A Phase I Study of Repeated Neural Stem Cell Based Virotherapy in Combination With N-Acetylcysteine Amid (NACA) and Standard Radiation and Chemotherapy for Newly Diagnosed High Grade Glioma

The goal of this clinical trial is to learn about the safety and feasibility of administering repeated doses of neural stem cell (NSC)-conditionally replicative adenovirus (CRAd)-survivin (S)-protomer (p)k7, in persons with newly diagnosed high grade glioma.
The main questions it aims to answer are:
* whether multiple doses of NSC-CRAd-S-pk7 are safe and feasible
* how multiple doses of NSC-CRAd-S-pk7 influence tumor response, overall survival, time to tumor progression, and quality of life.
Participants will:
* undergo a biopsy to confirm high grade glioma, then receive the first dose of NSC-CRAd-S-pk7 into the brain
* about 2 weeks later, undergo surgery to remove the tumor and receive the second dose of NSC-CRAd-S-pk7 into the brain
* start chemoradiation about 2 weeks after surgery, then about 2 weeks later, receive the 3rd dose of NSC-CRAd-S-pk7 into the brain
* four weeks later, at the end of chemoradiation, receive a fourth dose of NSC-CRAd-S-pk7 into the brain.
* after radiation is finished, receive standard of care chemotherapy and tumor-treating fields. Two additional doses of NSC-CRAd-S-pk7 will be given every 4 weeks.
Every other patient enrolled will receive N-acetylcysteine amide (NACA), from registration until the day prior to surgery and the second dose of NSC-CRAd-S-pk7.

开始日期2025-05-30

申办/合作机构

Northwestern University

NCT05994534

/ Recruiting临床1/2期

A Phase 1/2 Pharmacokinetic and Pharmacodynamic Study of NPI-001 Oral Solution Compared to Cysteamine Bitartrate in Cystinosis Patients

Safety, pharmacokinetics, and pharmacodynamics of NPI-001 oral solution in cystinosis patients compared with cysteamine.

开始日期2023-10-29

申办/合作机构

Nacuity Pharmaceuticals, Inc.

IRCT20231017059756N1

/ Recruiting临床3期IIT

Investigating the therapeutic effect of statin relative to the N-acetyl cysteine in the treatment of Helicobacter pylori patients

开始日期2023-10-23

申办/合作机构

Mashhad University of Medical Sciences

100 项与 N-Acetylcysteine amide 相关的临床结果

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100 项与 N-Acetylcysteine amide 相关的转化医学

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100 项与 N-Acetylcysteine amide 相关的专利（医药）

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项与 N-Acetylcysteine amide 相关的文献（医药）

2025-03-01·TALANTA

Rational engineering of ligands & metal nodes: MOF-on-MOF based probe for efficient integration of anti-interference and triple signal amplification

Article

作者: Wang, Jiaqing ; Ma, Zhanfang ; Han, Hongliang

Accurate and sensitive detection of slight fluctuations in insulin concentration is essential for the assessment and treatment of early diabetes. The requirement posed a serious challenge to the responsiveness of electrochemical sensors in analyzing slight fluctuation in concentration and their ability to accurately identify the target analyte. Therefore, a MOF-on-MOF (NH₂-MIL-88B-on-NH₂-UiO-66) probe with integrated anti-interference and signal-amplification capabilities was designed by engineering functionalized ligands and metal nodes, which ensures the sensitivity of sensors while improving the detection accuracy. Signal-amplification: the bimetallic ion nodes of MOF-on-MOF act synergistically at the sensing interface (sodium citrate doped poly-thionine/poly (3, 4-ethylenedioxythiophene) film) to impede the electron transfer, leading to a decrease in the poly-thionine (PThi) oxidation current signal. The complexed Fe³⁺ triggers a Fenton reaction, further leading to a decrease in the PThi oxidation current signal. A triple amplification of the PThi oxidation current signal response induced by changes in concentration per unit of analyte was achieved. Anti-interference: The growth of a more hydrophilic MOF on the surface of another less hydrophilic MOF and the introduction of a large number of amino groups resulted in a significant anti-interference property of the MOF-on-MOF based immunoprobes. The proposed sensor not only achieves detection limits of insulin at the fg level, but also has a signal response per unit change in insulin concentration being 5-50 times higher than existing electrochemical sensors. The integration of the hydrophilic and triple signal amplification strategy is significant in the monitoring of slight fluctuations of analyte concentrations.

