ABCG2

Summary

ABCG2 (ATP binding cassette subfamily G member 2 (JR blood group), HGNC:74) is a protein-coding gene on chromosome 4q22.1, encoding Broad substrate specificity ATP-binding cassette transporter ABCG2 (Q9UNQ0). Broad substrate specificity ATP-dependent transporter of the ATP-binding cassette (ABC) family that actively extrudes a wide variety of physiological compounds, dietary toxins and xenobiotics from cells.

The membrane-associated protein encoded by this gene is included in the superfamily of ATP-binding cassette (ABC) transporters. ABC proteins transport various molecules across extra- and intra-cellular membranes. ABC genes are divided into seven distinct subfamilies (ABC1, MDR/TAP, MRP, ALD, OABP, GCN20, White). This protein is a member of the White subfamily. Alternatively referred to as a breast cancer resistance protein, this protein functions as a xenobiotic transporter which may play a major role in multi-drug resistance. It likely serves as a cellular defense mechanism in response to mitoxantrone and anthracycline exposure. Significant expression of this protein has been observed in the placenta, which may suggest a potential role for this molecule in placenta tissue. Multiple transcript variants encoding different isoforms have been found for this gene.

Source: NCBI Gene 9429 — RefSeq curated summary.

At a glance

• GWAS associations: 96
• Clinical variants (ClinVar): 97 total — 14 pathogenic, 1 likely-pathogenic
• Phenotypes (HPO): 5
• Druggable target: yes — 92 molecules with ChEMBL bioactivity
• MANE Select transcript: NM_004827

Identifiers

Gene identifiers

Field	Value
HGNC ID	HGNC:74
Approved symbol	ABCG2
Name	ATP binding cassette subfamily G member 2 (JR blood group)
Location	4q22.1
Locus type	gene with protein product
Status	Approved
Aliases	EST157481, MXR, BCRP, ABCP, CD338
Ensembl gene	ENSG00000118777
Ensembl biotype	protein_coding
OMIM	603756
Entrez	9429

Gene structure

Transcript identifiers

Ensembl transcripts: 31 — 31 protein_coding

ENST00000237612, ENST00000503830, ENST00000505480, ENST00000515655, ENST00000650821, ENST00000889078, ENST00000889079, ENST00000889080, ENST00000889081, ENST00000889082, ENST00000889083, ENST00000889084, ENST00000889085, ENST00000889086, ENST00000889087, ENST00000889088, ENST00000889089, ENST00000889090, ENST00000889091, ENST00000889092, ENST00000889093, ENST00000889094, ENST00000889095, ENST00000889096, ENST00000889097, ENST00000889098, ENST00000889099, ENST00000889100, ENST00000962213, ENST00000962214, ENST00000962215

RefSeq mRNA: 7 — MANE Select: NM_004827 NM_001257386, NM_001348985, NM_001348986, NM_001348987, NM_001348988, NM_001348989, NM_004827

CCDS: CCDS3628, CCDS58910

Canonical transcript exons

ENST00000237612 — 16 exons

Exon	Start	End
ENSE00000801180	88095520	88095609
ENSE00000801181	88097453	88097607
ENSE00000801182	88099324	88099448
ENSE00000801183	88101230	88101319
ENSE00000801184	88107184	88107266
ENSE00000801185	88113303	88113553
ENSE00000801186	88114957	88115058
ENSE00000801187	88118109	88118260
ENSE00000801188	88121635	88121792
ENSE00000801189	88131061	88131213
ENSE00000801190	88131803	88131917
ENSE00000801191	88132576	88132635
ENSE00001196220	88090269	88092381
ENSE00001342969	88158386	88158639
ENSE00003465456	88139793	88140014
ENSE00003501294	88094577	88094659

Expression profiles

Bgee: expression breadth ubiquitous, 245 present calls, max score 98.97.

FANTOM5 (CAGE): breadth broad, TPM avg 4.4675 / max 251.9052, expressed in 755 samples.

FANTOM5 promoters (16 alternative TSS)

Promoter ID	TPM avg	Samples expressed
53072	1.3827	451
53070	1.1039	397
53066	0.4975	171
53069	0.3850	139
53076	0.3049	124
53067	0.2214	126
53071	0.1631	70
53075	0.1283	47
53068	0.1024	57
53078	0.0481	15

Top tissues by expression

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

Tissue	Anatomy ID	Expression score	Quality
jejunal mucosa	UBERON:0000399	98.97	gold quality
ileal mucosa	UBERON:0000331	98.67	gold quality
endothelial cell	CL:0000115	95.45	gold quality
mucosa of transverse colon	UBERON:0004991	94.43	gold quality
seminal vesicle	UBERON:0000998	94.20	gold quality
duodenum	UBERON:0002114	93.87	gold quality
substantia nigra	UBERON:0002038	87.82	gold quality
medial globus pallidus	UBERON:0002477	87.80	gold quality
colonic mucosa	UBERON:0000317	87.50	gold quality
placenta	UBERON:0001987	87.32	gold quality
midbrain	UBERON:0001891	87.31	gold quality
C1 segment of cervical spinal cord	UBERON:0006469	87.00	gold quality
inferior vagus X ganglion	UBERON:0005363	86.89	gold quality
subthalamic nucleus	UBERON:0001906	86.88	gold quality
spinal cord	UBERON:0002240	85.98	gold quality
globus pallidus	UBERON:0001875	85.81	gold quality
dorsal motor nucleus of vagus nerve	UBERON:0002870	85.53	gold quality
postcentral gyrus	UBERON:0002581	85.52	gold quality
putamen	UBERON:0001874	85.06	gold quality
rectum	UBERON:0001052	85.03	gold quality
caudate nucleus	UBERON:0001873	85.00	gold quality
prefrontal cortex	UBERON:0000451	84.87	gold quality
lateral nuclear group of thalamus	UBERON:0002736	84.87	gold quality
Ammon’s horn	UBERON:0001954	84.66	gold quality
hypothalamus	UBERON:0001898	84.63	gold quality
mucosa of sigmoid colon	UBERON:0004993	84.53	gold quality
medulla oblongata	UBERON:0001896	83.88	gold quality
parietal lobe	UBERON:0001872	83.83	gold quality
amygdala	UBERON:0001876	83.69	gold quality
superior vestibular nucleus	UBERON:0007227	83.32	gold quality

Single-cell (SCXA)

Detected in 8 experiment(s), a significant marker in 7.

Experiment	Marker?	Max mean expression
E-MTAB-6819	yes	387.44
E-MTAB-10018	yes	209.80
E-MTAB-6701	yes	45.98
E-GEOD-135922	yes	45.47
E-MTAB-9388	yes	7.38
E-GEOD-137537	yes	6.21
E-MTAB-2983	no	495.00
E-ANND-3	no	0.00

Regulation

Is transcription factor: no

Upstream regulators (CollecTRI, top): AHR, E2F1, ESR1, ESR2, FOXC1, GLI1, HIF1A, KLF5, MSX2, NFE2L2, NFKB1, NFKB, NR1I2, NR3C1, PGR, PPARA, PPARG, RARA, RELA, SALL4, SMAD2, SMARCA4, SP1, SP3, TP53

miRNA regulators (miRDB)

104 targeting ABCG2, top 30 by miRDB confidence (max_score; target_count = how many genes the miRNA targets in total — lower means more specific):

miRNA	Max score	Avg score	miRNA target_count
HSA-MIR-6873-3P	100.00	71.42	2626
HSA-MIR-6833-3P	100.00	70.63	3197
HSA-MIR-3163	100.00	77.23	8605
HSA-MIR-4768-5P	100.00	69.49	2861
HSA-MIR-5011-5P	100.00	83.46	5820
HSA-MIR-4747-5P	100.00	67.90	2681
HSA-MIR-5196-5P	100.00	67.98	2761
HSA-MIR-196A-1-3P	99.99	72.15	2772
HSA-MIR-186-5P	99.99	70.83	3707
HSA-MIR-12136	99.98	72.81	5713
HSA-MIR-5696	99.98	72.36	4487
HSA-MIR-548N	99.98	71.94	4170
HSA-MIR-4482-3P	99.98	72.50	3147
HSA-MIR-512-3P	99.97	67.35	1049
HSA-MIR-302E	99.96	70.74	2669
HSA-MIR-570-3P	99.96	72.41	4910
HSA-MIR-548AT-5P	99.96	70.83	2666
HSA-MIR-3143	99.93	71.96	3104
HSA-MIR-218-5P	99.93	72.22	2103
HSA-MIR-3119	99.92	71.34	2390
HSA-MIR-3529-3P	99.90	73.55	3045
HSA-MIR-302A-3P	99.89	71.23	1777
HSA-MIR-302B-3P	99.89	71.23	1777
HSA-MIR-302C-3P	99.89	71.20	1778
HSA-MIR-302D-3P	99.89	71.25	1777
HSA-MIR-7845-5P	99.88	64.88	771
HSA-MIR-12119	99.87	68.35	1653
HSA-MIR-579-3P	99.86	71.66	3628
HSA-MIR-520E-3P	99.84	70.55	1698
HSA-MIR-373-3P	99.84	70.68	1668

Literature-anchored findings (GeneRIF, showing 40)

• role in transport of 7-ethyl-10-hydroxycamptothecin (SN-38) in human lung cancer cells (PMID:11688982)
• ABCG2 protein effluxes hoechst 33342 in hematopoietic stem cells (PMID:11801536)
• homodimer formation is essential for BCRP drug resistance (PMID:11807788)
• There is no indication that elevated BCRP expression in breast carcinomas confers resistance to anthracyclines. (PMID:11948115)
• Increased expression in relapsed or refractory acute myeloid leukemia compared to level at diagnosis. (PMID:11986944)
• Expression of the BCRP gene reduces the response to chemotherapy in AML and that BCRP expression is higher at the time of relapse. (PMID:12145683)
• Overexpression of wild-type breast cancer resistance protein mediates methotrexate resistance. (PMID:12208758)
• involvement of ABCG2 in multidrug resistance in cancer, especially with regard to acute myeloid leukemia (PMID:12369998)
• characterization of different isoforms without possible endogenous dimerization partners (PMID:12374800)
• ABCG2 expression conferred resistance to mitoxantrone & topotecan, but not to idarubicin. High levels of ABCG2 mRNA expression in adult AML blast specimens are relatively uncommon. ABCG2 expression may be limited to a small cell subpopulation. (PMID:12393637)
• The breast cancer resistance protein protects against pheophorbide a, a major chlorophyll-derived dietary phototoxin, and protoporphyria. (PMID:12429862)
• In both normal and tumour brain tissue, BCRP is located at the blood-brain barrier, mainly at the luminal surface of microvessel endothelium. It may pose an additional barrier to drug access to the brain. (PMID:12438926)
• results showed that ABCG2 confers resistance to indolocarbazoles by transporting them in an energy-dependent manner (PMID:12459192)
• polymorphisms in BCRP1 result in low amounts of BCRP, which results in hypersensitivity of normal cells to such anticancer drugs as irinotecan and mitoxantrone (PMID:12479221)
• RNA expression of this protein in breast cancer correlates with response to chemotherapy. (PMID:12576456)
• Function of the ABC transporters, P-gp, multidrug resistance protein and BCRP, in minimal residual disease in acute myeloid leukemia (PMID:12604403)
• BCRP may play a role in the transport of sterols in human, in addition to its ability to transport multiple drugs and toxins. (PMID:12668685)
• transports sulfated conjugates of steroids and xenobiotics (PMID:12682043)
• functional study on polymorphism and determination of critical role of arginine-482 in methotrexate transport (PMID:12741957)
• Wld-type as well as R482T BCRP mediates cellular efflux of 9-AC but not 9-NC. Zplar groups at the 9 or 10 position of the CPT A ring facilitate interaction with BCRP. (PMID:12810652)
• ABCG2 is a component of the energy-dependent efflux system for certain folates and antifolates. (PMID:12874005)
• ABCG2 is expressed and functions as a transporter in the brain. (PMID:12958161)
• Single amino acid substitutions in the transmembrane domains of breast cancer resistance protein (BCRP) alter cross resistance patterns in transfectants (PMID:14566825)
• REVIEW: multidrug resistance in breast cancer (PMID:14576842)
• Higher ABCG2 expression is associated with T-lineage acute lymphoblastic leukemia (PMID:14613996)
• human ABCG2 likely exists and functions as a homotetramer (PMID:15001581)
• BCRP may serve as a molecular target for reducing drug resistance to chemotherapy in advanced lung cancer patients. (PMID:15014021)
• VX-710 modulates Pgp, MRP-1, and BCRP(R482), and has potential as a clinical broad-spectrum multidrug resistance modulator in malignancies. (PMID:15014037)
• ABCG2 overexpression is associated with drug resistance to SN38 in colon cancer cells and in irinotecan-treated metastases (PMID:15027118)
• Bcrp/ABCG2 enhances hypoxic cell survival through interactions with heme (PMID:15044468)
• subcellular localization of ABCG2 in gallbladder adenocarcinoma (PMID:15146167)
• As a method of circumventing ABCG2-associated drug resistance, low-polarity camptothecin analogues are considered to be potent lead compounds. (PMID:15170677)
• BCRP mRNA levels, antibody staining and function correlated strongly in cell lines but not in acute myeloid leukemia samples, suggesting complex biology of BCRP in AML and incomplete modeling by cell lines (PMID:15208643)
• imatinib is a substrate for BCRP; it competes with mitoxantrone for drug export (PMID:15251980)
• GXXXG motif promotes proper packing of the transmembrane segments in the functional ABCG2 homodimer, although it does not solely arbitrate dimerization (PMID:15260487)
• Results suggest that 1) the predominant allelic expression pattern of BCRP in placental samples is biallelic, and 2) the mutation C421A is not a genetic variant acting in cis, but is considered to influence translation efficiency. (PMID:15475413)
• conformational changes of the ABCG2 multidrug transporter modify its interaction with a monoclonal antibody on the cell surface (PMID:15557326)
• Cyclosporin A is neither a substrate nor an inhibitor of the human ABCG2 transporter. (PMID:15598974)
• The breast cancer resistance protein (BCRP) can be expressed in AML and BCRP mRNA expression was determined in samples from 40 AML patients by real-time RT-PCR. (PMID:15607361)
• The ABCG2, a member of the ATP binding cassette (ABC) transporters, has been identified as a molecular determinant for bone marrow stem cells and proposed as a universal marker for stem cells. (PMID:15625123)

