Ascendis to Share Its Latest Endocrinology Rare Disease Data at ESPE 2026

Ascendis will showcase new achondroplasia, hypoparathyroidism and growth hormone program data at the ESPE 2026 paediatric endocrinology congress.

Rhea-AI Summary

Ascendis Pharma (ASND) will present new data from its Endocrinology Rare Disease programs at ESPE 2026 in Marseille, France, from September 8-10, 2026.

The company highlights a podium presentation of first sentinel cohort data from the pivotal reACHin trial of TransCon CNP (navepegritide) in infants with achondroplasia aged 0 to <2 years. Additional presentations cover hypoparathyroidism patient-reported outcomes and growth hormone indications, including the HighLiGHts phase 3 trial design of lonapegsomatropin in children with several short-stature conditions and a systematic literature review on pediatric growth hormone deficiency prevalence.

Key Terms

sentinel cohort, achondroplasia, hypoparathyroidism, neuromusculoskeletal

4 terms

sentinel cohort medical

"first sentinel cohort data from the pivotal reACHin Trial"

A sentinel cohort is a small initial group of participants or patients given a new drug, vaccine or treatment and watched closely for safety and early effectiveness before more people are enrolled or the product is rolled out. For investors it matters because results from this ‘test group’ can reveal safety issues or promising signs that shape regulatory approval, development timelines and the company’s valuation—like taking a prototype for a short test drive before full production.

achondroplasia medical

"infants with achondroplasia aged 0 to <2 years"

A genetic condition that causes the most common form of short stature, where a specific change in a growth-control gene makes the long bones grow more slowly, producing a distinct body proportion and sometimes breathing, spinal or joint issues. It matters to investors because it defines a clear patient population, predictable medical needs and regulatory pathways for drugs or devices—similar to a niche market with steady, long-term demand for effective treatments or supportive care.

hypoparathyroidism medical

"treatments for people living with hypoparathyroidism"

A disorder in which small glands in the neck fail to make enough of a hormone that keeps blood calcium at the right level, causing low calcium with symptoms such as muscle cramps, tingling or fatigue. For investors, it matters because chronic, hard-to-manage conditions like this create demand for medicines, medical devices and diagnostic tests, shaping clinical trial needs, regulatory review and potential market opportunity much like a persistent mechanical problem creates ongoing demand for repairs.

neuromusculoskeletal medical

"mitigate the neuromusculoskeletal complications"

Relating to the nerves, muscles and skeleton together as a functional system of the body; it covers how nerve signals control muscles and how those muscles act on bones and joints. Think of it as the body's wiring (nerves), motors (muscles) and frame (bones) working as one. Investors encounter the term when assessing medical devices, drugs or diagnostics that target movement, pain or injury in this combined system because those products' success affects market potential and regulatory paths.

AI-generated analysis. How Rhea-AI works. Not financial advice.

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COPENHAGEN, Denmark, Sept. 07, 2026 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced it will share the latest data from its Endocrinology Rare Disease programs during ESPE 2026, the annual congress of the European Society for Paediatric Endocrinology (ESPE) being held September 8-10, 2026, in Marseille, France. Updates will include a podium presentation of the first sentinel cohort data from the pivotal reACHin Trial of TransCon CNP (navepegritide) in infants with achondroplasia aged 0 to <2 years.

“Ascendis is building leadership with its growing portfolio of treatments for people living with hypoparathyroidism and growth disorders, which we look forward to showcasing at ESPE 2026,” said Aimee Shu, M.D., Executive Vice President, Chief Medical Officer at Ascendis Pharma. “We are especially excited to share these early data from our trial of TransCon CNP in infants with achondroplasia. We believe early medical intervention with TransCon CNP in these youngest children may help mitigate the neuromusculoskeletal complications and co-morbidities associated with this condition.”