2025-03-01·Journal of Pharmaceutical Analysis

Strategy for cysteine-targeting covalent inhibitors screening using in-house database based LC-MS/MS and drug repurposing

Article

作者: Li, Na ; Wu, Jian-Lin ; Hu, Xiaolan ; He, Quan ; Xiong, Zhi-Qi

Targeted covalent inhibitors, primarily targeting cysteine residues, have attracted great attention as potential drug candidates due to good potency and prolonged duration of action. However, their discovery is challenging. In this research, a database-assisted liquid chromatography-tandem mass spectrometry (LC-MS/MS) strategy was developed to quickly discover potential cysteine-targeting compounds. First, compounds with potential reactive groups were selected and incubated with N-acetyl-cysteine in microsomes. And the precursor ions of possible cysteine-adducts were predicted based on covalent binding mechanisms to establish in-house database. Second, substrate-independent product ions produced from N-acetyl-cysteine moiety were selected. Third, multiple reaction monitoring scan was conducted to achieve sensitive screening for cysteine-targeting compounds. This strategy showed broad applicability, and covalent compounds with diverse structures were screened out, offering structural resources for covalent inhibitors development. Moreover, the screened compounds, norketamine and hydroxynorketamine, could modify synaptic transmission-related proteins in vivo, indicating their potential as covalent inhibitors. This experimental-based screening strategy provides a quick and reliable guidance for the design and discovery of covalent inhibitors.

2025-03-01·AMERICAN JOURNAL OF VETERINARY RESEARCH

Oxidative stress induces plasma membrane phosphatidylserine externalization in canine erythrocytes in vitro, mitigated by thiol antioxidants

Article

作者: Santos, Andrea Pires dos ; Woolcock, Andrew D. ; Moore, George E. ; Kao, Yun-Fan ; Serpa, Priscila B. S.

Abstract:

OBJECTIVE:

To determine if oxidative stress induces phosphatidylserine (PS) externalization in canine erythrocytes and if exposure to antioxidants prevents such changes.

METHODS:

This was an in vitro, experimental study using 5 healthy, adult, purpose-bred research Beagles. Fresh EDTA-anticoagulated blood samples were collected from each dog, and erythrocytes were harvested. For objective 1, erythrocytes were exposed to the pro-oxidant agents tert-butyl hydroperoxide (TBHP) at 2, 3, or 4 mM or 2,2′-azobis(2-amidinopropane) dihydrochloride at 30, 40, or 50 mM. For objective 2, erythrocytes were exposed to 3 mM TBHP and the antioxidant N-acetylcysteine-amide (NACA) at various concentrations (0, 1, or 3 mM). Erythrocytes incubated with benzoylbenzoyl-ATP were used as positive control, whereas erythrocytes incubated with sodium chloride medium with 0.1% bovine serum albumin, DMSO, and NACA were used as negative controls. Erythrocytes were stained with allophycocyanin-conjugated Annexin V, and PS externalization was assessed by flow cytometry. The degree of PS externalization of each sample was recorded as median fluorescence intensity and percentage of PS positivity.

RESULTS:

TBHP at 3 and 4 mM caused increased PS externalization in canine erythrocytes. 2,2′-Azobis(2-amidinopropane) dihydrochloride at all concentrations caused increased PS externalization. N-acetylcysteine-amide at all concentrations prevented significant PS externalization measured by median fluorescence intensity and percentage of PS positivity from erythrocytes exposed to TBHP.

CONCLUSIONS:

Oxidative stress causes PS externalization in canine erythrocytes, and NACA ameliorates this effect.

CLINICAL RELEVANCE:

Future studies are needed to determine if increased PS externalization in erythrocytes occurs in dogs with immune-mediated hemolytic anemia and its role in promoting thromboembolism.

