Biomarker & Intervention Discovery

NAFLD / MASH (Metabolic-Associated Steatohepatitis)

This page lists 12 druggable gene targets, 22 approved or trial-tested drug candidates and 15 recent clinical trials for NAFLD / MASH (Metabolic-Associated Steatohepatitis), 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.

NIH funding$387MFY2025 · RCDC estimate
Spontaneous remissionMASH (formerly NASH) with fibrosis progresses to cirrhosis in ~20% over 10 years on standard care (which until 2024…
Best-intervention remissionResmetirom (Rezdiffra) — first FDA-approved pharmacotherapy for MASH (March 2024): 26-30% histological resolution at…
Gap sizeLarge — first drug only just approved in 2024 after decades of failed trials; practice still dominated by lifestyle…
Primary barrierbiological (historical) + access (current)
Full remission profile →

Remission & chronicity

Spontaneous remission
MASH (formerly NASH) with fibrosis progresses to cirrhosis in ~20% over 10 years on standard care (which until 2024 was lifestyle modification only); fibrosis regression occurs spontaneously in ~25% over 5 years with sustained weight loss
Best-intervention remission
Resmetirom (Rezdiffra) — first FDA-approved pharmacotherapy for MASH (March 2024): 26-30% histological resolution at 52 weeks vs 10% placebo; bariatric surgery achieves MASH resolution in 85% and fibrosis regression in 35% at 5 years; >7-10% weight loss resolves MASH in ~50%
Gap size
Large — first drug only just approved in 2024 after decades of failed trials; practice still dominated by lifestyle advice alone
Primary barrier
biological (historical) + access (current)
Until March 2024, there was literally no approved pharmacotherapy for MASH despite it being a leading cause of cirrhosis and liver transplant; the field repeatedly failed Phase 3 (obeticholic acid, elafibranor, selonsertib, cenicriviroc — all negative); resmetirom is a genuine breakthrough but access and prescribing uptake are still ramping up

Biomarker Targets & Therapeutic Agents

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

FGF21 1GLP1R 294IRS1 16NR1H4 218PNPLA3 12PPARA 202PPARD 292PPARG 231SREBF1 30TGFB1 62THRB 253TNF 277
Therapeutic Agent Effect Direction Evidence Tier Potency Source
PEGBELFERMIN ? unclear Mechanistic
Therapeutic Agent Effect Direction Evidence Tier Potency Source
CHEMBL:CHEMBL607309 ? unclear Mechanistic
CHEMBL:CHEMBL585444 ? unclear Mechanistic
CHEMBL:CHEMBL1327172 ? unclear Mechanistic
CHEMBL:CHEMBL1224483 ? unclear Mechanistic
CGP13501 ? unclear Mechanistic
CHEMBL:CHEMBL580340 ? unclear Mechanistic
EFPEGLENATIDE ↑ activates Mechanistic
LIRAGLUTIDE ? unclear Mechanistic
DULOXETINE HYDROCHLORIDE ? unclear Mechanistic
CHEMBL:CHEMBL597251 ? unclear Mechanistic
ANGUSTIBALIN ? unclear Mechanistic
DULAGLUTIDE ↑ activates Mechanistic
EXENATIDE ↑ activates Mechanistic 0.66 nM (IC50)
MOFEBUTAZONE ? unclear Mechanistic
CHEMBL:CHEMBL532412 ? unclear Mechanistic
NORTRIPTYLINE HYDROCHLORIDE ? unclear Mechanistic
CHEMBL:CHEMBL533602 ? unclear Mechanistic
PEGSEBRENATIDE ↑ activates Mechanistic
OLANZAPINE ? unclear Mechanistic
CHEMBL:CHEMBL317115 ? unclear Mechanistic
CHEMBL:CHEMBL1549738 ? unclear Mechanistic
CYCLOASPEPTIDE A ? unclear Mechanistic
SCOULERINE ? unclear Mechanistic
KEPONE ? unclear Mechanistic
PEGAPAMODUTIDE ↑ activates Mechanistic
CHEMBL:CHEMBL603154 ? unclear Mechanistic
ETOPOSIDE ? unclear Mechanistic
CHEMBL:CHEMBL405914 ? unclear Mechanistic
CHEMBL:CHEMBL1308346 ? unclear Mechanistic
FIDUXOSIN HYDROCHLORIDE ? unclear Mechanistic
CLOZAPINE ? unclear Mechanistic
CHEMBL:CHEMBL578515 ? unclear Mechanistic
CHEMBL:CHEMBL1439833 ? unclear Mechanistic
CHEMBL:CHEMBL601351 ? unclear Mechanistic
CHEMBL:CHEMBL580727 ? unclear Mechanistic
AMORFRUTIN A ? unclear Mechanistic
PROPIONYLPROMAZINE HYDROCHLORIDE ? unclear Mechanistic
CHEMBL:CHEMBL588038 ? unclear Mechanistic
CHEMBL:CHEMBL1420370 ? unclear Mechanistic
CHEMBL:CHEMBL580207 ? unclear Mechanistic

