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Vertex Starting Phase I Trial of VX-407 in Autosomal Dominant Polycystic Kidney Disease

NEW YORK – The US Food and Drug Administration has granted Vertex Pharmaceuticals permission to begin studying VX-407, a treatment for autosomal dominant polycystic kidney disease (ADPKD), in a Phase I trial of healthy volunteers, the firm said Thursday.

Most cases of ADPKD are caused by variants in the PKD1 gene that, when functional, encodes for the polycystin 1 (PC1) protein. These variants, however, lead to a loss of PC1 function, resulting in cysts that impair kidney function and, ultimately, kidney failure requiring dialysis or kidney transplantation.

Vertex has designed VX-407, an investigational small molecule corrector, to restore PC1 protein function in ADPKD patients with certain PKD1 variants, representing about 10 percent of the ADPKD patient population. 

The FDA's clearance of the Boston-based firm's investigational new drug application for VX-407 makes ADPKD the 10th disease area in which Vertex has launched a drug trial. Vertex expects to initiate the Phase I study this month.