Biomarker & Intervention Discovery

Polycythaemia Vera / Myeloproliferative Neoplasms

This page lists 11 druggable gene targets, 54 approved or trial-tested drug candidates and 15 recent clinical trials for Polycythaemia Vera / Myeloproliferative Neoplasms, 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 remissionPV/ET/MF: no spontaneous remission; progressive disorders with risk of transformation to MF (PV/ET) and blast phase…
Best-intervention remissionJAK2 inhibitor (ruxolitinib) for MF/PV: 35-42% spleen volume reduction, significant symptom improvement…
Gap sizeModerate — ruxolitinib is established but HSCT underused in eligible MF patients; ropeginterferon is superior in PV…
Primary barrieraccess/practice
Full remission profile →
Research Tracker Chronic Disease Interventions Polycythaemia Vera / Myeloproliferative Neoplasms

Remission & chronicity

Spontaneous remission
PV/ET/MF: no spontaneous remission; progressive disorders with risk of transformation to MF (PV/ET) and blast phase (all); median survival PV >20 years, MF 5-7 years
Best-intervention remission
JAK2 inhibitor (ruxolitinib) for MF/PV: 35-42% spleen volume reduction, significant symptom improvement; ropeginterferon alfa-2b: superior complete haematological response vs hydroxyurea (PROUD-PV); allogeneic HSCT (MF): curative in 30-60% of transplant-eligible patients
Gap size
Moderate — ruxolitinib is established but HSCT underused in eligible MF patients; ropeginterferon is superior in PV but access varies
Primary barrier
access/practice
Hydroxyurea remains the most common cytoreductive agent for PV/ET despite ropeginterferon demonstrating superior molecular response; HSCT for MF is underutilised in patients who could benefit; JAK2 mutation molecular monitoring not performed systematically

Biomarker Targets & Therapeutic Agents

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

BCL2L1 110BRD4 217CALR 10HAMP 6IFNAR1 20JAK2 307KDM1A 169MDM2 161MPL 180TERT 211TET2 68

BCL2L1

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
APOGOSSYPOL ? unclear Mechanistic DGIdb
NAVITOCLAX ↓ inhibits Mechanistic DGIdb
SANGIVAMYCIN ? unclear Mechanistic DGIdb
GOSSYPOL ? unclear Mechanistic 500.0 nM (IC50) DGIdb
ABT-737 ↓ inhibits Mechanistic DGIdb
FOSELUTOCLAX ↓ inhibits Mechanistic DGIdb
A-1331852 ↓ inhibits Mechanistic DGIdb
PELCITOCLAX ? unclear Mechanistic DGIdb
APG-1252-M1 ↓ inhibits Mechanistic DGIdb
LISAFTOCLAX ↓ inhibits Mechanistic DGIdb
VENETOCLAX ↓ inhibits Mechanistic DGIdb
OBATOCLAX ↓ inhibits Mechanistic DGIdb
AZD4320 ↓ inhibits Mechanistic DGIdb
OBATOCLAX MESYLATE ↓ inhibits Mechanistic DGIdb
CHEMBL332055 ↓ inhibits Mechanistic 180000.0 nM (IC50) ChEMBL
CHEMBL330959 ↓ inhibits Mechanistic 10000.0 nM (IC50) ChEMBL
CHEMBL334615 ↓ inhibits Mechanistic 2600.0 nM (IC50) ChEMBL
PURPUROGALLIN ↓ inhibits Mechanistic 2200.0 nM (IC50) ChEMBL
CHEMBL339835 ↓ inhibits Mechanistic 7400.0 nM (IC50) ChEMBL
CHEMBL135650 ↓ inhibits Mechanistic 73000.0 nM (IC50) ChEMBL
CHEMBL28137 ↓ inhibits Mechanistic 781.0 nM (Ki) ChEMBL
CHEMBL28872 ↓ inhibits Mechanistic 1785.0 nM (Ki) ChEMBL
CHEMBL286715 ↓ inhibits Mechanistic 839.0 nM (Ki) ChEMBL
CHEMBL28478 ↓ inhibits Mechanistic 7631.0 nM (Ki) ChEMBL
CHEMBL281240 ↓ inhibits Mechanistic 3313.0 nM (Ki) ChEMBL
CHEMBL284686 ↓ inhibits Mechanistic 1852.0 nM (Ki) ChEMBL
CHEMBL433422 ↓ inhibits Mechanistic 5846.0 nM (Ki) ChEMBL
CHEMBL28926 ↓ inhibits Mechanistic 10920.0 nM (Ki) ChEMBL
CHEMBL28504 ↓ inhibits Mechanistic 10960.0 nM (Ki) ChEMBL
CHEMBL282624 ↓ inhibits Mechanistic 1013.0 nM (Ki) ChEMBL
CHEMBL371861 ↓ inhibits Mechanistic 122.0 nM (IC50) ChEMBL
CHEMBL383399 ↓ inhibits Mechanistic 1.7 nM (Ki) ChEMBL
CHEMBL380709 ↓ inhibits Mechanistic 0.8 nM (Ki) ChEMBL
CHEMBL382648 ↓ inhibits Mechanistic 0.9 nM (Ki) ChEMBL
CHEMBL202964 ↓ inhibits Mechanistic 250.0 nM (Ki) ChEMBL
CHEMBL379013 ↓ inhibits Mechanistic 3.1 nM (Ki) ChEMBL
CHEMBL378820 ↓ inhibits Mechanistic 75.0 nM (Ki) ChEMBL
CHEMBL198903 ↓ inhibits Mechanistic 1.1 nM (Ki) ChEMBL
CHEMBL370836 ↓ inhibits Mechanistic 1.0 nM (Ki) ChEMBL
CHEMBL370835 ↓ inhibits Mechanistic 1.2 nM (Ki) ChEMBL

