NurExone Biologic Receives FDA Orphan-Drug Designation, Accelerating Development of ExoPTEN Therapy for Acute Spinal Cord Injury Treatment
NurExone Biologic Receives FDA Orphan-Drug Designation, Accelerating Development of ExoPTEN Therapy for Acute Spinal Cord Injury Treatment
TORONTO and HAIFA, Israel, Oct. 30, 2023 (GLOBE NEWSWIRE) -- 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 limitedi.
多伦多和以色列海法,2023年10月30日(Global Newswire)--领先的生物技术公司NurExone生物公司(多伦多证券交易所股票代码:NRX)(FSE:J90)(NRX.V)(以下简称公司)欣喜若狂地宣布,美国食品和药物管理局(FDA)已批准其ExoPTEN疗法获得孤儿药物称号(ODD),认识到这种突破性的再生疗法治疗急性脊髓损伤的潜力,这种疾病的有效治疗方法有限我。
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 Statesii. 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.
孤儿药物的指定为制药公司开发治疗罕见疾病的药物提供了重大好处,即那些在美国影响不到20万人的药物II。这些好处包括市场排他性、财政激励、监管援助和对药物开发的支持。总体而言,该指定激励和支持某些治疗的开发,增加患者获得治疗的机会。
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.
获得孤儿药物称号对公司来说是一个重要的里程碑。该设计包括使用间充质干细胞(MSC)来源的小细胞外小泡(EV),装载针对磷酸酶和紧张素同源蛋白(PTEN)蛋白的短和修改的干扰RNA(SiRNA),用于治疗急性脊髓损伤,如该公司正在开发的ExoPTEN药物中实施的那样。
"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.
NurExone生物公司首席执行官沙尔蒂尔博士说:“孤儿药物的指定有望简化我们的上市流程,缩短我们的监管程序,为公司节省数百万美元,并提供宝贵的市场独占性。我们感谢正式承认我们的治疗方法对急性脊髓损伤患者的生活有潜在影响。”
About NurExone Biologic Inc.
关于NurExone生物公司
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.
NurExone生物公司是一家在多伦多证券交易所上市的制药公司,正在开发一个生物引导的体外治疗平台,以非侵入性的方式向遭受创伤性脊髓损伤的患者提供治疗。体外疗法在以色列理工学院的动物研究中得到了概念性的证明。通过其全资子公司NurExone生物有限公司,NurExone正在将这种治疗转化为人类,该公司拥有来自Technion和特拉维夫大学的独家全球许可证,用于该技术的开发和商业化。
For additional information, please visit or follow NurExone on LinkedIn, Twitter, Facebook, or YouTube.
欲了解更多信息,请访问或跟随NurExone OnLinkedIn,推特,Facebook,或YouTube。
For more information, please contact:
如需更多信息,请联系:
Dr. Lior Shaltiel
Chief Executive Officer and Director
Phone: +972-52-4803034
Email: info@nurexone.com
Lior Shaltiel博士
董事首席执行官兼首席执行官
电话:+972-52-4803034
电子邮件:info@nurexone.com
Thesis Capital Inc.
Investment Relation - Canada
Phone: +1 905-347-5569
Email: IR@nurexone.com
论文资本公司。
投资关系-加拿大
电话:+1905-347-5569
电子邮件:ir@nurexone.com
Dr. Eva Reuter
Investment Relation - Germany
Phone: +49-69-1532-5857
Email: e.reuter@dr-reuter.eu
伊娃·罗伊特博士
投资关系-德国
电话:+49-69-1532-5857
电子邮件:e.reuter@dr-reur.eu
FORWARD-LOOKING STATEMENTS
前瞻性陈述
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 Company's ExoTherapy drug, ExoPTEN and the potential benefits to the Company of the orphan-drug designation of ExoPTEN. These statements reflect management's current beliefs and are based on information currently available to management as at the date hereof.
本新闻稿包含某些“前瞻性陈述”,这些陈述反映了公司目前对其未来业绩的预期和预测。只要有可能,“可能”、“将”、“应该”、“可能”、“预期”、“计划”、“打算”、“预期”、“相信”、“估计”、“预测”或“潜在”或这些词语的否定或其他变体,或类似的词语或短语,都被用来识别这些前瞻性表述。本新闻稿中的前瞻性陈述包括但不限于与该公司的外部治疗药物ExoPTEN有关的陈述以及指定孤儿药物对公司的潜在好处ExoPTEN的基因。这些陈述反映了管理层目前的信念,并基于截至本文发布之日管理层目前掌握的信息。
In developing the forward-looking statements in this press release, we have applied several material assumptions, including our ability to retain key personnel, our ability to continue investing in research and development, our ability to secure available funding and to continue as a going concern, our ability to meet the post-approval requirements in respect of the orphan-drug designation, the general business and economic conditions of the industries and countries in which we operate, that the Company's operations in Israel will not be material disrupted by unrest in Israel and the Israel-Hamas War, our ability to execute on our business strategy, that there will be certain amount of demand for the Company's potential product, inflation will remain stable, and that the results of our studies reflect results that can be extrapolated.
在制定本新闻稿中的前瞻性陈述时,我们应用了几个重要的假设,包括我们留住关键人员的能力,我们继续投资研发的能力,我们获得可用资金并继续作为一家持续经营的公司的能力,我们满足审批后要求的能力关于孤儿药物的名称,我们经营的行业和国家的一般商业和经济状况,公司在以色列的业务不会受到以色列动乱和以色列-哈马斯战争的实质性干扰,我们执行业务战略的能力,对公司潜在产品的一定需求,通货膨胀将保持稳定,我们的研究结果反映了可以推断的结果。
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 Company not being able to demonstrate clinical superiority of its ExoTherapy drug, ExoPTEN, Company's early stage of development, lack of revenues to date, government regulation, market acceptance for its products, rapid technological change, dependence on key personnel, protection of the Company's intellectual property, dependence on the Company's strategic partners, the effect of the Israel-Hamas War on the Company's operations 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 . 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.
前瞻性陈述涉及重大风险、不确定性和假设。许多因素可能导致实际结果、业绩或成就与前瞻性陈述中讨论或暗示的结果大相径庭。这些风险和不确定性包括,但不限于,与公司不是存在能够展示ITS的临床优势体外治疗药物ExoPTEN,公司的早期发展阶段,到目前为止的收入不足,政府监管,其产品的市场接受度,快速的技术变化,对关键人员的依赖,对公司知识产权的保护,对公司战略的依赖以色列-哈马斯战争对公司运营的影响,以及在公司日期为2023年3月30日的年度信息表格第29至36页“风险因素”标题下讨论的风险,该表格的副本可在公司的SEDAR+简介下查阅,网址:。这些因素应仔细考虑,读者不应过分依赖前瞻性陈述。尽管本新闻稿中包含的前瞻性陈述是基于管理层认为合理的假设,但公司不能向读者保证实际结果将与这些前瞻性陈述一致。这些前瞻性陈述是截至本新闻稿发布之日作出的,除非法律另有要求,否则公司没有义务更新或修改这些陈述以反映新的事件或情况。
Neither TSX Venture Exchange nor its Regulation Services Provider (as that term is defined in the policies of the TSX Venture Exchange) accepts responsibility for the adequacy or accuracy of this release.
多伦多证券交易所风险交易所及其监管服务提供商(该术语在多伦多证券交易所的政策中定义)均不对本新闻稿的充分性或准确性承担责任。
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