ViaCyte Announces Highly Anticipated Encapsulation Clinical Trial Site Expansion Into Canada

TORONTO, ON--(Marketwired - July 29, 2015) - ViaCyte, Inc. announced the opening of a second site in its Phase 1/2 trial called STEP ONE, or Safety, Tolerability and Efficacy of VC-01™ Combination Product in Type One Diabetes (T1D), at the University of Alberta Hospital in Edmonton, Alberta funded in part by the JDRF Canadian Clinical Trial Network (CCTN). The VC-01™ product candidate, developed by ViaCyte, utilizes PEC-01™ pancreatic progenitor cells and the proprietary Encaptra® drug delivery system which is designed to protect the transplanted cells from a patient's immune system.

"This trial is the first of its kind in Canada and holds out the promise of a big step forward for people living with T1D," said Dave Prowten, President and CEO, JDRF Canada, "The outcome of this trial could transform the daily regimen and lifestyle for T1D patients. Imagine being able to avoid dangerous blood sugar highs and lows without monitoring your blood glucose or taking insulin injections."

The encapsulation device holds pancreatic progenitor cells that are produced by a proprietary method from a stem cell line, and together the combination product candidate is intended to address the current limitations of islet transplantation. The device will be implanted under the skin of the patient in an outpatient surgical procedure. The cells are then expected to develop into mature pancreatic endocrine cells that will secrete insulin and other factors, thereby regulating blood sugar levels. If effective, this therapy could reduce or even eliminate the need for insulin injections.

The lead Edmonton investigator is JDRF CCTN-funded researcher, Dr. James Shapiro. Dr. Shapiro's laboratory developed the Edmonton Protocol for the transplant of pancreatic islets as a treatment for T1D. He also holds a Canada Research Chair in Transplant Surgery and Regenerative Medicine.

Dr. Shapiro is renowned for determining how to administer human islets, resulting in insulin independence for many diabetes patients. Dr. Shapiro says, "This is a remarkable opportunity that could free T1D patients from severe complications and health issues such as hypoglycemia, eye, kidney and cardiovascular diseases, all without the requirement of powerful lifelong immunosuppression drugs."

"JDRF has been a longtime supporter of the important work we are doing towards a transformative therapy for patients with type 1 diabetes," said Dr. Paul Laikind, President and CEO of ViaCyte. "With their help we have now moved VC-01™ into clinical testing in both the US and Canada. This is a critical step, and while there is still much work to do, we are excited by the progress and the potential of our efforts."

The STEP ONE trial, launched in 2014 through the University of California San Diego Health System, calls for the evaluation of the product candidate in two groups, or cohorts of T1D patients. Assuming success with the first cohort of patients that are currently being studied, both cohorts are expected to be evaluated at the Canadian clinical trial site.

Cohort 1 patients are receiving a low, sub-therapeutic dose of cells to test the safety of VC-01™ product candidate in the human body to determine whether the body will accept the device and encapsulated cells, to optimize the procedural aspects of administration and to determine if the encapsulation device is an effective barrier to the human immune system.

Assuming the safety is demonstrated, and the procedure is optimized with Cohort 1, the trial will progress to the second group of patients who will receive a higher dose of cells, calculated to provide therapeutic production of insulin and other regulatory proteins, allowing further evaluation of safety and an initial evaluation of dosing and device performance.

The STEP ONE clinical trials in Edmonton are also being supported by a Collaborative Research and Innovation Opportunities (CRIO) grant from Alberta Innovates - Health Solutions (AIHS).

About JDRF Canada

JDRF is the leading global organization funding type 1 diabetes (T1D) research. JDRF's goal is to progressively remove the impact of T1D from people's lives until we achieve a world without T1D. JDRF collaborates with a wide spectrum of partners and is the only organization with the scientific resources, regulatory influence, and a working plan to better treat, prevent, and eventually cure T1D. As the largest charitable supporter of T1D research, JDRF is currently sponsoring $530 million in scientific research in 17 countries. For more information, please visit jdrf.ca.

About JDRF Canadian Clinical Trial Network

The JDRF Canadian Clinical Trial Network (JDRF CCTN) is an initiative to accelerate solutions for the management, care, and cure of type 1 diabetes (T1D). The JDRF CCTN creates a strong clinical research network to develop and conduct leading-edge clinical trials in T1D and its complications, in order to accelerate delivery of the benefits of research advances to the community. Through funding from the Canadian Federal Government, JDRF and the WB Family Foundation, $36.9 million has been invested to accelerate the testing of new technologies and treatments benefiting Canadians and individuals around the world living with T1D and its complications. For more information, visit jdrf.ca/cctn.

About ViaCyte

ViaCyte is a privately-held regenerative medicine company focused on developing a novel cell therapy for the treatment of diabetes. ViaCyte is conducting a Phase 1/2 clinical trial of the Company's lead VC-01™ product candidate in patients with type 1 diabetes who have minimal to no insulin-producing beta cell function. ViaCyte's VC-01™ combination product is based on the production of pancreatic progenitor cells derived from human pluripotent stem cells. These progenitor cells are implanted in a durable and retrievable encapsulation device. Once implanted and matured, these cells are designed to secrete insulin and other regulatory factors in response to blood glucose levels. The VC-01™ product is being developed as a potential long-term diabetes treatment without immune suppression, and without risk of hypoglycemia or other diabetes-related complications.

ViaCyte is headquartered in San Diego, California with additional operations in Athens, Georgia. The Company is funded in part by the California Institute for Regenerative Medicine (CIRM) and JDRF.