Positive data for zimislecel in type 1 diabetes presented at ADA 2025
Vertex presented updated data from the Phase 1/2 portion of the Phase 1/2/3 FORWARD-101 clinical trial of zimislecel during the American Diabetes Association 85th Scientific Sessions.
The clinical trial analysed how people with type 1 diabetes with impaired hypoglycaemic awareness and severe hypoglycaemic events can benefit from zimislecel – an investigational stem cell-derived, fully differentiated islet cell therapy.
The data were featured in an oral presentation at the American Diabetes Association (ADA) annual conference in Chicago as part of the symposium, ‘Innovation and Progress in Stem Cell–Derived Islet-Cell Replacement Therapy,’ and published online by the New England Journal of Medicine.
The data are from 12 people who received the full dose of zimislecel as a single infusion and were followed for at least one year, as of October 2024.
Results from the study to date continue to demonstrate the transformative potential of zimislecel with consistent and durable patient benefit with longer follow-up.
All 12 participants:
Demonstrated engraftment with glucose-responsive endogenous C-peptide production, which was durable through one year of follow-up
Achieved the ADA targets of HbA1c <seven per cent and time in range of >70 per cent
Were free of severe hypoglycaemic events from day 90 onwards
Had a reduction in exogenous insulin use (mean reduction in daily insulin dose: 92 per cent)
A total of 10/12 (83 per cent) no longer required exogenous insulin at month 12
Achieved the Phase 1/2 primary endpoint of elimination of severe hypoglycaemic events with HbA1c <seven per cent.
Zimislecel continues to be generally well tolerated. Most adverse events were mild or moderate, and there were no serious adverse events related to zimislecel treatment.
As previously reported, two patient deaths occurred, both unrelated to treatment with zimislecel.
The safety profile is generally consistent with the immunosuppressive regimen used in the study, the infusion procedure, and complications from long-standing diabetes.