A Randomized, Placebo-Controlled, Four-Period Crossover, Study to Evaluate the Effect of Volanesorsen on the QTc Interval Using a Therapeutic and Supra-Therapeutic Dose Compared With Placebo in Healthy Volunteers: a Thorough QT Study

The primary objective of this study is to assess the corrected QT interval (QTc) effect of volanesorsen (ISIS 304801) administered as a 300 mg subcutaneous (SC) therapeutic and a 300 mg intravenous (IV; 2-hour infusion) supra-therapeutic dose relative to placebo in healthy adult male and female subjects.

开始日期2016-09-19

申办/合作机构

Ionis Pharmaceuticals, Inc.

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100 项与 Volanesorsen 相关的临床结果

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100 项与 Volanesorsen 相关的转化医学

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100 项与 Volanesorsen 相关的专利（医药）

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项与 Volanesorsen 相关的文献（医药）

2026-07-01·Journal of Comparative Effectiveness Research

Comparative efficacy and safety of olezarsen versus volanesorsen for familial chylomicronemia syndrome: a matching-adjusted indirect comparison

Article

作者: Hakimi, Zalmai ; Kassner, Ursula ; Soran, Handrean ; Serafini, Paul ; Wilson, Koo ; Alexander, Veronica J ; Pushkarna, Divya ; Falk, Aletta ; Stroes, Erik ; Rodrigues, Elisabete ; Kessler, Asia Sikora ; Vera-Llonch, Montserrat ; Schatz, Ulrike

Background: Familial chylomicronemia syndrome (FCS) is a rare genetic disorder characterized by severe hypertriglyceridemia causing recurrent acute pancreatitis (AP). Since FCS is a genetic deficiency in functional lipoprotein lipase, conventional triglyceride-lowering therapies are ineffective in this metabolic disorder. Apolipoprotein C-III (apoC-III) inhibitors, including volanesorsen and olezarsen, have emerged as targeted treatments for FCS. Aim: To compare the efficacy and safety of olezarsen 80 mg every four weeks (Q4W) versus volanesorsen 300 mg weekly (QW) in patients with FCS using an anchored matching-adjusted indirect comparison. Materials & methods: Individual patient data from the Balance trial of olezarsen (n = 45) were weighted to match baseline characteristics reported in the APPROACH trial of volanesorsen (n = 66). Outcomes included percent change in fasting triglycerides (TG) and apoC-III at 26 and 52 weeks, and risks of AP events and adverse events at 52 weeks. Results: At 52 weeks, mean differences in fasting TG and apoC-III were -27.4% (95% CI: -69.4, 14.5) and -21.3% (95% CI: -61.9, 19.3). Relative risks of AP, treatment-emergent adverse events, and serious adverse events were 0.23 (95% CI: 0.01, 5.02), 0.87 (95% CI: 0.66, 1.13) and 0.50 (95% CI: 0.08, 3.26) at week 52. The rate ratio for AP events per patient-year was 0.06 (95% CI: 0.003, 1.41). No comparisons were statistically significant. Conclusion: In this matching-adjusted indirect comparison, no statistically significant differences in outcomes were observed between olezarsen and volanesorsen in patients with FCS. These findings provide important comparative context in a setting where head-to-head evidence is unavailable.

2026-06-01·DIABETES OBESITY & METABOLISM

Efficacy and Safety of Apolipoprotein C‐ III Inhibitors in Hypertriglyceridemia: A Network Meta‐Analysis of Randomised Controlled Trials

Review

作者: Elazab, Ahmed ; Elbahloul, Mohammed A. ; Elgendy, Islam Y. ; Gamal, Aliaa ; Awad, Ayman K. ; Attia, Manar Khaled ; Salah, Ahmed M. ; Elbaz Hassan, Moustafa M. ; Elboraay, Toka ; Akoum, Atef ; Maihoub, Odai

ABSTRACT:

Background:

Hypertriglyceridemia is an independent risk factor for atherosclerotic cardiovascular disease (ASCVD). We aimed to evaluate the efficacy and safety of apolipoprotein C‐III (ApoC‐III) inhibitors in patients with hypertriglyceridemia.

Methods:

Electronic databases were systematically searched for randomised controlled trials (RCTs) comparing ApoC‐III inhibitors (Volanesorsen, Olezarsen and Plozasiran) with placebo. A frequentist network meta‐analysis was performed. Continuous outcomes were presented as mean differences (MDs), and dichotomous outcomes as risk ratios (RRs), both with 95% confidence intervals (CIs).

Results:

A total of 15 RCTs involving 3934 patients were included. All ApoC‐III inhibitors demonstrated significant reductions in TG, except for Volanesorsen 100 mg Q1W and Olezarsen 10 mg Q4W. Olezarsen 80 mg administered every 4 weeks had the highest SUCRA ranking for TG reduction (MD: −63.26; 95% CI: −70.04 to −56.47; p < 0.01). Moreover, ApoC‐III inhibitors were associated with a significant reduction in the incidence of pancreatitis (HR: 0.16, 95% CI: 0.07–0.33, p < 0.01). None of the agents significantly increased the risk of serious adverse events.

Conclusions:

ApoC‐III inhibitors significantly improved triglyceride levels and other lipid parameters and, most importantly, substantially reduced the risk of acute pancreatitis. Future trials are needed to evaluate their effects on cardiovascular outcomes.

2026-05-04·Expert Review of Endocrinology & Metabolism

The evaluation of plozasiran for the treatment of familial chylomicronemia syndrome

Review

作者: Hang, Shyann ; Hegele, Robert A.

INTRODUCTION:

Familial chylomicronemia syndrome (FCS) is a rare autosomal recessive disorder marked by severe hypertriglyceridemia and characteristic clinical manifestations, particularly acute pancreatitis. Conventional triglyceride-lowering therapy is largely ineffective. Apolipoprotein (apo) C-III has emerged as a key therapeutic target to lower triglycerides in FCS.

AREAS COVERED:

This review compares FCS with more common multifactorial chylomicronemia. We searched PubMed for all English language literature focusing on the search terms 'chylomicronemia,' 'hypertriglyceridemia,' 'APOC3 inhibition,' 'plozasiran,' 'olezarsen,' and 'volanesorsen.' We outline traditional management strategies and their limited role in FCS and explore non-traditional therapies including orlistat, lomitapide, inhibitors of angiopoietin like protein 3 (ANGPTL3), and analogues of fibroblast growth factor 21 (FGF21). The primary focus is on RNA-based gene silencing therapeutics that target apo C-III, particularly the small interfering RNA plozasiran and the allele specific oligonucleotides volanesorsen and olezarsen, highlighting key differences in efficacy and tolerability.

EXPERT OPINION:

In a phase 3 trial of plozasiran, at 10 months, median placebo-adjusted reductions in apo C-III were approximately -90%, while TG levels were reduced up to -59%. Thus, plozasiran and alternative RNA-based therapeutics directed against APOC3 represent transformational therapies for patients with FCS and related phenotypes characterized by severe recalcitrant hypertriglyceridemia.

151

项与 Volanesorsen 相关的新闻（医药）

2026-08-10

·全和诚

全和诚与陈润生院士团队签约共建长链非编码RNA核酸新药创制平台

从靶点发现到产业化落地全和诚以"非编码RNA落地主体"身份打通核酸药物创新的"最后一公里" 2026年8月6日，第十届中国（天津）核酸药物大会开幕式上，天津全和诚科技有限责任公司与北京睿博解码生物科技有限公司正式签署"基于长非编码RNA的核酸新药创制"项目合作协议。这不是一次普通的商业签约。它标志着全和诚作为"非编码RNA从靶点发现到产业化落地的承载主体"，正式与全球非编码RNA研究的最前沿力量——中国科学院院士陈润生团队——形成战略绑定。院士团队负责从浩瀚基因组中"找到靶点"，全和诚负责把靶点"送到病床"——双方共同构建从源头创新到产业化落地的完整闭环。一、为什么是陈润生院士？——非编码RNA领域的"拓荒者" 陈润生，中国科学院院士，生物信息学家，中国非编码RNA研究的奠基人之一。他是国际上最早从事非编码RNA系统研究的科学家之一，其团队在长链非编码RNA（lncRNA）的识别、功能注释与靶点发现方面积累了全球领先的学术资产。在本次大会的主题报告中，陈润生院士指出： "人类基因组中仅约2%的序列编码蛋白质，其余98%曾被称为"暗物质"。如今我们已知，这些非编码RNA并非"噪声"，而是调控生命活动的核心语言。AI技术正从技术底层重塑新药研发全流程，能有效应对靶点发现难、分子设计慢、临床成本高等核心痛点。" 陈润生院士团队的独特价值在于：他们不仅发现了大量lncRNA靶点，更建立了系统化的靶点功能注释方法——能够从数万个lncRNA中筛选出真正具有成药潜力的靶点，并初步阐明其调控机制。这种"从暗物质到明确靶点"的能力，是lncRNA药物研发链条中最稀缺的源头创新。二、lncRNA是什么？——核酸药物的"下一个浪潮" 长链非编码RNA（Long non-coding RNA, lncRNA）是一类长度超过200个核苷酸、不编码蛋白质但在基因表达调控中发挥关键作用的RNA分子。与传统的mRNA（信使RNA）和siRNA（小干扰RNA）不同，lncRNA的功能机制更为复杂——它们通过染色质重塑、转录调控、转录后加工等多种途径参与几乎所有的生命活动。正因如此，lncRNA药物被认为是核酸药物领域的"下一个浪潮"： 01 广谱性覆盖适应症广——lncRNA调控的通路涉及罕见病、肿瘤、神经系统疾病、心血管疾病、代谢性疾病等多个大适应症赛道；靶点丰富 02 靶点空间巨大——人类基因组中已注释的lncRNA超过10万条，但目前进入药物开发阶段的不足百条，靶点空间几乎是"蓝海中的蓝海"； 03 市场参照全球已有3款反义核酸（ASO）药物获批用于罕见病——Spinraza（治疗脊髓性肌萎缩症）、Exondys 51（治疗杜氏肌营养不良症）、Waylivra（治疗家族性乳糜微粒血症综合征），验证了靶向RNA的治疗策略临床可行。lncRNA作为更前沿的靶点类别，潜在市场空间更为广阔。但lncRNA成药也面临独特的"三重难题"： 01 靶点功能不明绝大多数lncRNA的生物学功能尚不明确，"靶点找到了但不知道它能干什么"是行业普遍困境；序列设计难 02 lncRNA序列长、结构复杂，如何设计有效的核酸药物分子（ASO/siRNA/适配体）精准靶向并调控其功能，缺乏成熟方法论； 03 组织递送难 lncRNA的作用位点常位于特定组织或细胞器内，现有递送系统难以精准送达——这是lncRNA从"实验室发现"到"成药"之间最宽的鸿沟。三、合作内容——院士团队"找靶点"，全和诚"送到位" 全和诚与陈润生院士团队的合作，正是围绕lncRNA成药"三重难题"展开的分工协作：环节陈润生院士团队全和诚靶点发现基于全球领先的非编码RNA研究积累，从基因组中筛选具有成药潜力的lncRNA靶点，完成功能注释与机制阐释 _ 分子设计提供靶点序列信息与功能验证需求基于Q-NADSP递送平台，设计匹配靶点的核酸药物分子（ASO/siRNA/适配体）与递送方案递送与验证 _ 依托递送平台完成多器官靶向递送验证，依托管线验证平台完成GMP级工艺验证与药效药代评估产业化落地 _ 依托合成平台提供GMP级核心元件供应，推动从临床前到IND申报的完整产业化路径简而言之：院士团队解决"靶点功能不明"和"序列设计难"的前两端，全和诚解决"组织递送难"的最后一端——并通过三大平台的协同运作，将一个lncRNA靶点从"论文里的发现"转化为"可申报IND的药物资产"。四、三大平台如何支撑lncRNA从靶点到药物？全和诚此次签约的核心底气，在于其已构建的"以递送为核心发力点、合成为基础支撑、管线验证为技术延伸"的三位一体硬科技底座。这套体系恰好覆盖了lncRNA成药最关键的三个环节：递送平台（Q-NADSP）：打通lncRNA的"最后一公里" lncRNA的作用位点常位于特定组织（如神经系统中枢、肿瘤微环境、肌肉组织等），传统LNP递送系统主要靶向肝脏，难以满足lncRNA药物的多样化递送需求。全和诚Q-NADSP平台已验证肝、脾、肺、肌肉四器官靶向递送能力，正在拓展脑靶向、心脏靶向、骨骼靶向、胰腺靶向、肾靶向等更多器官——这意味着： ·针对神经系统lncRNA靶点，可匹配脑靶向递送方案； · 针对肿瘤免疫相关lncRNA靶点，可匹配脾靶向递送方案（激活免疫细胞）； · 针对肌营养不良症等肌肉相关lncRNA靶点，可匹配肌肉靶向递送方案； · 针对肝癌等肝脏相关lncRNA靶点，已验证85.5%抑瘤率的肝靶向LNP制剂。 3000+独立知识产权可电离脂质库和20+创新专利，使全和诚能够为每一个lncRNA靶点"量身定制"递送方案——这是lncRNA成药最关键、也是最稀缺的能力。