ASH 2021 | Phase II trial of sotatercept for anemia of MPN-associated myelofibrosis
Prithviraj Bose, MD, The University of Texas MD Anderson Cancer, Houston, TX, shares the findings of a Phase II open-label study (NCT01712308) of sotatercept to treat anemia in patients with myeloproliferative neoplasm (MPN)-associated myelofibrosis (MF). The first-in-class activin receptor type IIA ligand trap was administered subcutaneously to 46 evaluable patients as a monotherapy or in combination with stable dose ruxolitinib. Anemia responses, defined as the achievement of transfusion independence or a sustained increase in baseline hemoglobin level in non-dependent patients, were achieved in 30% when used alone and 32% in combination with ruxolitinib. Sotatercept was well-tolerated overall. Grade 3 adverse events included hypertension, and limb or back pain. This interview took place at the 63rd ASH Annual Meeting and Exposition congress in Atlanta, GA.
Transcript (edited for clarity)
This is an investigator sponsor trial that we’ve been conducting for a while, and I’ve presented results from this before, but these are the final results, and we found a response rate actually very similar in our two cohorts, right around 30%, one was 30, the other was 32. These cohorts being the monotherapy cohort and the cohort of patients who are on a stable dose of ruxolitinib. So sotatercept just to back up a little bit is an activin receptor-ligand trap, a novel mechanism to combat anemia, in this case in myelofibrosis...
This is an investigator sponsor trial that we’ve been conducting for a while, and I’ve presented results from this before, but these are the final results, and we found a response rate actually very similar in our two cohorts, right around 30%, one was 30, the other was 32. These cohorts being the monotherapy cohort and the cohort of patients who are on a stable dose of ruxolitinib. So sotatercept just to back up a little bit is an activin receptor-ligand trap, a novel mechanism to combat anemia, in this case in myelofibrosis. The drug has been studied in MDS as well. And we found that it’s clearly active, it’s very safe. In some patients, they get a remarkable response, they even have to hold the drug for multiple cycles because the hemoglobin overshoots and you wait until it comes back down.
But of course, in others, it does not have much of an effect, but clearly, an active drug, novel mechanism, luspatercept which is sort of a counterpart of it, or I should say, close a cousin of sotatercept is the one being developed, and that’s already on the market for beta-thalassemia and MDS and is being developed for myelofibrosis. So this should be… This class of drugs, if not sotatercept, this class of drugs should be a very helpful addition to the arsenal for anemia and myelofibrosis. And so we hope to see luspatercept approved and available for MF patients.
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