Two years have passed since GlaxoSmithKline anointed a startup called Regulus Therapeutics as a leader in the bleeding-edge world of microRNA drugs, and now the pharma giant has made clear it likes what it sees so far.
Regulus, a Carlsbad, CA-based spinoff from neighboring Isis Pharmaceuticals and Cambridge, MA-based Alnylam Pharmaceuticals, is announcing today it has clinched a second collaboration with Glaxo to develop a microRNA drug for hepatitis C. The new deal provides undisclosed upfront cash, milestone payments potentially worth $150 million, and escalating royalties worth a double-digit percentage of worldwide sales if the drug reaches the market. Glaxo’s upfront payment reimburses Regulus for its R&D work to date, and the big company will assume the expenses of clinical trials.
This is the second time Glaxo has pulled out its checkbook for Regulus, after it agreed to pay as much as $600 million in April 2008 to develop drugs against four microRNA targets for inflammatory diseases. It was a strong statement for the field of microRNA therapies, which are thought to have broad potential as a new class of treatment because they can affect not just one gene or protein in isolation, but full networks of genes—which might be useful in treating complex diseases like diabetes or cancer. Regulus hasn’t yet advanced any of these drugs that work this way into a clinical trial. But this new deal will allow it to move its lead candidate, a blocker of microRNA-122, into its first clinical trial as a hepatitis C therapy in the second half of 2011, according to CEO Kleanthis Xanthopoulos.
“GSK really made a bold and visionary move in the fledgling field of microRNA a couple of years ago, and by selecting us as the lead company,” Xanthopoulos says.
Xanthopoulos picked Glaxo as the partner on this lead program partly because it shows the big company is happy with Regulus’ progress, the terms were good for a single drug program, and he personally knows Glaxo’s senior vice president of anti-infectives, Zhi Hong, has the right expertise to move ahead with the miR-122 blocker.
Regulus and Glaxo are moving ahead fast with this program partly because of the work done by a competitor, Denmark-based Santaris Pharma, Xanthopoulos says. Santaris published a big paper in Science in December that said its own miR-122 blocker produced a powerful effect against the hepatitis C virus in chimpanzees—an animal model that is thought to be highly predictive of what will happen next in humans. That study confirmed what Regulus was seeing in its own animal experiments, and helped accelerate its program by providing some more clarity on the appropriate dosing, Xanthopoulos says.
The unhappy result of the Santaris paper is that it potentially puts the companies on a collision course over who owns the relevant intellectual property. Regulus contends
