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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159

项与贝派地酸相关的文献（医药）

2026-04-01·ATHEROSCLEROSIS

In silico prediction of the lipid-lowering efficacy of the oral PCSK9 inhibitor enlicitide using a mechanistic computational model

Article

作者: Mahé, Guillaume ; Bechet, Emmanuelle ; Cariou, Bertrand ; Boccara, Franck ; Angoulvant, Denis ; Wang, Yishu ; Steg, Philippe Gabriel

BACKGROUND AND AIMS:

Regulatory agencies increasingly support model-informed drug development, including quantitative systems pharmacology (QSP) models, but confidence in model-based predictions for decision making remains limited. We previously developed a mechanistic QSP model of atherosclerosis to simulate LDL-C responses to lipid-lowering therapies, which was extensively calibrated and retrospectively validated using published data from prior clinical trials. The recent completion of two phase 3 trials of the oral PCSK9 inhibitor enlicitide decanoate, CORALreef Lipids (NCT05952856) and CORALreef AddOn (NCT06450366), provided an opportunity for prospective validation, the highest standard for establishing predictive credibility.

METHODS:

The model was recalibrated using phase 1 PK/PD data and phase 2b LDL-C response data to incorporate enlicitide's effect. Predictions for both trials were generated and publicly posted, independently of the sponsor and prior to any data release.

RESULTS:

For CORALreef Lipids, the predicted week-24 LDL-C reduction was -54.5% (95% PPI: -56.1%, -53.0%), closely matching the subsequently reported -55.8% (95% CI: -60.9%, -50.7%). For CORALreef AddOn, predicted week-8 LDL-C reductions were -59.4% (95% PPI: -65.7, -53.4) with enlicitide, -18.0% (-20.1, -15.8) with bempedoic acid, -24.6% (-26.6, -22.7) with ezetimibe, and -39.8% (-42.8, -36.9) with their combination; comparison with actual trial outcomes is pending trial readout.

CONCLUSIONS:

This first successful prospective validation of our mechanistic QSP model against a large cardiovascular phase 3 trial, confirms the model's credibility. It supports the contribution of in silico trials to accelerate the development and evaluation of innovative lipid-lowering therapies to improve cardiovascular outcomes.

2026-03-01·THROMBOSIS AND HAEMOSTASIS

Strategic Lowering of LDL-Cholesterol Post Ischemic Stroke: Identifying Optimal Targets, Timing, and Therapeutic Approaches

Review

作者: Kolios, Nikolaos Gavriil ; Barkas, Fotios ; Milionis, Haralampos ; Ntaios, George

Stroke remains a leading cause of morbidity and mortality worldwide, with ischemic stroke dominating the landscape. Dyslipidemia, particularly elevated low-density lipoprotein cholesterol (LDL-C), is a critical modifiable risk factor in both primary and secondary prevention of stroke. While statins have established their role in stroke prevention, questions remain regarding optimal treatment strategies for specific patient groups, the role of non-statin therapies, and lipid level targets for maximal benefit. Emerging evidence underscores the potential of novel lipid-lowering agents, namely, PCSK9 injectable therapies targeting PCSK9, bempedoic acid, and icosapent ethyl, to complement statins in specific patient groups, including those with statin intolerance, those needing further LDL reduction, and those at high residual risk because of high triglycerides. Furthermore, the variability in stroke subtypes and patient comorbidities highlights the need for individualized, evidence-based approaches to lipid management. This narrative review provides a comprehensive overview of available lipid-lowering therapies for post-stroke patients, proposing practical algorithms for “who” to treat, “how” to treat them, and “how much” LDL-C reduction is needed. We aim to bridge gaps between clinical evidence and practice, focusing on individualized approaches to improve outcomes in this high-risk population.

2026-02-01·MOLECULAR THERAPY

Helper-dependent adenoviral vector-mediated expression of an LDL receptor/transferrin chimeric protein in muscle safely reduces atherosclerosis in LDLR-deficient mice.

Article

作者: Pagliaro, Raffaella ; Coluccino, Ludovica ; Tripodi, Lorella ; Castaldo, Giuseppe ; Pastore, Lucio ; Vitale, Maria ; Cerullo, Vincenzo ; Bianco, Andrea ; D'Agostino, Anna ; Scialò, Filippo

Familial hypercholesterolemia is a genetic disorder caused by mutations in the LDL receptor, leading to impaired uptake of low-density lipoprotein and its accumulation in arterial walls and other tissues. This accumulation results in cardiovascular disease and early mortality. Treatments, including statins, ezetimibe, and PCSK9 inhibitors, and bempedoic acid, are often insufficient in homozygous FH patients, particularly those with null mutations in the LDL receptor. To address this unmet need, we have developed two helper-dependent adenoviral vectors for the expression of the murine and human versions of a protein composed of the extracellular portion of the LDLR fused to transferrin. In both cassettes, expression is driven by the murine creatine kinase promoter to obtain high levels of expression restricted to muscle cells, to mitigate host response to the fusion protein. Both human and murine proteins restored LDL uptake in LDLR-deficient cells, correcting the phenotype in vitro. A single intramuscular administration of the HD-Ad vector induced expression of the murine fusion protein led to a twelve-month improving the lipid profile with a reduction of aortic atherosclerosis in LDLR-deficient mice. Furthermore, we observe no major systemic toxicity indicating that the present strategy may represent more effective therapy for FH patients.

