New Data Shows Improvements in Growth and Bone Morphometry in Children with Achondroplasia Treated with TransCon® CNP (Navepegritide)
Ascendis Pharma (ASND) announced positive Week 52 data from its pivotal ApproaCH Trial of TransCon CNP (navepegritide) in children with achondroplasia. The double-blind placebo-controlled trial, involving 84 children aged 2-11 years, demonstrated superior annualized growth velocity compared to placebo. The drug showed a favorable safety profile with no treatment-related serious adverse events, symptomatic hypotension, fractures, or bone age acceleration.
Key improvements were observed in bone morphometry, including better lower limb alignment, proportional growth, and increased spinal canal dimensions. The trial featured a 2:1 randomization ratio (TransCon CNP:placebo) for 52 weeks, followed by an open-label extension period. The results support the company's goal to provide benefits beyond linear growth and potentially reduce future complications associated with achondroplasia.
Ascendis Pharma (ASND) ha annunciato dati positivi alla settimana 52 del suo trial ApproaCH, fondamentale per TransCon CNP (navepegritide) nei bambini con acondroplasia. Lo studio randomizzato, in doppio cieco e controllato con placebo, che ha coinvolto 84 bambini di età compresa tra 2 e 11 anni, ha mostrato una velocità di crescita annualizzata superiore rispetto al placebo. Il farmaco ha evidenziato un profilo di sicurezza favorevole, senza eventi avversi gravi correlati al trattamento, ipotensione sintomatica, fratture o accelerazione dell'età ossea.
Significativi miglioramenti sono stati riscontrati nella morfometria ossea, inclusi un migliore allineamento degli arti inferiori, una crescita proporzionale e un aumento delle dimensioni del canale spinale. Il trial ha previsto un rapporto di randomizzazione 2:1 (TransCon CNP: placebo) per 52 settimane, seguito da un periodo di estensione in aperto. I risultati supportano l'obiettivo dell'azienda di offrire benefici oltre la crescita lineare e di ridurre potenzialmente le complicanze future associate all'acondroplasia.
Ascendis Pharma (ASND) anunció datos positivos a la semana 52 de su ensayo pivotal ApproaCH con TransCon CNP (navepegritide) en niños con acondroplasia. El estudio doble ciego controlado con placebo, que incluyó a 84 niños de 2 a 11 años, demostró una velocidad de crecimiento anualizada superior en comparación con el placebo. El medicamento mostró un perfil de seguridad favorable sin eventos adversos graves relacionados con el tratamiento, hipotensión sintomática, fracturas ni aceleración de la edad ósea.
Se observaron mejoras clave en la morfometría ósea, incluyendo mejor alineación de las extremidades inferiores, crecimiento proporcional y aumento de las dimensiones del canal espinal. El ensayo tuvo una proporción de aleatorización 2:1 (TransCon CNP: placebo) durante 52 semanas, seguida de un período de extensión abierto. Los resultados respaldan el objetivo de la compañía de proporcionar beneficios más allá del crecimiento lineal y potencialmente reducir complicaciones futuras asociadas con la acondroplasia.
Ascendis Pharma (ASND)는 연골무형성증 아동을 대상으로 한 중추적 ApproaCH 임상시험에서 TransCon CNP (나베페그리타이드)의 52주차 긍정적 데이터를 발표했습니다. 2세에서 11세 사이의 84명의 아동을 대상으로 한 이 이중맹검 위약대조 시험은 연간 성장 속도에서 위약 대비 우수한 결과를 보였습니다. 약물은 치료 관련 중대한 이상반응, 증상성 저혈압, 골절 또는 골 연령 가속화 없이 우호적인 안전성 프로필을 나타냈습니다.
골 형태계측에서 주요 개선이 관찰되었으며, 하체 정렬 개선, 비례 성장, 척추관 크기 증가 등이 포함됩니다. 임상시험은 2:1 무작위 배정 비율(TransCon CNP: 위약)로 52주간 진행되었으며, 이후 공개 라벨 연장 기간이 이어졌습니다. 이 결과는 선형 성장 이상의 혜택을 제공하고 연골무형성증과 관련된 미래 합병증을 줄일 가능성을 회사의 목표로 뒷받침합니다.
Ascendis Pharma (ASND) a annoncé des données positives à la semaine 52 de son essai pivot ApproaCH portant sur TransCon CNP (navepégritide) chez des enfants atteints d’achondroplasie. L’essai en double aveugle contrôlé par placebo, impliquant 84 enfants âgés de 2 à 11 ans, a démontré une vélocité de croissance annualisée supérieure par rapport au placebo. Le médicament a présenté un profil de sécurité favorable, sans événements indésirables graves liés au traitement, hypotension symptomatique, fractures ou accélération de l’âge osseux.
Des améliorations clés ont été observées en morphométrie osseuse, notamment un meilleur alignement des membres inférieurs, une croissance proportionnelle et une augmentation des dimensions du canal rachidien. L’essai comportait un ratio de randomisation de 2:1 (TransCon CNP: placebo) sur 52 semaines, suivi d’une période d’extension en ouvert. Ces résultats soutiennent l’objectif de la société d’apporter des bénéfices au-delà de la croissance linéaire et de réduire potentiellement les complications futures associées à l’achondroplasie.
