A Randomized Double-blind, Placebo-controlled, Multicenter Study to Evaluate the Efficacy, Safety and Tolerability of Pelacarsen (TQJ230) in US Black/African American & Hispanic Patient Populations With Elevated Lp(a) and Established Atherosclerotic Cardiovascular Disease

Study CTQJ230A12303 is a randomized, double-blind placebo-controlled, Phase IIIb study to evaluate the efficacy, safety and tolerability of pelacarsen (TQJ230) 80 mg s.c. QM compared with placebo s.c. QM in US Black/African American and Hispanic participants with established ASCVD and elevated levels of Lp(a) who are treated for cardiovascular (CV) risk factors according to local practice/guidelines for the reduction of cardiovascular risk.

开始日期2024-04-24

申办/合作机构

Novartis Pharmaceuticals Corp.

NCT05646381 / Recruiting临床2期

A Randomized Double-blind, Placebo-controlled, Multicenter Trial Assessing the Impact of Lipoprotein(a) Lowering With Pelacarsen (TQJ230) on the Progression of Calcific Aortic Valve Stenosis [Lp(a)FRONTIERS CAVS]

The purpose of this study is to evaluate the efficacy, safety and tolerability of pelacarsen (TQJ230) administered subcutaneously once monthly compared to placebo in slowing the progression of calcific aortic valve stenosis.

开始日期2024-03-07

申办/合作机构

Novartis Pharmaceuticals Corp.

NCT05900141 / Recruiting临床3期

A Single Arm, Open Label Extension (OLE), Multicenter Study to Evaluate Long-term Safety and Tolerability of Pelacarsen (TQJ230) in Patients With Cardiovascular Disease Who Have Successfully Completed the Apheresis Parent Study.

This non-randomized, open-label extension study will provide post-trial access to pelacarsen (TQJ230) to participants in Germany with hyperlipoproteinemia(a) and established cardiovascular disease who have successfully completed the double-blind parent study (CTQJ230A12302).

开始日期2023-09-29

申办/合作机构

Novartis Pharmaceuticals Corp.

100 项与 Pelacarsen 相关的临床结果

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100 项与 Pelacarsen 相关的转化医学

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100 项与 Pelacarsen 相关的专利（医药）

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项与 Pelacarsen 相关的文献（医药）

2024-08-01·Journal of Clinical Lipidology

Early health technology assessment of gene silencing therapies for lowering lipoprotein(a) in the secondary prevention of coronary heart disease

Article

作者: Norman, Richard ; Watts, Gerald F ; Burvill, Angela ; Ademi, Zanfina ; Watts, Gerald F.

BACKGROUND:

Olpasiran and pelacarsen are gene-silencing therapies that lower lipoprotein(a). Cardiovascular outcome trials are ongoing. Mendelian randomisation studies estimated clinical benefits from lipoprotein(a) lowering.

OBJECTIVE:

Our study estimated prices at which olpasiran and pelacarsen, in addition to standard-of-care, would be deemed cost-effective in reducing risk of recurrent coronary heart disease (CHD) events in the Australian healthcare system.

METHODS:

We developed a decision tree and lifetime Markov model. For olpasiran, participants had CHD and lipoprotein(a) 260 nmol/L at baseline and three-monthly injections, profiled on OCEAN(a) Outcomes trial (NCT05581303). Baseline risks of CHD, costs and utilities were obtained from published sources. Clinical trial data were used to derive reductions in lipoprotein(a) from treatment. Mendelian randomisation study data were used to estimate downstream clinical benefits. Annual discounting was 5 %. For pelacarsen, participants had CHD and lipoprotein(a) 226 nmol/L at baseline and one- monthly injections, profiled on Lp(a) HORIZON (NCT04023552) trial.

RESULTS:

Olpasiran in addition to standard-of-care saved 0.87 discounted quality-adjusted life years (QALYs) per person. Olpasiran in addition to standard-of-care would be cost- effective at annual prices of AU$1867 (AU$467 per dose) at threshold AU$28,000 per QALY. Pelacarsen would be cost-effective at annual prices of AU$984 (AU$82 per dose). For ICER threshold AU$50,000 per QALY, olpasiran and pelacarsen would be cost-effective at annual prices AU$4207 and AU$2464 respectively.

