Regulus Therapeutics - 2008 Fierce 15

Regulus Therapeutics 

Based: Carlsbad, CA
Founded: 2007 
Website: www.regulusrx.com [1] 

The Scoop: Spawned by two powerhouses in the RNA field--Isis Pharmaceuticals and Alnylam Pharmaceuticals--Regulus Therapeutics got started at an advanced stage in the emerging microRNA field. And the company was quick to capitalize on its hefty IP with a $600 million pact with GlaxoSmithKline. Anyone tracking microRNA drug development should pay close attention. 

What makes it Fierce: For a small, newly minted biotech company, Regulus has some very big allies. Alnylam Pharmaceuticals and Isis Pharmaceuticals spawned the mcroRNA developer and gifted it not only with years of IP but a backup group that can lend assistance to Regulus as it advances new therapies in the pipeline. 

That's something that GlaxoSmithKline understood early on; another reason why the pharma giant was willing to commit to a $600 million development deal with the startup. 

"It surprised even me that the deal went so quickly," says CEO Kleanthis Xanthopoulis, who joined the company last December. "I don't recall a company that was formed and so early on was able to conclude a $600 million deal. It reflects the excitement in the pharma industry in a very new company with years of research." 

About 500 microRNAs, a new category of RNA, have been identified that regulate the expression of some 30 percent of all human genes. Understanding how that works has the potential to deliver a range of new therapies covering multiple disease categories. 

Regulus' lead therapy targets miR-122 in the quest for a new anti-viral drug for hepatitis C. Other programs are being developed for cancer, metabolic diseases and inflammatory conditions. The collaboration with Glaxo is focused on developing four new therapies for rheumatoid arthritis and other inflammatory conditions. 

The founders are clearly set on creating a new player in the biotech industry with an independent future. 

"We want to build a company that discovers, develops and selectively commercializes microRNA therapeutics," says Xanthopoulos. "That's the bigger goal. We are not building the company for an acquisition." 

What to look for: Regulus says the Glaxo deal was just the first. More pacts are expected in the next year or two.  At the same time, Regulus plans to add two more development programs to its pipeline. That expansion will drive more hires, as the company grows to up to 45 employees by the end of '09. In 12 to 18 months, look for a new financing round. 

The 2008 Fierce 15 is brought to you by Quintiles [2]