Vertex Pharmaceuticals pursuing research-stage T1Dd programs

Zimislecel, 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 (MHRA). Consistent with this progress, Vertex is investing in expanding its manufacturing and commercial capabilities to ensure launch readiness. If approved, eligible patients across the U.S. and Europe with recurrent SHEs despite best available care could benefit from zimislecel, and Vertex anticipates that initial approval could serve approximately 60,000 people with severe T1D. Vertex is also pursuing research-stage T1D programs to evaluate additional approaches that could provide transformative benefit to people with T1D and reduce or eliminate the need for standard immunosuppressive regimens. These approaches include alternative immunosuppressive regimens, gene-edited hypoimmune stem-cell derived islet cell therapies, and novel devices to encapsulate islet cells. “We’d like to thank the patients, physicians and T1D community who participated in the VX-264 study. Today’s data show that more work needs to be done to advance the ‘cells plus device’ program, and we are committed to doing so,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer, Vertex Pharmaceuticals (VRTX). “Equally, we are very pleased with the rapid progress of our zimislecel program, which is on track to complete enrollment and dosing in the Phase 3 study this summer, positioning us for global regulatory submissions in 2026. We’re excited for the opportunity to bring the promise of zimislecel to patients as quickly as possible.”