San Diego, California, November 6, 2014 — ViaCyte, Inc., a privately-held regenerative medicine company developing a stem cell-derived islet replacement therapy for the treatment of diabetes, announced today it has been granted U.S. patent 8,859,286, which describes methods for manufacture of human pancreatic progenitor cells from definitive endoderm cells, by the U.S. Patent and Trademark Office. These methods further strengthen ViaCyte’s intellectual property platform, providing alternative ways to produce pancreatic progenitor and endocrine cells that have potential for use in cell replacement therapies.
Pancreatic progenitor cell cultures are precursors to endocrine cells, and are designed to mature into cells found in the normal human islet that produce insulin and other regulatory hormones, after implantation in the patient. ViaCyte’s clinical stage islet replacement therapy, the VC-01™ product candidate, uses a progenitor-containing population of cells designated PEC-01™ cells. The newly-issued patent covers alternative methods for making PEC-01 cells, as well as methods for producing insulin-expressing cells that are closer to fully differentiated beta cells, in vitro.
“This patent further expands our intellectual property platform, which includes over 100 issued and 150 pending patents covering drug delivery devices and human pluripotent stem cells and their derivatives,” said Dr. Paul Laikind, President and CEO of ViaCyte. “The newly-issued patent is important because it covers yet additional methods of creating PEC-01 cells, which resemble the precursor cells that occur in natural human pancreatic development and are integral to our lead product candidate, VC-01. Our Phase 1/2 clinical trial of VC-01 for the treatment of type 1 diabetes is underway and we expect initial data from the first cohort of subjects in this study in early 2015.”
The loss or malfunction of insulin-producing beta cells is the primary cause of type 1 diabetes, and the therapeutic potential of cells that might replace lost or malfunctioning beta cells is tremendous. ViaCyte’s VC-01 product candidate is designed to deliver a therapeutic dose of PEC-01 cells via implantation just under a patient’s skin. In order to protect the implanted cells from immune rejection, the PEC-01 cells are encapsulated in a proprietary device with a selectively porous membrane called the Encaptra® drug delivery system. In addition to providing protection from immune rejection, the Encaptra device is also designed to permanently contain the cells, and in this way prevent their distribution away from the implantation site, as well as to provide a platform for vascularization (growth of blood supply).
Because PEC-01 cells resemble the precursor cells that occur in natural human pancreatic development, ViaCyte believes that they have properties particularly well-suited for use in a cell replacement therapy product. When first implanted under the skin of the patient, the cells must exist in an oxygen-deprived environment and over time need to promote vascularization. Vascularization is critically important to provide oxygen and nutrients to the cells, allowing them to sense blood glucose levels and distribute the insulin and others factors throughout the body. As the PEC-01 pancreatic progenitor cells represent a cell type that is present in a developing embryo, they are designed by nature to function in a hypoxic environment, promote vascularization, and differentiate into the critical endocrine cells that populate the pancreatic islets. Thus, ViaCyte believes that the use of PEC-01 has advantages over the implantation of more fully differentiated cells such as insulin-producing beta cells; however, the company continues to evaluate other approaches, as indicated by this patent.
ViaCyte is conducting a Phase 1/2 clinical trial to evaluate the VC-01 product candidate 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 demonstrate 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.
The newly-issued patent forms part of ViaCyte’s broad intellectual property (IP) portfolio of 100 issued patents and more than 150 pending patent applications around its novel cell therapy and lead product candidate, VC-01. The current patent portfolio covers many of the intermediary cell types generated when pluripotent stem cells progress along the differentiation pathway to pancreatic endoderm cells, including mesendoderm and definitive endoderm. Methods of making the cells and implantable devices, as well as their use, are also protected.
After implant into animals in pre-clinical studies of VC-01, the PEC-01 cells mature into the various human cell types of the endocrine pancreas, including those expressing insulin (INS, blue), glucagon (GCG, red), and somatostatin (SST, green).
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.
Paul Laikind, Ph.D.
President and Chief Executive Officer