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iadademstat + venetoclax
- Development stage
- Unknown
- Lead developer
- Oryzon Genomics
- Modality
- Small Molecules
- Administration
- Oral
01
Overview
Iadademstat + venetoclax is an investigational drug combination being evaluated for the treatment of acute myeloid leukemia (AML). The combination consists of iadademstat (ORY-1001), an oral small-molecule inhibitor of lysine-specific demethylase 1 (LSD1/KDM1A) developed by Oryzon Genomics, and venetoclax, an approved oral small-molecule B-cell lymphoma 2 (BCL-2) inhibitor marketed by AbbVie and Genentech. In clinical trials, this combination is typically administered as a triplet regimen alongside the hypomethylating agent azacitidine. Iadademstat works by blocking the enzymatic activity of LSD1 and disrupting its interaction with the GFI-1 transcription factor, thereby promoting differentiation of leukemic blasts, while venetoclax induces apoptosis by selectively inhibiting BCL-2. The combination is being evaluated in Phase I clinical trials (such as ALICE-2 and an NCI-sponsored study) for newly diagnosed, treatment-naive AML in older or unfit patients.
- Other names
- iadademstat and venetoclaxiadademstat plus venetoclaxvenetoclax + iadademstatvenetoclax and iadademstatvenetoclax plus iadademstat
02
Targets
BCL-2 (BCL-2 family)DAT (Dopamine plasma membrane transport protein)GFI1 (Zinc finger protein Gfi-1)I2R (Imidazoline I2 receptor)KDM1A (LSD1)CACNA1C (Voltage-dependent L-type calcium channel subunit alpha-1C)NET (Norepinephrine Transporter)SMOX (Spermine oxidase)
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