Autosomal dominant polycystic kidney disease (ADPKD), the most commonly inherited kidney disease, is characterized by the development of cysts in the kidney that impair function. Of those affected, half will progress to end-stage kidney disease by age 60, requiring dialysis or kidney transplant. To date, no effective and safe therapies exist for this deadly disease. Tolvaptan (Tol), the only FDA-approved drug for treatment of ADPKD, has some benefit in slowing kidney disease progression, but Tol causes frequent urination and thirst and also injures the liver in a small number of patients. The investigators' goal, therefore, is to develop new strategies to treat ADPKD that are safe and tolerable.
The development of cysts in ADPKD patients results from two main cellular processes. The first is cell growth with an increase in the number of kidney cells that make up the outer surface of the cyst. The second is an increase in fluid secretion into the cysts that develop. The investigators have shown that an enzyme, AMP-activated protein kinase (AMPK), when activated can inhibit both of those processes. Moreover, genetic mutations that cause ADPKD may alter the energy metabolism of the cell, which in turn inhibits AMPK activity. Bempedoic acid (BA), a medication that is FDA-approved for the treatment of individuals with high cholesterol and has a good safety record, activates AMPK. In addition to activating AMPK, BA inhibits a second enzyme called ATP-citrate lyase (ACLY), which is involved in cholesterol synthesis. ACLY has received growing attention as a novel target for cancer treatment. ACLY inhibition blocks increases of cell numbers by inhibiting the lipid synthesis that is required for creation of new cell membranes. This study will test whether targeting these pathways through treatment with BA will help reverse dysfunctional metabolism in individuals with ADPKD and slow disease progression.
The investigators will test this using a phase 2 clinical trial in which 120 individuals with rapidly progressive ADPKD and an estimated glomerular filtration rate of 35 or greater will be treated with either BA or placebo (inactive look-alike pill) for two years. Participants on or off a stable dose of Tol will be included in the study. Participants will be recruited from the U. of Vermont, U. of Maryland, and Tufts University, which have active PKD clinics and are recognized by the PKD Foundation as Centers of Excellence. Through follow-up visits and lab work, the investigators will assess the safety and tolerability of BA in the participants as the primary outcomes. The secondary goals are to assess preliminary efficacy and effects of BA on quality of life in study participants. The growth of cysts results in increased volume or size of the kidneys and liver. Total and cyst volumes of the kidney and liver and visceral abdominal fat content via magnetic resonance imaging (MRI) will be measured to gauge the effectiveness of this drug. The investigators also predict that proteins and small molecules involved in regulating cell energy metabolism, inflammation, and injury, as well as proteins directly involved in AMPK and ACLY function, will be altered in ADPKD patients. Levels of these proteins and small molecules may then subsequently change with BA therapy. Exploratory, mechanistic goals of this study are to identify prognostic and predictive urinary biomarkers in study participants. Successful completion of this study would have a significant impact on individuals with ADPKD by laying the groundwork for a new treatment strategy as well as by providing a new way to help guide treatment decisions.
In summary, the goals of this phase 2 randomized, double-blind, placebo-controlled clinical trial are to test the safety, tolerability and preliminary efficacy of the drug bempedoic acid, FDA-approved to lower cholesterol, when used in ADPKD patients.

开始日期2026-02-01

申办/合作机构

University of Vermont

[+5]

CTIS2025-521086-28-00

/ Not yet recruiting临床4期IIT

Randomized clinical trial to assess the efficacy and safety of a strategy for primary prevention of cardiovascular disease in patients with inflammatory rheumatic diseases based on the use of carotid ultrasound.

开始日期2026-01-01

申办/合作机构-

NCT07255820

/ Not yet recruiting临床4期IIT

Comparative Efficacy and Safety of Dual Versus Triple Lipid-Lowering Therapies (Rosuvastatin/Ezetimibe, Bempedoic Acid/Ezetimibe, and Rosuvastatin/Ezetimibe/Bempedoic Acid ) in Type 2 Diabetes Mellitus Patients With Elevated LDL Cholesterol

This Open-label, randomized clinical trial evaluates the comparative efficacy and safety of dual versus triple lipid-lowering therapy using rosuvastatin, ezetimibe, and bempedoic acid in patients with type 2 diabetes mellitus and elevated LDL cholesterol. The study aims to determine whether adding bempedoic acid to standard dual therapy provides superior lipid control without compromising safety. The 126 participants, aged 35 - 60 years will be randomly assigned to one of three treatment groups for 12 weeks, and their lipid profiles, glycemic control, and adverse effects will be monitored.The total duration of study will be 6 months, with a 3 months individual treatment period.

