Vertex’s Breakthrough: Remarkable Results Revealed for Stem Cell-Based Type 1 Diabetes Therapy

Vertex Pharmaceuticals has unveiled promising longer-term results from parts A and B of its Phase I/II trial for the investigational stem cell-derived therapy VX-880, which is being evaluated for the treatment of type 1 diabetes (T1D).

The data, presented at the 59th Annual Meeting of the European Association for the Study of Diabetes, included positive outcomes such as insulin independence and the elimination of serious hypoglycemic events.

In this early-stage trial, a third patient achieved insulin independence after receiving Vertex’s VX-880. Two other patients, followed for at least 12 months, met the study’s endpoint by eliminating serious hypoglycemic events between 90 days and 12 months.

These results build on earlier data from June 2023, which reported the first two patients achieving insulin independence and meeting the study’s primary endpoint. The first patient remained insulin-independent for 24 months, having had T1D for nearly 42 years, while the second patient achieved insulin independence for 12 months before briefly resuming insulin treatment at a lower dose.

Additionally, the Phase I/II study found that VX-880 induced islet cell engraftment in all participants, allowing for glucose-responsive insulin production and improved glycemic control.

VX-880 is an investigational allogeneic stem cell-based therapy that delivers fully differentiated and insulin-producing islet cells. It aims to restore glucose-responsive insulin production and improve glucose control when infused via the hepatic portal vein. Patients receiving VX-880 need to be on an immunosuppressive regimen.

Vertex’s T1D program also includes VX-264, another investigational therapy that encapsulates stem cell-derived islet cells in a protective device for implantation into patients. Unlike VX-880, VX-264 is being studied without the use of immunosuppressive therapies, as the device shields the therapeutic cells from the body’s response.

Data from the Phase I/II study of VX-880 holds particular significance for the development of VX-264, as both therapies use the same VX-880 cells as their foundation. This promising progress could potentially lead to innovative treatment options for individuals living with T1D.

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