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AOC 1020

Phase 2

FSHD | Small molecule | Neurology |Atrium Therapeutics, Inc.|Last Updated: Jul 16, 2026

Target and mechanism

ModalitySmall molecule

Also known as AOC-1020

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials2
Total Enrollment174

FDA Designations

FAST_TRACKORPHAN_DRUG

Clinical trial landscape

AOC 1020 · 2 trials · 27 indications

Phase 2 1Phase 1 1
NCT06547216Phase 2 Open-label Extension Study of AOC 1020 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)FSHD
ACTIVE NOT_RECRUITING84 Analytics
PHASE2ACTIVE NOT_RECRUITING
Phase 2 Open-label Extension Study of AOC 1020 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)
FSHDUnlock trial analytics

Study Endpoints

Primary Endpoints

Incidence of treatment-emergent adverse events
Through study completion, up to Day 729
Incidence of treatment-emergent adverse events (Cohorts A & B)
Through study completion, up to Day 365
Change in plasma KHDC1L (Part C)
Across months 3 to 12

Ratio to Baseline

Secondary Endpoints

Plasma pharmacokinetic (PK) parameters of AOC 1020 (Cohorts A & B)
Through study completion; up to Day 365
Muscle drug concentration (Cohorts A & B)
Day 120
Change in circulating creatine kinase (Cohort C)
Across months 3 to 12
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
AOC 1020 RegimenEXPERIMENTALAOC 1020 Dose Regimen; Thirty-two doses administered intravenously over 46 months. All participants will receive AOC 1020 at a dose level of 2mg/kg.
AOC 1020 Regimen 1EXPERIMENTALCohort A: AOC 1020 Dose Regimen 1; Five doses administered intravenously over 9 months
AOC 1020 Regimen 2EXPERIMENTALCohort B1: AOC 1020 Dose Regimen 2; Five doses administered intravenously over 9 months
AOC 1020 Regimen 3EXPERIMENTALCohort C: AOC 1020 Dose Regimen 3; Eight doses administered intravenously over approximately 10 months
Placebo (Saline) Regimen 1PLACEBO_COMPARATORCohort A \& B: Placebo; Five doses administered intravenously over 9 months
Placebo (Saline) Regimen 2PLACEBO_COMPARATORCohort C: Placebo; Eight doses administered intravenously over approximately 10 months

Interventions

NameTypeDescription
AOC 1020DRUGAOC 1020 will be administered via intravenous (IV) infusion
PlaceboDRUGPlacebo will be administered via intravenous (IV) infusion
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Eligibility Criteria

Age Range16 Years to 72 Years
SexALL
Healthy VolunteersNo
Study Sites17

Inclusion Criteria: 1. Ability to provide written informed consent (signed and dated) and any authorizations required by local law and be willing and able to comply with all study requirements. When enrolling participants who are minors, it is necessary to also obtain consent from a legally designa...

Countries:United StatesCanadaUnited Kingdom
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Recent Changes (Last 90 Days)

MEDIUMAug 16, 2026NCT05747924TRIAL_REMOVED: changed
MEDIUMAug 16, 2026NCT05747924TRIAL_REMOVED: changed
MEDIUMAug 16, 2026NCT05747924TRIAL_REMOVED: changed
MEDIUMAug 16, 2026NCT05747924TRIAL_REMOVED: changed
LOWJul 14, 2026NCT06547216lastUpdatePostDate: changed
LOWJul 14, 2026NCT06547216lastUpdatePostDate: changed

Frequently asked questions about AOC 1020

What is AOC 1020 used for?

AOC 1020 is an investigational small molecule being developed for the treatment of Facioscapulohumeral Muscular Dystrophy (FSHD), a neurological condition. It is currently in Phase 2 clinical development and has received Fast Track and Orphan Drug designations from the FDA.

Who makes AOC 1020?

AOC 1020 is being developed by Atrium Therapeutics, Inc., a biopharmaceutical company. The company's stock is traded under the ticker symbol RNA.

What phase is AOC 1020 in?

AOC 1020 is currently in Phase 2 clinical development. It has completed a Phase 1/2 study and is being evaluated in an ongoing Phase 2 open-label extension study for FSHD. It is not yet approved by the FDA.

What clinical trials is AOC 1020 in?

AOC 1020 has been studied in clinical trials including NCT05747924, a completed Phase 1/2 study in participants with FSHD, and NCT06547216, an active Phase 2 open-label extension study. These trials have enrolled participants in the United States, Canada, and the United Kingdom.

Is AOC 1020 the same as Del-brax?

Yes, AOC 1020 is also referred to as Del-brax. A Phase 3 clinical trial (NCT07038200) is currently recruiting participants to evaluate Del-brax, also known as AOC 1020, in individuals with FSHD.