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ARO-AAT is designed to knock down the hepatic production of the mutant alpha-1 antitrypsin (Z-AAT) protein, the cause of progressive liver disease in AATD patients, is being developed to treat the liver disease associated with alpha-1 antitrypsin deficiency (AATD).
Lead Product(s): ARO-AAT
Therapeutic Area: Genetic Disease Product Name: ARO-AAT
Highest Development Status: Phase II/ Phase III Product Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable November 01, 2021
Details:
Results suggest that investigational ARO-AAT consistently reduced the production of the toxic mutant Z-AAT protein, which has been identified as the cause of progressive liver disease in patients with AATD.
Lead Product(s): ARO-AAT
Therapeutic Area: Genetic Disease Product Name: ARO-AAT
Highest Development Status: Phase II Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable July 29, 2021
Details:
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).
Lead Product(s): ARO-AAT
Therapeutic Area: Genetic Disease Product Name: ARO-AAT
Highest Development Status: Phase II Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable June 26, 2021
Details:
Under the terms of the agreement, Takeda and Arrowhead will co-develop ARO-AAT which, if approved, will be co-commercialized in the United States under a 50/50 profit-sharing structure.
Lead Product(s): ARO-AAT
Therapeutic Area: Genetic Disease Product Name: ARO-AAT
Highest Development Status: Phase II/ Phase III Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Takeda Pharmaceutical
Deal Size: $1,040.0 million Upfront Cash: $300.0 million
Deal Type: Licensing Agreement November 24, 2020
Details:
Three doses of ARO-AAT over 24-weeks resulted in consistent reductions of the disease-causing mutant Z protein (Z-AAT) and improvements in clinically relevant biomarkers of liver disease. Serum Z-AAT reductions of 86-93%.
Lead Product(s): ARO-AAT
Therapeutic Area: Genetic Disease Product Name: ARO-AAT
Highest Development Status: Phase II Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable November 13, 2020
Details:
Takeda and Arrowhead will co-develop ARO-AAT which, if approved, will be co-commercialized in the U.S under a 50/50 profit-sharing structure. Outside the U.S., Takeda will lead the global marketing strategy and receive an exclusive license to commercialize ARO-AAT.
Lead Product(s): ARO-AAT
Therapeutic Area: Genetic Disease Product Name: ARO-AAT
Highest Development Status: Phase II/ Phase III Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Takeda Pharmaceutical
Deal Size: $1,040.0 million Upfront Cash: $300.0 million
Deal Type: Collaboration October 08, 2020
Details:
The results show clear evidence of a meaningful pharmacodynamic effect by ARO-AAT, leading to improvements in relevant biomarkers, including substantial reductions in intra-hepatic mutant AAT protein (Z-AAT), both Z-AAT monomer and Z-AAT polymer.
Lead Product(s): ARO-AAT
Therapeutic Area: Genetic Disease Product Name: ARO-AAT
Highest Development Status: Phase II Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable September 16, 2020
