Ascendis Pharma A/S (NASDAQ:ASND) used its fourth-quarter 2025 earnings call to highlight continued commercial growth for its two marketed rare endocrine disease products, updates across its growth-disorder pipeline, and its expectation to reach operating cash flow of around €500 million in 2026. Management also reiterated a long-term ambition of at least €5 billion in annual product revenue by 2030 as the company expands indications and geographies for current products and advances additional TransCon programs.
YORVIPATH (TransCon PTH) launch momentum and label expansion plans
CEO Jan Møller Mikkelsen said the fourth quarter marked “strong execution” for YORVIPATH, which is approved in the U.S. for hypoparathyroidism in adults and has marketing authorization in the EU and U.K. Ascendis reported fourth-quarter YORVIPATH revenue of €187 million, bringing full-year 2025 YORVIPATH revenue to €477 million.
Outside the U.S., Ascendis said YORVIPATH is available commercially or through named patient programs in more than 30 countries, with full commercial reimbursement in four Europe Direct markets and two international markets. In Japan, partner Teijin launched YORVIPATH commercially in November 2025. Looking ahead, management said it expects full commercial launches in 10 additional new countries in 2026.
Ascendis also discussed efforts to broaden the product’s label. In the U.S., it is working to expand the range of doses studied in the PaTHway trial, and it is advancing trials intended to expand use to patients under 18 globally.
Once-weekly PTH candidate and management’s view on potential competition
Management highlighted ongoing work on a once-weekly TransCon PTH candidate aimed at patients stabilized on daily therapy. Mikkelsen referenced preclinical data shared at the J.P. Morgan Healthcare Conference that, according to the company, support a target profile of matching the released PTH exposure achieved with daily YORVIPATH over an entire week, with comparable efficacy and safety.
In response to a question about a potential competitive entrant (Encaleret) in chronic hypoparathyroidism, Mikkelsen argued that a calcium-sensing approach would not serve as hormone replacement therapy for patients missing PTH and said Ascendis has not factored such a product into its long-term outlook for YORVIPATH.
SKYTROFA growth and expanded growth-hormone development
Ascendis reported fourth-quarter SKYTROFA revenue of €53 million and full-year 2025 SKYTROFA revenue of €206 million. SKYTROFA is approved in the U.S. for pediatric growth hormone deficiency and adult growth hormone deficiency. Management said SKYTROFA’s overall U.S. market share is around 7%.
During the quarter, the company initiated a Phase 3 basket trial to evaluate TransCon Growth Hormone in additional established growth hormone indications, including:
- Idiopathic short stature (ISS)
- SHOX deficiency
- Turner syndrome
- Small for gestational age (SGA)
Management said these indications comprise up to half of the growth hormone market and represent a meaningful opportunity to expand SKYTROFA’s role over time.
TransCon CNP regulatory review, launch expectations, and combination data in achondroplasia
Mikkelsen described TransCon CNP as expected to be the first once-weekly treatment for children with achondroplasia and emphasized outcomes beyond linear growth, including improvement in leg bowing versus placebo and increased spinal canal dimension in its pivotal trial, along with a safety and tolerability profile described as similar to placebo. He also noted a “very low rate” of injection site reactions and no cases of symptomatic hypotension in that trial.
In the U.S., Ascendis said its NDA remains under review with a PDUFA date of Feb. 28. In the EU, the company said the MAA review is underway following an October submission, with a decision expected in Q4 2026. Management added that recruitment in its trial in infants with achondroplasia is progressing, with full enrollment anticipated later in 2026.
Asked about confidence heading into the PDUFA date, Mikkelsen responded that he expects TransCon CNP will be approved, drawing a comparison to his prior confidence ahead of the TransCon PTH decision.
The company also discussed 52-week Phase 2 combination data evaluating TransCon CNP plus TransCon Growth Hormone in achondroplasia. Management said the combination showed improvements in achondroplasia-specific height scores that were three to four times those seen in monotherapy trials over the same period, along with accelerated improvements in body proportionality and a reported improvement in arm span, without compromising safety or tolerability. Executives said all children completed 52 weeks and remain in the trial. Ascendis said it held end-of-Phase 2 meetings with the FDA and EU scientific advice meetings to align on a Phase 3 trial design, and it expects additional COACH trial updates at week 78 by mid-year and week 104 by year-end.
On commercialization outside the U.S., Mikkelsen said the company’s global infrastructure—built through YORVIPATH’s launch—positions it to launch TransCon CNP broadly, and he noted that partners such as Teijin (Japan) and VISEN (China) have collaborations covering multiple products. Management did not provide specific U.S. uptake cadence details, but emphasized product attributes such as once-weekly dosing, injection-site reaction expectations versus daily therapy, and broader efficacy outcomes. In a separate response, management said TransCon CNP was not included in the company’s €500 million operating cash flow expectation for 2026, and the company would provide more guidance after seeing early launch dynamics.
Financial results, cash position, and 2026 outlook commentary
CFO Scott Smith reviewed results that management said were largely pre-announced at J.P. Morgan. Total Q4 2025 revenue was €248 million and full-year 2025 revenue was €720 million. The company reported Q4 operating expenses of €214 million and full-year 2025 operating expenses of €761 million. Ascendis posted Q4 operating profit of €10 million and Q4 operating cash flow of €73 million.
Smith noted that below operating profit, results were influenced by non-cash accounting related to convertible notes. Net finance expense was driven primarily by non-cash items, including a remeasurement loss of financial liabilities of €106 million, resulting in €93 million net finance expense. He added that net cash financial expense for full-year 2025 was about €8 million, and that the company may introduce a non-IFRS EPS measure in future periods to improve comparability.
Ascendis ended 2025 with €616 million in cash and cash equivalents, up from €560 million at the end of 2024.
For 2026 modeling, management said it expects:
- YORVIPATH to continue strong revenue growth in 2026 with some seasonality.
- SKYTROFA to follow a similar seasonal pattern as 2025, with revenue growth tracking prescription growth, and longer-term growth supported by geographic and label expansion.
On expenses, Smith said using Q4 operating expenses as a run rate for 2026 is “not a bad way” to think about the year, while noting the company would update investors if expectations change.
About Ascendis Pharma A/S (NASDAQ:ASND)
Ascendis Pharma A/S is a Denmark‐based biopharmaceutical company focused on developing innovative therapies for rare endocrine diseases. Founded in 2015 and headquartered in Hellerup, the company leverages its proprietary TransCon drug delivery platform to create long‐acting prodrugs designed to improve safety, efficacy and patient convenience. Ascendis Pharma maintains research and development operations in Europe and the United States, with clinical studies spanning North America, Europe and Asia.
The company’s lead product, lonapegsomatropin (Skytrofa®), is a once‐weekly growth hormone therapy approved by the U.S.