项与 N-Acetylcysteine amide 相关的新闻（医药）

2025-04-02

·PRNewswire

Arctic Therapeutics research suggests preventative strategy against HCCAA

N-acetylcysteine (NAC) shows promise to treat hereditary cystatin C amyloid angiopathy (HCCAA) HCCAA – a subtype of cerebral amyloid angiopathy (CAA) – is an ultra-rare Icelandic amyloid disease due to a mutation in the CST3 gene encoding for the protease inhibitor, cystatin C. HCCAA causes cerebral hemorrhages in asymptomatic carriers of the L68Q variant due to amyloid aggregation with precipitation in brain vessels and tissue. Findings could guide the development of preventative therapies for related CAAs. REYKJAVIK, Iceland, April 2, 2025 /PRNewswire/ -- Icelandic drug discovery and development company Arctic Therapeutics (ATx) today announced the publication of research in JAMA Neurology, which informs a possible therapeutic strategy to treat a rare form of familial dementia. The publication, titled N-Acetylcysteine for hereditary cystatin C amyloid angiopathy (HCCAA), a nonrandomised clinical trial, details a new treatment approach for HCCAA, a rare and fatal genetic brain disorder that primarily affects young Icelandic adults, with the disease process beginning in childhood. The research provides insights into the potential of N-acetylcysteine (NAC) as a treatment for HCCAA, a disease with no currently approved therapeutic options. The study highlights how NAC may reduce disease-associated biomarkers and slow disease progression. The study is the first phase 2a clinical trial to assess the safety and therapeutic potential of high-dose NAC in patients carrying the L68Q-CST3 sequence variation of HCCAA. Results indicate that NAC is well tolerated, reduces disease specific biomarkers and stabilises disease progression, suggesting NAC may offer a preventative strategy against HCCAA. These findings could accelerate the development of preventative therapies for individuals at risk of HCCAA and other forms of CAAs. "These promising results provide hope to HCCAA patients and their families," Dr Ásbjörg Ósk Snorradóttir, lead author of the study explained. Senior author, Dr. Hákon Hákonarson, director of the Center for Applied Genomics at the Children's Hospital of Philadelphia and founder of Arctic Therapeutics added: "This study provides the first clinical evidence that NAC, by breaking disulfide bonds and other aberrant molecular interactions, can disrupt cystatin C oligomers and prevent amyloid aggregation that ultimately accumulates in brain vessels. By targeting the root biochemical process behind HCCAA pathology, NAC may ultimately offer a novel disease-modifying approach, not only for this devastating condition, but potentially for other forms of CAAs and protein aggregation disorders where toxic oligomers are central to disease progression." The open-label phase 2a clinical trial was conducted at Iceland's Landspitali University Hospital from March 2019 to December 2021. A total of 17 participants were enrolled, all confirmed carriers of the L68Q-CST3 mutation. The study assessed: Safety & tolerability: NAC was well tolerated, with no drug-related severe adverse events. Five participants experienced cerebral hemorrhages, but none resulted in permanent neurological damage. Biomarker reduction: Significant reductions were observed in collagen IV, fibronectin, vimentin, and SMAD levels—key indicators of disease progression. Plasma & skin biomarker changes: NAC was shown to increase reduced glutathione levels, a protective antioxidant, and reduce high-molecular-weight cystatin C aggregates, which are linked to amyloid buildup in the brain. Cognitive stability: No measurable decline in cognitive function was observed during the study period. Building on these findings, as well as internal in-vitro data that demonstrates that NAC-amide (NACA) has more than 10-fold higher potency in preventing or dispersing amyloid complex aggregates than NAC and increased ability to penetrate the blood brain barrier and get into cells involved in the disease pathogenesis, ATx has launched a European Medicines Agency-approved registration study to monitor safety, tolerability, and efficacy using NACA in patients with HCCAA. For more details, read the full study in JAMA Neurology: Original Investigation. About Arctic Therapeutics Arctic Therapeutics is an Icelandic drug discovery and development company established in 2015, which has licensed intellectual property from Children's Hospital of Philadelphia. ATx leverages the transformative power of applied genomics, steering the course of drug development towards safer, more effective treatments for some of the world's most challenging diseases. The company has operations in Iceland, the US and multiple collaborations across Europe. For more information, please visit: and follow us on LinkedIn For media inquires Gulli Arnason, Chief Strategy Officer [email protected] +354 660 0053 This information was brought to you by Cision The following files are available for download: WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

