NurExone Biologic Gains EMA Orphan Status for ExoPTEN in Spinal Cord Injury

On November 13, 2024, NurExone Biologic Inc. (TSXV: NRX) (OTCQB: NRXBF) (Germany: J90), a biopharmaceutical company specializing in exosome-based regenerative therapies, announced a significant milestone. The European Medicines Agency (EMA) has granted Orphan Medicinal Product Designation to the company's ExoPTEN therapy. This designation is a critical step in making this potential treatment accessible to patients with acute spinal cord injuries in Europe. The designation facilitates the development of ExoPTEN and accelerates its entry into the European market, where there is anticipated high demand for effective spinal cord injury treatments. ExoPTEN is designed to promote nerve regeneration and functional recovery post-spinal cord injury by utilizing mesenchymal stem cell-derived extracellular vesicles loaded with siRNA targeting PTEN, a protein crucial for nerve regeneration.

The Orphan Medicinal Product Designation from EMA provides several benefits, including 10 years of market exclusivity upon approval, and access to grants and incentives from both the European Commission and member states. Additionally, the designation allows the company to receive free or reduced-cost scientific advice and assistance with clinical trial design, which can streamline the regulatory process and reduce development costs. Some European Union countries also offer tax credits and other financial incentives to support orphan drug development.

Dr. Lior Shaltiel, CEO of NurExone, expressed his gratitude for the EMA's recognition of ExoPTEN, highlighting that the designation significantly advances the company's ability to enter the European market and provides hope to those affected by acute spinal cord injuries. He noted that this designation, along with the recent United States Food and Drug Administration’s Orphan Drug Designation, enhances their capability to accelerate the global development of ExoPTEN and address the critical unmet needs of patients worldwide.

The acute spinal cord injury market, according to the EMA, faces substantial challenges, with approximately 20,000 new cases in the European Union annually. These patients often require lifelong care, and effective therapeutic options are limited. ExoPTEN’s innovative approach to promoting spinal cord recovery aims to fill this gap, offering a much-needed solution within the European healthcare system.

Dr. Ina Sarel, Head of CMC Quality and Regulation at NurExone, highlighted that the EMA’s designation not only acknowledges the potential of ExoPTEN but also facilitates essential regulatory support as they prepare to advance into clinical trials. She emphasized their eagerness to collaborate closely with the EMA and other agencies to expedite ExoPTEN’s development and deliver this pioneering treatment to SCI patients across Europe.

NurExone Biologic Inc. is listed on both the TSX Venture Exchange (TSXV) and OTCQB. The company is developing a platform for biologically guided exosome-based therapies that can be administered non-invasively to patients with Central Nervous System injuries. Its first product, ExoPTEN for acute spinal cord injury, has demonstrated a 75% recovery rate in motor function in laboratory rats when administered intranasally. ExoPTEN has already received Orphan Drug Designation from the FDA. The NurExone platform technology is anticipated to provide innovative solutions for drug companies interested in non-invasive targeted drug delivery for various indications.