开始日期2026-01-01

申办/合作机构

Bahria University

100 项与贝派地酸相关的临床结果

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100 项与贝派地酸相关的转化医学

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100 项与贝派地酸相关的专利（医药）

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205

项与贝派地酸相关的文献（医药）

2026-02-01·AMERICAN HEART JOURNAL

Evolocumab before percutaneous coronary intervention for acute myocardial infarction: Design of the AMUNDSEN trial

Article

作者: Chioccioli, Marco ; Duband, Benjamin ; Montalescot, Gilles ; Ferrari, Emile ; Esposito, Giovanni ; Cequier-Fillat, Angel ; Guedeney, Paul ; Redjimi, Nassim ; Bartus, Stanislaw ; Mach, François ; Batias-Moreau, Laure ; Bouleti, Claire ; Vicaut, Eric ; Range, Grégoire ; Silvain, Johanne ; Braik, Nassim ; Brochard, Karine ; Picchi, Andréa ; Bolognese, Leonardo ; Zeitouni, Michel ; Souteyrand, Géraud ; Jukema, J Wouter ; Thiele, Holger

RATIONALE:

PCSK9 inhibitors are currently recommended as third-line therapy for post-myocardial infarction (MI) patients, added to high-dose statin, ezetimibe, and potentially bempedoic acid when the LDL-C target of <55 mg/dL is not achieved. These guideline-driven indications result in delayed initiation of PCSK9 inhibitors, potentially missing the opportunity for benefit during the acute phase. Recent studies have demonstrated that early PCSK9 inhibition promotes anti-inflammatory effects and plaque stabilization, suggesting a potential role early in MI management.

METHODS:

The AMUNDSEN trial is a randomized, international, phase IV study using a PROBE (Prospective Randomized Open, Blinded Endpoint) design to evaluate the effects of early PCSK9 inhibition in high-risk acute MI patients undergoing percutaneous coronary intervention (PCI). A total of 2,166 patients were enrolled, including those with ST-elevation MI undergoing primary PCI and those with non-ST-elevation MI with an indication for PCI, all presenting with at least 1 high-risk clinical characteristic. Patients were randomized to receive either immediate evolocumab prior to PCI alongside standard care or standard care alone. The primary objective is to achieve both a ≥ 50% reduction in LDL-C from baseline and a target LDL-C < 55 mg/dL at 12 months. The main clinical objective is to assess the composite of all-cause death or unplanned hospitalization for a cardiovascular (CV) reason at 12 months.

CURRENT STATUS:

Enrollment and randomization are complete. Lipid and clinical follow-up are ongoing. Results from this study will clarify the lipid-lowering efficacy and clinical impact of early evolocumab initiation in the acute MI setting.

CONCLUSION:

The AMUNDSEN trial will provide critical insights into the potential benefits of early PCSK9 inhibition in high-risk MI patients undergoing PCI.

CLINICAL TRIAL REGISTRATION:

NCT (clinicaltrials.gov): 04951856 - EUCT number: 2024-518195-31-00.

2026-01-01·American Journal of Cardiovascular Drugs

Cost-Effectiveness of Bempedoic Acid in High Cardiovascular Risk Patients with Statin Intolerance: An Analysis of the CLEAR Outcomes Trial

Article

作者: Ray, Kausik K ; Lincoff, A Michael ; Gillard, Kristin K ; Nissen, Steven E ; Elsea, David ; Migliaccio-Walle, Kristen ; Bloedon, LeAnne ; Sarnes, Evelyn ; Nicholls, Stephen J

BACKGROUND:

In the CLEAR Outcomes study, 13,970 high cardiovascular risk patients with hypercholesterolemia and statin intolerance were randomized to treatment with bempedoic acid or standard of care (placebo). Bempedoic acid reduced the risk of major adverse cardiovascular events by 13%. However, the cost-effectiveness of bempedoic acid in this patient population is unknown.

METHODS:

Markov modeling estimated cost-effectiveness of bempedoic acid versus standard of care alone to reduce cardiovascular risk from a US third-party payer perspective. Baseline risk was estimated by applying individual patient characteristics from the trial to established risk equations. Treatment benefit was extrapolated over a lifetime horizon using hazard ratios for individual major adverse cardiovascular event (MACE) components from CLEAR Outcomes. Scenario analyses included on-treatment analysis, alternate bempedoic acid costs, and modeling effects of the fixed-dose combination with ezetimibe on low-density lipoprotein cholesterol (LDL-C) reduction and predicted MACE.

RESULTS:

Bempedoic acid was estimated to reduce lifetime MACE (1.58 versus 1.95 per patient) versus standard of care. At list price, bempedoic acid was associated with increased costs (+ $22,600) and improved quality-adjusted life-years (QALYs, + 0.14), resulting in an incremental cost-effectiveness ratio (ICER) of $166,830 per QALY. The on-treatment analysis resulted in an ICER of $70,279 per QALY. Reduction in bempedoic acid price by 25% resulted in lower incremental total costs and an ICER of $99,993 per QALY. Modeling the effects of the fixed-dose combination resulted in an ICER of $40,317 per QALY.

CONCLUSIONS:

Use of bempedoic acid offers improved lifetime cardiovascular (CV) risk reduction over standard of care in patients with or at high risk for CV disease (CVD) at common cost-effectiveness thresholds ($150,000 per QALY).

TRIAL REGISTRATION:

ClinicalTrials.gov identifier: NCT02993406 (CLEAR Outcomes study).

