Emerging novel therapies in R/R AML
| Phase . | Regimen . | Mechanism of action . | Number of patients . | Median age (range) . | Response . | Reference . |
|---|---|---|---|---|---|---|
| 1 | SNDX-5613 | Menin inhibitor | 68 (56 R/R AML) | 51 (19-79) among adults | ORR: 53% (32/60) CR/CRi: 38% (23/60) ORR in KMT2A: 59% (27/46) ORR in NPM1: 36% (5/14) | Wang et al26 |
| 1 | HDM201 | MDM2 inhibitor | 208 (91 R/R AML) | ∼70 (23-85) | CR/CRi: 13.2% (12/91) CR/CRi in 45 mg: 22.2% (6/28) | Klossowski et al28 |
| 1/2 | HM43239 | FLT3/SYK inhibitor | 28 | 60 (35-83) | CR/CRi in 80 mg: 26.3% (5/19) CR/CRi in FLT3 80 mg: 37.5% (3/8) | Konopleva et al31 |
| 1b/2 | IMGN632 + AZA + Ven | CD123 antibody-drug conjugate | 35 (29 efficacy evaluable R/R AML) | 69 (range not available) | ORR: 55% (16/29) CR/CRi: 31% (9/29) | Daver et al35 |
| 1/2 | Magrolimab + AZA + Ven | CD47 inhibitor | 74 (29 R/R, 45 newly diagnosed) | Not available | ORR in R/R Ven-naïve: 75% ORR in R/R Ven prior: 12% | Kuruvilla et al38 |
| 1b/2 | APR-246 | p53 reactivator | 55 (11 R/R oligoblastic AML) | 66 (34-85) | ORR: 64% (n = 7) CR: 36% (n = 4) Median OS 10.8 months | Daver et al39 |
| Phase . | Regimen . | Mechanism of action . | Number of patients . | Median age (range) . | Response . | Reference . |
|---|---|---|---|---|---|---|
| 1 | SNDX-5613 | Menin inhibitor | 68 (56 R/R AML) | 51 (19-79) among adults | ORR: 53% (32/60) CR/CRi: 38% (23/60) ORR in KMT2A: 59% (27/46) ORR in NPM1: 36% (5/14) | Wang et al26 |
| 1 | HDM201 | MDM2 inhibitor | 208 (91 R/R AML) | ∼70 (23-85) | CR/CRi: 13.2% (12/91) CR/CRi in 45 mg: 22.2% (6/28) | Klossowski et al28 |
| 1/2 | HM43239 | FLT3/SYK inhibitor | 28 | 60 (35-83) | CR/CRi in 80 mg: 26.3% (5/19) CR/CRi in FLT3 80 mg: 37.5% (3/8) | Konopleva et al31 |
| 1b/2 | IMGN632 + AZA + Ven | CD123 antibody-drug conjugate | 35 (29 efficacy evaluable R/R AML) | 69 (range not available) | ORR: 55% (16/29) CR/CRi: 31% (9/29) | Daver et al35 |
| 1/2 | Magrolimab + AZA + Ven | CD47 inhibitor | 74 (29 R/R, 45 newly diagnosed) | Not available | ORR in R/R Ven-naïve: 75% ORR in R/R Ven prior: 12% | Kuruvilla et al38 |
| 1b/2 | APR-246 | p53 reactivator | 55 (11 R/R oligoblastic AML) | 66 (34-85) | ORR: 64% (n = 7) CR: 36% (n = 4) Median OS 10.8 months | Daver et al39 |