Vertex Pharmaceuticals announced the discontinuation of its VX-264 (cells + device) program for Type 1 Diabetes (T1D) following the completion of enrollment and dosing in Parts A and B of the Phase 1/2 study. While VX-264 was generally safe and well tolerated, the study did not meet its efficacy endpoint, as necessary increases in C-peptide were not observed.
In contrast, Vertex's Zimislecel (formerly VX-880) program, an investigational fully differentiated islet cell therapy with standard immunosuppression, is progressing as planned. The pivotal Phase 3 portion of its clinical trial is on track to complete enrollment and dosing in the first half of 2025.
Vertex anticipates global regulatory submissions for Zimislecel in 2026, if data are supportive. Zimislecel has received Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations from the U.S. FDA, and Priority Medicines (PRIME) designation from the European Medicines Agency (EMA), targeting approximately 60,000 severe T1D patients initially.
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