Ascendis to Share Latest Endocrinology Rare Disease Data in 3 Oral Presentations at ASBMR 2025

Rhea-AI Summary

Ascendis Pharma (NASDAQ:ASND) will present three oral presentations featuring its latest clinical data at the ASBMR 2025 conference in Seattle from September 5-8, 2025. The presentations will showcase new analyses for treatments in achondroplasia and hypoparathyroidism.

Key presentations include:

• New analysis correlating leg bowing improvements with physical functioning in children treated with TransCon CNP
• First presentation of clinical trial data combining TransCon CNP and TransCon hGH therapy in children with achondroplasia
• Clinical data showing 4-year sustained skeletal improvements in adults with hypoparathyroidism treated with TransCon PTH

Insights

Endocrinology Clinical Researcher

Ascendis shares promising clinical data for rare disease treatments at ASBMR 2025, demonstrating therapeutic progress across multiple endocrine conditions.

Ascendis Pharma's upcoming presentations at ASBMR 2025 represent significant clinical progress for their rare endocrine disease portfolio. The three oral presentations highlight critical advancements in both achondroplasia and hypoparathyroidism treatments.

For achondroplasia, they're presenting a new analysis correlating leg bowing improvements with enhanced physical functioning in children treated with navepegritide (TransCon CNP). This establishes an important connection between anatomical improvements and functional outcomes, strengthening the clinical value proposition of their therapy.

Perhaps more notably, Ascendis is unveiling the first-ever medical congress presentation of combination therapy data using both TransCon CNP and TransCon hGH (lonapegsomatropin) in children with achondroplasia. Combination approaches could potentially offer synergistic benefits beyond monotherapy, addressing multiple aspects of this complex growth disorder.

For hypoparathyroidism, their presentation demonstrates sustained improvements in skeletal dynamics over a remarkable 4-year (214-week) treatment period with palopegteriparatide (TransCon PTH). Such long-term data is particularly valuable for chronic conditions requiring lifelong management.

The caliber of presenting investigators—from leading institutions in Canada, Ireland, and the United States—lends additional credibility to these findings. The consistent positive messaging around these therapies suggests Ascendis is building a compelling case for their TransCon technology platform across multiple endocrine indications.

Biotechnology Investment Analyst

Ascendis strengthens clinical validation for multiple rare disease programs with promising data presentations, supporting potential market expansions.

Ascendis Pharma's upcoming presentations at ASBMR 2025 represent meaningful clinical validation for their TransCon technology platform across multiple rare endocrine indications. The company is effectively executing a dual-franchise strategy in achondroplasia and hypoparathyroidism.

For the achondroplasia program, two key developments stand out. First, the correlation between improved leg bowing and physical functioning from the ApproaCH trial provides important patient-centric outcomes data that supports the clinical meaningfulness of TransCon CNP treatment. Second, and perhaps more strategically significant, is the presentation of combination therapy data from the Phase 2 COACH trial, which could potentially expand their market opportunity in achondroplasia through a differentiated treatment approach.

The hypoparathyroidism data extends treatment benefits to 214 weeks (over 4 years), which is crucial for chronic conditions requiring lifelong management. This long-term efficacy data strengthens TransCon PTH's clinical profile and potential market position.

The selection of these data for oral presentations (rather than just posters) at a respected medical conference indicates their scientific significance and potential impact on treatment paradigms. Importantly, these presentations showcase Ascendis' ability to leverage their TransCon technology platform across different therapeutic proteins (CNP, hGH, and PTH), demonstrating the platform's versatility.

The involvement of established clinical investigators from renowned institutions adds further credibility and helps build key opinion leader support, which is essential for clinical adoption of novel therapies in rare diseases where treatment networks are often highly specialized and concentrated.