临床2期临床结果

2025-01-27

·药时空

12 家制药公司在融资赛道上 “狂飙” 啦

家人们，大消息来咯！投融资数据库掐指一算（其实是认真统计啦），截至 1 月 24 日的上周，全球制药圈那叫一个热闹，足足发生了 12 起投融资大事件！这就好比制药界的一场 “财富狂欢派对”，各个公司都来分一杯羹。从药物类型上看，资本的目光那叫一个 “毒辣”，紧紧盯着 PROTAC、分子胶、siRNA、CAR-NK、小分子这些超前沿的研发领域，感觉就像是在寻宝，就盼着能挖到下一个 “神药” 宝藏。再瞧瞧地区分布，12 家拿到融资的公司里，一大半都来自咱们中国，亚盛医药、微光基因、塔吉瑞、英百瑞、达歌生物等等，这些公司就像一群 “潜力股小猎豹”，在融资赛道上一路飞奔。下面，数据库要挑几个超有看点的融资案例，给大家好好唠一唠，纯当大伙茶余饭后的 “趣味谈资” 哈。亚盛医药：美股上市 “双响炮”，赚钱治病两不误北京时间 1 月 24 日晚上，亚盛医药在美国纳斯达克闪亮登场，成功挂牌上市，直接拿下 2025 年美股上市生物医药公司的 “头把交椅” ！按照亚盛医药港股公告，人家这次一口气发行了 732.5 万股，每股定价 17.25 美元，轻轻松松就募资约 1.26 亿美元，这赚钱能力，简直绝绝子！亚盛医药早在 2009 年就 “出道” 了，一直扎根在肿瘤创新药研发领域，董事长兼 CEO 杨大俊博士就是他们的 “带头大哥”。2019 年 10 月，亚盛医药已经在香港联交所主板 “小试牛刀”，这次美股上市，直接让它成为港股 18A 企业里首个在美股双重上市的 “狠角色”。他家研发的奥雷巴替尼已经获批上市啦，而且还是中国首个且唯一的第三代 BCR-ABL 抑制剂，妥妥的 “独一份”。自 2021 年 1 月上市后，这款药一路 “卖爆”，累计销售额约 5 亿元人民币。2024 年 6 月，亚盛医药更是大手一挥，以 13 亿美元的高价，把这款产品的独家选择权卖给了武田，这波操作，简直赚麻了！另外，亚盛医药的产品管线也超厉害，抑制 Bcl-2、IAP 或 MDM2-p53 等细胞凋亡通路关键蛋白的抑制剂，还有针对癌症治疗中激酶突变体的新一代抑制剂，都是他们的 “秘密武器”。现在，亚盛医药已经在中国、美国、澳大利亚、欧洲等地开展了 40 多项临床试验，其中 13 项还是注册临床研究，未来发展那叫一个不可限量！微光基因：Pre-A 轮融资 “补血”，立志攻克常见病 1 月 24 日传来消息，微光基因成功完成了数千万元人民币的 Pre-A 轮融资，这可把公司乐开了花。本轮融资由广州产投集团和广州优玖投资 “慷慨解囊”，这些钱都将砸进常见病表观遗传编辑管线研究里。微光基因 2021 年 9 月才成立，CEO 胡洋博士带着团队一路 “打怪升级”。他们手握表观遗传编辑底层工具技术和递送系统这两大 “法宝”，专注通过 RNP/mRNA-VLP/LNP 递送系统，开发新一代体内基因编辑药物，目标就是要一次性治愈代谢疾病、感染性疾病这些 “顽固分子”，真可谓野心勃勃！听说微光基因还有个独有的基因编辑工具进化平台，就像一个神奇的 “魔法工厂”，能生产出高效又安全的表观遗传编辑工具，精准调控肝内 / 肝外基因，给患者提供高安全性、高有效性、高可及性的治疗方案，这要是成功了，那可真是患者的福音！塔吉瑞：5000 万美元 C 轮融资到账，海外临床冲冲冲 1 月 22 日，塔吉瑞生物宣布完成了 5000 万美元的 C 轮融资，这钱一到账，公司干劲更足了。塔吉瑞 2014 年就成立了，创始人兼 CEO 王义汉博士一直带着团队在靶向创新药研发领域深耕。