Myelodysplastic Syndromes Pipeline 2026: Drugs & Trials

Remission & chronicity

Spontaneous remission

Essentially zero; progressive cytopaenia and transformation to AML in 30-40% of higher-risk MDS over 2-5 years; median survival low-risk MDS ~5 years, high-risk ~1 year without treatment

Best-intervention remission

Allogeneic HSCT: only curative option; 5-year OS 35-50% in fit patients; hypomethylating agents (azacitidine, decitabine): ORR 15-17%, overall survival improvement 9 months vs SC; oral azacitidine (CC-486): 10.7 vs 8.9 months OS in AML maintenance post-remission

Gap size

Large — MDS has limited treatment options; curative HSCT used in <15% of patients (age, comorbidity, donor availability); existing drugs are not curative

Primary barrier

biological + access

MDS predominantly affects elderly patients who are often HSCT-ineligible; hypomethylating agents provide modest benefit without cure; luspatercept (TGF-β trap) approved for LR-MDS with ring sideroblasts 2020 — improved transfusion independence; imetelstat for LR-MDS — Phase 3 (IMerge) showed independence from transfusion in 40% vs 15% placebo

Biomarker Targets & Therapeutic Agents

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

ACVR2B 33 BCL2 214 CD47 27 CRBN 151 DNMT1 232 HAVCR2 78 IDH1 531 IDH2 163 SF3B1 6 TERT 211 TET2 68 TP53 652

ACVR2B

UniProt Ensembl DGIdb Open Targets

Therapeutic Agent	Effect Direction	Evidence Tier	Potency	Source
DIBOTERMIN ALFA	↑ activates	Mechanistic		DGIdb
RAMATERCEPT	? unclear	Mechanistic		DGIdb
BIMAGRUMAB	↓ inhibits	Mechanistic		DGIdb
DASATINIB	↓ inhibits	Mechanistic	570.0 nM (Kd)	ChEMBL
ALVOCIDIB	↓ inhibits	Mechanistic	2400.0 nM (Kd)	ChEMBL
PAZOPANIB	↓ inhibits	Mechanistic	2400.0 nM (Kd)	ChEMBL
JNJ-7706621	↓ inhibits	Mechanistic	5900.0 nM (Kd)	ChEMBL
SB-202190	↓ inhibits	Mechanistic	4800.0 nM (Kd)	ChEMBL
STAUROSPORINE	↓ inhibits	Mechanistic	4600.0 nM (Kd)	ChEMBL
VX-702	↓ inhibits	Mechanistic	92.0 nM (Kd)	ChEMBL
LESTAURTINIB	↓ inhibits	Mechanistic	3800.0 nM (Kd)	ChEMBL
CHEMBL1808264	↓ inhibits	Mechanistic	10.0 nM (IC50)	ChEMBL
TAE-684	↓ inhibits	Mechanistic	4300.0 nM (Kd)	ChEMBL
PLX-4720	↓ inhibits	Mechanistic	3200.0 nM (Kd)	ChEMBL
KW-2449	↓ inhibits	Mechanistic	370.0 nM (Kd)	ChEMBL
CHEMBL1241674	↓ inhibits	Mechanistic	7.6 nM (Kd)	ChEMBL
AXITINIB	↓ inhibits	Mechanistic	5200.0 nM (Kd)	ChEMBL
AST-487	↓ inhibits	Mechanistic	1400.0 nM (Kd)	ChEMBL
CHEMBL386051	↓ inhibits	Mechanistic	9.1 nM (Kd)	ChEMBL
CHEMBL3823101	↓ inhibits	Mechanistic	7680.0 nM (IC50)	ChEMBL
CHEMBL3991933	↓ inhibits	Mechanistic	901.0 nM (Kd)	ChEMBL
AZD-7762	↓ inhibits	Mechanistic	253.0 nM (Kd)	ChEMBL
SARACATINIB	↓ inhibits	Mechanistic	3878.0 nM (Kd)	ChEMBL
TG100-801	↓ inhibits	Mechanistic	419.0 nM (Kd)	ChEMBL
GSK-1070916	↓ inhibits	Mechanistic	9900.0 nM (Kd)	ChEMBL
CHEMBL4283638	↓ inhibits	Mechanistic	3130.0 nM (IC50)	ChEMBL
CHEMBL513147	↓ inhibits	Mechanistic	9300.0 nM (IC50)	ChEMBL
CHEMBL5595138	↓ inhibits	Mechanistic	68.9 nM (IC50)	ChEMBL
CHEMBL5594704	↓ inhibits	Mechanistic	6344.0 nM (IC50)	ChEMBL
CHEMBL5591770	↓ inhibits	Mechanistic	17.9 nM (IC50)	ChEMBL
CHEMBL5653589	↓ inhibits	Mechanistic	3.019 nM (Kd)	ChEMBL
CHEMBL3752910	↓ inhibits	Mechanistic	3181.45 nM (Kd)	ChEMBL
CHEMBL6172244	↓ inhibits	Mechanistic	364.0 nM (IC50)	ChEMBL

