Cedazuridine

Summary

Cedazuridine (CHEMBL3237547) is an approved small molecule targeting CDA; indicated across 11 conditions including myelodysplastic syndrome and acute myeloid leukemia.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• Targets: 1 (CDA)
• Indications: 11 conditions
• Clinical trials: 39
• Chemistry: 268.21 Da · C9H14F2N2O5

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL3237547
Name	Cedazuridine
Type	Small molecule
Max phase	4
FDA approved	no
PubChem CID	25267009
Molecular formula	C9H14F2N2O5
Molecular weight	268.21
InChIKey	VUDZSIYXZUYWSC-DBRKOABJSA-N

SMILES: C1CN(C(=O)N[C@@H]1O)[C@H]2C([C@@H]([C@H](O2)CO)O)(F)F

IUPAC name: (4R)-1-[(2R,4R,5R)-3,3-difluoro-4-hydroxy-5-(hydroxymethyl)oxolan-2-yl]-4-hydroxy-1,3-diazinan-2-one

Also known as: Cedazuridina, Cedazuridine, CEDAZURIDINE COMPONENT OF ASTX-727, CEDAZURIDINE COMPONENT OF ASTX727, Cedazuridine component of inqovi, E-7727, E7727, CEDAZURIDINE

Patent coverage: 124 distinct patent families (310 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

Primary targets (GtoPdb curated mechanism): the Cancer dependency column is the DepMap CRISPR fitness signal (% of screened cell lines dependent on the target).

Gene	Target	Action	pAffinity	Cancer dependency	UniProt
CDA	cytidine deaminase	Inhibition	6.4	0.1%	P32320

Broader ChEMBL bioactivity targets: 1 (assay-derived). Sample: Cytidine deaminase.

Bioactivity

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

Target	pChembl	Type	Value	Unit	Activity ID
CDA	6.4	IC50	400	nM	CHEMBL_ACT_14565314

Target pathways

Aggregated over 1 target gene(s): CDA.

Top Reactome pathways

7 total, by targets touching each:

Pathway	Targets	Genes
Metabolism	1	CDA
Metabolism of nucleotides	1	CDA
Innate Immune System	1	CDA
Immune System	1	CDA
Neutrophil degranulation	1	CDA
Pyrimidine salvage	1	CDA
Nucleotide salvage	1	CDA

Dominant GO biological processes

GO term	Targets
CMP catabolic process	1
dCMP catabolic process	1
pyrimidine-containing compound salvage	1
cytosine metabolic process	1
UMP salvage	1
cellular response to external biotic stimulus	1
obsolete cytidine deamination	1
nucleobase-containing small molecule metabolic process	1
pyrimidine-containing compound metabolic process	1
carbohydrate derivative metabolic process	1

Indications & clinical

Indications

11 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
myelodysplastic syndrome	4	MONDO:0018881	EFO:0000198
acute myeloid leukemia	2	MONDO:0018874	EFO:0000222
neoplasm	1	MONDO:0005070	EFO:0000616
peripheral T-cell lymphoma, not otherwise specified	1	MONDO:0004964	EFO:0000211
prostate carcinoma	1	MONDO:0005159	EFO:0001663
triple-negative breast carcinoma	1	MONDO:0005494	EFO:0005537
chronic myelomonocytic leukemia	1	MONDO:0020311	EFO:1001779
head and neck squamous cell carcinoma	1	MONDO:0010150	EFO:0000181
leukemia	1	MONDO:0005059	EFO:0000565
myeloid leukemia	1	MONDO:0004643	MONDO:0004643

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

Clinical trials

Total trials: 39.

