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Mycophenolate Mofetil

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Also known as ArzipCellceptMyclausenMycophenolate mofetil tevaMycophenylate mofetilMyfenaxMyhibbinNSC-724229NSC-758905RS-61443SID29215409SID144205239Morpholinoethyl esterMYCOPHENOLATE_MOFETILSID170464859MycophenolateMycophenolate mofetilÊMycophenolate mofetilÂMYCOPHENOLATE-MOFETIL

Summary

Mycophenolate Mofetil (CHEMBL1456) is an approved small-molecule immunosuppressive agent targeting IMPDH1 and IMPDH2; indicated across 127 conditions including iga glomerulonephritis and nephrotic syndrome.

At a glance

  • Status: Approved (max clinical phase 4)
  • Modality: Small molecule
  • Targets: 2 (IMPDH1, IMPDH2)
  • Indications: 127 conditions
  • Clinical trials: 608
  • Chemistry: 433.5 Da · C23H31NO7

Identifiers

Drug identity and classification

FieldValue
ChEMBL IDCHEMBL1456
NameMycophenolate Mofetil
TypeSmall molecule
Max phase4
FDA approvedyes
PubChem CID5281078
ChEBICHEBI:8764
Molecular formulaC23H31NO7
Molecular weight433.5
InChIKeyRTGDFNSFWBGLEC-SYZQJQIISA-N

SMILES: CC1=C2COC(=O)C2=C(C(=C1OC)C/C=C(\C)/CCC(=O)OCCN3CCOCC3)O

IUPAC name: 2-morpholin-4-ylethyl (E)-6-(4-hydroxy-6-methoxy-7-methyl-3-oxo-1H-2-benzofuran-5-yl)-4-methylhex-4-enoate

ChEBI definition: A carboxylic ester resulting from the formal condensation between the carboxylic acid group of mycophenolic acid and the hydroxy group of 2-(morpholin-4-yl)ethanol. In the liver, it is metabolised to mycophenolic acid, an immunosuppressant for which it is a prodrug. It is widely used to prevent tissue rejection following organ transplants as well as for the treatment of certain autoimmune diseases.

Pharmacological roles (ChEBI): immunosuppressive agent, prodrug, EC 1.1.1.205 (IMP dehydrogenase) inhibitor, anticoronaviral agent.

Also known as: Arzip, Cellcept, Myclausen, Mycophenolate mofetil, Mycophenolate mofetil teva, Mycophenylate mofetil, Myfenax, Myhibbin, NSC-724229, NSC-758905, RS-61443, mycophenolate Mofetil

Parent form; salt/anhydrous children: CHEMBL1200955

Patent coverage: 13,242 distinct patent families (50,719 SureChEMBL compound mentions), from 2 matched compound structure(s). One matched structure accounts for 50,445 (99%) 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
IMPDH1inosine monophosphate dehydrogenase 1Inhibition8.8%P20839
IMPDH2inosine monophosphate dehydrogenase 2Inhibition48.5%P12268

Broader ChEMBL bioactivity targets: 9 (assay-derived). Sample: Prelamin-A/C, 15-hydroxyprostaglandin dehydrogenase [NAD(+)], ATP-binding cassette sub-family C member 4, Sodium-dependent serotonin transporter, Voltage-gated inwardly rectifying potassium channel KCNH2, cGMP-inhibited 3’,5’-cyclic phosphodiesterase 3A, Nuclear receptor subfamily 1 group I member 2, Aldehyde dehydrogenase 1A1, Bile salt export pump.

Bioactivity

ChEMBL activities: 2 potent at pChembl ≥ 5 of 9 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

TargetpChemblTypeValueUnitActivity ID
LMNA6.25Potency562.3nMCHEMBL_ACT_3655871
NR1I25.17AC506800nMCHEMBL_ACT_25188204

Target pathways

Aggregated over 2 target gene(s): IMPDH1, IMPDH2.