BCL2

UniProt Ensembl DGIdb Open Targets

Therapeutic Agent	Effect Direction	Evidence Tier	Potency	Source
NONSTEROIDAL ANTIINFLAMMATORY DRUG	? unclear	Mechanistic		DGIdb
ENDOTHELIN RECEPTOR ANTAGONIST	? unclear	Mechanistic		DGIdb
BORTEZOMIB	? unclear	Mechanistic		DGIdb
DOXORUBICIN HYDROCHLORIDE	? unclear	Mechanistic		DGIdb
MITOXANTRONE HYDROCHLORIDE	? unclear	Mechanistic		DGIdb
VENETOCLAX	↓ inhibits	Mechanistic		DGIdb
NAVITOCLAX	↓ inhibits	Mechanistic		DGIdb
ISOTRETINOIN	? unclear	Mechanistic		DGIdb
BRENTUXIMAB VEDOTIN	? unclear	Mechanistic		DGIdb
OXALIPLATIN	? unclear	Mechanistic		DGIdb
TAZAROTENE	? unclear	Mechanistic		DGIdb
LISAFTOCLAX	↓ inhibits	Mechanistic		DGIdb
GOSSYPOL	? unclear	Mechanistic	320.0 nM (Ki)	DGIdb
EDARAVONE	? unclear	Mechanistic		DGIdb
TENIPOSIDE	? unclear	Mechanistic		DGIdb
DITIOCARB	? unclear	Mechanistic		DGIdb
CARBOPLATIN	? unclear	Mechanistic		DGIdb
CILENGITIDE	? unclear	Mechanistic		DGIdb
ESTRAMUSTINE	? unclear	Mechanistic		DGIdb
RECOMBINANT INTERFERON	? unclear	Mechanistic		DGIdb
CISPLATIN	? unclear	Mechanistic		DGIdb
AZD4320	↓ inhibits	Mechanistic		DGIdb
PELCITOCLAX	? unclear	Mechanistic		DGIdb
ORAL CONTRACEPTIVE	? unclear	Mechanistic		DGIdb
ANG1005	? unclear	Mechanistic		DGIdb
OBATOCLAX	↓ inhibits	Mechanistic		DGIdb
ANTI-CD19 CAR T CELLS AT101	↓ inhibits	Mechanistic		DGIdb
FOSBRETABULIN DISODIUM	? unclear	Mechanistic		DGIdb
APG-1252-M1	↓ inhibits	Mechanistic		DGIdb
SONROTOCLAX	↓ inhibits	Mechanistic		DGIdb
DULANERMIN	? unclear	Mechanistic		DGIdb
RALTITREXED	? unclear	Mechanistic		DGIdb
EPIRUBICIN HYDROCHLORIDE	? unclear	Mechanistic		DGIdb
ETOPOSIDE	? unclear	Mechanistic		DGIdb
MULTIDRUG RESISTANCE MODULATOR	? unclear	Mechanistic		DGIdb
BCL-2 INHIBITOR BCL201	? unclear	Mechanistic		DGIdb
PROTUBOXEPIN A	? unclear	Mechanistic		DGIdb
RIBAVIRIN	? unclear	Mechanistic		DGIdb
DIETHYLSTILBESTROL	? unclear	Mechanistic		DGIdb
APRINOCARSEN	? unclear	Mechanistic		DGIdb

Showing top 40 of 214 agents ranked by evidence tier.

CD47

UniProt Ensembl DGIdb Open Targets

Therapeutic Agent	Effect Direction	Evidence Tier	Potency	Source
ABT-510	? unclear	Mechanistic		DGIdb
EVORPACEPT	? unclear	Mechanistic		DGIdb
ONTORPACEPT	? unclear	Mechanistic		DGIdb
MAGROLIMAB	? unclear	Mechanistic		DGIdb
LIGUFALIMAB	? unclear	Mechanistic		DGIdb
THROMBOSPONDIN 1	? unclear	Mechanistic		DGIdb
ANTI-CD47 MONOCLONAL ANTIBODY CC-90002	? unclear	Mechanistic		DGIdb
CHEMBL3911226	↓ inhibits	Mechanistic	18000.0 nM (Kd)	ChEMBL
CHEMBL3939184	↓ inhibits	Mechanistic	27000.0 nM (Kd)	ChEMBL
CHEMBL3900522	↓ inhibits	Mechanistic	6200.0 nM (Kd)	ChEMBL
CHEMBL3971661	↓ inhibits	Mechanistic	43000.0 nM (Kd)	ChEMBL
CHEMBL3891508	↓ inhibits	Mechanistic	40000.0 nM (Kd)	ChEMBL
CHEMBL3974452	↓ inhibits	Mechanistic	19000.0 nM (Kd)	ChEMBL
CHEMBL3946082	↓ inhibits	Mechanistic	1500.0 nM (Kd)	ChEMBL
CHEMBL3975422	↓ inhibits	Mechanistic	10000.0 nM (Kd)	ChEMBL
CHEMBL3899400	↓ inhibits	Mechanistic	42000.0 nM (Kd)	ChEMBL
CHEMBL4449880	↓ inhibits	Mechanistic	21000.0 nM (Kd)	ChEMBL
CHEMBL4471112	↓ inhibits	Mechanistic	36000.0 nM (Kd)	ChEMBL
CHEMBL4545895	↓ inhibits	Mechanistic	48000.0 nM (Kd)	ChEMBL
CHEMBL4470419	↓ inhibits	Mechanistic	38000.0 nM (Kd)	ChEMBL
CHEMBL4474790	↓ inhibits	Mechanistic	8600.0 nM (Kd)	ChEMBL
CHEMBL4462869	↓ inhibits	Mechanistic	15000.0 nM (Kd)	ChEMBL
CHEMBL4585124	↓ inhibits	Mechanistic	17000.0 nM (Kd)	ChEMBL
CHEMBL4570565	↓ inhibits	Mechanistic	13000.0 nM (Kd)	ChEMBL
CHEMBL4462459	↓ inhibits	Mechanistic	9500.0 nM (Kd)	ChEMBL
CHEMBL4573126	↓ inhibits	Mechanistic	20000.0 nM (Kd)	ChEMBL
CHEMBL4522427	↓ inhibits	Mechanistic	190.0 nM (Kd)	ChEMBL

CRBN

UniProt Ensembl DGIdb Open Targets

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

Showing top 40 of 151 agents ranked by evidence tier.

