Ascendis Reports Positive Interim Data from Achondroplasia Trial

Ascendis Pharma shared promising 26-week interim results from its Phase II COACH trial evaluating a weekly combination of TransCon CNP (navepegritide) and TransCon hGH (lonapegsomatropin) in children with achondroplasia. The treatment improved growth velocity, height Z-scores, and body proportionality in both TransCon CNP-naive and previously treated patients aged 2–11. Bone age remained aligned with chronological age.

The safety profile of the combo matched that of individual therapies. The open-label trial includes two cohorts and administers TransCon CNP at 100 µg/kg/week and TransCon hGH at 0.30 mg/kg/week. A Phase III trial is expected to start this quarter, with 52-week data due in Q4.

TransCon CNP, a long-acting CNP prodrug, is under FDA priority review. TransCon hGH, approved as Skytrofa, is already used for pediatric growth hormone deficiency.

18-06-2025