IMerge: Updated Safety and Efficacy Data on Imetelstat for Lower-Risk MDS

Hematology experts highlight recent data updates from the IMerge trial on the telomerase inhibitor imetelstat, which is being studied for lower-risk MDS.


Amer Zeidan, MBBS: We can talk more about the advances we’ve seen in lower-risk MDS [myelodysplastic syndromes]. There have been a couple of updates on 2 of the most promising drugs that we have in the lower-risk MDS space: imetelstat and luspatercept. Luspatercept is already approved. Imetelstat has completed its phase 3 trial, and we’re expecting results in 2023. Can you highlight your thoughts and some of the recent updates?

Rami Komrokji, MD: Let’s start with imetelstat. This is a telomerase inhibitor that finished phase 3 study. Hopefully, we’ll have the readout in January or February. We hope it will be positive because that will lead to a new drug approval for lower-risk MDS. The phase 2 study has been published in Journal of Clinical Oncology. Dr [Uwe] Platzbecker presented data on a subset of patients at ASH [American Society of Hematology Annual Meeting] that had durable response. Imetelstat is an IV [intravenous] infusion given every 4 weeks. In the phase 2 data, around 42% of the patients became transfusion independent, and a subset of patients had a durable transfusion independence for more than a year.

At ASH, there was a focus on that subset of patients. Eleven patients had more than 1 year of transfusion independency. Most of them [had] MDS with ring sideroblasts or the SF3B1-mutant type. Those patients had durable responses. Interestingly, in patients with SF3B1, we see a signal of reduction on the allele burden, suggesting that this can be disease modifying. In terms of safety, the drug does cause myelosuppression, so patients can develop grade 3 or 4 thrombocytopenia or neutropenia. Most of them are reversible by the second round of treatment. This was an update on the transfusion dependency, but the data look strong from the phase 2 trial, and we hope that the phase 3 trial will confirm the signal and lead to approval.

Transcript edited for clarity.

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