IMPDH2

Summary

IMPDH2 (inosine monophosphate dehydrogenase 2, HGNC:6053) is a protein-coding gene on chromosome 3p21.31, encoding Inosine-5’-monophosphate dehydrogenase 2 (P12268). Catalyzes the conversion of inosine 5’-phosphate (IMP) to xanthosine 5’-phosphate (XMP), the first committed and rate-limiting step in the de novo synthesis of guanine nucleotides, and therefore plays an important role in the regulation of cell growth. It is a selective cancer dependency (DepMap: 48.5% of cell lines).

This gene encodes the rate-limiting enzyme in the de novo guanine nucleotide biosynthesis. It is thus involved in maintaining cellular guanine deoxy- and ribonucleotide pools needed for DNA and RNA synthesis. The encoded protein catalyzes the NAD-dependent oxidation of inosine-5’-monophosphate into xanthine-5’-monophosphate, which is then converted into guanosine-5’-monophosphate. This gene is up-regulated in some neoplasms, suggesting it may play a role in malignant transformation.

Source: NCBI Gene 3615 — RefSeq curated summary.

At a glance

• Gene–disease (curated): neurodevelopmental disorder (Strong, GenCC)
• GWAS associations: 14
• Clinical variants (ClinVar): 96 total — 4 likely-pathogenic
• Phenotypes (HPO): 34
• Druggable target: yes — 2 molecules with ChEMBL bioactivity
• Cancer dependency (DepMap): dependent in 48.5% of screened cell lines
• MANE Select transcript: NM_000884

Identifiers

Gene identifiers

Field	Value
HGNC ID	HGNC:6053
Approved symbol	IMPDH2
Name	inosine monophosphate dehydrogenase 2
Location	3p21.31
Locus type	gene with protein product
Status	Approved
Ensembl gene	ENSG00000178035
Ensembl biotype	protein_coding
OMIM	146691
Entrez	3615

Gene structure

Transcript identifiers

Ensembl transcripts: 68 — 37 protein_coding, 29 retained_intron, 2 nonsense_mediated_decay

ENST00000326739, ENST00000429182, ENST00000442157, ENST00000462980, ENST00000463903, ENST00000466147, ENST00000472328, ENST00000481274, ENST00000484872, ENST00000485500, ENST00000491610, ENST00000496837, ENST00000676607, ENST00000676627, ENST00000676708, ENST00000676864, ENST00000677010, ENST00000677108, ENST00000677168, ENST00000677185, ENST00000677205, ENST00000677344, ENST00000677480, ENST00000677519, ENST00000677593, ENST00000677740, ENST00000677991, ENST00000678001, ENST00000678085, ENST00000678177, ENST00000678603, ENST00000678724, ENST00000678920, ENST00000679019, ENST00000679117, ENST00000679339, ENST00000881190, ENST00000881191, ENST00000881192, ENST00000881193, ENST00000881194, ENST00000881195, ENST00000881196, ENST00000937810, ENST00000937811, ENST00000937812, ENST00000937813, ENST00000937814, ENST00000937815, ENST00000937816, ENST00000937817, ENST00000937818, ENST00000937819, ENST00000937820, ENST00000937821, ENST00000937822, ENST00000937823, ENST00000937824, ENST00000937825, ENST00000937826, ENST00000968719, ENST00000968720, ENST00000968721, ENST00000968722, ENST00000968723, ENST00000968724, ENST00000968725, ENST00000968726

RefSeq mRNA: 4 — MANE Select: NM_000884 NM_000884, NM_001410759, NM_001410760, NM_001410761

CCDS: CCDS2786, CCDS93267, CCDS93268, CCDS93269

Canonical transcript exons

ENST00000326739 — 14 exons

Exon	Start	End
ENSE00001269198	49024495	49024578
ENSE00001269218	49025126	49025269
ENSE00001269252	49027710	49027916
ENSE00001269259	49028248	49028322
ENSE00001923105	49029253	49029398
ENSE00003468791	49026960	49027047
ENSE00003469699	49026519	49026609
ENSE00003471529	49024896	49025040
ENSE00003477985	49028758	49028806
ENSE00003503351	49026324	49026419
ENSE00003553096	49024659	49024802
ENSE00003597955	49028431	49028532
ENSE00003685190	49026687	49026886
ENSE00003688138	49024325	49024404

Expression profiles

Bgee: expression breadth ubiquitous, 294 present calls, max score 99.26.

FANTOM5 (CAGE): breadth ubiquitous, TPM avg 148.5913 / max 657.4576, expressed in 1820 samples.

FANTOM5 promoters (2 alternative TSS)

Promoter ID	TPM avg	Samples expressed
42191	140.1784	1820
42192	8.4128	1721

Top tissues by expression

295 total, by Bgee expression score (0-100, higher = more expressed):

Tissue	Anatomy ID	Expression score	Quality
left ovary	UBERON:0002119	99.26	gold quality
body of pancreas	UBERON:0001150	99.17	gold quality
cartilage tissue	UBERON:0002418	99.16	gold quality
right ovary	UBERON:0002118	99.14	gold quality
tongue squamous epithelium	UBERON:0006919	99.01	gold quality
right uterine tube	UBERON:0001302	98.85	gold quality
ovary	UBERON:0000992	98.80	gold quality
gingival epithelium	UBERON:0001949	98.79	gold quality
lower esophagus mucosa	UBERON:0035834	98.79	gold quality
ventricular zone	UBERON:0003053	98.77	gold quality
gingiva	UBERON:0001828	98.68	gold quality
endocervix	UBERON:0000458	98.64	gold quality
mammalian vulva	UBERON:0000997	98.56	gold quality
skin of leg	UBERON:0001511	98.56	gold quality
embryo	UBERON:0000922	98.55	gold quality
skin of abdomen	UBERON:0001416	98.55	gold quality
pancreas	UBERON:0001264	98.50	gold quality
zone of skin	UBERON:0000014	98.46	gold quality
ectocervix	UBERON:0012249	98.45	gold quality
nipple	UBERON:0002030	98.35	gold quality
body of uterus	UBERON:0009853	98.31	gold quality
esophagus mucosa	UBERON:0002469	98.28	gold quality
ganglionic eminence	UBERON:0004023	98.25	gold quality
gastrocnemius	UBERON:0001388	98.20	gold quality
penis	UBERON:0000989	98.18	gold quality
upper leg skin	UBERON:0004262	98.17	gold quality
rectum	UBERON:0001052	98.15	gold quality
left uterine tube	UBERON:0001303	98.14	gold quality
skin of hip	UBERON:0001554	98.13	gold quality
islet of Langerhans	UBERON:0000006	98.06	gold quality

Single-cell (SCXA)

Detected in 14 experiment(s), a significant marker in 13.

Experiment	Marker?	Max mean expression
E-GEOD-149689	yes	688.99
E-HCAD-4	yes	77.91
E-CURD-114	yes	68.36
E-HCAD-6	yes	29.68
E-HCAD-13	yes	26.68
E-MTAB-9067	yes	20.90
E-CURD-112	yes	19.46
E-CURD-122	yes	17.74
E-GEOD-125970	yes	17.20
E-MTAB-10042	yes	14.32
E-HCAD-1	yes	11.27
E-MTAB-9801	yes	6.66
E-MTAB-7008	no	472.75
E-ANND-3	no	0.00

Regulation

Is transcription factor: yes

Downstream targets (CollecTRI)

4 targets.

Target	Regulation
CCND1	Activation
CDKN1A	Repression
CDKN1B	Repression
MKI67	Activation

Upstream regulators (CollecTRI, top): ATF2, EGR1, MYC, NR1I2, SP1, TFAP2A

Functional genomics

DepMap (CRISPR cell-line fitness): dependent in 48.5% of screened cell lines.

Literature-anchored findings (GeneRIF, showing 40)

