Payload Information

General Information of This Payload
Payload ID
PAY0JRATG
Name
DUX4 siRNA
Synonyms
DUX4 siRNA
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Target Double homeobox protein 4 (DUX4)
 Target Info 
The activity data of This Payload
Standard Type Value Units Cell line Disease Model Cell line ID Reference
Methylation Fold Change ≈2.5 .
FMUFAHi001-A cells
Facioscapulohumeral dystrophy
CVCL_C0FU 
[1]
Each Antibody-drug Conjugate Related to This Payload
Full Information of The Activity Data of The ADC(s) Related to This Payload
AOC-1020 [Phase 2]
 ADC Info 
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 [2]
Related Clinical Trial
NCT Number NCT05747924  Phase 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 [3]
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  Phase 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 [4]
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  Phase 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 DICER/AGO-dependent epigenetic silencing of D4Z4 repeats enhanced by exogenous siRNA suggests mechanisms and therapies for FSHD. Hum Mol Genet. 2015 Sep 1;24(17):4817-28.
Ref 2 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 3 Phase 1/2 Study of AOC 1020 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)
Ref 4 Phase 2 Open-label Extension Study of AOC 1020 in Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)