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RESTORE phase II results: Elritercept as monotherapy and in combination with ruxolitinib in MF
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Dysregulated signaling of TGF-β superfamily ligands, including activin A, contribute to BM dysfunction and BM fibrosis in MF, in which anemia is highly prevalent.1 Elritercept (KER-050) is an investigational drug that restores ineffective hematopoiesis by inhibiting activin A and select TGF-β superfamily ligands.1 Results from the phase II RESTORE trial (NCT05037760) evaluating the safety and efficacy of elritercept in patients with MF and high disease burden (n = 73; 29 received elritercept monotherapy, 44 received elritercept plus ruxolitinib) were presented by Claire Harrison at the 66th ASH Annual Meeting and Exposition.1 |
| Key learnings |
| The most frequent TEAEs were thrombocytopenia (in 20.5% of patients overall) and diarrhea (in 19.2% of patients overall). Grade ≥3 thrombocytopenia occurred in 16.4% of patients overall, and one patient experienced a DLT of ≥2.0 g/dL Hb increase with elritercept monotherapy. |
| Among non-TD6 patients (n = 29), increases in Hb were seen in both monotherapy and combination arms; with 51.7%, 20.7%, and 10.3% of patients showing increases of ≥1.0 g/dL, ≥1.5 g/dL, and ≥2.0 g/dL, respectively, within the first 24 weeks. |
| Among TD3 patients (n = 41) in both arms, 39% showed a ≥50% reduction in transfusion burden and 24% achieved TI within the first 24 weeks. Both arms showed stabilized or improved platelet counts, reductions in spleen size, and improved TSS. |
| Findings from the RESTORE trial show that elritercept was tolerated and achieved hematologic improvements in patients with MF, both as monotherapy and in combination with ruxolitinib. Part 2 of the trial is ongoing at an RP2D of elritercept 3.75 mg/kg. |
Abbreviations: ASH, American Society of Hematology; BM, bone marrow; DLT, dose-limiting toxicity; Hb, hemoglobin; MF, myelofibrosis; RP2D, recommended phase II dose; TD, transfusion dependent; TD3, receiving ≥3 units of red blood cell within 12 weeks prior to first dose; TD6, receiving ≥6 units of red blood cell within 12 weeks prior to first dose; TEAE, treatment-emergent adverse event; TI, transfusion independence; TSS, total symptom score; TGF-β, transforming growth factor beta.
- Harrison C. Oral abstract #997. Hematological improvement and other clinical benefits of elritercept as monotherapy and in combination with ruxolitinib in participants with myelofibrosis from the ongoing phase 2 Restore trial. 66th American Society of Hematology (ASH) Annual Meeting and Exposition; Dec 9, 2024; San Diego, US.
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