ViaCyte, Inc., a privately-held regenerative medicine company, 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.”

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.