Cross-species orthologs

12 orthologs

Organism	Symbol	Gene ID
danio_rerio	abcg2b	ENSDARG00000079361
mus_musculus	Abcg3	ENSMUSG00000029299
rattus_norvegicus	Abcg3l4	ENSRNOG00000064381
rattus_norvegicus	Abcg3l4	ENSRNOG00000065003
rattus_norvegicus	Abcg3l1	ENSRNOG00000069481
rattus_norvegicus	Abcg3	ENSRNOG00000070774
drosophila_melanogaster	CG31689	FBGN0031449
drosophila_melanogaster	CG31121	FBGN0051121
drosophila_melanogaster	CG32091	FBGN0052091
caenorhabditis_elegans		WBGENE00006522
caenorhabditis_elegans		WBGENE00015479
caenorhabditis_elegans		WBGENE00017179

Paralogs (4): ABCG5 (ENSG00000138075), ABCG8 (ENSG00000143921), ABCG1 (ENSG00000160179), ABCG4 (ENSG00000172350)

Protein

Protein identifiers

Broad substrate specificity ATP-binding cassette transporter ABCG2 — Q9UNQ0 (reviewed: Q9UNQ0)

Alternative names: ATP-binding cassette sub-family G member 2, Breast cancer resistance protein, CDw338, Mitoxantrone resistance-associated protein, Placenta-specific ATP-binding cassette transporter, Urate exporter

All UniProt accessions (2): Q9UNQ0, F8S0F2

UniProt curated annotations — full annotation on UniProt →

Function. Broad substrate specificity ATP-dependent transporter of the ATP-binding cassette (ABC) family that actively extrudes a wide variety of physiological compounds, dietary toxins and xenobiotics from cells. Involved in porphyrin homeostasis, mediating the export of protoporphyrin IX (PPIX) from both mitochondria to cytosol and cytosol to extracellular space, it also functions in the cellular export of heme. Also mediates the efflux of sphingosine-1-P from cells. Acts as a urate exporter functioning in both renal and extrarenal urate excretion. In kidney, it also functions as a physiological exporter of the uremic toxin indoxyl sulfate. Also involved in the excretion of steroids like estrone 3-sulfate/E1S, 3beta-sulfooxy-androst-5-en-17-one/DHEAS, and other sulfate conjugates. Mediates the secretion of the vitamins riboflavin and biotin into milk. Involved in the excretion of the riboflavin-derived compound lumichrome into the intestinal lumen and in its secretion into milk. Extrudes pheophorbide a, a phototoxic porphyrin catabolite of chlorophyll, reducing its bioavailability. Plays an important role in the exclusion of xenobiotics from the brain. It confers to cells a resistance to multiple drugs and other xenobiotics including mitoxantrone, pheophorbide, camptothecin, methotrexate, azidothymidine, and the anthracyclines daunorubicin and doxorubicin, through the control of their efflux. In placenta, it limits the penetration of drugs from the maternal plasma into the fetus. May play a role in early stem cell self-renewal by blocking differentiation. In inflammatory macrophages, exports itaconate from the cytosol to the extracellular compartment and limits the activation of TFEB-dependent lysosome biogenesis involved in antibacterial innate immune response.

Subunit / interactions. Homodimer; disulfide-linked. The minimal functional unit is a homodimer, but the major oligomeric form in plasma membrane is a homotetramer with possibility of higher order oligomerization up to homododecamers.

Subcellular location. Cell membrane. Apical cell membrane. Mitochondrion membrane.

Tissue specificity. Highly expressed in placenta. Low expression in small intestine, liver and colon. Expressed in brain (at protein level).

Post-translational modifications. N-glycosylated. Glycosylation-deficient ABCG2 is normally expressed and functional. Phosphorylated. Phosphorylation at Thr-362 by PIM1 is induced by drugs like mitoxantrone and is associated with cells increased drug resistance. It regulates the localization to the plasma membrane, the homooligomerization and therefore, the activity of the transporter.

Activity regulation. Specifically inhibited by the fungal toxin fumitremorgin C and Ko143.

Domain organisation. The extracellular loop 3 (ECL3) is involved in binding porphyrins and transfer them to other carriers, probably albumin.

Polymorphism. Genetic variations in ABCG2 define the blood group Junior system (JR) [MIM:614490]. Individuals with Jr(a-) blood group lack the Jr(a) antigen on their red blood cells. These individuals may have anti-Jr(a) antibodies in their serum, which can cause transfusion reactions or hemolytic disease of the fetus or newborn. Although the clinical significance of the Jr(a-) blood group has been controversial, severe fatal hemolytic disease of the newborn has been reported. The Jr(a-) phenotype has a higher frequency in individuals of Asian descent, compared to those of European descent. The Jr(a-) phenotype is inherited as an autosomal recessive trait. Genetic variations in ABCG2 influence the variance in serum uric acid concentrations and define the serum uric acid concentration quantitative trait locus 1 (UAQTL1) [MIM:138900]. Excess serum accumulation of uric acid can lead to the development of gout, a common disorder characterized by tissue deposition of monosodium urate crystals as a consequence of hyperuricemia.

Similarity. Belongs to the ABC transporter superfamily. ABCG family. Eye pigment precursor importer (TC 3.A.1.204) subfamily.

Isoforms (2)

UniProt ID	Names	Canonical?
Q9UNQ0-1	1	yes
Q9UNQ0-2	2

RefSeq proteins (7): NP_001244315, NP_001335914, NP_001335915, NP_001335916, NP_001335917, NP_001335918, NP_004818* (*=MANE)

Domains & families (InterPro)

ID	Name	Type
IPR003439	ABC_transporter-like_ATP-bd	Domain
IPR003593	AAA+_ATPase	Domain
IPR013525	ABC2_TM	Domain
IPR027417	P-loop_NTPase	Homologous_superfamily
IPR043926	ABCG_dom	Domain
IPR050352	ABCG_transporters	Family

Pfam: PF00005, PF01061, PF19055

Enzyme classification (BRENDA):

• EC 7.6.2.2 — ABC-type xenobiotic transporter (BRENDA: 49 organisms, 716 substrates, 471 inhibitors, 280 Km, 31 kcat entries)
• EC 7.6.2.3 — ABC-type glutathione-S-conjugate transporter (BRENDA: 8 organisms, 145 substrates, 63 inhibitors, 16 Km, 0 kcat entries)

Substrate kinetics (BRENDA)

75 substrates with measured Km, best-characterized 15. Km ranges are aggregated across organisms/conditions.

Substrate	Km (mM)	Measurements
ATP	0.0009–9	105
VERAPAMIL/IN	0.0006–0.0058	11
VINBLASTINE/IN	0.0002–0.1455	11
ESTRADIOL 17-BETA-D-GLUCURONIDE/IN	0.013–0.17	7
LEUKOTRIENE C4/IN	—	7
METHOTREXATE/IN	0.24–0.776	6
NICARDIPINE/IN	0.0004–0.0026	6
PROGESTERONE/IN	0.0038–0.0207	6
CYCLIC GUANOSINE MONOPHOSPHATE/IN	0.36–2	5
VERAPAMIL	0.0022–0.0115	5
ATP	0.0865–0.91	5
COLCHICINE/IN	0.037–0.72	4
FOLIC ACID/IN	0.13–0.26	4
PACLITAXEL/IN	0.0007–0.0009	4
RHODAMINE 123/IN	0.0118–0.0354	4

Catalyzed reactions (Rhea), 12 shown:

• urate(in) + ATP + H2O = urate(out) + ADP + phosphate + H(+) (RHEA:16461)
• sphing-4-enine 1-phosphate(in) + ATP + H2O = sphing-4-enine 1-phosphate(out) + ADP + phosphate + H(+) (RHEA:38951)
• 17beta-estradiol 17-O-(beta-D-glucuronate)(in) + ATP + H2O = 17beta-estradiol 17-O-(beta-D-glucuronate)(out) + ADP + phosphate + H(+) (RHEA:60128)
• indoxyl sulfate(in) + ATP + H2O = indoxyl sulfate(out) + ADP + phosphate + H(+) (RHEA:61332)
• estrone 3-sulfate(in) + ATP + H2O = estrone 3-sulfate(out) + ADP + phosphate + H(+) (RHEA:61348)
• riboflavin(in) + ATP + H2O = riboflavin(out) + ADP + phosphate + H(+) (RHEA:61352)
• methotrexate(in) + ATP + H2O = methotrexate(out) + ADP + phosphate + H(+) (RHEA:61356)
• pheophorbide a(in) + ATP + H2O = pheophorbide a(out) + ADP + phosphate + H(+) (RHEA:61360)
• dehydroepiandrosterone 3-sulfate(in) + ATP + H2O = dehydroepiandrosterone 3-sulfate(out) + ADP + phosphate + H(+) (RHEA:61364)
• 4-methylumbelliferone sulfate(in) + ATP + H2O = 4-methylumbelliferone sulfate(out) + ADP + phosphate + H(+) (RHEA:61368)
• 4-methylumbelliferone beta-D-glucuronate(in) + ATP + H2O = 4-methylumbelliferone beta-D-glucuronate(out) + ADP + phosphate + H(+) (RHEA:61372)
• 5,7-dimethyl-2-methylamino-4-(3-pyridylmethyl)-1,3-benzothiazol-6-yl sulfate(in) + ATP + H2O = 5,7-dimethyl-2-methylamino-4-(3-pyridylmethyl)-1,3-benzothiazol-6-yl sulfate(out) + ADP + phosphate + H(+) (RHEA:61376)

UniProt features (139 total): helix 31, sequence variant 26, mutagenesis site 25, strand 17, topological domain 7, sequence conflict 7, transmembrane region 6, turn 5, binding site 4, domain 2, site 2, disulfide bond 2, splice variant 2, chain 1, modified residue 1, glycosylation site 1

Structure

Experimental structures (PDB)

29 structures.

