Aprea Therapeutics receives FDA breakthrough therapy designation for APR-246 in combination with azacitidine for the treatment of myelodysplastic syndromes with a TP53 mutation

Aprea Therapeutics

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.

Read Aprea Therapeutics press release

Michael Wonder

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Michael Wonder