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AOC-1020

Phase 3

Facioscapulohumeral Muscular Dystrophy | Small molecule | Neurology |Atrium Therapeutics, Inc.|Last Updated: Jul 27, 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 Trials1
Total Enrollment200

FDA Designations

FAST_TRACKORPHAN_DRUG

Clinical trial landscape

AOC-1020 · 1 trial · 17 indications

Phase 3 1
NCT07038200A Study to Evaluate Del-brax (Also Referred to as AOC 1020) in Participants With FSHDFacioscapulohumeral Muscular Dystrophy
RECRUITING200 Analytics
PHASE3RECRUITING
A Study to Evaluate Del-brax (Also Referred to as AOC 1020) in Participants With FSHD
Facioscapulohumeral Muscular DystrophyUnlock trial analytics

Study Endpoints

Primary Endpoints

Change from Baseline to Week 78 in Quantitative Muscle Testing (QMT) total composite score (PPN)
Baseline through Week 78

Assessment of muscle strength using standardized quantitative measurements

Secondary Endpoints

Change from Baseline to Week 78 in 10MWRT
Baseline through Week 78
Change from Baseline to Week 78 in Timed Up-and-Go (TUG)
Baseline through Week 78
Change from Baseline to Week 78 in Patient-Reported Outcomes Measurement Information System (PROMIS) Physical Function Item Bank v2.0-Physical Function-Short Form 20a Questionnaire
Baseline through Week 78
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Del-braxEXPERIMENTALDel-brax (AOC 1020) will be administered 13 times
PlaceboPLACEBO_COMPARATORSaline will be administered 13 times

Interventions

NameTypeDescription
AOC-1020DRUGDel-brax will be administered by intravenous (IV) infusion.
PlaceboDRUGPlacebo will be administered by intravenous (IV) infusion.
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Eligibility Criteria

Age Range16 Years to 70 Years
SexALL
Healthy VolunteersNo
Study Sites46

Inclusion Criteria: * Clinical and genetic diagnosis of FSHD1 or FSHD2 * Ability to walk independently at pre-specified walking speed (orthoses and ankle braces allowed) for at least 10 meters at screening * Adequate muscle strength based on QMT composite score Exclusion Criteria: * Breastfeeding...

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

MEDIUMJul 27, 2026NCT07038200lastUpdatePostDate: changed
MEDIUMJul 27, 2026NCT07038200lastUpdatePostDate: changed
MEDIUMJul 27, 2026NCT07038200lastUpdatePostDate: 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.