Showing top 40 of 110 agents ranked by evidence tier.

BRD4

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
COMPOUND 11H [PMID: 29808961] ↓ inhibits Mechanistic DGIdb
MIVEBRESIB ↓ inhibits Mechanistic DGIdb
ARV-771 ↓ inhibits Mechanistic DGIdb
CHEMBL:CHEMBL52030 ? unclear Mechanistic DGIdb
(+)-JQ1 ↓ inhibits Mechanistic DGIdb
FEDRATINIB ↓ inhibits Mechanistic DGIdb
ALPRAZOLAM ? unclear Mechanistic 2460.0 nM (Kd) DGIdb
MS436 ↓ inhibits Mechanistic DGIdb
BRD4 INHIBITOR PLX2853 ? unclear Mechanistic DGIdb
COMPOUND 6 [PMID: 30125504] ↓ inhibits Mechanistic DGIdb
BMS-986158 ↓ inhibits Mechanistic DGIdb
ESTAZOLAM ? unclear Mechanistic DGIdb
MZ1 ↓ inhibits Mechanistic DGIdb
I-BET282 ↓ inhibits Mechanistic DGIdb
DBET6 ↓ inhibits Mechanistic DGIdb
I-BET151 ↓ inhibits Mechanistic DGIdb
ZAVABRESIB ↓ inhibits Mechanistic DGIdb
COMPOUND 1 [PMID: 25408830] ↓ inhibits Mechanistic DGIdb
LY-294002 ↓ inhibits Mechanistic DGIdb
MIDAZOLAM HYDROCHLORIDE ? unclear Mechanistic DGIdb
NHWD-870 ↓ inhibits Mechanistic DGIdb
ISOXAZOLE AZEPINE COMPOUND 3 ↓ inhibits Mechanistic DGIdb
BIVALENT BRD4 INHIBITOR AZD5153 ? unclear Mechanistic DGIdb
PELABRESIB ↓ inhibits Mechanistic DGIdb
AMREDOBRESIB ↓ inhibits Mechanistic DGIdb
COMPOUND 7D [PMID: 25703523] ↓ inhibits Mechanistic DGIdb
MS417 ↓ inhibits Mechanistic DGIdb
BI-2536 ↓ inhibits Mechanistic DGIdb
DUAL BRD4/PLK1 INHIBITOR 23 ↓ inhibits Mechanistic DGIdb
TEC4 ↓ inhibits Mechanistic DGIdb
TRIAZOLAM ? unclear Mechanistic DGIdb
MOLIBRESIB ↓ inhibits Mechanistic 55.2 nM (Kd) DGIdb
N-METHYL-2-PYRROLIDONE ↓ inhibits Mechanistic DGIdb
COMPOUND 36 [PMID: 24000170] ↓ inhibits Mechanistic DGIdb
PFI-1 ↓ inhibits Mechanistic DGIdb
ABBV-744 ? unclear Mechanistic DGIdb
TRICHOSTATIN A ? unclear Mechanistic DGIdb
BZT-7 ↓ inhibits Mechanistic DGIdb
COMPOUND 3 [PMID: 25408830] ↓ inhibits Mechanistic DGIdb
XY-06-007 ↓ inhibits Mechanistic DGIdb

