Text of ViaCyte's Statement on its New Proposed Diabetes Product

The California Stem Cell Report this morning queried Paul Laikind, the CEO of ViaCyte, Inc., of San Diego, about the company's upcoming $4 million award from the California stem cell agency and the award's relationship to the ViaCyte product known as VC-01. Here is the text of his response.

"ViaCyte is continuing development of VC-01 (what we now call PEC-EnCap).  PEC-EnCap is the first encapsulated allogeneic cell therapy to enter the clinic where the device is designed to protect the cells from the host adaptive immune system.

 "We have early clinical demonstration of the feasibility of the PEC-EnCap approach; the Encaptra Drug Delivery Device appears to be functioning as designed and we have shown engraftment and differentiation to beta cells is achievable at 12 weeks.  However, work is continuing to ensure a robust and consistent engraftment before we move to the second cohort of patients and seek to demonstrate efficacy.

 "We can’t overemphasize enough the importance the clinical results we are obtaining, not only for the further development of PEC-EnCap but for the stem cell-derived cell therapy field in general. This important work has, of course, received important support from CIRM. "In parallel with the development of PEC-EnCap we are preparing to initiate clinical development of VC-02 (PEC-Direct).  PEC-Direct delivers the same biologic component as PEC-EnCap, PEC-01 pancreatic progenitor cells, but does so in a device that allows direct vascularization.  This direct vascularization, based in part on what we have learned with PEC-EnCap during the clinical evaluation, is expected to allow for a very robust engraftment and cellular performance.

"Given the open nature of the device, patients implanted with PEC-Direct, as with other transplants, would require chronic immune suppression.  Thus it is being developed to treat patients with type 1 diabetes that are at high risk for acute complications such as severe hypoglycemic events associated with hypoglycemia unawareness syndrome.

 "This high risk patient population is the same population that would be eligible for cadaver islet transplants, a procedure that has been demonstrated as very effective but suffers from a severe lack of donor material and high cost.  PEC-Direct is expected to overcome these limitations by providing an unlimited supply of cells for transplant and a safer more optimized route of administration.

"ViaCyte remains committed to the development of PEC-EnCap which we view as a potentially transformational therapy for the majority of insulin utilizing patients with diabetes, both type 1 and type 2.  PEC-Direct represents a potentially nearer term therapy for the patients at the highest risk."