Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Fazisiran · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| Fazirsiran 25 mg DB/200 mg OL | EXPERIMENTAL | Double-blind (DB) Period: Participants with no fibrosis: Administered on Day 1 and Week 4. Participants with fibrosis: Administered on Day 1, Week 4, and Week 16, then every 12 weeks up to 18 total doses. Open-label (OL) Period: Participants with fibrosis at Screening who received double-blind (DB) fazirsiran 25 mg and who completed the post-dose liver biopsy at Week 48 (or Week 72 or Week 96) entered the open-label phase and received fazirsiran 200 mg every 12 weeks for the duration of the study. |
| Fazirsiran 100 mg DB/200 mg OL | EXPERIMENTAL | DB Period: Participants with no fibrosis: Fazirsiran 100 mg administered on Day 1 and Week 4. Participants with fibrosis: Fazirsiran 100 mg administered on Day 1, Week 4, and Week 16, then every 12 weeks up to 18 total doses. OL Period: Participants with fibrosis at Screening who received double-blind (DB) fazirsiran 100 mg and who completed the post-dose liver biopsy at Week 48 (or Week 72 or Week 96) entered the open-label phase and received fazirsiran 200 mg every 12 weeks for the duration of the study. |
| Fazirsiran 200 mg DB/200 mg OL | EXPERIMENTAL | DB Period: Participants with no fibrosis: Fazirsiran 200 mg administered on Day 1 and Week 4. Participants with fibrosis: Fazirsiran 200 mg administered on Day 1, Week 4, and Week 16, then every 12 weeks up to 18 total doses. OL Period: Participants with fibrosis at Screening who received double-blind (DB) fazirsiran 200 mg and who completed the post-dose liver biopsy at Week 48 (or Week 72 or Week 96) entered the open-label phase and received fazirsiran 200 mg every 12 weeks for the duration of the study |
| Placebo DB / Fazirsiran 200 mg OL | PLACEBO_COMPARATOR | DB Period: Participants with no fibrosis: Placebo administered on Day 1 and Week 4. Participants with fibrosis: Placebo administered on Day 1, Week 4, and Week 16, then every 12 weeks up to 18 total doses. OL Period: Participants with fibrosis at Screening who received double-blind (DB) placebo and who completed the post-dose liver biopsy at Week 48 (or Week 72 or Week 96) entered the open-label phase and received fazirsiran 200 mg every 12 weeks for the duration of the study. |
| Name | Type | Description |
|---|---|---|
| Fazisiran Injection | DRUG | solution for subcutaneous (sc) injection |
| Placebo | OTHER | sterile normal saline (0.9% NaCl), calculated to match active comparator, for sc injection |
Inclusion Criteria: * Diagnosis of AATD * Liver biopsy at Screening indicating liver fibrosis (score less than F4); a patient with no fibrosis may participate based on a previous biopsy conducted within one year * Women of childbearing potential must have a negative pregnancy test, cannot be breast...
Fazisiran is an investigational small molecule being developed for the treatment of Alpha 1-Antitrypsin Deficiency, a rare genetic condition. It is currently in Phase 2 clinical development and has not been approved by regulatory authorities.
Fazisiran is being developed by Arrowhead Pharmaceuticals, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol ARWR. The drug is also known as TAK-999 and ARO-AAT in clinical research.
Fazisiran is in Phase 2 clinical development. It is an investigational drug and has not been approved for commercial use. A Phase 2 clinical trial has been completed to evaluate its safety, tolerability, and pharmacodynamic effects in patients with Alpha 1-Antitrypsin Deficiency.
Fazisiran has one completed Phase 2 clinical trial registered under NCT03945292. The trial, titled 'Safety, Tolerability and Pharmacodynamic Effect of Fazirsiran (TAK-999, ARO-AAT)', enrolled 40 participants with Alpha 1-Antitrypsin Deficiency across the United States, Germany, Italy, Netherlands, Portugal, and Spain.
Yes, Fazisiran is also known as TAK-999 and ARO-AAT. These alternative names appear in clinical trial records and research publications. The drug is being developed by Arrowhead Pharmaceuticals under the primary name Fazisiran.
Yes, the completed Phase 2 trial of Fazisiran (NCT03945292) was a randomized, double-blind, placebo-controlled study. It enrolled 40 adult participants aged 18 years and older with Alpha 1-Antitrypsin Deficiency, and did not include healthy volunteers.