Showing top 40 of 217 agents ranked by evidence tier.

CALR

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
ANGIOGENESIS INHIBITOR ? unclear Mechanistic DGIdb
TRETINOIN ? unclear Mechanistic DGIdb
PEGINTERFERON ALFA-2A ? unclear Mechanistic DGIdb
GENTAMICIN ? unclear Mechanistic DGIdb
THERAPEUTIC GLUCOCORTICOID ? unclear Mechanistic DGIdb
OLTIPRAZ ? unclear Mechanistic DGIdb
RETINOIC ACID AGENT ? unclear Mechanistic DGIdb
VACCINIA VACCINE ? unclear Mechanistic DGIdb
CHEMBL5653589 ↓ inhibits Mechanistic 24526.01 nM (Kd) ChEMBL
CHEMBL3752910 ↓ inhibits Mechanistic 24341.74 nM (Kd) ChEMBL

HAMP

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
LEXAPTEPID PEGOL ? unclear Mechanistic DGIdb
LY-2787106 ↓ inhibits Mechanistic DGIdb
CHEMBL5199417 ↓ inhibits Mechanistic 28.0 nM (IC50) ChEMBL
CHEMBL5184000 ↓ inhibits Mechanistic 7.3 nM (IC50) ChEMBL
CHEMBL5186341 ↓ inhibits Mechanistic 58.0 nM (IC50) ChEMBL
CHEMBL5181536 ↓ inhibits Mechanistic 34.0 nM (IC50) ChEMBL

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

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.

KDM1A

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
CHEMBL:CHEMBL2182009 ? unclear Mechanistic DGIdb
CC-90011 ↓ inhibits Mechanistic DGIdb
OG-L002 ↓ inhibits Mechanistic DGIdb
NAMOLINE ? unclear Mechanistic 51000.0 nM (IC50) DGIdb
RN-1 ↓ inhibits Mechanistic DGIdb
NCL-1 ↓ inhibits Mechanistic DGIdb
BOMEDEMSTAT ↓ inhibits Mechanistic DGIdb
ORY-1001 ↑ activates Mechanistic DGIdb
CHEMBL:CHEMBL1179 ? unclear Mechanistic DGIdb
TRANYLCYPROMINE HYDROCHLORIDE ? unclear Mechanistic 46300.0 nM (Kd) DGIdb
GSK2879552 ↓ inhibits Mechanistic DGIdb
TAK-418 ↓ inhibits Mechanistic DGIdb
GSK-LSD1 ↓ inhibits Mechanistic DGIdb
TRANYLCYPROMINE ↓ inhibits Mechanistic 21000.0 nM (Ki) DGIdb
SALVIANOLIC ACID B ↓ inhibits Mechanistic DGIdb
CHEMBL1086217 ↓ inhibits Mechanistic 16600.0 nM (Ki) ChEMBL
CHEMBL1086216 ↓ inhibits Mechanistic 15600.0 nM (IC50) ChEMBL
PHENELZINE ↓ inhibits Mechanistic 17600.0 nM (Ki) ChEMBL
CHEMBL1215658 ↓ inhibits Mechanistic 2000.0 nM (IC50) ChEMBL
CHEMBL1797639 ↓ inhibits Mechanistic 2000.0 nM (IC50) ChEMBL
CHEMBL1795981 ↓ inhibits Mechanistic 1000.0 nM (Ki) ChEMBL
CHEMBL1797640 ↓ inhibits Mechanistic 1000.0 nM (Ki) ChEMBL
CHEMBL1797641 ↓ inhibits Mechanistic 1000.0 nM (Ki) ChEMBL
CHEMBL1797642 ↓ inhibits Mechanistic 9000.0 nM (IC50) ChEMBL
CHEMBL1797643 ↓ inhibits Mechanistic 9000.0 nM (IC50) ChEMBL
CHEMBL1797644 ↓ inhibits Mechanistic 9000.0 nM (IC50) ChEMBL
CHEMBL1795980 ↓ inhibits Mechanistic 610.0 nM (Ki) ChEMBL
CHEMBL1797645 ↓ inhibits Mechanistic 1500.0 nM (Ki) ChEMBL
CHEMBL1797646 ↓ inhibits Mechanistic 1800.0 nM (Ki) ChEMBL
CHEMBL1797647 ↓ inhibits Mechanistic 50.0 nM (Ki) ChEMBL
CHEMBL1797650 ↓ inhibits Mechanistic 900.0 nM (Ki) ChEMBL
CHEMBL1797651 ↓ inhibits Mechanistic 700.0 nM (Ki) ChEMBL
CHEMBL1797652 ↓ inhibits Mechanistic 4400.0 nM (Ki) ChEMBL
CHEMBL1797649 ↓ inhibits Mechanistic 2800.0 nM (Ki) ChEMBL
CHEMBL1797648 ↓ inhibits Mechanistic 8300.0 nM (Ki) ChEMBL
CHEMBL1797653 ↓ inhibits Mechanistic 2700.0 nM (Ki) ChEMBL
CHEMBL1797654 ↓ inhibits Mechanistic 6110.0 nM (Ki) ChEMBL
CHEMBL1797655 ↓ inhibits Mechanistic 24200.0 nM (Ki) ChEMBL
CHEMBL1179 ↓ inhibits Mechanistic 21000.0 nM (IC50) ChEMBL
CHEMBL1797705 ↓ inhibits Mechanistic 1600.0 nM (IC50) ChEMBL

