ASH 2025 | An overview of ongoing Phase III trials investigating novel treatment options for myelofibrosis

We’re in an exciting era of numerous Phase III trials looking at combinations for patients with myelofibrosis. And I think starting with that JAK inhibitor base, JAK inhibitors make a big impact. Spleen symptoms, they have some impact on disease progression. They’re good drugs. But how do we build upon that? So I think leading candidates, pelabresib as a BET inhibitor clearly has won. That’s probably been the most active to date in terms of further reduction in splenomegaly as well as, you know, at least as good for controlling symptoms. There’s additional BET inhibitors that are in the pipeline as well, but pelabresib I think is the most impressive data and again the gathering additional data that hopefully will help to support a registration. Other key ones include everything from the MDM2 inhibitor from Kartos that is navtemadlin, that’s having impressive activity and the POIESIS trial which is ongoing very interesting where they have a run-in of ruxolitinib and suboptimal responders. Then we’ll get randomized to receive the therapy. We’re hopeful to see that incremental benefit. The PIM kinase inhibitor, nuvisertib, is interesting. The combination with selinexor, you know, amongst many others that are coming. So I’m excited by both the number, the preliminary activity that these have seen. The data around the actual Phase IIIs will be very important as we try to get a sense of where they might fit in the treatment paradigm, but excited to see more options coming.

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