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APX2044 is a preclinical-stage small molecule inhibitor developed by Apexian Pharmaceuticals. It specifically targets the redox signaling function of Apurinic/apyrimidinic endonuclease 1/Redox effector factor 1 (APE1/Ref-1). APE1/Ref-1 is a dual-function protein; while its endonuclease activity is central to base excision repair of DNA, its redox activity regulates the DNA-binding state of several transcription factors involved in disease progression, including NF-κB, AP-1, and HIF-1α. By inhibiting this redox function, APX2044 modulates downstream pathways associated with inflammation, angiogenesis, and cell survival. The drug is being investigated for the treatment of solid tumors, inflammatory bowel disease (IBD), ocular diseases such as diabetic retinopathy, and various forms of neuropathy.
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