Phase distribution

Phase	Trials
PHASE2	16
PHASE1	14
PHASE1/PHASE2	7
PHASE2/PHASE3	1
PHASE3	1

Top trials by phase / activity

NCT	Phase	Status	Title
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)
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
NCT04340843	PHASE2	ACTIVE_NOT_RECRUITING	Testing the Combination of Belinostat and SGI-110 (Guadecitabine) or ASTX727 for the Treatment of Unresectable and Metastatic Conventional Chondrosarcoma
NCT04655755	PHASE1/PHASE2	ACTIVE_NOT_RECRUITING	Venetoclax in Combination With ASTX727 for the Treatment of Treatment-Naive High-Risk Myelodysplastic Syndrome or Chronic Myelomonocytic Leukemia
NCT04657081	PHASE1/PHASE2	ACTIVE_NOT_RECRUITING	Pharmacokinetics, Safety, and Efficacy of ASTX727 in Combination With Venetoclax in Acute Myeloid Leukemia (AML)
NCT04746235	PHASE2	ACTIVE_NOT_RECRUITING	Venetoclax and ASTX727 for the Treatment of Relapsed, Refractory, or Newly Diagnosed Acute Myeloid Leukemia
NCT04774393	PHASE1/PHASE2	RECRUITING	Decitabine/Cedazuridine and Venetoclax in Combination With Ivosidenib or Enasidenib for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia
NCT04817241	PHASE1/PHASE2	ACTIVE_NOT_RECRUITING	Testing Oral Decitabine and Cedazuridine (ASTX727) in Combination With Venetoclax for Higher-Risk Acute Myeloid Leukemia Patients
NCT04975919	PHASE2	ACTIVE_NOT_RECRUITING	Venetoclax in Combination With Decitabine and Cedazuridine for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia
NCT05007873	PHASE2	RECRUITING	ASTX727 and Dasatinib for the Treatment of Newly Diagnosed Philadelphia Chromosome or BCR-ABL Positive Chronic Myeloid Leukemia in Chronic Phase
NCT05010122	PHASE1/PHASE2	RECRUITING	ASTX727, Venetoclax, and Gilteritinib for the Treatment of Newly Diagnosed, Relapsed or Refractory FLT3-Mutated Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome
NCT05564390	PHASE2	RECRUITING	MYELOMATCH: A Screening Study to Assign People With Myeloid Cancer to a Treatment Study or Standard of Care Treatment Within myeloMATCH (MyeloMATCH Screening Trial)
NCT05600894	PHASE2	ACTIVE_NOT_RECRUITING	Venetoclax in Combination With ASTX727 for the Treatment of Chronic Myelomonocytic Leukemia and Other Myelodysplastic Syndrome/Myeloproliferative Neoplasm
NCT06091267	PHASE1/PHASE2	ACTIVE_NOT_RECRUITING	PK/Efficacy Bridging Study of ASTX727 in Chinese Subjects With Myelodysplastic Syndromes
NCT06445959	PHASE1/PHASE2	RECRUITING	Phase 1b/2 Study of Decitabine and Venetoclax in Combination With the Targeted Mutant IDH1 Inhibitor Olutasidenib
NCT06577441	PHASE2	RECRUITING	Testing the Addition of an IDH2 Inhibitor, Enasidenib, to Usual Treatment (Cedazuridine-Decitabine) for Higher-Risk Myelodysplastic Syndrome (MDS) With IDH2 Mutation (A MyeloMATCH Treatment Trial)
NCT06672146	PHASE2	RECRUITING	Comparing New Treatments for People With Newly Diagnosed Acute Myeloid Leukemia That Has an IDH2 Gene Change (A MyeloMATCH Treatment Trial)
NCT07148947	PHASE2	RECRUITING	Pacritinib With Standard of Care Azacitidine or Decitabine as a Bridge to Allogeneic Hematopoietic Stem Cell Transplant for Patients With Accelerated and Blast Phase Myeloproliferative Neoplasms
NCT07153497	PHASE2	NOT_YET_RECRUITING	Testing the Use of an IDH1 Inhibitor, Olutasidenib, in Acute Myeloid Leukemia Added to ASTX727 and Venetoclax; in High-Risk MDS Added to ASTX727; and Alone in Low Risk MDS (A MyeloMATCH Treatment Substudy)
NCT07437950	PHASE2	NOT_YET_RECRUITING	Comparing Different Treatment Lengths for Venetoclax in Older People With Newly Diagnosed Acute Myeloid Leukemia (A MyeloMATCH Treatment Trial)
NCT04985656	PHASE2	WITHDRAWN	A Study of Pevonedistat Combined With Decitabine and Cedazuridine in Adults With Higher-risk Myelodysplastic Syndromes