Top Reactome pathways

14 total, by targets touching each:

PathwayTargetsGenes
Metabolism2IMPDH1, IMPDH2
Metabolism of nucleotides2IMPDH1, IMPDH2
Disease2IMPDH1, IMPDH2
Innate Immune System2IMPDH1, IMPDH2
Immune System2IMPDH1, IMPDH2
Infectious disease2IMPDH1, IMPDH2
Neutrophil degranulation2IMPDH1, IMPDH2
Purine ribonucleoside monophosphate biosynthesis2IMPDH1, IMPDH2
Nucleotide biosynthesis2IMPDH1, IMPDH2
Potential therapeutics for SARS2IMPDH1, IMPDH2
SARS-CoV Infections2IMPDH1, IMPDH2
Drug ADME2IMPDH1, IMPDH2
Azathioprine ADME2IMPDH1, IMPDH2
Viral Infection Pathways2IMPDH1, IMPDH2

Dominant GO biological processes

GO termTargets
GMP biosynthetic process2
GTP biosynthetic process2
lymphocyte proliferation2
‘de novo’ XMP biosynthetic process2
purine nucleotide biosynthetic process2
circadian rhythm1
cellular response to interleukin-41

Indications & clinical

Indications

127 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
IgA glomerulonephritis3MONDO:0005342EFO:0004194
nephrotic syndrome3MONDO:0005377EFO:0004255
pemphigus vulgaris3MONDO:0008219EFO:0004719
hepatitis C virus infection3MONDO:0005231EFO:0003047
myasthenia gravis3MONDO:0009688EFO:0004991
graft versus host disease3MONDO:0013730EFO:0004599
cardiovascular disorder3MONDO:0004995EFO:0000319
uveitis3MONDO:0020283EFO:1001231
hepatocellular carcinoma3MONDO:0007256EFO:0000182
Hodgkins lymphoma3MONDO:0004952EFO:0000183
myelodysplastic syndrome3MONDO:0018881EFO:0000198
acute lymphoblastic leukemia3MONDO:0004967EFO:0000220
acute myeloid leukemia3MONDO:0018874EFO:0000222
chronic myeloid leukemia3MONDO:0011996EFO:0000339
diffuse large B-cell lymphoma3MONDO:0018905EFO:0000403
leukemia3MONDO:0005059EFO:0000565
neoplasm3MONDO:0005070EFO:0000616
plasma cell myeloma3MONDO:0009693EFO:0001378
kidney disorder3MONDO:0005240EFO:0003086
heart disorder3MONDO:0005267EFO:0003777
focal segmental glomerulosclerosis3MONDO:0100313EFO:0004236
interstitial lung disease3MONDO:0015925EFO:0004244
membranous glomerulonephritis3MONDO:0005376EFO:0004254
anti-neutrophil antibody associated vasculitis3MONDO:0005435EFO:0004826
granulomatosis with polyangiitis3MONDO:0012105EFO:0005297
lupus nephritis3MONDO:0005556EFO:0005761
non-Hodgkin lymphoma3MONDO:0018908EFO:0005952
autoimmune thrombocytopenic purpura3MONDO:0008558EFO:0007160
cauda equina syndrome3MONDO:0005693EFO:0007196
microscopic polyangiitis3MONDO:0019124EFO:1000784
Takayasu arteritis3MONDO:0017991EFO:1001857
Waldenstrom macroglobulinemia3MONDO:0100280EFO:0009441
mantle cell lymphoma3MONDO:0018876EFO:1001469
kidney failure3MONDO:0001106EFO:1002048
pure red-cell aplasia3MONDO:0001705MONDO:0001705
diabetic kidney disease3MONDO:0005016EFO:0000401
neuromyelitis optica3MONDO:0019100EFO:0004256
interstitial cystitis3MONDO:0018301EFO:0008507
type 1 diabetes mellitus3MONDO:0005147MONDO:0005147
systemic lupus erythematosus3MONDO:0007915MONDO:0007915
chronic kidney disease3MONDO:0005300MONDO:0024327
diffuse scleroderma2MONDO:0005019EFO:0000404
vasculitis2MONDO:0018882EFO:0006803
liver disorder2MONDO:0005154EFO:0001421
Sjogren syndrome2MONDO:0010030EFO:0000699
B-cell chronic lymphocytic leukemia2MONDO:0004948EFO:0000095
Crohn disease2MONDO:0005011EFO:0000384
