| Chemical Properties | Back Directory | [form ]
Solid | [color ]
White to off-white | [Sequence]
DNA, d([2′-O-(2-methoxyethyl)]m5rU-sp-[2′-O-(2-methoxyethyl)]rG-[2′-O-(2-methoxyethyl)]m5rC-[2′-O-(2-methoxyethyl)]m5rU-[2′-O-(2-methoxyethyl)]m5rC-m5C-sp-G-sp-T-sp-T-sp-G-sp-G-sp-T-sp-G-sp-m5C-sp-T-sp-[2′-O-(2-methoxyethyl)]m5rU-[2′-O-(2-methoxyethyl)]rG-[2′-O-(2-methoxyethyl)]m5rU-sp-[2′-O-(2-methoxyethyl)]m5rU-sp-[2′-O-(2-methoxyethyl)]m5rC), 5′-[26-[[2-(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]-14,14-bis[[3-[[6-[[2-(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]hexyl]amino]-3-oxopropoxy]methyl]-8,12,19-trioxo-16-oxa-7,13,20-triazahexacos-1-yl hydrogen phosphate] |
| Hazard Information | Back Directory | [Uses]
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]. | [in vivo]
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. | [References]
[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. DOI:10.1038/mtna.2016.26 [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. DOI:10.1089/nat.2016.0623 [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. DOI:10.1016/j.jacc.2021.12.032 |
|
|