Azacitidine

Summary

Azacitidine (CHEMBL1489) is an approved small-molecule antineoplastic agent (ATC L01BC07); indicated across 68 conditions including acute myeloid leukemia and myelodysplastic syndrome; with CIViC clinical evidence for 3 variant-indication associations (e.g. IDH1 Mutation in acute myeloid leukemia).

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: L01BC07
• Indications: 68 conditions
• Clinical trials: 644
• Precision-oncology evidence (CIViC): 3 variant–indication associations
• Chemistry: 244.2 Da · C8H12N4O5

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL1489
Name	Azacitidine
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	9444
ChEBI	CHEBI:2038
ATC	L01BC07
Molecular formula	C8H12N4O5
Molecular weight	244.2
InChIKey	NMUSYJAQQFHJEW-KVTDHHQDSA-N

SMILES: C1=NC(=NC(=O)N1[C@H]2[C@@H]([C@@H]([C@H](O2)CO)O)O)N

IUPAC name: 4-amino-1-[(2R,3R,4S,5R)-3,4-dihydroxy-5-(hydroxymethyl)oxolan-2-yl]-1,3,5-triazin-2-one

ChEBI definition: An N-glycosyl-1,3,5-triazine that is 4-amino-1,3,5-triazin-2(1H)-one substituted by a β-D-ribofuranosyl residue via an N-glycosidic linkage. An antineoplastic agent, it is used in the treatment of myeloid leukaemia.

Pharmacological roles (ChEBI): antineoplastic agent.

Also known as: Azacitidina, Azacitidine, Azacitidine accord, Azacitidine betapharm, Azacitidine celgene, Azacitidine kabi, Azacitidine mylan, Ladakamycin, NSC-102816, Onureg, U-18,496, U-18496

Parent form; salt/anhydrous children: CHEMBL3250420, CHEMBL3250421

Patent coverage: 24,527 distinct patent families (97,123 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: 15 (assay-derived). Sample: Prelamin-A/C, RecQ-like DNA helicase BLM, Thrombopoietin, Geminin, Ras-related protein Rab-9A, Peripheral myelin protein 22, DNA (cytosine-5)-methyltransferase 1, Beta-lactamase, Muscarinic acetylcholine receptor M1, Serine/threonine-protein kinase mTOR.

Bioactivity

ChEMBL activities: 17 potent at pChembl ≥ 5 of 23 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
BLM	8.8	Potency	1.6	nM	CHEMBL_ACT_4745984
BLM	8.8	Potency	1.6	nM	CHEMBL_ACT_4925881
MTOR	6.93	Potency	116.7	nM	CHEMBL_ACT_4787550
DNMT1	6.52	IC50	300	nM	CHEMBL_ACT_18337672
MTOR	6.13	Potency	736.2	nM	CHEMBL_ACT_3919375
P08482	5.95	Potency	1122	nM	CHEMBL_ACT_4803347
HBB	5.9	Potency	1259	nM	CHEMBL_ACT_3770475
LMNA	5.8	Potency	1585	nM	CHEMBL_ACT_3656584
NFKB1	5.65	Potency	2239	nM	CHEMBL_ACT_3675751
NFKB1	5.65	Potency	2239	nM	CHEMBL_ACT_4588933
LMNA	5.5	Potency	3162	nM	CHEMBL_ACT_4403281
THPO	5.5	Potency	3162	nM	CHEMBL_ACT_4805544
TP53	5.5	Potency	3162	nM	CHEMBL_ACT_4858606
GMNN	5.5	Potency	3162	nM	CHEMBL_ACT_5065020
THPO	5.5	Potency	3162	nM	CHEMBL_ACT_5070295
LMNA	5.25	Potency	5623	nM	CHEMBL_ACT_3646866
P00811	5	Potency	10000	nM	CHEMBL_ACT_4668007

