New 3-Year Phase 3 Data Confirmed Sustained Response to TransCon® PTH (Palopegteriparatide) Therapy in Adults with Hypoparathyroidism, Including Improvements in Biochemistries, Kidney Function, and Quality of Life
Ascendis Pharma (Nasdaq: ASND) announced promising 3-year Phase 3 data for TransCon PTH (palopegteriparatide) in treating adults with hypoparathyroidism. The PaTHway Trial demonstrated significant sustained efficacy with 88% of patients achieving normal calcium levels and 96% becoming independent from conventional therapy at Week 156.
Key outcomes include improved kidney function with mean eGFR increase of 8.76 mL/min/1.73 m2 across all participants, and 13.98 mL/min/1.73 m2 in patients with lower baseline eGFR. The trial showed high retention with 89% completion rate, while demonstrating sustained improvements in patient symptoms and quality of life. The treatment was generally well-tolerated with no new safety concerns identified.
Ascendis Pharma (Nasdaq: ASND) ha annunciato dati promettenti a 3 anni della Fase 3 per TransCon PTH (palopegteriparatide) nel trattamento degli adulti con ipoparatiroidismo. Lo studio PaTHway ha dimostrato una efficacia sostenuta significativa, con l'88% dei pazienti che ha raggiunto livelli normali di calcio e il 96% che è diventato indipendente dalla terapia convenzionale alla settimana 156.
I risultati chiave includono un miglioramento della funzione renale con un aumento medio della eGFR di 8,76 mL/min/1,73 m2 in tutti i partecipanti e di 13,98 mL/min/1,73 m2 nei pazienti con eGFR basale più bassa. Lo studio ha mostrato una alta retention con un tasso di completamento dell'89%, evidenziando miglioramenti duraturi nei sintomi dei pazienti e nella qualità della vita. Il trattamento è stato generalmente ben tollerato senza nuove preoccupazioni di sicurezza identificate.
Ascendis Pharma (Nasdaq: ASND) anunció datos prometedores a 3 años de la Fase 3 para TransCon PTH (palopegteriparatida) en el tratamiento de adultos con hipoparatiroidismo. El ensayo PaTHway demostró una eficacia sostenida significativa, con el 88% de los pacientes alcanzando niveles normales de calcio y el 96% volviéndose independientes de la terapia convencional en la semana 156.
Los resultados clave incluyen una mejora en la función renal con un aumento medio del eGFR de 8,76 mL/min/1,73 m2 en todos los participantes y de 13,98 mL/min/1,73 m2 en pacientes con eGFR basal más bajo. El ensayo mostró una alta retención con una tasa de finalización del 89%, demostrando mejoras sostenidas en los síntomas de los pacientes y en la calidad de vida. El tratamiento fue generalmente bien tolerado sin identificarse nuevas preocupaciones de seguridad.
Ascendis Pharma (나스닥: ASND)는 성인 저부갑상선증 치료를 위한 TransCon PTH(팔로페그테리파타이드)의 3년차 3상 임상 데이터를 발표했습니다. PaTHway 시험은 유의미한 지속적 효능을 입증했으며, 156주차에 88%의 환자가 정상 칼슘 수치를 달성했고 96%가 기존 치료에서 독립했습니다.
주요 결과로는 모든 참가자에서 평균 eGFR이 8.76 mL/min/1.73 m2 증가했고, 기저 eGFR이 낮은 환자군에서는 13.98 mL/min/1.73 m2 증가하는 신장 기능 개선이 포함됩니다. 시험은 89%의 높은 완료율을 보이며 높은 유지율을 나타냈고, 환자의 증상 및 삶의 질이 지속적으로 개선됨을 보여주었습니다. 치료는 대체로 잘 견뎌졌으며 새로운 안전성 문제는 발견되지 않았습니다.
Ascendis Pharma (Nasdaq : ASND) a annoncé des données prometteuses à 3 ans de la Phase 3 pour TransCon PTH (palopegteriparatide) dans le traitement des adultes atteints d'hypoparathyroïdie. L'essai PaTHway a démontré une efficacité soutenue significative, avec 88 % des patients atteignant des niveaux normaux de calcium et 96 % devenant indépendants de la thérapie conventionnelle à la semaine 156.
Les résultats clés incluent une amélioration de la fonction rénale avec une augmentation moyenne du eGFR de 8,76 mL/min/1,73 m2 chez tous les participants, et de 13,98 mL/min/1,73 m2 chez les patients ayant un eGFR basal plus faible. L'essai a montré une forte rétention avec un taux de complétion de 89 %, tout en démontrant des améliorations durables des symptômes des patients et de leur qualité de vie. Le traitement a été généralement bien toléré sans nouvelles préoccupations de sécurité identifiées.
Ascendis Pharma (Nasdaq: ASND) gab vielversprechende 3-Jahres-Daten der Phase-3-Studie für TransCon PTH (Palopegteriparatid) bei der Behandlung von Erwachsenen mit Hypoparathyreoidismus bekannt. Die PaTHway-Studie zeigte eine signifikante und anhaltende Wirksamkeit, wobei 88 % der Patienten normale Kalziumwerte erreichten und 96 % nach 156 Wochen von der konventionellen Therapie unabhängig wurden.
Zu den wichtigsten Ergebnissen zählen eine verbesserte Nierenfunktion mit einem durchschnittlichen Anstieg der eGFR um 8,76 mL/min/1,73 m2 bei allen Teilnehmern und um 13,98 mL/min/1,73 m2 bei Patienten mit niedrigerem Ausgangs-eGFR. Die Studie zeigte eine hohe Verweildauer mit einer Abschlussrate von 89 % und demonstrierte anhaltende Verbesserungen der Symptome und der Lebensqualität der Patienten. Die Behandlung wurde im Allgemeinen gut vertragen, ohne neue Sicherheitsbedenken.
