Pegzilarginase

Summary

Pegzilarginase (CHEMBL4297803) is an approved enzyme (ATC A16AB24); indicated across 6 conditions including arginase deficiency and myelodysplastic syndrome.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Enzyme
• ATC class: A16AB24
• Indications: 6 conditions
• Clinical trials: 7

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL4297803
Name	Pegzilarginase
Type	Enzyme
Max phase	4
ATC	A16AB24

Also known as: AEB-1102, Aeb1102, AEB1102, Co-argi-peg, Loargys, Pegzilarginasa, Pegzilarginase, PEGZILARGINASE

Targets

Targets

No target linkage available.

Bioactivity

No ChEMBL bioactivity rows at pChembl ≥ 5 (expected for biologics / antibodies).

Target pathways

No target-pathway data for this drug (no mapped target genes).

Indications & clinical

Indications

6 indications (1 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).

Indication	Trial phase	MONDO	EFO
arginase deficiency	4	MONDO:0008814	Orphanet:90
myelodysplastic syndrome	2	MONDO:0018881	EFO:0000198
acute myeloid leukemia	2	MONDO:0018874	EFO:0000222
small cell lung carcinoma	1	MONDO:0008433	EFO:0000702
neoplasm	1	MONDO:0005070	MONDO:0004992

1 further indication record had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.

Clinical trials

Total trials: 7.

Phase distribution

Phase	Trials
PHASE3	3
PHASE1/PHASE2	3
PHASE2	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT03921541	PHASE3	COMPLETED	Efficacy and Safety of Pegzilarginase in Patients With Arginase 1 Deficiency
NCT05676853	PHASE3	TERMINATED	A Study of Safety of Weekly Subcutaneous Pegzilarginase in Subjects With Arginase 1 Deficiency
NCT06582524	PHASE3	COMPLETED	Pegzilarginase in Subjects <24 Months Old With Arginase 1 Deficiency
NCT02488044	PHASE1/PHASE2	COMPLETED	A Phase 1/2 Study of AEB1102 in Patients With Arginase I Deficiency
NCT02561234	PHASE1/PHASE2	COMPLETED	A Multiple Dose, Dose Escalation Trial of AEB1102 in Patients With Advanced Solid Tumors
NCT03371979	PHASE1/PHASE2	COMPLETED	Pegzilarginase and Pembrolizumab for Extensive Disease Small Cell Lung Cancer
NCT03378531	PHASE2	COMPLETED	A Study of AEB1102 (Pegzilarginase) in Patients With Arginase I Deficiency

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No PharmGKB pharmacogenomic data curated for this drug.

Related molecules

Related molecules

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).