◎ Sugi Atlas

Atlas / Drug / Asciminib

Asciminib

drug
On this page

Also known as Abl-001ABL001NVP-ABL001AY7ASCIMINIB (ABL001)

Summary

Asciminib (CHEMBL4208229) is an approved small molecule (ATC L01EA06) targeting ABL1; indicated across 5 conditions including neoplasm and chronic myeloid leukemia; with CIViC clinical evidence for 3 variant-indication associations (e.g. BCR::ABL1 Fusion in chronic myeloid leukemia).

At a glance

  • Status: Approved (max clinical phase 4)
  • Modality: Small molecule
  • ATC class: L01EA06
  • Targets: 1 (ABL1)
  • Indications: 5 conditions
  • Clinical trials: 34
  • Precision-oncology evidence (CIViC): 3 variant–indication associations
  • Chemistry: 449.8 Da · C20H18ClF2N5O3

Identifiers

Drug identity and classification

FieldValue
ChEMBL IDCHEMBL4208229
NameAsciminib
TypeSmall molecule
Max phase4
FDA approvedyes
PubChem CID72165228
ATCL01EA06
Molecular formulaC20H18ClF2N5O3
Molecular weight449.8
InChIKeyVOVZXURTCKPRDQ-CQSZACIVSA-N

SMILES: C1CN(C[C@@H]1O)C2=C(C=C(C=N2)C(=O)NC3=CC=C(C=C3)OC(F)(F)Cl)C4=CC=NN4

IUPAC name: N-[4-[chloro(difluoro)methoxy]phenyl]-6-[(3R)-3-hydroxypyrrolidin-1-yl]-5-(1H-pyrazol-5-yl)pyridine-3-carboxamide

Also known as: Abl-001, ABL-001, ABL001, Asciminib, NVP-ABL001, AY7, ASCIMINIB, ASCIMINIB (ABL001)

Parent form; salt/anhydrous children: CHEMBL4297220

Patent coverage: 353 distinct patent families (882 SureChEMBL compound mentions), from 2 matched compound structure(s). One matched structure accounts for 729 (83%) 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

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

GeneTargetActionpAffinityCancer dependencyUniProt
ABL1ABL proto-oncogene 1, non-receptor tyrosine kinaseNegative9.521.2%P00519

Broader ChEMBL bioactivity targets: 4 (assay-derived). Sample: Tyrosine-protein kinase ABL1, Bcr/Abl fusion protein, Voltage-gated inwardly rectifying potassium channel KCNH2, Protein cereblon/Tyrosine-protein kinase ABL1/Breakpoint cluster region protein.

Bioactivity

ChEMBL activities: 10 potent at pChembl ≥ 5 of 11 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

TargetpChemblTypeValueUnitActivity ID
ABL19.89EC500.13nMCHEMBL_ACT_25662974
ABL19.35Kd0.45nMCHEMBL_ACT_24754914
ABL19.3IC500.5nMCHEMBL_ACT_18557369
ABL19.3Kd0.5nMCHEMBL_ACT_18557469
ABL18.41EC503.89nMCHEMBL_ACT_25662975
ABL18.24EC505.8nMCHEMBL_ACT_20666981
ABL18.19EC506.45nMCHEMBL_ACT_25662976
ABL18.13EC507.39nMCHEMBL_ACT_25662982
ABL17.8IC5016nMCHEMBL_ACT_29002802
ABL17.55EC5028.19nMCHEMBL_ACT_25662966

Target pathways

Aggregated over 1 target gene(s): ABL1.

Top Reactome pathways

53 total, by targets touching each:

PathwayTargetsGenes
Hemostasis1ABL1
Developmental Biology1ABL1
Signal Transduction1ABL1
Cell Cycle1ABL1
Disease1ABL1
Innate Immune System1ABL1
Immune System1ABL1
Signaling by Rho GTPases1ABL1
RHO GTPase Effectors1ABL1
Fcgamma receptor (FCGR) dependent phagocytosis1ABL1
Regulation of actin dynamics for phagocytic cup formation1ABL1
Epigenetic regulation of gene expression1ABL1
Generic Transcription Pathway1ABL1
Signaling by ROBO receptors1ABL1
Axon guidance1ABL1
Role of ABL in ROBO-SLIT signaling1ABL1
Mitotic G1 phase and G1/S transition1ABL1
Myogenesis1ABL1
Infectious disease1ABL1
RHO GTPases Activate WASPs and WAVEs1ABL1
HDR through Single Strand Annealing (SSA)1ABL1
DNA Double-Strand Break Repair1ABL1
Homology Directed Repair1ABL1
Recruitment and ATM-mediated phosphorylation of repair and signaling proteins at DNA double strand breaks1ABL1
HDR through Homologous Recombination (HRR) or Single Strand Annealing (SSA)1ABL1
DNA Double Strand Break Response1ABL1
Cyclin D associated events in G11ABL1
G1 Phase1ABL1
Cell Cycle, Mitotic1ABL1
RNA Polymerase II Transcription1ABL1

