Skip to main content

Table 2 Targeted Therapies in MPN

From: Molecular pathogenesis of the myeloproliferative neoplasms

  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)
  1. aEuropean Medicines Agency approval only. FDA approval pending
  2. This table summarises the diversity of targeted therapies approved or undergoing clinical trials investigation in MPN either as single agent or combination therapies separated on the basis of sections of this review. The list is not exhaustive and an example of an active or recently completed clinical trial listed on clinicaltrials.gov platform has been provided for each drug. Other trials may be available