Ascendis Pharma A/S has provided a comprehensive update on its business and strategic roadmap for 2025, highlighting key milestones and growth opportunities. This update was presented by Jan Mikkelsen, President and CEO, at the 43rd Annual J.P. Morgan Healthcare Conference. The company outlined its advancements in endocrinology rare disease therapies, leveraging its TransCon™ technology platform to achieve rapid drug development and significant value creation for stakeholders.
Ascendis Pharma Strategic Vision and Growth Opportunities
Ascendis Pharma’s strategy centers on its innovative TransCon™ technology platform and its three lead programs: SKYTROFA® (lonapegsomatropin), YORVIPATH® (palopegteriparatide), and TransCon CNP (navepegritide). The company’s approach aims to unlock the full potential of its pipeline while addressing critical unmet needs in endocrinology and rare diseases.
“Ascendis Pharma With the successful launch of SKYTROFA and YORVIPATH and the planned regulatory submissions for TransCon CNP, we are positioned for substantial growth,” said Mikkelsen. “Our expanding TransCon technology platform continues to demonstrate its capability to deliver innovative therapies that address significant medical needs, driving value for patients and stakeholders alike.”
Key Updates and Milestones
TransCon hGH (SKYTROFA®)
SKYTROFA, the first product approved from Ascendis’ TransCon platform, has demonstrated robust growth:
- Revenue Growth: SKYTROFA’s full-year 2024 revenue is estimated at approximately €202 million (excluding prior-year sales deductions). This includes a preliminary revenue estimate of €197 million plus €5 million in sales adjustments.
- Market Penetration: U.S. SKYTROFA volume increased by 84% year-over-year in 2024, capturing an estimated 6.5% of the total U.S. growth hormone market based on third-party prescription data.
- Pipeline Expansion: Ascendis expects a Prescription Drug User Fee Act (PDUFA) goal date of July 27, 2025, for FDA review of its supplemental Biologics License Application (sBLA) for adult growth hormone deficiency treatment. Pending approval, a U.S. commercial launch is planned for Q4 2025. Additionally, the company plans to submit an Investigational New Drug (IND) application in Q3 2025 for evaluating TransCon hGH in additional indications.
TransCon PTH (YORVIPATH®)
YORVIPATH, targeting hypoparathyroidism, continues its commercial rollout:
- Revenue and Market Access: Unaudited preliminary revenue for 2024 is estimated at approximately €29 million. YORVIPATH became commercially available in Germany and Austria in early 2024, with early access provided in other markets via named-patient programs. By the end of 2024, approximately 700 patients were receiving treatment in Europe.
- U.S. Launch: Commercial availability began in late December 2024. As of January 9, 2025, 324 patients have been enrolled in the Ascendis Signature Access Program or connected with specialty pharmacies, with over half being new to YORVIPATH. The company aims to expand its commercial footprint to at least five additional European countries in 2025.
TransCon CNP (navepegritide)
TransCon CNP, developed for achondroplasia, achieved significant milestones:
- Regulatory Progress: Following a pre-New Drug Application (NDA) meeting with the FDA, Ascendis plans to submit the NDA in Q1 2025. A Marketing Authorization Application (MAA) submission to the European Medicines Agency is expected in Q3 2025.
- Clinical Data: New data from the pivotal ApproaCH trial demonstrated significant improvements in leg bowing, a common achondroplasia complication, compared to placebo. The Phase 2 COACH trial, evaluating TransCon CNP in combination with TransCon hGH, is expected to deliver topline results by Q2 2025.
- Pipeline Expansion: The company plans to submit an IND for the treatment of hypochondroplasia in Q4 2025.
Expanding the TransCon Platform
Ascendis is leveraging its TransCon technology to develop innovative therapies across various areas:
- Protein Degrader Platform: A new TransCon protein degrader platform aims to enable efficient clearance of hormones and cytokines. The first product candidate targets excess FGF-23 hormone levels in X-linked hypophosphatemia.
- Pipeline Development: The platform’s versatility continues to open opportunities for addressing complex diseases, with a focus on long-term innovation.
Financial Highlights
Ascendis Pharma reported strong financial performance, reflecting its growth trajectory:
- Revenue: Total unaudited preliminary product revenue for 2024 is estimated at approximately €226 million, comprising €202 million from SKYTROFA and €29 million from YORVIPATH. Non-product revenue includes a $100 million milestone payment from Novo Nordisk.
- Cash Position: As of December 31, 2024, the company’s pro forma cash balance is estimated at €655 million, including the Novo Nordisk milestone payment.
2025 Corporate Goals
Ascendis Pharma’s objectives for 2025 focus on growth and innovation:
- Commercial Expansion: Increase market penetration for SKYTROFA and YORVIPATH in existing and new territories.
- Regulatory Milestones: Secure approvals for TransCon CNP and expand its use in achondroplasia treatment.
- Pipeline Advancements: Initiate new trials for TransCon hGH and TransCon CNP in additional indications.
- Platform Innovation: Advance the TransCon protein degrader program and nominate new candidates for clinical development.
J.P. Morgan Healthcare Conference
Jan Mikkelsen’s presentation at the 43rd Annual J.P. Morgan Healthcare Conference will provide a detailed overview of Ascendis’ achievements and future plans. The live webcast is scheduled for January 13, 2025, at 11:15 a.m. ET and will be accessible via the company’s website. A replay will be available for 30 days.
