Double-Blind, Placebo-Controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Plozasiran in Adults With Severe Hypertriglyceridemia at High Risk of Acute Pancreatitis (SHASTA-5 Study)

This study will evaluate the efficacy and safety of plozasiran in approximately 140 adult participants with severe hypertriglyceridemia (SHTG) and history of at least two prior acute pancreatitis (AP) events not attributed to other etiologies, with at least one occurring within the last 12 months prior to screening. Eligible participants will be randomly assigned in a double-blind manner to either receive plozasiran 25 mg by subcutaneous (SC) injection every three months (Q3M) or matching placebo. Enrolled participants will be counseled to remain on the specified low-fat diet and background medications throughout the study. Following completion of the double-blind treatment period, or if the participant has a positively adjudicated AP event (whichever occurs first), participants will transition to the 12-month Open-Label Extension (OLE) treatment period receiving plozasiran 25 mg by SC injection Q3M.

开始日期2025-04-01

申办/合作机构

Arrowhead Pharmaceuticals, Inc.

NCT06822790

/ Not yet recruiting临床3期

A Phase 3 Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Plozasiran in Adults With Hypertriglyceridemia (SHASTA-10 Study)

This is an open-label study to be conducted in adults with hypertriglyceridemia (HTG) and severe hypertriglyceridemia (SHTG). Each participant must have completed all required visits per protocol in the parent study AROAPOC3-3001 (NCT05089084), AROAPOC3-3003 (NCT06347003), AROAPOC3-3004 (NCT06347016) or AROAPOC3-3009 (NCT06347133). All eligible participants will receive plozasiran administered subcutaneously (SC) approximately every 3 months for 24 months. Participants will be counseled to remain on the specified low-fat diet throughout the study in accordance with local standard of care.

开始日期2025-03-01

申办/合作机构

Arrowhead Pharmaceuticals, Inc.

CTR20243478

/ Recruiting临床3期

一项在重度高甘油三酯血症成人患者中评价Plozasiran的疗效和安全性的双盲、安慰剂对照、III期研究（SHASTA-3研究）。

主要目的：证明Plozasiran在降低空腹血清TG水平方面的疗效。次要目的：达到TG水平降低目标的受试者比例；证明 Plozasiran在降低残余胆固醇（极低密度脂蛋白胆固醇[VLDL-C]）和非高密度脂蛋白胆固醇（非 HDL-C）方面的疗效；评价Plozasiran在裁定的腹部临床事件发生率方面的疗效。安全性目的：评价Plozasiran的安全性和耐受性；评价裁定的主要不良心血管事件发生率；评价试验用药品的免疫原性；评价Plozasiran对肝脏脂肪含量的影响。探索性目的：证明Plozasiran在血脂相关指标变化方面的疗效；评价Plozasiran对血液中心血管代谢疾病相关炎症和探索性血清生物标志物的影响。

开始日期2024-10-30

申办/合作机构

Ajinomoto Althea, Inc.

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项与普乐司兰钠相关的文献（医药）

2025-12-01·Current Atherosclerosis Reports

Novel Therapeutics for Familial Chylomicronemia Syndrome

Review

作者: Izar, Maria Cristina ; Fonseca, Francisco Antonio Helfenstein

PURPOSE OF REVIEW:

This review discusses new treatment approaches for familial chylomicronemia syndrome (FCS), a rare disorder affecting triglyceride metabolism. The focus is on antisense oligonucleotides (ASO) and small-interfering RNA (siRNA) therapies targeting APOC3 and angiopoietin-like protein 3 (ANGPTL3).

RECENT FINDINGS:

Volanesorsen, an ASO targeting APOC3, has shown effectiveness in managing FCS, multifactorial chylomicronemia, and familial partial lipodystrophy, but its use is limited by thrombocytopenia. Emerging therapies, Olezarsen (ASO anti-APOC3) and Plozasiran (siRNA anti-APOC3), both conjugated with GalNAc, show promise in reducing acute pancreatitis risk without platelet concerns. ANGPTL3 inhibition requires residual lipoprotein lipase (LPL) activity, with only siRNA-based therapies-zodasiran and solbinsiran-under investigation. Suppressing APOC3 expression and targeting ANGPTL3 via siRNA offer significant potential, but long-term studies are needed to confirm their efficacy and safety. Future research may explore gene-editing strategies using lipid nanoparticle-based CRISPR-Cas9 delivery for more durable treatment outcomes.

