箭头收到监管机构的批准，开始2B乙肝候选ARC-520的研究附加

StephenW

Arrowhead Receives Regulatory Clearance to Begin Additional Phase 2b Studies of Hepatitis B Candidate ARC-520


    PASADENA, Calif.--(BUSINESS WIRE)--      Arrowhead Research Corporation (NASDAQ: ARWR), a biopharmaceutical      company developing targeted RNAi therapeutics, today announced that its      Clinical Trial Application for ARC-520, its clinical candidate for      chronic hepatitis B infection (HBV), has been approved by Germany's      Federal Institute for Drugs and Medical Devices. The company now has      regulatory clearance in Germany for two additional Phase 2b      multiple-dose studies of ARC-520 to be conducted in parallel. Arrowhead      awaits final IRB approval from the sites, which are expected shortly,      and in addition, the company is engaged with regulatory authorities from      Hong Kong and South Korea to open additional sites for these studies.      Arrowhead will provide guidance in the future on timing for release of      data from these studies.   
          "ARC-520 is leading the way in studying the use of RNAi drugs as new      treatment options for chronic HBV. We are very pleased to receive      regulatory clearance in Europe to conduct the parallel design Phase 2b      multiple-dose studies that we proposed," said Bruce D. Given, M.D.,      Arrowhead's Chief Operating Officer. "We view these studies as the front      end of a set of global multiple-dose studies designed to identify the      right agents, doses, and regimens that can achieve functional cures.      Based on multiple-dose data from our recently disclosed chimpanzee study      and the long duration of activity ARC-520 has demonstrated in the      single-dose Phase 2a study in patients, we are starting with doses of      1mg/kg and 2mg/kg in parallel. We have incorporated an interim analysis      in both studies to get an early read on whether we may want to      interrogate higher doses. ARC-520 continues to be well tolerated and we      have not seen any signal suggesting that we are approaching a maximum      tolerated dose through 4mg/kg, the highest dose studied in humans to      date."   
          The studies titled Heparc-2002 and Heparc-2003 will enroll patients with      chronic HBV infection that are e-antigen (HBeAg) negative and HBeAg      positive, respectively. Each study is a multicenter, randomized,      double-blind, placebo-controlled, multi-dose study intended to evaluate      two dose levels in parallel of ARC-520 in combination with entecavir or      tenofovir in patients with immune active chronic HBV infection. The 2002      and 2003 studies are planned to enroll up to 60 and 90 patients,      respectively. In each study, patients will be randomized to receive four      doses, once every 4 weeks, of either 1 mg/kg ARC-520, 2 mg/kg ARC-520,      or placebo at a ratio of 1:1:1.   
          For each patient, the duration of the study is approximately 33 weeks,      from screening to the Day 169 follow-up visit. For each study, a      planning interim analysis will be performed when 54 patients have      completed their Day 71 visit. This analysis will be used to plan future      studies only and will not impact the enrollment or conduct of      Heparc-2002 and -2003. Based on the interim results, the company will      decide whether higher multiple-doses would be likely to lead to      increased activity, in which case higher doses may be evaluated in one      or more additional studies.   
          The primary objective of the 2002 and 2003 studies is to evaluate the      depth of hepatitis B surface antigen (HBsAg) decline in response to      multiple doses of ARC-520 compared to placebo as a measure of drug      activity. Secondary objectives include the assessment of safety and      tolerability and multi-dose pharmacokinetics of ARC-520 when      co-administered with entecavir or tenofovir, in addition to other      exploratory objectives.   
          About ARC-520   
          Arrowhead's RNAi-based candidate ARC-520 is being investigated in the      treatment of chronic HBV infection. The small interfering RNAs (siRNAs)      in ARC-520 intervene at the mRNA level, upstream of the reverse      transcription process where current standard of care nucleotide and      nucleoside analogues act. Arrowhead is investigating ARC-520      specifically to determine if it can be used to achieve a functional      cure, which is an immune clearant state characterized by hepatitis B      s-antigen negative serum with or without sero-conversion. Arrowhead has      completed a Phase 1 single ascending dose study in normal volunteers and      the company is conducting single dose Phase 2a studies and multiple dose      Phase 2b studies in chronic HBV patients. Approximately 350-400 million      people worldwide are chronically infected with the hepatitis B virus,      which can lead to cirrhosis of the liver and is responsible for 80% of      primary liver cancers globally.   