Showing top 40 of 169 agents ranked by evidence tier.

MDM2

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
LISSOCLINIDINE B ? unclear Mechanistic 58600.0 nM (IC50) DGIdb
THIRAM ? unclear Mechanistic DGIdb
PEMBROLIZUMAB ? unclear Mechanistic DGIdb
TCDD ? unclear Mechanistic DGIdb
MILADEMETAN TOSYLATE ? unclear Mechanistic DGIdb
ALRIZOMADLIN ? unclear Mechanistic DGIdb
IPILIMUMAB ? unclear Mechanistic DGIdb
SIREMADLIN ↓ inhibits Mechanistic DGIdb
MILADEMETAN ? unclear Mechanistic DGIdb
SERDEMETAN ? unclear Mechanistic DGIdb
NAVTEMADLIN ↓ inhibits Mechanistic DGIdb
SULANEMADLIN ? unclear Mechanistic DGIdb
MEK INHIBITOR PD-98059 ? unclear Mechanistic DGIdb
ANGIOGENESIS INHIBITOR ? unclear Mechanistic DGIdb
NUTLIN-3 ? unclear Mechanistic 36.0 nM (Ki) DGIdb
CISPLATIN ? unclear Mechanistic DGIdb
JN122 ↓ inhibits Mechanistic DGIdb
IDASANUTLIN ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL1492017 ? unclear Mechanistic DGIdb
DIPLAMINE B ? unclear Mechanistic DGIdb
NIVOLUMAB ? unclear Mechanistic DGIdb
HELENALIN ? unclear Mechanistic DGIdb
HDM2 INHIBITOR MK-8242 ? unclear Mechanistic DGIdb
MANGOSTIN ? unclear Mechanistic DGIdb
MDM2 ANTAGONIST RO5045337 ? unclear Mechanistic DGIdb
GAMBOGIC ACID ? unclear Mechanistic DGIdb
RG7112 ↓ inhibits Mechanistic DGIdb
CHEMBL178578 ↓ inhibits Mechanistic 1700.0 nM (IC50) ChEMBL
CHEMBL361103 ↓ inhibits Mechanistic 200.0 nM (IC50) ChEMBL
CHEMBL182051 ↓ inhibits Mechanistic 30000.0 nM (IC50) ChEMBL
CHEMBL360540 ↓ inhibits Mechanistic 13000.0 nM (IC50) ChEMBL
CHEMBL427316 ↓ inhibits Mechanistic 3600.0 nM (IC50) ChEMBL
CHEMBL425528 ↓ inhibits Mechanistic 15000.0 nM (IC50) ChEMBL
CHEMBL267823 ↓ inhibits Mechanistic 6670.0 nM (Ki) ChEMBL
CHEMBL409889 ↓ inhibits Mechanistic 39.0 nM (Ki) ChEMBL
CHEMBL378638 ↓ inhibits Mechanistic 4000.0 nM (Ki) ChEMBL
CHEMBL379173 ↓ inhibits Mechanistic 3.0 nM (Ki) ChEMBL
CHEMBL210789 ↓ inhibits Mechanistic 44.0 nM (Ki) ChEMBL
CHEMBL377775 ↓ inhibits Mechanistic 170.0 nM (Ki) ChEMBL
CHEMBL210611 ↓ inhibits Mechanistic 38.0 nM (Ki) ChEMBL

