Table 2.

Novel agents being investigated in MF

Agent nameMechanism of actionNCT identifier; references
Novel single-agent investigations*(or both as single agent and combined with ruxolitinib) in MF   
 ABT-888* PARP inhibitor 61  
 Alisertib Aurora kinase inhibitor NCT02530619; 62  
 Durvalumab Anti-PDL1 antibody NCT02871323 
 Glasdegib* Hedgehog pathway inhibitor NCT02226172; 63  
 Imetelstat Telomerase inhibitor NCT01731951; NCT0242608; 64  
 LCL 161 SMAC inhibitor NCT02098161; 65  
 OGX-427 Heat shock protein 66  
 Mirabegron Sympathicomimetic agonist NCT02311569; 67  
 Nivolumab Anti-PD1 antibody NCT02421354 
 Pembrolizumab* Anti-PD1 antibody NCT03065400 
 PRM151* Antifibrosing agent NCT01981850; 68  
 Pracinostat HDAC inhibitor NCT01200498; NCT02267278; 69  
 SL-401 Recombinant IL-3 with diphtheria toxin NCT02268253; 70  
 Sotatercept* ACTRIIa NCT01712308; 71  
 Umbralisib* PI3K inhibitor NCT01730248; 72  
Novel combinational investigations with ruxolitinib in MF   
 Vismodegib Hedgehog pathway inhibitor NCT02593760; 73  
 PIM447, LEE001, and JAK1/2 Pan-PIM inhibitor, CDK4/6 inhibitor, JAK1/2 inhibitor NCT02370706; 74  
 Sonidegib Hedgehog pathway inhibitor NCT01787552; 75  
JAK2 inhibitor investigations (published phase 3 investigations and above only)   
 Fedratinib JAK2 inhibitor NCT00724334; 37,38  
 Momelotinib JAK1/2 inhibitor NCT01969838; NCT02124746; NCT03441113; 42  
 Pacritinib JAK2, FLT3 inhibitor NCT03165734; 41,76  
Agent nameMechanism of actionNCT identifier; references
Novel single-agent investigations*(or both as single agent and combined with ruxolitinib) in MF   
 ABT-888* PARP inhibitor 61  
 Alisertib Aurora kinase inhibitor NCT02530619; 62  
 Durvalumab Anti-PDL1 antibody NCT02871323 
 Glasdegib* Hedgehog pathway inhibitor NCT02226172; 63  
 Imetelstat Telomerase inhibitor NCT01731951; NCT0242608; 64  
 LCL 161 SMAC inhibitor NCT02098161; 65  
 OGX-427 Heat shock protein 66  
 Mirabegron Sympathicomimetic agonist NCT02311569; 67  
 Nivolumab Anti-PD1 antibody NCT02421354 
 Pembrolizumab* Anti-PD1 antibody NCT03065400 
 PRM151* Antifibrosing agent NCT01981850; 68  
 Pracinostat HDAC inhibitor NCT01200498; NCT02267278; 69  
 SL-401 Recombinant IL-3 with diphtheria toxin NCT02268253; 70  
 Sotatercept* ACTRIIa NCT01712308; 71  
 Umbralisib* PI3K inhibitor NCT01730248; 72  
Novel combinational investigations with ruxolitinib in MF   
 Vismodegib Hedgehog pathway inhibitor NCT02593760; 73  
 PIM447, LEE001, and JAK1/2 Pan-PIM inhibitor, CDK4/6 inhibitor, JAK1/2 inhibitor NCT02370706; 74  
 Sonidegib Hedgehog pathway inhibitor NCT01787552; 75  
JAK2 inhibitor investigations (published phase 3 investigations and above only)   
 Fedratinib JAK2 inhibitor NCT00724334; 37,38  
 Momelotinib JAK1/2 inhibitor NCT01969838; NCT02124746; NCT03441113; 42  
 Pacritinib JAK2, FLT3 inhibitor NCT03165734; 41,76  

ACTRIIa, activin A receptor type IIA; CDK, cyclin-dependent kinase; FLT3, FMS-like tyrosine kinase 3; IL-3, interleukin-3; PARP, polyadenosine 5′-diphosphate-ribose polymerase; PD1, programmed T-cell death 1; PDL1, programmed death-ligand 1; PI3K, phosphatidyl 3-kinase; PIM, proto-oncogene serine/threonine-protein kinase.

*

Designates that this is being investigated both alone and in combination with ruxolitinib.

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