213

项与贝派地酸相关的新闻（医药）

2026-02-26

·红米粒FH研究进展

【2.28国际罕见病日】纯合子家族性高胆固醇血症最全治疗手段（总结篇）

类别 · 前言 · 每年2月的最后一天，是国际罕见病日。 2026年2月28日，我们也将迎来第19个国际罕见病日，今年的全球主题为不止罕见(More than you can imagine)，意在告诉世界：罕见病群体的困境，远不止疾病本身；他们的生命，也远不止“罕见”二字。作为患者组织，我们也将在罕见病日前后组织一系列的科普宣传活动。今天我们对纯合子家族性高胆固醇血症（Homozygous Familial Hypercholesterolemia，HoFH）的现有治疗手段进行了总结，方便大家查看了解。一、传统降脂治疗（LDL-R依赖性） 01. 他汀类药物（Statins）他汀类药物序号类别内容 01 代表药物阿托伐他汀、瑞舒伐他汀、普伐他汀等 02 上市时间 1987年（洛伐他汀首个获批） 03 治疗原理竞争性抑制HMG-CoA还原酶，减少肝细胞内胆固醇合成，反馈性上调LDL-R表达，促进LDL清除 04 降脂效果 30%-60%（剂量依赖性） 05 HoFH应用基础治疗，但对LDL受体阴性患者效果有限 06 临床试验/数据EXCEL研究（8245例普通高胆固醇血症患者）：证实了他汀类药物改善血脂的疗效和安全性；HoFH患者中需大剂量（阿托伐他汀40-80mg或瑞舒伐他汀20-40mg） 07 主要副作用肝损伤（转氨酶升高>3倍约1%）、肌病、妊娠期禁用 02. 依折麦布（Ezetimibe）依折麦布序号类别内容 01 上市时间 2002 02 治疗原理抑制肠道NPC1L1转运蛋白，阻断胆固醇（饮食和胆汁）的肠道吸收，减少肝脏胆固醇储备，间接上调LDL-R 03 降脂效果单药5%-14%，与他汀联用可达18%-21% 04 HoFH应用二线治疗，与他汀联合使用 05临床试验/数据 50例HoFH患者研究：依折麦布+他汀40/80mg组较单用他汀80mg组LDL降低更显著（-21.4% vs -6.6%）；日本6例HoFH患者研究显示可减少血液净化后LDL反弹 06 主要副作用腹痛、腹泻、头痛、乏力（ALT升高1.3%）二、PCSK9抑制剂（LDL-R依赖性，需残留受体功能） 01. 依洛尤单抗（Evolocumab）伊洛尤单抗序号类别内容 01 上市时间 2015年（美国） 02 治疗原理全人源化单克隆抗体，结合循环PCSK9蛋白，阻止其与LDL-R结合，减少LDL-R降解，增加LDL清除 03 降脂效果 HoFH患者15%-32%（取决于残余LDL-R活性） 04 用法用量皮下注射，每2周140mg或每4周420mg 05临床试验/数据 TESLA B研究（1050例≥12岁HoFH患者，12周）：Evolocumab 420mg每4周 vs 安慰剂，LDL-C降低显著；TAUSSIG研究（94例）：非血液净化亚组降低23%，血液净化亚组降低19%；疗效与残余LDL-R活性密切相关（2%-30%活性者有效，<2%几乎无效） 06 主要副作用鼻咽炎、上呼吸道感染、头痛、背痛、肌痛（ALT升高3.0%） 02. 阿利西尤单抗（Alirocumab）阿利西尤单抗序号类别内容 01 上市时间 2015年（美国） 02 治疗原理全人源化单克隆抗体，结合循环PCSK9蛋白，阻止其与LDL-R结合，减少LDL-R降解，增加LDL清除 03 降脂效果 HoFH患者26% 04 用法用量皮下注射，每2周75-150mg 05临床试验/数据 ODYSSEY研究（最大规模HoFH随机对照试验）：12周时LDL-C较安慰剂降低35.6%；显著降低apoB、非HDL-C、Lp(a)；对至少2%功能性LDL-R的HoFH患者有益 06 主要副作用鼻咽炎、注射部位反应、流感、尿路感染、肌痛（过敏事件8.6% vs 安慰剂7.8%） 03. 英克司兰（Inclisiran）英克司兰序号项目内容 01 上市时间 2020年12月（欧盟首批） 02 治疗原理首个GalNAc偶联siRNA，特异性被肝细胞去唾液酸糖蛋白受体（ASGPR）摄取，进入细胞后整合入沉默复合体（RISC）复合物，切割降解PCSK9 mRNA，从源头上抑制PCSK9蛋白合成 03 降脂效果一般人群50%-52%，HoFH患者12%-37% 04 用法用量皮下注射，第1天、第90天各284mg，此后每6个月一次 05临床试验/数据 ORION-2研究（HoFH患者）：300mg剂量有效降低PCSK9和LDL-C，效果持续时间比单抗更长；ORION-5研究（56例HoFH）：未显著改善LDL-C（尽管PCSK9大幅降低），但对部分LDL-R功能亚组可能有效；ORION-13研究（13例12-18岁青少年）：330天时LDL-C降低33.3%，PCSK9降低60.2%，耐受性良好 06 主要副作用注射部位反应（红斑、疼痛、皮疹），均为轻中度且短暂（ALT升高0.6%） 07 优势半年一次给药，依从性极佳，适合青少年患者三、非他汀类降脂药（部分LDL-R依赖性） 01. 