SAN DIEGO, Oct. 29, 2014 /PRNewswire/ — ViaCyte, Inc., a privately-held regenerative medicine company, announced today that the first patient in its Phase 1/2 study was successfully implanted with VC-01™, its embryonic stem cell-derived islet replacement product candidate being developed as a treatment for type 1 diabetes. This Phase 1/2 clinical trial, designed to evaluate the VC-01 product candidate directly in patients with type 1 diabetes, is initially being conducted at UC San Diego Health System, with the support of the UC San Diego Sanford Stem Cell Clinical Center, under the direction of Principal Investigator Robert Henry, MD.
Dr. Paul Laikind, President and CEO of ViaCyte, said, “We are very excited to begin the clinical stage of development in our quest to transform the way patients with type 1 diabetes are impacted by the disease. To our knowledge, this is the first time that an embryonic stem cell-derived cell replacement therapy for diabetes has been studied in human subjects, and it represents the culmination of a decade of effort by the ViaCyte team, our collaborators, and our supporters at the California Institute for Regenerative Medicine and at JDRF.”
The loss or malfunction of insulin-producing beta cells is the primary cause of type 1 diabetes, making it a promising target disease for cell replacement therapy. ViaCyte’s VC-01 product candidate delivers pancreatic precursor cells (called PEC-01™ cells) that are designed to further differentiate and mature after surgical implantation, not only to fully functioning insulin-producing beta cells, but also to other endocrine cell types that make up the normal human pancreatic islet. Thus, along with the critically important insulin, the implanted cells are expected to produce other hormones that are important for the regulation of glucose (sugar) in the blood, including, for example, glucagon, somatostatin, and amylin.
The PEC-01 cells are delivered under the skin in a proprietary device with a selectively porous cell-impermeable membrane, called the Encaptra® drug delivery system. The Encaptra device is designed to protect the implanted cells from possible immune rejection, to permanently contain the cells and prevent their distribution away from the implantation site, and to provide a platform for product vascularization.
The ongoing Phase 1/2 clinical trial is evaluating the VC-01 product candidate directly in patients with type 1 diabetes who have minimal to no insulin-producing beta cell function. In addition to determining the safety of the product candidate in these patients, the study is designed to evaluate the effectiveness of the VC-01 product candidate in replacing the lost endocrine function that is central to the disease. In an open-label, dose-escalating format, ViaCyte expects to enroll approximately 40 patients in the study at multiple clinical sites.
About Type 1 Diabetes
Type 1 diabetes mellitus (previously called juvenile diabetes) is a life-threatening chronic condition in which the pancreas produces little or no insulin, a hormone needed to allow glucose to enter cells to produce energy. It is typically diagnosed during childhood or adolescence, though it can also arise in adults. Though less common than type 2 diabetes, which occurs when the body becomes resistant to insulin, type 1 diabetes affects several million Americans, according to JDRF, the leading global organization focused on type 1 diabetes research. Currently, there is no cure for type 1 diabetes and the risk of long-term complications is high even with diligent treatment. Standard treatment involves multiple daily injections of insulin and rigorous management of diet and lifestyle.
ViaCyte is a privately-held regenerative medicine company focused on developing a novel cell therapy for the treatment of diabetes. ViaCyte’s lead product candidate, the 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. VC-01 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 and JDRF. For more information, please visit www.viacyte.com.
About Sanford Stem Cell Clinical Center at UCSD
The Sanford Stem Cell Clinical Center was recently created to advance leading-edge stem cell medicine and science, protect and counsel patients, and accelerate innovative stem cell research into patient diagnostics and therapies.
Paul Laikind, Ph.D.
President and Chief Executive Officer
In-Site Communications, Inc.