Pelacarsen

• CAT Number: I043301
• CAS Number: 1637637-70-7
• Molecular Weight: 8636.00
• Purity: ≥95%

Pelacarsen (AKCEA-APO(a)-LRx) is a liver-specific antisense oligonucleotide against apolipoprotein(a) that reduces lipoprotein(a) up to 80% with good tolerability[1].
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.

Reference

[1]. Yu RZ, Graham MJ, Post N, et al. Disposition and Pharmacology of a GalNAc3-conjugated ASO Targeting Human Lipoprotein (a) in Mice. Mol Ther Nucleic Acids. 2016;5(5):e317.
 [Content Brief]

[2]. Yu RZ, Gunawan R, Post N, et al. Disposition and Pharmacokinetics of a GalNAc3-Conjugated Antisense Oligonucleotide Targeting Human Lipoprotein (a) in Monkeys. Nucleic Acid Ther. 2016;26(6):372-380.
 [Content Brief]

[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.
 [Content Brief]