Biomarker & Intervention Discovery

Multiple Myeloma

This page lists 12 druggable gene targets, 53 approved or trial-tested drug candidates and 15 recent clinical trials for Multiple Myeloma, 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 remissionEssentially zero; MGUS (precursor) progresses to myeloma in 1%/year; median survival without treatment <1 year once…
Best-intervention remissionDaratumumab + VRd (daratumumab + bortezomib + lenalidomide + dexamethasone) induction + autologous SCT: 66%…
Gap sizeModerate — outcomes dramatically improved but cure remains elusive; CAR-T access critical unmet need
Primary barrieraccess + biological
Full remission profile →

Remission & chronicity

Spontaneous remission
Essentially zero; MGUS (precursor) progresses to myeloma in 1%/year; median survival without treatment <1 year once symptomatic
Best-intervention remission
Daratumumab + VRd (daratumumab + bortezomib + lenalidomide + dexamethasone) induction + autologous SCT: 66% MRD-negative at 24 months (PERSEUS); CAR-T (idecabtagene vicleucel, ciltacabtagene autoleucel): 71-75% overall response, 28-33% CR in heavily pretreated patients; 5-year survival now ~55% (vs ~20% in 2000)
Gap size
Moderate — outcomes dramatically improved but cure remains elusive; CAR-T access critical unmet need
Primary barrier
access + biological
BCMA-targeted CAR-T therapy is transformative but manufacturing delays (6+ weeks), $420K+ cost, and limited centre access restrict use; bispecific antibodies (teclistamab) offer alternative; a functional cure remains an aspiration not a reality for most patients

Biomarker Targets & Therapeutic Agents

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

CD38 203CRBN 151FGFR3 217GPRC5D 1HDAC6 233IKZF1 11IKZF3 37KRAS 233PSMB5 167SLAMF7 3TNFRSF17 19XPO1 151
Therapeutic Agent Effect Direction Evidence Tier Potency Source
ANTI-CD38 CAR T CELLS PREPARATION ? unclear Mechanistic
MEZAGITAMAB ~ modulates Mechanistic
THROMBIN ALFA ? unclear Mechanistic
ISATUXIMAB ? unclear Mechanistic
DARATUMUMAB ↓ inhibits Mechanistic
HUMAX-IL15 ↓ inhibits Mechanistic
MK-0159 ↓ inhibits Mechanistic
FELZARTAMAB ↓ inhibits Mechanistic
KUROMANIN ↓ inhibits Mechanistic 6300.0 nM (IC50)
DELPHINIDIN ↓ inhibits Mechanistic 14600.0 nM (IC50)
PELARGONIDIN ↓ inhibits Mechanistic 16300.0 nM (IC50)
MALVIDIN ↓ inhibits Mechanistic 17000.0 nM (IC50)
QUERCETAGETINIDIN ↓ inhibits Mechanistic 14200.0 nM (IC50)
PEONIDIN ↓ inhibits Mechanistic 20900.0 nM (IC50)
CYANIDIN ↓ inhibits Mechanistic 21800.0 nM (IC50)
DIOSMETINIDIN ↓ inhibits Mechanistic 32100.0 nM (IC50)
PETUNIDIN ↓ inhibits Mechanistic 39200.0 nM (IC50)
FISETINIDIN ↓ inhibits Mechanistic 70300.0 nM (IC50)
LUTEOLIN ↓ inhibits Mechanistic 8200.0 nM (IC50)
MYRICETIN ↓ inhibits Mechanistic 24800.0 nM (IC50)
QUERCETIN ↓ inhibits Mechanistic 37900.0 nM (IC50)
ROBINETIN ↓ inhibits Mechanistic 37900.0 nM (IC50)
QUERCETAGETIN ↓ inhibits Mechanistic 48600.0 nM (IC50)
LUTEOLINIDIN ↓ inhibits Mechanistic 6000.0 nM (IC50)
CHEMBL3342444 ↓ inhibits Mechanistic 3300.0 nM (IC50)
CHEMBL3342440 ↓ inhibits Mechanistic 6400.0 nM (IC50)
CHEMBL3342442 ↓ inhibits Mechanistic 33000.0 nM (IC50)
CHEMBL3342443 ↓ inhibits Mechanistic 27000.0 nM (IC50)
CHEMBL3422364 ↓ inhibits Mechanistic 14000.0 nM (IC50)
CHEMBL3422366 ↓ inhibits Mechanistic 26000.0 nM (IC50)
CHEMBL3426029 ↓ inhibits Mechanistic 55.0 nM (IC50)
CHEMBL3426030 ↓ inhibits Mechanistic 89.0 nM (IC50)
CHEMBL3426031 ↓ inhibits Mechanistic 52.0 nM (IC50)
CHEMBL3426032 ↓ inhibits Mechanistic 6.0 nM (IC50)
CHEMBL3426033 ↓ inhibits Mechanistic 3.9 nM (IC50)
CHEMBL3426034 ↓ inhibits Mechanistic 7.3 nM (IC50)
CHEMBL3426035 ↓ inhibits Mechanistic 10.0 nM (IC50)
CHEMBL3426036 ↓ inhibits Mechanistic 54.0 nM (IC50)
CHEMBL3426037 ↓ inhibits Mechanistic 3.8 nM (IC50)
CHEMBL3426038 ↓ inhibits Mechanistic 13.0 nM (IC50)

