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MELBOURNE, Australia, June 16, 2025 / Biotech Newswire / --

澳大利亚墨尔本，2025年6月16日 /生物技术新闻社/ --

Neurizon® Therapeutics Limited

纽里佐恩®治疗有限公司

(ASX: NUZ & NUZOA) (“Neurizon” or “the Company”), a clinical-stage biotech company dedicated to advancing innovative treatments for neurodegenerative diseases, is pleased to announce new preclinical data demonstrating significant neuroprotective effects of NUZ-001 and its active metabolite, NUZ-001 Sulfone, in a zebrafish model of Huntington’s disease (HD).

（ASX：NUZ 和 NUZOA）（“Neurizon”或“公司”），一家致力于推进神经退行性疾病创新治疗的临床阶段生物技术公司，欣然宣布新的临床前数据，显示了 NUZ-001 及其活性代谢物 NUZ-001 Sulfone 在亨廷顿病（HD）斑马鱼模型中显著的神经保护作用。

HD is a rare, inherited neurodegenerative disorder that causes progressive degeneration of motor function, cognition, and mental health..

HD是一种罕见的遗传性神经退行性疾病，会导致运动功能、认知能力和精神健康的进行性退化。

In this disease model of HD, the targeted knockdown of the Htt (huntingtin) protein mRNA knockdown approach, triggers characteristic HD-related deficits, including increased cell death (acridine orange staining), morphological malformations, impaired haemoglobin production (Benzidine staining), and reduced expression of brain-derived neurotrophic factor (BDNF), a critical biomarker of neuronal function and survival.

在这种HD疾病模型中，靶向敲低Htt（亨廷顿蛋白）mRNA的方法触发了与HD相关的关键缺陷，包括细胞死亡增加（吖啶橙染色）、形态异常、血红蛋白生成受损（苯二胺染色）以及脑源性神经营养因子（BDNF）表达减少，这是神经元功能和存活的关键生物标志物。

Treatment with NUZ-001 or NUZ-001 Sulfone following Htt knockdown prevented developmental and morphological abnormalities, attenuated neuronal cell death, restored the delayed production of haemoglobin, and rescued BDNF expression, providing evidence of their potential to counteract early neurodegenerative damage..

NUZ-001 或 NUZ-001 砜在 Htt 基因敲低后进行处理，防止了发育和形态异常，减轻了神经细胞死亡，恢复了血红蛋白的延迟生成，并挽救了 BDNF 的表达，为其对抗早期神经退行性损伤的潜力提供了证据。

Dr. Michael Thurn

迈克尔·图恩博士

,

，

Managing Director and Chief Executive Officer, commented:

总经理兼首席执行官评论道：

“These results mark another important milestone in the realisation of the potential for NUZ-001 to treat a range of neurodegenerative diseases. HD is a devastating, rare genetic disorder that causes the progressive breakdown of nerve cells in the brain, leading to a range of symptoms including uncontrolled movements, cognitive decline, and emotional disturbances.

“这些结果标志着NUZ-001在治疗一系列神经退行性疾病潜力的实现上又达到了一个重要里程碑。亨廷顿病（HD）是一种毁灭性的罕见遗传疾病，会导致脑部神经细胞的逐步退化，引发包括不自主运动、认知能力下降及情绪障碍等一系列症状。”

HD affects between 2.7 and 4.8 per 100,000 people globally. There is no cure and no disease-modifying treatments, only treatments that manage symptoms. These exciting results demonstrate NUZ-001 has consistent neuroprotective effects beyond amyotrophic lateral sclerosis (ALS), strengthening our conviction in NUZ-001’s potential as a disease-modifying platform therapy across a range of neurodegenerative conditions.”.

