Biomarker & Intervention Discovery

Gout

Pharmacologically actionable gene targets, therapeutic agents ranked by evidence tier, and active clinical trials.

US DALYs263.8KWHO GHE 2021 · all ages
Global DALYs1.83MWHO GHE 2021 · worldwide
US mortality72.2 deaths/yrWHO GHE 2021
Global mortality3,355 deaths/yrWHO GHE 2021 · worldwide
Spontaneous remissionAcute flares self-resolve in 7-14 days without treatment; but urate crystal deposition continues; only 10-15% achieve…
Best-intervention remissionAllopurinol/febuxostat: serum urate target (<360 µmol/L) achieved in 60-70% if adequately dosed; treat-to-target with…
Gap sizeVery large — gout is one of the most undertreated diseases in medicine despite highly effective therapy
Primary barrierframing/practice
Full remission profile →

Remission & chronicity

Spontaneous remission
Acute flares self-resolve in 7-14 days without treatment; but urate crystal deposition continues; only 10-15% achieve serum urate <360 µmol/L spontaneously without ULT; progressive tophus formation and joint damage in untreated patients
Best-intervention remission
Allopurinol/febuxostat: serum urate target (<360 µmol/L) achieved in 60-70% if adequately dosed; treat-to-target with regular monitoring: 80-90% flare-free at 2 years; pegloticase: 40% complete tophus resolution at 6 months in refractory patients
Gap size
Very large — gout is one of the most undertreated diseases in medicine despite highly effective therapy
Primary barrier
framing/practice
Gout is widely (incorrectly) perceived as a diet-only disease by patients and many clinicians; ULT (urate-lowering therapy) is initiated in only 30-40% of eligible patients; of those who start ULT, only 30-40% reach serum urate target because doses are not escalated; gout management is the single most guideline-discordant area in rheumatology
Biomarker Targets & Therapeutic Agents

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

ABCG2 188IL1B 218NLRP3 138SLC22A12 200XDH 189
Therapeutic Agent Effect Direction Evidence Tier Potency Source
FOLIC ACID ? unclear Mechanistic
VORICONAZOLE ? unclear Mechanistic
AXITINIB ? unclear Mechanistic
OSIMERTINIB ? unclear Mechanistic
FELODIPINE ? unclear Mechanistic
TOPOTECAN HYDROCHLORIDE ? unclear Mechanistic
TELMISARTAN ? unclear Mechanistic
DELAVIRDINE ? unclear Mechanistic
SIROLIMUS ? unclear Mechanistic
SILYMARIN ? unclear Mechanistic
CYCLOSPORINE inhibits Mechanistic 63095.73 nM (IC50)
ALVOCIDIB ? unclear Mechanistic
ANHYDROUS TACROLIMUS ? unclear Mechanistic
CYCLOPHOSPHAMIDE ANHYDROUS ? unclear Mechanistic
TENOFOVIR ? unclear Mechanistic
ERLOTINIB HYDROCHLORIDE ? unclear Mechanistic
PACLITAXEL ? unclear Mechanistic
HUMAN CHORIONIC GONADOTROPIN ? unclear Mechanistic
ERLOTINIB ? unclear Mechanistic
EFAVIRENZ ? unclear Mechanistic
DOLUTEGRAVIR SODIUM ? unclear Mechanistic
IGURATIMOD ? unclear Mechanistic
SORAFENIB ? unclear Mechanistic
KETOCONAZOLE ? unclear Mechanistic
ELACRIDAR ? unclear Mechanistic 410.0 nM (IC50)
PRASTERONE ? unclear Mechanistic
RHODAMINE123 ? unclear Mechanistic
PRAZOSIN ? unclear Mechanistic
PRAVASTATIN SODIUM ? unclear Mechanistic
DOXORUBICIN HYDROCHLORIDE ? unclear Mechanistic
SN-38 ? unclear Mechanistic
IMATINIB ? unclear Mechanistic 4897.79 nM (IC50)
RISPERIDONE ? unclear Mechanistic
FLUVASTATIN ? unclear Mechanistic
ZIDOVUDINE ? unclear Mechanistic
NILOTINIB ? unclear Mechanistic
CHEMBL:CHEMBL428402 ? unclear Mechanistic
LOVASTATIN ? unclear Mechanistic
ITRACONAZOLE ? unclear Mechanistic
DIFLOMOTECAN ? unclear Mechanistic