2025-12-01·Current Atherosclerosis Reports

Highlights of Cardiovascular Disease Prevention Studies Presented at the 2024 American Heart Association Scientific Sessions

Review

作者: Khoja, Adeel ; Inam, Maha ; Murthy, Nikitha ; Epstein, Elizabeth ; Virani, Salim S ; Sheikh, Sana ; Vaughan, Elizabeth ; Mehta, Sandeep ; Minhas, Abdul Mannan Khan ; Slipczuk, Leandro ; Junaid, Vashma ; Chai, Zohar ; Hinkamp, Colin ; Hermel, Melody

PURPOSE OF REVIEW:

Focused review highlighting ten select studies presented at the 2024 American Heart Association (AHA) Scientific Sessions.

RECENT FINDING:

Included studies assessed effects of intensive blood pressure control in patients with type 2 diabetes (BPROAD); decision support system for physicians to optimize early lipid lowering therapies after acute coronary syndrome (ZODIAC); efficacy and safety of zerlasiran, a short interfering RNA targeting lipoprotein(a) (ALPACAR); efficacy and safety of muvalaplin an oral disrupter of the assembly of lipoprotein(a) particles (KRAKEN); safety and efficacy of obicetrapib in patients with heterozygous familial hypercholesterolemia (BROOKLYN); efficacy and safety of lerodalcibep, a third generation PCSK9 inhibitor in heterozygous familial hypercholesterolemia subjects (LIBerate-HeFH_OLE); personalized app-based coaching to improve physical activity in patients with HFpEF compared to standard care (MyoMobile); semaglutide to improve cardiovascular outcomes in patients with a history of coronary artery bypass surgery and overweight or obesity (the SELECT trial); efficacy and safety of plozasiran in adults with genetically or clinically defined familial chylomicronemia syndrome at high risk of acute pancreatitis (PALISADE); and transcriptomic signatures and predictors of evolocumab added to maximum statin therapy based on intra-coronary plaque characteristics (YELLOW III). Research presented at the 2024 AHA Scientific Sessions emphasized innovative strategies in cardiovascular disease prevention and management.

2025-06-01·METABOLISM-CLINICAL AND EXPERIMENTAL

Apolipoprotein C-III inhibitors for the treatment of hypertriglyceridemia: a meta-analysis of randomized controlled trials

Review

作者: Prizão, Vitória Martins ; de Athayde de Hollanda Morais, Beatriz Austregélio ; Mendes, Beatriz Ximenes ; Romaniello, Gabriela ; Defante, Maria Luiza Rodrigues ; Martins, Otávio Cosendey ; de Moura de Souza, Mariana

INTRODUCTION:

Hypertriglyceridemia is related to atherosclerotic cardiovascular risk and pancreatitis risk. The efficacy and safety of apolipoprotein C-III (APOC-III) inhibitors remains unclear.

AIM:

To investigate the effects of APOC-III inhibitors on hypertriglyceridemia and its complications.

METHODS:

We systematically searched PubMed, Embase, and Cochrane Central databases from inception to May 2024 for randomized controlled trials (RCTs) comparing APOC-III inhibitors to placebo in patients with hypertriglyceridemia. We pooled percentage standardized mean difference (SMD) changes and risk ratio (RR) for continuous and binary outcomes, respectively, with 95 % confidence interval (CI). Subgroup analyses were performed with APOC-III inhibitors drugs doses (Olezarsen, Volanesorsen and Plozasiran), and primary and secondary hypertriglyceridemia.

RESULTS:

10 RCTs with 1204 participants were included, of which 46 % were men. APOC-III inhibitors significantly reduced triglycerides (TG) (SMD: -60.56 %; 95 % CI -68.94 to -52.18; p < 0.00001), APOC-III (SMD: -75.44 %; 95 % CI -80.81 to -70.07; p < 0.00001) and non-HDL-c (SMD: -27.49 %; 95 % CI -34.16 to -20.82; p < 0.00001) levels. Consistent results were found for all subgroup analyses. APOC-III inhibitors were capable to normalize TG levels in patients with severe hypertriglyceridemia (RR: 7.92; 95 % CI 4.12 to 15.23; p < 0.00001). There was a significant increase in HDL-c (SMD: 43.92 %; 95 % CI 37.27 to 50.57; p < 0.00001) and LDL-c (SMD: 33.05 %; 95 % CI 9.08 to 57.01; p = 0.007) levels. There was a significant relative risk reduction in acute pancreatitis in the APOC-III inhibitors group (RR 0.17; 95 % CI 0.05 to 0.53; p = 0.007). Adverse events were similar in both groups.

