Kinzel Olaf, Goldberg Steven D, Cummings Maxwell D, Gege Christian, Steeneck Christoph, Xue Xiaohua, Albers Michael, Schlüter Thomas, Kleymann Gerald, Scott Brian, Sepassi Kia, Schoetens Freddy, Venkatesan Hariharan, Tanis Virginia M, Coe Kevin, Sales Zachary S, Spurlino John, Milligan Cynthia, Fourie Anne M, Edwards James P, Hoffmann Thomas
Phenex Pharmaceuticals AG, Waldhofer Strasse 104, 69123 Heidelberg, Germany.
Johnson & Johnson Innovative Medicine, 3210 Merryfield Row, San Diego, California92121-1126, United States.
J Med Chem. 2025 Apr 24;68(8):8713-8728. doi: 10.1021/acs.jmedchem.5c00390. Epub 2025 Apr 16.
The retinoic acid receptor-related orphan receptor gamma t (RORγt) is a nuclear transcription factor expressed in both innate and adaptive immune cells, driving Th17 cell differentiation and IL-17 production. The IL-23/IL-17 pathway is implicated in autoimmune and inflammatory diseases, and biologics that target IL-23/IL-17 signaling are efficacious in the treatment of psoriasis and psoriatic arthritis. RORγt, at the core of this pathway, represents an attractive opportunity for small-molecule intervention; however, combining high potency, nuclear receptor selectivity, and good physicochemical properties remains a challenge for RORγt inverse agonists. Recently, thiazole amides have been identified as potent RORγt inverse agonists; however, they often suffer from CYP450 autoinduction in the rat, precluding further development. Herein, we describe the discovery and development of potent and selective thiazole bisamide RORγt inverse agonists that avoid autoinduction in the rat. This effort culminated in the discovery of JNJ-61803534, which advanced into phase 1 clinical trials.
视黄酸受体相关孤儿受体γt(RORγt)是一种在先天性和适应性免疫细胞中均有表达的核转录因子,可驱动辅助性T细胞17(Th17)分化并产生白细胞介素-17(IL-17)。IL-23/IL-17信号通路与自身免疫性疾病和炎症性疾病相关,靶向IL-23/IL-17信号传导的生物制剂在治疗银屑病和银屑病关节炎方面有效。RORγt作为该信号通路的核心,是小分子干预的一个有吸引力的靶点;然而,要同时具备高效能、核受体选择性和良好的物理化学性质,对于RORγt反向激动剂来说仍是一项挑战。最近,噻唑酰胺已被确定为强效RORγt反向激动剂;然而,它们在大鼠中常常会导致细胞色素P450(CYP450)自身诱导,从而无法进一步开发。在此,我们描述了强效且选择性的噻唑双酰胺RORγt反向激动剂的发现与开发过程,该激动剂可避免在大鼠中产生自身诱导作用。这一研究最终发现了JNJ-61803534,并已进入1期临床试验阶段。