Biomarker & Intervention Discovery

Inflammatory Myopathies (Dermatomyositis / PM / IBM)

This page lists 11 druggable gene targets, 50 approved or trial-tested drug candidates and 15 recent clinical trials for Inflammatory Myopathies (Dermatomyositis / PM / IBM), 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 remissionSpontaneous remission in ~10-15% over 2 years without treatment; IBM (inclusion body myositis) is slowly progressive…
Best-intervention remissionHigh-dose corticosteroids + IVIG + steroid-sparing agent (methotrexate/azathioprine): clinical remission in 40-60% of…
Gap sizeLarge for IBM (no effective treatment); moderate for DM/PM
Primary barrierbiological (IBM) + practice (DM/PM undertreated)
Full remission profile →
Research Tracker Chronic Disease Interventions Inflammatory Myopathies (Dermatomyositis / PM / IBM)

Remission & chronicity

Spontaneous remission
Spontaneous remission in ~10-15% over 2 years without treatment; IBM (inclusion body myositis) is slowly progressive with no effective treatment; death from interstitial lung disease in dermatomyositis in 5-10%
Best-intervention remission
High-dose corticosteroids + IVIG + steroid-sparing agent (methotrexate/azathioprine): clinical remission in 40-60% of DM/PM; IVIG (ProQolity, FDA approved 2021 for DM): 79% response vs 44% placebo (PROPACK trial); rituximab in refractory: 77% response (RIM trial)
Gap size
Large for IBM (no effective treatment); moderate for DM/PM
Primary barrier
biological (IBM) + practice (DM/PM undertreated)
IBM (the most common myopathy in those >60 years) has no effective treatment despite multiple trial failures (bimagrumab, arimoclomol); DM/PM are managed with high-dose steroids first despite substantial side effects and emerging evidence for biologic first-line approaches

Biomarker Targets & Therapeutic Agents

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

C5 23CTLA4 33DHFR 133FCGRT 120HMGCR 214IFNAR1 20IMPDH2 193JAK1 233JAK2 307MS4A1 28NR3C1 264

C5

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
ZILUCOPLAN ↓ inhibits Mechanistic DGIdb
ECULIZUMAB ↓ inhibits Mechanistic DGIdb
TESIDOLUMAB ~ modulates Mechanistic DGIdb
OLENDALIZUMAB ↓ inhibits Mechanistic DGIdb
POZELIMAB ↓ inhibits Mechanistic DGIdb
CROVALIMAB ↓ inhibits Mechanistic DGIdb
AVACINCAPTAD PEGOL ↓ inhibits Mechanistic DGIdb
RAVULIZUMAB ↓ inhibits Mechanistic DGIdb
ZILUCOPLAN SODIUM ~ modulates Mechanistic DGIdb
VILOBELIMAB ↓ inhibits Mechanistic DGIdb
PEXELIZUMAB ↓ inhibits Mechanistic DGIdb
NOMACOPAN ↓ inhibits Mechanistic DGIdb
AVACINCAPTAD PEGOL SODIUM ? unclear Mechanistic Open Targets
CARPROFEN ↓ inhibits Mechanistic 580.0 nM (Kd) ChEMBL
OXAPROZIN ↓ inhibits Mechanistic 640.0 nM (Kd) ChEMBL
SULINDAC ↓ inhibits Mechanistic 570.0 nM (Kd) ChEMBL
RALOXIFENE ↓ inhibits Mechanistic 710.0 nM (Kd) ChEMBL
CHEMBL2204196 ↓ inhibits Mechanistic 440.0 nM (IC50) ChEMBL
CHEMBL2203296 ↓ inhibits Mechanistic 210.0 nM (IC50) ChEMBL
CHEMBL4756677 ↓ inhibits Mechanistic 620.0 nM (IC50) ChEMBL
CHEMBL5594087 ↓ inhibits Mechanistic 940.0 nM (Kd) ChEMBL
CHEMBL5594178 ↓ inhibits Mechanistic 700.0 nM (Kd) ChEMBL
CEMDISIRAN ? unclear Clinical Open Targets

CTLA4

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
TUVONRALIMAB ? unclear Mechanistic DGIdb
RIBAVIRIN ? unclear Mechanistic DGIdb
QUAVONLIMAB ? unclear Mechanistic DGIdb
THALIDOMIDE ? unclear Mechanistic DGIdb
IPILIMUMAB ↓ inhibits Mechanistic DGIdb
METHIMAZOLE ? unclear Mechanistic DGIdb
BLEOMYCIN ? unclear Mechanistic DGIdb
RECOMBINANT FAS LIGAND ? unclear Mechanistic DGIdb
PEGINTERFERON ALFA-2A ? unclear Mechanistic DGIdb
RETRO-INVERSO PEPTIDE ? unclear Mechanistic DGIdb
CYCLOSPORINE ? unclear Mechanistic DGIdb
PEGINTERFERON ALFA-2B ? unclear Mechanistic DGIdb
VISILIZUMAB ? unclear Mechanistic DGIdb
ANTIBIOTIC ? unclear Mechanistic DGIdb
CADONILIMAB ↓ inhibits Mechanistic DGIdb
PREDNISONE ? unclear Mechanistic DGIdb
ERFONRILIMAB ↓ inhibits Mechanistic DGIdb
ANTI-THYMOCYTE GLOBULIN ? unclear Mechanistic DGIdb
ZALIFRELIMAB ↓ inhibits Mechanistic DGIdb
WORTMANNIN ? unclear Mechanistic DGIdb
FF-10501-01 ? unclear Mechanistic DGIdb
ATEZOLIZUMAB ? unclear Mechanistic DGIdb
DEHYDRATED ALCOHOL ? unclear Mechanistic DGIdb
MELPHALAN ? unclear Mechanistic DGIdb
TETRADECANOYLPHORBOL ACETATE ? unclear Mechanistic DGIdb
ABATACEPT ? unclear Mechanistic DGIdb
NURULIMAB ? unclear Mechanistic DGIdb
INOSINE 5'-MONOPHOSPHATE DEHYDROGENASE INHIBITOR FF-10501-01 ? unclear Mechanistic DGIdb
TREMELIMUMAB ↓ inhibits Mechanistic DGIdb
BISPECIFIC ANTIBODY ? unclear Mechanistic DGIdb
SIROLIMUS ? unclear Mechanistic DGIdb
LORIGERLIMAB ? unclear Mechanistic DGIdb
CHEMBL5291170 ↓ inhibits Mechanistic 31000.0 nM (Kd) ChEMBL

DHFR

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
PRALATREXATE ↓ inhibits Mechanistic DGIdb
PEMETREXED TROMETHAMINE ↓ inhibits Mechanistic DGIdb
PEMETREXED DISODIUM ↓ inhibits Mechanistic DGIdb
TAMOXIFEN CITRATE ? unclear Mechanistic DGIdb
METHOTREXATE ↓ inhibits Mechanistic 0.82 nM (IC50) DGIdb
CH-1504 ? unclear Mechanistic DGIdb
PIRITREXIM ↓ inhibits Mechanistic 33.0 nM (Ki) DGIdb
CH-4051 ? unclear Mechanistic DGIdb
PERFOSFAMIDE ? unclear Mechanistic DGIdb
RALTITREXED ? unclear Mechanistic DGIdb
AZATHIOPRINE ? unclear Mechanistic DGIdb
METHOTREXATE SODIUM ↓ inhibits Mechanistic DGIdb
TRIMETREXATE ↓ inhibits Mechanistic 90.0 nM (IC50) DGIdb
PEMETREXED DIPOTASSIUM ↓ inhibits Mechanistic DGIdb
AMINOSALICYLIC ACID ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL22148 ? unclear Mechanistic DGIdb
ICLAPRIM ↓ inhibits Mechanistic DGIdb
ANTINEOPLASTIC ANTIBIOTIC ? unclear Mechanistic DGIdb
TALOTREXIN ? unclear Mechanistic DGIdb
COMPOUND 9 [PMID: 15615544] ↓ inhibits Mechanistic DGIdb
LEUCOVORIN CALCIUM ? unclear Mechanistic DGIdb
PYRIMETHAMINE ? unclear Mechanistic 250.0 nM (Ki) DGIdb
FOLATE ANTAGONIST ? unclear Mechanistic DGIdb
SULFASALAZINE ? unclear Mechanistic DGIdb
COBALAMIN ? unclear Mechanistic DGIdb
TRIMETHOPRIM ? unclear Mechanistic 194.98 nM (Ki) DGIdb
CYCLOSPORINE ? unclear Mechanistic DGIdb
FOLIC ACID ? unclear Mechanistic DGIdb
DIFLUNISAL ↓ inhibits Mechanistic DGIdb
MERCAPTOPURINE ? unclear Mechanistic DGIdb
PEMETREXED MONOHYDRATE ↓ inhibits Mechanistic DGIdb
MITOMYCIN ? unclear Mechanistic DGIdb
PEMETREXED ? unclear Mechanistic 2300.0 nM (IC50) Open Targets
CHEMBL7130 ↓ inhibits Mechanistic 93.33 nM (Ki) ChEMBL
CHEMBL33697 ↓ inhibits Mechanistic 22.39 nM (Ki) ChEMBL
CHEMBL281618 ↓ inhibits Mechanistic 776.25 nM (Ki) ChEMBL
CHEMBL20975 ↓ inhibits Mechanistic 42.66 nM (Ki) ChEMBL
CHEMBL20587 ↓ inhibits Mechanistic 218.78 nM (Ki) ChEMBL
CHEMBL6742 ↓ inhibits Mechanistic 1659.59 nM (Ki) ChEMBL
CHEMBL176987 ↓ inhibits Mechanistic 977.24 nM (Ki) ChEMBL

Showing top 40 of 133 agents ranked by evidence tier.

