TransCon hGH Meets Primary Endpoint in Turner Syndrome Trial
Key Insights
Ascendis Pharma's New InsiGHTS Phase 2 trial demonstrated that TransCon hGH achieved its primary objective in children with Turner syndrome (search) at Week 26.
The study showed comparable safety and tolerability between once-weekly TransCon hGH and daily somatropin, with no discontinuations related to the study drug.
Annualized height velocity in children treated with TransCon hGH was similar to that observed in those treated with daily somatropin.
Ascendis Pharma A/S announced positive topline results from the New InsiGHTS trial, a Phase 2 randomized, open-label, active-controlled study evaluating TransCon hGH (lonapegsomatropin) in prepubertal children with Turner syndrome (search). The trial, conducted in the U.S., compared the safety, tolerability, and efficacy of once-weekly TransCon hGH to daily somatropin.
The New InsiGHTS trial randomized and dosed 49 children with Turner syndrome (search), aged 1 to 10 years, into four treatment groups. These groups included three different starting doses of TransCon hGH (0.24, 0.30, or 0.36 mg/kg/week) and a daily somatropin comparator group (0.35 mg/kg/week). Doses were individualized based on IGF-1 levels.
Comparable Growth with TransCon hGH
The primary endpoint of annualized height velocity and the secondary endpoint of change from baseline in height SDS were met, with children treated with TransCon hGH showing similar growth compared to those treated with daily somatropin at Week 26, irrespective of the starting dose.
Safety and Tolerability
TransCon hGH was generally safe and well-tolerated, with a safety profile comparable to daily somatropin. There were no discontinuations due to the study drug.
Future Plans
"Week 26 topline data from the New InsiGHTS Trial showed that once-weekly TransCon hGH provided comparable safety and endocrine benefits to those seen with the established use of daily somatropin in Turner syndrome (search)," said Jan Mikkelsen, Ascendis Pharma's President and Chief Executive Officer. "These positive results lay the foundation for our planned basket trial in 2025 to support label expansion."
Ascendis Pharma anticipates the Week 52 results for lonapegsomatropin-treated children compared to the daily somatropin group in 2025.
About Turner Syndrome
Turner syndrome (search) affects approximately 1 in every 2,000 to 2,500 live female births. Short stature, linked to SHOX haploinsufficiency, is a common characteristic. The condition can also lead to cardiovascular, ovarian, endocrine, renal, liver, auditory, and neuropsychological complications.