APR-548

APR-548 is an orally bioavailable, next-generation small molecule prodrug designed to reactivate mutant p53 protein. It is an analog of eprenetapopt (APR-246) and has demonstrated higher oral bioavailability and enhanced potency compared to eprenetapopt in preclinical models. APR-548 works by restoring the wild-type conformation and function of mutant p53, thereby inducing programmed cell death (apoptosis) in cancer cells with TP53 mutations. The drug was developed primarily for the treatment of TP53-mutant myelodysplastic syndromes (MDS), but its mechanism suggests potential utility across a range of cancers characterized by p53 mutations[1][3][5][8].