Semma Therapeutics Announces Pre-Clinical Proof-of-Concept in Two Lead Programs in Type 1 Diabetes

On Jul 2, 2019

Semma Therapeutics, Inc., a biotechnology company pioneering the curative use of stem cells in regenerative medicine, announced the achievement of pre-clinical proof-of-concept based on data from its two lead programs for type 1 diabetes testing stem cell-derived islets (SC-islets) in both non-human primates and pigs. The data were presented by Felicia Pagliuca, Ph.D., Vice President of Cell Biology Research and Development, on Saturday, June 29^th in a plenary oral presentation at the International Society for Stem Cell Research (ISSCR) Annual Meeting 2019 in Los Angeles.

“Over the last 100 years, only incremental advances have been made to manage type 1 diabetes with insulin. These data demonstrate that our SC-islets can be delivered safely, maintain functionality and demonstrably reduce insulin requirements in non-human primates. Furthermore, when encapsulated in our device, our cell therapy shows rapid insulin response in pigs without immunosuppression,” said Bastiano Sanna, Ph.D., Chief Executive Officer. “We are thrilled to share our significant progress in two lead programs, pushing us a step closer to providing effective, curative therapeutic options for people living with type 1 diabetes.”

Cell Therapy in Non-Human Primates

In pre-clinical studies, SC-islets were delivered into diabetic non-human primates under immunosuppression. SC-islets were infused into the portal vein to mimic planned human clinical trials. The studies showed that:

SC-islets successfully engraft and are highly functional.
Functional cells persist over six weeks in the non-human primate liver.
Transplanted SC-islets reduce the requirement for insulin by more than 60 percent.

Cell Therapy with Immunoprotective Device in Pigs

In another pre-clinical study, pigs received SC-islets encapsulated in Semma Therapeutics’ novel immunoprotective device. The device is designed to keep the SC-islets alive and functional without a need for immunosuppression. The results showed that:

The device provides immunoprotection, maximizing cell survival while minimizing foreign body reaction.
The device encapsulated SC-islets rapidly secretes insulin in response to stimulation quantified by C-peptide, which measures beta cell function through insulin production.

Based upon the pre-clinical data, Semma Therapeutics plans to execute its two-part clinical strategy beginning in the first half of 2020 with a clinical trial in patients with difficult to treat diabetes and hypoglycemia unawareness. In the second half of 2020, Semma Therapeutics plans to initiate a second clinical trial for the broader adult type 1 diabetes population using SC-islets encapsulated in its immunoprotective device without immunosuppression.

“Semma’s team has achieved unprecedented pre-clinical results with both its SC-islets and immunoprotective device, which enables the path to clinical trials next year,” said Douglas Melton, Ph.D., Co-Chair of the Department of Stem Cell and Regenerative Biology at Harvard University, Co-Chair of the Harvard Stem Cell Institute and Founder of Semma Therapeutics. “These updates provide hope for the millions of patients with type 1 diabetes around the world who are affected by this debilitating disease.”