PASADENA, CA, USA I December 20, 2019 I Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced that it has dosed the first patient in AROAAT2002, an open-label Phase 2 clinical study of ARO-AAT, the company’s second generation investigational RNA interference (RNAi) therapeutic being developed as a treatment for the rare genetic liver disease associated with alpha-1 antitrypsin deficiency (AATD). Arrowhead is also conducting the SEQUOIA Phase 2/3 trial, which is an ongoing potentially pivotal study that began dosing patients in August 2019.

Javier San Martin, M.D., chief medical officer at Arrowhead, said: “We are committed to helping the alpha-1 community and would like to thank the participating investigators and patients, who currently have no available treatment options for alpha-1 liver disease other than liver transplant. AROAAT2002 is an important study within our ARO-AAT development program, which provides us with a key opportunity to assess patient response to treatment at various timepoints. This may prove to be helpful for future regulatory and commercial planning as the ARO-AAT development program progresses.”

AROAAT2002 (NCT03946449) is a pilot open-label, multi-dose, Phase 2 study to assess changes in a novel histological activity scale in response to ARO-AAT in patients with AATD associated liver disease. Approximately 12 participants will be enrolled in two sequential cohorts. All eligible participants will require a pre-dose biopsy and an end of study biopsy. Treated participants will also be offered the opportunity to continue treatment in an open-label extension (OLE). Including the OLE, changes in the novel histological grading scale will be assessed after 6 months, 12 months, 18 months, and 24 months of treatment with ARO-AAT.

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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SOURCE: Arrowhead Pharmaceuticals