Table 2.
Selected clinical trials investigating anti-CD123, immune checkpoint inhibitors (ICIs) and tumor vaccination in MPNs.
| Drug | Target (Mechanism) |
Condition(s) | Trial (Phase) |
Reference | Results/Comments |
|---|---|---|---|---|---|
|
Tagraxofusp
(SL-401) |
CD123 (MoAb) |
MF (or CMML) |
NCT02268253 (Phase I/II) |
Pemmaraju et al., 2019 [188] |
Still recruiting; SVR = 53%, SI = 45% |
| Nivolumab | PD-1 (MoAb) |
MF |
NCT02421354 (Phase II) |
- | Terminated early for lack of efficacy |
| Pembrolizumab | PD-1 (MoAb) |
PV, MF |
NCT03065400 (Phase II) |
Cimen Bozkus et al., 2019 [131] | Ongoing; ↑reactivity to CALR mutant epitopes in vivo and in vitro |
| Durvalumab | PD-L1 (MoAb) |
MF |
NCT02871323 (Phase I) |
Withdrawn before patients’ enrolment | |
| Ipilimumab * | CTLA-4 (MoAb) |
MPNs (among other conditions) |
NCT01822509 (Phase I/Ib) |
Ongoing; aims: assessment of AEs and best dose of ipilimumab or nivolumab | |
| CALRLong36 peptide (exon 9 mut) vaccine | Mutated CALR (vaccine) | CALR+ PMF and ET |
NCT03566446 (Phase I) |
Aims: assessment of AEs and T cell cytokine release against the target antigen | |
|
PD-L1Long (19–27)
ArgLong2 (169–206) vaccine |
PD-L1 and ARG1 (vaccine) |
ET, PV |
NCT04051307 (Phase I/II) |
Expected effects: ↑specific T cell responses, ↑killing of mutant cells, ↓ARG1, ↓PD-L1 |
MF: primary or post-essential thrombocythemia/polycythemia vera myelofibrosis; MoAb: monoclonal antibody; CTLA-4: cytotoxic T lymphocyte antigen-4; AEs: adverse events; SVR: spleen volume reduction; SI: symptom improvement; CMML: chronic myelomonocytic leukemia; ↓: decrease; ↑: increase; %: percentage of patients. * Ipilimumab or nivolumab in patients with various hematological neoplasms (including MPNs) relapsed after allogeneic hematopoietic stem cell transplantation.