The data will be presented at the Annual ALS Drug Development Summit, which is focused on identifying transformative ALS targets, seeking translational biomarkers and propelling more clinical approvals

这些数据将在年度ALS药物开发峰会上公布，该峰会的重点是确定变革性ALS目标，寻找转化生物标志物并推动更多的临床批准

NEW YORK, May 20, 2024 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of adult stem cell therapeutics for neurodegenerative diseases, will present new biomarker data suggesting that ALS patients may benefit from longer-term treatment with debamestrocel (NurOwn®).

2024年5月20日，纽约/PRNewswire/--BrainStorm Cell Therapeutics Inc.（纳斯达克：BCLI），一家针对神经退行性疾病的成人干细胞疗法的领先开发商，将提供新的生物标志物数据，表明ALS患者可能受益于长期使用debamestrocel（NurOwn®）治疗。

The Company will share the data with an international audience of patient advocacy groups, physicians, research organizations, industry representatives, key thought leaders and decision makers dedicated to ALS research at The 3rd Annual ALS Drug Development Summit, to take place May 21 to 23, 2024 in Boston MA.

该公司将在2024年5月21日至23日于马萨诸塞州波士顿举行的第三届年度ALS药物开发峰会上，与致力于ALS研究的患者倡导团体、医生、研究组织、行业代表、关键思想领袖和决策者的国际观众分享数据。

Stacy Lindborg PhD will deliver a presentation on new biomarker data from the NurOwn Expanded Access Program (EAP) along with data from the Phase 3 trial..

Stacy Lindborg博士将介绍NurOwn扩展访问计划（EAP）的新生物标志物数据以及3期试验的数据。。

'We are very pleased to share these important outcome data from the NurOwn EAP with the ALS community,' said Dr. Stacy Lindborg, Member of BrainStorm's Board of Directors. 'The data demonstrate a consistent reduction of neurofilament light (NfL) from baseline among participants who were randomized to receive NurOwn in the Phase 3 study.

BrainStorm董事会成员斯泰西·林德伯格（StacyLindborg）博士说，我们很高兴与ALS社区分享纽隆EAP的这些重要成果数据数据表明，在3期研究中随机接受NurOwn治疗的参与者中，神经丝光（NfL）从基线开始持续减少。

This reduction in NfL observed during the randomized Phase 3 trial, as well as in the subsequent EAP periods, indicate that patients treated with NurOwn during the Phase 3 study see benefits from the extended treatment. Furthermore, participants initially randomized to placebo in Phase 3 and were later treated with NurOwn during the EAP showed stabilization in Period 1 of the EAP, followed by reductions in NfL in Period 2 over their Phase 3 baseline levels.

在随机3期试验以及随后的EAP期间观察到的NfL减少表明，在3期研究期间接受NurOwn治疗的患者从延长治疗中获益。此外，参与者最初在第3阶段随机接受安慰剂治疗，后来在EAP期间接受NurOwn治疗，在EAP的第1阶段表现出稳定，随后在第2阶段的NfL比第3阶段的基线水平降低。

We looked forward to confirming this finding in the planned Phase 3b study.'.

我们期待着在计划的3b期研究中证实这一发现。”。

Presentation Title: Promising Longer-Term Biomarker Data from NurOwn Program in ALS: Spotlight on NfL in EAP Extension Cohort

演讲标题：来自ALS NurOwn计划的有希望的长期生物标志物数据：EAP扩展队列中NfL的焦点

Speaker: Stacy Lindborg, PhD

演讲者：Stacy Lindborg博士

Date/ time: May 22, 9am ET

日期/时间：美国东部时间5月22日上午9点

Location: Hyatt Regency, Boston MA

地点：马萨诸塞州波士顿凯悦酒店

Highlights

亮点

A fixed sample of participants in the Phase 3 NurOwn trial, who met eligibility criteria, had the opportunity to enroll in an FDA approved Expanded Access Program (EAP). The EAP was conducted over two periods of 28 weeks each, during which participants could receive a total of 6 doses of NurOwn, 3 doses of NurOwn in each period.

符合资格标准的3期NurOwn试验参与者的固定样本有机会参加FDA批准的扩展获取计划（EAP）。EAP在两个为期28周的时期内进行，在此期间，参与者可以接受总共6剂NurOwn，每个时期3剂NurOwn。

All participants in the EAP received NurOwn, including participants randomized to placebo in the Phase 3 trial..

EAP的所有参与者都接受了NurOwn，包括在3期试验中随机接受安慰剂的参与者。。

13 Cerebrospinal fluid (CSF) samples were drawn during Phase 3 trial and the EAP. Levels of neurofilament light chain (NfL) in CSF were monitored during Phase 3 and subsequent EAP periods. NfL is an important biomarker marker in ALS, which measures neurodegeneration and neural cell death. NfL values has been shown through published studies to be associated with clinical progression, and treatment driven reductions in NfL were the basis for a recent ALS drug approval..

