A Randomized, Double-Blind, Placebo-Controlled, Delayed-Start Study to Evaluate the Efficacy and Safety of Exaluren in Alport Syndrome Patients With Nonsense Mutations in COL4A3/4/5 Genes

This is a randomized, double-Blind, placebo-controlled study to evaluate the efficacy and safety of exaluren in Alport Syndrome patients with nonsense mutations in COL4A3/4/5 genes.
Targeted 24 patients aged 12 and older will be enrolled in the trial.
The study will be comprised of the following periods for each participant:
* a Screening period of up to 6 weeks (42 days)
* a total Treatment Period of exaluren 0.75 mg/kg or placebo administered daily subcutaneously for 32 weeks: Part 1: patients are randomized to either exaluren or placebo for 16 weeks. Part 2: all patients across both randomized arms receive exaluren for 16 additional weeks.
* a safety/efficacy Follow-up Period of 4 weeks after the last treatment

开始日期2026-05-30

申办/合作机构

Eloxx Pharmaceuticals, Inc.

NCT05448755

/ Completed临床2期

A Phase 2 Open Label Pilot Study to Evaluate the Safety and Efficacy of Subcutaneously Administered ELX-02 in Patients With Alport Syndrome With Col4A5 and Col4A3/4 Nonsense Mutation

This is a Phase 2 open label pilot study to evaluate the safety and efficacy of subcutaneously administered ELX-02 in patients with X-linked or autosomal recessive Alport Syndrome with Col4A5 and Col4A3/4 nonsense mutation.
In total, up to 8 participants, with a minimum of 3 adults, will be enrolled in the trial.
The study will be comprised of the following periods for each participant:
* a Screening period of up to 6 weeks (42 days)
* a total Treatment Period of 8 weeks (60 days)
* a safety/efficacy Follow-up Period of 12 weeks (90 days) after the last treatment The Treatment Period will be a treatment of ELX-02 0.75 mg/kg SC QD for 8 weeks.

开始日期2022-11-28

申办/合作机构

Eloxx Pharmaceuticals, Inc.

NCT04135495

/ Completed临床2期

A Phase 2 Open Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Dose Levels of Subcutaneously Administered ELX-02 in Patients With Cystic Fibrosis With at Least One G542X Allele

This is a Phase 2 open-label, dose-escalation study to evaluate the safety, tolerability, PK, and PD of multiple dose levels of SC administered ELX-02 with and without ivacaftor in patients with CF with at least one G542X allele.
In total, up to 16 patients will be enrolled in the trial; up to 4 patients will be homozygotes for G542X, and the remaining patients will be compound heterozygotes with one G542X or phenotypically similar nonsense allele and any Class 1 or Class 2 mutation.
Each patient will receive up to 5 escalating doses as follows:
* ELX-02 0.3 mg/kg per day SC
* ELX-02 0.75 mg/kg per day SC
* ELX-02 1.5 mg/kg per day SC
* An individualized dose of ELX-02, as high as 3.0 mg/kg per day SC, based on the patients observed safety and tolerability, PK at previous doses and the results of laboratory tests.
* ELX-02 1.5 mg/kg per day SC plus 150 mg ivacaftor every 12 bid

开始日期2019-11-25

申办/合作机构

Eloxx Pharmaceuticals, Inc.

100 项与 Exaluren 相关的临床结果

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100 项与 Exaluren 相关的转化医学

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100 项与 Exaluren 相关的专利（医药）

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项与 Exaluren 相关的文献（医药）

2026-03-01·PEDIATRIC NEPHROLOGY

From RAAS blockade to regenerative medicine: evolving treatment strategies in Alport syndrome

Review

作者: Kim, Jin-Ju ; Lo Re, Claudia ; Fornoni, Alessia

Abstract:

Alport syndrome (AS) is a hereditary glomerulopathy caused by mutations in the COL4A3 , COL4A4 , or COL4A5 genes, leading to progressive kidney decline and extrarenal manifestations. Advances in genetic testing have enabled the reclassification of AS into X-linked, autosomal recessive, and autosomal dominant forms, facilitating more accurate diagnosis and risk stratification. While renin-angiotensin-aldosterone system (RAAS) blockade remains the foundation of treatment to delay kidney failure, it does not directly target the underlying molecular pathology. Adjunctive commercially available metabolic modulators, including SGLT2i, mineralocorticoid receptor antagonists, ezetimibe and GLP-1 receptor agonists, may offer additional kidney protection. Ameliorating therapies being tested in Phase II trials include endothelin receptor antagonists (e.g., atrasentan), dual endothelin receptor antagonist and angiotensin II receptor inhibition (e.g., sparsentan) FXR agonists (e.g., vonafexor), inducers of cholesterol efflux (e.g., VAR200 and R3R01), and NOX1/4 inhibitors (e.g., setanaxib), several of which are currently being evaluated in clinical trials. Novel strategies such as exon skipping, gene editing, and nonsense mutation readthrough (e.g., ELX-02) are advancing toward precision medicine approaches as disease modifying agents targeting the genetic cause of AS. Moreover, therapies targeting mitochondrial function, such as mitophagy enhancers, have demonstrated preclinical promise. Stem cell-based approaches are also being explored for their regenerative and anti-fibrotic effects. This review summarizes the current landscape of AS classification and treatment, highlighting both standard interventions and experimental therapies. Emphasis is placed on the molecular mechanisms underlying podocyte injury and fibrosis, recent preclinical findings, and ongoing clinical trials that may shift future therapeutic paradigms. Graphical abstract

2026-01-01·JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Ribosomal read through as an alternative therapy for patients with hemophilia with nonsense mutations

Article

作者: Liu, Zhigang ; Srivastava, Vishal ; Hussain, Aabid ; Zhang, Bin

BACKGROUND:

Drug-induced ribosomal read through can rescue nonsense mutations by inserting a replacement amino acid at the premature termination codon. Hemophilia A (HA) is well suited for this therapy as even modest increases in cofactor activity can substantially improve severe bleeding symptoms. Identifying highly efficient, low-toxicity read through compounds and pinpointing responsive mutations are critical steps in advancing this therapy.

OBJECTIVES:

To compare the effects of different read through compounds and identify nonsense mutations in F8 that respond to read through treatments.

METHODS:

We used a cell-based model stably expressing B-domain-deleted factor (F)VIII-Gaussia luciferase fusion proteins to evaluate the efficacy of 2,6-diaminopurine (DAP), ELX-02, and G418. Rescue of nonsense mutants was assessed by measuring secreted luciferase activity, as well as FVIII activity and antigen levels. Immunoprecipitation-immunoblotting was used to detect intracellular and secreted FVIII. DAP efficacy was further investigated in vivo using a mouse model expressing nonsense mutant human FVIII delivered via hydrodynamic tail vein injection.

RESULTS:

We tested 26 nonsense mutations, including the most prevalent ones in hemophilia A. All 3 compounds induced varying levels of read through, but DAP showed higher efficacy than ELX-02 and G418 in most mutants. Immunoblotting confirmed the presence of intact fusion proteins in cell lysates and media after DAP and G418 treatments. Nonsense suppression was also confirmed in full-length FVIII. Rescue efficacy was determined by read through efficiency and the effects of replacement amino acids on FVIII secretion and activity. Finally, DAP treatment rescued the p.R355X mutant of human full-length FVIII expressed in mice, yielding detectable levels of antigen in plasma.

CONCLUSION:

Ribosomal read through therapy offers a promising small-molecule treatment option for managing bleeding symptoms in patients with hemophilia A based on individual mutation profiles.

2024-12-01·Molecular Therapy-Nucleic Acids

Reading through the nonsense: Gentamicin and ELX-02 as rescuing therapies for epidermolysis bullosa