CONCLUSION:

APOC-III inhibitors improve TG levels and other lipid panel parameters, as well as reduce episodes of acute pancreatitis in patients with primary and secondary hypertriglyceridemia.

156

项与普乐司兰钠相关的新闻（医药）

2025-03-28

·FiercePharma

Ionis, Arrowhead, Amarin sponsor new triglyceride group to educate physicians

The National Triglyceride Alliance plans to work with the American Society for Preventive Cardiology and other organizations with shared objectives to “provide evidence-based education, expert-driven resources and clinical guidance to healthcare providers.” Ionis Pharmaceuticals and Arrowhead Pharmaceuticals have found a common cause amid their emerging rivalry. While preparing to go head to head in a rare disease market, the companies have signed up to be the top two sponsors of the newly launched National Triglyceride Alliance (NTA). The biotechs are both targeting conditions characterized by elevated triglyceride levels in the blood. Ionis won FDA approval for its antisense oligonucleotide Tryngolza late last year, and the FDA is reviewing Arrowhead’s application for approval of RNAi therapy plozasiran in the same indication. Both companies are also working to bring their molecules to market in another indication in which elevated triglycerides drive disease.While Ionis and Arrowhead are in direct competition, that rivalry gives the companies shared interest. Enter the NTA. The working group will “offer educational programs, clinical resources and ongoing initiatives aimed at improving understanding and treatment strategies for high triglycerides,” according to Friday’s launch announcement.Ionis is a founding sponsor of the NTA, Arrowhead is a national sponsor and Amarin, which sells Vascepa for use in certain adults with elevated triglycerides, has come on board as a local sponsor. The NTA lists the founder, national and local sponsorship levels at $250,000, $50,000 and $15,000, respectively. The NTA plans to work with the American Society for Preventive Cardiology and other organizations with shared objectives to “provide evidence-based education, expert-driven resources and clinical guidance to healthcare providers.” Through such initiatives, the NTA is aiming to give healthcare professionals the education and tools to effectively manage patients with high triglyceride levels. Ionis and Arrowhead are also working independently to lay the foundations for sales growth. With FDA approval in the bag, Ionis recently kicked off Tryngolza campaigns aimed at patients and healthcare professionals. Arrowhead, meanwhile, is yet to win approval but began priming the market last year with “We’ll Get There Soon,” a push to spread the word about the importance of treating patients with triglycerides above 500 mg/dL.

