Vertex Continues Multiple Paths To Potential Islet Cell Cure
After ending the VX-264 trial, which used a device to protect lab-grown islet cells, the company will continue research on therapies with and without the need for immunosuppressive drugs.
Vertex Pharmaceuticals recently announced the discontinuation of its VX-264 trial, which tested whether lab-grown islet cells, transplanted into a small protective device, could produce insulin in people with type 1 diabetes. The VX-264 trial showed the treatment was safe, but the cells in the device didn't produce enough insulin to continue.
Researchers are analyzing the VX-264 trial results for insights that could guide future strategies for encapsulated transplants. The company says it remains committed to advancing device-based approaches and other strategies, such as alternative forms of immunosuppression treatment.
The company is also researching gene-edited hypoimmune stem-cell derived islet cell therapies, which don't require immunosuppressive drug therapy to protect the cells.
At the same time, the final phase continues for a trial of Vertex’s zimislecel (formerly VX-880), a cell-based therapy that does require immunosuppression to protect the immune system from rejecting the new beta cells. That study is aimed at treating individuals with type 1 diabetes who experience impaired awareness of hypoglycemia and severe hypoglycemic events.
In the zimislecel study, 11 of 12 participants reduced need or complete elimination of the need for insulin, with time in range increasing from 50% at the start of the trial to 90% a year into the study with no severe hypoglycemic episodes observed.
Vertex expects that trial to be fully recruited with 50 people who have type 1 by June 2025, and the company has accelerated its timeline for FDA submission from 2030 to 2026. With FDA fast track designation, zimislecel could become available as early as 2027.
Learn more about the search for a cure to type 1 diabetes: