Year 3

In Year 3 of the Strategic Partnership, the open-label VC01-101 STEP ONE* clinical trial continued to make significant progress. The observations from the STEP ONE study, testing a sub-therapeutic dose, suggest that the VC-01 (also known as PEC-Encap) product candidate is safe and well-tolerated by patients with type 1 diabetes. To date, no adverse events related to the implanted cells have been reported. Adverse events observed in the STEP ONE trial have generally been those that would be anticipated, related to the surgical procedure, such as discomfort during a post-operative recovery period. The Encaptra delivery device appears to be immuno-protective as designed, with no evidence of allo- or auto-immune rejection or sensitization in patients implanted to date. Methodological improvements have been developed and implemented over the course of the trial, and the potential for long-term cell survival has been demonstrated. This early observation shows that the team is on the right track for translating the promising animal results to humans. While the cumulative observations in the STEP ONE trial are encouraging, further product development is needed to ensure reliably robust engraftment in patients. ViaCyte is continuing both non-clinical and clinical efforts with the goal of reducing variability between patients and further improving cell engraftment with the PEC-Encap product candidate. Enrollment of the second, higher dose cohort of patients, seeking to demonstrate clinically relevant efficacy, will commence once the methods yielding robust reliable engraftment are established.

* Safety, Tolerability, and Efficacy of the VC-01 Combination Product in Type One Diabetes