Table 1 Examples of epigenetic inhibitors in current clinical development.
Inhibitor Target /Class | Drug Name | Alternative Drug Names | Clinical Trial Phase (ongoing / completed)* | Myeloid Neoplasm Trial Indication [Trial Phase] | Approval [Y/N, Year (FDA/EMA)] | Approval Indication** |
|---|---|---|---|---|---|---|
BET-Inhibitors | Pelabresib | CPI-0610 | 1–3 (completed) | Myeloid neoplasms [1/2], MF [1–3] | N | N.A. |
Ezobresib | BMS-986158 | 1/2 (completed) | MF | N | N. A. | |
DOT1L-Inhibitor | Pinometostat | EPZ-5676 | 1/2, 2 (completed) | AML (KMT2A-r) | N | N. A. |
HDAC-Inhibitors | Givinostat | ITF-2357 | 2–3 (ongoing) | PV [2,3] | N | N. A. |
Panobinostat | LBH589 | 2 (completed)* | CML, MDS, MF | N | N. A. | |
Vorinostat | Suberoylanilide Hydroxamic Acid | 1/2, 2 (completed) | AML [2], MDS [1/2] | N | N. A. | |
Hypomethylating Agents (HMAs) | Azacitidine | 5-Azacytidine | 1, 1/2 (ongoing) 3 (completed) | AML, MDS, MDS/MPN | Y (2004/2008) | MDS, JMML (FDA); MDS, AML (EMA) |
Oral Azacitidine | CC-486 | 1–2 (ongoing), 3 (completed) | AML, MDS, MPN | Y (2020/2021) | AML (maintenance) | |
Decitabine | 5-Aza-2′-deoxycytidine | 1–3 (completed), 1/2 (ongoing) 3 (not yet recruiting) | AML, MDS, MPN | Y (2006/2012) | MDS (FDA) AML (EMA) | |
Oral decitabine/ cedazuridine | ASTX727 | 1–3 (completed) 1/2 (ongoing) | AML, MDS | Y (2020/2021) | MDS (FDA) AML (EMA) | |
IDH-Inhibitors | Enasidenib (IDH2) | AG-221 | 1–3 (completed)1-2 (ongoing) | AML (IDH2-mutated) [1–3] MDS (IDH2-mutated) | Y (2017) | IDH2-mutated R/R AML (FDA) |
Ivosidenib (IDH1) | AG-120 | 1–3 (completed) 1–3 (ongoing) | AML (IDH1-mutated) MDS (IDH1-mutated) | Y (2018 and 2022/2023) | IDH1-mutated R/R AML (FDA); IDH1- AML (FDA/EMA) | |
Olutasidenib (IDH1) | FT-2102 | 1/2 (completed) 1–2 (ongoing) | AML (IDH1-mutated), Myeloid Neoplasm (IDH1-mutated) | N (2022) | IDH1-mutated R/R AML (FDA) | |
LSD1-Inhibitors | Bomedemstat | MK-3543, IMG-7289 | 1/2 (completed) 1–3 (ongoing) | Myeloid Neoplasms, ET, MF, PV [1/2], ET [3] | N | N. A. |
Menin-Inhibitors | Bleximenib | JNJ-7527661 | 1–3 (ongoing) | AL (KMT2A-r, NPM1-mutated, NUP98-r) [1–3] AML, HOX-high AML [1,2] | N | N. A. |
Enzomenib | DSP-5336 | 1/2 (ongoing) | AL (KMT2A-r, NPM1-mutated) | N | N. A. | |
Revumenib | SNDX-5613 (close [preclinical] homolog: VTP-50469) | 1/2 (completed) 1–3 (ongoing) | AML (KMT2A-r, NPM1-mutated) [1–3] AML, HOX-high AML [1,2] | Y (2024) | R/R KMT2A-r AL (FDA) | |
Ziftomenib | KO-539 | 1 (completed) 1–2 (ongoing) | AML (KMT2A-r, NPM1-mutated, NUP98-r) [1–3] AML, HOX-high AML [1,2] | N | N. A. | |
PRMT5-Inhibitors | Pemrametostat | GSK3326595, EPZ015938 | 1/2 (completed) | AML, MDS | N | N. A. |
Onametostat | JNJ-64619178 | 1/2 (completed) | MDS | N | N. A. |
