A Phase I, Double-blind, Randomized, Placebo-controlled Study to Assess the Safety and Pharmacokinetics of ATL-001 (Ciclopirox Olamine) in Healthy Volunteers

The goal of this clinical trial is to assess the safety, tolerability and pharmacokinetics of ATL-001 (ciclopirox olamine) in healthy volunteers

开始日期2023-03-31

申办/合作机构

Atlas Molecular Pharma SL初创企业

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项与 Atlas Molecular Pharma SL 相关的文献（医药）

2022-08-01·Journal of Neurology2区 · 医学

Description of the first Spanish case of Gerstmann–Sträussler–Scheinker disease with A117V variant: clinical, histopathological and biochemical characterization

2区 · 医学

Article

作者: Perez de Nanclares, Guiomar ; Díaz-Domínguez, Carlos M ; Navarro, Carmen ; Viéitez, Irene ; Geijo, Mariví ; Castilla, Joaquín ; Charco, Jorge M ; Rodríguez, Rosa ; Teijeira, Susana ; Yañez, Rosa ; San Millán, Beatriz ; Pereda, Arrate ; Eraña, Hasier

AbstractGerstmann–Sträussler–Scheinker disease (GSS) is a rare neurodegenerative illness that belongs to the group of hereditary or familial Transmissible Spongiform Encephalopathies (TSE). Due to the presence of different pathogenic alterations in the prion protein (PrP) coding gene, it shows an enhanced proneness to misfolding into its pathogenic isoform, leading to prion formation and propagation. This aberrantly folded protein is able to induce its conformation to the native counterparts forming amyloid fibrils and plaques partially resistant to protease degradation and showing neurotoxic properties. PrP with A117V pathogenic variant is the second most common genetic alteration leading to GSS and despite common phenotypic and neuropathological traits can be defined for each specific variant, strikingly heterogeneous manifestations have been reported for inter-familial cases bearing the same pathogenic variant or even within the same family. Given the scarcity of cases and their clinical, neuropathological, and biochemical variability, it is important to characterize thoroughly each reported case to establish potential correlations between clinical, neuropathological and biochemical hallmarks that could help to define disease subtypes. With that purpose in mind, this manuscript aims to provide a detailed report of the first Spanish GSS case associated with A117V variant including clinical, genetic, neuropathological and biochemical data, which could help define in the future potential disease subtypes and thus, explain the high heterogeneity observed in patients suffering from these maladies.

2021-07-01·Hepatology

Metabolic Landscape of the Mouse Liver by Quantitative 31P Nuclear Magnetic Resonance Analysis of the Phosphorome

Article

作者: Bernardo-Seisdedos, Ganeko ; Millet, Oscar ; Bilbao, Jon ; Mato, José M ; Lopitz-Otsoa, Fernando ; Bizkarguenaga, Maider ; Abril-Fornaguera, Jordi ; Nogueiras, Rubén ; Lu, Shelly C ; Diercks, Tammo ; Gutierrez de Juan, Virginia ; Fernández-Ramos, David ; Mateos, Borja ; Fondevila, Marcos F

Background and AimsThe liver plays a central role in all metabolic processes in the body. However, precise characterization of liver metabolism is often obscured by its inherent complexity. Phosphorylated metabolites occupy a prominent position in all anabolic and catabolic pathways. Here, we develop a 31P nuclear magnetic resonance (NMR)–based method to study the liver “phosphorome” through the simultaneous identification and quantification of multiple hydrophilic and hydrophobic phosphorylated metabolites.Approach and ResultsWe applied this technique to define the metabolic landscape in livers from a mouse model of the rare disease disorder congenital erythropoietic porphyria (CEP) as well as two well‐known murine models of nonalcoholic steatohepatitis: one genetic, methionine adenosyltransferase 1A knockout mice, and the other dietary, mice fed a high‐fat choline‐deficient diet. We report alterations in the concentrations of phosphorylated metabolites that are readouts of the balance between glycolysis, gluconeogenesis, the pentose phosphate pathway, the tricarboxylic acid cycle, and oxidative phosphorylation and of phospholipid metabolism and apoptosis. Moreover, these changes correlate with the main histological features: steatosis, apoptosis, iron deposits, and fibrosis. Strikingly, treatment with the repurposed drug ciclopirox improves the phosphoromic profile of CEP mice, an effect that was mirrored by the normalization of liver histology.ConclusionsIn conclusion, these findings indicate that NMR‐based phosphoromics may be used to unravel metabolic phenotypes of liver injury and to identify the mechanism of drug action.

