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Obicetrapib Lowers LDL-C by 36% in Familial Hypercholesterolaemia: BROOKLYN Trial Results

The BROOKLYN trial randomised 354 patients with heterozygous familial hypercholesterolaemia to obicetrapib 10 mg or placebo for 365 days. Obicetrapib achieved a placebo-adjusted LDL-C reduction of 36.3%, apoB reduction of 24.4%, and Lp(a) reduction of 45.9% at day 84. These results position CETP inhibition as a viable add-on therapy and underscore the central role of advanced lipid panel testing including apoB and Lp(a) quantification in monitoring treatment response.

The original study

Obicetrapib in patients with heterozygous familial hypercholesterolemia: the BROOKLYN randomized clinical trial.

Authors
Nicholls SJ, Nelson AJ, Ditmarsch M, Kastelein JJP, Ballantyne CM, Ray KK, et al.
Journal
Nature medicine
Type
Journal Article, Randomized Controlled Trial
PMID
41760952
Read the original study →

Original abstract

Most patients with heterozygous familial hypercholesterolemia fail to achieve adequate low-density lipoprotein (LDL) cholesterol lowering. Here we carried out a randomized trial to test the safety and efficacy of obicetrapib, a highly selective cholesteryl ester transfer protein inhibitor that lowers LDL cholesterol levels in patients with heterozygous familial hypercholesterolemia and an LDL cholesterol level ≥70 mg dl-1 on maximally tolerated lipid-lowering therapy. The trial enrolled 354 patients (190 women, 164 men) with a mean LDL cholesterol level of 122 mg dl-1 (87% on statins) who were randomized (2:1) to receive obicetrapib 10 mg or placebo daily for 365 days. For the primary endpoint, the change in LDL cholesterol from baseline to day 84, obicetrapib treatment resulted in a placebo-adjusted change in LDL cholesterol of -36.3% (95% confidence interval -42.2% to -30.4%, P < 0.0001). In analyses of secondary endpoints at day 84, treatment with obicetrapib resulted in placebo-adjusted reductions in apolipoprotein B of -24.4%, non-HDL cholesterol of -34.5% and lipoprotein(a) of -45.9%, as well as a placebo-adjusted increase in high-density lipoprotein cholesterol of +138.7%. Obicetrapib was well tolerated. These findings suggest that obicetrapib is an effective therapy for additional lipid lowering in patients with heterozygous familial hypercholesterolemia. ClinicalTrials.gov registration: NCT05425745 .