Ascendis Pharma (ASND) Study update presentation summary

Key clinical findings

• Combination therapy with TransCon CNP and TransCon hGH led to unprecedented improvements in arm span, with gains of +9.4 cm in treatment-naïve and +7.9 cm in previously treated cohorts, surpassing typical surgical outcomes.

• Enhanced spinal canal dimensions were observed with combination therapy, with an average interpedicular distance (IPD) improvement of +1.7 mm versus +0.6 mm for monotherapy.

• Combination therapy resulted in improved lower limb alignment, with a mean tibial femoral angle (TFA) change of -3.0 degrees, compared to -1.32 degrees for monotherapy.

• Mean annualized growth velocity at Week 52 exceeded the 97th percentile of average stature children, with no compromise in safety or acceleration of bone age.

• All children completed 52 weeks of treatment and remain on therapy, indicating strong tolerability and adherence.

Study design and population

• The COACH trial included children aged 2–11 years with achondroplasia, divided into treatment-naïve and previously treated cohorts.

• Primary efficacy endpoint was annualized growth velocity at Week 52; secondary endpoints included height Z-score, body proportionality, radiological measures, and safety.

• The trial population was representative of children with achondroplasia, with baseline characteristics well documented for both cohorts.

• Treatment consisted of weekly TransCon CNP 100 µg/kg and TransCon hGH 0.30 mg/kg.

• Safety endpoints focused on treatment-emergent adverse events, including injection site reactions.

Medical context and goals

• Achondroplasia is associated with complications such as leg bowing, shortened arm span, spinal abnormalities, and restricted mobility.

• Major treatment goals are to decrease incidence and severity of complications, increase height, promote proportional growth, and improve quality of life.