Vertex Pharma Reports Positive Results From Phase 2 Study Of VX-147 - Quick Facts

(RTTNews) - Vertex Pharmaceuticals Incorporated (VRTX) said, in a phase 2 proof-of-concept study in patients with APOL1-mediated focal segmental glomerulosclerosis, 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 at week 13 compared to baseline.

"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, Chief Medical Officer at Vertex.

Based on the results, the company plans to advance VX-147 into pivotal development in APOL1-mediated kidney disease, including FSGS, in first quarter 2022.