NCT05799079	PHASE2	TERMINATED	Decitabine and Cedazuridine in Combination With Venetoclax for the Treatment of Patients Who Have Relapsed Acute Myeloid Leukemia After Donor Stem Cell Transplant
NCT05835011	PHASE2	TERMINATED	A Study of Oral Decitabine/Cedazuridine in Combination With Magrolimab in Participants With Intermediate- to Very High-Risk Myelodysplastic Syndromes (MDS)
NCT05960773	PHASE2	SUSPENDED	Decitabine/Cedazuridine (INQOVI), an Oral DNA Demethylating Agent, in Subjects With BAP1 Cancer Predisposition Syndrome and Subclinical, Early-Stage Mesothelioma
NCT06661915	PHASE2	SUSPENDED	A Randomized Study of ASTX727 With or Without Iadademstat in Advanced Myeloproliferative Neoplasms (MPNs)
NCT03613532	PHASE1	ACTIVE_NOT_RECRUITING	Venetoclax Added to Fludarabine + Busulfan Prior to Transplant and to Maintenance Therapy for AML, MDS, and MDS/MPN
NCT03875287	PHASE1	ACTIVE_NOT_RECRUITING	Dose-Escalation Study of E7727, an Oral Cytidine Deaminase Inhibitor With Oral Decitabine in Subjects With Solid Tumors
NCT05010772	PHASE1	RECRUITING	Decitabine Alone or in Combination With Venetoclax, Gilteritinib, Enasidenib, or Ivosidenib as Maintenance Therapy for the Treatment of Acute Myeloid Leukemia in Remission
NCT05272384	PHASE1	RECRUITING	Testing the Combination of Nivolumab and ASTX727 for Relapsed or Refractory B-Cell Lymphoma
NCT05396859	PHASE1	ACTIVE_NOT_RECRUITING	Entrectinib in Combination With ASTX727 for the Treatment of Relapsed/Refractory TP53 Mutated Acute Myeloid Leukemia
NCT05403450	PHASE1	ACTIVE_NOT_RECRUITING	A Study of Tolinapant in Combination With Oral Decitabine/Cedazuridine and Oral Decitabine/Cedazuridine Alone in Participants With Relapsed/Refractory Peripheral T-cell Lymphoma (R/R PTCL)
NCT05673200	PHASE1	RECRUITING	Testing the Addition of an Anti-cancer Drug, ASTX727 (Cedazuridine, Decitabine), to Chemotherapy (Paclitaxel) and Immunotherapy (Pembrolizumab) for Metastatic Triple-Negative Breast Cancer
NCT06327685	PHASE1	RECRUITING	Avapritinib With Decitabine in Patients With SM-AHN
NCT06484062	PHASE1	RECRUITING	Testing the Anti-cancer Drug, Cirtuvivint, and Its Combination With ASTX727 to Improve Outcomes in Patients With Acute Myeloid Leukemia and Myelodysplastic Syndromes
NCT07012044	PHASE1	RECRUITING	A Study to Find the Highest Dose of Cedazuridine and Decitabine Combination With Filgrastim as a Treatment Option After Hematopoietic Stem Cell Transplant in Children With High-Risk Acute Myeloid Leukemia
NCT04953923	PHASE1	COMPLETED	Thorough QT Assessment of Cedazuridine in Healthy Subjects
NCT05037500	PHASE1	TERMINATED	Decitabine/Cedazuridine and Enzalutamide for the Treatment of Metastatic Castrate Resistant Prostate Cancer
NCT05456269	PHASE1	WITHDRAWN	A Study of Bisantrene Combined With Cytarabine or With Decitabine for Adult Subjects With Extramedullary AML and MDS
NCT06802523	PHASE1	SUSPENDED	Testing the Combination of Targeted Radiotherapy With Anti-Cancer Drugs, Venetoclax and ASTX-727, to Improve Outcomes for Adults With Newly Diagnosed Acute Myeloid Leukemia

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

Molecules sharing ≥1 of this drug’s curated primary targets, merged from two biobtree sources and ranked by shared-target count, then clinical phase: ChEMBL clinical-stage candidates (development phase ≥2) and PubChem drug-class bioactivity (approved / known drugs acting on the target). Deduplicated by drug name; the drug’s own salt forms are excluded. Note: for a drug with few primary targets a shared-target match can reflect off-target / promiscuous binding rather than the same therapeutic mechanism — the phase ordering surfaces bona-fide therapeutics first.

4 molecules share ≥1 primary target. Top 4 by shared-target count:

Molecule	Source	Status	Shared targets
TETRAHYDROURIDINE	ChEMBL	Phase 2	CDA
Cytarabine	PubChem	Approved	CDA
Gemcitabine	PubChem	Approved	CDA
Uridine	PubChem	Approved	CDA