Ascendis Pharma (ASND) Study result presentation summary

Product overview and mechanism

• TransCon CNP is a once-weekly prodrug of C-type natriuretic peptide designed for continuous exposure, counteracting FGFR3 signaling in achondroplasia.

• Active CNP released from TransCon CNP is identical to endogenous CNP and binds to NPR-B throughout the body.

• Approved by the FDA as YUVIWEL for increasing linear growth in children aged 2 years and older with achondroplasia.

Clinical trial design and population

• The ApproaCH trial enrolled treatment-naïve children aged 2–11 years with achondroplasia, randomized 2:1 to TransCon CNP or placebo.

• Double-blind period followed by open-label extension, with radiographs and growth assessments at baseline, Week 52, and Week 104.

• Baseline characteristics were balanced between groups, with mean ages around 5.6–6.0 years.

Efficacy outcomes

• TransCon CNP showed superior annualized growth velocity (AGV) at Week 52 compared to placebo (LS mean difference 1.49 cm/year, p<0.0001).

• Improvements in AGV and height Z-score were maintained through Week 104 in all children treated with TransCon CNP.