Vertex Touts Promising Data for Stem Cell-Based Type 1 Diabetes Treatment

Pictured: Illustration of pancreas/iStock, Rasi Bh

Pictured: Illustration of pancreas/iStock, Rasi Bh

New Phase I/II trial results show that one more type 1 diabetes patient achieved insulin independence after treatment with Vertex Pharmaceuticals’ investigational stem cell therapy VX-880.

Pictured: Illustration of pancreas/iStock, Rasi Bhadramani

Vertex Pharmaceuticals on Tuesday posted longer-term results from parts A and B of its Phase I/II trial of the investigational stem cell-derived therapy VX-880, being assessed for type 1 diabetes.

The early-stage data, presented at an oral session during the 59th Annual Meeting of the European Association for the Study of Diabetes, showed that a third patient reached insulin independence after being treated with Vertex’s candidate.

Two patients who had been followed for at least 12 months likewise met the study’s endpoint of the elimination of serious hypoglycemic events (SHE) between 90 days and 12 months.

Tuesday’s results follow an earlier data drop in June 2023, detailing the first two patients that achieved insulin independence and met the study’s primary endpoint.

The first patient achieved insulin independence 270 days into treatment, lasting through month 24 of the follow-up. The patient had type 1 diabetes (T1D) for “nearly 42 years,” according to Vertex’s announcement, and was taking 34 units of insulin daily.

Insulin independence came on day 180 for the second patient, persisting through 12 months of follow-up. The patient had T1D for 19 years and was taking 45.1 units of exogenous insulin daily. This patient had to restart insulin treatment at month 15, though at a much lower daily dose of four units.

The third patient stopped needing insulin at 180 days of treatment, which happened after the data cut-off, according to Vertex.

Aside from insulin independence, the new data from the Phase I/II study showed that VX-880 induced islet cell engraftment in all participants in parts A and B of the study at 90 days. In turn, these patients are now capable of endogenous glucose-responsive insulin production and demonstrated better glycemic control across various measures, including HbA1c and time-in-range.

Before receiving VX-880, all enrolled participants had long-standing T1D and showed no signs of endogenous insulin secretion and required 34.0 units of insulin per day on average, according to Vertex’s announcement on Tuesday. All patients also had histories of recurrent severe hypoglycemic events.

An investigational allogeneic stem cell-based therapeutic, VX-880 works by delivering fully differentiated and insulin-producing islet cells, in turn restoring the body’s glucose-responsive insulin production capabilities and boosting glucose control. VX-880 is designed to be delivered via an infusion through the hepatic portal vein. Patients need to be on an immunosuppressive regimen to receive the candidate.

Vertex’s T1D program also includes VX-264, an investigational therapy that encapsulates stem cell-derived islet cells in a protective device to be implanted into the patients’ bodies, according to the company’s website. Because the device is designed to shield the therapeutic cells from the body’s response, VX-264 is being studied without the use of immunosuppressive therapies.

Data from the Phase I/II study of VS-880 is also “particularly meaningful” for the development of VS-264, because “these same VX-880 cells are the foundation for our VX-264 cells-plus-device program,” Vertex CMO Carmen Bozic said in a statement on Tuesday.

Tristan Manalac is an independent science writer based in Metro Manila, Philippines. He can be reached at or

Tristan is an independent science writer based in Metro Manila, with more than eight years of experience writing about medicine, biotech and science. He can be reached at, or on LinkedIn.