Vertex Ends VX-264 Trials, Focuses on Zimislecel

Vertex Pharmaceuticals announced it will discontinue the clinical trials for its type 1 diabetes candidate VX-264 after failing to meet efficacy endpoints regarding insulin production in a Phase 1/2 study, leading to a $400 million impairment charge. Despite the therapy being safe and well-tolerated, increases in C-peptide, a marker of insulin production, were insufficient to warrant further development. However, Vertex's other diabetes candidate, zimislecel, continues to progress in clinical trials, with plans for regulatory submission in 2026. Zimislecel, which includes standard immunosuppression, remains promising as it advances through the Phase 3 portion of its trial. Meanwhile, Vertex remains committed to expanding its diabetes research, including gene-edited therapies and novel encapsulation devices.