IMerge Phase 3: Long-Term Results of Imetelstat in Lower-Risk MDS | Valeria Santini, MD

Hi, my name is Valeria Santini. I am the chair of the MDS unit in the Hematology Department of the University of Florence, Italy. Here at ASH, I presented the update of the eMERGE trial. The eMERGE trial is a study including patients with low-risk myelodysplastic syndrome who need transfusion. Not only that, but the patient who has a high burden of transfusion, meaning more than four red blood cell units in eight weeks. These patients were treated with Imethylstat, that is a new IV drug given every four weeks, and compared the result with the placebo. So the study was successful. The drug was approved on the basis of the achievement of 40% of transfusion independence, meaning that of all the patients, 40% could get rid of their dependence from the hospital and transfusions. And the increase of hemoglobin was quite important and relevant, achieving near normal values. Now, this study was updated ASH 2025, giving an idea of what we can expect from this increase of hemoglobin and achievement of transfusion independence in terms of prolongation of survival. So we had a comparison of patients treated with Imethylstat to patients treated with placebo with a long follow-up. So what we could see was that there was an advantage for survival to patients who received Imethylstat. And this was especially true in the patient that achieved more than one year of freedom from transfusion, which means a continuous period of transfusion independence longer than one year. This patient had, let's say, a nominal, so a little bit of advantage. We have to prolong our observation to be sure that we can see the difference. These are patients with low risk, so they have quite a long life expectancy. But we have to verify what we see now that is an advantage for the ones who were treated with Imethylstat. Imethylstat is a drug that gives temporary thrombocytopenia and neutropenia that are reversible. But anyway, it's a drug that is promising also because it has apparently disease modifying effects. So it lowers the burden of the disease. Please consider making a donation to help us continue this important work. Your gift will go three times as far when we reach $500,000 by the end of the year. Every contribution, big or small, makes a difference, and we're deeply grateful for your support.