Why Alnylam Pharmaceuticals Shares Soared

Although we don't believe in timing the market or panicking over market movements, we do like to keep an eye on big changes -- just in case they're material to our investing thesis.

What: Shares of biotechnology firm Alnylam Pharmaceuticals (Nasdaq: ALNY  ) rocketed higher by as much as 38% after the company reported positive clinical data.

So what: The catalyst behind the move was the announcement by Alnylam that ALN-TTR02, an RNAi therapeutic targeting the transthyretin (TTR) gene for the treatment of TTR-mediated amyloidosis, showed strong statistical significance in phase 1 studies. Overall, ALN-TTR02 led to a knockdown of serum TTR protein levels by 94%. Further data showed that just one dose showed efficacy in suppressing the TTR protein in for one month in nearly 80% of cases. The data provide ample human proof of concept with significant efficacy, and Alnylam plans to move forward with phase 2 clinical trials beginning in 2013.

Now what: Nowadays, it isn't just about the disease being treated, but the pathway by which that disease is treated. Alnylam is hitting an area of discovery still largely in its infancy, so the opportunity to carve out a huge niche in RNAi therapeutics is very possible. Then again, we must remember that today's results are simply representative of a single phase 1 clinical trial. Proof of concept in phase 1 doesn't always guarantee success down the road. I'm tempering my expectations after today but consider me mildly intrigued. I'd suggest adding Alnylam Pharmaceuticals to your Watchlist for future reference.

Craving more input? Start by adding Alnylam Pharmaceuticals to your free and personalized Watchlist so you can keep up on the latest news with the company.

Fool contributor Sean Williams has no material interest in any companies mentioned in this article. You can follow him on CAPS under the screen name TMFUltraLong, track every pick he makes under the screen name TrackUltraLong, and check him out on Twitter, where he goes by the handle @TMFUltraLong.

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  • Report this Comment On July 16, 2012, at 5:26 PM, GoNuke wrote:

    "Nowadays, it isn't just about the disease being treated, but the pathway by which that disease is treated. Alnylam is hitting an area of discovery still largely in its infancy..."

    It may be in its infancy but this "area of discovery" was not hard to predict. Alnylam's fortunes are tied to genomics and with the cost of sequencing a full human genome falling exponentially it was only a matter of time before genomics identified biological pathways (controlled by genes) that were responsible for diseases. Alnylam is positioned to be the "go to" company when seeking new treatments for cancer and mental illness, in my opinion. I have been holding Alnylam shares for some time now.

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