Norditropin (somatropin) / Novo Nordisk - LARVOL DELTA

Evaluating the Efficacy and Safety of GB08 Injection in Pediatric Patients With Growth Hormone Deficiency (clinicaltrials.gov) - P2/3 | N=268 | Not yet recruiting | Sponsor: Shenzhen Kexing Pharmaceutical Co., Ltd.

New P2/3 trial • Endocrine Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Pediatric) • Pediatrics • IGF1

Growth Hormone Replacement in Veterans With GWI and AGHD (GWIT) (clinicaltrials.gov) - P2 | N=20 | Recruiting | Sponsor: Baylor College of Medicine | Trial completion date: Sep 2025 ➔ Jul 2026 | Trial primary completion date: May 2025 ➔ Jul 2026

Trial completion date • Trial primary completion date • Endocrine Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Adult)

A Clinical Study of GenSci134 in Healthy Male Adults (clinicaltrials.gov) - P1 | N=64 | Recruiting | Sponsor: Changchun GeneScience Pharmaceutical Co., Ltd. | Not yet recruiting ➔ Recruiting

Enrollment open

Phase 1 Study and Population PK/PD Modeling of Long-Acting Growth Hormone, GB08, to Guide Pediatric Dosing. (PubMed, Eur J Endocrinol) - "GB08 provides notable advantages over traditional therapies, like short-acting GH, by enhancing treatment adherence and offering a potentially safer alternative. The findings from this study will guide dosing strategies for Phase 2 trial in children with GHD."

Clinical • Journal • P1 data • PK/PD data • Endocrine Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Adult) • Growth Hormone Deficiency (Pediatric) • Pediatrics

A Clinical Study of GenSci134 in Healthy Male Adults (clinicaltrials.gov) - P1 | N=64 | Not yet recruiting | Sponsor: Changchun GeneScience Pharmaceutical Co., Ltd.

New P1 trial

Efficacy, safety, and insulin-like growth factor I of weekly somapacitan in children with growth hormone deficiency: 3-year results from REAL4. (PubMed, Eur J Endocrinol) - P3 | "Sustained efficacy and tolerability were observed for continuous somapacitan treatment for 3 years, and for 2 years after the switching from daily GH treatment. HSDS in both groups was approaching mean mid-parental HSDS."

Clinical • Journal • Endocrine Disorders • Growth Hormone Deficiency • IGF1

Circulating IGF-1 Levels and Trends in Response to Daily and Weekly GH Therapies: A Comparative Analysis (ESPE-ESE 2025) - " Data were synthesized from eight studies analyzing IGF-1 response to various GH therapies, including daily rGH and weekly long-acting formulations such as somapacitan, PEG-rhGH, TransCon GH, and Nutropin Depot...Daily GH Therapies: Daily rGH therapy (e.g., Genotropin, Norditropin) resulted in more consistent IGF-1 levels with minor daily fluctuations, providing a steady profile throughout the week-(Lundberg et al., 2018)... Both weekly and daily GH therapies effectively elevate IGF-1 levels, with distinct advantages and limitations. Weekly formulations optimize patient adherence and long-term exposure, whereas daily rGH ensures consistent IGF-1 profiles. Table: IGF-1 Trends and Patterns Between Daily Vs Long-Acting-GH Therapies IGF-1 Response GH Therapy Type Key IGF-1 Trends IGF-1 Patterns Consistency Variable-Timing, Stable Higher consistency across Daily GH Fluctuates-Daily Long-Term days Dose-Dependent, Higher overall AUC due to Long-Acting GH Weekly-Peaks and..."

IGF1

Growth hormone therapy demonstrates no clinical impact on thyroid function in children with growth hormone deficiency (ESPE-ESE 2025) - "Methods Three randomized, multinational, open-labeled parallel-group phase 2 (REAL3, n=59) and phase 3 (REAL4, n=200; and REAL6, n=110) trials investigated efficacy and safety of once-weekly somapacitan compared to once-daily GH (Norditropin®, Novo Nordisk) administration in treatment-naïve, prepubertal children with GHD. Clinicians can be reassured that GH therapy does not pose a significant risk of inducing hypothyroidism. Cases with central GHD warrants special attention."

Clinical • Late-breaking abstract • Endocrine Disorders • Growth Hormone Deficiency

Evaluation of features for a digital application for growth hormone therapy using perceptions gathered from patients, caregivers and healthcare professionals (ESPE-ESE 2025) - "Insights from patients, caregivers and healthcare professionals (HCPs) were gathered to inform the development of MySoConnect® for Sogroya® (Novo Nordisk Health Care AG)...Patients were current users of the Norditropin® (somatropin) FlexPro® pen (Novo Nordisk Health Care AG)...HCPs were strongly in favour of adding an automated dose-logging feature, which corresponds to a connectivity element between the injection pen and the app, to encourage app use. Insights gathered from patients, caregivers and HCPs indicate that the use of an app for GHT may support adherence to treatment, with features such as tracking height gain over time, setting reminders and accessing instructional manuals and other relevant supporting information considered beneficial."

