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2026-09-21

申办/合作机构

University of Vermont

[+5]

NCT07474649

/ Not yet recruiting临床3期

Effects of Bempedoic Acid/Ezetimibe/High-intensity Statin on Plaque Regression and Stabilisation of Coronary Atherosclerosis Among Patients Without Cardiovascular Events

The overall objective of the trial is to evaluate the effect of the triple therapy consisting of bempedoic acid (BA), ezetimibe (EZE), and high-intensity atorvastatin or rosuvastatin on changes in coronary plaque burden and plaque morphology in patients with coronary atherosclerosis without significant obstructive coronary artery disease and without prior history of an ischemic vascular event.

开始日期2026-08-15

申办/合作机构

Daiichi Sankyo Co., Ltd.

NCT07623915

/ Not yet recruiting临床3期

A Multicenter, Randomized, Double-blind, Placebo-controlled Phase III Clinical Study to Evaluate the Efficacy and Safety of Bempedoic Acid in Patients With Hyperlipidemia Not Adequately Controlled by Statins

This trial is a multicenter randomized, double-blind, placebo-controlled phase III study to evaluate the efficacy and safety of Bempedoic Acid Tablets in patients with hyperlipidemia not adequately controlled by Statins.

开始日期2026-06-12

申办/合作机构

石药集团欧意药业有限公司

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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362

项与贝派地酸相关的文献（医药）

2026-12-31·RENAL FAILURE

Emerging roles of ATP citrate lyase in kidney diseases: from pathogenic driver to therapeutic target

Review

作者: Chen, Yinyin ; Yang, Yiya ; Tao, Mina ; Wei, Mengjiao ; Tan, Huifang ; Li, Guoli ; Jin, Zhiyuan ; Xiao, Zheng

ATP-citrate lyase (ACLY) is a crucial cytosolic and nuclear enzyme that catalyzes the synthesis of acetyl-CoA from citrate, serving as a central metabolic node linking carbohydrate metabolism to de novo lipogenesis and histone acetylation. Accumulating evidence positions ACLY not merely as a metabolic enzyme but as a key pathogenic driver in various kidney diseases. This review systematically examines the multifaceted roles of ACLY in renal physiology and pathology. During development, ACLY is essential for nephron progenitor cell maintenance and nephrogenesis. In diabetic kidney disease, hyperglycemia upregulates and promotes nuclear translocation of ACLY, fueling histone acetylation and the transcription of pro-fibrotic genes, while also contributing to oxidative stress and lipid peroxidation. In obesity-related kidney injury, ACLY drives renal ectopic lipid accumulation and inflammation by providing substrates for adipogenic enzymes and inducing histone hyperacetylation. ACLY also promotes renal fibrosis in chronic kidney disease via pathways such as AKT/ACLY signaling. In clear cell renal cell carcinoma, ACLY expression is upregulated through HIF-2α/LPCAT1/FBXW7 and VHL/PPARγ axes, promoting lipid synthesis, tumor proliferation, and metastasis. Furthermore, ACLY activity influences hypocitraturia, a key factor in nephrolithiasis, and is upregulated in polycystic kidney disease, where its inhibition attenuates cystic growth. Given its central role across diverse renal pathologies, ACLY emerges as a promising therapeutic target. Several inhibitors, including bempedoic acid, SB-204990, and natural compounds, show potential in modulating ACLY activity. This review consolidates current knowledge on ACLY in kidney diseases, highlighting its mechanistic contributions, and underscoring its significant potential for diagnostic and therapeutic innovation.

2026-09-01·Atherosclerosis plus

Systematic literature review on the real-world effectiveness and safety of bempedoic acid

Review

作者: Gautam, Raju ; Chhabra, Richa ; Anwar, Saeed ; Ghatak, Aishee ; Cannon, Christopher P ; Powell, Heather A ; Sarnes, Evelyn ; Cicero, Arrigo Francesco Giuseppe ; Jawla, Shantanu

Background and aims:

Cardiovascular disease remains a significant global health burden. Bempedoic acid is a novel, oral adenosine triphosphate-citrate lyase inhibitor that lowers low-density lipoprotein cholesterol (LDL-C). While randomized controlled trials (RCTs) have established its efficacy and safety, evidence from real-world clinical practice is needed.

Methods:

A systematic literature review (SLR) was conducted to identify studies reporting real-world use of bempedoic acid as monotherapy, in fixed dose combination with ezetimibe, or in combination with any other lipid lowering therapy (LLT). MEDLINE, EMBASE, and Cochrane Library were searched from inception to February 19, 2025. Two reviewers independently screened studies, with discrepancies resolved by a third. Data extraction and validation were conducted, and the risk of bias assessed using the Newcastle-Ottawa Scale.

Results:

Of 269 records, 28 reports on 22 unique studies met the eligibility criteria. Most studies evaluated LDL-C changes from baseline after initiation of bempedoic acid; one pragmatic randomized trial was identified. Bempedoic acid achieved LDL-C reductions of 15% to 39% when added to maximally tolerated statins, 22% to 38% in statin intolerant cohorts, and 18% to 42% in mixed statin intolerant cohorts. Safety data was limited; treatment discontinuation ranged from 9% to 36%, with higher rates in cohorts with greater statin-intolerance.

Discussion:

This SLR shows bempedoic acid, with or without other LLTs, achieves a minimum of approximately 20% LDL-C reductions, consistent with RCTs. Data suggests it may be effective as an adjunct to statins and as an oral alternative for statin-intolerant patients.

2026-07-07·Journal of the American Heart Association

Efficacy and Safety of Bempedoic Acid in Patients Aged ≥75 Years Stratified by Varying Statin Exposure: Results From Phase 3 Studies of Bempedoic Acid

Article

作者: Mukherjee, Debabrata ; Nicholls, Stephen J. ; Leiter, Lawrence A. ; Catapano, Alberico L. ; Duell, P. Barton ; Laufs, Ulrich ; Nissen, Steven E. ; Bloedon, LeAnne ; Powell, Heather A. ; Lincoff, A. Michael ; Broestl, Christine ; Li, Na ; Goldberg, Anne C. ; Mancini, G. B. John ; Herout, Peter M. ; Banach, Maciej

Background:

Lowering low‐density lipoprotein cholesterol (LDL‐C) reduces the risk of major vascular events across all age groups. We analyzed phase 3 studies of bempedoic acid to characterize the safety and efficacy in patients aged ≥75 years with and without a concomitant statin.

