预约演示

更新于：2025-05-07

Polycystic Kidney Diseases

多囊肾

更新于：2025-05-07

基本信息

别名

Disease, Polycystic Kidney、Disease, Polycystic Renal、Diseases, Polycystic Kidney

+ [58]

简介

Hereditary diseases that are characterized by the progressive expansion of a large number of tightly packed CYSTS within the KIDNEYS. They include diseases with autosomal dominant and autosomal recessive inheritance.

关联

项与多囊肾相关的药物

Bosutinib Monohydrate

伯舒替尼

靶点

Bcr-Abl x SRC

作用机制

Bcr-Abl抑制剂 [+1]

在研机构

原研机构

在研适应症

非在研适应症

最高研发阶段批准上市

首次获批国家/地区

美国

首次获批日期2012-09-04

Tolvaptan

托伐普坦

靶点

AVPR2

作用机制

AVPR2拮抗剂

在研机构

Otsuka Pharmaceutical Co., Ltd. [+5]

原研机构

Otsuka Holdings Co., Ltd.

在研适应症

腹水 [+7]

非在研适应症

慢性低钠血症 [+12]

最高研发阶段批准上市

首次获批国家/地区

美国

首次获批日期2009-05-19

Mozavaptan Hydrochloride

盐酸莫扎伐普坦

靶点

AVPR2

作用机制

AVPR2拮抗剂

在研机构

Otsuka Holdings Co., Ltd.

原研机构

Otsuka Holdings Co., Ltd.

在研适应症

低钠血症

非在研适应症

高血压 [+2]

最高研发阶段批准上市

首次获批国家/地区

日本

首次获批日期2006-07-26

297

项与多囊肾相关的临床试验

NCT06786260

/ Not yet recruitingN/AIIT

Di-PKD: A Pilot Trial of a 12-Month Targeted Dietary Intervention on Cyst Progression in Patients With Autosomal Dominant Polycystic Kidney Disease

The goal of this interventional study is to evaluate the impact of a specific diet (low salt, limited caffeine, high potassium, low phosphate, limited protein, limited carbohydrate intake, adequate water intake) on changes in serum and urinary biomarkers, total kidney volume (TKV), and cyst progression in subjects with autosomal dominant polycystic kidney disease (ADPKD).
Researchers will compare the results of subjects in the study diet (interventional) arm to the results of subjects in the regular diet (control) arm.

开始日期2025-07-01

申办/合作机构

Loma Linda University

NCT06902558

/ Not yet recruiting临床2期

A Phase 2, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Safety and Efficacy of ABBV-CLS-628 in Adult Subjects With Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Autosomal Dominant Polycystic Kidney Disease (ADPKD) is the most common genetic cause of kidney disease that causes fluid-filled cysts to develop in the kidneys. The purpose of this study is to assess the safety and efficacy of ABBV-CLS-628 for the treatment of ADPKD in adult participants.
ABBV-CLS-628 is an investigational drug being developed for the treatment of ADPKD. Participants are placed in 1 of 4 groups, called treatment arms. Each group receives a different treatment. There is a 1 in 4 chance that participants will be assigned to placebo. Around 240 adult participants with ADPKD will be enrolled at approximately 100 sites worldwide.
Participants will receive IntraVenous ABBV-CLS-628 or placebo every 4 weeks for 92 weeks. Participants will be followed for up to 15 weeks.
There may be higher treatment burden for participants in this trial compared to their standard of care . Participants will attend regular visits during the study at a hospital or clinic. The effect of the treatment will be checked by medical assessments, blood tests, checking for side effects and completing questionnaires.

开始日期2025-05-01

申办/合作机构

AbbVie, Inc.

[+1]

NCT06714006

/ Recruiting临床1期

A Phase 1 Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Intravenously Administered PYC-003, a Peptide-phosphorodiamidate Morpholino Oligonucleotide Conjugate, in Healthy Adult Participants and Adult Participants With Confirmed PKD1 Mutation-associated Autosomal Dominant Polycystic Kidney Disease

This is a Phase 1, First-in-Human study to evaluate the safety and tolerability of PYC-003 in healthy adult participants and adult participants with confirmed PKD1 mutation-associated ADPKD. There are 2 parts in this study, i.e. Part A and Part B.

