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Imetelstat

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First-in-class telomerase inhibitor approved for specified lower-risk MDS patients and still in Phase 3 myelofibrosis development.

Modality
telomerase-template antagonist oligonucleotide
Availability (US)
Approved in the United States and European Union for specified lower-risk MDS patients; Phase 3 myelofibrosis study Active, not recruiting
Biomarkers
red-blood-cell transfusion independence; hemoglobin; spleen response; symptom response; overall survival
Hallmark targeted
telomere maintenance, replicative immortality, malignant stem and progenitor cells
Kind
approved drug and investigational oncology therapy
Clinical trials
NCT04576156 — Phase 3, Active, not recruiting on July 8, 2026 update; NCT02598661 — Phase 2/Phase 3, Active, not recruiting on July 17, 2026 update