2026-01-01·Journal of Atherosclerosis and Thrombosis

High-Sensitivity C-Reactive Protein and Residual Inflammatory Risk in Coronary Artery Disease: The Pathophysiology, Prognosis, and Emerging Therapies

Review

作者: Katamine, Masahiro ; Ako, Junya ; Minami, Yoshiyasu

Inflammation plays a crucial role in the initiation, progression, and destabilization of atherosclerotic plaques and it contributes to recurrent cardiovascular events in patients with coronary artery disease (CAD). High-sensitivity C-reactive protein (hsCRP) is a well-established biomarker of systemic inflammation and it is a predictor of adverse outcomes, independent of low-density lipoprotein cholesterol (LDL-C) levels. Elevated hsCRP levels are consistently associated with higher event rates in both chronic and acute coronary syndromes, thus reflecting the residual inflammatory risk not addressed by lipid-lowering therapy or revascularization. Imaging studies have revealed that higher hsCRP levels correlate with a greater plaque burden and vulnerability. Recent trials have shown that anti-inflammatory therapies, including low-dose colchicine and interleukin-6 inhibition, can reduce this residual risk, while agents such as glucagon-like peptide-1 receptor agonists, sodium-glucose cotransporter 2 inhibitors, and bempedoic acid offer additional anti-inflammatory effects. The integration of anti-inflammatory strategies with intensive lipid management may thus provide additional cardiovascular benefits.

200

项与贝派地酸相关的新闻（医药）

2026-01-08

·Pharma信息差

【2026年1月8日全球药闻】

1. 葛兰素史克"功能性治愈"肝炎B疗法在III期试验中证明价值，为审批铺平道路葛兰素史克潜在的肝炎B"功能性治愈"药物在两项备受期待的III期研究中证明其价值，为向美国FDA提交审批申请奠定基础。 2. 安进与Disco达成6.18亿美元癌症靶点合作，聚焦新治疗靶点安进与德国生物技术公司Disco Pharmaceuticals达成价值6.18亿美元的合作协议，委托该机构针对其已识别的癌症治疗靶点开发疗法。 3. Soley Therapeutics获2亿美元C轮融资，推进AML细胞应激药物进入临床人工智能生物技术公司Soley Therapeutics获得2亿美元C轮融资，计划用于将其主导的急性髓系白血病资产推进至临床试验阶段。 4. 乐天医疗新增1亿美元融资，瞄准2028年向美国FDA提交申请乐天医疗再次完成大规模融资，获得1亿美元资金，推动其主导的光动力癌症疗法向2028年目标FDA审批申请迈进。 5. Pulmocide终止III期抗真菌试验，因反应率低和死亡率较高 Pulmocide的III期试验因接受其研究性吸入抗真菌药的患者反应率较低且死亡风险高于对照组患者而终止。 6. 和黄医药热推自身免疫竞赛，III期成功为中国申报铺路和黄医药自身免疫疾病候选药物sovleplenib的III期试验达到主要终点，为在中国提交申请注入动力。 7. 基立福授权Genesis在13个欧洲国家商业化白血病药物Vanflyta 根据协议，Genesis负责在13个欧洲国家销售第一三共2023年获批的急性髓系白血病药物Vanflyta（通用名：quizartinib）。 8. 美国FDA未标题信函批评Esperion关于降胆固醇药Nexlizet的电视广告声称美国FDA对Esperion Therapeutics降胆固醇药物Nexlizet（通用名：bempedoic acid）电视广告中"脂质潜伏者"相关声明提出质疑。 9. 生物技术并购市场2025年交易额达1140亿美元，显示行业复苏迹象尽管面临地缘政治和监管动荡，2025年生物制药行业并购活动活跃，总交易额超过1140亿美元，较2024年翻倍增长。 10. 美国海关与边境保护局将停止纸质退税，转向数字化处理随着进口商等待最高法院关于行政关税权力的裁决，美国海关与边境保护局将逐步转向数字化退税处理系统。信源整合 BioPharma Dive | MedTech Dive | Fierce Biotech | Fierce Pharma | PharmaVoice | Healthcare Dive | SupplyChain Dive | Manufacturing Dive

