GSK Thinks Small, Again

GSK said today it had entered a strategic alliance with microRNA specialist Regulus Therapeutics in the inflammatory disease area, marking its second move (two more moves than any other big pharma) in the young field of microRNA inhibition. MicroRNAs are a newly discovered, broad class of drug targets.

Regulus gets $20 million up-front from GSK which includes a $5 million note that will eventually convert to Regulus common stock. Regulus handles development of microRNA antagonists against four undisclosed inflammatory targets through proof-of-concept, at which point GSK can opt in; total milestones (both pre- and post-POC) are up to $144.5 million per project, and Regulus will receive up to double-digit royalties on sales.

If the deal seems a bit familiar that may be because only a few months ago, in December 2007, GSK's infectious disease CEDD inked a similar deal with Danish microRNA player Santaris for up to four different virology programs, in a deal with similar terms, that includes an option on Santaris' SPC3649 program targeting microRNA-122 in HCV (that program was recently the subject of a write-up in Nature). In both the Regulus and Santaris deals, GSK is specifying the targets.

Both Santaris and Regulus are developing single-stranded oligonucleotide (antisense) therapies against RNA; microRNAs are single-stranded RNA oligonucleotides that may act as master gene expression regulators. (For those of you trying to keeping score of oligo drug strategies at home, you can't use the RNAi mechanism to destroy miRNAs.)

Regulus president and CEO Kleanthis Xanthopoulos told IN VIVO Blog this morning that the firm could earn "frequent and significant" milestones leading up to Glaxo's proof-of-concept decision timing, presumably enough to cover development costs. Xanthopoulos estimated that molecules from the collaboration could begin hitting the clinic in the next two-to-three years.

Though only a few months old, Regulus is not a typical start-up; it is a well-funded JV between antisense leader Isis and RNAi leader Alnylam. (Look for a feature about Isis and its platform strategy in the April IN VIVO.) Part of Regulus's strategy is to quickly establish a base of IP around microRNA targets, as Isis did with antisense and Alnylam with RNAi.

But because miRNAs are targets, there is not likely to be any broadly enabling IP that would lock up the space--it'll have to be target specific. That should make microRNA players attractive collaborators for early-stage deals like GSK's. Expect more of them, soon.

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