Published Study Shows Similar In Vivo Time-to-Function Between In Vitro-Produced Beta-like Cells and Pancreatic Progenitor Cells Designed to Mature Into Beta Cells In Vivo.
SAN DIEGO, September 10, 2015 – ViaCyte, Inc., a privately-held regenerative medicine company with the first stem cell-derived islet replacement therapy for the treatment of diabetes in clinical trials, today announced publication of a study demonstrating that insulin-producing cells created in vitro from human embryonic stem cells can mature and function after being encapsulated and implanted into an animal model. In addition, the Company announced the addition of three patents to its extensive portfolio covering its cell therapy platform.
The article, entitled “Insulin-Producing Endocrine Cells Differentiated In Vitro From Human Embryonic Stem Cells Function in Macroencapsulation Devices In Vivo,” is published in Stem Cells Translational Medicine (October 2015 issue; e-published online August 24, 2015). In the June 2015 issue of the same journal, ViaCyte scientists published a review article summarizing the advances in bioprocessing and scale-up that have enabled ViaCyte’s VC-01™ product candidate to be the first stem cell-based treatment for type 1 diabetes to enter clinical testing.
The current article describes beta-like cells that have similar properties to the pluripotent stem cell-derived insulin-producing cells reported by other groups, except that ViaCyte produced populations with substantially higher endocrine cell content. The results demonstrated for the first time that nearly pure in vitro-produced endocrine cells can function in vivo. Populations comprised of up to 98% endocrine cells (and less than 2% pancreatic progenitor cells) exhibited robust glucose-responsive insulin production in a mouse model.
The cells in the study were differentiated further down the beta cell lineage than the PEC-01™ pancreatic progenitor cells being tested in ViaCyte’s VC-01 product candidate, which is currently in a Phase 1/2 clinical trial for treatment of type 1 diabetes. The animal study also demonstrated for the first time that when encapsulated in a device and implanted into mice, these more mature cells are capable of producing functional pancreatic beta cells. ViaCyte is also the first to show that these further differentiated cells can function in vivo following cryopreservation, a valuable process step when contemplating clinical and commercial application.
“The tremendous progress in understanding pancreatic cell differentiation has enabled development of the first clinical-stage therapy with potential to effectively cure type 1 diabetes,” said Paul Laikind, Ph.D., President and CEO of ViaCyte. “For a number of reasons we believe that the pancreatic progenitor cells that are the active component of the VC-01 product candidate are better suited for cell replacement therapy. However, the current work has expanded our fundamental knowledge of beta cell maturation and could lead to further advances for the field.”
In addition to demonstrating in vivo functionality, the time to functional maturation (i.e., glucose-responsive insulin production) following implantation of the more highly differentiated cells was compared to that of pancreatic progenitors. The results showed no appreciable differences between the two cell populations suggesting that engraftment and acquisition of a robust glucose response, not differentiation from progenitor cell to endocrine cell, are the rate-limiting steps following implantation.
In addition to the research progress, ViaCyte continues to build its large patent portfolio with three more U.S. patents having issued since November 2014. These patents are directed to: Methods for making and enriching pancreatic endocrine type cells (U.S. Patent No. 9,045,736); Scale-up technology directed to pluripotent stem cell aggregates in suspension in a roller bottle (U.S. Patent No. 8,895,300); and Pancreatic endoderm cell cultures with an ERBB receptor tyrosine kinase activating agent (U.S. Patent No. 9,109,245). These patents bolster and further protect the Company’s VC-01 product candidate and its cell therapy platform technology.
ViaCyte’s VC-01 product candidate, comprised of human PEC-01 pancreatic progenitor cells macroencapsulated in the Encaptra® drug delivery system, has previously been demonstrated to regulate glucose levels in animal models, and is in an ongoing clinical trial designed to evaluate safety and efficacy in type 1 diabetes patients. More information on the Phase 1/2 clinical trial, called STEP ONE, for Safety, Tolerability, and Efficacy of VC-01 Combination Product in Type One Diabetes, can be found at www.clinicaltrials.gov.
ViaCyte is a privately-held regenerative medicine company focused on developing a novel cell replacement therapy for the treatment of diabetes. ViaCyte is conducting a Phase 1/2 clinical trial of the Company’s lead VC-01 product candidate in patients with type 1 diabetes who have minimal to no insulin-producing beta cell function. ViaCyte’s VC-01 combination product candidate is based on the production of pancreatic progenitor cells derived from human pluripotent stem cells. These progenitor cells are implanted in a durable and retrievable encapsulation device. Once implanted and matured, these cells are designed to secrete insulin and other hormones in response to blood glucose levels. The VC-01 product candidate is being developed as a potential long-term diabetes treatment without immune suppression, and without risk of hypoglycemia or other diabetes-related complications.
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.