这次融到的钱，都要拿去推进多个创新药物的海外临床研究，准备在国际舞台上大显身手！塔吉瑞有两款 “拳头产品” 超厉害。一个是 TGRX-678，作为 Bcr-Abl 变构抑制剂，打算用来治疗慢粒白血病（CML），目前正在国内开展 Ⅱ 期临床，在美国开展 Ⅰ 期临床，就像两个赛场同时 “作战”；另一个是 TGRX-326，第三代 ALK 抑制剂，瞄准的是非小细胞肺癌（NSCLC），已经在国内进入 Ⅲ 期阶段，离成功上市又近了一步。2024 年 9 月，先声药业旗下的先声再明还和塔吉瑞达成合作，拿下了 TGRX-326 在中国大陆地区的独家商业化权益，看来大家都很看好这款药呢！威凯尔：近 2 亿元 C + 轮融资，创新药研发火力全开 1 月 22 日，威凯尔也迎来了好消息，完成了近 2 亿元的 C + 轮融资。本轮投资方阵容超豪华，星河创投、金龙投资、国投招商、长江产业集团纷纷 “入局”。这一轮融资过后，威凯尔累计获得的股权融资已经超过 10 亿元，简直是 “不差钱” 的节奏！威凯尔创立于 2010 年，联合创始人兼 CEO 龚彦春博士带着团队一路打拼。公司业务很广，创新药自主研发和药物全产业链服务都做得风生水起。这次融资的钱，主要会花在加速推进创新药临床在研管线，还有加强 CRO/CDMO 服务的产业链布局上。在新药研发方面，威凯尔以抗血栓新药「维卡格雷」为核心，构建了梯队式布局，已经有 5 款临床阶段在研新药，像 P2Y12 受体拮抗剂维卡格雷、NTRK 抑制剂 VC004、选择性 JAK1 抑制剂 VC005 等等。另外，还有 9 款临床前在研药物蓄势待发，适应症覆盖心脑血管、肿瘤、炎症、自免等多个领域，这研发实力，杠杠的！英百瑞：近亿元 A + 轮融资，免疫治疗创新药搞起来 1 月 21 日，英百瑞宣布完成近亿元 A + 轮融资，华睿投资带头领投，泰珑投资、孙子科技、华医资本、滨江 5050 基金也跟着 “上车”。英百瑞 2016 年成立，创始人苗振伟博士带着团队一头扎进免疫治疗创新药开发领域，不管是肿瘤，还是免疫类疾病、慢性病和 CNS 等非肿瘤类疾病，都是他们的 “目标战场”。这次融资的钱，主要用来推进通用型自然杀伤细胞疗法管线的注册临床进展。依托自有抗体偶联 NK 细胞平台技术，英百瑞开发了好几个通用现货型细胞治疗产品。IBR854 细胞注射液准备用来治疗晚期实体肿瘤，马上要进入 II 期临床试验；IBR733 细胞注射液针对急性髓系白血病，已经在 I 期临床试验第三剂量组阶段 “奋战”；IBR822 细胞注射液针对晚期实体瘤，也进入了 I 期临床；还有 IBR900，这款现货通用型 NK 细胞产品已经获批临床，专门治疗 65 岁以上 B 细胞淋巴瘤，或者现有治疗不耐受和失败的肿瘤患者，简直是患者的 “救命稻草”！达歌生物：超 2000 万美元 A + 轮融资，分子胶药物 “搞事情” 1 月 22 日，达歌生物也来 “分蛋糕” 啦，宣布完成超 2000 万美元的 A + 轮融资。这次投资方阵容强大，浙江省「4+1」生物医药与高端器械产业基金、Takeda Ventures、丰川资本、隆门资本、浩悦中国医疗健康基金以及 AIM-HI Accelerator Fund 等机构纷纷投资，老股东元生创投也继续 “加仓”。达歌生物 2019 年成立，联合创始人兼 CEO 邹丽晖博士带着团队专注于用专有的基于分子胶的靶向蛋白降解药物发现平台开发新药。他家的 GlueXplorer® 平台就像一个 “宝藏库”，构建了受知识产权保护的化合物库和筛选系统，专门开发针对以往无法成药靶点的新型小分子药物。 