A year and a half after betting $950 million on Semma Therapeutics and its stem cell-based diabetes treatments, Vertex is ushering one of those programs into the clinic and grabbing a fast-track tag from the FDA to boot.
Vertex is testing the treatment, VX-880, in patients who have Type 1 diabetes with severe hypoglycemia and impaired hypoglycemic awareness—that is, patients whose blood sugar drops to very low levels without showing symptoms that would otherwise warn them that their blood sugar is falling.
Previously known as STx-02, the treatment is designed to restore the body’s ability to regulate glucose by giving patients new insulin-producing pancreatic islet cells based on donor stem cells. The hope is to replace the insulin injections and chronic care that are the norm for people with Type 1 diabetes.
“This program has its roots in the groundbreaking work that began in Dr. Doug Melton’s lab, progressed at Semma Therapeutics, and has been accelerated and brought to the clinic by the team at Vertex,” said Bastiano Sanna, Ph.D., chief of cell and genetic therapies at Vertex, in a statement. Sanna was formerly the CEO of Semma Therapeutics.
The phase 1/2 study will enroll about 17 patients, who will receive VX-880 along with immunosuppressive therapy to prevent immune rejection of the cell transplant.
Along with this traditional immunosuppressive approach, Vertex is also working on a device originally developed at Semma that could free patients from lifelong immunosuppression. The device is designed to deliver and protect the transplanted cells from a patient’s immune system.