FCGRT

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
EFGARTIGIMOD ALFA ↓ inhibits Mechanistic DGIdb
BATOCLIMAB ? unclear Mechanistic DGIdb
DX-2507 ? unclear Mechanistic DGIdb
ZILTIVEKIMAB ? unclear Mechanistic DGIdb
NIPOCALIMAB ↓ inhibits Mechanistic DGIdb
ROZANOLIXIZUMAB ↓ inhibits Mechanistic DGIdb
ORILANOLIMAB ↓ inhibits Mechanistic DGIdb
CHEMBL508564 ↓ inhibits Mechanistic 1400.0 nM (IC50) ChEMBL
CHEMBL508411 ↓ inhibits Mechanistic 36000.0 nM (IC50) ChEMBL
CHEMBL505689 ↓ inhibits Mechanistic 87000.0 nM (IC50) ChEMBL
CHEMBL524509 ↓ inhibits Mechanistic 31000.0 nM (IC50) ChEMBL
CHEMBL503730 ↓ inhibits Mechanistic 34000.0 nM (IC50) ChEMBL
CHEMBL526683 ↓ inhibits Mechanistic 67000.0 nM (IC50) ChEMBL
CHEMBL207341 ↓ inhibits Mechanistic 24000.0 nM (IC50) ChEMBL
CHEMBL451944 ↓ inhibits Mechanistic 27000.0 nM (IC50) ChEMBL
CHEMBL506507 ↓ inhibits Mechanistic 40000.0 nM (IC50) ChEMBL
CHEMBL509774 ↓ inhibits Mechanistic 33000.0 nM (IC50) ChEMBL
PSYCIEGHIDGIYCFNA ↓ inhibits Mechanistic 49000.0 nM (IC50) ChEMBL
CHEMBL503219 ↓ inhibits Mechanistic 64000.0 nM (IC50) ChEMBL
CHEMBL499790 ↓ inhibits Mechanistic 33000.0 nM (IC50) ChEMBL
CHEMBL444514 ↓ inhibits Mechanistic 20000.0 nM (Kd) ChEMBL
CHEMBL525216 ↓ inhibits Mechanistic 34000.0 nM (Kd) ChEMBL
CHEMBL499664 ↓ inhibits Mechanistic 69000.0 nM (IC50) ChEMBL
CHEMBL499402 ↓ inhibits Mechanistic 57000.0 nM (IC50) ChEMBL
CHEMBL506644 ↓ inhibits Mechanistic 48000.0 nM (IC50) ChEMBL
CHEMBL525265 ↓ inhibits Mechanistic 420.0 nM (IC50) ChEMBL
CHEMBL503983 ↓ inhibits Mechanistic 1000.0 nM (IC50) ChEMBL
CHEMBL504423 ↓ inhibits Mechanistic 2100.0 nM (IC50) ChEMBL
CHEMBL525614 ↓ inhibits Mechanistic 1600.0 nM (IC50) ChEMBL
CHEMBL505899 ↓ inhibits Mechanistic 900.0 nM (IC50) ChEMBL
CHEMBL511030 ↓ inhibits Mechanistic 27000.0 nM (IC50) ChEMBL
CHEMBL446284 ↓ inhibits Mechanistic 2000.0 nM (IC50) ChEMBL
CHEMBL510716 ↓ inhibits Mechanistic 1700.0 nM (IC50) ChEMBL
CHEMBL504919 ↓ inhibits Mechanistic 16000.0 nM (IC50) ChEMBL
CHEMBL499766 ↓ inhibits Mechanistic 2000.0 nM (IC50) ChEMBL
CHEMBL524659 ↓ inhibits Mechanistic 2000.0 nM (IC50) ChEMBL
CHEMBL526861 ↓ inhibits Mechanistic 18000.0 nM (IC50) ChEMBL
CHEMBL506647 ↓ inhibits Mechanistic 2000.0 nM (IC50) ChEMBL
CHEMBL504226 ↓ inhibits Mechanistic 20000.0 nM (IC50) ChEMBL
CHEMBL507004 ↓ inhibits Mechanistic 21000.0 nM (IC50) ChEMBL

Showing top 40 of 120 agents ranked by evidence tier.

HMGCR

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
SIMVASTATIN ↓ inhibits Mechanistic 2.6 nM (Ki) DGIdb
FLUVASTATIN ↓ inhibits Mechanistic 28.0 nM (IC50) DGIdb
PRAVASTATIN SODIUM ↓ inhibits Mechanistic 1370.0 nM (IC50) DGIdb
CERIVASTATIN ↓ inhibits Mechanistic 10.0 nM (IC50) DGIdb
COMPOUND 9 [PMID: 1992149] ↓ inhibits Mechanistic DGIdb
COMPOUND 2T [PMID: 2296036] ↓ inhibits Mechanistic DGIdb
COMPOUND 18 [PMID: 8426367] ↓ inhibits Mechanistic DGIdb
COMPOUND 9A [PATIL ET AL., 1989] ↓ inhibits Mechanistic DGIdb
CRILVASTATIN ? unclear Mechanistic DGIdb
PITAVASTATIN ? unclear Mechanistic DGIdb
LAROPIPRANT ? unclear Mechanistic DGIdb
LOVASTATIN ↓ inhibits Mechanistic 37.0 nM (IC50) DGIdb
DFGYVAE ↓ inhibits Mechanistic DGIdb
COMPOUND 2F [PMID: 2153213] ↓ inhibits Mechanistic DGIdb
FLUVASTATIN SODIUM ↓ inhibits Mechanistic DGIdb
COMPOUND 4FF [PMID: 1656041] ↓ inhibits Mechanistic DGIdb
ATORVASTATIN ↓ inhibits Mechanistic 6.0 nM (IC50) DGIdb
PITAVASTATIN CALCIUM ↓ inhibits Mechanistic DGIdb
COMPOUND 17 [PMID: 15686906] ↓ inhibits Mechanistic DGIdb
COMPOUND 41 [PMID: 17574411] ↓ inhibits Mechanistic DGIdb
ASPIRIN ? unclear Mechanistic DGIdb
COMPOUND 1A [PMID: 2153213] ↓ inhibits Mechanistic DGIdb
ATORVASTATIN CALCIUM ↓ inhibits Mechanistic DGIdb
COMPOUND 2D [PMID: 2153213] ↓ inhibits Mechanistic DGIdb
FPYVAE PEPTIDE [PMID: 20494585] ↓ inhibits Mechanistic DGIdb
COMPOUND 4I [PMID: 2296036] ↓ inhibits Mechanistic DGIdb
HMG-COA REDUCTASE INHIBITOR ? unclear Mechanistic DGIdb
CERIVASTATIN SODIUM ↓ inhibits Mechanistic DGIdb
GLPDGG PEPTIDE [PMID: 20494585] ↓ inhibits Mechanistic DGIdb
COMPOUND 6A [PATIL ET AL., 1989] ↓ inhibits Mechanistic DGIdb
COMPOUND 11NN [PMID: 1656041] ↓ inhibits Mechanistic DGIdb
COMPOUND 2 [DREYER ET AL., 1991] ↓ inhibits Mechanistic DGIdb
TERIFLUNOMIDE ? unclear Mechanistic DGIdb
ROSUVASTATIN CALCIUM ↓ inhibits Mechanistic DGIdb
MEVASTATIN ↓ inhibits Mechanistic 28.4 nM (IC50) DGIdb
COMPOUND 29 [PMID: 1527791] ↓ inhibits Mechanistic DGIdb
COMPOUND 11JJ [PMID: 1656041] ↓ inhibits Mechanistic DGIdb
PITAVASTATIN SODIUM ↓ inhibits Mechanistic DGIdb
COMPOUND 3 [DREYER ET AL., 1991] ↓ inhibits Mechanistic DGIdb
ROSUVASTATIN ↓ inhibits Mechanistic 0.9 nM (Ki) DGIdb

