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Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced clinical data from a Phase 1 study of ARO-AAT, the company’s second generation subcutaneously administered RNA interference (RNAi) therapeutic being developed as a treatment for a rare genetic liver disease associated with alpha-1 antitrypsin (AAT) deficiency, will be presented in a late-breaking poster at The Liver Meeting®, the Annual Meeting of the American Association for the Study of Liver Disease (AASLD), being held in San Francisco.
In the AROAAT1001 study, 45 normal healthy volunteers (NHV) received a single dose of ARO-AAT (n=16), three monthly doses of ARO-AAT (n=12), or placebo (n=17). Key data presented include the following:
“The ARO-AAT data being presented at The Liver Meeting demonstrate the power of our Targeted RNAi Molecule (TRiMTM) platform, which is the basis of our growing pipeline of RNAi therapeutics targeting a diverse range of diseases,” said Bruce Given, M.D., Arrowhead’s chief operating officer and head of R&D. “These data indicate that ARO-AAT can produce robust and consistent reductions in the liver production of the AAT protein. In addition, the duration of response indicates that a dosing interval of once a quarter or even less frequent is feasible. Our next step is to initiate a Phase 2 study to evaluate whether this reduction can, over time, have an impact on the progressive liver disease in alpha-1 patients, whose only treatment option currently is liver transplant.”
A Phase 1 Single and Multiple Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Effect of ARO-AAT on Serum Alpha-1 Antitrypsin levels in Normal Adult Volunteers
A copy of the poster may be accessed on the Events and Presentations page under the Investors section of the Arrowhead website.
ARO-AAT is designed to silence the production of the misfolded Z-AAT protein with the intent to:
AROAAT1001 (NCT03362242) is a Phase 1 randomized, double-blind, placebo controlled single-ascending dose (SAD) and multiple-ascending dose (MAD) study to evaluate the safety, tolerability, pharmacokinetics, and effect of subcutaneous doses of ARO-AAT on serum alpha-1 antitrypsin levels in healthy adult volunteers. The SAD portion of the study included four cohorts at dose levels of 35, 100, 200, and 300 mg and the MAD portion of the study included three cohorts at dose levels of 100, 200, and 300 mg. Additional cohorts were planned at a dose of 400 mg, but were deemed unnecessary based on observed activity at lower doses. AROAAT1001 enrolled 45 healthy volunteers.
About Arrowhead Pharmaceuticals
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.
Source: Arrowhead Pharmaceuticals, Inc.
Arrowhead Pharmaceuticals, Inc.
Vince Anzalone, CFA
Investors and Media:
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