Showing top 40 of 294 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
AGANIRSEN ? unclear Mechanistic
CYCLOPHOSPHAMIDE ANHYDROUS ? unclear Mechanistic
FLUOROURACIL ? unclear Mechanistic
DEHYDRATED ALCOHOL ? unclear Mechanistic
PRASTERONE ? unclear Mechanistic
EPIRUBICIN ? unclear Mechanistic
INOSITOL ? unclear Mechanistic
GLIMEPIRIDE ? unclear Mechanistic
CHEMBL1685056 ↓ inhibits Mechanistic 50.0 nM (Kd)
CHEMBL1685055 ↓ inhibits Mechanistic 150.0 nM (Kd)
CHEMBL1681823 ↓ inhibits Mechanistic 50.0 nM (Kd)
CHEMBL4536878 ↓ inhibits Mechanistic 120.0 nM (Kd)
CHEMBL4591779 ↓ inhibits Mechanistic 16200.0 nM (Kd)
CHEMBL4530453 ↓ inhibits Mechanistic 1600.0 nM (Kd)
CHEMBL1572640 ↓ inhibits Mechanistic 3300.0 nM (Kd)
CHEMBL4576057 ↓ inhibits Mechanistic 16400.0 nM (Kd)
Therapeutic Agent Effect Direction Evidence Tier Potency Source
CHENODEOXYCHOLIC ACID ↑ activates Mechanistic
MELPHALAN ? unclear Mechanistic
SAQUINAVIR MESYLATE ? unclear Mechanistic
CHEMBL:CHEMBL269105 ? unclear Mechanistic
TO-901317 ? unclear Mechanistic
METHYLENE BLUE ? unclear Mechanistic
GANODERIC ACID TR ? unclear Mechanistic
GANODERMANONTRIOL ? unclear Mechanistic
7-O-METHYLCRYPTOCHINONE A ? unclear Mechanistic
OBETICHOLIC ACID ↑ activates Mechanistic 99.0 nM (EC50)
FLUOXASTROBIN ? unclear Mechanistic
TUBERATOLIDE A ? unclear Mechanistic
TRIBUTYLCHLOROSTANNANE ? unclear Mechanistic
HOMIDIUM BROMIDE ? unclear Mechanistic
TUBERATOLIDE B ? unclear Mechanistic
(R)-SARGACHROMENOL ? unclear Mechanistic
IMIPRAMINE HYDROCHLORIDE ? unclear Mechanistic
KETOCONAZOLE ? unclear Mechanistic
TROPIFEXOR ↑ activates Mechanistic
DIFENOCONAZOLE ? unclear Mechanistic
THEONELLASTEROL E ? unclear Mechanistic
CILOFEXOR ↑ activates Mechanistic
ERGOSTEROL PEROXIDE ? unclear Mechanistic
ZEARALENONE ? unclear Mechanistic
THEONELLASTEROL G ? unclear Mechanistic
CHEMBL:CHEMBL476302 ? unclear Mechanistic
CONICASTEROL D ? unclear Mechanistic
THEONELLASTEROL F ? unclear Mechanistic
HYDRAMETHYLNON ? unclear Mechanistic
THEONELLASTEROL B ? unclear Mechanistic
CHEMBL:CHEMBL297304 ? unclear Mechanistic
APOMINE ? unclear Mechanistic
LITHOCHOLIC ACID ? unclear Mechanistic 20000.0 nM (EC50)
CRYPTOCHINONE A ? unclear Mechanistic
PX-102 ↑ activates Mechanistic
NIMODIPINE ? unclear Mechanistic
SUVANINE'S SODIUM SALT ? unclear Mechanistic
2'-EPI-TUBERATOLIDE B ? unclear Mechanistic
CHEMBL:CHEMBL184450 ? unclear Mechanistic
THIMEROSAL ? unclear Mechanistic

