ARC-520

火星人

好消息，关注

bingdian1201

期待二期实验

hxzhu

许久不来论坛，终于看到了好消息。

齐欢畅2

顶

StephenW

5 Feb 2014
Extracts

Arrowhead's CEO Discusses F1Q 2014 Results - Earnings Call Transcript

Dr. Christopher Anzalone - President and CEO:

In November, we applied for regulatory approval in Hong Kong to conduct a single-dose Phase 2a study in patients with chronic hepatitis B. The two sites NPIs are well-known international KOL that have conducted many HBV trials. Ethics committees from both sites have approved our protocol and all necessary preparation -- preparations are completed.

We are waiting for final approval from the Hong Kong Department of Health to begin the study. Communications have been positive and we believe that we will receive approval and begin treating patients this quarter.

We're currently planning two dose groups of eight patients each and we expected to enroll quickly. We believe both cohorts will be at effective dose levels in our primary endpoint, our safety and tolerability as well as depth and duration of s-antigen knockdown.

We believe that dosing portion will be complete in the second quarter and we will follow patients until s-antigen levels return to baseline. While we cannot predict how long the duration of effect will be, we believe that topline data should be available sometime during the summer.

Our plan is to provide these data via press release and then present a full data set at a scientific meeting. We have a high degree of confidence that the Phase 2a will be successful. The Phase 1 suggested that we have a safe and well-tolerated drug at all dose study.

We saw no dose limiting toxicities. So we do not expect any safety concerns in the Phase 2a. We have generated a substantial amount of data in multiple animal models indicating highly protein knockdown. For instance, we publish data from electrotherapy describing ARC-520 administration in rodent HBV models that lead to three to four logs of greater than 99.9% knockdown of HBV gene products.

We have generated data in non-human primates using other siRNAs sequences demonstrating similar results. Most recently, we reported deep and durable knockdown in a chimpanzee with chronic HBV. Should the 2a -- should the 2a work out as we hope, it will represent a great leap forward in HBV research and potential treatment.

We believe it will be the first time anyone will have demonstrated consistent s-antigen reduction, which is thought to be a critical step in reaching a functional cure. Based on a Phase 2a data, we plan to move into a multi-dose base 2b in the second half of this year.

Preparations for this much longer study are underway and they include completion of our second external GMP manufacturing run, chronic GLP toxicology studies in multiple species, site and investigator recruitment, protocol development and applications for regulatory approval.

The Phase 2b will be a multinational study and will likely include sites in the U.S., Europe and Asia. We will be designing the trial to provide readout on ARC-520’s ability to achieve functional cures among other outcome measures.

As you can see, 2014 is an important year for ARC-520 and we expect substantial value to be created around this program. 2014 is also about leveraging ARC-520 to de-risk the DPC delivery platform and broadening out our pipeline.

So think of ARC-520 as the candidate drives value directly and as a proxy for other liver-based candidates. Our friends at Alnylam did the same thing with their TTR program over the past year and a half. We've demonstrated that DPCs are safe and well tolerated in humans and we hope that by this summer, we will have demonstrated that they are capable of inducing efficient, deep and durable gene products knockdown in humans.

StephenW

箭头的CEO讨论F1Q 2014个结果 - 收入电话抄本

克里斯托弗博士斯阿恩扎洛尼 - 总裁兼首席执行官：

在十一月，我们在香港申请监管部门的批准，进行单剂量2a期研究治疗慢性乙型肝炎的两个站点非营利机构已经进行了许多乙肝病毒试验国际知名KOL 。从两个站点伦理委员会已经批准了我们的方案和所有必要的准备 - 准备工作完成。

我们正在等待从健康的香港部门的最终批准，开始研究。沟通是积极的，我们相信我们将获得批准，并开始治疗的患者，本季度。

目前，我们正在筹划的每名患者2个剂量组，我们预计将很快加入。我们相信这两个同伙将在我们的主要终点，我们的安全性和耐受以及深度和表面抗原击倒时间有效剂量水平。

我们相信，定量给料部分将完成在第二季度，我们将按照患者直到表面抗原水平恢复至基线。虽然我们无法预测效果的持续时间会待多久，我们认为，在夏季营收数据应提供的某个时候。

我们的计划是通过新闻稿提供这些数据，然后呈现一个完整的数据集在一个科学会议。我们有高度的信心， 2a期一定会成功。第一阶段的建议，我们有一个安全和耐受性良好的药物在所有剂量的研究。

我们看到没有剂量限制性毒性。所以，我们不要指望在阶段2a任何安全问题。我们已经生成的显示高度蛋白敲除多种动物模型数据的大量。例如，我们发布描述ARC- 520管理在啮齿类动物模型乙肝导致的乙肝病毒基因产物大于99.9 ％击倒三到四原木电疗数据。

我们已经使用其他的siRNA序列，证明了相似的结果产生在非人类灵长类动物的数据。最近，我们在慢性HBV黑猩猩报告深刻而持久的击倒。假如甲 - 应该2A制定出我们希望，这将是一个巨大的飞跃乙肝病毒的研究和潜在的治疗。

我们相信这将是第一次有人会表现出一致的表面抗原减少，这被认为是在达到功能治愈的关键一步。根据2a期数据，我们计划进入今年下半年多剂量基地2B 。

筹备这一更长的研究正在进行中，它们包括我们的第二个外部GMP生产运行，在多个物种，现场调查和招聘，协议开发和监管部门的批准申请慢性GLP毒理学研究的完成。

阶段2b将是一个多国研究，并可能包括在美国，欧洲和亚洲的网站。我们将设计试验，以提供读出的ARC- 520的实现以及其他成果的措施功能的治疗能力。

正如你可以看到， 2014年的ARC- 520的重要一年，我们期望能够解决这个程序创建的重大价值。 2014年也即将利用ARC- 520去风险DPC交付平台，拓宽了我们的管道。

因此，认为ARC- 520作为直接的候选驱动器的价值和其他肝脏为基础的考生的代理。我们在Alnylam公司的朋友们与他们的TTR程序同样的事情在过去一年半的时间。我们已经证明， DPC的是安全和很好耐受的人，我们希望通过这个夏天，我们将证明，他们能够诱导击倒在人类高效，深入，持久的基因产物。

冰冻的心

期待着。

恰饭了某

效果这么好啊，无限期待。

银色黎明

期待人体试验能有好的效果！！

9病成医

这样的实验，应该跟朝鲜、古巴等国的政府合作，效率会高得多。多少人在急等结果？