Ascendis Pharma Provides Business and Strategic Roadmap Update at 43rd Annual J.P. Morgan Healthcare Conference

Rhea-AI Summary

Ascendis Pharma (ASND) provided a business update highlighting strong revenue growth from its two marketed Endocrinology Rare Disease medicines. SKYTROFA achieved ~€202 million in full-year 2024 revenue, with an 84% year-over-year volume increase and 6.5% U.S. market share. YORVIPATH generated ~€29 million in 2024 revenue, with ~700 patients in Europe and 324 patients enrolled in the U.S. program.

The company plans to submit an NDA for TransCon CNP for achondroplasia in Q1 2025 and expand to additional European markets. Total preliminary 2024 revenue is estimated at ~€364 million, including a $100 million Novo Nordisk milestone payment. The company reported a pro forma cash balance of ~€655 million as of December 31, 2024.

Positive

• SKYTROFA revenue reached ~€202 million in 2024 with 84% volume growth
• YORVIPATH launched successfully with ~€29 million revenue in 2024
• Total preliminary 2024 revenue of ~€364 million
• Strong cash position of ~€655 million (pro forma)
• Received $100 million milestone payment from Novo Nordisk

Negative

• None.

Insights

Financial Analyst

The financial performance metrics reveal robust growth trajectory. SKYTROFA generated €202 million in 2024 revenue with an impressive 84% year-over-year volume increase, capturing 6.5% market share. YORVIPATH contributed €29 million, bringing total product revenue to €226 million. Including the $100 million Novo Nordisk milestone payment, total revenue reached €364 million.

The pro forma cash position of €655 million provides substantial runway for commercial expansion and pipeline development. Market penetration metrics for both commercialized products show strong adoption rates, with YORVIPATH gaining traction in Europe and early U.S. launch momentum with 324 patients enrolled in just two weeks.

Medical Research Analyst

The pipeline progression demonstrates significant clinical advancement. TransCon CNP's planned NDA submission in Q1 2025 for achondroplasia treatment, supported by positive leg bowing data, represents a potential third commercial asset. The expansion into hypochondroplasia and the development of a novel TransCon protein degrader platform targeting FGF-23 for X-linked hypophosphatemia showcases strategic diversification in rare diseases.

The upcoming COACH Trial results combining TransCon CNP with TransCon hGH could potentially establish a new treatment paradigm. The basket trial strategy for TransCon hGH indicates potential label expansion opportunities beyond current indications.

Market Research Analyst

The commercial execution strategy shows strong market penetration potential. SKYTROFA's growth in pediatric GHD demonstrates successful market adoption, while YORVIPATH's rapid patient enrollment in the U.S. suggests strong market demand. The planned expansion to five additional European countries in 2025 indicates significant market opportunity.

The company's TransCon technology platform continues to demonstrate versatility and scalability across multiple therapeutic areas. The partnership with Novo Nordisk validates the platform's value proposition and potential for additional collaborations.

- Ascendis positioned to drive rapid revenue growth

COPENHAGEN, Denmark, Jan. 12, 2025 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today provided a business and strategic roadmap update, including planned 2025 key corporate milestones. Ascendis President and CEO Jan Mikkelsen will present this update tomorrow, January 13, during the 43^rd Annual J.P. Morgan Healthcare Conference.

“Ascendis is well-positioned for rapid revenue growth with the launch of our first two Endocrinology Rare Disease medicines, SKYTROFA and YORVIPATH, and, following our pre-NDA meeting, planned filings for our third, TransCon CNP for achondroplasia,” said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. “Our expanding TransCon technology platform and partnerships demonstrate that our business model built on fast, successful drug development can create substantial stakeholder value.”

