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Introduction <p>Systemic juvenile idiopathic arthritis (sJIA) is an autoinflammatory disorder that impacts children aged ≤ 16&#xa0;years. This study evaluated the efficacy and safety of firsekibart, a fully human anti-IL-1β monoclonal antibody, in children with active sJIA.</p> Methods <p>This multicenter, randomized, open-label, active-controlled phase 2 study (NCT05925452) enrolled participants aged 2– &lt; 18&#xa0;years with active sJIA across 14 sites in China. Participants were randomized (1:1:1) to firsekibart 3.0&#xa0;mg/kg or 4.0&#xa0;mg/kg subcutaneously every 4&#xa0;weeks, or tocilizumab intravenously every 2&#xa0;weeks, for 24&#xa0;weeks. The primary endpoint was modified JIA American College of Rheumatology Pediatric criteria (ACR Pedi) 30 response at day 28. Secondary endpoints included ACR Pedi 30/50/70/90 responses, corticosteroid tapering, immunogenicity, and safety.</p> Results <p>Fifty participants were randomized (firsekibart 3.0&#xa0;mg/kg: <i>n</i> = 17; 4.0&#xa0;mg/kg: <i>n</i> = 16; tocilizumab: <i>n</i> = 17). At day 28, ACR Pedi 30 response rates were 94.1% (95% CI 71.3–99.9), 75.0% (47.6–92.7) and 82.4% (56.6–96.2) in the firsekibart 3.0&#xa0;mg/kg, 4.0&#xa0;mg/kg, and tocilizumab groups, respectively. Firsekibart 3.0&#xa0;mg/kg achieved numerically higher ACR Pedi 70/90 responses. By the end of treatment, successful corticosteroid tapering occurred in 75.0%, 63.6%, and 62.5% of patients receiving firsekibart 3.0&#xa0;mg/kg, 4.0&#xa0;mg/kg, and tocilizumab, respectively. No MAS events or anti-drug or neutralizing antibodies were detected. Treatment-emergent adverse events (TEAEs) occurred in 70.6%, 93.8%, and 94.1% of participants in the firsekibart 3.0&#xa0;mg/kg, 4.0&#xa0;mg/kg, and tocilizumab groups, respectively. Grade ≥ 3 TEAEs were absent in the 3.0&#xa0;mg/kg group. Common TEAEs mainly included infections and laboratory abnormalities.</p> Conclusions <p>Firsekibart demonstrated rapid clinical improvement, steroid-sparing effects, and an acceptable safety profile. The 3.0&#xa0;mg/kg dose provided the most favorable benefit–risk balance, with efficacy and safety outcomes comparable to tocilizumab, supporting further evaluation as a convenient once-every-4-weeks IL-1β-targeted option in future phase 3 trials.</p> Trial Registration <p>ClinicalTrials.gov identifier, NCT05925452</p>

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Efficacy and Safety of Firsekibart in Treatment of Active Systemic Juvenile Idiopathic Arthritis: A Randomized Phase 2 Study

  • 期刊论文

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  • 发表日期：

    2026年3月11日

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  • Caifeng Li,
  • Junmei Zhang,
  • Haiguo Yu,
  • Meiping Lu,
  • Sirui Yang,
  • Cuihua Liu,
  • Ping Zeng,
  • Bo Zhao,
  • Xiaoqing Li,
  • Wenjie Zheng,
  • Yue Du,
  • Wei Zhang,
  • Zhihui Li,
  • Xiufen Hu,
  • Jianming Lai,
  • Qian Xu,
  • Xu Zhang,
  • Xianyang Wang,
  • Palasaiti Adili,
  • Wei Qu

摘要

Introduction

Systemic juvenile idiopathic arthritis (sJIA) is an autoinflammatory disorder that impacts children aged ≤ 16 years. This study evaluated the efficacy and safety of firsekibart, a fully human anti-IL-1β monoclonal antibody, in children with active sJIA.

Methods

This multicenter, randomized, open-label, active-controlled phase 2 study (NCT05925452) enrolled participants aged 2– < 18 years with active sJIA across 14 sites in China. Participants were randomized (1:1:1) to firsekibart 3.0 mg/kg or 4.0 mg/kg subcutaneously every 4 weeks, or tocilizumab intravenously every 2 weeks, for 24 weeks. The primary endpoint was modified JIA American College of Rheumatology Pediatric criteria (ACR Pedi) 30 response at day 28. Secondary endpoints included ACR Pedi 30/50/70/90 responses, corticosteroid tapering, immunogenicity, and safety.

Results

Fifty participants were randomized (firsekibart 3.0 mg/kg: n = 17; 4.0 mg/kg: n = 16; tocilizumab: n = 17). At day 28, ACR Pedi 30 response rates were 94.1% (95% CI 71.3–99.9), 75.0% (47.6–92.7) and 82.4% (56.6–96.2) in the firsekibart 3.0 mg/kg, 4.0 mg/kg, and tocilizumab groups, respectively. Firsekibart 3.0 mg/kg achieved numerically higher ACR Pedi 70/90 responses. By the end of treatment, successful corticosteroid tapering occurred in 75.0%, 63.6%, and 62.5% of patients receiving firsekibart 3.0 mg/kg, 4.0 mg/kg, and tocilizumab, respectively. No MAS events or anti-drug or neutralizing antibodies were detected. Treatment-emergent adverse events (TEAEs) occurred in 70.6%, 93.8%, and 94.1% of participants in the firsekibart 3.0 mg/kg, 4.0 mg/kg, and tocilizumab groups, respectively. Grade ≥ 3 TEAEs were absent in the 3.0 mg/kg group. Common TEAEs mainly included infections and laboratory abnormalities.

Conclusions

Firsekibart demonstrated rapid clinical improvement, steroid-sparing effects, and an acceptable safety profile. The 3.0 mg/kg dose provided the most favorable benefit–risk balance, with efficacy and safety outcomes comparable to tocilizumab, supporting further evaluation as a convenient once-every-4-weeks IL-1β-targeted option in future phase 3 trials.

Trial Registration

ClinicalTrials.gov identifier, NCT05925452