US-based biopharmaceutical firm Arrowhead has commenced its Phase II clinical trial of ARC-AAT to treat liver disease associated with alpha-1 antitrypsin deficiency (AATD).

ARC-AAT is characterised by an unlocked nucleobase analog (UNA) containing RNAi trigger molecule for a systemic delivery using the Dynamic Polyconjugate delivery system.

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It has proven its efficacy in affecting the Alpha-1 antitrypsin (AAT) gene transcript while decreasing the hepatic production of the mutant AAT (Z-AAT) protein in animal models.

The inflammatory Z-AAT protein is considered to be responsible for the cause of progressive liver disease in AATD patients.

"There remains no medical treatment for the liver disease associated with AATD, which is increasingly being recognised by patients and physicians as a serious problem."

The Phase II ARC-AAT trial has been designed as a multi-centre, open-label, multiple dose-escalation study of ARC-AAT and will include 12 participants.

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It is intended to test the safety, efficacy and tolerability of multiple doses of ARC-AAT in treating AATD.

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ARC-AAT will be administered in two dose levels and evaluated in two separate cohorts.

The subjects will receive a pre-dose biopsy, administered with seven doses of ARC-AAT and a post-dose biopsy after the 183rd day.

Arrowhead research and development CEO and head Bruce Given said: "There remains no medical treatment for the liver disease associated with AATD, which is increasingly being recognised by patients and physicians as a serious problem.

“Our Phase II 2001 study should give us, and the AATD community in general, the first insights into whether ARC-AAT can stop the progression of liver disease and possibly even allow the liver to recover and heal existing damage.

“This would be a significant breakthrough for patients."

The company is currently conducting a Phase I clinical trial of ARC-AAT with Part A involving healthy volunteers and Part B involving AATD patients.