作者: Cairo, Mitchell S ; Anderson-Crannage, Morgan ; Liao, Yanling

项与 Exaluren 相关的新闻（医药）

2025-12-30

·征战罕见D研习室

#004-4：治疗阿尔波特综合征（Alport Syndrome）的新药进展（2025）

Bringing medical advances from the lab to the clinic. （👆，征战罕见病，我们一起努力。）根据卫健委公布的罕见病目录（共两批，207种），美国Healsan恒祥医学科技将持续对所有罕见病进行解读和大数据分析，以其能够为从事罕见病研究的生物制药公司、医生科学家及患者提供一些有用的信息。从“无药可治”的遗传诅咒，到基因疗法与小分子药物的百花齐放。过去五年，是阿尔波特综合征（Alport Syndrome）从绝望走向希望的转折期。FDA的审批拉锯战、中国医保的强力覆盖、跨国药企的并购风云。本文将为您深度复盘这场发生在肾脏病领域的“静悄悄的革命”。 #004-1：深度解析阿尔波特综合征（Alport Syndrome）的基因密码 #004-2：肾脏罕见病的新曙光：阿尔波特综合征（Alport Syndrome）在最近3年有哪些诊疗最新进展？（2025） #004-3：研究阿尔波特综合征（Alport Syndrome）的机构及医学专家大数据分析（2025）被改写的命运时钟对于大多数人来说，阿尔波特综合征（以下简称AS）是一个陌生的名词。但在肾脏病罕见病领域，它是仅次于多囊肾的第二大遗传性肾病。长久以来，它被称为“沉默的杀手”：以血尿起病，悄无声息地侵蚀肾脏滤过功能，伴随着听力下降和眼部异常，最终将患者（多为男性青少年）推向终末期肾病（ESRD）的深渊。站在2025年的门槛回望，过去五年（2020-2025）是AS治疗史上最惊心动魄的五年。我们见证了明星药物的折戟沉沙，也目睹了老药新用的意外惊喜，更看到了中国在罕见病保障体系上的“弯道超车”。一、跌宕起伏的全球新药研发（2020-2025）在过去的五年里，全球药企针对AS的研发管线经历了“过山车”般的震荡。1.1 巴多索隆甲酯（Bardoxolone Methyl）：希望与争议的拉锯如果说AS研发史上有一个绕不开的名字，那就是Reata制药公司的巴多索隆甲酯（Bardoxolone Methyl）。高光时刻： 2020-2021年，该药一度被视为AS患者的“救命稻草”。其CARDINAL三期临床试验显示，药物能显著改善患者的估算肾小球滤过率（eGFR）。至暗时刻： 2022年2月，美国FDA发出了一封“完全回应函”（CRL），拒绝批准该药上市。FDA顾问委员会以全票通过的决议，质疑该药虽然提升了eGFR数据，但可能并未真正延缓肾衰竭进程，且存在加重心脏负担的风险。后续震荡：这一决定在当时重创了AS患者社区的信心。但在日本，该药（商品名Ayverdi）早前已获批用于糖尿病肾病，其在AS领域的探索并未完全终止，日本及部分亚洲地区的真实世界研究仍在继续，试图回答“安全性与疗效平衡”的终极问题。1.2 内皮素受体拮抗剂（ERA）的崛起：Atrasentan与Sparsentan 随着抗氧化通路的受挫，科学家们将目光转向了血流动力学与抗纤维化机制。 Atrasentan（阿曲生坦）的接力： 2023-2024年，Chinook Therapeutics（后被诺华Novartis收购）公布了Atrasentan治疗AS的AFFINITY研究数据。结果显示，该药能显著降低蛋白尿（UPCR），且耐受性良好。诺华的入局，标志着制药巨头开始重注这一罕见病领域。 Sparsentan（司帕生坦）的双重打击：作为一种双重内皮素血管紧张素受体拮抗剂（DEARA），Sparsentan此前已在IgA肾病上大获成功（商品名Filspari）。2024-2025年期间，其针对局灶节段性肾小球硬化（FSGS）和AS的临床数据备受瞩目。作为一种能同时阻断两条导致肾脏损伤通路的药物，它被认为是未来5年最有可能改变AS治疗格局的重磅炸弹。1.3 “老药新皇”：SGLT2抑制剂的跨界统治如果说上述新药还在审批的路上，那么SGLT2抑制剂（列净类药物）则是过去五年AS患者触手可及的“实惠红利”。 