• Pharmacogenetic analysis of IMPDH II may represent a novel approach to mycophenolate mofetil therapeutic monitoring. (PMID:15621150)
• Nine genetic variants were identified in the IMPDH2 gene, with frequencies of the rarer alleles ranging from 0.5 to 10.2%. (PMID:17496727)
• IMPDH2 is a marker gene useful for stratifying osteosarcoma patients into low- and high-risk groups and predicting therapy outcome. (PMID:17660802)
• Mycophenolate mofetil up-regulates IMPDH-I and IMPDH-II mRNA in peripheral blood mononuclear cells. May predict acute rejection. (PMID:17713475)
• The effect of diabetes mellitus on mycophenolic acid pharmacokinetics and catalytic activity of inosine monophosphate dehydrogenase 2 was investigated in maintenance kidney transplant recipients. (PMID:18043470)
• The synthesis and activity of several novel inhibitors of IMPDH2 are reported. (PMID:18583139)
• Analysis of in vivo C-MYC interactions with TS, IMPDH2 and PRPS2 genes confirmed that they are direct C-MYC targets. (PMID:18677108)
• The expression of IMPDH2 in tumor tissue was significantly higher in patients with colorectal cancer than that in healthy subjects. (PMID:19597826)
• the IMPDH type II 3757T > C polymorphism is associated with an increased IMPDH activity in MMF-treated renal transplant patients (PMID:19617864)
• IMPDH2 genetic polymorphism was investigated in 96 individuals of Caucasian origin. (PMID:19810816)
• In this small sample of pediatric heart transplant patients receiving MMF, ABCC2, IMPDH1 and IMPDH2 SNPs were associated with MMF GI intolerance and bone marrow toxicity. (PMID:20061166)
• in the absence of ligand the mobilities of “flap” and “loop” regions of IMPDH-II (1B3O) structures are mostly controlled by the four (W 1, W 2, W 3, and W 4) conserved water molecular centers and their dynamics (PMID:20135634)
• Potential associations between the most frequent single nucleotide polymorphisms in both IMPDH genes and clinical outcome in renal transplant recipients. (PMID:20679962)
• resequenced IMPDH1 and IMPDH2 using DNA from 288 individuals from three ethnic groups and performed functional genomic studies of the sequence variants observed; identfied 25 SNPs, 24 novel, in IMPDH2 (PMID:20718729)
• IMPDH2 is directly involved in the development of chemoresistance in osteosarcoma cells (PMID:20808934)
• This ethnic-related data might contribute to a better understanding of the variability in clinical outcome and/or dose adjustments of drugs that are IMPDH inhibitors such as mycophenolic acid. (PMID:21181270)
• IMPDHII plays an important role in the negative regulation of TLR2 signaling by modulating PI3K activity. (PMID:21460227)
• the 3757C allele was associated with higher lymphocyte counts and a reduced incidence of lymphopenia among kidney allograft recipients (PMID:21996196)
• Anti-IMPDH2 may cause difficulties in interpretation of immunofluorescence patterns in routine autoantibody testing. (PMID:22029192)
• Role of salt bridge dynamics in inter domain recognition of human IMPDH isoforms: an insight to inhibitor topology for isoform-II. (PMID:22066532)
• An insight to the dynamics of conserved water-mediated salt bridge interaction and interdomain recognition in hIMPDH isoforms. (PMID:22928911)
• Rods and rings (RR) are protein assemblies composed of CTPS1 and IMPDH2. Glutamine deprivation initiates reversible assembly of mammalian rods and rings. (PMID:24477477)
• Compared with non-cancerous prostate tissues, IMPDH2 mRNA and protein expression levels were both significantly upregulated in prostate cancer tissues. (PMID:24659377)
• the localization of IMPDH2 inside the nucleus of human cells as well as the ultrastructure of R&R inclusions (PMID:24980853)
• Expression of IMPDH mRNA after mycophenolate administration in male volunteers. (PMID:25105143)
• IMPDH2 is directly involved in the development of chemoresistance and radioresistance in osteosarcoma cells. (PMID:25392102)
• The findings suggest that overexpressed IMPDH2 can be used as a biomarker for kidney and bladder cancer diagnosis and is a potential therapeutic target for the diseases (PMID:25465060)
• Rod and ring formation might be an adaptive homeostatic mechanism, allowing IMPDH to sense changes in the one-carbon folate pathway. (PMID:27343244)
• Taking together, these results indicate that natural product myricetin exhibits potent anti-leukemia activity by interfering with purine nucleotides biosynthetic pathway through the suppression of hIMPDH1/2 catalytic activity. (PMID:27378425)
• inosine-5’-monophosphate dehydrogenase 2 (IMPDH2) is an intracellular target of the PPIA-Sanglifehrin A binary complex. (PMID:28076787)
• Our study demonstrates IMPDH2 may be served as an independent prognostic biomarker for NPC patients, in which high IMPDH expression suggests poor prognosis of NPC patients. (PMID:28389646)
• association of T/C and C/C genotypes in the SNP rs11706052 (IMPDH2) with the occurrence of rejection episodes considering only patients who received immunosuppressive drug mycophenolate mofetil associated with cyclosporine or tacrolimus and corticoids, demonstrated association with a protection against rejection 15.6-fold (PMID:30442353)
• Study verified that overexpressing IMPDH2 could promote G1/S phase cell cycle transition through activation of PI3K/AKT/mTOR and PI3K/AKT/FOXO1 pathways and facilitate cell invasion, migration and EMT by regulating PI3K/AKT/mTOR pathway. (PMID:30518405)
• that upregulation of IMPDH2 is a prerequisite for the occurance of aberrant nucleolar function and increased anabolic processes in glioblastoma (PMID:31371825)
• Cryo-EM structures demonstrate human IMPDH2 filament assembly tunes allosteric regulation. (PMID:31999252)
• The expression and prognostic role of IMPDH2 in ovarian cancer. (PMID:32305001)
• Genetic variants in the glucocorticoid pathway genes and birth weight. (PMID:32886236)
• IMPDH2 and HPRT expression and a prognostic significance in preoperative and postoperative patients with osteosarcoma. (PMID:34035425)
• IMPDH2: a new gene associated with dominant juvenile-onset dystonia-tremor disorder. (PMID:34305140)
• Circular RNA circPFKP promotes cell proliferation by activating IMPDH2 in prostate cancer. (PMID:34673127)

Cross-species orthologs

3 orthologs

Organism	Symbol	Gene ID
danio_rerio	impdh2	ENSDARG00000006900
mus_musculus	Impdh2	ENSMUSG00000062867
rattus_norvegicus	Impdh2	ENSRNOG00000031965

Paralogs (3): GMPR2 (ENSG00000100938), IMPDH1 (ENSG00000106348), GMPR (ENSG00000137198)

Protein

Protein identifiers

Inosine-5’-monophosphate dehydrogenase 2 — P12268 (reviewed: P12268)

Alternative names: Inosine-5’-monophosphate dehydrogenase type II

All UniProt accessions (8): A0A384N6C2, A0A7I2V2T3, A0A7I2V337, A0A7I2V5N6, A0A7I2YQK5, E7ETK5, P12268, H0Y4R1

UniProt curated annotations — full annotation on UniProt →

Function. Catalyzes the conversion of inosine 5’-phosphate (IMP) to xanthosine 5’-phosphate (XMP), the first committed and rate-limiting step in the de novo synthesis of guanine nucleotides, and therefore plays an important role in the regulation of cell growth. Could also have a single-stranded nucleic acid-binding activity and could play a role in RNA and/or DNA metabolism. It may also have a role in the development of malignancy and the growth progression of some tumors.

Subunit / interactions. Homotetramer (PubMed:7903306, Ref.28, Ref.29). Interacts with CLOCK; in a circadian manner. Interacts with ANKRD9; leading to its ubiquitination and degradation by the proteasome.

Subcellular location. Cytoplasm. Nucleus. Cytosol.

Tissue specificity. IMPDH1 is the main species in normal leukocytes and IMPDH2 predominates over IMPDH1 in the tumor.

Post-translational modifications. Ubiquitinated leading to its degradation by the proteasome. The N-terminus is blocked. Acetylated by CLOCK in a circadian manner.

Activity regulation. Mycophenolic acid (MPA) is a non-competitive inhibitor that prevents formation of the closed enzyme conformation by binding to the same site as the amobile flap. In contrast, mizoribine monophosphate (MZP) is a competitive inhibitor that induces the closed conformation. MPA is a potent inhibitor of mammalian IMPDHs but a poor inhibitor of the bacterial enzymes. MZP is a more potent inhibitor of bacterial IMPDH. Subject to product inhibition by XMP and NADH. Also inhibited by ADP.

Induction. Selectively up-regulated in neoplastic and replicating cells.

Pathway. Purine metabolism; XMP biosynthesis via de novo pathway; XMP from IMP: step 1/1.

Polymorphism. Genetic variants in the IMPDH2 gene are responsible for the large inter-individual variability in enzyme activity and may influence immunosuppressive efficacy and side effects in transplant recipients receiving mycophenolic acid [MIM:617995].

Miscellaneous. Because IMPDH activity is tightly linked with cell proliferation, it has been recognized as a target for cancer and viral chemotherapy and as a target for immunosuppressive drugs. The activities of the antitumor drug tiazofurin, the antiviral drug ribavirin, and the immunosuppressive drugs mizoribine and mycophenolic acid (MPA) are attributed to the inhibition of IMPDH. In addition, bacterial and parasitic IMPDH’s differ significantly from mammalian enzymes, which makes it a suitable target for anti-infective drugs.

Similarity. Belongs to the IMPDH/GMPR family.

RefSeq proteins (4): NP_000875, NP_001397688, NP_001397689, NP_001397690 (=MANE)

Domains & families (InterPro)

ID	Name	Type
IPR000644	CBS_dom	Domain
IPR001093	IMP_DH_GMPRt	Domain
IPR005990	IMP_DH	Family
IPR013785	Aldolase_TIM	Homologous_superfamily
IPR015875	IMP_DH/GMP_Rdtase_CS	Conserved_site

Pfam: PF00478, PF00571

Enzyme classification (BRENDA):

• EC 1.1.1.205 — IMP dehydrogenase (BRENDA: 70 organisms, 74 substrates, 895 inhibitors, 205 Km, 88 kcat entries)

Substrate kinetics (BRENDA)

12 substrates with measured Km, best-characterized 12. Km ranges are aggregated across organisms/conditions.

Substrate	Km (mM)	Measurements
NAD+	0.0016–3.2	87
IMP	0.0033–0.315	46
INOSINE 5’-PHOSPHATE	0.0017–31	34
THIAZOLE-4-CARBOXAMIDE ADENINE DINUCLEOTIDE	0.59–1.03	6
K+	2–17	4
INOSINE 5’-DIPHOSPHATE	0.0082–0.03	3
3-ACETYLPYRIDINE ADENINE DINUCLEOTIDE	0.19–0.22	2
5’-AMINO-5’-DEOXYINOSINE 5’-N-PHOSPHATE	0.038	1
5’-MERCAPTO-5’-DEOXYINOSINE 5’-S-PHOSPHATE	0.013	1
6-THIO-IMP	0.02	1
ACETYLPYRIDINE ADENINE DINUCLEOTIDE	0.19	1
INOSINE 5’-PHOSPHOROTHIOATE	0.21	1

Catalyzed reactions (Rhea), 1 shown:

• IMP + NAD(+) + H2O = XMP + NADH + H(+) (RHEA:11708)

UniProt features (88 total): strand 32, helix 21, binding site 13, turn 6, modified residue 5, cross-link 3, domain 2, active site 2, initiator methionine 1, chain 1, sequence variant 1, sequence conflict 1

Structure

Experimental structures (PDB)

25 structures.