临床3期临床成功

2026-01-08

·医脉通心血管

EHJ综述：新兴降脂靶点及前沿药物研发 | 图文

导语：尽管当前主流降脂疗法已显著改善心血管疾病预后，但部分患者仍存在残余心血管风险，且对现有疗法应答不佳或存在特定血脂谱异常的情况亟待解决。近年来，降脂治疗领域不断涌现新型靶点，针对这些靶点的药物研发为突破治疗瓶颈提供了可能。近日，《欧洲心脏杂志》（EHJ）发表的一篇综述，系统梳理了新兴降脂靶点及相关疗法。摘要图注：当前临床常用及新兴的降脂疗法，均通过多种作用机制靶向调控脂质与脂蛋白代谢通路中的关键靶点。他汀类药物与Bempedoic acid分别作用于胆固醇生物合成通路的不同环节，依折麦布抑制胆固醇吸收。洛美他派（Lomitapide）抑制微粒体甘油三酯转移蛋白（MTP），阻碍极低密度脂蛋白（VLDL）的生物合成。PCSK9抑制剂包括单克隆抗体类、小干扰RNA（siRNA）类，以及正在研发的Adnectin重组融合蛋白、大环肽、小分子化合物及CRISPR碱基编辑疗法。此外，针对载脂蛋白(a)/Lp (a)、血管生成素样蛋白3（ANGPTL3）、载脂蛋白C-III（APOC3）和胆固醇酯转移蛋白（CETP）等其他新型治疗靶点的单克隆抗体、反义寡核苷酸（ASO）、siRNA、小分子载脂蛋白(a)和基因沉默方法，目前正处于不同的研发阶段。图1 新型降脂疗法的细胞和分子靶点注：新型降脂疗法通过干扰特定的分子途径发挥作用：（i）PCSK9抑制剂，如Lerodalcibep、Enlicitide decanoate、AZD07080及基因编辑；（ii）CETP抑制剂，如Obicetrapib；（iii）ANGPTL3抑制剂，如Evinacumab（单抗）、Zodasiran（siRNA）、Vupanorsen（ASO，已停止研发）以及VERVE 201（基因编辑）。（iv）ApoC III抑制剂，如Olezarsen、普乐司兰（Plozasiran）；（v）载脂蛋白(a)靶向药物，包括基因沉默疗法和口服分子Muvalaplin，其中前者包括ASO类的Pelacarsen和siRNA类的Olpasiran、Zerlasiran和Lepodisiran。 01 血管生成素样蛋白3 血管生成素样蛋白3（ANGPTL3）仅在肝脏产生，在脂质代谢中发挥重要作用。ANGPTL3分泌到血液后，会抑制脂蛋白脂肪酶（LPL），从而减少富含甘油三酯脂蛋白的清除，升高血浆甘油三酯水平。研究者发现，抑制ANGPTL3可能是降低LDL-C水平和心血管风险的有效疗法。ANGPTL3抑制后观察到的LDL-C降低不依赖于LDLR活性，这对于可能几乎没有或完全没有残余LDLR功能的HoFH患者尤为重要。 Evinacumab是一种靶向循环ANGPTL3的单克隆抗体，可使HoFH患者的LDL-C水平降低50%，而与LDLR功能程度无关，其降脂效果可维持长达2.5年。 Zodasiran（ARO-ANG3）是一种靶向肝脏ANGPTL3 mRNA的GalNAc偶联小干扰RNA（siRNA）。在混合性血脂异常成人患者中，Zodasiran治疗24周可显著降低甘油三酯水平。 Solbinsiran（LY3561774）是另一种靶向ANGPTL3的GalNAc偶联siRNA，在混合性血脂异常患者的2期剂量递增研究中，中剂量组可使apoB降低14%（主要终点）、甘油三酯降低50%、LDL-C降低17%。尽管ANGPTL3的降低呈剂量依赖性（54%-77%），但低剂量和高剂量组的apoB降低无统计学意义。 02 载脂蛋白C-III 载脂蛋白C-III（apoC-III）通过抑制脂蛋白脂肪酶（LPL，负责水解血液中的甘油三酯）活性，在脂质代谢中发挥关键作用。apoC-III水平升高与高甘油三酯血症相关，这是由于富含甘油三酯脂蛋白的清除受损。旨在降低apoC-III水平的药物（包括两种ASO和一种siRNA）正在研发中，用于治疗高甘油三酯血症、预防动脉粥样硬化并降低急性胰腺炎风险，重点关注家族性乳糜微粒血症综合征（FCS）患者或高甘油三酯血症患者（表1）。表1 富含甘油三酯脂蛋白靶向治疗 Volanesorsen是第二代ASO，特异性靶向APOC3 mRNA。在高甘油三酯血症患者中，Volanesorsen单药治疗或与稳定的贝特类药物联合治疗时，可使apoC-III降低达80%，甘油三酯降低达71%，且对FCS患者也有效。Volanesorsen治疗与血小板减少症风险增加相关，因此需要探索肝细胞特异性更高的其他疗法。 Olezarsen是一种GalNAc偶联ASO，可选择性靶向肝脏apoC-III的合成。其核酸序列与Volanesorsen相同，但GalNAc部分的存在使其给药频率更低、剂量显著降低（50 mg或80 mg），且作用持续时间更长（每4周注射一次）。在中度高甘油三酯血症且有高心血管疾病风险或已确诊心血管疾病的患者中，Olezarsen可显著降低甘油三酯水平和致动脉粥样硬化脂蛋白水平。在FCS患者中，Olezarsen可显著降低apoC-III（81%）和甘油三酯（59%）水平；治疗53周时，安慰剂组发生11次急性胰腺炎，而Olezarsen各剂量组均仅发生1次。Olezarsen已获得美国食品药品监督管理局（FDA）批准，用于降低FCS患者的甘油三酯水平，并被欧洲药品管理局（EMA）指定为FCS治疗的孤儿药。Olezarsen的3期临床试验正在进行中，以进一步评估其安全性和有效性，涉及的终点包括肝脏脂肪含量变化、血浆甘油三酯水平变化以及冠状动脉斑块进展情况。 Plozasiran是一种GalNAc偶联siRNA，可抑制肝脏apoC-III的产生。在混合性血脂异常患者中，与安慰剂相比，第1天和第12周皮下注射Plozasiran 10 mg、25 mg或50 mg（每季度一次），甘油三酯水平显著降低50%-62%；第1天和第24周注射Plozasiran 50 mg（每半年一次），甘油三酯显著降低44%（MUIR；2期）。Plozasiran 25 mg剂量已用于正在进行的3期研究。Plozasiran对重度高甘油三酯血症也有效，90%的患者甘油三酯降低至< 500 mg/dL（这被认为是急性胰腺炎风险的阈值）（SHASTA-2；2期）；且在FCS患者中，Plozasiran可显著降低甘油三酯水平和胰腺炎发生率（PALISADE；3期）。