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Alnylam reports ALN-TTRsc achieves greater than 80% knockdown of serum TTR

Alnylam Pharmaceuticals announced positive top-line results from its ongoing Phase I trial of ALN-TTRsc, a subcutaneously administered RNAi therapeutic targeting the transthyretin gene for the treatment of TTR-mediated amyloidosis. The company is reporting that ALN-TTRsc achieved robust and statistically significant knockdown of serum TTR protein levels of greater than 80% in healthy volunteer subjects, in line with results for ALN-TTRsc previously reported in non-human primates. In addition, to date ALN-TTRsc was found to be generally safe and well tolerated These human study results are the first to be reported for Alnylam’s proprietary GalNAc-siRNA conjugate delivery platform, enabling subcutaneous dosing of RNAi therapeutics with a wide therapeutic index. Upon completion of the Phase I trial, the company plans to start a Phase II clinical study of ALN-TTRsc in FAC patients later this year and, assuming positive results, expects to start a pivotal Phase III trial for ALN-TTRsc in FAC patients in 2014.