ASH 2024 | The latest advancements in disease-modifying therapies for myelofibrosis

At this ASH meeting 2024, we have very, very new information coming from target therapy. For instance, we have the navtemadlin study. The navtemadlin study is a study including patients in the second line set, the suboptimal response, receiving navtemadlin. It is an MDM2 inhibitor. and the results obtained are quite good in terms of clinical efficacy, so with the reduction of the spleen and improvement of symptomatology, but also we present some translational results. And the translational results indicate that using this new compound we can have a reduction of the clone of JAK2, a reduction of bone marrow fibrosis, and also a reduction of CD34 positive cells that are progenitors. So we can really determine some alteration of the biology. And this drug now is under development in the first line setting in patients JAK inhibitor naive. The name of the protocol is POIESIS. Then we have other information from a PIM inhibitor, nuvisertib. And this PIM inhibitor demonstrated in the Phase II study that there is efficacy as single agent in those patients with myelofibrosis who are resistant or relapsing after JAK inhibitor treatment. And the nuvi demonstrate to be effective on symptomatology, a very robust signal on symptomatology, and also effective in terms of spleen improvement, platelet improvement, and hemoglobin improvement. So this is a promising drug. Then we have also data on elritercept, the name is KER-050. And this is a TGF-β inhibitor that demonstrates to be effective against anemia. And this is a good drug used as a single agent, but also used as a combination of ruxolitinib plus the drug. And the effect of the anemia is quite good, very sensible, with also efficacy in terms spleen reduction symptomatology improvement.

This transcript is AI-generated. While we strive for accuracy, please verify this copy with the video.