核磁共振分析显示，Plozasiran可使富含甘油三酯脂蛋白的数量减少约50%，并使LDL分布向更大、致动脉粥样硬化可能性更低的颗粒转移。 03 成纤维细胞生长因子21 成纤维细胞生长因子21（FGF21）主要由肝脏分泌，在脂肪组织、骨骼肌和胰腺中表达。FGF21调节脂质和葡萄糖代谢以及能量消耗，但其半衰期较短，限制了其作为药物靶点的潜在应用。 Pegozafermin是一种FGF21类似物，通过位点特异性糖基转移酶进行聚乙二醇化（PEG化），以延长其活性并减缓其分解代谢，目前正在研发用于治疗高甘油三酯血症和非酒精性脂肪性肝炎（NASH）。在重度高甘油三酯血症患者中，Pegozafermin总体可使甘油三酯降低44%，并改善NASH患者的纤维化程度。一项针对重度高甘油三酯血症患者的Pegozafermin 3期试验正在进行中。 04 胆固醇酯转移蛋白胆固醇酯转移蛋白（CETP）促进甘油三酯从VLDL和LDL转移至HDL，同时交换胆固醇酯。遗传研究表明，CETP活性降低可改善脂蛋白谱，并降低心血管事件发生率。这些发现促使科学家研究CETP抑制对血浆脂质和心血管疾病结局的影响，最初的研究重点是升高HDL-C水平。更强效的CETP抑制剂还可降低血浆LDL-C和apoB水平；尽管Anacetrapib对非HDL-C的降低作用不大，但这也解释了在REVEAL试验中其主要不良心血管事件发生率更低的原因。由于Anacetrapib半衰期较长，且会在脂肪组织中蓄积，其开发后来被终止，但REVEAL试验的观察结果为进一步研究CETP作为降低血浆LDL-C水平和改善心血管疾病结局的潜在靶点奠定了基础，随着随访时间延长观察到心血管事件减少幅度更大。迄今为止，Obicetrapib是临床研发中最强效的CETP抑制剂（表2）。在尽管使用了高强度他汀但LDL-C仍≥70 mg/dL的患者中，加用Obicetrapib可使LDL-C降低50%，并降低apoB、非HDL-C和Lp (a) 水平；Obicetrapib联合依折麦布可使LDL-C降低63%。研究显示，HeFH和/或ASCVD患者接受Obicetrapib治疗可进一步降低LDL-C水平。Obicetrapib的潜在获益似乎与apoB和LDL-C降低直接相关，这表明CETP抑制可降低不同类型的致动脉粥样硬化脂蛋白。表2 研发中的CETP靶向治疗 05 载脂蛋白(a) Lp(a) 是一种LDL样颗粒，其中apoB与载脂蛋白(a) [apo (a)] 共价结合。Lp(a) 水平升高是心血管疾病（包括主动脉瓣狭窄、心肌梗死和卒中）的风险因素。遗传和流行病学数据表明，除了控制其他心血管风险因素外，靶向Lp(a) 可能有助于降低心血管疾病风险，尤其在极高危患者中。目前的降脂疗法对Lp(a) 水平无影响或仅能降低20%-25%。现有数据表明，迄今为止最强效的CETP抑制剂Obicetrapib可使Lp(a)水平降低达56%，甲状腺激素受体β选择性激动剂Resmetirom可使Lp(a)降低约33%。然而，这样的降低幅度似乎不足以降低Lp(a)的致动脉粥样硬化潜力。事实上，据估计，Lp(a)水平降低> 80% 才具有临床有效性，这表明需要更强效的治疗方法。尽管目前尚获批的无专门用于降低血浆Lp(a)水平的药物，但已有多种疗法处于临床研发阶段，包括：（i）反义寡核苷酸；（ii）小干扰RNA；（iii）Lp(a)翻译后组装的特异性抑制剂；（iv）通过CRISPR-Cas9技术进行apo(a)基因编辑（表3）。表3 脂蛋白（a）靶向治疗 Pelacarsen是一种靶向apo(a)的ASO。在血浆Lp(a)水平升高的心血管疾病患者中进行的临床试验显示，Pelacarsen降低Lp(a)达66%-92%。HORIZON是一项3期心血管结局试验，纳入Lp(a)≥70 mg/dL的心血管疾病患者，随访4年，旨在评估每月皮下注射Pelacarsen 80mg对患者主要不良心血管事件风险的影响。目前至少有三种靶向apo(a) mRNA的siRNA处于研发阶段：Olpasiran、Zerlasiran和Lepodisiran。这些分子均采用GalNAc化学修饰以选择性靶向肝脏，但药代动力学特征不同。Olpasiran可使Lp(a)降低71%-98%，一项针对Lp(a)水平极高（≥200 nmol/L）患者的心血管结局试验OCEAN (a)-Outcomes-TIMI 75正在进行中。Zerlasiran和Lepodisiran可在短期内使Lp(a)降低超90%，且长期平均降低幅度超80%。Lepodisiran的3期心血管结局研究ACCLAIM-Lp(a)正在招募参与者。除了ASO和siRNA外，口服药物Muvalaplin通过阻断apo(a)与apoB结合来降低Lp(a)水平。研究表明，Muvalaplin可显著降低Lp(a)达50%-86%。尽管这些方法几乎可以完全清除循环中的Lp(a)，但在考虑将降低升高的Lp(a)水平纳入心血管风险降低策略之前，关键是要明确这种降低幅度对心血管保护的转化程度。在等待心血管结局试验结果的同时，监测长期血浆Lp(a)水平极低是否会导致葡萄糖代谢变化也至关重要，因为遗传性血浆Lp(a)水平极低的个体患糖尿病风险增加。文献索引：Christie M Ballantyne , Giuseppe D Norata. The evolving landscape of targets for lipid lowering: from molecular mechanisms to translational implications. European Heart Journal, 2025: 46(44): 4737–4750. 上篇推荐 EHJ综述：当前主流降脂治疗靶点和药物全景 | 图文医脉通是专业的在线医生平台，“感知世界医学脉搏，助力中国临床决策”是平台的使命。医脉通旗下拥有「临床指南」「用药参考」「医学文献王」「医知源」「e研通」「e脉播」等系列产品，全面满足医学工作者临床决策、获取新知及提升科研效率等方面的需求。

