Phase 2 ACcomplisH Trial Data on TransCon CNP for Achondroplasia in Children Presented at ICCHBH 2024

COPENHAGEN, Denmark, June 24, 2024 – Ascendis Pharma, Inc. announced new data from its Phase 2 ACcomplisH Trial of TransCon CNP (navepegritide) in children with achondroplasia. The findings were presented by Ravi Savarirayan, M.D., from Murdoch Children’s Research Center, during the International Conference on Children’s Bone Health (ICCBH) 2024 in Salzburg, Austria.

The trial's data revealed that children aged 2 to 10 treated for one year with TransCon CNP at a dose of 100μg/kg/week experienced significant improvements in well-being and physical functioning compared to a placebo group. This indicates that the positive effects of TransCon CNP extend beyond merely enhancing linear growth, suggesting potential additional direct benefits from the treatment.

Dr. Savarirayan highlighted the significance of the results, stating, “Once-weekly TransCon CNP has shown not only an improvement in linear growth with a good safety profile but also additional benefits beyond growth. As the first investigational drug for achondroplasia to show substantial improvements over placebo in health-related quality of life metrics, TransCon CNP at the pivotal dose could address the health and quality-of-life issues associated with this condition.”

TransCon CNP is an investigational prodrug of C-type natriuretic peptide (CNP), designed for sustained release and administered once weekly. The ACcomplisH trial is a Phase 2, multicenter, randomized, double-blind, placebo-controlled study involving 57 children with achondroplasia. Participants were assigned in a 3:1 ratio to receive either TransCon CNP in various dose-escalation cohorts or a placebo for 52 weeks. Post this period, participants could continue with TransCon CNP in an open-label extension phase at the 100μg/kg/week dose. Quality of life assessments compared data from the 52-week period between those on the pivotal TransCon CNP dose and the pooled placebo group.

The study observed statistically significant improvements in several exploratory endpoints. These included Daily Living Functioning and Emotional Well-Being domains of the Achondroplasia Child Experience Measure-Impact (ACEM) assessment, and the SF-10 Physical Summary for children aged 5 and older. Additionally, growth data across the entire trial population on the 100μg/kg/week dose for 52 weeks were consistent with earlier findings from this cohort.

No new safety concerns emerged, and there were no serious adverse events linked to the study drug. The majority of treatment-emergent adverse events (TEAEs) were mild to moderate, with a low rate of injection site reactions.

In summary, TransCon CNP has shown promise not only in improving growth but also in enhancing overall well-being and physical functioning in children with achondroplasia, without major safety issues. This investigational treatment could potentially address both health and quality-of-life complications associated with achondroplasia.