CONCLUSION:

This early health technology assessment model used inclusion criteria from clinical trials. Olpasiran and pelacarsen would be cost-effective if annual treatment prices were AU$1867 and AU$984 respectively, from the Australian healthcare perspective.

2024-03-01·Indian heart journal

Traditional and novel non-statin lipid-lowering drugs

Review

作者: Jain, Peeyush

Non-statin drugs find utility in the management of dyslipidaemia in mixed dyslipidaemia, patients with statin intolerance, and when guidelines directed low-density lipoprotein cholesterol (LDL-C) target cannot be achieved despite maximally tolerated statin. The most definite indication of fenofibrate monotherapy is fasting serum triglyceride >500 mg/dl to reduce the risk of acute pancreatitis It offers a modest reduction in cardiovascular events. The statin-ezetimibe combination is commonly used for lipid lowering particularly after ACS. Fish oils reduce serum triglycerides by about 25 %. EPA (and not DHA) seems to have cardioprotective effects. Despite cardiovascular outcome benefits, bile-exchange resins have limited use due to poor tolerance. Bempedoic acid added to maximally tolerated statin therapy is approved to lower LDL-C in adults with primary hypercholesterolemia or mixed dyslipidaemias, HeFH, in patients with ASCVD who require additional lowering of LDL-C, and in patients who are statin-intolerant. Inclisiran is a long-acting double-stranded small interfering RNA (siRNA) that inhibits the transcription of PCSK-9 leading to a decrease in PCSK9 generation in hepatocytes and an increase in LDL receptor expression in the liver cell membrane leading to about 50 % reduction in serum LDL-C levels. Lomitapide lowers plasma levels of all ApoB-containing lipoproteins, including VLDL, LDL, and chylomicrons by inhibiting the enzyme microsomal triglyceride transfer protein (MTP) and approved for the treatment of adult patients with homozygous familial hypercholesterolemia (HoFH). Close monitoring for hepatotoxicity is required. Mipomersen is a single-stranded synthetic antisense oligonucleotide (ASO) that affects the production and secretion of apoB-containing lipoproteins with demonstrated efficacy in both homozygous and heterozygous FH patients. It is approved for restricted use due to risk of hepatotoxicity. Pelacarsen is an antisense oligonucleotide that reduces the production of apo(a) in the liver.

2024-03-01·Indian heart journal

Lipoprotein a - Lp(a)

Review

作者: Ghose, Tapan

Lp(a) is a genetically determined, heritable, independent and causal risk factor for ASCVD. About 1 in 5 people worldwide have elevated Lp(a) (>50 mg/dL or >125 nmol/L) whereas in Indians it is 25 %. Epidemiological, genome-wide association and mendelian randomization studies have demonstrated an association between elevated Lp(a) levels and increased incidence of myocardial infarction, aortic valve stenosis, ischemic stroke, heart failure, CV and all-cause mortality. The increased Lp(a)-mediated CV risk is mediated by pro-inflammatory, pro-thrombotic and pro-atherogenic processes, leading to progression of atherosclerosis and increased risk of thrombosis. Lp(a) level reaches peak by 5 years of age and remains stable over time. Levels are not much influenced by dietary and environmental factors but it can vary in certain clinical situations like thyroid diseases, chronic kidney disease, inflammation and sepsis. It should be measured at least once in life time. Cascade testing for high Lp(a) is recommended in the settings of FH, family history of (very) high Lp(a), and personal or family history of ASCVD. In the absence of specific Lp(a)-lowering therapies, comprehensive risk factor management is recommended as per guidelines for individuals with elevated Lp(a). PCSK9 inhibitors and Inclisiran reduce Lp(a) by 25%. Pelacarsen is an antisense oligonucleotide and is found to reduce Lp(a) by 80%. In a recent Indian study of 1,021 CAD patients, presence of elevated Lp(a) (>50 mg/dL) correlated with severe angiographic disease. 37% of ACS patients exhibited elevated Lp(a) and it was higher in young CAD patients with FH (43%).

