Lilly to Acquire Phase III Molecule Teplizumab for the Treatment of Type 1 Diabetes

Companies to Collaborate on the Development of Autoimmune Disease Treatments.

As Pfizer takes a big step out of the diabetes market with its decision to halt production of inhaled insulin, Eli Lilly and Co. is taking another step into the market by buying the rights to develop a molecule being tested for treatment of type 1 diabetes.

The Indianapolis-based drug maker said Thursday it has bought the rights from MacroGenics, a seven-year-old private biotechnology company based in Rockville, Md.  The move adds to Lilly’s late-stage pipeline and bulks up its role as a biotech player.

The drug teplizumab may have the ability to preserve and protect the beta cells of the pancreas,  a humanized anti-CD3 monoclonal antibody, as well as other potential next generation anti-CD3 molecules for use in the treatment of autoimmune diseases. As part of the deal, Lilly will acquire the exclusive rights to the molecule. Teplizumab is currently being studied in the PROTÉGÉ trial, a global pivotal Phase II/III clinical trial for individuals with recent-onset type 1 diabetes. MacroGenics will continue to oversee the PROTÉGÉ trial.

 “Teplizumab is a significant addition to our growing portfolio of biotechnology-derived medicines, which are a key strategy for Lilly,” said Thomas Bumol, Ph.D., Lilly, vice president, biotechnology discovery research, and president, Applied Molecular Evolution. “Immune tolerance represents a novel therapeutic approach to the treatment of autoimmune disease, and we are excited to be partnering with MacroGenics to clinically evaluate this mechanism in type 1 diabetes.”

Teplizumab, also called MGA031 and hOKT3γ1(Ala-Ala), is a humanized, non-Fc receptor binding, anti-CD3 monoclonal antibody. Teplizumab binds to an epitope of the CD3-epsilon chain expressed on mature T lymphocytes and, by doing so, may modulate the pathological

immunologic responses underlying multiple autoimmune diseases. Specifically, teplizumab may inhibit unwanted effector T cells and enhances beneficial regulatory T cell functions, thus promoting immune tolerance.

The PROTÉGÉ trial, a global, pivotal Phase II/III clinical trial study, is evaluating the safety and efficacy of three teplizumab dosing regimens administered at the start of the study and again at six months in individuals with recent-onset type 1 diabetes ages 8 to 35 who are up to 12 weeks from their diagnosis.

Type 1 diabetes is an autoimmune disease in which the body’s immune system attacks and destroys the insulin-producing beta cells of the pancreas. Researchers believe teplizumab may control the T lymphocytes that mediate destruction of the insulin-producing beta cells of the islets of the pancreas. In doing so, they believe teplizumab may have the ability to preserve and protect the remaining beta cells of the pancreas.