Ascendis Pharma (ASND) gab positive Daten der Woche 52 aus der entscheidenden ApproaCH-Studie zu TransCon CNP (Navepegritid) bei Kindern mit Achondroplasie bekannt. Die doppelblinde, placebokontrollierte Studie mit 84 Kindern im Alter von 2 bis 11 Jahren zeigte eine überlegene annualisierte Wachstumsgeschwindigkeit im Vergleich zu Placebo. Das Medikament wies ein günstiges Sicherheitsprofil ohne behandlungsbedingte schwerwiegende Nebenwirkungen, symptomatischen Hypotonie, Frakturen oder beschleunigtes Knochenalter auf.
Wesentliche Verbesserungen wurden in der Knochenmorphometrie festgestellt, darunter eine bessere Ausrichtung der unteren Gliedmaßen, proportioniertes Wachstum und vergrößerte Wirbelsäulenkanaldimensionen. Die Studie verwendete ein 2:1-Randomisierungsverhältnis (TransCon CNP:Placebo) über 52 Wochen, gefolgt von einer offenen Verlängerungsphase. Die Ergebnisse unterstützen das Ziel des Unternehmens, Vorteile über das lineare Wachstum hinaus zu bieten und potenziell zukünftige Komplikationen im Zusammenhang mit Achondroplasie zu verringern.
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Insights
Ascendis' TransCon CNP shows positive growth and bone morphometry results in pivotal trial for achondroplasia, demonstrating benefits beyond linear growth.
The Phase 3 ApproaCH Trial for TransCon CNP (navepegritide) has yielded impressive efficacy data at the critical 52-week mark. This once-weekly prodrug demonstrated statistically superior annualized growth velocity compared to placebo in children with achondroplasia, the most common form of disproportionate short stature.
What's particularly noteworthy is that the improvements extend beyond simply increasing height. The data revealed meaningful bone morphometry improvements, including better lower limb alignment, proportional growth, and increased spinal canal dimensions. These structural changes could potentially address core pathological features of achondroplasia.
The safety profile appears robust with several key findings that differentiate it from other treatments in development: no treatment-related serious adverse events, absence of symptomatic hypotension (a concern with CNP-based therapies), no fractures, and no acceleration of bone age versus chronological age. The low rate of injection site reactions is particularly important for a chronic pediatric therapy requiring weekly administration.
The trial design incorporated a rigorous methodology with 84 children randomized 2:1 (treatment:placebo) and included comprehensive assessment measures. The dual presentation at ESPE & ESE 2025 provides scientific validation through peer review at a prestigious endocrinology conference.
These results position TransCon CNP to potentially address multiple clinical manifestations of achondroplasia beyond height, including complications like spinal stenosis that can cause significant disability. This comprehensive approach could differentiate Ascendis' therapy in an increasingly competitive therapeutic landscape for skeletal dysplasias.
COPENHAGEN, Denmark, May 13, 2025 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced new data showing improvements in growth and bone morphometry from Week 52 of its pivotal ApproaCH Trial of TransCon CNP (navepegritide) in children with achondroplasia. TransCon CNP is an investigational prodrug of C-type natriuretic peptide (CNP) administered once weekly providing continuous exposure of active CNP to receptors on tissues throughout the body, including growth plates and skeletal muscle.
The bone morphometry data were shared in an oral presentation on May 12 by Leanne Ward, M.D., Professor of Pediatrics in the Faculty of Medicine at the University of Ottawa (Canada) and the growth data were shared in an oral presentation on May 13 by Hanne B. Hove, M.D., DMSc., Chief Consultant in the Department of Pediatric & Adolescent Medicine at Copenhagen University Hospital Rigshospitalet (Denmark), during ESPE & ESE 2025, the joint congress of the European Society for Paediatric Endocrinology (ESPE) and the European Society of Endocrinology (ESE).
The double-blind placebo-controlled ApproaCH Trial included 84 children with achondroplasia (aged 2-11 years) randomized 2:1 (TransCon CNP:placebo) for 52 weeks, followed by an open-label extension period. At Week 52 of the trial, TransCon CNP demonstrated superiority over placebo in annualized growth velocity (AGV), with a safety and tolerability profile comparable to placebo that included a low rate of injection site reactions, no treatment-related serious adverse events (SAEs), no cases of symptomatic hypotension, no fractures, and no acceleration of bone age versus chronological age.
Analyses also showed that TransCon CNP improved aspects of bone morphometry at Week 52. This included improvement in lower limb alignment and proportional growth, as well as increases in spinal canal dimensions, versus placebo.
“The observed improvements in growth and bone morphometry seen in this trial support our goal to deliver benefits beyond linear growth,” said Aimee Shu, M.D., Executive Vice President of Endocrine & Rare Disease Medical Science and Chief Medical Officer at Ascendis Pharma. “We look forward to continuing to work with our investigators and the broader achondroplasia community to better understand how changing the trajectory of skeletal dysplasia in childhood could potentially reduce future complications associated with this condition, such as pain, impaired mobility, or the need for surgery.”
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 deformities, 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 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) Ascendis’ goal to deliver benefits beyond linear growth, (ii) Ascendis’ plans to continue to work with investigators and the broader achondroplasia community to better understand how changing the trajectory of skeletal dysplasia in childhood could potentially reduce future complications associated with the condition, (iii) Ascendis’ ability to apply its TransCon technology platform to make a meaningful difference for patients, and (iv) 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.
Ascendis, Ascendis Pharma, the Ascendis Pharma logo, the company logo, and TransCon are trademarks owned by the Ascendis Pharma group. © May 2025 Ascendis Pharma A/S.
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FAQ
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