Aprea Therapeutics (NASDAQ:APRE) announced today that the Food and Drug Administration has given its breakthrough therapy designation to the company's lead drug candidate, APR-246. It is being tested in a cocktail with Azacitidine (a type of chemotherapy) to treat myelodysplastic syndrome (MDS), a condition that often progresses to leukemia.
APR-246 is designed to help patients who have a genetic mutation in tp53, a key cancer-suppressing gene. This gene provides cells with the instructions for producing a protein (P53) that keeps cell division in check, thus suppressing cancers in the human body. When the gene mutates and stops working, cancer proliferates. P53 is the most common mutated gene in human cancers, occurring in approximately 50% of all tumors.
Why is APR-246 a breakthrough therapy?
The FDA reserves its breakthrough therapy designation for drugs that treat serious conditions when early clinical trials indicate they are superior to existing therapies. Treatments that receive it are given an automatic fast-track through the regulatory review process.
"Outcomes for MDS patients with a TP53 mutation are poor and there are no current therapeutic options specifically for these patients," said Aprea CEO Christian Schade. "We look forward to continued interaction with FDA regarding our ongoing Phase 3 clinical study and our clinical development program to advance APR-246."
Aside from MDS, Aprea is testing APR-246 for acute myeloid leukemia (AML) and ovarian cancer. It's the company's only molecule in clinical trials. The company is focusing all its research on developing drugs that reactivate the cancer-suppressing p53 protein. Aprea had its initial public offering last year and was one of the top-performing IPOs for 2019, ending the year up 131%.