上市批准

2025-03-22

·药明康德

强效降脂近90%的RNAi疗法；展现“史无前例”疗效的创新偶联疗法…… | TIDES周报

▎药明康德内容团队编辑近期，全球多肽和寡核苷酸（TIDES）领域迎来系列进展。美国FDA已批准Alnylam Pharmaceuticals公司为其RNA干扰（RNAi）疗法Amvuttra（vutrisiran）递交的补充新药申请，用于治疗野生型或遗传性伴有心肌病的转甲状腺素蛋白淀粉样变性（ATTR-CM）成人患者。维亚臻生物宣布，其用于治疗家族性乳糜微粒血症综合征（FCS）的RNAi疗法VSA001的3期临床试验达到主要疗效终点和所有关键次要终点。Avidity Biosciences公司和Dyne Therapeutics公司在近日举办的2025年肌肉萎缩症协会（MDA）临床与科学会议中各披露了1款用于治疗杜氏肌营养不良（DMD）的偶联疗法的积极早期数据。欢迎读者长按/扫描以下二维码，申请获取含有完整表格整理的《2025年3月第3期TIDES疗法进展盘点》。 ▲长按识别上方二维码，申请获取《2025年3月第3期TIDES疗法进展盘点》 Vutrisiran：补充新药申请获FDA批准 Alnylam Pharmaceuticals公司宣布，美国FDA已批准该公司为RNAi疗法vutrisiran递交的补充新药申请，用于治疗野生型或遗传性ATTR-CM成人患者，以减少心血管死亡、心血管住院和紧急心力衰竭就诊。Vutrisiran是一款皮下给药的RNAi疗法，它通过靶向和沉默特定的mRNA，阻断野生型和突变转甲状腺素蛋白的产生。Vutrisiran基于Alnylam的增强稳定化学（ESC）-GalNAc偶联递送平台设计，增加了效力和代谢稳定性，可允许不频繁的皮下注射。此次获批是基于3期临床试验HELIOS-B的结果，结果显示，与安慰剂相比，皮下注射vutrisiran显著降低了ATTR-CM患者因任何原因死亡或出现复发性心血管事件的风险，同时维持了患者的身体功能和生活质量。2022年6月，美国FDA批准vutrisiran上市，用于遗传性转甲状腺素蛋白淀粉样变性多发性神经病（hATTR-PN）。新闻稿指出，vutrisiran是首个获得FDA批准用于治疗成人ATTR-CM及hATTR-PN的治疗方法。 VSA001：公布3期临床试验数据维亚臻生物宣布，其旗下VSA001用于治疗中国家族性乳糜微粒血症综合征患者的3期临床试验获得积极顶线数据，成功达到主要疗效终点和所有关键次要终点。VSA001（plozasiran），之前也称为ARO-APOC3，最初由Arrowhead Pharmaceuticals公司开发，是一种潜在“first-in-class”的RNAi治疗药物，旨在减少载脂蛋白C-III（APOC3）的产生。APOC3是富含甘油三酯的脂蛋白（TRLs）的组成部分，也是甘油三酯代谢的关键调节因子。APOC3通过抑制脂蛋白脂酶对TRLs的分解以及肝脏中肝脏受体对TRLs残余物的摄取，导致血液中的甘油三酯水平升高。VSA001通过降低APOC3水平，从而减少甘油三酯并将脂质调节到正常水平。该研究的主要终点为与安慰剂组相比，第10个月的空腹血清甘油三酯水平相较于基线的中位变化百分比。VSA001在第10个月甘油三酯中位数降幅高达89%，APOC3中位数降幅高达93%，与安慰剂组相比具有统计学差异。除了成功达到研究主要终点外，VSA001还达到所有关键次要研究终点，包括第10个月和第12个月血清空腹甘油三酯相较于基线变化百分比，以及第10个月和第12个月APOC3水平相较于基线变化百分比。此外，VSA001在FCS患者中表现出良好的安全性。 Delpacibart zotadirsen：公布1/2期临床试验的新数据 Avidity Biosciences公司用于治疗DMD的在研疗法delpacibart zotadirsen（del-zota）在1/2期EXPLORE44临床试验中展现出“史无前例”的抗肌萎缩蛋白表达能力。DMD是由于编码抗肌萎缩蛋白的基因产生突变，导致抗肌萎缩蛋白缺失或功能失常，造成患者肌肉功能的终身、渐进性丧失。Del-zota由靶向1型转铁蛋白受体（TfR1）的单克隆抗体和促进编码抗肌萎缩蛋白的mRNA前体产生外显子44跳跃的寡核苷酸偶联而成，TfR1在肌肉组织中高度表达。这款疗法通过改变mRNA剪接过程，产生几乎与正常抗肌萎缩蛋白功能相同的蛋白。靶向TfR1的单克隆抗体可以通过与肌肉细胞表面的受体相结合，增强寡核苷酸的肌肉特异性递送。研究显示，剂量为5 mg/kg和10 mg/kg的del-zota均表现出强有力的骨骼肌递送能力，骨骼肌寡核苷酸水平达到200 nM。外显子44跳跃效率显著增加40%。抗肌萎缩蛋白生成显著增加，平均增加至正常水平的25%，最高达到正常水平的58%。肌酸激酶水平下降至接近正常水平，相比基线减少了超过80%。基于这些积极数据，Avidity决定在未来的临床研究中采用5 mg/kg（每6周一次给药）的剂量方案，并以此支持生物制品许可申请（BLA）的递交。公司计划最迟于2025年年底前向FDA提交BLA申请。 