Ascendis Pharma A/S (NASDAQ:ASND) highlighted accelerating product revenue growth, expanding international commercialization, and multiple 2026 clinical and regulatory milestones during a presentation and Q&A at the 44th Annual J.P. Morgan Healthcare Conference.
Revenue growth and profitability focus
Chief Executive Officer Jan Mikkelsen said Ascendis reported Q4 product revenue of about €240 million and full-year product revenue of about €683 million, describing the company as having achieved “two quarters of a profitable business.” He attributed the revenue base primarily to two commercial products, Yorvipath (TransCon PTH) and Skytrofa, and pointed to an upcoming regulatory decision for TransCon CNP with a PDUFA date at the end of February.
Yorvipath: U.S. penetration, ex-U.S. expansion, and weekly development
On Yorvipath, Mikkelsen presented market sizing estimates for hypoparathyroidism, citing 70,000–90,000 patients in the U.S., 150,000–200,000 in Europe, and a “huge” international market. He said revenue growth has been driven mainly by the U.S. so far, while ex-U.S. growth should expand over time as more countries launch and market penetration increases.
In a commercial update, Ascendis said it has generated 5,300 unique U.S. prescriptions from nearly 2,400 prescribers, and that penetration remains below 5%, which management characterized as leaving “a unique opportunity.” In Europe, Ascendis said it has launched in Germany, Austria, and Spain (with Spain starting in Q3), and expects up to 10 additional European countries to come online in 2026. Internationally, Ascendis said it currently supplies via named-patient programs but has distribution agreements covering about 75% of countries, and noted partner Teijin has launched in Japan following approval.
During Q&A, executive Jay (identified as a commercial leader) described three U.S. growth priorities for 2026:
- Provider education to broaden prescriber adoption and expand perceptions of eligible patients beyond conventional therapy.
- Patient activation, including digital outreach to patients without established care.
- Access and patient support to improve the treatment experience in a fragmented payer system.
Asked about reimbursement timelines, Jay said the company’s strategy is not expected to change materially, emphasizing that payer review processes require “time, patience, discipline,” and continued education, while noting the company feels good about coverage and approval rates early in launch.
Mikkelsen also discussed development of a once-weekly PTH option. He said Ascendis views weekly dosing as most appropriate for “stable” patients after titration on daily Yorvipath, estimating 35%–40% of patients could fit that profile. Management said the weekly candidate is designed to be bioequivalent across the week to daily Yorvipath by using the same active ingredient and changing the linker. R&D leader Kennett explained that a first-generation weekly design had a higher peak-to-trough profile, prompting new linker development to match Yorvipath’s exposure variation. In response to a question comparing the weekly product to “MBX,” Mikkelsen said the approaches are different and emphasized Ascendis’ focus on unmodified PTH replacement therapy.
Asked about peak sales expectations, Mikkelsen said he believes Yorvipath could reach $5–$8 billion worldwide.
Skytrofa: label expansion and additional indications
Mikkelsen said Skytrofa is approved for pediatric growth hormone deficiency and that in 2025 Ascendis expanded the label in the U.S. to include adult growth hormone deficiency. He said the company is running a “basket” trial across four additional indications to expand into established growth hormone indications and outlined a broader strategy to grow the franchise by consolidating share from daily growth hormone, expanding geographically, and adding both traditional and novel indications.
TransCon CNP: upcoming decision and combination strategy
For TransCon CNP in achondroplasia, Mikkelsen said the program was designed to provide sustained exposure of wild-type CNP via a prodrug to avoid injection site reactions. He cited reported findings including enhanced linear growth, benefit beyond linear growth versus placebo, no evidence of hypertension, low immunogenicity signals, no injection site reactions, and once-weekly administration. He said the company has observed durable response for up to three years in an open-label extension, with an EMA decision expected in Q4 following submission.
Mikkelsen also outlined a combination approach with growth hormone, describing it as a way to boost response after “removing the brake” of FGFR3 signaling. He presented combination-trial observations including changes in growth velocity and cited improvements in body proportionality and arm span, while emphasizing safety considerations in pediatrics. He said Ascendis has had two informal FDA meetings and has now initiated a Phase 3 trial, with the protocol signed and patient enrollment starting in Q1, and said week 78 core data are expected in Q2.
2026 milestones and capital allocation
Mikkelsen provided select financial and operational figures, including operating expenses of €762 million and a cash balance of €615 million, and said the company expects operating cash flow of at least €500 million. He also said Ascendis plans to buy back at least $120 million in shares in 2026.
He listed several 2026 milestones discussed during the presentation, including:
- Expected TransCon CNP approval decision in Q1 (with the PDUFA date at the end of February).
- 78-week combination-trial data in Q2.
- Plans to add at least 10 additional countries for Yorvipath launches in 2026.
- Additional trial progress updates, including completion of enrollment and recruitment across certain studies referenced in the presentation.
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, emphasizing continued investment in lifecycle management and pipeline development built on the company’s TransCon technology platform.
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.
