eprenetapopt + azacitidine

Eprenetapopt (APR-246) is a first-in-class small molecule developed by Aprea Therapeutics that restores wild-type p53 functions in TP53-mutant cells by restoring the protein's conformation and function. This combination therapy pairs eprenetapopt with azacitidine, a hypomethylating agent, for the treatment of TP53-mutant myelodysplastic syndromes (MDS). The combination was evaluated in a pivotal Phase 3 trial (ASPIRE) but failed to meet its primary endpoint of complete response rate compared to azacitidine alone. Despite this, the combination has previously received Breakthrough Therapy, Orphan Drug, and Fast Track designations from the FDA for MDS.

Brand names

Vidaza

Other names

APR-246 + azacitidineeprenetapopt + azacitidine