Ascendis Pharma A/S Q4 FY2024 earnings call

• SKYTROFA is firmly established as a high-value growth hormone brand with volume growth, and plans to expand its addressable market with adult growth hormone deficiency approval expected in 2024 and basket trial IND submission in Q3.
• YORVIPATH, the FDA-approved hypothyroidism treatment, launched in the U.S. with early demand and positive payer progress, expected to be a multi-billion-dollar product.
• TransCon CNP has promising clinical data for achondroplasia, on track for NDA submission to FDA in Q1 2025 and MAA to EMEA in Q3 2025, with combination treatment studies in progress.
• Expanded TransCon technology platform beyond endocrine rare diseases, including collaborations with Novo Nordisk in metabolic diseases and creation of Eyconis in ophthalmology, and incorporation of protein degraders.
• Strong financial position with €665 million cash on balance sheet, including $100 million upfront from Novo Nordisk.

SKYTROFA: In 2024, revenue reached around €200 million, with a 6.5% market share of the total growth hormone market in the U.S. and ~45% of the total U.S. long-acting growth hormone market based on third-party prescription data. In Q4 2024, SKYTROFA volume increased 37% compared to Q4 2023, with reported revenue of €58.5 million. YORVIPATH: Launched in the U.S. and has significant early demand; 2024 revenue was €28.7 million, with Q4 revenue at €13.6 million. TransCon CNP: Clinical data shows potential for achondroplasia treatment, on track to submit NDA to FDA this quarter and MAA to EMEA in Q3, with once-weekly data demonstrating superior linear growth and other benefits.

• YORVIPATH is expected to have a significant impact on financial profile in 2025, with plans to share launch parameters in coming quarters.
• SKYTROFA revenue growth expected to track closer to script growth unless substantial payer mix changes occur.
• Plan to provide revenue guidance for YORVIPATH when confidence in numbers is achieved.

• Uncertainty in insurance approvals for YORVIPATH, with initial estimates of 4-8 weeks but early launch phase may affect accuracy.
• Potential competition from other therapies with pivotal data expected in 2024.
• Challenges in commercial uptake and reimbursement for new products like YORVIPATH, especially with varying payer policies.

Q: Can you confirm if the 908 YORVIPATH figure is unique patients and not cumulative scripts?

A: Yes, it is unique patient enrollments.

Q: What proportion of EAP and OLE rollover patients are in the 908 number?

A: About 20% of the 908 are existing patients from TransCon PTH, EAP or clinical trials, 80% are new to YORVIPATH.

Q: How have discussions with payers gone for YORVIPATH and assumptions on prior authorization criteria?

A: Conversations are productive, focusing on clinical value proposition, and prior authorization criteria are expected to align with label.

Q: What's the expectation for CNP plus growth hormone combo data on AGV?

A: Expectation is for a good result with synergistic effect, likely reflecting improved linear growth and other benefits.

Q: What proportion of 900+ YORVIPATH prescriptions are severe vs moderate?

A: Data on patient severity is limited, but most severe patients see endos more frequently, likely including some of the early prescriptions.

Q: What type of docs are prescribing YORVIPATH and conversion from EAP to paid drugs?

A: Prescribers are broad across 44 states, over 50% reach for priority physicians; EAP patients converting to paid drugs starting in Feb and majority in Mar.

Q: Thoughts on U.S. YORVIPATH script cadence and reimbursement trends?

A: No clear data on script cadence trends yet, reimbursement approvals across payers but still early with varying policies.

Q: Thoughts on competing pivotal data for YORVIPATH and market evolution?

A: Awaiting results of other trials, but TransCon technology provides unique differentiation not seen in other compounds.

Q: Guidance on YORVIPATH revenue and CNP IND run-in activities?

A: No specific revenue guidance yet, working on optimal treatment design for hypochondroplasia including IND submission later in 2025.