- 88% of patients achieved normal albumin-adjusted serum calcium levels at Week 156
- 96% of patients became independent from conventional therapy
- Significant kidney function improvement with mean eGFR increase of 8.76 mL/min/1.73 m2
- High trial retention rate of 89% over 3.5 years
- Well-tolerated safety profile with no serious treatment-related adverse events
- None.
Insights
Ascendis' TransCon PTH shows strong 3-year efficacy data in hypoparathyroidism with sustained improvements in key clinical measures.
The new 3-year Phase 3 data for TransCon PTH (palopegteriparatide) demonstrates remarkably durable efficacy across multiple clinically significant endpoints. The 89% retention rate over 3.5 years is particularly impressive for a long-term trial, suggesting strong patient satisfaction with the treatment.
The efficacy results are compelling: 88% of patients maintained normal albumin-adjusted serum calcium levels and 96% became independent from conventional therapy. This represents a potential paradigm shift in hypoparathyroidism management, which has historically relied on calcium and active vitamin D supplementation that often fails to adequately control symptoms while increasing risk of complications.
Perhaps most significant is the sustained improvement in kidney function, with mean eGFR increasing by 8.76 mL/min/1.73 m² overall and by 13.98 mL/min/1.73 m² in patients with impaired renal function at baseline. This addresses a critical unmet need, as kidney complications represent a major source of morbidity in hypoparathyroidism patients.
The normalization of 24-hour urine calcium excretion is equally important, as hypercalciuria is a common complication of conventional therapy that contributes to renal calcification and decline in kidney function. The favorable safety profile, with predominantly mild-to-moderate adverse events and no treatment-related serious adverse events, further supports the potential of TransCon PTH as a transformative therapy for this challenging endocrine disorder.
COPENHAGEN, Denmark, July 14, 2025 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced new data from Week 156 of its Phase 3 PaTHway Trial confirming that long-term treatment with TransCon PTH (palopegteriparatide) continued to provide a durable response in adults with hypoparathyroidism regardless of its cause (post-surgical, autoimmune, genetic, or idiopathic), including improvements in biochemistries, kidney function, and quality of life. Results were shared over the weekend in an oral presentation by Aliya Khan, M.D., Clinical Professor of Medicine, Division of Endocrinology and Geriatrics and Director of the Calcium Disorders Clinic at McMaster University (Canada) at ENDO 2025, the annual meeting of the Endocrine Society being held in San Francisco.
PaTHway was a Phase 3, randomized, double-blind, placebo-controlled 26-week trial of 82 adults with chronic hypoparathyroidism, followed by an open-label extension period through Week 182. During the initial 26-week blinded period, participants were randomized 3:1 TransCon PTH:placebo. Renal function was assessed by estimated glomerular filtration rate (eGFR). Safety assessments included 24-hour urine-calcium and treatment-emergent adverse events (TEAEs). Hypoparathyroidism-related symptoms, functioning, and well-being were measured by the Hypoparathyroidism Patient Experience Scale (HPES). Overall retention in the trial was high, with 73 of the original 82 adults enrolled (
At Week 156, 64 patients (
“We are pleased to report these latest data from our second clinical trial of TransCon PTH showing sustained efficacy and improvements across key health and quality of life measures, beginning at the earliest timepoints in the Phase 3 PaTHway trial and continuing through three years of treatment,” said Aimee Shu, M.D. Executive Vice President of Endocrine & Rare Disease Medical Science and Chief Medical Officer at Ascendis Pharma. “These responses, including normalization of skeletal dynamics with significant and clinically meaningful improvements in kidney function, demonstrate the long-term benefits of this treatment option for the vast majority of adults with hypoparathyroidism, regardless of their disease etiology.”
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
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.
Forward-Looking Statements
This press release contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, included in this press release regarding Ascendis’ future operations, plans and objectives of management are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to (i) the long term benefits of TransCon PTH therapy for adults with hypoparathyroidism, regardless of disease etiology, (ii) Ascendis’ ability to apply its TransCon technology platform to make a meaningful difference for patients, and (iii) Ascendis’ application of its TransCon technologies to develop new therapies that demonstrate best-in-class potential to address unmet medical needs. Ascendis may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions, expectations and projections disclosed in the forward-looking statements. Various important factors could cause actual results or events to differ materially from the forward-looking statements that Ascendis makes, including the following: dependence on third party manufacturers, distributors and service providers for Ascendis’ products and product candidates; unforeseen safety or efficacy results in Ascendis’ development programs or on-market products; unforeseen expenses related to commercialization of any approved Ascendis products; unforeseen expenses related to Ascendis’ development programs; unforeseen selling, general and administrative expenses, other research and development expenses and Ascendis’ business generally; delays in the development of its programs related to manufacturing, regulatory requirements, speed of patient recruitment or other unforeseen delays; Ascendis’ ability to obtain additional funding, if needed, to support its business activities; the impact of international economic, political, legal, compliance, social and business factors, including tariffs and trade policies. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to Ascendis’ business in general, see Ascendis’ Annual Report on Form 20-F filed with the U.S. Securities and Exchange Commission (SEC) on February 12, 2025, and Ascendis’ other future reports filed with, or submitted to, the SEC. Forward-looking statements do not reflect the potential impact of any future licensing, collaborations, acquisitions, mergers, dispositions, joint ventures, or investments that Ascendis may enter into or make. Ascendis does not assume any obligation to update any forward-looking statements, except as required by law.
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FAQ
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