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PXS64 is a small molecule analogue of mannose-6-phosphate (M6P) developed as an anti-fibrotic agent. It acts as an inhibitor of the cation-independent mannose 6-phosphate receptor (CI-M6PR), thereby blocking the activation of latent transforming growth factor-beta 1 (TGF-β1). By inhibiting TGF-β1 signaling, PXS64 reduces the production of extracellular matrix proteins such as collagen and fibronectin and suppresses myofibroblast differentiation. Preclinical studies have demonstrated that PXS64 ameliorates fibrosis in models of idiopathic pulmonary fibrosis and kidney fibrosis by decreasing fibrotic and inflammatory markers in human lung fibroblasts, IPF patient fibroblasts, and human kidney tubular cells[1][2][3][4][8]. The drug is considered a potential candidate for further preclinical development in fibrotic diseases.
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