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    DRUG CLASS:

    RNA polymerase 1 inhibitor

    Related drugs:
    12d
    CX-5461-04: Study of CX-5461 in Patients With Solid Tumours and BRCA1/2, PALB2 or Homologous Recombination Deficiency (HRD) Mutation (clinicaltrials.gov)
    P1, N=52, Recruiting, Senhwa Biosciences, Inc. | Trial completion date: Dec 2026 --> Mar 2027 | Trial primary completion date: Dec 2025 --> Mar 2027
    Trial completion date • Trial primary completion date
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    BRCA1 (Breast cancer 1, early onset) • BRCA2 (Breast cancer 2, early onset) • CCNE1 (Cyclin E1) • KMT2D (Lysine Methyltransferase 2D) • CDK12 (Cyclin dependent kinase 12) • KMT2C (Lysine Methyltransferase 2C) • SLFN11 (Schlafen Family Member 11) • CHEK2 (Checkpoint kinase 2) • CREBBP (CREB binding protein) • RAD51 (RAD51 Homolog A) • FANCA (FA Complementation Group A) • RAD51B (RAD51 Paralog B) • BRIP1 (BRCA1 Interacting Protein C-terminal Helicase 1) • RAD51C (RAD51 paralog C) • RAD50 (RAD50 Double Strand Break Repair Protein) • RAD51D (RAD51 paralog D) • CHEK1 (Checkpoint kinase 1) • BARD1 (BRCA1 Associated RING Domain 1) • NBN (Nibrin Nijmegen Breakage Syndrome 1 (Nibrin)) • RAD54L (DNA Repair And Recombination Protein RAD54) • FANCF (FA complementation group F) • FANCL (FA Complementation Group L) • RBBP8 (RB Binding Protein 8, Endonuclease) • XRCC2 (X-Ray Repair Cross Complementing 2) • ERCC4 (ERCC Excision Repair 4, Endonuclease Catalytic Subunit) • FANCI (FA Complementation Group I) • FANCM (FA Complementation Group M) • MAD2L2 (Mitotic Arrest Deficient 2 Like 2) • CDC25C (Cell Division Cycle 25C) • FANCD2 (FA Complementation Group D2) • FANCE (FA Complementation Group E) • FANCG (FA Complementation Group G) • MUS81 (MUS81 Structure-Specific Endonuclease Subunit) • CDC25A (Cell Division Cycle 25A) • FANCB (FA Complementation Group B) • FANCC (FA Complementation Group C) • GNRP (Ras-Specific Guanine Nucleotide-Releasing Factor 1) • RAD17 (RAD17 Checkpoint Clamp Loader Component) • SLX4 (SLX4 Structure-Specific Endonuclease Subunit)
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    BRCA2 mutation • BRCA1 mutation • PALB2 mutation
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    pidnarulex (CX-5461)
    27d
    Testing the Effectiveness of the Anti-cancer Drug Pidnarulex (CX-5461), in Combination With Another Anti-cancer Drug Cemiplimab (REGN2810), in Treating Refractory Microsatellite Stable Colorectal Cancer (clinicaltrials.gov)
    P1/2, N=86, Recruiting, National Cancer Institute (NCI) | Not yet recruiting --> Recruiting | Initiation date: Mar 2026 --> Dec 2026
    Enrollment open • Trial initiation date
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    MSI (Microsatellite instability) • MYC (V-myc avian myelocytomatosis viral oncogene homolog) • FBXW7 (F-Box And WD Repeat Domain Containing 7)
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    Libtayo (cemiplimab-rwlc) • pidnarulex (CX-5461)
    3ms
    RNA polymerase I inhibitor CX-5461 suppresses cervical cancer cell growth by inducing DNA damage and mitotic catastrophe and enhances cisplatin sensitivity. (PubMed, Biochem Pharmacol)
    When combined with cisplatin, CX-5461 enhances the sensitivity of cervical cancer cells to this chemotherapeutic agent. In conclusion, CX-5461 demonstrates potential therapeutic value for cervical cancer, particularly as a new strategy for patients with primary or platinum-resistant disease.
    Journal
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    CDK1 (Cyclin-dependent kinase 1) • CCNB1 (Cyclin B1)
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    cisplatin • pidnarulex (CX-5461)
    3ms
    TFEB confers resistance against the chemotherapeutic agent CX-5461. (PubMed, Autophagy Rep)
    Interfering with TFEB improved the sensitivity of cancer cells to both CX-5461 and gemcitabine. Our findings suggest that TFEB provides broad protection against the stress caused by chemotherapeutic drugs, representing a promising target for intercepting chemoresistance and improving the efficacy of anticancer agents.
