Defeat Duchenne Canada looks forward to updated data from Avidity Biosciences who plans to share the safety and effectiveness data from their Phase 1/2 EXPLORE44 study of their investigational product delpacibart zotadirsen or del-zota. Del-zota is an Antibody Oligonucleotide Conjugate, a type of medicine that combines a monoclonal antibody (a protein made in a laboratory that acts like protein that occurs naturally in our bodies) and an oligonucleotides (short strands of DNA or RNA). Del-zota is designed to deliver phosphorodiamidate morpholino oligomers (PMOs, a molecule that is used to modify how a gene works) to skeletal muscle and heart tissue to specifically skip exon 44 of the dystrophin gene. The aim of del-zota is to increase the production of dystrophin for people with DMD who have mutations impacting exon 44.
“Successful readouts from our three clinical-stage programs in 2024 demonstrate the consistent and reproducible data of our AOC platform. We are extending our leadership position in the rare neuromuscular space as we plan to submit our first BLA for an AOC and prepare for three potential successive product launches to provide therapies for people living with rare neuromuscular diseases with limited or no treatment options”
Sarah Boyce, President and CEO, Avidity
Defeat Duchenne Canada looks forward to further updates from Avidity Biosciences and will share them with our audience as they become available. Please read the full press release below.
About AOC 1044
AOC 1044 is currently in Phase 1/2 development as part of the EXPLORE44™ trial for the treatment of DMD mutations amenable to exon 44 skipping. Data from the Phase 1/2 EXPLORE44 trial showed that AOC 1044 delivered unprecedented concentrations of PMO in skeletal muscle with up to 50-times greater concentrations of PMO in skeletal muscle following a single dose compared to peptide conjugated PMOs in healthy volunteers. AOC 1044 was well tolerated, demonstrated statistically significant exon 44 skipping compared to placebo of up to 1.5% in healthy volunteers after a single dose of 10 mg/kg AOC 1044 and increased exon skipping in all participants. Avidity plans to provide a first look at AOC 1044 data in people living with DMD44 in 2H 2024.
Want to know more about Duchenne Research?
Scientists and medical experts have been tirelessly pursuing diverse approaches to defeat Duchenne muscular dystrophy. From tackling the root cause to alleviating symptoms, the field of research is vast and promising. Explore the various strategies being developed and tested, and gain insights into the future of muscular dystrophy treatment: