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ARO-AAT · 2 trials · 1 indication
| Arm | Type | Description |
|---|---|---|
| ARO-AAT 100 mg Cohort 1b | EXPERIMENTAL | Primary Study Period (6-12 months): 100 mg dose of subcutaneous ARO-AAT for a minimum of 3 doses, with 2 optional treatment extension periods. Treatment Extension I (12 months): 100 mg dose of subcutaneous AROAAT every 12 weeks (Q12W). Treatment Extension II (up to 24 Months): 100 mg dose of subcutaneous ARO-AAT Q12W. |
| ARO-AAT 200 mg Cohort 1 | EXPERIMENTAL | Primary Study Period (6-12 months): 200 mg dose of subcutaneous ARO-AAT for a minimum of 3 doses, with 2 optional treatment extension periods. Treatment Extension I (12 months): 200 mg dose of subcutaneous AROAAT Q12W. Treatment Extension II (up to 24 Months): 200 mg dose of subcutaneous ARO-AAT Q12W. |
| ARO-AAT 200 mg Cohort 2 | EXPERIMENTAL | Primary Study Period (6-12 months): 200 mg dose of subcutaneous ARO-AAT for a minimum of 5 doses, with optional treatment extension periods. Treatment Extension I (12 months): 200 mg dose of subcutaneous AROAAT Q12W. Treatment Extension II (up to 24 Months): 200 mg dose of subcutaneous ARO-AAT Q12W. |
| ARO-AAT | ACTIVE_COMPARATOR | - |
| Placebo | PLACEBO_COMPARATOR | - |
| Name | Type | Description |
|---|---|---|
| ARO-AAT | DRUG | solution for subcutaneous injection |
| ARO-AAT Injection | DRUG | Single or multiple doses of ARO-AAT by subcutaneous (sc) injections |
| Sterile Normal Saline (0.9% NaCl) | OTHER | Calculated volume to match active comparator |
Inclusion Criteria: * Diagnosis of AATD * Liver biopsy indicating Metavir F1-F3 liver fibrosis based on local pathology read. * Women of childbearing potential must have a negative pregnancy test, cannot be breast feeding, and must be willing to use contraception * Willing to provide written inform...
ARO-AAT is an investigational drug being studied for the treatment of Alpha 1-Antitrypsin Deficiency, a rare genetic condition. It is being developed by Arrowhead Pharmaceuticals, Inc. (ARWR) and is currently in Phase 2 clinical development. ARO-AAT is also known as fazirsiran and TAK-999.
ARO-AAT is a small molecule designed to target the underlying cause of Alpha 1-Antitrypsin Deficiency. It works by reducing the production of the abnormal alpha-1 antitrypsin protein that accumulates in the liver and causes damage. This mechanism is intended to address the liver disease associated with the condition.
ARO-AAT is being developed by Arrowhead Pharmaceuticals, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker ARWR. The drug is also known as fazirsiran and TAK-999, and it is being studied for the treatment of Alpha 1-Antitrypsin Deficiency.
ARO-AAT is currently in Phase 2 clinical development. It has completed two clinical trials, including a Phase 1 study in healthy volunteers and a Phase 2 study in patients with Alpha-1 Antitrypsin Deficiency associated liver disease. The drug is investigational and has not been approved by regulatory authorities.
ARO-AAT has been studied in two completed clinical trials. The first, NCT03362242, was a Phase 1 study in 45 healthy adult volunteers in New Zealand. The second, NCT03946449, was a Phase 2 study in 16 patients with Alpha-1 Antitrypsin Deficiency associated liver disease, conducted in Austria, Germany, and the United Kingdom.
Yes, ARO-AAT is the same as fazirsiran and TAK-999. These names refer to the same investigational drug being developed by Arrowhead Pharmaceuticals for the treatment of Alpha 1-Antitrypsin Deficiency. The drug is currently in Phase 2 clinical development and has completed two trials.