Showing top 40 of 218 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
PREDNISOLONE ? unclear Mechanistic
DOXORUBICIN HYDROCHLORIDE ? unclear Mechanistic
DEHYDRATED ALCOHOL ? unclear Mechanistic
CYTARABINE ? unclear Mechanistic
THIOGUANINE ? unclear Mechanistic
ASPARAGINASE ? unclear Mechanistic
PEGASPARGASE ? unclear Mechanistic
METHOTREXATE ? unclear Mechanistic
MERCAPTOPURINE ? unclear Mechanistic
VINCRISTINE ? unclear Mechanistic
CYCLOPHOSPHAMIDE ANHYDROUS ? unclear Mechanistic
DAUNORUBICIN LIPOSOMAL ? unclear Mechanistic
Therapeutic Agent Effect Direction Evidence Tier Potency Source
INDEGLITAZAR ↑ activates Mechanistic
FENOFIBRIC ACID ↑ activates Mechanistic 30000.0 nM (EC50)
LINOLEIC ACID ? unclear Mechanistic
IMIGLITAZAR ↑ activates Mechanistic
LANIFIBRANOR ↑ activates Mechanistic
ZYH7 ↑ activates Mechanistic
OLEIC ACID ? unclear Mechanistic
SODELGLITAZAR ↑ activates Mechanistic
ELAFIBRANOR ↑ activates Mechanistic
PROSTAGLANDIN D2 ? unclear Mechanistic
TETRADECYLTHIOACETIC ACID ? unclear Mechanistic
PIRINIXIC ACID ? unclear Mechanistic 5000.0 nM (EC50)
PHENPROCOUMON ? unclear Mechanistic
METOPROLOL ? unclear Mechanistic
CIPROFIBRATE ↑ activates Mechanistic
LY-518674 ↑ activates Mechanistic
MYRISTIC ACID ? unclear Mechanistic
NAVEGLITAZAR ↑ activates Mechanistic
GFT14 ? unclear Mechanistic
CHEMBL:CHEMBL292352 ? unclear Mechanistic
ROMAZARIT ? unclear Mechanistic
DRF-10945 ↑ activates Mechanistic
GW590735 ↑ activates Mechanistic
CHEMBL:CHEMBL259327 ? unclear Mechanistic
METFORMIN ? unclear Mechanistic
PEMAFIBRATE ↑ activates Mechanistic
{4-[3-(4-ACETYL-3-HYDROXY-2-PROPYLPHENOXY)PROPOXY]PHENOXY}ACETIC ACID ? unclear Mechanistic
ATENOLOL ? unclear Mechanistic
GW7647 ? unclear Mechanistic
RAGAGLITAZAR ? unclear Mechanistic 3210.0 nM (EC50)
OXEGLITAZAR ? unclear Mechanistic
URSOLIC ACID ? unclear Mechanistic
BITHIONOL ? unclear Mechanistic
ALEGLITAZAR ↑ activates Mechanistic
CARVEDILOL ? unclear Mechanistic
LACTOFEN ? unclear Mechanistic
GW0742X ? unclear Mechanistic
LOBEGLITAZONE ? unclear Mechanistic
PHENFORMIN HYDROCHLORIDE ? unclear Mechanistic
CHEMBL:CHEMBL1089501 ? unclear Mechanistic