cutaneous melanoma2MONDO:0005012EFO:0000389
lymphoma2MONDO:0005062EFO:0000574
neuroblastoma2MONDO:0005072EFO:0000621
psoriasis2MONDO:0005083EFO:0000676
systemic sclerosis2MONDO:0005100EFO:0000717
HIV infectious disease2MONDO:0005109EFO:0000764
age-related macular degeneration2MONDO:0005150EFO:0001365
lymphoid neoplasm2MONDO:0005157EFO:0001642
primary myelofibrosis2MONDO:0009692EFO:0002430
gout2MONDO:0005393EFO:0004274
autoimmune disease2MONDO:0007179EFO:0005140
aplastic anemia2MONDO:0015909EFO:0006927
B-cell prolymphocytic leukemia2MONDO:0019461EFO:1000102
juvenile myelomonocytic leukemia2MONDO:0011908EFO:1000309
T-cell prolymphocytic leukemia2MONDO:0019468EFO:1000560
epidermolysis bullosa2MONDO:0006541EFO:1000690
Sezary syndrome2MONDO:0017844EFO:1000785
immunoglobulin A vasculitis2MONDO:0019167EFO:1000965
mycosis fungoides2MONDO:0009691EFO:1001051
alcoholic hepatitis2MONDO:0001505EFO:1001345
hematologic disorder2MONDO:0005570HP:0001871
peripheral T-cell lymphoma, not otherwise specified2MONDO:0004964EFO:0000211
angioimmunoblastic T-cell lymphoma2MONDO:0004977EFO:0000255
Burkitt lymphoma2MONDO:0007243EFO:0000309
acquired polycythemia vera2MONDO:0009891EFO:0002429
anaplastic large cell lymphoma2MONDO:0020325EFO:0003032
bullous pemphigoid2MONDO:0019082EFO:0007187
mast cell leukemia2MONDO:0020334EFO:0007359
Langerhans cell histiocytosis2MONDO:0018310EFO:1000318
chronic myelomonocytic leukemia2MONDO:0020311EFO:1001779
myeloproliferative neoplasm2MONDO:0020076EFO:0002428
kidney cancer2MONDO:0002367MONDO:0002367
follicular lymphoma2MONDO:0018906MONDO:0018906
nasal cavity and paranasal sinus lethal midline granuloma2MONDO:0006828MONDO:0019472
hematopoietic and lymphoid system neoplasm2MONDO:0002334MONDO:0044881
drug-induced liver injury2MONDO:0005359EFO:0004228
mucous membrane pemphigoid2MONDO:0018746EFO:1000680
MALT lymphoma2MONDO:0007650EFO:0000191
inborn error of immunity2MONDO:0003778HP:0002721
amyotrophic lateral sclerosis2MONDO:0004976MONDO:0004976
multiple sclerosis2MONDO:0005301MONDO:0005301
familial lipoprotein lipase deficiency2MONDO:0009387MONDO:0009387
Wiskott-Aldrich syndrome2MONDO:0010518MONDO:0010518
sickle cell disease2MONDO:0011382MONDO:0011382
Diamond-Blackfan anemia2MONDO:0015253MONDO:0015253
severe combined immunodeficiency2MONDO:0015974MONDO:0015974
Fanconi anemia2MONDO:0019391MONDO:0019391
cardiomyopathy2MONDO:0004994EFO:0000318
hemoglobinuria2MONDO:0003656MONDO:0100244
plasma cell neoplasm2MONDO:0004959EFO:0000200
Graves disease2MONDO:0005364EFO:0004237
limited cutaneous systemic sclerosis2MONDO:0016358MONDO:0016358
exocrine pancreatic carcinoma1MONDO:0005192EFO:0002618
hypertensive disorder1MONDO:0005044EFO:0000537
renal cell carcinoma1MONDO:0005086EFO:0000681
beta thalassemia1MONDO:0019402Orphanet:848
rheumatoid arthritis1MONDO:0008383EFO:0000685
gliosarcoma1MONDO:0016681EFO:1001465
glioblastoma1MONDO:0018177EFO:0000519
small cell lung carcinoma1MONDO:0008433EFO:0000702
DiGeorge syndrome1MONDO:0008564MONDO:0018923
lymphoid leukemia1MONDO:0005402EFO:0004289
myeloid leukemia1MONDO:0004643MONDO:0004643
neuroepithelial neoplasm0MONDO:0021193MONDO:0021193

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

Clinical trials

Total trials: 608.

