PPQ-102 CAS NO.931706-15-9

ppq-102 is a potent cftr inhibitor which can completely inhibited cftr chloride current with ic50 approximately 90 nm.
ic50 value: 90 nm [1]
target: cftr
in vitro: the most potent compound, 7,9-dimethyl-11-phenyl-6-(5-methylfuran-2-yl)-5,6-dihydro-pyrimido[4',5'-3,4]pyrrolo[1,2-a]quinoxaline-8,10-(7h,9h)-dione, ppq-102, completely inhibited cftr chloride current with ic(50) approximately 90 nm. the ppqs, unlike prior cftr inhibitors, are uncharged at physiological ph, and therefore not subject to membrane potential-dependent cellular partitioning or block efficiency. patch-clamp analysis confirmed voltage-independent cftr inhibition by ppq-102 and showed stabilization of the channel closed state [1]. the three gpslc26 anion transporters exhibited distinct pharmacological profiles of (36)cl(-) influx, including partial sensitivity to cftr inhibitors inh-172 and glyh101, but only slc26a11 was inhibited by ppq-102 [2]. airway epithelial nci-h292 cells and primary cultures of noncystic fibrosis human airway epithelial cells were treated with cystic fibrosis transmembrane conductance regulator (cftr) inhibitors (cftr-inh(172) or ppq-102) or transfected with a cftr small interfering (si)rna with or without a selective epidermal growth factor receptor tyrosine kinase inhibitor [3].
in vivo: ppq-102 prevented cyst expansion and reduced the size of preformed cysts in a neonatal kidney organ culture model of polycystic kidney disease. ppq-102 is the most potent cftr inhibitor identified to date [1].