DAPA-CKD与EMPA-KIDNEY研究：虽然这些大型研究主要针对普通慢性肾病，但其亚组分析和后续的真实世界证据表明，达格列净（Dapagliflozin）和恩格列净（Empagliflozin）对于非糖尿病肾病（包括AS）同样具有延缓肾功能恶化的作用。指南变更： 2023-2024年，全球主要肾脏病指南（KDIGO）陆续更新，SGLT2抑制剂正式成为包括AS在内的蛋白尿性肾病的“标准治疗”支柱之一，与RAS阻断剂（普利/沙坦类）并驾齐驱。二、前沿科技--直击基因核心（2025展望） AS的根源在于COL4A3、COL4A4或COL4A5基因突变，导致IV型胶原蛋白合成障碍。药物只能延缓，唯有基因疗法能谈“治愈”。2.1 无义突变通读疗法（Read-through Therapy）对于由于“提前终止密码子”（无义突变）导致的AS患者，Eloxx Pharmaceuticals研发的ELX-02曾寄予厚望。虽然其在囊性纤维化上的表现波折，但在2023-2025年间，针对AS的临床前及早期临床探索仍在进行。这种疗法试图让细胞“无视”错误的停止信号，继续合成完整的胶原蛋白。2.2 基因编辑与外显子跳跃外显子跳跃（Exon Skipping）：这一技术在杜氏肌营养不良（DMD）中已获批，目前正在向AS领域迁移。通过跳过带有突变的外显子，产生一种“缩短但仍有功能”的胶原蛋白，有望将重症AS转化为轻症。 CRISPR/Cas9： 2024年，多篇顶级学术期刊报道了利用基因编辑技术在小鼠模型中修复Col4a5突变的成功案例。虽然距离人体应用尚有距离，但这盏灯已经点亮。三、中国力量：政策红利与医保破局视线回到国内。过去五年，是中国罕见病保障体系从“荒芜”到“绿洲”巨变的五年。3.1 身份的确立：罕见病目录的护身符早在2018年，Alport综合征就被列入中国《第一批罕见病目录》。这看似简单的一行字，却是后续所有政策倾斜的基石。这意味着，AS相关的药物审批可以走“绿色通道”，科研项目可以获得国家专项支持。3.2 诊断的普及：基因检测平民化五年前，确诊AS往往需要进行痛苦的肾穿刺活检，且电镜检查普及率低。技术下沉： 2020-2025年，随着高通量测序（NGS）成本的断崖式下跌，基因检测已成为国内三甲医院诊断AS的“金标准”。医保覆盖探索：在上海、北京等地的部分医保试点中，遗传性疾病的基因检测费用开始尝试纳入报销或补充医疗保险范围，极大地降低了患者的确诊门槛。3.3 药物可及性：医保目录（NRDL）的强力托底中国患者最关心的永远是：“这药进医保了吗？” 普惠药（SGLT2抑制剂）：达格列净、恩格列净等SGLT2抑制剂早已进入国家医保目录，价格从最初的“贵族药”降至几块钱一片的“白菜价”。对于中国AS患者而言，这是一个每月仅需几十元就能用得起的顶级护肾方案。 RAAS阻断剂：传统的ACEI/ARB类药物早已集采，成本几乎可以忽略不计。特许药械政策：对于尚未在国内上市的海外新药（如部分处于临床后期的创新药），“港澳药械通”和海南博鳌乐城国际医疗旅游先行区提供了宝贵的窗口。过去三年，已有数位罕见病肾病患者通过这些渠道提前用上了全球最新药物。3.4 中国本土创新中国药企不再仅仅是旁观者。恒瑞医药、豪森药业等本土巨头在慢性肾病（CKD）抗纤维化领域的布局，虽然多从糖尿病肾病切入，但其机制往往覆盖AS。科创板生物科技公司正在积极开发针对胶原蛋白合成途径的siRNA（小干扰RNA）疗法，部分早期管线已在2024年公布了临床前数据。结语：黎明已至，静待花开 2020年时，我们还在为巴多索隆甲酯（Bardoxolone Methyl）的临床数据焦虑； 2022年时，我们曾因FDA的拒信而感到寒意；但到了2025年，随着SGLT2抑制剂的普及、内皮素受体拮抗剂的成熟以及基因疗法的萌芽，阿尔波特综合征的治疗拼图从未如此完整。这个疾病依然残酷，但它不再是不可战胜的“黑箱”。对于中国患者而言，背靠国家医保的强大议价能力和日益开放的新药准入政策，“活得更久、活得更好”已不再是奢望，而是触手可及的现实。我们相信，下一个五年，将是AS从“可控”走向“可治愈”的关键五年。征战罕见D研习社将持续发布罕见病最新研究进展和大数据分析报告，和各位同仁一起探索罕见病的优化诊疗方法。投稿及商务：加微信号healsan 网址： https://healsan.com/ （👆，征战罕见病，我们在行动！） ▼ Healsan医路成长更多免费资源：（点击👆图片，进入自己感兴趣的专辑。或点击“资源”，浏览本公众号所有资源）

