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SLS009 granted orphan drug designation by the U.S. FDA

Oct 17, 2023
Learning objective: After reading this article, learners will be able to cite a new development in AML.

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On October 10, 2023, the U.S. Food and Drug Administration granted orphan drug designation to SLS009 (formerly GFH009), a novel and highly selective CDK9 inhibitor, for the treatment of patients with acute myeloid leukemia (AML).1

The orphan drug designation is based on results from a phase I trial that showed SLS009 to be associated with:

  • 77.3% bone marrow blast reduction.1
  • Durable complete remission with no measurable residual disease.1
  • 24 hours >IC90 peripheral blood concentrations following the first infusion, with IC90 concentrations resulting in up to 97% of cancer cells being killed.1
  • Decrease in MCL1 and MYC in 97% of patients.1
  • No dose-limiting toxicities, no higher grade non-hematologic toxicities of any kind, and some treatable hematologic toxicities.1

SLS009 is currently being investigated in an open-label, single-arm, multi-center phase IIa trial (NCT04588922) to assess the safety, tolerability, and efficacy of SLS009 at two dose levels (once weekly at 45mg and 60mg) in combination with azacitidine and venetoclax in patients with relapsed/refractory AML.1

  1. SELLAS Life Sciences Group. SELLAS receives FDA orphan drug designation for SLS009 for treatment of acute myeloid leukemia. Published Oct 10, 2023. Accessed Oct 12, 2023.

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