Leniolisib (CDZ173) phosphate is a potent and selective PI3Kδ inhibitor. Leniolisib phosphate has the potential for immunodeficiency disorders treatment.
Expression of APDS mutant p110δ in cell lines and patient-derived lymphocytes lead to increased pathway activity, measured as phosphorylation of AKT or S6, which is suppressed by Leniolisib phosphate in a concentration dependent way[1].
References:
[1]. Rao V, et al. Effective 'Activated PI3Kd Syndrome' -targeted therapy with PI3Kd inhibitor leniolisib. The New England journal of medicine: NEJM. ISSN 0028-4793; 1533-4406
[2]. Hoegenauer K, et al. Discovery of CDZ173 (Leniolisib), Representing a Structurally Novel Class of PI3K Delta-Selective Inhibitors. ACS Med Chem Lett. 2017 Aug 25;8(9):975-980.