Ascendis Pharma Introduces Vision 2030

• Strategic roadmap to achieve blockbuster status for multiple products and expand
  the Company’s engine for future innovation
  
  

COPENHAGEN, Denmark, Jan. 07, 2024 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today introduced selected 2024 corporate milestones and Vision 2030, its strategic roadmap through 2030. Ascendis President and CEO Jan Mikkelsen will present the update tomorrow, January 8, at the 42^nd Annual J.P. Morgan Healthcare Conference.

“With unwavering focus on our values of Patients, Science, and Passion, we remain on track to fulfill Vision 3x3, with the approval already of two Endocrinology Rare Disease products and value leadership in the U.S. growth hormone market for SKYTROFA,” said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. “Today we are introducing Vision 2030, our strategic roadmap to attain blockbuster status for multiple products and expand our engine for future innovation.”

Selected Key Updates

• TransCon hGH
  (lonapegsomatropin, approved as SKYTROFA in the U.S. and EU)
  • Following positive topline results from Phase 3 foresiGHt Trial in adult growth hormone deficiency (GHD), plan to submit a supplemental Biologics License Application to FDA in the second quarter of 2024.
  • Topline results from Phase 2 trial in Turner syndrome expected in the fourth quarter of 2024.
  • Attained U.S. market value leadership in 2023.
  • SKYTROFA preliminary unaudited fourth quarter 2023 revenue is expected to be approximately €64 million.  
  • Full year 2024 SKYTROFA revenue expected to be €320 million to €340 million (based on average 2023 exchange rates).
• TransCon PTH
  (palopegteriparatide, approved as YORVIPATH in the EU)
  • First launch planned in Germany in January 2024, leveraging the Company’s existing commercial infrastructure.
  • FDA regulatory review for the treatment of adult patients with hypoparathyroidism continues, with a Prescription Drug User Fee Act (PDUFA) date of May 14, 2024; if approved, U.S. commercial launch planned in the third quarter of 2024.
• TransCon CNP
  (navepegritide)
  • Treatment with TransCon CNP at 100 mg/kg/week for one year in all 57 children with achondroplasia (age 2 to 10 years) in ACcomplisH, demonstrated growth consistent with results from the blinded period and achieved improvements in health-related quality of life and disease impacts assessed with SF-10 and Achondroplasia Child Experience Measure.
  • During the third quarter of 2023, submitted Clinical Trial Application (CTA) for infant trial (age 0-2 years).
  • Topline results from the pivotal ApproaCH Trial expected in the fourth quarter of 2024.
  • During the fourth quarter of 2024, plan to submit a New Drug Application to FDA for children with achondroplasia (age 2-11 years).
  • Week 26 topline data from the COACH Trial (TransCon hGH/TransCon CNP combination) expected in the fourth quarter of 2024.
  • During the fourth quarter of 2024, plan to submit an Investigational New Drug application or similar in adults with achondroplasia.
• Global commercial presence in Endocrinology Rare Disease
  • During the fourth quarter of 2023, entered into an exclusive license agreement with Teijin Limited for TransCon hGH, TransCon PTH, and TransCon CNP in Japan. Phase 3 PaTHway Japan Trial of TransCon PTH completed, and Phase 3 riGHt Trial of TransCon hGH fully enrolled.
  • VISEN’s TransCon hGH Phase 3 and TransCon CNP Phase 2 trials in China completed.
  • Expanding global reach through exclusive sales & distribution agreements with geographic market leaders in International Markets, with three regional agreements signed to date: Specialised Therapeutics Asia Pte Ltd, Er-Kim, and Vector Pharma FZCO.
• Oncology
  • During the fourth quarter of 2024, expect to complete enrollment in BelieveIT-201, a Phase 2 trial in advanced head and neck squamous cell carcinoma (HNSCC).
  • During the fourth quarter of 2024, plan to provide a clinical update from the Phase 2 portion of indication-specific, dose expansion cohorts in the IL-Believe Trial.
• Ophthalmology
  • Creation of Ophthalmology NewCo, financed by institutional investors, expected during the first quarter of 2024.
• Financial Update and Outlook Based on Current Plans
  • Preliminary unaudited December 31, 2023 cash, cash equivalents, and marketable securities of ~€400 million.
  • Full year 2024 SKYTROFA revenue expected to be €320 million to €340 million (based on average 2023 exchange rates).
  • Expect to provide YORVIPATH revenue update during 2024.
  • Expect total operating expenses (SG&A and R&D) of approximately €600 million for 2024.
  • Expect to be operating cash flow breakeven on a quarterly basis by the end of 2024.