Methods:

Post hoc analysis of bempedoic acid in high‐risk patients with cardiovascular disease by age from phase 3 placebo‐controlled, randomized clinical trials: two 52‐week primary hyperlipidemia studies with maximal background statin (“max‐statins pool”), and 1 cardiovascular outcomes trial (“statin‐intolerant pool”). Efficacy (LDL‐C, non–high‐density lipoprotein cholesterol, total cholesterol, high‐sensitivity C‐reactive protein) and safety were evaluated.

Results:

The max‐statins pool included 3009 (2010 bempedoic acid, 999 placebo) patients. The statin‐intolerant pool included 13 970 patients (6992 bempedoic acid, 6978 placebo). In total 7022 patients were aged 18 to <65 years; 7372 were aged 65 to <75 years; 2585 were aged ≥75 years. Placebo‐corrected mean percentage change in LDL‐C at week 12 in the max‐statin pool with bempedoic acid was −18.4% (95% CI, −21.3 to −15.6) for patients aged 18 to <65 years, −18.6% (95% CI, −21.2 to −16.1) for 65 to <75 years, and −18.3% (95% CI, −22.2 to −14.5) for ≥75 years. In the statin‐intolerant pool, LDL‐C change was −21.9% (95% CI, −23.1 to −20.7) for patients aged 18 to <65 years, −22.9% (95% CI, −24.0 to −21.8) for 65 to <75 years, and −24.5% (95% CI, −26.2 to −22.7) for ≥75 years. Frequency of adverse events compared with placebo was similar across the ages, but more frequent among patients ≥75 years regardless of background statin. Cardiovascular event reduction was statistically comparable across all ages.

Conclusions:

Bempedoic acid was well tolerated in patients aged ≥75 years with efficacy and safety comparable with younger subgroups regardless of background statin usage. Bempedoic acid may be considered a viable strategy for managing hypercholesterolemia in adults, regardless of age.

377

项与贝派地酸相关的新闻（医药）

2026-08-05

·BioSpace

HLS Therapeutics Provides Update on NILEMDO™ Market Access

Reimbursement secured with the largest private payers in Canada, estimated to represent approximately 80% of privately insured Canadians Canada's Drug Agency issues final recommendation, with its expert committee voting unanimously to recommend reimbursement of NILEMDO™ by public drug plans TORONTO , Aug. 5, 2026 /CNW/ -- HLS Therapeutics Inc. ("HLS" or the "Company") (TSX: HLS), a pharmaceutical company focused on addressing unmet needs in the treatment of psychiatric disorders and cardiovascular disease, announces positive developments in both private and public reimbursement coverage for NILEMDO™ (bempedoic acid) in Canada. Private Payer Coverage HLS has now secured reimbursement for NILEMDO with the largest private payers in Canada, representing approximately 80% of privately insured Canadians, the vast majority of whom can now access NILEMDO as a full benefit without restrictions. Several of these agreements are already in effect, with the remainder taking effect during the third quarter of 2026. Public Reimbursement On the public side, Canada's Drug Agency (CDA-AMC) has finalized its reimbursement recommendation for NILEMDO. The Canadian Drug Expert Committee voted unanimously to recommend that NILEMDO be reimbursed by participating public drug plans. This recommendation opens the window for HLS to begin negotiations with the pan-Canadian Pharmaceutical Alliance (pCPA) toward product listing agreements. HLS intends to commence those negotiations later this year, keeping it on track for initial provincial listings in the first half of 2027. A link to the CDA-AMC report can be found here: Bempedoic Acid (NILEMDO) "We are pleased with our progress on securing market access for NILEMDO," said Craig Millian, Chief Executive Officer at HLS. "For the many Canadians who are not reaching their LDL cholesterol targets on statins, whether due to intolerance or inadequate response, NILEMDO provides physicians and patients with a much-needed oral treatment option. Broad private coverage without restrictions, together with a clear pathway toward public reimbursement, means more Canadian patients will be able to access this medicine to help manage their cardiovascular risk." ezstandalone.cmd.push(function () { ezstandalone.showAds(131); }); ABOUT NILEMDO NILEMDO is indicated to reduce low-density lipoprotein cholesterol ("LDL-C") and the risk of major cardiovascular events in adults who require additional LDL-C lowering due to an inability to reach target levels with currently available therapies or due to statin intolerance. HLS in-licensed the exclusive rights to NILEMDO and NEXLIZET® (bempedoic acid and ezetimibe combination) for the Canadian market from Esperion Therapeutics, Inc. ABOUT HLS THERAPEUTICS INC. Formed in 2015, HLS is a pharmaceutical company focused on the acquisition and commercialization of late-stage development, commercial stage promoted and established branded pharmaceutical products in the North American markets. HLS's focus is on products targeting the treatment of psychiatric disorders and cardiovascular disease. HLS's management team is composed of seasoned pharmaceutical executives with a strong track record of success in these therapeutic areas and at managing products in each of these lifecycle stages. For more information visit: FORWARD LOOKING INFORMATION This release includes forward-looking statements regarding HLS and its business. Such statements are based on the current expectations and views of future events of HLS's management. In some cases the forward-looking statements can be identified by words or phrases such as "may", "will", "expect", "plan", "anticipate", "intend", "potential", "estimate", "believe" or the negative of these terms, or other similar expressions intended to identify forward-looking statements, including, among others, statements with respect to HLS's pursuit of additional product and pipeline opportunities in certain therapeutic markets, statements regarding growth opportunities, expectations regarding financial performance, and the NCIB and ASPP. The forward-looking events and circumstances discussed in this release may not occur and could differ materially as a result of known and unknown risk factors and uncertainties affecting HLS, including risks relating to the specialty pharmaceutical industry, risks related to the regulatory approval process, economic factors and many other factors beyond the control of HLS. Forward-looking statements and information by their nature are based on assumptions and involve known and unknown risks, uncertainties and other factors which may cause HLS's actual results, performance or achievements, or industry results, to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statement or information. A discussion of the material risks and assumptions associated with this release can be found in the Company's Annual Information Form dated March 11, 2026, and Management's Discussion and Analysis dated May 14, 2026, both of which have been filed on SEDAR+ and can be accessed at . Accordingly, readers should not place undue reliance on any forward-looking statements or information. Except as required by applicable securities laws, forward-looking statements speak only as of the date on which they are made and HLS undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events, or otherwise. SOURCE HLS Therapeutics Inc.

