Ascendis Pharma Provides Business and Strategic Plan Update at 43rd Annual JP Morgan Healthcare By Investing.com Conference

– Ascendis is positioned to drive rapid revenue growth

COPENHAGEN, Denmark, Jan. 12, 2025 (GLOBE NEWSWIRE) — Ascendis Pharma A/S (Nasdaq: NASDAQ: ) today provided an updated business and strategic roadmap, including planned key corporate milestones for 2025. Ascendis President and CEO Jan Mikkelsen will present this update tomorrow, January 13, during the 43rd Annual JP Morgan Healthcare Conference.

Ascendis is well positioned for rapid revenue growth with the launch of our first two endocrinology drugs for rare diseases, SKYTROFA and YORVIPATH, and, following our pre-NDA meeting, a filing is planned for our third, TransCon CNP for achondroplasia, said Jan Mikkelsen, Ascendis President of Pharma and Chief Executive Officer. Our growing TransCon technology platform and partnerships demonstrate that our business model built on rapid, successful drug development can create significant value for stakeholders.

Selected key updates and milestones

• TransCon™ hGH
  (lonapegsomatropin, sold as SKYTROFA®)
  • SKYTROFA’s full-year 2024 revenue, with one indication in pediatric GHD, is expected to be ~€202 million excluding prior-year sales deductions (based on SKYTROFA’s full-year unaudited preliminary revenue estimate 2024 of ~€197 million plus ~€5 million sales deductions related to previous years).
  • SKYTROFA volume in the US (mg) grew 84% in 2024 compared to the previous year, resulting in an estimated 6.5% market share of the total US growth hormone market in 2024 (based on third-party prescription data ).
  • Prescription Drug User Fee Act (PDUFA) July 27, 2025 target date for FDA review of supplemental BLA for treatment of adults with growth hormone deficiency; pending approval, commercial launch in the US is planned for 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, sold as YORVIPATH®)
  • The unaudited preliminary estimate of YORVIPATH’s revenue for the entire year 2024 is ~29 million euros.
  • YORVIPATH commercially available on prescription in Germany and Austria from January 2024. Outside Germany and Austria, provision of the product via early access routes, such as ˜named patient,’ until commercial reimbursement is established. ~700 patients on treatment in our Europe Direct and international markets at the end of 2024.
  • YORVIPATH commercially available by prescription from late December 2024 in the US As of January 9, 2025, 324 patients have enrolled in the Ascendis Signature Access Program or directly in specialty pharmacies, with more than half of prescriptions for patients new to YORVIPATH.
  • Expect a commercial launch in at least five additional Europe Direct countries in 2025.
• TransCon CNP
  (navepegritide)
  • After a pre-NDA meeting with the FDA, plan to submit a new drug application (NDA) for the treatment of children with achondroplasia during the first quarter of 2025 and submit a marketing application to the European Medicines Agency during the third quarter of 2025.
  • New data are presented showing significant improvements in leg flexion, a common complication of achondroplasia, seen with TransCon CNP compared to the worsening seen with placebo in the pivotal ApproaCH trial.
  • Topline Week 26 results from the Phase 2 COACH trial (TransCon CNP in combination with TransCon hGH) are expected in Q2 2025.
  • During the fourth quarter of 2025, plan to submit an IND or similar for the treatment of hypochondroplasia.
• Expansion of the TransCon platform and pipeline
  • A new TransCon protein degradation platform designed to enable efficient clearance of hormones, cytokines and other targets. The first planned TransCon protein degradation product designed to normalize excessive FGF-23 hormone levels for patients with X-linked hypophosphatemia.
• Financial update
  • Unaudited preliminary estimate of total product revenue for the entire year 2024 of ~ EUR 226 million:
    • SKYTROFA’s full year 2024 revenue is expected to be ~ EUR 202 million excluding prior year sales deductions (based on an unaudited preliminary full year 2024 SKYTROFA revenue estimate of ~ EUR 197 million plus ~ 5 million euros of sales deductions related to previous years).
    • YORVIPATH unaudited preliminary revenue estimate for the full year 2024
      of ~ 29 million euros.
  • Unaudited preliminary estimate of total full-year total revenue for 2024 of ~ EUR 364 million
    • Includes an upfront payment of $100 million from Novo Nordisk (NYSE: ) as non-product revenue.
  • Pro forma cash balance at 31 December 2024 of ~€655 million (based on unaudited preliminary estimate of 31 December 2024 cash balance of €560 million plus expected payment from Novo Nordisk of USD 100 million).