Clinical • Cognitive Disorders

Efficacy and safety of somapacitan versus daily growth hormone at 52 weeks in patients with growth hormone deficiency: pooled analysis of the randomised REAL 3, REAL 4 and REAL 6 clinical trials (ESPE-ESE 2025) - P2, P3 | "We present outcomes from a pooled analysis of data from three clinical trials (REAL 3 [NCT02616562], REAL 4 [NCT03811535] and REAL 6 [NCT04970654]) assessing the efficacy and safety of somapacitan versus daily GH (Norditropin®, Novo Nordisk). Outcomes from a pooled analysis of REAL 3, REAL 4 and REAL 6 demonstrate similar efficacy of somapacitan compared to daily GH over 52 weeks of treatment, with comparable safety and mean IGF-I SDS change in treatment-naïve children with GHD."

Retrospective data • Endocrine Disorders • Growth Hormone Deficiency • IGF1

Long-term efficacy and safety of once-weekly somapacitan in children with growth hormone deficiency: 7-year results from the randomised REAL 3 trial (ESPE-ESE 2025) - P2 | "REAL 3 (NCT02616562) is a phase 2, randomised, multinational, open-label, active-controlled trial designed to determine treatment efficacy and safety of somapacitan, a long-acting growth hormone (GH) derivative, vs daily GH (Norditropin®, Novo Nordisk). Similar responses were observed in patients treated with somapacitan throughout the trial and in those who switched from daily GH after 156 weeks. No side effects for somapacitan outside the known safety profile of daily GH were identified."

Clinical • Endocrine Disorders • Growth Hormone Deficiency • IGF1

Somapacitan is Effective and Well Tolerated in Chinese Children with Growth Hormone Deficiency: a Randomised Controlled Phase 3 Study. (PubMed, Horm Res Paediatr) - P3 | "Efficacy and safety profiles were comparable for Chinese children with GHD treated with somapacitan or daily GH. Both treatments similarly reduced disease burden, while treatment burden was reduced with somapacitan."

Journal • P3 data • Endocrine Disorders • Growth Hormone Deficiency • Pain • IGF1

Usability and Preference Evaluation of the Somapacitan Pen-Injector and Lonapegsomatropin Autoinjector: Results of a US-Based Simulated-Use Study with Adolescent Patients and Caregivers. (PubMed, Patient Prefer Adherence) - "The somapacitan device was preferred over the lonapegsomatropin device among adolescents with GRDs and caregivers of individuals with GRDs. Individuals receiving GH treatment via the somapacitan device may be more likely to have higher treatment adherence than those using the lonapegsomatropin device."

Journal

Skeletal Muscle Effects of GH in Boys (clinicaltrials.gov) - P=N/A | N=45 | Active, not recruiting | Sponsor: Nemours Children's Clinic | Trial primary completion date: Mar 2025 ➔ Jul 2025

Trial primary completion date • Endocrine Disorders • Growth Hormone Deficiency • Idiopathic Short Stature

Growth Response to Weekly Somapacitan Therapy in Children With GH Deficiency Is Related to GH Thresholds in GH Stimulation Testing. (PubMed, J Endocr Soc) - "Mean change in IGF-I SDS ranged from 3.13 to 3.01, 2.11 to 1.96, and 1.87 to 2.26 at week 52 and from 2.81 to 2.11, 1.85 to 1.62, and 1.28 to 1.71 at week 104 for the 3 GH peak concentrations. Patients with GHD in the ≤3 μg/L GH peak concentration group had greater HV and greater changes in height SDS and IGF-I SDS from baseline."

Journal • Endocrine Disorders • Growth Hormone Deficiency • IGF1

Adult Growth Hormone Deficiency (AGHD) and Outcomes (NordiNet and ANSWER). (PubMed, J Endocr Soc) - "Here we have analyzed the long-term effectiveness and safety of short-acting GH replacement therapy (GHRT) in treatment naïve and nonnaïve patients with AGHD using real-world data from the NordiNet® International Outcome Study and American Norditropin® Studies: Web Enabled Research Program...Mean IGF-I SDS levels reached a normal range (-2 to 2) in ≥80% of patients from all groups in the effectiveness analysis set by year 2. Together with previous reports of older patients, these results support the real-world safety and efficacy of short-acting GHRT among all ages of patients with AGHD."