Showing top 40 of 214 agents ranked by evidence tier.

IFNAR1

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
INTERFERON ALFA-N3 ↑ activates Mechanistic DGIdb
INTERFERON BETA-1A ↑ activates Mechanistic DGIdb
INTERFERON ALFA-N1 ? unclear Mechanistic DGIdb
ANIFROLUMAB ? unclear Mechanistic DGIdb
INTERFERON BETA ? unclear Mechanistic DGIdb
PEGINTERFERON ALFA-2A ↑ activates Mechanistic DGIdb
PEGINTERFERON BETA-1A ↑ activates Mechanistic DGIdb
INTERFERON ALFA-2A ↑ activates Mechanistic DGIdb
SIFALIMUMAB ? unclear Mechanistic DGIdb
INTERFERON-ALPHA ? unclear Mechanistic DGIdb
INTERFERON ALFACON-1 ↑ activates Mechanistic DGIdb
IFN-Β ↑ activates Mechanistic DGIdb
ALBINTERFERON ALFA-2B ↑ activates Mechanistic DGIdb
PEGINTERFERON ALFACON-1 ? unclear Mechanistic DGIdb
GLATIRAMER ? unclear Mechanistic DGIdb
INTERFERON BETA-1B ↑ activates Mechanistic DGIdb
PEGINTERFERON ALFA-2B ↑ activates Mechanistic DGIdb
INTERFERON ALFA-2B ↑ activates Mechanistic DGIdb
ROPEGINTERFERON ALFA-2B ~ modulates Mechanistic DGIdb
CHEMBL1617534 ↓ inhibits Mechanistic 4000.0 nM (Kd) ChEMBL

IMPDH2

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
THIOGUANINE ↓ inhibits Mechanistic DGIdb
MYCOPHENOLATE MOFETIL ↓ inhibits Mechanistic DGIdb
AVN-944 ↓ inhibits Mechanistic DGIdb
MYCOPHENOLATE ↓ inhibits Mechanistic DGIdb
CHEMBL:CHEMBL1683024 ? unclear Mechanistic DGIdb
MIZORIBINE ↓ inhibits Mechanistic DGIdb
MERIMEPODIB ↓ inhibits Mechanistic 10.0 nM (IC50) DGIdb
RIBAVIRIN ↓ inhibits Mechanistic DGIdb
AZATHIOPRINE ? unclear Mechanistic DGIdb
MYCOPHENOLATE MOFETIL HYDROCHLORIDE ↓ inhibits Mechanistic DGIdb
CYCLOSPORINE ? unclear Mechanistic DGIdb
MYCOPHENOLATE SODIUM ↓ inhibits Mechanistic DGIdb
MYCOPHENOLIC ACID ? unclear Mechanistic 10.0 nM (Ki) Open Targets
CHEMBL27948 ↓ inhibits Mechanistic 18.0 nM (IC50) ChEMBL
CHEMBL29926 ↓ inhibits Mechanistic 400.0 nM (IC50) ChEMBL
CHEMBL3144029 ↓ inhibits Mechanistic 110.0 nM (Ki) ChEMBL
CHEMBL348359 ↓ inhibits Mechanistic 470.0 nM (Ki) ChEMBL
CHEMBL416020 ↓ inhibits Mechanistic 1200.0 nM (IC50) ChEMBL
CHEMBL27528 ↓ inhibits Mechanistic 3800.0 nM (IC50) ChEMBL
CHEMBL26741 ↓ inhibits Mechanistic 150.0 nM (IC50) ChEMBL
CHEMBL27011 ↓ inhibits Mechanistic 550.0 nM (IC50) ChEMBL
CHEMBL27529 ↓ inhibits Mechanistic 740.0 nM (IC50) ChEMBL
CHEMBL282102 ↓ inhibits Mechanistic 3000.0 nM (IC50) ChEMBL
CHEMBL608195 ↓ inhibits Mechanistic 290.0 nM (Ki) ChEMBL
CHEMBL410744 ↓ inhibits Mechanistic 260.0 nM (Ki) ChEMBL
CHEMBL29927 ↓ inhibits Mechanistic 247.0 nM (IC50) ChEMBL
CHEMBL26155 ↓ inhibits Mechanistic 820.0 nM (IC50) ChEMBL
CHEMBL26258 ↓ inhibits Mechanistic 740.0 nM (IC50) ChEMBL
CHEMBL26419 ↓ inhibits Mechanistic 1660.0 nM (IC50) ChEMBL
CHEMBL608194 ↓ inhibits Mechanistic 950.0 nM (Ki) ChEMBL
CHEMBL162782 ↓ inhibits Mechanistic 400.0 nM (Ki) ChEMBL
CHEMBL285813 ↓ inhibits Mechanistic 60.0 nM (IC50) ChEMBL
CHEMBL2311206 ↓ inhibits Mechanistic 380.0 nM (Ki) ChEMBL
CHEMBL280793 ↓ inhibits Mechanistic 5.0 nM (IC50) ChEMBL
CHEMBL3144028 ↓ inhibits Mechanistic 300.0 nM (Ki) ChEMBL
CHEMBL305977 ↓ inhibits Mechanistic 22.0 nM (IC50) ChEMBL
CHEMBL67346 ↓ inhibits Mechanistic 25.0 nM (IC50) ChEMBL
CHEMBL302967 ↓ inhibits Mechanistic 17.0 nM (IC50) ChEMBL
CHEMBL417047 ↓ inhibits Mechanistic 20.0 nM (IC50) ChEMBL
CHEMBL64830 ↓ inhibits Mechanistic 12.0 nM (IC50) ChEMBL