项与 Pelacarsen 相关的新闻（医药）

2024-11-25

·药研网

诺华上调每年销售预期至6%，信心来源于这几款药物

11月21日，诺华上调了中期销售预期。受已上市药物的强劲势头和即将上市新药的推动，此前对 2023-2028 年销售复合年增长率为+5% 的指引上调至 +6%，预计到2028年的年增长率为6%,到2027 年核心营业利润率将增长超40%！目前，诺华研发管线中有 30 多种药物，大多数药物预计将在 2030 年或之后获得美国独占权。诺华业务发展势头强劲，8种已上市药物，有望带来30至80亿美元的年销售额峰值，具体来看，尽管其主要心脏病药物 Entresto 将于明年在美国失去专利保护，但诺华提高了5种主要药物的年度销售峰值预期：免疫学适应症Cosenty 预测销售峰值80亿美元适应症：用于治疗中重度斑块型银屑病、强直性脊柱炎及其他免疫介导的疾病。作用机制：靶向白介素-17A(IL-17A)的全人源单克隆抗体。乳腺癌Kisqali 预测销售峰值80亿美元适应症：用于治疗HR+/HER2-的局部晚期或转移性乳腺癌患者。作用机制：Kisqali是一种CDK4/6抑制剂，通过干扰细胞周期来抑制肿瘤细胞的生长和增殖。多发性硬化症 Kesimpta 预测销售峰值60亿美元适应症：用于治疗多发性硬化症（MS）。作用机制：是一种靶向CD20的单克隆抗体。前列腺癌 Pluvicto 预测销售峰值50亿美元适应症：用于PSMA阳性的转移性去势抵抗性前列腺癌。作用机制：Pluvicto是一款将靶向PSMA的小分子化合物与放射性同位素（177Lu）连接在一起的放射性配体疗法。胆固醇治疗 Leqvio 预测销售峰值40亿美元适应症：用于治疗成人高胆固醇血症（杂合子家族性和非家族性）及混合性血脂异常。作用机制：Leqvio是一款“first-in-class”、靶向PCSK9蛋白mRNA的RNAi疗法。这是诺华今年增长最快的药物，Q3销售值达到8亿美元，增幅高达118%。另外两款用于治疗慢性髓性白血病（CML）的Scemblix（Asciminib）和PNH药物Fabhalta（iptacopan），预测峰值均为30亿美元。诺华表示，近期将推出另外4 种重磅炸弹药物（口服 BTK 抑制剂瑞布替尼、与 Akcea 合作的 Lp(a) 药物pelacarsen、基因治疗药物OAV101 IT和抗BAFF-R抗体ianalumab），年销售额可能达到数10亿美元。 BTK抑制剂Remibrutinib瑞布替尼：正在开展荨麻疹的临床，明年提交用于治疗慢性自发性荨麻疹 (CSU) 的申请，此外其用于多发性硬化症和慢性诱导性荨麻疹 (CIndU) 的后续适应症预计将于2026 年公布。 Pelacarsen：是一种靶向Apo(a) mRNA的ASO药物，III期试验（NCT04023552）正在进行中,主要观察其对心血管疾病患者的主要心血管事件的影响,有望成为首个靶向强效降低脂蛋白(a)并带来心血管获益的药物。基因治疗药物OAV101（Zolgensma）：美国FDA批准的第一款治疗脊髓性肌萎缩症（SMA）的基因疗法，于2022年进入中国进行临床试验。抗BAFF-R抗体Ianalumab: 主要用于治疗自身免疫疾病（SLE、干燥综合症等)，预计2025年公布在治疗干燥综合征和作为免疫性血小板减少症 (ITP) 的二线治疗方面的结果，一线 ITP 研究将于2026 年产生数据。此外，诺华仍面临着再生障碍性贫血药物 Promacta、白血病治疗药物 Tasigna 以及 Entresto 明年可能出现的仿制药竞争压力。首席执行官Vas Narasimhan在接受采访时表示，“将考虑潜在的并购，继续追求成为未来几年美国五大制药公司之一的目标，努力扩大在美国的业务。截至第二季度，其销售额在美国排名第十。” 近年来，诺华开始对其业务组合进行重大调整，分拆了眼科专家爱尔康和仿制药生产商山德士，同时放弃了在竞争对手罗氏以及与英国GSK成立的消费者健康合资企业中的股份，完成了向创新药公司的转型。此外，诺华还进行了几项收购，今年收购了中国的信瑞诺医药、德国的 MorphoSys 和美国的 Mariana Oncology。此外，诺华经过重组并改革了组织结构并进行了裁员。总结诺华销售情况及利润率每年都在稳健提升，2018-2022年该公司销售额复合年增长率为7%，核心营业收入复合年增长率为14%，2022年核心营业收入增长为35%。而诺华上调中期目标的信心，主要来源于已上市产品的销售增长动力，以及在研管线候选药物的巨大潜力。 End 声明：本公众号所有发文章(包括原创及转载文章)系出于传递更多信息之目的，且注明来源和作者。本公众号欢迎分享朋友圈或大群，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载/商务/投稿 | 联系微信15618157102（sum_Gmi）商务合作稿件征集点击了解详情往期回顾 1 10月 | 20家生物医药裁员汇总 2 国产首款四价HPV疫苗拟纳入优先审评 3 3. 7亿美元!今年规模最大的生物医药融资诞生

