Biomarker & Intervention Discovery

Primary Sclerosing Cholangitis (PSC)

This page lists 10 druggable gene targets, 47 approved or trial-tested drug candidates and 15 recent clinical trials for Primary Sclerosing Cholangitis (PSC), with each agent ranked by the strength of its evidence (clinical, mechanistic or correlative). Candidates include existing drugs that could be repurposed. Sources are linked so you can check every claim.

Spontaneous remissionProgressive cholestatic disease; median time to liver failure or transplant 12-15 years; spontaneous remission…
Best-intervention remissionLiver transplantation: 5-year survival 80-85% (curative but recurrence in 20% post-transplant); ursodeoxycholic acid…
Gap sizeVery large — no approved disease-modifying therapy exists; liver transplantation is the only definitive treatment
Primary barrierbiological
Full remission profile →
Research Tracker Chronic Disease Interventions Primary Sclerosing Cholangitis (PSC)

Remission & chronicity

Spontaneous remission
Progressive cholestatic disease; median time to liver failure or transplant 12-15 years; spontaneous remission essentially zero; biliary strictures progress in majority; cholangiocarcinoma risk 10-15% lifetime
Best-intervention remission
Liver transplantation: 5-year survival 80-85% (curative but recurrence in 20% post-transplant); ursodeoxycholic acid (UDCA): improvement in LFTs but no transplant-free survival benefit (Phase 3 — no benefit, possibly harm at high dose); no pharmacotherapy proven to alter the natural history
Gap size
Very large — no approved disease-modifying therapy exists; liver transplantation is the only definitive treatment
Primary barrier
biological
PSC has resisted all pharmacotherapy attempts; UDCA widely prescribed despite no survival benefit (at high doses — possibly harmful); the field has 20+ failed Phase 2/3 trials; norUDCA, nor-UDCA, and several FXR agonists are in Phase 3; gut-liver axis and gut microbiome increasingly implicated (80% of PSC patients have concurrent IBD)

Biomarker Targets & Therapeutic Agents

Gene targets queried against DGIdb, Open Targets, ChEMBL, PubMed, and Europe PMC. Agents ranked: Clinical > Mechanistic > Correlative.

AOC3 201CCR2 175CCR5 182CYP7A1 5FGF19 2LOXL2 122NR1H4 228PPARA 219PPARD 299SLC10A2 206

AOC3

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
PHENELZINE ↓ inhibits Mechanistic 19.95 nM (IC50) DGIdb
VEPALIMOMAB ↓ inhibits Mechanistic DGIdb
PXS-4681A ↓ inhibits Mechanistic DGIdb
TIMOLUMAB ? unclear Mechanistic DGIdb
CLONIDINE HYDROCHLORIDE ? unclear Mechanistic DGIdb
HYDRALAZINE ? unclear Mechanistic DGIdb
PXS-4728A ↓ inhibits Mechanistic DGIdb
MOFEGILINE ↓ inhibits Mechanistic 1610.0 nM (Ki) DGIdb
CHEMBL534914 ↓ inhibits Mechanistic 8000.0 nM (IC50) ChEMBL
CHEMBL537624 ↓ inhibits Mechanistic 2000.0 nM (IC50) ChEMBL
CHEMBL537175 ↓ inhibits Mechanistic 100000.0 nM (IC50) ChEMBL
CHEMBL537402 ↓ inhibits Mechanistic 16000.0 nM (IC50) ChEMBL
CHEMBL557160 ↓ inhibits Mechanistic 56000.0 nM (IC50) ChEMBL
CHEMBL537628 ↓ inhibits Mechanistic 79000.0 nM (IC50) ChEMBL
CHEMBL541126 ↓ inhibits Mechanistic 5600.0 nM (IC50) ChEMBL
CHEMBL559376 ↓ inhibits Mechanistic 100000.0 nM (IC50) ChEMBL
CHEMBL537401 ↓ inhibits Mechanistic 170.0 nM (IC50) ChEMBL
CHEMBL536719 ↓ inhibits Mechanistic 3200.0 nM (IC50) ChEMBL
CHEMBL536484 ↓ inhibits Mechanistic 150.0 nM (IC50) ChEMBL
CHEMBL536951 ↓ inhibits Mechanistic 5000.0 nM (IC50) ChEMBL
CHEMBL538353 ↓ inhibits Mechanistic 130.0 nM (IC50) ChEMBL
CHEMBL539362 ↓ inhibits Mechanistic 1600.0 nM (IC50) ChEMBL
CHEMBL537845 ↓ inhibits Mechanistic 1700.0 nM (IC50) ChEMBL
CHEMBL534467 ↓ inhibits Mechanistic 440.0 nM (IC50) ChEMBL
CHEMBL537623 ↓ inhibits Mechanistic 20.0 nM (IC50) ChEMBL
CHEMBL536953 ↓ inhibits Mechanistic 16000.0 nM (IC50) ChEMBL
CHEMBL1243019 ↓ inhibits Mechanistic 389.05 nM (IC50) ChEMBL
CHEMBL1241430 ↓ inhibits Mechanistic 346.74 nM (IC50) ChEMBL
CHEMBL1241429 ↓ inhibits Mechanistic 467.74 nM (IC50) ChEMBL
CHEMBL1241428 ↓ inhibits Mechanistic 58.88 nM (IC50) ChEMBL
CHEMBL1241379 ↓ inhibits Mechanistic 269.15 nM (IC50) ChEMBL
CHEMBL1241378 ↓ inhibits Mechanistic 229.09 nM (IC50) ChEMBL
CHEMBL1241377 ↓ inhibits Mechanistic 398.11 nM (IC50) ChEMBL
CHEMBL1241376 ↓ inhibits Mechanistic 630.96 nM (IC50) ChEMBL
CHEMBL1241332 ↓ inhibits Mechanistic 416.87 nM (IC50) ChEMBL
CHEMBL1241331 ↓ inhibits Mechanistic 407.38 nM (IC50) ChEMBL
CHEMBL1241330 ↓ inhibits Mechanistic 323.59 nM (IC50) ChEMBL
CHEMBL1241329 ↓ inhibits Mechanistic 426.58 nM (IC50) ChEMBL
CHEMBL1241293 ↓ inhibits Mechanistic 407.38 nM (IC50) ChEMBL
CHEMBL1241292 ↓ inhibits Mechanistic 575.44 nM (IC50) ChEMBL

