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Arrow Pharmaceutical Company (NASDAQ:hero) Today announced the additional positive mid-term 48-week liver biopsy results of the ongoing AROAAT2002 study, which is an open label for the second-generation investigative RNA interference (RNAi) therapeutic drug ARO-AAT jointly developed by the company and Takeda Pharmaceuticals 2 Phase clinical research at the International Liver Congress-European Association for the Study of the Liver (EASL) Annual Meeting, Limited (“Takeda”) as a treatment for rare inherited liver diseases related to α-1 Antitrypsin Deficiency (AATD).
The results showed that in the AROAAT2002 study, investigative ARO-AAT treatment resulted in the improvement of various liver health indicators, including fibrosis, while the level of mutant AAT protein (Z-AAT) was significantly and continuously decreased. In addition, after up to 1 year of treatment, ARO-AAT treatment is usually well tolerated.
Arrowhead Chief Medical Officer Javier Saint Martin, MD, said: “We believe that the interim results announced at EASL today represent an important breakthrough in this field and are encouraging for patients with alpha-1 liver disease who currently have no available treatment options other than liver transplantation. The data shows that the research is The treatment of ARO-AAT is TAK-999 developed in cooperation with Takeda, which leads to a large, continuous and consistent reduction in the production of toxic mutant Z-AAT protein, which has been identified as the cause of progressive liver disease. α-1 Antitrypsin deficiency This reduction within 6 months and 12 months has led to multiple important signals related to the cure of patients’ liver disease. Importantly, we believe that ARO-AAT is the first to show this in α-patients. Benefits of investigative therapy 1 Liver disease. We would like to thank all researchers and patients for participating in this study. We look forward to obtaining this study and other results of our ongoing Sequoia ARO-AAT study. We expect the study to be completed in 2021. Fully registered in the third quarter.”
Pharmacodynamics and efficacy
After 24 weeks (cohort 1, n=4) and 48 weeks (cohort 2, n=5) treatment with investigative ARO-AAT in the AROAAT2002 study, the following results were observed:
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The serum Z-AAT level of all patients decreased
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The median drop in Z-AAT levels in the liver is:
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Total Z-AAT -80.1% (range -72 to -97%)
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Monomer -90% (range -79 to -97%)
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Polymer -81% (range* -42 to -97%)
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*Excluding 1 subject in cohort 1, whose Z-AAT polymer level was very low at baseline but increased at week 24
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The histological globule load of all 9 patients was reduced, and 2 of them reached complete regression (total globule load total score = 0)
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6 out of 9 patients (2/4 after 24 weeks and 4/5 after 48 weeks) achieved 1 or more stages of improvement in Metavir fibrosis, of which…
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