基因疗法临床3期并购

2025-03-26

·梅斯医学

3岁娃每天喝6升水、反复骨折！家长以为“缺钙”，真相竟是基因突变导致的罕见病！

3岁的男孩豆豆（化名）从1岁起就成了“水桶娃”——每天狂喝6升水，尿布两小时就湿透，半夜哭闹要喝水，家人以为他“天生爱喝水”。可随后他接连骨折三次：翻身压到手臂骨折、学走路崴脚骨折，甚至拍后背哄睡都能拍断肋骨！X光片显示他的骨头像“被虫蛀空的朽木”，骨密度比80岁老人还低。眼科医生用裂隙灯检查时，发现豆豆的角膜上布满“星尘”般的胱氨酸结晶，肾脏B超显示双肾弥漫性钙化，基因检测揪出罪魁祸首——CTNS基因突变，确诊为胱氨酸贮积症！一种因细胞“垃圾处理站”故障，胱氨酸结晶“填满”全身器官的罕见遗传病！ 1 常染色体隐性遗传病胱氨酸贮积症是一种常染色体隐性遗传病，是由编码溶酶体膜上的胱氨酸转运蛋白(site-directed mutagenesis)的CTNS基因突变引起，当该基因发生突变时，溶酶体中的胱氨酸便无法被正常转运到细胞质，从而在全身各处的细胞溶酶体内异常蓄积导致发病。 2 临床表现及分型胱氨酸贮积症分为婴儿型（肾病型）、迟发型（青少年型）和成人型（良性或眼型）。婴儿型为最常见且最严重，表现为蛋白尿、肾功能恶化和终末期肾病。肾脏病理表现包括足细胞脱落、胱氨酸结晶等。肾外器官也受累，眼部可见角膜结晶，导致视力丧失。营养不良和矿物质代谢紊乱可能导致骨病、肌肉萎缩和生长不良，身材矮小是慢性肾病患者的11倍。内分泌问题包括甲状腺功能减退、无精子症（男性）和青春期延迟（女性）。未经治疗者，20岁后可能出现神经系统症状，如行走和吞咽困难、智力减退等。 3 诊断胱氨酸贮积症的早期诊断对改善预后至关重要，未经治疗会导致全身脏器受累，预后极差。外周血白细胞中胱氨酸水平升高是确诊的金标准。裂隙灯检查可用于1岁以上儿童的角膜结晶诊断，而反射共焦显微技术（RCM）为年幼患者提供了快速、无痛的检测手段。近年来，新的筛查和诊断方法不断涌现。智能手机结合镜面反射可用于初步筛查角膜胱氨酸晶体，适用于基层诊所。皮内反射共聚焦显微镜和谱域光学相干断层扫描（SD-OCT）可以定量检测皮肤内胱氨酸结晶，并评估视网膜病变。角膜晶体评分可通过光密度测量直观了解角膜受累程度。对于高危妊娠，CTNS基因的分子分析可用于产前筛查，出生后通过DNA检测（如全外显子组测序或下一代测序）进行新生儿筛查，是最有效的二级预防措施。 4 治疗胱氨酸贮积症的治疗包括以下几个方面：一般治疗：包括高热量饮食、维持水电解质平衡等基础治疗。内分泌系统受累可使用激素替代治疗。终末期肾病患者可选择肾透析或肾移植。年轻男性可通过冷冻精子或睾丸精子提取手术保持生育机会。胱氨酸消耗疗法：半胱胺是唯一靶向治疗药物，常用速释酒石酸半胱胺，但因不良反应和给药频繁影响患者依从性，缓释剂型改善了这一问题，但可能导致纤维化结肠病。角膜结晶患者可使用半胱胺滴眼液，如Cystadrops或缓释半胱胺微球滴眼剂。血浆半胱胺或胱氨酸含量的监测可减少中毒风险。其他疗法：由于半胱胺对已出现的范可尼综合征、无精症和角膜结晶无效，研究提供了新的治疗方向。金雀异黄素和木犀草素可降低胱氨酸浓度并改善自噬。比卡鲁胺与半胱胺联合使用可纠正近端肾小管损伤。依维莫司等mTOC1通路抑制剂、IL-1受体拮抗剂等也在探索中。基因编辑技术和自体干/祖细胞移植在小鼠模型中已验证有效，可能为患者提供长效治疗。饮食疗法：通过补充赖氨酸或精氨酸，抑制肾脏巨蛋白途径，已在小鼠模型中证实有效，提供了新的饮食治疗选择。这些治疗方法的安全性和有效性正在不断验证，并为胱氨酸贮积症患者提供了多种潜在治疗途径。参考资料： [1]寇文韬,李燕.胱氨酸贮积症发病机制及诊疗新进展[J].疑难病杂志,2025,24(03):376-380. [2]李晓侨,巩纯秀.胱氨酸贮积症诊疗进展[J].临床儿科杂志,2020,38(02):156-160. [3] Kerem E.Elx-02:an investigational read-through agent for the treatment of nonsense mutation-related genetic disease[J].ExpertOpiniononInvestigational Drugs,2020,29(12):1347-1354.D0l:10.1080/13543784.2020.1828862. 来源 | 梅斯医学编辑 | wanny 神经系统罕见病交流群 ↓点击下方“阅读原文”，下载梅斯医学APP吧！

