Mycophenolic Acid

Summary

Mycophenolic Acid (CHEMBL866) is an approved small-molecule antineoplastic agent (ATC L04AA06) targeting IMPDH1 and IMPDH2; indicated across 19 conditions including immune system disorder and graft versus host disease.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: L04AA06
• Targets: 2 (IMPDH1, IMPDH2)
• Indications: 19 conditions
• Clinical trials: 53
• Chemistry: 320.3 Da · C17H20O6

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL866
Name	Mycophenolic Acid
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	446541
ChEBI	CHEBI:168396
ATC	L04AA06
Molecular formula	C17H20O6
Molecular weight	320.3
InChIKey	HPNSFSBZBAHARI-RUDMXATFSA-N

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

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

ChEBI definition: A member of the class of 2-benzofurans that is 2-benzofuran-1(3H)-one which is substituted at positions 4, 5, 6, and 7 by methyl, methoxy, (2E)-5-carboxy-3-methylpent-2-en-1-yl, and hydroxy groups, respectively. It is an antibiotic produced by Penicillium brevi-compactum, P. stoloniferum, P. echinulatum and related species. An immunosuppressant, it is widely used (partiularly as its sodium salt and as the 2-(morpholin-4-yl)ethyl ester prodrug, mycophenolate mofetil) to prevent tissue rejection following organ transplants and for the treatment of certain autoimmune diseases.

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

Other ChEBI roles (chemical / environmental): Penicillium metabolite, environmental contaminant, xenobiotic.

Also known as: Acide mycophenolique, Acido micofenolico, Mycophenolate, Mycophenolate mofetil impurity, mycophenolic acid-, Mycophenolic acid, NSC-129185, RS-61443 [AS MOFETIL], Mycophenolic Acid, mycophenolic acid, SID11112696, SID11112697

Parent form; salt/anhydrous children: CHEMBL2106643

Patent coverage: 21,382 distinct patent families (87,659 SureChEMBL compound mentions), from 2 matched compound structure(s). One matched structure accounts for 87,619 (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

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
IMPDH1	inosine monophosphate dehydrogenase 1	Inhibition	7.7	8.8%	P20839
IMPDH2	inosine monophosphate dehydrogenase 2	Inhibition	7.7	48.5%	P12268

Broader ChEMBL bioactivity targets: 14 (assay-derived). Sample: Lysine-specific demethylase 4E, Survival motor neuron protein, Prelamin-A/C, 15-hydroxyprostaglandin dehydrogenase [NAD(+)], Inosine-5’-monophosphate dehydrogenase 1, Inosine-5’-monophosphate dehydrogenase 2, Inosine-5’-monophosphate dehydrogenase (IMPDH), Cytochrome P450 1A2, Cytochrome P450 3A4, Aldehyde dehydrogenase 1A1.

Bioactivity

ChEMBL activities: 59 potent at pChembl ≥ 5 of 77 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
IMPDH2	8.22	Ki	6	nM	CHEMBL_ACT_92923
IMPDH2	8.15	Ki	7	nM	CHEMBL_ACT_2001019
IMPDH2	8.15	Ki	7	nM	CHEMBL_ACT_5241998
IMPDH2	8	Ki	10	nM	CHEMBL_ACT_2008226
IMPDH2	8	Ki	10	nM	CHEMBL_ACT_2259928
IMPDH2	8	Ki	10	nM	CHEMBL_ACT_2575558
IMPDH2	8	Ki	10	nM	CHEMBL_ACT_39950
IMPDH2	7.96	IC50	11	nM	CHEMBL_ACT_1747049
IMPDH2	7.92	IC50	12	nM	CHEMBL_ACT_3596387
IMPDH2	7.85	IC50	14	nM	CHEMBL_ACT_104732
IMPDH2	7.85	IC50	14	nM	CHEMBL_ACT_247760
IMPDH2	7.85	IC50	14	nM	CHEMBL_ACT_490697
IMPDH2	7.82	IC50	15	nM	CHEMBL_ACT_119352
IMPDH2	7.82	IC50	15	nM	CHEMBL_ACT_229371
IMPDH2	7.82	IC50	15	nM	CHEMBL_ACT_542727
IMPDH2	7.82	IC50	15	nM	CHEMBL_ACT_751419
IMPDH2	7.82	IC50	15	nM	CHEMBL_ACT_975090
IMPDH2	7.8	IC50	16	nM	CHEMBL_ACT_1909599
IMPDH1	7.72	IC50	19	nM	CHEMBL_ACT_3596393
IMPDH1	7.7	IC50	20	nM	CHEMBL_ACT_928730
IMPDH2	7.61	IC50	24.8	nM	CHEMBL_ACT_1102422
IMPDH2	7.6	IC50	25.1	nM	CHEMBL_ACT_1102421
IMPDH1	7.5	IC50	32	nM	CHEMBL_ACT_1747048
IMPDH1	7.48	Ki	33	nM	CHEMBL_ACT_2001007
IMPDH1	7.48	Ki	33	nM	CHEMBL_ACT_5241977
IMPDH1	7.48	Ki	33	nM	CHEMBL_ACT_92925
IMPDH1	7.4	Ki	40	nM	CHEMBL_ACT_2008219
IMPDH1	7.4	Ki	40	nM	CHEMBL_ACT_2259929
IMPDH1	7.4	Ki	40	nM	CHEMBL_ACT_2575557
IMPDH1	7.4	Ki	40	nM	CHEMBL_ACT_39949

