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.
I wonder why he goes to London for this 20 minutes, to a free show? This show is for retailers. I will be in London so I probably will go there, but there should be something else! I do not think this show is so important to CA for travelling across the ocean.
It has come to my attention that GSK might have an interest in ARWR (most likely for partnering imo) ....after doing more DD on the info and GSK's interest in the RNAi space this sounds like a plausible scenario. Let us know your thoughts regarding the presentation if you attend it.
I already have the ticket to visit the show, but I know he will not tell us any results yet. So I would like to ask CA about his sleeping habit! Does he have better sleep after 4 weeks
I have visited the Master Show, and I had a 10 minute personal conversation with CA. (with Buffet it could cost millions:). So as there was no Q&A section, after the presentation I met him alone because nobody wanted to ask anything, so that is why I call it personal meeting.
So he stated ARWR is a platform company, they intend to sell products. Means they do not want to go alone producing drugs. They are waiting to an offer. He pointed GILD and JnJ and also mentioned Merck. As soon as they have a great offer ARWR will sell ARC-520 (he said "HBV drug"). He of course did not mentioned the offers and the dates but these are the companies which are closely monitoring. Maybe others but he did not say any other names.
He denied any connection between Roche and the new lease place. This is absolute just a coincidence - said CA.
He was very happy when I mentioned the DPC. He knows this is the crown jewel. (but I forgot to ask what is the plan with this diamond, sorry:(
At the first 5 minutes there was a Chinese man. He said he represents a Chinese drug factory, but CA told they do not want to sell directly anything to China, he again mentioned Gilead as they have Chinese connection. (if I understood well)
Here I do not want to say about the presentation you probably will see on ARWR web page soon. ( I don't think there was any new info) They start the ARC 521 in clinic in June 2016 (I think we knew that)
Some highlights from the posters: ARC 520 IND or equivalent june 2016, monotherapy and with interferon actively enrolling now.
Post by retired7211 on Apr 25, 2016 8:02:55 GMT -5
Very curious presentation with update for 2001 OLE saying only "expected enrollment completion in 2016". Isn't it our belief that multi dose is well underway? What was purpose of attending? Something is up or this was a trip for the wife to go shopping in London.
Post by Think2Succeed on Apr 25, 2016 8:40:53 GMT -5
Here's Hparch take on this:
"The two things that I find of the most interest about this are:
1. The apparent deliberate elimination of both Roche and Novartis from the potential partners; not sure whether I read that as a negative or a positive both have vested interests in the success of ARWR and/or DPC produced drugs, and might enjoy watching others pay through the nose for a successful HBV treatment, and see their own pockets lined with no further effort (Novartis with 3m shares in ARWR, Roche with sales based royalties on the drug itself, which will have to sell at higher prices whenever possible to recover competing buying/licensing offers to ARWR at the front end). Of course, he might not have mentioned them to protect their counter offers which he knows will be coming after offers from the 3 he mentioned. 2. The use of the word "sell" instead of license for "HBV drug". At this point, they could only sell ARC-520, as it has the data to back up its performance, and we are awaiting results from 2001e and monarch. That's approximately 80m patients of the world's 400m. "Selling" could mean selling the rights to commercialize", or it could mean literally selling the drug's rights. Each has a different valuation. Anyway, it all depends on the data ARC-520 can generate. If its a slam dunk and FC's cHBV patients in most cases, ARC-521 would likely be included in such a sale, to assure that ARWR couldn't get back into the market with a competing product after the sale.
Things obviously are going well in trials. Im not sure about FC's yet, but, one could theoretically hope for some by now and not be overly anxious, due to Dr. Walsh's disclosure that s-AB complexes were developed in the NUC naive cohorts after only a single dose. That surely perked up the ears of big pharma, no matter who he mentioned - no one is going to ignore a potential 400m patient world wide market.
The next few months are going to be VERY interesting.