Ascendis Pharma Presents Long-Term Data on Rare Endocrine Disease Therapies at Major Medical Conferences
Key Insights
Ascendis Pharma will present 4-year efficacy and safety data from the Phase 2 PaTH Forward Trial of TransCon PTH (palopegteriparatide) for chronic hypoparathyroidism (search) at ESPE & ESE 2025.
The company will share 52-week results from the pivotal ApproaCH Trial of TransCon CNP (navepegritide) in children with achondroplasia (search), including growth and bone morphometry data.
Additional presentations at ENDO 2025 will feature 3-year data from the Phase 3 PaTHway Trial demonstrating sustained improvement in renal function and maintenance of normal biochemistries.
Ascendis Pharma A/S announced it will present comprehensive clinical data from its rare endocrine disease portfolio at two major medical conferences in 2025, showcasing long-term efficacy and safety results for therapies targeting hypoparathyroidism (search), achondroplasia (search), and growth hormone deficiency (search).
Hypoparathyroidism Program Shows Long-Term Benefits
The company will present 4-year efficacy and safety data from the Phase 2 PaTH Forward Trial of TransCon PTH (palopegteriparatide) in adults with chronic hypoparathyroidism (search) at ESPE & ESE 2025. Dr. Andrea Palermo will deliver the oral presentation on May 12, highlighting the long-term treatment outcomes for this rare endocrine condition.
At ENDO 2025, Dr. Aliya Khan will present 3-year results from the Phase 3 PaTHway Trial, demonstrating the safety and efficacy of long-term treatment with palopegteriparatide. The data shows maintenance of serum and urine biochemistries within normal levels and sustained improvement in renal function at Week 156.
Hypoparathyroidism (search) is caused by insufficient levels of parathyroid hormone (PTH), the primary regulator of calcium and phosphate balance in the body. Individuals with this condition may experience severe and potentially life-threatening complications, including neuromuscular irritability, renal complications, extra-skeletal calcifications, and cognitive impairment. Post-surgical hypoparathyroidism accounts for 70-80% of cases.
Achondroplasia Trial Results Demonstrate Growth Benefits
The pivotal ApproaCH Trial of TransCon CNP (navepegritide) in children with achondroplasia (search) will be featured in multiple presentations. Dr. Leanne Ward will present effects on bone morphometry at 52 weeks, while Dr. Hanne Hove will share growth data from the same timepoint at ESPE & ESE 2025.
At ENDO 2025, Dr. Carlos Bacino will present additional safety and tolerability data from Week 52 of the ApproaCH Trial, providing comprehensive insights into the treatment's profile in pediatric patients.
Achondroplasia (search) is a rare genetic condition arising from a systemic fibroblast growth factor receptor 3 (FGFR3 (search)) variant that affects more than 250,000 people worldwide. The condition causes serious muscular, neurological, and cardiorespiratory complications in addition to skeletal dysplasia. Medical complications vary across different life stages and can include spinal deformities, enlarged brain ventricles, impaired muscle strength, hearing deficits, upper airway obstructions, and chronic pain.
Growth Hormone Deficiency Program Advances
The foresiGHt Trial of TransCon hGH (lonapegsomatropin) in adults with growth hormone deficiency (search) will be presented by Dr. Aleksandra Gilis-Januszewska at ESPE & ESE 2025. The presentation will demonstrate the efficacy and safety of once-weekly treatment in adult patients.
Adult growth hormone deficiency (search) is a condition where the body does not produce enough growth hormone, leading to symptoms including central obesity, metabolic syndrome, decreased bone density, alterations in lipid profile, fatigue, and psychological symptoms such as cognitive impairment and depression.
TransCon Technology Platform
"As new treatments and clinical practices transform the outlook for rare endocrine diseases, we are pleased to partner with trial investigators and leading experts to share new data highlighting clinical benefits of our novel therapies," said Dr. Aimee Shu, Executive Vice President of Endocrine & Rare Disease Medical Science and Chief Medical Officer at Ascendis Pharma.
Dr. Shu emphasized the company's progress, stating, "Ascendis is making important advances in its growing Endocrinology Rare Disease portfolio. We are pleased to partner with renowned investigators during ENDO 2025 to share data demonstrating the long-term benefits and safety of our groundbreaking treatment for adults with hypoparathyroidism (search), as well as data that we believe supports the transformative potential of our investigational therapy for children with achondroplasia (search)."
The presentations will take place at ESPE & ESE 2025 in Copenhagen (May 10-13, 2025) and ENDO 2025 in San Francisco (July 12-15, 2025), providing the medical community with comprehensive updates on Ascendis' rare disease pipeline and the potential of its TransCon technology platform to address unmet medical needs in endocrinology.