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2026-06-30

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）

2026-06-01·Molecular therapy. Oncology

Identification of small molecules that enhance aminoglycoside-mediated suppression of CFTR and NF1 nonsense mutations

Article

作者: Kesterson, Robert A ; Moukha-Chafiq, Omar ; Sammons, Joshua ; Chen, Jianguo ; Du, Ming ; Bedwell, David M ; Wen, Hui ; Rowe, Steven M ; Augelli-Szafran, Corinne ; Thrasher, Kari ; Vinson, Paige ; Fu, Lianwu ; Wallis, Deeann ; Bostwick, J Robert ; Keeling, Kim M

Nonsense suppression rescues functional protein from mRNAs containing in-frame premature termination codons (PTCs). This approach employs small molecules that promote insertion of tRNAs at a PTC during translation (called "readthrough"), enabling translation to continue past the PTC to generate a full-length, functional protein. Some aminoglycosides such as G418 promote readthrough; however, their long-term use is thwarted by toxicity and modest potency. ELX-02, a novel synthetic aminoglycoside, was developed to have both enhanced readthrough activity and reduced toxicity in mammalian cells compared to traditional aminoglycosides. Recent studies suggest that at safe doses, aminoglycosides like ELX-02 may not possess sufficient efficacy to provide clinical benefits for most diseases. "Enhancer" molecules have been identified that do not possess readthrough activity themselves but enhance the efficiency of aminoglycoside-mediated readthrough. In this study, three newly identified series of enhancer molecules were tested for the ability to stimulate aminoglycoside-mediated readthrough of PTCs associated with cystic fibrosis and neurofibromatosis type 1. Although none of the enhancers alone induced readthrough, they all significantly increased readthrough via aminoglycosides at low doses (EC₁₀). Overall, our results suggest that enhancer compounds may be a viable way to overcome potency issues associated with aminoglycosides, enabling rescue of protein function from nonsense alleles while minimizing toxicity.

2026-05-01·EUROPEAN RESPIRATORY JOURNAL

Rescue of the CFTR chloride channel with nonsense mutations is markedly improved under inflammatory conditions

Article

作者: Ciciriello, Fabiana ; De Santis, Martina ; Galietta, Luis J V ; Venturini, Arianna ; Montemitro, Enza ; Borrelli, Anna ; Alghisi, Federico ; Guidone, Daniela

Loss of cystic fibrosis transmembrane conductance regulator (CFTR) chloride channel function is the basic defect in cystic fibrosis (CF), a genetic disease that particularly affects the respiratory system, with bacterial infection and severe inflammation. People with CF carrying the most frequent mutation (F508del) or many types of missense mutations can be efficiently treated with drugs, named correctors and potentiators, that improve CFTR protein processing and channel activity. These treatments are ineffective on nonsense mutations that lead to nonsense-mediated RNA decay (NMD) and CFTR protein truncation. We tested the efficacy of a triple combination of small molecules including the readthrough agent ELX-02, the CFTR corrector VX-809, and the eRF3A degrader CC-90009, on cultured airway epithelia from patients carrying the G542X mutation. The treatment resulted in a significant but relatively modest (three-fold) increase in CFTR function. Importantly, the efficacy of the triple drug combination was greatly amplified under inflammatory conditions, i.e. by exposing the epithelia to interleukin (IL)-4 (15-fold increase) or to IL-17A/tumour necrosis factor-α (nine-fold increase). Similar effects were also found in epithelia with the W1282X mutation. The large rescue of CFTR function under inflammatory conditions was paralleled by the appearance of full-length CFTR protein and by the increase in CFTR mRNA. The effect of inflammatory stimuli could be mediated by enhanced translational readthrough and/or reduced NMD. Our results suggest that rescue of CFTR with nonsense mutations could be more effective than expected in vivo . Our findings may also lead to the identification of novel targets to correct the effect of nonsense mutations in CF and other genetic diseases.