DYNE-251：公布1/2期临床试验的新数据 Dyne Therapeutics公司用于治疗DMD的在研疗法DYNE-251也1/2期DELIVER试验中展现出“史无前例”的持久功能提升和生物标志物改善。DYNE-251由磷酸二氨基甲酰吗啉寡核苷酸（PMO）与靶向TfR1的抗体片段（Fab）偶联而成。该疗法旨在实现靶向肌肉组织递送，促进核内外显子跳跃，从而使肌肉细胞生成截短但功能正常的抗肌萎缩蛋白，目标是停止或逆转疾病的进展。接受20 mg/kg和10 mg/kg剂量治疗的患者在多个关键功能指标上显著改善，包括北极星步行评估（NSAA）、步速95百分位数（SV95C）、10米步行/跑步时间（10-MWR）及从地面起身时间。在生物标志物方面，DYNE-251治疗组患者显示出高水平抗肌萎缩蛋白表达，肌肉含量调整后的平均绝对表达量达8.72%。Dyne Therapeutics已与FDA确认，抗肌萎缩蛋白表达可作为加速批准的替代终点。目前，DYNE-251的注册性扩展队列已完成患者招募，公司预计2025年晚些时候获得数据，并计划于2026年初向FDA递交BLA，寻求加速批准。限于篇幅，本文仅针对部分重要进展做简单介绍。欢迎读者长按/扫描以下二维码，申请获取含有完整表格整理的《2025年3月第3期TIDES疗法进展盘点》。 ▲长按识别上方二维码，申请获取《2025年3月第3期TIDES疗法进展盘点》 ▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放参考资料： [1] Alnylam Announces FDA Approval of AMVUTTRA® (vutrisiran), the First RNAi Therapeutic to Reduce Cardiovascular Death, Hospitalizations and Urgent Heart Failure Visits in Adults with ATTR Amyloidosis with Cardiomyopathy (ATTR-CM). Retrieved March 20, 2025, from https://investors.alnylam.com/press-release?id=28831 [2] 维亚臻宣布VSA001注射液在中国家族性乳糜微粒血症综合征患者的3期临床试验取得积极结果. Retrieved March 17, 2025, from https://www.prnasia.com/story/481821-1.shtml [3] Dyne Therapeutics Announces New Long-Term Clinical Data from Phase 1/2 DELIVER Trial of DYNE-251 in Duchenne Muscular Dystrophy Demonstrating Unprecedented and Sustained Functional Improvement Through 18 Months. Retrieved March 17, 2025, from https://investors.dyne-tx.com/news-releases/news-release-details/dyne-therapeutics-announces-new-long-term-clinical-data-phase-12 [4] Avidity Biosciences Announces Positive Topline Del-zota Data Demonstrating Consistent, Statistically Significant Improvements in Dystrophin, Exon Skipping and Creatine Kinase in People Living with Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping in Phase 1/2 EXPLORE44® Trial. Retrieved March 17, 2025, from https://aviditybiosciences.investorroom.com/2025-03-17-Avidity-Biosciences-Announces-Positive-Topline-Del-zota-Data-Demonstrating-Consistent,-Statistically-Significant-Improvements-in-Dystrophin,-Exon-Skipping-and-Creatine-Kinase-in-People-Living-with-Duchenne-Muscular-Dystrophy-Amenable-to-Exon-44 免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，聚焦全球生物医药健康创新

