Elritercept Well-Tolerated, Demonstrates Potential Efficacy for the Treatment of Patients With Myelofibrosis
Claire Harrison, MD, FRCP, shares results of the ongoing phase 2 RESTORE trial in which elritercept was generally well-tolerated and demonstrated potential efficacy for treating patients with myelofibrosis.
These results “demonstrate potential to address ineffective hematopoiesis and treat cytopenias associated with MF and ruxolitinib. Data also support potential for elritercept to reduce spleen size and improve symptoms,” Harrrison and coauthors wrote.
Results were presented at the 2024 European Hematology Association (EHA) Meeting in Madrid, Spain.
Transcript:
Hello, my name is Claire Harrison. I'm a hematologist working at [Guy’s and St. Thomas’ NHS Foundation Trust] in Central London. At the recent EHA 2024 meeting, I was very privileged to present an abstract concerning the efficacy of elritercept, or KER-050, for patients with myelofibrosis.
Oncology Learning Network: What is the significance of the data you presented on elritercept for the potential treatment of patients with myelofibrosis?
First, an introduction to elritercept which is an inhibitor of TGF-β superfamily ligands, in particular activin A. This agent potentially has the ability to restore various aspects of abnormalities that are present in our patients with myelofibrosis. The agent targets inflammation, it targets both early and late erythropoiesis, but also because inflammation is so central to the pathogenesis of myelofibrosis, potentially it may allow restoration of more normal hematopoiesis, restore the stem cell niche, and also various markers of inflammation and symptoms that patients have. I presented updated results from a study, which is recruiting 2 different cohorts of patients with myelofibrosis.
We have concluded dose escalation and continue to recruit patients into dose expansion. And the data that was presented was for 54 patients. The 2 cohorts of patients are either patients who are (Janus kinase) JAK inhibitor relapsed/refractory, say where the agent was given in monotherapy, or there was a second cohort for patients who had anemia transfusion dependence or were requiring transfusion in combination with ruxolitinib.
OLN: What do these results show?
In brief, the results are really intriguing and very complex. I want to give you the headlines, that basically we saw improvements in hemoglobin, reductions in requirements for transfusion without compromising platelet counts for patients. Patients' platelet counts generally stayed stable or increased. We also saw 3 patients who had significant spleen volume reductions (35%) and symptom improvements by varying amounts, but at least 1 patient had a 50% reduction in total symptom score.
These benefits were seen for patients across the dose expansions. So, 1 particular patient who was treated with monotherapy achieved transfusion independence, 35% spleen volume reduction, and symptom improvement at the lowest level, which was durable for over 100 weeks before dose escalation.
The predominant safety and tolerability issues related to nausea and some thrombocytopenia. Having said that, the monotherapy cohort in particular was very thrombocytopenic. There was 1 dose-limiting toxicity, which happily was an increase in hemoglobin by more than 2g/L meeting criteria for dose reduction.
So, this is an exciting agent. There was also data presented for this agent in [myelodysplastic syndromes] (MDS) with an ongoing partner study with the same dose, and also really interesting data with regard to iron stores, inflammation, and also anabolic effects of this drug, which I think could be a really exciting agent in the future for these chronic myeloid diseases. Thanks for listening.
Source:
Harrison C. Elritercept (Ker-050) demonstrated potential to treat myelofibrosis and mitigate ruxolitinib-associated cytopenias in the phase 2 restore trial. Presented at the 2024 European Hematology Association (EHA) Meeting. July 13–16, 2024; Madrid, Spain. Abstract S223