贝派地酸（Bempedoic Acid）贝派地酸序号类别内容 01 上市时间 2020年（美国、欧盟） 02 治疗原理前体药物，经肝脏ACSVL1激活为Bempedoic acid-CoA，抑制ATP-柠檬酸裂解酶（ACLY，位于HMG-CoA还原酶上游），减少胆固醇合成，反馈性上调LDL-R 03 降脂效果约10%（HoFH数据有限），总胆固醇降低16.5% 04 用法用量口服180mg/日，可与依折麦布固定复方（180mg/10mg） 05临床试验/数据 CLEAR Harmony & Wisdom试验（III期）：在他汀不耐受患者中显著降低心血管事件风险；CLEAR系列试验：常见不良反应包括上呼吸道感染、肌肉痉挛、贫血、肝酶升高 HoFH应用效果有限，主要用于他汀不耐受患者 06 主要副作用上呼吸道感染、肌肉痉挛、高尿酸血症、背痛、腹痛、贫血、肝酶升高（ALT升高1.0%）；≥60岁或联用氟喹诺酮/皮质类固醇者肌腱断裂风险增加 07 特点骨骼肌缺乏激活酶，无他汀相关肌病风险四、LDL-R非依赖性治疗（突破性疗法） 01.洛美他派（Lomitapide）洛美他派（Lomitapide）序号类别内容 01 上市时间 2012年12月（FDA），2013年7月（欧盟） 02 治疗原理口服微粒体甘油三酯转移蛋白（MTP）抑制剂，阻断肝脏和肠道内质网中apoB脂蛋白的组装，抑制VLDL和乳糜微粒形成，从源头减少LDL生成 03 降脂效果 24%-57%，中位肝脏脂肪含量10.2% 04 用法用量口服10-60mg/日，起始5mg/日，每2周递增 05临床试验/数据关键III期研究（29例成人HoFH，26周）：中位LDL-C降低50%，apoB降低49%，78周时38%患者维持疗效；扩展研究（294周）：74%患者达到LDL-C<100mg/dL，58%达到<70mg/dL；儿科研究APH-19（43例5-17岁）：24周时LDL-C平均降低53.5%，安全性良好 06 主要副作用腹泻、恶心、呕吐、腹痛、肝酶升高（ALT升高15.0%）、肝脂肪变性；需补充脂溶性维生素E（400IU/日）和必需脂肪酸 07 特点首个不依赖LDL-R的口服药，可减少或替代血液净化 02.依维苏单抗（Evinacumab）⭐ 明星药物依维苏单抗序号类别内容 01 上市时间 2021年2月（FDA加速批准，≥12岁） 2022年8月（EMA批准，≥12岁）； 2023年12月（EMA扩展至5-11岁）； 2025年1月（EMA批准≥6个月） 02 治疗原理全人源单克隆抗体，结合并抑制ANGPTL3蛋白，解除其对脂蛋白脂酶（LPL）和内皮脂酶（EL）的抑制，促进VLDL和IDL清除，减少LDL生成；完全不依赖LDL-R 03 降脂效果 47.1%（III期试验），对LDL-R阴性患者同样有效 04 用法用量静脉输注15mg/kg，每4周一次 05临床试验/数据 ELIPSE HoFH研究（III期，65例）：24周时LDL-C较安慰剂降低47.1%（安慰剂组+1.9%），包括LDLR null-null变异患者；长期研究（72周）：LDL-C持续降低45.5%；真实世界研究（7例，24个月）：显著降低总胆固醇、TG、HDL-C、非HDL-C、apoB、Lp(a)等；儿科研究（≥5岁）：疗效与成人相当；<5岁扩展研究（6例1-5岁）：LDL-C降低41.3%-77.3%；模型预测：<5岁患者疗效可能优于成人 06 主要副作用头痛、流感样症状、头晕、鼻咽炎、流涕、恶心、输液部位瘙痒（ALT升高<1.0%）；妊娠禁用（兔胎毒性） 07 特点目前唯一对LDL-R完全缺失患者有效的药物；2025年EMA批准年龄降至6个月，是HoFH治疗的重要里程碑五、非药物治疗 01.