Ascendis Pharma announces topline results from ACcomplisH trial

Ascendis Pharma A/S announced topline results from the ACcomplisH Trial, its Phase 2 randomized, double-blind, placebo-controlled, dose-escalation trial evaluating the safety and efficacy of once-weekly TransCon CNP compared to placebo in children with achondroplasia aged 2 to 10 years old. TransCon CNP is an investigational long-acting prodrug of C-type natriuretic peptide, designed to provide continuous exposure of CNP at safe, therapeutic levels via a single, weekly subcutaneous dose. The ACcomplisH Trial evaluated 57 children with achondroplasia aged 2 to 10 years old, randomized in a 3:1 ratio to receive either sequential ascending doses of once-weekly TransCon CNP or placebo for 52 weeks. All 57 randomized children completed the blinded portion of ACcomplisH and are currently continuing in the open label extension at the 100 microgram/kg/week dose. The trial met its primary objectives, demonstrating that TransCon CNP at 100 microgram/kg/week was superior to placebo on the primary efficacy endpoint of AHV at 52 weeks. TransCon CNP demonstrated a consistent dose-response in AHV across the four dose groups. Mean improvements in AHV for TransCon CNP-treated patients were consistent across age groups less than 5 years and greater than 5 years, with dose response established. TransCon CNP at 100 microgram/kg/week demonstrated superiority in change in ACH-specific height SDS compared to placebo. TransCon CNP was generally safe and well tolerated, with no discontinuations. No serious AEs related to treatment were reported; two unrelated SAEs were reported.