Aprea Therapeutics ‘Breakthrough’ for Azacitidine

FDA has granted Aprea Therapeutics a breakthrough therapy designation for APR-246 in combination with azacitidine for treating myelodysplastic syndromes (MDS) with a susceptible TP53 mutation. MDS is a hematopoietic stem cell malignancy where bone marrow fails to produce healthy blood cells, the company says. About 30-40% of MDS patients develop acute myeloid leukemia (AML), it says, adding that “mutation of the p53 tumor suppressor protein is thought to directly contribute to disease progression and a poor overall prognosis.”

Aprea describes APR-246 as a small molecule that has demonstrated reactivation of mutant and inactivated p53 proteins. Pre-clinical anti-tumor activity has been observed with APR-246 in a several solid and hematological cancers, including MDS, AML, and ovarian cancer, among others, it says. The therapy is currently undergoing a Phase 3 clinical study.