616. Acute Myeloid Leukemias: Investigational Therapies, Excluding Transplantation and Cellular Immunotherapies: Targeted Therapies and Novel Therapies


Pau Montesinos, Christian Recher, Susana Vives, et al.

The results of this study significantly demonstrate the clinical benefit of ivosidenib (IVO) + azacitidine (AZA) compared to placebo + AZA in a difficult-to-treat AML population: the combination improved event-free survival, overall survival, and clinical response (CR, CR + CRh, ORR) in these patients with IC-ineligible, newly diagnosed mIDH1-AML. The favorable safety profile of IVO + AZA was consistent with previous studies.



Jorge E. Cortes, Jordi Esteve, Ashish Bajel, et al.

Olutasidenib plus azacitidine induced durable CR/CRh in a subgroup of high-risk patients with mIDH1-AML. The combination was well tolerated. In all cohorts, some of those treated achieved transfusion independence. Further analyzes on safety and efficacy will be presented at the oral session.



Eytan M. Stein, Ibrahim Aldoss, John F. DiPersio, et al.

SNDX-5613 demonstrated an acceptable safety profile. Promising antileukemic activity has been observed in patients with heavily pretreated R / R MLLr and mNPM1 acute leukemia. The pre-established criteria for RP2D were met with two dose levels in each arm.



Eunice S. Wang, Pau Montesinos, Mark D. Minden, et al.

The combination of gilteritinib (GIL) + azacitidine (AZA) achieved significantly higher CRc rates in the absence of new safety signals, but similar OS compared to AZA alone. Patients with ECOG PS 0-1 and high FLT3-ITD allele ratio appeared to have greater benefit with the combination. Strange: the Ctrough values in patients with ND FLT3mut + AML who were not suitable for IIC were 2 times higher than in patients with R/R FLT3mut + AML. The study results confirm the safety, tolerability and activity of the combination compared to AZA.


701 ASH denotes this abstract as clinically relevant 

Curtis Lachowiez, Courtney D. DiNardo, Koichi Takahashi, et al.

Induction and consolidation with fludarabine, high-dose cytarabine (Ara-C0), idarubicin and granulocyte colony-stimulating factor (G-CSF) plus venetoclax (FLAG-IDA + VEN) leads to high MRD-negative CRc rates in ND-AML with an expected safety profile. After one year there was a sustained response with favorable results compared to historical cohorts of intensive therapy. With TP53 mutations there were inferior outcomes in comparison to wild-type TP53.



Naval Daver,  Kyoo Hyung Lee, Chul Won Jung, et al.

HM43239 is a preclinically effective FLT3 and SYK inhibitor. In the present study, the substance showed a favorable safety profile with only slight undesirable side effects and no redefining dose-limiting toxicity (DLT). At 80 mg, HM43239 shows clinical activity in both FLT3m and FLT3wt-AML. Updated data on response, safety and PK / PD will be presented in the oral session.