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vupanorsen
- Development stage
- Discontinued
- Lead developer
- Pfizer
- 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
Vupanorsen is an investigational N-acetyl galactosamine-conjugated antisense oligonucleotide designed to target and inhibit the hepatic production of angiopoietin-like 3 (ANGPTL3) protein by binding to ANGPTL3 mRNA in the liver. ANGPTL3 is a key regulator of triglyceride and cholesterol metabolism; its inhibition leads to reductions in triglycerides, non–high-density lipoprotein cholesterol (non-HDL-C), low-density lipoprotein cholesterol (LDL-C), and apolipoprotein B-containing lipoproteins. Vupanorsen was developed using Ionis Pharmaceuticals' LIgand Conjugated Antisense (LICA) technology platform. The drug was primarily investigated for cardiovascular risk reduction and severe hypertriglyceridemia, including in patients with diabetes, hepatic steatosis, and dyslipidemia. Clinical development was discontinued after Phase 2b due to insufficient efficacy for cardiovascular risk reduction or severe hypertriglyceridemia relative to safety concerns such as dose-dependent increases in liver fat and elevations in liver enzymes[1][2][5][7].
- Brand names
- vupanorsen
- Other names
- vupanorsen
02
Targets
ANGPTL3 (Angiopoietin-like protein 3)
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