Showing top 40 of 202 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
CHLORPROMAZINE HYDROCHLORIDE ? unclear Mechanistic
ALL-TRANS-RETINAL ? unclear Mechanistic
BIFENAZATE ? unclear Mechanistic
BITHIONOL ? unclear Mechanistic
DINOSEB ? unclear Mechanistic
VITAMIN K3 ? unclear Mechanistic
DIFENOCONAZOLE ? unclear Mechanistic
RALOXIFENE HYDROCHLORIDE ? unclear Mechanistic
ENCLOMIPHENE ? unclear Mechanistic
CHEMBL:CHEMBL1606 ? unclear Mechanistic
OCTYL_GALLATE ? unclear Mechanistic
PROMETHAZINE HYDROCHLORIDE ? unclear Mechanistic
ATORVASTATIN CALCIUM TRIHYDRATE ? unclear Mechanistic
ORYZALIN ? unclear Mechanistic
ELAFIBRANOR ↑ activates Mechanistic
E319 ? unclear Mechanistic
BEZAFIBRATE ↑ activates Mechanistic 20000.0 nM (EC50)
IDAZOXAN ? unclear Mechanistic
BAZEDOXIFENE ACETATE ? unclear Mechanistic
SERATRODAST ? unclear Mechanistic
FLUOXETINE HYDROCHLORIDE ? unclear Mechanistic
VITAMIN K3H2 ? unclear Mechanistic
CHIGLITAZAR ↑ activates Mechanistic
CHEMBL:CHEMBL172988 ? unclear Mechanistic
BISPHENOL A ? unclear Mechanistic
CLOXYQUIN ? unclear Mechanistic
CHEMBL:CHEMBL1257022 ? unclear Mechanistic
BIFONAZOLE ? unclear Mechanistic
IPCONAZOLE ? unclear Mechanistic
HEXACHLOROPHENE ? unclear Mechanistic
HYCANTHONE ? unclear Mechanistic
ASTEMIZOLE ? unclear Mechanistic
CHEMBL:CHEMBL292352 ? unclear Mechanistic
ALPHA-TERTHIENYL ? unclear Mechanistic
SALINAZID ? unclear Mechanistic
TRICLOCARBAN ? unclear Mechanistic
FENTICLOR ? unclear Mechanistic
HYMECROMONE ? unclear Mechanistic
CINACALCET HYDROCHLORIDE ? unclear Mechanistic
GW501516 ↑ activates Mechanistic

Showing top 40 of 292 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
METHOXYCHLOR ? unclear Mechanistic
CHEMBL:CHEMBL2208197 ? unclear Mechanistic
PROPYLPYRAZOLETRIOL ? unclear Mechanistic
LANIFIBRANOR ↑ activates Mechanistic
PROPYLPARABEN ? unclear Mechanistic
ABIETIC ACID ? unclear Mechanistic
KEPONE ? unclear Mechanistic
SPIRONOLACTONE ? unclear Mechanistic
FLUOXASTROBIN ? unclear Mechanistic
ROSIGLITAZONE MALEATE ↑ activates Mechanistic
CHEMBL:CHEMBL2178583 ? unclear Mechanistic
NAVEGLITAZAR ↑ activates Mechanistic
CELECOXIB ? unclear Mechanistic
SHINPTEROCARPIN ? unclear Mechanistic
PSAMMAPLIN A ? unclear Mechanistic
TECNAZENE ? unclear Mechanistic
MYCOPHENOLATE ? unclear Mechanistic
CHEMBL:CHEMBL1833984 ? unclear Mechanistic
IMIGLITAZAR ↑ activates Mechanistic
ROSIGLITAZONE ? unclear Mechanistic 250.0 nM (IC50)
CLX-0921 ↑ activates Mechanistic
AVE0847 ↑ activates Mechanistic
CHLORFENAPYR ? unclear Mechanistic
PIOGLITAZONE HYDROCHLORIDE ↑ activates Mechanistic
PROCHLORAZ ? unclear Mechanistic
MESALAMINE ↑ activates Mechanistic
BEZAFIBRATE ↑ activates Mechanistic
CHEMBL:CHEMBL312032 ? unclear Mechanistic
ISOSILYBIN A ? unclear Mechanistic
AMORFRUTIN A ? unclear Mechanistic
NALED ? unclear Mechanistic
TROGLITAZONE ↑ activates Mechanistic 302.0 nM (Ki)
PROSTAGLANDIN D2 ? unclear Mechanistic
INT131 ~ modulates Mechanistic
EFATUTAZONE HYDROCHLORIDE ↑ activates Mechanistic
FLUORODIFEN ? unclear Mechanistic
FLUDIOXONIL ? unclear Mechanistic
ESTRADIOL VALERATE ? unclear Mechanistic
CHEMBL:CHEMBL166112 ? unclear Mechanistic
BENSULIDE ? unclear Mechanistic