Showing top 40 of 188 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
ASPIRIN ? unclear Mechanistic
MAFOSFAMIDE ? unclear Mechanistic
DIACETYLRHEIN ? unclear Mechanistic
INFLIXIMAB ? unclear Mechanistic
LANSOPRAZOLE ? unclear Mechanistic
TILUDRONIC ACID ? unclear Mechanistic
GEVOKIZUMAB ~ modulates Mechanistic
LUTIKIZUMAB inhibits Mechanistic
CELASTROL ? unclear Mechanistic
CYTARABINE ? unclear Mechanistic
RILONACEPT inhibits Mechanistic
TT-301 ? unclear Mechanistic
METFORMIN ? unclear Mechanistic
ANTICONVULSANT AGENT ? unclear Mechanistic
ANTIDEPRESSANT AGENT ? unclear Mechanistic
ACITRETIN ? unclear Mechanistic
ERYTHROMYCIN ? unclear Mechanistic
ALTEPLASE ? unclear Mechanistic
OFLOXACIN ? unclear Mechanistic
PENTOXIFYLLINE ? unclear Mechanistic
USTEKINUMAB ? unclear Mechanistic
LITHIUM ? unclear Mechanistic
RALOXIFENE HYDROCHLORIDE ? unclear Mechanistic
MITOGEN-ACTIVATED PROTEIN KINASE INHIBITOR ? unclear Mechanistic
DRUGSATFDA.NDA:008378 ? unclear Mechanistic
NICARDIPINE ? unclear Mechanistic
GLUCOSAMINE ? unclear Mechanistic
BECLOMETHASONE DIPROPIONATE ? unclear Mechanistic
PAREGORIC ? unclear Mechanistic
CEFACLOR ANHYDROUS ? unclear Mechanistic
IBUDILAST ? unclear Mechanistic
MELATONIN ? unclear Mechanistic
PENTAMIDINE ? unclear Mechanistic
PRAVASTATIN SODIUM ? unclear Mechanistic
VERAPAMIL ? unclear Mechanistic
THYROGLOBULIN ? unclear Mechanistic
RISEDRONIC ACID ? unclear Mechanistic
AUTOANTIGEN ? unclear Mechanistic
CDP-484 inhibits Mechanistic
ETIDRONIC ACID ? unclear Mechanistic