Showing top 40 of 193 agents ranked by evidence tier.

JAK1

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
TRICETAMIDE ↓ inhibits Mechanistic DGIdb
NEZULCITINIB ↓ inhibits Mechanistic DGIdb
IVARMACITINIB ↓ inhibits Mechanistic DGIdb
OCLACITINIB ↓ inhibits Mechanistic DGIdb
MOMELOTINIB ↓ inhibits Mechanistic 11.0 nM (IC50) DGIdb
DECERNOTINIB ↓ inhibits Mechanistic DGIdb
GOLIDOCITINIB ↓ inhibits Mechanistic DGIdb
UPADACITINIB ↓ inhibits Mechanistic DGIdb
LEPZACITINIB ↓ inhibits Mechanistic DGIdb
BREPOCITINIB ↓ inhibits Mechanistic DGIdb
COMPOUND 30 [PMID: 37057760] ↓ inhibits Mechanistic DGIdb
WXFL10203614 ↓ inhibits Mechanistic DGIdb
LIRUCITINIB ↓ inhibits Mechanistic DGIdb
VEXICITINIB ↓ inhibits Mechanistic DGIdb
ILGINATINIB ↓ inhibits Mechanistic DGIdb
COMPOUND 5E [PMID: 28580438] ↓ inhibits Mechanistic DGIdb
COMPOUND 25AP [PMID: 37796543] ↓ inhibits Mechanistic DGIdb
ZEMPROCITINIB ↓ inhibits Mechanistic DGIdb
PEMBROLIZUMAB ? unclear Mechanistic DGIdb
AZD1480 ↓ inhibits Mechanistic DGIdb
CERDULATINIB ↓ inhibits Mechanistic DGIdb
LONDAMOCITINIB ↓ inhibits Mechanistic DGIdb
JAK INHIBITOR 20A ↓ inhibits Mechanistic DGIdb
FILGOTINIB ↓ inhibits Mechanistic DGIdb
GUSACITINIB ↓ inhibits Mechanistic DGIdb
COMPOUND 8L [PMID: 36053746] ↓ inhibits Mechanistic DGIdb
COMPOUND 13AC [PMID: 33256400] ↓ inhibits Mechanistic DGIdb
FEDRATINIB ↓ inhibits Mechanistic 100.0 nM (IC50) DGIdb
FILGOTINIB MALEATE ↓ inhibits Mechanistic DGIdb
PF-956980 ↓ inhibits Mechanistic DGIdb
SDC-1801 ↓ inhibits Mechanistic DGIdb
ROPSACITINIB ↓ inhibits Mechanistic DGIdb
COMPOUND 18E [PMID: 31670517] ↓ inhibits Mechanistic DGIdb
UPADACITINIB HEMIHYDRATE ↓ inhibits Mechanistic DGIdb
BLOVACITINIB ↓ inhibits Mechanistic DGIdb
DEURUXOLITINIB ↓ inhibits Mechanistic DGIdb
RUXOLITINIB PHOSPHATE ↓ inhibits Mechanistic DGIdb
SOLCITINIB ↓ inhibits Mechanistic DGIdb
CEE321 ↓ inhibits Mechanistic DGIdb
NIK INHIBITOR 12F ↓ inhibits Mechanistic DGIdb

Showing top 40 of 233 agents ranked by evidence tier.

