Final Results of a Phase 2 Study of Sotatercept (ACE-011) for Anemia of MPN-Associated Myelofibrosis

• Published in: Blood 2021-11, Vol.138 (Supplement 1), p.144-144
• Main Authors: Bose, Prithviraj, Masarova, Lucia, Pemmaraju, Naveen, Bledsoe, Sharon D., Daver, Naval, Jabbour, Elias J., 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 M., Richie, Mary Ann, Dobbins, Mackenzie H., McCrackin, Selene Andrea, Zhou, Lingsha, Pierce, Sherry A., Wang, Xuemei, Pike, Allison M., Garcia-Manero, Guillermo, Kantarjian, Hagop, Verstovsek, Srdan
• Format: Article
• Language: English
• ISSN: 0006-4971; 1528-0020
• DOI: 10.1182/blood-2021-150908

▪ 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 is a 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