ViaCyte Announces First Patients Implanted in Cohort 2 with Potentially Efficacious Dose of PEC-Direct Islet Cell Replacement Therapy
President and CEO Dr. Paul Laikind will give an update at Biotech Showcase in San Francisco
SAN DIEGO, January 5, 2018 — ViaCyte, Inc., a privately-held regenerative medicine company, today announced that the first patients have been implanted in Cohort 2 with a potentially efficacious dose of the company’s PEC-Direct islet cell replacement therapy. This novel cell replacement therapy is being developed as a potential functional cure for patients with type 1 diabetes who are at high risk for acute life-threatening complications. Paul Laikind, Ph.D., President and CEO of ViaCyte, will give an update at the 10th Annual Biotech Showcase™ Conference, on Monday, January 8 at 11:15 a.m. in Franciscan C at the Hilton San Francisco Union Square.
The PEC-Direct product candidate is currently in a Phase 1/2 open-label clinical trial at multiple sites in the United States and Canada to evaluate safety and efficacy. Cohort 2 is expected to enroll up to 40 patients, and the primary efficacy measurement 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.
“The implantation of a potentially efficacious dose of PEC-Direct marks an important milestone in the development of a functional cure for diabetes,” said Dr. Laikind. “While we are developing PEC-Direct to meet a critical unmet medical need for people who are most severely impacted by type 1 diabetes, we are also optimizing our other clinical-stage therapeutic candidate, PEC-Encap, which holds the promise of a therapy for all people with insulin-requiring diabetes. With cutting-edge technologies, data from clinical-stage product candidates, an extensive intellectual property estate including proprietary know-how, and a strong and dedicated team, we believe ViaCyte uniquely has all of the key elements needed to produce transformative therapies for diabetes.”
It is estimated that about 140,000 people in United States and Canada, or about 10 percent of type 1 diabetes patients, have high-risk type 1 diabetes. These patients are often eligible for islet transplants, a procedure that can be highly effective but suffers from a severe lack of donor material. The PEC-Direct product candidate could overcome some of the key limitations of islet transplant by providing a virtually unlimited supply of cells manufactured under quality-controlled conditions, with a potentially safer and more optimal route of administration.
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, which 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, will require immune suppression. Thus, PEC-Direct is being developed to treat patients with the greatest unmet medical need, including type 1 diabetes patients who already require immune suppression following a kidney transplant, as well as type 1 patients who are at high risk for acute complications, such as hypoglycemia unawareness, extreme glycemic lability, and/or severe hypoglycemic episodes.
ViaCyte recently announced funding from the California Institute for Regenerative Medicine (CIRM) to support yet another potentially transformative cell therapy approach that could be used for the treatment of diabetes. The company plans to engineer its well-characterized, regulatory-compliant CyT49 pluripotent stem cell line to be immune evasive, which, if successful, would open up off-the-shelf therapeutic applications in a number of fields.
The following institutions provide support for the PEC-Direct clinical trial: CIRM, JDRF, CIRM’s Alpha Clinic, the Sanford Stem Cell Clinical Center, the JDRF Canadian Clinical Trials Network, the Stem Cell Network, and Alberta Innovates Health Solutions. The clinical trial is under way at the UC San Diego School of Medicine’s Altman Clinical Trials Research Institute, the University of Alberta Hospital in Edmonton, Alberta, and the University of Minnesota, with additional sites planned in the US and Canada.
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 from stem cells, which are then implanted in durable and retrievable cell delivery devices. Once implanted and matured, these cells are designed to secrete insulin and other pancreatic hormones in response to blood glucose levels. ViaCyte has two product candidates in clinical-stage 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.