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Alnylam Starts Phase II Study of Subcutaneous ATTR Drug

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Alnylam Pharmaceuticals this week announced that it has begun a phase II trial of its subcutaneously delivered TTR amyloidosis treatment ALN-TTRsc.

ATTR is caused by mutations in the TTR gene, which triggers accumulation of abnormal amyloid proteins in the body. ALN-TTRsc is designed to inhibit both the wild-type and mutant forms of the protein and is formulated with the GalNAc technology, which facilitates liver delivery of siRNAs via uptake through the asialoglycoprotein receptors expressed on the surface of hepatocytes.

The phase II trial is designed to test the tolerability and clinical activity of the therapy in patients with a form of the disease called familial amyloidotic cardiomyopathy. It expects data to become available in late 2014, with a phase III study launching by the end of that year.

Earlier this year, Alnylam reported phase I data that showed the drug capable of cutting levels of its target protein by about 90 percent.

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