber protection, with participants experiencing statistically significant increases in dystrophin production and rapid reductions in creatine kinase (CK) levels. The functional improvements were maintained over 16 months of follow-up, with participants demonstrating improvements in multiple key functional measures such as Time to Rise from Floor (TTR), 4-Stair Climb (4SC), Performance of Upper Limb (PUL), and 10-Meter Walk/Run Test (10mWRT) Avidity Biosciences' Del-zota Demonstrated Reversal of Disease Progression Across Key Functional Endpoints in EXPLORE44® and EXPLORE44-OLE™ Phase 1/2 Trial in People Living with DMD44^[2].

Avidity Biosciences is on track to submit a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) at year-end 2025 for accelerated approval. The company's pipeline includes other RNA therapeutics for myotonic dystrophy type 1 and facioscapulohumeral muscular dystrophy, which are also being developed to address the unmet needs in these rare genetic conditions.

The positive results from the EXPLORE44® trial underscore the potential of Avidity Biosciences' targeted approach to RNA therapeutics. The company's commitment to delivering innovative treatments for rare genetic disorders has the potential to significantly impact the lives of patients and their families.