Omacetaxine Mepesuccinate

Summary

Omacetaxine Mepesuccinate (CHEMBL46286) is an approved small-molecule antineoplastic agent (ATC L01XX40); indicated across 6 conditions including neoplasm and leukemia; with CIViC clinical evidence for 1 variant-indication association (e.g. BCR::ABL1 Fusion AND ABL1 T315I in chronic myeloid leukemia).

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: L01XX40
• Indications: 6 conditions
• Clinical trials: 32
• Precision-oncology evidence (CIViC): 1 variant–indication association
• Chemistry: 545.6 Da · C29H39NO9

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL46286
Name	Omacetaxine Mepesuccinate
Type	Small molecule
Max phase	4
FDA approved	no
PubChem CID	285033
ChEBI	CHEBI:71019
ATC	L01XX40
Molecular formula	C29H39NO9
Molecular weight	545.6
InChIKey	HYFHYPWGAURHIV-JFIAXGOJSA-N

SMILES: CC(C)(CCC[C@@](CC(=O)OC)(C(=O)O[C@H]1[C@H]2C3=CC4=C(C=C3CCN5[C@@]2(CCC5)C=C1OC)OCO4)O)O

IUPAC name: 1-O-[(2S,3S,6R)-4-methoxy-16,18-dioxa-10-azapentacyclo[11.7.0.02,6.06,10.015,19]icosa-1(20),4,13,15(19)-tetraen-3-yl] 4-O-methyl (2R)-2-hydroxy-2-(4-hydroxy-4-methylpentyl)butanedioate

ChEBI definition: A cephalotaxine-derived alkaloid ester obtained from Cephalotaxus harringtonia; used for the treatment of chronic or accelerated phase chronic myeloid leukaemia.

Pharmacological roles (ChEBI): antineoplastic agent, protein synthesis inhibitor, apoptosis inducer, anticoronaviral agent.

Also known as: Ceflatonin, CGX-635, Homoharringtonin, HOMOHARRINGTONINE, Mepesuccinate d’omacetaxine, Mepesuccinato de omacetaxina, Myelostat, NSC-141633, Omacetaxine mepesuccinate, Omapro, Synribo, Homoharringtonine

Patent coverage: 1,240 distinct patent families (2,776 SureChEMBL compound mentions), from 2 matched compound structure(s). One matched structure accounts for 2,773 (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

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

3 approved indications. FDA phase 4, plus an anticancer drug’s labelled cancer uses (which ChEMBL often logs at phase 3).

Indication	Phase	MONDO	EFO
neoplasm	4	MONDO:0005070	EFO:0000616
leukemia	3	MONDO:0005059	EFO:0000565
acute myeloid leukemia	3	MONDO:0018874	EFO:0000222

2 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
chronic myeloid leukemia	2	MONDO:0011996	EFO:0000339
myelodysplastic syndrome	1	MONDO:0018881	EFO:0000198

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

Clinical trials

Total trials: 32.

