A study to investigate the safety and efficacy of the exon-skipping therapy, AOC 1044, for the treatment of Duchenne muscular dystrophy (DMD) was announced by Avidity Biosciences.

The phase 1/2 EXPLORE44™ clinical trial will study AOC 1044 in healthy volunteers and participants with DMD. The randomized, placebo-controlled, double-blind trial will assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of single and multiple ascending intravenous doses.

AOC 1044 is part of a new RNA therapeutic class called antibody oligonucleotide conjugates (AOCs™), and it is designed to treat DMD patients with mutations amenable to exon 44 skipping (DMD44). The therapy delivers phosphorodiamidate morpholino oligomers (PMOs) to help enable dystrophin production in skeletal and cardiac muscles. Avidity has 2 other therapies in the pipeline that will target exon 45 and exon 51.


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“Advancing the EXPLORE44 phase 1/2 clinical trial for AOC 1044 is a significant milestone for Avidity and our proprietary AOC platform. There are no therapies addressing the underlying mechanism of disease for the young people and their families living with DMD44, who have a progressively debilitating and often ultimately fatal condition,” Sarah Boyce, president and chief executive officer of Avidity Biosciences, said.

“With the ability to deliver to skeletal muscle and heart tissue, it is our hope that AOC 1044 will potentially provide patients with mutations amenable to exon 44 skipping with a life-changing treatment option,” Craig M. McDonald, MD, professor and chair of the Department of Physical Medicine and Rehabilitation and director of the Rehabilitation and Training Center in Neuromuscular Diseases at the University of California, Davis, said.

Avidity also has clinical trials for other therapies for rare diseases, including AOC 1001 for the treatment of myotonic dystrophy type 1 and AOC 1020 for the treatment of facioscapulohumeral muscular dystrophy. AOC 1001 is being investigated in the MARINA™ phase 1/2 clinical trial and its open-label extension trial MARINA-OLE™, while AOC 1020 is being evaluated in the phase 1/2 FORTITUDE™ trial.

“AOC 1044 is the first of multiple AOCs that we are developing for DMD and our first AOC PMO to advance into the clinic. With three clinical programs for three distinct rare diseases in clinical development, we have achieved significant progress in the past year toward our vision of profoundly improving people’s lives by revolutionizing the delivery of RNA therapeutics,” Boyce said.

Reference

Avidity Biosciences announces phase 1/2 EXPLORE44™ trial of AOC 1044 for Duchenne muscular dystrophy mutations amenable to exon 44 skipping. News release. Avidity Biosciences, Inc; October 11, 2022.