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Atiprimod + ursodiol is an experimental combination therapy formerly investigated for the treatment of multiple myeloma. Atiprimod is an oral small molecule azaspirane that acts as a STAT3 inhibitor, blocking IL-6 and VEGF signaling pathways and inducing apoptosis by downregulating anti-apoptotic proteins such as Bcl-2, Bcl-XL, and Mcl-1. Ursodiol (ursodeoxycholic acid) was co-administered in clinical trials to mitigate potential hepatobiliary toxicity associated with high-dose atiprimod. The regimen reached Phase II clinical development under AnorMED but was ultimately discontinued.
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