Ascendis Pharma Provides Business and Strategic Roadmap Update at 44th Annual J.P. Morgan Healthcare Conference

Rhea-AI Summary

Ascendis Pharma (Nasdaq: ASND) provided a 2026 business and strategic roadmap, commercial updates, regulatory timelines, and unaudited preliminary 2025 financials ahead of its Jan 12 J.P. Morgan presentation.

Key metrics include YORVIPATH 2025 revenue ~€477M, SKYTROFA 2025 revenue ~€206M, total product revenue ~€683M, total revenue ~€720M, gross margin ~87%, operating expenses ~€762M, cash ~€616M at Dec 31, 2025, and a $120M share repurchase program. Regulatory milestones include a TransCon CNP PDUFA date of Feb 28, 2026 and EMA decision expected Q4 2026.

Positive

• Total full-year 2025 product revenue ~€683M
• YORVIPATH 2025 revenue ~€477M
• SKYTROFA 2025 revenue ~€206M
• Unaudited cash balance ~€616M at Dec 31, 2025
• Board-authorized $120M share repurchase program

Negative

• Total full-year 2025 operating expenses ~€762M
• Operating expenses exceed 2025 product revenue (~€762M vs ~€683M)
• TransCon CNP PDUFA decision pending on Feb 28, 2026

Key Figures

YORVIPATH FY25 revenue €477 million Unaudited preliminary full-year 2025 product revenue

SKYTROFA FY25 revenue €206 million Unaudited preliminary full-year 2025 product revenue

Total FY25 product revenue €683 million Unaudited preliminary 2025 product revenue

Total FY25 revenue €720 million Unaudited preliminary 2025 total revenue

FY25 gross margin 87% Unaudited preliminary full-year 2025 gross margin

FY25 operating expenses €762 million Unaudited preliminary full-year 2025 operating expenses

Year-end 2025 cash €616 million Unaudited preliminary cash balance as of Dec 31, 2025

Share repurchase program $120 million Planned 2026 share repurchase authorization

Market Reality Check

$202.99 Last Close

Volume Volume 839,619 vs 20-day avg 670,040 (relative volume 1.25x) shows elevated trading interest ahead of the update. normal

Technical Price $202.99 is above 200-day MA $185.71 and about 11.72% below the 52-week high.

Peers on Argus

ASND fell 2.76% while key biotech peers were mixed: EXEL -0.54%, IONS -2.28%, BMRN -2.59% vs SMMT +3.58% and BBIO +3.72%, indicating a stock-specific move rather than a uniform sector reaction.

Historical Context

Date	Event	Sentiment	Move	Catalyst
Jan 05	Conference participation	Neutral	-0.1%	Simple notice of participation at the J.P. Morgan Healthcare Conference.
Nov 25	Regulatory delay	Negative	-1.1%	FDA classed new TransCon CNP data as major amendment, extending PDUFA date.
Nov 17	Pivotal trial results	Positive	+0.3%	JAMA Pediatrics publication showing improved growth and safety for TransCon CNP.
Nov 12	Earnings update	Positive	-1.0%	Q3 2025 revenue surge and operating profit but continued net loss and finance expense.
Nov 07	Clinical data update	Positive	-0.8%	Three-year pooled data showing sustained renal function gains with TransCon PTH.

Pattern Detected

Recent news often saw modest negative reactions, including some divergence where positive clinical or financial updates were followed by share price declines.

Recent Company History

Over the last few months, Ascendis has reported pivotal TransCon CNP trial success, strong Q3 2025 revenue growth, and incremental regulatory updates, including a PDUFA extension to February 28, 2026. Several of these were objectively positive milestones yet drew muted or slightly negative price moves. Today’s broad strategic roadmap, preliminary 2025 financials, and capital return plans build on that trajectory by highlighting commercial scale-up, late-stage regulatory catalysts and expanding indications across the TransCon platform.

Market Pulse Summary

This announcement outlines Ascendis’ transition into a broader biopharma platform, with preliminary 2025 product revenue of about €683 million, gross margin near 87%, and multiple late-stage TransCon programs approaching key decisions. It also adds a planned $120 million share repurchase and projected €500 million operating cash flow in 2026. Investors may watch upcoming PDUFA decisions, Phase 3 trial starts, and execution on global launches to assess how this roadmap translates into sustainable profitability.