Showing top 40 of 231 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
RECOMBINANT NEUROTROPHIC FACTOR ? unclear Mechanistic
INSULIN, REGULAR, HUMAN ? unclear Mechanistic
CALCIFEDIOL ANHYDROUS ↓ inhibits Mechanistic 1176.0 nM (IC50)
CHEMBL4876668 ↓ inhibits Mechanistic 1160.0 nM (IC50)
CHEMBL4851466 ↓ inhibits Mechanistic 722.7 nM (IC50)
CHEMBL4851396 ↓ inhibits Mechanistic 1600.0 nM (IC50)
CHEMBL1824673 ↓ inhibits Mechanistic 700.0 nM (IC50)
CHEMBL1824658 ↓ inhibits Mechanistic 5000.0 nM (IC50)
T091317 ↑ activates Mechanistic 20.0 nM (EC50)
CHEMBL5597106 ↑ activates Mechanistic 31000.0 nM (EC50)
CHEMBL5596392 ↑ activates Mechanistic 20000.0 nM (EC50)
CHEMBL5597291 ↑ activates Mechanistic 1500.0 nM (EC50)
CHEMBL5590383 ↑ activates Mechanistic 4300.0 nM (EC50)
CHEMBL5596520 ↑ activates Mechanistic 38000.0 nM (EC50)
CHEMBL5597394 ↑ activates Mechanistic 1300.0 nM (EC50)
CHEMBL5598279 ↑ activates Mechanistic 2800.0 nM (EC50)
CHEMBL5597319 ↑ activates Mechanistic 3000.0 nM (EC50)
CHEMBL5596811 ↑ activates Mechanistic 2900.0 nM (EC50)
CHEMBL5597943 ↑ activates Mechanistic 6900.0 nM (EC50)
CHEMBL5596386 ↑ activates Mechanistic 570.0 nM (EC50)
CHEMBL5596592 ↑ activates Mechanistic 12000.0 nM (EC50)
CHEMBL5596248 ↑ activates Mechanistic 190.0 nM (EC50)
CHEMBL5596939 ↑ activates Mechanistic 1800.0 nM (EC50)
CHEMBL5598331 ↑ activates Mechanistic 1300.0 nM (EC50)
CHEMBL5596815 ↑ activates Mechanistic 310.0 nM (EC50)
CHEMBL5597153 ↑ activates Mechanistic 240.0 nM (EC50)
CHEMBL5597982 ↑ activates Mechanistic 130.0 nM (EC50)
CHEMBL5598250 ↑ activates Mechanistic 770.0 nM (EC50)
CHEMBL5590706 ↑ activates Mechanistic 190.0 nM (EC50)
CHEMBL5596907 ↑ activates Mechanistic 8600.0 nM (EC50)
Therapeutic Agent Effect Direction Evidence Tier Potency Source
INTERFERON ALFA-2B ? unclear Mechanistic
PIOGLITAZONE HYDROCHLORIDE ? unclear Mechanistic
SILYMARIN ? unclear Mechanistic
RITUXIMAB ? unclear Mechanistic
VERAPAMIL ? unclear Mechanistic
PROTEASOME INHIBITOR ? unclear Mechanistic
FRESOLIMUMAB ? unclear Mechanistic
ATENOLOL ? unclear Mechanistic
BINTRAFUSP ALFA ~ modulates Mechanistic
NISEVOKITUG ? unclear Mechanistic
LUSPATERCEPT ↓ inhibits Mechanistic
TRETINOIN ? unclear Mechanistic
HYDROCORTISONE BUTYRATE ? unclear Mechanistic
VITAMIN E ? unclear Mechanistic
IRINOTECAN HYDROCHLORIDE ? unclear Mechanistic
INOSITOL ? unclear Mechanistic
NICOTINE POLACRILEX ? unclear Mechanistic
FENRETINIDE ? unclear Mechanistic
MELATONIN ? unclear Mechanistic
ISOTRETINOIN ? unclear Mechanistic
VACTOSERTIB ? unclear Mechanistic 12.9 nM (IC50)
TRIAMCINOLONE ? unclear Mechanistic
TAMOXIFEN ? unclear Mechanistic
ADRIAMYCIN ? unclear Mechanistic
HEPARAN SULFATE ? unclear Mechanistic
GOSSYPOL ? unclear Mechanistic
TCDD ? unclear Mechanistic
AMIFOSTINE ANHYDROUS ? unclear Mechanistic
RAMIPRIL ? unclear Mechanistic
ANTIOXIDANT ? unclear Mechanistic
SOTATERCEPT ? unclear Mechanistic
ASPIRIN ? unclear Mechanistic
GALUNISERTIB ? unclear Mechanistic
KEYHOLE LIMPET HEMOCYANIN ? unclear Mechanistic
PIRFENIDONE ? unclear Mechanistic
H2O2 ? unclear Mechanistic
THERAPEUTIC ANDROGEN ? unclear Mechanistic
SANDOSTATIN ? unclear Mechanistic
BROXURIDINE ? unclear Mechanistic
DISITERTIDE ? unclear Mechanistic