PDB	Method	Resolution (Å)
8QCM	ELECTRON MICROSCOPY	2.39
8U2C	ELECTRON MICROSCOPY	2.5
8Q7B	ELECTRON MICROSCOPY	2.56
8PXO	ELECTRON MICROSCOPY	3
8PY4	ELECTRON MICROSCOPY	3
8P7W	ELECTRON MICROSCOPY	3.04
6HBU	ELECTRON MICROSCOPY	3.09
6HZM	ELECTRON MICROSCOPY	3.09
6ETI	ELECTRON MICROSCOPY	3.1
7OJH	ELECTRON MICROSCOPY	3.1
8BHT	ELECTRON MICROSCOPY	3.1
7NEQ	ELECTRON MICROSCOPY	3.12
8BI0	ELECTRON MICROSCOPY	3.2
8P8A	ELECTRON MICROSCOPY	3.2
7NEZ	ELECTRON MICROSCOPY	3.39
7OJ8	ELECTRON MICROSCOPY	3.4
7OJI	ELECTRON MICROSCOPY	3.4
8P8J	ELECTRON MICROSCOPY	3.49
6VXF	ELECTRON MICROSCOPY	3.5
7NFD	ELECTRON MICROSCOPY	3.51
6FFC	ELECTRON MICROSCOPY	3.56
6HIJ	ELECTRON MICROSCOPY	3.56
6HCO	ELECTRON MICROSCOPY	3.58
6FEQ	ELECTRON MICROSCOPY	3.6
6VXI	ELECTRON MICROSCOPY	3.7
5NJ3	ELECTRON MICROSCOPY	3.78
5NJG	ELECTRON MICROSCOPY	3.78
6VXH	ELECTRON MICROSCOPY	4
6VXJ	ELECTRON MICROSCOPY	4.1

Predicted structure (AlphaFold)

Model	pLDDT	Fraction very-high
AF-Q9UNQ0-F1	85.54	0.66

Antibody-complex structures (SAbDab): 16 — 5NJ3, 5NJG, 6ETI, 6FEQ, 6HCO, 7NEQ, 7NEZ, 7NFD, 8P7W, 8P8A, 8P8J, 8PXO, 8PY4, 8Q7B, 8QCM, 8U2C

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): 418 (not glycosylated); 557 (not glycosylated)

Ligand- & substrate-binding residues (4): 80–87; 184–190; 211; 243

Post-translational modifications (1): 362

Disulfide bonds (2): 592–608, 603

Glycosylation sites (1): 596

Mutagenesis-validated functional residues (25):

Position	Phenotype
71	decreased protein abundance. no effect on substrate transmembrane transport.
86	decreased protein abundance. decreased localization to the plasma membrane and retained intracellularly. loss of atpase-
211	decreased estrone-3 sulfate atpase-coupled transmembrane transporter activity. decreased substrate-induced atp hydrolysi
362	loss of phosphorylation by pim1. decreased localization to the plasma membrane. decreased homooligomerization. loss of f
362	loss of phosphorylation by pim1. constitutive drug resistance independent of pim1.
383	loss of protein expression.
418	no effect.
435	no effect on stability. increased estrone-3 sulfate atpase-coupled transmembrane transporter activity. increased substra
435	no effect on stability. decreased estrone-3 sulfate atpase-coupled transmembrane transporter activity. decreased substra
436	no effect on stability. decreased estrone-3 sulfate atpase-coupled transmembrane transporter activity. decreased substra
439	no effect on stability. decreased estrone-3 sulfate atpase-coupled transmembrane transporter activity. decreased substra
482	decreases atpase activity.
482	increases atpase activity.
482	no change in atpase activity.
482	decreases transport activity.
546	no effect on stability. no effect on estrone-3 sulfate atpase-coupled transmembrane transporter activity. no effect on s
546	no effect on stability. decreased estrone-3 sulfate atpase-coupled transmembrane transporter activity. increased basal a
549	no effect on stability. no effect on estrone-3 sulfate atpase-coupled transmembrane transporter activity. no effect on s
554	no effect on stability. increased estrone-3 sulfate atpase-coupled transmembrane transporter activity. increased basal a
555	loss of protein expression.
557	no effect.
583	strongly reduced binding to hemin but not to ppix.
596	loss of glycosylation.
603	strongly reduced binding to hemin but not to ppix.
605	no effect on hemin binding.

Function

Pathways and Gene Ontology

Reactome pathways

23 pathways

ID	Pathway
R-HSA-1660661	Sphingolipid de novo biosynthesis
R-HSA-189451	Heme biosynthesis
R-HSA-189483	Heme degradation
R-HSA-2161517	Abacavir transmembrane transport
R-HSA-917937	Iron uptake and transport
R-HSA-9725554	Differentiation of Keratinocytes in Interfollicular Epidermis in Mammalian Skin
R-HSA-9753281	Paracetamol ADME
R-HSA-9793528	Ciprofloxacin ADME
R-HSA-9818032	NFE2L2 regulating MDR associated enzymes
R-HSA-1266738	Developmental Biology
R-HSA-1430728	Metabolism
R-HSA-189445	Metabolism of porphyrins
R-HSA-2161522	Abacavir ADME
R-HSA-2262752	Cellular responses to stress
R-HSA-382551	Transport of small molecules
R-HSA-428157	Sphingolipid metabolism
R-HSA-556833	Metabolism of lipids
R-HSA-8953897	Cellular responses to stimuli
R-HSA-9711123	Cellular response to chemical stress
R-HSA-9734767	Developmental Cell Lineages
R-HSA-9748784	Drug ADME
R-HSA-9755511	KEAP1-NFE2L2 pathway
R-HSA-9759194	Nuclear events mediated by NFE2L2

MSigDB gene sets: 315 (showing top): GSE18804_SPLEEN_MACROPHAGE_VS_TUMORAL_MACROPHAGE_UP, GOBP_EXCRETION, GOBP_CIRCULATORY_SYSTEM_PROCESS, PAL_PRMT5_TARGETS_UP, REACTOME_METABOLISM_OF_PORPHYRINS, VICENT_METASTASIS_UP, GOCC_CELL_SURFACE, GOBP_ORGANIC_ACID_TRANSPORT, KEGG_ABC_TRANSPORTERS, BOYAULT_LIVER_CANCER_SUBCLASS_G12_DN, OSWALD_HEMATOPOIETIC_STEM_CELL_IN_COLLAGEN_GEL_UP, GOBP_CELLULAR_RESPONSE_TO_TOXIC_SUBSTANCE, GOBP_SPHINGOLIPID_METABOLIC_PROCESS, BILD_E2F3_ONCOGENIC_SIGNATURE, ENGELMANN_CANCER_PROGENITORS_UP

GO Biological Process (14): obsolete organic anion transport (GO:0015711), biotin transport (GO:0015878), sphingolipid biosynthetic process (GO:0030148), riboflavin transport (GO:0032218), urate metabolic process (GO:0046415), transmembrane transport (GO:0055085), transepithelial transport (GO:0070633), renal urate salt excretion (GO:0097744), export across plasma membrane (GO:0140115), transport across blood-brain barrier (GO:0150104), cellular detoxification (GO:1990748), xenobiotic transport across blood-brain barrier (GO:1990962), lipid transport (GO:0006869), urate transport (GO:0015747)

GO Molecular Function (17): ATP binding (GO:0005524), obsolete organic anion transmembrane transporter activity (GO:0008514), ABC-type xenobiotic transporter activity (GO:0008559), urate transmembrane transporter activity (GO:0015143), biotin transmembrane transporter activity (GO:0015225), efflux transmembrane transporter activity (GO:0015562), ATP hydrolysis activity (GO:0016887), riboflavin transmembrane transporter activity (GO:0032217), ATPase-coupled transmembrane transporter activity (GO:0042626), identical protein binding (GO:0042802), protein homodimerization activity (GO:0042803), xenobiotic transmembrane transporter activity (GO:0042910), sphingolipid intramembrane carrier activity (GO:0046624), nucleotide binding (GO:0000166), protein binding (GO:0005515), protein dimerization activity (GO:0046983), ABC-type transporter activity (GO:0140359)

GO Cellular Component (9): nucleoplasm (GO:0005654), plasma membrane (GO:0005886), apical plasma membrane (GO:0016324), brush border membrane (GO:0031526), mitochondrial membrane (GO:0031966), membrane raft (GO:0045121), external side of apical plasma membrane (GO:0098591), mitochondrion (GO:0005739), membrane (GO:0016020)

Reactome top-level categories

Rollup of top-12 pathways:

Category	Pathways
Drug ADME	3
Metabolism of porphyrins	2
Metabolism	2
Sphingolipid metabolism	1
Abacavir ADME	1
Transport of small molecules	1
Developmental Cell Lineages of the Integumentary System	1
Nuclear events mediated by NFE2L2	1
Cellular responses to stimuli	1
Metabolism of lipids	1
Cellular responses to stress	1
Developmental Biology	1

GO top-level categories

Rollup of top GO terms by namespace:

Category	Terms
nitrogen compound transport	3
transport	3
vitamin transport	2
cellular process	2
xenobiotic transport	2
vitamin transmembrane transporter activity	2
transmembrane transporter activity	2
ATP-dependent activity	2
protein binding	2
cellular anatomical structure	2
apical plasma membrane	2
monocarboxylic acid transport	1
sulfur compound transport	1
sphingolipid metabolic process	1
lipid biosynthetic process	1
small molecule metabolic process	1
purine-containing compound metabolic process	1
renal tubular secretion	1
transmembrane transport	1
export from cell	1
vascular transport	1
cellular response to toxic substance	1
detoxification	1
transport across blood-brain barrier	1
lipid localization	1
adenyl ribonucleotide binding	1
purine ribonucleoside triphosphate binding	1
xenobiotic transmembrane transporter activity	1
ABC-type transporter activity	1
urate transport	1
salt transmembrane transporter activity	1
monocarboxylic acid transmembrane transporter activity	1
biotin transport	1
sulfur compound transmembrane transporter activity	1
ribonucleoside triphosphate phosphatase activity	1
riboflavin transport	1
primary active transmembrane transporter activity	1
ATP hydrolysis activity	1
identical protein binding	1
protein dimerization activity	1

Protein interactions and networks

STRING

3633 interactions, top by confidence (×1000):

Protein A	Protein B	Partner UniProt	Score
ABCG2	ABCC1	P33527	969
ABCG2	ABCC2	Q92887	889
ABCG2	SLCO1B1	Q9Y6L6	886
ABCG2	SLC2A9	Q9NRM0	874
ABCG2	SLC22A12	Q96S37	861
ABCG2	BSG	P35613	839
ABCG2	MRPS7	Q9Y2R9	813
ABCG2	ALB	P02768	806
ABCG2	SLC17A1	Q14916	803
ABCG2	SLC22A1	O15245	798
ABCG2	SLC22A8	Q8TCC7	796
ABCG2	SLCO1B3	Q9NPD5	790
ABCG2	PROM1	O43490	783
ABCG2	CYP3A4	P05184	778
ABCG2	SLCO2B1	O94956	767

IntAct

29 interactions, top by confidence:

A	B	Type	Score
ABCG2	ABCG2	psi-mi:“MI:0915”(physical association)	0.790
ABCG2	ABCG2	psi-mi:“MI:0407”(direct interaction)	0.790
ABCG2	PIM1	psi-mi:“MI:0915”(physical association)	0.640
PIM1	ABCG2	psi-mi:“MI:0915”(physical association)	0.640
PIM1	ABCG2	psi-mi:“MI:0403”(colocalization)	0.640
ABCG2	ENPP1	psi-mi:“MI:0407”(direct interaction)	0.600
ENPP1	ABCG2	psi-mi:“MI:0915”(physical association)	0.600
ABCG2	ENPP1	psi-mi:“MI:0915”(physical association)	0.600
Trim69		psi-mi:“MI:0915”(physical association)	0.400
GPC1	SNAP23	psi-mi:“MI:0915”(physical association)	0.400
GPC1	GANAB	psi-mi:“MI:0915”(physical association)	0.400
ABCG2	UBC	psi-mi:“MI:0915”(physical association)	0.400
UBC	ABCG2	psi-mi:“MI:0915”(physical association)	0.400
Npc1	ESYT2	psi-mi:“MI:0914”(association)	0.350
ARRDC5	PLPP1	psi-mi:“MI:0914”(association)	0.350
ABCG2	SCAMP1	psi-mi:“MI:0914”(association)	0.350
RHCG	TMEM223	psi-mi:“MI:0914”(association)	0.350
SLC34A2	SNAP23	psi-mi:“MI:0914”(association)	0.350
SLC6A7	ABCB1	psi-mi:“MI:0914”(association)	0.350
SLC7A2	SCAMP3	psi-mi:“MI:0914”(association)	0.350
SLC7A4	ESYT2	psi-mi:“MI:0914”(association)	0.350

BioGRID (81): ABCG2 (Affinity Capture-MS), ABCG2 (Affinity Capture-MS), ABCG2 (Affinity Capture-MS), ABCG2 (Affinity Capture-MS), ABCG2 (Affinity Capture-MS), ABCG2 (Affinity Capture-MS), ABCG2 (Affinity Capture-MS), ABCG2 (Affinity Capture-MS), ABCG2 (Affinity Capture-MS), ABCG2 (Affinity Capture-MS), ABCG2 (Affinity Capture-MS), ABCG2 (Affinity Capture-MS), ABCG2 (Affinity Capture-MS), ABCG2 (Affinity Capture-MS), ABCG2 (Affinity Capture-MS)

ESM2 similar proteins: A2AKQ0, A2VE55, A5GFZ5, C9WPN6, F1QGW6, F6RQL9, O60762, O70152, O77676, P00516, P0C605, P20461, P32189, P35250, P37273, P41091, P53033, P81795, Q05B83, Q0IID9, Q13126, Q13976, Q14409, Q15B89, Q1JQ93, Q2KHU8, Q2KJ61, Q2TBV5, Q2VIR3, Q3MHF7, Q5HZM6, Q5MB13, Q5R797, Q5RDC9, Q5RIC0, Q5ZHS1, Q5ZMS3, Q63060, Q641W4, Q64516

Diamond homologs: A0A059J0G5, A0A0M3R8G1, A0A0M4FLW6, A0A1U8QKX8, A2WSH0, A9YWR6, B8ALI0, B9G300, B9G5Y5, C7J6G6, D3GE74, D3ZCM3, F2PLH2, F2RSQ6, F2SHL1, H9BZ66, O24367, O80946, O81016, P10090, P25371, P45843, P45844, P58428, Q05360, Q08234, Q0JLC5, Q16928, Q17320, Q27256, Q2QV81, Q4GZT4, Q4WFQ4, Q54CG0, Q55DA0, Q55DR1, Q55DW4, Q55GB1, Q5MB13, Q64343

SIGNOR signaling

4 interactions.

