Ascendis Pharma reports positive Turner syndrome trial results for TransCon hGH

Ascendis Pharma A/S (NASDAQ: ASND) announced positive results from its Phase 2 New InsiGHTS trial evaluating once-weekly TransCon hGH compared to daily somatropin in children with Turner syndrome.

The randomized, open-label trial enrolled 49 prepubertal children with Turner syndrome aged 1 to 10 years. At week 52, children treated with TransCon hGH achieved an annualized height velocity of 9.05 cm/year compared to 9.04 cm/year for those receiving daily somatropin.

The mean dose for TransCon hGH was 0.22 mg/kg/week, while the daily somatropin group received 0.29 mg/kg/week. Both treatments were individualized based on IGF-1 levels.

TransCon hGH demonstrated a safety profile comparable to daily somatropin through up to 143 weeks of follow-up. Adverse events were mild to moderate in severity, with no events leading to treatment discontinuation. No cases of slipped capital femoral epiphysis occurred in either group.

"These new results demonstrated safety and efficacy comparable to daily growth hormone with up to 143 weeks of follow-up," said Jan Mikkelsen, Ascendis Pharma's President and Chief Executive Officer.

TransCon hGH is currently approved by the U.S. FDA for pediatric and adult growth hormone deficiency and approved in other territories for pediatric growth hormone deficiency. The company is conducting a Phase 3 HighLiGHts basket trial to support potential label expansion.

Turner syndrome affects approximately 1 in 2,000 to 2,500 live female births, with short stature being the most common clinical feature due to SHOX gene haploinsufficiency.

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