血脂净化/脂蛋白单采术（LA）血脂净化/脂蛋白单采术序号类别内容 01 应用时间 1967年（血浆置换），1975年首次有效治疗报道 02 治疗原理体外循环直接物理清除血浆中的LDL颗粒，快速降低LDL-C水平 03 适应症 LDL-C>500mg/dL（13 mmo/L）的HoFH；或>300mg/dL(8 mmol/L)且药物治疗无效；儿科：>300mg/dL(8 mmol/L)或合并ASCVD且>130mg/dL(3 mmol/L) 04 治疗方案每1-2周一次，每次2-3小时（常用Kaneka硫酸葡聚糖纤维素吸附系统） 05临床数据长期研究显示可显著改善冠状动脉粥样硬化并延长生存期；Reijman研究（2024）：儿童期启动LA者无事件生存期显著长于未治疗组 06 局限性成本高、有创、耗时、生活质量下降、需终身维持；非洲、亚洲部分地区不可及 07 联合策略与PCSK9抑制剂联用可减少反弹，降低净化频率 02.外科主动脉瓣置换术（SAVR）外科主动脉瓣置换术序号类别内容 01 适应症 HoFH患者严重主动脉瓣钙化/狭窄/反流（一项25名的加拿大HoFH队列中24%的患者需手术） 02 关键发现所有成人HoFH患者20岁前即出现早期严重主动脉钙化，与年龄相关 03 术式正中开胸体外循环，下置换机械瓣或生物瓣；无缝合瓣膜等微创技术正在发展中 03.肝移植肝移植序号类别内容 01 原理植入正常肝脏提供功能性LDL-R 02 效果 LDL-C降低可达80% 03 地位最后手段（其他治疗方式失败时） 04 局限性供体稀缺（尤其儿童需匹配小肝脏）、终身免疫抑制、显著死亡率和合并症风险、术后仍需LLT 05 数据 9例患者随访28年（平均11.7年）：多数未达正常血脂水平六、未来疗法（基因治疗）基因治疗技术原理载体/平台现状 CRISPR/Cas9基因编辑修复LDLR基因或敲除PCSK9基因 AAV载体、LNP脂质纳米颗粒、慢病毒、碳基纳米颗粒临床前/早期临床 AAV载体递送正常LDLR基因或CRISPR系统肝特异性启动子优化，衣壳工程改造长期表达潜力，但免疫原性和载荷限制 LNP-mRNA 递送Cas9 mRNA和sgRNA 可重复给药、低免疫原性已在Duchenne肌营养不良中验证 AI辅助药物设计加速靶点发现、虚拟筛选 ADMET预测机器学习/深度学习应用于HoFH新药研发七、治疗策略（总结图） HoFH治疗阶梯： ━━━━━━━━━━━━━━━━━━━━ 第一层（基础）：生活方式干预 + 他汀 + 依折麦布 ↓ 不达标第二层（强化）：加用PCSK9抑制剂（Evolocumab/Alirocumab/Inclisiran） ↓ 不达标（尤其LDL-R缺陷严重者）第三层（突破）：LDL-R非依赖性药物 • 口服：Lomitapide（MTP抑制剂） • 静脉：Evinacumab（ANGPTL3抑制剂）← 对null-null患者有效 ↓ 仍不达标第四层（有创）：血脂净化/脂蛋白单采术（LA） ↓ 终末期第五层（终极）：肝移植（最后手段） ━━━━━━━━━━━━━━━━━━━━ 关键目标：诊断后尽早（理想2岁前）启动治疗，LDL-C<55-70mg/dL（1.4-1.8 mmol/L）八、治疗方式关键里程碑(时间轴） 01.治疗方式关键里程碑(时间轴）时间轴序号年份内容 01 1967 首次血浆置换治疗HoFH 02 1987 首个他汀（洛伐他汀）获批 03 2002 依折麦布获批 042012 Lomitapide获批（首个非LDL-R依赖口服药） 05 2020 Inclisiran获批（首个PCSK9 siRNA） 06 2021 Evinacumab获批（首个ANGPTL3抑制剂，≥12岁） 07 2023 Evinacumab扩展至5-11岁；Bempedoic acid复方获批 08 2025 Evinacumab扩展至≥6个月（EMA） ▼ 往期精彩回顾 ▼ 突破壁垒：纯合子家族性高胆固醇血症的创新疗法（上篇）研究综述|突破壁垒：纯合子家族性高胆固醇血症的创新疗法（下篇）【2.28国际罕见病日】认识HoFH：一场关于“胆固醇”的罕见战争参考文献 [1]Huang SJ, Huang P, Song NN, Jia XH, Gao HL, Lu ZY, Wen XL. Breaking barriers: Innovative therapies for managing homozygous familial hypercholesterolemia. Exp Mol Pathol. 2025 Dec;144:105003. doi: 10.1016/j.yexmp.2025.105003. Epub 2025 Oct 16. PMID: 41106315 进病友群扫上方二维码红米粒微信号：HomyFH 如发现文内有误请联系我们编辑：雅雅审校：宋云排版：雅雅