Key Terms

PDUFA regulatory

"In the U.S., PDUFA goal date of February 28, 2026 for pediatric achondroplasia."

PDUFA, short for the Prescription Drug User Fee Act, is a law that allows drug companies to pay fees to the government to speed up the review process for new medicines. This helps bring important drugs to market more quickly, which can impact their availability and pricing. For investors, PDUFA timelines can influence the timing of a drug’s approval and potential market success.

marketing authorization application (MAA) regulatory

"Submitted marketing authorization application (MAA) to the European Medicines Agency (EMA)..."

A marketing authorization application (MAA) is a formal request submitted to a health regulator asking permission to sell a medicine or medical product in a market. Think of it like applying for a driver's license for a new drug: the regulator checks safety, quality and effectiveness before granting permission. For investors, the MAA stage matters because approval typically unlocks commercial sales and revenue, while rejection or delay creates major value and timing risk.

Biologic License Application (BLA) regulatory

"Biologic License Application (BLA) for TransCon hGH ... was accepted by China’s National Medical Products Administration..."

A Biologic License Application (BLA) is a formal request to the U.S. Food and Drug Administration to approve a biological product—such as vaccines, antibodies, or cell therapies—for commercial sale. It bundles clinical trial results, manufacturing details and labeling so regulators can judge safety and effectiveness; for investors, BLA approval is like a company earning the right to sell a complex, highly regulated product, often unlocking revenue, market access and reduced regulatory risk.

named patient programs regulatory

"Outside the U.S., now available commercially or through named patient programs in more than 30 countries..."

Named patient programs are arrangements that allow patients to access experimental or unapproved medicines outside of regular clinical trials, often through special approvals. They matter to investors because they can signal a company's efforts to bring new treatments to market and may impact perceptions of future sales potential or regulatory progress. These programs can also influence a company's reputation and relationships within the healthcare industry.

Phase 2 medical

"Week 52 topline results from Phase 2 COACH Trial, which demonstrated improvements..."

Phase 2 is the mid-stage clinical trial where a new drug or treatment is tested in a larger group of patients to see if it works and to keep checking safety after initial human testing. Think of it as a field test that proves whether a product actually delivers its promised benefit. Investors watch Phase 2 closely because its results strongly influence a medicine’s chances of reaching the market, the size of its potential sales, and the company’s valuation.

Phase 3 medical

"regarding a Phase 3 trial of TransCon CNP and TransCon hGH in pediatric achondroplasia."

Phase 3 is the late-stage clinical testing step for a new drug or medical treatment, where the product is given to large groups of patients to confirm effectiveness, monitor side effects, and compare it to standard care. Successful Phase 3 results are often the final scientific hurdle before regulators decide on approval and market launch—like passing a final exam before graduation—and can sharply change a company's valuation and future revenue prospects.

overall survival (OS) medical

"Expect to report median overall survival (OS) data for a cohort of 70 patients..."

Overall survival (OS) is the length of time from the start of a treatment or clinical study until death from any cause, essentially measuring how long patients live after a therapy begins. Investors watch OS because it is the most direct evidence a treatment extends life; stronger OS results can drive regulatory approvals, wider use and higher revenue expectations, much like sales figures proving a product actually works.

share repurchase program financial

"The Board of Directors of Ascendis has authorized a $120 million Share Repurchase Program."

A share repurchase program is when a company buys back its own shares from the marketplace. This reduces the total number of shares available, which can increase the value of each remaining share and signal confidence in the company's prospects. For investors, it often suggests that the company believes its stock is undervalued or that it has extra cash to return to shareholders.

AI-generated analysis. Not financial advice.

- Rapidly transforming into a leading global biopharma company

COPENHAGEN, Denmark, Jan. 09, 2026 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today provided 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 44^th Annual J.P. Morgan Healthcare Conference.

“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 97^th 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 https://investors.ascendispharma.com. 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 https://investors.ascendispharma.com.

About Ascendis Pharma A/S
Ascendis Pharma is applying its innovative TransCon technology platform to build a leading, fully integrated biopharma company focused on making a meaningful difference in patients’ lives. Guided by its core values of Patients, Science, and Passion, Ascendis uses its TransCon technologies to create new and potentially best-in-class therapies. Ascendis is headquartered in Copenhagen, Denmark and has additional facilities in Europe and the United States. Please visit ascendispharma.com to learn more.

