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AC-201CR is a controlled-release oral formulation of AC-201, a first-in-class, orally available small molecule developed by TWi Biotechnology. The drug acts primarily as an inhibitor of the NLRP3 inflammasome pathway, reducing the production and activity of interleukin-1β (IL-1β) and caspase-1. It also inhibits URAT1 (urate transporter 1), contributing to its urate-lowering effects. These mechanisms make it relevant for inflammatory diseases such as gout, arthritis (including osteoarthritis and hemophilic arthropathy), psoriasis, and type 2 diabetes. The active ingredient in AC-201 has been approved for chronic rheumatic diseases in parts of Europe and Asia since the mid-1990s but not in the US or other major markets[5][8][3]. Clinical trials have demonstrated anti-inflammatory effects, cartilage-sparing properties in animal models, reduction of serum uric acid levels, control of blood glucose/HbA1c in type 2 diabetes patients, and potential benefit for immunodermatology conditions[5][6][8].
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