Core Insights - Geron Corporation presented new data at the ASH 2025 Annual Meeting, reinforcing the potential of RYTELO (imetelstat) as a treatment for lower-risk myelodysplastic syndromes (LR-MDS) and myelofibrosis (MF) [1][2][4] Group 1: Clinical Efficacy of Imetelstat - Pooled analyses from the IMerge trial suggest that early treatment-emergent cytopenias may correlate with significant clinical outcomes, including hemoglobin increases and transfusion independence in LR-MDS patients [2][4][6] - The data indicate that patients experiencing ≥75% reductions in neutrophils or ≥50% reductions in platelets during the first two cycles of treatment had greater maximum hemoglobin increases compared to those without such reductions [5][6] - Long-term follow-up from the IMerge trial shows favorable trends in overall survival (OS) and progression-free survival (PFS) for imetelstat-treated patients compared to placebo, with a median follow-up of 45 months [7][8][9] Group 2: Mechanism of Action and Biological Insights - Imetelstat's mechanism involves telomerase inhibition, which is crucial for the uncontrolled cell division seen in LR-MDS, and the treatment has shown potential disease-modifying properties [19][20] - The correlation between early blood count changes and later improvements in hemoglobin provides valuable insights for clinicians regarding the treatment's biological effects [3][6] Group 3: Additional Studies and Future Directions - Ongoing studies, including the IMproveMF trial, are evaluating the safety and efficacy of imetelstat in combination with ruxolitinib for patients with intermediate-2 or high-risk myelofibrosis [13][14] - Preliminary results from the IMpress study indicate that imetelstat has a manageable safety profile in advanced myelodysplastic neoplasms or acute myeloid leukemia patients who are refractory to prior treatments [15][16][17]