Araim Pharmaceuticals, a clinical stage drug development company, announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation to its lead product candidate, Innate Repair Receptor activator ARA 290, for treatment to increase survival and improve functioning of pancreatic islets following transplantation. Araim was founded by Dr Anthony Cerami, who previously invented the hemoglobin A1c test that is used to manage diabetes worldwide.

"In an animal model, ARA 290 improved survival and function of the transplanted islets by 85%. Increased survival of islets cells following transplantation has been the greatest challenge to successful outcomes for patients with severe type 1 diabetes.  Protecting transplanted pancreatic islets from injury and subsequent loss underscores the potential beneficial role of Innate Repair Receptor Activation in conditions associated with acute or chronic stress and inflammation," said Dr. Michael Brines, co-founder and chief scientific officer of Araim Pharmaceuticals.

"Obtaining this Orphan Drug Designation for ARA 290 is another important regulatory milestone in advancing our platform towards the patient.  This fourth Orphan Drug Designation demonstrates the commitment of Araim Pharmaceuticals to prioritize and serve patients with critical unmet medical needs with our innovative Innate Repair Receptor platform" said Dr. Daiva Bajorunas, chief medical officer of Araim Pharmaceuticals.  "We are hopeful that improved transplant outcomes could become more widely available to the type 1 diabetic population with severe metabolic instability in need of additional treatment options."

Type 1 diabetes 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 type 1 diabetes characterized by significant metabolic instability and frequent life-threatening hypoglycemic episodes. When transplants are successful, they can result in normalized glucose without the need for insulin injections. However, despite the large number of islets and multiple transplantation procedures typically required for patients, long-term graft function and insulin independence are achieved in only a small minority.

Decreased function and survival of islet infusions are the most significant barriers to successful transplantation, limiting the clinical utility of PITx. Procedure-related cytokine-induced inflammation causes early damage to islets and graft loss after transplantation. Recently published data from a large active programme with ARA 290 in islet cell transplantation at the Karolinksa Institutet, Sweden demonstrate that ARA 290 significantly improves the outcome of islet cell transplantation in an animal model of type 1 diabetesi.

The Orphan Drug Designation programme provides orphan status to drugs and biologics, which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the US. This designation provides for a seven year marketing exclusivity period against competition, as well as certain incentives, including federal grants, tax credits and a waiver of PDUFA filing fees.

According to the Collaborative Islet Transplant Registry, 602 patients have received pancreatic islet allo-transplants between 1999 and 2012 in North America.  Significant obstacles to more widespread use of PITx are shortage of suitable islets from donors and process-related islet damage or destruction. The immediate inflammatory response associated with islet transplantation has been recognized as the primary cause of early damage to islets and graft loss,. Multiple different regimen with immune suppressive therapies that help prevent the long-term rejection of allogenic transplanted cells are under active study.  However, since a significant mass of islets is lost immediately, effective control of pre- and peri-transplant islet inflammation could improve post-transplant islet survival and in turn increase the benefits of islet cell transplantation for a greater number of patients.