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Alnylam licenses away hot PCSK9 prospect in 'small' $205M deal

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The Medicines Co. ($MDCO) has bought into one of the most exciting cardio drug targets in biotech, licensing a PCSK9 candidate from Alnylam Pharmaceuticals ($ALNY) for $25 million upfront and up to $180 million in milestones, the companies said today.

Taking out PCSK9 proteins robs the body of a key ingredient for cholesterol synthesis, reducing "bad" LDL cholesterol and lowering risk of heart disease. That is the promise that drives some of the biggest drugmakers on earth bonkers about this target.

While others such as Sanofi ($SNY) and Amgen ($AMGN) are pursuing monoclonal antibodies to inhibit PCSK9 proteins, Alnylam's ALN-PCS program is focused on injected RNA interference drugs that aim to silence a gene to shut down production of the protein at the source. And the drugs offer a way to combat high cholesterol in patients who don't respond to popular statins such as Pfizer's ($PFE) Lipitor and AstraZeneca's ($AZN) Crestor.

The Cambridge, MA-based biotech is handing over development of the program to The Medicines Co. after producing positive Phase I data for ALN-PCS02 in healthy volunteers with high cholesterol. The Medicines Co., which sells the anticlotting drug Angiomax, is expected to fund and handle future development and potential commercialization, with Alnylam eligible for double-digit royalties.

The Medicines Co. starts a ways behind Sanofi and Regeneron's ($REGN) candidate and Amgen's contender now in Phase III trials. As The New York Times' Andrew Pollack noted, this could explain the relatively "small" financials involved in the deal despite the heated interest in the target.

Yet Alnylam CEO John Maraganore, who had planned to license out the PCSK9 drug while his company focuses on its orphan disease programs, has notched another win for his strategy to advance more RNAi drugs from the company through either internal clinical development or partnerships.

The deal also brings up Maraganore's role as inventor of Angiomax during his days as a scientist at Biogen Idec ($BIIB), which licensed the drug to The Medicines Co.

- here's the release
- check out Pollack's article

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