CARLSBAD, Calif.—Operating on the belief that microRNA (miRNA) represents a completely new class of therapeutics that can impact diseases in fundamentally unique ways, Cambridge, Mass.-based Alnylam Pharmaceuticals Inc. and Carlsbad, Calif.-based Isis Pharmaceuticals Inc. have launched Regulus Therapeutics LLC, a joint venture focused on the discovery, development and commercialization of miRNA therapeutics.
"The opportunity to antagonize microRNAs could create a new frontier for pharmaceutical research where an entire disease pathway is targeted for intervention, not just a single disease target..[and] have profound implications for the treatment of a broad range of diseases including cancer, viral infection and metabolic disorders," said Dr. John Maraganore, president and CEO of Alnylam, in a statement announcing the deal. Combining Isis' and Alnylam's intellectual property (IP) and technologies to form Regulus creates what he calls, "an unmatched effort to establish the leading microRNA therapeutics company."
Both of the founding companies will grant Regulus exclusive licenses to their IP for miRNA therapeutic applications, as well as certain early fundamental patents in the miRNA field including the Tuschl III patent.
"John Maraganore has already said this, but this really is a case where one plus one truly equals five," says Alnylam's COO, Barry Greene. "By forming Regulus, we are putting together the premier miRNA therapeutics company. Isis is the best company in the world for ogligonucleotide chemistry and has more than 18 years experience in creating single-stranded RNA, while Alnylam brings leading expertise in RNAi. And by working together and sharing IP, we are creating an unmatched single force for investment in the science and creation of a broad portfolio of products in this field, plus improved opportunities at the business development end as well."
Dr. Stanley Crooke, chairman and CEO of Isis, agrees with that assessment, noting both companies have spent a lot of time on building up their IP for miRNA therapeutics and that both have considered the possibility of either traditional collaboration models or even the formation of a venture-capital-based company.
But the joint venture model offered some key advantages, Crooke notes. First, forming a separate enterprise ensures a high level of focus on miRNA exclusively and provides the opportunity for a scientific advisory board built around miRNA alone. Also, a separate company means that Alnylam and Isis can focus on locating a strong, independent leader for the enterprise who also will be focused on miRNA and not on any other areas that Isis and Alnylam pursue. By combining their strengths to do this, Alnylam and Isis are, Crooke says, essentially making a company that has all the strengths of a startup with none of the detriments.
"Finally, having a separate company makes it clear that Regulus is the one place to stop and shop for miRNA partnerships and deals," Crooke says. "It's not necessarily obvious to anyone that Alnylam or Isis are strong players in miRNA individually, but with a company devoted to miRNA, that focus becomes very clear to people outside the companies."
Regulus will be operated as an independent company with an independent board of directors and management team, but Alnylam and Isis will retain rights to develop and commercialize, on pre-negotiated terms, miRNA therapeutic products that Regulus decides not to develop either itself or with a partner. The new company will be based in Carlsbad because researchers at Isis have already put significant upfront work into creating miRNA antagonists and co-locating the companies is more efficient right now, though it is possible that Regulus' management team may choose to move at some point in the future, Crooke and Greene note.
The joint venture is ultimately a 50-50 split, but to get things started, Alnylam is making an initial investment of $10 million to "balance venture ownership." Thereafter, Isis and Alnylam will share funding of Regulus. As Crooke explains, the two companies agreed that the value of the combined intellectual property was more heavily weighted toward Isis' IP, and thus the $10 million represents a kind of "buy-in" fee for Alnylam in the joint venture.