Vertex Pharmaceuticals Incorporated has recently unveiled promising new data from their Phase 1/2 clinical trial of VX-880, an investigational therapy designed for people with type 1 diabetes (T1D) who experience impaired hypoglycemic awareness and severe hypoglycemic events (SHEs). VX-880 represents a cutting-edge approach in diabetes treatment, utilizing stem cell-derived, fully differentiated islet cells to restore the body’s ability to regulate glucose levels by re-establishing pancreatic islet cell function and glucose-responsive insulin production.

      VX-880 is administered via infusion into the hepatic portal vein and requires ongoing immunosuppressive therapy to protect the transplanted islet cells from immune system rejection. This investigational therapy holds the potential to significantly improve the lives of people with T1D.

      The latest data, gathered from an extended study involving 12 patients who received a full dose of VX-880 in Parts B and C of the trial, are encouraging. At the start of the study, all participants had undetectable fasting C-peptide levels (indicating no endogenous insulin secretion), a history of recurrent SHEs in the previous year, and required an average of 39.3 units of insulin per day.

      Following a single infusion of VX-880, all 12 patients showed successful islet cell engraftment and glucose-responsive insulin production by Day 90. Remarkably, all patients demonstrated improved glycemic control and met the American Diabetes Association (ADA) targets of an HbA1c below 7.0% and over 70% time-in-range on continuous glucose monitoring. Nearly all participants (11 out of 12) reduced or completely eliminated their need for external insulin by their last follow-up visit. Importantly, all patients were free from severe hypoglycemic events from Day 90 onward.

      Among the three patients who had at least one year of follow-up, all achieved the primary endpoint of eliminating SHEs with HbA1c levels below 7.0%, and the secondary endpoint of insulin independence.

      These groundbreaking results highlight the transformative potential of VX-880 therapy, offering new hope for those living with type 1 diabetes. As research progresses, VX-880 could become a vital tool in managing and potentially transforming the treatment landscape for T1D, significantly improving patients' quality of life.