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Alnylam, Isis Create miRNA Drugs JV, Give New Firm Exclusive IP License

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Alnylam Pharmaceuticals and Isis Pharmaceuticals announced last week that they have formed and bankrolled a joint venture called Regulus Therapeutics, which will discover, develop, and commercialize microRNA-targeting therapeutics.
 
Alnylam and Isis have also armed Regulus with exclusive licenses to their intellectual property related to therapeutic applications of miRNAs — a move the companies expect will give them a portion of the revenues Regulus generates through partnerships with pharma and biotech firms looking to get in on the miRNA therapeutics game.
 
Among the IP now consolidated in Regulus is the so-called Tuschl-3 miRNA patent family held jointly by Isis and Alnylam (see RNAi News, 3/8/2007), as well as a portfolio of more than 900 patents and patent applications owned by Isis pertaining to chemical modification of single-stranded oligonucleotides for therapeutic applications, John Maraganore, president and CEO of Alnylam, said during a conference call discussing the joint venture. 
Because Isis’ IP contribution to Regulus outweighs Alnylam’s, Maraganore said that his company will also make an initial $10 million investment in the JV to get it up and running.  Each company owns approximately half of Regulus, according to an Alnylam filing with the US Securities and Exchange Commission.
 
Future investments in Regulus, which he said could be in the range of $15 million to $20 million over the next two years, will be made equally by Alnylam and Isis.
 
Still, much of Regulus’ funding down the road is expected to come from drug-development alliances, and it is the venture’s IP estate that is likely to give it the clout to negotiate such deals on favorable terms, Maraganore noted during the call.
 
“We’ve seen the type of leverage this type of consolidation of IP and science can create, perhaps exemplified most recently by Alnylam’s partnership with Roche,” he said, referring to Alnylam’s recent deal to non-exclusively license its fundamental RNAi IP to the big pharma for as much as $1 billion (see RNAi News, 7/12/2007). “We fully expect that [Regulus] will create the same type, if not greater, leverage for both Alnylam and Isis” in the miRNA field. 
 
As they look to cash in on Regulus’ licensing and drug-development deals, Alnylam and Isis are likely to maintain a significant presence in their joint venture even though the new company has been outfitted to run independently with its own management team, board of directors, and scientific advisors.
 
In fact, during the conference call, Isis Chairman and CEO Stanley Crooke noted that Regulus was established as a joint venture — as opposed to a spin out, for example — because the structure is the “best way for us to remain in control of the technology and advance it as rapidly as possible,” while giving Alnylam and Isis the freedom to focus on their core technologies of RNAi and antisense, respectively.
 
Regulus is currently headquartered in Carlsbad, Calif., within Isis’ facilities, and will have a scientific advisory board that includes four members of Alnylam’s SAB, three of whom co-founded the RNAi company.
 
David Baltimore, a Nobel Laureate and a professor at the California Institute of Technology, will chair Regulus’ SAB. Other members of the advisory board are expected to include Alnylam co-founder and scientific advisor David Bartel, who also serves as a professor at the Massachusetts Institute of Technology; Scott Hammond, an assistant professor at the University of North Carolina School of Medicine; and Markus Stoffel, a professor at the Swiss Federal Institute of Technology and a scientific advisor to Alnylam.
 
Also slated to join Regulus’ SAB is Thomas Tuschl, an Alnylam co-founder and SAB member and associate professor at Rockefeller University; and Phil Zamore, a professor at the University of Massachusetts Medical School and Alnylam co-founder and scientific advisor.
 
Additionally, Alnylam and Isis expect to handle certain of Regulus’ general and administrative activities, which would give the parent companies another layer of control over the JV.
 
“Over the next year to two years, [Regulus’ staff] should be well in the range of 20 or more employees that will be actively engaged in the R&D activities,” Maraganore said. However, “there will be elements related to the support of the JV from both of Alnylam and Isis on certain functions that may not necessarily require JV-specific infrastructure.”
 
Crooke noted that a number of employees from both Isis and Alnylam who have been involved in miRNA research are expected to join Regulus “immediately so that it will hit the ground running.”
 
IP, R&D JV
 
According to Isis’ Crooke, Regulus builds on his company’s existing relationship with Alnylam, which began in early 2004 when the companies formed an alliance that included cross-licensing of each others’ IP portfolios (see RNAi News, 3/19/2004).
 

The companies chose to form a joint venture because it “the best way for us to remain in control of the technology and advance it as rapidly as possible.”

Since then, the companies have co-exclusively licensed key miRNA-related IP from Max Planck and Stanford University (see RNAi News, 3/8/2007 and 9/16/2005).
 
Alnylam and Isis have also been conducting miRNA drug research in parallel, including work showing that miR-122, a miRNA associated with replication of the hepatitis C virus, can be targeted and silenced with antisense agents.
 
Given their success with miR-122 as a target for HCV, the companies have selected it as Regulus’ lead drug-development program, Maraganore said during the conference call.
 
Other pipeline candidates will be determined based on ongoing and future research. Under the terms of Regulus’ organization, pre-negotiated, undisclosed terms enable Alnylam and Isis to pick up any product candidate the joint venture decides not to develop on its own or with a partner.
 
Interestingly, Isis is also developing antisense agents against miR-122 as a treatment for liver cancer under an ongoing partnership with miRNA firm Rosetta Genomics (see RNAi News, 2/23/2006).
 
In an e-mail to RNAi News this week, an Isis spokeswoman characterized her company’s arrangement with Rosetta as “productive,” and said that “we expect the relationship to continue.”
 
She was not available to elaborate or confirm whether the Rosetta deal would be transferred to Regulus or remain with Isis.
 
Officials from Rosetta were not available for comment.
 
Other alliances Alnylam and Isis have forged previously do not include miRNAs and will not have any impact on Regulus, Maraganore noted during the conference call.

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