New drug could prevent graft loss in pancreatic islet transplantation

The European Commission has granted orphan medicinal product designation in the EU to Araim Pharmaceutical’s lead product candidate, Innate Repair Receptor activator ARA 290, for prevention of graft loss in pancreatic islet transplantation.

The EC’s approval follows a positive opinion in July from the European Medicines Agency’s (EMA) Committee for orphan medicinal products. The US Food and Drug Administration (FDA) has previously granted designation to ARA 290 for treatment to increase survival and improve functioning of pancreatic islets following transplantation.

Type 1 diabetes (T1D) is an autoimmune disease in which the body attacks and destroys the insulin producing beta cells in the pancreas. Pancreatic islet cell transplantation (PITx) is a promising experimental treatment option for patients with severe T1D characterised by significant metabolic instability and frequent life-threatening hypoglycemic episodes.

However, current islet cell transplantation requires that each recipient receive a large number of islets, as the procedure causes an inflammatory reaction which destroys a significant proportion of the administered islets. 

“In a rodent model of islet transplantation, ARA 290 markedly reduced transplantation-induced inflammation, resulting in a marked increase in islet survival and function, such that 85% of ARA 290 animals achieved the target blood glucose concentration, compared to none of the control animals,” said Dr Michael Brines, Co-Founder and CSO of Araim Pharmaceuticals. “These effects of ARA 290 on islet preservation, if translated successfully for human disease, could result in the need for far fewer islets per transplant, permitting a substantial increase in the number of individuals with T1D who can undergo the potentially curative procedure.”

There are no products authorised in the EU for prevention of islet cell graft loss, signifying an important unmet medical need.

“This EC decision highlights the significant need for a drug that could transform an important treatment option for patients with T1D”, stated Dr Anthony Cerami, Founder of Araim Pharmaceuticals.  “As the developer of the HbA1c diagnostic test, I have had a very strong interest in improving outcomes for patients with diabetes.”