引进/卖出

2026-08-02

·知乎专栏

从“Biotech 寒冬”到结构性机会的2026酷暑

一场罕见病与新机制的投资人笔记本文基于 BioSpark Rare Disease Spotlight / 月度 Biotech 新闻讨论会内容，并结合截至 2026 年 8 月 2 日的公开资料，尝试从投资和新药研发两个角度，整理几个值得持续跟踪的方向：罕见肿瘤：Chordoma 与 Brachyury 靶点罕见心血管疾病：ATTR-CM 的机制分化慢病市场：PCSK9 抑制剂的口服化精神疾病：迷幻类资产获得大药企认可这些方向看上去彼此毫不相关，但背后其实存在一条共同主线：资本没有重新爱上所有 Biotech。它只是重新愿意为那些具有明确人类遗传学依据、临床可验证机制和清晰退出路径的少数资产付费。一、宏观背景：XBI 回暖，但不是所有公司都能获益2025 年，美国 SPDR S&P Biotech ETF，也就是大家熟悉的 XBI，全年上涨约 33%。与此同时，Biotech IPO 市场在经历连续数年的低迷之后，于 2026 年出现明显复苏迹象。2025 年全年，美国只有约 10 至 11 家 Biotech 公司完成 IPO，远低于 2021 年超过 100 家的盛况。但到 2026 年上半年，已经有约 18 家生物医药公司登陆公开市场，数量超过 2025 年全年。(Reuters⁠) 插播会议广告：欢迎报名参加波士顿MIT校园举办的BioSpark 2026 年会时间：2026 年 9 月 26 日地点：MIT 校园核心叙事：Inflection Point，也就是“拐点”会议关注心血管代谢、免疫、RNA、神经科学、罕见病以及中国 Biotech 等主题。据会议介绍，预计将有约 400 至 500 名参会者。公开活动页面并未单独披露人数，但已经明确把行业描述为进入“科学融合与资本纪律并存”的新阶段。(LinkedIn⁠)广告结束这里要特别注意，“回暖”不等于重新回到 2020 年至 2021 年的平台泡沫。现在的资金更关心三个问题：第一，你的靶点在人类疾病中真的成立吗？第二，你的技术能否解决过去做不到的事情？第三，临床数据出来之后，谁会买你？因此，2026 年的关键词并不是全面复苏，而是更残酷、更集中的筛选。二、罕见肿瘤 Chordoma：Brachyury 是一个典型的“少数派靶点”1. 一个每百万人不到一例的肿瘤Chordoma 中文常译为脊索瘤，起源于胚胎脊索残留组织，通常发生在骶骨、颅底或脊柱。其年发病率大约只有每百万人 0.8 至 1 例，属于真正意义上的超罕见肿瘤。治疗以手术和放射治疗为主，复发或转移之后，可选择的系统治疗仍然非常有限。(PubMed Central (PMC)⁠)对于传统投资人而言，这种疾病最大的问题非常直观：患者太少，临床试验招募困难，商业市场也不够大。但 Chordoma 又具有一个罕见优势：它可能拥有肿瘤学中最清晰的转录因子依赖之一，也就是 Brachyury。2. Brachyury 到底是什么Brachyury 由 TBXT 基因编码，是胚胎发育过程中决定中胚层和脊索形成的重要转录因子。在正常成年组织中，Brachyury 的表达非常有限；但在绝大多数 Chordoma 肿瘤中，它持续高表达，并且是肿瘤细胞维持生存和脊索表型的重要依赖因素。换句话说：Chordoma 不只是“表达 Brachyury”，而是很可能“离不开 Brachyury”。这使它具备了一个非常漂亮的靶点组合：疾病特异性较强正常组织表达较低具有明确的功能依赖存在潜在的肿瘤免疫原性问题也同样明显：Brachyury 是一个转录因子。过去药物化学领域经常把这类蛋白归入“不可成药靶点”，原因是它们通常缺少传统小分子可以进入的深口袋。3. 2025 年之后，事情开始发生变化2025 年，《Nature Communications》发表了一项重要研究，提供了 Brachyury 蛋白结构和配体结合位点的详细信息。研究人员通过结构分析和片段筛选，发现了多个可以被小分子占据的结合热点，并获得了一系列低微摩尔级 Brachyury 结合分子。这些化合物还远远称不上临床候选药物，但意义非常明确：Brachyury 从“理论上重要但碰不到”，变成了“至少在化学上可以接触”。这一步往往是一个转录因子靶点从学术概念走向药物项目的起点。(Nature⁠)到了 2026 年 AACR 年会，X-Chem 团队又报告了利用 DNA 编码化合物库，也就是 DEL 发现 Brachyury 结合分子的工作，并通过 SPR 对部分分子的直接结合进行了验证。(AACR Journals⁠)目前围绕 Brachyury 的药物研发大致可以分成三条路线：路线一：直接小分子抑制剂寻找能够影响 Brachyury DNA 结合、转录复合物形成或者蛋白稳定性的分子。这是最经典的药物化学路线，但也是难度最高的一条路线。路线二：靶向蛋白降解如果小分子只能“抓住”Brachyury，却无法有效抑制它，那么 PROTAC、分子胶或者其他蛋白降解技术，可能把弱结合转化为功能性降解。对这类缺乏传统酶活口袋的转录因子，降解往往比占位抑制更有吸引力。路线三：免疫治疗Brachyury 蛋白可以被加工成肽段，并由 MHC 分子呈递到肿瘤细胞表面。研究人员正在开发针对 Brachyury-MHC 复合物的 TCR-T、TCR mimic 抗体和其他细胞治疗。Chordoma Foundation 在 2025 年表示，相关团队计划于 2026 年推动首个人体临床试验。(Chordoma Foundation⁠)4. 投资人真正应该看什么Chordoma 的投资逻辑并不应该建立在患者数量上，而应该建立在靶点外溢价值上。Brachyury 还与肿瘤 EMT、侵袭、转移以及治疗耐药有关，在肺癌、乳腺癌、前列腺癌等肿瘤中也存在异常表达。因此，一个真正有效的 Brachyury 药物，最初可能在 Chordoma 中验证靶点，但最终商业边界未必停留在 Chordoma。不过，这里需要保持冷静：目前 Brachyury 具有很强的靶点验证，但尚未完成真正的药物验证。低微摩尔结合分子、体外降解以及细胞杀伤，都不等于人体临床疗效。投资判断：Brachyury 是一个高风险、高信息价值的项目。它可能不是最适合传统大基金的第一笔投资，却很适合具备药化、蛋白降解、结构生物学或肿瘤免疫能力的专业团队进行早期布局。三、ATTR-CM：同一个蛋白，已经出现四种完全不同的治疗逻辑1. ATTR-CM 不再是一个简单的“稳定剂市场”ATTR-CM，全称为转甲状腺素蛋白淀粉样变性心肌病。其核心病理过程是：正常的四聚体 TTR 蛋白解离，单体错误折叠并形成淀粉样纤维，随后沉积在心肌组织中，导致心室壁增厚、舒张功能障碍、心律失常和心力衰竭。过去，这个市场主要由 TTR 稳定剂定义。现在，ATTR-CM 已经形成至少四个机制层级：稳定蛋白：阻止 TTR 四聚体解离沉默表达：减少肝脏产生 TTR基因编辑：一次性关闭 TTR 基因清除沉积：移除已经形成的淀粉样蛋白这四种机制的临床价值并不相同。稳定剂主要解决“不要继续坏下去”。RNAi 和 ASO 解决“不要再生产这么多”。基因编辑试图实现“一次治疗，长期沉默”。淀粉样清除剂则试图处理最困难的问题，也就是“已经沉积在心脏里的东西怎么办”。