Showing top 40 of 161 agents ranked by evidence tier.

MPL

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
HETROMBOPAG OLAMINE ↑ activates Mechanistic DGIdb
HETROMBOPAG ↑ activates Mechanistic DGIdb
AVATROMBOPAG ↑ activates Mechanistic DGIdb
ELTROMBOPAG ↑ activates Mechanistic 38.0 nM (EC50) DGIdb
ROMIPLOSTIM ↑ activates Mechanistic DGIdb
THROMBOPOIETIN ↑ activates Mechanistic DGIdb
AVATROMBOPAG MALEATE ↑ activates Mechanistic DGIdb
ELTROMBOPAG OLAMINE ↑ activates Mechanistic DGIdb
RAFUTROMBOPAG ↑ activates Mechanistic DGIdb
ELTROMBOPAG CHOLINE ↑ activates Mechanistic DGIdb
LUSUTROMBOPAG ↑ activates Mechanistic DGIdb
TOTROMBOPAG ? unclear Mechanistic DGIdb
CHEMBL3144682 ↑ activates Mechanistic 400.0 nM (EC50) ChEMBL
CHEMBL125996 ↑ activates Mechanistic 2000.0 nM (EC50) ChEMBL
CHEMBL3144783 ↑ activates Mechanistic 70.0 nM (EC50) ChEMBL
CHEMBL122504 ↑ activates Mechanistic 2000.0 nM (EC50) ChEMBL
CHEMBL3144667 ↑ activates Mechanistic 10000.0 nM (EC50) ChEMBL
CHEMBL3144785 ↑ activates Mechanistic 2000.0 nM (EC50) ChEMBL
CHEMBL3144672 ↑ activates Mechanistic 200.0 nM (EC50) ChEMBL
CHEMBL3144775 ↑ activates Mechanistic 20.0 nM (EC50) ChEMBL
CHEMBL340040 ↑ activates Mechanistic 3000.0 nM (EC50) ChEMBL
CHEMBL3144788 ↑ activates Mechanistic 3000.0 nM (EC50) ChEMBL
CHEMBL3144786 ↑ activates Mechanistic 700.0 nM (EC50) ChEMBL
CHEMBL3144789 ↑ activates Mechanistic 2000.0 nM (EC50) ChEMBL
CHEMBL124367 ↑ activates Mechanistic 200.0 nM (EC50) ChEMBL
CHEMBL3144776 ↑ activates Mechanistic 500.0 nM (EC50) ChEMBL
CHEMBL3144677 ↑ activates Mechanistic 1000.0 nM (EC50) ChEMBL
CHEMBL121918 ↑ activates Mechanistic 600.0 nM (EC50) ChEMBL
CHEMBL3144778 ↑ activates Mechanistic 70.0 nM (EC50) ChEMBL
CHEMBL3144665 ↑ activates Mechanistic 10000.0 nM (EC50) ChEMBL
CHEMBL3144780 ↑ activates Mechanistic 2000.0 nM (EC50) ChEMBL
CHEMBL3144666 ↑ activates Mechanistic 100.0 nM (EC50) ChEMBL
CHEMBL3144790 ↑ activates Mechanistic 900.0 nM (EC50) ChEMBL
CHEMBL3144676 ↑ activates Mechanistic 7000.0 nM (EC50) ChEMBL
CHEMBL3144836 ↑ activates Mechanistic 8000.0 nM (EC50) ChEMBL
CHEMBL3144675 ↑ activates Mechanistic 5000.0 nM (EC50) ChEMBL
CHEMBL3144787 ↑ activates Mechanistic 2000.0 nM (EC50) ChEMBL
CHEMBL3144670 ↑ activates Mechanistic 300.0 nM (EC50) ChEMBL
CHEMBL3144781 ↑ activates Mechanistic 300.0 nM (EC50) ChEMBL
CHEMBL3144668 ↑ activates Mechanistic 5000.0 nM (EC50) ChEMBL

