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Alnylam Begins Clinical Trials of ATTR, Hypercholesterolemia Drugs

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NEW YORK (GenomeWeb) – Alnylam Pharmaceuticals this week announced that it has initiated two new clinical trials, a Phase III study of its TTR-mediated amyloidosis (ATTR) drug revusiran and a Phase I study of its hypercholesterolemia therapy ALN-PCSsc.

Both drugs are administered subcutaneously using Alnylam's GalNAc conjugates.

ATTR is a condition characterized by the accumulation of amyloid deposits in tissues due to mutations in the TTR gene. Revusiran is designed to silence both mutant and wild-type TTR.

The Phase III trial will test revusiran in up to 200 ATTR patients with a manifestation of the disease called familial amyloidotic cardiomyopathy. Primary endpoints of the study are changes in a measurement of how far patients walk over a six-minute period and the reduction in TTR burden between treated and placebo-controlled patients after 18 months.

ALN-PCSsc is designed to silence proprotein convertase subtilisn/kexin type 9, or PCSK9, which has been shown to regulate low-density lipoprotein receptor levels and play a role in plasma low-density lipoprotein clearance.

The study will test single ascending and multiple doses of ALN-PCSsc in up to 76 healthy volunteers.

ALN-PCSsc is being developed in collaboration with The Medicines Company. Initiation of the trial triggered a $10 million milestone payment to Alnylam.

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