Vertex shares updated data for islet cell therapy in diabetes

Vertex Pharmaceuticals shared updated positive data from the phase 1/2 portion of the phase 1/2/3 FORWARD-101 clinical trial of zimislecel, 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.

The data are from 12 patients who received the full dose of zimislecel as a single infusion and were followed for at least one year, as of October 2024. Results from the study to date continue to demonstrate the transformative potential of zimislecel with consistent and durable patient benefit with longer follow-up.

All patients with at least one year of follow-up who received a full dose of zimislecel as a single infusion achieved ADA-recommended target HbA1c levels <7% and >70% time-in-range, and 10/12 patients were insulin free.

The data were featured in an oral presentation at the American Diabetes Association annual conference in Chicago and published online in the New England Journal of Medicine.

Zimislecel is an investigational allogeneic stem cell-derived, fully differentiated, insulin-producing islet cell therapy manufactured using proprietary technology. It has the potential to restore the body’s ability to regulate glucose levels by restoring pancreatic islet cell function, including glucose-responsive insulin production. Zimislecel is delivered by an infusion into the hepatic portal vein and requires chronic immunosuppressive therapy to protect the islet cells from immune rejection.

Vertex scrapped its other islet cell therapy, VX-264, in March after a phase 1/2 study failed to meet the efficacy endpoint. The treatment, which was also being studied in type 1 diabetes, was encapsulated in a proprietary immunoprotective device. The diabetes cell therapy-device combo fail triggered a round of layoffs, revealed earlier this month.