APX3330

APX3330 is a first-in-class, orally bioavailable small molecule inhibitor of the redox function of apurinic/apyrimidinic endonuclease 1/reduction–oxidation effector factor 1 (APE1/Ref‑1; also known as APEX1). It selectively targets and binds to APE1/Ref‑1 to inhibit its redox-dependent signaling activity without affecting its DNA repair function. By blocking the reduction and activation of multiple oncogenic transcription factors regulated by Ref‑1—including NF-kB, AP‑1, STAT3, p53, NRF2 and HIF‑1α—APX3330 modulates downstream pathways involved in angiogenesis (VEGF), oxidative stress (Nrf2), and inflammation (NFkB). This multimodal mechanism is being investigated for slowing or preventing progression in diseases such as diabetic retinopathy (DR), diabetic macular edema (DME), age-related macular degeneration (AMD), hepatitis B/C infection and liver cirrhosis. The drug has demonstrated a favorable safety profile in clinical trials[3][4][5][8][9].

Other names

APE1/REF-1 redox inhibitor APX3300Undecanoic acid, 2-((4,5-dimethoxy-2-methyl-3,6-dioxo-1,4-cyclohexadien-1-yl)methylene)-, (2e)-