A	Effect	B	Mechanism
PPARG	“up-regulates quantity by expression”	ABCG2	“transcriptional regulation”
SALL4	“up-regulates quantity by expression”	ABCG2	“transcriptional regulation”
SMARCA4	“up-regulates quantity by expression”	ABCG2	“transcriptional regulation”
PIM1	“up-regulates activity”	ABCG2	phosphorylation

Disease & clinical

Cancer significance

Clinical variants and AI predictions

ClinVar

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

Classification	Count (floor)
Pathogenic	14
Likely pathogenic	1
Uncertain significance	46
Likely benign	5
Benign	8

Top pathogenic / likely-pathogenic (15)

Variant ID	HGVS	Classification
1341211	GRCh37/hg19 4q21.21-22.1(chr4:80467886-93362064)x1	Pathogenic
145649	GRCh38/hg38 4q21.21-22.3(chr4:80879777-94809447)x1	Pathogenic
148194	GRCh38/hg38 4q21.21-24(chr4:80427023-100855441)x1	Pathogenic
1527097	GRCh37/hg19 4q13.3-22.1(chr4:75737340-91131156)	Pathogenic
1527100	GRCh37/hg19 4q21.21-22.2(chr4:79780152-94873225)	Pathogenic
1527103	GRCh37/hg19 4q21.21-22.1(chr4:81054789-90667421)	Pathogenic
1527108	GRCh37/hg19 4q21.23-22.1(chr4:85839771-93071150)	Pathogenic
152923	GRCh38/hg38 4q21.23-22.2(chr4:85449365-93973194)x1	Pathogenic
2498966	GRCh37/hg19 4q22.1(chr4:88344058-89061168)x1	Pathogenic
2579270	GRCh38/hg38 4q21.21-23(chr4:79123548-99457773)x1	Pathogenic
3062764	GRCh37/hg19 4q21.21-22.3(chr4:81558759-95965995)x1	Pathogenic
394976	GRCh37/hg19 4q21.21-22.1(chr4:82283358-90341831)x1	Pathogenic
562937	GRCh37/hg19 4q21.21-22.3(chr4:81314915-96636651)x1	Pathogenic
59455	GRCh38/hg38 4q21.21-22.1(chr4:79575748-92412449)x1	Pathogenic
3062760	GRCh37/hg19 4q21.23-22.3(chr4:85139670-96295033)x3	Likely pathogenic

SpliceAI

2331 predictions. Top by Δscore:

Variant	Effect	Δscore
4:88094575:A:AC	donor_gain	1.0000
4:88094576:C:CC	donor_gain	1.0000
4:88094576:CG:C	donor_gain	1.0000
4:88094576:CGTTG:C	donor_gain	1.0000
4:88094658:GCCTA:G	acceptor_loss	1.0000
4:88094659:CCTA:C	acceptor_loss	1.0000
4:88094660:C:CC	acceptor_gain	1.0000
4:88094660:CT:C	acceptor_loss	1.0000
4:88094661:T:G	acceptor_loss	1.0000
4:88095605:AAAAT:A	acceptor_gain	1.0000
4:88095606:AAAT:A	acceptor_gain	1.0000
4:88095607:AAT:A	acceptor_gain	1.0000
4:88095608:AT:A	acceptor_gain	1.0000
4:88095610:C:CA	acceptor_loss	1.0000
4:88097615:A:T	acceptor_gain	1.0000
4:88107283:A:AC	acceptor_gain	1.0000
4:88107283:A:C	acceptor_gain	1.0000
4:88113353:TGGAA:T	donor_gain	1.0000
4:88113363:T:C	donor_gain	1.0000
4:88113556:T:TC	acceptor_gain	1.0000
4:88113563:C:CT	acceptor_gain	1.0000
4:88113564:A:T	acceptor_gain	1.0000
4:88113565:G:GC	acceptor_gain	1.0000
4:88113569:A:AC	acceptor_gain	1.0000
4:88113569:A:C	acceptor_gain	1.0000
4:88114952:TATAC:T	donor_loss	1.0000
4:88114954:TA:T	donor_loss	1.0000
4:88114955:A:T	donor_loss	1.0000
4:88114956:C:CG	donor_loss	1.0000
4:88131795:ATACT:A	donor_loss	1.0000

AlphaMissense

4276 scored. Top likely-pathogenic:

Variant	Protein change	am_pathogenicity
4:88118223:G:C	H243D	1.000
4:88118216:G:T	P245H	0.999
4:88121663:C:G	A221P	0.999
4:88121673:G:C	D217E	0.999
4:88121673:G:T	D217E	0.999
4:88121674:T:A	D217V	0.999
4:88121674:T:C	D217G	0.999
4:88121675:C:G	D217H	0.999
4:88121689:G:T	P212H	0.999
4:88121692:T:A	E211V	0.999
4:88132582:T:A	K86I	0.999
4:88095541:G:C	S572R	0.998
4:88095541:G:T	S572R	0.998
4:88095543:T:G	S572R	0.998
4:88121674:T:G	D217A	0.998
4:88121675:C:T	D217N	0.998
4:88121677:A:G	L216S	0.998
4:88121689:G:C	P212R	0.998
4:88121691:C:A	E211D	0.998
4:88121691:C:G	E211D	0.998
4:88121692:T:C	E211G	0.998
4:88121692:T:G	E211A	0.998
4:88121693:C:T	E211K	0.998
4:88121695:T:A	D210V	0.998
4:88121748:T:A	K192N	0.998
4:88121748:T:G	K192N	0.998
4:88131083:A:G	L170P	0.998
4:88131803:T:A	Q126H	0.998
4:88131803:T:G	Q126H	0.998
4:88132583:T:G	K86Q	0.998

dbSNP variants (sampled 300 via entrez): RS1000002258 (4:88094347 G>A), RS1000039382 (4:88207764 C>T), RS1000089015 (4:88222936 G>A,C), RS1000106772 (4:88161991 T>A), RS1000117689 (4:88092723 C>T), RS1000121656 (4:88223327 C>T), RS1000176118 (4:88204945 T>A), RS10001782 (4:88136188 T>C,G), RS1000204267 (4:88156050 C>G), RS1000205232 (4:88126323 T>A,C), RS1000239974 (4:88168209 C>A), RS1000255944 (4:88112437 T>A), RS1000261345 (4:88173566 C>A,T), RS1000266205 (4:88216810 A>G), RS1000275640 (4:88112151 T>G)

Disease associations

OMIM: gene MIM:603756 | disease phenotypes: MIM:613509

GenCC curated gene-disease

Mondo (3): chromosome 4q21 deletion syndrome (MONDO:0013292), autosomal dominant polycystic kidney disease (MONDO:0004691), hereditary breast ovarian cancer syndrome (MONDO:0003582)

Orphanet (3): 4q21 microdeletion syndrome (Orphanet:238750), Autosomal dominant polycystic kidney disease (Orphanet:730), Hereditary breast and/or ovarian cancer syndrome (Orphanet:145)

HPO phenotypes

5 total (5 of 5 shown, HPO-id order):

HPO	Term
HP:0000006	Autosomal dominant inheritance
HP:0000951	Abnormality of the skin
HP:0001369	Arthritis
HP:0002149	Hyperuricemia
HP:0033073	Urate tophus

GWAS associations

96 associations (top):

Study	Trait	p-value
GCST000242_3	Urate levels	3.000000e-60
GCST000418_5	Uric acid levels	1.000000e-10
GCST000418_9	Uric acid levels	2.000000e-18
GCST000581_5	Urate levels	1.000000e-13
GCST000818_3	Urate levels	3.000000e-23
GCST000818_5	Urate levels	1.000000e-75
GCST001247_2	Cardiovascular disease risk factors	2.000000e-17
GCST001268_3	Gout	3.000000e-12
GCST001269_4	Serum uric acid levels	2.000000e-20
GCST001374_6	Uric acid levels	5.000000e-06
GCST001408_3	Response to statins (LDL cholesterol change)	2.000000e-15
GCST001608_3	Renal function-related traits (urate)	4.000000e-30
GCST001713_23	Dental caries	7.000000e-08
GCST001726_3	Lipoprotein-associated phospholipase A2 activity change in response to statin therapy	2.000000e-10
GCST001790_4	Gout	2.000000e-32
GCST001791_5	Urate levels	1.000000e-134
GCST002355_1	Serum uric acid levels	3.000000e-42
GCST002355_2	Serum uric acid levels	3.000000e-18
GCST002355_3	Serum uric acid levels	4.000000e-20
GCST002650_2	Coffee consumption (cups per day)	9.000000e-08
GCST002773_1	Gout	7.000000e-54
GCST002828_1	Urate levels in obese individuals	9.000000e-08
GCST002828_32	Urate levels in obese individuals	1.000000e-07
GCST002829_31	Urate levels in overweight individuals	2.000000e-30
GCST002829_39	Urate levels in overweight individuals	2.000000e-11
GCST002829_41	Urate levels in overweight individuals	1.000000e-22
GCST002830_10	Urate levels in lean individuals	1.000000e-22
GCST002830_25	Urate levels in lean individuals	2.000000e-29
GCST002830_7	Urate levels in lean individuals	6.000000e-13
GCST002989_5	LDL peak particle diameter (total fat intake interaction)	9.000000e-06

EFO canonical traits (13, from GWAS)

EFO ID	Trait name
EFO:0004531	urate measurement
EFO:0004761	uric acid measurement
EFO:0007804	LDL cholesterol change measurement
EFO:0004746	lipoprotein-associated phospholipase A(2) measurement
EFO:0004330	coffee consumption
EFO:0006782	cups of coffee per day measurement
EFO:0007677	LDL peak particle diameter measurement
EFO:0007678	total fat intake measurement
EFO:0009104	hyperuricemia
EFO:0010089	bitter beverage consumption measurement
EFO:0006781	coffee consumption measurement
EFO:0010091	tea consumption measurement
EFO:0004346	neuroimaging measurement

MeSH disease descriptors (2)

Descriptor	Name	Tree numbers
D061325	Hereditary Breast and Ovarian Cancer Syndrome	C04.588.180.483; C04.588.322.455.431; C04.700.517; C12.050.351.500.056.630.705.431; C12.050.351.937.418.685.431; C12.100.250.056.630.705.431; C12.900.418.685.431; C16.320.700.517; C17.800.090.500.483; C19.344.410.431; C19.391.630.705.431
D016891	Polycystic Kidney, Autosomal Dominant	C12.050.351.968.419.403.875.500; C12.200.777.419.403.875.500; C12.950.419.403.875.500; C16.131.077.717.500; C16.320.184.625.500

Drugs & pharmacology

Drug and pharmacology data

Is drug target: yes

ChEMBL targets (1): CHEMBL5393 (SINGLE PROTEIN)

Molecules with ChEMBL bioactivity

92 molecules (phase ≥1), by development phase (incl. off-target/promiscuous compounds). Patent mentions across the top 20 by phase: 561,299 (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
CHEMBL1014	CANDESARTAN CILEXETIL	4	11,194
CHEMBL1017	TELMISARTAN	4	27,457
CHEMBL114	SAQUINAVIR	4	39,899
CHEMBL1163	ATAZANAVIR	4	22,094
CHEMBL1164729	FEBUXOSTAT	4	3,499
CHEMBL1171837	PONATINIB	4	8,955
CHEMBL1219	RABEPRAZOLE	4	12,441
CHEMBL1229211	DOLUTEGRAVIR	4	3,337
CHEMBL1289494	TIVOZANIB	4	4,455
CHEMBL1292	CLOFAZIMINE	4	15,481
CHEMBL1336	SORAFENIB	4	86,060
CHEMBL1397	POSACONAZOLE	4	541
CHEMBL1405	ESTRONE	4	36,722
CHEMBL1428	NIMODIPINE	4	32,587
CHEMBL1450	ATOVAQUONE	4	14,589
CHEMBL1484	NICARDIPINE	4	30,866
CHEMBL1487	ATORVASTATIN	4	68,788
CHEMBL1502	PANTOPRAZOLE	4	14,689
CHEMBL1503	OMEPRAZOLE	4	52,284
CHEMBL157101	KETOCONAZOLE	4	75,361
CHEMBL160	CYCLOSPORINE	4
CHEMBL163	RITONAVIR	4
CHEMBL1630575	CASPOFUNGIN	4
CHEMBL1738797	ALECTINIB	4
CHEMBL175691	RILPIVIRINE	4
CHEMBL191	LOSARTAN	4
CHEMBL193	NIFEDIPINE	4
CHEMBL1946170	REGORAFENIB	4
CHEMBL2095208	COBICISTAT	4
CHEMBL2103830	FOSTAMATINIB	4

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

PharmGKB clinical annotations

53 annotations.

