Vertex Presents Positive, Updated VX-880 Results From Ongoing Phase 1/2 Study in Type 1 Diabetes at the European Association


BOSTON--(BUSINESS WIRE)--Oct. 3, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today 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 (T1D) with impaired hypoglycemic awareness and severe hypoglycemic events (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 (SHEs) 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 (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 <7.0%. 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. This is a patient who has had T1D for 19 years and prior to trial enrollment was on 45.1 units of daily exogenous insulin. Starting at Month 15, this patient was started on four units of basal insulin daily, at the investigator’s discretion. After the data cut-off date, a third patient achieved insulin independence at Day 180.

VX-880 has been generally well tolerated in all patients dosed to date. The majority of adverse events (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.

“We continue to marvel at the impressive data from the VX-880 program as evidenced by the improvements in all treated patients across all glycemic measures,” said Trevor Reichman, M.D., Department of Surgery, University of Toronto. “This represents an incredibly promising investigational therapy, one with far-reaching potential.”

“These data are particularly meaningful in the context of our overall investigational T1D program, as these same VX-880 cells are the foundation for our VX-264 cells-plus-device program, and our hypoimmune islet cell program,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex. “We are moving with urgency to bring these potentially transformative therapies to patients who are waiting.”

These data were presented during the European Association for the Study of Diabetes 59th Annual Meeting on October 3, 2023, in Hamburg, Germany as an oral presentation, “Glucose-Dependent Insulin Production and Insulin-Independence in Patients with Type 1 Diabetes Infused with Stem Cell-Derived, Fully Differentiated Islet Cells (VX-880)” (abstract/publication #449).

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