Ascendis Pharma reported a mean +3.15 mm change in foramen magnum sagittal diameter among infants receiving once-weekly TransCon CNP. The data also showed stable or improved cranio-cervical health and linear growth, supporting continued evaluation of the treatment in infants with achondroplasia.

Key Highlights

  • Foramen magnum scores were stable or improved in all children in the sentinel cohort.
  • Mean change in ACH-specific supine length Z-Score reached +0.42.
  • Mean annualized growth velocity was 9.9 cm per year.
  • No decompression surgeries occurred during the treatment period.

Ascendis Pharma A/S (NASDAQ: ASND) said once-weekly TransCon CNP produced a mean +3.15 mm change from baseline in the sagittal diameter of the foramen magnum in infants with achondroplasia. The Week 52 data also showed stabilization or improvement in cranio-cervical junction health alongside linear growth benefits.

ReACHin Shows Stable Cranio-Cervical Health

Seven infants participated in the open-label sentinel cohort, with a mean age of 11.7 months. No decompression surgeries occurred during the 52-week treatment period.

Growth and Safety Findings

Ascendis Pharma reported a mean change from baseline of +0.42 in ACH-specific supine length Z-score, while mean annualized growth velocity was 9.9 cm per year. Pharmacokinetic results were comparable with those observed in older children, supporting the treatment level used in the study. No adverse event was assessed as treatment-related, and no adverse event led to treatment interruption, discontinuation or trial withdrawal.