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AOC 1044 · 2 trials · 4 indications
| Arm | Type | Description |
|---|---|---|
| AOC 1044 Multiple Dose Levels | EXPERIMENTAL | AOC 1044 will be IV infused every 6 weeks for approximately 2 years. |
| AOC 1044-CS1 Part A - Single Dose Levels 1-5 | EXPERIMENTAL | AOC 1044 will be administered once. |
| AOC 1044-CS1 Part A - Single Dose: Placebo | PLACEBO_COMPARATOR | Placebo will be administered once. |
| AOC 1044-CS1 Part B - Multiple Ascending Dose Levels 1-3 | EXPERIMENTAL | AOC 1044 will be administered three times. |
| AOC 1044-CS1 Part B - Multiple Ascending Dose: Placebo | PLACEBO_COMPARATOR | Placebo will be administered three times. |
| Name | Type | Description |
|---|---|---|
| AOC 1044 | DRUG | AOC 1044 will be administered via intravenous (IV) infusion |
| Placebo | DRUG | Placebo will be administered via intravenous (IV) infusion. |
Key Inclusion Criteria Rollover Participants: * Satisfactorily completed AOC 1044-CS1 (EXPLORE44) as determined by the Investigator and Sponsor * No significant tolerability issues with AOC 1044 De novo Participants: * Aged 7 to 27 years, inclusive, at the time of informed consent * Clinical dia...
AOC 1044 is an investigational small molecule being developed for Duchenne Muscular Dystrophy (DMD), specifically for patients with mutations amenable to exon 44 skipping. It is designed to target the dystrophin protein, which is deficient in DMD. The drug is currently in Phase 2 clinical development.
AOC 1044 targets the dystrophin protein, which is critical for muscle function and is deficient in Duchenne Muscular Dystrophy. By targeting dystrophin, the drug aims to address the underlying cause of the disease in patients with mutations amenable to exon 44 skipping.
AOC 1044 is being developed by Atrium Therapeutics, Inc., a biopharmaceutical company. The company is conducting clinical trials for this drug in the United States, focusing on Duchenne Muscular Dystrophy patients with exon 44 skipping mutations.
AOC 1044 is currently in Phase 2 clinical development. It has received Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the FDA. The drug is investigational and not yet approved for commercial use.
AOC 1044 has been studied in two clinical trials. NCT05670730 was a Phase 1 study in healthy volunteers and DMD patients, which is completed. NCT06244082 is an ongoing Phase 2 open-label study in DMD patients with exon 44 skipping mutations, with an enrollment of 39 participants.
AOC 1044 is a distinct investigational drug specifically designed for Duchenne Muscular Dystrophy patients with mutations amenable to exon 44 skipping. It is not the same as other DMD treatments, as it targets the dystrophin protein through a unique mechanism for this patient subgroup.