Potentially Transformative Therapy for People Living with T1D

JDRF is providing additional milestone-based funding for the continued development of ViaCyte’s VC-01™ encapsulated cell therapy product candidate for the treatment of T1D. ViaCyte is a leading regenerative medicine company. JDRF will fund up to $7 million to help ensure a rapid transition of the project into the clinical phase of development once ViaCyte’s investigational new drug application (IND) is filed with and accepted by the U.S. Food and Drug Administration (FDA). This commitment builds on JDRF’s previous support of ViaCyte’s preclinical development program focused on collecting the necessary animal safety and efficacy data to support introduction into clinical testing.

ViaCyte’s innovative VC-01 product candidate is a cell replacement therapy that could transform the way individuals with T1D manage their disease by supplying an alternative source of insulin-producing cells with the potential to free individuals from a dependence on external insulin use. The product candidate uses pancreatic progenitor cells derived from a stem cell line, called PEC-01™ cells, which are encapsulated inside a semi-permeable device called the Encaptra® drug delivery system. Both the cells and the device are ViaCyte proprietary technologies. The resulting VC-01 product candidate is designed to be inserted under the skin where, after maturation of the PEC-01 cells into islet-like structures including beta cells, they are expected to produce insulin and other pancreatic hormones in response to blood glucose levels, similar in manner to that of normal islets in the pancreas. If the product candidate performs as intended, it has the potential to provide individuals who have T1D with a replacement for the beta cells lost or impaired as a result of their disease.

Beta cell encapsulation research is a high priority research area for JDRF because of its potential benefits for individuals with T1D. JDRF has provided substantial funding for multiple scientific research programs to advance discovery and development research in this area. Based on earlier basic research support, JDRF began its partnership with ViaCyte in December 2011, and previously provided the Company with $6 million in milestone-based funding that has contributed to the progress in developing the VC-01 product candidate toward the filing of an IND.

ViaCyte is planning to file an IND with the FDA as early as next quarter to support initiation of clinical evaluation of the VC-01 product candidate. Assuming an IND filing next quarter and no objections from the FDA, the Company plans to begin testing in individuals with T1D around mid-year. The primary purpose of the first human study will be to establish that the product candidate is safe and well tolerated; however, efficacy will also be assessed. After initial safety is demonstrated in the first group of participants, ViaCyte plans to expand the trial to multiple clinical sites in the United States and Canada.

“We look forward to our continued work with ViaCyte as we help fund the upcoming clinical trials for the VC-01 product candidate, an important milestone to advance this promising encapsulated cell therapy,” said Jeffrey Brewer, JDRF president and chief executive officer. “ViaCyte has an innovative and advanced technology that we believe has the potential to significantly benefit people with type 1 diabetes. A product like VC-01 could someday be a key step in helping JDRF achieve its vision of creating a world without type 1 diabetes.”

Dr. Paul Laikind, ViaCyte’s president and chief executive officer, said, “JDRF has been and continues to be a valuable partner as we work to develop this potentially transformative new approach to controlling insulin-dependent diabetes. While their financial help has been welcomed, as leading experts on type 1 diabetes, JDRF’s advice and advocacy on our behalf has been equally if not more important. Together with JDRF, we will soon determine if the promising results demonstrated in preclinical studies translate to patients. If so, the VC-01 product candidate could potentially represent a practical cure for type 1 diabetes, and possibly an important therapy for patients with insulin-requiring type 2 diabetes as well.”