Vertexs second-generation diabetes cell therapy fails early-stage study

Vertex's VX-264 Diabetes Cell Therapy Fails in Early-Stage Trial

Key Facts

Vertex Pharmaceuticals has halted development of VX-264, its second-generation type 1 diabetes cell therapy1 7
The Phase I/II trial failed to meet efficacy endpoints, with insufficient increases in C-peptide levels1 7
VX-264 used the same cell therapy as VX-880 but encapsulated in a device to avoid immunosuppression8
Vertex will conduct further analyses to better understand the trial results1
The company continues to advance its VX-880 (zimislecel) program in Phase III trials1 7

Background

VX-264 was designed to deliver insulin-producing cells via an implantable device without requiring immunosuppression8. This was intended to improve upon Vertex's VX-880 therapy, which has shown promising results but requires immunosuppression2 3.

Trial Details

The trial enrolled patients with type 1 diabetes and severe hypoglycemia unawareness1
Primary endpoints were safety and change in peak C-peptide levels7
C-peptide increases were not observed at levels necessary for clinical benefit1

Impact and Next Steps

Vertex will not advance VX-264 further in clinical development1 7
The company plans to analyze explanted devices to understand the results1
Focus shifts to the ongoing Phase III trial of VX-880, expected to complete enrollment in H1 20251 7

Broader Context

This setback highlights the challenges in developing cell therapies for type 1 diabetes, particularly in protecting transplanted cells from immune attack without immunosuppression4. However, Vertex's VX-880 program continues to show promise, with 11 out of 12 patients achieving insulin independence in earlier trials2 3.