Investigators tout a new therapeutic approach to cardiovascular disease

Regulus Therapeutics thinks a recent batch of non-human primate data indicates that it may be on the right path to developing a new treatment for cardiovascular disease. One of the central focuses of several clinical programs now involves experimental treatments that can both raise the level of good cholesterol, HDL, while lowering the "bad" cholesterol, LDL. And investigators at NYU Langone Medical Center say that targeting microRNA-33 looks promising.

The scientists chose to advance beyond mouse models because non-human primates express two forms of micro-RNA33--A and B--just like humans. After 8 weeks of treatment the primates experienced an increase of up to 50% in HDL and a similar drop in LDL. In the next step scientists will observe if the treatment can flush cholesterol out of the arteries, helping to prevent atherosclerosis.

"It's a very big problem," says Ryan Temel, Ph.D., an assistant professor at Wake Forest Baptist and co-principal investigator. "The ideal therapy would not only reduce cholesterol accumulation in the arteries by lowering bad cholesterol but also increase the removal of existing cholesterol in the arteries by elevating good cholesterol. The combination of a statin and anti-miR-33 could potentially be this therapy. While there is still a lot of work that needs to be done with this drug before it can ever be used in humans, anti-miR-33 is showing strong potential as a new therapy for reducing coronary heart disease risk."

The investigators note that there's no currently available treatment that raises HDL and lowers LDL, but they don't say that there are a number of advanced clinical programs that do just that. In particular there's Merck's ($MRK) anacetrapib and dalcetrapib from Roche, which target the very same process that Regulus is interested in. Both of those programs are in late-stage development, and have been routinely touted as top blockbuster prospects.

- here's the press release