Viacyte, privately-held, leading regenerative medicine company, announced today that the first patients have been implanted with the PEC-Direct™ product candidate, a novel islet cell replacement therapy in development as a functional cure for patients with type 1 diabetes who are at high risk for acute life-threatening complications. The first implant procedures of the clinical trial took place at the University of Alberta Hospital in Edmonton, Alberta, and the UC San Diego School of Medicine’s Altman Clinical Trials Research Institute. The goal of the open-label clinical trial is to evaluate the PEC-Direct product candidate for safety and definitive evidence of efficacy. In the coming months, the company expects to expand the trial to additional centers including the University of Minnesota and other sites in the US and Canada.
The first cohort of type 1 diabetes patients is receiving multiple small-format cell-filled devices called sentinels in order to evaluate safety and implant viability. These sentinel units will be removed at specific time points and examined histologically to provide early insight into the progression of engraftment and maturation into pancreatic islet cells including insulin-producing beta cells. A second cohort of up to 40 patients is expected to begin enrolling later this year to evaluate both safety and efficacy. The primary efficacy measurement in the trial will be the clinically relevant production of insulin, as measured by the insulin biomarker C-peptide, in a patient population that has little to no ability to produce endogenous insulin at the time of enrollment. Other important endpoints will be evaluated including injectable insulin usage and the incidence of hypoglycemic events. ViaCyte’s goal is to demonstrate early evidence of efficacy in the first half of 2018 and definitive efficacy 6 to 12 months later.
“Islet transplants have been used to successfully treat patients with unstable, high-risk type 1 diabetes, but the procedure has limitations, including a very limited supply of donor organs and challenges in obtaining reliable and consistent islet preparations,” said trial investigator James Shapiro, MD, PhD, FRCSC, Director of the Clinical Islet Transplant Program, University of Alberta. “An effective stem cell-derived islet replacement therapy would solve these issues and has the potential to help a greater number of people.”
“Patients with high-risk type 1 diabetes complications, such as hypoglycemia unawareness, are at constant risk of life-threatening low blood glucose,” said Jeremy Pettus, MD, investigator in the clinical trial and Assistant Professor of Medicine at UC San Diego. “The PEC-Direct islet cell replacement therapy is designed to help patients with the most urgent medical need.”
The PEC-Direct product candidate is being developed for type 1 diabetes patients who have hypoglycemia unawareness, extreme glycemic lability, and/or severe hypoglycemic episodes. It is estimated that about 140,000 people in Canada and the US have such high-risk type 1 diabetes. In addition to providing an unlimited supply of cells for implantation, the PEC-Direct approach has other potential advantages relative to cadaver islet transplants such as delivering a more consistent product preparation under quality-controlled cGMP conditions, and a more straightforward and safe mode of delivery.
The clinical trial is being supported in part by JDRF, the leading global organization funding type 1 diabetes research. “JDRF remains dedicated to accelerating the delivery of beta cell replacement therapies to the T1D community, and we commend ViaCyte in its announcement of the first patients to be implanted with the PEC-Direct islet cell replacement therapy,” said Derek Rapp, JDRF President and Chief Executive Officer. “JDRF is excited to support this clinical development given its potential to help those people with type 1 diabetes that need it the most – those at high risk of life-threatening acute complications. JDRF and ViaCyte share a continuing commitment to realizing the potential of beta cell replacement strategies to deliver insulin independence without immune suppression for people living with type 1 diabetes, and ultimately, at JDRF we hope this will move us forward in fulfilling our vision of a world without type 1 diabetes.”
“There are limited treatment options for patients with high-risk type 1 diabetes to manage life-threatening hypoglycemic episodes,” said Paul Laikind, PhD, President and Chief Executive Officer of ViaCyte. “We believe that the PEC-Direct product candidate has the potential to transform the lives of these patients and we are excited to move closer to that goal with the initiation of clinical evaluation announced today. This also represents a step towards a broader application of the technology. We remain fully committed to developing a functional cure for all patients with insulin-requiring diabetes. To that end, we are hard at work on next-generation approaches as well, and expect the work with PEC-Direct to further advance our knowledge and drive progress.”
In addition to JDRF, the California Institute for Regenerative Medicine (CIRM)’s Alpha Clinic, the Sanford Stem Cell Clinical Center, the JDRF Canadian Clinical Trials Network (CCTN), the Stem Cell Network, and Alberta Innovates Health Solutions (AIHS) are all providing support for the trial.
About the PEC-Direct Product Candidate
The PEC-Direct product candidate delivers stem cell-derived pancreatic progenitor cells, called PEC-01™ cells, in a device designed to allow direct vascularization of the cells in the device. After implantation, these cells are expected to become mature human islet tissue including well-regulated beta cells producing insulin on demand. The direct vascularization of the implanted cells is expected to allow for robust and consistent engraftment but will necessitate the use of maintenance immune suppression therapy.
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 from stem cells, which are then implanted in a durable and retrievable cell delivery device. Once implanted and matured, these cells are designed to secrete insulin and other pancreatic hormones in response to blood glucose levels. ViaCyte has two products in clinical 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 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 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 headquartered in San Diego, California. The Company is funded in part by the California Institute for Regenerative Medicine (CIRM) and JDRF.