siRNA寡核苷酸核酸药物临床结果临床2期

2026-01-08

·BioSpace

Organon Enters into a Commercialization Agreement for Daiichi Sankyo’s Nilemdo® in France, Denmark, Iceland, Sweden, Finland and Norway

The agreement builds on Organon’s cardiovascular disease portfolio, leveraging expertise to expand access to new treatments for patients with dyslipidemia, especially statin-intolerant patients. This collaboration addresses a critical gap in care that disproportionately affects women, who are at a 47% higher risk of developing statin intolerance compared to men. i JERSEY CITY, N.J.--(BUSINESS WIRE)--Organon today announced that it has entered into an agreement with Daiichi Sankyo Europe to commercialize Nilemdo ® (bempedoic acid) in France, Denmark, Iceland, Sweden, Finland and Norway. Nilemdo ® is a new, first-in-class drug indicated for patients with high cholesterol and cardiovascular disease risk. It provides an alternative treatment for patients that cannot be treated effectively with statins. ii “This collaboration combines Organon’s commercial agility with Daiichi Sankyo’s expertise in cardiovascular innovation to bring Nilemdo ® to patients in France, Denmark, Iceland, Sweden, Finland and Norway,” says Thibault Crosnier Leconte, AVP & Managing Director at Organon Northwest Europe. “By offering a new treatment option for patients who cannot tolerate statins, we are helping to close a persistent gap in cardiovascular care—one that disproportionately affects women – whilst reinforcing our mission to deliver impactful treatments for a healthier every day.” Cardiovascular disease is the leading cause of death in Europe iii and for women worldwide, iv however it remains understudied, under-recognized, under-diagnosed and under-treated. v Nilemdo ® is the first and only treatment in its class available in these markets and provides healthcare professionals with a new therapy to reduce cardiovascular risk in patients unable to achieve adequate LDL-C reduction with statins or other lipid-lowering therapies, or in patients who are statin-intolerant or for whom statins are contraindicated. Under the terms of the agreement, Organon will distribute and promote Nilemdo ® in France, Denmark, Iceland, Sweden, Finland and Norway. Daiichi Sankyo Europe will remain the marketing authorization holder for the product and Organon will be local representative in the territory. About Nilemdo ® Nilemdo ® is a lipid-lowering drug containing bempedoic acid, which inhibits ATP-citrate lyase, a key enzyme in the cholesterol biosynthesis pathway. It was approved by the European Medicines Agency (EMA) in February 2020. Indications and use in the EU: Hypercholesterolaemia and mixed dyslipidaemia Nilemdo ® is indicated in adults with primary hypercholesterolaemia (heterozygous familial and non-familial) or mixed dyslipidaemia, as an adjunct to diet: in combination with a statin or statin with other lipid-lowering therapies in patients unable to reach low-density lipoprotein cholesterol (LDL-C) goals with the maximum tolerated dose of a statin or, alone or in combination with other lipid-lowering therapies in patients who are statin-intolerant, or for whom a statin is contraindicated. Cardiovascular disease Nilemdo ® is indicated in adults with established or at high risk for atherosclerotic cardiovascular disease to reduce cardiovascular risk by lowering LDL-C levels, as an adjunct to correction of other risk factors: in patients on a maximum tolerated dose of a statin with or without ezetimibe or, alone or in combination with ezetimibe in patients who are statin-intolerant, or for whom a statin is contraindicated. The recommended dose is one 180 mg tablet taken orally once daily. Nilemdo ® can be taken with or without food. If a dose is missed, the patient should take the tablet immediately and resume the usual schedule the next day. Nilemdo ® is intended for oral use only. Complete dosage and administration instructions are provided in the medication leaflet which can be found here: Nilemdo, INN-bempedoic acid . Please consult with your healthcare professional. Patients should be monitored regularly to assess the effectiveness and safety of the treatment, with adjustments made as needed based on individual response and tolerance. About Organon Organon (NYSE: OGN) is a global healthcare company with a mission to deliver impactful medicines and solutions for a healthier every day. With a portfolio of over 70 products across Women’s Health and General Medicines, which includes biosimilars, Organon focuses on addressing health needs that uniquely, disproportionately or differently affect women, while expanding access to essential treatments in over 140 markets. Headquartered in Jersey City, New Jersey, Organon is committed to advancing access, affordability, and innovation in healthcare. Learn more at and follow us on LinkedIn , Instagram , X , YouTube , TikTok and Facebook . Cautionary Note Regarding Forward-Looking Statements Except for historical information, this press release includes “forward-looking statements” within the meaning of the safe harbor provisions of the US Private Securities Litigation Reform Act of 1995, including, but not limited to, statements about Organon’s expectations about regarding its commercialization agreement for Daiichi Sankyo’s Nilemdo® in France, Denmark, Iceland, Sweden, Finland and Norway. Forward-looking statements may be identified by words such as “potential,” “mission,” “expects,” “will,” or words of similar meaning. These statements are based upon the current beliefs and expectations of Organon’s management and are subject to significant risks and uncertainties. If underlying assumptions prove inaccurate, or risks or uncertainties materialize, actual results may differ materially from those set forth in the forward-looking statements. Factors that could cause results to differ materially from those described in the forward-looking statements can be found in Organon’s filings with the SEC, including Organon’s most recent Annual Report on Form 10-K (as amended), Quarterly Reports on Form 10-Q (as amended), Current Reports on Form 8-K, and other SEC filings, available at the SEC’s Internet site ( ). Organon undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events or otherwise. _____________________________ i Bytyçi I, Penson PE, Mikhailidis DP, et al. Prevalence of statin intolerance: a meta-analysis. Eur Heart J. 2022;43(34):3213-3223. doi:10.1093/eurheartj/ehac015 ii European Medicines Agency, Nilemdo (bempedoic acid) Summary of product characteristics, available here: Nilemdo, INN-bempedoic acid [last accessed: January 2026] iii WHO, Cardiovascular diseases, available here: Cardiovascular diseases EURO [last accessed: December 2025] iv European Society of Cardiology, Cardiovascular disease in women, available here: Cardiovascular Disease in Women [last accessed: December 2025] v Vogel B, Acevedo M, Appelman Y, et al. The Lancet women and cardiovascular disease Commission: reducing the global burden by 2030. Lancet . 2021;397(10292):2385-2438. doi:10.1016/S0140-6736(21)00684-X Contacts Media: Karissa Peer (614) 314-8094 Felicia Bisaro (646) 703-1807 Investor: Jennifer Halchak (201) 275-2711