2. Acoramidis 和 Vutrisiran 改写了竞争格局2024 年 11 月，FDA 批准 acoramidis，商品名 Attruby，用于治疗野生型或遗传型 ATTR-CM 成人患者。它是一种口服 TTR 稳定剂，通过提高四聚体稳定性，减少后续错误折叠和淀粉样沉积。(U.S. Food and Drug Administration⁠)2025 年 3 月，FDA 又批准了 Alnylam 的 vutrisiran，商品名 Amvuttra，使其成为首个获批用于 ATTR-CM 的 RNAi 治疗。Vutrisiran 通过沉默肝细胞中的 TTR mRNA，减少循环 TTR 蛋白，每三个月皮下注射一次。HELIOS-B 研究显示，与安慰剂相比，vutrisiran 可以降低全因死亡和复发性心血管事件风险。(Alnylam Investor Relations⁠)这意味着 ATTR-CM 已经从“有没有药”，进入了“哪一种机制更适合哪一类患者”的阶段。3. 同一种机制，也可能出现完全不同的临床结果2026 年 7 月，Ionis 和 AstraZeneca 公布 eplontersen 的 CARDIO-TTRansform III 期研究结果。该研究未达到心血管死亡和复发性心血管事件复合终点的主要目标。公司指出，在部分单药治疗亚组中观察到了名义上的统计学改善，但整体研究仍然属于主要终点失败。(Ionis Pharmaceuticals, Inc.⁠)这件事给投资人一个非常重要的提醒：一个机制类别已经被验证，并不意味着同类的每一个分子、每一个剂量和每一种试验设计都能成功。ATTR-CM 临床试验还会受到许多因素影响：患者是否同时使用稳定剂患者处于早期还是晚期野生型和遗传型比例基础心衰治疗是否充分终点采用生物标志物、六分钟步行，还是死亡和住院事件随访时间是否足够长这些变量都会改变最终结果。4. 基因编辑的吸引力与代价Intellia 开发的 nexiguran ziclumeran，简称 nex-z，原名 NTLA-2001，利用脂质纳米颗粒将 CRISPR-Cas9 系统递送至肝脏，通过一次性编辑关闭 TTR 基因。早期研究显示，患者接受一次治疗后，血清 TTR 可持续下降约 90%。但在 III 期开发过程中，相关研究曾因严重肝脏安全事件受到临床暂停，随后在增加肝功能监测、预防性处理和排除部分高风险患者后恢复。(Oligonucleotide Therapeutics Society⁠)这里体现了基因编辑在慢病领域最核心的矛盾：一次治疗可能带来多年收益，但任何严重脱靶或器官毒性也可能难以逆转。投资判断：ATTR-CM 的赢家未必只有一个。稳定剂可能服务于早期和轻症患者，RNAi 可能提供更深的蛋白降低，基因编辑争夺一次性治疗市场，淀粉样清除剂则可能进入高负荷和进展期患者。未来真正值得关注的不是“谁能把 TTR 降得最低”，而是：谁能改善死亡和住院谁能在背景治疗下继续显示增益谁能建立清楚的患者分层四、PCSK9 口服化：从注射降脂药走向大众慢病市场1. PCSK9 一直是好靶点，只是给药方式限制了市场PCSK9 会促进肝细胞表面的 LDL 受体降解。抑制 PCSK9 后，更多 LDL 受体可以回到细胞表面，从血液中清除 LDL-C。过去，PCSK9 药物主要包括：单克隆抗体：每两周或每月注射siRNA：每半年注射这些药物降脂效果很好，但由于价格、注射方式、医生处方习惯和保险限制，使用范围一直远小于他汀类药物。因此，PCSK9 口服化一直被视为心血管领域最有价值的药物工程问题之一。2. 2026 年 7 月，第一个口服 PCSK9 抑制剂获批2026 年 7 月 16 日，FDA 批准默沙东的 enlicitide，商品名 Lipfendra，用于治疗成人高胆固醇血症。这是全球首个获批的每日一次口服 PCSK9 抑制剂。(Merck⁠)Enlicitide 不是传统意义上的小分子，而是一个经过环化和口服吸收优化的大环肽。它直接结合 PCSK9，阻止 PCSK9 与 LDL 受体相互作用，在功能上复制了注射型 PCSK9 抗体的机制。在 CORALreef 系列 III 期研究中，enlicitide 在标准降脂治疗基础上，可使 LDL-C 进一步下降约 57% 至 60%。在部分研究中，其降脂效果也优于 ezetimibe、bempedoic acid 及二者联合方案。(New England Journal of Medicine⁠)这不只是“把注射药做成口服药”。它可能改变整个降脂市场的治疗顺序：过去，患者通常先使用他汀，再加 ezetimibe，仍然控制不佳时才考虑注射 PCSK9 药物。现在，临床医生可能更早加入口服 PCSK9 抑制剂。深度学习推荐观看biospark嘉宾deep dive之环肽设计巅峰之作Enlicitide的故事3. AstraZeneca 也在追赶AstraZeneca 的 AZD0780 是另一种口服 PCSK9 抑制剂。与 enlicitide 的大环肽不同，AZD0780 更接近传统小分子开发逻辑，目前已经进入 III 期临床阶段。PURSUIT IIb 期研究显示，AZD0780 在标准治疗基础上可以进一步显著降低 LDL-C。(AstraZeneca⁠)因此，口服 PCSK9 市场最终可能演变成几类药物竞争：大环肽传统小分子固定剂量复方药物长效注射 RNAi单克隆抗体4. 口服化并不自动等于商业成功口服 PCSK9 抑制剂仍然面临几个问题：每日服药依从性可能低于每半年一次注射部分药物存在空腹服用等给药要求价格需要与他汀、ezetimibe 和 bempedoic acid 竞争长期心血管结局数据仍然需要积累支付方是否愿意把它提前到二线治疗仍不确定但从产业角度看，它验证了一条非常重要的路线：过去只能通过抗体阻断的蛋白相互作用，也可能通过大环肽和精心设计的小分子实现口服干预。这对口服 GLP-1、细胞因子抑制剂、蛋白相互作用抑制剂以及其他“抗体口服替代”项目，都具有示范价值。投资判断：PCSK9 的风险已经不再是靶点风险，而是产品竞争力和商业执行风险。未来决定胜负的可能不是 LDL-C 多降低几个百分点，而是：服药是否方便能否组成固定剂量复方医保是否愿意覆盖心血管结局是否得到验证五、迷幻类药物：大药企认可的是药物，不是文化1. 两笔交易改变了市场态度2025 年，AbbVie 宣布收购 Gilgamesh Pharmaceuticals 的 bretisilocin 项目，交易潜在总金额最高约 12 亿美元。Bretisilocin 是一种经过优化的 5-HT2A 受体激动剂，目标是在保留快速抗抑郁作用的同时，缩短传统裸盖菇素类药物持续数小时的精神体验。AbbVie 于 2025 年 10 月完成相关收购。(AbbVie News Center⁠)2026 年 7 月 16 日，礼来宣布收购 AtaiBeckley。交易包括约 28 亿美元预付款，潜在总金额最高约 38 亿美元。核心资产 BPL-003 是一种鼻腔给药的合成 5-MeO-DMT 制剂，主要开发方向包括难治性抑郁症。(Eli Lilly and Company⁠)这两笔交易说明，大药企已经不再把迷幻类药物视为边缘文化产品。但它们真正买入的，也不是“迷幻体验”本身。它们购买的是：快速起效的抗抑郁机制更短的精神活性时间可以标准化的临床流程可被专利保护的分子和制剂适合纳入精神科商业体系的产品2. COMP360 接近监管关键节点Compass Pathways 的 COMP360 psilocybin 已完成两项针对难治性抑郁症的 III 期研究。