Fattening up the IP

Alnylam licenses lipid technology from Inex for use in RNAi therapeutics work

By Jeffrey Bouley

CAMBRIDGE, Mass.—In a bid to further increase its market position in RNAi therapeutics discovery, development and commercialization, Alnylam Pharmaceuticals this winter negotiated a worldwide exclusive license to liposomal delivery formulation technology from British Columbia, Canada-based Inex Pharmaceuticals. In addition, the two companies have expanded their previous technology research and manufacturing alliance on lipid-based delivery technology.

Also in the deal, Inex will receive three InterfeRx license options, subject to Alnylam review and third-party obligations, to develop its own RNAi therapeutic products and Inex will also receive exclusive access to Alnylam's intellectual property to develop oligonucleotide drugs that act through an immune stimulation mechanism, outside the RNAi pathway.

What this means in hard U.S. dollars and cents is that Inex—a developer of proprietary drugs and drug delivery systems to improve the treatment of cancer—will receive an upfront payment of $8 million in newly issued shares of Alnylam common stock and/or cash, at Alnylam's option. In addition, Inex will receive $4 million in research and development funding over the next two years to continue to identify and develop lipid-based delivery systems for Alnylam. Inex will provide contract manufacturing services on an exclusive basis for Alnylam proprietary products in the collaboration and Alnylam will make available a $5 million loan for capital equipment expenditures related to manufacturing capabilities.

Moreover, Inex is eligible to receive $13 million in potential milestone payments for each product using Inex technology, of which $9.5 million is due upon regulatory approval and successful commercialization (defined as more than $500 million in cumulative product sales), plus royalties.

What this means in broader business terms, for Alnylam, is that the company will significantly "advance and solidify its market leadership in the area of RNAi," says Barry Greene, chief operating officer of Alnylam. "We've had collaborations with Inex where we utilized their unique technology and capabilities, which is how we came to the idea this would be a good strategic alliance. This gives us a leg up in terms of RNAi leadership, because lipid-based technologies are key to delivering RNAi therapeutics systemically, and it will accelerate our product pipeline. At the same time, Inex gets a boost in developing products for their own strategic goals outside of the RNAi realm."

Inex's intellectual property estate comprises certain key patents, such as those derived from the Wheeler and Semple patent series (U.S. Patent Nos. 5,976,567, 6,815,432, and 6,858,225), which are considered important for the development and commercialization of liposomal and lipid nanoparticle formulations of oligonucleotide therapeutics, including small interfering RNAs (siRNAs), the molecules that mediate RNAi.

Alnylam had published results in March 2006 demonstrating, for the first time, therapeutic gene silencing in primates with systemically delivered RNAi therapeutics, and those results were obtained using liposomal formulations. Alnylam reportedly intends to advance its RNAi therapeutic that targets PCSK9 for the treatment of hypercholesterolemia to an investigational new drug application in 2007. The systemically administered RNAi therapeutic is expected to employ a lipid nanoparticle formulation. "The licensing of Inex's delivery technology is another example of Alnylam's strategy of consolidating intellectual property critical for the development and commercialization of RNAi therapeutics," says Robert Millman, chief intellectual property counsel for Alnylam.