Our Pipeline for
Insulin-Requiring Diabetes

Multiple therapeutic approaches

ViaCyte is developing islet cell replacement therapy for insulin-requiring diabetes. Our two products in clinical research deploy the same active component, PEC-01 cells, but with different implantation technologies and addressing the unmet needs of different patient groups.

About Type 1 Diabetes

Type 1 diabetes is an autoimmune disease where a person’s own immune cells mistakenly attack and destroy the insulin-producing beta cells in the pancreas. Without beta cells, the body can no longer produce insulin, a hormone which controls blood glucose (sugar) levels. If not controlled by insulin, high blood sugar can be life-threatening. While donor (cadaver) islet transplants can be highly effective in treating type 1 diabetes, the procedure is only performed on a limited number of patients because of a severe lack of donor material and the requirement for immunosuppression. To deliver a functional cure for all patients with type 1 diabetes and an important new therapy for patients with type 2 diabetes who require insulin, ViaCyte is developing product candidates with numerous potential advantages, including a virtually unlimited supply of cells manufactured under quality-controlled conditions, and a potentially safer and more optimal route of administration.

ViaCyte’s Product Pipeline

PEC-Direct (VC-02)

ViaCyte’s PEC-Direct product candidate is in clinical development for patients with type 1 diabetes with the highest risk of life-threatening acute complications. For PEC-Direct, the pouch is designed to allow blood vessels to enter the device and directly interact with the implanted PEC-01 cells. The direct vascularization of the implanted cells is intended to allow for robust and consistent engraftment but will necessitate the use of immune suppression therapy because the implanted cells are not hidden from the immune system. Patients who receive PEC-Direct must take immunosuppressants to protect the cells, making it suitable only for people with high-risk type 1 diabetes.

The PEC-Direct product candidate is being developed for type 1 diabetes patients who have hypoglycemia unawareness and/or extreme glycemic lability. It is estimated that about 140,000 people in the United States and Canada have high-risk type 1 diabetes. In addition to a potentially unlimited supply of cells for implantation, the PEC-Direct approach has other potential advantages relative to cadaver islet transplants such as delivering a more consistent product preparation under quality-controlled cGMP conditions and a more straightforward and potentially safer mode of administration.

The PEC-Direct product candidate is in clinical development for patients with high-risk type 1 diabetes.

PEC-Encap (VC-01)

ViaCyte is developing the PEC-Encap product candidate as a functional cure for type 1 diabetes. For PEC-Encap, the pouch is designed to fully contain the implanted cells but still allow vital nutrients and proteins, such as oxygen, glucose, insulin, and other hormones, to travel between the cells inside the device and the blood vessels, which grow along the outside of the device. This device is also designed to prevent immune cells from directly contacting the implanted cells so they may thrive and function without provoking an immune response or being destroyed. In the case of PEC-Encap, the Encaptra® system has generally prevented immune rejection and immune sensitization and effectively protects implanted cells from the patient’s adaptive immune system.

Approximately 1.25 million people in the United States have type 1 diabetes (statistics from JDRF). Worldwide, an estimated 42 million people have type 1 diabetes (~10% of 420 million cases of all forms of diabetes, IDF).

The PEC-Encap product candidate is also in clinical trials and is being developed as a functional cure for patients with type 1 diabetes.

PEC-QT (VCTX210)

For PEC-QT, ViaCyte’s proprietary CyT49 pluripotent human stem cell line will be specifically engineered to avoid destruction by the patient’s immune system, potentially eliminating the need for immunosuppressants. The cell line will be differentiated into pancreatic endoderm cells, which will likely be housed in the same type of device used for PEC-Direct, which allows direct interaction between blood vessels and the implanted cells, but because the cells are being designed to be immune-evasive, we would not expect them to be rejected by the immune system. ViaCyte and CRISPR Therapeutics formed a partnership in 2018 to discover, develop, and commercialize gene-edited allogeneic stem cell-derived therapies which could be a next-generation functional cure for all insulin-requiring type 1 and type 2 diabetes.

The PEC-QT program is partnered with CRISPR Therapeutics to develop a next-generation therapy for all insulin-requiring diabetes.

The PEC-QT product candidate is in preclinical development and could be a next generation treatment for all insulin-requiring diabetes.

ViaCyte’s PEC-01 Cells Become Functional Islet Cells as Intended

Preliminary clinical results have shown that, following implant and when effectively engrafted, ViaCyte’s PEC-01 pancreatic progenitor cells mature into human islet tissue, including glucose-responsive insulin-secreting beta cells and other cells of the islet responsible for regulating blood glucose. In clinical trials to date, ViaCyte’s product candidates have been well-tolerated with minimal product-related side effects. Clinical evidence, both histological and measurements of C-peptide (insulin) production, shows that PEC-01 cells are functioning as intended when appropriately engrafted. PEC-01 cells are the active drug substance common to both PEC-Direct and PEC-Encap product candidates, as well as the PEC-QT project.

At ADA 2018, data were presented from the PEC-Encap clinical trial showing that differentiation into endocrine islet cells was observed in two-year explants in regions where there was good host tissue integration and vascularization.

ViaCyte’s Product Candidates Have the Potential to Overcome Donor Islet Transplant Hurdles

While donor (cadaver) islet transplants can be highly effective in treating type 1 diabetes, the procedure is only performed on a limited number of patients because of a severe lack of donor material and the requirement for immunosuppression.

To deliver a functional cure for all patients with type 1 diabetes and an important new therapy for patients with type 2 diabetes who require insulin, ViaCyte is developing product candidates with numerous potential advantages, including a virtually unlimited supply of cells manufactured under quality-controlled conditions, and a potentially safer and more optimal route of administration.

For the PEC-Direct product candidate, the direct vascularization of the implanted cells is intended to allow for robust and consistent engraftment but will necessitate the use of immune suppression therapy, making it suitable only for people with high-risk type 1 diabetes.

Islet transplants have been used to successfully treat patients with unstable, high-risk type 1 diabetes, but the procedure has limitations, including a very limited supply of donor organs and challenges in obtaining reliable and consistent islet preparations,” said trial investigator James Shapiro, MD, PhD, FRCSC, Director of the Clinical Islet Transplant Program, University of Alberta. “An effective stem cell-derived islet replacement therapy would solve these issues and has the potential to help a greater number of people. ”

About Our Development Approach

ViaCyte is currently in clinical trials with PEC-Direct and PEC-Encap. By pursuing multiple therapeutic approaches in parallel, ViaCyte has the potential to accelerate successful clinical outcomes.

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Our Pipeline for Insulin-Requiring Diabetes