临床3期信使RNA核酸药物放射疗法基因疗法

2024-11-20

·奇点网

JAMA双重磅：超毒脂蛋白克星来了！靶向脂蛋白(a)的两款新药同期公布II期临床数据，降低水平最高可超85%丨临床大发现

*仅供医学专业人士阅读参考最新捷报！近两年，多种降低脂蛋白(a)（Lp[a]）的药物临床试验已经进入白热化阶段。今天，又是谁带着成果上台呢？前日，两款Lp(a)降脂药的II期临床结果同时于《美国医学会杂志》期刊惊艳亮相。一款是Lp(a)的首个口服降脂药muvalaplin[1]，此次II期临床结果显示，相比于安慰剂，12周、每日一次240 mg的muvalaplin治疗可以使Lp(a)水平≥175 nmol/L、具有较高心血管事件风险的患者Lp(a)水平降低达85.8%。另一款是腹部皮下注射的小干扰 RNA（siRNA）药物zerlasiran[2]，相比于安慰剂，以450 mg、每24周两次的给药方式进行治疗36周后，zerlasiran使Lp(a)水平≥125 nmol/L、动脉粥样硬化性心血管疾病患者的Lp(a)平均水平降低85.6%，且药效持久，治疗结束半年内Lp(a)水平稳定较低。两个药物的安全性都可靠。脂蛋白(a)（Lp[a]）已被证明是动脉粥样硬化和主动脉瓣狭窄心血管疾病独立危险因素，高于50mg/dL（125nmol/L）通常被认为属于较高水平，临床上尚无获批药物进行治疗。很多研究喜欢拿Lp(a)和另一个心血管“杀手”进行掰头，也就是低密度脂蛋白（LDL）或者说低密度脂蛋白胆固醇（LDL-C）。结果发现，虽说LDL水平高在人群中更普遍存在，但是Lp(a)的毒性可谓是一节更比六节强，每颗粒Lp(a)是LDL的致动脉粥样硬化性的大约6.6倍[3]。即使求救“神奇海螺”他汀，将LDL-C降至较低水平也无法完全抵消由Lp(a)增加的疾病风险[4]。还有件棘手的事情，Lp(a)这东西难以通过改变生活饮食习惯来改变，几乎是刻在DNA里的（80%-90%由基因决定），于生命早期就已达到一定水平，随后基本保持稳定[5]。这么一比，LDL-C竟好似温柔了些。不能用他汀，不能靠改善饮食，现有的法子行不通，那就另寻他路了。 Lp(a)由载脂蛋白(a) (apo[a])与含有apo B100蛋白的LDL样颗粒结合通过非共价结合形成。针对它的结构和诱导心血管疾病的病理机制，应对之策主要有两个大方向：一是限制apo(a)的合成。apo(a)由LHA基因编码，在肝脏中合成。对LHA做手脚，可以开发靶向LHA的小干扰 RNA（siRNA）药物，代表有lepodisiran、olpasiran、zerlasiran；或者是靶向LHA的反义寡核苷酸（ASO）药物，代表有pelacarsen。二是限制Lp(a)的组装，代表有muvalaplin。不同于上述几种药物的注射疗法，muvalaplin是首个口服药，通过阻断apo(a)与apoB结合、抑制Lp(a)形成。脂蛋白（a）以及当前临床药物的主要研发方向[9] 截至目前，lepodisiran的I期临床试验[6]、olpasiran的II期临床试验[7]、pelacarsen的II期临床试验[8]已完成，数据喜人、优势不同。今天为大家献上的是zerlasiran和muvalaplin的最新临床结果。澳大利亚莫纳什大学维多利亚心脏研究所的Stephen J. Nicholls团队，领衔完成了Lp(a)口服降脂药muvalaplin的II期临床试验。试验共招募233名来自多个国家Lp(a)水平≥175 nmol/L、年龄≥40岁的患者，中位年龄为66岁，33%为女性。患者的心血管事件风险高，患有冠状动脉疾病或缺血性卒中、外周动脉疾病、2型糖尿病、家族性高胆固醇血症。患者被随机分配至四个治疗组，包括三个不同剂量的muvalaplin治疗组和一个安慰剂对照组，每天口服一次药物。 12周治疗后，muvalaplin在所有剂量组中均显著降低了Lp(a)浓度，且呈现明显的剂量依赖性，而安慰剂组未观察到明显变化。