Showing top 40 of 201 agents ranked by evidence tier.

CCR2

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
[125I]CCL7 (HUMAN) ↑ activates Mechanistic DGIdb
BMS-741672 ↓ inhibits Mechanistic DGIdb
BMS-813160 ? unclear Mechanistic DGIdb
RS-136270 ↓ inhibits Mechanistic DGIdb
RECOMBINANT MONOCYTE CHEMOATTRACTANT PROTEIN-1 ? unclear Mechanistic DGIdb
BMS-681 ↓ inhibits Mechanistic DGIdb
HIV-TAT ↑ activates Mechanistic DGIdb
PLOZALIZUMAB ? unclear Mechanistic DGIdb
MORPHINE SULFATE ? unclear Mechanistic DGIdb
INCB8761 ↓ inhibits Mechanistic DGIdb
CCR2-RA-[R] ~ modulates Mechanistic DGIdb
CHEMBL:CHEMBL41275 ? unclear Mechanistic DGIdb
VMIP-II ↓ inhibits Mechanistic DGIdb
RS-504393 ↓ inhibits Mechanistic DGIdb
SB-282241 ↓ inhibits Mechanistic DGIdb
CCL11 ↑ activates Mechanistic DGIdb
VCCL4 ↑ activates Mechanistic DGIdb
ILACIRNON ↓ inhibits Mechanistic DGIdb
ALITRETINOIN ? unclear Mechanistic DGIdb
CCL8 ↑ activates Mechanistic DGIdb
PF-04634817 ↓ inhibits Mechanistic DGIdb
CENICRIVIROC ↓ inhibits Mechanistic DGIdb
OPIATE ? unclear Mechanistic DGIdb
PLERIXAFOR ? unclear Mechanistic DGIdb
MK-0812 ? unclear Mechanistic DGIdb
RS-102895 ↓ inhibits Mechanistic DGIdb
COMPOUND 39 [PMID: 31742400] ~ modulates Mechanistic DGIdb
JNJ-41443532 ↓ inhibits Mechanistic DGIdb
CCL24 ↓ inhibits Mechanistic DGIdb
CCL16 ↑ activates Mechanistic DGIdb
CCL2 ↑ activates Mechanistic DGIdb
GSK COMPOUND 34 ↓ inhibits Mechanistic DGIdb
TEIJIN-LEAD_CMP_5 ↓ inhibits Mechanistic DGIdb
SIMVASTATIN ? unclear Mechanistic DGIdb
CCL26 ↓ inhibits Mechanistic DGIdb
MC148R ↓ inhibits Mechanistic DGIdb
CCL13 ↑ activates Mechanistic DGIdb
JNJ-27141491 ~ modulates Mechanistic DGIdb
AZD2423 ~ modulates Mechanistic DGIdb
CCL7 ↑ activates Mechanistic DGIdb

Showing top 40 of 175 agents ranked by evidence tier.

