According to the results of an ongoing Phase 2 clinical trial, Isis Pharmaceutical's cholesterol drug candidate, ISIS 301012, was able to cut cholesterol levels in half among patients with homozygous familial hypercholesterolemia (HoFH). ISIS 301012 is a second-generation drug that selectively targets apoB-100, a protein critical to the synthesis and transport of low density lipoprotein cholesterol (LDL) and very low density lipoprotein cholesterol (VLDL).

With ISIS 301012, the patients achieved 45%, 50% and 51% additional reductions in LDL-cholesterol beyond that achieved with maximally-tolerated lipid-lowering therapy, with similar reductions in apoB of 45%, 43% and 54%. ISIS 301012 continued to be well tolerated in the study.

"The magnitude of LDL-cholesterol response in these three HoFH patients has surpassed anything I have seen in the last 30 years, including with statins," stated Evan Stein, M.D., Ph.D., Director, Metabolic and Atherosclerosis Research Center, Cincinnati, Ohio, lead investigator in the study. "It offers for the first time the potential for good, life-long control of LDL without expensive and invasive therapies such as apheresis, liver transplantation and other heroic measures we have tried over the years."

ISIS 301012 has received orphan drug status for the treatment of HoFH, one of the most severe and treatment-resistant forms of hypercholesterolemia.

More details from Isis Pharma's press release. Following this news, shares of Isis rose 68 cents, or 6.9 percent, to $10.54 in electronic premarket activity.