Data presented by the MS team at Hadassah Medical Center, Jerusalem, led by Prof. Dimitrios Karussis, in the Americas Committee for Treatment and Research in Multiple Sclerosis Forum reveal consistent reductions in serum levels of both NfL and GFAP, the most reliable biomarkers for neurodegeneration and prediction of MS progression, in patients with progressive MS following treatment with NG-01 cells.

由Dimitrios Karussis教授领导的耶路撒冷哈达萨医学中心MS团队在美洲多发性硬化症治疗和研究委员会论坛上提供的数据显示，NfL和GFAP的血清水平持续下降，NfL和GFAP是神经退行性变和MS进展预测最可靠的生物标志物，用NG-01细胞治疗后进行性MS患者。

Improvement in physical and cognitive parameters of MS and in patient-reported outcomes, were also observed, following repeated treatments with NG-01

在用NG-01反复治疗后，还观察到MS的身体和认知参数以及患者报告的结局的改善

The FDA has cleared a Phase 2b, global, multi-center study with a double-blind, randomized, and placebo-controlled design to test the efficacy and safety of different doses of intrathecal administration of NG-01 in progressive MS patients

FDA已经批准了一项2b期全球多中心研究，采用双盲、随机和安慰剂对照设计，以测试不同剂量鞘内注射NG-01对进行性MS患者的疗效和安全性

ROCHESTER, N.Y., March 12, 2024 /PRNewswire/ -- NeuroGenesis Bio Inc., a clinical-stage biopharmaceutical company advancing innovative cell therapies to combat neuro-inflammation and demyelination in neurodegenerative diseases such as multiple sclerosis (MS) and Amyotrophic Lateral Sclerosis (ALS), announced today positive results from the interim analysis of the open-label extension period of a randomized, double-blind, placebo-controlled, Phase 2 clinical trial assessing the long-term effects of its lead asset, NG-01, on the two main serum biomarkers of MS activity and progression, on physical and cognitive assessments, and patient-reported outcomes in progressive MS.

纽约州罗切斯特，2024年3月12日/PRNewswire/--NeuroGenesis Bio Inc.，一家临床阶段的生物制药公司，推进创新细胞疗法，以对抗多发性硬化症（MS）和肌萎缩侧索硬化症（ALS）等神经退行性疾病中的神经炎症和脱髓鞘，今天宣布了一项随机，双盲，安慰剂对照的2期临床试验的开放标签延长期中期分析的积极结果，该试验评估了其主要资产NG-01对MS活动和进展的两种主要血清生物标志物的长期影响，身体和认知评估，以及患者报告的进展性MS结果。

The results were recently presented by the MS team at Hadassah Medical Center, Jerusalem, led by Prof. Dimitrios Karussis, at the Americas Committee for Treatment and Research in Multiple Sclerosis (ACTRIMS) Forum, held in West Palm Beach, Florida on March 2nd, 2024..

该结果最近由耶路撒冷哈达萨医学中心的MS团队在Dimitrios Karussis教授领导下于2024年3月2日在佛罗里达州西棕榈滩举行的美洲多发性硬化症治疗与研究委员会（ACTRIMS）论坛上发表。。

'There is a significant unmet medical need for progressive MS, which is a debilitating disease without effective treatment options,' said Prof.  Karussis, lead principal investigator of the study. 'We are therefore excited to see that NG-01 not only prevented the progression of the disease for a rather long period of up to two years, but also led to demonstrable improvement in patient condition measured by multiple parameters— clinical assessments, cognitive tests, and objective biomarkers.

该研究首席研究员卡鲁西斯教授说：“进展性多发性硬化症的医疗需求尚未得到满足，这是一种没有有效治疗选择的衰弱性疾病。”因此，我们高兴地看到，NG-01不仅在长达两年的相当长的时间内阻止了疾病的进展，而且还通过多种参数（临床评估，认知测试和客观生物标志物）测量了患者状况的明显改善。

Serum levels of NfL and GFAP were evaluated at repeated time points after each NG-01 treatment as the key biomarkers that correlate with neuroinflammation and neurodegeneration in progressive MS. We are encouraged to see that NG-01 treatment consistently reduced the levels of both biomarkers. Additionally, the positive effect on cognitive function after NG-01 treatment may point to a potential role of these cells in targeting compartmentalized CNS lesions, such as PRLs (paramagnetic lesions), that is to be tested in subsequent clinical trials.

