Rilzabrutinib, an Oral BTK Inhibitor, in Immune Thrombocytopenia
David J. Kuter, Merlin Efraim, Jiri Mayer, Marek Trněný, Vickie McDonald, Robert Bird, Thomas Regenbogen, Mamta Garg, Zane Kaplan, Nikolay Tzvetkov, Philip Y. Choi, A.J. Gerard Jansen, Milan Kostal, Ross Baker, Jaromir Gumulec, Eun-Ju Lee, Ilona Cunningham, Isaac Goncalves, Margaret Warner, Ralph Boccia, Terry Gernsheimer, Waleed Ghanima, Olga Bandman, Regan Burns, Ann Neale, Dolca Thomas, Puneet Arora, Beiyao Zheng, Nichola Cooper
Abstract
BACKGROUND: Rilzabrutinib, an oral, reversible covalent inhibitor of Bruton's tyrosine kinase, may increase platelet counts in patients with immune thrombocytopenia by means of dual mechanisms of action: decreased macrophage (Fcγ receptor)-mediated platelet destruction and reduced production of pathogenic autoantibodies. METHODS: per cubic millimeter without the use of rescue medication). RESULTS: per cubic millimeter was 65%. CONCLUSIONS: Rilzabrutinib was active and associated with only low-level toxic effects at all dose levels. The dose of 400 mg twice daily was identified as the dose for further testing. Overall, rilzabrutinib showed a rapid and durable clinical activity that improved with length of treatment. (Funded by Sanofi; ClinicalTrials.gov number, NCT03395210; EudraCT number, 2017-004012-19.).