hTERT reduction by imetelstat correlates with clinical activity in MDS

Imetelstat is a first-in-class competitive inhibitor of telomerase enzymatic activity, shown in the Phase II IMerge study (NCT02598661) to achieve durable transfusion independence in patents with lower-risk myelodysplastic syndromes (MDS) relapsed/refractory to erythropoiesis stimulating agents. Valeria Santini, MD, University of Florence, Florence, Italy, shares the findings of an investigation into the on-target activity of imetelstat and the clinical benefits demonstrated in the trial. An optimal pharmacodynamic (PD) effect was defined as ≥50% hTERT reduction by imetelstat. The study revealed a significant correlation between optimal PD effect and clinical benefits. Patients who achieved a ≥50% hTERT reduction had a greater reduction in transfusions and a significantly longer median transfusion independence duration, compared to patients who did not show optimal on-target activity. This on-target activity was not accompanied by higher rates of cytopenias or liver enzyme elevations. This interview took place at the 63rd ASH Annual Meeting and Exposition congress in Atlanta, GA.