CCR5

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
[3H]ANCRIVIROC ↓ inhibits Mechanistic DGIdb
[3H]MARAVIROC ↓ inhibits Mechanistic DGIdb
CCL16 ↑ activates Mechanistic DGIdb
[125I]CCL4 (HUMAN) ↑ activates Mechanistic DGIdb
CCL11 ↑ activates Mechanistic DGIdb
BP-CCL3 ↑ activates Mechanistic DGIdb
[125I]CCL5 (HUMAN) ↑ activates Mechanistic DGIdb
BMS-753426 ↓ inhibits Mechanistic DGIdb
CENICRIVIROC ↓ inhibits Mechanistic DGIdb
MARAVIROC ↓ inhibits Mechanistic DGIdb
TAK-220 ↓ inhibits Mechanistic DGIdb
VMIP-II ↓ inhibits Mechanistic DGIdb
GLATIRAMER ? unclear Mechanistic DGIdb
CCR5 MAB ? unclear Mechanistic DGIdb
LERONLIMAB ? unclear Mechanistic DGIdb
MLN-3897 ↓ inhibits Mechanistic DGIdb
PF-232798 ↓ inhibits Mechanistic DGIdb
[125I]CCL3 (HUMAN) ↑ activates Mechanistic DGIdb
COMPOUND 39 [PMID: 31742400] ~ modulates Mechanistic DGIdb
CCL13 ↑ activates Mechanistic DGIdb
APLAVIROC HYDROCHLORIDE ↓ inhibits Mechanistic DGIdb
CCL7 ↓ inhibits Mechanistic DGIdb
BMS-813160 ? unclear Mechanistic DGIdb
VICRIVIROC ↓ inhibits Mechanistic 2.1 nM (Ki) DGIdb
E913 ↓ inhibits Mechanistic DGIdb
CCL14 ↑ activates Mechanistic DGIdb
CCL3 ↑ activates Mechanistic DGIdb
VICRIVIROC MALEATE ↓ inhibits Mechanistic DGIdb
CCL5 ↑ activates Mechanistic DGIdb
INCB-9471 ↓ inhibits Mechanistic DGIdb
BMS-681 ↓ inhibits Mechanistic DGIdb
HGS-1025 ↓ inhibits Mechanistic DGIdb
FLU-CCL3 ↑ activates Mechanistic DGIdb
PF-04634817 ↓ inhibits Mechanistic DGIdb
BMS-741672 ↓ inhibits Mechanistic DGIdb
CCL8 ↑ activates Mechanistic DGIdb
CENICRIVIROC MESYLATE ? unclear Mechanistic DGIdb
[125I]CCL8 (HUMAN) ↑ activates Mechanistic DGIdb
APLAVIROC ↓ inhibits Mechanistic DGIdb
CCR5MAB004 ↓ inhibits Mechanistic DGIdb

Showing top 40 of 182 agents ranked by evidence tier.

CYP7A1

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
CHOLIC ACID ? unclear Mechanistic DGIdb
(2S,4S)-KETOCONAZOLE ↓ inhibits Mechanistic DGIdb
CHEMBL4288256 ↓ inhibits Mechanistic 1370.0 nM (IC50) ChEMBL
CHEMBL4289930 ↓ inhibits Mechanistic 880.0 nM (IC50) ChEMBL
CHEMBL5574621 ↓ inhibits Mechanistic 2100.0 nM (Kd) ChEMBL

FGF19

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
FGFR4 INHIBITOR H3B-6527 ? unclear Mechanistic DGIdb
FISOGATINIB ? unclear Mechanistic DGIdb