Variant	Type	Level	Drugs	Phenotypes
rs10011796	Efficacy	4	allopurinol
rs1061018	Other	3	dasatinib;imatinib;nilotinib
rs12505410	Efficacy	3	imatinib	Chronic myelogenous leukemia;BCR-ABL1 positive
rs12505410	Metabolism/PK	3	methotrexate	Osteosarcoma
rs13120400	Efficacy	3	methotrexate	Psoriasis
rs13120400	Efficacy	3	imatinib	Chronic myelogenous leukemia;BCR-ABL1 positive
rs13120400	Efficacy,Metabolism/PK	3	deferasirox	Beta-thalassemia and related diseases
rs13120400	Metabolism/PK	3	ceftriaxone	Central Nervous System Infectious Disorder
rs13120400	Metabolism/PK	3	methotrexate	Osteosarcoma
rs13137622	Metabolism/PK	3	methotrexate	Osteosarcoma
rs17731538	Efficacy	3	methotrexate	Psoriasis
rs2199936	Efficacy	3	rosuvastatin
rs2231135	Toxicity	3	methotrexate	Osteosarcoma
rs2231137	Toxicity	3	irinotecan	Non-Small Cell Lung Carcinoma
rs2231137	Other	3	dasatinib;imatinib;nilotinib
rs2231137	Dosage	3	imatinib	Drug Toxicity;Gastrointestinal Stromal Tumors
rs2231142	Metabolism/PK	1A	rosuvastatin
rs2231142	Metabolism/PK	3	tenofovir	HIV infectious disease
rs2231142	Metabolism/PK	3	dolutegravir	HIV infectious disease
rs2231142	Toxicity	3	methotrexate	Rheumatoid arthritis
rs2231142	Metabolism/PK	3	apixaban	Atrial Fibrillation
rs2231142	Efficacy,Toxicity	3	gemcitabine	Non-Small Cell Lung Carcinoma
rs2231142	Efficacy	3	Opioid anesthetics;Other general anesthetics;volatile anesthetics
rs2231142	Toxicity	3	cyclophosphamide;doxorubicin;fluorouracil	Breast Neoplasms
rs2231142	Toxicity	3	sunitinib	Neoplasms
rs2231142	Metabolism/PK	3	lamotrigine	Epilepsy
rs2231142	Other	3	atorvastatin
rs2231142	Toxicity	1A	rosuvastatin	statin-related myopathy
rs2231142	Metabolism/PK	3	simvastatin
rs2231142	Metabolism/PK	3	fluvastatin
rs2231142	Toxicity	3	atorvastatin
rs2231142	Toxicity	4	gefitinib	Lung Neoplasms
rs2231142	Efficacy	4	capecitabine;fluorouracil;leucovorin;oxaliplatin	Colorectal Neoplasms
rs2231142	Metabolism/PK	4	methotrexate	Acute lymphoblastic leukemia;Burkitt Lymphoma;Lymphoma;T-Cell;Osteosarcoma
rs2231142	Toxicity	4	methotrexate	Acute lymphoblastic leukemia;Burkitt Lymphoma;Drug Toxicity;Lymphoma;T-Cell;Osteosarcoma
rs2231142	Efficacy	1A	allopurinol	Gout
rs2231142	Dosage	1A	allopurinol	Gout
rs2231142	Efficacy	2A	rosuvastatin	Hypercholesterolemia;Myocardial Infarction
rs2231142	Toxicity	3	efavirenz	HIV infectious disease
rs2231142	Efficacy	3	sulfasalazine	Rheumatoid arthritis

PharmGKB variants

53 variants.

Variant	Genes	Level	Score	#Clin annots	Drugs
rs1061018	ABCG2	3	0.00	1	dasatinib;imatinib;nilotinib
rs1481012	ABCG2		0.00	0
rs2231135	ABCG2	3	2.25	1	methotrexate
rs2231137	ABCG2	3	3.75	3	irinotecan;dasatinib;imatinib;nilotinib;imatinib
rs2231142	ABCG2	1A	115.62	26	rosuvastatin;atorvastatin;cyclophosphamide;doxorubicin;fluorouracil;allopurinol;simvastatin;fluvastatin;efavirenz;tenofovir;dolutegravir;Opioid anesthetics;Other general anesthetics;volatile anesthetics
rs2231148	ABCG2		0.00	0
rs2231164	ABCG2		0.00	0
rs2622604	ABCG2		0.00	0
rs2622628	ABCG2		0.00	0
rs2725252	ABCG2	3	2.00	1	imatinib
rs2725256	ABCG2		0.00	0
rs3109823	ABCG2		0.00	0
rs3114018	ABCG2		0.00	0
rs3114020	ABCG2	3	0.75	1	lamotrigine
rs3219191	ABCG2		0.00	0
rs4148157	ABCG2	3	1.75	2	allopurinol;topotecan
rs7699188	ABCG2	3	6.00	1	fluorouracil;irinotecan;leucovorin
rs10011796	ABCG2	4	-1.50	1	allopurinol
rs12505410	ABCG2	3	2.00	2	methotrexate;imatinib
rs13120400	ABCG2	3	4.25	5	methotrexate;ceftriaxone;imatinib;deferasirox
rs17731538	ABCG2	3	2.75	1	methotrexate
rs17731799	ABCG2		0.00	0
rs41282401	ABCG2	3	0.00	1	dasatinib;imatinib;nilotinib
rs45605536	ABCG2	3	0.00	1	dasatinib;imatinib
rs58818712	ABCG2	3	0.00	1	dasatinib;imatinib
rs72552713	ABCG2	3	0.12	1	sulfasalazine
rs2725264	ABCG2		0.00	0
rs9999111	ABCG2		0.00	0
rs2725263	ABCG2		0.00	0
rs1564481	ABCG2		0.00	0
rs13137622	ABCG2	3	2.00	1	methotrexate
rs2622621	ABCG2		0.00	0
rs6857600	ABCG2		0.00	0
rs45499402	ABCG2		0.00	0
rs4148155	ABCG2	3	0.00	1	allopurinol
rs76979899	ABCG2	3	0.00	1	allopurinol
rs1448784	ABCG2		0.00	0
rs569310717	ABCG2		0.00	0
rs149106245	ABCG2		0.00	0
rs769734146	ABCG2		0.00	0

PharmGKB dosing guidelines

3 guidelines.

Source	Drug	Guideline	Dosing?	Recommendation?
CPIC	atorvastatin;fluvastatin;lovastatin;pitavastatin;pravastatin;simvastatin	Annotation of CPIC Guideline for atorvastatin, fluvastatin, lovastatin, pitavastatin, pravastatin, simvastatin and ABCG2
CPIC	rosuvastatin	Annotation of CPIC Guideline for rosuvastatin and ABCG2, SLCO1B1	yes	yes
DPWG	allopurinol	Annotation of DPWG Guideline for allopurinol and ABCG2	yes	yes

GtoPdb / IUPHAR curated pharmacology

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

Target class: transporter — ABCG subfamily

Most potent curated ligand interactions (5 total), top 5:

Ligand	Action	Affinity	Parameter
elacridar	Inhibition	6.71	pIC50
cyclosporin A	Inhibition	6.3	pKi
KS 176	Inhibition	6.23	pIC50
compound 14 [PMID: 30925062]	Non-competitive	5.62	pIC50
nobiletin	Inhibition	5.36	pIC50

Binding affinities (BindingDB)

48 measured of 52 human assays (53 total across all organisms); most potent 48 below. Values come from heterogeneous assays and are not directly comparable.

Ligand	Measure	Value	Patent
3-chloranyl-N-[2-chloranyl-5-(trifluoromethyl)phenyl]-6-(3,5-dimethylpyrazol-1-yl)pyridine-2-carboxamide	EC50	240 nM
2-methoxy-N-(2-morpholinophenyl)-5-(phthalimidomethyl)benzamide	EC50	340 nM
(E)-3-[3-(4-methoxyphenyl)-1-phenyl-4-pyrazolyl]-1-[4-(2-pyridinyl)-1-piperazinyl]-2-propen-1-one	EC50	410 nM
(4-cyanophenyl)methyl 3-nitro-4-pyrrolidin-1-yl-benzoate	EC50	450 nM
N-[(E)-[3-[[4-(4-fluorophenyl)-1-piperazinyl]methyl]-4-methoxyphenyl]methylideneamino]-4-hydroxy-3-methoxybenzamide	EC50	760 nM
Fumitremorgin C	IC50	790 nM
(6E)-6-[4-(3,5-dimethyl-1-piperidinyl)-1H-quinazolin-2-ylidene]-2-methoxy-1-cyclohexa-2,4-dienone	EC50	1040 nM
CHEMBL4209777	IC50	1060 nM
N-[5-(dimethylamino)-2-[4-morpholinyl-[3-(trifluoromethyl)anilino]phosphoryl]phenyl]-2-thiophenecarboxamide	EC50	1210 nM
3,4,5-trimethoxy-N-[4-(propan-2-ylsulfamoyl)phenyl]benzamide	EC50	1320 nM
(6-nitro-4H-1,3-benzodioxin-8-yl)methyl 2-(benzenesulfonamido)-3-methylbutanoate	EC50	1570 nM
3-keto-2-(2-methoxyethyl)-N-p-phenetyl-isoindoline-4-carboxamide	EC50	1600 nM
2-[[1-oxo-2-(2-pyrimidinylthio)ethyl]amino]-6-(phenylmethyl)-5,7-dihydro-4H-thieno[2,3-c]pyridine-3-carboxylic acid ethyl ester	EC50	1880 nM
(2S,5S,8S)-14-methoxy-5,13-dimethyl-2-(2-methylpropyl)-3,6,17-triazatetracyclo[8.7.0.03,8.011,16]heptadeca-1(10),11,13,15-tetraene-4,7-dione	IC50	1980 nM	US-9695174: Inhibitor of breast cancer resistance protein (BCRP)
N-[2-[diethylamino-[3-(trifluoromethyl)anilino]phosphoryl]-5-(dimethylamino)phenyl]-2-furamide	EC50	2290 nM
NSC19139	IC50	2600 nM
4-(dimethylamino)benzoic acid [2-(2-ethoxyanilino)-2-oxo-1-phenylethyl] ester	EC50	2750 nM
tert-butyl 3-[(2S,5S,8S)-2-(2-methylpropyl)-4,7-dioxo-3,6,17-triazatetracyclo[8.7.0.03,8.011,16]heptadeca-1(10),11,13,15-tetraen-5-yl]propanoate	IC50	3250 nM	US-9695174: Inhibitor of breast cancer resistance protein (BCRP)
NSC375985	IC50	3700 nM
2-[4-(4-cyanophenyl)phenoxy]-N-(2-phenylethyl)acetamide	EC50	3760 nM
2-[(2-fluoranyl-6-nitro-phenyl)methylsulfanyl]-4,6-dimethyl-pyridine-3-carbonitrile	EC50	3820 nM
NSC168201	IC50	3900 nM
(4E)-4-(dimethylaminomethylene)-5-methyl-2-(4-nitrophenyl)-2-pyrazolin-3-one	EC50	4000 nM
2-fluoranyl-N-[(E)-[3-[[4-(4-fluorophenyl)piperazin-1-yl]methyl]-4-methoxy-phenyl]methylideneamino]benzamide	EC50	4070 nM
NSC120688	IC50	4300 nM
2-[7-[butyl(methyl)amino]-4,4a,5,6-tetrahydro-3H-naphthalen-2-ylidene]malononitrile	EC50	4420 nM
cid_223753	IC50	4500 nM
NSC320852	IC50	4600 nM
4-[1-[benzyl-[2-(2-thienyl)acetyl]amino]-2-(cyclohexylamino)-2-keto-ethyl]benzoic acid methyl ester	EC50	4600 nM
2-[[4-chloranyl-6-[(4-nitrophenyl)amino]-1,3,5-triazin-2-yl]amino]ethanol	EC50	4630 nM
3-(5-bromanylfuran-2-yl)-5-(2-nitrophenyl)-1,2,4-oxadiazole	EC50	4880 nM
NSC306698	IC50	5400 nM
NSC303769	IC50	5800 nM
N-(4-fluorophenyl)-2-methyl-3-(2-pyridin-4-ylethyl)-1-indolizinecarboxamide	EC50	6840 nM
(Z)-N-[(E)-2-(3,5-dibromo-4-hydroxyphenyl)ethenyl]-3-(4-hydroxyphenyl)-2-methoxyprop-2-enamide	IC50	6900 nM	US-8470888: Botryllamides and method of inhibiting PGP in a mammal afflicted with cancer
Digitonin	IC50	9700 nM
(Z)-N-[(E)-2-(3,5-dibromo-4-methoxyphenyl)ethenyl]-3-(4-hydroxyphenyl)-2-methoxyprop-2-enamide	IC50	11200 nM	US-8470888: Botryllamides and method of inhibiting PGP in a mammal afflicted with cancer
Botryllamide B, 2	IC50	11200 nM
3-(quinoxaline-2-carbonylamino)benzoic acid ethyl ester	EC50	11800 nM
(2S,5S,8S)-14-methoxy-5-methyl-2-(2-methylpropyl)-3,6,17-triazatetracyclo[8.7.0.03,8.011,16]heptadeca-1(10),11,13,15-tetraene-4,7-dione	IC50	15400 nM	US-9695174: Inhibitor of breast cancer resistance protein (BCRP)
(Z)-N-[(E)-2-(3-bromo-4-methoxyphenyl)ethenyl]-3-(4-hydroxyphenyl)-2-methoxyprop-2-enamide	IC50	16400 nM	US-8470888: Botryllamides and method of inhibiting PGP in a mammal afflicted with cancer
Botryllamide D, 4	IC50	16400 nM
(Z)-3-(4-hydroxyphenyl)-N-[(E)-2-(4-hydroxyphenyl)ethenyl]-2-methoxyprop-2-enamide	IC50	16700 nM	US-8470888: Botryllamides and method of inhibiting PGP in a mammal afflicted with cancer
(Z)-3-(4-hydroxyphenyl)-2-methoxy-N-[(E)-2-(4-methoxyphenyl)ethenyl]prop-2-enamide	IC50	23300 nM	US-8470888: Botryllamides and method of inhibiting PGP in a mammal afflicted with cancer
1-keto-7,8-dimethoxy-isothiochromene-3-carboxylic acid isopropyl ester	EC50	24400 nM
Botryllamide J, 10	IC50	26900 nM
(Z)-3-(2-cyano-3-hydroxyphenyl)-2-methoxy-N-[(E)-2-phenylethenyl]prop-2-enamide	IC50	26900 nM	US-8470888: Botryllamides and method of inhibiting PGP in a mammal afflicted with cancer
Botryllamide I, 9	IC50	41400 nM

