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Alnylam Begins Phase I Testing of Hemophilia Drug


Alnylam Pharmaceuticals this week announced that it has begun a phase I trial of ALN-AT3, an RNAi-based treatment for hemophilia and other bleeding disorders.

The drug is designed to silence antithrombin and is administered using the company's GalNAc conjugates, which enable subcutaneous delivery.

The phase I trial is being conducted in the UK and is structured in two parts. The first will test single escalating doses of ALN-AT3 in 24 healthy volunteers, and will examine the agent's safety, as well as changes in antithrombin plasma levels at sub-pharmacologic doses.

The second part will evaluate multiple escalating doses of ALN-AT3 in 18 patients with moderate to severe hemophilia A or B. The primary objective of this arm of the trial is examine safety and tolerability, as well as clinical activity as determined by the knockdown of circulating antithrombin levels and increase in thrombin generation at pharmacologic doses.

Initial data from the study is expected to be released in late 2014.