Avidity Biosciences, Inc., a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates announced the Phase 1/2 EXPLORE44™ clinical trial of AOC 1044 in healthy volunteers and participants with Duchenne muscular dystrophy (DMD), a rare, genetic condition that is characterized by progressive muscle damage and weakness. AOC 1044 is designed for people living with DMD amenable to exon 44 skipping (DMD44) and is the first of multiple AOCs the company is developing for DMD. Currently, there are no approved therapies to treat the underlying mechanism of disease for people living with DMD mutations amenable to exon 44 skipping. Avidity has advanced three distinct rare disease programs – DM1, FSHD and DMD44 – into clinical development in a 14-month period.
DMD is an irreversible, progressive disease caused by a genetic mutation that prevents the body from producing a protein called dystrophin, which is an important protein that protects muscle cells from injury during contraction. The lack of functional dystrophin leads to stress and tears of muscle cell membranes, resulting in muscle cell death and progressive loss of muscle function. Those living with the condition often require special aid and assistance throughout their lives and have significantly shortened life expectancy.
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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,” said Sarah Boyce, president and chief executive officer. “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.”
AOC 1044 is designed to deliver phosphorodiamidate morpholino oligomers (PMO) to skeletal muscle and heart tissue to specifically skip exon 44 of DMD to enable dystrophin production. AOC 1044 is Avidity’s first AOC engineered to deliver PMO and the company has two additional programs for DMD, which target exon 45 and exon 51.