New 3-Year Skeletal Dynamics Data for Adults with Hypoparathyroidism Treated with TransCon™ PTH (Palopegteriparatide) Presented at ASBMR 2024
Ascendis Pharma A/S (Nasdaq: ASND) presented 3-year results from their ongoing Phase 2 PaTH Forward Trial at ASBMR 2024. The study, involving 57 adults with hypoparathyroidism, showed that long-term treatment with TransCon™ PTH (palopegteriparatide; marketed as YORVIPATH®) through Week 162 normalized bone remodeling.
Hypoparathyroidism is associated with low bone remodeling rates, accumulation of overly mature bone, and higher-than-average bone mineral density, potentially indicating poorer overall bone quality. The results suggest that long-term palopegteriparatide treatment promotes skeletal health parameters similar to those in parathyroid sufficiency.
TransCon PTH is a once-daily prodrug of parathyroid hormone (PTH [1-34]), designed to provide continuous PTH exposure over 24 hours. Dr. Mishaela Rubin, Associate Professor of Medicine at Columbia University, presented the findings in an oral presentation titled 'Palopegteriparatide Improves Skeletal Dynamics in Adults with Chronic Hypoparathyroidism: 3 Year Results from the Phase 2 PaTH Forward Trial' (#1091).
Ascendis Pharma A/S (Nasdaq: ASND) ha presentato i risultati triennali del loro in corso Studio di Fase 2 PaTH Forward all'ASBMR 2024. Lo studio, che ha coinvolto 57 adulti con ipoparatiroidismo, ha dimostrato che il trattamento a lungo termine con TransCon™ PTH (palopegteriparatide; commercializzato come YORVIPATH®) fino alla settimana 162 ha normalizzato il rimodellamento osseo.
L'ipoparatiroidismo è associato a basse percentuali di rimodellamento osseo, accumulo di tessuto osseo troppo maturo e una densità minerale ossea superiore alla media, indicando potenzialmente una qualità ossea complessivamente scadente. I risultati suggeriscono che il trattamento a lungo termine con palopegteriparatide promuove parametri di salute scheletrica simili a quelli nella sufficienza paratiroidea.
TransCon PTH è un pro-farmaco di ormone paratiroideo (PTH [1-34]), progettato per fornire un'esposizione continua all'ormone paratiroideo per 24 ore. La Dott.ssa Mishaela Rubin, Professore Associato di Medicina presso la Columbia University, ha presentato i risultati in una presentazione orale dal titolo 'Palopegteriparatide Migliora la Dinamica Scheletrica negli Adulti con Ipoparatiroidismo Cronico: Risultati Triennali dal Trial di Fase 2 PaTH Forward' (#1091).
Ascendis Pharma A/S (Nasdaq: ASND) presentó resultados de 3 años de su ensayo en curso de Fase 2 PaTH Forward en ASBMR 2024. El estudio, que involucró a 57 adultos con hipoparatiroidismo, mostró que el tratamiento a largo plazo con TransCon™ PTH (palopegteriparatide; comercializado como YORVIPATH®) a través de la Semana 162 normalizó el remodelado óseo.
El hipoparatiroidismo se asocia con tasas de remodelado óseo bajas, acumulación de hueso excesivamente maduro y una densidad mineral ósea superior a la media, lo que podría indicar una calidad ósea general deficiente. Los resultados sugieren que el tratamiento prolongado con palopegteriparatide promueve parámetros de salud esquelética similares a los de la suficiencia paratiroidea.
TransCon PTH es un profármaco diario de la hormona paratiroidea (PTH [1-34]), diseñado para proporcionar exposición continua a la PTH durante 24 horas. La Dra. Mishaela Rubin, Profesora Asociada de Medicina en la Universidad de Columbia, presentó los hallazgos en una presentación oral titulada 'Palopegteriparatide Mejora la Dinámica Esquelética en Adultos con Hipoparatiroidismo Crónico: Resultados de 3 Años del Ensayo de Fase 2 PaTH Forward' (#1091).
