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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 Alnylam Pharmaceuticals (NASDAQ: ALNY ) , a biopharmaceutical company developing treatments based on RNA interference, vaulted 19% higher after the company announced positive top-line data for ALN-TTRsc.
So what: In early-stage trials, initial data showed that a subcutaneous treatment targeting TTR-mediated amyloidosis achieved statistical significant results by knocking down serum TTR levels in patients by greater than 80%. The true excitement comes from the quickness of subcutaneous injection as compared to intravenous injection, and the fact that most patients are more willing to get medication subcutaneously than through an IV. Alnylam anticipates beginning a phase 2 trial for ALN-TTRsc later this year and, if all goes well, initiate a phase 3 trial sometime next year.
Now what: Both of Alnylam's TTR-mediated amyloidosis drugs have shown considerable promise in trials -- ALN-TTR02, an intravenous application to treat familial amyloidotic polyneuropathy (FAP), and ALN-TTRsc for familial amyloidotic cardiomyopathy (FAC). There are four times as many cases of FAC worldwide, so it's not hard to understand why investors are excited about this early stage news. As for me, I still remain skeptical of Alnylam's valuation at these levels. My suggestion would be to watch Alnylam closely, but do it from a safe distance!
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