开始日期2025-04-07

申办/合作机构

PYC Therapeutics Ltd.

100 项与多囊肾相关的临床结果

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100 项与多囊肾相关的转化医学

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0 项与多囊肾相关的专利（医药）

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13,889

项与多囊肾相关的文献（医药）

2025-12-31·Renal Failure

Effectiveness of abdominal bracing core exercises as rehabilitation therapy for reducing abdominal symptoms in patients with autosomal dominant polycystic kidney disease and significant polycystic liver disease

Article

作者: Sim, Gwangeon ; Yoo, Jaeyeong ; Kim, Jisu ; Choi, Kwang-Ho ; Kim, Hyunsuk ; Song, Young-Jin ; Cho, Myunkyu ; Yoon, Jong-Woo ; Kim, Jin Eop ; Jeon, Sohyun

In patients with autosomal dominant polycystic kidney disease (ADPKD) who also have polycystic liver disease (PLD), organomegaly often leads to abdominal symptoms. Abdominal bracing core (ABC) exercises have been validated as effective for alleviating chronic back pain. The purpose of this study was to assess the effectiveness of ABC rehabilitation exercises in reducing pain in ADPKD patients with significant PLD. Significant PLD was defined as a height-adjusted total liver volume (htTLV) exceeding 1,600 mL/m. Both the control groups (n = 11) and intervention (n = 12) and received an outpatient consultation on nutrition and exercise; however, only the intervention group participated in ABC exercises. After a 3-month biweekly intervention, changes in pain assessed by the Korean Oswestry Disability Index, abdominal symptoms, quality of life (QoL) by the second version of the short-form 36-item Health Survey, and bioelectrical impedance analysis (BIA) were analyzed. The participants comprised 23 individuals (male: 4, female: 19). Their mean age was 54, and the mean ± SD of htTLV was 2,706 ± 1,335 mL/m. The mean ± SD of eGFR was 53.9 ± 29.0 mL/min/1.73 m². After the intervention, pain and pressure-related symptoms significantly decreased in some cases; however, gastrointestinal symptoms did not improve. Pain (control vs. intervention: -1.9 vs. 1.0) and QoL (1.20 vs. 0.93) and showed significant improvements. The results of BIA indicated a noticeable change in the soft lean mass of the proximal body (0.4 vs. -0.2). Our study demonstrates that ABC exercise is effective in alleviating pain and increasing soft lean mass in ADPKD patients who have significant PLD.

2025-12-31·RNA Biology

Translational regulation of PKD1 by evolutionarily conserved upstream open reading frames

Review

作者: Chen, Lei ; Zhu, Ye ; Liu, Xiangshen ; Gao, Xia ; Wang, Dong

Mutations in PKD1 coding sequence and abnormal PKD1 expression levels contribute to the development of autosomal-dominant polycystic kidney disease, the most common genetic disorder. Regulation of PKD1 expression by factors located in the promoter and 3´ UTR have been extensively studied. Less is known about its regulation by 5´ UTR elements. In this study, we investigated the effects of uORFs and uORF-affecting variants by combining bioinformatic analyses, luciferase reporter assays, RT-qPCR and immunoblotting experiments. Our analyses demonstrate that PKD1 mRNA contains two evolutionarily conserved translation-inhibitory uORFs. uORF1 is translatable, and uORF2 is likely not translatable. The 5´ UTR and uORFs do not modulate downstream protein output under endoplasmic reticulum stress and oxidative stress conditions. Some of uORF-perturbing variants in the SNP database are predicted to affect gene translation. Luciferase reporter assays and RT-qPCR results reveal that rs2092942382 and rs1596636969 increase, while rs2092942900 decreases main gene translation without affecting transcription. Antisense oligos targeting the uORFs reduce luciferase protein levels without altering luciferase mRNA levels. Our results establish PKD1 as a novel target of uORF-mediated translational regulation and mutations that perturb uORFs may dysregulate PKD1 protein level.

