Ascendis Pharma Charts Path to Global Biopharma Leadership

COPENHAGEN, Denmark – January 09, 2026 – Ascendis Pharma has laid out an ambitious strategic roadmap, signaling its rapid evolution from a specialized biotech to a fully integrated global biopharmaceutical company. In a comprehensive update at the 44th Annual J.P. Morgan Healthcare Conference, the company showcased soaring revenues, a maturing pipeline with blockbuster potential, and a financial profile robust enough to initiate a significant share buyback program, solidifying its position as a formidable player in the industry.

Anchored by its proprietary TransCon® technology platform, Ascendis reported impressive unaudited preliminary financial results for 2025, with total product revenue hitting approximately €683 million. The company projects it will generate around €500 million in operating cash flow in 2026, a testament to its successful commercial execution and growing market presence.

“With our proven TransCon® technology platform, strong R&D capabilities, and a maturing commercial infrastructure and financial profile, Ascendis is rapidly transforming into a leading global biopharma company,” said President and Chief Executive Officer Jan Mikkelsen in a statement released ahead of his presentation.

The Commercial Engine: YORVIPATH and SKYTROFA Drive Growth

The company’s strong financial performance is fueled by the remarkable commercial success of its two flagship products. YORVIPATH® (palopegteriparatide), a treatment for hypoparathyroidism, has emerged as a major revenue driver, generating an estimated €477 million in 2025. The therapy’s strong uptake in the United States is evident, with over 5,300 unique patient enrollments by the end of the year. Beyond the U.S., Ascendis has successfully established a global footprint for the drug, making it available either commercially or through named patient programs in more than 30 countries, with plans to launch in 10 additional countries by the end of 2026.

The success of YORVIPATH addresses a significant unmet need for patients with hypoparathyroidism, a rare endocrine disorder where conventional treatments often fall short. Ascendis is also exploring a once-weekly formulation and pursuing label expansion trials to further solidify the product's market leadership.

Meanwhile, SKYTROFA® (lonapegsomatropin), a long-acting treatment for growth hormone deficiency (GHD), contributed an estimated €206 million in 2025 revenue. A key growth catalyst was its U.S. Food & Drug Administration (FDA) approval in July 2025 for adult GHD, significantly expanding its market beyond its initial pediatric indication. The product’s primary competitive advantage is its once-weekly dosing schedule, which offers a more convenient alternative to the daily injections required by older therapies, potentially improving patient adherence and quality of life. The company is not resting on its laurels, having initiated a Phase 3 basket trial to study SKYTROFA in several other growth-related conditions, including Turner syndrome and idiopathic short stature.

The Next Blockbuster? TransCon CNP Awaits Regulatory Verdict

While its current products thrive, all eyes are on the company's late-stage pipeline, particularly TransCon CNP (navepegritide), a potential first-in-class therapy for pediatric achondroplasia. The condition, the most common form of dwarfism, has limited treatment options. Ascendis is poised for a critical milestone with a PDUFA goal date of February 28, 2026, for a decision from the U.S. FDA. A parallel review in Europe is also advancing, with a decision from the European Medicines Agency (EMA) anticipated in the fourth quarter of 2026.

An approval would position TransCon CNP to compete in a market currently dominated by BioMarin Pharmaceutical's Voxzogo. The global achondroplasia market is projected to grow substantially, and a new, effective therapy could capture a significant share. Ascendis is further bolstering its position in this space with promising data from its combination therapy approach. Topline results from the Phase 2 COACH trial, which combined TransCon CNP with TransCon hGH (SKYTROFA), demonstrated significant improvements in growth velocity and body proportionality in children with achondroplasia. The company is already in discussions with the FDA to advance this combination therapy into a Phase 3 trial, suggesting a long-term strategy to establish a new standard of care.

A Platform for Partnerships and Pipeline Expansion

The engine behind Ascendis’s success is its versatile TransCon technology, a novel drug delivery platform that allows for the controlled release of a parent drug in the body. This versatility has not only spawned its internal pipeline but has also attracted high-profile partners, validating the technology and extending its application into massive therapeutic areas.

A multi-product collaboration with Novo Nordisk aims to develop new therapies for obesity and metabolic diseases, with the lead program, TransCon semaglutide, on track to enter clinical trials. This partnership provides Ascendis with a foothold in one of the largest and most competitive markets in medicine. Similarly, a strategic investment in Eyconis, Inc. has given Ascendis a 41% equity stake and a strategic entry into ophthalmology, with a lead candidate for wet age-related macular degeneration expected to enter the clinic in 2026.

Beyond these collaborations, Ascendis has built a robust global expansion strategy through regional partnerships. VISEN Pharmaceuticals holds exclusive rights for Ascendis's core endocrinology products in Greater China, while Teijin Limited is commercializing the portfolio in Japan. These partnerships provide crucial access and local expertise in key Asian markets, accelerating global growth without the cost of building commercial infrastructure from the ground up.

Maturing Financials and Shareholder Confidence

Ascendis’s strategic update paints a picture of a company that has reached financial maturity. Beyond the headline revenue figures, the company expects a strong gross margin of approximately 87% for 2025. The projection of generating around €500 million in operating cash flow in 2026, even without potential contributions from TransCon CNP, underscores the profitability of its core business.

This financial strength is allowing the company to begin returning capital to its investors. The Board of Directors has authorized a $120 million share repurchase program, a move typically associated with larger, more established pharmaceutical companies. Such a program signals management's confidence that its stock is undervalued and reflects a disciplined capital allocation strategy. By reducing the number of shares outstanding, the buyback can boost earnings per share and enhance shareholder value, marking another significant step in Ascendis's journey from a development-stage biotech to a self-sustaining biopharma powerhouse.

With a powerful commercial engine, a de-risked pipeline, and a network of strategic partnerships, Ascendis Pharma has firmly established the foundation for what it believes will be sustainable, long-term growth and a lasting impact on patient care.