PDB	Method	Resolution (Å)
9DMU	ELECTRON MICROSCOPY	1.82
8FOZ	ELECTRON MICROSCOPY	2
8FUZ	ELECTRON MICROSCOPY	2.1
9MUC	ELECTRON MICROSCOPY	2.1
9MUB	ELECTRON MICROSCOPY	2.5
6I0M	X-RAY DIFFRACTION	2.57
8G8F	ELECTRON MICROSCOPY	2.6
6I0O	X-RAY DIFFRACTION	2.62
1NF7	X-RAY DIFFRACTION	2.65
1B3O	X-RAY DIFFRACTION	2.9
1NFB	X-RAY DIFFRACTION	2.9
6UDP	ELECTRON MICROSCOPY	2.95
8G9B	ELECTRON MICROSCOPY	3
6U8E	ELECTRON MICROSCOPY	3.03
6U8S	ELECTRON MICROSCOPY	3.14
6UDO	ELECTRON MICROSCOPY	3.21
6UDQ	ELECTRON MICROSCOPY	3.27
6U8N	ELECTRON MICROSCOPY	3.29
6U9O	ELECTRON MICROSCOPY	3.36
6UA4	ELECTRON MICROSCOPY	3.65
6UA5	ELECTRON MICROSCOPY	3.79
6UAJ	ELECTRON MICROSCOPY	3.84
6U8R	ELECTRON MICROSCOPY	3.91
6UA2	ELECTRON MICROSCOPY	4.2
6UC2	ELECTRON MICROSCOPY	4.48

Predicted structure (AlphaFold)

Model	pLDDT	Fraction very-high
AF-P12268-F1	92.07	0.73

Functional residue map

Curated UniProt residues grouped by drug-discovery relevance — catalytic, ligand-binding, modification, and mutation-validated positions. Source: UniProtKB sequence features.

Catalytic / active sites (2): 331 (thioimidate intermediate); 429 (proton acceptor)

Ligand- & substrate-binding residues (13): 329; 331 (in other chain); 364–366; 387–388; 411–415; 441; 500; 501; 502; 274–276; 324–326; 326 (in other chain) …

Post-translational modifications (8): 122, 160, 400, 416, 511, 195, 208, 438

Function

Pathways and Gene Ontology

Reactome pathways

14 pathways

ID	Pathway
R-HSA-6798695	Neutrophil degranulation
R-HSA-73817	Purine ribonucleoside monophosphate biosynthesis
R-HSA-9679191	Potential therapeutics for SARS
R-HSA-9748787	Azathioprine ADME
R-HSA-1430728	Metabolism
R-HSA-15869	Metabolism of nucleotides
R-HSA-1643685	Disease
R-HSA-168249	Innate Immune System
R-HSA-168256	Immune System
R-HSA-5663205	Infectious disease
R-HSA-8956320	Nucleotide biosynthesis
R-HSA-9679506	SARS-CoV Infections
R-HSA-9748784	Drug ADME
R-HSA-9824446	Viral Infection Pathways

MSigDB gene sets: 387 (showing top): GOBP_CIRCADIAN_RHYTHM, MODULE_93, E2F_Q4_01, HORIUCHI_WTAP_TARGETS_DN, REACTOME_INNATE_IMMUNE_SYSTEM, SHIPP_DLBCL_VS_FOLLICULAR_LYMPHOMA_UP, WWTAAGGC_UNKNOWN, GRUETZMANN_PANCREATIC_CANCER_DN, GOBP_RESPONSE_TO_INTERLEUKIN_4, GOBP_RESPONSE_TO_PEPTIDE, MODULE_56, NKX25_02, GOCC_SECRETORY_GRANULE, MODULE_151, GOBP_ORGANOPHOSPHATE_METABOLIC_PROCESS

GO Biological Process (7): GMP biosynthetic process (GO:0006177), GTP biosynthetic process (GO:0006183), circadian rhythm (GO:0007623), lymphocyte proliferation (GO:0046651), cellular response to interleukin-4 (GO:0071353), ‘de novo’ XMP biosynthetic process (GO:0097294), purine nucleotide biosynthetic process (GO:0006164)

GO Molecular Function (8): nucleotide binding (GO:0000166), DNA binding (GO:0003677), RNA binding (GO:0003723), IMP dehydrogenase activity (GO:0003938), metal ion binding (GO:0046872), catalytic activity (GO:0003824), protein binding (GO:0005515), oxidoreductase activity (GO:0016491)

GO Cellular Component (9): extracellular region (GO:0005576), nucleus (GO:0005634), cytoplasm (GO:0005737), peroxisomal membrane (GO:0005778), cytosol (GO:0005829), membrane (GO:0016020), secretory granule lumen (GO:0034774), extracellular exosome (GO:0070062), ficolin-1-rich granule lumen (GO:1904813)

Reactome top-level categories

Rollup of top-10 pathways:

Category	Pathways
Innate Immune System	1
Nucleotide biosynthesis	1
SARS-CoV Infections	1
Drug ADME	1
Metabolism	1
Immune System	1
Disease	1
Metabolism of nucleotides	1
Viral Infection Pathways	1
Infectious disease	1

GO top-level categories

Rollup of top GO terms by namespace:

Category	Terms
cellular anatomical structure	4
purine ribonucleotide biosynthetic process	2
nucleic acid binding	2
purine ribonucleoside monophosphate biosynthetic process	1
GMP metabolic process	1
purine ribonucleoside triphosphate biosynthetic process	1
GTP metabolic process	1
rhythmic process	1
mononuclear cell proliferation	1
lymphocyte activation	1
response to interleukin-4	1
cellular response to cytokine stimulus	1
XMP biosynthetic process	1
purine nucleotide metabolic process	1
nucleotide biosynthetic process	1
purine-containing compound biosynthetic process	1
nucleoside phosphate binding	1
heterocyclic compound binding	1
oxidoreductase activity, acting on the CH-OH group of donors, NAD or NADP as acceptor	1
cation binding	1
molecular_function	1
binding	1
catalytic activity	1
intracellular membrane-bounded organelle	1
intracellular anatomical structure	1
peroxisome	1
microbody membrane	1
cytoplasm	1
secretory granule	1
cytoplasmic vesicle lumen	1
extracellular vesicle	1
intracellular organelle lumen	1
ficolin-1-rich granule	1

Protein interactions and networks

STRING

3842 interactions, top by confidence (×1000):

Protein A	Protein B	Partner UniProt	Score
IMPDH2	GMPS	P49915	877
IMPDH2	CTPS1	P17812	816
IMPDH2	ADSS2	P30520	797
IMPDH2	P0DN79	P0DN79	737
IMPDH2	ADSL	P30566	725
IMPDH2	APRT	P07741	673
IMPDH2	PPAT	Q06203	665
IMPDH2	ITPA	Q9BY32	657
IMPDH2	GART	P22102	648
IMPDH2	DHODH	Q02127	642
IMPDH2	ATIC	P31939	638
IMPDH2	HPRT1	P00492	634
IMPDH2	PNP	P00491	625
IMPDH2	ADK	P55263	622
IMPDH2	GDA	Q9Y2T3	614

IntAct

189 interactions, top by confidence:

A	B	Type	Score
CDK2	CCNE2	psi-mi:“MI:0914”(association)	0.940
MAPK14	RPS6KA4	psi-mi:“MI:0914”(association)	0.870
XPC	PARP1	psi-mi:“MI:0914”(association)	0.730
IMPDH2	CPLANE2	psi-mi:“MI:0915”(physical association)	0.720
CPLANE2	IMPDH2	psi-mi:“MI:0915”(physical association)	0.720
CFTR	ESYT2	psi-mi:“MI:2364”(proximity)	0.710
CFTR	ESYT2	psi-mi:“MI:0914”(association)	0.710
IMPDH2	STAT3	psi-mi:“MI:0915”(physical association)	0.670
SH3KBP1	USP27X	psi-mi:“MI:0914”(association)	0.640
NCBP2	KPNA3	psi-mi:“MI:0914”(association)	0.640
IMPDH2	APIP	psi-mi:“MI:0915”(physical association)	0.560
APIP	IMPDH2	psi-mi:“MI:0915”(physical association)	0.560
TAE1	IMPDH2	psi-mi:“MI:0915”(physical association)	0.560
IMPDH2	ISU1	psi-mi:“MI:0915”(physical association)	0.560
IMPDH2	TRAF2	psi-mi:“MI:0915”(physical association)	0.560
IMPDH2	IMPDH1	psi-mi:“MI:0915”(physical association)	0.560
IMPDH2	POU6F2	psi-mi:“MI:0915”(physical association)	0.560

BioGRID (556): IMPDH2 (Affinity Capture-MS), IMPDH2 (Two-hybrid), APIP (Two-hybrid), RSG1 (Two-hybrid), IMPDH2 (Affinity Capture-MS), IMPDH2 (Affinity Capture-MS), IMPDH2 (Affinity Capture-MS), IMPDH1 (Affinity Capture-MS), SKP2 (Affinity Capture-MS), SMCR8 (Affinity Capture-MS), ANKRD9 (Affinity Capture-MS), PUSL1 (Affinity Capture-MS), ASUN (Co-fractionation), CD2AP (Co-fractionation), DDX3X (Co-fractionation)

ESM2 similar proteins: A0A0B5L585, A0JNA3, B0UXP9, D3ZLZ7, E9BDA8, E9PU28, F1DBB2, F6S675, F7CYY5, O00086, O14344, O33655, O93937, P07259, P07756, P12268, P12269, P20839, P21620, P24547, P31327, P38697, P39567, P47996, P50094, P50095, P50096, P50097, P50098, Q07152, Q12658, Q3SWY3, Q49729, Q4QEB3, Q4VRV8, Q4WHZ9, Q54P92, Q54QQ0, Q59Q46, Q5KP44

Diamond homologs: A0A0B5L585, A0JNA3, A7Z8C3, A9A5Y7, B0UXP9, B1L5U5, B9DP67, C0MAM1, D3ZLZ7, E9BDA8, E9PU28, F1DBB2, F6S675, F7CYY5, O00086, O14344, O42831, O50316, O58045, O67820, P0ADG7, P0ADG8, P0ADG9, P0C0H6, P0C0H7, P0DB88, P0DB89, P12268, P12269, P20839, P21620, P21879, P24547, P31002, P38697, P39567, P42851, P44334, P47996, P49058

SIGNOR signaling

14 interactions.

