The U.S. Food and Drug Administration granted orphan-drug designation to SY-1425 (tamibarotene) for the treatment of patients with acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS) whose disease is characterized by abnormally high expression of the RARA or IRF8 genes. SY-1425 is an oral, first-in-class selective retinoic acid receptor alpha (RARα) agonist that targets super-enhancers that drive overexpression of those genes. Tamibarotene is licensed in Japan for treatment of acute promyelocytic leukemia.
SY-1425 is being evaluated as a single agent and in combination with azacitidine in an ongoing phase II study of patients with AML or MDS who express the biomarker. The study’s primary objective is to measure the overall response rate in AML and higher-risk MDS patients or transfusion independence in lower-risk MDS patients.
Source: Syros Pharmaceuticals press release, August 21, 2017.