Imetelstat is a human telomerase inhibitor used to treat myelodysplastic syndromes in patients with transfusion-dependent anemia. Imetelstat is a first-in-class 18-amino acid oligonucleotide that directly and competitively inhibits human telomerase.
- Mechanism
- Curator reviewed · 1 reference
Imetelstat exerts its therapeutic effects by binding to the RNA template component of telomerase, directly inhibiting its activity. This inhibition leads to the progressive shortening of telomeres in cancerous cells, ultimately resulting in cell death. The selectivity of imetelstat for malignant cells is due to a relatively higher telomerase activity in myelodysplastic syndrome and malignant stem and progenitor cells compared to normal cells.
- Primary indication
- Imetelstat is indicated for the treatment of adult patients with low- to intermediate-1 risk myelodysplastic syndromes (MDS) with transfusion-dependent anemia requiring four or more red blood cell units over eight weeks who have not responded...Curator reviewed · 1 structured indication
- Formula / weight
- C148H211N68O53P13S13 · 4610.18 g/mol (avg)
- First approval
- United States, 2024 · European Union, 2025
- Code names
- GRN-163L
- Brand names
Resolves to
LVZYXEALRXBLJZ-UHFFFAOYSA-NSMILESCCCCCCCCCCCCCCCC(=O)NCC(O)COP(S)(=O)OCC1OC(CC1NP(S)(=O)OCC1OC(CC1NP(S)(=O)OCC1OC(CC1NP(S)(=O)OCC1OC(CC1NP(S)(=O)OCC1OC(CC1NP(S)(=O)OCC1OC(CC1NP(S)(=O)OCC1OC(CC1NP(S)(=O)OCC1OC(CC1NP(S)(=O)OCC1OC(CC1NP(S)(=O)OCC1OC(CC1NP(S)(=O)OCC1OC(CC1NP(S)(=O)OCC1OC(CC1NP(S)(=O)OCC1OC(CC1N)N1C=NC2=C1N=CN=C2N)N1C=NC2=C1N=CN=C2N)N1C=CC(N)=NC1=O)N1C=NC2=C1N=CN=C2N)N1C=NC2=C1N=C(N)NC2=O)N1C=NC2=C1N=CN=C2N)N1C=C(C)C(=O)NC1=O)N1C=C(C)C(=O)NC1=O)N1C=NC2=C1N=C(N)NC2=O)N1C=NC2=C1N=C(N)NC2=O)N1C=NC2=C1N=C(N)NC2=O)N1C=NC2=C1N=CN=C2N)N1C=C(C)C(=O)NC1=OWhat you can answer from here — as of September 23, 2026
Which drugs share a target with Imetelstat, and which of those have an active Phase 3 trial?
Create a free account or log in to explore the full data card.
Create Account