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GSK3039294 is an orally available small molecule prodrug developed as an alternative to parenteral miridesap (GSK2315698) for the depletion of serum amyloid P component (SAP). Its mechanism of action is inhibition of SAP, a plasma protein that binds to amyloid fibrils and stabilizes them. By depleting SAP, the drug aims to facilitate the removal of systemic amyloid deposits, particularly in conditions such as systemic amyloidosis. The compound was designed to improve oral bioavailability while maintaining the pharmacological properties of miridesap. Development was led by GSK (GlaxoSmithKline), but clinical development has been discontinued due to safety observations in early human studies[1][2][3][6][7].
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