ChEMBL bioactivities

1955 potent at pChembl≥5 of 2203 total, top 50 by pChembl (potency: 10 = 0.1 nM, 6 = 1 µM).

pChembl	Type	Value	Unit	Molecule
10.99	EC50	0.01019	nM	CHEMBL2141796
10.99	EC50	0.01013	nM	CHEMBL2145384
10.96	EC50	0.01088	nM	CHEMBL2142245
10.92	EC50	0.01209	nM	CHEMBL2137928
10.64	EC50	0.02297	nM	CHEMBL2141666
10.63	EC50	0.02338	nM	CHEMBL2130718
10.39	EC50	0.04032	nM	CHEMBL2140166
10.39	EC50	0.04093	nM	CHEMBL2137680
10.35	EC50	0.04419	nM	CHEMBL2135227
10.32	EC50	0.04775	nM	CHEMBL2133727
9.60	EC50	0.254	nM	CHEMBL2132526
9.22	EC50	0.6	nM	CHEMBL4168440
9.05	EC50	0.9	nM	CHEMBL4561302
9.05	EC50	0.9	nM	CHEMBL4584493
8.96	EC50	1.1	nM	CHEMBL4453052
8.92	EC50	1.2	nM	CHEMBL4159635
8.85	EC50	1.4	nM	CHEMBL4561302
8.85	EC50	1.4	nM	CHEMBL4860031
8.82	IC50	1.5	nM	AVAPRITINIB
8.80	EC50	1.6	nM	CHEMBL4860031
8.77	EC50	1.7	nM	CHEMBL4454217
8.70	EC50	1.999	nM	CHEMBL2133413
8.70	EC50	2	nM	CHEMBL4101737
8.70	EC50	2	nM	CHEMBL4172744
8.70	EC50	2	nM	CHEMBL4591134
8.70	EC50	2	nM	CHEMBL4454217
8.68	EC50	2.1	nM	CHEMBL4474920
8.66	EC50	2.2	nM	CHEMBL4442810
8.66	EC50	2.2	nM	CHEMBL4453052
8.64	EC50	2.3	nM	CHEMBL4591134
8.62	EC50	2.4	nM	CHEMBL4440681
8.62	EC50	2.4	nM	CHEMBL4592756
8.60	EC50	2.5	nM	CHEMBL4453052
8.55	EC50	2.8	nM	CHEMBL4545977
8.55	EC50	2.8	nM	CHEMBL4584493
8.52	EC50	3	nM	CHEMBL4073213
8.51	EC50	3.1	nM	CHEMBL4291291
8.48	EC50	3.3	nM	CHEMBL4454945
8.48	EC50	3.3	nM	CHEMBL4589907
8.46	EC50	3.5	nM	CHEMBL3092485
8.46	EC50	3.5	nM	CHEMBL4463713
8.46	EC50	3.5	nM	CHEMBL4877122
8.44	EC50	3.6	nM	CHEMBL4564209
8.41	EC50	3.9	nM	CHEMBL4472194
8.40	EC50	4	nM	CHEMBL4082228
8.40	EC50	4	nM	CHEMBL4081708
8.40	EC50	4	nM	CHEMBL4094899
8.40	EC50	4	nM	CHEMBL4091528
8.40	EC50	4	nM	CHEMBL4072298
8.40	EC50	4	nM	CHEMBL4176228