2024-11-12

·GlobeNewswire-Health Care

Eloxx Pharmaceuticals Announces Key Corporate Accomplishments

Data from Eloxx’s proof-of-concept trial in patients with nonsense mutation Alport Syndrome (NMAS) presented in late-breaking presentation at American Society of Nephrology (ASN) Kidney Week New protein analyses confirm production of both collagen alpha 4 and collagen alpha 5 in patients post treatment with ELX-02 Results justify the need to conduct a larger clinical trial of ELX-02 in NMAS to confirm clinical benefit U.S. Food and Drug Administration (FDA) allows for continued dosing of subjects in Phase 1 trial of ZKN-013 WATERTOWN, Mass., Nov. 12, 2024 (GLOBE NEWSWIRE) -- Eloxx Pharmaceuticals, Inc. (OTC: ELOX), a leader in ribosomal RNA-targeted genetic therapies for rare diseases with nonsense mutations, today provided significant key corporate updates. “The demonstration of both collagen alpha 4 and collagen alpha 5 in patients in our proof-of-concept study of ELX-02 in NMAS patients is an important milestone in the development of this crucial potential therapy. These new results, presented in a late-breaking presentation at ASN Kidney Week, strongly justify the need to conduct a larger trial to confirm these encouraging results in this underserved patient population,” said Sumit Aggarwal, President and Chief Executive Officer of Eloxx. “Also, the FDA allowing for continued dosing of subjects in the Phase 1 trial for ZKN-013 is a substantial achievement for the program and makes Eloxx eligible for the next milestone payment in the company’s agreement with Almirall, once confirmed by our partner, Almirall.” Additional Alport Syndrome data presented at the ASN Kidney Week In late October, Daniel Gale, Professor of Nephrology at University College London Department of Renal Medicine, presented additional results from Eloxx’s proof-of-concept study in three NMAS patients treated with ELX-02 in a late-breaking presentation at ASN Kidney Week. The presentation was titled: “Small molecule premature termination codon readthrough therapy: a Phase 2 pediatric and adult trial in Nonsense Mutation Alport syndrome” at the ASN Kidney Week conference. “Although this was a small trial of limited duration, the appearance of new Type IV Collagen in the glomerular basement membranes is the first time a missing podocyte protein has been reconstituted in a human and provides evidence that ELX-02 is acting as intended in the kidneys of patients. A longer, controlled study will be needed to quantify the clinical effectiveness of this therapy,” said Professor Gale. New protein immunostaining and mass spectrometry analyses that were presented, confirmed the production of both collagen a4 (IV) and collagen a5 (IV) proteins in NMAS patients treated with ELX-02. Prior to treatment, collagen a4 (IV) and a5 (IV) were absent in the glomerulus in all three patients. Post treatment biopsy of the patient with the largest improvement in Filtration Slit Density showed a 34% increase in collagen a4 (IV) immunostaining intensity and presence of collagen a3/a4/a5 (IV) peptide with a greater than 6-fold increase in collagen a4 and detectable collagen a5 consistent with formation of a functional protein induction. No collagen alpha 5 was detected pre-treatment in this patient. These additional data support ELX-02’s mechanism of protein induction, and the observed morphology change and reduced foot process effacement in all the patients that was previously reported. The results also justify the conduct of a larger clinical trial with longer ELX-02 treatment duration to validate the observed clinical benefit in NMAS patients. The demonstrated readthrough of premature stop codons also suggests ELX-02 has potential in other rare kidney diseases, including autosomal dominant polycystic kidney disease and cystinosis. FDA allows for continued dosing of subjects in Phase 1 trial of ZKN-013 Following successful dosing of the initial subjects, FDA has allowed continued dosing of healthy volunteers for ZKN-013 for the treatment of recessive dystrophic epidermolysis bullosa and junctional epidermolysis bullosa. Eloxx is awaiting confirmation from Almirall to proceed with the study based on their review of the results and FDA feedback. ZKN-013 has been exclusively licensed