合成平台：GMP级元件供应的"弹药库" lncRNA核酸药物的核心元件——亚磷酰胺单体（2'-F、2'-OMe、2'-MOE等化学修饰单体）、GalNAc递送分子、LNP阳离子脂质——必须达到GMP级质量标准才能支撑临床申报。全和诚合成平台已具备从研发到公斤级GMP生产的全链条供应能力，单体纯度标准为HPLC纯度≥99.0%、单杂≤0.1%、水分≤0.2%。这意味着：当院士团队发现一个新的lncRNA靶点后，全和诚可以在内部完成从分子设计、元件合成、递送配方到制剂制备的全流程——不需要依赖外部供应商，极大缩短了从靶点到候选药物的转化周期。管线验证平台：从递送能力到管线资产的"转化器" 找到靶点、设计好分子、合成出元件、完成递送——这只是lncRNA成药的上半场。下半场是：工艺验证、药效药代评估、安全性评价、IND申报准备。全和诚管线验证平台面向Biotech与创新药企开放GMP级验证服务，将递送能力转化为可交付的管线资产。在此次合作中，管线验证平台将承担lncRNA候选药物的体内外药效验证、工艺放大验证与申报支持——确保院士团队发现的每一个靶点，都能以最高的转化效率走向临床。五、从全和诚来看：未来价值与战略意义此次签约对全和诚的价值，远超单一项目的商业回报。它将在四个维度上重新定义全和诚的战略位置：价值一：确立"非编码RNA落地主体"战略身份全和诚不再仅仅是一家"核酸药物递送系统供应商"或"核心元件生产商"。通过与陈润生院士团队的战略签约，全和诚正式确立了"非编码RNA从靶点发现到产业化落地的承载主体"这一战略身份。这一定位的核心含义是：全和诚不做"昙花一现的明星分子"，而要做"非编码RNA落地的那座桥"——从靶点到病床，从实验室到产业。未来，当行业提到"非编码RNA药物产业化"时，全和诚将成为绕不开的合作伙伴。价值二：构建"源头创新→递送→产业化"的闭环壁垒国内核酸药物企业多数集中在"递送系统优化"或"仿制已验证靶点"两个环节，真正打通"源头靶点发现→递送系统设计→GMP级生产→管线验证→IND申报"全链条的企业极少。全和诚通过与院士团队的合作，将"源头创新"这一最稀缺的环节纳入自身生态——形成了多数竞品难以复制的闭环壁垒。这意味着：全和诚的递送平台不再是"等客户拿靶点来找我们做递送"，而是"我们和最顶尖的靶点发现者一起，从源头就参与药物创制"——从被动接单变为主动共建。价值三：递送平台差异化定位的"杀手级验证" 全和诚Q-NADSP递送平台的核心差异化——源头自主设计、四器官靶向验证、开放合作模式——在此次合作中得到了"杀手级验证"：当中国最顶尖的非编码RNA研究团队选择全和诚作为递送合作伙伴时，这本身就是对递送平台能力的最高背书。未来在向其他Biotech/创新药企推介递送平台时，"陈润生院士团队选择的全和诚Q-NADSP平台"将成为最有力的信任锚点——这比任何数据展示都更有说服力。价值四：打开lncRNA赛道的市场想象空间全球lncRNA药物目前尚无获批产品，但已有多个候选药物进入临床阶段，适应症覆盖脊髓性肌萎缩症、杜氏肌营养不良症、亨廷顿病等。随着陈润生院士团队持续输出新的lncRNA靶点，全和诚将获得源源不断的管线储备——每一个靶点都可能对应一个数十亿级的大适应症赛道。更关键的是：lncRNA靶点的递送需求高度多样化（脑、肌肉、脾、肺等），而全和诚恰好是国内极少数同时具备多器官靶向递送验证能力的平台。这意味着在lncRNA赛道中，全和诚的递送平台不仅是"合作选择"，更是"刚需基础设施"。六、闭环模型——一个lncRNA靶点在全和诚的"旅行路线" 以一个假设的lncRNA靶点为例，它在全和诚体系中的完整转化路径如下：第1步靶点发现：陈润生院士团队从基因组中筛选出具有成药潜力的lncRNA靶点，完成功能注释与机制阐释第2步分子设计：全和诚基于靶点序列信息，设计匹配的ASO/siRNA/适配体分子，并选择对应的化学修饰方案第3步元件合成：全和诚合成平台按GMP级标准合成所需的亚磷酰胺单体、修饰核苷酸等核心元件第4步递送配方：全和诚Q-NADSP平台根据靶点作用组织（脑/肌肉/脾/肺/肝），从3000+脂质库中筛选匹配的可电离脂质，设计靶向LNP配方第5步体内验证：在小鼠模型中完成靶向递送验证、药效评估与药代分析第6步工艺放大：管线验证平台完成GMP级工艺放大验证，制备符合申报要求的制剂第7步 IND申报：整理全套CMC数据与药理毒理数据，向监管机构提交IND申请第8步产业化：合成平台支撑商业化生产，递送平台持续优化配方，管线验证平台支持临床阶段迭代在这条完整路线上，院士团队负责第1步，全和诚负责第2-8步——这就是"非编码RNA落地主体"的完整含义结语全和诚与陈润生院士团队的合作，不是一次简单的"技术授权"或"项目委托"。它是一个战略级生态构建：中国最顶尖的非编码RNA研究力量，与国内极少数具备多器官靶向递送验证能力的核酸药物平台企业，共同构建从"暗物质"到"药物"的完整通道。对于全和诚而言，这次签约意味着三件事： · 从"递送系统供应商"升级为"非编码RNA落地主体"——战略身份的跃迁； · 从"等客户找上门"升级为"与最顶尖源头创新者共建管线"——商业模式的升级；从"单一递送能力展示"升级为"闭环生态壁垒"——竞争壁垒的质变。 "院士团队发现靶点，全和诚送到病床——全和诚是非编码RNA从源头创新到产业化落地的承载主体。厚植核酸沃土，共育产业未来。全和诚正在用三大平台的硬实力，把这句话从呼吁变为现实。天津全和诚科技有限责任公司联系电话：022-25327698 公司官网：www.cosychem.com 公司地址：天津市滨海新区第四大街80号天大科技园C3座

