On January 30, 2020 Karolinska Development (Nasdaq Stockholm: KDEV) reported that its portfolio company Aprea Therapeutics has been granted Breakthrough Therapy Designation for APR-246 in combination with azacitidine for the treatment of myelodysplastic syndrome (MDS) with a TP53 mutation (Press release, Aprea, JAN 30, 2020, https://www.karolinskadevelopment.com/en/press-releases?page=/en/pressreleases/karolinska-development%2527s-portfolio-company-aprea-therapeutics-receives-fda-breakthrough-therapy-designation-1769167 [SID1234553684]). A Breakthrough Therapy Designation facilitates expedited development and regulatory review of a drug candidate.
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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 3 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 FDA for MDS, and Orphan Drug designation from the EMA 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, CEO, Karolinska Development AB, in response to the announcement.
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.
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.