DMC concludes Protege Phase 3 clinical trial for type 1 diabetes lacks efficacy

MacroGenics, Inc. and Eli Lilly and Company (NYSE: LLY) today announced that the Protege Data Monitoring Committee (DMC), composed of independent experts in the fields of diabetes and biostatistics, has completed a planned analysis of one-year safety and efficacy data of the Protege Phase 3 clinical trial of teplizumab, an investigational biologic under development for the treatment of individuals with recent-onset type 1 diabetes.

The DMC concluded that the primary efficacy endpoint of the study, a composite of a patient's total daily insulin usage and HbA1c level at 12 months, was not met. The DMC, noting that all administration of experimental drug had been completed, commented that appropriate safety monitoring is warranted. No unanticipated safety issues were identified in the DMC's review.

"We will comply with the DMC's recommendations," said Scott Koenig, M.D., Ph.D., President and Chief Executive Officer, MacroGenics. "We remain committed to discovering and developing novel biologics for the treatment of autoimmune disorders like type 1 diabetes."

Following careful evaluation of the DMC's recommendations for Protege, based on the lack of efficacy, the companies have decided to suspend further enrollment and dosing of patients in two other ongoing clinical trials of teplizumab in type 1 diabetes: the Protege Encore Trial, a second Phase 3 trial of the same design as Protege, and the SUBCUE trial, a Phase 1b trial that is exploring the subcutaneous administration in patients with type 1 diabetes.

"The failure to meet the primary endpoint is obviously disappointing for the millions of people who live with and treat type 1 diabetes," said Gwen Krivi, Vice President, Product Development, Lilly Diabetes. "Lilly and MacroGenics will be considering all options for teplizumab in type 1 diabetes as well as the impact of the DMC's recommendations on other potential indications."