What: Shares of biotechnology firm Alnylam Pharmaceuticals (Nasdaq: ALNY ) rocketed higher by as much as 38% after reporting 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 provides 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.