2020-09-01·British Journal of Haematology

Abnormal concentration of porphyrins in serum from COVID‐19 patients

Letter

作者: Gil-Redondo, Rubén ; García de Vicuña, Aitor ; Bernardo-Seisdedos, Ganeko ; San Juan, Itxaso ; Embade, Nieves ; Diercks, Tammo ; Arana, Eunate ; Bizkarguenaga, Maider ; Laín, Ana ; Gil-Martínez, Jon ; Mato, José M ; Bruzzone, Chiara ; Millet, Oscar ; Urquiza, Pedro ; Seco, Marisa

COVID 19 is an infectious disease caused by severe acute respiratory syndrome coronavirus 2.COVID 19 is not only a lung disease but rather a systemic syndrome where alterations in the blood may play a key role.Clin. evaluation of patients revealed low Hb levels along with increased total bilirubin and ferritin concentrationsAutopsies of deceased COVID-19 patients have revealed that the spleen is significantly altered in size, which is a normal physiol. response to anemia.Spontaneous or effector-induced chem. degradation likewise affects several intermediates in the haempathway to generate a number of toxic byproducts, including the oxidation of porphyrinogens to porphyrins.

项与 Atlas Molecular Pharma SL 相关的新闻（医药）

2022-12-23

·创鉴汇

聚焦西班牙，今年这12家“小而美”的创新药公司喜获融资 | 欧洲制药新风向

▎药明康德内容团队编辑我们“欧洲制药新风向系列”文章在前四期介绍了德国、英国、法国、瑞士生物技术公司本年度的融资情况，今天我们将把目光聚焦于西班牙。和这些国家有所不同，支撑起西班牙生物技术行业的大多是中小型企业。根据西班牙生物技术公司协会（ASEBIO）发布的报告，现有862家生物技术公司中，雇员少于10人的微型中小企业占比近53%（截止2020年）。在投融资方面，该行业仍是国际投资者关注的焦点。去年，西班牙生物技术行业吸引超过1.8亿欧元的融资，其中超过1亿欧元来自国外的投资者。据创鉴汇不完全统计，今年西班牙共有12家新药研发公司获得融资，绝大多数都是微小企业。其中，通过西班牙国家工业技术署（CDTI）获得资金的有三家公司：治疗罕见中枢神经系统疾病的Minoryx Therapeutics获得5100万欧元融资，将支持其小分子激动剂的上市许可申请工作；致力于推进罕见代谢疾病药物研发的Atlas Molecular Pharma以及抗肿瘤靶向药研发公司Peptomyc。此外，获得诺华等青睐的SpliceBio利用其源于普林斯顿大学的技术平台“切断”基因，克服病毒载体载荷有限的障碍；Oncomatryx靶向肿瘤微环境开发抗体偶联药物（ADC）......