2026 年 7 月，公司公布第二项 III 期研究的六个月随访数据，并表示已经启动滚动 NDA 申报，计划在 2026 年第四季度完成全部提交。若获得批准，COMP360 有可能成为美国首批进入市场的裸盖菇素类处方治疗之一。(Compass Pathways⁠)同月，FDA 发布了迷幻类药物临床研究的最终指导原则，对以下问题提出了更具体的要求：盲法和对照组设计心理支持人员的培训药物与心理干预效果的区分急性精神反应和自杀风险监测滥用潜力评估长期疗效和安全性随访(U.S. Food and Drug Administration⁠)这意味着监管路径正在变得清楚，但并不意味着监管标准正在放松。3. MDMA 的教训不能忘记2024 年，Lykos Therapeutics 的 MDMA 辅助治疗 PTSD 申请收到 FDA 完整回复函，未能获批。该项目暴露出迷幻药临床开发中特有的问题：受试者很容易猜到自己是否接受了活性药物，导致功能性破盲。治疗师的经验、行为和现场管理可能影响疗效。不同试验中心之间难以保持完全一致。药物效应和心理治疗效应难以拆分。长时间留观增加了诊所、人力和商业化成本。因此，大药企现在更偏爱作用时间较短、流程更简单、分子本身贡献更明确的下一代资产。(OpenFDA Download⁠)投资判断：AbbVie 和礼来的入场，是对“快速起效精神疾病药物”赛道的认可，而不是对所有迷幻类公司的集体背书。真正有价值的资产，往往具备以下特征：起效快精神体验时间短诊所占用时间有限安全窗口清楚不需要高度复杂的心理治疗配套可以被现有精神科商业体系承接六、2026 年，资本究竟在为什么付钱把 Brachyury、ATTR-CM、口服 PCSK9 和迷幻类药物放在一起，会发现资本关注的并不是某一种疾病，而是四种更底层的价值。1. 被人类疾病验证过的生物学Brachyury 是 Chordoma 的核心依赖。TTR 是 ATTR 的直接致病蛋白。PCSK9 拥有清楚的人类遗传学和临床验证。5-HT2A 及相关神经可塑性通路已经积累大量药理证据。在资本更加谨慎的环境中，validated biology 的价值反而上升。2. 新技术把旧靶点重新打开很多结构性机会并不是发现了一个全新的靶点，而是新技术让旧靶点第一次变得可开发：结构生物学和 DEL 让 Brachyury 可以被小分子接触。RNAi 和 CRISPR 让 TTR 可以被深度沉默。大环肽让 PCSK9 阻断首次实现口服。短效分子和新制剂让迷幻药更接近标准化精神科治疗。3. 临床路径足够清楚好的 Biotech 项目不仅需要机制漂亮，还需要回答：在哪一类患者中做选择什么终点多久能读出数据是否存在可靠生物标志物失败后能否解释原因能否设计出下一项试验投资人真正害怕的不是一次试验失败，而是失败之后仍然不知道靶点、分子、剂量和患者选择中，到底哪一个出了问题。4. 有明确的 BD 和退出路径在今天的融资环境中，很多早期公司并不需要独立完成全球商业化。它们需要证明的是：某个大药企在什么阶段会需要这个资产。Brachyury 可能被肿瘤免疫、蛋白降解或精准肿瘤公司收购。ATTR 项目可以进入心血管和罕见病巨头的产品组合。口服 PCSK9 可以成为大型心血管公司的复方核心。下一代迷幻类分子则可以补充大药企长期薄弱的精神疾病管线。结语：2026 年的机会，不属于“所有 Biotech”所谓 Biotech 拐点，并不是融资重新变得容易，也不是所有估值都会回到高点。真正发生的变化是：市场开始愿意重新讨论长期价值，但要求项目拿出更扎实的证据。2026 年最值得跟踪的资产，往往同时具备三点：疾病机制足够真实技术带来了过去不存在的解决方案临床和商业路径足够具体所以，与其问“Biotech 寒冬结束了吗”，不如换一个问题：在资本仍然谨慎的情况下，哪些靶点和机制已经强到足以穿越寒冬？Brachyury、ATTR-CM、口服 PCSK9 和下一代迷幻类药物，正在给出四种不同的答案。主要参考文献与新闻链接BioSpark 与行业环境2026 BioSpark Annual Conference：September 26 at MITBioSpark Monthly Biotech News ReviewReuters：US biotech sector poised for 2026 reboundBioSpace：Biotech IPOs surge in the first half of 2026Chordoma 与 BrachyuryNature Communications：Structural insights into human Brachyury and fragment bindersChordoma Foundation：Brachyury Drug Discovery InitiativeChordoma Foundation：TBXT ChallengeChordoma Foundation：Brachyury immunotherapy researchAACR 2026：DEL data-driven discovery of Brachyury bindersATTR-CMFDA：Approval of acoramidis for ATTR-CM⁠Alnylam：FDA approval of vutrisiran for ATTR-CM⁠NEJM：Vutrisiran in patients with transthyretin amyloidosis with cardiomyopathy⁠Ionis：CARDIO-TTRansform Phase III update⁠ Oligonucleotide Therapeutics Society：Nex-z trials resume after clinical holds⁠口服 PCSK9Merck：FDA approval of Lipfendra, the first oral PCSK9 inhibitor⁠NEJM：Oral enlicitide for hypercholesterolemia⁠Reuters：Enlicitide reduced LDL cholesterol in late-stage trial⁠AstraZeneca：AZD0780 PURSUIT Phase IIb results⁠迷幻类药物与精神疾病AbbVie：Acquisition of Gilgamesh bretisilocin program⁠Lilly：Acquisition of AtaiBeckley⁠Reuters：Lilly enters psychedelic drug race with AtaiBeckley deal⁠Compass Pathways：COMP360 Phase III six-month data⁠FDA：Final guidance for clinical investigations of psychedelic drugs⁠FDA：Complete Response Letter for MDMA-assisted therapy NDA⁠最后自保一句：本文仅用于行业研究、药物研发和投资逻辑讨论，不构成证券投资或医疗建议。