Showing top 40 of 62 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
METHYL PARATHIONE ? unclear Mechanistic
DIENOCHLOR ? unclear Mechanistic
AXITIROME ? unclear Mechanistic
3-METHYLCHOLANTHRENE ? unclear Mechanistic
CHEMBL:CHEMBL577415 ? unclear Mechanistic
DYCLONINE HYDROCHLORIDE ? unclear Mechanistic
LEVOTHYROXINE ↑ activates Mechanistic
ORTHO-PHTHALALDEHYDE ? unclear Mechanistic
QUINALIZARIN ? unclear Mechanistic
CHEMBL:CHEMBL297304 ? unclear Mechanistic
CHEMBL:CHEMBL1493528 ? unclear Mechanistic
RESMETIROM ↑ activates Mechanistic
CLOSANTEL ? unclear Mechanistic
IDARUBICIN ? unclear Mechanistic
FUMARPROTOCETRARIC ACID ? unclear Mechanistic
DIPYRIDAMOLE ? unclear Mechanistic
CHEMBL:CHEMBL1341830 ? unclear Mechanistic
CARUBICIN ? unclear Mechanistic
ALBUTEROL SULFATE ? unclear Mechanistic
PRIMAQUINE ? unclear Mechanistic
DEXTROTHYROXINE SODIUM ↑ activates Mechanistic
SOBETIROME ↑ activates Mechanistic 0.1 nM (Kd)
METHYLENE BLUE ANHYDROUS ? unclear Mechanistic
HEXACHLOROPHENE ? unclear Mechanistic
CHEMBL:CHEMBL1465049 ? unclear Mechanistic
COMPOUND 16G [PMID: 35507418] ↑ activates Mechanistic
CHLOROTHALONIL ? unclear Mechanistic
SLAZINIC ACID ? unclear Mechanistic
TRIAMTERENE ? unclear Mechanistic
CHEMBL:CHEMBL601952 ? unclear Mechanistic
CHEMBL:CHEMBL578741 ? unclear Mechanistic
CIRAZOLINE ? unclear Mechanistic
CHEMBL:CHEMBL581044 ? unclear Mechanistic
DAUNORUBICIN HYDROCHLORIDE ? unclear Mechanistic
RT3 ↑ activates Mechanistic
CHEMBL:CHEMBL592124 ? unclear Mechanistic
VK-2809 ↑ activates Mechanistic
CHEMBL:CHEMBL602969 ? unclear Mechanistic
EPROTIROME ↑ activates Mechanistic
TOLONIUM CHLORIDE ? unclear Mechanistic