Showing top 40 of 203 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
IBERDOMIDE ~ modulates Mechanistic 60.0 nM (IC50)
GOLCADOMIDE ? unclear Mechanistic
POMALIDOMIDE ↓ inhibits Mechanistic 1200.0 nM (IC50)
MG-277 ? unclear Mechanistic
LENALIDOMIDE HYDROCHLORIDE MONOHYDRATE ~ modulates Mechanistic
THALIDOMIDE ↓ inhibits Mechanistic 29900.0 nM (IC50)
TX-16 ? unclear Mechanistic
AK-2292 ? unclear Mechanistic
LENALIDOMIDE ↓ inhibits Mechanistic 1500.0 nM (IC50)
AVADOMIDE ? unclear Mechanistic 38700.0 nM (IC50)
DBRD9 ? unclear Mechanistic
TL12-186 ? unclear Mechanistic
SJ995973 ↓ inhibits Mechanistic
CBPD-268 ? unclear Mechanistic
MEZIGDOMIDE ? unclear Mechanistic
SK5527 ? unclear Mechanistic
SB-405483 ~ modulates Mechanistic
MS40 ? unclear Mechanistic
DTAG-13 ? unclear Mechanistic
PTD10 ? unclear Mechanistic
MD-222 ? unclear Mechanistic
NVP-DKY709 ? unclear Mechanistic
CHEMBL3764647 ↓ inhibits Mechanistic 7900.0 nM (Kd)
SUCCINIMIDE ↓ inhibits Mechanistic 34500.0 nM (IC50)
CHEMBL4449512 ↓ inhibits Mechanistic 2.0 nM (IC50)
CHEMBL4436772 ↓ inhibits Mechanistic 5.5 nM (IC50)
CHEMBL3693969 ↓ inhibits Mechanistic 1470.0 nM (IC50)
CHEMBL4226052 ↓ inhibits Mechanistic 28.0 nM (Kd)
CHEMBL4466169 ↓ inhibits Mechanistic 10000.0 nM (Kd)
CHEMBL4635421 ↓ inhibits Mechanistic 440.0 nM (Ki)
CHEMBL4648907 ↓ inhibits Mechanistic 130.0 nM (Ki)
CHEMBL4645406 ↓ inhibits Mechanistic 970.0 nM (Ki)
CHEMBL4648134 ↓ inhibits Mechanistic 800.0 nM (Ki)
CHEMBL4639892 ↓ inhibits Mechanistic 2200.0 nM (Ki)
DASABUVIR ↓ inhibits Mechanistic 4710.0 nM (Ki)
CHEMBL4758352 ↓ inhibits Mechanistic 67800.0 nM (Ki)
CHEMBL4797970 ↓ inhibits Mechanistic 65700.0 nM (IC50)
CHEMBL4759876 ↓ inhibits Mechanistic 84800.0 nM (IC50)
CHEMBL4798668 ↓ inhibits Mechanistic 1200.0 nM (IC50)
CHEMBL4749646 ↓ inhibits Mechanistic 460.0 nM (IC50)

Showing top 40 of 151 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
SEGIGRATINIB ↓ inhibits Mechanistic
RABEPRAZOLE ↓ inhibits Mechanistic
ORANTINIB ↓ inhibits Mechanistic
DABOGRATINIB ↓ inhibits Mechanistic
NINTEDANIB ESYLATE ↓ inhibits Mechanistic
DASATINIB ANHYDROUS ? unclear Mechanistic
VOFATAMAB ? unclear Mechanistic
INFIGRATINIB ↓ inhibits Mechanistic
FEXAGRATINIB ↓ inhibits Mechanistic
DERAZANTINIB ↓ inhibits Mechanistic
LY-2874455 ↓ inhibits Mechanistic
ROGARATINIB ↓ inhibits Mechanistic
NIVOLUMAB ? unclear Mechanistic
TOZASERTIB ? unclear Mechanistic 860.0 nM (Kd)
CETRELIMAB ? unclear Mechanistic
AVUTOMETINIB ? unclear Mechanistic
PAZOPANIB HYDROCHLORIDE ↓ inhibits Mechanistic
ENTRECTINIB ? unclear Mechanistic 158.49 nM (Ki)
ENMD-981693 ↓ inhibits Mechanistic
ENMD-2076 ↓ inhibits Mechanistic
BRIVANIB ↓ inhibits Mechanistic 160.0 nM (Kd)
PEMIGATINIB ↓ inhibits Mechanistic
CP-547632 ? unclear Mechanistic
NVP-TAE684 ? unclear Mechanistic
MLN-8054 ? unclear Mechanistic 1200.0 nM (Kd)
COMPOUND 29 [PMID: 36356320] ↓ inhibits Mechanistic
LY2874455 ↓ inhibits Mechanistic
ZOLIGRATINIB ? unclear Mechanistic
ASP5878 ↓ inhibits Mechanistic
TASURGRATINIB ? unclear Mechanistic
MASITINIB ↓ inhibits Mechanistic
PONATINIB ↓ inhibits Mechanistic
TRAFERMIN ? unclear Mechanistic
S-49076 ? unclear Mechanistic
GEFITINIB ? unclear Mechanistic
PALIFERMIN ? unclear Mechanistic
SINTILIMAB ? unclear Mechanistic
PICTILISIB ? unclear Mechanistic
FGFR INHIBITOR CPL304110 ? unclear Mechanistic
IRINOTECAN HYDROCHLORIDE ? unclear Mechanistic

