Sexual HealthGenzyme And Isis Announce That Mipomersen Phase 3 Study In Patients With Homozygous Familial Hypercholesterolemia Met Primary Endpoint
Genzyme Corp. (NASDAQ: GENZ) and Isis Pharmaceuticals Inc. (NASDAQ: ISIS) today announced that the phase 3 study of mipomersen in patients with homozygous familial hypercholesterolemia (hoFH) met its primary endpoint, with a 25 percent reduction in LDL cholesterol after 26 weeks of treatment, vs. 3 percent for placebo (pAbout Familial Hypercholesterolemia (FH)
FH is a genetic disorder in which patients are unable to properly metabolize LDL cholesterol, resulting in elevated LDL-C levels. FH patients experience a markedly increased risk of premature cardiovascular disease (CVD) and CVD-related death. There are two forms of FH: homozygous (hoFH), where the same defective gene is inherited from both parents, or heterozygous (heFH), where the defective gene is inherited from only one parent so that some function is preserved.
The homozygous form of FH is a very rare condition estimated to affect approximately one in a million people. HoFH patients can have LDL-C levels greater than 600 mg/dL and are at very high risk for early coronary events and sudden death. Because many patients are resistant to the lipid-lowering effects of currently available therapies, effective treatment of hoFH patients is difficult. HeFH is a more common form of the disorder, with a prevalence of approximately one in 500, and results in untreated LDL cholesterol levels of approximately 300 mg/dL, double those of the general population.
About Mipomersen
Mipomersen is a first-in-class apo-B synthesis inhibitor currently in late-stage development. It is intended to reduce LDL-C by preventing the formation of atherogenic lipoproteins. Genzyme and Isis are currently conducting several clinical trials of mipomersen, including:
- A phase 3 study in patients with heterozygous FH;
- A phase 3 study in patients with severe hypercholesterolemia;
- A phase 3 study in hypercholesterolemic patients at high risk for coronary heart disease; and
- A phase 2 study in high-risk, high-cholesterol patients who are intolerant to statins.
Data from these trials are expected to be available at the time of the initial hoFH regulatory filing, and will continue to build the body of clinical evidence around the treatment"s value in managing high-risk, high-cholesterol patients.
In 2008, Genzyme and Isis completed a licensing agreement that provides Genzyme with exclusive worldwide rights to mipomersen, which was discovered and initially developed by Isis.
Isis Pharmaceuticals Inc.