Ascendis Pharma (ASND) Study result presentation summary

Product overview and mechanism

• Navepegritide is a once-weekly prodrug of C-type natriuretic peptide (CNP) designed for continuous exposure, binding NPR-B to counteract FGFR3 signaling in achondroplasia.

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

Trial design and population

• Randomized, double-blind trial in treatment-naïve children aged 2–11 years, followed by an open-label extension (OLE) to Week 104.

• Subgroup analysis focused on children aged 5 years or older at enrollment.

• Baseline demographics were balanced between navepegritide and placebo groups, including age, sex, and height.

Efficacy results

• At Week 52, navepegritide significantly increased annualized growth velocity (AGV) compared to placebo: +1.49 cm/year in all children and +1.78 cm/year in those aged ≥5 years (both p<0.0001).

• AGV improvements were maintained through Week 104 in both the overall and ≥5-year-old subgroups.

• Height Z-scores (achondroplasia-specific and CDC-based) continued to improve through Week 104, with greater gains in children aged ≥5 years.