Arrowhead second generation subcutaneous RNAi therapy phase II study completed the first patient administration!

Arrowhead is a clinical biotechnology company, which is committed to using RNA interference (RNAi) to inhibit the posttranscriptional expression of pathogenic genes and develop innovative drugs for the treatment of refractory diseases Recently, the company announced that aroaat2002, a phase II study to evaluate the RNAi drug aro-aat, has completed the first patient medication At present, the company is also carrying out the seqoia II / III trial, which is an ongoing potential key study, and started to use drugs for patients in August 2019 Aro-aat is the company's second generation of subcutaneous RNAi therapy, which is currently being developed for the treatment of liver disease associated with alpha-1 antitrypsin deficiency (AATD), a rare genetic liver disease that seriously damages the liver and lungs of affected individuals Aro-aat aims to inhibit the production of mutant α - 1 antitrypsin (z-aat) protein in the liver, which is the cause of progressive liver disease in AATD patients Reducing the production of inflammatory z-aat protein is expected to prevent the progression of liver disease, and may make it 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 At present, there is no alternative to liver transplantation for the treatment of α - 1 liver disease Aroaat2002 is an important study in the aro-aat development project, which will provide us with a key opportunity to evaluate patients' response to treatment at different time points As the aro-aat development progresses, this may help with regulatory and business planning in the future " Aroaat2002 (nct0394449) is an open label, multi dose study designed to assess the changes of aro-aat response in patients with AATD related liver disease About 12 subjects will be put into two sequential cohorts All eligible subjects were required to undergo pre dose and end of study biopsies Subjects receiving treatment will also have the opportunity to continue to receive open extension (OLE) treatment Including Ole, changes in the new histological rating scale will be evaluated after 6, 12, 18 and 24 months of aro-aat treatment.