JAK2

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
CEP-33779 ↓ inhibits Mechanistic DGIdb
ITACNOSERTIB ↓ inhibits Mechanistic DGIdb
FILGOTINIB ↓ inhibits Mechanistic DGIdb
CEE321 ↓ inhibits Mechanistic DGIdb
MOMELOTINIB ↓ inhibits Mechanistic DGIdb
GILTERITINIB ? unclear Mechanistic DGIdb
PEMBROLIZUMAB ? unclear Mechanistic DGIdb
NEZULCITINIB ↓ inhibits Mechanistic DGIdb
GANDOTINIB ↓ inhibits Mechanistic DGIdb
PACRITINIB ↓ inhibits Mechanistic DGIdb
CEP-11981 ? unclear Mechanistic DGIdb
GUSACITINIB ↓ inhibits Mechanistic DGIdb
ITACITINIB ↓ inhibits Mechanistic DGIdb
ENTRECTINIB ? unclear Mechanistic DGIdb
MIDOSTAURIN ? unclear Mechanistic 94.0 nM (Kd) DGIdb
TOFACITINIB CITRATE ↓ inhibits Mechanistic DGIdb
DOVITINIB ? unclear Mechanistic 7000.0 nM (Kd) DGIdb
6-AZAURIDINE ? unclear Mechanistic DGIdb
BOMEDEMSTAT ? unclear Mechanistic DGIdb
AT-9283 ↓ inhibits Mechanistic 1.2 nM (IC50) DGIdb
UPADACITINIB ↓ inhibits Mechanistic DGIdb
RUXOLITINIB ↓ inhibits Mechanistic DGIdb
TOZASERTIB ? unclear Mechanistic 190.0 nM (Kd) DGIdb
AZD1208 ? unclear Mechanistic DGIdb
DECERNOTINIB ↓ inhibits Mechanistic DGIdb
RG-1530 ↓ inhibits Mechanistic DGIdb
HG-10-102-01 ↓ inhibits Mechanistic DGIdb
ROVADICITINIB ↓ inhibits Mechanistic DGIdb
JAK3 INHIBITOR II ↓ inhibits Mechanistic DGIdb
IZENCITINIB ↓ inhibits Mechanistic DGIdb
EMZELTRECTINIB ↓ inhibits Mechanistic DGIdb
FEDRATINIB ↓ inhibits Mechanistic DGIdb
LESTAURTINIB ↓ inhibits Mechanistic DGIdb
OLAPARIB ? unclear Mechanistic DGIdb
FEDRATINIB HYDROCHLORIDE ↓ inhibits Mechanistic DGIdb
JNJ-7706621 ↓ inhibits Mechanistic 220.0 nM (Kd) DGIdb
TOFACITINIB ↓ inhibits Mechanistic 11.0 nM (IC50) DGIdb
DELGOCITINIB ↓ inhibits Mechanistic DGIdb
BINIMETINIB ? unclear Mechanistic DGIdb
BIIB068 ↓ inhibits Mechanistic DGIdb

Showing top 40 of 307 agents ranked by evidence tier.

MS4A1

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
RITUXIMAB ~ modulates Mechanistic DGIdb
UBLITUXIMAB ~ modulates Mechanistic DGIdb
VELTUZUMAB ? unclear Mechanistic DGIdb
OFATUMUMAB ~ modulates Mechanistic DGIdb
OBINUTUZUMAB ~ modulates Mechanistic DGIdb
OCARATUZUMAB ~ modulates Mechanistic DGIdb
TOSITUMOMAB ~ modulates Mechanistic DGIdb
TISAGENLECLEUCEL ? unclear Mechanistic DGIdb
IODINE-131-TOSITUMOMAB ? unclear Mechanistic DGIdb
EPCORITAMAB ? unclear Mechanistic DGIdb
GLOFITAMAB ~ modulates Mechanistic DGIdb
TRU-015 ~ modulates Mechanistic DGIdb
ODRONEXTAMAB ? unclear Mechanistic DGIdb
OCRELIZUMAB ~ modulates Mechanistic DGIdb
ANTI-CD20 CAR T CELLS PREPARATION ? unclear Mechanistic DGIdb
DI-LEU16-IL2 ? unclear Mechanistic DGIdb
MONOCLONAL ANTIBODY 1F5 ? unclear Mechanistic DGIdb
MT-3724 ~ modulates Mechanistic DGIdb
MOSUNETUZUMAB ? unclear Mechanistic DGIdb
PLAMOTAMAB ? unclear Mechanistic DGIdb
AUTOLOGOUS ANTI-CD20 CAR TRANSDUCED CD4/CD8 ENRICHED T-CELLS MB-CART20.1 ? unclear Mechanistic DGIdb
FBT-A05 ~ modulates Mechanistic DGIdb
YTTRIUM Y 90 IBRITUMOMAB TIUXETAN ~ modulates Mechanistic DGIdb
BSAB (CD89-CD20) ? unclear Mechanistic DGIdb
LFB-R603 ~ modulates Mechanistic DGIdb
DIVOZILIMAB ? unclear Mechanistic DGIdb
IBRITUMOMAB TIUXETAN ? unclear Mechanistic DGIdb
TOSITUMOMAB 131I ~ modulates Mechanistic DGIdb