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

项与 Exaluren 相关的新闻（医药）

2026-06-11

·中广发资本管理

一家收入为0的临床阶段药企，从OTC粉单市场成功转板纳斯达克！

在生物医药领域，纳斯达克向来是全球创新药企资本化的制高点。从OTC粉单市场成功转板至纳斯达克，不仅意味着企业资本市场层级的跃升，更是对核心研发管线价值的一次系统性重估。近日，一家临床阶段生物制药企业用真实行动印证了这一路径的可行性——Eloxx Pharmaceuticals，一家聚焦RNA调控、小分子药物研发方向的临床阶段生物制药企业，在营收尚未成规模、研发持续投入的情况下，凭借核心研发资产的临床进展与公开市场合规记录，完成了从OTC粉单市场到纳斯达克的阶梯式升级。从OTC粉单市场，到纳斯达克资本市场美东时间2026年6月8日，Eloxx Pharmaceuticals, Inc.注册声明获美国证券交易委员会（SEC）审批生效。6月9日，公司普通股正式在纳斯达克资本市场挂牌交易，股票代码为“ELOX”，正式告别此前在Pink Limited Market粉单市场的交易状态，迈入主流资本市场的行列。回顾Eloxx的转板时间线：2026年5月初提交注册声明，6月1日递交首版S-1/A文件，6月8日注册声明正式生效。从文件提交到获批生效，前后仅约一个月，体现了转板通道的通畅与高效。近年来，OTC转板纳斯达克已形成成熟通道。据市场统计，企业满足财务、市值、流动性等核心指标后即可申请转板，审核流程相对简化，2026年转板规则优化后进一步取消等待期，达标即可申请。Eloxx的案例正是这一机制顺畅运行的生动注脚。核心管线：针对无义突变的小分子基因疗法 Eloxx是一家专注于核糖体RNA靶向遗传疗法的临床阶段生物制药企业，致力于开发口服小分子候选药物，用于治疗由无义突变（nonsense mutation）驱动的罕见遗传病。公司核心技术机制非常独特：通过小分子药物与核糖体RNA复合体结合，使核糖体能够“通读”过早出现的终止密码子，从而恢复全长功能性蛋白的产生，用小分子来模拟基因疗法的作用。Eloxx在SEC招股文件中将自身描述为“核糖体RNA靶向遗传疗法的领导者”，其TURBO-ZM化学平台与氨基糖苷类药物库的技术积累获得市场高度关注。管线构成方面，Eloxx建立了清晰的阶梯式开发序列： ELX-02是公司的核心候选药物，针对携带无义突变的奥尔波特综合征（Alport syndrome, NMAS）。这是一种罕见的遗传性肾病，目前尚无针对病因的获批准疗法。ELX-02已获得美国FDA孤儿药认定（ODD），公司计划在2026年第三季度启动2b期临床试验，初步顶线数据预计于2027年中读出，最终结果预计2027年底公布。 ZKN-013是Eloxx与Almirall全球授权合作的候选药物，针对营养不良性/交界性大疱性表皮松解症（RDEB/JEB）及家族性腺瘤性息肉病（FAP）中携带无义突变的患者群体。ZKN-013已进入临床1期试验阶段，FDA已批准继续给药。此外，Eloxx还布局了针对常染色体显性多囊肾病（ADPKD）、囊肿病以及由无义突变驱动的癌症和罕见眼科遗传病的临床前研发项目，形成了多适应症拓展的储备管线。零收入如何实现转板？美股资本市场定价逻辑的核心 Eloxx的财务状态是典型的临床阶段药企：尚未形成实质性营业收入，持续的研发投入带来亏损，员工总数仅18人，但同时被SEC归为“更小的报告公司”和“新兴成长型公司”。然而，财务现状并未成为其转板纳斯达克的门槛性障碍。