BRIEF—Aprea Therapeutics gains FDA Breakthrough Therapy designation

Aprea Therapeutics, a Karolinska Development portfolio company, has been granted Breakthrough Therapy designation for its APR-245 in combination with azacitidine for the treatment of myelodyplastic syndrome (MDS) with a TP53 mutation.

APR-246 is a small molecular drug candidate that binds, refolds and stabilizes mutant p53, occurring in approximately 50% of all human tumors.

A pivotal Phase III clinical trial of APR-246 and azacitidine for frontline treatment of TP53 mutant MDS is ongoing.

APR-246 has previously received Orphan Drug and Fast Track designations from the Food and Drug Administration for MDS, and Orphan Drug designation from the European Medicines Agency for MDS, acute myeloid leukemia (AML) and ovarian cancer.

“This is yet another success for Aprea Therapeutics, and we look forward to following the continued development of its potentially ground-breaking cancer therapy,” says Viktor Drvota, chief executive, Karolinska Development AB, in response to the announcement.