Ascendis Pharma (ASND) Study update presentation summary

Medical complications and treatment goals

• Achondroplasia is associated with complications such as spinal stenosis, hearing loss, sleep-disordered breathing, and skeletal dysplasia.

• Major treatment goals include reducing complications, increasing height, promoting proportional growth, and improving quality of life.

TransCon CNP mechanism and approval

• TransCon CNP is a once-weekly prodrug designed to provide continuous CNP exposure, counteracting FGFR3 signaling.

• Approved by the FDA to increase linear growth in children 2 years and older with achondroplasia and open epiphyses.

COACH trial: combination therapy efficacy and safety

• Combination of TransCon CNP + TransCon hGH in children aged 2–11 showed annualized growth velocity (AGV) at or above the 97th percentile for average-stature children at Week 78.

• Sustained increases in height Z-scores indicated a tripling of efficacy compared to monotherapy.

• Body proportionality improved through Week 78, aligning with height gains.

• Safety profile was consistent with monotherapies, with most adverse events mild or moderate and no discontinuations due to adverse events.

• 100% of children completed 78 weeks of treatment and remained in the trial.