NR3C1

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
METHYLPREDNISOLONE ↑ activates Mechanistic DGIdb
FOSDAGROCORAT ↑ activates Mechanistic DGIdb
ZK216348 ↑ activates Mechanistic DGIdb
PHENYLMERCURIC ACETATE ? unclear Mechanistic DGIdb
TNF-ALPHA ? unclear Mechanistic DGIdb
HYDROCORTAMATE HYDROCHLORIDE ↑ activates Mechanistic DGIdb
TRIAMCINOLONE ↑ activates Mechanistic DGIdb
BUDESONIDE ↑ activates Mechanistic DGIdb
ANTICONVULSANT AGENT ? unclear Mechanistic DGIdb
FLUTICASONE FUROATE ↑ activates Mechanistic DGIdb
METHYLPREDNISOLONE ACETATE ↑ activates Mechanistic DGIdb
DAGROCORAT ↑ activates Mechanistic DGIdb
MIFEPRISTONE ↓ inhibits Mechanistic 0.24 nM (Ki) DGIdb
TESTOSTERONE PROPIONATE ? unclear Mechanistic DGIdb
FLURANDRENOLIDE ↑ activates Mechanistic DGIdb
CHEMBL:CHEMBL1915271 ? unclear Mechanistic DGIdb
CHRYSIN ? unclear Mechanistic DGIdb
FLUOXYMESTERONE ? unclear Mechanistic DGIdb
LOTEPREDNOL ↑ activates Mechanistic DGIdb
DIFLUPREDNATE ↑ activates Mechanistic DGIdb
CORT 108297 ↓ inhibits Mechanistic DGIdb
PROGESTERONE ? unclear Mechanistic 30.5 nM (Ki) DGIdb
BETAMETHASONE SODIUM PHOSPHATE ↑ activates Mechanistic DGIdb
ANTIDEPRESSANT AGENT ? unclear Mechanistic DGIdb
HYDROCORTISONE BUTYRATE ↑ activates Mechanistic DGIdb
VELSECORAT ↑ activates Mechanistic DGIdb
FLUMETHASONE PIVALATE ↑ activates Mechanistic DGIdb
AMCINONIDE ↑ activates Mechanistic DGIdb
CYPROTERONE ACETATE ↓ inhibits Mechanistic DGIdb
C108297 ↑ activates Mechanistic DGIdb
COMPOUND 17 [PMID: 29741897] ↑ activates Mechanistic DGIdb
FLUOCINONIDE ↑ activates Mechanistic DGIdb
MAPRACORAT ↑ activates Mechanistic DGIdb
CLOBETASOL PROPIONATE ↑ activates Mechanistic DGIdb
CHEMBL:CHEMBL210411 ? unclear Mechanistic DGIdb
FLUOCINOLONE ACETONIDE ↑ activates Mechanistic DGIdb
BETAMETHASONE ↑ activates Mechanistic DGIdb
BECLOMETHASONE DIPROPIONATE ↑ activates Mechanistic DGIdb
FLUNISOLIDE ANHYDROUS ↑ activates Mechanistic DGIdb
CORTISONE ACETATE ↑ activates Mechanistic DGIdb

Showing top 40 of 264 agents ranked by evidence tier.

Clinical Trials

Drug, biologic and supplement trials (treatment or prevention) from ClinicalTrials.gov whose main condition is Inflammatory Myopathies (Dermatomyositis / PM / IBM): recruiting, active or completed, most recently updated first. Data retrieved 2026-10-28.

Topical Ruxolitinib Cream for Refractory Cutaneous Dermatomyositis Recruiting
NCT06857240 Phase: 2 Started: 2025-06-01

This study will assess the safety and efficacy of topical ruxolitinib for treating the refractory cutaneous manifestations in patients with dermatomyositis. The investigators' hypothesis is that topical ruxolitinib will be both safe and effective for such patients.

FAST for DM - Fatty Acid Supplementation Trial (FAST) for Dermatomyositis (DM) Recruiting
NCT07111065 Phase: 2 Started: 2026-11-02

Dermatomyositis (DM) is a rare autoimmune disease that causes muscle weakness, skin rashes, and other symptoms. Researchers think both genetic and environmental factors play a role in this disease. They want to find out more about how diet and lifestyle choices affect people with DM/JDM. Objective: ...

CARTIMMUNE: Study of Patients With Autoimmune Diseases Receiving KYV-101 Active Not Recruiting
NCT06152172 Phase: 1 Started: 2024-08-05

The purpose of this study is to assess the safety, tolerability, and clinical activity of KYV 101 (a fully-human anti-CD19 CAR T-cell therapy) in adult subjects with B cell-driven autoimmune diseases. The trial anticipates enrolling participants to reach a maximum of 24 participants who will receive...

A Study to Investigate the Efficacy and Safety of Brepocitinib in Adults With Dermatomyositis Completed
NCT05437263 Phase: 3 Started: 2022-10-31

This is a phase 3, multicenter, randomized, placebo-controlled, double-blind study of treatment with brepocitinib (TYK2/JAK1 inhibitor) in adults with dermatomyositis (DM). The primary objective of this study is to assess the efficacy of two dose levels of brepocitinib in comparison to placebo, as m...

Emapalumab MDA5 Rapidly Progressive Interstitial Lung Disease (RP-ILD) Study Recruiting
NCT07486869 Phase: 2 Started: 2026-07-23

This is a proof of concept study to determine if Emapalumab appears effective for the treatment of anti-MDA5 antibody positive rapidly progressive interstitial lung disease (MDA5 RP-ILD). Emapalumab is a medication that is currently used for a severe problem with the immune system, called macrophage...

A Study to Understand How the Study Medicine Dazukibart Works in People With Idiopathic Inflammatory Myopathies Recruiting
NCT06698796 Phase: 3 Started: 2025-01-22

The purpose of this study is to understand how the study medicine, dazukibart, works in people with active idiopathic inflammatory myopathies (dermatomyositis \[DM\] or polymyositis \[PM\]). Idiopathic inflammatory myopathies are a group of disorders that show inflammation of the muscles used for mo...

Phase 2 Study of Rapcabtagene Autoleucel in Myositis Active Not Recruiting
NCT06665256 Phase: 2 Started: 2024-12-17

A Phase 2, randomized, open-label, controlled study to evaluate the efficacy and safety of rapcabtagene autoleucel versus comparator in participants with severe refractory idiopathic inflammatory myopathies (IIM)

Baricitinib in Patients With Relapsing or naïve Dermatomyositis Recruiting
NCT04972760 Phase: 3 Started: 2022-08-31

Dermatomyositis (DM) is a rare and disabling condition with an important impairment of quality of life and possible life-threatening complications. Treatment is based on high doses of corticosteroids but this exposes patients to adverse events (cardiovascular mortality, glucocorticoids-induced muscl...

A Study Evaluating the Effects of GLPG3667 Given as Oral Treatment for up to 24 Weeks in Adults With Dermatomyositis Completed
NCT05695950 Phase: 2 Started: 2023-02-27

The purpose of this study is to evaluate the efficacy, safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of orally administered GLPG3667 once daily for 24 weeks in adult participants with dermatomyositis (DM), followed by an open-label extension (OLE) period until Week 48.