Showing top 40 of 180 agents ranked by evidence tier.

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.

Clinical Trials

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

MITHRIDATE: Ruxolitinib Versus Hydroxycarbamide or Interferon as First Line Therapy in High Risk Polycythemia Vera Recruiting
NCT04116502 Phase: 3 Started: 2019-10-25

The trial will be a phase III, randomised-controlled, multi-centre, international, open-label trial consisting of ruxolitinib versus best available therapy, where best available therapy is a choice of interferon alpha, any formulation permitted (IFN) or hydroxycarbamide (HC), and which will be elect...

INTREPID: A Study of Sapablursen Evaluating the Safety and Efficacy in Participants With Polycythemia Vera (PV) Recruiting
NCT07429266 Phase: 3 Started: 2026-05-25

The purpose of this study is to evaluate the efficacy and safety of sapablursen when added on to current standard of care (SOC) for Polycythemia Vera (PV) therapy. The study will be conducted in three sequential parts (Part 1a blinded treatment, Part 1b open-label treatment, \& Part 2 long-term exte...

A Clinical Trial of Bomedemstat in Participants With Polycythemia Vera (MK-3543-025). Recruiting
NCT07722611 Phase: 2/3 Started: 2026-10-21

Researchers are looking for new ways to treat polycythemia vera (PV). People with PV may receive treatment to lower the number of red blood cells in the blood, but the usual treatments may not work for everyone. Researchers want to learn if a trial medicine called bomedemstat, also called MK-3543, c...

A Study of Rusfertide in Adults With Polycythemia Vera (PV) Active Not Recruiting
NCT05210790 Phase: 3 Started: 2022-04-11

Polycythemia vera (PV) is a long-term condition in which the bone marrow makes too many red blood cells (RBCs). Bone marrow is the soft tissue inside the large bones where blood cells are made. When there are too many RBCs, called erythrocytosis, the blood can become thicker and move less easily thr...

Study of INCA036978 in Participants With Myeloproliferative Neoplasms Recruiting
NCT07441694 Phase: 1 Started: 2026-05-11

This study will be conducted to determine the safety, tolerability, dose-limiting toxicity (DLT)s, and maximum tolerated dose (MTD) and/or recommended dose for expansion (RDE)s of INCA036978 administered as monotherapy and in combination with a standard disease-directed therapy.

A Study of Rusfertide in Japanese Adults With Polycythemia Vera Recruiting
NCT07648030 Phase: 2 Started: 2026-07-14

Polycythemia vera (PV) is a rare blood cancer in which the body makes too many red blood cells. This can make the blood thicker and may increase the risk of serious health problems such as blood clots. Many people with PV need regular phlebotomy, which is a procedure to remove blood, to help keep th...

A Phase 1 Study of PRT12396 in Participants With Select Myeloproliferative Neoplasms Recruiting
NCT07469891 Phase: 1 Started: 2026-04-29

This is a first-in-human, open-label, multi-center Phase 1 study designed to evaluate the safety, tolerability, pharmacokinetics, and preliminary efficacy of PRT12396 in participants with high-risk polycythemia vera (PV) and myelofibrosis (MF), and to determine the maximum tolerated dose (MTD) and r...

Study of BEBT-507 Injection in Subjects With Polycythemia Vera (PV) Recruiting
NCT07012109 Phase: 1 Started: 2025-06-17

This is a multicenter, open-label Phase I clinical trial of BEBT-507 in subjects with polycythemia vera(PV). Phase Ia is a single-agent dose-escalation study designed to evaluate the safety, tolerability, pharmacokinetics (PK), preliminary efficacy, and pharmacodynamics of BEBT-507 in subjects with ...

