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Development of Potent Mcl-1 Inhibitors: Structural Investigations on Macrocycles Originating from a DNA-Encoded Chemical Library Screen
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    Development of Potent Mcl-1 Inhibitors: Structural Investigations on Macrocycles Originating from a DNA-Encoded Chemical Library Screen
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    • Koen F. W. Hekking*
      Koen F. W. Hekking
      Symeres, 6546BB Nijmegen, The Netherlands
      *Email: [email protected]
    • Sergio Maroto
      Sergio Maroto
      Symeres, 6546BB Nijmegen, The Netherlands
    • Kees van Kekem
      Kees van Kekem
      Symeres, 6546BB Nijmegen, The Netherlands
    • Frank S. Haasjes
      Frank S. Haasjes
      Symeres, 6546BB Nijmegen, The Netherlands
    • Jack C. Slootweg
      Jack C. Slootweg
      Symeres, 6546BB Nijmegen, The Netherlands
    • Patrick G. B. Oude Alink
      Patrick G. B. Oude Alink
      Symeres, 6546BB Nijmegen, The Netherlands
    • Ron Dirks
      Ron Dirks
      Symeres, 6546BB Nijmegen, The Netherlands
      More by Ron Dirks
    • Malvika Sardana
      Malvika Sardana
      Symeres, 6546BB Nijmegen, The Netherlands
    • Marjon G. Bolster
      Marjon G. Bolster
      Symeres, 6546BB Nijmegen, The Netherlands
    • Brian Kuijpers
      Brian Kuijpers
      Symeres, 6546BB Nijmegen, The Netherlands
    • Dennis Smith
      Dennis Smith
      Symeres, 6546BB Nijmegen, The Netherlands
      More by Dennis Smith
    • Robin Doodeman
      Robin Doodeman
      Symeres, 6546BB Nijmegen, The Netherlands
    • Marcel Scheepstra
      Marcel Scheepstra
      Symeres, 6546BB Nijmegen, The Netherlands
    • Birgit Zech
      Birgit Zech
      X-Rx, Inc., New York, New York 10016, United States
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    • Mark Mulvihill
      Mark Mulvihill
      X-Rx, Inc., New York, New York 10016, United States
    • Louis M. Renzetti
      Louis M. Renzetti
      X-Rx, Inc., New York, New York 10016, United States
    • Lee Babiss
      Lee Babiss
      X-Rx, Inc., New York, New York 10016, United States
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    • Paolo A. Centrella
      Paolo A. Centrella
      X-Chem, Inc., Waltham, Massachusetts 02453, United States
    • Matthew A. Clark
      Matthew A. Clark
      X-Chem, Inc., Waltham, Massachusetts 02453, United States
    • John W. Cuozzo
      John W. Cuozzo
      X-Chem, Inc., Waltham, Massachusetts 02453, United States
    • Marie-Aude Guié
      Marie-Aude Guié
      X-Chem, Inc., Waltham, Massachusetts 02453, United States
    • Eric Sigel
      Eric Sigel
      X-Chem, Inc., Waltham, Massachusetts 02453, United States
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    • Sevan Habeshian
      Sevan Habeshian
      X-Chem, Inc., Waltham, Massachusetts 02453, United States
    • Christopher D. Hupp
      Christopher D. Hupp
      X-Chem, Inc., Waltham, Massachusetts 02453, United States
    • Julie Liu
      Julie Liu
      X-Chem, Inc., Waltham, Massachusetts 02453, United States
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    • Heather A. Thomson
      Heather A. Thomson
      X-Chem, Inc., Waltham, Massachusetts 02453, United States
    • Ying Zhang
      Ying Zhang
      X-Chem, Inc., Waltham, Massachusetts 02453, United States
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    • Anthony D. Keefe*
      Anthony D. Keefe
      X-Chem, Inc., Waltham, Massachusetts 02453, United States
      *Email: [email protected]
    • Gerhard Müller
      Gerhard Müller
      Symeres, 6546BB Nijmegen, The Netherlands
    • Stijn Gremmen
      Stijn Gremmen
      Symeres, 6546BB Nijmegen, The Netherlands
    Other Access OptionsSupporting Information (2)

