Palm Beach, FL 7/16/12 (StreetBeat) – Alnylam Pharmaceuticals Inc (Nasdaq:ALNY) is trading up 34%, increasing $4.30 to $16.82, after announcing its achievement of positive clinical results from its Phase I Trial with ALN-TTRo2. The company is a leader in the RNAi therapeutics sector (a new class of gene-targeting drugs), is testing this drug on patients with a genetic disease known as TTR-mediated amyloidosis, or ATTR. This drug is an RNAi therapeutic that targets the transthyretin (TTR) gene for the treatment of ATTR.
Results from this study were highly significant, showing that ALN-TTRo2 leads to robust knockdown of serum TTR protein levels of up to 94%. TTR is the disease causing proten in ATTR, and its suppression was rapid, dose dependent, durable, and specific after just a single dose of this drug. The company recently reported its initiation of a Phase II study of ALN-TTRo2 in patients with ATTR and has guided that its goal is to start a pivotal trial in 2013.
“In our continued efforts to advance RNAi therapeutics to patients, we believe these data with ALN-TTR02 establish new industry milestones. First, these results demonstrate the highly robust level of target gene knockdown and attractive pharmacologic profile achievable with RNAi therapeutics. Further and for the first time, we have generated data using a control siRNA from a parallel study documenting the specificity for RNAi therapeutics in humans,” said John Maraganore, Ph.D., CEO of Alnylam. “Finally, these results mark important progress in our overall ‘Alnylam 5x15’ product strategy efforts. While there is more to do in our efforts to bring RNAi therapeutics to patients in need, we are very encouraged by this significant step forward.”
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