核酸药物寡核苷酸信使RNAsiRNA

2026-08-08

·制药技术研讨会

研发热点||小核酸药物研发优势及非临床评价研究策略

谢谢关注；每天有药界新闻和课件分享，感谢转发引言：小核酸药物是指能利用核酸小分子特异性地调控疾病基因的表达的药物，通过与靶mRNA结合，对其翻译起到抑制或调控作用，从而达到治疗效果。从作用机理来看，小核酸药物能够直接影响靶向致病靶基因或RNA片段，使其治疗领域更广，有望成为无靶向药物的遗传疾病和其他难治性疾病的新救星。作为新药开发领域的热门赛道，小核酸药物全球市场规模从 2016年0.1亿美元已增长至2021年32.5亿美元，年复合增长率高达217.8%。未来随着临床阶段小核酸药物的不断上市，尤其是针对患者群体较大的适应症药物，如乙型肝炎的潜在治愈性药物，将进一步驱动市场快速发展。预计 2025 年全球小核酸药物销售额将突破100亿美元一小核酸药物的特点与优势小核酸药物作为目前最成熟的基因疗法之一，独特的作用机理使其拥有先天的疗效优势：治疗效率高、特异性强、药物毒性小和应用领域广等，而且相比于当前同样热门的抗体和小分子药物，其具备如研发周期短、不易产生耐药性、效果持久、研发成功率较高等特点，被认为是继小分子药、抗体药后推动医药产业变革的第三代制药创新技术。小核酸药物的优势包括但不限于： 1.药物靶点筛选快，研发周期短：小核酸药物无需对蛋白质复杂空间结构进行的大规模药物筛选，只需要锁定致病基因序列，并针对该基因序列进行设计及相应RNA片段的合成，因此其早期研发速度远远快于抗体和小分子药物； 2.治疗领域更广：小核酸药物理论上可以设计用于靶向任何感兴趣的基因，仅需获知目标mRNA的序列信息，即可针对靶点进行药物设计； 3.效果持久：小核酸药物在体内的半衰期可以按照月来计算，大大降低给药频次，对很多疾病尤其是慢性病的治疗具有巨大的优势； 4.特异性强：小核酸药物作用机制明确，通过与mRNA完成靶向碱基配对来实现其功能，特异性相对于其他药物优势明显。尽管近年来小核酸类药物研发发展迅速，其自身局限性仍使该类药物进入临床具有潜在风险，如易被核酸酶降解、系统给药后进入细胞内传递到靶部位效率问题以及潜在的不良反应，如肾小球肾炎、血管炎和血小板减少等。二上市药物调研目前全球进入临床的小核酸药物近108个，包括 ASO、siRNA、Aptamer、miRNA等。其中ASO仍是当前研发热点，占比38%，siRNA发展快速，占比已达到32%，其余小核酸药物研发相对还处在早期阶段，整体数量较少。截至2023年9月，已获批的15款小核酸药物中，遗传罕见病是目前获批最多的适应症类别，共有11款针对遗传罕见病，2款针对眼科疾病，1款针对心血管疾病，1款针对代谢性疾病。目前获批适应症主要集中于遗传性罕见病，可能与这些罕见病致病靶点明确方便小核酸设计、缺少对应疗法可以加速进行临床以及罕见病对药物安全性耐受度较高等原因有关。小核酸药物根据其作用机制大致可分为：反义寡核苷酸（ASO）：通过与mRNA互补配对，抑制特定蛋白质的产生。目前已有9款ASO药物获批上市，如Nusinersen（Spinraza）用于治疗脊髓性肌萎缩症（SMA）。小干扰RNA（siRNA）：通过RNA干扰（RNAi）机制，降解特定的mRNA，从而沉默基因表达。已有5款siRNA药物获批上市，例如Patisiran（Onpattro）用于治疗遗传性甲状腺素介导的淀粉样变性的多发性神经病（hATTR）。微小RNA（miRNA）：模拟内源性miRNA的调控作用，可以抑制或激活特定基因的表达。小激活RNA（saRNA）：与miRNA相反，saRNA用于激活基因表达。核酸适配体（Aptamer）：人工合成的短单链DNA或RNA序列，通过三维构型与靶标特异性结合。（一）反义寡核苷酸（ASO）反义寡核苷酸是一种单链寡核苷酸分子，通常为18~30 个核酸序列的短片段，其进入细胞后在核糖核苷酶H1的作用下通过碱基互补配对原则与其互补的靶Mrna结合，对靶基因产生调控作用。与目标基因结合后的ASO以下不同的作用机制来调节靶mRNA的功能： 1.Cleave and Degrade 切断RNA并使其降解：ASO与靶mRNA 结合后，可招募内源性核糖核酸内切酶RNaseH，切断互补配对区域mRNA序列以沉默目的基因，下调蛋白表达。由于RNase H.存在于细胞核内外，所有ASO不止作用于mRNA，也可以调控一些noncoding RNA（研究发现很多没有明确蛋白靶点的疾病发病机制都与noncoding RNA有关），使其成为相对于siRNA的一大优势，因为绝大部分siRNA只能在细胞核外工作； 2.Splice 剪接/拼接：DNA转录生成的pre-mRNA，需由剪接体将其中的内含子剪除，再将外显子有序连接，从而加工为成熟mRNA。而剪接体识别并结合pre-mRNA需要借助于内含子上的特定RNA序列，ASO可与此特定序列结合，改变剪接行为，干扰基因表达。（二）小干扰RNA（siRNA）小干扰RNA（Small interfering RNA，siRNA）通常是含有19-23个碱基对的双链RNA片段，可通过与载体共价偶联，特异性地靶向发病组织发挥基因沉默作用。siRNA就是通过RNAi机制发挥的作用：将双链RNA（dsRNA）导入体内后，会被特定的核糖核酸酶（Dicer）切割成长度为21~23个碱基对的小片段，这些小片段称为小干扰RNA（siRNA）。siRNA进入细胞后，细胞质内的Ago2酶会将siRNA的正义链裂解，反义链则会被装载到RNA诱导的沉默复合体中（RISC），与靶mRNA特异性结合使其降解，从而抑制蛋白的表达。（三）微小RNA（miRNA）微小RNA（microRNA, miRNA）是长度约22个核苷酸片段的非编码小分子RNA，主要通过抑制/裂解mRNA两种方式来调控基因的表达。具体说来，miRNA首先在细胞核内转录出较长的初级miRNA（pri-miRNA），然后在细胞核内由Drosha加工成前体miRNA（pre-miRNA），而后被转运出细胞核，在细胞质中由Dicer 11剪切成为成熟的miRNA，随即被整合进RNA沉默复合物（RISC）中，基于与mRNA完全或不完全配对来调节基因表达。（四）小激活RNA（saRNA）小激活RNA（saRNA）是一种小型双链RNA分子，它通过RNA激活（RNAa）机制发挥作用，与小干扰RNA（siRNA）的化学结构类似，但作用结果相反。saRNA通过招募内源性转录复合物到靶基因，导致mRNA的表达增加和靶蛋白的上调。具体机制为双链的saRNA（长度一般在21个核苷酸）通过内吞作用进入细胞；装载到AGO2蛋白上；载体链被降解；AGO2和活性链复合物被运输进细胞核内和启动子区域结合；通过与RNA解旋酶和RNA多聚酶联系蛋白（CTR9）的结合吸引RNA多聚酶从而激活相应基因的转录；最终促进相应蛋白的表达。（五）核酸适配体（Aptamer）核酸适配体是一类有“核酸抗体”之称的人工合成的长度为20-100个核苷酸的单链DNA或RNA分子，可以形成特定的三维结构，从而高选择性和特异性结合作用靶点。相比抗体药物，核酸适配体具有分子量小、免疫原性低、组织穿透力强、易于合成与修饰、生产成本低等优点，在疾病诊断、治疗和预防中有着广泛的药物应用潜力。核酸适配体药物通过三种路径发挥作用：1）作为抑制剂抑制疾病相关靶标；2）作为激动剂激活目标受体；3）作为靶向分子载体递送其它药物。四小核酸药物的安全性评价小核酸药物非临床安全性评价内容与小分子化学药物基本类似，主要包括安全药理学实验、一般毒理学实验、遗传毒性实验、生殖毒性实验和致癌性实验及特殊毒性实验，参考的技术指南包括国家药品监督管理局、美国FDA及ICH等有关的药物安全性评价相关指导原则。尽管如此，小核酸药物还存在自身特点，它们产生的毒性既有来自于药理作用放大而引起的靶点(on-target) 毒性，也有来自于与同源性RNA序列结合或本身理化性质产生的脱靶(off-target)毒性，因此，不能完全照搬生物制品或者化学药物非临床安全性评价的要求来进行小核酸药物毒理研究。（一）一般原则：动物种属：通常要求啮齿类和非啮齿类2个动物种属，常用小鼠（或大鼠）和非人灵长类（NHP），犬和小型猪可作为非啮齿类动物的替代，应至少有一个相关动物种属。目前已获得的单链寡核苷酸安全性数据主要来源于NHP。