KDU 731

KDU 731 is an orally administered small-molecule pyrazolopyridine antiparasitic that selectively inhibits the Cryptosporidium phosphatidylinositol 4-kinase (PI4K, also termed PI(4)K), leading to potent blockade of parasite growth in vitro and in vivo with minimal host toxicity.[1][7][9] By competitively binding to the ATP site of the Cryptosporidium PI4K enzyme, KDU 731 disrupts parasite lipid signaling and replication while showing a >50-fold selectivity window over the human PI4K IIIβ homologue and little off-target activity in human safety panels.[1][2] The compound demonstrated strong efficacy in immunocompromised mouse models and neonatal calf models of cryptosporidiosis, markedly reducing oocyst shedding, diarrhea, and dehydration, and is being advanced primarily for the treatment of cryptosporidiosis in young children in resource-limited settings by Novartis in collaboration with academic partners.[1][2][5][7][9]

Other names

3-(4-(aminocarbonyl)phenyl)-N-(4-cyanophenyl)-N-methylpyrazolo(1,5-a)pyridine-5-carboxamidepyrazolopyridine KDU731