A	Effect	B	Mechanism
IMPDH2	“up-regulates quantity by expression”	CCND1	“transcriptional regulation”
IMPDH2	“up-regulates quantity by expression”	MKI67	“transcriptional regulation”
IMPDH2	“down-regulates quantity by repression”	CDKN1A	“transcriptional regulation”
IMPDH2	“down-regulates quantity by repression”	CDKN1B	“transcriptional regulation”
IMPDH2	“up-regulates activity”	AKT	binding
ribavirin	“down-regulates activity”	IMPDH2	“chemical inhibition”
“Sanglifehrin A”	“down-regulates activity”	IMPDH2	“chemical inhibition”
Merimepodib	“down-regulates activity”	IMPDH2	“chemical inhibition”
IMPDH2	“down-regulates quantity”	IMP	“chemical modification”
IMPDH2	“up-regulates quantity”	“5’-xanthylic acid”	“chemical modification”
MYC	“up-regulates quantity by expression”	IMPDH2	“transcriptional regulation”
“mycophenolic acid”	“down-regulates activity”	IMPDH2	“chemical inhibition”
AKT1	“up-regulates activity”	IMPDH2	phosphorylation

Enriched among interaction partners

Reactome pathways and GO biological processes over-represented among this gene’s 183 IntAct physical interaction partners (hypergeometric vs the genome-wide background, BH-FDR, gene-set size 15–500, ranked by fold). A functional readout of the neighbourhood — distinct from this gene’s own memberships above, and biased toward well-studied / hub proteins, so read it as themes rather than proof.

GO biological processes:

GO term	Partners	Fold	FDR
stress granule assembly	5	18.6×	5e-03
positive regulation of axon extension	5	15.8×	6e-03
DNA damage response	14	4.6×	3e-03

Disease & clinical

Clinical variants and AI predictions

ClinVar

96 variants total. Per-class counts are floors (≥ shown; pagination cap):

Classification	Count (floor)
Pathogenic	0
Likely pathogenic	4
Uncertain significance	68
Likely benign	3
Benign	2

Top pathogenic / likely-pathogenic (4)

Variant ID	HGVS	Classification
1164002	NM_000884.3(IMPDH2):c.93_96del (p.Tyr32fs)	Likely pathogenic
1803982	NM_000884.3(IMPDH2):c.338G>A (p.Gly113Glu)	Likely pathogenic
1966023	NM_000884.3(IMPDH2):c.713A>G (p.Lys238Arg)	Likely pathogenic
3383473	NM_000884.3(IMPDH2):c.619G>A (p.Gly207Arg)	Likely pathogenic

SpliceAI

2191 predictions. Top by Δscore:

Variant	Effect	Δscore
3:49024574:TGGCT:T	acceptor_gain	1.0000
3:49024577:CT:C	acceptor_gain	1.0000
3:49024578:TC:T	acceptor_loss	1.0000
3:49024579:C:CC	acceptor_gain	1.0000
3:49024579:C:T	acceptor_loss	1.0000
3:49024657:A:AC	donor_gain	1.0000
3:49024658:C:CC	donor_gain	1.0000
3:49024658:CCGGA:C	donor_gain	1.0000
3:49024798:CTTCA:C	acceptor_gain	1.0000
3:49024800:TCA:T	acceptor_gain	1.0000
3:49024800:TCAC:T	acceptor_loss	1.0000
3:49024801:CA:C	acceptor_gain	1.0000
3:49024801:CAC:C	acceptor_gain	1.0000
3:49024803:C:CC	acceptor_gain	1.0000
3:49024803:C:CG	acceptor_loss	1.0000
3:49024891:CCCA:C	donor_loss	1.0000
3:49024892:CCA:C	donor_loss	1.0000
3:49024894:A:T	donor_loss	1.0000
3:49024904:T:C	donor_gain	1.0000
3:49025036:CATGA:C	acceptor_gain	1.0000
3:49025037:ATGA:A	acceptor_gain	1.0000
3:49025038:TGA:T	acceptor_gain	1.0000
3:49025039:GA:G	acceptor_gain	1.0000
3:49025040:AC:A	acceptor_loss	1.0000
3:49025041:C:CC	acceptor_gain	1.0000
3:49025041:C:CG	acceptor_loss	1.0000
3:49025042:T:C	acceptor_loss	1.0000
3:49025044:C:CT	acceptor_gain	1.0000
3:49025048:C:CT	acceptor_gain	1.0000
3:49025121:CTCA:C	donor_loss	1.0000

AlphaMissense

3381 scored. Top likely-pathogenic:

Variant	Protein change	am_pathogenicity
3:49024773:C:T	G442E	1.000
3:49024774:C:G	G442R	1.000
3:49024774:C:T	G442R	1.000
3:49024949:C:A	M414I	1.000
3:49024949:C:G	M414I	1.000
3:49024949:C:T	M414I	1.000
3:49024964:C:A	K409N	1.000
3:49024964:C:G	K409N	1.000
3:49025182:C:T	G365E	1.000
3:49026337:G:C	C331W	1.000
3:49026848:C:G	A220P	1.000
3:49026880:A:G	L209S	1.000
3:49027759:C:A	R161M	1.000
3:49027774:C:T	G156D	1.000
3:49027775:C:G	G156R	1.000
3:49027824:G:C	F139L	1.000
3:49027824:G:T	F139L	1.000
3:49027826:A:G	F139L	1.000
3:49027899:G:C	F114L	1.000
3:49027899:G:T	F114L	1.000
3:49027901:A:G	F114L	1.000
3:49024517:C:G	G501R	0.999
3:49024710:C:T	G463D	0.999
3:49024711:C:G	G463R	0.999
3:49024719:A:G	L460P	0.999
3:49024764:C:T	G445D	0.999
3:49024779:G:T	A440D	0.999
3:49024903:A:G	Y430H	0.999
3:49024948:C:G	G415R	0.999
3:49024950:A:G	M414T	0.999

dbSNP variants (sampled 300 via entrez): RS1000245176 (3:49027122 G>T), RS1002423663 (3:49026858 A>G,T), RS1002729214 (3:49028476 A>G), RS1002784462 (3:49024589 G>A,C), RS1002815120 (3:49024927 T>C), RS1002853413 (3:49031227 A>C), RS1003326765 (3:49029536 G>A,C,T), RS1003518601 (3:49025837 G>A), RS1003573887 (3:49030127 G>A,C), RS1003579148 (3:49029689 A>G,T), RS1004472994 (3:49028218 G>A,T), RS1005539460 (3:49026046 C>G), RS1005979311 (3:49031185 T>C), RS1006378892 (3:49030959 C>G), RS1006463705 (3:49024914 C>T)

Disease associations

OMIM: gene MIM:146691 | disease phenotypes:

GenCC curated gene-disease

Disease	Classification	Inheritance
neurodevelopmental disorder	Strong	Autosomal dominant

Mondo (2): dystonic disorder (MONDO:0003441), neurodevelopmental disorder (MONDO:0700092)

Orphanet (0):

HPO phenotypes

34 total (30 of 34 shown, HPO-id order):

HPO	Term
HP:0000365	Hearing impairment
HP:0000473	Torticollis
HP:0000666	Horizontal nystagmus
HP:0000716	Depression
HP:0000722	Compulsive behaviors
HP:0000739	Anxiety
HP:0000821	Hypothyroidism
HP:0000822	Hypertension
HP:0001249	Intellectual disability
HP:0001251	Ataxia
HP:0001300	Parkinsonism
HP:0001348	Brisk reflexes
HP:0001370	Rheumatoid arthritis
HP:0001761	Pes cavus
HP:0001762	Talipes equinovarus
HP:0002063	Rigidity
HP:0002066	Gait ataxia
HP:0002067	Bradykinesia
HP:0002071	Abnormality of extrapyramidal motor function
HP:0002166	Impaired vibration sensation in the lower limbs
HP:0002174	Postural tremor
HP:0002360	Sleep disturbance
HP:0002395	Lower limb hyperreflexia
HP:0002451	Limb dystonia
HP:0002601	Paresis of extensor muscles of the big toe
HP:0002650	Scoliosis
HP:0003487	Babinski sign
HP:0003785	Decreased CSF homovanillic acid concentration
HP:0004373	Focal dystonia
HP:0005876	Progressive flexion contractures

GWAS associations

14 associations (top):

Study	Trait	p-value
GCST004131_23	Inflammatory bowel disease	1.000000e-33
GCST004132_17	Crohn’s disease	3.000000e-23
GCST004133_11	Ulcerative colitis	8.000000e-20
GCST007294_107	Body fat distribution (trunk fat ratio)	9.000000e-06
GCST007294_72	Body fat distribution (trunk fat ratio)	1.000000e-18
GCST007294_98	Body fat distribution (trunk fat ratio)	3.000000e-15
GCST007295_21	Body fat distribution (leg fat ratio)	8.000000e-06
GCST007295_45	Body fat distribution (leg fat ratio)	1.000000e-10
GCST007295_80	Body fat distribution (leg fat ratio)	1.000000e-14
GCST010698_80	Subcortical volume (min-P)	3.000000e-24
GCST010699_110	Brain morphology (min-P)	4.000000e-08
GCST010701_52	Cortical surface area (MOSTest)	1.000000e-16
GCST010702_36	Subcortical volume (MOSTest)	1.000000e-10
GCST010703_262	Brain morphology (MOSTest)	2.000000e-13

EFO canonical traits (2, from GWAS)

EFO ID	Trait name
EFO:0004341	body fat distribution
EFO:0004346	neuroimaging measurement

MeSH disease descriptors (2)

Descriptor	Name	Tree numbers
D020821	Dystonic Disorders	C10.228.662.300
D065886	Neurodevelopmental Disorders	F03.625

Drugs & pharmacology

Drug and pharmacology data

Is drug target: yes

ChEMBL targets (2): CHEMBL2002 (SINGLE PROTEIN), CHEMBL2111369 (PROTEIN FAMILY)

Molecules with ChEMBL bioactivity

2 molecules (phase ≥1), by development phase (incl. off-target/promiscuous compounds). Patent mentions across the top 20 by phase: 88,232 (via chembl_molecule»patent_compound — counts attach to the compound, not the gene–compound relationship, so off-target/promiscuous molecules can dominate).

