New InsiGHTS Trial of TransCon™ hGH (Lonapegsomatropin) in Turner Syndrome Achieved Primary Objective at Week 26
Ascendis Pharma (ASND) announced positive Week 26 topline results from its Phase 2 New InsiGHTS trial, evaluating TransCon hGH (lonapegsomatropin) in Turner syndrome patients. The trial compared three different weekly doses of TransCon hGH against daily somatropin in 49 prepubertal children aged 1-10 years.
The study achieved its primary objective, demonstrating that once-weekly TransCon hGH provided similar growth benefits to daily somatropin, with comparable safety and tolerability profiles. The trial showed improved growth across all three TransCon hGH starting dose cohorts (0.24, 0.30, or 0.36 mg/kg/week), matching the performance of daily somatropin (0.35 mg/kg/week).
Ascendis Pharma (ASND) ha annunciato risultati positivi della settimana 26 dallo studio di fase 2 New InsiGHTS, che valuta TransCon hGH (lonapegsomatropin) in pazienti con sindrome di Turner. Lo studio ha confrontato tre diverse dosi settimanali di TransCon hGH con il somatropin giornaliero in 49 bambini prepuberi di età compresa tra 1 e 10 anni.
Lo studio ha raggiunto il suo obiettivo primario, dimostrando che TransCon hGH una volta a settimana ha fornito benefici di crescita simili a quelli del somatropin giornaliero, con profili di sicurezza e tollerabilità comparabili. L'esperimento ha mostrato una crescita migliorata in tutti e tre i gruppi di dosaggio iniziali di TransCon hGH (0,24, 0,30 o 0,36 mg/kg/settimana), allineandosi alle prestazioni del somatropin giornaliero (0,35 mg/kg/settimana).
Ascendis Pharma (ASND) anunció resultados positivos de la semana 26 de su ensayo de fase 2 New InsiGHTS, evaluando TransCon hGH (lonapegsomatropin) en pacientes con síndrome de Turner. El ensayo comparó tres dosis semanales diferentes de TransCon hGH con somatropin diario en 49 niños prepubescentes de 1 a 10 años.
El estudio logró su objetivo principal, demostrando que TransCon hGH una vez a la semana proporcionó beneficios de crecimiento similares a los del somatropin diario, con perfiles de seguridad y tolerabilidad comparables. El ensayo mostró un crecimiento mejorado en los tres grupos de dosis iniciales de TransCon hGH (0.24, 0.30 o 0.36 mg/kg/semana), igualando el rendimiento del somatropin diario (0.35 mg/kg/semana).
Ascendis Pharma (ASND)는 터너 증후군 환자에서 TransCon hGH (lonapegsomatropin)을 평가하는 2상 New InsiGHTS 시험의 26주 긍정적인 주요 결과를 발표했습니다. 이 시험은 1세에서 10세 사이의 49명의 사춘기 이전 아동을 대상으로 TransCon hGH의 세 가지 다른 주간 용량을 하루 한 번 투여하는 소마트로핀과 비교했습니다.
이번 연구는 주 1회 TransCon hGH가 하루 소마트로핀과 유사한 성장 이점을 제공하고, 안전성 및 내약성 프로필이 비슷하다는 것을 증명하여 주요 목적을 달성했습니다. 이 시험은 TransCon hGH의 세 가지 초기 용량 그룹(0.24, 0.30 또는 0.36 mg/kg/주) 전반에 걸쳐 성장 개선을 보였으며, 하루 소마트로핀(0.35 mg/kg/주)의 성능과 일치했습니다.
Ascendis Pharma (ASND) a annoncé des résultats positifs de la semaine 26 de son essai de phase 2 New InsiGHTS, évaluant TransCon hGH (lonapegsomatropin) chez des patients atteints du syndrome de Turner. L'essai a comparé trois différentes doses hebdomadaires de TransCon hGH à du somatropin quotidien chez 49 enfants prépubères âgés de 1 à 10 ans.
L'étude a atteint son objectif principal, démontrant que TransCon hGH une fois par semaine offrait des bénéfices de croissance similaires à ceux du somatropin quotidien, avec des profils de sécurité et de tolérabilité comparables. L'essai a montré une amélioration de la croissance dans les trois groupes de dose de départ de TransCon hGH (0,24, 0,30 ou 0,36 mg/kg/semaine), correspondant à la performance du somatropin quotidien (0,35 mg/kg/semaine).
Ascendis Pharma (ASND) hat positive Ergebnisse der Woche 26 aus seiner Phase-2-Studie New InsiGHTS angekündigt, die TransCon hGH (lonapegsomatropin) bei Patienten mit Turner-Syndrom untersucht. Die Studie verglich drei verschiedene wöchentliche Dosen von TransCon hGH mit täglichem Somatropin bei 49 präpubertären Kindern im Alter von 1 bis 10 Jahren.
