DF-003

DF-003 is a first-in-class, oral, potent, and highly selective small molecule inhibitor of alpha-kinase 1 (ALPK1), including gain-of-function ALPK1 mutations such as the T237M variant that cause ROSAH syndrome. Developed by Drug Farm, DF-003 targets ALPK1 to modulate inflammation pathways implicated in rare genetic blindness (ROSAH syndrome) and cardio-renal diseases such as chronic kidney disease. Preclinical studies have shown significant activity in animal models of heart and kidney disease as well as ROSAH transgenic mouse models. The drug has favorable pharmacokinetic properties supporting once-daily oral dosing and a low risk for off-target effects or drug interactions. As of 2024, DF-003 is undergoing Phase 1 clinical trials for ROSAH syndrome and kidney/inflammatory diseases[1][2][4][5][6][8].