Press release
Arrowhead Interim Clinical Data Demonstrate ARO-AAT Treatment Improved Multiple Biomarkers of Alpha-1 Liver Disease
- Serum Z-AAT reductions of 86-93% - All patients demonstrated greater than 80% reduction in liver Z-AAT monomer - 3 of 4 patients had a decrease in liver
About this update from Arrowhead Pharmaceuticals, Inc.
[{"type":"text","content":"\n- Serum Z-AAT reductions of 86-93%\n\n- All patients demonstrated greater than 80% reduction in liver Z-AAT monomer\n\n- 3 of 4 patients had a decrease in liver globule involvement\n\n- 3 of 4 patients demonstrated reductions in Z-AAT polymer with a range of 68-97%\n\n- All patients showed ALT reductions ranging from 36-66%\n\n PASADENA, Calif.--(BUSINESS WIRE)--\nArrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced positive interim clinical data from 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). The data demonstrate that three doses of ARO-AAT over 24-weeks resulted in consistent reductions of the disease-causing mutant Z protein (Z-AAT) and improvements in clinically relevant biomarkers of liver disease. The results were presented in a late-breaking poster at The Liver Meeting Digital Experience, the Annual Meeting of the American Association for the Study of Liver Disease (AASLD).\n\nA copy of the poster may be accessed on the Events and Presentations page under the Investors section of the Arrowhead website.\n\nJavier San Martin, M.D., chief medical officer at Arrowhead, said: “These data presented at AASLD strongly suggest that ARO-AAT is doing what it’s designed to do, which is reduce the production of the misfolded mutant Z-AAT protein. Moreover, these compelling results indicate that the liver may have the ability to clear out accumulated Z-AAT and begin to heal itself faster than anticipated. Importantly, we saw reductions in serum Z-AAT and liver Z-AAT which led to improvements in multiple markers, such as liver globules, ALT/GGT and Pro-C3. These are all positive indications of a strong pharmacodynamic response and improvement in liver health, following just three doses of ARO-AAT. We anticipate data from additional patient cohorts will be available in the coming months, which will be included in our planned discussions with the U.S. Food and Drug Administration and other regulatory agencies, aimed at exploring areas where the ARO-AAT program could potentially be streamlined and accelerated.”\n\nIn the AROAAT2002 study, four patients with homozygous PiZZ alpha-1 antitrypsin deficiency and e...