Early Results from a Biomarker-Directed Phase 2 Trial of Sy-1425 in Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome (MDS) Demonstrate DHRS3 Induction and Myeloid Differentiation Following Sy-1425 Treatment

Introduction: A novel patient (pt) subset defined by RARA pathway activation was identified by super-enhancers (SEs) at the RARAand IRF8gene loci in AML and MDS. These SEs correlated with mRNA expression of each gene, as well as preclinical tumor cell differentiation and in vivoefficacy response to SY-1425 (tamibarotene), a potent and selective oral RARα agonist approved for treatment of relapsed/refractory (r/r) APL in Japan. This discovery formed the basis of a biomarker-directed phase 2 clinical study of SY-1425 as monotherapy and in combination with azacitidine for AML and MDS pts (NCT02807558).