Showing top 40 of 217 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
TALQUETAMAB ~ modulates Mechanistic
Therapeutic Agent Effect Direction Evidence Tier Potency Source
ABEXINOSTAT ↓ inhibits Mechanistic
TRICHOSTATIN A ↓ inhibits Mechanistic
PHENYLBUTYRYLHYDROXAMIC ACID ? unclear Mechanistic
KA2507 ↓ inhibits Mechanistic
BML-281 ↓ inhibits Mechanistic
BENZOHYDROXAMATE ? unclear Mechanistic
ROMIDEPSIN ↓ inhibits Mechanistic 14000.0 nM (IC50)
VORINOSTAT ↓ inhibits Mechanistic 100.0 nM (IC50)
COMPOUND 25AP [PMID: 37796543] ↓ inhibits Mechanistic
ENTINOSTAT ↓ inhibits Mechanistic
APICIDIN ? unclear Mechanistic 4300.0 nM (IC50)
GIVINOSTAT ↓ inhibits Mechanistic
INHIBITOR M9 [PMID: 38236416] ↓ inhibits Mechanistic
BUFEXAMAC ↓ inhibits Mechanistic
VALPROPYLHYDROXAMIC ACID ? unclear Mechanistic
PRACINOSTAT ? unclear Mechanistic
AR-42 ? unclear Mechanistic
FIMEPINOSTAT ↓ inhibits Mechanistic
SCRIPTAID ↓ inhibits Mechanistic
MARBOSTAT-100 ↓ inhibits Mechanistic
CITARINOSTAT ↓ inhibits Mechanistic
SUPRASTAT ↓ inhibits Mechanistic
QUISINOSTAT ↓ inhibits Mechanistic
HINOKITIOL ? unclear Mechanistic
CHEMBL:CHEMBL324053 ? unclear Mechanistic
TYA-018 ↓ inhibits Mechanistic
RICOLINOSTAT ↓ inhibits Mechanistic
RESMINOSTAT ↓ inhibits Mechanistic
SS-208 ↓ inhibits Mechanistic
URSOLIC ACID ? unclear Mechanistic
COMPOUND 23F [ZHANG ET AL., 2025] ↓ inhibits Mechanistic
SANTACRUZAMATE A ↓ inhibits Mechanistic
LARGAZOLE ? unclear Mechanistic
BUTYRYLHYDROXAMIC ACID ? unclear Mechanistic
PSAMMAPLIN A ? unclear Mechanistic
BUTYRIC ACID ? unclear Mechanistic
CUDC-101 ↓ inhibits Mechanistic
VALPROIC ACID ? unclear Mechanistic
CHR-3996 ↓ inhibits Mechanistic
KA1010 ↓ inhibits Mechanistic

