Arrowhead Pharmaceuticals Receives FDA Clearance to Begin Phase 2/3 Stu

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dyArrowhead Pharmaceuticals Receives FDA Clearance to Begin Phase 2/3 Stu of ARO-AAT for Treatment of Alpha-1 Liver Disease
Apr 15, 2019 at 7:30 AM EDT
PASADENA, Calif. --(BUSINESS WIRE)--Apr. 15, 2019-- Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced that it has received clearance from the U.S. Food and Drug Administration to proceed with an adaptive Phase 2/3 trial with the potential to serve as a pivotal registrational study of
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PASADENA, Calif.--(BUSINESS WIRE)--Apr. 15, 2019-- Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced that it has received clearance from the U.S. Food and Drug Administration to proceed with an adaptive Phase 2/3 trial with the potential to serve as a pivotal registrational study of ARO-AAT, the company’s second generation subcutaneously administered RNA interference (RNAi) therapeutic being developed as a treatment for a rare genetic liver disease associated with alpha-1 antitrypsin deficiency (AATD).

Arrowhead intends to initiate the adaptive design, Phase 2/3 study of ARO-AAT in patients with AATD associated liver disease at various sites in the U.S. in the second quarter of 2019, followed by various international sites in Europe, pending regulatory submission and review. The proposed primary objectives are to evaluate safety and pharmacodynamic dose response, and to evaluate efficacy, defined as an improvement in a histologic grading scale of AATD associated liver disease, and no worsening of liver fibrosis based on Ishak score on end of study biopsy. The company plans to provide additional study details following its initiation.

About Arrowhead Pharmaceuticals

Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them. Using a broad portfolio of RNA chemistries and efficient modes of delivery, Arrowhead therapies trigger the RNA interference mechanism to induce rapid, deep, and durable knockdown of target genes. RNA interference, or RNAi, is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a specific protein. Arrowhead’s RNAi-based therapeutics leverage this natural pathway of gene silencing.

For more information, please visit www.arrowheadpharma.com, or follow us on Twitter @ArrowheadPharma. To be added to the Company's email list and receive news directly, please visit http://ir.arrowheadpharma.com/email-alerts.

Safe Harbor Statement under the Private Securities Litigation Reform Act:

This news release contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. These statements are based upon our current expectations and speak only as of the date hereof. Our actual results may differ materially and adversely from those expressed in any forward-looking statements as a result of various factors and uncertainties, including the safety and efficacy of our product candidates, the duration and impact of regulatory delays in our clinical programs, our ability to finance our operations, the likelihood and timing of the receipt of future milestone and licensing fees, the future success of our scientific studies, our ability to successfully develop and commercialize drug candidates, the timing for starting and completing clinical trials, rapid technological change in our markets, and the enforcement of our intellectual property rights. Our most recent Annual Report on Form 10-K and subsequent Quarterly Reports on Form 10-Q discuss some of the important risk factors that may affect our business, results of operations and financial condition. We assume no obligation to update or revise forward-looking statements to reflect new events or circumstances.

Source: Arrowhead Pharmaceuticals, Inc.



View source version on businesswire.com: https://www.businesswire.com/news/home/20190415005133/en/

Source: Arrowhead Pharmaceuticals Inc.

Contacts:
Arrowhead Pharmaceuticals, Inc.
Vince Anzalone, CFA
626-304-3400
[email protected]

Investors and Media:
LifeSci Advisors, LLC
Brian Ritchie
212-915-2578
[email protected]
www.lifesciadvisors.com

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Arrowhead Presents Clinical Data on JNJ-3989 (ARO-HBV) at The International Liver Congress™
Apr 12, 2019 at 12:45 PM EDT
PASADENA, Calif. --(BUSINESS WIRE)--Apr. 12, 2019-- Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced the presentation of clinical data from an ongoing Phase 1/2 study (AROHBV1001) of JNJ-3989 (formerly ARO-HBV), a third-generation subcutaneously administered RNA interference (RNAi)
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PASADENA, Calif.--(BUSINESS WIRE)--Apr. 12, 2019-- Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced the presentation of clinical data from an ongoing Phase 1/2 study (AROHBV1001) of JNJ-3989 (formerly ARO-HBV), a third-generation subcutaneously administered RNA interference (RNAi) therapeutic candidate being developed as a potential treatment for patients with chronic hepatitis B virus (HBV) infection, at The International Liver Congress™ 2019 (ILC), the annual meeting of the European Association for the Study of the Liver (EASL).

