5 July 2022 - - The US Food and Drug Administration has lifted the clinical hold placed on the Phase 1/2 clinical trial of VX-880, an investigational stem cell-derived, fully differentiated pancreatic islet cell replacement therapy for people with type 1 diabetes with impaired hypoglycemic awareness and severe hypoglycemia, US-based Vertex Pharmaceuticals Inc. (NASDAQ: VRTX) said.

As a result, the Phase 1/2 trial will be reopened for screening, enrollment and dosing at multiple sites in the US.

To date, three patients have been dosed in the Phase 1/2 study with VX-880. Two patients received half the target dose of cells in Part A of the study.

A third patient has received the full target dose in Part B of the study.

Part B will evaluate safety and efficacy in five patients at the target dose before expanding to additional patients in Part C.

VX-880 is an investigational allogeneic stem cell-derived, fully differentiated, insulin-producing islet cell therapy manufactured using proprietary technology.

It is being evaluated for patients who have T1D with impaired hypoglycemic awareness and severe hypoglycemia.

According to the company, VX-880 has the potential to restore the body's ability to regulate glucose levels by restoring pancreatic islet cell function, including glucose responsive insulin production.

VX-880 is delivered by an infusion into the hepatic portal vein and requires maintenance immunosuppressive therapy to protect the islet cells from immune rejection.

The clinical trial is a Phase 1/2, multi-center, single-arm, open-label study in patients who have T1D with impaired hypoglycemic awareness and severe hypoglycemia.

This study is designed as a sequential, multi-part clinical trial to evaluate the safety and efficacy of VX-880.

In Part A, the first two patients received half the target dose. In Part B, five patients will receive the target dose, after which concurrent dosing at the full target dose will occur in Part C. Approximately 17 patients will be enrolled in the clinical trial.

Enrollment is ongoing in this study.

T1D results from the autoimmune destruction of insulin-producing islet cells in the pancreas, leading to loss of insulin production and impairment of blood glucose control.

Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London.