NurExone Biologic Inc.  a biopharmaceutical company developing exosome-based regenerative therapies, is pleased to announce that the European Medicines Agency (the “EMA”) has granted Orphan Medicinal Product Designation for the Company’s ExoPTEN therapy, marking a significant step towards making this potential treatment available for acute spinal cord injury patients across Europe. This designation supports the development of ExoPTEN and opens a pathway for faster entry into European markets, where the Company expects demand for effective spinal cord injury therapies to be high. Designed to provide nerve regeneration and functional recovery following spinal cord injury, ExoPTEN uses mesenchymal stem cell-derived extracellular vesicles loaded with siRNA targeting PTEN, a key protein in nerve regeneration.

The EMA’s Orphan Medicinal Product Designation offers valuable incentives, including 10 years of market exclusivity upon approval, access grants and incentives from the European Commission and Member States. Additionally, the Company may benefit from free or reduced-cost scientific advice and assistance with clinical trial design, which can streamline the regulatory process and reduce development costs. Moreover, some European Union countries also provide tax credits and other financial incentives to support orphan drug development.

“We are honored by the EMA’s recognition of ExoPTEN through the Orphan Medicinal Product Designation, which significantly advances our ability to enter the European market and offers hope to those impacted by acute spinal cord injuries,” said Dr. Lior Shaltiel, Chief Executive Officer of NurExone. “This designation, together with the recently granted United States Food and Drug Administration’s Orphan Drug Designation, reinforces our ability to accelerate the global development of ExoPTEN and NurExone as a company to address the urgent unmet needs of patients globally.”

According to the EMA, the acute spinal cord injury (“SCI”) market faces considerable challenges, with approximately 20,000¹ new cases in the European Union each year. These patients often require lifelong care and effective therapeutic options are limited. ExoPTEN’s innovative approach to promoting spinal cord recovery directly addresses this gap, with potential to meet a critical need in the European healthcare system.

Dr. Ina Sarel, NurExone’s Head of CMC Quality and Regulation added, “the EMA’s designation not only acknowledges ExoPTEN’s potential, but also paves the way for essential regulatory support as we prepare to advance into clinical trials. We are eager to work closely with the EMA and other agencies to accelerate ExoPTEN’s development and bring this innovative treatment to SCI patients across Europe.”