Amrubicin

Summary

Amrubicin (CHEMBL1186894) is a phase-3 clinical-stage small-molecule topoisomerase II inhibitor (ATC L01DB10); indicated across 9 conditions including neoplasm and small cell lung carcinoma; with CIViC clinical evidence for 2 variant-indication associations (e.g. NQO1 P187S in lung cancer).

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Small molecule
• ATC class: L01DB10
• Indications: 9 conditions
• Clinical trials: 19
• Precision-oncology evidence (CIViC): 2 variant–indication associations
• Chemistry: 483.5 Da · C25H25NO9

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL1186894
Name	Amrubicin
Type	Small molecule
Max phase	3
FDA approved	no
PubChem CID	3035016
ChEBI	CHEBI:135779
ATC	L01DB10
Molecular formula	C25H25NO9
Molecular weight	483.5
InChIKey	VJZITPJGSQKZMX-XDPRQOKASA-N

SMILES: CC(=O)[C@]1(C[C@@H](C2=C(C1)C(=C3C(=C2O)C(=O)C4=CC=CC=C4C3=O)O)O[C@H]5C[C@@H]([C@@H](CO5)O)O)N

IUPAC name: (7S,9S)-9-acetyl-9-amino-7-[(2S,4S,5R)-4,5-dihydroxyoxan-2-yl]oxy-6,11-dihydroxy-8,10-dihydro-7H-tetracene-5,12-dione

ChEBI definition: A synthetic anthracycline antibiotic with molecular formula C25H25NO9. A specific inhibitor of topoisomerase II, it is used (particularly as the hydrochloride salt) in the treatment of cancer, especially lung cancer, where it is a prodrug for the active metabolite, ambrucinol.

Pharmacological roles (ChEBI): topoisomerase II inhibitor, antineoplastic agent, prodrug.

Also known as: Amrubicin, Amrubicina, Amrubicine, AMRUBICIN, amrubicin

Parent form; salt/anhydrous children: CHEMBL481958

Patent coverage: 5,258 distinct patent families (22,882 SureChEMBL compound mentions), from 1 matched compound structure(s). Mentions count patents naming the compound (not distinct inventions), so promiscuous / reference molecules inflate the mention figure — families are the dedup metric.

Targets

Targets

Broader ChEMBL bioactivity targets: 4 (assay-derived). Sample: Thromboxane A2 receptor, Muscarinic acetylcholine receptor M1, Prostaglandin G/H synthase 1, Alpha-1A adrenergic receptor.

Bioactivity

ChEMBL activities: 2 potent at pChembl ≥ 5 of 5 total. Top 100 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
PTGS1	5.55	AC50	2815	nM	CHEMBL_ACT_25206473
PTGS1	5.44	AC50	3621	nM	CHEMBL_ACT_25205543

Target pathways

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

Indications & clinical

Indications

8 diseases in clinical trials (phase 1–3, investigational — not approved indications). Highest ChEMBL trial phase per disease; a non-cancer approved use is occasionally logged at phase 3 here.

Disease (in trials)	Phase	MONDO	EFO
neoplasm	3	MONDO:0005070	EFO:0000616
small cell lung carcinoma	3	MONDO:0008433	EFO:0000702
lung neoplasm	3	MONDO:0021117	MONDO:0008903
soft tissue sarcoma	2	MONDO:0018078	EFO:1001968
thymic carcinoma	2	MONDO:0006451	EFO:1000576
thymoma	2	MONDO:0006456	EFO:1000581
breast neoplasm	2	MONDO:0021100	MONDO:0007254
plasma cell myeloma	1	MONDO:0009693	EFO:0001378

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

Clinical trials

Total trials: 19.

