Thompson Andrew M, O'Connor Patrick D, Marshall Andrew J, Yardley Vanessa, Maes Louis, Gupta Suman, Launay Delphine, Braillard Stephanie, Chatelain Eric, Franzblau Scott G, Wan Baojie, Wang Yuehong, Ma Zhenkun, Cooper Christopher B, Denny William A
Auckland Cancer Society Research Centre, School of Medical Sciences, The University of Auckland , Private Bag 92019, Auckland 1142, New Zealand.
Faculty of Infectious & Tropical Diseases, London School of Hygiene & Tropical Medicine , Keppel Street, London WC1E 7HT, United Kingdom.
J Med Chem. 2017 May 25;60(10):4212-4233. doi: 10.1021/acs.jmedchem.7b00034. Epub 2017 May 11.
Within a backup program for the clinical investigational agent pretomanid (PA-824), scaffold hopping from delamanid inspired the discovery of a novel class of potent antitubercular agents that unexpectedly possessed notable utility against the kinetoplastid disease visceral leishmaniasis (VL). Following the identification of delamanid analogue DNDI-VL-2098 as a VL preclinical candidate, this structurally related 7-substituted 2-nitro-5,6-dihydroimidazo[2,1-b][1,3]oxazine class was further explored, seeking efficacious backup compounds with improved solubility and safety. Commencing with a biphenyl lead, bioisosteres formed by replacing one phenyl by pyridine or pyrimidine showed improved solubility and potency, whereas more hydrophilic side chains reduced VL activity. In a Leishmania donovani mouse model, two racemic phenylpyridines (71 and 93) were superior, with the former providing >99% inhibition at 12.5 mg/kg (b.i.d., orally) in the Leishmania infantum hamster model. Overall, the 7R enantiomer of 71 (79) displayed more optimal efficacy, pharmacokinetics, and safety, leading to its selection as the preferred development candidate.
在临床研究药物pretomanid(PA - 824)的一个备份项目中,从delamanid进行骨架跃迁启发了一类新型强效抗结核药物的发现,这些药物意外地对动基体疾病内脏利什曼病(VL)具有显著疗效。在确定delamanid类似物DNDI - VL - 2098为VL临床前候选药物后,对这种结构相关的7 - 取代2 - 硝基 - 5,6 - 二氢咪唑并[2,1 - b][1,3]恶嗪类进行了进一步探索,以寻找具有改善溶解性和安全性的有效备用化合物。从一个联苯先导化合物开始,用吡啶或嘧啶取代一个苯基形成的生物电子等排体显示出改善的溶解性和活性,而更多亲水性侧链则降低了VL活性。在杜氏利什曼原虫小鼠模型中,两种外消旋苯基吡啶(71和93)表现更优,前者在婴儿利什曼原虫仓鼠模型中以12.5 mg/kg(每日两次,口服)提供>99%的抑制率。总体而言,71的7R对映体(79)显示出更优的疗效、药代动力学和安全性,因此被选为首选的开发候选药物。