Dominant GO biological processes

GO termTargets
mitotic cell cycle1
neural tube closure1
B-1 B cell homeostasis1
B cell proliferation involved in immune response1
transitional one stage B cell differentiation1
mismatch repair1
regulation of DNA-templated transcription1
autophagy1
DNA damage response1
response to oxidative stress1
epidermal growth factor receptor signaling pathway1
positive regulation of cytosolic calcium ion concentration1
integrin-mediated signaling pathway1
canonical NF-kappaB signal transduction1
associative learning1

Indications & clinical

Indications

5 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).

IndicationTrial phaseMONDOEFO
neoplasm4MONDO:0005070EFO:0000616
chronic myeloid leukemia3MONDO:0011996EFO:0000339
acute lymphoblastic leukemia1MONDO:0004967EFO:0000220
kidney disorder1MONDO:0005240EFO:0003086

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

Clinical trials

Total trials: 34.

Phase distribution

PhaseTrials
PHASE213
PHASE16
PHASE35
Not specified5
PHASE1/PHASE23
PHASE41
EARLY_PHASE11

Top trials by phase / activity

NCTPhaseStatusTitle
NCT06427811PHASE4COMPLETEDClinical Study of Asciminib in Previously Treated Indian Patients With Ph+ CML-CP Without T315I Mutation and in Patients With Ph+ CML-CP With T315I Mutation
NCT04971226PHASE3ACTIVE_NOT_RECRUITINGA Study of Oral Asciminib Versus Other TKIs in Adult Patients With Newly Diagnosed Ph+ CML-CP
NCT05456191PHASE3ACTIVE_NOT_RECRUITINGA Study to Investigate Tolerability and Efficacy of Asciminib (Oral) Versus Nilotinib (Oral) in Adult Participants (≥18 Years of Age) With Newly Diagnosed Philadelphia Chromosome Positive Chronic Myelogenous Leukemia in Chronic Phase (Ph+ CML-CP)
NCT03106779PHASE3COMPLETEDStudy of Efficacy of CML-CP Patients Treated With ABL001 Versus Bosutinib, Previously Treated With 2 or More TKIs
NCT04666259PHASE3COMPLETEDAsciminib in Monotherapy for Chronic Myeloid Leukemia in Chronic Phase (CML-CP) With and Without T315I Mutation
NCT05413915PHASE3TERMINATEDAsciminib Used in Consolidation With Imatinib vs. Imatinib to Achieve TFR in CP-CML
NCT03906292PHASE2ACTIVE_NOT_RECRUITINGFrontline Asciminib Combination in Chronic Phase CML
NCT04838041PHASE2RECRUITINGProtocol Number: HJKC3-0003. Treatment Free Remission After Asciminib Based Therapy in Chronic Phase Chronic Myeloid Leukemia (CP-CML) Patients Who Relapsed After a Prior Attempt at TKI Discontinuation
NCT05384587PHASE2ACTIVE_NOT_RECRUITINGAsciminib Monotherapy, With Dose Escalation, for 2nd and 1st Line Chronic Myelogenous Leukemia
NCT06236724PHASE2RECRUITINGPhase II Study Assessing Efficacy and Safety of Asciminib in Patients With Newly Diagnosed Chronic Myeloid Leukemia in Chronic Phase.
NCT06308588PHASE1/PHASE2RECRUITINGPhase I/II Study of the Combination of Blinatumomab and Asciminib in Patients With Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia
NCT06368414PHASE2RECRUITINGA Study of Treatment-free Remission in Chronic Phase Chronic Myeloid Leukemia
NCT06409936PHASE2RECRUITINGPEARL Study: PotEntial of Asciminib in the eaRly Treatment of CML
NCT06514534PHASE2RECRUITINGOpen-label Study of Asciminib for CML-CP or CML-AP Patients With T315I Mutation Who Are Resistant, Intolerant or Ineligible to Ponatinib.
