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    DRUG CLASS:

    LSD1 inhibitor

    Related drugs:
    7d
    CL12-ORY-2001: A PHASE 1, OPEN-LABEL STUDY TO ASSESS THE ABSORPTION, DISTRIBUTION, METABOLISM, AND EXCRETION, INCLUDING THE MASS BALANCE, OF A SINGLE ORAL DOSE OF [14C]-VAFIDEMSTAT IN HEALTHY MALE PARTICIPANTS (2025-524832-20-00)
    P1, N=8, Not yet recruiting, Oryzon Genomics S.A.
    New P1 trial
    11d
    CL09-ORY-2001: A Clinical trial to evaluate the efficacy of vafidemstat in negative symptoms and cognitive impairment associated with schizophrenia. (EVOLUTION study) (2023-507372-42-00)
    P1/2, N=239, Recruiting, Oryzon Genomics S.A. | N=84 --> 239
    Enrollment change
    11d
    The lysine-specific demethylase 1 (LSD1) inhibitor bomedemstat in myelofibrosis: results from a phase 1/2 study. (PubMed, Blood Adv)
    Bomedemstat has manageable safety and clinical activity in participants with MF in need of an alternative therapy. This trial was registered at www.cinicaltrials.gov as #NCT03136185.
    P1/2 data • Journal
    |
    JAK2 (Janus kinase 2) • CALR (Calreticulin)
    |
    bomedemstat (MK-3543)
    1m
    Dose Determining Study of EXS74539 (REC-4539) in Participants With Select Solid Tumors (clinicaltrials.gov)
    P1, N=40, Recruiting, Exscientia AI Ltd., a wholly owned subsidiary of Recursion Pharmaceuticals, Inc.
    New P1 trial
    1m
    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)
    1m
    NCI-2020-14163: Seclidemstat and Azacitidine for the Treatment of Myelodysplastic Syndrome or Chronic Myelomonocytic Leukemia (clinicaltrials.gov)
    P1/2, N=24, Active, not recruiting, M.D. Anderson Cancer Center | Recruiting --> Active, not recruiting | N=44 --> 24
    Enrollment closed • Enrollment change
    |
    TP53 (Tumor protein P53) • NRAS (Neuroblastoma RAS viral oncogene homolog) • RUNX1 (RUNX Family Transcription Factor 1) • ASXL1 (ASXL Transcriptional Regulator 1) • SETBP1 (SET Binding Protein 1)
    |
    TP53 mutation • NRAS mutation • ASXL1 mutation
    |
    azacitidine • seclidemstat (SP2577)
    1m
    Uncovering BAP1 deubiquitination landscape enhances mechanism elucidation and therapeutic precision for BAP1-deficient pancancers. (PubMed, Sci Transl Med)
    Combined inhibition of LSD1 and PARP1, using SP2509/SP2577 and olaparib, respectively, synergistically hinders NER, induces apoptosis, reduces tumor burden, and prolongs the survival of multiple BAP1-deficient pancancer in vitro models and in vivo xenografts. In conclusion, our results provide a deubiquitination landscape of BAP1; elucidate the mechanisms of action of BAP1, LSD1, and PARP1 in pancancers; and describe a promising combination therapeutic strategy applicable across multiple cancers with BAP1 mutations.
    Journal • BRCA Biomarker • PARP Biomarker • Pan tumor
    |
    BAP1 (BRCA1 Associated Protein 1) • DDB1 (Damage Specific DNA Binding Protein 1) • RAD23B (RAD23 Homolog B)
    |
    BRCA1 mutation
    |
    Lynparza (olaparib) • seclidemstat (SP2577) • SP-2509
    2ms
    LSD1 inhibitor, TAS1440, disrupts INSM1-LSD1 complex activating tumor-suppressive pathways via transcriptional reprogramming in neuroendocrine SCLC. (PubMed, Nat Commun)
    Loss of LSD1 enzymatic activity or INSM1 knockout abrogated TAS1440 effects, defining its mode of action and chemoresistance. These findings support TAS1440 as a next-generation epigenetic therapy candidate for INSM1-high SCLC-A.
    Journal
    |
    TGFB1 (Transforming Growth Factor Beta 1) • ASCL1 (Achaete-Scute Family BHLH Transcription Factor 1) • SMAD2 (SMAD Family Member 2)
    2ms
    Phase 2 study of the lysine-specific demethylase 1 (LSD1) inhibitor bomedemstat for essential thrombocythemia. (PubMed, Blood Adv)
    Bomedemstat had clinically relevant activity and manageable safety in participants with ET. Registration: NCT04254978 (Study of Bomedemstat in Participants With Essential Thrombocythemia [IMG-7289-CTP-201/MK-3543-003]).
    P2 data • Journal
    |
    JAK2 (Janus kinase 2) • CALR (Calreticulin)
    |
    bomedemstat (MK-3543)
    2ms
    MK-3453-023: A Study of Bomedemstat (MK-3543) in Participants With Mild or Moderate Hepatic Impairment (MK-3543-023) (clinicaltrials.gov)
    P1, N=9, Completed, Merck Sharp & Dohme LLC | Active, not recruiting --> Completed | N=24 --> 9
    Trial completion • Enrollment change
    |
    bomedemstat (MK-3543)
    3ms
    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
    3ms
    Bomedemstat (IMG-7289) in Combination With Momelotinib in Patients With Myelofibrosis (clinicaltrials.gov)
    P2, N=40, Not yet recruiting, United Lincolnshire Hospitals NHS Trust
    New P2 trial
    |
    Ojjaara (momelotinib) • bomedemstat (MK-3543)