A multicenter, randomized, open‐label, phase 2 clinical study of telitacicept in adult patients with generalized myasthenia gravis
Jian Yin, Mingming Zhao, Xianhao Xu, Meini Zhang, Zucai Xu, Zunbo Li, Xinyue Qin, Zhuyi Li, Chongbo Zhao, Hongyu Zhou, Ying Ma, Wenfeng Cao, Guoping Wang, Yongzhong Lin, Jizhong Zhang, Xu Zhang, Hongbin Cai, Weidong Qian, Yiqi Wang, Xinghu Zhang, Guangzhi Liu, Jiawei Wang, Wei Qiu, Lianqiu Min, Jing Li, Hui Deng, Lan Chu, Yifan Zhang, Jianmin FangAbstract
Background and purpose
This study aimed to investigate the clinical efficacy and safety of telitacicept in patients with generalized myasthenia gravis (gMG) who tested positive for acetylcholine receptor antibodies or muscle‐specific kinase antibodies and were receiving standard‐of‐care therapy.
Methods
Patients meeting the eligibility criteria were randomly assigned to receive telitacicept subcutaneously once a week for 24 weeks in addition to standard‐of‐care treatment. The primary efficacy endpoint was the mean change in the quantitative myasthenia gravis (QMG) score from baseline to week 24. Secondary efficacy endpoints included mean change in QMG score from baseline to week 12 and gMG clinical absolute score from baseline to week 24. Additionally, safety, tolerability and pharmacodynamics were assessed.
Results
Twenty‐nine of the 41 patients screened were randomly selected and enrolled. The mean (± standard deviation [SD]) reduction in QMG score from baseline to week 24 was 7.7 (± 5.34) and 9.6 (± 4.29) in the 160 mg and 240 mg groups, respectively. At week 12, mean reductions in QMG scores for these two groups were 5.8 (± 5.85) and 9.5 (± 5.03), respectively, indicating rapid clinical improvement. Safety analysis revealed no adverse events leading to discontinuation or mortalities. All patients showed consistent reductions in serum immunoglobulin (Ig) A, IgG and IgM levels throughout the study.
Conclusion
Telitacicept demonstrated safety, good tolerability and reduced clinical severity throughout the study period. Further validation of the clinical efficacy of telitacicept in gMG will be conducted in an upcoming phase 3 clinical trial.