亨廷顿病全球每10万人中影响2.7至4.8人。目前尚无治愈方法，也无疾病修饰治疗，只有管理症状的治疗。这些令人兴奋的结果表明，NUZ-001在肌萎缩侧索硬化症（ALS）之外具有一致的神经保护作用，进一步增强了我们对NUZ-001作为跨多种神经退行性疾病的疾病修饰平台疗法潜力的信心。”

About the Zebrafish Disease Model

关于斑马鱼疾病模型

Wild-type zebrafish embryos were raised in standard conditions. Morpholino antisense oligonucleotides (MOs) targeting Htt were injected into one-cell stage embryos to decrease Htt expression. NUZ-001 or NUZ-001 Sulfone at 1 and 10 μM concentrations were added to the embryonic media 6 hours post-fertilisation to evaluate the protective effects of NUZ-001 and NUZ-001 Sulfone on Htt knockdown-induced deficits.

野生型斑马鱼胚胎在标准条件下饲养。将针对Htt的吗啉代反义寡核苷酸（MOs）注射到单细胞期胚胎中以降低Htt的表达。在受精后6小时，将1和10 μM浓度的NUZ-001或NUZ-001砜添加到胚胎培养基中，以评估NUZ-001和NUZ-001砜对Htt敲低诱导缺陷的保护作用。

Changes in morphology (eye size and hindbrain swelling), neuronal cell death (apoptosis), and the levels of BDNF expression were analysed 2 days post-fertilisation (See Figure 1)..

受精后 2 天分析了形态学（眼睛大小和后脑肿胀）、神经元细胞死亡（凋亡）以及 BDNF 表达水平的变化（见图 1）。

Figure 1

图1

: Htt knockdown and treatment procedure

：Htt敲低和治疗程序

For high resolution please click the image.

要获得高分辨率，请点击图像。

Knockdown of Htt (Htt MO) resulted in zebrafish embryos with smaller eyes and swollen hindbrain ventricles (See Figure 2a; arrows indicate hindbrain and eye regions) compared to the control group (Control MO). Partial rescue of eye size was observed following treatment with 1 μM and 10 μM NUZ-001 and NUZ-001 Sulfone, with full reversal of hind brain swelling at 10 μM NUZ-001 and NUZ-001 Sulfone.

敲低 Htt（Htt MO）导致斑马鱼胚胎的眼睛较小且后脑室肿胀（见图 2a；箭头指示后脑和眼部区域），与对照组（Control MO）相比。在使用 1 μM 和 10 μM NUZ-001 及 NUZ-001 砜处理后，观察到眼睛大小的部分恢复，并且在 10 μM NUZ-001 和 NUZ-001 砜处理下，后脑肿胀完全逆转。

(Figure 2b). At 2 dpf, neuronal cell death (Apoptosis; See Figure 3) was significantly higher in the Htt knockdown zebrafish embryos compared to the control group. Treatment with 1 μM and 10 μM NUZ-001, and 10 μM NUZ-001 Sulfone significantly reduced neuronal cell death. Haemoglobin levels (Benzidine staining; See Figure 4) were significantly decreased in the Htt knockdown group compared to controls, whereas treatment with 1 μM and 10 μM NUZ-001, and with 1 μM and 10 μM NUZ-001 Sulfone provided partial rescue.

（图2b）。在2 dpf时，与对照组相比，Htt敲低的斑马鱼胚胎中神经元细胞死亡（凋亡；见图3）显著增加。使用1 μM和10 μM的NUZ-001以及10 μM的NUZ-001砜处理显著减少了神经元细胞死亡。血红蛋白水平（联苯胺染色；见图4）在Htt敲低组中较对照组显著降低，而用1 μM和10 μM的NUZ-001以及1 μM和10 μM的NUZ-001砜处理则提供了部分挽救。

Expression of BDNF transcripts (See Figure 5) was significantly rescued following treatment with 10 μM NUZ-001 and 10 μM NUZ-001 Sulfone..