Showing top 40 of 233 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
DAUNORUBICIN LIPOSOMAL ? unclear Mechanistic
IMATINIB ? unclear Mechanistic
FLUDARABINE ? unclear Mechanistic
METHOTREXATE ? unclear Mechanistic
CYTARABINE ? unclear Mechanistic
IBERDOMIDE ↑ activates Mechanistic 1.0 nM (EC50)
POMALIDOMIDE ↑ activates Mechanistic 24.0 nM (EC50)
LENALIDOMIDE ↑ activates Mechanistic 67.0 nM (EC50)
CHEMBL5531974 ↑ activates Mechanistic 120.0 nM (EC50)
CHEMBL6074489 ↑ activates Mechanistic 35.0 nM (EC50)
CHEMBL6078004 ↑ activates Mechanistic 650.0 nM (EC50)
Therapeutic Agent Effect Direction Evidence Tier Potency Source
CYCLOPHOSPHAMIDE ANHYDROUS ? unclear Mechanistic
IBERDOMIDE ↑ activates Mechanistic 0.5 nM (EC50)
POMALIDOMIDE ↑ activates Mechanistic 22.0 nM (EC50)
LENALIDOMIDE ↑ activates Mechanistic 87.0 nM (EC50)
CHEMBL4645677 ↑ activates Mechanistic 0.1 nM (EC50)
CHEMBL4640365 ↑ activates Mechanistic 0.1 nM (EC50)
CHEMBL4638224 ↑ activates Mechanistic 0.1 nM (EC50)
CHEMBL4641034 ↑ activates Mechanistic 0.2 nM (EC50)
CHEMBL4645692 ↑ activates Mechanistic 0.3 nM (EC50)
CHEMBL4642904 ↑ activates Mechanistic 0.2 nM (EC50)
CHEMBL4636670 ↑ activates Mechanistic 0.1 nM (EC50)
CHEMBL4647512 ↑ activates Mechanistic 0.5 nM (EC50)
CHEMBL4647905 ↑ activates Mechanistic 0.1 nM (EC50)
CHEMBL4645564 ↑ activates Mechanistic 2.4 nM (EC50)
CHEMBL4644747 ↑ activates Mechanistic 2.1 nM (EC50)
CHEMBL4642316 ↑ activates Mechanistic 3.3 nM (EC50)
CHEMBL4645905 ↑ activates Mechanistic 1.5 nM (EC50)
CHEMBL4634342 ↑ activates Mechanistic 0.5 nM (EC50)
CHEMBL4647587 ↑ activates Mechanistic 0.1 nM (EC50)
CHEMBL4642197 ↑ activates Mechanistic 0.3 nM (EC50)
CHEMBL4634712 ↑ activates Mechanistic 0.7 nM (EC50)
CHEMBL4642361 ↑ activates Mechanistic 0.4 nM (EC50)
CHEMBL4639846 ↑ activates Mechanistic 0.4 nM (EC50)
CHEMBL4642706 ↑ activates Mechanistic 1.0 nM (EC50)
CHEMBL4637801 ↑ activates Mechanistic 0.1 nM (EC50)
CHEMBL4642035 ↑ activates Mechanistic 3.0 nM (EC50)
CHEMBL4638669 ↑ activates Mechanistic 7.5 nM (EC50)
CHEMBL4645982 ↑ activates Mechanistic 0.1 nM (EC50)
CHEMBL4641342 ↑ activates Mechanistic 0.6 nM (EC50)
CHEMBL4646021 ↑ activates Mechanistic 0.3 nM (EC50)
CHEMBL4647343 ↑ activates Mechanistic 0.5 nM (EC50)
CHEMBL4643542 ↑ activates Mechanistic 1.0 nM (EC50)
CHEMBL4636184 ↑ activates Mechanistic 0.1 nM (EC50)
CHEMBL4648954 ↑ activates Mechanistic 0.3 nM (EC50)
CHEMBL4644660 ↑ activates Mechanistic 0.1 nM (EC50)
CHEMBL4637622 ↑ activates Mechanistic 0.1 nM (EC50)
CHEMBL4645264 ↑ activates Mechanistic 0.6 nM (EC50)
Therapeutic Agent Effect Direction Evidence Tier Potency Source
GEMCITABINE ? unclear Mechanistic
VEDROPREVIR ↓ inhibits Mechanistic
COBIMETINIB ? unclear Mechanistic
BEVACIZUMAB ? unclear Mechanistic
RAS PEPTIDE CANCER VACCINE ? unclear Mechanistic
PACLITAXEL PROTEIN-BOUND ? unclear Mechanistic
SOTORASIB ? unclear Mechanistic 68.0 nM (IC50)
AMG410 ~ modulates Mechanistic
NIVOLUMAB ? unclear Mechanistic
PIMASERTIB ? unclear Mechanistic
METFORMIN ? unclear Mechanistic
TIZATERKIB ? unclear Mechanistic
GARSORASIB ↓ inhibits Mechanistic
CAPECITABINE ? unclear Mechanistic
ENCORAFENIB ? unclear Mechanistic
DACTOLISIB ? unclear Mechanistic
IRINOTECAN HYDROCHLORIDE ? unclear Mechanistic
PICTILISIB ? unclear Mechanistic
PEMBROLIZUMAB ? unclear Mechanistic
ARS-1620 ↓ inhibits Mechanistic
DASATINIB ANHYDROUS ? unclear Mechanistic
COMPOUND 25 [PMID: 32023060] ↓ inhibits Mechanistic
DANOPREVIR ↓ inhibits Mechanistic
DOCETAXEL ANHYDROUS ? unclear Mechanistic
MUC1-TARGETED PEPTIDE GO-203-2C ? unclear Mechanistic
THERAPEUTIC TUMOR INFILTRATING LYMPHOCYTES ? unclear Mechanistic
AZACITIDINE ? unclear Mechanistic
B-RAF/VEGFR-2 INHIBITOR RAF265 ? unclear Mechanistic
PARITAPREVIR ↓ inhibits Mechanistic
VANIPREVIR ↓ inhibits Mechanistic
DUAL IGF-1R/INSR INHIBITOR BMS-754807 ? unclear Mechanistic
SORAFENIB ? unclear Mechanistic
CILUPREVIR ↓ inhibits Mechanistic
TRASTUZUMAB ? unclear Mechanistic
TEMSIROLIMUS ? unclear Mechanistic
DECITABINE ? unclear Mechanistic
SALIRASIB ↓ inhibits Mechanistic
PHA-665752 ? unclear Mechanistic
MITOGEN-ACTIVATED PROTEIN KINASE KINASE INHIBITOR ? unclear Mechanistic
SELUMETINIB ↓ inhibits Mechanistic