LOXL2

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
COMPOUND 21B [PMID: 31430136] ↓ inhibits Mechanistic DGIdb
SIMTUZUMAB ↓ inhibits Mechanistic DGIdb
LENUMLOSTAT ↓ inhibits Mechanistic DGIdb
CCT365623 ↓ inhibits Mechanistic DGIdb
CHEMBL4068784 ↓ inhibits Mechanistic 190.0 nM (IC50) ChEMBL
CHEMBL1618272 ↓ inhibits Mechanistic 83.0 nM (IC50) ChEMBL
CHEMBL4072524 ↓ inhibits Mechanistic 321.0 nM (IC50) ChEMBL
CHEMBL4099444 ↓ inhibits Mechanistic 203.0 nM (IC50) ChEMBL
CHEMBL3467241 ↓ inhibits Mechanistic 309.0 nM (IC50) ChEMBL
CHEMBL4066086 ↓ inhibits Mechanistic 1180.0 nM (IC50) ChEMBL
CHEMBL4079586 ↓ inhibits Mechanistic 431.0 nM (IC50) ChEMBL
CHEMBL4103840 ↓ inhibits Mechanistic 385.0 nM (IC50) ChEMBL
CHEMBL4096085 ↓ inhibits Mechanistic 999.0 nM (IC50) ChEMBL
CHEMBL4060123 ↓ inhibits Mechanistic 6040.0 nM (IC50) ChEMBL
CHEMBL4088360 ↓ inhibits Mechanistic 8210.0 nM (IC50) ChEMBL
CHEMBL1188378 ↓ inhibits Mechanistic 1220.0 nM (IC50) ChEMBL
CHEMBL4075843 ↓ inhibits Mechanistic 902.0 nM (IC50) ChEMBL
CHEMBL4098510 ↓ inhibits Mechanistic 4970.0 nM (IC50) ChEMBL
CHEMBL187301 ↓ inhibits Mechanistic 1110.0 nM (IC50) ChEMBL
CHEMBL1778128 ↓ inhibits Mechanistic 3540.0 nM (IC50) ChEMBL
CHEMBL148986 ↓ inhibits Mechanistic 3620.0 nM (IC50) ChEMBL
CHEMBL4068773 ↓ inhibits Mechanistic 670.0 nM (IC50) ChEMBL
CHEMBL1235374 ↓ inhibits Mechanistic 719.0 nM (IC50) ChEMBL
CHEMBL12392 ↓ inhibits Mechanistic 26000.0 nM (IC50) ChEMBL
CHEMBL148476 ↓ inhibits Mechanistic 47000.0 nM (IC50) ChEMBL
CHEMBL4098672 ↓ inhibits Mechanistic 35000.0 nM (IC50) ChEMBL
BENZYLAMINE ↓ inhibits Mechanistic 64700.0 nM (IC50) ChEMBL
PHENETHYLAMINE ↓ inhibits Mechanistic 60100.0 nM (IC50) ChEMBL
CHEMBL4071963 ↓ inhibits Mechanistic 110.0 nM (IC50) ChEMBL
CHEMBL4092740 ↓ inhibits Mechanistic 82.0 nM (IC50) ChEMBL
CHEMBL4063337 ↓ inhibits Mechanistic 74.0 nM (IC50) ChEMBL
CHEMBL4084982 ↓ inhibits Mechanistic 35.0 nM (IC50) ChEMBL
CHEMBL4100783 ↓ inhibits Mechanistic 77.0 nM (IC50) ChEMBL
CHEMBL4082808 ↓ inhibits Mechanistic 43.0 nM (IC50) ChEMBL
CHEMBL4097435 ↓ inhibits Mechanistic 58.0 nM (IC50) ChEMBL
CHEMBL4079398 ↓ inhibits Mechanistic 78.0 nM (IC50) ChEMBL
CHEMBL4105137 ↓ inhibits Mechanistic 96.0 nM (IC50) ChEMBL
CHEMBL4087273 ↓ inhibits Mechanistic 130.0 nM (IC50) ChEMBL
CHEMBL4081837 ↓ inhibits Mechanistic 570.0 nM (IC50) ChEMBL
CHEMBL4059919 ↓ inhibits Mechanistic 42.0 nM (IC50) ChEMBL

Showing top 40 of 122 agents ranked by evidence tier.

NR1H4

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
SAQUINAVIR MESYLATE ? unclear Mechanistic DGIdb
DIFENOCONAZOLE ? unclear Mechanistic DGIdb
CONICASTEROL D ? unclear Mechanistic DGIdb
FLUAZINAM ? unclear Mechanistic DGIdb
DY268 ↓ inhibits Mechanistic DGIdb
TUBERATOLIDE B ? unclear Mechanistic DGIdb
METHYLENE BLUE ? unclear Mechanistic DGIdb
GENISTEIN ? unclear Mechanistic DGIdb
PENTACHLOROPHENOL ? unclear Mechanistic DGIdb
COMPOUND 3F [FESTA ET AL., 2019] ↓ inhibits Mechanistic DGIdb
PHENOLPHTHALEIN ? unclear Mechanistic DGIdb
LITHOCHOLIC ACID ? unclear Mechanistic 20000.0 nM (EC50) DGIdb
CHEMBL:CHEMBL476302 ? unclear Mechanistic DGIdb
GANODERMANONTRIOL ? unclear Mechanistic DGIdb
ERGOSTEROL PEROXIDE ? unclear Mechanistic DGIdb
TROPIFEXOR ↑ activates Mechanistic DGIdb
DOXORUBICIN HYDROCHLORIDE ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL184450 ? unclear Mechanistic DGIdb
22R-HYDROXYCHOLESTEROL ↑ activates Mechanistic DGIdb
COMPOUND 25 [PMID: 32687365] ↑ activates Mechanistic DGIdb
TUROFEXORATE ISOPROPYL ↑ activates Mechanistic DGIdb
LEVOKETOCONAZOLE ? unclear Mechanistic DGIdb
CONICASTEROL B ? unclear Mechanistic DGIdb
CRYPTOCHINONE B ? unclear Mechanistic DGIdb
SUVANINE'S SODIUM SALT ? unclear Mechanistic DGIdb
TUBERATOLIDE A ? unclear Mechanistic DGIdb
CHENODEOXYCHOLIC ACID ↑ activates Mechanistic DGIdb
CRYPTOCHINONE A ? unclear Mechanistic DGIdb
TERN-101 ↑ activates Mechanistic DGIdb
APOMINE ? unclear Mechanistic DGIdb
ZEARALENONE ? unclear Mechanistic DGIdb
GUGGULSTERONE ↓ inhibits Mechanistic 100000.0 nM (IC50) DGIdb
PX-102 ↑ activates Mechanistic DGIdb
BETA SITOSTEROL ↑ activates Mechanistic DGIdb
BMS-986339 ↑ activates Mechanistic DGIdb
OBETICHOLIC ACID ↑ activates Mechanistic 99.0 nM (EC50) DGIdb
NIDUFEXOR ↑ activates Mechanistic DGIdb
THEONELLASTEROL E ? unclear Mechanistic DGIdb
TRIBUTYLCHLOROSTANNANE ? unclear Mechanistic DGIdb
(R)-SARGACHROMENOL ? unclear Mechanistic DGIdb

Showing top 40 of 228 agents ranked by evidence tier.