与安慰剂相比，使用完整Lp(a)测定法时，10 mg/天、60 mg/天、240 mg/天剂量的muvalaplin治疗分别使Lp(a)水平降低47.6%、81.7%、85.8%；使用基于apo(a)的测定法时，分别降低40.4%、70.0%、68.9%。次要终点中，完整Lp(a)测定法显示，接受60 mg/天或240 mg/天剂量治疗的患者里，超过95%的人在12周内将Lp(a)水平降低至125 nmol/L以下。另外，接受60 mg/天或240 mg/天剂量治疗的患者LDL-C水平在治疗12周后分别降低16%、21.3%，任何剂量均未观察到高超敏C反应蛋白（hs-CRP）的变化，也没有安全性或耐受性问题。 zerlasiran的II期临床试验由美国克利夫兰医学中心的Steven E. Nissen团队完成。试验共招募178名Lp(a)水平≥125 nmol/L、年龄在18-80岁之间的患者，均患有动脉粥样硬化性心血管疾病。患者的中位年龄63.7岁，女性占25.8%，被随机分配至五个治疗组，包括两个安慰剂组和三个不同剂量的zerlasiran治疗组，均通过腹部皮下注射给药。 36周治疗后，将两个安慰剂组的数据进行合并，发现zerlasiran各治疗组均可显著降低Lp(a）水平。相比于合并安慰剂组，接受450 mg、每24周两次治疗的患者Lp(a)平均水平降低85.6%、中位水平降低94.5%；接受300 mg每16周三次治疗的患者Lp(a)平均水平降低82.8%，中位水平降低96.4%；接受300mg、每24周两次治疗的患者Lp(a)水平降低81.3%、中位水平降低90.0%。每24周450 mg、每16周300 mg、每24周300 mg的zerlasiran治疗，还可以使患者的LDL-C水平在为期36周的疗程后平均降低25.1%、31.9%、29.7%。研究者们对患者进行后续随访。结果显示，zerlasiran在不同剂量组中均表现出较长的持续疗效，首次给药后48周时患者Lp(a)水平仍相较于合并安慰剂组降低78.7%-83.0%，60周时降低幅度可维持在71.8%-79.2%。 zerlasiran治疗最常见的不良事件是轻度注射部位反应，给药后第一天有2.3%-7.1%的参与者反映轻度疼痛，无治疗相关严重不良反应。新的风暴已经出现，怎么能够停滞不前？Lp(a)的恶行被陆续揭露，靶向Lp(a)的降脂药走向临床也指日可待了。参考文献： [1]Nicholls, S. J., Ni, W., Rhodes, G. M., Nissen, S. E., Navar, A. M., Michael, L. F., Haupt, A., & Krege, J. H. (2024). Oral Muvalaplin for Lowering of Lipoprotein(a): A Randomized Clinical Trial. JAMA, 10.1001/jama.2024.24017. Advance online publication. https://doi.org/10.1001/jama.2024.24017 [2]Nissen, S. E., Wang, Q., Nicholls, S. J., Navar, A. M., Ray, K. K., Schwartz, G. G., Szarek, M., Stroes, E. S. G., Troquay, R., Dorresteijn, J. A. N., Fok, H., Rider, D. A., Romano, S., Wolski, K., & Rambaran, C. (2024). Zerlasiran-A Small-Interfering RNA Targeting Lipoprotein(a): A Phase 2 Randomized Clinical Trial. JAMA, 10.1001/jama.2024.21957. Advance online publication. https://doi.org/10.1001/jama.2024.21957 [3]Björnson E, Adiels M, Taskinen M-R, et al. Lipoprotein(a) is markedly more atherogenic than LDL: an apolipoprotein B-based genetic analysis. J Am Coll Cardiol. 