DNMT1

UniProt Ensembl DGIdb Open Targets

Therapeutic Agent	Effect Direction	Evidence Tier	Potency	Source
MAHANINE	? unclear	Mechanistic		DGIdb
PALIFOSFAMIDE	? unclear	Mechanistic		DGIdb
HYDROXYUREA	? unclear	Mechanistic		DGIdb
ANTROQUINONOL	? unclear	Mechanistic		DGIdb
CHEMBL:CHEMBL1490332	? unclear	Mechanistic		DGIdb
IFOSFAMIDE	? unclear	Mechanistic		DGIdb
FARNESYL TRANSFERASE INHIBITOR	? unclear	Mechanistic		DGIdb
GUADECITABINE	? unclear	Mechanistic		DGIdb
ORAL AZACITIDINE	? unclear	Mechanistic		DGIdb
DEMETHOXYCURCUMIN	? unclear	Mechanistic		DGIdb
SELENIUM	? unclear	Mechanistic		DGIdb
TCDD	? unclear	Mechanistic		DGIdb
CURCUMIN	? unclear	Mechanistic		DGIdb
DAPHNORIN	? unclear	Mechanistic	7560.0 nM (IC50)	DGIdb
DECITABINE	↓ inhibits	Mechanistic		DGIdb
CM-272	↓ inhibits	Mechanistic		DGIdb
MITOXANTRONE HYDROCHLORIDE	? unclear	Mechanistic		DGIdb
AZACITIDINE	↓ inhibits	Mechanistic		DGIdb
GSK3685032	↓ inhibits	Mechanistic		DGIdb
SGI-1027	↓ inhibits	Mechanistic		DGIdb
RODUCITABINE	? unclear	Mechanistic		DGIdb
CISPLATIN	? unclear	Mechanistic		DGIdb
TETRAHYDROCURCUMIN	? unclear	Mechanistic		DGIdb
CEPHALOTHIN	? unclear	Mechanistic	7280.0 nM (IC50)	DGIdb
DECITABINE AND CEDAZURIDINE	? unclear	Mechanistic		DGIdb
BROMO-DEAZA-SAH	↓ inhibits	Mechanistic		DGIdb
MG98	? unclear	Mechanistic		DGIdb
DIETHYLSTILBESTROL	? unclear	Mechanistic		DGIdb
ARSENIC TRIOXIDE	? unclear	Mechanistic		DGIdb
DOXORUBICIN HYDROCHLORIDE	? unclear	Mechanistic		DGIdb
ZEBULARINE	? unclear	Mechanistic		DGIdb
FLOXURIDINE	? unclear	Mechanistic		DGIdb
CHEMBL:CHEMBL2063052	? unclear	Mechanistic		DGIdb
BENZANTHRONE	? unclear	Mechanistic		DGIdb
HEXAHYDROCURCUMIN	? unclear	Mechanistic		DGIdb
CHEMBL:CHEMBL599013	? unclear	Mechanistic		DGIdb
BISDEMETHOXYCURCUMIN	? unclear	Mechanistic		DGIdb
CHEMBL:CHEMBL580076	? unclear	Mechanistic		DGIdb
CHEMBL382617	↓ inhibits	Mechanistic	100000.0 nM (IC50)	ChEMBL
CHEMBL383475	↓ inhibits	Mechanistic	50000.0 nM (IC50)	ChEMBL

Showing top 40 of 232 agents ranked by evidence tier.

HAVCR2

UniProt Ensembl DGIdb Open Targets

Therapeutic Agent	Effect Direction	Evidence Tier	Potency	Source
PD1 INHIBITOR	? unclear	Mechanistic		DGIdb
SABATOLIMAB	↓ inhibits	Mechanistic		DGIdb
ANTI-TIM3 MONOCLONAL ANTIBODY LY3321367	? unclear	Mechanistic		DGIdb
ANTI-TIM-3 MONOCLONAL ANTIBODY	? unclear	Mechanistic		DGIdb
COBOLIMAB	↓ inhibits	Mechanistic		DGIdb
LOMVASTOMIG	↓ inhibits	Mechanistic		DGIdb
CHEMBL1569082	↓ inhibits	Mechanistic	810000.0 nM (Kd)	ChEMBL
CHEMBL4876694	↓ inhibits	Mechanistic	330000.0 nM (Kd)	ChEMBL
CHEMBL4866110	↓ inhibits	Mechanistic	250000.0 nM (Kd)	ChEMBL
CHEMBL4860123	↓ inhibits	Mechanistic	40000.0 nM (Kd)	ChEMBL
CHEMBL4870128	↓ inhibits	Mechanistic	40000.0 nM (Kd)	ChEMBL
CHEMBL4852144	↓ inhibits	Mechanistic	50000.0 nM (Kd)	ChEMBL
CHEMBL4864057	↓ inhibits	Mechanistic	90000.0 nM (Kd)	ChEMBL
CHEMBL4871549	↓ inhibits	Mechanistic	90000.0 nM (Kd)	ChEMBL
CHEMBL4870200	↓ inhibits	Mechanistic	40000.0 nM (Kd)	ChEMBL
CHEMBL4854696	↓ inhibits	Mechanistic	40000.0 nM (Kd)	ChEMBL
CHEMBL4863477	↓ inhibits	Mechanistic	10000.0 nM (Kd)	ChEMBL
CHEMBL4865760	↓ inhibits	Mechanistic	130000.0 nM (Kd)	ChEMBL
CHEMBL4876887	↓ inhibits	Mechanistic	450000.0 nM (Kd)	ChEMBL
CHEMBL1610810	↓ inhibits	Mechanistic	460000.0 nM (Kd)	ChEMBL
CHEMBL1462057	↓ inhibits	Mechanistic	530000.0 nM (Kd)	ChEMBL
CHEMBL4854485	↓ inhibits	Mechanistic	740000.0 nM (Kd)	ChEMBL
CHEMBL69086	↓ inhibits	Mechanistic	670000.0 nM (Kd)	ChEMBL
CHEMBL4864031	↓ inhibits	Mechanistic	780000.0 nM (Kd)	ChEMBL
CHEMBL2132792	↓ inhibits	Mechanistic	1400000.0 nM (Kd)	ChEMBL
CHEMBL1576751	↓ inhibits	Mechanistic	850000.0 nM (Kd)	ChEMBL
CHEMBL4848599	↓ inhibits	Mechanistic	2000.0 nM (Kd)	ChEMBL
CHEMBL4873902	↓ inhibits	Mechanistic	250.0 nM (Kd)	ChEMBL
CHEMBL4859588	↓ inhibits	Mechanistic	1600.0 nM (Ki)	ChEMBL
CHEMBL4860976	↓ inhibits	Mechanistic	2100.0 nM (Ki)	ChEMBL
CHEMBL4874818	↓ inhibits	Mechanistic	1500.0 nM (Ki)	ChEMBL
CHEMBL4851756	↓ inhibits	Mechanistic	1800.0 nM (Ki)	ChEMBL
CHEMBL4872496	↓ inhibits	Mechanistic	2200.0 nM (Ki)	ChEMBL
CHEMBL4876721	↓ inhibits	Mechanistic	2300.0 nM (Ki)	ChEMBL
CHEMBL4864303	↓ inhibits	Mechanistic	2600.0 nM (Ki)	ChEMBL
CHEMBL4846039	↓ inhibits	Mechanistic	1500.0 nM (Ki)	ChEMBL
CHEMBL4875904	↓ inhibits	Mechanistic	1300.0 nM (Ki)	ChEMBL
CHEMBL4868118	↓ inhibits	Mechanistic	970.0 nM (Ki)	ChEMBL
CHEMBL4873816	↓ inhibits	Mechanistic	550.0 nM (Ki)	ChEMBL
CHEMBL4863017	↓ inhibits	Mechanistic	750.0 nM (Ki)	ChEMBL

Showing top 40 of 78 agents ranked by evidence tier.

