Vertex Pharmaceuticals Incorporated announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug Application (IND) for VX-407, an investigational first-in-class small molecule corrector that targets the underlying cause of autosomal dominant polycystic kidney disease (ADPKD) in patients with a subset of PKD1 genetic variants. ADPKD is the most common inherited kidney disease, with an estimated 250,000 people in the U.S. and Europe living with ADPKD; however, there are no treatments currently available that address the underlying causal biology of the disease. ADPKD is a life-shortening genetic kidney disease characterized by the growth of numerous kidney-en largering cysts that impair kidney function and can ultimately lead to kidney failure, requiring dialysis or kidney transplantation, and premature death.

The majority of ADPKD cases are caused by variants in the PKD1 gene, which encodes the polycystin 1 (PC1) protein. These inherited variants lead to a loss of PC1 function that results in cyst growth. VX-407 is a first-in-class small molecules corrector that is designed to target the underlying cause of ADPKD in a subset of patients with PKD1 variants, estimated at 25,000 (or 10%) of the overall ~250,000 ADPKD patient population, by restoring function to the variant PC1 protein.

Vertex plans to initiate a Phase 1 clinical trial of VX-407 in healthy volunteers this month.