EPF-001

EPF-001 (also known as RK-701) is a first-in-class, highly selective small molecule inhibitor of the histone methyltransferase G9a (also known as EHMT2). Developed by EpiFrontier Therapeutics in collaboration with RIKEN and the Tokyo University of Pharmacy and Life Sciences, the drug is primarily indicated for the treatment of beta-globin disorders, including sickle cell disease and beta-thalassemia. The mechanism of action involves the selective inhibition of G9a, which leads to the induction of fetal hemoglobin (HbF) expression. By increasing HbF levels, EPF-001 aims to functionally compensate for the defective or deficient adult hemoglobin that characterizes these genetic blood disorders. The compound was identified through a large-scale screening campaign and optimized using X-ray crystallographic data. Preclinical studies have demonstrated its ability to significantly increase HbF levels in human cells and animal models with a favorable safety and pharmacokinetic profile. As of early 2026, the program has received significant non-dilutive funding to advance through Phase 2 clinical development.

Other names

G9a inhibitorG-9a inhibitorG 9a inhibitor