APR-548 + azacitidine

APR-548 + azacitidine is a combination therapy under investigation for the treatment of TP53-mutant myelodysplastic syndromes (MDS). APR-548 is a next-generation small molecule that acts as a p53 reactivator, designed to restore the function of mutant p53 protein commonly found in various cancers. It has demonstrated high oral bioavailability and enhanced potency compared to earlier agents like eprenetapopt. Azacitidine is an approved demethylating agent that works by promoting normal blood cell production and killing abnormal cells in the bone marrow. The combination aims to target TP53-mutant MDS by both reactivating dysfunctional tumor suppressor pathways (via APR-548) and modifying epigenetic regulation (via azacitidine). The primary developer of APR-548 is Aprea Therapeutics[1][2][3][6].