Novel investigational agents
| Drug . | Mechanism of action . | Patient population . | Trial no. . |
|---|---|---|---|
| Luspatercept | ActRII ligand trap | MF with Hgb <10 g/dL | NCT03194542 |
| Sotatercept | ActRII ligand trap | MF with Hgb <10 g/dL | NCT01712308 |
| Selumetinib | MEK inhibitor | High-risk myeloid neoplasms | NCT03326310 |
| Imetelstat | Telomerase inhibitor | MF ET with at least 1 prior therapy MF with previous JAKi exposure | NCT01731951 NCT01243073 NCT02426086 |
| Bomedemstat | LSD-1 inhibitor | MF with previous JAKi exposure ET or PV with at least 1 prior therapy ET previously treated with hydroxyurea ET with at least 1 prior therapy | NCT03136185 NCT04262141 NCT04081220 NCT04254978 |
| KRT-232 (in combination with decitabine or low-dose cytarabine) | MDM2 inhibitor | TP53 wild-type BP MPNs | NCT04113616 |
| Venetoclax (in combination with decitabine) | BCL2 inhibitor | TP53-mutated BP MPNs | NCT03844815 |
| Drug . | Mechanism of action . | Patient population . | Trial no. . |
|---|---|---|---|
| Luspatercept | ActRII ligand trap | MF with Hgb <10 g/dL | NCT03194542 |
| Sotatercept | ActRII ligand trap | MF with Hgb <10 g/dL | NCT01712308 |
| Selumetinib | MEK inhibitor | High-risk myeloid neoplasms | NCT03326310 |
| Imetelstat | Telomerase inhibitor | MF ET with at least 1 prior therapy MF with previous JAKi exposure | NCT01731951 NCT01243073 NCT02426086 |
| Bomedemstat | LSD-1 inhibitor | MF with previous JAKi exposure ET or PV with at least 1 prior therapy ET previously treated with hydroxyurea ET with at least 1 prior therapy | NCT03136185 NCT04262141 NCT04081220 NCT04254978 |
| KRT-232 (in combination with decitabine or low-dose cytarabine) | MDM2 inhibitor | TP53 wild-type BP MPNs | NCT04113616 |
| Venetoclax (in combination with decitabine) | BCL2 inhibitor | TP53-mutated BP MPNs | NCT03844815 |
See Tables 1 and 3 for expansion of abbreviations.