Enthera Pharmaceuticals, Boston Children’s Hospital and the University of Milan reveal use of novel pathway to restore pancreatic cell function in diabetes

Enthera Pharmaceuticals, a Sofinnova Partners portfolio company, together with scientific partners Boston Children’s Hospital (Harvard Medical School) and the University of Milan, announced on Feb. 3 a study detailing the discovery of a harmful cellular pathway that causes the death of pancreatic beta cells that make insulin.

Enthera aims to develop a therapeutic to block the pathway to treat type 1 diabetes. The study, published Feb. 3 in Nature Communications, used three mouse models of diabetes, and found protective effects when the pathway was blocked genetically or with an antibody. Studies of human cells and diabetes patients were consistent with the mouse findings.

Data from the study reinforced the idea that the ‘IGFBP3/TMEM219’ apoptosis pathway is a key regulator of pancreatic beta cells and a novel therapeutic target for type 1 diabetes.

“The common thought for type 1 diabetes is that it autoimmune,” said Paolo Fiorina, MD, PhD, Enthera's founder. “But immunotherapy doesn’t completely cure diabetes. We think that dysregulation of beta cell homeostasis also plays a role and that IGFBP3 acts as a ‘betatoxin.’”

Enthera, founded in 2016, is developing biologics based on these discoveries. First-in-human tests of an antibody treatment to block the IGFBP3/TMEM219 pathway could begin as early as September 2022 in Europe.

For more information, read:
The article in Nature Communications

The Enthera press release

The news release from Boston Children's Hospital