Target pathways

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

Indications & clinical

Indications

68 indications (7 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
acute myeloid leukemia	4	MONDO:0018874	EFO:0000222
myelodysplastic syndrome	4	MONDO:0018881	EFO:0000198
myelodysplastic syndrome with excess blasts	4	MONDO:0019454	EFO:0003811
neoplasm	4	MONDO:0005070	EFO:0000616
chronic myelomonocytic leukemia	4	MONDO:0020311	EFO:1001779
leukemia	3	MONDO:0005059	EFO:0000565
thrombocytopenia	3	MONDO:0002049	HP:0001873
diffuse large B-cell lymphoma	3	MONDO:0018905	EFO:0000403
lymphoma	3	MONDO:0005062	EFO:0000574
angioimmunoblastic T-cell lymphoma	3	MONDO:0004977	EFO:0000255
peripheral T-cell lymphoma, not otherwise specified	3	MONDO:0004964	EFO:0000211
mature T-cell and NK-cell non-Hodgkin lymphoma	3	MONDO:0000430	MONDO:0000430
myeloid leukemia	3	MONDO:0004643	MONDO:0004643
non-small cell lung carcinoma	2	MONDO:0005233	EFO:0003060
exocrine pancreatic carcinoma	2	MONDO:0005192	EFO:0002618
B-cell chronic lymphocytic leukemia	2	MONDO:0004948	EFO:0000095
Hodgkins lymphoma	2	MONDO:0004952	EFO:0000183
cutaneous melanoma	2	MONDO:0005012	EFO:0000389
plasma cell myeloma	2	MONDO:0009693	EFO:0001378
metastatic melanoma	2	MONDO:0005191	EFO:0002617
male breast carcinoma	2	MONDO:0005628	EFO:0006861
acute erythroid leukemia	2	MONDO:0017858	EFO:1001257
beta thalassemia	2	MONDO:0019402	Orphanet:848
acute lymphoblastic leukemia	2	MONDO:0004967	EFO:0000220
prostate adenocarcinoma	2	MONDO:0005082	EFO:0000673
rectal cancer	2	MONDO:0006519	EFO:1000657
breast neoplasm	2	MONDO:0021100	MONDO:0007254
ovarian cancer	2	MONDO:0008170	MONDO:0008170
follicular lymphoma	2	MONDO:0018906	MONDO:0018906
colonic neoplasm	2	MONDO:0005401	MONDO:0021063
hematopoietic and lymphoid system neoplasm	2	MONDO:0002334	MONDO:0044881
primary myelofibrosis	2	MONDO:0009692	MONDO:0044903
lymphoid neoplasm	2	MONDO:0005157	EFO:0001642
nasopharyngeal neoplasm	2	MONDO:0005375	EFO:0004252
lymphoid leukemia	2	MONDO:0005402	EFO:0004289
acute monocytic leukemia	2	MONDO:0007896	EFO:0000221
paraganglioma	2	MONDO:0000448	EFO:1000453
chronic myeloid leukemia	1	MONDO:0011996	EFO:0000339
liposarcoma	1	MONDO:0005060	EFO:0000569
renal cell carcinoma	1	MONDO:0005086	EFO:0000681
squamous cell carcinoma	1	MONDO:0005096	EFO:0000707
non-Hodgkin lymphoma	1	MONDO:0018908	EFO:0005952
head and neck cancer	1	MONDO:0005627	EFO:0006859
ependymoma	1	MONDO:0016698	EFO:1000028
central nervous system cancer	1	MONDO:0002714	EFO:0000326
osteosarcoma	1	MONDO:0009807	EFO:0000637
myelodysplastic/myeloproliferative disease	1	MONDO:0020077	MONDO:0020077
head and neck squamous cell carcinoma	1	MONDO:0010150	EFO:0000181
acute kidney injury	1	MONDO:0002492	MONDO:0002492
graft versus host disease	1	MONDO:0013730	MONDO:0013730
tuberculosis	1	MONDO:0018076	MONDO:0018076
sarcoma	1	MONDO:0005089	EFO:0000691
CD4+/CD56+ hematodermic neoplasm	1	MONDO:0019467	EFO:0010580
pancreatic ductal adenocarcinoma	1	MONDO:0005184	MONDO:0005184
colorectal neoplasm	1	MONDO:0005335	MONDO:0005575
acute biphenotypic leukemia	1	MONDO:0020322	MONDO:0019460
brain neoplasm	0	MONDO:0021211	EFO:0003833

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

Clinical trials

Total trials: 644.

Phase distribution

Phase	Trials
PHASE2	236
PHASE1	161
PHASE1/PHASE2	126
PHASE3	63
Not specified	31
PHASE2/PHASE3	12
PHASE4	8
EARLY_PHASE1	7

