The US Food and Drug Administration (FDA) has granted the fourth Orphan Drug Designation to Araim Pharmaceuticals’ ARA 290, an innate repair receptor (IRR) activator for increased survival and improved function of pancreatic islets after transplant in patients with Type 1 diabetes (T1B).
“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, the company’s chief medical officer in a press release. “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.”
“Orphan” status is granted to drugs under development that are intended for conditions rare enough that the marketing value is not considered profitable for the pharmaceutical company, but moving forward with development is important to public need.
Type 1 diabetes (T1B) is a form of diabetes mellitus that results from the autoimmune destruction of the pancreatic beta cells that produce insulin. The subsequent lack of insulin leads to increased glucose in the blood and urine.
Pancreatic islet cell transplant (PITx), is an experimental treatment for T1B. Once transplanted, the islets begin to produce insulin, actively regulating the level of glucose in the blood.
The most significant barriers to successful PITx are the decreased function and survival of islet infusions. Cytokine-induced inflammation associated with PITx causes early damage to islets and graft loss.
ARA 290 is a first-in-class synthetic peptide that activates the innate repair mechanism in the setting of tissue injury. ARA 290 selectively binds to the IRR activating tissue protective, reparative, and anti-inflammatory signaling pathways.
Results from a large program with ARA 290 following transplant demonstrated via animal models of T1B, that ARA 290 improved the outcome of PITx by 85 percent.
“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.