Die Studie erreichte ihr Hauptziel und zeigte, dass wöchentliches TransCon hGH ähnliche Wachstumsgewinne wie tägliches Somatropin lieferte, mit vergleichbaren Sicherheits- und Verträglichkeitsprofilen. Die Studie zeigte eine verbesserte Wachstumsrate in allen drei Anfangsdosierungsgruppen von TransCon hGH (0,24, 0,30 oder 0,36 mg/kg/Woche) und erreichte die Leistung von täglichem Somatropin (0,35 mg/kg/Woche).
- Achieved primary endpoint with comparable efficacy to daily treatment
- Demonstrated favorable safety profile with no drug-related discontinuations
- Successfully expanded potential application beyond growth hormone deficiency
- Positive results support planned basket trial in 2025 for label expansion
- None.
Insights
The positive Phase 2 trial results for TransCon hGH in Turner syndrome represent a significant milestone for Ascendis Pharma. The data demonstrates that the once-weekly treatment matches daily somatropin's efficacy in height velocity and safety profile - a important outcome for potential market expansion. The three dosing cohorts (0.24, 0.30 and 0.36 mg/kg/week) provide valuable insights for optimal dosing strategies.
Most importantly, this trial marks TransCon hGH's first successful venture beyond growth hormone deficiency, validating the platform's versatility. With 49 patients showing positive outcomes and no treatment-related discontinuations, these results strongly position Ascendis for their planned 2025 basket trial aimed at label expansion.
This clinical success significantly strengthens Ascendis Pharma's market position in the growth hormone space. Turner syndrome represents a valuable expansion opportunity beyond the current pediatric growth hormone deficiency indication. The positive safety profile and comparable efficacy to daily treatment reduce regulatory risks for future approvals.
The potential label expansion could substantially increase TransCon hGH's addressable market. The convenience of weekly versus daily administration provides a compelling competitive advantage that could drive market share gains. With planned basket trials in 2025, this positions ASND for potential revenue growth in 2026-2027, assuming successful trial completion and regulatory approval.
- Results for all three TransCon hGH starting dose cohorts, in first clinical trial of an indication outside of growth hormone deficiency, showed a safety and tolerability profile comparable to daily somatropin
- Annualized height velocity was similar at Week 26 in once-weekly TransCon hGH-treated and daily somatropin-treated children
COPENHAGEN, Denmark, Dec. 16, 2024 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced positive Week 26 topline results from New InsiGHTS, its Phase 2 randomized, open-label, active-controlled trial in the U.S. to investigate the safety, tolerability, and efficacy of once-weekly TransCon hGH (lonapegsomatropin; approved for pediatric growth hormone deficiency) compared to daily somatropin in prepubertal children with Turner syndrome.
New InsiGHTS randomized and dosed 49 children with Turner syndrome aged 1 to 10 years old into one of four treatment groups 1:1:1:1 – one of three starting doses of TransCon hGH (0.24, 0.30, or 0.36 mg/kg/week) or an active comparator of daily somatropin with a starting dose of 0.35 mg/kg/week. Doses were individualized based on IGF-1.
- On the primary endpoint of annualized height velocity and secondary endpoint of change from baseline in height SDS, children treated with TransCon hGH demonstrated improved growth similar to daily somatropin at Week 26, independent of starting dose.
- In the trial, TransCon hGH was generally safe and well tolerated, with no discontinuations related to study drug and with comparable safety and tolerability to daily somatropin.
“Week 26 topline data from the New InsiGHTS Trial showed that once-weekly TransCon hGH provided comparable safety and endocrine benefits to those seen with the established use of daily somatropin in Turner syndrome,” said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. “These positive results lay the foundation for our planned basket trial in 2025 to support label expansion.”
Ascendis expects Week 52 results for lonapegsomatropin-treated children compared to daily somatropin-treated children in 2025.
About Turner Syndrome
Turner syndrome is the most common congenital sex chromosomal condition in females, with an estimated prevalence of 1 out of every 2,000 to 2,500 live female births. Short stature, associated with short stature homeobox-containing gene (SHOX) haploinsufficiency, is the most common clinical feature of Turner syndrome. Clinical manifestations of Turner syndrome affect multiple organ systems and are associated with significant and potentially life-threatening complications including cardiovascular disease, ovarian dysfunction, endocrine disease, renal malformation, liver disease, sensorineural hearing loss, and varied neuropsychological manifestations.
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
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 timing of Week 52 results for lonapegsomatropin-treated patients compared to daily somatropin-treated patients, (ii) Ascendis’ plans for a basket trial in 2025 and SKYTROFA’s potential label expansion, (iii) Ascendis’ ability to apply its TransCon technology platform to build a leading, fully integrated biopharma company, and (iv) Ascendis’ use of its TransCon technologies to create new and potentially best-in-class therapies. 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. 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’ prospectus supplement filed on September 20, 2024 and Ascendis’ current and future reports filed with, or submitted to, the U.S. Securities and Exchange Commission (SEC), including its Annual Report on Form 20-F filed with the SEC on February 7, 2024. 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. © December 2024 Ascendis Pharma A/S.
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FAQ
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