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Also known as AOC 1001 (del-desiran)
AOC 1001 · 3 trials · 19 indications
video Hand Opening Time (vHOT)
Cumulative number of participants with treatment emergent adverse events at the end of study.
| Arm | Type | Description |
|---|---|---|
| Del-desiran | EXPERIMENTAL | Del-desiran (AOC 1001) will be administered seven times |
| Placebo | PLACEBO_COMPARATOR | Saline will be administered seven times |
| AOC 1001 | EXPERIMENTAL | AOC 1001 will initially be administered quarterly. On Day 43 patients will receive an additional dose. Treatment assignment will be based on treatment received in AOC 1001-CS1. If participant did not receive AOC 1001 on Day 43 in AOC 1001-CS1, participant will receive AOC 1001 treatment on Day 43 in AOC 1001-CS2. Beginning in September 2024, AOC 1001 will be administered every 8 weeks. |
| AOC 1001 (with Placebo at Day 43) | EXPERIMENTAL | AOC 1001 will initially be administered quarterly. On Day 43 patients will receive an additional dose. Treatment assignment will be based on treatment received in AOC 1001-CS1. If participant received AOC 1001 on Day 43 in AOC 1001-CS1, participant will receive blinded placebo treatment on Day 43 in AOC 1001-CS2. Beginning in September 2024, AOC 1001 will be administered every 8 weeks. |
| Part A Single Dose: AOC 1001 Dose Level 1 | EXPERIMENTAL | AOC 1001 will be administered once. |
| Part A Single Dose: Placebo | PLACEBO_COMPARATOR | Saline will be administered once. |
| Part B Multiple Ascending Dose: AOC 1001 Dose Levels 2 & 3 | EXPERIMENTAL | AOC 1001 will be administered three times. |
| Part B Multiple Ascending Dose: Placebo | PLACEBO_COMPARATOR | Saline will be administered three times. |
| Name | Type | Description |
|---|---|---|
| AOC 1001 (del-desiran) | DRUG | Del-desiran will be administered by intravenous (IV) infusion. |
| Placebo | DRUG | Placebo will be administered by intravenous (IV) infusion. |
| AOC 1001 | DRUG | AOC 1001 will be administered by intravenous (IV) infusion. |
Key Inclusion Criteria: * Clinical and genetic diagnosis (CTG repeat ≥ 100) of DM1 * Ability to walk independently (orthoses and ankle braces allowed) for at least 10 meters at screening Key Exclusion Criteria: * Breastfeeding, pregnancy, or intent to become pregnant during the study * Unwilling ...
AOC 1001 is an investigational therapy being developed for myotonic dystrophy type 1 (DM1), also called Steinert disease. DM1 is a rare genetic neuromuscular disorder. The drug is designed to address the underlying cause of the condition rather than only managing symptoms, and it is being studied in adults and adolescents aged 16 and older.
AOC 1001 targets DMPK, the gene that encodes dystrophia myotonica protein kinase. In DM1, the DMPK gene produces toxic expanded RNA repeats that interfere with normal cellular function. By targeting DMPK at the gene level, AOC 1001 aims to reduce production of the toxic RNA that drives the disease.
AOC 1001 is being developed by Atrium Therapeutics, Inc., which trades under the ticker RNA. The company is advancing the candidate for myotonic dystrophy type 1 as part of its rare disease portfolio.
AOC 1001 is in Phase 3 development for myotonic dystrophy type 1. It is an investigational drug and has not been approved by the FDA. Three clinical trials have been completed to date, including a Phase 1, a Phase 2 extension, and a global Phase 3 study.
AOC 1001 has been studied in three completed trials: NCT05027269 (Phase 1), NCT05479981 (Phase 2 extension of the MARINA study), and NCT06411288 (Phase 3 global study). The Phase 3 trial enrolled 159 patients across the United States, Canada, Europe, and Japan. Total enrollment across all three trials was 235 participants.
Yes, AOC 1001 is also known as del-desiran. Del-desiran is the international nonproprietary name for the same investigational compound developed by Atrium Therapeutics for myotonic dystrophy type 1. Both names refer to the identical drug candidate studied in the Phase 1, Phase 2, and Phase 3 clinical trials.