    Journal of Medicinal Chemistry

    Cite this: J. Med. Chem. 2024, 67, 4, 3039–3065
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    https://doi.org/10.1021/acs.jmedchem.3c02206
    Published February 2, 2024
    Copyright © 2024 American Chemical Society

    Abstract

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    Abstract Image

    Evasion of apoptosis is critical for the development and growth of tumors. The pro-survival protein myeloid cell leukemia 1 (Mcl-1) is an antiapoptotic member of the Bcl-2 family, associated with tumor aggressiveness, poor survival, and drug resistance. Development of Mcl-1 inhibitors implies blocking of protein–protein interactions, generally requiring a lengthy optimization process of large, complex molecules. Herein, we describe the use of DNA-encoded chemical library synthesis and screening to directly generate complex, yet conformationally privileged macrocyclic hits that serve as Mcl-1 inhibitors. By applying a conceptual combination of conformational analysis and structure-based design in combination with a robust synthetic platform allowing rapid analoging, we optimized in vitro potency of a lead series into the low nanomolar regime. Additionally, we demonstrate fine-tuning of the physicochemical properties of the macrocyclic compounds, resulting in the identification of lead candidates 57/59 with a balanced profile, which are suitable for future development toward therapeutic use.

    Copyright © 2024 American Chemical Society

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    Supporting Information

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    The Supporting Information is available free of charge at https://pubs.acs.org/doi/10.1021/acs.jmedchem.3c02206.

    • Experimental details combinatorial array synthesis; assay results for all combinatorial compounds; NMR spectra and HPLC traces of key compounds; single-molecule crystallography data for compound 2, including Ortep-drawing; protein–ligand crystallography data for compound 1; and conformational analysis NMR data (PDF)

    • Molecular formula strings for all final compounds (CSV)

    Accession Codes

    Coordinates for compound 1 in complex with Mcl-1 have been deposited into the Protein Data Bank (PDB: 8QSO). The authors will release the atomic coordinates upon article publication.

    Terms & Conditions

    Most electronic Supporting Information files are available without a subscription to ACS Web Editions. Such files may be downloaded by article for research use (if there is a public use license linked to the relevant article, that license may permit other uses). Permission may be obtained from ACS for other uses through requests via the RightsLink permission system: http://pubs.acs.org/page/copyright/permissions.html.

    Cited By

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    This article is cited by 3 publications.

    1. Millicent Dockerill, Pramod M. Sabale, Francesco Russo, Sofia Barluenga, Nicolas Winssinger. Translation of Deoxyribonucleic Acid into Synthetic Alpha Helical Peptides for Darwinian Evolution. JACS Au 2024, 4 (10) , 4013-4022. https://doi.org/10.1021/jacsau.4c00738
    2. Yanrui Suo, Kaige Li, Xing Ling, Kenian Yan, Weiwei Lu, Jinfeng Yue, Xiaohua Chen, Zhiqiang Duan, Xiaojie Lu. Discovery Small-Molecule p300 Inhibitors Derived from a Newly Developed Indazolone-Focused DNA-Encoded Library. Bioconjugate Chemistry 2024, 35 (8) , 1251-1257. https://doi.org/10.1021/acs.bioconjchem.4c00307
    3. Jiasheng Guo, Chenning Yu, Kenan Li, Yijian Zhang, Guoqiang Wang, Shuhua Li, Hao Dong. Retrosynthesis Zero: Self-Improving Global Synthesis Planning Using Reinforcement Learning. Journal of Chemical Theory and Computation 2024, 20 (11) , 4921-4938. https://doi.org/10.1021/acs.jctc.4c00071

    Journal of Medicinal Chemistry

    Cite this: J. Med. Chem. 2024, 67, 4, 3039–3065
    Click to copy citationCitation copied!
    https://doi.org/10.1021/acs.jmedchem.3c02206
    Published February 2, 2024
    Copyright © 2024 American Chemical Society

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