30 January 2020 - Aprea Therapeutics today announced that the U.S. FDA has granted breakthrough therapy designation for APR-246 in combination with azacitidine for the treatment of myelodysplastic syndromes with a susceptible TP53 mutation.
APR-246 is a small molecule that has demonstrated reactivation of mutant and inactivated p53 protein – by restoring wild-type p53 conformation and function – and thereby induce programmed cell death in human cancer cells.
A pivotal Phase 3 clinical trial of APR-246 and azacitidine for frontline treatment of TP53 mutant myelodysplastic syndrome (MDS) is ongoing. APR-246 has received orphan drug and fast track designations from the FDA for MDS, and orphan drug designation from the EMA for MDS, AML and ovarian cancer.