Table 2 Targeted Therapies in MPN
From: Molecular pathogenesis of the myeloproliferative neoplasms
| Â | Drug class | Drug | Approved/trial |
|---|---|---|---|
JAK-STAT signalling | JAK inhibition | Ruxolitinib | Approved |
Fedratinib | Approved | ||
Momelotinib | Phase III trial (NCT04173494) | ||
Pacritinib | Phase III trials (NCT03165734) | ||
Non-JAK/STAT intracellular signalling | PI3K inhibition | Parsaclisib | Phase III (NCT04551066) |
PIM inhibition | PIM447 | Phase I (NCT02370706) | |
Targeted inhibition of mutated proteins | IDH2 inhibition | Enasidenib | Phase II (NCT04281498) |
Cell of origin | Interferon-α | Peginterferon-alpha-2A | Approved |
Ropeginterferon-alpha-2B | Approveda | ||
Predisposing factors | Telomerase inhibition | Imetelstat | Phase III (NCT04576156) |
Epigenetic dysregulation | Hypomethylating agents | Azacitidine | Phase II (NCT01787487) |
Decitabine | Phase II (NCT0428187) | ||
Histone deacetylase (HDAc) inhibitor | Panobinostat | Phase I/II (NCT01693601) | |
Givinostat | Phase II (NCT01761968) | ||
BET inhibitors | CPI-0610 | Phase I/II (NCT02158858) | |
LSD1 inhibitors | IMG-7289 (bomedemstat) | Phase II (NCT03136185) | |
Other | IMiDs | Thalidomide | Phase II (NCT03069326) |
BCL2/BCL-Xl inhibitors | Navitoclax | Phase II (NCT03222609) | |
MDM2 inhibition | KRT-232 | Phase II (NCT03662126) | |
Aurora kinase inhibition | Alisertib | Phase N/A (NCT02530619) | |
PD-1 inhibition | Pembrolizumab | Phase II (NCT03065400) | |
TGF-beta signalling interference | Luspatercept | Phase II (NCT04717414) | |
Sotatercept | Phase II (NCT01712308) | ||
Anti-CD123 | Tagraxofusp | Phase II (NCT02268253) |