Expert: First-in-class telomerase inhibitor that targets the RNA template of human telomerase reverse transcriptase (hTERT). Binds to the active site of telomerase with high affinity, inhibiting enzymatic activity and leading to progressive telomere shortening in malignant clones. In MDS, selectively depletes the aberrant hematopoietic stem cells harboring driver mutations (SF3B1, TET2, DNMT3A) while sparing normal hematopoiesis, as evidenced by molecular response data showing >50% VAF reductions in driver mutations.

Everyday: Telomerase is an enzyme that cancer and abnormal blood cells use to keep dividing indefinitely - like an immortality switch. Imetelstat blocks this enzyme, causing those abnormal cells to eventually run out of fuel and die off, allowing healthy blood cell production to recover.

Targets: []

Primary EP: [{"id":"imerge-ep1","name":"RBC transfusion independence >=8 weeks","type":"PRIMARY","unit":"%","p_value":"<0.001","results":[{"ci":"30.9-49.3","arm":"imerge-imet","label":"39.8% (95% CI: 30.9-49.3)",

Safety: High rates of Grade 3-4 cytopenias: neutropenia 68% (vs 3% placebo), thrombocytopenia 62% (vs 8%). Cytopenias most frequent in cycles 1-3, generally manageable with dose modifications. ALT elevation 1

Rytelo - Lower-risk MDS - EU Country-by-Country Launch Throughout 2026

First-in-class telomerase inhibitor. FDA approved June 2024 for transfusion-dependent anemia in lower-risk MDS (non-del(5q), ESA-relapsed/refractory). 10-year orphan exclusivity in EU (EC approved March 2025). Geron holds US/ROW rights; SOBI commercializes EU. Key differentiator: disease-modifying potential via molecular responses (VAF reductions in SF3B1, TET2, DNMT3A driver mutations). Main safety concern: high-grade cytopenias (68% Gr3-4 neutropenia, 62% Gr3-4 thrombocytopenia) requiring close monitoring.