Phase IIa Study of Flonoltinib Maleate Tablets in the Treatment of Patients With Polycythemia Vera Recruiting
NCT07232290 Phase: 2 Started: 2025-09-23

This trial adopts an open, randomized, parallel controlled, multicenter clinical trial design planning to enroll patients with polycythemia vera who are resistant/intolerant to hydroxyurea or interferon. The study divided into two stages: dose exploration stage: three dose groups are set, with three...

A Phase 2, Open-Label Study of DISC-3405 in Participants With Polycythemia Vera (PV) Active Not Recruiting
NCT06985147 Phase: 2 Started: 2025-08-12

This open-label, multicenter, within-participant dose escalation study examining up to 2 dose levels of DISC-3405 will assess the safety, tolerability, efficacy, pharmacokinetics, and pharmacodynamics of DISC-3405 in participants with polycythemia vera (PV).

A Study to Evaluate Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx) in Patients With Polycythemia Vera Completed
NCT05143957 Phase: 2 Started: 2021-12-30

The main purpose of this study is to evaluate the efficacy of sapablursen in reducing the frequency of phlebotomy and in improving quality of life assessments in participants with polycythemia vera.

A Clinical Study of Gecacitinib Combined With Pegylated Interferon in Patients With PV Recruiting
NCT07445893 Phase: N/A Started: 2026-04-02

The goal of this clinical trial is to evaluate the efficacy and safety of Gecacitinib in combination with pegylated interferon for the treatment of polycythemia vera (PV).The main question it aims to answer is: Can PV patients achieve hematological remission after receiving the combination therapy? ...

A Study of Bomedemstat (IMG-7289/MK-3543) in Participants With Polycythemia Vera (IMG-7289-CTP-203/MK-3543-004) Completed
NCT05558696 Phase: 2 Started: 2023-09-07

This study will evaluate the safety, efficacy, pharmacokinetics (PK), and pharmacodynamics of the orally administered lysine-specific demethylase 1 (LSD1) inhibitor bomedemstat, in participants with polycythemia vera (PV). At Week 36 of dosing, participants will be assessed for eligibility to receiv...

Study on Efficacy and Safety of Givinostat Versus Hydroxyurea in Patients With Polycythemia Vera Recruiting
NCT06093672 Phase: 3 Started: 2024-03-26

The goal of this clinical trial is to compare the efficacy and safety of givinostat to hydroxyurea in Jak2V617F-positive high risk polycythemia vera patients.

AVAJAK: Apixaban/Rivaroxaban Versus Aspirin for Primary Prevention of Thrombo-embolic Complications in JAK2V617F-positive Myeloproliferative Neoplasms Recruiting
NCT05198960 Phase: 3 Started: 2022-07-13

Philadelphia-negative myeloproliferative neoplasms (MPN) are frequent and chronic myeloid malignancies including Polycythemia Vera (PV), essential thrombocythemia (ET), Primary Myelofibrosis (PMF) and Prefibrotic myelofibrosis (PreMF). These MPNs are caused by the acquisition of mutations affecting ...

Agents Found By Disease-Level Search

Searched directly from “Polycythaemia Vera / Myeloproliferative Neoplasms” 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).

54 agents shown (approved or with trial evidence) — 45 not found by the gene-target search above.

Ruxolitinib

Approved · 15 trials · source: ClinicalTrials.gov, Open Targets
NCT05714072 NCT04116502 NCT04644211

fludarabine phosphateNOT in gene-target list

· 8 trials · source: ClinicalTrials.gov
NCT00112593 NCT00003816 NCT00134004

cyclophosphamideNOT in gene-target list

Phase 2 · 8 trials · source: ClinicalTrials.gov, Open Targets
NCT00002792 NCT00003816 NCT00134004

busulfanNOT in gene-target list

· 6 trials · source: ClinicalTrials.gov
NCT00002792 NCT00003816 NCT00054340

tacrolimusNOT in gene-target list

· 6 trials · source: ClinicalTrials.gov
NCT00002792 NCT00134004 NCT00104975

cyclosporineNOT in gene-target list

Phase 2 · 5 trials · source: ClinicalTrials.gov, Open Targets
NCT00112593 NCT00002792 NCT00080925

mycophenolate mofetilNOT in gene-target list

· 5 trials · source: ClinicalTrials.gov
NCT00112593 NCT00134004 NCT00245037

methotrexateNOT in gene-target list

Phase 2 · 4 trials · source: ClinicalTrials.gov, Open Targets
NCT00002792 NCT00054340 NCT00296023

