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Arrowhead's second-generation subcutaneous RNAi therapy phase II study completes first patient dosing!

Release date: 2020-01-06 Views: 0

Source: Biovalley  

Arrowhead is a clinical stage biotechnology company dedicated to using RNA interference (RNAi) to suppress the post-transcriptional expression of disease-causing genes and to develop innovative drugs for the treatment of refractory diseases. Recently, the company announced that AROAAT2002, a phase II study evaluating the RNAi drug ARO-AAT, has completed the first patient drug treatment. Currently, the company is also conducting Phase II / III trials of SEQUOIA, a potentially key study underway, which began patient medication in August 2019.

ARO-AAT is the company's second-generation subcutaneous RNAi therapy and is currently being developed to treat liver diseases associated with alpha-1 antitrypsin deficiency (AATD), a rare genetic liver disease with severe damage Liver and lungs of affected individuals.

ARO-AAT aims to inhibit the production of mutated alpha-1 antitrypsin (Z-AAT) protein in the liver, which is the cause of progressive liver disease in patients with AATD. Reducing the production of inflammatory Z-AAT protein is expected to prevent the progression of liver disease and may allow it to regenerate and repair.

Dr. Javier San Martin, Chief Medical Officer of Arrowhead, said: "We are committed to helping the alpha-1 community and thank the researchers and patients who participated in the study. Currently, there is no other treatment for alpha-1 liver disease other than liver transplantation in this type of patients. Choice. AROAAT2002 is an important study in the ARO-AAT development project, which will provide us with a key opportunity to assess the patient's response to treatment at different points in time. As the ARO-AAT development project progresses, this may oversee future regulation And business planning. "

AROAAT2002 (NCT0394449) is an open-label, multi-dose study designed to assess changes in the new histological activity scale for ARO-AAT responses in patients with AATD-related liver disease. Approximately 12 subjects will be enrolled in two sequential cohort. All eligible subjects are required to undergo a pre-dose biopsy and study end biopsy. Subjects receiving treatment will also have the opportunity to continue with open extension (OLE) treatment. Including OLE, changes to the new histological rating scale will be assessed after 6, 12, 18, and 24 months of ARO-AAT treatment.

Arrowhead is committed to developing drugs to treat refractory diseases by suppressing the genes that cause them. Utilizing extensive RNA chemistry and effective delivery methods, the company aims to trigger RNA interference (RNAi) mechanisms and induce rapid, deep, long-lasting silencing of target genes. RNA interference (ie, RNAi) is a mechanism that exists in cells and inhibits the expression of specific genes, thereby affecting the production of specific proteins.

Arrowhead's RNAi therapy uses a natural approach to gene silencing. At present, the company has entered a number of experimental RNAi therapies into clinical development, and reached strategic cooperation with a number of pharmaceutical companies, including Johnson & Johnson's Janssen Biotechnology Company (development of hepatitis B RNAi therapy) and Amgen (development of cardiovascular disease RNAi therapy). ).

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