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Description: Leniolisib phosphate (formerly known as CDZ173 phosphate) is a novel, potent and selective PI3K (phosphatidylinositol 3-kinase inhibitor) inhibitor with IC50 values of 0.244, 0.424, 2.23 and 0.011 uM for PI3Kalpha, PI3Kbeta, PI3Kgamma and PI3Kdelta, respectively. It is currently in phase II/III clinical trials for the treatment of immunodeficiency disorders. Oral leniolisib led to a dose-dependent reduction in PI3K/AKT pathway activity assessed ex vivo and improved immune dysregulation. Leniolisib was well tolerated and improved laboratory and clinical parameters in APDS, supporting the specific inhibition of PI3Kdelta as a promising new targeted therapy in APDS and other diseases characterized by overactivation of the PI3Kdelta pathway. This trial was registered at www.clinicaltrials.gov as #NCT02435173.
References: Blood. 2017 Nov 23; 130(21):2307-2316.
Related CAS#:1354690-24-6 (free base)
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