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news.Isis Pharmaceuticals has initiated a development program of ISIS 301012 in patients with familial hypercholesterolemia, a genetic disorder that causes extremely high cholesterol levels and results in the early onset of heart disease.
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The studies of the drug in familial hypercholesterolemia (FH) are part of a wider developmental program for the drug as a treatment for high cholesterol.
ISIS 301012 reduces levels of apoB-100, a protein critical to the synthesis and transport of low density lipoprotein cholesterol, or the “bad” cholesterol involved in heart disease.
FH sufferers have elevated levels of “bad” cholesterol from birth which results in heart attacks at an early age. Most men with the disorder experience a heart attack before they reach their 60th birthday.
The phase II studies will evaluate the safety and efficacy of the drug in patients with FH who are not achieving their cholesterol target levels on lipid-lowering therapy. In the studies, patients will be dosed with 50 mg, 100 mg or 200 mg of ISIS 301012 for 6 weeks treatment period and then followed for 5 months.
“Demonstrating efficacy of ISIS 301012 in patients with FH may lead to an advance in therapy for these desperately ill patients and establish ISIS 301012 as a powerful new cholesterol-lowering drug. The results, if positive, could also provide a faster route to commercialization of the drug because of the unmet medical need in this patient population,” said Dr Mark Wedel, chief medical officer at Isis Pharmaceuticals.