Phase distribution

Phase	Trials
PHASE2	9
PHASE3	6
PHASE1/PHASE2	2
PHASE1	2

Top trials by phase / activity

NCT	Phase	Status	Title
NCT05740566	PHASE3	ACTIVE_NOT_RECRUITING	Study Comparing Tarlatamab With Standard of Care Chemotherapy in Relapsed Small Cell Lung Cancer
NCT06203210	PHASE3	RECRUITING	A Study of Ifinatamab Deruxtecan Versus Treatment of Physician’s Choice in Subjects With Relapsed Small Cell Lung Cancer
NCT07365241	PHASE3	NOT_YET_RECRUITING	A Study to Evaluate Adverse Events and Change in Disease Activity of Intravenous ABBV-706 Versus Standard of Care in Adult Participants With Relapsed/Refractory Small Cell Lung Cancer
NCT00547651	PHASE3	COMPLETED	AMR PH GL 2007 CL001 Phase 3 Trial in Patients With Small Cell Lung Cancer After Failure of First-Line Chemotherapy
NCT01207011	PHASE3	COMPLETED	Amrubicin (AMR) Trial in Patients With Non-small Cell Lung Cancer
NCT02481830	PHASE3	COMPLETED	Effectiveness Study of Nivolumab Compared to Chemotherapy in Patients With Relapsed Small-cell Lung Cancer
NCT00319969	PHASE2	COMPLETED	Study Comparing Amrubicin Versus Topotecan in Patients With Small Cell Lung Cancer Who Have Responded to Prior Therapy.
NCT00375193	PHASE2	COMPLETED	Study of Amrubicin in Patients With Small Cell Lung Cancer Refractory or Progressive to Prior Therapy
NCT00380835	PHASE2	WITHDRAWN	Study of Amrubicin With or Without Herceptin in Patients With Metastatic Breast Cancer
NCT00388960	PHASE2	COMPLETED	Study of Amrubicin With or Without Cisplatin Versus Etoposide-cisplatin for Extensive Stage Small Cell Lung Cancer
NCT01033032	PHASE1/PHASE2	COMPLETED	Trial of Amrubicin as Treatment for Patients With HER2-Negative Metastatic Breast Cancer
NCT01076504	PHASE2	COMPLETED	A Trial of Amrubicin and Carboplatin With Pegfilgrastim in Patients With Extensive-Stage Small Cell Lung Cancer
NCT01259375	PHASE2	COMPLETED	Amrubicin Chemotherapy as First Line in Metastatic or Unresectable Soft Tissue Sarcoma
NCT01331824	PHASE2	TERMINATED	Trial of Amrubicin as Second-Line Therapy in Patients With Advanced/Metastatic Refractory Urothelial Carcinoma
NCT01355705	PHASE1/PHASE2	COMPLETED	Phase 1-2 Amrubicin in Combo With Lenalidomide + Weekly Dexamethasone in Relapsed/Refractory Multiple Myeloma
NCT01364727	PHASE2	COMPLETED	A Phase 2 Study of Amrubicin in Relapsed or Refractory Thymic Malignancies
NCT01904253	PHASE2	TERMINATED	A Phase 2 Study Comparing TAS-102 Versus Topotecan or Amrubicin to Treat Small Cell Lung Cancer Following Platinum-Based Chemotherapy
NCT00890955	PHASE1	COMPLETED	Amrubicin + Cyclophosphamide in Advanced Solid Organ Malignancies
NCT00915083	PHASE1	COMPLETED	A Phase I Study to Assess the Safety, Pharmacokinetics, and Potential Effects of Amrubicin on the QT/QTc Interval in Cancer Patients With Advanced Solid Tumors.

Clinical evidence (CIViC)

Variant × indication × effect (2 predictive associations from 2 curated evidence items):

Variant	Indication	Effect	Therapy	Level	CIViC
NQO1 P187S	Lung Cancer	Sensitivity/Response	Amrubicin	CIViC D	EID941
NQO1 EXPRESSION	Lung Cancer	Resistance	Amrubicin	CIViC D	EID940

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).