Drugs / ARC-AAT
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1 | NCT02363946 | Feb 2015 → Nov 2016 | alpha 1-antitrypsin deficiency | Arrowhead Pharmaceuticals | Terminated | No outcome recorded |
News releases announcing trial results or a regulatory action · 12
| Date | Issuer | Release |
|---|---|---|
| 2019-04-12 | Arrowhead Pharmaceuticals | Results Arrowhead Preclinical Data Demonstrates RNAi Therapy Prevents and Reverses Alpha-1 Liver Disease at The International Liver Congress™ arrowheadpharma.com ↗
The results presented today at EASL from a long-term term preclinical study of our prior generation compound, ARC-AAT, suggest that RNAi holds great promise for the treatment of patients with AATD-associated liver disease. |
| 2017-04-24 | Arrowhead Pharmaceuticals | Results Arrowhead Pharmaceuticals Presents ARC-AAT Clinical Data at The International Liver Congress™ arrowheadpharma.com ↗
The data demonstrated that an RNA interference (RNAi) therapeutic can achieve deep durable, and dose dependent reductions of alpha-1 antitrypsin (AAT), with the highest dose studied in healthy volunteers achieving near full suppression of the liver production of the AAT protein. |
| 2017-04-05 | Arrowhead Pharmaceuticals | Results Arrowhead Pharmaceuticals Announces Presentations at The International Liver Congress™ arrowheadpharma.com ↗
Hepatic targeted RNA interference provides deep and prolonged knockdown of alpha-1 antitrypsin levels in ZZ patients |
| 2016-11-14 | Arrowhead Pharmaceuticals | Results Arrowhead Pharmaceuticals Presents New Data on ARC-AAT arrowheadpharma.com ↗
The data indicate that in a first-in-human clinical study, ARC-AAT was well tolerated and induced deep and durable reduction of the target AAT protein. |
| 2016-10-20 | Arrowhead Pharmaceuticals | Results Arrowhead Pharmaceuticals to Present Data on ARC-AAT at The Liver Meeting® arrowheadpharma.com ↗
The clinical abstract shows that ARC-AAT achieved reductions in serum AAT of up to 90%. |
| 2016-01-19 | Arrowhead Pharmaceuticals | Regulatory Arrowhead's ARC-AAT Granted EMA Orphan Drug Designation arrowheadpharma.com ↗
Arrowhead Research Corporation (NASDAQ: ARWR), a biopharmaceutical company developing targeted RNAi therapeutics, today announced that the European Medicines Agency (EMA) granted Orphan Drug Designation to ARC-AAT for treatment of congenital alpha-1 antitrypsin deficiency. |
| 2015-07-08 | Arrowhead Pharmaceuticals | Regulatory Arrowhead Receives Regulatory Clearance for ARC-AAT Phase 1 in the United Kingdom and New Zealand arrowheadpharma.com ↗
Arrowhead Research Corporation (NASDAQ: ARWR), a biopharmaceutical company developing targeted RNAi therapeutics, today announced that it received regulatory permission in the United Kingdom and New Zealand to proceed with Part B of its Phase 1 study of ARC-AAT, Arrowhead's RNAi-based drug candidate for the treatment of liver disease associated with the rare genetic disorder alpha-1 antitrypsin deficiency (AATD). |
| 2015-06-10 | Arrowhead Pharmaceuticals | Regulatory Arrowhead Receives Orphan Drug Designation for ARC-AAT arrowheadpharma.com ↗
Arrowhead Research Corporation (NASDAQ: ARWR), a biopharmaceutical company developing targeted RNAi therapeutics, today announced the United States Food and Drug Administration (FDA) has granted ARC-AAT orphan drug designation. |
| 2015-05-05 | Arrowhead Pharmaceuticals | Results Arrowhead Completes Dosing Healthy Volunteers and Initiates Transition to Patients in Phase 1 Study of ARC-AAT arrowheadpharma.com ↗
All three dose levels tested appear to be generally well tolerated and the data safety committee has cleared the study to move into patients with AATD. |
| 2014-11-18 | Arrowhead Pharmaceuticals | Regulatory Arrowhead Files for Regulatory Permission to Begin Phase 1 Trial of RNAi Therapeutic ARC-AAT arrowheadpharma.com ↗
The application was submitted to an ethics committee in compliance with the Clinical Trial Notification process of the Australian Department of Health and Ageing, Therapeutic Goods Administration. |
| 2014-11-10 | Arrowhead Pharmaceuticals | Results Arrowhead Presents Data on ARC-520 and ARC-AAT at AASLD The Liver Meeting® 2014 arrowheadpharma.com ↗
Repeat dosing of ARC-AAT in primates shows reduction of approximately 90% of serum alpha 1 antitrypsin (AAT) with long duration of effect suggesting that monthly or less frequent dosing may be sufficient for sustained suppression of hepatic AAT production |
| 2014-06-19 | Arrowhead Pharmaceuticals | Results Arrowhead Announces New Clinical Candidate ARC-AAT for Treatment of Alpha-1 Antitrypsin Deficiency-Associated Liver Disease arrowheadpharma.com ↗
RNAi Therapeutic ARC-AAT Induces Dose Dependent Reductions in Mutant AAT Protein of Greater than 95 Percent in Preclinical Studies |
All press releases naming this drug 15 releases
Evidence & citations 4 cited values
Every value below carries the sentence it was read from. 2 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | ARC-AAT | ClinicalTrials.gov intervention name — accepted as the source's own label NCT02363946 ↗ |
| Action | Inhibit | “This study evaluated an RNAi therapeutic to silence production of AAT.” PMID 29572094 ↗ Mar 2018 |
| Modality | Small interfering RNA (siRNA) | “RNA interference-based, liver-targeted therapeutic” NCT02363946 ↗ |
| Route | Intravenous | “Healthy volunteers and AATD patients will be randomized to receive a single intravenous injection of either ARC-AAT or Placebo in double-blind fashion.” NCT02363946 ↗ |