IDH1

UniProt Ensembl DGIdb Open Targets

Therapeutic Agent	Effect Direction	Evidence Tier	Source
OLAPARIB	? unclear	Mechanistic	DGIdb
CHEMBL:CHEMBL596631	? unclear	Mechanistic	DGIdb
LOMUSTINE	? unclear	Mechanistic	DGIdb
PD173074	? unclear	Mechanistic	DGIdb
LYNGBYABELLIN A	? unclear	Mechanistic	DGIdb
GALANTAMINE HYDROBROMIDE	? unclear	Mechanistic	DGIdb
CHEMBL:CHEMBL580609	? unclear	Mechanistic	DGIdb
SAQUINAVIR	? unclear	Mechanistic	DGIdb
CHEMBL:CHEMBL1998001	? unclear	Mechanistic	DGIdb
CHEMBL:CHEMBL1588679	? unclear	Mechanistic	DGIdb
IDH305	↓ inhibits	Mechanistic	DGIdb
ANGUSTIBALIN	? unclear	Mechanistic	DGIdb
CYCLOHEXIMIDE	? unclear	Mechanistic	DGIdb
CHEMBL:CHEMBL22062	? unclear	Mechanistic	DGIdb
CHLORPROMAZINE	? unclear	Mechanistic	DGIdb
BROXYQUINOLINE	? unclear	Mechanistic	DGIdb
CHEMBL:CHEMBL600734	? unclear	Mechanistic	DGIdb
CHEMBL:CHEMBL600908	? unclear	Mechanistic	DGIdb
CHEMBL:CHEMBL548615	? unclear	Mechanistic	DGIdb
VORASIDENIB	↓ inhibits	Mechanistic	DGIdb
PANOBINOSTAT	? unclear	Mechanistic	DGIdb
CHEMBL:CHEMBL606532	? unclear	Mechanistic	DGIdb
ENNIATIN B	? unclear	Mechanistic	DGIdb
AZACITIDINE	? unclear	Mechanistic	DGIdb
CHEMBL:CHEMBL1490332	? unclear	Mechanistic	DGIdb
REV-5901	? unclear	Mechanistic	DGIdb
CHEMBL:CHEMBL1312050	? unclear	Mechanistic	DGIdb
CHEMBL:CHEMBL426	? unclear	Mechanistic	DGIdb
CUCURBITACIN B	? unclear	Mechanistic	DGIdb
XD1	? unclear	Mechanistic	DGIdb
HELENALIN	? unclear	Mechanistic	DGIdb
AZELASTINE HYDROCHLORIDE	? unclear	Mechanistic	DGIdb
CHEMBL:CHEMBL576208	? unclear	Mechanistic	DGIdb
CHEMBL:CHEMBL473721	? unclear	Mechanistic	DGIdb
CHEMBL:CHEMBL576409	? unclear	Mechanistic	DGIdb
CHEMBL:CHEMBL530291	? unclear	Mechanistic	DGIdb
CEPHALOCHROMIN	? unclear	Mechanistic	DGIdb
PURPURIN	? unclear	Mechanistic	DGIdb
METHOTREXATE	? unclear	Mechanistic	DGIdb
DIPHYLLIN	? unclear	Mechanistic	DGIdb

Showing top 40 of 531 agents ranked by evidence tier.

IDH2

UniProt Ensembl DGIdb Open Targets

Therapeutic Agent	Effect Direction	Evidence Tier	Potency	Source
ENASIDENIB	↓ inhibits	Mechanistic	340.0 nM (IC50)	DGIdb
VORASIDENIB	↓ inhibits	Mechanistic	130.0 nM (IC50)	DGIdb
DASATINIB ANHYDROUS	? unclear	Mechanistic		DGIdb
RANOSIDENIB	? unclear	Mechanistic		DGIdb
QUIZARTINIB	? unclear	Mechanistic		DGIdb
LY3410738	↓ inhibits	Mechanistic		DGIdb
CHEMBL:CHEMBL3392845	? unclear	Mechanistic		DGIdb
IVOSIDENIB	? unclear	Mechanistic		DGIdb
DUBERMATINIB	? unclear	Mechanistic		DGIdb
DECITABINE	? unclear	Mechanistic		DGIdb
VENETOCLAX	? unclear	Mechanistic		DGIdb
VORASIDENIB CITRATE	↓ inhibits	Mechanistic		DGIdb
SORAFENIB	? unclear	Mechanistic		DGIdb
CRENOLANIB	? unclear	Mechanistic		DGIdb
IDH2 MUTANT INHIBITOR SH1573	? unclear	Mechanistic		DGIdb
MIDOSTAURIN	? unclear	Mechanistic		DGIdb
CERALASERTIB	? unclear	Mechanistic		DGIdb
DAUNORUBICIN LIPOSOMAL	? unclear	Mechanistic		DGIdb
IDH2 MUTANT INHIBITOR TQB3455	? unclear	Mechanistic		DGIdb
CYTARABINE	? unclear	Mechanistic		DGIdb
GILTERITINIB	? unclear	Mechanistic		DGIdb
AZACITIDINE	? unclear	Mechanistic		DGIdb
ENASIDENIB MESYLATE	↓ inhibits	Mechanistic	123.6 nM (IC50)	DGIdb
TALAZOPARIB	? unclear	Mechanistic		DGIdb
OLAPARIB	? unclear	Mechanistic		DGIdb
ALECTINIB	? unclear	Mechanistic		DGIdb
CHEMBL3947537	↓ inhibits	Mechanistic	3800.0 nM (IC50)	ChEMBL
CHEMBL4290152	↓ inhibits	Mechanistic	50.0 nM (IC50)	ChEMBL
CHEMBL4280581	↓ inhibits	Mechanistic	50.0 nM (IC50)	ChEMBL
CHEMBL4282029	↓ inhibits	Mechanistic	50.0 nM (IC50)	ChEMBL
CHEMBL4292196	↓ inhibits	Mechanistic	50.0 nM (IC50)	ChEMBL
CHEMBL4280463	↓ inhibits	Mechanistic	50.0 nM (IC50)	ChEMBL
CHEMBL4281900	↓ inhibits	Mechanistic	50.0 nM (IC50)	ChEMBL
CHEMBL4284624	↓ inhibits	Mechanistic	50.0 nM (IC50)	ChEMBL
CHEMBL4288431	↓ inhibits	Mechanistic	50.0 nM (IC50)	ChEMBL
CHEMBL4286718	↓ inhibits	Mechanistic	50.0 nM (IC50)	ChEMBL
CHEMBL4284814	↓ inhibits	Mechanistic	50.0 nM (IC50)	ChEMBL
CHEMBL4289977	↓ inhibits	Mechanistic	100.0 nM (IC50)	ChEMBL
CHEMBL4282071	↓ inhibits	Mechanistic	100.0 nM (IC50)	ChEMBL
CHEMBL4277498	↓ inhibits	Mechanistic	50.0 nM (IC50)	ChEMBL

