Glaxo-Regulus Kicks Off an miRNA Land Grab

(MoneyWatch)  GlaxoSmithKline's willingness to bet up to $600 million on a tiny biotech called Regulus Therapeutics kicks off the latest pharma land grab -- this one for treatments that rely on tiny molecules called microRNAs, or miRNAs for short.

New drug technologies -- or "modalities," as the industry terms them -- bloom like perennials in this industry, and often fade just as fast. But no one wants to pass entirely, only to find themselves on the sidelines whenever the technique finally proves itself. Think of the now-dead biotechs that gave up on monoclonal antibodies years before Genentech proved they could yield blockbuster drugs.

Thus the latest enthusiasm for miRNAs, a new way of "regulating" genes. In lab experiments, these tiny nucleic-acid molecules act as cellular dimmer switches that can turn beneficial genes "up bright" or "switch off" the activity of harmful gene mutants. (In this respect they resemble their cousins siRNAs -- short for "small interfering RNAs" -- the main difference being that miRNAs are coded directly by the genome instead of being produced as a side effect of other cellular processes, as I understand it.)

Since many miRNAs seem to be associated with cancer, researchers think it might be possible to use them as early-warning detectors for tumors. Making drugs that interfere with other miRNAs -- kind of like hitting a bullet with a bullet -- might yield useful treatments.

Glaxo's deal with Regulus takes the form of a fairly standard pharma-biotech partnership, in which the big company puts up cash over time in exchange for an option to promising drugs as they emerge from development. Regulus gets $20 million up front, plus up to $144.5 million in milestone payments for each of up to four drug candidates.

This is Glaxo's second deal in miRNA -- last December, it reached a similar arrangement with the Danish biotech Santaris Pharma for new antiviral drugs that could be worth over $700 million. (Remember, though, that most partnerships of this sort never amount to more than a fraction of these headline numbers.) One other recent rumble: The Texas biotech Asuragen just spun out its miRNA operations as a new startup called Mirna Therapeutics and seeded it with $3 million.

With two potentially big deals on the table, expect other pharmas and big biotechs to be planting their own flags in this virgin territory before long.

(Photo by Flickr user mav.mbecker.net, CC 2.0)