Genzyme
Corp. (NASDAQ: GENZ) and Isis
Pharmaceuticals Inc. (NASDAQ: ISIS) today announced that data from a
phase 3 study of mipomersen in patients with homozygous familial
hypercholesterolemia (hoFH) were published in The Lancet. This
study met its primary endpoint, resulting in an average LDL-C reduction
of greater than 100 mg/dL in this very high-risk patient population.
“Mipomersen has the potential to set a new standard of
care for this difficult-to-treat disease.”
“Currently available treatments do not provide adequate lipid lowering
for hoFH patients, leaving them at extraordinarily high risk for
cardiovascular events,” said Professor Frederick J. Raal, M.D., Ph.D.,
Director of the Carbohydrate and Lipid Metabolism Research Unit at the
University of the Witwatersrand in South Africa, and the study’s primary
investigator. “Mipomersen has the potential to set a new standard of
care for this difficult-to-treat disease.”
The trial, one of the largest conducted to date in this rare patient
population, was designed to test the efficacy and safety of adding
mipomersen to substantial lipid-lowering therapy. Patients treated with
mipomersen had a 25 percent LDL-C reduction in an intent-to-treat
analysis. In addition to meeting its primary endpoint, the trial also
met each of its secondary and tertiary endpoints, which included
statistically significant reductions in apolipoprotein-B, total
cholesterol, non-HDL cholesterol, Lp(a), VLDL-C and triglycerides.
Familial hypercholesterolemia (FH) is a genetic disorder that results in
elevated LDL cholesterol levels. FH patients experience a markedly
increased risk of premature cardiovascular disease (CVD) and CVD-related
death. There are two forms of FH: homozygous, estimated to affect
approximately one in a million people worldwide; and heterozygous, a
more common form of the disorder with a prevalence of approximately one
in 500. In patients with hoFH, the first cardiovascular events can occur
in childhood or adolescence. Without lipid-lowering therapy, hoFH
patients rarely live beyond age 30.
Although all but one of the 51 patients in the phase 3 study were being
treated with lipid-lowering therapy, their average LDL-C at baseline was
greater than 400 mg/dL. The LDL-C reductions observed in the study were
in addition to those achieved with the patients’ existing therapeutic
regimens.