2024 年 5 月，达歌生物就和武田达成合作，用 GlueXplorer® 平台开发针对肿瘤学、神经科学和炎症领域多个靶点的创新分子胶降解剂。没想到，武田旗下的 Takeda Ventures 这次还参与了 A + 轮融资，这合作关系真是越来越紧密啦！从达歌生物的产品管线布局来看，涵盖了肿瘤、炎症 / 代谢疾病、罕见遗传疾病等多个领域，未来发展潜力无限。安炎达医药：数千万元 Pre-A 轮融资，剑指免疫炎症疾病 1 月 21 日，安炎达医药成功完成数千万元人民币 Pre-A 轮融资，高创溪创投独家领投。安炎达医药 2022 年才成立，创始人兼 CEO 是 Micky D. Tortorella。这家公司专注开发针对免疫炎症领域的创新药，癌症、疼痛、神经系统疾病、骨关节炎等疾病都是他们的 “敌人”。安炎达医药的科学团队有个独特的想法，他们觉得组织微环境就像一个 “大指挥官”，决定了机体免疫系统的反应。细胞外基质稍微有点变化，免疫 / 炎症反应就可能 “失控”，导致疾病发生。所以，他们的药物专门针对组织微环境中的关键机制和途径，就像给失控的反应 “踩刹车”，纠正病理状态下的免疫炎症反应。现在，安炎达医药有 4 种临床候选药物正在开发中，这次融资的钱主要用来支持核心管线 MAX-001 在中国 / 澳大利亚针对晚期 / 难治性实体瘤的首次人体临床试验，希望能早日取得好成果！Sen-Jam Pharmaceutical：战略投资到手，抗炎代谢疗法要发力 1 月 22 日，Sen-Jam Pharmaceutical 宣布获得了 5 Horizons Capital 的金融和战略投资。Sen-Jam 这家公司致力于开发改变炎症和代谢紊乱的疗法，他们的专有技术 “多效抗炎疗法”(PAIR) 就像一个神奇的 “魔法棒”，希望能提供和人体免疫系统协同工作的精准疗法，减轻慢性炎症相关的系统性风险，让人能一直健健康康的。 Sen-Jam 的核心产品 SJP-001 已经获得澳大利亚人类研究伦理委员会批准开展临床试验，主要评估这款疗法在减轻炎症、促进代谢健康方面的效果。有了投资方 5 Horizons Capital 的支持，Sen-Jam 准备和 CRO 公司 Novotech 合作，一起把临床试验安排得明明白白！Arctic Therapeutics：2650 万欧元 A 轮融资，对抗疑难病有新招 1 月 22 日，冰岛的 Arctic Therapeutics（简称 ATx 公司）成功完成 2650 万欧元超额认购的 A 轮融资，这可把公司高兴坏了。Arctic 公司 2015 年成立，是美国应用基因组学中心 (CAG) 的 “亲儿子”。他们想利用应用基因组学的强大力量，给药物研发 “指方向”，为那些超难搞的疾病提供更安全、更有效的治疗方法。这次融到的钱，主要用来推动 ATx 公司两个领先候选药物 AT-001 和 AT-004 的研究。AT-001 是一种口服治疗药物，目标是防止有害淀粉样蛋白在大脑里 “捣乱”，聚集起来搞破坏，打算用来治疗家族性痴呆症、阿尔茨海默病等。2024 年，欧洲药品管理局 (EMA) 已经批准了一项关键的 IIb/III 期临床试验，就为了看看 AT-001 治疗遗传性胱抑素 C 淀粉样血管病 (HCCAA，一种罕见的家族性痴呆症）到底有没有效果。AT-004 则是治疗炎症性皮肤病的新方法，目前正在开展 Ⅱ 期临床，期待它们都能早日成功上市，造福患者！除了上面这些公司，上周还有不少制药公司也在融资上取得了进展。但篇幅有限，就不一一介绍啦。要是大家还知道更多上周的融资信息，请评论处留言！识别微信二维码，可添加药时空小编请注明：姓名+研究方向！

