SAN DIEGO, Aug. 19, 2014 /PRNewswire/ -- ViaCyte, Inc., a privately-held regenerative medicine company, announced today that the U.S. Food and Drug Administration ("FDA") has accepted the company's Investigational New Drug Application ("IND") for its VC-01™ candidate cell replacement therapy to treat type 1 diabetes. The company plans to promptly initiate a Phase 1/2 clinical trial which it believes to be the first ever clinical evaluation of a stem cell-derived islet replacement therapy for the treatment of patients with type 1 diabetes.
"We are pleased to have received FDA acceptance for our clinical trial protocol and look forward to initiating this study shortly" said Paul Laikind, Ph.D., President and Chief Executive Officer of ViaCyte. "The commencement of this clinical trial marks a significant milestone for ViaCyte, as we begin studying the use of the VC-01 product candidate for the treatment of type 1 diabetes. Instrumental in our ability to achieve this milestone are JDRF, the leading global organization focused on type 1 diabetes (T1D) research, and the California Institute for Regenerative Medicine, a leading organization focused on advancing stem cell research and regenerative medicine. Both organizations have been strong supporters of the work we are doing at ViaCyte."
ViaCyte's VC-01 product candidate consists of pancreatic progenitor cells, called PEC-01™ cells, which are derived from a proprietary human embryonic stem cell line, encapsulated in ViaCyte's proprietary Encaptra® device. When implanted under the skin, the PEC-01 cells are specifically designed to mature and further differentiate into insulin-producing beta and other endocrine cells that regulate blood glucose in a manner similar or identical to the islets that normally comprise the endocrine pancreas.
The Phase 1/2 clinical study will evaluate 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 evaluating 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.
A primary goal of this first clinical trial will be to assess the safety and tolerance of the VC-01 product candidate in patients with type 1 diabetes. In addition, insulin production in the study subjects will be evaluated by measuring C-peptide, a biomarker for insulin produced by beta cells that is expected to provide a sensitive measure of efficacy in these patients. As proposed, the trial would also evaluate clinically-relevant secondary endpoints related to the need for administration of pharmaceutical insulin to control type 1 diabetes and the incidence of hypoglycemia, a common side effect associated with pharmaceutical insulin usage.
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 begin in adults. Though far less common than type 2 diabetes, which occurs when the body becomes resistant to insulin, type 1 diabetes affects several million Americans, according to the JDRF. Currently, there is no cure for type 1 diabetes. 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. The VC-01 combination 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 and JDRF.