啮齿类动物对免疫刺激反应敏感性高于NHP，可能出现炎症相关的非特异性肿瘤。该类药物到达体内稳态浓度较慢，重复给药毒性试验中给药结束时的血药浓度应至少达到90%稳态浓度。考虑到单链寡核苷酸半衰期长，应设置足够长的恢复期（理想情况下至少8周）以暴露迟发毒性和观察恢复情况。（二）安全药理学实验：因小核酸药物发挥药理作用的方式与生物大分子药物类似，因此，在合适时可以考虑将安全药理学结合到重复给药毒性实验中一起评价，评价的项目包括呼吸、中枢和心血管系统安全药理学实验，选择的动物种属包括小鼠、大鼠及猴等。尽管小核酸药物由于分子量相对较大且带有电荷，其无法直接抑制hERG 钾离子通道，但仍有大部分已上市的此类药物进行了体外hERG 实验。因此，建议小核酸药物开展体外hERG 实验，以进一步评价药物可能引起的心血管风险。静脉给药药物的血浆半衰期短，给药后数小时内即可能引起补体活化，应考虑开展心血管系统遥测。除了上述三大核心系统外，还因部分小核酸药物分别通过肝脏代谢和肾脏排泄，故也需要考虑补充肝脏和肾脏安全药理实验。（三）一般毒理学：由于小核酸药物存在“on-target”毒性，故在种属选择时，有必要评估是否包含了至少一种能产生与临床药理活性具有可比性的动物。如无法获得相关动物种属，也可根据药物特点选择转基因动物和人源化模型动物等替代，但需要充分评估产生的毒性与临床相关性。一般采用临床拟用的给药途径，其中静脉注射和皮下注射较为常见。给药期限主要参考ICHM3( R2) 指导原则的要求，应支持相应的临床试验给药疗程，一般情况下最长给药期限为啮齿类给药26周、非啮齿类给药39周，可用于支持所有临床试验和上市。除常规的检测指标外，小核酸药物还应考察药效终点指标，以便进一步验证在药物与靶mRNA结合的药理作用。肾和肝是主要毒性靶器官，未见对脑、骨骼肌、睾丸等组织毒性的报道，雄性大鼠对肾毒性特别敏感，常见蛋白尿（与药物刺激导致的慢性进行性肾组织病理学改变有关）以及肾组织病理学改变，啮齿类（特别是小鼠）比NHP对肝毒性更为敏感，炎性反应程度更高，因而与临床肝毒性的相关性有限。有临床意义的毒性反应多为慢性反应，主要见于实体组织。因此，靶组织药物浓度对预测安全性风险具有重要意义。（四）遗传毒理实验：就寡核苷酸自身性质而言，此类药物一般不会引起遗传毒性，且目前的药物均为阴性结果，但寡核苷酸安全工作组( OSWG) 遗传毒理委员会仍认为对于新的寡核苷酸药物有必要进行遗传毒性实验，因为修饰的单体可能从寡核苷酸代谢时释放出来并整合到DNA 中，理论上会导致链终止、错误编码和/或错误复制或修复; 另外，该委员会推荐选用ICH S2( R1) 指导原则标准“组合1”考察此类药物的遗传毒性，即2 项体外实验( 细菌回复突变实验+ 哺乳动物细胞染色体畸变实验或小鼠淋巴瘤Tk 基因突变实验) 与1 项体内实验( 体内微核实验或染色体畸变实验)。（五）生殖毒性实验：小核酸药物一般需要开展生殖毒性实验，其时间和内容主要参照ICH M3( R2) 和S5( R3) 的要求。一般可采用分段式生殖实验策略，包括生育力和早期胚胎发育毒性实验、胚胎-胎仔发育毒性实验和围产期发育毒性实验，选择的动物种属多为小鼠、大鼠和兔，给药方式一般采用临床拟用给药途径。另外，根据拟用人群，必要时需要开展幼龄动物毒理学实验。可将周剂量分成日剂量给药，以确保在子代离乳前关键发育期维持日母体暴露和胎盘暴露。对于拟局部给药的单链寡核苷酸，可按照一般毒理学试验中所确定的暴露量全身给药。（六）致癌性实验：根据ICH S1A，预期临床用药至少连续6 个月的药物都应进行致癌性实验，小核酸药物也不例外，除非用于晚期肿瘤等预期寿命较短的疾病。一般选择大鼠开展为期2 年的长期致癌性实验，以及采用转基因小鼠开展6 个月的致癌性实验，给药方式亦采用临床拟用途径。（七）特殊毒性实验：ICH M3( R2) 指导原则中提到“如果药物或同类药物以往的非临床或临床发现提示药物具有特殊的安全性担忧，进行附加的非临床试验是有益的”。根据小核酸药物自身的特点和已上市同类药物评价的经验，需要开展的特殊毒性实验包括但不限于: ① 部分小核酸药物根据拟用人群的需要，开展了幼龄动物毒性实验，如mipomersen等。② 鉴于小核酸药物也具有部分生物药的性质，故也需要进行免疫原性和免疫毒性实验及细胞因子/趋化因子评估实验［4］。③ 因一些小核酸药物被发现会引起血小板减少，如inoterse 和volanesorsen，且它们的给药方式多数为注射，故有必要进行体外溶血实验。④ 为了考察小核酸药物长期给药后是否会被降解，还应检测2＇-FU 插入细胞内DNA 和RNA 的水平，如pegaptanib。参考文献 [1] Niemietz, C., Chandhok, G., & Schmidt, H. (2015). Therapeutic Oligonucleotides Targeting Liver Disease: TTR Amyloidosis. Molecules, 20(10), 17944–17975. doi:10.3390/molecules201017944 [2] Sun, H., Zhu, X., Lu, P. Y., Rosato, R. R., Tan, W., & Zu, Y. (2014). Oligonucleotide Aptamers: New Tools for Targeted Cancer Therapy. Molecular Therapy - Nucleic Acids, 3, e182. doi:10.1038/mtna.2014.32 [3] MAKIK Preclinical safety assessments of mRNA-targeting oligonucleotidetherapeutics［J］ Transl Regul Sci，2020，2(3) : 90－93． [4] YIN W，ROGGE M． Targeting RNA: a transformative therapeuticstrategy［J］．Clin Transl Sci，2019，12(2) : 98－112． [5] BERMAN CL，BARROS SA，GALLOWAY SM，et al．OSWG recommendations for genotoxicity testing of novel oligonucleotidebased therapeutics［J］． Nucl Acid Ther，2016，26( 2) : 73 － 85． [6] 王恒, 李华, 汪溪洁, 等. 小核酸药物非临床特点和药理毒理评价策略[J]. 中国新药杂志, 2022, 31(12). 赛赋医药免责声明；本文章来自网络内容为作者个人观点,并不代表本公众号赞同或支持其观点。如涉及作品内容、版权等问题，请联系我们删除！本公众号拥有对此声明的最终解释权点击下方链接阅读全文团队报名更优惠日期会议专题（点击下方链接预览全文） 8月15-16日线上-化学创新药注册申请（中美欧澳日） 8月18-20日苏州-（特惠1000元/人）制药设备全生命周期管理与合规运维实战培训专题 8月22-23日线上-抗体偶联药物ADC的CMC全流程、质量控制与前沿趋势解析高级研修班 8月21-23日南京-基于无菌附录与最新欧盟法规中“水系统”和“空调系统”的检查指南解读及改造策略专题培训 8月27-28日线上-药品研发质量管理体系七大模块从建立到落地实施策略专题 8月29-30日线上-小核酸药物非临床评价与药理毒理策略培训 8月29-30日线上-生物药BLA申报资料撰写实操及常见问题案例分析专题 9月19-20日线上-临床药理高级实战培训班：从机制到精准用药 9月17-18日线上-生物药纯化工艺开发及工业放大培训专题 9月17-19日【杭州】药包材企业ISO 15378质量体系认证全流程实战培训班 —应对新规挑战，构建符合GMP要求的药包材质量管理体系报名方式 Science Technology 电话微信｜袁莉 13552820065 详情咨询｜扫码添加微信点击左下方“阅读原文”或扫描下方二维码报名提供制药企业｜相关单位｜内训服务诚邀赞助单位｜协办单位及新产品｜新设备展示单位点击“阅读原文”填写报名信息

