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Vertex moves kidney disease drug into late-stage testing

Dive Brief:

  • Vertex Pharmaceuticals on Monday said it will advance an experimental kidney disease drug into Phase 3 testing and expand enrollment to include adolescents between the ages of 10 and 17.
  • The Phase 3 test will evaluate Vertex’s medicine inaxaplin, or VX-147, as a daily oral treatment for kidney disease caused by mutations in a gene called APOL1. The company will assess kidney function and urine protein levels over time, compared to a placebo.
  • Vertex is planning an interim analysis after 48 weeks that, if positive, could support an accelerated approval request to the Food and Drug Administration. The final study analysis will occur after at least two years of data is collected from study participants.

Dive Insight:

Vertex, as it sought to find success outside of the cystic fibrosis drugs that made its name, made a bet a few years ago on targeting APOL1-mediated kidney disease.

The company’s resulting drug inaxaplin works by blocking proteins encoded by the APOL1 gene. Two variants in this gene are known to increase the risk of “proteinuric” kidney disease and, in those who already have the disease, hasten progression. The gene variants are more common in people of African ancestry.

A small study in 2021 showed that treatment could reduce urine protein levels in people who had a manifestation of kidney disease called focal segmental glomerulosclerosis, or FSGS. This kidney scarring impacts the organ’s ability to filter blood, causing higher protein levels in urine.

Based on that data, Vertex advanced inaxaplin into a Phase 2/3 trial. Data from the first Phase 2 portion of the trial were published last year in The New England Journal of Medicine and again showed a decrease in proteinuria.

The Phase 3 part of the study will broadly enroll people with proteinuric APOL1-mediated kidney disease, or AMKD. There is currently no disease-specific therapy for AMKD, with treatment consisting of standard care for chronic kidney disease.

More than 200 trial sites are open in the U.S. and Europe, Vertex said.