IPO放射疗法

2026-07-31

·BioSpace

Daiichi Sankyo Reports First Quarter Fiscal Year 2026 Financial Results

Revenue increased 21.1% to 574.7 billion yen year-over-year driven by Enhertu ® and Datroway ® Fiscal year 2026 guidance for revenue and operating profit revised upward Conference call to take place on Friday, July 31 at 5:30 pm Japan Standard Time TOKYO--(BUSINESS WIRE)--Daiichi Sankyo (TSE: 4568) reported financial results for the first quarter of fiscal year 2026, ending June 30, 2026. “Daiichi Sankyo delivered strong revenue growth in the first quarter of fiscal year 2026 as we began execution of our new Five-Year Business Plan,” said Hiroyuki Okuzawa, President and CEO of Daiichi Sankyo. “Three new U.S. approvals for Enhertu and Datroway were received in a single week in May, underscoring the continued strength of our portfolio where we anticipate delivering more than 20 new indications across five medicines by 2030. These results reinforce the durability of our growth trajectory and support our outlook for fiscal 2026 and beyond.” Q1 FY2026 Results Global revenue increased 21.1% year-over-year to 574.7 billion yen, driven by the company’s global oncology products. Operating profit decreased 12.0% year-over-year to 85.1 billion yen, primarily due to EU Specialty Business restructuring expenses. Global revenue from oncology products was driven by Enhertu ® and Datroway ® , the company's flagship antibody drug conjugates (ADCs). Enhertu revenue, including milestone payments, was 239.8 billion yen, a 48.9% increase. Datroway revenue, including milestone payments, was 23.9 billion yen, a 176.4% increase. ezstandalone.cmd.push(function () { ezstandalone.showAds(131); }); Revenue from the Japan Business was 120.0 billion yen, driven by Lixiana ® and Tarlige ® . ASCA (Asia, South and Central America) Business revenue was 68.7 billion yen, driven by Enhertu. EU Specialty Business revenue was 74.7 billion yen, driven by Lixiana and Nilemdo ® /Nustendi ® . Full fiscal year 2026 guidance has been revised upward from the forecast issued in May 2026. Global revenue is anticipated to increase from 2.28 to 2.34 trillion yen, driven by stronger sales anticipated in the U.S. and favorable foreign exchange rates. Operating profit is anticipated to increase from 315.0 to 320.0 billion yen. Further details on financial performance can be found in the Financial Results Presentation Material . Daiichi Sankyo will host an investor and media conference call and webcast to discuss Q1 FY2026 results on Friday, July 31 at 5:30 pm Japan Standard Time. Q4 FY2025 Results Revision Operating profit has increased from 229.1 billion yen to 258.0 billion yen. Following the announcement of the Q4 FY2025 financial results on May 11, 2026, the Company identified an error in the aggregation process for selling, general and administrative (SG&A) expenses. As a result of the decrease in SG&A expenses, operating profit has been revised upward. Portfolio and Pipeline Highlights Daiichi Sankyo continued to advance its portfolio and pipeline with several milestones across its oncology portfolio, demonstrating momentum towards its goal of launching 20 new indications across five medicines by 2030. Three new breast cancer indications were approved within one week in the U.S. for Enhertu and Datroway in May 2026, including simultaneous approval of two new indications for Enhertu in the neoadjuvant and adjuvant HER2 positive early breast cancer setting based on the DESTINY-Breast11 and DESTINY-Breast05 phase 3 trials, followed by one new indication for Datroway in the first-line metastatic triple negative breast cancer (TNBC) setting for patients not candidates for immunotherapy based on the TROPION-Breast02 phase 3 trial. Datroway received approval in the EU for the same TNBC indication and Enhertu received a positive CHMP opinion for the first-line treatment of HER2 positive metastatic breast cancer based on the DESTINY-Breast09 phase 3 trial in July 2026. A Biologics License Application (BLA) was accepted and granted priority review in the U.S. in April 2026 for ifinatamab deruxtecan (I-DXd) for the treatment of adult patients with extensive-stage small cell lung cancer with disease progression on or after platinum-based chemotherapy, with a Prescription Drug User Fee Act (PDUFA) date of October 10, 2026. Additional regulatory approvals were received in June 2026 for medicines in other major countries/regions. Enhertu received approval in the EU for a HER2 positive metastatic tumor agnostic indication and Vanflyta ® was approved in China in the newly diagnosed setting of FLT3 -ITD positive acute myeloid leukemia (AML). Data from more than 25 abstracts across multiple cancers were presented at the 2026 American Society of Clinical Oncology (#ASCO26) in June 2026. Medicine Indication Q1 FY2026 Update Enhertu Breast Enhertu Approved in the U.S. for Two New Indications for Patients with HER2 Positive Early Breast Cancer ( May 2026 ) Enhertu Plus Pertuzumab Recommended for Approval in the EU by CHMP as First-Line Treatment for Patients with HER2 Positive Metastatic Breast Cancer ( July 2026 ) Tumor Agnostic Enhertu Approved in the EU as First Tumor Agnostic HER2 Directed Therapy and Antibody Drug Conjugate for Patients with Previously Treated HER2 Positive Metastatic Solid Tumors ( June 2026 ) Datroway Breast Datroway Approved in the U.S. as First