In the preclinical study of Plexxikon’s polycystic kidney disease (PKD) drug candidate, doses of the drug candidate were administered orally daily for 14 days. A marked reduction in kidney cyst burden was observed in the treatment group compared to vehicle treated mice.
Levels of blood urea nitrogen, an indicator of kidney function, were also significantly improved in the drug-treated groups compared with the vehicle-treated group, suggesting that improved renal function accompanied reduced cyst growth in treated animals.
Plexxikon’s novel small molecule kinase inhibitor is said to be a highly selective and potent inhibitor of Raf kinase, a critical mediator of PKD pathology.