2024;83:385-395. [4]Harpreet S. Bhatia, Simon Wandel, Peter Willeit, et al. Independence of Lipoprotein(a) and Low-Density Lipoprotein Cholesterol–Mediated Cardiovascular Risk: A Participant-Level Meta-Analysis. Circulation. DOI: 10.1161/CIRCULATIONAHA.124.069556. [5]Wilson DP, Jacobson TA, Jones PH, et al. Use of lipoprotein(a) in clinical practice: a biomarker whose time has come. A scientific statement from the National Lipid Association. J Clin Lipidol.2022;16:e77–e95. [6]Nissen SE, Linnebjerg H, Shen X, et al. Lepodisiran, an Extended-Duration Short Interfering RNA Targeting Lipoprotein(a): A Randomized Dose-Ascending Clinical Trial. JAMA. 2023;330(21):2075–2083. doi:10.1001/jama.2023.21835 [7]O'Donoghue, M. L., Rosenson, R. S., Gencer, B., López, J. A. G., Lepor, N. E., Baum, S. J., Stout, E., Gaudet, D., Knusel, B., Kuder, J. F., Ran, X., Murphy, S. A., Wang, H., Wu, Y., Kassahun, H., Sabatine, M. S., & OCEAN(a)-DOSE Trial Investigators (2022). Small Interfering RNA to Reduce Lipoprotein(a) in Cardiovascular Disease. The New England journal of medicine, 387(20), 1855–1864. https://doi.org/10.1056/NEJMoa2211023 [8]Tsimikas S, Karwatowska-Prokopczuk E, Gouni-Berthold I, et al. Lipoprotein(a) reduction in persons with cardiovascular disease. N Engl J Med 2020;382:244-255. [9]Nicholls SJ, Nissen SE, Fleming C, et al. Muvalaplin, an Oral Small Molecule Inhibitor of Lipoprotein(a) Formation: A Randomized Clinical Trial. JAMA. 2023;330(11):1042–1053. doi:10.1001/jama.2023.16503 本文作者 | 张艾迪