TROP2 Directed Antibody Drug Conjugate for First-Line Treatment of Patients with Metastatic Triple Negative Breast Cancer Who Are Not PD-1/PD-L1 Inhibitor Candidates ( May 2026 ) Datroway Approved in the EU as Only TROP2 Directed Medicine with Overall Survival Benefit for the First-Line Treatment of Patients with Metastatic TNBC Who Are Not Candidates for Immunotherapy ( July 2026 ) I-DXd Lung Ifinatamab Deruxtecan Granted Priority Review in the U.S. for Adult Patients with Previously Treated Extensive-Stage Small Cell Lung Cancer who Experienced Disease Progression on or After Platinum-Based Chemotherapy ( April 2026 ) Vanflyta AML Vanflyta Approved in China as First and Only FLT3 Inhibitor for Patients with Newly Diagnosed FLT3 -ITD Positive AML ( June 2026 ) Pipeline Various Daiichi Sankyo Showcases Progress Across Industry-Leading Oncology Portfolio with Latest Research Updates at ASCO ( May 2026 ) Corporate Highlights Daiichi Sankyo announced its next Five-Year Business Plan (FY2026 - FY2030) in May 2026 outlining how the company plans to deliver more than 2.3 trillion yen in oncology revenue by 2030 and be a global top five oncology company by 2035 as part of its larger 2035 Vision to be recognized as a “trusted healthcare innovator transforming the lives of people through our science and technology.” As part of the company’s continued transformation into a global oncology company, Daiichi Sankyo announced in April 2026 that it will transfer in stages all of its shares of Daiichi Sankyo Healthcare Co., Ltd, its over-the-counter drug business in Japan, to Suntory Holdings Limited. The deal is estimated to be completed by June 2029. About Daiichi Sankyo Daiichi Sankyo (TSE: 4568) is a global healthcare company committed to becoming a trusted healthcare innovator, transforming the lives of people through its strength in science and technology. The company discovers and develops new standards of care to address diverse medical needs to fulfill its purpose of contributing to the enrichment of quality of life around the world. With a strategic focus on oncology, Daiichi Sankyo is advancing an industry-leading antibody drug conjugate portfolio along with identifying new breakthrough generating technologies to deliver practice-changing medicines to patients, healthcare professionals and society. For more information, please visit . Forward-Looking Statements Management strategies and plans, financial forecasts, future projections and policies, and R&D information that Daiichi Sankyo discloses in this material are all classified as Daiichi Sankyo’s future prospects. These forward-looking statements, including words such as “anticipate,” “estimated,” “favorable,” “goal,” “momentum,” “outlook,” “trajectory,” were determined by Daiichi Sankyo based on information obtained as of today with certain assumptions, premises and future forecasts, and thus, there are various inherent risks as well as uncertainties involved. As such, please note that actual results of Daiichi Sankyo may diverge materially from the outlook of Daiichi Sankyo or the content of this material. Furthermore, there is no assurance that any forward-looking statements in this material will be realized. Regardless of the actual results or facts, Daiichi Sankyo is not obliged and does not have in its policy the duty to update the content of this material from the date of this material onward. Some of the compounds under discussion are investigational agents and are not approved by the FDA or any other regulatory agency worldwide as a treatment for indications under investigation. Efficacy and safety have not been established in areas under investigation. There are no guarantees that these compounds will become commercially available in indications under investigation. Daiichi Sankyo takes reasonable care to ensure the accuracy of the content of this material but shall not be obliged to guarantee the absolute accuracy, appropriateness, completeness and feasibility, etc. of the information described in this material. Furthermore, any information regarding companies, organizations or any other matters outside the Daiichi Sankyo Group that is described within this material has been compiled or cited using publicly available information or other information, and Daiichi Sankyo has not performed in-house inspection of the accuracy, appropriateness, completeness and feasibility, etc. of such information, and does not guarantee the accuracy thereof. The information described in this material may be changed hereafter without notice. Accordingly, this material or the information described herein should be used at your own judgment, together with any other information you may otherwise obtain. This material does not constitute a solicitation of application to acquire or an offer to sell any security in the United States, Japan or elsewhere. This material disclosed here is for reference purposes only. Final investment decisions should be made at your own discretion. Daiichi Sankyo assumes no responsibility for any damages resulting from the use of this material or its content, including without limitation damages related to the use of erroneous information. Contacts MEDIA CONTACTS: Global: Jennifer Brennan jennifer.brennan@daiichisankyo.com +1 908 900 3183 (mobile) INVESTOR RELATIONS CONTACT: DaiichiSankyoIR_jp@daiichisankyo.com Japan: DS-PR_jp@daiichisankyo.com

