SAN DIEGO, July 21, 2016 — ViaCyte, Inc., a privately-held regenerative medicine company, today announced that the California Institute for Regenerative Medicine (CIRM) approved a grant of $3.9 million to support pre-clinical development of ViaCyte’s PEC-Direct cell therapy product candidate which is being developed for a subset of type 1 diabetes patients. The PEC-Direct product candidate delivers stem cell-derived PEC-01 pancreatic progenitor cells in a device designed to allow direct vascularization of the cells, and is being developed for type 1 diabetes patients that have severe hypoglycemic episodes, extreme glycemic lability, and/or impaired awareness of hypoglycemia.
“We are grateful to CIRM and the citizens of California for their continued support of our potentially transformative stem cell-derived, cell replacement therapies for diabetes,” said Paul Laikind, Ph.D., President and CEO of ViaCyte. “With PEC-Direct we intend to address a critical unmet medical need for people who are most severely impacted by type 1 diabetes. These funds will support our pre-clinical studies as we prepare to initiate clinical development of PEC-Direct early next year.”
ViaCyte is also actively developing the PEC-EnCap (formerly known as VC-01™) product candidate, which delivers pancreatic progenitor cells in an immunoprotective device. PEC-EnCap is currently being evaluated in a Phase 1/2 trial in patients with type 1 diabetes who have minimal to no insulin-producing beta cell function. PEC-EnCap is the first pluripotent stem cell-derived islet replacement therapy for the treatment of type 1 diabetes in clinical-stage development.
“We have early clinical demonstration of the principle of the PEC-EnCap approach, having shown that engraftment and differentiation to beta cells is feasible based on analyses at twelve weeks post-implant. Optimization efforts are continuing to ensure robust and consistent engraftment with PEC-EnCap before exploring higher doses to demonstrate efficacy,” said Dr. Laikind. “ViaCyte remains committed to the development of PEC-EnCap, which we view as a potentially transformational therapy for the majority of people who use insulin to manage their diabetes. While PEC-Direct development could advance more rapidly and make a major impact for the subset of type 1 diabetes patients at highest risk, we view PEC-EnCap as holding the promise of a functional cure for all patients with type 1 diabetes as well as an important therapy for many patients with type 2 diabetes.”
PEC-Direct delivers the same PEC-01 pancreatic progenitor cells as PEC-EnCap but does so in a device designed to allow direct vascularization. This direct vascularization is expected to allow for a robust engraftment and cellular performance similar to the anatomy of a normal islet. Given the open nature of the device, patients implanted with PEC-Direct, as with other transplants, would require chronic immune suppression. Thus it is being developed to treat patients with type 1 diabetes that are at high risk for acute complications such as severe hypoglycemic episodes, including patients with extreme glycemic lability and/or impaired awareness of hypoglycemia.
“This high-risk patient population is the same population that would be eligible for cadaver islet transplants, a procedure that can be highly effective but suffers from a severe lack of donor material,” said Dr. Laikind. “We believe PEC-Direct could overcome the limitations of islet transplant by providing an unlimited supply of cells, manufactured under cGMP conditions, and a safer, more optimal route of administration.”
For more information about ViaCyte’s clinical trials, please visit: https://clinicaltrials.gov/.
ViaCyte is a privately-held regenerative medicine company developing novel cell replacement therapies as potential long-term diabetes treatments to reduce the risk of hypoglycemia and diabetes-related complications. ViaCyte’s product candidates are based on the derivation of pancreatic progenitor cells, which are then implanted in durable and retrievable delivery devices. Once implanted and matured, these cells are designed to secrete insulin and other regulatory factors in response to blood glucose levels. ViaCyte has two products in development. The PEC-Direct product candidate delivers the pancreatic progenitor cells in a non-immunoprotective device and is being developed for type 1 diabetes patients that have severe hypoglycemic episodes, extreme glycemic lability, and/or impaired awareness of hypoglycemia. The PEC-EnCap (formerly VC-01™) product candidate delivers pancreatic progenitor cells in the Encaptra® immunoprotective device, and is currently being evaluated in a Phase 1/2 trial in patients with type 1 diabetes who have minimal to no insulin-producing beta cell function. 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.