Phase distribution

PhaseTrials
PHASE2242
PHASE498
PHASE378
PHASE166
Not specified60
PHASE1/PHASE245
PHASE2/PHASE313
EARLY_PHASE16

Top trials by phase / activity

NCTPhaseStatusTitle
NCT02081755PHASE4ENROLLING_BY_INVITATIONSafety and Efficacy of Everolimus Treatment in Liver Transplantation for Liver Cancer
NCT05193565PHASE4ACTIVE_NOT_RECRUITINGStudy to Compare the Efficacy and Safety After Conversion to RaparoBell® or My-Rept® in Kidney Transplant Patients
NCT05207358PHASE4RECRUITINGMinimizing Glucocorticoid Administration in Patients With Proliferative Lupus Nephritis
NCT05916781PHASE4RECRUITINGEffectiveness of Mycophenolate Mofetil Combined With Tacrolimus for Steroid Tapering in Systemic Lupus Erythematosus
NCT07352566PHASE4NOT_YET_RECRUITINGUtilization of a Microdevice for Psoriasis and Atopic Dermatitis
NCT00048165PHASE4COMPLETEDA Study to Evaluate the Efficacy and Safety of Zenapax in Combination With CellCept, Cyclosporine, and Corticosteroids in Patients Undergoing Cardiac Transplantation
NCT00087581PHASE4COMPLETEDStudy of Therapeutic Monitoring of Mycophenolate Mofetil (MMF/CellCept) After Kidney Transplantation
NCT00113269PHASE4COMPLETEDSafety/Efficacy of Induction Agents With Tacrolimus, MMF, and Rapid Steroid Withdrawal in Renal Transplant Recipients
NCT00133172PHASE4TERMINATEDEffect of Rapid Steroid Withdrawal on Subclinical Markers of Rejection
NCT00160966PHASE4COMPLETEDImpact of Immunosuppressive Regimens on Polyomavirus-related Transplant Nephropathy
NCT00166244PHASE4COMPLETEDFixed Dose MMF vs Concentration Controlled MMF After Renal Transplantation
NCT00166829PHASE4UNKNOWNThe Effect of Sirolimus on the Pharmacokinetics of Tacrolimus
NCT00189150PHASE4COMPLETEDPharmacokinetics of Mmf and Valganciclovir
NCT00195988PHASE4COMPLETEDComparison of Immunosuppression Protocols After LTx in Children
NCT00199667PHASE4UNKNOWNConcentration Controlled Versus Fixed Dose of MMF in Kidney Transplant Recipients
NCT00200551PHASE4COMPLETEDA Study of Mycophenolate Mofetil and Cyclosporin, Without Concomitant Corticosteroids, After a First Renal Transplant
NCT00206076PHASE4COMPLETEDMycophenolate Mofetil Immunosuppression Without/With Reduced Dose Calcineurin Inhibitor Long After Liver Transplantation
NCT00217152PHASE4TERMINATEDA Kidney Transplant Study to Look at the Effects of Taking Fixed Doses of CellCept Versus Taking Doses of CellCept Based on the Concentration of CellCept in the Blood When Taking Full or Reduced Dose Calcineurin Inhibitors
NCT00261820PHASE4COMPLETEDStudy Comparing Two Immunosuppressive Regimens in De Novo Renal Allograft Recipients
NCT00275535PHASE4COMPLETEDThe Comparison of Tacrolimus and Sirolimus Immunosuppression Based Drug Regimens in Kidney Transplant Recipients
NCT00307671PHASE4COMPLETEDTreatment of Necrotizing Vasculitides for Patients Older Than 65 Years
NCT00352092PHASE4COMPLETEDPilot Study for HLA Identical Living Donor Renal Transplant Recipients
NCT00371319PHASE4COMPLETEDComparing the Efficacy of Tacrolimus and Mycophenolate Mofetil for the Initial Therapy of Active Lupus Nephritis
NCT00371826PHASE4COMPLETEDSOCRATES: Steroid or Cyclosporine Removal After Transplantation Using Everolimus
NCT00374231PHASE4COMPLETEDSingle Center Pilot Study of Corticosteroid Discontinuation in Liver Transplant Recipients
NCT00374647PHASE4COMPLETEDStudy to Evaluate Safety and Efficacy of Early Calcineurin Inhibitor Withdrawal in Primary Renal Allografts
NCT00400400PHASE4COMPLETEDEnteric-coated Mycophenolate Sodium (EC-MPS) and Mycophenolate Mofetil (MMF) in Renal Transplant Patients With Gastrointestinal (GI) Intolerance
NCT00400647PHASE4COMPLETEDGastrointestinal and Health-related Quality of Life in Kidney Transplant Patients Treated With Mycophenolate Mofetil
NCT00411515PHASE4COMPLETEDA Prospective Randomized Trial on Mycophenolate Mofetil in Risk Penetrating Keratoplasty
NCT00420537PHASE4TERMINATEDShift to Everolimus (RAD) Kidney Sparing Study
NCT00494741PHASE4COMPLETEDMMF vs. AZA for Kidney Transplantation
NCT00522548PHASE4TERMINATEDMyfortic or CellCept Gastrointestinal Effects in African American Kidney Recipients
NCT00545402PHASE4COMPLETEDA Study of CellCept (Mycophenolate Mofetil) in Combination Therapy in Liver Transplant Patients.
NCT00556933PHASE4COMPLETEDImproved Induction and Maintenance Immunosuppression in Kidney Transplantation
NCT00596947PHASE4TERMINATEDPrednisone Withdrawal Versus Prednisone Maintenance After Kidney Transplant
NCT00650468PHASE4COMPLETEDA Study to Compare Early Steroid Withdrawal and Long-Term Steroid Maintenance Therapy in Kidney Transplant Patients
NCT00656266PHASE4TERMINATEDTrial of Calcineurin Inhibitor-Sparing Immunosuppression Regimen in Pediatric Liver Transplantation
NCT00717314PHASE4COMPLETEDA Study of CellCept (Mycophenolate Mofetil) Combined With Calcineurin Inhibitors in Liver Transplant Patients.
NCT00717379PHASE4COMPLETEDStudy of Tacrolimus Immunosuppressive Therapy After Kidney Transplantation
NCT00717470PHASE4COMPLETEDA Study in Kidney Transplant Subjects to Investigate the Optimal Suppression of Immunity to Help Prevent Kidney Rejection

