Planned Phase 3b trial Designed to Confirm Efficacy and Safety of NurOwn in mild-to-moderate ALS patients

计划进行的3b期试验旨在确认NurOwn在轻度至中度ALS患者中的有效性和安全性

NEW YORK, Feb. 27, 2024 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of adult stem cell therapeutics for neurodegenerative diseases, today announced that it will present a poster (#M201) outlining the proposed design for a Phase 3b trial of NurOwn® in amyotrophic lateral sclerosis (ALS) at the MDA Clinical and Scientific Conference, on Monday, March 4 between 6-8 p.m.

2024年2月27日，纽约/PRNewswire/--BrainStorm Cell Therapeutics Inc.（纳斯达克股票代码：BCLI），神经退行性疾病成人干细胞治疗的领先开发商，今天宣布将在周一的MDA临床和科学会议上发布一张海报（#M201），概述NurOwn®肌萎缩侧索硬化症（ALS）3b期试验的拟议设计，3月4日下午6点至8点。

at the Hilton Orlando Hotel, Florida. The design is for a two-part, multicenter, Phase 3b study to assess the efficacy and safety of NurOwn in participants with ALS. Eligible patients will initially enter a 24-week randomized, double-blind, placebo-controlled period (Part A), followed by a 24-week open-label extension period (Part B).

这项设计是一项由两部分组成的多中心3b期研究，旨在评估NurOwn对ALS患者的疗效和安全性。符合条件的患者最初将进入24周的随机，双盲，安慰剂对照期（a部分），然后是24周的开放标签延长期（B部分）。

The entry criteria will enroll people living with mild-to-moderate ALS. The primary endpoint will be based on the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R)..

入选标准将招募轻度至中度ALS患者。主要终点将基于修订的肌萎缩侧索硬化功能评定量表（ALSFRS-R）。。

BrainStorm recently submitted a Special Protocol Assessment (SPA) request to the U.S. Food and Drug Administration (FDA) for this Phase 3b study of NurOwn. A SPA agreement would indicate concurrence by FDA with the adequacy and acceptability of the overall protocol design for the planned Phase 3b study, intended to support a future marketing application.

头脑风暴（BrainStorm）最近向美国食品和药物管理局（FDA）提交了一份特别方案评估（SPA）请求，以进行NurOwn的这项3b期研究。SPA协议将表明FDA同意计划的3b期研究的总体方案设计的充分性和可接受性，旨在支持未来的营销应用。

The details are currently under review by the FDA and the company plans to finalize the details following the receipt of input from the Agency..

FDA目前正在审查细节，该公司计划在收到FDA的意见后最终确定细节。。

'We are committed to doing everything we can to make NurOwn available to ALS patients in need and we are pleased to present an outline of our planned confirmatory Phase 3b trial,' said Chaim Lebovits, President and Chief Executive Officer of BrainStorm. 'Together with our advisors, we have designed a trial that we believe will generate robust and conclusive data.

BrainStorm总裁兼首席执行官夏姆·勒博维茨（ChaimLebovits）说：“我们致力于尽一切努力为有需要的ALS患者提供NurOwn，我们很高兴为我们计划的3b期确认试验提供一个大纲。”我们与我们的顾问一起设计了一个试验，我们相信它将产生可靠和确凿的数据。

Our goal is to develop an efficacious treatment option that, if approved, can help patients and generate value for our stakeholders.'.

我们的目标是开发一种有效的治疗方案，如果获得批准，可以帮助患者并为我们的利益相关者创造价值。”。

Bob Dagher, MD, commented, 'We have given careful consideration to a Phase 3b trial that will conclusively demonstrate the efficacy and safety of NurOwn and support a new NDA.  Our previous studies have provided us with valuable insight into the NurOwn treatment effect. We believe that by incorporating these learnings into the design of a confirmatory study we can maximize the prospects of a successful outcome.

医学博士鲍勃·达格（BobDagher）评论道：“我们已经仔细考虑了一项3b期试验，该试验将最终证明NurOwn的有效性和安全性，并支持新的NDA。我们之前的研究为我们提供了对NurOwn治疗效果的宝贵见解。我们相信，通过将这些学习纳入验证性研究的设计中，我们可以最大限度地提高成功结果的前景。

The planned study will enroll mild-to-moderate ALS patients, as we believe that this is the appropriate patient population in which the course of the disease can be accurately measured using ALSFRS-R. We are grateful for the FDA's ongoing engagement and look forward to its feedback and to finalizing the SPA.'      .

计划中的研究将招募轻度至中度ALS患者，因为我们认为这是使用ALSFRS-R可以准确测量疾病进程的合适患者人群。我们感谢FDA的持续参与，并期待其反馈和完成SPA。”。

The abstract for this presentation can be viewed here. Brainstorm will post a copy of the poster to its corporate website at the conclusion of the presentation.

可以在此处查看此演示文稿的摘要。演讲结束后，头脑风暴将在其公司网站上发布海报副本。

Special Protocol Assessment    A Special Protocol Assessment (SPA) is a process in which drug developers may ask to meet with the FDA to reach agreement on the design and size of certain clinical trials to determine if they adequately address scientific and regulatory requirements for a study that could support marketing approval.

特别方案评估特别方案评估（SPA）是一个过程，在这个过程中，药物开发人员可能会要求与FDA会面，就某些临床试验的设计和规模达成协议，以确定它们是否充分满足可能支持上市批准的研究的科学和监管要求。

An SPA agreement indicates concurrence by FDA with the adequacy and acceptability of specific critical elements of overall protocol design for a study intended to support a future marketing application. These elements are critical to ensuring that the trial conducted under the protocol can be considered an adequate and well-controlled study that can support marketing approval.

SPA协议表明FDA同意总体方案设计的特定关键要素的充分性和可接受性，以支持未来的营销应用。这些因素对于确保根据协议进行的试验可以被认为是一项充分且控制良好的研究，可以支持营销批准至关重要。

Feedback on these issues provides the greatest benefit to companies in planning late-phase development strategy. An SPA agreement does not indicate FDA concurrence on every protocol detail..

对这些问题的反馈为公司规划后期发展战略提供了最大的好处。SPA协议并不表示FDA同意每个协议细节。。

About 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.

关于BrainStorm Cell Therapeutics Inc.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            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.

关于前瞻性声明的通知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 BLA PDUFA行动日期的时间，NurOwn作为治疗ALS的疗法的临床发展，NurOwn对患者的未来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”等词语或其他类似词语的否定词来识别，尽管并非所有前瞻性声明都包含这些词语。

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，以及表格10-K上的头脑风暴年度报告和表格10-Q上的季度报告中详述的其他因素，请访问http://www.sec.gov.

These factors should be considered care.

应谨慎考虑这些因素。

CONTACT:

联系人：

Media: Lisa GuitermanPhone: +1 202-330-3431[email protected]

媒体：Lisa GuitermanPhone：+1 202-330-3431[受电子邮件保护]

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SOURCE BrainStorm Cell Therapeutics Inc.

来源：BrainStorm Cell Therapeutics Inc。