Alnylam reported positive results from a Phase I trial with ALN-TTRsc in 28 healthy volunteers at the Heart Failure Society of America 17th Annual Scientific Meeting held in September 2013. The results of the clinical study showed that ALN-TTRsc administration led to robust, consistent, and statistically significant (p<0.01) knockdown of serum TTR of up to 94%. Knockdown of TTR was found to be rapid, dose-dependent, and durable. ALN-TTRsc was found to be generally safe and well tolerated in the study. Importantly, these human study results were 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.In 2012, Alnylam entered into an exclusive alliance with Genzyme, a Sanofi company, to develop and commercialize RNAi therapeutics, including patisiran and ALN-TTRsc, for the treatment of ATTR in Japan and the broader Asian-Pacific region. Alnylam plans to develop and commercialize the ALN-TTR program in North and South America, Europe, and rest of the world.
Alnylam Initiates Phase II Clinical Trial With ALN-TTRsc, A Subcutaneously Delivered RNAi Therapeutic Targeting Transthyretin (TTR) In Development For The Treatment Of TTR-Mediated Amyloidosis (ATTR)
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