Ascendis Pharma A/S (NASDAQ: ASND)는 2024년 ASBMR에서 진행 중인 2상 PaTH Forward 시험의 3년 결과를 발표했습니다. 이 연구는 57명의 저파라타이로이드증 성인 참가자를 포함하였으며, TransCon™ PTH (팔로펙테리파라타이드; YORVIPATH®로 판매됨)로 장기 치료가 162주차까지 뼈 재형성을 정상화했다는 결과를 보여주었습니다.
저파라타이로이드증은 낮은 뼈 재형성 비율, 과도하게 성숙한 뼈의 축적, 평균보다 높은 뼈 미네랄 밀도와 관련이 있으며, 이는 전반적인 뼈 질이 저하될 가능성을 시사합니다. 이러한 결과는 팔로펙테리파라타이드의 장기 치료가 부갑상선 적정 상태와 유사한 골격 건강 지표를 촉진한다는 것을 시사합니다.
TransCon PTH는 부갑상선 호르몬(PTH [1-34])의 일일 프로약물로 설계되어 24시간 동안 지속적인 PTH 노출을 제공합니다. 콜롬비아 대학교의 내과 교수인 미샤엘라 루빈 박사는 '#1091'이라는 제목으로 '팔로펙테리파라타이드가 만성 저파라타이로이드증 성인에서 골격 역학을 개선한다: 2상 PaTH Forward 시험의 3년 결과'라는 구두 발표에서 연구 결과를 발표했습니다.
Ascendis Pharma A/S (Nasdaq: ASND) a présenté les résultats sur 3 ans de son essai en cours de phase 2 PaTH Forward lors de l'ASBMR 2024. L'étude, impliquant 57 adultes atteints d'hypoparathyroïdie, a révélé que le traitement à long terme avec TransCon™ PTH (palopegteriparatide; commercialisé sous le nom de YORVIPATH®) jusqu'à la semaine 162 normalisait le remodelage osseux.
L'hypoparathyroïdie est associée à des taux de remodelage osseux faibles, à l'accumulation de tissus osseux trop matures, et à une densité minérale osseuse supérieure à la moyenne, ce qui peut indiquer une qualité osseuse globale inférieure. Les résultats suggèrent que le traitement à long terme avec palopegteriparatide favorise des paramètres de santé squelettique semblables à ceux de la suffisance parathyroïdienne.
TransCon PTH est un pro-médicament administré une fois par jour de l'hormone parathyroïdienne (PTH [1-34]), conçu pour fournir une exposition continue à la PTH sur 24 heures. Dr. Mishaela Rubin, professeure associée de médecine à l'Université de Columbia, a présenté les résultats lors d'une présentation orale intitulée 'Palopegteriparatide améliore la dynamique squelettique chez les adultes atteints d'hypoparathyroïdie chronique : résultats de 3 ans de l'essai de phase 2 PaTH Forward' (#1091).
Ascendis Pharma A/S (Nasdaq: ASND) hat auf der ASBMR 2024 die 3-Jahres-Ergebnisse ihrer laufenden Phase-2-Studie PaTH Forward präsentiert. Die Studie, an der 57 Erwachsene mit Hypoparathyreoidismus teilnahmen, zeigte, dass die Langzeitbehandlung mit TransCon™ PTH (Palopegteriparatid; vermarktet als YORVIPATH®) bis zur Woche 162 die Knochenneubildung normalisierte.
Hypoparathyreoidismus wird mit niedrigen Raten der Knochenneubildung, der Ansammlung von überreifen Knochen und einer überdurchschnittlichen Knochendichte in Verbindung gebracht, was möglicherweise auf eine insgesamt schlechtere Knochenqualität hindeutet. Die Ergebnisse lassen darauf schließen, dass eine langfristige Behandlung mit Palopegteriparatid die Skelettgesundheitsparameter fördert, die mit einer ausreichenden Parathormonaktivität vergleichbar sind.
