Ascendis Pharma A/S presented financial results and operational updates at the 44th Annual J.P. Morgan Healthcare Conference. Chief Executive Officer Jan Mikkelsen reported product revenue of approximately €240 million for the fourth quarter and about €683 million for the full year. He stated the company has achieved two consecutive quarters of profitability.
Mikkelsen attributed the revenue primarily to two commercial products, Yorvipath and Skytrofa. He noted an upcoming regulatory decision for TransCon CNP, with a PDUFA date set for the end of February. The CEO expressed confidence in reaching more than €5 billion in revenue by 2030, with significant contributions expected from both U.S. and international markets.
Regarding Yorvipath, Mikkelsen provided market size estimates, citing 70,000 to 90,000 patients in the U.S. and 150,000 to 200,000 in Europe for hypoparathyroidism. The company reported generating approximately 5,300 unique U.S. prescriptions from nearly 2,400 prescribers, with market penetration remaining below 5%. Launches have occurred in Germany, Austria, and Spain, with Spain starting in the third quarter. Up to 10 additional European countries are expected to launch Yorvipath in 2026.
Internationally, Ascendis currently supplies through named-patient programs but has distribution agreements covering about 75% of countries. Partner Teijin has launched the drug in Japan following approval. During a question-and-answer session, a commercial leader named Jay outlined three U.S. growth priorities for 2026: provider education, patient activation, and access and patient support. Jay emphasized that payer review processes require time, patience, and continued education.
Mikkelsen discussed development of a once-weekly PTH option, estimating 35% to 40% of patients could be suitable after titration on daily Yorvipath. He said the weekly candidate is designed to be bioequivalent across the week to the daily version. Research and development leader Kennett explained that new linker development was undertaken to match Yorvipath's exposure profile. Mikkelsen stated he believes Yorvipath could reach $5 to $8 billion in worldwide peak sales.
For Skytrofa, Mikkelsen said the drug is approved for pediatric growth hormone deficiency, with the U.S. label expanded in 2025 to include adult growth hormone deficiency. The company is conducting a basket trial across four additional indications to expand into established growth hormone markets.
Regarding TransCon CNP for achondroplasia, Mikkelsen cited reported findings including enhanced linear growth, benefit beyond linear growth versus placebo, no evidence of hypertension, low immunogenicity, no injection site reactions, and once-weekly administration. He said durable response has been observed for up to three years in an open-label extension, with an EMA decision expected in the fourth quarter following submission.
Mikkelsen outlined a combination approach with growth hormone, describing it as a way to boost response. He presented combination-trial observations including changes in growth velocity and cited improvements in body proportionality and arm span. A Phase 3 trial has been initiated, with the protocol signed and patient enrollment starting in the first quarter. Week 78 core data are expected in the second quarter.
Mikkelsen provided select financial figures, including operating expenses of €762 million and a cash balance of €615 million. He said the company expects operating cash flow of at least €500 million and plans to buy back at least $120 million in shares in 2026.
He listed several 2026 milestones, including an expected TransCon CNP approval decision in the first quarter, 78-week combination-trial data in the second quarter, and plans to add at least 10 additional countries for Yorvipath launches. Mikkelsen concluded that Ascendis has built a global commercial organization over the last two years and believes it is on the edge of becoming a leading biopharma company.