Showing top 40 of 218 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
ANAKINRA ? unclear Mechanistic
ALAPROCLATE HYDROCHLORIDE ? unclear Mechanistic 8020.0 nM (IC50)
ENCLOMIPHENE CITRATE ? unclear Mechanistic
CHEMBL:CHEMBL585591 ? unclear Mechanistic
4'-METHOXYFLAVONE ? unclear Mechanistic 31000.0 nM (IC50)
DAPANSUTRILE inhibits Mechanistic
CHEMBL:CHEMBL602526 ? unclear Mechanistic
TRICLOCARBAN ? unclear Mechanistic 1490.0 nM (IC50)
CLIOXANIDE ? unclear Mechanistic 1610.0 nM (IC50)
CHEMBL1336269 inhibits Mechanistic 8010.0 nM (IC50)
CHEMBL1596681 inhibits Mechanistic 9020.0 nM (IC50)
CHEMBL1447078 inhibits Mechanistic 53900.0 nM (IC50)
CHEMBL1457403 inhibits Mechanistic 4790.0 nM (IC50)
CHEMBL1539913 inhibits Mechanistic 35400.0 nM (IC50)
CHEMBL1366588 inhibits Mechanistic 2070.0 nM (IC50)
CHEMBL1709001 inhibits Mechanistic 14900.0 nM (IC50)
CHEMBL1329507 inhibits Mechanistic 11000.0 nM (IC50)
CHEMBL1507547 inhibits Mechanistic 76600.0 nM (IC50)
CLOMIPHENE inhibits Mechanistic 29300.0 nM (IC50)
CHEMBL1339092 inhibits Mechanistic 11700.0 nM (IC50)
CHEMBL1574982 inhibits Mechanistic 3530.0 nM (IC50)
CHEMBL1558285 inhibits Mechanistic 14300.0 nM (IC50)
CHEMBL3193158 inhibits Mechanistic 7300.0 nM (IC50)
CHEMBL1339245 inhibits Mechanistic 9700.0 nM (IC50)
CHEMBL1386774 inhibits Mechanistic 4910.0 nM (IC50)
CHEMBL1412902 inhibits Mechanistic 10300.0 nM (IC50)
CHEMBL1347185 inhibits Mechanistic 6600.0 nM (IC50)
CHEMBL1343658 inhibits Mechanistic 1940.0 nM (IC50)
CHEMBL1602195 inhibits Mechanistic 2900.0 nM (IC50)
CHEMBL1334557 inhibits Mechanistic 5920.0 nM (IC50)
CHEMBL1711405 inhibits Mechanistic 23300.0 nM (IC50)
CHEMBL3192672 inhibits Mechanistic 45500.0 nM (IC50)
CHEMBL1563728 inhibits Mechanistic 3160.0 nM (IC50)
CHEMBL1505335 inhibits Mechanistic 41800.0 nM (IC50)
CHEMBL1447669 inhibits Mechanistic 8190.0 nM (IC50)
CHEMBL1427059 inhibits Mechanistic 1970.0 nM (IC50)
CHEMBL1505307 inhibits Mechanistic 43400.0 nM (IC50)
CHEMBL602526 inhibits Mechanistic 1620.0 nM (IC50)
CHEMBL1398317 inhibits Mechanistic 13700.0 nM (IC50)
CHEMBL3191011 inhibits Mechanistic 24300.0 nM (IC50)

Showing top 40 of 138 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
VERINURAD inhibits Mechanistic 23.0 nM (IC50)
ARHALOFENATE inhibits Mechanistic
PRATOSARTAN inhibits Mechanistic
IDARUBICIN ? unclear Mechanistic
DOTINURAD inhibits Mechanistic
BENZBROMARONE inhibits Mechanistic 300.0 nM (IC50)
LOSARTAN POTASSIUM ? unclear Mechanistic
SHR-4640 inhibits Mechanistic 33.7 nM (IC50)
FLUDARABINE ? unclear Mechanistic
GEMTUZUMAB OZOGAMICIN ? unclear Mechanistic
CYTARABINE ? unclear Mechanistic
LESINURAD inhibits Mechanistic 6700.0 nM (IC50)
SULFINPYRAZONE inhibits Mechanistic 100000.0 nM (IC50)
MORIN inhibits Mechanistic 2000.0 nM (IC50)
PROBENECID inhibits Mechanistic 50000.0 nM (IC50)
CHEMBL1767090 inhibits Mechanistic 2490.0 nM (IC50)
CHEMBL1767088 inhibits Mechanistic 2460.0 nM (IC50)
BENZARONE inhibits Mechanistic 2800.0 nM (IC50)
CHEMBL1767079 inhibits Mechanistic 138.0 nM (IC50)
CHEMBL1767091 inhibits Mechanistic 6680.0 nM (IC50)
CHEMBL1767094 inhibits Mechanistic 33650.0 nM (IC50)
CHEMBL1767097 inhibits Mechanistic 3920.0 nM (IC50)
CHEMBL1767099 inhibits Mechanistic 1130.0 nM (IC50)
CHEMBL1767100 inhibits Mechanistic 19690.0 nM (IC50)
CHEMBL1767105 inhibits Mechanistic 3940.0 nM (IC50)
CHEMBL1767106 inhibits Mechanistic 42.0 nM (IC50)
CHEMBL1767107 inhibits Mechanistic 189.0 nM (IC50)
CHEMBL1767108 inhibits Mechanistic 358.0 nM (IC50)
CHEMBL1767109 inhibits Mechanistic 83.0 nM (IC50)
CHEMBL1767110 inhibits Mechanistic 1440.0 nM (IC50)
CHEMBL1767111 inhibits Mechanistic 287.0 nM (IC50)
CHEMBL1767112 inhibits Mechanistic 111.0 nM (IC50)
CHEMBL1767113 inhibits Mechanistic 1650.0 nM (IC50)
CHEMBL1767114 inhibits Mechanistic 177.0 nM (IC50)
CHEMBL1767115 inhibits Mechanistic 23460.0 nM (IC50)
CHEMBL1767116 inhibits Mechanistic 667.0 nM (IC50)
CHEMBL1767117 inhibits Mechanistic 772.0 nM (IC50)
CHEMBL3745776 inhibits Mechanistic 158.8 nM (IC50)
CHEMBL3745813 inhibits Mechanistic 1189.0 nM (IC50)
CHEMBL3747053 inhibits Mechanistic 333.4 nM (IC50)

