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.
Today is a disaster and even the YMB is not working. I am unfortunately deep in ARWR and has no clue what will come after all these knews. I was following a lot of great people here and on YMB but I think everybody had a shock what CA said. Can we have a discussion about ARWR future?
I am with you. I am trying to figure out if I should average down and try and get out at a break even. Although a couple of days letting the dust settle is probably the best idea. I would also welcome ideas on what to do.
Post by brokerphil on Nov 29, 2016 20:43:12 GMT -5
I too am crushed today but will also sit tight. Not much of a choice.
I kid you not- At 4:10 PM I was at the traffic light at the corner of Binney and Third Streets in Kendall Square in Cambridge........staring at the Alnylam sign on their HQ when my brother called me to tell me the Arrowhead news. Me, the one who reveled in ALNY's halt; the one who smugly thought our SAE-free trials and last-man-standing trajectory was bullet-proof. I'm a big believer in the "you reap what you sow" mantra and now I sit here in the Hyatt on Memorial Drive looking like a deer in the headlights. Was my location upon hearing the news a coincidence or Karma. Who the hell knows, but it sure is ironic any way you look at it.
300 people dosed 800 times; drugs doing what they were designed to do; a new partnership (from a company you can be damn sure looked under the hood very closely) and until three weeks ago, not a cloud in the sky. The drugs are still the same; there will be no more bee venom in sub-q formulations and an IP portfolio that alone has to be worth more than the current MC. There will be another day....there could be an acquisition now w/ or w/o the 200 M share shelf- the stock is low enough; there could be a Chinese play now; there could be an acquisition (hey, as much as it would hurt, ALNY is still sitting on billions and if anyone could use ARWR's IP); there could be ultimate victory with patience.
My take is let the dust settle. My only lingering fear/suspicion tucked in the way back of my mind is something someone more devious than I pointed out once.....there's two sets of brothers running things. Down $360k today. F'in ouch.