Arrowhead entered into a license agreement in October 2018 with Janssen Pharmaceuticals, Inc., part of the Janssen Pharmaceutical Companies of Johnson & Johnson, to develop and commercialize ARO-HBV.

Key results from this interim analysis include the following:

JNJ-3989 rapidly reduced hepatitis B surface antigen (HBsAg) in patients that had 24 weeks or more of HBsAg assay results (n=40) to thresholds possibly associated with improved chances of HBsAg seroclearance1 in many patients, after only 3 doses
100% of patients (40 of 40) achieved ≥1.0 Log10 IU/mL HBsAg reduction
88% of patients (35 of 40) achieved HBsAg <100 IU/mL
43% of patients (17 of 40) achieved HBsAg <10 IU/mL
13% of patients (5 of 40) achieved HBsAg <1 IU/mL
JNJ-3989 reduced all measurable viral products, including HBsAg in hepatitis B e-antigen (HBeAg) positive or HBeAg negative patients
JNJ-3989 administered subcutaneously was well tolerated at doses up to 400 mg in all chronic hepatitis B (CHB) patients in cohorts 2b-11 (n=56)
168 total doses administered to 56 CHB patients (cohorts 2b through 11)
No drug related serious adverse events (SAE) reported
Unrelated SAE of menorrhagia
Unrelated SAE of anxiety/depression
All patients received all 3 scheduled doses; No dropouts
No dose related pattern of adverse changes in laboratory values (e.g. ALT, AST, total bilirubin, creatinine)
17 total AEs at injection site (10% of injections) reported (e.g. erythema, tenderness, bruising), all were mild
Oral Presentation Details:

Short term RNA interference (RNAi) therapy in chronic hepatitis B (CHB) using JNJ-3989 brings majority of patients to HBsAg <100 IU/ml

Presentation Reference: PS-080
Session: Parallel session: Hepatitis B - drug development
Session Date and Time: April 12, 2019 at 5:45 p.m. CET
Authors: Man-Fung Yuen, et al.
Additional details, including the presentation abstract, can be found on the ILC website at https://ilc-congress.eu/. A copy of presentation materials can be accessed by visiting the Events section under the Investors tab of the Arrowhead website.

AROHBV1001 (NCT03365947) is a Phase 1/2 clinical study evaluating the safety, tolerability, and pharmacokinetic effects of single-ascending doses (SAD) of ARO-HBV in healthy adult volunteers, as well as the safety, tolerability, and pharmacodynamic effects of multiple-ascending doses (MAD) of ARO-HBV in patients with chronic HBV.

Hepatitis B infection is a life-threatening viral infection of the liver, which can cause cirrhosis — scarring of liver tissue — and liver cancer if the infection becomes chronic. The World Health Organization cites that hepatitis B is a global public health problem with 257 million people living with the disease, resulting in 887,000 deaths in 2015.2 While a preventive vaccine is available, cure rates for those infected remain low and most patients will endure lifelong therapy.

