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 partimefriend on Jan 30, 2016 13:44:26 GMT -5
Oligonucleotide Therapeutics and Delivery 4 April, 2016 - 5 April, 2016 Hyatt Regency, Cambridge, MA, USA
ANTIVIRAL DEVELOPMENT
9:00 Using DPC Technology in RNAi Therapeutics for Chronic HBV Infection and Factor 12-Mediated Diseases David Lewis, Ph.D., CSO, Arrowhead Research Corporation
Post by partimefriend on Mar 28, 2016 18:42:13 GMT -5
Hello Qb, yeah don't know of anything new really. I have been following Arwr for only a few months now, and they seem to PR the conferences they present at. Just thought we could see somthing about the Oligonucleotide Therapeutics and Delivery conference.
I am trying to identify if Mauro Ferrari is using DPC technology in his cancer research.
Ferrari’s team developed porous silicon particles as drug carriers, able to travel unimpeded through healthy blood vessels thanks to their small size and disc-like shape. At the tumor site, the silicon breaks down, generating the cancer-killing nanoparticles. The nanoparticles, coiled into tiny balls that resemble cancer cells, are absorbed and taken to the cancer nucleus, where the drug is released.
The particles encase doxorubicin, a potent chemotherapy that, used conventionally, prevents tumor cells from dividing but causes adverse effects in the heart. No approach in humans has been able to deliver it only to the tumor, a limitation to the drug’s effectiveness and safety.
The strategy benefitted not just the 50 percent of mice who were disease-free eight months later, but also the half that didn’t respond as well. They lived an average of 160 days, the human equivalent of three to six years longer in humans than metastatic breast cancer patients on standard chemotherapy. The study involved triple-negative breast cancer, a hard-to-treat variety that doesn’t respond to strategies that effectively target the more common types.
“If this bears out in the clinic, even in a small fraction of what we’ve seen in lab experiments, it will be transformational, the first-ever demonstration of a cure of metastatic disease to the lungs,” said Mauro Ferrari, president and CEO of the Houston Methodist Research Institute and the study’s principal investigator.
The wording and timing of information coming from our company and progress being made simultaneously by Dr. Ferrari can not be coincidental.
Also, was CLRX101 in companies portfolio with work being done with Calonado? I do not remember the correct name of company which got divested from ARWR portfolio. Is there also a link with CERU work?
Anyone able to find information on what was presented today? I could not find anything posted or links from today's presentations.
Hello Wayne, I have not been able to find any information on this conference. I think it could have been changed, the pdf shows ContraVir Pharmaceuticals Inc. in the 9:00am time slot on Tues April 5. The web site shows Arrowhead in the same time slot.