Aptitude for Aptamers

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CAMBRIDGE, Mass.—January 17, 2007—Archemix announced two major collaboration agreements that will see it leverage its expertise in aptamer technology to target cancer for Merck KGaA. Archemix will receive upfront payments and research funding of up to $10 million, and could also receive milestone and royalty payments on any successful products. The deal comes 8 days after Archemix signed a similar agreement with Pfizer for the development and commercialization of aptamers against three unspecified disease targets. Although financial details were not disclosed, this deal will similarly see Archemix receive upfront cash payments, as well as research funding and development milestones, for their early-phase efforts, as well as possible royalties on any marketed products.
CAMBRIDGE, Mass.—Seeking to capitalize on the market potential of aptamers in much the same way other companies have with antibodies over the past decade, Archemix Corp. recently entered into a multi-year, multi-product alliance with neuroscience-based biotech company Elan Corp. that is potentially worth well over $300 million.
"I like to call aptamers 'chemical antibodies' because functionally they do what antibodies do but you get the controllability and manufacturing benefits that you typically see with small-molecule drugs," says Dr. Errol De Souza, president and CEO of Archemix. "Back in the mid-90s, there was a lot skepticism on the big pharma side about whether antibodies were really drugs, and at the time, there was only one antibody therapy on the market, and now they have taken off. Aptamers are in kind of the same place right now."
Specifically, the companies will seek to develop aptamer therapeutics to interleukin 23 (IL-23), a cytokine that has emerged as a mediator in chronic autoimmune inflammatory diseases—such as mul­tiple sclerosis, Crohn's disease, psoriasis, and rheu­matoid arthritis—and additional protein targets.
"Most compounds target both IL-12 and IL-23 in a non-selective manner," De Souza notes. "One of the strengths we have with the work we are doing with Elan is our demonstrated ability to build in selectivity to IL-23."
This is important, he says, because recent preclinical data suggest that IL-23 exerts its pro-inflammatory effects principally at the site of inflammation. The working hypothesis is that specific blockades of IL-23 may control clinical symptoms at the level of the inflamed tissue without generally suppressing the patient's immune system.
Under the terms of the agree­ment, Archemix will receive an upfront payment of $7 million. Depending upon the number of products successfully commer­cialized under the collaboration, Archemix is eligible to receive development and sales milestones in excess of $350 million. Archemix is also entitled to receive a royalty on any marketed products devel­oped under the collaboration.
Archemix also has an option to participate in the co-development of some products that may emerge from this collaboration, most nota­bly for psoriasis or non-parenteral administration of the compounds.
"This is not strictly an outli­censing deal whereby you get the upfront, milestone and royalties," De Souza says. "Even though our ability to co-develop is only for limited applications, I think that striking a deal like this shows we can be cognizant of the needs of the collaboration without losing sight of the needs of our shareholders."
The collaboration combines Archemix's extensive expertise in aptamer therapeutics with Elan's experience in the development and commercialization of new thera­pies for autoimmune diseases, as well as its knowledge of regulatory issues, De Souza says. Moreover, he notes, Elan is a company that "bet on antibodies before and now has a couple in clinical trials," and he believe that their backing of aptamer compounds now is a good sign of their potential suc­cess and a validation of everything Archemix has worked toward.
"By combining our strengths, this collaboration can accelerate the development of new therapies for chronic, debilitating diseases with continued unmet medi­cal needs," said G. Kelly Martin, Elan's president and CEO in a news release about the deal.

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