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

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