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Alnylam Publishes Phase I Data on ATTR Drugs


Alnylam Pharmaceuticals and collaborators this week published data from phase I trials of the siRNA-based transthyretin amyloidosis drugs ALN-TTR01 and ALN-TTR02 in the New England Journal of Medicine.

The data, which had previously been released by the company (GSN 12/01/2011 & 7/19/2012), show that RNAi-based drugs can achieve rapid, dose-dependent, durable, and specific knockdown of the mutant protein that causes the disease, Alnylam said.

The paper can be found here.

"The phase I studies of ALN-TTR01 and ALN-TTR02 demonstrate key human proof of concept for RNAi therapeutics targeting TTR but also for liver-expressed target genes in general," Alnylam CMO Akshay Vaishnaw said in a statement.

"This new paper describes our clinical trial experience with ALN-TTR01 and ALN-TTR02, which utilize first- and second-generation lipid nanoparticle formulations, respectively," Alnylam Vice President of Clinical Research Jared Gollob added. "We believe the findings published today … suggest that the robust, sustained knockdown of TTR levels observed in our clinical trials with ALN-TTR02 may ameliorate the course of disease in patients with ATTR."

Alnylam has stopped development of ALN-TTR01 in order to focus on ALN-TTR02, which is poised to enter phase III testing before the end of this year. The company is also working on a subcutaneously delivered version of the drug, called ALN-TTRsc, that is slated to enter phase II by the end of 2013.