PubChem BioAssay actives

1905 with measured affinity, of 5039 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
N-(3-methylsulfanylphenyl)-2-(3-nitrophenyl)quinazolin-4-amine	1501277: Activation of ABCG2 ATPase activity (unknown origin) expressed in baculovirus infected high5 cell membranes after 20 mins by ascorbic acid ammonia molybdate reaction based colorimetric assay	ec50	0.0006	uM
methyl 3-[[2-[4-[2-[2-[2-[4-[4-[4-(6-methyl-4-oxochromen-2-yl)phenoxy]butyl]triazol-1-yl]ethoxy]ethoxy]ethoxy]phenyl]-4-oxochromen-3-yl]oxymethyl]benzoate	1525920: Inhibition of BCRP in human MCF7/MX100 cells assessed as reversal of topotecan resistance after 5 days by MTS/PMS assay	ec50	0.0009	uM
methyl 3-[[4-oxo-2-[4-[2-[2-[2-[4-[4-(4-oxo-2-phenylchromen-7-yl)oxybutyl]triazol-1-yl]ethoxy]ethoxy]ethoxy]phenyl]chromen-3-yl]oxymethyl]benzoate	1525919: Inhibition of BCRP (unknown origin) transfected in HEK293/R2 cells assessed as reversal of topotecan resistance after 5 days by MTS/PMS assay	ec50	0.0009	uM
methyl 3-[[2-[4-[2-[2-[4-[3-[1-[2-[2-[4-[3-[(3-methoxycarbonylphenyl)methoxy]-4-oxochromen-2-yl]phenoxy]ethoxy]ethyl]triazol-4-yl]phenyl]triazol-1-yl]ethoxy]ethoxy]phenyl]-4-oxochromen-3-yl]oxymethyl]benzoate	1525920: Inhibition of BCRP in human MCF7/MX100 cells assessed as reversal of topotecan resistance after 5 days by MTS/PMS assay	ec50	0.0011	uM
N-(3,4-dimethoxyphenyl)-2-(4-methoxyphenyl)quinazolin-4-amine	1501277: Activation of ABCG2 ATPase activity (unknown origin) expressed in baculovirus infected high5 cell membranes after 20 mins by ascorbic acid ammonia molybdate reaction based colorimetric assay	ec50	0.0012	uM
methyl 3-[[2-[4-[2-[2-[2-[4-[[benzyl(2-hydroxyethyl)amino]methyl]triazol-1-yl]ethoxy]ethoxy]ethoxy]phenyl]-4-oxochromen-3-yl]oxymethyl]benzoate	1769259: Inhibition of BCRP (unknown origin) transfected in HEK293/R2 cells assessed as reversal of topotecan resistance after 5 days by MTS/PMS assay	ec50	0.0014	uM
Avapritinib	1916975: Inhibition of ABCG2 (unknown origin)	ic50	0.0015	uM
methyl 3-[[4-oxo-2-[4-[2-[2-[2-[4-[2-(4-oxo-2-phenylchromen-7-yl)oxyethoxymethyl]triazol-1-yl]ethoxy]ethoxy]ethoxy]phenyl]chromen-3-yl]oxymethyl]benzoate	1525919: Inhibition of BCRP (unknown origin) transfected in HEK293/R2 cells assessed as reversal of topotecan resistance after 5 days by MTS/PMS assay	ec50	0.0017	uM
4-[[2-(3-methoxyphenyl)pyrido[2,3-d]pyrimidin-4-yl]amino]phenol	1479734: Activation of recombinant human ABCG2 ATPase activity expressed in baculovirus infected High five insect cell membranes after 20 mins by ascorbic acid ammonia molybdate reaction-based colorimetric method	ec50	0.0020	uM
N,2-bis(3-nitrophenyl)quinazolin-4-amine	1501277: Activation of ABCG2 ATPase activity (unknown origin) expressed in baculovirus infected high5 cell membranes after 20 mins by ascorbic acid ammonia molybdate reaction based colorimetric assay	ec50	0.0020	uM
methyl 3-[[2-[4-[2-[2-[2-[4-[3-[4-(7-fluoro-4-oxochromen-2-yl)phenoxy]propyl]triazol-1-yl]ethoxy]ethoxy]ethoxy]phenyl]-4-oxochromen-3-yl]oxymethyl]benzoate	1525919: Inhibition of BCRP (unknown origin) transfected in HEK293/R2 cells assessed as reversal of topotecan resistance after 5 days by MTS/PMS assay	ec50	0.0020	uM
methyl 3-[[2-[4-[2-[2-[4-[[1-[2-[2-[4-[3-[(3-methoxycarbonylphenyl)methoxy]-4-oxochromen-2-yl]phenoxy]ethoxy]ethyl]triazol-4-yl]methoxymethyl]triazol-1-yl]ethoxy]ethoxy]phenyl]-4-oxochromen-3-yl]oxymethyl]benzoate	1525920: Inhibition of BCRP in human MCF7/MX100 cells assessed as reversal of topotecan resistance after 5 days by MTS/PMS assay	ec50	0.0021	uM
methyl 3-[[2-[4-[2-[2-[4-[[N-[[1-[2-[2-[4-[3-[(3-methoxycarbonylphenyl)methoxy]-4-oxochromen-2-yl]phenoxy]ethoxy]ethyl]triazol-4-yl]methyl]anilino]methyl]triazol-1-yl]ethoxy]ethoxy]phenyl]-4-oxochromen-3-yl]oxymethyl]benzoate	1525920: Inhibition of BCRP in human MCF7/MX100 cells assessed as reversal of topotecan resistance after 5 days by MTS/PMS assay	ec50	0.0022	uM
methyl 3-[[2-[4-[2-[2-[4-[3-[1-[2-[2-[4-[3-[(3-methoxycarbonylphenyl)methoxy]-4-oxochromen-2-yl]phenoxy]ethoxy]ethyl]triazol-4-yl]propyl]triazol-1-yl]ethoxy]ethoxy]phenyl]-4-oxochromen-3-yl]oxymethyl]benzoate	1525920: Inhibition of BCRP in human MCF7/MX100 cells assessed as reversal of topotecan resistance after 5 days by MTS/PMS assay	ec50	0.0024	uM
methyl 3-[[2-[4-[2-[2-[2-[4-[4-[1-[2-[2-[2-[4-[3-[(3-methoxycarbonylphenyl)methoxy]-4-oxochromen-2-yl]phenoxy]ethoxy]ethoxy]ethyl]triazol-4-yl]phenyl]triazol-1-yl]ethoxy]ethoxy]ethoxy]phenyl]-4-oxochromen-3-yl]oxymethyl]benzoate	1525919: Inhibition of BCRP (unknown origin) transfected in HEK293/R2 cells assessed as reversal of topotecan resistance after 5 days by MTS/PMS assay	ec50	0.0024	uM
methyl 3-[[2-[4-[2-[2-[2-[4-[3-[1-[2-[2-[2-[4-[3-[(3-methoxycarbonylphenyl)methoxy]-4-oxochromen-2-yl]phenoxy]ethoxy]ethoxy]ethyl]triazol-4-yl]phenyl]triazol-1-yl]ethoxy]ethoxy]ethoxy]phenyl]-4-oxochromen-3-yl]oxymethyl]benzoate	1525919: Inhibition of BCRP (unknown origin) transfected in HEK293/R2 cells assessed as reversal of topotecan resistance after 5 days by MTS/PMS assay	ec50	0.0028	uM
4-[[2-(3-methoxyphenyl)pyrido[2,3-d]pyrimidin-4-yl]amino]benzonitrile	1479734: Activation of recombinant human ABCG2 ATPase activity expressed in baculovirus infected High five insect cell membranes after 20 mins by ascorbic acid ammonia molybdate reaction-based colorimetric method	ec50	0.0030	uM
7-fluoro-2-[4-[3-[1-[2-[2-[2-[4-(6-fluoro-4-oxochromen-2-yl)phenoxy]ethoxy]ethoxy]ethyl]triazol-4-yl]propoxy]phenyl]chromen-4-one	1525919: Inhibition of BCRP (unknown origin) transfected in HEK293/R2 cells assessed as reversal of topotecan resistance after 5 days by MTS/PMS assay	ec50	0.0031	uM
methyl 3-[[2-[4-[2-[2-[2-[4-[[benzyl-[[1-[2-[2-[2-[4-[3-[(3-methoxycarbonylphenyl)methoxy]-4-oxochromen-2-yl]phenoxy]ethoxy]ethoxy]ethyl]triazol-4-yl]methyl]amino]methyl]triazol-1-yl]ethoxy]ethoxy]ethoxy]phenyl]-4-oxochromen-3-yl]oxymethyl]benzoate	1525919: Inhibition of BCRP (unknown origin) transfected in HEK293/R2 cells assessed as reversal of topotecan resistance after 5 days by MTS/PMS assay	ec50	0.0033	uM
methyl 3-[[2-[4-[2-[2-[4-[[benzyl-[[1-[2-[2-[4-[3-[(3-methoxycarbonylphenyl)methoxy]-4-oxochromen-2-yl]phenoxy]ethoxy]ethyl]triazol-4-yl]methyl]amino]methyl]triazol-1-yl]ethoxy]ethoxy]phenyl]-4-oxochromen-3-yl]oxymethyl]benzoate	1525920: Inhibition of BCRP in human MCF7/MX100 cells assessed as reversal of topotecan resistance after 5 days by MTS/PMS assay	ec50	0.0033	uM
methyl 3-[[4-oxo-2-[4-[2-[2-[2-[4-[3-(4-oxo-2-phenylchromen-7-yl)oxypropyl]triazol-1-yl]ethoxy]ethoxy]ethoxy]phenyl]chromen-3-yl]oxymethyl]benzoate	1525919: Inhibition of BCRP (unknown origin) transfected in HEK293/R2 cells assessed as reversal of topotecan resistance after 5 days by MTS/PMS assay	ec50	0.0035	uM
methyl 3-[[2-[4-[2-[2-[4-[[benzyl(methyl)amino]methyl]triazol-1-yl]ethoxy]ethoxy]phenyl]-4-oxochromen-3-yl]oxymethyl]benzoate	1769261: Inhibition of BCRP (unknown origin) expressed in MCF7/MX100 cells assessed as reversal of topotecan resistance after 5 days by MTS/PMS assay	ec50	0.0035	uM
2-(3,4-dimethoxyphenyl)-N-(3-nitrophenyl)quinazolin-4-amine	1501277: Activation of ABCG2 ATPase activity (unknown origin) expressed in baculovirus infected high5 cell membranes after 20 mins by ascorbic acid ammonia molybdate reaction based colorimetric assay	ec50	0.0035	uM
methyl 3-[[2-[4-[2-[2-[4-[4-[4-(6-methyl-4-oxochromen-2-yl)phenoxy]butyl]triazol-1-yl]ethoxy]ethoxy]phenyl]-4-oxochromen-3-yl]oxymethyl]benzoate	1525919: Inhibition of BCRP (unknown origin) transfected in HEK293/R2 cells assessed as reversal of topotecan resistance after 5 days by MTS/PMS assay	ec50	0.0036	uM
methyl 3-[[2-[4-[2-[2-[2-[4-[[N-[[1-[2-[2-[2-[4-[3-[(3-methoxycarbonylphenyl)methoxy]-4-oxochromen-2-yl]phenoxy]ethoxy]ethoxy]ethyl]triazol-4-yl]methyl]anilino]methyl]triazol-1-yl]ethoxy]ethoxy]ethoxy]phenyl]-4-oxochromen-3-yl]oxymethyl]benzoate	1525920: Inhibition of BCRP in human MCF7/MX100 cells assessed as reversal of topotecan resistance after 5 days by MTS/PMS assay	ec50	0.0039	uM
tert-butyl 3-[(2S,5S,8S)-14-methoxy-2-(2-methylpropyl)-4,7-dioxo-3,6,17-triazatetracyclo[8.7.0.03,8.011,16]heptadeca-1(10),11,13,15-tetraen-5-yl]propanoate	1635795: Inhibition of human ABCG2 expressed in baculovirus infected Sf9 cell membrane assessed as inhibition of vanadate sensitive basal ATPase activity after 20 mins by colorimetric method	ic50	0.0040	uM
N-(3-nitrophenyl)-2-pyridin-3-ylpyrido[2,3-d]pyrimidin-4-amine	1479734: Activation of recombinant human ABCG2 ATPase activity expressed in baculovirus infected High five insect cell membranes after 20 mins by ascorbic acid ammonia molybdate reaction-based colorimetric method	ec50	0.0040	uM
N-(3-nitrophenyl)-2-pyridin-4-ylquinazolin-4-amine	1447895: Activation of ABCG2 (unknown origin) ATPase activity expressed in baculovirus infected high five cell membranes by ascorbic acid/ammonia molybdate reaction-based colorimetric analysis	ec50	0.0040	uM
4-[(2-pyridin-3-ylquinazolin-4-yl)amino]benzonitrile	1447895: Activation of ABCG2 (unknown origin) ATPase activity expressed in baculovirus infected high five cell membranes by ascorbic acid/ammonia molybdate reaction-based colorimetric analysis	ec50	0.0040	uM
N-(4-fluorophenyl)-2-(3-methoxyphenyl)pyrido[2,3-d]pyrimidin-4-amine	1479734: Activation of recombinant human ABCG2 ATPase activity expressed in baculovirus infected High five insect cell membranes after 20 mins by ascorbic acid ammonia molybdate reaction-based colorimetric method	ec50	0.0040	uM
3-[[2-(3-methoxyphenyl)pyrido[2,3-d]pyrimidin-4-yl]amino]benzonitrile	1479734: Activation of recombinant human ABCG2 ATPase activity expressed in baculovirus infected High five insect cell membranes after 20 mins by ascorbic acid ammonia molybdate reaction-based colorimetric method	ec50	0.0040	uM
N-(3,4-dimethoxyphenyl)-2-(3-methoxyphenyl)quinazolin-4-amine	1501277: Activation of ABCG2 ATPase activity (unknown origin) expressed in baculovirus infected high5 cell membranes after 20 mins by ascorbic acid ammonia molybdate reaction based colorimetric assay	ec50	0.0040	uM
N-(4-bromo-3-methoxyphenyl)-2-(3,4-dimethoxyphenyl)quinazolin-4-amine	1501277: Activation of ABCG2 ATPase activity (unknown origin) expressed in baculovirus infected high5 cell membranes after 20 mins by ascorbic acid ammonia molybdate reaction based colorimetric assay	ec50	0.0040	uM
2-(3-methoxyphenyl)-N-(3-nitrophenyl)quinazolin-4-amine	1501277: Activation of ABCG2 ATPase activity (unknown origin) expressed in baculovirus infected high5 cell membranes after 20 mins by ascorbic acid ammonia molybdate reaction based colorimetric assay	ec50	0.0040	uM
methyl 3-[[4-oxo-2-[4-[2-[2-[4-[2-(4-oxo-2-phenylchromen-7-yl)oxyethoxymethyl]triazol-1-yl]ethoxy]ethoxy]phenyl]chromen-3-yl]oxymethyl]benzoate	1525919: Inhibition of BCRP (unknown origin) transfected in HEK293/R2 cells assessed as reversal of topotecan resistance after 5 days by MTS/PMS assay	ec50	0.0041	uM
2-[(2S,5S,8S)-14-methoxy-2-(2-methylpropyl)-4,7-dioxo-3,6,17-triazatetracyclo[8.7.0.03,8.011,16]heptadeca-1(10),11,13,15-tetraen-5-yl]ethyl 2,2-dimethylpropanoate	1668468: Reversal of human ABCG2-mediated multidrug resistance in HEK293/R482 cells assessed as effect on mitoxantrone-induced cytotoxicity by measuring mitoxantrone IC50 at 1 uM after 72 hrs by CCK8 assay (Rvb = 50.66 +/- 6.88 nM)	ic50	0.0046	uM
2-(3,4-dimethoxyphenyl)-N-(4-nitrophenyl)quinazolin-4-amine	1501277: Activation of ABCG2 ATPase activity (unknown origin) expressed in baculovirus infected high5 cell membranes after 20 mins by ascorbic acid ammonia molybdate reaction based colorimetric assay	ec50	0.0047	uM
N-(4-nitrophenyl)-2-phenylpyrido[2,3-d]pyrimidin-4-amine	1479734: Activation of recombinant human ABCG2 ATPase activity expressed in baculovirus infected High five insect cell membranes after 20 mins by ascorbic acid ammonia molybdate reaction-based colorimetric method	ec50	0.0050	uM
2-nitro-4-[[2-(3-nitrophenyl)quinazolin-4-yl]amino]phenol	1501277: Activation of ABCG2 ATPase activity (unknown origin) expressed in baculovirus infected high5 cell membranes after 20 mins by ascorbic acid ammonia molybdate reaction based colorimetric assay	ec50	0.0050	uM
tert-butyl 3-[(2S,5S,8S)-14-methoxy-2-(2-methylpropyl)-7-oxo-3,6,17-triazatetracyclo[8.7.0.03,8.011,16]heptadeca-1(10),11,13,15-tetraen-5-yl]propanoate	1769268: Inhibition of BCRP (unknown origin) expressed in human HEK293/R2 cells assessed as reversal of mitoxantrone resistance after 5 days by MTS/PMS assay	ec50	0.0050	uM
methyl 3-[[4-oxo-2-[4-[2-[2-[4-[4-(4-oxo-2-phenylchromen-7-yl)oxybutyl]triazol-1-yl]ethoxy]ethoxy]phenyl]chromen-3-yl]oxymethyl]benzoate	1525919: Inhibition of BCRP (unknown origin) transfected in HEK293/R2 cells assessed as reversal of topotecan resistance after 5 days by MTS/PMS assay	ec50	0.0052	uM
2-(3-methoxyphenyl)-N-(4-nitrophenyl)quinazolin-4-amine	1501277: Activation of ABCG2 ATPase activity (unknown origin) expressed in baculovirus infected high5 cell membranes after 20 mins by ascorbic acid ammonia molybdate reaction based colorimetric assay	ec50	0.0060	uM
methyl 3-[[2-[4-[4-(1-benzyltriazol-4-yl)butoxy]phenyl]-4-oxochromen-3-yl]oxymethyl]benzoate	1769261: Inhibition of BCRP (unknown origin) expressed in MCF7/MX100 cells assessed as reversal of topotecan resistance after 5 days by MTS/PMS assay	ec50	0.0060	uM
(E)-3-[4-hydroxy-2-methoxy-5-(2-methylbut-3-en-2-yl)phenyl]-1-(4-hydroxyphenyl)prop-2-en-1-one	1668466: Reversal of human ABCG2-mediated multidrug resistance in HEK293/R482 cells assessed as effect on mitoxantrone-induced cytotoxicity by measuring mitoxantrone IC50 at 3 uM after 72 hrs by CCK8 assay (Rvb = 50.66 +/- 6.88 nM)	ic50	0.0062	uM
7-[2-[2-[4-[3-[4-(7-fluoro-4-oxochromen-2-yl)phenoxy]propyl]triazol-1-yl]ethoxy]ethoxy]-2-phenylchromen-4-one	1525919: Inhibition of BCRP (unknown origin) transfected in HEK293/R2 cells assessed as reversal of topotecan resistance after 5 days by MTS/PMS assay	ec50	0.0064	uM
methyl 3-[[2-[4-[2-[2-[4-[3-[4-(7-fluoro-4-oxochromen-2-yl)phenoxy]propyl]triazol-1-yl]ethoxy]ethoxy]phenyl]-4-oxochromen-3-yl]oxymethyl]benzoate	1525920: Inhibition of BCRP in human MCF7/MX100 cells assessed as reversal of topotecan resistance after 5 days by MTS/PMS assay	ec50	0.0065	uM
2-nitro-4-[(2-pyridin-3-ylquinazolin-4-yl)amino]phenol	1447895: Activation of ABCG2 (unknown origin) ATPase activity expressed in baculovirus infected high five cell membranes by ascorbic acid/ammonia molybdate reaction-based colorimetric analysis	ec50	0.0070	uM
methyl 3-[[4-oxo-2-[4-[2-[2-[4-[3-(4-oxo-2-phenylchromen-7-yl)oxypropyl]triazol-1-yl]ethoxy]ethoxy]phenyl]chromen-3-yl]oxymethyl]benzoate	1525919: Inhibition of BCRP (unknown origin) transfected in HEK293/R2 cells assessed as reversal of topotecan resistance after 5 days by MTS/PMS assay	ec50	0.0072	uM
7-fluoro-2-[4-[3-[1-[2-[2-[4-(4-oxochromen-2-yl)phenoxy]ethoxy]ethyl]triazol-4-yl]propoxy]phenyl]chromen-4-one	1525919: Inhibition of BCRP (unknown origin) transfected in HEK293/R2 cells assessed as reversal of topotecan resistance after 5 days by MTS/PMS assay	ec50	0.0072	uM
7-fluoro-2-[4-[3-[1-[2-[2-[2-[4-(4-oxochromen-2-yl)phenoxy]ethoxy]ethoxy]ethyl]triazol-4-yl]propoxy]phenyl]chromen-4-one	1525920: Inhibition of BCRP in human MCF7/MX100 cells assessed as reversal of topotecan resistance after 5 days by MTS/PMS assay	ec50	0.0074	uM

