Phase 2 study of the lysine-specific demethylase 1 inhibitor bomedemstat for essential thrombocythemia

Blood Advances 2026 Open access

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Authors: Harinder Gill, Francesca Palandri, David M. Ross, Joachim R Göthert, Tara Cochrane, Stephen Larsen, Anna B. Halpern, Jake Shortt, James M. Rossetti, James Liang, Monia Marchetti, Andrew J. Wilson
+10 more Andrew J. Innes, Merit Hanna, Nicola Vianelli, William Stevenson, Alessandro M. Vannucchi, Maria Kleppe, Jessica A. Flynn, Georges Natsoulis, Claire N. Harrison, Hugh Young Rienhoff
DOI: 10.1182/bloodadvances.2025017575
Cited by: 3
References: 24

Abstract

ABSTRACT: Novel treatments that can improve outcomes of essential thrombocythemia (ET) are needed. In a phase 2 trial, participants with ET who required cytoreduction and had inadequate response to, or were intolerant of, ≥1 standard therapy received bomedemstat at a starting dose of 0.6 mg/kg per day, titrated to achieve a target platelet count (200 × 109/L to 400 × 109/L). Primary end points were safety and response, defined as a platelet count of ≤400 × 109/L without new thromboembolic events. Of 73 who received bomedemstat, at 24 weeks, 49 of 64 evaluable participants (77%) achieved a response. Durable reductions in platelet count (≤400 × 109/L for ≥12 weeks) were observed in 52 of 72 participants (72%). Durable reduction in white blood cell count (

Fields of Study & Subjects

Myeloproliferative Neoplasms: Diagnosis and Treatment Platelet Disorders and Treatments Blood groups and transfusion

Publication details

Journal / source
Blood Advances
Publisher
American Society of Hematology
Volume
10
Issue
10
Pages
3494–3504
Publication date
27 February 2026
Language
EN