Table 2.
Novel Agents for Myelofibrosis in Clinical Development
| Class | Agent | Target | Phase | NCT identifier |
|---|---|---|---|---|
| JAK inhibitors | Itacitinib, alone or with ruxolitinib (+RUX) | JAK1 | 2 | NCT03144687 |
| Pacritinib | JAK2/FLT3 | 2 | NCT03165734 | |
| NS-018 | JAK2/Src | 2 | NCT01423851 | |
| Epigenetic Agents | Pracinostat (+RUX) | HDAC | 2 | NCT02267278 |
| Panobinostat | HDAC | 1/2 | NCT01693601 | |
| IMG-7289 | LSD-1 | 1 | NCT03136185 | |
| Azacitidine (+RUX) | DNA methylation | 2 | NCT01787487 | |
| SGI-110 | DNA methylation | 2 | NCT03075826 | |
| PI3K/AKT/mTOR Pathway Inhibitors |
INCB050465 (+RUX) | PI3K | 2 | NCT02718300 |
| Buparlisib (+RUX) | PI3K | 1 | NCT01730248 | |
| TGR-1202 (+RUX) | PI3Kδ | 1 | NCT02493530 | |
| Hedgehog Pathway Inhibitors |
Vismodegib (+ RUX) | SMO | 1/2 | NCT02593760 |
| Sonidegib (+ RUX) | SMO | 1/2 | NCT01787552 | |
| Glasdegib | SMO | 2 | NCT02226172 | |
| Other Small Molecule Inhibitors |
CPI-0610 | BET | 1 | NCT02158858 |
| PIM447 (+ RUX) | pan-PIM kinases | 1b | NCT02370706 | |
| Ribocicilib (+ RUX) | CDK4/6 | 1b | NCT02370706 | |
| Alisertib | Aurora kinase A | 1 | NCT02530619 | |
| Selumetinib | MEK kinase | 1 | *pending | |
| Checkpoint Inhibitors |
Durvalumab | PD-L1 | 1 | NCT02871323 |
| Pembrolizumab | PD-1 | 2 | NCT03065400 | |
| Nivolumab | PD-1 | 2 | NCT02421354 | |
| Nivolumab | PD-1 | 1/1b | NCT01822509 | |
| Ipilimumab | CTLA4 | 1/1b | NCT01822509 | |
| Other Agents | Imetelstat | Telomerase | 2 | NCT02426086 |
| PRM-151 | Pentraxin 2 | 2 | NCT01981850 | |
| Sotatercept | TGFβ | 2 | NCT01712308 | |
| SL-401 | IL3 receptor (CD123) | 1/2 | NCT02268253 | |
| P1101 | Peg-Interferon α | 2 | NCT02370329 | |
| LCL-161 | SMAC mimetic | 2 | NCT02098161 |
*
Clinical trials.gov listing is pending at time of manuscript submission