Nanotechnology Now

Our NanoNews Digest Sponsors
Heifer International

Wikipedia Affiliate Button

Home > Press > Arrowhead Pharmaceuticals Begins Dosing in Phase 1 Study of ARO-ANG3 for Treatment of Dyslipidemias and Metabolic Diseases

Abstract:
Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced that it has dosed the first subjects in a Phase 1 clinical study of ARO-ANG3, an RNAi-based investigational medicine targeting angiopoietin like protein 3 (ANGPTL3) being developed for the treatment of dyslipidemias and metabolic diseases.

Arrowhead Pharmaceuticals Begins Dosing in Phase 1 Study of ARO-ANG3 for Treatment of Dyslipidemias and Metabolic Diseases

Pasadena. CA | Posted on January 7th, 2019

Bruce Given, M.D., chief operating officer and head of R&D at Arrowhead, said: “We continue to advance our broad pipeline of RNAi therapeutics with the dosing of the first subjects in our Phase 1 study of ARO-ANG3. ANGPTL3 has emerged as a potentially important target to address dyslipidemias, such as elevated cholesterol and triglycerides that are not well-controlled with currently available medicines, and also metabolic diseases, such as NAFLD and NASH. The preclinical data for ARO-ANG3 have been promising, and our prior clinical results from the ARO-HBV and ARO-AAT programs, which also leverage the Targeted RNAi Molecule, or TRiMTM, platform, give us confidence in the potential for ARO-ANG3 to address significant unmet medical needs.”

AROANG1001 (NCT03747224) is a Phase 1 single and multiple dose study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of ARO-ANG3 in adult healthy volunteers and patients with dyslipidemia. The study is designed to enroll up to 70 subjects.

The single-ascending dose (SAD) portion of the study is designed to include up to 4 cohorts of 10 adult healthy volunteers per cohort (6 active: 4 placebo). Each SAD subject will receive a single-dose administration of either placebo or ARO-ANG3 at dose levels of 35, 100, 200, or 300 mg. The multiple-dose portion is designed to include up to 4 patient cohorts, including patients with non-alcoholic fatty liver disease (NAFLD), patients on a stable statin treatment regimen with elevated low-density lipoprotein cholesterol (LDL-C) and triglycerides, patients with heterozygous or homozygous familial hypercholesterolemia, and patients with severe hypertriglyceridemia.

Dyslipidemia is a major risk factor for atherosclerotic cardiovascular disease (Kersten, 2017). While the current standard of care is effective at lowering LDL-C, a large unmet medical need for lipid lowering and risk modifying therapies with novel mechanisms of action persists. Hypertriglyceridemia and elevations in triglyceride-rich lipoproteins represent causative risks for atherosclerosis, and elevated triglycerides also manifest in the form of metabolic syndrome, pancreatitis and hepatic steatosis. Metabolic syndrome is a complex of interrelated risk factors for cardiovascular disease and type II diabetes mellitus.

ANGPTL3 has emerged as an important regulator of plasma lipoprotein levels (including triglycerides, LDL-C, high-density lipoprotein cholesterol, and very low-density lipoprotein cholesterol) by inhibition of enzymes including lipoprotein lipase and endothelial lipase. ANGPTL3 may also be involved in regulating apolipoprotein B particle containing synthesis and hepatocyte clearance of LDL-C through mechanisms independent of the low-density lipoprotein receptor (LDLR) (Xu et al., 2018). This LDLR-independent feature makes ANGPTL3 inhibition potentially applicable as a therapeutic for LDLR-deficient hypercholesterolemic patients.

Intrahepatic targeting of ANGPTL3 may also improve hepatic steatosis which can progress to nonalcoholic steatohepatitis (NASH). Additionally, human genetic studies indicate that ANGPTL3-deficient homozygotes show lower serum insulin, lower serum glucose, and improved measures of insulin resistance compared to non-carriers (Robciuc et al., 2013).

####

About Arrowhead Pharmaceuticals, Inc.
Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them. Using a broad 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. RNA interference, or RNAi, is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a specific protein. Arrowhead’s RNAi-based therapeutics leverage this natural pathway of gene silencing.

For more information, please visit www.arrowheadpharma.com, or follow us on Twitter @ArrowheadPharma. To be added to the Company's email list and receive news directly, please visit http://ir.arrowheadpharma.com/email-alerts .

