COPENHAGEN, Denmark — Ascendis Pharma A/S (Nasdaq: ASND) today announced that its participation at ISDS 2026, the annual meeting of the International Skeletal Dysplasia Society being held in Toronto, Canada, from August 26-29, 2026, will include an oral presentation of Week 104 data from its pivotal ApproaCH Trial of once-weekly TransCon CNP (navepegritide) in children with achondroplasia. The presentation will be given by Carlos Bacino, M.D., FACMG, Professor of Molecular and Human Genetics, Baylor College of Medicine and Texas Children’s Hospital.
“This long-term data reinforces the benefits seen in clinical trials of once-weekly TransCon CNP, which have ranged from durable improvements in height to improvements in lower-extremity alignment, body proportionality, spinal canal dimensions, muscle function, and physical functioning – with a safety and tolerability profile similar to placebo and a low rate of injection site reactions,” said Aimee Shu, Executive Vice President, Chief Medical Officer at Ascendis Pharma. “The results align with improvements in health-related quality of life identified as important to the achondroplasia community and we look forward to sharing them with experts focused on advancing treatment of individuals living with skeletal dysplasia.”
Ascendis presentations at ISDS 2026 include:
| ORAL PRESENTATION | |
| Friday August 28 10:00-10:15am Session 4 |
Abstract #45 Improved Growth and Physical Functioning in Children with Achondroplasia Treated with Navepegritide in the ApproaCH Trial Open-Label Extension Presented by Carlos Bacino, M.D. |
| POSTER | |
| Wednesday – Saturday August 26-29 Poster Boards |
Number Needed to Harm Analysis for Injection Site Reactions When Indirect Treatment Comparison (ITC) Is Not Suitable Authors: Manoj Chevli et al |
About TransCon CNP
TransCon CNP is a prodrug of C-type natriuretic peptide (CNP) administered once weekly, designed to provide continuous exposure of active CNP to receptors on tissues throughout the body to counteract the overactive FGFR3 signaling in achondroplasia. In February 2026, TransCon CNP was approved by the U.S. Food & Drug Administration (FDA) under the trade name YUVIWEL® to increase linear growth in pediatric patients 2 years of age and older with achondroplasia with open epiphyses. Ascendis Pharma’s Marketing Authorisation Application for YUVIWEL is under review by the European Medicines Agency, with a regulatory decision anticipated in the fourth quarter of 2026.
About Achondroplasia
Achondroplasia is a rare genetic condition arising from a systemic fibroblast growth factor receptor 3 (FGFR3) variant that leads to an imbalance in the effects of the FGFR3 and CNP signaling pathways, estimated to affect more than 250,000 people worldwide. While historically considered a bone growth disorder, the FGFR3 variant seen in achondroplasia is expressed in tissues throughout the body, causing serious muscular, neurological, and cardiorespiratory complications in addition to skeletal dysplasia. Medical complications of achondroplasia vary across different stages of life. Throughout infancy and childhood, observed complications include spinal abnormalities, enlarged brain ventricles, impaired muscle strength and stamina, hearing deficits and chronic ear infections, upper airway obstructions, sleep-disordered breathing, hip problems, leg bowing, and chronic pain; many of these persist or worsen in adulthood. These medical complications can affect physical well-being and quality of life, and may be impacted by a range of individual, clinical, and social factors. Some individuals with achondroplasia require multiple procedures and surgeries to address specific functional or anatomical concerns.
About Ascendis Pharma A/S
Ascendis Pharma is a global biopharmaceutical company focused on applying our innovative TransCon technology platform to make a meaningful difference for patients. Guided by our core values of Patients, Science, and Passion, and following our algorithm for product innovation, we apply TransCon to develop new therapies that demonstrate best-in-class potential to address unmet medical needs. Ascendis is headquartered in Copenhagen, Denmark, and has additional facilities in Europe and the United States. Please visit ascendispharma.com to learn more.
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