临床结果

2026-02-17

·esperion.com

Esperion Reaches Settlement Agreement with Fifth ANDA Filer Not to Market Generic Version of NEXLETOL® (bempedoic acid) & NEXLIZET® (bempedoic acid & ezetimibe) Prior to April 19, 2040

ANN ARBOR, Mich., Feb. 17, 2026 (GLOBE NEWSWIRE) -- Esperion (NASDAQ: ESPR) today announced that it has entered into a settlement agreement with Alkem Laboratories Ltd. (Alkem). This agreement resolves the patent litigation brought by Esperion against Alkem in response to Alkem’s Abbreviated New Drug Applications (ANDAs) seeking approval to market generic versions of NEXLETOL and NEXLIZET prior to the expiration of the applicable patents. Pursuant to the agreement, Alkem has agreed not to market a generic version of either NEXLETOL or NEXLIZET in the United States prior to April 19, 2040, unless certain limited circumstances customarily included in these types of agreements occur. The pending patent litigation against the remaining defendants (Aurobindo Pharma Limited (along with an affiliate); MSN Pharmaceuticals Inc. (along with an affiliate); Renata Limited; and Sandoz Inc.) is ongoing, and there can be no assurance whether such ongoing patent litigation will allow a generic version of NEXLETOL and/or NEXLIZET, as applicable, to be marketed in the U.S. prior to April 19, 2040. Esperion Therapeutics Esperion Therapeutics, Inc. is a commercial-stage biopharmaceutical company dedicated to developing and delivering innovative cardiometabolic and rare/orphan disease therapies. The Company leverages deep domain expertise in ACLY biology to develop and commercialize transformative medicines for patients worldwide. Esperion currently markets two oral, once-daily, non-statin therapies for patients struggling to maintain their low-density lipoprotein cholesterol (LDL-C) levels and are at risk of cardiovascular disease. With a broad U.S. commercial infrastructure and global approvals across more than 40 countries, Esperion is well positioned to serve as a partner-of-choice for global innovators seeking U.S. market access through acquisition, in-license, co-promotion and revenue share opportunities. In tandem, the Company is advancing its leadership in ACLY biology to build a diversified pipeline of novel product candidates, including treatments for Primary Sclerosing Cholangitis and renal diseases. For more information, visit esperion.com and follow Esperion on LinkedIn and X. Forward-Looking Statements This press release contains forward-looking statements that are made pursuant to the safe harbor provisions of the federal securities laws, including statements regarding marketing strategy and commercialization and business development plans, current and planned operational expenses, expected profitability, future operations, commercial products, clinical development, including the timing, designs and plans for the CLEAR Outcomes study and its results, plans for potential future product candidates, financial condition and outlook, including expected cash runway and profitability, and other statements containing the words “anticipate,” “believe,” “estimate,” “expect,” “intend,” “may,” “plan,” “predict,” “project,” “suggest,” “target,” “potential,” “will,” “would,” “could,” “should,” “continue,” and similar expressions. Any express or implied statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Forward-looking statements involve risks and uncertainties that could cause Esperion’s actual results to differ significantly from those projected, including, without limitation, the net sales, profitability, and growth of Esperion’s commercial products, clinical activities and results, supply chain, commercial development and launch plans, business development, the outcomes and anticipated benefits of legal proceedings and settlements, and the risks detailed in Esperion’s filings with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Esperion disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law. Esperion Contact Information: Investors: Alina Venezia investorrelations@esperion.com (734) 887-3903 Media: Tiffany Aldrich corporateteam@esperion.com (616) 443-8438