Showing top 40 of 233 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
IXAZOMIB ↓ inhibits Mechanistic 3.4 nM (IC50)
BORTEZOMIB ↓ inhibits Mechanistic 7.9 nM (IC50)
OPROZOMIB ↓ inhibits Mechanistic 10.0 nM (IC50)
WLL-VS ↓ inhibits Mechanistic
BELACTOSIN A ? unclear Mechanistic 1440.0 nM (IC50)
BORTEZOMIB D-MANNITOL ↓ inhibits Mechanistic
MARIZOMIB ↓ inhibits Mechanistic 3.5 nM (IC50)
MG-132 ↓ inhibits Mechanistic 3.7 nM (Ki)
LACTACYSTIN ? unclear Mechanistic 370.0 nM (IC50)
NIC-0102 ↓ inhibits Mechanistic
CARFILZOMIB ↓ inhibits Mechanistic
ZETOMIPZOMIB ↓ inhibits Mechanistic
CHEMBL:CHEMBL490829 ? unclear Mechanistic
IXAZOMIB CITRATE ? unclear Mechanistic
CHEMBL381735 ↓ inhibits Mechanistic 2.4 nM (IC50)
CHEMBL383529 ↓ inhibits Mechanistic 260.0 nM (IC50)
CHEMBL377532 ↓ inhibits Mechanistic 0.72 nM (Ki)
CHEMBL207336 ↓ inhibits Mechanistic 17.0 nM (IC50)
CHEMBL208015 ↓ inhibits Mechanistic 6.1 nM (IC50)
CHEMBL208019 ↓ inhibits Mechanistic 170.0 nM (IC50)
CHEMBL205687 ↓ inhibits Mechanistic 130.0 nM (Ki)
CHEMBL204518 ↓ inhibits Mechanistic 2500.0 nM (IC50)
CHEMBL207162 ↓ inhibits Mechanistic 2.5 nM (IC50)
CHEMBL206206 ↓ inhibits Mechanistic 2.9 nM (IC50)
CHEMBL204992 ↓ inhibits Mechanistic 1.1 nM (IC50)
CHEMBL207016 ↓ inhibits Mechanistic 2.1 nM (IC50)
CHEMBL376815 ↓ inhibits Mechanistic 6.7 nM (IC50)
CHEMBL381884 ↓ inhibits Mechanistic 430.0 nM (IC50)
CHEMBL207598 ↓ inhibits Mechanistic 0.5 nM (Ki)
CHEMBL207394 ↓ inhibits Mechanistic 21000.0 nM (IC50)
CHEMBL383122 ↓ inhibits Mechanistic 38.0 nM (Ki)
CHEMBL205757 ↓ inhibits Mechanistic 0.51 nM (Ki)
CHEMBL207425 ↓ inhibits Mechanistic 32.0 nM (Ki)
CHEMBL207196 ↓ inhibits Mechanistic 24.0 nM (IC50)
CHEMBL207669 ↓ inhibits Mechanistic 10.0 nM (IC50)
CHEMBL383673 ↓ inhibits Mechanistic 540.0 nM (IC50)
CHEMBL437311 ↓ inhibits Mechanistic 45.0 nM (Ki)
CHEMBL381931 ↓ inhibits Mechanistic 140.0 nM (Ki)
CHEMBL379476 ↓ inhibits Mechanistic 500.0 nM (Ki)
EPOXOMYCIN ↓ inhibits Mechanistic 3.7 nM (IC50)

