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    DRUG:

    iadademstat (ORY-1001)

    i
    Other names: ORY-1001, RG 6016, RO7051790, RO-7051790, RG6016, RO 7051790, ORY1001, ORY 1001, RG-6016
    Company:
    Oryzon
    Drug class:
    LSD1 inhibitor
    Related drugs:
    1m
    ALICE: Iadademstat in Combination With Azacitidine and Venetoclax in Treating Newly Diagnosed Acute Myeloid Leukemia (clinicaltrials.gov)
    P1, N=30, Recruiting, OHSU Knight Cancer Institute | Trial completion date: May 2026 --> May 2028 | Trial primary completion date: Mar 2026 --> Mar 2027
    Trial completion date • Trial primary completion date
    |
    ABL1 (ABL proto-oncogene 1)
    |
    ABL1 fusion
    |
    Venclexta (venetoclax) • azacitidine • iadademstat (ORY-1001)
    2ms
    A Randomized Study of ASTX727 With or Without Iadademstat in Advanced Myeloproliferative Neoplasms (MPNs) (clinicaltrials.gov)
    P2, N=62, Suspended, National Cancer Institute (NCI) | Recruiting --> Suspended
    Trial suspension
    |
    Inqovi (decitabine/cedazuridine) • iadademstat (ORY-1001)
    4ms
    Iadademstat With Hypomethylating Agent in Patients With Myelodysplastic Syndrome (clinicaltrials.gov)
    P1, N=12, Recruiting, Medical College of Wisconsin | Trial completion date: Oct 2027 --> Dec 2027 | Trial primary completion date: Oct 2026 --> Dec 2026
    Trial completion date • Trial primary completion date
    |
    iadademstat (ORY-1001)
    6ms
    CL06-ORY-1001: Phase 2 Study of iadademstat for the treatment of patients with essential thrombocythemia resistant/intolerant to hydroxyurea. (2025-523864-19-00)
    P1/2, N=36, Not yet recruiting, Oryzon Genomics S.A.
    New P1/2 trial
    |
    JAK2 (Janus kinase 2)
    |
    iadademstat (ORY-1001) • hydroxyurea
    7ms
    TIARA: Iadademstat + SBRT With Atezo in ES-SCLC (clinicaltrials.gov)
    P1, N=15, Recruiting, Yale University | Not yet recruiting --> Recruiting
    Enrollment open
    |
    Tecentriq (atezolizumab) • iadademstat (ORY-1001)
    9ms
    Co-targeting menin and LSD1 dismantles oncogenic programs and restores differentiation in MLL-rearranged AML. (PubMed, bioRxiv)
    This uncovered consistent synergy between menin and lysine-specific demethylase 1 (LSD1) inhibition, including with the clinical agent iadademstat...In vivo, the combination produced potent antileukemic effects in both MOLM-13 and MLL-r patient-derived xenografts, markedly reducing leukemic burden and extending survival without overt toxicity. These findings identify LSD1 as a critical cofactor of the menin-MLL-LEDGF axis and establish concurrent menin and LSD1 inhibition as a mechanistically informed combinatorial therapeutic approach in MLL-r AML.
    Journal
    |
    FLT3 (Fms-related tyrosine kinase 3) • KMT2A (Lysine Methyltransferase 2A) • CDK6 (Cyclin-dependent kinase 6) • HOXA9 (Homeobox A9) • PBX3 (PBX Homeobox 3)
    |
    MLL rearrangement
    |
    iadademstat (ORY-1001)
    11ms
    RNA processing kinase inhibitors and epigenetic inhibitors in combination with oncology drugs or investigational agents in multi-cell type patient-derived tumor cell line spheroids. (PubMed, Cancer Chemother Pharmacol)
    These findings may provide guidance for development of clinical trial combination regimens including cirtuvivint, CC-671 or iadademstat. Full data sets are available on PubChem.
    Preclinical • Journal • IO biomarker
    |
    KRAS (KRAS proto-oncogene GTPase)
    |
    KRAS mutation • KRAS G12D
    |
    MRTX1133 • eltanexor (KPT-8602) • iadademstat (ORY-1001) • cirtuvivint (SM08502)
    11ms
    Dual targeting of CDK6 and LSD1 is synergistic and overcomes differentiation blockade in AML. (PubMed, EMBO Mol Med)
    While many AML samples exhibit only modest responses to LSD1 inhibition, co-targeting CDK6 restores the expected transcription response associated with LSD1 inhibition. Given the availability of clinical-grade CDK6 and LSD1 inhibitors, this combination holds significant potential for implementation in clinical settings through drug repositioning.
    Journal
    |
    CDK6 (Cyclin-dependent kinase 6)
    |
    Ibrance (palbociclib) • iadademstat (ORY-1001)
    12ms
    Curing the Incurable: TP53 Mutated Myeloid Neoplasms. (PubMed, Clin Lymphoma Myeloma Leuk)
    The differential prognosis of the mutation in various chromosomal and VAF settings will be explored. Lastly, we will outline therapeutic options, promising treatments on the horizon, and whether allogeneic stem cell transplant is curative.
    Review • Journal
    |
    TP53 (Tumor protein P53)
    |
    TP53 mutation
    |
    flotetuzumab (MGD006) • iadademstat (ORY-1001)
    12ms
    TIARA: Iadademstat + SBRT With Atezo in ES-SCLC (clinicaltrials.gov)
    P1, N=15, Not yet recruiting, Yale University
    New P1 trial
    |
    Tecentriq (atezolizumab) • iadademstat (ORY-1001)
    1year
    Iadademstat in Combination With Paclitaxel in Relapsed/Refractory SCLC and Extrapulmonary High Grade NET (clinicaltrials.gov)
    P2, N=20, Terminated, Fox Chase Cancer Center | N=42 --> 20 | Trial completion date: Aug 2026 --> Jul 2025 | Recruiting --> Terminated; Closed to accrual due to low probability of successful outcomes
    Enrollment change • Trial completion date • Trial termination
    |
    paclitaxel • iadademstat (ORY-1001)