Target pathways

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

Top Reactome pathways

14 total, by targets touching each:

Pathway	Targets	Genes
Metabolism	2	IMPDH1, IMPDH2
Metabolism of nucleotides	2	IMPDH1, IMPDH2
Disease	2	IMPDH1, IMPDH2
Innate Immune System	2	IMPDH1, IMPDH2
Immune System	2	IMPDH1, IMPDH2
Infectious disease	2	IMPDH1, IMPDH2
Neutrophil degranulation	2	IMPDH1, IMPDH2
Purine ribonucleoside monophosphate biosynthesis	2	IMPDH1, IMPDH2
Nucleotide biosynthesis	2	IMPDH1, IMPDH2
Potential therapeutics for SARS	2	IMPDH1, IMPDH2
SARS-CoV Infections	2	IMPDH1, IMPDH2
Drug ADME	2	IMPDH1, IMPDH2
Azathioprine ADME	2	IMPDH1, IMPDH2
Viral Infection Pathways	2	IMPDH1, IMPDH2

Dominant GO biological processes

GO term	Targets
GMP biosynthetic process	2
GTP biosynthetic process	2
lymphocyte proliferation	2
‘de novo’ XMP biosynthetic process	2
purine nucleotide biosynthetic process	2
circadian rhythm	1
cellular response to interleukin-4	1

Indications & clinical

Indications

19 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
immune system disorder	4	MONDO:0005046	EFO:0000540
graft versus host disease	3	MONDO:0013730	EFO:0004599
nephrotic syndrome	3	MONDO:0005377	EFO:0004255
lupus nephritis	3	MONDO:0005556	EFO:0005761
intermediate uveitis	3	MONDO:0006806	EFO:1000986
type 1 diabetes mellitus	3	MONDO:0005147	MONDO:0005147
systemic lupus erythematosus	3	MONDO:0007915	MONDO:0007915
pulmonary sarcoidosis	3	MONDO:0001708	DOID:13406
cytomegalovirus infection	3	MONDO:0005132	EFO:0001062
chronic kidney disease	2	MONDO:0005300	EFO:0003884
acute myeloid leukemia	2	MONDO:0018874	EFO:0000222
Crohn disease	2	MONDO:0005011	EFO:0000384
dry eye syndrome	2	MONDO:0006733	EFO:1000906
leukemia	2	MONDO:0005059	EFO:0000565
lymphoma	2	MONDO:0005062	EFO:0000574
systemic sclerosis	1	MONDO:0005100	EFO:0000717
myelodysplastic syndrome	1	MONDO:0018881	EFO:0000198
primary myelofibrosis	1	MONDO:0009692	MONDO:0044903

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

Clinical trials

Total trials: 53.

