AOC-1044 is under clinical development by Avidity Biosciences and currently in Phase II for Duchenne Muscular Dystrophy. According to GlobalData, Phase II drugs for Duchenne Muscular Dystrophy have a 65% phase transition success rate (PTSR) indication benchmark for progressing into Phase III. GlobalData’s report assesses how AOC-1044’s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks. Buy the report here.

GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval.

AOC-1044 overview

AOC-1044 is under development for the treatment of Duchenne muscular dystrophy. The drug candidate is an antibody oligonucleotide conjugate (AOC) comprising monoclonal antibody that binds to the transferrin receptor 1 (TfR1) conjugated with PMO targeting exon 44 of dystrophin mRNA.

Avidity Biosciences overview

Avidity Biosciences, formerly Avidity NanoMedicines is a biotech company that develops antibody oligonucleotide conjugates (AOCs). Its proprietary platform technology has enhanced pharmacokinetic and biodistribution properties that improve the delivery of multiple therapeutic oligonucleotides. Its pipeline of therapeutic programs is intended for the treatment of rare muscle disorders and other serious diseases, including Duchenne muscular dystrophy, myotonic dystrophy and muscle atrophy. The company has research collaborations with leading pharma companies, to discover antibody-based drug candidates against various therapeutic targets. Avidity Biosciences is headquartered in San Diego, California, the US.

For a complete picture of AOC-1044’s drug-specific PTSR and LoA scores, buy the report here.


GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

GlobalData’s Likelihood of Approval analytics tool dynamically assesses and predicts how likely a drug will move to the next stage in its clinical pathway (PTSR), as well as how likely the drug will be approved (LoA). This is based on a proprietary algorithm built from the drugs’ sales forecast, regulatory milestones, cost forecasts, WACC rate and other proprietary data sources found on GlobalData’s Pharmaceutical Intelligence Center.