Isis Pharmaceuticals Inc. (ISIS), Carlsbad, Calif., said an experimental drug for treating high cholesterol showed promise in mid-stage trials.
The drug, known only as "Isis 301012," proved effective in lowering blood levels of low-density lipoprotein - the so-called "bad" cholesterol - and related molecules such as triglycerides when administered alone or in combination with cholesterol-fighting drugs called statins.
Isis 301012, however, still hasn't demonstrated long-term safety, a task that could take several years. Some doctors worry that the drug, which blocks the production of an intermediate cholesterol molecule called ApoB-100, may leave fat trapped in the liver. If proven safe and effective, Isis 301012 could be one of the first practical applications of a so-far unproven drug technology called antisense, which aims to treat disease by blocking the function of individual genes.
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