BDNF转录本的表达（见图5）在用10 μM NUZ-001和10 μM NUZ-001砜处理后显著恢复。

Figure 2a & b

图2a和b

: Rescue of morphological abnormalities induced by

：由诱导的形态异常的拯救

htt

htt

knockdown

击倒

Figure 2a

图2a

For high resolution please click the image.

为了获得高分辨率，请点击图像。

Figure 2b

图2b

Figure 3

图3

: Reduction in neuronal cell death

减少神经细胞死亡

Figure 4

图4

: Partial rescue of haemoglobin levels

：部分恢复血红蛋白水平

Figure 5

图5

: Rescue of BDNF levels

：BDNF水平的恢复

NUZ-001 is currently in clinical development for amyotrophic lateral sclerosis (ALS), where it has shown preclinical efficacy in enhancing proteostasis, reducing pathological protein aggregation, and preserving neuronal function. The new findings in the HD model further underscore NUZ-001’s potential as a platform therapy that targets fundamental cellular stress and clearance mechanisms common to multiple neurodegenerative diseases..

NUZ-001 目前正在肌萎缩侧索硬化症（ALS）的临床开发中，其在增强蛋白质稳态、减少病理性蛋白聚集和保护神经元功能方面已显示出临床前疗效。在亨廷顿病（HD）模型中的新发现进一步强调了 NUZ-001 作为一种平台疗法的潜力，该疗法针对多种神经退行性疾病共有的基本细胞应激和清除机制。

Neurizon plans to advance additional preclinical studies in mammalian models of Huntington’s disease as part of its broader strategy to expand the therapeutic applications of NUZ-001 into other progressive neurological disorders with high unmet need.

Neurizon计划在其更广泛的策略中，推进NUZ-001在亨廷顿病哺乳动物模型中的额外临床前研究，以将该药物的治疗应用扩展到其他具有高度未满足需求的进展性神经系统疾病。

About

关于

Huntington’s Disease

亨廷顿病

Huntington’s disease (HD) is a rare, inherited (genetic) neurodegenerative disorder that causes progressive degeneration of motor function, cognition, and mental health. It is caused by a CAG trinucleotide repeat expansion in the Htt gene, leading to the production of a mutant huntingtin protein (mHtt) with an abnormally long polyglutamine (polyQ) tract.

亨廷顿病 (HD) 是一种罕见的遗传性神经退行性疾病，会导致运动功能、认知能力和精神健康的进行性退化。它由 **Htt 基因**中的 CAG 三核苷酸重复扩增引起，导致产生带有异常长的多聚谷氨酰胺（polyQ）序列的突变亨廷顿蛋白（mHtt）。

This mutant protein misfolds and forms toxic aggregates in neurons, particularly in the striatum and cortex, disrupting cellular functions such as gene transcription, protein degradation, and mitochondrial activity. Over time, these aggregates contribute to neuronal dysfunction and death, resulting in the hallmark symptoms of chorea (involuntary movements), cognitive decline, and behavioural disturbances..

这种突变蛋白发生错误折叠，并在神经元中形成有毒的聚集体，特别是在纹状体和皮层中，破坏基因转录、蛋白质降解和线粒体活动等细胞功能。随着时间的推移，这些聚集体导致神经元功能障碍和死亡，从而引发舞蹈症（不自主运动）、认知衰退和行为异常等标志性症状。

HD is most often diagnosed between the ages of 30 and 50, but it can also occur earlier, including in children and adolescents (a form known as juvenile-onset Huntington’s disease (JHD)). The condition typically progresses over 15 to 20 years, during which individuals experience a gradual decline in their ability to work, communicate, manage daily activities, and maintain independence..

HD 最常在 30 至 50 岁之间被诊断出来，但也可能更早发生，包括儿童和青少年（一种称为青少年起病的亨廷顿病 (JHD) 的形式）。该病通常在 15 到 20 年内逐渐进展，在此期间，个体的工作能力、沟通能力、管理日常活动以及保持独立性的能力会逐渐下降。

At present, there are no approved therapies that modify the underlying progression of HD. Current treatment approaches focus on symptom management. Chorea may be treated with dopamine-depleting agents, while psychiatric symptoms are typically managed with antidepressants or antipsychotics. However, these treatments do not alter the course of the disease..

