Eprenetapopt (APR-246) and Azacitidine in TP53-Mutant Myelodysplastic Syndromes.

ABSTRACT:

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: NCT03072043).

Results

Fifty-five patients (40 MDS, 11 AML, and four MDS/myeloproliferative neoplasms) with at least one TP53 mutation were treated. The overall response rate was 71% with 44

SUBMITTER: Sallman DA 

PROVIDER: S-EPMC8099410 | biostudies-literature | 2021 May

REPOSITORIES: biostudies-literature