从纳斯达克资本市场的上市标准来看，企业可以选择基于净资产、净利润或市值等多种财务路径达标。对于临床阶段生物医药企业而言，获得纳斯达克上市资格通常可通过达成足够的市值与股价等标准实现，而非单纯依赖当期盈利，这为尚未商业化的创新药企提供了可行的上市路径。 Eloxx便是凭借核心管线的临床价值、合规记录以及市场对其进展的认可，满足了纳斯达克资本市场层级的挂牌要求，最终获得交易所审核通过。 2026年6月9日上市首日，Eloxx股价收盘于11.01美元，较前一交易日上涨约10%。其普通股在OTC市场的交易表现、本次公开发行的定价区间及其与核心管线临床价值的关联程度，共同构成了本次转板的逻辑闭环。 OTC市场作为“孵化器”，为企业发展提供了重要的平台基础 Eloxx在OTC市场的挂牌经历并非临时过渡，而是一个相对完整的培育阶段，在此期间持续进行合规信息披露、公司治理优化以及财务体系规范化，为转板到纳斯达克奠定了扎实的合规基础。从资本市场全局来看，这种“OTC挂牌—规范运营—合规达标—主板转板”的阶梯式发展路径，并不局限于Eloxx个案。据市场统计，2025年美国OTC市场共有34家企业成功转板至纳斯达克或纽交所主板，其中56%来自OTCQB板块；纳达斯克因流动性充沛，全年吸引了23家企业转板落户，占比超过68%。对于临床阶段生物医药企业而言，先在美国OTC市场挂牌，一方面可提前融入美国证券监管体系，积累经PCAOB审计的财务记录与合规披露历史，另一方面可以较低的成本获得一定的公开市场曝光，为核心管线的融资和研发提供持续的资金支持。转板纳斯达克：融资通道进一步拓宽，融资成本持续优化 Eloxx从OTC粉单市场成功转板纳斯达克，所带来的资本跃升效应已初步显现。更高的品牌曝光度与市场关注度：纳斯达克作为全球科技与创新企业的首选上市地，具有极高的流动性、机构投资者覆盖率和媒体关注度。Eloxx在转板首日即成为生物技术板块的重点受关注对象之一。更宽的机构投资者基础：纳斯达克上市意味着公司股票可被纳入更多机构投资者的配置范围，包括指数基金、养老金等长线资金，有利于改善股东结构和估值水平。更强的融资能力和融资成本改善趋势：转板为企业后续融资提供了更优的市场环境和更灵活的资本工具选择。更顺畅的资本市场进阶路径：从粉单市场到纳斯达克的跨越，标志着公司完成了初步的资本市场培育和规范运营阶段，未来有望进一步向全球交易所更高层级迈进。总结与展望 Eloxx Pharmaceuticals的成功转板为临床阶段生物医药企业提供了一条清晰的参考路径——不必等待商业化收入的出现，而是让市场用对核心研发资产的定价，来验证上市融资的可行性。从技术平台的角度看，Eloxx的RNA调控与核糖体通读技术在罕见肾病和罕见遗传病领域的差异化定位，构成了其管线价值的基础，为其后续融资和研发推进提供了持续的支撑和验证条件。对正在考虑赴美上市路径的医药企业而言，Eloxx此次“先OTC挂牌、再合规转板至纳斯达克”的经验，在路径选择和节奏规划上提供了有价值的参照——纳斯达克为创新药企敞开着大门，关键是如何找准自己的节奏，做好充足的准备，将研发价值转化为资本价值。责声明：本文信息均来源于公开资料，本公司对上述信息的来源、准确性及完整性不作任何保证。在任何情况下，本文中的信息仅供参考。文章中可能有部分数据、资料及配图来源于其他专业媒体或个人的网络公开报道，如果侵犯您的权益请及时告知我们，我们将于第一时间删除。精彩推荐中广发资本2024冬季项目分享会圆满落幕中广发资本辅导网班科技成功在美国纳斯达克上市上市公司为何需要市值管理？3大核心逻辑 VIE架构2.0：中美监管协调后的合规操作指南

