Core Insights - Ascendis Pharma announced positive topline results from the Week 52 of the COACH trial, demonstrating that the combination therapy of once-weekly TransCon CNP and TransCon hGH showed durable growth without compromising safety or tolerability [2][3] Group 1: Trial Results - The combination therapy resulted in an annualized growth velocity (AGV) that exceeded the 97th percentile of average stature children [11] - For the treatment-naïve cohort, the mean AGV was 8.80 cm/year, with a mean ACH height Z-score improvement of +1.02, indicating a tripling of efficacy compared to TransCon CNP monotherapy [5][6] - The treated cohort showed a mean AGV of 8.42 cm/year, with an increase from baseline of 3.28 cm/year and a mean ACH height Z-score improvement of +0.86, increasing from 1.28 to 2.15 over 52 weeks [5][6] Group 2: Safety and Tolerability - The combination therapy was generally well tolerated, with mild treatment-emergent adverse events (TEAEs) consistent with those observed for the monotherapies [2][11] - Safety and tolerability profiles were consistent with TransCon CNP and TransCon hGH monotherapies, indicating a low incidence of injection site reactions [11] Group 3: Implications for Treatment - The results suggest that TransCon CNP may become a foundational therapy for achondroplasia, with TransCon hGH providing complementary benefits [2][3] - The trial highlights the potential for dual-agent regimens to set new standards of care in treating achondroplasia and other growth disorders [3][11] - Improvements in body proportionality and arm span were observed, aligning with the increase in linear growth, indicating benefits beyond just height [2][11] Group 4: Future Developments - Ascendis Pharma has submitted a protocol for a Phase 3 trial of TransCon CNP and TransCon hGH in pediatric achondroplasia and held an end of Phase 2 meeting with the FDA [7][11] - The U.S. FDA has granted Priority Review for TransCon CNP, with a PDUFA target action date of February 28, 2026 [3][11]

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 A/S will present new data on its hypoparathyroidism, achondroplasia, and growth hormone deficiency programs at the ESPE & ESE 2025 congress in Copenhagen from May 10-13, 2025 [1][2] Hypoparathyroidism - The company will showcase 4-year efficacy and safety data from the Phase 2 PaTH Forward Trial of TransCon PTH (palopegteriparatide) in adults with chronic hypoparathyroidism [2][4] - Presentations will include a retrospective matched cohort study using real-world data from England to estimate the risk of chronic kidney disease progression in chronic hypoparathyroidism [4][6] Achondroplasia - Ascendis will present Week 52 growth and bone morphometry data from the pivotal ApproaCH Trial of TransCon CNP (navepegritide) in children with achondroplasia [2][4] - The congress will feature discussions on assessing health-related quality of life (HRQoL) in achondroplasia across the life course [4][6] Growth Hormone Deficiency - The company will present results from the Phase 3 foresiGHt Trial supporting the efficacy and safety of once-weekly lonapegsomatropin in adults with growth hormone deficiency [2][6] - The presentations will highlight the importance of growth hormone in maintaining normal body composition and cardiometabolic health throughout adulthood [9] Company Overview - Ascendis Pharma is a global biopharmaceutical company focused on innovative therapies using its TransCon technology platform to address unmet medical needs [10] - The company is headquartered in Copenhagen, Denmark, with additional facilities in Europe and the United States [10]