PPARA

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
FENOFIBRATE MICRONIZED ↑ activates Mechanistic DGIdb
BEZAFIBRATE ↑ activates Mechanistic 50000.0 nM (EC50) DGIdb
LANIFIBRANOR ↑ activates Mechanistic DGIdb
CIPROFIBRATE ↑ activates Mechanistic DGIdb
AD-5061 ↑ activates Mechanistic DGIdb
MURAGLITAZAR ↑ activates Mechanistic DGIdb
N-OLEOYLETHANOLAMIDE ↑ activates Mechanistic DGIdb
PHENFORMIN HYDROCHLORIDE ? unclear Mechanistic DGIdb
MK-0767 ↑ activates Mechanistic DGIdb
ALECTINIB ? unclear Mechanistic DGIdb
GW590735 ↑ activates Mechanistic DGIdb
IMIGLITAZAR ↑ activates Mechanistic DGIdb
INDEGLITAZAR ↑ activates Mechanistic DGIdb
K-877 ↑ activates Mechanistic DGIdb
GEMFIBROZIL ↑ activates Mechanistic DGIdb
LY-465608 ↑ activates Mechanistic DGIdb
ILOPROST ? unclear Mechanistic DGIdb
BROMOXYNIL ? unclear Mechanistic DGIdb
RAGAGLITAZAR ↑ activates Mechanistic 3210.0 nM (EC50) DGIdb
CHEMBL:CHEMBL292352 ? unclear Mechanistic DGIdb
GFT14 ? unclear Mechanistic DGIdb
SAROGLITAZAR ↑ activates Mechanistic DGIdb
GW409544 ↑ activates Mechanistic DGIdb
ELAFIBRANOR ↑ activates Mechanistic DGIdb
SELADELPAR ↑ activates Mechanistic DGIdb
CHEMBL:CHEMBL259327 ? unclear Mechanistic DGIdb
ROMAZARIT ? unclear Mechanistic DGIdb
CLOFIBRATE ↑ activates Mechanistic 55000.0 nM (EC50) DGIdb
PIRINIXIC ACID ↑ activates Mechanistic 5000.0 nM (EC50) DGIdb
TESAGLITAZAR ? unclear Mechanistic DGIdb
CHOLINE FENOFIBRATE ↑ activates Mechanistic DGIdb
GW9578 ↑ activates Mechanistic DGIdb
NAVEGLITAZAR ? unclear Mechanistic DGIdb
GW7647 ↑ activates Mechanistic DGIdb
AZD4619 ↑ activates Mechanistic DGIdb
GW501516 ? unclear Mechanistic DGIdb
GW6471 ↓ inhibits Mechanistic DGIdb
LY-510929 ↑ activates Mechanistic 28.0 nM (IC50) DGIdb
CHIGLITAZAR ? unclear Mechanistic DGIdb
PEMAFIBRATE ↑ activates Mechanistic DGIdb

Showing top 40 of 219 agents ranked by evidence tier.

PPARD

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
CISPLATIN ? unclear Mechanistic DGIdb
MBX-8025 ? unclear Mechanistic DGIdb
ZIRAM ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL594772 ? unclear Mechanistic DGIdb
ISOCONAZOLE ? unclear Mechanistic DGIdb
GW7647 ? unclear Mechanistic 6200.0 nM (EC50) DGIdb
ENCLOMIPHENE ? unclear Mechanistic DGIdb
CINACALCET HYDROCHLORIDE ? unclear Mechanistic DGIdb
ABAMECTIN ? unclear Mechanistic DGIdb
COMPOUND 25 [PMID: 32687365] ↑ activates Mechanistic DGIdb
CER-002 ↑ activates Mechanistic DGIdb
DEHYDRATED ALCOHOL ? unclear Mechanistic DGIdb
RO3280 ? unclear Mechanistic DGIdb
TRICLOCARBAN ? unclear Mechanistic DGIdb
PROPRANOLOL HYDROCHLORIDE ? unclear Mechanistic DGIdb
MITOXANTRONE HYDROCHLORIDE ? unclear Mechanistic DGIdb
IDARUBICIN HYDROCHLORIDE ? unclear Mechanistic DGIdb
FONADELPAR ↑ activates Mechanistic DGIdb
CHEMBL:CHEMBL598608 ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL172988 ? unclear Mechanistic DGIdb
(R)-16 [PMID: 32267688] ↑ activates Mechanistic DGIdb
CHEMBL:CHEMBL333177 ? unclear Mechanistic DGIdb
ORYZALIN ? unclear Mechanistic DGIdb
FLUSPIRILENE ? unclear Mechanistic DGIdb
GOSSYPOL ? unclear Mechanistic DGIdb
BIFENAZATE ? unclear Mechanistic DGIdb
{4-[3-(4-ACETYL-3-HYDROXY-2-PROPYLPHENOXY)PROPOXY]PHENOXY}ACETIC ACID ↑ activates Mechanistic DGIdb
ETOXAZOLE ? unclear Mechanistic DGIdb
DOCETAXEL ANHYDROUS ? unclear Mechanistic DGIdb
OCHRATOXIN A ? unclear Mechanistic DGIdb
GW501516 ↑ activates Mechanistic DGIdb
BAZEDOXIFENE ACETATE ? unclear Mechanistic DGIdb
CAPECITABINE ? unclear Mechanistic DGIdb
PIMOZIDE ? unclear Mechanistic DGIdb
RALOXIFENE HYDROCHLORIDE ? unclear Mechanistic DGIdb
DIFENOCONAZOLE ? unclear Mechanistic DGIdb
THIOSEMICARBAZIDE ? unclear Mechanistic DGIdb
LANIFIBRANOR ↑ activates Mechanistic DGIdb
MURAGLITAZAR ? unclear Mechanistic DGIdb
PHENYLMERCURIC ACETATE ? unclear Mechanistic DGIdb