Presentation at the JP Morgan Healthcare conference on Monday 13 January
A live webcast of the event will be available through the Investors and News section of Ascendis Pharma’s website at https://investors.ascendispharma.com. The presentation will begin at 11:15 AM ET / 8:15 AM PT. The replay of the webcast will be available for 30 days.

The Company’s slides from the JP 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 biopharmaceutical company focused on making a meaningful difference in patients’ lives. Driven 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. Visit ascendispharma.com to learn more.

Forward-looking statements
This press release contains forward-looking statements that involve significant risks and uncertainties. All statements, other than statements of historical fact, included in this press release regarding the future operations, plans and objectives of Ascendis management are forward-looking statements. Examples of such statements include, but are not limited to, statements regarding (i) Ascendis’ potential for rapid revenue growth, (ii) planned regulatory filings for TransCon CNP and TransCon hGH, (iii) the ability of Ascendis’ business model to create significant shareholder value, (iv) expected SKYTROFA, YORVIPATH and total revenues for the full year 2024, (v) PDUFA date of additional BLA for SKYTROFA for the treatment of adults with growth hormone deficiency and Ascendis’ plans for US commercial launch, if approved, (vi) Ascendis’ expectations regarding the commercial launch of YORVIPATH in additional Europe Direct countries, (vii) warm-up time results from the TransCon CNP Phase 2 COACH trial, (viii) Ascendis’ expectations in connection with the TransCon protein degrader platform and the first TransCon degrader product candidate, (ix) the expected payment from Novo Nordisk, (x) Ascendis’ ability to apply its TransCon technology platform to build leading, fully integrated biopharmaceutical companies, and (xi) Ascendis’ use of its TransCon technologies to create new and potentially best-in-class therapies. Ascendis may not actually achieve the plans, implement the intentions or meet the expectations or projections expressed in the forward-looking statements and you should not place undue reliance on such forward-looking statements. Actual results or events may differ materially from the plans, intentions, expectations and projections expressed in the forward-looking statements. Various important factors could cause actual results or events to differ materially from the forward-looking statements Ascendis makes, including the following: dependence on third-party manufacturers, distributors and service providers for Ascendis’ products and product candidates; unanticipated safety or efficacy in Ascendis’ development programs or marketed products; incidental costs associated with the commercialization of any approved Ascendis product; unforeseen expenses related to the development programs of Ascendis; unforeseen selling, general and administrative expenses, other research and development expenses and business of Ascendis in general; delays in the development of its programs related to manufacturing, regulatory requirements, the speed of patient recruitment or other unforeseen delays; Ascendis’ ability to obtain additional funding, if necessary, to support its business activities; the influence of international economic, political, legal factors, compliance factors, 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 generally, see Ascendis’ prospectus supplement filed on September 20, 2024 and Current and Ascendis’ future reports filed with or filed with the US Securities and Exchange Commission (SEC), including its Annual Report on Form 20-F filed with the SEC February 7, 2024 Forward-looking statements do not reflect the potential effect of any future licensing, collaboration, acquisition, merger, disposition, joint venture or investment that Ascendis may enter into or make. Ascendis undertakes no obligation to update any forward-looking statements, except as required by law.

Ascendis, Ascendis Pharma, Ascendis Pharma logo, company logo, TransCon, SKYTROFA ®,
and YORVIPATH ® are trademarks owned by the Ascendis Pharma group. © January 2025 Ascendis Pharma A/S.

Source: Ascendis Pharma