Drugs / Fazirsiran
Fazirsiran
Trials 6
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 11 trial | ||||||
| Phase 1 | NCT05891158 | Oct 2023 → Sep 2025 | — | Takeda | Completed | No outcome recorded |
| Phase 22 trials · 1 met primary | ||||||
| Phase 2 | NCT03946449 | Oct 2019 → Apr 2022 | alpha 1-antitrypsin deficiency | Arrowhead Pharmaceuticals | Completed | No outcome recorded |
| Phase 2 | NCT03945292 | Aug 2019 → Nov 2021 | alpha 1-antitrypsin deficiency | Arrowhead Pharmaceuticals | Completed | Met primary |
| Phase 33 trials | ||||||
| Phase 3 | NCT06165341 | Mar 2024 → Aug 2028 expected | alpha 1-antitrypsin deficiency | Takeda | Recruiting | No outcome recorded |
| Phase 3 | NCT05899673 | Aug 2023 → May 2033 expected | alpha 1-antitrypsin deficiency | Takeda | Active not recruiting | No outcome recorded |
| Phase 3 | NCT05677971 | Mar 2023 → Jan 2029 expected | alpha 1-antitrypsin deficiency | Takeda | Recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 21
| Date | Issuer | Release |
|---|---|---|
| 2023-06-24 | Arrowhead Pharmaceuticals | Results Arrowhead Presents Updated Data from Phase 2 SEQUOIA Study of Investigational RNAi Therapy Fazirsiran in Patients with Alpha-1 Antitrypsin Deficiency Liver Disease arrowheadpharma.com ↗
Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today presented updated results from the Phase 2 SEQUOIA clinical study of investigational fazirsiran (TAK-999/ARO-AAT) for the treatment of liver disease associated with alpha-1 antitrypsin deficiency (AATD-LD). |
| 2023-01-09 | Takeda | Results Arrowhead and Takeda Announce Topline Results from SEQUOIA Phase 2 Study of Fazirsiran in Patients with Alpha-1 Antitrypsin Deficiency-Associated Liver Disease takeda.com ↗
Takeda ( TSE:4502/NYSE:TAK ) and Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced topline results from the Phase 2 SEQUOIA clinical study of investigational fazirsiran (TAK-999/ARO-AAT) for the treatment of liver disease associated with alpha-1 antitrypsin deficiency (AATD-LD). |
| 2023-01-09 | Arrowhead Pharmaceuticals | Results Arrowhead and Takeda Announce Topline Results from SEQUOIA Phase 2 Study of Fazirsiran in Patients with Alpha-1 Antitrypsin Deficiency-Associated Liver Disease arrowheadpharma.com ↗
today announced topline results from the Phase 2 SEQUOIA clinical study of investigational fazirsiran (TAK-999/ARO-AAT) for the treatment of liver disease associated with alpha-1 antitrypsin deficiency (AATD-LD). |
| 2022-06-27 | Takeda | Results Results from Phase 2 Study of Fazirsiran in Patients with Alpha-1 Antitrypsin Deficiency Published in New England Journal of Medicine takeda.com ↗
The NEJM article was published online ahead of print and is titled, “Fazirsiran for Liver Disease Associated with Alpha-1 Antitrypsin Deficiency.” |
| 2022-06-27 | Arrowhead Pharmaceuticals | Results Results from Phase 2 Study of Fazirsiran in Patients with Alpha-1 Antitrypsin Deficiency Published in New England Journal of Medicine arrowheadpharma.com ↗ |
| 2021-11-12 | Arrowhead Pharmaceuticals | Results Arrowhead Presents Additional Clinical Data on Investigational ARO-AAT Treatment at AASLD Liver Meeting arrowheadpharma.com ↗ |
| 2021-11-01 | Arrowhead Pharmaceuticals | Results Arrowhead Pharmaceuticals to Participate in Upcoming November 2021 Conferences arrowheadpharma.com ↗
Title: ARO-AAT Treatment Reduces Intra-hepatic Z-AAT Leading to Improved Parameters of Liver Health and Fibrosis Regression |
| 2021-06-26 | Arrowhead Pharmaceuticals | Results Arrowhead Presents Additional Clinical Data on Investigational ARO-AAT Treatment in Patients with Alpha-1 Liver Disease at EASL International Liver Congress arrowheadpharma.com ↗
The results demonstrate that, in the AROAAT2002 study, investigational ARO-AAT treatment led to improvements in multiple measures of liver health, including fibrosis, with substantial and sustained reductions in the level of mutant AAT protein (Z-AAT). |
| 2021-04-28 | Arrowhead Pharmaceuticals | Results Arrowhead Announces Improvement in Fibrosis after ARO-AAT Treatment in Patients with Alpha-1 Liver Disease arrowheadpharma.com ↗
Four of the five patients achieved a 1 or greater stage improvement in Metavir fibrosis stage, with no worsening of fibrosis in the fifth patient |
| 2020-11-13 | Arrowhead Pharmaceuticals | Results Arrowhead Interim Clinical Data Demonstrate ARO-AAT Treatment Improved Multiple Biomarkers of Alpha-1 Liver Disease arrowheadpharma.com ↗
