RESTORE phase II updated results: Elritercept in MF

Updated results from the phase II RESTORE trial (NCT05037760), investigating elritercept as monotherapy or in combination with ruxolitinib in patients with myelofibrosis (MF) and anemia, were presented by Ciro Rinaldi at the 67th American Society of Hematology (ASH) Annual Meeting and Exposition, December 6–9, 2025, Orlando, US. Outcomes of 38 patients receiving elritercept + ruxolitinib were reported (n = 23 transfusion-dependent [TD], n = 15 non-transfusion-dependent [NTD]). Analyses included treatment effects on hematologic parameters, spleen size, symptoms, fatigue, and gene mutations. The MPN Hub previously reported the primary results from this trial.

Key data: The most common treatment-emergent adverse events (TEAEs) were thrombocytopenia (28.9%; 10.5% Grade 3), diarrhea (23.8%, all Grade 1–2), fatigue (23.7%; 5.3% Grade 3), and anemia (23.7%; 7.9% Grade 3). At 36 weeks, 43.5% of TD patients achieved red blood cell (RBC) transfusion independence ≥12 weeks and 65.2% achieved ≥50% reduction in RBC transfusions. In NTD patients, 35.7% achieved mean hemoglobin increases ≥1.5 g/dL. Platelet improvements were observed in 39.7% of patients in the pharmacodynamic population with evaluable platelet values (N = 58). In patients with baseline spleen volume ≥450 cm³, 20.8% achieved spleen volume reduction ≥25%. Symptoms improved in 88.9% of evaluable patients. 

Key learning: Elritercept as an add-on treatment demonstrated clinically meaningful improvements in anemia, thrombocytopenia, spleen volume, and symptoms, supporting the continued evaluation of elritercept in patients with MF and anemia receiving ruxolitinib.