ViaCyte files investigational new drug application and device master file with FDA for novel cell replacement therapy …

Published 18 July 2014

ViaCyte a privately held regenerative medicine company developing a cell replacement therapy for the treatment of diabetes, announced that it has filed an Investigational New Drug application (IND) with the United States Food and Drug Administration (FDA) seeking to initiate a Phase 1/2 clinical trial in patients with type 1 diabetes.

The trial would evaluate the safety and efficacy of ViaCyte's VC-01 product candidate, a stem cell-derived, encapsulated cell replacement therapy. In a related development, ViaCyte submitted a Medical Device Master File (called MAF) to the FDA in support of the Encaptra drug delivery system, the device component of the VC-01 product candidate.

"The filing of this IND represents the culmination of many years of research and development by a dedicated team focused on developing a cell replacement therapy for patients with type 1 diabetes and advancing our VC-01 product candidate to human clinical trials," said Paul Laikind, Ph.D., President and Chief Executive Officer of ViaCyte. "The ViaCyte team has been assisted and supported by the California Institute for Regenerative Medicine (CIRM) a leading organization focused on advancing the field of stem cell-based technologies, and JDRF, the leading advocacy organization for patients with type 1 diabetes," added Dr. Laikind.

ViaCyte's VC-01 product candidate consists of pancreatic progenitor cells, called PEC-01 cells, which are derived from a proprietary human embryonic stem cell line. These cells are then encapsulated by use of ViaCyte's Encaptra device. When implanted under the skin, the PEC-01 cells are designed to mature and further differentiate into insulin-producing beta and other endocrine cells that regulate blood glucose in a manner similar or identical to the normal islets that comprise the endocrine pancreas.

Based on a pre-IND meeting with the FDA and subsequent consultations, ViaCyte is proposing to initiate clinical evaluation of the VC-01 product candidate directly in patients with type 1 diabetes who have minimal to no insulin-producing beta cell function. In addition to evaluating the safety of the product candidate in these patients, the study is designed to demonstrate the effectiveness of the VC-01 product candidate in replacing lost endocrine function that is central to the disease.

In the proposed clinical trial, insulin production from the VC-01 implant would be assessed by measuring C-peptide, a biomarker for insulin produced by beta cells that is expected to provide a sensitive measure of efficacy in these patients. As proposed, the trial would also evaluate secondary end-points related to the need for administration of pharmaceutical insulin to control the disease and the incidence of hypoglycemia, a common side effect associated with pharmaceutical insulin usage.

ViaCyte's proprietary Encaptra device is designed to contain the implanted cells, preventing biodistribution, as well as shielding them from the immune system. Although PEC-01 cells are human cells, they are not the patient's actual cells. As such, they are considered an allogeneic graft, which typically requires immunosuppression in order for the recipient to tolerate the implant. However, the Encaptra device is designed to prevent the patient's immune system from accessing the implanted cells, thereby facilitating successful engraftment and subsequent maturation to islets.

The VC-01 product candidate is designed to be placed under the skin of the patient and can be monitored and readily removed, if or when required. The option to remove the cells is designed to provide an important safety benefit for this novel stem cell-derived cell therapy candidate. It is being regulated as a biologic through interaction with the Office of Cell, Tissue and Gene Therapy within CBER at the FDA. Given the combination product nature of the product candidate, the Center for Devices and Radiological Health at the FDA is also involved in its regulation.

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ViaCyte files investigational new drug application and device master file with FDA for novel cell replacement therapy ...