Showing top 40 of 167 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
XBP1-US/XBP1-SP/CD138/CS1 MULTIPEPTIDE VACCINE PVX-410 ? unclear Mechanistic
AZINTUXIZUMAB VEDOTIN ~ modulates Mechanistic
ELOTUZUMAB ↓ inhibits Mechanistic
Therapeutic Agent Effect Direction Evidence Tier Potency Source
CILTACABTAGENE AUTOLEUCEL ? unclear Mechanistic
IDECABTAGENE VICLEUCEL ? unclear Mechanistic
LINVOSELTAMAB ? unclear Mechanistic
AUTOLOGOUS ANTI-BCMA-CAR-EXPRESSING CD4+/CD8+ T-LYMPHOCYTES FCARH143 ? unclear Mechanistic
P-BCMA-101 ? unclear Mechanistic
AUTOLOGOUS ANTI-BCMA-CAR-MRNA-TRANSFECTED CD8+ T-LYMPHOCYTES ? unclear Mechanistic
KITE-585 ? unclear Mechanistic
APRIL ? unclear Mechanistic
ORVACABTAGENE AUTOLEUCEL ? unclear Mechanistic
ELRANATAMAB ~ modulates Mechanistic
PACANALOTAMAB ? unclear Mechanistic
TECLISTAMAB ~ modulates Mechanistic
BELANTAMAB MAFODOTIN ? unclear Mechanistic
AUTOLOGOUS ANTI-BCMA-CAR-4-1BB-CD3ZETA-EXPRESSING MEMORY T-LYMPHOCYTES BB21217 ? unclear Mechanistic
AUTOLOGOUS ANTI-BCMA-CAR-4-1BB-CD3ZETA-EXPRESSING T-CELLS C-CAR088 ? unclear Mechanistic
AUTOLOGOUS ANTI-BCMA-CAR-TCRZ/4-1BB-EXPRESSING T-LYMPHOCYTES CART-BCMA ? unclear Mechanistic
AUTOLOGOUS BCMA/TACI-TARGETED CAR T CELLS AUTO2 ? unclear Mechanistic
BAFF ? unclear Mechanistic
ANTI-BCMA CAR T CELLS PREPARATION ? unclear Mechanistic
Therapeutic Agent Effect Direction Evidence Tier Potency Source
VERDINEXOR ↓ inhibits Mechanistic 24.0 nM (EC50)
SELINEXOR ↓ inhibits Mechanistic 18.74 nM (Kd)
ELTANEXOR ↓ inhibits Mechanistic
LEPTOMYCIN B ↓ inhibits Mechanistic 1.0 nM (IC50)
FELEZONEXOR ? unclear Mechanistic
GONIOTHALAMIN ? unclear Mechanistic
RIFAMPIN ? unclear Mechanistic
OSTHOLE ? unclear Mechanistic 1600.0 nM (IC50)
CHEMBL1084286 ↓ inhibits Mechanistic 6400.0 nM (IC50)
CHEMBL3929869 ↓ inhibits Mechanistic 1000.0 nM (IC50)
CHEMBL3891836 ↓ inhibits Mechanistic 1000.0 nM (IC50)
CHEMBL3964319 ↓ inhibits Mechanistic 1000.0 nM (IC50)
CHEMBL3896208 ↓ inhibits Mechanistic 1000.0 nM (IC50)
CHEMBL3899053 ↓ inhibits Mechanistic 1000.0 nM (IC50)
CHEMBL3951325 ↓ inhibits Mechanistic 1000.0 nM (IC50)
CHEMBL3929349 ↓ inhibits Mechanistic 1000.0 nM (IC50)
CHEMBL3919410 ↓ inhibits Mechanistic 1000.0 nM (IC50)
CHEMBL3895766 ↓ inhibits Mechanistic 1000.0 nM (IC50)
CHEMBL3893042 ↓ inhibits Mechanistic 1000.0 nM (IC50)
CHEMBL3911673 ↓ inhibits Mechanistic 1000.0 nM (IC50)
CHEMBL3911830 ↓ inhibits Mechanistic 1000.0 nM (IC50)
CHEMBL3914451 ↓ inhibits Mechanistic 1000.0 nM (IC50)
CHEMBL3890257 ↓ inhibits Mechanistic 1000.0 nM (IC50)
CHEMBL3928302 ↓ inhibits Mechanistic 1000.0 nM (IC50)
CHEMBL3925662 ↓ inhibits Mechanistic 1000.0 nM (IC50)
CHEMBL3898004 ↓ inhibits Mechanistic 1000.0 nM (IC50)
CHEMBL3908175 ↓ inhibits Mechanistic 1000.0 nM (IC50)
CHEMBL3915859 ↓ inhibits Mechanistic 1000.0 nM (IC50)
CHEMBL3956187 ↓ inhibits Mechanistic 1000.0 nM (IC50)
CHEMBL3904126 ↓ inhibits Mechanistic 1000.0 nM (IC50)
CHEMBL4088268 ↓ inhibits Mechanistic 10.0 nM (IC50)
CHEMBL4077955 ↓ inhibits Mechanistic 25.0 nM (IC50)
CHEMBL4277221 ↑ activates Mechanistic 900.0 nM (EC50)
CHEMBL4293588 ↑ activates Mechanistic 1800.0 nM (EC50)
CHEMBL4278766 ↑ activates Mechanistic 3800.0 nM (EC50)
CHEMBL4282126 ↑ activates Mechanistic 1800.0 nM (EC50)
CHEMBL4283174 ↑ activates Mechanistic 900.0 nM (EC50)
CHEMBL4294538 ↑ activates Mechanistic 30000.0 nM (EC50)
CHEMBL4294903 ↑ activates Mechanistic 30000.0 nM (EC50)
CHEMBL4280077 ↑ activates Mechanistic 15000.0 nM (EC50)

Showing top 40 of 151 agents ranked by evidence tier.


