Vertex Announces Positive Results From Phase 2 Study of VX-147 in APOL1-Mediated Focal Segmental Glomerulosclerosis
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that, in a Phase 2 proof-of-concept (POC) study in patients with APOL1-mediated focal segmental glomerulosclerosis (FSGS), VX-147 on top of standard of care achieved a statistically significant, substantial and clinically meaningful mean reduction of 47.6% in the urine protein to creatinine ratio (UPCR) at Week 13 compared to baseline. VX-147 was well tolerated. These results provide the first clinical evidence and POC that an oral small molecule APOL1 inhibitor can decrease proteinuria in patients with APOL1-mediated kidney disease. Based on these results, Vertex plans to advance VX-147 into pivotal development in APOL1-mediated kidney disease, including FSGS, in Q1 2022.
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Figure 1. Primary Endpoint Results: Mean percent change from baseline in UPCR at Week 13 (Graphic: Business Wire)
A total of 16 patients were enrolled in the study. According to the pre-specified statistical analysis plan, three patients who were noncompliant with treatment were not included in the primary efficacy analysis. In the 13 evaluable patients, treatment with VX-147 on top of standard of care resulted in a rapid, statistically significant and clinically meaningful mean change in proteinuria from baseline of -47.6% (95% CI: -60.0%, -31.3%) following 13 weeks of treatment. Reduction in proteinuria was observed early and continued throughout the 13-week treatment period. Results were consistent regardless of baseline proteinuria or background therapy.
There were no treatment discontinuations due to adverse events (AEs), and there were no serious adverse events considered related to study drug. All AEs were mild or moderate in severity. The most common AEs (occurring in > 15% of subjects) were headache, back pain and nausea.
“VX-147 is the first investigational treatment targeting the underlying cause of APOL1-mediated kidney disease. These results demonstrate that inhibition of the APOL1 protein can substantially reduce proteinuria in patients with two APOL1 genetic variants, FSGS and significant proteinuria,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex. “We are working with urgency to advance this molecule into pivotal development with the goal of bringing this first-in-class therapy to the more than 100,000 patients in the U.S. and Europe living with APOL1-mediated kidney disease.”