NCT06629584PHASE2RECRUITINGPhase II Study of Asciminib for Second-line Treatment of Chronic Phase Chronic Myeloid Leukemia
NCT07136428PHASE1/PHASE2NOT_YET_RECRUITINGAsciminib in HER2+ Breast Cancer Brain Metastases
NCT07493408PHASE2NOT_YET_RECRUITINGAsciminib & Standard-of-Care Integration in Maintenance Therapy for POST Allogeneic Stem Cell Transplant (Allo-HSCT) of Patient With Ph+ B-ALL or Blastic Transformed CML
NCT07604233PHASE1/PHASE2NOT_YET_RECRUITINGPhase 1/2 FLAG-IDA, VEN and Asciminib in CML and Ph+ AML
NCT03578367PHASE2COMPLETEDStudy of Efficacy and Safety of Asciminib in Combination With Imatinib in Patients With Chronic Myeloid Leukemia in Chronic Phase (CML-CP) Who Have Been Previously Treated With Imatinib and Have Not Achieved Deep Molecular Response.
NCT03874858PHASE2COMPLETEDA Study of Full Treatment-free Remission in Patients With Chronic Myeloid Leukemia
NCT04216563PHASE2TERMINATEDABL001 for the Treatment of Chronic Myeloid Leukemia in Patients Who Are on Therapy With Tyrosine Kinase Inhibitor
NCT04795427PHASE2COMPLETEDStudy of Efficacy and Safety of CML-CP Patients Treated With Asciminib Versus Best Available Therapy, Previously Treated With 2 or More Tyrosine Kinase Inhibitors
NCT03595917PHASE1RECRUITINGABL001 + Dasatinib + Prednisone + Blinatumomab in BCR-ABL+ B-ALL or CML
NCT07040982PHASE1NOT_YET_RECRUITINGAsciminib as Maintenance Treatment After Cellular Therapies for Adults With Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia
NCT07250087PHASE1RECRUITINGAsciminib Maintenance Therapy Following alloHCT or CAR T to Prevent Relapse in Adults With Ph+ALL
NCT02081378PHASE1COMPLETEDA Phase I Study of Oral Asciminib (ABL001) in Patients With CML or Ph+ ALL
NCT02857868PHASE1COMPLETEDA Trial to Evaluate the Pharmacokinetics of ABL001 in Healthy and Hepatic Impaired Subjects
NCT03605277PHASE1COMPLETEDPharmacokinetics Study of Asciminib in Subjects With Impaired Renal Function Compared to Matched Healthy Volunteers
NCT07039760EARLY_PHASE1NOT_YET_RECRUITINGAsciminib With or Without Sildenafil for Brain Tumors
NCT04360005Not specifiedAVAILABLEManaged Access Programs for ABL001, Asciminib
NCT05943522Not specifiedRECRUITINGAsciminib RMP Study
NCT06092879Not specifiedACTIVE_NOT_RECRUITINGAsciminib Prospective Non Interventional Study as 3rd Line Therapy or More to Treat Adult Patients With CML- CP in Real World Setting in France
NCT06684964Not specifiedRECRUITINGRWE,NIS,Prospective Study for the Effectiveness, Tolerability and Adherence of Asciminib in Saudi Arabia. (ASC4REAL)
NCT05421091Not specifiedCOMPLETEDSpecial Drug Use-results Surveillance of Scemblix Tablets

Clinical evidence (CIViC)

Variant × indication × effect (3 predictive associations from 4 curated evidence items):

VariantIndicationEffectTherapyLevelCIViC
BCR::ABL1 FusionChronic Myeloid LeukemiaSensitivity/ResponseAsciminibCIViC AEID11223 +1
ABL1 T315IChronic Myeloid LeukemiaSensitivity/ResponseAsciminibCIViC AEID11225
ZC3HAV1::ABL2 FusionB-lymphoblastic Leukemia/lymphoma With IAMP21Sensitivity/ResponseAsciminib + BCR-ABL Inhibitor HS-10382CIViC DEID12504

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline or drug-level clinical/variant annotations in PharmGKB for this molecule.

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.

