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Arrowhead Files to Start Phase I Trial of Alpha-1 Antitrypsin Deficiency Drug

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NEW YORK (GenomeWeb) – Arrowhead Research announced today that it has filed an application with regulators to begin a Phase I trial of its alpha-1 antitrypsin deficiency (AAT) drug ARC-AAT in Australia.

AAT deficiency is a genetic disease that leads to decreased activity of the protease inhibitor alpha-1 antitrypsin in the blood and lungs causing lung dysfunction. It can also feature the accumulation of a mutant AAT protein known as Z-AAT in liver tissue, which leads to liver injury, fibrosis, cirrhosis, and potentially liver cancer.

Upon approval of its application, Arrowhead said that it will start the trial of the drug in up to 48 healthy volunteers and AAT patients to evaluate ARC-AAT's safety, tolerability, pharmacokinetics, and effect on circulating alpha-1 antitrypsin levels.

The study will test escalating doses of ARC-AAT until predetermined levels of alpha-1 antitrypsin reduction are reached, Arrowhead said. The study will transition into a population of patients with ZZ genotype alpha-1 antitrypsin deficiency to further evaluate escalating doses of the drug.

Arrowhead added ARC-AAT, which combines its unlocked nucleobase analog-modified siRNAs with its dynamic polyconjugate delivery vehicles, to its pipeline this summer.

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