A Study Comparing Imetelstat Versus Best Available Therapy for the Treatment of Intermediate-2 or High-risk Myelofibrosis (MF) Who Have Not Responded to Janus Kinase (JAK)-Inhibitor Treatment
Key claim: This phase III trial evaluates whether imetelstat improves overall survival compared to best available therapy in intermediate-2 or high-risk myelofibrosis patients who are relapsed or refractory to JAK inhibitor treatment.
Abstract
The purpose of the study is to evaluate the overall survival of participants treated with imetelstat compared to best available therapy with intermediate-2 or high-risk Myelofibrosis (MF) who are relapsed/refractory (R/R) to Janus Kinase (JAK)-Inhibitor treatment.
Why this matters
JAK-inhibitor-refractory intermediate-2/high-risk myelofibrosis remains the highest-unmet-need PMF subpopulation, where survival is dismal and no agent has yet demonstrated an OS benefit in a randomized trial. Imetelstat’s telomerase-inhibition mechanism is orthogonal to the dense JAK/BET/XPO1/PI3Kδ/CALR landscape accumulating on the PMF page, and an OS-powered Phase III readout (versus the more common SVR35/TSS50 endpoints) would set a new prognostic benchmark for the post-JAKi PMF natural history.