    Journal
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    TFEB (Transcription Factor EB 2)
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    gemcitabine • pidnarulex (CX-5461)
    3ms
    Testing the Safety of Anti-Cancer Drug, CX-5461 (Pidnarulex), in Treating Lymphoma With Specific Changes in the MYC Gene (clinicaltrials.gov)
    P1/2, N=50, Recruiting, National Cancer Institute (NCI) | Not yet recruiting --> Recruiting | Trial completion date: Sep 2027 --> Jan 2030 | Initiation date: Mar 2026 --> Oct 2026 | Trial primary completion date: Sep 2027 --> Jan 2030
    Enrollment open • Trial completion date • Trial initiation date • Trial primary completion date
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    BCL2 (B-cell CLL/lymphoma 2) • BCL6 (B-cell CLL/lymphoma 6)
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    pidnarulex (CX-5461)
    4ms
    Testing the Safety of the Combination of Anti-Cancer Drugs CX-5461 (Pidnarulex) and Trastuzumab Deruxtecan (T-DXd) for Human Epidermal Growth Factor Receptor 2 (HER2)-Positive Solid Tumors and Breast Cancer (clinicaltrials.gov)
    P1, N=36, Recruiting, National Cancer Institute (NCI) | Not yet recruiting --> Recruiting | Initiation date: Mar 2026 --> Oct 2026
    Enrollment open • Trial initiation date
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    HER-2 (Human epidermal growth factor receptor 2) • ER (Estrogen receptor) • PGR (Progesterone receptor)
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    HER-2 positive
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    Enhertu (fam-trastuzumab deruxtecan-nxki) • pidnarulex (CX-5461)
    4ms
    Testing the Safety of the Combination of Anti-Cancer Drugs CX-5461 (Pidnarulex) and Trastuzumab Deruxtecan (T-DXd) for Human Epidermal Growth Factor Receptor 2 (HER2)-Positive Solid Tumors and Breast Cancer (clinicaltrials.gov)
    P1, N=36, Not yet recruiting, National Cancer Institute (NCI) | Trial completion date: Mar 2027 --> Jan 2028 | Trial primary completion date: Mar 2027 --> Jan 2028
    Trial completion date • Trial primary completion date
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    HER-2 (Human epidermal growth factor receptor 2) • ER (Estrogen receptor) • PGR (Progesterone receptor)
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    HER-2 positive
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    Enhertu (fam-trastuzumab deruxtecan-nxki) • pidnarulex (CX-5461)
    5ms
    Testing the Safety of Anti-Cancer Drug, CX-5461 (Pidnarulex), in Treating Lymphoma With Specific Changes in the MYC Gene (clinicaltrials.gov)
    P1/2, N=50, Not yet recruiting, National Cancer Institute (NCI) | Initiation date: Dec 2025 --> Mar 2026
    Trial initiation date
    |
    BCL2 (B-cell CLL/lymphoma 2)
    |
    pidnarulex (CX-5461)
    5ms
    Testing the Effectiveness of the Anti-cancer Drug Pidnarulex (CX-5461) in Combination With Another Anti-cancer Drug Cemiplimab (REGN2810), in Treating Refractory Microsatellite Stable Colorectal Cancer (clinicaltrials.gov)
    P1/2, N=86, Not yet recruiting, National Cancer Institute (NCI) | Initiation date: Dec 2025 --> Mar 2026
    Trial initiation date
    |
    Libtayo (cemiplimab-rwlc) • pidnarulex (CX-5461)
    5ms
    Testing the Safety of the Combination of Anti-Cancer Drugs CX-5461 (Pidnarulex) and Trastuzumab Deruxtecan (T-DXd) for Human Epidermal Growth Factor Receptor 2 (HER2)-Positive Solid Tumors and Breast Cancer (clinicaltrials.gov)
    P1, N=36, Not yet recruiting, National Cancer Institute (NCI) | Initiation date: Dec 2025 --> Mar 2026
    Trial initiation date
    |
    Enhertu (fam-trastuzumab deruxtecan-nxki) • pidnarulex (CX-5461)
    6ms
    Reply to: The chemotherapeutic drug CX-5461 is a potent mutagen in cultured human cells. (PubMed, medRxiv)
    This is particularly important given that several clinical trials, including a Phase Ib study of CX-5461 in patients with solid tumours [ NCT04890613 ] and a National Cancer Institute sponsored Phase I trial [ NCT06606990 ] are currently enrolling patients. These ongoing and planned clinical efforts highlight both the therapeutic promise of CX-5461 and the importance of evaluating this agent within rigorous and clinically relevant frameworks.
    Journal • Tumor mutational burden
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    TMB (Tumor Mutational Burden)
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    pidnarulex (CX-5461)
    7ms
    High-throughput combination screening of Pidnarulex and other G-quadruplex ligands in multi-cell type tumor spheroids. (PubMed, SLAS Discov)
    To investigate the therapeutic potential of three G4 ligands-pidnarulex, APTO-253, and BRACO-19-a high-throughput drug combination screen was conducted in thirty-one multi-cell type tumor spheroids derived from patient tumors and established cancer cell lines. Brightfield imaging corroborated enhanced spheroid growth suppression from synergistic combinations. These findings underscore the context-dependent activity of G4 ligands and support the use of integrated functional and imaging-based approaches to characterize potential therapeutic combinations in physiologically relevant 3D cancer models.
    Journal
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    PIM1 (Pim-1 Proto-Oncogene)
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    pidnarulex (CX-5461) • APTO-253