欢迎访问融资库小程序，获取更多VIP专享资讯 Minoryx完成5100万欧元C轮融资，推动小分子激动剂治疗罕见CNS疾病成立日期：2011年关键词：中枢神经系统融资金额：5100万欧元C轮投资机构：Columbus Venture Partners、Caixa Capital Risc、CDTI Innvierte、Fund+、Ysios Capital 5月31日，Minoryx Therapeutics宣布完成5100万欧元C轮融资，将支持其候选药物leriglitazone的上市许可申请（MAA）及在欧洲的上市准备工作，用于治疗患有肾上腺髓质神经病（AMN）的成年男性X连锁肾上腺脑白质营养不良（X-ALD）患者，此外还将适应症扩展到整个X-ALD人群。 Minoryx致力于开发罕见中枢神经系统疾病的新疗法，其主导项目leriglitazone（MIN-102）是一种新型选择性PPAR γ激动剂，目前正处于临床开发阶段。leriglitazone的主要适应症是X连锁肾上腺脑白质营养不良（X-ALD），这是一种破坏性神经退行性疾病，以两种形式存在：肾上腺脊髓神经病（AMN）和肾上腺脑白质营养不良（cALD）。该药物在相关疾病动物模型中展示了强大的临床前概念验证，并成功完成了1期临床试验。 SpliceBio完成5000万欧元A轮融资，推动蛋白剪接平台发展及在研基因疗法进入临床开发关键词：基因疗法融资金额：5000万欧元A轮投资机构：UCB Ventures、Ysios Capital、恩颐投资（New Enterprise Associates，NEA）、Gilde Healthcare、诺华风险投资基金（Novartis Venture Fund）、Asabys Partners 2月16日，SpliceBio宣布完成5000万欧元的A轮融资，达成超额认购。所得资金将推动其基于蛋白剪接（protein splicing）的主打在研基因疗法进入临床开发，用于治疗眼底黄色斑点症（又名Stargardt病），并且扩展其研发管线，治疗目前尚无法治疗的遗传性疾病。 SpliceBio是一家利用蛋白剪接技术开发新型基因疗法的生物技术公司，其技术平台旨在利用腺相关病毒（AAV）载体高效递送大基因。该平台是基于普林斯顿大学（Princeton University）科学家20多年对内含肽（intein）和蛋白质工程学的开拓性研究。该公司的蛋白剪接技术可以将原本无法包装进一个AAV载体的基因分成两部分，分别表达携带内含肽的部分蛋白，随后内含肽在细胞内介导蛋白剪接，合成具有功能的全长治疗性蛋白，从而克服AAV载体载荷有限的障碍。 Oncomatryx完成1500万欧元增资，推进抗体偶联药物研发成立日期：2009年关键词：抗体偶联药物融资金额：1500万欧元 4月19日，Oncomatryx已完成1500万欧元增资，将用于临床开发靶向肿瘤微环境的抗体偶联药物（ADC）管线。Oncomatryx是一家肿瘤靶向疗法研发公司，研究重点在于将治疗性抗体与细胞毒性分子结合，其专有连接头可提高ADC的抗肿瘤功效。据官网显示，Oncomatryx研发的ADC包含人IgG1抗体，靶向癌症相关的成纤维细胞和内皮细胞，与抑制微管蛋白聚合并破坏微管稳定性的780 Da细胞毒剂偶联。其主要候选药物OMTX705已经在小鼠和非人灵长类动物模型中进行实验，实验结果显示出良好的抗肿瘤作用与安全性。此外，Oncomatryx还开发了针对肿瘤的诊断产品，包括伴随诊断产品。 Geneos Therapeutics完成1700万美元A2轮融资，开发个体化癌症疫苗成立日期：2021年3月关键词：癌症疫苗融资金额：1700万美元A2轮投资机构：Flerie Invest、Inovio Pharmaceuticals、Korea Investment Partners、Santé Ventures 3月24日，Geneos Therapeutics完成1700万美元A2轮融资，将扩展其在研个体化癌症疫苗GNOS-PV02的1b/2a期临床试验，治疗晚期肝细胞癌（HCC）患者。