TransCon PTH ist ein einmal täglich zu verabreichendes Prodrug des Parathormons (PTH [1-34]), das für eine kontinuierliche PTH-Exposition über 24 Stunden ausgelegt ist. Dr. Mishaela Rubin, außerordentliche Professorin für Medizin an der Columbia University, präsentierte die Ergebnisse in einem mündlichen Vortrag mit dem Titel 'Palopegteriparatide verbessert die Skelettdynamik bei Erwachsenen mit chronic Hypoparathyreoidismus: 3-Jahres-Ergebnisse aus der Phase-2-Studie PaTH Forward' (#1091).
- TransCon PTH normalized bone remodeling in hypoparathyroidism patients after 162 weeks of treatment
- Long-term palopegteriparatide treatment promotes skeletal health parameters similar to parathyroid sufficiency
- Positive 3-year results from ongoing Phase 2 PaTH Forward Trial
- None.
Insights
The 3-year results from the Phase 2 PaTH Forward Trial of TransCon™ PTH (palopegteriparatide) in adults with hypoparathyroidism are significant for Ascendis Pharma. The data shows that long-term treatment with palopegteriparatide normalized bone remodeling, addressing a key issue in hypoparathyroidism patients who typically have low bone turnover and potentially compromised bone quality.
Key points:
- Treatment through Week 162 drove bone remodeling into the normal range
- Results suggest palopegteriparatide promotes skeletal health parameters similar to those with normal parathyroid function
- TransCon PTH is designed for once-daily dosing with continuous 24-hour PTH exposure
This data strengthens the clinical profile of YORVIPATH®, potentially supporting its long-term use and efficacy. For investors, positive long-term data could translate to sustained market presence and potential revenue growth. However, it's important to note that while promising, Phase 2 data still requires confirmation in larger Phase 3 trials for full validation of long-term benefits and safety.
COPENHAGEN, Denmark, Sept. 30, 2024 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) shared 3-year results from the Company’s ongoing Phase 2 PaTH Forward Trial of 57 adults with hypoparathyroidism showing that long-term treatment with TransCon™ PTH (palopegteriparatide; marketed as YORVIPATH®) through Week 162 drove bone remodeling into the normal range. Deficiency of parathyroid hormone is associated with low rates of bone remodeling, accumulation of overly mature bone, and higher-than-average bone mineral density that may correspond with poorer overall bone quality compared to that seen in the general population.1,2,3,4 In contrast, these results suggest that long-term palopegteriparatide treatment promotes attainment of skeletal health parameters in line with those expected with states of parathyroid sufficiency.
TransCon PTH is a prodrug of parathyroid hormone (PTH [1-34]), administered once daily, designed to provide continuous exposure to released PTH over the 24-hour dosing period.
Lead author Mishaela Rubin, M.D., Associate Professor of Medicine at Columbia University and an expert at the forefront of characterizing the dynamic and structural abnormalities of bone in hypoparathyroidism, shared the results in an oral presentation during ASBMR 2024, the annual meeting of the American Society for Bone & Mineral Research held in Toronto September 27-30.
Additional information associated with Dr. Rubin’s presentation, titled Palopegteriparatide Improves Skeletal Dynamics in Adults with Chronic Hypoparathyroidism: 3 Year Results from the Phase 2 PaTH Forward Trial (#1091), is available to ASBMR members and registered attendees at https://asbmr.org.
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 kidneys 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 applying its innovative TransCon technology platform to build a leading, fully integrated biopharma company focused on making a meaningful difference in patients’ lives. Guided by its core values of Patients, Science, and Passion, Ascendis uses its TransCon technologies to create new and potentially best-in-class therapies. 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
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Ascendis, Ascendis Pharma, the Ascendis Pharma logo, the company logo, TransCon, and YORVIPATH are trademarks owned by the Ascendis Pharma group. © September 2024 Ascendis Pharma A/S.
1. Rubin MR, et al. J Bone Miner Res 2008;23(12):2018-24.
2. Rubin, MR, et al. J Bone Miner Res 2018;33(11):1931-9.
3. Misof BM, et al. J Bone Miner Res 2016;31(1):180-9.
4. Cianferotti L, et al. J Endocrinol Invest 2023;46(7):1283-1304.
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FAQ
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