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BXCL701 granted orphan drug designation by the US FDA for the treatment of AML

, Sep 9, 2019 , Expert Opinion

The United States (US) Food & Drug Administration (FDA) granted Orphan Drug Designation (ODD) to BXCL701 for the treatment of acute myeloid leukemia (AML).1 BXCL701 has already received ODD for the treatment of melanoma and pancreatic cancer.1

The company responsible for the development of the drug is planning to test BXCL701 as monotherapy, and as combination therapy, in specific patients with AML for whom there is a high unmet medical need.1

About BXCL7011
  • Orally-available systemic innate immunity activator:
    • Inhibits dipeptidyl dipeptidases DPP8 and DPP9
    • Blocks immune evasion via targeting of the fibroblast activation protein (FAP)
    • Exerts an immuno-stimulatory effect by initiating pro-inflammatory, programmed, lytic cell death (pyroptosis)
  • In a study published by Darren C. Johnson and colleagues in Nature Medicine, small-molecule inhibitors of the DPP8 and DPP9 were shown to:2
    • Cause pyroptosis in most human AML cell lines and primary AML samples, whilst leaving cells from other lineages unaffected
    • Prevent AML progression in mouse models
References
  1. Global newswire. BioXcel Therapeutics Receives FDA Orphan Drug Designation for BXCL701 for the Treatment of Acute Myeloid Leukemia (AML). https://www.globenewswire.com/news-release/2019/09/04/1910740/0/en/BioXcel-Therapeutics-Receives-FDA-Orphan-Drug-Designation-for-BXCL701-for-the-Treatment-of-Acute-Myeloid-Leukemia-AML.html [Accessed 2019 Sep 05]
  2. Johnson D.C. et al., DPP8/DPP9 inhibitor-induced pyroptosis for treatment of acute myeloid leukemia. Nature Medicine. 2019 Jul 02. DOI: 10.1038/s41591-018-0082-y
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