Press release
Vertex Announces Program Updates for Type 1 Diabetes Portfolio
- VX-264 Phase 1/2 enrollment and dosing complete in Parts A and B: VX-264 was generally safe and well tolerated; efficacy data are not supportive of further
About this update from Vertex Pharmaceuticals Incorporated
[{"type":"text","content":"\n- VX-264 Phase 1/2 enrollment and dosing complete in Parts A and B: VX-264 was generally safe and well tolerated; efficacy data are not supportive of further clinical advancement -\n\n- Zimislecel (VX-880) pivotal trial on track to complete enrollment and dosing in H1 2025; Vertex expects to submit marketing applications to global regulators in 2026 -\n\n- Continue to progress multiple novel, research-stage immunoprotective approaches -\n\n BOSTON--(BUSINESS WIRE)--\nVertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced several updates on the Company’s type 1 diabetes (T1D) portfolio.\n\nVX-264 Update\nVertex has completed enrollment and dosing in Parts A and B of the Phase 1/2 VX-264 (cells + device) 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 (MMTT) 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.\n\nZimislecel Update\nZimislecel (formerly VX-880), Vertex’s investigational fully differentiated islet cell therapy with standard immunosuppression, is in the Phase 3 portion of the Phase 1/2/3 study in patients with T1D with severe hypoglycemic events (SHEs) and impaired awareness of hypoglycemia. This pivotal trial is well underway and on track to complete enrollment and dosing in the first half of 2025, setting up global regulatory submissions in 2026. Zimislecel has previously been granted Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations from the U.S. Food and Drug Administration, Priority Medicines (PRIME) designation from the European Medicines Agency (EMA), and has secured an Innovation Passport under the Innovative Licensing and Access Pathway (ILAP) from the UK Medicines and Healthcare products Regulatory Agency (M...