Safe Harbor Statement under the Private Securities Litigation Reform Act:

This news release contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. These statements are based upon our current expectations and speak only as of the date hereof. Our actual results may differ materially and adversely from those expressed in any forward-looking statements as a result of various factors and uncertainties, including the safety and efficacy of our product candidates, the duration and impact of regulatory delays in our clinical programs, our ability to finance our operations, the likelihood and timing of the receipt of future milestone and licensing fees, the future success of our scientific studies, our ability to successfully develop and commercialize drug candidates, the timing for starting and completing clinical trials, rapid technological change in our markets, and the enforcement of our intellectual property rights. Our most recent Annual Report on Form 10-K and subsequent Quarterly Reports on Form 10-Q discuss some of the important risk factors that may affect our business, results of operations and financial condition. We assume no obligation to update or revise forward-looking statements to reflect new events or circumstances.

For more information, please click here

Contacts:
Arrowhead Pharmaceuticals, Inc.
Vince Anzalone, CFA
626-304-3400


Investors and Media:
LifeSci Advisors, LLC
Brian Ritchie
212-915-2578

www.lifesciadvisors.com

Copyright © Arrowhead Pharmaceuticals, Inc.

If you have a comment, please Contact us.

Issuers of news releases, not 7th Wave, Inc. or Nanotechnology Now, are solely responsible for the accuracy of the content.

Bookmark:
Delicious Digg Newsvine Google Yahoo Reddit Magnoliacom Furl Facebook

Related News Press

News and information

New method to reduce uranium concentration in contaminated water March 18th, 2019

Review of the recent advances of 2D nanomaterials in Lit-ion batteries March 15th, 2019

Converting biomass by applying mechanical force Nanoscientists discover new mechanism to cleave cellulose effectively and in an environmentally friendly way March 15th, 2019

Exotic “second sound” phenomenon observed in pencil lead: At relatively balmy temperatures, heat behaves like sound when moving through graphite, study reports March 15th, 2019

Nanomedicine

Arrowhead Pharmaceuticals Begins Dosing in Phase 1 Study of ARO-APOC3 for Treatment of Hypertriglyceridemia March 11th, 2019

New optical imaging system could be deployed to find tiny tumors: Near-infrared technology pinpoints fluorescent probes deep within living tissue; may be used to detect cancer earlier March 8th, 2019

Computer-designed vaccine elicits potent antibodies against RSV: The nanoparticle platform for this respiratory syncytial virus study will be applied to vaccine research on flu, HIV, and more; Seattle startup Icosavax will advance related clinical trials March 8th, 2019

CEA-Leti Breakthrough Opens Path to New Vaccine for HIV: Lipidots Platform Strengthens Immune Response to Protein That Is Key to HIV Vaccine; Results Presented in Nature Publishing Group’s npj Vaccines February 27th, 2019

Announcements

New method to reduce uranium concentration in contaminated water March 18th, 2019

Review of the recent advances of 2D nanomaterials in Lit-ion batteries March 15th, 2019

Converting biomass by applying mechanical force Nanoscientists discover new mechanism to cleave cellulose effectively and in an environmentally friendly way March 15th, 2019

Exotic “second sound” phenomenon observed in pencil lead: At relatively balmy temperatures, heat behaves like sound when moving through graphite, study reports March 15th, 2019

Nanobiotechnology

Arrowhead Pharmaceuticals Begins Dosing in Phase 1 Study of ARO-APOC3 for Treatment of Hypertriglyceridemia March 11th, 2019

Computer-designed vaccine elicits potent antibodies against RSV: The nanoparticle platform for this respiratory syncytial virus study will be applied to vaccine research on flu, HIV, and more; Seattle startup Icosavax will advance related clinical trials March 8th, 2019

Nanotechnology Gives Mice Night Vision—Are Humans Next? March 2nd, 2019

CEA-Leti Breakthrough Opens Path to New Vaccine for HIV: Lipidots Platform Strengthens Immune Response to Protein That Is Key to HIV Vaccine; Results Presented in Nature Publishing Group’s npj Vaccines February 27th, 2019

NanoNews-Digest
The latest news from around the world, FREE



  Premium Products
NanoNews-Custom
Only the news you want to read!
 Learn More
NanoStrategies
Full-service, expert consulting
 Learn More











ASP
Nanotechnology Now Featured Books




NNN

The Hunger Project