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AOC 1044 · 1 trial · 14 indications
| Arm | Type | Description |
|---|---|---|
| AOC 1044 | EXPERIMENTAL | Participants will receive AOC 1044 intravenously (IV) every 6 weeks (Q6W) during the 54-week double-blind treatment period, followed by a 54-week open-label treatment period in which the same participants will continue to receive AOC 1044 IV Q6W |
| Placebo | PLACEBO_COMPARATOR | Participants will receive placebo (saline) IV Q6W during the 54-week double-blind treatment period, followed by a 54-week open-label treatment period in which all participants will then receive AOC 1044 IV Q6W |
| Name | Type | Description |
|---|---|---|
| AOC 1044 | DRUG | AOC 1044 will be administered by intravenous (IV) infusion |
| Placebo | DRUG | Placebo will be administered by intravenous (IV) infusion |
Key Inclusion Criteria: * Ambulatory males with clinical and genetic diagnosis of DMD * Acceptable genetic test confirming dystrophin gene mutation amenable to exon 44 skipping * 7 to 16 years of age at time of consent * TTR and NSAA assessment completed within the protocol specified parameters at ...
AOC 1044 is an investigational therapy being developed for Duchenne muscular dystrophy (DMD) in people with gene mutations amenable to exon 44 skipping. It is in Phase 3 clinical development and is not yet approved for any indication.
AOC 1044 targets dystrophin, the protein missing or defective in Duchenne muscular dystrophy. By targeting dystrophin production through exon 44 skipping, the therapy aims to address the underlying cause of the disease in patients with specific genetic mutations.
AOC 1044 is being developed by Avidity Biosciences, Inc., which trades on the Nasdaq under the ticker RNAM. The company is advancing the program for muscular dystrophies, specifically Duchenne muscular dystrophy with exon 44 skipping mutations.
AOC 1044 is in Phase 3 clinical development. The ongoing trial is a randomized, double-blind, placebo-controlled study, indicating it has advanced to late-stage testing. It is not yet approved by the FDA or any regulatory agency.
AOC 1044 is being evaluated in a Phase 3 study, NCT07587242, titled "A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 Skipping." The trial is currently recruiting and plans to enroll 70 male participants aged 7 years and older across multiple countries.
Yes, AOC 1044 is also known as delpacibart zotadirsen. The two names refer to the same investigational therapy being developed by Avidity Biosciences for Duchenne muscular dystrophy with exon 44 skipping mutations.