Geneos Therapeutics致力于利用人体自身免疫系统特异性攻击肿瘤新抗原，开发新型癌症疗法。其专有的GT-EPIC新抗原靶向技术平台基于高度优化的合成DNA质粒，能够针对个体患者体内肿瘤的独特新抗原（癌细胞产生的异常突变）开发高度个体化的治疗方案。该平台的开发具有多种优势：能够驱动强效和广泛的T细胞免疫应答；能够在单一制剂中容纳多种新抗原；以及快速的生产周转时间。由GT-EPIC平台开发的GNOS-PV02，是一款基于DNA质粒递送的新抗原疫苗。通过对每例患者肿瘤进行测序，发现基于每个患者独特的肿瘤突变产生的新抗原，然后将表达这些新抗原的DNA序列导入到DNA质粒中。在该项1b/2a期临床试验中，GNOS-PV02与编码细胞因子IL-12的DNA质粒，和PD-1检查点抑制剂Keytruda联用。Atlas Molecular Pharma获300万美元融资，推动罕见代谢疾病治疗药物的临床开发成立日期：2015年9月融资金额：300万美元投资机构：Inveready、Centre for the Development of Industrial Technology、Ezten Venture Capital Fund2月1日，Atlas Molecular Pharma完成300万美元融资，将推进其候选产品ATL-001的临床开发，用于治疗先天性红细胞生成性卟啉病（CEP）。Atlas Molecular Pharma是由生物科学研究合作中心（CIC bioGUNE）拆分而来，致力于利用药理学伴侣（pharmacological chaperon）开发治疗罕见病的新型疗法。其在研管线ATL-001作为药理学伴侣稳定和恢复UROIIIS的功能。UROIIIS的缺陷导致高浓度的卟啉在体内堆积，对人体组织造成损害，使患者出现一系列显著症状。Nanoligent完成280万欧元的种子轮融资，推进抗肿瘤药物的临床前开发成立日期：2017年3月关键词：纳米药物、靶向疗法融资金额：280万欧元种子轮投资机构：I&I Biotech Fund、Avanteca Partners、IAG Capital Partners今年，Nanoligent完成280万欧元的种子轮融资，将推进其抗肿瘤药物的临床前开发。Nanoligent是巴塞罗那自治大学（UAB）和圣保罗医院研究所（IIB Sant Pau）的衍生公司，致力于利用蛋白质工程和纳米生物技术开发抗肿瘤的靶向药物。根据官网信息，其技术平台可以设计新的蛋白质偶联物，并将其组装成纳米颗粒，避免肾脏清除的同时选择性地靶向细胞。这种蛋白质结构在体内给药时高度稳定，不仅可以实现持久的全身循环，还可以实现特定部位的药物输送。该公司的在研管线NNL-121靶向CXCR4受体，是一种自组装蛋白纳米颗粒，与抗肿瘤药物偶联，能够选择性地杀死表达受体的肿瘤细胞。VIP专享！纵览全球大健康领域融资事件（每日更新）请点击下图访问我们的小程序免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：本文由药明康德内容团队根据公开资料整理编辑，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转发/复制至其他平台。转发授权请在「创鉴汇」微信公众号留言联系我们。读者们请星标⭐创鉴汇，第一时间收到我们的推送新型疗法专题PROTAC疗法 | 细胞疗法 ‍| 基因疗法 | 溶瘤病毒 | mRNA | 反义寡核苷酸 | RNAi | 寡核苷酸疗法 | 抗体偶联药物 | 人工智能Medtech专题生物材料 | 液体活检 | 二尖瓣技术 | 手术机器人 | 神经介入 | POCT | 脑机接口 | 人工心脏 |投资机构专题高瓴创投 | 礼来亚洲基金 | 红杉中国 | 诺华 | RA Capital Management | 启明创投 | 奥博资本 | Alexandria Venture Investments | Flagship pioneering | 腾讯国家园区专题以色列 | 日本 | 英国 | 张江点击“在看”，分享创鉴汇健康新动态