2026-06-11

·埃登華爾環球资本有限公司

生物制药企业Eloxx告别OTC粉单市场，成功转板纳斯达克，募资总额达6600万美元

埃登華爾環球资本背书文件（长按识别下方二维码可查看）美东时间2026年6月8日，一家临床阶段生物制药企业 Eloxx Pharmaceuticals, Inc. （以下简称“Eloxx”或“公司”）注册声明获美国证券交易委员会（SEC）审批生效，6月9日，该企业普通股正式在纳斯达克资本市场（Nasdaq Capital Market）挂牌交易，股票代码为ELOX保持不变，正式告别此前交易的 Pink Limited Market 粉单市场。公开资料显示，Eloxx本次募资总额在扣除发行费用前最高可达6600万美元，承销商预计将在美东时间6月10日左右交付股票及预融资认股权证，并抵扣付款，情况详细如下：本次公开发行，共发行2,975,000股普通股，每股面值0.01美元，发行价为11美元/股。同时，针对部分特定投资者，公司不向其直接配售普通股，改为发行预融资认股权证，最多可认购3,025,000股普通股，单份预融资认股权证发行价为10.99美元，该价格=普通股发行价11美元−每份权证0.01美元行权价。每份预融资认股权证对应可认购1股普通股，行权价0.01美元/股。权证自发行之日起可随时行权，但行权存在持股比例上限约束。同时公司不计划将预融资认股权证在纳斯达克或任何全国性证券交易所、全国公认交易系统挂牌交易。从财务数据看，Eloxx仍处于典型临床阶段药企状态：营业收入尚未形成规模，持续投入研发，亏损仍然存在。但该案例的核心参考价值，并非企业当期盈利表现，而是美股资本市场以临床管线价值、研发核心资产、募资资金投向、公开市场进阶路径为核心维度的综合估值定价逻辑。 Eloxx 专注研发创新小分子候选药物，该类药物能够调节核糖体功能，实现对无义突变（NMs）所生成的提前终止密码子进行通读，从而促使细胞合成全长完整蛋白。以核糖体亚基为作用靶点，为多种遗传性疾病开辟了全新治疗路径。根据人类基因突变数据库的数据，针对任意一种遗传性疾病，无义突变致病的患者占比约10% 至12%。目前已知遗传性疾病逾7000种，全球累计受此类疾病影响的患者达3.5亿人。 Eloxx现阶段核心任务是推进核心候选药物 Exaluren 的临床研发进程，用于治疗各类罕见肾脏疾病。同时还将另一候选药物 ZKN-013 的全球独家开发权授权给Almirall, S.A.，由该企业负责开发，用于治疗罕见皮肤类疾病。此次Eloxx从OTC市场转板至纳斯达克，不仅是公司自身发展的重要里程碑，也为全球中小企业提供了“OTC市场孵化→合规升级→转板纳斯达克”的可借鉴路径。转板至纳斯达克后，不仅有望实质性改善Eloxx的股票交易活跃度、提升其估值定价效率，更能进一步吸引机构投资者与长期资金入场，持续完善投资者多元化结构，为公司核心业务升级、产业链布局及业务规模化扩张筑牢资本根基。关于埃登華爾環球资本埃登華爾環球资本是资本界知名的专业美股FA，成立至今已持续为中国及欧美等地的公司，提供了十六年的海外上市服务，是中国企业赴美上市最早的FA。已协助数十家中外企业登陆美国资本市场，同时每年为各上市公司组织多场大型海外路演活动，其中，包括泽尔西西医疗集团以S1形式登陆美国OTC市场的案例，这是中国企业以S1的形式在美国OTC市场上市的里程碑，以及华景假期上市案例，是埃登華爾環球资本辅导加拿大公司在美国上市的另一代表作。自埃登華爾環球资本成立以来，与OTC市场管理层一直保持着紧密合作关系，埃登華爾環球资本总裁Mary女士与美国OTC市场副总裁及董事Jason Paltrowitz 先生会面时，Jason Paltrowitz首先衷心地表示感谢埃登華爾環球资本十多年为OTC市场输送的大量优质企业，同时他还当面再次介绍了OTC市场的概况并分析了当前环境下OTC市场的优势，同时双方还达成了新的合作共识，埃登華爾環球资本将持续推荐和扶持中小企业赴OTC市场上市。埃登華爾環球资本总裁Mary表示：“有相当一部分中小企业更适合在OTC市场上市，OTC市场对中概股参与者的过渡很有帮助，我们将会帮助更多的中小企业在OTC市场上市，合作是互利的，在帮到企业的同时，也促进了OTC市场的发展，如近日埃登華爾環球资本宣布助力全球上市公司以股票跨境交易方式进入美国资本市场的决定，将会在全球范围内帮助已在全球50余个国家股票交易市场的上市公司，实现这部分符合条件的上市公司股票通过快捷和简化的方式在美国资本市场交易。” 埃登華爾環球资本已协助数十家中外企业登陆美国资本市场，如有赴美上市需求，欢迎通过以下方式联系咨询。联系信息： Mary | 埃登華爾環球资本电话: +86 135 1001 6379 邮箱: mary20116688@163.com 微信: Maryxing55

2026-06-10

·allsci.com

Eloxx Pharma raises USD 66m in public offering to advance nonsense mutation platform

