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iadademstat + azacitidine
- Development stage
- Phase 1
- Lead developer
- Oryzon Genomics
- Modality
- Small Molecules
- Administration
- Intravenous, Oral, Subcutaneous
01
Overview
Iadademstat (ORY-1001) plus azacitidine is an investigational combination regimen consisting of iadademstat, an oral, highly selective, covalent small molecule inhibitor of lysine-specific histone demethylase 1A (LSD1/KDM1A), and azacitidine, a standard-of-care hypomethylating agent and DNA methyltransferase inhibitor. Developed by Oryzon Genomics, this combination is designed to synergistically target epigenetic dysregulation in hematological malignancies. LSD1 inhibition by iadademstat promotes differentiation of leukemic stem cells and blasts, while azacitidine induces DNA hypomethylation and reactivates silenced tumor suppressor genes. The combination has been evaluated in clinical trials, including the Phase IIa ALICE trial, demonstrating high objective response rates and a manageable safety profile in elderly or unfit patients with newly diagnosed acute myeloid leukemia (AML). It is also under investigation for myelodysplastic syndrome (MDS).
- Other names
- ORY-1001 + azacitidineazacitidine + ORY-1001azacitidine + iadademstatazacitidine and iadademstatiadademstat and azacitidine
02
Targets
DNMT1 (DNA (cytosine-5)-methyltransferase 1)DNMT3A (DNA methyltransferase 3 alpha)DNMT3B (DNA (cytosine-5)-methyltransferase 3B)KDM1A (LSD1)
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