专利侵权专利到期生物类似药

2026-02-12

·国际糖尿病idiabetes

循证更新，精准降脂——ESC/EAS 2025血脂管理指南更新解读及美国新版指南前瞻

点击“蓝字”关注我们刚刚过去的2025年，血脂异常管理再度成为关注焦点，这一定程度上源于欧洲心脏病学会（ESC）/欧洲动脉粥样硬化学会（EAS）于ESC 2025发布的《2019 ESC/EAS血脂异常管理指南：2025重点更新》，以及美国心脏协会（AHA）/美国心脏病学会（ACC）即将推出的相关指南。AHA/ACC指南更新的具体内容暂未公布，预计将于2026年第一季度正式发布。在此期间，欧洲指南建议、近期发表的研究证据及专家观点，为美国新版指南的潜在更新方向提供了参考。来自Medscape的一篇医学新闻，聚焦ESC/EAS 2025血脂指南重点更新及AHA/ACC新版指南前瞻，整合权威专家观点与最新循证证据，为临床医师解读了血脂管理的新趋势、新策略。新型风险评估方案 2025年8月发布的血脂异常管理指南更新版中，新增推荐包括：采用SCORE2风险评分工具、重视冠状动脉钙化评估，以及为部分高危人群设定更低的LDL-C目标值。欧美指南未来可能持续存在的一项核心差异，是心血管总体风险长期评估工具的应用。欧洲的SCORE2及SCORE2-OP风险评分已于去年替代了既往算法。美国2019版指南最初推荐使用ACC/AHA心血管风险评分工具，该工具于2023年被AHA PREVENT评分替代。PREVENT评分纳入了年轻人群、肥胖人群，以及合并肾功能不全或其他代谢心血管危险因素的患者。年轻人群风险升高临床心脏病专家接诊的确诊心血管疾病年轻患者数量不断增加，这也引发了是否应更早启动降脂治疗的讨论。美国心脏病学专家N. Shah指出，即便患者血脂检测指标处于正常范围，临床医师也应关注风险增强因素，例如脂蛋白(a)［Lp(a)］水平升高、存在冠状动脉钙化、子痫前期或早发性绝经病史、早发炎症性疾病家族史、慢性肾脏病，以及南亚裔血统等。瑞士心脏病专家、欧洲新版指南联合主席K. Koskinas表示，年轻时期小幅降低血脂水平，其获益远大于老年时期或心血管疾病确诊后大幅降脂的效果。 K. Koskinas称：“流行病学、遗传学及影像学研究均提供了确凿且不断丰富的证据，对于血脂水平升高的人群，早期治疗确实可降低远期心血管疾病发生风险。”但他同时补充，该推荐不适用于血脂水平正常且无心血管高危因素的人群。ESC/EAS指南同时建议，应从儿童及青少年时期开始，尽早养成健康的生活方式。住院治疗：干预的关键时机欧洲专家团队提出的另一项新推荐为：患者因心肌梗死或其他急性冠状动脉综合征首次住院期间，即可启动降脂治疗。 N. Shah对该方案表示支持：“明确的研究数据表明，动脉粥样硬化性心血管疾病事件发生后，启动LDL-C降低治疗的时间越早，且低LDL-C水平维持时间越长，患者预后越好。相关研究还证实，该治疗策略可降低患者死亡率。”首次住院治疗对患者而言，也是改变生活方式的重要契机。N. Shah表示：“这段时期会给患者带来极大的触动，他们会高度配合、主动学习相关知识。这是关键的干预窗口期，能帮助患者明确自身LDL-C目标，使其主动参与健康管理，为自身健康负责。” 超越LDL-C的评估维度美国心血管医学教授R. Rosenson介绍，长期以来，指南均将LDL-C作为动脉粥样硬化的替代评估指标。“但我们深知，LDL-C可能低估心血管疾病风险，尤其是合并代谢综合征特征的人群。”他表示，这类人群的危险因素包括肥胖、高甘油三酯、低高密度脂蛋白（HDL）、糖尿病前期及糖尿病。 R. Rosenson认为，LDL颗粒数量对该类人群的心血管风险预测准确性更高。“仅以LDL-C为靶点的治疗，可能无法根据患者体内LDL颗粒的实际水平，给予足够强度的干预。”目前尚不清楚美国新版指南是否会推荐LDL-C以外的血脂检测项目。 Bempedoic Acid ESC/EAS指南推荐，部分患者可使用Bempedoic Acid（贝派地酸）降低LDL-C水平。该药物主要在他汀类药物不耐受患者中开展研究，同时也可作为他汀治疗的联合用药。例如CLEAR研究证实，Bempedoic Acid可使他汀不耐受的高危患者LDL-C水平降低21%~30%，主要不良心血管事件风险降低13%~15%；随访4年发现，该药物还可使心肌梗死风险降低23%，冠状动脉血运重建风险降低19%。但R. Rosenson对此提出担忧：“多数医保方案不覆盖该药物，其价格十分昂贵。我的大部分医保患者都无法承担。”此外，Bempedoic Acid治疗会增加血尿酸升高及痛风的发生风险，因此需要加强监测与检测。重新关注Lp(a) ESC/EAS重点更新指南推荐，患者一生中至少应检测1次Lp(a)水平，纳入整体风险评估体系。 N. Shah表示：“我完全认同该推荐，但全球范围内该检测的临床落地情况并不理想。”例如，他牵头的2024年研究显示，美国近60万患者中，Lp(a)检测率不足1%。筛查率偏低的原因，可能是医师对检测结果的临床应用缺乏认知，或目前尚无获批用于治疗Lp(a)异常升高的药物。但N. Shah认为：“Lp(a)可作为风险评估指标，指导是否启动降脂治疗。” R. Rosenson建议，至少检测2次Lp(a)，以校正生物学变异或单次异常结果的误差。他同时指出，他汀类药物可能升高Lp(a) 水平，但他汀带来的风险降低获益可抵消这一影响。营养补充剂 AHA/ACC 2026版指南是否会推荐营养补充剂尚未可知，但ESC/EAS专家团队因缺乏充分的获益证据，未对此类补充剂作出推荐。 N. Shah称：“目前对营养补充剂的认知仍十分有限，我通常不建议患者使用，更倾向于推荐循证治疗方案。” 但营养补充剂的临床应用仍十分普遍。K. Koskinas表示：“希望降低血脂水平的患者，会广泛使用营养补充剂。部分补充剂或许对血脂有一定影响，但效果微乎其微。”他补充道：“此外，部分补充剂存在安全性隐患，指南中已对此进行详细阐述。” N. Shah认为，无论AHA/ACC更新指南包含哪些内容，各类共识指南的临床落地执行，始终是需要关注的现实问题。“即便推出新版指南，若无法落实其中的研究结论与推荐建议，便无法产生实际价值。这也是推动临床诊疗进步的关键。” 信源：Damian McNamara, MA.Recent Dyslipidemia Research Prompts New Guidance.Medscape Medical News.February 05, 2026 声明：本文仅供医疗卫生专业人士了解最新医药资讯参考使用，不代表本平台观点。该等信息不能以任何方式取代专业的医疗指导，也不应被视为诊疗建议，如果该信息被用于资讯以外的目的，本站及作者不承担相关责任。最新《国际糖尿病》读者专属微信交流群建好了，快快加入吧！扫描左边《国际糖尿病》小助手二维码（微信号：guojitnb），回复“国际糖尿病读者”，ta会尽快拉您入群滴！（来源：《国际循环》编辑部）

