AOC 1020

Phase 3Small molecule

AOC 1020 is one of 6 small molecule programs targeting Facioscapulohumeral Muscular Dystrophy. It is currently in Phase 3.

Indications

Total Trials

Active Trials

Competitors

Pipeline

Status by Indication[3][1]

Indication

Facioscapulohumeral Muscular DystrophyRecruiting

FSHDActive, not recruiting

DMDActive, not recruiting

Facioscapulohumeral Muscular DystrophyPhase 3

Recruiting

FSHDPhase 2

Active, not recruiting

DMDPhase 2

Active, not recruiting

Competition

Competitive Landscape

3 competitors in Facioscapulohumeral Muscular Dystrophy

#DrugCompanyPhaseTrials

AOC 1020

AAvidity Biosciences, Inc.

Phase 3

1 2

Apitegromab

SScholar Rock, Inc.

Phase 2

1 3

Proair HFA

GGlaxo Dba Glaxosmithkline

Preclinical

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q2 2027

Phase 2Active, not recruiting

DMD

NCT06244082

Q3 2028

Phase 3Recruiting

Facioscapulohumeral Muscular Dystrophy

NCT07038200

Q2 2030

Phase 2Active, not recruiting

FSHD

NCT06547216

Clinical

Trials by Indication[1]

DM12 trials

A Phase 2 Extension Study to Evaluate the Long-Term Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) Patients

Completed·AAvidity Biosciences, Inc.·Phase 2·NCT05479981·37 enrolled

A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Global Study to Evaluate the Efficacy and Safety of Intravenous AOC 1001 for the Treatment of Myotonic Dystrophy Type 1

Completed·AAvidity Biosciences, Inc.·Phase 3·NCT06411288·159 enrolled

FSHD2 trials

A Randomized, Double-blind, Placebo-controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of AOC 1020 Administered Intravenously to Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)

Completed·AAvidity Biosciences, Inc.·Preclinical·NCT05747924·90 enrolled

A Phase 2 Open-label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of AOC 1020 Administered Intravenously to Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)

Active, not recruiting·AAvidity Biosciences, Inc.·Phase 2·NCT06547216·84 enrolled

Myotonic Dystrophy Type 11 trial

Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients

Completed·AAvidity Biosciences, Inc.·Phase 1/2·NCT05027269·39 enrolled

Duchenne Muscular Dystrophy1 trial

Study of AOC 1044 in Healthy Adult Volunteers and Participants With Duchenne Muscular Dystrophy (DMD) Mutations Amenable to Exon 44 Skipping

Completed·AAvidity Biosciences, Inc.·Phase 1/2·NCT05670730·70 enrolled

DMD1 trial

A Phase 2 Open-label Study to Evaluate the Pharmacodynamics and Long-Term Safety and Tolerability of AOC 1044 Administered Intravenously to DMD Participants With Mutations Amenable to Exon 44 Skipping

Active, not recruiting·AAvidity Biosciences, Inc.·Phase 2·NCT06244082·39 enrolled

1 more indication

Facioscapulohumeral Muscular Dystrophy1 trial

A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1020 for the Treatment of Facioscapulohumeral Muscular Dystrophy (FSHD)

Recruiting·AAvidity Biosciences, Inc.·Phase 3·NCT07038200·200 enrolled

Sources

References

[1]
ClinicalTrials.gov — 8 trial records for AOC 1020. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — AOC 1020 compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for AOC 1020. EMBL-EBI & Wellcome Sanger Institute.
[4]
SEC EDGAR — Avidity Biosciences, Inc. company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Small molecule[2]
Highest Phase: Phase 3[1]
Company: AAvidity Biosciences, Inc.

AOC 1020

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q2 2027

Q3 2028

Q2 2030

Trials by Indication[1]

DM12 trials

FSHD2 trials

Myotonic Dystrophy Type 11 trial

Duchenne Muscular Dystrophy1 trial

DMD1 trial

Facioscapulohumeral Muscular Dystrophy1 trial

References

Drug Info

Quick Facts

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