Press release
Vertex Advances VX-147 Into Pivotal Clinical Development for People With APOL1-Mediated Kidney Disease
– Single pivotal trial to initiate later this month targets the broad patient population with two APOL1 mutations and proteinuric kidney disease – – Pathway
About this update from Vertex Pharmaceuticals Incorporated
[{"type":"text","content":"\n– Single pivotal trial to initiate later this month targets the broad patient population with two APOL1 mutations and proteinuric kidney disease –\n\n– Pathway for accelerated approval using an interim analysis at Week 48 of eGFR slope, supported by reduction in proteinuria –\n\n– Final analysis evaluating eGFR slope at approximately two years serves as basis for U.S. approval –\n\n BOSTON--(BUSINESS WIRE)--\nVertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the company has advanced VX-147 into pivotal development for people living with proteinuric kidney disease mediated by two mutations in the APOL1 gene (AMKD), which affects approximately 100,000 people in the U.S. and Europe. VX-147 is the first investigational therapy aimed at treating the underlying cause of AMKD. The pivotal study will open for enrollment later this month.\n\nThe company will evaluate the safety and efficacy of VX-147 in a single pivotal clinical trial designed to assess the impact of VX-147, on top of standard of care, on kidney function and proteinuria in people with AMKD. The primary endpoint of this study is reduction in the rate of decline of kidney function as measured by estimated glomerular filtration rate (eGFR) slope versus placebo at approximately two years. The study is also designed to have a pre-planned interim analysis at Week 48 evaluating eGFR slope in VX-147 versus placebo, supported by a percent change from baseline in proteinuria. 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.\n\n“VX-147 holds the potential to be a first-in-class and best-in-class treatment for patients with AMKD, based upon Phase 2 results demonstrating a 47.6% reduction in proteinuria in APOL1-mediated focal segmental glomerulosclerosis (FSGS),” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex. “We are very pleased to have concluded our discussions with the FDA, enabling us to initiate the pivotal development program evaluating VX-147 in the broad patient population with AMKD and to have a clear path forward for potential accelerated approval in the U.S.”\n\nAbout the VX-147 Pivotal Program\nThis randomized, double-blind, placebo-controlled Phase 2/3 adaptive stu...