Baricitinib in the Treatment of New-onset Juvenile Dermatomyositis (MYOCIT) Completed
NCT05524311 Phase: 2 Started: 2022-11-10

The MYOCIT study aims to evaluate the efficacy and safety of baricitinib in association with corticosteroids in new-onset patients with juvenile dermatomyositis (JDM) in a phase II trial with the objective to obtain a better efficacy than the conventional combination methotrexate (MTX) and corticost...

RCT of Tocilizumab for Anti-MDA5+DM Recruiting
NCT07377058 Phase: N/A Started: 2025-11-01

The goal of this clinical trial is to learn if tocilizumab works to treat anti-MDA5+ dermatomyositis (anti-MDA5+DM) in adults. It will also learn about the safety of tocilizumab. The main questions it aims to answer are: Does tocilizumab improve patients' clinical symptoms? Does tocilizumab improve ...

A Study to Evaluate Safety and Efficacy of Empasiprubart in Adults With Dermatomyositis Active Not Recruiting
NCT06284954 Phase: 2 Started: 2024-08-20

This study will evaluate the safety and efficacy of empasiprubart compared with placebo in adult participants with dermatomyositis (DM). The study duration will be approximately 92 weeks for all participants. After the screening period, eligible participants will be randomized in a 2:1 ratio to rece...

Clinical Study of BCMA/CD70-targeted CAR-T Therapy for Refractory Pediatric Rheumatic Diseases Recruiting
NCT07184450 Phase: 1 Started: 2025-09-01

This is an investigator-initiated trial to evaluate the efficacy and safety of BCMA/CD70-targeted CAR-T in the treatment of refractory pediatric rheumatic diseases.

An Exploratory Study of CD19/CD22/BCMA CAR-T Cells (BZE2204) in Subjects With Relapsed or Refractory Autoimmune Diseases Recruiting
NCT07174843 Phase: Early 1 Started: 2025-09

This is a single arm, open-label, dose escalation and expansion study to evaluate the safety, tolerability and preliminary efficacy of autologous chimeric antigen receptor T (CAR-T) cells targeting CD19/CD22/BCMA(BZE2204) in patients with relapsed or refractory active autoimmune diseases, including ...

Long-term Extension Study of Ulviprubart (ABC008) in Subjects With Inclusion Body Myositis Active Not Recruiting
NCT06450886 Phase: 2/3 Started: 2024-10-21

ABC008-IBM-202 is an open-label, multicenter study to evaluate the safety and efficacy of long-term administration of ulviprubart (ABC008) in subjects with IBM who have completed either Study ABC008-IBM-101 or Study ABC008-IBM-201. Subjects may be enrolled in this study if they meet study eligibilit...

Agents Found By Disease-Level Search

Searched directly from “Inflammatory Myopathies (Dermatomyositis / PM / IBM)” via ClinicalTrials.gov interventions and Open Targets' disease→drug data — independent of the 11-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).

50 agents shown (approved or with trial evidence) — 48 not found by the gene-target search above.

FludarabineNOT in gene-target list

· 3 trials · source: ClinicalTrials.gov
NCT06733935 NCT06308978 NCT07115745

CyclophosphamideNOT in gene-target list

Phase 1 · 3 trials · source: ClinicalTrials.gov, Open Targets
NCT06733935 NCT06308978 NCT07115745

Baricitinib

Phase 2 · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT04972760 NCT05524311

anti-CD19 CAR T cell therapyNOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT06828042 NCT06347718

EtanerceptNOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT00802815 NCT00112385

ICG318, BCMA-CD19-IL-15/IL-15 sushi Compound CAR-TNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07715136

bupivacaine and acupunctureNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00635037

CD34 selectionNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00010335

CabozantinibNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03816345

FluoropyrimidineNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03816345

Ipilimumab

· 1 trial · source: ClinicalTrials.gov
NCT03816345

NivolumabNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03816345

Platinum CompoundNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03816345

Platinum DoubletNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03816345

Non intervention studyNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03059394

omega 7NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03669575

Octagam 10%NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02728752

Kinesio TapingNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02501915

Apremilast 30mgNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03529955

Cytosorb therapyNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04913298

Abatacept Active TreatmentNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01315938

Abatacept Delayed-Onset TreatmentNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01315938

pharmacokinetics studyNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05524311

dosage of cytokinesNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05524311

transcriptomic analysisNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05524311

CytoSorbNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05146336

FT839NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07800871

CD19 or CD19-BCMA CAR-TNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06548607

YTS109 cellNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07123519

NKX019NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06733935

Single nucleotide polymorphisms (SNP)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06696027

3'mRNA sequencingNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06696027

Enzyme-linked immunosorbent assay (ELISA) and Legendplex ArrayNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06696027

Flow CytometryNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06696027

Sodium Thiosulfate (STS)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06672822

RapamycinNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02481453

DIMAC02NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02789800

Anti-Beta Interferon (PF-06823859)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05192200

Shoulder arthroplastyNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03573765

Elbow arthroplastyNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03573765

Hand and wrist arthroplastyNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03573765

Nerve decompressionNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03573765

Tendon repairNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03573765

Joint stabilisationNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03573765

Excision/division/release of palmar fasciaNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03573765

Soft tissue release/debridement/excisionNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03573765

Intra-articular injectionNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03573765

CefadroxilNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03802552

CephalexinNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03802552

Electrical activity during maximal voluntary contraction of the rectus femorisNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07074028

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