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline, but PharmGKB curates 20 clinical and 99 variant annotation(s) for this drug (gene-keyed; see PharmGKB).

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.

24 molecules share ≥1 primary target. Top 24 by shared-target count:

MoleculeSourceStatusShared targets
MYCOPHENOLIC ACIDChEMBL + PubChemPhase 4 (approved)IMPDH1, IMPDH2
MERIMEPODIBChEMBLPhase 2IMPDH1, IMPDH2
AdenosinePubChemApprovedIMPDH1
AfatinibPubChemApprovedIMPDH2
BinimetinibPubChemApprovedIMPDH2
CobimetinibPubChemApprovedIMPDH2
CrizotinibPubChemApprovedIMPDH2
dacomitinibPubChemApprovedIMPDH2
ErlotinibPubChemApprovedIMPDH2
FedratinibPubChemApprovedIMPDH2
FostamatinibPubChemApprovedIMPDH2
GefitinibPubChemApprovedIMPDH2
IdelalisibPubChemApprovedIMPDH2
LapatinibPubChemApprovedIMPDH2
NadidePubChemApprovedIMPDH1
PazopanibPubChemApprovedIMPDH2
regorafenibPubChemApprovedIMPDH2
RibavirinPubChemApprovedIMPDH1
SelumetinibPubChemApprovedIMPDH2
SorafenibPubChemApprovedIMPDH2
ThioguaninePubChemApprovedIMPDH1
TirbanibulinPubChemApprovedIMPDH2
TrametinibPubChemApprovedIMPDH2
VorinostatPubChemApprovedIMPDH1

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