Pelacarsen (AKCEA-APO(a)-LRx) is a liver-specific antisense oligonucleotide against apolipoprotein(a) that reduces lipoprotein(a) up to 80% with good tolerability.
Mice dosed with Pelacarsen at dose levels of 0.3, 1, 3, and 10 mg/kg once weekly for 6 weeks had dose-dependent reductions in target mRNA expression. The model-estimated doses for Pelacarsen that produced 50% of maximum drug-induced inhibitory effect (ED50) for liver apo(a) mRNA and plasma apo(a) protein levels were 0.32 and 0.54 mg/kg/week, respectively.
<p >References:
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[3]. Yeang C, et al. Effect of Pelacarsen on Lipoprotein(a) Cholesterol and Corrected Low-Density Lipoprotein Cholesterol. J Am Coll Cardiol. 2022 Mar 22;79(11):1035-1046.