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Aprea Therapeutics to Begin Phase I Trial for APR-548 in Myelodysplastic Syndromes

NEW YORK – Aprea Therapeutics said on Wednesday that it will initiate a Phase I trial exploring the activity of APR-548 in TP53-mutant myelodysplastic syndromes (MDS).

APR-548 is a small molecule reactivator of mutant p53 that Boston-headquartered Aprea is developing for oral administration. Half of all tumors harbor mutations in TP53, making it one of the most common genetic aberrations involved in cancer, and cancer patients with TP53 mutations in their tumors often have poor overall survival and tend to develop resistance to available therapies.

Aprea's drug has demonstrated activity against TP53-mutant tumor cells in preclinical studies. The firm will now advance APR-548 into Phase I human studies for the treatment of TP53-mutant MDS after the US Food and Drug Administration accepted its investigational new drug application for the agent.

"The IND acceptance by FDA is an important milestone for APR-548 and Aprea's development of next-generation reactivators of mutant p53," Aprea CEO Christian Schade said in a statement. "We look forward to initiating the Phase I clinical trial of APR-548 in MDS and to expanding our leadership in the development of needed therapeutic options for patients with p53-mutated cancers."

This is the second therapy targeting p53 that Aprea has moved into trials. Its other investigational treatment, eprenetapopt (APR-246), entered a Phase III trial earlier this year for the treatment of front-line TP53-mutant MDS. Eprenetapopt is also being studied to treat TP53-mutant acute myeloid leukemia.