StephenW

箭头收到监管机构的批准，开始2B乙肝候选ARC-520的研究附加相


加利福尼亚州帕萨迪纳 - （BUSINESS WIRE） - 箭头研究公司（纳斯达克股票代码：ARWR），一家生物制药公司开发有针对性的RNAi疗法，今天宣布其临床试验申请ARC-520，其临床候选慢性乙型肝炎病毒感染（ HBV），已通过德国联邦药物和医疗器械。公司目前拥有的ARC-520的另外两个阶段2b多剂量研究将并行进行的德国监管部门的批准。箭头等待来自网站，这是短期内有望最终批准IRB，此外，该公司是一家从事与来自香港和韩国监管当局打开其他网站进行这些研究。箭头将提供关于这些研究数据发布的时间在未来的指导意见。

“ARC-520一路领先在研究利用RNAi药物作为慢性HBV新的治疗方案。我们很高兴能够获得监管部门的批准在欧洲进行的并行设计阶段2b，我们建议多剂量研究，”说布鲁斯D.考虑，MD，箭头的首席运营官。 “我们认为这些研究为一组全球多剂量研究旨在查明代理权，剂量和治疗方案，可以实现功能治愈。根据我们最近披露的黑猩猩研究和长多剂量数据的前端活动的ARC-520的持续时间已经证明在患者的单剂量2a期研究中，我们已开始与剂量为1mg / kg和为2mg / kg的并联。我们已经并入在这两项研究的中期分析，以得到一个早的读取上我们是否可能想询问较高剂量。ARC-520继续耐受性好，我们还没有看到这表明，我们通过4毫克/千克，研究在人类迄今的最高剂量接近最大耐受剂量的任何信号。“

该研究题为Heparc-2002和Heparc 2003年将招收慢性HBV感染是e抗原（HBeAg）阳性阴性和HBeAg阳性，分别。每个研究是一项多中心，随机，双盲，安慰剂对照，多剂量研究旨在评估患者免疫活性慢性HBV感染两个剂量水平中的ARC-520的平行结合恩替卡韦或替诺福韦。在2002年和2003年的研究计划招收多达60和90例，分别为。在每个研究中，患者将被随机接受4剂量的1mg / kg的ARC-520，2毫克/公斤的ARC-520，或安慰剂，每4周一次，以1：1的比率：1。

对于每一个病人，研究的持续时间约33周，从筛选到169天随访。对于每一个研究中，当54名患者完成了71天的访问规划中期分析将被执行。这一分析将用于只打算未来的研究并不会影响Heparc-2002和-2003入学或行为。根据该中间结果，该公司将决定更高多剂量是否很可能会导致增加的活性，在这种情况下，更高的剂量可以在一个或多个另外的研究进行评估。

在2002和2003年的研究的主要目的是评估响应于多剂量ARC-520的相比，安慰剂作为药物活性的量度的B型肝炎表面抗原（HBsAg）的下降深度。次要目的包括ARC-520的安全性和耐受性和多剂量药代动力学评估当共同施用恩替卡韦或替诺福韦，除了其他试探性的目标。

关于ARC-520

箭头的基于RNAi的候选人ARC-520正在调查慢性HBV感染的治疗。小干扰RNA（siRNA）的ARC-520介入在mRNA水平，反转录过程，其中护理核苷酸的目前标准和核苷类似物起作用的上游。箭头正在调查的ARC-520特异性以确定它是否可用于实现一个功能治愈，这是其特征在于由乙肝表面抗原阴性血清或无血清转化的免疫clearant状态。箭头已经完成了正常的志愿者一期单剂量递增研究，该公司正进行单剂量2a期研究和多次给药阶段2b研究慢性乙肝患者。全世界大约350-400百万人慢性感染了乙肝病毒，其可以导致肝硬化，并负责全球80％的原发性肝癌。

feitianyu

crystalandtiger

2b阶段的结果到底什么时候能够完全公开披露？明年一月份？今年第三季度公布的是哪些数据呢？

newchinabok

加油

四十己过

总算有点小消息，也有个小盼头

相信会幸福

加油！

9病成医

好消息，值得进一步期待。

苏小三

离成功还有一大截距离，原来在央电视台也报道过类似消息，唉！