ARC's new poster on HBV Meeting 2015

hao2014

在我印象中，恩替是在全球包括中国做了三期

跨国大公司啊

小公司没这实力

齐欢畅2

回复 HBVCURER 的帖子

现在看。联合用药势在必行。我们拭目以待。

guilin2013

回复 HBVCURER 的帖子

如果美国FDA，面对那么多患者的一个群体，这么多患者的迫切需求而无动于冲的话，那他们那届总统都不用当了，更不用说美国FDA的职员等

guilin2013

回复 hao2014 的帖子

小公司没实力，更应来中国做实验，现在全世界有那个国家有那么多患者，有那个国家的人愿意花全部家产都医这个病，只有在中国，在中国，所以这是全世界最大的市场，没有之一，占领这个市场，不单止造福人类，还挣上万亿人民币都有可能，所以说最大问题还是国内管理问题

guilin2013

回复 hao2014 的帖子

若我是一个制药公司老板，就会花有限的资源占领最大的市场，这是基本商业逻辑和规则，除非政治管理问行不通，若同经济角度考虑，就应这样做，所以就好奇怪，国外的好药就是不能在中国最先做实验，最先上市，真是悲哀

齐欢畅2

回复 HBVCURER 的帖子

现在看。联合用药势在必行。我们拭目以待。

guilin2013

回复 hao2014 的帖子

所以说，如果'''REP2139这个药是真是话，就应走快速通道，5年前就应引进中国做实验，说不定效果像他说的那么好的话，若国内监管给力的话，现在已经上市了，大家就解散了，HBV就像感冒一样了

HBVCURER

hao2014 发表于 2015-10-13 23:28
在我印象中，恩替是在全球包括中国做了三期

跨国大公司啊

BMS对CFDA有妥协，允许国内有限度的生产ETV仿制药，算是交了投名状吧，自然给绿灯了。反例是Gilead，非要和CFDA死磕，结果一直拖到14年TDF才被允许上市。当然，Gilead这下也学聪明了，TAF的进程自然也就快的多。

齐欢畅2

Arrowhead Presents Overview of Its Broad RNAi Delivery Platform and Introduces New Subcutaneously Administered Format

PASADENA, Calif.--(BUSINESS WIRE)--

Arrowhead Research Corporation (NASDAQ: ARWR), a biopharmaceutical company developing targeted RNAi therapeutics, today presented an overview of its broad RNAi delivery platform at the Annual Meeting of the Oligonucleotide Therapeutics Society in Leiden, the Netherlands. Included in this presentation was data from a new program, ARC-LPA, which is designed to reduce production of apolipoprotein A [apo(a)], a key component of lipoprotein(a) [Lp(a)]. Lp(a) has been genetically linked with increased risk of cardiovascular diseases, independent of cholesterol and LDL levels. Using a new subcutaneous delivery construct that Arrowhead has developed, an initial lead compound has achieved up to 90% knockdown of apo(a) in mice.

"Our ability to generate RNAi drugs for a wide variety of indications has expanded dramatically with our advances in delivery," said Chris Anzalone, Ph.D., Arrowhead's president and chief executive officer. "We currently have two drugs in the clinic, ARC-520 and ARC-AAT, that validate our capabilities in IV liver delivery and provide additional confidence in follow-on candidates ARC-F12 and ARC-521. We are now able to deliver to tumors, as exemplified by ARC-HIF2, and report data today on subcutaneous administration for liver delivery with ARC-LPA. These capabilities provide broad opportunities to fight diverse diseases and enable us to drive an aggressive pipeline."

In a presentation titled, "Development of RNAi-Based Therapeutics using Dynamic Polyconjugates™ (DPC™) Technology," Bruce Given, M.D., Arrowhead's chief operating officer, discussed three examples of Arrowhead's broad RNAi delivery platform: DPC™ for liver delivery with select data from the ARC-520 clinical program; DPC™ for extra-hepatic delivery with select preclinical data from the ARC-HIF2 program; and, a new proprietary subcutaneous delivery vehicle with initial preclinical data from the ARC-LPA program.

Arrowhead uses DPC™, or Dynamic Polyconjugates™, for liver delivery in both of its clinical stage drugs, ARC-520 for chronic hepatitis B infection and ARC-AAT for alpha-1 antitrypsin deficiency, as well as additional preclinical stage programs, including ARC-F12 for hereditary angioedema and ARC-521 for chronic hepatitis B infection. Arrowhead's delivery platform can produce deep and sustained knockdown of mRNA and proteins, as evidenced by data from the ARC-520 program showing dramatic reductions in hepatitis B e-antigen, core-related antigen, and s-antigen in humans. ARC-520 achieved a maximum reduction of s-antigen of 99% (1.9 log), which is the highest reported single-dose knockdown in humans with any RNAi therapeutic.

DPC™ for extra-hepatic delivery is being deployed in Arrowhead's ARC-HIF2 candidate for the treatment of clear cell renal cell carcinoma (ccRCC). As Arrowhead previously reported, in an orthotopic ccRCC tumor model in mice ARC-HIF2 reduced the expression of HIF2α, a well-validated oncogene in this disease, by more than 80%, leading to statistically significant reductions in tumor size and weight, extensive tumor cell death, reduction in the tumor-expressed VEGF-A biomarker, and destruction of the blood vessels feeding the tumors. DPC™ for extra-hepatic delivery employs a masked polymer designed to induce endosomal escape, with RNAi trigger attached directly to the delivery vehicle. This molecule is actively guided to tumor cells with a proprietary targeting agent that binds to αVβ3 integrin, which is highly expressed on the surface of ccRCC cells as well as various other tumor types.

Dr. Given also introduced Arrowhead's new hepatic delivery format being developed for subcutaneous administration and a new target being explored for cardiovascular disease. The new delivery construct discussed includes an RNAi trigger against apo(a). Apo(a) is a critical component of Lp(a) and effective knockdown results in marked Lp(a) reductions. Single dose knockdown of around 90% in apo(a) has been achieved in mice. Initial data through day 15 suggest a good duration of effect (study ongoing). Similar to the intravenously administered vehicle used for hepatic delivery, this construct employs N-acetyl galactosamine for hepatic targeting. However, it does not employ the same peptide-based active endosomal escape. An active lead compound has been identified and additional lead optimization is ongoing. This program has not been officially designated as entering pre-IND development.

A copy of the presentation can be accessed by visiting the Events section of the company's website at http://ir.arrowheadresearch.com/events.cfm after the presentation concludes.

默然10

其实我觉得，一种新药物而且是舶来品的实验药物，还是要谨慎对待，这个群体这么大，目前大家都太想治愈。
参考《生化危机》
官方应该把握合适的度来引进药物，这个倒是真的。