Forward-Looking Statements
This press release contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, included in this press release regarding Ascendis’ future operations, plans and objectives of management are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to (i) Ascendis’ potential to rapidly transform into a leading global biopharma company, (ii) anticipated commercial launches of TransCon PTH into additional countries, (iii) the PDUFA goal date of TransCon CNP for pediatric achondroplasia and EMA regulatory decision date on the potential use of TransCon CNP in pediatric achondroplasia, (iv) timing of week 78 COACH data and planned new trials to support TransCon CNP and TransCon hGH treatment in additional indications, (v) planned release of data from the IL-Believe Trial of TransCon IL-2 β/γ, (vi) clinical status of TransCon aVEGF, (vii) NMPA’s anticipated decision of TransCon hGH as a potential treatment for PGHD, (viii) the expected collaboration with Novo Nordisk and progress of TransCon semaglutide, (ix) expected full year 2025 SKYTROFA, YORVIPATH and total revenues, operating expense, cash balance and operating cash flow, (x) planned share repurchase program, (xi) Ascendis’ ability to apply its TransCon technology platform to build a leading, fully integrated biopharma company, and (xii) Ascendis’ use of its TransCon technologies to create new and potentially best-in-class therapies. Ascendis may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions, expectations and projections disclosed in the forward-looking statements. Various important factors could cause actual results or events to differ materially from the forward-looking statements that Ascendis makes, including, without limitation: dependence on third‑party manufacturers, distributors, and service providers for Ascendis’ products and product candidates; risks related to regulatory review and approval, including the possibility of delays, requests for additional data or analyses, restrictions or limitations on use, approval with labeling that is more limited than expected, or failure to obtain approval in the United States, European Union, or other jurisdictions; clinical development risks, including that results from ongoing or future trials may not confirm earlier data; unforeseen safety or efficacy findings in development programs or on‑market products; manufacturing, supply chain, quality, or logistics issues that could delay development or commercialization; unforeseen expenses related to commercialization of any approved Ascendis products; unforeseen research and development or selling, general and administrative expenses and other costs impacting Ascendis’ business generally; market acceptance, pricing, and reimbursement challenges, including payer coverage decisions and health technology assessments; competitive developments, including new or improved therapies; intellectual property protection, freedom‑to‑operate, and litigation risks; Ascendis’ ability to obtain additional funding, if needed, to support its business activities; cybersecurity, data privacy, and information technology disruptions; and the impact of international economic, political, legal, compliance, public health, and business factors, including tariffs, trade policies, currency fluctuations, and geopolitical events. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to Ascendis’ business in general, see Ascendis’ current and future reports filed with, or submitted to, the U.S. Securities and Exchange Commission (SEC), including its Annual Report on Form 20-F filed with the SEC on February 12, 2025. Forward-looking statements do not reflect the potential impact of any future licensing, collaborations, acquisitions, mergers, dispositions, joint ventures, or investments that Ascendis may enter into or make. Ascendis does not assume any obligation to update any forward-looking statements, except as required by law.

Ascendis, Ascendis Pharma, the Ascendis Pharma logo, the company logo, TransCon, SKYTROFA^®, and YORVIPATH^® are trademarks owned by the Ascendis Pharma group. © January 2026 Ascendis Pharma A/S.

Investor Contacts: Chad Fugere Ascendis Pharma ir@ascendispharma.com	Media Contact: Melinda Baker Ascendis Pharma media@ascendispharma.com
Patti Bank ICR Healthcare +1 (415) 513-1284 patti.bank@icrhealthcare.com

FAQ

What 2025 revenue did Ascendis Pharma (ASND) report for YORVIPATH?

YORVIPATH full-year 2025 revenue ~€477 million (unaudited preliminary).

How much cash did Ascendis (ASND) hold at December 31, 2025?

Unaudited preliminary cash balance ~€616 million as of Dec 31, 2025.

What is Ascendis Pharma’s (ASND) share repurchase authorization for 2026?

The Board authorized a $120 million share repurchase program in 2026.

When is the TransCon CNP (navepegritide) PDUFA date for ASND in the U.S.?

The PDUFA goal date is February 28, 2026 for pediatric achondroplasia.

What 2025 operating expense level did Ascendis (ASND) report?

Total full-year 2025 operating expenses were ~€762 million (unaudited preliminary).