Photomicrograph of a liver biopsy from a patient with alpha-1 antitrypsin deficiency. Credit: Jerad M Gardner, MD.

Arrowhead Pharmaceuticals has initiated a potentially pivotal Phase II/III clinical trial of ARO-AAT, its second generation subcutaneously administered RNA interference (RNAi) therapeutic.

As part of the initiation, the company dosed the first patient in the SEQUOIA (AROAAT2001) trial.

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Arrowhead is developing the RNAi therapeutic as a treatment for a rare genetic liver disease associated with alpha-1 antitrypsin deficiency (AATD).

Arrowhead Pharmaceuticals chief operating officer and R&D head Bruce Given said: “ARO-AAT is the first RNAi therapeutic derived from our proprietary Targeted RNAi Molecule, or TRiM, platform to reach a potentially pivotal study.

“This is a significant milestone for Arrowhead and, more importantly, it represents potential hope for patients living with alpha-1 liver disease, who currently have no available treatment options other than liver transplant.”

The placebo-controlled, adaptive design Phase II/III SEQUOIA (NCT03945292) trial will evaluate the safety, efficacy, and tolerability of the drug candidate ARO-AAT administered subcutaneously to patients with AATD associated liver disease.

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A total of 120 patients will be given at least nine doses, or approximately two years worth of treatment, with ARO-AAT or placebo.

Doses will then be administered on day one, 29, and roughly every 12 weeks.

Part A of the study will be four-arm and placebo-controlled and will feed seamlessly into a two-arm placebo-controlled Part B component.

The primary objective for Part A is to select a single dose level for use in Part B of the study, while the primary objective for Part B is to evaluate the efficacy of ARO-AAT.

In September last year, Arrowhead completed dosing patients in a Phase I clinical trial (AROAAT1001) of ARO-AAT for the treatment of a liver disease caused by AATD.