Ascendis reports positive 52-week trial data for achondroplasia treatment

Ascendis Pharma A/S (NASDAQ: ASND) announced results from a 52-week Phase 2 trial evaluating combination therapy with TransCon CNP and TransCon hGH in children with achondroplasia, a rare genetic condition affecting bone growth.

The COACH trial included 21 children aged 2 to 11 years, divided into two cohorts: 12 treatment-naive children and 9 children who had previously received TransCon CNP therapy. The study tested once-weekly doses of TransCon CNP at 100 µg/kg/week combined with TransCon hGH at 0.30 mg/kg/week.

In the treatment-naive cohort, mean annualized growth velocity reached 8.80 cm/year with an improvement in achondroplasia height Z-score of +1.02 over 52 weeks. The previously treated cohort showed mean annualized growth velocity of 8.42 cm/year, representing an increase of 3.28 cm/year from baseline, with Z-score improvement of +0.86.

Children in both groups exceeded the 97th percentile growth velocity of average-stature children. The combination therapy also showed improvements in body proportionality and arm span measurements. All 21 children completed the 52-week treatment period and continue in the trial.

The treatment was generally well-tolerated with mild treatment-emergent adverse events consistent with individual monotherapies. Safety profiles remained comparable to standalone TransCon CNP and TransCon hGH treatments.

TransCon CNP is under Priority Review by the FDA with a target action date of February 28, 2026. The company submitted a Phase 3 trial protocol to the FDA in the fourth quarter of 2025.

Achondroplasia affects more than 250,000 people worldwide and causes various medical complications beyond growth issues, including spinal abnormalities, breathing difficulties, and chronic pain throughout a patient's life.

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