Zeidan Discusses Imetelstat for Lower-Risk MDS

Amer Zeidan, MBBS, associate professor at Yale School of Medicine, discusses imetelstat and the IMerge/MDS3001 study (NCT02598661) which evaluated the agent in patients with lower-risk myelodysplastic syndromes (MDS) with anemia who are transfusion-dependent and ineligible for erythropoiesis stimulating agents (ESA).

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0:09 | Imetelstat is a new first-in-class drug that has demonstrated activity in patients with lower-risk MDS who are anemic, needing blood transfusions after failure of ESA or who are ineligible, meaning unlikely to benefit from ESA. The clinical trial that hopefully [is] leading to the approval is a phase 3 trial called IMerge, which randomized patients against placebo and around 180 patients were included internationally. The primary end point was the transfusion independence among patients who are transfusion dependent at baseline.Currently, the new drug submission to the FDA is being considered, and hopefully, will be decided on soon.

1:04 | IMerge was a well designed and very well conducted study. It was, as I mentioned, randomized in a double blinded fashion in patients with lower-risk MDS who are anemic and needing transfusions…The drug was shown to be effective, it was better than placebo. The transfusion dependence at 8 weeks of around 40% compared with 15%. Also importantly, the duration of transfusion dependence was a median duration of around 52 weeks, and the median rise in hemoglobin for responders was quite good at around 3.6 grams rise in the responders for patients in the imetelstat arm compared with around a gram for patients in the placebo arm.

1:58 | And another important thing in this trial is that the drug, which is given subcutaneously every 4 weeks, was associated with treatment-related neutropenia and thrombocytopenia, which were grade 3 and higher in around 2 thirds of patients. However, most of those were reversible within 2 weeks of holding the dose or delaying the cycle or reducing the dose and did not lead to increase in the clinically significant adverse events such as bleeding and febrile neutropenia. I think overall, the efficacy and safety profile is favorable, and I think this drug would be a good addition to our patients.