Koester Anja, Witcher Derrick R, Lee Mark, Demarest Stephen J, Potter Scott, Werle Katie, Bauer Scott, Ruiz Diana, Malherbe Laurent, Poorbaugh Josh, Glasebrook Andrew, Preuss Christoph, Datta Gourab, Wang Ziqiao, Knorr Jack, Manner David, Patel Dipak, Schmitz Carsten, Klekotka Paul, Nirula Ajay
Lilly Biotechnology Center, San Diego, Eli Lilly and Company, Immunology Research, San Diego, CA, USA.
Lilly Research Laboratories, Eli Lilly and Company, Biotechnology, Indianapolis, IN, USA.
Nat Commun. 2025 May 1;16(1):4082. doi: 10.1038/s41467-025-59147-w.
CD200R is a checkpoint inhibitory receptor central to the pathogenesis of inflammatory skin disease. Here we describe the development and phase 1 clinical study (NCT03750643) of ucenprubart, a CD200R agonist antibody to downregulate immune system inflammation. Preclinical studies find ucenprubart inhibiting Fcγ receptor-induced cytokine secretion from myeloid cells in vitro and demonstrating efficacy in a mouse contact hypersensitivity model. The randomized, placebo-controlled, NCT03750643 trial assesses safety and pharmacokinetics in healthy subjects, and efficacy in atopic dermatitis patients. The primary efficacy outcome is the proportion of patients achieving Validated Investigator's Global Assessment for Atopic Dermatitis (vIGA-AD) 0 or 1 with ≥2-point improvement from baseline at week 12. Secondary outcomes are proportions of patients achieving the primary outcome and mean changes in Eczema Area and Severity Index (EASI) and SCORing Atopic Dermatitis (SCORAD) across weeks 1 through 12, and cutoffs at week 12. Sixty-two healthy participants and 40 patients are enrolled. No serious adverse events or discontinuations due to adverse events is seen with ucenprubart. The primary endpoint is not met; however, overall improvements are observed in EASI-75 and SCORAD through 12 weeks. CD200R may be a promising therapeutic target for treating autoimmune disease, including inflammatory skin diseases.
CD200R是一种在炎症性皮肤病发病机制中起核心作用的检查点抑制受体。在此,我们描述了ucenprubart的研发及1期临床研究(NCT03750643),ucenprubart是一种用于下调免疫系统炎症的CD200R激动剂抗体。临床前研究发现,ucenprubart在体外可抑制Fcγ受体诱导的髓样细胞分泌细胞因子,并在小鼠接触性超敏反应模型中显示出疗效。随机、安慰剂对照的NCT03750643试验评估了健康受试者的安全性和药代动力学,以及特应性皮炎患者的疗效。主要疗效指标是在第12周时达到特应性皮炎验证研究者整体评估(vIGA-AD)为0或1且较基线改善≥2分的患者比例。次要指标是在第1周至第12周期间达到主要指标的患者比例、湿疹面积和严重程度指数(EASI)及特应性皮炎评分(SCORAD)的平均变化,以及第12周时的截断值。共招募了62名健康参与者和40名患者。未观察到ucenprubart导致的严重不良事件或因不良事件而停药的情况。主要终点未达到;然而,在12周内EASI-75和SCORAD总体上有所改善。CD200R可能是治疗自身免疫性疾病(包括炎症性皮肤病)的一个有前景的治疗靶点。