836-P: Glucose-Dependent Insulin Production and Insulin-Independence in Type 1 Diabetes from Stem Cell–Derived, Fully Differentiated Islet Cells—Updated Data from the VX-880 Clinical Trial

作者：Trevor Reichman, CAMILLO RICORDI, ALI NAJI, James F. Markmann, BRUCE A. PERKINS, Martin Wijkstrom, Steven Paraskevas, Bote G. Bruinsma, Gautham Marigowda, Judy L. Shih, Chenkun Wang, Douglas A. Melton, FELICIA PAGLIUCA, Bastiano Sanna, Leslie S. Kean, Anne L. Peters, PIOTR WITKOWSKI, Michael R. Rickels · 发表于：Diabetes · 年份：2023 · DOI：10.2337/db23-836-p · 被引用次数：41 · 研究领域：Pancreatic function and diabetes、Diabetes Management and Research、Diabetes and associated disorders

VX-880 is an investigational allogeneic stem cell-derived, fully differentiated, pancreatic islet cell replacement therapy being evaluated in a phase 1/2 clinical trial in patients with T1D and impaired hypoglycemic awareness and severe hypoglycemia. The phase 1/2 trial has three parts: Part A in which 2 patients are enrolled sequentially and receive half the target dose (presented at ADA 2022), Part B in which 5 patients are enrolled sequentially and receive the target (full) dose, and Part C where 10 patients are enrolled concurrently and receive the target (full) dose. The first two patients infused with VX-880 at half the target dose (in Part A) had restored insulin production and glucose control. One of these patients achieved and has maintained insulin independence, defined as at least one week off exogenous insulin, HbA1C ≤7%, post-prandial serum glucose at 90 minutes ≤180 mg/dL, fasting serum glucose ≤126 mg/dL, and fasting or stimulated C-peptide ≥166 pmol/L (latter 3 measured during mixed-meal tolerance test). The safety profile was consistent with the immunosuppressive regimen used in the study and the perioperative period. Part B is now fully enrolled and multiple patients have been dosed with the full (target) dose. Longer-term data on both patients in Part A and new data on patients who received the full (target) dose in Part B will be provided in the presentation. These results are the first from a clinical trial of allogeneic, fully differentiated, insulin pro...