Product Description
Iadademstat (ORY-1001) is a small oral molecule, which acts as a covalent and highly selective inhibitor of the epigenetic enzyme Lysine Specific Demethylase-1, LSD1 (KDM1A). Iadademstat does not only block the demethylating activity of LSD1, but also its scaffolding function. As the molecule binds covalently to the FAD cofactor in LSD1's catalytic center, it protrudes in a way that impairs the anchoring of LSD1 with GFI-1 (a phenomena denominated steric hindrance). This uncoupling produces a strong effect forcing undifferentiated blasts to differentiate. This reduces leukemic stem cell capacity and proliferation and induces leukemic blast differentiation, and may produce therapeutic benefit, as has been reported by Oryzon scientists. (Sourced from: https://www.oryzon.com/en/programs/iadademstat)
Mechanisms of Action: LSD1 Inhibitor
Novel Mechanism: No
Modality: Small Molecule
Route of Administration: Oral
FDA Designation: Orphan Drug - Acute Leukemia|Acute Myeloid Leukemia|Leukemia|Myeloid LeukemiaOrphan Drug - Lung Cancer|Small Cell Lung Cancer *
Approval Status: Not Approved
Approved Countries: None
Approved Indications: None
Known Adverse Events: None
Company: Hoffmann-La Roche
Company Location:
Company Founding Year: 1896
Additional Commercial Interests: None
Clinical Description
Countries in Clinic: Spain, United States
Active Clinical Trial Count: 5
Recent & Upcoming Milestones
Highest Development Phases
Phase 2: Small Cell Lung Cancer|Thrombocythemia, Essential
Phase 1: Acute Myeloid Leukemia
Trial ID |
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
Latest Trial Update Date |
Data Updated |
|---|---|---|---|---|---|---|---|---|
NCT07113691 |
TIARA | P1 |
Recruiting |
Small Cell Lung Cancer |
2030-01-01 |
50% |
2026-01-09 |
Primary Completion Date|Primary Endpoints|Start Date|Study Completion Date|Treatments|Trial Status |
NCT05546580 |
FRIDA | P1 |
Recruiting |
Acute Myeloid Leukemia |
2025-11-30 |
39% |
2024-07-16 |
Primary Endpoints |
2025-523864-19-00 |
CL06-ORY-1001 | P2 |
Not yet recruiting |
Thrombocythemia, Essential |
2100-01-01 |
|||
2018-000482-36 |
ALICE | P2 |
Active, not recruiting |
Unknown |
2020-10-25 |
2022-03-13 |
Treatments |
|
2018-000469-35 |
CLEPSIDRA | P2 |
Active, not recruiting |
Small Cell Lung Cancer |
2020-09-19 |
2022-03-13 |
Treatments |
Recent News Events
Date |
Type |
Title |
|---|---|---|
|
02/09/2026 |
News Article |
ORYZON Announces First Patient Dosed in an Investigator-Initiated Phase Ib Study of Iadademstat in Extensive Stage Small Cell Lung Cancer |
|
01/08/2026 |
News Article |
ORYZON to Participate in Upcoming Events in January |
|
12/09/2025 |
News Article |
ORYZON Presents Data for Iadademstat Combinations in AML at the American Society of Hematology (ASH) 67th Annual Meeting |
|
11/04/2025 |
News Article |
ORYZON Announces Positive Clinical Data of Iadademstat at ASH-2025 |
|
05/14/2024 |
PubMed |
Anticancer Drugs of Lysine Specific Histone Demethylase-1 (LSD1) Display Variable Inhibition on Nucleosome Substrates. |
|
01/25/2024 |
PubMed |
Combination Therapy and Dual-Target Inhibitors Based on LSD1: New Emerging Tools in Cancer Therapy. |
|
10/20/2023 |
PubMed |
Lysine-Specific Demethylase 1 (LSD1) Inhibitors: Peptides as an Emerging Class of Therapeutics. |