ASH 2020 | Imetelstat for transfusion-dependent R/R MDS

The IMerge study was actually presented, the Phase II part, presented at the ASH meeting. It’s about imetelstat in lower-risk MDS patients, being ESA refractory or not eligible for ESA or resistant. And this study actually investigates imetelstat in these patients and patients actually with a very high transfusion burden, actually median number of eight units of red blood cells within eight weeks prior to start of the therapy. So really, patients where nothing works, and therefore, the data and the results of the study were quite impressive with regards to the overall rate of transfusion independency, which was about 40%. And also, 29% of the patients who are transfusion-independent beyond one year. So also not only that the rate of transfusion independency was quite high, also, it was durable in approximately one third of the patients, which I think is quite remarkable results.

The study also did some translational research and found out that the allelic burden of mutations like SF3B1 declined at the time also of the response to imetelstat single-agent treatment, suggesting that the drug has disease modifying activity. These study results of the Phase II are the backbone for the ongoing Phase III trial with a 2:1 randomization imetelstat versus placebo in exactly the same patient population, so lower-risk MDS patients being refractory or resistant to ESA therapy and being transfusion-dependent. And the trial is running, and I think, hopefully, the study will pave the way for a potential registration of a novel agent in low-risk MDS patients.