Emerging therapy in LR-MDS
| Agent . | Mechanism of action . | Phase . | Population . | Identifier . | Ref. . |
|---|---|---|---|---|---|
| Ivosidenib | IDH1 inhibitor | 2 | Treatment-naive HR-MDS R/R (HMA) HR-MDS R/R (ESA) LR-MDS with anemia All with IDH1m | NCT03503409 | 20 |
| Enasidenib | IDH2 inhibitor | 2 | Treatment-naive HR-MDS R/R (HMA) HR-MDS R/R (ESA) LR-MDS with anemia All with IDH2m | NCT03744390 | 21 |
| 2, with AZA | MDS, excess blats, AML, CMML with IDH2m | NCT03383575 | |||
| Olutasidenib (FT-2102) | IDH1 inhibitor | 2, with/without AZA/L-DAC | SMD and AML with IDH1m | NCT02719574 | |
| H3B-8800 | Splicing modulator | 1 | SMD, AML, CMML | NCT02841540 | 24 |
| Roxadustat | HIF inhibitor | 3 | LR-MDS with anemia, low transfusion burden | NCT03263091 | 29 |
| 2/3 | LR-MDS with anemia | NCT03263091 | |||
| Imetelstat | Telomerase inhibitor | 2/3 | R/R (ESA) LR-MDS | NCT02598661 | 25 |
| KER-050 | TGF-β inhibitor | 2 | R/R (ESA) LR-MDS | NCT04419649 | |
| Canakinumab | Il-1β inhibitor | 1/2, with darbepoietin | R/R (ESA) LR-MDS | NCT04798339 | |
| 2 | R/R (ESA) LR-MDS | NCT05237713 | |||
| 2 | R/R (ESA/HMA) LR-MDS/CMML | NCT04239157 | |||
| Emavusertib (CA-4948) | IRAK4 inhibitor | 2 | Treatment-naive and R/R (ESA) LR-MDS | NCT05178342 | |
| BMS-986253 | IL-8 inhibitor | 1/2, with/without DEC/cedazuridine | R/R (HMA) HR-MDS R/R (ESA/LEN/Luspa) LR-MDS | NCT05148234 | |
| SX-682 | CXCR1 and CXCR2 inhibitor | 1 | R/R (ESA/LEN) LR-MDS | NCT04245397 | |
| Tomaralimab | TLR2 inhibitor | 1/2 | R/R (ESA/LEN) LR-MDS | NCT02363491 |
| Agent . | Mechanism of action . | Phase . | Population . | Identifier . | Ref. . |
|---|---|---|---|---|---|
| Ivosidenib | IDH1 inhibitor | 2 | Treatment-naive HR-MDS R/R (HMA) HR-MDS R/R (ESA) LR-MDS with anemia All with IDH1m | NCT03503409 | 20 |
| Enasidenib | IDH2 inhibitor | 2 | Treatment-naive HR-MDS R/R (HMA) HR-MDS R/R (ESA) LR-MDS with anemia All with IDH2m | NCT03744390 | 21 |
| 2, with AZA | MDS, excess blats, AML, CMML with IDH2m | NCT03383575 | |||
| Olutasidenib (FT-2102) | IDH1 inhibitor | 2, with/without AZA/L-DAC | SMD and AML with IDH1m | NCT02719574 | |
| H3B-8800 | Splicing modulator | 1 | SMD, AML, CMML | NCT02841540 | 24 |
| Roxadustat | HIF inhibitor | 3 | LR-MDS with anemia, low transfusion burden | NCT03263091 | 29 |
| 2/3 | LR-MDS with anemia | NCT03263091 | |||
| Imetelstat | Telomerase inhibitor | 2/3 | R/R (ESA) LR-MDS | NCT02598661 | 25 |
| KER-050 | TGF-β inhibitor | 2 | R/R (ESA) LR-MDS | NCT04419649 | |
| Canakinumab | Il-1β inhibitor | 1/2, with darbepoietin | R/R (ESA) LR-MDS | NCT04798339 | |
| 2 | R/R (ESA) LR-MDS | NCT05237713 | |||
| 2 | R/R (ESA/HMA) LR-MDS/CMML | NCT04239157 | |||
| Emavusertib (CA-4948) | IRAK4 inhibitor | 2 | Treatment-naive and R/R (ESA) LR-MDS | NCT05178342 | |
| BMS-986253 | IL-8 inhibitor | 1/2, with/without DEC/cedazuridine | R/R (HMA) HR-MDS R/R (ESA/LEN/Luspa) LR-MDS | NCT05148234 | |
| SX-682 | CXCR1 and CXCR2 inhibitor | 1 | R/R (ESA/LEN) LR-MDS | NCT04245397 | |
| Tomaralimab | TLR2 inhibitor | 1/2 | R/R (ESA/LEN) LR-MDS | NCT02363491 |
AML, acute myelogenous leukemia; AZA, azacitidine; CMML, chronic myelomonocytic leukemia; DEC, decitabine; HIF, hypoxia-inducible factor; L-DAC, low-dose aracytine; LEN, lenalidomide; MDS, myelodysplastic syndrome; R/R, relapse/refractory.