COPENHAGEN, Denmark — Ascendis Pharma A/S (Nasdaq: ASND) today announced three oral presentations of its latest achondroplasia and hypoparathyroidism data at ASBMR 2025, the annual meeting of the American Society for Bone & Mineral Research being held in Seattle from September 5-8, 2025. These include a new analysis of pivotal trial data correlating improvements in leg bowing with improved physical functioning in children with achondroplasia treated with TransCon® CNP (navepegritide); the first medical congress presentation of clinical trial data for children with achondroplasia treated with TransCon CNP and TransCon hGH (lonapegsomatropin) combination therapy; and clinical trial data demonstrating the sustained improvements in skeletal dynamics in adults with hypoparathyroidism treated for 4 years with TransCon PTH (palopegteriparatide).
| Achondroplasia | |
| Saturday September 6 12:00 – 12:15 pm General Session Room 6BC |
Oral Presentation Abstract 1039 (See also poster PM-155.) Improvements in Lower Extremity Alignment Are Associated with Physical Functioning in Children with Achondroplasia Treated with Navepegritide: 52-Week Results From the ApproaCH TrialPresented by Leanne Ward, M.D. Children’s Hospital of Eastern Ontario, University of Ottawa (Canada) |
| Monday September 8 11:15 – 11:30 am General Session Room 6BC |
Oral Presentation Abstract 1126 (See also poster PM-118.) Once-Weekly Lonapegsomatropin (TransCon hGH) Added to Once-Weekly Navepegritide (TransCon CNP) in Children with Achondroplasia: 26-Week Results from the Phase 2 COACH TrialPresented by Ciara McDonnell, M.D. Children’s Health Ireland at Temple Street and University of Dublin, Trinity College (Ireland) |
| Hypoparathyroidism | |
| Saturday September 6 11:45 am – 12:00 pm General Session Room 6BC |
Oral Presentation Abstract 1038 Improved Skeletal Dynamics in Adults Treated with Palopegteriparatide for Chronic Hypoparathyroidism: 214-Week Results from the Phase 2 PaTH Forward TrialPresented by Mishaela Rubin, M.D. Columbia University (United States) |
“We are pleased to continue our partnership with these renowned investigators and believe that our latest data reinforces the transformative potential of the mono- and combination therapies that form the foundation of our growth disorder portfolio,” said Aimee Shu, M.D., Executive Vice President of Endocrine & Rare Disease Medical Sciences and Chief Medical Officer at Ascendis Pharma. “And, through this presentation of long-term skeletal dynamics data in the United States, we are pleased to reinforce the long-term benefits of our groundbreaking treatment for adults with hypoparathyroidism.”
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 have detrimental effects on quality of life, physical functioning, and psychosocial function. Individuals with achondroplasia often require multiple surgeries and procedures to alleviate the condition’s many complications.
About Hypoparathyroidism
Hypoparathyroidism is an endocrine disease caused by insufficient levels of parathyroid hormone (PTH), the primary regulator of calcium and phosphate balance in the body, acting directly on bone and kidney and indirectly on the intestine. Individuals with hypoparathyroidism may experience a range of severe and potentially life-threatening short-term and long-term complications, including neuromuscular irritability, renal complications, extra-skeletal calcifications, and cognitive impairment. Post-surgical hypoparathyroidism accounts for the majority of cases (70-80%), while other etiologies include autoimmune and idiopathic causes.
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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