ViaCyte to Present Preliminary PEC-Direct Clinical Data at Cell & Gene Meeting on the Mesa

First demonstration of insulin production in patients from a stem cell-derived islet replacement therapy

SAN DIEGO, October 3, 2019ViaCyte, Inc., a privately-held regenerative medicine company, will present preliminary data today from the company’s PEC-Direct clinical trial at the Cell & Gene Meeting on the Mesa taking place in Carlsbad, California. While optimization of the procedure is continuing, preliminary data show that implanted cells, when effectively engrafted, are capable of producing circulating C-peptide, a biomarker for insulin, in patients with type 1 diabetes.

“ViaCyte is the first and only company in human clinical trials with a stem cell-derived islet replacement therapy candidate, and we are now the first to demonstrate production of C-peptide in patients receiving implanted stem cell-derived islets. These data show that our PEC-01 cells are functioning as intended when appropriately engrafted,” said Paul Laikind, Ph.D., Chief Executive Officer and President of ViaCyte. “While there is still more work to be done, this is an important milestone. We plan to present additional data in the near future.”

PEC-01 cells are pancreatic precursor cells derived from ViaCyte’s proprietary CyT49 pluripotent stem cell line. PEC-01 cells are implanted in both the PEC-Direct and PEC-Encap product candidates, and gene-edited immune-evasive PEC-01 cells are being developed for the PEC-QT program in collaboration with CRISPR Therapeutics. The data show initial detection of C-peptide and insulin production through histological and biochemical measurements at multiple time points in multiple patients; detection of C-peptide has correlated with engraftment efficiency. C-peptide is a short chain of amino acids that is co-produced in the formation of insulin by the pancreas and is co-released into the blood proportionally to insulin. It is a standard biomarker in the field for assessing the amount of functional insulin-producing pancreatic beta cells, used because its measurement is not confounded by injected insulin. The entry criteria of the study require patients to be C-peptide-negative upon enrollment. Previous histological data have shown that, after PEC-01 cells are implanted under the skin and when properly engrafted, they can mature into functional beta cells and other cells of the islet that are responsible for controlling blood glucose levels.

Laikind concluded, “ViaCyte has achieved a number of firsts in this field. Now with the first demonstration of insulin production in patients who have received PEC-Direct, we are confident we can be the first to deliver an effective stem cell-derived islet replacement therapy for type 1 diabetes.”

The presentation will take place at 1:45 pm today in the Cognate Services Ballroom at the Park Hyatt Aviara Resort.

About ViaCyte

ViaCyte is a privately held regenerative medicine company developing novel cell replacement therapies as potential long-term diabetes treatments to achieve glucose control targets and reduce the risk of hypoglycemia and diabetes-related complications. ViaCyte’s product candidates are based on directed differentiation of pluripotent stem cells into pancreatic islet progenitor cells, which are then implanted in durable and retrievable cell delivery devices. Over a decade ago, ViaCyte scientists were the first to report on the production of pancreatic cells from a stem cell starting point and the first to demonstrate in an animal model of diabetes that, once implanted and matured, these cells secrete insulin and other pancreatic hormones in response to blood glucose levels, and can be curative.  ViaCyte has two product candidates in clinical-stage development. The PEC-Direct™ product candidate delivers the pancreatic islet progenitor cells in a non-immunoprotective device and is being developed for type 1 diabetes patients who have hypoglycemia unawareness, extreme glycemic lability, and/or recurrent severe hypoglycemic episodes. The PEC-Encap™ (also known as VC-01) product candidate delivers the same pancreatic islet progenitor cells in an immunoprotective device and is being developed for all patients with diabetes, type 1 and type 2, who use insulin. ViaCyte is also developing immune-evasive stem cell lines, from its proprietary CyT49 cell line, which have the potential to further broaden the availability of cell therapy for diabetes and other potential indications. To accelerate and expand its efforts, ViaCyte has established collaborative partnerships with leading companies including CRISPR Therapeutics and W.L. Gore & Associates.  ViaCyte is headquartered in San Diego, California. ViaCyte is funded in part by the California Institute for Regenerative Medicine (CIRM) and JDRF.