Vertex Pharmaceuticals Announces Updates on Type 1 Diabetes Portfolio

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Carmen Bozic

BOSTONVertex Pharmaceuticals Incorporated (Nasdaq: VRTX) provided key updates on its Type 1 Diabetes (T1D) portfolio, which includes progress on clinical trials for both VX-264 and Zimislecel (VX-880), as well as advancements in its research-stage T1D programs.

VX-264 Update
Vertex has completed enrollment and dosing in Parts A and B of the Phase 1/2 VX-264 (cells + device) study. The study focused on assessing the investigational fully differentiated pancreatic islet cell therapy encapsulated in a proprietary immunoprotective device. The primary endpoints of the trial included safety and changes in peak C-peptide levels, a marker of insulin production, during a mixed-meal tolerance test (MMTT) at Day 90.

The results showed that VX-264 was generally safe and well tolerated. However, the study did not meet the efficacy endpoint, as increases in C-peptide were not observed at levels necessary to demonstrate clinical benefit. As a result, Vertex will not pursue further clinical trials for VX-264. The company plans to conduct additional analyses, including an examination of explanted devices, to better understand the findings.

“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 of Global Medicines Development and Medical Affairs, and Chief Medical Officer of Vertex Pharmaceuticals.

Zimislecel Update
Vertex also provided an update on Zimislecel (formerly VX-880), its investigational fully differentiated islet cell therapy. The therapy, combined with standard immunosuppression, is currently in the Phase 3 portion of a Phase 1/2/3 study involving patients with T1D who experience severe hypoglycemic events (SHEs) and impaired awareness of hypoglycemia. Vertex is on track to complete enrollment and dosing in the first half of 2025, positioning the company to submit global regulatory applications in 2026.

Zimislecel has received Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations from the U.S. Food and Drug Administration, as well as Priority Medicines (PRIME) designation from the European Medicines Agency (EMA). Additionally, the therapy has secured an Innovation Passport under the Innovative Licensing and Access Pathway (ILAP) from the UK Medicines and Healthcare products Regulatory Agency (MHRA).

“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,” added Bozic. “We’re excited for the opportunity to bring the promise of zimislecel to patients as quickly as possible.”

T1D Research Update
In addition to its clinical programs, Vertex continues to advance research-stage T1D programs aimed at exploring transformative approaches that could reduce or eliminate the need for standard immunosuppressive regimens. These programs include alternative immunosuppressive strategies, gene-edited hypoimmune stem-cell derived islet cell therapies, and novel devices for encapsulating islet cells.

About Type 1 Diabetes
T1D is an autoimmune condition in which the body’s immune system attacks and destroys insulin-producing beta cells in the pancreas. As a result, individuals with T1D rely on lifelong insulin therapy to manage their blood glucose levels. Despite advancements in insulin delivery systems and glucose monitoring, people with T1D often experience dangerous fluctuations in blood sugar, which can result in severe complications. There is currently no cure for T1D, and existing treatments do not address the underlying cause of the disease.

Vertex Pharmaceuticals remains committed to advancing its innovative programs in the hopes of providing patients with better therapeutic options to manage or potentially overcome T1D.

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