Investigational Allogeneic Human Stem Cell Therapy for Type 1 Diabetics Awarded FDA Fast Track Designation While Phase 1/2 Clinical Trial Commences

Investigational Allogeneic Human Stem Cell Therapy for Type 1 Diabetics Awarded FDA Fast Track Designation While Phase 12 Clinical Trial Commences

VX-880, formerly known as STx-02, is an investigational allogeneic human stem cell-derived islet cell therapy that is being evaluated for patients who have type 1 diabetes (T1D) with impaired hypoglycemic awareness and severe hypoglycemia. Developed by Massachusetts-based Vertex Pharmaceuticals, VX-880 has the potential to restore the body’s ability to regulate glucose levels by restoring pancreatic islet function, including insulin production. This novel regenerative therapy just received U.S. Food and Drug Administration (FDA) Fast Track Designation while, in parallel, the company has initiated a clinical trial for the study therapy in T1D patients with severe hypoglycemia and impaired hypoglycemic awareness. This particular study involves an infusion of fully differentiated, functional islet cells, as well as the chronic administration of concomitant immunosuppressive therapy to protect the islet cells from immune reaction.

What follows is a brief breakdown of this novel effort.

The Disease

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. The absence of insulin leads to abnormalities in how the body processes nutrients, leading to high blood glucose levels. High blood glucose can lead to diabetic ketoacidosis and over time, to complications such as kidney disease/failure, eye disease (including vision loss), heart disease, stroke, nerve damage and even death.

Due to the limitations and complexities of insulin delivery systems, it can be difficult to achieve and maintain balance in glucose control in patients with T1D. Hypoglycemia often results because of the difficulty in balancing the different factors that impact glucose levels, including insulin, diet and exercise. Hypoglycemia remains a critical limiting factor in glycemic management, and severe hypoglycemia can cause loss of consciousness, coma, seizures, injury, and can be fatal. Over time, patients with T1D can develop impaired awareness of hypoglycemia, meaning they are no longer able to perceive the early signs of a hypoglycemic event, which can be dangerous and result in life threatening events. There are currently limited treatment options beyond insulin for the management of T1D.

Who is behind this research originally?

Initially developed by Dr. Doug Melon’s lab at Semma Therapeutics but the novel therapy has since then been acerated and brought to the clinic by the Vertex team, reports Bastiano Sanna, PhD, Executive Vice President and Chief of Cell and Genetic Therapies at Vertex.

How is this regenerative approach different?

According to Vertex’s Sanna, “Ours is the only approach that produces fully differentiated and fully differentiated and fully functional insulin-secreting pancreatic islets. We are very pleased to have received FDA’s Fast Track Designation, which facilitates the development and expedites the review of drugs that treat serious conditions and fill an unmet medical need. We continue to work with urgency to bring this innovative therapy to patients.”

Camillo Ricordi with University of Miami is a notable stem cell therapy expert and Steering Committee Chair of the VX-880 trial—what’s his point of view?

“It’s a remarkable time for T1D research efforts worldwide, as this investigational treatment enters the clinic,” declared the Professor of Surgery and Director of the Diabetes Research Institute (DRI) and the Cell Transplant Center at the University of Miami Miller School of Medicine. The key opinion leader continued, “The field’s experience with the limited cadaveric islet transplants available, where some patients have experienced prolonged insulin independence for years, provides important proof-of-concept for the potential of cell therapy to be transformative for patients living with T1D.”

The Clinical Trial

The study (NCT04786262) is a Phase 1/2, single-arm, open-label study in patients who have T1D with impaired hypoglycemic awareness and severe hypoglycemia. This will be a sequential, multi-part clinical trial to evaluate the safety and efficacy of different doses of VX-880. Approximately 17 patients will be enrolled in the study.

The first clinical trial sites at the University of Miami Health System, the University of Pennsylvania and Massachusetts General Hospital are open for enrollment, and additional sites will be activated this year. 

About Vertex 

Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) — a rare, life-threatening genetic disease — and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of cell and genetic therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

The company was founded in 1989 in Cambridge, MA, and its global headquarters is now located in Boston’s Innovation District with an international headquarter office in London, UK.

Lead Research/Investigator

Camillo Ricordi, MD, Professor of Surgery and Director of the Diabetes Research Institute (DRI) and the Cell Transplant Center at the University of Miami Miller School of Medicine, University of Miami 

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