在3期试验和EAP期间抽取了13份脑脊液（CSF）样本。在第3阶段和随后的EAP期间监测CSF中神经丝轻链（NfL）的水平。NfL是ALS中重要的生物标志物，可测量神经变性和神经细胞死亡。已发表的研究表明，NfL值与临床进展有关，治疗驱动的NfL降低是最近ALS药物批准的基础。。

Recently published NfL data from the Phase 3 trial showed that participants treated with NurOwn had an 11% decline from baseline in NfL. Participants randomized to placebo had NfL values similar to baseline across the trial. (Lindborg et al. Muscle and Nerve 2024).

最近公布的来自第三阶段试验的NfL数据显示，接受NurOwn治疗的参与者在NfL中比基线下降了11%。随机接受安慰剂治疗的参与者在整个试验中的NfL值与基线相似。（Lindborg等人，肌肉和神经2024）。

10 trial participants who completed Phase 3 were enrolled in the EAP. 8 participants completed EAP period 1, and 6 completed EAP period 2. The participants in the EAP had lower NfL values at Phase 3 baseline as compared to the entire Phase 3 population.

完成第3阶段的10名试验参与者参加了EAP。8名参与者完成了EAP第1阶段，6名参与者完成了EAP第2阶段。与整个3期人群相比，EAP参与者在3期基线时的NfL值较低。

The new EAP data to be presented at the ALS Summit showed that, for participants randomized to NurOwn, there was a 4% decrease from baseline in NfL in Phase 3 and a 27% and 36% decrease from baseline, at the ends of Period 1 and Period 2 of the EAP, respectively. These results suggest continual benefit from extended treatment of NurOwn..

将在ALS峰会上提交的新EAP数据显示，对于随机分配到纽隆的参与者，在EAP的第1阶段和第2阶段结束时，第3阶段NfL的基线下降了4%，比基线下降了27%和36%。这些结果表明，纽隆的长期治疗持续受益。。

For participants randomized to placebo and subsequently treated with NurOwn in the EAP, there was a 37% increase in NfL from baseline to study end in Phase 3. Following treatment with NurOwn in the EAP, the same patients had a 17% increase in NfL from baseline during Period 1 and a 5% decrease from baseline in NfL during Period 2..

对于随机接受安慰剂治疗并随后在EAP中接受NurOwn治疗的参与者，从基线到第3阶段研究结束，NfL增加了37%。在EAP中接受NurOwn治疗后，同样的患者在第1阶段的NfL比基线增加了17%，在第2阶段的NfL比基线减少了5%。。

BrainStorm hopes to confirm these results in the planned Phase 3b trial of NurOwn.

头脑风暴（BrainStorm）希望在NurOwn计划的3b期试验中证实这些结果。

Workshop and Panel

车间和面板

Antonio Trejo, VP of Regulatory Affairs at BrainStorm, will participate in a workshop Reinventing the Surrogate Landscape: Innovating Regulatory Acceptable Endpoints Most Meaningful to People Living with ALS at 2pm on May 21. The co-presenters in the workshop will be Christopher Ocampo, Senior Medical Director, AbbVie; Angela Genge, Director, ALS Centre of Excellence, McGill University; and Marjan Sepassi, Vice President, Medical Affairs, Clene Nanomedicine..

BrainStorm监管事务副总裁安东尼奥·特雷霍（AntonioTrejo）将于5月21日下午2点参加一个重塑替代景观的研讨会：创新监管可接受的终点，对ALS患者最有意义。研讨会的共同主持人将是艾伯维高级医学主任克里斯托弗·奥坎波；安吉拉·根奇，麦吉尔大学ALS卓越中心主任；和Clene Nanomedicine医疗事务副总裁Marjan Sepassi。。

Mary Kay Turner, Senior Vice President, Global Patient Advocacy & Public Affairs and Kylan Morris Senior Manager, Patient Advocacy at Brainstorm, will participate in a panel discussion Leveraging Insight from Real-Life Experiences: How Do People Living with ALS Evaluate Which Trial They Want to be Enrolled in? at 4pm on May 22..

全球患者倡导与公共事务高级副总裁玛丽·凯·特纳（MaryKayTurner）和头脑风暴（Brainstorm）患者倡导高级经理凯兰·莫里斯（KylanMorris）将参加一次小组讨论，利用现实生活经验的洞察力：ALS患者如何评估他们想要参加哪项试验？5月22日下午4点。。

About NurOwn®

关于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 harvested from each person with ALS and are manufactured using an innovative and proprietary process, to secrete neurotrophic factors to target specific neurodegenerative diseases.

NurOwn®技术平台（自体MSC-NTF细胞）代表了一种有前途的研究性治疗方法，用于靶向神经退行性疾病中重要的疾病途径。MSC-NTF细胞是从每个患有ALS的人身上收获的，并使用创新和专有的方法制造，以分泌神经营养因子以靶向特定的神经退行性疾病。

The lead program for NurOwn is for the treatment of ALS. BrainStorm's long-term commitment to ALS is demonstrated in preclinical research and a series of clinical studies, all of which have been published in peer-reviewed journals..

