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

1. Ascendis Pharma presents new data at ASBMR 2025 in Seattle. 2. Key findings include improvements in children’s leg bowing and physical function. 3. Long-term data shows sustained skeletal improvements in hypoparathyroidism adults. 4. The studies strengthen Ascendis' growth disorder treatment portfolio potential. 5. Executives emphasize the significance of TransCon technology in patient care.

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Why Bullish?

The positive trial data can enhance investor sentiment and likely demand for ASND.

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The findings could attract attention from investors and healthcare providers, influencing ASND's price positively.

Why Long Term?

The sustained improvements may lead to market penetration over time.

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MANPREGNVRTX
September 02, 2025 16:01 ET  | Source: Ascendis Pharma 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). “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 AchondroplasiaAchondroplasia 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 HypoparathyroidismHypoparathyroidism 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/SAscendis 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.

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