Molecule	Name	Phase	Patents
CHEMBL866	MYCOPHENOLIC ACID	4	87,659
CHEMBL304087	MERIMEPODIB	2	573

PharmGKB: 1 entry (VIP=true, CPIC=false)

PharmGKB clinical annotations

2 annotations.

Variant	Type	Level	Drugs	Phenotypes
rs11706052	Efficacy,Toxicity	4	mycophenolate mofetil;mycophenolic acid	Kidney Transplantation
rs11706052	Efficacy	4	cyclosporine;mycophenolate mofetil	Kidney Transplantation

PharmGKB variants

3 variants.

Variant	Genes	Level	Score	#Clin annots	Drugs
rs470119	IMPDH2, TYMP		0.00	0
rs11706052	DALRD3, IMPDH2, MIR191, MIR425	4	-3.00	2	cyclosporine;mycophenolate mofetil;mycophenolate mofetil;mycophenolic acid
rs121434586	IMPDH2		0.00	0

GtoPdb / IUPHAR curated pharmacology

(IUPHAR/BPS Guide to Pharmacology — expert-curated)

Target class: enzyme — 1.-.-.- Oxidoreductases

Most potent curated ligand interactions (3 total), top 3:

Ligand	Action	Affinity	Parameter
merimepodib	Inhibition	8.0	pIC50
mycophenolic acid	Inhibition	7.7	pIC50
ribavirin	Inhibition	6.0	pIC50

Binding affinities (BindingDB)

39 measured of 90 human assays (90 total across all organisms); most potent 39 below. Values come from heterogeneous assays and are not directly comparable.

Ligand	Measure	Value
({[(2R,3S,4R,5R)-5-(6-amino-2-ethyl-9H-purin-9-yl)-3,4-dihydroxyoxolan-2-yl]methoxy}(hydroxy)phosphoryl)oxyphosphinic acid	KI	1 nM
VX-497 (2)	IC50	3.5 nM
{[(2R,3S,4R,5R)-5-(4-carbamoyl-1,3-thiazol-2-yl)-3,4-dihydroxyoxolan-2-yl]methoxy}[({[(2R,3S,4R,5R)-5-(2,6-diamino-9H-purin-9-yl)-3,4-dihydroxyoxolan-2-yl]methoxy}(hydroxy)phosphoryl)oxy]phosphinic acid	KI	7 nM
N-{2-[3-(dimethylsulfamoyl)phenyl]propan-2-yl}-2-fluoro-9-oxo-9,10-dihydroacridine-3-carboxamide	IC50	7.9 nM
({[(2R,3S,4R,5R)-5-(6-amino-2-ethynyl-9H-purin-9-yl)-3,4-dihydroxyoxolan-2-yl]methoxy}(hydroxy)phosphoryl)oxyphosphinic acid	KI	10 nM
N-{2-[4-(dimethylsulfamoyl)phenyl]propan-2-yl}-2-fluoro-9-oxo-9,10-dihydroacridine-3-carboxamide	IC50	10 nM
2-fluoro-9-oxo-N-(2-phenylpropan-2-yl)-9,10-dihydroacridine-3-carboxamide	IC50	11 nM
2-fluoro-9-oxo-N-[2-(pyridin-3-yl)propan-2-yl]-9,10-dihydroacridine-3-carboxamide	IC50	11 nM
2-fluoro-N-{2-[5-methyl-6-(morpholin-4-yl)pyridin-3-yl]propan-2-yl}-9-oxo-9,10-dihydroacridine-3-carboxamide	IC50	11 nM
2-fluoro-N-[2-(4-methanesulfonylphenyl)propan-2-yl]-9-oxo-9,10-dihydroacridine-3-carboxamide	IC50	12 nM
2-fluoro-N-[2-(3-methanesulfonylphenyl)propan-2-yl]-9-oxo-9,10-dihydroacridine-3-carboxamide	IC50	12 nM
N-{2-[2-(dimethylamino)pyridin-4-yl]propan-2-yl}-2-fluoro-9-oxo-9,10-dihydroacridine-3-carboxamide	IC50	14 nM
[({[(2R,3S,4R,5R)-5-(6-amino-2-ethyl-9H-purin-9-yl)-3,4-dihydroxyoxolan-2-yl]methoxy}(hydroxy)phosphoryl)methyl][2-(4-hydroxy-6-methoxy-7-methyl-3-oxo-1,3-dihydro-2-benzofuran-5-yl)ethoxy]phosphinic acid	KI	16 nM
2-fluoro-9-oxo-N-(2-{2H,3H,4H-pyrido[3,2-b][1,4]oxazin-7-yl}propan-2-yl)-9,10-dihydroacridine-3-carboxamide	IC50	17 nM
Acridone-Based Inhibitor, 4m	IC50	17 nM
({[(2R,3S,4R,5R)-5-(6-amino-2-iodo-9H-purin-9-yl)-3,4-dihydroxyoxolan-2-yl]methoxy}(hydroxy)phosphoryl)oxyphosphinic acid	KI	19 nM
9-oxo-N-(2-phenylpropan-2-yl)-9,10-dihydroacridine-3-carboxamide	IC50	19 nM
({[(2R,3S,4R,5R)-5-(6-amino-2-phenyl-9H-purin-9-yl)-3,4-dihydroxyoxolan-2-yl]methoxy}(hydroxy)phosphoryl)oxyphosphinic acid	KI	20 nM
N-(2-{4-[2-(dimethylamino)ethoxy]phenyl}propan-2-yl)-2-fluoro-9-oxo-9,10-dihydroacridine-3-carboxamide	IC50	20 nM
2-fluoro-N-{2-[6-(morpholin-4-yl)pyridin-3-yl]propan-2-yl}-9-oxo-9,10-dihydroacridine-3-carboxamide	IC50	20 nM
2-fluoro-N-{2-[5-methoxy-6-(morpholin-4-yl)pyridin-3-yl]propan-2-yl}-9-oxo-9,10-dihydroacridine-3-carboxamide	IC50	20 nM
N-{2-[6-(dimethylamino)pyridin-3-yl]propan-2-yl}-2-fluoro-9-oxo-9,10-dihydroacridine-3-carboxamide	IC50	22 nM
2-fluoro-N-[2-(6-methylpyridin-3-yl)propan-2-yl]-9-oxo-9,10-dihydroacridine-3-carboxamide	IC50	23 nM
2-fluoro-9-oxo-N-[2-(pyridin-2-yl)propan-2-yl]-9,10-dihydroacridine-3-carboxamide	IC50	29 nM
9-oxo-N-[2-(pyridin-3-yl)propan-2-yl]-9,10-dihydroacridine-3-carboxamide	IC50	42 nM
({[(2R,3S,4R,5R)-5-[6-amino-2-(phenylamino)-9H-purin-9-yl]-3,4-dihydroxyoxolan-2-yl]methoxy}(hydroxy)phosphoryl)oxyphosphinic acid	KI	45 nM
({[(2R,3S,4R,5R)-5-[6-amino-2-(benzylamino)-9H-purin-9-yl]-3,4-dihydroxyoxolan-2-yl]methoxy}(hydroxy)phosphoryl)oxyphosphinic acid	KI	45 nM
9-oxo-N-[2-(pyridin-2-yl)propan-2-yl]-9,10-dihydroacridine-3-carboxamide	IC50	53 nM
9-oxo-N-[2-(pyridin-4-yl)propan-2-yl]-9,10-dihydroacridine-3-carboxamide	IC50	53 nM
ethyl 9-oxo-9,10-dihydroacridine-3-carboxylate	IC50	59 nM
N-tert-butyl-9-oxo-9,10-dihydroacridine-3-carboxamide	IC50	59 nM
[({[(2R,3S,4R,5R)-5-(6-amino-2-phenyl-9H-purin-9-yl)-3,4-dihydroxyoxolan-2-yl]methoxy}(hydroxy)phosphoryl)methyl][2-(4-hydroxy-6-methoxy-7-methyl-3-oxo-1,3-dihydro-2-benzofuran-5-yl)ethoxy]phosphinic acid	KI	66 nM
({[(2R,3S,4R,5R)-5-{6-amino-2-[(2-phenylethyl)amino]-9H-purin-9-yl}-3,4-dihydroxyoxolan-2-yl]methoxy}(hydroxy)phosphoryl)oxyphosphinic acid	KI	73 nM
9-oxo-N-[(1R)-1-phenylethyl]-9,10-dihydroacridine-3-carboxamide	IC50	95 nM
({[(2R,3S,4R,5R)-5-(6-amino-2-ethenyl-9H-purin-9-yl)-3,4-dihydroxyoxolan-2-yl]methoxy}(hydroxy)phosphoryl)oxyphosphinic acid	KI	96 nM
NSC 358285	KI	110 nM
[({[(2R,3S,4R,5R)-5-(6-amino-9H-purin-9-yl)-3,4-dihydroxyoxolan-2-yl]methoxy}(hydroxy)phosphoryl)methyl][2-(4-hydroxy-6-methoxy-7-methyl-3-oxo-1,3-dihydro-2-benzofuran-5-yl)ethoxy]phosphinic acid	KI	330 nM
9-oxo-N-[(1S)-1-phenylethyl]-9,10-dihydroacridine-3-carboxamide	IC50	431 nM
N-benzyl-9-oxo-9,10-dihydroacridine-3-carboxamide	IC50	2200 nM

ChEMBL bioactivities

676 potent at pChembl≥5 of 729 total, top 50 by pChembl (potency: 10 = 0.1 nM, 6 = 1 µM).

