Ascendis Pharma (ASND) Study result presentation summary

Mechanism and product overview

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

• FDA-approved for increasing linear growth in children aged 2+ with achondroplasia and open epiphyses.

ApproaCH trial design and participant characteristics

• Randomized, double-blind, placebo-controlled trial in children aged 2–11, followed by an open-label extension (OLE).

• Primary endpoint: annualized growth velocity (AGV) at Week 52; secondary endpoints included height Z-scores and safety.

• At OLE start, mean age was 6.8 years, with balanced demographics and baseline growth characteristics.

Efficacy results through 104 weeks

• Navepegritide showed superior AGV at Week 52 (LS mean difference 1.49 cm/year, p<0.0001).

• Children continuing navepegritide maintained AGV through 104 weeks; those switching from placebo saw comparable gains.

• Achondroplasia-specific and CDC-based height Z-scores improved consistently through OLE.

• Numeric improvement in upper-to-lower body segment ratio indicated promotion of proportional growth.