ViaCyte said today that it will begin clinical trials of its novel islet cell replacement therapy in development as a potential cure for Type I diabetes, after landing regulatory wins from the FDA and Health Canada.

The company’s PEC-Direct candidate delivers stem cell-derived pancreatic progenitor cells in a device that is designed to enable direct vascularization of the cells. Following implantation, ViaCyte said the cells can proliferate and mature into human islet tissue that can produce insulin. The therapy is being developed for Type I diabetes patients who are at high risk for acute, life-threatening complications.

The therapy will necessitate the use of maintenance immune suppression therapy, ViaCyte reported.

The company’s newly-acquired funds will support a 40-patient clinical to measure the therapy’s ability to promote clinically relevant insulin production in a patient population that has little to no ability to produce endogenous insulin at the time of enrollment. Other endpoints will include injectable insulin use and the incidence of hypoglycemic events, ViaCyte said.

“The loss of insulin-producing beta cells leads to Type I diabetes, making it an ideal target for cell replacement therapy,” Dr. James Shapiro, director of the Clinical Islet Transplant Program at the University of Alberta, said in prepared remarks.  “Islet transplants from scarce organ donors have offered great promise for those with unstable, high-risk type 1 diabetes, but the procedure has many limitations.  With an unlimited supply of new islets that the stem cell-derived therapy promises, we have real potential to benefit far more patients with islet cell replacement.”

“ViaCyte was the 1st to differentiate human stem cells into glucose-responsive, insulin-producing cells, and now we are running the 1st and only clinical trials of stem cell-derived islet replacement therapies for Type I diabetes,” Viacyte’s president & CEO Paul Laikind said.  “While insulin therapy transformed Type I diabetes from a death sentence to a chronic illness, it is far from a cure.  Type I diabetes patients continue to deal with the daily impact of the disease and remain at risk for often severe long-term complications.  This is especially true for the patients with high-risk type 1 diabetes, who face challenges such as hypoglycemia unawareness and life-threatening severe hypoglycemic episodes.  These patients have a particularly urgent unmet medical need and could benefit greatly from cell replacement therapy.”

PEC-Direct is 1 of 2 candidates that ViaCyte is developing to treat patients with diabetes. The company is also working on PEC-Encap to deliver the same cell therapy using the Encaptra Cell Delivery System designed to protect the cells from the patient’s immune system. ViaCyte is developing PEC-Encap to treat all patients who require insulin to control their condition.

ViaCyte also said today that it raised $10 million to support development from investors that included W.L. Gore and JDRF.

“In addition to our recently announced collaborative research agreement with ViaCyte, Gore’s investment should strengthen our collaboration with ViaCyte and accelerate the development of the PEC-Encap product candidate,” Edward Gunzel, technical leader for Gore PharmBIO Products, said. “This effort supports Gore’s mission to improve lives through advanced materials, and aligns well with a broader portfolio of step change products which we will be launching for the biotech industry over the next few years.  We believe that ViaCyte’s capabilities in cell therapy for diabetes are the most advanced in the industry, and we are pleased to bring our material and device expertise to the challenge of developing a functional cure for insulin-requiring diabetes.”

“With JDRF’s support, ViaCyte has achieved the important goal of advancing innovative islet replacement therapies into clinical trials,” JDRF’s chief mission officer Aaron Kowalski added.  “We are excited to support the preclinical and clinical development work with the PEC-Direct product candidate, given its potential to help the Type I diabetes patients with the most urgent medical need, those at high risk of life-threatening acute complications.  In addition, we believe that success with PEC-Direct is an important step on the path to a functional cure for all patients with Type I diabetes, helping to fulfill our commitment of turning Type One into Type None.”