EHA 2025 | Early findings on the safety and efficacy of targeting calreticulin in ET and myelofibrosis

That’s a really exciting stuff coming into the field of patients with mutated calreticulin, which we have discovered just a few years ago. It is an immunogenic compound because it goes on the cell surface, so we could target it. So there are companies developing monoclonal antibodies that could bind to calreticulin and then literally disembark it. So we’re kind of looking at eradicating calreticulin mutated MPNs, super exciting for ET and myelofibrosis. So here at the EHA, there is actually a session in the plenary presented on data on ET patients who were treated with the monoclonal antibody, which is the naked one that enrolled high-risk ET patients that required therapies, and more exciting to me, it’s safety – there was no safety concern even in the higher dose established, of course, it’s a phase one dose-finding study, the different dose escalations, but even in the highest dose, there have been any DLT’s or significant concerns for continuation of these therapies, which is very important for somebody who has ET because this is a very benign risk where patients could live very long with the disease. Very nice data, all preliminary, of course, of efficacy, because it’s an early phase, so efficacy is going to be explored more on what we call the recommended phase two dose, which will be the effective dose of the antibody, but so far, the data look very nice in terms of control of the blood counts, particularly platelets, as well as LDH, and then there are also promising data basically emerging on fibrosis, and that’s what I’m really excited into calreticulin levels, because although there is no clinical significance or relevance right now, because we have never really had a drug that will go deeper inside and attack the calreticulin, remove it, and see what it could mean for patients. So exciting era to see more targeted calreticulin-directed. There are more drugs, of course, coming, and there will be, I guess, more approaches to it, to making these monoclonals even more smart, not naked, but we’ll see how they go. But I think this is a really breakthrough, and I really do hope this is a new era of what I would say calreticulin mutated MPNs. So when hopefully a couple of years will enter where we will be actually reclassifying these patients to be belonging somewhere else where we could cure them, not only prolong their lives.

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