Ascendis Pharma reported that its Phase 2 New InsiGHTS trial of the once‑weekly TransCon hGH (lonapegsomatropin) in pre‑pubertal children with Turner syndrome produced Week 52 topline data from 49 patients. The trial’s primary endpoint, annualized height velocity, was 9.05 cm per year for TransCon hGH, virtually identical to the 9.04 cm per year observed with daily somatropin, and no cases of slipped capital femoral epiphysis were recorded.
The comparable efficacy and safety profile of a once‑weekly therapy versus a daily injection is a key differentiator that could improve patient adherence and quality of life. These results will be incorporated into Ascendis’s upcoming Phase 3 HighLiGHts basket trial, which aims to expand labeling for TransCon hGH across multiple short‑stature indications.
Jan Mikkelsen, Ascendis’s President and CEO, said, "These new results demonstrated safety and efficacy comparable to daily growth hormone with up to 143 weeks of follow‑up, and support the potential of TransCon hGH as a differentiated therapy for short stature in the setting of growth hormone sufficiency and is being further studied in our recently initiated Phase 3 HighLiGHts basket trial to support label expansion."
The New InsiGHTS trial, which enrolled children aged 1 to 10 years and had a primary completion date of October 18, 2024, represents a significant milestone for Ascendis’s rare‑endocrine portfolio. By showing that a once‑weekly prodrug can match the growth velocity of daily somatropin without additional safety signals, the company strengthens its competitive position in the growth‑hormone market and sets the stage for broader regulatory submissions and potential market expansion.
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