New InsiGHTS Trial of TransCon® hGH (Lonapegsomatropin) in Turner Syndrome Demonstrated Comparable Efficacy and Safety to Daily Somatropin at Week 52

- Annualized height velocity of 9.05 cm/year (LS mean) for TransCon hGH-treated children was similar at Week 52 to daily somatropin-treated children 

- TransCon hGH showed a safety and tolerability profile comparable to daily somatropin with no occurrences of slipped capital femoral epiphysis (SCFE)

COPENHAGEN, Denmark, March 17, 2026 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (NASDAQ:ASND) today announced positive Week 52 topline results from New InsiGHTS, its Phase 2 randomized, open-label, active-controlled trial in the U.S. investigating the safety, tolerability, and efficacy of once-weekly TransCon hGH (lonapegsomatropin; U.S. FDA-approved for pediatric and adult growth hormone deficiency (GHD) and approved in other territories for pediatric GHD) 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. They were treated with either TransCon hGH or daily somatropin. Doses were subsequently individualized based on IGF-1.

• At Week 52, children treated with TransCon hGH demonstrated improved annualized height velocity (AHV) similar to daily somatropin, independent of starting dose, with an LS mean AHV of 9.05 cm/year for all TransCon hGH-treated children, compared to 9.04 cm/year for those treated with daily somatropin.
• At Week 52, the mean dose for TransCon hGH was 0.22 mg/kg/week, while the mean dose for the daily somatropin cohort was 0.29 mg/kg/week.

• In the trial, TransCon hGH demonstrated a safety and tolerability profile similar to daily somatropin through follow-up of up to 143 weeks. Adverse events (AEs) were mild to moderate in severity, with no AEs leading to discontinuation of study drug. There were no occurrences of slipped capital femoral epiphysis (SCFE), consistent with the low rate of occurrence (<1%) in long-term safety data of daily somatropin use in Turner syndrome from published literature^1,2.
  
  
  
  

"These new results demonstrated safety and efficacy comparable to daily growth hormone with up to 143 weeks of follow-up, and support the potential of TransCon hGH as a differentiated therapy for short stature in the setting of growth hormone sufficiency and is being further studied in our recently initiated Phase 3 HighLiGHts basket trial to support label expansion," said Jan Mikkelsen, Ascendis Pharma's President and Chief Executive Officer.

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 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) the potential of TransCon hGH as a differentiated therapy for short stature in the setting of growth hormone sufficiency, (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, 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, and TransCon are trademarks owned by the Ascendis Pharma group. © March 2026 Ascendis Pharma A/S.

¹Darendeliler F, Karagiannis G, Wilton P. Horm Res. 2007;68 Suppl 5:41-47. doi:10.1159/000110474

²Bell J, Parker KL, Swinford RD, et al. J Clin Endocrinol Metab 2010;95(1):167–177. doi:10.1210/jc.2009-0178.