Ascendis Reports Presentation Of Data From Phase 2 ACcomplisH Trial Of TransCon CNP (Navepegritide) In Children Aged 2 To 10 Years Old With Achondroplasia; Says Data Show Significant Improvements In Well-being
$ASND
Biotechnology: Pharmaceutical Preparations
Health Care
The data showed that patients treated for one year at the pivotal 100μg/kg/week dose demonstrated significant improvements in well-being and physical functioning compared to placebo.
These improvements could not be explained by changes in linear growth only, supporting a potential additional direct treatment effect of TransCon CNP beyond linear growth.
TransCon CNP is an investigational prodrug of C-type natriuretic peptide (CNP) administered once weekly, designed to provide sustained release of active CNP.
No new safety signals were observed, and no serious adverse events related to the study drug were reported. Most treatment-emergent adverse events (TEAEs) were Grade 1-2, with a low frequency of injection site reactions.