Top trials by phase / activity

NCT	Phase	Status	Title
NCT05144243	PHASE4	ACTIVE_NOT_RECRUITING	Study to Assess Adverse Events and Change in Disease State of Oral Venetoclax in Combination With Subcutaneous (SC) Azacitidine in Newly Diagnosed Adult Participants With Acute Myeloid Leukemia (AML) Who Are Ineligible for Intensive Chemotherapy in China
NCT06370000	PHASE4	RECRUITING	Oral Azacitidine in Transplant-Eligible Patients With Acute Myeloid Leukemia (AML) Suffering From Health-Inequality
NCT07044687	PHASE4	RECRUITING	Study to Assess Adverse Events and Change in Disease Activity of Oral Venetoclax in Combination With Subcutaneous (SC) or Intravenous (IV) Azacitidine in Newly Diagnosed Adult Participants With Acute Myeloid Leukemia (AML) Who Are Ineligible for Standard Induction Therapy in India
NCT07046182	PHASE4	ACTIVE_NOT_RECRUITING	Clinical Study of CEP+Dasatinib + Azacytidine in First-line Treatment of. Angioimmunoblastoma Foresight
NCT01011283	PHASE4	TERMINATED	To Demonstrate Superiority of Decitabine Over Azacitidine in Subjects With Intermediate- or High-risk MDS.
NCT01201811	PHASE4	COMPLETED	Study of Azacitidine in Adult Taiwanese Subjects With Higher-Risk Myelodysplastic Syndromes (MDS)
NCT03873311	PHASE4	UNKNOWN	Azacytidine + HAG Regimen vs. Azacytidine for Elderly Patients With Newly Diagnosed Myeloid Malignancy
NCT06004765	PHASE4	UNKNOWN	Efficacy and Safety of Lenalidomide Combined With Azacitidine vs Azacitidine in the Treatment of MDS-RS
NCT03173248	PHASE3	ACTIVE_NOT_RECRUITING	Study of AG-120 (Ivosidenib) vs. Placebo in Combination With Azacitidine in Participants With Previously Untreated Acute Myeloid Leukemia With an IDH1 Mutation
NCT03703375	PHASE3	ACTIVE_NOT_RECRUITING	Efficacy and Safety of Oral Azacitidine (CC-486) Compared to Investigator’s Choice Therapy in Patients With Relapsed or Refractory Angioimmunoblastic T Cell Lymphoma
NCT04090736	PHASE3	ACTIVE_NOT_RECRUITING	Study to Compare Azacitidine Plus Pevonedistat Versus Azacitidine in Patients With Acute Myeloid Leukemia Not Eligible for Standard Chemotherapy
NCT04173533	PHASE3	ACTIVE_NOT_RECRUITING	Randomised Study of Oral Azacitidine vs Placebo Maintenance in AML or MDS Patients After Allo-SCT
NCT04184505	PHASE3	RECRUITING	Feasibility of Allogeneic Stem Cell Transplantation in Higher-risk-MDS (ACROBAT)
NCT04229979	PHASE3	ACTIVE_NOT_RECRUITING	Galinpepimut-S Versus Investigator’s Choice of Best Available Therapy for Maintenance in AML CR2/CRp2
NCT04256317	PHASE2/PHASE3	RECRUITING	A Multi-phase Study of ASTX030 (Azacitidine and Cedazuridine) in Myeloid Neoplasm Alone or in Combination With Venetoclax in AML (AZTOUND Study)
NCT04401748	PHASE3	ACTIVE_NOT_RECRUITING	Study Of Venetoclax Tablet With Intravenous or Subcutaneous Azacitidine to Assess Change in Disease Activity In Adult Participants With Newly Diagnosed Higher-Risk Myelodysplastic Syndrome
NCT04799275	PHASE2/PHASE3	ACTIVE_NOT_RECRUITING	Testing CC-486 (Oral Azacitidine) Plus the Standard Drug Therapy in Patients 75 Years or Older With Newly Diagnosed Diffuse Large B Cell Lymphoma
NCT05183035	PHASE3	RECRUITING	Venetoclax in Children With Relapsed Acute Myeloid Leukemia (AML)
NCT05404906	PHASE2/PHASE3	RECRUITING	AZA + Venetoclax as Maintenance Therapy in Younger Adults With AML in First Remission
NCT05469737	PHASE2/PHASE3	ACTIVE_NOT_RECRUITING	A Study to Compare the Efficacy and Safety of Oral Azacitidine Plus Best Supportive Care (BSC) Versus Placebo Plus BSC in Participants With International Prognostic Scoring System Revised (IPSS-R) Low- or Intermediate-risk Myelodysplastic Syndrome (MDS)
NCT05586074	PHASE3	RECRUITING	HEC73543 Versus Salvage Chemotherapy in R/R FLT3-ITD AML
