On January 9, 2026 Ascendis Pharma A/S (Nasdaq: ASND) reported a business and strategic roadmap update, including planned 2026 key corporate milestones. Ascendis President and CEO Jan Mikkelsen will present this update on Monday, January 12, during the 44th Annual J.P. Morgan Healthcare Conference.
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"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 Jan Mikkelsen, President and Chief Executive Officer at Ascendis Pharma. "Over the next year, this inflection will be driven by further global penetration and commercial expansion, a third potential TransCon product approval with blockbuster potential, and an advancing R&D pipeline of highly differentiated internal and external TransCon product candidates, all of which create the foundation for sustainable long-term growth."
Selected Key Updates and Milestones
TransCon PTH
(palopegteriparatide, marketed as YORVIPATH)
YORVIPATH fourth-quarter 2025 and full-year 2025 unaudited preliminary revenue expected to be ~€187 million and ~€477 million, respectively.
More than 5,300 unique U.S. patient enrollments, with nearly 2,400 unique prescribing healthcare providers at year end.
Outside the U.S., now available commercially or through named patient programs in more than 30 countries, with full commercial launches anticipated in 10 additional countries by year end 2026.
Confirmed product profile for once-weekly TransCon PTH targeting patients receiving stable YORVIPATH doses.
Ongoing label expansion trials through PaTHway60 (adults) and PaTHway Adolescent.
TransCon hGH
(lonapegsomatropin, marketed as SKYTROFA)
SKYTROFA fourth-quarter 2025 and full year 2025 unaudited preliminary revenue expected to be ~€53 million and ~€206 million, respectively.
Received first label expansion in July 2025 with U.S. Food & Drug Administration (FDA) approval for adult growth hormone deficiency (GHD).
Initiated Phase 3 basket trial for additional indications: idiopathic short stature (ISS), SHOX deficiency, Turner syndrome, and small for gestational age (SGA).
TransCon CNP
(navepegritide)
In the U.S., PDUFA goal date of February 28, 2026 for pediatric achondroplasia.
Submitted marketing authorization application (MAA) to the European Medicines Agency (EMA) in October 2025, with a regulatory decision on potential use in pediatric achondroplasia anticipated in the fourth quarter of 2026.
TransCon CNP + TransCon hGH Combination Therapy
(navepegritide plus lonapegsomatropin)
On January 8, 2026, announced Week 52 topline results from Phase 2 COACH Trial, which demonstrated improvements in annualized growth velocity (AGV) across both TransCon CNP treatment-naïve and TransCon CNP-treated children that exceeded the 97th percentile of average stature children, along with improvements in body proportionality and arm span, and a safety profile consistent with those observed for monotherapies of TransCon CNP and TransCon hGH.
In Q4 2025, Ascendis submitted a protocol and held an end of Phase 2 meeting with the FDA regarding a Phase 3 trial of TransCon CNP and TransCon hGH in pediatric achondroplasia.
Week 78 COACH data update anticipated in second quarter of 2026.
Planned new trials to support TransCon CNP + TransCon hGH treatment in additional indications, such as hypochondroplasia.
TransCon IL-2 β/γ
(onvapegleukin alfa)
Expect to report median overall survival (OS) data for a cohort of 70 patients with late-line platinum-resistant ovarian cancer (PROC) from the IL-Believe Trial of TransCon IL-2 β/γ + weekly paclitaxel in the second quarter of this year.
Strategic Collaborations & Investments
Novo Nordisk A/S
Ongoing multi-product collaboration with Novo Nordisk for TransCon technology-based therapies in obesity and metabolic diseases.
Lead program TransCon semaglutide remains on track to enter the clinic as anticipated.
Eyconis, Inc.
Ascendis has granted Eyconis exclusive rights to develop and commercialize TransCon ophthalmology products globally and holds a 41% equity position in the company.
Lead program TransCon aVEGF (EYC-0305) in development for wet AMD and other retinal diseases anticipated to enter the clinic in 2026.
VISEN Pharmaceuticals
Ascendis has granted VISEN Pharmaceuticals exclusive rights to develop and commercialize TransCon hGH, TransCon PTH, and TransCon CNP in Greater China and maintains a 39% ownership interest in the public company.
Biologic License Application (BLA) for TransCon hGH (lonapegsomatropin) as a potential treatment for pediatric growth hormone deficiency (PGHD) was accepted by China’s National Medical Products Administration (NMPA) in March 2024, with an approval decision anticipated in the first quarter of 2026.
Teijin Limited
Ascendis has granted Teijin Limited an exclusive license agreement for the further development and commercialization of TransCon hGH, TransCon PTH, and TransCon CNP for rare endocrinology diseases in Japan.
In August 2025, YORVIPATH received MAA approval from Japan’s Ministry of Health, Labour, and Welfare.
Financial Update
Unaudited preliminary estimate of 2025 financial results:
Total full-year 2025 product revenue of ~€683 million:
YORVIPATH full-year 2025 revenue of ~€477 million
SKYTROFA full-year 2025 revenue of ~€206 million
Total full-year 2025 revenue of ~€720 million
Full-year 2025 gross margin expected to be ~87%
Total full-year 2025 operating expenses of ~€762 million
December 31, 2025 unaudited preliminary cash balance of ~€616 million
Expect operating cash flow of ~€500 million in 2026, based on current plans, exchange rates and excluding any contribution from TransCon CNP
Planned $120 million share repurchase program in 2026
Share Repurchase Program
The Board of Directors of Ascendis has authorized a $120 million Share Repurchase Program. Purchases under the Share Repurchase Program may be made from time to time through a variety of methods, which may include open-market purchases, privately negotiated transactions, or other methods permitted under applicable securities laws. The timing and amount of any repurchases pursuant to the Share Repurchase Program will be determined based on market conditions, share price and other factors. The Share Repurchase Program does not require Ascendis to repurchase any specific number of shares, and may be modified, suspended or terminated at any time without notice.
Presentation at J.P. Morgan Healthcare Conference on Monday, January 12
A live webcast of the event will be available via the Investors & News section of the Ascendis Pharma website at View Source The presentation will begin at 10:30 a.m. Eastern Time / 7:30 a.m. Pacific Time. A webcast replay will be available for 30 days.
The Company’s slides from the J.P. Morgan presentation will be available on the same Investors & News website at View Source
(Press release, Ascendis Pharma, JAN 9, 2026, View Source [SID1234661888])