Showing top 40 of 163 agents ranked by evidence tier.

SF3B1

UniProt Ensembl DGIdb Open Targets

Therapeutic Agent	Effect Direction	Evidence Tier	Potency	Source
OLAPARIB	? unclear	Mechanistic		DGIdb
ETOPOSIDE	? unclear	Mechanistic		DGIdb
H3B-8800	? unclear	Mechanistic		DGIdb
CHEMBL5653589	↓ inhibits	Mechanistic	3000.8 nM (Kd)	ChEMBL
CHEMBL3752910	↓ inhibits	Mechanistic	2847.44 nM (Kd)	ChEMBL
MOLIBRESIB	↓ inhibits	Mechanistic	7501.0 nM (Kd)	ChEMBL

TERT

UniProt Ensembl DGIdb Open Targets

Therapeutic Agent	Effect Direction	Evidence Tier	Potency	Source
CHEMBL:CHEMBL17442	? unclear	Mechanistic		DGIdb
IMETELSTAT	↓ inhibits	Mechanistic		DGIdb
ARSENIC TRIOXIDE	? unclear	Mechanistic		DGIdb
OMACETAXINE MEPESUCCINATE	? unclear	Mechanistic		DGIdb
CHEMBL:CHEMBL190474	? unclear	Mechanistic		DGIdb
CHEMBL:CHEMBL91163	? unclear	Mechanistic		DGIdb
IMETELSTAT SODIUM	↓ inhibits	Mechanistic		DGIdb
SODIUM METAARSENITE	↓ inhibits	Mechanistic		DGIdb
HLA-A*0201 RESTRICTED TERT(572Y)/TERT(572) PEPTIDES VACCINE VX-001	? unclear	Mechanistic		DGIdb
HTERT VACCINE V934/V935	? unclear	Mechanistic		DGIdb
9,10-PHENANTHRENEQUINONE	? unclear	Mechanistic		DGIdb
CHEMBL:CHEMBL89977	? unclear	Mechanistic		DGIdb
IODINE I-131	? unclear	Mechanistic		DGIdb
TERTOMOTIDE	? unclear	Mechanistic		DGIdb
HTERT-LAMP MRNA-LOADED AUTOLOGOUS DENDRITIC CELL VACCINE GRNVAC1	? unclear	Mechanistic		DGIdb
ANTISENSE OLIGONUCLEOTIDES	? unclear	Mechanistic		DGIdb
TELOMESTATIN	? unclear	Mechanistic		DGIdb
CHEMBL:CHEMBL1254091	? unclear	Mechanistic		DGIdb
RECOMBINANT BRAIN-DERIVED GROWTH FACTORS	? unclear	Mechanistic		DGIdb
CHEMBL:CHEMBL89250	? unclear	Mechanistic		DGIdb
CHEMBL:CHEMBL481161	? unclear	Mechanistic		DGIdb
RALOXIFENE HYDROCHLORIDE	? unclear	Mechanistic		DGIdb
CHEMBL:CHEMBL520095	? unclear	Mechanistic		DGIdb
CHEMBL:CHEMBL457246	? unclear	Mechanistic		DGIdb
CHEMBL:CHEMBL608862	? unclear	Mechanistic		DGIdb
CHEMBL:CHEMBL2336666	? unclear	Mechanistic		DGIdb
ISOBONDUCELLIN	? unclear	Mechanistic		DGIdb
DENDRITIC CELL VACCINE	? unclear	Mechanistic		DGIdb
ERIBULIN	? unclear	Mechanistic		DGIdb
EPITALON	? unclear	Mechanistic		DGIdb
CHEMBL:CHEMBL144757	? unclear	Mechanistic		DGIdb
SURATADENOTUREV	? unclear	Mechanistic		DGIdb
RECOMBINANT VASCULAR ENDOTHELIAL GROWTH FACTOR	? unclear	Mechanistic		DGIdb
BERBERINE	? unclear	Mechanistic		DGIdb
CHEMBL:CHEMBL481160	? unclear	Mechanistic		DGIdb
CHEMBL314057	↓ inhibits	Mechanistic	7300.0 nM (IC50)	ChEMBL
CHEMBL86984	↓ inhibits	Mechanistic	9500.0 nM (IC50)	ChEMBL
CHEMBL262163	↓ inhibits	Mechanistic	45200.0 nM (IC50)	ChEMBL
CHEMBL405650	↓ inhibits	Mechanistic	11100.0 nM (IC50)	ChEMBL
CHEMBL313020	↓ inhibits	Mechanistic	10600.0 nM (IC50)	ChEMBL

Showing top 40 of 211 agents ranked by evidence tier.