newchinabok

hp121

哪位说说啥内容呢

泡泡苹苹

Arrowhead于
2019年4月12日美国东部时间下午12:45在
加利福尼亚州帕萨迪纳市举行的国际肝病大会上向JNJ-3989（ARO-HBV）提供临床数据- （美国商业资讯） - 4月。Arrowhead Pharmaceuticals，Inc。（纳斯达克股票代码：ARWR）今天宣布，正在进行的第1/2期研究（AROHBV1001）JNJ-3989（原ARO-HBV）的临床数据报告，第三代皮下注射RNA干扰（RNAi）
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加利福尼亚州帕萨迪纳 - （美国商业资讯） - 4月 Arrowhead Pharmaceuticals，Inc。（纳斯达克股票代码：ARWR）今天宣布，正在进行的第1/2期研究（AROHBV1001）JNJ-3989（原ARO-HBV）的临床数据报告，第三代皮下注射RNA干扰（RNAi）治疗候选药物被开发为慢性乙型肝炎病毒（HBV）感染患者的潜在治疗药物，参加国际肝病大会2019年（ILC），欧洲肝脏研究协会年会（ EASL）。

Arrowhead于2018年10月与Janssen Pharmaceuticals，Inc。（强生公司的Janssen制药公司的一部分）签订了许可协议，以开发和商业化ARO-HBV。

此中期分析的主要结果如下：

JNJ-3989迅速降低了HBsAg检测结果24周或更长时间（n = 40）患者的乙型肝炎表面抗原（HBsAg）阈值可能与许多患者HBsAg血清清除率增加1相关，仅3次给药后
100％患者（40人中的40人）达到≥1.0Log10IU / mL HBsAg减少
88％的患者（40人中的35人）达到HBsAg <100 IU / mL
43％的患者（17人中40人）达到HBsAg <10 IU / mL
13％患者（40
人中的5人）达到HBsAg <1 IU / mL JNJ-3989减少所有可测量的病毒产品，包括乙型肝炎e抗原（HBeAg）阳性的HBsAg或
皮下注射的HBeAg阴性患者JNJ-3989在剂量高达所有慢性乙型肝炎（CHB）患者中有400 mg患者为2b-11（n = 56）
向56名CHB患者（组合2b至11）施用168次总剂量
没有药物相关的严重不良事件（SAE）报告
月经过多的
SAE与焦虑/抑郁的无关SAE
所有患者均接受了所有3个预定剂量; 没有辍学
没有剂量相关的实验室值的不利变化模式（如ALT，AST，总胆红素，肌酐）
17注射部位的总AE（10％的注射）报告（如红斑，触痛，瘀伤），均为轻度
口服表现详细信息：

使用JNJ-3989对慢性乙型肝炎（CHB）进行短期RNA干扰（RNAi）治疗，使大多数患者的HBsAg <100 IU / ml

表现参考：PS-080
会议：平行会议：乙型肝炎 - 药物开发
会议日期和时间：2019年4月12日下午5:45 CET
作者：Man-Fung Yuen，et al。
其他详细信息，包括演示摘要，可在ILC网站https://ilc-congress.eu/上找到。可以通过访问Arrowhead网站“投资者”选项卡下的“活动”部分访问演示材料的副本。

AROHBV1001（NCT03365947）是一项1/2期临床研究，评估ARO-HBV单次递增剂量（SAD）对健康成年志愿者的安全性，耐受性和药代动力学作用，以及安全性，耐受性和药效学作用。慢性HBV患者ARO-HBV的多次递增剂量（MAD）。

乙型肝炎感染是一种危及生命的肝脏病毒感染，如果感染变成慢性，可导致肝硬化 - 肝组织瘢痕形成 - 和肝癌。世界卫生组织援引乙型肝炎是一个全球公共卫生问题，有2.57亿人患有这种疾病，2015年造成887,000人死亡.2虽然可以获得预防性疫苗，但感染者的治愈率仍然很低，大多数患者将忍受终身治疗。

mingbai

效果不错，增加用药次数应该更确切降表面抗原吧？

newchinabok

mingbai 发表于 2019-4-18 18:52
效果不错，增加用药次数应该更确切降表面抗原吧？

做了试验，曾加频率不改变降hbsag

喜从天降

效果很好了。小于 100后打长效干扰素得金牌概率极高。我在干扰素吧观察到有大量的500以下打干扰素转阴的帖子，就算打不出100多的抗体，打成10以内的表抗没有压力。