Vertex Pharmaceuticals says VX-264 study did not meet primary endpoint

Vertex Pharmaceuticals (VRTX) announced several updates on the company’s type 1 diabetes portfolio. Vertex has completed enrollment and dosing in Parts A and B of the Phase 1/2 VX-264 study and the planned analysis at Day 90 for Part B. In Part B of the study, participants received the full dose of the investigational fully differentiated pancreatic islet cell therapy encapsulated in a proprietary immunoprotective device. There were two primary endpoints in Part B, safety and change in peak C-peptide during a mixed-meal tolerance test from baseline at Day 90. VX-264 was generally safe and well tolerated; however, the study did not meet the efficacy endpoint. Increases in C-peptide, a marker of insulin production, were not observed at levels necessary to deliver benefit. Therefore, VX-264 will not be advancing further in clinical trials. Vertex plans to conduct further analyses, including of explanted devices, to better understand these findings.