NurExone Biologic Secures EMA Orphan Status for ExoPTEN in Spinal Cord Injury, Accelerating Pathway to European Markets
NurExone Biologic has received Orphan Medicinal Product Designation from the European Medicines Agency (EMA) for its ExoPTEN therapy, designed to treat acute spinal cord injury. This designation provides significant benefits, including 10 years of market exclusivity upon approval, access to grants, and reduced costs for scientific advice. The therapy targets approximately 20,000 new cases of acute spinal cord injury annually in the European Union. ExoPTEN uses mesenchymal stem cell-derived extracellular vesicles loaded with siRNA targeting PTEN protein to promote nerve regeneration and functional recovery. This designation follows a similar recognition from the FDA, strengthening NurExone's global market position.
NurExone Biologic ha ricevuto la Designazione di Medicinale Orfano dall'Agenzia Europea dei Medicinali (EMA) per la sua terapia ExoPTEN, progettata per trattare il trauma acuto del midollo spinale. Questa designazione offre vantaggi significativi, tra cui 10 anni di esclusiva di mercato dopo l'approvazione, accesso a finanziamenti e costi ridotti per consulenze scientifiche. La terapia si rivolge a circa 20.000 nuovi casi di trauma acuto del midollo spinale annualmente nell'Unione Europea. ExoPTEN utilizza vescicole extracellulari derivate da cellule staminali mesenchimali caricate con siRNA mirante alla proteina PTEN, al fine di promuovere la rigenerazione nervosa e il recupero funzionale. Questa designazione segue un riconoscimento simile da parte della FDA, rafforzando la posizione di mercato globale di NurExone.
NurExone Biologic ha recibido la Designación de Medicamento Huérfano de la Agencia Europea de Medicamentos (EMA) para su terapia ExoPTEN, diseñada para tratar lesiones agudas de la médula espinal. Esta designación proporciona beneficios significativos, que incluyen 10 años de exclusividad en el mercado tras la aprobación, acceso a subvenciones y reducción de gastos para asesoría científica. La terapia se dirige a aproximadamente 20,000 nuevos casos de lesiones agudas de la médula espinal anualmente en la Unión Europea. ExoPTEN utiliza vesículas extracelulares derivadas de células madre mesenquimatosas cargadas con siRNA que tiene como objetivo la proteína PTEN para promover la regeneración nerviosa y la recuperación funcional. Esta designación sigue un reconocimiento similar de la FDA, fortaleciendo la posición de NurExone en el mercado global.
NurExone Biologic는 급성 척수 손상을 치료하기 위해 설계된 ExoPTEN 치료법에 대해 유럽 의약품청(EMA)으로부터 희귀 의약품 지정(Orphan Medicinal Product Designation)을 받았습니다. 이 지정은 승인 후 10년간의 시장 독점권, 보조금 접근, 과학적 조언에 대한 비용 절감 등 중요한 혜택을 제공합니다. 이 치료법은 매년 유럽 연합에서 발생하는 약 20,000건의 새로운 급성 척수 손상 사례를 대상으로 합니다. ExoPTEN은 PTEN 단백질을 목표로 하는 siRNA로 로드된 중간엽 줄기세포 유래의 세포외 소포를 사용하여 신경 재생과 기능적 회복을 촉진합니다. 이 지정은 FDA의 유사한 인정 이후 이루어져 NurExone의 글로벌 시장 위치를 강화합니다.
NurExone Biologic a reçu la désignation de médicament orphelin de l'Agence européenne des médicaments (EMA) pour sa thérapie ExoPTEN, conçue pour traiter les blessures aiguës de la moelle épinière. Cette désignation offre des avantages significatifs, comme 10 ans d'exclusivité sur le marché après approbation, l'accès à des subventions et des coûts réduits pour des conseils scientifiques. La thérapie cible environ 20 000 nouveaux cas de blessures aiguës de la moelle épinière chaque année dans l'Union européenne. ExoPTEN utilise des vésicules extracellulaires dérivées de cellules souches mésenchymateuses chargées de siRNA ciblant la protéine PTEN pour promouvoir la régénération nerveuse et le rétablissement fonctionnel. Cette désignation fait suite à une reconnaissance similaire de la FDA, renforçant la position de marché mondial de NurExone.
NurExone Biologic hat von der Europäischen Arzneimittel-Agentur (EMA) die Orphan Medicinal Product Designation für seine ExoPTEN-Therapie erhalten, die zur Behandlung akuter Rückenmarksverletzungen entwickelt wurde. Diese Auszeichnung bietet erhebliche Vorteile, darunter 10 Jahre Marktexklusivität nach der Genehmigung, Zugang zu Zuschüssen und reduzierte Kosten für wissenschaftliche Beratung. Die Therapie richtet sich an etwa 20.000 neue Fälle akuter Rückenmarksverletzungen jährlich in der Europäischen Union. ExoPTEN verwendet aus mesenchymalen Stammzellen gewonnene extrazelluläre Vesikel, die mit siRNA geladen sind, um das PTEN-Protein zu zielen und die Nervenregeneration und funktionelle Erholung zu fördern. Diese Auszeichnung folgt einer ähnlichen Anerkennung durch die FDA, die die globale Marktposition von NurExone stärkt.
