Alnylam, Studying History of Genzyme, Eyes Drug to Make Big Impact for Rare Disease

12/14/09Follow @xconomy

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a lot of other complicating factors. The company’s small interfering RNA drugs can block all 100 known mutated forms of the TTR gene. Evidence is mounting that the Alnylam drug can reduce TTR protein levels in the blood by more than 80 percent in mice and non-human primates, and the effect lasts more than three weeks after a single shot. The protein is produced in the liver, and that’s one organ where Alnylam believes it can effectively deliver RNAi drugs with the help of lipid nanoparticles from its partner, Vancouver, BC-based Tekmira Pharmaceuticals.

There’s also a strong business case to make. Patients with TTR amyloidosis have a severe disease and need better treatments. The only real competition is liver transplant, although one other drug from Cambridge, MA-based FoldRx Pharmaceuticals is in clinical trials. The Alnylam treatment can be delivered in small doses, which means lower costs for raw materials for manufacturing, and therefore higher profit margins. And importantly, the clinical trial pathway is relatively quick, because researchers will be able to tell early on with objective criteria whether the drug is working.

Imporantly, the market is concentrated enough with a small number of specialty physicians that a small company can probably build a sales force large enough to commercialize such a product on its own, and keep all the profits to itself.

The plan is to begin clinical trials in the first half of 2010, Maraganore says. This puts the amyloidosis program at least a couple years behind the RSV program, which is still most likely to be Alnylam’s first marketed RNAi product, Maraganore says. But the amyloidosis treatment is “not that far behind,” Maraganore says.

Alnylam has stockpiled enough money in the bank—$453 million when it last reported financials at the end of September—that it can afford to roll the dice on clinical trials of this program on its own. That’s especially true if it continues to rack up milestone payments from partners who support its other programs. Having partners basically allows Alnylam gets to take a swing for the fence with TTR, but without betting the company’s life and death on a single drug.

“We’re excited about the potential of this. It can be a fundamental game-changer,” Maraganore says.

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  • Joan Fox

    Yes!!! We are excited about this new drug. My husband has this disease and we also had one relative die of this 20 years ago. My husband we believe may have started it with his carpal tunnel in his 40′s . Misdiagnosed almost two years ago he is now in his 4th or 5th years of symptoms. Officially diagnosed correctly l4 months ago . Have other family member with this disease. We will keep looking for more info on this. We have lost alot of time trying to find help for this disease. A daughter is negative and a son will be tested soon. Two cousins also have the mutation. I believe we heard about this process in Chicago at a Support Meeting.

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