临床3期上市批准寡核苷酸临床2期临床结果

2025-03-17

·美通社

维亚臻宣布VSA001注射液在中国家族性乳糜微粒血症综合征患者的3期临床试验取得积极结果

中国 FCS 3 期临床试验达到主要终点和全部关键次要终点 VSA001 在第 10 个月甘油三酯中位数降幅高达 89% ， APOC3 中位数降幅高达 93% ，与安慰剂组相比具有统计学差异 VSA001 具有良好的安全性苏州 2025年3月17日 /美通社/ -- 维亚臻生物技术（苏州）有限公司（"维亚臻"）宣布，由维亚臻开展的针对中国家族性乳糜微粒血症综合征（FCS）患者的3期临床试验（CTR20231418/NCT05902598）获得积极顶线数据，成功达到主要疗效终点和所有关键次要终点。维亚臻首席执行官邹晓明博士表示："FCS是一种严重危及患者健康和生命的疾病，在中国尚未有针对该适应症的获批药物。我们很高兴看到VSA001注射液作为维亚臻首个进入3期临床试验的siRNA药物，在针对中国FCS患者的3期临床试验中获得积极结果，展现出显著的疗效和良好的安全性，并与海外已完成的FCS 3期试验结果高度相似。我们向参与此次研究的患者、护理人员、研究人员和团队表示诚挚的谢意，并将继续推动VSA001在中国的研发注册及商业化进程，让中国FCS和高甘油三酯血症患者早日从该药物获益。" 本试验是一项随机、双盲、安慰剂对照、多中心的3期临床试验，共37名FCS患者随机接受每3个月一次，持续12个月的VSA001 25 mg、50 mg或安慰剂皮下注射治疗，旨在中国FCS成人患者中评估VSA001的有效性和安全性。该研究的主要终点为与安慰剂组相比，第10个月的空腹血清甘油三酯水平相较于基线的中位变化百分比。第10个月接受25 mg和50 mg VSA001治疗的FCS患者空腹血清甘油三酯水平相较于基线的降幅分别达到86%和89%。第12个月接受25 mg和50 mg VSA001治疗的FCS患者空腹血清甘油三酯水平相较于基线的降幅分别达到72%和79%。在VSA001 25mg 治疗组中，90%的FCS患者空腹血清甘油三酯水平在第10个月降至500 mg/dL以下。第10个月接受25 mg和50 mg VSA001治疗的FCS患者血清载脂蛋白C3（APOC3）中位值相较于基线降低幅度分别达到93%和92%。除了成功达到研究主要终点外，VSA001还达到所有关键次要研究终点，包括第10个月和第12个月血清空腹甘油三酯相较于基线变化百分比，以及第10个月和第12个月APOC3水平相较于基线变化百分比。 VSA001在FCS患者中表现出良好的安全性。经历治疗相关不良事件（TEAEs）的患者例数在VSA001治疗组及安慰剂组之间相似，而在VSA001治疗组中，严重不良事件的发生率低于安慰剂组。关于家族性乳糜微粒血症综合征（ FCS）家族性乳糜微粒血症综合征（FCS）是一种严重且罕见的疾病，通常由多种单基因突变引起。FCS导致甘油三酯（TG）水平极度升高，通常超过880 mg\/dL，可能导致多种严重的体征和症状，包括急性和潜在致命的胰腺炎、慢性腹痛、糖尿病、肝脂肪变性和认知障碍。目前，针对FCS的治疗选择有限，难以满足患者的医疗需求。关于 VSA001 （Plozasiran） VSA001 （Plozasiran），之前也称为ARO-APOC3，是一种同类首创的RNA干扰（RNAi）治疗药物，旨在减少载脂蛋白C-III（APOC3）的产生。APOC3是富含甘油三酯的脂蛋白（TRLs）的组成部分，也是甘油三酯代谢的关键调节因子。APOC3通过抑制脂蛋白脂酶对TRLs的分解以及肝脏中肝脏受体对TRLs残余物的摄取，导致血液中的甘油三酯水平升高。VSA001通过降低APOC3水平，从而减少甘油三酯并将脂质调节到正常水平。在多项临床研究中，VSA001已在家族性乳糜微粒血症综合征（FCS）、严重高甘油三酯血症（SHTG）和混合性高脂血症患者中显示出甘油三酯和多种致动脉粥样硬化脂蛋白的减少。到目前为止，VSA001总体耐受性良好，报道的治疗相关不良事件通常与研究人群的合并症和基础疾病相关。在临床研究和研究人群中，拟申请上市的25 mg剂量组的最常见治疗相关不良事件包括COVID-19、上呼吸道感染、头痛、2型糖尿病和腹痛。 VSA001正在开展SUMMIT临床计划中的一系列研究，包括针对FCS患者的PALISADE 3期研究、针对SHTG患者的SHASTA研究，以及针对混合性高脂血症患者的MUIR和CAPITAN研究。 VSA001已在FCS适应症获得美国食品药品监督管理局（FDA）的突破性疗法、孤儿药和快速通道认定，欧洲药品管理局（EMA）的孤儿药认定，以及中国国家药品监督管理局（NMPA）的突破性疗法、优先审评认定。目前plozasiran尚未批准用于治疗任何疾病。关于维亚臻维亚臻是一家立足于中国、放眼全球的小核酸药物治疗公司。公司成立于2022年，与国际领先的小核酸药物企业Arrowhead Pharmaceuticals（NASDAQ：ARWR）建立长期战略合作关系。目前公司产品管线拥有四款在同类竞品中处于领先位置、针对心血管代谢及自身免疫疾病领域的临床阶段小核酸药物，旨在打造从研发、生产和商业化的整合式小核酸药物技术平台。更多信息请访问 www.visirna.com，或联系 info@visirna.com