细胞疗法蛋白降解靶向嵌合体siRNA核酸药物免疫疗法

2025-01-22

·PRNewswire

Arctic Therapeutics Announces €26.5M Oversubscribed Series A Financing

Funding will accelerate the company's anti-amyloid treatment for rare and common forms of dementia. As well as bringing forward a new class of treatment for inflammatory skin diseases. Sanos Group CEO Jeppe Ragnar Andersen joins the board. REYKJAVIK, Iceland and PHILADELPHIA, Jan. 22, 2025 /PRNewswire/ -- Icelandic drug discovery and development company Arctic Therapeutics (ATx) today announced the successful closing of a €26.5 million oversubscribed Series A financing from a broad syndicate of international investors. The syndicate includes the EIC Fund, Iceland's largest privately-held investment firm Kaldbakur, Investcorp-backed Sanos Group, Swiss-based Cerebrum DAO, The Lurie Family Foundation – established by US billionaire Jeffery Lurie – as well as a consortium led by early investors and co-founders of Icelandic unicorn, Kerecis, and Copenhagen-listed Chemometec. Icelandic institutional investors and family offices also participated in the round. "This funding represents a critical milestone in our journey to address significant unmet medical needs, from familial forms of dementia and Alzheimer's to novel treatments for inflammatory skin diseases. The new investors bring expertise and strategic value to the table, enabling us to accelerate the development of groundbreaking treatments," said Ivar Hakonarson, ATx co-founder and CEO. The proceeds from the Series A financing will be used to advance ATx's two frontrunners, AT-001 and AT-004, further into the clinic. Last year, the European Medicines Agency (EMA) approved a pivotal phase IIb/III clinical trial evaluating AT-001 for the treatment of Hereditary Cystatin C Amyloid Angiopathy (HCCAA), a rare form of familial dementia. The new funding will allow ATx to explore the potential of AT-001, an oral treatment designed to prevent the aggregation of harmful amyloid proteins in the brain, in other forms of familial dementia, and eventually Alzheimer's disease. "Recent advances in understanding the role of amyloid proteins and the critical importance of preventing and dissolving plaque formation in the brain underscore the transformative potential of our approach to significantly slow or even prevent these devastating diseases," Hakonarson added. In addition to the AT-001 clinical trial, ATx is also planning to launch a phase IIa clinical trial for AT-004 in Europe post-financing. The therapeutic goal of this approach is to demonstrate safety and efficacy in acne vulgaris before expanding into other inflammatory skin diseases, including atopic dermatitis, rosacea and psoriasis. Additionally, ATx announced the appointment of Jeppe Ragnar Andersen, CEO of Sanos Group, to its board of directors. "Sanos Group's proven expertise in dermatology and recent acquisition of NeuroScios, a CRO specialized in clinical studies in Alzheimer's, strengthens our alignment with ATx's therapeutic focus even further," Andersen said. "We look forward to supporting the ATx team on achieving its goal of bringing forward innovative treatments for dementia and skin diseases to millions of patients," he added. Last year, ATx was selected to join the prestigious EIC Scaling Club, an exclusive community of Europe's most promising scaleups. The Club aims to accelerate the growth of its members, with a goal of scaling 20% of them into unicorns. This aligns with the EIC Fund's investment mandate to provide funding to European companies, which have the potential to meet the world's most pressing challenges. "Investing in innovative solutions to address the global healthcare challenge of dementia is both a responsibility and an opportunity," said Svetoslava Georgieva, Chair of the EIC Fund Board. "In Europe alone, dementia affects over ten million people – placing immense strain on families, healthcare systems, and economies. By providing an equity investment in Arctic Therapeutics, the EIC Fund is supporting groundbreaking preventive treatments that have the potential to change the trajectory of this crisis, improve quality of life, and alleviate the burden on healthcare infrastructure worldwide," Georgieva added. About Arctic Therapeutics Arctic Therapeutics is an Icelandic drug discovery and development company established in 2015 as a spin-off from the US-based Center for Applied Genomics (CAG), a research center at the Children's Hospital of Philadelphia led by ATx founder Hakon Hakonarson, MD, PhD. ATx leverages the transformative power of applied genomics, steering the course of drug development towards safer, more effective treatments for some of the world's most challenging diseases. The company has operations in Iceland, the US and multiple collaborations across Europe. For more information, please visit: and follow us on LinkedIn. About Sanos Group Sanos Group is a leading specialist provider of clinical study services in the global pharma and biotech industry. The group consists of around 200 employees, mainly in Denmark and the United States, but also in Europe and Hong Kong. Last year, Sanos acquired Austrian NeuroScios, which is a contract research organization (CRO) focusing on Alzheimer's and other diseases of the central nervous system. The acquisition is part of the group's strategic development as a multi-niche CRO, adding a third specialist area to the two current scientific areas of osteoarthritis and dermatology. About EIC Fund The European Innovation Council Fund from the European Commission is an agnostic fund: it invests across all technologies and verticals, and all EU countries and countries associated to Horizon Europe. It provides the investment component of the EIC Accelerator blended finance. The European Investment Bank acts as investment adviser to the EIC Fund. The EIC Fund aims to fill a critical financing gap and its main purpose is to support companies in the development and commercialization of disruptive technologies, bridging with and crowding in market players, and further sharing risk by building a large network of capital providers and strategic partners suitable for co-investments and follow-on funding. The fund pays particular attention to the empowerment and support of female founders as well as the ambition to reduce the innovation divide among EU countries. For media inquires Gulli Arnason, Chief Strategy Officer [email protected] +354 660 0053 This information was brought to you by Cision . The following files are available for download: SOURCE Arctic Therapeutics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

高管变更并购

100 项与 N-Acetylcysteine amide 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	-	-	DB14480

