Aprea's MDS candidate leads to 95% ORR in Phase Ib/II

Aprea Therapeutics AB (Stockholm, Sweden) reported data from 20 evaluable patients with myelodysplastic syndrome (MDS) or acute myelogenous leukemia (AML) in a Phase Ib/II trial showing that APR-246 plus Vidaza azacitidine led to an overall response rate (ORR) of 95%, including a complete remission rate of 70%. Data were presented at the American Society of Hematology (ASH) meeting in San Diego. The H. Lee Moffitt Cancer Center and Research Institute sponsored the trial.

The open-label, dose-escalation, U.S. trial is enrolling about 49 patients with p53 (TP53) mutant MDS. APR-246 is a small molecule quinuclidinone that promotes correct folding of p53...