On the surface, Alnylam Pharmaceuticals
But Alnylam's data for ALN-TTR02 come from a phase 1 trial in healthy volunteers. There's no direct proof the drug will successfully treat TTR-mediated amyloidosis.
So what's got investors so excited? ALN-TTR02 knocked down the level of TTR protein in healthy volunteers, which should translate well to treating patients who have mutations in their TTR genes. The mutant protein clumps, which causes damage to the peripheral nerves and heart and all kinds of bad things to happen. If there's less mutant protein, there should be fewer amyloid clumps.
Or so the theory goes. The only way to know for sure is to see the drug in action in patients with TTR amyloidosis.
More important in the overall scheme of things, and possibly a bigger influence on the stock jump yesterday, the clinical trial confirms the approach that Alnylam is taking. The biotech is developing RNAi technology that targets RNA, the intermediary step between DNA and proteins. ALN-TTR02 was able to knock down the production of the TTR protein in a few days and keep it down for a few weeks. If it works this well on TTR, it should work well on other proteins.
Even after the 50% jump, Alnylam still has a market cap under $1 billion, lower than it was back in 2008 when there was a lot less proof that RNAi could work as a therapeutic. The valuation back then was a bit crazy, but seems considerably more reasonable now.
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