研发状态

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
胱氨酸贮积症	临床2期	澳大利亚	Nacuity Pharmaceuticals, Inc.	2023-10-29
Usher综合征	临床2期	澳大利亚	Nacuity Pharmaceuticals, Inc.	2020-09-03
阿尔茨海默症	临床2期	美国	Arctic Therapeutics	-
脑淀粉样血管病	临床2期	美国	Arctic Therapeutics	-
家族性淀粉样脑血管病	临床申请批准	冰岛	Nacuity Pharmaceuticals, Inc.	2024-03-12
家族性淀粉样脑血管病	临床申请批准	冰岛	Arctic Therapeutics	2024-03-12
创伤性脑损伤	临床前	美国	Brighton Biotech, Inc.	2024-10-15
认知障碍	临床前	美国	Brighton Biotech, Inc.	2024-10-15

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT00585559 (CTgov)	临床3期	颅内动脉瘤1 \| 颅内血管痉挛	44	N-acetylcysteine (N-acetylcysteine IV Infusion at 0.5 gm Hourly)	網壓鹹衊鹹獵鏇觸築選(餘願壓顧醖繭鬱願憲網) = 膚淵蓋選蓋鏇網餘鏇鬱鬱網襯膚窪鹽鑰願製夢 (範獵鹽鏇襯鬱醖獵壓艱, 壓壓遞簾鑰醖憲積憲餘 ~ 壓顧憲餘遞鏇願範壓網) 更多	-	2025-04-18
				Placebo+N-acetylcysteine (Placebo for N-acetylcysteine IV Infusion at 0.5 gm Hourly)	網壓鹹衊鹹獵鏇觸築選(餘願壓顧醖繭鬱願憲網) = 餘壓構鹽醖選窪衊憲餘鬱網襯膚窪鹽鑰願製夢 (範獵鹽鏇襯鬱醖獵壓艱, 餘餘夢簾膚蓋構廠廠鏇 ~ 醖鹹製鹹築繭壓蓋窪繭) 更多
NCT04632069 (CTgov)	早期临床1期	糖尿病患者妊娠 \| 糖尿病 \| 应激氧化	10	N acetyl cysteine + vagus nerve stimulation	網遞鬱顧鑰範窪廠餘構(淵壓壓鹽醖願積艱醖夢) = 獵願網鏇憲蓋蓋構鹽簾遞積簾鹽鹽衊襯製鹹範 (廠夢窪選衊繭願膚廠構, 0.4) 更多	-	2024-08-29
NCT01251315 (CTgov)	临床1期	应激氧化	24	Placebo (Placebo-A)	鑰選醖醖構選餘夢壓鹽(鏇衊窪遞選構鏇蓋鹹糧) = 餘鬱顧選觸製醖壓築鏇齋製鹹遞襯膚觸簾糧膚 (壓艱餘憲夢憲繭構鹹鏇, 133.7) 更多	-	2014-03-17
				N Acetyl cysteine (N Acetyl Cysteine-A)	鑰選醖醖構選餘夢壓鹽(鏇衊窪遞選構鏇蓋鹹糧) = 淵選範鹹壓醖顧淵繭製齋製鹹遞襯膚觸簾糧膚 (壓艱餘憲夢憲繭構鹹鏇, 131.2) 更多
NCT00332605 (CTgov)	临床2期	甲基苯丙胺依赖	45	Placebo	糧憲觸糧願鹹鏇齋膚襯(衊糧簾築築窪窪蓋顧廠) = 齋鏇憲獵糧鑰鑰膚構選鹽襯簾餘獵襯積網壓積 (鑰積鬱觸獵鹽製壓壓獵, 6.9) 更多	-	2013-07-24
ARVO2008	N/A	黄斑成群色素沉着	-	N-acetylcysteine amide (NACA)	構繭襯鹽窪製觸顧衊壓(膚簾獵顧觸獵齋顧鑰淵) = 觸獵齋範觸築襯製淵遞醖鑰鬱壓鑰鬱憲壓鏇繭 (窪艱顧觸繭範繭糧夢鏇 )	-	2008-05-01
				N-Acetylcysteine Amide (tert-butylhydroperoxide (tBH))	構繭襯鹽窪製觸顧衊壓(膚簾獵顧觸獵齋顧鑰淵) = 構顧顧顧鏇鏇膚獵鬱憲醖鑰鬱壓鑰鬱憲壓鏇繭 (窪艱顧觸繭範繭糧夢鏇 )