to Almirall, S.A. (BME:ALM), who has global rights to develop and commercialize ZKN-013. Under the terms of the license agreement, Eloxx is eligible for additional development, regulatory and sales milestones of up to $470 million as well as tiered royalties on any potential global sales. About Eloxx Pharmaceuticals Eloxx Pharmaceuticals, Inc. is engaged in the science of ribosome modulation, leveraging its innovative TURBO-ZM™ chemistry technology platform in an effort to develop novel Ribosome Modulating Agents (RMAs) and its library of Eukaryotic Ribosome Selective Glycosides (ERSGs). Eloxx’s lead investigational product candidate, ELX-02, is a small molecule drug candidate designed to restore production of full-length functional proteins. ELX-02 is in Phase 2 clinical development for the treatment of Alport syndrome in patients with nonsense mutations. For more information, please visit www.eloxxpharma.com. Forward-looking StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements other than statements of historical facts contained in this press release, including without limitation, statements regarding the potential future payments and future benefits under the license agreement, achievement of key milestones under the license agreement, the expected timeline for clinical development, the efficacy of the Company’s product candidates ZKN-013 and ELX-02, the Company’s expected and planned communications with and submissions to the FDA and anticipated funding under the bridge loan are forward-looking statements. Forward-looking statements can be identified by the words “aim,” “may,” “will,” “would,” “should,” “expect,” “explore,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “contemplate,” “believe,” “estimate,” “predict,” “potential,” “seeks,” or “continue” or the negative of these terms similar expressions, although not all forward-looking statements contain these words. Forward-looking statements are based on management's current plans, estimates, assumptions and projections based on information currently available to us. Forward-looking statements are subject to known and unknown risks, uncertainties and assumptions, and actual results or outcomes may differ materially from those expressed or implied in the forward-looking statements due to various important factors, including, but not limited to: the Company’s incurrence of significant losses; the Company has identified conditions and events that raise substantial doubt about its ability to continue as a going concern; its ability, or its licensees’ ability, to progress any product candidates in preclinical or clinical trials; the uncertainty of clinical trial results and the fact that positive results from preclinical studies are not always indicative of positive clinical results; the scope, rate and progress of the Company’s, and its licensees’ preclinical studies and clinical trials and other research and development activities; the competition for patient enrollment from drug candidates in development; the Company’s ability to obtain the capital necessary to fund its operations; the cost of filing, prosecuting, defending and enforcing any patent claims and other intellectual property rights; the Company’s ability to obtain financing in the future through product licensing, public or private equity or debt financing or otherwise; general business conditions, regulatory environment, competition and market for the Company’s products; and business ability and judgment of personnel, and the availability of qualified personnel and other important factors discussed under the caption “Risk Factors” in the Company’s Quarterly Report on Form 10-Q for the quarterly period ended September 30, 2023, as any such factors may be updated from time to time in the Company’s other filings with the SEC, accessible on the SEC’s website at www.sec.gov and the “Financials & Filings” page of the Company’s website at https://investors.eloxxpharma.com/financials-filings. All forward-looking statements speak only as of the date of this press release and, except as required by applicable law, the Company has no obligation to update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise. ContactJohn Woolfordjohn.woolford@westwicke.com 443.213.0506 Source: Eloxx Pharmaceuticals

