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factor XI antisense oligonucleotide
- Development stage
- Discontinued
- Lead developer
- Ionis Pharmaceuticals
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Subcutaneous
01
Overview
Factor XI antisense oligonucleotide (IONIS-FXIRx, also known as BAY-2306001, ISIS-416858, or ISIS-404071) is an investigational second-generation antisense oligonucleotide (ASO) developed by Ionis Pharmaceuticals in collaboration with Bayer. It is designed to target and degrade the mRNA of coagulation factor XI (FXI) in the liver, thereby reducing hepatic synthesis and circulating plasma levels of FXI. By selectively inhibiting the intrinsic pathway of coagulation, the drug aims to provide effective antithrombotic protection with a significantly lower risk of bleeding compared to traditional anticoagulants. It has been clinically evaluated in Phase 2 trials for the prevention of venous thromboembolism (VTE) in patients undergoing elective total knee arthroplasty and for the prevention of thrombosis in patients with end-stage renal disease (ESRD) on hemodialysis. Further development of this first-generation unconjugated ASO was discontinued in favor of fesomersen (IONIS-FXI-LRx), a second-generation GalNAc-conjugated version that allows for less frequent monthly dosing.
- Other names
- FXI ASOFXI-ASOfactor XI antisense inhibitor
02
Targets
RNASEH1 (Ribonuclease H1)F11 (Coagulation Factor XI)
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