Avidity Biosciences, Inc., a biopharmaceutical company specializing in RNA therapeutics, has announced that its investigational therapy, AOC 1044, has been granted Rare Pediatric Disease designation by the US Food and Drug Administration (FDA). AOC 1044 is designed to address Duchenne muscular dystrophy (DMD) in individuals with mutations amenable to exon 44 skipping (DMD44).

The announcement marks a significant milestone in Avidity's efforts to combat DMD, a rare genetic condition characterized by progressive muscle damage and weakness due to the loss of dystrophin protein, particularly affecting children from a young age. Presently, there are no approved therapies targeting exon 44, highlighting the urgent need for innovative treatments.

AOC 1044, currently undergoing assessment in the Phase 1/2 EXPLORE44™ trial, is the first of multiple Antibody Oligonucleotide Conjugates (AOCs™) developed by Avidity for DMD. The therapy has not only received Rare Pediatric Disease designation but also Orphan Designation from both the FDA and the European Medicines Agency (EMA), as well as Fast Track Designation from the FDA.

Dr. Steve Hughes, Chief Medical Officer at Avidity, expressed satisfaction with the FDA's decision, emphasizing the dire need for effective treatments for DMD44. "We are pleased that the FDA has granted Rare Pediatric Disease designation to AOC 1044, adding to the Orphan Drug and Fast Track designations already granted. The effects of DMD44 are devastating, with symptoms often starting in childhood. These designations by the FDA underscore the urgent need for innovative treatments and validate the potential of AOC 1044 to address the unmet need of people living with Duchenne muscular dystrophy," he said. 

The positive outlook for AOC 1044 is further bolstered by encouraging data from the EXPLORE44 trial, where the therapy demonstrated unprecedented delivery of therapeutic oligonucleotide in skeletal muscle and consistent exon skipping in healthy volunteers. The company plans to share data from the study involving individuals with DMD44 later in 2024.

Avidity's EXPLORE44 trial is a pivotal Phase 1/2 study evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of AOC 1044 in both healthy volunteers and participants with DMD44 mutations. With an anticipated enrollment of approximately 40 healthy volunteers and 24 DMD44 participants aged seven to 27 years old, the trial aims to assess exon skipping and dystrophin protein levels, offering hope for potential therapeutic breakthroughs in the management of DMD.