These results provide the first clinical evidence that an oral small molecule APOL1 inhibitor can decrease proteinuria in patients with AMKD. The most common adverse events (occurring in >15% of subjects) were headache, back pain and nausea. Inaxaplin was generally well tolerated in the study.

Results were consistent regardless of baseline proteinuria or background standard-of-care therapy. Reduction in proteinuria was observed early and continued throughout the 13-week treatment period. The manuscript presents results from the Phase 2 study of inaxaplin, demonstrating a statistically significant and clinically meaningful mean reduction in proteinuria of 47.6% (95% CI: 60.0%, 31.3%) at 13 weeks compared to baseline, which was the primary endpoint of the study. Inaxaplin is an APOL1 inhibitor aimed at treating the underlying cause of AMKD. AMKD is caused by variants of the APOL1 gene and affects approximately 100,000 people in the U.S.

Vertex Pharmaceuticals Incorporated VRTX today announced publication in the New England Journal of Medicine (NEJM) of results from preclinical studies and a Phase 2 study evaluating the efficacy and safety of inaxaplin (VX-147) on top of standard-of-care in people with focal segmental glomerulosclerosis (FSGS) and two APOL1 variants, a severe, rapidly progressive form of chronic kidney disease also known as APOL1-mediated kidney disease (AMKD).

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