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5月19日，BrainStorm 宣布，美国食品药品监督管理局 (FDA) 已批准公司启动 NurOwn® 针对 ALS 的 Phase 3b 临床试验。这项即将启动的 Phase 3b 试验的设计细节已经与 FDA 在一项 特殊方案评估 (SPA) 下达成一致。这意味着研究的终点和统计方法被认为足以支持未来的生物制品许可申请 (BLA) 提交。截止发稿前，公司股价大涨40%。肌萎缩侧索硬化症（ALS），俗称渐冻症，是一种神经退行性疾病，选择性地导致大脑皮层、脑干和脊髓运动神经元的退化。随着运动神经元的死亡，患者逐渐丧失肌肉力量和运动功能，最终大多数患者在诊断后的3到5年内死亡。目前，还没有治疗方法能够阻止或逆转 ALS 的疾病进展。BrainStorm 作为一家专注于神经退行性疾病成人干细胞疗法，其专有的 NurOwn® 技术平台，采用了一种创新的自体细胞疗法方法。简单来说，它利用患者自身的骨髓来源的间充质干细胞 (MSCs)。这些 MSCs 在体外经过专有技术处理和分化，使其能够分泌高水平的神经营养因子 (NTFs)。这些经过分化的细胞被称为 MSC-NTF 细胞，然后被收集起来准备注射回患者体内。MSC-NTF 细胞未经过基因改造。MSC-NTF 细胞被认为能够通过靶向神经退行性疾病中的重要疾病通路，有效传递多种 NTFs 和免疫调节细胞因子，直接作用于损伤部位。这有望激活神经保护和免疫调节通路，从而减缓或稳定疾病进展。迄今为止，已有大约 170 名 ALS 患者在四项已完成的临床试验中接受了 Autologous MSC-NTF 细胞的治疗。这些试验包括在以色列进行的两项开放标签试验以及在美国进行的两项多中心、双盲、安慰剂对照试验。总体而言，试验结果表明，Autologous MSC-NTF 细胞的给药是安全且耐受性良好的。在早期的试验中，观察到疾病进展速度的改善和生物标志物的积极变化。虽然在美国完成的 Phase 3a 试验 (NCT03280056) 在总体患者人群中显示，病情较轻患者的预先指定分析显示出了有意义的临床结果，并在神经炎症、神经保护和神经退化方面显示出强大的生物标志物结果。即将启动的Phase 3b 试验计划招募约200名参与者，将在美国领先的学术医疗中心进行。试验设计分为两个部分：首先是一个为期 24 周的随机、双盲、安慰剂对照阶段 (A 部分)，随后是一个为期 24 周的开放标签扩展阶段 (B 部分)，在此阶段所有参与者都将接受 NurOwn® 治疗。参与者将按 1:1 的比例随机分配到 NurOwn® 组和安慰剂组。NurOwn® 或安慰剂将每八周通过鞘内注射给药。该试验的目标人群是处于疾病早期阶段的 ALS 参与者，这是基于之前 Phase 3a 试验中对病情较轻患者的分析显示出有意义的临床结果的发现。试验的主要终点是从基线到第 24 周时，ALS 功能评定量表修订版 (ALSFRS-R) 分数的变化。除了 ALS，BrainStorm 的管线还包括在进展性多发性硬化症 (MS) 中的研究（已完成 Phase 2 开放标签试验），以及在亨廷顿病 (HD)、帕金森病 (PD) 和自闭症谱系障碍 (ASD) 等其他疾病中的临床前研究。公司还在推进一个专有的异体外泌体平台。参考资料：公司官网推文用于传递知识，如因版权等有疑问，请于本文刊发30日内联系医药速览。原创内容未经授权，禁止转载至其他平台。有问题可发邮件至yong_wang@pku.edu.cn获取更多信息。©2021 医药速览 保留所有权利往期链接“小小疫苗”养成记 | 医药公司管线盘点 人人学懂免疫学| 人人学懂免疫学（语音版）综述文章解读 | 文献略读 | 医学科普|医药前沿笔记PROTAC技术| 抗体药物| 抗体药物偶联-ADC核酸疫苗 | CAR技术| 化学生物学温馨提示医药速览公众号目前已经有近12个交流群（好学，有趣且奔波于医药圈人才聚集于此）。进群加作者微信（yiyaoxueshu666）或者扫描公众号二维码添加作者，备注“姓名/昵称-企业/高校-具体研究领域/专业”，此群仅为科研交流群，非诚勿扰。简单操作即可星标⭐️医药速览，第一时间收到我们的推送①点击标题下方“医药速览”  ②至右上角“...”  ③点击“设为星标

Conference call and webcast rescheduled for 8:30 a.m. Eastern Time on Monday, May 19 NEW YORK, May 15, 2025 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of adult stem cell therapeutics for neurodegenerative diseases, today announced financial results for the first quarter ended March 31, 2025, and provided a corporate update. "BrainStorm continues to make meaningful progress in advancing our NurOwn® development program. Our priority continues to be initiation of a Phase 3b trial, designed to confirm the product's efficacy in early stage ALS patients and support a new BLA," said Chaim Lebovits, President and Chief Executive Officer of BrainStorm. "We have a clear path forward and ongoing support from the ALS community. We recently submitted an amendment to our IND, which includes updated documentation that is essential for regulatory compliance and trial integrity. We are in the process of finalization clinical trial agreements with leading academic centers and completing other necessary steps on trial execution and manufacturing. We believe that NurOwn, if approved, has the potential to become a valuable treatment option for ALS patients. Our team is fully aligned and executing with discipline to position BrainStorm for success." Recent Highlights NurOwn (MSC-NTF) for ALS IND amendment on NurOwn submitted to FDA BrainStorm submitted an Investigational New Drug (IND) amendment to the U.S. Food and Drug Administration for NurOwn. This important milestone sets the stage for the initiation of the planned Phase 3b clinical trial, which has been designed in collaboration with the FDA under a Special Protocol Assessment (SPA). The trial will have a primary efficacy endpoint assessing changes in ALSFRS-R scores from baseline to