Arlington, Massachusetts-based Eloxx Pharmaceuticals, Inc. (Nasdaq: ELOX) announced the pricing of a USD 66 million public offering of common stock and pre-funded warrants, with proceeds intended to advance its small molecule platform targeting nonsense mutations — a class of genetic defects that account for roughly 10%–15% of all inherited disease cases. The offering coincides with the company’s uplisting to the Nasdaq Capital Market, a structural step that broadens access to institutional investors and provides a more liquid trading venue for ELOX stock. The offering comprises 2,975,000 shares of common stock priced at USD 11.00 per share and pre-funded warrants to purchase up to 3,025,000 shares at USD 10.99 per warrant, with an exercise price of USD 0.01 per share. All securities are being sold by Eloxx, with no secondary component. Leerink Partners and Guggenheim Securities are acting as joint bookrunning managers, with LifeSci Capital serving as passive bookrunner. The Eloxx USD 66 million offering was expected to close on June 10, 2026, subject to customary closing conditions. Company overview and pipeline Eloxx is a clinical-stage biopharmaceutical company focused on developing small molecule product candidates designed to modulate the ribosome and promote readthrough of premature stop codons induced by nonsense mutations. In cells carrying a nonsense mutation, the ribosome encounters a premature stop codon and terminates protein synthesis early, producing a truncated, typically nonfunctional protein. Eloxx’s compounds are designed to allow the ribosome to read through these aberrant stop codons and generate full-length functional protein, potentially addressing the root cause of disease rather than managing downstream symptoms. The company’s lead clinical asset is ELX-02, a small molecule aminoglycoside analogue being developed for rare genetic diseases caused by nonsense mutations, including currently recruiting Phase II trials for cystic fibrosis. ELX-02 has received Orphan Drug Designation from the US FDA for the treatment of Alport Syndrome, a progressive rare kidney disease caused by mutations in genes encoding type IV collagen. The designation provides Eloxx with potential market exclusivity benefits and reduced regulatory fees upon approval. Eloxx’s second program centers on ZKN-013, a molecule derived from its proprietary TURBO-ZM platform, which represents a next-generation approach to nonsense mutation readthrough with a differentiated chemical scaffold compared to ELX-02. In March 2024, Eloxx out-licensed global exclusive rights to ZKN-013 to Almirall, S.A., a Barcelona-based specialty pharmaceutical company, for development in rare dermatological indications including Recessive Dystrophic Epidermolysis Bullosa, Junctional Epidermolysis Bullosa, and Familial Adenomatous Polyposis with nonsense mutations. The transaction included a USD 3 million upfront payment and up to USD 470 million in development, regulatory, and commercial milestones, plus tiered royalties. ZKN-013 was Phase I-ready at the time of signing. Eloxx’s intellectual property foundation rests on a research and license agreement with the Technion Research and Development Foundation in Israel, which underpins the platform technology for both ELX-02 and the TURBO-ZM series. This article was generated with AI assistance and reviewed and edited by the AllSci editorial team Explore more at AllSci News: https://allsci.com/news/ Your email address will not be published. Required fields are marked * Comment * Name * Email * Website Copyright © 2026. AllSci Corp. All rights reserved.

100 项与 Exaluren 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	-	-	DB15080

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
隐性Alport综合征	临床2期	美国	Eloxx Pharmaceuticals, Inc.	2026-06-30
隐性Alport综合征	临床2期	英国	Eloxx Pharmaceuticals, Inc.	2026-06-30
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	網夢構餘窪蓋鏇艱艱齋(鏇鹹築衊範範鏇鬱顧鹹) = 廠顧遞範顧襯獵憲鏇積壓壓簾獵網憲鹹餘鏇壓 (鹽願襯艱顧築蓋淵鹽醖, 觸艱積蓋築獵壓構遞簾 ~ 鹹膚糧獵廠積艱壓願廠) 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	膚蓋艱願夢艱顧醖鹽築(網觸觸顧憲艱築簾積窪) = No ELX-02 related serious adverse events 鹹艱鑰蓋膚襯窪鬱蓋築 (願鏇鹽構觸構製鹽膚築 )	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	糧鏇糧餘鏇餘艱憲醖鑰(壓網膚鏇繭衊繭糧積廠) = 艱網衊鹹夢膚積膚鬱醖鏇襯齋鬱鏇鏇齋壓鬱簾 (淵遞構積鹽壓窪齋鬱顧 ) 更多	积极	2021-11-17
NCT03309605 (Pubmed \| Clin Pharmacol Drug Dev) 人工标引	临床1期	-	62	ELX-02	鑰積醖簾憲憲簾觸網鹽(獵憲醖壓齋築憲製艱積) = 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. 淵襯願獵鑰觸鏇糧願築 (壓鏇醖憲夢鏇蓋積鹽鬱 ) 更多	积极	2021-08-01