2025-12-31·Renal Failure

Autosomal dominant polycystic kidney disease: an overview of recent genetic and clinical advances

Review

作者: Chebib, Fouad T. ; Salih, Mahdi ; Rahbari-Oskoui, Frederic F. ; Borghol, Abdul Hamid ; Hanna, Christian ; Bou Antoun, Marie Therese

Autosomal Dominant Polycystic Kidney Disease (ADPKD) is the most common inherited kidney disease, characterized by the progressive development of multiple kidney cysts, leading to a gradual decline in kidney function. ADPKD is also the fourth leading cause of kidney failure (KF) in adults. In addition to kidney manifestations, ADPKD is associated with various extrarenal features, including liver cysts, cardiovascular abnormalities, intracranial aneurysms, and chronic pain with significant impact on patients' quality of life. While several disease-modifying agents have been tested in ADPKD, tolvaptan remains the only approved drug by the US Food and Drug Administration. The Mayo Imaging Classification is currently the most practical tool for predicting rate of kidney disease progression in ADPKD. This review provides a comprehensive overview of ADPKD, focusing on its genetics, pathophysiology, clinical presentation, management, and prognostic tools. Advances in diagnostic imaging and genetic testing have improved the early detection of ADPKD, allowing better classification of patients and prediction of KF. The review also discusses current therapeutic approaches to ADPKD, including tolvaptan, a vasopressin V2-receptor antagonist. Additionally, we address specific issues in children and pregnant individuals with ADPKD. Despite substantial progress in understanding ADPKD, there is a large need for additional effective treatments and prognostic markers to provide a more personalized care for these patients.

551

项与多囊肾相关的新闻（医药）

2025-05-05

·药时代

8亿美元！诺华制药拟收购肾脏疾病药物研发公司Regulus

正文共： 1348字 8图预计阅读时间： 4分钟诺华计划以8亿美元预付款收购位于圣地亚哥的生物科技公司Regulus Therapeutics，该公司专注于microRNA药物的研发。此次收购的核心资产是farabursen，这是一种针对与肾病相关的miR-17的候选药物，刚完成针对多囊肾病患者的1b期临床试验。收购Regulus的协议中，股东将获得每股7美元的现金，这一价格是周二收盘价的两倍多。此外，诺华还承诺，若达到某些未公开的审批标准，将额外支付每股7美元。Regulus成立于2007年，由Alnylam和Ionis联合创立，其技术灵感来源于《自然》杂志上一篇关于合成寡核苷酸抑制microRNA的论文。公司成立初期发展势头良好，曾与赛诺菲和阿斯利康等大型药企合作。然而，近年来研发遭遇连续失败，股价自2021年以来一直未能突破4美元。farabursen于2022年进入临床试验，针对的是研究人员认为与肾病相关的miR-17。目前，多囊肾病患者治疗选择有限，主要依赖托伐普坦等药物来延缓肾功能恶化。诺华在肾病领域的布局并非首次。2023年，诺华以30亿美元收购Chinook Therapeutics，获得了治疗IgA肾病的新药Venrafia。此外，诺华还计划扩展其已获批的IgA肾病药物Fabhalta的适应症，以覆盖更多肾病类型。诺华首席医疗官Shreeram Aradhye在领英上表示，此次收购加强了公司在肾病产品线上的实力，延续了在IgA肾病和补体3肾小球病治疗领域获批后的创新势头。诺华生物医学研究总裁Fiona Marshall此前向BioPharma Dive透露，公司一直在寻找有潜力的早期项目，并将多囊肾病视为肾病产品线的重点攻克目标。她强调，虽然内部研发项目很重要，但如果市场上有更优秀的项目，诺华也会果断收购。参考资料：Novartis to acquire Regulus Therapeutics and farabursen, an investigational microRNA inhibitor to treat ADPKD, the most common genetic cause of renal failureNovartis to acquire Regulus in deal for kidney disease drug其它公开资料图片来源：微信订阅号AI38笔！596亿美元！跨国药企2025年重磅交易达成，中国公司贡献很大2025-05-02【直击现场】「星起点」宣讲会直播：50万资金+专家辅导，寻找下一个代谢病创新药突破者！2025-04-30百济神州索托克拉冲刺上市，引领BCL2抑制剂新纪元！2025-04-30版权声明/免责声明本文为原创文章。本文仅作信息交流之目的，不提供任何商用、医用、投资用建议。文中图片、视频、字体、音乐等素材或为药时代购买的授权正版作品，或来自微信公共图片库，或取自公司官网/网络，部分素材根据CC0协议使用，版权归拥有者，药时代尽力注明来源。如有任何问题，请与我们联系。衷心感谢!药时代官方网站:www.drugtimes.cn联系方式:电话:13651980212微信:27674131邮箱:contact@drugtimes.cn点击这里，查看更多精彩!