week 24, and is designed to enroll approximately 200 ALS participants with early stage disease. Successful completion of the double blind part of the study (Part A) is expected to generate the clinical data to support a new BLA submission. Phase 3b trial listed on Clinicaltrials.gov. Details of the trial, known as ENDURANCE, are now available on ClinicalTrials.gov ID NCT06973629. Included is a list of 15 clinical trial sites that are expected to participate in the trial. NurOwn® data selected as Breakthrough Science for Presentation at ISCT 2025 Meeting The new pharmacogenomic were delivered in a oral presentation at the International Society for Cell & Gene Therapy (ISCT) 2025 Annual Meeting, in New Orleans. The data highlight the impact of the UNC13A genotype on clinical outcomes for ALS patients treated with NurOwn. The presentation was featured in the ISCT public announcement regarding the meeting, which indicated that the data were "carefully reviewed and selected by the ISCT 2025 Planning Faculty, to explore the latest breakthroughs in the clinical translation of Mesenchymal Stem/Stromal Cells and how they will shape the future of cell therapies." Biomarker insights supporting NurOwn's mechanism and clinical impact presented at the 2025 ALS Drug Development Summit BrainStorm's senior leadership team (Dr. Bob Dagher, Dr. Netta Blondheim-Shraga and May Kay Turner) participated in sessions at the summit that highlighted insights and expertise gained throughout the NurOwn® development program. These sessions included a presentation on cerebrospinal fluid (CSF) biomarker pathways associated with NurOwn treatment, including their relationship to clinical outcomes and disease heterogeneity in ALS. Financial Results for the First Quarter Ended March 31, 2025 Cash, cash equivalents, and restricted cash were approximately $1.8 million as of March 31, 2025. Research and development expenditures, net, for the quarter ended March 31, 2025 were $1.3 million, compared to $1.0 million for the quarter ended March 31, 2024. General and administrative expenses for the quarter ended March 31, 2025 were approximately $1.8 million, compared to approximately $1.5 million for the quarter ended March 31, 2024. Net loss for the quarter ended March 31, 2025, was approximately $2.9 million, as compared to a net loss of approximately $3.4 million for the quarter ended March 31, 2024. Net loss per share for the three months ended March 31, 2025, and 2024 was $0.45 and $0.75, respectively. Conference Call and Webcast Monday, May 19, 2025, at 8:30 a.m. U.S. Eastern Time Participant Numbers: U.S. dial in: 888-506-0062 International: 973-528-0011 Participant Access Code: 621608 Webcast URL: The replay of the conference call can be accessed by dialing the numbers below and will be available for 14 days. Teleconference Replay Number: Toll Free: 877-481-4010 International: 919-882-2331 Passcode: 52457 About NurOwn® The NurOwn® technology platform (autologous MSC-NTF cells) represents a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors (NTFs). Autologous MSC-NTF cells are designed to effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. About BrainStorm Cell Therapeutics Inc. BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI) is a leading developer of autologous adult stem cell therapies for debilitating neurodegenerative diseases. The company's proprietary NurOwn® platform uses autologous mesenchymal stem cells (MSCs) to produce neurotrophic factor-secreting cells (MSC-NTF cells), designed to deliver targeted biological signals that modulate neuroinflammation