General AML

The FDA grants SY-1425 Orphan Drug Designation for the treatment of AML

On 21st August 2017, the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to SY-1425 (tamibarotene) for the treatment of patients with Acute Myeloid Leukemia (AML).1

Highly specialized regions known as super-enhancers, associated with selective Retinoic Acid Receptor Alpha (RARα) and Monocyte Specific Factor (IRF8) genes, have been identified in blast cells of some patients with AML. These super-enhancers have been shown to drive high expression of RARα and IRF8, thus driving AML blasts to be in an immature, undifferentiated, and proliferative state. SY-1425 is an oral, first-in-class, selective RARα agonist. SY-1425 binds to RARα, which leads to reactivation of differentiation and prolongation of survival in preclinical models of AML with high RARα expression.2

Currently, SY-1425 is being evaluated in a biomarker directed phase II study (NCT02807558) aiming to assess its safety and efficacy alone or in combination with azacitidine in newly diagnosed or relapsed/refractory AML patients who are positive for either RARα or IRF8. The primary endpoint of the study is Overall Response Rate (ORR).

  1. BuisnessWire: Syros Receives FDA Orphan Drug Designation for SY-1425 for Treatment of AML. 2017 Aug 21. [Accessed 2017 Aug 21].
  2. McKeown M.R. et al. Super-Enhancer Analysis Defines Novel Epigenomic Subtypes of Non-APL AML Including an RARα Dependency Targetable by SY-1425, a Potent and Selective RARα Agonist. Cancer Discov. 2017 Jul 20. DOI: 10.1158/2159-8290.CD-17-0399. [Epub ahead of print].
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