The drug, known as ALN-PCS, inhibits a protein in the body known as PCSK9. Such drugs might one day be used to treat millions of people who do not achieve sufficient cholesterol-lowering from commonly used statins, such as Lipitor.
The Medicines Company will pay $25 million initially and as much as $180 million later if certain development and sales goals are met, under the deal expected to be formally announced Monday. It will also pay Alnylam, which is based in Cambridge, Mass., double-digit royalties on global sales.
That is small payment for a drug with presumably a huge potential market, probably reflecting that Alnylam is still in the first of three phases of clinical trials, well behind some far bigger competitors.
The team of Sanofi and Regeneron Pharmaceuticals is already entering the third and final stage of trials with their PCSK9 inhibitor, as is Amgen. Pfizer and Roche are in midstage trials.
ALN-PCS is different from the other drugs. It uses a gene-silencing mechanism called RNA interference, aimed at shutting off production of the PCSK9 protein. The other drugs are proteins called monoclonal antibodies that inhibit the action of PCSK9 after it has been formed.
Alnylam and the Medicines Company hope that turning off the faucet, as it were, will be more efficient than mopping the floor, allowing their drug to be given less frequently and in smaller amounts.
But that has yet to be proved. No drug using RNA interference has reached the market.
The Medicines Company, based in Parsippany, N.J., generates almost all of its revenue from one product — Angiomax, an anticlotting drug used when patients receive stents to open clogged arteries.
Dr. Clive A. Meanwell, chief executive of the company, said that PCSK9 inhibitors are likely to be used at first mainly by patients with severe lipid problems under the care of interventional cardiologists, the same doctors who use Angiomax. “It really is quite adjacent to what we do,” he said.
The Medicines Company licensed Angiomax from Biogen Idec, where the drug was invented and initially developed under a team led by Dr. John M. Maraganore, who is now the chief executive of Alnylam.
“It’s a bit like getting the band back together,” Dr. Maraganore said.
Medicines Co. Licenses Rights to Cholesterol Drug
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Medicines Co. Licenses Rights to Cholesterol Drug