TET2

UniProt Ensembl DGIdb Open Targets

Therapeutic Agent	Effect Direction	Evidence Tier	Potency	Source
SELINEXOR	? unclear	Mechanistic		DGIdb
AZACITIDINE	? unclear	Mechanistic		DGIdb
RITUXIMAB	? unclear	Mechanistic		DGIdb
QUIZARTINIB	? unclear	Mechanistic		DGIdb
TALAZOPARIB	? unclear	Mechanistic		DGIdb
HYDROCHLOROTHIAZIDE	? unclear	Mechanistic		DGIdb
ELTANEXOR	? unclear	Mechanistic		DGIdb
OLAPARIB	? unclear	Mechanistic		DGIdb
DASATINIB ANHYDROUS	? unclear	Mechanistic		DGIdb
VEMURAFENIB	? unclear	Mechanistic		DGIdb
DOXORUBICIN HYDROCHLORIDE	? unclear	Mechanistic		DGIdb
HYDROXYUREA	? unclear	Mechanistic		DGIdb
CHEMBL4457636	↓ inhibits	Mechanistic	73000.0 nM (IC50)	ChEMBL
CHEMBL4872206	↓ inhibits	Mechanistic	1200.0 nM (IC50)	ChEMBL
N-OXALYLGLYCINE	↓ inhibits	Mechanistic	19700.0 nM (IC50)	ChEMBL
CHEMBL4851104	↓ inhibits	Mechanistic	9400.0 nM (IC50)	ChEMBL
CHEMBL4855677	↓ inhibits	Mechanistic	54800.0 nM (IC50)	ChEMBL
CHEMBL4872110	↓ inhibits	Mechanistic	11000.0 nM (IC50)	ChEMBL
CHEMBL4860506	↓ inhibits	Mechanistic	10500.0 nM (IC50)	ChEMBL
CHEMBL4870693	↓ inhibits	Mechanistic	3420.0 nM (Kd)	ChEMBL
CHEMBL4859276	↓ inhibits	Mechanistic	2050.0 nM (Kd)	ChEMBL
DEFEROXAMINE	↓ inhibits	Mechanistic	46000.0 nM (IC50)	ChEMBL
CHEMBL5193408	↓ inhibits	Mechanistic	13000.0 nM (IC50)	ChEMBL
CHEMBL5205660	↓ inhibits	Mechanistic	2300.0 nM (IC50)	ChEMBL
CHEMBL5199439	↓ inhibits	Mechanistic	86600.0 nM (IC50)	ChEMBL
CHEMBL5177215	↓ inhibits	Mechanistic	23900.0 nM (IC50)	ChEMBL
CHEMBL1230640	↓ inhibits	Mechanistic	2270.0 nM (IC50)	ChEMBL
CHEMBL316034	↓ inhibits	Mechanistic	5128.61 nM (IC50)	ChEMBL
CHEMBL3646117	↓ inhibits	Mechanistic	18620.87 nM (IC50)	ChEMBL
CHEMBL4475458	↓ inhibits	Mechanistic	5495.41 nM (IC50)	ChEMBL
VADADUSTAT	↓ inhibits	Mechanistic	5011.87 nM (IC50)	ChEMBL
CHEMBL1982368	↓ inhibits	Mechanistic	3019.95 nM (IC50)	ChEMBL
CHEMBL1398529	↓ inhibits	Mechanistic	1548.82 nM (IC50)	ChEMBL
PANOBINOSTAT	↓ inhibits	Mechanistic	3801.89 nM (IC50)	ChEMBL
CHEMBL1615211	↓ inhibits	Mechanistic	12882.5 nM (IC50)	ChEMBL
CHEMBL1614745	↓ inhibits	Mechanistic	15488.17 nM (IC50)	ChEMBL
CHEMBL92309	↑ activates	Mechanistic	11481.54 nM (EC50)	ChEMBL
CHEMBL5527999	↓ inhibits	Mechanistic	250.0 nM (IC50)	ChEMBL
CHEMBL5565375	↓ inhibits	Mechanistic	250.0 nM (IC50)	ChEMBL
CHEMBL5557372	↓ inhibits	Mechanistic	23988.33 nM (IC50)	ChEMBL

Showing top 40 of 68 agents ranked by evidence tier.

TP53

UniProt Ensembl DGIdb Open Targets

Therapeutic Agent	Effect Direction	Evidence Tier	Source
PIRARUBICIN	? unclear	Mechanistic	DGIdb
OXALIPLATIN	? unclear	Mechanistic	DGIdb
SIREMADLIN	↓ inhibits	Mechanistic	DGIdb
CARBOPLATIN	? unclear	Mechanistic	DGIdb
DAUNORUBICIN HYDROCHLORIDE	? unclear	Mechanistic	DGIdb
CHEMBL:CHEMBL260560	? unclear	Mechanistic	DGIdb
MELPHALAN	? unclear	Mechanistic	DGIdb
LAPACHONE	? unclear	Mechanistic	DGIdb
VITAMIN K3	? unclear	Mechanistic	DGIdb
CHEMBL:CHEMBL260451	? unclear	Mechanistic	DGIdb
DINOSEB	? unclear	Mechanistic	DGIdb
RIMCAZOLE	? unclear	Mechanistic	DGIdb
TRACAZOLATE	? unclear	Mechanistic	DGIdb
CAPIVASERTIB	? unclear	Mechanistic	DGIdb
AZD7648	? unclear	Mechanistic	DGIdb
QUINALIZARIN	? unclear	Mechanistic	DGIdb
INOSITOL	? unclear	Mechanistic	DGIdb
ECONAZOLE NITRATE	? unclear	Mechanistic	DGIdb
L-733,060	? unclear	Mechanistic	DGIdb
U50488	? unclear	Mechanistic	DGIdb
CHEMBL:CHEMBL260028	? unclear	Mechanistic	DGIdb
CHEMBL:CHEMBL601140	? unclear	Mechanistic	DGIdb
CHEMBL:CHEMBL408563	? unclear	Mechanistic	DGIdb
ENALAPRIL MALEATE	? unclear	Mechanistic	DGIdb
HOMIDIUM BROMIDE	? unclear	Mechanistic	DGIdb
IOXYNIL	? unclear	Mechanistic	DGIdb
ACALABRUTINIB	? unclear	Mechanistic	DGIdb
INDOPROFEN	? unclear	Mechanistic	DGIdb
MERCAPTOPURINE	? unclear	Mechanistic	DGIdb
AUTOLOGOUS DENDRITIC CELLS TRANSDUCED WITH WILD-TYPE P53 ADENOVIRUS VACCINE	? unclear	Mechanistic	DGIdb
ZIRAM	? unclear	Mechanistic	DGIdb
IFOSFAMIDE	? unclear	Mechanistic	DGIdb
DOCETAXEL ANHYDROUS	? unclear	Mechanistic	DGIdb
SOTULETINIB	? unclear	Mechanistic	DGIdb
SERTRALINE HYDROCHLORIDE	? unclear	Mechanistic	DGIdb
PD166285	? unclear	Mechanistic	DGIdb
SAPANISERTIB	? unclear	Mechanistic	DGIdb
ENZALUTAMIDE	? unclear	Mechanistic	DGIdb
PYRITHIONE ZINC	? unclear	Mechanistic	DGIdb
CHEMBL:CHEMBL1169388	? unclear	Mechanistic	DGIdb

