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Alnylam Maps Out First Steps in ‘RNA Decade’

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specific antibodies was  first discovered in 1975, but it took 20 years for the first approved product to arrive. It was longer than that before antibodies blossomed into an estimated $30 billion worldwide market. Maraganore clarified that RNAi will demonstrate its effectiveness over the coming decade, but he wouldn’t go so far as to say the markets will also materialize in such a big way over the next 10 years.

Of course, 10 years is a long time, and there are a lot of milestones along the way that investors want to see. Last year at this time, Alnylam predicted it would deliver two more major partnerships during 2009, which didn’t happen. The company certainly made progress, by starting a clinical trial of its first RNAi treatment that can circulate throughout the bloodstream, forming a plan to move ahead into later-stage testing of its lead drug for respiratory syncytial virus in adults and children, and preparing to bring its third product into clinical trials for a rare genetic disorder that offers potential for a Genzyme-like domination of a pioneering treatment in a niche market.

But investors showed signs of impatience. Alnylam stock fell almost 29 percent in 2009.

So what are the incremental markers of success Alnylam has staked out for 2010? What steps does Alnylam hope will keep investors excited about the journey of developing RNA-based therapies? Here’s a rundown of some highlights.

—Before the end of March, Alnylam is planning to move its lead drug candidate, ALNRSV01, into a Phase IIb trial in adult patients who are infected with respiratory syncytial virus (RSV). Alnylam’s partner, Lexington, MA-based Cubist Pharmaceuticals will take the lead in advancing a next-generation form of this drug that the two companies consider a better candidate for children with RSV.

—Alnylam expects to have the first results from an early-stage clinical trial of ALN-VSP for treating liver cancer. This is the first-of-its-kind drug Alnylam has developed with help from Vancouver, BC-based Tekmira Pharmaceuticals that delivers the RNAi drug in lipid nanoparticles so that it can travel throughout the bloodstream.

—The next product candidate, for which Alnylam envisions building a Genzyme-like niche franchise around someday, will enter clinical trials before the end of June. This is ALN-TTR, a drug for TTR amyloidosis. I profiled this drug and the strategy last month.

—Fourth on the list is ALN-PCS, which is another RNAi treatment that can be delivered throughout the bloodstream to shut down the PCSK9 gene, which is involved in … Next Page »

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