临床1期抗体药物偶联物基因疗法引进/卖出

2022-11-25

·Business Wire

2022 Polycystic Kidney Disease Drugs in Development Report: Featuring Angion Biomedica, ATLAS Molecular Pharma, Bristol-Myers Squibb and Camurus Among Others - ResearchAndMarkets.com

DUBLIN--( BUSINESS WIRE )--The "Polycystic Kidney Disease Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update" report has been added to ResearchAndMarkets.com's offering. This publisher's latest Pharmaceutical and Healthcare disease pipeline guide Polycystic Kidney Disease - Drugs In Development, 2022, provides an overview of the Polycystic Kidney Disease (Genetic Disorders) pipeline landscape. Companies Mentioned AceLink Therapeutics Inc Amathus Therapeutics Inc AmMax Bio Inc Angion Biomedica Corp ATLAS Molecular Pharma SL Bristol-Myers Squibb Co Camurus AB Centessa Pharmaceuticals Plc Chimerna Therapeutics Inc Chinook Therapeutics Inc DiscoveryBiomed Inc Eloxx Pharmaceuticals Inc Galapagos NV Generian Pharmaceuticals Inc GLG Pharma SA Goldfinch Bio Inc Healx Ltd IC-MedTech Inc Ipsen SA Johnson & Johnson Kadmon Holdings Inc ManRos Therapeutics Mironid Ltd Mitotherapeutix LLC Otsuka Pharmaceutical Co Ltd Pano Therapeutics Inc Poxel SA Reata Pharmaceuticals Inc Regulus Therapeutics Inc Shanghai Alebund Pharmaceuticals Ltd Surrozen Inc XORTX Therapeutics Inc Polycystic kidney disease (PKD) is a disorder in which clusters of cysts develop primarily within kidneys. Polycystic kidney disease symptoms may include high blood pressure, back or side pain, headache, blood in urine, frequent urination and kidney failure. The predisposing factors include age and family history. Treatment includes antihypertensive drugs and diuretics. The report provides comprehensive information on the therapeutics under development for Polycystic Kidney Disease (Genetic Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. The Polycystic Kidney Disease (Genetic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Polycystic Kidney Disease and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase III, Phase II, Phase I, Preclinical and Discovery stages are 2, 4, 3, 18 and 14 respectively. Similarly, the Universities portfolio in Phase II, Preclinical and Discovery stages comprises 1, 2 and 2 molecules, respectively. Polycystic Kidney Disease (Genetic Disorders) pipeline guide helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage. The guide is built using data and information sourced from proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis. Scope The pipeline guide provides a snapshot of the global therapeutic landscape of Polycystic Kidney Disease (Genetic Disorders). The pipeline guide reviews pipeline therapeutics for Polycystic Kidney Disease (Genetic Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources. The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages. The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities. The pipeline guide reviews key companies involved in Polycystic Kidney Disease (Genetic Disorders) therapeutics and enlists all their major and minor projects. The pipeline guide evaluates Polycystic Kidney Disease (Genetic Disorders) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type. The pipeline guide encapsulates all the dormant and discontinued pipeline projects. The pipeline guide reviews latest news related to pipeline therapeutics for Polycystic Kidney Disease (Genetic Disorders) Reasons to Buy Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies. Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage. Find and recognize significant and varied types of therapeutics under development for Polycystic Kidney Disease (Genetic Disorders). Classify potential new clients or partners in the target demographic. Develop tactical initiatives by understanding the focus areas of leading companies. Plan mergers and acquisitions meritoriously by identifying key players and it's most promising pipeline therapeutics. Formulate corrective measures for pipeline projects by understanding Polycystic Kidney Disease (Genetic Disorders) pipeline depth and focus of Indication therapeutics. Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and scope. Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline. For more information about this report visit https://www.researchandmarkets.com/r/ii4537

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药物(靶点)	适应症	全球最高研发状态

ATL-001b	卟啉症更多	临床前
ATL-003	朊病毒疾病更多	临床前
环吡酮	红细胞生成性卟啉病更多	临床前
ATL-002 ( FAH )	I型酪氨酸血症更多	药物发现