Ascendis presentations at ESPE 2026 include:

PODIUM PRESENTATION
Achondroplasia
Tuesday September 8 3:00-4:00p.m. CEST Free Communications 3: Growth & Syndromes Les Goudes 1	Abstract FC4.6 Navepegritide Therapy in Infants with Achondroplasia Less Than 2 Years: Sentinel Data from the reACHin Trial Presented by Genevieve Baujat, M.D.
POSTERS
Hypoparathyroidism
Tuesday-Thursday September 8-10 Exhibition Hall	Poster 1496 Listening to the Patient Voice: Understanding and Assessing the Impact of Hypoparathyroidism on the Daily Lives of Adolescents Authors: S. Ravendeen on behalf of Meryl Brod
	Poster 1534 Understanding and Assessing the Adolescent Patient Perspective of Symptoms in Hypoparathyroidism Authors: S. Ravendeen on behalf of Alden Smith
Growth Hormone Indications
Tuesday-Thursday September 8-10 Exhibition Hall	HighLiGHts Phase 3 Trial Design: Lonapegsomatropin in Children with Short Stature or Growth Failure Due to Turner Syndrome, SHOX Deficiency, Small for Gestational Age, or Idiopathic Short Stature Authors: T. Rohrer et al
e-poster Tuesday-Thursday September 8-10 onsite screens and ESPE meeting platform	Prevelance of Pediatric Growth Hormone Deficiency: A Systematic Literature Review Authors: S. Ravendeen et al

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 within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. Examples of such statements include, but are not limited to, statements relating to (i) Ascendis’ planned podium presentation and posters at ESPE 2026, (ii) the first sentinel cohort data from the pivotal reACHin Trial of TransCon CNP (navepegritide) in infants with achondroplasia aged 0-2 years, (iii) Ascendis’ ability to build leadership with its growing portfolio of treatments for people living with hypoparathyroidism and growth disorders, (iv) the potential for early medical intervention in infants with achondroplasia to help mitigate some of the more serious complications and comorbidities associated with this condition, (v) Ascendis’ clinical development activities, including the Phase 3 trial design for lonapegsomatropin in children with short stature or growth failure due to Turner syndrome, SHOX deficiency, small for gestational age, or idiopathic short stature, (vi) Ascendis’ ability to apply its TransCon technology platform to make a meaningful difference for patients and (vii) Ascendis’ use of TransCon to create new and potentially best-in-class therapies 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, without limitation: dependence on third‑party manufacturers, distributors, and service providers for Ascendis’ products and product candidates; risks related to regulatory review and approval, including the possibility of delays, requests for additional data or analyses, restrictions or limitations on use, approval with labeling that is more limited than expected, or failure to obtain approval in the United States, European Union, or other jurisdictions; clinical development risks, including that results from ongoing or future trials may not confirm earlier data; unforeseen safety or efficacy findings in development programs or on‑market products; manufacturing, supply chain, quality, or logistics issues that could delay development or commercialization; unforeseen expenses related to commercialization of any approved Ascendis products; unforeseen research and development or selling, general and administrative expenses and other costs impacting Ascendis’ business generally; market acceptance, pricing, and reimbursement challenges, including payer coverage decisions and health technology assessments; competitive developments, including new or improved therapies; intellectual property protection, freedom‑to‑operate, and litigation risks; Ascendis’ ability to obtain additional funding, if needed, to support its business activities; cybersecurity, data privacy, and information technology disruptions; and the impact of international economic, political, legal, compliance, public health, and business factors, including tariffs, trade policies, currency fluctuations, and geopolitical events. 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 11, 2026, 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. © September 2026 Ascendis Pharma A/S.

Investor Contacts:	Media Contact:
Chad Fugere	Melinda Baker
Ascendis Pharma	Ascendis Pharma
+1 (650) 519-7494	+1 (650) 709-8875

FAQ

When and where is ESPE 2026 being held?

ESPE 2026, the annual congress of the European Society for Paediatric Endocrinology, is scheduled for September 8-10, 2026, in Marseille, France.

What is the focus of Ascendis Pharma’s podium presentation at ESPE 2026?

The podium presentation will share first sentinel cohort data from the pivotal reACHin trial of TransCon CNP (navepegritide) in infants with achondroplasia less than 2 years old.

Which hypoparathyroidism topics will Ascendis address at ESPE 2026?

Ascendis will present posters on understanding and assessing the impact of hypoparathyroidism on the daily lives of adolescents and on the adolescent patient perspective of symptoms in hypoparathyroidism.

What growth hormone-related work is Ascendis presenting?

The company will present the HighLiGHts phase 3 trial design of lonapegsomatropin in children with short stature or growth failure due to Turner syndrome, SHOX deficiency, small for gestational age, or idiopathic short stature, and an e-poster on the prevalence of pediatric growth hormone deficiency based on a systematic literature review.