Lipoprotein(a) Reduction in Persons with Cardiovascular Disease

This randomized, double-blind, placebo-controlled phase 2 trial assessed the efficacy and safety of AKCEA-APO(a)-LRx, an antisense oligonucleotide targeting hepatic apolipoprotein(a) production, in 286 patients with established cardiovascular disease and elevated lipoprotein(a) levels. Patients received various dosing regimens of APO(a)-LRx or placebo. The therapy yielded dose-dependent reductions in lipoprotein(a) levels, with the highest dose achieving a mean reduction of 80%. The proportion of patients reaching a lipoprotein(a) level of ≤50 mg/dL (125 nmol/L) reached 98% in the highest-dose group. No serious safety concerns were identified. Injection-site reactions were the most common adverse event. These results support the potential of APO(a)-LRx as a targeted therapy for elevated lipoprotein(a) in cardiovascular disease.

This randomized, double-blind, placebo-controlled phase 2 trial assessed the efficacy and safety of AKCEA-APO(a)-LRx, an antisense oligonucleotide targeting hepatic apolipoprotein(a) production, in 286 patients with established cardiovascular disease and elevated lipoprotein(a) levels. Patients received various dosing regimens of APO(a)-LRx or placebo. The therapy yielded dose-dependent reductions in lipoprotein(a) levels, with the highest dose achieving a mean reduction of 80%. The proportion of patients reaching a lipoprotein(a) level of ≤50 mg/dL (125 nmol/L) reached 98% in the highest-dose group. No serious safety concerns were identified. Injection-site reactions were the most common adverse event. These results support the potential of APO(a)-LRx as a targeted therapy for elevated lipoprotein(a) in cardiovascular disease.