Showing top 40 of 299 agents ranked by evidence tier.

SLC10A2

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
ELOBIXIBAT ↓ inhibits Mechanistic DGIdb
DOCETAXEL ANHYDROUS ? unclear Mechanistic DGIdb
SC-435 ↓ inhibits Mechanistic DGIdb
MARALIXIBAT CHLORIDE ↓ inhibits Mechanistic 890.0 nM (IC50) DGIdb
264W94 ↓ inhibits Mechanistic DGIdb
THALIDOMIDE ? unclear Mechanistic DGIdb
[3H]TAUROCHOLIC ACID ? unclear Mechanistic DGIdb
ODEVIXIBAT ↓ inhibits Mechanistic DGIdb
VOLIXIBAT ↓ inhibits Mechanistic DGIdb
MARALIXIBAT ↓ inhibits Mechanistic 0.28 nM (IC50) DGIdb
ODEVIXIBAT SESQUIHYDRATE ↓ inhibits Mechanistic DGIdb
LINERIXIBAT ↓ inhibits Mechanistic DGIdb
TAUROCHOLIC ACID ? unclear Mechanistic 41500.0 nM (Ki) DGIdb
CHEMBL17614 ↓ inhibits Mechanistic 2800.0 nM (IC50) ChEMBL
CHEMBL17966 ↓ inhibits Mechanistic 770.0 nM (IC50) ChEMBL
CHEMBL17687 ↓ inhibits Mechanistic 1070.0 nM (IC50) ChEMBL
CHEMBL332974 ↓ inhibits Mechanistic 1.6 nM (IC50) ChEMBL
CHEMBL123319 ↓ inhibits Mechanistic 580.0 nM (IC50) ChEMBL
CHEMBL122745 ↓ inhibits Mechanistic 44.0 nM (IC50) ChEMBL
CHEMBL123444 ↓ inhibits Mechanistic 690.0 nM (IC50) ChEMBL
CHEMBL332370 ↓ inhibits Mechanistic 48.0 nM (IC50) ChEMBL
CHEMBL340291 ↓ inhibits Mechanistic 22.0 nM (IC50) ChEMBL
CHEMBL125997 ↓ inhibits Mechanistic 26.0 nM (IC50) ChEMBL
CHEMBL332606 ↓ inhibits Mechanistic 2500.0 nM (IC50) ChEMBL
CHEMBL434814 ↓ inhibits Mechanistic 200.0 nM (IC50) ChEMBL
CHEMBL124715 ↓ inhibits Mechanistic 570.0 nM (IC50) ChEMBL
CHEMBL124239 ↓ inhibits Mechanistic 970.0 nM (IC50) ChEMBL
CHEMBL332973 ↓ inhibits Mechanistic 3.0 nM (IC50) ChEMBL
CHEMBL331235 ↓ inhibits Mechanistic 1200.0 nM (IC50) ChEMBL
CHEMBL331631 ↓ inhibits Mechanistic 320.0 nM (IC50) ChEMBL
CHEMBL331910 ↓ inhibits Mechanistic 310.0 nM (IC50) ChEMBL
CHEMBL123542 ↓ inhibits Mechanistic 1200.0 nM (IC50) ChEMBL
CHEMBL341104 ↓ inhibits Mechanistic 8.0 nM (IC50) ChEMBL
CHEMBL122229 ↓ inhibits Mechanistic 83.0 nM (IC50) ChEMBL
CHEMBL341082 ↓ inhibits Mechanistic 35.0 nM (IC50) ChEMBL
CHEMBL122498 ↓ inhibits Mechanistic 6.0 nM (IC50) ChEMBL
CHEMBL331015 ↓ inhibits Mechanistic 320.0 nM (IC50) ChEMBL
CHEMBL96648 ↓ inhibits Mechanistic 4400.0 nM (IC50) ChEMBL
CHEMBL96642 ↓ inhibits Mechanistic 7100.0 nM (IC50) ChEMBL
CHEMBL96145 ↓ inhibits Mechanistic 13300.0 nM (IC50) ChEMBL

Showing top 40 of 206 agents ranked by evidence tier.

