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ISIS-APO(a)Rx
- Development stage
- Phase 2
- Lead developer
- Ionis Pharmaceuticals
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Subcutaneous
01
Overview
ISIS-APO(a)Rx (also known as ISIS 494372) is a first-generation antisense oligonucleotide (ASO) developed by Ionis Pharmaceuticals for the treatment of elevated lipoprotein(a) [Lp(a)] levels, a significant risk factor for cardiovascular disease. The drug is designed to specifically bind to the messenger RNA (mRNA) of the LPA gene, which encodes apolipoprotein(a), the distinguishing protein component of Lp(a) particles. By promoting the RNase H-mediated degradation of this mRNA, ISIS-APO(a)Rx inhibits the synthesis and secretion of Lp(a) from the liver. Although Phase 2 clinical trials demonstrated its ability to significantly lower plasma Lp(a) concentrations, development of this specific unconjugated molecule was discontinued in favor of a more potent, second-generation ligand-conjugated antisense (LICA) version, ISIS-APO(a)-LRx (now known as pelacarsen), which incorporates a triantennary N-acetylgalactosamine (GalNAc) moiety for targeted delivery to hepatocytes.
- Other names
- ISIS APO(a)Rx
02
Targets
LPA (Lysophosphatidic acid)
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