上市批准免疫疗法临床3期ASCO会议财报

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
高低密度脂蛋白胆固醇血症	加拿大	HLS Therapeutics, Inc.	2026-03-05
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

未上市

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
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
脑血管疾病	临床3期	智利	Esperion Therapeutics, Inc.	2016-12-22

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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


NCT05694260 (CLEAR Path 1, CTgov)	临床2期	杂合子家族性高胆固醇血症	31	Bempedoic Acid (Cohort 1 (16 to <30 Kg) Bempedoic Acid 60 mg)	鏇艱醖淵糧觸齋網襯鏇(顧鑰遞簾壓襯衊襯觸鏇) = 積衊鹹膚繭鏇構選蓋鏇醖淵鬱鬱觸鏇鑰壓網衊 (襯鏇齋壓獵醖鬱鹽製繭, 39.6) 更多	-	2026-07-17
				Bempedoic Acid (Cohort 1 (16 to <30 Kg) Bempedoic Acid 90 mg)	鏇艱醖淵糧觸齋網襯鏇(顧鑰遞簾壓襯衊襯觸鏇) = 選遞鹽衊餘艱繭範觸簾醖淵鬱鬱觸鏇鑰壓網衊 (襯鏇齋壓獵醖鬱鹽製繭, 20.4) 更多
NCT02993406 (CLEAR Outcomes, Pubmed \| JAMA Cardiol)	临床3期	静脉血栓栓塞	13,970	Bempedoic acid	鑰膚鑰鹹蓋淵醖鹹鹹衊(憲糧醖醖範鹹繭構觸膚) = 鹹鹽衊襯鑰餘築鹽憲鏇網襯餘襯憲淵襯膚餘夢 (廠壓壓築獵鏇糧艱顧糧 )	积极	2026-05-27
				Placebo	鑰膚鑰鹹蓋淵醖鹹鹹衊(憲糧醖醖範鹹繭構觸膚) = 艱獵糧願鑰憲壓鏇糧鹹網襯餘襯憲淵襯膚餘夢 (廠壓壓築獵鏇糧艱顧糧 )
NCT06925100 (CLEAR Taiwan, Pubmed \| J Am Heart Assoc)	临床4期	高胆固醇血症	180	Bempedoic acid	鏇積淵顧鹽廠衊簾範繭(製餘簾齋簾網簾餘築鬱) = 齋遞糧遞築鏇鑰廠選顧廠憲齋鬱襯醖簾範觸鹽 (繭鹽顧艱鹹築夢窪鹹構 )	积极	2026-05-19
NCT05687071 (CTgov)	临床3期	高低密度脂蛋白胆固醇血症	130	ETC-1002	膚膚簾遞網糧艱製廠餘 = 鏇艱簾鬱壓範衊膚鬱憲鹽遞顧膚獵餘齋觸醖積 (壓繭餘齋鹽鑰淵顧壓艱, 餘鏇鬱壓鏇壓醖積餘積 ~ 網製蓋餘鬱廠憲鹽構鬱) 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	構鹹製範獵繭齋遞選範(襯餘製夢齋鏇膚餘醖糧) = 積顧鬱構齋構壓艱築餘蓋糧糧醖窪鬱醖遞糧獵 (淵範鬱觸艱壓憲醖簾鹹 )	积极	2025-08-20
				Placebo	構鹹製範獵繭齋遞選範(襯餘製夢齋鏇膚餘醖糧) = 淵觸鏇願憲願鏇壓獵糧蓋糧糧醖窪鬱醖遞糧獵 (淵範鬱觸艱壓憲醖簾鹹 )
NCT05683340 (CTgov)	临床3期	高低密度脂蛋白胆固醇血症	96	Placebo	襯憲製壓襯齋壓廠繭淵(襯願構齋襯齋膚觸衊醖) = 繭壓顧選衊襯觸齋遞廠鹹廠選簾構糧鹹顧鹹選 (蓋積膚襯齋壓製餘選遞, 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	鹽繭獵遞憲窪觸鬱廠築(簾構觸鬱夢願鏇窪築憲) = 壓網鹹膚鹽鬱醖製顧網鹹遞繭窪鬱繭淵壓壓鏇 (積顧鏇顧範襯觸鑰顧築, 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	網膚蓋繭選淵築鏇選廠(鑰衊襯遞積窪膚淵糧齋) = 製遞積獵繭願範範顧範製襯觸淵鏇醖憲糧選製 (遞願鬱願醖選製鏇襯繭 ) 达到	积极	2024-05-20
				Placebo	網膚蓋繭選淵築鏇選廠(鑰衊襯遞積窪膚淵糧齋) = 窪夢窪顧窪壓範壓糧糧製襯觸淵鏇醖憲糧選製 (遞願鬱願醖選製鏇襯繭 ) 达到
NCT04784442 (CTgov)	临床2期	高胆固醇血症	188	Placebo	鑰顧獵壓鬱糧膚餘餘觸(觸獵鏇製鏇膚觸繭範構) = 蓋遞夢鬱鹹築鑰餘憲製觸襯鹽餘鹽繭鹹鏇築夢 (願廠觸構鹹蓋窪齋鏇淵, 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)	壓構鏇蓋鬱醖糧餘壓憲 = 簾築鬱鹽窪鬱製蓋齋夢憲餘繭膚遞鏇獵獵簾範 (襯範鬱憲築糧蓋網鏇淵, 廠繭憲蓋餘襯餘齋淵願 ~ 製醖膚餘鹹願憲獵遞鏇) 更多	-	2024-01-03
				Matching placebo tablet (Placebo Comparator)	壓構鏇蓋鬱醖糧餘壓憲 = 壓醖淵廠鏇積繭願夢鏇憲餘繭膚遞鏇獵獵簾範 (襯範鬱憲築糧蓋網鏇淵, 遞廠蓋夢選蓋齋鑰憲觸 ~ 夢夢鹹積蓋製襯遞鏇願) 更多