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SAP-001 is a novel, orally administered small molecule inhibitor of uric acid transporter 1 (URAT1), developed by Shanton Pharma for the treatment of gout and management of hyperuricemia, particularly in patients refractory to standard-of-care xanthine oxidase inhibitor (XOI) therapy. It is designed to lower serum uric acid (sUA) levels by inhibiting URAT1 in the kidney, thereby reducing urate reabsorption and promoting its excretion. Clinical studies have shown that SAP-001 can achieve nearly 100% efficacy in lowering sUA below target levels (<6 mg/dL), with more than half of patients on higher doses reaching sUA <3 mg/dL. The drug has demonstrated an excellent safety profile and sustained efficacy over several months, with potential benefits including elimination or prevention of tophi and reduction in recurrent gout attacks. As of early 2025, SAP-001 is in Phase IIb clinical trials for refractory and tophaceous gout[1][2][4][5][6][8].
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