siRNA临床3期临床结果

100 项与普乐司兰钠相关的药物交易

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研发状态

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
家族性乳糜微粒血症综合征	申请上市	美国	Arrowhead Pharmaceuticals, Inc.	2024-11-18
高甘油三酯血症	临床3期	美国	Ajinomoto Althea, Inc.	2024-04-26
高甘油三酯血症	临床3期	美国	维亚臻生物技术（苏州）有限公司	2024-04-26
高甘油三酯血症	临床3期	美国	Arrowhead Pharmaceuticals, Inc.	2024-04-26
高甘油三酯血症	临床3期	阿根廷	Ajinomoto Althea, Inc.	2024-04-26
高甘油三酯血症	临床3期	阿根廷	维亚臻生物技术（苏州）有限公司	2024-04-26
高甘油三酯血症	临床3期	阿根廷	Arrowhead Pharmaceuticals, Inc.	2024-04-26
高甘油三酯血症	临床3期	澳大利亚	Ajinomoto Althea, Inc.	2024-04-26
高甘油三酯血症	临床3期	澳大利亚	Arrowhead Pharmaceuticals, Inc.	2024-04-26
高甘油三酯血症	临床3期	澳大利亚	维亚臻生物技术（苏州）有限公司	2024-04-26

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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


NCT05902598 \| CTR20231418 (PRNewswire) 人工标引	临床3期	家族性乳糜微粒血症综合征	37	VSA001 25 mg	夢齋積範獵壓膚憲遞襯(網鹽夢艱鏇獵齋餘製製) = 網鹽蓋網網願網壓廠淵糧構範願簾選壓廠繭願 (遞範選鑰憲鑰鏇遞願獵 ) 达到更多	积极	2025-03-17
				VSA001 50 mg	夢齋積範獵壓膚憲遞襯(網鹽夢艱鏇獵齋餘製製) = 衊齋壓艱獵廠艱鏇膚壓糧構範願簾選壓廠繭願 (遞範選鑰憲鑰鏇遞願獵 ) 达到更多
NCT04998201 \| NCT04720534 (MUIR, Company_Website) 人工标引	临床2期	高甘油三酯血症 \| 家族性混合型高脂血症	418	Plozasiran (MUIR)	壓鹽網遞膚鹹鏇壓膚範(觸壓繭衊鏇醖鑰構淵顧) = 廠蓋襯繭膚膚醖蓋糧築壓膚選夢積遞襯顧壓製 (醖鬱衊鹹憲積簾憲觸鬱 )	积极	2024-11-18
				Plozasiran (SHASTA-2)	壓鹽網遞膚鹹鏇壓膚範(觸壓繭衊鏇醖鑰構淵顧) = 積獵齋積積壓願築壓窪壓膚選夢積遞襯顧壓製 (醖鬱衊鹹憲積簾憲觸鬱 )
NCT05089084 (PALISADE, Company_Website) 人工标引	临床3期	家族性乳糜微粒血症综合征	75	Plozasiran 25 mg	衊繭餘網襯鹽鏇夢構蓋(願糧選願顧艱夢蓋艱觸) = 顧醖餘膚鹽製糧淵鏇選鏇選壓獵衊窪衊鑰鬱壓 (鬱襯壓網網鹽憲鬱蓋蓋 ) 更多	积极	2024-11-18
				Plozasiran 50 mg	衊繭餘網襯鹽鏇夢構蓋(願糧選願顧艱夢蓋艱觸) = 構醖餘鑰齋鏇窪膚觸選鏇選壓獵衊窪衊鑰鬱壓 (鬱襯壓網網鹽憲鬱蓋蓋 ) 更多
NCT05089084 (PALISADE, BusinessWire) 人工标引	临床3期	家族性乳糜微粒血症综合征	75	Plozasiran 25 mg	鏇範襯膚齋齋簾壓簾鬱(繭願觸憲衊願蓋糧艱築) = 繭顧構積網簾製醖製齋繭鏇繭窪窪窪夢構壓願 (襯餘壓鬱範夢鏇鑰窪廠 ) 更多	积极	2024-09-02