Showing top 40 of 253 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
NERELIMOMAB ↓ inhibits Mechanistic
CROMOLYN SODIUM ? unclear Mechanistic
PLACULUMAB ↓ inhibits Mechanistic
METHYLENE BLUE ? unclear Mechanistic
ALTEPLASE ? unclear Mechanistic
CARBOPLATIN ? unclear Mechanistic
GENTAMICIN ? unclear Mechanistic
GOLIMUMAB ↓ inhibits Mechanistic
BUPIVACAINE ? unclear Mechanistic
PENTOXIFYLLINE ? unclear Mechanistic 85000.0 nM (IC50)
BENZO[E]PYRENE ? unclear Mechanistic
THALIDOMIDE ? unclear Mechanistic
PYRAZINAMIDE ? unclear Mechanistic
GLIMEPIRIDE ? unclear Mechanistic
OMEPRAZOLE ? unclear Mechanistic
LENERCEPT ↓ inhibits Mechanistic
ADALIMUMAB ↓ inhibits Mechanistic
PRAZOSIN HYDROCHLORIDE ? unclear Mechanistic
REMTOLUMAB ? unclear Mechanistic
MIDOSTAURIN ? unclear Mechanistic
LENABASUM ? unclear Mechanistic
MAGNESIUM SULFATE ANHYDROUS ? unclear Mechanistic
PICIBANIL ? unclear Mechanistic
FLUOCINOLONE ACETONIDE ? unclear Mechanistic
MILTEFOSINE ? unclear Mechanistic
HOMIDIUM BROMIDE ? unclear Mechanistic
OZORALIZUMAB ↓ inhibits Mechanistic
PEGSUNERCEPT ↓ inhibits Mechanistic
CERTOLIZUMAB PEGOL ↓ inhibits Mechanistic
THERAPEUTIC HORMONE ? unclear Mechanistic
NAFAMOSTAT ? unclear Mechanistic
FOLIC ACID ? unclear Mechanistic
ABBV-257 ? unclear Mechanistic
RISPERIDONE ? unclear Mechanistic
COVA322 ? unclear Mechanistic
HYDROXYCHLOROQUINE ? unclear Mechanistic
CERTOLIZUMAB ? unclear Mechanistic
AN0128 ? unclear Mechanistic
5,7-DIHYDROXY-4-METHYLCOUMARIN ? unclear Mechanistic
SORAFENIB ? unclear Mechanistic

Showing top 40 of 277 agents ranked by evidence tier.


Clinical Trials

Drug, biologic and supplement trials (treatment or prevention) from ClinicalTrials.gov whose main condition is NAFLD / MASH (Metabolic-Associated Steatohepatitis): recruiting, active or completed, most recently updated first. Data retrieved 2026-10-28.

NCT07631637 Phase: 2 Started: 2026-07-06

This study will test a Regeneron study drug called ALN-CIDEB to find out whether it may help treat a liver disease called MASH. In this study, researchers are looking at the effect of ALN-CIDEB on reducing liver fat, liver injury, and liver scarring. The study will compare ALN-CIDEB with placebo to ...

NCT06419374 Phase: 3 Started: 2024-05-24

The study will assess the efficacy and safety of pegozafermin administered in participants with biopsy-confirmed and clinically confirmed compensated cirrhosis due to MASH .

NCT06318169 Phase: 3 Started: 2024-03-13

The study will assess the efficacy and safety of 2 dose regimens of pegozafermin compared to placebo for the treatment of liver fibrosis stage F2 or F3 in adult participants with MASH.

NCT07553663 Phase: 1 Started: 2026-04-30

A Phase 1b, Open-Label Study Evaluating the Pharmacokinetics and Safety of Miricorilant in Adult Patients With Presumed Metabolic Dysfunction-Associated Steatohepatitis (MASH)

NCT06528314 Phase: 3 Started: 2024-10-04

This is a multi-center evaluation of efruxifermin (EFX) in a randomized, double-blind, placebo-controlled study in subjects with compensated cirrhosis due to NASH/MASH.

NCT07288138 Phase: 2 Started: 2025-12-15

The primary objective of this trial is to evaluate the dose-dependent and comparative effects of ECC4703 (low and high dose), ECC0509 (low and high dose), and their combination on hepatic fat reduction as assessed by change in magnetic resonance imaging proton density fat fraction (MRI-PDFF) at Week...