pChembl	Type	Value	Unit	Molecule
8.30	IC50	5	nM	CHEMBL153794
8.30	IC50	5	nM	CHEMBL152910
8.30	IC50	5	nM	CHEMBL150777
8.30	IC50	5	nM	CHEMBL280793
8.22	IC50	6	nM	CHEMBL422508
8.22	IC50	6	nM	CHEMBL153118
8.22	IC50	6	nM	CHEMBL118988
8.22	Ki	6	nM	MYCOPHENOLIC ACID
8.15	Ki	7	nM	MYCOPHENOLIC ACID
8.15	IC50	7	nM	CHEMBL109312
8.15	IC50	7	nM	MERIMEPODIB
8.15	IC50	7	nM	CHEMBL101754
8.15	Ki	7	nM	CHEMBL23843
8.10	IC50	7.9	nM	CHEMBL230256
8.10	IC50	8	nM	CHEMBL23843
8.07	IC50	8.51	nM	CHEMBL131856
8.05	IC50	9	nM	CHEMBL347721
8.00	IC50	10	nM	CHEMBL25138
8.00	IC50	10	nM	CHEMBL426531
8.00	Ki	10	nM	MYCOPHENOLIC ACID
8.00	IC50	10	nM	MERIMEPODIB
7.96	IC50	11	nM	MYCOPHENOLIC ACID
7.96	IC50	11	nM	CHEMBL231660
7.96	IC50	11	nM	CHEMBL230734
7.96	IC50	11	nM	CHEMBL228694
7.96	IC50	11	nM	CHEMBL423952
7.96	IC50	11	nM	CHEMBL112967
7.96	IC50	11	nM	MERIMEPODIB
7.96	IC50	11	nM	CHEMBL68075
7.92	IC50	12	nM	CHEMBL230148
7.92	IC50	12	nM	CHEMBL230091
7.92	IC50	12	nM	CHEMBL155617
7.92	IC50	12	nM	CHEMBL357309
7.92	IC50	12	nM	MYCOPHENOLIC ACID
7.92	IC50	12	nM	CHEMBL64830
7.90	IC50	12.6	nM	CHEMBL132070
7.90	Ki	12.6	nM	CHEMBL3098163
7.89	IC50	13	nM	CHEMBL182139
7.89	IC50	13	nM	CHEMBL208921
7.89	IC50	13	nM	CHEMBL153389
7.89	IC50	13	nM	CHEMBL66614
7.89	IC50	13	nM	CHEMBL67036
7.85	IC50	14	nM	MYCOPHENOLIC ACID
7.85	Ki	14	nM	CHEMBL3098189
7.85	IC50	14	nM	CHEMBL228957
7.85	Ki	14	nM	CHEMBL392203
7.85	IC50	14	nM	CHEMBL357949
7.85	IC50	14	nM	CHEMBL154752
7.85	Ki	14	nM	CHEMBL1683637
7.82	IC50	15	nM	MYCOPHENOLIC ACID

PubChem BioAssay actives

730 with measured affinity, of 1482 total; 50 most potent distinct compounds. Largely complementary to BindingDB; screening values are coarse (µM, 4 dp), so sub-nM hits tie at the floor.

