Oryzon's Iadademstat Shows Strong Efficacy in AML Trials, Setting Stage for Registrational Study

Event summary

  • Oryzon presented updated clinical data for iadademstat in AML at EHA 2026, showing 100% ORR and 89% CRc in the ALICE-2 trial.
  • All patients with TP53 or RAS pathway mutations achieved complete response in the ALICE-2 trial.
  • FRIDA trial reported a 67% CRc rate in FLT3-mutated refractory-relapsed AML patients.
  • Oryzon plans to report final ALICE-2 data by year-end and advance toward a potential registrational study in 2027.

The big picture

Oryzon's positive clinical data for iadademstat in AML reinforces the potential of LSD1 inhibition as a novel triplet strategy, particularly for patients without targetable mutations. The results position Oryzon to compete in the AML space, where existing treatments like azacitidine plus venetoclax leave a significant portion of patients unresponsive. The company's focus on adverse-risk populations could carve out a niche in a market increasingly driven by personalized medicine.

What we're watching

Regulatory Pathway
Whether the favorable safety and efficacy data will support a successful registrational study and eventual approval for iadademstat in AML.
Clinical Development
The pace at which Oryzon can enroll and complete the ALICE-2 trial, and initiate subsequent studies in adverse-risk AML populations.
Competitive Positioning
How iadademstat's performance in genomically defined adverse-risk subgroups will differentiate it from existing AML treatments.