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.
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