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Alnylam Files to Begin Phase I Trial of Subcutaneous TTR Drug in UK


Alnylam Pharmaceuticals announced this week that it has filed with UK regulators to begin phase I testing of a subcutaneous version of its TTR-mediated amyloidosis treatment called ALN-TTRsc.

Currently, Alnylam is testing an intravenously delivered form of the drug called ALN-TTR02, which it anticipates entering phase III testing by the end of this year. However, the firm has long been planning to incorporate its GalNAc conjugate delivery technology into its TTR amyloidosis program.

“Our pre-clinical studies with ALN-TTRsc have confirmed the ability of achieving over 80 percent TTR knockdown at single digit mg/kg doses with a very wide therapeutic index,” Alnylam CMO Akshay Vaishnaw said in a statement. “We very much look forward to the continued advancement of ALN-TTRsc, including the start of this phase I clinical trial in healthy volunteers early in the year with data expected mid-year."

In mid-2012, Alnylam reported positive phase I data on ALN-TTR02, showing the drug triggered up to 94 percent reductions in levels of its target protein after a single dose (GSN 7/19/2012).