				Plozasiran 50 mg	鏇範襯膚齋齋簾壓簾鬱(繭願觸憲衊願蓋糧艱築) = 廠艱範鑰範糧艱願蓋繭繭鏇繭窪窪窪夢構壓願 (襯餘壓鬱範夢鏇鑰窪廠 ) 更多
NCT05089084 (PALISADE, Corporate Publications) 人工标引	临床3期	家族性乳糜微粒血症综合征	75	Plozasiran 25 mg	壓觸鬱鏇願鏇糧獵壓鹹(構簾鑰淵鏇艱鏇醖選獵) = 齋範簾窪淵鹽壓膚鹹窪衊遞獵齋願願繭襯艱襯 (鹽襯夢鹹鏇鬱鹹蓋觸廠 ) 达到更多	积极	2024-06-03
				Plozasiran 50 mg	壓觸鬱鏇願鏇糧獵壓鹹(構簾鑰淵鏇艱鏇醖選獵) = 築鑰鑰獵衊醖遞鏇窪願衊遞獵齋願願繭襯艱襯 (鹽襯夢鹹鏇鬱鹹蓋觸廠 ) 达到更多
NCT04998201 (MUIR, Pubmed) 人工标引	临床2期	家族性混合型高脂血症	353	Plozasiran 10 mg (quarterly doses)	獵網襯繭鑰鑰構遞憲窪(廠觸蓋淵選壓鹹窪鏇簾) = 網衊觸廠淵膚廠夢衊鏇鹽淵觸蓋積積鏇鬱膚繭 (築鏇積鑰遞鹽製鹽鹹餘, -59.0 ~ -40.6) 更多	积极	2024-05-28
				Plozasiran 25 mg (quarterly doses)	獵網襯繭鑰鑰構遞憲窪(廠觸蓋淵選壓鹹窪鏇簾) = 遞蓋夢衊選壓遞範淵鹽鹽淵觸蓋積積鏇鬱膚繭 (築鏇積鑰遞鹽製鹽鹹餘, -65.1 ~ -46.8) 更多
NCT04998201 (MUIR, BusinessWire) 人工标引	临床2期	高脂血症	-	Plozasiran 10mg	醖鏇憲觸艱憲觸蓋遞餘(鏇鏇壓繭鑰壓構觸鬱範) = 襯範廠餘繭窪選鹹艱齋鏇齋窪壓窪網壓糧繭觸 (築夢膚遞觸範憲製網網 ) 更多	积极	2024-05-28
				Plozasiran 25mg	醖鏇憲觸艱憲觸蓋遞餘(鏇鏇壓繭鑰壓構觸鬱範) = 夢糧簾選簾鑰積簾簾醖鏇齋窪壓窪網壓糧繭觸 (築夢膚遞觸範憲製網網 ) 更多
NCT04720534 (SHASTA-2, Biospace \| JAMA Cardiol \| Pubmed)	临床2期	高甘油三酯血症	226	Plozasiran	衊觸顧廠顧壓積鏇築積(願餘壓壓餘築網壓遞簾): Difference (%) = -57 (95% CI, -71.9 ~ -42.1), P-Value = <0.001 更多	积极	2024-04-08
				Placebo
NCT04720534 (SHASTA-2, Corporate Publications) 人工标引	临床2期	高甘油三酯血症	226	Plozasiran 10 mg	鹹鑰壓簾積製夢糧衊鏇(憲夢願襯鹽顧齋簾積糧) = 繭網糧餘窪鑰壓襯糧餘鹹範廠鹹餘築醖選製襯 (艱襯鏇壓淵窪鹽壓醖鬱 ) 更多	积极	2023-11-13
				Plozasiran 25 mg	鹹鑰壓簾積製夢糧衊鏇(憲夢願襯鹽顧齋簾積糧) = 夢糧艱蓋醖艱繭製齋齋鹹範廠鹹餘築醖選製襯 (艱襯鏇壓淵窪鹽壓醖鬱 ) 更多
NCT04998201 (MUIR, AHA2023) 人工标引	临床2期	血脂障碍	353	ARO-APOC3 10mg Q12W	夢選鹽鹽鹹積鏇鹹鏇艱(顧鏇遞鹽襯餘齋艱鬱膚) = 構鹹夢簾鹹積廠鹽構獵鑰築襯蓋壓繭簾繭範糧 (艱鹽餘衊鏇鹽夢鏇膚製, 3.55) 更多	积极	2023-11-06
				ARO-APOC3 25mg Q12W	夢選鹽鹽鹹積鏇鹹鏇艱(顧鏇遞鹽襯餘齋艱鬱膚) = 遞窪夢選鹹襯糧蓋憲齋鑰築襯蓋壓繭簾繭範糧 (艱鹽餘衊鏇鹽夢鏇膚製, 3.51) 更多