Phase distribution

Phase	Trials
PHASE4	16
PHASE2	11
PHASE3	9
Not specified	7
PHASE1/PHASE2	6
PHASE1	3
PHASE2/PHASE3	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT07352566	PHASE4	NOT_YET_RECRUITING	Utilization of a Microdevice for Psoriasis and Atopic Dermatitis
NCT00371826	PHASE4	COMPLETED	SOCRATES: Steroid or Cyclosporine Removal After Transplantation Using Everolimus
NCT00866684	PHASE4	TERMINATED	Prevention of Skin Cancer in High Risk Patients After Conversion to a Sirolimus-based Immunosuppressive Protocol
NCT01336296	PHASE4	COMPLETED	Evaluate Effects and Safety of Pre-load Myfortic® in Transplant Patients
NCT01560572	PHASE4	COMPLETED	Steroid Free Immunosuppression or Calcineurin Inhibitor Minimization After Basiliximab Induction Therapy in Kidney Transplantation: Comparison With a Standard Quadruple Immunosuppressive Regimen
NCT01927588	PHASE4	UNKNOWN	Evaluation of Early Use of Everolimus (EVE) on Cytomegalovirus (CMV) Infection in Renal Transplant Recipients
NCT02036554	PHASE4	UNKNOWN	Evaluate Efficacy Study of Combination Therapy of Everolimus and Low Dose Tacrolimus in Renal Allograft Recipients
NCT02123108	PHASE4	COMPLETED	Safety and Efficacy of Basiliximab, Delayed Dose Tacrolimus Plus ECMPA (Enteric Coated Mycophenolic Acid) Following Liver Transplantation
NCT02152345	PHASE4	COMPLETED	Belatacept Compared to Tacrolimus in Deceased Donor Renal Transplant Recipients
NCT02328963	PHASE4	COMPLETED	Proportion of CMV Seropositive Kidney Transplant Recipients Who Will Develop a CMV Infection When Treated With an Immunosuppressive Regimen Including Everolimus and Reduced Dose of Cyclosporine Versus an Immunosuppressive Regimen With Mycophenolic Acid and Standard Dose of Cyclosporine A
NCT02453867	PHASE4	UNKNOWN	Efficacy and Safety of a Reduced Immunosuppression vs. Standard Triple Therapy in Senior Renal Transplant Recipients
NCT02683291	PHASE4	COMPLETED	Sirolimus Associated With Tacrolimus at Low Doses in Elderly Kidney Transplant Patients
NCT02974686	PHASE4	TERMINATED	Conversion From MPA to Zortress (Everolimus) for GI Toxicity Post-renal Transplantation
NCT03099122	PHASE4	COMPLETED	A Study of Thymoglobuline® Induction Therapy in Adult Recipients of Donated After Cardiac Death Kidney Transplant
NCT03468478	PHASE4	COMPLETED	Comparison of the Efficacy and Safety of Sirolimus Versus Everolimus Versus Mycophenolate in Kidney Transplantation
NCT04424602	PHASE4	COMPLETED	Comparison between2 Drugs in Lupus Nephritis
NCT00149864	PHASE3	COMPLETED	Follow-up Study of Safety of Enteric-coated Mycophenolate Sodium in Patients Who Successfully Completed Study CERL080A302
NCT00149903	PHASE3	COMPLETED	Study of Enteric-coated Mycophenolate Sodium Versus Mycophenolate Mofetil in Adult de Novo Renal Transplant Patients
NCT00149916	PHASE3	COMPLETED	Follow-up Study of Safety of Enteric-coated Mycophenolate Sodium in Patients Who Successfully Completed Study CERL080A0107
NCT00149929	PHASE3	COMPLETED	Follow-up Study of Safety of Enteric-coated Mycophenolate Sodium in Patients Who Successfully Completed Study CERL080A301
NCT00262132	PHASE3	TERMINATED	Mycophenolate for Pulmonary Sarcoidosis
NCT00533624	PHASE2/PHASE3	COMPLETED	Myfortic vs. Cellcept in Kidney Transplant Recipients
NCT00966836	PHASE3	UNKNOWN	Prevention of Transplant Atherosclerosis With Everolimus and Anti-cytomegalovirus Therapy
NCT01299922	PHASE3	WITHDRAWN	Clinical Trial Treatment in Lupus Nephritis
NCT01625377	PHASE3	COMPLETED	A National Multi-center Randomized, Open Label Study to Evaluate Efficacy and Safety of Everolimus With EC-MPS Compared to Standard Treatment Combination Tacrolimus and EC-MPS in de Novo Liver Transplant Recipients
NCT04945096	PHASE3	UNKNOWN	Outcomes of Patients After Allo-HSCT With Decitabine and NAC
NCT05156710	PHASE2	ACTIVE_NOT_RECRUITING	BIVV020 (SAR445088) in Prevention and Treatment of Antibody-mediated Rejection (AMR)
NCT05436418	PHASE1/PHASE2	RECRUITING	The Lowest Effective Dose of Post-Transplantation Cyclophosphamide in Combination With Sirolimus and Mycophenolate Mofetil as Graft-Versus-Host Disease Prophylaxis After Reduced Intensity Conditioning and Peripheral Blood Stem Cell Transplantation
NCT07138898	PHASE2	NOT_YET_RECRUITING	Immunosuppressant Management in Rheumatology Patients Undergoing Elective Total Shoulder Arthroplasty
NCT00403416	PHASE1/PHASE2	COMPLETED	Efficacy and Safety of AEB071 Plus Tacrolimus (Converted to Mycophenolic Acid After 3 Months), in Renal Transplant Patients
NCT00622895	PHASE1/PHASE2	COMPLETED	Allogeneic Hematopoietic Cell Transplantation for Severe Systemic Sclerosis
NCT01053221	PHASE2	TERMINATED	Mycophenolic Acid Monotherapy in Recipients of HLA-identical Living-Related Transplantation
NCT01064791	PHASE2	COMPLETED	Efficacy, Safety, Tolerability, and Pharmacokinetics of Sotrastaurin Combined With Tacrolimus vs. a Mycophenolic Acid-tacrolimus Regimen in Renal Transplant Patients
NCT01379209	PHASE1/PHASE2	COMPLETED	Intravenous Administration of RGI-2001 in Patient Undergoing Allogenic Hematopoietic Stem Cell Transplantation (AHSCT)
NCT01490723	PHASE2	COMPLETED	Zevalin-Containing Nonmyeloablative Conditioning for Stem Cell Transplantation (SCT)
NCT01570348	PHASE2	TERMINATED	Crohn’s Allogeneic Transplant Study
NCT02137239	PHASE2	COMPLETED	Regimen Optimization Study
NCT02532777	PHASE2	UNKNOWN	The Research of Standard Diagnosis and Treatment for HSPN in Children
NCT02550652	PHASE2	COMPLETED	A Study to Evaluate the Safety and Efficacy of Obinutuzumab Compared With Placebo in Participants With Lupus Nephritis (LN)
NCT02711826	PHASE1/PHASE2	COMPLETED	Treg Therapy in Subclinical Inflammation in Kidney Transplantation

