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Also known as AOC 1001 (del-desiran)
AOC 1001 · 3 trials · 19 indications
video Hand Opening Time (vHOT)
| 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, also known as del-desiran, is an investigational small molecule being developed for the treatment of myotonic dystrophy type 1 (DM1), a rare genetic disorder. It is currently in Phase 3 clinical development and has not been approved by regulatory authorities.
AOC 1001 targets the DMPK gene, which is implicated in myotonic dystrophy type 1 (DM1). By targeting this gene, the therapy aims to address the underlying cause of the disease. The drug is being studied in patients with DM1.
AOC 1001 is being developed by Atrium Therapeutics, Inc., a biopharmaceutical company. The company's stock is traded under the ticker symbol RNA. Atrium Therapeutics is conducting clinical trials to evaluate the safety and efficacy of AOC 1001 in patients with DM1.
AOC 1001 is currently in Phase 3 clinical development for the treatment of myotonic dystrophy type 1 (DM1). It is an investigational drug and has not yet received regulatory approval. The Phase 3 trial is a global, randomized, double-blind, placebo-controlled study.
AOC 1001 has been studied in three clinical trials. The Phase 1 trial (NCT05027269) and a Phase 2 extension study (NCT05479981) have been completed. A Phase 3 trial (NCT06411288) has also been completed, enrolling 159 participants across multiple countries.
Yes, AOC 1001 is also known as del-desiran. Both names refer to the same investigational drug being developed for myotonic dystrophy type 1 (DM1). The drug is currently in Phase 3 clinical development and has not been approved.