and promote neuroprotection. NurOwn® is BrainStorm's lead investigational therapy for amyotrophic lateral sclerosis (ALS) and has received Orphan Drug designation from both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). A Phase 3 trial in ALS (NCT03280056) has been completed, and a second Phase 3b trial is set to launch under a Special Protocol Assessment (SPA) agreement with the FDA. The NurOwn clinical program has generated valuable insights into ALS disease biology, including pharmacogenomic response associated with the UNC13A genotype, biomarker data collected at seven longitudinal time points, and a comprehensive analysis of the "Floor Effect" — a critical challenge in measuring clinical outcomes in advanced ALS. BrainStorm has published its findings in multiple peer-reviewed journals. In addition to ALS, BrainStorm has completed a Phase 2 open-label multicenter trial (NCT03799718) of MSC-NTF cells in progressive multiple sclerosis (MS), supported by a grant from the National MS Society. BrainStorm is also advancing a proprietary, allogeneic exosome-based platform designed to deliver therapeutic proteins and nucleic acids. The company recently received a Notice of Allowance from the U.S. Patent and Trademark Office for a foundational patent covering its exosome technology, further strengthening BrainStorm's growing IP portfolio in this emerging area of regenerative medicine. To learn more, visit . Notice Regarding Forward-Looking Statements This press release contains "forward-looking statements" that are subject to substantial risks and uncertainties, including statements regarding meetings with the U.S. Food and Drug Administration (FDA), Special Protocol Assessment (SPA), the clinical development of NurOwn as a therapy for the treatment of ALS, the future availability of NurOwn to patients, and the future success of BrainStorm. All statements, other than statements of historical fact, contained in this press release are forward-looking statements. Forward-looking statements contained in this press release may be identified by the use of words such as "anticipate," "believe," "contemplate," "could," "estimate," "expect," "intend," "seek," "may," "might," "plan," "potential," "predict," "project," "target," "aim," "should," "will" "would," or the negative of these words or other similar expressions, although not all forward-looking statements contain these words. Forward-looking statements are based on BrainStorm's current expectations and are subject to inherent uncertainties, risks and assumptions that are difficult to predict. These potential risks and uncertainties include, without limitation, management's ability to successfully achieve its goals, BrainStorm's ability to raise additional capital, BrainStorm's ability to continue as a going concern, prospects for future regulatory approval of NurOwn, whether BrainStorm's future interactions with the FDA will have productive outcomes, and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available at . These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations, and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance, or achievements. CONTACTS Investors: Michael Wood Phone: +1 646-597-6983 [email protected] Media: Uri Yablonka, Chief Business Officer Phone: +1 917-284-2911 [email protected] The accompanying notes are an integral part of the consolidated financial statements. The accompanying notes are an integral part of the consolidated financial statements. Logo: SOURCE BrainStorm Cell Therapeutics Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED