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Aprea Therapeutics receives FDA Breakthrough Therapy Designation


Aprea Therapeutics has announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for APR-246 in combination with azacitidine for the treatment of myelodysplastic syndromes (MDS) with a susceptible TP53 mutation.

MDS represents a spectrum of hematopoietic stem cell malignancies in which bone marrow fails to produce sufficient numbers of healthy blood cells. Approximately 30-40% of MDS patients progress to acute myeloid leukemia (AML) and mutation of the p53 tumor suppressor protein is thought to directly contribute to disease progression and a poor overall prognosis.

“Breakthrough Therapy Designation further supports our development program for APR-246 in combination with azacitidine in MDS patients with a TP53 mutation,” said Christian S. Schade, Chief Executive Officer of Aprea. “Outcomes for MDS patients with a TP53 mutation are poor and there are no current therapeutic options specifically for these patients. We look forward to continued interaction with FDA regarding our ongoing Phase 3 clinical study and our clinical development program to advance APR-246.”

The FDA’s Breakthrough Therapy Designation is intended to expedite the development and review of a drug candidate that is planned to treat a serious or life-threatening disease or condition when preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapies on one or more clinically significant endpoints.

Image caption: p53 prevents cancer formation and acts as a guardian of the genome. Mutations in the p53 gene contribute to about half of the cases of human cancer.