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On December 1, 2021, Vertex Pharmaceuticals announced that a phase 2 clinical trial of the oral small molecule APOL1 inhibitor VX-147 has obtained positive results
APOL1-mediated kidney disease is a chronic kidney disease caused by mutations in the APOL1 gene
Image source: Vertex's official website
The primary endpoint of this phase 2 clinical trial is the percentage change from baseline in patients' UPCR at week 13, and the secondary endpoints are drug safety and pharmacokinetics
▲The decrease of proteinuria index was observed in the early stage of VX-147 treatment, and it continued to decrease during the 13-week course of treatment (picture source: reference [1])
In terms of safety, there were no treatment discontinuations due to adverse events (AE) in the trial, and no serious adverse events thought to be related to the study drug
Reference materials:
[1] Vertex Announces Positive Results From Phase 2 Study of VX-147 in APOL1-Mediated Focal Segmental Glomerulosclerosis.