Pasadena’s Arrowhead Pharmaceuticals Opens New Phase Study of Liver Treatment Drug

Published : Friday, August 9, 2019 | 4:43 AM

Research at Arrowhead Pharmaceutical. Courtesy photo

Pasadena-based Arrowhead Pharmaceuticals Inc. said Aug. 8, that it has administered a dose of SEQUOIA (AROAAT2001) on its first patient.

SEQUOIA is a Phase 2/3 clinical study of ARO-AAT, the company’s second-generation subcutaneously administered RNAi (RNA interference) therapeutic. It is being developed as a treatment for a rare genetic liver disease associated with alpha-1 antitrypsin deficiency or AATD.

“ARO-AAT is the first RNAi therapeutic derived from our proprietary Targeted RNAi Molecule, or TRiM, platform to reach a potentially pivotal study,”  said Dr. Bruce Given, Arrowhead’s Chief Operating Officer and head of research. “It represents potential hope for patients living with alpha-1 liver disease, who currently have no available treatment options other than liver transplant.”

An Arrowhead statement said SEQUOIA is a placebo-controlled, adaptive design Phase 2/3 study to evaluate the safety, efficacy, and tolerability of ARO-AAT administered to patients with AATD associated liver disease.

The study will enroll 120 patients who will receive at least nine doses, or approximately two years of treatment, with ARO-AAT or placebo.

Doses will be administered on Day 1, 29, and approximately every 12 weeks thereafter, the statement continued. The four-arm placebo-controlled Part A component of the study 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, based on the effects of each dose.

The primary objective for Part B is to evaluate efficacy, as assessed by the proportion of ARO-AAT treated patients achieving a two-point improvement in a histologic grading scale of AATD-associated liver disease, and no worsening of liver fibrosis on end of study biopsy.

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 RNAi mechanism to induce rapid, deep, and durable knockdown of target genes. RNAi is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a specific protein.

For more information about Arrowhead and its work, visit

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