Showing top 40 of 652 agents ranked by evidence tier.

Clinical Trials

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

A Study of TAK-226 for Anemia in Japanese Patients With Lower-Risk Myelodysplastic Syndromes Recruiting

NCT07319845 Phase: 2 Started: 2026-04-22

The main aim of the study is to evaluate how TAK-226 improves symptoms of transfusion-dependent anemia in Japanese patients with lower-risk myelodysplastic syndromes. The study consists of Screening Period (up to 6 weeks), Treatment Period, Safety Follow-Up Period (8 weeks), and Long-Term Follow-Up ...

Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms Recruiting

NCT06303193 Phase: 1/2 Started: 2026-11-02

Background: Myelodysplastic syndrome (MDS) and myelodysplastic/myeloproliferative neoplasm (MDS/MPN) are blood disorders that can cause serious complications in children and adults. MDS and MDS/MPN can also progress to acute myeloid leukemia. Treatments for these disorders are risky and not always e...

A Study of Elritercept to Treat Anemia in Adults With Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS) Who Need Regular Blood Transfusions Recruiting

NCT06499285 Phase: 3 Started: 2025-05-06

The main aim of this study is to find out how well elritercept works in lowering the need for RBC transfusions. Other aims are to learn how well elritercept works in reducing the need for RBC transfusions over longer periods of time or in adults with high transfusion needs. The study will also check...

Sonrotoclax Plus Azacitidine in Patients With MDS-IB2 Recruiting

NCT07885397 Phase: 1/2 Started: 2026-10-15

This study will evaluate the safety and preliminary effectiveness of sonrotoclax combined with azacitidine in adults aged 18-70 years with newly diagnosed myelodysplastic neoplasms with increased blasts-2 (MDS-IB2). Sonrotoclax blocks BCL-2, a protein that helps cancer cells survive. Combining sonro...

Bexmarilimab + Azacitidine Versus Placebo + Azacitidine in Participants With Treatment-naïve Higher-risk Myelodysplastic Syndromes Recruiting

NCT07672769 Phase: 2 Started: 2026-10

This Phase IIb study (BEXERA) will evaluate the safety and efficacy of bexmarilimab (FP-1305), an antibody targeting Clever-1, given in combination with azacitidine compared with azacitidine plus placebo in adults with treatment-naïve higher-risk myelodysplastic syndromes (HR-MDS). Participants will...

Study of Efficacy and Safety of Eltrombopag in Lower-risk MDS Patients With Platelet Transfusion Dependence Active Not Recruiting

NCT04797000 Phase: 2 Started: 2021-05-25

This study is designed to evaluate the efficacy and safety of eltrombopag monotherapy in Japanese adult patients with platelet transfusion-dependent lower-risk Myelodysplastic syndromes (LR-MDS).

A Study of Elritercept to Treat Anemia in Adults With Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS) Recruiting

NCT04419649 Phase: 2 Started: 2020-08-19

The main aim of this study is to learn how safe elritercept is and how well adults with anemia associated with lower-risk MDS tolerate treatment with different doses of elritercept. Other aims are to learn how safe elritercept is by looking at how many participants have MDS that worsens during the s...

PK/Efficacy Bridging Study of ASTX727 in Chinese Subjects With Myelodysplastic Syndromes Active Not Recruiting

NCT06091267 Phase: 1/2 Started: 2023-10-16

This is an Open-Label, Crossover, Pharmacokinetic and Efficacy Bridging Study of Oral ASTX727 versus IV Decitabine in Chinese Subjects with Myelodysplastic Syndromes

Danvatirsen Monotherapy Followed by Combination With Venetoclax in Relapsed/Refractory MDS & AML Recruiting

NCT05986240 Phase: 1 Started: 2024-05-08

This is a Phase 1 study investigating the safety and efficacy of Danvatirsen as a monotherapy followed by combination with Venetoclax in patients with relapsed/refractory myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML). Funding Source: FDA OOPD

Venetoclax-containing Therapy Combined With Microtransplant for Intermediate-risk and Higher MDS Recruiting

NCT07238686 Phase: 2 Started: 2025-06-20

This study aims to evaluate the safety and efficacy of a Venetoclax and hypomethylating agent-based regimen combined with infusion of HLA-mismatched donor G-CSF mobilized peripheral blood mononuclear cells (GPBMC) in patients with intermediate-risk and higher myelodysplastic syndromes who are inelig...

A Multi-phase Study of ASTX030 (Azacitidine and Cedazuridine) in Myeloid Neoplasm Alone or in Combination With Venetoclax in AML (AZTOUND Study) Recruiting

NCT04256317 Phase: 2/3 Started: 2020-05-21

Study ASTX030-01 is a multi-phase study comprising of Phases 1-3 Monotherapy arms, and Phase 1 and Phase 2 Combination Therapy arms. Phase 1 Monotherapy consists of an open-label Dose Escalation Stage (Stage A) using multiple cohorts at escalating dose levels of oral cedazuridine and azacitidine (on...

Study to Evaluate Imetelstat in Patients With High-Risk MDS or AML Failing HMA-based Therapy Completed

NCT05583552 Phase: 2 Started: 2023-06-05

The purpose of this study is to evaluate the efficacy, in terms of hematologic improvement, and safety of imetelstat in participants with high-risk (HR) myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) that is relapsed/refractory to hypomethylating agents (HMAs) treatment. Responding p...

