Phase 1/2 Data Show Potential of VX-880 for Insulin Independence in T1D

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New results from the ongoing clinical trial of VX-880 suggested all patients with T1D treated with the novel stem cell-derived islet cell therapy showed restored endogenous insulin secretion and improved glycemic control.

Updated findings from the ongoing, phase 1/2 clinical trial investigating VX-880 continued to show the potential of the novel stem cell-derived islet cell therapy as a future treatment option for patients with type 1 diabetes (T1D).1

The data, presented at the 83rd Scientific Sessions of the American Diabetes Association (ADA 2023), showed all 6 patients treated with VX-880 demonstrated restored insulin secretion, improved glycemic control and time-in-range, reduction or elimination of exogenous insulin usage, and complete absence of severe hypoglycemic events in an evaluation period.

“These new findings demonstrate the potential of stem cell-derived islets as a future treatment for patients with type 1 diabetes, signaling a new era that could potentially remove the need for exogenously administered insulin to achieve glycemic control,” Trevor W. Reichman, MD, PhD, surgical director of pancreas and islet cell transplantation at Ajmera Transplant Center at the University of Toronto, said in a statement. “We are hopeful that this first-of-its-kind research could be a game changer for the treatment of type 1 diabetes.”

Common in those with T1D, hypoglycemia occurs due to imbalances in insulin administration relative to an individual’s requirements at a particular time that can be affected by various factors, such as diet and sleep. People with T1D can lose awareness of hypoglycemia and could experience severe hypoglycemic events as a result, which can present with loss of consciousness, seizure, coma, or serious injury. Standards of care do not address the underlying cause of the disease and there are limited treatment options beyond exogenous insulin for T1D management.2

The patient population of the current analysis included adults with T1D and impaired hypoglycemic awareness and severe hypoglycemia. All patients treated with VX-880 had undetectable insulin secretion and a history of recurrent severe hypoglycemic events in the year prior to treatment. The study’s primary efficacy endpoint was the elimination of severe hypoglycemic events between Day 90 and Month 12 with an HbA1c of <7%.

In the study, 2 patients treated with VX-880 (1 in Part A who received half the target dose and 1 in Part B who received the full target dose) were treated for ≥12 months of follow-up and were evaluable for the study’s primary efficacy endpoint. Both patients treated for ≥12 months were insulin independent. According to the analysis, Patient A1 had HbA1c of 5.3% at Month 21 (8.6% at baseline) and Patient B1 had HbA1c of 6.0% at Month 12 (7.6% at baseline).

Investigators noted this level of glucose control is unusual in patients with T1D treated with exogenous insulin, as recent data suggests that approximately 25% of people with T1D meet the recommended HbA1c target (7.0%). Moreover, both patients showed HbA1c levels below the diagnostic threshold for diabetes (6.5%) and over 95% time-in-range, above the recommended target of 70%.

For the 3 patients in Part B, each patient was administered the full target dose of VX-880 as a single infusion, had up to 90 days of follow-up, and additionally showed insulin production, reduction in HbA1c, time-in-range improvements, and reductions in daily insulin usage. The trajectory was considered consistent with that of those observed in 2 patients with ≥1 year of follow-up at equivalent periods of follow-up after VX-880 infusion.

Regarding safety signals, VX-880 is reported to be generally safe and well-tolerated in all patients dosed, with a profile consistent with immunosuppressant therapy and cadaveric islet cell transplantation. Most adverse events were considered mild or moderate and there were no serious adverse events reported related to VX-880 treatment.

Due to positive safety and efficacy data in Parts A and B, an independent committee has recommended moving to Part C of the trial. Part C will allow for concurrent dosing of patients at the full target dose of VX-880. The trial will expand to sites in Norway, Switzerland, and the Netherlands.


  1. Novel stem cell-derived islet cell therapy continues to show promise for achieving insulin independence for individuals with type 1 diabetes. Novel Stem Cell-Derived Islet Cell Therapy Continues to Show Promise for Achieving Insulin Independence for Individuals with Type 1 Diabetes | ADA. June 23, 2023. Accessed June 24, 2023.
  2. Hypoglycemia (low blood glucose). Hypoglycemia (Low Blood Glucose) | ADA. Accessed June 24, 2023.