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.
Post by littlestevie on Apr 14, 2016 7:09:58 GMT -5
I don't know if some of you went through the posters published yesterday by Arrowhead, but I have a question regarding the posters "Predicting HBsAg clearance responses during ARC-520 RNA interference (RNAi) therapy based on HBsAg epitope profile analysis".
I really don't get the figure "HBsAG CP (baseline vs on-therapy)" figure. Why in the "baseline" box there is already 18/40 patients with a clearance profile? Does it mean that about half of the patients have already a clearance profile before the ARC-520 treatment (is due to due to entecavir)? In this case why the number of patients with a clearance profile treated with placebo decrease in the "on therapy" box?
I would really appreciate if someone could help me understand this figure, I am clearly missing something to get it right.
Also in connection with this poster, while looking for information reagrding the "19plex HBsAg epitope mapping assay", I came across this presentation by Pr. Locarnini (who is the senior author on the poster): www.informedhorizons.com/hepdart2015/pdf/Presentations/Locarnini.pdf
It shows that the data from this poster where already presented in December 2015 at the Hep DART conference (see slide 23). I don't know why there were not released sooner. Interestingly there is also a graph regarding the Anti-HB (see slide 28). As stated in the posted significance is not reached.