Compound	Assay	Type	Value	Unit
oxolan-3-yl N-[[3-[[3-methoxy-4-(1,3-oxazol-5-yl)phenyl]carbamoylamino]phenyl]methyl]carbamate	1803439: Enzyme Assay from Article 10.3109/14756366.2013.793184: “Design, synthesis and biological evaluation of novel inosine 5’-monophosphate dehydrogenase (IMPDH) inhibitors.”	ic50	0.0035	uM
7-methoxy-2-[4-methyl-3-(oxolan-2-ylmethoxy)phenyl]-6-(1,3-oxazol-5-yl)-1H-quinolin-4-one	92740: Inhibitory activity against inosine monophosphate dehydrogenase IMPDH II	ic50	0.0050	uM
7-methoxy-2-[4-methyl-3-(2-morpholin-4-ylethoxy)phenyl]-6-(1,3-oxazol-5-yl)-1H-quinolin-4-one	92740: Inhibitory activity against inosine monophosphate dehydrogenase IMPDH II	ic50	0.0050	uM
7-methoxy-2-(4-methoxyphenyl)-6-(1,3-oxazol-5-yl)-1H-quinolin-4-one	92624: Inhibition of human inosine monophosphate dehydrogenase IMPDH II	ic50	0.0050	uM
7-methoxy-2-(3-methylphenyl)-6-(1,3-oxazol-5-yl)-1H-quinolin-4-one	92624: Inhibition of human inosine monophosphate dehydrogenase IMPDH II	ic50	0.0050	uM
2-(3,4-dimethylphenyl)-7-methoxy-6-(1,3-oxazol-5-yl)-1H-quinolin-4-one	92624: Inhibition of human inosine monophosphate dehydrogenase IMPDH II	ic50	0.0050	uM
2-(4-bromophenyl)-7-methoxy-6-(1,3-oxazol-5-yl)-1H-quinolin-4-one	92624: Inhibition of human inosine monophosphate dehydrogenase IMPDH II	ic50	0.0050	uM
7-methoxy-2-(4-methyl-3-morpholin-4-ylphenyl)-6-(1,3-oxazol-5-yl)-1H-quinolin-4-one	92740: Inhibitory activity against inosine monophosphate dehydrogenase IMPDH II	ic50	0.0050	uM
2-[3-(dimethylamino)-2,3-dihydro-1H-inden-5-yl]-7-methoxy-6-(1,3-oxazol-5-yl)-1H-quinolin-4-one	92740: Inhibitory activity against inosine monophosphate dehydrogenase IMPDH II	ic50	0.0050	uM
N-isoquinolin-6-yl-5-phenyl-1,3-oxazol-2-amine	3134: Inhibitory activity against inosine 5’-inosine monophosphate dehydrogenase type II (IMPDH II)	ic50	0.0050	uM
7-methoxy-2-(4-methylphenyl)-6-(1,3-oxazol-5-yl)-1H-quinolin-4-one	92624: Inhibition of human inosine monophosphate dehydrogenase IMPDH II	ic50	0.0050	uM
2-[3-(azetidin-1-yl)-2,3-dihydro-1H-inden-5-yl]-7-methoxy-6-(1,3-oxazol-5-yl)-1H-quinolin-4-one	92740: Inhibitory activity against inosine monophosphate dehydrogenase IMPDH II	ic50	0.0060	uM
[(2R)-1-cyanobutan-2-yl] N-[[3-[(4-cyano-3-methoxyphenyl)carbamoylamino]phenyl]methyl]carbamate	92742: Inhibitory activity tested against inosine-5’-monophosphate dehydrogenase 2 (IMPDH-II) enzyme	ic50	0.0060	uM
Mycophenolic Acid	92748: Inhibitory activity against human Inosine-5’-monophosphate dehydrogenase 2 (IMPDH type II isoform); Range is 6-10 nM	ki	0.0060	uM
[2-[N-methyl-2-[2-[[3-(1,3-oxazol-5-yl)-1H-indol-6-yl]amino]-1,3-oxazol-5-yl]anilino]-2-oxoethyl] acetate	92742: Inhibitory activity tested against inosine-5’-monophosphate dehydrogenase 2 (IMPDH-II) enzyme	ic50	0.0070	uM
[(3S)-oxolan-3-yl] N-[[3-[[3-methoxy-4-(1,3-oxazol-5-yl)phenyl]carbamoylamino]phenyl]methyl]carbamate	93053: Inhibition of inositol-5-monophosphate dehydrogenase (IMPDH); range 7-8 nM	ic50	0.0070	uM
(2S)-2-[(2E)-2-[2-(4-amino-6-methoxy-7-methyl-3-oxo-1H-2-benzofuran-5-yl)ethylidene]cyclopentyl]propanoic acid	93053: Inhibition of inositol-5-monophosphate dehydrogenase (IMPDH); range 7-8 nM	ic50	0.0070	uM
7-methoxy-6-(1,3-oxazol-5-yl)-2-phenyl-1H-quinolin-4-one	92750: Inhibitory activity against IMPDH II with respect to IMP and NAD	ki	0.0070	uM
N-[2-[3-(dimethylsulfamoyl)phenyl]propan-2-yl]-2-fluoro-9-oxo-10H-acridine-3-carboxamide	1797842: IMPDH2 Enzyme Inhibition and Human T-Lymphoblast (CEM) Proliferation Inhibition Assays from Article 10.1021/jm070299x: “Acridone-based inhibitors of inosine 5’-monophosphate dehydrogenase: discovery and SAR leading to the identification of N-(2-(6-(4-ethylpiperazin-1-yl)pyridin-3-yl)propan-2-yl)-2- fluoro-9-oxo-9,10-dihydroacridine-3-carboxamide (BMS-566419).”	ic50	0.0079	uM
(E)-6-(6-ethenyl-4-hydroxy-7-methyl-3-oxo-1H-2-benzofuran-5-yl)-4-methylhex-4-enoic acid	92618: Inhibition of human recombinant inosine monophosphate dehydrogenase type II.	ic50	0.0085	uM
7-methoxy-6-(1,3-oxazol-5-yl)-2-thiophen-3-yl-1H-quinolin-4-one	92624: Inhibition of human inosine monophosphate dehydrogenase IMPDH II	ic50	0.0090	uM
N-[2-[4-(dimethylsulfamoyl)phenyl]propan-2-yl]-2-fluoro-9-oxo-10H-acridine-3-carboxamide	1797842: IMPDH2 Enzyme Inhibition and Human T-Lymphoblast (CEM) Proliferation Inhibition Assays from Article 10.1021/jm070299x: “Acridone-based inhibitors of inosine 5’-monophosphate dehydrogenase: discovery and SAR leading to the identification of N-(2-(6-(4-ethylpiperazin-1-yl)pyridin-3-yl)propan-2-yl)-2- fluoro-9-oxo-9,10-dihydroacridine-3-carboxamide (BMS-566419).”	ic50	0.0100	uM
7-methoxy-6-(1,3-oxazol-5-yl)-2-(8-oxo-6,7-dihydro-5H-naphthalen-2-yl)-1H-quinolin-4-one	92740: Inhibitory activity against inosine monophosphate dehydrogenase IMPDH II	ic50	0.0100	uM
N’-tert-butyl-N-[3-methoxy-4-(1,3-oxazol-5-yl)phenyl]oxamide	92613: Inhibition of inosine-5’-monophosphate dehydrogenase 2.	ic50	0.0100	uM
[6-[7-methoxy-6-(1,3-oxazol-5-yl)-4-oxo-1H-quinolin-2-yl]-2,3-dihydro-1H-inden-1-yl] N,N-dimethylcarbamate	92740: Inhibitory activity against inosine monophosphate dehydrogenase IMPDH II	ic50	0.0110	uM
2-fluoro-9-oxo-N-(2-phenylpropan-2-yl)-10H-acridine-3-carboxamide	1797842: IMPDH2 Enzyme Inhibition and Human T-Lymphoblast (CEM) Proliferation Inhibition Assays from Article 10.1021/jm070299x: “Acridone-based inhibitors of inosine 5’-monophosphate dehydrogenase: discovery and SAR leading to the identification of N-(2-(6-(4-ethylpiperazin-1-yl)pyridin-3-yl)propan-2-yl)-2- fluoro-9-oxo-9,10-dihydroacridine-3-carboxamide (BMS-566419).”	ic50	0.0110	uM
2-fluoro-9-oxo-N-(2-pyridin-3-ylpropan-2-yl)-10H-acridine-3-carboxamide	1797842: IMPDH2 Enzyme Inhibition and Human T-Lymphoblast (CEM) Proliferation Inhibition Assays from Article 10.1021/jm070299x: “Acridone-based inhibitors of inosine 5’-monophosphate dehydrogenase: discovery and SAR leading to the identification of N-(2-(6-(4-ethylpiperazin-1-yl)pyridin-3-yl)propan-2-yl)-2- fluoro-9-oxo-9,10-dihydroacridine-3-carboxamide (BMS-566419).”	ic50	0.0110	uM
2-fluoro-N-[2-(5-methyl-6-morpholin-4-yl-3-pyridinyl)propan-2-yl]-9-oxo-10H-acridine-3-carboxamide	1797842: IMPDH2 Enzyme Inhibition and Human T-Lymphoblast (CEM) Proliferation Inhibition Assays from Article 10.1021/jm070299x: “Acridone-based inhibitors of inosine 5’-monophosphate dehydrogenase: discovery and SAR leading to the identification of N-(2-(6-(4-ethylpiperazin-1-yl)pyridin-3-yl)propan-2-yl)-2- fluoro-9-oxo-9,10-dihydroacridine-3-carboxamide (BMS-566419).”	ic50	0.0110	uM
1-(4-methylphenyl)-3-[3-(1,3-oxazol-5-yl)-1H-indol-6-yl]urea	92742: Inhibitory activity tested against inosine-5’-monophosphate dehydrogenase 2 (IMPDH-II) enzyme	ic50	0.0110	uM
N-[2-[2-[3-methoxy-4-(1,3-oxazol-5-yl)anilino]-1,3-oxazol-5-yl]phenyl]-N-methyl-2-pyrazol-1-ylacetamide	92622: Inhibition of human inosine-5’-monophosphate dehydrogenase 2	ic50	0.0110	uM
7-methoxy-2-[3-(methylamino)-2,3-dihydro-1H-inden-5-yl]-6-(1,3-oxazol-5-yl)-1H-quinolin-4-one	92740: Inhibitory activity against inosine monophosphate dehydrogenase IMPDH II	ic50	0.0120	uM
2-methoxy-N-[6-[7-methoxy-6-(1,3-oxazol-5-yl)-4-oxo-1H-quinolin-2-yl]-2,3-dihydro-1H-inden-1-yl]-N-methylacetamide	92740: Inhibitory activity against inosine monophosphate dehydrogenase IMPDH II	ic50	0.0120	uM
2-fluoro-N-[2-(3-methylsulfonylphenyl)propan-2-yl]-9-oxo-10H-acridine-3-carboxamide	1797842: IMPDH2 Enzyme Inhibition and Human T-Lymphoblast (CEM) Proliferation Inhibition Assays from Article 10.1021/jm070299x: “Acridone-based inhibitors of inosine 5’-monophosphate dehydrogenase: discovery and SAR leading to the identification of N-(2-(6-(4-ethylpiperazin-1-yl)pyridin-3-yl)propan-2-yl)-2- fluoro-9-oxo-9,10-dihydroacridine-3-carboxamide (BMS-566419).”	ic50	0.0120	uM
2-fluoro-N-[2-(4-methylsulfonylphenyl)propan-2-yl]-9-oxo-10H-acridine-3-carboxamide	1797842: IMPDH2 Enzyme Inhibition and Human T-Lymphoblast (CEM) Proliferation Inhibition Assays from Article 10.1021/jm070299x: “Acridone-based inhibitors of inosine 5’-monophosphate dehydrogenase: discovery and SAR leading to the identification of N-(2-(6-(4-ethylpiperazin-1-yl)pyridin-3-yl)propan-2-yl)-2- fluoro-9-oxo-9,10-dihydroacridine-3-carboxamide (BMS-566419).”	ic50	0.0120	uM
N-[2-[2-[3-methoxy-4-(1,3-oxazol-5-yl)anilino]-1,3-oxazol-5-yl]phenyl]-N-methyl-2-morpholin-4-ylacetamide	92622: Inhibition of human inosine-5’-monophosphate dehydrogenase 2	ic50	0.0120	uM
(E)-6-(6-ethyl-4-hydroxy-7-methyl-3-oxo-1H-2-benzofuran-5-yl)-4-methylhex-4-enoic acid	92618: Inhibition of human recombinant inosine monophosphate dehydrogenase type II.	ic50	0.0126	uM
(E)-N-[(2R)-1-[[(2R,3S,4R,5R)-5-(6-aminopurin-9-yl)-3,4-dihydroxyoxolan-2-yl]methylamino]-1-oxo-3-phenylpropan-2-yl]-6-(4-hydroxy-6-methoxy-7-methyl-3-oxo-1H-2-benzofuran-5-yl)-4-methylhex-4-enamide	1060471: Inhibition of IMPDH2 (unknown origin)	ki	0.0126	uM
7-methoxy-6-(1,3-oxazol-5-yl)-2-(3-pyrrolidin-1-yl-2,3-dihydro-1H-inden-5-yl)-1H-quinolin-4-one	92740: Inhibitory activity against inosine monophosphate dehydrogenase IMPDH II	ic50	0.0130	uM
7-methoxy-6-(1,3-oxazol-5-yl)-3-(2-pyridin-4-ylethyl)-2-sulfanylidene-1H-quinazolin-4-one	241614: In vitro inhibitory concentration against inosine-5’-monophosphate dehydrogenase	ic50	0.0130	uM
2-[[(E)-4-(6-ethyl-4-hydroxy-7-methyl-3-oxo-1H-2-benzofuran-5-yl)-2-methylbut-2-enyl]amino]ethylphosphonic acid	267726: Inhibition of human IMPDH2	ic50	0.0130	uM
N-[2-[2-[3-methoxy-4-(1,3-oxazol-5-yl)anilino]-1,3-oxazol-5-yl]phenyl]-N-methyl-2-(1,2,4-triazol-1-yl)acetamide	92622: Inhibition of human inosine-5’-monophosphate dehydrogenase 2	ic50	0.0130	uM
N-[2-[2-[3-methoxy-4-(1,3-oxazol-5-yl)anilino]-1,3-oxazol-5-yl]phenyl]-N-methyl-3-morpholin-4-ylpropanamide	92622: Inhibition of human inosine-5’-monophosphate dehydrogenase 2	ic50	0.0130	uM
2-(4-acetyl-2,3-dihydro-1,4-benzoxazin-6-yl)-7-methoxy-6-(1,3-oxazol-5-yl)-1H-quinolin-4-one	92740: Inhibitory activity against inosine monophosphate dehydrogenase IMPDH II	ic50	0.0140	uM
N-[2-[2-(dimethylamino)-4-pyridinyl]propan-2-yl]-2-fluoro-9-oxo-10H-acridine-3-carboxamide	1797842: IMPDH2 Enzyme Inhibition and Human T-Lymphoblast (CEM) Proliferation Inhibition Assays from Article 10.1021/jm070299x: “Acridone-based inhibitors of inosine 5’-monophosphate dehydrogenase: discovery and SAR leading to the identification of N-(2-(6-(4-ethylpiperazin-1-yl)pyridin-3-yl)propan-2-yl)-2- fluoro-9-oxo-9,10-dihydroacridine-3-carboxamide (BMS-566419).”	ic50	0.0140	uM
[[(2R,3S,4R,5R)-5-(6-amino-2-ethylpurin-9-yl)-3,4-dihydroxyoxolan-2-yl]methoxy-hydroxyphosphoryl] [(2R,3S,4R,5R)-5-(4-carbamoyl-1,3-thiazol-2-yl)-3,4-dihydroxyoxolan-2-yl]methyl hydrogen phosphate	1797841: IMPDH Type 2 Enzyme Assay from Article 10.1021/jm070568j: “Probing Binding Requirements of Type I and Type II Isoforms of Inosine Monophosphate Dehydrogenase with Adenine-Modified Nicotinamide Adenine Dinucleotide Analogues.”	ki	0.0140	uM
[[(2S,3R,4S,5S)-5-(6-amino-2-ethylpurin-9-yl)-3,4-dihydroxyoxolan-2-yl]methoxy-hydroxyphosphoryl] [(2R,3S,4R,5R)-5-(4-carbamoyl-1,3-thiazol-2-yl)-3,4-dihydroxyoxolan-2-yl]methyl hydrogen phosphate	580126: Inhibition of human IMPDH2 by Spectrophotometer	ki	0.0140	uM
[(2R,3S,4R,5R)-5-(6-amino-2-pyridin-4-ylpurin-9-yl)-3,4-dihydroxyoxolan-2-yl]methoxy-[[hydroxy-[2-(4-hydroxy-6-methoxy-7-methyl-3-oxo-1H-2-benzofuran-5-yl)ethoxy]phosphoryl]methyl]phosphinic acid	1060471: Inhibition of IMPDH2 (unknown origin)	ki	0.0140	uM
7-methoxy-2-(2-methyl-1,3-dihydroisoindol-5-yl)-6-(1,3-oxazol-5-yl)-1H-quinolin-4-one	92740: Inhibitory activity against inosine monophosphate dehydrogenase IMPDH II	ic50	0.0140	uM
2-[(2R,3R)-3-(dimethylamino)-2-methoxy-2,3-dihydro-1H-inden-5-yl]-7-methoxy-6-(1,3-oxazol-5-yl)-1H-quinolin-4-one	92740: Inhibitory activity against inosine monophosphate dehydrogenase IMPDH II	ic50	0.0150	uM
2-[8-(dimethylamino)-5,6,7,8-tetrahydronaphthalen-2-yl]-7-methoxy-6-(1,3-oxazol-5-yl)-1H-quinolin-4-one	92740: Inhibitory activity against inosine monophosphate dehydrogenase IMPDH II	ic50	0.0150	uM

CTD chemical–gene interactions

78 total (human), top 30 by PubMed support.

