Myelodysplastic Syndrome

The phase 2 KER050-MD-201 trial investigates elritercept, an activin receptor ligand trap, for treating lower-risk myelodysplastic neoplasms (LR-MDS) with anaemia. The study, as of September 2023, involved 79 participants, evaluating safety, tolerability, and response to treatment. Most participants experienced mild-to-moderate adverse events, with dyspnea and diarrhoea being most common. Of those evaluated for at least 24 weeks (N=60), half achieved haematological improvement or transfusion independence, particularly those with lower erythropoietin levels. Responses were durable, with 72% maintaining independence beyond 24 weeks. Elritercept shows promise for addressing LR-MDS and improving quality of life. Further updates will provide long-term data

The IMerge Phase 3 study (NCT02598661) is a phase 2/3 global study investigating imetelstat, a first-in-class telomerase inhibitor, for patients with red blood cell transfusion-dependent non-del(5q) lower risk-myelodysplastic syndromes (LR-MDS) relapsed/refractory (R/R) to erythropoiesis-stimulating agents (ESAs). We were joined by Dr Rami Komrokji (Moffitt Cancer Center, Tampa, FL, USA) to discuss the unmet needs in […]