目前，尚无获批的疗法能够改变亨廷顿病 (HD) 的潜在病程。当前的治疗策略侧重于症状管理。舞蹈样动作可使用耗竭多巴胺的药物进行治疗，而精神症状通常通过抗抑郁药或抗精神病药物来管理。然而，这些治疗并不能改变疾病的进程。

This announcement has been authorized for release by the Board of Neurizon Therapeutics Limited.

本公告已获Neurizon Therapeutics Limited董事会授权发布。

About

关于

Neurizon Therapeutics Limited

纽瑞森治疗有限公司

Neurizon Therapeutics Limited (ASX: NUZ) is a clinical-stage biotechnology company dedicated to advancing treatments for neurodegenerative diseases. Neurizon is developing its lead drug candidate, NUZ-001, for the treatment of ALS, which is the most common form of motor neurone disease.  Neurizon’s strategy is to accelerate access to effective ALS treatments for patients while exploring NUZ-001’s potential for broader neurodegenerative applications.

Neurizon Therapeutics Limited（ASX：NUZ）是一家临床阶段的生物技术公司，致力于推进神经退行性疾病的治疗方法。Neurizon正在开发其主要候选药物NUZ-001，用于治疗最常见的运动神经元疾病——肌萎缩侧索硬化症（ALS）。Neurizon的战略是加快患者获得有效的ALS治疗，同时探索NUZ-001在更广泛的神经退行性疾病中的潜力。

Through international collaborations and rigorous clinical programs, Neurizon is dedicated to creating new horizons for patients and families impacted by complex neural disorders..

通过国际合作和严格的临床项目，Neurizon致力于为受复杂神经疾病影响的患者和家庭创造新的希望。

Neurizon® is a registered trademark of Neurizon Therapeutics Limited

Neurizon® 是 Neurizon Therapeutics Limited 的注册商标

Contacts

联系人

Neurizon Therapeutics Limited

纽瑞森治疗有限公司

Dr. Michael Thurn

迈克尔·图恩博士

Managing Director and Chief Executive Officer

总经理兼首席执行官

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Lidija Damjanovic

莉迪亚·达米亚诺维奇

Head of Marketing and Corporate Affairs

市场营销与企业事务主管

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+61 (0) 425 700 504

+61 (0) 425 700 504

Keywords: Zebrafish; Brain-Derived Neurotrophic Factor; Neuroprotective Agents; Mental Health; Huntington Disease; Neurodegenerative Diseases; Hemoglobins; Cognition; Cell Death; Neurizon Therapeutics; NUZ-001; NUZ-001 Sulfone; zebrafish model; Huntington’s disease (HD); neuroprotective effects; neuronal cell death; haemoglobin production; BDNF expression; Htt protein knockdown; developmental abnormalities; morphological abnormalities; amyotrophic lateral sclerosis (ALS); platform therapy; neurodegenerative diseases; Htt knockdown; neuronal function; juvenile-onset Huntington’s disease (JHD); ASX: NUZ.

关键词：斑马鱼；脑源性神经营养因子；神经保护剂；心理健康；亨廷顿病；神经退行性疾病；血红蛋白；认知；细胞死亡；Neurizon Therapeutics；NUZ-001；NUZ-001 亚砜；斑马鱼模型；亨廷顿病 (HD)；神经保护作用；神经元细胞死亡；血红蛋白生成；BDNF 表达；Htt 蛋白敲低；发育异常；形态学异常；肌萎缩侧索硬化症 (ALS)；平台疗法；神经退行性疾病；Htt 敲低；神经元功能；青少年发病亨廷顿病 (JHD)；ASX: NUZ。

Source: Biotech Newswire

来源：生物技术新闻专线