并购申请上市

2025-05-03

·药明康德

疾病控制率超90%的抗癌多肽偶联药物；诺华核药新适应症监管申请获受理…… | TIDES周报

近期，全球多肽和寡核苷酸（TIDES）领域迎来系列进展。Avacta Therapeutics公司公布其主打在研多肽偶联药物AVA6000（FAP-Dox）的1期临床试验积极数据。接受≥250 mg/m²剂量的唾液腺癌患者，疾病控制率达到91%。诺华（Novartis）的放射配体疗法（RLT）Pluvicto（177Lu vipivotide tetraxetan，镥[177Lu]特昔维匹肽注射液）的新适应症监管申请获得中国国家药监局药品审评中心（CDE）受理。此外，诺华达成合并协议收购Regulus Therapeutics。欢迎读者长按/扫描以下二维码，申请获取含有完整表格整理的《2025年5月第1期TIDES疗法进展盘点》。▲长按识别上方二维码，申请获取《2025年5月第1期TIDES疗法进展盘点》AVA6000：1期试验结果公布Avacta Therapeutics公司日前公布了其主打在研多肽偶联药物AVA6000的1期临床试验积极数据。AVA6000是一种在肿瘤微环境中特异性激活的多柔比星（doxorubicin）前药，旨在减少传统化疗的全身性副作用。在1a期剂量递增研究中，无论是每三周一次（Q3W）还是每两周一次（Q2W）的给药方案，AVA6000均显示出良好的耐受性。即使剂量升高至每三周385 mg/m²，也未达到最大耐受剂量（MTD）。在唾液腺癌患者（n=11）中，接受≥250 mg/m²剂量水平的AVA6000治疗后，多位患者获得确认缓解，疾病控制率达到91%。中位无进展生存期（PFS）尚未达到，当前中位随访时间已超过25周。Avacta Therapeutics公司目前正在1b期扩展队列中持续招募包括唾液腺癌、三阴性乳腺癌以及高级别软组织肉瘤的患者，相关数据预计将于2025年底公布。Pluvicto：新适应症监管申请获得受理日前，中国国家药监局药品审评中心官网公示，诺华的前列腺特异性膜抗原（PSMA）靶向放射配体疗法Pluvicto的新适应症监管申请获得受理，并被拟纳入优先审评，用于治疗既往接受过雄激素受体通路抑制剂（ARPI）治疗的PSMA阳性转移性去势抵抗性前列腺癌（mCRPC）成年患者。针对该项适应症，Pluvicto于今年3月获得了美国FDA批准。Pluvicto是通过静脉注射的放射配体疗法，由靶向配体与治疗性放射性核素镥结合而成。进入血液后，Pluvicto可靶向结合表达PSMA的前列腺癌细胞。结合后，放射性同位素释放的能量可破坏靶细胞，抑制其复制能力和/或引发肿瘤细胞死亡。图片来源：CDE官网今年3月，诺华宣布FDA批准Pluvicto用于接受过一种ARPI治疗且被认为适合延迟化疗的PSMA阳性的mCRPC患者。此适应症获批是基于3期PSMAfore临床研究的结果，将符合Pluvicto治疗条件的患者人群扩大约三倍。研究结果显示，与更换ARPI方案相比，Pluvicto将PSMA阳性mCRPC患者的影像学进展或死亡风险降低59%，Pluvicto组的中位影像学无进展生存期显著延长（9.3个月vs. 5.6个月，p<0.0001）。诺华达成协议收购Regulus TherapeuticsRegulus Therapeutics日前宣布，已与诺华达成一项合并协议。根据该协议，诺华将以8亿美元的初始付款收购Regulus。此外，Regulus也将在该公司的主打在研产品farabursen达成潜在监管里程碑时，获得额外9亿美元的款项，使交易总额最高可达约17亿美元。Regulus Therapeutics是一家专注于发现和开发靶向微RNA（microRNA）创新药物的生物医药公司。该公司充分发挥其在寡核苷酸药物发现和开发领域的专长，已建立了一系列在研药物管线。Regulus Therapeutics的主打在研疗法farabursen是一款靶向miR-17的新型、下一代寡核苷酸药物，用于治疗常染色体显性多囊肾病（ADPKD）。该药物近期已完成一项1b期多剂量递增临床试验。限于篇幅，本文仅针对部分重要进展做简单介绍。欢迎读者长按/扫描以下二维码，申请获取含有完整表格整理的《2025年5月第1期TIDES疗法进展盘点》。▲长按识别上方二维码，申请获取《2025年5月第1期TIDES疗法进展盘点》参考资料：[1] Avacta Therapeutics Presents Data from Lead pre|CISION® Candidate FAP-Dox (AVA6000) at the 2025 AACR Annual Meeting. Retrieved May 1, 2025 from https://www.globenewswire.com/news-release/2025/04/28/3069067/0/en/Avacta-Therapeutics-Presents-Data-from-Lead-pre-CISION-Candidate-FAP-Dox-AVA6000-at-the-2025-AACR-Annual-Meeting.html[2] 中国国家药监局药品审评中心（CDE）官网. Retrieved Apr 28，2025, From https://www.cde.org.cn/main/xxgk/listpage/4b5255eb0a84820cef4ca3e8b6bbe20c[3] FDA expands Pluvicto’s metastatic castration-resistant prostate cancer indication. Retrieved May 2, 2025 from https://www.fda.gov/drugs/resources-information-approved-drugs/fda-expands-pluvictos-metastatic-castration-resistant-prostate-cancer-indication[4] Regulus Therapeutics Enters into Agreement to be Acquired by Novartis AG. Retrieved April 30, 2025 from https://www.prnewswire.com/news-releases/regulus-therapeutics-enters-into-agreement-to-be-acquired-by-novartis-ag-302442023.html免责声明：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：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载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，聚焦全球生物医药健康创新

