FDA Grants Breakthrough Designation to APR-246 for MDS

The FDA granted breakthrough therapy designation to APR-246 in combination with azacitidine for the treatment of patients with myelodysplastic syndromes (MDS) and a TP53 mutation.

APR-246 is a small-molecule therapy that reactivates mutant and inactivated p53 protein by restoring p53 conformation and function, thereby inducing programmed cell death in cancer cells. In pre-clinical trials, APR-246 demonstrated anti-tumor activity in a variety of solid and hematologic malignancies; in phase I/II studies, the agent’s adverse event profile was acceptable to most patients, and treatment was associated with clinical responses in patients with TP53-mutated hematologic malignancies.

Breakthrough-therapy designation is intended to expedite the development and review of drugs for the treatment of serious or life-threatening conditions and allows a sponsor to schedule more frequent meetings with the FDA. APR-246 previously received orphan-drug and fast-track designations from the FDA for MDS.

Source: Aprea Therapeutics press release, January 30, 2020.