Ascendis Pharma A/S (ASND) revealed positive topline data from a pivotal Phase 3 clinical trial evaluating TransCon CNP (navepegritide), an investigational prodrug, in children with achondroplasia, a genetic condition resulting in dwarfism. The trial, involving 84 children, showcased promising results for TransCon CNP in enhancing growth velocity compared to placebo.
TransCon CNP, administered once weekly, is designed to provide sustained release and continuous exposure of C-type natriuretic peptide (CNP). CNP is a naturally occurring hormone that regulates blood flow, blood pressure, and the reactivity of platelets and leukocytes.
The primary endpoint of the trial, measuring annualized growth velocity (AGV) at Week 52, showed a significant difference between the TransCon CNP and placebo groups. Children treated with TransCon CNP exhibited an LS mean AGV of 5.89 cm/year compared to 4.41 cm/year in the placebo arm, demonstrating a statistically significant difference (p<0.0001).
Further analysis revealed that TransCon CNP's efficacy extended across various age subgroups. Children aged 2 to <5 years treated with TransCon CNP showed an LS mean AGV of 6.07 cm/year at Week 52 compared to 5.06 cm/year in the placebo arm. Similarly, children aged 5-11 demonstrated an LS mean AGV of 5.79 cm/year at Week 52 with TransCon CNP, surpassing the placebo group's 4.02 cm/year.
The trial also assessed secondary endpoints, including changes in ACH Height Z-score and CDC Height Z-score. Children treated with TransCon CNP demonstrated significant improvements in both these measures compared to the placebo arm.
In addition to growth-related outcomes, the study found numerical improvements in health-related quality of life for children receiving TransCon CNP. This positive impact was observed across various domains measured by the Achondroplasia Child Experience Measure (ACEM).
Importantly, TransCon CNP demonstrated a safety profile comparable to placebo, with generally mild treatment-emergent adverse events (TEAEs). The drug was well-tolerated, showing no evidence of a hypotensive effect and a low frequency of mild injection site reactions.
Based on these positive results, Ascendis Pharma plans to submit a marketing application to the FDA for TransCon CNP for the treatment of achondroplasia in the first quarter of 2025. A similar application is planned for the European market in the third quarter of 2025.
The news of these encouraging trial results has positively impacted ASND stock, which saw a 17.4% increase to $140 at the last check on Monday.
This data underscores the potential of TransCon CNP as a meaningful treatment option for children with achondroplasia. The positive safety profile and significant improvement in growth velocity indicate a promising future for this investigational drug. The upcoming FDA and European applications are eagerly anticipated by the medical community and families impacted by achondroplasia.