Selected Key Updates and Milestones

• TransCon^™ hGH
  (lonapegsomatropin, marketed as SKYTROFA^®)
  • SKYTROFA full-year 2024 revenue, with a single indication in pediatric GHD, expected to be ~€202 million excluding sales deductions related to prior years (based on unaudited preliminary estimate of full-year 2024 SKYTROFA revenue of ~€197 million plus ~€5 million of sales deductions related to prior years).
  • U.S. SKYTROFA volume (mg) increased 84% in 2024 year-over-year resulting in an estimated 6.5% market share of the total U.S. growth hormone market for 2024 (based on third party prescription data).
  • Prescription Drug User Fee Act (PDUFA) goal date of July 27, 2025 for FDA review of supplemental BLA for the treatment of adults with growth hormone deficiency; pending approval, U.S. commercial launch planned in the fourth quarter of 2025.
  • During the third quarter of 2025, plan to submit an Investigational New Drug (IND) application or similar for a basket trial evaluating TransCon hGH in additional indications.
• TransCon PTH
  (palopegteriparatide, marketed as YORVIPATH^®)
  • YORVIPATH full-year 2024 unaudited preliminary revenue estimate of ~€29 million.
  • YORVIPATH commercially available for prescription in Germany and Austria since January 2024. Outside Germany and Austria, providing product through early access routes, such as ‘named patient,’ until commercial reimbursement established. ~700 patients on treatment in our Europe Direct and International Markets at the end of 2024.
  • YORVIPATH commercially available for prescription since late December 2024 in the U.S. As of January 9, 2025, 324 patients enrolled into the Ascendis Signature Access Program or direct with specialty pharmacy, with over half of prescriptions for patients new to YORVIPATH.
  • Expect commercial launch in at least five additional Europe Direct countries in 2025.
• TransCon CNP
  (navepegritide)
  • Following pre-NDA meeting with FDA, plan to submit New Drug Application (NDA) for the treatment of children with achondroplasia during the first quarter of 2025, and submit Marketing Authorisation Application to the European Medicines Agency during the third quarter of 2025.
  • Presented new data demonstrating significant improvements in leg bowing, a common complication in achondroplasia, observed with TransCon CNP compared to worsening observed with placebo in pivotal ApproaCH Trial.
  • Topline Week 26 results from Phase 2 COACH Trial (TransCon CNP in combination with TransCon hGH) expected in the second quarter of 2025.
  • During the fourth quarter of 2025, plan to submit an IND or similar for the treatment of hypochondroplasia.
• Expanding the TransCon Platform & Pipeline
  • New TransCon protein degrader platform designed to enable efficient clearance of hormones, cytokines, and other targets. First planned TransCon protein degrader product candidate designed to normalize excess FGF-23 hormone levels for patients with X-linked hypophosphatemia.
• Financial Update
  • Unaudited preliminary estimate of total full-year 2024 product revenue of ~€226 million:
    • SKYTROFA full-year 2024 revenue expected to be ~€202 million excluding sales deductions related to prior years (based on unaudited preliminary estimate of full-year 2024 SKYTROFA revenue of ~€197 million plus ~€5 million of sales deductions related to prior years).
    • YORVIPATH full-year 2024 unaudited preliminary revenue estimate
      of ~€29 million.
  • Unaudited preliminary estimate of total full-year total 2024 revenue of ~€364 million
    • Includes $100 million Novo Nordisk milestone payment as non-product revenue.
  • December 31, 2024 pro forma cash balance of ~€655 million (based on unaudited preliminary estimate of December 31, 2024 cash balance of €560 million plus expected payment from Novo Nordisk of $100 million).
    

Presentation at J.P. Morgan Healthcare Conference on Monday, January 13
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 11:15 a.m. Eastern Time / 8:15 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 Investor Relations 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 for rapid revenue growth, (ii) planned regulatory submissions for TransCon CNP and TransCon hGH, (iii) the ability of Ascendis’ business model to create substantial stakeholder value, (iv) expected full-year 2024 SKYTROFA, YORVIPATH and total revenues, (v) the PDUFA date of the supplemental BLA for SKYTROFA for the treatment of adults with growth hormone deficiency and Ascendis’ plans for a U.S. commercial launch, if approved, (vi) Ascendis’ expectations regarding the commercial launch of YORVIPATH in additional Europe Direct Countries, (vii) the timing of topline results from the Phase 2 COACH Trial of TransCon CNP, (viii) Ascendis’ expectations regarding its TransCon protein degrader platform and first TransCon degrader product candidate, (ix) the expected payment from Novo Nordisk, (x) Ascendis’ ability to apply its TransCon technology platform to build a leading, fully integrated biopharma company, and (xi) 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 the following: dependence on third party manufacturers, distributors and service providers for Ascendis’ products and product candidates; unforeseen safety or efficacy results in Ascendis’ development programs or on-market products; unforeseen expenses related to commercialization of any approved Ascendis products; unforeseen expenses related to Ascendis’ development programs; unforeseen selling, general and administrative expenses, other research and development expenses and Ascendis’ business generally; delays in the development of its programs related to manufacturing, regulatory requirements, speed of patient recruitment or other unforeseen delays; Ascendis’ ability to obtain additional funding, if needed, to support its business activities; the impact of international economic, political, legal, compliance, social and business factors. 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’ prospectus supplement filed on September 20, 2024 and 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 7, 2024. 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 2025 Ascendis Pharma A/S.

Investor Contacts:	Media Contact:
Scott Smith	Melinda Baker
Ascendis Pharma	Ascendis Pharma
ir@ascendispharma.com	+1 (650) 709-8875
	media@ascendispharma.com
Patti Bank
ICR Healthcare
+1 (415) 513-1284
patti.bank@icrhealthcare.com

FAQ

What was Ascendis Pharma's (ASND) total revenue for 2024?

Ascendis Pharma's preliminary total revenue for 2024 was ~€364 million, including ~€202 million from SKYTROFA, ~€29 million from YORVIPATH, and a $100 million milestone payment from Novo Nordisk.

What is SKYTROFA's market share in the U.S. growth hormone market as of 2024?

SKYTROFA achieved an estimated 6.5% market share of the total U.S. growth hormone market in 2024, with an 84% year-over-year volume increase.

When will Ascendis Pharma (ASND) submit the NDA for TransCon CNP?

Ascendis Pharma plans to submit the New Drug Application (NDA) for TransCon CNP for treating children with achondroplasia during the first quarter of 2025.

How many patients are currently on YORVIPATH treatment in Europe?

Approximately 700 patients were on YORVIPATH treatment in Ascendis Pharma's Europe Direct and International Markets at the end of 2024.

What is Ascendis Pharma's (ASND) cash position as of December 2024?

Ascendis Pharma reported a pro forma cash balance of approximately €655 million as of December 31, 2024, including the expected $100 million payment from Novo Nordisk.