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Publication in New England Journal of Medicine of results from phase II study of inaxaplin for focal segmental glomerulosclerosis.- Vertex

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Published:17th Mar 2023

Vertex Pharmaceuticals Incorporated announced publication in the New England Journal of Medicine (NEJM) of results from preclinical studies and a Phase II 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). AMKD is caused by variants of the APOL1 gene and affects approximately 100,000 people in the U.S. and Europe. Inaxaplin is an APOL1 inhibitor aimed at treating the underlying cause of AMKD.


The manuscript presents results from the Phase II 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. Reduction in proteinuria was observed early and continued throughout the 13-week treatment period. Results were consistent regardless of baseline proteinuria or background standard-of-care therapy. Inaxaplin was generally well tolerated in the study. The most common adverse events (occurring in greater than 15% of subjects) were headache, back pain and nausea. These results provide the first clinical evidence that an oral small molecule APOL1 inhibitor can decrease proteinuria in patients with AMKD.

Earlier last year, Vertex initiated a pivotal Phase II/III, randomized, double-blind, placebo-controlled study to assess the impact of inaxaplin, on top of standard-of-care, on kidney function and proteinuria in people with AMKD. The primary endpoint of the study is reduction in the rate of decline of kidney function as measured by estimated glomerular filtration rate (eGFR) slope in patients receiving inaxaplin compared to placebo after a minimum follow-up of approximately two years. The study is also designed to have a pre-planned interim analysis at Week 48 evaluating eGFR slope, supported by a percent change from baseline in proteinuria, in the inaxaplin arm versus placebo.

If positive, the interim analysis may serve as the basis for Vertex to seek accelerated approval of VX 147 in the U.S. for patients with AMKD. Enrollment in the study is ongoing, with more than 100 sites open in the U.S. and internationally, with 250 planned sites in total globally.

See- "Inaxaplin for Proteinuric Kidney Disease in Persons with Two APOL1 Variants"-O. Egbuna and Others. N Engl J Med 2023; 388:969-979';In a phase IIa study, 16 participants with APOL1 nephropathy received inaxaplin, an inhibitor of APOL1 channel function. Among 13 evaluable participants, the mean reduction in proteinuria was 47.6% at week 13.

Condition: Focal Segmental Glomerulosclerosis
Type: drug

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