COPENHAGEN, Denmark, Sept. 02, 2025 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced three oral presentations of its latest achondroplasia and hypoparathyroidism data at ASBMR 2025, the annual meeting of the American Society for Bone & Mineral Research being held in Seattle from September 5-8, 2025. These include a new analysis of pivotal trial data correlating improvements in leg bowing with improved physical functioning in children with achondroplasia treated with TransCon^® CNP (navepegritide); the first medical congress presentation of clinical trial data for children with achondroplasia treated with TransCon CNP and TransCon hGH (lonapegsomatropin) combination therapy; and clinical trial data demonstrating the sustained improvements in skeletal dynamics in adults with hypoparathyroidism treated for 4 years with TransCon PTH (palopegteriparatide).

Achondroplasia
Saturday September 6 12:00 – 12:15 pm General Session Room 6BC	Oral Presentation Abstract 1039 (See also poster PM-155.) Improvements in Lower Extremity Alignment Are Associated with Physical Functioning in Children with Achondroplasia Treated with Navepegritide: 52-Week Results From the ApproaCH Trial Presented by Leanne Ward, M.D. Children’s Hospital of Eastern Ontario, University of Ottawa (Canada)
Monday September 8 11:15 – 11:30 am General Session Room 6BC	Oral Presentation Abstract 1126 (See also poster PM-118.) Once-Weekly Lonapegsomatropin (TransCon hGH) Added to Once-Weekly Navepegritide (TransCon CNP) in Children with Achondroplasia: 26-Week Results from the Phase 2 COACH Trial Presented by Ciara McDonnell, M.D. Children’s Health Ireland at Temple Street and University of Dublin, Trinity College (Ireland)​
Hypoparathyroidism
Saturday September 6 11:45 am – 12:00 pm General Session Room 6BC	Oral Presentation Abstract 1038 Improved Skeletal Dynamics in Adults Treated with Palopegteriparatide for Chronic Hypoparathyroidism: 214-Week Results from the Phase 2 PaTH Forward Trial Presented by Mishaela Rubin, M.D. Columbia University (United States)

“We are pleased to continue our partnership with these renowned investigators and believe that our latest data reinforces the transformative potential of the mono- and combination therapies that form the foundation of our growth disorder portfolio,” said Aimee Shu, M.D., Executive Vice President of Endocrine & Rare Disease Medical Sciences and Chief Medical Officer at Ascendis Pharma. “And, through this presentation of long-term skeletal dynamics data in the United States, we are pleased to reinforce the long-term benefits of our groundbreaking treatment for adults with hypoparathyroidism.”

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 abnormalities, 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 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 (70-80%), while other etiologies include autoimmune and idiopathic causes.

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 transformative potential and long-term benefits of Ascendis’ growth disorder portfolio, (ii) Ascendis’ ability to apply its TransCon technology platform to make a meaningful difference for patients, and (iii) Ascendis’ ability to apply TransCon 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. © September 2025 Ascendis Pharma A/S.

Investor Contacts: Chad Fugere Ascendis Pharma ir@ascendispharma.com	Media Contact: Melinda Baker Ascendis Pharma media@ascendispharma.com
Patti Bank ICR Healthcare +1 (415) 513-1284 patti.bank@icrhealthcare.com

FAQ

When and where will Ascendis Pharma present at ASBMR 2025?

Ascendis Pharma will present at ASBMR 2025 in Seattle from September 5-8, 2025, with presentations scheduled for September 6 and 8.

What clinical data will Ascendis Pharma (ASND) present for achondroplasia treatment?

Ascendis will present data on leg bowing improvements with TransCon CNP and the first clinical trial results of combination therapy using TransCon CNP with TransCon hGH in children with achondroplasia.

What are the latest results for Ascendis Pharma's hypoparathyroidism treatment?

The presentation will show 214-week (4-year) clinical trial data demonstrating sustained improvements in skeletal dynamics in adults treated with TransCon PTH (palopegteriparatide).

Who will be presenting the Ascendis Pharma (ASND) clinical data at ASBMR 2025?

The presentations will be given by Dr. Leanne Ward from University of Ottawa, Dr. Ciara McDonnell from University of Dublin, and Dr. Mishaela Rubin from Columbia University.