Clinical Trials

Drug, biologic and supplement trials (treatment or prevention) from ClinicalTrials.gov whose main condition is Primary Sclerosing Cholangitis (PSC): recruiting, active or completed, most recently updated first. Data retrieved 2026-10-28.

A Study to Assess Safety and Effectiveness of Elafibranor in Adult Participants With Primary Sclerosing Cholangitis. Active Not Recruiting
NCT05627362 Phase: 2 Started: 2022-12-29

This study will evaluate the effects of elafibranor (the study drug) in participants with Primary Sclerosing Cholangitis (PSC) during the double-blind period, an initial 96 week open-label extension (OLE) period, and an optional open-label extension long-term (OLE-LT) period beyond OLE Week 96. PSC ...

A Study to Assess How Well and Safely Elafibranor Works in Adult Participants With Primary Sclerosing Cholangitis Recruiting
NCT07387549 Phase: 3 Started: 2026-05-14

The purpose of this study is to find out how well and safely elafibranor works compared to placebo in adult participants with Primary sclerosing cholangitis (PSC). PSC is a rare disease that causes inflammation and scarring of the bile ducts in the liver. Over time, this can lead to liver damage and...

A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Sclerosing Cholangitis (PSC) Active Not Recruiting
NCT04663308 Phase: 2 Started: 2020-12-18

The purpose of this clinical research study is to learn more about the use of the study medicine, volixibat, for the treatment of pruritus (itching) associated with Primary Sclerosing Cholangitis (PSC), and to assess the possible impact on the disease progression of PSC.

A POC and Dose-Ranging Study of HTD1801 in PSC Patients Completed
NCT03333928 Phase: 2 Started: 2018-02-09

The study was a dose-ranging, 18-week study comparing two doses of HTD1801 (500 mg BID and 1000 mg BID) to placebo in adult subjects with PSC.

S-adenosylmethionine (SAMe) in Patients With Primary Sclerosing Cholangitis (PSC) Completed
NCT06026865 Phase: N/A Started: 2023-09-01

The aim of this study is to investigate clinical effects (liver biochemistries, health-related quality of life, liver stiffness) and underlying mechanisms of hepatoprotection of S-adenosylmethionine in patients with primary sclerosing cholangitis. The study will be performed in a randomized and plac...

A Study of HTD1801 in Healthy Subjects Completed
NCT03099603 Phase: 1 Started: 2017-03-24

This is a randomized, double blind, single center, ascending single dose study to evaluate the safety, tolerability, and PK of HTD1801.

Detoxification of the Liver In PSC (Dolphin) Recruiting
NCT05835505 Phase: 2 Started: 2023-12-12

This study is a clinical trial being done to investigate the efficacy of drug BRS201 (hydroxocobalamin) as a treatment in patients with primary sclerosing cholangitis. Participation in this study will take 8 weeks long and the study is structured as a cross-over study in which participants will take...

Pharmacologic Approaches to Preventing Primary Sclerosing Cholangitis Recurrence After Liver Transplantation Recruiting
NCT06905054 Phase: 2 Started: 2025-04-15

This study aims to determine the efficacy of 36 months once-daily fenofibrate in preventing clinically-detectable recurrence of primary sclerosing cholangitis after liver transplantation, compared with a historical control cohort that was not treated with

CM-101 in PSC Patients -The SPRING Study Completed
NCT04595825 Phase: 2 Started: 2020-10-01

This study is designed to assess the safety, tolerability and activity of the anti-human CCL24 monoclonal antibody CM-101 in adult subjects with Primary Sclerosing Cholangitis (PSC). At least 68 subjects at approximately 50 sites will be randomized to receive either CM-101 at doses of 10 mg/kg or 20...

Evaluation of an Oral Microbiota-based Therapeutic as a Treatment Option for PSC Recruiting
NCT06197308 Phase: Early 1 Started: 2024-03-01

This pilot clinical trial will evaluate the initial safety and feasibility of microbiota transplant therapy (MTT) inpatients with primary sclerosing cholangitis (PSC). This trial will inform development of future trials in treatment of PSC.

