FDA批准NUZ-001进入HEALEY ALS平台试验-动脉网

FDA Clears NUZ-001 for Entry into HEALEY ALS Platform Trial

B3C newswire 等信源发布 2025-12-11 21:15

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MELBOURNE, Australia, December 11, 2025 / Biotech Newswire / --

澳大利亚墨尔本，2025年12月11日 /生物技术新闻社/ --

Neurizon® Therapeutics Limited

纽里宗®治疗有限公司

(ASX: NUZ & NUZOA; OTCQB: NUZTF) ('Neurizon' or 'the Company'), a clinical-stage biotech company dedicated to advancing innovative treatments for neurodegenerative diseases, is pleased to announce that the Sean M. Healey & AMG Center for ALS at Mass General Brigham has received clearance from the US Food and Drug Administration (FDA) to proceed with Neurizon’s NUZ-001 regimen in the HEALEY ALS Platform Trial (ClinicalTrial.gov identifier: NCT04297683) following completion of the FDA’s 30-day review.

(ASX: NUZ 和 NUZOA；OTCQB: NUZTF)（“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，马萨诸塞总医院Sean M. Healey & AMG ALS中心已获得美国食品药品监督管理局 (FDA) 的批准，可以在完成FDA 30天审查后，在HEALEY ALS平台试验 (ClinicalTrial.gov标识符：NCT04297683) 中继续进行Neurizon的NUZ-001方案。

NUZ-001 is officially Regimen I..

NUZ-001正式为方案I。

The FDA’s acceptance of the protocol amendment allows the commencement of clinical activities under the Sean M. Healey & AMG Center for ALS Investigational New Drug (IND). It represents another significant regulatory milestone for Neurizon, enabling the Company and the HEALEY ALS Platform Trial team to progress to the next operational phases, including Institutional Review Board (IRB) submissions and approvals, activation of clinical trial sites, and initiation of study start-up activities.

美国食品药品监督管理局（FDA）接受协议修正案后，Sean M. Healey & AMG肌萎缩侧索硬化症（ALS）研究性新药（IND）的临床活动得以启动。这标志着Neurizon公司又一重要的监管里程碑，使得该公司和HEALEY ALS平台试验团队能够进入下一操作阶段，包括提交机构审查委员会（IRB）审批、激活临床试验站点以及启动研究筹备活动。

The first patients are expected to be enrolled early in 2026..

预计2026年初将招募到首批患者。

The HEALEY ALS Platform Trial is a multicenter, double-blind, placebo-controlled, adaptive trial for ALS created in partnership with the Network of Excellence for ALS (NEALS). The goal of the HEALEY ALS Platform Trial is to accelerate the development of potential new ALS therapies. The trial tests and evaluates multiple investigational drugs simultaneously, shares infrastructure across trial sites, and improves start-up and enrollment efficiencies, allowing for fast results..

HEALEY ALS平台试验是一项多中心、双盲、安慰剂对照、适应性ALS试验，由卓越ALS网络（NEALS）合作创建。HEALEY ALS平台试验的目标是加速潜在ALS新疗法的开发。该试验同时测试和评估多种研究药物，跨试验站点共享基础设施，并提高启动和入组效率，从而实现快速获得结果。

NUZ-001 is in development for treating amyotrophic lateral sclerosis (ALS). It aims to address key pathological mechanisms, including TDP-43 protein aggregation and impaired autophagy, which are central features across multiple neurodegenerative diseases. Preclinical and clinical data to date indicate favourable oral bioavailability, central nervous system penetration, an encouraging long‑term safety profile, and early signs of potential clinical benefit..

NUZ-001 正在开发用于治疗肌萎缩侧索硬化症（ALS）。它旨在解决关键的病理机制，包括TDP-43蛋白聚集和自噬功能障碍，这些是多种神经退行性疾病的共同核心特征。迄今为止的临床前和临床数据表明，其具有良好的口服生物利用度、中枢神经系统渗透性、令人鼓舞的长期安全性，并显示出潜在临床益处的早期迹象。

Dr Michael Thurn, Managing Director & CEO, commented:

Dr Michael Thurn，董事总经理兼首席执行官，评论道：

“Receiving the FDA’s clearance is a major milestone for Neurizon and a critical step in advancing NUZ-001 as a potential new treatment option for people living with ALS. The HEALEY ALS Platform Trial represents the gold standard for efficient and collaborative clinical development in this field, and we are proud to progress into the next phase of activation.

