Ascendis Pharma A/S (ASND) announced positive topline 52-week results from its Phase 2 trial, dubbed New InsiGHTS, evaluating once-weekly TransCon hGH (Lonapegsomatropin) versus daily somatropin in prepubertal children with Turner syndrome.
Turner syndrome is one of the most common congenital sex chromosomal conditions in females and is associated with clinical manifestations, including short stature and ovarian insufficiency. Turner syndrome has an estimated prevalence of 1 out of every 2,000 to 2,500 live female births, according to Ascendis Pharma.
TransCon hGH (Lonapegsomatropin) is an investigational treatment for Growth Hormone Deficiency (GHD) and is FDA-approved for paediatric and adult GHD, and approved in other territories for paediatric GHD. TransCon hGH is also being investigated in adult GHD and Turner syndrome.
New InsiGHTS phase 2 trial is a randomised, open-label, active-controlled trial in the U.S. investigating the safety, tolerability, and efficacy of once-weekly TransCon hGH compared with daily somatropin in prepubertal children with Turner syndrome.
In the New InsiGHTS trial, 49 children with Turner syndrome aged 1 to 10 years old were randomised and dosed either with TransCon hGH or daily somatropin.The mean dose for TransCon hGH was 0.22 mg/kg/week, while the mean dose for the daily somatropin cohort was 0.29 mg/kg/week at week 52.
The key findings at week 52 include,
-Annualised height velocity (AHV) similar to daily somatropin, independent of starting dose, with an LS mean AHV of 9.05 cm/year for all TransCon hGH-treated children, compared to 9.04 cm/year for those treated with daily somatropin.
-TransCon hGH demonstrated a safety and tolerability profile similar to daily somatropin through follow-up of up to 143 weeks.
-Adverse events (AEs) were mild to moderate in severity, with no AEs leading to discontinuation of the study drug.
-There were no occurrences of slipped capital femoral epiphysis (SCFE), consistent with the low rate of occurrence (<1%) in long-term safety data of daily somatropin use in Turner syndrome from published literature.
Jan Mikkelsen, Ascendis Pharma's President and Chief Executive Officer, said, "These new results demonstrated safety and efficacy comparable to daily growth hormone with up to 143 weeks of follow-up".
TransCon hGH is being further studied in a recently initiated Phase 3 HighLiGHts basket trial to support label expansion across multiple indications, including Turner syndrome, Idiopathic Short Stature, SHOX Deficiency, and Small for Gestational Age.
ASND has traded between $124.06 and $248.60 in the last year. The stock closed Tuesday's trade at $228.06, down 1.28%.
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