Antibody-drug Conjugate Information

General Information of This Antibody-drug Conjugate (ADC)
ADC ID
DRG0AVJCO
ADC Name
AOC-1020
Synonyms
AOC 1020; AOC-1020; AOC-FSHD; AOC1020
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Drug Status
Phase 2
Antibody Name
Delpacibart
  Antibody Info 
Antigen Name
Transferrin receptor protein 1 (TFRC)
  Antigen Info 
Payload Name
DUX4 siRNA
  Payload Info 
Therapeutic Target
Double homeobox protein 4 (DUX4)
  Target Info 
Linker Name
Undisclosed
Conjugate Type
Random conjugation through reduced inter-chain cysteines.
Combination Type
Braxlosiran
Special Approval(s)
Fast track (FDA); Orphan drug (FDA)
The indication landscape of This ADC(2027 Update)
The clinical trial pipeline of This ADC(2027 Update)
Indication Phase 1 Phase 1/2 Phase 2 Phase 2/3 Phase 3 New Drug Application Approved
Cardiovascular
1 Trials
Trial ID
NCT05747924
1 Trials
Trial ID
NCT06547216
General Information of The Activity Data Related to This ADC
Identified from the Human Clinical Data
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Standard Type NCT Number Clinical Status Clinical Trial Description
Undisclosed  NCT05747924
Phase 1
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 adult participants with facioscapulohumeral muscular dystrophy (FSHD).

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Undisclosed  NCT05747924
PHASE1|||PHASE2
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)

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Undisclosed  NCT06547216
PHASE2
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)

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Full List of Activity Data of This Antibody-drug Conjugate
Identified from the Human Clinical Data
Click To Hide/Show 3 Activity Data Related to This Level
Experiment 1 Reporting the Activity Date of This ADC [1]
Related Clinical Trial
NCT Number NCT05747924  Clinical Status Phase 1
Clinical Description 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 adult participants with facioscapulohumeral muscular dystrophy (FSHD).
Experiment 2 Reporting the Activity Date of This ADC [2]
Patients Enrolled
Key inclusion: Genetically confirmed FSHD1/2 (sponsor-tested), ambulatory (10m walk capacity), ≥1 biopsy-suitable muscle region, and documented upper/lower body weakness
Administration Dosage
AOC 1020 Dose Regimen 1; Five doses administered intravenously over 9 months
Related Clinical Trial
NCT Number NCT05747924  Clinical Status PHASE1|||PHASE2
Clinical Description 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)
Primary Endpoint
Primary endpoints include treatment-emergent AE incidence (Cohorts A/B) [up to Day 365], DUX4-regulated gene expression changes in muscle biopsies (Cohort C) [Day 120], and circulating FSHD biomarker changes (Cohort C) [Day 120].
Other Endpoint
Secondary endpoints comprise AOC 1020 PK parameters (Cmax, half-life, AUC [up to Day 365]), muscle drug concentration [Day 120], and creatine kinase level changes [up to Day 365].
Experiment 3 Reporting the Activity Date of This ADC [3]
Patients Enrolled
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 designated representative and the participant will receive information in a way adapted to their age and mental maturity.

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Administration Dosage
AOC 1020 Dose Regimen; Sixteen doses administered intravenously over 22 months. All participants will receive AOC 1020 at a dose level of 2mg/kg.
Related Clinical Trial
NCT Number NCT06547216  Clinical Status PHASE2
Clinical Description 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)
Primary Endpoint
Incidence of treatment-emergent adverse events [Time Frame: Through study completion, up to Day 729]
References
Ref 1 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 Adult Participants With Facioscapulohumeral Muscular Dystrophy (FSHD), NCT05747924
Ref 2 Phase 1/2 Study of AOC 1020 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)
Ref 3 Phase 2 Open-label Extension Study of AOC 1020 in Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)