放射疗法临床3期优先审批临床2期临床结果

2025-05-01

·Pharmaceutical Technology

Novartis to strengthen renal disease portfolio with Regulus

The upfront cash payment represents a significant premium over Regulus’ recent stock prices. Credit: Judith Linine/Shutterstock. Novartis has agreed to acquire US-based Regulus Therapeutics and its oligonucleotide, farabursen, to bolster its renal disease offerings. The upfront cash payment represents a significant premium over Regulus’ recent stock prices and a contingent value right (CVR) tied to regulatory milestones. Total consideration for the acquisition, including the CVR, could reach $1.7bn if the regulatory milestone is met. The transaction has received unanimous approval from both companies’ boards of directors. Novartis will commence a tender offer through an indirect wholly owned subsidiary to purchase all outstanding Regulus shares. Shareholders of Regulus will receive $7 per share in cash on closure, along with the CVR worth up to an additional $7 per share, contingent upon regulatory approval of the therapy. Post completion, Novartis plans to combine the acquiring subsidiary with Regulus, making it an indirect wholly owned subsidiary of the company. GlobalData Strategic Intelligence US Tariffs are shifting - will you react or anticipate? Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis. By GlobalData Learn more about Strategic Intelligence Anticipated closure will take place in the second half of 2025, contingent on customary closing conditions, including the tender of a majority of Regulus’ common stock outstanding shares and necessary regulatory approvals. Both companies will operate as independent entitiesuntil then. Evercore is the exclusive financial advisor to Regulus, and Latham & Watkins is providing legal counsel. Regulus’ farabursen targets microRNA (miR)-17 with preferential kidney exposure and is intended for treating autosomal dominant polycystic kidney disease (ADPKD). The treatment decreases cyst growth and the size of the kidney, potentially delaying ADPKD’s severity. In March 2025, Regulus completed its Phase Ib multiple-ascending dose trial of the therapy, demonstrating safety and clinical efficacy. Novartis Development president and chief medical officer Shreeram Aradhye stated: “With limited treatment options currently available for patients suffering from ADPKD, farabursen represents a potential first-in-class medicine with a profile that may provide enhanced efficacy, tolerability and safety versus standard of care.” In February 2025, Novartis agreed to acquire Anthos Therapeutics, a Boston-based clinical-stage biopharmaceutical company, for an upfront payment of $925m.

并购临床1期寡核苷酸