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100 项与贝派地酸相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D10691	贝派地酸	C10AX15	DB11936

研发状态

批准上市

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
高胆固醇血症	日本	大塚製薬株式会社	2025-09-19
心血管疾病	美国	Esperion Therapeutics, Inc.	2024-03-22
心肌梗塞	美国	Esperion Therapeutics, Inc.	2024-03-22
原发性高脂血症	美国	Esperion Therapeutics, Inc.	2024-03-22
血脂障碍	欧盟	Daiichi Sankyo Europe GmbH	2020-04-01
血脂障碍	冰岛	Daiichi Sankyo Europe GmbH	2020-04-01
血脂障碍	列支敦士登	Daiichi Sankyo Europe GmbH	2020-04-01
血脂障碍	挪威	Daiichi Sankyo Europe GmbH	2020-04-01
原发性高胆固醇血症	欧盟	Daiichi Sankyo Europe GmbH	2020-04-01
原发性高胆固醇血症	冰岛	Daiichi Sankyo Europe GmbH	2020-04-01
原发性高胆固醇血症	列支敦士登	Daiichi Sankyo Europe GmbH	2020-04-01
原发性高胆固醇血症	挪威	Daiichi Sankyo Europe GmbH	2020-04-01
动脉粥样硬化	美国	Esperion Therapeutics, Inc.	2020-02-21
杂合子家族性高胆固醇血症	美国	Esperion Therapeutics, Inc.	2020-02-21

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适应症	最高研发状态	国家/地区	公司	日期
高低密度脂蛋白胆固醇血症	临床3期	日本	Otsuka Pharmaceutical Co., Ltd.	2023-02-13
高脂血症	临床3期	美国	Esperion Therapeutics, Inc.	2017-10-23
脑血管疾病	临床3期	美国	Esperion Therapeutics, Inc.	2016-12-22
脑血管疾病	临床3期	阿根廷	Esperion Therapeutics, Inc.	2016-12-22
脑血管疾病	临床3期	澳大利亚	Esperion Therapeutics, Inc.	2016-12-22
脑血管疾病	临床3期	奥地利	Esperion Therapeutics, Inc.	2016-12-22
脑血管疾病	临床3期	比利时	Esperion Therapeutics, Inc.	2016-12-22
脑血管疾病	临床3期	巴西	Esperion Therapeutics, Inc.	2016-12-22
脑血管疾病	临床3期	保加利亚	Esperion Therapeutics, Inc.	2016-12-22
脑血管疾病	临床3期	加拿大	Esperion Therapeutics, Inc.	2016-12-22

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临床结果

适应症

分期

评价

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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


