[HTML][HTML] Eprenetapopt (APR-246) and azacitidine in TP53-mutant myelodysplastic syndromes

DA Sallman, AE DeZern, G Garcia-Manero… - Journal of clinical …, 2021 - ncbi.nlm.nih.gov
DA Sallman, AE DeZern, G Garcia-Manero, DP Steensma, GJ Roboz, MA Sekeres
Journal of clinical oncology, 2021ncbi.nlm.nih.gov
PURPOSE Approximately 20% of patients with TP53-mutant myelodysplastic syndromes
(MDS) achieve complete remission (CR) with hypomethylating agents. Eprenetapopt (APR-
246) is a novel, first-in-class, small molecule that restores wild-type p53 functions in TP53-
mutant cells. METHODS This was a phase Ib/II study to determine the safety, recommended
phase II dose, and efficacy of eprenetapopt administered in combination with azacitidine in
patients with TP53-mutant MDS or acute myeloid leukemia (AML) with 20%-30% marrow …
PURPOSE
Approximately 20% of patients with TP53-mutant myelodysplastic syndromes (MDS) achieve complete remission (CR) with hypomethylating agents. Eprenetapopt (APR-246) is a novel, first-in-class, small molecule that restores wild-type p53 functions in TP53-mutant cells.
METHODS
This was a phase Ib/II study to determine the safety, recommended phase II dose, and efficacy of eprenetapopt administered in combination with azacitidine in patients with TP53-mutant MDS or acute myeloid leukemia (AML) with 20%-30% marrow blasts (ClinicalTrials. gov identifier:
ncbi.nlm.nih.gov