Clinical Trials

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

NCT06948084 Phase: 2 Started: 2027-05-22

This phase II trial compares the effect of the combination of daratumumab-hyaluronidase (daratumumab) and teclistamab to the usual treatment of daratumumab, pomalidomide, dexamethasone or daratumumab, carfilzomib and dexamethasone in treating patients with multiple myeloma that has not responded to ...

NCT07106710 Phase: 2 Started: 2025-08-15

This is a prospective, single-arm, phase II study to evaluate the efficacy and safety of autologous stem cell transplantation combined with BCMA CAR-T therapy followed by GPRC5D/CD3 bispecific antibody maintenance in transplant-eligible patients with ultra-high-risk multiple myeloma.

NCT06465316 Phase: 1 Started: 2024-12-06

This phase Ib trial tests the safety, side effects, and best dose of iberdomide in combination with teclistamab in treating multiple myeloma that has come back after a period of improvement (relapsed) or that does not respond to treatment (refractory). Iberdomide is a medication that belongs to a gr...

NCT07609940 Phase: 4 Started: 2026-07-15

This study is researching whether the use of tocilizumab before the first dose of linvoseltamab will decrease the risk of Cytokine Release Syndrome (CRS) in participants who have Relapsed or Refractory Multiple Myeloma (RRMM) who have already been treated with at least four lines of treatment for th...

NCT07322159 Phase: Early 1 Started: 2026-01-08

This study is an open-label, single-arm early exploratory clinical study, aiming to evaluate the safety, tolerability and preliminary efficacy of IASO206 Injection(In Vivo CAR-T) in Patients with Relapsed/Refractory Multiple Myeloma

NCT06953960 Phase: 1/2 Started: 2025-07-23

Multiple myeloma (MM) is a plasma cell disease characterized by the growth of clonal plasma cells in the bone marrow. The purpose of this study is to assess the safety and change in disease activity of surzetoclax in adult participants with relapsed/refractory (R/R) MM. Adverse events and change in ...

NCT07891923 Phase: 2 Started: 2025-10-28

The goal of this clinical trial is to evaluate whether a modified (less frequent) dosing schedule of daratumumab, when added to standard VRd therapy, can be an effective induction regimen for treating newly diagnosed multiple myeloma (NDMM). The main questions it aims to answer are: 1. Does the modi...

NCT06952478 Phase: 3 Started: 2025-08-12

This study is a phase 1/3 study to Compare Pharmacokinetics, Efficacy and Safety of Subcutaneous CT-P44 and Darzalex Faspro in Combination with Lenalidomide and Dexamethasone in Patients with Refractory or Relapsed Multiple Myeloma

NCT07106736 Phase: 2 Started: 2025-08-12

This is a prospective, single-center, clinical study to evaluate the efficacy and safety of a fully immunotherapy-based strategy guided by MRD-driven dynamic risk stratification in transplant-ineligible patients with newly diagnosed multiple myeloma.

NCT07106684 Phase: 2 Started: 2025-08-09

This is a prospective, single-arm, phase II study to evaluate the efficacy and safety of autologous stem cell transplantation combined with BCMA CAR-T therapy followed by GPRC5D/CD3 bispecific antibody maintenance in transplant-eligible patients with primary plasma cell leukemia.

NCT05623020 Phase: 3 Started: 2022-11-10

Elranatamab is a bispecific antibody: binding of elranatamab to CD3-expressing T-cells and BCMA-expressing multiple myeloma cells causes targeted T-cell-mediated cytotoxicity. The main purpose of the study is to evaluate if the combination of Elranatamab, Daratumumab and Lenalidomide offers superior...

NCT06103838 Phase: 2 Started: 2024-03-25

Background: Multiple myeloma (MM) is an incurable cancer of certain blood cells. MM often returns after treatment, and most people survive only 5 to 8 years after diagnosis. To improve survival, researchers need to find ways to identify returning disease earlier. Objective: To find out if the radiot...

NCT03761108 Phase: 1/2 Started: 2019-01-23

The main purpose of this study is to learn about the safety of linvoseltamab and to find out what is the best dose of linvoseltamab to give to patients with multiple myeloma and to look for any signs that linvoseltamab can effectively treat cancer. The study is looking at several other research ques...

NCT04822337 Phase: 1/2 Started: 2021-05-19

This research study is being done to learn if the study drug belantamab mafodotin, in combination with other standard medications, can improve multiple myeloma. This study will also help determine what effects, good and/or bad, this combination of study drugs have on subjects and their cancer, and t...

