Idarubicin

Summary

Idarubicin (CHEMBL1117) is an approved small molecule (ATC L01DB06); indicated across 28 conditions including neoplasm and myelodysplastic syndrome; with CIViC clinical evidence for 1 variant-indication association (e.g. DNMT3A R882 in acute myeloid leukemia).

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: L01DB06
• Indications: 28 conditions
• Clinical trials: 203
• Precision-oncology evidence (CIViC): 1 variant–indication association
• Chemistry: 497.5 Da · C26H27NO9

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL1117
Name	Idarubicin
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	42890
ChEBI	CHEBI:42068
ATC	L01DB06
Molecular formula	C26H27NO9
Molecular weight	497.5
InChIKey	XDXDZDZNSLXDNA-TZNDIEGXSA-N

SMILES: C[C@H]1[C@H]([C@H](C[C@@H](O1)O[C@H]2C[C@@](CC3=C2C(=C4C(=C3O)C(=O)C5=CC=CC=C5C4=O)O)(C(=O)C)O)N)O

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

Also known as: Idarubicin, Idarubicina, Idarubicine, idarubicin, SID50106402, SID50106404, SID90340569, SID50106403, IDARUBICINE, IDARUBICIN, SID124891657, ML069

Parent form; salt/anhydrous children: CHEMBL1200976

Patent coverage: 32,512 distinct patent families (136,065 SureChEMBL compound mentions), from 3 matched compound structure(s). One matched structure accounts for 135,887 (100%) of the total. 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: 47 (assay-derived). Sample: Microtubule-associated protein tau, Lysine-specific demethylase 4E, Ubiquitin carboxyl-terminal hydrolase 2, Nuclear receptor ROR-gamma, Survival motor neuron protein, Prelamin-A/C, ATP-dependent DNA helicase Q1, RecQ-like DNA helicase BLM, Inositol monophosphatase 1, Ferritin light chain.

Bioactivity

ChEMBL activities: 25 potent at pChembl ≥ 5 of 64 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
TOP2A	8.59	IC50	2.6	nM	CHEMBL_ACT_25724247
NR3C1	6.96	AC50	110	nM	CHEMBL_ACT_25176089
LMNA	6.6	Potency	251.2	nM	CHEMBL_ACT_4403321
LMNA	6.5	Potency	316.2	nM	CHEMBL_ACT_3639530
MTOR	6.38	Potency	414	nM	CHEMBL_ACT_3919392
PMP22	6.37	Potency	425.6	nM	CHEMBL_ACT_4358519
HTT	6.2	Potency	631	nM	CHEMBL_ACT_3760513
SMN1	6.2	Potency	631	nM	CHEMBL_ACT_3878105
MEN1	5.75	Potency	1778	nM	CHEMBL_ACT_3630890
P97697	5.7	Potency	1995	nM	CHEMBL_ACT_4407029
GMNN	5.7	Potency	1995	nM	CHEMBL_ACT_5065072
MTOR	5.63	Potency	2328	nM	CHEMBL_ACT_4393074
MAPT	5.45	Potency	3548	nM	CHEMBL_ACT_4050864
MEN1	5.35	Potency	4467	nM	CHEMBL_ACT_3634701
PGR	5.24	AC50	5800	nM	CHEMBL_ACT_25223241
SOS1	5.22	IC50	6000	nM	CHEMBL_ACT_25697635
BLM	5.19	Potency	6462	nM	CHEMBL_ACT_4280272
SLC6A4	5.14	AC50	7300	nM	CHEMBL_ACT_25150595
GHSR	5.11	AC50	7700	nM	CHEMBL_ACT_25173012
SOS1	5.1	IC50	8000	nM	CHEMBL_ACT_25874369
MAPT	5.1	Potency	7943	nM	CHEMBL_ACT_3966706
SLC6A2	5.06	AC50	8700	nM	CHEMBL_ACT_25145263
HTR2C	5.03	AC50	9300	nM	CHEMBL_ACT_25132064
ADRA2C	5	AC50	10000	nM	CHEMBL_ACT_25148149
HRH1	5	AC50	10000	nM	CHEMBL_ACT_25212782

