ESPE Abstracts (2021) 94 P1-161

ESPE2021 ePoster Category 1 Growth B (10 abstracts)

Continued Safety and Efficacy of Weekly Lonapegsomatropin (TransCon hGH) for up to Two Years in Children with Growth Hormone Deficiency (GHD)

Elena M. Aghajanova 1 , Samuel J. Casella 2 , Ulhas Nadgir 3 , Paul Hofman 4 , Paul Saenger 5 , Wenjie Song 6 , Meng Mao 6 , Steven Chessler 6 , Allison S. Komirenko 6 , Michael Beckert 7 , Aimee D. Shu 6 , Paul S. Thornton 8 & Aristides K. Maniatis 9

1Yerevan State Medical University, Yerevan, Armenia; 2Children’s Hospital at Dartmouth, Lebanon, USA; 3Center of Excellence in Diabetes and Endocrinology, Sacramento, USA; 4Liggins Institute, University of Auckland, Auckland, New Zealand; 5New York University Langone, Mineola, USA; 6Ascendis Pharma, Inc., Palo Alto, USA; 7Ascendis Pharma, A/S, Hellerup, Denmark; 8Cook Children’s Medical Center, Fort Worth, USA; 9Rocky Mountain Pediatric Endocrinology, Centennial, USA

Lonapegsomatropin (TransCon hGH) is an investigational once-weekly prodrug of somatropin for the treatment of GHD. Previous trials in treatment-naïve (52-week heiGHt Trial) and treatment-experienced children (26-week fliGHt Trial) have reported the efficacy and safety of lonapegsomatropin. Subjects were eligible to enter the open-label extension enliGHten Trial, which continues to evaluate weekly lonapegsomatropin in pediatric GHD. In heiGHt, treatment-naïve subjects received lonapegsomatropin (Group A; vial/syringe) or daily somatropin (Group B; pen device). In fliGHt, treatment-experienced subjects switched to lonapegsomatropin from daily somatropin (Group C; vial/syringe). Lonapegsomatropin was administered at a starting dose of 0.24 mg hGH/kg/wk. Upon entry into enliGHten, all subjects received lonapegsomatropin (vial/syringe), and switched to TransCon hGH Auto-Injector pending regional availability. A total of 298 (of 303 eligible) subjects continued into enliGHten (A: n = 103; B: n = 55; C: n = 140). The mean dose remained approximately 0.24 mg hGH/kg/wk for Group A/B at Week 104 and was 0.20 mg hGH/kg/wk for Group C at Week 78. Subjects starting heiGHt on daily somatropin who switched to lonapegsomatropin continued to approach their average parental height, with height standard deviation score (SDS) improving from -3.0 at baseline to -1.52 at Week 104. Subjects from the fliGHt Trial also continued to approach their average parental height, with height SDS improving from −1.42 at fliGHt baseline to −0.69 at Week 78. Observed Mean annualized height velocity (AHV) at Week 78 was 8.4 cm/year. heiGHt subjects starting lonapegsomatropin continued to approach their average parental height, with height SDS improving from -2.89 at baseline to -1.37 at Week 104. As of the data cut (01 June 2020), 5 subjects have met or exceeded their target height and 11 subjects have grown to within 2 cm of their target height. Mean (SD) average IGF-1 SDS remained stable and generally within the expected range for all groups (Week 104, A: 0.95 [1.22], B: 1.04 [1.25]; Week 78, C: 1.81 [1.08]). Across all phase 3 trials, lonapegsomatropin produced a dose linear IGF-1 response. With lonapegsomatropin treatment, the adverse event (AE) profile remained consistent with what was observed in the parent trials, with no new safety signals, comparable tolerability, and a mean body mass index (BMI) SDS approaching zero. Across the broad population of the phase 3 program, subjects treated with lonapegsomatropin for up to 2 years continued to approach their average parental height, with a safety profile comparable to daily somatropin, including a similar AE profile, tolerability, and stable BMI.

Volume 94

59th Annual ESPE (ESPE 2021 Online)

22 Sep 2021 - 26 Sep 2021

European Society for Paediatric Endocrinology 

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