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From a novel HTS hit to potent, selective, and orally bioavailable KDM5 inhibitors.
Liang, Jun; Labadie, Sharada; Zhang, Birong; Ortwine, Daniel F; Patel, Snahel; Vinogradova, Maia; Kiefer, James R; Mauer, Till; Gehling, Victor S; Harmange, Jean-Christophe; Cummings, Richard; Lai, Tommy; Liao, Jiangpeng; Zheng, Xiaoping; Liu, Yichin; Gustafson, Amy; Van der Porten, Erica; Mao, Weifeng; Liederer, Bianca M; Deshmukh, Gauri; An, Le; Ran, Yingqing; Classon, Marie; Trojer, Patrick; Dragovich, Peter S; Murray, Lesley.
Afiliación
  • Liang J; Genentech Inc., 1 DNA Way, South San Francisco, CA 94080, USA. Electronic address: liang.jun@gene.com.
  • Labadie S; Genentech Inc., 1 DNA Way, South San Francisco, CA 94080, USA.
  • Zhang B; Genentech Inc., 1 DNA Way, South San Francisco, CA 94080, USA.
  • Ortwine DF; Genentech Inc., 1 DNA Way, South San Francisco, CA 94080, USA.
  • Patel S; Genentech Inc., 1 DNA Way, South San Francisco, CA 94080, USA.
  • Vinogradova M; Genentech Inc., 1 DNA Way, South San Francisco, CA 94080, USA.
  • Kiefer JR; Genentech Inc., 1 DNA Way, South San Francisco, CA 94080, USA.
  • Mauer T; Genentech Inc., 1 DNA Way, South San Francisco, CA 94080, USA.
  • Gehling VS; Constellation Pharmaceuticals Inc., 215 First Street, Suite 200, Cambridge, MA 02142, USA.
  • Harmange JC; Constellation Pharmaceuticals Inc., 215 First Street, Suite 200, Cambridge, MA 02142, USA.
  • Cummings R; Constellation Pharmaceuticals Inc., 215 First Street, Suite 200, Cambridge, MA 02142, USA.
  • Lai T; WuXi AppTec Co., Ltd., 288 Fute Zhong Road, Waigaoqiao Free Trade Zone, Shanghai 200131, China.
  • Liao J; WuXi AppTec Co., Ltd., 288 Fute Zhong Road, Waigaoqiao Free Trade Zone, Shanghai 200131, China.
  • Zheng X; WuXi AppTec Co., Ltd., 288 Fute Zhong Road, Waigaoqiao Free Trade Zone, Shanghai 200131, China.
  • Liu Y; Genentech Inc., 1 DNA Way, South San Francisco, CA 94080, USA.
  • Gustafson A; Genentech Inc., 1 DNA Way, South San Francisco, CA 94080, USA.
  • Van der Porten E; Genentech Inc., 1 DNA Way, South San Francisco, CA 94080, USA.
  • Mao W; WuXi AppTec Co., Ltd., 288 Fute Zhong Road, Waigaoqiao Free Trade Zone, Shanghai 200131, China.
  • Liederer BM; Genentech Inc., 1 DNA Way, South San Francisco, CA 94080, USA.
  • Deshmukh G; Genentech Inc., 1 DNA Way, South San Francisco, CA 94080, USA.
  • An L; Genentech Inc., 1 DNA Way, South San Francisco, CA 94080, USA.
  • Ran Y; Genentech Inc., 1 DNA Way, South San Francisco, CA 94080, USA.
  • Classon M; Genentech Inc., 1 DNA Way, South San Francisco, CA 94080, USA.
  • Trojer P; Constellation Pharmaceuticals Inc., 215 First Street, Suite 200, Cambridge, MA 02142, USA.
  • Dragovich PS; Genentech Inc., 1 DNA Way, South San Francisco, CA 94080, USA.
  • Murray L; Genentech Inc., 1 DNA Way, South San Francisco, CA 94080, USA.
Bioorg Med Chem Lett ; 27(13): 2974-2981, 2017 07 01.
Article en En | MEDLINE | ID: mdl-28512031
A high-throughput screening (HTS) of the Genentech/Roche library identified a novel, uncharged scaffold as a KDM5A inhibitor. Lacking insight into the binding mode, initial attempts to improve inhibitor potency failed to improve potency, and synthesis of analogs was further hampered by the presence of a C-C bond between the pyrrolidine and pyridine. Replacing this with a C-N bond significantly simplified synthesis, yielding pyrazole analog 35, of which we obtained a co-crystal structure with KDM5A. Using structure-based design approach, we identified 50 with improved biochemical, cell potency and reduced MW and lower lipophilicity (LogD) compared with the original hit. Furthermore, 50 showed lower clearance than 9 in mice. In combination with its remarkably low plasma protein binding (PPB) in mice (40%), oral dosing of 50 at 5mg/kg resulted in unbound Cmax ∼2-fold of its cell potency (PC9 H3K4Me3 0.96µM), meeting our criteria for an in vivo tool compound from a new scaffold.
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Pirazoles / Inhibidores Enzimáticos / Proteína 2 de Unión a Retinoblastoma / Ensayos Analíticos de Alto Rendimiento Tipo de estudio: Prognostic_studies Límite: Animals / Humans Idioma: En Revista: Bioorg Med Chem Lett Asunto de la revista: BIOQUIMICA / QUIMICA Año: 2017 Tipo del documento: Article Pais de publicación: Reino Unido

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Pirazoles / Inhibidores Enzimáticos / Proteína 2 de Unión a Retinoblastoma / Ensayos Analíticos de Alto Rendimiento Tipo de estudio: Prognostic_studies Límite: Animals / Humans Idioma: En Revista: Bioorg Med Chem Lett Asunto de la revista: BIOQUIMICA / QUIMICA Año: 2017 Tipo del documento: Article Pais de publicación: Reino Unido