Oryzon Advances Iadademstat into Phase II Trial for Essential Thrombocythemia

Event summary

  • Oryzon initiated patient enrollment in the IDEAL Phase II trial of iadademstat for essential thrombocythemia (ET) on June 2, 2026.
  • The multicenter, single-arm study will evaluate safety, tolerability, and clinical activity in ET patients resistant or intolerant to hydroxyurea.
  • Iadademstat will be administered for up to 24 weeks, with a potential 24-week extension for responding patients.
  • Updated ALICE-2 study results for first-line AML will be presented at EHA 2026, showing 100% overall response rate (ORR).
  • FRIDA study in relapsed/refractory FLT3-mutated AML demonstrated a 67% composite complete remission (CRc) rate.

The big picture

Oryzon's expansion of iadademstat into essential thrombocythemia reflects its strategy to leverage LSD1 inhibition across multiple hematologic indications. The Phase II trial follows promising results in AML, positioning iadademstat as a potential differentiated treatment in chronic myeloproliferative diseases. The company's broad clinical pipeline underscores its focus on epigenetics-driven therapies in both oncology and non-malignant hematology.

What we're watching

Clinical Efficacy
How iadademstat's performance in ET compares to its results in AML and other hematologic malignancies.
Regulatory Pathway
Whether the IDEAL trial results could support a broader indication for iadademstat in myeloproliferative disorders.
Pipeline Momentum
The pace at which Oryzon can advance its diverse clinical programs, including RESTORE for sickle cell disease.

Related topics

Oncology