Arrowhead 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). |
| 2020-09-16 | Arrowhead Pharmaceuticals | Results Arrowhead ARO-AAT Phase 2 Interim Results in Patients with Alpha-1 Liver Disease Demonstrate Improvements in Key Parameters after Six Months of Treatment arrowheadpharma.com ↗
After 24 weeks of treatment with investigational ARO-AAT in the AROAAT2002 study, serum and total intra-hepatic Z-AAT decreased in all four patients by up to 93% and 95%, respectively. |
| 2019-06-27 | Arrowhead Pharmaceuticals | Regulatory Arrowhead Pharmaceuticals Receives Fast Track Designation for ARO-AAT for Treatment of Alpha-1 Liver Disease arrowheadpharma.com ↗ |
| 2019-04-15 | Arrowhead Pharmaceuticals | Regulatory Arrowhead Pharmaceuticals Receives FDA Clearance to Begin Phase 2/3 Study of ARO-AAT for Treatment of Alpha-1 Liver Disease arrowheadpharma.com ↗ |
| 2019-03-18 | Arrowhead Pharmaceuticals | Regulatory Arrowhead Pharmaceuticals Files IND for Pivotal Phase 2/3 Study of ARO-AAT for Treatment of Alpha-1 Liver Disease arrowheadpharma.com ↗ |
| 2018-11-09 | Arrowhead Pharmaceuticals | Results Arrowhead Pharmaceuticals Presents Late-Breaking Clinical Data on ARO-AAT at Liver Meeting® 2018 arrowheadpharma.com ↗
Single-doses of 200 and 300 mg resulted in greater than 91% serum AAT reduction, with 3 of 4 subjects having concentrations below the level of quantitation (BLQ) |
| 2018-06-29 | Arrowhead Pharmaceuticals | Results Arrowhead Presents New Clinical Data on ARO-AAT at Alpha-1 National Education Conference arrowheadpharma.com ↗
In the AROAAT1001 Phase 1 clinical study, a single, open-label dose of 100 mg of ARO-AAT in four subjects achieved 93% maximum serum AAT knockdown and 87% mean maximum serum AAT knockdown. |
| 2018-06-27 | Arrowhead Pharmaceuticals | Regulatory Arrowhead Receives Positive EMA Opinion on Orphan Designation for ARO-AAT for Treatment of Congenital Alpha-1 Antitrypsin Deficiency arrowheadpharma.com ↗
Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) issued a positive opinion on Arrowhead’s application for orphan designation of its RNAi candidate, ARO-AAT, for the treatment of congenital alpha-1 antitrypsin deficiency. |
| 2018-02-22 | Arrowhead Pharmaceuticals | Regulatory Arrowhead Receives Regulatory Clearance to Begin Phase 1 Study of ARO-AAT for Treatment of Alpha-1 Liver Disease arrowheadpharma.com ↗ |
| 2018-02-15 | Arrowhead Pharmaceuticals | Regulatory Arrowhead Pharmaceuticals Receives Orphan Drug Designation for ARO-AAT arrowheadpharma.com ↗
Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced the United States Food and Drug Administration (FDA) has granted orphan drug designation to ARO-AAT, Arrowhead's second-generation investigational medicine for the treatment of a rare genetic liver disease associated with alpha-1 antitrypsin deficiency. |
| 2017-12-20 | Arrowhead Pharmaceuticals | Regulatory Arrowhead Pharmaceuticals Files for Regulatory Clearance to Begin Phase 1 Study of ARO-AAT arrowheadpharma.com ↗
Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced that it filed a regulatory submission to begin a first-in-human study of ARO-AAT, a second-generation investigational medicine for the treatment of alpha-1 liver disease that leverages Arrowhead's subcutaneously administered Targeted RNAi Molecule (TRiMTM) technology. |
| 2017-10-23 | Arrowhead Pharmaceuticals | Results Arrowhead Presents Promising Preclinical Data on Development of ARO-AAT for Treatment of Alpha-1 Liver Disease at Liver Meeting® 2017 arrowheadpharma.com ↗ |
All press releases naming this drug 32 releases
Evidence & citations 6 cited values
Every value below carries the sentence it was read from. 8 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Fazirsiran | “Safety, Tolerability and Pharmacodynamic Effect of Fazirsiran (TAK-999, ARO-AAT)” NCT03945292 ↗5“Fazirsiran for Liver Disease Associated with Alpha(1)-Antitrypsin Deficiency.” PMID 35748699 ↗ Jun 2022 |
| Known as | ADS-001 | ChEMBL registry synonym — accepted as the source's own label CHEMBL5095435 ↗ |
| Known as | ADS-001 FREE ACID | ChEMBL registry synonym — accepted as the source's own label CHEMBL5095435 ↗ |
| Known as | ARO-AAT | “Study of Fazirsiran (TAK-999, ARO-AAT) in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATD)” NCT03946449 ↗ |
| Action | Inhibit | “All the patients had reduced accumulation of Z-AAT in the liver” PMID 35748699 ↗ Jun 2022 |
| Route | Subcutaneous | “administered subcutaneously” NCT03945292 ↗ |