Target pathways

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

Indications & clinical

Indications

28 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
neoplasm	4	MONDO:0005070	EFO:0000616
myelodysplastic syndrome	3	MONDO:0018881	EFO:0000198
acute lymphoblastic leukemia	3	MONDO:0004967	EFO:0000220
acute monocytic leukemia	3	MONDO:0007896	EFO:0000221
acute myeloid leukemia	3	MONDO:0018874	EFO:0000222
acute promyelocytic leukemia	3	MONDO:0012883	EFO:0000224
leukemia	3	MONDO:0005059	EFO:0000565
lymphoma	3	MONDO:0005062	EFO:0000574
plasma cell myeloma	3	MONDO:0009693	EFO:0001378
myelodysplastic syndrome with single lineage dysplasia	3	MONDO:0005272	EFO:0003802
non-Hodgkin lymphoma	3	MONDO:0018908	EFO:0005952
acute erythroid leukemia	3	MONDO:0017858	EFO:1001257
soft tissue sarcoma	3	MONDO:0018078	EFO:1001968
acute myelomonocytic leukemia M4	3	MONDO:0018871	EFO:0000223
myeloproliferative neoplasm	3	MONDO:0020076	EFO:0002428
acute megakaryoblastic leukemia	3	MONDO:0018872	EFO:0003025
acute myeloblastic leukemia without maturation	3	MONDO:0005224	EFO:0003027
myelodysplastic syndrome with excess blasts	3	MONDO:0019454	EFO:0003811
chronic myelomonocytic leukemia	3	MONDO:0020311	EFO:1001779
plasma cell neoplasm	3	MONDO:0004959	EFO:0000200
hepatocellular carcinoma	2	MONDO:0007256	EFO:0000182
chronic myeloid leukemia	2	MONDO:0011996	EFO:0000339
neutropenia	2	MONDO:0001475	MONDO:0001475
acute biphenotypic leukemia	2	MONDO:0020322	MONDO:0019460

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

Clinical trials

Total trials: 203.