CTD chemical–gene interactions

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

Chemical	Actions (top 5)	PubMed papers
Mitoxantrone	increases transport, decreases activity, decreases export, affects export, decreases abundance (+12 more)	24
3-(6-isobutyl-9-methoxy-1,4-dioxo-1,2,3,4,6,7,12,12a-octahydropyrazino(1’,2’-1,6)pyrido(3,4-b)indol-3-yl)propionic acid tert-butyl ester	decreases export, decreases activity, increases expression, affects reaction, decreases reaction (+6 more)	14
Tetrachlorodibenzodioxin	decreases reaction, increases expression, affects cotreatment	12
Benzo(a)pyrene	decreases activity, decreases methylation, increases activity, increases reaction, increases expression (+4 more)	11
Estradiol	increases expression, increases reaction, affects transport, decreases reaction, increases uptake (+6 more)	11
Cisplatin	affects expression, affects response to substance, decreases expression, affects cotreatment, decreases response to substance (+2 more)	10
Methotrexate	increases export, affects response to substance, decreases response to substance, increases transport, decreases reaction (+3 more)	10
tryptoquivaline	increases export, affects transport, affects uptake, decreases activity, decreases export (+2 more)	9
bisbenzimide ethoxide trihydrochloride	decreases activity, decreases export, decreases abundance, increases transport, increases reaction (+6 more)	9
Irinotecan	affects response to substance, affects metabolic processing, increases response to substance, affects export, decreases response to substance (+2 more)	9
Doxorubicin	affects transport, affects localization, affects cotreatment, decreases reaction, increases export (+7 more)	9
Topotecan	increases expression, affects export, increases response to substance, decreases activity, decreases export (+6 more)	9
estrone sulfate	decreases reaction, affects export, affects transport, increases export, increases transport (+2 more)	7
Sulfasalazine	decreases reaction, increases expression, increases activity, increases transport, affects cotreatment (+4 more)	7
Valproic Acid	affects cotreatment, increases expression, affects expression	7
Cyclosporine	decreases reaction, increases export, increases transport, decreases activity, increases expression (+2 more)	7
2-amino-1-methyl-6-phenylimidazo(4,5-b)pyridine	increases secretion, decreases reaction, increases expression, affects transport, increases export (+4 more)	6
Resveratrol	increases export, decreases expression, affects transport, increases transport, increases expression (+2 more)	5
Arsenic Trioxide	affects expression, decreases expression, increases expression, decreases reaction, increases reaction	5
Fulvestrant	affects cotreatment, increases expression, affects response to substance, affects reaction, decreases expression (+1 more)	5
Cadmium	decreases uptake, affects cotreatment, increases abundance, affects response to substance, affects uptake (+3 more)	5
Fluorouracil	decreases expression, decreases reaction, decreases response to substance, affects cotreatment, affects response to substance (+1 more)	5
Tobacco Smoke Pollution	increases expression, increases reaction, affects expression, decreases reaction, increases export	5
Cadmium Chloride	affects response to substance, decreases response to substance, decreases activity, affects cotreatment, decreases expression (+7 more)	5
bisphenol A	increases transport, decreases activity, decreases transport, decreases expression, affects reaction (+1 more)	4
chrysin	decreases expression, increases expression, decreases reaction, increases secretion, increases transport (+2 more)	4
Acetaminophen	decreases expression, decreases activity, increases expression	4
Chlorpyrifos	decreases response to substance, increases expression, affects cotreatment, affects expression	4
Novobiocin	decreases activity, decreases reaction, decreases export, increases abundance, increases uptake	4
Phenobarbital	affects expression, increases expression	4

ChEMBL screening assays

878 unique, capped per target: 651 binding, 115 admet, 111 functional, 1 toxicity

Representative assays (with source publication via chembl_document):

Assay ID	Type	Description	Source paper
CHEMBL1035250	Binding	Inhibition of BCRP expressed in human MCF7 MX cells at 31.6 uM by Hoechst 33342 staining relative to 10 uM XR9577	Aromatic 2-(thio)ureidocarboxylic acids as a new family of modulators of multidrug resistance-associated protein 1: synthesis, biological evaluation, and structure-activity relationships. — J Med Chem
CHEMBL1743158	ADMET	Substrates of transporters of clinical importance in the absorption and disposition of drugs, BCRP	Membrane transporters in drug development. — Nat Rev Drug Discov
CHEMBL2075183	Functional	TP_TRANSPORTER: inhibition of mitoxantrone efflux in BCRP-expressing MCF7-MX cells	Transport of glyburide by placental ABC transporters: implications in fetal drug exposure. — Placenta

Cellosaurus cell lines

19 cell lines: 16 cancer cell line, 2 spontaneously immortalized cell line, 1 induced pluripotent stem cell

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

Cellosaurus	Name	Category	Sex
CVCL_0H87	S1-M1-80	Cancer cell line	Female
CVCL_5227	OCI-AML3/HaABCG2 clone 6.2	Cancer cell line	Male
CVCL_A2KC	MDCK-hBCRP cMDR1-KO	Spontaneously immortalized cell line	Female
CVCL_A5XR	NCI-H460/TPT10 ABCG2 KO	Cancer cell line	Male
CVCL_B1EH	Abcam A-549 ABCG2 KO	Cancer cell line	Male
CVCL_B5ZS	C2BBe1 BCRP KO	Cancer cell line	Male
CVCL_B5ZU	C2BBe1 MDR1/BCRP KO	Cancer cell line	Male
CVCL_B5ZX	C2BBe1 MRP2/BCRP KO	Cancer cell line	Male
CVCL_B6A8	HepaRG BCRP KO	Cancer cell line	Female
CVCL_D6NG	OCI-AML3/HaABCG2 clone 3.3	Cancer cell line	Male

Clinical trials (associated diseases)

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

Trial	Phase	Status	Title
NCT00414440	PHASE4	COMPLETED	Efficacy, Safety and Tolerability of Everolimus in Preventing End-stage Renal Disease in Patients With Autosomal Dominant Polycystic Kidney Disease
NCT03273413	PHASE4	ACTIVE_NOT_RECRUITING	Statin Therapy in Patients With Early Stage ADPKD
NCT03949894	PHASE4	COMPLETED	Evaluating the Safety and effectivenesS in Adult KorEaN Patients Treated With Tolvaptan for Management of Autosomal domInAnt poLycystic Kidney Disease
NCT02562170	PHASE4	COMPLETED	Protexa® Versus TiLoopBra® in Immediate Breast Reconstruction- A Pilot Study
NCT00309283	PHASE3	COMPLETED	Somatostatin in Polycystic Kidney: a Long-term Three Year Follow up Study
NCT00346918	PHASE3	COMPLETED	Sirolimus (Rapamune®) for Autosomal Dominant Polycystic Kidney Disease (ADPKD)
NCT00428948	PHASE3	COMPLETED	Tolvaptan Phase 3 Efficacy and Safety Study in Autosomal Dominant Polycystic Kidney Disease (ADPKD)
NCT01022424	PHASE3	COMPLETED	A Long-term Administration Study of OPC-41061 in Patients With Autosomal Dominant Polycystic Kidney Disease (ADPKD) (2) [Extension of Study 156-05-002]
NCT01214421	PHASE3	COMPLETED	Tolvaptan Extension Study in Participants With ADPKD
NCT01377246	PHASE3	COMPLETED	Somatostatin In Patients With Autosomal Dominant Polycystic Kidney Disease And Moderate To Severe Renal Insufficiency
NCT01616927	PHASE3	UNKNOWN	Study of Lanreotide to Treat Polycystic Kidney Disease
NCT01853553	PHASE3	COMPLETED	Mineralocorticoid Antagonism and Endothelial Dysfunction in Autosomal Dominant Polycystic Kidney Disease (ADPKD)
NCT02115659	PHASE3	UNKNOWN	Triptolide-Containing Formulation as Treatment for Autosomal Dominant Polycystic Kidney Disease (ADPKD)
NCT02134899	PHASE3	COMPLETED	The Efficacy of Everolimus in Reducing Total Native Kidney Volume in Polycystic Kidney Disease Transplanted Recipients
NCT02160145	PHASE3	COMPLETED	Efficacy and Safety of Tolvaptan in Subjects With Chronic Kidney Disease Between Late Stage 2 to Early Stage 4 Due to Autosomal Dominant Polycystic Kidney Disease
NCT02964273	PHASE3	COMPLETED	Safety, Pharmacokinetics, Tolerability and Efficacy of Tolvaptan in Children and Adolescents With ADPKD (Autosomal Dominant Polycystic Kidney Disease)
NCT03764605	PHASE3	UNKNOWN	Metformin vs Tolvaptan for Treatment of Autosomal Dominant Polycystic Kidney Disease
NCT03918447	PHASE3	TERMINATED	A Trial of Bardoxolone Methyl in Patients With ADPKD - FALCON
NCT04064346	PHASE3	TERMINATED	Efficacy and Safety of Lixivaptan in the Treatment of Autosomal Dominant Polycystic Kidney Disease
NCT04152837	PHASE3	TERMINATED	Safety of Lixivaptan in Subjects Previously Treated With Tolvaptan for Autosomal Dominant Polycystic Kidney Disease
NCT04939935	PHASE3	RECRUITING	Implementation of Metformin theraPy to Ease Decline of Kidney Function in Polycystic Kidney Disease (IMPEDE-PKD)
NCT05373264	PHASE3	RECRUITING	HYDROchlorothiazide to PROTECT Polycystic Kidney Disease Patients and Improve Their Quality of Life
NCT00673335	PHASE3	COMPLETED	Letrozole in Preventing Breast Cancer in Postmenopausal Women With a BRCA1 or BRCA2 Mutation
NCT00685256	PHASE3	COMPLETED	Standard Genetic Counseling With or Without a Decision Guide in Improving Communication Between Mothers Undergoing BRCA1/2 Testing and Their Minor-Age Children
NCT03162276	PHASE3	UNKNOWN	Trial of Inquiry Based Stress Reduction (IBSR) Program for BRCA1/2 Mutation Carriers
NCT00841568	PHASE2	COMPLETED	A Long-term Administration Study of OPC-41061 in Patients With Autosomal Dominant Polycystic Kidney Disease (ADPKD) [Extension of Study 156-04-001]
NCT01210560	PHASE2	COMPLETED	Dose-finding Study of New Tolvaptan Formulation in Subjects With ADPKD
NCT01336972	PHASE2	COMPLETED	Short-term Renal Hemodynamic Effects of Tolvaptan in Subjects With Autosomal Dominant Polycystic Kidney Disease (ADPKD)
NCT01451827	PHASE2	COMPLETED	8-Week Study of Tolvaptan Dose Forms in Autosomal Dominant Polycystic Kidney Disease (ADPKD)
NCT01670110	PHASE2	COMPLETED	Pasireotide LAR in Severe Polycystic Liver Disease
NCT01932450	PHASE2	UNKNOWN	Radiofrequency Ablation for ADPKD Blood Pressure and Disease Progression Control
NCT02527863	PHASE2	COMPLETED	Effect of the Aquaretic Tolvaptan on Nitric Oxide System
NCT02616055	PHASE2	TERMINATED	Long-Term Treatment and Follow up of Subjects Completing 24 Months of Treatment With Tesevatinib on Study KD019-101
NCT03203642	PHASE2	COMPLETED	Study of the Efficacy and Safety of Tesevatinib in Subjects With ADPKD
NCT03487913	PHASE2	COMPLETED	The ELiSA Study - Evaluation of Lixivaptan in Subjects With Autosomal Dominant Polycystic Kidney Disease
NCT03541447	PHASE2	COMPLETED	Tolvaptan-Octreotide LAR Combination in ADPKD
NCT04284657	PHASE2	COMPLETED	Pravastatin and Alkali Therapy in Patients With Autosomal Dominant Polycystic Kidney Disease
NCT04578548	PHASE2	TERMINATED	A Study to Evaluate the Effects of GLPG2737 in Participants With Autosomal Dominant Polycystic Kidney Disease (ADPKD)
NCT05190744	PHASE2	COMPLETED	Probenecid (PB) to Treat Hereditary Nephrogenic Diabetes Insipidus (NDI), ADPKD Treated With Tolvaptan, and Severely Polyuric Patients With Previous Lithium Administration
NCT05870007	PHASE2	ENROLLING_BY_INVITATION	Atorvastatin and Alkali Therapy in Patients With Autosomal Dominant Polycystic Kidney Disease