在每次NG-01治疗后的重复时间点评估血清NfL和GFAP水平，作为与进行性MS中神经炎症和神经变性相关的关键生物标志物。我们鼓励看到NG-01治疗持续降低两种生物标志物的水平。此外，NG-01治疗后对认知功能的积极影响可能表明这些细胞在靶向区室化CNS病变（如PRL（顺磁性病变））中的潜在作用，这将在随后的临床试验中进行测试。

The extension study results underscore the possibility of a novel, safe, and long-acting therapy for stabilizing and/or reversing the progression of MS, especially in its progressive form, known as PIRA (Progression Independent of Relapse Activity), that advances 'silently' and is very resistant to the existing conventional medical treatments.'.

扩展研究结果强调了一种新的，安全的，长效的治疗方法的可能性，用于稳定和/或逆转MS的进展，特别是以其进展形式，称为PIRA（独立于复发活动的进展），其进展“无声”并且对现有的常规医学治疗具有很强的抵抗力。

'We are extremely pleased with these promising results of our NG-01 cells in patients with progressive MS,' said Tal Gilat, CEO of NeuroGenesis. 'Following the observations on the longitudinal decrease in NfL and GFAP, the FDA has cleared our Phase 2b, global, multi-center study with a double-blind, randomized, and placebo-controlled design to primarily test the efficacy and safety of different doses of intrathecal administration of NG-01 in progressive MS patients.

神经发生首席执行官塔尔吉拉特（TalGilat）说：“我们对NG-01细胞在进行性MS患者中取得的这些有希望的结果感到非常满意。”在观察到NfL和GFAP的纵向下降后，FDA通过双盲，随机和安慰剂对照设计，批准了我们的2b期全球多中心研究，主要测试不同剂量鞘内注射NG-01对进行性MS患者的疗效和安全性。

We are now preparing for the Phase 2 double-blinded study and have already initiated work with multiple leading hospitals around the world. We look forward to the continued development of this cutting-edge treatment that has shown promise not only in MS but also in ALS, and thus may offer hope for hundreds of thousands of patients worldwide who currently have no alternative medical solution.'.

我们现在正在准备第二阶段双盲研究，并已开始与世界各地多家领先医院合作。我们期待着这种尖端治疗的持续发展，这种治疗不仅在MS中而且在ALS中都显示出前景，因此可能为全球数十万目前没有替代医疗解决方案的患者提供希望。”。

The interim analysis was conducted on 23 out of 40 patients who were given at least two administrations of NG-01,in addition to their standard-of-care, 3-6 months apart with a follow up of 12-18 months. In this pre-specified interim analysis, safety, tolerability and various efficacy measurements— changes in EDSS and Functional systems, timed 25-feet (25FT) walking, cognitive functions, and changes in the serum levels of the biomarkers NfL (neurofilaments-light chains) and GFAP (that indicate neurodegeneration and neuroinflammation)— were assessed..

对40名患者中的23名进行了中期分析，这些患者除接受标准治疗外，还接受了至少两次NG-01治疗，间隔3-6个月，随访12-18个月。在这个预先指定的中期分析中，评估了安全性，耐受性和各种功效测量-EDS和功能系统的变化，定时25英尺（25英尺）行走，认知功能以及生物标志物NfL（神经丝轻链）和GFAP（表明神经变性和神经炎症）的血清水平变化。。

The main findings include:

主要发现包括：

Results show a significant reduction in serum levels of NfL and GFAP, the two most reliable biomarkers that are correlated with MS-activity, neurodegeneration and prediction of progression of the disease. The mean reduction in NfL was 33.2% in 20 patients from baseline to the last observation after 2nd dosing (p <0.0008; Wilcoxon matched-pair test), and the mean reduction in GFAP was 22% in 23 patients from baseline to the last observation after 2nd dosing (p <0.00008; Wilcoxon matched pair test)..

结果显示血清NfL和GFAP水平显着降低，这两种最可靠的生物标志物与MS活性，神经变性和疾病进展预测相关。从基线到第二次给药后最后一次观察，20例患者的NfL平均降低33.2%（p＜0.0008；Wilcoxon配对检验），23例患者的GFAP平均降低22%（p＜0.00008；Wilcoxon配对检验）。。

There was a significant improvement in walking ability as measured by 25-foot walking time (T25FW) in 16 patients after 12 months (p=0.031)

通过12个月后的25英尺步行时间（T25FW）测量，16名患者的步行能力有了显着改善（p=0.031）

The SDMT cognitive test was improved by a mean of ~3 degrees (p=0.0008). In addition, Patient reported outcomes (mental and fatigue questionaries) were all improved by 18-33% (p <0.05) from baseline to the last observation since the 2nd dose (>12 months).

