Ascendis Pharma announces data from ApproaCH trial of TransCon CNP

Ascendis Pharma announced topline data from the pivotal double-blind placebo-controlled ApproaCH Trial of TransCon CNP, which included 84 children with achondroplasia randomized 2:1. TransCon CNP is an investigational prodrug of C-type natriuretic peptide administered once weekly and designed to provide sustained release and continuous exposure of active CNP. In the trial, children treated with once-weekly TransCon CNP demonstrated annualized growth velocity superior to placebo. TransCon CNP also demonstrated statistically significant improvements in other growth parameters, including height Z-score and change from baseline AGV. Primary Endpoint For the primary endpoint of AGV at Week 52, children treated with TransCon CNP demonstrated an LS mean AGV of 5.89 cm/year compared to 4.41 cm/year in the placebo arm, an LS mean difference of 1.49 cm/year. Children aged 2 to less than5 years, treated with TransCon CNP demonstrated a change from baseline AGV at Week 52 of 1.57 cm/year compared to 0.43 cm/year in the placebo arm. For the secondary endpoint of change in ACH Height Z-score, children treated with TransCon CNP demonstrated an LS mean change from baseline ACH Height Z-score of 0.30 compared to 0.01 in the placebo arm