TORONTO and HAIFA, Israel — NurExone Biologic Inc. (TSXV: NRX) (FSE: J90) (NRX.V) (the “Company” or “NurExone”), a pioneering biotechnology company, is thrilled to announce that the U.S. Food and Drug Administration (FDA) has granted Orphan-Drug Designation (ODD) for its ExoPTEN therapy, recognizing the potential of this groundbreaking regenerative therapy for acute spinal cord injury, a condition where effective treatments are limited.
The orphan drug designation provides significant benefits to pharmaceutical companies developing drugs for rare diseases, i.e. those impacting fewer than 200,000 people in the United States. These benefits include market exclusivity, financial incentives, regulatory assistance, and support with drug development. Overall, the designation incentivizes and supports the development of certain treatments, increasing access to therapies for patients.
Earning orphan-drug designation is a significant milestone for the Company. The designation covers the use of mesenchymal stem cell (MSC) derived small extracellular vesicles (EVs) loaded with short and modified interfering RNA (siRNA) targeting the phosphatase and tensin homolog (PTEN) protein for acute spinal cord injury, as implemented in the Company’s ExoPTEN drug under development.
“Orphan-drug designation is expected to streamline our go-to-market, shorten our regulatory process saving the Company millions of dollars, and provide valuable market exclusivity. We appreciate the formal recognition of the potential impact of our therapy on the lives of patients suffering from acute spinal cord injuries,” said Dr. Shaltiel, CEO of NurExone Biologic, Ltd.
About NurExone Biologic Inc.
NurExone Biologic Inc. is a TSXV listed pharmaceutical company that is developing a platform for biologically-guided ExoTherapy to be delivered, non-invasively, to patients who suffered traumatic spinal cord injuries. ExoTherapy was conceptually demonstrated in animal studies at the Technion, Israel Institute of Technology. Via its fully-owned subsidiary, NurExone Biologic Ltd, NurExone is translating the treatment to humans, and the Company holds an exclusive worldwide license from the Technion and Tel Aviv University for the development and commercialization of the technology.