NCT02993406 (CLEAR Outcomes, Pubmed \| Am J Cardiovasc Drugs)	临床3期	高胆固醇血症	13,970	Bempedoic acid	襯鑰齋鬱壓獵齋鏇遞積(選蓋衊積淵齋艱糧夢蓋) = 蓋餘齋鑰鏇餘築襯簾憲積膚鏇網鏇鹽積簾獵壓 (淵製網襯淵範獵醖鑰壓 )	积极	2025-08-20
				Placebo	襯鑰齋鬱壓獵齋鏇遞積(選蓋衊積淵齋艱糧夢蓋) = 鏇襯衊窪鬱膚觸鏇網簾積膚鏇網鏇鹽積簾獵壓 (淵製網襯淵範獵醖鑰壓 )
NCT05683340 (CTgov)	临床3期	高低密度脂蛋白胆固醇血症	96	Placebo	憲衊繭簾壓繭築簾夢糧(獵廠獵夢鑰廠淵鑰鹹獵) = 艱糧構憲憲蓋獵艱糧鑰構築鹽膚簾膚築鑰願網 (窪鏇鏇製醖鑰壓鏇選鏇, 1.901) 更多	-	2025-03-13
NCT02993406 (CLEAR Outcomes, Pubmed \| J Am Heart Assoc)	临床3期	肥胖	6,177	Bempedoic Acid 180 mg	顧壓齋窪淵壓鬱選蓋遞(積鏇築網糧獵鹹憲艱醖) = 網遞範積獵簾齋襯網鹽範醖餘壓簾願網窪蓋繭 (範鏇獵願壓艱構築網淵, 0.76 [0.63 ~ 0.92]) 更多	积极	2025-02-18
				Placebo	-
NCT05683340 (Company_Website) 人工标引	临床3期	高胆固醇血症	96	Bempedoic acid 180 mg	醖積廠選簾衊憲遞襯廠(襯鬱醖襯廠鑰糧遞艱淵) = 鬱簾網窪壓觸顧膚衊鑰襯膚獵糧築製鹽顧糧簾 (鬱築網獵製憲製製遞鏇 ) 达到	积极	2024-05-20
				Placebo	醖積廠選簾衊憲遞襯廠(襯鬱醖襯廠鑰糧遞艱淵) = 鏇簾製鹽窪積範觸餘糧襯膚獵糧築製鹽顧糧簾 (鬱築網獵製憲製製遞鏇 ) 达到
NCT04784442 (CTgov)	临床2期	高胆固醇血症	188	Placebo	鹽齋築鹹廠蓋壓鑰淵簾(壓齋製願夢齋鹽觸構餘) = 淵淵餘願願壓醖糧獵獵獵糧築範廠製鬱餘鏇蓋 (鹽範觸遞夢鏇鬱簾齋糧, 2.117) 更多	-	2024-03-04
NCT02993406 (CLEAR Outcomes, CTgov)	临床3期	心血管疾病 \| 冠心病 \| 脑血管疾病 ... 更多	13,970	Bempedoic acid 180 mg tablet (Bempedoic Acid 180 mg)	網壓鑰網網鹹選糧餘觸 = 鹽鏇齋繭選築選積鑰願窪遞選淵鏇醖廠選齋襯 (糧艱遞憲餘餘製選獵蓋, 蓋鏇醖夢膚衊膚鬱糧艱 ~ 壓鏇餘獵齋鹹醖憲壓製) 更多	-	2024-01-03
				Matching placebo tablet (Placebo Comparator)	網壓鑰網網鹹選糧餘觸 = 繭鏇淵淵鹽積齋窪鏇膚窪遞選淵鏇醖廠選齋襯 (糧艱遞憲餘餘製選獵蓋, 範鑰遞觸糧構衊選壓鹹 ~ 襯構鹹鏇壓願艱繭範壓) 更多
NCT02993406 (CLEAR Outcomes, ENDO2023)	临床3期	心血管疾病 \| 冠心病 \| 脑血管疾病 ... 更多	-	Bempedoic Acid	艱窪夢憲願繭壓齋艱觸(艱鏇鏇膚鬱鏇構淵糧餘) = 鏇淵製網鏇糧膚蓋糧繭衊構鏇鹹膚鏇淵艱鑰範 (遞鹹築製廠膚齋鹽鹹網 )	积极	2023-10-05
NCT02993406 (Pubmed \| JAMA)	临床3期	-	4,206	Bempedoic Acid	餘餘鹽艱網構衊觸築齋(製選廠願艱觸夢醖獵衊): HR = 0.61 (95% CI, 0.39 ~ 0.98) 更多	积极	2023-06-24
				Placebo
NCT02178098 (CTgov)	临床2期	高血压 \| 高胆固醇血症	143	Placebo	襯壓襯壓窪淵餘糧積餘(遞遞繭鏇襯築醖糧衊願) = 窪襯窪簾艱構襯廠憲鑰願襯選願觸構餘遞網壓 (製鑰繭膚鑰鹹鑰窪遞餘, 2.133) 更多	-	2023-04-04
NCT02993406 (CLEAR Outcomes, Pubmed \| N Engl J Med)	临床3期	-	14,014	Bempedoic acid	膚鏇網獵範鏇網遞艱餘(積襯壓鬱鹽觸糧願夢衊) = 2.2% vs. 1.2% 獵廠獵築獵蓋構鏇選餘 (顧願衊鑰觸夢夢遞廠鹹 ) 更多	积极	2023-03-04
				Placebo