108 molecules share ≥1 primary target. Top 60 by shared-target count:

MoleculeSourceStatusShared targets
AFATINIBChEMBL + PubChemPhase 4 (approved)ABL1
CRIZOTINIBChEMBL + PubChemPhase 4 (approved)ABL1
PAZOPANIBChEMBL + PubChemPhase 4 (approved)ABL1
REGORAFENIBChEMBL + PubChemPhase 4 (approved)ABL1
AFATINIB DIMALEATEChEMBLPhase 4 (approved)ABL1
AXITINIBChEMBLPhase 4 (approved)ABL1
BIOTINChEMBLPhase 4 (approved)ABL1
BOSUTINIBChEMBLPhase 4 (approved)ABL1
BRIGATINIBChEMBLPhase 4 (approved)ABL1
CABOZANTINIBChEMBLPhase 4 (approved)ABL1
CERITINIBChEMBLPhase 4 (approved)ABL1
DABRAFENIBChEMBLPhase 4 (approved)ABL1
DASATINIBChEMBLPhase 4 (approved)ABL1
ENTRECTINIBChEMBLPhase 4 (approved)ABL1
ERLOTINIBChEMBLPhase 4 (approved)ABL1
FEDRATINIBChEMBLPhase 4 (approved)ABL1
FILGOTINIBChEMBLPhase 4 (approved)ABL1
GEFITINIBChEMBLPhase 4 (approved)ABL1
IBRUTINIBChEMBLPhase 4 (approved)ABL1
IMATINIBChEMBLPhase 4 (approved)ABL1
INFIGRATINIBChEMBLPhase 4 (approved)ABL1
LAPATINIBChEMBLPhase 4 (approved)ABL1
LENVATINIBChEMBLPhase 4 (approved)ABL1
MEBENDAZOLEChEMBLPhase 4 (approved)ABL1
MIDOSTAURINChEMBLPhase 4 (approved)ABL1
NERATINIBChEMBLPhase 4 (approved)ABL1
NILOTINIBChEMBLPhase 4 (approved)ABL1
NINTEDANIBChEMBLPhase 4 (approved)ABL1
PONATINIBChEMBLPhase 4 (approved)ABL1
QUIZARTINIBChEMBLPhase 4 (approved)ABL1
RUXOLITINIBChEMBLPhase 4 (approved)ABL1
SORAFENIBChEMBLPhase 4 (approved)ABL1
SUNITINIBChEMBLPhase 4 (approved)ABL1
TIRBANIBULINChEMBLPhase 4 (approved)ABL1
TIVOZANIBChEMBLPhase 4 (approved)ABL1
TOFACITINIBChEMBLPhase 4 (approved)ABL1
TOVORAFENIBChEMBLPhase 4 (approved)ABL1
VANDETANIBChEMBLPhase 4 (approved)ABL1
ALISERTIBChEMBLPhase 3ABL1
ALVOCIDIBChEMBLPhase 3ABL1
BRIVANIBChEMBLPhase 3ABL1
CANERTINIBChEMBLPhase 3ABL1
CEDIRANIBChEMBLPhase 3ABL1
DEFACTINIBChEMBLPhase 3ABL1
DOVITINIBChEMBLPhase 3ABL1
FLUMATINIBChEMBLPhase 3ABL1
LESTAURTINIBChEMBLPhase 3ABL1
LINIFANIBChEMBLPhase 3ABL1
MASITINIBChEMBLPhase 3ABL1
MOTESANIBChEMBLPhase 3ABL1
OLVEREMBATINIBChEMBLPhase 3ABL1
RADOTINIBChEMBLPhase 3ABL1
RESVERATROLChEMBLPhase 3ABL1
SARACATINIBChEMBLPhase 3ABL1
SEMAXANIBChEMBLPhase 3ABL1
TANESPIMYCINChEMBLPhase 3ABL1
TESEVATINIBChEMBLPhase 3ABL1
ADAVOSERTIBChEMBLPhase 2ABL1
AEE-788ChEMBLPhase 2ABL1
APITOLISIBChEMBLPhase 2ABL1

About this page

Generated deterministically by Sugi Atlas from primary biomedical databases via BioBTree. Every record on this page traces to a primary source.

Generated
Atlas version
v1.1.2
BioBTree
v2.0.2

Sources 30

This page pulls from 30 datasets indexed by BioBTree:

chebichembl_activitychembl_moleculechembl_targetcivic_evidenceclingen_dosageclinical_trialsdepmapefoensemblentrezgogoparentgtopdbgtopdb_interactiongtopdb_ligandhgncmeshmondomsigdbpatent_compoundpharmgkbpharmgkb_guidelinepubchempubchem_activityreactomereactomeparentrefseqtranscriptuniprot