Orphan drug designation received for acute myeloid leukaemia treatment

The US Food and Drug Administration (FDA) has granted orphan drug designation to SY-1425, which is an oral first-in-class selective retinoic acid receptor alpha (RARα) agonist for the treatment of acute myeloid leukaemia (AML) from Syros Pharmaceuticals.

This therapy is currently undergoing evaluation in a Phase II clinical trial including genomically defined subsets of patients with AML and myelodysplastic syndrome (MDS). The ongoing trial is assessing the safety and efficacy of SY-1425 as a single agent in four AML and MDS patient populations, as well as in combination with azacitidine, a standard-of-care therapy, in newly diagnosed AML patients who are not suitable candidates for standard chemotherapy.

“Treatment of AML remains a significant unmet medical need, with many patients lacking adequate therapeutic options,” said Dr David A. Roth, Syros’ chief medical officer. “We believe that SY-1425 may provide a meaningful benefit for subsets of AML patients whose disease is driven by abnormally high expression of the RARA or IRF8 genes. Receiving orphan drug designation is an important regulatory milestone in the development of SY-1425. We’re pleased with the continued progress of the ongoing Phase II clinical trial, and we look forward to presenting initial clinical data in the fourth quarter of this year.”