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Alnylam Launches Phase II Trial of Second-Generation Amyloidosis Drug

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Alnylam Pharmaceuticals announced this week that it has begun a phase II trial of its TTR-mediated amyloidosis drug ALN-TTR02 to test the compound's clinical activity, safety, and tolerability.

In the study, approximately 20 ATTR patients will receive increasing doses of the drug once every four weeks for two cycles, the company said. “The primary objectives of the study are to evaluate the safety and tolerability of multiple doses of ALN-TTR02 and to measure clinical activity based on serial measurement of circulating serum TTR levels.”

Alnylam recently unveiled phase I data on a previous version of the drug called ALN-TTR01, which was able to trigger dose-dependent and durable reductions in target protein levels in patients (GSN 5/10/2012).

The company has publicly stated that it only intends on advancing ALN-TTR02, which uses an improved lipid-delivery systems, toward commercialization.

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