siRNA临床结果寡核苷酸临床2期

2024-11-19

·echemi

Eli Lilly's New Drug Trial Fails, Cholesterol Reduction Only 70%

At a medical meeting on Monday, Eli Lilly presented data from a study showing that its experimental drug muvalaplin significantly reduced levels of inherited high cholesterol in a mid-stage trial. Researchers reported at an American Heart Association meeting in Chicago that the drug muvalaplin was able to reduce lipoprotein (a) or Lp(a) levels by 70 percent with a traditional blood test and by nearly 86 percent with a more specific test developed by the company. Lilly's drug is currently the only oral treatment, while several injectable therapies are currently being tested to treat high lipoprotein (a), a risk factor for heart disease in one in five people worldwide. Unlike low-density lipoprotein (LDL), this so-called bad cholesterol can be treated with diet and statins, while Lp(a) currently has no approved treatment and few people are aware they have it. Elevated lipoprotein (a) significantly increases the risk of heart attack, stroke, aortic stenosis, and peripheral artery disease (fatty plaque buildup in the arteries), especially in people of African and South Asian descent. The trial compared the effects of three daily doses of muvalaplin (10,60 and 240 mg) with placebo in 233 adults with high levels of Lp(a). The researchers used a traditional blood test and a new method to measure intact Lp(a) particle levels in the blood to test for Lp(a) levels. Compared with placebo, muvalaplin reduced Lp(a) by 47.6% at the 10 mg dose, 81.7% at the 60 mg dose, and 85.8% at the 240 mg dose. Compared with traditional methods, the results were reduced by 40.4%, 70.0% and 68.9%, respectively. The rates of adverse events were similar in the muvalaplin and placebo groups. Ruth Gimeno, Eli Lilly's group vice president for diabetes and metabolic research, said the company is considering moving the drug into late-stage trials. "We need to have discussions with regulators, but we're very excited about it," she said in an interview. She stressed that although the drug reduced cardiovascular risk factors, a large number of trials are still needed to prove that lowering lipoprotein (a) levels actually reduces heart attacks and other adverse cardiovascular events. At the same meeting, London-based Silence Therapeutics reported results from a 60-week phase II trial of zerlasiran in 180 patients. zerlasiran inhibits LPA gene activity through short interfering RNA (siRNA) technology, resulting in high levels of Lp(a). Injections of 300 mg or 450 mg of zerlasiran every 16 or 24 weeks resulted in an 80 to 85 percent reduction in Lp(A) over a follow-up period of 36 to 60 weeks with no significant safety concerns. Dr. Curtis Rambaran, the chief medical officer of Silence Therapeutics, saidin an interview, "We observed significant reductions to the same extent as in the Phase 1 trial." Silence Therapeutics plans to test a 300-milligram dose in a late-stage trial that will begin in the middle of next year, he noted. The results of both studies were published in the Journal of the American Medical Association. Other injectable Lp(a) treatments in clinical trials include Eli Lilly's lepodisiran, Amgen's olpasiran and Novartis' pelacarsen.

临床结果AHA会议临床1期临床2期

100 项与 Pelacarsen 相关的药物交易

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KEGG	Wiki	ATC	Drug Bank
-	-	-	DB16284

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适应症	最高研发状态	国家/地区	公司	日期
动脉粥样硬化	临床3期	美国	Novartis Pharmaceuticals Corp.	2024-04-24
脂蛋白(a)高脂蛋白血症	临床3期	德国	Novartis Pharmaceuticals Corp.	2022-08-19
心血管疾病	临床3期	美国	Novartis Pharma AG	2019-12-12
心血管疾病	临床3期	美国	Novartis Pharma Stein AG	2019-12-12
心血管疾病	临床3期	美国	诺华（中国）生物医学研究有限公司	2019-12-12
心血管疾病	临床3期	中国	Novartis Pharmaceuticals Corp.	2019-12-12
心血管疾病	临床3期	日本	Novartis Pharmaceuticals Corp.	2019-12-12
心血管疾病	临床3期	日本	诺华（中国）生物医学研究有限公司	2019-12-12
心血管疾病	临床3期	日本	Novartis Pharma Stein AG	2019-12-12
心血管疾病	临床3期	日本	Novartis Pharma AG	2019-12-12

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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


NCT03070782 (CTgov)	临床2期	心血管疾病 \| 脂蛋白(a)高脂蛋白血症	286	ISIS 681257 (Cohort A: ISIS 681257: 20 mg Q4W)	蓋壓壓鹽鹹網繭窪淵顧(夢鏇製築鹽鏇齋網膚醖) = 衊範範觸襯顧網範憲網觸顧製艱網膚衊襯觸網 (憲廠鑰鏇築網獵鹽獵鏇, 夢蓋蓋獵膚壓願顧壓夢 ~ 壓構範選築鏇構衊製鹽) 更多	-	2020-10-30
				ISIS 681257 (Cohort B: ISIS 681257: 40 mg Q4W)	蓋壓壓鹽鹹網繭窪淵顧(夢鏇製築鹽鏇齋網膚醖) = 衊鹹襯願觸壓襯鬱糧鑰觸顧製艱網膚衊襯觸網 (憲廠鑰鏇築網獵鹽獵鏇, 壓製鹽繭蓋膚觸膚鏇糧 ~ 廠餘襯獵範網夢顧簾廠) 更多