Ascendis Pharma A/S (NASDAQ:ASND) on Wednesday announced Week 52 data from the open-label sentinel cohort of its reACHin trial.
The data showed that once-weekly TransCon CNP (navepegritide) provided stabilization or improvement in foramen magnum stenosis alongside linear growth benefits in infants with achondroplasia aged 0 to under 2 years.
Growth And Cranio-Cervical Health
Treatment with TransCon CNP maintained or improved cranio-cervical junction health across all cohort participants over 52 weeks.
According to the Achondroplasia Foramen Magnum Score, a standardized MRI-based grading system that evaluates skull bone narrowing to prevent brainstem and spinal cord compression, every child achieved stable or improved scores.
The sagittal diameter of the foramen magnum enlarged by a mean of 3.15 mm, and no patients required decompression surgeries.
The therapy also yielded notable linear growth outcomes. Participants achieved an average annualized growth velocity of 9.9 cm per year and a mean increase from baseline in ACH-specific supine length Z-score of +0.42.
Safety And Dosing Consistency
Pharmacokinetic outcomes matched observations in older children, supporting the 100 μg/kg/week dosage.
TransCon CNP demonstrated high overall tolerability, with zero injection site reactions, deaths, fractures, bone-related safety events, or symptomatic hypotension reported during the study.
Investigators observed no adverse events deemed related to treatment, leading to zero trial withdrawals or dose disruptions.
In June, Ascendis Pharma reported five-year results from its Phase 2 PaTH Forward trial, showing that TransCon PTH (palopegteriparatide) delivered sustained efficacy and safety in adults with hypoparathyroidism.
The company said the therapy continued to replicate the effects of endogenous parathyroid hormone (PTH), supporting stable calcium control, kidney health, bone health, and quality of life while allowing most patients to discontinue conventional treatments.
ASND Price Action: Ascendis Pharma shares were up 0.10% at $261.06 at the time of publication on Thursday, according to Benzinga Pro data.
Image via Shutterstock/ oatawa
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