Skip to main content
Premium Trial:

Request an Annual Quote

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

Premium

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.

The Scan

Booster Push

New data shows a decline in SARS-CoV-2 vaccine efficacy over time, which the New York Times says Pfizer is using to argue its case for a booster, even as the lower efficacy remains high.

With Help from Mr. Fluffington, PurrhD

Cats could make good study animals for genetic research, the University of Missouri's Leslie Lyons tells the Atlantic.

Man Charged With Threatening to Harm Fauci, Collins

The Hill reports that Thomas Patrick Connally, Jr., was charged with making threats against federal officials.

Nature Papers Present Approach to Find Natural Products, Method to ID Cancer Driver Mutations, More

In Nature this week: combination of cryogenic electron microscopy with genome mining helps uncover natural products, driver mutations in cancer, and more.