NurOwn的主要项目是治疗ALS。BrainStorm对ALS的长期承诺已在临床前研究和一系列临床研究中得到证实，所有这些研究均已在同行评审期刊上发表。。

The NurOwn clinical program has generated valuable insights into the pathology of ALS, as well as disease progression and treatment. Since the initial Phase 3 readout, BrainStorm has shared the full dataset through rigorous peer-reviewed analysis, including: quantification of Floor Effect, which had been noted, but never before explored in depth; evaluation of multiple pre-specified biomarkers, collected at seven different points across 20 weeks during the trial, allowing a longitudinal view; and analysis of genetic data, which represents one of the first ALS trials to prospectively invoke pharmacogenomic analysis of clinical outcome, offering great promise for the development of future treatments for ALS..

NurOwn临床计划对ALS的病理学以及疾病进展和治疗产生了宝贵的见解。自最初的第三阶段读数以来，头脑风暴通过严格的同行评审分析共享了完整的数据集，包括：地板效应的量化，这已经被注意到，但之前从未深入探讨过；评估在试验期间20周内在七个不同点收集的多个预先指定的生物标志物，允许纵向观察；和遗传数据分析，这是第一个前瞻性调用药物基因组学分析临床结果的ALS试验之一，为ALS未来治疗的发展提供了巨大的希望。。

About BrainStorm Cell Therapeutics Inc.

关于BrainStorm Cell Therapeutics Inc。

BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. BrainStorm holds the rights to clinical development and commercialization of the NurOwn® technology platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement.

BrainStorm Cell Therapeutics Inc.是用于衰弱神经退行性疾病的创新性自体成体干细胞疗法的领先开发商。BrainStorm拥有通过独家全球许可协议生产自体MSC-NTF细胞的NurOwn®技术平台的临床开发和商业化权利。

Autologous MSC-NTF cells have received Orphan Drug designation status from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm has completed a Phase 3 trial in ALS (NCT03280056); this trial investigated the safety and efficacy of repeat-administration of autologous MSC-NTF cells and was supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989), and another grant from the ALS Association and I AM ALS.

自体MSC-NTF细胞已获得美国食品和药物管理局（FDA）和欧洲药品管理局（EMA）的孤儿药物指定状态，用于治疗肌萎缩侧索硬化症（ALS）。头脑风暴已完成ALS的3期试验（NCT03280056）；该试验研究了重复施用自体MSC-NTF细胞的安全性和有效性，并得到了加利福尼亚再生医学研究所（CIRM CLIN2-0989）的资助，以及ALS协会和我是ALS的另一笔资助。

BrainStorm completed under an investigational new drug application a Phase 2 open-label multicenter trial (NCT03799718) of autologous MSC-NTF cells in progressive MS and was supported by a grant from the National MS Society (NMSS)..

头脑风暴在一项研究性新药申请下完成了一项进行性MS中自体MSC-NTF细胞的2期开放标签多中心试验（NCT03799718），并得到了美国国家MS学会（NMSS）的资助。。

Notice Regarding Forward-Looking Statements

关于前瞻性声明的通知

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), ADCOM meeting related to NurOwn, the timing of a PDUFA action date for the BLA for NurOwn, 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.

本新闻稿包含具有重大风险和不确定性的“前瞻性声明”，包括与美国食品和药物管理局（FDA）的会议，特别协议评估（SPA），与NurOwn相关的ADCOM会议，NurOwn的BLA PDUFA行动日期的时间，NurOwn作为ALS治疗药物的临床开发，NurOwn未来对患者的可用性以及头脑风暴的未来成功。

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.

除历史事实声明外，本新闻稿中的所有声明均为前瞻性声明。本新闻稿中包含的前瞻性陈述可以通过使用诸如“预期”、“相信”、“沉思”、“可能”、“估计”、“期望”、“打算”、“寻求”、“可能”、“可能”、“计划”、“潜力”、“预测”、“项目”、“目标”、“目标”、“应该”、“将会”或这些词语的否定词或其他类似表达来识别，尽管并非所有前瞻性陈述都包含这些词语。

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 http://www.sec.gov.

前瞻性陈述基于头脑风暴当前的预期，并受到难以预测的固有不确定性、风险和假设的影响。这些潜在的风险和不确定性包括但不限于管理层成功实现其目标的能力、集思广益筹集额外资本的能力、集思广益持续经营的能力、纽隆未来监管批准的前景、集思广益未来与FDA的互动是否会产生成效，以及集思广益10-K表格年度报告和10-Q表格季度报告中详述的其他因素http://www.sec.gov.

These factors should be considered carefully, and readers should not place undue reliance on B.

应仔细考虑这些因素，读者不应过度依赖B。

CONTACTS

联系人

Media:

媒体：

Lisa Guiterman

Lisa Guiterman

Phone: +1 202-330-3431

电话：+1 202-330-3431

lisa.guiterman@gmail.com

lisa.guiterman@gmail.com

IR:

红外光谱：

Michael Wood

迈克尔·伍德

Phone: +1 646-597-6983

电话：+1 646-597-6983

mwood@lifesciadvisors.com

mwood@lifesciadvisors.com

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SOURCE BrainStorm Cell Therapeutics Inc.

来源：BrainStorm Cell Therapeutics Inc。

Company Codes: NASDAQ-SMALL:BCLI

公司代码：NASDAQ-SMALL：BCLI