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	英国	Esperion Therapeutics, Inc.	2017-02-20
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

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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


NCT05687071 (CTgov)	临床3期	高低密度脂蛋白胆固醇血症	130	ETC-1002	艱積網積遞範鬱鹽窪糧 = 顧範艱憲繭廠窪積網製淵鹹淵觸製簾壓築衊鹽 (簾膚製鹽餘淵廠膚蓋糧, 觸鹽膚夢鑰壓醖構鏇獵 ~ 鹽醖築簾壓選築簾繭壓) 更多	-	2026-02-20
NCT02993406 (CLEAR Outcomes, Pubmed \| Am J Cardiovasc Drugs)	临床3期	高胆固醇血症	13,970	Bempedoic acid	襯鑰夢築糧簾襯艱餘製(範網顧糧窪蓋糧憲簾衊) = 鑰艱顧艱齋製壓繭遞構膚齋夢憲蓋淵願淵顧獵 (壓醖簾積夢獵襯鹹繭夢 )	积极	2025-08-20
				Placebo	襯鑰夢築糧簾襯艱餘製(範網顧糧窪蓋糧憲簾衊) = 構憲遞鏇築鹽範獵餘蓋膚齋夢憲蓋淵願淵顧獵 (壓醖簾積夢獵襯鹹繭夢 )
NCT05683340 (CTgov)	临床3期	高低密度脂蛋白胆固醇血症	96	Placebo	壓鑰壓糧製齋範鑰淵構(網築築艱艱製壓憲簾觸) = 醖獵糧製壓積艱選衊製範餘選壓遞網製醖範蓋 (顧獵顧廠網窪襯醖獵艱, 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	衊憲繭鹽構顧網顧觸糧(選顧獵夢網淵遞鑰築壓) = 膚齋膚襯網淵齋觸壓顧選齋選壓廠壓憲獵繭襯 (觸齋簾壓遞網願顧艱網, 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	醖遞壓鏇製製範網鹹蓋(衊廠積選遞衊鬱範衊憲) = 淵糧餘餘夢觸齋獵鹽顧獵襯膚齋獵壓遞淵遞醖 (襯鏇廠壓遞製獵壓夢襯 ) 达到	积极	2024-05-20
				Placebo	醖遞壓鏇製製範網鹹蓋(衊廠積選遞衊鬱範衊憲) = 範齋鑰鏇遞觸廠遞齋獵獵襯膚齋獵壓遞淵遞醖 (襯鏇廠壓遞製獵壓夢襯 ) 达到
NCT04784442 (CTgov)	临床2期	高胆固醇血症	188	Placebo	壓繭獵築淵鏇襯糧願廠(顧鹽選淵憲築鹹遞積選) = 遞餘網窪範夢壓鑰觸鏇鹽範鬱糧膚選餘憲夢網 (膚選蓋齋願構膚築獵憲, 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)	簾鏇齋遞糧膚鹽鹹範顧 = 鹹遞構繭夢襯繭鹽壓鑰夢衊簾鬱構窪願鏇醖構 (顧選窪願願鬱壓蓋醖糧, 廠膚獵鹹衊壓鬱構夢憲 ~ 艱製鹹鏇製醖衊糧構衊) 更多	-	2024-01-03
				Matching placebo tablet (Placebo Comparator)	簾鏇齋遞糧膚鹽鹹範顧 = 鑰觸遞蓋齋鏇鬱艱鹽鏇夢衊簾鬱構窪願鏇醖構 (顧選窪願願鬱壓蓋醖糧, 積糧壓選廠選蓋網憲積 ~ 齋鹹淵膚餘壓簾餘積醖) 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	製簾壓願廠顧襯鏇鏇襯(壓窪積齋遞廠顧糧膚壓) = 艱窪鹹鏇艱艱簾範壓獵網淵製顧鹹壓繭遞鏇製 (製憲膚壓鑰願範憲蓋膚 )	积极	2023-10-05
NCT02993406 (Pubmed \| JAMA)	临床3期	-	4,206	Bempedoic Acid	糧遞糧觸鹽繭廠構鹹鏇(構壓顧簾糧積壓鏇築選): 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	餘簾鬱繭製鏇襯壓壓鬱(鏇遞簾願鹹獵窪範淵簾) = 鬱衊築範鬱壓繭鹽鏇觸築選構繭鹽鏇醖選築遞 (遞遞淵顧夢艱鏇願壓構, 2.133) 更多	-	2023-04-04