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.
Response: There is absolutely no doubt in my mind that a deal ia coming sooner than we think. Why? Dr. A said, starting with "I don't lose any sleep over it....access to multiple sources of non-dilutive capital." On the Feb 9 CC. That was a SHARP contrast to what he said on the 12/14 CC. Then he made his multiple comments at the ASM discussing non-dilutive capital, actually NAMING three partners. If he is in any way being disingenuous, they would get a Wells Notice in a nano-second. Dr. A. is a VERY savy person and there is simply NO WAY he would say that unless he were looking in the rear view mirror. I suspect they have a signed joint DPC+outside pharma drug development agreement with an upfront payment and milestones. I further believe that they can run several of these development programs in parallel. Maybe the first payment is contingent upon program launch. But think about it. If they aren't adding new pipeline drugs until recapitalized and yet they are hiring droves of new chemists, what are they all going to be working on? Recall the chemistry of DPCs can be optimized to maximum targeting efficiency. That's not molecular biology. That's chemistry. Hence the hiring of more chemists to optimize .ARWR DPCs with partner pharma drugs.
BTW...this is not a revolutionary concept. Both Tigercapital and I (and I think HP) posted about this possibility nine months or so ago. I pinged a friend at the company to inquire. His response was, "Sure, if someone shows up with a pile of cash we would do it but that hasn't happened to date and we are really focused on our core pipeline. So what's changed??? Someone showed up with a pile of money!!!
I would add that I was very gratified to hear that Dr.A said they would prefer not to partner the HBV platform until they produce more data to give them negotiating leverage. Said that nobody is going to pay them what it is worth until they generate some more data. Monarch dose 4 coming 10 days or so from Monday.
It's cold Juniper Juice in a triangle glass time at Camp Holden.
Post by marcpuckett on Mar 11, 2016 22:09:20 GMT -5
From your lips to God's ears. ....
You will recall it was I who elicited the 'not losing sleep' comment during the earnings call....he used the same expression in response to another question during the annual meeting.
He also reiterated his option of building a sales force for the US and Europe if needed. While he better have something to sell, he also better have some capital to work with in that event.
No insider trading, no volume is what leads me to see news soon.