NCT05678933	PHASE3	ENROLLING_BY_INVITATION	AC-CHOP Versus CHOP in Patients With Previously Untreated PTCL-TFH
NCT05883956	PHASE3	ACTIVE_NOT_RECRUITING	A Study Comparing Treatment Preference Between Oral Decitabine/Cedazuridine and Azacitidine in Myelodysplastic Syndrome, Low-Blast Acute Myeloid Leukemia, or Chronic Myelomonocytic Leukemia
NCT05907057	PHASE3	RECRUITING	An Open-label Phase 3b Study of Ivosidenib in Combination With Azacitidine in Adult Patients Newly Diagnosed With IDH1m Acute Myeloid Leukemia (AML) Ineligible for Intensive Induction Chemotherapy.
NCT06345365	PHASE3	RECRUITING	MA+AZA Regimen for the Treatment of Newly Diagnosed Acute Myeloid Leukemia (AML)
NCT06389292	PHASE3	RECRUITING	A Pivotal Study of APG-2575 (Lisaftoclax) Combined With Azacitidine in the Treatment of Acute Myeloid Leukemia
NCT06465953	PHASE3	RECRUITING	Ivosidenib (IVO) Monotherapy and Azacitidine (AZA) Monotherapy in Patients With Hypomethylating Agent (HMA) Naive Myelodysplastic Syndromes (MDS) With an IDH1 Mutation
NCT06641414	PHASE3	RECRUITING	Lisaftoclax (APG-2575) Combined With Azacytidine (AZA) in the Treatment of Patients With Higher-risk Myelodysplastic Syndrome (GLORA-4).
NCT06647862	PHASE3	RECRUITING	IMM01+Azacitidine VS Placebo +Azacitidine in Patients With Newly Diagnosed Chronic Myelomonocytic Leukemia (CMML1-2)
NCT06852222	PHASE3	RECRUITING	A Study of Bleximenib, Venetoclax and Azacitidine For Treatment of Participants With Newly Diagnosed Acute Myeloid Leukemia (AML)
NCT06927232	PHASE3	NOT_YET_RECRUITING	AZA+Lus VS AZA Monotherapy in HR-MDS
NCT07007312	PHASE3	RECRUITING	Studies to Assess Ziftomenib in Combination With Ven+Aza or 7+3 in Patients With Untreated NPM1-m or KMT2A-r AML
NCT07082452	PHASE3	NOT_YET_RECRUITING	A Multicenter Trial Evaluating Efficacy and Safety of A Reduced Venetoclax Exposure To Seven Days Versus Standard Continuous Venetoclax Exposure Combined With Azacitidine in Treatment Naïve Subjects With Acute Myeloid Leukemia Who Are Ineligible for Intensive Induction
NCT07132684	PHASE3	RECRUITING	Comparison of VA and D/IA Induction Regimens in Elderly Fit Acute Myeloid Leukemia Patients
NCT07255872	PHASE2/PHASE3	NOT_YET_RECRUITING	A Study of BL-M11D1 in Combination With Cytarabine + Daunorubicin or Venetoclax + Azacitidine in Patients With Acute Myeloid Leukemia
NCT07389616	PHASE2/PHASE3	RECRUITING	A Clinical Trial of Cidabenamine Plus Azacitidine to Prevent Post-Transplant Progression in High-Risk Peripheral T-Cell Lymphoma
NCT07407140	PHASE3	NOT_YET_RECRUITING	VAG Versus Standard Chemotherapy With FLT3 Inhibitor in Adult Patients With FLT3-Mutated AML
NCT07407660	PHASE3	NOT_YET_RECRUITING	Shortened Venetoclax Duration Based on Day 14 BM Blasts Versus Standard Therapy in Elderly or Frail Patients With AML Patients Treated With Azacitidine Plus Venetoclax
NCT07425808	PHASE2/PHASE3	NOT_YET_RECRUITING	FLT3-ITD Targeted Therapy in Fit AML Patients
NCT07469046	PHASE3	NOT_YET_RECRUITING	VAH vs VA in Newly Diagnosed Elderly AML

Clinical evidence (CIViC)

Variant × indication × effect (3 predictive associations from 3 curated evidence items):

Variant	Indication	Effect	Therapy	Level	CIViC
IDH1 Mutation	Acute Myeloid Leukemia	Sensitivity/Response	Ivosidenib + Azacitidine	CIViC A	EID10313
TP53 Mutation	Myelodysplastic Syndrome	Sensitivity/Response	Azacitidine + Eprenetapopt	CIViC B	EID12029
ZMIZ1::ABL1 Fusion	Chronic Myelomonocytic Leukemia	Sensitivity/Response	Azacitidine + Dasatinib	CIViC C	EID12636

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline, but PharmGKB curates 0 clinical and 6 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).