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RX-0047 is a first-in-class small molecule inhibitor of the hypoxia-inducible factor 1 (HIF-1) transcription factor, which regulates genes involved in angiogenesis, cancer cell survival, invasion, and radiation resistance. Developed by Rexahn Pharmaceuticals, it demonstrated potent inhibition of cancer cell proliferation at nanomolar concentrations in preclinical studies across human solid tumors, including lung and prostate cancers, reduced tumor growth in xenograft models, and inhibited lung metastasis formation. A nanoliposomal formulation, RX-0047-Nano, was designed to enhance efficacy, extend circulation time, and reduce toxicity, with lipid-albumin nanoparticles (LAN-RX-0047) showing prominent HIF-1α downregulation and tumor growth inhibition in vivo.
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