Vertex Pharmaceuticals reports data from Phase 1/2 clinical trial of VX-880

Vertex Pharmaceuticals presented longer-term data on patients dosed in Parts A and B of its Phase 1/2 clinical trial of VX-880, an investigational stem cell-derived, fully differentiated islet cell therapy in people with type 1 diabetes, or T1D, with impaired hypoglycemic awareness and severe hypoglycemic events, or SHEs. Prior to VX-880 treatment, all six patients enrolled had long-standing T1D with no endogenous insulin secretion, required an average of 34.0 units of insulin per day, and had a history of recurrent severe hypoglycemic events in the year prior to screening. All patients in Part A and B now have more than 90 days of follow-up and have demonstrated islet cell engraftment and endogenous glucose-responsive insulin production on the Day 90 mixed-meal tolerance test, or MMTT. All patients demonstrated improved glycemic control across all measures, including decreases in HbA1c, improved time-in-range on continuous insulin monitoring, and reduction or elimination of exogenous insulin use. The two patients with at least 12 months of follow-up after VX-880 infusion, who were therefore evaluable for the study’s primary efficacy endpoint, met the criteria for the primary endpoint of elimination of SHEs between Day 90 and Month 12 with an HbA1c less than7.0%. The first patient achieved insulin independence at Day 270 through Month 24. This is a patient who has had T1D for nearly 42 years and prior to trial enrollment was on 34 units of daily exogenous insulin. The second patient achieved insulin independence at Day 180 through Month 12. VX-880 has been generally well tolerated in all patients dosed to date. The majority of adverse events, or AEs, were mild or moderate, and there were no serious AEs related to VX-880 treatment. As previously reported, one subject had SHEs in the perioperative period. There have been no other SHEs in the study.