Table 1.

New drugs for lower risk patients.

New Active Principle	Mechanism of Action	Efficacy in Evaluable Patients	Clinical Trial
Imetelstat	Telomerase inhibitor	N = 38 R/R to ESAs 8-week TI rate of 37% for a median duration of 20 months	NCT02598661 phase II/III
Pexmetinib	p38/Tie2 inhibitor	N = 44 HI rate of 32%	NCT00916227 phase I
Galunisertib	TGF-β receptor type 1 kinase (ALK5) oral inhibitor	N = 41 HI rate of 24.4%	NCT02008318 phase II
Oral azacytidine (CC-486)	HMA DNA/RNA methyltransferases inhibitor	N = 216 RBC-TI rate of 31% with a median duration of 11.1 months	NCT01566695 phase III
Tomaralimab (OPN-305)	TLR-2 inhibitor	N = 22 TI for at least 2 cycles in 27% (major responders)	NCT02363491 phase I/II
Roxadustat	HIF-PH inhibitor	N = 24 TI rate of 37.5% for ≥56 consecutive days within the first 28 weeks	NCT03263091 phase III
Cedazuridine/decitabine (ASTX727)	Fixed-dose combination of the HMA decitabine and the novel cytidine deaminase inhibitor cedazuridine	N = 27 RBC-TI rate of 48%	NCT03502668 phase I/II

R/R, relapsed/refractory; ESAs, erythropoiesis stimulating agents; TI, transfusion independence; HI, hematologic improvement; TGF-β, transforming growth factor-beta; RBC, red blood cell; TLR-2, Toll-like receptor 2; HIF-PH, hypoxia-inducible factor prolyl hydroxylase; HMA, hypomethylating agent; DNA, deoxyribonucleic acid; RNA, ribonucleic acid.