Vertex to send kidney disease drug to phase 2/3 trials

March 23, 2022

Vertex Pharmaceuticals is ready to get its phase 2/3 clinical trial of new kidney medication VX-147 on the road.

The trial will look at how the drug impacts kidney function and levels of protein in urine in participants with a type of genetic kidney disease caused by mutations in the APOL1 gene. The study will run for approximately two years, with an interim analysis scheduled halfway through the trial, which could be a chance for the company to seek accelerated approval from the U.S. FDA if the results are positive.

Vertex believes that targeting the APOL1 gene may be key in treating kidney disease. If the company is correct, there is potential for VX-147 to be a first-in-class and best-in-class treatment for patients, said Carmen Bozic, chief medical officer at Vertex. 

APOL1-mediated kidney disease is a chronic kidney disease caused by genetic mutations on the APOL1 gene. According to Vertex’s estimates, about 100,000 people in the U.S. and Europe have elevated protein levels and two APOL1 mutations, which can cause more aggressive forms of kidney disease. 

Early signs of hope for the drug came in December, when Vertex reported results from a phase 2 proof-of-concept study. The company, known for its cystic fibrosis treatments, said that the study provided statistically significant evidence that VX-147 can reduce levels of protein in urine, known as proteinuria, which tend to rise as kidneys lose their ability to filter blood.

The company is expected to start enrolling patients in its trial next month.