Phase distribution

Phase	Trials
PHASE2	71
PHASE3	45
PHASE1/PHASE2	29
PHASE1	29
PHASE4	12
PHASE2/PHASE3	8
Not specified	8
EARLY_PHASE1	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT07561892	PHASE4	RECRUITING	Study of the Effectiveness and Safety of Daunorubicin /Idarubicin ± Silibinin in Treating Newly Diagnosed AML (Non-M3).
NCT00180128	PHASE4	UNKNOWN	AIDA2000 - Risk-Adapted Therapy for Patients With Acute Promyelocytic Leukemia
NCT00198978	PHASE4	COMPLETED	German Multicenter Trial for Treatment of Elderly Patients With Newly Diagnosed Acute Lymphoblastic Leukemia
NCT00198991	PHASE4	COMPLETED	German Multicenter Trial for Treatment of Newly Diagnosed Acute Lymphoblastic Leukemia in Adults (07/2003)
NCT00199056	PHASE4	COMPLETED	German Multicenter Trial for Treatment of Newly Diagnosed Acute Lymphoblastic Leukemia in Adults (06/99)
NCT00199095	PHASE4	COMPLETED	Treatment of Elderly Patients (>65 Years) With Acute Lymphoblastic Leukemia
NCT00199147	PHASE4	UNKNOWN	Efficacy of G-CSF-Priming in Elderly AML Patients
NCT00408278	PHASE4	COMPLETED	Treatment of Newly Diagnosed Patients With Acute Promyelocytic Leukemia (PETHEMA LPA 2005)
NCT00464217	PHASE4	COMPLETED	Treatment of the Acute Myeloblastic Leukaemia in Patients Over 65 Years
NCT00487448	PHASE4	COMPLETED	SMD_FLAG-IDA_98: FLAG-IDA in Induction Treatment of High Risk Myelodysplastic Syndromes or Secondary Acute Myeloblastic Leukemia
NCT01366898	PHASE4	UNKNOWN	Protocol For the Treatment Acute Lymphoblastic Leukemia With Ph ‘Negative in Elderly Patients (> 55 Years)
NCT02020161	PHASE4	UNKNOWN	Clinical Guidelines for APL Treatment
NCT02339740	PHASE3	ACTIVE_NOT_RECRUITING	Tretinoin and Arsenic Trioxide in Treating Patients With Untreated Acute Promyelocytic Leukemia
NCT02416388	PHASE2/PHASE3	RECRUITING	Study to Improve OS in 18 to 60 Year-old Patients, Comparing Daunorubicin Versus High Dose Idarubicin Induction Regimens, High Dose Versus Intermediate Dose Cytarabine Consolidation Regimens, and Standard Versus MMF Prophylaxis of GvHD in Allografted Patients in First CR
NCT03117751	PHASE2/PHASE3	ACTIVE_NOT_RECRUITING	Total Therapy XVII for Newly Diagnosed Patients With Acute Lymphoblastic Leukemia and Lymphoma
NCT05177731	PHASE3	ACTIVE_NOT_RECRUITING	Venetoclax + Decitabine vs. 7+3 Induction Chemotherapy in Young AML
NCT06744504	PHASE3	RECRUITING	Standard-dose vs Intermediate-dose Cytarabine Induction in the Treatment of Acute Myeloid Leukemia With RUNX1-RUNX1T1
NCT07132684	PHASE3	RECRUITING	Comparison of VA and D/IA Induction Regimens in Elderly Fit Acute Myeloid Leukemia Patients
NCT07232134	PHASE3	RECRUITING	The Efficacy of Therapy in Patients With Acute Myeloid Leukemia and Down Syndrome in Russia
NCT07486479	PHASE3	NOT_YET_RECRUITING	Venetoclax, Azacitidine, and Mitoxantrone Hydrochloride Liposome Versus Idarubicin and Cytarabine in Newly Diagnosed AML
NCT00002517	PHASE3	COMPLETED	Combination Chemotherapy in Treating Children With Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome
NCT00002549	PHASE3	UNKNOWN	Combination Chemotherapy Followed by Bone Marrow or Peripheral Stem Cell Transplantation in Treating Patients With Acute Myelogenous Leukemia
NCT00002565	PHASE3	COMPLETED	Combination Chemotherapy in Treating Patients With Intermediate-Grade or Immunoblastic Non-Hodgkin’s Lymphoma
NCT00002658	PHASE3	UNKNOWN	Combination Chemotherapy, Biological Therapy, and Bone Marrow Transplantation in Treating Patients With Acute Myeloid Leukemia
NCT00002701	PHASE3	UNKNOWN	Combination Chemotherapy With or Without Bone Marrow Transplantation in Treating Patients With Acute Promyelocytic Leukemia
NCT00002719	PHASE3	COMPLETED	Combination Chemotherapy With or Without G-CSF in Treating Older Patients With Acute Myeloid Leukemia
NCT00002771	PHASE3	UNKNOWN	Chemotherapy, Interferon, and Bone Marrow Transplantation in Treating Patients With Chronic Myelogenous Leukemia
NCT00002798	PHASE3	COMPLETED	Combination Chemotherapy With or Without Bone Marrow Transplantation in Treating Children With Acute Myelogenous Leukemia or Myelodysplastic Syndrome
NCT00002812	PHASE3	COMPLETED	Combination Chemotherapy in Treating Children With Acute Lymphocytic Leukemia
NCT00002816	PHASE3	COMPLETED	Combination Chemotherapy in Treating Children With Relapsed Acute Lymphoblastic Leukemia
NCT00002835	PHASE3	COMPLETED	Combination Chemotherapy in Treating Patients With Lymphoma
NCT00002868	PHASE3	COMPLETED	Interferon-alfa With or Without Chemotherapy and Peripheral Stem Cell Transplantation in Treating Patients With Newly Diagnosed Chronic Myelogenous Leukemia
NCT00002926	PHASE3	UNKNOWN	Combination Chemotherapy With or Without Peripheral Stem Cell Transplantation in Treating Patients With Myelodysplastic Syndrome or Acute Myelogenous Leukemia
NCT00002945	PHASE3	COMPLETED	High Dose Chemotherapy, Peripheral Stem Cell Transplantation, and Interleukin-2 in Treating Patients With Acute Myeloid Leukemia
NCT00002989	PHASE3	UNKNOWN	Combination Chemotherapy With or Without Idarubicin and Peripheral Stem Cell Transplantation in Treating Patients With Leukemia or Myelodysplastic Syndrome
NCT00003602	PHASE3	UNKNOWN	Combination Chemotherapy in Treating Older Patients With Acute Myeloid Leukemia
NCT00003603	PHASE3	UNKNOWN	Chemotherapy Plus Steroid Therapy in Treating Patients With Multiple Myeloma
NCT00003639	PHASE3	UNKNOWN	Combination Chemotherapy With or Without Interferon Alfa in Treating Patients With Low-Grade Non-Hodgkin’s Lymphoma
NCT00005823	PHASE3	COMPLETED	Intensive Compared With Nonintensive Chemotherapy in Treating Older Patients With Acute Myeloid Leukemia or Myelodysplastic Syndrome
NCT00006232	PHASE3	COMPLETED	Combination Chemotherapy in Treating Patients With Stage II or Stage III Multiple Myeloma

Clinical evidence (CIViC)

Variant × indication × effect (1 predictive associations from 1 curated evidence items):

Variant	Indication	Effect	Therapy	Level	CIViC
DNMT3A R882	Acute Myeloid Leukemia	Sensitivity/Response	Idarubicin	CIViC B	EID18

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline, but PharmGKB curates 15 clinical and 28 variant annotation(s) for this drug (gene-keyed; see PharmGKB).

Related molecules

Related molecules

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