NCT05519475 Phase: 2 Started: 2023-02-09

This study is researching an investigational drug, ALN-HSD called "study drug". This study is focused on participants who are known to have Metabolic dysfunction-Associated SteatoHepatitis (MASH). MASH is a form of Metabolic dysfunction-Associated Steatotic Liver Disease (MASLD). MASH occurs when fa...

NCT06947304 Phase: 1 Started: 2025-08-22

A Phase 1, Open-Label Study Evaluating the Effect of Miricorilant on Hepatic Lipids in Patients with Presumed Metabolic Dysfunction-Associated Steatohepatitis (MASH)

NCT06108219 Phase: 2 Started: 2023-10-25

A Phase 2b, Randomized, Double-Blind, Placebo-Controlled Study Evaluating the Efficacy and Safety of Miricorilant in Adult Patients with Nonalcoholic Steatohepatitis/Metabolic Dysfunction-Associated Steatohepatitis (MONARCH)

NCT07527910 Phase: 2 Started: 2026-05-22

This study will test a study drug called ALN-PNP with and without another drug that is used for controlling blood sugar, appetite, and weight (for example, tirzepatide), to see if it can help treat MASLD, also known as fatty liver disease. ALN-PNP reduces the amount of Patatin-like phospholipase dom...

NCT07581951 Phase: 3 Started: 2026-06-30

A phase 3, randomized, double-blind, placebo-controlled study evaluating the safety and tolerability of HSK31679 compared to placebo in patients with metabolic dysfunction-associated steatohepatitis (MASH) .

NCT07221188 Phase: 3 Started: 2025-12-12

This study will evaluate the safety and tolerability of Efimosfermin Alfa for participants with known or suspected MASH with fibrosis consistent with stage F2 or F3.

NCT06374875 Phase: 4 Started: 2024-07-11

Metabolic dysfunction-associated steatotic liver disease (MASLD), formerly known as non-alcoholic fatty liver disease (NAFLD), a major global public health concern, is commonly associated with obesity, diabetes, and dyslipidemia. MASLD is currently the most common cause of chronic liver disease affe...

NCT07221227 Phase: 3 Started: 2025-10-24

The purpose of this study is to assess the safety and efficacy of efimosfermin alfa in the resolution of steatohepatitis and improvement of liver-related clinical outcome compared to placebo in individuals with MASH and biopsy-confirmed F2- or F3-stage fibrosis.

NCT06920043 Phase: 2 Started: 2025-04-09

The purpose of this study is to evaluate the safety, tolerability, preliminary efficacy, and pharmacokinetics (PK) of efimosfermin in participants with metabolic dysfunction associated steatohepatitis (MASH) and compensated cirrhosis consistent with stage F4 fibrosis.


Agents Found By Disease-Level Search

Searched directly from “NAFLD / MASH (Metabolic-Associated Steatohepatitis)” via ClinicalTrials.gov interventions and Open Targets' disease→drug data — independent of the 12-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).

22 agents shown (approved or with trial evidence) — 20 not found by the gene-target search above.

Efimosfermin alfaNOT in gene-target list

· 3 trials · source: ClinicalTrials.gov
NCT07221227 NCT07221227 NCT07221188

ALN-PNPNOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT05648214 NCT07527910

EfruxiferminNOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT06161571 NCT06528314

MiricorilantNOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT07553663 NCT06947304

PegozaferminNOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT06419374 NCT06318169

ECC4703NOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT07288138 NCT07288138

denifanstat (TVB-2640)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04906421

Goserelin Acetate 3.6 mg inj, implantNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06523530

ALG-055009NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06342947

Miricorilant (Cohort A)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06108219

Miricorilant (Cohort B)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06108219

ALN-HSDNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05519475

Incretin-Based TherapyNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06374875

TVB-2640 25 mg (US)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03938246

TVB-2640 50 mg (US)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03938246

TVB-2640 50 mg (China)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03938246

TVB-2640 75 mg (US)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03938246

Tirzepatide

· 1 trial · source: ClinicalTrials.gov
NCT07527910

Digoxin

· 1 trial · source: ClinicalTrials.gov
NCT06588699

DR10624 InjectionNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07024212

ECC0509NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07288138

Menopausal hormone therapyNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06704516

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