- Secured EMA Orphan Medicinal Product Designation for ExoPTEN
- Gained 10 years of market exclusivity in Europe upon approval
- Access to grants and reduced costs for scientific advice
- Potential market of 20,000 new spinal cord injury cases annually in EU
- Additional financial incentives through EU tax credits
- Product still in pre-clinical phase, requiring extensive development
- No current revenue generation from ExoPTEN
TORONTO and HAIFA, Israel, Nov. 13, 2024 (GLOBE NEWSWIRE) -- NurExone Biologic Inc. (TSXV: NRX) (OTCQB: NRXBF) (Germany: J90) ("NurExone" or the "Company"), 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.”
About NurExone
NurExone Biologic Inc. is a TSX Venture Exchange (“TSXV”) and OTCQB listed pharmaceutical company that is developing a platform for biologically guided exosome-based therapies to be delivered, non-invasively, to patients who have suffered Central Nervous System injuries. The Company’s first product, ExoPTEN for acute spinal cord injury, was proven to recover motor function in
For additional information and a brief interview, please watch Who is NurExone?, visit www.nurexone.com or follow NurExone on LinkedIn, Twitter, Facebook, or YouTube.
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1 Jazayeri, S. B., Safdarian, M., Zadegan, S. A., Ghodsi, Z., & Rahimi-Movaghar, V. (2023). Incidence of traumatic spinal cord injury worldwide: A systematic review, data integration, and update. World Neurosurgery: X, 18, 100171. https://doi.org/10.1016/j.wnsx.2023.100171
For more information, please contact:
Dr. Lior Shaltiel
Chief Executive Officer and Director
Phone: +972-52-4803034
Email: info@nurexone.com
Oak Hill Financial Inc.
2 Bloor Street, Suite 2900
Toronto, Ontario M4W 3E2
Investor Relations - Canada
Phone: +1-647-479-5803
Email: info@oakhillfinancial.ca
Dr. Eva Reuter
Investor Relations - Germany
Phone: +49-69-1532-5857
Email: e.reuter@dr-reuter.eu
Allele Capital Partners
Investor Relations - US
Phone: +1 978-857-5075
Email: aeriksen@allelecapital.com
FORWARD-LOOKING STATEMENTS
This press release contains certain “forward-looking statements” that reflect the Company’s current expectations and projections about its future results. Wherever possible, words such as “may”, “will”, “should”, “could”, “expect”, “plan”, “intend”, “anticipate”, “believe”, “estimate”, “predict” or “potential” or the negative or other variations of these words, or similar words or phrases, have been used to identify these forward-looking statements. Forward-looking statements in this press release include, but are not limited to, statements relating to: the receipt of the Orphan Medicinal Product Designation having the intended benefits and incentives on the Company and its business as set out herein; the Company entering the European market and bringing its products to patients across Europe; the Company preparing to advance into clinical trials; the Company working with the EMA and other agencies to accelerate the development of ExoPTEN; and the NurExone platform technology offering novel solutions to drug companies interested in non-invasive targeted drug delivery for other indications.
These statements reflect management’s current beliefs and are based on information currently available to management as at the date hereof. In developing the forward-looking statements in this press release, we have applied several material assumptions, including: the receipt of the Orphan Medicinal Product Designation having the intended benefits and incentives on the Company and its business as set out herein; the Company will enter the European market and bring its products to patients across Europe; the Company will advance into clinical trials; the Company will work with the EMA and other agencies to accelerate the development of ExoPTEN; and the NurExone platform technology will offer novel solutions to drug companies interested in non-invasive targeted drug delivery for other indications.
Forward-looking statements involve significant risk, uncertainties and assumptions. Many factors could cause actual results, performance or achievements to differ materially from the results discussed or implied in the forward-looking statements. These risks and uncertainties include, but are not limited to risks related to: the receipt of the Orphan Medicinal Product Designation not having the intended benefits and incentives on the Company and its business as set out herein; the Company not entering the European market and bringing its products to patients across Europe; the Company not advancing into clinical trials; the Company not working with the EMA and other agencies to accelerate the development of ExoPTEN; the NurExone platform technology not offering novel solutions to drug companies interested in non-invasive targeted drug delivery for other indications; and the risks discussed under the heading “Risk Factors” on pages 29 to 36 of the Company’s Annual Information Form dated March 30, 2023, a copy of which is available under the Company’s SEDAR+ profile at www.sedarplus.ca. These factors should be considered carefully, and readers should not place undue reliance on the forward-looking statements. Although the forward-looking statements contained in this press release are based upon what management believes to be reasonable assumptions, the Company cannot assure readers that actual results will be consistent with these forward-looking statements. These forward-looking statements are made as of the date of this press release, and the Company assumes no obligation to update or revise them to reflect new events or circumstances, except as required by law.
Neither TSXV nor its Regulation Services Provider (as that term is defined in the policies of the TSXV) accepts responsibility for the adequacy or accuracy of this release.
FAQ
What benefits did NurExone (NRXBF) receive from the EMA Orphan designation?
How many new spinal cord injury cases occur annually in the EU for NurExone's (NRXBF) ExoPTEN therapy?
How does NurExone's (NRXBF) ExoPTEN therapy work for spinal cord injury?
