Phase 2 Study of the ALK5 Inhibitor Galunisertib in Very Low-, Low-, and Intermediate-Risk Myelodysplastic Syndromes

Santini, Valeria; Valcárcel, David; Platzbecker, Uwe; Komrokji, Rami S; Cleverly, Ann; Lahn, Michael M; Janssen, Jan; Zhao, Yumin; Chiang, Alan; Giagounidis, Aristoteles; Guba, Susan C; Sridharan, Ashwin; Gueorguieva, Ivelina; Girvan, Alicia; Da Silva Ferreira, Mariana; Bhagat, Tushar D; Pradhan, Kith; Steidl, Ulrich; Will, Britta; Verma, Amit

doi:10.1158/1078-0432.CCR-19-1338

Over-activation of TGF-b signaling is observed in myelodysplastic syndromes and is associated with dysplastic hematopoietic differentiation. Galunisertib, a first-in-class oral inhibitor of the TGF-b receptor type 1 kinase (ALK5) has shown effectiveness in preclinical models of myelodysplastic syndromes (MDS) and acceptable toxicity in phase 1 studies of solid malignancies.

Phase 2 Study of the ALK5 Inhibitor Galunisertib in Very Low-, Low-, and Intermediate-Risk Myelodysplastic Syndromes / Santini, V., Valcárcel, D., Platzbecker, U., Komrokji, R.S., Cleverly, A., Lahn, M.M., Janssen, J., Zhao, Y., Chiang, A., Giagounidis, A., Guba, S.C., Sridharan, A., Gueorguieva, I., Girvan, A., da Silva Ferreira, M., Bhagat, T.D., Pradhan, K., Steidl, U., Will, B., Verma, A.. - In: CLINICAL CANCER RESEARCH. - ISSN 1078-0432. - STAMPA. - 25:(2019), pp. 6976-6985. [10.1158/1078-0432.CCR-19-1338]