Vertex Pharmaceuticals (Nasdaq:VRTX) announced today that the FDA lifted the clinical hold placed on the Phase 1/2 trial of VX-880.
Boston-based Vertex designed VX-880 as a stem cell-derived fully differentiated pancreatic islet cell replacement therapy for people with type 1 diabetes with impaired hypoglycemic awareness and severe hypoglycemia. It could potentially restore the body’s ability to regulate glucose levels by restoring pancreatic islet cell function, including glucose-responsive insulin production.
Delivered by an infusion into the hepatic portal vein, VX-880 requires maintenance immunosuppressive therapy to protect the islet cells from immune rejection.
According to a news release, the Phase 1/2 trial will be reopened for screening, enrollment and dosing at multiple sites in the U.S.
To date, three patients have been dosed in the trial, with two receiving half the target dose of cells in “Part A” of the study. A third patient received the full target dose in “Part B,” which will evaluate safety and efficacy in five patients at the target dose before expanding to additional patients in “Part C.”
Vertex designed its multi-center, single-arm, open-label study as a sequential, multi-part clinical trial to evaluate the safety and efficacy of VX-880. Approximately 17 patients will be enrolled in the trial.