Chemical	Actions (top 5)	PubMed papers
sodium arsenite	increases abundance, increases expression, affects binding, increases reaction, decreases expression	4
4-(5-benzo(1,3)dioxol-5-yl-4-pyridin-2-yl-1H-imidazol-2-yl)benzamide	affects cotreatment, decreases expression	2
bisphenol S	increases expression	2
Phenylmercuric Acetate	affects cotreatment, decreases expression	2
Tretinoin	increases expression, decreases expression	2
Valproic Acid	decreases expression, increases methylation	2
Genistein	increases expression	2
Particulate Matter	decreases expression, increases abundance	2
aristolochic acid I	increases expression	1
FR900359	affects phosphorylation	1
bisphenol F	increases expression	1
TAK-243	increases sumoylation	1
2,4,6-tribromophenol	increases expression	1
methylmercuric chloride	decreases expression	1
triphenyl phosphate	affects expression	1
bisphenol A	increases expression	1
chlorophyllin	decreases expression	1
sodium arsenate	decreases expression	1
decabromobiphenyl ether	decreases expression	1
oridonin	decreases activity	1
beta-lapachone	decreases expression	1
o,p’-DDT	increases expression	1
tetrabromobisphenol A	increases expression	1
tanshinone	decreases activity	1
perfluorooctanoic acid	increases expression	1
ochratoxin A	increases expression	1
periodate-oxidized adenosine	affects expression	1
cupric chloride	decreases expression	1
microcystin RR	increases expression	1
chloropicrin	increases expression	1

ChEMBL screening assays

111 unique, capped per target: 104 binding, 6 functional, 1 admet

Representative assays (with source publication via chembl_document):

Assay ID	Type	Description	Source paper
CHEMBL1032990	Binding	Inhibition of human recombinant IMPDH2 expressed in Escherichia coli guaB assessed as NADH production by fluorescence assay	Triazole inhibitors of Cryptosporidium parvum inosine 5’-monophosphate dehydrogenase. — J Med Chem
CHEMBL4676180	ADMET	Inhibition of human IMPDH2 expressed in Escherichia coli assessed as apparent inhibition constant using NAD as substrate by fluorescence assay	Mycophenolic anilides as broad specificity inosine-5’-monophosphate dehydrogenase (IMPDH) inhibitors. — Bioorg Med Chem Lett
CHEMBL699978	Functional	Compound was tested for its effect on IMPDH (Inosine 5`-monophosphate dehydrogenase) activity in K562 cells	Synthesis, structure, and antiproliferative activity of selenophenfurin, an inosine 5’-monophosphate dehydrogenase inhibitor analogue of selenazofurin. — J Med Chem

Cellosaurus cell lines

4 cell lines: 4 cancer cell line

First 10 cell lines (id-ordered, not curated):

Cellosaurus	Name	Category	Sex
CVCL_C8FK	HAP1 IMPDH2 (-) 1	Cancer cell line	Male
CVCL_C8FL	HAP1 IMPDH2 (-) 2	Cancer cell line	Male
CVCL_F1PU	HyCyte HeLa KO-hIMPDH2	Cancer cell line	Female
CVCL_F1SB	HyCyte NCI-H460 KO-hIMPDH2	Cancer cell line	Male

Clinical trials (associated diseases)

371 trials via MONDO — disease-level, not drug-specific.

Trial	Phase	Status	Title
NCT04586348	PHASE4	UNKNOWN	Prenatal Iodine Supplementation and Early Childhood Neurodevelopment
NCT04873115	PHASE4	UNKNOWN	Double-blind, Placebo-controlled, Randomized Clinical Trial Comparing the Efficacy and Safety of Sialanar Plus orAl rehabiLitation Against Placebo Plus Oral Rehabilitation for chIldren and Adolescents With seVere Sialorrhoea and Neurodisabilties,
NCT00142259	PHASE4	UNKNOWN	Efficacy and Safety of DBS of the GPi in Patients With Primary Generalized and Segmental Dystonia
NCT00950196	PHASE4	COMPLETED	Amantadine for Improving Neurologic Symptoms in Ataxia-Telangiectasia
NCT00998660	PHASE4	COMPLETED	RECHARGE Sub-Study to the Implantable Systems Performance Registry (ISPR)
NCT02263417	PHASE4	COMPLETED	A Randomized Controlled Trail Comparing Subthalamic and Pallidal Deep Brain Stimulation for Dystonia
NCT02559102	PHASE3	COMPLETED	Dexmedetomidine Sedation Versus General Anaesthesia for Inguinal Hernia Surgery in Infants
NCT02757079	PHASE3	COMPLETED	Study of the Efficacy and Safety of NPC-15 for Sleep Disorders of Children With Neurodevelopmental Disorders
NCT06915480	PHASE3	RECRUITING	Reducing Missed Appointments
NCT07377032	PHASE3	RECRUITING	TAP-GRIN: Interventional Study on Patients With GRIN-related Neurodevelopmental Disorders
NCT00169403	PHASE3	UNKNOWN	Pallidal Stimulation in Patients With Idiopathic Generalised Dystonia
NCT03232320	PHASE3	COMPLETED	Meditoxin® Treatment in Patients With Cervical Dystonia
NCT02909959	PHASE2	COMPLETED	Sulforaphane for the Treatment of Young Men With Autism Spectrum Disorder
NCT06081348	PHASE2	RECRUITING	Sertraline vs. Placebo in the Treatment of Anxiety in Children and AdoLescents With NeurodevelopMental Disorders
NCT06352372	PHASE2	COMPLETED	Safety and Efficacy of tPBM for Epileptiform Activity in Autism
NCT00001784	PHASE2	COMPLETED	Mexiletine for the Treatment of Focal Dystonia
NCT00105430	PHASE2	COMPLETED	Deep Brain Stimulation for Cervical Dystonia
NCT00106782	PHASE2	COMPLETED	Transcranial Electrical Polarization to Treat Focal Hand Dystonia
NCT00122044	PHASE2	COMPLETED	Childhood Hypertonia of Central Origin: A Trial of Anticholinergic Treatment Effects
NCT00169338	PHASE2	COMPLETED	Pallidal Stimulation in Patients With Post-anoxic and Idiopathic Dystonia
NCT00331669	PHASE2	UNKNOWN	Efficacy and Safety of Deep Brain Stimulation (DBS) of the Pallidal (GPi) in Patients With Tardive Dystonia
NCT02107261	PHASE2	COMPLETED	Incobotulinum Toxin A (Xeomin®) As A Treatment For Focal Task-Specific Dystonia Of The Musician’s Hand
NCT02470325	PHASE2	UNKNOWN	The Effects of Cannabis on Dystonia and Spasticity on Pediatric Patients
NCT05027997	PHASE2	COMPLETED	Exploratory Study of Dipraglurant (ADX48621) for the Treatment of Patients With Blepharospasm
NCT06412653	PHASE2	COMPLETED	Prospective Pilot Trial to Address Feasibility and Safety of Oral Zinc in GNAO1 Associated Disorders
NCT07304089	PHASE2	RECRUITING	A Study to Evaluate the Efficacy, Safety, and Tolerability of VIM0423 in Individuals With Isolated Dystonia
NCT00503191	PHASE1	COMPLETED	NeuroModulation Technique Treatment of Autism
NCT04475848	PHASE1	COMPLETED	A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Food Effect of RO6953958 in Healthy Participants
NCT06300398	PHASE1	COMPLETED	IAMA-6 Oral Dose Study in Healthy Adults
NCT01433757	PHASE1	COMPLETED	Ampicillin for DYT-1 Dystonia Motor Symptoms
NCT01698450	PHASE1	COMPLETED	Magnetic Resonance (MR) Guided Functional Ultrasound-Neurosurgery for Movement Disorders
NCT02982304	PHASE1	UNKNOWN	Multi-Target Pallidal and Thalamic Deep Brain Stimulation for Hemi-Dystonia
NCT06117020	PHASE1	COMPLETED	Single and Multiple Ascending Dose Study of MTR-601 in Healthy Individuals
NCT06554288	PHASE1	RECRUITING	Pharmacogenomic Contributions to Trihexyphenidyl Biotransformation and Response in Children With Dystonic Cerebral Palsy
NCT01783041	PHASE2/PHASE3	COMPLETED	Effect of Early L-Carnitine Supplementation on Neurodevelopmental Outcomes in Very Preterm Infants
NCT05767385	PHASE2/PHASE3	RECRUITING	Fetal Cerebrovascular Autoregulation in Congenital Heart Disease and Association With Neonatal Neurobehavior
NCT05675098	EARLY_PHASE1	NOT_YET_RECRUITING	Central Nervous System Stimulants and Physical Function in Children With Cerebral Palsy
NCT00783783	Not specified	COMPLETED	CYP2D6 Pharmacogenetics in Risperidone-Treated Children
NCT01778504	Not specified	RECRUITING	Studying Childhood-onset Behavioral, Psychiatric, and Developmental Disorders
NCT01850784	Not specified	UNKNOWN	High Energy Formula Feeding in Infants With Congenital Heart Disease