Showing top 40 of 200 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
THERAPEUTIC GLUCOCORTICOID ? unclear Mechanistic
MERCAPTOPURINE ? unclear Mechanistic
QUERCETIN ? unclear Mechanistic 3000.0 nM (IC50)
DIDANOSINE ? unclear Mechanistic
INSULIN, REGULAR, HUMAN ? unclear Mechanistic
BENZODIAZEPINE ? unclear Mechanistic
DOXORUBICIN HYDROCHLORIDE ? unclear Mechanistic
ROXITHROMYCIN ? unclear Mechanistic
FEBUXOSTAT inhibits Mechanistic 30.0 nM (IC50)
IMMUNOTHERAPEUTIC AGENT ? unclear Mechanistic
FOSDENOPTERIN ? unclear Mechanistic
FENTANYL CITRATE ? unclear Mechanistic
ALLOPURINOL inhibits Mechanistic 4200.0 nM (IC50)
ALLOPURINOL SODIUM inhibits Mechanistic
VINCRISTINE ? unclear Mechanistic
PAPAVERINE ? unclear Mechanistic
RICIN ? unclear Mechanistic
RECOMBINANT INTERFERON ? unclear Mechanistic
FOLIC ACID ? unclear Mechanistic
AZITHROMYCIN ANHYDROUS ? unclear Mechanistic
CHEMOPREVENTIVE AGENT ? unclear Mechanistic
METHYLENE BLUE ANHYDROUS ? unclear Mechanistic
TIGULIXOSTAT ? unclear Mechanistic
ERYTHROMYCIN ? unclear Mechanistic
CARVEDILOL ? unclear Mechanistic
MISONIDAZOLE ? unclear Mechanistic
SIALYL TN ANTIGEN ? unclear Mechanistic
CURCUMIN ? unclear Mechanistic
NITROFURANTOIN, MACROCRYSTALS ? unclear Mechanistic
HYPOXANTHINE ? unclear Mechanistic
MITOMYCIN ? unclear Mechanistic
THIOGUANINE ? unclear Mechanistic 92420.0 nM (IC50)
DAUNORUBICIN LIPOSOMAL ? unclear Mechanistic
OXYPURINOL inhibits Mechanistic 1000.0 nM (Ki)
CHINESE HERBS ? unclear Mechanistic
TOPIROXOSTAT inhibits Mechanistic
BENDAZAC inhibits Mechanistic
OTERACIL POTASSIUM ? unclear Mechanistic
CLARITHROMYCIN ? unclear Mechanistic
CHEMBL87562 inhibits Mechanistic 330.0 nM (Ki)

Showing top 40 of 189 agents ranked by evidence tier.


Clinical Trials

Recruiting and recently completed trials from ClinicalTrials.gov. Data retrieved 2026-07-06.

No recent clinical trials found on ClinicalTrials.gov for this condition.


Agents Found By Disease-Level Search

Searched directly from “Gout” via ClinicalTrials.gov interventions and Open Targets' disease→drug data — independent of the 5-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).

Not yet run. python -m biomarker_pipeline.run_disease_agent_discovery --disease "..."

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