“获得FDA的批准是Neurizon的一个重要里程碑，也是推进NUZ-001作为ALS患者潜在新治疗选择的关键一步。HEALEY ALS平台试验代表了该领域高效且协作的临床开发的黄金标准，我们很自豪能够进入激活的下一阶段。”

We look forward to working closely with the HEALEY team as we move toward site initiations and study start-up, bringing us one step closer to addressing this significant unmet medical need.”.

我们期待在推进现场启动和研究开始的过程中，与HEALEY团队密切合作，这将使我们更进一步解决这一重要的未满足医疗需求。

Professor Merit Cudkowicz, Principal Investigator, HEALEY ALS Platform Trial & Director, Sean M. Healey & AMG Center for ALS, Mass General Brigham, commented:

梅里特·库德科维奇教授，HEALEY ALS平台试验首席研究员，肖恩·M·希利和AMG ALS中心主任，麻总百瀚医疗集团评论道：

“We are pleased to welcome Neurizon’s NUZ-001 regimen into the HEALEY ALS Platform Trial. The HEALEY ALS Platform Trial Therapy Evaluation Committee previously selected NUZ-001 for inclusion as a new regimen. The FDA’s clearance to proceed with the NUZ-001 regimen is an important next step. We look forward to initiating clinical sites and beginning enrollment so we can evaluate the potential of NUZ-001 to benefit people with ALS.”.

“我们很高兴欢迎Neurizon的NUZ-001方案加入HEALEY ALS平台试验。HEALEY ALS平台试验治疗评估委员会此前已选择NUZ-001作为新方案纳入。FDA批准继续进行NUZ-001方案是重要的下一步。我们期待启动临床站点并开始招募，以便评估NUZ-001为ALS患者带来益处的潜力。”

Neurizon remains dedicated to developing novel, disease-modifying therapies for ALS and other serious neurodegenerative diseases, leveraging international collaborations and world-leading clinical platforms to accelerate clinical progress.

纽里兹恩仍然致力于开发新颖的、改变疾病的肌萎缩侧索硬化症（ALS）和其他严重神经退行性疾病的疗法，利用国际合作和世界领先的临床平台来加速临床进展。

Semantic keywords: Neurodegenerative Diseases, Amyotrophic Lateral Sclerosis, Motor Neuron Disease, Clinical Trials, Phase II as Topic, FDA, NUZ-001, HEALEY ALS Platform Trial, Sean M. Healey & AMG Center, Neurizon Therapeutics, ALS, FDA clearance, Investigational New Drug, IND, IRB approval, clinical trial sites, patient enrollment, adaptive trial, NEALS, Network of Excellence for ALS, TDP-43, protein aggregation, autophagy, oral bioavailability, CNS penetration, regulatory milestone.

语义关键词：神经退行性疾病、肌萎缩侧索硬化症、运动神经元病、临床试验、II期试验、FDA、NUZ-001、HEALEY ALS平台试验、Sean M. Healey & AMG中心、Neurizon Therapeutics、ALS、FDA许可、研究性新药、IND、伦理审查委员会批准、临床试验地点、患者招募、适应性试验、NEALS、ALS卓越网络、TDP-43、蛋白质聚集、自噬、口服生物利用度、中枢神经系统渗透、监管里程碑。

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 the potential of NUZ-001 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，用于治疗肌萎缩侧索硬化症（ALS），这是运动神经元病最常见的形式。Neurizon的战略是加快患者获得有效ALS治疗的速度，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. NUZ-001 is an investigational product and is not approved for commercial use in any jurisdiction..

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。NUZ-001是一种研究性产品，在任何司法管辖区均未获批准用于商业用途。

Neurizon® is a registered trademark of Neurizon Therapeutics Limited

Neurizon® 是 Neurizon Therapeutics Limited 的注册商标

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神经元治疗学

Lidija Damjanovic

莉迪亚·达米亚诺维奇

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