Ascendis Pharma (ASND) 43rd Annual J.P. Morgan Healthcare Conference 2025 summary

Strategic Vision and Technology Platform

• Aims to lead rare endocrine diseases with three independent blockbuster products by 2030, leveraging TransCon technology and product innovation algorithm.

• Expanding TransCon technology into oncology, metabolic, cardiovascular, and ophthalmology, with partnerships including Novo Nordisk and Eyconis.

• Pursuing accelerated approval in oncology and broadening the pipeline with new chemical entities and protein degrader platforms.

• Strategic roadmap includes global commercialization through direct sales, partnerships, and exclusive distribution agreements in over 50 countries.

• Emphasizes rapid product development, achieving three product approvals from preclinical stage in seven years.

Product Performance and Market Expansion

• Skytrofa generated over EUR 202 million in 2023, with 2024 revenue expected at ~€202 million, mainly from the U.S., and label expansion for adult GHD pending FDA decision by July 27, 2025.

• Yorvipath launched in Germany, Austria, and U.S., generating EUR 29 million in 2023, with 2024 revenue expected at ~€29 million, rapid patient uptake, and plans to expand to at least five more European countries in 2025.

• Yorvipath achieved in 10 months what Natpara did in four years in Europe, with 700 patients on treatment and 150 unique prescribers in the U.S.

• TransCon CNP pivotal phase III trial met primary endpoint in pediatric achondroplasia; NDA filing planned for U.S. in Q1 2025 and EU in Q3 2025.

• Ongoing trials for label expansion in growth hormone and CNP, including basket and cross trials to address broader patient populations.

Financial Outlook and Guidance

• 2024 total product revenue expected at ~€226 million, with total revenue (including Novo Nordisk milestone) at ~€364 million; cash position at ~€655 million, including €100 million from Novo Nordisk.

• Confident in achieving cash flow break-even, potentially by the end of 2025, depending on Yorvipath's trajectory.

• Major revenue drivers for 2025 expected from research and development milestones, especially CNP.

• German reference pricing for Yorvipath expected to finalize mid-year, likely between EUR 80,000 and EUR 105,000 per patient annually.

• Plan to remain well-capitalized through cash flow breakeven and beyond, with stable R&D investment as portfolio matures.