Table 3 Investigational agents in clinical development for AML and/or MDS
From: Recent advances in targeted therapies in acute myeloid leukemia
Class/Pathway | Investigational agent | Alternative names | Proposed MOA/Target | AML/MDS trials |
|---|---|---|---|---|
FLT3 inhibitor | Quizartinib | Â | Type I FLT3 inhibitor | NCT04107727, NCT03135054, NCT04493138, NCT04128748, NCT03735875, NCT02668653, NCT01892371, NCT03661307, NCT03793478, NCT04047641, NCT04687761, NCT04112589, NCT02984995, NCT03723681, NCT02039726, NCT02834390, NCT01565668, NCT02675478, NCT00462761, NCT01468467, NCT01390337, NCT00989261, NCT01411267 |
Crenolanib | Â | Type I FLT3 inhibitor | NCT03258931, NCT02400255, NCT03250338, NCT01657682, NCT01522469, NCT02400281, NCT02626338, NCT02283177, NCT02270788 | |
Ponatinib | Â | Type I FLT3 inhibitor | NCT02428543, NCT03690115 | |
Luxeptinib | CG-806 | Type I FLT3 inhibitor | NCT04477291 | |
HM43239 | Â | Type I FLT3 inhibitor | NCT03850574 | |
FF-10101 | Â | Type I FLT3 inhibitor | NCT03194685, NCT02193958 | |
IDH inhibitor | LY3410738 | Â | IDH1/2 inhibitor | NCT04603001 |
BCL2/MCL1 | S65487 | VOB560 | BCL2 inhibitor | NCT04742101, NCT03755154, NCT04702425 |
S55746 | BCL201 | BCL2 inhibitor | NCT02920541 | |
AMG 176 | Â | MCL1 inhibitor | NCT05209152, NCT02675452 | |
AZD5991 | Â | MCL1 inhibitor | NCT03013998 | |
S64315 | MIK665 | MCL1 inhibitor | NCT04629443, NCT03672695, NCT02979366, NCT02992483, NCT04702425 | |
Mutant P53 | Eprenetapopt | APR-246 | Mutant p53 reactivation, cellular redox modification, ferroptosis | NCT03931291, NCT04214860, NCT03745716, NCT03072043, |
Epigenetic pathways | Ziftomenib | KO-539 | Menin-MLL inhibitor | NCT04067336 |
Revumenib | SNDX-5613, VTP50469 | Menin inhibitor | NCT05406817, NCT05326516, NCT04065399, NCT05360160, NCT03013998 | |
Pinometostat | EPZ5676 | DOT1L inhibitor | NCT03701295 | |
Iadademstat | ORY-1001 | KDM1A inhibitor | NCT05546580 | |
Entinostat | SYNDX-275, MS-275 | HDAC1/3 inhibitor | NCT00101179, NCT00313586, NCT00462605, NCT00015925, NCT02936752 | |
Other oncogenic pathways | Pevonedistat | TAK-924, MLN4924 | NEDD8 activator | NCT03459859, NCT03772925, NCT04266795, NCT03813147, NCT03268954 |
Entospletinib | GS-9973 | Syk inhibitor | NCT05020665, NCT03013998 | |
Olaparib | Â | PARP inhibitor | NCT03953898 | |
Milademetan | DS-3032, RAIN-32 | MDM2/HDM2 inhibitor | NCT03671564, NCT03634228 | |
Siremadlin | HDM201 | MDM2/HDM2 inhibitor | NCT05447663, NCT05155709 | |
Uproleselan | GMI-1271 | E-selectin antagonist | NCT04964505, NCT05569512, NCT05054543, NCT04839341, NCT04848974, NCT03616470, NCT02306291 | |
Tamibarotene | SY-1425 | Retinoic acid receptor alpha agonist | NCT04797780, NCT04905407, NCT02807558 | |
JNJ-74856665 | Â | Dihydroorotate dehydrogenase inhibitor | NCT04609826 | |
PRT543 | Â | PRMT5 inhibitor | NCT03886831 | |
H3B-8800 | RVT-2001 | Splicing modulator | NCT0281540 | |
Emavusertib | CA-4948 | IRAK4 inhibitor | NCT04278768, NCT05178342 | |
Targeted Immune inhibitors | Magrolimab | Hu5F9-G4, ONO-7913 | Anti-CD47 | NCT04313881, NCT05367401, NCT05079230, NCT04435691, NCT03248479, NCT04778397, NCT04778410, NCT02678338 |
Lemzoparlimab | TJ011133, TJC4 | Anti-CD47 | NCT04202003, NCT04912063 | |
Ipilimumab | Â | Anti-CTLA4 | NCT01757639, NCT02890329, NCT03600155, NCT02397720, NCT03912064, NCT02846376, NCT00060372, NCT01822509, NCT02530463 | |
Nivolumab | Â | Anti-PD-1 | NCT03417154, NCT02530463, NCT02464657, NCT03600155, NCT03358719, NCT03092674, NCT02846376, NCT02532231, NCT02397720, NCT01822509, NCT04913922, NCT03825367, NCT02275533, NCT04277442 | |
Pembrolizumab | Â | Anti-PD-1 | NCT02996474, NCT02845297, NCT02708641, NCT03769532, NCT02768792, NCT02771197, NCT04284787, NCT04214249, NCT03969446, NCT04372706, NCT03761914, NCT03144245, NCT02981914, NCT03094637, NCT02936752, NCT01953692 | |
Spartalizumab | PDR001 | Anti-PD-1 | NCT03066648 | |
Relatlimab | Â | Anti-LAG3 | NCT04913922 | |
Sabatolimab | MBG453 | Anti-TIM3 | NCT04812548, NCT04623216, NCT04878432, NCT05367401, NCT05201066, NCT04150029, NCT03946670, NCT04266301 | |
Decoy receptor | Evorpacept | ALX148 | Anti-CD47 | NCT04755244, NCT04417517 |
ADC | IMGN632 | Â | Anti-CD123 | NCT03386513, NCT04086264, NCT05320380 |
Cusatuzumab | ARGX-110 | Anti-CD70 | NCT04023526, NCT04241549, NCT04150887, NCT03030612 | |
Vadastuximab | SGN-CD33A | Anti-CD33 | NCT02326584, NCT01902329 | |
BITE/DART | AMV564 | Â | CD33 x CD3 | NCT03144245, NCT03516591 |
AMG 427 | Â | FLT3 x CD3 | NCT03541369 | |
Flotetuzumab | MGD006 | CD123 x CD3 | NCT05506956, NCT04158739, NCT04582864, NCT04681105 | |
Vibecotamab | XmAB14045 | CD123 x CD3 | NCT02730312, NCT05285813 | |
APVO436 | Â | CD123 x CD3 | NCT03647800 |