Seclidemstat

Seclidemstat is an orally available, reversible, noncompetitive small molecule inhibitor of lysine-specific demethylase 1 (LSD1, also known as KDM1A). By inhibiting LSD1’s enzymatic and scaffolding functions, seclidemstat increases methylation at histone H3K4 and H3K9 sites. This leads to enhanced expression of tumor suppressor genes and reduced transcription of tumor-promoting genes. Seclidemstat has demonstrated antineoplastic activity in preclinical models by reprogramming cancer cell differentiation, reducing tumor burden, and prolonging survival. It is being developed primarily for the treatment of Ewing sarcoma and hematologic malignancies such as myelodysplastic syndrome (MDS) and chronic myelomonocytic leukemia (CMML), with ongoing clinical trials evaluating its efficacy both as a single agent and in combination with other therapies[1][2][4][5][7]. Seclidemstat also blocks the transcriptional function of multiple FET fusion proteins implicated in various sarcomas[6]. The drug has shown manageable safety profiles in early-phase clinical trials for relapsed/refractory Ewing sarcoma[7], with additional studies ongoing for MDS/CMML[5].

Other names

1423715-37-0