Ascendis Pharma announces new data from ApproaCH Trial of TransCon CNP

Ascendis Pharma (ASND) announced new data showing improvements in growth and bone morphometry from Week 52 of its pivotal ApproaCH Trial of TransCon CNP in children with achondroplasia. TransCon CNP is an investigational prodrug of C-type natriuretic peptide administered once weekly providing continuous exposure of active CNP to receptors on tissues throughout the body, including growth plates and skeletal muscle. The double-blind placebo-controlled ApproaCH Trial included 84 children with achondroplasia randomized 2:1 for 52 weeks, followed by an open-label extension period. At Week 52 of the trial, TransCon CNP demonstrated superiority over placebo in annualized growth velocity, with a safety and tolerability profile comparable to placebo that included a low rate of injection site reactions, no treatment-related serious adverse events, no cases of symptomatic hypotension, no fractures, and no acceleration of bone age versus chronological age. Analyses also showed that TransCon CNP improved aspects of bone morphometry at Week 52. This included improvement in lower limb alignment and proportional growth, as well as increases in spinal canal dimensions, versus placebo.