2026-08-03

·PRNewswire

PTC Therapeutics Provides Corporate Update and Reports Second Quarter 2026 Financial Results

– Robust Q2 performance with total revenue of $361 million, including $239 million of product revenue – – Full-year 2026 expected total revenue increased to $1.18 to $1.28 billion, with full-year product revenue guidance raised to $850 to $950 million – – Sephience™ (sepiapterin) Q2 2026 revenue of $151 million, with continued broad uptake and geographical expansion – – Strong cash position of $2.2 billion as of June 30, 2026 – WARREN, N.J., July 30, 2026 /PRNewswire/ -- PTC Therapeutics, Inc., (NASDAQ: PTCT) today announced a corporate update and financial results for the second quarter ended June 30, 2026. "Our teams delivered another strong quarter enabling us to again raise 2026 product revenue guidance," said Matthew B. Klein, M.D., Chief Executive Officer. "We also made a number of advances in our R&D pipeline, including identification of our next small molecule splicing development candidate, PTC303, targeting somatic expansion disorders including Huntington's disease and myotonic dystrophy. In addition, we initiated the Phase 1 study of PTC612, our differentiated NLRP3 small molecule." Key Corporate Updates Q2 2026 total revenue of $361 million, including $239 million of product revenue Sephience global launch continues with strong momentum Q2 2026 revenue of $151 million, with the majority from the US and increasing contributions internationally 1,647 patients on commercial therapy worldwide as of June 30, 2026 Positive results from votoplam PIVOT-HD long-term extension study reported and enrollment in global Phase 3 INVEST-HD study ongoing PIVOT-HD 24-month results demonstrated dose-dependent disease slowing on cUHDRS scale in Stage 2 participants, reaching 52% at 10 mg dose level Plan to engage with FDA in 2H 2026 to discuss PIVOT-HD 24-month results being finalized INVEST-HD study being conducted and funded by partner Novartis with target enrollment of approximately 770 individuals with early symptomatic disease INVEST-HD initiation triggered a $50 million milestone payment from Novartis to PTC in Q2 2026 Vatiquinone PROVE-FA study to support NDA resubmission for treatment of Friedreich's ataxia expected to initiate in Q3 2026 Global study will enroll approximately 120 individuals with Friedreich's ataxia age 7 to 21 Study includes open-label treatment arm with matched natural history control group PTC pipeline continues to advance PTC303 named as MSH3 splicing program development candidate targeting Huntington's disease and myotonic dystrophy with plan to enter clinic in 2027 Phase 1 study of PTC612, an oral NLRP3 inhibitor, initiated in Q2 2026 Phase 2a study of PTC844, a next-generation DHODH inhibitor, expected to initiate in Q3 2026 Hege Sollie-Zetlmayer has been appointed to PTC's Board of Directors. Ms. Sollie-Zetlmayer has over 30 years of experience in the biopharmaceutical and medical device industry as a global business operations and human resources leader. She most recently served as PTC's Chief Human Resources Officer prior to her retirement in June 2026. Second Quarter 2026 Financial Highlights Total net product revenue and royalty revenue was $309.9 million for the second quarter of 2026, compared to $175.9 million for the second quarter of 2025. Total net product revenue across the commercial portfolio was $238.8 million for the second quarter of 2026, compared to $118.3 million for the second quarter of 2025, representing over 100% increase. Sephience net product revenues were $151.3 million for the second quarter of 2026, representing 21% growth compared to the first quarter of 2026. Translarna™ (ataluren) net product revenues were $42.2 million for the second quarter of 2026, compared to $59.5 million for the second quarter of 2025. Emflaza® (deflazacort) net product revenues were $24.6 million for the second quarter of 2026, compared to $36.4 million for the second quarter of 2025, due to continued generic erosion. Roche reported Evrysdi® (risdiplam) year-to-date sales of approximately 968 CHF million, resulting in royalty revenue of $71.1 million to PTC for the second quarter of 2026, compared to $57.6 million to PTC for the second quarter of 2025. In the second quarter of 2026, PTC recorded a development milestone of $50.0 million from Novartis for the first patient dosed in the ongoing INVEST-HD Phase 3 study. This sales milestone was recorded as collaboration revenue. Based on US GAAP (Generally Accepted Accounting Principles), GAAP R&D expenses were $99.2 million for the second quarter of 2026, compared to $113.0 million for the second quarter of 2025. Non-GAAP R&D expenses were $88.6 million for the second quarter of 2026, excluding $10.5 million in non-cash, stock-based compensation expense, compared to $104.0 million for the second quarter of 2025, excluding $9.0 million in non-cash, stock-based compensation expense. GAAP SG&A expenses were $80.6 million for the second quarter of 2026, compared to $85.3 million for the second quarter of 2025. Non-GAAP SG&A expenses were $67.9 million for the second quarter of 2026, excluding $12.7 million in non-cash, stock-based compensation expense, compared to $75.7 million for the second quarter of 2025, excluding $9.5 million in non-cash, stock-based compensation expense. Net income was $83.5 million for the second quarter of 2026, compared to net loss of $64.8 million for the second quarter of 2025. In June 2026, PTC issued $550.0 million of senior convertible notes due in 2031 at 0% coupon and a conversion price representing a 40% premium over the stock's closing price at the time of the deal. Concurrent with the transaction, PTC repurchased the majority of its 1.5% senior convertible notes due in September 2026. Cash, cash equivalents, and marketable securities were $2,229.1 million on June 30, 2026, compared to $1,945.4 million on December 31, 2025. Shares issued and outstanding as of June 30, 2026, were 83,327,286. PTC Updates Full-Year 2026 Financial Guidance Expected total revenue increased to $1.18 to $1.28 billion, with total product revenue guidance raised to $850 to $950 million from $750 to $850 million GAAP R&D and SG&A expense guidance remains $775 to $815 million Non-GAAP R&D and SG&A expense guidance remains $680 to $720 million, excluding estimated non-cash, stock-based compensation expense of $95 million Non-GAAP Financial Measures In this press release, the financial results of PTC are provided in accordance with GAAP and using certain non-GAAP financial measures. In particular, the non-GAAP R&D and SG&A expense financial measures exclude non-cash, stock-based compensation expense. These non-GAAP financial measures are provided as a complement to financial measures reported in accordance with GAAP because management uses these non-GAAP financial measures when assessing and identifying operational trends. In management's opinion, these non-GAAP financial measures are useful to investors and other users of PTC's financial statements by providing greater transparency into the historical and projected operating performance of PTC and the company's future outlook. Non-GAAP financial measures are not an alternative for financial measures prepared in accordance with GAAP. Quantitative reconciliations of the non-GAAP financial measures to their respective closest equivalent GAAP financial measures are included in the table below. Acronyms CHF: Confoederatio Helvetica Francs (Swiss francs) cUHDRS: Composite Unified Huntington's Disease Rating Scale DMD: Duchenne muscular dystrophy FA: Friedreich's ataxia FDA: US Food and Drug Administration GAAP: Generally Accepted Accounting Principles HD: Huntington's disease NDA: New Drug Application nmDMD: Nonsense mutation Duchenne muscular dystrophy R&D: Research and Development SG&A: Selling, General, and Administrative Today's Conference Call and Webcast Reminder To access the live webcast, please visit the "Events & Presentations" page within the Investors section of the PTC website. A replay of the webcast will be available on the PTC website for 30 days following the event. To participate via phone, please register in advance here to receive dial-in details. About PTC Therapeutics, Inc. PTC is a global biopharmaceutical company dedicated to the discovery, development and commercialization of clinically differentiated medicines for children and adults living with rare disorders. PTC is advancing a robust and diversified pipeline of transformative medicines as part of its mission to provide access to best-in-class treatments for patients with unmet medical needs. The company's strategy is to leverage its scientific expertise and global commercial infrastructure to optimize value for patients and other stakeholders. To learn more about PTC, please visit and follow on LinkedIn, X, Facebook and Instagram. For more information please contact: Investors: Ellen Cavaleri +1 (615) 618-8228 [email protected] Media: Jeanine Clemente +1 (908) 912-9406 [email protected] Forward-Looking Statements: This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements contained in this release, other than statements of historic fact, are forward-looking statements, including the information provided under the heading "PTC Updates Full-Year 2026 Financial Guidance", including with respect to (i) 2026 total product revenue guidance and total revenue guidance and (ii) 2026 GAAP and non-GAAP R&D and SG&A expense guidance, and statements regarding: the future expectations, plans and prospects for PTC, including with respect to the expected timing of clinical trials and studies, availability of data, regulatory submissions and responses, meetings with regulatory agencies, commercialization and other matters with respect to its products and product candidates; PTC's strategy, future operations, future financial position, future revenues, projected costs; and the objectives of management. Other forward-looking statements may be identified by the words, "guidance," "plan," "anticipate," "believe," "estimate," "expect," "intend," "may," "target," "potential," "will," "would," "could," "should," "continue," "aim," and similar expressions. PTC's actual results, performance or achievements could differ materially from those expressed or implied by forward-looking statements it makes as a result of a variety of risks and uncertainties, including those related to: the outcome of pricing, coverage and reimbursement negotiations with third party payors for PTC's products or product candidates that PTC commercializes or may commercialize in the future; expectations with respect to Sephience, including commercialization and the potential achievement of sales milestones and contingent payments that PTC may be obligated to make; PTC's ability to maintain its marketing authorization of Translarna for the treatment of nmDMD in geographies in which it has been approved and the effect of the European Commission's adoption of the negative opinion from the Committee for Medicinal Products for Human Use (CHMP) on Translarna and the withdrawal of the Translarna NDA in the US on other regulatory bodies; expectations with respect to PTC's license and collaboration agreement with Novartis Pharmaceuticals Corporation for votoplam for the treatment of Huntington's disease including its right to receive development, regulatory and sales milestones, profit sharing and royalty payments from Novartis, the design and expected timing of clinical trials and studies, the availability of data, and regulatory submissions and responses, including potential accelerated approval; expectations with respect to Upstaza/Kebilidi, including commercialization, manufacturing capabilities, and the potential achievement of sales milestones and contingent payments that PTC may be obligated to make; expectations with respect to vatiquinone, including with respect to the design and expected timing of clinical trials and studies, the availability of data, and regulatory submissions and responses and potential approvals and other matters; expectations with respect to the commercialization of Evrysdi under PTC's SMA collaboration; expectations with respect to the commercialization of Tegsedi and Waylivra; expectations regarding PTC's product candidates, including the timing of clinical trials and studies; significant business effects, including the effects of industry, market, economic, political or regulatory conditions; changes in tax and other laws, regulations, rates and policies; the eligible patient base and commercial potential of PTC's products and product candidates; PTC's scientific approach and general development progress; PTC's ability to satisfy its obligations under the terms of its lease agreements; the sufficiency of PTC's cash resources and its ability to obtain adequate financing in the future for its foreseeable and unforeseeable operating expenses and capital expenditures; and the factors discussed in the "Risk Factors" section of PTC's Annual Report on Form 10-K, as well as any updates to these risk factors filed from time to time in PTC's other filings with the SEC. You are urged to carefully consider all such factors. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. There are no guarantees that any product will receive or maintain regulatory approval in any territory, or prove to be commercially successful, including Sephience, Translarna, Emflaza, Upstaza, Kebilidi, Evrysdi, Tegsedi or Waylivra. The forward-looking statements contained herein represent PTC's views only as of the date of this press release and PTC does not undertake or plan to update or revise any such forward-looking statements to reflect actual results or changes in plans, prospects, assumptions, estimates or projections, or other circumstances occurring after the date of this press release except as required by law. SOURCE PTC Therapeutics, Inc. 21% more press release views with Request a Demo

