Imetelstat in intermediate-2 or high-risk myelofibrosis refractory to JAK inhibitor: IMpactMF phase III study design
John Mascarenhas, Claire Harrison, Jean‐Jacques Kiladjian, Rami S. Komrokji, Steffen Koschmieder, Alessandro M. Vannucchi, Tymara Berry, Denise Redding, Laurie Sherman, Souria Dougherty, Lixian Peng, Libo Sun, Fei Huang, Ying Wan, Faye Feller, Aleksandra Rizo, Srđan Verstovšek
Abstract
Imetelstat, a first-in-class telomerase inhibitor, demonstrated meaningful clinical benefit including a robust symptom response rate and potential overall survival benefit in IMbark, a phase II study in intermediate-2 or high-risk myelofibrosis (MF) patients who have relapsed after or are refractory to JAK inhibitors. We describe the rationale and design for the phase III trial, IMpactMF (NCT04576156), an open-label evaluation of imetelstat versus best available therapy, excluding JAK inhibitors, in MF patients refractory to JAK inhibitor. Imetelstat 9.4 mg/kg is administered as an intravenous infusion every 21 days. Primary objective is to assess overall survival. Secondary objectives include symptom and spleen responses, progression-free survival, clinical response assessment, bone marrow fibrosis reduction, safety and pharmacokinetics. Biomarker, cytogenetics and mutation analyses will be performed.