Sotatercept (ACE-011) for Anemia of Myelofibrosis: A Phase 2 Study

Background Anemia is common in patients (pts) with myeloproliferative neoplasm (MPN)-associated myelofibrosis (MF). Furthermore, anemia is an on-target effect of therapeutic Janus kinase 2 (JAK2) inhibition, and may be the most frequent cause of ruxolitinib (rux) discontinuation (d/c) in clinical pr...

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Published in:Blood 2020-11, Vol.136 (Supplement 1), p.10-11
Main Authors: Bose, Prithviraj, Pemmaraju, Naveen, Masarova, Lucia, Bledsoe, Sharon D., Daver, Naval, Jabbour, Elias, Kadia, Tapan M., Estrov, Zeev E., Kornblau, Steven M., Andreeff, Michael, Cortes, Jorge E., Jain, Nitin, Borthakur, Gautam, Alvarado, Yesid, Huynh-Lu, Julie, Nguyen-Cao, Madeleine, Richie, Mary Ann, Dobbins, Mackenzie H., Zhou, Lingsha, Pierce, Sherry A., Wang, Xuemei, Pike, Allison M., Garcia-Manero, Guillermo, Kantarjian, Hagop M., Verstovsek, Srdan
Format: Article
Language:English
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Summary:Background Anemia is common in patients (pts) with myeloproliferative neoplasm (MPN)-associated myelofibrosis (MF). Furthermore, anemia is an on-target effect of therapeutic Janus kinase 2 (JAK2) inhibition, and may be the most frequent cause of ruxolitinib (rux) discontinuation (d/c) in clinical practice (Kuykendall, Ann Hematol 2018). Current therapies for anemia of MF (erythropoietin and analogs, danazol, IMiDs®) are unsatisfactory. Sotatercept (ACE-011) is a first-in-class, activin receptor type IIA ligand trap that may improve anemia by sequestering stromal transforming growth factor beta superfamily ligands that suppress terminal erythropoiesis (Iancu-Rubin, Exp Hematol 2013). Methods This is a phase 2, investigator-initiated, open-label, single institution study of sotatercept, administered subcutaneously every 3 weeks, in 2 cohorts of anemic pts (Hgb
ISSN:0006-4971
1528-0020
DOI:10.1182/blood-2020-140441