Ascendis Pharma's Program Hits Primary Goal In Children With Short-Limbed Dwarfism

Ascendis Pharma A/S ASND has announced positive topline results from the ACcomplisH Phase 2 trial of once-weekly TransCon CNP compared to a placebo in children with achondroplasia aged 2 to 10 years old.
Achondroplasia is a form of short-limbed dwarfism.
The ACcomplisH Trial evaluated 57 children. All 57 randomized children completed the blinded portion of ACcomplisH and are currently continuing in the open-label extension (OLE) at the 100 µg/kg/week dose.
The trial met its primary objectives, demonstrating that TransCon CNP at 100 µg/kg/week was superior to the placebo on the primary efficacy endpoint of annualized height velocity (AHV) at 52 weeks.
Data showed robust and consistent results in prespecified analyses across age groups and dose levels, supporting continued development at the selected dose of 100 µg/kg/week.
Patients treated ≥6 months at 100 µg/kg/week in the blinded or OLE period demonstrated a consistent and sustained response, with a mean AHV of 5.39 cm/year (n=40).
TransCon CNP was generally well tolerated with a low frequency of injection site reactions; all 57 randomized children continued, with the longest treatment duration beyond two years.
In August, Ascendis Pharma submitted an FDA marketing application for TransCon PTH for adult patients with hypoparathyroidism.
Price Action: ASND shares closed 2.22% higher at $134.90 during after-hours trading on Friday.