Ascendis Pharma Business and Strategic Roadmap Update at J.P. Morgan Healthcare

 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 43rd 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.

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