Vertex Announces Positive Results for VX-880 in Type 1 Diabetes Treatment

1. Positive Results: Vertex Pharmaceuticals announced positive results from the ongoing Phase 1/2 study of VX-880, a stem cell-derived islet cell therapy, for the treatment of type 1 diabetes.
2. Islet Cell Engraftment: All 12 patients who received the full dose of VX-880 demonstrated islet cell engraftment and glucose-responsive insulin production by Day 90.
3. Glycemic Control: All patients achieved ADA-recommended target HbA1c levels <7.0% and >70% time-in-range (70-180 mg/dL), and 11 of 12 patients reduced or eliminated use of exogenous insulin.
4. Elimination of Severe Hypoglycemic Events: Three patients with at least 12 months of follow-up met the primary endpoint of elimination of severe hypoglycemic events (SHEs) with HbA1c <7.0%, and the secondary endpoint of insulin independence. 5. Trial Expansion: The trial has been expanded to enroll approximately 37 participants to generate clinical data that will support the goal of making VX-880 available for people living with type 1 diabetes in the future. 6. Safety Profile: VX-880 has been generally well tolerated, with the majority of adverse events being mild or moderate, and no serious adverse events related to VX-880 treatment. 7. Potential for Insulin Independence: The study suggests that VX-880 stem cell-derived islets can restore physiological islet function and glycemic control, potentially relieving some people with type 1 diabetes of the need for daily insulin injections.