Table 3.

Key results from randomized placebo-controlled phase III clinical trials

% Change from Baseline	Drug	HoFH [18] Mipomersen (n = 34) Placebo (n = 17) [%]	Severe HC [36] Mipomersen (n = 39) Placebo (n = 18) [%]	HeFH with CAD [37] Mipomersen (n = 82) Placebo (n = 41) [%]	HC at high risk [38] Mipomersen (n = 101) Placebo (n = 50) [%]
LDL-C*	Mipomersen	−25	−36	−28	−37
	Placebo	−3	+13	+5	−5
apoB*	Mipomersen	−27	−36	−26	−38
	Placebo	−3	+11	+7	−4
Lp(a)*	Mipomersen	−31	−33	−21	−26
	Placebo	−8	−2	0	−0
TG**	Mipomersen	−17	−9	−14	−25
	Placebo	+0.4	+27	+1	+11
HDL-C***	Mipomersen	+15.1	+6	+3	+2
	Placebo	+3.9	+3	+6	+2

Data shown is based on the analysis of the intent-to-treat population (n), defined as those who received at least one dose of study drug and had at least one post-baseline LDL-C measurement

apoB apolipoprotein B, CAD coronary artery disease, HC hypercholesterolemia, HDL-C high-density lipoprotein cholesterol, HeFH heterozygous familial hypercholesterolem ia, HoFH homozygous familial hypercholesterolemia, Lp(a) lipoprotein(a), LDL-C low-density lipoprotein cholesterol, TG triglyceride

p-Values (vs. placebo): * p < 0.001 all populations, ** p < 0.05 all populations, *** p < 0.05 in HoFH population