Skip to main content
ENDO 2026 Main banner

Pediatric and Adolescent Endocrinology

ORF47-04 - PK/PD Modeling, Efficacy and Safety of Pegpesen in Children with Non-Growth Hormone Deficiency Short Stature: A Basket Trial

Monday, June 15, 2026
2:00 PM - 2:15 PM CT
Location: Room W187

    Senior Author/Primary Investigator(s)

  • YL

    Yan Liang, n/a

    Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology
    Wuhan, China (People's Republic)

    • Co-Author(s)

  • YL

    Yan Liang, n/a

    Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology
    Wuhan, China (People's Republic)

  • HY

    Hui Yao, n/a

    Wuhan Children’s Hospital, Tongji Medical College, Huazhong University of Science and Technology
    Wuhan, China (People's Republic)

  • HW

    Haiyan Wei, n/a

    Children’s Hospital Affiliated to Zhengzhou University, Henan Children’s Hospital, Zhengzhou Children’s Hospital
    Henan, China (People's Republic)

  • QL

    Qun Lian, n/a

    The First Affiliated Hospital of Xiamen University
    Xiamen, United States

  • FY

    Fan Yang, Chief Physician, Department of Pediatrics

    Department of Pediatrics, West China Second University Hospital, Sichuan University
    Chengdu, China (People's Republic)

  • RX

    Rongrong Xie, n/a

    Children's Hospital of Soochow University
    Suzhou, China (People's Republic)

  • YY

    Yu Yang, n/a

    Jiangxi Provincial Children’s Hospita
    Nanchang, China (People's Republic)

  • XC

    Xinran Cheng, n/a

    University of Electronic Science and Technology of China, Chengdu Women’s and Children’s Central Hospital
    Chengdu, United States

  • XL

    Xiaoming Luo, n/a

    Zhejiang Provincial People's Hospital
    Hangzhou, China (People's Republic)

  • LL

    Liyang Liang, n/a

    Sun Yat-Sen Memorial Hospital, Sun Yat-Sen University
    Guangzhou, China (People's Republic)

  • YZ

    Yan Zhong, n/a

    Hunan Children’s Hospital
    Changsha, China (People's Republic)

  • HZ

    Hua Zhang, n/a

    Sanya Central Hospital
    Sanya, China (People's Republic)

  • SZ

    Shunye Zhu, n/a

    The Third Affiliated Hospital, Sun Yat-sen University
    Guangzhou, China (People's Republic)

  • PL

    Pin Li, n/a

    Children’s Hospital of Shanghai, Shanghai Jiao Tong University
    Shanghai, China (People's Republic)

  • YM

    Yaping Ma, n/a

    Affiliated Hospital of Jiangnan University
    Wuxi, China (People's Republic)

  • DL

    Deyun Liu, n/a

    The Second Hospital of Anhui Medical University
    Hefei, China (People's Republic)

  • XC

    Xiangao Cheng, n/a

    The First Affiliated Hospital of Anhui Medical University,Anhui Public Health Clinical Center
    Hefei, China (People's Republic)

  • RH

    Ruoyi He, n/a

    Xiamen Amoytop Biotech Co., Ltd
    Xiamen, China (People's Republic)

  • YY

    Yalin Yin, n/a

    Xiamen Amoytop Biotech Co., Ltd
    Xiamen, China (People's Republic)

  • LS

    Li Sun, n/a

    Xiamen Amoytop Biotech Co., Ltd
    Xiamen, China (People's Republic)

    • Oral Abstract Presenter(s)

  • Xiaoping Luo, MD, PhD

    Tongji Hospital of Tongji Medical College, Huazhong University of Science and Technology
    Wuhan, China

    • Senior Author/Primary Investigator(s)

  • Xiaoping Luo, MD, PhD

    Tongji Hospital of Tongji Medical College, Huazhong University of Science and Technology
    Wuhan, China

    • Co-Author(s)

  • Xiaoping Luo, MD, PhD

    Tongji Hospital of Tongji Medical College, Huazhong University of Science and Technology
    Wuhan, China

Disclosure(s):
Xiaoping Luo, MD, PhD: Amoytop: Advisory Board (), Grant Recipient (); GenSci: Advisory Board (), Grant Recipient (), Speakers' Bureau (); Ipsen: Advisory Board (), Grant Recipient (); Kyowa Kirin: Advisory Board (), Grant Recipient (); Lumos: Advisory Board (), Grant Recipient (); Medtronic: Advisory Board (), Grant Recipient (); Novo Nordisk: Advisory Board (), Grant Recipient (), Speakers' Bureau (); Sanofi: Advisory Board (), Grant Recipient (); Takeda: Advisory Board (), Grant Recipient (); Visen: Advisory Board (), Grant Recipient ()



Objectives: To address the critical gap in pharmacokinetic/pharmacodynamic (PK/PD) and dose-response data for long-acting growth hormones in non-growth hormone deficiency (non-GHD) populations, this study evaluated the dose-response relationship and safety of Pegpesen, a novel Y-shaped 40 kDa PEGylated recombinant human growth hormone (rhGH), in children with idiopathic short stature (ISS), small for gestational age (SGA), and Turner syndrome (TS).

Methods: This multicenter, randomized, open-label, active-controlled, phase 2 basket trial enrolled treatment-naïve, prepubertal children with ISS, SGA, or TS. Participants were randomized (2:2:1:1) to receive either once-weekly Pegpesen (140 or 280 µg/kg/week) or daily rhGH (245 or 470 µg/kg/week) for 52 weeks. The primary objectives were to characterize the dose-response relationship between Pegpesen, insulin-like growth factor-1 (IGF-1), and growth velocity (GV) via population PK/PD modeling, alongside assessments of efficacy and safety.

Results: A total of 78 subjects were randomized. The population PK/PD model demonstrated no significant drug accumulation with Pegpesen 280 μg/kg/week at steady state. Mean IGF-1 SDS remained below +2, with 90.4% of subjects maintaining trough IGF-1 SDS > 0. Dose-dependent efficacy improvements were observed, with Pegpesen 280 μg/kg/week showing comparable GV to daily rhGH 470 μg/kg/week across ISS, SGA, and TS cohorts (10.91 vs. 10.80, 11.98 vs. 11.30, 9.37 vs. 9.95 cm/year). Safety profiles were similar between treatment groups.

Conclusions: Once-weekly Pegpesen 280 μg/kg/week demonstrated comparable efficacy and safety to daily rhGH 470 μg/kg/week consistently across ISS, SGA, and TS populations, with sustained IGF-1 exposure and reduced dosing frequency. These findings support its further development as a weekly therapeutic option for non-GHD short stature.