Ascendis Pharma publishes results from ApproaCH Trial in JAMA Pediatrics

Ascendis Pharma (ASND) announced that pivotal Week 52 results from its randomized double-blind, placebo-controlled ApproaCH Trial of investigational once-weekly TransCon CNP in children with achondroplasia have been published in JAMA Pediatrics, a journal of the American Medical Association. In the publication, titled “Once-Weekly Navepegritide in Children with Achondroplasia: The ApproaCH Randomized Clinical Trial,” the authors report that treatment with TransCon CNP led to significantly higher annualized growth velocity at Week 52 compared to placebo, as well as improved lower-limb alignment and body proportionality and positive changes in health-related quality of life, with a safety and tolerability profile similar to placebo. In addition to the key primary endpoint of annualized growth velocity superior to placebo, favorable impacts on body proportionality and leg bowing were reported at Week 52. These analyses showed treatment with TransCon CNP decreased upper-to-lower body segment ratio from baseline to Week 52 and improved tibial-femoral angle, mechanical axis deviation and fibula-to-tibia length ratio from baseline to Week 52 compared to placebo. In the trial, treatment with TransCon CNP resulted in numerical improvements in health-related quality of life compared to placebo, as measured across several Achondroplasia Child Experience Measure domains. The benefits of TransCon CNP were achieved without accelerating bone age or negatively affecting spinal curvature. In the trial, TransCon CNP demonstrated a safety and tolerability profile similar to placebo, with the majority of adverse events mild or moderate. Injection site reaction rates were low, and no observed symptomatic hypotension or bone fractures were reported.