NCT04973605 Phase: 1/2 Started: 2021-09-16

The purpose of this study is to assess the safety, tolerability, and efficacy of sonrotoclax as monotherapy and in various combinations in patients with relapsed/refractory (R/R) multiple myeloma (MM) and chromosomal translocation t(11;14). The study investigates sonrotoclax alone and in combination...


Agents Found By Disease-Level Search

Searched directly from “Multiple Myeloma” 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).

53 agents shown (approved or with trial evidence) — 41 not found by the gene-target search above.

DexamethasoneNOT in gene-target list

Approved · 15 trials · source: ClinicalTrials.gov, Open Targets
NCT03029234 NCT01478581 NCT07341867

Bortezomib

Approved · 8 trials · source: ClinicalTrials.gov, Open Targets
NCT00514371 NCT07341867 NCT00006184

LENALIDOMIDE

Approved · 8 trials · source: ClinicalTrials.gov, Open Targets
NCT07341867 NCT06932562 NCT01698801

Carfilzomib

Approved · 4 trials · source: ClinicalTrials.gov, Open Targets
NCT03029234 NCT02056756 NCT01677858

cyclophosphamideNOT in gene-target list

Approved · 4 trials · source: ClinicalTrials.gov, Open Targets
NCT00003116 NCT00005854 NCT07341867

cyclosporineNOT in gene-target list

Phase 2 · 4 trials · source: ClinicalTrials.gov, Open Targets
NCT00003116 NCT00005854 NCT00112593

fludarabine phosphateNOT in gene-target list

Phase 2 · 4 trials · source: ClinicalTrials.gov, Open Targets
NCT01658319 NCT00856388 NCT00112593

melphalanNOT in gene-target list

Approved · 4 trials · source: ClinicalTrials.gov, Open Targets
NCT00792142 NCT00856388 NCT00006244

busulfanNOT in gene-target list

Phase 3 · 3 trials · source: ClinicalTrials.gov, Open Targets
NCT00003116 NCT00943319 NCT01237951

Pomalidomide

Approved · 3 trials · source: ClinicalTrials.gov, Open Targets
NCT07825064 NCT00833833 NCT02103335

Revlimid, Melphalan, Prednisone, ThalidomideNOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT00961467 NCT00961467

filgrastimNOT in gene-target list

Phase 3 · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT00003116 NCT02593123

Pembrolizumab

Phase 3 · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT01953692 NCT07341867

Daratumumab

Approved · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT07341867 NCT06932562

Teclistamab

Approved · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT06100237 NCT06171685

Talquetamab

Approved · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT06100237 NCT07093554

Linvoseltamab

Approved · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT06932562 NCT07455851

Doxorubicin hydrochlorideNOT in gene-target list

Approved · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT00006184 NCT00036790

Methotrexate

Phase 2 · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT00006184 NCT00005854

cytogenetic analysisNOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT00792142 NCT00898066

fluorescence in situ hybridizationNOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT00792142 NCT00898066

Lenalidomide, endoxan, prednisoneNOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT01352338 NCT01352338

mycophenolate mofetilNOT in gene-target list

Phase 2 · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT00112593 NCT02593123

sodium seleniteNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02184533

NEX-20ANOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05643248

LNA-i-miR-221NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04811898

Routine Blood Draw - Phase 1 onlyNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06224309

anti-BCMA CAR-TNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06793449

ASCTNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06793449

methoxyamineNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01658319

western blottingNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01658319

DNA analysisNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01658319

ibrutinib (PCI-32765)NOT in gene-target list

Approved · 1 trial · source: ClinicalTrials.gov
NCT01478581

Fludarabine

Phase 3 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT00943319

CampathNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00943319

tanespimycinNOT in gene-target list

Phase 3 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT00514371

Lenalidomide 20 mgNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01953692

Lenalidomide 25 mgNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01953692

Carboplatin

Phase 2 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT07341867

PaclitaxelNOT in gene-target list

Phase 2 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT07341867

DoxorubicinNOT in gene-target list

Approved · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT07341867

CevostamabNOT in gene-target list

Phase 3 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT07825064

TocilizumabNOT in gene-target list

Phase 1 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT07825064

Daratumumab SCNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06100237

ABBV-438NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07409246

Ixazomib (MLN9708)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02697383

nivolumab-daratumumabNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03184194

nivolumab-daratumumab with low-dose cyclophosphamideNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03184194

Cohort 1: Carfilzomib 15 mg/m2NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02148913

Cohort 2: Carfilzomib 20 mg/m2NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02148913

Cohort 2b: Carfilzomib 20 mg/m2NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02148913

Cohort 3b: Carfilzomib 27 mg/m2NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02148913

PI3K-beta Inhibitor GSK2636771NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04439188

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