COPENHAGEN, Denmark — Ascendis Pharma A/S (Nasdaq: ASND) today announced positive Week 52 topline results from New InsiGHTS, its Phase 2 randomized, open-label, active-controlled trial in the U.S. investigating the safety, tolerability, and efficacy of once-weekly TransCon hGH (lonapegsomatropin; U.S. FDA-approved for pediatric and adult growth hormone deficiency (GHD) and approved in other territories for pediatric GHD) compared to daily somatropin in prepubertal children with Turner syndrome.
New InsiGHTS randomized and dosed 49 children with Turner syndrome aged 1 to 10 years old. They were treated with either TransCon hGH or daily somatropin. Doses were subsequently individualized based on IGF-1.
- At Week 52, children treated with TransCon hGH demonstrated improved annualized 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.
- At Week 52, 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.
- In the trial, 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 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 literature1,2.
“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,” said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer.
About Turner Syndrome
Turner syndrome is the most common congenital sex chromosomal condition in females, with an estimated prevalence of 1 out of every 2,000 to 2,500 live female births. Short stature, associated with short stature homeobox-containing gene (SHOX) haploinsufficiency, is the most common clinical feature of Turner syndrome. Clinical manifestations of Turner syndrome affect multiple organ systems and are associated with significant and potentially life-threatening complications including cardiovascular disease, ovarian dysfunction, endocrine disease, renal malformation, liver disease, sensorineural hearing loss, and varied neuropsychological manifestations.
About Ascendis Pharma A/S
Ascendis Pharma is a global biopharmaceutical company focused on applying our innovative TransCon technology platform to make a meaningful difference for patients. Guided by our core values of Patients, Science, and Passion, and following our algorithm for product innovation, we apply TransCon to develop new therapies that demonstrate best-in-class potential to address unmet medical needs. Ascendis is headquartered in Copenhagen, Denmark, and has additional facilities in Europe and the United States. Please visit ascendispharma.com to learn more.
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