COPENHAGEN, Denmark, March 17, 2026
Ascendis Pharma A/S announced positive Week 52 topline results from its Phase 2 New InsiGHTS clinical trial, demonstrating that once-weekly TransCon® hGH (lonapegsomatropin) achieved comparable efficacy and safety to daily somatropin in children with Turner syndrome, a significant milestone in advancing long-acting growth hormone therapies. The randomized, open-label, active-controlled trial evaluated 49 pediatric patients aged 1 to 10 years, highlighting the potential of a less frequent dosing regimen to improve treatment adherence while maintaining clinical outcomes.
Comparable Efficacy Demonstrated at Week 52
The study results showed that annualized height velocity (AHV) reached 9.05 cm/year in patients treated with TransCon hGH, closely matching 9.04 cm/year observed in the daily somatropin group, confirming non-inferior efficacy across dosing strategies. Importantly, this outcome was consistent regardless of initial dosing variations, reinforcing the robustness of the therapy’s performance across patient subsets. The ability to achieve equivalent growth outcomes with a once-weekly regimen represents a major advancement in pediatric endocrinology, potentially reducing treatment burden for patients and caregivers.
Additionally, dose optimization based on IGF-1 levels enabled personalized treatment approaches, supporting precision medicine strategies in growth hormone therapy. This level of individualized dosing is particularly relevant in regulated clinical settings where patient-specific response and safety monitoring are critical components of Good Clinical Practice (GCP).
Strong Safety and Tolerability Profile
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Beyond efficacy, the trial demonstrated a favorable safety and tolerability profile for TransCon hGH, comparable to the established daily somatropin standard. Adverse events reported were primarily mild to moderate, with no discontinuations due to safety concerns. Notably, there were no cases of slipped capital femoral epiphysis (SCFE), a rare but serious complication associated with growth hormone therapy, aligning with long-term safety expectations from existing treatments.
The safety data extended up to 143 weeks of follow-up, providing a comprehensive assessment of long-term tolerability. This reinforces confidence among clinicians and regulatory stakeholders regarding the therapy’s risk-benefit profile. Such data are crucial for regulatory submissions and label expansion strategies, especially in pediatric populations where safety thresholds are stringent.
Advancing Toward Late-Stage Clinical Development
The positive Phase 2 findings support the continued development of TransCon hGH in the Phase 3 HighLiGHts basket trial, aimed at expanding its therapeutic indications. Turner syndrome, a genetic condition affecting approximately 1 in 2,000 to 2,500 female births, is characterized by short stature and multiple systemic complications, making effective and patient-friendly treatments essential.
Ascendis Pharma’s TransCon technology platform, which enables sustained drug release, is central to this innovation, offering a differentiated approach to hormone therapy. The transition to Phase 3 trials marks a critical step toward regulatory approval and commercialization, positioning the therapy as a potential best-in-class option in growth disorders.
From a cGxP perspective, this development holds significant relevance across GCP (clinical trials), GMP (manufacturing of long-acting biologics), and regulatory compliance frameworks, as the therapy progresses through late-stage development. The results not only validate the scientific approach but also underscore the importance of innovative delivery systems in improving patient outcomes and adherence.
The positive Week 52 data from the New InsiGHTS trial represent a major advancement in the treatment of Turner syndrome, demonstrating that once-weekly TransCon hGH can deliver comparable efficacy and safety to daily therapy while offering improved convenience. As Ascendis Pharma advances into Phase 3 development, this therapy has the potential to reshape growth hormone treatment paradigms and enhance patient quality of life, making it a highly relevant development for stakeholders across clinical, regulatory, and pharmaceutical domains.
Source: ascendis pharma press release