Phase distribution

Phase	Trials
PHASE2	17
PHASE1/PHASE2	5
PHASE3	4
PHASE2/PHASE3	2
Not specified	2
PHASE1	1
EARLY_PHASE1	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT05726110	PHASE3	RECRUITING	Selinexor in Combination With HAD or CAG Rregimens in Relapsed or Refractory Acute Myeloid Leukemia
NCT05805098	PHASE2/PHASE3	RECRUITING	Venetoclax Combined With Homoharringtonine and Cytarabine in Induction for AML
NCT06361329	PHASE3	RECRUITING	Comparing the Efficacy of VHAG and Traditional Chemotherapy Regimens in Newly Diagnosed ETP-ALL
NCT06855810	PHASE2/PHASE3	RECRUITING	Newly-diagnosed Pediatric T-cell ALL Protocol
NCT07469046	PHASE3	NOT_YET_RECRUITING	VAH vs VA in Newly Diagnosed Elderly AML
NCT00004933	PHASE3	TERMINATED	Homoharringtonine Compared With Hydroxyurea for Chronic Myelogenous Leukemia That Has Not Responded to Interferon Alfa
NCT06221683	PHASE2	RECRUITING	Clinical Study of Induction Therapy Options Based on Molecular Subtyping and MRD in Children and Adolescents With AML
NCT06483906	PHASE2	RECRUITING	Venetoclax and Azacitidine Combined With Homoharringtonine, Followed by Allo-HSCT for Intermediate and High-risk AML.
NCT06742463	PHASE2	RECRUITING	VHAG in Treating R/R T-ALL/LBL
NCT06744556	PHASE2	ACTIVE_NOT_RECRUITING	HAV Versus DAV/IAV Induction Regimen in Elderly Patients With AML
NCT07159906	PHASE1/PHASE2	NOT_YET_RECRUITING	Homoharringtonine, BCL-2 Inhibitor, Rituximab, and Prednisone in Relapsed/Refractory Diffuse Large B-Cell Lymphoma
NCT07163910	PHASE2	RECRUITING	Homoharringtonine Plus Androgen Deprivation Therapy in the Neoadjuvant Treatment of Prostate Cancer: A Single-Arm Clinical Study
NCT07505160	PHASE2	NOT_YET_RECRUITING	Efficacy and Safety of Lisafotoclax Plus Decitabine and Homoharringtonine in Venetoclax/Azacitidine Pretreated AML Patients
NCT00002574	PHASE2	COMPLETED	Homoharringtonine and Interferon Alfa in Treating Patients With Chronic Myelogenous Leukemia
NCT00003239	PHASE2	COMPLETED	Chemotherapy and Biological Therapy in Treating Patients With Chronic Phase Chronic Myelogenous Leukemia
NCT00003694	PHASE2	COMPLETED	Homoharringtonine Plus Low-Dose Cytarabine in Treating Patients With Newly Diagnosed Chronic Myelogenous Leukemia in Chronic Phase
NCT00006364	PHASE2	COMPLETED	Homoharringtonine in Treating Patients With Chronic Phase Chronic Myelogenous Leukemia
NCT00030355	PHASE1/PHASE2	WITHDRAWN	Homoharringtonine in Treating Patients With Refractory Acute Promyelocytic Leukemia
NCT00114959	PHASE2	TERMINATED	Homoharringtonine With Oral Gleevec in Chronic, Accelerated and Blast Phase Chronic Myeloid Leukemia (CML)
NCT00375219	PHASE2	COMPLETED	Homoharringtonine (Omacetaxine Mepesuccinate) in Treating Patients With Chronic Myeloid Leukemia (CML) With the T315I BCR-ABL Gene Mutation
NCT00462943	PHASE2	COMPLETED	Open Label Study of Subcutaneous Homoharringtonine (Omacetaxine Mepesuccinate) in Patients With Advanced CML
NCT02029417	PHASE2	TERMINATED	Omacetaxine Mepesuccinate, Cytarabine, and Decitabine in Treating Older Patients With Newly Diagnosed Acute Myeloid Leukemia
NCT02078960	PHASE1/PHASE2	TERMINATED	A Study to Evaluate the Pharmacokinetics, Safety,and Efficacy of Omacetaxine Given Subcutaneously as a Fixed Dose in Patients With Chronic Phase (CP) or Accelerated Phase (AP) Chronic Myeloid Leukemia (CML) (Referred to as the SYNSINCT Study)
NCT02440568	PHASE1/PHASE2	TERMINATED	AML-02: Omacetaxine With Standard-of-Care Induction With Cytarabine & Idarubicin in Newly-Diagnosed AML Patients
NCT04126681	PHASE2	COMPLETED	A Pivotal Study of HQP1351 in Patients With Chronic Myeloid Leukemia in Chronic Phase
NCT04248595	PHASE2	UNKNOWN	Study of Azacitidine Combined With Homoharringtonie Based Regimens in AML
NCT04874194	PHASE1/PHASE2	TERMINATED	Omacetaxine and Venetoclax for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome Harboring Mutant RUNX1
NCT05906914	PHASE2	COMPLETED	Cladribine Plus Homoharringtonine and Cytarabine Regimen (CHA) for de Novo Acute Myeloid Leukemia
NCT01844869	PHASE1	COMPLETED	An Open-Label Study to Investigate the Pharmacokinetics of Omacetaxine Mepesuccinate
NCT07516704	EARLY_PHASE1	NOT_YET_RECRUITING	Safety and Efficacy of Docetaxel, Darolutamide, and Homoharringtonine Combined With Androgen Deprivation Therapy in Neoadjuvant Treatment of High-Risk Prostate Cancer: A Multicenter Prospective, Single-Arm Clinical Study
NCT04505995	Not specified	UNKNOWN	Azacitidine and Homoharringtonine in JMML
NCT05805072	Not specified	UNKNOWN	Selinexor and HAAG With/Without HMA in Relapsed/Refractory Acute Leukemia (AML) Patients

Clinical evidence (CIViC)

Variant × indication × effect (1 predictive associations from 2 curated evidence items):

Variant	Indication	Effect	Therapy	Level	CIViC
BCR::ABL1 Fusion AND ABL1 T315I	Chronic Myeloid Leukemia	Sensitivity/Response	Omacetaxine Mepesuccinate	CIViC B	EID6197 +1

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).