Clinical evidence (CIViC)

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

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline, but PharmGKB curates 11 clinical and 57 variant annotation(s) for this drug (gene-keyed; see PharmGKB).

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.

23 molecules share ≥1 primary target. Top 23 by shared-target count:

Molecule	Source	Status	Shared targets
MERIMEPODIB	ChEMBL	Phase 2	IMPDH1, IMPDH2
Adenosine	PubChem	Approved	IMPDH1
Afatinib	PubChem	Approved	IMPDH2
Binimetinib	PubChem	Approved	IMPDH2
Cobimetinib	PubChem	Approved	IMPDH2
Crizotinib	PubChem	Approved	IMPDH2
dacomitinib	PubChem	Approved	IMPDH2
Erlotinib	PubChem	Approved	IMPDH2
Fedratinib	PubChem	Approved	IMPDH2
Fostamatinib	PubChem	Approved	IMPDH2
Gefitinib	PubChem	Approved	IMPDH2
Idelalisib	PubChem	Approved	IMPDH2
Lapatinib	PubChem	Approved	IMPDH2
Nadide	PubChem	Approved	IMPDH1
Pazopanib	PubChem	Approved	IMPDH2
regorafenib	PubChem	Approved	IMPDH2
Ribavirin	PubChem	Approved	IMPDH1
Selumetinib	PubChem	Approved	IMPDH2
Sorafenib	PubChem	Approved	IMPDH2
Thioguanine	PubChem	Approved	IMPDH1
Tirbanibulin	PubChem	Approved	IMPDH2
Trametinib	PubChem	Approved	IMPDH2
Vorinostat	PubChem	Approved	IMPDH1