Healthcare Industry News: ezetimibe
News Release - June 1, 2006
Isis Pharmaceuticals Receives Orphan Drug Status for ISIS 301012 for the Treatment of Homozygous Familial Hypercholesterolemia
CARLSBAD, Calif., June 1 (HSMN NewsFeed) -- Isis Pharmaceuticals, Inc. (Nasdaq: ISIS ) announced today that the U.S. Food and Drug Administration has granted orphan drug status to ISIS 301012 for the treatment of patients with homozygous familial hypercholesterolemia (HoFH). HoFH is a rare genetic disorder that results in extremely high cholesterol. Patients with this disease display signs of high cholesterol early in life and may experience heart attacks from early childhood. Treatment for HoFH is inadequate and many patients undergo an expensive and time consuming procedure called apheresis, a process similar to kidney dialysis to remove the "bad" cholesterol from the blood."This orphan drug designation underscores the need for improved therapies for familial hypercholesterolemia (FH). Isis is committed to rapidly developing ISIS 301012 for FH, as well as more routine forms of high cholesterol," said Mark Wedel, MD, JD, Senior Vice President of Development and Chief Medical Officer at Isis Pharmaceuticals.
ABOUT ORPHAN DRUG DESIGNATION
The Orphan Drug Act provides for economic incentives to encourage the development of drugs for diseases affecting fewer than 200,000 people in the United States. Orphan drug designation entitles Isis Pharmaceuticals to seven years of market exclusivity for ISIS 301012 for the treatment of patients with homozygous familial hypercholesterolemia. Additional incentives include tax credits related to clinical trial expenses, an exemption from the FDA-user fee, and FDA assistance in clinical trial design.
ABOUT ISIS 301012
ISIS 301012, a second-generation antisense drug, inhibits apoB-100, a protein critical to the synthesis and transport of the "bad" cholesterol involved in heart disease -- low density lipoprotein cholesterol (LDL) and very low density lipoprotein (VLDL). Lowering cholesterol and triglyceride levels is a key component in the prevention and management of cardiovascular disease.
Isis plans to rapidly develop ISIS 301012 for FH patients. This development plan has the potential to provide an accelerated pathway to commercialization because of the unmet medical need in this desperate patient population. In parallel, Isis plans to address the larger commercial market represented by the traditional population of patients with high cholesterol, who are not achieving their targeted cholesterol levels.
In April 2006, Isis announced initial data from the three low-dose cohorts of a Phase 2 clinical trial of ISIS 301012 as a single-agent in patients with high cholesterol. ISIS 301012 produced rapid, dose-dependent and prolonged reductions of its target, apoB-100, with concomitant reductions in LDL, VLDL, total cholesterol and triglyceride levels in patients with high cholesterol. At a dose of 200 mg/wk for three months, ISIS 301012 achieved a median percent reduction from baseline of 47% in apoB-100, 42% in LDL, 34% in total cholesterol and 46% in triglycerides at day 99. Isis is continuing this Phase 2 single-agent trial using higher dosing (300 and 400 mg/wk) of ISIS 301012.
In Phase 1 trials, ISIS 301012 produced rapid, dose-dependent and prolonged reductions of apoB-100 with concomitant reductions in LDL, VLDL, total cholesterol and triglycerides in normal subjects with elevated cholesterol. In a drug-drug interaction study, ISIS 301012 did not interact with simvastatin or ezetimibe, currently available lipid lowering drugs with which ISIS 301012 may be dosed in combination. ISIS 301012 has been well tolerated in all clinical trials to date.
ABOUT CHOLESTEROL AND CARDIOVASCULAR DISEASE
According to the American Heart Association, an estimated 106.9 million American adults have total blood cholesterol values of 200 mg/dL and higher, and of these about 37.7 million American adults have levels of 240 or above. In adults, total cholesterol levels of 240 mg/dL or higher are considered "high risk". Levels from 200 to 239 mg/dL are considered "borderline-high risk". LDL, known as the "bad" cholesterol, can clog arteries, increasing the risk of heart attack and stroke.
According to the World Health Organization (WHO), heart disease and stroke kill 17 million people a year, which is almost one-third of all deaths globally. By 2020, the WHO projects that heart disease and stroke will become the leading cause of both death and disability worldwide, with the number of fatalities projected to increase to over 20 million a year and by 2030 to over 24 million a year.
ABOUT FAMILIAL HYPERCHOLESTEROLEMIA
Familial hypercholesterolemia is a dominantly inherited genetic condition that results in markedly elevated LDL cholesterol levels beginning at birth, and resulting in heart attacks at an early age. Affected people have consistently high levels of LDL, which leads to premature atherosclerosis of the coronary arteries. It is possible for a person to inherit two genes for this disorder (homozygous familial hypercholesterolemia). This magnifies the severity of the condition. Cholesterol values in individuals with HoFH may exceed 600 mg/dL. Affected individuals develop waxy plaques (xanthomas) beneath the skin over their elbows, knees, buttocks. These are deposits of cholesterol in the skin. In addition, they develop cholesterol deposits in tendons and around the cornea of the eye. Atherosclerosis begins before puberty in HoFH patients and heart attacks and death may occur before age 20.
ABOUT ISIS PHARMACEUTICALS, INC.
Isis is exploiting its expertise in RNA to discover and develop novel drugs for its product pipeline and for its partners. The Company has successfully commercialized the world's first antisense drug and has 15 drugs in development. Isis' drug development programs are aimed at treating cardiovascular, metabolic and inflammatory diseases. Isis' partners are focused in disease areas such as inflammatory, ocular, viral and neurodegenerative diseases, and cancer. In its Ibis division, Isis is developing and commercializing the IBIS biosensor system, a revolutionary system to identify infectious organisms. As an innovator in RNA-based drug discovery and development, Isis is the owner or exclusive licensee of approximately 1,500 issued patents worldwide. Additional information about Isis is available at www.isispharm.com.
This press release includes forward-looking statements regarding the development, therapeutic potential and safety of ISIS 301012 to lower high cholesterol. Any statement describing Isis' goals, expectations, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement, including those statements that are described as Isis' goals. Such statements are subject to certain risks and uncertainties, particularly those inherent in the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, in developing and commercializing systems to identify infectious organisms that are effective and commercially attractive, and in the endeavor of building a business around such products. Isis' forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Isis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Isis. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Isis' programs are described in additional detail in Isis' annual report on Form 10-K for the year ended December 31, 2005, and its quarterly report on Form 10-Q for the quarter ended March 31, 2006, which are on file with the U.S. Securities and Exchange Commission (SEC). Copies of these and other documents are available from the Company.
Source: Isis Pharmaceuticals
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