Aprea Therapeutics (NASDAQ:APRE) received FDA breakthrough designation for APR-246, in combination with azacytidine, for the treatment of myelodysplastic syndromes (MDS) with a susceptible tumor suppressor protein p53 (TP53) mutation.
MDS is a type of cancer caused by the bone marrow’s inability to produce a sufficient amount of blood cells. A TP53 mutation contributes to progression of the disease to acute myeloid leukemia (AML), which occurs in some 30% to 40% of MDS patients. APR-246 is designed to reactivate mutant TP53.
Aprea is conducting an ongoing Phase 3 clinical trial of APR-246 and azacitidine for frontline treatment of TP53 mutant MDS.
“Outcomes for MDS patients with a TP53 mutation are poor and there are no current therapeutic options specifically for these patients,” Christian Schade, Aprea’s CEO, said in a statement.
APR-246 has received FDA orphan drug and fast track designations for MDS; and EMA orphan drug designation for MDS, AML and ovarian cancer.
