Ascendis Pharma (ASND) Morgan Stanley 24th Annual Global Healthcare Conference summary

Strategic and financial updates

• Announced $400 million share repurchase, reflecting strong financial transformation and confidence in future cash flows, with no plans to raise additional capital.

• Vision 2030 targets €5 billion in product revenue, driven by three approved products and global expansion, with a focus on both U.S. and ex-U.S. markets.

• Revenue recognized in over 40 countries, with plans to add 10–20 new fully commercial countries annually through 2028.

• Operating cash flow expected to exceed €500 million, potentially doubling next year due to successful product launches.

• No material increase in R&D expenses anticipated; revenue growth expected to outpace expense growth significantly.

Product portfolio and commercialization

• Three approved products—SKYTROFA, YORVIPATH, and YUVIWEL—are the foundation for revenue growth, with ongoing label expansions and late-stage trials.

• YORVIPATH is achieving blockbuster status, with investments in prescriber education, patient activation, and digital infrastructure to accelerate U.S. growth.

• Ex-U.S. commercialization is expanding rapidly, using a mixed model of direct presence and distribution agreements to maximize reach.

• YUVIWEL launch is outperforming previous analogs, with strong uptake across new starts, switches, and previously untreated patients.

• SKYTROFA continues to grow and serves as a platform for future development and combination therapies.

Pipeline and future growth

• At least one new chemical entity (NCE) will enter clinical development annually, expanding beyond rare disease endocrinology into new therapeutic areas.

• Combination therapies, such as YUVIWEL with SKYTROFA, show promising results, including significant improvements in achondroplasia outcomes.

• Upcoming clinical milestones include two to three phase III readouts next year and continued data releases from ongoing trials.

• Once-weekly TransCon PTH is in development to provide more options for stable patients, aiming for bioequivalence with daily therapy.

• Cardiometabolic franchise regained, with plans for phase I/II trials and no need for additional capital to fund expansion.