CS0159 in Chinese Patients With PSC (Primary Sclerosing Cholangitis) Active Not Recruiting
NCT05896137 Phase: 2 Started: 2023-08-07

A phase II Study of CS0159 in Chinese patients with PSC(Primary Sclerosing Cholangitis)

Sulfasalazine for the Treatment of Primary Sclerosing Cholangitis Completed
NCT03561584 Phase: 2 Started: 2018-07-01

This is a multicenter, randomized, double-blinded placebo controlled trial to assess the benefit of sulfasalazine in the treatment of PSC. The specific objectives of this study are to determine if sulfasalazine treatment 1) results in reduced serum ALP and other biomarkers of liver injury in PSC; 2)...

Phase 2a Evaluation of Safety, Tolerability, and Pharmacokinetics of PLN-74809 in Patients With Primary Sclerosing Cholangitis (PSC) Completed
NCT04480840 Phase: 2 Started: 2020-07-27

A Phase 2a, multicenter, randomized, double-blind, dose-ranging, placebo-controlled, study to evaluate the safety, tolerability, and PK of PLN-74809 in participants with primary sclerosing cholangitis and suspected liver fibrosis

A Study to Assess the Safety and Efficacy of LB-P8 in Patients With PSC Recruiting
NCT06699121 Phase: 2 Started: 2025-11

The study is designed to assess the safety and efficacy of LB-P8 in patients with primary sclerosing cholangitis.

Effect of Simvastatin on the Prognosis of Primary Primary Sclerosing Cholangitis (PSC) Active Not Recruiting
NCT04133792 Phase: 3 Started: 2020-10-01

This is a randomized, double-blind, placebo controlled multicenter study. A total of 700 patients will be included. After an updated powercalculation 560 was condidered enough The study will include patients with primary sclerosing cholangitis (PSC) for daily intake of 40 mg simvastatin/placebo for ...

Agents Found By Disease-Level Search

Searched directly from “Primary Sclerosing Cholangitis (PSC)” via ClinicalTrials.gov interventions and Open Targets' disease→drug data — independent of the 10-gene target panel above. This surfaces agents whose mechanism doesn't route through a curated gene (combination therapies, standard-of-care drugs, targets outside the panel).

47 agents shown (approved or with trial evidence) — 42 not found by the gene-target search above.

VancomycinNOT in gene-target list

Phase 3 · 5 trials · source: ClinicalTrials.gov, Open Targets
NCT02464020 NCT01085760 NCT01085760

Oral VancomycinNOT in gene-target list

· 3 trials · source: ClinicalTrials.gov
NCT02137668 NCT05876182 NCT01802073

MetronidazoleNOT in gene-target list

Phase 1 · 3 trials · source: ClinicalTrials.gov, Open Targets
NCT03069976 NCT01085760 NCT01085760

CS0159NOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT05896137 NCT05082779

RituximabNOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT01584388 NCT02705638

Cilofexor

Phase 3 · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT02808312 NCT02943460

CurcuminNOT in gene-target list

Phase 1/2 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT02978339

Elafibranor 80 mgNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05627362

Elafibranor 120 mgNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05627362

Elafibranor

Phase 3 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT07387549

BRS201NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05835505

Anti-human CCL24 monoclonal antibody (CM-101)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04595825

Obeticholic Acid (OCA)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02177136

Hymecromone

Phase 2 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT05295680

bexotegrast (PLN-74809)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04480840

Docosahexaenoic Acid (DHA)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00325013

Standardized mealNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07235670

maralixibat (LUM001)NOT in gene-target list

Approved · 1 trial · source: ClinicalTrials.gov
NCT02061540

ursodeoxycholic acid (UDCA)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01088607

Endoscopic retrograde Cholangio- pancreatographyNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05187312

cladribineNOT in gene-target list

Phase 2 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT00004762

Vidofludimus calciumNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03722576

SulfasalazineNOT in gene-target list

Phase 2 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT03561584

Simvastatin 40mgNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04133792

Genetic AnalysisNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01161992

XifaxanNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01695174

RosuvastatinNOT in gene-target list

Early Phase 1 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT05912387

Oral all-trans retinoic acid (ATRA)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01456468

Volixibat

· 1 trial · source: ClinicalTrials.gov
NCT04663308

Interleukin 2NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01988506

LenalidomideNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02705638

SiplizumabNOT in gene-target list

Phase 1 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT06455280

aldafermin (NGM282)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02704364

Simtuzumab

Phase 2 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT01672853

Cenicriviroc 150 mgNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02653625

Fenofibrate (drug)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06905054

berberine ursodeoxycholate (HTD1801)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03333928

11C-CSarNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01879735

ICGNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01879735

SpyGlass DS I - IINOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03766035

MRCP+NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03809559

EP547NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05525520

oral vancomycin and oral amoxicillinNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06197308

S-Adenosyl-L-methionine (SAMe)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06026865

LB-P8 low-doseNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06699121

LB-P8 high-doseNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06699121

TAK-781NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07229911

Generated by the Open Source Medicine Foundation Biomarker Pipeline — 2026-10-08. Data sources: DGIdb, Open Targets, ChEMBL, PubMed, ClinicalTrials.gov. Not medical advice.