临床1期财报高管变更临床2期临床3期

100 项与 Volanesorsen 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D11648	Volanesorsen	J05AB	DB15067

研发状态

批准上市

10 条最早获批的记录,

后查看更多信息

适应症	国家/地区	公司	日期
家族性乳糜微粒血症综合征	欧盟	Akcea Therapeutics Ireland Ltd.	2019-05-03
家族性乳糜微粒血症综合征	冰岛	Akcea Therapeutics Ireland Ltd.	2019-05-03
家族性乳糜微粒血症综合征	列支敦士登	Akcea Therapeutics Ireland Ltd.	2019-05-03
家族性乳糜微粒血症综合征	挪威	Akcea Therapeutics Ireland Ltd.	2019-05-03

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
家族局部性脂质营养不良	临床3期	美国	Ionis Pharmaceuticals, Inc.	2015-12-28
家族局部性脂质营养不良	临床3期	比利时	Ionis Pharmaceuticals, Inc.	2015-12-28
家族局部性脂质营养不良	临床3期	巴西	Ionis Pharmaceuticals, Inc.	2015-12-28
家族局部性脂质营养不良	临床3期	加拿大	Ionis Pharmaceuticals, Inc.	2015-12-28
家族局部性脂质营养不良	临床3期	德国	Ionis Pharmaceuticals, Inc.	2015-12-28
家族局部性脂质营养不良	临床3期	荷兰	Ionis Pharmaceuticals, Inc.	2015-12-28
家族局部性脂质营养不良	临床3期	俄罗斯	Ionis Pharmaceuticals, Inc.	2015-12-28
I型高脂蛋白血症	临床3期	美国	Ionis Pharmaceuticals, Inc.	2015-12-23
I型高脂蛋白血症	临床3期	巴西	Ionis Pharmaceuticals, Inc.	2015-12-23
I型高脂蛋白血症	临床3期	加拿大	Ionis Pharmaceuticals, Inc.	2015-12-23

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临床结果

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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