临床结果引进/卖出临床2期临床1期

100 项与 Exaluren 相关的药物交易

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KEGG	Wiki	ATC	Drug Bank
-	-	-	DB15080

研发状态

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适应症	最高研发状态	国家/地区	公司	日期
隐性Alport综合征	临床2期	美国	Eloxx Pharmaceuticals, Inc.	2026-05-30
隐性Alport综合征	临床2期	英国	Eloxx Pharmaceuticals, Inc.	2026-05-30
X-Linked Alport Syndrome	临床2期	澳大利亚	Eloxx Pharmaceuticals, Inc.	2022-11-28
X-Linked Alport Syndrome	临床2期	英国	Eloxx Pharmaceuticals, Inc.	2022-11-28
囊性纤维化	临床2期	澳大利亚	Eloxx Pharmaceuticals, Inc.	2019-11-05
囊性纤维化	临床2期	德国	Eloxx Pharmaceuticals, Inc.	2019-11-05
囊性纤维化	临床2期	以色列	Eloxx Pharmaceuticals, Inc.	2019-11-05
胱氨酸贮积症	临床2期	加拿大	Eloxx Pharmaceuticals, Inc.	2019-08-02
遗传病	临床1期	-	Eloxx Pharmaceuticals, Inc.	2016-07-01

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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


NCT05448755 (CTgov)	临床2期	X-Linked Alport Syndrome \| Alport 综合征	3	ELX-02	餘窪積鑰壓網積糧齋觸(膚艱觸糧選獵齋襯餘鬱) = 膚鑰構簾襯廠廠鹹鬱築夢淵鹽廠鹹鹹範膚繭鹽 (蓋膚獵鏇廠獵淵遞網艱, 糧廠繭鹹遞範膚鑰醖窪 ~ 獵觸簾艱淵願憲鬱鬱鹽) 更多	-	2026-02-24
NCT04126473 \| NCT04135495 (Corporate Publications) 人工标引	临床2期	囊性纤维化	31	ELX-02	醖網顧鹹窪衊鏇製膚壓(範糧廠餘壓鑰遞築夢廠) = No ELX-02 related serious adverse events 窪範範簾襯窪鹽鑰範鏇 (觸構範醖膚選製鹽窪願 )	不佳	2022-09-14
NCT04126473 \| NCT04135495 (Corporate Publications) 人工标引	临床2期	囊性纤维化	31	Ivacaftor+ELX-02		不佳	2022-09-14
NCT04126473 \| NCT04135495 (Corporate Publications) 人工标引	临床2期	囊性纤维化	-	ELX-02	齋顧獵鹹淵選鏇鏇顧艱(齋窪夢衊夢窪餘範廠鹹) = 鹽鬱蓋廠遞鬱衊顧網顧憲鹹積夢廠鬱淵繭繭夢 (艱製鹹壓鏇遞築範餘壓 ) 更多	积极	2021-11-17
NCT03309605 (Pubmed \| Clin Pharmacol Drug Dev) 人工标引	临床1期	-	62	ELX-02	鑰衊廠顧蓋製願壓壓願(積膚選淵蓋鑰醖範鬱獵) = At the top dose of 5.0 mg/kg, 1 of 6 subjects experienced auditory threshold changes in which ototoxicity could not be clearly ruled out, and 2 of 6 had hearing threshold changes consistent with possible ototoxicity. Two of 3 subjects receiving placebo in the 5.0 mg/kg group also had significant hearing threshold changes. 繭積壓艱範鏇壓艱壓壓 (積廠範憲築範遞衊網願 ) 更多	积极	2021-08-01