Biomarker & Intervention Discovery

NAFLD / MASH (Metabolic-Associated Steatohepatitis)

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

Disease Overview

Spontaneous remission
Not established
Best-intervention remission
Gap size
Primary barrier
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

Recruiting and recently completed trials from ClinicalTrials.gov. Data retrieved 2026-07-03.

NCT07527910 Phase: PHASE2 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...

NCT06138821 Phase: NA Started: 2025-06-24

Metabolic dysfunction-associated steatotic liver disease (MASLD) is the most common chronic liver disease globally. While weight loss through lifestyle modification is the standard treatment, most patients regain weight limiting ultimate improvement in liver disease. On the other end of the spectrum...

NCT06528314 Phase: PHASE3 Started: 2024-09-04

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: PHASE2 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...

NCT06419374 Phase: PHASE3 Started: 2024-05-24

The study will assess the efficacy and safety of pegozafermin administered in participants with compensated cirrhosis due to MASH (biopsy-confirmed fibrosis stage F4 MASH \[previously known as nonalcoholic steatohepatitis, NASH\]).

NCT06318169 Phase: PHASE3 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: PHASE1 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)

NCT06588699 Phase: PHASE2 Started: 2025-06-05

Nonalcoholic steatohepatitis (NASH) is a severe subtype of nonalcoholic fatty liver disease (NAFLD) which affects 1 in 3 Americans. The mainstay of treatment for NASH, which was recently renamed metabolic associated steatohepatitis (MASH), involves lifestyle interventions to promote weight loss and ...

NCT05519475 Phase: PHASE2 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...

NCT06108219 Phase: PHASE2 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 (MONARCH)

NCT06161571 Phase: PHASE3 Started: 2023-11-10

The aim of this study is to assess the safety and tolerability of EFX compared to placebo in subjects with non-invasively diagnosed NASH/MASH and NAFLD/MASLD.

NCT05648214 Phase: PHASE1, PHASE2 Started: 2022-12-27

This study is researching an experimental drug called ALN-PNP (called "study drug"). This is a first in human study. The study drug is not approved by any public health agency such as the United States Food and Drug Administration (FDA) for any kind of treatment. This study consists of 3 parts. Par...

NCT07221227 Phase: PHASE3 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.

NCT07221188 Phase: PHASE3 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.

NCT06947304 Phase: PHASE1 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)


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).

34 agents shown (approved or with trial evidence) — 32 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

Placebo (PB)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05648214

TVB-2640NOT 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

ESG + lifestyle modificationNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06138821

Lifestyle modificationNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06138821

ALG-055009NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06342947

Thermoacoustic Enhanced UltrasoundNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07321925

Liver ultrasoundNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06661655

Miricorilant (Cohort A)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06108219

Placebo (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

Placebo (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

Metabolic surgeryNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06374875

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

Placebo (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

Placebo (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

Standard of care acceptance-based behavioral weight loss programNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06121999

Occupational therapy dietary and lifestyle modificationsNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06121999

Part of the Open Source Medicine Foundation Network

Open Source Medicine Foundation The OSMF hub — advancing open-source biomedical research infrastructure, tools, and evidence synthesis.
SpikeProtein.site Database and research resource on SARS-CoV-2 spike protein biology, persistence, and clinical implications.
Vaccine Data Navigator Open-access navigation tool for vaccine adverse event reporting and surveillance data.
VitalScan4PACVS Remote physiological monitoring study for post-acute COVID-19 vaccination syndrome.
PACVS Research Summit Annual summit convening researchers, clinicians, and patients on PACVS science.