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About Arrowhead Arrowhead Pharmaceuticals is a biopharmaceutical company developing targeted RNAi therapeutics.
Targeting Innovation
Arrowhead Pharmaceuticals develops novel drugs to treat intractable diseases by silencing the genes that cause them. Using the broadest 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. Arrowhead’s most advanced drug candidate in clinical development is ARC-520, which is designed to treat chronic hepatitis B infection by inhibiting the production of all HBV gene products. The goal is to reverse the immune suppression that prevents the body from controlling the virus and clearing the disease. Arrowhead’s second clinical candidate is ARC-AAT, a treatment for a rare liver disease associated with a genetic disorder that causes alpha-1 antitrypsin deficiency.

Lead Products
ARC-520 is an RNAi-based therapeutic designed to treat chronic hepatitis B virus (HBV) infection. It is the first clinical-stage drug candidate from Arrowhead’s Dynamic Polyconjugate® delivery platform. It is designed to treat chronic HBV infection by reducing the expression and release of new viral particles and key viral proteins with the goal of achieving a functional cure for HBV.

ARC-AAT is a novel unlocked nucleobase analog (UNA)-containing RNAi-based therapeutic for the treatment of liver disease associated with Alpha-1 Antitrypsin Deficiency (AATD), a rare genetic disease that can severely damage the liver and lungs of affected individuals. The goal of treatment with ARC-AAT is to reduce the production of the mutant Z-AAT protein to prevent and potentially reverse accumulation-related liver injury and fibrosis.

The companys pre-clinical stage drug candidates include ARC-521, an RNAi-based therapeutic for the treatment of chronic hepatitis B virus; ARC-F12, an RNAi-based therapeutic to treat hereditary angioedema and thromboembolic diseases; ARC-HIF2, an RNAi-based therapeutic to treat renal cell carcinoma; and ARC-LPA, an RNAi-based therapeutic for the treatment of cardiovascular diseases. It also holds patents related to Adipotide for the treatment of obesity and related metabolic disorders. The company has research collaboration and license agreement with Shire AG to develop and commercialize targeted peptide-drug conjugates.

Platform Delivery Technology
The Dynamic Polyconjugate (DPC®) platform is an RNAi delivery system that has been demonstrated to preferentially deliver to hepatocytes, induce efficient endosomal escape, promote high levels of gene knockdown in multiple animal models, and appears to be well tolerated using a variety of RNAi trigger molecules. It is a modular system that can be optimized on a target-by-target basis and may be targeted in the future to address multiple organ systems and cell types.

Pipeline Development Strategy
Arrowhead’s internal drug pipeline is intended to drive value directly through the clinical development of novel therapeutics and to provide proof of concept for our platform technologies. In addition to our two lead product candidates, ARC-520 and ARC-AAT, we intend to nominate additional clinical candidates that utilize the DPC delivery system. Our core areas of focus for expanding our internal pipeline of RNAi therapeutics are: (1) develop intravenous (IV) administered liver-targeted candidates; (2) develop subcutaneously administered liver-targeted candidates; and (3) explore extra-hepatic targets, including oncology.

To learn more about Arrowhead visit the company's website at: http://arrowheadpharma.com/

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520/521 with cholesterol reducing drug

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qbeing1010 ARWR Junior Member Posts: 68 ARWR shares held: >5 Registered: Nov 25, 2015 8:39:52 GMT -5	520/521 with cholesterol reducing drug Jan 20, 2016 12:36:58 GMT -5 Think2Succeed likes this Quote Select Post Deselect Post Link to Post Member Give Gift Back to Top Post by qbeing1010 on Jan 20, 2016 12:36:58 GMT -5 BioBoyScout tweeted this last week. I can't find the source. Any thoughts?
	[Go to Top] [Go to Bottom] Last Edit: Jan 21, 2016 5:38:19 GMT -5 by Think2Succeed

Think2Succeed
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Sentiment: Strong buy !

Posts: 182
ARWR shares held: 20,000

Registered: Nov 24, 2015 2:03:46 GMT -5

520/521 with cholesterol reducing drug Jan 21, 2016 5:47:03 GMT -5

Quote

Post by Think2Succeed on Jan 21, 2016 5:47:03 GMT -5

I can't find the source either QB however I found a link that confirms that this patent was granted to Arrowhead on Jan 12t, 2016: patents.justia.com/assignee/arrowhead-research-corporation

It's certainly an interesting theory that adding a lowering cholesterol drug would make the path to FC more efficient...however I have no idea how they arrived at this conclusion. Maybe management is going to mention something about this in one the CC's.

T2S

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Bio Boy Scout
ARWR New Member

Posts: 14

Registered: Dec 23, 2015 11:14:28 GMT -5

520/521 with cholesterol reducing drug Jan 21, 2016 23:23:42 GMT -5

Quote

Post by Bio Boy Scout on Jan 21, 2016 23:23:42 GMT -5

As taken directly from the patent application: "by administering a statin drug, the level of competing cholesterol in plasma is reduced and the level of LDL receptors for binding cholesterol-siRNA in the liver are increased. The invention thus provides a method for increased uptake of cholesterol labeled siRNA wherein the siRNA is administered in conjunction with a statin whereby the level of competing cholesterol in the serum is reduced, allowing for more efficient uptake of cholesterol labeled siRNA by hepatocytes. The statin can be administered before, with or after the administration of the cholesterol-siRNA."

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