Vertex Announces Positive Results From Phase 2 Study of VX-147 in APOL1-Mediated Focal Segmental Glomerulosclerosis - Seite 2

“These results are very promising. For decades, I have cared for patients who have suffered from the rapid onset of kidney disease that progresses quickly to kidney failure, which we now know may have been caused by APOL1 gene variants,” said Glenn Chertow, M.D., M.P.H., Professor of Medicine, Stanford University School of Medicine, and Chair of the Vertex APOL1 Program Steering Committee. “This approach has tremendous potential, as it targets the underlying genetic driver of kidney disease in these patients. These data demonstrate the potential of VX-147 as a targeted treatment for a patient population at unusually high risk of progression to kidney failure.”

Based on these data, Vertex plans to advance VX-147 into pivotal development in Q1 2022 as a potential first-in-class therapy for the more than 100,000 patients with proteinuric kidney disease mediated by variants in the APOL1 gene, including, but not limited to, FSGS. Consistent with the company’s serial innovation strategy, Vertex will also continue to investigate additional small molecule APOL1 inhibitors in the clinic.

About the VX-147 Phase 2 Study

The Phase 2 open-label, single-arm study evaluated the efficacy, safety and pharmacokinetics of VX-147 in patients with APOL1-mediated FSGS. Patients with biopsy-confirmed FSGS, two APOL1 genetic variants, proteinuria defined by at least 0.7 g/g in the UPCR and an estimated glomerular filtration rate (eGFR) of at least 27 mL/min/1.73 m² were eligible for enrollment in the study. Patients were on a stable standard-of-care regimen, which could include an angiotensin-converting enzyme (ACE) inhibitor, an angiotensin II receptor blocker (ARB), immunosuppressants and/or low doses of corticosteroids. Patients were treated with VX-147 for a total of 13 weeks. The primary endpoint was percent change from baseline in UPCR at Week 13. The secondary endpoints were safety and pharmacokinetics. In addition, there is a 28-day safety follow-up period after the last dose of treatment and an optional off-treatment follow-up period of up to 12 weeks after the last dose of treatment. The study is ongoing for these follow-up periods.

About APOL1-Mediated Kidney Disease

APOL1-mediated kidney disease is a form of chronic kidney disease caused by variants of the APOL1 gene. Over 100,000 people in the U.S. and Europe have two APOL1 genetic variants and proteinuric kidney disease. People who inherit two risk variants in the APOL1 gene have a course of disease that is far more aggressive than in the absence of APOL1 genetic variants. Inherited APOL1 genetic variants cause kidney disease through a toxic gain of function, which leads to podocyte injury. This injury disrupts filtration, resulting in proteinuria and rapidly progressive kidney disease. Progressive kidney disease can result in dialysis, kidney transplant or death.