• Arrowhead Completes Dosing in Phase 1 Study of ARO-AAT for Treatment of Alpha-1 Liver Disease

    • September 4, 2018
    • Posted By : admin
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    Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced that it has completed dosing of a Phase 1 clinical 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 deficiency. Arrowhead intends to submit a late-breaking abstract with initial clinical data on ARO-AAT to the Liver Meeting ®, the Annual Meeting of the American Association for the Study of Liver Disease (AASLD), being held in November 2018.

    AROAAT1001 ( NCT03362242 ) is a Phase 1 single- and multiple-ascending dose study to evaluate the safety, tolerability, pharmacokinetics, and effect of ARO-AAT on serum alpha-1 antitrypsin levels in healthy adult volunteers. The study includes 7 cohorts in which subjects receive placebo, a single dose of ARO-AAT, or three monthly doses of ARO-AAT at doses of 35 (single dose only), 100, 200, or 300 mg. Additional cohorts were planned at a dose of 400 mg, but were deemed unnecessary based on observed activity at lower doses.

    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.


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