Vertex Pharmaceuticals has announced updated data from the Phase 1/2 portion of its ongoing Phase 1/2/3 FORWARD-101 clinical trial evaluating zimislecel (VX-880), an investigational, stem cell-derived, fully differentiated islet cell therapy for patients with type 1 diabetes (T1D) who have impaired hypoglycemic awareness and severe hypoglycemic events (SHEs).
The data were presented at the American Diabetes Association (ADA) annual conference and simultaneously published in the New England Journal of Medicine.
The study evaluated 12 patients, each of whom received a single infusion of the full dose of zimislecel and was monitored for at least one year. Key outcomes observed in the trial include:
- All 12 patients demonstrated lasting engraftment with glucose-responsive endogenous C-peptide production through one year.
- All patients achieved ADA-recommended glycemic control targets: HbA1c levels below 7% and greater than 70% time-in-range (70-180 mg/dL).
- No severe hypoglycemic events (SHEs) occurred from day 90 onwards.
- Patients experienced a mean reduction of 92% in daily insulin dose; 10 of 12 (83%) were insulin-free by month 12.
- The primary endpoint of the Phase 1/2 study, which was the elimination of SHEs with HbA1c levels under 7%, was met.
Zimislecel was generally well tolerated, with most adverse events reported as mild or moderate. There were no serious adverse events directly related to the therapy. Two patient deaths occurred during the study period, neither of which were related to zimislecel treatment. Reported adverse events were consistent with the immunosuppressive regimen and infusion procedure used, as well as complications typically associated with long-standing diabetes.
Michael R. Rickels, M.D., M.S., Medical Director of the Pancreatic Islet Cell Transplant Program at the Perelman School of Medicine at the University of Pennsylvania and presenting author and steering committee co-chair for the zimislecel clinical program, commented: “It’s remarkable to see 12 out of 12 patients with baseline HbA1c above 7% and multiple severe hypoglycemic events reach consensus targets for glycemic control by both HbA1c and time in range as well as elimination of severe hypoglycemic events. As I think about my patients and the unmet need in the type 1 diabetes community, the results we’ve seen so far for restoring endogenous insulin secretion with a stem cell-derived islet therapy bring me hope and confidence for a transformative treatment option for individuals with type 1 diabetes in the not-so-distant future.”
About Zimislecel (VX-880)
Zimislecel is an investigational allogeneic stem cell-derived therapy intended to restore pancreatic islet cell function, including glucose-responsive insulin production. It is administered via infusion into the hepatic portal vein and requires chronic immunosuppressive therapy to prevent immune rejection. The clinical trial for zimislecel is actively enrolling patients across multiple sites in the U.S., Canada, and Europe.
Zimislecel has received the following regulatory designations:
- Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations from the U.S. Food and Drug Administration (FDA)
- Priority Medicines (PRIME) designation from the European Medicines Agency (EMA)
- Innovation Passport under the Innovative Licensing and Access Pathway (ILAP) from the UK Medicines and Healthcare products Regulatory Agency (MHRA)
Zimislecel remains investigational and has not yet been approved by health authorities globally.
