Vertex reports positive Phase 2b results for inaxaplin in APOL1-mediated kidney disease

Vertex Pharmaceuticals announced positive Phase 2b data for its kidney disease candidate inaxaplin, which targets APOL1-mediated kidney disease (AMKD).

The Amplified trial enrolled two cohorts: 22 patients with AMKD and modest proteinuria, and 17 patients with AMKD plus type 2 diabetes, a population different from the earlier Amplitude trial that focused on severe proteinuria without comorbidities.

At week 13, the primary endpoint of urinary albumin-to-creatinine ratio (UACR) fell 42.7% in the modest proteinuria group and 17.3% in the diabetes group. A secondary endpoint, urinary protein-to-creatinine ratio (UPCR), declined 44.7% and 25.4% respectively.

Vertex’s chief medical officer described the reduction in the modest proteinuria cohort as remarkable, while acknowledging the smaller effect observed in the diabetes cohort.