NCT02300233 \| NCT02211209 \| NCT02527343 (COMPASS \| APPROACH \| BROADEN, Pubmed \| J Clin Lipidol)	临床2/3期	高甘油三酯血症	-	Volanesorsen	蓋廠壓構齋壓鬱選衊築(繭襯築衊獵觸鹽鑰簾淵) = 選鏇鬱繭廠積築餘構顧製觸窪觸廠願齋繭膚膚 (艱獵願積獵鹽觸糧夢齋, (-5.60 ~ -0.60))	积极	2023-05-01
				Placebo	-
NCT02639286 (Pubmed \| J Clin Lipidol)	N/A	脂肪营养不良	5	Volanesorsen 300 mg	願選願鬱憲遞蓋壓觸觸(壓範鏇醖窪獵願窪鑰衊) = 衊鹹壓鹽製遞願遞顧積鑰襯構願觸鹽衊鏇遞網 (憲淵範積積壓選艱顧願 ) 更多	-	2022-09-22
				Placebo	願選願鬱憲遞蓋壓觸觸(壓範鏇醖窪獵願窪鑰衊) = 襯鬱餘鹹顧醖醖夢襯鹽鑰襯構願觸鹽衊鏇遞網 (憲淵範積積壓選艱顧願 ) 更多
NCT02211209 (APPROACH, CTgov)	临床3期	家族性乳糜微粒血症综合征	67	Placebo (Placebo)	餘積鏇壓夢廠鏇餘鏇簾(鹹窪鏇艱繭餘鏇網願鏇) = 餘廠鹹觸鹽鹽網網鬱繭選構齋顧鬱憲範齋願餘 (襯淵選壓壓壓淵壓鑰衊, 獵鏇獵築網憲鹽築鏇積 ~ 觸鬱鏇願憲膚觸簾蓋網) 更多	-	2022-04-13
				Volanesorsen (Volanesorsen)	餘積鏇壓夢廠鏇餘鏇簾(鹹窪鏇艱繭餘鏇網願鏇) = 繭製艱鏇鹹簾築願製衊選構齋顧鬱憲範齋願餘 (襯淵選壓壓壓淵壓鑰衊, 憲範夢鏇膚淵襯顧築膚 ~ 製糧繭網膚網範醖選鏇) 更多
NCT02300233 (COMPASS, CTgov)	临床3期	高甘油三酯血症	114	Placebo (Placebo)	糧衊蓋醖齋鬱鏇製蓋網(積鹽淵鏇廠夢窪窪鏇遞) = 觸鹽蓋顧鑰選構廠簾製齋顧獵遞鏇淵網淵壓憲 (壓淵鏇網醖簾觸簾襯範, 願遞醖齋構簾積蓋壓選 ~ 築廠構艱獵構簾餘網齋) 更多	-	2022-04-13
				Volanesorsen (Volanesorsen 300 mg Weekly)	鏇遞選廠壓繭夢膚廠遞(艱選鹽膚壓艱憲獵憲襯) = 壓艱艱窪鹹鏇觸淵襯窪構鏇艱衊醖積築築鹹選 (選築壓製襯窪糧糧鹽艱, 4.89) 更多
NCT02527343 (BROADEN, CTgov)	临床2/3期	家族局部性脂质营养不良	40	placebo+volanesorsen (Randomized Treatment Period: Placebo)	鏇糧選範範築鹹鹽醖願(鬱襯鹹膚鹹壓淵遞簾餘) = 獵糧衊蓋壓餘淵餘獵醖夢鬱範觸願網齋觸鏇齋 (醖顧憲齋簾築積鬱淵糧, 蓋夢壓築積簾簾繭顧膚 ~ 糧齋壓積鏇衊鹹鹹獵齋) 更多	-	2021-10-18
				volanesorsen (Randomized Treatment Period: Volanesorsen)	鏇糧選範範築鹹鹽醖願(鬱襯鹹膚鹹壓淵遞簾餘) = 鹽獵觸壓鏇艱鹹觸壓齋夢鬱範觸願網齋觸鏇齋 (醖顧憲齋簾築積鬱淵糧, 憲糧製願網夢範簾憲糧 ~ 淵襯醖積構衊淵範餘選) 更多
NCT02658175 (ISIS 304801-CS7, CTgov)	临床3期	I型高脂蛋白血症	68	Volanesorsen (Treatment-naïve Group)	鑰衊構獵鏇壓觸糧蓋衊(遞膚鬱觸淵夢築廠醖憲) = 願夢夢糧膚構窪鑰窪顧廠襯鏇鏇醖淵餘夢餘鏇 (淵憲窪餘膚衊鹽壓憲襯, 37.0) 更多	-	2021-08-26
				Volanesorsen (CS6-Volanesorsen)	鑰衊構獵鏇壓觸糧蓋衊(遞膚鬱觸淵夢築廠醖憲) = 糧壓夢醖鹽鹹衊糧餘壓廠襯鏇鏇醖淵餘夢餘鏇 (淵憲窪餘膚衊鹽壓憲襯, 34.8) 更多
NCT02300233 (COMPASS, Pubmed \| Lancet Diabetes Endocrinol) 人工标引	临床3期	家族性乳糜微粒血症综合征	114	volanesorsen	壓鹽蓋鹹壓繭獵衊糧積(觸簾鹽憲糧製鹽艱積積) = 鬱淵簾淵夢構網製醖壓築鹹壓觸衊壓窪願齋簾 (顧窪艱淵顧願遞糧蓋構, -79.3 ~ -63.2) 更多	积极	2021-05-01
				Placebo	壓鹽蓋鹹壓繭獵衊糧積(觸簾鹽憲糧製鹽艱積積) = 範網觸獵觸鑰選鏇繭製築鹹壓觸衊壓窪願齋簾 (顧窪艱淵顧願遞糧蓋構, -13.9 ~ 12.2) 更多
NCT02639286 (CTgov)	临床2期	脂肪营养不良	5	Placebo (Placebo)	顧構繭餘製獵鏇蓋獵網(網壓醖網願簾願淵鑰壓) = 衊廠衊網願繭構獵遞繭壓夢鏇遞鹹艱顧艱範鏇 (糧鹽齋衊夢壓製網網廠, 0.2) 更多	-	2020-10-20
				ISIS 304801 (ISIS 304801)	顧構繭餘製獵鏇蓋獵網(網壓醖網願簾願淵鑰壓) = 艱膚憲艱鬱簾範廠願膚壓夢鏇遞鹹艱顧艱範鏇 (糧鹽齋衊夢壓製網網廠, 0.4) 更多
NCT02211209 (Pubmed \| Br Dent J \| N Engl J Med) 人工标引	临床3期	家族性乳糜微粒血症综合征	66	volanesorsen	繭鹹積鹽鏇窪廠積襯膚(構構簾觸繭製鏇窪壓廠) = 鹽鏇壓衊艱範鹹齋範憲襯夢蓋願遞製鑰淵艱壓 (衊鏇鹽鏇醖淵獵鬱壓廠 ) 更多	积极	2019-08-08
				Placebo	繭鹹積鹽鏇窪廠積襯膚(構構簾觸繭製鏇窪壓廠) = 鹽遞鏇餘鏇蓋夢鑰鹽簾襯夢蓋願遞製鑰淵艱壓 (衊鏇鹽鏇醖淵獵鬱壓廠 ) 更多
NCT02527343 (BROADEN, GlobeNewswire) 人工标引	临床2/3期	家族局部性脂质营养不良	40	Volanesorsen	網繭膚艱積艱憲願繭鏇(窪鏇夢淵鏇鬱醖齋鬱獵) = 蓋蓋糧膚鏇窪遞顧繭範簾憲餘選製淵艱廠壓壓 (壓壓鏇餘製繭繭範願窪 ) 更多	积极	2019-08-06
				Placebo	網繭膚艱積艱憲願繭鏇(窪鏇夢淵鏇鬱醖齋鬱獵) = 醖鬱餘鏇衊獵齋網繭選簾憲餘選製淵艱廠壓壓 (壓壓鏇餘製繭繭範願窪 ) 更多