Press release
Vertex Announces Positive Results From Phase 2 Study of VX-147 in APOL1-Mediated Focal Segmental Glomerulosclerosis
– Treatment with VX-147 led to a statistically significant, substantial and clinically meaningful mean reduction in proteinuria of 47.6% at 13 weeks compared
About this update from Vertex Pharmaceuticals Incorporated
[{"type":"text","content":"\n– Treatment with VX-147 led to a statistically significant, substantial and clinically meaningful mean reduction in proteinuria of 47.6% at 13 weeks compared to baseline and was well tolerated –\n\n– Results provide clinical proof of concept for the first genetically targeted approach to treating kidney disease –\n\n– Vertex to advance VX-147 into pivotal development in Q1 2022 –\n\n– Vertex to hold a conference call on these results today, December 1, at 8:30 am ET –\n\n BOSTON--(BUSINESS WIRE)--\nVertex 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.\nThis press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20211201005492/en/Figure 1. Primary Endpoint Results: Mean percent change from baseline in UPCR at Week 13 (Graphic: Business Wire)\nA 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.\n\nThere 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 (occurri...