COPENHAGEN, Denmark, Nov. 17, 2025 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced that pivotal Week 52 results from its randomized double-blind, placebo-controlled ApproaCH Trial of investigational once-weekly TransCon® CNP (navepegritide) in children with achondroplasia have been published in JAMA Pediatrics, a journal of the American Medical Association. In the publication, titled “Once-Weekly Navepegritide in Children with Achondroplasia: The ApproaCH Randomized Clinical Trial,” t ...

Core Insights - Ascendis Pharma A/S announced three oral presentations of data related to achondroplasia and hypoparathyroidism at ASBMR 2025, highlighting the effectiveness of their therapies [1][2] Group 1: Achondroplasia - Achondroplasia is a rare genetic condition affecting over 250,000 people globally, leading to various complications beyond skeletal dysplasia, including muscular and neurological issues [3] - The company presented new analyses correlating improvements in leg bowing with enhanced physical functioning in children treated with TransCon® CNP [1] - The first medical congress presentation of clinical trial data for children treated with TransCon CNP and TransCon hGH combination therapy was also shared [1] Group 2: Hypoparathyroidism - Hypoparathyroidism is characterized by insufficient parathyroid hormone levels, leading to severe complications, with post-surgical cases accounting for 70-80% of instances [4] - Clinical trial data demonstrated sustained improvements in skeletal dynamics in adults treated for four years with TransCon PTH [1][2] Group 3: Company Overview - Ascendis Pharma is a global biopharmaceutical company focused on innovative therapies using its TransCon technology platform to address unmet medical needs [5] - The company emphasizes its commitment to patient care, scientific innovation, and passion in its operations [5]

Core Insights - Ascendis Pharma announced positive interim results from the COACH Trial, demonstrating that the combination of TransCon CNP and TransCon hGH significantly improves growth and body proportionality in children with achondroplasia after 26 weeks of treatment [2][4][12] Group 1: Clinical Trial Results - For treatment-naïve children, the combination treatment resulted in a mean annualized growth velocity (AGV) of 9.14 cm/year, with a height Z-score improvement of +0.53 over 26 weeks [1][12] - For children previously treated with TransCon CNP, the mean AGV was 8.25 cm/year, with a height Z-score improvement of +0.44 over the same period [1][12] - The combination treatment led to accelerated improvement in body proportionality, aligning with increased linear growth [1][12] Group 2: Safety and Tolerability - The safety and tolerability profile of the combination treatment was consistent with that of the individual monotherapies, with generally mild treatment-emergent adverse events (TEAEs) reported [1][4][12] Group 3: Trial Design and Future Plans - The COACH Trial is a Phase 2 open-label trial investigating the efficacy, safety, and tolerability of the combination treatment in children aged 2 to 11 years with achondroplasia [5] - The interim analysis will be followed by Week 52 data, expected in Q4 2025, with plans to initiate a Phase 3 trial in the same timeframe [5][11] Group 4: Product Information - TransCon CNP is under priority review by the FDA as a monotherapy for children with achondroplasia, while TransCon hGH is already approved and marketed as SKYTROFA for pediatric growth hormone deficiency [3][10] - Both products utilize Ascendis Pharma's innovative TransCon technology platform, which aims to provide sustained release of active compounds [3][10]