Journal • Endocrine Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Adult) • Rare Diseases • IGF1

Efficacy in Japanese adults with growth hormone deficiency receiving weekly somapacitan or daily growth hormone: results from phase 3 REAL 1 trial. (PubMed, Front Endocrinol (Lausanne)) - P3 | "Participants received once-weekly somapacitan or daily GH (Norditropin®), both administered subcutaneously. Somapacitan provides an effective alternative to daily GH in AGHD. http://www.clinicaltrials.gov, identifier NCT02229851."

Clinical • Journal • P3 data • Endocrine Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Adult)

REAL 8: A Research Study to Compare Somapacitan Once a Week With Norditropin® Once a Day in Children Who Need Help to Grow (clinicaltrials.gov) - P3 | N=399 | Active, not recruiting | Sponsor: Novo Nordisk A/S | Recruiting ➔ Active, not recruiting

Enrollment closed • Pan tumor • Genetic Disorders • Idiopathic Short Stature • Small for Gestational Age • Turners Syndrome • IGF1

Pharmacodynamic Endpoints After Once-Weekly Somapacitan in Children With GHD: 3-year results from REAL4 phase 3 study (ESPE 2024) - P3 | "The trial randomised (2:1) 200 GH-treatment-naïve, prepubertal children with GHD to once-weekly somapacitan (0.16 mg/kg; n=132) or once-daily GH (Norditropin® 0.034 mg/kg; n=68) for 52 weeks. IGF-I SDS >2.0 at two consecutive visits occurred in 2 (1.7%) and 1 (1.7%), respectively, with no observed trend in the amount or type of AEs. In conclusion, after three years of somapacitan and two years of somapacitan following switch from daily GH, IGF-I SDS, IGFBP-3 SDS, and IGF-I/IGFBP-3 molar ratio were similar between groups, and mean IGF-I SDS and IGFBP-3 SDS were within normal range."

Clinical • Late-breaking abstract • P3 data • PK/PD data • IGF1 • IGFBP3

Skeletal Muscle Effects of GH in Boys (clinicaltrials.gov) - P=N/A | N=45 | Active, not recruiting | Sponsor: Nemours Children's Clinic | Trial primary completion date: Oct 2024 ➔ Mar 2025

Trial primary completion date • Endocrine Disorders • Growth Hormone Deficiency • Idiopathic Short Stature

Phase 2 Study of LUM-201 in Children With Growth Hormone Deficiency (OraGrowtH210 Trial) (clinicaltrials.gov) - P2 | N=82 | Completed | Sponsor: Lumos Pharma | Active, not recruiting ➔ Completed

Trial completion • Endocrine Disorders • Growth Hormone Deficiency

Investigating Efficacy and Safety of Once-weekly NNC0195-0092 (Somapacitan) Treatment Compared to Daily Growth Hormone Treatment (Norditropin® FlexPro®) in Growth Hormone Treatment naïve Pre-pubertal Children With Growth Hormone Deficiency (clinicaltrials.gov) - P2 | N=74 | Completed | Sponsor: Novo Nordisk A/S | Recruiting ➔ Completed

Trial completion • Endocrine Disorders • Growth Hormone Deficiency • IGF1

Sustained Efficacy of Weekly Somapacitan in Children With GHD: 3-year Results From Phase 3 REAL4 Study (AAP-NCE 2024) - P3 | "Participants received either once-weekly somapacitan (0.16 mg/kg; n=132) or once-daily GH (Norditropin® 0.034 mg/kg; n=68) for 52 weeks. Once-weekly somapacitan showed sustained efficacy and tolerability for three years, as well as for two years following the switching from daily GH treatment in this pivotal phase 3 REAL4 trial. The safety profile of somapacitan was similar to the well-known safety profile of daily GH."

Clinical • P3 data • Growth Hormone Deficiency • Growth Hormone Deficiency (Adult)

REAL 8: A Research Study to Compare Somapacitan Once a Week With Norditropin® Once a Day in Children Who Need Help to Grow (clinicaltrials.gov) - P3 | N=399 | Recruiting | Sponsor: Novo Nordisk A/S | Trial completion date: May 2027 ➔ Oct 2027

Trial completion date • Genetic Disorders • Idiopathic Short Stature • Small for Gestational Age • Turners Syndrome • IGF1

Efficacy and Safety of Somapacitan Relative to Somatrogon and Lonapegsomatropin in Pediatric Growth Hormone Deficiency: Systematic Literature Review and Network Meta-analysis. (PubMed, Adv Ther) - "No efficacy and safety differences were identified in comparisons of once weekly somapacitan versus somatrogon and lonapegsomatropin, as well as daily GH. All treatments were generally well tolerated, with the exception of observed injection site pain for somatrogon."

Journal • Retrospective data • Review • Endocrine Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Pediatric) • Pain • Pediatrics