PEGASYSNOT in gene-target list

· 4 trials · source: ClinicalTrials.gov
NCT01259817 NCT02407080 NCT01387763

FludarabineNOT in gene-target list

Phase 2 · 4 trials · source: ClinicalTrials.gov, Open Targets
NCT00683046 NCT00475020 NCT05031897

MelphalanNOT in gene-target list

· 4 trials · source: ClinicalTrials.gov
NCT00683046 NCT05031897 NCT00003816

anti-thymocyte globulinNOT in gene-target list

· 4 trials · source: ClinicalTrials.gov
NCT00003816 NCT00054340 NCT00006379

therapeutic allogeneic lymphocytesNOT in gene-target list

· 4 trials · source: ClinicalTrials.gov
NCT00080925 NCT00104975 NCT00296023

Best Available Therapy (BAT)NOT in gene-target list

· 3 trials · source: ClinicalTrials.gov
NCT02101268 NCT03952039 NCT02218047

pomalidomide (CC-4047)NOT in gene-target list

Approved · 3 trials · source: ClinicalTrials.gov
NCT00669578 NCT00946270 NCT00946270

Azacitidine

· 2 trials · source: ClinicalTrials.gov
NCT03862157 NCT02129101

fedratinib (SAR302503)NOT in gene-target list

Approved · 2 trials · source: ClinicalTrials.gov
NCT01420783 NCT01692366

Momelotinib

Phase 2 · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT01969838 NCT02101268

etoposideNOT in gene-target list

Phase 2 · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT00003816 NCT00080925

thiotepaNOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT00003816 NCT00104975

filgrastimNOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT00080925 NCT00296023

graft-versus-tumor induction therapyNOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT00080925 NCT00006379

prednisoneNOT in gene-target list

Phase 2 · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT00080925 NCT00946270

recombinant interferon alfaNOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT00002504 NCT00003451

Low-dose aspirinNOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT04182100 NCT05198960

PegIntronNOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT01387763 NCT01387763

AbemaciclibNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05714072

PEG-P-INF alpha-2b (P1101)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01193699

bomedemstat (IMG-7289)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04262141

Open-label rusfertideNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06033586

HydroxycarbamideNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04116502

Interferon-Alpha

· 1 trial · source: ClinicalTrials.gov
NCT04116502

luspatercept (ACE-536)NOT in gene-target list

Approved · 1 trial · source: ClinicalTrials.gov
NCT04717414

sonidegib (LDE225)NOT in gene-target list

Approved · 1 trial · source: ClinicalTrials.gov
NCT01787552

ruxolitinib (INC424)NOT in gene-target list

Approved · 1 trial · source: ClinicalTrials.gov
NCT01787552

PXS-5505NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04676529

INCB057643NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04279847

Pembrolizumab

· 1 trial · source: ClinicalTrials.gov
NCT03065400

Apixaban 2.5 MG Oral Tablet [ELIQUIS]NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04243122

Aspirin 81 mgNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04243122

PevonedistatNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03862157

Venetoclax

· 1 trial · source: ClinicalTrials.gov
NCT03862157

PracinostatNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02267278

panobinostat

· 1 trial · source: ClinicalTrials.gov
NCT02386800

forodesine hydrochlorideNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00073944

umbralisib (TGR-1202)NOT in gene-target list

Approved · 1 trial · source: ClinicalTrials.gov
NCT02493530

Talazoparib

· 1 trial · source: ClinicalTrials.gov
NCT06218628

pacritinib

· 1 trial · source: ClinicalTrials.gov
NCT06218628

AspirinNOT in gene-target list

Phase 2 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT01259817

Placebo to match momelotinibNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01969838

Placebo to match ruxolitinibNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01969838

momelotinib (CYT387)NOT in gene-target list

Approved · 1 trial · source: ClinicalTrials.gov
NCT00935987

Stem cellsNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00683046

CampathNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00683046

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