Study Evaluating Combination of Luspatercept in LR-MDS Without RS Having Failed or Being Ineligible to ESA Recruiting

NCT05181735 Phase: 1/2 Started: 2022-05-18

Study of the combination of luspatercept in low-risk myelodysplastic syndrom (LR-MDS) without ring sideroblasts (RS) having failed or being ineligible to ESA

Omacetaxine + Azacitidine in Untreated Patients With High Grade MDS Active Not Recruiting

NCT03564873 Phase: 1/2 Started: 2018-09-17

This study will treat patients with previously untreated high grade myleodysplastic syndromes (MDS) with both omacetaxine mepesuccinate and azacitidine.

A Study of Tebapivat (AG-946) in Participants With Anemia Due to Lower-Risk Myelodysplastic Syndromes (LR-MDS) Active Not Recruiting

NCT05490446 Phase: 2 Started: 2022-11-07

This purpose of this study is to establish proof of concept of tebapivat in participants with LR-MDS in Phase 2a and to evaluate the effect of tebapivat on transfusion independence (TI) in participants with LR-MDS in phase 2b.

Agents Found By Disease-Level Search

Searched directly from “Myelodysplastic Syndromes (MDS)” 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).

48 agents shown (approved or with trial evidence) — 33 not found by the gene-target search above.

cyclosporineNOT in gene-target list

Phase 3 · 10 trials · source: ClinicalTrials.gov, Open Targets
NCT00357565 NCT00003116 NCT00005854

cyclophosphamideNOT in gene-target list

Phase 3 · 8 trials · source: ClinicalTrials.gov, Open Targets
NCT00003116 NCT00005854 NCT00322101

busulfanNOT in gene-target list

Phase 3 · 7 trials · source: ClinicalTrials.gov, Open Targets
NCT00357565 NCT00003116 NCT00003335

fludarabine phosphateNOT in gene-target list

Phase 3 · 6 trials · source: ClinicalTrials.gov, Open Targets
NCT00357565 NCT00856388 NCT00112593

melphalan

Phase 3 · 6 trials · source: ClinicalTrials.gov, Open Targets
NCT00357565 NCT00856388 NCT00003335

mycophenolate mofetilNOT in gene-target list

Phase 3 · 6 trials · source: ClinicalTrials.gov, Open Targets
NCT00357565 NCT00112593 NCT02593123

Azacitidine

Approved · 6 trials · source: ClinicalTrials.gov, Open Targets
NCT02995655 NCT02038777 NCT03814005

Cytarabine

Phase 3 · 6 trials · source: ClinicalTrials.gov, Open Targets
NCT02038777 NCT05554406 NCT02521493

FLUDARABINENOT in gene-target list

Phase 3 · 5 trials · source: ClinicalTrials.gov, Open Targets
NCT04473911 NCT05534620 NCT04872595

decitabine

Approved · 5 trials · source: ClinicalTrials.gov, Open Targets
NCT00619099 NCT00619099 NCT00479232

PF-04449913NOT in gene-target list

· 5 trials · source: ClinicalTrials.gov
NCT02038777 NCT02038777 NCT02038777

methotrexate

Phase 3 · 5 trials · source: ClinicalTrials.gov, Open Targets
NCT00005854 NCT00322101 NCT00521430

filgrastimNOT in gene-target list

Approved · 4 trials · source: ClinicalTrials.gov, Open Targets
NCT00357565 NCT00003116 NCT00002832

anti-thymocyte globulinNOT in gene-target list

· 4 trials · source: ClinicalTrials.gov
NCT00856388 NCT00003335 NCT00521430

talmapimod (SCIO-469)NOT in gene-target list

· 4 trials · source: ClinicalTrials.gov
NCT00113893 NCT00113893 NCT00113893

Venetoclax

Phase 3 · 4 trials · source: ClinicalTrials.gov, Open Targets
NCT05554406 NCT04628026 NCT04988555

Liposome-encapsulated Daunorubicin-CytarabineNOT in gene-target list

· 3 trials · source: ClinicalTrials.gov
NCT04493164 NCT04802161 NCT05554406

TacrolimusNOT in gene-target list

Phase 3 · 3 trials · source: ClinicalTrials.gov, Open Targets
NCT01471444 NCT01491958 NCT00322101

Peripheral blood drawNOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT02995655 NCT04263181

Pembrolizumab

Phase 2 · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT01953692 NCT02936752

Ruxolitinib

Phase 2 · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT07238712 NCT04279847

PevonedistatNOT in gene-target list

Phase 3 · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT03814005 NCT03862157

Thiotepa

Phase 3 · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT04872595 NCT01746849

Lenalidomide

Approved · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT02279654 NCT00502112

Bone Marrow AspirationNOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT05554406 NCT03953898

Daunorubicin Hydrochloride

Phase 3 · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT05554406 NCT02521493

MY008211A tabletsNOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT06933914 NCT06050226

tosedostatNOT in gene-target list

Phase 2 · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT01567059 NCT02452346

methylprednisolone

Phase 2/3 · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT00003335 NCT00521430

EntinostatNOT in gene-target list

Phase 2 · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT02936752 NCT00015925

Lenalidomide and melphalanNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00744536

Ivosidenib

Phase 3 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT04493164

CX-01NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02995655

becatecarinNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00087204

GTB-360NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06594445

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

TBINOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04473911

Sirolimus

Phase 3 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT04473911

RGI-2001NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04473911

bezuclastinibNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04996875

Low dose ARA-C (LDAC)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02038777

DaunorubicinNOT in gene-target list

Phase 3 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT02038777

LDACNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02038777

VEN (Venetoclax)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07047183

TreosulfanNOT in gene-target list

Phase 3 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT05534620

Bortezomib

Phase 2 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT00789256

Melphalan and bortezomibNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00789256

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

Myelodysplastic Syndromes (MDS)

Remission & chronicity

Biomarker Targets & Therapeutic Agents

ACVR2B

BCL2

CD47

CRBN

DNMT1

HAVCR2

IDH1

IDH2

SF3B1

TERT

TET2

TP53

Clinical Trials

Agents Found By Disease-Level Search

The research patients need, made open source.

Conditions

Data

Tools

Foundation