FAMILIAL HYPERCHOLESTEROLEMIA
Familial Hypercholesterolemia (FH) is an autosomal
dominate disease which occurs about 1 in every 500
people (also known as
Heterozygous Familial Hypercholesterolemia (HeFH).
The homozygous form (HoFH) is rarer, occurring with
the frequency of about 1 in a million. The
statistics for the homozygous form is not surprising
though; since patients suffering from two alleles of
this gene usually do not survive pass their teens.
The condition of hypercholesterolemia in FH patients
is detectable at birth or shortly thereafter. The
cholesterol levels in heterozygous patients are
between 350 to 500 mg/dL, and in homozygous, the
levels are between 700 to 1,200 mg/dL.
SLx-4090
was designed to act selectively on MTP in gut
enterocytes while avoiding systemic exposure and the
resultant liver toxicity that has prevented
development of other compounds of this class. A
first time in man (FTIM) study was initiated in
March 2006 and dosing was completed in June 2006. A
repeat dose study (RDS) and a Phase IIa study in
patients with familial hypercholesterolemia study is
expected to start in Q3 2006. |