SDMT认知测试平均提高了约3度（p=0.0008）。此外，自第二次给药（>12个月）以来，从基线到最后一次观察，患者报告的结果（精神和疲劳问卷）均改善了18-33%（p＜0.05）。

No serious adverse events were observed during the whole period of the trial. Minor adverse events included headache in 9 subjects and backache in 2 subjects, which were considered by the investigators, as procedure-related and resolved within 48 hours after the lumbar puncture.

在整个试验期间没有观察到严重的不良事件。轻微不良事件包括9名受试者的头痛和2名受试者的背痛，研究人员认为这与手术有关，并在腰椎穿刺后48小时内得到解决。

The Phase 2, randomized, double-blind, placebo-controlled, clinical trial (NCT02166021), which was performed by the Hadassah-MS team led by Prof. Karussis, Dr. Petrou and Dr. Kassis, assessed the safety, tolerability, and efficacy of transplantation of NG-01 in people with progressive MS. The trial demonstrated significant improvement and repair results which were published in BRAIN 2020 as the Editor's Choice.

由Karussis教授，Petrou博士和Kassis博士领导的Hadassah MS团队进行的2期随机，双盲，安慰剂对照临床试验（NCT02166021）评估了NG-01在进行性MS患者中移植的安全性，耐受性和有效性。该试验显示出显着的改善和修复结果，并作为编辑的选择发表在BRAIN 2020上。

The original study enrolled 48 participants with progressive MS who were randomized into 3 groups, receiving either an intrathecal (IT) or intravenous (IV) NG-01 injection, or a placebo injection. In the extension phase, patients were treated with 1-3 intrathecal injections of NG-01 at intervals of 3-6 months..

最初的研究招募了48名进行性MS患者，他们被随机分为3组，接受鞘内（IT）或静脉（IV）NG-01注射或安慰剂注射。在延长期，患者每隔3-6个月鞘内注射1-3次NG-01。。

About NG-01NG-01 is a proprietary population of autologous bone marrow-derived stem cells designed to secrete high levels of remyelinating and neurotrophic factors (NG-01) at the central nervous system site of injury. The NG-01 cells are injected directly into the central nervous system (through the cerebrospinal fluid), where the cells settle close to the damaged areas or lesions in the brain and spinal cord, and exert significant neuroprotective, remyelinating, and anti-inflammatory effects..

关于NG-01NG-01是一种专有的自体骨髓干细胞群，旨在在中枢神经系统损伤部位分泌高水平的髓鞘再生和神经营养因子（NG-01）。NG-01细胞直接注射到中枢神经系统（通过脑脊液），在那里细胞靠近大脑和脊髓的受损区域或病变，并发挥显着的神经保护，髓鞘再生和抗炎作用。。

About NeuroGenesisNeuroGenesis is developing cell therapies for neurodegenerative disorders based on a unique approach that allows sustained delivery of high levels of neuroprotective, neurotrophic, and anti-inflammatory proteins at the site of CNS injury using the patient's own stem cells. The company's lead product is NG-01 that is under clinical development for the treatment of progressive Multiple Sclerosis, an indication for which a placebo-controlled Phase 2 study as well as two open label trials have been successfully completed.

关于神经发生NeuroGenesisNeuroGenesis正在开发针对神经退行性疾病的细胞疗法，该疗法基于一种独特的方法，可以使用患者自身的干细胞在中枢神经系统损伤部位持续递送高水平的神经保护，神经营养和抗炎蛋白。该公司的主导产品是NG-01，该产品正在临床开发中，用于治疗进行性多发性硬化症，一项安慰剂对照的2期研究以及两项开放标签试验已成功完成。

NG-01 was also tested in two successful Phase 2a trials in ALS patients. To date, more than 160 progressive MS and ALS patients combined, have been treated globally with NeuroGenesis' lead product via clinical trials and compassionate use treatments..

NG-01也在ALS患者的两项成功的2a期试验中进行了测试。迄今为止，通过临床试验和同情使用治疗，全球已有160多名进行性MS和ALS患者接受了神经发生领先产品的治疗。。

Media contact:Tsipi HaitovskyGlobal Media LiaisonNeuroGenesis+972-52-5989-892Tsipihai5@gmail.com

媒体联系人：齐皮·海托夫斯基全球媒体联络人神经发生+972-52-5989-892Tsipihai5@gmail.com

SOURCE NeuroGenesis Bio Inc.

SOURCE NeuroGenesis Bio Inc.公司。